Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PTX-022 · 6 trials · 4 indications
| Arm | Type | Description |
|---|---|---|
| PTX-022 | EXPERIMENTAL | - |
| Open label treatment arm | EXPERIMENTAL | PTX-022 QTORIN |
| Open label | EXPERIMENTAL | - |
| Experimental: PTX-022 | EXPERIMENTAL | PTX-022 QTORIN |
| Active | EXPERIMENTAL | - |
| Vehicle | PLACEBO_COMPARATOR | - |
| Open label period | EXPERIMENTAL | - |
| Randomized period - Dosing A | PLACEBO_COMPARATOR | - |
| Randomized period - Dosing B | EXPERIMENTAL | - |
| Randomized period - Dosing C | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| PTX-022 | DRUG | QTORIN rapamycin 3.9% anhydrous gel |
| Vehicle comparator | DRUG | Vehicle comparator topical gel |
| Placebo | DRUG | Placebo topical |
Key Inclusion Criteria: * Completed the PALV-05 (VAPAUS) study * Agree to contraceptive use Key Exclusion Criteria: * Females who are pregnant or breastfeeding * Concomitant use of sirolimus or any sirolimus-containing medications (systemic or topical) * Any significant concurrent condition that ...
PTX-022 is an investigational topical sirolimus gel being studied for angiokeratomas, basal cell carcinomas (BCCs) in Gorlin syndrome patients, microcystic lymphatic malformation, and pachyonychia congenita. It is a small molecule mTOR inhibitor developed by Palvella Therapeutics, Inc. (PVLA).
PTX-022 targets mTOR, a key regulator of cell growth and proliferation. By inhibiting mTOR, the drug aims to reduce abnormal cell growth associated with the conditions it is being studied for, including angiokeratomas and basal cell carcinomas in Gorlin syndrome patients.
PTX-022 is being developed by Palvella Therapeutics, Inc., a biopharmaceutical company traded under the ticker PVLA. The company is conducting clinical trials to evaluate the drug's safety and efficacy in multiple rare dermatologic conditions.
PTX-022 is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. Clinical trials are ongoing or completed for several indications, including a Phase 2 study for angiokeratomas that is currently recruiting participants.
PTX-022 has three clinical trials listed on ClinicalTrials.gov. NCT07552077 is a recruiting Phase 2 study for angiokeratomas. NCT05643872 is a recruiting Phase 3 study for pachyonychia congenita. NCT05050149 and NCT04893486 are completed Phase 2 studies for microcystic lymphatic malformation and BCCs in Gorlin syndrome patients, respectively.
PTX-022 is also known as QTORIN sirolimus or QTORIN rapamycin. Clinical trial titles refer to it as QTORIN Rapamycin 3.9% Anhydrous Gel and QTORIN Sirolimus, indicating these names refer to the same investigational drug.