United Therapeutics and Ascendis Pharma signed a collaboration agreement to use the latter’s TransCon drug formulation and delivery platform in the development of a controlled-release, long-acting formulation of United Therapeutics’ pulmonary arterial hypertension drug (PAH) treprostinil, which patients could self-administer. The drug, tradenamed Remodulin®, is currently administered via a continuous infusion pump. Under terms of the collaboration United Therapeutics also has exclusive rights to develop prostacyclin, prostacyclin analog, and prostacyclin-related products for treating PAH using the TransCon technology, and will retain worldwide commercial rights to all relevant products.
Ascendis’ TransCon platform is a prodrug technology that enables the transient conjugation of peptides, proteins, and small molecules to various carriers. The linker families are a diverse group of structures with inherent chemical self-cleaving properties, making drug release independent of enzyme activity and tissue conditions. Key to the platform is the reversibility of conjugation, which means the drug molecule can be released unmodified in a precise, time-controlled fashion. The firm says conventional conjugation technologies are unable to achieve this type of slow-release mechanism because the carrier molecule, such as PEG, can’t be uncoupled from the drug.
“Preclinical studies in multiple animal models with a lead compound of TransCon-linked treprostinil have already produced encouraging results, demonstrating a sustained relase profile supportive of once-daily injection,” states Roger Jeffs, Ph.D., United Therapeutics’ president and CEO. “The application of TransCon technology to treprostinil may also minimize injection site pain and reactions that frequently occur with continuously infused subcutaneous Remodulin.”
Ascendis' in-house pipeline includes TransCon-formulated growth hormone, paliperidone, pramipexole, interferon alpha, and Factor IX candidates. The firm is also working with Sanofi to develop TransCon-based insulin and GLP-1 drugs for diabetes therapy.