Shares of biotechnology company Intellia Therapeutics Inc (NASDAQ: NTLA) are trading near 13-month lows with the stock down over 70% compared to the all-time high set last June. Intellia is a pioneering biotechnology company that develops biopharmacueticals using CRISPR/cas9 genome editing.
FDA Designation Obtained
In March, Intellia announced it obtained the orphan drug designation from the U.S. Food and Drug Administration (FDA) for its ex vivo investigational T cell receptor (TCR)-T cell therapy, NTLA-5001 to treat patients suffering from acute myeloid leukemia (AML).
NTLA-5001 refers to an autologous TCR-T cell therapy developed by Intellia to target the Wilms’ Tumor (WT1) antigen, which is often present in patients with acute myeloid leukemia, as well as other hematologic and solid tumors.
At the moment, the therapy is being tested in the Phase 1/2a study in adults suffering from persistent or recurrent AML who have been previously treated.
“The FDA’s decision to grant orphan drug designation for NTLA-5001 reflects the serious need for novel treatment options for people living with AML, a disease with notably poor long-term survival,” said John Leonard, M.D., CEO, and President of Intellia Therapeutics.
The Orphan Drug Designation program is an FDA program that grants orphan status to therapies developed for the treatment, diagnosis, or prevention of rare illnesses that are present in less than 200,000 people in the U.S.
The status makes the drug developers eligible for development incentives such as tax credits for qualified clinical studies, 7-year marketing exclusivity upon obtaining the status, and exemption from the prescription drug user fee program (PDUFA).
In February, Intellia published positive interim data from the Phase 1 study of NTLA-200 therapy. The data showed that the NTLA-2001 candidate reduced serum TTR, recording maximal reductions by day 28, including mean reductions of 52%, 87%, and 86% in three patients who were separated into three 0.1 mg/kg, 0.3 mg/kg, and 0.7 mg/kg dose groups. Furthermore, the reduction level rose to 93% among the six patients who were in the 1.0 mg/kg dose group.
The recorded reductions remained sustained during the observation period, which ranged from 2 to 12 months. Furthermore, the serum TTR reduction also remained consistent among all patients who were in dose groups higher than 0.3 mg/kg, while six patients who received the 1.0 mg/kg dose have seen more than 80% reduction, four of which achieved a maximum reduction of 90% by day 28.
In general, the NTLA-2001 treatment was well tolerated across all four dose groups, with most of the reported adverse effects remaining at mild levels. The majority of adverse events included headache, back pain, rash, and nausea.
Business Objectives Presented
Intellia has clear strategic priorities and landmarks it expects to achieve this year including:
- Advancing the clinical validation of in vivo pipeline. In other words, the company expects to continue characterizing the efficacy and safety of its NTLA-2001 drug, as well as set up the initial clinical profile of NTLA-2002 as a single-dose therapy to treat patients suffering from hereditary angioedema (HAE).
- Grow its in vivo and ex vivo pipelines, including the establishment of the initial safety profile of NTLA-5001 to treat AML and nominate a number of new development candidates.
- Enhance the Intellia platform by expanding its genome editing, delivery, and cell engineering abilities.
“Unequivocally, 2021 was a landmark year for Intellia. We demonstrated that our proprietary CRISPR-based platform and LNP technology can turn revolutionary science into potentially transformational medicines. Our platform enables us to advance genome editing approaches, which maximizes our ability to target a multitude of life-threatening diseases,” added Leonard.
He added that Intellia is well-positioned to considerably expand the full-spectrum pipeline of its therapies, with plans to nominate a minimum of two more in vivo candidates as well as the company’s first allogeneic development candidate later this year.
The company anticipates sharing new data from the current NTLA-2001 study and interim results of the NTLA-2002 Phase 1/2 study, which it expects will emphasize the modularity of its genome editing platform.
While Intellia continues to share positive business updates, shares are still under pressure in response to the tightening financial conditions. Breakthrough biopharmaceutical products are extremely costly to develop and pose ongoing financial obstacles for biotech companies. Intellia stock price trades in a downward channel with the supporting trend line coming in at around $40.
However, analysts offering 12-month price forecasts for Intellia have a median target of $165, representing a +201% increase from the recent $54.80 price. With a high estimate of $207 and low of $83, Intellia is a buy for investors willing to wait for the company’s bounce back over the next 12 months.
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