Shares of Biogen Idec Inc. (NASDAQ: BIIB) touched a new 52-week high of $73.53 on Thursday. The company and its partner Knopp Biosciences announced enrollment of the first patient in EMPOWER, a multi-national Phase 3 study evaluating the efficacy, safety and pharmacokinetics of dexpramipexole in patients with amyotrophic lateral sclerosis (ALS).
The Weston, Massachusetts-based Biogen Idec said ALS, also known as Lou Gehrig’s disease and motor neuron disease (MND), is a rapidly progressive and degenerative disease of motor neurons in the brain and spinal cord. It causes muscle atrophy and spasticity, resulting in weakness, progressive paralysis and, ultimately, death usually by respiratory failure.
Biogen Idec said the first patient dosed in the EMPOWER study triggers a $10 million milestone payment from Biogen Idec to Knopp.
Biogen Idec is committed to working with the ALS community to find new treatment options for this deadly disease and to improve the lives of people with ALS. Based on promising data from earlier clinical trials, we believe that dexpramipexole has the potential to be a significant advance for people suffering from ALS, and we will fully explore its potential as a new treatment for this devastating disease, said Alfred Sandrock, Senior Vice President of Development at Biogen Idec.
EMPOWER is a Phase 3, randomized, double-blind, placebo-controlled, parallel-group, multi-center study that will assess the safety and efficacy of dexpramipexole in people with familial or sporadic ALS.
The companies plan to enroll about 804 patients, who will be randomized in a one-to-one ratio to receive either dexpramipexole 150 mg twice daily or placebo and will be followed for a period of at least 1 year. Investigators also have the option to maintain treatment with the current standard-of-care in ALS for all patients enrolled in the study.
The primary objective will be assessed using a joint ranking of functional outcomes adjusted for mortality on the ALS Functional Rating Scale, Revised (ALSFRS-R), a validated rating instrument used by physicians for monitoring the progression of disability in patients with ALS.
Biogen Idec said it has agreed with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for the design of the Phase III clinical trial of dexpramipexole. Under an SPA, the FDA evaluates a clinical trial protocol to assess whether it is adequate to meet current scientific and regulatory requirements for a potential application to market a new drug.
Dexpramipexole was well-tolerated in three Phase 1 studies in healthy volunteers who received dexpramipexole in single doses up to 300 mg or multiple doses of up to 150 mg twice daily for four-and-a-half days.
Dexpramipexole was also well-tolerated in a two-part Phase 2 study (CL201) that evaluated about 100 ALS patients treated for up to nine months and in a subsequent Phase 2 extension study (CL211) that has continued to follow ALS patients for about two additional years.
In the first part of CL201, dexpramipexole at 150 mg twice daily showed a trend toward slowing functional decline over a 12-week period compared to placebo. Following re-randomization in the second part of CL201, dexpramipexole at 150 mg twice daily again showed a trend toward slowing functional decline and also showed a trend toward improving survival compared to low dose over a six-month period.
In CL201, the joint ranking, which incorporates both function and survival and which is the primary endpoint of the Phase 3 trial, significantly favored dexpramipexole 150 mg twice daily compared to 25 mg twice daily.
Biogen Idec stock gapped open sharply higher March 31 at $71.95 compared to previous day's close of $71.90. The stock touched a new high of $73.53 on Thursday. The stock closed Thursday's regular trading up 2.18 percent at $73.47 on a volume of 2.51 million shares on the NASDAQ Stock Market. The stock traded between $45.96 and $73.53 during the past 52 weeks.