Benevolentai Ltd. is betting on its oral phosphodiesterase 10 inhibitor BEN-8744 as having first-in-class potential for moderate to severe ulcerative colitis, after healthy volunteers in a phase Ia trial remained free of central nervous system-associated side effects.
Fennec Pharmaceuticals Inc. has out-licensed its chemoprotective formulation of sodium thiosulfate, Pedmarqsi, to Norgine BV for €40 million (US$44 million) up front and up to an additional €210 million (US$229 million) in commercial and regulatory milestones. The injectable drug reduces the risk of cisplatin-induced ototoxicity in pediatric patients 1 month and older with localized, nonmetastatic solid tumors.
Aquestive Therapeutics Inc.’s oral epinephrine prodrug to treat life-threatening allergic reactions, Anaphylm, produced a faster time to maximum concentration than currently available autoinjectors in healthy adults, early pivotal phase III data show, meeting both pharmacokinetic (PK) primary and secondary endpoints.
With early phase II trial data from Cognition Therapeutics Inc.’s lead candidate, CT-1812, for Alzheimer’s disease expected in mid-2024, the firm is hoping to raise $11.5 million from an underwritten public offering of about 6.6 million shares, priced at $1.75 each.
Exonate Ltd. is preparing to advance its eye drop candidate to treat retinal vascular diseases, EXN-407, into a phase IIb study following promising tolerability and early signs of efficacy in a phase Ib/IIa study. A small-molecule SRPK1 inhibitor, EXN-407 is designed to be administered twice daily and works by selectively targeting specific (proangiogenic) isoforms of VEGF that lead to vascular retinal disease progression via aberrant growth of leaky blood vessels within the eye.
With its sights set on expanding the commercial launch of Xdemvy (lotilaner ophthalmic solution, 0.25%) to treat Demodex blepharitis in the U.S., Tarsus Pharmaceuticals Inc. has priced an underwritten public offering of common stock, selling 2.8 million shares for $32 each, taking the expected gross proceeds to about $100 million.
Baseimmune Ltd. has raised $11.3 million through a series A to accelerate the development of its deep learning AI technology for predicting future pathogen mutations to generate a series of longer-lasting, multistrain vaccines.
Seven new therapies, including two influenza vaccines, were recommended for approval by the EMA’s Committee for Medicinal Products for Human Use during its February 2024 meeting. One of these was sparsentan by CSL Vifor Pharma Inc. and Travere Therapeutics Inc. for the treatment of IgA nephropathy (IgAN), for which the committee has awarded conditional marketing authorization.
Inventiva SA has halted enrollment in its pivotal phase III Nativ3 trial with nonalcoholic steatohepatitis (NASH) candidate lanifibranor after a patient experienced raised liver enzymes indicative of autoimmune hepatitis. The resulting delays to the study could spell trouble for the firm, which estimates its cash runway will only see it through to the start of the third quarter 2024.
The European Commission approved two therapies for progressive, genetic diseases: Biogen Inc.’s Friedreich’s ataxia drug, Skyclarys (omaveloxolone), and Crispr Therapeutics AG’s CRISPR/Cas9 gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia, Casgevy (exagamglogene autotemcel, exa-cel).