Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) experienced a pronounced decrease in its share price during Monday's market session, falling roughly 42% to close near $19.84. This sharp decline followed the announcement of Phase 3 clinical trial outcomes involving setrusumab (UX143), a therapeutic candidate aimed at treating Osteogenesis Imperfecta (OI), a rare genetic disorder characterized by fragile bones prone to fractures.
Alongside Mereo BioPharma, Ultragenyx disclosed the results from two Phase 3 trials known as the ORBIT and COSMIC studies. These trials evaluated the efficacy of setrusumab in reducing fracture rates among patients with Osteogenesis Imperfecta. Unfortunately, neither study met its pre-defined primary endpoints: statistical significance in reducing the annualized clinical fracture rate compared to placebo or bisphosphonate treatments. Mereo BioPharma Group plc also saw its shares plummet by 90%, down to $0.23, reflecting investor response to the disappointing trial data.
Osteogenesis Imperfecta encompasses several genetic disorders affecting collagen production, resulting in brittle bones that significantly increase fracture susceptibility. The failure of setrusumab to demonstrate meaningful clinical benefit in these pivotal studies constitutes a setback for both companies' ambitions in this therapeutic area.
Market Highlights: Noteworthy Gainers
Despite challenges for Ultragenyx and Mereo BioPharma, a range of other equities showed substantial upside amid the mid-day trading session. Republic Power Group Ltd (NASDAQ: RPGL) surged by 163%, reaching $0.69. Brand Engagement Network Inc (NASDAQ: BNAI) climbed 84.3% to $2.21, buoyed by the announcement of a Vendor Services Project Agreement with a prominent global advertising and communications firm.
Additional notable gainers included:
- Globavend Holdings Ltd (NASDAQ: GVH) +58.5% to $4.12
- CCSC Technology International Holdings Ltd (NASDAQ: CCTG) +57.3% to $0.20
- 3 E Network Technology Group Ltd (NASDAQ: MASK) +34.8% to $0.25
- Eightco Holdings Inc (NASDAQ: ORBS) +29.1% to $2.15; confidence bolstered by a newly announced $125 million share repurchase program
- Greenwich LifeSciences Inc (NASDAQ: GLSI) +25.7% to $26.00 following an extension of the lock-up period until September 30, 2026
- TROOPS Inc (NASDAQ: TROO) +23.3% to $2.38
- Fitell Corp (NASDAQ: FTEL) +21.2% to $0.88
- TryHard Holdings Ltd (NASDAQ: THH) +19.2% to $20.19
- Palisade Bio Inc (NASDAQ: PALI) +18.6% to $2.55; Piper Sandler analyst Yasmeen Rahimi initiated coverage with an Overweight rating and a $25 price target
- Regencell Bioscience Holdings Ltd (NASDAQ: RGC) +16.3% to $24.60
- Praxis Precision Medicines Inc (NASDAQ: PRAX) +13% to $304.00 after the FDA conferred Breakthrough Therapy Designation for its experimental compound ulixacaltamide
- Zura Bio Ltd (NASDAQ: ZURA) +13% to $5.26
- DigitalBridge Group Inc (NYSE: DBRG) +9.9% to $15.30 in response to a SoftBank Group acquisition announcement valued at approximately $4 billion
- Precigen Inc (NASDAQ: PGEN) +8.6% to $4.52
- Autolus Therapeutics PLC (NASDAQ: AUTL) +7.8% to $1.80 following a maintained Buy rating and price target increase from $10 to $11 by Needham analyst Gil Blum
- LightPath Technologies Inc (NASDAQ: LPTH) +6.6% to $9.62
Stocks Facing Declines
Several other companies recorded significant share price decreases in conjunction with adverse news or broader sector weakness. Beyond Ultragenyx and Mereo, shares in X3 Holdings Co Ltd (NASDAQ: XTKG) dropped 42.1% to $0.080. ECD Automotive Design Inc (NASDAQ: ECDA) fell 38.3% to $0.71, while CID HoldCo Inc (NASDAQ: DAIC) decreased by 34.6% to $0.41.
Carbon Revolution PLC (NASDAQ: CREV) dipped nearly 28% to $1.82, and Northann Corp (NYSE: NCL) declined 26.5% to $0.23. Security Matters PLC (NASDAQ: SMX) also registered a 26.5% loss, closing at $85.60.
Inovio Pharmaceuticals Inc (NASDAQ: INO) shed 22.3%, closing at $1.78. This followed FDA acceptance of its Biologics License Application (BLA) for INO-3107, though regulators expressed reservations concerning the accelerated approval pathway.
Additional losers included:
- 374Water Inc (NASDAQ: SCWO) -19.6% to $1.90, even as the company added Charles Weiser to its Board of Directors
- Hycroft Mining Holding Corporation (NASDAQ: HYMC) -14% to $21.61, reflective of retreating precious metal prices
- Coincheck Group NV (NASDAQ: CNCK) -11.5% to $3.08
- CapsoVision Inc (NASDAQ: CV) -11.2% to $11.55
- Americas Gold and Silver Corporation (NYSE: USAS) -11% to $5.48, impacted by softer precious metal commodity prices
- Uxin Ltd (NASDAQ: UXIN) -10.8% to $3.41 despite reporting strong quarterly revenue, vehicle sales, and income metrics, alongside a significant equity raise
Market Context and Sector Notes
Precious metal stocks showed broad weakness with declines across multiple companies. The pullback followed a period where silver, in particular, reached record highs but then reversed course.
Overall, the market displayed considerable volatility, with biotechnology firms particularly responsive to clinical trial data and regulatory developments. Shareholder confidence was notably variable, as illustrated by divergences between companies announcing growth initiatives or regulatory designations versus those confronting negative trial outcomes.
This trading activity underscores the sensitivity of biotechnology equities to clinical advancement news, regulatory decisions, and strategic corporate actions such as share repurchase programs and partnership agreements.