Agios Announces FDA Fast Track Designation Granted to AG-120; Regeneron and Sanofi Announce Positive Pivotal Phase 2b Dupilumab Data Print
By William Kent   
Monday, 18 May 2015 20:53
Below is a look at some of the headlines for companies that made news in the healthcare sector on May 18 2015.
Agios Pharmaceuticals, Inc.  (NASDAQ: AGIO), a leader in the fields of cancer metabolism and rare genetic disorders of metabolism, announced the U.S. FDA has granted Fast Track designation to AG-120 for the treatment of patients with acute myelogenous leukemia (AML) who harbor an isocitrate dehydrogenase-1 (IDH1) mutation. AG-120 is a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein being evaluated in two Phase 1 clinical trials, one in hematologic malignancies that recently initiated three expansion cohorts, and one in advanced solid tumors, including glioma.
"We are pleased that now both AG-120 and AG-221 have been granted Fast Track designation, demonstrating the FDA's commitment to facilitate the development and expedite the review of our lead IDH programs as important new therapies for people with AML who carry these mutations," said Chris Bowden, M.D., chief medical officer of Agios. "We look forward to presenting new data from the ongoing Phase 1 study at the EHA Annual Congress next month and remain on track to initiate a global, registration-enabling Phase 3 study in collaboration with Celgene in AML patients who harbor an IDH1 mutation in the first half of 2016."
The FDA's Fast Track Drug Development Program is designed to expedite clinical development and submission of New Drug Applications (NDA) for medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs. Specifically, Fast Track designation facilitates frequent interactions with the FDA review team, including meetings to discuss all aspects of development to support approval, and also provides the opportunity to submit sections of an NDA on a rolling basis as data become available.
 
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Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi shared positive results from an interim analysis of a pivotal Phase 2b study of dupilumab in adult patients with moderate-to-severe asthma, who are uncontrolled despite treatment with inhaled corticosteroids and long-acting beta agonists (ICS/LABA). As previously reported, the study met its primary endpoint of improving lung function in asthma patients with high blood eosinophil counts (HEos, greater than or equal to 300 eosinophilic cells/microliter). Such high counts are thought to be a marker for patients more likely to have "atopic" or "allergic" asthma. New data presented on secondary endpoints at the American Thoracic Society 2015 International Conference included positive results in study patients with low blood eosinophil counts (LEos, less than 300 eosinophilic cells/microliter), who are thought to be less likely to suffer from "allergic" asthma and
thus less likely to respond to TH2 targeted therapies. Dupilumab is an investigational therapy that inhibits signaling of IL-4 and IL-13, two cytokines required for the TH2 (or Type 2) immune response.
Based on discussions with the U.S. Food and

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Drug Administration (FDA), this Phase 2b study may be considered one of two pivotal efficacy studies required for a potential dupilumab biologics license application (BLA) in asthma. The companies also announced the initiation of a Phase 3 clinical trial of dupilumab in patients with uncontrolled persistent asthma, known as LIBERTY ASTHMA QUEST, which will serve as the second required pivotal efficacy study. The global, placebo-controlled Phase 3 study will enroll more than 1,600 patients with uncontrolled persistent asthma and will evaluate two doses of dupilumab, 200 milligrams (mg) and 300 mg, subcutaneously administered every other week (Q2W).
The new results focused on LEos asthma patients. In this population, patients treated with either 200 mg or 300 mg Q2W doses of dupilumab showed a greater than 8 percent improvement in forced expiratory volume over one second (FEV1, a standard measure of lung function) at Week 12 (p less than 0.001), in comparison to placebo, both in combination with ICS/LABA. Additionally, the 200 mg and
300 mg Q2W doses of dupilumab in combination with ICS/LABA showed 68 percent and 62 percent reductions, respectively, in adjusted annualized rate of severe exacerbations in the LEos population (p less than 0.01 and p less than 0.05), in comparison to placebo in combination with ICS/LABA.
These results are consistent with previously reported positive results in HEos asthma patients and the overall patient population, in which the two Q2W doses (200 mg and 300 mg) of dupilumab in combination with ICS/LABA demonstrated a statistically significant 12 to 15 percent improvement in FEV1 over placebo at Week 12 and a 64 to 75 percent improvement in annualized rate of severe exacerbations over placebo.
Dupilumab also significantly reduced mean fractional exhaled nitric oxide (FeNO) across both Q2W doses tested (200 and 300 mg) and the three patient populations (overall, LEos and HEos), in a roughly dose-dependent manner. FeNO is recommended by the American Thoracic Society clinical practice guidelines to assess airway inflammation, since higher than normal levels of nitric oxide may be released when a patient has a chronic airway disease, such as asthma.
The most common adverse event was injection site reaction, which was more frequent in the dupilumab dose groups (13 to 25 percent) compared to placebo (12 percent). Other common adverse events in the study included upper respiratory tract infection (10 to 13 percent dupilumab; 13 percent placebo), headache (5 to 10 percent dupilumab; 8 percent placebo), nasopharyngitis (3 to 10 percent dupilumab; 6 percent placebo) and bronchitis (5 to 8 percent dupilumab; 8 percent placebo). The incidence of infections was balanced across treatment groups (42 to 45 percent dupilumab; 46 percent placebo), as was the incidence of serious adverse events (3 to 7 percent dupilumab; 5 percent placebo).
"Despite available treatments, many patients with asthma continue to have symptoms and recurring attacks, which have a serious and detrimental impact on their daily lives," said Sally Wenzel, M.D., lead investigator from The University of Pittsburgh, Division of Pulmonary, Allergy and Critical Care Medicine. "In the study, dupilumab added to standard-of-care therapy demonstrated fewer exacerbations and improved lung function across both the high and low baseline eosinophil groups. We look forward to the continued clinical development of dupilumab as a potential option for a broad population of patients with uncontrolled asthma."
The slide set presented at ATS can be found on Regeneron's website here. Dupilumab is an investigational agent under clinical development and its safety and efficacy have not been fully evaluated by any regulatory agency.
These results were based on a pre-specified interim analysis, which occurred when all patients had reached Week 12 of the 24-week treatment period; the average treatment duration at the time of the analysis was 21.4 weeks. The primary endpoint of the study was improvement from baseline in FEV1 at Week 12 in the HEos group. Final analyses on exacerbations and safety will be conducted after 24 weeks of treatment and a 16-week follow-up period.
 
Also Monday:
Anavex Life Sciences Corp. (OTCQX:AVXL), a clinical-stage biopharmaceutical company developing drug candidates to treat Alzheimer's disease, other central nervous system (CNS) diseases, pain and various types of cancer, revealed full preclinical anti-seizure data for its lead drug candidate, ANAVEX 2-73, in a presentation on Friday, May 15, 2015 at the Antiepileptic Drug Trials XIII Conference. ANAVEX 2-73 demonstrated convincing data in three well-established and according to experts highly predictive preclinical anti-seizure models with potentially more favorable side effect profile than currently marketed epilepsy drugs. 
 
Applied Genetic Technologies Corporation (Nasdaq:AGTC), a clinical stage biotechnology company developing adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, today announced new preclinical data which further supports the safety of the company's AAV vector gene therapies for the treatment of achromatopsia (ACHM) and X-linked retinoschisis (XLRS). 
Corium International, Inc. (Nasdaq:CORI), a commercial-stage biopharmaceutical company focused on the development, manufacture and commercialization of specialty transdermal products, today announced the publication of promising data on the use of its needle-free transdermal system to deliver influenza vaccine in the June 9, 2015 issue of Vaccine, a pre-eminent publication on vaccines and vaccination. 
ERBA Diagnostics, Inc. (NYSE MKT:ERB), a fully integrated in vitro diagnostics company, reported its financial results for the year ended December 31, 2014.
Fate Therapeutics, Inc. (Nasdaq:FATE), a biopharmaceutical company engaged in the development of programmed cellular therapeutics for the treatment of severe, life-threatening diseases, today announced that it has commenced an underwritten public offering of 6,000,000 shares of its common stock. 
Idera Pharmaceuticals, Inc. (Nasdaq:IDRA), a clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with cancer and rare diseases, today announced the achievement of key development milestones for its product candidate IMO-9200, an antagonist of Toll-like receptors (TLRs) 7, 8 and 9. 
iKang Healthcare Group, Inc. (Nasdaq:KANG), China's largest private preventive healthcare services provider, today announced that it has acquired a 100% share of Ommay Health Management Co., Ltd., a middle to high-end private medical examination service center in Chengdu that was officially founded in June 2007.
Inovalon (Nasdaq:INOV), a leading technology company providing advanced cloud-based analytics and data-driven intervention platforms to the healthcare industry, will present results from two studies related to Medicaid and Medicare Advantage quality performance measurement during the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Annual Meeting in Philadelphia. 
Intuitive Surgical, Inc. (Nasdaq:ISRG) today reported that equity awards approved by the Compensation Committee of the Board of Directors, which consists entirely of Independent Directors, were made to 37 new employees. Pursuant to NASDAQ Marketplace Rule 5635(c)(4), the equity awards were granted under the Intuitive Surgical, Inc. 2009 Commencement Incentive Plan, which the Board of Directors of Intuitive Surgical, Inc. adopted for the granting of equity awards to new employees. 
Lumenis Ltd. (Nasdaq:LMNS), the world's largest energy-based medical device company for surgical, ophthalmology, and aesthetic applications announced today that its novel laser urology platform, the Lumenis Pulse 120H, has received the Red Dot Award: Product Design 2015.
Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative epilepsy and other neuropsychiatric therapeutics, announced that Gail M. Farfel, Ph.D., Chief Clinical and Regulatory Officer of Marinus, presented clinical data on ganaxolone in drug-resistant pediatric epilepsies at the Antiepileptic Drug and Device Trials (AED) XIII conference on Friday, May 15, 2015.
Minerva Neurosciences, Inc. (Nasdaq:NERV) today announced the appointment of Fouzia Laghrissi-Thode, M.D. to its board of directors, effective immediately.
Natus Medical Incorporated (Nasdaq:BABY) today announced that Jim Hawkins, President and Chief Executive Officer, will present at the Jefferies 2015 Global Health Care Conference in New York City. 
Otonomy, Inc. (Nasdaq:OTIC), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapeutics for diseases and disorders of the inner and middle ear, today announced the appointment of Eric Loumeau as general counsel and chief compliance officer.
Premier, Inc. (Nasdaq:PINC) has been recognized for environmental excellence by Practice Greenhealth, a leading healthcare community driving environmental stewardship and sustainable practices.
Quotient Limited (Nasdaq:QTNT), a commercial-stage diagnostics company, today reported financial results for its fourth quarter and fiscal year ended March 31, 2015.
RedChip Companies, Inc., today announced it has issued a research update on Cancer Genetics, Inc. (Nasdaq:CGIX), an emerging leader in DNA-based cancer diagnostics.

RedHill Biopharma Ltd. (Nasdaq:RDHL) (TASE:RDHL), an Israeli biopharmaceutical company primarily focused on late clinical-stage, proprietary, orally-administered small molecule drugs for inflammatory and gastrointestinal diseases, including gastrointestinal cancers, held an interactive Investor Webcast Forum and conference call on Helicobacter pylori (H. pylori) bacterial infection and RHB-105 for the treatment of H. pylori infection, on Thursday, May 14, 2015. 

StemCells, Inc. (Nasdaq:STEM) announced today that management will make a presentation on the Company's programs, pipeline and operations at the 2015 Marcum MicroCap Conference. 

Wellness Center USA, Inc. (OTCQB:WCUI), today announced the appointment of Seth B. Forman, MD as Medical Director of Psoria-Shield, Inc. (PSI). Dr. Forman is an internationally recognized expert on the treatment of psoriasis and other auto-immune skin diseases, as well as an early adopter of the Psoria-Light®. 

XBiotech (Nasdaq:XBIT), developer of Xilonix anti-cancer therapy for the treatment of colorectal cancer, announced today that the Data Safety Monitoring Board (DSMB) has recommended continuation of the Company's Phase 3 registration study underway in Europe. 



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