Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Recombinant human C1 inhibitor · 4 trials · 3 indications
The time to beginning of relief of symptoms has been assessed by using a patient-reported visual analogue scale ("VAS") ranging from 0 mm (no symptoms at all) to 100 mm (extremely disabling). Time to beginning of relief of symptoms at the location that showed first "VAS" score decrease of at least 20 mm from baseline score (t= 0 min) to the next assessment time-point). Assessment time-points were taken on pre-scheduled time-points after drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to beginning of relief has been calculated as median time, by using the exact time-points on which each assessment was performed.
Average number of HAE attacks normalized to a 28 day period
Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).
| Arm | Type | Description |
|---|---|---|
| 100 IU/kg "rhC1INH" | EXPERIMENTAL | 100 IU/kg recombinant human C1 inhibitor |
| Saline | PLACEBO_COMPARATOR | Saline solution |
| rhC1INH twice weekly | EXPERIMENTAL | rhC1INH administered twice weekly |
| rhC1INH once weekly | EXPERIMENTAL | rhC1INH administered once weekly |
| Placebo (Saline) twice weekly | PLACEBO_COMPARATOR | Placebo (Saline) administered twice weekly |
| Recombinant Human C1 Inhibitor | OTHER | Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor |
| 100 IU/kg rhC1INH | EXPERIMENTAL | 100 IU/kg Recombinant human C1 inhibitor |
| 50 IU/kg rhC1INH | EXPERIMENTAL | 50 IU/kg Recombinant human C1 inhibitor |
| Name | Type | Description |
|---|---|---|
| recombinant human C1 inhibitor | DRUG | IV |
| Placebo | DRUG | IV |
Inclusion Criteria: * Clear clinical and laboratory diagnosis of HAE * Baseline plasma level of functional C1INH of less than 50% of normal * Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack Exclusion Cri...
| 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 |