|BrainStorm Announces Treatment of Final Patient in US Multicenter Double Blinded Phase 2 ALS Trial; Nektar Submits Investigational New Drug Application (IND) for NKTR-214|
|By Marilyn Mullen|
|Wednesday, 07 October 2015 18:43|
Below is a look at some of the headlines for companies that made news in the healthcare sector on October 7, 2015.
BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), announced the final patient has been treated in its multicenter Phase 2 clinical trial investigating the company's proprietary NurOwn™ stem cell technology. The trial is a double blind, placebo-controlled, clinical trial which targeted enrollment of 48 patients with Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease.
BrainStorm Cell Therapeutics -- The study was designed to evaluate the safety and efficacy of transplantation of Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors ("MSC-NTF" or NurOwn™). The trial is being conducted at the Massachusetts General Hospital (MGH) in Boston, MA, University of Massachusetts Memorial (UMass) Hospital in Worcester, MA, and Mayo Clinic in Rochester, Minnesota.
"We are pleased to have successfully completed all transplantations," said principal investigator, Prof. Anthony J. Windebank, MD, "In six months, we expect to complete follow-up visits on the last patients, and we are looking forward to seeing the analysis of the complete results. The initial safety profile has been excellent and the treatment generally well-tolerated – which is a primary goal of the study."
"We are proud to announce the completion of dosing of this multicenter study on schedule, which is one of the company's most important milestones to date. Importantly, this is the first trial to compare NurOwn with placebo," said Chaim Lebovits, CEO of BrainStorm. "We are extremely thankful for having the opportunity to collaborate with three top-tier US medical centers – MGH, UMass and Mayo Clinic. And we would like to express our special appreciation and thanks to our team of PI's – Prof. Robert H. Brown, MD, DPhil, Chair of the Neurological Department at UMass, Prof. Merit Cudkowicz, MD, MSc, Chair of the MGH Neurological Clinical Research Institute (NCRI), and Prof. Anthony J. Windebank, MD, Head of the Regenerative Neurobiology Laboratory in the Department of Neurology at Mayo Clinic."
Nektar Therapeutics (NASDAQ: NKTR) announced it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for NKTR-214, its lead immuno-oncology candidate. NKTR-214 is a CD122-biased immune-stimulatory cytokine that is designed to stimulate the patient's own immune system to destroy cancer cells. The company plans to initiate a Phase 1/2 clinical study by the end of 2015. The study will evaluate the safety, tolerability and efficacy of NKTR-214 in patients with solid tumor malignancies and will include expansion cohorts that will evaluate NKTR-214 both as a single-agent and in combination with a checkpoint inhibitor.
"As a new cytokine with biased receptor activity and an antibody-like dosing schedule, NKTR-214 could emerge as a differentiated immuno-oncology therapy that specifically stimulates T-cell growth to fight cancer," said Stephen Doberstein, PhD, Senior Vice President and Chief Scientific Officer of Nektar. "In preclinical studies with NKTR-214, we not only observed single-agent efficacy in multiple tumor models, but when administered in combination with a checkpoint inhibitor, we see a dramatic immune-educating vaccine-like effect with NKTR-214. We are excited to start our first-in-human study and we expect to have initial data from the dose-escalation phase of the trial by the second half of 2016."
The Phase 1/2 clinical program will be conducted at multiple clinical sites including MD Anderson Cancer Center and Yale Cancer Center. In addition to the Phase 1/2 clinical program, Nektar and MD Anderson will conduct translational research to identify predictive biomarkers that can be used in the future development of NKTR-214.
Adaptimmune Therapeutics plc (Nasdaq:ADAP), a clinical stage biopharmaceutical company focused on the use of T-cell therapy to treat cancer, will announce financial results for its 2015 full fiscal year ended June 30 before the open of the U.S. markets on Tuesday, October 13, 2015. Following the announcement, the Company will host a live teleconference and webcast at 8:00 a.m. Eastern Time (1:00 p.m. BST) on Tuesday, October 13, 2015, at which time management will provide a business update and discuss the 2015 full fiscal year financial results. The press release and the live webcast of the conference call will be available via Adaptimmune’s corporate website at www.adaptimmune.com. The webcast will be made available on the events page. An archive will be available after the call at the same address. To participate in the live conference call, if preferred, please dial (877) 280-1254 (U.S.) or +44 (0) 20 3427 1919 or 0800 279 4992 (United Kingdom). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (7224348).
Allscripts (Nasdaq:MDRX) announced a solidified management team under an enhanced organizational structure. This new structure will enable more effective client partnerships and better support the Company's long-term growth. Chief Financial Officer Rick Poulton is promoted to President, responsible for day-to-day operational leadership. He will continue to report to Chief Executive Officer Paul Black, who has held the dual position of President and CEO since joining the Company in December 2012. The Company has commenced a search for a new CFO, and Poulton will maintain the CFO title and responsibilities until a new CFO is appointed. In addition, the Allscripts Board of Directors and Paul Black agreed to extend Black's employment agreement through at least 2018. The initial term of his previous contract was set to expire in December 2015.
Applied Genetic Technologies Corporation (Nasdaq:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, today announced that data from a study evaluating the natural history of X-linked retinoschisis (XLRS) will be presented at The Retina Society 48th Annual Scientific Meeting, taking place in Paris, France from October 7-11. "These results provided useful information to inform the design of AGTC's ongoing phase 1/2 clinical trial to evaluate a candidate AAV-based gene therapy product for treating XLRS, a rare inherited retinal disease that can lead to a significant reduction in visual acuity and other serious complications," said Sue Washer, President and CEO of AGTC. "We recently reached an enrollment milestone in our dose escalation study and plan to share initial topline safety data from the first treatment cohorts in the coming months."
Avant Diagnostics, Inc. (OTCQB:AVDX), an innovative in vitro diagnostics company, was notified by DOCRO, its independent clinical research organization, that it has received notification that the previously purchased specimens have been approved and are available for use in the forthcoming validation study to be used to support a pre-Submission package to the United States Food and Drug Administration (“FDA”). Avant expects to commence the FDA validation study shortly after the completion of the ongoing calibration testing. Once the calibration testing is completed, Avant intends to test the previously purchased set of ovarian cancer specimens, including serial sets obtained from women diagnosed previously with ovarian cancer, which will serve as the validation study and form the basis of the pre-Submission package that will be submitted to FDA for review and comment prior to the commencement of the OvaDx® 510(k) trial. The OvaDx® microarray test is intended for use as an aid in monitoring women diagnosed previously with ovarian cancer. The validation study and 510(k) trial will be conducted in a double-blinded environment supervised by DOCRO, Inc., an independent clinical research organization. The results from the validation study are expected to be published in a peer-reviewed scientific journal within six months of test completion and data analysis, however there can be no assurance that such publication will be completed within this time frame.
Chimerix, Inc. (NASDAQ:CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, announced today preliminary data from liver transplant patients who received brincidofovir for adenovirus infection through the ongoing AdVise trial and the brincidofovir expanded access program. These data will be presented at IDWeek in San Diego. Twelve pediatric patients and one adult patient who were infected with adenovirus following receipt of a liver transplant, either alone or as part of a multi-organ transplantation, were identified from the AdVise trial and the brincidofovir expanded access program. Eight out of the thirteen patients had disseminated adenovirus disease, with high levels of adenovirus in the blood and/or adenovirus recovered from multiple organ systems. Patients were treated with brincidofovir for a median of 79 days (range: 4 to 180 days). Patients were assessed for changes in adenovirus viral load from baseline, time to nadir viral load, survival, and adverse events.
Dermira, Inc. (NASDAQ:DERM), a specialty biopharmaceutical company focused on bringing innovative and differentiated products to dermatologists and their patients, today announced the presentation of data for DRM01, a novel, topical sebum inhibitor in development for the treatment of acne. Topline data from the company's Phase 2a study showed that DRM01 met all of the primary efficacy endpoints, demonstrating statistically significant improvements relative to vehicle in the reduction of lesion counts and the proportion of patients who achieved a successful improvement in Investigator's Global Assessment (IGA) score at week 12. In addition, newly presented data suggest that a treatment difference in these endpoints was observed as early as the first efficacy assessment, which was conducted after four weeks of the 12-week treatment period. As previously reported, topical treatment with DRM01 was generally well tolerated. These results were presented today at the European Academy of Dermatology and Venereology (EADV) Congress 2015 being held October 7-11, 2015 in Copenhagen.
Dyadic International, Inc. (OTCQX:DYAI), a global biotechnology company focused on the discovery, development, manufacture and sale of enzymes and other proteins for the biopharmaceutical, bioenergy, bio-based chemicals, food and feed industries, announced today it has received the test results from the mice trials in our research program with Sanofi Pasteur. The data generated by Sanofi Pasteur indicates that the C1 produced antigen generated an equal, or better, immune response in mice than the industry standard antigen. "We are very encouraged by these results and remain optimistic as to the potential benefits our C1 technology can bring to the biopharmaceutical industry. We believe our C1 Expression System has the potential to aid in the discovery, development and manufacture of new, better and more affordable human and animal vaccines, antibodies, biosimilars and other therapeutic proteins," said Dyadic’s founder & CEO, Mark Emalfarb. "Our expansion into biopharmaceutical’s is taking shape. With the promising Sanofi results and our participation in the EU funded Zoonoses Anticipation and Preparedness Initiative or ZAPI, which was launched earlier this year, we expect to further develop our capabilities and establish our C1 Technology as a competitive manufacturing platform for use in producing novel, and low cost vaccines, biosimilars and other biologics."
The Electronic Healthcare Network Accreditation Commission (EHNAC), a non-profit standards development organization and accrediting body for organizations that electronically exchange healthcare data, announced today that it has posted new versions of program criteria for public review. EHNAC seeks interested parties to provide opinions, comments and suggestions for the 16 upgraded 2016 criteria versions that have been developed for each of EHNAC’s accreditation programs, as well as the two new accreditation programs available. The open process for adopting criteria will last 60 days, commencing on Oct. 7, 2015 and ending on Dec. 4, 2015. Feedback is encouraged with regard to the necessity, appropriateness and workability of the criteria versions proposed for adoption.
Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing novel protein therapeutics for cancer and inflammatory diseases, today announced that preliminary data from rheumatoid arthritis (RA) patients in part 3 of Five Prime's ongoing Phase 1 trial of FPA008 will be presented during the upcoming 2015 American College of Rheumatology (ACR)/Association of Rheumatology Health Professionals Annual Meeting, to be held November 7-11, 2015 in San Francisco. FPA008 is Five Prime's anti-CSF1R antibody that blocks the binding of CSF1 and IL34 ligands to CSF1R, resulting in inhibition of the activation and survival of inflammatory macrophages and osteoclasts. Abstract #2749 entitled, "A Phase 1 Study of FPA008, an Anti-Colony Stimulating Factor 1 Receptor (anti-CSF1R) Antibody in Patients (pts) with Rheumatoid Arthritis (RA): Preliminary Results," is accessible on the meeting website and includes data as of May 13, 2015. The poster presentation will take place during the session, Rheumatoid Arthritis - Small Molecules, Biologics and Gene Therapy Poster III, scheduled from 9:00-11:00 am Pacific Time on Tuesday, November 10, 2015. The poster is expected to include study data up to October 2015, which will be detailed in a press release during the conference.
FutureWorld Corp. (FWDG), a cannabis technology accelerator and a leading provider of advanced technologies and solutions to the global cannabis industry, announced today that Cameron Cox, former executive VP President of FutureWorld Corp. and CEO of FutureLand Corp. (FUTL) along with officers from some of the subsidiaries of FutureWorld will be presenting on the Cannabis Investor Webcast on Thursday, October 8, 2015 at 10:00 - 10:45 AM ET (08:00 - 08:45 AM MT). The Cannabis Investor Webcast will include presentations from privately-held and publicly-traded industry companies and industry professionals. FutureWorld Corp. presentation will be 30-minutes long and followed by 15-minutes of Q&A. The Cannabis Investor Webcast is a great opportunity for the audience to research FutureWorld Corp. without taking time-off from work, paying registration fees and incurring travel-related expenses.
Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin proteins to treat fibrosis and cancer, today announced that James Czirr, the Company's Executive Chairman will present at the Robins Equity Research Roundup Conference taking place October 8-10, 2015. Mr. Czirr's presentation, entitled Developing A Therapy for a Silent Epidemic - What Galectin Therapeutics is doing to Develop Treatments for NASH with Liver Cirrhosis and Other Unmet Medical Needs is scheduled for Friday, October 9 at 11:15 a.m. Pacific time. The Robins Equity Research Roundup is presented by Catalyst Research Management Group. An audio replay of Mr. Czirr's presentation will be posted to the Investor Relations section of the company's website the following week. The conference is being held at Skamania Lodge, in the Cascade Range Gorge above the Columbia River. The Robins Equity Research Roundup is a small-cap investor conference focused on introducing new and interesting company equities and their managements to clients and potential shareholders. The audience will include investors and brokers from the region as well as professional money managers from different parts of the country.
Heat Biologics, Inc. (Nasdaq:HTBX), a clinical-stage cancer immunotherapy company, announced Taylor Schreiber, M.D., Ph.D., Chief Scientific Officer, will serve on a panel at the 22nd Annual Prostate Cancer Foundation (PCF) Scientific Retreat in Washington, D.C. on October 7-10, 2015. In addition, Dr. Schreiber will present an overview on ComPACT, the company's next-generation combination immunotherapy platform. ComPACT combines a pan-antigen T cell priming vaccine and T cell co-stimulator in a single product, offering the potential benefits of combination immunotherapy in a single drug without the need for multiple independent biologic products. Heat expects to announce its selection of the first product candidate based on the ComPACT platform in the first quarter of 2016. For the past twenty-one years, the PCF has been hosting an annual scientific retreat that brings together diverse researchers in a collaborative forum to present and discuss new and largely unpublished findings for prostate cancer diagnosis, prognosis, and treatment and defines the challenges that impede progress toward better treatments and cures. For more information please visit PCF's website at www.pcf.org.
Kite Pharma, Inc. (Nasdaq:KITE) today announced that Steven A. Rosenberg, M.D., Ph.D., Chief of Surgery at the National Cancer Institute (NCI) and a special advisor to Kite, has received three significant awards for his achievements and career dedicated to advancing cancer research. The American Cancer Society (ACS), the largest voluntary health organization in the United States, awarded Dr. Rosenberg its Medal of Honor for his pioneering leadership in cancer immunotherapy. The Medal of Honor is the ACS' highest honor and was presented to Dr. Rosenberg at a ceremony held in Washington, DC, on September 30, 2015.
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. As consideration for the license, Lion will make an upfront payment to the NIH, payable half within 60 days of closing and the balance a year later. Additional milestone payments, which will vary according to indication, will be based on completion of specific clinical, regulatory and commercial milestones. The agreement also calls for royalties to be payable to the NIH based on revenues, and certain additional payments under different sublicense scenarios.
Myriad Genetics, Inc. (NASDAQ:MYGN) today announced that it has signed a three-year contract with Tufts Health Plan through which the plan will provide coverage of Prolaris® for members diagnosed with localized prostate cancer. Prolaris is the first and only biopsy test validated against prostate cancer specific mortality. Tufts Health Plan is a leading health plan in the Northeast with more than one million members. Tufts Health Plan's members will benefit from this agreement, which is designed to drive optimal treatment because men can confidently pursue active surveillance for tumors identified as being less aggressive with Myriad's extensively validated Prolaris combined score. Recent data show that many men realize no benefit from interventional treatment, and many suffer needlessly from the side effects associated with radical prostatectomy and radiation. "Myriad is excited to offer Prolaris testing to Tufts Health Plan's members. We believe it will help to improve patient care and lower healthcare costs," said Nicole Lambert, general manager, Urology Business Unit, Myriad Genetic Laboratories. "The Prolaris test has been validated in multiple clinical studies and provides substantially stronger prognostic power than standard pathology. The Prolaris test enables physicians to confidently tailor treatment plans for their patients."
Natus Medical Incorporated (NASDAQ:BABY) today announced that the Company will release its 2015 third quarter financial results before the market opens on Wednesday, October 21st. Natus management will host an investment-community conference call the same day beginning at 8:00 a.m. Pacific Time (11:00 a.m. Eastern Time) to discuss those results and to answer questions. Individuals interested in listening to the conference call may do so by dialing 1-800-510-0146 for domestic callers, or 1-617-614-3449 for international callers, and entering reservation code 80435628. A telephone replay will be available for 48 hours following the conclusion of the call by dialing 1-888-286-8010 for domestic callers, or 1-617-801-6888 for international callers, and entering reservation code 23210688. The conference call also will be available real-time via the Internet at http://investor.natus.com and a recording of the call will be available on the Company's Web site for 90 days following the completion of the call.
Otonomy, Inc. (Nasdaq:OTIC), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapeutics for diseases and disorders of the ear, today held an investor and analyst day which included a review of the commercialization plans for OTIPRIO™ (formerly known as AuriPro™) in patients undergoing ear tube surgery, the Phase 3 clinical trial program for OTO-104 in Ménière's disease, an introduction to the OTO-311 program for tinnitus that has been cleared for Phase 1 testing, other pipeline activities as well as a general corporate update. A replay of the event webcast is available online at www.Otonomy.com in the events and presentations section under the investors tab.
Drug discovery and development company SCYNEXIS, Inc. (Nasdaq:SCYX) today announced the appointment of Rajeshwar Motheram, Ph.D., as Vice President, Pharmaceutical Development, effective October 1, 2015. Dr. Motheram will be responsible for pharmaceutical research and development, including chemistry, manufacturing and controls (CMC) and regulatory CMC strategy for SCYNEXIS' lead clinical asset, SCY-078, in Phase 2 development for the treatment of invasive fungal infections. Dr. Motheram will report to David Angulo, M.D., SCYNEXIS' Chief Medical Officer. "Rajeshwar brings a wealth of experience in drug development and manufacturing to SCYNEXIS as we continue to advance the clinical development of SCY-078 for life threatening and treatment-resistant fungal infections," said Marco Taglietti, M.D., SCYNEXIS' President and Chief Executive Officer. "His background serves as a complement to the skills of our management team as we continue to build the company and plan our growth strategies." "SCY-078 has the potential to become a compelling new treatment for patients and physicians in need of improved antifungal alternatives," said Dr. Motheram. "I'm thrilled to be joining David and the broader team at SCYNEXIS and advancing this important clinical candidate, as well as guiding the pharmaceutical development strategy going forward."
Sunesis Pharmaceuticals, Inc. (Nasdaq:SNSS) today announced that the company intends to submit a Marketing Authorization Application (MAA) for vosaroxin as a treatment for acute myeloid leukemia (AML) with the European Medicines Agency (EMA) by the end of 2015. The company recently announced that it met separately with the Rapporteur (United Kingdom) and Co-Rapporteur (Netherlands) assigned to provide advice and guidance to the company through the MAA process. Based on these discussions, the company is proceeding with an MAA filing for the indication of relapsed/refractory AML in patients age 60 years and older, a population with the greatest medical need and for whom the greatest benefit was observed in the vosaroxin/cytarabine treatment arm of VALOR, the company's pivotal Phase 3 study of vosaroxin and cytarabine in adult patients with relapsed or refractory AML.