|First Human Patient Treated in Phase 1b Study of Advaxis's ADXS-HER2; RXi Pharma Granted Patent in China for Lead Clinical Candidate RXI-109|
|By David Fowler|
|Monday, 28 September 2015 19:19|
Below is a look at some of the headlines for companies that made news in the healthcare sector on September 28, 2015.
The Phase 1b clinical trial is the first-in-human study of Advaxis's lead Lm Technology™ immunotherapy product for HER2 expressing cancers. The dose escalation portion of the study will investigate the safety and tolerability of ADXS-HER2 as a monotherapy in approximately 18 patients diagnosed with metastatic HER2 expressing solid tumors, which include breast, gastric, esophageal and osteosarcoma. Once the maximum tolerated dose (MTD) and recommended Phase 2 dose have been identified, up to 80 patients may be enrolled in up to four HER2 expressing tumor specific cohorts in the expansion phase of the study.
"We are pleased to enroll our first patient in the dose escalation portion of this Phase 1b study of ADXS-HER2 in HER2 expressing solid tumors," said Daniel J. O'Connor, President and Chief Executive Officer of Advaxis. "With the initiation of this study, we now have three clinical constructs in development and seven ongoing clinical trials, which reinforce our long-term commitment to evaluate the potential of our immunotherapy platform across different tumor types."
Advaxis plans to establish the MTD from the Phase 1b study in pediatric patients and work with Children's Oncology Group (COG) to potentially launch a pivotal trial in pediatric osteosarcoma in 2016. The COG, a National Cancer Institute supported clinical trials group, is the world's largest organization devoted exclusively to childhood and adolescent cancer research.
This Phase 1b study builds upon efficacy and safety data from Phase 1 clinical studies of ADXS-HER2 conducted in dogs with osteosarcoma, which may have important translational relevance for human patients with osteosarcoma and other HER2 expressing cancers. Preliminary data from a Phase 1 clinical trial in canine osteosarcoma presented at the 2014 American College of Veterinary Internal Medicine (ACVIM) Forum suggested ADXS-HER2 safely delayed or prevented the development of metastatic disease and prolonged overall survival in pet dogs with osteosarcoma when administered after amputation and chemotherapy. Results from this trial led to ADXS-HER2 being considered by the U.S. Department of Agriculture (USDA) for expedited approval to treat canine osteosarcoma. Furthermore, preliminary data from a second ongoing canine Phase 1/2 trial presented at the 2015 American Association for Cancer Research (AACR) Annual Meeting suggested that ADXS-HER2 in combination with palliative radiation delayed tumor progression and prolonged overall survival in pet dogs with spontaneous osteosarcoma that are not candidates for primary tumor removal (amputation).
"The Phase 1 safety and efficacy data with ADXS-HER2 in canine osteosarcoma supports our clinical development of ADXS-HER2 in HER2 expressing solid tumors," said David J. Mauro, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Advaxis. "We look forward to translating this canine evidence into the first human trial of ADXS-HER2 in metastatic HER2 expressing solid tumors."
RXi Pharmaceuticals Corporation (NASDAQ: RXII), a biotechnology company focused on discovering and developing innovative therapies primarily in the areas of dermatology and ophthalmology, today announced that it has been granted a patent from the State Intellectual Property Office of the People's Republic of China (SIPO) for the composition and methods of use for RXI-109 and other connective tissue growth factor (CTGF) targeting self-delivering RNAi compounds (sd-rxRNA®) for the treatment of fibrotic disorders, including skin fibrosis. The patent is scheduled to expire in 2031.
RXI-109 and other CTGF-targeting sd-rxRNA® compounds may be beneficial for the treatment of fibrotic diseases, including dermal scarring, a condition with a much higher prevalence in people with darker skin. Skin pigmentation is classified in VI categories known as the Fitzpatrick scale. In China, individuals tend to have skin types between III and V. The prevalence rates for hypertrophic scarring in individuals with higher-scoring Fitzpatrick phototypes can be up to 40-70% following surgery, making China a significant market for RXI-109 in the treatment of dermal scarring. According to Persistence Market Research, China is predicted to be one of the fastest growing markets for scar treatments in Asia.
"We are pleased with the issuance of this core patent which covers RXI-109 in China," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He added, "Not only does this patent further strengthen our intellectual property portfolio, it also expands regional out-licensing and partnering opportunities available with our self-delivering platform. This comes at an important time as we accelerate our business development efforts, one of our seven key initiatives for increasing shareholder value."
ACETO Corporation (NASDAQ:ACET), a global leader in the marketing, sale and distribution of products for Human Health, Pharmaceutical Ingredients and Performance Chemicals, today announced that Rising Pharmaceuticals, Inc., its generic finished dosage form subsidiary, has reached agreement to purchase three ANDAs from a subsidiary of Endo International plc for the products Methimazole Tablets, Glycopyrrolate Tablets and Meclizine Tablets. Methimazole, the generic version of Pfizer's Tapazole®, is indicated for the treatment of hyperthyroidism and has IMS-reported generic sales of $12.3 million. Glycopyrrolate, the generic version of Shionogi's Robinul® and Robinul® Forte, which are indicated for the treatment of peptic ulcers have IMS-reported generic sales of approximately $20 million. Meclizine, the generic version of Citron's Antivert®, is indicated for the treatment or prevention of nausea, vomiting and dizziness caused by motion sickness, and has IMS-reported generic sales of $38 million.
Adaptimmune Therapeutics plc (Nasdaq:ADAP), a clinical stage biopharmaceutical company focused on the use of T-cell therapy to treat cancer, today announced that Adrian Rawcliffe, Chief Financial Officer of Adaptimmune, will present at the Leerink Partners Inaugural Immuno-Oncology Roundtable at 2:20 PM ET (7:20 PM BST) on Thursday, October 1, 2015. The conference is being held at the Le Parker Meridien Hotel in New York City. Adaptimmune's presentation will be webcast live for investors through the investor section of www.adaptimmune.com and available for a period of 30 days following the conference.
ADMA Biologics, Inc. (Nasdaq:ADMA), a late-stage biopharmaceutical company that develops, manufactures, and intends to commercialize specialty plasma-based biologics for the treatment and prevention of certain infectious diseases, announced today that the Company has been recognized as one of the 50 fastest growing companies in New Jersey by NJBIZ, a leading New Jersey-focused weekly business new publication. The award program produced by NJBIZ, New Jersey's premiere business news publication, is presented by Weiser Mazars and Comcast Business and sponsored by United Healthcare, and Archer & Greiner. "ADMA Biologics is honored to be recognized as one of the fastest growing companies in New Jersey, and we extend our congratulations to the other honorees," stated Adam Grossman, President and CEO of ADMA Biologics. "We are proud of the rapid evolution of our Company, and this distinction is a tremendous honor as we look towards the future. At ADMA Biologics, our mission is to offer treatment options to immune-compromised patients, and we remain committed to fulfilling that mission."
Aethlon Medical, Inc. (Nasdaq: AEMD), today announced that it has entered into an extension of its contract with the Defense Advanced Research Projects Agency, or DARPA, part of the Department of Defense. The Company originally entered into the initial DARPA contract on September 30, 2011. DARPA entered into this contract extension in order to exercise its option to continue the contract for year five, the final year of the contract. Under the DARPA contract, the company has been engaged to develop a therapeutic device to reduce the incidence of sepsis, a fatal bloodstream infection that often results in the death of combat-injured soldiers. The award from the Defense Advanced Research Projects Agency is a fixed-price contract that requires the achievement of multiple, incremental milestones to receive the full award during each year of the contract. The Company has achieved 23 milestones under the contract, which have resulted in revenues of approximately $4,872,000.
Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing new therapeutics and molecular diagnostics that target the underlying mechanisms of cancer, today announced that Mr. Avanish Vellanki, Senior Vice President and Chief Business Officer, will present at the 15th Annual Biotech in Europe Forum for Global Partnering and Investment on Tuesday September 29th at 8:30 a.m. ET / 2:30 p.m. CEST at the Congress Centre Basel, Switzerland in the Darwin presentation room. The presentation slides will be accessible by visiting the following link on the Aptose Biosciences Inc. website: http://ir.aptose.com/phoenix.zhtml?c=116148&p=irol-calendar The Annual Biotech in Europe Forum for Global Partnering and Investment brings together a cross-section of early-stage/pre-IPO, late-stage and public companies with leading investors, analysts, money managers and pharma licensing executives. For more information, visit www.sachsforum.com.
biOasis Technologies Inc. (TSX VENTURE: BTI) (OTCQB: BIOAF) wishes to announce, that as part of its going forward strategy, its current listing on the OTCQX will be changing to a listing on the OTCQB. The Company's trading symbol will remain BIOAF. The Company remains in compliance with all relevant TSX Venture and OTCQB reporting standards and will continue trading on both exchanges.
BioNano Genomics, Inc., the leader in physical genome mapping, today announced that it will present five posters supporting the utility of its Irys® System in human genome research at the upcoming 2015 American Society of Human Genetics (ASHG) Annual Meeting being held October 6 – 10, 2015 at the Baltimore Convention Center in Baltimore, Maryland. Erik Holmlin, Ph.D., President and Chief Executive Officer of BioNano Genomics, commented, "We are excited to present findings from a host of different studies that underscore the utility of the Irys System for next generation mapping. The Irys System is proving to be an essential tool for genome analysis because it provides a comprehensive view of genomic architecture. The findings reported in these studies show our ability to provide independent detection of known structural variations and to identify novel large complex structural variations that are beyond the limits of next generation sequencing. We also demonstrate the compatibility of next generation mapping with sequencing to produce highly contiguous hybrid scaffolds that reveal chromosome architecture at a reduced time and cost compared to traditional methods of generating complete genome assemblies."
Cara Therapeutics, Inc. (Nasdaq:CARA), a biotechnology company focused on developing and commercializing new chemical entities designed to alleviate pain and pruritus by selectively targeting kappa opioid receptors, today announced that Chief Medical Officer Joseph Stauffer, D.O., M.B.A., will present two posters at the American Academy of Physical Medicine and Rehabilitation (AAPM&R) Annual Assembly, to be held October 1-4 at the Hynes Convention Center in Boston. These posters will include positive data from Cara's Phase 2 acute pain and human abuse liability studies of CR845, the Company's first-in-class peripherally-selective kappa opioid agonist, which has potential to be the only Schedule V or non-scheduled opioid for acute pain.
Cellectar Biosciences, Inc. (NASDAQ:CLRB) today announced the pricing of a registered direct offering of 1,017,272 shares of its common stock and Series B pre-funded warrants to purchase 482,728 shares of common stock at a price of $2.20 per share. Concurrently in a private placement, Cellectar is issuing Series A warrants to purchase 1,500,000 shares of common stock at an exercise price of $2.83 per share, which are not exercisable for six months from issuance and are exercisable for five years thereafter. Pursuant to a registration rights agreement, the Company has agreed to file a registration statement for the resale of the shares of common stock issuable upon exercise the Series A Warrants. Gross proceeds from this offering are expected to be approximately $3,300,000, before deducting the estimated offering expenses payable by the company. The offering is expected to close on or about September 30, 2015, subject to customary closing conditions.
Cellular Biomedicine Group Inc. (NASDAQ:CBMG), a biomedicine firm engaged in the development of effective stem cell therapies for degenerative diseases and immunotherapies for cancer, today announced results from an expanded Phase I/II clinical trial evaluating the safety, feasibility and anti-tumor activity of its Chimeric Antigen Receptor-Modified T-Cells (CAR-T) immunotherapy (CBM-EGFR.1) targeting wild type EGFR (Epidermal Growth Factor Receptor) for the treatment of patients with EGFR expressing advanced relapsed/refractory solid tumors. Based on the results from 24 patients treated with CBM-EGFR.1 (17 patients with non-small cell lung cancer (NSCLC), 5 patients with cholangiocarcinoma, 1 patient with pancreatic cancer and 1 patient with renal cell carcinoma (RCC)), the early results showed that CBM-EGFR.1 immunotherapy was safe, well tolerated, and had positive signal of clinical activity in several indications.
The Centers for Disease Control and Prevention (CDC) estimate there are 1.2 million people living with Human Immunodeficiency Virus (HIV) in the United States today, as well as nearly 50,000 new diagnoses yearly, with certain populations being adversely affected. Reaffirming Patient Advocate Foundation's (PAF) commitment to improving health access to patients nationwide, the Co-Pay Relief program is pleased to announce the launch of a new HIV and Acquired Immune Deficiency Syndrome (AIDS) treatment and prevention fund available immediately to financially qualified, insured patients. "We understand the financial challenges faced by those who have already been diagnosed with HIV or AIDS, and are grateful to have this opportunity to provide assistance that reduces their financial burden," Fran Castellow, MsED., President of Operations at PAF says. The CDC estimates a patient's lifetime HIV treatment costs were $367,134, with an annual cost of over $20,000 in care. Patients diagnosed with HIV and AIDS are seeing increased out-of-pocket costs and insurance co-payment responsibilities, as well as higher medication costs.
CareDx, Inc. (Nasdaq:CDNA), a molecular diagnostics company focused on the discovery, development and commercialization of clinically differentiated, high-value diagnostic surveillance solutions for transplant recipients, is responding to the Centers for Medicare & Medicaid Services (CMS) Clinical Laboratory Fee Schedule (CLFS) Preliminary Determinations for CY 2016 issued on September 25, 2015. In the draft CLFS, CMS is proposing drastic changes in reimbursement for a number of established molecular diagnostic tests, including AlloMap. The draft CLFS is subject to an open comment period until November 24, 2015. CareDx and other stakeholders believe that additional information provided by stakeholders during the open comment period should be reflected in the final guidance. Final changes to the CLFS will go into effect on January 1, 2016.
Catalyst Biosciences, Inc. (NASDAQ:CBIO), a clinical-stage biopharmaceutical company focused on creating and developing novel medicines to address serious medical conditions, today announced the appointment of Andrew Hetherington as Vice President of Manufacturing Operations. Mr. Hetherington brings to Catalyst Biosciences more than two decades of experience in global commercial and clinical manufacturing, technology transfer and product development. "We are extremely pleased to have Andrew join our executive team as we expect to advance multiple clinical programs during the course of 2015 and 2016," said Nassim Usman, Ph.D., President and Chief Executive Officer of Catalyst. "Given Andrew's significant operational experience, especially with the manufacturing of Bayer's Factor VIII product, he has an excellent combination of skills necessary to lead our manufacturing operations and support our most advanced hemophilia programs."
Cellular Biomedicine Group Inc. (NASDAQ:CBMG), a biomedicine firm engaged in the development of effective stem cell therapies for degenerative diseases and immunotherapies for cancer, today responded to inquiries from investors and the scientific community about its Immuno-Oncology cell therapy platform as it relates to information surrounding CD19, CD20 and CD30. Dr. William (Wei) Cao, Chief Executive Officer of Cellular Biomedicine Group, commented, "We are taking a proactive approach to addressing inquiries from the investment and scientific community on the Company's Immuno-Oncology cell therapy platform on a more real-time basis. This area has been well followed and we would like to take this opportunity to expand on frequently asked questions and dispel misinformation in the marketplace. We will continue to keep our stakeholders apprised of ongoing business and pipeline developments."
Cesca Therapeutics Inc. (NASDAQ:KOOL), an autologous cell-based regenerative medicine company, today announced that Kenneth L. Harris is stepping down as President of the Company and from the Board of Directors in the U.S., as well as in the Indian subsidiaries, effective immediately. Mr. Harris will remain involved with the Company as an independent advisor for up to 18 months. "Ken has spent the best part of the last ten years building Cesca's clinical programs from concept to their current state," commented Craig W. Moore, Cesca's Chairman of the Board. "This has taken creativity, perseverance and many long hours. We appreciate his trust in the Cesca team to bring his ideas to market, and look forward to working with him in his new role," he added. "We have made very significant progress over the last 18 months," said Mr. Harris. "With the FDA approval to commence Cesca's Phase III pivotal clinical trial for CLI secured, and the AMI program approaching Phase II, it is time for me to transition day-to-day leadership responsibility to other members of the Cesca team. I look forward to working with the Company in more of a consultative role, and helping to accelerate the delivery of these important new therapies to the clinic," he added.
Esperion Therapeutics, Inc. (NASDAQ: ESPR), a pharmaceutical company focused on developing and commercializing a first-in-class, oral therapy for low-density lipoprotein cholesterol (LDL-C) lowering for the treatment of patients with hypercholesterolemia, provided an update on the design and timing of its planned pivotal Phase 3 clinical development program following receipt of the official End-of-Phase 2 Meeting Minutes from the U.S. Food and Drug Administration (FDA). Esperion plans to conduct multiple Phase 3 clinical trials that will separately evaluate patients with statin intolerance, as well as patients who are inadequately treated despite maximally tolerated statin therapy. This dual strategy will leverage the profile of ETC-1002 to differentiate the drug in the statin intolerant patient population, while also preserving the opportunity to develop the drug as an add-on to maximally tolerated statin therapy.
Galena Biopharma, Inc. (NASDAQ:GALE), a biopharmaceutical company developing and commercializing innovative, targeted oncology therapeutics that address major medical needs across the full spectrum of cancer care, today announced that data from the GALE-301 Phase 2a portion of the Phase 1/2a clinical trial was presented at the European Cancer Congress 2015 in Vienna, Austria. GALE-301 is Galena's cancer immunotherapy that consists of a peptide (E39) derived from Folate Binding Protein (FBP). GALE-301 is combined with the immune adjuvant, granulocyte macrophage-colony stimulating factor (GM-CSF) and administered via intradermal injection for the prevention of recurrence in ovarian and endometrial cancers.
Intra-Cellular Therapies, Inc. (Nasdaq:ITCI), a biopharmaceutical company, today announced the completion of its previously announced underwritten public offering of 6,900,000 shares of its common stock at a public offering price of $43.50 per share. In addition, the underwriters have exercised an option to purchase an additional 1,035,000 shares of common stock at the public offering price, less the underwriting discounts and commissions. All of the shares in the offering were sold by Intra-Cellular Therapies, with gross proceeds to Intra-Cellular of approximately $345 million and net proceeds of approximately $327.4 million, after deducting underwriting discounts and commissions and estimated offering expenses. Leerink Partners LLC, Cowen and Company, LLC, RBC Capital Markets, LLC and Guggenheim Securities, LLC acted as joint book-running managers for the offering. Ladenburg Thalmann & Co. Inc. and SunTrust Robinson Humphrey, Inc. acted as co-managers for the offering.
Intrexon Corporation (NYSE:XON), a leader in synthetic biology, announced today it has formed a new Exclusive Channel Collaboration (ECC) with ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, for the treatment and prevention of graft-versus-host disease (GvHD), a major complication of allogeneic hematopoietic stem-cell transplantation (HSCT) which significantly impairs the quality of life and survival of many recipients. The collaboration will focus on addressing the underlying pathologies of GvHD through engineered cell platforms to express and deliver interleukin-2 (IL-2), a cytokine critical for modulation of the immune system. "The combined expertise and the knowledge gained from our current research programs with Intrexon in adoptive T-cell therapies and cytokine modulation for treatment of cancer, position us well to develop and implement therapeutic approaches addressing an area of high unmet medical need for patients with GvHD," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM.
Lipocine Inc. (NASDAQ:LPCN), a specialty pharmaceutical company, today announced the first subject dosed in its multi-dose PK dose finding clinical study for LPCN 1107, the company's oral hydroxyprogesterone caproate ("HPC") product candidate, being developed as a potential therapy for the prevention of pre-term birth ("PTB"). The primary objective of the study will be study pharmacokinetics over an extended period of time with multiple dose strengths of oral administration of LPCN 1107 in pregnant women. "We are pleased to initiate this study following recent feedback we received from the U.S. Food and Drug Administration," said Dr. Mahesh Patel, President and CEO of Lipocine Inc. "Pre-term birth continues to be a significant unmet medical need, and LPCN 1107 as potentially the first oral product, may offer patients with an important new treatment option."
Molina Healthcare, Inc. (NYSE:MOH) today announced that it will report its earnings for the third quarter and nine months ended September 30, 2015, after the market closes on Thursday, October 29, 2015. The Company's management will host a conference call and webcast to discuss the earnings release at 5:00 p.m. Eastern time on the same day. The number to call for the interactive teleconference is (212) 231-2926. A telephonic replay of the conference call will be available from 7:00 p.m. Eastern time on Thursday, October 29, 2015, through 6:00 p.m. on Friday, October 30, 2015, by dialing (800) 633-8284 and entering confirmation number 21777769. A live broadcast of Molina Healthcare's conference call will be available on the Company's website, molinahealthcare.com. A 30-day online replay will be available approximately an hour following the conclusion of the live broadcast.
Neothetics, Inc. (NASDAQ:NEOT) today announced that it has named Jeffrey Nugent and Hani Zeini to its Board of Directors. Mr. Nugent is a respected business leader who brings a broad range of valuable experiences to Neothetics. He has over 25 years of experience in healthcare, which includes building successful dermatology and consumer healthcare companies, forging industry leading partnerships, and leading M&A transactions. Currently, Mr. Nugent serves on the Board of Sientra, Inc and is Chairman of the Compensation Committee. Mr. Nugent served as Chief Executive Officer and Worldwide President of Neutrogena, where he led the company's acquisition by Johnson & Johnson. In 2010, he founded Precision Dermatology, a multi-channel dermatology and skincare company and served as Chief Executive Officer. Three years later, the company was sold to Valeant Pharmaceuticals. He began his career at Jonson & Johnson, progressing to a number of senior positions with responsibilities in Research, Marketing, Finance and Manufacturing across Pharmaceutical, Medical Device and Consumer franchises. He was appointed VP of Worldwide Quality, leading an initiative of corporate competitiveness improvement, reporting to the Chairman and CEO. Mr. Nugent holds an MBA in Finance and Marketing from Loyola University of Chicago and BS in Mathematics from St. Joseph's College.
Novadaq® Technologies Inc. (NASDAQ:NVDQ) (TSX:NDQ), the leading developer and provider of clinically relevant fluorescence imaging solutions for use in surgical and diagnostic procedures, today announced the unveiling of SPY-Q Case Manager (SPY-QCM), at the Symposium on Advanced Wound Care ("SWAC") Fall 2015 meeting being held in Las Vegas, Nevada from September 26-28. SPY-QCM is an image acquisition and analysis software toolkit that will also enable physicians treating patients with compromised blood flow and tissue perfusion to assess the effectiveness of chosen treatments over time using new case management functionality. SPY-QCM will compile image data acquired using NOVADAQ's Fluorescence Angiography System over the course of a treatment period, and will provide a clear history of a patient's recovery progress and, if appropriate, allow them to alter treatment strategies to maximize the likelihood of a successful outcome. "Despite the many advances in treatments, monitoring of the wound microcirculation during treatment has been one of the major unmet needs," commented Dr. William W. Li, President of the non-profit Angiogenesis Foundation. "The introduction of a powerful new case management software like SPY-QCM will allow clinicians to perform perfusion assessments serially over a course of treatment and to compare those assessments. Results are displayed immediately and provide arterial inflow and venous outflow intensity and rate maps that, when plotted over time, give clinicians valuable insight into the restoration of the critical local blood supply toward baseline or healthy tissue."
PharmaCyte Biotech, Inc. (OTCQB:PMCB), a clinical stage biotechnology company focused on developing targeted treatments for cancer and diabetes using its signature live-cell encapsulation technology, released today the second in a series of articles that will serve to educate the public on its technology and how it is used in the treatment of advanced pancreatic cancer. This latest article, written by Dr. Matthias Löhr of the famed Karolinska Institute in Stockholm, Sweden, and the Chairman of PharmaCyte Biotech’s Scientific Advisory Board, discusses the unmet clinical need of a large group of patients suffering from locally advanced pancreatic cancer. PharmaCyte Biotech’s Chief Executive Officer, Kenneth L. Waggoner, commented on Dr. Löhr’s article, “Dr. Löhr understands the severity of the unmet medical need he addresses in his article as much as anyone. Dr. Löhr explains his rationale for believing there is an urgent need to develop a therapy for patients with pancreatic cancer that is not currently being addressed by the medical community. Our other prominent oncologists also believe that new therapeutic options are needed for those with locally advanced pancreatic cancer. We agree with Dr. Löhr that PharmaCyte Biotech’s pancreatic cancer treatment has the potential to address squarely this unmet medical need.”
Revance Therapeutics, Inc. (NASDAQ:RVNC), a specialty biopharmaceutical company developing botulinum toxin products for use in aesthetic and therapeutic indications, today announced it has commenced dosing patients in the Phase 3 pivotal study to evaluate the safety and efficacy of its RT001 investigational topical drug product candidate for the treatment of lateral canthal lines, or crow's feet. The Phase 3 trial will evaluate the safety and efficacy of a single, bilateral administration of RT001 topical gel compared to placebo in patients with moderate to severe crow's feet. The company plans to release interim results from this Phase 3 study in the first half of 2016. "Transporting large molecules, such as botulinum toxin, through the skin has the potential to offer many clinical benefits. We anticipate it will be easier for medical professionals to administer a topical product and that patients will be able to avoid the pain, bruising and downtime associated with needles," said Dan Browne, President and Chief Executive Officer at Revance. "Topical delivery has the potential to transform the $3 billion global neurotoxin market and change the way many other drugs are administered in the future. We've invested significant capital and resources to refine our proprietary TransMTS® technology platform, generate and publish meaningful clinical data, build manufacturing capabilities and develop cutting-edge performance analytics. We believe these have positioned Revance to become the first and only company to move into Phase 3 clinical trials with a topical botulinum toxin approach.
Rexahn Pharmaceuticals, Inc. (NYSE MKT:RNN), a clinical stage biopharmaceutical company developing next generation therapeutics for the treatment of cancer, announced today that preliminary clinical data from ongoing Phase I studies of its anti-cancer compounds, Supinoxin™ and RX-3117, were presented on Sunday, September 27, 2015 in two poster presentations at the joint 18th ECCO – 40th ESMO European Cancer Congress 2015 – a biennial congress focused on improving the prevention, diagnosis, treatment and care of cancer patients, taking place in Vienna, Austria, September 25-29, 2015. “We were pleased to have the opportunity to showcase promising preliminary data from both the Supinoxin and RX-3117 clinical programs at this prestigious cancer meeting,” commented Dr. Peter D. Suzdak, Chief Executive Officer of Rexahn Pharmaceuticals. “The initial clinical results demonstrate that both Supinoxin and RX-3117 appear to be safe and well tolerated and also show encouraging preliminary evidence of clinical activity. We believe these results underscore the unique mechanism of action of both compounds and their ability to selectively target key molecular pathways involved in cancer biology.”
Stemline Therapeutics, Inc. (Nasdaq:STML) announced today that Ivan Bergstein, M.D., Stemline's CEO, will present at the Ladenburg Thalmann 2015 Healthcare Conference on Tuesday, September 29, 2015 at 2:00 PM ET. The conference will be held at the Sofitel New York in New York, NY. A live webcast of the presentation can be viewed on the company's website at www.stemline.com.
VBL Therapeutics (NASDAQ:VBLT), today announced full Phase 2 data on VB-111 in combination with Bevacizumab (Avastin™) in rGBM. Data were presented yesterday at the European Cancer Congress 2015, in Vienna, Austria by principal investigator Andrew J. Brenner, MD, PhD. Data showed statistically significant overall survival benefit in patients treated with VB-111 continued with VB-111 in combination with Avastin™ upon disease progression (continuous exposure cohort), compared to patients treated with VB-111 followed by Avastin™ alone, upon disease progression (limited exposure cohort). Patients treated with continuous exposure of VB-111 had median overall survival (mOS) of 15 months, compared to mOS of 8 months in patients with limited VB-111 exposure (p=0.048). In addition, for the first time the company reported overall response rate (ORR) data of 29% (7/24) with 2 complete responders in the continuous exposure cohort, compared 9% (2/22) with no complete responders in the limited exposure cohort.
Vital Therapies, Inc. (Nasdaq:VTL), a biotherapeutic company developing ELAD®, a cell-based therapy targeting the treatment of liver failure, today outlined plans for a possible new phase 3 clinical trial designed to confirm ELAD's safety and efficacy in alcohol-induced liver decompensation (AILD), including severe acute alcoholic hepatitis, based on trends identified in subset analyses of the Company's recently completed VTI-208 clinical trial. On August 21, 2015, the Company announced that while its first phase 3 trial, VTI-208, did not reach its primary or secondary endpoints, medically pertinent pre-specified subsets based on age and disease severity did show promising trends toward efficacy. Since then, the Company has conducted a series of pre-specified and post-hoc analyses of subsets of the data in order to determine if there is a basis for designing and implementing a new clinical trial to confirm the efficacy and safety signals identified in these subsets.
Xtant Medical Holdings, Inc. (OTCQX:BONE), a leader in the development of regenerative medicine products and medical devices, today announced that its wholly owned subsidiaries, X-spine Systems and Bacterin International will be attending the Congress of Neurological Surgeons Annual Meeting held in New Orleans, Louisiana, September 26-30, 2015. Both subsidiaries will be displaying their product portfolio with an emphasis on the Axle® Interspinous System, Irix-A™ Lumbar Integrated Fusion System, Silex® Sacroiliac Joint Fusion System, the Xpress™ Minimally Invasive Pedicle Screw System, OsteoSponge®, and OsteoSelect® DBM Putty. To learn more about Xtant products, visit Booth #1003 at CNS 2015.
Zafgen, Inc. (Nasdaq:ZFGN), a biopharmaceutical company dedicated to significantly improving the health and well-being of patients affected by obesity, today announced the appointment of Robert Perez and Geoffrey McDonough, M.D., to its Board of Directors. Mr. Perez is the former Chief Executive Officer of Cubist Pharmaceuticals, and brings over 28 years of experience from various leadership and senior management positions in the life sciences industry. Mr. Perez has a wealth of experience in guiding corporate strategy in the biotechnology industry, with a focus on drug development and commercialization. Prior to joining Cubist, Mr. Perez served as Vice President of the CNS Business Unit at Biogen, where he was responsible for leading the U.S. neurology franchise. From 1995 to 2001 he served as a Regional Director, Director of Sales, and Avonex Commercial Executive at Biogen. From 1987 to 1995, Mr. Perez held various sales and marketing positions at Zeneca Pharmaceuticals.
ZIOPHARM Oncology, Inc. (Nasdaq: ZIOP), a biopharmaceutical company focused on the development and commercialization of new cancer immunotherapies, today announced the appointment of Scott Tarriff to the Company's Board of Directors. Mr. Tarriff, who serves as President, Chief Executive Officer and a Director of Eagle Pharmaceuticals, brings more than 25 years of pharmaceutical industry experience to ZIOPHARM. "Scott's leadership experience extends from research and development to regulatory and commercial strategy, all of which will be invaluable to ZIOPHARM as its pipeline of leading-edge viro- and cell-based cancer immunotherapies expands," said Sir Murray Brennan, M.D., Lead Director of ZIOPHARM. "We look forward to Scott's contributions as a Director at this important stage of growth for the Company."