Wave Life Sciences Q2 2024 Financial Results and Business Update

16 August 2024

Wave Life Sciences Ltd., a clinical-stage biotechnology company specializing in RNA medicines, has released its second-quarter financial results for 2024 and shared significant business updates. The company's RNA medicines platform has recently received further validation through positive clinical data from the SELECT-HD trial in Huntington's Disease (HD) patients.

Wave Life Sciences has made notable advancements in several key areas:

Huntington's Disease (HD):
Wave's WVE-003 is an allele-selective oligonucleotide designed to lower mutant huntingtin (mHTT) protein while preserving the healthy, wild-type huntingtin (wtHTT) protein. Positive data from the Phase 1b/2a SELECT-HD study of WVE-003 demonstrated significant, potent, and durable reductions in CSF mHTT and preservation of healthy protein. The study showed a correlation between mHTT reductions and the slowing of caudate atrophy, suggesting potential benefits. WVE-003 was generally safe and well-tolerated. The company has submitted an opt-in package to its partner, Takeda, and initiated discussions with regulators on a path to accelerated approval.

Duchenne Muscular Dystrophy (DMD)
Wave's WVE-N531 is an exon-skipping oligonucleotide designed to induce the production of functional dystrophin protein in DMD patients amenable to exon 53 skipping. Earlier studies showed industry-leading exon skipping levels and significant muscle tissue concentrations. The FORWARD-53 trial is currently evaluating WVE-N531, with the potential to become a registrational trial. Positive results could pave the way for a broader DMD pipeline of PN-modified oligonucleotides targeting other exons.

Alpha-1 Antitrypsin Deficiency (AATD): 
WVE-006 is a GalNAc-conjugated RNA editing oligonucleotide aimed at treating both lung and liver disease associated with AATD. Unlike other treatments, WVE-006 does not require a lipid-nanoparticle delivery system. The RestorAATion-2 Phase 1b/2a study is currently underway, with dosing initiated in the third quarter of 2024. Proof-of-mechanism data is expected by the fourth quarter of 2024.

Obesity: 
WVE-007 is a GalNAc-siRNA designed to target the INHBE gene, which plays a role in inducing lipolysis while preserving muscle mass. Preclinical studies have shown promising results, with potent and durable gene silencing and significant weight loss. Wave plans to initiate a clinical trial for WVE-007 in the first quarter of 2025.

Looking ahead, Wave is set to hold an R&D Day in Fall 2024, where it will present new preclinical data and highlight its innovations in RNA medicines. The company is also expanding its pipeline of RNA therapeutics, targeting a range of hepatic and extra-hepatic conditions. This expansion is supported by genetic datasets and deep learning models, which help identify new RNA editing targets.

Financial Highlights:
As of June 30, 2024, Wave reported cash and cash equivalents of $154.0 million, down from $200.4 million at the end of 2023. The company expects its current cash reserves to fund operations into the fourth quarter of 2025. Revenue for the second quarter of 2024 was $19.7 million, compared to $22.1 million in the same period of 2023. Research and development expenses increased to $40.4 million, while general and administrative expenses were $14.3 million. The net loss for the second quarter of 2024 was $32.9 million.

Wave Life Sciences continues to push forward with its innovative RNA medicines platform, aiming to provide transformative treatments for a range of serious diseases. The company's recent clinical successes and ongoing research efforts are positioning it to make significant strides in the biotech industry.

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