Phathom Pharmaceuticals, Inc., a biopharmaceutical company, focuses on developing and commercializing novel treatments for gastrointestinal, or GI, diseases.
The company’s approved products, VOQUEZNA, VOQUEZNA TRIPLE PAK, and VOQUEZNA DUAL PAK, contain vonoprazan, an oral small molecule potassium-competitive acid blocker, or PCAB. PCABs are a novel class of medicines that block acid secretion in the stomach. Vonoprazan is the first gastric anti-secretory agent from a novel class approved in the...
Phathom Pharmaceuticals, Inc., a biopharmaceutical company, focuses on developing and commercializing novel treatments for gastrointestinal, or GI, diseases.
The company’s approved products, VOQUEZNA, VOQUEZNA TRIPLE PAK, and VOQUEZNA DUAL PAK, contain vonoprazan, an oral small molecule potassium-competitive acid blocker, or PCAB. PCABs are a novel class of medicines that block acid secretion in the stomach. Vonoprazan is the first gastric anti-secretory agent from a novel class approved in the United States, Europe, or Canada in over 30 years; and has shown rapid, potent, and durable anti-secretory effects. Vonoprazan has also demonstrated clinical benefits over the current standard of care as a single agent in the treatment of erosive gastroesophageal reflux disease, or Erosive GERD, and in combination with antibiotics for the treatment of Helicobacter pylori, or H. pylori, infection. Takeda Pharmaceutical Company Limited, or Takeda, developed vonoprazan and has received marketing approval in numerous countries in Asia and Latin America, as well as Russia. In May 2019, the company in-licensed the U.S., European, and Canadian rights to vonoprazan from Takeda.
In May 2022, the U.S. Food and Drug Administration, or FDA, approved the NDAs for vonoprazan triple therapy, under the brand name VOQUEZNA TRIPLE PAK, and vonoprazan dual therapy, under the brand name VOQUEZNA DUAL PAK. Subsequently, on November 1, 2023, the FDA approved vonoprazan, under the brand name VOQUEZNA, as a treatment for adults for the healing of all grades of Erosive GERD, maintenance of healing of all grades of Erosive GERD, and relief of heartburn associated with Erosive GERD, as well as in combination with amoxicillin, with or without clarithromycin, for the treatment of H. pylori infection in adults. The company initiated commercial launch for VOQUEZNA for both the Erosive GERD and H. pylori indications, and VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK for the treatment of H. pylori infection in the fourth quarter of 2023. In September 2023, the company submitted an NDA seeking approval of vonoprazan as a once-daily treatment for heartburn symptoms associated with Non-Erosive GERD in adults. On July 17, 2024, the FDA approved VOQUEZNA 10 mg tablets for the relief of heartburn associated with Non-Erosive GERD, the largest category of GERD.
The company is independently commercializing VOQUEZNA, VOQUEZNA TRIPLE PAK, and VOQUEZNA DUAL PAK in the United States. The company’s commercial launch continues to build momentum and early launch data show strong physician and patient demand. As of February 21, 2025, over 300,000 prescriptions for VOQUEZNA tablets, VOQUEZNA Triple Pak, and VOQUEZNA Dual Pak had been filled since launch. These prescriptions were written by more than 20,000 prescribers. In addition, due to increasing commercial demand, the company continues to make progress in securing broad commercial coverage for VOQUEZNA, with over 120 million, or over 80%, of total U.S. commercial lives with access to VOQUEZNA tablets.
The company continues to evaluate potential commercial partnerships for vonoprazan in Europe and Canada, expand development of vonoprazan into other indications, dosing regimens, and alternative formulations and packaging, and evaluate the in-license or acquisition of additional clinical or commercial stage product candidates for the treatment of GI diseases in a capital-efficient manner.
In May 2021, the FDA granted qualified infectious disease product, or QIDP, designations for VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK, and the company thereby received an extension of five years of new chemical entity, or NCE, exclusivity based on the vonoprazan component in the applicable NDAs. In December 2024, the company submitted a citizen petition requesting that the FDA update the Orange Book listings to reflect the same ten-year period of NCE exclusivity for VOQUEZNA as reflected on the VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK Orange Book listings.
Vonoprazan has demonstrated clinical advantages over the PPI lansoprazole in the treatment of Erosive GERD and H. pylori infection in completed Phase 3 clinical trials conducted in the United States and Europe, including: more complete healing of Erosive GERD in patients with moderate to severe disease after two weeks; more durable healing of Erosive GERD in patients with all grades of disease; higher H. pylori eradication rates in combination with antibiotics compared to standard of care triple therapy; and more flexible dosing, including dosing independent of food and time of day.
Moreover, vonoprazan’s anti-secretory mechanism has the potential to contribute to additional clinical advantages over PPIs, such as rapid symptom relief through ‘as-needed’ dosing in the treatment of patients with Non-Erosive GERD.
Erosive GERD: The FDA approval of VOQUEZNA for the healing and maintenance of healing of all grades of erosive esophagitis and relief of heartburn associated with erosive esophagitis in adults was based on the results from PHALCON-EE, the company’s Phase 3 clinical trial conducted in the United States and Europe. This trial assessed vonoprazan versus lansoprazole in the healing and maintenance of healing of adult patients with Erosive GERD. In PHALCON-EE, vonoprazan met its primary healing endpoint, demonstrating non-inferiority to lansoprazole in the number of patients who showed complete healing of Erosive GERD after eight weeks of treatment. Further, in a pre-specified secondary endpoint, vonoprazan demonstrated superior healing after two weeks of treatment in patients with moderate to severe Erosive GERD compared to lansoprazole. After two weeks of treatment, 70% of patients with moderate to severe Erosive GERD were healed after treatment with vonoprazan versus 53% with lansoprazole (p=0.0008). In the maintenance phase of the trial, both doses of vonoprazan (10 mg and 20 mg) met the primary endpoint of non-inferiority compared to lansoprazole in the number of all patients who maintained healing of Erosive GERD through week 24. Further, both vonoprazan doses also met a pre-specified secondary endpoint demonstrating superiority of maintenance of healing versus lansoprazole (79% for vonoprazan 10 mg, 81% for vonoprazan 20 mg compared to 72% for lansoprazole 15 mg) (p<0.0001 for both non-inferiority comparisons; p=0.0436 for vonoprazan 10 mg superiority comparison; p=0.0272 for vonoprazan 20 mg superiority comparison). Both vonoprazan doses also met the pre-specified secondary endpoint of demonstrating superiority of the percentage of patients with moderate-to-severe disease who maintained healing of Erosive GERD through week 24 (75% vonoprazan 10 mg, 77% vonoprazan 20 mg v. 61% lansoprazole 15 mg) (p=0.0490 for vonoprazan 10 mg superiority comparison; p=0.0196 for vonoprazan 20 mg superiority comparison).
In PHALCON-EE, vonoprazan 20 mg met the secondary endpoint of showing non-inferiority to lansoprazole 30 mg in the mean percentage of 24-hour heartburn-free days over the healing period, and both vonoprazan doses met the secondary endpoint of showing non-inferiority to lansoprazole 15 mg in the mean percentage of 24-hour heartburn-free days over the maintenance period. Finally, vonoprazan 20 mg was also compared to lansoprazole 30 mg in a superiority test for onset of sustained resolution of heartburn by day three of the healing phase but did not achieve statistical significance (p=0.2196).
Non-Erosive gastroesophageal reflux disease (Non-Erosive GERD): In PHALCON-NERD-301, a Phase 3 study evaluating the efficacy and safety of vonoprazan for the daily treatment of adults with Non-Erosive GERD, both doses of vonoprazan (10 mg and 20 mg) met the primary endpoint evaluating the mean percentage of 24-hour heartburn-free days through week four by demonstrating statistical significance versus placebo (mean 45% vonoprazan 10 mg, 44% vonoprazan 20 mg, compared to 28% for placebo; p<0.0001 for both vonoprazan 10 mg and 20 mg versus placebo). The median percentage of 24-hour heartburn-free days was 48%, 46%, and 17% for vonoprazan 10 mg, vonoprazan 20 mg, and placebo, respectively. Patients randomized to vonoprazan 10 mg and 20 mg during the initial 4-week period remained on their blinded treatment assignment for a 20-week extension period. The mean percentage of heartburn-free days reported over the 20-week extension period for these patients was 63% for vonoprazan 10 mg and 61% for vonoprazan 20 mg. Additionally, patients randomized to placebo during the initial 4-week period were re-randomized to either vonoprazan 10 mg or 20 mg for the 20-week extension period. For these patients, the mean percentage of heartburn-free days reported over the 20-week extension period was 62% for vonoprazan 10 mg and 63% for vonoprazan 20 mg. Based on the results of PHALCON-NERD-301, on July 17, 2024, the FDA approved VOQUEZNA 10 mg tablets for the relief of heartburn associated with Non-Erosive GERD.
In PHALCON-NERD-201, a Phase 2 study evaluating three doses of vonoprazan (10 mg, 20 mg, and 40 mg) as an as-needed therapy for relief of episodic heartburn in subjects with Non-Erosive GERD, all three vonoprazan doses successfully met the primary endpoint evaluating the percentage of heartburn episodes completely relieved within three hours, with relief sustained for over 24 hours and were statistically significant (p<0.0001) when compared to placebo. Within three hours, vonoprazan 10 mg, 20 mg, and 40 mg achieved complete and sustained relief in 56%, 61%, and 70% of evaluable heartburn episodes, respectively, as compared to 27% of episodes for placebo. An evaluable heartburn episode is a heartburn episode for which the participant completes a minimum of one timed assessment after taking study medication.
With the company’s recent launch of VOQUEZNA for Non-Erosive GERD, the company has begun generating real-world data which are being reviewed to understand consumer usage patterns and prescribing habits of healthcare providers.
H. pylori: The FDA’s approval of VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK was based on the results from PHALCON-HP, the company’s Phase 3 clinical trial in the United States and Europe studying two vonoprazan-based treatment regimens for the eradication of H. pylori infection, both of which successfully met their primary and all secondary endpoints. The trial studied vonoprazan triple therapy and vonoprazan dual therapy compared to lansoprazole in combination with amoxicillin and clarithromycin, or lansoprazole triple therapy. The objective of the PHALCON-HP trial was to compare eradication rates in all treated subjects, as well as in two pre-identified subgroups of patients: those patients with clarithromycin-resistant strains of H. pylori, and those patients who did not have clarithromycin or amoxicillin-resistant strains of H. pylori. For regulatory purposes, the primary endpoint of this study was a non-inferiority comparison in the non-resistant subgroup for each of vonoprazan triple therapy and vonoprazan dual therapy compared to lansoprazole triple therapy.
In PHALCON-HP, both vonoprazan-based regimens successfully met their primary endpoints. In the modified intent-to-treat, or mITT, population, H. pylori eradication rates were 84.7% for vonoprazan triple therapy and 78.5% for vonoprazan dual therapy compared to 78.8% with lansoprazole triple therapy (p<0.0001 and p=0.0073, respectively, for non-inferiority). In the pre-specified per protocol population, a subset of the mITT population consisted of patients who were protocol compliant, H. pylori eradication rates were 90.4% with vonoprazan triple therapy and 81.2% with vonoprazan dual therapy compared to 82.1% with lansoprazole triple therapy (p<0.0001 and p=0.0155, respectively, for non-inferiority).
In PHALCON-HP, vonoprazan triple therapy and vonoprazan dual therapy also met all secondary endpoints, demonstrating superior eradication rates versus lansoprazole triple therapy in all patients and in the subgroup of patients with clarithromycin-resistant strains of H. pylori. Among all patients, the H. pylori eradication rate of vonoprazan triple therapy was superior to that of lansoprazole triple therapy in both the mITT population (80.8% vs. 68.5%; p=0.0001) and the per protocol population (85.7% vs. 70.0%; p<0.0001). In the subset of patients with H. pylori strains resistant to clarithromycin, the H. pylori eradication rate with vonoprazan triple therapy was superior to that of lansoprazole triple therapy in both the mITT population (65.8% vs. 31.9%; p<0.0001) and the per protocol population (67.2% vs. 29.0%; p<0.0001).
Among all patients, the H. pylori eradication rate of vonoprazan dual therapy was superior to that of lansoprazole triple therapy in both the mITT population (77.2% vs. 68.5%; p=0.0127) and the per protocol population (81.1% vs. 70.0%; p=0.0027). The H. pylori eradication rate of vonoprazan dual therapy was also superior to that of lansoprazole triple therapy in the subset of patients with H. pylori strains resistant to clarithromycin in both the mITT population (69.6% vs. 31.9%; p<0.0001) and the per protocol population (79.5% vs. 29.0%; p<0.0001).
Strategy
The company’s strategy is focused on continuing its commercial efforts of approved VOQUEZNA products and further developing vonoprazan as a first-in-class PCAB in the United States for the treatment of acid-related GI diseases. Key elements of this strategy are to continue its efforts to ensure the successful commercialization of VOQUEZNA, VOQUEZNA TRIPLE PAK, and VOQUEZNA DUAL PAK in the U.S.; further expand the development of vonoprazan across indications, including EoE, and alternative formulations and packaging; evaluate commercial partnerships to maximize the vonoprazan opportunity outside of the United States; and in-license or acquisition of additional clinical or commercial stage product candidates for the treatment of GI diseases in a capital-efficient manner.
Sales and Marketing
The company has established robust marketing, sales, and distribution capabilities to support the launch of its approved products, and it is independently commercializing VOQUEZNA, VOQUEZNA TRIPLE PAK, and VOQUEZNA DUAL PAK in the United States through an experienced national sales force. The company’s strategy is to target approximately 52,000 core high-volume PPI prescribers in the treatment of GERD and H. pylori infection. In the United States, the company sells primarily to pharmaceutical wholesale distributors, which are the principal means of distributing its products to healthcare providers.
The company also markets certain products through direct-to-consumer channels, including across streaming platforms, traditional broadcast and cable television, and consumer-facing digital channels, including Facebook, Instagram, waiting room TVs in doctors’ offices, and digital banner ads. Since launch, the campaign has reached millions of GERD sufferers and is driving strong patient engagement, increasing prescription requests, and motivating patients to explore a treatment option that works differently than PPIs.
Intellectual Property
As of December 31, 2024, the company’s patent portfolio covering vonoprazan consisted predominantly of exclusively licensed patents and patent applications from Takeda. Subject to the terms of the license agreement the company entered into with Takeda on May 7, 2019, or the Takeda License, the company has licensed from Takeda exclusive rights in the United States, Europe, and Canada to patents and patent applications covering the composition of matter, formulation, use, and/or manufacture of vonoprazan. The company’s patent portfolio comprises 11 distinct patent families protecting the technology relating to the compound vonoprazan and its synthetic intermediates, methods of synthesizing vonoprazan and related compounds, various formulations of vonoprazan products, as well as methods of treating diseases with vonoprazan and related compounds. As of December 31, 2024, the company’s portfolio consisted of approximately 25 issued U.S. patents, 4 pending U.S. applications, 16 issued European patents subsequently validated in individual European countries, 2 pending European applications, 6 issued Canadian patents, and 3 pending Canadian applications. The issued patents and pending applications have nominal expiration dates ranging from 2024 to 2038 without accounting for any available patent term adjustments or extensions. The issued U.S. patent covering the composition of matter of vonoprazan is expected to expire in August 2028, not including patent term extension. The issued U.S. patent covering the formulation of vonoprazan is expected to expire in August 2030, not including patent term extension.
License Agreement with Takeda Pharmaceutical Company Limited
On May 7, 2019, the company and Takeda entered into an exclusive license, or the Takeda License, pursuant to which Takeda granted the company an exclusive, sublicensable (with Takeda’s reasonable consent) license under certain patents and know-how relating to vonoprazan and owned or controlled by Takeda during the term of the Takeda License to commercialize vonoprazan products using specified formulations for all human therapeutic uses in the United States, Europe, and Canada, and a non-exclusive license under such patents and know-how to develop and manufacture such vonoprazan products anywhere in the world (subject to Takeda’s consent as to each country) for the purposes of commercializing the vonoprazan products in the United States, Europe, and Canada.
The company granted Takeda a non-exclusive, royalty-free, sublicensable license under the company’s rights in any patents and know-how that are necessary or useful to enable Takeda to develop and manufacture vonoprazan products anywhere in the world for the purposes of commercialization outside the United States, Europe, and Canada. The company also granted Takeda an exclusive, royalty-free license under its rights in certain patents and know-how owned or controlled by the company and necessary for the exploitation of vonoprazan products, in each case for Takeda to commercialize any vonoprazan product outside of the United States, Canada, and Europe, and for purposes other than human therapeutic use.
During the term of the Takeda License, the company and its affiliates are not permitted to commercialize any pharmaceutical product, other than vonoprazan, that treats acid-related disorders, except for certain generic and OTC competing products in specified circumstances. The company will be responsible, at its cost, for the development, manufacture, and commercialization of the vonoprazan products. The company is required to use commercially reasonable efforts to develop and commercialize the vonoprazan products in its licensed territory.
Under the Takeda License, Takeda has the sole right and authority, with the company’s input, to prepare, file, prosecute, and maintain all Takeda and joint patents on a worldwide basis at its own cost. The company is responsible, at its cost, for preparing, filing, prosecuting, and maintaining patents on inventions made solely by the company in connection with vonoprazan, subject to input from Takeda. The company has the first right to enforce the licensed patent rights with respect to certain infringing products in the United States, Europe, and Canada.
Sandoz Supply and Packaging Agreement
In December 2020, the company entered into a Supply and Packaging Services Agreement with Sandoz GmbH, or the Sandoz Supply Agreement, pursuant to which Sandoz has agreed to supply commercial quantities of amoxicillin capsules and clarithromycin tablets, to package these antibiotics with vonoprazan drug product in finished convenience packs, and to supply the company with these convenience packs.
Catalent Commercial Supply Agreement
In July 2021, the company entered into a Commercial Supply Agreement, or the Tablet Supply Agreement, with Catalent Pharma Solutions, LLC, or Catalent, pursuant to which Catalent has agreed to supply the company with commercial quantities of vonoprazan fumarate tablets.
Pursuant to the Tablet Supply Agreement, as amended, Catalent has agreed to supply the company with, and the company has agreed to purchase from Catalent, finished vonoprazan tablets at an agreed-upon price per unit. The price per unit may be adjusted annually based on increases in costs incurred by Catalent. The Tablet Supply Agreement requires the company to purchase a specified percentage of its requirements of finished vonoprazan tablets from Catalent, which percentage is subject to adjustment following January 1, 2027.
Evonik Commercial Supply Agreement
In August 2022, the company entered into a Commercial Supply Agreement, or the API Supply Agreement, with Evonik Operations GmbH, or Evonik, pursuant to which Evonik has agreed to supply the company with commercial quantities of vonoprazan drug substance, or API.
Pursuant to the API Supply Agreement, Evonik has agreed to supply the company with, and the company has agreed to purchase, certain quantities of API at an agreed-upon price which varies based on the volume of product ordered.
Unless terminated earlier, the API Supply Agreement has an initial period that expires in August 2027. This initial term was extended by two years from 2027 to 2029 upon Evonik's successful qualification of a second manufacturing facility to produce API in December of 2024.
Government Regulation
The company is enrolled or participates in the MDRP, the 340B program, the VA/FSS program, and the TRICARE retail pharmacy program, and has price reporting and payment obligations under these and other programs.
To obtain regulatory approval of a product candidate in the EU, the company must submit a marketing authorization (MA) Application, or MAA.
The company is also subject to the European Union General Data Protection Regulation, or the EU GDPR, and to the United Kingdom General Data Protection Regulation and Data Protection Act 2018, or collectively, the UK GDPR (the EU GDPR and UK GDPR together referred to as the ‘GDPR’), which imposes comprehensive data privacy compliance obligations in relation to the company’s collection, processing, sharing, disclosure, transfer, and other use of data relating to an identifiable living individual or ‘personal data’, including a principle of accountability and the obligation to demonstrate compliance through policies, procedures, training, and audit. Failure to comply with applicable data privacy and security laws can result in the imposition of significant civil and/or criminal penalties and private litigation.
Research and Development
The company’s research and development expenses were $34.1 million for the year ended December 31, 2024.
History
Phathom Pharmaceuticals, Inc. was incorporated under the laws of the state of Delaware in 2018.
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