9 Meters Biopharma Inc., of Raleigh, N.C., said it will enter a collaboration with the Duke Clinical Research Institute to support clinical development of the company’s NM-002, a long-acting GLP-1 agonist, currently in phase II development for short bowel syndrome.
Akers Biosciences Inc., of Baltimore, and Mymd Pharmaceuticals Inc., also of Baltimore, said Akers obtained sufficient votes during its stockholder meeting to consummate the proposed merger between the two firms. Upon closing, the combined firm will be named Mymd Pharmaceuticals Inc. and will remain listed on Nasdaq under the new ticker MYMD, starting April 19. The company plans to focus on development and commercialization of immunotherapy assets, including MYMD-1, a drug aimed at treating autoimmune and age-related diseases, including extending the human lifespan.
Ampio Pharmaceuticals Inc., of Englewood, Colo., reported preclinical findings that show Ampio’s lead candidate, Ampion, provides some of the same anti-inflammatory effects as the steroid dexamethasone but without the significant adverse events associated with steroids. Data showed Ampion regulated 102 of the same transcripts as dexamethasone, according to RNA sequencing and differential gene expression analysis, while Ingenuity Pathway Analysis modeling predicted significantly similar regulation of downstream molecules by dexamethasone and Ampion based on directionally correlated transcripts. However, there were 30 transcripts where Ampion's activity was different than known regulation by dexamethasone – up-regulating when dexamethasone was down, or vice versa – in at least one of the treatment conditions. Dexamethasone also targets a wide range of molecules that are not regulated by Ampion.
Applied Genetic Technologies Corp., of Alachua, Fla., said it entered a licensing agreement that provides its cone specific promoter technology to Sparingvision SAS, of Paris, a genomic medicine company developing vision-saving treatments for ocular diseases. AGTC’s PR1.7 cone specific receptor promoter is designed to help drive increased gene expression in cone photoreceptors only, thereby allowing enhanced targeting of gene therapies for indications in which the gene defect is cone specific and limiting expression of the gene in other cells that could be undesirable. Under the terms, Sparingvision gets nonexclusive rights for use of the promoter in development of two noncompeting products, with an opportunity to obtain rights for one additional product in the future. AGTC will receive an undisclosed up-front fee and be eligible to receive milestone payments and royalties on a per-product basis.
Catalyst Biosciences Inc., of South San Francisco, said preclinical data published in Blood from its study in hemophilia B mice demonstrated superiority of the company's CB 2679d-GT gene therapy candidate over that of the R338L-Padua variant, which is currently used in clinical trials. Results showed CB 2679d-GT safely and effectively achieved sustained factor IX levels and significantly outperformed the R338L-Padua variant. A significantly reduced bleeding time and total blood loss with CB 2679d-GT gene therapy compared to mice treated with R338L-Padua demonstrated a more rapid and robust hemostatic correction.
Citrine Medicine Co. Ltd., of Shanghai, and Sinopharm Group, of Beijing, said they partnered to accelerate the commercialization of rare disease drugs in China. The two firms will work together to build a rare disease ecosystem in China and enable the availability of more rare disease therapies to Chinese patients in need. As part of the collaboration, Sinopharm will support Citrine in building a supply chain and securing distribution channels for delivering rare disease therapies.
Hyundai Bioscience Co. Ltd., of Seoul, South Korea, said its major shareholder biotech company, Cnpharm, reported preclinical data showing its COVID-19 candidate, Poly-COV01, could sustain an effective concentration in the blood that inhibits virus activity by 100% (IC100) for more than 24 hours. In an experiment conducted on animals at Knotus, its nonclinical CRO, when 2 mg/kg of Poly-COV01 was administered, the concentration of IC100 or higher was maintained in the blood for more than 24 hours, and it was confirmed that the drug in the delivery system was continuously released during that time.
Inflammasome Therapeutics Inc., of Newton, Mass., said research published in Signal Transduction and Targeted Therapy continues to confirm the potential of its Kamuvudines to be an effective treatment for geographic atrophy (GA), a severe form of age-related macular degeneration (AMD). Researchers found that deposits of amyloid beta protein in the retina of AMD patients trigger a part of the immune system, leading to death of cells that make up one of the inner layers of the eye. When those cells die, so does the overlying retina and vision is lost. The investigators also found that a class of existing AIDS drugs, nucleoside reverse transcriptase inhibitors and safer derivatives of those drugs (Kamuvudines) halt inflammasome activation and prevent cell death.
Kazia Therapeutics Ltd., of Sydney, said it entered a worldwide exclusive licensing agreement and a master services agreement with Evotec SE, of Hamburg, Germany, for EVT-801, a small-molecule oncology drug candidate. Kazia expects to launch a phase I trial of EVT-801 in 2021. Under the terms, Kazia gets exclusive global rights in exchange for an up-front fee of €1 million (US$1.2 million), contingent milestones of up to €308 million related to achievement of clinical, regulatory and commercial outcomes over the lifetime of the drug, and a tiered single-digit royalty on net sales.
Krystal Biotech Inc., of Pittsburgh, reported results from IND-enabling GLP toxicology study of KB-407, an inhaled gene therapy candidate for the treatment of cystic fibrosis (CF), in nonhuman primates. Repeat doses were well-tolerated, and the no-observed-adverse-effect level was at the highest dose tested. KB-407 was distributed throughout the lung tissue, including the bronchioles and alveoli, with little-to-no vector detected in all other tissues and fluids tested. A phase I trial is expected to begin in the third quarter of 2021. Krystal also reported initial preclinical proof-of-concept data for its second genetic pulmonary disease candidate, KB-408, for the treatment of alpha-1 antitrypsin deficiency (AATD). KB-408, an inhaled (nebulized) formulation of a vector designed to deliver two copies of the SERPINA1 gene, successfully transduced primary airway epithelial cells in vitro, leading to production and secretion of full length, normal human AAT protein. In healthy immunocompetent mice administered a single dose of KB-408 or vehicle control to the airways, analyses of lung tissue samples 24 hours post-dose show efficient vector transduction and human AAT transgene expression. An IND package for KB-408 has been submitted to the FDA.
Neurona Therapeutics Inc., of San Francisco, presented preclinical data from its lead inhibitory neuron cell therapy program, which is being developed for the treatment of drug-resistant focal epilepsy, demonstrating that after a single administration in a rodent model of drug-resistant temporal lobe epilepsy, the injected neuronal cells exhibited long-term persistence in the target tissue, reduced temporal lobe pathology, and resulted in robust and reproducible seizure suppression.
Oblique Therapeutics AB, of Gothenburg, Sweden, in collaboration with Karolinska Institutet, Gothenburg University and several local biotechs, published results in Science Advances describing in how Oblique’s Abiprot technology can be used to discover and develop pharmacologically tailored antibodies against clinically important targets widely considered undruggable with antibodies. Two example antibodies presented in the article involve targeting hTRPV1, a clinically validated pain target, and KRAS, a highly relevant oncogene of critical importance in the etiology of many aggressive cancers.
Ovid Therapeutics Inc., of New York, said it will discontinue development of OV-101 (gaboxadol), a delta-selective GABAA receptor agonist, in Angelman syndrome. The company also said it does not plan to initiate further clinical studies of OV-101 in fragile X syndrome. The news follows a phase III failure of OV-101 in Angelman syndrome late last year. Ovid will prioritize its resources to focus on the development of its early stage pipeline, including OV-882, a short hairpin RNA therapy targeting UBE3A gene expression in neurons, as a potential treatment for Angelman syndrome. Following those updates, Ovid is reiterating its previous guidance of anticipated quarterly operating expenses, excluding noncash expenses, to be in the range of $8 million to $10 million starting in the second quarter of 2021.
Phico Therapeutics Ltd., of Cambridge, U.K., said it was awarded a grant of up to $18.2 million from Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator (CARB-X), a global nonprofit partnership dedicated to accelerating antibacterial research to tackle the global rising threat of drug-resistant bacteria. The funding will support the progression of Phico’s lead product, Saspject PT3.9, through clinical trials, with $5.3 million available immediately and a further $12.9 million contingent on reaching specific project milestones.
Portage Biotech Inc., of Westport, Conn., said it plans to voluntarily delist its common stock from the Canadian Securities Exchange. Shares will continue trading on Nasdaq under the ticker PRTG.
Saniona AB, of Copenhagen, Denmark, said it entered a partnership with the Foundation for Prader-Willi Research to increase awareness of Saniona’s phase IIb trial of Tesomet for treating Prader-Willi syndrome (PWS). Saniona said it expects to initiate the study in the first half of 2021. Saniona evaluated Tesomet in a randomized, double-blind, placebo-controlled phase IIa trial in adults and adolescents with PWS. Adult patients receiving Tesomet achieved a statistically significant reduction in hyperphagia, uncontrollable hunger, as well as a clinically meaningful reduction in body weight at a dose of 0.5 mg per day. A smaller study extension in an adolescent population showed that Tesomet appeared to be well-tolerated at lower doses (0.125 mg/day and 0.25 mg/day) and suggested dose-dependent effects on weight and hyperphagia. Tesomet is an investigational fixed-dose combination therapy of tesofensine, a triple monoamine reuptake inhibitor and metoprolol, a beta-1 selective blocker.
Sanofi SA, of Paris, said it completed its $358 million acquisition of Amsterdam-based Kiadis Pharma NV, which is developing off-the-shelf natural killer cell therapies. The platform has the potential to make products rapidly and economically available for a broad patient population across a wide range of liquid and solid tumors, according to Sanofi, and create synergies with its immuno-oncology pipeline. The cell-based medicines will be developed alone and in combination with Sanofi’s existing pipeline and platforms.
Sunshine Biopharma Inc., of Montreal, said it completed a maximum tolerated dose study of its PLpro inhibitor in mice The results fall within the optimum range for use in humans, the company said. Next, Sunshine Biopharma said it will use the test dose indicated by those results to conduct the efficacy studies in hACE2-transgenic mice. PLpro is a protease present only in SARS coronaviruses and is involved in shutting down the host innate immune system, which leads to greater morbidity.
New data from Therapeutic Solutions International Inc., of Elk City, Idaho, implicated a key role in the protein brain-derived neurotrophic factor (BDNF) in the therapeutic activities of Narcostem, an umbilical cord blood-derived biologic demonstrated to possess positive animal data in preclinical models of addiction and drug-induced brain damaged. BDNF is considered a master molecule in controlling brain function and has been reported by numerous groups to associate with efficacy of antidepressants, as well as the ability to overcome addictive tendencies, the company said. It is almost impossible to provide recombinant BDNF directly into the brain due to its size and half-life, the company added. Accordingly, preliminary data suggest that administration of Narcostem can directly stimulate the brain to produce its own BDNF, the company added.
Tonix Pharmaceuticals Holding Corp., of Chatham, N.J., said it entered an exclusive worldwide licensing agreement for an antiviral inhibitor of SARS-CoV-2, TNX-3500 (sangivamycin, formerly OYA1), for treating COVID-19 and potentially other viral disorders. The active ingredient of TNX-3500 has been studied for safety in humans in prior studies on cancer patients at the U.S. National Cancer Institute. TNX-3500 has shown strong dose-dependent antiviral activity against live SARS-CoV-2 virus in cell culture infectivity studies, the company said. TNX-3500 was demonstrated to be approximately 65 times more potent in head-to-head comparisons at inhibiting SARS-CoV-2 than remdesivir, the active ingredient of Veklury (Gilead Sciences Inc.). In addition, combining TNX-3500 and remdesivir has demonstrated additive activity against SARS-CoV-2 in cell culture infectivity studies, the company said.
Valo Therapeutics Ltd., of Helsinki, Finland, said it acquired intellectual property rights for the Peptibac technology from the University of Helsinki. Peptibac uses the Bacillus Calmette-Guèrin (BCG) vaccine as a backbone for delivery of disease-specific peptides. The technology has preventive potential in infectious diseases, specifically tuberculosis as well as therapeutic potential in cancer, according to Valo. The BGC vaccine is safe and well-tolerated, with more than 100 million doses dispensed a year, Valo said. Peptibac is designed to drive a T-cell immune response against new strains of tuberculosis by targeting multiple disease-specific antigens simultaneously.
New research from Viewpoint Molecular Targeting Inc., of Coralville, Iowa, demonstrated that the combination of VMT-01 and immune checkpoint inhibitors induced synergistic antitumor effect and 43% complete tumor responses. The company said it plans to launch a phase I study, with results expected in the fourth quarter of 2021, followed by a phase I/IIa study for treating metastatic melanoma. VMT-01 is a specialized peptide designed to target the melanocortin 1 receptor (MC1R) on tumor cells. The results from the preclinical study indicate that MC1R-targeted therapies, such as peptide receptor radionuclide therapies, are a promising alternative to current therapies for metastatic melanoma, the company added.
Results of an animal study from Virpax Pharmaceuticals Inc., of Berwyn, Pa., showed that MMS-019, its antiviral product candidate for treating respiratory viruses, inhibited viral replication in the nasal passages. MMS-019 is high-density molecular masking spray the company is developing as an antiviral barrier. Virpax said it intends for the formulation to be delivered using a preassembled device and cartridge to propel the formulation into the nose. The animal study model included transgenic mice expressing the human ACE2 protein under the human cytokeratin 18 promoter, a type of protein found on epithelial cells, inside and outside of the body. Each experimental group consisted of 10 animals with 14 animals in the control group. MMS-019 was administered once daily intranasally in the treatment group, and the control group received remdesivir intramuscularly. Initially, the animals were infected intranasally with the SARS-CoV-2 virus.