Acorda Therapeutics, Inc. (Nasdaq: ACOR) today announced that a proof-of-concept trial found dalfampridine extended release (ER) tablets, marketed as AMPYRA® (dalfampridine) Extended Release Tablets, 10 mg, improved walking in people with post-stroke deficits. Post-stroke deficits refer to chronic neurological deficits, such as impaired walking, motor and sensory function and manual dexterity that persist in people who have had a stroke.
"There were clear efficacy signals in the dalfampridine-ER post-stroke deficits trial and we therefore plan to proceed with a clinical development program for this indication. A top-line analysis of the data found dalfampridine-ER improved walking for people with mobility impairment resulting from ischemic stroke. Dalfampridine-ER treatment was also associated with a positive change versus placebo on a scale of functional independence in this study," said Ron Cohen, M.D., Acorda's President and Chief Executive Officer. "We are analyzing the data further to better understand the entirety of the results. After we complete the analysis, we plan to discuss the development program with the FDA. There are more than seven million stroke survivors in the United States, and approximately half of them have some lasting mobility impairment. There are no medications currently available for these patients, so new therapies are desperately needed."
This study included 83 participants who had experienced an ischemic stroke at least six months prior to enrollment and had chronic motor deficits. As part of the crossover design, participants received both dalfampridine-ER 10 mg and placebo for 14 days twice daily, with a wash-out period in between during which participants received placebo. The primary goals of the study were to assess safety and tolerability, as well as to explore various efficacy measures.
Key Safety Findings from Post-Stroke Deficits Trial
The safety findings in this study were consistent with previous clinical trials and post-marketing experience of AMPYRA in multiple sclerosis (MS).
The most common adverse events reported in the study were dizziness (10.4% dalfampridine-ER, 2.5% placebo), nausea (3.9% dalfampridine-ER, 6.2% placebo), fatigue (5.2% dalfampridine-ER, 3.7% placebo), insomnia (5.2% dalfampridine-ER, 2.5% placebo) and arthralgia (2.6% dalfampridine-ER, 3.7% placebo).
Three participants experienced a seizure during the study. One occurred while the participant was taking placebo (without prior exposure to dalfampridine-ER), one occurred while the participant was taking dalfampridine-ER, and one occurred due to an intentional overdose of dalfampridine-ER. The overdose was judged by the study investigator to be a suicide attempt related to a recent family tragedy. All three participants recovered fully.
Key Efficacy Findings from Post-Stroke Deficits Trial
Improvement in walking was measured by the Timed 25-Foot Walk (T25FW). Using the full crossover design, walking speed increased while participants were taking dalfampridine-ER compared to placebo (p < 0.05).
Participants also showed a positive change on the Functional Independence Measurement (FIM) scale while taking dalfampridine-ER compared to placebo. The FIM scale assesses an individual's ability to perform daily tasks such as bathing, grooming, eating, and walking independently.
Other exploratory efficacy measures in the study are currently being analyzed.
Cerebral Palsy Study Update
A separate proof-of-concept trial including 24 participants explored the use of dalfampridine-ER 10 mg dosed twice daily in adults with cerebral palsy (CP).
The safety findings in this study were consistent with previous clinical trials and post-marketing experience of AMPYRA in MS. The most commonly reported adverse events were headache (12.5% dalfampridine-ER, 4.2% placebo), fatigue (12.5% dalfampridine-ER, 0% placebo), insomnia (8.3% dalfampridine-ER, 4.2% placebo), diarrhea (4.2% dalfampridine-ER, 4.2% placebo) and nausea (4.2% dalfampridine-ER, 4.2% placebo). There were no serious adverse events reported.
Efficacy data from this study suggested potential treatment activity on measures of walking and hand strength; however, these data are still being analyzed to determine if they are sufficiently robust to warrant further clinical studies.
The Company plans to present data from the post-stroke deficits and CP trials in appropriate medical forums following additional analysis of the data.
AMPYRA is approved by the U.S. Food and Drug Administration (FDA) for the improvement of walking in people with MS. This was demonstrated by an increase in walking speed. The findings in post-stroke deficits and CP do not impact AMPYRA's proven safety and efficacy profile in people with MS.
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