Chinese pharmaceutical and biotech companies are leading development of glucagon-like peptide-1 receptor agonists (GLP-1RA) as Novo Nordisk A/S and Eli Lilly and Co. edge closer to launching blockbuster therapies in China. At the heart of the GLP-1 boom is a nationwide obesity problem driven by a confluence of factors, including the rise of a modern, sedentary lifestyle, according to Clarivate. Despite the rising prevalence of obesity and type 2 diabetes, the gap in obesity therapeutics is “substantial and leaves a solid market opportunity for weight loss drugs,” Karan Verma, principal analyst of health care research and data analytics at Clarivate, said.
Rare disease failure batters Fulcrum shares
After missing the primary and secondary endpoints in its phase III study of losmapimod in treating the rare disease facioscapulohumeral muscular dystrophy (FHSD), Fulcrum Therapeutics Inc. is yanking the program’s plug. The selective p38α/β mitogen activated protein kinase inhibitor had a lot of money behind it. It was originally in-licensed from GSK plc and then in May Sanofi SA signed on to help Fulcrum develop and commercialize losmapimod for FHSD worldwide excluding the U.S. in a deal worth $1.06 billion. FHSD, the second most common muscular dystrophy, has no approved therapy. The company’s stock (NASDAQ:FULC) was taking a direct hit at midday, with shares sagging 60% at $3.53 each.
Moderna falls on R&D cuts, filing delays, terminated programs
Moderna Inc.’s shares (NASDAQ:MRNA) sank 19% to a $64.11 low in early trading Sept. 12 as investors learned during its annual R&D Day event of a $1.1 billion reduction to R&D and the U.S. FDA’s reluctance to support an accelerated approval filing for the company’s individualized neoantigen therapy for melanoma. Next the company will file for approval by year-end of three of its assets, using priority review vouchers for each: its next-generation COVID-19 vaccine, mRNA-1283; its COVID-19 and flu combination vaccine, mRNA-1083; and a label expansion for its respiratory syncytial virus vaccine, Mresvia (mRNA-1345), for high-risk adults ages 18 to 59. That pushes back some other programs and completely cuts others. The company also pushed its cash flow breakeven to 2028, expecting about $6 billion in revenue. “These updates put to rest key elements of the bull thesis and reflect a worsening financial position,” said analyst Mani Foroohar, of Leerink Partners.
Vironexis exits stealth with 10+ AAV-delivered immuno-oncology candidates
Vironexis Biotherapeutics Inc. came out of stealth mode today, disclosing that it has more than 10 product candidates it’s been developing over the last three years. The therapies are built on the company’s AAV-based platform, Transjoin, which is designed to have patients' livers express bispecific antibodies that bind to both CD3 on T-cells and various targets on tumor cells. The drug candidates include one for CD19+ acute lymphoblastic leukemia that’s ready to start a clinical trial next month and another for the prevention of metastatic HER2-positive cancer that’s expected to enter the clinic in 2025.
Partial epigenetic reprogramming to treat ischemic optic neuropathy
A novel gene therapy that leads to cellular rejuvenation could restore vision after non-arteritic anterior ischemic optic neuropathy and glaucoma. The technique is based on a reprogramming process that reverses the epigenetic DNA alterations caused by aging. Preclinical studies in glaucoma mice and nonhuman primates models for this stroke-like disorder that affects the eye, showed an improvement of vision and restoration of the damaged axons of the optic nerve.
Drug developers index falls as cancer trials hit setbacks
The BioWorld Drug Developers Index (BDDI) declined in August, ending the month down 1.11% for the year and diverging from its previous trend of mirroring the Nasdaq Biotechnology Index (NBI) and the Dow Jones Industrial Average (DJIA). By the close of August, the BDDI had slipped, while the NBI gained 11.7% year-to-date and the DJIA rose 7.05%.