RARE: Upcoming Study Data Will Drive Risk Reward Into 2027 Profitability

Ultragenyx Pharmaceutical's analyst price target has been revised downward by analysts, dropping from $84.10 to $83.40. This change reflects recent updates to revenue outlooks and profit margin expectations following lighter-than-anticipated quarterly revenues and sector-wide target adjustments.

Analyst Commentary

Recent Street research on Ultragenyx Pharmaceutical has highlighted both opportunities and challenges the company faces following its latest quarterly performance and sector-wide target reviews.

Bullish Takeaways

• Bullish analysts reaffirmed their positive outlook on Ultragenyx's mid- and long-term guidance, with particular confidence in the company's pathway to profitability by fiscal year 2027.
• Recent performance of key products, including Crysvita and Dojolvi, met or exceeded quarterly expectations, supporting continued confidence in revenue drivers.
• Upcoming clinical study updates for setrusumab, particularly in the ORBIT and COSMIC studies, are seen as significant catalysts. Major institutions highlight a favorable risk-reward profile at current share price levels.
• Maintained Buy and Overweight ratings from several analysts reflect enduring optimism in Ultragenyx's growth trajectory and execution capability, despite some revenue headwinds.

Bearish Takeaways

• Bearish analysts have lowered price targets due to lighter-than-expected total revenues, signaling uncertainty over near-term execution and commercial momentum.
• Recent target adjustments across multiple firms suggest broader sector caution and possible challenges in achieving aggressive top-line growth estimates.
• While key products performed well, total revenues lagged. This raises concerns about the company's ability to consistently deliver on revenue expectations in upcoming quarters.
• The extent of upside from new therapies remains subject to successful clinical outcomes and subsequent market adoption. This carries inherent risk and could impact valuation if not achieved.

What's in the News

• Ultragenyx Pharmaceutical reaffirmed its earnings guidance for 2025, projecting total revenue between $640 million and $670 million. This represents an estimated 14-20% growth over 2024. (Key Developments)
• The first patient has been dosed in the global Aurora study of GTX-102 (apazunersen), expanding clinical trials to include a broader Angelman syndrome patient population and regions not covered in previous studies. (Key Developments)
• Enrollment for the global Phase 3 Aspire study of GTX-102 in Angelman syndrome was completed ahead of schedule. Data is expected in the second half of 2026. (Key Developments)
• Ultragenyx announced positive longer-term Phase 3 results for DTX401 gene therapy in glycogen storage disease type Ia. Results showed sustained reductions in daily cornstarch intake and improved glycemic control through 96 weeks, with an acceptable safety profile. (Key Developments)

Valuation Changes

• Consensus Analyst Price Target has decreased moderately from $84.10 to $83.40. This reflects recent adjustments in analyst expectations.
• Discount Rate has risen slightly, increasing from 7.45% to 7.46%. This change indicates a marginal increase in perceived investment risk.
• Revenue Growth projections have fallen modestly, moving from 32.49% to 31.88%.
• Net Profit Margin estimate has dropped significantly, declining from 9.78% to 5.65%. This suggests a more cautious outlook on future profitability.
• Future P/E ratio has increased sharply, rising from 79.3x to 136.8x. This reflects reduced earnings expectations relative to Ultragenyx’s share price.