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September 22, 2021updated 21 Sep 2021 5:37pm

Potential FDA Approval on the Way for Amylyx’s AMX0035 in ALS Ahead of Time

On September 15, Amylyx Pharmaceuticals announced that its bid to submit a new drug application (NDA) for its amyotrophic lateral sclerosis (ALS) investigational asset AMX0035, using data from the Phase II CENTAUR trial, has been permitted following recent discussions with the FDA.

On September 15, Amylyx Pharmaceuticals announced that its bid to submit a new drug application (NDA) for its amyotrophic lateral sclerosis (ALS) investigational asset AMX0035, using data from the Phase II CENTAUR trial, has been permitted following recent discussions with the FDA. As a result of the FDA’s initial request in April 2021 for Amylyx to conduct an additional Phase III trial before filing for AMX0035’s approval, the company planned to initiate a global Phase III PHOENIX study on a larger pool of 600 participants that was expected to conclude in Q4 2023. However, with the recent shift in the FDA’s stance, it is likely that AMX0035 will be available for ALS patients much sooner than anticipated. Given AMX0035’s clinical evidence and commercial potential, it is poised to become a welcome addition to the ALS space and to establish a new treatment paradigm in this market, where there are limited players and a lack of effective treatment.

AMX0035 is a combination of two orally administered small molecules, tauroursodeoxycholic acid (TUDCA) and sodium phenylbutyrate, that protects nerve cells from damage by targeting the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS patients. These molecules have been used clinically in various indications and therefore they have a good safety and tolerability profile. The data from the Phase II CENTAUR study showed that AMX0035 met its primary endpoint of slowing decline in function as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) and modestly prolonged patient survival by 6.5 months versus placebo. Based on those results, AMX0035 has been filed for approval in Canada in June 2021 and is expected to be filed in the EU by the end of 2021.

As AMX0035 targets a serious neurological condition with huge clinical unmet needs, it could be granted an FDA accelerated approval within one year from the NDA filing if the FDA follows a similar path to the controversial approval of Biogen’s Aduhelm in Alzheimer’s disease earlier this year. Key opinion leaders (KOLs) previously interviewed by GlobalData believed that AMX0035 has a favorable oral route of administration and well-known safety and tolerability profile. As such, they indicated that if AMX0035 continues to show therapeutic benefits in trials, they will likely prescribe it to the majority of their patients upon approval, as it would address some clinical unmet needs and expand the existing treatment options, which currently consist of only two drugs: Sanofi’s Rilutek (riluzole) and Mitsubishi Tanabe’s Radicava (edaravone). Additionally, they hoped that the drug would be priced reasonably due to the availability of its two constituents.

KOLs also noted that given the devastating nature of ALS and the average short life expectancy of patients, there is strong public pressure to get more drugs approved for this disease despite showing modest benefits on survival and slowing disease progression. As such, GlobalData expects AMX0035 to gain a moderate slice of the

ALS market, which is expected to grow at a Compound Annual Growth Rate of 13.9% and reach $1.04B in global sales by 2029, driven by pipeline launches including AMX0035’s.

Overall, GlobalData anticipates that the need for a cure remains. The potential approval of new therapies in this stagnant market will likely encourage further investments in developing innovative pipeline agents that try to halt or slow the progression of the disease instead of reformulating available ALS treatments.

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