Following Amylyx’s announcement that AMX0035, its amyotrophic lateral sclerosis (ALS) pipeline drug, showed potential to prolong patient survival in addition to slowing rapid disease progression in the open label extension of Phase II/III clinical trial CENTAUR;

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

“The new functional and survival benefits of AMX0035 will significantly strengthen the drug’s position to enter the ALS market over the coming years as a potential disease-modifying therapy, given its high tolerability profile and convenient oral administration. Key opinion leaders (KOLs) interviewed by GlobalData expressed that if AMX0035 continued to show therapeutic benefits in trials, they would likely prescribe it to the majority of their patients upon approval, as it would address some major unmet needs and expand existing treatment options in the ALS market.

“The announced positive results support AMX0035’s clinical superiority as there was a 44% risk reduction for death over the course of follow-up in the open-label extension study. This will certainly advance the drug’s regulatory approval processes and could encourage other companies to focus on developing pipeline agents with novel mechanism of action that try to halt or slow the progression of the disease, rather than reformulating current ALS treatment.

“Further opportunities could arise for another pipeline agent with a similar molecule. Bruschettini’s Tudcabil (TUDCA) is currently undergoing a Multi-Centre Phase III trial in Europe to evaluate its safety and efficacy as an add-on treatment in ALS patients. GlobalData anticipates that Tudcabil will launch in 2022 in Europe, contributing $3.5m of sales to the global ALS market that is forecasted to reach $1.04bn in global sales by 2029 at a compound annual growth rate (CAGR) of 13.9%, driven by pipeline launches. There is still a pressing need to find a drug with curative and disease-modifying properties. Despite the rarity of the disease, there remains high level of unmet need in the current treatment landscape and significant opportunities for pharmaceutical companies to address.”