Verseon has announced the launch of a drug program developing oral treatments for hereditary angioedema (HAE), a rare, life-threatening genetic disease. In their interim report published yesterday, the Company presented data from a well-established HAE disease model which demonstrate the efficacy of their plasma kallikrein inhibitors in reducing swelling.
HAE is a rare genetic condition affecting approximately 1 in every 50,000 people. The disease is caused by low levels or improper function of C1 inhibitor, a serine protease inhibitor needed to regulate vasodilation and edema. As a result, HAE patients suffer from recurring episodes of severe swelling (edema), which can affect the face, extremities, intestinal tract, and airways. Upper airway edema is considered especially dangerous and can even be life-threatening.
An oral therapeutic would be a life-changing development for HAE patients who currently rely on treatments administered via intravenous or subcutaneous injections. Verseon is building on expertise established in its diabetic macular edema program to develop small-molecule plasma kallikrein inhibitors suitable for oral dosing. Plasma kallikrein is an established target central to the HAE disease pathway, which is used in existing HAE treatments, including Shire’s injectable HAE drug Kalbitor.
Verseon has developed a range of plasma kallikrein inhibitors using its computationally driven drug discovery platform. Many of these inhibitors have demonstrated good oral pharmacokinetic exposure, which is critical for convenient once-a-day oral dosing. In their interim report, Verseon presented data on a lead candidate that reduces swelling with efficacy comparable to a positive control in a carrageenan-induced paw edema model, a standard preclinical model for HAE.
“We are excited about the promising preclinical results for our hereditary angioedema candidates. We are continuing to optimize our drug candidates for oral bioavailability, efficacy, and safety to develop an effective and convenient treatment, which has the potential to change the standard of care for HAE patients,” Dr. David Kita, Vice President of R&D at Verseon, said.
In the interim report, Verseon also presented substantial progress on its other drug programs. The Company is finalizing phase I preparations for the first development candidate in its anticoagulation program and has recently presented promising efficacy results in their diabetic macular edema program.