CYP2D6

SOLANA BEACH, Calif., April 28, 2025 (GLOBE NEWSWIRE) -- Evoke Pharma, Inc. (NASDAQ: EVOK), a specialty pharmaceutical company focused on treatments for gastrointestinal (GI) disorders with an emphasis on GIMOTI®, today announced that a new abstract comparing the incidence of tardive dyskinesia (TD) in patients receiving continuous versus intermittent oral metoclopramide (OMCP) treatment has been accepted for presentation at Digestive Disease Week® (DDW) 2025, taking place May 3–6, 2025, in San Diego, CA. The analysis leverages real-world data from over 100 million U.S. patients and highlights differences in TD incidence rates and timing based on metoclopramide dosing patterns. “This research builds on our ongoing commitment to understanding metoclopramide’s long-term safety profile,” said Matt D’Onofrio, CEO of Evoke Pharma. “As the only FDA-approved treatment for diabetic gastroparesis, it’s critical that we continue evaluating both efficacy and safety, particularly around concerns like tardive dyskinesia. This real-world data helps clarify risk profiles and supports more informed prescribing decisions for physicians interested in Gimoti. We’re excited to share these findings with the DDW community.” Details of the poster presentation are as follows: Abstract Title: Comparison of the Incidence of Tardive Dyskinesia in Patients Receiving Continuous vs Intermittent Oral Metoclopramide Presenter & Lead Author: Pierantonio Russo, MD, EVERSANA Life Sciences Presentation Session: Gastroparesis and Small Intestinal Dysmotility Date & Time: Monday, May 5, 2025, from 12:30 – 1:30 p.m. PT The abstract will be available via the DDW ePosters site and accessible to attendees through the DDW meeting planner and mobile app after 12:01 a.m. PT on Sunday, May 4, 2025. Visit Evoke Pharma at DDW 2025:To learn more about GIMOTI and our latest research, visit Evoke Pharma at booth #917 during DDW. About Evoke Pharma, Inc. Evoke is a specialty pharmaceutical company focused primarily on the development of drugs to treat GI disorders and diseases. The company developed, commercialized and markets GIMOTI, a nasal spray formulation of metoclopramide, for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adults. Diabetic gastroparesis is a GI disorder affecting millions of patients worldwide, in which the stomach takes too long to empty its contents resulting in serious GI symptoms as well as other systemic complications. The gastric delay caused by gastroparesis can compromise absorption of orally administered medications. Prior to FDA approval to commercially market GIMOTI, metoclopramide was only available in oral and injectable formulations and remains the only drug currently approved in the United States to treat gastroparesis. Visit www.EvokePharma.com for more information. Follow Evoke Pharma on LinkedInFollow Evoke Pharma on Twitter About Digestive Disease Week® (DDW) Digestive Disease Week® (DDW) is the largest international gathering of physicians, researchers and academics in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery. Jointly sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA), the American Society for Gastrointestinal Endoscopy (ASGE) and the Society for Surgery of the Alimentary Tract (SSAT), DDW is an in-person and online meeting from May 3-6, 2025. The meeting showcases nearly 6,000 abstracts and 1,000 invited talks on the latest advances in GI research, medicine, and technology. More information can be found at www.ddw.org. About Gimoti® (metoclopramide) nasal spray GIMOTI is indicated for the relief of symptoms in adults with acute and recurrent diabetic gastroparesis. Important Safety Information WARNING: TARDIVE DYSKINESIA Metoclopramide can cause tardive dyskinesia (TD), a serious movement disorder that is often irreversible. The risk of developing TD increases with duration of treatment and total cumulative dosage.Discontinue GIMOTI in patients who develop signs or symptoms of TD. In some patients, symptoms may lessen or resolve after metoclopramide is stopped.Avoid treatment with metoclopramide (all dosage forms and routes of administration) for longer than 12 weeks because of the increased risk of developing TD with longer-term use. GIMOTI is not recommended for use in: Pediatric patients due to the risk of developing tardive dyskinesia (TD) and other extrapyramidal symptoms as well as the risk of methemoglobinemia in neonates.Moderate or severe hepatic impairment (Child-Pugh B or C), moderate or severe renal impairment (creatinine clearance less than 60 mL/minute), and patients concurrently using strong CYP2D6 inhibitors due to the risk of increased drug exposure and adverse reactions. GIMOTI is contraindicated: In patients with a history of tardive dyskinesia (TD) or a dystonic reaction to metoclopramide.When stimulation of gastrointestinal motility might be dangerous (e.g., in the presence of gastrointestinal hemorrhage mechanical obstruction, or perforation).In patients with pheochromocytoma or other catecholamine-releasing paragangliomas. Metoclopramide may cause a hypertensive/pheochromocytoma crisis, probably due to release of catecholamines from the tumor.In patients with epilepsy. Metoclopramide may increase the frequency and severity of seizures.In patients with hypersensitivity to metoclopramide. Reactions have included laryngeal and glossal angioedema and bronchospasm. Potential adverse reactions associated with metoclopramide include: Tardive dyskinesia (TD), other extrapyramidal effects (EPS), parkinsonism symptoms, motor restlessness, neuroleptic malignant syndrome (NMS), depression, suicidal ideation and suicide, hypertension, fluid retention, hyperprolactinemia, effects on the ability to drive and operate machinery. Most common adverse reactions (≥5%) for GIMOTI are: dysgeusia, headache, and fatigue. These are not all of the possible side effects of GIMOTI. Call your doctor for medical advice about whether you should take GIMOTI and the possible risk factors and side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. Safe Harbor Statement Evoke cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should, ”expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential” or “continue” or the negatives of these terms or other similar expressions. These statements are based on the company’s current beliefs and expectations. These forward-looking statements include statements regarding: guidance regarding 2024 net product sales; potential future prescribing trends for GIMOTI based on Evoke’s or EVERSANA’s marketing efforts; Evoke’s commercialization plans, the potential market opportunity for GIMOTI, Evoke’s partnership with ASPN Pharmacies, growth in prescriptions, patients taking GIMOTI and the conversion of prescriptions to fills, and Evoke’s expected cash runway. The inclusion of forward-looking statements should not be regarded as a representation by Evoke that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Evoke’s business, including, without limitation: Evoke may not be able to achieve its guidance for 2024 including as a result of decreased demand for GIMOTI; Evoke’s and EVERSANA’s ability to successfully drive market demand for GIMOTI; Evoke’s ability to obtain additional financing as needed to support its operations; Evoke may use its capital resources sooner than expected; warrant holders may choose not to exercise any of the outstanding warrants; Evoke’s dependence on third parties for the manufacture of GIMOTI; Evoke is entirely dependent on the success of GIMOTI; inadequate efficacy or unexpected adverse side effects relating to GIMOTI that could result in recalls or product liability claims; Evoke’s ability to maintain intellectual property protection for GIMOTI; and other risks and uncertainties detailed in Evoke’s prior press releases and in the periodic reports it files with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Evoke undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor & Media Contact:Daniel Kontoh-BoatengDKB PartnersTel: 862-213-1398dboateng@dkbpartners.net

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 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. 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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. 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

New Analyses Related to Aficamten Expand on its Metabolism Pathways, Treatment Effect Associated with Combination Therapy with Disopyramide and Longer-Term Effect on Cardiac Structure and Function SOUTH SAN FRANCISCO, Calif., March 17, 2025 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced five presentations related to aficamten, an investigational cardiac myosin inhibitor, and hypertrophic cardiomyopathy (HCM), at the American College of Cardiology (ACC) Annual Scientific Session & Expo taking place from March 29, 2025–March 31, 2025 in Chicago, IL. “We are pleased to be sharing several new analyses relating to aficamten at the upcoming ACC Scientific Session & Expo,” said Stephen Heitner, M.D., Vice President, Head of Clinical Research. “The presentations describe the drug metabolism of aficamten, the safety of combination therapy with the standard of care medication disopyramide, and the effect of longer-term use of aficamten. Together these analyses add to the strong and growing evidence base supporting the potential for aficamten in patients with obstructive HCM and inform how it may be used in clinical practice.” Evaluation of Cytochrome P450 2C9, 2C19, and 2D6 Inhibition on the Pharmacokinetics of Aficamten in Healthy Participants (1091-139) Poster Presentation, March 29, 2025, 2:00-3:00 PM CT, South Hall. Neha Maharao, Ph.D., Senior Clinical Pharmacologist, Cytokinetics. Data from an open-label, fixed-sequence drug-drug interaction (DDI) study of aficamten in healthy participants will be presented in a poster presentation. A previous study showed that aficamten is metabolized, in part, by the cytochrome P450 (CYP) enzyme 3A41. To further characterize its metabolic pathways, aficamten was evaluated with concomitant administration of three strong inhibitors of one or more of the CYP pathways: fluconazole (inhibitor of 2C9, 2C19, and 3A4), paroxetine (inhibitor of 2D6) and fluoxetine (inhibitor of 2C19 and 2D6). The data show that aficamten was eliminated by multiple CYP pathways, primarily by CYP2C9 (fraction metabolized [fm]=50%), with contributions from CYP3A (fm=26%), CYP2D6 (fm=21%) and CYP2C19 (fm=3%). Safety and Outcomes of Concomitant Aficamten and Disopyramide Use and Withdrawal in Patients with Obstructive Hypertrophic Cardiomyopathy: An Analysis of REDWOOD-HCM Cohort 3, SEQUOIA-HCM, and FOREST-HCM Trials (411-06) Oral Presentation, March 31, 2025, 9:11-9:18 AM CT, S406b. Ahmad Masri, M.D., MS, Director of the Hypertrophic Cardiomyopathy Center at Oregon Health & Science University. Data from an analysis of concomitant treatment with aficamten and disopyramide from completed and ongoing clinical trials of aficamten in patients with obstructive HCM will be presented in an oral presentation. The analysis included 50 participants from Cohort 3 of REDWOOD-HCM (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM), SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM) and FOREST-HCM (Follow-up, Open-Label, Research Evaluation of Sustained Treatment with Aficamten in HCM) who were receiving disopyramide at baseline. Participants were separated into four groups: those on disopyramide who underwent withdrawal of aficamten due to end of treatment in Cohort 3 of REDWOOD-HCM or SEQUOIA-HCM (n=29), patients on disopyramide receiving placebo in SEQUOIA-HCM (n=20), patients on aficamten who underwent disopyramide withdrawal in FOREST-HCM (n=17) and patients on aficamten who maintained treatment with disopyramide in FOREST-HCM (n=27). Combination therapy with aficamten and disopyramide was well-tolerated; the analysis suggests that combination of disopyramide with aficamten did not result in lower left ventricular outflow tract (LVOT) gradients compared to treatment with aficamten alone. The analysis suggests that withdrawal of disopyramide while receiving aficamten did not reduce the efficacy of aficamten and further that withdrawal of aficamten while on disopyramide resulted in the return of LVOT obstruction and symptoms, with an increase in NT-proBNP. There were no safety events reported with either aficamten or disopyramide withdrawal, and no episodes of atrial fibrillation after disopyramide withdrawal were reported. Effect of Aficamten Treatment for Up to 72 Weeks on Cardiac Structure and Function in Patients with Obstructive Hypertrophic Cardiomyopathy: The SEQUOIA-HCM and FOREST-HCM CMR Sub-studies (964-09) Moderated Poster Presentation, March 30, 2025, 12:06-12:13 PM CT, Theater 5. Ahmad Masri, M.D., MS, Director of the Hypertrophic Cardiomyopathy Center at Oregon Health & Science University. New data from the cardiac magnetic resonance (CMR) imaging sub-studies of FOREST-HCM and SEQUOIA-HCM will be presented in a moderated poster presentation. At the time of the current analysis, 64 patients had completed a baseline CMR, including 36 patients who had completed a follow-up CMR at 72 weeks, and 28 patients who had completed a follow-up CMR at 48 weeks. Longer-term treatment with aficamten resulted in statistically significant improvements (mean ±SD) in measures of cardiac structure and function including left ventricular mass index (-9.8 g/m2 ±18.1, p<0.0001), maximum left ventricular septal wall thickness (-2.4 mm ±2.3, p<0.0001), left atrial volume (-17.9 ml ±28.3, p<0.0001) and mitral regurgitant volume (-18.1 ml ±19.2, p<0.0001) and fraction (-14.2% ±15.6, p<0.0001). These changes were accompanied by stable replacement fibrosis (late gadolinium enhancement mass = -0.3 g ±5.0, p=0.51) and reduced interstitial fibrosis (global extracellular volume (ECV) = -1.2 % ±2.8, p = 0.002; Native T1 = -36.6 ms ±55.7, p<0.0001). Understanding the Impact of Placebo on Patient-Reported Health Status: An Analysis from SEQUOIA-HCM (1029-176) Poster Presentation, March 29, 2025, 9:30-10:30 AM CT, South Hall. Charles F. Sherrod, M.D., M.Sc., Cardiology Fellow, UMKC Healthcare Institute for Innovations in Quality, Saint Luke’s Mid America Heart Institute. A new analysis from SEQUOIA-HCM of the placebo effect on Kansas City Cardiomyopathy Questionnaire Overall Summary Scores (KCCQ-OSS) will be presented in a poster presentation. In patients randomized to placebo in SEQUOIA-HCM, the change in KCCQ-OSS was evaluated from baseline to Week 24 and following blinded treatment withdrawal from Week 24 to Week 28. Among the 140 (47%) patients on placebo, the median KCCQ-OSS at baseline was 67.2 (95% CI: 62.9, 71.5) and the median improvement at Week 24 was 5.7 points (95% CI: 3.2, 8.3; p<0.01). After withdrawal from Week 24 to Week 28, the median score decreased by only 2.1 points (95% CI: -3.5, -0.7; p<0.01). These results suggest that about one-third of the improvement observed in KCCQ-OSS in patients receiving placebo was due to a placebo effect, further suggesting that other changes in health status following treatment with placebo may be due to other factors involved in clinical trial participation, such as receiving treatment at expert centers and changes in patient behavior. Association Between Race/Ethnicity and Outcomes in Patients with Non-Obstructive Hypertrophic Cardiomyopathy (1251-173) Poster Presentation, March 31, 2025, 10:30-11:30 AM CT, South Hall. Nosheen Reza, M.D., Assistant Professor of Medicine, Division of Cardiovascular Medicine, the Hospital of the University of Pennsylvania. Data from a new health economics and outcomes research (HEOR) analysis of the association between race/ethnicity and outcomes in non-obstructive HCM patients will be presented in a poster presentation. This retrospective cohort study included adult patients diagnosed with non-obstructive HCM from January 1, 2013 to December 31, 2021. Of the 9,842 patients included, 74.2% were non-Hispanic white, 19.5% were non-Hispanic Black/African American, 4.2% were Hispanic and 2.1% were non-Hispanic Asian. White patients had greater rates of atrial fibrillation and cardiovascular hospitalization compared to Asian and Hispanic patients. Compared to white patients, Black patients had increased rates of stroke (risk ratio [RR] 1.76), heart failure (RR 1.73), ventricular tachycardia (RR 1.24), sudden cardiac arrest (RR 1.90), cardiovascular hospitalization (RR 1.42) and cardiovascular rehospitalization (RR 1.31) and a lower rate of atrial fibrillation (RR 0.74; all p<0.001). All-cause mortality was highest among Black patients (p<0.001). Compared to white patients, non-Hispanic Black patients had the highest rate of adverse cardiovascular outcomes and all-cause mortality, while Asian and Hispanic patients experienced lower rates. These results highlight an urgent need to address drivers of race/ethnicity-based disparities in patients with nHCM. About Aficamten Aficamten is an investigational selective, small molecule cardiac myosin inhibitor discovered following an extensive chemical optimization program that was conducted with careful attention to therapeutic index and pharmacokinetic properties and as may translate into next-in-class potential in clinical development. Aficamten was designed to reduce the number of active actin-myosin cross bridges during each cardiac cycle and consequently suppress the myocardial hypercontractility that is associated with HCM. In preclinical models, aficamten reduced myocardial contractility by binding directly to cardiac myosin at a distinct and selective allosteric binding site, thereby preventing myosin from entering a force producing state. The development program for aficamten is assessing its potential as a treatment that improves exercise capacity as measured by peak oxygen uptake (pVO2) and relieves symptoms in patients with HCM. Aficamten was evaluated in SEQUOIA-HCM, a positive pivotal Phase 3 clinical trial in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Aficamten received Breakthrough Therapy Designation for the treatment of symptomatic obstructive HCM from the U.S. Food & Drug Administration (FDA) and from the National Medical Products Administration (NMPA) in China. Aficamten is currently under regulatory review in the U.S, where the FDA is reviewing a New Drug Application (NDA) for aficamten, which was assigned standard review and a Prescription Drug User Fee Act (PDUFA) target action date of September 26, 2025. Additionally, the European Medicines Agency (EMA) is reviewing a Marketing Authorization Application (MAA) for aficamten, and The Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) is reviewing an NDA for aficamten with Priority Review. Aficamten is also currently being evaluated in MAPLE-HCM, a Phase 3 clinical trial of aficamten as monotherapy compared to metoprolol as monotherapy in patients with obstructive HCM; ACACIA-HCM, a Phase 3 clinical trial of aficamten in patients with non-obstructive HCM; CEDAR-HCM, a clinical trial of aficamten in a pediatric population with obstructive HCM; and FOREST-HCM, an open-label extension clinical study of aficamten in patients with HCM. About Hypertrophic Cardiomyopathy Hypertrophic cardiomyopathy (HCM) is a disease in which the heart muscle (myocardium) becomes abnormally thick (hypertrophied). The thickening of cardiac muscle leads to the inside of the left ventricle becoming smaller and stiffer, and thus the ventricle becomes less able to relax and fill with blood. This ultimately limits the heart’s pumping function, resulting in reduced exercise capacity and symptoms including chest pain, dizziness, shortness of breath, or fainting during physical activity. HCM is the most common monogenic inherited cardiovascular disorder, with approximately 280,000 patients diagnosed, however, there are an estimated 400,000-800,000 additional patients who remain undiagnosed in the U.S.2,3,4 Two-thirds of patients with HCM have obstructive HCM (oHCM), where the thickening of the cardiac muscle leads to left ventricular outflow tract (LVOT) obstruction, while one-third have non-obstructive HCM (nHCM), where blood flow isn’t impacted, but the heart muscle is still thickened. People with HCM are at high risk of also developing cardiovascular complications including atrial fibrillation, stroke and mitral valve disease.5 People with HCM are at risk for potentially fatal ventricular arrhythmias and it is one of the leading causes of sudden cardiac death in younger people or athletes.6 A subset of patients with HCM are at high risk of progressive disease leading to dilated cardiomyopathy and heart failure necessitating cardiac transplantation. About Cytokinetics Cytokinetics is a leading muscle biology specialty biopharmaceutical company focused on discovering, developing and commercializing muscle biology-directed drug candidates as potential treatments for debilitating diseases in which muscle performance is compromised. As a pioneer in muscle and the mechanics of muscle performance, Cytokinetics is intent on meaningfully improving the lives of patients through global access to innovative medicines. Cytokinetics is readying for potential regulatory approvals and commercialization of aficamten, a potential next-in-class cardiac myosin inhibitor, following positive results from SEQUOIA-HCM, the pivotal Phase 3 clinical trial in patients with obstructive hypertrophic cardiomyopathy (HCM). Aficamten is also being evaluated in additional clinical trials enrolling patients with obstructive and non-obstructive HCM. Cytokinetics is also developing omecamtiv mecarbil, a cardiac myosin activator, in patients with heart failure with severely reduced ejection fraction (HFrEF), CK-586, a cardiac myosin inhibitor with a mechanism of action distinct from aficamten, for the potential treatment of heart failure with preserved ejection fraction (HFpEF) and CK-089, a fast skeletal muscle troponin activator with potential therapeutic application to a specific type of muscular dystrophy and other conditions of impaired skeletal muscle function. For additional information about Cytokinetics, visit www.cytokinetics.com and follow us on X, LinkedIn, Facebook and YouTube. Forward-Looking Statements This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics disclaims any intent or obligation to update these forward-looking statements and claims the protection of the Act’s Safe Harbor for forward-looking statements. Examples of such statements include, but are not limited to, statements express or implied relating to the properties, treatment effect or potential benefits of aficamten or any of our other drug candidates or our ability to obtain regulatory approval for aficamten for the treatment of obstructive hypertrophic cardiomyopathy or any other indication from FDA or any other regulatory body in the United States or abroad. Such statements are based on management’s current expectations, but actual results may differ materially due to various risks and uncertainties, including, but not limited to the risks related to Cytokinetics’ business outlines in Cytokinetics’ filings with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and Cytokinetics’ actual results of operations, financial condition and liquidity, and the development of the industry in which it operates, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that Cytokinetics makes in this press release speak only as of the date of this press release. Cytokinetics assumes no obligation to update its forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release. CYTOKINETICS® and the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries. Contact: Cytokinetics Diane Weiser Senior Vice President, Corporate Affairs (415) 290-7757 References: Source: Cytokinetics, Incorporated