VOQUEZNA is now approved and available to treat the largest category of Gastroesophageal Reflux Disease (GERD)
VOQUEZNA met its primary endpoint in its Phase 3 pivotal trial by demonstrating a significant and rapid reduction of heartburn with daily treatment
VOQUEZNA represents the first major innovation in GERD treatment in over 30 years and the only FDA-approved treatment of its kind available in the U.S.
FLORHAM PARK, N.J., July 18, 2024 (GLOBE NEWSWIRE) – Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal (GI) diseases, announced the U.S. Food and Drug Administration (FDA) has approved VOQUEZNA® (vonoprazan) 10 mg tablets for the relief of heartburn associated with Non-Erosive Gastroesophageal Reflux Disease (Non-Erosive GERD) in adults. Non-Erosive GERD represents a substantial segment of the U.S. GERD population, affecting millions of individuals suffering from frequent heartburn. This is the third FDA approval for VOQUEZNA, which is also approved to treat all severities of Erosive Esophagitis (EE), also referred to as Erosive GERD, and in combination with antibiotics for the eradication of Helicobacter pylori (H. pylori) infection.
“Today marks a significant milestone for millions of GERD patients as we proudly announce the approval of VOQUEZNA for the treatment of Non-Erosive GERD,” said Terrie Curran, President, and Chief Executive Officer at Phathom. “For decades GERD sufferers had no new class of treatment to turn to in the U.S. This approval provides patients and healthcare providers with immediate access to the first and only FDA-approved treatment of its kind, from a new class of acid suppression therapy, and the power to help provide complete 24-hour heartburn-free days and nights. We are very excited to introduce VOQUEZNA to the broader GERD community and look forward to its potential to help change the way this disease is treated.”
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 living with Non-Erosive GERD, and approximately 15 million are treated with a prescription medicine annually. Despite longstanding treatment options, many patients remain dissatisfied with such therapies and continue to suffer from heartburn symptoms which may impact overall quality of life with episodic heartburn, occurring during the day and at night.
“Millions of patients with Non-Erosive GERD continue to suffer from heartburn despite current treatment options,” said Colin W. Howden, M.D., Professor Emeritus, University of Tennessee College of Medicine. “The pivotal study that led to this approval showed that VOQUEZNA significantly reduced heartburn episodes in patients with Non-Erosive GERD along with an established safety profile. Today’s approval of VOQUEZNA provides physicians with a novel, first-in-class treatment that can quickly and significantly reduce heartburn for many adult patients.”
This approval is supported by the positive results from the PHALCON-NERD-301 study (NCT05195528), a Phase 3 randomized, placebo-controlled, double-blind, multi-site U.S. study evaluating the efficacy and safety of VOQUEZNA for the daily treatment of adults with Non-Erosive GERD. The trial enrolled 772 adult patients with Non-Erosive GERD who experienced four or more days of heartburn per week, with the majority having six to seven days of heartburn per week, and compared patients treated with VOQUEZNA 10 mg to placebo in the relief of heartburn over four weeks. The trial also included a 20-week extension period where all patients received VOQUEZNA to evaluate long-term treatment.
In the pivotal trial, VOQUEZNA quickly and significantly reduced heartburn with daily treatment through week 4. VOQUEZNA demonstrated the power of more complete all-day and all-night heartburn-free days with significantly more 24-hour heartburn-free days through week 4 versus placebo, the primary endpoint. The mean percentage of heartburn-free days for patients taking VOQUEZNA was 45% versus 28% for placebo (p<0.001), and the median percentage of 24-hour heartburn-free days was 48% versus 17%, respectively. Improvements for those taking VOQUEZNA were also seen in the percentage of each of heartburn-free days and nights, in addition to the percentage of days without rescue antacid use. Results from the pivotal study were previously presented at Digestive Disease Week® (DDW) 2024 and also published in Clinical Gastroenterology and Hepatology.
The most common adverse reactions (≥2%) reported in patients treated with VOQUEZNA during the four-week placebo-controlled trial include abdominal pain, constipation, diarrhea, nausea, and urinary tract infection. Upper respiratory tract infection and sinusitis were also reported in patients who received VOQUEZNA in the 20-week extension phase of the trial.
Phathom offers savings programs for eligible patients who face coverage or affordability issues, including co-pay assistance for patients with commercial insurance. For more information, please visit: voquezna.com/savings.
VOQUEZNA is marketed exclusively by Phathom Pharmaceuticals, Inc. and is currently available via prescription. To learn more about VOQUEZNA, please visit: voquezna.com.
About PHALCON-NERD-301 Study
PHALCON-NERD-301 was a phase 3, randomized, double-blind, multicenter, 4-week study conducted in U.S. patients with heartburn related to Non-Erosive GERD. The primary endpoint was the percentage of days without daytime or nighttime heartburn (24-hour heartburn-free days) over the 4-week placebo-controlled treatment period. The trial also included a 20-week long-term extension period to further evaluate the treatment of VOQUEZNA. A total of 776 patients with Non-Erosive GERD who experienced four or more days of heartburn per week, with the majority having six to seven days of heartburn per week, were enrolled and randomized in the multisite U.S. trial.
About Non-Erosive Gastroesophageal Reflux Disease
Non-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.
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.
REDHILL BIOPHARMA R&D UPDATE
Newly Published Positive Phase 3 Data Demonstrates 64% Increased Efficacy with RedHill’s RHB-104 in Crohn’s Disease
RedHill Biopharma (Nasdaq: RDHL) announced the new publication of ground-breaking positive data from its Phase 3 Crohn’s disease study with RHB-104, showing that RHB-104 plus standard of care (SoC), targeting Mycobacterium avium subspecies paratuberculosis (MAP), was 64% more effective than SoC alone in the study, supporting the hypothesis of a Mycobacterial basis to the disease.
Newly published in the peer-reviewed journal Antibiotics, the 331-patient Phase 3 Crohn’s disease study data shows the primary endpoint of clinical remission at week 26 was achieved, with high statistical significance, in 36.7% (61/166) of orally administered RHB-104 plus standard of care (SoC) patients, compared to 22.4% (37/165) of placebo plus SoC patients (p=0.0048); Safety profile similar to placebo. Study conducted across more than 100 sites.
The advanced stage clinical data demonstrating the potential efficacy of oral RHB-104 triple antimicrobial therapy targeting Mycobacterium avium subspecies paratuberculosis (MAP,) supports the paradigm-changing hypothesis of a Mycobacterial basis to Crohn’s disease, where high unmet medical need exists.
“This ground-breaking data shows that RHB-104, which contains antimicrobial therapy directed against Mycobacterium paratuberculosis, or MAP, which typically infects cattle, appears to be effective for the treatment of Crohn’s disease – potentially opening a new avenue of therapy directed against its possible cause,” said Dr. David Y. Graham, Professor of Medicine and Molecular Virology and Microbiology at Baylor College of Medicine, the lead investigator of the study. Dr. Graham added: “It is particularly important to note that RHB-104 proved beneficial to patients receiving anti-TNF agents, corticosteroids or immunosuppressive agents, and may also have a role as an add-on therapy for patients not responding to their current treatment.”
The Crohn’s disease market was valued at more than $13 billion in 2023. Commonly used therapies in the treatment of Crohn’s include: Abbvie’s Humira® (adalimumab), Janssen’s Remicade® (infliximab) and Stelara® (ustekinumab), BMS’s Zeposia® (ozanimod) and Pfizer’s Xeljanz® (tofacitinib).
“This study was designed based on the hypothesis that infection with MAP is the primary cause of Crohn’s disease and that antimicrobial therapy designed to treat MAP would favorably influence the outcome of Crohn’s disease,” said study lead investigator, Dr. David Y. Graham, Professor of Medicine and Molecular Virology and Microbiology at Baylor College of Medicine and the Michael E. DeBakey Veterans Administration Hospital in Houston, Texas, USA. “We believe that this data shows that treatment with RedHill’s RHB-104 appears to be effective for the treatment of Crohn’s disease – this is important as an effective and safe oral therapy for Crohn’s could be highly beneficial to the patients and treating community. A unique feature of this Phase 3 study was that patients were permitted concomitant treatment with infliximab, adalimumab and/or corticosteroids at study entry. To our knowledge, no other similar Crohn’s trial has allowed tumor necrosis factor (TNF) agents to be continued throughout the treatment period. It is therefore also important to note that RedHill’s RHB-104 proved beneficial to patients receiving corticosteroids, immunosuppressive agents, or anti-TNF agents and may also have a role as an add-on therapy for patients not responding to their current treatment.”
“Crohn’s disease causes immense suffering to millions of patients globally and sometimes leads to death due to various complications. Recognizing this, RedHill is firmly committed to the RHB-104 potential paradigm-changing Phase 3-stage program. It has been more than 100 years since Scottish surgeon, Dalziel, first sowed the seeds of a possible link between MAP and Crohn’s. Evidence has grown of MAP involvement in the etiology of Crohn’s, being associated with immune signaling dysregulation and being identified in over 50% of Crohn’s patients,” said Dror Ben-Asher, RedHill’s Chief Executive Officer. “This makes the new peer-reviewed publication of these data highly exciting as Crohn’s is a terrible disease that remains steadfastly resistant to the search for a cure – despite the enormous research efforts made in the field of immunosuppression. We have a long-held conviction in the MAP approach – vindicated by this compelling data. Planning ahead, the advent of an accurate and reliable MAP diagnostic, allied to this proven triple antimicrobial therapeutic strategy, has the potential to change the treatment paradigm in inflammatory bowel disease (IBD). Accordingly, we have been pursuing creative partnership models to advance the development of RHB-104 and intend to update the market in due course.”
Learn more at:
redhillbio.com