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TransCon PTH · 2 trials · 3 indications
The primary endpoint was a multi-component endpoint that included the percentage of participants who met the following criteria at 26 weeks of blinded treatment: 1) albumin-adjusted serum calcium measured within 4 weeks prior to and on Week 26 visit within the normal range (8.3 to 10.6 mg/dL), and 2) independence from active vitamin D within 4 weeks prior to Week 26 visit (i.e., all daily standing dose of active vitamin D equal to zero AND use of PRN ≤7 days during the 4 weeks), and 3) independence from therapeutic doses of calcium within 4 weeks prior to Week 26 visit (i.e., average daily standing dose of elemental calcium ≤600 mg AND use of PRN doses on ≤7 days during the 4 weeks), and 4) no increase in prescribed study drug within 4 weeks prior to Week 26 visit.
| Arm | Type | Description |
|---|---|---|
| TransCon PTH | EXPERIMENTAL | TransCon PTH at a starting dose of 18 mcg delivered once daily by subcutaneous injection and titrated to an optimal dose |
| Placebo | PLACEBO_COMPARATOR | Placebo for TransCon PTH delivered once daily by subcutaneous injection |
| Double Blind: TransCon PTH 15 mcg | EXPERIMENTAL | TransCon PTH 15 mcg delivered once daily by subcutaneous injection |
| Double Blind: TransCon PTH 18 mcg | EXPERIMENTAL | TransCon PTH 18 mcg delivered once daily by subcutaneous injection |
| Double Blind: TransCon PTH 21 mcg | EXPERIMENTAL | TransCon PTH 21 mcg delivered once daily by subcutaneous injection |
| Open-Label Extension Period: TransCon PTH | EXPERIMENTAL | Participants who completed the 4-week double-blind period, continued into the open-label extension period and received treatment with TransCon PTH up to Week 266, with up to an initial 14 weeks of TransCon PTH titration and standard of care optimization, followed by approximately 248 weeks of individualized doses of TransCon PTH (allowable dose range: 6 to 60 mcg/day). |
| Name | Type | Description |
|---|---|---|
| TransCon PTH | COMBINATION_PRODUCT | TransCon PTH drug product is supplied as a solution with a concentration of 0.3 mg PTH(1-34)/mL in a single-patient-use prefilled pen intended for subcutaneous injection. |
| Placebo | COMBINATION_PRODUCT | Placebo is supplied as a solution containing the formulation buffer for TransCon PTH in a single-patient-use prefilled pen intended for subcutaneous injection. |
| Placebo for TransCon PTH | COMBINATION_PRODUCT | Placebo is supplied as a clear solution containing the formulation buffer for TransCon PTH in a pre-filled pen intended for subcutaneous injection. |
Inclusion Criteria: 1. Males and females, ≥18 years of age 2. Subjects with postsurgical chronic HP, or auto-immune, genetic, or idiopathic HP for at least 26 weeks. Diagnosis of HP is established based on historic hypocalcemia in the setting of inappropriately low serum PTH levels 3. Requirement f...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| AstraZeneca PLC | AZN | 1 | PHASE3 | eneboparatide |
| Ascendis Pharma A/S | ASND | 3 | PHASE3 | Palopegteriparatide Experimental Arm, Palopegteriparatide Control Arm |
| MBX Biosciences, Inc. | MBX | 1 | PHASE2 | 400 µg of MBX 2109 once-weekly by, 200-1600 µg of MBX 2109 once-weekly by |
TransCon PTH is an investigational small molecule being developed for the treatment of hypoparathyroidism in adults. It is currently in Phase 3 clinical development and has not been approved by the FDA. The drug is designed to address the underlying hormone deficiency associated with this endocrine disorder.
TransCon PTH is being developed by Ascendis Pharma A/S, a biopharmaceutical company listed on the NASDAQ under the ticker symbol ASND. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy for hypoparathyroidism.
TransCon PTH is currently in Phase 3 clinical development for hypoparathyroidism. It has received Priority Review designation from the FDA, indicating the agency's commitment to expedite its review. The drug remains investigational and is not yet approved for commercial use.
TransCon PTH has completed two clinical trials. NCT04009291 was a Phase 2 study with 59 participants, and NCT04701203 was a Phase 3 study with 84 participants. Both trials were randomized, double-blind, and placebo-controlled, enrolling adults with hypoparathyroidism across multiple countries including the United States, Canada, and several European nations.
TransCon PTH is not FDA approved. It is an investigational drug currently in Phase 3 clinical development for hypoparathyroidism. The FDA has granted Priority Review designation, which may accelerate the review process, but the drug has not yet received marketing approval.