Intellia (NTLA Quick Quote – ) shares are down 27% in the past month, compared to a 2.6% decline.
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Intellia's stock price declined primarily due to a decision adopted by the United States Patent and Trademark Office ("USPTO") Patent and Trial Appeals Board ("PTAB") on February 28th regarding patent interference litigation between two groups –The first group consists of the University of California, the University of Vienna, and Emmanuelle Charpentier (collectively, "CVC"), and the other group consists of the Broad Institute, Harvard University, and the Massachusetts Institute of Technology (collectively, the "Broad Institute"). the "Broad Institute") . Since 2016, the Broad Institute and the CVC have been engaged in a patent dispute over the use of CRISPR/Cas9 genome editing systems in eukaryotic cells (i.e., plants and animals).
On February 28, the PTAB passed a ruling in favor of the Broad Institute, saying it was the first company to invent the use of CRISPR/Cas9 genome editing in eukaryotic cells. This resulted in the invalidation of 14 of CVC's patent applications.
Intellia Therapeutics has been focused on developing therapeutic therapies using CRISPR/Cas9 technology. NTLA received an exclusive license from CVC for the development and commercialization of CRISPR/Cas9-based human therapeutics, which explains the stock price decline.
Based on an exclusive license from CVC, Intellia has filed multiple patent applications, including delivery applications and product candidates, for its technology innovations in CRISPR/Cas9 therapeutics. In a press release issued earlier this month, NTLA stated that the PTAB's decision will not affect any of the company's patent applications covering its technology innovations, nor will it affect the company's ability to develop and commercialize CRISPR-based therapeutics in the future.
At the same time, Intellia continues to rapidly develop its pipeline of candidates.
Earlier this week, the company announced that its first wholly-owned ex vivo gene therapy candidate, NTLA-5001, received orphan drug designation from the FDA for the treatment of acute myeloid leukemia. NTLA began dosing its first patient with NTLA-5001 in a Phase I/IIa study in adult participants with persistent or recurrent AML.
Last month, the company and partner Regeneron Pharmaceuticals (REGN Quick Quote – ) announced positive interim data from a Phase I study evaluating its lead in vivo genome editing candidate, NTLA-2001, in transgenic patients with polyneuropathy (ATTRv-). thyrotropin amyloidosis patients. (PN). Data from the study showed a dose-dependent reduction in serum TTR levels in patients treated with NTLA-2001, with mean reductions of 52%, 87%, 86% and 93% at day 28 for the 0.1 mg/kg, 0.3 mg, 0.7 mg/kg and 1.0 mg/kg dose groups. Both Intellia and Regeneron plan to move forward with a fixed dose of 80 mg and evaluate it in a dose expansion cohort in a Phase I study expected to begin in the first quarter of 2022.
We remind investors that Intellia is the lead party for NTLA-2001 and that Regeneron shares 25% of the development costs and commercial profits. Intellia and Regeneron are also developing treatments for hemophilia A and hemophilia B.
Intellia also initiated a Phase I/II study in December 2021 to evaluate NTLA-2002 for the treatment of hereditary angioedema ("HAE"). NTLA-2002 is designed to prevent the onset of HAE by inhibiting plasma kinin-releasing enzyme activity.
Another company affected by the PTAB decision is CRISPR Therapeutics (CRSP Quick Quote -). Similar to Intellia, CRSP has acquired the rights to develop CRISPR-based therapies from CVC.
CRISPR Therapeutics' lead drug candidate is CTX001, an investigational in vitro CRISPR gene editing therapy currently being evaluated in two independent Phase III studies for the treatment of transfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD). CRSP expects to file regulatory submissions for CTX001 in TDT and SCD indications by the end of 2022.
Zacks Rank and Stock to Consider
Intellia currently owns a Zacks Rank #4 (Sell). A higher-ranked stock in the same sector is BioDelivery Sciences (BDSI Quick Quote – ), which owns a Zacks Rank #2 (Buy). You can see.
BioDelivery Sciences' earnings per share estimates for 2022 have increased from 33 cents to 35 cents in the last 30 days. To date, BDSI's shares have soared 80%.
BioDelivery Sciences has beaten earnings estimates in three of the last four quarters and missed estimates once, with an average surprise of 33.7%.