NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ: NASDAQ:RARE) announced on Thursday that its Phase 3 study of DTX401, a gene therapy for glycogen storage disease type Ia (GSDIa), met its primary endpoint. The treatment led to a significant reduction in daily cornstarch intake while maintaining glucose control in patients aged eight and older.
The 48-week GlucoGene study demonstrated a 41.3% average reduction in cornstarch consumption for the DTX401 group versus a 10.3% reduction for the placebo group. The study also met key secondary endpoints, including fewer cornstarch doses per day and non-inferiority in glucose control.
Eric Crombez, M.D., chief medical officer at Ultragenyx, highlighted the consistency of these results with previous Phase 1/2 findings. He acknowledged the study participants and their support of the GSDIa community.
The trial included 46 patients, with 44 in the modified intention-to-treat population. All patients treated with DTX401 experienced a reduction in cornstarch intake, with significant percentages achieving more than 30% and 50% reductions.
Rebecca Riba-Wolman, M.D., director of the Glycogen Storage Disease Program & Disorders of Hypoglycemia at Connecticut Children's Medical Center/University of Connecticut Medical School, noted the potential of DTX401 to improve quality of life for GSDIa patients.
Safety outcomes were in line with previous studies, showing manageable hepatic effects with a prophylactic corticosteroid regimen and no observed dorsal root ganglion toxicity or thrombotic microangiopathy.
In addition to the Phase 3 study, long-term data from the Phase 1/2 trial of DTX401 presented at the American Society of Gene and Cell Therapy 2024 showed sustained reductions in cornstarch intake up to five years post-treatment.
Ultragenyx plans to present full 48-week data from the Phase 3 study at a scientific conference later this year and discuss these results with regulatory authorities to support a marketing application in 2025.
GSDIa is a rare inherited condition affecting an estimated 6,000 patients worldwide, characterized by the inability to regulate blood sugar due to a defective gene.
DTX401 has received orphan drug, regenerative medicine advanced therapy (RMAT), and Fast Track designations from the U.S. FDA, as well as PRIME and orphan drug designation from the European Medicines Agency.
InvestingPro Insights
As Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announces promising results from its Phase 3 study for DTX401, investors and industry observers are closely monitoring the financial health and market positioning of the company. According to InvestingPro data, Ultragenyx has a market capitalization of $3.21 billion, reflecting the market's valuation of the company's potential. Despite the clinical advancements, the company's financial metrics show challenges, with a negative P/E ratio of -4.85 and an adjusted P/E ratio for the last twelve months as of Q1 2024 at -5.18, indicating that the company is not currently profitable.
InvestingPro Tips suggest that the stock is in oversold territory based on the RSI, which could interest value-seeking investors. However, it is important to note that analysts do not anticipate Ultragenyx will be profitable this year, which aligns with the company's reported negative gross profit margin of -60.26% for the same period. Additionally, the company's high Price / Book multiple of 22.78 suggests a premium valuation relative to its book value. This is particularly relevant as the company does not pay dividends, meaning investors are likely banking on future growth and profitability for returns.
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