Palvella Therapeutics, Inc. Common Stock Chardan's 10th Annual Healthcare Conference

Sure. Thanks, Gautam. Thanks for having us. Palvella Therapeutics is a company focused on developing drugs to improve skin and vascular anomaly diseases. We do that by focusing on diseases where we can be first, and we focus on serious diseases that have no approved treatments. We transitioned to a public company a couple of years ago. At the time, we were developing QTORIN rapamycin, our lead product for just one disease, microcystic lymphatic malformations. Today, we're in four different diseases. We've, since the time we went public, announced our Phase III data in microcystic lymphatic malformations, our Phase II data in cutaneous venous malformations.

We've started a Phase II trial in clinically significant angiokeratomas, our third indication with our lead product, QTORIN rapamycin. Then we've announced our second program, QTORIN pitavastatin, where we're intending to start a Phase II trial later this quarter. We've progressed all that over the course of the last couple of years. We've successfully raised $230 million earlier this year. So we're funded through all of those projects to take them all to the next couple of years of development. That's the setup for now. Going forward, we will be expecting to take our lead product, which we filed our NDA for, to be commercial. We expect to hear back from the FDA any day now. We filed our rolling submission in the end of August, and we should expect to hear by the end of October about when our PDUFA date might be.

Yeah. We expect that to be sometime in the first half next year, likely in the end of April timeframe, and then we'll transition to a commercial company.

Sure. Microcystic lymphatic malformations is a serious, rare, chronic, debilitating, progressive disease.

Right. It results from a genetic mutation.

It's present at birth, and it's a PI3K mutation that leads to overactive or hyperactivated mTOR in your lymphatic cells. It ends up with your lymphatic system growing progressively out through the skin and protruding out. The most serious symptom of microcystic lymphatic malformations tends to be lymphorrhea, or the leaking of your lymph fluid out onto your skin, which breaks down the skin barrier and ends up with basically an open wound to the outside environment and results in serious infections in both the skin and deeper. There are more than 30,000 patients in the U.S., which we've verified through various different claims analysis, and today, there's very poor treatment. There's things like surgery, sclerotherapy, and others that have temporary or ineffective results, and sometimes off-label rapamycin gets used either orally or topically.

Yeah. We're progressing towards our commercialization by putting in place a team that we think is world-class.

We started about 18 months ago with our Chief Commercial Officer, Ashley Kline. She was previously at Dompé, launched a drug called OXERVATE.

Very successfully drove that to over $500 million very quickly. She's put in place her core lieutenants. We brought on Kent Taylor to head sales. Kent, previously from Incyte and then most recently Arcutis. We brought on Jen J. McDonough to head market access, and she's most recently from Krystal Biotech, leading VYJUVEK through its launch from a market access standpoint.

Yeah. With those two folks, Vimal Patel and Peter Finlayson on marketing and head of medical affairs, we've got a commercial team today that's spending their time on disease education and disease awareness, going out and meeting with the core folks that ultimately will be our prescriber base.

We're seeding the market today with all this disease education. From an ultimate size standpoint, we've had good conversations so far with the payer community. We expect to price the drug at $100,000-$200,000 per patient per year. That translates into a multi-billion dollar TAM. What we've said is we think it should be at least a billion-dollar peak sales product for us.