Affiris AG, of Vienna, is presenting in vivo data with its antibody, mAB C6-17, against the mutant huntingtin protein at the 16th annual Huntington's Disease Therapeutics Conference, held virtually. The company has developed mAB C6-17 to target a particularly exposed region of the HTT protein. In vitro testing has already shown that mAB C6-17 is able to significantly inhibit mtHTT uptake in cultured cells. The latest study showed that the monoclonal antibody quickly distributes in the body, including into peripheral organs and the central nervous system, in animals.
Awakn Life Sciences Inc., of Toronto, disclosed a new chemical entity development program and appointed Evotec SE, of Hamburg, Germany, as its research partner and the selection of David Nutt, one of the world's leading experts on addiction, as program lead. The company said it will focus on addictions where the consequences for the patient, their family and society are especially severe. The molecules involved will bring together the therapeutic characteristics of MDMA with a more rapid onset of activity and a short half-life, Awakn said.
Clarus Therapeutics Inc., of Northbrook, Ill., and Blue Water Acquisition Corp., of Greenwich, Conn., a special purpose acquisition company, disclosed a business combination agreement that will result in Clarus becoming a publicly traded company. The deal values Clarus at $379 million on a fully diluted basis, assuming no redemptions by Blue Water stockholders. Clarus stakeholders will invest an additional $25 million in Clarus following the disclosure of the deal, expected to close in the third quarter of this year.
Compugen Ltd., of Holon, Israel, disclosed the publication of a peer-reviewed article, titled "COM-902, a Novel Therapeutic Antibody Targeting TIGIT Augments Anti-Tumor T Cell Function in Combination with PVRIG or PD-1 Pathway Blockade" in Cancer Immunology, Immunotherapy. The preclinical data discussed in the paper highlight the potential of COM-902, Compugen's anti-TIGIT therapeutic antibody, to enhance antitumor immune responses.
New postmarketing study data from Dynavax Technologies Corp., of Emeryville, Calif., assessing rates of occurrence of acute myocardial infarction (AMI) in persons receiving Heplisav-B compared with Engerix-B showed an AMI rate per 1,000 person years of 1.67 for Heplisav-B and 1.86 for Engerix-B. The hazard ratio comparing the rate of AMI in the Heplisav-B group with the Engerix-B group was 0.92 with a 95% confidence interval of 0.63 to 1.32. The upper bound of the 95% confidence interval of the hazard ratio comparing the rate of AMI in the Heplisav-B group to the Engerix-B group was less than 2, meeting the primary endpoint. The results provide evidence, the company said, that there is no increased risk of AMI associated with vaccination with Heplisav-B compared to Engerix-B. The results, the company added, reinforce its previous clinical data regarding the safety of Heplisav-B and support its confidence in its ability to prevent hepatitis B infection in adults. Heplisav-B is an adult hepatitis B vaccine that combines hepatitis B surface antigen with Dynavax's Toll-like receptor 9 agonist CpG 1018 to enhance the immune response.
Enalare Therapeutics Inc., of Princeton, N.J., said it received an initial contract from the U.S. Biomedical Advanced Research and Development Authority (BARDA) to support development of the company's lead product, ENA-001. The funding will be used to advance development of an intramuscular formulation for ENA-001 as an agnostic respiratory stimulant. The funding is to support development of therapeutic candidates against a range of chemical threats. Enalare is developing therapies for patients suffering from life threatening acute respiratory and critical care conditions, including respiratory depression caused by drug overdose, post-surgery and respiratory infections.
Fresh study results from Epivax Inc., of Providence, R.I., demonstrated antigen-specific tolerance induction in allergy with the company’s Tregitopes, which are peptides found in human immunoglobulin. Administration of IgG-derived Tregitopes was found to induce suppressive antigen-specific T regulatory cells in ovalbumin and ragweed-allergic murine mouse models, attenuating allergen-induced airway hyperresponsiveness and lung inflammation, the company said. Tregitopes are capable of engaging regulatory T cells and down-regulating inflammation in a wide range of disease models, according to Epivax.
Generex Biotechnology Corp., of Miramar, Fla., said it received a $2 million development payment from Beijing Youfeng Biological Technology Ltd. to provide research results, data, regulatory documents and manufacturing methods to support filing an IND for the Ii-Key-SARS-CoV-2 vaccine in China. The company’s partners in China are responsible for development and regulatory approval. All clinical trials will be conducted under guidelines of the International Council on Harmonisation, so the data can be used for submission to the FDA, EMA and other international regulatory agencies, according to Generex.
Gilead Sciences Inc., of Foster City, Calif., said it is providing voluntary licensing partners in India with technical assistance, support for the addition of local manufacturing facilities and donation of active pharmaceutical ingredient to scale up production of remdesivir to address shortages of the drug during the surge in COVID-19 infections in the country. Remdesivir is approved in India for restricted emergency use to treat suspected or laboratory confirmed COVID-19 in adults and children hospitalized with severe disease. Gilead also plans to donate at least 450,000 vials of Veklury (remdesivir) to help address the immediate needs of patients in India.
Glauconix Biosciences Inc., of Albany, N.Y., and Nicox SA, of Sophia Antipolis, France, said they co-authored a manuscript published in Investigative Ophthalmology and Visual Sciences that showed the ability of NCX-667, a nitric oxide donor prostaglandin analogue, to lower intraocular pressure (IOP). The studies were performed using a 3D bioengineered conventional outflow tract model developed by Glauconix that’s composed of human donor trabecular meshwork and Schlemm's canal cells. The article reported that the 3D human tissue model was effective in retaining biological and physiological features found in human trabecular meshwork and Schlemm's canal tissue. The model's relevance to the pathophysiology of glaucoma enabled validation of efficacy in development compounds now translated to commercialized products, such as ROCK inhibitors. The article also featured data showing robust and dose-dependent decreases in IOP produced by NCX-667 in various animal models, including rabbit, dog and monkey.
Guardant Health Inc., of Redwood City, Calif., and Daiichi Sankyo Inc., of Tokyo, will collaborate to develop the Guardant360 CDx blood test as a companion diagnostic for Enhertu (fam-trastuzumab deruxtecan-nxki), a HER2-directed antibody-drug conjugate being studied as a treatment for patients with advanced metastatic non-small-cell lung cancer. The blood test is FDA-approved to provide genomic profiling results for guiding solid tumor treatment.
Inmed Pharmaceuticals Inc., of Vancouver, British Columbia, said it provided written notice to the Toronto Stock Exchange of the voluntary delisting of its common shares in light of strong trading data since its Nov. 12, 2020, listing on the Nasdaq, where the stock trades as INM. On April 27, INM closed at $3.38 for a loss of 18 cents.
Inveniai LLC, of Guilford, Conn., said it formed a multitarget drug discovery collaboration with Shionogi & Co. Ltd., of Osaka, Japan, involving its artificial intelligence platform, Alphameld. Shionogi will synthesize drugs against targets proposed by Inveniai for diseases of interest and will develop, manufacture and commercialize any candidates. Inveniai is eligible for up to $200 million per program through a combination of up-front payments, development and commercial milestones, and royalties.
Logicbio Therapeutics Inc., of Lexington, Mass., said it formed a strategic collaboration with and granted an option to Canbridge Pharmaceuticals Inc., of Beijing, for an exclusive license in greater China to LB-001 to treat methylmalonic acidemia. The in vivo gene editing therapy is based on Logicbio's gene insertion platform, Generide. Should it exercise the option, Canbridge will assume responsibility and costs for development, regulatory, commercial and manufacturing activities in the licensed territory. Logicbio also granted Canbridge a global license for the adeno-associated virus sL65, the first capsid produced from its Saavy platform, and agreed to provide development support on gene therapy candidates to treat Fabry and Pompe disease along with two optional indications. Logicbio is eligible for an up-front payment of $10 million along with options payments, milestone payments of up to $581 million and up to double-digit royalties on net sales. Separately, Logicbio said it formed a research collaboration with and granted an exclusive option agreement to Daiichi Sankyo Co. Ltd., of Tokyo, to develop treatments for two undisclosed indications based on the Generide platform. Daiichi Sankyo secured the option to negotiate and acquire global licenses for the development programs. Financial terms were not disclosed.
Merck and Co. Inc., of Kenilworth, N.J., said it formed nonexclusive voluntary licensing agreements for molnupiravir with established Indian generics manufacturers Cipla Ltd., Dr. Reddy’s Laboratories Ltd., Emcure Pharmaceuticals Ltd., Hetero Labs Ltd. and Sun Pharmaceutical Industries Ltd., which have World Health Organization pre-qualified manufacturing facilities and experience as suppliers to key procurers for low- and middle-income countries (LMICs). Merck will provide licenses to the manufacturers to supply molnupiravir to India and more than 100 LMICs, accelerating availability following approvals or emergency authorizations by local regulatory agencies. Merck also is in discussions with the Medicines Patent Pool to explore the potential for additional licenses. An RNA polymerase inhibitor, molnupiravir is in phase III development in a collaboration with Ridgeback Biotherapeutics LP, of Miami, to treat non-hospitalized people with confirmed COVID-19 infection.
Moderna Inc. and DNA Script SAS, of Paris, San Francisco and Boston, will collaborate to develop a prototype for rapid mobile manufacturing of vaccines and therapeutics as part of the Defense Advanced Research Projects Agency's Nucleic Acids On-Demand World-Wide program. The partnership will employ DNA Script's enzymatic synthesis platform to generate high-fidelity nucleic acids without using hazardous chemical solvents to power Moderna's manufacturing technology to produce mRNA therapeutics and vaccines. DNA Script will receive up to $5 million in grant funding from the agency.
Nascent Biotech Inc., of San Diego, said it preemptively paid and eliminated a convertible debt note of $186,000 held by JSJ Investments LLC, preventing a dilutive conversion. JSJ had the right to convert the value of the note into common shares at a discount to current average trading levels in NBIO shares, which trade on the OTCQB. On April 27, the stock closed at 7.6 cents.
Ose Immunotherapeutics SA, of Nantes, France, said it granted a license to Veloxis Pharmaceuticals Inc., a subsidiary of Asahi Kasei Pharma Corp., of Tokyo, for global rights to develop, manufacture and commercialize FR-104, a CD28 antagonist monoclonal antibody fragment, in all transplant indications. Ose, which retained rights to develop the asset in autoimmune diseases, is set to receive up to €315 million (US$380.2 million), including €7 million up front and the remainder in development, registration and commercialization milestone payments along with tiered sales royalties. Veloxis assumed production, development and commercialization costs in transplant indications and said it plans to advance FR-104 as prophylaxis for organ rejection in people receiving solid organ transplants.
Pyxis Oncology Inc., of Cambridge, Mass., which in March 2021 secured $152 million in a series B to advance its portfolio, disclosed targets and preclinical data supporting three antibody-drug conjugate (ADC) candidates. The company said PYX-201 is a non-internalizing ADC that targets extra domain-B of fibronectin, a key component of the tumor extracellular matrix that is expressed across non-small-cell lung, ovarian, breast and pancreatic cancers and other solid tumors. PYX-201 is designed to release an auristatin payload with bystander activity into the extracellular space to induce immunogenic cell death. PYX-202 is an internalizing ADC targeting delta-like 1 homolog, a tumor antigen expressed in small-cell lung cancer, soft tissue sarcoma, hepatocellular carcinoma, neuroblastoma and other tumor types. PYX-202 uses a monomethyl auristatin payload and a beta-glucuronide cleavable linker to increase stability in circulation and reduce off-target toxicities. Finally, PYX-203 is an internalizing ADC targeting CD123 that uses a lysosomal cleavable linker and cyclopropylpyrroloindole DNA-damaging payload to achieve positive treatment outcomes while limiting adverse side effects.
Sarepta Therapeutics Inc., of Cambridge, Mass., said it received notice of an unsolicited mini-tender offer by TRC Capital Investment Corp., to purchase up to 2 million of its common shares (NASDAQ:SRPT), or approximately 2.5% of its outstanding shares, at $68.50 apiece in cash. The offering price represents a discount of approximately 4.6% to the closing price of $71.80 for SRPT on April 16, 2021, the last business day prior to commencement of TRC’S offer. Sarepta said it does not endorse the unsolicited mini-tender offer and recommended that stockholders do not tender their shares due to the offer’s below-market price and conditions. On April 27, Sarepta’s shares closed at $70.51 for a loss of $2.79.
Sentia Medical Sciences Inc., of San Diego, said it formed a research collaboration with Neurocrine Biosciences Inc., also of San Diego, to discover long-acting corticotropin-releasing factor (CRF) receptor antagonist peptide therapeutics to treat hypothalamic-pituitary-adrenal axis modulated diseases. Sentia agreed to provide Neurocrine an exclusive license to existing preclinical CRF receptor antagonist peptide compounds. The companies also will collaborate to discover peptide antagonists targeting CRF and advance them to development candidate stage, with Neurocrine then assuming sole responsibility for additional development, manufacturing, regulatory and commercial activities. Sentia is set to receive an up-front payment and committed research funding from Neurocrine along with milestone and royalty payments, all undisclosed.
Shasqi Inc., of San Francisco, said it was awarded a $2 million direct-to-phase-II Small Business Innovation Research grant from the U.S. National Cancer Institute to support immune biomarker analysis of patient samples from the company’s phase I trial of lead candidate SQ-3370, a dual DNA polymerase/topoisomerase II inhibitor, as well as manufacturing process development.
Storm Therapeutics Ltd., of Cambridge, U.K., said a paper published in Nature offered additional validation of its research on targeting RNA-modifying enzymes to develop cancer therapeutics and showed that its METTL3 inhibitors provided antitumor efficacy in physiologically relevant, proof-of-concept animal models of acute myeloid leukemia (AML) and solid tumors. METTL3 small-molecule inhibition prolonged survival in multiple AML models by targeting key stem cell subpopulations. Findings also confirmed antitumor activity against different AML driver mutations, suggesting that targeting METTL3 is not limited by specific mutations.
Zogenix Inc., of Emeryville, Calif., said it launched a global access program for Fintepla (fenfluramine), a serotonin reuptake inhibitor approved in the U.S. and EU as add-on therapy to treat seizures associated with Dravet syndrome in people 2 and older. The program expands prescribing access in other parts of the world, where local regulations allow, including European countries where reimbursement is not established. Zogenix partnered with Durbin, a unit of Uniphar Group’s Product Access Division, to manage the program.