Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as ADX-324 Dose Level 1, siRNA
ADX-324 · 3 trials · 5 indications
Incidence and severity of treatment-emergent adverse events
The time-normalized number of Investigator-confirmed HAE attacks per month for ADX-324 Dose Level 1 vs placebo
To evaluate the safety and tolerability of ADX-324 in HVs by incidence, relationship, and severity of adverse events and serious adverse events
To evaluate the safety and tolerability of ADX-324 in HAE by incidence, relationship, and severity of adverse events and serious adverse events
| Arm | Type | Description |
|---|---|---|
| ADX-324 Dose Level 1 | EXPERIMENTAL | - |
| ADX-324 Dose Level 2 | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | - |
| PART A - Active ADX-324 administered to HV | EXPERIMENTAL | For each cohort in Part A (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-324): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule. |
| PART A- Placebo administered to HV | PLACEBO_COMPARATOR | For each cohort in Part A (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-324): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule. |
| PART B - ADX-324 administered to HAE participants | EXPERIMENTAL | This will be initiated at the dose level determined by the Safety Review Committee from SAD in HVs. The treatment of HAE participants is an open-label study. |
| Name | Type | Description |
|---|---|---|
| ADX-324 Dose Level 1 | DRUG | siRNA duplex oligonucleotide |
| ADX-324 Dose Level 2 | DRUG | siRNA duplex oligonucleotide |
| ADX-324 | DRUG | siRNA duplex oligonucleotide |
| Placebo | DRUG | saline |
Inclusion Criteria: * Have a documented diagnosis of HAE (Type I or II) * Completed Study ADX-324-301 * Have access to an acute therapy to treat HAE attacks (such as plasma derived or recombinant C1-INH concentrate or a BK2-receptor antagonist) Exclusion Criteria: * A negative reaction to study d...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Intellia Therapeutics, Inc. | NTLA | 3 | PHASE3 | NTLA-2002 |
| BioCryst Pharmaceuticals, Inc. | BCRX | 2 | PHASE3 | Berotralstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | Donidalorsen |
| ADARx Pharmaceuticals, Inc. | ADRX | 3 | PHASE3 | ADX-324 |
| Pharvaris N.V. | PHVS | 1 | PHASE2 | Deucrictibant |
| BioMarin Pharmaceutical Inc. | BMRN | 1 | PHASE1 | BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |