- Initiation of registrational Phase 3 study of VOQUEZNA® (vonoprazan) for as-needed (PRN) use in Non-Erosive Reflux Disease (NERD) builds on previously reported positive Phase 2 data
- Topline results expected in the first quarter of 2028
- pHalcon-NERD-302, if successful, is intended to support a potential VOQUEZNA label expansion to include as-needed treatment of heartburn episodes in patients with Non-Erosive GERD, a treatment regimen of interest to patients for which proton pump inhibitors (PPIs) are not currently approved in the U.S.
FLORHAM PARK, N.J., Sept. 29, 2026 (GLOBE NEWSWIRE) -- Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on the development and commercialization of novel treatments for gastrointestinal diseases, today announced the initiation of patient screening in pHalcon-NERD-302, a registrational Phase 3 study evaluating an as-needed (PRN or on-demand) investigational dosing regimen of VOQUEZNA® (vonoprazan) tablets in adults with Non-Erosive Gastroesophageal Reflux Disease, or Non-Erosive GERD (NERD). Topline results are expected in the first quarter of 2028. If successful, the study is intended to support a potential regulatory submission seeking a label expansion for VOQUEZNA tablets for this treatment regimen.
“With pHalcon-NERD-302, we are studying what we believe could be an important new treatment approach for patients with Non-Erosive GERD. The study is designed to assess whether as-needed VOQUEZNA can provide rapid relief of heartburn when symptoms occur, with relief sustained over 24 hours. We have heard from many physicians and patients about the desire for an as-needed option. If successful, this study could support a potential label expansion for VOQUEZNA and an important opportunity to potentially broaden how VOQUEZNA may be used in the future to meet the needs of patients with Non-Erosive GERD,” said Steve Basta, President and Chief Executive Officer of Phathom Pharmaceuticals.
VOQUEZNA Phase 3 As-Needed ProgramBuilding on previously reported positive Phase 2 results, pHalcon-NERD-302 is a multicenter, randomized, double-blind, placebo-controlled Phase 3 study investigating VOQUEZNA 10 mg as an as-needed treatment in adults with Non-Erosive GERD. The Phase 3 pHalcon-NERD-302 study is expected to enroll approximately 500 adults with Non-Erosive GERD who report heartburn at least four days per week. The study is planned to be conducted at approximately 80 sites in the U.S. Following a four-week, open-label run-in period with VOQUEZNA 10 mg once daily, patients without heartburn during the final seven days of the run-in period will be randomized 1:1 to receive VOQUEZNA 10 mg or placebo, as-needed, for 24 weeks of treatment. The primary endpoint, assessed at Week 12, is the percentage of heartburn episodes in which patients experience complete relief within two hours and sustained relief for 24 hours post-dosing.
“Non-Erosive GERD is characterized by episodic, often unpredictable heartburn. Proton-pump inhibitor acid-suppressing therapies generally rely on continuous daily use,” said Ronnie Fass, M.D., Division Director of Gastroenterology and Hepatology and Medical Director of the Digestive Health Center at MetroHealth Medical Center, Professor of Medicine at Case Western Reserve University School of Medicine, and an investigator for pHalcon-NERD-302. “An as-needed treatment approach would need to provide acid suppression that starts rapidly and is sustained long enough to control heartburn over 24 hours. The results from the earlier Phase 2 as-needed study of VOQUEZNA provide a strong rationale for evaluating its efficacy and safety in this Phase 3 trial.”
“One of the practical questions in managing Non-Erosive GERD is whether patients with intermittent symptoms need continuous daily therapy. This registrational study is designed to evaluate whether an as-needed treatment approach can provide effective heartburn relief when symptoms occur,” said Philip O. Katz, M.D., Professor of Medicine in the Division of Gastroenterology and Hepatology at Weill Cornell Medicine, gastroenterologist at NewYork-Presbyterian/Weill Cornell Medical Center, and an investigator for pHalcon-NERD-302. Dr. Katz serves as a consultant for Phathom Pharmaceuticals.
For more information about the pHalcon-NERD-302 study (NCT07752407), visit ClinicalTrials.gov.
About Non-Erosive Gastroesophageal Reflux DiseaseNon-Erosive Gastroesophageal Reflux Disease (NERD) is the largest subcategory of gastroesophageal reflux disease (GERD) and is characterized by reflux-related symptoms in the absence of esophageal mucosal erosions. More than 22 million U.S. patients are estimated to be treated annually with prescription medication for GERD, and approximately 15 million of these patients are believed to have NERD. Symptoms of NERD may impact overall quality of life and can include episodic heartburn, regurgitation, problems swallowing, and chest pain.
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 (