Rare Daily Staff

PTC Therapeutics and Akcea Therapeutics said they will collaborate to commercialize two of Akcea’s rare disease drugs in Latin America, a deal that will provide Akcea with $18 million in upfront payments.

The first drug, Akcea’s Tegsedi, won marketing authorization approval from the European Commission for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis. Approval is pending in the United States and Canada.

The second drug, Waylivra, is under regulatory review in the United States, Europe, and Canada for the treatment of people with familial chylomicronemia syndrome. Waylivra recently received a positive vote from the U.S. Food and Drug Administration’s Division of Metabolism and Endocrinology Products Advisory Committee and the agency is expected to act on the company’s application to market the drug by the end of August. Waylivra is also in clinical development for familial partial lipodystrophy.

Under terms of the agreement, PTC will gain exclusive rights to Tegsedi and Waylivra in Latin America and certain Caribbean countries. PTC will pay Akcea an upfront licensing fee of $18 million, $12 million which is due on signing and $6 million of which will be paid on the earlier of FDA or EMA approval of Waylivra.

In addition, PTC will pay Akcea regulatory milestones in the PTC territory up to a total of $8 million. Akcea is also eligible to receive royalties from PTC in the mid-20 percent range on net sales of each drug in the PTC territory. PTC’s obligation to pay Akcea royalties begins on the earlier of 12 months after the first commercial sale of a product in Brazil or the date that PTC recognizes revenue of at least $10 million in Latin America.

The collaboration will be governed by a joint steering committee with representation from both parties. Akcea gained the global rights to Tegsedi and Waylivra through licenses from Ionis. Milestone payments and royalties that Akcea receives from PTC for Tegsedi will be split 60 percent to Ionis and 40 percent to Akcea. All Waylivra milestone payments and royalties that Akcea receives from PTC will be split evenly with Ionis.

“Our decision to partner with PTC to accelerate commercial access for patients in Latin America reflects our commitment to bringing Tegsedi and Waylivra to patients as rapidly as possible,” said Paula Soteropoulos, CEO of Akcea Therapeutics. “PTC’s established rare disease team in Latin America has a proven record of success in patient identification, in physician and patient education and support programs, and in efficiently obtaining market access.”

August 2, 2018
Photo: Paula Soteropoulos, CEO of Akcea Therapeutics

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