Provectus Biopharmaceuticals Concludes FDA Meeting for PV-10 Phase 3; Successful Completion of AB Science's Futility Test For Masitinib Print E-mail
By Marilyn Mullen   
Monday, 09 February 2015 20:48
Below is a look at some of the headlines for companies that made news in the healthcare sector on February 9, 2015.
Provectus Biopharmaceuticals (NYSE: PVCT) announced it has held a Type C meeting with the U.S. Food and Drug Administration to review certain operational aspects of the protocol for its planned phase 3 clinical trial of intralesional PV-10, its novel investigational drug for cancer, as a treatment for melanoma. The meeting was held by teleconference on January 29, 2015.

Eric Wachter, PhD, CTO of Provectus, stated, “As noted in our press release of December 22, 2014, when we submitted the protocol to the Agency in November 2014, we included a brief list of questions about certain operational aspects of the protocol. The FDA subsequently indicated that a formal meeting was appropriate to assure that these questions were addressed in a timely and comprehensive manner. As is typical for such meetings, we provided a more extensive list of questions in the formal meeting package. This led to a very thorough and helpful review of the protocol as a result of the meeting.”

Topics formally reviewed included subject eligibility requirements, primary and secondary study end points, and study lesion definitions and conventions for defining disease progression.

Dr. Wachter noted, “We are extremely grateful for the careful review conducted by the Agency. The outcome of the review does not affect the fundamental design of the study nor the patient population, but does affect certain details concerning some secondary end points and statistical analysis matters, such as the treatment of missing data. We are making a number of small changes to the protocol in light of this review, and will issue a final version later this month.”

Dr. Wachter added, “We have eight sites, four in the U.S. and four in Australia, in our expanded access program currently using PV-10 for melanoma and other cutaneous malignancies. We expect that they will provide a path to quickly starting enrollment upon completion of the review period. In addition, we have been qualifying additional sites that will join the study pending action by their respective Institutional Review Boards.”


AB Science SA (NYSE Euronext - FR0010557264 - AB)
, a pharmaceutical company specialized in research, development and marketing of protein kinase inhibitors (PKIs), today announced the successful completion of a futility analysis related to the masitinib phase 3 trial for the treatment of mild to moderate Alzheimer's disease. Based on these results, the Independent Data Safety Monitoring Committee (IDMC) has recommended the continuation of the study.

Phase 3 status -- The ongoing phase 3 trial is a double-blind, randomized, placebo-controlled study (AB09004) designed to assess the safety and efficacy of masitinib in patients with confirmed mild to moderate Alzheimer's disease. The treatment period is 24 weeks and masitinib is being given as an add-on therapy to cholinesterase inhibitor (donepezil, rivastigmine or galantamine and/or memantine). The main measures are the change in two commonly used clinical assessments: the effect on ADCS-ADL, which measures self-care and activities of daily living assessed, and the effect on ADAS-Cog, which measures the effect on cognition and memory. The study is intended to enroll about 600 patients.

The study was recently assessed as non-futile by the IDMC. A futility analysis tests the inability of a clinical study to achieve its efficacy objective. Therefore, a conclusion that a study is not futile suggests that a clinical study has the potential to achieve its stated efficacy objective. The IDMC analysis was performed after about one third of the patients were enrolled into the study and had reached the 24 week treatment duration period.

The study previously successfully passed all safety data reviews by the IDMC, indicating that there is no major or unexpected safety concern with masitinib in this patient population.

Previous establishment proof of concept -- As a reminder, proof of concept for the evaluation of masitinib in Alzheimer's disease was established through a 35 patient double-blind, placebo-controlled phase 2 study. In this study, the rate of clinically relevant cognitive decline, according to the primary endpoint, ADAS-Cog response (increase >4 points), was significantly lower with masitinib treatment compared with placebo after 12 and 24 weeks (6% versus 50% for both; p=0.040 and p=0.046, respectively). Moreover, while the placebo treatment-arm demonstrated worsening mean ADAS-Cog, ADCS-ADL and MMSE scores, the masitinib treatment-arm reported improvements with statistical significance between treatment-arms at weeks 12 and/or 24 (respectively, p=0.016 and 0.030; p=0.035 and 0.128; and p=0.047 and 0.031). Adverse events occurred more frequently with masitinib treatment (65% versus 38% of patients); however, the majority of events were mild or moderate and transient. The phase 2 results were published in Alzheimers Res Ther. 2011 Apr 19;3(2):16. doi: 10.1186/alzrt75.

Scientific rationale -- The potential therapeutic benefit of masitinib in Alzheimer's disease is linked to two possible mechanisms of action: the role of mast cells in neuroinflammation and regulation of the blood-brain-barrier (BBB) permeability; and the inhibition of the protein kinase Fyn, which is involved in A-beta signaling and Tau phosphorylation.

Neuroinflammation is thought to be a major contributor in the pathogenesis of Alzheimer's disease1,2,3. Mast cells release large amounts of proinflammatory mediators and therefore play an important role in sustaining the inflammatory network of the central nervous system. Furthermore; mast cells are found on both sides of the BBB and also have the ability to rapidly cross the BBB, thereby increasing their numbers in response to physiological stimuli. Given that the neural pool of mast cells is influenced by their ability to rapidly cross the BBB, inhibition of mast cells peripheral to the BBB could impact upon neurodegenerative disease outcome. Therefore, masitinib could be an effective drug in Alzheimer's disease because it blocks mast cells through the inhibition of the tyrosine kinases c-Kit and Lyn.

In addition to blocking mast cell activity, masitinib may exert an effect through its inhibition of the tyrosine kinase Fyn4,5,6. Alzheimer's disease is associated with the pathological aggregation of amyloid-beta (A-beta) plaques and tau-positive neurofibrillary tangles. Several lines of evidence implicate Fyn in the pathogenesis of Alzheimer's disease through its dual role in A-beta signaling and Tau phosphorylation. Masitinib, by inhibiting Fyn, could possibly disrupt the A-beta signaling cascade and modulate the phosphorylation of tau protein, thus and preventing neurofibrillary tangles.

Targeted population -- The meta-analysis of epidemiologic studies indicates that between 5 and 10 million people suffer from Alzheimer's disease in the USA and Europe. Alzheimer's disease is the most common type of dementia among western countries, corresponding to about 60% of cases. Alzheimer's disease is already the sixth leading cause of all deaths in USA and the fifth leading cause among Americans over 65 years of age.7,8,9 Worldwide, it is thought that there are more than 15 million people affected by Alzheimer's disease.8

Currently, there are only five products approved for the treatment of Alzheimer's disease, four of which belong to the pharmacological class of anticholinesterases, the fifth being an NMDA inhibitor. Therefore, this remains an area of significant unmet medical need.  Accordingly, the FDA recently issued new guidance (21 CFR 149 314.510) that allows for the potential of conditional approval.

Also Monday: 

Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today announced updated interim results from the ongoing interferon-free, ribavirin-free, Phase 2 study to evaluate the efficacy, safety, and tolerability of six weeks of 50 mg of ACH-3102 and 400 mg of sofosbuvir, a marketed nucleotide polymerase inhibitor, in treatment-naïve genotype 1 HCV-infected patients.

Akers Biosciences, Inc. (Nasdaq:AKER) (AIM:AKR.L)
, a medical device company focused on reducing the cost of healthcare through faster, easier diagnosis, announces that the Company's Management System has been certified by SAI Global, a leading global management systems Certification Body, to ISO 13485 (the "Certification").

Anavex Life Sciences Corp. (OTCQX:AVXL)
today confirmed positive preclinical data for its lead drug candidate ANAVEX 2-73 for the potential treatment of epilepsy, validating it also as a prospective platform drug for the treatment of other neurodegenerative diseases beyond Alzheimer's.

Atara Biotherapeutics, Inc. (Nasdaq:ATRA)
, a biopharmaceutical development company with a focus on innovative therapies for patients with debilitating diseases, today announced that it has commenced an underwritten public offering of 3,000,000 shares of its common stock.

Biocept, Inc. (Nasdaq:BIOC)
, a molecular oncology diagnostics company specializing in biomarker analysis of circulating tumor DNA and circulating tumor cells, today announced the pricing of an underwritten public offering of 8,000,000 shares of its common stock and warrants to purchase up to an aggregate of 8,000,000 shares of its common stock at a combined offering price of $1.25.

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN)
announced today that the company would make two data presenations in addition to presenting eight posters at the Lysosomal Disease Network's 11th Annual WORLD Symposium™ from February 9-13 in Orlando, Florida.

BSD Medical Corporation (Nasdaq:BSDM)
, a leading provider of medical systems that utilize heat therapy to treat cancer, today announced the resignation of Gerhard W. Sennewald, Ph.D., and Douglas P. Boyd, Ph.D., as members of the Board of Directors. Mr. Boyd's resignation was effective on January 28, 2015 and Mr. Sennewald's resignation was effective February 4, 2015.

Celldex Therapeutics, Inc. (Nasdaq:CLDX)
announced today that Anthony Marucci, President and Chief Executive Officer, and Tom Davis, M.D., Executive Vice President and Chief Medical Officer, are scheduled to present a corporate overview at the 2015 Leerink Global Healthcare Conference on Thursday, February 12, 2015 at 10:20 am ET at the Waldorf Astoria in New York City.

Cellular Dynamics International, Inc. (Nasdaq:ICEL)
today announced that it has manufactured, under current Good Manufacturing Practices (cGMP), stem cell lines from two HLA "superdonors."

Cellular Biomedicine Group Inc. (Nasdaq:CBMG)
, a biomedicine company engaged in the development of cellular therapies for degenerative and cancerous diseases, today announced its acquisition of Chinese PLA General Hospital's ("PLAGH", Beijing, also known as "301 Hospital") Chimeric Antigen Receptor T cell (CAR-T) therapy, its recombinant expression vector CD19, CD20, CD30 and Human Epidermal Growth Factor Receptor's (EGFR or HER1) Immuno-Oncology patents (all pending), and Phase I/II clinical data of the aforementioned therapies and manufacturing knowledge.

Cesca Therapeutics Inc. (Nasdaq:KOOL)
, an autologous cell-based regenerative medicine company, announced today that the Company will be presenting at the 17th Annual BIO CEO & Investor Conference.

Chembio Diagnostics, Inc. (Nasdaq:CEMI)
, a leader in point-of-care (POC) diagnostic tests for infectious diseases, has received approval for commercial use of its DPP® HIV-Syphilis Assay by the Brazilian regulatory agency, Agência Nacional de Vigilância Sanitária (ANVISA).

The Ensign Group, Inc. (Nasdaq:ENSG)
, the parent company of the Ensign™ group of skilled nursing, rehabilitative care services, home health, home care, hospice care, assisted living and urgent care companies, today reported operating results for the fourth quarter and full year 2014.

The Ensign Group, Inc. (Nasdaq:ENSG)
, the parent company of the Ensign™ group of skilled nursing, rehabilitative care services, assisted living, home health, home care, hospice care and urgent care companies, announced today that it has commenced an underwritten public offering of 2,500,000 shares of its common stock.

Galena Biopharma, Inc. (Nasdaq:GALE)
, a biopharmaceutical company developing and commercializing innovative, targeted oncology treatments that address major medical needs across the full spectrum of cancer care, today announced enrollment of the 700th patient in the NeuVax™ (nelipepimut-S) Phase 3 PRESENT (Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer with Low to Intermediate HER2 Expression with NeuVax Treatment) clinical trial. 

Based upon numerous Hemispherx stockholder requests, Hemispherx Biopharma, Inc. (NYSE MKT:HEB) has posted the report and findings of the recent efficacy study of Ampligen® in a mouse model of Ebola virus infection performed by scientists at the U.S. Army Medical Research Institute of Infectious Disease (USAMRIID).

ICU Medical, Inc., (Nasdaq:ICUI)
, a leader in innovative medical devices used in vascular therapy, oncology and critical care applications, today announced financial results for the fourth quarter and fiscal year ended December 31, 2014.

Islet Sciences, Inc. (OTCQB:ISLT)
, a biopharmaceutical company developing new medicines and technologies for the treatment of metabolic disease, announced today the engagement of Summer Street Research Partners ("Summer Street"), a leading healthcare-focused investment bank, as financial advisor.

Integra LifeSciences Holdings Corporation (Nasdaq:IART)
today announced that it will present at the following conference in February:  At 11:30AM ET on Wednesday, February 25, 2015, Mr. Peter Arduini, Integra's President and Chief Executive Officer, will present at the RBC Capital Markets' Global Healthcare Conference in New York City.

Juno Therapeutics, Inc. (Nasdaq:JUNO)
today announced that it has entered into a lease agreement for a facility in Bothell, Washington to manufacture the company's cell therapy products.

La Jolla Pharmaceutical Company (Nasdaq: LJPC)
, a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, today announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assesment (SPA) for its Phase 3 clinical trial of LJPC-501 for the treatment of catecholamine-resistant hypotension (CRH).

LifeVantage Corporation (Nasdaq:LFVN)
, announced that its flagship product, Protandim, has earned certification from NSF International.

Medical Marijuana, Inc.'s (OTC Pink:MJNA)
Kannaway™, is rapidly ramping up its mission to bring hemp back to consumers across the nation. Today, Kannaway™ announced plans to open three new regional offices in Miami, Orange County, and Atlanta before the end of May 2015.

Melinta Therapeutics announced today that it has entered into a $30 million growth capital debt financing agreement with Hercules Technology Growth Capital, Inc. (NYSE:HTGC).

Nanosphere, Inc. (Nasdaq:NSPH)
, a company enhancing medicine through targeted molecular diagnostics, today announced that it will report its results for the fourth quarter and fiscal year ended December 31, 2014 after the market close on Wednesday, February 11, 2015.

Orexigen (NASDAQ: OREX)
today announced an update on the European marketing authorization application (MAA) for Mysimba™ (naltrexone HCl / bupropion HCl prolonged release) which is being reviewed for use as an adjunct to a reduced-calorie diet and increased physical activity, for the management of weight in adult patients (≥18 years) with an initial Body Mass Index (BMI) of ≥ 30 kg/m2 (obese), or ≥ 27 kg/m2 to ˂ 30 kg/m2 (overweight) in the presence of one or more weight-related co-morbidities (e.g., type 2 diabetes, dyslipidaemia, or controlled hypertension).

Portola Pharmaceuticals (Nasdaq:PTLA)
today announced the appointment of Laura Brege and Dennis Fenton, Ph.D., to its board of directors.

PRA Health Sciences, Inc. (Nasdaq:PRAH)
today announced the appointment of Matthew P. Young to the Board of Directors of the Company.

Receptos, Inc. (Nasdaq:RCPT)
, a biopharmaceutical company developing therapeutic candidates for the treatment of immune and metabolic diseases, today announced that Faheem Hasnain, its President and Chief Executive Officer, will be presenting at the Leerink Global Healthcare Conference at the Waldorf Astoria hotel in New York City.

RedHill Biopharma Ltd. (Nasdaq:RDHL) (TASE:RDHL)
, an Israeli biopharmaceutical company primarily focused on late clinical-stage, proprietary, orally-administered drugs for inflammatory and gastrointestinal diseases, including gastrointestinal cancers, today announced that it intends to offer its American Depository Shares ("ADSs"), each representing ten of its ordinary shares, in an underwritten public offering.

Regulus Therapeutics Inc.  (Nasdaq:RGLS)
, a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, will visit the Nasdaq MarketSite in Times Square.

Rexahn Pharmaceuticals, Inc. (NYSE MKT:RNN)
, a clinical stage biopharmaceutical company developing best-in-class therapeutics for the treatment of cancer, today announced the release of an online publication describing preclinical results for Supinoxin™ (RX-5902) in the peer reviewed medical journal, Journal of Cellular Biochemistry, in an article titled, "A Novel Anti-Cancer Agent, 1-(3,5-Dimethoxyphenyl)-4-[(6-fluoro-2-methoxyquinoxalin-3-yl)aminocarbonyl] piperazine (RX-5902), Interferes with β-Catenin Function through Y593 phospho-p68 RNA Helicase."

Sabra Health Care REIT, Inc. (Nasdaq:SBRA)
announced today that it will issue its 2014 fourth quarter earnings release after close of market on February 18, 2015.

today announced the expansion of its UPS Temperature True® Packaging service, which further broadens the scope of its cold-chain shipping offerings.

VBL Therapeutics (Nasdaq:VBLT)
, a clinical-stage biotechnology company committed to the discovery, development and commercialization of first-in-class treatments for cancer and immune-inflammatory disease, today announced that Eyal Breitbart, Ph.D., Vice President of Research and Operations of VBL, will present preclinical results evaluating the Company's lead Lecinoxoid candidate, VB-201, for the treatment of non-alcoholic steatohepatitis (NASH) and liver fibrosis, in an oral presentation at the Keystone Symposium on Liver Metabolism and Nonalcoholic Fatty Liver Disease (NAFLD), taking place on March 22 - 27, 2015 in Whistler, British Columbia, Canada.

Wright Medical Group, Inc. (Nasdaq:WMGI)
today announced its intention to commence an offering, subject to market and other conditions, of $400 million aggregate principal amount of cash convertible senior notes due 2020 (the "notes"), to be offered and sold to initial purchasers who would resell the notes to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended.

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