Pasadena Pharmaceutical Company Earns $100 Million in Drug Trial Milestone

Arrowhead Pharmaceuticals, based in Pasadena, has secured a $100 million payment from Sarepta Therapeutics for hitting a key milestone in its early-stage trial of a potential treatment for a rare form of muscular dystrophy.

The payment was triggered after the company successfully enrolled participants in a Phase 1/2 clinical trial and received approval to increase dosage levels.

The drug, known as ARO-DM1, is being developed to treat type 1 myotonic dystrophy, an inherited condition that causes progressive muscle weakness.

If enrollment continues as planned, Arrowhead could get an additional $200 million payout by year’s end.

This milestone is part of a broader partnership struck between Arrowhead and Sarepta earlier this year.

Under the deal, Sarepta paid $500 million upfront and invested another $325 million to acquire Arrowhead stock at a premium.

Arrowhead is also set to receive $250 million over the next five years and is eligible for up to $300 million in short-term bonuses — including the $100 million already earned.

Beyond this, Arrowhead could earn hundreds of millions more in development and sales incentives for each drug the companies advance under the arrangement.

Potential treatments for various rare diseases that affect muscles, the brain, and lungs, some of which are already in clinical trials, are under development while others are approaching readiness for human testing.

Arrowhead remains responsible for making the drugs and supplying them for testing.

Sarepta can propose up to six new disease targets for Arrowhead to explore under the current five-year agreement.

Arrowhead Pharmaceuticals is headquartered in Pasadena.

More details can be found at arrowheadpharma.com.