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Arrowhead and Sarepta link for treatments of rare genetic diseases

Posted on by Sweet
Arrowhead and Sarepta link for treatments of rare genetic diseases

The collaboration encompasses several clinical and preclinical programs targeting rare diseases affecting the muscles, lungs and central nervous system. Credit: Stock-Asso/Shutterstock.

Arrowhead Pharmaceuticals has entered into a global licensing and partnership agreement with Sarepta Therapeutics to develop treatments for rare genetic diseases.

Arrowhead will receive an $825 million payout: $500 million in cash and a $325 million equity investment at a 35% premium.

It is also expected to receive $250 million, distributed in equal installments over five years, and benefit from an additional $300 million in future payments linked to the enrollment of certain cohorts in a phase I/II trial.

The company is also expected to receive royalties on commercial sales and up to $10 billion in potential future milestone payments.

The collaboration encompasses clinical and preclinical programs targeting rare diseases affecting the muscles, lungs and central nervous system (CNS).

Sarepta can propose up to six new targets for which Arrowhead will carry out discovery and preclinical development activities leveraging the Targeted RNAi Molecule (TRiM) platform.

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Sarepta will receive an exclusive license for these programs and manage activities related to their clinical development and commercialization.

The deal could result in development milestone payments for Arrowhead ranging from $110 million to $410 million, and sales milestone payments of $500 million to $700 million, per program.

Additionally, Arrowhead is entitled to tiered royalties on commercial sales.

Sarepta President and CEO Doug Ingram is expected to join Arrowhead’s board of directors.

Arrowhead is responsible for manufacturing clinical medicines for all licensed and collaborative programs, as well as commercial pharmaceutical products for the four programs currently under study.

The transaction is expected to close in early 2025, subject to the expiration or termination of the waiting period imposed by the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as well as other conditions standard.

Christopher Anzalone, CEO and President of Arrowhead Pharmaceuticals, said: “We believe this transaction extends Arrowhead’s cash flow through 2028 and potentially through multiple new drug launches, including both wholly-owned and partnered programs. .

“As a company, we are now focused on launching investigational plozasiran for the treatment of familial chylomicronemia syndrome, potentially in 2025, pending FDA review and approval, which would be our first commercial product . »

In September 2024, the U.S. Food and Drug Administration granted breakthrough therapy status to Arrowhead’s plozasiran.


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