Recent Insider Transactions Derivative • May 22
Chief Operating Officer notifies of intention to sell stock Kathleen Goin intends to sell 4k shares in the next 90 days after lodging an Intent To Sell Form on the 20th of May. If the sale is conducted around the recent share price of US$111, it would amount to US$476k. Company insiders have collectively sold US$2.3m more than they bought, via options and on-market transactions in the last 12 months. Announcement • May 22
Palvella Therapeutics Inc Presents New Selva And Toiva Data Supporting Qtorin Rapamycin As A Potential First-In-Disease Therapy For Multiple Serious Rare Vascular Malformations Palvella Therapeutics, Inc. announced new clinical data from the Phase 3 SELVA and Phase 2 TOIVA studies were reported at the International Society for the Study of Vascular Anomalies World Congress 2026 in Philadelphia, PA. In SELVA Phase 3 study, 100% of participants (13/13) aged 6–11 years were rated as “Much Improved” (+2) or “Very Much Improved” (+3) on the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA) scale at Week 24, with a mean improvement of +2.46. 87% of participants (20/23) in SELVA with moderate or worse leaking/bleeding at baseline were rated as “Much Improved” (+2) or “Very Much Improved” (+3) on the mLM-IGA Leaking/Bleeding at Week 24, with a mean improvement of +2.48. 100% of SELVA participants who completed the efficacy evaluation period (43/43) were at least somewhat satisfied with QTORIN rapamycin on the TSQM-9 overall satisfaction item at Week 24, with 84% reporting extremely satisfied, very satisfied, or satisfied. Blinded independent review demonstrated pre-treatment stability during the 8-week run-in period, followed by marked improvement on QTORIN rapamycin, supporting SELVA’s single-arm, baseline-controlled design. TOIVA Phase 2 study demonstrated statistically significant improvements in both cVM-MCSS Height and cVM-MCSS Appearance at all measured time points, with increasing clinical response observed with longer duration of QTORIN rapamycin therapy. QTORIN rapamycin has the potential to become the first FDA-approved therapy and standard of care for microcystic lymphatic malformations and cutaneous venous malformations. SELVA is a Phase 3, single-arm, baseline-controlled clinical trial of QTORIN rapamycin administered topically once daily for a 24-week efficacy evaluation period followed by an open-label extension period, for microcystic lymphatic malformations (microcystic LMs). Palvella previously announced positive topline results from this study in February 2026, including achieving statistical significance on the primary endpoint, key secondary endpoint, and all pre-specified secondary efficacy endpoints. Additional data presented highlight: In participants aged 6–11 years (n=13), QTORIN rapamycin demonstrated statistically significant improvement on the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA) of +2.46 at Week 24. At Week 24, 100% of participants elected to continue QTORIN rapamycin in the Treatment Extension period, reflecting strong interest in ongoing therapy. In microcystic LMs, bleeding and leaking represent some of the most debilitating and hardest-to-control disease manifestations. In participants with moderate or worse leaking/bleeding at baseline (n=23), QTORIN rapamycin demonstrated statistically significant improvement on the mLM-IGA Leaking/Bleeding of +2.48 at Week 24. Palvella plans to initiate a Phase 3 trial of QTORIN rapamycin for the treatment of cutaneous venous malformations in the second half of 2026. QTORIN rapamycin has received FDA Fast Track Designation for cutaneous venous malformations, and Palvella has submitted an application to FDA for Breakthrough Therapy Designation in this indication. QTORIN rapamycin and QTORIN pitavastatin are for investigational use only and neither has been approved by the FDA or by any other regulatory agency for any indication. Announcement • May 16
Palvella Therapeutics Announces New Data from the Phase 2 Toiva Trial of Qtorin Rapamycin in Cutaneous Venous Malformations Palvella Therapeutics, Inc. announced new data from the Phase 2 TOIVA trial of QTORIN rapamycin in patients with cutaneous venous malformations (VMs) were presented at the 83rd Annual Meeting of the Society for Investigative Dermatology (SID) in Chicago, IL. TOIVA is a Phase 2, single-arm, open-label, baseline-controlled clinical trial of QTORIN™ rapamycin administered topically once daily for a 12-week efficacy evaluation period followed by a 12-week treatment extension period, for cutaneous VMs. As previously announced in December 2025, TOIVA achieved statistical significance on multiple pre-specified clinician-reported and patient-reported efficacy endpoints. New data presented at the 83rd Annual Meeting of the Society for Investigative Dermatology demonstrated statistically significant reduction in lesion bleeding in TOIVA and highlighted the multidimensional quality-of-life (QoL) burden for individuals living with cutaneous venous malformations as captured by baseline qualitative patient interviews. cVM-IGA: Bleeding improvement in patients with bleeding at baseline: Patients with bleeding at baseline (n=4) demonstrated a statistically significant improvement in change in cVM-IGA Bleeding scores at Week 12 with a mean effect size of +2.5 (p=0.003). cVM-IGA Bleeding is a 7-point clinician-assessed dynamic scale measuring change in bleeding from baseline to Week 12, ranging from “Very Much Worse” (-3) to “Very Much Improved” (+3). At week 12, 100% of patients (4/4) were either “Much Improved” (+2) or “Very Much Improved” (+3) on the cVM-IGA Bleeding at Week 12. 100% of patients with bleeding at baseline reported being "satisfied" or "very satisfied" with QTORIN™ rapamycin on the overall satisfaction item of the Treatment Satisfaction Questionnaire for Medication at Week 12. Baseline qualitative patient interviews highlight multidimensional quality-of-life (QoL) burden of cutaneous VMs: A pre-specified patient qualitative interview sub-study was incorporated to systematically capture the patient experience, including the symptoms, functional impacts, and treatment-related changes most meaningful to patients, consistent with FDA’s Patient-Focused Drug Development framework. QoL burden extends beyond clinical lesion severity, including bluish discoloration, pain/discomfort, swelling, protrusions, and bleeding or leakage. Patients reported limitations in physical activity, social participation, and work/school functioning, as well as emotional distress related to lesion visibility. Treatment priorities included improving appearance, reducing pain, and decreasing lesion size, highlighting patient-important domains not fully captured by traditional clinical severity assessments. Findings underscore the patient-experienced burden of moderate-to-severe cutaneous VMs and reinforce the value of patient-reported outcomes in clinical development. 
