Goldman Comments On Vertex After FDA Approval; Arrowhead Initiates Dosing of ARC-AAT in Patients Print E-mail
By William Kent   
Thursday, 02 July 2015 22:51
Below is a look at some of the headlines for companies that made news in the healthcare sector on July 2, 2015.
Shares of Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) were trading higher by nearly 4 percent on Thursday after the FDA approved the company's Orkambi (lumacafor/ivacaftor) for the treatment of cystic fibrosis patients 12 years and older.
Commenting on the FDA's approval, Terence Flynn, PhD of Goldman Sachs noted that the timing and decision by the FDA was in-line with the July 5 PDUFA date and follows the FDA AdCom's 12-1 recommendation for approval in May.
Flynn noted that the drug's label is not yet available, but based on the FDA AdCom he isn't expecting any surprises. The analyst also added that pricing of Orkambi is not yet available, but is expected to be on par with Kalydeco ($312,000 a year) in the U.S. given a strong secondary endpoint data. Other reasons to support the $312,000 per year cost includes the fact that the analyst is not aware of any precedents to significantly cut prices on already approved drugs and an analysis of historical orphan drug pricing.
Looking forward, the analyst is now expecting investor focus to shift to the drug's launch trajectory and the company's earnings power. The launch is expected to boost the company to profitability and the analyst is now modeling the company to report a non-GAAP earnings per share of $6.54 in 2016 and $9.75 in 2017.
Shares remain Buy rated with an unchanged price target of $142 based on a 70/30 percent blend of DCF ($133) and M&A ($165).
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, announced it dosed the first patient in Part B of a Phase 1 clinical trial of ARC-AAT. ARC-AAT is Arrowhead's RNAi-based drug candidate for the treatment of liver disease associated with the rare genetic disorder alpha-1 antitrypsin deficiency (AATD) that was recently granted orphan drug designation by the United States Food and Drug Administration. The clinical trial is currently enrolling patients at a single center in Australia and the company intends to open additional sites for enrollment in Europe, pending regulatory permission to proceed. The company expects to complete enrollment of the Phase 1 study by the end of 2015.
"Dosing the first alpha-1 patient with ARC-AAT is a milestone for Arrowhead and for patients with AATD. The goal of treatment with ARC-AAT is to halt progression and possibly reverse the liver injury and fibrosis associated with AATD, which currently has no approved therapy short of liver transplant. This is becoming a larger clinical problem that we believe ARC-AAT holds great potential to address," said Bruce D. Given, M.D., Arrowhead's Chief Operating Officer. "We would also like to thank the Alpha-1 Foundation and The Alpha-1 Project, who have agreed to help support the development of ARC-AAT through funding and assistance with patient recruitment."
The ongoing Phase 1 trial of ARC-AAT is a multi-center, randomized, placebo-controlled, double-blind, single dose-escalation, first-in-human study to evaluate the safety, tolerability and pharmacokinetics of ARC-AAT and the effect on circulating AAT levels. The study has been enrolling in dose cohorts of six participants each, with participants randomized at a ratio of 2:1 (active:placebo) to receive a single intravenous injection of either ARC-AAT or placebo (normal saline). The study consists of two parts; Part A in healthy volunteers, which has been completed, and Part B to be conducted in patients with PiZZ genotype AATD. Dosing in patients begins at the highest dose level used in healthy volunteers and then continued dose escalation may proceed under the protocol. The study evaluates participants for 28 days following dosing, with additional follow-up if needed every 2 weeks until AAT levels return to baseline.
Also Thursday:
Adaptimmune Therapeutics plc (Nasdaq:ADAP), a clinical stage biopharmaceutical company focused on the use of T-cell therapy to treat cancer, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's investigational new drug (IND) application for autologous genetically modified T-cells expressing enhanced T cell receptors (TCRs) specific for MAGE A10 (MAGE-A10 T) in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC), and that the IND is now active.
Amyris, Inc. (Nasdaq:AMRS), the industrial bioscience company, today announced an update and clarification with respect to media reports regarding its inactive manufacturing joint venture with Usina São Martinho, SMA Indústria Química S.A, or SMA., noting that, at present, its existing Brotas facility is exceeding targets and provides adequate capacity to meet its near and mid-term business needs.
AngioDynamics (NASDAQ:ANGO), a leading provider of innovative, minimally invasive medical devices for vascular access, surgery, peripheral vascular disease and oncology, today announced it will report financial results for its fiscal 2015 fourth quarter ended May 31, 2015, on Thursday, July 16, 2015, after the close of the U.S. financial markets.
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 that the Company will host a conference call and webcast to discuss its recently announced collaboration with Biogen (NASDAQ:BIIB). 
BioCorRx, Inc. (OTCQB: BICX), developer of the Start Fresh Program, announces the mutual release of Sobriety and Addiction Solutions, LLC (SAS) from its license agreements with the Company.  They were previously offering the Start Fresh Program for locations in Walnut Creek, Fresno and Washington D.C. under MyLife Recovery Centers (MyLife). 
CareTrust REIT, Inc. (Nasdaq:CTRE) today announced that it has acquired Shamrock Nursing and Rehabilitation Center, a 105-bed skilled nursing facility located in Dublin, Georgia, for $8.3 million. 
Celldex Therapeutics, Inc. (NASDAQ:CLDX) announced today that Tibor Keler, Ph.D., Executive Vice President and Chief Scientific Officer, will present a corporate overview at the Cantor Fitzgerald Inaugural Healthcare Conference on July 8, 2015 at 8:30 a.m. EDT in New York City.
Cyclacel Pharmaceuticals, Inc. (NASDAQ:CYCC) (NASDAQ:CYCCP), a biopharmaceutical company developing oral therapies that target the various phases of cell cycle control for the treatment of cancer and other serious disorders today announced that the first patient has been dosed in an investigator sponsored trial (IST) of the Company's oral cyclin dependent kinase (CDK) inhibitor seliciclib in Cushing's disease (CD)1. 
EDAP TMS SA (Nasdaq:EDAP), the global leader in therapeutic ultrasound, today announced that Eric Soyer, Chief Financial Officer, has resigned from the Company to accept a top management position at a publicly traded biotechnology company.
Endocyte, Inc. (Nasdaq:ECYT), a biopharmaceutical company and leader in developing targeted small molecule drug conjugates (SMDCs) and companion imaging agents for personalized therapy in cancer and other serious diseases, today announced that the company's management team will present at the Cantor Fitzgerald Healthcare Conference on Wednesday, July 8, at 4:15 p.m. EDT. 
Inogen, Inc. (NASDAQ:INGN), a medical technology company offering innovative respiratory products for use in the homecare setting, announced today that Bob Fary, Inogen VP of Strategic Alliances, has been selected to join the American Association of Homecare Board of Directors.
Integra LifeSciences Holdings Corporation (NASDAQ:IART) today announced that it has completed the previously announced tax-free spin-off of its orthobiologics and spinal fusion hardware business, now known as SeaSpine Holdings Corporation (NASDAQ:SPNE).
K2M Group Holdings, Inc. (Nasdaq:KTWO), a global medical device company focused on designing, developing and commercializing innovative and proprietary complex spine technologies, techniques and minimally invasive procedures, today announced the U.S. commercial launch of the MESA® 2 Deformity Spinal System, the Company's next-generation pedicle screw system designed to address the most complex spinal pathologies. 
In a video statement, Stuart W. Titus, PhD and Chief Executive Officer of Medical Marijuana, Inc. (OTC Pink:MJNA), makes it clear what the Company's vision and mission is: inspire change. 
Medical Marijuana, Inc. (OTC Pink:MJNA), an industrial hemp industry innovator, is proud to announce that its portfolio company, Dixie Botanicals™, one of the most widely recognized brands in the cannabis industry, has launched Hemp Stalk Oil Dew Drops® in an all-new flavor: peppermint. 
NeuroDerm Ltd. (NASDAQ:NDRM), a clinical stage pharmaceutical company developing drugs for central nervous system (CNS) diseases, today announced that it has filed a registration statement on Form F-1 with the U.S. Securities and Exchange Commission for a proposed public offering of its ordinary shares. 
Onconova Therapeutics, Inc. (Nasdaq:ONTX) a clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, today announced that Ramesh Kumar, Ph.D., President and Chief Executive Officer, will present a company update, including details of a new global Phase 3 trial in higher-risk myelodysplastic syndromes at the Cantor Fitzgerald Healthcare Conference in New York, NY.
Revance Therapeutics, Inc. (NASDAQ:RVNC), a specialty biopharmaceutical company developing botulinum toxin products for the use in aesthetic and therapeutic indications, announced today that Jacob Waugh, M.D. will relinquish his role as Chief Scientific Officer, effective July 2, 2015, and consult on key programs while serving as an advisor to the CEO and the Science & Technology Committee of the Company's Board.
Relypsa, Inc. (Nasdaq:RLYP), today announced that John A. Orwin, president and chief executive officer, will present at the Cantor Fitzgerald Inaugural Healthcare Conference on July 8, 2015 at 11:45 a.m. ET.
SeaSpine Holdings Corporation (NASDAQ:SPNE), a global medical technology company focused on surgical solutions for the treatment of spinal disorders, today announced the completion of its tax-free spinoff from Integra LifeSciences Holdings Corporation (NASDAQ:IART). 
The Spectranetics Corporation (Nasdaq:SPNC) today announced that the U.S. District Court for the Northern District of California has ruled in favor of its wholly-owned subsidiary, AngioScore Inc., in a lawsuit filed by AngioScore seeking damages for breach of fiduciary duty against Eitan Konstantino, a former board member of AngioScore and founder of TriReme Medical, LLC, Quattro Vascular Pte Ltd. and QT Vascular Ltd. (SGX:5I0). 
Stereotaxis, Inc. (NASDAQ:STXS), a global leader in innovative technologies for the treatment of cardiac arrhythmias, today announced that William C. Mills, CEO, and Martin C. Stammer, CFO, will present at the Cantor Fitzgerald Inaugural Healthcare Conference on Wednesday, July 8, 2015 at 2:30 p.m. Eastern Time. 

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