Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Encaleret · 3 trials · 2 indications
* cCa within 8.3-10.7 mg/dL (2.08-2.68 millimoles per liter \[mmol/L\]) * 24-hr UCa within the reference range (\< 300 mg/day for men \[7.5 mmol/day\], \< 250 mg/day for women \[6.25 mmol/day\])
Adverse events (AEs) were defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related. Treatment-emergence was defined as any AE(s) regardless of relationship to investigational medicinal product (IMP), that had an onset or worsened in severity on or after the first dose of IMP.
| Arm | Type | Description |
|---|---|---|
| Encaleret | EXPERIMENTAL | Participants will receive encaleret at a dose as needed based on calcium levels. |
| Standard of Care (SoC) | OTHER | Participants will continue receiving calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.) |
| Cohort 1: Ascending + Steady-State Dose | EXPERIMENTAL | Period 1: Participants will receive an ascending dose of encaleret once daily for the first 3 days. Participants will then receive an individualized dose of encaleret twice daily for 2 days. Period 2: Participants will receive encaleret twice daily for 5 days at a single dose level based on responses from Period 1. Period 3: After completion of Period 2, participants will be eligible to receive encaleret for an additional 24 weeks. Long-Term Extension (LTE): At the end of the study, participants will also have an option to receive encaleret for up to an additional 2 years. |
| Cohort 2: Steady-State Dose | EXPERIMENTAL | Participants will directly be enrolled into Period 2, and receive encaleret twice daily at a dose based on data and responses from Cohort 1 Period 1. Period 2: Participants will receive encaleret twice daily for 5 days. Period 3: After completion of Period 2, participants will be eligible to receive encaleret for an additional 24 weeks. LTE: At the end of the study, participants will also have an option to receive encaleret for up to an additional 2 years. |
| Name | Type | Description |
|---|---|---|
| Encaleret | DRUG | Administered as film-coated tablet for oral use |
| Standard of Care | DIETARY_SUPPLEMENT | Calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.) |
Key Inclusion Criteria: 1. Participants must have a documented pathogenic or likely pathogenic activating variant, or variant of uncertain significance, of the calcium sensing receptor (CASR) gene associated with biochemical findings of hypoparathyroidism. 2. Participants must have a documented his...
Encaleret is an investigational small molecule being developed for Autosomal Dominant Hypocalcemia (ADH) and Autosomal Dominant Hypocalcemia Type 1 (ADH1), rare genetic disorders characterized by low blood calcium levels. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.
Encaleret is being developed by BridgeBio Pharma, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol BBIO. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Autosomal Dominant Hypocalcemia.
Encaleret is in Phase 3 clinical development for Autosomal Dominant Hypocalcemia Type 1 (ADH1). It is an investigational drug, meaning it has not yet been approved by the FDA or other regulatory agencies. A Phase 3 trial comparing it to standard of care is currently active but not recruiting participants.
Encaleret has been studied in several clinical trials. A completed Phase 2 trial (NCT04581629) evaluated its safety and efficacy in 13 participants with ADH1. An active Phase 3 trial (NCT05680818) is comparing it to standard of care in 67 participants. A recruiting Phase 2 trial (NCT07080385) is studying it in pediatric patients.
Encaleret is a small molecule that targets the calcium-sensing receptor (CaSR), which plays a key role in regulating blood calcium levels. By modulating this receptor, Encaleret aims to correct the underlying calcium imbalance in patients with Autosomal Dominant Hypocalcemia Type 1.
Encaleret is a distinct investigational compound developed by BridgeBio Pharma. It is not known to be the same as any other marketed drug. Its unique mechanism of action targeting the calcium-sensing receptor differentiates it from standard treatments for hypocalcemia.