Wave Life Sciences Reports Third Quarter 2022 Financial Results and Provides Business Update


Wave Life Sciences Ltd

And as we share today, 006, our first AIMer for Alpha-1 antitrypsin deficiency, we shared that one of the advantages to being able to get hold and durable editing was a function of being able to apply PN chemistry on top of our coyote control molecules. As it relates to C9, we’ll have continued data on both then multi and single dose so again, continue to be able to look at the pharmacokinetics in target engagement. Turn it over, Mike for any additional follow up on that, but that’s going to be the key. The robust data set across multiple programs, all related to PN that answer key questions related to target engagement and various tissues to speak, CNS versus muscle, as well as being able to look at individual disease indications for cross silencing and splicing.

Approximately 30,000 people in the United States have symptomatic HD and more than 200,000 others are at risk for developing the disease. There are currently no approved disease-modifying therapies available. Wave expects that its existing cash, cash equivalents and short-term investments will enable the company to fund its operating Wave Life Sciences Ltd and capital expenditure requirements into the second quarter of 2023. Pfizer hasn’t exercised its option to license any Wave programs yet, but big pharma isn’t about to walk away from its up-front investment without an objective assessment. Additional single-dose biomarker and safety clinical data expected in 1H 2023.

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Suvodirsen is the first of what the company hoped would be a long line of “stereopure” RNA-based drugs, and an improvement over the current exon-51-skipping therapy for DMD patients, Exondys 51 from Sarepta Therapeutics. Now that the first stereopure candidate to enter clinical trials has failed, though, there won’t be much enthusiasm out there for the next candidates produced by Wave’s proprietary drug discovery platform. Yes, I mean, yes, no, it’s great question, because I think it’s data that we’ve shared, and we’re happy to continue to share more. Well, we have shared in the past is with the PN edition of PN chemistry, we do see much deeper, broader penetration across the CNS. So that’s translating the uptake across tissues, the data that we share, related to Huntington reduction from the backage D model, it’s in the corporate deck, is striatal data. So what we’ve seen differently with the application of PN is broader, deeper penetration. So these data continue to help, will help clarify for us preclinical, or clinical translation as we measure this, but we’ve seen that it’s not tissue specific either.

Wave intends to use the additional capital to accelerate its RNA editing capability, led by its AATD program. Duchenne muscular dystrophy is a fatal X-linked genetic neuromuscular disorder caused predominantly by out-of-frame deletions in the dystrophin gene, resulting in absent or defective dystrophin protein. Dystrophin protein is needed for normal muscle maintenance and operation. Because of the genetic mutations in DMD, the body cannot produce functional dystrophin, which results in progressive and irreversible loss of muscle function, including the heart and lungs.

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PRISM is Wave Life Sciences’ proprietary discovery and drug development platform that enables genetically defined diseases to be targeted with stereopure oligonucleotides across multiple therapeutic modalities, including silencing, splicing and editing. PRISM combines the company’s unique ability to construct stereopure oligonucleotides with a deep understanding of how the interplay among oligonucleotide sequence, chemistry and backbone stereochemistry impacts key pharmacological properties. Over the past decade, we have established a disruptive https://www.wave-accounting.net/ oligonucleotide platform and are now seeing these innovations translated in the clinic. Our unique guide strands enable us to target diverse biology with multiple therapeutic modalities. In addition to what we have shared today around antisense, splicing, and RNA editing, we have previously demonstrated how PRISM can significantly improve SI RNA silencing in vivo over industry, state-of-the-art and advanced DSD plus chemistry. These capabilities, together with our GMP manufacturing position as well to become a leading genetic medicines company.

In June, we completed a successful following offering which put Wave in a strong well capitalized position entering the second half of 2022. We continue to execute on multiple pillars of value creation, including delivering clinical updates, and rapidly advancing our novel RNA based editing pipeline led by WVE-006. We also continue to advance discussions with potential strategic partners who share our vision for broad applications of Wave platform and support expansion of our pipeline for unlocking value in GMP manufacturing. In the second quarter, we continue to make meaningful progress both on our therapeutic program and our platform.