Phase one launched last week after patients who had been on placebo in Biogen’s pivotal phase III VALOR study were transitioned to active therapy.
Lisa Stockman Mauriello and family/Photo courtesy of Lisa Stockman Mauriello
Late Thursday evening, it was announced that Lisa Stockman Mauriello had received her first treatment of Amyotrophic Lateral Sclerosis (ALS) drug, tofersen. After months of advocacy, the moment was bittersweet for the Mauriello family and the supporters who have been drawn to Lisa’s plight, as only time will tell whether the medicine will have an impact on her condition at this late stage.
On June 25, Biogen opened applications for its hard-won compassionate use program for the drug, an antisense oligonucleotide (ASO) which targets a mutation in the superoxide dismutase 1 (SOD1) gene, believed to be the genetic driver in a familial form of the disease called SOD1-ALS. Tofersen is being co-developed by Biogen and collaborator, Ionis Pharmaceuticals.
In phase one of the two-part early access program (EAP), access to tofersen is being granted on an individual basis to SOD1-ALS patients with the most rapidly progressing disease, classified as an ALS Functional Rating Scale - Revised (ALSFRS-R) slope decline greater than or equal to two points per month. Then, if safety and efficacy data are positive, Biogen will offer the drug to the broader SOD1-ALS population in Fall, 2021.
Phase one launched last week after patients who had been on placebo in the pivotal phase III VALOR study were transitioned to active therapy.
During a time when Americans have come face-to-face with their own mortality, Stockman Mauriello’s petition to receive access to tofersen through the “Right-to-Try” act sparked the compassion of a nation.
Stockman Mauriello suffers from a particularly devastating AD5 variant of SOD1-ALS, which typically kills patients within 12 months of the diagnosis. The New Jersey mother of three began exhibiting symptoms nearly one year ago, in August of 2020.
“We are thankful that Lisa finally got access to Tofersen - and that it happened within a week of the start of the EAP program. It was a fantastic belated birthday present,” Bob Mauriello told BioSpace. Lisa’s birthday was on July 15. “Our hope is that this is the beginning of a new day for ALS SOD1 patients and hopefully, it will lead to positive changes in the way that experimental drugs are developed and approved.”
While ultimately ceding to the pressure of over 100 000 petitioners and vocal criticism from the ALS community, Biogen did not compromise on its primary ethical argument.
BioSpace originally reported that the neurodegenerative disease leader was uncomfortable offering Tofersen more broadly while a control group of patients was still receiving a placebo in the 2:1 randomized trial. As of mid-July, Biogen stated that these patients would have transitioned to active therapy.
“We do not believe it is fair to ask participants in this study to continue to receive placebo while other SOD1-ALS patients are offered access to tofersen, but we do believe that access could be provided as soon as the placebo-controlled study has ended. However, until the safety and efficacy have been established, we will prioritize early access for a subset of the most rapidly progressing patients with this rare, severe disease,” Biogen said in a statement announcing the early access program.
Former Biogen senior vice president of corporate affairs, Daniel McIntyre, weighed in on the matter.
“The effort to support Lisa Mauriello has never been about simply giving her tofersen to the detriment of others. It’s true that the research sponsor’s first obligation is to those in the trial, which is why Lisa has asked to be randomized in the same manner as those participating in the VALOR trial,” McIntyre told BioSpace. “This started together as help for Lisa but not ONLY for Lisa, and arguments focused on trial participants on placebo does a disservice to both Lisa and other members of the ALS community who are seeking a more transparent, humane and accountable system that works for everyone.”
In Thursday’s statement, The Mauriellos went on to thank, among others, the ALS community and Lisa’s physician, Dr. Neil Shneider.
“Sadly, there are few expanded access programs for promising #ALS therapies, so this is an important step,” the family said in a statement. “We appreciate that Biogen finally agreed to offer this program and accelerated the timeline. While we wish this all would have occurred sooner, we are glad the day has finally come.”
