Amryt Pharma Holdings Ltd., of Dublin, said the NICE approved Myalepta (metreleptin) for reimbursement in England and Wales as an adjunct to diet as replacement therapy to treat leptin deficiency in congenital or acquired general lipodystrophy in adults and children two years and older and in familial or acquired partial lipodystrophy in adults and children 12 years and older. Amryt gained the recombinant form of the human hormone leptin in its 2019 acquisition of Aegerion Pharmaceuticals Inc.
Aruvant Sciences Holdings Ltd., of London, said it selected Lonza Ltd., of Basel, Switzerland, to help develop and manufacture sickle cell gene therapy candidate ARU-1801 for an upcoming pivotal trial. Under Aruvant's direction, Lonza is beginning process development and technology transfer activities from its Houston center of excellence. Once complete, Lonza will deliver cGMP material supply for the trial.
Brainstorm Cell Therapeutics Inc., of New York, said a preclinical study published in Stem Cell and Research Therapy showed that intratracheal administration of Nurown (neurotrophic factor-producing mesenchymal stem cells)-derived exosomes in a mouse model of acute respiratory distress syndrome (ARDS) led to a statistically significant reduction in lung disease severity score (p < 0.05) based on criteria established by the American Thoracic Society, along with improvements in clinically relevant lipopolysaccharide-induced ARDS markers such as lung function, fibrin presence, neutrophil accumulation, cytokine expression and blood oxygenation levels. The improvements were deemed superior to those observed following administration of naïve mesenchymal stem cell-derived exosomes.
Bridgebio Pharma Inc., of Palo Alto, Calif., and Eidos Therapeutics Inc., of San Francisco, said their stockholders approved Bridgebio’s acquisition of outstanding Eidos shares that Bridgebio – which launched Eidos – does not already own. Terms call for holders of Eidos common shares to receive, for each share, either 1.85 common shares of Bridgebio or $73.26 in cash, prorated to ensure that total cash payable in the merger does not exceed $175 million. The merger, designed to provide greater resources to advance acoramidis, a transthyretin modulator in development by Eidos to treat transthyretin amyloidosis, is expected to close by Jan. 26. Bridgebio’s shares (NASDAQ:BBIO) lost $1.67 on Jan. 20 to close at $67.71 while Eidos (NASDAQ:EIDX) finished at $125.07 for a loss of $3.07.
Gene therapy platform company Dinaqor Ltd., of Pfäffikon, Switzerland, said it acquired EHT Technologies GmbH, of Hamburg, whose engineered heart tissue technology platform is based on research on human induced pluripotent stem cells at the University Medical Center Hamburg-Eppendorf. Financial terms were not disclosed.
Gritstone Oncology Inc., of Emeryville, Calif., said it reached agreement with privately held Genevant Sciences Ltd., of Vancouver, British Columbia, for a nonexclusive license to Genevant’s lipid nanoparticle (LNP) technology to develop and commercialize self-amplifying (SAM) RNA vaccines against SARS-CoV-2. Genevant’s LNP platform is part of Gritstone’s SAM neoantigen-based cancer immunotherapy, which is in phase II testing. Genevant is eligible to receive up to $192 million in up-front and contingent milestone payments per product plus royalties ranging from the mid-single to mid-double digits. Should Gritstone outlicense the COVID-19 program, Genevant may be entitled to a percentage of amounts paid to Gritstone. On Jan. 20, Gritstone (NASDAQ:GRTS) shares spiked 14.5%, or $3.22, to finish at $25.49.
Hoth Therapeutics Inc., of New York, said in vitro data showed the therapeutic potential of its Toll-like receptor 2 (TLR2) agent, HT-003, to block acne pathogenic gene expression in human keratinocytes, the major cell type of the epidermis and the outermost of the layers of the skin. Data generated from a series of experiments showed TLR2 was significantly inhibited by HT-003, even in the presence of TLR2 agonists. Baseline expression of TLR2 and of NF-KB, a transcription factor for skin inflammation, also were inhibited by HT-003.
Kiromic Biopharma Inc., of Houston, said it completed and certified its GMP facility in Houston to meet FDA regulatory guidelines to support the first-in-human ovarian cancer trial of its allogenic CAR-T candidate, which targets chPD1 gamma-delta and anti-ISOMSLN. The company is conducting gamma-delta-T cell GMP manufacturing test batch optimization and qualification studies.
Contract research and drug discovery organization Metrion Biosciences Ltd., of Cambridge, U.K., and the medical research charity Lifearc said they agreed to a second 12-month extension of their neuroscience drug discovery collaboration. The program, which has progressed to hit-to-lead optimization, is focused on discovery of selective small molecular modulators of a specific two-pore domain potassium ion channel target thought to be involved in neurological pathogenesis. Metrion is providing validated ion channel and electrophysiology-based assays and safety profiling services while Lifearc is identifying modulators of central nervous system ion channel targets, with Metrion then evaluating the pharmacological activities of these compounds.
Neuronascent Inc., of Clarksville, Md., said preclinical findings showed its glycogen synthase kinase-3 beta modulator, NNI-362, allosterically stimulated neural regeneration in human cultures and in vivo models of aging and disease. The short report in Stem Cell Research & Therapy showed that NNI-362’s mechanism of action reversed neural cell formation and survival deficit in the hippocampus, with allosteric stimulation of the pleiotropic kinase, p70S6 kinase, by NNI-362 appearing to promulgate regenerative capacity in a degeneration model and even under extreme aging.
Neuvogen Inc., of San Diego, said it was formed to pursue whole cell cancer vaccine technology targeting solid tumors by maximizing immune priming against an array of tumor targets combined into a single platform. The company’s platform directly addresses key limitations of cancer vaccine development, including inadequate tumor target breadth, inadequate immune priming and inadequate focus on overcoming immune suppressive mechanisms in the tumor microenvironment. Neuvogen aims to employ a combination of genetic modifications to each of six whole cell tumor lines in each of its vaccines to create a robust immune priming adjuvant effect that may overcome peripheral tolerance to self-antigens and to prime against an array of targets in patient populations where cancer vaccine monotherapy showed efficacy signals in large studies.
Oncorus Inc., of Cambridge, Mass., said preclinical data on its lead oncolytic herpes simplex virus (HSV) candidate, ONCR-177, published online in Cancer Immunology Research, showed potent and durable antitumor activity in multiple immune-competent tumor models. The activity of ONCR-177 was shown to be driven by the combination of five complementary immunomodulatory transgene payloads in addition to its retention of gamma-34.5, an HSV-1 gene that allows the virus to replicate in the presence of host antiviral immune responses.
Relief Therapeutics Holding AG, of Geneva, Switzerland, and Advita Lifescience GmbH, of Gundelfingen, Germany, signed a binding term sheet for Relief to acquire all shares of Advita in exchange for €25 million (US$30.2 million) of Relief common shares, plus possible future contingent milestone payments of up to €20 million. The is expected to occur in the second quarter of this year. Under the terms, Relief will advance a €2 million convertible secured loan to AdVita. The first tranche of €1 million will be advanced concurrent with the execution of the binding term sheet and the second tranche of the same amount upon the successful conclusion of Relief's phase IIb/III clinical trial of intravenous RLF-100 in the treatment of critical COVID-19 patients with respiratory failure. Top-line data from this trial are expected at the end of January or early February of this year.
Satellos Bioscience Inc., of Hackensack, N.J., was the subject of a $1 million investment by Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne muscular dystrophy (DMD). The money will support the development of a new regenerative medicine for the therapeutic treatment of DMD. Satellos' technology is based on the discovery of what controls muscle stem cell differentiation, the company said.
Sensorion SAS, of Montpellier, France, highlighted preclinical data showing that the combination of its SENS-401 molecule with cochlear implants reduced loss of residual hearing at a frequency located beyond the electrode array. SENS-401 also showed a statistically significant electrode impedance reduction. The impedance is associated with electrode array insertion trauma and increased foreign body response around the electrode array, the company noted.
Sorrento Therapeutics Inc., of San Diego, is presenting preliminary results from an ongoing SARS-CoV-2 mutation surveillance program for its neutralizing antibodies currently in clinical and preclinical development for treatment of patients with COVID-19 disease. Findings will be disclosed from the SARS-CoV-2 Spike protein variant screening program at PepTalk 2021. Data will provide evidence of maintained binding potency by STI-2020 in in vitro assays including the spike amino acid changes found in SARS-CoV-2 viruses of the B.1.1.7 lineage initially identified in the U.K., which has since been detected in 10 U.S states. The discovery might signify that the STI-2020 antibody currently in clinical trials is not anticipated to behave differently against the new virus variant, the company said.
T-Cure Bioscience Inc., of Sherman Oaks, Calif., and Immunotech Biopharm Ltd., of Beijing, signed a license agreement for the research and development of T-Cure's 800TCR product candidate, a HERV-E targeting T-cell receptor therapy for renal cell cancer, in China. Under the terms of the agreement, Immunotech will make an upfront payment with T-Cure being eligible to receive additional future development milestone payments and tiered royalties on net sales of the product. Details were not disclosed. T-Cure is currently developing 800TCR in the U.S. in collaboration with the National Heart Lung and Blood Institute.
Therapeutic Solutions International Inc., of Oceanside, Calif., reported positive animal data in the 4T1 breast cancer model using its clinically tested Stemvacs cellular immunotherapy. In a series of experiments, it was demonstrated that Stemvacs administration resulted in regression of established breast cancer in mice, that regression was dependent on natural killer cells, and that immunity to breast cancer could be transferred by CD4 T cells to naïve mice. Previously the company disclosed positive safety data in 10 patients treated with Stemvacs.
Turnstone Biologics Corp., of New York, acquired Myst Therapeutics Inc., of Los Angeles, a privately held biotechnology company focused on advancing novel T-cell therapies for solid tumors. The acquisition adds a pipeline of tumor-infiltrating lymphocyte programs to Turnstone’s portfolio of oncolytic virus candidates. Financial terms were not disclosed.
Viewpoint Molecular Targeting Inc., of Coralville, Iowa, was awarded a total of $5 million in phase II grants from the National Cancer Institute under the SBIR program and the NIH. The grants will be used to advance the company’s image-guided alpha-particle radiotherapies. Viewpoint's VMT-01 program is intended to meet an unmet clinical need with the use of a new imaging agent to guide Viewpoint's radiopharmaceutical therapy against metastatic melanoma, the firm said.