PulmoSIM Therapeutics Granted Orphan Drug Designation for PT001 by the FDA to Treat Pulmonary Arterial Hypertension

PulmoSIM Therapeutics (PulmoSIM), the pharmaceutical subsidiary of VeriSIM Life, Inc., is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for asset PT001 for the treatment of Pulmonary Arterial Hypertension (PAH). PT001 targets multiple responsible pathways in PAH, to provide a more efficacious curative treatment.

The FDA’s Office of Orphan Products Development (OOPD) grants orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 people in the United States. Orphan drug designation will allow PulmoSIM to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of PT001, a waiver of the Prescription Drug User Fee Act (PDUFA) filing fees, and tax credits in clinical research expenses along with regulatory and strategic assistance.

“We’re thrilled that the FDA has granted this designation for PT001, and look forward to advancing this drug candidate into a clinical trial program very soon,” said Dr. Jo Varshney, DVM, PhD, CEO and Founder at PulmoSIM. “PAH is a devastating disease, and there still remains a high unmet need for more effective and less-burdensome treatment options for patients living with PAH. Leveraging VeriSIM Life’s Virtual Drug Development Engine, BIOiSIM, we will be fast-tracking development of curative therapeutics for rare diseases with highly unmet needs.”

PAH is a progressive and life-threatening disorder characterized by increased pressure in the arteries that carry blood from the heart to the lungs. The increased pressure strains the heart, which can limit physical activity, result in heart failure and reduce life expectancy. It affects 15-50 persons per million within the United States and Europe, and the average survival rate after diagnosis is less than 3 years. Current approved therapies are unable to target the underlying condition, making PAH a ‘rare disease epidemic’ with no long-term treatment option.

“We greatly appreciate the FDA’s support of our efforts to evaluate our PT001 asset for the treatment of PAH,” said Dr. Vivek Gupta, PhD, Scientific Founder at PulmoSIM. “There is an urgent need for new therapies that have the potential to address the underlying cause of PAH, and we are committed to advancing PT001 as a novel approach to treating this debilitating disease. The preclinical results in this indication have been very encouraging. The development of this new therapy will directly improve the standard of care for PAH patients by reducing morbidity and enhancing the quality of life at a reasonable cost.”

Orphan drug designation status is one of many critical milestones for PT001 and we look forward to continuing to work with our partners and advisors as we embark on our proof-of-concept clinical efficacy trials.