The US Food and Drug Administration (FDA) has granted clearance for Intellia Therapeutics’ investigational new drug (IND) application for in vivo CRISPR-based investigational therapy, NTLA-2002, enabling the company to initiate a trial to treat hereditary angioedema (HAE).
This clearance will allow the company to include the US in the international Phase II portion of its ongoing Phase I/II trial.
The multi-national Phase I/II trial is assessing the pharmacokinetics, tolerability, pharmacodynamics, and safety of NTLA-2002 in Type I or Type II HAE adult patients.
It also includes plasma kallikrein protein level measurements, as well as clinical activity as determined by HAE attack rate measures.
The trial’s Phase I section is a single ascending dose, and open-label design for identifying two NTLA-2002 dose levels that will be further assessed in the placebo-controlled, randomised Phase II part of the trial.
Last year, the company reported positive interim data from the Phase I trial, which showed significant, dose-dependent reductions in plasma kallikrein, as well as reductions in HAE attacks.
Intellia president and CEO John Leonard said: “The FDA’s acceptance of our IND application to initiate clinical evaluation of NTLA-2002 brings us one step closer to introducing a potentially paradigm-shifting treatment for people living with hereditary angioedema.
“The NTLA-2002 IND clearance marks an important milestone for Intellia as we continue our track record of execution as the leader in the genome editing field.
“We look forward to presenting additional data from the first-in-human, Phase 1 portion of the study later this year.”
NTLA-2002 has been designed for preventing the potentially life-threatening swelling attacks in HAE patients.
Recently, the trial’s Phase II stage commenced patient screening outside of the US.
