Update on Tofersen Clinical Program

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A message from Alfred Sandrock, EVP Research & Development about ALS investigational therapy

I want to extend my deepest gratitude to the many of you who have expressed to me and others at Biogen your concern for people living with ALS. Their experiences have touched all of us at Biogen and reinforced our commitment to finding effective and safe therapies to treat this terrible disease. 

Biogen has been working to find effective therapies for ALS for more than a decade. Unfortunately, this has been a difficult and often disappointingly unsuccessful path. In the past, we have had great hope for therapies that seemed very promising in early studies, only to find in later studies they provided no benefit or posed unjustified risk to patients. This is why even when a drug shows early promise, it is essential to conduct confirmatory studies.

In the case of tofersen, a well-controlled study is the fastest and most certain way to establish the benefit-risk profile and ultimately ensure access for SOD1-ALS patients around the world. At this point, our ongoing study includes people living with SOD1-ALS, all of whom are affected by rapidly progressing forms of the disease. Only some of these patients are receiving tofersen, while others are receiving placebo. These patients agreed to participate in our study acknowledging the risk that they may not receive tofersen, and with hope that tofersen could be shown to work and be approved for all patients. Our research and the hope of developing an effective and safe therapy would not be possible without the commitment of these brave study participants, to whom we are extremely grateful.

We cannot overlook these patients when considering questions of broader access, and cannot keep them on placebo while at the same time offering tofersen to those outside of our study. Offering tofersen outside of the study would risk failing to complete the study and risk failing to obtain access for all SOD1-ALS patients. Decisions such as these are among the most difficult and challenging we face as a company. To ensure that we are considering all aspects, we have consulted with several ethicists to help us grapple with the difficult tension that arises when access for some patients today could risk access for all future patients. While they too acknowledge the impossibility of an outcome satisfactory to everyone, our collective conclusion is that early access to tofersen should only be provided after study participants are no longer randomized to placebo.

We remain hopeful that when the tofersen study reads out later this year, it will be positive. There is more information on our website about the ongoing tofersen clinical trial.  If the study data show tofersen to have a favorable benefit-risk profile, and we do not require further controlled clinical trials, we will immediately open an Early Access Program for individuals with SOD1-ALS.

We take the responsibility that we have to ALS patients around the world very seriously. We carefully consider the needs of all patients when making these decisions. We look forward to advancing our work so that someday these hard choices may no longer be necessary.

Thank you for your support and compassion for people living with ALS,

Alfred Sandrock, M.D., Ph.D

Executive Vice President Research and Development