There is broad agreement that psychiatric diagnoses in their current form are not reflective of any underlying biology, and that this is one of the things hampering psychiatric drug development. “We are still fully reliant on descriptive diagnoses that yield heterogeneous patient cohorts,” Steve Hyman told the audience at the European Congress of Neuropsychopharmacology (ECNP) Roadmap Meeting on Precision Psychiatry in Amsterdam in January.
Vanda Pharmaceuticals Inc. gained U.S. FDA approval for Bysanti (milsaperidone), cleared for use in acute bipolar I disorder and schizophrenia and ensuring continuation of the firm’s atypical antipsychotic franchise, with Fanapt (iloperidone) set to start losing patent protection in 2027. It also offered a rare straightforward regulatory win for Vanda, which has tussled with the FDA in recent years.
Reviva Pharmaceuticals Holdings Inc. may be headed back to the clinic for another phase III study of brilaroxazine in treating schizophrenia. With a successful series of early and mid-stage studies behind the treatment, the U.S. FDA recommended a second study for the serotonin-dopamine and neuroinflammatory signaling modulator after a pre-NDA meeting in order to net more efficacy results and expand the safety dataset.
Maplight Therapeutics Inc.’s pricing of a $258.9 million financing this week revived the debate over whether targeting the M1 as well as the M4 muscarinic receptor – as Bristol Myers Squibb Co. does with U.S. FDA-approved Cobenfy (xanomeline and trospium chloride) for schizophrenia – is a better strategy than going after M4 alone.
Positive allosteric modulators (PAMs) of the muscarinic acetylcholine M4 receptor show potential against schizophrenia because they can enhance endogenous acetylcholine signaling, which in turn may mitigate psychotic symptoms as well as improve attention and working memory in individuals with the disorder. However, the efficacy of a promising PAM, CVL-231, has been disappointing in clinical trials.
LB Pharmaceuticals Inc. became the first traditional biopharma company to price an IPO in the U.S. since February, raising $285 million through the upsized sale of 19 million shares at $15 apiece. Shares, now listed on Nasdaq under the ticker symbol LBRX, rose 15% to close Sept. 11 at $17.30.
With new and positive phase III data in treating agitation in patients – at home instead of in a clinic – with bipolar disorders or schizophrenia, Bioxcel Therapeutics Inc. is preparing to submit an sNDA in the first quarter of 2026 to expand the label of the company’s Igalmi (BXCL-501). These data also bring the company closer to having the first such treatment that is approved by the U.S. FDA for home use. The unmet need for patients is huge, Vimal Mehta, the CEO of Bioxcel, told the BioWorld Insider podcast. Watch the interview, below.
Dysregulation of cholinergic neurotransmission through muscarinic receptors, particularly the M1 and M4 subtypes, has been implicated in the pathophysiology of schizophrenia. Muscarinic M4 receptor agonists and positive allosteric modulators (PAMs) have demonstrated the ability to modulate the disrupted neural circuits associated with this disorder.
