Biom Therapeutics filed Orphan Drug Designation for BIO017 with the FDA

Published: December 17, 2020

SARASOTA, FL–(December 17, 2020) – Biom Therapeutics announces today that it has applied for orphan drug designation for its lead candidate BIO017. The filing is for indication Angelman Syndrome, rare epilepsy under developmental and epileptic encephalopathies (DEE).

Angelman syndrome (AS) is a complex, heterogeneous, and severe life-long neurodevelopmental disorder that includes developmental delay, sleep disorders, speech and motor impairment, anxiety, and electroencephalography (EEG) abnormalities and seizures. The frequency, severity, and pharmacological intractability of the seizures exact a heavy toll on individuals with AS and their caregivers. It is estimated that the US currently has around 30,000-33,000 AS patients. Despite the considerable impact on individuals and caregivers, no disease-modifying treatments are available.

Biom Therapeutics has developed BIO017 as a treatment for AS. In a well-defined preclinical study published in the reputed Journal of Clinical Investigation, we show that BIO017 (100 mg/kg) treatment attenuated hyperthermia- and acoustically induced seizures in a mouse model of AS. Further, BIO017 has shown positive human safety data in phase 2 studies as a treatment against epileptic patients. Biom is planning to initiate for the pivotal randomized controlled study by Q2 of 2021.

‘With our compelling preclinical data, we are pretty confident that FDA will give us an orphan drug designation for BIO017 as a treatment for Angelman Syndrome patients, a huge unmet medical need. said Bobban Subhadra CEO at Biom Therapeutics. ‘The orphan disease status is vital for our next clinical and commercializing planning’, he added.

About Biom Therapeutics
Biom Therapeutics, a clinical stage biopharmaceutical company, founded by a team of business leaders, scientists and clinicians with a focus on developing therapeutics for hard-to-treat neurological disease. Biom Therapeutics is positioning to be a world-class biotech to improve human health and quality of life by developing innovative drugs for treating and possibly reversing the effects of hard-to-treat diseases such as rare epilepsies and Autism. Biom’s initial focus is on rare pediatric epilepsy such as developmental and epileptic encephalopathies and Angelman syndrome. Our talented team of scientists, scientific advisors, and board directors is working to accelerate breakthrough treatments for patients with significant unmet medical needs.

Investor & Media Contact:
John Allen, President