In phase one of the two-part program, access to tofersen will be granted on an individual basis to SOD1-ALS patients with an ALS Functional Rating Scale - Revised (ALSFRS-R) slope decline greater than or equal to 2 points per month.
Lisa Stockman Mauriello and family/Photo courtesy of Lisa Stockman Mauriello.
In a reversal of course that may come too late for Lisa Stockman Mauriello, Biogen will open a compassionate use program for Amyotrophic Lateral Sclerosis (ALS drug), tofersen. The program will begin in mid-July, after the completion of the pivotal phase III VALOR study, but before the data reads out.
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” drew the attention of the nation.
Tofersen, developed by Biogen with collaborator Ionis Pharmaceuticals, is 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.
In phase one of the two-part program, access to tofersen will be granted on an individual basis to SOD1-ALS patients with an ALS Functional Rating Scale - Revised (ALSFRS-R) slope decline greater than or equal to 2 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.
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 in August of 2020, which may put the start date of Biogen’s program out of reach for her.
“We are pleased our efforts have contributed to some people with ALS getting access to tofersen faster than they would have otherwise. Unfortunately, it still falls short for ALS A5V patients like me who are deteriorating rapidly. For us, this is a death sentence because we most likely will not make it to mid-July when the tofersen expanded access trial will start, and if we do, we will be in such bad shape that we may not see any benefit,” stated Stockman Mauriello this morning.
Yesterday, Biogen denied Stockman-Mauriello’s fifth request for compassionate use of tofersen.
While finally 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 will 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.”
