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.
After Recognify Life Sciences Inc.’s phase IIb failure in treating the cognitive impairment in those with schizophrenia, the indication is no closer to a U.S. FDA approved therapy. The placebo-controlled study of inidascamine missed its primary endpoint to improve cognition, joining a list of companies that have tried and failed to find a successful treatment.
The acquisition of Karuna Therapeutics Inc. by Bristol Myers Squibb Co. (BMS) was announced in December 2023, and closed as the BioEurope Spring meeting was convening in March. Along with the acquisition of Cerevel Therapeutics Inc. by Abbvie Inc., the deal signaled that big pharma companies were ready to get back into the brain diseases space.
Despite positive findings from an earlier trial, Alto Neuroscience Inc.’s BDNF-targeting candidate, ALTO-100, failed to best placebo in a phase IIb study in major depressive disorder, sending shares of the company to their lowest price since going public in a February 2024 IPO, as investors worried about readthrough to Alto’s biomarker-based approach for treating psychiatric disorders.
The discussion that preceded the June 4 U.S. FDA advisory committee vote against the approval of Lykos Therapeutics Inc.’s midomafetamine as a treatment for post-traumatic stress disorder could shine some light on the way forward for other sponsors developing psychedelics for approved medical use.
Alto Neuroscience Inc.’s start this spring of the phase II double-blind, single- and multiple-dose study to test the pharmacodynamics of ALTO-203 in major depressive disorder represents another stake planted in a notoriously difficult indication. But getting attention as well is the push by Los Altos, Calif.-based Alto in post-traumatic stress disorder (PTSD) with a separate compound.
Precision psychiatry got some love at two quite different meetings this week, the European Congress of Neuropsychopharmacology’s New Frontiers meeting and BioEurope Spring. The New Frontiers Meeting, an annual two-day meeting dedicated to cutting-edge issues in brain disease research, focused on big-picture and scientific – at times almost philosophical – questions of how to get to a classification scheme for brain disorders that aligns with the underlying biology.
The week closed out with two IPOs on their way in. Alto Neuroscience Inc. (NASDAQ:ANRO) and Fractyl Health Inc. (NASDAQ:GUTS) both debuted on Wall Street with offerings looking to raise combined $238.6 million.
The first IPO of 2024 is a greatly upsized one from CG Oncology Inc., which is selling 20 million shares (NASDAQ:CGON) of its common stock at $19 each. Shares closed Jan. 25 95.6% higher at $37.17 each. The company initially had looked to raise about $200.6 million by selling its shares somewhere from $16 to $18 each but adjusted its thinking before the Jan. 25 debut, now anticipating gross proceeds of $389 million.
Brain disorders have not yet profited from advances in precision medicine to the same extent that other disorders have. With the advent of magnetic resonance imaging and other technologies, watching the brain at work has made great strides in recent decades. But those data have often been shoehorned into the categories of the Diagnostic and Statistical Manual of Mental Disorders. Researchers are working to bring diagnostic categories in line with a modern understanding the brain.
