Precigen Triple-Gene Provides Six-month Follow-up Data from Phase I Study of INXN-4001, a Multigenic Investigational Therapeutic Candidate for Heart Failure
The primary objectives of the Phase I study are to evaluate the safety of INXN-4001 infusion into the myocardium of left ventricular assist device (LVAD) patients and to demonstrate safety and feasibility of RCSI. Secondary endpoints include improvements in 6-min walk test (6MWT) duration and distance and quality of life, as assessed by Kansas City Cardiomyopathy Questionnaire (KCCQ) responses.
Twelve chronic heart failure patients were treated with INXN-4001 in either Cohort 1 (80 mg/40 ml; n=6) or Cohort 2 (80 mg/80 mL; n=6) and 10 were evaluated for six-month follow-up. The study has met the primary endpoints to evaluate safety and feasibility for INXN-4001. The infusions of INXN-4001 were overall well tolerated, with no adverse events attributed to INXN-4001, whereas one serious adverse event was considered related to the infusion procedure.
Furthermore, preliminary assessment of the secondary endpoints show improvement by at least five points in total KCCQ score, a validated tool qualified by the FDA for Clinical Outcome Assessment, was observed in 50% of patients evaluable (5 out of 10) at the six-month follow-up visit. These included four patients treated in Cohort 1 and one patient treated in Cohort 2. Of the evaluable patients, 50% (4 out of 8) had an improvement in the 6MWT (> 50 meters in distance walked) at six months post-infusion compared to pre-treatment baseline.
"Overall the data support that INXN-4001 is well tolerated in patients with chronic heart failure on an LVAD," said
"Heart failure is a devastating disease with high unmet medical need. We are encouraged that INXN-4001 successfully met the primary safety and feasibility endpoints and that the preliminary assessment of the secondary endpoints show promising improvements in the quality of life six months after treatment for these patients," said
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