PVLA — what changed in the latest 10-Q

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

• We are a clinical-stage biopharmaceutical company whose vision is to become the leading rare disease biopharmaceutical company focused on developing and, if approved, commercializing novel therapies to treat patients suffering from serious, rare skin diseases and vascular malformations for which the…

• 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…

Text removed vs the prior filing · source: 10-Q · 2025-11-12

• We are a clinical-stage biopharmaceutical company whose vision is to become the leading rare disease biopharmaceutical company focused on developing and, if approved, commercializing novel therapies to treat patients suffering from serious, rare skin diseases for which there are no FDA-approved ther…

• In May 2025, we received initial proceeds of $0.5 million from our previously announced grant from the FDA Office of Orphan Products Development supporting our ongoing Phase 3 SELVA trial.

• In June 2025, the United States Patent and Trademark Office (USPTO) granted us our sixth U.S. patent (No. 12,329,748) covering 0.1-20% anhydrous compositions of rapamycin and other mTOR inhibitors.

• In June 2025, we announced the successful completion of enrollment in our Phase 3 SELVA trial with 51 subjects enrolled.

• In September 2025, we announced the successful completion of enrollment in our Phase 2 TOIVA trial with 16 subjects enrolled.

Text added 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).

Text removed vs the prior filing · source: 10-Q · 2025-11-12

• 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…

• November 18, 2026 or such earlier date upon completion of all trades under the plan (or the 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 August

• 19th, Kathleen Goin, our Chief Operating Officer, entered into a Rule 10b5-1 trading plan that provides for the potential future sale of a limited portion of her Common Stock, as part of Ms. Goin’s long-term financial planning objectives. The plan was adopted during an open trading window, at a time…

• 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 September 30, 2025.