Amylyx's ALS drug Relyvrio gets off the ground as company eyes new brain disorder

Amylyx Pharmaceuticals’ amyotrophic lateral sclerosis (ALS) drug Relyvrio continues to wow Wall Street.

Approved in the U.S. as Relyvrio and in Canada under a different brand name, the drug brought in $98.2 million in revenue during the second quarter, Amylyx said Thursday. The haul topped analysts’ consensus estimates of below $92 million, according to Mizuho Securities.

Having annual revenue now tracking at $400 million after just four quarters of commercialization is no easy feat, Mizuho analyst Graig Suvannavejh, Ph.D., said in a Friday note. 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,” he said.

The beat was fueled by better-than-expected pricing discounts, according to Suvannavejh.

New patient starts actually slowed. By the end of June, Relyvrio added 800 more patients compared with the end of March, bringing the total tally to 3,800, Amylyx’s co-CEO Justin Klee told investors during a call Thursday. That’s compared with a gain of 1,700 new patients reported in the first quarter. The decline in starts, as Suvannavejh noted, was expected.

These patients are staying on treatment, too. About 70% of patients remained on the drug after treatment, which is similar to that observed in Relyvrio’s phase 2 clinical trial, Klee said. People may stop treatment because of death or disease progression, he added.

Almost all patients living with ALS have an insurance policy covering Relyvrio, Klee said. Around 75% of the top 500 prescribers and almost all top ALS treatment centers in the U.S. have prescribed the drug, Amylyx’s commercial chief Margaret Olinger said. The turnaround time between prescription and product shipment has also improved to about 25 days from around 30 days in the first quarter.

But given that Amylyx didn’t provide sales guidance, debates will likely continue about the drug’s future performance and its momentum in bringing on new patients, Suvannavejh said.

Outside North America, Amylyx is fighting a negative opinion from drug reviewers at the European Medicines Agency. The EU regulator is expected to make a final decision this fall.

Relyvrio’s FDA approval in ALS was a hard-fought one with questions still lingering about its efficacy. Suvannavejh also acknowledged that uncertainty exists around whether Relyvrio wil succeed in the confirmatory phase 3 Phoenix trial, which is expected to read out in the first half of 2024. But he argued that Relyvrio, also known as AMX0035, is set to become a “foundational standard of care” in ALS, with “little near-term, mid-term and likely longer-term threat.” 

The FDA in April approved Biogen and Ionis’ Qalsody to treat ALS associated with a mutation in the SOD1 gene which represents a meager 2% of the entire ALS population. Earlier this year, Apellis and Sobi called it quits on the development of their C3 inhibitor pegcetacoplan—sold as Empaveli and Syfovre in other diseases—for ALS.

As Amylyx awaits the phase 3 ALS readout, the company expects to launch a new phase 3 trial called Orion, for the drug to treat another rare neurological disorder called progressive supranuclear palsy (PSP) by year-end. The plan is to enroll 600 patients for a disease that affects seven in 100,000 people worldwide.