Collaborations Pharmaceuticals, Inc. Awarded A new $3.9M SBIR Commercialization Readiness Pilot Grant For Batten Disease

The National Institute of Neurological Disorders and Stroke has awarded a $3,953,139 SBIR grant to Collaborations Pharmaceuticals, Inc. for a Rare Disease

RALEIGH, NORTH CAROLINA, USA, March 7, 2024 /EINPresswire.com/ -- “We are very excited and grateful to be awarded this Phase II SBIR Commercialization Readiness Pilot (CRP) Program Technical Assistance and Late-Stage Development grant by the National Institutes of Health / National Institute of Neurological Disorders and Stroke for “Manufacture of an intracerebroventricular Enzyme Replacement Therapy for CLN1Batten Disease” said Collaborations Pharmaceuticals, Inc. (CPI) CEO Dr. Sean Ekins. “This is also the biggest grant we have been awarded to date by the NIH, as the 12th highest NIH funded organization in North Carolina for 2023.”

“CLN1 Batten Disease is an incurable neurodegenerative storage disorder primarily affecting the brain and the retina of children and young adults, leading to dementia, blindness, epilepsy, and early death. There are currently no treatments available (other than palliative therapies) for this fatal disease.”

“Funding from NINDS has previously enabled us and collaborators to demonstrate efficacy for monthly dosing of the rhPPT1 enzyme replacement therapy, rescue of enzyme activity and improved the phenotype in animal models of the disease. We have since worked with a CDMO (STC Biologics) to manufacture the protein. In 2023 we had our preIND meeting with the FDA supported by a team from RTI International.”

“We now propose in this project production of GMP protein, continued development of quality procedures, prepare a clinical trial protocol, and submit an IND in collaboration with STC Biologics, Long Scientific LLC, RTI International and CTI Clinical Trial Services, Inc. respectively, in order to translate this potential treatment to the clinic.”

“We have already obtained an Orphan Drug Designation and rare pediatric disease designation from the FDA for rhPPT1 as a biological product for a “rare pediatric disease” which offers several benefits in future upon FDA approval, including marketing exclusivity for 7 years and the potential to obtain a rare pediatric disease voucher.”

“We are very grateful to the many CLN1 families who have provided their support over the years and we believe are positioned to continue the development of this potential treatment and bring it closer to a clinical trial. We would welcome discussions with potential partners to help bring this to the families and children who are awaiting a treatment”.

About Batten Disease
The infantile onset form CLN1 disease is caused by mutations in the CLN1/PPT1 gene, which codes for the lysosomal enzyme palmitoyl-protein thioesterase-1 (PPT1) resulting in a reduction or absence of enzyme activity. CLN1 disease usually presents between 6 and 24 months of age. It is likely there are many hundreds of children affected by this disease globally.