Amgen announced positive top-line results for erenumab (AMG 334) from a phase 3, RandomIzed, double-blind, placebo-controlled Study to Evaluate the efficacy and safety of erenumab in migraine prevention (ARISE). These data showed the ARISE study met the primary endpoint, demonstrating a statistically significant reduction from baseline in monthly migraine days in patients with episodic migraine treated with erenumab compared with placebo at 12 weeks. Erenumab is specifically designed to prevent migraine by blocking the Calcitonin Gene-Related Peptide (CGRP) receptor, which is believed to have a critical role in mediating the incapacitating pain of migraine.

"People with episodic migraine lose a substantial part of their lives to migraine, and many face intolerable pain and physical impairment, frequently accompanied by a significant disruption of their daily activities. Unfortunately, there are limited preventive treatment options currently available for these patients," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "These positive results, along with the recent chronic migraine results,  contribute to the growing body of evidence supporting erenumab as an innovative treatment option for people who are suffering from this debilitating disease."

A total of 577 patients were randomized to receive either placebo or erenumab 70 mg subcutaneously, once monthly. Patients enrolled in the ARISE study were experiencing between four and 14 migraine days each month, with an average of eight migraine days per month at baseline. Those receiving erenumab experienced a statistically significant 2.9-day reduction from baseline in monthly migraine days, as compared to a 1.8-day reduction in the placebo arm.

The safety profile of erenumab was similar to placebo and consistent with previously reported studies. The most common adverse events were upper respiratory tract infection, injection site pain and nasopharyngitis.

Further analysis of these data is ongoing and will be submitted to a future medical conference and for publication. The STRIVE study, a second phase 3 episodic migraine study evaluating both 70 mg and 140 mg doses for 24 weeks, is expected to be completed by the end of this year. Positive results from a phase 2 study of erenumab in chronic migraine prevention were also announced earlier this year.

Erenumab is being co-developed by Amgen and Novartis. As part of the collaboration, Amgen retained commercialization rights in the US, Canada and Japan, and Novartis has rights in Europe and the rest of the world.

The ARISE study (20120297) is a global phase 3, multicenter, randomized, 12-week, double-blind, placebo-controlled study evaluating the safety and efficacy of erenumab in episodic migraine prevention. In the study, 577 patients were randomized to receive once-monthly subcutaneous placebo or erenumab (70 mg) in a 1:1 ratio. Trial participants who completed the double-blinded portion had the option to continue in a long-term safety extension. Patients enrolled in ARISE were experiencing between four to 14 migraine days each month. The primary endpoint was change in monthly migraine days from baseline to the last four weeks of the 12-week treatment phase (the number of migraine days between weeks 9 and 12). Secondary study endpoints included reduction of at least 50 percent from baseline in monthly migraine days and change from baseline in monthly acute migraine-specific medication treatment days. The Migraine Physical Function Impact Diary (MPFID), a scale developed to measure impact of migraine on physical function and impact on everyday activities, assessed two other secondary endpoints.

Erenumab is a fully human monoclonal antibody specifically designed for the prevention of migraine. Erenumab targets and blocks the Calcitonin Gene-Related Peptide (CGRP) receptor, thought to be pivotal in the genesis of migraine. Erenumab is currently being studied in several large global, randomized, double-blind, placebo-controlled trials to assess its safety and efficacy in migraine prevention.

In August 2015, Amgen entered into a global collaboration with Novartis to jointly develop and commercialize pioneering treatments in the field of migraine and Alzheimer's disease (AD). The collaboration focuses on investigational Amgen drugs in the migraine field, including erenumab (currently in Phase 3 studies for episodic migraine) and AMG 301 (currently in a Phase 1 study). For the migraine program, Amgen retains commercialization rights in the US, Canada and Japan, and Novartis has commercialization rights in Europe and rest of world. Also, the companies are partnering in the development and commercialization of a beta-secretase 1 (BACE) inhibitor program in AD. Novartis' oral therapy CNP520 (currently in a phase 2 study for AD) will be the lead molecule and further compounds from both companies' pre-clinical BACE inhibitor programs may be considered as follow-on molecules.