CHARDAN HEALTHCARE CONFERENCE – 10 YEARS OF DISRUPTIVE INNOVATION - PALVELLA THERAPEUTICS INC

Explicit Guidance:

• NDA Approval Timeline: Expect FDA feedback regarding the rolling submission (filed end of August) "any day now"; PDUFA date expected "any time" by end of October, likely finalized in the "first half of next year, likely at the end of April timeframe."
• Commercial Peak Sales: Anticipate the lead product (Cuturin Rapamycin) to generate "at least a billion-dollar peak sales" from the Microcystic Lymphatic Malformations (MLM) indication, with a combined peak sales target of "$1 to $3 billion" across MLM and Cutaneous Venous Malformations (CVM).
• Pricing Strategy: Target annual pricing of "$100,000 to $200,000 per patient per year."
• Revenue Split: Project a revenue distribution of approximately "one-third ($1B) for MLM" and "two-thirds ($2B) for CVM."
• Product Portfolio Count: Targeting a portfolio of "three drugs with six diseases" by the end of the current year.
• Capital Position: Funded through $230 million raised earlier this year to support development for the "next couple of years."

Business-Segment Outlook:

• Cuturin Rapamycin (Lead Product):
  • MLM Indication: Transitioning to commercial status upon FDA approval; currently filing NDA for microcystic lymphatic malformations (30,000+ US patients).
  • CVM Indication: Phase II data completed; pivoting focus to designing the most efficient Phase III trial following an imminent end-of-phase two meeting with the FDA.
  • Angiokeratomas: Phase II trial for clinically significant angiokeratomas is ongoing.
  • New Indication: A fourth indication for Cuturin Rapamycin will be announced "this year."
• Cuturin Pitavastatin:
  • DSAP Indication: Phase II trial for disseminated superficial actinic porokeratosis scheduled to commence "later this quarter."
  • New Product: A third distinct active ingredient (new Cuturin product) targeting a new disease will be announced "this year."
• Commercial Infrastructure: A world-class commercial team has been assembled, including a Chief Commercial Officer, Head of Sales, Head of Market Access, and heads of Medical Affairs and Marketing. The team is currently "seeding the market" with disease education and awareness.

Market and Competitive Outlook:

• Demand Trends: MLM is a serious, chronic, progressive genetic disease with no approved treatments; current alternatives (surgery, sclerotherapy) offer only temporary or ineffective results. CVM affects >75,000 patients and causes significant functional impairment (e.g., inability to bend limbs, wear clothing).
• Market Size: MLM represents a "billion-dollar peak sales" opportunity; CVM is estimated to be "at least double the size" of MLM due to a patient population >75,000.
• Competitive Positioning: Focuses on diseases where the company can be "first." The Cuturin platform (anhydrous gel) creates new IP for every molecule paired, offering manufacturing efficiencies and tunable drug loading.
• Pricing Power: Expects "multibillion-dollar TAM" based on payer conversations and pricing strategy; higher revenue per patient in CVM due to larger average lesion size.

Macro / Industry Outlook:

• Regulatory Pathway: Prioritizing speed to market over regulatory designations (e.g., previously missed Breakthrough Therapy); willing to reapply for Breakthrough Therapy if determined by the FDA after trial design finalization.
• Treatment Landscape: Anticipates structural shift from ineffective off-label rapamycin use and invasive procedures to a proprietary topical gel solution for both MLM and CVM.

Strategic Initiatives:

• Platform Expansion: Leveraging the "tunable" Cuturin platform to rapidly move from molecule identification to clinical development for new rare diseases.
• Pipeline Acceleration:
  • Launch Phase II for Cuturin Pitavastatin (DSAP) in the current quarter.
  • Announce a new disease indication for Cuturin Rapamycin and a new Cuturin product within the current year.
• Commercial Ramp: Immediate focus on disease education and prescriber engagement to prepare for the post-approval commercial transition.

Risks and Headwinds to Outlook:

• Regulatory Uncertainty: The specific timing of the PDUFA and the design of the Phase III trial for CVM remain dependent on FDA discussions; Breakthrough Therapy designation is not guaranteed and may be pursued later.
• Trial Execution: The efficiency of the Phase III trial design is a critical variable for speed to market.
• Market Adoption: While pricing discussions with payers have been positive, actual reimbursement rates and formulary placement remain external variables.

Management Tone on the Future:

• Confidence Level: High confidence in the commercial potential of the lead product ("world-class" team, clear pricing strategy, significant unmet need).
• Strategic Focus: Shift in priority from seeking regulatory designations to "getting drug to patients as fast as possible."
• Operational Status: Expresses satisfaction with the current funding position ("funded through all of those projects") and the progress of the pipeline, viewing the next 12-18 months as a transition from development to commercialization.

Miscellaneous:

• Corporate Structure: Transitioning from a public biotech development company to a commercial entity immediately following NDA approval.
• Investor Relations: Plans to announce Phase III trial design details "imminently" following the end-of-phase two meeting.