Genea Biocells Announces FDA Orphan Drug Designation for GBC0905 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

May 31, 2018 |

5 min read

This small molecule agent potently suppresses DUX4 function, the molecular cause of the disease.

SAN DIEGO--(BUSINESS WIRE)-- Genea Biocells, a preclinical-stage company focused on drug development to treat neuromuscular diseases, today announced that it has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its therapeutic candidate, GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD). This small molecule agent potently suppresses DUX4 function, the molecular cause of the disease. FSHD is one of the most common muscular dystrophies and results in progressive muscle weakness.

“We are very pleased that the FDA granted Orphan Drug Designation to GBC0905 as an accomplishment for our team and an important milestone in our journey toward bringing our lead product candidate to the clinic. We anticipate GBC0905 to be the first targeted therapy for patients suffering from FSHD, a relentless and debilitating disease,” said Dr. Uli Schmidt, President of Genea Biocells. “The Orphan Drug Designation also validates our skeletal muscle technology which is a highly useful platform for modeling any number of other myogenic indications as we expand our pipeline.”

Genea Biocells’ lead scientist for the FSHD program, Amanda Rickard, added, “Our targeted therapeutic candidate GBC0905 potently suppresses DUX4 function, prevents the activation of DUX4 target genes, and protects affected skeletal muscle cells in a dose-dependent fashion and, importantly, without affecting normal myogenesis. Aberrant DUX4 expression is the underlying cause of the disease and results in muscle fiber death. The FSHD field generally believes that blocking DUX4 will be curative. We are looking forward to thorough and expeditious development of this much-needed therapy.”

Currently, Genea Biocells is seeking capital to advance GBC0905 to clinical trials, complete preclinical work on their Spinal Muscular Atrophy (SMA) program, and expand the pipeline to other orphan muscle diseases. Under the U.S. Orphan Drug Act, the FDA grants Orphan Drug Designation status to novel therapeutics for rare diseases, defined as those affecting fewer than 200,000 people in the U.S. All of Genea Biocells’ product candidates are ultimately expected to benefit from Orphan Drug Designation. Advantages include tax credits for qualified clinical trials costs and seven years of market exclusivity upon regulatory product approval.

About FSHD

Facioscapulohumeral Dystrophy (FSHD) is an inheritable muscle disease affecting up to 1:8,000 people. There is no cure or treatment strategy for patients with FSHD. This debilitating disease slowly consumes skeletal muscle, robbing people of the active, healthy, and independent years of their lives. It is caused by the sporadic de-repression of a toxic gene, DUX4, in the muscles of FSHD patients.

About Genea Biocells

Genea Biocells US Inc. is a neuromuscular disease-focused preclinical stage company based on a proprietary technology platform for the accelerated development of drugs to treat neuromuscular diseases - typically rare orphan diseases that present an area of particularly high unmet medical need with patients in many cases being children for which no treatment options exist. Genea Biocells has developed a proprietary skeletal muscle differentiation method used to generate disease affected skeletal muscles for disease modeling and assay development. Genea Biocells’ screening approach makes use of a highly curated repurposed chemical library of compounds with favorable toxicity profiles, paving an accelerated path for regulatory approval of lead compound hits. Genea Biocells is based in San Diego, California and originated from the Australian Genea group, a public, unlisted company that has been operating world leading IVF clinics since 1985. Genea Biocells’ FSHD program was supported by FSHD Global and Friends of FSH Research in the past and the company works closely with the FSH Society now.

For more information, visit geneabiocells.com or contact: info@geneabiocells.com

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