An experimental drug for treating diabetes and obesity has been shown to lower blood sugar levels and increase fat burning. It is a β2-adrenergic receptor (β2AR) agonist that mimics the effects of physical exercise by activating skeletal muscle metabolism. Unlike GLP-1-based treatments such as semaglutide and tirzepatide, this new compound, developed by researchers at the Karolinska Institute, Stockholm University, and the biotech company Atrogi AB, does not suppress appetite or cause muscle loss.
Edgewise Therapeutics Inc. CEO Kevin Koch speculated that “perhaps a different environment at the FDA” from four months ago led to reviewers’ caution on sevasemten, his firm’s fast skeletal myelin inhibitor for Becker muscular dystrophy and Duchenne muscular dystrophies.
Just a month after laying off 147 employees and announcing plans to mull “strategic alternatives,” Vor Biopharma Inc. reported raising $175 million in private placement in public equity financing and inking a new $4.23 billion license deal for Yantai Rongchang Biotechnologies (Remegen) Co. Ltd.’s telitacicept, a dual-target fusion protein drug approved in China for three autoimmune indications. The news was disclosed after U.S. market hours June 25. Vor’s shares (NASDAQ:VOR) gained 34 cents, or 60.5%, to close June 26 at 89 cents. The company’s shares had risen for eight consecutive trading days since June 17.
The realignment within the U.S. FDA continued with reports of the removal of two high level executives. When asked by BioWorld if the Center for Biologics Evaluation and Research’s (CBER) Office of Therapeutic Products director and deputy director had been forced out and if so, why, an HHS spokesperson responded on background with a single sentence: “Center directors deserve to be supported by managers that are aligned with aggressive goals to expeditiously advance therapeutics for rare diseases using the gold standard of science.”
University of Lausanne has reported promising preclinical data on SAN-523, a first-in-class positive allosteric modulator of cystathionine gamma-lyase (CSE), developed as a potential therapeutic for microcrystalline arthropathies, including gout and calcium pyrophosphate deposition (CPPD) disease.
The revised trial protocol that means a delay in filing for U.S. approval of DYNE-101 to treat myotonic dystrophy type 1 (DM1) dented shares of Dyne Therapeutics Inc. (NASDAQ:DYN), which closed June 17 at $10.86, down $2.96, or 21%.
Rheumatoid arthritis (RA) is a chronic autoimmune disease characterized by inflammation and damage of the joints. PET/CT imaging is a useful tool for early disease assessment and monitoring treatment efficacy.
How the U.S. FDA might respond became a serious question for Wall Street as Sarepta Therapeutics Inc. made known a second death due to acute liver failure with gene therapy Elevidys (delandistrogene moxeparvovec), cleared for Duchenne muscular dystrophy (DMD). Shares of Cambridge, Mass.-based Sarepta (NASDAQ:SRPT) closed June 16 at $20.94, down $15.24, or 42%, as Wall Street digested the news.
Researchers at Ileadbms Co. Ltd. have announced promising results of their novel drug IL-21120033 in a preclinical model of rheumatoid arthritis. The drug acts as a selective agonist of the chemokine receptor CXCR7.
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