We use cookies to understand how you use our site and to improve your experience.
This includes personalizing content and advertising.
By pressing "Accept All" or closing out of this banner, you consent to the use of all cookies and similar technologies and the sharing of information they collect with third parties.
You can reject marketing cookies by pressing "Deny Optional," but we still use essential, performance, and functional cookies.
In addition, whether you "Accept All," Deny Optional," click the X or otherwise continue to use the site, you accept our Privacy Policy and Terms of Service, revised from time to time.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
RARE's GTX-102 Gets FDA's Breakthrough Status for Angelman Syndrome
Read MoreHide Full Article
Key Takeaways
The FDA granted Breakthrough Therapy designation to Ultragenyx's GTX-102 for Angelman syndrome.
GTX-102 showed rapid, lasting improvements in AS patients during a phase I/II study over up to 3 years.
RARE is enrolling for a phase III Aspire study and planning a broader phase II/III Aurora study for GTX-102.
Ultragenyx Pharmaceuticals (RARE - Free Report) announced that the FDA has granted the Breakthrough Therapy designation to its investigational antisense oligonucleotide therapy, GTX-102 (apazunersen), to treat Angelman syndrome (AS).
Benefits of the FDA’s Breakthrough Therapy Designation Grant
The FDA’s Breakthrough Therapy designation is a process that speeds up the development and review of drugs for serious or life-threatening conditions. This designation is granted when early clinical evidence suggests the drug may significantly improve over existing treatments on one or more important clinical measures. Drugs with this designation receive more intensive guidance and organizational support from senior FDA managers.
Year to date, shares of Ultragenyx Pharmaceuticals have lost 13.2% compared with the industry’s 3.4% decline.
Image Source: Zacks Investment Research
The FDA’s decision is supported by early clinical data, including results from a phase I/II study involving 74 AS patients aged 4 to 17 with a complete maternal UBE3A gene deletion. The study showed that participants experienced consistent developmental progress with rapid, sustained and ongoing improvements across multiple symptom areas during up to three years of treatment.
AS is a rare neurogenetic disorder that affects around 60,000 people in accessible markets and leads to severe developmental challenges, including cognitive and motor impairments, speech loss, balance issues, seizures, anxiety, and sleep disturbances, taking a severe toll on the quality of life. Current treatment options for AS are limited, with no approved therapies.
Ultragenyx Pharmaceuticals is currently enrolling patients in the phase III Aspire study, evaluating AS patients with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The company also plans to initiate a phase II/III clinical study named Aurora to evaluate the safety and efficacy of GTX-102 for treating other AS genotypes in other patient age groups. This additional study aims to enable treatment for a broader range of AS patients and is expected to be initiated in the second half of 2025.
Other Key Clinical Programs in RARE’s Pipeline
Ultragenyx Pharmaceuticals has several other interesting gene therapy candidates in its pipeline. RARE and its partner, Mereo BioPharma, are jointly developing UX143 (setrusumab) monoclonal antibody forpediatric and young adult patients with osteogenesis imperfecta (OI) in two late-stage studies, Orbit and Cosmic. In October 2024, the FDA granted the Breakthrough Therapy designation to UX143 for the OI indication.
The company is also evaluating UX701, an investigational AAV9 gene therapy, in a phase I/II/III Cyprus2+ study to treat Wilson disease. In 2024, Ultragenyx announced that the phase III GlucoGene study (NCT05139316), evaluating its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia patients aged eight years and older, achieved its primary endpoint with statistical significance. The GlucoGene study also met its key secondary endpoints. The next step for the company involves discussing these results with regulatory authorities to support a marketing application soon.
A regulatory application, seeking the FDA approval of UX111, an AAV gene therapy candidate being developed for the treatment of patients with Sanfilippo syndrome type A, is currently under Priority Review. A final decision is expected on Aug. 18, 2025.
Ultragenyx Pharmaceutical Inc. Price and Consensus
In the past 90 days, loss per share estimates for Immunocore’s 2025 have improved from $1.57 to 86 cents. Loss per share estimates for 2026 have narrowed from $1.80 to $1.33 during the same period. IMCR stock has gained 5.5% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed the mark once, delivering an average surprise of 76.18%.
BAYRY’s 2025 earnings per share estimate has increased from $1.19 to $1.25 for 2025 over the past 90 days, while that for 2026 has gone up from $1.28 to $1.31 over the same timeframe. Year to date, shares of Bayer have surged 61.3%.
BAYRY’s earnings beat estimates in one of the trailing four quarters, matched twice and missed on the remaining occasion, the average negative surprise being 13.91%.
In the past 90 days, Agenus’ bottom-line estimates for 2025 have significantly improved from a loss of $4.66 per share to earnings of $1.56. During the same timeframe, estimates for 2026 loss per share have narrowed from $5.02 to $1.99. AGEN stock has soared 73.7% so far this year.
Agenus’ earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 22.71%.
See More Zacks Research for These Tickers
Normally $25 each - click below to receive one report FREE:
Image: Bigstock
RARE's GTX-102 Gets FDA's Breakthrough Status for Angelman Syndrome
Key Takeaways
Ultragenyx Pharmaceuticals (RARE - Free Report) announced that the FDA has granted the Breakthrough Therapy designation to its investigational antisense oligonucleotide therapy, GTX-102 (apazunersen), to treat Angelman syndrome (AS).
Benefits of the FDA’s Breakthrough Therapy Designation Grant
The FDA’s Breakthrough Therapy designation is a process that speeds up the development and review of drugs for serious or life-threatening conditions. This designation is granted when early clinical evidence suggests the drug may significantly improve over existing treatments on one or more important clinical measures. Drugs with this designation receive more intensive guidance and organizational support from senior FDA managers.
Year to date, shares of Ultragenyx Pharmaceuticals have lost 13.2% compared with the industry’s 3.4% decline.
Image Source: Zacks Investment Research
The FDA’s decision is supported by early clinical data, including results from a phase I/II study involving 74 AS patients aged 4 to 17 with a complete maternal UBE3A gene deletion. The study showed that participants experienced consistent developmental progress with rapid, sustained and ongoing improvements across multiple symptom areas during up to three years of treatment.
AS is a rare neurogenetic disorder that affects around 60,000 people in accessible markets and leads to severe developmental challenges, including cognitive and motor impairments, speech loss, balance issues, seizures, anxiety, and sleep disturbances, taking a severe toll on the quality of life. Current treatment options for AS are limited, with no approved therapies.
Ultragenyx Pharmaceuticals is currently enrolling patients in the phase III Aspire study, evaluating AS patients with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The company also plans to initiate a phase II/III clinical study named Aurora to evaluate the safety and efficacy of GTX-102 for treating other AS genotypes in other patient age groups. This additional study aims to enable treatment for a broader range of AS patients and is expected to be initiated in the second half of 2025.
Other Key Clinical Programs in RARE’s Pipeline
Ultragenyx Pharmaceuticals has several other interesting gene therapy candidates in its pipeline. RARE and its partner, Mereo BioPharma, are jointly developing UX143 (setrusumab) monoclonal antibody forpediatric and young adult patients with osteogenesis imperfecta (OI) in two late-stage studies, Orbit and Cosmic. In October 2024, the FDA granted the Breakthrough Therapy designation to UX143 for the OI indication.
The company is also evaluating UX701, an investigational AAV9 gene therapy, in a phase I/II/III Cyprus2+ study to treat Wilson disease. In 2024, Ultragenyx announced that the phase III GlucoGene study (NCT05139316), evaluating its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia patients aged eight years and older, achieved its primary endpoint with statistical significance. The GlucoGene study also met its key secondary endpoints. The next step for the company involves discussing these results with regulatory authorities to support a marketing application soon.
A regulatory application, seeking the FDA approval of UX111, an AAV gene therapy candidate being developed for the treatment of patients with Sanfilippo syndrome type A, is currently under Priority Review. A final decision is expected on Aug. 18, 2025.
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 Immunocore (IMCR - Free Report) , Bayer (BAYRY - Free Report) and Agenus (AGEN - Free Report) . While IMCR sports a Zacks Rank #1 (Strong Buy), BAYRY and AGEN carry a Zacks Rank #2 (Buy) each at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 90 days, loss per share estimates for Immunocore’s 2025 have improved from $1.57 to 86 cents. Loss per share estimates for 2026 have narrowed from $1.80 to $1.33 during the same period. IMCR stock has gained 5.5% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed the mark once, delivering an average surprise of 76.18%.
BAYRY’s 2025 earnings per share estimate has increased from $1.19 to $1.25 for 2025 over the past 90 days, while that for 2026 has gone up from $1.28 to $1.31 over the same timeframe. Year to date, shares of Bayer have surged 61.3%.
BAYRY’s earnings beat estimates in one of the trailing four quarters, matched twice and missed on the remaining occasion, the average negative surprise being 13.91%.
In the past 90 days, Agenus’ bottom-line estimates for 2025 have significantly improved from a loss of $4.66 per share to earnings of $1.56. During the same timeframe, estimates for 2026 loss per share have narrowed from $5.02 to $1.99. AGEN stock has soared 73.7% so far this year.
Agenus’ earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 22.71%.