Phathom Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) has approved VOQUEZNA® (vonoprazan) tablets 10 mg and 20 mg, a novel potassium-competitive acid blocker (PCAB), as a new treatment for adults for the healing of all grades of Erosive Esophagitis, also known as Erosive GERD (gastroesophageal reflux disease), maintenance of healing of all grades of Erosive GERD, and relief of heartburn associated with Erosive GERD. Erosive GERD, also referred to as Erosive Esophagitis or Erosive Acid Reflux, is a major type of GERD that affects approximately 20 million people in the U.S. In addition to experiencing troubling heartburn symptoms, patients with inadequately treated Erosive GERD may develop more severe diseases including Barrett?s esophagus, a condition in which esophageal tissue changes can progress to cancer. This approval is based on positive results from the Phase 3 PHALCON-EE study (NCT04124926).

The pivotal trial was a randomized, double-blind, multicenter study that enrolled 1,024 patients with Erosive GERD in the U.S. and Europe and compared VOQUEZNA to the PPI lansoprazole in the healing and maintenance of healing of Erosive GERD and associated heartburn symptom relief. Results showed that VOQUEZNA 20 mg met the primary endpoint of non-inferiority (p<0.0001) for complete healing by Week 8 in patients with all grades of Erosive GERD with a healing rate of 93% compared to 85% for lansoprazole 30 mg, with superior rates of healing demonstrated in a secondary endpoint in patients with moderate-to-severe disease (LA Grade C/D) at Week 2 compared to lansoprazole (70% for VOQUEZNA 20 mg and 53% for lansoprazole 30 mg) (p=0.0008). VOQUEZNA 20 mg also demonstrated non-inferiority to lansoprazole 30 mg in the mean percentage of 24-hour heartburn free days over the healing period.

In the maintenance phase of the trial, VOQUEZNA 10 mg was superior to lansoprazole 15 mg in maintaining healing at six months in all randomized patients (79% for VOQUEZNA 10 mg, compared to 72% for lansoprazole 15 mg) as well as in the subset of patients with moderate-to-severe Erosive GERD (75% for VOQUEZNA 10 mg, compared to 61% for lansoprazole 15 mg) (p=0.0490). In addition, VOQUEZNA 10 mg was evaluated as a secondary endpoint for relief of heartburn in Erosive GERD patients and demonstrated non-inferiority to lansoprazole 15 mg over six months. Adverse event (AE) rates for VOQUEZNA were comparable to lansoprazole in the trial.

The most common AEs in the healing phase (= 2% in the VOQUEZNA treatment arm) were gastritis (3.0% for VOQUEZNA 20 mg and 2.0% for lansoprazole 30 mg), diarrhea (2.0% for VOQUEZNA 20 mg and 3.0% for lansoprazole 30 mg), abdominal distension (2.0% for VOQUEZNA 20 mg and 1.0 % for lansoprazole 30 mg), abdominal pain (2.0% for VOQUEZNA 20 mg and 1.0% for lansoprazole 30 mg) and nausea (2.0% for VOQUEZNA 20 mg and 1.0% for lansoprazole 30 mg). The most common AEs in the maintenance phase (= 3% in the VOQUEZNA treatment arm) for VOQUEZNA 10 mg compared to lansoprazole 15 mg were gastritis (6.0% vs. 3.0%), abdominal pain (4.0% vs.

2.0%), dyspepsia (4.0% vs. 3.0%), hypertension (3.0% vs. 2.0%), and urinary tract infection (3.0% vs.

2.0%). These are not all of the potential side effects associated with the use of VOQUEZNA. Please see Important Safety information below and the full Prescribing Information for VOQUEZNA for more information.

VOQUEZNA is expected to be available in the U.S. in December 2023 and will be marketed exclusively by Phathom Pharmaceuticals, Inc. Based on the terms of Phathom?s revenue interest financing agreement, the FDA approval of VOQUEZNA for Erosive GERD also entitles the company to receive a $175.0 million payment. This non-dilutive capital will help fund the commercial launch.