Spasm

CHESHIRE, UK I April 28, 2025 I BlackfinBio Ltd, a clinical stage gene therapy company focused on the development of treatments for rare neurological diseases, announces today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for a Phase 1/2 clinical trial of its novel adeno-associated virus (AAV) gene therapy, BFB-101, in children with Hereditary Spastic Paraplegia, Type 47 (SPG47). The trial will be conducted in the U.S. at Boston Children’s Hospital and is expected to commence recruiting by the end of 2025. The FDA has granted an orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to BFB-101 for the treatment of SPG47. SPG47 is a rare, autosomal-recessive, neurological disorder characterised by progressive lower-limb spasticity, developmental delays and intellectual disability in children. It is caused by deleterious changes in the AP4B1 gene. Currently, there is no cure or effective treatment available for this devastating condition. BFB-101 has been designed specifically to address the underlying genetic cause of SPG47 by delivering a functional copy of the AP4B1 gene, with the goal of halting or reversing disease progression. Preclinical results with BFB-101 have demonstrated promising activity and safety (1). The Phase 1/2 clinical trial is a single-center open-label study with a primary objective to assess the safety and efficacy of a single injection into the cerebrospinal fluid at the base of the brainstem near the spinal cord, termed an ‘ICM’ administration. This type of delivery generally allows for rapid and efficient delivery of agents to the central nervous system. BFB-101 will be administered by ICM in up to five children with AP4B1-associated SPG47. The secondary objectives will aim to evaluate the impact of BFB-101 on motor function, development and health-related quality of life. “The FDA’s clearance of the BFB-101 IND is an important milestone for our rare neurological disease program and the company. We look forward to initiating enrollment in the U.S. later this year and are working closely with the investigator team at Boston Children’s Hospital to evaluate the therapeutic utility of this gene therapy in children with SPG47,” said Peter Nolan, Founder and Chief Executive Officer of BlackfinBio Ltd. “We are proud to be leading this important clinical trial at Boston Children’s Hospital, bringing forward a precision medicine approach for children affected by SPG47. As clinicians, we see firsthand the impact of this disorder and the urgency for effective treatments. BFB-101 represents a promising gene therapy candidate, and this trial is a critical step toward delivering meaningful change for patients and families affected by AP-4-associated hereditary spastic paraplegia and related conditions,” said Dr. Darius Ebrahimi-Fakhari, M.D., Ph.D., Boston Children’s Hospital (Principal Investigator). “Having overseen this therapeutic innovation from discovery stage, it is a huge source of excitement to reach this important IND milestone. Given the incredibly high unmet need for children with this devastating condition, we are on a mission at BlackfinBio to transform treatment options for these young lives and make a real impact to the lives of their families,” added Professor Mimoun Azzouz, Founder and Chief Scientific Officer of BlackfinBio Ltd. Reference: (1): Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47 Disclosure Statement: Dr. Darius Ebrahimi-Fakhari has consulted for BlackfinBio. About BFB-101 BFB-101 aims to address the underlying genetic cause of Hereditary Spastic Paraplegia, Type 47 (SPG47) by delivering a functional copy of the AP4B1 gene, with the goal of halting or reversing disease progression. It is administered via the brain as a single lifetime dose and has shown potential in restoring AP-4 function in vitro and improving motor function in AP4B1 mutant mice. BFB-101 was originally developed by Professor Mimoun Azzouz at the University of Sheffield with the support of Cure AP-4 ( https://cureap4.org ), LifeArc ( www.lifearc.org ). This project has also received partial funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 945473 to support AAV9 capsid synthesis for immune response study. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA. The FDA has granted an orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to BFB-101 for the treatment of SPG47. About BlackfinBio Ltd BlackfinBio is a clinical stage gene therapy company focused on the development of treatments for rare neurological diseases. The pipeline comprises BFB-101, a clinical stage AP4B1 replacement AAV gene therapy for spastic paraplegia 47 – an ultra-rare genetic neurological disease for which no treatment currently exists. BFB-201 is a preclinical stage gene therapy to treat several rare dopamine deficiency disorders. For more information, please visit https://blackfin.bio and follow us on LinkedIn . SOURCE: BlackfinBio

PRINCETON, N.J., April 6, 2025 /PRNewswire/ -- Luye Pharma Group today announced that ERZOFRI® (paliperidone palmitate) extended-release injectable suspension is now available for commercial sale in the U.S. for the treatment of adults with schizophrenia and as a monotherapy or adjunct therapy for the treatment of adults with schizoaffective disorder. ERZOFRI, administered once a month, delivers the active ingredient, paliperidone, through a long-acting, extended-release delivery system. It simplifies treatment by enabling single-dose initiation due to its unique 351 mg initiation dose. The currently prescribed monthly paliperidone palmitate long-acting injectable (LAI) requires two initiation doses, on Day 1 and Day 8. ERZOFRI may cause serious side effects, including: Increased risk of death in elderly people with dementia-related psychosis. ERZOFRI increases the risk of death in elderly people who have lost touch with reality (psychosis) due to confusion and memory loss (dementia). ERZOFRI is not for the treatment of people with dementia-related psychosis. The most common side effects of ERZOFRI include injection site reactions, sleepiness or drowsiness, dizziness, feeling restless or like you need to move, and abnormal muscle movements including tremor (shaking), shuffling, uncontrolled involuntary movements, and abnormal movements of your eyes. Both schizophrenia and schizoaffective disorder are severe, chronic psychiatric disorders characterized by recurrent relapses[1]. Non-adherence to medication is a major challenge in patients with such conditions. Failure of follow-up care after discharge significantly increases patients' non-adherence to prescribed medications, relapse and rehospitalization[2]. LAI antipsychotics, with a reduced dosage frequency compared to oral antipsychotics, effectively improve medication adherence and reduce the risk of healthcare providers not knowing if their patients fail to follow the prescribed dosage regimen[3]. ERZOFRI's approval by the FDA is based on the results of an open-label, randomized, multiple-dose, parallel-group study evaluating its pharmacokinetic (PK) profile and relative bioavailability at steady state versus an established paliperidone palmitate LAI drug for reference. The study demonstrated the bioequivalence of ERZOFRI to the listed drug at steady state (treatment day 8) without the need for a second initiation injection. Comparable total drug exposure and a safety profile consistent with the listed drug were established. In addition to the unique 351 mg/2.25mL initiation dose, ERZOFRI is available in single-use kits in the following strengths: 39 mg/0.25mL, 78 mg/0.5 mL, 117 mg/0.75 mL, 156 mg/mL, and 234 mg/1.5 mL. "This newly approved long-acting injectable antipsychotic for the management of schizophrenia can help address treatment initiation complexities and provides an exciting alternative to help patients reach their treatment goals." said Christoph U Correll, MD, Professor of Psychiatry, Zucker School of Medicine at Hofstra/Northwell, New York, USA. Rongbing Yang, President of Luye Pharma Group, added: "Medication adherence and discontinuation present a major challenge in the treatment of schizophrenia and schizoaffective disorder. Using LAIs is effective in increasing medication adherence. ERZOFRI, with its simplified dosage regimen eliminating the need for a second dose when initiating treatment is expected to increase treatment adherence in the initial stage. Its commercial launch in the U.S. marks a major milestone in our commitment to addressing the unmet medical needs of patients in the U.S. The central nervous system is a key therapeutic area of focus for Luye Pharma, and we are committed to providing innovative therapies to combat mental disorders and expand treatment options." ERZOFRI® (paliperidone palmitate) extended-release injectable suspension INDICATIONS What is ERZOFRI® (paliperidone palmitate)? ERZOFRI is a prescription medicine given by injection by a healthcare provider and used to treat: schizophrenia in adults schizoaffective disorder in adults either alone or with other medicines such as mood stabilizers or antidepressants It is not known if ERZOFRI is safe and effective in children. IMPORTANT SAFETY INFORMATION FOR ERZOFRI (paliperidone palmitate) ERZOFRI may cause serious side effects, including: Increased risk of death in elderly people with dementia-related psychosis. ERZOFRI increases the risk of death in elderly people who have lost touch with reality (psychosis) due to confusion and memory loss (dementia). ERZOFRI is not for the treatment of people with dementia-related psychosis. Do not receive ERZOFRI if you are allergic to paliperidone, risperidone, or any of the ingredients in ERZOFRI. What are the possible serious side effects of ERZOFRI? Cardiovascular problems (including stroke) in elderly people with dementia-related psychosis that can lead to death. Neuroleptic Malignant Syndrome (NMS) a serious condition that can lead to death. Call your healthcare provider right away or go to your nearest emergency room right away if you get any of the following signs or symptoms: high fever stiff muscles confusion sweating changes in your breathing, pulse, heart rate, or blood pressure Problems with your heartbeat. These heart problems can cause death. Call your healthcare provider right away if you get any of these symptoms: passing out or feeling like you will pass out dizziness feeling as if your heart is pounding or missing beats Uncontrolled body movements (tardive dyskinesia). ERZOFRI may cause movements that you cannot control in your face, tongue, or other body parts. Tardive dyskinesia may not go away, even if you stop taking ERZOFRI. Tardive dyskinesia may also start after you stop taking ERZOFRI. Problems with your metabolism such as: High blood sugar (hyperglycemia) and diabetes. Increases in blood sugar can happen in some people who are treated with ERZOFRI. Extremely high blood sugar can lead to coma or death. If you have diabetes or risk factors for diabetes such as being overweight, or a family history of diabetes, your healthcare provider should check your blood sugar before you start treatment and during your treatment with ERZOFRI. Call your healthcare provider if you get any of these symptoms of high blood sugar during treatment with ERZOFRI: feel very thirsty feel very hungry feel sick to your stomach need to urinate more than usual feel weak or tired feel confused, or your breath smells fruity Increased fat levels (cholesterol and triglycerides in your blood). Your healthcare provider may check your cholesterol and triglyceride levels during treatment with ERZOFRI. Weight gain. You and your healthcare provider should check your weight regularly during treatment with ERZOFRI. Decreased blood pressure (orthostatic hypotension) and fainting. You may feel lightheaded or faint when you rise too quickly from a sitting or lying position. Falls. ERZOFRI may make you sleepy or dizzy, may cause a decrease in your blood pressure when changing position (orthostatic hypotension), and can slow your thinking and motor skills which may lead to falls that can cause fractures or other injuries. Low white blood cell counts. Your healthcare provider may do blood tests during treatment with ERZOFRI. Increased prolactin levels in your blood (hyperprolactinemia). ERZOFRI may cause a rise in the blood levels of a hormone called prolactin that may cause side effects including missed menstrual periods, a reversible reduction in fertility in females who are able to become pregnant, leakage of milk from the breasts, development of breasts in men, or problems with erection. Sleepiness, drowsiness feeling tired, difficulty thinking and doing normal activities. Seizures (convulsions). Difficulty swallowing that can cause food or liquid to get into your lungs. Prolonged or painful erection lasting more than 4 hours (priapism). Call your healthcare provider or go to your nearest emergency room right away if you have an erection that lasts more than 4 hours. Problems with control of your body temperature so that you feel too warm. Before taking ERZOFRI, tell your healthcare provider about all of your medical conditions including if you: have never taken paliperidone or risperidone before have had neuroleptic malignant syndrome (NMS) have or had heart problems, including heart attack, heart failure, abnormal heart rhythm, or long QT syndrome have or had low levels of potassium or magnesium in your blood have or had uncontrolled movements of your tongue, face, mouth or jaw (tardive dyskinesia) have or had kidney or liver problems have or had high blood sugar, diabetes or have a family history of diabetes have or had high levels of total cholesterol, LDL cholesterol, or triglycerides, or low levels of HDL cholesterol have Parkinson's disease or a type of dementia called Lewy Body Dementia have or had problems with dizziness or fainting or are being treated for high blood pressure have or had a low white blood cell count have or had seizures or epilepsy are pregnant or plan to become pregnant. It is not known if ERZOFRI will harm your unborn baby. If you become pregnant during treatment with ERZOFRI, talk to your healthcare provider about registering with the National Pregnancy Registry for Atypical Antipsychotics. You can register by calling 1-866-961-2388 or visit . Babies born to women who are treated with ERZOFRI during their third trimester of pregnancy may experience symptoms such as tremors, irritability, excessive sleepiness, eye twitching, muscle spasms, decreased appetite, difficulty breathing, or abnormal movement of arms and legs. Let your healthcare provider know if these symptoms occur. are breastfeeding or plan to breastfeed. ERZOFRI can pass into your breastmilk. Talk to your healthcare provider about the best way to feed your baby if you receive ERZOFRI. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. ERZOFRI and certain other medicines may affect each other causing possible serious side effects or affect the way each other works. Know the medicines you take. Keep a list of them to show to your healthcare provider or pharmacist when you get a new medicine. The most common side effects of ERZOFRI include: injection site reactions sleepiness or drowsiness dizziness feeling restless or like you need to move abnormal muscle movements including tremor (shaking), shuffling, uncontrolled involuntary movements, and abnormal movements of your eyes Tell your healthcare provider if you have any side effect that bothers you or does not go away. These are not all of the possible side effects of ERZOFRI. Call your doctor for medical advice about side effects. You may report side effects to FDA at or call 1-800- FDA-1088. About Luye Pharma Group Luye Pharma Group is an international pharmaceutical company dedicated to the R&D, manufacturing and sale of innovative medications. The company has established R&D centers in China, the U.S. and Europe, with a robust pipeline of over 20 drug candidates in China and more than 10 drug candidates in other international markets. Luye Pharma maintains high-level international standards in novel drug delivery technologies including microspheres, liposomes, and transdermal drug delivery systems. The company has achieved multiple innovations in new chemical entities and antibodies, and is also actively making strategic developments in the fields of cell therapies and gene therapies. Luye Pharma is developing a global supply chain of 8 manufacturing sites built up around the world, with GMP quality management and control systems established in line with international standards. With more than 30 products covering the central nervous system, oncology, cardiovascular, metabolism and other therapeutic areas, business is conducted in over 80 countries and regions around the world, including the largest pharmaceutical markets -- China, the U.S., Europe and Japan, as well as in fast-growing emerging markets. SOURCE Luye Pharma Group WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

For the first time, Thompson shares his journey living with gastroesophageal reflux disease (GERD) and experience using VOQUEZNA, as part of the new GERD IS NO JOKE campaignVOQUEZNA is a potassium competitive acid blocker (PCAB), the first and only FDA-approved treatment of its kind for both types of GERD1 A Media Snippet accompanying this announcement is available by clicking on this link. FLORHAM PARK, N.J., March 31, 2025 (GLOBE NEWSWIRE) -- Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal (GI) diseases, announced today a partnership with actor, comedian, and author Kenan Thompson, best known as the longest-running cast member on Saturday Night Live. For the first time ever, Thompson is publicly speaking out about his experience with GERD as part of the GERD IS NO JOKE campaign to raise awareness about the condition and encourage others to talk with their doctor about treatment options. Thompson, who has been diagnosed with the most common form of GERD, called Non-Erosive GERD, found heartburn relief with VOQUEZNA® (vonoprazan) after struggling with other treatments. VOQUEZNA is the first and only treatment of its kind approved by the U.S. Food and Drug Administration (FDA) to help manage his condition.1 VOQUEZNA is approved for use in adults for the relief of heartburn associated with Non-Erosive GERD and for the treatment of all severities of Erosive Esophagitis, commonly referred to as Erosive GERD, and relief of related heartburn.1 As one of more than 65 million people in the U.S. living with GERD2—a chronic condition where stomach acid flows into the esophagus, causing irritation and heartburn3—Thompson knows firsthand how disruptive symptoms can be. His goal is to help elevate the discussion around GERD and help others recognize the importance of finding an effective treatment to better manage their disease. “This is the first time I’m sharing my story with GERD, and trust me—it's no joke,” said Thompson. “I’ve dealt with terrible burning in my chest, avoided some of my favorite foods, and even had my heartburn affect my voice and sleep, which is tough when your job is to make people laugh. After struggling with treatments that didn’t work, my doctor recommended VOQUEZNA, and it helped me kick some acid and reduce my heartburn! I’m excited to partner with Phathom to encourage others to talk to their doctor and find the right treatment for them.” While Thompson has not experienced side effects while taking VOQUEZNA, some patients taking VOQUEZNA do. The most common side effects of VOQUEZNA for the relief of heartburn related to Non-Erosive GERD include stomach pain, constipation, diarrhea, nausea, and urinary tract infection.1 Please see additional Important Safety Information below. Thompson shares more about his personal story with GERD at GERDIsNoJoke.com, including a downloadable guide from his own unique point of view, and helpful tips for people to talk to their doctor about the condition. “Despite using over-the-counter medications and prescription treatments like antacids, histamine-2 receptor antagonists, or proton pump inhibitors—also called PPIs—many people with GERD continue to experience persistent, bothersome symptoms which could affect their daily lives,” said Kavita Kongara, M.D., Motility Clinical Chair and Georgia Physician Executive Committee Member, United Digestive, Physician, Atlanta Gastroenterology Associates. “It’s crucial that patients engage in open, honest conversations with their healthcare providers to explore all available therapeutic approaches. VOQUEZNA, a first-in-class PCAB, works differently than traditional medications and provides rapid and durable acid suppression, resulting in effective relief from heartburn and making it an important and differentiated option for GERD management.” As part of the campaign, Thompson is also taking on the comedic role of a chef in a “Kick Some Acid Cooking Show," a humorous nod to how VOQUEZNA has helped him better control his GERD-related heartburn. This multi-channel campaign will feature a new commercial airing next month on broadcast television and streaming platforms—including Prime Video, Hulu, and Peacock—and will also extend across digital platforms such as Facebook and Instagram, and in waiting rooms at doctors’ offices. The goal of the light-hearted campaign is to empower individuals experiencing persistent heartburn to consult their healthcare providers. “Phathom is passionate about supporting the millions of patients living with GERD and working to advance innovative treatments to address unmet needs," said Martin Gilligan, Chief Commercial Officer at Phathom. “We are excited to partner with Kenan Thompson, who has been making audiences laugh and feel good for decades. Kenan's story is inspiring and relatable to so many GERD sufferers. By sharing his journey for the first time, he’s helping to spark conversations and encourage others to talk to their doctors about VOQUEZNA.” To learn more about Thompson’s story, tips for discussing GERD with your healthcare provider, and VOQUEZNA as a treatment option, visit GERDIsNoJoke.com. A Media Snippet accompanying this announcement is available by clicking on this link. INDICATION AND IMPORTANT SAFETY INFORMATION What is VOQUEZNA? VOQUEZNA® (vonoprazan) is a prescription medicine used in adults: for 8 weeks to heal acid-related damage to the lining of the esophagus (called Erosive Esophagitis) and for relief of heartburn related to Erosive Esophagitis.for up to 6 months to maintain healing of Erosive Esophagitis and for relief of heartburn related to Erosive Esophagitis.for 4 weeks for relief of heartburn related to gastroesophageal reflux disease (GERD). It is not known if VOQUEZNA is safe and effective in children. Do not take VOQUEZNA if you: are allergic to vonoprazan or any of the other ingredients in VOQUEZNA. Allergic reaction symptoms may include trouble breathing, rash, itching, and swelling of your face, lips, tongue or throat.are taking a medicine that contains rilpivirine (EDURANT, COMPLERA, JULUCA, ODEFSEY) used to treat HIV-1 (Human Immunodeficiency Virus). Before taking VOQUEZNA, tell your healthcare provider about all your medical conditions, including if you: have low magnesium, calcium, or potassium in your blood, or you are taking a medicine to increase urine (diuretic).have kidney or liver problems.are pregnant, think you may be pregnant, or plan to become pregnant. It is not known if VOQUEZNA will harm your unborn baby.are breastfeeding or plan to breastfeed. It is not known if VOQUEZNA passes into your breast milk. You and your healthcare provider should decide if you will take VOQUEZNA or breastfeed. You should not do both. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine. VOQUEZNA may affect how other medicines work, and other medicines may affect how VOQUEZNA works. Especially tell your healthcare provider if you take medicine that contains rilpivirine (EDURANT, COMPLERA, JULUCA, ODEFSEY). What are the possible side effects of VOQUEZNA? VOQUEZNA may cause serious side effects including: A type of kidney problem (acute tubulointerstitial nephritis): Some people who take VOQUEZNA may develop a kidney problem called acute tubulointerstitial nephritis. Call your healthcare provider right away if you have a decrease in the amount that you urinate or if you notice blood in your urine.Diarrhea caused by an infection (Clostridioides difficile) in your intestines: Call your healthcare provider right away if you have watery stools, stomach pain, and fever that does not go away.Bone fractures (hip, wrist, or spine): Bone fractures in the hip, wrist, or spine may happen in people who take multiple daily doses of another type of medicine that reduces acid in your stomach known as proton pump inhibitors (PPI medicines) for a long period of time (a year or longer). Tell your healthcare provider if you have a bone fracture, especially in the hip, wrist, or spine.Severe skin reactions: VOQUEZNA can cause rare, but severe skin reactions that may affect any part of your body. These serious skin reactions may need to be treated in a hospital and may be life threatening: Skin rash which may have blistering, peeling, or bleeding on any part of your skin.You may also have fever, chills, body aches, shortness of breath, or enlarged lymph nodes. If you experience any of these symptoms, stop taking VOQUEZNA and call your healthcare provider right away. These symptoms may be the first sign of a severe skin reaction. Low vitamin B-12 levels: VOQUEZNA lowers the amount of acid in your stomach. Stomach acid is needed to absorb Vitamin B12 properly. Tell your healthcare provider if you have symptoms of low vitamin B12 levels, including irregular heartbeat, shortness of breath, lightheadedness, tingling or numbness in the arms or legs, muscle weakness, pale skin, feeling tired, or mood changes. Talk with your healthcare provider about the risk of low vitamin B12 levels if you have been on VOQUEZNA for a long time.Low magnesium levels in the body can happen in people who take VOQUEZNA. Tell your healthcare provider right away if you have symptoms of low magnesium levels, including seizures, dizziness, irregular heartbeat, jitteriness, muscle aches or weakness, or spasms of the hands, feet, or voice.Stomach growths (fundic gland polyps): A certain type of stomach growth called fundic gland polyps may happen in people who take another type of medicine that reduces acid in your stomach known as proton pump inhibitors (PPI medicines) for a long time. Talk with your healthcare provider about the possibility of fundic gland polyps if you have been on VOQUEZNA for a long time. The most common side effects of VOQUEZNA for treatment of Erosive Esophagitis and/or relief of heartburn related to gastroesophageal reflux disease include: • stomach inflammation• indigestion• diarrhea• constipation• stomach bloating• high blood pressure• stomach pain• urinary tract infection• nausea These are not all the possible side effects of VOQUEZNA. For more information, ask your healthcare provider or pharmacist. Call your healthcare provider for medical advice about side effects. You are encouraged to report suspected adverse reactions by contacting Phathom Pharmaceuticals at 1-888-775-PHAT (7428) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Please see Patient Information and full Prescribing Information for VOQUEZNA. About Erosive and Non-Erosive Gastroesophageal Reflux Disease (GERD)Erosive GERD, also referred to as Erosive Esophagitis (EE), is a subtype of gastroesophageal reflux disease (GERD) characterized by erosions in the gastric mucosa caused by acidic reflux of stomach contents into the esophagus.4 There are estimated to be over 65 million individuals with GERD in the U.S.,2 of which approximately 30% have Erosive GERD.3 In addition to experiencing troubling symptoms including heartburn, patients with inadequately treated Erosive GERD may progress to more severe diseases including Barrett’s esophagus and esophageal cancer.5 Non-Erosive GERD is the largest category of GERD and is characterized by reflux-related symptoms in the absence of esophageal mucosal erosions.3 It is estimated that approximately 70% of the overall GERD population have Non-Erosive GERD.3 Symptoms of Non-Erosive GERD may impact overall quality of life and can include episodic heartburn, especially at night, regurgitation, problems swallowing, and chest pain.2,3 About VOQUEZNA®VOQUEZNA® (vonoprazan) tablets contain vonoprazan, an oral small molecule potassium-competitive acid blocker (PCAB). PCABs are a novel class of medicines that block acid secretion in the stomach. VOQUEZNA is approved in the U.S. for the treatment of adults with Erosive Esophagitis, also known as Erosive GERD, the relief of heartburn associated with Erosive GERD, the relief of heartburn associated with Non-Erosive GERD, and for the treatment of H. pylori infection in combination with either amoxicillin or amoxicillin and clarithromycin. Phathom in-licensed the U.S. rights to vonoprazan from Takeda, which markets the product in Japan and numerous other countries in Asia and Latin America. About Phathom Pharmaceuticals, Inc.Phathom Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of novel treatments for gastrointestinal diseases. Phathom has in-licensed the exclusive rights to vonoprazan, a first-in-class potassium-competitive acid blocker (PCAB) that is currently marketed in the United States as VOQUEZNA® (vonoprazan) tablets for the relief of heartburn associated with Non-Erosive GERD in adults, the healing and maintenance of healing of Erosive GERD in adults and relief of associated heartburn, in addition to VOQUEZNA® TRIPLE PAK® (vonoprazan tablets, amoxicillin capsules, clarithromycin tablets) and VOQUEZNA® DUAL PAK® (vonoprazan tablets, amoxicillin capsules) for the treatment of H. pylori infection in adults. For more information about Phathom, visit the company’s website at www.phathompharma.com and follow on LinkedIn and X. Forward-Looking StatementsThis press release contains forward-looking statements. Investors are cautioned not to place undue reliance on these forward-looking statements, including statements about the Company’s estimates of the number of patients with GERD. The inclusion of forward-looking statements should not be regarded as a representation by Phathom 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 Phathom’s business, including, without limitation: the Company’s estimates regarding patient population could prove to be inaccurate and other risks described in the Company’s prior press releases and the Company’s filings with the Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in the Company’s most recent Annual Report on Form 10-K and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Phathom undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. References VOQUEZNA. Prescribing Information. Phathom Pharmaceuticals; 2024.Machicado JD, Greer JB, Yadav D. (2021) Epidemiology of Gastrointestinal Diseases. In: Pitchumoni CS, Dharmarajan T, (eds). Geriatric Gastroenterology. Springer, Cham. https://doi.org/10.1007/978-3-030-30192-7_7Antunes C, Aleem A, Curtis SA. Gastroesophageal reflux disease. NCBI Bookshelf. Accessed January 21, 2024. Available at: https://www.ncbi.nlm.nih.gov/books/NBK441938/?report=printableDickman R, Maradey-Romero C, Gingold-Belfer R, Fass R. Unmet Needs in the Treatment of Gastroesophageal Reflux Disease. J Neurogastroenterol Motil. 2015 Jul 30;21(3):309-19. doi: 10.5056/jnm15105. Savarino E, de Bortoli N, De Cassan C, et al. The natural history of gastro-esophageal reflux disease: A comprehensive review. Dis Esophagus. 2017;30(2):1-9. MEDIA CONTACTNick Benedetto1-877-742-8466media@phathompharma.com INVESTOR CONTACTEric Sciorilli1-877-742-8466ir@phathompharma.com © 2025 Phathom Pharmaceuticals. All rights reserved. VOQUEZNA, VOQUEZNA TRIPLE PAK, VOQUEZNA DUAL PAK, Phathom Pharmaceuticals, and their respective logos are registered trademarks of Phathom Pharmaceuticals, Inc. 03/25US-VPZ-24-0652