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News Article | November 7, 2016
Site: globenewswire.com

SALT LAKE CITY, Nov. 07, 2016 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, today announced that it will submit its BRACAnalysis CDx® test for approval by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) in parallel with the PMDA review of AstraZeneca’s novel PARP inhibitor, olaparib. BRACAnalysis CDx is the leading genetic test to identify patients who are likely to benefit from treatment with olaparib, which is a novel PARP inhibitor that targets tumor DNA repair pathway deficiencies to preferentially kill cancer cells. "BRACAnalysis CDx launched precision medicine for ovarian cancer patients by identifying patients more likely to respond to PARP inhibitors," said Mark C. Capone, president and CEO, Myriad Genetics.  "As a global leader in personalized medicine, Myriad is committed to providing the highest quality molecular tests in the fight against the world's most challenging diseases, including breast and ovarian cancers." Today’s announcement builds on a long-term companion diagnostic collaboration with AstraZeneca that began in 2007.  In December 2014, the U.S. Food and Drug Administration (FDA) approved BRACAnalysis CDx to identify ovarian cancer patients who may be appropriate for treatment with Lynparza™ (olaparib).  It was the first time the FDA had approved a laboratory developed test (LDT) under the premarket approval application process. About BRACAnalysis CDx® BRACAnalysis CDx is an in vitro diagnostic device intended for the qualitative detection and classification of variants in the protein coding regions and intron/exon boundaries of the BRCA1 and BRCA2 genes using genomic DNA obtained from whole blood specimens.  It is approved for use as a companion diagnostic with AstraZeneca’s Lynparza (olaparib) for patients with advanced ovarian cancer in the United States and Europe. About Olaparib Olaparib is the first in a class of drugs that act as inhibitors of poly(ADP)-ribose polymerase (PARP), which exploit tumor DNA repair pathway deficiencies to preferentially kill cancer cells.  It is marketed in the United States and Europe under the brand name Lynparza for patients with advanced ovarian cancer. About Myriad Genetics Myriad Genetics Inc., is a leading personalized medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics.  Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs.  Myriad is focused on three strategic imperatives:  transitioning and expanding its hereditary cancer testing markets, diversifying its product portfolio through the introduction of new products and increasing the revenue contribution from international markets.  For more information on how Myriad is making a difference, please visit the Company's website: www.myriad.com. Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, EndoPredict, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice HRD, Vectra, Prolaris and GeneSight are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G Note to Editors: Lynparza is a trademark of AstraZeneca. Safe Harbor Statement This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to  submitting the Company’s BRACAnalysis CDx® test for approval by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) in parallel with the PMDA review of AstraZeneca’s novel PARP inhibitor, olaparib; expanding the Company’s companion diagnostic collaboration with AstraZeneca; and the Company’s strategic directives under the caption “About BRACAnalysis CDx” and “About Myriad Genetics.”  These “forward-looking statements” are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those described or implied in the forward-looking statements. These risks include, but are not limited to: the risk that sales and profit margins of our existing molecular diagnostic tests and pharmaceutical and clinical services may decline or will not continue to increase at historical rates; risks related to our ability to transition from our existing product portfolio to our new tests; risks related to changes in the governmental or private insurers’ reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services tests and any future tests are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities; risks related to public concern over our genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire, including but not limited to our acquisition of Assurex, Sividon and the Clinic; risks related to our projections about the potential market opportunity for our products; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decision in the lawsuit brought against us by the Association for Molecular Pathology et al; risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements;  the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading “Risk Factors” contained in Item 1A of our Annual report on Form 10-K for the fiscal year ended June 30, 2016, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K.


DURHAM, North Carolina, Nov. 29, 2016 /PRNewswire/ -- Micell Technologies, Inc. (Micell) announced enrollment of the first patient in DESSOLVE J: a prospective, randomized, balanced, controlled, double-blind, multi-center study comparing MiStent SES® Sirolimus Eluting Absorbable Polymer Coronary Stent System (MiStent) to XIENCE V Everolimus Eluting Coronary Stent System (Xience) in Japan. Shigeru Saito, M.D., Vice Director, Shonan Kamakura General Hospital and Director of Cardiology and Catheterization Laboratories, is the principal investigator. DESSOLVE J is an all-comers trial with a primary endpoint of target lesion failure (TLF) comparing 12-month clinical outcomes between MiStent and Xience. Candidates for trial participation suffer from symptomatic coronary artery disease, including those with chronic stable angina, silent ischemia, acute coronary syndromes and those who qualify for percutaneous coronary interventions. Enrollment is planned at approximately 10 clinical sites throughout Japan. "We are pleased to enroll the first patient in this important study," said Dr. Saito. "This study is a historic one for Japan. It will provide us with valuable insights into the potential benefits of MiStent's unique pharmacokinetic profile, with a rapidly absorbing polymer and extended elution of crystalline sirolimus." DESSOLVE J is intended to provide Japanese-specific data that is supportive of DESSOLVE III, an all-comers clinical study that includes 1,400 patients randomized to MiStent or Xience at 20 sites in Europe. DESSOLVE III completed enrollment in 2015. Following the completion of DESSOLVE J, Micell intends to file a pre-market approval application with Japan's Pharmaceuticals and Medical Devices Agency for approval by its Ministry of Health, Labour, and Welfare. Micell's patented supercritical fluid technology allows for a rigorously controlled coating of the drug and polymer, whereby the drug is applied to a bare-metal stent in a dry powder, crystalline form. This preserves its morphology and optimizes its pharmacokinetic (distribution and absorption) profile. MiStent also leverages the benefits of a cobalt chromium coronary stent system -- a state-of-the-art, ultra-thin-strut metallic stent that has demonstrated excellent deliverability, conformability and flexibility. Arthur J. Benvenuto, Chairman and Chief Executive Officer of Micell said, "Cardiovascular disease poses a significant health risk in Japan and even with recent improvements in coronary artery disease treatment, additional advances in drug-eluting stents are still needed. Studies of MiStent to date have demonstrated a desirable lack of late lumen loss over 18 months, a characteristic that makes MiStent a clinically meaningful improvement that could provide clinicians and patients worldwide with enhanced treatment options." MiStent is designed to optimize healing and clinical performance in patients with coronary artery disease. The rapidly absorbable coating of MiStent, which contains crystalline drug (sirolimus) and an absorbable polymer, is intended to precisely and consistently provide for extended local drug delivery and limit the duration of polymer exposure. These characteristics potentially reduce the safety risks associated with currently commercially available drug-eluting stents and improve long-term clinical outcomes. EU approval of MiStent was supported by clinical data from two studies, DESSOLVE I and II, which demonstrated superior in-stent late lumen loss rates and an excellent safety profile. Micell also completed enrollment in December 2015 of DESSOLVE III, a 1,400 patient, 20 center, randomized clinical trial comparing MiStent to Xience Everolimus Eluting Coronary Stent System® (Xience). DESSOLVE III is a prospective, balanced, randomized, controlled, single-blind, multi-center study comparing clinical outcomes between MiStent and Xience in a "real world, all-comers" patient population. Patients in the trial suffered from symptomatic coronary artery disease, including those with chronic stable angina, silent ischemia, or acute coronary syndrome, and qualified for percutaneous coronary interventions. The primary endpoint for this trial is a non-inferiority comparison of target lesion failure (TLF) of the MiStent group versus the Xience group at 12 months post-procedure. The 12-month primary endpoint results for DESSOLVE III are expected to be released in the first half of 2017. MiStent has received CE marking, but is not approved for sale in the United States. This press release contains forward-looking statements that can be identified by the fact that they do not relate strictly to historical or current facts. Forward-looking statements include words such as "anticipates," "estimates," "expects," "projects," "intends," "plans," "believes" and words and terms of similar substance in connection with the results of a post-marketing clinical program and the commercialization and sale of MiStent in Europe and other markets. We caution readers that the forward-looking statements contained in this press release are predictions based on our current analysis of, and expectations about, future events and speak only as of the date of this press release. These forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties, including, but not limited to, the following: the results of any further clinical trials and studies; our ability to obtain regulatory approval of the MiStent in other jurisdictions; the successful development and commercialization of MiStent in Europe and other markets; the ability of MiStent to effectively and successfully compete with current commercially available drug-eluting stent technologies in Europe and other markets; and our ability to maintain and protect our proprietary stent coating technology. Actual results, performance or achievements could differ materially and adversely from those expressed or implied by any forward-looking statement contained in this press release. Micell, Micell Technologies, the Micell Logo, MiStent and MiStent SES are among the trademarks of Micell Technologies, Inc.


Similar PK/PD data between US and Japanese children treated with somavaratan support using the same dose in the ongoing US and Japan Phase 3 trials MENLO PARK, Calif., March 02, 2017 (GLOBE NEWSWIRE) -- Versartis, Inc. (NASDAQ:VSAR), an endocrine-focused biopharmaceutical company that is developing somavaratan (VRS-317), a novel, long-acting form of recombinant human growth hormone (rhGH) for growth hormone deficiency (GHD), announced that a comparison of data from its U.S. and Japanese Phase 2 studies was accepted as a late-breaking poster presentation at the Endocrine Society's 99th Annual Meeting & Expo (ENDO 2017), to be held April 1-4, in Orlando, Florida. The abstract has been published online and is available on the ENDO 2017 website. This is the sixth Versartis abstract that has been accepted for presentation at the conference, including five that were announced previously. Comparison of the pharmacokinetic and pharmacodynamic (PK/PD) properties between U.S. and Japanese pediatric subjects showed similar responses achieved at the same range of somavaratan doses in both populations. In addition, variability between the two patient sets was minor, with no effect on treatment outcomes. These similarities support the utilization of the same 3.5 mg/kg twice-monthly US Phase 3 dose in the ongoing Japanese Phase 3 trial, which has been reviewed with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA). Below are details on each of the presentations. ORAL PRESENTATION (Oral Session: OR31-1) Monday, April 3, 2017 - 11:15 AM - 12:45 PM 31135 - Safety and Efficacy of Somavaratan (VRS-317), a Long-Acting Recombinant Human Growth Hormone (rhGH), in Children with Growth Hormone Deficiency (GHD): 3-Year Update of the VERTICAL & VISTA Trials (NCT01718041, NCT02068521) POSTER PRESENTATION (Poster Board: SAT 016) Saturday, April 1, 2017 - 1:00 – 3:00 PM 31202 - Achievement of a Suitable Basis of Comparison in Phase 2 and Phase 3 Pediatric Somavaratan Clinical Trials (VERTICAL, VISTA, and VELOCITY Studies) and for the Comparison of Somavaratan to Daily Recombinant Human Growth Hormone (rhGH) POSTER PRESENTATION (Poster Board: SAT 015) Saturday, April 1, 2017 - 1:00 – 3:00 PM 29268 - IGF Family Biomarkers in the Diagnosis of Pediatric Growth Hormone Deficiency (PGHD) in Somavaratan Clinical Trials About Somavaratan Somavaratan is Versartis' investigational, novel, long-acting form of recombinant human growth hormone (rhGH). This fusion protein consists of rhGH and specific sequences of hydrophilic amino acids based on a proprietary XTEN®1 technology. Somavaratan has been designed with the goal of improving therapeutic outcomes for children and adults with growth hormone deficiency (GHD), including enhanced adherence and convenience with a twice-monthly dosing schedule, fine gauge needle autoinjector device and room temperature storage. Somavaratan is currently being evaluated for the treatment of pediatric GHD in the pivotal Phase 3 VELOCITY trial in the U.S., Canada and Europe, for which data are anticipated in September 2017, and the J14VR5 Phase 2/3 trial in Japan. Confirmatory safety and efficacy data from 36 months of dosing in the Phase 2 trial and VISTA long-term safety study are scheduled to be presented during the Endocrine Society 2017 annual meeting. In adult GHD, results have been reported from the Phase 2 VITAL trial in the U.S., Europe and Australia and a Phase 3 trial is expected to begin by the end of 2017. 1XTEN is a registered trademark of Amunix Operating Inc. About Versartis, Inc. Versartis, Inc. is an endocrine-focused biopharmaceutical company initially developing somavaratan, a novel, long-acting form of recombinant human growth hormone in late-stage clinical trials for the treatment of GHD in children and adults.  Somavaratan is intended to reduce the burden of daily injection therapy by requiring significantly fewer injections, potentially improving adherence and, therefore, treatment outcomes.  For more information on Versartis, visit www.versartis.com. Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements regarding our intentions or current expectations concerning, among other things, plans and timing of our clinical trials and the potential for eventual regulatory approval of somavaratan. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: our success being heavily dependent on somavaratan; somavaratan being a new molecular entity; the risk that somavaratan may not have favorable results in clinical trials or receive regulatory approval; potential delays in our clinical trials due to regulatory requirements or difficulty identifying qualified investigators or enrolling patients; the risk that somavaratan may cause serious side effects or have properties that delay or prevent regulatory approval or limit its commercial potential; the risk that we may encounter difficulties in manufacturing somavaratan; if somavaratan is approved, risks associated with its market acceptance, including pricing and reimbursement; potential difficulties enforcing our intellectual property rights; our reliance on our license of intellectual property from Amunix Operating, Inc. and our need for additional funds to support our operations. We discuss many of these risks in greater detail under the heading "Risk Factors" contained in our Annual Report on Form 10-K for the year ended December 31, 2015 and in our Quarterly Report on Form 10-Q for the three months ended September 30, 2016, which are on file with the Securities and Exchange Commission (SEC). Forward-looking statements are not guarantees of future performance, and our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.


News Article | February 15, 2017
Site: globenewswire.com

New Phase 3 Retrospective Analysis Shows Correlation between Reduction in Disease Substrate (Kidney Interstitial Capillary GL-3) and Improved Diarrhea in Fabry Patients with Amenable Mutations treated with Migalastat Supportive Study for Japanese New Drug Application (J-NDA) Demonstrates Migalastat Exposure is Similar in Japanese and non-Japanese Individuals SAN DIEGO and CRANBURY, N.J., Feb. 14, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biotechnology company at the forefront of therapies for rare and orphan diseases, today announced new positive data analyses for the oral small molecule pharmacological chaperone migalastat HCl (“migalastat”) for Fabry disease at WORLDSymposium™ 2017 in San Diego, California. Jay Barth, Chief Medical Officer of Amicus Therapeutics, Inc., stated, “The new analyses highlighted at this year’s WORLDSymposium add to the already significant body of data which demonstrate the multiple benefits of treatment with migalastat in Fabry patients with amenable mutations. Here, the correlation of the reduction in disease substrate with the reduction in diarrhea symptoms provides further evidence that migalastat is having a positive effect on an important gastrointestinal symptom in Fabry. These findings in diarrhea symptoms support our current Fabry regulatory strategy for migalastat in the U.S., which is based upon improvement in diarrhea in this patient population. Also, we remain on track in Japan with our submission to the Pharmaceuticals and Medical Devices Agency based on completed Phase 1 and Phase 3 studies. We also remain committed to providing access to this important medicine to patients in the EU, where it is already approved, in addition to other major geographies.” Data Highlights for Migalastat for Fabry Disease at WORLDSymposium 2017 Phase 3 Retrospective Analysis - Correlation between Substrate Reduction and Reduction in Diarrhea In a poster1 from Study 011 (FACETS) in Fabry patients who were naïve to ERT, a retrospective analysis from baseline to month 6 demonstrated that migalastat reduces disease substrate (KIC GL-3) and improves diarrhea symptoms (GSRS-D) in patients with Fabry disease with amenable mutations. Key highlights were as follows: To support full approval in the U.S., which represents approximately 25% of the global Fabry market, Amicus plans to confirm the clinical beneficial effects of migalastat in a GI symptom study. The GI study is anticipated to begin in 2017 in approximately 35 Fabry patients who are naïve to treatment and who have an amenable mutation and diarrhea and other GI symptoms. More than 50% of patients with Fabry disease report or show GI signs and symptoms, including diarrhea, abdominal pain, constipation, nausea, and vomiting.2 Supportive Pharmacokinetics (PK) Study for Japanese Regulatory Submission As previously announced, Amicus plans to submit a J-NDA in Japan in the first half of 2017. The J-NDA will be based on data from completed clinical studies with migalastat, including two pivotal Phase 3 studies as well as a Phase 1 study that evaluated the pharmacokinetics (PK) of migalastat in Japanese volunteers. The results from this Phase 1 study are being highlighted in a poster3 at WORLDSymposium 2017 with key highlights as follows: Japan represents the second largest Fabry market in the world by country, with approximately 13% of the $1.2B global Fabry ERT sales generated in Japan in 2015.4 The Pharmaceuticals and Medical Devices Agency (PMDA) in Japan previously confirmed that completed studies of migalastat meet J-NDA submission requirements without the need to conduct an additional clinical study in Japan. The PMDA took into account data from Japanese patients included in the Phase 3 program and the similar PK properties in Japanese and non-Japanese individuals. Migalastat is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations. On May 30, 2016, the European Commission granted full approval for migalastat, under the trade name Galafold™, as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation. This EU approval may serve as the basis for regulatory approvals in more than two-thirds of the global Fabry market that is outside the U.S. Amicus has commenced the commercial launch of Galafold in Germany and is undergoing the EU country-by-country processes to launch in the majority of EU countries throughout 2016 and 2017. The Company has also initiated expanded access programs (EAP) in the EU and other territories outside the U.S. that provide this mechanism for reimbursed access prior to formal approval. Galafold™ (migalastat) is a first-in-class chaperone therapy approved in the EU as a monotherapy for Fabry disease in patients with amenable mutations. Galafold works by stabilizing the body’s own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations.  A proprietary in vitro assay (Galafold Amenability Assay) was used to classify more than 800 known GLA mutations as “amenable” or “not amenable” to treatment with Galafold. The current EU label includes 313 GLA mutations that have been identified and determined to be amenable based on the Galafold Amenability Assay, which represent between 35% and 50% of the currently diagnosed Fabry population. Healthcare providers in the EU may access the website www.galafoldamenabilitytable.com to quickly and accurately identify which mutations are categorized as “amenable” or “not amenable” to Galafold. Amicus expects to submit updates to the label as additional GLA mutations are identified and tested in the Galafold Amenability Assay. Treatment with GALAFOLD should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. GALAFOLD is not recommended for use in patients with a nonamenable mutation. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu. Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb ). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease, and stroke. The symptoms can be severe, differ from patient to patient, and begin at an early age. All Fabry disease is progressive and may lead to organ damage regardless of the time of symptom onset. Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases. 1D. Germain, WORLDSymposium 2017, Effects of Treatment With Migalastat on the Combined Endpoint of Kidney Globotriaosylceramide Accumulation and Diarrhea in Patients With Fabry Disease: Results From the Phase 3 FACETS Study 2Hoffmann B et al. Clin Gastroenterol Hepatol. 2007;5(12):1447-1453. 3F. Johnson, WORLDSymposium 2017, Migalastat exposures in Japanese healthy volunteers and non-Japanese subjects provide evidence that they are similar to Japanese patients with Fabry disease 4Company filings and Amicus estimates Forward Looking Statements This press release contains "forward- looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to data, future studies and ongoing regulatory strategies for migalastat.  Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” "confidence," "encouraged," “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “could,” and similar expressions or words identify forward-looking statements.  The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the data reported herein will not be predictive of future results, that later study results will not support  further development of migalastat, or even if such later  results are favorable, that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully commercialize migalastat.  In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2015 and Quarterly Report on 10-Q for the Quarter ended September 30, 2016.  As a consequence, actual results may differ materially from those set forth in this press release or the accompanying conference call or webcast.  You are cautioned not to place undue reliance on these forward looking statements, which speak only of the date hereof.  All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof.


CHAPEL HILL, N.C., Dec. 05, 2016 (GLOBE NEWSWIRE) -- Cempra, Inc. (Nasdaq:CEMP), a clinical-stage pharmaceutical company focused on developing antibiotics to meet critical medical needs in the treatment of bacterial infectious diseases, today announced that Toyama Chemical Co., Ltd. (Toyama) a subsidiary of FUJIFILM Holdings Corporation, has begun Phase 3 clinical trials with solithromycin in Japan, the world's second largest antibiotic market, for patients with community-acquired bacterial pneumonia (CABP) and other respiratory infections. Earlier this year, Toyama completed a Phase 2 multi-center, randomized, double-blinded study of 135 Japanese patients with mild to moderate CABP. Patients were randomized to either oral solithromycin or oral levofloxacin for five days. Overall safety and tolerability was similar in both treatment groups and all efficacy outcome measures favored solithromycin. These data, and the data from Cempra’s studies, were reviewed by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) before finalizing the Phase 3 study protocol. Toyama owns exclusive rights to develop and commercialize solithromycin in Japan for respiratory tract infections and other indications in adults and pediatric patients. In November 2016, Cempra announced it had received a $10 million milestone payment when Toyama decided to progress to Phase 3 studies. Cempra has received $40 million of upfront and milestone payments from Toyama and can earn an additional $30 million, for a total of $70 million, in payments from Toyama based on the achievement of certain objectives. If approved, Toyama would pay tiered royalties, adjusted based on sales, to Cempra following launch of solithromycin in Japan. “We are excited and encouraged to see Toyama commencing their Phase 3 program and moving another step closer to the potential approval of solithromycin in Japan, where already high antibiotic resistance in pneumococcus strains and other CABP pathogens is rising, highlighting the urgent unmet medical need for new therapies,” said Prabhavathi Fernandes, Ph.D., chief executive officer of Cempra. “We are also pleased that the Toyama Phase 3 trial will be against levofloxacin as the comparator, which is the fluoroquinolone used most frequently in outpatient CABP treatment,” Fernandes added. Cempra, Inc. is a clinical-stage pharmaceutical company focused on developing antibiotics to meet critical medical needs in the treatment of bacterial infectious diseases. Cempra's two lead product candidates are currently in advanced clinical development. Solithromycin has been successfully evaluated in two Phase 3 clinical trials for community acquired bacterial pneumonia (CABP) and applications for approval for both intravenous and oral capsule formulations have been accepted for review by the FDA and the EMA. Solithromycin is licensed to strategic commercial partner Toyama Chemical Co., Ltd., a subsidiary of FUJIFILM Holdings Corporation, for certain exclusive rights in Japan. Solithromycin is also in a Phase 3 clinical trial for uncomplicated urogenital urethritis caused by Neisseria gonorrhoeae or chlamydia. Cempra is contracted with BARDA for the development of solithromycin for pediatric use and has commenced enrollment in a global Phase 2/3 trial to evaluate the safety and efficacy of solithromycin versus standard of care antibiotics in children and adolescents from two months to 17 years of age. Fusidic acid is Cempra's second product candidate, which has completed enrollment of an initial Phase 3 trial comparing fusidic acid to linezolid in patients with acute bacterial skin and skin structure infections (ABSSSI).  Cempra also has an ongoing exploratory study of fusidic acid for chronic oral treatment of refractory infections in bones and joints. Both products seek to address the need for new treatments targeting drug-resistant bacterial infections in the hospital and in the community. Cempra has also synthesized novel macrolides for non-antibiotic uses such as the treatment of chronic inflammatory diseases, endocrine diseases and gastric motility disorders. Cempra was founded in 2006 and is headquartered in Chapel Hill, N.C. For additional information about Cempra please visit www.cempra.com. Please Note: This press release contains forward-looking statements regarding future events. These statements are just predictions and are subject to risks and uncertainties that could cause the actual events or results to differ materially. These risks and uncertainties include, among others: the costs, sources of funds, enrollment, timing, regulatory review and results of our studies and clinical trials and those of our strategic commercial partners; results of our and our strategic commercial partners’ pre-clinical studies and clinical trials are not predictive of results from subsequent clinical trials for any possible therapy; our and our strategic commercial partners' ability to obtain FDA and foreign regulatory approval of our product candidates, including solithromycin; our dependence on the success of solithromycin and fusidic acid; the unpredictability of the size of the markets for, and market acceptance of, any of our products, including solithromycin and fusidic acid; our ability to commercialize and launch, whether on our own or with a strategic partner, any product candidate that receives regulatory approval; our ability to produce and sell any approved products and the price we are able to realize for those products; our ability to retain and hire necessary employees and to staff our operations appropriately; our need to obtain additional funding and our ability to obtain future funding on acceptable terms; our anticipated capital expenditures and our estimates regarding our capital requirements; the possible impairment of, or inability to obtain, intellectual property rights and the costs of obtaining such rights from third parties; our ability to compete in our industry; innovation by our competitors; and our ability to stay abreast of and comply with new or modified laws and regulations that currently apply or become applicable to our business. The reader is referred to the documents that we file from time to time with the Securities and Exchange Commission.


News Article | December 9, 2015
Site: www.nature.com

Japan has been working feverishly to stay at the cutting edge of research and clinical applications in regenerative medicine. It has invested billions of yen in induced pluripotent stem (iPS) cells — made by reprogramming an individual’s adult cells so that they can develop into any body tissue — and has overhauled its drug regulations to create a fast track to bring regenerative therapies to market. The strategy is working, up to a point — in September, the first treatments were approved under the new law. According to bullish regenerative-medicine firms in Japan, the scheme is the fastest way to meet patients’ needs. Without it, they argue, treatments get bogged down in phased clinical trials that can take several years and cost hundreds of millions of dollars. But it is not clear whether the acceleration will benefit patients or help Japan’s overburdened national health system. One of the approved treatments, HeartSheet, is made of skeletal-muscle stem cells that are taken from a patient’s thigh and grown in the lab. The sheet, made by the company Terumo, is then applied to the hearts of people who have severe cardiac failure. Japan’s health ministry gave “conditional approval” for clinical use of the treatment after the company carried out a phase II trial, which hinted at its safety and efficacy in seven patients (Y. Sawa et al. Circ. J. 79, 991–999; 2015). The company can market and sell the treatment. The approval comes with the condition that, within 5 years, Terumo must provide data from at least 60 patients treated with HeartSheet and 120 controls to show that the treatment is effective. Officials at the Pharmaceuticals and Medical Devices Agency, which approves new treatments, say that the examination of these data will be just as strict as it would be for a conventional phase III clinical trial. Such approvals feed two Japanese obsessions. First, they allow Japan to be at the forefront of regenerative medicine, something that it has pursued doggedly since iPS cells — which would go on to win one of the country’s scientists a Nobel prize — became a national project. Second, Japan is determined to find new engines of economic growth, because it has enjoyed few successes in biotechnology so far. Biotech firms around the world are excited about the approval, too. Stories of commercialization are a welcome counterpoint to the narrative of failure. California biotech firm Geron, once a trailblazer in regenerative medicine, has given up on embryonic stem-cell therapies and, just this year, Masayo Takahashi of the RIKEN Center for Developmental Biology in Kobe decided to halt her trial of iPS-cell-derived retinal grafts to treat age-related macular degeneration. Patients are willing to pay, and pay dearly: the HeartSheet treatment costs nearly ¥15 million (US$122,000). Last month, the health ministry added it to the procedures covered by national health insurance, which will help. But patients still pay 10–30% of the cost for a drug that is not known to be effective. As they do so, they basically subsidize the company’s clinical trial. Japan has turned the drug-discovery model on its head. Usually, the investment — and thus the risk — is borne by drug companies, because they stand to gain in the long run. Now the risk is being outsourced. By the time it is clear whether a treatment works or not, the companies will have already made revenue from it. The government argues that its system will encourage firms to bring to market regenerative-medicine treatments that might work. They will, at least, work well enough to make it past small initial trials. Many drugs do that, and then most of them fail at phase III. Biotech companies in other countries are keen on the idea and have pushed their own regulatory bodies to follow Japan’s lead. This is a bad move. Regulatory agencies around the world should resist pressure to create such fast-track systems, at least until Japan has proved that its system works. That will take time. The country will have to demonstrate that its health-care system can withstand the costs of the new regenerative-medicine treatments, and that patients do not feel cheated. What happens when, inevitably, one of the fast-track drugs turns out to be ineffective? Company officials and government representatives say that patients will not be reimbursed, even though some might have paid up to ¥4.5 million (the rest covered by health insurance) for an ineffective treatment. Japan’s drug authority must guarantee that the post-commercialization evaluation of the drugs will be as rigorous as it says. It will not be easy to rein in a drug that has already been approved, whether that approval is conditional or not. If lax evaluation means that ineffective drugs are not revealed, or are not taken out of circulation, Japan could find itself flooded with unsuccessful treatments. And that would not be good for patients, the government or the biotech companies that want to see their truly effective medicines noted as such.


Moritoyo T.,University of Tokyo | Moritoyo T.,Pharmaceuticals and Medical Devices Agency
Clinical Therapeutics | Year: 2015

Purpose The Ministry of Health, Labour and Welfare (MHLW) of Japan launched a regulatory science research project in which the aim is to promote the establishment of guidelines for the development of innovative drugs thorough interactions between academia and Japan's regulatory agency, the Pharmaceuticals and Medical Devices Agency (PMDA). In this project, a research system with the aim of developing a guideline for the clinical evaluation of drugs intended for the treatment of Alzheimer's disease (AD) was established. Methods Two research groups were set up: (1) the Biomarker and Clinical Evaluation Group to establish biomarker-based criteria for the clinical evaluation of drugs for AD, and (2) the Modeling and Simulation (M&S) Group to create a disease model of AD using M&S techniques based on data from the Alzheimer's Disease Neuroimaging Initiative (ADNI). Furthermore, a human resource exchange between the University of Tokyo Hospital and the PMDA is conducted to establish a guideline that is suitable for regulatory use. Findings As an interim report of this project, issues that require consideration for the clinical evaluation and development were summarized, including topics such as the use of biomarkers in the inclusion criteria, the efficacy endpoint, and the clinical data package required for application in Japan. Implications As the result of collaboration between the University of Tokyo Hospital and PMDA, this document is the first to summarize perspectives on the development of drugs for AD in Japan. © 2015 Elsevier HS Journals, Inc.© 2015 ElsevierHSJournals, Inc. All rights reserved.


Hirakawa A.,Pharmaceuticals and Medical Devices Agency | Hirakawa A.,Tokyo University of Science
Statistics in Medicine | Year: 2012

In this study, we developed a novel adaptive dose-finding approach for inclusion of correlated bivariate binary and continuous outcomes in designing phase I oncology trials. For this approach, binary toxicity and continuous efficacy outcomes are modeled jointly with a factorization model. The basic strategy of the proposed approach is based primarily on the Bayesian method. We based the dose escalation/de-escalation decision rules on the posterior distributions of both toxicity and efficacy outcomes. We compared the operating characteristics of the proposed and existing methods through simulation studies under various scenarios. We found that the recommendation rate of the true recommended dose (RD) in the proposed method was more favorable than that in the existing method when the true RD was relatively at the tail end among the tested doses. It was similar to that of the existing method when the true RD was relatively at the top end. © 2011 John Wiley & Sons, Ltd.


Li H.-B.,Rutgers University | Li H.-B.,Yale University | Ohno K.,Rutgers University | Ohno K.,Pharmaceuticals and Medical Devices Agency | And 2 more authors.
PLoS Genetics | Year: 2013

Polycomb bodies are foci of Polycomb proteins in which different Polycomb target genes are thought to co-localize in the nucleus, looping out from their chromosomal context. We have shown previously that insulators, not Polycomb response elements (PREs), mediate associations among Polycomb Group (PcG) targets to form Polycomb bodies. Here we use live imaging and 3C interactions to show that transgenes containing PREs and endogenous PcG-regulated genes are targeted by insulator proteins to different nuclear structures depending on their state of activity. When two genes are repressed, they co-localize in Polycomb bodies. When both are active, they are targeted to transcription factories in a fashion dependent on Trithorax and enhancer specificity as well as the insulator protein CTCF. In the absence of CTCF, assembly of Polycomb bodies is essentially reduced to those representing genomic clusters of Polycomb target genes. The critical role of Trithorax suggests that stable association with a specialized transcription factory underlies the cellular memory of the active state. © 2013 Li et al.


HAIFA Israel, TOKYO, Dec. 20, 2016 (GLOBE NEWSWIRE) -- Corporate Venture Capital Ltd., (Subsidiary of Sosei Group Corporation, a Tokyo Stock Exchange Mothers listed company), and Pluristem Therapeutics Inc. (Nasdaq:PSTI), (TASE: PSTI), a leading developer of placenta-based cell therapy products, today announced the signing of a binding term sheet for the establishment of a new Japanese corporation (NewCo) for the clinical development and commercialization of Pluristem’s PLX-PAD cell therapy product in Japan.  Following completion of fund-raising, the parties plan to establish NewCo in Japan. Pluristem will own 35% of NewCo in return for its contribution of a perpetual license to commercialize PLX-PAD for Critical Limb Ischemia (CLI) in Japan. All proprietary rights related to PLX-PAD will be exclusively owned by Pluristem. Sosei CVC’s investment fund, Sosei RMF1, together with additional Japanese investors, will raise and invest approximately $11 million, equivalent to approximately ‎¥‎1.3 billion, in return for ownership of 65% of NewCo. The first indication to be developed by NewCo will be CLI. The design of a 75-patient study of PLX-PAD in CLI was previously agreed upon with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) under an accelerated regulatory pathway for regenerative medicine. This single study may lead to early conditional marketing approval and early reimbursement. Future marketing activities are planned to be undertaken by NewCo. The parties plan to enter into a definitive agreement no later than March 31, 2017. “We are pleased to partner with Sosei CVC to commercialize PLX-PAD in Japan. The development of our CLI program through the accelerated regulatory pathway could allow a more rapid entrance into the sizeable Japanese market, as has been our strategy,” stated Zami Aberman, Pluristem’s Chairman and CEO. “Our cooperation with Sosei CVC also creates the potential to develop additional indications in this market, by drawing on our robust portfolio of cell therapy product candidates in development,” Mr. Aberman added. “We are eager to begin the joint development of PLX-PAD in critical limb ischemia with the goal of commercialization, and believe that our cooperation could lead to an efficient and successful entry into Japan’s substantial market,” said Toshimi Miyoshi, Director, Sosei CVC. “The NewCo is expected to be a meaningful investment by Sosei RMF1 into the regenerative medicine space, and will support our goal to stimulate the biotechnology industry in Japan,” Miyoshi concluded. About Japan’s Conditional Time-limited Approval for Regenerative Medicine Products Japan’s Act on the Safety of Regenerative Medicine went into effect in November 2014. Its purpose is to facilitate faster commercialization of cellular therapies and other regenerative medicine treatments. Per the Act, these therapies can receive conditional, time-limited approval for marketing, and be eligible for reimbursement, upon proof of safety and a signal of effectiveness but prior to verification of efficacy. Safety and efficacy need to be confirmed via collection of observational data from treated patients after the conditional approval. Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells and is entering late-stage trials in several indications. The cell products release a range of therapeutic proteins in response to inflammation, ischemia, hematological disorders, and radiation damage. PLX cell products are grown using the Company's proprietary three-dimensional expansion technology. They are off-the-shelf, requiring no tissue matching prior to administration. Pluristem has a strong intellectual property position; Company-owned and operated, GMP-certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team. Sosei Corporate Venture Capital (Sosei CVC) is the corporate venture arm of Sosei Group Corporation, a Tokyo Stock Exchange Mothers listed company.  Along with its partners, Sosei CVC currently manages Sosei RMF1, a ¥2 billion fund focusing on investing in companies with innovative regenerative medicine technology. This press release contains express or implied forward-looking statements within the Private Securities Litigation Reform Act of 1995 and other U.S. Federal securities laws. For example, we are using forward-looking statements when we discuss the establishment of a Japanese new corporation, our expected holdings in the new corporation and the investment of approximately $11 million in the new corporation by Sosei RMF1 and additional partners; when we discuss the development and marketing plans of the new corporation; when we discuss PLX-PAD’s potential eligibility for early conditional marketing approval, and for reimbursement, following a single, PMDA-approved clinical trial; when we discuss the parties’ plan to enter into a definitive agreement and the proposed date of execution of such agreement; when we discuss the potential of the accelerated regulatory pathway to allow us a more rapid entrance into the sizeable Japanese market; and when we discuss the potential of our cooperation with Sosei CVC to develop additional indications in the Japanese market. Further, although Pluristem has signed a binding term sheet, it may not be successful in negotiating definitive documentation by the date expected or at all, and even if successful, the transaction may not be completed if the conditions to closing are not met. These forward-looking statements and their implications are based on the current expectations of the management of Pluristem only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications; loss of market share and pressure on pricing resulting from competition, which could cause the actual results or performance of Pluristem to differ materially from those contemplated in such forward-looking statements. Except as otherwise required by law, Pluristem undertakes no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. For a more detailed description of the risks and uncertainties affecting Pluristem, reference is made to Pluristem's reports filed from time to time with the Securities and Exchange Commission.

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