PPI

FLORHAM PARK, N.J., Oct. 27, 2024 (GLOBE NEWSWIRE) -- Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal (GI) diseases, today announced that the company will present data from multiple investigational studies for its first-in-class treatment VOQUEZNA® (vonoprazan) tablets at the American College of Gastroenterology (ACG) 2024 Annual Scientific Meeting, being held October 25-30 in Philadelphia, PA. VOQUEZNA is approved for the relief of heartburn associated with Non-Erosive Gastroesophageal Reflux Disease (Non-Erosive GERD), for the treatment of all severities of Erosive Esophagitis, commonly referred to as Erosive GERD, and relief of related heartburn, and in combination with antibiotics for the eradication of Helicobacter pylori (H. pylori) infection.1 VOQUEZNA is the first and only U.S. Food and Drug Administration (FDA)-approved potassium-competitive acid blocker (PCAB).2 Evaluating On-Demand VOQUEZNA Treatment from the PHALCON-NERD-201 Trial3In an oral presentation being presented Monday, October 28 at ACG, Ronnie Fass, M.D., director of the Division of Gastroenterology and Hepatology and medical director of the Digestive Health Center at MetroHealth in Cleveland, Ohio, and lead investigator of the PHALCON-NERD-201 trial, will provide results from a post-hoc analysis of the Phase 2 study. This research, recognized with the ACG Outstanding Research Award in the ‘Esophagus’ category, evaluated the efficacy of As Needed (On-Demand) dosing of VOQUEZNA, compared to placebo, for relief of episodic heartburn in participants with Non-Erosive GERD following a 4-week VOQUEZNA daily dosing “run-in” period. As previously reported, the primary analysis of the study demonstrated that all three VOQUEZNA doses tested (10 mg, 20 mg and 40 mg) had higher percentages of heartburn completely relieved within three hours of dosing and sustained for 24 hours, with significant differences reported as early as one hour. Use of As Needed dosing in this population is investigational as VOQUEZNA has been neither evaluated nor approved by the FDA as an As Needed treatment for Non-Erosive GERD. To understand the practical application of As Needed dosing for VOQUEZNA, the new analysis examined daily heartburn symptom burden at baseline, during daily VOQUEZNA treatment, and upon switching to As Needed treatment after achieving symptom control during the run-in period. It also further evaluated the rate of symptom improvement upon treating new onset heartburn episodes during the As Needed dosing period.3 “As a healthcare provider, the concept of a novel As Needed dosing option for Non-Erosive GERD is highly compelling. I am pleased that ACG has recognized this data as a recipient of the ACG Outstanding Research Award, further reinforcing its importance to the GI community,” said Ronnie Fass, M.D. “The low frequency of heartburn after discontinuing daily VOQUEZNA treatment, coupled with the rapid onset of improvement following As Needed dosing, suggests that transitioning from daily dosing to As Needed treatment is a viable option for patients who previously respond well to daily therapy of VOQUEZNA. These promising results warrant further investigation in a larger and extended As Needed study period.” The double-blind, placebo-controlled PHALCON-NERD Phase 2 study randomized 207 patients who were eligible for On-Demand (“As Needed”) treatment following a 4-week daily dosing run-in period in which patients received VOQUEZNA 20 mg once-daily. Patients without heartburn in the last seven days of the run-in period were evenly randomized to receive 10 mg, 20 mg or 40 mg of VOQUEZNA, or placebo, for six weeks. Results of the study were published in Alimentary Pharmacology & Therapeutics in September 2023.4 Results:3 Patients eligible for the On-Demand period (n=207) were highly symptomatic with a reported mean of 16.1% [95% CI: (13.5%, 18.7%)] heartburn-free days during screening. Heartburn-free days increased during the run-in period to a mean of 82.9% [95% CI: (80.4%, 85.4%)] and remained well above pre-treatment levels (means 71% to 75%) throughout the six-week On-Demand period.The difference in the improvement of treated heartburn episodes between the active and placebo groups became evident within the first hour of dosing (10 mg [75.5%, p<0.0001], 20 mg [69.1%, p = 0.0010], 40 mg [75.5%, p<0.0001], placebo [57.0%]).Over 90% of heartburn episodes treated with VOQUEZNA improved within two hours. Non-Erosive GERD is the largest category of GERD and is characterized by reflux-related symptoms in the absence of esophageal mucosal erosions. An estimated 45 million U.S. adults are living with Non-Erosive GERD, and approximately 15 million are treated with a prescription medicine annually. Despite longstanding treatment options, many patients continue to suffer from heartburn symptoms. As Needed dosing is a unique and differentiated dosing regimen for which proton pump inhibitors (PPIs), a mainstay of GERD treatment, are not approved in the U.S.5 In July 2024, VOQUEZNA was approved by the FDA as a daily dosing treatment for the relief of heartburn associated with Non-Erosive GERD in adults.1 Nocturnal GERD Symptoms in Non-Erosive GERD6 In a poster presentation on Monday, October 28 at ACG, which has received ACG’s Presidential Poster Award, Dr. Catiele Antunes, M.D., Yale New Haven Hospital, will present data looking at the common, yet infrequently evaluated, nocturnal symptoms among patients with Non-Erosive GERD. The study included an exploratory analysis of a validated patient-reported outcomes scale, the Nocturnal Gastro-esophageal reflux disease Symptom Severity and Impact Questionnaire (N-GSSIQ), to determine the severity, morning impact, and concern about nocturnal GERD. “We are very pleased to continue to add to the body of clinical research evaluating the potential benefits of VOQUEZNA for GERD patients,” said Eckhard Leifke, M.D., Chief Medical Officer at Phathom. “In our study, patients who experienced nocturnal GERD symptoms prior to VOQUEZNA treatment found effective and meaningful relief with daily dosing, reinforcing its potent and durable acid suppression profile in helping to address both daytime and nighttime heartburn. While these findings are exploratory, they show promise for the many individuals suffering from nocturnal GERD symptoms.” Results:6 Among 772 subjects, the mean percentage of heartburn-free nights during the screening period was 29.6%, 25.8% and 31.1%, for patients randomized to placebo, VOQUEZNA 10 mg and 20 mg, respectively.After 4 weeks, the least-square (LS) mean percentage of heartburn-free nights was significantly better with 59.9% for VOQUEZNA 10 mg (LS mean difference=16.5%, p<0.0001 vs. placebo), and 56.4% for VOQUEZNA 20 mg (LS mean difference=13.1%, p<0.0001 vs. placebo), compared to 43.3% for placebo. The median percentage of heartburn-free nights was 70.4% for VOQUEZNA 10 mg and 71.0% for VOQUEZNA 20 mg, compared to 45.5% for placebo.N-GSSIQ scores showed significant improvement with VOQUEZNA compared to placebo in total score (LS mean difference vs. placebo of -2.9 and -1.8 for VOQUEZNA 10 mg and 20 mg, respectively; p<0.005 for both comparisons), nocturnal symptom severity (LS mean difference vs. placebo of -5.4 and -3.5 for VOQUEZNA 10 mg and 20 mg; p<0.001 for both comparisons), and concern about nocturnal GERD (LS mean difference vs. placebo of -2.0 for both VOQUEZNA 10 mg and 20 mg; p<0.0001). In addition to these data presentations, Phathom will sponsor a product theater highlighting VOQUEZNA as an approved treatment for GERD and will also have a presence on the exhibit floor at booth #757 throughout the conference. Following the conclusion of ACG 2024, the abstracts will be posted to the publications and scientific section of the Phathom company website. VOQUEZNA is marketed exclusively by Phathom Pharmaceuticals, Inc. and is currently available via prescription. Please visit voqueznapro.com to learn more about VOQUEZNA. About Non-Erosive Gastroesophageal Reflux DiseaseNon-Erosive GERD is the largest category of GERD and is characterized by reflux-related symptoms in the absence of esophageal mucosal erosions. There are over 65 million U.S. patients living with GERD, and it is estimated that approximately 70% of this population have Non-Erosive GERD. 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.7,8 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 treatment of heartburn associated with Non-Erosive GERD in adults, the healing and maintenance of healing of Erosive GERD in adults and 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. INDICATIONS AND USAGE VOQUEZNA® (vonoprazan) is a potassium-competitive acid blocker (PCAB) indicated in adults: for the healing of all grades of Erosive Esophagitis (Erosive Gastroesophageal Reflux Disease or Erosive GERD) and relief of heartburn associated with Erosive GERD.to maintain healing of all grades of Erosive GERD and relief of heartburn associated with Erosive GERD.for the relief of heartburn associated with Non-Erosive GERD.in combination with amoxicillin and clarithromycin for the treatment of Helicobacter pylori (H. pylori) infection.in combination with amoxicillin for the treatment of H. pylori infection. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS VOQUEZNA is contraindicated in patients with a known hypersensitivity to vonoprazan or any component of VOQUEZNA, or in patients receiving rilpivirine-containing products. For information about contraindications of antibacterial agents (clarithromycin and amoxicillin) indicated in combination with VOQUEZNA, refer to the Contraindications section of the corresponding prescribing information. WARNINGS AND PRECAUTIONS Presence of Gastric Malignancy: In adults, symptomatic response to therapy with VOQUEZNA does not preclude the presence of gastric malignancy. Consider additional follow-up and diagnostic testing in patients who have a suboptimal response or an early symptomatic relapse after completing treatment with VOQUEZNA. In older patients, also consider endoscopy. Acute Tubulointerstitial Nephritis: Acute tubulointerstitial nephritis (TIN) has been reported with VOQUEZNA. If suspected, discontinue VOQUEZNA and evaluate patients with suspected acute TIN. Clostridioides difficile-Associated Diarrhea: Published observational studies suggest that proton pump inhibitors (PPIs) may be associated with an increased risk of Clostridioides difficile-associated diarrhea (CDAD), especially in hospitalized patients. VOQUEZNA may also increase the risk of CDAD. Consider CDAD in patients with diarrhea that does not improve. Use the shortest duration of VOQUEZNA appropriate to the condition being treated. CDAD has been reported with use of nearly all antibacterial agents. For more information specific to antibacterial agents (clarithromycin and amoxicillin) indicated for use in combination with VOQUEZNA, refer to Warnings and Precautions section of the corresponding prescribing information. Bone Fracture: Several published observational studies suggest that PPI therapy may be associated with an increased risk for osteoporosis-related fractures of the hip, wrist, or spine, especially in patients receiving high dose (multiple daily doses) and long-term therapy (a year or longer). Bone fracture, including osteoporosis-related fracture, has also been reported with vonoprazan. Use the shortest duration of VOQUEZNA appropriate to the condition being treated. Patients at risk for osteoporosis-related fractures should be managed according to the established treatment guidelines. Severe Cutaneous Adverse Reactions (SCAR): Severe cutaneous adverse reactions, including Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) have been reported with VOQUEZNA. Discontinue VOQUEZNA at the first signs or symptoms of SCAR or other signs of hypersensitivity and consider further evaluation. Vitamin B12 (Cobalamin) Deficiency: Long-term use of acid-suppressing drugs can lead to malabsorption of Vitamin B12 caused by hypo- or achlorhydria. Vitamin B12 deficiency has been reported postmarketing with vonoprazan. If clinical symptoms consistent with vitamin B12 deficiency are observed in patients treated with VOQUEZNA, consider further workup. Hypomagnesemia and Mineral Metabolism: Hypomagnesemia has been reported postmarketing with vonoprazan. Hypomagnesemia may lead to hypocalcemia and/or hypokalemia and may exacerbate underlying hypocalcemia in at-risk patients. Consider monitoring magnesium levels prior to initiation of VOQUEZNA and periodically in patients expected to be on prolonged treatment, in patients taking drugs that may have increased toxicity in the presence of hypomagnesemia or drugs that may cause hypomagnesemia. Treatment of hypomagnesemia may require magnesium replacement and discontinuation of VOQUEZNA. Consider monitoring magnesium and calcium levels prior to initiation of VOQUEZNA and periodically while on treatment in patients with a preexisting risk of hypocalcemia. Supplement with magnesium and/or calcium, as necessary. If hypocalcemia is refractory to treatment, consider discontinuing VOQUEZNA. Interactions with Diagnostic Investigations for Neuroendocrine Tumors: Serum chromogranin A (CgA) levels increase secondary to drug-induced decreases in gastric acidity. The increased CgA level may cause false positive results in diagnostic investigations for neuroendocrine tumors. Temporarily discontinue VOQUEZNA treatment at least 4 weeks before assessing CgA levels and consider repeating the test if initial CgA levels are high. Fundic Gland Polyps: Use of VOQUEZNA is associated with a risk of fundic gland polyps that increases with long-term use, especially beyond one year. Fundic gland polyps have been reported with vonoprazan in clinical trials and during postmarketing use with PPIs. Most patients who developed fundic gland polyps were asymptomatic and fundic gland polyps were identified incidentally on endoscopy. Use the shortest duration of VOQUEZNA appropriate to the condition being treated. ADVERSE REACTIONS: Healing of Erosive GERD: The most common adverse reactions (≥2% of patients in the VOQUEZNA arm) include gastritis (3%), diarrhea (2%), abdominal distention (2%), abdominal pain (2%), and nausea (2%). Maintenance of Healed Erosive GERD: The most common adverse reactions (≥3% of patients in the VOQUEZNA arm) include gastritis (6%), abdominal pain (4%), dyspepsia (4%), hypertension (3%), and urinary tract infection (3%). Relief of Heartburn Associated with Non-Erosive GERD: The most common adverse reactions (≥2% of patients in the VOQUEZNA arm) include abdominal pain (2%), constipation (2%), diarrhea (2%), nausea (2%), and urinary tract infection (2%). Treatment of H. Pylori Infection (VOQUEZNA and Amoxicillin): The most common adverse reactions (≥2% in any treatment arm) include diarrhea (5%), abdominal pain (3%), vulvovaginal candidiasis (2%), nasopharyngitis (2%), dysgeusia (1%), headache (1%), and hypertension (1%). Treatment of H. Pylori Infection (VOQUEZNA, Amoxicillin and Clarithromycin): The most common adverse reactions (≥2% in any treatment arm) include dysgeusia (5%), diarrhea (4%), vulvovaginal candidiasis (3%), headache (3%), abdominal pain (2%), hypertension (2%), and nasopharyngitis (<1%). For more information on adverse reactions and laboratory changes with amoxicillin or clarithromycin, refer to Adverse Reactions section of the corresponding prescribing information. DRUG INTERACTIONS VOQUEZNA has the potential for clinically important drug interactions, including interactions with drugs dependent on gastric pH for absorption, drugs that are substrates for certain CYP enzymes, and some diagnostic tests. Avoid concomitant use of VOQUEZNA with atazanavir or nelfinavir. See full Prescribing Information for more details about important drug interactions. Consult the labeling of concomitantly used drugs to obtain further information about interactions with vonoprazan. For information about drug interactions, contraindications, and warnings and precautions of antibacterial agents (amoxicillin or clarithromycin) indicated in combination with VOQUEZNA, refer to their corresponding prescribing information. USE IN SPECIFIC POPULATIONS Lactation: Breastfeeding is not recommended during treatment. Because of the potential risk of adverse liver effects shown in animal studies with vonoprazan, advise patients not to breastfeed during treatment with VOQUEZNA. Renal Impairment: For the healing of Erosive GERD, dosage reduction is recommended in patients with severe renal impairment (eGFR < 30 mL/min). Use of VOQUEZNA is not recommended for the treatment of H. pylori infection in patients with severe renal impairment. Hepatic Impairment: For the healing of Erosive GERD, dosage reduction is recommended in patients with moderate to severe hepatic impairment (Child-Pugh Class B and C). Use of VOQUEZNA is not recommended for the treatment of H. pylori infection in patients with moderate to severe hepatic impairment. 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 full Prescribing Information for VOQUEZNA. Forward-Looking StatementsThis press release contains forward-looking statements. Words such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential” or continue” and similar expressions, are intended to identify forward-looking statements. Investors are cautioned not to place undue reliance on these forward-looking statements, including statements about the ability of vonoprazan to relieve symptoms of GERD following As Needed dosing and to suppress nocturnal symptoms among patients with Non-Erosive 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: additional data related to vonoprazan to relieve symptoms of GERD following As Needed dosing may be inconsistent with the data produced as of the date hereof, and further analysis of existing data and analysis of new data may lead to conclusions different from those established as of the date hereof; the inherent risks of clinical development of vonoprazan; Phathom’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; regulatory developments in the United States and foreign countries; unexpected adverse side effects or inadequate efficacy of vonoprazan that may limit its development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; Phathom’s ability to obtain and maintain intellectual property protection and non-patent regulatory exclusivity for vonoprazan; Phathom’s estimates regarding patient population and commercial coverage 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.Data on file. Phathom Pharmaceuticals, Inc. Florham Park, NJFass R, et al. Heartburn Frequency and Symptom Improvement Rates of Treated Episodes when Switching From Daily to On-Demand Vonoprazan Treatment for Non-Erosive Reflux Disease; American College of Gastroenterology (ACG) 2024 abstract; October 25-30, Philadelphia, PA.Fass R, Vaezi M, Sharma P, et al. Randomised clinical trial: Efficacy and safety of on-demand vonoprazan versus placebo for non-erosive reflux disease. Accessed September 17, 2024. Available at: https://doi.org/10.1111/apt.17728Antunes 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=printableAntunes C, et al. Vonoprazan Improves Nocturnal Gastroesophageal Reflux Symptoms in Non-Erosive Reflux Disease; American College of Gastroenterology (ACG) 2024 abstract; October 25-30, Philadelphia, PA.Machiacado 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=printable MEDIA CONTACTNick Benedetto1-877-742-8466media@phathompharma.com INVESTOR CONTACTEric Sciorilli1-877-742-8466ir@phathompharma.com © 2024 Phathom Pharmaceuticals. All rights reserved.VOQUEZNA, VOQUEZNA DUAL PAK, VOQUEZNA TRIPLE PAK, Phathom Pharmaceuticals, and their respective logos are registered trademarks of Phathom Pharmaceuticals, Inc. 10/24US-VPZ-24-0557

LA JOLLA, Calif.--( BUSINESS WIRE )-- Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced that it will present data at the International League Against Epilepsy (ILAE) 15 th Annual European Epilepsy Congress which is taking place in Rome, Italy from September 7-11, 2024. “ ILAE is one of the premier medical organizations in the epilepsy space and we look forward to participating in this important meeting, which brings together key opinion leaders from across Europe and the rest of world. As we prepare for the initiation of our global Phase 3 program this year, this conference will help us continue to broaden our engagement with potential investigators and gain important insights,” stated Dr. Randall Kaye, Longboard’s Chief Medical Officer. “ We are pleased that our PACIFIC study open label extension interim data was accepted as a late breaker for this conference, and we look forward to discussing these data as well as additional data that highlights the potential of bexicaserin for individuals living with a broad range of Developmental and Epileptic Encephalopathies, or DEEs.” EVENT DETAILS: LATE BREAKER Title: Safety and Tolerability of Bexicaserin in Adolescents and Adults with Developmental and Epileptic Encephalopathies: Interim Results of the Phase 1b/2a PACIFIC Study Open-Label Extension (OLE) Event Type : In-Person Posters Poster Number : P082 Title: Efficacy and Safety of Bexicaserin (LP352) in Adolescent and Adult Participants with Developmental and Epileptic Encephalopathies: Results of The Phase 1b/2a PACIFIC Study Session : Platform Session – Drug Therapy Event Type : Platform Session Presentation Time : Sunday, September 8, 2024, from 12:30 PM – 12:38 PM CET Title: Bexicaserin has Negligible CYP Or P-glycoprotein Interaction Potential, Minimizing Therapeutic Complexity In Epilepsy Patients with a High Burden of Polypharmacy Event Type : In-Person Posters Poster Number : P321 Title: A Phase 1 Study Of 5-HT2C Superagonist LP352 Shows Robust Brain Penetration, a Strong Correlation Between Plasma and CSF Pharmacokinetics and QEEG Changes Reflecting Receptor Engagement Event Type : In-Person Posters Poster Number : P262 Title: Bexicaserin, A 5-HT2C Superagonist, has Broad Antiepileptic Activity in Preclinical Seizure Models Event Type : In-Person Posters Poster Number : P261 ABOUT LONGBOARD PHARMACEUTICALS Longboard Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases. Longboard is working to advance a portfolio of centrally acting product candidates designed to be highly selective for specific G protein-coupled receptors (GPCRs). Longboard’s small molecule product candidates are based on more than 20 years of GPCR research. Longboard plans to advance bexicaserin (LP352), an oral, centrally acting 5-hydroxytryptamine 2C (5-HT2C) receptor superagonist, with no observed impact on 5-HT2B and 5-HT2A receptor subtypes, into a global Phase 3 program. The FDA has granted Breakthrough Therapy designation for bexicaserin for the treatment of seizures associated with Developmental and Epileptic Encephalopathies (DEEs) for patients two years of age and older. Earlier this year, Longboard reported positive topline data from a Phase 1b/2a clinical trial (the PACIFIC Study) evaluating bexicaserin in participants with DEEs. Longboard is also evaluating LP659, an oral, centrally acting, sphingosine-1-phosphate (S1P) receptor subtypes 1 and 5 modulator, which is in development for the potential treatment of rare neuroinflammatory conditions. Longboard recently completed a Phase 1 single-ascending dose (SAD) clinical trial for LP659 in healthy volunteers. Bexicaserin and LP659 are investigational compounds that are not approved for marketing by the U.S. Food and Drug Administration (FDA) or any other regulatory authority. FORWARD-LOOKING STATEMENTS Certain statements in this press release are forward-looking statements that involve a number of risks and uncertainties. In some cases, you can identify forward-looking statements by words such as “focus”, “will”, “look forward to”, “prepare for”, “potential”, “working to”, “designed to”, “plans”, “potential” or the negative, plural or other tenses of these words, references to future dates or time periods, or other comparable language, and they may include, without limitation, statements about Longboard’s presentations, product candidates, clinical and preclinical programs, focus and work. For such statements, Longboard claims the protection of the Private Securities Litigation Reform Act of 1995. Actual events or results may differ materially from Longboard’s expectations. Factors that could cause actual results to differ materially from those stated or implied by Longboard’s forward-looking statements are disclosed in Longboard’s filings with the Securities and Exchange Commission (SEC). These forward-looking statements represent Longboard’s judgment as of the time of this release. Longboard disclaims any intent or obligation to update these forward-looking statements, other than as may be required under applicable law.

Merger with Trawsfynydd Therapeutics, Inc (“Trawsfynydd”) and concurrent private placement of $14 million (cash runway to support planned operations through year end), with recently achieved clinical milestones, put Traws on track to achieve key readouts for the clinical pipeline in H2 2024 and beyond Lead antiviral program, tivoxavir marboxil, for influenza, including avian flu, is dosing the first cohort in the Phase 1 dose extension study in AustraliaCOVID-19 program, ratutrelvir, completed the Phase 1 single-ascending dose (SAD) and multiple-ascending dose (MAD) study in Australia Oncology strategy includes investigator-sponsored trials (ISTs), planned to begin in H2 2024, adding a new dimension to the narazaciclib program, with continued investigator support for rigosertib Corporate update call planned for August 15, 2024 at 8:00 AM ET NEWTOWN, Pa., Aug. 15, 2024 (GLOBE NEWSWIRE) -- Traws Pharma, Inc. (“Traws” or “Traws Pharma”), a clinical stage biopharmaceutical company developing oral small molecules for respiratory viral diseases and cancer, today outlined recent business highlights and reported unaudited financial results for the second quarter ended June 30, 2024. The Company intends to host a conference call and live webcast on Thursday, August 15, 2024 at 8:00 AM ET. “The last several months have been transformational for Traws Pharma, with strategic and product development progress and an increase in capital,” said Werner Cautreels, PhD, Chief Executive Officer of Traws Pharma. “Completion of the merger agreement that created Traws Pharma expanded our investor base to include recognized healthcare investors, Orbimed and Torrey Pines, while broadening our portfolio to include compounds that we believe have best-in-class potential for influenza (flu), including bird flu, and COVID-19. In addition, ISTs are enhancing our multi-kinase inhibitors for cancer, narazaciclib and rigosertib.” “We expect the antiviral program to advance over the next 6+ months as we complete preparations to report Phase 1 results and initiate Phase 2 studies in flu and COVID-19. Preclinical data and topline data from SAD studies from our influenza candidate, tivoxavir marboxil, suggest that it has the potential to achieve our target product profile as a single dose treatment that is active against pandemic-potential viruses as well as oseltamivir and baloxavir resistant viruses,” continued Dr. Cautreels. “In addition, clinical-trial enabling studies for our COVID-19 candidate, ratutrelvir, suggest that it has the potential to be effective without the requirement for co-administration of a CYP-inhibitor, and active against virus strains that may be resistant to other approved agents such as nirmatrelvir. In the coming months, we look forward to completing the Phase 1 studies, finalizing the dosing plan and initiating the Phase 2 program for both tivoxavir marboxil and ratutrelvir. We believe these data could pave the way for key readouts in 2025.” Upcoming Milestones Tivoxavir marboxil -- Targets the influenza cap-dependent endonuclease, which is highly conserved across flu strains, including avian flu. The compound is intended to be administered as a single dose per treatment Q4 2024: Announcement of topline Phase 1 dose extension results from AustraliaQ4 2024/Q1 2025: Initiation of Phase 2 efficacy study Ratutrelvir – Targets Mpro (3CL protease). The compound does not require combination use of a CYP-inhibitor such as ritonavir Q4 2024: Announcement of topline results from Phase 1 SAD and MAD studiesQ4 2024/Q1 2025: Initiation of Phase 2 efficacy study Narazaciclib – our multi-kinase inhibitor, including CKD4+, with potential for use in multiple solid tumors H2 2024: Release topline results from our Phase 1/2 study at an upcoming medical meeting. In addition, we plan to announce the recommended Phase 2 dose (RP2D) and initiate ISTs in multiple myeloma, breast cancer and other indications Rigosertib – our multi-kinase inhibitor targeting cell cycle proteins including PLK-1, with potential for use in the ultra-rare disease, advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC, RDEB) Continue to support the IST-led program, including compassionate use for patients with RDEB Recent Developments Start of dosing in Phase 1 dose extension study for tivoaxavir marboxil, a placebo-controlled trial designed to evaluate three doses, including two doses from the successful Phase 1 dose escalation study (80 and 120 mg) and one new, increased dose (240 mg) in healthy volunteers. The study was designed to assess pharmacokinetics, pharmacodynamics and safety, to define the dosing plan for the upcoming Phase 2 efficacy study. The expected dosing schedule will be one single oral dose.Completion of a Phase 1 dose escalation study for ratutrelvir in Australia, a SAD/MAD study conducted in healthy volunteers. The study was designed to assess SAD (5 doses ranging from 15 mg to 600 mg) and MAD (2 doses, 150 mg, and 600 mg, daily for 10 days) and provide pharmacokinetics, pharmacodynamics and safety data to guide the dosing plan for the upcoming Phase 2 efficacy study. The expected dosing schedule will be one oral dose per day for 10 days.Completion of Phase 1/2 dose escalation studies for narazaciclib, evaluated as a monotherapy and in combination with letrozole in patients with recurrent metastatic low-grade endometrioid endometrial cancer and other gynecologic malignancies. The studies were designed to define the dose limiting toxicity (DLT) and maximally tolerated dose (MTD) of the combination, and the RP2D for further clinical trials. Topline data are are being analyzed.Presentation of further data from ongoing ISTs for rigosertib, shared at the Society for Investigative Dermatology (SID) held in July, 2024 which highlighted ongoing studies for RDEB-associated SCC, conducted at the University Hospital in Salzburg, Austria and Thomas Jefferson University, Philadelphia, PA.Completion of the merger with Trawsfynydd and concurrent $14 million private placement in April expanded the clinical portfolio of the combined entity to include two potentially best-in-class oral small molecule programs, for influenza and COVID-19, as well as oral two agents for solid tumor cancers. The transaction also enhanced the Company’s investor base, with the addition of Orbimed and Torrey Pines. Financial Results: Traws financial results for the quarter ended June 30, 2024 represent the first post-transaction quarterly report of the combined company. The financial results for the comparable period in 2023 represent a consolidated summary of the combined entity. For simplicity, the explanatory statements below provide a high-level summary of expenses for the quarter ended June 30, 2024. Cash, cash equivalents and short-term investments: As of June 30, 2024, the Company had cash, cash equivalents, and short-term investments of approximately $16.9 million, compared to cash, cash equivalents, and short-term investments of approximately $20.8 million at December 31, 2023. The company believes its cash balance is adequate to support planned operations through year end 2024. Acquired in-process R&D related to our transaction early in the second quarter resulted in a non-cash charge of $117.5 million. Research and development (R&D) expense for the three months ended June 30, 2024, totaled $4.0 million, compared to $2.5 million for the comparable period in 2023. Q2 2024 R&D expenses mainly reflect the cost of the Phase 1 SAD/MAD COVID-19 study, the Phase 1 extension study in flu, completion of the narazaciclib Phase 1/2 dose escalation study, regulatory expenses to support planned upcoming studies, and expenses related to stock-based compensation and restructuring costs related to the recent transaction. General and administrative (G&A) expense for the three months ended June 30, 2024, totaled $2.0 million compared to $2.2 million for the comparable period in 2023. Q2 2024 G&A expenses include stock-based compensation and restructuring costs related to the merger. Net loss: The net loss for the three months ended June 30, 2024 was $123.1 million, or $4.87 per basic and diluted common share, which reflects a non-cash charge of $117.5 million related to in-process R&D from Onconova’s April 2024 acquisition of Trawsfynydd. This compares with a net loss of $4.3 million, or $0.20 per basic and diluted common share, for the same period in 2023. Conference Call and Webcast Information Traws Pharma will host a conference call and webcast today, August 15, 2024, at 8:00 AM ET to discuss recent business progress and second quarter financial results. To access the call, please dial: 1 (877) 407-0789 (United States) or 1 (201) 689-8562 (International) and reference the conference ID “13748066”. To access the webcast, please click: Traws Pharma Corporate Update Call. The live and archived webcast can also be accessed by visiting the “Corporate Events & Presentations” tab of the Events and Presentations section of the Investor Relations page. A replay of the webcast will be archived for 90 days. About Traws Pharma, Inc. Traws Pharma is a clinical stage biopharmaceutical company developing oral small molecule therapies for the treatment of respiratory viral diseases and cancer. The viral respiratory disease program includes two potentially best-in-class oral small molecules in Phase 1 studies: tivoxavir marboxil, a novel oral antiviral drug candidate for influenza and avian flu, targeting the influenza cap-dependent endonuclease, and ratutrelvir, targeting Mpro (3CL protease). In the cancer program, Traws is developing the novel, proprietary multi-kinase CDK4-plus inhibitor narazaciclib, with potential for refractory endometrial cancer and potentially other solid tumor cancers, and rigosertib, multi-kinase inhibitor targeting cell cycle proteins including PLK-1, with potential for use in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC, RDEB). Traws Pharma is committed to delivering novel compounds for unmet medical needs using state-of-the-art drug development technology. With a focus on product safety and a commitment to patients in need or that are specifically vulnerable, we aim to build solutions for important medical challenges and alleviate the burden of viral infections and cancer. Forward-Looking StatementsSome of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties including statements regarding Traws Pharma, its respective businesses, the merger agreement with Trawsfynydd Therapeutics, Inc. and concurrent private placement and the use of proceeds from such financing and the Company’s financing strategies, as well as statements regarding the milestones, preclinical studies and clinical studies for its four product candidates, tivoxavir marboxil for influenza including avian flu, ratutrelvir for COVID-19, and narazaciclib and rigosertib for cancer, related to the design, timing and potential results and the timing of next steps. Traws has attempted to identify forward-looking statements by terminology including “believes”, “estimates”, “anticipates”, “expects”, “plans”, “intends”, “may”, “could”, “might”, “will”, “should”, “preliminary”, “encouraging”, “approximately” or other words that convey uncertainty of future events or outcomes. Although Traws believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Traws’ clinical trials, collaborations, merger integration, market conditions and those discussed under the heading “Risk Factors” in Traws’ filings with the Securities and Exchange Commission. Any forward-looking statements contained in this release speak only as of its date. Traws undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events. Traws Pharma Contact:Mark GuerinTraws Pharma, Inc.267-759-3680www.trawspharma.com Investor Contact:Bruce Mackle LifeSci Advisors, LLC646-889-1200bmackle@lifesciadvisors.com Traws Pharma, Inc.Consolidated Balance Sheets June 30, December 31, 2024 (unaudited) 2023Assets Current assets: Cash and cash equivalents$16,886,000 $20,821,000 Receivables 18,000 18,000 Prepaid expenses and other current assets 1,767,000 1,821,000 Total current assets 18,671,000 22,660,000 Property and equipment, net 14,000 22,000 Other non-current assets 1,000 1,000 Total assets$18,686,000 $22,683,000 Liabilities, redeemable convertible preferred stock and stockholders’ (deficit) equity Current liabilities: Accounts payable$6,174,000 $5,619,000 Accrued expenses and other current liabilities 3,509,000 3,375,000 Deferred revenue 227,000 226,000 Total current liabilities 9,910,000 9,220,000 Deferred revenue, non-current 2,677,000 2,791,000 Total liabilities 12,587,000 12,011,000 Commitments and contingencies Series C redeemable convertible preferred stock; $0.01 par value, 5,000,000 shares authorized; 12,472 and zero shares issued and outstanding at June 30, 2024 and December 31, 2023, respectively 111,619,000 — Stockholders’ (deficit) equity: Common stock, $0.01 par value, 125,000,000 shares authorized, 25,306,509 and 21,003,409 shares issued and outstanding at June 30, 2024 and December 31, 2023, respectively 253,000 210,000 Additional paid in capital 505,017,000 493,116,000 Accumulated deficit (610,757,000) (482,631,000)Accumulated other comprehensive loss (33,000) (23,000)Total stockholders’ (deficit) equity (105,520,000) 10,672,000 Total liabilities, redeemable convertible preferred stock and stockholders’ (deficit) equity$18,686,000 $22,683,000 Traws Pharma, Inc.Condensed Consolidated Statements of Operations (unaudited) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023Revenue$57,000 $57,000 $113,000 $113,000 Operating expenses: Acquired in-process research and development 117,464,000 — 117,464,000 — Research and development 3,964,000 2,456,000 5,876,000 6,536,000 General and administrative 1,977,000 2,211,000 5,333,000 4,324,000 Total operating expenses 123,405,000 4,667,000 128,673,000 10,860,000 Loss from operations (123,348,000) (4,610,000) (128,560,000) (10,747,000)Other income, net 205,000 360,000 434,000 722,000 Net loss$(123,143,000) $(4,250,000) $(128,126,000) $(10,025,000)Net loss per share, basic and diluted$(4.87) $(0.20) $(5.53) $(0.48)Basic and diluted weighted average shares outstanding 25,310,774 20,979,766 23,177,117 20,970,022