Amylyx Pharmaceuticals, Inc.

Good afternoon, and thank you for joining us today to discuss our first quarter 2023 earnings. With me on the call are Josh Cohen and Justin Klee, our co-CEOs, Margaret Olinger, our chief commercial officer, and Jim Friedes, our chief financial officer. Before we begin, I would like to remind everyone that any statements we make or information presented on this call that are not historical facts are forward-looking statements that are based on our current beliefs, plans, and expectations, and are made pertinent to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, our expectations with results to Relivrio and Albreoza, statements regarding regulatory developments and the expected timing thereof, our business strategy and outlook, and our expected financial performance. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risks, uncertainties, and other factors, including those set forth in our most recent filings with the SEC and any other future filings that we may make with the SEC. You are cautioned not to place any undue reliance on these forward-looking statements, and AMALX disclaims any obligation to update such statements unless required by law. Now, I will turn the call over to Justin.

Thank you, Lindsay, and good afternoon. As everyone on this call knows, there is an enormous unmet need in ALS and other relentlessly progressive and fatal neurodegenerative diseases. It is our mission at Amelix to one day end the suffering caused by these diseases. Our treatment Relivrio enables us to make meaningful advances toward and continued investment in our mission. During the first quarter, we generated 71.4 million in net product revenues, reflecting the significant progress we continue to make toward our goal of ensuring every eligible person living with ALS has access to Relivrio. Relivrio is the first and only approved treatment for ALS that, in a randomized placebo-controlled trial, both met its pre-specified primary outcome demonstrating a statistically significant benefit in function and showed a benefit on survival in a longer-term post-hoc analysis. These data suggest that ALS can, in fact, be treated. The full approval of Relivrio is an important step toward ALS one day being a manageable disease. The ALS community has been waiting for meaningful treatment options since the disease was first characterized over 150 years ago, and we believe that Relivrio will eventually be a foundational therapy for ALS. In the first quarter, we saw a continued high level of interest from the ALS community in Relivrio, broadened insurance coverage, and high levels of engagement with our AMLEX care team, also known as ACTS. Just two quarters into launch, over 10% of the approximately 29,000 people living with ALS in the U.S. are now on Relivrio. Even with that success in our first six months, we have more to do. There remain many more thousands of people living with ALS in the U.S., and at least 200,000 people living with ALS globally. We are still in the early stages of our journey, and our team remains hard at work. Shifting to our plans internationally, we are diligently preparing to help bring our medicine to all eligible people living with ALS around the world. The regulatory review process in Europe remains ongoing, and we continue to expect an opinion from CHMP mid-year and a decision at the earliest in Q3. Overall, we believe we have a strong scientific position that is supported broadly by the ALS medical and advocacy community. and a capable team who is leading our process. We continue to prepare to execute a successful launch in the EU if approved. We are proud of our team's progress on advancing our mission. And now, I'll turn the call over to Margaret to share a commercial update.

Thank you, Justin. As we enter the seventh month since our U.S. launch, we remain focused on three key priorities. The first, is our effort to drive awareness and education about Relivrio among people living with ALS and clinicians. This includes educating that Relivrio is the first approved drug for adults with ALS to demonstrate a statistically significant benefit and function in a clinical trial, as well as a benefit on survival in a longer-term post-hoc analysis. Our efforts to drive awareness are yielding strong results. We are seeing continued interest and demand for Relivrio. As of March 31st, there were roughly 3,000 people on Relivrio in the US, more than double the number of people on Relivrio at the start of the quarter. We are pleased that this many people have gained access to our important treatment. I think it's worth spending a minute to provide some additional context on the strength of our launch. While we knew there was pent-up demand, the fourth quarter and first quarter were still well ahead of our expectations. The rate of net patients' ads has begun to moderate as expected. However, we still see significant demand from people living with ALS and physicians alike. Importantly, we still have plenty of room for growth, both at the top ALS centers and the broader neurology community. Now, let me run through a few metrics that show our progress but also the growth opportunities ahead of us. By the end of the first quarter, approximately 65% of the top 500 U.S. prescribers and approximately 95% of the key ALS centers had prescribed Rolivrio to at least one person since launch. Prescribing remains fairly concentrated with roughly 80 prescribers, mostly at major ALS centers representing approximately half of all relivrio prescriptions during the quarter. While we are encouraged with these data points, we see an opportunity for broader and deeper uptake at KALS centers and the opportunity to continue to penetrate the group of top prescribers. Additionally, we believe we have a large untapped opportunity for additional growth outside of this group of top prescribers as we expand our outreach and education efforts more broadly. Our second priority is engaging with payers to work towards our goal of ensuring that every eligible person who can benefit from Rolivrio has access as quickly and efficiently as possible. At the end of the first quarter, U.S. insurers representing approximately 50% of covered lives have published formal coverage policies. including many of the key national and regional plans. The vast majority of these insurers provide broad access to Relivrio. For people living with ALS who go through the medical exception process, we are pleased that approximately 80% of prior authorization requests have been approved on the first admission. We continue to see a wide range of people living with ALS. In terms of time since initial diagnosis, interested in and gaining access to Relivrio. Our team remains engaged with insurers across the country, and we continue to anticipate most plans will formalize their policies during the first half of this year. Our third priority is ensuring eligible people living with ALS have positive interactions through their treatment journey with Relivrio, and ALS clinics have positive interactions with Amilex. This includes facilitating an organized, clear process to get people prescribed Rolivrio access to the therapy as quickly as possible and optimizing people's experience accessing Rolivrio as best we can. Our team is working expeditiously to get people living with ALS who have been prescribed Rolivrio on therapy. In the first quarter, on average, it took about 30 days between receiving an enrollment form And we're real being shipped down from a little more than 45 days in the fourth quarter. And we expect to make additional progress on this metric in the second quarter with the benefit of more insurers having formal coverage decisions. In the limited cases where access hasn't been granted through an insurer, our interim access program and our patient assistance program are available for eligible patients. In the first quarter, roughly 10% of people taking Relivrio were part of these programs. We are pleased with the progress we've made so far on our goal to ensure that every eligible person living with ALS has access to Relivrio, which we see as a potential foundational therapy in neurodegenerative disease. Our launch is off to a strong start, and it is our hope that eventually, Relivrio will become the most commonly used ALS medication. While we are clearly focused on the launch of Relivrio in the U.S., we also have a large opportunity internationally. ALS is a global disease affecting more than 200,000 people worldwide. Interest in Albreoza remains high in Canada, and we continue to negotiate public insurance coverage consistent with the expected public timelines for coverage. In addition, a few weeks ago, We appointed Masako Nakamura to lead our efforts in Asia Pacific and Latin America as we pursue additional opportunities to bring AMX 35 to as many people with ALS globally as possible. He brings 30 years of commercial, general management, and operational leadership experience in the biopharmaceutical industry with a strong track record of introducing rare disease therapies worldwide across multiple therapeutic areas. We are very pleased that she decided to join our team to further explore opportunities for AMX 35 access around the world. I will now turn the call over to Josh to provide an important update on our R&D pipeline.