Alexion (ALXN) In-licenses Amyloidosis Candidate In Japan

 | Sep 09, 2019 11:39PM ET

Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced a license agreement with BridgeBio Pharma, Inc.’s (NASDAQ:BBIO) subsidiary Eidos Therapeutics, Inc. (NASDAQ:EIDX) .

Per the terms of the deal, Alexion will obtain an exclusive license from Eidos to develop and commercialize an investigational, orally-administered small molecule AG10 in Japan. In exchange, the latter will receive an upfront payment of $25 million and an equity investment of $25 million at a premium to the market price from the former upon the deal execution. Also, a possibility of additional milestones and royalty-dependent fees remains.

AG10 has been designed to treat the root cause of transthyretin amyloidosis (ATTR) — destabilized and misfolded transthyretin (TTR) protein — by binding and stabilizing TTR in the blood. We note that Eidos is currently evaluating the candidate in a phase III study in the United States and Europe for ATTR cardiomyopathy (ATTR-CM). Part A of the study will assess the change from baseline in 6-minute walk distance (6MWD) at 12 months while Part B will evaluate reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations at 30 months. This apart, Eidos plans to initiate a phase III study of AG10 for ATTR polyneuropathy (ATTR-PN) in the second half of 2019.

While the deal expands Alexion’s amyloidosis portfolio, it provides Eidos with an opportunity to leverage the company’s substantial development and commercial experience in expanding its AG10 program in Japan.

We remind investors that in January 2019, Alexion entered into an agreement with the biotechnology company Caelum Biosciences, which is developing CAEL-101 for light chain (AL) amyloidosis. CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ functions by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. A phase Ia/Ib study on CAEL-101 has already been completed.

Per the terms of the pact, Alexion acquired a minority equity interest in the preferred stock of Caelum and an exclusive option to also buy the remaining stake in the same based on the phase II data.

Shares of the company have gained 9.3% in the year so far against the .

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