Amylyx Pharmaceuticals continues to impress the financial world with its amyotrophic lateral sclerosis (ALS) drug, Relyvrio. The medication, known by a different name in Canada, generated an impressive $98.2 million in revenue during the second quarter, surpassing analysts’ estimates of around $92 million, according to Mizuho Securities.
“Having annual revenue now tracking at $400 million after just four quarters of commercialization is no easy feat. Besides, a strong balance sheet—Amylyx had $357 million cash as of June—means the company faces no existential need to raise capital—an increasingly daunting challenge given a difficult equity capital markets environment. The beat was fueled by better-than-expected pricing discounts.”
– Mizuho analyst Graig Suvannavejh, Ph.D.
Despite a slowdown in new patient enrollments compared to the previous quarter, with 800 additional patients joining by the end of June, co-CEO Justin Klee assured investors during a recent call that this decline was anticipated. The total patient count reached 3,800, in contrast to the 1,700 new patients in the first quarter.
The drug’s retention rate is notable, with roughly 70% of patients staying on the treatment after its initiation. Klee highlighted that this retention rate aligns with the outcomes observed in Relyvrio’s Phase II clinical trial. However, some patients might discontinue treatment due to disease progression or unfortunate outcomes.
Amylyx’s commercial chief, Margaret Olinger, revealed that nearly all ALS patients with insurance coverage can access Relyvrio. Furthermore, approximately 75% of the top 500 prescribers and the majority of leading ALS treatment centers in the US. have already prescribed the drug. The company has also managed to streamline the process, reducing the turnaround time between prescription and product shipment to about 25 days from the initial 30 days.
Nevertheless, since Amylyx refrained from providing sales projections, discussions about the drug’s future performance and its ability to attract new patients are likely to persist, according to insights shared by Suvannavejh.
Beyond North America, Amylyx faces a challenge in the form of a negative opinion from European Medicines Agency reviewers. A final decision from the EU regulator is anticipated in the upcoming fall.
The path to FDA approval for Relyvrio in ALS was marked by challenges and ongoing questions about its effectiveness. Suvannavejh acknowledged that uncertainties also linger around the drug’s potential success in the confirmatory Phase III Phoenix trial, projected to conclude in the first half of 2024. However, he contended that Relyvrio, also known as AMX0035, is positioned to establish itself as a “foundational standard of care” in the ALS landscape, with minimal near- and long-term threats.
In a contrasting development, the FDA granted approval to Biogen and Ionis’ Qalsody in April, targeting ALS associated with a SOD1 gene mutation—a condition affecting just 2% of the ALS population. Additionally, Apellis and Sobi discontinued their C3 inhibitor pegcetacoplan’s development for ALS this year.
While awaiting the results of the Phase III ALS trial, Amylyx is gearing up to initiate the Orion Phase III trial by year-end. Orion will focus on evaluating Relyvrio’s potential to treat progressive supranuclear palsy (PSP), a rare neurological disorder affecting seven in every 100,000 individuals globally.
