Intellia Therapeutics Inc (NASDAQ:NTLA) recently announced that it received regulatory approval from the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to commence it’s phase 1 clinical trial of NTLA-2001.
The clinical trial will evaluate NTLA-2001 as a potential treatment for transthyretin amyloidosis (ATTR). They will particularly focus on treating the hereditary form of the disease with polyneuropathy (hATTR-PN). If the clinical study yields positive findings, then NTLA-2001 will become the first treatment for ATTR.
Intellia has been working towards developing NTLA-2001’s mechanism such that a single course of treatment will possibly lead to the reduction of the transthyretin (TTR) protein in patients over time. Achieving this would make NTLA-2001 an effective treatment against ATTR. The pipeline treatment is administered using the non-viral lipid nanoparticle platform that Intellia developed for in vivo therapies that target multiple diseases including ATTR.
“Starting our global NTLA-2001 Phase 1 trial for ATTR patients is a major milestone in Intellia’s mission to develop medicines to cure severe and life-threatening diseases,” stated Intellia’s CEO, Dr. John Leonard.
Intellia is excited about the possibilities that may come from the study and related developments
The CEO also noted that the phase 1 clinical trial represents the first opportunity for Intellia to show that its unique therapeutic approach can deliver permanent effects that can potentially reverse ATTR. He also pointed out that the goal of the study will be to establish the optimal dose, as well as the safety, tolerability, and efficacy profile of the pipeline therapy. The company also plans to expedite the process so that it can get to pivotal studies in which it will evaluate cardiomyopathy and polyneuropathy patients.
Intellia plans to start dosing the first patient in the clinical trial before the end of the year. It also plans to conduct the NTLA-2001 study as a multi-center and open-label study that will be divided into two parts. It will focus on patients that have been diagnosed with hereditary ATTR. The biopharma plans to sign up to 38 patients in the phase 1 trial. The study is an important milestone for the company and also for patients with rare diseases such as ATTR who do not have therapeutic options.