CORAL SPRINGS, Florida, October 8, 2015 /PRNewswire/ --
With recent reports, studies and collaborations between cell and tissue biologists, geneticists, biotech companies and other industry experts, treatment of skin diseases and disorders fuel the development of biological substitutes to restore damaged tissue with the possibilities of actually re-growing skin or maintain tissue function. In addition to skin substitute therapies; rare disease & conditions, human genetics, cystic fibrosis and cancer immunotherapy advancements are leading the biotech sector led by Amarantus Bioscience Holdings, Inc. (OTCQX: AMBS), Pacific Biosciences of California, Inc., (NASDAQ: PACB), Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX), Juno Therapeutics (NASDAQ: JUNO), Nivalis Therapeutics, Inc. (NASDAQ: NVLS).
Amarantus Bioscience Holdings, Inc. (OTCQX: AMBS), a biotechnology company developing therapeutic and diagnostic product candidates in orphan indications and neurology, reported on preliminary 13-year longitudinal follow-up data presented on Engineered Skin Substitute (ESS) for the treatment of pediatric severe burns and Congenital Giant Hair Nevus at the Tissue Products for Emergency Preparedness Symposium at the National Archives in Washington, DC in May 2015.
Amarantus completed the acquisition of ESS in July 2015, and has focused the Company's near-term execution strategy on the advancement of ESS in several therapeutic areas including Congenital Giant Hairy Nevus and pediatric severe burns, two rare pediatric diseases that the Company believes would be eligible for a Rare Pediatric Disease Designation (RPDD) by the US Food and Drug Administration (FDA). The Sponsor of a treatment approved under the RPDD pathway is eligible to receive a PRV that may be sold to other companies. Two PRVs have been sold in the last 6 months for an aggregate of $595 million to major pharmaceutical companies. The proceeds from the sale of a PRV are in addition to any potential revenues that could be generated from product sales. The Company intends to apply to the FDA for both orphan drug designation (ODD) and RPDD for ESS treatment of Congenital Giant Hairy Nevus in the fourth quarter of 2015.
Read the full AMBS Press Release at http://www.financialnewsmedia.com/profiles/ambs.html
Congenital Giant Hairy Nevus is a rare pediatric disease defined by one or more large, darkly pigmented and sometimes hairy patches with a projected adult diameter of over 40 cm with hypertrichosis. The condition is sometimes known as giant congenital melanocytic nevus. The estimated prevalence for the largest forms is 0.002% of births, affecting approximately 1 in 50,000 to 1 in 500,000 births annually. Melanocytic Nevi often grow proportionally to the body size as the child matures. As they mature, they often develop thickness, and become elevated, although these features can also be present from birth. Prominent terminal hairs often form, especially after puberty. With maturity, the nevus can have variation in color, and the surface might be textured with proliferative growths. Neurocutaneous melanosis is associated with the presence of either giant congenital melanocytic nevi or non-giant nevi of the skin. It is estimated that neurocutaneous melanosis is present in 2% to 45% of patients with giant congenital melanocytic nevi. Neurocutaneous melanosis is characterized by the presence of congenital melanocytic nevi on the skin and melanocytic tumors in the leptomeninges of the central nervous system.
Gerald E. Commissiong, President & CEO of Amarantus stated "Congenital Giant Hairy Nevus can be a devastating condition affecting not only the physical, but also the psychosocial maturation of newborns as they grow into childhood. Any clinical benefit that could be provided in terms of cosmesis outcomes will be heralded both by children and their families. ESS represents a potential paradigm shift in this indication, as not only is there potential initial cosmesis benefit, but also a potential long-term cosmesis improvement, as well as the potential reduction or elimination in reconstructive procedures due to the unique properties of ESS growing with pediatric patients - which distinguishes ESS from all other available treatment options in the market or in development that we are aware of. This property makes ESS potentially especially beneficial for the pediatric dermatologic population which is in dire need of a permanent full thickness skin replacement options."
In other recent biotech developments of importance for the sector: Pacific Biosciences of California, Inc., (NASDAQ: PACB) a pioneer and leader in long-read sequencing using its Single Molecule, Real-Time (SMRT(R)) Technology, recently announced it has launched a new nucleic acid sequencing platform. The Sequel(TM) System provides higher throughput, more scalability, a reduced footprint and lower sequencing project costs compared to the PacBio(R) RS II System, while maintaining the existing benefits of the company's SMRT Technology. Pacific Biosciences showcased the new product at the American Society of Human Genetics annual meeting that took place in Baltimore, Maryland held on October 6, 2015. The core of the Sequel System is the capacity of its redesigned SMRT Cells, which contain one million zero-mode waveguides (ZMWs) at launch, compared to 150,000 ZMWs in the PacBio RS II. Active individual polymerases are immobilized within the ZMWs, providing windows to observe and record DNA sequencing in real time.
Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) announced that the U.S. Food and Drug Administration (FDA) has accepted for review a supplemental New Drug Application (sNDA) for the use of KALYDECO® (ivacaftor) in people with cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations. The FDA granted Vertex's request for Priority Review of this sNDA, and a target review date of February 6, 2016 was set under the Prescription Drug User Fee Act (PDUFA) for the FDA's decision on the sNDA. The submission was based on preclinical and clinical data showing the effect of ivacaftor on CFTR function in certain residual function mutations. "Given the severity of cystic fibrosis, we are committed to getting KALYDECO to more people as quickly as possible," said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. "Based on the established safety profile of KALYDECO and our increasing understanding of the biology of these specific residual function mutations and their response to ivacaftor, we believe that people with these mutations would benefit from treatment with this medicine."
Seattle immunotherapy biotech Juno Therapeutics (NASDAQ: JUNO) is responding well to a bullish analyst report released Tuesday by FBR Co. Juno's stock shot up nearly 10 percent Tuesday, closing at $47.45 per share, and continued to climb, closing 3 percent higher Wednesday. FBR set a price target of $73 per share for Juno - higher than the stock has ever been - and gave it a rating of "outperform," expecting it to fare slightly better than the market average. Read the full article at http://www.bizjournals.com/seattle/blog/health-care-inc/2015/10/juno-stock-gets-a-big-bump-after-analyst-sets.html?ana=yahoo
Nivalis Therapeutics, Inc. (NASDAQ: NVLS), a clinical stage pharmaceutical company focused on treating people with cystic fibrosis (CF), announced positive topline results from a Phase 1b clinical study evaluating N91115 that demonstrated favorable safety, tolerability and pharmacokinetics in adult CF patients with two copies of the F508del-CFTR mutation. N91115 is the Company's novel stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In this randomized, double-blind, placebo-controlled, parallel group study, 51 adult CF patients were randomly assigned to receive placebo or N91115 at doses of 50, 100 or 200 mg administered twice daily for 28 days.
Lion Biotechnologies (NASDAQ: LBIO), a biotechnology company that is developing novel cancer immunotherapies based on tumor-infiltrating lymphocytes (TIL), today announced that it has obtained an exclusive, worldwide license from the National Institutes of Health (NIH) to develop and commercialize TIL therapy in four additional tumor indications. Under the agreement, the NIH has granted Lion exclusive rights to certain patents to develop TIL in the treatment of bladder, lung, breast and HPV-associated cancers, including cervical and head and neck. The agreement was executed as an amendment to Lion's existing exclusive licensing agreement with the NIH for the development and commercialization of TIL in the treatment of metastatic melanoma.
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