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RARE Reports Positive Long-Term Data From Metabolic Disorder Study
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Key Takeaways
RARE's DTX401 showed a lasting 61% mean reduction in daily cornstarch needs at week 96 in GSDIa patients.
Patients reported fewer hypoglycemic events, better fasting tolerance, and higher quality of life.
RARE has begun rolling BLA submission for DTX401, with completion expected later this year.
Ultragenyx Pharmaceutical (RARE - Free Report) reported positive longer-term data from a late-stage study of its AAV gene therapy, DTX401, for the treatment of glycogen storage disease type Ia (GSDIa). GSDIa is a rare, life-threatening metabolic disorder that leads to severe hypoglycemia (excess glycogen storage) and other complications. Managing the disease relies on frequent cornstarch intake, but current approaches are burdensome, and no approved pharmacologic treatments exist. GSDIa affects around 6,000 people in commercially accessible regions.
Per Ultragenyx, GSDIa patients achieved greater reductions in daily cornstarch intake while sustaining low hypoglycemia, enhanced euglycemia, and better fasting tolerance following 96 weeks of treatment with the candidate.
Last year, RARE reported achieving the primary endpoint in the same phase III GSDIa study. Per the results, treatment with DTX401 led to a statistically significant and clinically meaningful reduction in daily cornstarch intake of 41% at week 48 compared to a 10% reduction in the placebo group.
RARE’s Longer-Term Metabolic Disorder Study Data in Detail
Per Ultragenyx’s 96-week data readout, GSDIa patients in the phase III study continued to show strong improvements, with both the DTX401 group and the crossover group achieving a mean reduction in daily cornstarch requirements of 61% from baseline. Patients in both groups also demonstrated statistically significant gains across other cornstarch-related measures.
Specifically, the DTX401 group saw a 70% mean reduction in nighttime cornstarch, while the crossover group achieved a 75% reduction. Across both groups, two-thirds of patients eliminated at least one nighttime cornstarch dose following DTX401 treatment. Notably, patients maintained low hypoglycemia rates and improved levels in the euglycemic range throughout the second year of the phase III study, despite substantial reductions in daily cornstarch requirements. Additionally, patients receiving DTX401 showed enhanced fasting tolerance in a controlled fasting challenge through 96 weeks of the study, indicating protection against severe hypoglycemia.
Year to date, RARE shares have lost 25.1% against the industry’s 4.5% growth.
Image Source: Zacks Investment Research
Additionally, Ultragenyx reported that the clinical benefits of DTX401 were reflected in meaningful improvements in patient-reported quality of life at week 96. According to the Patient Global Impression of Change, 83% of patients in the DTX401 group and 95% in the crossover group reported reduced disease burden. Interviews with patients at Weeks 48 and 96 of the phase III study further highlighted key benefits, including reduced cornstarch intake, fewer hypoglycemic events, decreased tiredness and improvements in physical, social, and daily regimen functioning — aligning closely with patients’ baseline treatment priorities. The results were presented at a recent medical conference in Japan.
The candidate was also well-tolerated by GSDIa patients throughout the 96 weeks and demonstrated an acceptable safety profile consistent with that observed in an earlier-stage study. Early results from an open-label cohort in Japan, comprising three pediatric patients (ages 8–17) treated with DTX401, further support the phase III data. By Week 24, all pediatric patients achieved a 95% average reduction in daily cornstarch intake and fully discontinued cornstarch intake by weeks 24-36. Glycemic control was maintained or improved, hypoglycemia was reduced, and no serious adverse events were reported.
RARE’s Regulatory Approval Pathway for DTX401
Around mid-August, Ultragenyx initiated the rolling submission of a biologics license application (BLA) to the FDA for DTX401 for GSDIa. The company has already submitted the non-clinical and clinical modules, with the chemistry, manufacturing, and controls (CMC) module expected to follow in the fourth quarter of 2025.
This step allows the FDA to begin reviewing key data early, while Ultragenyx addresses outstanding CMC and facility-related questions raised in the complete response letter issued by the FDA against its BLA for UX111, which is being developed for Sanfilippo syndrome type A.
Ultragenyx expects to finalize the DTX401 BLA later this year, moving closer to delivering a potential first-in-class therapy for this rare metabolic disorder.
Ultragenyx Pharmaceutical Inc. Price and Consensus
In the past 60 days, estimates for CorMedix’s earnings per share have increased from $1.10 to $1.52 for 2025. During the same time, earnings per share estimates for 2026 have increased from $1.46 to $2.12. Year to date, shares of CRMD have surged 57.4%.
CorMedix’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 34.85%.
In the past 60 days, estimates for Pharming Group’s 2025 loss per share have narrowed from 40 cents to 10 cents. For 2026, the estimate for PHAR’s earnings per share has improved from 7 cents to 27 cents. PHAR stock has rallied 44.3% year to date.
Pharming Group’s earnings beat estimates in two of the trailing four reported quarters and missed on the remaining two occasions, delivering an average negative surprise of 39.14%.
In the past 60 days, estimates for Kiniksa Pharmaceuticals’ 2025 earnings per share have increased from 74 cents to $1.03. Earnings per share estimates for 2026 have increased from $1.19 to $1.60 during the same period. KNSA stock has surged 84.9% year to date.
Kiniksa Pharmaceuticals’ earnings beat estimates in two of the trailing four reported quarters and missed on the remaining two occasions, delivering an average negative surprise of 330.56%.
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RARE Reports Positive Long-Term Data From Metabolic Disorder Study
Key Takeaways
Ultragenyx Pharmaceutical (RARE - Free Report) reported positive longer-term data from a late-stage study of its AAV gene therapy, DTX401, for the treatment of glycogen storage disease type Ia (GSDIa). GSDIa is a rare, life-threatening metabolic disorder that leads to severe hypoglycemia (excess glycogen storage) and other complications. Managing the disease relies on frequent cornstarch intake, but current approaches are burdensome, and no approved pharmacologic treatments exist. GSDIa affects around 6,000 people in commercially accessible regions.
Per Ultragenyx, GSDIa patients achieved greater reductions in daily cornstarch intake while sustaining low hypoglycemia, enhanced euglycemia, and better fasting tolerance following 96 weeks of treatment with the candidate.
Last year, RARE reported achieving the primary endpoint in the same phase III GSDIa study. Per the results, treatment with DTX401 led to a statistically significant and clinically meaningful reduction in daily cornstarch intake of 41% at week 48 compared to a 10% reduction in the placebo group.
RARE’s Longer-Term Metabolic Disorder Study Data in Detail
Per Ultragenyx’s 96-week data readout, GSDIa patients in the phase III study continued to show strong improvements, with both the DTX401 group and the crossover group achieving a mean reduction in daily cornstarch requirements of 61% from baseline. Patients in both groups also demonstrated statistically significant gains across other cornstarch-related measures.
Specifically, the DTX401 group saw a 70% mean reduction in nighttime cornstarch, while the crossover group achieved a 75% reduction. Across both groups, two-thirds of patients eliminated at least one nighttime cornstarch dose following DTX401 treatment. Notably, patients maintained low hypoglycemia rates and improved levels in the euglycemic range throughout the second year of the phase III study, despite substantial reductions in daily cornstarch requirements. Additionally, patients receiving DTX401 showed enhanced fasting tolerance in a controlled fasting challenge through 96 weeks of the study, indicating protection against severe hypoglycemia.
Year to date, RARE shares have lost 25.1% against the industry’s 4.5% growth.
Image Source: Zacks Investment Research
Additionally, Ultragenyx reported that the clinical benefits of DTX401 were reflected in meaningful improvements in patient-reported quality of life at week 96. According to the Patient Global Impression of Change, 83% of patients in the DTX401 group and 95% in the crossover group reported reduced disease burden. Interviews with patients at Weeks 48 and 96 of the phase III study further highlighted key benefits, including reduced cornstarch intake, fewer hypoglycemic events, decreased tiredness and improvements in physical, social, and daily regimen functioning — aligning closely with patients’ baseline treatment priorities. The results were presented at a recent medical conference in Japan.
The candidate was also well-tolerated by GSDIa patients throughout the 96 weeks and demonstrated an acceptable safety profile consistent with that observed in an earlier-stage study. Early results from an open-label cohort in Japan, comprising three pediatric patients (ages 8–17) treated with DTX401, further support the phase III data. By Week 24, all pediatric patients achieved a 95% average reduction in daily cornstarch intake and fully discontinued cornstarch intake by weeks 24-36. Glycemic control was maintained or improved, hypoglycemia was reduced, and no serious adverse events were reported.
RARE’s Regulatory Approval Pathway for DTX401
Around mid-August, Ultragenyx initiated the rolling submission of a biologics license application (BLA) to the FDA for DTX401 for GSDIa. The company has already submitted the non-clinical and clinical modules, with the chemistry, manufacturing, and controls (CMC) module expected to follow in the fourth quarter of 2025.
This step allows the FDA to begin reviewing key data early, while Ultragenyx addresses outstanding CMC and facility-related questions raised in the complete response letter issued by the FDA against its BLA for UX111, which is being developed for Sanfilippo syndrome type A.
Ultragenyx expects to finalize the DTX401 BLA later this year, moving closer to delivering a potential first-in-class therapy for this rare metabolic disorder.
Ultragenyx Pharmaceutical Inc. Price and Consensus
Ultragenyx Pharmaceutical Inc. price-consensus-chart | Ultragenyx Pharmaceutical Inc. Quote
RARE’s Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are CorMedix (CRMD - Free Report) , Pharming Group (PHAR - Free Report) and Kiniksa Pharmaceuticals (KNSA - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for CorMedix’s earnings per share have increased from $1.10 to $1.52 for 2025. During the same time, earnings per share estimates for 2026 have increased from $1.46 to $2.12. Year to date, shares of CRMD have surged 57.4%.
CorMedix’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 34.85%.
In the past 60 days, estimates for Pharming Group’s 2025 loss per share have narrowed from 40 cents to 10 cents. For 2026, the estimate for PHAR’s earnings per share has improved from 7 cents to 27 cents. PHAR stock has rallied 44.3% year to date.
Pharming Group’s earnings beat estimates in two of the trailing four reported quarters and missed on the remaining two occasions, delivering an average negative surprise of 39.14%.
In the past 60 days, estimates for Kiniksa Pharmaceuticals’ 2025 earnings per share have increased from 74 cents to $1.03. Earnings per share estimates for 2026 have increased from $1.19 to $1.60 during the same period. KNSA stock has surged 84.9% year to date.
Kiniksa Pharmaceuticals’ earnings beat estimates in two of the trailing four reported quarters and missed on the remaining two occasions, delivering an average negative surprise of 330.56%.