SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C.- United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced that the U.S. Food and Drug Administration (FDA) has granted clearance under the company’s Investigational New Drug application to proceed with a clinical trial of Treprostinil-Iloprost SMI.
The phase 1, open-label, single ascending dose study will evaluate the safety, tolerability, and pharmacokinetics of Treprostinil-Iloprost SMI in healthy volunteers. Assuming a successful outcome in the study, United Therapeutics anticipates commencing a subsequent phase 2 study in patients with PAH in 2027.
Treprostinil-Iloprost SMI combines two potent prostacyclin analogues, treprostinil and iloprost. The therapy is intended to be used as needed to provide episodic symptom relief for PAH patients, who may need a bridge between doses of their ongoing prostacyclin therapy due to acute symptom flare-ups.
“Many people living with PAH experience moments when their symptoms flare despite their maintenance medication, and today they have no therapy designed specifically for those moments. Treprostinil-Iloprost SMI is intended to explore whether an as-needed, combination prostacyclin therapy could help fill that gap. FDA clearance to begin clinical testing is an important first step, and we look forward to advancing this program,” said Ravi Patel, PharmD, Senior Vice President, Global Patient Safety and Strategic Clinical Development, United Therapeutics.
“As a company known for pioneering prostacyclin therapies, Treprostinil-Iloprost SMI represents another important advancement in United Therapeutics’ PAH treatment portfolio. It also reflects continued innovation from our product development platform and our culture of running down the corridors of indifference in pulmonary medicine,” said Martine Rothblatt, Ph.D., Chairperson and Chief Executive Officer of United Therapeutics.
Treprostinil-Iloprost SMI is an investigational-stage product, and is not yet approved in any jurisdiction for any indication.
About Pulmonary Arterial Hypertension
PAH is a life-threatening disease that affects the blood vessels in the lungs and is characterized by increased pressure in the pulmonary arteries, due to increased pulmonary vascular resistance in the small precapillary vessels (i.e., the small blood vessels leading from the heart to lungs). The elevated pressure in the pulmonary arteries strains the right side of the heart as it pumps blood to the lungs. This eventually leads to right heart failure and, ultimately, death. PAH is characterized by structural changes in blood vessel walls, aggregation of platelets, and alteration of smooth muscle cell proliferation. PAH affects about 500,000 individuals worldwide, with around 50,000 people affected in the United States. Increases in the number of people diagnosed with the disease have been observed, but due to the rarity of the disease and the complexity of diagnosing it, only a fraction of patients with PAH are treated.
About United Therapeutics
Founded by CEO Martine Rothblatt to discover a cure for her daughter’s life-threatening rare disease, pulmonary arterial hypertension, United Therapeutics transforms the treatment of rare diseases and pioneers alternatives to expand the supply of transplantable organs. From our innovative therapies to our groundbreaking manufactured organs, we are bold and unconventional. We move quickly from scientific theory to practical technologies that can save lives. As a public benefit corporation, even our legal structure reflects our commitments. We serve patients, act with integrity, create long-term shareholder value, and operate with sustainable practices that protect the future we are working to build. Visit us at www.unither.com and follow us on LinkedIn, Facebook, and Instagram.
For Further Information Contact: Investor Inquiries https://ir.unither.com/contact-ir
Media Inquiries [email protected]
Source: United Therapeutics, ir.unither.com. Republished with credit to the original author. Read the original article.
