Jefferies analyst Maury Raycroft said in a note to investors that Thursday’s mid-stage readout pointed to the “unprecedented” complete response rate of Intellia’s investigational in vivo gene editing therapy in the disorder.
Intellia Therapeutics on Thursday unveiled Phase II data for its investigational CRISPR treatment NTLA-2002, touting a steep reduction in monthly attack rates in patients with hereditary angioedema, a disorder characterized by recurrent episodes of severe swelling.
The results, to be presented this weekend at the 2024 Scientific of the American College of Allergy, Asthma & Immunology in Boston, showed that a one-time, 25-mg dose of NTLA-2002 reduced monthly attacks by 80% from weeks five through 16 of follow-up. The higher 50-mg dose lowered attacks by 81% during this time period.
Expanding the observation period from weeks one through 16, the 25-mg and 50-mg doses of NTLA-2002 resulted in a 75% and 77% decrease in attack rate, respectively.
According to Intellia, eight of the 11 patients given a single 50-mg infusion reached complete response, experiencing “no attacks at all” during the 16-week observation period. Complete treatment response was durable in the eight patients through the latest follow-up at a median of eight months. Four of the 10 patients in the 25-mg group achieved this outcome, while none of the placebo comparators did so.
In terms of safety, both doses of NTLA-2002 were well-tolerated with the most common side effects being headaches, fatigue and nasopharyngitis. There were no serious toxicities, except for one patient in the placebo arm who developed grade 4 tongue edema that compromised breathing. There were also no clinically significant laboratory abnormalities.
Intellia CEO John Leonard in a statement said that the biotech is “highly encouraged” by these mid-stage findings, which “underscore the tremendous potential of our in vivo CRISPR gene editing therapy to be a functional cure” for hereditary angioedema (HAE) and “redefine the treatment paradigm” for this condition.
“What was previously an unimaginable potential to be free of chronic therapy is one step closer to becoming a reality for the HAE community,” Leonard said. Intellia will take NTLA-2002’s 50-mg dose into late-stage development. The Phase III HEALO study is currently actively screening patients, according to the company.
Analysts appear to be optimistic about NTLA-2002’s prospects—though cautiously so. Jefferies analyst Maury Raycroft in a note to investors said that Intellia’s readout on Thursday pointed to the “unprecedented complete response rate” of NTLA-2002 in HAE. The strong efficacy showing is further bolstered by NTLA-2002’s one-time dosing profile, which “is the key differentiator vs other … programs and points to potential for a functional cure for HAE,” Raycroft wrote.
Shareholders, however, seem to be underwhelmed. Intellia’s shares were down 9% in premarket trading on Thursday, following the Phase II data, according to Seeking Alpha.
Maury chalked the negative stock reaction up to an “overreaction” from investors, who had expected better monthly attack rate reductions based on promising Phase I data. Meanwhile, Truist Securities analyst Joon Lee pointed to competitive—and potentially better—HAE data from Pharvaris, which touted a 93% reduction in monthly attack rate for its oral daily pill.
William Blair analyst Myles Minter said that despite these pressures, “we still see potential for NTLA-2002 in HAE given the impressive magnitude of response when achieved.” Nevertheless, HAE is a “competitive space,” not only with several currently available treatment options, but also with upcoming therapies with strong potential, according to Minter.
