Ultragenyx Pharmaceutical (RARE) Q3 2024 earnings summary

Executive summary

• Q3 2024 revenue grew 42% year-over-year to $139–$139.5 million, driven by strong Crysvita and Dojolvi sales, global expansion, and royalty income.

• Net loss for Q3 2024 was $133.5–$134 million ($1.40 per share), an improvement from $159.6–$160 million ($2.23 per share) in Q3 2023, reflecting higher revenues and moderated expense growth.

• Four approved products across five indications, with a robust rare disease pipeline and rapid development timelines; treating patients in ~34 countries.

• Advanced multiple late-stage programs (Sanfilippo syndrome, GSDIa, osteogenesis imperfecta, Angelman Syndrome, Wilson Disease) toward regulatory submissions, with potential for first-ever approved treatments.

• Received key regulatory designations, including FDA Breakthrough Therapy for UX143 and positive regulatory feedback for Dojolvi in Japan.

Financial highlights

• Q3 2024 total revenue was $139–$139.5 million; Crysvita contributed $98 million, Dojolvi $21 million, Evkeeza $11 million, and Mepsevii $10 million.

• Operating expenses for Q3 2024 were $271–$271.5 million, including $170 million R&D, $80 million SG&A, and $21 million cost of sales; net loss per share was $1.40.

• Cash, cash equivalents, and marketable securities totaled $824.7–$825 million as of September 30, 2024.

• Net cash used in operations was $67 million for Q3 and $334.7–$335 million for the nine months ended September 30, 2024.

• Product sales rose 82% year-over-year to $77.3 million in Q3 2024, led by Crysvita (up 85%), Dojolvi (up 29%), and Evkeeza.

Outlook and guidance

• 2024 total revenue guidance reaffirmed at $530–$550 million.

• Crysvita revenue expected toward the upper end of $375–$400 million; Dojolvi revenue expected between $75–$80 million.

• Path to GAAP profitability targeted by end of 2026, supported by product launches, PRV monetization, and expense management.

• Multiple BLA submissions planned within the next year, with UX111 submission expected by year-end and DTX401 in mid-2025.

• Aspire Phase 3 study for Angelman Syndrome to initiate by end of 2024.