Ultragenyx

Andelyn Biosciences announced it is now manufacturing FAYUVI, Ultragenyx Pharmaceutical Inc.'s FDA-approved gene therapy for Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA, for commercial supply at its Columbus, Ohio facility. The move follows the U.S. Food and Drug Administration's approval of FAYUVI, making it the first FDA-approved gene therapy manufactured using the Andelyn AAV Curator Platform process. Sanfilippo syndrome type A is a rare, fatal lysosomal storage disease that primarily affects the central nervous system and is marked by rapid neurodegeneration beginning in early childhood; it is estimated to affect 3,000 to 5,000 patients worldwide, with a median life expectancy of 15 years. Andelyn Chief Executive Officer Wade Macedone said the company is proud to manufacture an FDA-approved gene therapy for commercial use using an AAV Curator Platform process, calling the milestone a reflection of the purpose behind Andelyn's founding. Andelyn, a full-service FDA-inspected cell and gene therapy commercial CDMO with more than 20 years of experience, has produced clinical and commercial material for more than 500 cGMP batches and 85 global clinical trials.

The FDA has granted full approval to Ultragenyx's Fayuvi, also known as UX111, for treating the neurologic manifestations of mucopolysaccharidosis type IIIA, or Sanfilippo syndrome Type A, in pediatric patients with preserved neurodevelopmental function. The approval makes Fayuvi the first FDA-approved treatment for this rare and fatal neurodegenerative disorder and marks Ultragenyx's second gene therapy approval and sixth FDA approval overall. The decision was supported by nearly eight years of clinical data, including a 23.5-point higher mean change in Bayley-III Cognitive raw score versus an untreated natural-history cohort. Ultragenyx shares ended the last trading session 12.6% higher at $14.5 on above-average volume, though the stock has lost 50.9% over the past four weeks. The company is expected to post a quarterly loss of $0.65 per share, a year-over-year change of +64.1%, on revenues of $185.2 million, up 15.8% from the year-ago quarter, with its consensus EPS estimate revised 6.4% higher over the last 30 days.

Ultragenyx Pharmaceutical Inc. announced the publication of 96-week data from its Phase 3 study of GENGLYCOS AAV gene therapy for glycogen storage disease type Ia in The Journal of Inherited Metabolic Disease, showing that participants achieved a mean reduction in daily cornstarch intake of 61% while maintaining glycemic control. The study met its primary endpoint at Week 48, with DTX401-treated patients experiencing a 41% reduction in cornstarch compared to 10% for placebo, and by Week 96, 67% of participants in both the original and crossover groups eliminated at least one nighttime cornstarch dose, with 33% and 42% respectively eliminating nighttime dosing entirely. Patient-reported outcomes showed that 83% of DTX401-treated participants met or exceeded their own expectations for meaningful cornstarch reduction at Week 48, and the therapy was generally well tolerated with an acceptable safety profile. GENGLYCOS was recently approved by the FDA for patients ages eight and older with 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.

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.

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 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 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.