Our bimonthly Clinical Research & Trials Update keeps you up to date on what’s happening in ALS clinical research. We unpack trial press releases, flag upcoming studies, and highlight important new publications, including care guidelines, long-term therapy data, and off label treatment insights.
We have developed a Research Glossary that contains a list of scientific and medical terms and definitions relevant to ALS. Click here to download a copy.
On December 29, 2025, QurAlis announced Phase 1 proof-of-mechanism data from its QRL-101 trial. The study was single-dose, placebo-controlled, and enrolled 12 participants.
QRL101 works by opening the Kv7.2/7.3 potassium channel, which helps stabilize neurons and prevent them from becoming overactive.*
This pathway is already validated in epilepsy, and the data showed target engagement of the ion channel opener. Phase 1 trials are not designed to prove efficacy, but the company states the drug demonstrated reduced motor neuron hyperexcitability across different assessments.
With a safety and tolerability profile reported as consistent with prior studies of no serious adverse effects, these findings support advancement of QRL-101 into Phase 2 proof-of-concept trials.
*Harmful overactivation of motor neurons (hyperexcitability) is speculated to contribute to disease progression.
On February 23, 2026, QurAlis announced Phase 1/2 proof-of-concept data from its QRL-201 trial. The study was double-blind and placebo-controlled, with a total of 69 participants.
The press release reports that the therapeutic achieved its intended biological target by significantly increasing STMN2 levels in the nervous system. While we still don’t yet know whether boosting STMN2 can influence disease progression, showing target engagement in humans can be a meaningful first step.
The company also reported a trend toward slowing disease progression, but it’s important to remember that Phase 1 and 2 trials are not designed to prove whether a drug works, so trends should be interpreted with caution until a larger Phase 3 study confirms the data. QurAlis also reported a significant slowing down of the disease according to the ALSFRS-R, but only in a subgroup of participants with lower baseline NfL levels, which can naturally correlate with slower progression. The press release doesn’t indicate how large this subgroup was. Although the ALSFRSR is helpful and widely used, it is based on patient reports rather than a biological marker, so it can also be influenced by variability.
The study also showed a reduction in phosphorylated neurofilament heavy (pNfH) in the low dose group but doesn’t state the number of participants in this group or further details. pNfH is a type of protein released when nerve cells are damaged, and it is being investigated as a biomarker for ALS, like neurofilament light chain (NfL), but we are still learning how to best interpret this biomarker in clinical trials.
On the safety side, which is the main purpose of early trials, the therapy was reported as generally safe and well tolerated. Based on these signals so far, regulators in Canada have approved an open‑label extension, allowing all eligible participants to continue receiving QRL‑201. QurAlis is now preparing for a pivotal Phase 3 trial planned for 2027.
On December 8, 2025, Amylyx Pharmaceuticals announced initial safety and tolerability data from cohort 1 of its Phase 1 LUMINA trial of AMX0114 in ALS, reporting no treatment-related serious adverse events in 12 participants.
Enrollment for the second cohort began in Canada in December 2025. Read more in our clinical trials page.
On December 5, 2025, AL-S Pharma reported Phase 2 results from its double-blind, placebo-controlled study of AP-101, with 73 participants living with ALS.
According to the press release, the study met its primary goal, showing the drug to be safe and well-tolerated. Although Phase 2 studies of this size are not sufficient to prove efficacy, the company states that exploratory analyses showed encouraging signs of clinical benefit. They report that participants who started AP-101 immediately experienced longer survival and a delayed need for ventilatory support compared with participants who first received placebo, and that these positive effects were seen in both sporadic ALS and SOD1ALS.
The company states that the therapy also appeared to stabilize disease stage and slow functional decline on the ALSFRS-R in people with elevated misfolded SOD1, though the company did not state if this was statistically significant and provide further data. The company also states that the drug produced favorable changes in neurofilament biomarkers, though they did not release further details.
AL-S Pharma is preparing a confirmatory Phase 3 study.
On December 12, 2025, Prilenia and Ferrer announced FDA clearance to initiate PREVAiLS, a 500-participant Phase 3 randomized, placebo-controlled trial of pridopidine in early, rapidly progressive ALS, designed to confirm Phase 2 HEALEY ALS Platform Trial subgroup findings showing slowed progression and improved survival.
Canadian sites are still under consideration, and our clinical trials database will be updated once we receive more information.
Other trials are expected to start at Canadian sites soon in 2026, and more information will be shared once available.
“ALS clinical trials now recruiting at UBC”
We would like to congratulate Dr. Erik Pioro and the team for all their efforts in getting clinical trials to Vancouver.
*Pathogenic variants (mutations) in the SOD1 gene are a known risk factor for ALS and contribute to about 1.5 to 7% of all ALS cases.
Long-term results for tofersen, now approved in Canada as Qalsody, continue to show ongoing benefits for people with SOD1-ALS.*
In the Phase 3 VALOR trial and its open label extension, people who started the drug six months early had a slower decline in clinical function (according to the ALSFRS-R score), respiratory function, muscle strength, and overall quality of life over almost three years. About 25% of early start participants even showed improvements in strength and function.
These findings support early treatment initiation, despite the fact that the study didn’t show a statistically significant difference between early- and delayed-start groups at the three-year point. For tofersen, this is likely a testament to it being an effective treatment, as a large difference would not be expected if individuals in the delayed-start group were benefitting for years, following only six months on placebo. Overall trends still favoured earlier treatment, and there is an expectation that the benefit of earlier intervention would be much more pronounced in individuals with faster progressing disease. This is being tested in a landmark, presymptomatic clinical trial called ATLAS, which is currently underway. In this study, both groups did better than what’s typically seen in SOD1-ALS.
Plasma NfL levels, a biomarker of nerve damage, dropped the most around 16 weeks after starting treatment. By about three years, NfL levels were still much lower than at the start: about 67% lower in the early-start group and 64% lower in the group that began treatment six months later.
The long-term safety profile remained consistent with earlier trials, and all the data together strongly support Qalsody as an effective long-term treatment option, especially when started early. Like any treatment, many people are thriving on Qalsody but side effects may occur in some individuals. Biogen published a separate paper on the potential safety risks of Qalsody as a commitment to transparency.
Watch the trailer for Another Tomorrow, a documentary coming up spring 2026 about Qalsody’s impact.
ALS Untangled is a program founded by Dr. Richard Bedlack that reviews alternative and off-label treatments for people living with ALS.
N-acetylcysteine has theoretical biological rationale for ALS, speculated to reduce oxidative stress, regulate neuroinflammation, and mitigate mitochondrial dysfunction*. However, preclinical studies, case reports, and clinical trials have not shown meaningful benefit. Because current evidence does not support its effectiveness in slowing disease progression, ALS Untangled does not recommend its use as an ALS treatment.
Clenbuterol has biological properties that might be relevant to ALS, such as inducing muscle growth, improving mitochondrial function*, and reducing neuroinflammation. However, existing mouse studies and small open label trials are too limited to show clear benefit. Given its significant side effects and high dropout rates, ALS Untangled cannot currently recommend clenbuterol as an ALS treatment, though further research is encouraged.
*Mitochondria are small structures inside our cells that act like power plants, creating the energy the cell needs to function and stay healthy. In ALS, mitochondria can become damaged or work less efficiently, which may contribute to the loss of motor neurons. Supporting healthy mitochondrial function is therefore an area of interest in ALS research.
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