WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) said it remains on track to submit a New Drug Application in the second half of 2026 for QTORIN rapamycin after reporting positive Phase 3 data for the treatment of microcystic lymphatic malformations, a rare vascular disorder with no approved therapies.
The company expects a potential U.S. commercial launch in the first half of 2027 and is expanding development of the QTORIN platform into additional rare dermatologic and vascular conditions.
“Following the positive Phase 3 SELVA topline results, we believe Palvella is on a clear path toward near-term NDA submission for QTORIN rapamycin in microcystic lymphatic malformations, with the potential for approval and U.S. commercial launch in the first half of 2027,” Chief Executive Officer Wes Kaupinen said.
Palvella reported first-quarter cash, cash equivalents and short-term investments of $261.9 million as of March 31, bolstered by a February equity offering that generated net proceeds of $215.8 million.
The company completed a $230 million upsized public offering earlier this year, including the underwriters’ full exercise of an option to purchase additional shares.
Palvella stated that the Phase 3 SELVA trial for QTORIN rapamycin met its primary endpoint and all pre-specified secondary endpoints in patients with microcystic lymphatic malformations.
Results from the study are scheduled to be presented May 20 at the International Society for the Study of Vascular Anomalies World Congress 2026. The company also confirmed that the FDA granted a pre-NDA meeting, expected during the second quarter.
Beyond microcystic lymphatic malformations, Palvella is advancing QTORIN rapamycin in cutaneous venous malformations and angiokeratomas, while also developing QTORIN pitavastatin for disseminated superficial actinic porokeratosis, a rare genetic skin disease.
The company submitted an application for FDA Breakthrough Therapy Designation for QTORIN rapamycin in cutaneous venous malformations during the second quarter and expects to begin a Phase 3 study in the second half of 2026.
Palvella also dosed the first patients in its Phase 2 LOTU trial evaluating QTORIN rapamycin for clinically significant angiokeratomas, with topline results expected in the second half of 2027.
Research and development expenses increased to $9.3 million in the quarter ended March 31 from $4.1 million a year earlier, driven by manufacturing activities, consulting costs and higher headcount.
General and administrative expenses rose to $5.5 million from $3.8 million, reflecting increased staffing and expenses associated with operating as a public company.
Net loss widened to $15.8 million, or $1.20 per share, compared with a loss of $8.2 million, or $0.74 per share, during the same period last year.
Palvella stated that it plans to announce an additional QTORIN product candidate and a fourth clinical indication for QTORIN rapamycin in the second half of 2026.
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