Eiger BioPharmaceuticals has expanded its licensing agreement with Merck to include rights to develop the investigational farnesyltransferase inhibitor lonafarnib for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria), a rare and fatal genetic condition characterized by accelerated aging in children. The expanded agreement provides Eiger with commercial and distribution rights to lonafarnib across the licensed and approved indications in the future. Concurrently, Eiger announced that it has completed a collaboration agreement with The Progeria Research Foundation (PRF). Eiger, at its sole cost and expense, will provide lonafarnib for ongoing clinical trials and expanded access in Progeria and be responsible for any potential filing of an NDA for the Progeria indication based on PRF data. Eiger plans to seek FDA guidance regarding a potential regulatory approval for lonafarnib in Progeria.
Merck previously provided lonafarnib free of charge for clinical studies supported by PRF in Progeria. Following the transfer of manufacturing technology for lonafarnib from Merck to Eiger in 2015, Eiger continued to provide lonafarnib for investigational use, and a collaboration with PRF emerged. Eiger plans to seek guidance from the FDA regarding data already generated in multiple clinical studies conducted and completed by PRF evaluating lonafarnib in Progeria. Under the agreement with Merck, Eiger will be responsible for regulatory execution, commercialization and distribution activities of lonafarnib for Progeria. Eiger is also preparing to evaluate lonafarnib in a Phase 3 clinical trial for the treatment of hepatitis delta virus (HDV) infection.
"Continued patient access to lonafarnib was the fundamental motivation for these agreements," said David Cory, President and CEO of Eiger. "Eiger will provide lonafarnib for ongoing clinical trials and expanded access in Progeria and work together with PRF to seek regulatory guidance on a pathway toward regulatory approval of lonafarnib for use in children with Progeria."
"Our mission at PRF is to discover treatments and the cure for Progeria, and its aging-related disorders, including heart disease," said Leslie Gordon, MD, PhD, Medical Director and Co-Founder of The Progeria Research Foundation. "In a relatively short time, we have achieved extraordinary progress towards our mission including the Progeria gene discovery in 2003, the first clinical trial in Progeria initiated in 2007, and clinical evidence of a survival benefit for children administered lonafarnib. We are indebted to Merck for supplying lonafarnib free of charge to PRF-supported clinical trials, and for facilitating our new partnership with Eiger. We look forward to collaborating with Eiger as we pursue pathways for regulatory approval of lonafarnib in Progeria."