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

Explicit Guidance:

• NDA/PDUFA Timeline: Expect FDA feedback on the rolling submission by the end of October; PDUFA date expected in the first half of next year, likely around the end of April.
• Commercial Transition: Targeted transition from a development-stage company to a commercial company following NDA approval.
• Pricing Strategy: Projected price range of $100,000 to $200,000 per patient per year.
• Peak Sales Projections: Forecast for Cuturin rapamycin combined peak sales between $1 billion and $3 billion, with a 1/3 to 2/3 split ($1B for microcystic lymphatic malformations [MLM]; $2B for cutaneous venous malformations [CVM]).
• Capitalization: Fully funded through $230 million raised earlier this year to cover development for the next couple of years.

Business-Segment Outlook:

• Lead Product (Cuturin rapamycin): Advancing to commercialization for MLM (NDA filed); initiating Phase 2 trial for clinically significant angiokeratomas (third indication).
• Second Program (Cuturin pitavastatin): Intending to start Phase 2 trial for disseminated superficial actinic porokeratosis (DSAP) later this quarter.
• Pipeline Expansion: Two additional new programs (one fourth indication for Cuturin rapamycin and one new Cuturin product with a new active ingredient) to be announced by the end of the year.
• Target End State: Portfolio of three drugs targeting six diseases by year-end.
• Phase 2 CVM Status: Phase 2 data already announced; focus shifting to designing the Phase 3 pivotal trial.
• Indication Scope (CVM): Drug will treat cutaneous manifestations of venous malformations, excluding deep/internal manifestations.

Market and Competitive Outlook:

• TAM Estimation: MLM addressable market identified as >30,000 patients in the US; CVM identified as >75,000 patients (approx. double MLM size).
• Competitive Landscape: Current standard of care noted as ineffective or temporary (surgery, sclerotherapy, off-label compounded rapamycin); positioning Cuturin as a differentiated, platform-based solution.
• Market Penetration: Commercial team actively seeding the market with disease education and engaging core prescribers and the payer community.

Macro / Industry Outlook:

• Regulatory Pathway: Anticipating FDA interaction to finalize Phase 3 trial design for CVM via an End of Phase 2 meeting (imminent).
• Breakthrough Therapy: Will reapply for Breakthrough Therapy designation if deemed the most efficient path by the FDA; otherwise will prioritize immediate trial initiation.

Strategic Initiatives:

• Commercial Team Buildout: Completed hiring of a core executive team, including a Chief Commercial Officer (ex-Dompe), Head of Sales (ex-Insight), Head of Market Access (ex-Crystal Biotech), Head of Marketing, and Head of Medical Affairs.
• Technology Platform: Leveraging the "Cuturin" anhydrous gel platform to enable tunable drug loading, high manufacturing efficiency, and generation of new IP for future molecule combinations.
• Resource Allocation: Utilizing recent capital raise to execute multiple parallel development tracks across the pipeline.

Risks and Headwinds to Outlook:

• Regulatory Uncertainty: Failure to secure FDA agreement on the Phase 3 trial design for CVM or potential delays in NDA approval/PDUFA timing could delay commercial launch.
• Breakthrough Therapy Status: Failure to obtain Breakthrough Therapy designation for CVM could potentially impact the speed of the regulatory pathway or trial design efficiency.
• Execution Risk: Transitioning from clinical development to full commercial operations introduces execution risks regarding sales force ramp-up and payer negotiations.

Management Tone on the Future:

• Confidence: High confidence in the platform's ability to "unlock billion-dollar opportunities" rapidly through repeated molecule-pairing.
• Urgency: Strong emphasis on speed to market ("get drug to patients as fast as possible") and willingness to forgo certain designations (like initial Breakthrough Therapy) to accelerate trial initiation.
• Growth Outlook: Optimistic view on the commercial potential, citing "world-class" team capabilities and a multi-billion dollar TAM across the pipeline.

Miscellaneous:

• Company Structure: Transitioned from private to public a couple of years ago; currently operating as a biotech focused on rare skin and vascular anomaly diseases.
• Product Platform: Cuturin rapamycin is an anhydrous gel developed after testing 80 prototypes over two years.