MONTREAL--(BUSINESS WIRE)--Neurenati Therapeutics Inc., a biotech company focused on rare pediatric diseases, is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to NEU-001, a novel combination therapy utilizing a neurotrophic growth factor, for the treatment of Hirschsprung disease (HD).
NEU-001 is a first-in-class, once-in-a-lifetime treatment administered intrarectally to regenerate the enteric nervous system in newborns with HD. The primary goal is to restore gastrointestinal motility after birth, eliminating the need for pull-through surgery, the current standard of care. This surgery is an invasive, non-curative procedure that is costly and associated with significant complications. Neurenati’s IND-enabling preclinical program for NEU-001 is ongoing with plans to initiate a first-in-human trial by the end of H1 2026.
The FDA’s Office of Orphan Drug Products grants ODD and RPDD to encourage the development of medicines for rare conditions affecting less than 200,000 people in the U.S. The ODD offers several key benefits, including tax credits for qualified clinical trials, exemptions from FDA user fees, as well as eligibility for seven years of marketing exclusivity upon marketing approval. RPDD is granted to therapies targeting serious or life-threatening diseases that primarily affect children. Upon marketing approval, NEU-001 may be eligible for a Rare Pediatric Disease Priority Review Voucher (PRV), which can expedite the FDA review process for a future marketing application offering significant strategic advantages.
“With a U.S. prevalence of about 100,000 cases, and the congenital nature of HD, the FDA’s decision to grant both ODD and RPDD to NEU-001 underscores the strong preclinical proof of concept demonstrated in gold standard animal models. This recognition further validates our approach and reinforces our commitment to advancing NEU-001 through clinical development. We believe that NEU-001 has the potential to be a curative therapy for babies diagnosed with HD, a devastating disease with no approved treatment options," stated Dr. Maxime Ranger, CEO of Neurenati Therapeutics. “We hope for a cure, one child at a time.”
Extended Seed financing. Neurenati is pleased to announce the successful closing of $1.7M in extended seed financing led by Genson Capital and Invest Quebec, mandated by the Government of Quebec, through its Impulsion PME program, with additional support from new and existing angels. The funding will provide sufficient capital to pursue the cGMP production of the neurotrophic growth factor, initiate the IND-enabling preclinical studies for NEU-001 and expand Neurenati’s core team.
Appointment of Chief Development Officer. Neurenati is pleased to announce the appointment of Dr. Meriam Kabbaj, DPharm, PhD, as Chief Development Officer. In this role, Dr. Kabbaj will lead the company’s overall R&D strategy, including regulatory affairs, preclinical and clinical development of NEU-001. Dr. Kabbaj brings extensive experience in drug development, particularly in rare pediatric diseases. Prior to joining Neurenati, she served as Chief Technology Officer at Genfit S.A., following its acquisition of Versantis, a Switzerland-based company she co-founded and led as Chief Operations Officer.
Dr. Kabbaj holds a Doctor of Pharmacy (DPharm) from the University of Geneva and a PhD in Pharmacology from the Université de Montréal.
ABOUT NEURENATI
Neurenati Therapeutics is a Québec-based biotech company dedicated to developing innovative therapies for rare diseases. Its lead program focuses on Hirschsprung disease (HD), a life-threatening congenital gastrointestinal (GI) disorder caused by the absence of nerve cells in parts of the lower GI tract. Neurenati is advancing a first-in-class combination therapy incorporating a growth factor, aiming to eliminate the need for surgery and minimize associated complications.
Contacts
MEDIA CONTACT
Maxime Ranger, PhD MBA
President and CEO
Neurenati Therapeutics Inc
E: maxime@neurenati.com
T: +1.514.825.9035
