BioCryst Pharmaceuticals (BCRX) H.C. Wainwright 26th Annual Global Investment Conference summary

Commercial performance and revenue outlook

• ORLADEYO has shown strong patient growth for three consecutive quarters, with increasing physician and patient confidence in its efficacy and convenience as an oral therapy for hereditary angioedema (HAE).

• Over 90% reduction in attack rates is observed in responsive patients, matching injectable therapies, and once-daily dosing is a key differentiator.

• Revenue guidance for 2024 was raised due to higher-than-expected conversion of free drug patients to paid status and robust underlying demand.

• Patient growth is expected to continue, with a target of $1 billion in peak revenue and an average of 200 new net paid patients per year through 2029.

• Paid patient percentage is progressing toward the 85% target, up from 74.4% recently.

Market dynamics and patient trends

• About half of new patients are switching from prior prophylactic therapies, 20% are treatment-naive, and 30% are switching from acute/on-demand therapies.

• The market is shifting toward more prophylactic treatment, with the acute-only patient pool expected to shrink as prophylaxis adoption rises.

• There will always be a need for acute therapies for breakthrough attacks, but growth is expected mainly from newly diagnosed and prophylactic patients.

Patient retention, compliance, and support

• Retention rate remains steady at 60% at 12 months, with high compliance around 90%.

• Main reasons for discontinuation are perceived lack of efficacy and transient GI tolerability issues, which are addressed through physician education.

• Efforts to improve retention focus on supporting patients and physicians during the transition onto therapy.