Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Patidegib · 2 trials · 2 indications
All serious adverse events (SAEs) and all other non-serious adverse events (AEs) regardless of causality are located in the Reported AE Module. The number of AEs (including both serious and non-serious) considered related to study drug by the Investigator are presented below. Treatment-emergent AEs are those with an onset after use of study drug.
GLI1 change is a biomarker of the HH signaling pathway. A change in GLI1 mRNA levels reflect a change in the HH pathway. Surgically eligible basal cell carcinomas (SEBs) were defined as clinically diagnosed basal cell carcinoma (BCC) 5 to 20 millimeters (mm) in diameter on the face, excluding the nose and periorbital skin, and 9 to 20 mm at sites other than the face. A single baseline BCC designated as a treatment-targeted tumor at Baseline was biopsied first at Baseline and again following 12 weeks of treatment. This was used to assess percent change in GLI1 mRNA levels as follows: (Baseline - Week 12) / Baseline \* 100, with positive numbers to represent increases and negative numbers to represent decreases. Any missing values were not imputed; all available data is summarized.
SEBs were defined as clinically diagnosed basal cell carcinoma (BCC) 5 millimeters (mm) or greater in diameter on the face, excluding the nose and periorbital skin, and 9 mm or greater at sites other than the face. The percent change in greatest diameters of treatment-targeted surgically eligible basal cell carcinomas (SEBs) from Baseline to Week 26 was calculated as follows: (sum \[Baseline\] - sum \[Week 26\] / sum \[Baseline\] \* 100), where sum = the greatest diameters of Baseline treatment-targeted SEBs and positive numbers represent decrease in tumor size and negative numbers to represent increase in tumor size. Missing values were imputed using Last-Observation Carried Forward (LOCF).
SEBs were defined as clinically diagnosed BCC 5 mm or greater in diameter on the face, excluding the nose and periorbital skin, and 9-mm or greater at sites other than the face. A single baseline SEB designated as a treatment targeted tumor at Baseline was biopsied first at Baseline and again following 6 weeks of treatment. This was used to assess percent change in GLI1 mRNA levels as follows: (Baseline - Week 6) / Baseline \* 100, where positive numbers to represent decrease in GL1 mRNA level and negative numbers to represent increase in GL1 mRNA level. Any missing values were not imputed; all available data is summarized.
All serious adverse events (SAEs) and all other non-serious adverse events (AEs) regardless of causality are located in the Reported AE Module. AEs considered as related where categorized by the Investigator as either definitely related, probably related, or possibly related. Treatment-emergent AEs are those with an onset after use of study drug.
All SAEs and all other non-serious AEs, regardless of causality, are located in the Reported AE Module. The number of participants reporting administrative-site, skin condition treatment-emergent AEs considered related to study drug by the Investigator are presented below. Treatment-emergent AEs are those with an onset after use of study drug.
| Arm | Type | Description |
|---|---|---|
| Patidegib gel 2% - Cohort 1 | EXPERIMENTAL | Patidegib gel 2%, applied topically, once daily for 12 weeks (Cohort 1) |
| Patidegib gel 4% - Cohort 2 | EXPERIMENTAL | Patidegib gel 4%, applied topically, once daily for 12 weeks (Cohort 2) |
| Vehicle gel - Cohort 1 | PLACEBO_COMPARATOR | Vehicle gel, applied topically, once daily for 12 weeks (Cohort 1) |
| Patidegib gel 2% - Cohort 3 | EXPERIMENTAL | Patidegib gel 2%, applied topically, twice daily for 12 weeks (Cohort 3) |
| Patidegib gel 4% - Cohort 4 | EXPERIMENTAL | Patidegib gel 4%, applied topically, twice daily for 12 weeks (Cohort 4) |
| Vehicle gel - Cohort 2 | PLACEBO_COMPARATOR | Vehicle gel, applied topically, once daily for 12 weeks (Cohort 2) |
| Vehicle gel - Cohort 3 | PLACEBO_COMPARATOR | Vehicle gel, applied topically, twice daily for 12 weeks (Cohort 3) |
| Vehicle gel - Cohort 4 | PLACEBO_COMPARATOR | Vehicle gel, applied topically, twice daily for 12 weeks (Cohort 4) |
| Patidegib gel 2% | EXPERIMENTAL | Patidegib gel 2%, applied topically, twice daily for 26 weeks |
| Patidegib gel 4% | EXPERIMENTAL | Patidegib gel 4%, applied topically, twice daily for 26 weeks |
| Vehicle gel | PLACEBO_COMPARATOR | Vehicle gel, applied topically, twice daily for 26 weeks |
| Name | Type | Description |
|---|---|---|
| Patidegib | DRUG | - |
| Vehicle gel | DRUG | - |
Inclusion Criteria: 1. The participant is from 18 to 85 years of age, inclusive. 2. The participant must provide electronic informed consent prior to any study procedures. 3. If the participant is a woman of childbearing potential, she is willing to use two effective methods of birth control during...
Patidegib is an investigational small molecule being studied for the treatment of Basal Cell Nevus Syndrome (Gorlin Syndrome) and Basal Cell Carcinomas. It is a topical gel formulation evaluated in Phase 2 clinical trials to reduce the number of surgically eligible basal cell carcinomas in patients with Gorlin Syndrome and to decrease the GLI1 biomarker in sporadic nodular basal cell carcinomas.
Patidegib targets the hedgehog signaling pathway, specifically inhibiting the Smoothened (SMO) receptor. By blocking this pathway, Patidegib aims to reduce the growth of basal cell carcinomas. The drug's effect on the GLI1 biomarker, a downstream target of the hedgehog pathway, was measured in clinical trials to assess its activity.
Patidegib is being developed by BridgeBio Pharma, Inc., a biopharmaceutical company listed on NASDAQ under the ticker symbol BBIO. The company is conducting clinical trials to evaluate the safety and efficacy of Patidegib gel for the treatment of basal cell carcinoma-related conditions.
Patidegib is in Phase 2 clinical development. Two Phase 2 trials have been completed, one in the United Kingdom for Basal Cell Nevus Syndrome and one in the United States for sporadic nodular Basal Cell Carcinomas. Patidegib is investigational and has not been approved by regulatory authorities.
Patidegib has been studied in two completed Phase 2 clinical trials. NCT02762084 evaluated Patidegib gel 2% and 4% versus vehicle in 17 patients with Basal Cell Nevus Syndrome in the United Kingdom. NCT02828111 evaluated Patidegib gel 2% and 4% applied once or twice daily in 36 patients with sporadic nodular Basal Cell Carcinomas in the United States.
Patidegib is a hedgehog pathway inhibitor that targets Smoothened (SMO), similar to other drugs in this class. However, Patidegib is formulated as a topical gel for local application to basal cell carcinomas, which distinguishes it from systemically administered hedgehog inhibitors. No alternative names for Patidegib have been reported.