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ALN-PNP · 3 trials · 5 indications
Part A
| Arm | Type | Description |
|---|---|---|
| Part A: A1 | EXPERIMENTAL | - |
| Part A: A2 | EXPERIMENTAL | - |
| Part B: B1 | EXPERIMENTAL | - |
| Part B: B2 | EXPERIMENTAL | - |
| Part B: B3 | EXPERIMENTAL | - |
| Part B: B4 | PLACEBO_COMPARATOR | - |
| Part A: Placebo | PLACEBO_COMPARATOR | Participants who are homozygous for the PNPLA3 rs738409:G risk allele will be randomized 1:1:1:1 |
| Part A: Low Dose | EXPERIMENTAL | Participants who are homozygous for the PNPLA3 rs738409:G risk allele will be randomized 1:1:1:1 |
| Part A: Mid Dose | EXPERIMENTAL | Participants who are homozygous for the PNPLA3 rs738409:G risk allele will be randomized 1:1:1:1 |
| Part A: High Dose | EXPERIMENTAL | Participants who are homozygous for the PNPLA3 rs738409:G risk allele will be randomized 1:1:1:1 |
| Part B: Placebo | PLACEBO_COMPARATOR | Participants who are homozygous for the PNPLA3 rs738409:G risk allele will be randomized 1:1:1:1 |
| Part B : Low Dose | EXPERIMENTAL | Participants who are homozygous for the PNPLA3 rs738409:G risk allele will be randomized 1:1:1:1 |
| Part B: Mid Dose | EXPERIMENTAL | Participants who are homozygous for the PNPLA3 rs738409:G risk allele will be randomized 1:1:1:1 |
| Part B: High Dose | EXPERIMENTAL | Participants who are homozygous for the PNPLA3 rs738409:G risk allele will be randomized 1:1:1:1 |
| Part C: Placebo (Optional) | PLACEBO_COMPARATOR | Sponsor may elect to enroll participants who are heterozygous for the PNPLA3 rs738409:G risk allele and may be randomized 1:1 |
| Part C: High Dose (Optional) | EXPERIMENTAL | Sponsor may elect to enroll participants who are heterozygous for the PNPLA3 rs738409:G risk allele and may be randomized 1:1 |
| Part A: Cohorts | EXPERIMENTAL | - |
| Part A: Optional Cohort | EXPERIMENTAL | - |
| Part A: JPN Cohorts | EXPERIMENTAL | - |
| Part B: Cohorts | EXPERIMENTAL | - |
| Part C: Cohorts | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| ALN-PNP | DRUG | Administered per the protocol |
| Tirzepatide | DRUG | Administered per the protocol |
| Placebo | DRUG | Administered per the protocol Placebo matching ALN-PNP |
| Placebo (PB) | DRUG | Administered per the protocol |
Key Inclusion Criteria: Part A and Part B: 1. Homozygous for the PNPLA3 p.I148M genotype 2. Liver fat by Magnetic Resonance Imaging-Proton Density Fat Fraction (MRI-PDFF) ≥15% at visit 3 3. Has a Body Mass Index (BMI) ≥30 to \<45 kg/m\^2 at visit 2 Part A: To be eligible for randomization on stud...
ALN-PNP is an investigational small molecule being developed for Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD) and Nonalcoholic Fatty Liver Disease (NAFLD). It is also being studied in patients with a genetic risk factor for these liver conditions. It has not been approved for any indication and remains in clinical development.
ALN-PNP is being developed by Regeneron Pharmaceuticals, Inc., which trades on Nasdaq under the ticker REGN. Regeneron is the sponsor of the clinical program evaluating ALN-PNP in MASLD and NAFLD, including studies that combine the drug with a GLP1R agonist.
ALN-PNP is in Phase 2 clinical development. The Phase 2a study, NCT07527910, is recruiting adults with homozygous PNPLA3-related MASLD and is testing ALN-PNP with and without a GLP1R agonist. Earlier Phase 1 studies of ALN-PNP have also been conducted.
ALN-PNP has been studied in three registered trials. NCT07527910 is a recruiting Phase 2a study in homozygous PNPLA3-related MASLD. NCT05648214 is a recruiting Phase 1 study in healthy adults and adults with MASLD. NCT06024408 was a completed Phase 1 study in NAFLD and NASH patients with a genetic risk factor.
Across the ALN-PNP clinical program, total enrollment is 376 participants. The Phase 2a study NCT07527910 plans to enroll 204 patients, the Phase 1 study NCT05648214 plans to enroll 172 participants, and the completed Phase 1 study NCT06024408 enrolled 9 participants. The trials are randomized, double-blind, and controlled.
Yes. The Phase 2a trial NCT07527910 is evaluating ALN-PNP both with and without a GLP1R agonist in adult patients with homozygous PNPLA3-related MASLD. This design allows researchers to assess ALN-PNP as a monotherapy and in combination with a GLP1 receptor agonist.