US-based biotechnology company Intellia Therapeutics has begun the Phase III HAELO clinical trial of NTLA-2002, an investigational in-vivo gene editing therapy for treating hereditary angioedema (HAE).
The multinational, placebo-controlled study will assess the therapy’s efficacy and safety in 60 adult subjects diagnosed with Type I or Type II HAE.
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Participants will be randomised in a 2:1 ratio to receive either a single 50mg infusion of NTLA-2002 or a placebo.
Those in the placebo group will have the option to be transferred to the NTLA-2002 treatment after 28 weeks.
Variation in the frequency of HAE attacks between weeks five and 28 will be the trial’s primary endpoint.
Intellia Therapeutics began screening subjects for the HAELO study following an end-of-Phase II discussion with the US Food and Drug Administration (FDA) and the submission of an amended investigational new drug application.
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By GlobalDataThe company previously reported positive safety and efficacy data from the Phase I/II study of NTLA-2002.
Interim results from the Phase I trial indicated substantial reductions in HAE attack rates, as well as consistent, significant and sustained decreases in kallikrein levels.
Intellia Therapeutics president and CEO John Leonard said: “Initiation of the HAELO Phase III trial is a significant milestone for Intellia as we enter the final stage of clinical development for NTLA-2002 for people living with hereditary angioedema.
“Data from the ongoing Phase I/II study showed great promise that a single-dose treatment can lead to a complete response – no more attacks and no further treatment required.
“We are working urgently to bring forward NTLA-2002 to address the real world needs of people suffering from this disease and, ultimately, believe it will bring significant value to patients, physicians and payers.”
Based in Massachusetts, Intellia Therapeutics develops gene therapy programmes designed to enable the precise editing of disease-causing genes directly inside the human body.
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