Positive Results from QTORIN Study for Microcystic LM

Palvella Therapeutics

WAYNE, PAPalvella Therapeutics, Inc. this week announced positive topline results from the Company’s Phase 2 study of QTORIN™ rapamycin in Microcystic Lymphatic Malformations (Microcystic LM). Microcystic LM is a rare, chronically debilitating genetic disease caused by dysregulation of the PI3K/mTOR pathway. The disease is characterized by localized masses of malformed lymphatic vessels that protrude through the skin barrier and persistently leak lymph fluid (lymphorrhea) and bleed, often leading to recurrent serious infections and cellulitis. There are no FDA-approved treatments for the estimated more than 30,000 individuals living with Microcystic LM in the U.S.

“People living with Microcystic LMs face daily challenges and frequent, sometimes life-threatening hospitalizations due to cellulitis. Current treatments are inadequate, invasive, and do not address the underlying mechanisms of this debilitating disease,” said James Treat, MD, a pediatric dermatologist at Children’s Hospital of Philadelphia and study investigator. “The encouraging results from the Phase 2 study of QTORIN™ rapamycin build upon the large, growing evidence base supporting targeted therapeutic intervention of Microcystic LMs via the mTOR pathway. We look forward to potentially initiating a pivotal Phase 3 study of QTORIN™ rapamycin in the second half of 2023, and to sharing the results from that study when they are available.”

The proof-of-concept Phase 2, multi-center, open-label study featured multiple efficacy assessments, including clinician and patient global impression assessments as well as assessments of individual clinical manifestations that are important disease burdens for individuals living with Microcystic LM (lesion height, leaking, bleeding, erythema, and crusting/hyperkeratosis). Twelve individuals (n=12) with Microcystic LMs received QTORIN™ rapamycin once-daily for 12 weeks. Key findings from among the prespecified efficacy endpoints comparing end of treatment (Week 12) to the pre-treatment baseline period demonstrated improvements in several clinician and patient-reported outcomes:<

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 Efficacy Endpoints Week 12
Nominal, Two-sided
 Clinician Global Impression of Change (CGI-C) 2.42 <0.0001
 Clinician Global Impression of Severity (CGI-S) – Overall -1.33 <0.0001
•  CGI-S Height -1.67 <0.0001
•  CGI-S Leaking -0.92 0.0047
•  CGI-S Bleeding -0.92 0.0197
•  CGI-S Erythema -1.08 0.0016
•  CGI-S Crusting/Hyperkeratosis -1.17 0.0012
 Patient Global Impression of Change (PGI-C) 2.08 <0.0001
 CGI-C and PGI-C improvements are represented by increases; CGI-S improvements are represented by reductions
CGI-C and PGI-C are 7-points scales ranging from “Very Much Worse” (-3) to “Very Much Improved” (+3)
CGI-S is a 5-point lesion severity scale
p-values are nominal as there was no adjustment for multiplicity amongst efficacy endpoints
All p-values from paired t-tests vs mean change of 0 as compared to baseline
All twelve participants in the study demonstrated improvements on the PGI-C and CGI-C scales with all rated as either “Much Improved” or “Very Much Improved” by the clinicians after twelve weeks of treatment with QTORIN™ rapamycin. To help contextualize changes on efficacy endpoints and, specifically, better understand any patient quality of life impact resulting from QTORIN™ rapamycin, qualitative exit interviews were conducted by a third-party interviewer with a subset of participants from the Phase 2 study.

In the study, QTORIN™ rapamycin was generally well-tolerated with the most common adverse events being application site pain and pruritus. No participants experienced drug related serious adverse events, and no unexpected adverse events occurred. No rapamycin was detected in the systemic circulation for all participants at all timepoints in the study. Palvella plans to present additional results of the Phase 2 study, including the results of the qualitative exit interviews, at an upcoming scientific meeting.

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Palvella completed an End of Phase 2 Meeting with the FDA in February 2023. Pending additional interactions with FDA, Palvella anticipates potentially initiating a pivotal Phase 3 study of QTORIN™ rapamycin in the second half of 2023. The FDA has granted Orphan Drug and Fast Track Designations to QTORIN™ rapamycin for the treatment of Microcystic LM. The European Medicines Agency (EMA) has also granted Orphan Drug Designation to QTORIN™ rapamycin for the treatment of microcystic LMs.

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