• Collaboration Revenue: $19.7 million in Q2 2024, compared to $22.1 million in the prior year quarter.
• Research and Development Expenses: $40.4 million in Q2 2024, up from $33.3 million in the prior year quarter.
• General and Administrative Expenses: $14.3 million in Q2 2024, compared to $12.3 million in the prior year quarter.
• Net Loss: $32.9 million for Q2 2024, compared to $21.1 million in the prior year quarter.
• Cash and Cash Equivalents: $154 million at the end of Q2 2024.
• Cash Runway: Expected to fund operations into Q4 2025.

• Warning! GuruFocus has detected 6 Warning Signs with WVE.

Release Date: August 08, 2024

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• WAVE Life Sciences Ltd (NASDAQ:WVE) reported positive results from the SELECT-HD clinical trial for WVE-003, showing potent and durable mutant protein lowering in Huntington's disease.
• The company has initiated engagement with regulators on a clinical development path for WVE-003 that could support accelerated approval.
• WVE-N531, the exon-skipping candidate for Duchenne Muscular Dystrophy (DMD), demonstrated industry-leading exon skipping and unprecedented muscle concentrations in Part A of the clinical trial.
• WVE-006, the first-in-class GalNAc RNA editing candidate for AATD, has shown promising preclinical results, including increased wild-type protein levels and reduced liver aggregates.
• WVE-007, a novel siRNA approach for obesity, demonstrated highly potent and durable silencing in preclinical studies, with potential for infrequent dosing intervals of once or twice a year.

Negative Points

• WAVE Life Sciences Ltd (NASDAQ:WVE) reported a net loss of $32.9 million for the second quarter of 2024, an increase from $21.1 million in the prior year quarter.
• Research and development expenses increased to $40.4 million, driven by spending on the INHBE program and other pipeline initiatives.
• The company faces significant scientific gaps in the functional benefits and durability of current DMD therapies, highlighting the need for better treatment options.
• There are uncertainties regarding the regulatory feedback and opt-in decision from Takeda, which could impact the future of the HD program.
• The competitive landscape for AATD treatments poses challenges, with questions about the translatability of preclinical dose-finding studies to humans.

Q & A Highlights

Q: Regarding the conversations with regulators, is Takeda involved in those conversations? Or is it just between you and the FDA? And what has been the feedback so far on the possibility of using imaging as a surrogate biomarker? A: Takeda is involved in our interactions with regulators. We don't comment on specific regulatory interactions at this point in time.

Q: On DMD, looking at the approved antisense oligos in DMD, about 42% exon skipping for the 53 from Viltepso leads to around 4.8% dystrophin. Is the bar for N531 around 5%? A: We aim for dystrophin expression greater than 5%. The 42% exon skipping from Viltepso was after six months of dosing, while we saw 53% after just three doses at six weeks. Consistency of response is key, and we believe our approach will drive a differentiated profile.

Q: Could you clarify if the feedback from Takeda and the regulatory body will be one event or two separate events? A: These are two independent events. The Takeda opt-in decision is based on data from the prior study and not predicated on regulatory feedback. Updates will be provided as they occur, with timelines taking us to the end of the year.

Q: If Takeda chooses not to opt in, what are your options for the program? A: We are not waiting for Takeda's decision and have received interest from other strategics. We are ensuring the program is positioned well to continue to patients, should there be alignment with regulators on a path to accelerated registration.

Q: For the RestorAATion-2 update in Q4, do you plan to show changes in AAT protein concentration or just the proportion of corrected M-AAT versus Z-AAT? A: The initial proof of mechanism data will evaluate both M-AAT protein and total protein. This will be the first demonstration of RNA editing in humans.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.