Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as CLTX-305
Encaleret · 4 trials · 4 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 during the 24-week double-blind treatment period. Participants will then have the option to enter an open-label long-term extension (LTE) period to continue receiving encaleret for up to 192 weeks. |
| Placebo | PLACEBO_COMPARATOR | Participants will receive placebo during the 24-week double-blind treatment period. Participants will then have the option to enter an open-label LTE period to receive encaleret for up to 192 weeks. |
| 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 | Oral tablets |
| Placebo | DRUG | Oral tablets |
| Standard of Care | DIETARY_SUPPLEMENT | Calcium supplements and/or active Vitamin D (calcitriol, alfacalcidol, falecalcitriol, etc.) |
Key Inclusion Criteria: * Participants must have had a clinically confirmed diagnosis of chronic HP, with disease duration of at least 26 weeks prior to screening. Cause of chronic HP can be postsurgical, autoimmune or idiopathic. * For participants with nonsurgical chronic HP etiology, sufficient ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Ascendis Pharma A/S | ASND | 4 | PHASE3 | Palopegteriparatide |
| AstraZeneca PLC | AZN | 1 | PHASE3 | eneboparatide |
| MBX Biosciences, Inc. | MBX | 2 | PHASE3 | Canvuparatide |
| BridgeBio Pharma, Inc. | BBIO | 1 | PHASE3 | Encaleret |
Encaleret is an investigational small molecule being developed for autosomal dominant hypocalcemia (ADH), including autosomal dominant hypocalcemia type 1 (ADH1), and for chronic hypoparathyroidism. It is a rare disease therapy that has not been approved by the FDA and remains in clinical development.
Encaleret targets the calcium-sensing receptor, or CaSR. By acting on this receptor, the drug is designed to address the underlying calcium regulation defect in conditions such as autosomal dominant hypocalcemia type 1 and hypoparathyroidism. Encaleret is a small molecule administered as an oral therapy.
Encaleret is being developed by BridgeBio Pharma, Inc., which trades on the Nasdaq under the ticker BBIO. BridgeBio holds the development program for encaleret across its rare disease indications, including autosomal dominant hypocalcemia type 1 and chronic hypoparathyroidism.
Encaleret is in late-stage development, with an NDA or BLA submission stage reached. It is investigational and has not been approved by the FDA. The program has received FDA Fast Track, Orphan Drug, and Priority Review designations. Clinical work includes Phase 2 and Phase 3 trials.
Encaleret trials include NCT05680818, a Phase 3 study comparing encaleret to standard of care in ADH1 that is active but not recruiting, and NCT07805356, a recruiting Phase 3 trial in chronic hypoparathyroidism. NCT07080385 is a recruiting Phase 2 pediatric ADH1 study, and NCT04581629 was a completed Phase 2 ADH1 trial.
Encaleret is not FDA approved. It is an investigational therapy still in clinical development, with the program at the NDA or BLA stage. The FDA has granted encaleret Fast Track, Orphan Drug, and Priority Review designations, which support the review process but do not constitute approval.