Arrowhead Pharmaceuticals has entered a worldwide licensing and partnership agreement with Sarepta Therapeutics to develop treatments for rare genetic diseases.
Arrowhead will receive an $825m payment: $500m in cash and a $325m equity investment at a 35% premium.
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It is also set to receive $250m, disbursed in equal instalments over five years, and qualifies for an extra $300m in forthcoming payments linked to the enrolment of certain cohorts in a Phase I/II trial.
The company also stands to gain royalties on commercial sales and up to $10bn in potential future milestone payments.
The collaboration encompasses clinical and preclinical programmes targeting rare diseases affecting the muscles, lungs and central nervous system (CNS).
Sarepta can propose up to six new targets for which Arrowhead will perform discovery and preclinical development activities leveraging the Targeted RNAi Molecule (TRiM) platform.
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By GlobalDataSarepta will receive an exclusive licence for those programmes and handle activities related to their clinical development and commercialisation.
The deal could yield development milestone payments for Arrowhead ranging from $110m to $410m, and sales milestone payments from $500m to $700m, per programme.
Additionally, Arrowhead is entitled to tiered royalties on commercial sales.
Sarepta’s president and CEO Doug Ingram is set to join the Arrowhead board of directors.
Arrowhead is tasked with manufacturing clinical drug supply for all programmes under the licence and collaboration, as well as commercial drug products for the four programmes that are in studies at present.
The completion of the transaction is anticipated in early 2025, contingent upon the expiration or termination of the waiting period mandated by the Hart-Scott-Rodino Antitrust Improvements Act of 1976, along with other standard conditions.
Arrowhead Pharmaceuticals CEO and president Christopher Anzalone stated: “We estimate that this transaction extends Arrowhead’s cash runway into 2028 and potentially through multiple new drug launches, including wholly owned and partnered programmes.
“We now turn our focus as a company to launching investigational plozasiran for the treatment of familial chylomicronaemia syndrome potentially in 2025, pending FDA review and approval, which would be our first commercial product.”
In September 2024, the US Food and Drug Administration granted breakthrough therapy status to Arrowhead’s plozasiran.
