Biogen's Dexpramipexole Misses Endpoints in Phase III

By: Mari Sullivan

Bloomberg news article: Biogen Sinks as ALS Drug Fails to Show Efficacy in Trial

Statement from the ALS Therapy Development Institute:

This morning, Biogen Idec announced that dexpramipexole failed to demonstrate sufficient effectiveness in slowing down the progression of ALS or extending survival in a phase III clinical trial.

The information put out to date is limited to only that which appeared in the press release. We need to see the actual data to understand the implications of these findings. We hope that Biogen will release more results soon.

The ALS Therapy Development Institute shares in the disappointment of another drug that failed to meet its endpoints in a clinical trial. Rather than let this news be a discouragement, this fuels our urgency for the biotech and patient community to accelerate the clinical exploration of additional potential treatments for ALS. PALS’ participation cannot be undervalued. Without PALS willingness to participate in clinical research, we would not have been able to determine whether this drug held promise as a treatment for ALS. It is crucial that PALS continue to participate in clinical studies to truly know whether a drug is effective in combating this horrific disease.

We thank Biogen Idec for their continued commitment and investment in ALS and their continued partnership in the effort to end ALS.

When will data from this trial be presented?

We expect Biogen Idec CEO George Scangos to present more detailed information about the dexpramipexole phase III clincial trial at a future medical meeting. He presented phase 2 clincial trial results at the JP Morgan Healthcare Conference last year. Biogen is scheduled to speak next week during this year's meeting. However, we do not know if additional information will be reported at that time or at a future meeting. There are several conferences scheduled in the first part of 2013, including the American Academy of Neurology meeting in San Diego in March.

Did I do harm to myself by participating in this trial?

No significant adverse effects were reported in today's press release. However, PALS who were in the trial should always consult with their medical team regarding any side effects that they think they might have experienced due to this experimental medication. It is likely that Biogen will be reaching out to all trial participants soon to review end of trial steps.

Will I continue to have access to the drug?

We expect Biogen to contact all trial participants. There is precedent that when a trial does not meet its endpoints, patients no longer have access to the drug, however we do not know if that will be the case as this trial wraps up or not. We encourage PALS who participated in the trial to contact their clinical trial enrollment site for more information about what to do with remaining medication as soon as possible.

Why did the trial go so long?

The clinical trial ran for 12 months from the last patient enrolled: 2 years from the trial annoucement.  The trial design dictated the length needed to fully evaluate the potential efficacy of the medication. The trial would likely have been stopped earlier if there were significant signs of adverse events.

What did we learn from this trial?

We learned from this trial that the ALS patient population is ready and willing to participate in clinical research and are eager for opportunities to access potentially effective medicines. Without seeing any of the actual data, it is difficult to draw conclusions at this time specifically about this trial. It is likely that there will be many lessons learned as people sift through this huge database.One possible early lesson from this is that Phase II clinical trials are crucial to the development process and the field may need to revisit how to sufficiently power them.

What does it mean for Biogen’s interest and investment in ALS?

Biogen Idec has grown its interest and commitment in ALS within the past few years. Among these investments is the research collaboration with TDI on TDI00846 (CD40L).

In Biogen’s press release today, Douglas Williams, Ph.D., EVP of Research and Development at Biogen Idec, reiterated that “Biogen Idec is committed to advancing ALS science. We continue to work with researchers around the world to understand the causes of ALS and find potential treatments for people with ALS.”

Who should participants contact for more information about the trial?

We would suggest that PALS first contact their medical team if they were in the trial to determine what they should do with any remaining medication. Those interested in learning more about Biogen Idec and their announcement should contact Biogen’s public affairs office: 781-464-3260, 

MEDIA CONTACT AT ALS TDI: Mari Sullivan,, 617-441-7220