CDKL5 Deficiency Disorder

Today, a brief rundown of news from GSK and Amgen, as well as updates from Roche, AbbVie, and Marinus Pharmaceuticals that you may have missed.Amgen set a list price of $1,665 per shot for its biosimilar version of Regenerons Eylea, a 10% discount from the eye drugs current wholesale cost, RBC analyst Brian Abrahams wrote in an Oct. 25 note to clients. On Tuesday, Amgen said it would launch its biosimilar, branded Pavblu, without a legal settlement with Regeneron following a court decision that lifted a temporary injunction on its launch. Such at risk launches are uncommon, and could result in steep penalties for Amgen if subsequent legal judgments side with Regeneron. The small discount means the market impact of Pavblu may be more muted than Amgen investors feared, Abrahams wrote. Further price erosion could occur when additional biosimilars launch. The FDA has approved four others. Jonathan GardnerGSK is making its largest investment in U.S. manufacturing, committing $800 million to expand a site in Marietta, Pennsylvania with new drugmaking facilities. One will handle production of sterile liquid vaccines and medicines, and should be ready by the end of 2028. Another will include a drug substance facility set to start operations by the end of 2027 as well as a pilot plant for making clinical trial medicines. Ned PagliaruloRoche has inked a second research collaboration with gene therapy specialist Dyno Therapeutics. The companies originally linked up in 2020 and, similar to that deal, the one announced Thursday revolves around neurological diseases. Roche and Dyno think the latter's technology can be used to create better “viral vectors“ the microscopic tools that shuttle genetic medicines to a desired target. The new deal tasks Dyno with designing these improved vectors, which will then be applied to potential gene therapies for nervous system disorders. Roche will be responsible for testing the therapies and, if they make it to the market, selling them. Dyno is getting $50 million upfront, plus research payments. It's also eligible for royalties on resulting products, and could nab north of $1 billion more by hitting certain milestones. Jacob BellLongtime partners AbbVie and Gideon Richter are working together on another brain-focused project. Through a sparsely detailed agreement announced Thursday, the companies hope to discover new targets that could be used to treat neuropsychiatric conditions. They plan to share the costs of the necessary research and development. Richter will receive $25 million upfront, and could take home more through milestone and royalty payments. AbbVie, meanwhile, will get worldwide rights to programs that advance to commercialization, except in traditional markets of Richter, such as Europe and Russia. Jacob BellMarinus Pharmaceuticals will lay off staff, cut costs and consider strategic alternatives, after results from a Phase 3 study showed the companys seizure drug ganaxolone did not meet the trials goal. On Thursday, Marinus said the data, while finding nominal reductions in seizure frequency that favored ganaxolone, were likely not enough to support an approval application. The study tested ganaxolone in people with epilepsy related to tuberous sclerosis complex; the drug is already cleared for use treating seizures in people with a condition called CDKL5 deficiency disorder. Ned Pagliarulo '

Marinus Pharmaceuticals is reducing its workforce again and taking other measures to reduce costs after its epilepsy treatment failed a late-stage trial. The TrustTSC trial investigating oral ganaxolone for the treatment of seizures associated with tuberous sclerosis complex (TSC) missed the primary endpoint of percent change in TSC-associated frequency of seizures at 28 days. While there was a reduction in seizures in the treatment arm, the results weren’t statistically significant. The median reduction in seizure frequency was 19.7% for patients on ganaxolone compared with 10.2% for placebo, earning a p-value of 0.09. The company’s stock $MRNS tumbled on Thursday morning, dropping about 80%. “We are disappointed that the results of the TrustTSC trial are not likely to be sufficient for an sNDA filing,” Marinus CEO Scott Braunstein said in a statement . Marinus now plans to discontinue all clinical development of ganaxolone, and it started a “strategic alternatives” process that includes layoffs. The company didn’t specify how many employees it would let go. As of Dec. 31, Marinus had 165 full-time employees, though it cut about 20% of its workforce in May when it also implemented other cost-reducing measures. Since 2022, ganaxolone has been approved as Ztalmy for the treatment of seizures associated with CDKL5 deficiency disorder.

Danish drugmaker Lundbeck will buy La Jolla, CA-based Longboard Pharmaceuticals for $2.6 billion in cash, the companies said Monday morning, marking the largest biopharma M&A deal since July . Neuroscience-focused Lundbeck will get access to a 5-HT2C superagonist that just entered Phase 3 last month for Dravet syndrome, a severe type of epilepsy found in infancy. The experimental medicine, known as bexicaserin, could also apply to other developmental and epileptic encephalopathies like Lennox-Gastaut syndrome and CDKL5 deficiency disorder, the company has said. Lundbeck projects a launch of bexicaserin in the last quarter of 2028 and global peak sales potential of up to $2 billion. The drugmaker also gets an S1P receptor modulator dubbed LP659 that is in Phase 1 and could be tested in multiple neurological diseases. Lundbeck CEO Charl van Zyl said Longboard will “become a cornerstone in Lundbeck’s neuro-rare franchise.” “Bexicaserin addresses a critical unmet need for patients suffering from rare and severe epilepsies, for which there are very few good treatment options available,” van Zyl said in a statement. Longboard spun out of Arena Pharmaceuticals with $56 million in October 2020. It went public five months later. Pfizer then bought Arena for $6.7 billion in December 2021. The deal comes amid a relatively dull past few months for public biopharma M&A, with many of the recent deals falling below $1.3 billion. There has been no deal of $5 billion or higher yet in 2024. Lundbeck itself has been relatively quiet on the acquisition front since its $1.95 billion Alder BioPharmaceuticals deal in September 2019. That led to a migraine drug approval a few months later for Vyepti . The Longboard deal is slated to close this quarter, and both boards have approved it, the companies said. Longboard will be bought for $60 per share $LBPH . Its share price skyrocketed 50% to $58 in premarket trading.