Hepion Pharmaceuticals Announces Positive Topline Data from Phase 2a ‘AMBITION’ NASH Trial; All Primary Endpoints Achieved
- Safety, Tolerability and Pharmacokinetics (“PK”) Primary Endpoints Met
- Reductions in Liver Transaminases Indicate Dose-Related Improvements in Key
- Concentration-Effect Relationship Demonstrated for ALT Reductions
- Multiomic Analyses and Machine Learning Paving the Way for Phase 2b, Anticipated to Start Later in 2021
AMBITION was a Phase 2a randomized, multi-center, placebo controlled, single-blind trial designed to investigate once daily oral administration of CRV431 at doses of 75 mg and 225 mg administered as soft gelatin capsules to presumed F2 and F3
The primary outcome measure of the AMBITION trial was the incidence of safety and tolerability events of CRV431 versus placebo. CRV431 at both study doses was well tolerated, and there were no serious adverse events (“SAEs”), and the few adverse events (“AEs”) observed were mostly mild and unrelated to study drug.
In the AMBITION trial, CRV431 blood concentrations after oral dosing of either 75 mg or 225 mg once daily were in the anticipated effective range for
It has been reported in recent literature that reductions in serum alanine aminotransferase (“ALT”) may be used as a surrogate measure for histologic improvement in
|CRV431 75 mg
|CRV431 225 mg
|ALT (% change)||-6.1±13.3
|1465.1 ± 810.9||1190.5 ± 712.1||859.9 ± 387.0**|
* Pooled 75 mg & 225 mg statistically significant from placebo, p < 0.05, unpaired t-test.
**Statistically significant from placebo, p < 0.05, ANOVA with Bonferroni Post-Hoc
“The observed changes in serum ALT at this early timepoint, along with the safety and tolerability data are very encouraging and suggest a potential positive impact of CRV431 on hepatocyte health relative to placebo,” commented
“Statistical significance in a dose response on ALT is very encouraging, suggesting a rapid drug effect. A thorough review of literature by our group suggested that a 10% to 15% decline in ALT in four weeks over placebo would indicate a beneficial drug effect,” stated
“In order to embark on a phase 2b program in
“CRV431 is a cyclophilin inhibitor that represents a new approach to treating NASH,” commented
1Hoofnagle, JH, et al. Aliment Pharmacol Ther. 2013; 38:134-143.
2Loomba, R. Clin Gastroenterol Hepatol. 2014; 12:1731-1732.
The Company's lead drug candidate, CRV431, is a potent inhibitor of cyclophilins, which are involved in many disease processes. CRV431 is currently in clinical-phase development for the treatment of
Hepion has created a proprietary AI platform, called AI-POWR™, which stands for Artificial Intelligence - Precision Medicine; Omics (including genomics, proteomics, metabolomics, transcriptomics, and lipidomics); World database access; and Response and clinical outcomes. Hepion intends to use AI-POWR™ to help identify which
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimated,” and “intend,” among others. These forward-looking statements are based on Hepion Pharmaceuticals’ current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, substantial competition; our ability to continue as a going concern; our need for additional financing; uncertainties of patent protection and litigation; risks associated with delays, increased costs and funding shortages caused by the COVID-19 pandemic; uncertainties with respect to lengthy and expensive clinical trials, that results of earlier studies and trials may not be predictive of future trial results; uncertainties of government or third party payer reimbursement; limited sales and marketing efforts and dependence upon third parties; and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. As with any drug candidates under development, there are significant risks in the development, regulatory approval, and commercialization of new products. There are no guarantees that future clinical trials discussed in this press release will be completed or successful, or that any product will receive regulatory approval for any indication or prove to be commercially successful.
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Source: Hepion Pharmaceuticals, Inc.