|Merrimack Confirms U.S. FDA Approval of ONIVYDE(TM); CardioCell Utilizes BioLife's CryoStor® in Phase II Clinical Trial for Heart Failure|
|By Mary Davila|
|Thursday, 22 October 2015 19:40|
Below is a look at some of the headlines for companies that made news in the healthcare sector on October 22, 2015.
Merrimack (Nasdaq: MACK) announced ONIVYDE™ (irinotecan liposome injection) has been approved by the U.S. Food and Drug Administration (FDA) in combination with fluorouracil (5-FU) and leucovorin for the treatment of patients with metastatic adenocarcinoma of the pancreas after disease progression following gemcitabine-based therapy. ONIVYDE is not indicated for use as a single agent. With this approval, ONIVYDE in combination with 5-FU and leucovorin becomes the first and only FDA-approved treatment option for patients in this setting.
"This is an important day for patients facing pancreatic cancer," said Andrea Wang-Gillam, M.D., Ph.D., Associate Professor of Medicine, Clinical Director of GI Oncology Program, Division of Oncology, at Washington University School of Medicine, St. Louis, and a key investigator on the ONIVYDE Phase 3 NAPOLI-1 study. "With a long history of failed clinical studies in the post-gemcitabine setting, this approval is a significant achievement in the oncology community. It brings a new therapy to the many patients who are facing this aggressive disease and are in need of treatment options."
"We are thrilled to be able to deliver this groundbreaking therapy to patients battling metastatic pancreatic cancer," said Robert Mulroy, President and CEO of Merrimack. "Pancreatic cancer is an aggressive and devastating disease, with very few patients surviving beyond one year. ONIVYDE provides a clinically significant treatment option to a patient population where there is currently no standard of care. We are grateful to the many patients, clinicians, researchers and partners that worked with us to make ONIVYDE available. Today's approval by the FDA is a pivotal milestone in our company's history, representing years of hard work and commitment to our mission of engineering new treatment options for cancer patients in need."
The FDA approval is based on the results of an international Phase 3 randomized, controlled study (NAPOLI-1). In this study, ONIVYDE in combination with 5-FU and leucovorin achieved its primary endpoint of a significant improvement in overall survival (p=0.014, unstratified HR=0.68, 95% CI: [0.50–0.93]) with a 45% improvement in median overall survival of 6.1 months for patients receiving the ONIVYDE combination regimen compared to 4.2 months for patients who received 5-FU and leucovorin alone. The ONIVYDE combination also demonstrated improvement in progression free survival (3.1 months vs. 1.5 months, HR=0.55, 95% CI: [0.41-0.75]). The monotherapy regimen in this study did not achieve its primary endpoint and, therefore, ONIVYDE is not indicated as a single agent. The most common adverse reactions (>20 %) of ONIVYDE were diarrhea, fatigue/asthenia, vomiting, nausea, decreased appetite, stomatitis and pyrexia, and the most common severe laboratory abnormalities (> 10% Grade 3 or 4) were lymphopenia and neutropenia. For additional safety information, please see Important Safety Information including Boxed Warning below.
"This is a pivotal achievement for the pancreatic cancer community because it provides a new treatment option for some patients facing this difficult disease," said Julie Fleshman, President and CEO of the Pancreatic Cancer Action Network. "We applaud the dedication of those involved in this advancement, knowing it will impact our goal to double pancreatic cancer survival by 2020."
There are approximately 49,000 patients diagnosed with pancreatic cancer each year in the United States1, the overwhelming majority of whom have adenocarcinoma2. Most patients receive gemcitabine-based therapy during either adjuvant/neoadjuvant treatment for locally advanced disease or during first- or second-line therapy for metastatic disease, but are left with no standard of care therapy upon progression3. ONIVYDE in combination with 5-FU and leucovorin is now approved for these patients whose disease has progressed following gemcitabine-based therapy.
Merrimack expects ONIVYDE to be available in the United States next week and is committed to supporting rapid physician and patient access to this therapy. PROVYDE™ (ONIVYDE Access Center) offers a variety of reimbursement support services for healthcare providers and financial assistance services for patients. For more information, please call 1-844-ONIVYDE or visit www.ONIVYDE.com.
Baxalta Incorporated (NYSE: BXLT) is responsible for the development and commercialization of ONIVYDE outside of the United States and Taiwan under the exclusive licensing agreement that Merrimack and Baxalta entered into in September 2014. In May 2015, the European Medicines Agency (EMA) accepted for review Baxalta's marketing authorization application (MAA) for ONIVYDE based on the same clinical results.
"We are excited that ONIVYDE (nal-IRI) will now be available to people living with metastatic pancreatic cancer in the U.S. after they progress from gemcitabine-based therapy," said David Meek, Executive Vice President and President of Oncology at Baxalta. "Looking ahead, Baxalta continues to work toward marketing authorization in Europe with the goal of providing nal-IRI to patients in more countries around the world in need of new options."
PharmaEngine, Inc. (Taipei, Taiwan) holds the rights to commercialize ONIVYDE in Taiwan. PharmaEngine filed a New Drug Application (NDA) with the Taiwan FDA in May 2015.
Merrimack to Host Conference Call -- Merrimack will host an investor conference call and webcast at 4:30 p.m., Eastern Time, today, October 22, where it will review the details of the approval. Investors and the general public are invited to listen to the call by dialing (877) 564-1301 (domestic) or (224) 357-2394 (international) five minutes prior to the start of the call and providing the passcode 67436538.
A listen-only webcast of the call can be accessed in the Investors section of Merrimack's website, investors.merrimack.com, and a replay of the call will be archived there for six weeks.
BioLife Solutions, Inc. (NASDAQ: BLFS), the leading developer, manufacturer and marketer of proprietary clinical grade cell and tissue hypothermic storage and cryopreservation freeze media and a related cloud hosted biologistics cold chain management app for smart shippers, today announced that CardioCell, a Stemedica Cell Technologies company, recently published details of its phase II heart failure clinical trial in the Journal of Cardiovascular Medicine. In the article, titled, Rationale and design of a randomized controlled trial of allogeneic mesenchymal stem cells in patients with nonischemic cardiomyopathy, the use of BioLife's clinical grade CryoStor freeze media was disclosed. Details of the study can be found here: A Study to Assess the Effect of Intravenous Dose of (aMBMC) to Subjects With Non-ischemic Heart Failure.
Over the last several years, adoption of BioLife's biopreservation media products including CryoStor and HypoThermosol in the developing and high growth regenerative medicine market has increased significantly due to the products' ability to extend stability and improve viability and functional recovery of starting material and manufactured cell products.
Mike Rice, BioLife's President & CEO, remarked, "We are very proud to support CardioCell and Stemedica in their quest to commercialize potentially life saving cell based therapies for heart failure patients and other indications. HypoThermosol and CryoStor are now embedded in over 200 pre-clinical validations and clinical trials of numerous cell based products and therapies. With this critical mass of regenerative medicine customer clinical trials, and our biologistex™ cloud based cold chain management service that we are just rolling out, we have tremendous opportunities to positively impact the manufacturing and delivery logistics of biologic based medicine and to create significant value for our shareholders."
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 the European Medicines Agency (EMA) has granted an orphan drug designation to its investigational gene therapy product for the treatment of achromatopsia caused by mutations in the CNGA3 gene. AGTC is developing products for achromatopsia based on mutations in the CNGB3 and CNGA3 genes, which together account for 75 percent of the total achromatopsia patient population. The Company has demonstrated proof-of-concept in a naturally occurring dog model of the CNGB3 form of the disease, and previously received orphan drug designation from the U.S. Food and Drug Administration and the EMA for its investigational gene therapy product for the treatment of achromatopsia caused by mutations in the CNGB3 gene. More recent studies conducted by AGTC's collaborators showed that in sheep affected by achromatopsia caused by mutations in the CNGA3 gene, delivery of an AAV vector carrying a normal copy of CNGA3 restored vision as measured by the ability to navigate an obstacle maze. "Receiving EU orphan drug designation is an important milestone that delivers significant commercial benefits to our company as we advance our innovative portfolio of gene therapy products that allow a patient's own body to treat their rare eye disorders," said Sue Washer, President and CEO of AGTC. "There is an enormous unmet need for effective treatment options for patients with achromatopsia, and we believe that our approach, delivered as a single injection into the eye, has the potential to provide long-lasting treatment, leading to a better quality of life for patients worldwide." Orphan medicinal product designation is granted by the European Commission, following a positive opinion from the Directorate-General for Competition (COMP), to a medicinal product that is intended for the diagnosis, prevention or treatment of a life-threatening or a chronically debilitating condition affecting not more than five in 10,000 persons in the Community when the application for designation is submitted. An orphan designation allows a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market.
Avinger, Inc., (NASDAQ:AVGR) a developer and manufacturer of image-guided, catheter-based systems for the treatment of peripheral arterial disease (PAD) and pioneer of the Lumivascular approach to treating vascular disease, today announced that it will release its financial results for the third quarter of 2015 after the close of trading on Thursday, November 5, 2015. The Company will host a corresponding conference call beginning at 1:30pm PT/4:30pm ET. Individuals interested in listening to the conference call may do so by dialing (855) 638-4817 for domestic callers or (262) 912-6051 for international callers and referencing Conference ID: 57934241. To listen to a live webcast, please visit the investor relations section of Avinger's website at: www.avinger.com. A replay of the call will be available beginning November 5, 2015 at 4:30pm PT/7:30pm ET through midnight on November 6, 2015. To access the replay, dial (855) 859-2056 or (404) 537-3406 and reference Conference ID: 57934241. The webcast will also be available on Avinger's website for one year following the completion of the call.
Cempra, Inc. (Nasdaq:CEMP), a clinical-stage pharmaceutical company focused on developing antibiotics to meet critical medical needs in the treatment of bacterial infectious diseases, today reported financial results for the quarter ended September 30, 2015 and provided an update on recent corporate developments. The company will host a webcast and conference call today at 8:00 a.m. EDT. Conference Call and Webcast --- Cempra management will host a webcast and conference call regarding this announcement at 8:00 a.m. EDT today. The live call may be accessed by dialing (877) 377-7553 for domestic callers and (253) 237-1151 for international callers and using conference ID # 58583734. A live webcast of the call will be available from the investor relations section of the company website at www.cempra.com, and will be archived there for 30 days. A telephone replay of the call will be available by dialing (855) 859-2056 for domestic callers, or (404) 537-3406 for international callers, and entering the conference ID # 58583734.
Chimerix (NASDAQ:CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced initiation of dosing in SURPASS (ClinicalTrials.gov ID: NCT02439957), one of the two Phase 3 trials in the brincidofovir kidney transplant program. Both the SUSTAIN (ClinicalTrials.gov ID: NCT02439970) and SURPASS trials are evaluating brincidofovir for the prevention of cytomegalovirus (CMV) disease in kidney transplant recipients; both trials are now actively enrolling. Brincidofovir is an oral nucleotide analog that has shown in vitro antiviral activity against all five families of DNA viruses that affect humans, including herpesviruses and adenovirus, and demonstrated a clinically and statistically significant reduction in CMV infection in a Phase 2 trial in hematopoietic cell transplant (HCT) recipients. Based on that successful study, Chimerix designed and conducted the pivotal SUPPRESS trial for the prevention of clinically significant CMV infection in patients undergoing HCT which completed enrollment in June. Topline data from SUPPRESS are anticipated in early 2016. "SUSTAIN and SURPASS address the need for new CMV prevention therapies in the kidney transplant population," said W. Garrett Nichols, MD, MS, Chief Medical Officer of Chimerix. "We are excited about the opportunity to explore the potential for brincidofovir to prevent CMV disease in kidney transplant recipients, as well as to evaluate the potential to prevent other DNA viral infections and to preserve renal function in these patients. Initiation of these trials marks another significant milestone for the brincidofovir program following the completion of enrollment in SUPPRESS in June."
CNS Response (OTCQB:CNSO) won the Platinum Innovations in Healthcaresm Award by the Adaptive Business Leaders Organization (ABL), a healthcare CEO organization focused on growing great companies. The ABBY awards, held yesterday in Long Beach, CA, recognize the most innovative national firms that improve the delivery and quality of healthcare, while lowering its cost. “CNS Response addresses a major health issue for a significant portion of our population who wrestle with mental health issues,” said ABL President Mimi Grant. “CNS’ PEER Online solution impressed the judges on ABL’s Finalists Selection Committee that it was not only innovative, but has significant clinical and financial metrics that prove their service is reducing the cost of providing quality care. Increasingly, it will be innovative companies, like CNS Response, that will not only win ABBY Awards, but will be big winners as the healthcare industry shifts from ‘volume to value.’” The finalist event was held before a live audience of CEO’s, ABL members and supporters. ABBY Award Winners were chosen by secret ballot following live presentations made by the chief executives of the nine Finalist organizations. This is CEO George Carpenter’s 2nd ABBY award. He previously won the Platinum Award for Workwell Systems in 2004. “The ABBY award is a win for the more than 10,000 patients and their doctors who contributed their outcomes data to help others receive more targeted therapy for mental health illnesses,” said Chairman of the Board Dr. Robin Smith.
Crescendo Bioscience, a wholly-owned subsidiary of Myriad Genetics, Inc. (NASDAQ:MYGN), today announced that new data on Vectra DA will be presented at the American College of Rheumatology/Association of Rheumatology Health Professionals (ACR/ARHP) meeting being held Nov. 7-11, 2015 in San Francisco, Calif.
Cyclacel Pharmaceuticals, Inc. (NASDAQ:CYCC) (NASDAQ:CYCCP), a biopharmaceutical company developing oral therapies that target the various phases of cell cycle control for the treatment of cancer and other serious disorders today announced the dosing of the first patient in a Phase 1 trial of CYC065, the Company’s novel second generation CDK (cyclin-dependent kinase) 2/9 inhibitor, for the treatment of advanced solid tumors. CYC065 was selected from the Company's discovery program in Dundee, Scotland and its development was supported in part by a UK government grant. In preclinical studies CYC065 has demonstrated anti-tumor activity as a single agent in hematological malignancies and solid tumors, including drug-resistant models. CYC065 also combined effectively with other targeted anticancer agents in drug-resistant solid tumor in vivo models. The objective of the Phase 1 trial is to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of CYC065 in advanced cancer patients. The trial is being conducted at the Dana Farber Cancer Institute in Boston. “The recent accelerated approval of palbociclib, a CDK4/6 targeted inhibitor, for breast cancer demonstrates that CDK inhibitors can be an effective class of anti-cancer therapeutics,” said Geoffrey I. Shapiro, M.D., Ph.D., Associate Professor of Medicine at Harvard Medical School and Director of the Early Drug Development Center, Dana Farber Cancer Institute. “CYC065 works differently from palbociclib, since it targets CDKs 2 and 9. In preclinical models, the drug induces apoptosis, or programmed death, of cancer cells irrespective of retinoblastoma (RB) pathway status and has been shown to reverse drug resistance associated with the addiction of cancer cells to cyclin E, a partner protein of CDK2. CYC065 has also been shown to inhibit CDK9-dependent oncogenic and leukemogenic pathways, such as those driven by MYC amplification or Mixed Lineage Leukemia gene rearrangement. Cancer cells manage to survive and evade other treatments by activating these pathways.”
CytoDyn Inc. (OTCQB:CYDY), a biotechnology company focused on the development of new therapies for combating human immunodeficiency virus (HIV) infection, today announced that the first patient in its Phase 3 clinical trial has been injected with PRO 140, the Company's monoclonal antibody for the treatment of HIV. PRO 140 is the first self-injectable antibody in a Phase 3 protocol involving 300 HIV patients that will each be evaluated over a 25-week period. The Company believes that upon successful completion of this Phase 3 study, CytoDyn will have the opportunity to seek accelerated approval based on previously FDA granted fast-track candidate designation. Additionally, CytoDyn intends to apply for a breakthrough designation for PRO 140, as the first self-injectable antibody for HIV monotherapy for patients with resistance issues to the current standard of care, referred to as HAART (Highly Active AntiRetroviral Therapy). The Company's recently completed Phase 2b treatment substitution trial demonstrated that 98% of patients treated with PRO 140 successfully passed four weeks of monotherapy without virologic failure. At the completion of the study some of these patients were offered the option to continue in an extension trial. CytoDyn recently announced that the first group of patients in the extension trial passed one year of monotherapy with complete viral load suppression. Currently, 11 patients are in the extension trial and all 11 have now passed one year of monotherapy with PRO 140.
Emergent BioSolutions Inc. (NYSE:EBS) will host a conference call on Thursday, November 5, 2015 at 5:00 pm (Eastern Time) to discuss the financial results for the third quarter of 2015, recent business developments, financial outlook for full year 2015, and revenue guidance for the fourth quarter of 2015. This conference call can be accessed live by telephone or through Emergent’s website: Live Teleconference Information: Dial in number: (855) 766-6521; International dial in: (262) 912-6157; Passcode: 48261059. Live Webcast Information: Visit www.emergentbiosolutions.com; and select the “Investors” section. Pre-registering for the live call will expedite access and minimize hold times. You will be issued a passcode to bypass the operator and connect directly. To pre-register for the call, visit the following website: http://edge.media-server.com/m/p/xhoiec67/lan/en. A replay of the call can be accessed on Emergent’s website http://www.emergentbiosolutions.com under “Investors.”
Entellus Medical, Inc. (NASDAQ:ENTL), a medical technology company focused on delivering superior patient and physician experiences through products designed for the minimally invasive treatment of chronic and recurrent sinusitis patients, today announced that it will release its financial results for the third quarter of 2015 after the close of trading on Thursday, November 5, 2015. The Company's management team will host a corresponding conference call beginning at 3:30pm CT/4:30pm ET to discuss the financial results and recent business developments. Individuals interested in listening to the conference call may do so by dialing (877) 930-5751 for domestic callers or (253) 336-7277 for international callers, using Conference ID: 50165626. To listen to a live webcast, please visit the investor relations section of the Entellus Medical website at: www.entellusmedical.com. A replay of the call will be available beginning November 5, 2015 at 6:30pm CT/7:30pm ET through midnight ET on November 6, 2015. To access the replay, dial (855) 859-2056 or (404) 537-3406 and reference Conference ID: 50165626. The webcast will also be available on the Entellus Medical website for one month following the completion of the call.
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 Company's NeuVax™ (nelipepimut-S) and GALE-301 and GALE-302 clinical programs will be presented at two upcoming medical conferences. Details of the presentations are as follows: Conference: Society for Immunotherapy of Cancer (SITC) 30th Anniversary Annual Meeting; Poster #: 166; Abstract #: 156 – the abstract will be available on November will be published in the Journal for ImmunoTherapy of Cancer on Wednesday, November 4, 2015; Title: Preliminary report of a clinical trial supporting the sequential use of an attenuated E39 peptide (E39') to optimize the immunologic response to the FBP (E39+GM-CSF) vaccine; Date: Friday, November 6, 2015; Time: 6:15 p.m. to 7:30 p.m. Eastern time; Location: Prince George's Exhibition Hall at the Gaylord National Hotel & Convention Center in National Harbor, Maryland. GALE-301 (E39) and GALE-302 (E39' – variant of E39; previously named J65) are folate binding protein (FB) peptides targeting FBP expressing tumors. The data to be presented at SITC is from a single-center, randomized, single-blinded, three-arm Phase 1b study of GALE-301 and GALE-302 in patients with breast or ovarian cancer diagnosis who have been treated by standard of care and are without evidence of disease. The primary endpoint of the trial is immunological and designed to determine which of the three primary vaccination sequence strategies maximizes long-term specific immunity defined as E39-specific cytotoxic T lymphocytes (CTLs) six months following completion of the primary vaccination series.
Globus Medical, Inc. (NYSE:GMED), a leading musculoskeletal implant manufacturer, announced the introduction of the CREO MIS™ Pedicle Screw System. CREO MIS™ is a thoracolumbar posterior stabilization system featuring extended modular screw heads. The screw heads are one of the smallest diameters available on the market which helps to minimize muscle disruption and to reduce clutter at difficult levels such as L5-S1. The extremely low profile design of CREO MIS™ continues to provide the strength and rigidity of a traditional percutaneous reduction system allowing for manipulation and deformity correction. With reduction options up to 30mm and the ability to assemble the extended screw heads to a wide range of screw offerings, CREO MIS™ is a comprehensive and customizable solution for all cases. “Globus continues to lead the way with a growing portfolio of MIS related products, including this important addition to our CREO® Pedicle Screw System platform. Surgeons appreciate the intuitive simplicity of the CREO MIS™ platform including the ability to insert the locking cap through the screw head in a controlled, guided manner where the rod is then captured, reduced and locked in one simplified step,” commented Andrew Iott, Senior Vice President of Product Development. “CREO MIS has been used in over 300 spine procedures since its launch in the second quarter of 2015, and the feedback from surgeons has been extremely positive. We are proud to add yet another innovative solution to our suite of MIS products. This product is designed to help surgeons treat their patients with less tissue disruption leading to faster recovery times. We will continue to provide innovative solutions that allow our surgeons to better improve the lives of their patients.”
Health Insurance Innovations, Inc. (NASDAQ:HIIQ), a leading developer of affordable health plans, will announce its third quarter 2015 financial results on Monday, November 9, 2015, after market close. The announcement will be followed by a live earnings conference call that same day at 5 p.m. Eastern time. The conference call can be accessed live over the phone by dialing 1-877-407-9039, or for international callers, 1-201-689-8470. A replay will be available two hours after the call and can be accessed by dialing 1-877-870-5176, or for international callers, 1-858-384-5517. The passcode for the live call and the replay is 13623035. The replay will be available until November 16, 2015. Interested investors and other parties may also listen to a simultaneous webcast of the conference call by logging onto the Investor Relations section of the Company's website at investor.hiiquote.com. The online replay will be available for a limited time beginning immediately following the call.
IPC Healthcare, Inc. (Nasdaq:IPCM), a leading national acute hospitalist and post-acute provider organization, announced today that it has acquired Hospital Medicine Consultants, LLC in Chicago, Illinois. The acquisition complements IPC Healthcare's existing market presence for acute and post-acute services in the northwestern suburbs of the Chicagoland area. Commenting on the acquisition, IPC's Chicago Executive Director Jennifer Miraglia said, "We are excited to have Hospital Medicine Consultants join an organization that will help their practice to continue to grow and adapt to the changes occurring in markets across the country in both acute and post-acute care. It is increasingly difficult for independent groups to meet all of the new demands of value-based and more highly coordinated care. The unique combination of infrastructure, specialized expertise, and breadth of experience that IPC brings to its clinicians, offers a support system that is unequaled by other clinician management organizations." "HMC's talented clinicians will help boost IPC's presence in Chicago's northwestern suburbs and give us an improved capability to service both the acute and post-discharge needs of patients in the market," said R. Jeffrey Taylor, president and COO of IPC Healthcare. "Hospital Medicine Consultants is an impressive organization that has built a loyal customer base by providing high quality care and superior service, and we are pleased to welcome the group's clinicians to our IPC family." This acquisition is expected to add more than 20,000 encounters on a combined annualized basis.
LHC Group, Inc. (NASDAQ:LHCG), a national provider of home health, hospice and comprehensive post-acute healthcare services, announced today details for the release of its results for the third quarter and nine months ended September 30, 2015. LHC Group plans to issue its earnings release for the third quarter ended September 30, 2015, after the market closes on Wednesday, November 4, 2015, and will host a conference call on Thursday, November 5, 2015, at 11:00 a.m. Eastern time. The toll-free number to call for this interactive teleconference is (866) 393-1608 (international callers should call (973) 890-8327). A telephonic replay of the conference call will be available through midnight on Wednesday, November 11, 2015, by dialing (855) 859‑2056 (international callers should call (404) 537-3406) and entering confirmation number 45313318. A live broadcast of LHC Group's conference call will be available under the Investor Relations section of the Company's website, www.LHCGroup.com. A one-year online replay will be available approximately an hour following the conclusion of the live broadcast.
Matinas BioPharma Holdings, Inc. (OTCQB:MTNB), a clinical-stage biopharmaceutical company focused on identifying and developing safe and effective therapeutics for the treatment of serious and life-threatening infections, today announced that it has been selected as a Cavendish Innovation Partner and will present at the Cavendish Global Health Impact Forum co-hosted by The Cleveland Clinic, which takes place October 25th to October 28th at The Cleveland Clinic, Cleveland, Ohio. The purpose of the Forum is to help family offices and foundations develop and implement their individual pro-social impact investing, grant-giving, and philanthropy programs within health and the life sciences. To accomplish this mission, the Forum showcases presentations and panel discussions by leading family offices, accomplished foundations, research institutions, and private-sector companies engaged in developing innovations with the potential for transformational impact on disease prevention, diagnosis and treatment. A unique gathering of family offices, Cavendish Impact Forums are hosted by leading institutions around the world and take place three times each year. The next Forum is being co-hosted by The Cleveland Clinic and Cleveland Clinic Innovations. Michael Moffat, Cavendish co-founder and Chief Executive Officer explains, “The theme of our Cleveland Clinic hosted Forum is a ‘Celebration of Philanthropy, Impact Investing and Innovation that is Changing the World.’ With the help of our expert advisors, we conduct a global search of research institutions and private-sector companies in order to identify organizations that meet the required standard of excellence. The quality and originality of Matinas BioPharma’s research and scientific insights in the utilization of lipid-crystal, nanoparticle cochleate drug formulations have the potential to transform the way potent medicines are administered, providing physicians and patients with more tolerable, safer, effective and powerful anti-infective options and positions them to make a major contribution to the battle against the growing global multi-drug resistant ‘superbug’ public health threat.” “We are honored to be selected as a Cavendish Global Innovation Partner and to present at this unique event,” added Jerome D. Jabbour, Co-Founder and Chief Business Officer of Matinas BioPharma. “Our selection is further recognition of our innovative approach to nano-encapsulating existing drugs to formulate safe and effective broad spectrum anti-infective therapies to treat serious and life-threatening infections on a global basis. We welcome this opportunity to introduce our disruptive and potentially transformational products and platform technology to many of world’s most accomplished scientists, thought-leaders and generous philanthropic individuals and families who are all dedicated to the common cause of improving the health and lives of people around the world.”
Medgenics, Inc. (NYSE:MDGN) today announced financial results for the three and nine months ended September 30, 2015. Management Commentary --- "We are very pleased with the acquisition this quarter of our Phase 2-ready CNS asset, NFC-1. This represents the first of what we hope to be many programs to come from our ongoing collaboration with the Center for Applied Genomics at Children's Hospital of Philadelphia, " stated Mike Cola, Chief Executive Officer of Medgenics. "Following the successful completion of our recent equity offering, we look forward to advancing the clinical development of NFC-1 in mGluR mutation positive ADHD and 22Q Deletion Syndrome patients." A recent study of NFC-1, the GREAT trial, a 30 patient Phase 1b study in adolescents with ADHD and disruptions in the metabotropic glutamate receptor (mGluR) gene network, was recently completed. The objectives of the study were to evaluate the safety, tolerability, and pharmacokinetics of NFC-1 and to evaluate the effect of NFC-1 on ADHD during four weeks of continuous treatment following one week of placebo therapy in several validated ADHD scales in mGluR+ adolescents with ADHD symptoms. The treatment effect of NFC-1 appeared more robust over time and at higher doses. NFC-1 was well tolerated, with no treatment-related serious adverse events reported. Additionally, 20 of the 30 enrolled patients elected to continue in a long-term safety trial in order to maintain access to therapy. Results from the GREAT trial will be presented at the upcoming 62nd Annual Meeting of the American Academy of Child and Adolescent Psychiatry (AACAP) meeting on Saturday, October 31st at 10:00AM in San Antonio, TX. "We look forward to the results of the GREAT study being presented at the upcoming AACAP meeting, and our entire team is excited to further the development of NFC-1 for the treatment of patients with serious CNS disease," stated Dr. Garry Neil, CSO of Medgenics.
Nature's Sunshine Products, Inc. (NASDAQ:NATR), a leading natural health and wellness company engaged in the manufacture and direct selling of nutritional and personal care products, announced today that it has made several remarkable discoveries regarding the ability of its ProArgi-9+ formula to support circulatory function and inhibit myeloperoxidase, an enzyme that is known to oxidize LDL cholesterol. Following these discoveries by the Company's scientists at The Hughes Center for Research and Innovation, Nature's Sunshine filed a United States provisional patent application on the ProArgi-9+ formula. "Numerous studies have shown the cardiovascular benefits of L-arginine," explained Nature's Sunshine's Chief Scientific Officer, Dr. Matthew Tripp. "However, our preliminary in vitro and human studies showed that the unique combination of ingredients in our ProArgi-9+ formula enhances the effects of L-arginine within the body in several ways." The body converts the amino acid L-arginine into nitric oxide to support healthy circulatory and cardiovascular function. Although L-arginine alone can increase the surrogate marker of saliva nitric oxide, this is a gradual process. In testing ProArgi-9+, Nature's Sunshine found that its formula significantly and rapidly increases the level of nitric oxide in the body. Previous studies have also shown that L-arginine has no significant effect on myeloperoxidase, an enzyme that inhibits nitric oxide production. Additionally, L-arginine has no significant influence on inhibiting the oxidation of LDL cholesterol. The Hughes Center's preliminary study discovered that the unique combination of ingredients found in Synergy WorldWide's ProArgi-9+ potently inhibited myeloperoxidase and helps to reduce the oxidation of LDL cholesterol. "This is why we invested millions of dollars to build The Hughes Center for Research and Innovation," said Nature's Sunshine's Chairman and Chief Executive Officer, Gregory L. Probert. "The knowledge we gain through the Hughes Center truly advances the science of feeling better and helps us transform lives around the world. These are the discoveries that set our brand apart from competitors in the market and help make Nature's Sunshine the world's premier provider of natural health supplements."
Novavax, Inc. (Nasdaq:NVAX), a clinical-stage vaccine company focused on the discovery, development and commercialization of recombinant nanoparticle vaccines and adjuvants, today announced that enrollment has begun in a Phase 2 rollover clinical trial of its respiratory syncytial virus F-protein nanoparticle vaccine candidate (RSV F Vaccine) in older adults enrolled in the prior Phase 2 trial. The trial is a randomized, observer-blinded, placebo-controlled rollover trial designed to enroll the same 1600 adults 60 years of age and older (older adults) who participated in the recently concluded prior Phase 2 trial. Participants previously randomized to receive 135µg RSV F Vaccine or placebo will be re-enrolled and re-randomized in the current trial to receive either 135µg RSV F Vaccine or placebo. This will result in analysis of four separate study arms: a) participants receiving RSV F Vaccine in both the first trial and second trial; b) participants receiving placebo in the first trial and RSV F Vaccine in the second trial; c) participants receiving RSV F Vaccine in the first trial and placebo in the second trial; and d) participants receiving a placebo in both the first trial and second trial. The primary endpoints of the trial will evaluate safety and serum anti-F IgG antibody concentrations in response to immunization with the RSV F Vaccine. Secondary endpoints will examine palivizumab-competing antibody (PCA) concentration and neutralizing antibody titer to at least one RSV/A and one RSV/B strain. “It is estimated that 2.4 million adults 65 years of age or older are infected with RSV annually in the U.S., leading to as many as 900,000 medical interventions and 14,000 deaths each year,” said Stanley C. Erck, President and CEO. “This RSV F Vaccine rollover trial will provide important information on the amplitude and duration of immunogenicity in older adults, which will be a key data set as we develop the RSV F Vaccine for annual, seasonal vaccination.”
RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL), an Israeli biopharmaceutical company primarily focused on late clinical-stage, proprietary, orally-administered, small molecule drugs for inflammatory and gastrointestinal diseases, including cancer, today announced that the U.S. National Cancer Institute ("NCI") has awarded Apogee Biotechnology Corporation ("Apogee") a $225,000 Small Business Innovation Research Program ("SBIR") grant to support a pre-clinical study with YELIVA™ (ABC294640) for the treatment of prostate cancer. In March 2015, RedHill acquired from Apogee the exclusive worldwide rights to YELIVA™ (ABC294640), a proprietary, first-in-class, orally-administered sphingosine kinase-2 ("SK2") selective inhibitor. Following prior pre-clinical studies in early-stage and advanced prostate cancer models, the NCI grant is intended to support additional studies with YELIVA™ (ABC294640) to determine its therapeutic efficacy in in vitro and in vivo models of prostate cancer in combination with radiotherapy. These pre-clinical studies could potentially support future clinical studies with YELIVA™ (ABC294640) for this important indication with over 200,000 estimated new cases of prostate cancer in the U.S. in 2015. The previous prostate cancer pre-clinical study, supported by a grant from the Pennsylvania Department of Health, a Prostate Cancer Foundation Young Investigator award, and a Prostate Cancer Foundation Mazzone Challenge award, is described in an article authored by scientists from Apogee and from the Kimmel Cancer Center at Thomas Jefferson University, and was initially published online in Molecular Cancer Research. The article, entitled "Downregulation of Critical Oncogenes by the Selective SK2 Inhibitor ABC294640 Hinders Prostate Cancer Progression", describes the findings of the pre-clinical study, suggesting that oral administration of YELIVA™ (ABC294640) disrupts multiple oncogenic signaling pathways that are deregulated in prostate cancer, including significant inhibition of tumor growth, proliferation and cell cycle progression. In particular, the article noted that YELIVA™ (ABC294640) inhibited, in vitro, several very resistant types of prostate cancer. The authors of the article conclude that their pre-clinical findings support the hypotheses that SK2 activity is required for prostate cancer growth and that YELIVA™ (ABC294640) could represent a new pharmacological agent for the treatment of aggressive prostate cancer. A Phase I/II clinical study was recently initiated in the U.S. evaluating YELIVA™ (ABC294640) in patients with refractory/relapsed diffuse large B-cell lymphoma (DLBCL), primarily in patients with HIV-related DLBCL. The study is being conducted at the Louisiana State University Health Sciences Center (LSUHSC) in New Orleans and is supported by a grant awarded to Apogee from the NCI Small Business Technology Transfer (STTR) program.
Sucampo Pharmaceuticals, Inc. (NASDAQ:SCMP), a global biopharmaceutical company, today announced the closing of a $250.0 million credit facility in connection with the financing of its acquisition of R-Tech Ueno (TSE:4573:JP). The loans under the credit facility were fully allocated to institutional investors. The loans under the credit facility bear interest at a LIBOR (subject to a 1% floor) plus 7.25% or base rate (subject to a 2% floor) plus 6.25% and a final maturity date of October 16, 2021. Sucampo has funded all shares and stock acquisition rights tendered during the previously announced tender offer and has also closed its share purchase agreement with R-Tech Ueno's founders and a related entity. As a result, upon settlement of the share transfer from the founders, Sucampo will control approximately 98% of R-Tech Ueno shares. Sucampo intends to acquire all remaining outstanding shares of R-Tech Ueno through a squeeze-out process under Japanese law. Sucampo continues to expect to close the acquisition of the remaining shares in the fourth quarter of 2015.
Vical Incorporated (Nasdaq:VICL) today announced that the company will report financial results for the three and nine months ended September 30, 2015, before the opening of trading on Thursday, October 29, and conduct a conference call and webcast to discuss the financial results and provide a company update at noon Eastern Time on Thursday, October 29. The call will be open on a listen-only basis to any interested parties. The company will also provide a business update, including details on independent and partnered development programs in the conference call and webcast. Conference Call -- To listen to the conference call, dial in approximately ten minutes before the scheduled call to (719) 325-2376 (preferred), or (888) 401-4669 (toll-free), and reference confirmation code 3993142. A replay of the call will be available for 48 hours beginning about two hours after the call. To listen to the replay, dial (719) 457-0820 (preferred) or (888) 203-1112 (toll-free) and enter replay passcode 3993142. The call also will be available live and archived through the events page at www.vical.com. Invited participants may ask questions during the conference call. Others may submit questions before the call by e-mail addressed to [email protected] or by fax to (858) 646-1150. Submitted questions will be screened for appropriateness and general interest. Selected questions received with sufficient notice before the call will be answered as time permits at the end of the call. For further information, contact Vical's Investor Relations department by phone at (858) 646-1127 or by email at [email protected]
Vital Therapies, Inc. (Nasdaq:VTL), a biotherapeutic company developing the ELAD® System, a cell-based therapy targeting the treatment of liver failure, today announced that it intends to offer approximately $30,000,000 of shares of its common stock in an underwritten public offering. In addition, Vital Therapies expects to grant the underwriter of the offering a 30-day option to purchase up to an additional approximately $4,500,000 of shares of its common stock at the public offering price, less the underwriting discounts and commissions. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering. Vital Therapies currently plans to use the net proceeds from this offering for the continuing clinical development of the ELAD System, working capital and other general corporate purposes. BofA Merrill Lynch is acting as sole book-running manager for the offering.
WaferGen Bio-systems (NASDAQ:WGBS) announced today that the Company has signed a supply agreement with Reprogenetics, the largest U.S. genetics laboratory specializing in Preimplantation Genetic Screening (PGS) and Preimplantation Genetic Diagnosis (PGD), for use of WaferGen's Apollo 324TM Next Generation Sequencing (NGS) sample preparation system. The Apollo 324™ will enable automation of NGS library preparation for PGS testing used in in-vitro fertilization (IVF). The Apollo 324™ is a compact and versatile platform that enables rapid and full walk-away library prep automation for NGS applications. It combines precise liquid handling and proven chemistries to provide a streamlined sample-to-answer solution for robust library preparation on all major NGS platforms. "We look forward to supporting the leader in PGS and PGD testing, as Reprogenetics continues to deliver high-quality personalized care in the IVF field," said Rollie Carlson, CEO of WaferGen Bio-systems. "The Apollo 324™ NGS sample preparation system will enable improved workflow and will create additional resources for Reprogenetics' transition of their PGS services to NGS."
Xenon Pharmaceuticals Inc. (Nasdaq:XENE), a clinical-stage biopharmaceutical company, today announced that the Company's partner Genentech, a member of the Roche Group, has advanced a second Nav1.7 inhibitor into clinical development. GDC-0310 is a selective, oral Nav1.7 small-molecule inhibitor, identified as part of the companies' pain collaboration focused on the Nav1.7 target. Genentech has initiated a Phase 1 clinical trial in healthy volunteers for GDC-0310. Genentech is also developing GDC-0276, a selective, oral Nav1.7 small-molecule inhibitor for pain which is currently in a Phase 1 clinical trial. "We are delighted with the advancement of a second Nav1.7 inhibitor into clinical development, underscoring the potential of this novel class of compounds as a new way to treat pain, and the productivity of our broad Nav1.7-focused discovery and development collaboration with Genentech," said Dr. Simon Pimstone, President and Chief Executive Officer of Xenon. "We believe that Nav1.7 is a validated pain target and that both GDC-0276 and GDC-0310 are promising candidates."
Zafgen, Inc. (Nasdaq:ZFGN), a biopharmaceutical company dedicated to significantly improving the health and well-being of patients affected by obesity and complex metabolic disorders, today announced a clinical update for beloranib, the Company’s lead MetAP2 inhibitor product candidate. After review of its ongoing clinical trials, the Company has elected to proceed with efficacy and safety data analysis and close the randomized portion of its Phase 3 ZAF-311 clinical trial of beloranib in patients with Prader-Willi syndrome (PWS) and its ZAF-203 Phase 2b clinical trial of beloranib in patients with severe obesity complicated by type 2 diabetes. Zafgen believes that a sufficient number of patients have completed randomized treatment in both clinical trials to assess the efficacy of beloranib and help inform next steps for the beloranib program. Following the partial clinical hold announced last week, the Company believes it can best preserve the integrity of the data in each clinical trial by closing the randomized portion of the clinical trials early. The Company, based on consultation with the U.S. Food and Drug Administration (FDA), expects ZAF-311 to remain a pivotal clinical trial. The Company expects to report top-line results from both the ZAF-311 and ZAF-203 clinical trials in the first quarter of 2016. Zafgen will continue the six-month open label extension (OLE) of the ZAF-311 clinical trial in PWS to obtain important ongoing efficacy and safety data. As previously planned, the Company is continuing to offer an open-ended, unblinded extension study after patients have completed six months of OLE. In consultation with the FDA, a full assessment of the safety and efficacy data from ZAF-311 will be performed to inform the design of ZAF-312, the Company’s second PWS Phase 3 clinical trial. The FDA has informed the Company that it will review the ZAF-311 clinical trial results on the basis of an abbreviated data package. “The strategic decision to analyze results from the ZAF-311 and ZAF-203 clinical trials early allows us to better preserve data integrity by limiting dose interruption in both clinical trials, and inform the future development of this important product candidate,” said Dennis Kim, M.D., Chief Medical Officer of Zafgen. “PWS is a life-threatening and complex rare disorder that severely impacts the quality of life of both patients and their families, and we continue to believe that beloranib may have an important role in addressing hyperphagia and underlying obesity associated with this disorder. We will continue to treat, closely monitor and follow patients in the ZAF-311 extension portion of the clinical trial.”