BOSTON--(BUSINESS WIRE)--Nido Biosciences (Nido Bio), a clinical stage company developing precision medicines for debilitating neurological diseases, announced today that the European Medicines Agency (EMA) has granted Orphan Drug Designation to their lead clinical candidate NIDO-361, a novel small molecule in development for the treatment of Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy’s disease. This designation is awarded as the company has completed enrollment for a phase 2 study of the drug.
The EMA’s designation illustrates the lack of satisfactory treatment options for patients with the disease. It enables the company to seek scientific advice as it continues development of NIDO-361 and allows for priority review and reduced regulatory fees, amongst other benefits.
The company has fully enrolled 54 patients in the Phase 2 trial being carried out in the European Union, United Kingdom and South Korea. The study will assess changes from baseline in total and thigh lean muscle volume (measured by MRI) as the primary endpoint, with secondary clinical endpoints including change from baseline in the SBMA functional rating scale, 6-minute walk test, grip strength assessment among others. Patients will receive a single oral dose of NIDO-361 daily. This dosage will be continually reviewed throughout the study to assess optimal dosing levels, informing subsequent Phase 3 trials.
“There are no treatments currently approved for SBMA in the EU or the US, so the Orphan Drug Designation for NIDO-361 granted by the EMA marks an important step towards developing a new medicine that can transform the lives of patients suffering from this rare and debilitating neuromuscular disease,” said Vissia Viglietta, M.D., Ph.D., CMO of Nido Bio. “At Nido Bio, we are at a critical juncture with a fully enrolled phase 2 study for this program, and we are grateful for the opportunities that this designation provides as we eye the next stages of its advancement.”
About Spinal and Bulbar Muscular Atrophy (SBMA) and NIDO-361
SBMA, also known as Kennedy’s disease, is a rare inherited X-linked neuromuscular disorder caused by a genetic mutation of the androgen receptor (AR) that results in the loss of skeletal muscle and motor neuron function. Manifesting in men, SBMA causes progressive weakness and wasting of limb, facial and swallowing muscles, which results in impaired mobility, speech, and swallowing. NIDO-361 is a novel small molecule that binds to a distinct site on the androgen receptor and corrects transcriptional dysregulation to restore healthy cell function.
About Nido Biosciences
Nido Bio is translating today’s neuroscience breakthroughs into tomorrow’s treatments for severe neurological diseases. Leveraging human genetics, we develop precision medicines that address the fundamental biology of disease and restore healthy cell function. NIDO-361, our clinical-stage candidate, is a treatment for Spinal and Bulbar Muscular Atrophy which is a rare and debilitating neuromuscular disease. Additional pipeline programs center around a novel target with the potential to address multiple disease mechanisms and that has broad clinical application across neurodegenerative and peripheral inflammatory diseases. We are creating a sustainable pipeline for the company by utilizing a functional genomics discovery platform based on human cell lines to identify novel targets. Through our approach we seek to transform patient lives in meaningful ways. www.nidobio.com.
