NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a $2.77 billion market cap biotech company whose shares have declined over 22% in the past week, announced Friday that the U.S. Food and Drug Administration has issued a Complete Response Letter for its gene therapy UX111 (ABO-102) intended to treat Sanfilippo syndrome type A (MPS IIIA). According to InvestingPro data, the stock is currently trading near its 52-week low of $29.06.
The FDA cited specific chemistry, manufacturing and controls (CMC) related observations that the company believes are "readily addressable." According to the press release statement, these issues are related to facilities and processes rather than product quality. Despite the setback, InvestingPro analysis shows the company maintains strong liquidity with a current ratio of 2.4, indicating sufficient resources to address these challenges.
Ultragenyx plans to work with the FDA over the coming months to resolve the observations, after which it will resubmit the Biologics License Application (BLA). The company anticipates up to a six-month review period following resubmission, potentially delaying approval until 2026.
The FDA acknowledged that the clinical data provided for UX111 are "robust" and that biomarker data offer supportive evidence. The regulatory agency did not raise concerns about the clinical data package or clinical inspections in its response.
UX111 is a one-time intravenous gene therapy designed to deliver a functional copy of the SGSH gene to address the enzyme deficiency that causes the rare lysosomal storage disease. The therapy has received multiple special designations including Regenerative Medicine Advanced Therapy and Fast Track in the U.S.
Sanfilippo syndrome type A is a rare fatal disorder characterized by rapid neurodegeneration beginning in early childhood. It affects approximately 3,000 to 5,000 patients in commercially accessible regions and has no approved treatments, with a median life expectancy of 15 years.
Emil D. Kakkis, CEO and president of Ultragenyx, stated that the company's goal remains getting UX111 to patients as quickly as possible, noting its importance to the Sanfilippo community. The company has demonstrated strong revenue growth of 33.46% over the last twelve months, and InvestingPro analysis indicates the stock is currently trading below its Fair Value.
In other recent news, Ultragenyx Pharma's Phase 3 Orbit study for setrusumab has faced a setback, missing its predetermined interim analysis thresholds. Despite this, the trial will continue to its final analysis, with results expected by the end of 2025. Several analyst firms have maintained their ratings on Ultragenyx. TD Cowen reiterated a Buy rating with a price target of $86, expressing confidence in the therapy's potential despite the interim results. Morgan Stanley also upheld an Overweight rating and a $65 price target, citing the unmet need in osteogenesis imperfecta and the scientific rationale for setrusumab's efficacy. Wells Fargo lowered its price target to $65 from $88 but maintained an Overweight rating, suggesting the program shouldn't be fully written off. Canaccord Genuity maintained a Buy rating with a $136 price target, viewing the stock decline as excessive. BofA Securities also retained a Buy rating and an $80 price target, indicating a 60% probability of success for the program.
These developments underscore the ongoing interest and varied perspectives among analysts regarding Ultragenyx's potential.