Ucb S A Stock Price, News & Analysis

UCB (OTC:UCBJY) announced positive Phase 3 study results for fenfluramine in treating CDKL5 deficiency disorder (CDD), an ultra-rare developmental and epileptic encephalopathy. The study met its primary endpoint of reducing countable motor seizure frequency and most key secondary endpoints.

The randomized, double-blind trial involved 87 patients aged 1-35 with CDD diagnosis and uncontrolled seizures. CDD affects approximately 1 in 40,000 to 60,000 live births. The drug demonstrated a safety profile consistent with previous studies in Dravet syndrome and Lennox-Gastaut syndrome. UCB plans to pursue regulatory approval for this potential new treatment option.

UCB announced its participation in the UMDF Mitochondrial Medicine 2025 Conference, presenting three key research posters on thymidine kinase 2 deficiency (TK2d). The presentations include findings from disease course studies in untreated patients, examining progression and outcomes over time, as well as insights into caregiver burden from the Assessment of TK2d Patient Perspectives study. The research focuses on both pediatric (≤12 years) and adult (>12 years) onset patients, representing data from the largest international TK2d dataset. Dr. Kimberly A. Moran, SVP & Head of US Rare Diseases at UCB, emphasized the company's commitment to advancing understanding of this rare mitochondrial disease, which has historically lacked research attention.

UCB has awarded 15 scholarships to individuals affected by myasthenia gravis (MG) in the U.S. through its inaugural 2025 UCB U.S. Myasthenia Gravis Scholarship program. The recipients include both people living with MG and caregivers, with one-third being high school seniors and others pursuing undergraduate, graduate degrees, or certification programs. The selection committee comprised medical professionals and people impacted by MG. Kim Moran, SVP & Head of U.S. Rare Diseases at UCB, announced that the scholarship program will continue into 2026. The initiative demonstrates UCB's commitment to supporting the rare disease community beyond medical treatment by helping recipients manage educational costs and advance their career goals.

UCB (UCBJY) has announced plans to build a new state-of-the-art biologics manufacturing facility in the United States, representing a major expansion of its U.S. presence. The investment is expected to generate an economic impact of approximately $5 billion and create 300 permanent high-skilled jobs plus 500 construction positions. Since 2017, UCB has grown its U.S. workforce by 73% to 2,000 employees and invested $4.5 billion in acquisitions and capital improvements. The company has achieved 15 FDA approvals or indication expansions, with 8 occurring in the past two years. The new facility aims to serve UCB's growing U.S. patient base while strengthening its global supply chain. The company is currently conducting a feasibility study to determine the optimal location, focusing on areas with strong talent pipelines and innovation ecosystems.

UCB announced compelling three-year data for BIMZELX (bimekizumab-bkzx) in treating psoriatic arthritis (PsA) and axial spondyloarthritis (axSpA) at EULAR 2025. The data showed sustained efficacy across multiple endpoints. In PsA patients, ACR50 response was maintained at 53.2% for biologic-naive and 55.2% for TNFi-experienced patients. For axSpA, ASAS40 responses were sustained at 60.4% for nr-axSpA and 60.1% for AS patients. The drug demonstrated consistent control of inflammation across all patient groups, with nearly 60% achieving elimination of swollen joints in PsA and similar proportions reaching low disease activity in axSpA. BIMZELX, as the first dual inhibitor of IL-17A and IL-17F, maintained a favorable safety profile with no new safety signals over three years.

UCB (UCBJY) and Domino Data Lab have announced a strategic collaboration to modernize Statistical Computing Environment (SCE) for life sciences. The partnership aims to create a unified cloud-enabled platform that integrates analytical tools like SAS, R, and Python while maintaining compliance with GxP, FDA 21 CFR Part 11, and GDPR standards.

The modernized SCE will enhance research capabilities by embedding critical metadata and workflow management for searchable data re-use, supporting UCB's scientific innovation strategy. The platform will combine Domino's cloud expertise with UCB's clinical research knowledge to accelerate drug development and enable more efficient clinical studies through advanced data analysis.

UCB (UCBJY) announced the presentation of 24 scientific abstracts at the American Academy of Neurology (AAN) meeting in San Diego, April 5-9, 2025. The research focuses on treatments for rare epilepsies (Dravet syndrome and Lennox-Gastaut syndrome), generalized myasthenia gravis (gMG), and thymidine kinase 2 deficiency (TK2d).

Key presentations include:

• Final long-term safety data for fenfluramine (FINTEPLA) in Lennox-Gastaut syndrome
• Results from rozanolixizumab (RYSTIGGO) self-administration study in gMG
• Zilucoplan (ZILBRYSQ) Phase 3 study outcomes in gMG
• Disease course data from the largest international TK2d dataset
• BRIVIACT long-term clinical outcomes in pediatric patients
• Investigation of STACCATO alprazolam for seizure management

The company will also host its inaugural US Rare Disease Connect in Neurology Annual Summit and two symposia focusing on epileptic encephalopathy and seizure emergencies.

UCB presented positive clinical data for its investigational therapy doxecitine (dC) and doxribtimine (dT) for treating thymidine kinase 2 deficiency (TK2d) at the MDA Conference 2025. The data demonstrated significant benefits in survival and motor function improvements.

Key findings show that in patients with symptom onset ≤12 years, treatment reduced death risk by 92-94% from symptom onset and 87-95% from treatment start. Additionally, 75% of patients regained at least one lost motor milestone, with 22.5% regaining four or more. Ventilatory support needs decreased, with 16.1% reducing usage and another 16.1% discontinuing support entirely.

The therapy was generally well-tolerated, with diarrhea (84.6%-90.9%) being the most common side effect. The treatment is currently under review by US FDA and EMA regulators.

UCB announced the publication of final analysis results from a long-term open-label extension study of FINTEPLA® (fenfluramine) in treating Dravet syndrome (DS) patients. The study demonstrated that FINTEPLA was generally well-tolerated with significant efficacy in reducing seizures.

Key findings include:

• 66.8% median reduction in monthly convulsive seizure frequency from baseline to end of study
• 64.2% of patients achieved ≥50% reduction in seizure frequency
• 20.3% median increase in seizure-free days
• Only 2.9% of patients discontinued due to adverse events

Common side effects included pyrexia, nasopharyngitis, and decreased appetite. Notably, no valvular heart disease or pulmonary arterial hypertension was observed. The study included both children and adults, with patients exposed to FINTEPLA for up to 3.5 years. Caregivers and investigators rated approximately 62% of patients as much/very much improved.