Amylyx Pharmaceuticals Q1 2024 Financial Results

Amylyx Pharmaceuticals, Inc. released its first-quarter financial results for 2024, highlighting key developments in its clinical programs and financial performance. The company has taken significant steps to focus on its crucial clinical and preclinical projects, aiming to address unmet needs in neurodegenerative diseases.

Amylyx plans to engage with the FDA based on interim data from its Phase 2 HELIOS trial for AMX0035 in Wolfram syndrome. The trial showed improvements in pancreatic function, glycemic control, and vision among participants. The company expects to release topline data from all 12 participants by fall 2024. Additionally, Amylyx is set to begin a clinical trial of AMX0114 in ALS in the latter half of 2024, after completing IND-enabling studies.

In April 2024, Amylyx decided to discontinue marketing authorizations for RELYVRIO®/ALBRIOZA™ in the U.S. and Canada based on the Phase 3 PHOENIX trial results. This decision was informed by engagement with regulatory authorities and discussions with the ALS community. Current patients will be transitioned to a free drug program. This strategic move is part of a broader restructuring to allocate financial resources toward key initiatives and extend the cash runway into 2026.

Interim results from the HELIOS trial involving eight adults with Wolfram syndrome indicated positive outcomes in pancreatic beta cell function, glycemic control, and vision. AMX0035 was well-tolerated, with no dropouts reported. This rare genetic disorder affects around 3,000 people in the U.S. AMX0035 targets ER stress and mitochondrial dysfunction, showing potential benefits.

The ORION study on AMX0035 for progressive supranuclear palsy (PSP) expects interim data by mid-2025. This Phase 3 trial aims to evaluate the efficacy, safety, and tolerability of AMX0035 in PSP patients, a rare neurological disorder characterized by neurodegeneration linked to tau protein deposition. Previous studies indicated that AMX0035 could reduce cerebrospinal fluid tau levels.

Amylyx plans to initiate a clinical trial for AMX0114 in ALS in the second half of 2024. AMX0114, a potent antisense oligonucleotide, targets the inhibition of calpain-2. Preclinical studies have shown promising results in reducing neurofilament light chain levels and enhancing motor neuron survival.

Financially, Amylyx reported net product revenue of $88.6 million for the first quarter of 2024, an increase from $71.4 million in the same period of 2023. However, the company also reported a net loss of $118.8 million, primarily due to non-cash charges associated with inventory write-downs and firm purchase commitments related to the discontinuation of RELYVRIO/ALBRIOZA. Research and development expenses rose to $36.6 million, driven by increased spending on the Phase 3 ORION study and preclinical activities. Selling, general, and administrative expenses also increased to $57.8 million.

As of March 31, 2024, Amylyx had $373.3 million in cash, cash equivalents, and short-term investments, providing an expected cash runway into 2026. This financial position is expected to support upcoming milestones, including data readouts from the HELIOS and ORION trials and the planned AMX0114 trial.

The management team hosted a webcast to discuss the interim data from the HELIOS trial and financial results. The company's strategic focus on its key clinical programs aims to bring innovative therapies to patients with neurodegenerative diseases, fulfilling its mission to alleviate suffering caused by these conditions.