Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as DX-88
ecallantide · 4 trials · 1 indication
Mean Symptom Complex Severity (MSCS) score is a validated point-in-time measure of symptom severity. At baseline and 4 hrs, patients rated the severity on a categorical scale (0=normal, 1=mild, 2=moderate, 3=severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.
The Mean Symptom Complex Severity (MSCS) score is a validated, comprehensive point-in-time measure of symptom severity. At baseline and 4 hours, patients rated the severity on a categorical scale (0 = normal, 1 = mild, 2 = moderate, 3 = severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.
Treatment Outcome Score (TOS) is a validated, comprehensive measure of symptom response to treatment. At 4 hours , patient assessment of response characterized by their change from baseline in symptom severity and collected by anatomic site of attack involvement, was recorded on a categorical scale (significant improvement \[100\] to significant worsening \[-100\]). The response at each anatomic site was weighted by baseline severity and then the weighted scores across all involved sites were averaged to calculate the TOS. Clinically meaningful improvement was indicated by a TOS of 30 or higher.
successful outcome is defined as attack resolution begun within 4 hrs after treatment and maintained for 24 hours
partial response is defined as an initial response to dosing followed by a relapse within 4 to 24 hours
| Arm | Type | Description |
|---|---|---|
| DX-88 (ecallantide) | EXPERIMENTAL | DX-88 (ecallantide) Patients were treated with DX-88 (ecallantide) when they experienced an HAE attack. 30 mg dose of ecallantide given via 3 SC injections; a second 30 mg dose can be administered if needed. Patients were to be assessed until 4 hrs post-dose. Patients were asked to return for 3 follow-up visits: 7 days, 28 days and 90 days post-dose. |
| Placebo | PLACEBO_COMPARATOR | Placebo, Phosphate Buffer Saline (PBS), pH 7.0 given as 3 subcutaneous injections. |
| 5mg/m2 DX-88 IV | EXPERIMENTAL | 5mg/m2 DX-88 (ecallantide)administered intravenously |
| 10mg/m2 DX-88 IV | EXPERIMENTAL | 10mg/m2 DX-88(ecallantide)administered intravenously |
| 20mg/m2 DX-88 IV | EXPERIMENTAL | 20mg/m2 DX-88 (ecallantide) administered intravenously |
| 30 mg DX-88 SC | EXPERIMENTAL | 30mg DX-88(ecallantide)administered subcutaneously |
| Name | Type | Description |
|---|---|---|
| ecallantide | DRUG | solution for SC injection, one 30 mg dose per HAE attack |
| Phosphate Buffer Saline (PBS), pH 7.0 | DRUG | given as three 1mL subcutaneous injections. |
| Phosphate Buffer Saline (PBS), | DRUG | given as three 1mL subcutaneous injections. |
| DX-88 (ecallantide) | DRUG | solution for injection 10 mg/mL |
Inclusion Criteria: * 10 years of age or older * Documented diagnosis of HAE (Type I or II) * Willing and able to give informed consent * Acute HAE attack at time of presentation Exclusion Criteria: * Receipt of an investigational drug or device, within 30 days prior to study treatment, other tha...
| 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 |
| Pharvaris N.V. | PHVS | 1 | PHASE2 | Deucrictibant |
| BioMarin Pharmaceutical Inc. | BMRN | 1 | PHASE1 | BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |
Ecallantide is used to treat acute attacks of hereditary angioedema (HAE), a rare inherited condition marked by sudden swelling episodes. It is a small molecule developed by Takeda Pharmaceutical Company Limited (ticker TAK) and has been studied in Phase 3 clinical trials in patients with HAE.
Ecallantide targets KLKB1, also known as plasma kallikrein. It acts as an inhibitor of this enzyme. By inhibiting KLKB1, ecallantide is designed to reduce the generation of bradykinin, the peptide mediator that drives the swelling and pain characteristic of hereditary angioedema attacks.
Ecallantide is developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is the company associated with the clinical development program for ecallantide in hereditary angioedema.
Ecallantide is in Phase 3 clinical development for hereditary angioedema. Three Phase 3 trials have been completed, and no trials are currently listed as active. The program has enrolled 334 participants in total across randomized, double-blind, placebo-controlled studies.
Ecallantide has been evaluated in three completed Phase 3 trials: NCT00457015, NCT00456508, and NCT00262080. These studies examined ecallantide for acute attacks of hereditary angioedema. Enrollment was 96, 147, and 91 participants respectively, and the trials were conducted in the United States, Canada, and Jordan.
Yes. Ecallantide was studied under the name DX-88, which appears in the titles of its clinical trials. Searches for DX-88 refer to the same small molecule developed by Takeda Pharmaceutical Company Limited for hereditary angioedema.