Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701, an adeno-associated AAV9 gene therapy which is in Phase 2 clinical trial for the treatment of Wilson liver disease. The company has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Regeneron; and Abeona. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
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FDA Approves Ultragenyx's Genglycos Gene Therapy for GSDIa
The FDA approved Ultragenyx Pharmaceutical's Genglycos, the first gene therapy for glycogen storage disease type Ia, sending shares up 7.5% in after-hours trading. The one-time treatment will carry a U.S. list price of approximately $2.7 million per patient and is expected to become available through qualified treatment centers within 30 to 60 days. Ultragenyx estimates the disorder affects only 1,500 to 2,500 Americans, and the company must now prove it can diagnose, secure reimbursement for, and treat enough patients to generate meaningful revenue. In the Phase 3 GlucoGene trial, Genglycos reduced mean daily cornstarch intake by 41% at Week 48 compared with 10% for placebo, and longer-term data showed a 61% reduction at Week 96. The FDA has required two years of additional safety and efficacy data, and Ultragenyx will manufacture the therapy at its Bedford, Massachusetts facility.
Deere jumps on upbeat Q3, Wolfspeed plunges on wider loss
U.S. stock futures were little changed on Thursday as investors assessed a fresh batch of corporate earnings and analyst actions. CrowdStrike shares fell 2.8% in premarket trading after Bloomberg reported that the cybersecurity company's chief technology officer is departing to launch an artificial intelligence-focused cybersecurity fund. Advance Auto Parts stock plunged 15.9% after its second-quarter results revealed a significant difference between headline earnings and underlying performance, with about $0.31 of the reported $1.03 adjusted diluted earnings per share coming from one-time tariff refunds. Deere shares jumped 5.1% after the agricultural and construction equipment maker delivered a stronger-than-expected fiscal third quarter, with diluted earnings per share of $5.10 and net sales of about $11 billion. Etsy gained 3.9% in premarket trading after BofA Securities upgraded the online marketplace to Buy from Neutral and raised its price target to $105 from $88. Ultragenyx surged 11.1% after the U.S. Food and Drug Administration granted accelerated approval to GENGLYCOS, also known as DTX401, a gene therapy for glycogen storage disease type Ia in patients aged eight and older. Moderna shares fell 13% in premarket trading, retreating after more than doubling in the previous session following the company's major cancer-vaccine trial announcement with Merck. Wolfspeed plunged 10.5% after the semiconductor company reported a sharply wider-than-expected fiscal fourth-quarter loss, with an adjusted loss of $2.26 per share and revenue of $149.6 million. Webull rose 13.8% after the digital brokerage reported its strongest quarterly results since going public, with second-quarter revenue of $198.8 million and adjusted earnings of $0.05 per share.
Webull, Ultragenyx lead gainers; Wolfspeed, Coty slide
Stock futures were lower early Thursday as persistent geopolitical tensions in the Middle East drove Brent crude over $92 a barrel, reinforcing broader inflation concerns. Webull shares climbed 14% after the digital investment platform reported record Q2 revenue of $198.8M, up 51% Y/Y and 24% Q/Q, beating estimates of $180.7M, while Ultragenyx Pharmaceutical surged 12% after the FDA granted accelerated approval to Genglycos, its gene therapy for glycogen storage disease type Ia. Circle Internet Group gained 5% alongside a broader rally in crypto-linked stocks as Bitcoin climbed more than 9% to around $71K, its highest level since June 2, following the U.S. Treasury's surprise decision to double planned buybacks of longer-dated Treasuries to at least $4B per operation. Wolfspeed plunged 10% after reporting a wider-than-expected FQ4 adjusted loss of $2.26 per share, versus the $1.47 consensus, while revenue rose 24% Y/Y to $149.6M, and Coty tumbled 9% after mixed FQ4 results with LFL revenue down 1% and adjusted operating income plunging 31%.
Ultragenyx Q2 revenue rises to $214 million, net loss narrows to $92 million
Ultragenyx Pharmaceutical reported second-quarter revenue of US$214 million, up from US$167 million a year earlier, while its quarterly net loss narrowed to US$92 million from US$115 million. Basic loss per share from continuing operations improved to US$0.90 from US$1.17. For the first half, revenue reached US$350 million versus US$306 million, though the six-month net loss widened to US$277 million from US$266 million. The FDA has accepted the UX111 BLA with a PDUFA date of September 19, 2026, a key near-term catalyst.
Ultragenyx sets August 4 call for second quarter 2026 results
Ultragenyx Pharmaceutical will host a conference call at 5:00 p.m. Eastern Time on Tuesday, August 4, 2026, to discuss its financial results and corporate update for the quarter ending June 30, 2026. The live and replayed webcast will be available through the company’s website, with the replay accessible for three months.
Ultragenyx grants 65,886 restricted stock units to 39 new non-executive hires
Ultragenyx Pharmaceutical granted 65,886 restricted stock units to 39 newly hired non-executive officers under its Employment Inducement Plan. The awards, approved by the compensation committee with a grant date of July 16, 2026, vest over four years with 25% of the underlying shares vesting on each anniversary of the grant date, subject to continued employment. The grants were made as an inducement material to the new employees entering into employment in accordance with Nasdaq Listing Rule 5635(c)(4).
Johnson Fistel Investigates Ultragenyx Officers Over Setrusumab Disclosures
Johnson Fistel, PLLP is investigating potential claims on behalf of long-term shareholders of Ultragenyx Pharmaceutical Inc. against certain officers and directors for alleged breaches of fiduciary duty. Shareholders who have held Ultragenyx shares continuously since prior to August 3, 2023, may have standing to seek corporate governance reforms, the return of funds, and a court-approved incentive award at no cost. The investigation follows a previously filed securities class action alleging that Ultragenyx and certain executives made materially false and misleading statements about setrusumab and the Phase III ORBIT study in patients with osteogenesis imperfecta, creating a false impression of reliable information while minimizing the risk that the study would fail to achieve a statistically significant reduction in annualized fracture rate. The complaint further alleges that the company's optimism was misplaced because the threshold figures were based on Phase II results lacking a placebo control group.
Ultragenyx shares surged 5.2% in the last trading session to close at $30.77, driven by higher-than-usual trading volume. The stock has gained 25.8% over the past four weeks, fueled by investor optimism around its growing portfolio of marketed rare disease therapies and a promising late-stage pipeline. Key products Crysvita, Dojolvi, and Mepsevii continue to drive revenue growth, while upcoming catalysts include an FDA decision on DTX401 for glycogen storage disease type Ia expected in August 2026, positive late-stage data for DTX301 in OTC deficiency, and readouts for UX701 in Wilson disease and GTX-102 in Angelman syndrome in 2026. The company is expected to report a quarterly loss of $1.25 per share on revenues of $185.77 million. Analysts have kept consensus estimates unchanged over the past 30 days, and the stock carries a Zacks Rank of 3, or Hold.