Applied Genetic Technologies Corp., of Gainesville, Fla., said preclinical data validating the transgene (hRPGRco) that is being evaluated in the company’s ongoing phase I/II clinical trial in patients with X-linked retinitis pigmentosa (XLRP) have been published in Human Gene Therapy. The studies, which evaluated the safety and efficacy of hRPGRco and another XLRP transgene in a canine model of XLRP, demonstrated stronger expression of hRPGRco than the other transgene at all dose levels evaluated. Following subretinal administration in the company’s rAAV2tYF vector, each of the XLRP transgenes corrected rod-cone opsin mislocalization, which are early markers of the disease, but the hRPGRco transgene demonstrated a broader therapeutic index.
Altimmune Inc., of Gaithersburg, Md., disclosed additional positive results from the preclinical studies of its single-dose intranasal COVID-19 vaccine candidate, AdCOVID. The studies were conducted as part of Altimmune’s ongoing collaboration with the University of Alabama at Birmingham. The latest study showed potent stimulation of antigen-specific CD4+ and CD8+ T cells in the lungs of CD-1 mice as early as 10 days following a single intranasal vaccination, with responses strongly biased toward CD8+ T cells, the company said.
Asalyxa Bio Inc., of Ann Arbor, Mich., a spinout from the University of Michigan focused on severe neutrophil-mediated diseases, disclosed its formation and seed funding to accelerate ASX-100 into first-in-human trials in 2021. The company was co-founded by Orange Grove Bio (OGB) a New York-based venture operating company and leading scientists at the university. Marc Appel, CEO of OGB, will serve as the CEO of Asalyxa, with Lola Eniola-Adefeso, a Michigan professor, serving as chief scientific officer. ASX-100 is a spherical polymer particle that degrades into salicylic acid – an active metabolite of aspirin – and a food additive that is listed as generally regarded as safe by the FDA.
Bioage Labs Inc., of Richmond, Calif., in-licensed a clinical-stage therapy with potential for treating immune aging in older patients hospitalized with COVID-19. The compound, BGE-175, is an orally administered inhibitor of the prostaglandin D2 DP1 signaling pathway associated with increased risk of mortality, and susceptibility to infections. The company recently generated preclinical data showing significant immune-modulating and anti-viral activity of BGE-175 which resulted in 100% survival in a preclinical model of the SARS 1 virus.
Brainstorm Cell Therapeutics Inc., of New York, reported data to be presented at the MSVirtual2020 meeting from a subset of patients in the observational Climb study comparing magnetic resonance imaging (MRI) results and measures of functional improvement. MRI measurements of whole brain volume (p=0.03) and gray matter volume (p=0.063) at follow-up correlated with results from the 9-hole peg functional test. Deep white matter FLAIR/T2 lesion volume at baseline (p=0.03) and follow-up (p=0.02) correlated with timed-25-foot-walk scores. Brainstorm is using MRIs in its ongoing phase II study of Nurown (MSC-NTF cells) in patients with progressive MS.
Cabaletta Bio Inc., of Philadelphia, disclosed that preclinical study results evaluating DSG3-CAART (desmoglein 3 chimeric autoantibody receptor T cells), its lead product candidate for patients with mucosal pemphigus vulgaris, were published in The Journal of Clinical Investigation. The manuscript, titled “Antigen-specific B-cell depletion for precision therapy of mucosal pemphigus vulgaris,” includes the preclinical data that enabled the DSG3-CAART IND submission and opening of the DesCAARTes clinical trial. The preclinical studies, conducted at the Perelman School of Medicine at the University of Pennsylvania and led by the laboratory of Aimee Payne, assessed the potential toxicity and specific target engagement of DSG3-CAART in inducing antigen-specific B cell depletion.
Carisma Therapeutics Inc., of Philadelphia, signed a scientific research and licensing agreement with Nathaniel Landau and NYU Langone Health through which Carisma will gain exclusive rights to develop and commercialize their Vpx lentiviral vector globally for all indications. The Vpx lentiviral and Carisma's Ad5f35 vectors are the only two vectors known to be effective in engineering macrophages, a pivotal aspect of Carisma's approach, the company said. The Vpx lentiviral vector, proven highly effective at transducing human monocytes, macrophages and dendritic cells, is expected to broaden the utility of Carisma’s platforms by enabling integration of transgenes for long-term, durable expression.
Contrafect Corp., of Yonkers, N.Y., said it inked an initial funding agreement with the Cystic Fibrosis Foundation to investigate the utility of direct lytic agents (DLAs), including lysins and amurin peptides, against resistant gram-negative pathogens that infect individuals with cystic fibrosis (CF). The first stage of the agreement will provide funding to assess the in vitro activity of CF-370, Contrafect’s engineered lysin targeting Pseudomonas aeruginosa, and amurin peptides against bacterial specimens obtained from CF patients at different stages of disease. With supportive data, Contrafect plans to evaluate development of CF-370 and/or amurin peptides as therapeutic candidates to treat exacerbations in CF lung disease.
Cyclerion Therapeutics Inc., of Cambridge, Mass., said it was awarded a grant from the Alzheimer's Association’s Part the Cloud-Gates Partnership Grant Program to support the planned phase II trial of guanylate cyclase stimulator IW-6463 in Alzheimer’s disease with vascular pathology. The award provides Cyclerion with $2 million in funding over two years.
Elixiron Immunotherapeutics Inc., of Taipei, Taiwan, said it was awarded a $1 million grant from the Alzheimer's Association under the 2020 Part the Cloud-Gates Partnership Grant Program to support a U.S. phase I trial of lead candidate, EI-1071, an oral colony-stimulating factor 1 receptor inhibitor. The company aims to treat Alzheimer's disease by targeting microglia-mediated neuroinflammation.
Intravacc, of Bilthoven, the Netherlands, said it partnered with Buffalo, N.Y.-based Therapyx Inc. to optimize Ngoxim, a prophylactic vaccine against gonorrhea, after Therapyx received a $2.8 million SBIR phase IIB grant to advance the candidate. Ngoxim is a microsphere vaccine with encapsulated interleukin-12 and outer bacterial membrane vesicles from Neisseria gonorrhoeae, formulated for mucosal immunization. The partners will seek to enhance the vaccine’s adaptive immune response for long-term protection against the gonorrhea bacteria in non-human primates prior to testing in humans.
Mediwound Ltd., of Yavne, Israel, and U.S. commercial partner Vericel Corp., of Cambridge, Mass., said the Biomedical Advanced Research and Development Authority (BARDA) accepted the first shipment of the wound healing agent Nexobrid (bromelain gel) under its national emergency response preparedness mission. The initial BARDA procurement of the endopeptidase stimulator is valued at $16.5 million, which includes quarterly deliveries through the end of 2021. BARDA holds an option to procure additional quantities of Nexobrid through funding of up to $50 million. On June 30, 2020, the companies submitted a BLA to the FDA seeking the approval of Nexobrid for debridement in adults with deep partial-thickness and/or full-thickness thermal burns. While the BLA is under review, burn centers across the U.S. are treating patients under the Nexobrid expanded access protocol.
Morphic Therapeutic Inc., of Waltham, Mass., said Abbvie Inc., of North Chicago, exercised a license option under their R&D collaboration to develop Morphic’s alpha-V/beta-6 integrin inhibitors to treat fibrotic diseases, including idiopathic pulmonary fibrosis (IPF). The license covers inhibitors discovered from Morphic’s Mint platform, including MORF-720 and MORF-627, which Morphic advanced through preclinical studies. The option exercise triggered a $20 million payment to Morphic, which remains eligible for development milestone payments and sales royalties under the agreement with Abbvie, which now assumes responsibility for development and commercialization.
Otsuka America Pharmaceutical Inc., a unit of Otsuka Pharmaceutical Company Ltd., of Tokyo, said it purchased the assets of Proteus Digital Health Inc., of Redwood City, Calif., including material goods and intellectual property for its ingestible and wearable sensor technology, in a bid to strengthen its R&D efforts and implement the next phase of its digital medicine and digital therapeutics programs. Financial details of the transaction were not disclosed. Otsuka was an early partner of Proteus for digital medication adherence monitoring with its drug-device combination of Abilify Mycite and an ingestible sensor.
Oxford Biomedical Research Centre, of Oxford, U.K., published a retrospective study in Lancet Rheumatology on the safety of hydroxychloroquine and azithromycin in patients with rheumatoid arthritis. Using health records from 956,374 users of hydroxychloroquine, 310,350 users of sulfasalazine and 323,122 users of hydroxychloroquine plus azithromycin, researchers found long term use of hydroxychloroquine had a 65% relative increase in cardiovascular-related mortality compared to sulfasalazine. Hydroxychloroquine plus azithromycin had a cardiovascular mortality risk that was more than twice as high as the comparative treatment, even on a short-term basis. The combination also increased the rate of angina/chest pain and heart failure by 15% to 20%.
Sinovac Biotech Ltd., of Beijing, said it signed two agreements in Hainan, China, with PT Bio Farma, of Bandung, West Java, Indonesia, covering the supply, local production and technology licensing of Coronavac, Sinovac’s inactivated vaccine candidate against COVID-19. Sinovac committed to supply Bio Farma bulk vaccine to produce at least 40 million doses in Indonesia prior to March 2021. After that time, Sinovac will supply needed quantity of the bulk vaccine until year-end 2021. Bio Farma is currently conducting the phase III trial of Coronavac in Bandung, Indonesia.
Starpharma Holdings Ltd., of Melbourne, Australia, said it reformulated the hyaluronidase inhibitor SPL-7013 (astodrimer), which showed activity against COVID-19 infection, into several nasal spray formulations; identified a manufacturer and device components; instituted pilot manufacturing; and compiled regulatory documentation in preparation for submission. Based on its discussions with regulators, the company expects to expedite review of the nasal spray by leveraging existing nonclinical and clinical data from approved and marketed antiviral dendrimer products, which include SPL-7013 as the active ingredient.
Sutro Biopharma Inc., of South San Francisco, said that it achieved a clinical supply milestone under its collaboration and license agreement with the health care division of Merck KGaA, of Darmstadt, Germany, which entitled Sutro to a milestone payment. The amount was not disclosed. The candidate, M-1231, a MUC1-EGFR bispecific antibody drug conjugate targeting solid tumors, was discovered using Sutro's Xpresscf and Xpresscf+ discovery and manufacturing technologies and includes a linker-warhead discovered by Sutro.
Xphyto Therapeutics Corp., of Vancouver, British Columbia, said its wholly owned German subsidiary, Vektor Pharma TF GmbH, completed process implementation for the manufacture of its transdermal patch for rotigotine, a non-ergoline dopamine agonist approved in the U.S. and Europe to treat Parkinson's disease and restless legs syndrome. Xphyto said clinical sample manufacturing and analytical work will begin in Vektor's EU GMP laboratory and manufacturing facility in preparation for human bioavailability studies in Europe, which are planned for the third quarter of 2020. Results of those studies are expected in the fourth quarter of 2020.