Servier Invests $450M in Kaerus' Fragile X Syndrome Therapy, Expanding Neuroscience Portfolio

French pharmaceutical company Servier has announced a significant investment in Kaerus Bioscience's fragile X syndrome candidate, KER-0193, in a deal worth up to $450 million. This move marks Servier's latest effort to strengthen its neuroscience pipeline and expand its presence in the rare disease market.

KER-0193: A Promising Therapy for Fragile X Syndrome

KER-0193 is an investigational small-molecule drug designed to modulate BK channels, calcium-activated ion channels that play a crucial role in central nervous system function. The therapy aims to address the underlying mechanisms of fragile X syndrome, the most common genetic cause of autism spectrum disorder.

Kaerus Bioscience, a U.K.-based startup, has demonstrated promising results in preclinical studies, showing improvements in behavioral, sensory, and cognitive symptoms related to fragile X syndrome. In March, the company completed a Phase I study of KER-0193, reporting "significant pharmacodynamic effects" on relevant parameters of brain activity.

The potential of KER-0193 has been recognized by the U.S. Food and Drug Administration (FDA), which granted the therapy both orphan drug and rare pediatric drug designations for fragile X syndrome in May.

Servier's Strategic Expansion in Rare Diseases

Under the terms of the agreement, Servier will acquire KER-0193 and assume responsibility for its further development. The company plans to launch a Phase II study in 2026, targeting patients in both America and Europe.

This deal follows closely on the heels of Servier's recent collaborations in the rare disease space. Just last week, the company invested $210 million upfront in a partnership with IDEAYA Biosciences to advance darovasertib, a kinase inhibitor for uveal melanoma. Earlier this year, Servier also licensed Black Diamond's BDTX-4933 for solid tumors in a deal worth up to $780 million.

Addressing an Unmet Medical Need

Fragile X syndrome affects approximately 1 in 7,000 males and 1 in 11,000 females worldwide. The condition is characterized by cognitive and developmental delays, behavioral problems, and various mental health issues. Currently, there are no approved treatments specifically targeting the underlying cause of fragile X syndrome, highlighting the significant unmet medical need that KER-0193 aims to address.

As Servier continues to expand its neuroscience portfolio, the acquisition of KER-0193 represents a strategic investment in innovative therapies for rare genetic disorders. The development of this promising treatment could potentially offer new hope to patients and families affected by fragile X syndrome.