Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Deucrictibant · 6 trials · 18 indications
Time-normalized number of Investigator-confirmed AAE attacks during Treatment Phase
Time to symptom relief, Patient Global Impression of Change (PGI-C) rating of at least "better"
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation
Hematology, blood chemistry, and urinalysis. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Digital triplicate 12-lead ECG. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Descriptive in nature, no formal statistical hypothesis testing will be performed.
Systolic and diastolic blood pressure will be measured. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Descriptive in nature, no formal statistical hypothesis testing will be performed.
hematology, blood chemistry, urinalysis
The primary endpoint of the study was the change of the VAS-3 (3-symptom composite visual analogue scale) score from pre-treatment to 4 hours post-treatment. The VAS-3 was calculated as the mean of the VAS scores of the 3 major HAE symptoms: skin swelling, skin pain, and abdominal pain. The VAS scores of the 3 major HAE symptoms (skin swelling, skin pain, and abdominal pain) could range between 0 (No swelling/No pain) and 100 (Extreme swelling/Excruciating pain)
| Arm | Type | Description |
|---|---|---|
| Part 1 - Arm 1 - Active | EXPERIMENTAL | - |
| Part 1 - Arm 2 - Placebo | PLACEBO_COMPARATOR | - |
| Part 2 - Arm 1 | EXPERIMENTAL | - |
| Part 2 - Arm 2 | EXPERIMENTAL | - |
| Part 3 - Open-label | EXPERIMENTAL | - |
| Deucrictibant | EXPERIMENTAL | Deucrictibant |
| Active | EXPERIMENTAL | Deucrictibant 40mg extended-release tablet by mouth once daily |
| Placebo | EXPERIMENTAL | Placebo 1 tablet by mouth once daily |
| Part A: Deucrictibant, blinded dose | EXPERIMENTAL | Participants will receive the dose of deucrictibant they were randomized to in the PHA022121-C201 study (low, medium, or high dose, each consisting of 3 capsules of deucrictibant or matching placebo) for oral use for on-demand treatment of HAE attacks. |
| Part B: Deucrictibant, open-label | EXPERIMENTAL | Participants will receive deucrictibant soft capsules for oral use for on-demand treatment of HAE attacks. |
| Part 1: Low dose | EXPERIMENTAL | BID low dose of deucrictibant |
| Part 1: High dose | EXPERIMENTAL | BID high dose of deucrictibant |
| Part 1: Placebo | PLACEBO_COMPARATOR | BID placebo |
| Part 2: Open-label | EXPERIMENTAL | BID high dose of deucrictibant |
| Low dose/placebo | OTHER | Single low dose of deucrictibant or placebo |
| Medium dose/placebo | OTHER | Single medium dose of deucrictibant or placebo |
| High dose/placebo | OTHER | Single high dose of deucrictibant or placebo |
| Name | Type | Description |
|---|---|---|
| Deucrictibant | DRUG | Part 1: Deucrictibant 40 mg extended-release tablet for once daily oral use |
| Placebo | DRUG | Part 1: Placebo Comparator tablet for once daily oral use |
| Deucrictibant low dose | DRUG | Deucrictibant softgel capsules for oral use (PHVS416) |
| Deucrictibant high dose | DRUG | Deucrictibant softgel capsules for oral use (PHVS416) |
Inclusion Criteria: * Provision of written informed consent * Male or female (sex at birth) aged ≥18 years * Diagnosis of AAE-C1INH * History of AAE-C1INH attacks prior to the Screening Visit: * Participants enrolling in Part 1 must have stable underlying disease of AAE-C1INH * The underlying co...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Intellia Therapeutics, Inc. | NTLA | 3 | PHASE3 | NTLA-2002, Normal Saline Administration |
| BioCryst Pharmaceuticals, Inc. | BCRX | 2 | PHASE3 | Berotralstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | Donidalorsen |
| Pharvaris N.V. | PHVS | 1 | PHASE2 | deucrictibant |
| BioMarin Pharmaceutical Inc. | BMRN | 1 | PHASE1 | Dose 1 of BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |