TK2

24 scientific abstracts reflect UCB's ongoing commitment to improving outcomes for people living with neurological conditions, including rare epilepsies Dravet syndrome and Lennox-Gastaut syndrome, as well as generalized myasthenia gravis and thymidine kinase 2 deficiency. Research includes disease course data of untreated patients with thymidine kinase 2 deficiency from largest international dataset. Also features new data on generalized myasthenia gravis treatments RYSTIGGO®1 (rozanolixizumab-noli) and ZILBRYSQ®2 (zilucoplan) including an open-label extension study investigating rozanolixizumab self-administration and a phase 3 study on the effect of zilucoplan on specific outcome scores. Data on FINTEPLA®3 (fenfluramine), BRIVIACT®4 (brivaracetam) CV, and investigational therapy STACCATO®* alprazolam showcase commitment to people living with epilepsies and their unmet needs. BRUSSELS, April 3, 2025 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced it will present 24 abstracts from its expansive neurology portfolio, regarding rare epilepsies Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), generalized myasthenia gravis (gMG), and thymidine kinase 2 deficiency (TK2d), at this year's American Academy of Neurology (AAN) meeting, San Diego, California, April 5-9, 2025. "We at UCB are deeply committed to driving innovation to improve the treatment and care of people living with severe neurological and neuromuscular conditions such as epilepsy, gMG, and TK2d. The data presented at AAN is a testament to our dedication to exploring new frontiers of science and improving care in areas of high unmet need. It is our mission to address critical gaps in care and bring meaningful solutions that make real improvements in the lives of the people we serve, now and into the future," said Donatello Crocetta, Chief Medical Officer at UCB. Highlights of data to be presented at AAN include: Epilepsy DS and LGS: the final long-term safety and effectiveness data from a fenfluramine (FFA) open-label extension (OLE) study of 247 participants with LGS6, data from an observational analysis assessing sleep apnea association with increased mortality in patients 1-17 years old with severe epilepsy7, and data from a Phase 1 study to assess the safety, tolerability, pharmacokinetics, and efficacy of fenfluramine when combined with cannabidiol in a small cohort of patients with DS or LGS.8 Focal onset seizures: data include brivaracetam long-term clinical outcomes in pediatric patients with primary generalized seizures* from an open-label Phase 3 follow-up study9 and healthcare resource utilization of brivaracetam monotherapy† from a claims analysis.10 Women of childbearing age: data include results of a social media listening analysis on the experiences and challenges of women living with epilepsy during their motherhood journey.11 Quality of life: including data from a survey evaluating the disruptive impact of developmental and epileptic encephalopathies on patients' and families' quality of life12 and data from a study exploring the impact of prolonged seizures on patients' and caregivers' quality of life.13 UCB pipeline: data include investigational therapy STACCATO alprazolam*, being studied in management of stereotypical prolonged seizures.5,14 gMG Rozanolixizumab self-administration study: Phase 3, open-label, randomized study which observed the safety and efficacy of manual push (MP) and syringe driver (SD) self-administration of rozanolixizumab was consistent with known profile.15 Ocular symptoms study in gMG: results from post hoc analyses from the randomized, placebo-controlled, Phase 3 MycarinG study investigating the effect of rozanolixizumab on ocular symptoms in patients with gMG.16 Study investigating switching to zilucoplan: Phase 3b study on safety, efficacy, and patient preference and satisfaction for subcutaneous zilucoplan in myasthenia gravis after switching from intravenous complement component 5 inhibitors.17 MG-specific subdomain scores: results from analysis from the randomized, placebo-controlled, Phase 3 RAISE study investigating the effect of zilucoplan on MG-ADL and QMG subdomain scores.18 Quality of life with gMG: a new international patient registry in gMG linking clinical and patient-reported outcomes data aims to improve understanding of the symptoms and quality of life to optimize future disease management.19 TK2d Disease course: findings from the largest international TK2d dataset evaluating the disease course of people living with TK2d who were ≤12 years at TK2d symptom onset20 and over 12 at TK2d symptom onset and not on treatment.21 * STACCATO® alprazolam is an investigational treatment, and its safety and efficacy has not been established. It is not currently approved for use by any regulatory authority worldwide. Rare Disease Connect in Neurology (RDCN) UCB is proud to host its inaugural US Rare Disease Connect in Neurology (RDCN) Annual Summit at AAN, to provide a forum for needs-driven medical education for the gMG community. This event is being held exclusively for healthcare providers who are involved in patient care. Symposia AAN 2025 will feature two UCB-supported symposia: Earlier identification and treatment of patients with a developmental and epileptic encephalopathy: taking a close look at Lennox-Gastaut syndrome: Saturday, April 5, 11:45 am-12:45 pm. Rethinking seizure emergencies: expert perspectives on a new paradigm: Monday, April 7, 6 pm. For further information, contact UCB: Global Communications Anna Clark T: +44.73.8.668.67.79 email: [email protected] Corporate Communications, Media Relations Laurent Schots T: +32.2.559.92.64 email: [email protected] Rare Disease Communications Daphne Teo T: +1 (770) 880-7655 email: [email protected] Epilepsy and Rare Syndromes Communications Becky Malone T: +1 (919) 605-9600 email: [email protected] Investor Relations Antje Witte T: +32.2.559.94.14 email: [email protected] About UCB UCB, Brussels, Belgium () is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9 000 people in approximately 40 countries, the company generated revenue of € 6.15 billion in 2024. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news. 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Important Safety Information about BRIVIACT (brivaracetam) in the US4 Indication BRIVIACT® (brivaracetam) CV is indicated for the treatment of partial-onset seizures in patients 1 month of age and older.4 IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Suicidal Behavior and Ideation: Antiepileptic drugs, including BRIVIACT, increase the risk of suicidal behavior and ideation. Monitor patients taking BRIVIACT for the emergence or worsening of depression; unusual changes in mood or behavior; or suicidal thoughts, behavior, or self-harm. Advise patients, their caregivers, and/or families to be alert for these behavioral changes and report them immediately to a healthcare provider. Neurological Adverse Reactions: BRIVIACT causes somnolence, fatigue, dizziness, and disturbance in coordination. Monitor patients for these signs and symptoms and advise them not to drive or operate machinery until they have gained sufficient experience on BRIVIACT. Psychiatric Adverse Reactions: BRIVIACT causes psychiatric adverse reactions, including non-psychotic and psychotic symptoms in adult and pediatric patients. Advise patients to report these symptoms immediately to a healthcare provider. Hypersensitivity: BRIVIACT can cause hypersensitivity reactions. Bronchospasm and angioedema have been reported. Discontinue BRIVIACT if a patient develops a hypersensitivity reaction after treatment. BRIVIACT is contraindicated in patients with a prior hypersensitivity reaction to brivaracetam or any of the inactive ingredients. Withdrawal of Antiepileptic Drugs: As with all antiepileptic drugs, BRIVIACT should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus. DOSING CONSIDERATIONS Dose adjustments are recommended for patients with all stages of hepatic impairment. When BRIVIACT is co-administered with rifampin, an increase in the BRIVIACT dose is recommended. ADVERSE REACTIONS In adult adjunctive therapy placebo-controlled clinical trials, the most common adverse reactions (at least 5% for BRIVIACT and at least 2% more frequently than placebo) were somnolence and sedation, dizziness, fatigue, and nausea and vomiting symptoms. Adverse reactions reported in clinical studies of pediatric patients were generally similar to those in adult patients. Adverse reactions with BRIVIACT injection in adult and pediatric patients were generally similar to those observed with BRIVIACT tablets. Other adverse events that occurred in adult patients who received BRIVIACT injection included dysgeusia, euphoric mood, feeling drunk, and infusion site pain. BRIVIACT is a Schedule V controlled substance. Please refer to the full Prescribing Information. Important Safety Information about FINTEPLA (fenfluramine) in the US3 INDICATIONS FINTEPLA is indicated for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients 2 years of age and older.3 IMPORTANT SAFETY INFORMATION BOXED WARNING: VALVULAR HEART DISEASE and PULMONARY ARTERIAL HYPERTENSION There is an association between serotonergic drugs with 5-HT2B receptor agonist activity, including fenfluramine (the active ingredient in FINTEPLA), and valvular heart disease and pulmonary arterial hypertension. Echocardiogram assessments are required before, during, and after treatment with FINTEPLA. FINTEPLA is available only through a restricted program called the FINTEPLA REMS. CONTRAINDICATIONS FINTEPLA is contraindicated in patients with hypersensitivity to fenfluramine or any of the excipients in FINTEPLA and with concomitant use, or within 14 days of the administration, of monoamine oxidase inhibitors because of an increased risk of serotonin syndrome. WARNINGS AND PRECAUTIONS Valvular Heart Disease and Pulmonary Arterial Hypertension (see Boxed Warning): Because of the association between serotonergic drugs with 5–HT2B receptor agonist activity, including fenfluramine (the active ingredient in FINTEPLA), and valvular heart disease (VHD) and pulmonary arterial hypertension (PAH), cardiac monitoring via echocardiogram is required prior to starting treatment, during treatment, and after treatment with FINTEPLA concludes. Cardiac monitoring via echocardiogram can aid in early detection of these conditions. In clinical trials for DS and LGS of up to 3 years in duration, no patient receiving FINTEPLA developed VHD or PAH. Monitoring: Prior to starting treatment, patients must undergo an echocardiogram to evaluate for VHD and PAH. Echocardiograms should be repeated every 6 months, and once at 3-6 months post treatment with FINTEPLA. The prescriber must consider the benefits versus the risks of initiating or continuing treatment with FINTEPLA if any of the following signs are observed via echocardiogram: valvular abnormality or new abnormality; VHD indicated by mild or greater aortic regurgitation or moderate or greater mitral regurgitation, with additional characteristics of VHD (eg, valve thickening or restrictive valve motion); PAH indicated by elevated right heart/pulmonary artery pressure (PASP >35 mmHg). FINTEPLA REMS Program (see Boxed Warning): FINTEPLA is available only through a restricted distribution program called the FINTEPLA Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified by enrolling in the FINTEPLA REMS. Prescribers must counsel patients receiving FINTEPLA about the risk of VHD and PAH, how to recognize signs and symptoms of VHD and PAH, the need for baseline (pretreatment) and periodic cardiac monitoring via echocardiogram during FINTEPLA treatment, and cardiac monitoring after FINTEPLA treatment. Patients must enroll in the FINTEPLA REMS and comply with ongoing monitoring requirements. The pharmacy must be certified by enrolling in the FINTEPLA REMS and must only dispense to patients who are authorized to receive FINTEPLA. Wholesalers and distributors must only distribute to certified pharmacies. Further information is available at or by telephone at 1-877-964-3649. Decreased Appetite and Decreased Weight: FINTEPLA can cause decreases in appetite and weight. Decreases in weight appear to be dose related. Approximately half of the patients with LGS and most patients with DS resumed the expected measured increases in weight during the open-label extension studies. Weight should be monitored regularly during treatment with FINTEPLA, and dose modifications should be considered if a decrease in weight is observed. Somnolence, Sedation, and Lethargy: FINTEPLA can cause somnolence, sedation, and lethargy. Other central nervous system (CNS) depressants, including alcohol, could potentiate these effects of FINTEPLA. Prescribers should monitor patients for somnolence and sedation and should advise patients not to drive or operate machinery until they have gained sufficient experience on FINTEPLA to gauge whether it adversely affects their ability to drive or operate machinery. Suicidal Behavior and Ideation: Antiepileptic drugs (AEDs), including FINTEPLA, increase the risk of suicidal thoughts or behaviors in patients taking these drugs for any indication. Patients treated with an AED for any indication should be monitored for the emergence or worsening of depression, suicidal thoughts or behaviors, or any unusual changes in mood or behavior. Anyone considering prescribing FINTEPLA or any other AED must balance the risk of suicidal thoughts or behaviors with the risks of untreated illness. Epilepsy and many other illnesses for which AEDs are prescribed are themselves associated with morbidity and mortality and an increased risk of suicidal thoughts and behaviors. Should suicidal thoughts and behaviors emerge during treatment, consider whether the emergence of these symptoms in any given patient may be related to the illness being treated. Withdrawal of Antiepileptic Drugs: As with most AEDs, FINTEPLA should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus. If withdrawal is needed because of a serious adverse reaction, rapid discontinuation can be considered. Serotonin Syndrome: Serotonin syndrome, a potentially life-threatening condition, may occur with FINTEPLA, particularly during concomitant administration of FINTEPLA with other serotonergic drugs, including, but not limited to, selective serotonin-norepinephrine reuptake inhibitors (SNRIs), selective serotonin reuptake inhibitors (SSRIs), tricyclic antidepressants (TCAs), bupropion, triptans, dietary supplements (eg, St. John's Wort, tryptophan), drugs that impair metabolism of serotonin (including monoamine oxidase inhibitors [MAOIs], which are contraindicated with FINTEPLA), dextromethorphan, lithium, tramadol, and antipsychotics with serotonergic agonist activity. Patients should be monitored for the emergence of signs and symptoms of serotonin syndrome, which include mental status changes (eg, agitation, hallucinations, coma), autonomic instability (eg, tachycardia, labile blood pressure, hyperthermia), neuromuscular signs (eg, hyperreflexia, incoordination), and/or gastrointestinal symptoms (eg, nausea, vomiting, diarrhea). If serotonin syndrome is suspected, treatment with FINTEPLA should be stopped immediately and symptomatic treatment should be started. Increase in Blood Pressure : FINTEPLA can cause an increase in blood pressure. Rare cases of significant elevation in blood pressure, including hypertensive crisis, has been reported in adult patients treated with fenfluramine, including patients without a history of hypertension. In clinical trials for DS and LGS of up to 3 years in duration, no pediatric or adult patient receiving FINTEPLA developed hypertensive crisis. Monitor blood pressure in patients treated with FINTEPLA. Glaucoma: Fenfluramine can cause mydriasis and can precipitate angle closure glaucoma. Consider discontinuing treatment with FINTEPLA in patients with acute decreases in visual acuity or ocular pain. ADVERSE REACTIONS The most common adverse reactions observed in DS studies (incidence at least 10% and greater than placebo) were decreased appetite; somnolence, sedation, lethargy; diarrhea; constipation; abnormal echocardiogram; fatigue, malaise, asthenia; ataxia, balance disorder, gait disturbance; blood pressure increased; drooling, salivary hypersecretion; pyrexia; upper respiratory tract infection; vomiting; decreased weight; fall; status epilepticus. The most common adverse reactions observed in the LGS study (incidence at least 10% and greater than placebo) were diarrhea; decreased appetite; fatigue; somnolence; vomiting. DRUG INTERACTIONS Strong CYP1A2, CYP2B6, or CYP3A Inducers: Coadministration with strong CYP1A2, CYP2B6, or CYP3A inducers will decrease fenfluramine plasma concentrations. If coadministration of a strong CYP1A2, CYP2B6, or CYP3A inducer with FINTEPLA is necessary, monitor the patient for reduced efficacy and consider increasing the dosage of FINTEPLA as needed. If a strong CYP1A2, CYP2B6, or CYP3A inducer is discontinued during maintenance treatment with FINTEPLA, consider gradual reduction in the FINTEPLA dosage to the dose administered prior to initiating the inducer. Strong CYP1A2 or CYP2D6 Inhibitors: Coadministration with strong CYP1A2 or CYP2D6 inhibitors will increase fenfluramine plasma concentrations. If FINTEPLA is coadministered with strong CYP1A2 or CYP2D6 inhibitors, the maximum daily dosage of FINTEPLA is 20 mg. If a strong CYP1A2 or CYP2D6 inhibitor is discontinued during maintenance treatment with FINTEPLA, consider gradual increase in the FINTEPLA dosage to the dose recommended without CYP1A2 or CYP2D6 inhibitors. If FINTEPLA is coadministered with stiripentol and a strong CYP1A2 or CYP2D6 inhibitor, the maximum daily dosage of FINTEPLA is 17 mg. USE IN SPECIFIC POPULATIONS In patients with severe impairment of kidney function (estimated glomerular filtration rate [eGFR]) 15 to 29 mL/min/1.73m2, dosage adjustments are recommended. FINTEPLA has not been studied in patients with kidney failure (eGFR <15 mL/min/1.73m2). Combined molar exposures of fenfluramine and norfenfluramine were increased in subjects with various degrees of hepatic impairment (Child-Pugh Class A, B, and C), necessitating a dosage adjustment in these patients. To report SUSPECTED ADVERSE REACTIONS, contact UCB, Inc. at 1–844-599-2273 or FDA at 1-800-FDA-1088 or . Please refer to the full Prescribing Information . Important Safety Information about RYSTIGGO (rozanolixizumab-noli) in the US1 INDICATION RYSTIGGO (rozanolixizumab-noli) is a neonatal Fc receptor blocker indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.1 IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Infections: RYSTIGGO may increase the risk of infection. Delay RYSTIGGO administration in patients with an active infection until the infection is resolved. During treatment with RYSTIGGO, monitor for clinical signs and symptoms of infection. If serious infection occurs, administer appropriate treatment and consider withholding RYSTIGGO until the infection has resolved. Immunization Immunization with vaccines during RYSTIGGO treatment has not been studied. The safety of immunization with live or live-attenuated vaccines and the response to immunization with any vaccine are unknown. Because RYSTIGGO causes a reduction in IgG levels, vaccination with live-attenuated or live vaccines is not recommended during treatment with RYSTIGGO. Evaluate the need to administer age-appropriate vaccines according to immunization guidelines before initiation of a new treatment cycle with RYSTIGGO. Aseptic Meningitis: Serious adverse reactions of aseptic meningitis (also called drug-induced aseptic meningitis) have been reported in patients treated with RYSTIGGO. If symptoms consistent with aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care. Hypersensitivity Reactions: Hypersensitivity reactions, including angioedema and rash, were observed in patients treated with RYSTIGGO. Management of hypersensitivity reactions depends on the type and severity of the reaction. Monitor patients during treatment with RYSTIGGO and for 15 minutes after for clinical signs and symptoms of hypersensitivity reactions. If a reaction occurs, institute appropriate measures if needed. ADVERSE REACTIONS In a placebo-controlled study, the most common adverse reactions (reported in at least 10% of RYSTIGGO-treated patients) were headache, infections, diarrhea, pyrexia, hypersensitivity reactions, and nausea. Serious infections were reported in 4% of patients treated with RYSTIGGO. Three fatal cases of pneumonia were identified, caused by COVID-19 infection in two patients and an unknown pathogen in one patient. Six cases of infections led to discontinuation of RYSTIGGO. Please refer to the full Prescribing Information. Important Safety Information about ZILBRYSQ (zilucoplan) in the US2 INDICATION ZILBRYSQ is a complement inhibitor indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.2 IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING WARNING: SERIOUS MENINGOCOCCAL INFECTIONS ZILBRYSQ, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis. Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update meningococcal vaccination (for serogroups A, C, W, and Y, and B) at least 2 weeks prior to the first dose of ZILBRYSQ, unless the risk of delaying therapy outweigh the risks of developing a meningococcal infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccination against meningococcal bacteria in patients receiving a complement inhibitor. Patients receiving ZILBRYSQ are at increased risk for invasive disease caused by Neisseria meningitidis, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious meningococcal infections and evaluate immediately if infection is suspected. Because of the risk of serious meningococcal infections, ZILBRYSQ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called ZILBRYSQ REMS. CONTRAINDICATIONS ZILBRYSQ is contraindicated for initiation in patients with unresolved Neisseria meningitidis infection. WARNINGS AND PRECAUTIONS Serious Meningococcal Infections ZILBRYSQ, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by meningococcal bacteria (septicemia and/or meningitis) in any serogroup, including non-groupable strains. Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of ZILBRYSQ treatment is contraindicated in patients with unresolved serious Neisseria meningitidis infection. Complete or update meningococcal vaccination (for serogroups A, C, W, Y and B) at least 2 weeks prior to administration of the first dose of ZILBRYSQ, according to current ACIP recommendations for patients receiving a complement inhibitor. If urgent ZILBRYSQ therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer meningococcal vaccines as soon as possible. Closely monitor patients for early signs and symptoms of meningococcal infection and evaluate patients immediately if infection is suspected. Consider interruption of ZILBRYSQ in patients who are undergoing treatment for serious meningococcal infection, depending on the risks of interrupting treatment in the disease being treated. ZILBRYSQ REMS Due to the risk of meningococcal infections, ZILBRYSQ is available only through a restricted program under a REMS called ZILBRYSQ REMS. Under the ZILBRYSQ REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risk of serious meningococcal infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated with meningococcal vaccines. Additional information on the REMS requirements is available at or 1-877-414-8353. Other Infections Serious infections with Neisseria species (other than Neisseria meningitidis), including disseminated gonococcal infections, have been reported in patients treated with complement inhibitors. ZILBRYSQ blocks terminal complement activation; therefore, patients may have increased susceptibility to infections, especially with encapsulated bacteria, such as infections caused by Neisseria meningitidis but also Streptococcus pneumoniae, Haemophilus influenzae, and to a lesser extent, Neisseria gonorrhoeae. Administer vaccinations for the prevention of Streptococcus pneumoniae infection according to ACIP recommendations. Patients receiving ZILBRYSQ are at increased risk for infections due to these organisms, even if they develop antibodies following vaccination. Pancreatitis And Other Pancreatic Conditions Pancreatitis and pancreatic cysts have been reported in patients treated with ZILBRYSQ. Patients should be informed of this risk before starting ZILBRYSQ. Obtain lipase and amylase levels at baseline before starting treatment with ZILBRYSQ. Discontinue ZILBRYSQ in patients with suspected pancreatitis and initiate appropriate management until pancreatitis is ruled out or has resolved. ADVERSE REACTIONS In a placebo-controlled study, the most common adverse reactions (reported in at least 10% of gMG patients treated with ZILBRYSQ) were injection site reactions, upper respiratory tract infections, and diarrhea. Please refer to the full Prescribing Information . BRIVIACT®, FINTEPLA®, RYSTIGGO® and ZILBRYSQ® are registered trademarks of the UCB Group of Companies. STACCATO® is a registered trademark of Alexza Pharmaceuticals, Inc., and is used by UCB Pharma under license. ©2025 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-BR-2500017 1. Rystiggo® US PI. . Accessed February 2025. 2. Zilbrysq® US PI. Accessed February 2025. 3. Fintepla® US PI. . Accessed February 2025. 4. Briviact® US PI *1a42elk*_ga*NjA2OTM3NDAwLjE2ODI1ODQwNzc.*_ga_TXC8S80N6W*MTY5OTAyNTU2NS4yNy4xLjE2OTkwMjU1NzYuNDkuMC4w Accessed February 2025. 5. UCB acquires Engage Therapeutics: Staccato[®] Alprazolam - A potential solution for acute on-demand seizure management for people living with epilepsy. . Accessed February 2025. 6. Knupp, et al. Safety and Effectiveness of Fenfluramine for the Treatment of Seizures in Lennox-Gastaut Syndrome: Results From the Final Analysis of an Open-Label Extension Study. 7. Dedeurwaerdere, et al. Sleep Apnea is Associated with High Mortality Risk in Children with Severe Epilepsies: Observational Analysis from Large Scale US Claims Data. 8. Zhang Roper, et al. Safety, Tolerability, Pharmacokinetics, and Efficacy of Fenfluramine in Combination With Cannabidiol: Results From a Phase 1 Study. 9. Lagae, et al. Long-term Tolerability and Efficacy of Adjunctive Brivaracetam in Pediatric Patients With Primary Generalized Seizures: Subgroup Analysis of an Open-label Follow-up Trial. 10. Bessons, et al. Brivaracetam Monotherapy Patient Characteristics, Treatment Patterns, and Healthcare Resource Utilization Among Patients With Epilepsy: A Cohort Study Using US Claims Data. 11. Baker, et al. Experiences of Women of Childbearing Age with Epilepsy Throughout their Motherhood Journey: Results From a Social Media Listening Study. 12. Bailey, et al. Disruptive Impacts of Developmental and Epileptic Encephalopathies on Patient and Family Life: a Quality-of-Life Survey. 13. Kaye, et al. Impact of Prolonged Seizures on Patients' and Caregivers' Quality of Life. 14. Daniels, et al. Inhalation as an Efficient Delivery Route of Alprazolam for the Treatment of Acute Seizures: Randomized Study of Staccato® Alprazolam Compared to Oral Alprazolam. 15. Bril, et al. Self-Administration of Rozanolixizumab in Patients With Generalized Myasthenia Gravis: The MG0020 Study. 16. Mahuwala, et al. Effect of Rozanolixizumab on Ocular Symptoms in Patients With Generalized Myasthenia Gravis: A Post Hoc Item-Level Analysis of Myasthenia Gravis-Specific Outcomes in MycarinG. 17. Freimer, et al. Switching to Subcutaneous Zilucoplan From Intravenous Complement Component 5 Inhibitors in Myasthenia Gravis: Patient Preference and Satisfaction From a Phase 3b Study. 18. Weiss, et al. Effect of Zilucoplan on Myasthenia Gravis–Specific Outcome Subdomain Scores in RAISE: A Phase 3 Study. 19. Amini, et al. A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-Reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry. 20. Hirano, et al. The Disease Course of Untreated Patients with Thymidine Kinase 2 Deficiency (TK2d) Aged ≤12 Years at TK2d Symptom Onset: Findings from the Largest International TK2d Dataset. 21. Cristina Dominguez Gonzalez, et al. The Disease Course of Untreated Patients with Thymidine Kinase 2 Deficiency (TK2d) Aged >12 Years at TK2d Symptom Onset: Findings from the Largest International TK2d Dataset. 22. Nabbout, et al. Impact of Fenfluramine on Convulsive Seizure Frequency in Dose-Capped Patients With Dravet Syndrome. 23. Ameen, et al. Understanding the Incidence, Prevalence, Characteristics, and Healthcare Resource Utilization for Patients With Dravet and Lennox-Gastaut Syndromes. 24. Lhatoo, et al. Real-world Use of Fenfluramine for Dravet Syndrome: a Retrospective Cohort Study Using a National Pharmacy Database. 25. Bass, et al. Interim Results of the US Fenfluramine Oral Solution Cardiovascular Safety Registry Study. 26. Nabbout, et al. A Stratified Analysis of Efficacy and Safety of Fenfluramine in Patients With Dravet Syndrome. 27. Ameen, et al. A Retrospective Claims Study Evaluating Mortality in Patients with Lennox-Gastaut Syndrome or Dravet Syndrome in the United States. 28. Pascuzzi, et al. Correlation Between MG Symptoms PRO and Existing MG-Specific Outcome Scores in the Phase 3 MycarinG Study: Post Hoc Analysis. 29. Thompson, et al. Solutions to Address the Unmet Needs of the gMG Patient Journey in the US: A Multistakeholder Delphi Consensus Study (RWE0970). SOURCE UCB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical data highlight survival benefits and improvement in functional motor outcomes associated with treatment with doxecitine (dC) and doxribtimine (dT) - an investigational pyrimidine nucleoside therapy - in people living with thymidine kinase 2 deficiency (TK2d).1,2,3 Additional patient experience data emphasize the profound physical challenges and severe psychological strain that come with living with TK2d, and the heavy emotional and physical burden experienced by caregivers.4,5 Across all study populations, pyrimidine nucleoside and/or nucleotide therapy was generally well tolerated.1,2,3 TK2d is an ultra-rare, life-threatening, genetic mitochondrial disease with no currently approved treatment options.6,7,8,9,10,11 BRUSSELS, March 19, 2025 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced positive data from studies involving its investigational pyrimidine nucleoside therapy, doxecitine (dC) and doxribtimine (dT), in people living with thymidine kinase 2 deficiency (TK2d), at this year's MDA Clinical and Scientific Conference, Dallas, Texas, March 16-19, 2025. The data show that in individuals with TK2d who were aged 12 years or less when their symptoms first appeared, treatment with pyrimidine nucleoside and/or nucleotide therapy significantly decreased mortality and increased survival.2,3 In addition, treatment also improved functional outcomes irrespective of age of onset, including retaining or regaining motor milestones, and helped stabilize ventilatory and feeding support use.1,2,3 Across all study populations, pyrimidine nucleoside and/or nucleotide therapy was generally well tolerated with diarrhea being the most common treatment emergent adverse event.1,2,3 Thymidine kinase 2 deficiency is an ultra-rare, life-threatening, genetic mitochondrial disease characterized by progressive and severe muscle weakness (myopathy), which can impact the ability to walk, eat, and breathe independently.6,7,8,9,10 "There are no approved therapies or international clinical guidelines for the management of TK2d, therefore we are very excited to share this data with the medical community at MDA," said Donatello Crocetta, Chief Medical Officer at UCB. "The data highlight the positive impact this investigational treatment could have on the lives of people living with this debilitating and life-threatening condition." Data from participants treated in the doxecitine (dC) and doxribtimine (dT) clinical program were pooled from retrospective and prospective sources and a company-supported expanded access program (EAP). Data from untreated participants were pooled from literature reviews of case series and reports, and a retrospective chart review study. Subgroups were stratified by age of TK2d symptom onset categories and reported for participants with age of TK2d symptom onset ≤12 years and >12 years. Doxecitine and doxribtimine is currently under regulatory review by US and EU regulatory authorities. The safety and efficacy have not been established, and doxecitine and doxribtimine has not been approved by the US Food and Drug Administration (FDA) nor the European Medicines Agency (EMA). Key findings from the studies Improvement in survival: Among people living with TK2d with an age of symptom onset 12 years or less, treatment with nucleoside and/or nucleotide therapy reduced risk of death by 92–94% (hazard ratio=0.06–0.08; p<0.0001) and 87–95% (hazard ratio=0.05–0.13; p<0.0001) in the time from symptom onset and starting treatment, respectively.3 Enhanced functional outcomes: Among people living with TK2d who were aged 12 years or less when their symptoms first appeared, following treatment with pyrimidine nucleoside and/or nucleotide therapy, 75.0% (30/40) regained at least one previously lost motor milestone, with 22.5% (9/40) regaining four or more motor milestones.2 In addition, ventilatory support dependency decreased, with 16.1% (5/31) of patients reducing usage time and 16.1% (5/31) discontinuing ventilatory support altogether after treatment.2 Safety profile: Across all study populations, pyrimidine nucleoside and/or nucleotide therapy was generally well tolerated. Diarrhea (range from 84.6%-90.9%) was the most reported treatment-emergent adverse event.1,2,3 Impact on quality of life on individuals and caregivers: In addition to the data presented on nucleoside and/or nucleotide therapy, patient experience data presented highlight the debilitating physical impacts and severe psychological strain associated with living with TK2d, as well as its impact on caregivers. Many individuals reported the 'extreme' impact the condition has on their quality of life including walking, breathing and eating/swallowing difficulties. Caregivers reported that the constant demands of caregiving and minimal support/respite caused persistent stress and emotional burnout.4,5 Key data presented at MDA About thymidine kinase 2 deficiency (TK2d) Thymidine kinase 2 deficiency is an ultra-rare, life-threatening, genetic mitochondrial disease characterized by progressive and severe muscle weakness (myopathy), which can impact the ability to walk, eat, and breathe independently.6,7,8,9,10 It is estimated that the worldwide prevalence of TK2d is 1.64 [0.5, 3.1] cases per 1,000,000 people.12 The age of TK2d symptom onset is categorized as ≤12 years and >12 years.6,7,8 TK2d profoundly affects multiple health, physical, quality-of-life, and psychosocial domains, as children struggle to achieve developmental milestones or lose them, and adults lose functional independence with challenges in breathing, eating, and walking.11,13 About doxecitine and doxribtimine Doxecitine and doxribtimine is a nucleoside therapy that supports mitochondrial DNA replication resulting in increased or stabilized mitochondrial function in patients with TK2d. Pre-clinical data suggest the primary mechanism of action of doxecitine and doxribtimine is the incorporation of nucleosides deoxycytidine (dC) and deoxythymidine (dT) into skeletal muscle mitochondrial deoxyribonucleic acid (mtDNA), which has the potential to restore mitochondrial DNA copy number and improve skeletal muscle function in patients with TK2d.14,15,16 In the U.S., the application has been granted a priority review, Breakthrough Therapy Designation and Rare Pediatric Disease Designation.17,18 For further information, contact UCB: US Rare Diseases Communications Daphne Teo T: +1 770.880.7655 [email protected] Global Communications Nick Francis T: +44 7769 307745 [email protected] Corporate Communications, Media Relations Laurent Schots T +32.2.559.92.64 [email protected] Investor Relations Antje Witte T +32.2.559.94.14 [email protected] About UCB UCB, Brussels, Belgium () is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With more than 9 000 people in approximately 40 countries, the company generated revenue of € 6.1 billion in 2024. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news Forward-looking statements This press release may contain forward-looking statements including, without limitation, statements containing the words "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will", "continue" and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to differ materially from those that may be expressed or implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: the global spread and impact of COVID-19, changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring and retention of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products, which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB's efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB's products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB's data and systems. Given these uncertainties, you should not place undue reliance on any of such forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time, nor can there be any guarantee that such products will be or will continue to be commercially successful in the future. UCB is providing this information, including forward-looking statements, only as of the date of this press release and it does not reflect any potential impact from the evolving COVID-19 pandemic, unless indicated otherwise. UCB is following the worldwide developments diligently to assess the financial significance of this pandemic to UCB. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations. Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction. References: Scaglia F, et al. Survival and Functional Outcomes in Patients with Thymidine Kinase 2 Deficiency Aged >12 Years at Symptom Onset Who Received Pyrimidine Nucleos(t)ides. . Accessed February 2025. Garone C, et al. Functional Outcomes in Patients with Thymidine Kinase 2 Deficiency Aged ≤12 Years at Symptom Onset Who Received Pyrimidine Nucleos(t)ide Therapy. . Accessed February 2025. Hirano M, et al. Survival Analyses in Patients with Thymidine Kinase 2 Deficiency Aged ≤12 Years at Symptom Onset Who Received Pyrimidine Nucleos(t)ide Therapy. . Accessed February 2025. Yeske P, et al. Patients' Lived Experience of Thymidine Kinase 2 Deficiency (TK2d): Results from the Assessment of TK2d Patient Perspectives (ATP) Study. . Accessed February 2025. Yeske P, et al. Burden and impact of caring for those with thymidine kinase 2 deficiency (TK2d): results from the Assessment of TK2d Patient Perspectives (ATP) study. . Accessed February 2025. Berardo A, et al. Advances in Thymidine Kinase 2 Deficiency: Clinical Aspects, Translational Progress, and Emerging Therapies. J Neuromuscul Dis. 2022;9(2):225-35. Wang J, et al. TK2-Related Mitochondrial DNA Maintenance Defect, Myopathic Form. 2018. In: Adam MP, et al. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2025. Garone C, et al. Retrospective natural history of thymidine kinase 2 deficiency. J Med Genet. 2018;55(8):515-21. Domínguez-González C, et al. Late-onset Thymidine Kinase 2 Deficiency: A Review of 18 cases. Orphanet J Rare Dis. 2019;14(1):100. National Institutes of Health. TK2-related mitochondrial DNA depletion syndrome, myopathic form. . Accessed February 2025. U.S. FDA TK2d Patient Listening Session. . Accessed February 2025. Ma Y. Prevalence Estimation of Thymidine Kinase 2 Deficiency: An Ultra-Rare Autosomal Recessive Mitochondrial Disease. Poster presented at: ISPOR Europe; 2023, 12-15 November; Denmark. Amtmann D, et al. The impact of TK2 deficiency syndrome and its treatment by nucleoside therapy on quality of life. Mitochondrion. 2023;68:1-9. Lopez-Gomez C, et al. Deoxycytidine and Deoxythymidine Treatment for Thymidine Kinase 2 Deficiency. Ann Neurol. 2017;81(5):641-52. Lopez-Gomez C, et al. Bioavailability and cytosolic kinases modulate response to deoxynucleoside therapy in TK2 deficiency. EBioMedicine. 2019;46:356-367. National Library of Medicine. A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve). . Accessed February 2025. U.S. FDA. Orphan Drug Designations and Approvals. . Accessed February 2025. UCB Press Release. . Accessed February 2025. ©2025 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-MT-2500041 SOURCE UCB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Aug. 15, 2022 13:00 UTC Physician-scientist and biopharmaceutical executive with extensive experience in novel drug development and pipeline strategy REDWOOD CITY, Calif.--(BUSINESS WIRE)-- Nuvig Therapeutics, Inc. a private biotech company developing novel therapeutics to restore immune homeostasis and treat chronic autoimmune diseases, announced today the appointment of Joanne Quan, M.D., as Chief Medical Officer. Dr. Quan has more than 25 years of experience advancing novel drug candidates in all stages of development, from preclinical compounds to marketed products. “Joanne has a passion and commitment to developing new therapies for patients with high unmet medical needs and has a successful track record in advancing novel drug candidates for a wide range of therapeutic areas, including autoimmune disorders and rare and orphan diseases,” said Pamela Conley, Ph.D., Chief Executive Officer and Cofounder of Nuvig Therapeutics. “In addition, Joanne has built nimble and creative teams and has led multiple successful collaborations and partnerships to support the progression of new therapies to commercialization.” Dr. Quan added, “What attracted me to Nuvig was the incredible science and the potential to develop therapies for multiple immunologic diseases, many of which have few or no treatment options. It’s exciting to join Nuvig at this stage as we refine the scientific and clinical strategy and build the company to maximize the reach of this novel approach. I look forward to demonstrating proof-of-concept as we move into the clinic.” Prior to Nuvig, Dr. Quan was Chief Medical Officer at Modis Therapeutics, a company focused on developing disease-modifying therapies for patients with rare genetic diseases. After Modis was acquired by Zogenix, Dr. Quan, as a member of the Executive Team, continued to lead drug development for mitochondrial diseases, including advancing nucleoside therapy for thymidine kinase 2 deficiency toward filing in U.S. and Europe. Before Modis, she held positions of increasing responsibility at Eiger Biopharmaceuticals, InterMune, Arena Pharmaceuticals, Bayhill Therapeutics, ALZA Corporation (Johnson and Johnson), Genentech, and PathoGenesis. Dr. Quan received a B.A. in molecular biology at the University of California, Berkeley and an M.D. at Stanford University School of Medicine. She completed a residency in Internal Medicine at Massachusetts General Hospital and a fellowship in Pulmonary and Critical Care Medicine at the University of Washington. About Nuvig Therapeutics Nuvig Therapeutics is advancing innovative and transformational therapies that are designed to induce natural mechanisms to restore immune homeostasis, rebalance immune function following inflammation, and provide better treatments for patients. Nuvig is building a pipeline of novel immune therapeutics for chronic inflammatory and autoimmune diseases. For more info, visit .