Last update 09 Dec 2024

Lovastatin

Last update 09 Dec 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

(1S,3R,7S,8S,8aR)-1,2,3,7,8,8a-hexahydro-3,7-dimethyl-8-(2-(2R,4R)-(tetrahydro-4-hydroxy-6-oxo-2H-pyran-2-yl)ethyl)-1-naphthalenyl (S)-2-methyl-butyrate, 2β,6α-dimethyl-8alpha-(2-methyl-1-oxobutoxy)-mevinic acid lactone, 6-alpha-methylcompactin

+ [32]

Target

HMG-CoA reductase

Mechanism

HMG-CoA reductase inhibitors(HMG-CoA reductase inhibitors)

Therapeutic Areas

Cardiovascular DiseasesEndocrinology and Metabolic Disease

Active Indication

Coronary DiseaseHyperlipidemias

Inactive Indication

AtherosclerosisCoronary Artery DiseaseHypercholesterolemia

Originator Organization

Merck & Co., Inc.

Active Organization

Covis Pharma Holdings BV

Inactive Organization

Merck Research Laboratories Massachusetts LLC

Merck Sharp & Dohme Corp.

Drug Highest PhaseApproved

First Approval Date

US (31 Aug 1987),

Coronary DiseaseHypercholesterolemia

Regulation-

Structure

Molecular FormulaC24H36O5

InChIKeyPCZOHLXUXFIOCF-BXMDZJJMSA-N

CAS Registry75330-75-5

Clinical Trials associated with Lovastatin

NCT06636734 / Not yet recruitingPhase 2IIT

A Phase II Trial of Lovastatin and Pembrolizumab in Patients with RM HNSCC (LAPP)

This phase II trial tests how well lovastatin and pembrolizumab work in treating patients with head and neck cancer that has come back after a period of improvement (recurrent) or that has spread from where it first started (primary site) to other places in the body (metastatic). Lovastatin is a drug used to lower the amount of cholesterol in the blood and may also cause tumor cell death. In addition, studies have shown that lovastatin may make the tumor cells more sensitive to immunotherapy. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving lovastatin and pembrolizumab may kill more tumor cells in patients with recurrent or metastatic head and neck cancer.

Start Date31 Dec 2024

Sponsor / Collaborator

Emory University

[+1]

NCT06262685 / Not yet recruitingPhase 4IIT

A Multicenter, Controlled, Randomized, Single-blind Phase IV Trial Assesses Efficacy, Safety, and Cost of Pre-emptive Genotyping in a Cardiovascular Risk Population Eligible for High/Moderate-intensity Statins

This is a Phase IV multicentre adaptive single-blinded randomized clinical trial if preemptively genotyping populations at risk of cardiovascular disease susceptible of receiving high or moderate doses of statin therapy is efficacious, cost-efficacious, and feasible within the Spanish National Health System when compared to the current standard of care. This trial is nested within the iPHARMGx master protocol

Start Date04 Mar 2024

Sponsor / Collaborator

Hospital Universitario La Paz

[+1]

IRCT20140318017053N12 / CompletedPhase 3IIT

Comparison of the effects of lovastatin and alendronate with nanobone grafts on the preservation of tooth sockets

Start Date25 Feb 2021

Sponsor / Collaborator

Islamic Azad University

100 Clinical Results associated with Lovastatin

100 Translational Medicine associated with Lovastatin

100 Patents (Medical) associated with Lovastatin

4,433

Literatures (Medical) associated with Lovastatin

01 Feb 2025·FOOD CHEMISTRY

A bio-guided search of anti-steatotic compounds in Opuntia stricta var. dillenii by fast centrifugal partition chromatography

Article

Author: Lobo, M Gloria ; Serrano-Sandoval, Sayra N ; Parralejo-Sanz, Sara ; Serrano-Sandoval, Sayra N. ; Antunes-Ricardo, Marilena ; Cano, M Pilar ; Cano, M. Pilar ; Parralejo-SanZ, Sara ; Lobo, M. Gloria

The fruit extract of Opuntia stricta var. dillenii (OSDE) has been recognized for its effects on hepatic steatosis, but the compounds responsible for this activity have yet to be precisely identified. This work aimed to evaluate the anti-steatotic effect of OSDE and its different fractions obtained by fast centrifugal-partition chromatography (FCPC) to identify the compounds potentially responsible for this biological activity. Hepatic lipid accumulation and triglyceride content were evaluated, as well as cellular antioxidant activity and inhibition of lipid peroxidation. Pool 1-Pool 4 showed lower lipid accumulation than OSDE in liver cells, while a greater reduction in triglyceride levels, even lower than OSDE and lovastatin (LOV), was observed for Pool 1, 9, and 10. Compared to OSDE, Pools 1,6, 7, and 12 showed higher cellular antioxidant effects, whereas OSDE showed better lipid peroxidation inhibition than all of Pools. Quinic and piscidic acids were the main bioactive present in Pool 1, exhibiting +1597 % and + 997 % increases in their content related to OSDE, respectively. Likewise, the most abundant compounds in Pool 2- Pool 4 were betalains such as betanin and isobetanin, with +163 % and + 162 % of increases in their concentration related to OSDE, respectively. Antioxidant effects in Pools 6 and 7 correlated with higher phenolic acid concentration. OSDE significantly reduced triglyceride levels in a steatotic-induced model. Although OSDE showed anti-steatotic effects, they were more pronounced for some of its constituents in FCPC Pools. Results suggested that these compounds might be potentially responsible for this anti-steatotic effect. FCPC fractionation facilitated the separate biological evaluation of OSDE constituents and thus identified them. Future studies should focus on validating these anti-steatotic effects in in vivo models.

01 Jan 2025·INTERNATIONAL IMMUNOPHARMACOLOGY

Lovastatin-mediated pharmacological inhibition of Formin protein DIAPH1 suppresses tumor immune escape and boosts immunotherapy response

Article

Author: Zhu, Yichao ; Ding, Junli ; Zhou, Ji ; Mei, Jie ; Li, Qing ; He, Zhicheng ; Zhou, Jiaofeng ; Xue, Ningyi ; Wan, Mengyun ; Zong, Xinyu

BACKGROUND:

The immunosuppressive tumor microenvironment (TME) is a key characteristic of human cancer. Immunotherapy has emerged as a promising treatment strategy to overcome immune escape and has gained widespread use in recent years. In particular, the blockade of PD-1/PD-L1 interaction holds significant importance in oncotherapy. Combining anti-PD-1/PD-L1 with small molecule inhibitors targeting key pathways represents an emerging trend in therapeutic development.

METHODS:

To validate our findings biologically, we employed qRT-PCR or Western blotting and immunofluorescence staining techniques to assess the expression levels of DIAPH1 and PD-L1 in cells. Additionally, CCK8 and clone formation assays were utilized to evaluate cell proliferation ability, while flow assays were conducted to detect apoptosis in T cells.

RESULTS:

Knockdown of DIAPH1 restored the tumor-killing capacity of T cells, effectively suppressing tumor immune escape. We observed a highly positive correlation between the expression levels of DIAPH1 and 3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR), which can be competitively inhibited by lovastatin. Through Sybyl analysis followed by confirmation via micro scale thermophoresis, we identified lovastatin as a potential inhibitor targeting DIAPH1. Lovastatin downregulated DIAPH1 expression both in tumor cell lines and xenograft lung cancer tissues within a mouse lung cancer model. Furthermore, we found that lovastatin degraded DIAPH1 through lysosomal degradation pathway. Treatment with lovastatin was strongly associated with improved response rates and prolonged overall survival among patients with lung adenocarcinoma. Finally, overexpression of DIAPH1 reversed the inhibitory effects mediated by lovastatin on tumor development.

CONCLUSIONS:

Lovastatin downregulates PD-L1 expression by targeting DIAPH1 and restores the tumor-killing ability of T cells to block tumor immune escape. Lovastatin may become a potential drug for cancer patients to enhance immunotherapy response in the clinic.

02 Dec 2024·MOLECULAR PHARMACEUTICS

[⁶⁴Cu]Cu-NOTA-Trastuzumab and [⁸⁹Zr]Zr-DFO-Trastuzumab in Xenografts with Varied HER2 Expression

Article

Author: Simó, Cristina ; Vanover, Alex ; Shmuel, Shayla ; Pereira, Patrícia M. R.

Positron emission tomography (PET) has potential as a complementary technique to biomarker analysis, especially for human epidermal growth factor receptor 2 (HER2)-expressing tumors characterized by high heterogeneity. In this study, zirconium-89 (⁸⁹Zr) and copper-64 (⁶⁴Cu) labeled trastuzumab were employed to monitor varying levels of tumoral HER2 expression. Additionally, we studied the use of the cholesterol-depleting lovastatin as a pharmacological approach to enhance cell-surface HER2 expression in tumors with moderate to low HER2 levels, aiming to increase antibody accumulation in these tumor types. Both ⁸⁹Zr- and ⁶⁴Cu-labeled trastuzumab effectively monitor HER2 expression levels in xenografts exhibiting varying HER2 expression. No significant difference in tumor uptake was observed between ⁸⁹Zr- or ⁶⁴Cu-labeled trastuzumab, and tumor uptake for both radioimmunoconjugates positively correlated with HER2 protein levels. These findings underscore the potential of PET to monitor HER2 protein levels across heterogeneous tumors. Furthermore, our results suggest that further optimization of statin dosing and timing could offer a promising strategy to enhance trastuzumab accumulation in HER2-high, HER2-moderate, and HER2-low tumors.

News (Medical) associated with Lovastatin

15 Apr 2024

·www.biospace.com

Fresenius Accelerates Momentum in its (Bio)Pharma Business and Launches Tyenne®, its Third Approved Biosimilar in the U.S.

Tyenne® is the first tocilizumab biosimilar by Fresenius Kabi, an operating company of Fresenius SE, with an intravenous and subcutaneous formulation approved by the FDA. The tocilizumab biosimilar provides increased access and an affordable, high-quality, and safe treatment option for U.S. patients. Excellent performance of the (Bio)Pharma business in 2024 so far, also driven by Tyenne® which became the first tocilizumab biosimilar available in Europe in November 2023. The launch of the tocilizumab biosimilar contributes directly to growing Fresenius’ (Bio)Pharma platform, a substantial cornerstone of the #FutureFresenius strategy. BAD HOMBURG, Germany--(BUSINESS WIRE)-- Fresenius, via its operating company Fresenius Kabi, announced today the immediate U.S. availability of Tyenne® (tocilizumab-aazg), a biosimilar of Actemra® (tocilizumab). Tyenne®, for use in the treatment of chronic autoimmune diseases, is available in an intravenous (IV) formulation. This press release features multimedia. View the full release here: Fresenius Kabi announced that Tyenne® (tocilizumab-aazg), a biosimilar of Actemra® (tocilizumab) is now available in the U.S. in an IV presentation. Tyenne tocilizumab-aazg) is for use in the treatment of chronic autoimmune diseases. (Photo: Business Wire) Michael Sen, CEO of Fresenius: “With the launch of Tyenne® in the U.S., we have reached another important milestone in accelerating our strong (Bio)Pharma momentum. Growing this platform is a substantial cornerstone of our #FutureFresenius journey. Overall, we have seen an excellent performance of our (Bio)Pharma business in 2024 so far. We are particularly happy with the good progress of our majority-owned biotechnology company mAbxience and the traction of Tyenne®, the first tocilizumab biosimilar available in Europe since November 2023.” Tyenne® is the first tocilizumab biosimilar with an intravenous and subcutaneous formulation approved by the FDA. The biosimilar received FDA approval on March 5, 2024. Tyenne® is Fresenius’ third approved biosimilar available in the U.S. and the second within its immunology portfolio. The biologic medicine is indicated for the treatment of several autoimmune diseases, including rheumatoid arthritis, giant cell arteritis, polyarticular juvenile idiopathic arthritis, and systemic juvenile idiopathic arthritis. Pierluigi Antonelli, CEO of Fresenius Kabi: “Tyenne® will impact the treatment landscape for inflammatory and immune diseases in the U.S. Reaching ever more patients with our state-of-the-art biopharma portfolio signals a clear growth path in a highly promising market segment. We will continue to roll out our comprehensive pipeline of autoimmune and oncology biosimilars with several molecules in late-stage development.” Supported by Fresenius Kabi’s support program for health care professionals and patients, the company’s biologic medicine provides wider access to more treatment options and contributes to the viability of health care systems. Next to its two available biosimilars, Idacio® (adalimumab) and Stimufend® (pegfilgrastim), Fresenius Kabi has a growing pipeline of autoimmune and oncology biosimilars with several molecules in late-stage development. To learn more about how Fresenius Kabi provides comprehensive patient support for Tyenne® in the U.S. please click here. With #FutureFresenius, Fresenius successfully set the course last year to become a leading therapy-focused company. In line with the strategy, Fresenius has simplified its structure, is sharpening its focus by concentrating on its operating companies Fresenius Kabi and Fresenius Helios and is continuously enhancing its performance. About Tyenne®, a Tocilizumab Biosimilar Tyenne® (tocilizumab-aazg), a biosimilar to Actemra® (tocilizumab), is a prescription medicine called an Interleukin-6 (IL-6) receptor antagonist. It was developed by Fresenius Kabi using advanced analytical and manufacturing technologies for use in the treatment of several autoimmune diseases, including rheumatoid arthritis, giant cell arteritis, polyarticular juvenile idiopathic arthritis, and systemic juvenile idiopathic arthritis. Serious infections leading to hospitalization or death including tuberculosis (TB), bacterial, invasive fungal, viral, and other opportunistic infections have occurred in patients receiving the product. Tyenne is contraindicated in patients with known hypersensitivity to tocilizumab products. For more information about Tyenne, please see the full prescribing information for the U.S. here. IMPORTANT SAFETY INFORMATION RISK OF SERIOUS INFECTIONS Patients treated with TYENNE® (tocilizumab-aazg) are at increased risk for developing serious infections that may lead to hospitalization or death, including tuberculosis (TB), bacterial, invasive fungal, viral, or other opportunistic infections. If a serious infection develops, interrupt TYENNE until the infection is controlled. Reported infections include: Active tuberculosis, which may present with pulmonary or extrapulmonary disease. Patients should be tested for latent tuberculosis before TYENNE use and during therapy. Treatment for latent infection should be initiated prior to TYENNE use. Invasive fungal infections, including candidiasis, aspergillosis, and pneumocystis. Patients with invasive fungal infections may present with disseminated, rather than localized, disease. Bacterial, viral and other infections due to opportunistic pathogens. The risks and benefits of treatment with TYENNE should be carefully considered prior to initiating therapy in patients with chronic or recurrent infection. Patients should be closely monitored for the development of signs and symptoms of infection during and after treatment with TYENNE, including the possible development of tuberculosis in patients who tested negative for latent tuberculosis infection prior to initiating therapy. CONTRAINDICATION TYENNE is contraindicated in patients with known hypersensitivity to tocilizumab products. WARNINGS AND PRECAUTIONS Gastrointestinal Perforations Events of gastrointestinal (GI) perforation have been reported in clinical trials, primarily as complications of diverticulitis in patients treated with tocilizumab. Use TYENNE with caution in patients who may be at increased risk for GI perforation. Promptly evaluate patients presenting with new-onset abdominal symptoms for early identification of GI perforation. Hepatotoxicity Serious cases of hepatic injury have been observed in patients taking intravenous or subcutaneous tocilizumab products. Some of these cases have resulted in liver transplant or death. Time to onset for cases ranged from months to years after treatment initiation. Most cases presented with marked elevations of transaminases (> 5 times ULN), and some cases presented with signs or symptoms of liver dysfunction and only mildly elevated transaminases. Treatment with tocilizumab was associated with a higher incidence of transaminase elevations; increased frequency and magnitude of these elevations were observed when tocilizumab was used in combination with potentially hepatotoxic drugs (e.g., methotrexate). It is not recommended to initiate TYENNE treatment in RA, GCA, PJIA, and SJIA patients with elevated transaminases ALT or AST greater than 1.5x ULN. In patients who develop elevated ALT or AST greater than 5x ULN discontinue TYENNE. Measure liver tests promptly in patients who report symptoms that may indicate liver injury. If the patient is found to have abnormal liver tests, TYENNE treatment should be interrupted. TYENNE should only be restarted in patients with another explanation for the liver test abnormalities after normalization of the liver tests. Laboratory Parameters Laboratory monitoring is recommended due to potential consequences of treatment-related laboratory abnormalities in neutrophils, platelets, lipids, and liver function tests. Dosage modifications may be required. Neutropenia: Treatment with tocilizumab products was associated with a higher incidence of neutropenia. It is not recommended to initiate TYENNE treatment in RA, GCA, PJIA, and SJIA patients with a low neutrophil count i.e., absolute neutrophil count (ANC) less than 2000 per mm3. In patients who develop an ANC less than 500 per mm3 treatment is not recommended. Thrombocytopenia: Treatment with tocilizumab products was associated with a reduction in platelet counts. It is not recommended to initiate TYENNE in RA, GCA, PJIA, and SJIA patients with a platelet count below 100,000 per mm3. In patients who develop a platelet count less than 50,000 per mm3, treatment is not recommended. Elevated Liver Enzymes: It is not recommended to initiate TYENNE treatment in patients with elevated transaminases ALT or AST >1.5x ULN. In patients who develop elevated ALT or AST >5x ULN, treatment is not recommended. Lipid Abnormalities: Treatment with tocilizumab products was associated with increases in lipid parameters such as total cholesterol, triglycerides, LDL cholesterols, and/or HDL cholesterol. Immunosuppression The impact of treatment with tocilizumab products on the development of malignancies is not known, but malignancies were observed in clinical studies with tocilizumab. TYENNE is an immunosuppressant, and treatment with immunosuppressants may result in an increased risk of malignancies. Hypersensitivity Reactions Hypersensitivity reactions, including anaphylaxis, have been reported in association with tocilizumab products and anaphylactic events with a fatal outcome have been reported with intravenous infusion of tocilizumab products. TYENNE for intravenous use should only be infused by a healthcare professional with appropriate medical support to manage anaphylaxis. For TYENNE subcutaneous injection, advise patients to seek immediate medical attention if they experience any symptoms of a hypersensitivity reaction. If anaphylaxis or other hypersensitivity reaction occurs, stop administration of TYENNE immediately and discontinue TYENNE permanently. Do not administer TYENNE to patients with known hypersensitivity to tocilizumab products. Demyelinating Disorders The impact of treatment with tocilizumab products on demyelinating disorders is not known, but multiple sclerosis and chronic inflammatory demyelinating polyneuropathy were reported rarely in clinical studies. Monitor patients for signs and symptoms of demyelinating disorders. Prescribers should exercise caution in considering the use of TYENNE in patients with preexisting or recent onset demyelinating disorders. Active Hepatic Disease and Hepatic Impairment Treatment with TYENNE is not recommended in patients with active hepatic disease or hepatic impairment. Vaccinations Avoid use of live vaccines concurrently with TYENNE. No data are available on the secondary transmission of infection from persons receiving live vaccines to patients receiving TYENNE or on the effectiveness of vaccination in patients receiving TYENNE. Patients should be brought up to date on all recommended vaccinations prior to initiation of TYENNE therapy, if possible. ADVERSE REACTIONS Most common adverse reactions (incidence of at least 5%): upper respiratory tract infections, nasopharyngitis, headache, hypertension, increased ALT, injection site reactions. DRUG INTERACTIONS In GCA patients, no effect of concomitant corticosteroid on tocilizumab exposure was observed. Cytochrome P450s in the liver are down-regulated by infection and inflammation stimuli including cytokines such as IL-6. Inhibition of IL-6 signaling in RA patients treated with tocilizumab products may restore CYP450 activities to higher levels than those in the absence of tocilizumab products leading to increased metabolism of drugs that are CYP450 substrates. Exercise caution when coadministering TYENNE with CYP3A4 substrate drugs where decrease in effectiveness is undesirable, e.g., oral contraceptives, lovastatin, atorvastatin, etc. USE IN PREGNANCY The limited available data with tocilizumab products in pregnant women are not sufficient to determine whether there is a drug-associated risk for major birth defects and miscarriage. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to Fresenius Kabi at (800) 551-7176. Please see additional Important Safety Information in full Prescribing Information, including Boxed Warning. INDICATIONS TYENNE is indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response to one or more Disease-Modifying Anti-Rheumatic Drugs (DMARDs). TYENNE is indicated for the treatment of giant cell arteritis (GCA) in adult patients. TYENNE is indicated for the treatment of active polyarticular juvenile idiopathic arthritis (PJIA) in patients 2 years of age and older. TYENNE is indicated for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in patients 2 years of age and older. Tyenne® demonstrates Fresenius Kabi’s commitment to providing access to affordable and cost-effective biosimilars to more patients living with autoimmune diseases around the world while enabling savings for health care systems. KabiCare, Fresenius Kabi’s comprehensive patient support program, will be available to patients and health care providers in the U.S. from launch. About Fresenius SE Fresenius SE & Co. KGaA (Frankfurt/Xetra: FRE) is a global healthcare company headquartered in Bad Homburg v. d. Höhe, Germany. In the 2023 fiscal year, Fresenius generated €22.3 billion in annual revenue with its more than 190,000 employees. Fresenius offers solutions to the social challenges posed by a growing and ageing population and the resulting need for affordable, high-quality healthcare. The Fresenius Group comprises the operating companies Fresenius Kabi and Fresenius Helios as well as the investment companies Fresenius Vamed and Fresenius Medical Care. With 140 hospitals and countless outpatient facilities, Fresenius Helios is the leading private hospital operator in Germany and Spain, treating around 26 million patients every year. Fresenius Kabi’s product portfolio includes a range of highly complex biopharmaceuticals, clinical nutrition, medical technology, and generic intravenous drugs. Fresenius was established in 1912 by the Frankfurt pharmacist Dr. Eduard Fresenius. After his death, Else Kröner took over management of the company in 1952. She laid the foundations for a global enterprise that today pursues the goal of improving people’s health. The largest shareholder is the non-profit Else Kröner-Fresenius Foundation, which is dedicated to advancing medical research and supporting humanitarian projects. For more information visit the company website at . Follow us on social media: . About Fresenius Kabi Fresenius Kabi is a global healthcare company that specializes in lifesaving medicines and technologies for infusion, transfusion and clinical nutrition. The company’s products and services are used for the therapy and care of critically and chronically ill patients. Its product portfolio comprises a range of highly complex biopharmaceuticals, clinical nutrition, medical technologies, and I.V. generic drugs. Within biopharmaceuticals, Fresenius Kabi offers, among others, biosimilar drugs with a focus on autoimmune diseases and oncology. The company’s clinical nutrition offering includes a wide selection of enteral and parenteral nutrition products. In the segment of medical technologies, its offering includes vital disposables, infusions pumps, apheresis machines, cell therapy devices, and more. Fresenius Kabi puts essential medicines and technologies in the hands of people who help patients and finds the best answers to the challenges they face. Following its strategy “Vision 2026”, which is a key part of the #FutureFresenius program of the Fresenius healthcare group, the company is furthermore committed to increase efficiencies in the therapy and care of patients and improve access to high-quality healthcare around the globe. Fresenius Kabi aspires to be leading globally in its product segments – all for the benefit of patients, its customers, and its stakeholders. Actemra® is a registered trademark of Chugai Seiyaku Kabushiki Kaisha Corp., a member of the Roche Group. Tyenne® is a registered trademark of Fresenius Kabi Deutschland GmbH. Idacio® and Stimufend® are registered trademarks of Fresenius Kabi Deutschland GmbH in selected countries. View source version on businesswire.com: Contacts Matt Kuhn (847) 220-3033 matt.kuhn@fresenius-kabi.com Source: Fresenius Kabi Smart Multimedia Gallery Photo Fresenius Kabi announced that Tyenne® (tocilizumab-aazg), a biosimilar of Actemra® (tocilizumab) is now available in the U.S. in an IV presentation. Tyenne tocilizumab-aazg) is for use in the treatment of chronic autoimmune diseases. (Photo: Business Wire) Logo View this news release and multimedia online at:

Drug Approval

28 Nov 2023

·www.globenewswire.com

Phathom Pharmaceuticals Announces Commercial Availability of VOQUEZNA® (vonoprazan) Tablets, a Powerful First-In-Class PCAB for the Treatment of Erosive GERD and Relief of Associated Heartburn

VOQUEZNA, the first and only FDA-approved potassium-competitive acid blocker (PCAB), is now available through major retail pharmacies and BlinkRx, an end-to-end digital fulfillment channelVOQUEZNA tablets in 30-count bottles are now commercially available for the healing and maintenance of healing of all severities of Erosive GERD, and relief of heartburn associated with Erosive GERD1 FLORHAM PARK, N.J., Nov. 28, 2023 (GLOBE NEWSWIRE) -- Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal (GI) diseases, today announced the U.S. commercial availability of VOQUEZNA® (vonoprazan). VOQUEZNA is now available for the treatment of adults with Erosive Esophagitis, also known as Erosive GERD (gastroesophageal reflux disease), and the relief of heartburn associated with Erosive GERD.1 As the first and only approved potassium-competitive acid blocker (PCAB) in the U.S., this milestone brings the power of a new class of acid suppression treatment to a disease with high unmet need. “We are thrilled to announce the commercial availability of our first-in-class medication, VOQUEZNA, now available for the millions of people in the U.S. suffering from Erosive GERD,” said Martin Gilligan, Chief Commercial Officer at Phathom Pharmaceuticals. “For over three decades, there has been no major innovation in this category. We are excited to introduce VOQUEZNA to patients and healthcare providers, as it has been shown to provide rapid, potent, and durable acid suppression and has the power to help heal Erosive GERD for patients seeking a new and effective treatment option.” The U.S. Food and Drug Administration (FDA) recently approved VOQUEZNA for the healing of all severities (grades) of Erosive GERD, maintenance of healing of all severities of Erosive GERD, and relief of heartburn associated with Erosive GERD in adults.1 Its novel mechanism of action (MOA) provides rapid, potent, and durable acid suppression in a way that is distinct from other prescription and over-the-counter medications.2 Additionally, VOQUEZNA does not have the burden of mealtime dosing, whereas most PPIs must always be taken with food.1 In a Phase 3, randomized clinical study, VOQUEZNA 20 mg met the primary endpoint of non-inferiority for complete healing by Week 8 in patients with all severities of Erosive GERD, demonstrating a strong 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 at Week 2 compared to lansoprazole (70% for VOQUEZNA 20 mg and 53% for lansoprazole 30 mg). In the maintenance phase of the study, 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). The most common side effects of VOQUEZNA for the treatment of Erosive GERD include stomach inflammation, diarrhea, stomach bloating, stomach pain, nausea, indigestion, high blood pressure, and urinary tract infection. “Erosive GERD is a highly prevalent condition affecting over 20 million people in the U.S.,3,4 many of whom experience troubling symptoms, including painful heartburn. When not properly treated, Erosive GERD can lead to complications such as scarring, narrowing of the esophagus, and bleeding,4” said Colin Howden, M.D., Professor Emeritus, University of Tennessee College of Medicine. "With many patients unsatisfied with their current therapy,5 the introduction of VOQUEZNA provides healthcare providers and patients with an important new treatment option that offers a novel mechanism of action and has demonstrated superiority in comparison to a standard-of-care PPI across several clinically meaningful endpoints.2" Prescriptions for VOQUEZNA may be filled at major retail pharmacies and through BlinkRx, an end-to-end digital fulfillment channel. Phathom is offering programs for eligible patients who face coverage or affordability issues, including co-pay assistance for patients with commercial insurance. For more information, please visit www.voquezna.com/savings. In addition, VOQUEZNA® TRIPLE PAK® (vonoprazan tablets, amoxicillin capsules, clarithromycin tablets) and VOQUEZNA® DUAL PAK® (vonoprazan tablets, amoxicillin capsules), two treatment regimens for adults with H. pylori infection, are expected to be commercially available in mid-December 2023. VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK each contain 14-days of VOQUEZNA-based treatment co-packaged with antibiotics in convenient blister packs. VOQUEZNA is marketed exclusively by Phathom Pharmaceuticals, Inc. Please visit www.voquezna.com to learn more about VOQUEZNA. INDICATION AND IMPORTANT SAFETY INFORMATION What is VOQUEZNA?VOQUEZNA® (vonoprazan) is a prescription medicine used in adults: for 8 weeks to heal acid-related damage to the lining of the esophagus (called Erosive Esophagitis or Erosive Acid Reflux) and for relief of heartburn related to Erosive Acid Reflux.for up to 6 months to maintain healing of Erosive Acid Reflux and for relief of heartburn related to Erosive Acid Reflux. It is not known if VOQUEZNA is safe and effective in children. Do not take VOQUEZNA if you: are allergic to vonoprazan or any of the other ingredients in VOQUEZNA. Allergic reaction symptoms may include trouble breathing, rash, itching and swelling of your face, lips, tongue, or throat.are taking a medicine that contains rilpivirine (EDURANT, COMPLERA, JULUCA, ODEFSEY, CABENUVA) used to treat HIV-1 (Human Immunodeficiency Virus). Before taking VOQUEZNA, tell your healthcare provider about all your medical conditions, including if you: have low magnesium, calcium, or potassium in your blood, or you are taking a medicine to increase urine (diuretic).have kidney or liver problems.are pregnant, think you may be pregnant, or plan to become pregnant. It is not known if VOQUEZNA will harm your unborn baby.are breastfeeding or plan to breastfeed. It is not known if VOQUEZNA passes into your breast milk. You and your healthcare provider should decide if you will take VOQUEZNA or breastfeed. You should not do both. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine. VOQUEZNA may affect how other medicines work, and other medicines may affect how VOQUEZNA works. Especially tell your healthcare provider if you take medicine that contains rilpivirine (EDURANT, COMPLERA, JULUCA, ODEFSEY, CABENUVA). What are the possible side effects of VOQUEZNA? VOQUEZNA may cause serious side effects including: A type of kidney problem (acute tubulointerstitial nephritis): Some people who take VOQUEZNA may develop a kidney problem called acute tubulointerstitial nephritis. Call your healthcare provider right away if you have a decrease in the amount that you urinate or if you notice blood in your urine. Diarrhea caused by an infection (Clostridioides difficile) in your intestines: Call your healthcare provider right away if you have watery stools, stomach pain, and fever that does not go away. Bone fractures (hip, wrist, or spine): Bone fractures in the hip, wrist, or spine may happen in people who take multiple daily doses of another type of medicine that reduces acid in your stomach known as proton pump inhibitors (PPI medicines) for a long period of time (a year or longer). Tell your healthcare provider if you have a bone fracture, especially in the hip, wrist, or spine. Severe skin reactions: VOQUEZNA can cause rare, but severe skin reactions that may affect any part of your body. These serious skin reactions may need to be treated in a hospital and may be life threatening: Skin rash which may have blistering, peeling, or bleeding on any part of your skin.You may also have fever, chills, body aches, shortness of breath, or enlarged lymph nodes.If you experience any of these symptoms, stop taking VOQUEZNA and call your healthcare provider right away. These symptoms may be the first sign of a severe skin reaction. Low vitamin B-12 levels: VOQUEZNA lowers the amount of acid in your stomach. Stomach acid is needed to absorb Vitamin B12 properly. Tell your healthcare provider if you have symptoms of low vitamin B12 levels, including irregular heartbeat, shortness of breath, lightheadedness, tingling or numbness in the arms or legs, muscle weakness, pale skin, feeling tired, or mood changes. Talk with your healthcare provider about the risk of low vitamin B12 levels if you have been on VOQUEZNA for a long time. Low magnesium levels in the body can happen in people who take VOQUEZNA. Tell your healthcare provider right away if you have symptoms of low magnesium levels, including seizures, dizziness, irregular heartbeat, jitteriness, muscle aches or weakness, or spasms of hands, feet, or voice. Stomach growths (fundic gland polyps): A certain type of stomach growth called fundic gland polyps may happen in people who take another type of medicine that reduces acid in your stomach known as proton pump inhibitors (PPI medicines) for a long time. Talk with your healthcare provider about the possibility of fundic gland polyps if you have been on VOQUEZNA for a long time. The most common side effects of VOQUEZNA for treatment of Erosive Acid Reflux include: stomach inflammationdiarrheastomach bloatingstomach painnauseaindigestionhigh blood pressureurinary tract infection These are not all the possible side effects of VOQUEZNA. For more information, ask your healthcare provider or pharmacist. Call your healthcare provider for medical advice about side effects. You are encouraged to report suspected adverse reactions by contacting Phathom Pharmaceuticals at 1-888- 775-PHAT (7428) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Please see Patient Information and full Prescribing Information for VOQUEZNA. What are VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK? VOQUEZNA® TRIPLE PAK® (vonoprazan tablets, amoxicillin capsules, clarithromycin tablets) and VOQUEZNA® DUAL PAK® (vonoprazan tablets, amoxicillin capsules) are co-packaged prescription medicines for the treatment of a Helicobacter pylori (H. pylori) bacterial infection in adults.It is not known if VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK are safe and effective in children.To reduce the development of drug-resistant bacteria and maintain the effectiveness of VOQUEZNA TRIPLE PAK, VOQUEZNA DUAL PAK and other antibacterial drugs, these products should be used only to treat or prevent infections that are proven or strongly suspected to be caused by susceptible bacteria. Do not take VOQUEZNA TRIPLE PAK if you are known to have an allergy or be sensitive to the components of VOQUEZNA TRIPLE PAK (vonoprazan, amoxicillin, clarithromycin), macrolide antibiotics (such as azithromycin and erythromycin), or penicillin.are taking: medicines that contain rilpivirine (Edurant, Complera, Odefsey)pimozidecolchicine, if you have kidney or liver problemslomitapide, lovastatin, and simvastatinergot alkaloids (ergotamine or dihydroergotamine)lurasidone have a history of yellowing of the skin (jaundice) or liver problems when taking clarithromycin. Do not take VOQUEZNA DUAL PAK if you are known to have an allergy or be sensitive to the components of VOQUEZNA DUAL PAK (vonoprazan, amoxicillin) or penicillin.are taking medicines that contain rilpivirine (Edurant, Complera, Odefsey). Before you take VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK, tell your healthcare provider about all of your medical conditions, including if you are breastfeeding. If breastfeeding, pump and discard breast milk during treatment and for 2 days after treatment.have severe kidney disease.have moderate to severe liver disease.have myasthenia gravis. Additionally, do not take VOQUEZNA TRIPLE PAK if: you are pregnant or plan to become pregnant. Clarithromycin, a medicine in VOQUEZNA TRIPLE PAK may harm your unborn baby. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. There can be serious side effects when some are used in combination with this product. Serious adverse reactions can occur with VOQUEZNA TRIPLE PAK due to drug interactions of clarithromycin with colchicine, some lipid lowering agents, some calcium channel blockers, and other drugs. What are the possible side effects of VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK? VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK can cause serious side effects including: Allergic reactions (hypersensitivity): Call your doctor right away if you have rash, hives, or other skin changes, face swelling or difficulty breathing.Severe skin reactions: VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK can cause severe skin reactions, such as skin rash or allergic reaction on or in any part of your body. Symptoms can also include, but are not limited to, fever, chills, body aches or shortness of breath. If you experience any of these symptoms, stop taking VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK immediately and call your doctor right away.Severe diarrhea: Call your doctor right away if you have watery stool, stomach pain, and fever that does not go away while taking VOQUEZNA DUAL PAK or VOQUEZNA TRIPLE PAK or after therapy is completed.Rash in patients with mononucleosis: Amoxicillin (a component of VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK) may cause a rash in patients who have mononucleosis. Stop taking VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK if you are diagnosed with mononucleosis and call your doctor right away.Altered test results for some tumors: VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK lower stomach acid which can cause increased levels of a certain protein (CgA) in your blood. When this level is increased it may alter test results for detecting some tumors. Notify your doctor of the use of VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK prior to blood tests. Additionally, VOQUEZNA TRIPLE PAK can cause: Irregular heartbeats: Clarithromycin may cause irregular heartbeats. Call your doctor right away if you feel faint, light-headed, or feel your heart beating irregularly. Liver problems: Call your doctor right away if you have any of the following symptoms: weight loss, yellowing of the skin and eyes (jaundice), dark urine, rash, or pain on the right side of your abdomen. The most common side effects may include: diarrheatemporary changes in sense of tastevaginal yeast infectionstomach painheadachehigh blood pressure andcold-like symptoms These are not all of the possible side effects of VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK. Call your healthcare provider for medical advice about side effects. General information about the safe and effective use of VOQUEZNA.Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK for a condition for which it was not prescribed. Do not give VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK to other people, even if they have the same symptoms you have. It may harm them. For more information, ask your healthcare provider or pharmacist. You are encouraged to report suspected adverse reactions by contacting Phathom Pharmaceuticals at 1-888-775-PHAT (7428) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Please see full Prescribing Information for VOQUEZNA TRIPLE PAK AND VOQUEZNA DUAL PAK. About Erosive GERDErosive GERD, also referred to as Erosive Esophagitis or Erosive Acid Reflux, is a major type of GERD that affects over 20 million people in the U.S. and is characterized by erosions in the gastric mucosa caused by acidic reflux of stomach contents into the esophagus.3 There are estimated to be over 65 million individuals with GERD in the U.S., of which approximately 30% have erosive GERD.4,6,7 In addition to experiencing troubling heartburn symptoms and painful erosions, patients with inadequately treated erosive GERD may progress to more severe diseases including Barrett’s esophagus and esophageal cancer.4 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. Vonoprazan provides rapid, potent, and durable acid suppression. Phathom in-licensed the U.S., European, and Canadian 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 in the U.S., Europe, and Canada to vonoprazan, a first-in-class potassium-competitive acid blocker (PCAB). For more information about Phathom, visit the Company’s website at www.phathompharma.com and follow the Company on LinkedIn and X (formerly Twitter). Forward Looking StatementThis press release contains forward-looking statements. Investors are cautioned not to place undue reliance on these forward-looking statements, including statements about the size of the Erosive GERD patient population and the potential of vonoprazan to satisfy the large unmet medical need. The inclusion of forward-looking statements should not be regarded as a representation by Phathom that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Phathom’s business, including, without limitation: we may not be able to successfully commercialize VOQUEZNA which will depend on a number of factors including coverage and reimbursement levels from governmental authorities and health insurers as well as market acceptance by healthcare providers; future data generated from our stability program may be different from the data submitted to the FDA to date and may not demonstrate that our mitigation efforts will continue to maintain the level of the nitrosamine impurity below the acceptable intake (AI) level throughout the shelf life of products containing vonoprazan, which could result in market action or shelf life reduction; risks associated with product manufacturing or formulation changes required to be made in connection with achieving the AI; the inherent risks of clinical development of vonoprazan; Phathom’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; regulatory developments in the United States and foreign countries; unexpected adverse side effects or inadequate efficacy of vonoprazan that may limit its development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; Phathom’s ability to access additional capital under its term loan facility and royalty interest finance agreements is subject to certain conditions; Phathom’s ability to obtain and maintain intellectual property protection for vonoprazan; Phathom’s ability to comply with its license agreement with Takeda; and other risks described in the Company’s prior press releases and the Company’s filings with the Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Phathom undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. MEDIA CONTACTNick Benedetto1-877-742-8466media@phathompharma.com INVESTOR CONTACTEric Sciorilli1-877-742-8466ir@phathompharma.com © 2023 Phathom Pharmaceuticals. All rights reserved. VOQUEZNA, Phathom Pharmaceuticals, and their respective logos are registered trademarks or trademarks of Phathom Pharmaceuticals, Inc. 11/23 US-VPZ-23-0227 1 VOQUEZNA Prescribing Information, Phathom Pharmaceuticals, 2023.2 Laine L, DeVault K, Katz P, et al. Vonoprazan Verses Lansoprazole for Healing and Maintenance of Healing of Erosive Esophagitis: A Randomized Trial. Gastroenterology. October 10, 2022. https://doi.org/10.1053/j.gastro.2022.09.0413 Dickman R, Maradey-Romero C, Gingold-Belfer R, Fass R. Unmet Needs in the Treatment of Gastroesophageal Reflux Disease. J Neurogastroenterol Motil. 2015 Jul 30;21(3):309-19. doi: 10.5056/jnm15105.4 Savarino E, de Bortoli N, De Cassan C, et al. The natural history of gastro-esophageal reflux disease: A comprehensive review. Dis Esophagus. 2017;30(2):1-9.5 Vaezi MF, Brunton S, Mark Fendrick A, et al. Patient journey in erosive esophagitis: real-world perspectives from US physicians andpatients. BMJ Open Gastroenterology 2022;9:e000941. doi: 10.1136/bmjgast-2022-0009416 Machicado J.D., Greer J.B., Yadav D. (2020) Epidemiology of Gastrointestinal Diseases. In: Pitchumoni C., Dharmarajan T. (eds) Geriatric Gastroenterology. Springer, Cham. https://doi.org/10.1007/978-3-319-90761-1_7-1.7 U.S. Census Bureau. U.S. and World Population Clock. Accessed May 2022. https://www.census.gov/popclock.

Clinical ResultDrug ApprovalPhase 3

30 Oct 2023

·www.globenewswire.com

Phathom Pharmaceuticals Announces FDA Approval of Reformulated Vonoprazan Tablets for VOQUEZNA® TRIPLE PAK® (vonoprazan, amoxicillin, clarithromycin) and VOQUEZNA® DUAL PAK® (vonoprazan, amoxicillin) for the Treatment of H. pylori Infection in Adults

Planning for a December 2023 U.S. launch for H. pylori, together with the U.S. launch of vonoprazan for Erosive GERD, if approved FLORHAM PARK, N.J., Oct. 30, 2023 (GLOBE NEWSWIRE) -- Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal (GI) diseases, announced that the U.S. Food and Drug Administration (FDA) has approved the Prior Approval Supplement (PAS) for the reformulation of vonoprazan tablets for both VOQUEZNA TRIPLE PAK (vonoprazan tablets, amoxicillin capsules, clarithromycin tablets) and VOQUEZNA DUAL PAK (vonoprazan tablets, amoxicillin capsules), for the treatment of Helicobacter pylori (H. pylori) infection in adults. VOQUEZNA treatment regimens contain antibiotics conveniently packaged with vonoprazan, a novel potassium-competitive acid blocker (PCAB) and the first innovative acid suppressant from a new drug class approved in the U.S. in over 30 years. “We are very pleased with the FDA approval of our reformulated vonoprazan tablets for both VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK, enabling Phathom to market two new first-line treatment options that offer strong H. pylori eradication rates,” said Terrie Curran, President and Chief Executive Officer at Phathom. “H. pylori eradication failures are due to rising antibiotic resistance, inadequate acid suppression, and complex treatment regimens. We are excited about VOQUEZNA TRIPLE and DUAL PAKs, a new class of acid suppression therapy, that has the potential to address these issues and enhance H. pylori eradication. Our teams are making the final preparations for a combined December 2023 U.S. commercial launch for H. pylori along with the anticipated launch of vonoprazan for Erosive GERD, if approved. Thank you to Phathom employees and our manufacturing partners, Catalent and Evonik, who contributed to today’s PAS approval.” These initial product approvals were based on safety and efficacy data from the Phase 3 PHALCON-HP trial, the largest U.S. registrational trial ever conducted in H. pylori, randomizing 1,046 patients.1 In the modified intent-to-treat population, both VOQUEZNA treatment regimens demonstrated non-inferiority to lansoprazole triple therapy in patients without a clarithromycin or amoxicillin resistant strain of H. pylori at baseline. The H. pylori eradication rate was 84.7% with VOQUEZNA TRIPLE PAK compared to 78.8% with lansoprazole triple therapy [95% CI: -0.8, 12.6] and 78.5% for VOQUEZNA DUAL PAK compared to 78.8% with lansoprazole triple therapy [95% CI: -7.4, 6.8]. VOQUEZNA TRIPLE PAK and DUAL PAK demonstrated superior eradication rates compared to PPI-based triple therapy (lansoprazole with amoxicillin and clarithromycin) among all patients, including in patients with clarithromycin resistant strains of H. pylori.1 The H. pylori eradication rate with VOQUEZNA TRIPLE PAK was 80.8% versus 68.5% with lansoprazole triple therapy in the overall study population [95% CI: 5.7, 18.8] and in patients who had a clarithromycin-resistant strain of H. pylori, 65.8% vs. 31.9%, respectively [95% CI: 17.7, 48.1].1 H. pylori eradication rates for VOQUEZNA DUAL PAK were 77.2% versus 68.5% with lansoprazole triple therapy in the overall study population [95% CI: 1.9, 15.4] and in patients who had a clarithromycin-resistant strain of H. pylori, 69.6% vs. 31.9%, respectively [95% CI: 20.5, 52.6].1 Adverse event (AE) rates for the vonoprazan-based regimens were comparable to lansoprazole triple therapy in the trial.1 The most common AEs (>2.0%) reported in the VOQUEZNA TRIPLE PAK, VOQUEZNA DUAL PAK, and lansoprazole triple therapy arms, respectively, were diarrhea (4.0%, 5.2%, 9.6%), dysgeusia (4.6%, 0.6%, 6.1%), vulvovaginal candidiasis (3.2%, 2.0%, 1.4%), abdominal pain (2.3%, 2.6%, 2.9%), headache (2.6%, 1.4%, 1.4%), hypertension (2.0%, 1.1%, 0.9%) and nasopharyngitis (0.3%, 2.0%, 0.9%).1 VOQUEZNA TRIPLE and DUAL PAKs are expected to be available in the U.S. in December 2023 and marketed exclusively by Phathom Pharmaceuticals, Inc. Phathom is planning for a combined U.S. commercial launch of VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK, together with vonoprazan for Erosive GERD, if approved. Phathom plans to host an investor conference call in November 2023, following FDA action on the pending Erosive GERD New Drug Application, which has a PDUFA target action date of November 17, to discuss the Company’s U.S. commercial launch plans. The full Prescribing Information for VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK can be found here. INDICATION AND IMPORTANT SAFETY INFORMATION What are VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK? VOQUEZNA® TRIPLE PAK® (vonoprazan tablets, amoxicillin capsules, clarithromycin tablets) and VOQUEZNA® DUAL PAK® (vonoprazan tablets, amoxicillin capsules) are co-packaged prescription medicines for the treatment of a Helicobacter pylori (H. pylori) bacterial infection in adults.It is not known if VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK are safe and effective in children.To reduce the development of drug-resistant bacteria and maintain the effectiveness of VOQUEZNA TRIPLE PAK, VOQUEZNA DUAL PAK and other antibacterial drugs, these products should be used only to treat or prevent infections that are proven or strongly suspected to be caused by susceptible bacteria. Do not take VOQUEZNA TRIPLE PAK if you are known to have an allergy or be sensitive to the components of VOQUEZNA TRIPLE PAK (vonoprazan, amoxicillin, clarithromycin), macrolide antibiotics (such as azithromycin and erythromycin), or penicillin.are taking: medicines that contain rilpivirine (Edurant, Complera, Odefsey)pimozidecolchicine, if you have kidney or liver problemslomitapide, lovastatin, and simvastatinergot alkaloids (ergotamine or dihydroergotamine)lurasidone have a history of yellowing of the skin (jaundice) or liver problems when taking clarithromycin. Do not take VOQUEZNA DUAL PAK if you are known to have an allergy or be sensitive to the components of VOQUEZNA DUAL PAK (vonoprazan, amoxicillin) or penicillin.are taking medicines that contain rilpivirine (Edurant, Complera, Odefsey). Before you take VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK, tell your healthcare provider about all of your medical conditions, including if you are breastfeeding. If breastfeeding, pump and discard breast milk during treatment and for 2 days after treatment.have severe kidney disease.have moderate to severe liver disease.have myasthenia gravis. Additionally, do not take VOQUEZNA TRIPLE PAK if: you are pregnant or plan to become pregnant. Clarithromycin, a medicine in VOQUEZNA TRIPLE PAK may harm your unborn baby. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. There can be serious side effects when some are used in combination with this product. Serious adverse reactions can occur with VOQUEZNA TRIPLE PAK due to drug interactions of clarithromycin with colchicine, some lipid lowering agents, some calcium channel blockers, and other drugs. What are the possible side effects of VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK? VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK can cause serious side effects including: Allergic reactions (hypersensitivity): Call your doctor right away if you have rash, hives, or other skin changes, face swelling or difficulty breathing.A type of kidney problem (acute tubulointerstitial nephritis): Some people who take VOQUEZNA may develop a kidney problem called acute tubulointerstitial nephritis. Call your healthcare provider right away if you have a decrease in the amount that you urinate or if you notice blood in your urine.Severe skin reactions: VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK can cause severe skin reactions, such as skin rash or allergic reaction on or in any part of your body. Symptoms can also include, but are not limited to, fever, chills, body aches or shortness of breath. If you experience any of these symptoms, stop taking VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK immediately and call your doctor right away.Severe diarrhea: Call your doctor right away if you have watery stool, stomach pain, and fever that does not go away while taking VOQUEZNA DUAL PAK or VOQUEZNA TRIPLE PAK or after therapy is completed.Rash in patients with mononucleosis: Amoxicillin (a component of VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK) may cause a rash in patients who have mononucleosis. Stop taking VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK if you are diagnosed with mononucleosis and call your doctor right away.Altered test results for some tumors: VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK lower stomach acid which can cause increased levels of a certain protein (CgA) in your blood. When this level is increased it may alter test results for detecting some tumors. Notify your doctor of the use of VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK prior to blood tests. Additionally, VOQUEZNA TRIPLE PAK can cause: Irregular heartbeats: Clarithromycin may cause irregular heartbeats. Call your doctor right away if you feel faint, light-headed, or feel your heart beating irregularly.Liver problems: Call your doctor right away if you have any of the following symptoms: weight loss, yellowing of the skin and eyes (jaundice), dark urine, rash, or pain on the right side of your abdomen. The most common side effects may include: diarrheatemporary changes in sense of tastevaginal yeast infectionstomach painheadachehigh blood pressure andcold-like symptoms These are not all of the possible side effects of VOQUEZNA TRIPLE PAK and VOQUEZNA DUAL PAK. Call your healthcare provider for medical advice about side effects. General information about the safe and effective use of VOQUEZNA.Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK for a condition for which it was not prescribed. Do not give VOQUEZNA TRIPLE PAK or VOQUEZNA DUAL PAK to other people, even if they have the same symptoms you have. It may harm them. For more information, ask your healthcare provider or pharmacist. You are encouraged to report suspected adverse reactions by contacting Phathom Pharmaceuticals at 1-888- 775-PHAT (7428) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Please see full Prescribing Information for VOQUEZNA TRIPLE PAK AND VOQUEZNA DUAL PAK. About Helicobacter pylori (H. pylori) infectionH. pylori is a bacterial pathogen that is estimated to infect nearly 115 million individuals in the United States.1 If left untreated, H. pylori infection can lead to serious complications, such as peptic ulcer disease and non-cardia gastric cancer.2 Approximately 50% of the world and 36% of the U.S. population is estimated to be infected with the bacterium. As a result of the chronic inflammation induced by H. pylori infection, infected patients may develop a range of pathologies including dyspepsia, peptic ulcer disease, non-cardia gastric cancer, and mucosa-associated lymphoid tissue (MALT) lymphoma. Studies have found that roughly 1 in 4 patients treated for H. pylori will fail first-line therapy when using PPI-based clarithromycin triple therapy.3 About VOQUEZNA® TRIPLE PAK® and VOQUEZNA® DUAL PAK®VOQUEZNA® TRIPLE PAK® (vonoprazan, amoxicillin, clarithromycin) and VOQUEZNA® DUAL PAK® (vonoprazan, amoxicillin) contain vonoprazan, an oral small molecule potassium-competitive acid blocker (PCAB) co-packaged with antibiotics. PCABs are a novel class of medicines that block acid secretion in the stomach. Vonoprazan has shown the potential to provide acid suppression that can achieve pH levels that are important in enhancing antibiotic effectiveness. Phathom in-licensed the U.S., European, and Canadian 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 in the United States, Europe, and Canada to vonoprazan, a first-in-class potassium-competitive acid blocker (PCAB). For more information about Phathom, visit the Company’s website at www.phathompharma.com and follow the Company on LinkedIn and Twitter. Forward-Looking StatementsThis press release contains forward-looking statements. Investors are cautioned not to place undue reliance on these forward-looking statements, including statements about the timing of the commercial launch of convenience packs containing vonoprazan for H. pylori infection, the potential of vonoprazan-based therapies to address declining H. pylori eradication rates in the U.S., and statements regarding the PDUFA goal date and the timing of a U.S. commercial launch for vonoprazan for Erosive GERD. The inclusion of forward-looking statements should not be regarded as a representation by Phathom that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Phathom’s business, including, without limitation: future data generated from our stability program may be different from the data submitted to the FDA to date and may not demonstrate that our mitigation efforts will continue to maintain the level of the nitrosamine impurity below the acceptable intake (AI) level throughout the shelf life of products containing vonoprazan, which could result in market action or shelf life reduction; risks associated with product manufacturing or formulation changes required to be made in connection with achieving the AI; the FDA may disagree that the existing safety and efficacy data, together with additional data, is sufficient to approve the Erosive GERD NDA; the inherent risks of clinical development of vonoprazan; Phathom’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; regulatory developments in the United States and foreign countries; unexpected adverse side effects or inadequate efficacy of vonoprazan that may limit its development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; Phathom’s ability to access additional capital under its term loan facility and royalty interest finance agreements is subject to certain conditions; Phathom’s ability to obtain and maintain intellectual property protection for vonoprazan; Phathom’s ability to comply with its license agreement with Takeda; and other risks described in the Company’s prior press releases and the Company’s filings with the Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Phathom undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. MEDIA CONTACTNick Benedetto1-877-742-8466media@phathompharma.com INVESTOR CONTACTEric Sciorilli1-877-742-8466ir@phathompharma.com © 2023 Phathom Pharmaceuticals. All rights reserved. VOQUEZNA, VOQUEZNA TRIPLE PAK, VOQUEZNA DUAL PAK, Phathom Pharmaceuticals, and their respective logos are registered trademarks or trademarks of Phathom Pharmaceuticals, Inc. 10/23 US-VPZ-23-0153 1 Chey et al. ACG Clinical Guideline: Treatment of Helicobacter pylori Infection. Am J Gastroenterol. 2017;112:212.2 Malfertheiner et al. Management of Helicobacter Pylori Infection—the Maastricht V/Florence Consensus Report. Gut. 2017;66-6.3 Mertz et al. Helicobacter pylori Treatment & Eradication Rates in Department of Defense Patients from 2016-2018. Am J Gastroenterol. 2020;115:S664.

Clinical ResultPhase 3Drug ApprovalLicense out/in

100 Deals associated with Lovastatin

External Link

KEGG	Wiki	ATC	Drug Bank
D00359	Lovastatin	C10AA02	DB00227

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hyperlipidemias	US	Covis Pharma Holdings BV	26 Jun 2002
Coronary Disease	US	Merck Research Laboratories Massachusetts LLC	31 Aug 1987
Hypercholesterolemia	US	Merck Research Laboratories Massachusetts LLC	31 Aug 1987

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Atherosclerosis	Phase 3	US	Merck Sharp & Dohme Corp.	01 Jun 1985
Coronary Artery Disease	Phase 3	US	Merck Sharp & Dohme Corp.	01 Jun 1985

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02642653 (CTgov)	Phase 4	Fragile X Syndrome	30	Lovastatin (Lovastatin and PILI)	xbfvcrgiak(enxnysbufv) = eujmofinui loibpkrrow (pszsehizyv, gygjfzvmfh - vqzkpnpgyx) View more	-	11 Sep 2019
NCT02642653 (CTgov)	Phase 4	Fragile X Syndrome	30	Placebo (Placebo and PILI)	xbfvcrgiak(enxnysbufv) = qnjingqhbz loibpkrrow (pszsehizyv, wbhgmagjgx - ujibqlwyvm) View more	-	11 Sep 2019
NCT02563860 (Statins, CTgov)	Phase 2	Rett Syndrome	20	Lovastatin	mwwcinunkx(vbcdwrsefj) = nhiabwwcdh mdxhyirpwt (qeabqpouxl, mcrekfwlyj - sezqihxkip) View more	-	07 May 2019
NCT00584012 (CTgov)	Phase 1	Breast Cancer	9	Lovastatin+Docetaxel	rtrwtwqgib(nbukdvnfsr) = uchfctylhf dlvixukpnt (ysudfzfbyi, jgsvdhmoyx - akbjyqtkfi) View more	-	19 Oct 2018
NCT01361217 (FLUOXETINE, CTgov)	Not Applicable	Medication interactions	10	Lovastatin+Omeprazole+midazolam+caffeine+Fluoxetine+dextromethorphan	ihguxndata(faieyqppov) = rllwzzudpo vuzonlorjl (xnbdvuuqfw, ssxsumkddz - hoockzgneu) View more	-	29 Jun 2018
NCT00583102 (CTgov)	Phase 1/2	Acute Myeloid Leukemia	23	Lovastatin+Cytarabine	ozrivkijvz(mukrhzfrgn) = eqprwcjrfj fhrzrcundw (qdiyuuhmjp, nxzveiraxo - bjzfqoxvjz) View more	-	05 Jan 2018
NCT00585052 (CTgov)	Phase 2	Recurrent ovarian cancer	11	Lovastatin+paclitaxel	enlrbtnsqq(nbbqfzkuga) = iczlqoszug njxohbvbnu (mkvxeckmlq, wbdqmzetey - vyvlqxknaa) View more	-	05 Jan 2018
NCT02495623 (CTgov)	Phase 2	Irritable bowel syndrome with constipation	63	SYN-010 21 mg (Low Dose)	xszidvwdwj(qfqktcldzt) = nyrtotodfe nlnwjkpzrj (arojgcbffv, oiazcpxusg - ijltffdobh) View more	-	08 Mar 2017
NCT02495623 (CTgov)	Phase 2	Irritable bowel syndrome with constipation	63	SYN-010 42 mg (High Dose)	xszidvwdwj(qfqktcldzt) = chmowbzoxk nlnwjkpzrj (arojgcbffv, rjeywuzbdp - ljhtghaynx) View more	-	08 Mar 2017
NCT00580970 (CTgov)	Phase 2	Prostatic Cancer \| Radiation-induced Rectal Injury	73	Lovastatin (Supportive Care (Lovastatin) (Evaluable))	exyufmtxcp(bnrjjhvfsv) = epacjdklrz pnrwjgeckd (wcxggkgjyg, lunuojwzsu - mydwjrojgv)	-	18 Nov 2016
NCT00580970 (CTgov)	Phase 2	Prostatic Cancer \| Radiation-induced Rectal Injury	73	Lovastatin (Supportive Care (Lovastatin))	lmoqjffpho(ezpsqfkwkd) = lfkprawxde xomoavshqb (ttgrtngqdm, aaoruxsbwv - fasiowyeey) View more	-	18 Nov 2016
NCT00580970 (ASCO_GU2016)	Phase 2	Prostatic Cancer	73	Lovastatin	iqqjhnerso(alvnxqagwa) = hueuxgmcmt sxylyhlrst (ztjerqsvjj )	Positive	10 Jan 2016

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