2023-11-14 17:19:58 ET
Summary
- Intellia Therapeutics, Inc. enrollment completion for the phase 2 study using NTLA-2002 for the treatment of hereditary angioedema is expected in Q4 2023; Results expected in 2024.
- Pending positive feedback from regulators, potential to initiate a pivotal phase 3 study using NTLA-2002 for hereditary angioedema in Q3 of 2024.
- It is expected that the global hereditary angioedema market size is projected to reach $6.53 billion by 2025.
- FDA clearance received to initiate a phase 3 trial of NTLA-2001 for the treatment of transthyretin amyloidosis with cardiomyopathy by the end of 2023.
Intellia Therapeutics, Inc. ( NTLA ) has made good progress in being able to advance its pipeline. It has an extensive pipeline to look into, but one program to consider would be with the use of its in vivo CRISPR product candidate NTLA-2002, which is being advanced in a phase 2 study for the treatment of patients with hereditary angioedema [HAE]. It is on track to complete enrollment of this particular mid-stage study in Q4 of 2023 and it is highly likely that results from this study are to be released at some point from it in 2024.
Not only that, but pending positive feedback from the FDA, it expects to initiate a phase 3 pivotal study for this program by Q3 of 2024 as well. Another catalyst to consider is that it was able to receive clearance from the FDA to initiate the phase 3 MAGNITUDE pivotal trial, which is going to use another in vivo CRISPR product candidate NTLA-2001 for the treatment of patients with transthyretin [ATTR] amyloidosis with cardiomyopathy by the end of 2023. It has an extensive pipeline and it hopes to advance other in vivo CRISPR drugs like NTLA-3001 for the treatment of patients with alpha-1 antitrypsin deficiency [AATD] lung disease.
NTLA-2002 For The Treatment Of Patients With Hereditary Angioedema
The good thing about Intellia Therapeutics is that it is on track to complete enrollment for its phase 2 study . This would be a mid-stage trial, which is using NTLA-2002 for the treatment of patients with hereditary angioedema [HAE]. Matter of fact, it expects to complete enrollment of this study at some point during Q4 of 2023. Why I believe it was important to go over this company is because with enrollment nearing completion, it is highly likely that it will be able to release results from this phase 2 study in 2024. This sets up a major catalyst opportunity for investors to look forward to in the coming year.
It is expected that the global hereditary angioedema market size is projected to reach $6.53 billion by 2025 . The phase 1/2 study is evaluating the safety and efficacy of NTLA-2002 for the treatment of adults with type I and Type II hereditary angioedema [HAE]. This clinical trial was split into two different portions of study in order to see if this in vivo CRISPR candidate is enough to act as a one-time treatment option for these patients. The trial was split into the following:
- Phase 1 dose escalation portion of the study
- Phase 2 placebo-controlled portion of the study.
As I stated in the beginning above, Intellia is getting close to completing enrollment of the 2 portion of the study. It is expected that results from this mid-stage study are likely to be released in 2024 at some point. Not only that, but it is also expected that it might be able to initiate a global pivotal phase 3 study for this program as well. Should it receive positive regulatory feedback from the FDA to do so, then it believes that it could initiate such a late-stage study in HAE patients by at least Q3 of 2024. This program has been coming along well, especially since the release of interim data from the phase 1 dose-escalation portion of this study. The phase 1 dose escalation portion recruited a total of 10 adult patients with HAE, who were split into specific dose group cohorts as follows:
- 3 patients received a single dose of 25 mg NTLA-2002
- 4 patients received a single dose of 50 mg NTLA-2002
- 3 patients received a single dose of 75 mg NTLA-2002.
Such data, with a cut-off date of September 28th 2022, showed that all patients given a single dose of NTLA-2002 were able to achieve robust reductions of plasma kallikrein level and HAE attack rates as well. Not only that, but all patients in the 25 mg and 75 mg cohorts had an attack-free interval through that latest follow-up . What this in vivo CRISPR drug showed is that a single treatment was able to have an extended attack-free interval of 5.5 month to 10.6 months.
Financials
According to the 10-Q SEC Filing , Intellia Therapeutics had cash, cash equivalents and marketable securities of $992.5 million as of September 30th 2023. The company spent about $343.2 million during the quarter to fund itself. The reason for the lower amount of cash spend during its most recently reported quarter was because of an offset in that it obtained $37.4 million of income through interest and about $12.6 million of reimbursement from its collaborators. Lastly, it was able to obtain about $16.2 million of net equity proceeds with the use of the "At-The-Market" offering program, along with $7.7 million in proceeds from employee-based stock plans.
The company is well capitalized for now and there is no risk of near-term dilution. Why do I state that? That's because in its 10-Q SEC Filing it states that it has enough cash to fund its operations beyond the next 24-month period. With this type of cash runway, I don't believe there is a major risk of near-term dilution.
Risks To Business
There are several risks that investors should be aware of before investing in Intellia Therapeutics. The first risk to consider would be with respect to the completion of enrollment of the phase 2 portion of the study using NTLA-2002 for the treatment of patients with Hereditary Angioedema. That's because it is highly likely that results from this mid-stage study are going to be released in 2024. There is no assurance that the plasma kallikrein or HAE attack reductions observed in the prior phase 1 portion with NTLA-2002 will be seen again in the randomized phase 2 portion of this study.
A second risk to then consider would be with respect to the phase 3 MAGNITUDE study, which is going to use NTLA-2001 for the treatment of patients with ATTR amyloidosis with cardiomyopathy [ATTR-CM]. This trial is expected to begin by end of 2023 and then the first patient is expected to be dosed in early 2024.
If and once trial results from this late-stage study are released, there is no guarantee that the composite endpoint will be met. Especially, since such an endpoint has two items that must be met with statistical significance. One part of the composite endpoint is looking at cardiovascular related mortality and then the other part of this endpoint is looking at cardiovascular related events.
The third and final risk to consider would be with respect to the advancement of NTLA-2001 for the treatment of patients with hereditary ATTR Amyloidosis with polyneuropathy [ATTRv-PN]. That's because Intellia is in discussions with regulatory authorities to possibly initiate a phase 3 study for this indication as well. There is no assurance that one or more regulatory of these regulatory authorities will give it the green light so that it can initiate a late-stage study for this specific program.
Conclusion
Intellia Therapeutics has made great progress in being able to advance the use of several of its in vivo CRISPR candidates for the treatment of patients with several indications. I noted that it is gearing up to complete enrollment of its phase 2 study using NTLA-2002 for the treatment of patients with Hereditary Angioedema. This is going to be an important event, because then that sets up the company to be able to release results from this mid-stage study in 2024. Not only that, but pending positive discussions with regulators, it could also initiate a phase 3 study for this program by Q3 of 2024 as well. This is going to be a good indication for it to go after.
It is expected that the global hereditary angioedema market size is projected to reach $6.53 billion by 2025. One good thing about NTLA-2002 is that it is going to go beyond just being the same as existing treatments out there. In essence, the goal is to get this drug to be a one-time treatment option for these patients. Along with the possibility of preventing any breakthrough attacks from sneaking by. It is in the process of initiating a phase 3 study using NTLA-2001 for ATTR Amyloidosis with cardiomyopathy before the end of this year and then will look to initiate another phase 3 program as well. This other phase 3 is going to be with respect to the advancement of NTLA-2001 for the treatment of patients with hereditary Amyloidosis with polyneuropathy.
For further details see:
Intellia: Soon-To-Be Enrollment Completion Brings About Catalysts In 2024