Avidity Biosciences unveiled "impressive" results from a study of its treatment for a rare form of muscular dystrophy. The top-rated biotech stock soared to a record high.
The company is working on a treatment called del-brax for patients with facioscapulohumeral muscular dystrophy, or FSHD. In this condition, mutations in a gene called DUX4 cause loss of muscle function, pain, fatigue and progressive disability.
After four months, patients treated with del-brax had a greater than 50% average reduction in DUX4 regulated genes. All patients showed reductions greater than 20%. Avidity previously said it would have been happy with a 20% change, Leerink Partners analyst Joseph Schwartz said in a report.
"Overall, we see this data as impressive, especially considering the data is from the first and lowest dose (group)," he said. "Furthermore, the data support that targeting DUX4 directly can have a positive impact on function as early as four months and are clearly the fruit of Avidity's rigorous approach in FSHD."
Avidity stock rocketed 32.6% to 38.36. Earlier, shares hit a record high at 39.25. The biotech stock has a best-possible IBD Digital Composite Rating of 99. This means shares have performed in the top 1% of all stocks over the past 12 months.
Biotech Stock: Targeting The Underlying Cause
It's important to note, abnormal expression of the DUX4 gene is the root cause of the disease, Chardan Research analyst Keay Nakae said in a report. He has a buy rating and 45 price target on Avidity stock.
Notably, patients treated with del-brax also showed improvements in upper and lower limb strength and muscle function compared to the placebo group and the natural course of the disease.
Biomarkers of FHSD also declined by an average of 25% or greater.
The results "provide further validation of Avidity's (antibody oligonucleotide conjugates) design platform as an enabling technology for the successful development of small RNA therapeutics in (outside the liver) targets," Nakae said.
Antibody oligonucleotide conjugates are drugs that brings medicine to a specific cell.
Leerink's Schwartz also reiterated his outperform rating on the biotech stock.
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