Treprostinil

NEWARK, Calif.--(BUSINESS WIRE)--Corsair Pharma, Inc., a private biopharmaceutical company, today announced the successful completion of its first-in-human Phase 1 clinical trial evaluating the TRX-248 Transdermal System, a once-daily transdermal patch designed for the treatment of pulmonary arterial hypertension (PAH). “We are excited by these first-in-human results demonstrating steady, therapeutically meaningful treprostinil levels using our proprietary prodrug and transdermal technology,” said Dr. Bobby Singh, President and COO, Corsair Pharma. The positive results demonstrated that the TRX-248 Transdermal System achieved desired, therapeutically relevant, steady and continuous blood levels of treprostinil. The findings support transdermal delivery as a potentially superior alternative to existing treprostinil administration methods and provide a foundation for advancing the program into further clinical development. Prostacyclins remain a standard of care drug class for the treatment of PAH. Corsair’s proprietary transdermal patch is designed to provide steady and continuous systemic exposure to treprostinil by administering an inactive prodrug, TRX-248, which is absorbed through the skin, enters systemic circulation, and is rapidly converted to active treprostinil by the liver. The once-daily patch is designed to offer a convenient, noninvasive alternative that may also have potential applications in other forms of pulmonary hypertension. Phase 1 Trial Overview The Phase 1 clinical trial evaluated the safety and pharmacokinetics (PK) of treprostinil following single-dose administration of the TRX-248 Transdermal System. The study enrolled nine female volunteers, each of whom received a single patch application designed for 24-hour wear per treatment period. Key findings include: Steady and continuous treprostinil plasma levels over 24 hours with low peak-to-trough fluctuation, Pharmacokinetic profiles comparable with published data for subcutaneous delivery, Data supporting dose proportionality across patch sizes, which would enable ease of dose titration, and Acceptable safety and skin tolerability. “We are excited by these first-in-human results demonstrating steady, therapeutically meaningful treprostinil levels using our proprietary prodrug and transdermal technology,” said Dr. Bobby Singh, President and COO, Corsair Pharma. “These data support the potential of our transdermal platform as a clinically meaningful alternative to existing treprostinil delivery modalities.” “These Phase 1 results are exciting and reinforce our prior preclinical findings,” said George Mahaffey, CEO, Corsair Pharma. “The prostacyclin drug class generates over $3.3 billion in annual U.S. sales, with treprostinil accounting for about $2 billion. Each current delivery system for treprostinil has significant challenges for patients. These positive results provide further evidence that the Corsair Transdermal System may improve the lives of PAH patients.” About PAH and Treprostinil Pulmonary arterial hypertension (PAH) is a serious, progressive, and ultimately fatal disease that causes shortness of breath and markedly reduces quality of life. In the U.S., approximately 45,000 patients are currently taking treatments labeled for PAH. Current therapies clearly improve function and outcomes, but there remains a tremendous unmet medical need. Importantly, many patients are unwilling or unable to utilize prostanoids, one of the most effective therapies, and Corsair believes that the TRX-248 Transdermal System could provide an important new option for them. Treprostinil is commonly used in the treatment of PAH and exerts its pharmacological effects through vasodilation, reduced platelet aggregation, and inhibition of smooth muscle proliferation. Treprostinil is currently marketed by United Therapeutics and Liquidia in different forms to alleviate symptoms, maintain or improve functional class, delay disease progression, and enhance quality of life in patients with PAH. The U.S. prostacyclin market for PAH is estimated at approximately $3.3 billion annually. About Corsair Pharma, Inc. Corsair Pharma is developing transformative solutions to improve the therapeutic profile of medications and provide superior treatment options for patients. The company is focused on developing novel prodrugs of treprostinil to treat patients with pulmonary arterial hypertension using a once-daily transdermal patch. The company intends to pursue a 505(b)(2) regulatory pathway, which is a streamlined process to develop new versions of approved drugs. For more information, visit www.corsairpharma.com.

After success with the drug Winrevair, a band of entrepreneurs has found their next challenge in a difficult-to-treat lung disease. On Tuesday, Oorja Bio launched with a $30 million Series A round, all from Westlake BioPartners, to study a new treatment for idiopathic pulmonary fibrosis (IPF) that it licensed from the Korean biotech company NIBEC in an up to $435 million deal. Oorja is led by former Acceleron leaders Sujay Kango and Janethe Pena. In 2021, Merck paid $11.5 billion to buy Acceleron and Winrevair, a pulmonary arterial hypertension treatment. Current therapies for IPF only slow down the disease, but Oorja is seeking to build a treatment that can reverse or improve the lung-scarring condition. The company plans to start a Phase 2 study of its drug this year. “Looking at the biotech landscape, I realized that the fibrotic space was quite unserved, and there was a lot more that could be done,” said Kango, Oorja’s CEO. Kango was most recently CEO at the cell therapy startup Tmunity. After Tmunity sold to Gilead in 2023, Westlake’s founding managing director and Tmunity chair Beth Seidenberg asked him to incubate a new biotech. He took an interest in fibrotic diseases, where scar tissue builds up in the organs. Around that time, Kango’s father was also diagnosed with IPF and then died. “As a caregiver for IPF, I could see firsthand, and I experienced the challenges the patients were facing, the limitations that current therapies were providing,” Kango said. “The gap was finding therapies that would actually halt the disease progression or even repair or remodel the fibrotic tissues.” IPF is a disease that has long challenged drugmakers, but recently new treatments have shown promise. Boehringer Ingelheim last year won FDA approval for the IPF drug Jascayd, which marked the first new treatment for the disease in over a decade. United Therapeutics is also seeking FDA approval of its drug Tyvaso in IPF after reporting encouraging Phase 3 successes. Kango enlisted Pena as chief medical officer and Connie Coulomb as chief business officer, and they scoured the globe for an IPF asset that they believed had the potential to do that. “We started with a large haystack, got into a smaller haystack, and then to get to the needles, we evaluated the novelty of the biology, the pathway, how it would work together with the current drugs,” Pena said. Oorja is developing ORJ-001, a peptide that’s an agonist of the transmembrane protein β1 integrin. The experimental therapy is meant to restore function of alveolar epithelial type 2 cells, which are stem cells important to the repair of the lining of the lung air sacs. At the American Thoracic Society annual meeting on Tuesday, Oorja is sharing preclinical data as well as preliminary safety data in healthy volunteers. Kango said the biotech’s primary goal with the fundraise was to run the Phase 2 trial, but the $30 million raise also “has the ability for us to further advance our mission of building a pipeline.”