The U.S. FDA has approved Relyvrio, the therapy formerly known as AMX-0035, for treating amyotrophic lateral sclerosis (ALS) in adults. Relyvrio, from Amylyx Pharmaceuticals Inc., is the third approved treatment option available for ALS, perhaps best known as Lou Gehrig’s disease, in the U.S. and the first to demonstrate a significant slowing of disease progression and functional decline, as well as extended survival, in a randomized, placebo-controlled trial.

Japan’s MHLW approves raft of new drugs

Japan’s Ministry of Health, Labor and Welfare (MHLW) approved a slew of new drugs in the last week of September, including Japan’s first sustained release GIP/GLP-1 receptor agonist for type 2 diabetes from Eli Lilly and Co. and Mitsubishi Tanabe Pharma Corp; a self-administered injectable drug for rheumatoid arthritis from Eisai Co. Ltd. and Nippon Medac Co. Ltd.; a CAR T from Legend Biotech Corp. and Janssen Pharmaceutical KK; and two drugs from Astrazeneca plc, one in asthma and the other in pediatric plexiform neurofibromas in neurofibromatosis type 1.

Solid raising $75M to buy Aavantibio, advance Duchenne therapy 

Solid Biosciences Inc. signed a deal to acquire privately held Aavantibio Inc., made possible by way of a $75 million private placement with institutional investors. The combined company will operate under Solid’s name and continue to trade under its ticker. Bo Cumbo, Aavantibio’s CEO, will serve in that role at the merged company, as well as president. The pipeline is led by gene therapy SGT-003 for Duchenne muscular dystrophy, bound for an IND application in mid-2023, with patient dosing targeted for later that year. Shares of Solid (NASDAQ:SLDB) were trading midday at 53 cents, up about 6 cents, or 13.2%.

Calithera, Kronos, others exploring Syk inhibition

Calithera Biosciences Inc.’s in-licensing deal to take ownership of a pair of oncology assets from Takeda Pharmaceutical Co. Ltd. may be on its way to paying off, perhaps especially with regard to the oral Syk/FLT3 inhibitor mivavotinib, formerly known as CB-659/TAK-659. Also busy is Kronos Bio Inc., with entospletinib (GS-9973) and lanraplenib (GS-9876). In August of this year, the company kicked off a phase Ib/II trial in acute myeloid leukemia, combining lanraplenib with Xospata (gilterinib, Astellas Pharma Inc.).

Simcere out-licenses autoimmune drug to Almirall in $507M deal

Simcere Pharmaceutical Group has out-licensed its autoimmune drug candidate SIM-0278 for all indications to Almirall SA in a deal worth up to $507 million. Simcere granted exclusive global rights to the candidates to Almirall for development and commercialization excluding mainland China, Hong Kong, Macau, and Taiwan. In return, Almirall will pay Simcere $15 million up front. An Almirall spokesperson told BioWorld its team expects to file IND applications in U.S. and Europe in the second half of 2023.

After EU approval, Biomarin heads back to FDA with refile for pricey gene therapy

After winning the backing of European regulators, Biomarin Pharmaceutical Inc. is returning to the U.S. FDA with its hemophilia A gene therapy following a rejection in August 2020, armed with data it hopes will assuage concerns about long-term safety and benefits. The company said the new submission included its response to the FDA’s complete response letter for valoctocogene roxaparvovec and further feedback, including two-year outcomes from the global GENEr8-1 phase III study, plus data from five years of follow-up from an ongoing phase I/II dose escalation study.

Cerecin closes first tranche of new financing to advance CER-0001

Cerecin Inc. has successfully closed the first tranche of a series IIB financing with investors from South Korea, with SK Securities Co. Ltd. and KNT Investment LLC leading the round. Proceeds from the financing, of undisclosed value, will help the company advance clinical studies of its CER-0001 (tricaprilin) in migraine and infantile spasm. The new funds will also be used to build Cerecin’s pipeline and advance plans for a pivotal trial in Alzheimer’s disease, an indication first pursued by the company under its former name, Accera Inc.

The BioWorld Insider Podcast – Psychedelic evolution: Mindset Pharma looks to change mental health treatment

An analyst recently observed that mental health treatments are stuck where cancer was 50 years ago. However, there have been major advancements in developing psychedelic medicines to address that problem. BioWorld staff writer Lee Landenberger talked with James Lanthier, the CEO of Mindset Pharma, which is developing what it calls “next-generation” psychedelic medicines to treat neurological and psychiatric disorders. Lanthier discussed challenges facing companies developing psychedelic treatments and how to overcome the one-size-fits-all, trial-and-error approach to mental health treatment that often seems stuck in the past.

Also in the news

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