Nido Biosciences has announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to NIDO-361, a novel small molecule being developed for the treatment of Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy’s disease. This designation underscores the urgent need for effective treatments for this rare and debilitating neuromuscular disorder, as there are currently no approved therapies in the EU or the US. The company has completed enrollment in its Phase 2 clinical trial evaluating NIDO-361.
The EMA's Orphan Drug Designation provides several key benefits to Nido Biosciences, including the opportunity to seek scientific advice from the agency, eligibility for priority review of the marketing authorization application, and reduced regulatory fees. These incentives are designed to encourage the development of treatments for rare diseases that affect a small number of people.
Phase 2 Trial Details
The Phase 2 trial, which has fully enrolled 54 patients across the European Union, United Kingdom, and South Korea, is designed to assess the efficacy and safety of NIDO-361. The primary endpoint of the study is the change from baseline in total and thigh lean muscle volume, as measured by MRI. Secondary endpoints include changes from baseline in the SBMA functional rating scale, 6-minute walk test, and grip strength assessment. Patients receive a single oral dose of NIDO-361 daily, with the dosage continually reviewed to determine optimal levels for subsequent Phase 3 trials.
Mechanism of Action and SBMA
SBMA is a rare inherited X-linked neuromuscular disorder caused by a genetic mutation of the androgen receptor (AR). This mutation leads to the loss of skeletal muscle and motor neuron function, resulting in progressive weakness and wasting of limb, facial, and swallowing muscles. NIDO-361 is designed to bind to a distinct site on the androgen receptor, correcting transcriptional dysregulation and restoring healthy cell function.
“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.
Nido Biosciences is focused on translating neuroscience breakthroughs into treatments for severe neurological diseases. Their approach involves leveraging human genetics to develop precision medicines that address the fundamental biology of disease and restore healthy cell function. In addition to NIDO-361, Nido Bio is developing other pipeline programs targeting multiple disease mechanisms with broad clinical application across neurodegenerative and peripheral inflammatory diseases.
