Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Study drug
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 topical therapy being studied for basal cell carcinomas and basal cell nevus syndrome, also known as Gorlin syndrome. It is applied as a gel and is intended to reduce tumor burden in these skin cancers. It has not been approved by the FDA and remains in clinical development.
Patidegib targets the SMO protein, a key component of the Hedgehog signaling pathway. It acts as an antagonist, blocking SMO activity to inhibit pathway signaling that drives basal cell carcinoma growth. This mechanism is relevant because aberrant Hedgehog signaling is a known driver in these tumors.
Patidegib is being developed by BridgeBio Pharma, Inc., which trades on the Nasdaq under the ticker BBIO. The company is advancing the drug as a potential treatment for basal cell carcinomas and basal cell nevus syndrome.
Patidegib is in Phase 2 clinical development. It has not been approved by the FDA for any indication. The completed Phase 2 trials evaluated the drug in patients with basal cell carcinomas and basal cell nevus syndrome.
Patidegib has been studied in two completed Phase 2 trials. NCT02828111 evaluated Patidegib gel 2%, 4%, and vehicle in 36 patients with sporadic nodular basal cell carcinomas in the United States. NCT02762084 tested the gel in 17 patients with basal cell nevus syndrome in the United Kingdom.
Patidegib is also known by its developmental code name IPI-926. It should not be confused with other Hedgehog pathway inhibitors, as it is a distinct small molecule developed by BridgeBio Pharma. The name Patidegib refers to the same investigational agent.