PulmoSIM Planning to Bring PAH Therapy, PT001, Into Clinical Testing
PulmoSIM Therapeutics has joined with scientists at National Jewish Health hospital and Brown University to advance PT001, its investigational and potentially disease-modifying therapy for pulmonary arterial hypertension (PAH), into clinical trials.
The partnership follows PT001 being designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status aims to encourage the development of therapies for rare and serious diseases, through benefits such as seven years of market exclusivity if granted approval and exemption from FDA fees.
PulmoSIM plans to file a trial application with the FDA, a mandatory step in obtaining regulatory clearance to begin testing a medicine in people.
“We’re thrilled as we embark on our clinical trials program for PT001, and look forward to applying for the Investigational New Drug Application (IND) very soon,” Jo Varshney, PhD, and the CEO of PulmoSIM, said in a press release.
According to PulmoSIM, PT001 modulates several of the pathways playing a role in PAH. The investigational therapy was designed to more effectively alter the underlying biology that drives the disease, with a goal of becoming the “first curative treatment for PAH.”
“The great benefit of PT001 is its potential to not only ease symptoms but further halt or reverse the progression of PAH,” said Vivek Gupta, PhD, scientific founder of PulmoSIM. “Current treatments for PAH do not stop the underlying drivers of the disease, hence there is an urgent need for therapies that can address the progression checkpoints.”
“PAH is a chronic disease and we recognize the importance of developing a curative treatment,” said Tim Lahm, MD, who is joining National Jewish Health as its director of pulmonary vascular biology. “We are excited to work with the PulmoSIM team and clinical investigator-partner from Brown University to develop PT001 further to show its clinical efficacy.”
PulmoSIM, which opened earlier this year, is the pharmaceutical subsidiary of VeriSIM Life. The parent company also owns BIOiSIM, a platform that uses artificial intelligence to predict the likely benefit of compounds to treat disorders.
“We are leveraging VeriSIM Life’s Virtual Drug Development Engine, BIOiSIM, to prioritize the development of PT001, which, unlike currently available therapeutics, has the ability to stop and reverse the progression of PAH,” Varshney said.