Shares of Soleno Therapeutics Inc. (NASDAQ:SLNO) soared by $15.64, or 353%, to trade midday at $20.07 on word of positive top-line results from the randomized withdrawal period of phase III Study C602, a long-term treatment experiment with DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS). The primary endpoint was the change from baseline in hyperphagia-related behaviors as assessed by the hyperphagia questionnaire for clinical trials, also known as HQ-CT, a caregiver-completed nine-item validated questionnaire for assessing hyperphagia in PWS. The U.S. FDA has told Soleno that data from the trial could support an NDA submission for DCCR, which has orphan drug designation for the treatment of PWS in the U.S. and EU, along with fast track designation from the FDA.
With positive phase III data, Ionis looks to approval in rare disease
Positive top-line phase III study results for olezarsen in treating the rare disorder familial chylomicronemia syndrome has Ionis Pharmaceuticals Inc. looking down the road to U.S. FDA approval. CEO Brett Monia told investors in a morning conference call that an NDA filing could come by early 2024 with a potential approval before that year ends. There is no currently approved therapy in the U.S. for the disease that results in excessive triglycerides and often organ failure. The genetic affliction affects 1 million to 2 million people globally. Placebo-controlled phase III study data showed the antisense medicine met its primary efficacy endpoint with a statistically significant reduction in triglyceride levels. Ionis stock (NASDAQ:IONS) was up 7% at midday with shares at $45.80 each.
Acurastem, Takeda strike $580M licensing deal for ALS, neurodegenerative drugs
Acurastem Inc. struck an out-licensing deal potentially worth $580 million with Takeda Pharmaceutical Co. Ltd. to develop drugs for amyotrophic lateral sclerosis and other PIKfyve gene-targeting therapeutics. Under the terms, Takeda obtains exclusive worldwide rights to Acurastem’s therapeutics, including its lead AS-202 asset, an antisense oligonucleotide therapy to treat ALS. In return, Acurastem is eligible for $580 million in up-front and milestone payments, provided that all clinical, regulatory and commercial milestones are met within a specified timeline, along with tiered royalties on net sales.
Italian firm Alfasigma offers $794M to buy Intercept
Three months after Intercept Pharmaceuticals Inc. received its second complete response letter for obeticholic acid to treat fibrosis due to nonalcoholic steatohepatitis, causing it to pull out of development for the indication, Alfasigma SpA agreed to acquire the company for $19 per share in cash, or about $793.8 million, based on the 41.78 million shares outstanding as of the end of the second quarter. The transaction price is an 82% premium to Intercept’s Sept. 25 closing price of $10.44. Shares (NASDAQ:ICPT) soared 79% on the news and were trading midday on Sept. 26 at $18.70. With the acquisition, Alfasigma gains a broader gastroenterology and hepatology portfolio, as well as a presence in the U.S. market, adding Ocaliva (obeticholic acid) to its list of medicines. Ocaliva, a farnesoid X receptor agonist, is approved in the U.S. as a second-line treatment for primary biliary cholangitis.
Going forward, regulatory flexibility essential in biosimilar realm
As biosimilar development expands beyond monoclonal antibodies to more complex biologics, the flexibility built into regulatory paths across the world will become more essential. Rather than making wholesale changes to those pathways, regulators need to follow the science in exercising the flexibility they already have, Leah Christl, executive director of global biosimilars regulatory affairs and R&D policy at Amgen Inc., told BioWorld. In doing so, “we do need to look forward to what might be coming down the pipeline,” in addition to looking at what types of biosimilars have already been approved, she said.
Japan’s MHLW approves slew of new drugs, including Eisai/Biogen’s Leqembi for Alzheimer’s
Thanks to a raft of new approvals by Japan’s Ministry of Health, Labor and Welfare, patients in Japan will soon have access to Biogen Inc./Eisai Co. Ltd.’s Leqembi (lecanemab), an amyloid beta binder, for slowing progression of mild cognitive impairment and mild dementia due to Alzheimer's disease. Other approvals included Alvotech hf’s biosimilar to Stelara (ustekinumab) and Ardelyx Inc.’s tenapanor.
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