Biom Therapeutics submits Orphan Drug Designation filing for rare epilepsy disease
SARASOTA, FL, December 31, 2020 /24-7PressRelease/ — Biom Therapeutics announced today that it has applied for an orphan drug designation for its lead drug candidate, BIO017. The filing is for treatment of patients with an indication of Angelman Syndrome, a rare epilepsy that falls 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 these seizures take a heavy toll on individuals with Angelman syndrome and their caregivers. According to recent estimates, the U.S. has about 30,000 to 33,000 AS patients. Despite the considerable impact on individuals and caregivers, no disease-modifying treatments are currently 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, Biom Therapeutics demonstrated that BIO017 (100 mg/kg) treatment attenuated hyperthermia-induced and acoustically-induced seizures in a mouse model of AS. Furthermore, BIO017 has shown positive human safety data in an open-label human clinical study as an appropriate treatment for epileptic patients. Biom Therapeutics plans to initiate preparations for a pivotal randomized controlled study by the third quarter of 2021.
“With our compelling preclinical data, we are very confident the FDA will give us an orphan drug designation for BIO017 to treat Angelman Syndrome patients because they have a huge unmet medical need,” said Bobban Subhadra, CEO at Biom Therapeutics. “The orphan disease status is key for our next clinical and commercialization planning,” he added.
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 diseases. Biom Therapeutics is positioning itself to be a world-class biotech firm that improves 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. The firm’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 hard to accelerate breakthrough treatments for patients with significant unmet medical needs. www.biomtherapeutics.com
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