• Total Revenue: $139 million for Q3 2024, representing 42% growth year-over-year.
• CRISPRIDA Revenue: $98 million, with $56 million from North America, $36 million from Latin America and Turkey, and $6 million from Europe.
• Dojolvi Revenue: $21 million for the quarter.
• Evkeeza Revenue: $11 million for the quarter.
• Mepsevii Revenue: $10 million for the quarter.
• Operating Expenses: $271 million, including R&D expenses of $170 million and SG&A expenses of $80 million.
• Net Loss: $134 million or $1.40 per share for Q3 2024.
• Cash and Equivalents: $825 million as of September 30, 2024.
• Net Cash Used in Operations: $67 million for Q3 2024 and $335 million for the nine months ended September 30, 2024.
• 2024 Revenue Guidance: Reaffirmed total revenue range of $530 million to $550 million.
• CRISPRIDA Full Year Revenue Expectation: Towards the upper end of $375 million to $400 million.
• Dojolvi Full Year Revenue Expectation: Between $75 million and $80 million.

• Warning! GuruFocus has detected 4 Warning Signs with RARE.

Release Date: November 05, 2024

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) reported a 42% year-over-year revenue growth for the third quarter of 2024, reaching $139 million.
• The company is on track for three near-term BLA submissions and potential approvals for treatments targeting Sanfilippo Syndrome Type A, GSD1A, and osteogenesis imperfecta.
• Ultragenyx has expanded its geographic access, with significant revenue contributions from Latin America, Europe, the Middle East, and Japan.
• The FDA granted breakthrough therapy designation for UX143 (setrusumab) for osteogenesis imperfecta, highlighting its potential clinical benefits.
• Ultragenyx has successfully launched Evkeeza in Japan and is progressing with the conditional filing package for Dojolvi, strengthening its rare disease business in the region.

Negative Points

• Ultragenyx reported a net loss of $134 million for the third quarter of 2024, with total operating expenses reaching $271 million.
• The company anticipates a net cash usage of around $400 million for the year, indicating significant cash burn.
• There is variability in revenue from Latin America due to uneven ordering patterns, which could affect financial projections.
• The commercialization of new therapies may require substantial investment in salesforce expansion and manufacturing capabilities.
• The path to profitability is projected by the end of 2026, indicating a longer timeline before achieving financial self-sufficiency.

Q & A Highlights

Q: Who is the ideal patient for the Wilson gene therapy program? Would it be suited for patients well-controlled on copper chelators or zinc, or only for the most severe patients? A: Emil Kakkis, President, CEO, and Director, explained that about 20% of Wilson patients are not well-controlled or can't tolerate medications, making them the most addressable population. While some patients are stable, others not optimally managed could benefit from the treatment. The potential market remains large due to the size of the disease population.

Q: Regarding the interim analyses for the OI-AML study, if the study continues past the first interim, does it affect the likelihood of success? How would the timeline to commercialization vary? A: Emil Kakkis noted that whether the first interim hits or not doesn't impact the outcome, as it relates to how fast the lines separate. The commercialization timeline could be shortened if the first interim is successful, but the second interim would not significantly delay the BLA filing.

Q: What data should we focus on for the upcoming Angelman syndrome updates at medical conferences? A: Emil Kakkis mentioned that the focus will be on long-term data through day 338, showing the transformation of raw scores and GSV, and overall safety. This will help set up for the Phase III trial.

Q: When can we expect data from the additional stage one cohort for Wilson's disease, and what would you want to see to gain confidence in the regulatory and commercial outlook? A: Emil Kakkis stated that the study is expected to start early next year, with data taking most of the year to collect. They aim to see consistent reduction in standard care in the majority of patients, indicating a potent gene therapy.

Q: For UX111 for Sanfilippo syndrome, will heparan sulfate be the sole biomarker in the BLA, or will other measurements be included? A: Emil Kakkis confirmed that in addition to heparan sulfate, gangliosides, NSL data, and brain volume data will be included to support the efficacy chain leading to Bayley scores.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.