Palvella Therapeutics (PVLA) Chardan's 10th Annual Healthcare Conference summary

Pipeline progress and funding

• Since becoming public, advanced QTORIN rapamycin from one indication to four; reported Phase III data in microcystic lymphatic malformations (MLM) and Phase II data in cutaneous venous malformations (CVM).

• Phase II is ongoing in clinically significant angiokeratomas; Phase II for disseminated superficial actinic porokeratosis (DSAP) with QTORIN pitavastatin is expected to start this quarter.

• Raised $230 million earlier this year, with funding expected to support the programs through the next couple of years of development.

• By year-end, plans to announce two diseases: a fourth QTORIN rapamycin indication and a new QTORIN product; expects three active ingredients across six diseases in development.

MLM filing and commercialization

• Rolling NDA submission for QTORIN rapamycin was filed at the end of August; expects FDA feedback by the end of October on the PDUFA date, anticipated in H1 next year, likely around late April.

• Plans to transition to a commercial-stage business following the anticipated FDA decision; a commercial team is conducting disease education and awareness with prospective prescribers.

• MLM is a congenital, progressive disease linked to a PI3K mutation and mTOR activation; lymphatic growth can protrude through skin, with lymphorrhea, wounds and serious infections among severe complications.

• Estimates more than 30,000 U.S. patients; existing options include surgery, sclerotherapy and off-label rapamycin, with temporary or ineffective results reported.

• Expected pricing is $100,000-$200,000 per patient annually; estimates multi-billion-dollar TAM and at least $1 billion in peak sales for MLM.

QTORIN platform and CVM opportunity

• QTORIN is an anhydrous topical gel platform developed through more than two years of work and 80 prototypes; designed for tunable drug loading and release, skin delivery, new IP and manufacturing efficiencies.

• CVM affects more than 75,000 patients, at least twice the MLM population; symptoms include bleeding, thrombosis, pain and functional impairment.

• QTORIN treatment is focused on cutaneous CVM manifestations, not internal disease manifestations; Phase II patients reported functional limitations, and a case described improvement in daily activities after 12 weeks.

• Following an end-of-Phase II meeting expected this quarter, plans to finalize and announce a Phase III design with FDA; may reapply for breakthrough therapy designation if FDA requests, while prioritizing an efficient trial start.

• Combined MLM and CVM peak sales opportunity estimated at $1 billion-$3 billion, with roughly a one-third/two-thirds split; same product and pricing, with larger average lesions and patient population supporting greater CVM revenue potential.