| Indication | Eosinophilic Esophagitis |
| Drug | vonoprazan |
| Mechanism of Action | potassium-competitive acid blocker |
| Company | Phathom Pharmaceuticals, Inc. |
| Trial Phase | Phase 2 |
| Trial Acronym | pHalcon-EoE-201 |
| Category | Clinical Trial Event |
| Sub Category | Patient Enrollment Milestone |
| Q1 2026 Net Revenues | $58.3 million |
| Q1 2025 Net Revenues | $28.5 million |
| Year-over-Year Revenue Growth | 104% |
| Q1 2026 Operating Expenses | $61.8 million |
| Q1 2026 Non-GAAP Operating Expenses | $56.2 million |
| Q1 2026 Net Cash Usage | ~$15 million |
| Q1 2026 Net Loss | $30.4 million |
| Cash and Cash Equivalents (March 31, 2026) | $180.9 million |
| Net Proceeds from Public Equity Offering | $122.0 million |
| Debt Repayment | $55.8 million |
| Total VOQUEZNA Prescriptions to Date | ~1.35 million |
| Q1 2026 VOQUEZNA Prescriptions | ~268,000 |
| Q1 2026 VOQUEZNA Prescriptions Year-over-Year Growth | 115% |
| Q1 2026 Covered Prescriptions Year-over-Year Growth | 91% |
| Q1 2026 Prescriptions Covered by Insurance | ~63% |
| FY 2026 Net Revenues Guidance | $320–$345 million |
| FY 2026 Non-GAAP Operating Expenses Guidance | $235–$255 million |
| Expected Operating Profitability Timeline | Beginning Q3 2026 and for FY 2026 |
| Expected Cash Flow Positivity Timeline | 2027 |
| Topline Results Anticipation | Late Q4 2026 or early Q1 2027 |
| Licensed Territory | U.S., Europe, Canada |
| Combination Partner (Dual Pak) | amoxicillin |
| Combination Partner (Triple Pak) | amoxicillin, clarithromycin |
Phathom Pharmaceuticals Reports Strong Q1 2026 Revenue Growth and EoE Trial Progress
Phathom Pharmaceuticals reported strong financial results for Q1 2026, with net revenues more than doubling year-over-year to $58.3 million, driven by the commercial success of VOQUEZNA. The company achieved a net loss of $30.4 million and managed net cash usage of approximately $15 million, reflecting significant cost reductions. VOQUEZNA saw approximately 268,000 prescriptions filled in Q1, a 115% increase from the prior year. Phathom also provided a positive business update, maintaining its full-year 2026 financial guidance and anticipating operating profitability starting in Q3 2026. Additionally, the Phase 2 pHalcon-EoE-201 trial for VOQUEZNA in eosinophilic esophagitis is enrolling ahead of schedule, with topline results expected in late Q4 2026 or early Q1 2027.
- Phathom Pharmaceuticals demonstrated robust financial growth in Q1 2026, achieving $58.3 million in net revenues, a 104% increase compared to Q1 2025. The company significantly reduced operating expenses by $41.9 million year-over-year to $61.8 million, leading to a net loss of $30.4 million, a substantial improvement from $94.3 million in Q1 2025. This performance reflects effective cost management and a strategic pivot to GI.
- VOQUEZNA continues its strong market penetration, with approximately 1.35 million total prescriptions filled to date. In the first quarter of 2026 alone, 268,000 prescriptions were filled, marking a 115% increase over Q1 2025. The company noted strengthening prescription trends in late March and early April, with covered prescriptions growing 91% year-over-year and about 63% of Q1 prescriptions covered by insurance.
- The Phase 2 pHalcon-EoE-201 trial evaluating VOQUEZNA for eosinophilic esophagitis (EoE) is progressing ahead of schedule, with topline results now anticipated in late Q4 2026 or early Q1 2027. Phathom maintains its full-year 2026 financial guidance, projecting net revenues of $320–$345 million and non-GAAP operating expenses of $235–$255 million, with operating profitability expected to commence in Q3 2026 and for the full year.
Addressing Key Challenges in Eosinophilic Esophagitis Treatment
Current treatment approaches for eosinophilic esophagitis face significant challenges that impact both clinical practice and patient outcomes. These limitations span from insufficient evidence for established therapies to emerging safety concerns with newer treatment modalities. Understanding these constraints is essential for optimizing therapeutic strategies and advancing clinical care.
• Limited evidence base for swallowed topical steroids - Current recommendations are based on relatively few studies employing various doses and formulations, with a notable paucity of direct comparisons between available pharmacologic treatments
• Inadequate efficacy of low-dose maintenance therapy - Low-dose budesonide (0.25 mg twice daily) demonstrated limited effectiveness in maintaining remission, with esophageal eosinophil loads increasing from 0.4 to 31.8 eosinophils/high-power field over 50 weeks and poor symptom control
• Suboptimal treatment adherence - Studies report treatment adherence issues in 29.4% of patients, potentially compromising therapeutic outcomes and disease management
• Diagnostic delays due to symptom overlap - Similarity with other gastrointestinal disorders frequently delays accurate diagnosis, which may result in complications such as esophageal strictures
• Safety concerns with oral immunotherapy - Eosinophilic esophagitis develops as a complication in 12% of patients receiving oral immunotherapy for food allergy, with 79% experiencing allergic reactions during maintenance phase and anaphylaxis occurring in 33% of patients
• Limited treatment options for refractory cases - Patients with positive IgG4 histological staining exhibit significantly higher rates of endoscopic edema (16.1% vs. 2.1%) and greater need for second-line treatment (64.5% vs. 41.3%)
• Insufficient pediatric research - While several emerging biologics targeting inflammatory pathways are under investigation in adults, studies are notably lacking in much of the pediatric population
• Procedural limitations - Esophageal dilations serve only as adjunct therapy for fibrostenotic disease and do not address the underlying inflammatory process
Unpacking the pHalcon-EoE-201 Trial Design for VOQUEZNA
Recent clinical trials for eosinophilic esophagitis have employed sophisticated study designs to evaluate novel therapeutic interventions. These studies have established standardized endpoints focusing on histologic remission, symptom control, and endoscopic improvements, with particular emphasis on the ≤6 eosinophils per high-power field threshold for histologic response.
| Study | Phase | Design | Sample Size | Duration | Primary Endpoints |
|---|---|---|---|---|---|
| MESSINA (Benralizumab) | 3 | Multicenter, double-blind, randomized, placebo-controlled | 211 patients (ages 12-65) | 24 weeks | Histologic response (≤6 eos/hpf); Change in DSQ score (0-84 scale) |
| LIBERTY EoE TREET (Dupilumab) | 3 | Randomized, double-blind, placebo-controlled | 239 patients (median age 24) | 52 weeks | Peak eosinophil count ≤6/hpf; Absolute change in DSQ score |
| Cendakimab Trial | 3 | Multicenter, multinational, randomized, double-blind | 399 patients (planned) | 48 weeks | Mean change in dysphagia days; Eosinophil histologic response ≤6/hpf |
| Etrasimod Trial | 2 | Double-blind, randomized | 108 patients (ages 18-65) | 24 weeks + 28-week extension | Percentage change in peak eosinophil count at week 16 |
| ESO-101 (Mometasone) | 2 | Randomized, placebo-controlled | 43 adults | 28 days | Absolute change in peak eosinophil count |
| EoE KIDS (Dupilumab) | 3 | Weight-tiered dosing study | Children aged 1-12 years | Up to 52 weeks | Weight-adjusted efficacy endpoints |
Understanding VOQUEZNA's Safety Profile for Future Indications
Published safety and tolerability data across multiple indications demonstrate that vonoprazan exhibits a generally favorable safety profile comparable to conventional proton pump inhibitors. Comprehensive analyses spanning from 2017 to 2026, including data from over 7,900 participants across various clinical trials, indicate that vonoprazan is well tolerated with manageable adverse event profiles across its therapeutic applications.
• Comparable overall safety to PPIs: A 2023 systematic review of 18 randomized controlled trials involving 7,932 participants showed no significant increase in adverse event incidence compared to PPIs (95% CI = 0.987-1.095, p = 0.141), with vonoprazan actually demonstrating superior tolerability in some aspects
• Consistent serious adverse event rates: Integrated analysis data from 14 clinical trials showed serious adverse event incidence rates of 10.39 per 100 person-years for vonoprazan versus 10.65 for PPIs, with four deaths occurring across studies, none considered related to study drug
• Mild and manageable common adverse events: The most frequently reported adverse events include abdominal pain, constipation, diarrhea, nausea, and dyspepsia, with nasopharyngitis being the only adverse event reported by ≥5% of patients (6.94% vonoprazan vs 5.07% PPI vs 4.49% placebo)
• Reversible hypergastrinemia without long-term sequelae: Mean serum gastrin levels were elevated during vonoprazan treatment compared to lansoprazole but normalized within 4 weeks after discontinuation, though this requires continued long-term safety monitoring
• Favorable gastrointestinal tolerability: Vonoprazan treatment was associated with statistically significant reduction in diarrhea and loose stools compared to PPIs (95% CI = 0.661-0.966, p = 0.021), with no significant differences in other gastrointestinal symptoms
• Minimal drug-drug interaction risk: Studies demonstrated no clinically meaningful interactions when co-administered with low-dose aspirin or NSAIDs, with vonoprazan not influencing antiplatelet aggregation effects of aspirin
• Low treatment discontinuation rates: Across multiple studies, treatment discontinuation due to adverse events was rare, with most reported adverse events being mild in severity and unrelated to study medication
VOQUEZNA's Ascent: Commercial Success and Strategic EoE Expansion
Phathom Pharmaceuticals' recent financial update paints a picture of a company gaining significant momentum, largely propelled by the impressive commercial uptake of VOQUEZNA. The doubling of revenues and substantial increase in prescriptions for this potassium-competitive acid blocker (P-CAB) highlight its growing acceptance and efficacy in the treatment landscape for acid-related disorders. VOQUEZNA's demonstrated superiority over traditional proton pump inhibitors (PPIs) in healing severe erosive esophagitis and managing PPI-refractory GERD positions it as a formidable player, offering patients a more potent and sustained acid suppression.
Beyond its current success, the company's strategic foresight is evident in its accelerated Phase 2 trial for VOQUEZNA in eosinophilic esophagitis (EoE). This move is particularly compelling given existing evidence suggesting vonoprazan's effectiveness in EoE patients who have not responded to PPIs. A positive outcome here could unlock a significant new market, transforming VOQUEZNA from a leading acid suppressant into a broader therapeutic agent for inflammatory gastrointestinal conditions. This expansion would not only diversify Phathom's portfolio but also address a critical unmet need in EoE management.
However, this promising trajectory is not without considerations. While the potential for market expansion into EoE is substantial, the uncertainty of clinical trial outcomes remains a key risk; Phase 2 results, though anticipated, are not guaranteed. Furthermore, as vonoprazan gains wider and longer-term use, the long-term safety profile will require continued scrutiny, particularly given early reports of gastric mucosal changes like white globe appearance lesions in some patients. The evolving competitive landscape within the P-CAB class also necessitates ongoing innovation and differentiation to maintain market leadership. Phathom's ability to navigate these factors will be crucial as it aims for sustained operating profitability and solidifies VOQUEZNA's role across a broader spectrum of gastrointestinal diseases.
Frequently Asked Questions
References
- [1] Al Hayek M, Lucendo AJ et al.. Comparative efficacy and safety of potassium-competitive acid blockers and proton pump inhibitors for erosive esophagitis: a network meta-analysis of randomized controlled trials. EClinicalMedicine. 2026 Mar. 41768276
- [2] Straumann A, Conus S et al.. Long-term budesonide maintenance treatment is partially effective for patients with eosinophilic esophagitis. Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association. 2011 May. 21277394
- [3] Han S, Choi HY et al.. Randomised clinical trial: safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple oral doses of tegoprazan (CJ-12420), a novel potassium-competitive acid blocker, in healthy male subjects. Alimentary pharmacology & therapeutics. 2019 Oct. 31437865
- [4] Thavamani A, Ryan MJ et al.. Safety and efficacy of a novel ultrathin gastroscope for unsedated transnasal endoscopy in children and adults for evaluation of upper GI disorders. iGIE : innovation, investigation and insights. 2024 Mar. 41648889
- [5] Falk GW, Pesek R. Pharmacologic Management of Eosinophilic Esophagitis. Immunology and allergy clinics of North America. 2024 May. 38575221
- [6] Han RS, Hao JW et al.. Efficacy and safety of triple therapy with vonoprazan for Helicobacter pylori eradication: A multicenter, prospective, randomized controlled trial. World journal of gastroenterology. 2025 Jul 28. 40741473
- [7] Wechsler JB, Hirano I. Biological therapies for eosinophilic gastrointestinal diseases. The Journal of allergy and clinical immunology. 2018 Jul. 29859203
- [8] Howden CW, Katz P et al.. Integrated Analysis of Vonoprazan Safety for Symptomatic Gastro-Oesophageal Reflux Disease or Erosive Oesophagitis. Alimentary pharmacology & therapeutics. 2025 Mar. 39720884
- [9] Kasai T, Suzuki S et al.. High Body Mass Index Is Correlated with the Success of Vonoprazan-Based Second-Line Therapy for Helicobacter Pylori Infection. The Tohoku journal of experimental medicine. 2021 Feb. 33536385
- [10] Huang KZ, Weber HC. Potassium-competitive acid blockers and acid-related disorders. Current opinion in endocrinology, diabetes, and obesity. 2024 Jun 1. 38483115
- [11] Smadi Y, Deb C et al.. Blind esophageal brushing offers a safe and accurate method to monitor inflammation in children and young adults with eosinophilic esophagitis. Diseases of the esophagus : official journal of the International Society for Diseases of the Esophagus. 2018 Dec 1. 29905784
- [12] Sugano K. [Vonoprazan: a novel potassium-competitive acid blocker]. Nihon rinsho. Japanese journal of clinical medicine. 2015 Jul. 26165074
- [13] Akazawa Y, Fukuda D et al.. Vonoprazan-based therapy for Helicobacter pylori eradication: experience and clinical evidence. Therapeutic advances in gastroenterology. 2016 Nov. 27803739
- [14] Meng X, Zhu X et al.. Efficacy and safety of proton pump inhibitors and H2 receptor antagonists in the initial non‑eradication treatment of duodenal ulcer: A network meta‑analysis. Experimental and therapeutic medicine. 2023 Jun. 37206569
- [15] Hwang I, Ji SC et al.. Randomised clinical trial: Safety, tolerability, pharmacodynamics and pharmacokinetics of zastaprazan (JP-1366), a novel potassium-competitive acid blocker, in healthy subjects. Alimentary pharmacology & therapeutics. 2023 Apr. 36732884
- [16] Pesek RD, Rothenberg ME. Eosinophilic gastrointestinal disease below the belt. The Journal of allergy and clinical immunology. 2020 Jan. 31669097
- [17] Macías E, Menéndez-Ramos A et al.. Esophageal tissue immunoglobulin G4 in eosinophilic esophagitis and its correlation with serum-specific IgG4 to six foods. Frontiers in allergy. 2026. 41668700
- [18] Zhou S, Xie L et al.. Keverprazan, a novel potassium-competitive acid blocker: Multiple oral doses safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy subjects. Clinical and translational science. 2023 Oct. 37533172
- [19] Rachid R, Keet CA. Current Status and Unanswered Questions for Food Allergy Treatments. The journal of allergy and clinical immunology. In practice. 2018 Mar-Apr. 29162426
- [20] Park KS, Kim HS et al.. Randomized, Double-Blind, Active-Controlled, Parallel, Phase 3 Clinical Trial for Evaluating the Efficacy and Safety of Zastaprazan in Patients with Gastric Ulcers. Gut and liver. 2026 Jan 16. 41540828




















