Principia Achieves $10 Million in Additional Milestones

Principia Biopharma Inc. recently announced the achievement of $10 million in additional milestones related to successful development activities conducted by Principia as part of the clinical development of PRN2246. PRN2246, also known as SAR442168, is a Bruton’s tyrosine kinase (BTK) inhibitor that crosses the human blood-brain barrier and modulates immune cell function in the brain for the potential treatment of central nervous system (CNS) diseases. PRN2246 is being developed under a collaboration agreement with Sanofi, a company committed to discovering and developing new treatment options for people living with multiple sclerosis (MS).

“Principia is pleased that, with the achievement of these additional milestones, we have now earned, in the aggregate, $25 million in milestones in 2018 related to our successful development efforts for PRN2246,” said Martin Babler, Chief Executive Officer of Principia. “We intend to present the results of our Phase 1 clinical trial at a future scientific conference and we look forward to Sanofi commencing Phase 2 development in MS following Principia’s completion of our remaining Phase 1 activities.”

As previously disclosed, in the Phase 1 clinical trial, PRN2246 was considered generally safe and well-tolerated following both single and multiple dose administration to healthy human volunteers for 10 days. There were no serious adverse events or deaths reported in the trial, and all drug-related adverse events were classified as mild. Importantly, in a dedicated arm of the trial, pharmacologically-relevant cerebral spinal fluid (CSF) exposure to PRN2246 was confirmed, highlighting the potential for PRN2246 to impact B-cell driven inflammation in both the periphery and the CNS.

PRN2246 is being developed to potentially treat MS and other CNS diseases, in part by penetrating the blood-brain barrier and modulating B cells and other immune cells in the CNS. During neuro-inflammation, the number of B cells in the brain increases, which is thought to play a central role in the pathology of MS and other CNS diseases. In late 2017, Principia formed a collaboration with Sanofi under which Principia granted Sanofi an exclusive, worldwide license to develop and commercialize PRN2246. Principia is responsible for completion of Phase 1 activities.

Principia is a clinical-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology. Principia’s proprietary Tailored Covalency platform enables Principia to design and develop reversible and irreversible covalent, small molecule inhibitors with potencies and selectivities that have the potential to rival those of injectable biologics, yet maintain the convenience of a pill. PRN1008, a reversible covalent BTK inhibitor, is being evaluated in a Phase 2 clinical trial in patients with pemphigus, an orphan autoimmune disease, and in a Phase 2 clinical trial in patients with immune thrombocytopenic purpura, a rare hematological disease. PRN2246, a covalent BTK inhibitor which crosses the blood-brain barrier, has completed a Phase 1 clinical trial in healthy volunteers, and has been partnered with Sanofi for development in multiple sclerosis and, potentially, for other diseases of the CNS. PRN1371, a covalent inhibitor of Fibroblast Growth Factor Receptor (FGFR), is being evaluated in a Phase 1 trial in patients with solid tumors. For more information, please visit www.principiabio.com.