Forecast

The analysts' average price target is €23.85 (+91.57%). The median is €21.53 (+72.93%).

Buy	26
Hold	6
Sell	1

News Go to news feed

Ultragenyx stock plunges 45% after Phase‑3 trial failure for Apazunersen

The drug candidate Apazunersen (GTX‑102) failed in the approval‑relevant Phase‑3 Aspire trial for Angelman syndrome, missing both the primary and key secondary endpoints. The news triggered a premarket drop of about 45%. Analysts cut ratings and sharply reduced price targets (e.g., Evercore, Baird, JPMorgan, William Blair), since Apazunersen had been seen as a central pillar of Ultragenyx’s pipeline. The stock now faces a reassessment because an important growth driver is gone. » More on deraktionaer.de
Ultragenyx share falls 40% after failed phase‑3 study of apazunersen

Ultragenyx shares plunged more than 40% in after‑hours trading after the phase‑3 Aspire study of apazunersen in Angelman syndrome missed its primary and key secondary endpoints (cognitive Bayley‑4 raw score and MDRI). The safety profile matched phase‑1/2 results but showed no efficacy differences versus control. Ultragenyx will evaluate the apazunersen program and make a decision on its future, and will review business activities to define and implement significant cost reductions. » More on de.investing.com
Ultragenyx presents positive 96-week data on GSDIa gene therapy GENGLYCOS

In the phase 3 study of GENGLYCOS (DTX401), there was an average 61% reduction in daily cornstarch intake versus baseline after 96 weeks among treated and crossover participants. 67% of the original treatment group and 72% of the crossover group achieved at least a 50% reduction in cornstarch intake, and a proportion of patients were able to discontinue nighttime cornstarch entirely. GENGLYCOS received accelerated approval in the US for patients aged eight and older; continued approval depends on confirmatory studies. Common treatment-related adverse events included transient liver enzyme elevations, and several serious adverse events were reported during the efficacy analysis, including anaphylaxis and adrenal insufficiency. » More on de.investing.com

Historical dividends Dividends

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In 2022, Ultragenyx Pharmaceutical has already paid €0.27 in dividends. The last payout was in July 2022.

Financials

In the last quarter, Ultragenyx Pharmaceutical had revenue of €187.51M and net profit of -€80.61M

(EUR)	June 2026	YoY
Revenue	187.51M	32.74%
Gross profit	157.72M	29.55%
Net profit	-80.61M	17.34%
EBITDA	-53.45M	31.03%

Fundamentals

Metric	Value
Market capitalization	€1.21B
Number of shares	98.49M
52-week high/low	€34.35 - €11.89
Dividends	No
Beta	0.32
P/E ratio	-2.45
PEG ratio	0.39
P/B ratio	-5.01
P/S ratio	1.97

Company profile

Ultragenyx Pharmaceutical Inc. ist ein biopharmazeutisches Unternehmen, das sich auf die Identifizierung, den Erwerb, die Entwicklung und die Vermarktung neuartiger Produkte für die Behandlung seltener und extrem seltener genetischer Krankheiten in Nordamerika, Europa und international konzentriert. Zu den biologischen Produkten des Unternehmens gehören Crysvita (Burosumab), ein Antikörper gegen den Fibroblasten-Wachstumsfaktor 23 zur Behandlung der X-chromosomalen Hypophosphatämie und der tumorbedingten Osteomalazie; Mepsevii, eine Enzymersatztherapie zur Behandlung von Kindern und Erwachsenen mit Mukopolysaccharidose VII; Dojolvi zur Behandlung von Störungen der Oxidation langkettiger Fettsäuren; und Evkeeza (Evinacumab) zur Behandlung der homozygoten familiären Hypercholesterinämie. Zu den Produktkandidaten des Unternehmens gehören DTX401, ein Gentherapiekandidat mit dem Adeno-assoziierten Virus 8 (AAV8) zur Behandlung von Patienten mit der Glykogenspeicherkrankheit Typ Ia; DTX301, eine AAV8-Gentherapie zur Behandlung von Patienten mit Ornithintranscarbamylase; UX143, ein humaner monoklonaler Antikörper zur Behandlung der Osteogenesis imperfecta; GTX-102, ein Antisense-Oligonukleotid zur Behandlung des Angelman-Syndroms; UX701 zur Behandlung der Wilson-Krankheit; und UX053 zur Behandlung der Glykogenspeicherkrankheit Typ III. Ultragenyx Pharmaceutical Inc. hat Kooperations- und Lizenzvereinbarungen mit Kyowa Kirin Co, Ltd, der Saint Louis University, dem Baylor Research Institute, REGENXBIO Inc, Bayer Healthcare LLC, GeneTx, Mereo, der University of Pennsylvania, Arcturus Therapeutics Holdings Inc, Solid Biosciences Inc. und Daiichi Sankyo Co, Ltd. Ultragenyx Pharmaceutical Inc. wurde im Jahr 2010 gegründet und hat seinen Hauptsitz in Novato, Kalifornien.

Name	Ultragenyx Pharmaceutical
CEO	Emil D. Kakkis
Headquarters	Novato, ca USA
Website	ultragenyx.com
Sector	Materials
Industry	Chemicals
IPO	01/31/2014
Employees	1,371