A huge breakthrough as schizophrenia patients get approval for the first novel approach drug in 30 years as the FDA gives the green light to muscarinic agonist KarXT (Cobenfy). This is fantastic news - not just for the BMS/Karuna team and patients - setting the stage for subsequent approvals in the space and underlining the value of investment in neuropsychiatry.
Although KarXT has led the headlines, it isn't the only good news in the regulatory space this week! We see two approvals for Niemann Pick Disease Type C from Zevra Therapeutics and IntraBio. Longboard Pharmaceuticals also get Rare Paediatric Disease Designation and Orphan Drug Designation for Dravet treatment.
In deals, Genespire raises a Series B and positive clinical trial results are announced for Sanofi, Biogen/UCB, and Biohaven.
- In long-awaited news, the
百时美施贵宝
and
Karuna Therapeutics
team receive FDA approval for KarXT (Cobenfy). The muscarinic agonist is the first novel approach drug to be approved for schizophrenia in the past 30 years. Until now, schizophrenia has been treated by dopamine-blocking antipsychotics which pose significant safety risks. KarXT works through a dual activation of the M1 and M4 muscarinic acetylcholine receptors in the central nervous system, limiting the side effects seen associated with traditional antipsychotics. According to the NJ-pharma, which came into possession of KarXT after the 2023 acquisition of Karuna Therapeutics, the company is ready to launch the drug, having made significant progress building out the organisation. BMS is planning to make KarXT available to patients in late October.
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Longboard Pharmaceuticals
achieves FDA rare paediatric disease designation and orphan drug designation for bexicaserin in Dravet Syndrome. They are the first company to receive Breakthrough Therapy Designation for the treatment of seizures associated with Developmental and Epileptic Encephalopathies. Both designations will support the development of this asset from a regulatory and investment standpoint. The company will be initiating its global Phase III DEEp program in the coming weeks.
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Zevra Therapeutics
announces FDA approval for MIPLYFFA? (arimoclomol) to treat patients with ultra-rare neurodegenerative disease, Niemann-Pick Disease Type C. The asset is indicated for use in combination with miglustat to treat neurological manifestations of NPC in adults and paediatric patients 2 years' of age or older.
- IntraBio Inc announces FDA approval of AQNEURSA for the treatment of Niemann-Pick Disease Type C. This drug is the only FDA-approved stand-alone therapy for the disease and is indicated for adult patients and paediatric patients weighing over 15kg.
- Genespire raises €46.6 million (~$52 million) in a Series B co-led by Sofinnova Partners , XGEN Venture, and CDP Venture Capital SGR, forming a strong syndicate with Indaco SGR. Funds from this Series B will be directed towards advancing off-the-shelf paediatric gene therapy for rare disease, Methylmalonic Acidemia (MMA) into the clinic, and bolstering the Company’s gene therapy pipeline.
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Sanofi
presents positive data at ECTRIMS for tolebrutinib, a brain-penetrant BTK inhibitor. The drug demonstrated a 31% delay in time to onset of confirmed disability progression in non-relapsing secondary progressive multiple sclerosis phase 3 study. Global regulatory submissions for the asset will begin in H2 2024.
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Biogen
and
优时比
announce positive topline results from Phase III study of dapirolizumab pegol to treat lupus erythematosus. The primary endpoint was met as the drug demonstrated clinical improvement in moderate-to-severe systemi9c lupus erythematosus. Based on these results, the Biogen/UCB team will be initiating a second Phase III trial in 2024.
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Biohaven
announces positive topline results in pivotal study assessing troriluzole in spinocerebellar ataxia. The oral drug met the study's primary endpoint with modified functional scale for the assessment and rating of Ataxia. The company plans to submit a New Drug Application to the FDA in Q4 2024.
