ISIS-TTRRx, an antisense product, will be evaluated as a treatment for transthyretin amyloidosis.
Isis Pharmaceuticals earned a $5 million milestone payment from GlaxoSmithKline (GSK) tied to the launch of a Phase I study of ISIS-TTRRx in transthyretin amyloidosis. The compound is the first antisense drug to enter development under Isis’ alliance with the pharma giant, which covers six therapeutic programs.
“In a little over a year, we have begun clinical development on the first drug in this collaboration, and we look forward to moving additional drugs from this collaboration into our pipeline,” says B. Lynne Parshall, COO and CFO of Isis. “This year, we anticipate making significant progress in our drugs to treat severe and rare diseases such as transthyretin amyloidosis.”
Parshall notes that Isis has already earned $48 million in payments including $8 million this year since entering into the agreement with GSK to develop RNA therapeutics for rare and infectious diseases. Isis received $35 million up front and has earned $10 million in milestones for ISIS-TTRRx.
Isis is eligible for license and milestone fees totaling nearly $1.5 billion if all six drug programs covered by the deal are successfully developed for one or more indications, then commercialized through to pre-agreed sales targets. On May 16, three days before its announcement of the $5 million payment, Isis said it received $3 million from GSK toward development of the sixth drug.
For each program, Isis is eligible to receive on average up to $20 million in milestone payments up to Phase II. GSK has the option to license compounds at proof-of-concept and will be responsible for all further development and commercialization. In addition, Isis will receive up to double-digit royalties on sales from any product that is successfully commercialized.
ISIS-TTRRx targets transthyretin (TTR) for the treatment of transthyretin amyloidosis, a genetic disease for which the only approved available option is a liver transplant. ISIS-TTRRx will initially be developed for patients with familial amyloid polyneuropathy, or FAP, who have TTR build up in their peripheral nerves and experience the loss of motor functions such as walking.