Early trial success for Dravet syndrome and ALS therapeutic

Hormones in the brain concept

Bloom Science has announced positive topline data from a Phase I clinical trial of BL-001, a potential first-in-class therapeutic the company is developing for Dravet syndrome and amyotrophic lateral sclerosis (ALS).

In the trial, BL-001 demonstrated a favourable safety profile and was well tolerated with no serious adverse events (SAEs) across all four dose cohorts.

“Dravet syndrome is a rare and devastating form of childhood epilepsy in which there is an unmet need for patients whose seizures remain difficult to control and who experience significant side effects with current medications,” said Paolo Baroldi, Chief Medical Officer of Bloom Science.

“BL-001 shows promise as a novel treatment option that can change lives. With these results we plan to proceed into Phase II clinical development with doses we expect to be within the therapeutic window in both Dravet syndrome and ALS.”

BL-001 has also received Rare Pediatric Disease Designation from the US Food & Drug Administration (FDA) for Dravet syndrome.

“By taking a breakthrough approach to therapeutic development via the Gut-Brain Axis, we believe Bloom has the potential to develop completely novel, transformational treatments with superior safety and efficacy profiles that can have a significant impact on the lives of the patients suffering from rare diseases,” said Christopher Reyes, Founder and CEO of Bloom Science.

“This milestone builds on many years of groundbreaking work that validate Bloom’s IrisRx platform and ability to select strains optimised for both safety and activity.”

Based on the preclinical profile together with these Phase 1 results, Bloom intends to advance BL-001 into Phase II studies in both Dravet syndrome and ALS patients in 2024.

Edited by Diana Spencer, Senior Digital Content Editor, Drug Discovery World

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