Indian startup develops drug for key orphan diseases, partners US firm

Bengaluru: In an announcement made on 5th February 2018, the Oraxion Therapeutics, a spin-off from Bangalore based Aten Porus Lifesciences has informed that the company has entered into an option agreement with a US-based biopharmaceutical company.

The agreement provides the biopharma partner with the exclusive option to license its lead asset ORX-301 for the treatment of Niemann-Pick Type C disorder (NPC) and Focal Segmental Glomerulosclerosis (FSGS). The total aggregate payments under the agreement have the potential to reach upwards of $125 million as well as typical royalties and associated sales milestone payments.

“We are excited to be working with a partner that shares our vision to develop ORX-301 for NPC and FSGS. Our partner has expertise in rare diseases, with deep clinical development, regulatory experience and related capabilities. We look forward to seeing these promising programs yield important drugs that will benefit underserved patients around the globe,” said Arun B Papaiah, Founder and Chief Executive Officer, Oraxion Therapeutics, Inc. “We would also like to acknowledge the support from the Biotechnology Industry Research Association Council (BIRAC), an initiative of the Department of Biotechnology, Government of India, for initial grant support for our work,” he added.

Niemann-Pick Type C disorder is a rare lysosomal storage disorder characterized by excess accumulation of cholesterol in organs, specifically, the spleen, liver and brain. NPC is almost always fatal with currently no clinically approved treatment and it affects 3000 patients worldwide. Focal Segmental Glomerulosclerosis is a rare disease that attacks the kidney’s filtering units (glomeruli) causing serious scarring which leads to permanent kidney damage and even failure. FSGS affects greater than 50,000 patients in the US and 5000 new patients diagnosed every year and is one of the causes of a serious condition known as Nephrotic Syndrome.

“We have confirmed the therapeutic efficacy of our lead compound, ORX-301, in the relevant preclinical models of these diseases and now plan to transfer the asset to our partner for clinical development,” said Aditya Kulkarni, Ph.D., Founder and Chief Scientific Officer, Oraxion Therapeutics, Inc.

“We’re continually looking for opportunities to advance critical therapies to treat underserved disease targets such as NPC and FSGS, through novel technology, like ORX-301,” said Tom Johnston, Chief Business Officer, Oraxion Therapeutics, Inc.

“This deal is a validation of our science and our platform and will allow us to expand our pipeline of treatments with the potential to improve other health outcomes in areas of unmet medical need,” said Srinivasan Namala, Founder and Director, Oraxion Therapeutics, Inc.