Amylyx Pharmaceuticals, Inc.

Good morning. My name is Morgan, and I will be your conference operator today. At this time, I would like to welcome everyone to the Amelix Pharmaceuticals first quarter 2024 earnings conference call. All participants will be in a listen-only mode. After today's presentation, there will be an opportunity to ask questions. To ask a question, please press star, then the number one on your telephone keypad. If you wish to withdraw your question after you have entered the queue, please press star then the number one again. Please limit your questions to one with one follow-up. If you have additional questions, you may rejoin the queue. Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to your host, Lindsay Allen, Head of Investor Relations and Communications. Please proceed.

Good morning, and thank you for joining us today to discuss our first quarter of 2024 financial results. With me on the call are Josh Cohen and Justin Klee, our co-CEOs, Jim Fradies, our chief financial officer, and Dr. Camille Bedrosian, our chief medical 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 made based on our current police plans and expectations and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, our plans with respect to AMX 35 and AMX 114, statements regarding current and planned clinical trials, statements regarding regulatory developments and the expected timing thereof, our business strategy and outlook, and our expected financial performance and cash runway. Actual events and results could differ materially from those expressed or implied by any forward-looking statement 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 AMLEX disclaims any obligation to update such statements unless required by law. Now, I will turn the call over to Justin.

Good morning, and thank you all for joining us today. The first quarter of this year was a difficult one for AMLEX as an organization, and especially for the ALS community. The top line results from the Phoenix trial of AMX 35 and ALS were deeply disappointing and surprising, given the prior central trial results that had showed a meaningful benefit of AMX 35 for people living with ALS and supported an FDA approval. But as admission , the next steps were clear. We moved quickly to restructure our organization in order to continue our work for one day ending the suffering caused by neurodegenerative diseases. Not long after the Phoenix trial results in early April, we announced the planned interim analysis results from our study of AMX35 and Wolfram syndrome, a program we have been developing for nearly seven years. The data showed stabilization or even improvement across the major outcomes in the trial. These data strengthen our belief that we've been investigating for over a decade that targeting cell death mechanisms is an important approach across many different diseases. It is therefore critical to continue to focus our resources on matching the mechanism of our treatment candidates with the mechanisms of disease and ideally incorporating measurable biomarkers in our development that are consistent with disease progression. In that spirit and in pursuit of our mission, we continue to advance our three key programs that have been in the works for several years, AMX35 for the treatment of Wolfram Syndrome, AMX-35 for the treatment of progressive supernuclear palsy or PST. And AMX-114, our anti-sense oligonucleotide for the treatment of ALS. For Wolfram, we are seeing early evidence of benefit across multiple organ systems based on well-established outcomes such as C-peptide response. These results are consistent with our prior preclinical studies. We plan to meet with FDA to discuss next steps in the program. The PSP community continues to be excited about our trial because of the tau-lowering effects we saw with AMX35 in a prior Alzheimer's study. We expect to have data from an interim analysis of this study mid-next year. AMX114 is a potent antisense oligonucleotide targeting inhibition of calcane-2, a well-established target in a number of neurological diseases. And published data suggests calcine-2 is the primary protease that cleaves neurofilament light chain. We expect to enroll the first participants in our ALS trial later this year. Each of these programs address neurodegenerative diseases with well-defined mechanistic rationales, well-defined and measurable biomarkers, and a foundation of rigorous preclinical data upon which we have based our clinical work. all with the goal of bringing important therapies to communities with high unmet needs. And critically, we currently have the cash runway into 2026 through meaningful milestones in each of these programs. Since founding AMLEX more than 10 years ago, we have built a foundation focused on the science behind what drives cell death and degeneration. These pathways remain critically important across many diseases of high unmet medical need, and we will continue the work to further our mission. I will now turn the call over to Camille to provide an update on these key clinical programs.