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Alexion & Caelum Begin Studies on CAEL-101 in AL Amyloidosis

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Alexion Pharmaceuticals, Inc. (ALXN - Free Report) and partner Caelum Biosciences announced the initiation of the phase III program, Cardiac Amyloid Reaching for Extended Survival (CARES), to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care (SoC) therapy in AL amyloidosis.

The CARES program includes two parallel double-blind, randomized, event-driven global phase III studies, which are evaluating the efficacy and safety of CAEL-101 in AL amyloidosis patients who are newly diagnosed and naïve to SoC treatment (cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy). While one study is enrolling approximately 260 patients with Mayo stage IIIa disease, the other study is enrolling approximately 110 patients with Mayo stage IIIb disease. The studies will be conducted at approximately 70 sites across North America, the United Kingdom, Europe, Israel, Japan and Australia.

In each study, participants are being randomized in a 2:1 ratio to receive either CAEL-101 plus SoC or placebo plus SoC once weekly for four weeks. This will be followed by a maintenance dose administered every two weeks until the last patient enrolled completes at least 50 weeks of treatment. Patients will continue follow-up visits every 12 weeks. The primary objective of the clinical program is to assess overall survival, and safety and tolerability of CAEL-101.

Per the company, current treatments address the bone marrow disorder that creates the misfolded amyloid proteins but there are no approved therapies for the significant damage AL amyloidosis causes to the heart and kidney.  CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis.

Long-term survival data from AL amyloidosis patients treated with CAEL-101 in the phase Ia/Ib study showed that 78% were still alive after a median follow-up time of more than three years.

We note that CAEL-101 has received the Orphan Drug designation from both the FDA and the European Medicine Agency as a therapy for patients with AL amyloidosis.

Last year, Alexion entered into a collaboration agreement with Caelum, whereby the former acquired a minority equity interest in the latter and an exclusive option to acquire the remaining equity based on the phase III CAEL-101 data.

Alexion’s shares have gained 0.6% in the year so far compared with the industry’s growth of 1.4%.


The company is looking to diversify its portfolio and reduce dependence on its blockbuster drug, Soliris, through collaborations.

Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) ’s Onpattro has obtained FDA approval for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Akcea Therapeutics, Inc. , an affiliate of Ionis Pharmaceuticals, Inc. (IONS - Free Report) , has also obtained FDA approval of Tegsedi (inotersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Alexion currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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