UBS Says Sunesis Leukemia Pivotal Trial Success 'Likely'; Asterias Biotherapeutics Receives U.S. FDA Clearance To Initiate Phase 1/2a Clinical Trial Of AST-OPC1 Print E-mail
By Staff and Wire Reports   
Wednesday, 27 August 2014 18:49

Below is a look at some of the headlines for companies that made news in the healthcare sector on August 27, 2014.
Sunesis Pharmaceuticals (NASDAQ: SNSS) gained 10 percent Wednesday to near a year high after an analyst said success is "more likely than not" for the company's development drug for leukemia.

With key data from a Phase III pivotal trial expected in coming weeks, UBS' Andrew Peters expects an "approvable" outcome.

But if indeed the drug, called Qinprezo, can be approved by the U.S. Food and Drug Administration, that still begs the question of the degree of its benefits.

"The FDA has historically approved oncology drugs with only a 'modest' benefit only to see sales disappoint," Peters said in a note maintaining a Buy rating and $14 target.

"The entire data set will be critically important to consider Qinprezo as a value proposition," Peters said.

While Peters is optimistic Qinprezo will deliver significant benefits, even merely clearing the approval hurdle in his view would offer upside to Sunesis shares.

Shares closed at $8.23, up 9.8 percent.



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BioTime, Inc. (NYSE MKT: BTX) announced that its subsidiary Asterias Biotherapeutics, Inc. (OTCBB:ASTY) has received clearance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1/2a clinical trial of its product, AST-OPC1, in patients with complete cervical spinal cord injury. The approved trial follows the successful completion of the Phase 1 clinical study of the product, and is designed to assess safety and activity of escalating doses of AST-OPC1 in patients with complete cervical spinal cord injuries, the first targeted indication for AST-OPC1 and the first of future product registration clinical trials.
 
“We would like to acknowledge the scientists, clinical investigators, and FDA for working with us to develop AST-OPC1,” stated Pedro Lichtinger, President and CEO of Asterias. “We are especially enthusiastic about working with our new partner, CIRM, in executing this clinical trial. The FDA clearance provides Asterias with imminent access to the previously announced $14.3 million CIRM grant, which provides non-dilutive funding to support both the clinical trial and other product development activities for AST-OPC1.”
 
AST-OPC1 is a population of cells derived from human embryonic stem cells (hESCs) that contains oligodendrocyte progenitor cells (OPCs). OPCs and oligodendrocytes perform supportive functions for nerve cells in the central nervous system. The foundation for this newly cleared Phase 1/2a clinical trial comes from results from the Phase 1 clinical trial of AST-OPC1, which met its primary endpoints of safety and feasibility when administered to five patients with neurologically-complete, thoracic spinal cord injury. These five patients were administered a low dose of two million AST-OPC1 cells and have been followed to date for 2 to 3 years. No serious adverse events were observed associated with the delivery of the cells, the cells themselves, or the short-course immunosuppression regimen used. There was no evidence of expanding masses, expanding cysts, infections, cerebrospinal fluid leaks, increased inflammation, neural tissue deterioration or immune responses targeting AST-OPC1 in these patients. In four of the five subjects, serial MRI scans performed throughout the 2 to 3 year follow-up period indicate that reduced spinal cord cavitation may have occurred and that AST-OPC1 may have had some positive effects in reducing spinal cord tissue deterioration.
 
The new Phase 1/2a clinical trial will be an open-label, single-arm study testing three escalating doses of AST-OPC1 in 13 patients with subacute, C5-C7, neurologically-complete cervical spinal cord injury. These individuals have essentially lost all sensation and movement below their injury site with severe paralysis of the upper and lower limbs. AST-OPC1 will be administered 14 to 30 days post-injury. Patients will be followed by neurological exams to assess the safety and activity of the product. Selection of the clinical trial sites is well underway and Asterias expects to begin patient enrollment during the first quarter of 2015.
 
The new clinical trial differs from the original clinical study in that doses up to 10 times higher will be tested. In addition, the trial will focus on patients with neurologically-complete cervical spinal cord injuries. Because of the anatomy of the spinal cord and the existence of more sensitive outcomes measures to assess movement of the arms and hands, it is currently believed that detection of efficacy is much more likely to occur in patients with cervical injuries. It is this patient population that Asterias anticipates will be the target for the first registration clinical trials of AST-OPC1. Asterias expects that the results of the Phase 1/2a clinical trial will provide support for a Phase 2b expansion study that will be conducted to more thoroughly demonstrate safety and efficacy of the product.
 
There are currently no approved therapies for the treatment of spinal cord injury (SCI), and the complex pathology of the injury is unlikely to be addressed by a traditional small molecule or protein therapeutic. AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown to have three potentially reparative functions which address the complex pathologies observed at the SCI injury site. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.




Also Wednesday:



Amarantus Bioscience Holdings, Inc. (OTCQB:AMBS)
, a biotechnology company focused on the development of diagnostic and therapeutic interventions for Alzheimer's disease, Parkinson's disease and orphan ophthalmological disorders, reported financial results for the second quarter ended June 30, 2014 and announced a business update call on September 2nd, 2014.

Auspex Pharmaceuticals, Inc. (Nasdaq:ASPX)
, a late clinical stage biopharmaceutical company focused on developing and commercializing novel medicines for the treatment of orphan diseases, today announced that Pratik Shah, Ph.D., President and Chief Executive Officer, will present at the Baird 2014 Healthcare Conference on Wednesday, September 3, 2014 at 2:55 p.m. EDT at the New York Palace Hotel in New York City.

BioReference Laboratories, Inc. (Nasdaq:BRLI)
announces record third quarter results for revenues and patient volumes.

Cancer Genetics, Inc. (Nasdaq:CGIX)
, an emerging leader in DNA-based cancer diagnostics, announced today that the National Institutes of Health's (NIH) Genetic Testing Registry now features the company's proprietary diagnostic and prognostic genomic tests.

CareDx, Inc. (Nasdaq:CDNA)
a molecular diagnostics company focused on the development and commercialization of clinically differentiated, high value, non-invasive surveillance solutions for transplant recipients today reported financial results for the three and six months ended June 30, 2014.

Cellular Biomedicine Group Inc. (Nasdaq:CBMG)
a biomedicine firm engaged in the development of new treatments for degenerative and cancerous diseases, today announced that Dr. William (Wei) Cao, CEO, will be presenting at the Rodman & Renshaw 16th Annual Global Investment Conference on Tuesday, September 9 at 4:10 P.M. EDT.

Curis, Inc. (Nasdaq:CRIS)
, an oncology-focused biotechnology company developing novel drug candidates for the treatment of human cancers, today announced that the Company will present at the following upcoming conferences:  Baird's 2014 Health Care Conference, Wednesday, September 3, 2014 at 10:15 a.m. ET. A corresponding webcast of this presentation can be accessed by visiting: http://wsw.com/webcast/baird39/cris; Rodman & Renshaw 16th Annual Global Investment Conference, Wednesday, September 10, 2014 at 11:15 a.m. ET. A corresponding webcast of this presentation can be accessed by visiting:   http://wsw.com/webcast/rrshq24/cris.

Endocyte, Inc. (Nasdaq:ECYT)
, a leader in developing targeted small molecule drug conjugates (SMDCs) and companion imaging agents for personalized therapy, today announced that the company's management team will present at the Baird 2014 Health Care Conference on Wednesday, Sept. 3, at 2:20 p.m. EDT.

India Globalization Capital, Inc. (NYSE MKT: IGC)
, announced that its 2014 Annual Meeting of Stockholders scheduled for, and convened on August 25, 2014, was adjourned due to the lack of requisite quorum. 

NanoString Technologies, Inc., (Nasdaq:NSTG)
a provider of life science tools for translational research, and molecular diagnostic products, today announced the unexpected death of J. Wayne Cowens, M.D., the company's Chief Medical Officer.

Marrone Bio Innovations, Inc., (Nasdaq:MBII)
, a leading provider of bio-based pest management and plant health products for the agriculture, turf and ornamental and water treatment markets, today announced that its VENERATE™ Bioinsecticide is now registered in California.

Medical Transcription Billing, Corp. (Nasdaq:MTBC)
, a leading provider of proprietary, web-based electronic health records, practice management and mHealth solutions, today announced financial and operational results for the second quarter of fiscal year 2014.

RedHill Biopharma Ltd. (Nasdaq:RDHL) (TASE:RDHL)
, an Israeli biopharmaceutical company focused on late clinical-stage drugs for inflammatory and gastrointestinal diseases, including gastrointestinal cancers, today provided an update on the ongoing RHB-105 Phase III ERADICATE Hp study.

TetraLogic Pharmaceuticals Corporation (Nasdaq:TLOG)
today announced that data from the preclinical studies that provide the rationale for SMAC-mimetics as a potential therapy for Hepatitis B will be presented at the 2014 International Meeting on Molecular Biology of Hepatitis B Viruses to be held in Los Angeles at UCLA from September 3-6, 2014.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE)
, a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Tom Kassberg, the company's Chief Business Officer, will be presenting at the Robert W. Baird & Co. 2014 Health Care Conference on September 3, 2014 at 1:45pm ET in New York.




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