Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ACER-002 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| ACER-002 (celiprolol) 200 mg BID | EXPERIMENTAL | ACER-002 200 mg twice daily (BID) (after titration): 200 mg morning and 200 mg evening: 400 mg total daily dose Titration: Day 1 to Month 1 - 100 mg once daily (QD) evening: 100 mg total daily dose Month 2 to Month 3 - 100 mg morning and 100 mg evening: 200 mg total daily dose Month 3 to Month 4 - 100 mg morning and 200 mg evening: 300 mg total daily dose Month 4 to End of Treatment Period (BID) - 200 mg morning and 200 mg evening: 400 mg total daily dose |
| Placebo BID | EXPERIMENTAL | Placebo twice daily (BID) Placebo given orally to mimic ACER-002 (celiprolol) administration |
| Name | Type | Description |
|---|---|---|
| ACER-002 (celiprolol) 200 mg BID | DRUG | ACER-002 (celiprolol) 200 mg BID |
| Placebo BID | DRUG | placebo for ACER-002 |
Inclusion Criteria: 1. Willingness to obtain magnetic resonance angiogram (MRA) image at local imaging facility. 2. A genetic test confirming the presence of a pathogenic COL3A1 variant (classified as likely pathogenic or pathogenic according to ACMG/AMP Guidelines. 3. Patients must be ≥ 15 years o...
ACER-002 is an investigational small molecule being developed for the treatment of Vascular Ehlers-Danlos Syndrome, a rare genetic condition affecting connective tissue. It is currently in Phase 3 clinical development and has not been approved by the FDA.
ACER-002 is a small molecule, but its specific molecular target has not been disclosed. It is being studied for its potential to treat Vascular Ehlers-Danlos Syndrome, a rare disease, though the exact mechanism of action is not publicly detailed.
ACER-002 is being developed by Zevra Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol ZVRA. The company is conducting a Phase 3 clinical trial to evaluate the drug's efficacy in patients with Vascular Ehlers-Danlos Syndrome.
ACER-002 is currently in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. The ongoing Phase 3 trial is recruiting participants in the United States to assess the drug's efficacy compared to placebo.
ACER-002 is being studied in a Phase 3 clinical trial registered as NCT05432466. This randomized, double-blind, placebo-controlled trial is recruiting 150 patients with Vascular Ehlers-Danlos Syndrome in the United States. The study is currently active and enrolling participants aged 15 years and older.
ACER-002 is also known as celiprolol, specifically at a dose of 200 mg taken twice daily. In clinical trials, ACER-002 refers to this formulation of celiprolol being investigated for the treatment of Vascular Ehlers-Danlos Syndrome.