ALS Global Day reiterates the need for an elusive cure despite promising late-stage pipeline

The International Alliance of Amyotrophic Lateral Sclerosis (ALS)/ Motor Neuron Disease (MND) Associations, recognizes June 21 as the ALS Global Day. The goal of this yearly campaign is to promote global awareness and raise funds for ALS research activities.

Sarah Elsayed, Pharma Analyst at GlobalData, a leading data and analytics company, offers her view on the opportunities and challenges in this market:

“ALS is a rare but fatal progressive neurodegenerative disease that belongs to a wider group of disorders known as MNDs. To date, patients diagnosed with ALS face an uphill battle against the disease as they have to take daily medication with limited efficacy and moderate survival benefits for the rest of their lives. Key opinion leaders (KOLs) interviewed by GlobalData have cited that if any drug treatment shows some benefits to halt or reverse the disease, it would revolutionize life for ALS patients.

“There is a pressing need to shift the treatment paradigm toward new disease modifying drugs (DMDs) that can ultimately become an elusive cure for ALS patients rather than symptomatic treatment. The current treatment algorithm offers only two therapies for the treatment of ALS; Sanofi’s Rilutek (riluzole), a glutamate antagonist that is genericized across the eight major markets (8MM*), and Mitsubishi’s Radicava (edaravone), a free radical scavenger, only available in the US, Canada and Japan, neither of which can stop or reverse the progression of the disease and, at best, they increase a patient’s life span by up to six months. As such, there are significant opportunities for pharmaceutical companies to target this major unmet need.

“ALS pipeline includes 267 diverse candidates across all active stages of development in the 8MM, of which 14 drugs (5%) are in the late-stage development with a potential to present promising therapies into market over the next five years. The most promising pipeline candidates are Biogen’s tofersen, an antisense therapy and BrainStorm’s NurOwn, a stem-cell therapy. Most KOLs think that if these therapies get approved, they will provide a significant breakthrough in the treatment of ALS due to their neuro-regenerative properties. However, tofersen is being investigated only in patients with superoxide dismutase-1 (SOD-1) mutations who represent 2% to 5% of the total ALS population, yet the early signs show that it can be effective to reverse and stop the disease in this rapidly progressive subset of ALS patients. Thus, tofersen can also replace riluzole in this group of patients if it shows positive results in Phase III trial as riluzole will no longer be needed.

“The ALS market represents one with several future opportunities, but at the same time several potential pitfalls such as the high clinical trial failure rate, the lack of diagnostic biomarkers and the non-linear disease progression in the different subsets of patients. Given that, GlobalData expects that with the exception of tofersen and NurOwn, the remaining late-stage pipeline products that will succeed to reach the market will likely be prescribed as an add-on therapy to the current standard of care (SOC), riluzole and in a combination therapy approach with other future approved treatments.”

*8MM: US, France, Germany, Italy, Spain, UK, Canada and Japan

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