Despite the many challenges caused by COVID-19 this past year, ALS research made strides toward finding effective treatments for ALS. As 2020 winds down, we wanted to share an update from our partners at Biogen on some of their ongoing research projects.

In its recent statement, Biogen spoke of its urgent work in targeting genetic forms of ALS caused by mutations in some of the most common ALS-associated genes, such as SOD1 and C9orf72.

Biogen’s phase 3 VALOR trial targeting the SOD1 gene is close to achieving full enrollment. According to Biogen: “We extend our deepest gratitude to the entire community for your ongoing support as we near our enrollment target.”

Biogen is also beginning a new study designed for people that carry SOD1 gene mutations, but who do not yet show symptoms (pre-symptomatic), to understand if the drug tofersen is beneficial and safe in this population. This study is the first of its kind in the ALS trial space.

Biogen also provided updates on three other ongoing ALS trials that are progressing despite challenges stemming from the COVID-19 pandemic.

Read Biogen’s full statement below:

December 10, 2020

Dear members of the ALS community,

As Biogen continues to advance ALS research, we have a few updates to share with you about our clinical development programs, including tofersen (an investigational drug being studied in people with ALS with a confirmed SOD1 mutation), investigational BIIB078, investigational BIIB100 and investigational BIIB105:

Tofersen

Our tofersen clinical development program continues to progress. We are close to fully enrolling the Phase 3 VALOR study evaluating the safety and efficacy in SOD1-ALS participants and are on track to share data by the end of 2021. We extend our deepest gratitude to the entire community for your ongoing support as we near our enrollment target. Details about the study can be found at www.alsvalorstudy.com and https://www.clinicaltrials.gov/ct2/show/NCT02623699

Additionally, following productive discussions with the U.S. Food and Drug Administration, Biogen is in the process of initiating a new study of the safety and efficacy of tofersen in individuals with a confirmed SOD1 mutation and who are pre-symptomatic - do not have clinical symptoms or signs of disease. This study, called ATLAS, aims to determine whether pre-symptomatic intervention with tofersen can potentially delay clinical onset and/or slow the progression of disease and will include a sub-set of adult participants who are found to carry certain rapidly progressive SOD1 gene mutations. The study is being designed in collaboration with Dr. Michael Benatar from the University of Miami Miller School of Medicine, with critical insights the Pre-Symptomatic Familial ALS (Pre-fALS) study that he initiated in 2007 We anticipate the study to commence next year and look forward to sharing updates.

BIIB078

Since our last update regarding BIIB078, we have added an additional group to the Phase 1 study assessing the safety, tolerability and pharmacokinetics of this investigational drug. The study continues to evaluate varying doses of BIIB078 in individuals who have ALS with a confirmed C9orf72 mutation, targeting completion in 2022, and now aims to enroll about 115 individuals. Details about the study can be found at https://clinicaltrials.gov/ct2/show/NCT03626012

BIIB100

The safety and tolerability of escalating doses of BIIB100 are being assessed in the Phase 1 study. Details about the study can be found at https://clinicaltrials.gov/ct2/show/NCT03945279

BIIB105

In September Biogen dosed the first patient in a Phase 1 study of BIIB105. The study is evaluating the safety and tolerability of BIIB105 and aims to enroll approximately 70 participants. Details about the study can be found at https://clinicaltrials.gov/ct2/show/NCT04494256

For information on clinical trial enrollment for any of our ongoing studies, please contact patientcenter@biogen.com.  

We thank you for your continued support of our efforts and we will share further updates on our ALS clinical development program.

Sincerely,

Biogen