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Jazz Pharmaceuticals is reaching into its wallet once again, this time buying Actio Biosciences and an experimental therapy for KCNT1+ epilepsy, a form of the disease with no FDA-approved treatments.
Definium Therapeutics’ closely watched anxiety readout could further validate its single-dose LSD candidate, while Compass Pathways advances a psilocybin program through a rolling FDA filing.
News of the joint venture comes months after AstraZeneca vowed to spend $15 billion in China by the end of the decade.
Patients treated with Tenax Therapeutics’ heart failure drug TNX-103 did not show significantly better functional performance—though Leerink analysts found biomarker improvements and subgroup analyses in elderly and more severe patients “very encouraging.”
BlossomHill Therapeutics will use its $150 million IPO proceeds to power the Phase 1/2 development of a non-small cell lung cancer drug, while Latigo Therapeutics will bring in more than $345 million for its late-stage non-opioid pain pill.
BioMarin’s discontinued drug showed biomarker benefits in a Phase 3 study for ENPP1 deficiency but these failed to translate to clinical improvements.
After launching in April, Hunter syndrome drug Avlayah surprised analysts with a strong first quarter, as patients rush to get access to Denali Therapeutics’ new option.
The shift from ex vivo to in vivo gene delivery is rewriting the rules of viral vector manufacturing, forcing developers to rethink scale, purity, cell lines and analytics as vectors themselves become the drug. AGC Biologics is betting on stable producer lines, next‑gen purification and deeper characterization to meet the far stricter demands of in vivo therapies.
Three years after Qalsody was approved by the FDA as the first drug to target a genetic cause of ALS, some patients are reporting not only disease stabilization but also improvement of symptoms.
Despite the recent emergence of a new drug class, schizophrenia remains one of the most challenging neuropsychiatric diseases to treat—and Cobenfy’s first steps into the market indicate there is still room to grow.