Shares of Wave Life Sciences WVE surged 74.1% on Wednesday after the company reported positive proof-of-mechanism data from an ongoing early to mid-stage study of its investigational candidate, WVE-006, for alpha-1 antitrypsin deficiency (AATD), a type of genetic disorder.
WVE-006 is a GalNAc-conjugated oligonucleotide that enables A-to-I RNA editing and is administered subcutaneously. Developed with Wave Life Sciences’ advanced oligonucleotide chemistry platform, it is specifically designed to target lung disease, liver disease, or both, related to AATD.
Per the company, approximately 200,000 individuals in the United States and EU have AATD due to the SERPINA1 Z mutation. Current treatments are limited to weekly IV augmentation therapy, which targets lung disease and generated more than $1.4 billion in global sales in 2023. However, no approved therapies exist for AATD-related liver disease, often leading to liver transplants for affected individuals.
WVE Achieves First-Ever Therapeutic RNA Editing in AATD Study
In the latest press release, Wave Life Sciences reported proof-of-mechanism data from the first single-dose cohort (200 mg) of the phase Ib/IIa RestorAATion-2 study of WVE-006 involving the first two patients with “ZZ” AATD (Pi*ZZ AATD), who reached day 57 of the treatment period.
This is a landmark achievement for the company as the results constitute the first-ever clinical demonstration of RNA editing in humans.
People with Pi*ZZ AATD do not naturally produce wild-type alpha-1 antitrypsin (M-AAT) protein. Thus, detecting the M-AAT protein confirms that mutant Z-AAT mRNA has been successfully edited. Restoring 50% M-AAT levels would be similar to having the heterozygous “MZ” genotype, which is associated with a lower risk of AATD-related lung and liver disease.
Year to date, shares of Wave Life Sciences have skyrocketed 195% against the industry’s 1% decline.
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By day 15 in the RestorAATion-2 study of WVE-006, circulating wild-type M-AAT protein in plasma reached an average of 6.9 micromolar, accounting for more than 60% of total AAT. This increase corresponded with enhanced neutrophil elastase inhibition, indicating functional M-AAT production.
Total AAT protein rose from undetectable levels at baseline to 10.8 micromolar at day 15, reaching levels required for regulatory approval of AAT augmentation therapies. Both M-AAT and total AAT increases were observed as early as day three and continued through day 57.
Wave Life Sciences also reported that WVE-006 was overall well-tolerated and demonstrated a favorable safety profile. Adverse events related to treatment with the candidate to date were mild to moderate in severity.
The company expects to report multidose data from the phase Ib/IIa RestorAATion-2 study of WVE-006 for AATD in 2025. Based on the encouraging single-dose results, management expects M-AAT levels to continue to increase with repeat dosing of WVE-006.
Please note that Wave Life Sciences has an existing collaboration agreement with GSK plc GSK, which grants the latter an exclusive global license for WVE-006. Under the terms of the agreement, GSK will assume all developmental and commercialization responsibilities after Wave completes the RestorAATion-2 study of the candidate. In consideration for the license grant, WVE is eligible to receive up to $525 million in milestone payments, as well as tiered royalties on net sales, from GSK, for WVE-006.
WVE News Sparks a Stock Price Rally for Korro Bio
Shares of Korro Bio KRRO, a preclinical-stage company, also rallied 93.7% on Wednesday following the data readout by Wave Life Sciences. Korro Bio is currently focusing on developing its lead pre-clinical candidate, KRRO-110, for the treatment of AATD, using its proprietary RNA editing approach.
Positive proof-of-mechanism data from WVE’s mid-stage study of WVE-006 for the same indication is likely to instill faith in investors regarding KRRO-110’s potential for success in treating AATD, which follows a similar mechanism of action, driving the stock price rally for Korro Bio.
Korro Bio is currently conducting preclinical studies on KRRO-110 in preparation for a potential regulatory filing by the end of 2024.
WAVE Life Sciences Ltd. Price and Consensus
WAVE Life Sciences Ltd. Price and Consensus
WAVE Life Sciences Ltd. price-consensus-chart | WAVE Life Sciences Ltd. Quote
WVE’s Pipeline Programs Beyond AATD
Apart from the AATD indication, there are several other candidates like WVE-N531, WVE-003 and WVE-007 in Wave Life Sciences’ pipeline, which are being developed for rare and prevalent diseases, including Duchenne muscular dystrophy, Huntington’s disease and obesity, respectively.
In the same press release, the company stated that it will introduce the next RNA editing programs and provide an update on its obesity program at its Research Day on Oct. 30, 2024.
WVE’s Zacks Rank and Other Stock to Consider
Wave Life Sciences currently carries a Zacks Rank #2 (Buy).
Another top-ranked stock in the biotech sector is Amicus Therapeutics FOLD, which carries a Zacks Rank #2 at present. You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.
In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share (EPS) have moved up from $4.69 to $4.81. EPS estimates for 2025 have improved from $5.37 to $5.86, during the same period. Year to date, shares of ANIP have gained 8.2%.
ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 31.32%.
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