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Last update 25 Mar 2025

Vonafexor

Last update 25 Mar 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

EYP-001, EYP-001A, EYP001

+ [3]

Target

FXR

Action

agonists

Mechanism

FXR agonists(Bile acid receptor FXR agonists)

Therapeutic Areas

Congenital DisordersSkin and Musculoskeletal DiseasesUrogenital Diseases+ [1]

Active Indication

Nephritis, Hereditary

Inactive Indication

Diabetes Mellitus, Type 2Hepatitis BHepatitis B, Chronic+ [2]

Originator Organization

Poxel SA

Active Organization

ENYO Pharma SAStartup

Inactive Organization

Poxel SA

Drug Highest PhasePhase 2

First Approval Date-

RegulationOrphan Drug (United States), Orphan Drug (European Union)

Structure/Sequence

Molecular FormulaC19H15Cl3N2O5S

InChIKeyXLGQSYUNOIJBNR-UHFFFAOYSA-N

CAS Registry1192171-69-9

Clinical Trials associated with Vonafexor

CTIS2023-509638-20-00

/ RecruitingPhase 2

Vonafexor fixed dose-escalation safety and proof-of-concept study in patients with at risk of progression Alport syndrome - EYP001-208

Start Date07 Oct 2024

Sponsor / Collaborator

ENYO Pharma SAStartup

NCT06425055

/ Active, not recruitingPhase 2

Vonafexor Fixed Dose-escalation Safety and Proof-of-concept Study in Patients with At Risk of Progression Alport Syndrome

This study is a proof-of-concept trial of vonafexor safety, its effects on kidney function in subjects with at risk of progression Alport syndrome.

Start Date01 Aug 2024

Sponsor / Collaborator

ENYO Pharma SAStartup

CTIS2023-509192-16-00

/ Not yet recruitingPhase 2

A study to Assess the Effect of Vonafexor on Kidney Function in Subjects With Impaired Renal Function and Suspected MASH - EYP001-210

Start Date14 Mar 2024

Sponsor / Collaborator

ENYO Pharma SAStartup

100 Clinical Results associated with Vonafexor

100 Translational Medicine associated with Vonafexor

100 Patents (Medical) associated with Vonafexor

Literatures (Medical) associated with Vonafexor

01 Apr 2023·European journal of medicinal chemistry

Machine learning- and structure-based discovery of a novel chemotype as FXR agonists for potential treatment of nonalcoholic fatty liver disease

Article

Author: Zhang, Wenxuan ; Shen, Zhufang ; Xia, Jie ; Qin, Tong ; Feng, Jing ; Hu, Yuhua ; Lei, Lei ; Zhang, Liangren ; Liu, Zhenming ; Huan, Yi ; Gao, Xuefeng ; Wu, Song

Farnesoid X receptor (FXR) is a promising target for drug discovery against nonalcoholic fatty liver disease (NAFLD). However, no FXR agonist has been approved for NAFLD so far. The R & D of FXR agonists are somewhat hindered by the lack of effective and safe chemotypes. To this end, we developed a multi-stage computational workflow to screen the Specs and ChemDiv chemical library for FXR agonists, which consisted of machine learning (ML)-based classifiers, shape-based and electrostatic-based models, a FRED-based molecular docking protocol, an ADMET prediction protocol and substructure search. As a result, we identified a novel chemotype that has never been reported before, with compound XJ02862 (ChemDiv ID: Y020-6413) as the representative. By designing an asymmetric synthesis strategy, we were able to prepare four isomers of compound XJ02862. Interestingly, one of the isomers, 2-((S)-1-((2S,4R)-2-methyl-4-(phenylamino)-3,4-dihydroquinolin-1(2H)-yl)-1-oxopropan-2-yl)hexahydro-1H-isoindole-1,3(2H)-dione (XJ02862-S2), showed potent FXR agonistic activity in HEK293T cells. The molecular docking, molecular dynamics simulations and site-directed mutagenesis suggested the hydrogen bond between compound XJ02862-S2 and HIS294 of FXR is essential for ligand binding. We further demonstrated that compound XJ02862-S2 had no agonistic effect on TGR5. Further biological experiments have shown that compound XJ02862-S2 could ameliorate hypercholesterolemia, hepatic steatosis, hyperglycemia, insulin resistance (IR) in high-fat-diet induced obese (DIO) mice. In term of molecular mechanism, compound XJ02862-S2 regulates the expression of FXR downstream genes involved in lipogenesis, cholesterol transport and bile acid biosynthesis and transport. Taken together, we have discovered a novel chemotype as potent FXR agonists for NAFLD by computational modeling, chemical synthesis and biological evaluation.

01 Mar 2023·Biochemical pharmacology

Pharmacological characterization of second generation FXR agonists as effective EphA2 antagonists: A successful application of target hopping approach

Article

Author: Lodola, Alessio ; Giorgio, Carmine ; Galvani, Francesca ; Zappia, Alfonso ; Mor, Marco ; Ballabeni, Vigilio ; Barocelli, Elisabetta ; Bertoni, Simona ; Scalvini, Laura ; Tognolini, Massimiliano ; Ferrari, Francesca Romana

It is well demonstrated the key role of Eph-ephrin system, specifically of EphA2 receptor, in supporting tumor growth, invasion, metastasis and neovascularization. We previously identified FXR agonists as eligible antagonists of Eph-ephrin system. Herein we characterize new commercially available FXR (Farnesoid X Receptor) agonists as potential Eph ligands including Cilofexor, Nidufexor, Tropifexor, Turofexorate isopropyl and Vonafexor. Our exploration based on molecular modelling investigations and binding assays shows that Cilofexor binds specifically and reversibly to EphA2 receptor with a Ki value in the low micromolar range. Furthermore, Cilofexor interferes with the phosphorylation of EphA2 and the cell retraction and rounding in PC3 prostate cancer cells, both events depending on EphA2 activation. In conclusion, we can confirm that target hopping can be a successful approach to discover new moiety of protein-protein inhibitors.

01 Mar 2023·Journal of hepatology

Hepatic and renal improvements with FXR agonist vonafexor in individuals with suspected fibrotic NASH

Article

Author: Darteil, Raphaël ; Harrison, Stephen A ; Lawitz, Eric ; Alkhouri, Naim ; Francque, Sven ; Bureau, Christophe ; Vonderscher, Jacky ; Sanyal, Arun ; Scalfaro, Pietro ; Abdelmalek, Manal ; Wolf, Myles ; Loustaud-Ratti, Véronique ; Girma, Hugo ; Couchoux, Harold ; Ratziu, Vlad

BACKGROUND & AIMS:

The LIVIFY trial investigated the safety, tolerability, and efficacy of vonafexor, a second-generation, non-bile acid farnesoid X receptor agonist in patients with suspected fibrotic non-alcoholic steatohepatitis (NASH).

METHODS:

This double-blind phase IIa study was conducted in two parts. Patients were randomised (1:1:1:1) to receive placebo, vonafexor 100 mg twice daily (VONA-100BID), vonafexor 200 mg once daily (VONA-200QD), or 400 mg vonafexor QD (VONA-400QD) in Part A (safety run-in, pharmacokinetics/pharmacodynamics) or placebo, vonafexor 100 mg QD (VONA-100QD), or VONA-200QD (1:1:1) in Part B. The primary efficacy endpoint was a reduction in liver fat content (LFC) by MRI-proton density fat fraction, while secondary endpoints included reduced corrected T1 values and liver enzymes, from baseline to Week 12.

RESULTS:

One hundred and twenty patients were randomised (Part A, n = 24; Part B, n = 96). In Part B, there was a significant reduction in least-square mean (SE) absolute change in LFC from baseline to Week 12 for VONA-100QD (-6.3% [0.9]) and VONA-200QD (-5.4% [0.9]), vs. placebo (-2.3% [0.9], p = 0.002 and 0.012, respectively). A >30% relative LFC reduction was achieved by 50.0% and 39.3% of patients in the VONA-100QD and VONA-200QD arms, respectively, but only in 12.5% in the placebo arm. Reductions in body weight, liver enzymes, and corrected T1 were also observed with vonafexor. Creatinine-based glomerular filtration rate improved in the active arms but not the placebo arm. Mild to moderate generalised pruritus was reported in 6.3%, 9.7%, and 18.2% of participants in the placebo, VONA-100QD, and VONA-200QD arms, respectively.

CONCLUSIONS:

In patients with suspected fibrotic NASH, vonafexor was safe and induced potent liver fat reduction, improvement in liver enzymes, weight loss, and a possible renal benefit.

CLINICAL TRIAL NUMBER (EUDRACT):

2018-003119-22.

CLINICALTRIALS:

GOV IDENTIFIER:

NCT03812029.

IMPACT AND IMPLICATIONS:

Non-alcoholic steatohepatitis (NASH) has become a leading cause of chronic liver disease worldwide. Affected patients are also at higher risk of developing chronic kidney disease. There are no approved therapies and only few options to treat this population. The phase IIa LIVIFY trial results show that single daily administration of oral vonafexor, an FXR agonist, leads in the short term to a reduction in liver fat, liver enzymes, fibrosis biomarkers, body weight and abdominal circumference, and a possible improvement in kidney function, while possible mild moderate pruritus (a peripheral FXR class effect) and an LDL-cholesterol increase are manageable with lower doses and statins. These results support exploration in longer and larger trials, with the aim of addressing the unmet medical need in NASH.

News (Medical) associated with Vonafexor

30 Jan 2025

·www.prnewswire.com

Metabolic Dysfunction-Associated Steatohepatitis Market to Register Stunning Growth at a CAGR of 17.9% in the US by 2034 | DelveInsight

The MASH treatment landscape is evolving rapidly, driven by a diverse array of therapeutic classes, each with unique mechanisms and potential impacts. The forecast period reveals significant shifts and emerging trends that will shape future market dynamics. LAS VEGAS, Jan. 30, 2025 /PRNewswire/ -- DelveInsight's Metabolic Dysfunction-Associated Steatohepatitis Market Insights report includes a comprehensive understanding of current treatment practices, MASH emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Metabolic Dysfunction-Associated Steatohepatitis Market Report According to DelveInsight's analysis, the market size for MASH was found to be USD 2.1 billion in the 7MM in 2023. In 2023, there were an estimated 42 million prevalent cases of MASH in the 7MM. Out of these, a total of ~15 million cases were diagnosed, and this number is projected to increase by the end of 2034 in the 7MM. Leading MASH companies such as Inventiva Pharma, Novo Nordisk A/S, Cirius Therapeutics, Akero Therapeutics, 89bio, Boehringer Ingelheim, Zealand Pharma, Galectin Therapeutics, Lipocine, Viking Therapeutics, Eli Lilly and Company, Boston Pharmaceuticals, Pfizer, HighTide Biopharma, CytoDyn, Merck & Co., Hanmi Pharmaceutical, Hepagene (Shanghai), Hepion Pharmaceuticals, Enyo Pharmaceuticals, Gilead Sciences, Poxel SA, Zydus Therapeutics, Sagimet Biosciences, Ionis Pharmaceuticals, Corcept Therapeutics, and others are developing novel MASH drugs that can be available in the MASH market in the coming years. The promising MASH therapies in the pipeline include Lanifibranor (IVA337), Semaglutide, Azemiglitazone (MSDC-0602K), Efruxifermin (EFX), BIO89-100 (Pegozafermin), Survodutide (BI 456906), GR-MD-02 (Belapectin), LPCN1144, VK2809, Tirzepatide, BOS-580, Ervogastat (PF-06865571) + Clesacostat (PF-05221304), HTD1801, Leronlimab (PRO 140), Efinopegdutide, HPG1860, Rencofilstat (CRV431), EYP001 (Vonafexor), Semaglutide/Cilofexor/Firsocostat, PXL065, Saroglitazar Magnesium, Denifanstat (TVB-2640), ION224, Miricorilant (CORT118335), and others. In March 2024, Madrigal Pharmaceuticals' groundbreaking product, REZDIFFRA (resmetirom), a once-daily, oral THR-ß agonist, received accelerated endorsement from the US FDA based on results from the Phase III MAESTRO-NASH trial. This approval marks a significant stride in the medical landscape, as REZDIFFRA becomes the inaugural and sole FDA-sanctioned therapy for adults afflicted with non-cirrhotic MASH, accompanied by moderate to advanced liver scarring (fibrosis) corresponding to stages F2–F3 fibrosis. Discover which therapies are expected to grab the major MASH market share @ Metabolic Dysfunction-Associated Steatohepatitis Market Report Metabolic Dysfunction-Associated Steatohepatitis Overview Metabolic dysfunction-associated steatohepatitis (MASH) is a progressive liver disease that stems from metabolic dysfunction, often linked to obesity, diabetes, and other conditions of metabolic syndrome. MASH is characterized by the accumulation of fat in liver cells, accompanied by inflammation and liver cell injury, which can progress to fibrosis, cirrhosis, or even liver cancer. The primary drivers of MASH include insulin resistance, obesity, type 2 diabetes, and dyslipidemia. Genetic predisposition and a sedentary lifestyle also play significant roles. Environmental factors, such as a poor diet high in sugars and fats, exacerbate the condition. MASH is often asymptomatic in its early stages. When symptoms occur, they can include fatigue, vague abdominal discomfort, or pain in the upper right quadrant. In advanced stages, signs of liver dysfunction such as jaundice, swelling of the abdomen or legs, and confusion may arise. Diagnosis involves a combination of clinical history, physical examination, and diagnostic tests. Blood tests measuring liver enzymes (ALT, AST) often indicate liver inflammation. Imaging techniques like ultrasound, MRI, or FibroScan can identify liver fat and fibrosis. In some cases, a liver biopsy is required to confirm the diagnosis and assess disease severity. Metabolic Dysfunction-Associated Steatohepatitis Epidemiology Segmentation The MASH epidemiology section provides insights into the historical and current MASH patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The MASH market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Prevalent Cases of MASH Diagnosed Prevalent Cases of MASH Gender-specific Diagnosed Prevalent Cases of MASH Severity-specific Diagnosed Prevalent Cases of MASH Metabolic Dysfunction-Associated Steatohepatitis Treatment Market The approval of REZDIFFRA (resmetirom) in March 2024 represents a pivotal achievement in medical innovation, transforming the treatment landscape for MASH disease. This groundbreaking therapy addresses the root causes of MASH, offering renewed hope to patients grappling with this challenging condition. Clinical trials have shown impressive results, with REZDIFFRA effectively reducing symptoms like inflammation and fibrosis, enhancing liver function, and improving patients' quality of life. By providing healthcare professionals with a robust treatment option, this approval addresses a critical unmet need and has the potential to significantly alleviate the complications linked to advanced liver disease. The prevalence of MASLD is strongly linked to type 2 diabetes mellitus and obesity, particularly in individuals with a higher body mass index. However, MASLD occurrence is reduced in T2DM patients receiving treatments such as sodium-glucose cotransporter-2 (SGLT2) inhibitors, GLP-1 receptor agonists, and insulin. Vitamin E, with its antioxidant properties, is regarded as a first-line pharmacological option for managing MASH, especially when dietary and lifestyle interventions are insufficient. Antifibrotic agents can help prevent the progression of liver fibrosis and MASLD into fibrotic MASH. The role of pioglitazone, an anti-diabetic medication, in improving MASH histology in T2DM patients is well-documented, but concerns remain regarding potential side effects like weight gain, fluid retention, cancer risk, and bone fractures. Additional therapeutic targets for MASLD and MASH include G protein-coupled receptors (GPCRs), estrogen-related receptor alpha (ERRα), bone morphogenetic proteins (BMPs), and KLFs. Bariatric surgery, or weight loss surgery, is the most effective intervention for treating obesity and diabetes, as it reduces food absorption while influencing gut hormone secretion and metabolic function. To know more about MASH treatment guidelines, visit @ Metabolic Dysfunction-Associated Steatohepatitis Management Metabolic Dysfunction-Associated Steatohepatitis Pipeline Therapies and Key Companies Lanifibranor (IVA337): Inventiva Pharma Semaglutide: Novo Nordisk A/S Azemiglitazone (MSDC-0602K): Cirius Therapeutics Efruxifermin (EFX): Akero Therapeutics BIO89-100 (Pegozafermin): 89bio Survodutide (BI 456906): Boehringer Ingelheim/Zealand Pharma GR-MD-02 (Belapectin): Galectin Therapeutics LPCN1144: Lipocine VK2809: Viking Therapeutics Tirzepatide: Eli Lilly and Company BOS-580: Boston Pharmaceuticals Ervogastat (PF-06865571) + Clesacostat (PF-05221304): Pfizer HTD1801: HighTide Biopharma Leronlimab (PRO 140): CytoDyn Efinopegdutide: Merck & Co./Hanmi Pharmaceutical HPG1860: Hepagene (Shanghai) Rencofilstat (CRV431): Hepion Pharmaceuticals EYP001 (Vonafexor): Enyo Pharmaceuticals Semaglutide/ Cilofexor/ Firsocostat: Gilead Sciences PXL065: Poxel SA Saroglitazar Magnesium: Zydus Therapeutics Denifanstat (TVB-2640): Sagimet Biosciences ION224: Ionis Pharmaceuticals Miricorilant (CORT118335): Corcept Therapeutics Discover more about MASH drugs in development @ Metabolic Dysfunction-Associated Steatohepatitis Clinical Trials Metabolic Dysfunction-Associated Steatohepatitis Market Dynamics The MASH market dynamics are expected to change in the coming years. Growing research activities and multiple clinical trials for MASH, driven by the rapid surge in its prevalence due to rising obesity and type 2 diabetes rates, highlight an active drug development pipeline and an expanding market size. The large pool of patients and lucrative growth opportunities present attractive prospects for key players, further supported by ongoing preclinical studies aimed at advancing imaging techniques for MASH diagnosis, potentially eliminating the need for invasive biopsy-based histopathological confirmation. Furthermore, potential therapies are being investigated for the treatment of MASH, and it is safe to predict that the treatment space will significantly impact the MASH market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the MASH market in the 7MM. However, several factors may impede the growth of the MASH market. Lack of awareness and negligence in the early stages of MASH by physicians often lead to disease progression, culminating in irreversible damage where liver transplantation becomes the only viable option. Diagnosing advanced MASH typically requires procedures like liver biopsy, which are costly, invasive, and risky. Regulatory challenges also pose hurdles, as the FDA mandates achieving one MASH endpoint for approval, while the EMA's draft guidance requires efficacy in both endpoints, potentially delaying first-mover approvals in major European markets. Additionally, access to expensive MASH treatments may be limited in certain regions, further hindering patient adoption. Moreover, MASH treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, MASH market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact MASH market growth. Scope of the Metabolic Dysfunction-Associated Steatohepatitis Market Report Therapeutic Assessment: Metabolic Dysfunction-Associated Steatohepatitis current marketed and emerging therapies Metabolic Dysfunction-Associated Steatohepatitis Market Dynamics: Key Market Forecast Assumptions of Emerging Metabolic Dysfunction-Associated Steatohepatitis Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Metabolic Dysfunction-Associated Steatohepatitis Market Access and Reimbursement Download the report to understand which factors are driving MASH market trends @ Metabolic Dysfunction-Associated Steatohepatitis Market Trends Table of Contents Related Reports Non-Alcoholic Steatohepatitis Epidemiology Forecast Non-Alcoholic Steatohepatitis Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted NASH epidemiology in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, Spain), the United Kingdom, and Japan. Nonalcoholic Steatohepatitis Pipeline Nonalcoholic Steatohepatitis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key nonalcoholic steatohepatitis companies, including Madrigal Pharmaceuticals, Intercept Pharmaceuticals, Cirius Therapeutics, Novo Nordisk, Inventiva, Galmed Pharmaceuticals, AstraZeneca, Galectin Therapeutics, Viking Therapeutics, Eli Lilly and Company, Terns Pharmaceuticals, Sinew Pharma, 89bio, Inc., Eccogene, Novartis Pharmaceuticals, Poxel SA, AngioLab, Pfizer, Arrowhead Pharma, LG Chem, Redx Pharma, Lipocine, Inc., CytoDyn, Inc., Alnylam Pharmaceuticals, Inc., Chemomab Therapeutics, NuSirt Biopharma, HK inno. N, Aligos Therapeutics, Altimmune, Inc., Kowa Pharmaceutical, Ionis Pharmaceuticals, NorthSea Therapeutics, Rivus Pharmaceuticals, Hanmi Pharmaceutical, Hepagene Therapeutics, HighTide Biopharma, Akero Therapeutics, Merck Sharp & Dohme LLC, Cascade Pharmaceuticals, Hepion Pharmaceuticals, Chipscreen Biosciences, Boston Pharmaceuticals, Bristol-Myers Squibb, Sunshine Lake Pharma, GSK plc., Future Medicine, Gilead Sciences, ENYO Pharma, Histogen, among others. Non-Alcoholic Fatty Liver Disease Market Non-Alcoholic Fatty Liver Disease Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key NAFLD companies, including MediciNova, Eli Lilly and Company, AstraZeneca, Inventiva Pharma, Oasis Pharmaceuticals, LLC, Madrigal Pharmaceuticals, Inc., BioMarin Pharmaceutical, GlaxoSmithKline, Zydus Therapeutics Inc., Akero Therapeutics, Inc, Pfizer, Boehringer Ingelheim, Neuraly, Inc., Merck Sharp & Dohme LLC, Rivus Pharmaceuticals, Inc., Hepion Pharmaceuticals, Inc. , among others. Non-Alcoholic Fatty Liver Disease Pipeline Non-Alcoholic Fatty Liver Disease Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key NAFLD companies, including BeiGene, Inventiva Pharma, Cirius Therapeutics, Madrigal Pharma, Novo Nordisk, Galmed Pharmaceuticals, AstraZeneca, Galectin Therapeutics, Viking Therapeutics, Dr. Falk Pharma GmbH, Sagimet Biosciences, Eli Lilly and Company, Terns Pharmaceuticals, Sinew Pharma, Novartis Pharmaceuticals, Afimmune, Poxel SA, AngioLab, Pfizer, Oramed Pharmaceuticals, Can Fite Biopharma, MediciNova, Metacrine, Inc., Lipocine, Inc., CytoDyn, Inc., Alnylam Pharmaceuticals, Inc., Mitsubishi Tanabe Pharma, Chemomab Therapeutics, NuSirt Biopharma, HK inno.N, Kowa Pharmaceutical, Ionis Pharmaceuticals, NorthSea Therapeutics, Rivus Pharmaceuticals, Hanmi Pharmaceutical, Hepagene Therapeutics, HighTide Biopharma, Akero Therapeutics, Enanta Pharmaceuticals, Cascade Pharmaceuticals, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3

20 Jun 2024

·www.enyopharma.com

The Phase 2 ALPESTRIA-1 clinical study has started with Vonafexor on Alport syndrome

Published on 20/06/2024 The ALPESTRIA-1 Phase 2 clinical study (NCT06425055) is an open-label, single arm, fixed dose escalation of the investigational drug Vonafexor. This study is a proof-of-concept trial of Vonafexor safety, its effects on kidney function in subjects with at risk of progression Alport syndrome. The study aims to recruit 20 patients in the US and EU (France, Spain and Germany). The first site for patient recruitment is now open in Idaho (US) and more sites will open in the coming weeks. More information about the study here: The first patient dosed in the Phase 2 ALPESTRIA-1 clinical study of Vonafexor for Alport syndrome Orphan Drug Designation (ODD) granted to Vonafexor for Alport syndrome by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) New publication: Hepatic and renal improvements with FXR agonist Vonafexor in individuals with suspected fibrotic NASH New publication : An anti-diabetic drug targets NEET (CISD) proteins through destabilization of their [2Fe-2S] clusters Swiss NASH Foundation sponsorship

Orphan DrugPhase 2

03 Jan 2024

·www.businesswire.com

ENYO Pharma Announces a €39 Million Series C Financing and FDA Clearance to Advance Vonafexor in a Phase 2 Clinical Trial for Patients With Alport Syndrome

LYON, France--( BUSINESS WIRE )--ENYO Pharma (“ENYO”) announced that it has received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) to initiate a Phase 2 clinical study of Vonafexor, a highly selective FXR agonist, for the treatment of Alport Syndrome. Furthermore, ENYO announced the first closing of a 39 million € Series C equity financing, positioning ENYO as an emerging biotechnology company in the field of renal diseases. The financing will support the Phase 2 Alpestria-1 study and further profiling of Vonafexor in other kidney diseases, such as Autosomal Dominant Polycystic Kidney Disease (ADPKD). The financing was co-led by OrbiMed and Morningside Ventures with participation from other existing investors including AndEra Partners, Bpifrance InnoBio and Bpifrance Large Ventures. The clearance of ENYO’s IND represents a significant milestone for the company as it embarks on its first clinical trial focusing on renal disease. ENYO’s lead candidate Vonafexor, as well as fast follower EYP651, are highly specific FXR agonists given as once-daily oral treatments. Both compounds have strong fibrolytic and anti-inflammatory properties that are broadly applicable across several renal diseases. Vonafexor effect on renal function (eGFR) was already shown in the Phase 2 LIVIFY study of patients with both kidney impairment and fibrotic liver disease. Preclinical studies in both Alport syndrome and Chronic Kidney Disease mouse models showed beneficial effects of Vonafexor on kidney remodeling and function in a curative mode. In 2023, Vonafexor was granted as Orphan Drug Designation (ODD) by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) for Alport syndrome. ” We are delighted with this support from our existing investors” said Dr. Jacky Vonderscher, CEO at ENYO Pharma. “ There are huge unmet needs in many kidney diseases with fibrosis or inflammatory components. These patients almost invariably progress to end-stage kidney disease despite current therapies. The results already obtained with Vonafexor, our highly differentiated anti-inflammatory and fibrolytic lead compound, in patients with moderate kidney impairment and in several preclinical protocols, make us confident that it will greatly benefit to those patients with rare kidney diseases like Alport syndrome. “ ” Kidney diseases were somewhat neglected for decades, and it is refreshing to see a team like ENYO pursuing with persistence the development of Vonafexor for the benefit of these patients. “ said Iain Dukes of OrbiMed . About ENYO Pharma: For more information : ENYO Pharma – Developing therapeutics for diseases with impaired kidney function About OrbiMed: For more information : OrbiMed

Phase 2Orphan DrugIND

100 Deals associated with Vonafexor

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Nephritis, Hereditary	Phase 2	United States	ENYO Pharma SAStartup	01 Aug 2024
Nephritis, Hereditary	Phase 2	France	ENYO Pharma SAStartup	01 Aug 2024
Nephritis, Hereditary	Phase 2	Spain	ENYO Pharma SAStartup	01 Aug 2024
Hepatitis B, Chronic	Phase 2	Australia	ENYO Pharma SAStartup	12 May 2020
Hepatitis B, Chronic	Phase 2	Hong Kong	ENYO Pharma SAStartup	12 May 2020
Hepatitis B, Chronic	Phase 2	Poland	ENYO Pharma SAStartup	12 May 2020
Hepatitis B, Chronic	Phase 2	South Korea	ENYO Pharma SAStartup	12 May 2020
Nonalcoholic Steatohepatitis	Phase 2	United States	ENYO Pharma SAStartup	30 Jan 2019
Nonalcoholic Steatohepatitis	Phase 2	Belgium	ENYO Pharma SAStartup	30 Jan 2019
Nonalcoholic Steatohepatitis	Phase 2	France	ENYO Pharma SAStartup	30 Jan 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03812029 (CTgov)	Phase 2	Nonalcoholic Steatohepatitis	120	Placebo (Placebo Part B)	invjzlcbdj(jfcfohrhmk) = oauwouadvp mraeawoifv (huqvmcdtip, kkebmrjswk - uovikxqvge) View more	-	06 May 2023
				Vonafexor (Vonafexor 100 mg QD Part B)	invjzlcbdj(jfcfohrhmk) = tmmlfuuhly mraeawoifv (huqvmcdtip, dqufeygpbo - pppitphore) View more
NCT04465916 (CTgov)	Phase 2	Hepatitis B, Chronic	26	Nucleotide analogue (Entecavir or Tenofovir Disoproxil)+EYP001a (Experimental Arm)	fojzmpmnph(augdewmwla) = roucungmla uskoyqedop (vkyqifmwnn, 0.0195) View more	-	12 Oct 2022
				Nucleotide analogue (Entecavir or Tenofovir Disoproxil) (Control Arm)	fojzmpmnph(augdewmwla) = okqzoxwipt uskoyqedop (vkyqifmwnn, 0.0348) View more
NCT04365933 (EYP001-203, BusinessWire) Manual	Phase 2	Hepatitis B, Chronic	20	Entecavir+Peginterferon alfa-2a+Vonafexor	lsqnfbzdxw(effjwirdwc) = kceeaiamgt nbhdcdtxne (qovshgipfn )	Positive	30 Jul 2021
				Peginterferon alfa-2a+Vonafexor	lsqnfbzdxw(effjwirdwc) = jnikqytawg nbhdcdtxne (qovshgipfn )
UEGW2019	Not Applicable	-	-	EYP001	gctqwmblfo(dmvlsrqpgl) = FGF19 protein was also stimulated significantly by EYP001 gyjvzkbdju (krulhijwwe )	-	01 Oct 2019

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Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis