Neuroscience Pioneer NeuroSense Joins Forces with Biogen in the Race to Transform ALS Treatment

Patients with amyotrophic lateral sclerosis (ALS) are witnessing a treatment revolution brewing for over 20 years, and more innovative therapies are still on the way.

Before Mitsubishi Tanabe Pharma's Radicava receivedFDA approvalin 2017, the ALS treatment landscape was barren. The first drug approved for ALS, riluzole, came to market in 1995, and other companiesfailedto successfully develop effective therapies until Radicava arrived. A few years later, in 2022, Amylyx Pharmaceuticals' combination therapy Relyvrio receivedFDA approval, becoming the first drug to slow disease progression in ALS.

Now, after the viral "Ice Bucket Challenge" of 2014 raisedmore than $100 millionfor ALS research, more companies are advancing their own combination therapies, offering hope for new pathways for patients with this devastating disease. Among them, NeuroSense Therapeutics and partner Biogen are pushing the oral therapy PrimeC toward its final stages—after the drug showedpositive safety and efficacy in a mid-stage trial. As a partner, Biogen is exploring an ALS biomarker called neurofilament protein to mark disease progression and treatment viability.

"We now recognize that ALS is a complex disease involving multiple targets, and if you target only a single target, the likelihood of achieving clinical benefit is relatively low. There have been many failures in this field."

—Alon Ben-Noon, CEO and co-founder of NeuroSense

Clinical-stage NeuroSense also has a mid-stage program for Alzheimer's disease and is conducting preclinical research in Parkinson's disease. PrimeC will be its first attempt at a commercial product.

Here, NeuroSense CEO and co-founder Alon Ben-Noon discusses the rapidly evolving ALS treatment landscape, PrimeC's place within it, and the therapeutic and regulatory challenges ahead.

This interview has been edited for length and style.

PHARMAVOICE: The recent approvals and launches of new ALS therapies highlight a gap of more than 20 years without new drugs. Why did it take so long to meet this need?

ALON BEN-NOON:It's the result of multiple factors. In the early 2000s, public awareness of the disease was low, and the few companies that tried to tackle it mostly used single-target mechanisms. We now recognize that ALS is a complex disease involving multiple targets, and targeting only one has a relatively low chance of clinical benefit—there have been many failures in this field. Since the Ice Bucket Challenge, public awareness has risen dramatically, and we've learned more about the different mechanisms of motor neuron degeneration from subsequent research. Currently, three companies are developing combination therapies: Amylyx (whose drug is approved), us (using a combination of ciprofloxacin and celecoxib), and Coya Therapeutics.

How does NeuroSense's PrimeC position itself in the future treatment landscape?

First, PrimeC is safe and well-tolerated, and we observed a 29% difference between the placebo and active drug groups in 68 patients, showing meaningful patient benefit. Overall, clinicians say that as long as there are no drug-drug interactions, they would prescribe any approved drug to their patients. From their perspective, patients should be on as many drugs as possible, forming a "drug cocktail," because we don't yet know which type works best for each patient. This strategy is fairly common among ALS clinicians and makes sense.

So we don't see it as necessarily competitive between companies—if it helps patients, they should have access. Of course, the potential issue is reimbursement: insurers might say they can't cover Relyvrio at $160,000 per year, Radicava at $140,000 per year, and future PrimeC costs all at once. We think it will ultimately come down to the magnitude of efficacy shown in pivotal studies, safety profiles, and pricing.

Biogen is one of the companies with the new therapy Qalsody, and you've been collaborating since 2018. Can you talk about how that partnership came about and the dynamics of working with such an established biotech?

Biogen had been watching us and was impressed by the data we presented. Of course, they were quite cautious before getting definitive results in this complex indication. They are experts in the field and wanted to analyze blood samples even before we had results, and we were happy to collaborate on that. They have a right of first refusal and will continue to conduct statistical analyses. Biogen is not only an expert in neurofilament proteins but also genuinely cares about ALS patients and wants to provide more effective treatments. We see them as ideal partners to advance our program to market.

Speaking of Biogen, they're no stranger to regulatory controversies in neuroscience. Do these factors need to be considered in advancing PrimeC toward approval?

I need to choose my words carefully to avoid overpromising to patients and investors. But clearly, if we get excellent neurofilament data that correlates with clinical outcomes and additional supportive biomarkers, we plan to discuss accelerated approval options with the FDA. At the same time, we'll also discuss the Phase 3 study design—even if we get excellent data before accelerated approval, we'll still conduct a Phase 3 study, as Amylyx did.

As this wave of ALS drugs reaches patients, what exciting plans does NeuroSense have next?

The most exciting thing, of course, is the upcoming neurofilament data. Additionally, the Alzheimer's program is advancing with more collaborations, and discussions with regulators will guide our path to bringing PrimeC to patients.

It's hard to describe exactly how each patient feels, but there is indeed great hope. We know we're doing something that benefits patients, and that brings immense satisfaction. Patients believe we can influence the disease course and slow progression, which is very gratifying for us.