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Intrabio research update – August 2018

We are pleased to share the below from Intrabio and their research into a potential treatment for Tay-Sachs and Sandhoff disease.

I am pleased to share highlights of the significant progress IntraBio Inc. has made during 2018. These regulatory, clinical, and corporate accomplishments have prepared us for planned, multinational clinical studies with N-Acetyl-L-Leucine for Niemann-Pick Type C (NPC), Tay-Sachs disease, and inherited Cerebellar Ataxias. Enrollment is aimed to begin in late 2018 and the trials should be completed by mid- 2019.

Multi-National Clinical Trials
IntraBio has met with regulators in the US and Europe to discuss the clinical trial protocols, including the US Food and Drug Administration (FDA; July 2018), the UK Medicines and Healthcare Products Regulatory Agency (MHRA), the Dutch Medicines Evaluation Board (MEB), and the Spanish Agency of Medicines and Medical Devices (AEMPS).

Based upon the regulators’ feedback, IntraBio will submit applications for Clinical Trial Authorizations (CTA) with select EU national regulatory authorities, and Investigational New Drug (IND) applications in the US to initiate the studies. The trials will be conducted at key specialist centers to clearly establish the safety and efficacy of N-Acetyl-L-Leucine for the treatment of these rare, life-threatening conditions.

IntraBio has partnered with the world’s preeminent orphan-drug regulatory consultants and clinical trial organizations to best ensure the successful completion of the clinical studies. Our regulatory consultants, Granzer Regulatory Consulting, have assisted their clients with developing over 70 orphan drugs in the EU and US and are involved with over 50% of all orphan drug marketing authorizations worldwide. PSR Orphan, a clinical research organization (CRO) which have coordinated over 120 rare disease clinical studies, will be responsible for managing IntraBio’s clinical trials. The Patheon division of Fisher Clinical Services is producing the clinical trial supplies of N-Acetyl-L-Leucine. It is planned to have Thermo Fisher Scientific package, label, and distribute the blinded drug products.

In addition, IntraBio is immensely grateful to receive strong support from over fifteen Niemann-Pick, Tay- Sachs, and Ataxia patient organizations across the US and Europe. These close collaborations have greatly aided the development of N-Acetyl-Leucine as a novel treatment for these disorders with unmet medical needs.

Intellectual Property and Market Exclusivity Protections
N-Acetyl-Leucine has been granted several Orphan Drug Designations (ODD) in 2018 from regulatory authorities in the US and the EU. In addition to the ODD granted to N-Acetyl-Leucine by the FDA for the treatment of NPC (Feb 2018) and GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease; March 2018), the compound has most recently been granted ODD in both the US (July 2018) and EU (August 2018) for the treatment of Spinocerebellar Ataxias (of which there are 40 known subtypes).

These designations carry significant financial benefits in addition to broadening our Intellectual Property rights. With marketing authorization, the designations will provide additional layers of protection (7 years exclusivity US/ 10 years exclusivity EU) to method of use patents (20 years for Lysosomal Storage Disorders, Neurodegenerative Diseases, and Mobility and Cognitive Function in the Elderly) and the data exclusivity or protection provided for a new chemical entity (10 Years in the EU and 5 Years in the US).

IntraBio has also been granted a Rare Pediatric Disease Designation (RPDD) for N-Acetyl-Leucine by the FDA for the treatment of NPC (April 2018). This RPDD makes N-Acetyl-Leucine eligible for, and expedites the request of, a Rare Pediatric Disease Priority Review Voucher (PRV) granted at the time of marketing approval.

In the US, the PRV can be sold or transferred an unlimited number of times and used for a marketing application of a different product. In 2016, four vouchers sold for an average price of $200 million.

Funding Round
In July 2018, IntraBio completed its “Series B” funding round. Based on the current budget, we believe our existing resources will be sufficient to conduct our clinical studies described above.

Clinical Pipeline
In compassionate use programs, preliminary data has demonstrated that N-Acetyl-Leucine has potential clinical efficacy in 19 different indications. Most recently, the first confirmed Lewy Body Dementia patient was successfully treated. This adds to the list of neurodegenerative disorders where N-Acetyl-Leucine has been observed to have clinical benefit (eg. Multiple System Atrophy (Parkinsonism) and Restless Leg Syndrome).

In addition to N-Acetyl-Leucine, IntraBio has made significant progress evolving a robust pipeline of compounds. IntraBio has been granted Orphan Drug Designation in the US and EU for Ursodeoxycholic Acid (UDCA) for the treatment of NPC. A series of NAADP analogues are in the early pre-clinical stage and are being developed as potential scientific tools to elucidate new molecular pathways, and developed as new drugs that create opportunities to treat a wide range of diseases.

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