Alto Neuroscience Announces Positive Phase 1 Results for ALTO-101, a Novel PDE4 Inhibitor in Development for Schizophrenia

Alto Neuroscience, Inc. (“Alto”) (NYSE: ANRO) today announced positive results from its healthy volunteer Phase 1 study of ALTO-101, a novel PDE4 inhibitor in development for cognitive impairment associated with schizophrenia (CIAS). Results from the study demonstrated favorable tolerability and improved pharmacokinetics of ALTO-101 when administered via a transdermal delivery system (TDS) compared to oral administration. The TDS formulation is being developed in partnership with MEDRx. The drug exposure levels achieved with the transdermal formulation were significantly greater than systemic exposure with oral administration while also reducing typical class-wide adverse events. The Company plans to initiate a proof-of-concept study evaluating ALTO-101 in patients with CIAS in the first half of 2024, with topline data expected in the second half of 2025.

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The pharmacokinetic curve illustrates stable drug exposure and achievement of the desired plasma concentration with the transdermal formulation as compared to orally administered ALTO-101. (Graphic: Alto Neuroscience, Inc.)

“These positive results provide a strong indication that ALTO-101 could become an important therapeutic option for a broad range of indications,” said Amit Etkin, M.D., Ph.D., founder and chief executive officer of Alto Neuroscience. “Oral PDE4 inhibitors have shown promise as pro-cognitive therapeutics but are associated with burdensome and dose-limiting side effects. Despite these dosing and tolerability challenges, PDE4 inhibitors are used widely in other non-CNS indications, often with sub-optimal dosing regimens. We have now demonstrated transdermal administration of ALTO-101 results in significantly greater, stable drug concentration with a once-daily formulation that is well-tolerated. We believe the reduction in typical class-wide adverse events observed with ALTO-101 provides a significant point of differentiation compared to other PDE4 inhibitors currently available, or in development. We look forward to advancing this product candidate for patients with CIAS who currently have few treatment options.”