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Ultragenyx (RARE) Aligns With FDA on Phase III Neuro Study Plans
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Ultragenyx Pharmaceutical (RARE - Free Report) announced that it has reached alignment with the FDA regarding the phase III study design and endpoints for GTX-102, an antisense oligonucleotide for Angelman syndrome, following the completion of a successful end-of-phase II meeting with the regulatory body. The company expects to initiate the same by the end of 2024.
In the meeting, Ultragenyx mainly discussed the positive interim data from the phase I/II study, announced in April 2024, with the FDA. The study evaluated the safety and tolerability of GTX-102 administered by intrathecal injection to pediatric patients with Angelman syndrome, a rare and neurogenetic disorder, with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.
Per RARE, the pivotal phase III sham-controlled study will evaluate the safety and primary efficacy of GTX-102 in approximately 120 Angelman syndrome patients, with a genetically confirmed diagnosis of full maternal UBE3A gene deletion over a 48-week treatment period. The primary endpoint of the late-stage study will be the improvement in cognition assessed by the Bayley-4 cognitive raw score. Patients in the control group who complete the study can switch to treatment once the double-blind phase concludes.
Year to date, shares of Ultragenyx have lost 8.2% compared with the industry’s 1% decline.
Image Source: Zacks Investment Research
The company also reported that the phase III study will assess the Multi-domain Responder Index across cognition, receptive communication, behavior, gross motor function and sleep as a key secondary endpoint. Individual secondary endpoints for communication, behavior, motor function and sleep have been agreed upon with the FDA as well. Further feedback on the conduct and analysis of these endpoints may be provided by the FDA's Division of Clinical Outcomes Assessment.
In the EU, Ultragenyx has met with the European Medicines Agency in a PRIME meeting, receiving approval for the phase III study design, dosing and evaluations. It also plans to meet with the Japanese regulatory body in the coming weeks to discuss the same.
Alongside the planned pivotal phase III study, RARE also discussed with the FDA its plans to initiate an open-label clinical study to evaluate the safety and efficacy of GTX-102 for treating other Angelman syndrome genotypes in other patient age groups. This additional study aims to enable treatment for a broader range of Angelman syndrome patients.
At present, there are no approved disease-modifying treatments for Angelman syndrome, which represents a significant unmet medical need for patients suffering from the disease.
Ultragenyx Pharmaceutical Inc. Price and Consensus
In the past 60 days, estimates for ANI Pharmaceuticals 2024 earnings per share (EPS) have improved from $4.41 to $4.44. EPS estimates for 2025 have improved from $4.85 to $5.42. Year to date, shares of ANIP have jumped 14.2%.
ANIP’s earnings beat estimates in each of the trailing four quarters, the average surprise being 53.90%.
In the past 60 days, estimates for Adaptive Biotechnologies’ 2024 loss per share have narrowed from $1.30 to $1.29, while loss per share estimates for 2025 have narrowed from $1.08 to $1.02. Year to date, shares of ADPT have lost 11.2%.
ADPT’s earnings beat estimates in two of the trailing four quarters, meeting the same once and missing on the remaining occasion, the average surprise being 0.65%.
In the past 60 days, estimates for RAPT Therapeutics’ 2024 loss per share have narrowed from $2.94 to $2.93. Loss per share estimates for 2025 have narrowed from $2.06 to $2.05. Year to date, shares of RAPT have plunged 85.4%.
RAPT’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 3.19%.
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Ultragenyx (RARE) Aligns With FDA on Phase III Neuro Study Plans
Ultragenyx Pharmaceutical (RARE - Free Report) announced that it has reached alignment with the FDA regarding the phase III study design and endpoints for GTX-102, an antisense oligonucleotide for Angelman syndrome, following the completion of a successful end-of-phase II meeting with the regulatory body. The company expects to initiate the same by the end of 2024.
In the meeting, Ultragenyx mainly discussed the positive interim data from the phase I/II study, announced in April 2024, with the FDA. The study evaluated the safety and tolerability of GTX-102 administered by intrathecal injection to pediatric patients with Angelman syndrome, a rare and neurogenetic disorder, with a genetically confirmed diagnosis of full maternal UBE3A gene deletion.
Per RARE, the pivotal phase III sham-controlled study will evaluate the safety and primary efficacy of GTX-102 in approximately 120 Angelman syndrome patients, with a genetically confirmed diagnosis of full maternal UBE3A gene deletion over a 48-week treatment period. The primary endpoint of the late-stage study will be the improvement in cognition assessed by the Bayley-4 cognitive raw score. Patients in the control group who complete the study can switch to treatment once the double-blind phase concludes.
Year to date, shares of Ultragenyx have lost 8.2% compared with the industry’s 1% decline.
Image Source: Zacks Investment Research
The company also reported that the phase III study will assess the Multi-domain Responder Index across cognition, receptive communication, behavior, gross motor function and sleep as a key secondary endpoint. Individual secondary endpoints for communication, behavior, motor function and sleep have been agreed upon with the FDA as well. Further feedback on the conduct and analysis of these endpoints may be provided by the FDA's Division of Clinical Outcomes Assessment.
In the EU, Ultragenyx has met with the European Medicines Agency in a PRIME meeting, receiving approval for the phase III study design, dosing and evaluations. It also plans to meet with the Japanese regulatory body in the coming weeks to discuss the same.
Alongside the planned pivotal phase III study, RARE also discussed with the FDA its plans to initiate an open-label clinical study to evaluate the safety and efficacy of GTX-102 for treating other Angelman syndrome genotypes in other patient age groups. This additional study aims to enable treatment for a broader range of Angelman syndrome patients.
At present, there are no approved disease-modifying treatments for Angelman syndrome, which represents a significant unmet medical need for patients suffering from the disease.
Ultragenyx Pharmaceutical Inc. Price and Consensus
Ultragenyx Pharmaceutical Inc. price-consensus-chart | Ultragenyx Pharmaceutical Inc. Quote
Zacks Rank & Stocks to Consider
Ultragenyx currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are ANI Pharmaceuticals, Inc. (ANIP - Free Report) , Adaptive Biotechnologies Corporation (ADPT - Free Report) and RAPT Therapeutics, Inc. (RAPT - Free Report) , each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, estimates for ANI Pharmaceuticals 2024 earnings per share (EPS) have improved from $4.41 to $4.44. EPS estimates for 2025 have improved from $4.85 to $5.42. Year to date, shares of ANIP have jumped 14.2%.
ANIP’s earnings beat estimates in each of the trailing four quarters, the average surprise being 53.90%.
In the past 60 days, estimates for Adaptive Biotechnologies’ 2024 loss per share have narrowed from $1.30 to $1.29, while loss per share estimates for 2025 have narrowed from $1.08 to $1.02. Year to date, shares of ADPT have lost 11.2%.
ADPT’s earnings beat estimates in two of the trailing four quarters, meeting the same once and missing on the remaining occasion, the average surprise being 0.65%.
In the past 60 days, estimates for RAPT Therapeutics’ 2024 loss per share have narrowed from $2.94 to $2.93. Loss per share estimates for 2025 have narrowed from $2.06 to $2.05. Year to date, shares of RAPT have plunged 85.4%.
RAPT’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 3.19%.