QNRX

Clinical Progress

• Released first clinical data from Netherton syndrome trial under open investigational new drug application (IND) with US FDA, initial data was encouraging with no safety signals. Submitted protocol amendments including increasing study sizes, changing dosing frequency to twice daily, eliminating lower dose from blinded study, and lowering eligibility age to 14, all cleared by FDA and implemented.

Commercial Efforts

• Entered 9 commercial partnerships for QRX003 covering 61 countries, plan to establish own commercial infrastructure in US and Western Europe for post-approval sales.

Financial Strengthening

• Completed $6.5 million public offering, entered $8 million equity line of credit, strengthening balance sheet to fund operations into 2026.

M&A Strategy

• Focused on expanding product portfolio via acquisition/in-licensing within rare and orphan diseases, disciplined in approach and will not execute deals unless makes sense for the company

Clinical Guidance

• Open-label study readout expected this summer, blinded study data targeted for early 2025. Plan to enroll as many pediatric patients as eligible in studies, with eligibility age reduced to 14 skewing towards younger population.

Financial and M&A Guidance

• Will continue exploring M&A opportunities within rare and orphan diseases, timing of deals depends on structure; expect to speak with FDA about rare pediatric, orphan priority sometime in 2024. Strong cash position enables completion of clinical testing for lead asset for Netherton syndrome

• Forward-looking statements involve risks and uncertainties where actual results may differ materially. Other companies filing INDs or initiating Netherton studies face timelines being pushed or abandoned

Q: How many pediatric patients plan to enroll in the randomized portion of the study and what's the safety database need from FDA?

A: As many as possible, eligibility age reduced to 14 has skewed towards younger population, FDA hasn't specified a hard number for safety database Q: When will the next data readout occur?

A: Open-label study readout expected this summer, blinded study data targeted for early 2025 Q: How have patients tolerated twice daily dosing and any discontinuation of systemic therapy?

A: Patients remain on systemic therapy during open-label study, no safety concerns with twice daily dosing observed so far Q: What are M&A opportunities looking like?

A: Focus on rare and orphan diseases, later stage assets with clinical data, may need additional raise for deals depending on structure Q: Updates on talking to FDA about rare pediatric, orphan priority?

A: Sometime in 2024