American Cryostem Corp., of Eatonton, N.J., said it entered a license and collaboration agreement with Racemd to apply its technologies to develop a therapy for treating Duchenne muscular dystrophy (DMD). The technologies include autologous cell retrieval, storage, expansion and multiple administrations. Racemd is a 501(c)3 focused on creating treatments for DMD patients.
Aradigm Corp., of Newark, Calif., said the Bankruptcy Court entered an order approving its sale to Grifols SA, of Barcelona, Spain, of assets and intellectual property that pertain to Lipoquin, Free Ciprofloxacin, Apulmiq and any derivatives. The sale excludes Aradigm's cash and cash equivalents, accounts or notes receivable, certain contracts, tangible personal property, the company name, its accounts and records, all benefit plans and company shares. The purchase price is $3.2 million cash payable at closing; waiver of proof of claim No. 4 for $19.95 million filed by Grifols in Aradigm's bankruptcy case; waiver of proof of claim No. 5 for $11.8 million filed by Grifols’ affiliate in the bankruptcy case; a milestone payment of $2 million payable upon approval of any Aradigm product by the FDA; a milestone payment of $1 million payable upon approval of any Aradigm product by the EMA; and, during the royalty term, Aradigm will receive 25% of any royalties received by Grifols. Aradigm filed for bankruptcy protection in February 2019, a month after the company received a complete response letter for Linhaliq, to treat noncystic fibrosis bronchiectasis chronic lung infections with Pseudomonas aeruginosa. Citing inconsistent efficacy data and concern over pivotal trial endpoints, FDA advisors voted it down.
Brainstorm Cell Therapeutics Inc., of New York and Petach Tikvah, Israel, said it received a new grant of about $1.5 million from the Israel Innovation Authority. The grant enables Brainstorm to continue developing cellular manufacturing capabilities for treating neurodegenerative disorders. The authority has supported Brainstorm since 2007 by providing it grants of about $11.4 million. Brainstorm will be required to pay mid-single-digit royalties to the authority based on sales of the products, up to the cumulative amount of the grants received plus accumulated interest.
Capricor Therapeutics Inc., of Los Angeles, said it is providing CAP-1002, its cell therapy for treating advanced COVID-19, under the compassionate use act, allowing the FDA to immediately collect data on experimental treatments and then make decisions about safety and efficacy. Capricor also said it submitted an expanded access IND to investigate use of CAP-1002 in certain COVID-19 patients. CAP-1002 consists of allogeneic cardiosphere-derived cells, a cardiac cell therapy designed to modify the immune system’s ability to encourage cellular regeneration.
South Korea’s Celltrion Inc. said it successfully transitioned to the second phase of developing an antiviral COVID-19 treatment. The first phase secured 300 different types of antibodies that bind to the antigen. The antibody library was created using the blood of recovered patients in Korea. In cooperation with the Korea Centers for Disease Control and Prevention, Celltrion said it screens the antibodies to find the most effective in neutralizing SARS-CoV-2, the virus responsible for COVID-19. Celltrion said it anticipates the candidate screening to conclude by mid-April.
Emergent Biosolutions Inc., of Gaithersburg, Md., said it is partnering with the Biomedical Advanced Research and Development Authority (BARDA) to develop a plasma-derived therapy for treating COVID-19. Emergent has received $14.5 million from BARDA to support its COVID-HG program, one of two hyperimmune development programs announced by Emergent in March. COVID-HIG is a human hyperimmune product being developed for treating COVID-19 in severe hospitalized patients and high-risk, acute symptomatic patients to prevent progression to severe symptoms.
Glaxosmithkline plc, of London, disclosed on its website that it will collaborate with Xiamen Innovax Biotech Co. Ltd., of Xiamen, China, to evaluate a recombinant protein-based coronavirus vaccine candidate (COVID-19 XWG-03), being developed by Innovax with Xiamen University. GSK will provide Innovax with its pandemic adjuvant system for preclinical evaluation of the vaccine. The COVID-19 XWG-03 vaccine candidate technology is based on a series of truncated S (spike) proteins, which will be screened during preclinical testing, with a lead candidate to be determined by immunogenicity data. GSK also said it entered the COVID-19 Therapeutics Accelerator research effort and will contribute by making available compounds from its libraries for screening for activity against COVID-19.
Incyte Corp., of Wilmington, Del., said it is working with the FDA to initiate a phase III trial to evaluate the efficacy and safety of ruxolitinib (Jakafi) plus standard-of-care (SoC), compared to SoC therapy alone, in patients with COVID-19-associated cytokine storm. The study is sponsored by Incyte in the U.S. and Basel, Switzerland-based Novartis AG everywhere else. Incyte also said it intends to initiate a separate open-label emergency expanded access program in the U.S., allowing eligible patients with severe COVID-19-associated cytokine storm to receive ruxolitinib while it is being studied. Ruxolitinib is a JAK1/JAK2 inhibitor. Because many patients with severe respiratory disease due to COVID-19 have features consistent with cytokine storm and increased activation of the JAK-STAT pathway, Incyte said ruxolitinib may be a possible treatment.
Innate Pharma SA, of Paris, joined French Immunology cluster Marseille Immunopole (MI) and a group of Marseille-based hospitals, including the emergency, intensive care and internal medicine units at La Timone, North and Laveran hospitals and the MI/AP-HM immunoprofiling laboratory at La Timone hospital to launch an exploratory research task force named EXPLORE COVID-19. The goal of the research project is to gain translational insights to better understand the immune response in COVID-19 patients and identify potential new ways to fight the viral infection. Work will include a focus on Innate Pharma targets that may have a role in controlling viral infection or SARS-Cov-2-induced inflammation.
Ligand Pharmaceuticals Inc., of San Diego, said it acquired the core assets of Icagen Inc.’s North Carolina operations. The purchase price was $15 million in cash, with Icagen entitled to receive additional cash payments based on certain revenue achievements. Icagen focused on ion channels, transporters and X-ray fluorescence. Ion channels are key components in a variety of biological processes involving rapid changes in cells and have therapeutic applicability toward cancer, metabolic disease, pain, neurological diseases and infectious diseases.
Neurocrine Biosciences Inc., of San Diego, said it is unsure if it can acquire an adequate commercial inventory of opicapone so it is delaying its potential commercial launch until later in 2020. Opicapone, a catechol-O-methyltransferase inhibitor for treating Parkinson’s disease, has an April 26 PDUFA date.
Regenerx Biopharmaceuticals Inc., of Rockville, Md., said a new scientific study demonstrating a mechanism of action of thymosin beta 4 (Tβ4) on human corneal epithelial cell migration and the downstream signaling pathways has been published online in Current Eye Research. Although Tβ4 is in phase III trials for treatment of eye disorders such as dry eye syndrome and neurotrophic keratitis, the effect of Tβ4 on human corneal epithelial cell (HCEC) migration and proliferation and the precise downstream signaling pathway(s) have not been completely identified. The results of the study suggest that Tβ4-mediated HCEC proliferation and migration are associated with increased ATP levels, P2X7 R-mediated Ca2+ influx, and the ERK1/2 signaling pathway, which describes one of the mechanisms of action for Tβ4-mediated corneal healing and regeneration.
Ridgeback Biotherapeutics LP, of Miami, said it was awarded a contract from the U.S. Biomedical Advanced Research and Development Authority for $10.9 million to support the development of ansuvimab (MAb-114), an experimental Ebola treatment. The contract includes options for an additional $139.8 million to be awarded.
Savara Inc., of Austin, Texas, said it entered an exclusive license and collaboration agreement with Barcelona-based Grifols SA for apulmiq (inhaled liposomal ciprofloxacin). Also known as Linhaliq in Europe, apulmiq is a late-stage investigational inhaled antibiotic in phase III development for the treatment of noncystic fibrosis bronchiectasis. Savara provided an up-front payment and, if regulatory approval is obtained for the compound, Grifols will be eligible for regulatory milestone payments as well as royalties and potential tiered sales milestones upon commercialization.
Seneca Biopharma Inc., of Germantown, Md., said it completed its cell manufacturing facility in Suzhou, China, that will be used to manufacture NSI-566, its lead stem cell therapy, for clinical trials within China, including a non-GCP-compliant phase II trial currently underway for the treatment of chronic ischemic stroke in Beijing.
Siga Technologies Inc., of New York, said the Canadian Department of National Defence has awarded a contract to Meridian Medical Technologies Inc., in which it will purchase up to 15,325 courses of oral Tpoxx (tecovirimat). The four-year contract is valued at $14.3 million, with an initial purchase of 2,500 courses amounting to $2.3 million. In 2018, the FDA approved oral Tpoxx, a small-molecule antiviral, for the treatment of smallpox to mitigate the impact of a potential outbreak or bioterror attack. Siga is currently targeting a Canadian regulatory filing for the drug in the second half of 2020, with an anticipated regulatory approval in 2021. In June last year, Siga entered an international promotion agreement with Meridian that will promote the sale of oral Tpoxx for the treatment of smallpox in all international markets, except the U.S. and South Korea.
Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan, and the Proteo-Science Center at Ehime University said the preclinical development project for a new malaria transmission-blocking vaccine (TBV), which the two organizations are jointly conducting with the nonprofit Path in the U.S., has been awarded a grant from the Global Health Innovative Technology Fund. The candidate Plasmodium falciparum (malaria) vaccine incorporates an optimized immunogen, Pfs230D1+ discovered by Ehime University and Path, and was formulated with Sumitomo’s TLR7 adjuvant (DSP-0546E). It could block parasite transmission from human to mosquito, the parties believe. When launched, the formulation could be a transformational tool as the world’s first malaria TBV to aid the elimination of malaria, they said.
Voltron Therapeutics Inc., of New York, signed a sponsored research agreement with the Vaccine and Immunotherapy Center (VIC) of the Massachusetts General Hospital. The goal of the collaboration is to co-develop a new vaccine designed to protect patients at risk of COVID-19 infection, leveraging the self-assembling vaccine platform developed by the VIC and licensed exclusively to Voltron. Halovax, a subsidiary of Voltron, will spearhead the development.