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    DRUG:

    QTORIN 3.9% (rapamycin topical)

    i
    Other names: PTX-022, PTX 022, TD201
    Associations

    News

    Trials
    Company:
    Ligand, Palvella Therapeutics
    Drug class:
    mTOR inhibitor
    Related drugs:
    Associations

    News

    Trials
    1year
    TOIVA: A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 (Sirolimus) Topical Gel 3.9% in the Treatment of Cutaneous Venous Malformations (clinicaltrials.gov)
    P2, N=15, Recruiting, Palvella Therapeutics, Inc. | Not yet recruiting --> Recruiting
    Enrollment open
    |
    QTORIN 3.9% (rapamycin topical)
    1year
    TOIVA: A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 (Sirolimus) Topical Gel 3.9% in the Treatment of Cutaneous Venous Malformations (clinicaltrials.gov)
    P2, N=20, Not yet recruiting, Palvella Therapeutics, Inc.
    New P2 trial
    |
    QTORIN 3.9% (rapamycin topical)
    over1year
    Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita (clinicaltrials.gov)
    P2/3, N=73, Completed, Palvella Therapeutics, Inc. | Unknown status --> Completed
    Trial completion
    |
    QTORIN 3.9% (rapamycin topical)
    over1year
    VAPAUS: A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita (clinicaltrials.gov)
    P3, N=87, Completed, Palvella Therapeutics, Inc. | Recruiting --> Completed
    Trial completion
    |
    QTORIN 3.9% (rapamycin topical)
    over1year
    VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita (clinicaltrials.gov)
    P3, N=36, Completed, Palvella Therapeutics, Inc. | Active, not recruiting --> Completed | Phase classification: P3b --> P3
    Trial completion • Phase classification
    |
    QTORIN 3.9% (rapamycin topical)
    over1year
    Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations (clinicaltrials.gov)
    P2, N=12, Completed, Palvella Therapeutics, Inc. | Recruiting --> Completed
    Trial completion
    |
    QTORIN 3.9% (rapamycin topical)
    over1year
    PALV-04: CODY: A Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel For The Prevention Of Basal Cell Carcinomas (BCCs) In Patients With Gorlin Syndrome (clinicaltrials.gov)
    P2, N=73, Completed, Palvella Therapeutics, Inc. | Recruiting --> Completed | Phase classification: P2b --> P2
    Trial completion • Phase classification
    |
    QTORIN 3.9% (rapamycin topical)
    over1year
    SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations (clinicaltrials.gov)
    P3, N=40, Recruiting, Palvella Therapeutics, Inc. | Not yet recruiting --> Recruiting | Trial completion date: Jan 2026 --> Jul 2026
    Enrollment open • Trial completion date
    |
    QTORIN 3.9% (rapamycin topical)
    over1year
    SELVA: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations (clinicaltrials.gov)
    P3, N=50, Not yet recruiting, Palvella Therapeutics, Inc. | Initiation date: Apr 2024 --> Jul 2024
    Trial initiation date
    |
    QTORIN 3.9% (rapamycin topical)
    almost2years
    SELVA: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations (clinicaltrials.gov)
    P3, N=50, Not yet recruiting, Palvella Therapeutics, Inc.
    New P3 trial
    |
    QTORIN 3.9% (rapamycin topical)
    over4years
    Theasaponin E Inhibits Platinum-Resistant Ovarian Cancer Cells through Activating Apoptosis and Suppressing Angiogenesis. (PubMed, Molecules)
    The results showed that TSE1 had more potent cell growth inhibitory effects on ovarian cancer OVCAR-3 and A2780/CP70 cells than cisplatin and was lower in cytotoxicity to normal ovarian IOSE-364 cells...Combination treatment of TSE1 with the Notch1 signaling inhibitor tert-butyl (2S)-2-[[(2S)-2-[[2-(3,5-difluorophenyl)acetyl]amino]propanoyl]amino]-2-phenylacetate (DAPT), or the Akt signaling inhibitor wortmannin, showed a stronger inhibition toward HIF-1α activation compared with single compound treatment. Taken together, TSE1 might be a potential candidate compound for improving platinum-resistant ovarian cancer treatment via Dll4/Jagged1-Notch1-Akt-HIF-1α axis.
    Journal
    |
    PTEN (Phosphatase and tensin homolog) • NOTCH1 (Notch 1) • mTOR (Mechanistic target of rapamycin kinase) • HIF1A (Hypoxia inducible factor 1, alpha subunit) • EIF4EBP1 (Eukaryotic translation initiation factor 4E binding protein 1)
    |
    HIF1A expression • VEGFA expression
    |
    cisplatin • QTORIN 3.9% (rapamycin topical)
    over4years
    Different Apples, Same Tree: Visualizing Current Biological and Clinical Insights into CTLA-4 Insufficiency and LRBA and DEF6 Deficiencies. (PubMed, Front Pediatr)
    Successful treatment options include regular administration of soluble CTLA-4-Ig fusion protein, Treg cell-sparing immune suppressants like sirolimus or mycophenolate mofetil, and hematopoietic stem cell transplantation. This mini-review highlights the most relevant biological and clinical features as well as treatment options for CTLA-4 insufficiency and LRBA and DEF6 deficiencies.
    Clinical • Review • Journal • IO biomarker
    |
    CTLA4 (Cytotoxic T-Lymphocyte Associated Protein 4) • CD70 (CD70 Molecule) • CD27 (CD27 Molecule)
    |
    CTLA4 expression
    |
    sirolimus • QTORIN 3.9% (rapamycin topical)