August 02, 2023
How High Is the Bar for Future ALS Treatments? Although Neurologists Express Frustration with Current Treatments, They Are Quick to Adopt Therapies that Promise to Delay ALS Progression
According to Spherix Global Insights first market landscape study on Amyotrophic Lateral Sclerosis (ALS), general neurologists lack awareness of therapies in development for ALS.
EXTON, Pa., August 2, 2023 – A quiet but significant advance was made during the last year: not one, but two medicines were FDA approved to delay disease progression in the fatal disease, Amyotrophic Lateral Sclerosis, or ALS. Amylyx’s Relyvrio™ (sodium phenylbutyrate and taurursodiol) launched 10 months ago, and Ionis/Biogen’s Qalsody™ (tofersen) was just approved in 2023. In both cases, the FDA granted advanced marketing authorization based on phase 2 trial results and the promise on the part of manufacturers to provide updated phase 3 clinical trial results.
Regarding the FDA granting advanced marketing authorizations based on phase 2 trial results, neurologists in Spherix Global Insight’s specialist network acknowledge that phase 3 results don’t always confirm phase 2 results. However, in the case of ALS, they say they want to offer these treatments to their patients right away, along with counseling to set realistic expectations.
According to data just published in Spherix’s first market landscape study of ALS – Market Dynamix™: Amyotrophic Lateral Sclerosis (US), about one quarter of patients with ALS have already been started on Relyvrio™ and roughly one in twenty – those testing positive for the SOD-1 gene – are receiving Qalsody™. The rapid adoption of these agents is largely being driven by patient or caregiver request. 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.
The 103 surveyed neurologists in Spherix’s ALS study also indicated they are fairly disappointed with these and other current treatments: In fact, less than a fifth are fully satisfied with their patients’ response to their ALS treatment. Consistent with that attitude, neurologists in the study acknowledge a very high unmet need for new ALS treatments – saying they seek truly disease-modifying treatments. The Market Dynamix™: ALS study also asked neurologists what they would deem to be a meaningful therapeutic development in a new therapy. That feedback boiled down to, “Provide added years of life (maintaining quality of life), not months of life.”
“The current treatments really do very little to change the clinical course of the disease in my opinion.”
Neurologists have little to no knowledge of assets in clinical development despite an active pipeline with Ionis’ ION363 (Ulefnersen) for FUS gene mutations, AB Science’s masitinib, and Biogen’s BIIB105 among those assets. Neurologists are hopeful that an emerging mechanism of action may offer some promise but also are skeptical as so many investigational medicines have failed to reach the market. Neurologists emphasize that the first step is uncovering more details about the pathophysiology of ALS so that developing treatments have a specific target.
In the absence of substantial advances in ALS treatment, doctors will likely continue to try new therapies with the hope of incrementally improving patient quality of life or modest increases in overall lifespan.
Spherix monitors the quarterly changes in the ALS treatment market through its Launch Dynamix™: Relyvrio tracking study.
About Market Dynamix™
Market Dynamix™ is an independent market landscape study, conducted once a year to characterize current treatment approaches, profile the patient and project what the market may look like in 3-5 years. The reports provide an unbiased view of the competitive landscape within rapidly evolving specialty markets, fueled by robust HCP primary research and our in-house team of experts.
About Launch Dynamix™
Launch Dynamix™ is an independent service providing monthly benchmarking of newly launched products for the first eighteen months of commercial availability, augmented by a quarterly deep dive into promotional activity, barriers to uptake, and patient types gravitating to the launch brand.
About Spherix Global Insights
Spherix Global Insights is a leading provider of market research, business intelligence and advisory services to the global life sciences industry. The company’s unique service offerings are powered by deep therapeutic knowledge, the Spherix Network specialty physician panel, and commercially relevant analyses to enable strategic decision-making by our valued customers.
A trusted advisor and industry thought leader, Spherix Global Insights provides specialized market expertise in six (6) focused therapeutic areas including: dermatology, gastroenterology, nephrology, neurology, rheumatology and ophthalmology.
Spherix Global Insights Contact
Blaine Cloud, Ph.D., Neurology Franchise Head
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