Neurologists frustrated with ALS treatments now on market: Study

But most clinicians offering patients 2 new approved therapies

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by Steve Bryson, PhD |

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While neurologists are frustrated with the treatments for amyotrophic lateral sclerosis (ALS) now on the market, they rapidly adopt new therapies that may delay disease progression for use among their patients with the neurodegenerative disorder.

That’s according to Spherix Global Insights, a company providing market research and business intelligence in the life sciences, which has completed its first market landscape study on ALS.

That study, involving 103 neurologists in Spherix’s specialist network, found that many clinicians are already prescribing the two new ALS medications — Relyvrio and Qalsody — approved in the U.S. in the last year.

“With no cure, and no way to stop or reverse progression of the disease, neurologists appear willing to prescribe these treatments without typical real-world data,” Spherix stated in a company press release, adding, “They say they want to offer these treatments to their patients right away, along with counseling to set realistic expectations.”

Interestingly, data provided by Spherix also suggested that neurologists lack awareness of investigational ALS treatments. That information was reported in the company’s Market Dynamix: Amyotrophic Lateral Sclerosis (U.S.) — an ongoing survey of healthcare providers in the U.S. on the use of ALS therapies.

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Neurologists offering new ALS treatments Relyvrio, Qalsody

ALS is a progressive neurological disorder marked by the loss of motor neurons, which are the nerve cells that control muscle movements. As a result of such damage, patients experience muscle weakness and atrophy (shrinking), eventually leading to paralysis.

Four treatments have been approved to date for ALS. Clinical trials demonstrate that these therapies can slow disease progression and extend survival by a few months.

Of these, Relyvrio (sodium phenylbutyrate and taurursodiol) and Qalsody (tofersen) were most recently approved by the U.S. Food and Drug Administration. The decisions were based mainly on Phase 2 trial results, with the companies now marketing the therapies — Amylyx for Relyvrio and Biogen for Qalsody — promising to provide updates on Phase 3 trial data.

In the survey, the neurologists acknowledged that Phase 3 trials don’t always confirm Phase 2 results. Regardless, these physicians reported that they planned to offer the treatments immediately to their patients, while counseling them and their caregivers on realistic expectations.

To date, about 25% of ALS patients have already been started on Relyvrio, and roughly 5% are receiving Qalsody — those carrying a mutation in the SOD1 gene,  for whom Qalsody is approved. Requests from patients or caregivers are the main drivers for neurologists rapidly adopting these therapies, the survey found.

Despite a lack of real-world data, the neurologists indicated they were willing to prescribe these treatments because there is no cure for ALS and no currently available therapy can stop or reverse disease progression. Yet, they are disappointed with these and other therapies, with fewer than 20% in the survey being fully satisfied with treatment responses.

The current treatments really do very little to change the clinical course of the disease in my opinion.

Consistently, neurologists acknowledged a very high unmet need for new ALS therapeutics, especially effective disease-modifying treatments that alter the course of ALS. When asked about new therapies, most neurologists wanted those that “Provide added years of life (maintaining quality of life), not months of life.”

According to one surveyed neurologist, “The current treatments really do very little to change the clinical course of the disease in my opinion.”

Survey data also demonstrated that neurologists have little to no knowledge of investigational treatments now in clinical development, including masitinib, BIIB105 and ION363 (ulefnersen).

Because so many experimental medicines have failed approval, the neurologists indicated they were skeptical. But they also were hopeful that a therapy with a new mechanism of action may offer some promise.

More research is needed to reveal underlying mechanisms that drive ALS disease, according to the neurologists, who noted that new data could potentially provide specific targets for novel treatments.

Without such options, clinicians will likely continue to prescribe new therapies hoping to improve quality of life or extend survival, no matter how modest the benefit, according to the Spherix study.