The fibroblast growth factor 21 (FGF21) analogue space took another turn as Novo Nordisk A/S said during its second-quarter financial update that work with zalfermin (NNC0194-0499) will be discontinued. A phase II study testing the compound in combination with glucagon-like peptide-1 therapy failed to hit the primary endpoint related to liver fibrosis in metabolic dysfunction-associated steatohepatitis (MASH), without MASH worsening.
Deep learning tools for protein design can also be used to create molecules that bind to them. Certain peptides, such as intrinsically disordered proteins (IDPs), are challenging to target due to their variable nature. However, scientists from the lab of Nobel laureate David Baker have developed a method to generate binders for IDPs by searching the world’s largest protein database with their AI-powered tool RFdiffusion.
Madrigal Pharmaceuticals Inc.’s long-awaited business development pact became reality by way of an exclusive global license agreement that could be worth more than $2 billion with CSPC Pharmaceutical Group Ltd., of Shijiazhuang, China, for SYH-2086. The candidate is a preclinical oral, small-molecule glucagon-like peptide-1 (GLP-1) receptor agonist and orforglipron derivative.
With reduced sales and operating expectations for the rest of the year, Novo Nordisk A/S is on the receiving end of a huge stock drop. For a big pharma, where the stock drops are usually more modest, the shares (NASDAQ:NVO) fell off a cliff on July 29, closing 21.8% downward to $53.93 each, the lowest price per share in the past 12 months. Lower U.S. sales of semaglutide blockbusters Wegovy and Ozempic for treating obesity and diabetes are at the heart of the plunge. In May, the Danish company had expected 13% to 21% sales growth but now is looking at only 8% to 14%.
PTC Therapeutics Inc. will be launching its oral phenylketonuria therapy, Sephience (sepiapterin) in both the U.S. and Europe this summer, following the U.S. FDA approval just ahead of its July 29 PDUFA date. Sephience previously gained marketing authorization by the European Commission, roughly three months after a thumbs up from the EMA’s Committee for Medicinal Products for Human Use.
SML Biopharm Co. Ltd. is harnessing mRNA technology to develop novel immunotherapy-based cancer vaccines, including two candidates for cervical and head and neck cancers caused by human papillomavirus (HPV) infection.
The U.S. FDA on July 15 cleared Biocon Biologics Ltd.’s Kirsty (insulin aspart-xjhz) as the first and only interchangeable biosimilar product referencing Novo Nordisk A/S’ Novolog (insulin aspart), a rapid-acting diabetes medication.
Launched out of 5AM Ventures’ 4:59 Initiative, Renasant Bio Inc. has secured $54.5 million in seed funding to support development of treatments for autosomal dominant polycystic kidney disease (ADPKD), a genetic disease caused by mutations in the PKD1 and PKD2 genes, which encode polycystin proteins PC1 and PC2 that come together to form a calcium ion channel vital for kidney function.
A glucagon-like peptide-1 and glucose-dependent insulinotropic polypeptide receptor dual agonist developed by Jiangsu Hengrui Pharmaceuticals Co. Ltd. and licensed by Kailera Therapeutics Inc. has shown a mean weight loss of 19.2% at the 6-mg dose with no plateau over 48 weeks in a phase III trial conducted in China.
Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a complete response letter (CRL) regarding the BLA for its gene therapy to treat Sanfilippo syndrome type A, saying it needs more details and improvements made about CMC after having finished manufacturing facility inspections.
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