Longboard Pharma Gets Breakthrough Therapy Nod for Bexicaserin (LP352)

Longboard Pharmaceuticals, Inc., a biopharmaceutical company listed on Nasdaq under the symbol LBPH, has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for its investigational drug, bexicaserin. This designation pertains to the treatment of seizures associated with Developmental and Epileptic Encephalopathies (DEEs) in patients aged two years and older.

Dr. Randall Kaye, the Chief Medical Officer of Longboard, expressed excitement about the FDA's decision, emphasizing that it validates the company's innovative approach to treating DEEs. He highlighted that the designation allows for closer collaboration with the FDA, which will provide guidance to streamline the further development of bexicaserin. With the global Phase 3 program on the horizon, Longboard is gearing up to commence this critical phase of clinical trials later in the year.

Gabrielle Conecker, Executive Director and Co-Founder of Decoding Developmental Epilepsies, also lauded the designation, pointing out its significance for the DEE community. She noted that many individuals with DEEs have lacked access to new medications and opportunities to participate in condition-specific trials. The designation is seen as a step towards greater equity and access for underserved patients and their families, ensuring broader participation in clinical trials and potential treatments.

Breakthrough Therapy designation is designed to accelerate the development and review process for drugs intended to treat serious or life-threatening conditions. To qualify, preliminary clinical evidence must suggest that the drug offers substantial improvement over existing therapies on at least one significant clinical endpoint. This designation not only promises faster development timelines but also involves intensive guidance and a higher level of organizational commitment from the FDA's senior management.

Longboard Pharmaceuticals is dedicated to developing novel, transformative medications for neurological diseases. The company's product candidates are designed to selectively target specific G protein-coupled receptors (GPCRs), based on over two decades of GPCR research. Bexicaserin (LP352), a key focus of Longboard's efforts, is an oral, centrally acting 5-hydroxytryptamine 2C (5-HT2C) receptor superagonist. Notably, it has shown no impact on the 5-HT2B and 5-HT2A receptor subtypes.

Earlier this year, Longboard announced positive topline results from a Phase 1b/2a clinical trial, known as the PACIFIC Study, which evaluated bexicaserin in patients aged 12 to 65 with DEEs, including Lennox-Gastaut syndrome and Dravet syndrome. These promising results set the stage for the upcoming global Phase 3 program.

In addition to bexicaserin, Longboard is working on LP659, an oral, centrally acting modulator of sphingosine-1-phosphate (S1P) receptor subtypes 1 and 5. LP659 is being developed for the treatment of rare neuroinflammatory conditions. The company recently completed a Phase 1 single-ascending dose (SAD) clinical trial of LP659 in healthy volunteers.

Both bexicaserin and LP659 remain investigational compounds and have not yet received marketing approval from the FDA or any other regulatory authority. Nevertheless, the Breakthrough Therapy designation for bexicaserin marks a significant milestone for Longboard Pharmaceuticals and signals hope for patients suffering from DEEs. The company's continued efforts in advancing its clinical programs underscore its commitment to bringing new, effective treatments to the market.

In summary, Longboard Pharmaceuticals is making significant strides in neurological disease treatment, particularly with its innovative drug, bexicaserin. The FDA's Breakthrough Therapy designation accelerates the drug's developmental timeline, offering hope for improved therapies for DEE patients.