News Article | February 22, 2017
Thermo Fisher Scientific to develop and then commercialize its assay for measuring the concentration of plazomicin SOUTH SAN FRANCISCO, Calif., Feb. 22, 2017 (GLOBE NEWSWIRE) -- Achaogen, Inc. (NASDAQ:AKAO), a clinical-stage biopharmaceutical company developing novel antibacterials addressing multi-drug resistant (MDR) gram-negative infections, today announced that they have achieved a strategic milestone in their ongoing efforts to develop an assay enabling therapeutic drug management (TDM) of plazomicin. Achaogen is developing plazomicin for the potential treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae (CRE). In Achaogen’s Phase 3 CARE trial in patients with serious infections due to CRE, an investigational assay enabling plazomicin TDM was used to help ensure that targeted exposures of plazomicin were achieved in the critically ill patients enrolled in the trial. If plazomicin is approved, Achaogen and Thermo Fisher plan to develop and have a commercial assay for plazomicin available at product launch. Achaogen plans to submit a New Drug Application (NDA) for plazomicin to the Food and Drug Administration (FDA) in the second half of 2017. Achaogen also plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in 2018. “Thermo Fisher is a world leader in developing and providing high-quality commercial assays to achieve precise and accurate quantitative results crucial for monitoring drug levels. We are pleased with the progress made in our collaboration with Thermo Fisher and with achieving this important milestone of demonstrating assay feasibility, a key step toward enabling therapeutic drug management of plazomicin for those patients most likely to benefit from TDM,” said Blake Wise, Achaogen’s Chief Operating Officer. “In certain high-risk patient populations, such as the critically ill, we believe TDM has the potential to provide significant utility in optimizing plazomicin dosing.” The two companies have been collaborating on assay development since 2015. Under terms of their collaboration agreement, Thermo Fisher leads the development, regulatory approval, and commercialization of an assay for measuring plazomicin drug levels. Achaogen brings plazomicin expertise to the collaboration, including the discovery of plazomicin-specific antibodies utilizing their state-of-the-art antibody discovery platform. “The agreement reflects our mutual commitment to providing a broadly-available plazomicin assay at launch so that healthcare providers can measure plazomicin drug levels in critically ill patients with bacterial infections,” said John Kody, Vice President/General Manager Clinical Diagnostics - Niche Products at Thermo Fisher Scientific. “During the feasibility period, our two teams have developed an assay that compares very well to traditional analytical chemistry techniques. Thermo Fisher’s QMS™ TDM assays are conveniently ready-to-use, with excellent precision and accuracy, and are optimized for performance on a wide range of analyzers.” In December 2016, Achaogen announced positive results from its plazomicin Phase 3 clinical trials in complicated urinary tract infections (cUTI) and infections due to CRE. In the Phase 3 EPIC registration trial in patients with cUTI and acute pyelonephritis (AP), plazomicin met the objective of non-inferiority compared to meropenem for FDA-specified primary efficacy endpoints, and achieved superiority for the EMA-specified primary efficacy endpoints. In addition, in the Phase 3 CARE trial in patients with serious infections due to CRE, a lower rate of mortality or serious disease-related complications was observed for plazomicin-treated patients compared with those on colistin therapy. In the Phase 3 CARE trial, TDM with an investigational assay helped to confirm that the targeted-exposure of plazomicin was achieved in these critically ill patients. About Therapeutic Drug Management and Plazomicin Therapeutic Drug Management is the practice of measuring the concentration of medication in blood and adjusting the dose of that drug based on the results. Healthcare providers routinely use TDM for certain drugs to help improve patient care by individually adjusting the dose as appropriate. Critically ill patients with bacterial infections often have abnormal and fluctuating renal function as well as an altered volume of drug distribution in the body. This can lead to these patients being either under or over-dosed with what are potentially life-saving therapies. Initial data from Achaogen’s plazomicin CARE study in critically ill patients with CRE infections confirmed drug concentration variability within and among patients and importantly, showed that TDM helped to ensure that the targeted-exposure of plazomicin was achieved in these patients. About Achaogen Achaogen is a clinical-stage biopharmaceutical company passionately committed to the discovery, development, and commercialization of novel antibacterials to treat MDR gram-negative infections. Achaogen is developing plazomicin, Achaogen’s lead product candidate, for the treatment of serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae. Achaogen’s plazomicin program is funded in part with Federal funds from the Biomedical Advanced Research and Development Authority, Office of the Assistant Secretary for Preparedness and Response, Office of the Secretary, Department of Health and Human Services, under Contract No. HHSO100201000046C. Plazomicin is the first clinical candidate from Achaogen’s gram-negative antibiotic discovery engine. Achaogen has other programs in early and late preclinical stages focused on other MDR gram-negative infections, including LpxC inhibitors for the treatment of serious bacterial infections including MDR gram-negative bacteria. Achaogen's LpxC inhibitor program has been funded in part with Federal funds from the National Institute of Allergy and Infectious Diseases, National Institutes of Health, Department of Health and Human Services, under Contract No. HHSN272201500009C. LpxC inhibitors are the second class of molecules from Achaogen's gram-negative antibiotic discovery engine. For more information, please visit www.achaogen.com. Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical facts contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, Achaogen’s expectations regarding potential regulatory approval of plazomicin, Achaogen’s commercial objectives and Achaogen’s pipeline of product candidates. Such forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause Achaogen's actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the preclinical and clinical development process; the risk of failure to successfully validate, develop and obtain regulatory clearance or approval for the in vitro diagnostic (IVD) assay for plazomicin; the risks and uncertainties of the regulatory approval process; the risks and uncertainties of commercialization and gaining market acceptance; the risk when bacteria will evolve resistance to plazomicin; Achaogen's reliance on third-party contract manufacturing organizations to manufacture and supply its product candidates and certain raw materials used in the production thereof; risk of third party claims alleging infringement of patents and proprietary rights or seeking to invalidate Achaogen's patents or proprietary rights; and the risk that Achaogen's proprietary rights may be insufficient to protect its technologies and product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward- looking statements, as well as risks relating to Achaogen's business in general, see Achaogen's current and future reports filed with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, and its Annual Report on Form 10-K for the fiscal year ended December 31, 2015. Achaogen does not plan to publicly update or revise any forward-looking statements contained in this press release, whether as a result of any new information, future events, changed circumstances or otherwise.
News Article | February 20, 2017
Bangalore, India, February 19, 2017 --( The inspection confirmed the site to be compliant with principles of current Good Manufacturing Practices (cGMPs) and no Form 483 observations were issued. The audit was triggered by an ANDA filed for a customer. This facility is part of Kemwell’s pharmaceutical business for which Kemwell has signed a purchase agreement with Recipharm. “We are proud of the result achieved during the FDA inspection. In the last 10 years we have successfully faced 3 USFDA and 3 EMA inspections. This success is testament to our on-going commitment towards our quality systems,” says Anurag Bagaria, Chairman. “We are now preparing for our first Japanese inspection in the same facility and our first EMA/FDA inspection in our biotech facilities.” Kemwell will continue to leverage its 30-year experience in being a leading contract development and manufacturing service provider to provide services in the biotech space. Its’ biotech facilities are the largest contract manufacturing facilities in India. With 2x2000L of cGMP cell culture capacity along with process development and drug product manufacturing (liquid and lyophilized vials) Kemwell is an end-to-end service provider for mAbs and therapeutic proteins. About Kemwell: Kemwell Biopharma, is a 100% CDMO that provides customized product development and cGMP compliant manufacturing solutions to pharmaceutical and biopharmaceutical organizations worldwide. Kemwell delivers a full product portfolio of services for sterile and non-sterile products from preformulation to commercial supply. Kemwell facilities have been inspected by USFDA, EMA, ANVISA and other health authorities. Founded in 1980, Kemwell employs 1200 people in India. Read more about Kemwell on For further queries please contact: Karan Bagaria, VP Corporate Development or +91-80-39285450 Bangalore, India, February 19, 2017 --( PR.com )-- Kemwell Biopharma, a global bio/pharmaceutical contract development and manufacturing company, announced today that its oral solids manufacturing facility located in Bangalore, India has successfully completed a U.S. Food and Drug Administration (USFDA) inspection.The inspection confirmed the site to be compliant with principles of current Good Manufacturing Practices (cGMPs) and no Form 483 observations were issued. The audit was triggered by an ANDA filed for a customer. This facility is part of Kemwell’s pharmaceutical business for which Kemwell has signed a purchase agreement with Recipharm.“We are proud of the result achieved during the FDA inspection. In the last 10 years we have successfully faced 3 USFDA and 3 EMA inspections. This success is testament to our on-going commitment towards our quality systems,” says Anurag Bagaria, Chairman. “We are now preparing for our first Japanese inspection in the same facility and our first EMA/FDA inspection in our biotech facilities.”Kemwell will continue to leverage its 30-year experience in being a leading contract development and manufacturing service provider to provide services in the biotech space. Its’ biotech facilities are the largest contract manufacturing facilities in India. With 2x2000L of cGMP cell culture capacity along with process development and drug product manufacturing (liquid and lyophilized vials) Kemwell is an end-to-end service provider for mAbs and therapeutic proteins.About Kemwell:Kemwell Biopharma, is a 100% CDMO that provides customized product development and cGMP compliant manufacturing solutions to pharmaceutical and biopharmaceutical organizations worldwide. Kemwell delivers a full product portfolio of services for sterile and non-sterile products from preformulation to commercial supply. Kemwell facilities have been inspected by USFDA, EMA, ANVISA and other health authorities. Founded in 1980, Kemwell employs 1200 people in India.Read more about Kemwell on www.kemwellbiopharma.com For further queries please contact:Karan Bagaria, VP Corporate Development firstname.lastname@example.org or +91-80-39285450 Click here to view the list of recent Press Releases from Kemwell Biopharma Private Limited
News Article | February 22, 2017
SMis Paediatric Clinical Trials show returns to London for the 11th year on 20th-21st March 2017. London, United Kingdom, February 22, 2017 --( Through a series of interactive conference sessions, presentations and a workshop led by industry experts; the 2017 agenda will discuss current clinical trials, implementation, drug development, recruitment and retention, ethical issues and regulations. Key presentations not to be missed: - Bianca McDade, Director Regulatory Affairs, GSK - Tom Willgoss, Senior Outcomes Research Scientist, Patient-Centred Outcomes Research, Roche - Karl-Heinz Huemer, Scientific Committee Member and Expert, EMA, PDCO - Hernando Patino, Paediatric Drug Development Lead, Johnson & Johnson - Deborah Lee, VP Clinical Development, Insys Therapeutics - Andy Kenwright, Senior Statistical Scientist, Roche - Robert Kahn, Former Senior Safety Science Leader, Global Pediatric Oncology, Genentech Highlights for 2017: - Update from the EMA on the PDCO's 10 year review into paediatric investigation plans - Discuss clinical trial legislation in the EU and US - Review challenges in paediatric drug development for rare diseases - Optimise approaches to paediatric drug formulation to improve clinical success - Evaluate recruitment and retention - Discuss hot topic of data extrapolation In the lead up to the event SMi have released some pre-conference interviews with some of the speakers. For further insight into the topics being discussed at this year’s conference and an overall look into the paediatric trials field visit the download centre of the event website to access the 2017 speaker interview series. Interviews available to download include: Roche, Insys Therapeutics, Paediatric Research Consultancy, The Birmingham Children’s Hospital and Klausrose Consulting. Countries attending Paediatric Clinical Trials 2017 include: Australia, Austria, Belgium, Denmark, France, Germany, Netherlands, Spain, Switzerland, United Kingdom & USA. For those who are interested in attending register online at the event website www.paediatric-trials.co.uk/prcom Paediatric Clinical Trials 20-21 March 2017 Copthorne Tara Hotel, London, UK www.paediatric-trials.co.uk/prcom Sponsorship enquiries: Contact Alia Malick on: +44 (0) 20 7827 6168 or email email@example.com Group bookings: Contact Ameenah Begum on: +44 (0) 20 7827 6166 or email firstname.lastname@example.org About SMi Group: Established since 1993, the SMi Group is a global event-production company that specializes in Business-to-Business Conferences, Workshops, Masterclasses and online Communities. We create and deliver events in the Defence, Security, Energy, Utilities, Finance and Pharmaceutical industries. We pride ourselves on having access to the world’s most forward thinking opinion leaders and visionaries, allowing us to bring our communities together to Learn, Engage, Share and Network. More information can be found at http://www.smi-online.co.uk London, United Kingdom, February 22, 2017 --( PR.com )-- With just 4 weeks to go, registration will be closing soon for SMi’s Paediatric Clinical Trials 2017. The event will bring together Clinical Operations Leads and Heads of Clinical Trials to review the developments leading to the advancement of paediatric medicines.Through a series of interactive conference sessions, presentations and a workshop led by industry experts; the 2017 agenda will discuss current clinical trials, implementation, drug development, recruitment and retention, ethical issues and regulations.Key presentations not to be missed:- Bianca McDade, Director Regulatory Affairs, GSK- Tom Willgoss, Senior Outcomes Research Scientist, Patient-Centred Outcomes Research, Roche- Karl-Heinz Huemer, Scientific Committee Member and Expert, EMA, PDCO- Hernando Patino, Paediatric Drug Development Lead, Johnson & Johnson- Deborah Lee, VP Clinical Development, Insys Therapeutics- Andy Kenwright, Senior Statistical Scientist, Roche- Robert Kahn, Former Senior Safety Science Leader, Global Pediatric Oncology, GenentechHighlights for 2017:- Update from the EMA on the PDCO's 10 year review into paediatric investigation plans- Discuss clinical trial legislation in the EU and US- Review challenges in paediatric drug development for rare diseases- Optimise approaches to paediatric drug formulation to improve clinical success- Evaluate recruitment and retention- Discuss hot topic of data extrapolationIn the lead up to the event SMi have released some pre-conference interviews with some of the speakers. For further insight into the topics being discussed at this year’s conference and an overall look into the paediatric trials field visit the download centre of the event website to access the 2017 speaker interview series. Interviews available to download include: Roche, Insys Therapeutics, Paediatric Research Consultancy, The Birmingham Children’s Hospital and Klausrose Consulting.Countries attending Paediatric Clinical Trials 2017 include: Australia, Austria, Belgium, Denmark, France, Germany, Netherlands, Spain, Switzerland, United Kingdom & USA. For those who are interested in attending register online at the event website www.paediatric-trials.co.uk/prcomPaediatric Clinical Trials20-21 March 2017Copthorne Tara Hotel, London, UKwww.paediatric-trials.co.uk/prcomSponsorship enquiries: Contact Alia Malick on: +44 (0) 20 7827 6168 or email email@example.comGroup bookings: Contact Ameenah Begum on: +44 (0) 20 7827 6166 or email firstname.lastname@example.orgAbout SMi Group:Established since 1993, the SMi Group is a global event-production company that specializes in Business-to-Business Conferences, Workshops, Masterclasses and online Communities. We create and deliver events in the Defence, Security, Energy, Utilities, Finance and Pharmaceutical industries. We pride ourselves on having access to the world’s most forward thinking opinion leaders and visionaries, allowing us to bring our communities together to Learn, Engage, Share and Network. More information can be found at http://www.smi-online.co.uk Click here to view the list of recent Press Releases from SMi Group
News Article | February 2, 2017
Sports Direct’s Mike Ashley and French Connection’s Stephen Marks both don’t enjoy being told how to run their business. Thus excellent entertainment could be in prospect now that Sports Direct has bought control of an 11% stake in the struggling fashion chain. Marks, who owns 42% of French Connection, is already sparring with a hedge fund that thinks he shouldn’t be both chairman and chief executive of a company that has made losses for five years in a row. Ashley’s arrival in the ring makes the script more unpredictable. Read nothing into French Connection’s statement that it sees the purchase “as a vote of confidence in the true potential of our company”. Ashley is not a sit-and-hold merchant. But, while we sit back and await developments, Sports Direct’s outside shareholders may not be so relaxed. Ashley is supposed to be running a sportswear retailer, not a speculative investment fund. He owns 56% of Sports Direct but the owners of the other 44% are quite capable of forming their own view of French Connection’s prospects. The funds for this adventure are the company’s, not Ashley’s, remember. He’s done this sort of thing in the past – everywhere from Tesco to House of Fraser to Debenhams. The (thin) justification is usually that the “investments” somehow serve Sports Direct’s broader strategic aims. At a push, one could construct an elaborate theory that a relationship with French Connection could help Sports Direct to get bigger in fashion. But, come on, you don’t need to buy a big block of loose shares to open a commercial discussion. It seems more likely that Ashley simply can’t resist a flutter when he spies a lively situation. It’s almost as if he has heard the objections to past share-buying adventures, plus shareholders’ multiple other governance worries, and decided to send a message that he doesn’t give a fcuk. If your shares have performed like Reckitt Benckiser’s – £24 to £71 in the past decade – investors are inclined to smile on any big proposed acquisition. Sure enough, the share price improved 4% as Reckitt said it is in advanced discussions to buy Mead Johnson Nutrition, a US baby milk firm, for $16.7bn (£13.3bn) in cash. But isn’t this a very un-Reckitt-like deal? One could say that baby milk falls within Reckitt’s “consumer healthcare” bucket, even if the current infant-related range seems to run to little more than the kiddie formulation of Nurofen. Or perhaps the appeal of Mead is the fact that it’s big in China and the rest of Asia, territory that all consumer goods giants are inclined to want to populate. But, at $16.7bn, plus a billion if you include Mead’s borrowings, this deal would be a major departure from Reckitt’s old diet of small purchases that can be slotted seamlessly into a reliable distribution network. Nurofen, Durex condoms and the Scholl footwear brand fitted that bill. With baby milk, Reckitt would be up against the commanding marketing budgets of Nestlé and Danone. Then there is the proposed takeover price of $90 a share. Mead expects to earn $3.05-$3.20 a share in 2017, so Reckitt would be paying about 29 times earnings, which is enormous. It would be easier to justify if the target was growing rapidly or carrying fat. But Mead’s sales, measured at constant currencies, fell 3% last year, and were even down in Asia. Profit margins are already 24%, which doesn’t obviously suggest inefficiency. Rakesh Kapoor, Reckitt’s chief executive, has been warming up his investors for a big move for a while but they thought he had in mind an unloved consumer healthcare division of a big pharmaceutical firm. Mead isn’t that, and yet Reckitt is seemingly happy to push its own borrowing ratios to the maximum to buy it. It all suggests Kapoor failed to find what he really wants to buy and has settled for second-best. When he is free to speak, he will offer counter-arguments, but investors’ lack of initial scepticism is odd. Just because it’s Reckitt, it doesn’t mean the deal must be brilliant. Aren’t our big pharmaceutical firms meant to be depressed about Brexit and the prospect of the European Medicines Agency (EMA), the pan-EU regulator, moving out of London? Pascal Soriot, chief executive of AstraZeneca, didn’t exactly sound excited but he made a couple of interesting points about the EMA’s departure, which he views as inevitable. First, it will be important for the new UK body and EMA to work collaboratively. “That should be possible because it is in the interests of everybody and all the regulatory and scientific standards today are common,” he said. Second, he argued the UK could be more “agile” in encouraging innovation. “If it’s done well, it could be helpful,” he said. That is a qualified remark but it is another welcome sign of pragmatism on Brexit from British business. It’s the only way, unfortunately.
News Article | February 28, 2017
MOUNTAIN VIEW, Calif., Feb. 28, 2017 (GLOBE NEWSWIRE) -- ChemoCentryx, Inc., (Nasdaq:CCXI), today announced that Thomas J. Schall, Ph.D., President and Chief Executive Officer, will present at the Cowen and Company 37th Annual Health Care Conference on Tuesday, March 7, 2017 at 10:00 a.m. ET. The conference will be held at the Boston Marriott Copley Place Hotel in Boston, MA. A live audio webcast of the presentation can be accessed through the Investors section of the Company's website at www.ChemoCentryx.com. A replay of the webcast will be available on the Company's website for two weeks following the live presentation. ChemoCentryx is a biopharmaceutical company developing new medications targeted at inflammatory and autoimmune diseases, and cancer. ChemoCentryx targets the chemokine and chemoattractant systems to discover, develop and commercialize orally-administered therapies. ChemoCentryx is currently focusing on its late stage drug candidates for patients with rare kidney diseases, avacopan (CCX168) and CCX140. Avacopan is an orally-administered small molecule that is a selective inhibitor of the complement C5a receptor, or C5aR. Avacopan is in Phase III development for the treatment of anti-neutrophil cytoplasmic auto-antibody-associated vasculitis (AAV). In clinical studies to date, avacopan was shown to be safe, well tolerated and provided effective control of the disease while allowing elimination of high-dose steroids, part of the current standard of care. Avacopan is also being developed in patients with C3 glomerulopathy (C3G) and atypical hemolytic uremic syndrome (aHUS). The U.S. Food and Drug Administration has granted avacopan orphan-drug designation for AAV, and aHUS. The European Commission has granted orphan medicinal product designation for avacopan for the treatment of two forms of AAV: microscopic polyangiitis and granulomatosis with polyangiitis (formerly known as Wegener's granulomatosis). Avacopan was also granted access to the European Medicines Agency's (EMA) PRIority MEdicines (PRIME) initiative, which supports accelerated assessment of investigational therapies addressing unmet medical need. The Company’s other late stage drug candidate is CCX140, an inhibitor of the chemokine receptor known as CCR2, which is currently being developed for patients with focal segmental glomerulosclerosis (FSGS), a debilitating kidney disease. ChemoCentryx’s Kidney Health Alliance with Vifor Pharma provides Vifor Pharma with exclusive rights to commercialize avacopan and CCX140 in markets outside of the U.S. and China. ChemoCentryx also has early stage drug candidates that target chemoattractant receptors in other Inflammatory and autoimmune diseases and in cancer.
News Article | February 24, 2017
Basel, February 24, 2017 - Novartis today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending approval of Tafinlar® (dabrafenib) in combination with Mekinist® (trametinib) to treat patients with advanced or metastatic non-small cell lung cancer (NSCLC) whose tumors express the BRAF V600 mutation. If approved, Tafinlar + Mekinist will be the first targeted treatment available for patients with BRAF V600-positive NSCLC. Of the estimated 1.8 million new cases of lung cancer diagnosed worldwide each year, 1-3%, may be driven by the BRAF mutation. "At Novartis, we are committed to finding treatments for rare cancers with an unmet need. Today's CHMP opinion marks a major milestone for NSCLC patients with the BRAF V600 mutation, who have very limited treatment options," said Bruno Strigini, CEO, Novartis Oncology. "We welcome the CHMP's opinion as a first step towards that goal, and look forward to continuing to work with European health authorities to make Tafinlar + Mekinist available for appropriate NSCLC patients." The positive CHMP opinion was based on safety and efficacy data from a Phase II study of Tafinlar + Mekinist in patients with BRAF V600-positive NSCLC (36 treatment-naïve and 57 previously treated with chemotherapy). The 57 patients who had tumor progression on at least one platinum based chemotherapy, receiving 150 mg of Tafinlar twice daily and 2 mg of Mekinist once daily, demonstrated an overall response rate (ORR) of 63.2% (95% confidence interval [CI], 49.3%, 75.6%) and duration of response of 9.0 months (95% CI, 6.9, 18.3 months). The most common adverse events (incidence >20%) were pyrexia, nausea, vomiting, diarrhea, asthenia, decreased appetite, dry skin, chills, peripheral edema, cough and rash. Updated data from the previously treated and treatment-naïve cohorts were included in the overall data package for EMA review and will also be presented at upcoming medical meetings. The European Commission (EC) typically adheres to the recommendation of the CHMP and usually delivers its final decision within two months. The decision will be applicable to all 28 European Union (EU) member states plus Iceland and Norway. In Europe, Tafinlar and Mekinist is approved for the treatment of patients with unresectable or metastatic melanoma who have a BRAF V600 mutation. The US Food and Drug Administration (FDA) granted Tafinlar + Mekinist Breakthrough Therapy Designation for advanced or metastatic BRAF V600-positive NSCLC patients in 2015 and Priority Review in November 2016. Combination use of Tafinlar + Mekinist is also approved in the US, Australia, Canada and additional countries for patients with unresectable or metastatic melanoma whose tumors tested positive for the BRAF V600 mutation. Worldwide, lung cancer causes more deaths than colon, breast, and prostate cancer combined, and an estimated 1.8 million new cases of lung cancer are diagnosed each year. Among patients with NSCLC, roughly 30% have an actionable mutation that may be targeted with available therapies,,,. To determine that treatment, medical organizations recommend genetic testing for patients with lung cancer. Novartis Commitment to Lung Cancer Novartis Oncology's research into targeted therapies has helped transform treatment approaches for patients living with mutation-driven types of lung cancer. Patients with a mutation-driven NSCLC may be candidates for treatment with targeted therapies. Novartis continues its commitment to the global lung cancer community through ongoing studies, as well as the exploration of investigational compounds that target genetic biomarkers in NSCLC. About Tafinlar + Mekinist Combination Combination use of Tafinlar + Mekinist in patients with unresectable or metastatic melanoma who have a BRAF V600 mutation is approved in the US, EU, Australia, Canada and other countries. Tafinlar and Mekinist target different kinases within the serine/threonine kinase family - BRAF and MEK1/2, respectively - in the RAS/RAF/MEK/ERK pathway, which is implicated in non-small cell lung cancer (NSCLC) and melanoma, among other cancers. When Tafinlar is used with Mekinist, the combination has been shown to slow tumor growth more than either drug alone. The combination of Tafinlar + Mekinist is currently being investigated in an ongoing clinical trial program across a range of tumor types conducted in study centers worldwide. The safety and efficacy profile of the Tafinlar + Mekinist combination has not yet been established outside of the approved indications. Tafinlar and Mekinist are also indicated in more than 40 countries worldwide, including the US and EU, as single agents to treat patients with unresectable or metastatic melanoma with a BRAF V600 mutation. Tafinlar + Mekinist combination may cause serious side effects. Tafinlar in combination with Mekinist should only be used to treat melanoma with a change (mutation) in the BRAF gene; therefore, doctors should test their patients before treatment, as patients without a BRAF mutation and with a RAS mutation can be at risk of increased cell proliferation in the presence of a BRAF inhibitor. Doctors should also consider other treatment options for their patients if they had been previously treated with a BRAF inhibitor as single agent, as the limited data available have shown that the efficacy of Tafinlar + Mekinist is lower in these patients. When Tafinlar is used in combination with Mekinist, or when Tafinlar is administered as monotherapy, it can cause new cancers (both skin cancer and non-skin cancer). Patients should be advised to contact their doctor immediately for any new lesions, changes to existing lesions on their skin, or signs and symptoms of other malignancies. Tafinlar in combination with Mekinist, or Mekinist alone, can cause severe bleeding, and in some cases can lead to death. Patients should be advised to call their healthcare provider and get medical help right away if they have headaches, dizziness, or feel weak, cough up blood or blood clots, vomit blood or their vomit looks like "coffee grounds," have red or black stools that look like tar, or any unusual signs of bleeding. Tafinlar in combination with Mekinist, or either drug alone, can cause severe eye problems that can lead to blindness. Patients should be advised to call their healthcare provider right away if they get these symptoms of eye problems: blurred vision, loss of vision, or other vision changes, seeing color dots, halo (seeing blurred outline around objects), eye pain, swelling, or redness. Tafinlar in combination with Mekinist, or Tafinlar alone, can cause fever which may be serious. When taking Tafinlar in combination with Mekinist, fever may happen more often or may be more severe. In some cases, chills or shaking chills, too much fluid loss (dehydration), low blood pressure, dizziness, or kidney problems may happen with the fever. Patients should be advised to call their healthcare provider right away if they get a fever above 38.5oC (101.3oF) while taking Tafinlar. Tafinlar in combination with Mekinist, or Mekinist alone, can affect how well the heart pumps blood. A patient's heart function should be checked before and during treatment. Patients should be advised to call their healthcare provider right away if they have any of the following signs and symptoms of a heart problem: feeling like their heart is pounding or racing, shortness of breath, swelling of their ankles and feet, or feeling lightheaded. Tafinlar in combination with Mekinist, or Tafinlar alone, can cause abnormal kidney function or inflammation of the kidney. Abnormal kidney function may happen more often for patients with fever or too much fluid loss. Patients should be advised to call their healthcare provider right away if they have a fever above 38.5oC (101.3oF), decreased urine, fatigue, loss of appetite or discomfort in lower abdomen or back. Tafinlar has not been studied in patients with renal insufficiency (defined as creatinine > 1.5 x ULN) therefore caution should be used in this setting. Tafinlar in combination with Mekinist, or Mekinist alone, can cause abnormal liver function. A patient may feel tired, lose appetite, yellow skin, dark urine colour, or discomfort in abdomen. The liver function abnormality needs to be assessed by laboratory test of the blood. Patients should consult their healthcare provider if they have such experience. Administration of Tafinlar or Mekinist should be done with caution in patients with moderate to severe hepatic impairment. Elevations in blood pressure have been reported in association with Mekinist in combination with Tafinlar, or with Mekinist alone, in patients with or without pre-existing hypertension. Patients should be advised to monitor blood pressure during treatment with Mekinist and control potential hypertension by standard therapy, as appropriate. Tafinlar in combination with Mekinist, or Mekinist alone, can cause inflammation of the lung tissue. Patients should notify their doctor if they experience any new or worsening symptoms of lung or breathing problems, including shortness of breath or cough. Rash is a common side effect of Tafinlar in combination with Mekinist, or with Mekinist alone. Tafinlar in combination with Mekinist, or Mekinist alone, can also cause other skin reactions which can be severe, and may need to be treated in a hospital. Patients should be advised to call their healthcare provider if they get any of the following symptoms: skin rash that bothers them or does not go away, acne, redness, swelling, peeling, or tenderness of hands or feet, skin redness. Tafinlar in combination with Mekinist, or Mekinist alone, can cause muscle breakdown, a condition called Rhabdomyolysis. Patients experiencing muscle pain, tenderness, weakness or a swelling of their muscles should contact their healthcare provider immediately. Tafinlar in combination with Mekinist, or Tafinlar alone, can uncommonly cause an inflammation of the pancreas (pancreatitis). Patients should be promptly investigated if they experience unexplained abdominal pain and closely monitored if they re-start Tafinlar after a prior episode of pancreatitis. Tafinlar in combination with Mekinist, or Mekinist alone, can cause blood clots in the arms or legs, which can travel to the lungs and can lead to death. Patients should be advised to get medical help right away if they have the following symptoms: chest pain, sudden shortness of breath or trouble breathing, pain in their legs with or without swelling, swelling in their arms or legs, or a cool or pale arm or leg. Mekinist, alone or in combination with Tafinlar, may increase the risk of developing holes in the stomach or intestine (gastrointestinal perforation). Treatment with Mekinist alone or in combination with Tafinlar should be used with caution in patients with risk factors for gastrointestinal perforation, including concomitant use of medications with a recognised risk of gastrointestinal perforation. Tafinlar and Mekinist both can cause harm to an unborn baby when taken by a pregnant woman. Tafinlar can also render hormonal contraceptives ineffective. The most common side effects of Tafinlar + Mekinist combination include fever, tiredness, nausea, headache, chills, diarrhea, rash, joint pain, high blood pressure, vomiting and cough. The incidence and severity of fever is increased when Mekinist is used in combination with Tafinlar. Patients should tell their doctor of any side effect that bothers them or does not go away. These are not all of the possible side effects of Tafinlar + Mekinist combination. For more information, patients should ask their doctor or pharmacist. Patients should take Tafinlar + Mekinist combination exactly as their health care provider tells them. Patients should not change their dose or stop taking Tafinlar + Mekinist combination unless their health care provider advises them to. Mekinist should be taken only once daily (either in the morning or evening, at the same time as Tafinlar). The first and second doses of Tafinlar should be taken approximately 12 hours apart. Patients should take Tafinlar + Mekinist at least 1 hour before or 2 hours after a meal. Do not take a missed dose of Tafinlar within 6 hours of the next dose of Tafinlar. Do not open, crush, or break Tafinlar capsules. Do not take a missed dose of Mekinist within 12 hours of the next dose of Mekinist. Please see full Prescribing Information for Tafinlar and Mekinist. Disclaimer The foregoing release contains forward-looking statements that can be identified by words such as "positive CHMP opinion," "will," "recommending," "committed," "first step," "goal," "look forward," "upcoming," "recommendation," "usually," "Breakthrough Therapy designation," "Priority Review," "commitment," "may," "ongoing," "investigational," "being investigated," "yet," or similar terms, or by express or implied discussions regarding potential new indications or labeling for Tafinlar + Mekinist, or regarding potential future revenues from Tafinlar and Mekinist, both as single agents and in combination with the other. You should not place undue reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that Tafinlar + Mekinist will be submitted or approved for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that Tafinlar and Mekinist, either as single agents or in combination with the other will be commercially successful in the future. In particular, management's expectations regarding Tafinlar and Mekinist, both as single agents and in combination with the other could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain proprietary intellectual property protection; general economic and industry conditions; global trends toward health care cost containment, including ongoing pricing pressures; safety, quality or manufacturing issues, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. About Novartis Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has leading positions globally in each of these areas. In 2016, the Group achieved net sales of USD 48.5 billion, while R&D throughout the Group amounted to approximately USD 9.0 billion. Novartis Group companies employ approximately 118,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world. For more information, please visit http://www.novartis.com. Novartis is on Twitter. Sign up to follow @Novartis at http://twitter.com/novartis and @NovartisCancer at http://twiter.com/novartiscancer For Novartis multimedia content, please visit www.novartis.com/news/media-library For questions about the site or required registration, please contact email@example.com  Planchard D et al. Dabrafenib plus trametinib in patients with previously treated BRAFV600E-mutant metastatic non-small cell lung cancer: An open-label, multicentre Phase II trial. 2016. Lancet Oncol 17: 984-93.  World Health Organization. International Agency for Research on Cancer. GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide in 2012. Lung Cancer. Available at http://globocan.iarc.fr/Pages/fact_sheets_cancer.aspx?cancer=lung. Accessed February 2, 2017.  Pao W, Girard N. New driver mutations in non-small-cell lung cancer. 2011. Lancet Oncol 12: 175-180.  World Health Organization. Estimated number of deaths, both sexes, worldwide in 2012. World Health Organization. http://gco.iarc.fr/today/online-analysis-pie?mode=cancer&mode_population= continents&population=900&sex=0&cancer=11&type=1&statistic=0&prevalence=0&color _palette=default. Accessed on January 19, 2017.  Riess JW, Wakelee, HA. Metastatic Non-Small Cell Lung Cancer Management: Novel Targets and Recent Clinical Advances. Clinical Advances in Hematology & Oncology. 2012; 10: 226-224.  Pao W, Girard N. New driver mutations in non-small-cell lung cancer. Lancet Oncol. 2011;12:175-180.  Paik PK, Arcila ME, Fara M, et al. Clinical Characteristics of Patients With Lung Adenocarcinomas Harboring BRAF Mutations. J Clin Oncol. 2011;29:2046-2051.  Takeuchi, K, Soda M, Togashi Y, et al. RET, ROS1 and ALK fusions in lung cancer. Nature. 2012;378-381.  Lindeman, N.I., et al. Molecular Testing Guideline for Selection of Lung Cancer Patients for EGFR and ALK Tyrosine Kinase Inhibitors. Arch Pathol Lab Med. 2013; 137: 828-1174.
News Article | March 1, 2017
- Trial 006 demonstrated a statistically significant and clinically meaningful reduction in OFF-time and increase in proportion of patients “ON” by 8:00 am (primary and key secondary endpoints) - - The trial also showed statistically significant reduction in troublesome dyskinesia and a complete reduction of OFF-time to zero hours in 66% of responders (post hoc sub-groups analyses) - - Company to host conference call and webcast today at 8:30 a.m. ET- REHOVOT, Israel, March 01, 2017 (GLOBE NEWSWIRE) -- NeuroDerm Ltd. (Nasdaq:NDRM), a clinical stage pharmaceutical company developing drug-device combinations for central nervous system (CNS) disorders, today announced that a preliminary analysis of trial 006 demonstrated that the trial successfully met its primary, key secondary and additional secondary endpoints. Trial 006 was an international open label, blinded rater, phase II study of ND0612H, NeuroDerm's high dose continuous, subcutaneously delivered levodopa/carbidopa (LD/CD) liquid formulation, in patients with advanced Parkinson's disease. NeuroDerm also announced that it has modified its EU clinical and regulatory development strategy following a meeting with the European Medicines Agency (EMA). The modified strategy will be based on NeuroDerm's restarted and amended iNDiGO phase III efficacy study (trial 007), rather than on bioequivalent pharmacokinetic (PK) studies. Based on discussions with the EMA, NeuroDerm believes that this strategy should allow it to pursue a broader EU label, with no effect on its clinical and regulatory timelines. “The very prominent responder effect, as well as the significant reductions in OFF-time and troublesome dyskinesia observed in trial 006, are extremely encouraging and demonstrate the substantial potential for ND0612 to make a meaningful difference in the lives of patients living with Parkinson’s disease,” said Oded S. Lieberman, PhD, CEO of NeuroDerm. “We believe that restarting and amending the iNDiGO trial, incorporating both an ND0612H arm and new endpoints that reflect these very positive preliminary results from trial 006, should support a broader label in the EU and increase the clinical and commercial potential of ND0612 while not affecting our clinical timelines. We are committed to improving the lives of Parkinson's patients by achieving our clinical and regulatory objectives as quickly as possible and providing Parkinson’s disease patients with a safer and more effective alternative to current treatment options.” Trial 006 Endpoints The primary endpoint of this study was to assess the change from baseline to day 28 in daily OFF-time (normalized to 16 waking hours) as assessed by a blinded rater. A key secondary endpoint was to assess the percentage of subjects who were “ON” by 8:00am and 9:00am. Additional secondary endpoints were also evaluated as well as safety and tolerability. Trial 006 Design Trial 006 was a 28-day multicenter, international (US, EU and Israel), parallel-group, blinded rater, randomized phase II study that investigated the efficacy, safety, tolerability and pharmacokinetics of two dosing regimens (R1 and R2) of ND0612H and compared them to the baseline of standard optimized oral therapy: All patients could add oral LD/CD therapy at any time as needed. The trial enrolled 38 patients with advanced Parkinson’s disease. Trial 006 Preliminary Results The 38 enrolled subjects had typical characteristics for patients with advanced Parkinson’s disease including: an average age of 63.5 years, 11.5 years since diagnosis and an average baseline OFF-time of 5.3 hours per day. OFF-time (primary endpoint): The primary endpoint was met in R1. From 5.5 hours at baseline, the OFF-time was reduced by 2.8 hours (p equals 0.004). There was a smaller, non-statistically significant reduction of 1.3 hours in OFF-time in R2. “ON” by 8:00am and 9:00am (key secondary endpoint): In R1, the proportion of patients who achieved the first “ON” by 8:00am (as reported by the patient) increased from 11% at baseline to 50% by day 28 (p equals 0.020), and, by 9:00am, from 26% at baseline to 75% (p equals 0.004). In R2, dosing began in the morning and there was therefore no improvement from baseline at either timepoint. Complete reduction of OFF-time (post-hoc analysis): In R1, 42% of patients had a complete reduction in OFF-time to zero hours (in R2, 11% experienced complete resolution of OFF-time). Patients who experienced reduction in OFF-time (greater than 0 hours change) during the trial were defined as “Responders” and constituted 68% of all patients (12 patients in R1 and 14 patients in R2). Eight (66%) of the Responders in R1 (and two (14%) of the Responders in R2) experienced a complete reduction of their OFF-time to zero hours; all Responders in R1 experienced a reduction of more than 50% in their OFF-time. “Good” ON (secondary endpoint): Good ON (defined as “ON” with no or mild dyskinesia, as assessed by the blinded rater) increased in R1 from 9.2 hours by 3.7 hours (p less than 0.001), and in R2 from 8.5 hours by 2.8 hours (p equals 0.003). Unified Parkinson’s Disease Rating Scale (UPDRS) III by 8:00am (post-hoc analysis): UPDRS III score by 8:00am decreased in R1 from 37.4 at baseline by 19.1 points (p less than 0.001) and from 37.3 at baseline by 10.7 points in R2 (p equals 0.001). Troublesome Dyskinesia (post hoc analysis): Troublesome dyskinesia (defined as “ON” with moderate or severe dyskinesia as assessed by the blinded rater) decreased from 5.1 hours at baseline by 3.5 hours (p equals 0.011) in the subgroup of all patients who had at least 1 hour of troublesome dyskinesia at baseline (N equals 14, R1 and R2 combined). Oral LD Dosing and Frequency (post hoc analysis): Average dosing frequency of oral levodopa decreased in all patients from 6.6 times at baseline to 2.3 times per day by day 28. The average dose of oral LD decreased from approximately1100mg at baseline to approximately 330mg. Safety and Tolerability: 33 subjects (87%) out of 38 completed the study with 5 who did not complete the study, two of which were due to adverse events: one due to an infection at the infusion site and the other due to worsening of symptoms. Infusion site reactions (nodules, bruising and erythema) were common yet generally well tolerated. These results corroborate the safety and tolerability data obtained in previous studies and did not raise new safety or tolerability concerns. Preliminary Results: Preliminary trial 006 results demonstrate that the R1 dosing regimen provides a significant reduction in OFF-time and a significant increase in ON-time with no or mild dyskinesia. A substantial percentage of subjects experienced complete resolution of OFF-time. The treatment was associated with some nodules, consistent with prior trials, but otherwise did not raise new safety or tolerability concerns. Benefits were also seen with the R2 regimen in spite of the study design whereby patients started levodopa therapy later in the morning. ND0612H devices were generally found to be reliable with only few minor, correctable malfunctions reported. No inconvenience related to the wearing of the device was reported for either day or night administration. Detailed trial results will be presented at a future medical meeting. ND0612 EU Clinical and Regulatory Development NeuroDerm recently received minutes from a meeting held in January 2017 with the EMA's Scientific Advice Working Party. Based on this meeting and on the preliminary results of trial 006, NeuroDerm has modified its EU clinical and regulatory development path. Upon completion of its ongoing trials, NeuroDerm plans to submit a marketing application based on the results of an amended iNDiGO phase III efficacy study and the ongoing BeyoND (trial 012) long-term safety trial, seeking to obtain a broader label for ND0612 than the label that could have been granted under a PK regulatory route in the EU. The previously planned PK trial (trial 009) will not be carried out. Anticipated timelines for submission of the EU marketing application remain unchanged. NeuroDerm's U.S. clinical and regulatory development timelines also remain unchanged. iNDiGO Trial NeuroDerm's iNDiGO phase III efficacy study (trial 007) will be restarted and amended to support a broad label claim in the EU for ND0612. The trial will be expanded from 150 to 240 patients by adding a third treatment arm of ND0612H to the current ND0612L and control arms. Furthermore, new endpoints that reflect the recent trial 006 results, including a responder analysis, will be incorporated into this trial. NeuroDerm believes that these should enable the company to seek approval for both the low- and high-dose versions of ND0612 in the EU. It is anticipated that iNDiGO will be completed in 2018, in parallel to the ongoing long term BeyoND safety trial (Trial 012). Conference Call Details NeuroDerm will host a conference call at 8:30 a.m. ET today. Individuals can access the webcast in the Events and Presentations section of the Company’s website, by clicking here, or by dialing 844-452-2810 (U.S.) or 574-990-9831 (outside of the U.S.). The passcode is 79280228. A webcast will be archived on the website. About ND0612 ND0612 is designed to significantly reduce motor complications in Parkinson's disease patients through continuous, subcutaneous delivery of LD/CD solution. Previously completed Phase II trials demonstrated that the low dose ND0612L maintained steady, therapeutic levodopa plasma concentrations that were associated with major changes in several clinical parameters including “OFF” time reductions when added to optimal oral standard of care. The high dose ND0612H, intended for severe Parkinson’s disease patients, was shown to reach even higher levodopa steady plasma levels, indicating that it may provide an effective therapy alternative to current treatments requiring surgery such as deep brain stimulation and LD/CD Intestinal Gel. About Parkinson's disease Parkinson's disease is a progressive neurodegenerative illness characterized by reduced dopamine in the brain, resulting in a debilitating decrease in the patient's motor and non-motor functions. Its symptoms, such as trembling in the extremities and face, slowness of movement and impaired balance and coordination, worsen over time and gravely impact the patient's quality of life. Levodopa is the most effective treatment for Parkinson’s disease. However, chronic oral levodopa treatment is associated with fluctuations in motor response as result of which, despite the benefits of the drug, patients can experience periods of impaired motor and non-motor functions, also referred to as “OFF” time. In addition, mainly as a result of excessive/intermittent oral doses of levodopa aimed at treating the “OFF” time, some patients experience involuntary movements, or dyskinesia. The “OFF” time and dyskinesia affect the majority of levodopa-treated Parkinson's disease patients and can interfere with day-to-day functions, causing patients to become severely disabled. Current evidence suggests that intermittent dosing with standard oral formulations of levodopa contributes to the development of these motor complications. By contrast, it has been shown that continuous administration of levodopa can effectively treat motor fluctuations in Parkinson's disease patients without increasing troublesome dyskinesia; however, a convenient route for continuous administration has not been introduced to date. About NeuroDerm NeuroDerm is a clinical-stage pharmaceutical company developing drug-device combinations for central nervous system (CNS) disorders that are designed to overcome major deficiencies of current treatments and achieve enhanced clinical efficacy through continuous, controlled administration. NeuroDerm has three product candidates in different stages of development which offer a solution for almost every Parkinson’s disease patient from the moderate to the very severe stage of the disease. NeuroDerm has developed a line of levodopa and carbidopa (LD/CD) product candidates administered through small belt pumps that deliver a continuous, controlled dose of LD/CD. The LD/CD product candidates are ND0612L and ND0612H, which are used for treatment of moderate and advanced Parkinson’s disease patients, respectively, and which are delivered subcutaneously. In addition, NeuroDerm is developing ND0701, a novel subcutaneously delivered apomorphine formulation for patients who suffer from moderate to severe Parkinson’s disease and who do not respond well to LD/CD. NeuroDerm is headquartered in the Weizmann Science Park in Rehovot, Israel. Forward-Looking Statements This press release contains forward-looking statements, within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended that involve risks and uncertainties. Such forward-looking statements may include projections regarding our future performance and may be identified by words like "anticipate," "assume," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "future," "will," "seek" and similar terms or phrases. The forward-looking statements contained in this press release are based on management's current expectations and projections about future events. There are important factors that could cause our actual results, levels of activity, performance or achievements to differ materially from the results, levels of activity, performance or achievements expressed or implied by the forward-looking statements. In particular, you should consider the risks provided under "Risk Factors" in our annual report on Form 20-F for the year ended December 31, 2015 filed with the Securities and Exchange Commission. Any forward-looking statement made by us in this press release speaks only as of the date hereof. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.
News Article | February 16, 2017
BRUSSELS, BELGIUM and WASHINGTON, DC--(Marketwired - Feb 16, 2017) - The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader regenerative medicine and advanced therapies sector, today announced it has published its position on Hospital Exemption (HE) in response to vastly different interpretations and implementations across the European Union of Article 3(7) of Directive 2001/83/EC within article 28 (2) of the Advanced Therapy Medicinal Products (ATMP) Regulation. ARM believes that HE is a useful pathway to enable patients to receive an ATMP under controlled conditions in cases where no authorised medicinal product is available for an indication with a high unmet medical need. However, varying interpretations and implementations of HE among European Member States may lead to the supply of a medicine under HE when there is an existing licensed alternative that has undergone rigorous quality testing and demonstrated proven safety and efficacy. This undermines the case for investment in the licensing of ATMP, and as such, may act as a deterrent to developers and could ultimately limit the supply of products for European patients. "Throughout our efforts to advocate for the consistent interpretation and implementation of HE across the EU -- a topic that has been widely debated for several years -- we have been focused on understanding and defining an approach that will generate optimal outcomes for patients," said Jacqueline Barry, Chief Clinical Officer at Cell & Gene Therapy Catapult and chair of ARM EU Regulatory Committee. "This includes fostering a regulatory pathway for the development and widespread availability of innovative, disruptive medicines across the European community." In its position paper, ARM has outlined a series of proposals aimed at creating consistent implementation of HE across European Member States, including: "ARM has taken an important leadership role in advocating for a consistent interpretation and implementation of HE among European Union Member States," said Eduardo Bravo, Managing Director and CEO of TiGenix, member of the ARM Executive Committee. "We believe that ARM's initiative for greater clarity on HE addresses the hurdle to patients' access to effective treatments that the current divergent implementation of HE across the EU poses. Important for companies in our sector, clearly, is market access, and the position proposes a way forward to ensure HE is not misused to circumvent the applicable legal instruments for the authorisation of safe and effective medicinal products in Europe." ARM's official position on HE is available here. "We are very pleased that a series of planned activities, published by the EMA on 2nd February 2017 to foster ATMP development and enable expanded patient access in the EU, includes support of more transparency in HE, as proposed in our position paper. We encourage the stakeholders involved in the elaboration of the EU plan seeking to optimize the current regulatory framework to consider the other recommendations proposed in ARM's position on HE," concludes Annie Hubert, ARM's director of European public policy. About The Alliance for Regenerative Medicine The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine and advanced therapies worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Today, ARM has more than 250 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.
News Article | February 15, 2017
Enterprise Management Associates (EMA), a leading IT and data management research and consulting firm, today announced it will host a webinar titled “Responsible Rationalization: Reducing the Cost of IT Operations” featuring Steve Brasen, managing research director of enterprise mobile and endpoint management at EMA, and Gary Henderson, director at ASG Technologies. While enterprise IT infrastructures are the key driver for the success of nearly every modern business, their costs for initial deployment and operational management can be astonishingly expensive. Costs related to the provisioning and maintenance of a complex IT ecosystem can grossly erode operational budgets and marginalize business successes and profitability. Faced with this reality, most organizations are pressured to reduce IT-related costs without diminishing service availability or performance. Brasen and Henderson will discuss how rationalization practices can substantially reduce the cost of maintaining IT infrastructures while accelerating effectiveness and reliability, as well as cover: The webinar is Thursday, January 26 at 2:00 p.m. Eastern. Those who wish to attend can register at: http://research.enterprisemanagement.com/responsible-rationalization-webinar-pr.html Founded in 1996, Enterprise Management Associates (EMA) is a leading industry analyst firm that specializes in providing deep insight across the full spectrum of IT and data management technologies. EMA analysts leverage a unique combination of practical experience, insight into industry best practices, and in-depth knowledge of current and planned vendor solutions to help its clients achieve their goals. Learn more about EMA research, analysis, and consulting services for enterprise line of business users, IT professionals and IT vendors at http://www.enterprisemanagement.com or blogs.enterprisemanagement.com.
News Article | February 15, 2017
DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of the "Hepatitis D - Pipeline Insights, 2017" drug pipelines to their offering. Hepatitis D - Pipeline Insights, 2017 provides in depth insights on the pipeline drugs and their development activities around the Hepatitis D. This report covers the product profiles in various stages of development including Discovery, Pre-clinical, IND, Phase I, Phase II, Phase III and Preregistration. Report covers the product clinical trials information and other development activities including technology, licensing, collaborations, acquisitions, fundings, patent and USFDA & EMA designations details. This report also provides detailed information on the discontinued and dormant drugs that have gone inactive over the years for Hepatitis D. This report also assesses the Hepatitis D therapeutics by Monotherapy, Combination products, Molecule type and Route of Administration. For more information about this drug pipelines report visit http://www.researchandmarkets.com/research/8t3rcn/hepatitis_d