Rare Daily Staff

Eiger BioPharmaceuticals said it has expanded its licensing agreement with Merck to include rights to develop the experimental drug lonafarnib for the treatment of progeria, a rare and fatal genetic condition characterized by accelerated aging in children.

Progeria, also known as Hutchinson-Gilford Progeria Syndrome, is caused by a mutation in the lamin A gene, which results in the production of an aberrant protein, progerin. Researchers believe that defective lamin A protein makes the nucleus of the cell unstable. That cellular instability leads to the process of premature aging in Progeria.

The manifestations of progeria include growth failure, loss of body fat and hair, aged-looking skin, stiff joints, hip dislocation, cardiovascular disease, and stroke. Children with the condition live on average to a little more than 14 and die from heart disease typically associated with old age. There are no approved drugs to treat the condition.

Lonafarnib inhibits an enzyme that is involved in the biological pathway that leads to the production of the mutated protein that drives the condition. The U.S. Food and Drug Administration granted Orphan Drug Designation to Lonafarnib for Progeria.

Under the expanded agreement with Merck, Eiger will gain commercial and distribution rights to lonafarnib across the licensed and approved indications in the future. Eiger will be responsible for regulatory execution, commercialization, and distribution activities of lonafarnib for Progeria. Eiger is also preparing to evaluate lonafarnib in a late-stage clinical trial for the treatment of hepatitis delta virus infection.

Separately, Eiger announced that it has completed a collaboration agreement with the Progeria Research Foundation that calls for Eiger, at its own expense, to provide lonafarnib for ongoing clinical trials and expanded access in progeria. Eiger will also be responsible for any potential filing of a regulatory approval to market the drug for progeria indication based on data from the  Progeria Research Foundation.

Merck previously provided lonafarnib free of charge for clinical studies supported by the foundation 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 the foundation emerged.

Eiger plans to seek guidance from the FDA regarding data already generated in multiple clinical studies conducted and completed by the foundation evaluating lonafarnib in progeria.

“Continued patient access to lonafarnib was the fundamental motivation for these agreements,” said David Cory, president and CEO of Eiger.

May 16, 2018
Photo: David Cory, president and CEO of Eiger

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