Today, a brief rundown of news from Ipsen and AstraZeneca, as well as updates from Sobi and the Food and Drug Administration that you may have missed.
French pharmaceutical company Ipsen will acquire the California-based company Kartos Therapeutics in a deal that will hand Ipsen an experimental drug for a rare blood cancer. Ipsen will pay $450 million up front at closing, the company said Monday, and is eligible to receive goal-based payments of up to $1.3 billion, inclusive of “a significant regulatory approval milestone” and sales-based milestones. At the center of the deal is Kartos’ myelofibrosis drug, dubbed navtemadlin, which blocks “MDM2,” a key regulator for the protein p53 that keeps tumors in check. Early data of the drug indicate navtemadlin has potential to treat intermediate and high-risk myelofibrosis, and is currently in Phase 3 testing. The acquisition is expected to close in the third quarter. — Delilah Alvarado
AstraZeneca unveiled data Sunday from a Phase 3 trial testing its experimental enzyme replacement therapy efzimfotase alfa in children with a rare condition that affects bone development. The company said the therapy displayed a “statistically significant and clinically meaningful improvement” in children aged 2 to 12 years with hypophosphatasia at 25 weeks compared to placebo and who had not been treated with the enzyme replacement therapy Strensiq. The company additionally said the treatment showed improvements in secondary endpoints of physical function and quality of life at week 25. Full data from another trial in children aged 12 years and older will be shared at a later medical meeting. — Delilah Alvarado
The Swedish pharmaceutical company Sobi said Friday it received a complete response letter from the Food and Drug Administration for its drug NASP as a treatment for adults with uncontrolled gout. The FDA is seeking additional data related to manufacturing of the drug, also referred to as nanoencapsulated sirolimus plus pegadricase, and to address contract manufacturing facility deficiencies. The FDA did not identify efficacy or safety issues with the treatment. Sobi will meet with the FDA to discuss feedback and next steps. — Delilah Alvarado
BeOne Medicines said Tuesday its drug Brukinsa combined with Rituxan significantly delayed disease progression or death in people with the rare cancer mantle cell lymphoma when compared to Rituxan and chemotherapy in a Phase 3 trial. The Brukinsa-Rituxan combination reduced the risk of progression or death by 43% in people who’d never been treated for their illness before, BeOne said, adding that researchers saw a strong but not statistically significant trend toward improved overall survival. The combination, if approved by regulators, would offer patients the first chemotherapy- and infusion-free treatment regimen in mantle cell lymphoma, which could be more tolerable and offer an acceptable alternative for older, frailer patients. BeOne said it will present the data at an upcoming medical meeting as well as submit it for regulatory approval by the end of 2026. — Jonathan Gardner
The FDA said it has selected the first seven facilities to take part in its “pre-check”pilot program to streamline regulatory review and speed construction of new pharmaceutical manufacturing plants in the U.S. From more than 80 applications received between Feb. 1 and March 1, the agency selected new factories being built by Amneal Pharmaceutical, Cellares, Eli Lilly, FUJIFILM Biotechnologies, Kriya Therapeutics, Kyowa Kirin and Regeneron Pharmaceuticals. The two-part program will consist of a “facility readiness” stage that will provide early technical guidance before a company submits a product application; and an “application submission” phase that will entail facility focused meetings to allow for facility inspections earlier in the review cycle. — Jonathan Gardner
