Vertex Treatment May Have High Risk of Failure; Agios Announces Orphan Drug Designation of AG-348 Print E-mail
By Josh Gee   
Tuesday, 24 March 2015 18:56
Below is a look at some of the headlines for companies that made news in the healthcare sector on March 24, 2015.
Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) recent results from a prospective cystic fibrosis treatment suggests the product has a high risk of failure, an analyst said Tuesday.
Maxim Group's Jason Kolbert maintained a Hold rating on Vertex with a $121 target.  Vertex is off 5 percent since reporting the results Monday. The company traded recently at $125.16, down 0.5 percent on the day.  Kolbert said results of the Phase II study of Vertex's VX-661 drug in combination with its ivacaftor drug were "disappointing."
Patients suffering from cystic fibrosis inherit mutated genes from each parent, with the mutations either "homozygous," the same, or "heterozygous," different.  The two classifications create subgroups for treatment, and Kolbert said success for Vertex must encompass both sets of patients.  Kolbert said the recent study suggests that the risk of failure in the heterozygous population is high."
The company said lung function improved 4.4 percent among patients in the homozygote population after four weeks of treatment, but gains fell to 3 percent after 12 weeks.  "There are more variables in the heterozygous population," Kolbert said.  Some investors had reportedly hoped for lung function gains 6 percent.
Agios Pharmaceuticals, Inc. (Nasdaq:AGIO), announced that the United States Food and Drug Administration (FDA) has granted the company orphan drug designation for its investigational medicine AG-348 for the treatment of pyruvate kinase (PK) deficiency, a rare form of hemolytic anemia. AG-348, a first-in-class, orally available activator of pyruvate kinase-R (PKR) enzymes, met its primary endpoints in two Phase 1 healthy volunteer studies - a single ascending dose study and multiple ascending dose study. In addition, data presented in December 2014 at the 54th Annual Meeting of the American Society of Hematology (ASH) provided early proof-of-mechanism for AG-348. Based on these findings, Agios plans to initiate a Phase 2 clinical trial in patients with PK deficiency in the first half of 2015.
"We are pleased to achieve another milestone in the clinical program for AG-348, the first medicine in development designed to treat the underlying cause of PK deficiency," said Chris Bowden, M.D., chief medical officer of Agios. "PK deficiency can result in lifelong medical problems and is an example of our focus on underserved diseases with significant medical needs."
The FDA's Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders that affect fewer than 200,000 people in the U.S. This designation provides certain benefits, including market exclusivity upon regulatory approval, if received, exemption of FDA application fees and tax credits for qualified clinical trials.
About AG-348 Clinical Development Plans and Upcoming Milestones -- Based on findings presented at ASH, Agios expects to initiate a Phase 2 study of AG-348 in patients with PK deficiency in the first half of 2015. The company plans to provide final results from the Phase 1 multiple ascending dose study at a medical conference in mid-2015. A natural history study of PK deficiency is also ongoing and Agios expects to report initial data from this study at a medical conference in mid-2015.
Also Tuesday:
Affimed N.V. (Nasdaq:AFMD), a clinical-stage biopharmaceutical company developing targeted cancer immunotherapies today announced the achievement of the second milestone in its development partnership with Amphivena Therapeutics, Inc. (Amphivena). 
Aldeyra Therapeutics, Inc. (Nasdaq:ALDX), a biotechnology company focused on the development of products to treat diseases related to free aldehydes, today announced that it has enrolled the first patient in its Phase II clinical trial of NS2 for the treatment of Sjögren-Larsson Syndrome (SLS). 
Arno Therapeutics, Inc. (OTCQB:ARNI), a clinical stage biopharmaceutical company primarily focused on the development of oncology therapeutics, today announced that data from a preclinical study demonstrate its histone-deacetylase (HDAC) inhibitor AR-42 in combination with cisplatin has a synergistic anti-tumor effect in bladder cancer models. 
BG Medicine, Inc. (Nasdaq:BGMD) today announced that the company will host a conference call and webcast on Tuesday, March 31, 2015 at 8:30 am Eastern Time to discuss its fiscal year 2014 financial results before the market opens.
Biocept, Inc. (Nasdaq:BIOC), a molecular oncology diagnostics company specializing in biomarker analysis of circulating tumor DNA (ctDNA) and circulating tumor cells (CTCs), today announced that it has entered into a clinical collaboration with University of California, San Diego Moores Cancer Center to determine the clinical utility of detecting biomarkers present in CTCs and ctDNA in blood samples for non-small cell lung cancer patients using Biocept's OncoCEE™ LU platform and CEE-Selector™ technology.
Cellectar Biosciences, Inc. (Nasdaq:CLRB), a clinical stage biopharmaceutical company developing innovative agents for the detection and treatment of cancer, is providing an overview of its development programs and financial results for the year ended December 31, 2014.
Celladon Corporation (Nasdaq:CLDN), a clinical-stage cardiovascular gene therapy company and Novasep, a leading supplier of services and technologies for the life sciences and chemical industries, today announce that they have signed a Development, Manufacturing and Supply Agreement pursuant to which, if supported by upcoming MYDICAR clinical data, Novasep would manufacture MYDICAR drug substance through 2018 with extension options through 2020.
Drexel University College of Medicine renewed its agreement with Allscripts (Nasdaq:MDRX) and the use of Allscripts TouchWorks EHR as its core electronic health record (EHR) solution. In addition, Drexel implemented the Allscripts FollowMyHealth as its patient engagement platform.
DS Healthcare Group, Inc. (NASDAQ:DSKX), today will introduce Revita styling products to its current line of Revita treatment products. These products have been designed to complement the Company's existing industry-leading hair care  and topical treatment products that promote healthy hair growth.
DS Healthcare Group, Inc. (NASDAQ:DSKX), today will introduce Revita styling products to its current line of Revita treatment products.
Emergent BioSolutions Inc. (NYSE:EBS) announced today that it has signed a contract with the Biomedical Advanced Research and Development Authority (BARDA) for the advanced development of NuThraxTM (anthrax vaccine adsorbed with CPG 7909 adjuvant), also known as AV7909, the company's next generation anthrax vaccine candidate. 
Enzymotec Ltd. (Nasdaq:ENZY), a developer, manufacturer and marketer of innovative bio-active lipid ingredients, reported today that on March 23, 2015 the US Patent and Trademark Office issued a decision in an Inter Partes Review ("IPR") of Neptune Technologies & Bioressources' U.S. Patent No. 8,278,351 ('351 patent). 
FlexPower, Inc. (OTCMKTS:FLXP) is pleased to announce its attendance at the upcoming Northwest Athletic Trainers' Association (NWATA) annual meeting and clinical symposium, Rocky Mountain Athletic Trainers' Association (RMATA) symposium, as well as the Far West Athletic Trainers' Association (FWATA) annual meeting and clinical symposium between March 27th and April 19th.
Galectin Therapeutics (Nasdaq:GALT), the leading developer of therapeutics that target galectin proteins to treat fibrosis and cancer, announces that Peter G. Traber, M.D., president, chief executive officer and chief medical officer, delivered an invited presentation of the Company's research with lead compound GR-MD-02 in non-alcoholic steatohepatitis (NASH) at the American Association for the Study of Liver Diseases (AASLD) Industry Colloquium. 
HealthEquity, Inc. (Nasdaq:HQY), one of the largest health savings account ("HSA") non-bank custodians, today announced financial results for its fourth quarter and year ended January 31, 2015.
Hemispherx Biopharma, Inc. (NYSE MKT:HEB) announces that the European subsidiary, "Hemispherx Biopharma Europe N.V./S.A" gained a positive opinion from the COMP (Committee on Medical Products) regarding its Orphan Medicinal Product Application for Ampligen®, an experimental therapeutic, to treat Ebola Virus Disease (EVD).
Herborium® Group, Inc. (OTC Pink: HBRM), a Botanical Therapeutics® Company and the provider of unique all  botanical medicines (Botanical Therapeutics®)  announced today that last week its Board of Directors approved an increase in the number of Company authorized common shares to 10 billion shares.
Kips Bay Medical, Inc. (OTCQB:KIPS), a medical device company focused on manufacturing and commercializing its external saphenous vein support technology, eSVS® Mesh, for use in coronary artery bypass grafting surgery, today announced that the Company has entered into a securities purchase agreement with several investors, including Manny Villafaña, the Company's Chairman and Chief Executive Officer, and Kips Bay Investments, LLC, one of the Company's principal stockholders, pursuant to which such investors have agreed to purchase up to $3.25 million in shares of the Company's common stock in four equal tranches, subject to certain terms and conditions.
RestorGenex Corporation (OTCQB:RESX), a specialty biopharmaceutical company focused on developing products for oncology, ophthalmology and dermatology, today announced that Stephen M. Simes, chief executive officer of RestorGenex, was interviewed by
StemCells, Inc. (Nasdaq:STEM), a leading stem cell company developing novel cell-based therapeutics for disorders of the central nervous system, today announced that Ian Massey, D. Phil., has joined its executive team as President and Chief Operating Officer, effective March 23, 2015. 
Wright Medical Group, Inc. (Nasdaq:WMGI) today announced that it will highlight recent innovations at the American Academy of Orthopaedic Surgeons (AAOS) 2015 Annual Meeting taking place at the Venetian/Sands EXPO in Las Vegas, Nevada from March 24-28, 2015.

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