Innovative approaches are poised to revolutionize the treatment of amyotrophic lateral sclerosis (ALS), a challenging and rare disease. The current therapies have limitations in efficacy, prompting a surge in exploration of novel disease-modifying drugs (DMD). With 109 pipeline candidates in various stages of development, the ALS treatment landscape is primed for transformation, offering hope for improved outcomes and potential commercial success in the pharmaceutical sector, says GlobalData, a leading data and analytics company.

Currently, the available treatments for ALS include Sanofi’s Rilutek, a glutamate antagonist, Mitsubishi Tanabe’s Radicava/Radicut, a free radical scavenger, Amyylyx’s antisense therapy Relyvrio and Biogen’s apoptotic regulator Qalsody.

The key opinion leaders (KOL’s) interviewed by GlobalData expressed that all the available treatments, including newly approved Relyvrio and Qalsody lack efficacy in their modes of actions (MOA’s) and only succeed in prolonging life by a moderate margin

Momna Ali, Healthcare Analyst at GlobalData, comments: ‘‘To address the lack of efficacious options, the treatment horizon is now abuzz with exploration of novel approaches. This not only offers pharmaceutical companies’ lucrative opportunities for profit but also presents a ray of hope for patients eagerly waiting for disease-modifying drugs’’.

The ALS product pipeline comprises of 109 candidates in various stages of development from Phase 0 all the way to Phase III, across the 8MM (The US, France, Germany, Italy, Spain, the UK, Canada, and Japan).

Ali adds: “Among the impressive array of 109 pipeline drugs, a promising dozen are in Phases II-III and are expected to enter the market within the next five years, signalling commercial success.”

The developing products include tyrosine kinase inhibitors (TKIs), leukotriene receptor antagonists (LTRAs), interleukin-2 antagonists (IL-2), monoclonal antibodies, stem cell therapies, poly(A)-binding proteins (PAB), d2 dopamine receptors (D2) and vitamin B12, which are all explored further in GlobalData’s upcoming “Amyotrophic Lateral Sclerosis Updated Market Analysis and Forecast to 2029.”

Ali concludes: “The imperative to transition towards DMDs capable of curing ALS is widely acknowledged in the industry. As the current diverse pipeline indicates, GlobalData anticipates significant evolution in the treatment landscape over the coming decade. This forecast underscores lucrative prospects for pharmaceutical companies to innovate and deliver safe, efficacious therapies.”