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News Article | February 28, 2017
Site: globenewswire.com

OXFORD, United Kingdom, Feb. 28, 2017 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium difficile infection, recognises the tenth annual Rare Disease Day taking place today, 28 February 2017. The Rare Disease Day 2017 theme, ‘with research, possibilities are limitless,’ emphasises the importance of scientific research in helping to understand, diagnose and treat rare diseases that affect millions of people and their families worldwide. Summit seeks to remain at the forefront of utrophin modulation research through its strategic alliance with the University of Oxford, under the guidance of Professor Kay Davies. The collaboration is focussed on developing future-generation utrophin modulators for the potential treatment of all patients with the progressive muscle wasting disorder, DMD. To date, the research team has identified two series of novel utrophin modulators, one of which has a mechanism of action potentially distinct from ezutromid, the Company’s lead utrophin modulator that is in a Phase 2 clinical trial in DMD patients. “In our quest to bring a potentially disease-modifying treatment to all patients with DMD, we have collaborated with the preeminent expert in utrophin modulation biology, Professor Kay Davies, and her research team at the University of Oxford,” said Glyn Edwards, Chief Executive Officer of Summit. “We applaud EURORDIS, the organisation representing rare disease patients in Europe, for bringing an annual spotlight to the plight of millions of people affected by rare diseases and in this year, recognising the immense impact that research is having and will continue to have for those living with rare diseases.” In the European Union a rare disease is defined as one that affects fewer than 5 in 10,000 of the general population, while in the United States, it is defined as a disease that affects fewer than 200,000 people. There are between 6,000 and 8,000 known rare diseases with around five new rare diseases described in the literature each week. Rare diseases are often chronic and life threatening and include rare conditions, such as childhood cancers, and some other well-known conditions including cystic fibrosis and DMD. Rare Disease Day takes place on the last day of February each year, and its objective is to raise awareness among the general public and decision-makers about rare diseases and their impact on patients' lives. Rare Disease Day was launched in Europe in 2008 by EURORDIS. It is now observed in more than 80 nations, and is sponsored in the US by the National Organization for Rare Disorders (NORD). For more information, please visit www.rarediseaseday.org. About Utrophin Modulation in DMD DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties. Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin has a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company’s lead utrophin modulator, ezutromid, is an orally administered, small molecule. DMD is an orphan disease, and the US Food and Drug Administration (‘FDA’) and the European Medicines Agency have granted orphan drug status to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the FDA. About Summit Therapeutics Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc). For more information, please contact: Forward-looking Statements Any statements in this press release about Summit’s future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of Summit’s product candidates, the therapeutic potential of Summit’s product candidates, and the timing of initiation, completion and availability of data from clinical trials, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from on-going and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for Summit’s foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that Summit makes with the Securities and Exchange Commission including Summit’s Annual Report on Form 20-F for the fiscal year ended January 31, 2016. Accordingly readers should not place undue reliance on forward looking statements or information. In addition, any forward looking statements included in this press release represent Summit’s views only as of the date of this release and should not be relied upon as representing Summit’s views as of any subsequent date. Summit specifically disclaims any obligation to update any forward-looking statements included in this press release.


News Article | February 28, 2017
Site: globenewswire.com

Oxford, UK, 28 February 2017 - Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, recognises the tenth annual Rare Disease Day taking place today, 28 February 2017. The Rare Disease Day 2017 theme, 'with research, possibilities are limitless,' emphasises the importance of scientific research in helping to understand, diagnose and treat rare diseases that affect millions of people and their families worldwide. Summit seeks to remain at the forefront of utrophin modulation research through its strategic alliance with the University of Oxford, under the guidance of Professor Kay Davies. The collaboration is focussed on developing future-generation utrophin modulators for the potential treatment of all patients with the progressive muscle wasting disorder, DMD. To date, the research team has identified two series of novel utrophin modulators, one of which has a mechanism of action potentially distinct from ezutromid, the Company's lead utrophin modulator that is in a Phase 2 clinical trial in DMD patients. "In our quest to bring a potentially disease-modifying treatment to all patients with DMD, we have collaborated with the preeminent expert in utrophin modulation biology, Professor Kay Davies, and her research team at the University of Oxford," said Glyn Edwards, Chief Executive Officer of Summit. "We applaud EURORDIS, the organisation representing rare disease patients in Europe, for bringing an annual spotlight to the plight of millions of people affected by rare diseases and in this year, recognising the immense impact that research is having and will continue to have for those living with rare diseases." In the European Union a rare disease is defined as one that affects fewer than 5 in 10,000 of the general population, while in the United States, it is defined as a disease that affects fewer than 200,000 people. There are between 6,000 and 8,000 known rare diseases with around five new rare diseases described in the literature each week. Rare diseases are often chronic and life threatening and include rare conditions, such as childhood cancers, and some other well-known conditions including cystic fibrosis and DMD. Rare Disease Day takes place on the last day of February each year, and its objective is to raise awareness among the general public and decision-makers about rare diseases and their impact on patients' lives. Rare Disease Day was launched in Europe in 2008 by EURORDIS. It is now observed in more than 80 nations, and is sponsored in the US by the National Organization for Rare Disorders (NORD). For more information, please visit www.rarediseaseday.org. DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties. Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin has a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company's lead utrophin modulator, ezutromid, is an orally administered, small molecule. DMD is an orphan disease, and the US Food and Drug Administration ('FDA') and the European Medicines Agency have granted orphan drug status to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the FDA. Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc). For more information, please contact: Any statements in this press release about Summit's future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of Summit's product candidates, the therapeutic potential of Summit's product candidates, and the timing of initiation, completion and availability of data from clinical trials, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from on-going and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for Summit's foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that Summit makes with the Securities and Exchange Commission including Summit's Annual Report on Form 20-F for the fiscal year ended January 31, 2016. Accordingly readers should not place undue reliance on forward looking statements or information. In addition, any forward looking statements included in this press release represent Summit's views only as of the date of this release and should not be relied upon as representing Summit's views as of any subsequent date. Summit specifically disclaims any obligation to update any forward-looking statements included in this press release.


News Article | December 15, 2016
Site: globenewswire.com

Summit Therapeutics plc ('Summit', the 'Company' or the 'Group') SUMMIT THERAPEUTICS REPORTS FINANCIAL RESULTS FOR THE THIRD QUARTER ENDED 31 OCTOBER 2016 AND OPERATIONAL PROGRESS Oxford, UK, 15 December 2016 - Summit Therapeutics plc (AIM: SUMM, NASDAQ: SMMT), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and C. difficile infection ('CDI'), today reports its financial results for the third quarter ended 31 October 2016. Mr Glyn Edwards, Chief Executive Officer of Summit commented: "During the third quarter, we strengthened our DMD programme by entering into an exclusive collaboration and license agreement with Sarepta Therapeutics, granting European rights to our utrophin modulator pipeline, including our lead candidate, ezutromid. This collaboration gives us access to Sarepta's DMD development, regulatory and commercial expertise, while strengthening our financial position with global research and development cost sharing and the potential for future milestones. Ultimately, we believe that combining our strengths through this collaboration could help to bring utrophin modulators to market, where we have the potential to benefit all patients with this muscle wasting disease. "PhaseOut DMD, our Phase 2 proof of concept trial of ezutromid, is enrolling now in the UK and the US, and we are on track to report data from the first group of 24-week biopsies in the second or third quarter of 2017. These biopsies have the potential to demonstrate proof of mechanism for ezutromid through a change in the pattern of utrophin expression from baseline and an association of utrophin with mature muscle fibres - a phenomenon that we expect would only occur with drug treatment. This trial is expected to evaluate the F3 and F6 formulations of ezutromid that both have the potential to modulate utrophin over a wide range of exposures that could help us to maximise safety and efficacy in patients over longer-term dosing. "With our CDI programme, we continue preparations for Phase 3 trials of ridinilazole, a novel antibiotic with potential as a front-line treatment for patients suffering from this serious bacterial infection. We look forward to a productive 2017 with both programmes." Exclusive Collaboration and License Agreement with Sarepta Therapeutics Inc. ('Sarepta') About Summit Therapeutics Summit is a biopharmaceutical company focused on the discovery, development and commercialization of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc). For more information, please contact: Forward Looking Statements Any statements in this press release about our future expectations, plans and prospects, including statements about development and potential commercialisation of our product candidates, the therapeutic potential of our product candidates, the timing of initiation, completion and availability of data from clinical trials, the potential benefits and future operation of the collaboration with Sarepta Therapeutics Inc., including any potential future payments thereunder, any other potential third-party collaborations and expectations regarding the sufficiency of our cash balance to fund operating expenses and capital expenditures, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for our foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that we make with the Securities and Exchange Commission, including our Annual Report on Form 20-F for the fiscal year ended 31 January 2016. In addition, any forward-looking statements included in this press release represent our views only as of the date of this release and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update any forward-looking statements included in this press release. Summit is seeking to develop a treatment for all patients affected with the fatal disorder DMD using its utrophin modulation technology. Summit is also advancing the development of a highly selective antibiotic to treat CDI. Summit's DMD utrophin modulation programme is a treatment approach independent of the underlying mutations in the dystrophin gene that cause the disease. Therefore, we believe this approach has the potential to benefit the entire patient population. Summit has established a leadership position in the field of utrophin modulation and is developing a pipeline of orally administered small molecule utrophin modulator product candidates. Summit's most advanced utrophin modulator is ezutromid which is currently being evaluated in a Phase 2 proof of concept trial. Ezutromid has received orphan drug designation in the US and Europe, and Fast Track and Rare Pediatric Disease designations in the US. Summit's CDI product candidate is ridinilazole, a novel class antibiotic that has the potential to treat the initial infection and reduce recurrent disease, the key clinical issue in CDI. Ridinilazole markedly reduced recurrence rates and had a statistically superior rate of sustained clinical response ('SCR') compared to vancomycin in a Phase 2 proof of concept trial. Summit is currently evaluating its options for advancing ridinilazole into Phase 3 clinical trials. Ridinilazole has received Qualified Infectious Disease Product, or QIDP, designation and has been granted Fast Track designation in the US. Exclusive Collaboration and License Agreement with Sarepta Therapeutics Inc. ('Sarepta') In October 2016, Summit announced an exclusive collaboration and license agreement with Sarepta. This granted Sarepta rights to Summit's utrophin modulator pipeline in the European Union, Switzerland, Norway, Iceland, Turkey and the Commonwealth of Independent States, with an option for Latin American rights. Summit retains commercialization rights in all other countries, including the key markets of the US and Japan. Under the terms of the agreement, Summit has received an upfront payment of $40 million, and will be eligible for future ezutromid-related development, regulatory and sales milestone payments totalling up to $522 million. This includes $42 million in respect of specified development milestones (including a $22 million milestone upon the first dosing of the last patient in Summit's PhaseOut DMD trial, payable on or after 1 April 2017), $150 million in respect of specified regulatory milestones and a potential $330 million from specified sales milestones. In addition, Summit is eligible for escalating royalties ranging from a low to high teens percentage of net sales in the licensed territories. Beginning in 2018, Summit and Sarepta will share at a 55%/45% split specified global research and development costs related to ezutromid and future generation utrophin modulators. If Sarepta elects to exercise its option for Latin American rights, Summit would be entitled to additional fees, milestones and royalties. Summit will also be eligible to receive development and regulatory milestones related to potential future generation utrophin modulator candidate(s). Ezutromid: Phase 2 Proof of Concept Trial PhaseOut DMD is a Phase 2 clinical trial evaluating ezutromid in patients with DMD, and it aims to establish proof of concept for this utrophin modulator. The 48-week open-label trial is expected to enrol up to 40 boys ranging in age from their fifth to their tenth birthdays. Enrolment of patients into PhaseOut DMD is ongoing at sites in the UK and US. The Company anticipates reporting 24-week muscle biopsy data from the first group of patients in Q2 or Q3 2017. DMD is characterised by high levels of muscle degeneration caused by the absence of functional dystrophin. Muscle fibres consequently enter into a cycle of repair and degeneration that over time leads to fat infiltrating into muscle, loss of ambulation and loss of other functional abilities. Ezutromid aims to maintain production of utrophin so that it can substitute for the missing dystrophin. This has potential to allow muscle fibres to mature and so reduce the level of muscle degeneration, reduce the rate of fat infiltration and reduce the rate of decline in functional abilities. PhaseOut DMD is assessing all these factors through various techniques including use of muscle biopsy to evaluate utrophin expression and muscle fibre regeneration and maturity, magnetic resonance imaging to measure fat infiltration, and various functional tests including the North Star Ambulatory Assessment and the six minute walk test. Ezutromid: Phase 1 New Formulation Trial Summit announced in August 2016 results from a Phase 1 clinical trial that showed a new formulation of ezutromid called F6 achieved a substantial increase in plasma exposure in patients compared to the current clinical formulation called F3. The trial evaluated the pharmacokinetics and safety of three fixed doses (250mg, 500mg and 1,000mg twice a day) of the F6 formulation in patients aged between five and nine years who followed a modified diet. At the highest dose, the five evaluable patients achieved an average maximum concentration of 390ng/mL on day 7, the final day of dosing. This was a six-fold increase in maximum plasma levels compared to formulation F3 but were achieved with two-fifths of the dose of ezutromid. Summit plans to test the F6 formulation alongside the F3 formulation in the ongoing PhaseOut DMD clinical trial. It is anticipated that up to ten of the patients enrolled in the US will be dosed with F6 to evaluate safety and efficacy. The two formulations of ezutromid have the potential to modulate the expression of utrophin, and the inclusion of F6 will provide a greater understanding of the potential relationship between drug exposure and clinical benefit. Ezutromid: Commercialisation Strategy Summit outlined in August 2016 its strategy for the future development of ezutromid. The Company plans to conduct a randomised, placebo controlled trial designed with the potential to support accelerated and conditional regulatory approvals in the United States and Europe respectively. Assuming positive interim data from PhaseOut DMD, it is anticipated that this trial would start in the second half of 2017, with data available for potential regulatory filings in 2019. Summit also expects to conduct a separate confirmatory clinical trial designed to support full product approvals in major territories. Development of Biomarkers As highlighted, a key endpoint in the PhaseOut DMD trial is measurement of utrophin and muscle regeneration biomarkers from muscle biopsies. Summit, in collaboration with Flagship Biosciences Inc., has been developing an automated, digital analysis tool to precisely measure muscle maturity and integrity and utrophin expression in individual fibres, and data from this research were presented at the 21st Congress of the World Muscle Society held in Granada, Spain, in October 2016. This research represents an important step in helping to further our understanding of the potential benefits of utrophin modulator therapies such as ezutromid. Fast Track and Rare Pediatric Disease Designations In September, ezutromid was granted two separate designations by the US FDA in the treatment of DMD: Fast Track and Rare Pediatric Disease. Fast Track designation provides the Company with advantages such as opportunities for more frequent interactions with the FDA during all aspects of development, submission of a New Drug Application ('NDA') on a rolling basis, and eligibility for accelerated approval and priority review. Rare Pediatric Disease designation could qualify Summit for a Priority Review Voucher upon the approval of ezutromid, which could be used for a subsequent marketing application or sold or transferred an unlimited number of times (although only used once). The Priority Review Voucher programme was extended on 13 December 2016 through the enactment of the 21st Century Cures Act, under which a drug designated as a Rare Pediatric Disease can receive a voucher if approved before 1 October 2022. DMD Community Website On 12 September 2016, Summit launched www.utrophintrials.com, an online resource on utrophin and Summit's utrophin modulator clinical trials, in an effort to broaden the Company's relationship with the Duchenne community as it advances ezutromid and other utrophin modulators. Summit has generated a comprehensive package of data supporting the potential of the novel class antibiotic ridinilazole as a new front-line treatment of infections caused by the bacteria Clostridium difficile. The Phase 2 proof of concept trial called CoDIFy showed ridinilazole outperformed the antibiotic vancomycin, which is the current standard of care. Ridinilazole demonstrated a large numerical reduction in rates of recurrent disease compared to vancomycin with this difference driven by ridinilazole outperforming vancomycin in the preservation of the gut microbiome. Recurrence of CDI, and the failure to subsequently achieve a sustained clinical response after treatment, is a major issue in the management of the disease, as collateral damage to the gut microbiome by antibiotics such as vancomycin leaves patients vulnerable to disease recurrence. Phase 3 preparations are ongoing as the Company continues to explore options to support the future development of ridinilazole with a view to maximising the potential commercial value of this antibiotic. Summit's preference remains finding a third party collaborator although the Company continues to actively explore other potential options, including funding from government and non-profit organisations. In parallel, Summit is undertaking an exploratory Phase 2 trial called CoDIFy 2 to evaluate ridinilazole against the antibiotic fidaxomicin. This trial is intended to further understand the impact of ridinilazole on a number of disease parameters, including its impact on patients' gut microbiomes to help inform the design of the Phase 3 clinical programme for ridinilazole. Dosing of patients in this trial has now completed, and Summit expects to report top-line data, including analysis of the microbiome, during Q2 2017. The development of ridinilazole has been financially supported by Seeding Drug Discovery and Translational Awards both from the Wellcome Trust. As part of the exclusive collaboration and license agreement entered into with Sarepta, the Company received an upfront payment of £32.9 million. Of this amount, £0.6 million was recognised during the three months ended 31 October 2016. The remaining £32.3 million of the upfront payment is classified as deferred income and will be recognised as revenue over the development period. See Note 1, 'New accounting policy - Revenue Recognition.' Other operating income for the three months ended 31 October 2016 was £nil compared to £0.3 million for the three months ended 31 October 2015. Other operating income for the nine months ended 31 October 2016 was £0.07 million compared to £1.1 million (adjusted - see Note 1, 'Change in accounting policy') for the nine months ended 31 October 2015. All monies and income attributed to the funding agreement with the Wellcome Trust have now been received and accounted for with the completion of our CoDIFy Phase 2 clinical trial of ridinilazole. Income recognised as part of the funding from Innovate UK for the DMD programme was lower in the nine months ended 31 October 2016 as compared to the nine months ended 31 October 2015, with no income recognised in the three months ended 31 October 2016. The decrease in income is in line with the achievement of milestones under the funding agreement. Further, in September 2016, the Company elected to withdraw from the Innovate UK funding agreement in order to enable the Company to take advantage of more tax efficient opportunities related to research and development expenditure. Operating Expenses Research and Development Expenses Research and development expenses decreased by £0.5 million to £4.0 million for the three months ended 31 October 2016 from £4.5 million for the three months ended 31 October 2015. The decrease is driven by the completion of our CoDIFy Phase 2 clinical trial of ridinilazole. Research and development expenses increased by £2.3 million to £14.2 million for the nine months ended 31 October 2016 from £11.9 million for the nine months ended 31 October 2015. This increase reflected an overall increase in investment in the DMD programme and an increase in research and development related staffing costs driven by an increase in research and development related headcount. General and Administration Expenses General and administration expenses increased by £0.6 million to £1.9 million for the three months ended 31 October 2016 from £1.3 million for the three months ended 31 October 2015. General and administration expenses increased by £1.8 million to £5.2 million for the nine months ended 31 October 2016 from £3.4 million for the nine months ended 31 October 2015. These increases were primarily due to continuing additional corporate costs associated with being a publicly traded company in the United States as well as in the United Kingdom and an increase in staff related costs offset by a favourable exchange rate variance for both the three months ended 31 October 2016 and nine months ended 31 October 2016. Finance Costs Following an IFRS IC agenda decision in May 2016 on the application of IAS 20 'Government Grants,' the Company has changed its accounting policy regarding charitable funding arrangements from the Wellcome Trust and US Not for Profit organisations. See Note 1 - 'Change in accounting policy.' Finance costs relate to the subsequent re-measurement of the financial liability recognised in respect of funding arrangements and the unwinding of the discounts associated with the liabilities. Finance costs decreased by £0.3 million to £0.2 million for the three months ended 31 October 2016 from £0.5 million for the three months ended 31 October 2015 (adjusted). Finance costs decreased by £0.1 million to £0.7 million for the nine months ended 31 October 2016 from £0.8 million for the nine months ended 31 October 2015 (adjusted). Operating Activities For the nine months ended 31 October 2016, the Company generated £17.9 million in cash from operating activities. This compares to net cash used in operating activities of £11.2 million for the nine months ended 31 October 2015. This net movement of £29.1 million was driven by the receipt of a £32.9 million upfront payment received as part of the exclusive collaboration and licensing agreement entered into with Sarepta. This was offset by an increase in research and development expenditure and general and administrative expenditure during the nine months ended 31 October 2016, offset by a £1.6 million increase in the amount of research and development tax credit received during the nine months ended 31 October 2016, which totalled £3.0 million. Investing Activities Net cash used in investing activities for the nine months ended 31 October 2016 and the nine months ended 31 October 2015 includes the net amount of bank interest received on cash deposits less amounts paid to acquire property, plant and equipment. Financing Activities Net cash inflow from financing activities for the nine months ended 31 October 2016 relates to proceeds from the exercise of warrants and the exercise of share options during the nine months ended 31 October 2016. For the nine months ended 31 October 2015, the Company received net cash inflow related to proceeds from the sale of the Company's equity securities and exercise of share options, net of expenses. As at 31 October 2016, total cash and cash equivalents held were £34.6 million (31 January 2016: £16.3 million). CONSOLIDATED STATEMENT OF COMPREHENSIVE INCOME (unaudited) For the three months ended 31 October 2016 CONSOLIDATED STATEMENT OF COMPREHENSIVE INCOME (unaudited) For the nine months ended 31 October 2016 CONSOLIDATED STATEMENT OF FINANCIAL POSITION (unaudited) As at 31 October 2016 CONSOLIDATED STATEMENT OF CASH FLOWS (unaudited) For the nine months ended 31 October 2016 CONSOLIDATED STATEMENT OF CHANGES IN EQUITY (unaudited) NOTES TO THE FINANCIAL STATEMENTS For the three and nine months ended 31 October 2016 The unaudited consolidated interim financial statements of Summit and its subsidiaries (the 'Group') for the nine months ended 31 October 2016 have been prepared in accordance with International Financial Reporting Standards ('IFRS') and International Financial Reporting Interpretations Committee ('IFRIC') interpretations as issued by the International Accounting Standards Board and as adopted by the European Union and with those parts of the Companies Act 2006 applicable to companies reporting under IFRS including those applicable to accounting periods ending 31 January 2017 and the accounting policies set out in Summit's consolidated financial statements. They do not include all the statements required for full annual financial statements, and should be read in conjunction with the consolidated financial statements of the Group as at 31 January 2016 (the '2016 Accounts'). The 2016 Accounts, on which the Company's auditors delivered an unqualified audit report, have been delivered to the Registrar of Companies following the 2016 Annual General Meeting. The interim financial statements are prepared in accordance with the historical cost convention. Whilst the financial information included in this announcement has been prepared in accordance with IFRSs as issued by the International Accounting Standards Board and adopted for use in the European Union, this announcement does not itself contain sufficient information to comply with IFRSs. The interim financial statements have been prepared assuming the Group will continue on a going concern basis. The financial information for the three and nine month periods ended 31 October 2016 and 2015 are unaudited. Solely for the convenience of the reader, unless otherwise indicated, all pound sterling amounts stated in the Consolidated Balance Sheet as at 31 October 2016, in the Consolidated Income Statement for the three and nine months ended 31 October 2016 and in the Consolidated Cash Flow Statement for the nine months ended 31 October 2016 have been translated into US dollars at the rate on 31 October 2016 of $1.2212 to £1.00. These translations should not be considered representations that any such amounts have been, could have been or could be converted into US dollars at that or any other exchange rate as at that or any other date. The Board of Directors of the Company approved this statement on 15 December 2016. Change in accounting policy Following an IFRS IC agenda decision in May 2016 on the application of IAS 20 'Government Grants,' the Company has changed its accounting policy regarding charitable funding arrangements from the Wellcome Trust and US Not for Profit organisations. In exchange for the funding provided, these arrangements require the company to pay royalties on potential future revenues generated from these projects and also give the counterparties certain rights over the intellectual property if the compound is not exploited. The IFRIC decision has clarified that such arrangements result in a financial liability. The estimate of the financial liability is initially recognised at fair value using a discounted cash flow model with the difference between the fair value of the liability and the cash received considered to represent a charitable grant. When determining the fair value on initial recognition, the significant assumptions in the model include the estimation of the timing and the probability of successful development leading to commercialisation of the project related results and related estimates of future cash flows. Estimated future cash flows include expected sources of revenue (including commercial sales and upfront payments, milestone payments and royalties from potential licensing arrangements) and are calculated using estimated geographical market share and associated pricing. The financial liability is subsequently measured at amortised cost using a discounted cash flow model which calculates the risk adjusted present values of estimated potential future cash flows for the respective projects related to the Wellcome Trust and US Not for Profit agreements. The financial liability is re-measured when there is a specific significant event that provides evidence of a significant change in the probability of successful development such as the completion of a phase of research or changes in use or market for a product. The model will be updated for changes in the clinical probability of success and other associated assumptions with the discount rate remaining consistent within the model. Re-measurements of the financial liability are recognised in the income statement as finance costs. Grant income is recognised as other operating income in accordance with International Accounting Standard 20, 'Accounting for Government Grants and Disclosure of Government Assistance,' at the same time as the underlying expenditure is incurred, provided that there is reasonable assurance that the Group will comply with the conditions. This change in accounting policy has been reflected retrospectively in these financial statements. The impact of this change in accounting policy on the consolidated financial statements is a reduction in other income historically recognised, a change in the level of accrued income accounted for as grant income and the recognition of a financial liability and finance costs associated with the unwinding of the discount. New accounting policy - Revenue Recognition  As a result of the exclusive collaboration and licensing agreement entered into with Sarepta Therapeutics Inc., the following revenue recognition policy has been adopted. See Note 4 - 'Collaboration and License Agreement with Sarepta Therapeutics Inc.' Revenue is measured at the fair value of the consideration received or receivable and represents amounts receivable for goods and services provided in the normal course of business net of value added tax and other sales-related taxes. The Group recognises revenue when the amount can be reliably measured; when it is probable that future economic benefits will flow to the Group; and when specific criteria have been met for each of the Group's activities. Collaboration revenues consist of revenues generated from collaborative research and development arrangements. Such agreements may consist of multiple elements and provide for varying consideration terms, such as upfront, development, regulatory and sales milestones and sales royalties and similar payments. Where such arrangements can be divided into separate units of accounting (each unit constituting a separate earnings process), the arrangement consideration is allocated to the different units based on their relative fair values and recognised over the respective performance period. Revenues from non-refundable, upfront payments are assessed as to whether they relate to the provision of a license or development services. Upfront payments classified as the provision of a license are recognised in full immediately while revenue related to further development services are initially reported as deferred income on the Consolidated Statement of Financial Position and are recognised as revenue over the development period. Development and approval milestone payments are recognised as revenue based on the percentage of completion method on the assumption that all stages will be completed successfully, but with cumulative revenue recognised limited to non-refundable amounts already received or reasonably certain to be received. Royalty revenue is recognised on an accrual basis in accordance with the substance of the relevant agreement, provided that it is probable that the economic benefits will flow to the Group and the amount of revenue can be measured reliably. Sales related milestone payments are recognised in full in the period in which the relevant milestone is achieved. The loss per Ordinary Share has been calculated by dividing the loss for the period by the weighted average number of Ordinary Shares in issue during the nine month period to 31 October 2016: 61,457,313 and during the three month period to 31 October 2016: 61,571,215 (for the nine month period to 31 October 2015: 58,354,036 and for the three month period to 31 October 2015: 61,290,740). Since the Group has reported a net loss, diluted loss per ordinary share is equal to basic loss per ordinary share. On 14 April 2016, the number of Ordinary Shares increased to 61,467,785 following the exercise of warrants over 177,045 Ordinary Shares at an exercise price of 60 pence per share. The issue of shares raised net proceeds of £0.1 million. During the nine month period to 31 October 2016, the following exercise of share options took place: The total net proceeds from exercised share options during the period was £0.27 million. Following the exercise of the above share options, the number of Ordinary Shares in issue was 61,815,316. All new Ordinary Shares rank pari passu with existing Ordinary Shares. 4. Collaboration and License Agreement with Sarepta Therapeutics Inc. On 4 October 2016, Summit announced its entry into an exclusive Collaboration and License Agreement (the 'Collaboration Agreement') with Sarepta Therapeutics Inc. ('Sarepta'), pursuant to which the Company granted Sarepta the exclusive right to commercialize products in the Company's utrophin modulator pipeline in the European Union, Switzerland, Norway, Iceland, Turkey and the Commonwealth of Independent States (the 'Licensed Territory'). Such products include the Company's lead product candidate, ezutromid, for the treatment of Duchenne muscular dystrophy and its second generation and future generation small molecule utrophin modulators. The Company also granted Sarepta an option to expand the Licensed Territory to include Latin America. The Company retains commercialization rights in the rest of the world. Under the terms of the Collaboration Agreement, Summit received an upfront payment of $40.0 million (£32.9 million) from Sarepta. The terms of the contract have been assessed and the Company believe the development services to be indistinguishable and as a result the upfront payment has been initially reported as deferred income on the Consolidated Statement of Financial Position and is being recognised as revenue over the development period. In addition, the Company will be eligible to receive specified development, regulatory and potential sales milestones related to ezutromid and Summit's second generation and future generation small molecule utrophin modulators. Summit is also eligible for escalating royalties ranging from a low to high teens percentage of net sales in the Licensed Territories. This announcement contains inside information for the purposes of Article 7 of EU Regulation 596/2014 (MAR).


OXFORD, United Kingdom, Dec. 15, 2016 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (AIM:SUMM) (NASDAQ:SMMT), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and C. difficile infection (‘CDI’), today reports its financial results for the third quarter ended 31 October 2016. Mr Glyn Edwards, Chief Executive Officer of Summit commented: “During the third quarter, we strengthened our DMD programme by entering into an exclusive collaboration and license agreement with Sarepta Therapeutics, granting European rights to our utrophin modulator pipeline, including our lead candidate, ezutromid. This collaboration gives us access to Sarepta’s DMD development, regulatory and commercial expertise, while strengthening our financial position with global research and development cost sharing and the potential for future milestones. Ultimately, we believe that combining our strengths through this collaboration could help to bring utrophin modulators to market, where we have the potential to benefit all patients with this muscle wasting disease. “PhaseOut DMD, our Phase 2 proof of concept trial of ezutromid, is enrolling now in the UK and the US, and we are on track to report data from the first group of 24-week biopsies in the second or third quarter of 2017. These biopsies have the potential to demonstrate proof of mechanism for ezutromid through a change in the pattern of utrophin expression from baseline and an association of utrophin with mature muscle fibres – a phenomenon that we expect would only occur with drug treatment. This trial is expected to evaluate the F3 and F6 formulations of ezutromid that both have the potential to modulate utrophin over a wide range of exposures that could help us to maximise safety and efficacy in patients over longer-term dosing. “With our CDI programme, we continue preparations for Phase 3 trials of ridinilazole, a novel antibiotic with potential as a front-line treatment for patients suffering from this serious bacterial infection. We look forward to a productive 2017 with both programmes.” Exclusive Collaboration and License Agreement with Sarepta Therapeutics Inc. (‘Sarepta’) About Summit Therapeutics Summit is a biopharmaceutical company focused on the discovery, development and commercialization of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc). For more information, please contact: Forward Looking Statements Any statements in this press release about our future expectations, plans and prospects, including statements about development and potential commercialisation of our product candidates, the therapeutic potential of our product candidates, the timing of initiation, completion and availability of data from clinical trials, the potential benefits and future operation of the collaboration with Sarepta Therapeutics Inc., including any potential future payments thereunder, any other potential third-party collaborations and expectations regarding the sufficiency of our cash balance to fund operating expenses and capital expenditures, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for our foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that we make with the Securities and Exchange Commission, including our Annual Report on Form 20-F for the fiscal year ended 31 January 2016. In addition, any forward-looking statements included in this press release represent our views only as of the date of this release and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update any forward-looking statements included in this press release. Summit is seeking to develop a treatment for all patients affected with the fatal disorder DMD using its utrophin modulation technology. Summit is also advancing the development of a highly selective antibiotic to treat CDI. Summit’s DMD utrophin modulation programme is a treatment approach independent of the underlying mutations in the dystrophin gene that cause the disease. Therefore, we believe this approach has the potential to benefit the entire patient population. Summit has established a leadership position in the field of utrophin modulation and is developing a pipeline of orally administered small molecule utrophin modulator product candidates. Summit’s most advanced utrophin modulator is ezutromid which is currently being evaluated in a Phase 2 proof of concept trial. Ezutromid has received orphan drug designation in the US and Europe, and Fast Track and Rare Pediatric Disease designations in the US. Summit’s CDI product candidate is ridinilazole, a novel class antibiotic that has the potential to treat the initial infection and reduce recurrent disease, the key clinical issue in CDI. Ridinilazole markedly reduced recurrence rates and had a statistically superior rate of sustained clinical response (‘SCR’) compared to vancomycin in a Phase 2 proof of concept trial. Summit is currently evaluating its options for advancing ridinilazole into Phase 3 clinical trials. Ridinilazole has received Qualified Infectious Disease Product, or QIDP, designation and has been granted Fast Track designation in the US. Exclusive Collaboration and License Agreement with Sarepta Therapeutics Inc. (‘Sarepta’) In October 2016, Summit announced an exclusive collaboration and license agreement with Sarepta. This granted Sarepta rights to Summit’s utrophin modulator pipeline in the European Union, Switzerland, Norway, Iceland, Turkey and the Commonwealth of Independent States, with an option for Latin American rights. Summit retains commercialization rights in all other countries, including the key markets of the US and Japan. Under the terms of the agreement, Summit has received an upfront payment of $40 million, and will be eligible for future ezutromid-related development, regulatory and sales milestone payments totalling up to $522 million. This includes $42 million in respect of specified development milestones (including a $22 million milestone upon the first dosing of the last patient in Summit’s PhaseOut DMD trial, payable on or after 1 April 2017), $150 million in respect of specified regulatory milestones and a potential $330 million from specified sales milestones. In addition, Summit is eligible for escalating royalties ranging from a low to high teens percentage of net sales in the licensed territories. Beginning in 2018, Summit and Sarepta will share at a 55%/45% split specified global research and development costs related to ezutromid and future generation utrophin modulators. If Sarepta elects to exercise its option for Latin American rights, Summit would be entitled to additional fees, milestones and royalties. Summit will also be eligible to receive development and regulatory milestones related to potential future generation utrophin modulator candidate(s). Ezutromid: Phase 2 Proof of Concept Trial PhaseOut DMD is a Phase 2 clinical trial evaluating ezutromid in patients with DMD, and it aims to establish proof of concept for this utrophin modulator. The 48-week open-label trial is expected to enrol up to 40 boys ranging in age from their fifth to their tenth birthdays. Enrolment of patients into PhaseOut DMD is ongoing at sites in the UK and US. The Company anticipates reporting 24-week muscle biopsy data from the first group of patients in Q2 or Q3 2017. DMD is characterised by high levels of muscle degeneration caused by the absence of functional dystrophin. Muscle fibres consequently enter into a cycle of repair and degeneration that over time leads to fat infiltrating into muscle, loss of ambulation and loss of other functional abilities. Ezutromid aims to maintain production of utrophin so that it can substitute for the missing dystrophin. This has potential to allow muscle fibres to mature and so reduce the level of muscle degeneration, reduce the rate of fat infiltration and reduce the rate of decline in functional abilities. PhaseOut DMD is assessing all these factors through various techniques including use of muscle biopsy to evaluate utrophin expression and muscle fibre regeneration and maturity, magnetic resonance imaging to measure fat infiltration, and various functional tests including the North Star Ambulatory Assessment and the six minute walk test. Ezutromid: Phase 1 New Formulation Trial Summit announced in August 2016 results from a Phase 1 clinical trial that showed a new formulation of ezutromid called F6 achieved a substantial increase in plasma exposure in patients compared to the current clinical formulation called F3. The trial evaluated the pharmacokinetics and safety of three fixed doses (250mg, 500mg and 1,000mg twice a day) of the F6 formulation in patients aged between five and nine years who followed a modified diet. At the highest dose, the five evaluable patients achieved an average maximum concentration of 390ng/mL on day 7, the final day of dosing. This was a six-fold increase in maximum plasma levels compared to formulation F3 but were achieved with two-fifths of the dose of ezutromid. Summit plans to test the F6 formulation alongside the F3 formulation in the ongoing PhaseOut DMD clinical trial. It is anticipated that up to ten of the patients enrolled in the US will be dosed with F6 to evaluate safety and efficacy. The two formulations of ezutromid have the potential to modulate the expression of utrophin, and the inclusion of F6 will provide a greater understanding of the potential relationship between drug exposure and clinical benefit. Ezutromid: Commercialisation Strategy Summit outlined in August 2016 its strategy for the future development of ezutromid. The Company plans to conduct a randomised, placebo controlled trial designed with the potential to support accelerated and conditional regulatory approvals in the United States and Europe respectively. Assuming positive interim data from PhaseOut DMD, it is anticipated that this trial would start in the second half of 2017, with data available for potential regulatory filings in 2019. Summit also expects to conduct a separate confirmatory clinical trial designed to support full product approvals in major territories. Development of Biomarkers As highlighted, a key endpoint in the PhaseOut DMD trial is measurement of utrophin and muscle regeneration biomarkers from muscle biopsies. Summit, in collaboration with Flagship Biosciences Inc., has been developing an automated, digital analysis tool to precisely measure muscle maturity and integrity and utrophin expression in individual fibres, and data from this research were presented at the 21st Congress of the World Muscle Society held in Granada, Spain, in October 2016. This research represents an important step in helping to further our understanding of the potential benefits of utrophin modulator therapies such as ezutromid. Fast Track and Rare Pediatric Disease Designations In September, ezutromid was granted two separate designations by the US FDA in the treatment of DMD: Fast Track and Rare Pediatric Disease. Fast Track designation provides the Company with advantages such as opportunities for more frequent interactions with the FDA during all aspects of development, submission of a New Drug Application (‘NDA’) on a rolling basis, and eligibility for accelerated approval and priority review. Rare Pediatric Disease designation could qualify Summit for a Priority Review Voucher upon the approval of ezutromid, which could be used for a subsequent marketing application or sold or transferred an unlimited number of times (although only used once). The Priority Review Voucher programme was extended on 13 December 2016 through the enactment of the 21st Century Cures Act, under which a drug designated as a Rare Pediatric Disease can receive a voucher if approved before 1 October 2022. DMD Community Website On 12 September 2016, Summit launched www.utrophintrials.com, an online resource on utrophin and Summit’s utrophin modulator clinical trials, in an effort to broaden the Company’s relationship with the Duchenne community as it advances ezutromid and other utrophin modulators. Summit has generated a comprehensive package of data supporting the potential of the novel class antibiotic ridinilazole as a new front-line treatment of infections caused by the bacteria Clostridium difficile. The Phase 2 proof of concept trial called CoDIFy showed ridinilazole outperformed the antibiotic vancomycin, which is the current standard of care. Ridinilazole demonstrated a large numerical reduction in rates of recurrent disease compared to vancomycin with this difference driven by ridinilazole outperforming vancomycin in the preservation of the gut microbiome. Recurrence of CDI, and the failure to subsequently achieve a sustained clinical response after treatment, is a major issue in the management of the disease, as collateral damage to the gut microbiome by antibiotics such as vancomycin leaves patients vulnerable to disease recurrence. Phase 3 preparations are ongoing as the Company continues to explore options to support the future development of ridinilazole with a view to maximising the potential commercial value of this antibiotic. Summit’s preference remains finding a third party collaborator although the Company continues to actively explore other potential options, including funding from government and non-profit organisations. In parallel, Summit is undertaking an exploratory Phase 2 trial called CoDIFy 2 to evaluate ridinilazole against the antibiotic fidaxomicin. This trial is intended to further understand the impact of ridinilazole on a number of disease parameters, including its impact on patients’ gut microbiomes to help inform the design of the Phase 3 clinical programme for ridinilazole. Dosing of patients in this trial has now completed, and Summit expects to report top-line data, including analysis of the microbiome, during Q2 2017. The development of ridinilazole has been financially supported by Seeding Drug Discovery and Translational Awards both from the Wellcome Trust. As part of the exclusive collaboration and license agreement entered into with Sarepta, the Company received an upfront payment of £32.9 million. Of this amount, £0.6 million was recognised during the three months ended 31 October 2016. The remaining £32.3 million of the upfront payment is classified as deferred income and will be recognised as revenue over the development period. See Note 1, ‘New accounting policy – Revenue Recognition.’ Other operating income for the three months ended 31 October 2016 was £nil compared to £0.3 million for the three months ended 31 October 2015. Other operating income for the nine months ended 31 October 2016 was £0.07 million compared to £1.1 million (adjusted – see Note 1, ‘Change in accounting policy’) for the nine months ended 31 October 2015. All monies and income attributed to the funding agreement with the Wellcome Trust have now been received and accounted for with the completion of our CoDIFy Phase 2 clinical trial of ridinilazole. Income recognised as part of the funding from Innovate UK for the DMD programme was lower in the nine months ended 31 October 2016 as compared to the nine months ended 31 October 2015, with no income recognised in the three months ended 31 October 2016. The decrease in income is in line with the achievement of milestones under the funding agreement. Further, in September 2016, the Company elected to withdraw from the Innovate UK funding agreement in order to enable the Company to take advantage of more tax efficient opportunities related to research and development expenditure. Operating Expenses Research and Development Expenses Research and development expenses decreased by £0.5 million to £4.0 million for the three months ended 31 October 2016 from £4.5 million for the three months ended 31 October 2015. The decrease is driven by the completion of our CoDIFy Phase 2 clinical trial of ridinilazole. Research and development expenses increased by £2.3 million to £14.2 million for the nine months ended 31 October 2016 from £11.9 million for the nine months ended 31 October 2015. This increase reflected an overall increase in investment in the DMD programme and an increase in research and development related staffing costs driven by an increase in research and development related headcount. General and Administration Expenses General and administration expenses increased by £0.6 million to £1.9 million for the three months ended 31 October 2016 from £1.3 million for the three months ended 31 October 2015. General and administration expenses increased by £1.8 million to £5.2 million for the nine months ended 31 October 2016 from £3.4 million for the nine months ended 31 October 2015. These increases were primarily due to continuing additional corporate costs associated with being a publicly traded company in the United States as well as in the United Kingdom and an increase in staff related costs offset by a favourable exchange rate variance for both the three months ended 31 October 2016 and nine months ended 31 October 2016. Finance Costs Following an IFRS IC agenda decision in May 2016 on the application of IAS 20 'Government Grants,' the Company has changed its accounting policy regarding charitable funding arrangements from the Wellcome Trust and US Not for Profit organisations. See Note 1 – ‘Change in accounting policy.’ Finance costs relate to the subsequent re-measurement of the financial liability recognised in respect of funding arrangements and the unwinding of the discounts associated with the liabilities. Finance costs decreased by £0.3 million to £0.2 million for the three months ended 31 October 2016 from £0.5 million for the three months ended 31 October 2015 (adjusted). Finance costs decreased by £0.1 million to £0.7 million for the nine months ended 31 October 2016 from £0.8 million for the nine months ended 31 October 2015 (adjusted). Operating Activities For the nine months ended 31 October 2016, the Company generated £17.9 million in cash from operating activities. This compares to net cash used in operating activities of £11.2 million for the nine months ended 31 October 2015. This net movement of £29.1 million was driven by the receipt of a £32.9 million upfront payment received as part of the exclusive collaboration and licensing agreement entered into with Sarepta. This was offset by an increase in research and development expenditure and general and administrative expenditure during the nine months ended 31 October 2016, offset by a £1.6 million increase in the amount of research and development tax credit received during the nine months ended 31 October 2016, which totalled £3.0 million. Investing Activities Net cash used in investing activities for the nine months ended 31 October 2016 and the nine months ended 31 October 2015 includes the net amount of bank interest received on cash deposits less amounts paid to acquire property, plant and equipment. Financing Activities Net cash inflow from financing activities for the nine months ended 31 October 2016 relates to proceeds from the exercise of warrants and the exercise of share options during the nine months ended 31 October 2016. For the nine months ended 31 October 2015, the Company received net cash inflow related to proceeds from the sale of the Company’s equity securities and exercise of share options, net of expenses. As at 31 October 2016, total cash and cash equivalents held were £34.6 million (31 January 2016: £16.3 million). CONSOLIDATED STATEMENT OF COMPREHENSIVE INCOME (unaudited) For the three months ended 31 October 2016 CONSOLIDATED STATEMENT OF COMPREHENSIVE INCOME (unaudited) For the nine months ended 31 October 2016 CONSOLIDATED STATEMENT OF FINANCIAL POSITION (unaudited) As at 31 October 2016 CONSOLIDATED STATEMENT OF CASH FLOWS (unaudited) For the nine months ended 31 October 2016 CONSOLIDATED STATEMENT OF CHANGES IN EQUITY (unaudited) NOTES TO THE FINANCIAL STATEMENTS For the three and nine months ended 31 October 2016 The unaudited consolidated interim financial statements of Summit and its subsidiaries (the ‘Group’) for the nine months ended 31 October 2016 have been prepared in accordance with International Financial Reporting Standards (‘IFRS’) and International Financial Reporting Interpretations Committee (‘IFRIC’) interpretations as issued by the International Accounting Standards Board and as adopted by the European Union and with those parts of the Companies Act 2006 applicable to companies reporting under IFRS including those applicable to accounting periods ending 31 January 2017 and the accounting policies set out in Summit’s consolidated financial statements. They do not include all the statements required for full annual financial statements, and should be read in conjunction with the consolidated financial statements of the Group as at 31 January 2016 (the ‘2016 Accounts’). The 2016 Accounts, on which the Company’s auditors delivered an unqualified audit report, have been delivered to the Registrar of Companies following the 2016 Annual General Meeting. The interim financial statements are prepared in accordance with the historical cost convention. Whilst the financial information included in this announcement has been prepared in accordance with IFRSs as issued by the International Accounting Standards Board and adopted for use in the European Union, this announcement does not itself contain sufficient information to comply with IFRSs. The interim financial statements have been prepared assuming the Group will continue on a going concern basis. The financial information for the three and nine month periods ended 31 October 2016 and 2015 are unaudited. Solely for the convenience of the reader, unless otherwise indicated, all pound sterling amounts stated in the Consolidated Balance Sheet as at 31 October 2016, in the Consolidated Income Statement for the three and nine months ended 31 October 2016 and in the Consolidated Cash Flow Statement for the nine months ended 31 October 2016 have been translated into US dollars at the rate on 31 October 2016 of $1.2212 to £1.00. These translations should not be considered representations that any such amounts have been, could have been or could be converted into US dollars at that or any other exchange rate as at that or any other date. The Board of Directors of the Company approved this statement on 15 December 2016. Change in accounting policy Following an IFRS IC agenda decision in May 2016 on the application of IAS 20 'Government Grants,' the Company has changed its accounting policy regarding charitable funding arrangements from the Wellcome Trust and US Not for Profit organisations. In exchange for the funding provided, these arrangements require the company to pay royalties on potential future revenues generated from these projects and also give the counterparties certain rights over the intellectual property if the compound is not exploited. The IFRIC decision has clarified that such arrangements result in a financial liability. The estimate of the financial liability is initially recognised at fair value using a discounted cash flow model with the difference between the fair value of the liability and the cash received considered to represent a charitable grant. When determining the fair value on initial recognition, the significant assumptions in the model include the estimation of the timing and the probability of successful development leading to commercialisation of the project related results and related estimates of future cash flows. Estimated future cash flows include expected sources of revenue (including commercial sales and upfront payments, milestone payments and royalties from potential licensing arrangements) and are calculated using estimated geographical market share and associated pricing. The financial liability is subsequently measured at amortised cost using a discounted cash flow model which calculates the risk adjusted present values of estimated potential future cash flows for the respective projects related to the Wellcome Trust and US Not for Profit agreements. The financial liability is re-measured when there is a specific significant event that provides evidence of a significant change in the probability of successful development such as the completion of a phase of research or changes in use or market for a product. The model will be updated for changes in the clinical probability of success and other associated assumptions with the discount rate remaining consistent within the model. Re-measurements of the financial liability are recognised in the income statement as finance costs. Grant income is recognised as other operating income in accordance with International Accounting Standard 20, ‘Accounting for Government Grants and Disclosure of Government Assistance,’ at the same time as the underlying expenditure is incurred, provided that there is reasonable assurance that the Group will comply with the conditions. This change in accounting policy has been reflected retrospectively in these financial statements. The impact of this change in accounting policy on the consolidated financial statements is a reduction in other income historically recognised, a change in the level of accrued income accounted for as grant income and the recognition of a financial liability and finance costs associated with the unwinding of the discount. As a result of the exclusive collaboration and licensing agreement entered into with Sarepta Therapeutics Inc., the following revenue recognition policy has been adopted. See Note 4 – ‘Collaboration and License Agreement with Sarepta Therapeutics Inc.’ Revenue is measured at the fair value of the consideration received or receivable and represents amounts receivable for goods and services provided in the normal course of business net of value added tax and other sales-related taxes. The Group recognises revenue when the amount can be reliably measured; when it is probable that future economic benefits will flow to the Group; and when specific criteria have been met for each of the Group’s activities. Collaboration revenues consist of revenues generated from collaborative research and development arrangements. Such agreements may consist of multiple elements and provide for varying consideration terms, such as upfront, development, regulatory and sales milestones and sales royalties and similar payments. Where such arrangements can be divided into separate units of accounting (each unit constituting a separate earnings process), the arrangement consideration is allocated to the different units based on their relative fair values and recognised over the respective performance period. Revenues from non-refundable, upfront payments are assessed as to whether they relate to the provision of a license or development services. Upfront payments classified as the provision of a license are recognised in full immediately while revenue related to further development services are initially reported as deferred income on the Consolidated Statement of Financial Position and are recognised as revenue over the development period. Development and approval milestone payments are recognised as revenue based on the percentage of completion method on the assumption that all stages will be completed successfully, but with cumulative revenue recognised limited to non-refundable amounts already received or reasonably certain to be received. Royalty revenue is recognised on an accrual basis in accordance with the substance of the relevant agreement, provided that it is probable that the economic benefits will flow to the Group and the amount of revenue can be measured reliably. Sales related milestone payments are recognised in full in the period in which the relevant milestone is achieved. The loss per Ordinary Share has been calculated by dividing the loss for the period by the weighted average number of Ordinary Shares in issue during the nine month period to 31 October 2016: 61,457,313 and during the three month period to 31 October 2016: 61,571,215 (for the nine month period to 31 October 2015: 58,354,036 and for the three month period to 31 October 2015: 61,290,740). Since the Group has reported a net loss, diluted loss per ordinary share is equal to basic loss per ordinary share. On 14 April 2016, the number of Ordinary Shares increased to 61,467,785 following the exercise of warrants over 177,045 Ordinary Shares at an exercise price of 60 pence per share. The issue of shares raised net proceeds of £0.1 million. During the nine month period to 31 October 2016, the following exercise of share options took place: The total net proceeds from exercised share options during the period was £0.27 million. Following the exercise of the above share options, the number of Ordinary Shares in issue was 61,815,316. All new Ordinary Shares rank pari passu with existing Ordinary Shares. 4. Collaboration and License Agreement with Sarepta Therapeutics Inc. On 4 October 2016, Summit announced its entry into an exclusive Collaboration and License Agreement (the ‘Collaboration Agreement‘) with Sarepta Therapeutics Inc. (‘Sarepta‘), pursuant to which the Company granted Sarepta the exclusive right to commercialize products in the Company’s utrophin modulator pipeline in the European Union, Switzerland, Norway, Iceland, Turkey and the Commonwealth of Independent States (the ‘Licensed Territory‘). Such products include the Company’s lead product candidate, ezutromid, for the treatment of Duchenne muscular dystrophy and its second generation and future generation small molecule utrophin modulators. The Company also granted Sarepta an option to expand the Licensed Territory to include Latin America. The Company retains commercialization rights in the rest of the world. Under the terms of the Collaboration Agreement, Summit received an upfront payment of $40.0 million (£32.9 million) from Sarepta. The terms of the contract have been assessed and the Company believe the development services to be indistinguishable and as a result the upfront payment has been initially reported as deferred income on the Consolidated Statement of Financial Position and is being recognised as revenue over the development period. In addition, the Company will be eligible to receive specified development, regulatory and potential sales milestones related to ezutromid and Summit’s second generation and future generation small molecule utrophin modulators. Summit is also eligible for escalating royalties ranging from a low to high teens percentage of net sales in the Licensed Territories. This announcement contains inside information for the purposes of Article 7 of EU Regulation 596/2014 (MAR).


News Article | December 13, 2016
Site: globenewswire.com

SUMMIT THERAPEUTICS TO REPORT FINANCIAL RESULTS FOR THE THIRD QUARTER ENDED 31 OCTOBER 2016 ON 15 DECEMBER 2016 Oxford, UK, 13 December 2016 - Summit Therapeutics plc (AIM: SUMM, NASDAQ: SMMT), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy and C. difficile infection, will be announcing its financial results for the third quarter and nine months ended 31 October 2016 on Thursday, 15 December 2016. About Summit Therapeutics Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease DMD and the infectious disease Clostridium difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc). For more information, please contact:


SUMMIT ENROLS PATIENTS IN THE UNITED STATES INTO PhaseOut DMD, A PHASE 2 CLINICAL TRIAL OF EZUTROMID IN PATIENTS WITH DMD Oxford, UK, 16 November 2016 - Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, today announces that it has enrolled its first patients at trial sites in the US into PhaseOut DMD, a Phase 2 proof of concept clinical trial of ezutromid in patients with DMD. Ezutromid dosing is expected to follow within a screening period of up to 28 days. Enrolment and dosing of patients into PhaseOut DMD in the UK is ongoing. Ezutromid is a utrophin modulator and represents a potential disease modifying treatment for all patients with DMD. "Ezutromid has shown great promise in preclinical testing as a universal treatment that has the potential to slow or stop disease progression in all patients with DMD, regardless of their underlying dystrophin gene mutation," said John Jefferies, MD, of Cincinnati Children's Hospital Medical Center, and the US coordinating investigator in PhaseOut DMD. "We are excited to participate in PhaseOut DMD and contribute to the clinical development of this utrophin modulator." Ralf Rosskamp, MD, Chief Medical Officer of Summit added, "Our PhaseOut DMD clinical trial is an important component of bringing ezutromid to patients and families who are in urgent need of a disease modifying therapy, and we are making progress with patient enrolment in this clinical trial, with enrolment ongoing in the UK and now in the US." This announcement contains inside information for the purposes of Article 7 of EU Regulation 596/2014 (MAR). PhaseOut DMD aims to provide proof of concept for ezutromid and utrophin modulation by measuring muscle fat infiltration, as well as by measuring utrophin protein and muscle fibre regeneration in muscle biopsies. The primary endpoint of the open-label trial is the change from baseline in magnetic resonance imaging parameters related to fat infiltration and inflammation of the leg muscles. Exploratory endpoints include the six-minute walk distance, the North Star Ambulatory Assessment and patient reported outcomes. PhaseOut DMD is a 48-week open-label trial expected to enrol up to 40 boys ranging in age from their fifth to their tenth birthdays at sites in the UK and the US. Each patient will receive two biopsies, one at baseline and the second either at 24 or 48 weeks. Data from the initial group of 24-week biopsies are expected Q2/Q3 2017. Further information is available at: https://clinicaltrials.gov/ct2/show/NCT02858362 and www.utrophintrials.com. DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties. Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin has a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company's lead utrophin modulator, ezutromid, is an orally administered, small molecule. DMD is an orphan disease, and the US Food and Drug Administration ('FDA') and the European Medicines Agency have granted orphan drug status to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the FDA. Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc). For more information, please contact: Any statements in this press release about Summit's future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of Summit's product candidates, the therapeutic potential of Summit's product candidates, and the timing of initiation, completion and availability of data from clinical trials, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from on-going and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for Summit's foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that Summit makes with the Securities and Exchange Commission including Summit's Annual Report on Form 20-F for the fiscal year ended January 31, 2016. Accordingly readers should not place undue reliance on forward looking statements or information. In addition, any forward looking statements included in this press release represent Summit's views only as of the date of this release and should not be relied upon as representing Summit's views as of any subsequent date. Summit specifically disclaims any obligation to update any forward-looking statements included in this press release.


News Article | February 23, 2017
Site: globenewswire.com

Oxford, UK, 23 February 2017 - Summit Therapeutics plc (AIM: SUMM, NASDAQ: SMMT), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy and C. difficile infection, announces that it has received notice and payment from the University of Oxford to exercise warrants over 50,000 ordinary shares of 1 penny each (the "New Ordinary Shares") at an exercise price of 20 pence per ordinary share. Application has been made for the admission to trading on AIM of the New Ordinary Shares, which will rank pari passu with the Company's existing ordinary shares, and admission is expected to occur on or around 1 March 2017. Following admission of the New Ordinary Shares to trading on AIM, the total number of ordinary shares with voting rights in issue will be 61,891,566.  This figure may be used by shareholders as the denominator for the calculations by which they will determine if they are required to notify their interest in, or a change to their interest in, the Company under the FCA's Disclosure and Transparency Rules. This announcement contains inside information for the purposes of Article 7 of EU Regulation 596/2014. For more information, please contact:


OXFORD, United Kingdom, Dec. 13, 2016 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (AIM:SUMM) (NASDAQ:SMMT), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy and C. difficile infection, will be announcing its financial results for the third quarter and nine months ended 31 October 2016 on Thursday, 15 December 2016. About Summit Therapeutics Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease DMD and the infectious disease Clostridium difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc). For more information, please contact:


OXFORD, United Kingdom, Nov. 16, 2016 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium difficile infection, today announces that it has enrolled its first patients at trial sites in the US into PhaseOut DMD, a Phase 2 proof of concept clinical trial of ezutromid in patients with DMD. Ezutromid dosing is expected to follow within a screening period of up to 28 days. Enrolment and dosing of patients into PhaseOut DMD in the UK is ongoing. Ezutromid is a utrophin modulator and represents a potential disease modifying treatment for all patients with DMD. “Ezutromid has shown great promise in preclinical testing as a universal treatment that has the potential to slow or stop disease progression in all patients with DMD, regardless of their underlying dystrophin gene mutation,” said John Jefferies, MD, of Cincinnati Children’s Hospital Medical Center, and the US coordinating investigator in PhaseOut DMD. ”We are excited to participate in PhaseOut DMD and contribute to the clinical development of this utrophin modulator.” Ralf Rosskamp, MD, Chief Medical Officer of Summit added, “Our PhaseOut DMD clinical trial is an important component of bringing ezutromid to patients and families who are in urgent need of a disease modifying therapy, and we are making progress with patient enrolment in this clinical trial, with enrolment ongoing in the UK and now in the US.” This announcement contains inside information for the purposes of Article 7 of EU Regulation 596/2014 (MAR). About PhaseOut DMD PhaseOut DMD aims to provide proof of concept for ezutromid and utrophin modulation by measuring muscle fat infiltration, as well as by measuring utrophin protein and muscle fibre regeneration in muscle biopsies. The primary endpoint of the open-label trial is the change from baseline in magnetic resonance imaging parameters related to fat infiltration and inflammation of the leg muscles. Exploratory endpoints include the six-minute walk distance, the North Star Ambulatory Assessment and patient reported outcomes. PhaseOut DMD is a 48-week open-label trial expected to enrol up to 40 boys ranging in age from their fifth to their tenth birthdays at sites in the UK and the US. Each patient will receive two biopsies, one at baseline and the second either at 24 or 48 weeks. Data from the initial group of 24-week biopsies are expected Q2/Q3 2017. Further information is available at: https://clinicaltrials.gov/ct2/show/NCT02858362 and www.utrophintrials.com. About Utrophin Modulation in DMD DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties. Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin has a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company’s lead utrophin modulator, ezutromid, is an orally administered, small molecule. DMD is an orphan disease, and the US Food and Drug Administration (‘FDA’) and the European Medicines Agency have granted orphan drug status to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the FDA. About Summit Therapeutics Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc). For more information, please contact: Forward-looking Statements Any statements in this press release about Summit’s future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of Summit’s product candidates, the therapeutic potential of Summit’s product candidates, and the timing of initiation, completion and availability of data from clinical trials, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from on-going and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for Summit’s foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that Summit makes with the Securities and Exchange Commission including Summit’s Annual Report on Form 20-F for the fiscal year ended January 31, 2016. Accordingly readers should not place undue reliance on forward looking statements or information. In addition, any forward looking statements included in this press release represent Summit’s views only as of the date of this release and should not be relied upon as representing Summit’s views as of any subsequent date. Summit specifically disclaims any obligation to update any forward-looking statements included in this press release.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, today reported financial results for the three and twelve months ended December 31, 2016. “2016 was a transformative year, with the FDA accelerated approval of EXONDYS 51. In 2017, we are focused on our strategy to build shareholder value by executing a successful launch of EXONDYS 51 in the US, reaching more patients through global expansion, and rapidly advancing our pipeline through internal and external development efforts,” said Edward Kaye, Sarepta’s chief executive officer. “We are pleased with the interest from the patient and physician community for EXONDYS 51, and with the progress we have made in discussions with payers. We believe this positions us well for potential growth and towards our goal at Sarepta Therapeutics to help all boys with Duchenne muscular dystrophy.” For the fourth quarter of 2016, Sarepta reported a net loss of $88.5 million, or $1.62 per share, compared to a net loss of $64.7 million for the same period of 2015, or $1.44 per share. The incremental loss of $23.8 million was primarily driven by expense recorded in connection with an up-front payment of $40.0 million related to an exclusive license agreement with Summit Therapeutics plc. (“Summit”) offset by lower manufacturing expenses that were previously captured as research and development expenses, which are now capitalized as inventory because of the approval of EXONDYS 51 by the Food and Drug Administration (“FDA”). Non-GAAP net loss for the fourth quarter of 2016 was $38.7 million, or $0.71 per share, compared to a non-GAAP net loss of $58.3 million for the fourth quarter of 2015, or $1.30 per share. The reduction of $19.7 million in Non-GAAP net loss was primarily driven by the capitalization of inventory upon the approval of EXONDYS 51 by the FDA. For the year ended December 31, 2016, Sarepta reported a net loss of $267.3 million, or $5.49 per share, compared to a net loss of $220.0 million for the prior year, or $5.20 per share. The incremental loss of $47.2 million was primarily driven by $47.9 million of research and development expenses recorded in connection with up-front license and milestone payments related to certain license and collaboration agreements and increased costs for our on-going clinical trials primarily due to increased patient enrollment, partially offset by lower manufacturing expenses because of the capitalization of inventory upon the approval of EXONDYS 51 by the FDA. Non-GAAP net loss for the year ended December 31, 2016 was $192.0 million, or $3.94 per share, compared to a non-GAAP net loss of $187.9 million for the prior year, or $4.44 per share. The incremental loss of $4.0 million was primarily driven by increased costs for our on-going clinical trials primarily due to increased patient enrollment partially offset by lower manufacturing expenses because of the capitalization of inventory upon the approval of EXONDYS 51 by the FDA. The Company commenced shipments of EXONDYS 51 to customers at the end of the third quarter of 2016 following the accelerated approval by the FDA on September 19, 2016. For both the fourth quarter and full-year of 2016, the Company recognized net revenues of $5.4 million. For the same periods of 2015, the Company recognized $1.3 million of revenue from the contract finalization of the Ebola portion of the July 2010 Department of Defense contract. Research and development expenses were $70.7 million for the fourth quarter of 2016, compared to $41.4 million for the same period of 2015, an increase of $29.4 million, which was primarily driven by expense recorded in connection with an up-front payment of $40.0 million related to the exclusive license agreement with Summit offset by lower manufacturing expenses because of the capitalization of inventory upon the approval of EXONDYS 51 by the FDA. Non-GAAP research and development expenses were $27.8 million for the fourth quarter of 2016, compared to $38.6 million for the same period of 2015, a decrease of $10.8 million, which was primarily driven by lower manufacturing expense because of the capitalization of inventory upon the approval of EXONDYS 51 by the FDA. Research and development expenses were $188.3 million for the year ended December 31, 2016, compared to $146.4 million for the prior year, an increase of $41.9 million, which was primarily driven by $47.9 million of expenses recorded in connection with up-front license and milestone payments related to certain license and collaboration agreements and increased clinical trial costs, partially offset by lower manufacturing expenses because of the capitalization of inventory upon the approval of EXONDYS 51 by the FDA. Non-GAAP research and development expenses were $136.0 million for both the year ended December 31, 2016, and the prior year. Selling, general and administrative expenses were $22.9 million for the fourth quarter of 2016, compared to $24.3 million for the same period of 2015, a decrease of $1.4 million, which was primarily driven by decreased external professional services due to lower litigation activities partially offset by increases in restructuring expense and stock-based compensation expense. Non-GAAP selling, general and administrative expenses were $16.1 million for the fourth quarter of 2016, compared to $20.7 million for the same period of 2015, a decrease of $4.6 million, which was primarily driven by decreased external professional fees due to lower litigation activities. Selling, general and administrative expenses for the year ended December 31, 2016 were $83.7 million, compared to $75.0 million for the prior year, an increase of $8.7 million, which was primarily driven by increases in compensation expenses due to increases in commercial headcount and restructuring expenses offset by decreases in severance expense related to the resignation of our former CEO in March 2015 and professional services primarily due to lower litigation activities. Non-GAAP selling, general and administrative expenses were $60.7 million for the year ended December 31, 2016, compared to $53.3 million for the same period of 2015, an increase of $7.4 million, which was primarily driven by increase in compensation expenses due to increased commercial headcount offset by decreased external professional fees due to lower litigation activities. The Company had $329.3 million in cash, cash equivalents and restricted cash and investments as of December 31, 2016 compared to $204.0 million as of December 31, 2015, an increase of $125.4 million. The increase was driven by the net proceeds received from the Company’s public offerings in June and September 2016, offset by the use of cash to fund the Company’s ongoing operations. In addition to the GAAP financial measures set forth in this press release, the Company has included certain non-GAAP measurements: non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP operating expense adjustments, non-GAAP net loss, and non-GAAP basic and diluted net loss per share, which present operating results on a basis adjusted for stock-based compensation and restructuring expenses and other items. Stock-based compensation expenses represent non-cash charges related to equity awards granted by Sarepta. Although these are recurring charges to operations, management believes the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within management's control. Therefore, management believes that excluding these charges from non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP net loss and non-GAAP net loss per share facilitates comparisons of the Company’s operational performance in different periods. Restructuring expenses have been excluded from non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP net loss and non-GAAP net loss per share as the Company believes that the adjustments for these items represent more closely the sustainability of the Company’s operating performance and financial results. Management evaluates other items of expense and income on an individual basis. It takes into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relates to the Company’s ongoing business operations, and (c) whether the Company expects the items to continue on a regular basis. These other items include the up-front and options payments related to existing collaboration and option agreements. The Company uses these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. The Company also believes these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating the Company’s performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company’s financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP operating expense adjustments, non-GAAP net loss, and non-GAAP basic and diluted net loss per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table "Reconciliation of GAAP to Non-GAAP Net Loss." -Sarepta Therapeutics Agrees to Sale of Priority Review Voucher for $125M -Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program -Sarepta Therapeutics Enters into License Agreement with Nationwide Children’s Hospital for Galgt2 Gene Therapy Program -Sarepta Therapeutics Announces EMA Validation of Eteplirsen Authorization Application for Treatment of Duchenne Muscular Dystrophy Amenable to Exon Skipping 51 Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy drug candidates. For more information, please visit us at www.sarepta.com. In order to provide Sarepta’s investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “may,” “intends,” “prepares,” “looks,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to Sarepta’s future operations, financial performance and projections, business plans, priorities and development of product candidates including: Sarepta’s plans for 2017, including executing a successful launch of EXONDYS 51 in the US, reaching more patients through global expansion and rapidly advancing Sarepta’s pipeline through internal and external development efforts, and Sarepta’s belief that it is well positioned for potential growth and towards its goal to help all boys with Duchenne muscular dystrophy. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: we may not be able to meet expectations with respect to EXONDYS 51 sales or attain profitability and positive cash-flow from operations; we may not be able to comply with all FDA post-approval commitments and requirements with respect to EXONDYS 51 in a timely manner or at all; we may not be able to complete clinical trials required by the FDA for approval of our product candidates; the results of our ongoing research and development efforts and clinical trials for our product candidates may not be positive or consistent with prior results or demonstrate a safe treatment benefit; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, bringing EXONDYS 51 to markets outside the United States and bringing our product candidates to market, for various reasons including possible limitations of Company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2016 or Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. You should not place undue reliance on forward-looking statements. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except to the extent required by applicable law or SEC rules. We routinely post information that may be important to investors in the 'For Investors' section of our web site at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

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