2024 was another banner year for GLP-1 receptor agonists (GLP-1RAs) on multiple fronts. They continued to expand into new indications, and provide their developers with both rich remuneration and scientific acclamation. There are now seven approved GLP-1RAs. Commercially, the most successful one so far is semaglutide, sold under the brand name Wegovy or Ozempic depending on the indication.

With Alhemo (concizumab-mtci) from Novo Nordisk A/S, a third hemophilia drug in the past eight months has been approved by the U.S. FDA. The once-daily injectable prophylaxis is to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B with inhibitors.

Kura Oncology, Inc. and Kyowa Kirin Co. Ltd.’s selective oral menin inhibitor ziftomenib showed encouraging data across multiple studies, the most encouraging of which were in combination with other standard of care therapies in patients with NPM1-mutant and KMT2A-rearranged  acute myeloid leukemia.

Kura Oncology, Inc. and Kyowa Kirin Co. Ltd.’s selective oral menin inhibitor ziftomenib showed encouraging data across multiple studies, the most encouraging of which were in combination with other standard of care therapies in patients with NPM1-mutant and KMT2A-rearranged  acute myeloid leukemia.

Takeda Pharmaceutical Co. Ltd. is licensing Keros Therapeutics Inc.’s activin inhibitor elritercept in a global development and commercialization deal worth up to $1.3 billion that excludes mainland China, Hong Kong and Macau.

At the 66th American Society of Hematology Annual Meeting, a plethora of companies presented clinical trial data highlighting their drugs targeting Bruton tyrosine kinase (BTK) in patients with blood cancers.

Agios Pharmaceuticals Inc. is learning the hard way the downside of having a pipeline in a product. When a side effect crops up, investors are likely to worry that it may affect the potential of the drug in the numerous diseases the drug could potentially treat.