|Threshold Pharma Receives Two U.S. Patents for Tarloxotinib Bromide; Portola Announces FDA Grants Fast Track Designation to Betrixaban|
|By Josh Gee|
|Tuesday, 13 October 2015 19:28|
Below is a look at some of the headlines for companies that made news in the healthcare sector on October 13, 2015.
Threshold Pharmaceuticals, Inc. (NASDAQ: THLD) announced the U.S. Patent and Trademark Office (USPTO) has issued the first two U.S. patents protecting tarloxotinib bromide*, or "tarloxotinib," the Company's proprietary hypoxia-activated, irreversible epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor exclusively licensed from the University of Auckland, New Zealand. The first patent, U.S. Patent No. 9,073,916, generically covers a structural class of EGFR tyrosine kinase inhibitor prodrugs, including tarloxotinib, and pharmaceutical compositions containing this structural class of prodrugs. The second patent, U.S. Patent No. 9,101,632, specifically covers the compound tarloxotinib and pharmaceutical compositions containing it.
"These two patents provide fundamental intellectual property protection for tarloxotinib, which is the subject of two ongoing Phase 2 clinical trials for the treatment of patients with mutant EGFR-positive non-small cell lung cancer and patients with squamous cell cancers of the head and neck or skin," said Barry Selick, Ph.D., Chief Executive Officer of Threshold. "Currently, treatment with EGFR inhibitors has limited efficacy because side effects can prevent effective doses from being achieved. We believe that more effective dosing may be achievable with tarloxotinib, which is designed to release an irreversible EGFR tyrosine kinase inhibitor selectively under hypoxic conditions commonly found within solid tumors. Tumor-selective activation of tarloxotinib may allow for greater inhibition of EGFR signaling within the tumor while limiting systemic side effects, potentially leading to better outcomes for patients with EGFR-dependent cancers."
*Tarloxotinib bromide is the proposed International Nonproprietary Name.
Portola Pharmaceuticals (NASDAQ: PTLA) announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to betrixaban for extended-duration prevention of venous thromboembolism (VTE), or blood clots, in acute medically ill patients (i.e., those who are hospitalized for serious medical conditions, such as heart failure, stroke, infection and pulmonary disease). Betrixaban is an investigational oral anticoagulant that directly inhibits the activity of Factor Xa, an important validated target in the blood coagulation pathway, to prevent life-threatening thrombosis.
Portola is currently evaluating betrixaban in the global, pivotal, 7,500-patient Phase 3 APEX (Acute Medically Ill VTE Prevention with Extended Duration Betrixaban) Study for the prevention of VTE in acute medically ill patients. The Company expects to complete patient enrollment in APEX by the end of 2015 and report top-line data in the first quarter of 2016. If the trial is successful, the Company expects to submit a New Drug Application (NDA) later in 2016 under the Fast Track designation.
The FDA's Fast Track process is designed to facilitate the development and expedite the review of drugs used to treat serious conditions and fill an unmet medical need. Fast Track designation enables a company to have early and frequent communication with the FDA throughout the drug development and review process, often leading to earlier drug approval.
"FDA's Fast Track designation of betrixaban recognizes the unmet medical need in the prevention of VTE in acute medically ill patients. Importantly, it raises public awareness of this life-threatening condition in a large, underserved patient population. Betrixaban has the potential to become the first anticoagulant approved for extended-duration prevention of VTE," said John T. Curnutte, M.D., Ph.D., executive vice president, research and development, for Portola. "We're now focused on completing the APEX trial and reporting results in early 2016."
BioCorRx, Inc. (OTCQB:BICX), developer of the BioCorRx® Recovery Program, a Medication-Assisted Treatment (MAT) program, announces a new distribution agreement with an existing wellness center in the Atlanta area. The agreement is part of the Company’s continued focus to distribute its addiction treatment program specific to long-lasting Naltrexone therapy to wellness centers nationally through a partnership with Myriad Medical Marketing (MMM). The new center, Superior Health Care LLC, is run by Dr. Steve Peyroux and is located at 2050 Cumming Way, #100 Canton, GA 30115. “The availability of our newly rebranded program, the BioCorRx® Recovery Program, in this new clinical setting in the heavily populated Southeast is great news for those suffering from addiction in that area of the country. These individuals will soon have access to the benefits of our program in an environment that offers many other services that can be beneficial to them during their recovery,” says BioCorRx COO & interim CEO, Brady Granier.
BioNano Genomics, the leader in physical genome mapping, today announced that the Company has entered into a research collaboration agreement with the University of California, Los Angeles (UCLA). The research is intended to study the role of structural variations in human genetic pediatric disorders using BioNano Genomics' revolutionary genome mapping platform, the Irys® System. Principal investigator of this collaboration will be Dr. Eric Vilain, M.D., Ph.D., of the David Geffen School of Medicine at UCLA. Previously, Dr. Vilain led a study that investigated 814 cases with presumed genetic disorders that have remained undiagnosed despite exhaustive testing efforts. This study mainly focused on sequencing the protein coding regions of a patient's genome to uncover any genetic influences on disease. The results obtained from the study indicated that only 26% of the patient population showed molecular diagnosis. Technologies including next-generation sequencing (NGS) were unsuccessful in identifying the cause of genetic disorders in the remaining 74% of the study patient population. This research collaboration will focus on studying the role of structural variations in the undiagnosed patient population using next-generation mapping (NGM) capabilities of the Irys System. Dr. Vilain and his team will utilize two Irys Systems to conduct research on samples obtained from approximately 80 undiagnosed patients, with a predominantly pediatric population.
Caladrius Biosciences, Inc. (NASDAQ:CLBS), a company combining a leading cell therapy service provider with a therapeutics pipeline including a Phase 3 clinical program in immuno-oncology and a portfolio of earlier clinical phase projects in immune modulation and ischemic repair, announces the promotions of Joseph Talamo from Vice President, Corporate Controller and Chief Accounting Officer to Chief Financial Officer and Todd Girolamo, Esq. from Vice President, Legal to General Counsel. In addition, the Company announces that Robert S. Vaters, is no longer serving as an employee of the Company and has resigned from the Company’s board of directors. “Bob Vaters joined Caladrius in January 2015 to help me accelerate the Company’s near-term organizational and strategic evolution. He has been instrumental in identifying and affecting multiple changes that have contributed to the Company’s new course. We greatly appreciate the contributions he made and wish him continued success in his future endeavors,” said David J. Mazzo, Ph.D., Chief Executive Officer of Caladrius Biosciences. “We are delighted to promote Joe and Todd, both experienced executives who have demonstrated their leadership, competence and diligence in the execution of their duties. I am confident each will continue to contribute meaningfully to the Company’s success as we expand our leadership position as the premier cell therapy service provider and advance our pipeline of clinical development programs,” added Dr. Mazzo.
CymaBay Therapeutics, Inc. (NASDAQ:CBAY), a clinical-stage biopharmaceutical company developing therapies to treat metabolic diseases with high unmet medical need, today announced that Robert J. Wills, Ph.D., a member of the Board of Directors since March 2015, has been appointed Chairman of the Board of Directors. Dr. Wills replaces retiring Chairman, Louis G. Lange, M.D., Ph.D. Dr. Lange has been a member of CymaBay's Board since 2003 and has served as Chairman since 2009. "I would like to thank Dr. Lange for his 12 years of valued leadership and service to CymaBay," said Harold Van Wart, Ph.D., Chief Executive Officer of CymaBay Therapeutics. "On behalf of the Board of Directors, we wish him well in his future endeavors. It is also my pleasure to welcome Dr. Wills as our new Chairman of the Board. Dr. Wills has had a distinguished career in the pharmaceutical industry and has already been a significant strategic asset to CymaBay since he joined our Board in March of this year. We look forward to his continued contributions to CymaBay as we remain focused on further advancing our clinical pipeline, including arfhalofenate in gout and MBX-8025 in high unmet need and orphan diseases."
Fibrocell Science, Inc., (NASDAQ:FCSC), an autologous cell and gene therapy company focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet needs, and Intrexon Corporation (NYSE:XON), a leader in synthetic biology, today announced that a poster entitled “Pre-Clinical Development of a Genetically-Modified Human Dermal Fibroblast (FCX-007) for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)” will be presented at the American Society of Human Genetics Annual Meeting from October 6-10, 2015 in Baltimore, Maryland. The poster contains additional details of the previously reported positive in vivo pre-clinical data for FCX-007, Fibrocell’s orphan gene-therapy product candidate for the treatment of RDEB. RDEB is caused by a mutation of the COL7A1 gene—the gene which encodes for type VII collagen (COL7), a protein that forms anchoring fibrils to hold together the layers of skin. Without these fibrils, skin layers separate causing severe blistering, open wounds and scarring in response to any kind of friction, including normal daily activities like rubbing or scratching.
Heat Biologics, Inc. (Nasdaq:HTBX), a clinical-stage cancer immunotherapy company, announced that it has completed enrollment of the full 75 patients in the blinded, randomized, placebo-controlled arms of the Phase 2 clinical trial of HS-410 (vesigenurtacel-L) for the treatment of high-risk, non-muscle invasive bladder cancer (NMIBC). In these three arms of the Phase 2 trial, Heat is evaluating the ability of HS-410 in combination with standard of care, Bacillus Calmette-Guerin (BCG), to stimulate the immune system and eliminate remaining cancer cells to prevent recurrence. The primary endpoint for the Phase 2 trial is one-year disease free survival. As previously announced, Heat is enrolling an additional 25 patients to evaluate HS-410 as a monotherapy in an unblinded, open-label arm, which the company expects to complete by late 2015/early 2016. "This enrollment of the 75 randomized patients represents a significant milestone for the company and we continue to remain on track to report topline efficacy, immune-response and safety results in the fourth quarter of 2016," said Melissa Price, Ph.D., Vice President of Product Development, Heat Biologics. "These data will help guide the trial design, including patient selection and biomarker strategy, for our registration-directed trial as we move forward in our commitment to address the unmet needs of patients living with bladder cancer." HS-410 is an investigational product candidate for NMIBC based on Heat's proprietary ImPACT™ immunotherapy platform that is designed to generate killer T cells to attack cancers.i In March 2015, Heat received U.S. FDA Fast Track Designation for HS-410 for the treatment of NMIBC, which is designed to facilitate the development and expedite the review of potential therapies. Furthermore, Heat expects to report one-year safety and immune-response data from its Phase 1 trial evaluating HS-410 in patients with NMIBC by the end of 2015.
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 plans to release third quarter of fiscal year 2015 financial results after market close on November 3rd. Management will host a conference call at 5:00 p.m. Eastern Time on November 3rd to discuss the results of the quarter and to host a question and answer session. Those who would like to participate may dial 888-430-8705 (719-325-2402 for international callers) and provide access code 376216 approximately 10 minutes prior to the start of the call. A live webcast of the call will also be provided on the investor relations section of the Company's website at http://Investors.K2M.com/. For those unable to participate, a replay of the call will be available for two weeks at 888-203-1112 (719-457-0820 for international callers); access code 376216. The webcast will be archived on the investor relations section of the Company's website.
LDR Holding Corporation (NASDAQ:LDRH), a global medical device company focused on designing and commercializing novel and proprietary surgical technologies for the treatment of patients suffering from spine disorders, today announced preliminary estimated revenue results for the third quarter ended September 30, 2015. For the third quarter ended September 30, 2015, the company expects to report: Total revenue of approximately $39.3 million, an increase of 9.5% over the third quarter of 2014, or an increase of 14.2% on a constant currency basis; Revenue from exclusive technology products of approximately $36.7 million, an increase of 14.5% over the third quarter of 2014, or an increase of 18.0% on a constant currency basis; Revenue in the United States of approximately $32.3 million, an increase of 15.0% over the third quarter of 2014, and representing 82.2% of total revenue; International revenue of approximately $7.0 million, a decrease of 10.3% over the third quarter of 2014. On a constant currency basis, international revenue increased 11.4%. Revenue from the Company’s exclusive cervical products is expected to be approximately $27.5 million, an increase of 21.0% over the third quarter of 2014, or an increase of 25.2% on a constant currency basis, due principally to the growth from sales of the Mobi-C® Cervical Disc. Additionally, revenue from LDR's exclusive lumbar products is expected to be approximately $9.2 million, a decrease of 1.5% over the third quarter of 2014, or an increase of 0.5% on a constant currency basis.
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, announced today that the U.S. Environmental Protection Agency has approved its latest product, MAJESTENE™, a broad spectrum, high performance natural bionematicide for controlling nematodes (roundworms) on a wide range of agricultural crops. MAJESTENE was developed from MBI's in-house discovery screening process and provides growers with a new mode of action for safely controlling nematodes by reducing or stopping eggs from hatching, preventing root galling and reducing nematode population density. Nematodes cause approximately $80 billion annually in damages to crops globally. "We are pleased with MAJESTENE'S approval by the EPA, as this new bionematicide meets a large market need due to heavy restrictions on toxic chemicals used to control nematodes and the need for easier-to-use biologicals that reduce multiple species of nematode populations," said Pam Marrone, MBI's CEO. "Today's growers are faced with ever-increasing regulatory restrictions on existing conventional products to reduce exposure to people nearby, to protect non-target beneficial insects, to reduce air and water pollution, to ensure worker safety and to be export-ready. Growers who traditionally employ conventional nematicides for crop protection can now use MAJESTENE, which has multiple modes of action, a low risk to beneficial insects, and an exemption from residue tolerances. MAJESTENE brings growers expanded options for an environmentally responsible and effective solution for managing pests in conventional and organic crops," said Brian Ahrens, MBI's Vice President of Sales.
MedAssets (NASDAQ:MDAS) today announced that Scripps Health, a non-profit regional integrated health system based in San Diego, Calif., has expanded its relationship with MedAssets to include Invoice Management Services, powered by Ariba, an SAP company. This is MedAssets newest component of its procure-to-pay solution set, and builds on Scripps Health's comprehensive program with MedAssets to reduce the total cost of care, enhance operational efficiency, align clinical delivery and improve revenue performance. MedAssets Invoice Management Services is a best-in-class solution that leverages Ariba's cloud-based invoice management applications to enable a "smart invoicing" process through which invoices are automatically validated against 80+ provider-customized validation rules to ensure that only accurate and approved invoices reach accounts payable. "Many companies have implemented solutions to digitize invoices. But with one in five invoices still containing an overcharge or other error, it's clear that digitization only leads to inaccurate invoices being delivered faster," said Alex Atzberger, president, Ariba. "MedAssets delivers an intelligent invoicing process using Ariba's Invoice Management solutions that reduces the number of invoice exceptions materially and provides true "touchless" invoice processing, resulting in significant labor efficiency gains to providers."
Medical Marijuana, Inc. (OTC Pink:MJNA), the first-ever publicly traded U.S. cannabis company, is pleased to announce to shareholders and the general public that Stuart W. Titus, PhD and Chief Executive Officer of Medical Marijuana, Inc. will be presenting on the Cannabis Investor Webcast on Thursday, October 8 at 4:00 – 4:45 PM ET (1:00 – 1:45 PM PT). The Cannabis Investor Webcast will include presentations from both privately held and publicly traded industry companies and industry professionals. Medical Marijuana, Inc.’s presentation on hemp industry innovations will be 30 minutes long and will then be followed by a 15-minute Q&A. Along with learning about economic potential of re-introducing hemp into American culture, attendees at the Cannabis Investor can research Medical Marijuana, Inc. without taking time off from work, paying registration fees and incurring travel-related expenses. “Medical Marijuana, Inc. is very excited to share an overview on the company, hemp industry innovations and other recent developments with the Cannabis Investor Webcast,” states Stuart W. Titus, PhD and Chief Executive Officer of Medical Marijuana, Inc. “With our flagship product Real Scientific Hemp Oil generating global headlines and other exciting developments from our portfolio companies that are conducting clinical research, it’s a very exciting time for the company.”
Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, today announced that new clinical outcomes and clinical utility data for myPath® Melanoma will be featured at the American Society of Dermatopathology (ASDP) 52nd meeting being held Oct. 8 to 11, 2015 in San Francisco, Calif. The findings add to the growing body of knowledge for myPath Melanoma and will support the Company's clinical reimbursement dossier for the product. "The accurate diagnosis of melanoma can be challenging based on histologic findings alone and there are potentially severe consequences of a misdiagnosis, including the under-treatment or overtreatment of patients," said Loren Clarke, M.D., vice president, Medical Affairs for Dermatology, Myriad Genetic Laboratories. "Our studies show that myPath Melanoma accurately differentiates malignant melanoma from benign skin lesions and helps physicians deliver a more objective and confident diagnosis for their patients."
SANUWAVE Health, Inc. (OTCQB:SNWV) today announced top line and preliminary data analysis from the Company's pivotal Phase III, Investigational Device Exemption (IDE) supplemental clinical trial comparing the rates of 100% wound closure at 12 weeks between dermaPACE® and Sham control (non-active treatment), when both are combined with the current standard of care for the treatment of diabetic foot ulcers (DFUs). This study supplemented the Company's earlier 206 patient Phase III trial of identical endpoints which demonstrated the exceptional safety profile and clinical benefit of the dermaPACE device for the treatment of diabetic foot ulcers; an area of significant unmet medical need and represents a $2 billion market in the United States alone. The design of this randomized, double-blind, parallel-group, Sham-controlled, multicenter, 26-week clinical trial was intended to quantify the effectiveness of up to eight, non-invasive procedures with dermaPACE, delivered over a 10-week period. This supplemental study ultimately enrolled and randomized 130 subjects and was run under a Bayesian statistical plan, designed with FDA input and utilizing the strength of the results from the first clinical trial as an informative prior.
The Spectranetics Corporation (Nasdaq:SPNC) today announced that the Company will release 2015 third quarter financial results after market close on Thursday, October 22, 2015. Company management will host an investment-community conference call beginning at 2:30 p.m. Mountain Time (4:30 p.m. Eastern Time) on Thursday, October 22 to discuss those results and to answer questions. Individuals interested in listening to the conference call may do so by dialing (877) 561-2747 for domestic callers, or (973) 409-9689 for international callers (Conference ID: 48806203), or from the webcast on the investor relations section of the Company's website at: www.spectranetics.com. The webcast will be available on the Company's website for 14 days following the completion of the call.
VBI Vaccines Inc. (Nasdaq:VBIV) nnounced that it has applied its eVLP Platform in the development of a novel therapeutic vaccine candidate for glioblastoma multiforme ("Glioblastoma" or "GBM"). Columbia University's Brain Tumor Center is performing research to evaluate VBI's GBM immunotherapy candidate in ex vivo studies using GBM patient samples. Glioblastoma is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, 12,000 new cases are diagnosed each year. The current standard of care for GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and is exceptionally lethal, with median patient survival of less than sixteen months. A growing body of research has demonstrated that GBM tumors are particularly susceptible to infection by cytomegalovirus ("CMV"), with over 90% of GBM tumors expressing CMV antigens. In addition, recent research has demonstrated that dendritic cell priming combined with dendritic cell vaccination against CMV can extend overall survival in patients with glioblastoma.
WaferGen Bio-systems, Inc. (Nasdaq:WGBS) today announced preliminary, unaudited revenue for the third quarter of 2015. The Company expects total revenue for the third quarter of 2015 to be in the range of $1.9 million to $2.1 million, which would represent an increase of 52-68% when compared to the $1.3 million reported for the third quarter of 2014. This also represents a sequential increase of 18-30% compared to the $1.6 million generated in the second quarter of 2015. Revenue for the third quarter is expected to be the highest in WaferGen's history, and represents the second consecutive quarter of revenue growth. The primary driver of revenue growth was sales of the Company's SmartChipTM products and services, which comprised approximately 67% of total revenue, compared to 53% in the second quarter of 2015 and 58% in the third quarter of 2014. WaferGen is also updating the Company's full-year 2015 revenue guidance from between $7.5 million and $7.8 million, excluding sales of the recently launched ICELL8™ Single-Cell System, to between $7.8 million and $8.2 million, including ICELL8-related revenue. "The third quarter will represent both our highest ever quarterly revenue, and the Company's second consecutive quarter of revenue growth," said Rollie Carlson, President and CEO of WaferGen. "With our base business now performing well, and the recent commercial launch of our ICELL8 Single-Cell System, we are well-positioned for meaningful growth in our business."