Shares of Wave Life Sciences (WVE) surged to a four-year high Wednesday after the company announced "impressive" proof-of-concept results for its new technology in two patients with a genetic disease.
The biotech company tested its experimental treatment in patients with alpha-1 antitrypsin deficiency. In this condition, the liver doesn't make enough — or any — of the AAT protein. This protein helps protect the lungs from damage. Without it, the lungs can become more easily damaged from smoking, pollution or dust.
But Wave's new means of editing RNA helped the patients generate a robust amount of the missing AAT protein 15 days after receiving a single dose. RNA is the messenger from DNA that carries the instructions for making proteins.
"This is the first clinical data point for RNA editing (from Wave Life Sciences or any others, as far as we know)," Mizuho Securities analyst Salim Syed said in a report. "And proof-of-concept for the platform."
Wave Life Sciences stock skyrocketed 73.9% to 14.88. Shares touched their highest point since September 2020, according to MarketSurge. The results also bolstered shares of Korro Bio (KRRO), which launched 131.2% to 91.94. William Blair analyst Myles Minter says Korro could have the best-in-class messenger RNA-editing platform.
It's important to note, both patients are considered homozygous. This means they have two copies of the problematic gene attached to alpha-1 antitrypsin deficiency, or AATD.
"Therefore any production (of AAT protein) at all is proof-of-mechanism," Mizuho's Syed said.
More than 60% of the AAT protein in patients' plasma was wild-type — meaning unmutated. The threshold for clinical meaningfulness is 50%. This could be enough to lower the disease's risk, Syed said. Wave Life Sciences is also planning to test higher doses with results expected in 2025.
He has an outperform rating and 19 price target on Wave stock.
Leerink Partners analyst Joseph Schwartz, who classified Wave's results as "impressive," also noted Takeda Pharmaceutical opted out of another Wave Life Sciences program in a filing late Tuesday. Now, Wave is free to go alone or shop around for another partner for its Huntington's disease drug.
https://www.investors.com/news/technology/wave-life-sciences-rna-editing-alpha-1-antitrypsin/
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