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Biogen is still shocked by the crucial failure of aducanumab in Alzheimer's disease, but he is celebrating positive news from another active neurology class.
Preliminary results from a phase 1 trial showed that tofersen (BIIB067), in which Biogen was licensed by Ionis Pharmaceuticals, was well tolerated by patients with amyotrophic lateral sclerosis (ALS). The results suggest that the drug may slow the progression of the disease in people with a mutant SOD1 gene.
Mutations in SOD1 can cause misfolding of the protein expressed by the gene as toxic nerve cells in the brain and spinal cord, leading to loss of muscle control, paralysis and possibly death. About 10% of all cases of ALS result from inherited genetic mutations, and of these familial cases about 20% are caused by the SOD1 gene.
Tofersen is an antisense oligonucleotide that aims to reduce the production of toxic protein. It works by binding to and inhibiting the SOD1 mRNA, disabling the failed gene.
In the Phase 1 trial, 50 ALS patients with SOD1 mutation received 20 mg, 40 mg, 60 mg or 100 mg tofersen or placebo via lumbar puncture for approximately three months. The researchers found that the 10 people who received the highest dose of tofersen had a 37% reduction in SOD1 protein in their cerebrospinal fluid compared to 12 people in placebo, according to a study presented at the 71st Annual Meeting. from the American Academy of Neurology to Philadelphia Cream.
"Lower concentrations of the protein in the cerebrospinal fluid suggest that there were also lower concentrations in the brain and spinal cord. Such reductions could lead to the preservation of motor neurons and the slow progression of the disease, but further studies are needed to deepen this issue, "said lead author Timothy Miller of Washington University in St. Louis, in a statement.
In addition, patients receiving the 100 mg dose scored better on tests that measure the clinical decline of ALS, including respiratory capacity, muscle strength, and other bodily functions. On a score of 48, patients under 100 mg of tofersen experienced an average decline of 1.1 points, while those on placebo suffered an average fall of 5.3 points, according to Miller's team.
RELATED: Neurimmune anti-SOD1 antibodies show promise in mouse ALS studies
Since the factors underlying most cases of ALS are unknown, the targeting of SOD1, a known cause of the familial form of the disease, provides a clear strategy for researchers. Swiss Biotech Neurimmune relies on the memory cells of healthy elderly people to develop a recombinant antibody targeting SOD1. (The company previously used the same technology to discover aducanumab before yielding it to Biogen.) Neurimmune's drug, called α-miSOD1, has recently been shown to be promising in post mortem spinal cord specimens removed. in ALS patients and in mouse models with SOD1 mutations.
A team of scientists from the University of Umeå in Sweden analyzed the SOD1 clumps present in ALS, providing a roadmap for researchers developing drugs targeting gene mutation.
The Biogen tofersen phase 1 trial offers a proof of concept and, based on a positive analysis first revealed last December, the company has exercised its option to gain full control of the product. Antisense drug from Ionis. Biogen is now moving the drug forward in pivotal trials, with the goal of proving that it works in larger patient groups over longer periods of time.
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