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.
Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from its first five patients. However, the company’s stock (NASDAQ:RGNX) shuddered on June 5 as shares closed at $8.36 each, a drop of 17% on the day.
Inflamax Pharmaceuticals (Guangzhou) Co. Ltd. has disclosed proprotein convertase subtilisin/kexin-type 6 (PCSK6; PACE4; PC7A) inhibitors reported to be useful for the treatment of osteoarthritis.
Developing a therapy for an ultra-rare condition has its challenges, including finding enough patients for clinical enrollment and convincing regulatory authorities that limited data prove the candidate is safe and effective. For that reason, Stealth Biotherapeutics Inc. has faced numerous roadblocks getting its mitochondria-targeting elamipretide across the finish line for Barth syndrome, a condition that affects about 230 to 250 males worldwide, including fewer than 150 in the U.S.
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