Adamis Pharmaceuticals Corp., of San Diego, said it has conducted, in collaboration with the Human Immune Monitoring Center at Stanford University, a study to investigate the effects of Tempol on immune cells from COVID-19 patients. Preliminary data show that Tempol decreases cytokines from stimulated cells from COVID-19 patients. The collaboration expects to submit the final data to a peer-reviewed journal.
Aeterna Zentaris Inc., of Charleston, S.C., said it licensed, from the Julius-Maximilians-University Wuerzburg, the exclusive worldwide rights to develop, manufacture and commercialize targeted, highly specific, autoimmunity modifying proteins for treating neuromyelitis optica spectrum disorder. In collaboration with the university and the university’s clinic, Aeterna said it plans to conduct further preclinical research to identify and characterize the development candidate for treating the disorder, including meeting with regulatory authorities to confirm further preclinical data.
Applied Genetic Technologies Corp., of Gainesville, Fla., said new data from its studies of patients with achromatopsia provide the first reported quantitative evidence of improvements in visual sensitivity, supporting the positive patient reported outcomes and provide a path forward to collect additional data. Seven of the 16 patients in the three highest dose groups in the ACHMB3 trial showed improvements in visual sensitivity, in the treated area, as measured by static perimetry, the company said. Achromatopsia is an inherited retinal disease that is present from birth and characterized by the lack of cone photoreceptor function. The company is developing AAV-based therapies for treating rare diseases.
Asklepios Biopharmaceutical Inc. (Askbio), of Research Triangle Park, N.C., said the clinical team from Brain Neurotherapy Bio Inc., of Columbus, Ohio, has joined Askbio to expand its clinical pipeline for treating neurodegenerative disorders, specifically development efforts in treating Parkinson’s disease and multiple system atrophy. The lead clinical program, glial cell-derived neurotrophic factor gene therapy for Parkinson’s disease, is recruiting and treating patients in a phase Ib study in the U.S. A phase I/II study for eligible patients with multiple system atrophy is expected to begin enrollment and treatment in early 2021, Askbio added.
Betterlife Pharma Inc., of Vancouver, British Columbia, said it entered an agreement with Eurofins Discovery for TD-0148A’s IND-enabling pharmacology studies. TD-0148A is a second-generation lysergic acid diethylamide (LSD) derivative molecule that Betterlife said it believes will mimic the projected therapeutic potential of LSD without causing the undesirable psychoactive dissociative side effects, such as hallucinations. Eurofins Discovery has a portfolio of standard and custom in vitro safety and pharmacology assays and panels for drug screening and profiling.
Cybrexa Therapeutics Inc., of New Haven, Conn., said it completed the GLP toxicology study of its lead candidate, CBX-12. The positive results will serve as a guide for dosing regimen for the planned phase I trial of CBX-12, the company said, adding that it plans to file the IND during the first quarter 2021. In the GLP toxicology studies, CBX-12 was delivered via I.V. infusion, and based on multiple parameters, including mortality, clinical observations, body weights and food consumption, the results established a significant therapeutic index of about 20X over exatecan for CBX-12, the company said. CBX-12 is a novel treatment for solid tumors that includes a highly potent topoisomerase I inhibitor payload.
New data from Genocea Biosciences Inc., of Cambridge, Mass., showed its Atlas bioassay targets tumor mutations that are either neoantigens that activate antitumor responses or inhibitory antigens that are targets of pro-tumor responses, in both CD8+ (killer) and CD4+ (helper) T cells. The data improve neoantigen immunotherapies by potentially ensuring they are targeting the right neoantigens and excluding inhibitory antigens, the company said. The Atlas profiling of neoantigen-specific T-cell responses in a cohort of lung cancer patients revealed that many of tumor-specific helper and killer T cells were inhibitory and shut off neighboring beneficial T-cell responses.
Hoth Therapeutics Inc., of New York, extended a research collaboration agreement with Weill Cornell Medicine to continue investigating the therapeutic mechanism of action and safety of HT-003, a novel retinoic acid metabolism blocking agent, for the treatment of acne. The one-year agreement extension is based on positive results generated from the prior research agreement that support a therapeutic effect of HT-003 in key signaling pathways contributing to the development of acne.
Imaginab Inc., of Los Angeles and Neuvogen Inc., of San Diego, signed a multiyear nonexclusive licensing agreement. Imaginab will work with Neuvogen to implement clinical doses of 89Zr CD8 Immuno-PET imaging agents into the therapeutic vaccine clinical trials, and provide technical and clinical support to Neuvogen's development teams. Imaginab will receive license fees and other contingent payments. No other terms were disclosed.
Eli Lilly and Co., of Indianapolis, and Asahi Kasei Pharma Corp., of Tokyo, disclosed a license agreement whereby Lilly will acquire the exclusive rights for AK-1780 from Asahi. The drug is an orally bioavailable P2X7 receptor antagonist that recently completed phase I single and multiple ascending-dose and clinical pharmacology studies. P2X7 receptors have been consistently implicated in neuroinflammation, a driving force in chronic pain conditions, the companies noted. Lilly will be responsible for future global development and regulatory activities for AK-1780. Lilly will pay Asahi $20 million up front and Asahi may be eligible for up to $210 million in potential development and regulatory milestone payments. Asahi will retain the right to promote AK-1780 in Japan and China (including Hong Kong and Macau). If the drug is successfully commercialized, Asahi would be eligible for up to $180 million in potential sales milestones, as well as tiered royalties ranging from the mid-single to low-double digits.
Liminal Biosciences Inc., of Laval, Quebec, provided an update on corporate strategy for its small-molecule therapeutics and plasma-derived based therapeutics platforms. The firm has decided to refocus its resources on its small-molecule therapeutics business, and determined plasma-derived therapeutics are no longer aligned with that overall strategy. Liminal said it has commenced a process to evaluate potential strategic alternatives for that portion of its business aimed at minimizing cash burn.
Mydecine Innovations Group Inc., of Denver, said it is sponsoring a study, titled “Facilitating extinction of fear with psilocybin: Model development of biomarkers and mechanisms,” at the University of Maryland School of Medicine in Baltimore. The research will test a novel hypothesis on the mechanism of psilocybin action in a lab setting. The goal of the research is to get a pointed understanding of the heightened plasticity that permits the therapeutic response to psilocybin in order to obtain a better understanding of when, where and how psilocybin is most appropriate for use in human therapeutics.
Nevakar Inc., of Bridgewater, N.J., and Laboratoires Théa SAS, of Clermont-Ferrand, France, signed an exclusive licensing agreement for the commercialization of NVK-002 in Europe. NVK-002 is an investigational, preservative-free eye drop administered nightly and intended for slowing the progression of myopia in children ages 3 to 17. It is currently under clinical evaluation in the Childhood Atropine for Myopia Progression study, a phase III effort in the U.S. and Europe. Nevakar will develop and seek to obtain EMA approval for the product, and Théa will launch, distribute and support it in Europe, as well as in parts of North Africa. In total, Nevakar has the potential to receive up to $135 million in payments upon the successful attainment of several key regulatory and sales milestones. In exchange, Nevakar will be entitled to tiered royalty payments, ranging from the mid-to-upper teens, on net sales of NVK-002 in the territory.
Provention Bio Inc., of Red Bank, N.J., reported positive data from a preclinical proof-of-concept study for PRV-3279, a DART (bispecific antibody-based molecule) targeting the B-cell surface proteins CD32B and CD79B, conducted in a murine model of gene therapy for Pompe disease. A PRV-3279 mouse surrogate was tested in mice transgenic for human CD32B, which received gene therapy with an adeno-associated virus (AAV) vector AAV9 encoding for the enzyme acid-alpha-glucosidase (GAA) gene. Errors in the GAA gene cause glycogen storage disease type II, or Pompe disease. In the study, the PRV-3279 surrogate reduced anti-AAV9 vector antibody levels in a dose-dependent fashion.
Redhill Biopharma Ltd., of Tel Aviv, Israel, said it inked an agreement with Cosmo Pharmaceuticals NV, of Dublin, to expand manufacturing capacity for Yeliva (opaganib), Redhill’s sphingosine kinase-2 inhibitor, to address potential demand in the event of global emergency use authorizations. A third interim independent data and safety monitoring board futility analysis is expected in the coming days in the global phase II/III study of opaganib in severe COVID-19 pneumonia, assessing data from the first 135 participants to reach the primary endpoint of the proportion who required intubation and mechanical ventilation by day 14 vs. placebo.
Therapix Biosciences Ltd., of Tel Aviv, Israel, said it changed its corporate name to Scisparc Ltd., with the change effected by the Israeli Registrar of Companies.
Valneva SE, of Saint-Herblain, France, said it began production of its inactivated, adjuvanted COVID-19 vaccine candidate, VLA-2001, in parallel with ongoing studies to accelerate the timeline for potential vaccine deliveries. The phase I/II study, which has fully enrolled 150 healthy adults, is expected to report initial results in April 2021.