Health and Healthcare

Alexion Wins Big in Late-Stage Trial

Chris Lange

Published: March 15, 2018 12:25 pm

Last Updated: January 12, 2020 2:23 am

Shares of Alexion Pharmaceuticals Inc. (NASDAQ: ALXN) saw a solid gain on Thursday after the firm announced positive results from its late-stage trial of ALXN1210 in patients with paroxysmal nocturnal hemoglobinuria (PNH). One analyst even took this opportunity to weigh in on the stock.

Ultimately, ALXN1210 demonstrated noninferiority to Soliris (eculizumab) based on the co-primary endpoints of transfusion avoidance and normalization of lactate dehydrogenase (LDH) levels, a direct marker of complement-mediated hemolysis in PNH.

The study also demonstrated noninferiority on all four key secondary endpoints: percentage change from baseline in LDH levels, change from baseline in quality of life as assessed by the Functional Assessment of Chronic Illness Therapy Fatigue scale, proportion of patients with breakthrough hemolysis and proportion of patients with stabilized hemoglobin levels.

Additionally, numeric results for all six endpoints favored ALXN1210. And there were no notable differences in the safety profiles for ALXN1210 and Soliris.

John Orloff, M.D., executive vice president and head of Research & Development at Alexion, commented:

We are very pleased with these positive data for ALXN1210 in the first and only head-to-head study versus Soliris, and the results reinforce our ambition to establish ALXN1210 as the new standard of care for patients with PNH. The data are also consistent with our hypothesis that immediate, complete, and sustained C5 inhibition is critical for patients with this potentially life-threatening disease. Soliris has established a high bar for efficacy. Achieving non-inferiority on both co-primary and all key secondary endpoints, as well as seeing numeric results in favor of ALXN1210, in such a rigorous study met a very high hurdle. We look forward to regulatory submissions of ALXN1210 in PNH in the U.S., EU, and Japan in the second half of 2018.

Merrill Lynch maintained its Buy rating on Alexion with a $164 price objective. The firm said that ALXN1210 was shown to be noninferior to Soliris in Phase 3 study of naive PNH patients. A key secondary endpoint of breakthrough hemolysis narrowly missed superiority, but there is a strong trend favoring ALXN1210. Safety was clean with no meningococcal infections in Phase 3. These results should lead to favorable reimbursement for ALXN1210.

Shares of Alexion were last seen up 6% at $130.13, with a consensus analyst price target of $158.50 and a 52-week range of $96.18 to $149.34.

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