PVLA — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-08-04

• In April 2026, we dosed our first patients in the Phase 2 LOTU trial, a baseline-controlled clinical study of QTORIN rapamycin administered topically once daily for the treatment of clinically significant angiokeratomas. Topline results are expected in the second half of 2027.

• In April 2026, we submitted an application to the FDA for Breakthrough Therapy Designation for our cutaneous venous malformation program. Following the FDA’s recent decision in July 2026 to not grant the

• designation at this time, we are currently planning a resubmission incorporating TOIVA patient interview transcripts and new 24-week data.

• In May 2026, at the 83rd Annual Meeting of the Society for Investigative Dermatology (“SID”), we presented new data from our Phase 2 TOIVA trial of QTORIN rapamycin for the treatment of cutaneous VMs demonstrating 100% of patients with bleeding at baseline demonstrated improvement on the Cutaneous V…

• In May 2026, additional clinical data from our Phase 3 SELVA and Phase 2 TOIVA studies was presented by James Treat, MD at the International Society for the Study of Vascular Anomalies (“ISSVA”) World Congress 2026, which demonstrated that QTORIN rapamycin produced clinical improvements across multi…

Text removed vs the prior filing · source: 10-Q · 2026-05-07

• In January 2026, we completed a Preliminary Breakthrough Therapy Designation Advice meeting with the FDA for our cutaneous venous malformation program. Based on that meeting, we submitted an application to the FDA for Breakthrough Therapy Designation in the second quarter of 2026.

• In February 2026, we announced positive topline results from SELVA, a Phase 3, single-arm, baseline-controlled study, which evaluated the safety and efficacy of QTORIN rapamycin for the treatment of microcystic LMs in patients 3 years and older. In March 2026, we submitted a pre-NDA meeting request …

• the FDA, which was subsequently granted. We anticipate the meeting to occur during the second quarter of 2026.

• In February 2026, we received written feedback from the FDA on the proposed design of a Phase 2 study of approximately 10-20 patients to evaluate QTORIN rapamycin for the treatment of clinically significant angiokeratomas. In April 2026, we dosed our first patients in the Phase 2 LOTU trial, a basel…

• In March 2026, we announced the launch of "BEYOND mLM," a new disease state awareness campaign and website, BEYONDmLM.com, designed to educate, engage, and empower patients, caregivers, and healthcare professionals about microcystic LMs.

Text added vs the prior filing · source: 10-Q · 2026-08-04

• Consistent with Item 408 of Regulation S-K, the following table reflects Rule 10b5-1 trading arrangements and non-Rule 10b5-1 trading arrangements (as defined in Item 408 of Regulation S-K) entered into by any director or officer (as defined in Rule 16a-1(f) of the Exchange Act) during the quarter e…

• May 12, 2027 or such earlier date upon completion of all trades under the plan (or expiration of the orders relating to such trades without execution) or the occurrence of such other termination events as specified in the plan.

• Our officers and directors from time to time may adopt trading plans to transact in our securities for reasons such as satisfying vesting-related income tax requirements, investment diversification, or other personal reasons. On June 12, 2026, George Jenkins, a member of our Board of Directors, ente…

• of material non-public information and was reviewed and approved in accordance with the Company’s Insider Trading Policy. The plan establishes fixed criteria for potential transactions and removes discretion over the timing of trades.

• Other than as disclosed above, no director or officer (as defined in Rule 16a-1(f) of the Exchange Act) adopted or terminated a Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement (as defined in Item 408 of Regulation S-K) during the quarter ended June 30, 2026.

Text removed vs the prior filing · source: 10-Q · 2026-05-07

• During the quarter ended March 31, 2026, none of our directors or officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted or terminated a “Rule 10b5-1 trading arrangement” or “non-Rule 10b5-1 trading arrangement” (as each term is defined in Item 408(a) of Regulation S-K).