Pharmosa Biopharm announced on the 16th that its North American licensing partner, Liquidia, will present the mid-term analysis results of the Phase III clinical trial of the new formulation drug L606 for treating PAH (Pulmonary Arterial Hypertension) at the American Thoracic Society (ATS) conference. The current results show positive outcomes, with participants successfully transitioning from Tyvaso to L606 treatment, demonstrating tolerability when used in combination with other medications and showing no significant side effects.
This interim clinical achievement, besides aiding in the planning for subsequent clinical advancements, also positively impacts the ongoing negotiations for regional licensing outside North America. CEO Gan Pei reiterated that the goal of completing licensing agreements this year remains unchanged.
Pharmosa Biopharm stated that the etiology of PAH is unclear, and its initial diagnosis is challenging, posing fatal risks without timely treatment. Gan Pei pointed out that PAH is a highly complex rare disease. Although over ten therapies have been approved, because of their distinct pharmacological actions and response pathways, clinical treatment guidelines recommend adopting combination therapies involving two or three pathways to provide patients with more beneficial treatment. However, to date, there has been no other more effective treatment, indicating unmet medication needs.
Gan Pei further stated that among the currently approved PAH therapies, there are three standard therapies based on prostacyclin, endothelin, and nitric oxide pathways. Prostacyclin pathway drugs have a market size of $3 billion, accounting for 50% of the total market and are predominant. These drugs come in oral, inhaled, and continuous injection formulations, with sales of approximately $2 billion, $500 million, and $500 million, respectively, in the past year.
Pharmosa Biopharm's self-developed L606 belongs to the prostacyclin class, which is an inhaled new formulation drug encapsulated in liposomes containing treprostinil, compared to Tyvaso by United Therapeutics (UT). According to the clinical data presented, the daily tolerable dose of L606 for patients is approximately two to three times that of Tyvaso. Additionally, while Tyvaso requires dosing every four hours, L606 only needs to be administered twice a day, providing continuous drug coverage throughout the day and reducing upper respiratory tract irritation and fluctuations in drug concentration that can cause side effects.
Gan Pei pointed out that in March of this year, the U.S. FDA re-approved two new PAH treatments: Sotatercept by Merck and Opsynvi by Actelion. Sotatercept, a novel mechanism drug administered via injection every three weeks, aims to enhance exercise capacity and reduce the risk of disease progression when used in conjunction with existing therapies. Opsynvi by Actelion is a combination tablet integrating two existing oral medications (macitentan and tadalafil) to prolong the sales lifecycle of Opsumit, representing a non-innovative mechanism drug.
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