Abionyx Pharma SA, of Toulouse, France, reported that results from a named patient temporary authorization for use of CER-001 by the French Drug Safety Agency were published in Annals of Internal Medicine. The patient, who had inherited mutations in the lecithin-cholesterol acyltransferase gene, developed glomerulopathy and corneal lipid deposits and displayed low circulating levels of high-density lipoprotein and apolipoprotein A1 (apoA-I). Despite rapid decline in renal function, he was able to avoid dialysis during treatment with CER-001, an apoA-I agonist. Although eGFR decreased from 41 to 19 mL/min/1.73 m2 during the nine months that preceded the start of CER-001, eGFR only decreased from 19 to 17 mL/min/1.73 m2 during the 11 months following introduction of treatment. Urinary protein-to-creatinine ratio decreased from 7 g/g to 0.25 g/g at day 10, with undetectable albuminuria at that time, but returned to initial values thereafter. Nephrotic syndrome disappeared, with serum albumin increasing from 29 g/L to 37 g/L during treatment and follow-up. Additionally, the patient’s visual blurring disappeared, with improvement in visual function still observed after one year of follow-up.
Agex Therapeutics Inc., of Alameda, Calif., and Lygenesis Inc., of Pittsburgh, are negotiating to form a subsidiary of Agex to merge into Lygenesis. At closing, Agex would issue Lygenesis stockholders shares of Agex common stock representing two-thirds the total number of shares of Agex common stock. If the merger is completed, Agex’s premerger stockholders would receive an 80% economic interest in Agex’s induced tissue regeneration technology. Terms and structure of such economic interest are undetermined.
Arsanis Inc., of Waltham, Mass., and Adimab LLC, of Lebanon, N.H., signed an agreement under which Arsanis has secured the exclusive, worldwide license to antibodies targeting respiratory syncytial virus (RSV) that were discovered by Adimab. Arsanis will initially focus on the selection of a lead RSV antibody candidate and has received a grant of up to $9.3 million from the Bill & Melinda Gates Foundation to advance the selected antibody to IND filing. Arsanis has exclusively licensed a panel of RSV antibodies for the purpose of evaluating and selecting the best therapeutic leads under an exclusive global development and commercialization license. Adimab will be entitled to receive license fees and development milestones, as well as a royalty on net sales.
Artizan Biosciences Inc., of New Haven, Conn., has raised $11 million in a series A-2 financing and said it will use the proceeds to advance preclinical R&D of its lead program for inflammatory bowel disease (IBD), which is anticipated to enter the clinic in 2022, as well as to explore additional disease targets. The financing was led by existing investor Hatteras Venture Partners and new investor Biohaven Therapeutics Ltd., also of New Haven, Conn. Artizan also entered an option and license agreement with Biohaven to develop and commercialize Artizan’s IBD treatments in the U.S. Biohaven has the rights to exercise an option on up to three IBD product candidates. Artizan is eligible to receive option exercise payments and milestone payments, which are based on the market potential of each product candidate, as well as tiered royalties.
Astrazeneca plc, of Cambridge, U.K., said it has begun supplying its hundreds of millions of doses of its COVID-19 vaccine to low- and middle-income countries, including Ghana and Cote D’Ivoire in Africa. More will soon arrive, the company added, in the Philippines, Indonesia, Fiji, Mongolia and Moldova. The vaccine can be stored, transported and handled at normal refrigerated conditions (2-8 degrees Celsius/36-46 degrees Fahrenheit) for at least six months and administered within existing health care settings.
Awakn Life Sciences Inc., of London, said it has an exclusive license to use and deliver the ketamine used in the Reduction of Alcoholic Relapse (KARE) psychotherapy intervention at the University of Exeter. In a four-armed, placebo-controlled phase II trial assessing ketamine combined with the KARE psychotherapy in the treatment for alcohol use disorder, the primary endpoints were percentage days abstinent and relapse at six months, with secondary endpoints including depressive symptoms, craving and quality of life. Awakn is studying psychedelic medicine to treat addiction and other mental health conditions.
Cynata Therapeutics Ltd., of Melbourne, Australia, said it initiated a new study to investigate in a preclinical model of lung disease the potential molecular mechanisms involved in the observed high potency of Cynata’s Cymerus mesenchymal stem cells (MSCs). The model mimics features of idiopathic pulmonary fibrosis. The company said the study should last six months. Cymerus uses induced pluripotent stem cells and a precursor cell known as mesenchymoangioblast to achieve economic manufacture of cell therapy products, including MSCs, at commercial scale without the limitation of multiple donors, the company said.
Fusion Pharmaceuticals Inc., of Hamilton, Ontario, and Boston, said it entered an asset purchase agreement (APA) to acquire Paris-based Ipsen SA's intellectual property and assets related to IPN-1087, a small molecule targeting neurotensin receptor 1 (NTSR1), a protein expressed on multiple solid tumor types. Fusion said it intends to use IPN-1087 to create an alpha-emitting radiopharmaceutical, FPI-2059, targeting solid tumors expressing NTSR1. Fusion will issue Ipsen 400,000 shares of common stock upon closing and another 200,000 shares on achieving a patent-related milestone. Fusion will also be obligated to pay Ipsen up to an additional €67.5 million (US$81.59 million) upon achieving certain development and regulatory milestones, low single-digit royalties on potential future net sales and up to €350 million in net sales milestones, in each case, relating to products covered by the APA. Fusion will be responsible for paying to a third-party licensor up to €70 million in development milestone payments and mid-single to low-double-digit royalties on potential future net sales of products covered by the license agreement.
Iktos SAS, of Paris, said it will use its artificial intelligence technology for de novo design in selected small-molecule discovery programs with Pfizer Inc., of New York. Iktos develops artificial intelligence solutions applied to chemical research, more specifically medicinal chemistry and new drug design. Iktos’ deep learning generative models enables the design of molecules that are optimized in silico.
Innovation Pharmaceuticals Inc. (formerly Cellceutix Corp.), of Wakefield, Mass., said a peer-reviewed article published in Viruses showed that brilacidin exerted inhibitory effects on SARS-CoV-2 in cell culture by decreasing viral load in different cell types, including ACE2-positive human lung cells, and in both the Washington and Italian strains of the coronavirus. The dual PDE3/PDE4 inhibitor is being assessed in a global phase II trial in people hospitalized with COVID-19.
Junshi Biosciences Co. Ltd., of Shanghai, and Eli Lilly and Co., of Indianapolis, said the U.S. government agreed to purchase a minimum of 100,000 doses of etesevimab (JS-016 or LY-CoV016) 1,400 mg and bamlanivimab (LY-CoV555) 700 mg for $210 million. The doses will be delivered through March 31, 2021. The government has an option to purchase an additional 1.1 million doses through Nov. 25, 2021, under the same terms as the base agreement and subject to agreement from Lilly and product availability.
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda, said it launched the Resonance registry in pediatric and adult patients with recurrent pericarditis, an autoinflammatory cardiovascular disease. The registry is a voluntary patient database led by physician researchers with experience in managing patients with recurrent pericarditis and was created to increase the understanding of recurrent pericarditis by encouraging the collaboration and sharing of information and expertise.
Lexeo Therapeutics LLC, of New York, said it licensed worldwide intellectual property rights and preclinical data from Adverum Biotechnologies Inc., of Redwood City, Calif., to its Friedreich's ataxia gene therapy program. With exclusive rights to data from seven preclinical studies now combined, Lexeo said it will advance LX-2006 through final IND-enabling studies and into a phase I trial in 2021. LX-2006 is an adeno-associated virus-mediated treatment delivered through intravenous administration to address the Friedreich's ataxia cardiac disease pathology. In other preclinical studies, Lexeo said, LX-2006 has demonstrated efficacy in reversing the abnormal cardiac phenotype in Friedreich's ataxia disease models by transferring a normal frataxin gene to the heart.
Lidds AB, of Uppsala, Sweden, said it will develop an oncology product with Johnson and Johnson Enterprise Innovation Inc. for an undisclosed indication. Lidds’ drug delivery technology is designed to provide a controlled and sustained release of active drug substances for up to six months.
Mitokinin Inc., of San Francisco, said North Chicago-based Abbvie Inc. purchased an exclusive right to acquire Mitokinin following completion of IND-enabling studies on Mitokinin's lead PINK1 compound, which selectively increases the activity of PINK1, a master regulator of mitochondrial quality control that is genetically linked to Parkinson's disease. Mitokinin will receive an up-front payment and will continue developing its PINK1 activator program through completion of the studies.
Nanoviricides Inc., of Shelton, Conn., reported strong pan-coronavirus effectiveness of its two COVID-19 drug candidates for which the company is preparing a pre-IND application. NV-CoV-2 is the company's broad-spectrum anti-coronavirus clinical lead drug candidate for the treatment of COVID-19 patients based on the Nanoviricides platform. The company is also developing NV-CoV-2-R, a drug treatment that combines the power of both NV-CoV-2 and remdesivir in a single drug that encapsulates and protects remdesivir inside NV-CoV-2.
Oncosec Medical Inc., of Pennington, N.J. published data on Tavo (tavokinogene telseplasmid), a DNA-based interleukin-12, in Clinical Cancer Research. In mouse models, treatment with Tavo induced expression of a CXCR3 gene signature, enhanced antigen presentation, T-cell infiltration and expansion and PD-1/PD-L1 expression. In patients, treatment enriched CXCR3-GS in tumors that exhibited an enhancement of CD8+ T-cell infiltration. One patient who was unresponsive to anti-PD-L1 therapy had increased CXCR3-GS after treatment with Tavo and subsequently had a significant clinical response after receiving additional anti-PD-1 therapy.
Oscotec Inc., of Seongnam, South Korea, and Beactica Therapeutics AB, of Stockholm, disclosed a research development and licensing agreement. Oscotec and Beactica will initially jointly collaborate concerning research and early preclinical development of cancer drug candidates arising out of Beactica's LSD1 program. Oscotec may thereafter opt to take full responsibility for clinical development and commercialization. Oscotec is entitled to gain exclusive global rights for further development and commercialization of Beactica's program. Beactica is eligible to receive up to €149 million (US$$179.8 million) in preclinical, clinical and regulatory milestone payments, including an up-front and equity payment from Oscotec upon signing of the agreement. Furthermore, Beactica could also collect double-digit royalties on commercial sales of the products resulting from the partnership, plus revenue shares from any related future licensing activities by Oscotec.
Perrigo Co. plc, of Dublin, reached an agreement to sell its generic pharmaceutical business to Altaris Capital Partners LLC for $1.5 billion in cash. Altaris will assume more than $50 million in R&D milestone payments and contingent purchase obligations with third-party partners. Perrigo plans to use the cash to advance its consumer self-care strategy, likely through acquisitions in the space.
Phio Pharmaceuticals Corp., of Marlborough, Mass., and Agonox Inc., of Portland, Ore., are collaborating to develop a T cell-based cancer immunotherapy. The companies will use Agonox's double positive CD8 tumor-infiltrating lymphocytes (DP CD8 TIL) treated with Phio's PH-762, a self-delivering RNAi compound that targets the checkpoint protein PD-1. In a preclinical model, the companies showed that treating DP CD8 TIL with PH-762 produces a twofold increase in tumor killing activity compared to DP CD8 TIL alone.
Presage Biosciences Inc., of Seattle, closed a $13 million financing and commenced a new research collaborations with Merck & Co. Inc., of Kenilworth, N.J., and Maverick Therapeutics Inc., of Brisbane, Calif. The research collaborations with Merck and Maverick will utilize Presage’s Comparative In Vivo Oncology intratumoral microdosing platform to evaluate investigational oncology agents.
PYC Therapeutics, of Perth, Australia, said its lead investigational drug, VP-001, for the treatment of RP11 has restored function of the retinal pigment epithelium, the target cells for the therapy, in patient-derived models of the disease. The result builds further conviction in VP-001 as the firm heads toward clinical testing. Breakdown of the blood-retina barrier is a major driver of vision loss in patients with retinitis pigmentosa 11, the company noted.
Qualigen Inc., of Carlsbad Calif., entered a material evaluation and option agreement with the University College London (UCL) to evaluate using G-quadruplex binders (GQBs) developed in Stephen Neidle's lab at UCL with Qualigen's aptamer-linked anti-nucleolin (ALAN). Qualigen said the GQBs will help target ALAN to pancreatic tumors.
Rallybio Inc., of New Haven, Conn., made public the details of two previously undisclosed pipeline programs. RLYB-212 is a monoclonal antibody to human platelet antigen-1a in preclinical development for the prevention of fetal and neonatal alloimmune thrombocytopenia, a potentially life-threatening rare disorder that can cause uncontrolled bleeding in fetuses and newborns. RLYB-116 is a small protein inhibitor of complement factor 5 suitable for subcutaneous administration in preclinical development for the treatment of patients with severe and rare diseases of complement dysregulation. Rallybio plans to submit clinical trial applications for both in the second half of 2021.
Q Biomed Inc., of New York, signed an agreement for Strontium89 (strontium chloride Sr-89 injection) for relief of bone pain in bone metastases to be included on the federal supply schedule. The agreement provides U.S. veterans and other federal government agencies access to Strontium89 for eligible patients. The final contract is effective March 1, 2021, and has a five-year term. The FSS contract will extend access to Strontium89 to over 9 million VA beneficiaries and others within the federal system.
Sorrento Therapeutics Inc., of San Diego, and Scilex Holdings Co., a majority owned subsidiary of Sorrento, have signed an exclusive licensing term sheet with Aardvark Therapeutics Inc., of San Diego, to acquire Aardvark’s formulation of delayed burst release low-dose naltrexone (DBR-LDN), or ARD-301, for the treatment of chronic pain, fibromyalgia and chronic post-COVID-19 in multiple phase II programs planned for this year. Scilex plans to work with Aardvark to initiate a new phase II trial this year for fibromyalgia.
Starton Therapeutics Inc., of Paramus, N.J., licensed the rights to sell its Star-Olz, a once-weekly transdermal olanzapine delivery system for nausea and vomiting, in mainland China to Haisco Pharmaceutical Group Co. Ltd., of Chengdu, China. Starton will receive an undisclosed up-front cash payment and is eligible for additional development and regulatory milestone payments, plus double-digit royalties on annual net sales of Star-Olz in the territory.
Strata Oncology Inc., of Ann Arbor, Mich., disclosed the Strata Precision Indications for Approved Therapies, or Strata PATH, trial, a multidrug, multi-arm, biomarker-driven study designed to evaluate the efficacy of FDA-approved targeted- and immunotherapies in new Strata-defined biomarker indications. Pfizer Inc., of New York, will provide marketed, targeted drugs for four study arms at no cost to participants. Additional pharmaceutical companies are expected to participate after the study launch. The trial will use Strata-defined biomarker profiles from comprehensive DNA and RNA profiling to match patients to one of several different investigational treatment arms.
Tauriga Sciences Inc., of New York, acquired an equity stake in an undisclosed Israeli biotechnology company that develops drugs based on cannabinoid molecules. The company plans to disclose details of the equity stake in a Form 8-K to be filed within the next several days.
Verisim Life Inc., of San Francisco, was awarded a National Science Foundation Small Business Innovation Research grant for $255,908 to support using its artificial intelligence-driven biosimulation platform, Bioisim, to evaluate optimizing combinations and dosing strategies of repurposed FDA approved drugs for treating COVID-19.
Viacyte Inc., of San Diego, W. L. Gore & Associates Inc., of Newark, Del., expanded their partnership to develop membrane and device technologies to deliver cell replacement therapies for diabetes. Through their previous partnership, the companies developed a closed encapsulation system, VC01-103, which is being tested in a phase II study in patients with type 1 diabetes.
Vyne Therapeutics Inc., of Bridgewater, N.J., reported preclinical data on FMX-114, a topical gel formulation of the pan-JAK inhibitor tofacitinib and the sphingosine 1-phosphate receptor modulator fingolimod. In an atopic dermatitis mouse model, the high dose FMX-114 produced an 89% reduction in the modified atopic dermatitis index score relative to the control group at day seven. Both doses had comparable efficacy to triamcinolone 0.1% cream. Vyne plans to start a phase IIa study of FMX-114 in the third quarter of 2021.
Wugen Inc., of St. Louis, licensed rights to fusion molecules from HCW Biologics Inc., of Miramar, Fla. Wugen plans to use the proteins to manufacture its cellular therapeutics for the treatment of cancer.
Yuyu Pharma Inc., of Seoul, South Korea, is collaborating with the University of California, Los Angeles, to test drugs developed in the labs of Rhonda Voskuhl and Michael Jung as potential treatments for either relapsing-remitting or progressive multiple sclerosis.