Benzinga | Dec 7, 2021 04:41AM EST

Ionis Announced Collaboration With AstraZeneca to Develop and Commercialize Eplontersen; Ionis to Receive $200M Upfront, Up to $485M in Development and Approval Milestones, Up to $2.9B in Sales-related Milestone Payments

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), the leader in RNA-targeted therapies, today announced it has entered into a strategic collaboration agreement with AstraZeneca to develop and commercialize eplontersen, Ionis' investigational antisense medicine for the treatment of transthyretin amyloidosis (ATTR).

Eplontersen, formerly known as IONIS-TTR-LRx, is designed to reduce the production of transthyretin, or TTR protein, to treat ATTR, a systemic, progressive and fatal disease.It uses Ionis' advanced LIgand-Conjugated Antisense (LICA) technology.

The companies will develop a global strategy for developing, manufacturing and commercializing eplontersen. A breakdown of responsibilities of the collaboration includes, but is not limited to:

Ionis will continue to lead the conduct of the global Phase 3 clinical trials in patients with hereditary ATTR amyloidosis (hATTR) with polyneuropathy (NEURO-TTRansform) and cardiomyopathy (CARDIO-TTRansform).Ionis will manufacture and supply eplontersen for the ongoing clinical trials and process qualifications. AstraZeneca will be responsible for commercial supply, with transition timing to be agreed by both companies.Ionis and AstraZeneca will have shared responsibility for medical affairs and commercial activities in the U.S.AstraZeneca will have an exclusive license for eplontersen outside the U.S. except certain countries in Latin America."We believe that bringing together Ionis' industry-leading experience in RNA-targeted therapeutics and deep knowledge of the TTR amyloidosis market with AstraZeneca's global scale and leadership in cardiovascular drug development and commercialization will enable faster and deeper market penetration for the benefit of patients," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "In addition to being the best strategy to maximize patient and shareholder value for eplontersen, this agreement also represents a key step in bolstering our commercial organization as we prepare to launch multiple products."

Hereditary ATTR amyloidosis (hATTR) with polyneuropathy is expected to be the first indication for which the companies will seek regulatory approval for eplontersen, with the potential to file a new drug application (NDA) with the U.S. Food and Drug Administration by the end of 2022.

Under the terms of the agreement, Ionis will receive a $200 million upfront payment, up to $485 million in development and approval milestones, and up to $2.9 billion in sales-related milestone payments. The collaboration includes territory-specific development, commercial and medical affairs cost-sharing provisions. Ionis is also eligible to earn royalties in the range of low double-digit to mid-20s percentage depending on region.

The agreement is expected to become effective by the end of 2021, subject to the satisfaction of requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 1976.