A Double-Blind, Multicenter, Placebo-Controlled, Randomized, Parallel, Multiple-Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Pegbelfermin in Healthy Overweight and Obese Chinese and Korean Participants

The purpose of this study is to evaluate the safety, tolerability, and drug level of Pegbelfermin in healthy overweight and obese Chinese and Korean participants.

Start Date21 Jun 2021

Sponsor / Collaborator

Bristol Myers Squibb Co.

NCT04634149 / CompletedPhase 1

An Open-label, Single-dose Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of BMS-986036 in Participants With Normal Hepatic Function and Participants With Moderate and Severe Hepatic Impairment

The purpose of this study is to investigate the effect of impaired liver function on the drug levels, safety, and tolerability of BMS-986036 in participants with moderate and severe liver impairment. Results from this study will be used to determine whether dose adjustment is required for patients with decreased liver function.

Start Date23 Nov 2020

Sponsor / Collaborator

Bristol Myers Squibb Co.

NCT04493567 / CompletedPhase 1

An Open-label, Randomized, Two-period Cross-over Study to Investigate the Relative Bioavailability of BMS-986036 in Healthy, Overweight, and Obese Participants Following Subcutaneous Administration Via Auto-injector Versus Pre-filled Syringe

The purpose of this study is to develop an auto-injector (AI) device for the BMS-986036 subcutaneous formulation that can be self-administered conveniently by participants.

Start Date29 Jul 2020

Sponsor / Collaborator

Bristol Myers Squibb Co.

100 Clinical Results associated with Pegbelfermin(Ambrx, Inc.)

100 Translational Medicine associated with Pegbelfermin(Ambrx, Inc.)

100 Patents (Medical) associated with Pegbelfermin(Ambrx, Inc.)

Literatures (Medical) associated with Pegbelfermin(Ambrx, Inc.)

01 Jul 2024·Clinical pharmacology and therapeutics

Efficacy and Safety of Fibroblast Growth Factor‐21 Analogs for the Treatment of Metabolic Dysfunction‐Associated Steatohepatitis: A Systematic Review and Meta‐Analysis

Review

Author: Kim, Min‐Soo ; Jeon, Nakyung ; Rhee, Su‐jin ; Han, Nayoung ; Staatz, Christine E. ; Baek, In‐hwan ; Jeong, Changhyeon

Fibroblast growth factor (FGF)‐21 analogs are potential therapeutic candidates for metabolic dysfunction‐associated steatohepatitis (MASH). This systematic review and meta‐analysis aimed to assess the efficacy and safety of the FGF‐21 analogs, efruxifermin, pegbelfermin, and pegozafermin for MASH treatment. A comprehensive systematic review and meta‐analysis of randomized controlled trials from five major databases was conducted. Primary efficacy outcomes focused on liver histological improvement, while secondary efficacy outcomes encompassed reductions in liver fat content and improvements in biochemical parameters. Safety outcomes examined included treatment‐emergent adverse events (TEAEs), treatment‐related TEAEs, TEAEs leading to discontinuation, and serious TEAEs. Eight eligible studies involving 963 patients were included in this review. Compared with the placebo group, the FGF‐21 analog‐treated group exhibited significantly improved primary efficacy outcomes, specifically ≥1 stage improvement in fibrosis with no worsening of MASH (risk ratio [RR] = 1.83; 95% confidence interval [CI] = 1.27–2.62) and at least two‐point improvement in the non‐alcoholic fatty liver disease activity score with no worsening of fibrosis (RR = 2.85; 95% CI = 2.06–3.95). Despite an increased risk of TEAEs (RR = 1.17; 95% CI = 1.08–1.27) and treatment‐related adverse events (RR = 1.75; 95% CI = 1.40–2.19), FGF‐21 analogs exhibited an acceptable safety profile. FGF‐21 analogs were significantly better in achieving liver histological improvements and beneficial biochemical outcomes compared with placebo, with a tolerable safety pattern. These findings shed light on the efficacy and safety of FGF‐21 analogs and provide valuable evidence for their application as MASH therapeutics.

01 Jan 2024·Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association

Pegbelfermin in Patients With Nonalcoholic Steatohepatitis and Compensated Cirrhosis (FALCON 2): A Randomized Phase 2b Study

Article

Author: Harrison, Stephen A ; Lawitz, Eric J ; Goodman, Zachary D ; Neuschwander-Tetri, Brent A ; Jacobson, Ira M ; Yamaguchi, Masayuki ; Shevell, Diane E ; Abdelmalek, Manal F ; Tirucherai, Giridhar S ; Akahane, Takemi ; Charles, Edgar D ; Halegoua-DeMarzio, Dina ; Imajo, Kento ; Du, Shuyan ; Boone, Bradly ; Gunn, Nadege ; Sanyal, Arun J ; Nakajima, Atsushi ; Loomba, Rohit ; Chatterjee, Arkendu

BACKGROUND & AIMS:

Pegbelfermin is a polyethylene glycol-conjugated analog of human fibroblast growth factor 21, a nonmitogenic hormone that regulates energy metabolism. This phase 2b study evaluated 48-week pegbelfermin treatment in patients with nonalcoholic steatohepatitis (NASH) with compensated cirrhosis.

METHODS:

FALCON 2 (NCT03486912) was a randomized (1:1:1:1), double-blind, placebo-controlled study. Eligible adults had biopsy-confirmed NASH and stage 4 fibrosis. Pegbelfermin (10, 20, or 40 mg) or placebo was injected subcutaneously once weekly. The primary endpoint was 1 or more stages of improvement in the NASH Clinical Research Network fibrosis score without NASH worsening at week 48; pegbelfermin dose response was assessed using a Cochran-Armitage trend test across proportions (1-sided α = .05). Additional endpoints included histologic and noninvasive measures of steatosis, fibrosis, and liver injury/inflammation.

RESULTS:

Overall, 155 patients were randomized, and 154 patients received treatment. At week 48, 24% to 28% of the pegbelfermin arms had primary endpoint responses vs 31% of the placebo arm (P = .361). Nonalcoholic fatty liver disease activity score improvements were more frequent with pegbelfermin vs placebo and were driven primarily by reduced lobular inflammation. Numerically higher proportions of the pegbelfermin arms had liver stiffness (magnetic resonance elastography) and steatosis (magnetic resonance imaging-proton density fat fraction) improvements vs placebo; these differences were not statistically significant. Mean N-terminal type III collagen propeptide, alanine aminotransferase, and aspartate aminotransferase values were numerically lower in the 20- and/or 40-mg pegbelfermin arms compared with placebo. Serious adverse events were more frequent with pegbelfermin vs placebo, although none were treatment related. One patient (40-mg pegbelfermin) discontinued treatment because of a treatment-emergent adverse event (worsening ascites).

CONCLUSIONS:

FALCON 2 did not meet its primary endpoint of 1 or more stages of improvement in the NASH Clinical Research Network fibrosis without NASH worsening assessed via biopsy. Pegbelfermin generally was well tolerated in this advanced NASH population.

01 Jan 2024·Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association

Pegbelfermin in Patients With Nonalcoholic Steatohepatitis and Stage 3 Fibrosis (FALCON 1): A Randomized Phase 2b Study

Article

Author: Loomba, Rohit ; Abdelmalek, Manal F ; Goodman, Zachary D ; Lawitz, Eric J ; Harrison, Stephen A ; Gunn, Nadege ; Neuschwander-Tetri, Brent A ; Shevell, Diane E ; Yamaguchi, Masayuki ; Ravendhran, Natarajan ; Tirucherai, Giridhar S ; Akahane, Takemi ; Charles, Edgar D ; Imajo, Kento ; Boone, Bradly ; Sanyal, Arun J ; Du, Shuyan ; Nakajima, Atsushi ; Chatterjee, Arkendu

BACKGROUND & AIMS:

Pegbelfermin is a polyethlene glycol-conjugated analog of human fibroblast growth factor 21, a nonmitogenic hormone that regulates energy metabolism. This phase 2b study evaluated 48-week pegbelfermin treatment in patients with nonalcoholic steatohepatitis (NASH) and stage 3 (bridging) fibrosis.

METHODS:

The FALCON 1 study (NCT03486899) was a multicenter, randomized (1:1:1:1), double-blind, placebo-controlled study. Patients with biopsy-confirmed NASH and stage 3 fibrosis (N = 197) received weekly subcutaneous pegbelfermin (10, 20, or 40 mg) or placebo injections for 48 weeks. The week 24 primary endpoint was a ≥1-point decrease in fibrosis score without NASH worsening or NASH improvement without fibrosis worsening; pegbelfermin dose response was assessed using a Cochran-Armitage trend test across proportions (1-sided α = 0.05). Secondary/exploratory endpoints included histological and noninvasive measures of steatosis, fibrosis, and liver injury/inflammation.

RESULTS:

At week 24, the primary endpoint was met by 14% (placebo) vs 24%-31% (pegbelfermin arms); statistical significance was not reached due to lack of pegbelfermin dose response (P = .134). At weeks 24 and 48, more patients who received pegbelfermin had ≥30% relative reductions in hepatic fat fraction (magnetic resonance imaging-proton density fat fraction) vs placebo, although no differences reached statistical significance. In the pegbelfermin arms, improvements in liver fibrosis (magnetic resonance elastography and N-terminal type III collagen propeptide) and liver injury/inflammation (alanine aminotransferase, aspartate aminotransferase) were observed vs placebo. Adverse events occurred at similar frequencies across arms. No treatment-related serious adverse events were observed.

CONCLUSIONS:

The FALCON 1 study did not meet its primary endpoint; a ≥1-point decrease in fibrosis score without NASH worsening or NASH improvement without fibrosis worsening assessed via biopsy. Pegbelfermin was generally well tolerated during 48 weeks of treatment.

News (Medical) associated with Pegbelfermin(Ambrx, Inc.)

12 Dec 2022

·www.prnewswire.com

Propulsion of Nonalcoholic Steatohepatitis Pipeline as Novel and Extensive 150+ Therapies Likely to Enter in the Treatment Domain | DelveInsight

The prevalence of nonalcoholic steatohepatitis has been rising since the past few years, which prompts the growing demand for treatment options. The increasing prevalence of Nonalcoholic Steatohepatitis and the growing research and development activities drive the market. The Companies developing the potential therapies in the last stage of development include Madrigal Pharmaceuticals, Inventiva, Boehringer Ingelheim, and several others. LAS VEGAS, Dec. 12, 2022 /PRNewswire/ -- DelveInsight's ' Nonalcoholic Steatohepatitis Pipeline Insight – 2022 ' report provides comprehensive global coverage of available, marketed, and pipeline nonalcoholic steatohepatitis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the nonalcoholic steatohepatitis pipeline domain. Key Takeaways from the Nonalcoholic Steatohepatitis Pipeline Report DelveInsight's nonalcoholic steatohepatitis pipeline report depicts a robust space with 130+ active players working to develop 150+ pipeline therapies for nonalcoholic steatohepatitis treatment. Key nonalcoholic steatohepatitis companies such as Madrigal Pharmaceuticals, Intercept Pharmaceuticals, Cirius Therapeutics, Novo Nordisk, Galmed Pharmaceuticals, AstraZeneca, Galectin Therapeutics, Viking Therapeutics, Eli Lilly and Company, Terns Pharmaceuticals, Sinew Pharma, Novartis Pharmaceuticals, Poxel SA, AngioLab, Pfizer, 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, 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, and others are evaluating nonalcoholic steatohepatitis drugs to improve the treatment landscape. Promising nonalcoholic steatohepatitis pipeline therapies in various stages of development include TERN-101, SNP-610, LJN452 (tropifexor + licogliflozin), PXL065, ALS-L1023, PF-06865571 + PF-05221304, PF-06835919, PF-06865571, ORMD-0801, Norucholic acid, NNC0194-0499, MN-001, MK-3655, MET642, MET409, LPCN 1144, LIK066, Leronlimab, ALN-HSD, MT-3995, CM101, Leu-Mit-Sil (NS-0200), IN-A010, Efruxifermin (EFX), Efinopegdutide, EDP-305, JKB-122, CS0159 (Linafexor), CRV431, Chiglitazar sodium, BOS-580, BMS-986263, HEC96719, GSK4532990, FM101, Firsocostat, EPY001a, Emricasan, BMS-986036, BIO89-100, BI 456906, BFKB8488A, AXA1125, ASC 41, GS-9674 (Cilofexor), ZSP1601, and others. In November 2022, Sagimet Biosciences announced positive interim data from its Phase IIb clinical trial (FASCINATE-2) with denifanstat, a fatty acid synthase (FASN) inhibitor, in non-alcoholic steatohepatitis patients. Data showed statistically significant improvements across key markers of NASH, reinforcing results observed in earlier studies, including statistically significant reductions in markers of liver fat, inflammation, and fibrosis. There were no treatment-related serious adverse events, with the majority of adverse events mild to moderate in nature (Grade 1 and 2). Additional interim data are expected in early 2023. In November 2022, Inventiva announced that the United States Patent and Trademark Office granted a patent (U.S. Patent No. 11,504,380) that protects the use of lanifibranor for the treatment of cirrhotic patients at risk of progressing from compensated stage to decompensated stage. This patent will expire on November 8, 2039. This patent further expands the intellectual property protection of lanifibranor in the United States for use in patients with cirrhotic NASH. In October 2022, Galectin Therapeutics, Inc. reported the positive outcome of its second data and safety monitoring board (DSMB) meeting for NAVIGATE, its seamless, adaptive, phase IIb/III study of belapectin in patients with liver cirrhosis caused by non-alcoholic steatohepatitis (NASH). The objective of this second independent DSMB was further to review the emerging tolerance and safety profiles of belapectin. Based on its deliberation, the DSMB concluded that NAVIGATE could continue as designed, without modifications. In September 2022, Inventiva and Chia Tai-Tianqing Pharmaceutical Group entered into a licensing and collaboration agreement to develop and commercialize lanifibranor, if approved, for the treatment of nonalcoholic steatohepatitis and potentially other metabolic diseases in mainland China, Hong Kong, Macau, and Taiwan. In June 2022, Poxel SA announced that the U.S. Patent and Trademark Office (PTO) has issued to Poxel US Patent No. 11319313, which represents a new patent for PXL065, a novel, proprietary deuterium-stabilized R-stereoisomer of pioglitazone which is being investigated in Phase II stage of clinical trial evaluation for the treatment of Nonalcoholic steatohepatitis (NASH). In May 2022, Pfizer Inc. announced the U.S. Food and Drug Administration (FDA) had granted Fast Track designation to Pfizer's investigational combination therapy for the treatment of non-alcoholic steatohepatitis (NASH) with liver fibrosis: ervogastat (PF-06865571, a diacylglycerol O-acyltransferase 2 inhibitor, or DGAT2i) and clesacostat (PF-05221304, an acetyl-CoA carboxylase inhibitor, or ACCi). In May 2022, Hepion Pharmaceuticals, Inc. announced that it has entered into a clinical collaboration with HepQuant, a Denver-based, privately held company with novel, proprietary investigational technology for evaluating liver function and health in patients with chronic liver diseases. Hepion will incorporate the HepQuant 'SHUNT' test into a dedicated Phase IIb clinical trial in presumed NASH F3 subjects. In February 2022, the US Food and Drug Administration (FDA) granted a Fast-Track Designation to Axcella Therapeutics' AXA1125 to treat non-alcoholic steatohepatitis (NASH) with liver fibrosis. In January 2022, LISCure Biosciences announced that it had executed a research collaboration agreement with Mayo Clinic for new drug development for rare liver diseases. LISCure Biosciences Inc. has executed a research collaboration agreement with US based-Mayo Clinic for new drug development for rare liver diseases. Request a sample and discover the nash drugs in development 2022 @ Nonalcoholic Steatohepatitis Pipeline 2022 Report The nonalcoholic steatohepatitis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage nonalcoholic steatohepatitis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the nonalcoholic steatohepatitis clinical trial landscape. Nonalcoholic Steatohepatitis Overview Nonalcoholic steatohepatitis (NASH) is a type of liver inflammation and damage caused by fat buildup. It is one of several conditions known as nonalcoholic fatty liver disease. Nonalcoholic fatty liver disease is classified into two types: isolated fatty liver, in which only fat accumulates, and nonalcoholic steatohepatitis, in which fat, inflammation, and liver cell damage occur. NASH does not usually cause symptoms. Most people with NASH feel fine and are unaware that they have it. As NASH progresses and liver damage worsens, a patient may experience fatigue, weight loss, general weakness, and acne in the upper right part of the belly. Screening for nonalcoholic fatty liver disease in the general population is not advised. It is usually considered after discovering unexplained liver enzyme levels, or imaging reveals hepatic steatosis. A patient's medical history, a physical exam, and tests for nonalcoholic steatohepatitis are used by doctors. Want to discover how the Nash treatment pipeline will look like? Request for sample @ NASH Drug Pipeline 2022 A snapshot of the Nonalcoholic Steatohepatitis Pipeline Drugs mentioned in the report: Want to know how nash dash the pipeline race is for new agents? Download the sample page @ NASH Clinical Trials Nonalcoholic Steatohepatitis Therapeutics Assessment The nonalcoholic steatohepatitis pipeline report proffers an integral view of nonalcoholic steatohepatitis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Nonalcoholic Steatohepatitis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Intravenous, Parenteral, Oral, Subcutaneous Therapeutics Assessment By Molecule Type: Antibody, Biologic, Cell therapy, Peptide, Protein, Small molecule Therapeutics Assessment By Mechanism of Action: Adiponutrin inhibitor, Diacylglycerol O acyltransferase inhibitor, Adiponutrin modulator, RNA interference, Glucagon like peptide 1 receptor agonist, Ornithine decarboxylase stimulant, Farnesoid X-activated receptor agonist, Thyroid hormone receptor beta agonist, Mitochondrial membrane transport protein modulator, Thyroid hormone receptor beta agonists, Peroxisome proliferator-activated receptor alpha agonist Key Nonalcoholic Steatohepatitis Companies:Madrigal Pharmaceuticals, Intercept Pharmaceuticals, Cirius Therapeutics, Novo Nordisk, Galmed Pharmaceuticals, AstraZeneca, Galectin Therapeutics, Viking Therapeutics, Eli Lilly and Company, Terns Pharmaceuticals, Sinew Pharma, Novartis Pharmaceuticals, Poxel SA, AngioLab, Pfizer, 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, 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, and others. Key Nonalcoholic Steatohepatitis Pipeline Therapies: TERN-101, SNP-610, LJN452(tropifexor + licogliflozin), PXL065, ALS-L1023, PF-06865571 + PF-05221304, PF-06835919, PF-06865571, ORMD-0801, Norucholic acid, NNC0194-0499, MN-001, MK-3655, MET642, MET409, LPCN 1144, LIK066, Leronlimab, ALN-HSD, MT-3995, CM101, Leu-Mit-Sil (NS-0200), IN-A010, Efruxifermin (EFX), Efinopegdutide, EDP-305, JKB-122, CS0159 (Linafexor), CRV431, Chiglitazar sodium, BOS-580, BMS-986263, HEC96719, GSK4532990, FM101, Firsocostat, EPY001a, Emricasan, BMS-986036, BIO89-100, BI 456906, BFKB8488A, AXA1125, ASC 41, GS-9674 (Cilofexor), ZSP1601, and others. Dive deep into rich insights for nonalcoholic steatohepatitis research; visit @ NASH Treatment Pipeline Table of Contents For further information on the most promising nash drugs, reach out @ NASH Medication Pipeline Related Reports Nonalcoholic Steatohepatitis Epidemiology Nonalcoholic Steatohepatitis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical, and forecasted nonalcoholic steatohepatitis epidemiology in the 7MM. Nonalcoholic Steatohepatitis Market Nonalcoholic Steatohepatitis Market Insight, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key nonalcoholic steatohepatitis companies, including Boehringer Ingelheim, Oramed Pharmaceuticals, ENYO Pharma, Terns Pharmaceuticals, Cirius Therapeutics, among others. Non-Alcoholic Fatty Liver Disease Market Non-Alcoholic Fatty Liver Disease Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key non-alcoholic fatty liver disease companies including AstraZeneca, Novartis, Pfizer, Roche, among others. Non-Alcoholic Fatty Liver Disease Pipeline Non-Alcoholic Fatty Liver Disease Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key advanced non-alcoholic fatty liver disease companies, including AstraZeneca, Novartis, Pfizer, Roche, among others. Non-Alcoholic Fatty Liver Disease Epidemiology Non-Alcoholic Fatty Liver Disease Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical, and forecasted non-alcoholic fatty liver disease epidemiology in the 7MM. Other Trending Reports Alcohol Use Disorder Market | Liver Cancer Market | Alcoholic Hepatitis - Market | Acute Liver Failure - Market | Liver Angiosarcoma - Market | Liver Cirrhosis - Market | Chronic Liver Disease - Market | Advanced Liver Cancer - Market | Acute On Chronic Liver Failure (ACLF) Market Related Healthcare Services Healthcare Business Consulting Healthcare Competitive Intelligence Services Healthcare Asset Prioritization Services About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. Connect with us at LinkedIn Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research LLP

Phase 2License out/inFast Track

20 Jun 2022

·www.biospace.com

PharmaNest to Present Eight Digital Pathology and AI Abstracts at the International Liver Congress 2022PharmaNest

PRINCETON, N.J., June 20, 2022 (GLOBE NEWSWIRE) --Pharmanest, an emerging leader in quantitative Digital Pathology and Artificial Intelligence, announces today that it will be present, alongside with its collaborators, eight presentations at this year's International Liver Congress (ILC) in London, June 22-25, 2022. The abstracts highlight the utility and performance of FibroNest, the first multi-vendor quantitative digital Pathology image analysis and translational platform for the assessment of the severity and progression of Fibrosis and disease activity in NASH. "The automated quantification of the phenotype of fibrosis severity from the same slides reviewed by pathologists offers a robust and scalable method to generate continuous scores with a detection threshold that can detect faint changes and drug effects," said Mathieu Petitjean, CEO and founder of Pharmanest. Because the approach is fully quantitative and is not based on existing staging or grading paradigms, it can be used along the drug discovery and development process, and across etiologies of fibrosis. The first abstract is a retrospective analysis of liver biopsies from the ARMOR Phase 2 trial of aramchol. It shows how Fibronest's continuous fibrosis score detects drug effects that are missed by conventional histological methods (in partnership with Galmed Pharmaceuticals). A second abstract includes results from a prospective analysis of liver biopsies from the FALCON1 Phase 2 trial of pegbelfermin. It demonstrates the sensitivity of FibroNest's method to detect treatment and dose effects, with a performance that benchmarks imaging-based measurements (in partnership with Bristol Myers Squibb). A third abstract discusses the performance of novel FibroNest computational methods for the continuous histological quantification of Ballooning and Inflammation in liver biopsies and related correlations with NASH-CRN histological grades. Another abstract in collaboration with the University of Oxford uses FibroNest to develop an etiology-independent composite score for the automated histological assessment of fibrosis in liver biopsies. Finally, several abstracts highlight the value of FibroNest to quantify the histological phenotype of fibrosis in the context of in-vitro assays for mid-throughput screening of antifibrotic compounds (in partnership with InSphero AG) and the validation for lead antifibrotic drug candidate in rodent models (in collaboration with Therapeutic Discovery department at Mount Sinai) with a performance that outperforms any alternative methods, when they exist. The specific details of these abstracts are available on PharmaNest website at fibronest.com/science. Media Contact: Dr. Li Chen Principal Investigator Contact Li.chen@pharmanest.com Related Images Image 1: FibroNest Augmented Pathology of a NASH Biopsy FibroNest generates augmented pathology images to visualize and quantify the histologic hallmarks of NASH This content was issued through the press release distribution service at Newswire.com. Attachment FibroNest Augmented Pathology of a NASH Biopsy FibroNest Augmented Pathology of a NASH Biopsy FibroNest Augmented Pathology of a NASH Biopsy

Collaborate

14 Nov 2021

·www.biospace.com

89bio Presents New Data from Phase 1b/2a NASH Study Showing BIO89-100 Reduces Spleen Volume

New sub-analysis presented at The Liver Meeting® 2021 shows treatment with BIO89-100 reduced spleen volume by an average of 11.8% in NASH patients Oral presentation was highlighted by the AASLD Scientific Program Committee as a key presentation in The Liver Meeting® 2021 SAN FRANCISCO, Nov. 14, 2021 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardio-metabolic diseases, today announced the presentation of new data from a post-hoc analysis of the Phase 1b/2a proof-of-concept study evaluating BIO89-100 in patients with nonalcoholic steatohepatitis (NASH) at The Liver Meeting® 2021 of the American Association for the Study of Liver Diseases (AASLD). The sub-analysis, which assessed the correlation between liver fat and spleen volume (SV), demonstrated that treatment with BIO89-100 reduced spleen volume by an average of 11.8% in patients with NASH. These data will be presented virtually on Sunday, November 14, as an oral presentation entitled “Treatment with BIO89-100 Led to Decreased Spleen Volume That was Correlated with Relative Change in Liver Fat Volume and Pro-C3 Level in a Phase 1b/2a, Placebo-controlled, Double-blind, NASH Proof of Concept (POC) Study” during the Parallel 21: NAFLD and NASH: Clinical Trials of Novel Therapeutics session. “Prior to this study, correlations between increased spleen volume and improvements in NASH had not been systematically evaluated,” said Rohit Loomba, MD, MHSc, Director of the NAFLD Research Center and Director of Hepatology at UC San Diego School of Medicine. “These exciting data from the BIO89-100 study demonstrate that improvement in liver fat and liver volume result in decreasing spleen volume likely due to improved portal flow.” The post-hoc analysis of the Phase 1b/2a study assessed the effect of BIO89-100 on SV in NASH patients without advanced fibrosis. SV was evaluated in all eligible patients on BIO89-100 27mg every week dose (n=8), BIO89-100 36mg every two-week dose (n=8) and 16 patients on placebo. These patients were assessed by MRI at baseline, on Day 50 and on Day 92. At baseline, it was observed that SV was correlated with liver volume, vibration-controlled transient elastography (VCTE) score and body mass index (BMI), and negatively correlated with platelet count. Findings at study Day 50 and Day 92 demonstrated that treatment with BIO89-100 led to a progressive and significant decrease in spleen volume compared to placebo (on Day 50, treated patients saw an average of 7.4% decrease in SV and by Day 92 patients saw an average 11.8% decrease in SV). These preliminary observations suggest that increased fat in the liver and inflammation may lead to subclinical worsening of portal blood flow, which in turn leads to increased spleen volume in NASH patients without advanced fibrosis. Based on these study findings, portal flow may be improved with treatment that significantly reduces liver fat. “We are encouraged by the preliminary findings that suggest BIO89-100 has the potential to promote meaningful reductions in spleen volume, which correlate with reductions in liver fat by MRI-PDFF and liver fat volume,” said Hank Mansbach, Chief Medical Officer of 89bio. “These exciting data continue to advance our understanding of fatty liver disease and provide new insights about evaluating treatment response in NASH.” In addition to being presented at the meeting, the abstract has been selected by members of the AASLD Scientific Program Committee as a key presentation in The Best of Liver Meeting 2021. A copy of the oral presentation will be accessible under ‘Scientific Publications’ in the pipeline section of 89bio’s website. About the Phase 1b/2a Study This multicenter, randomized, double-blind, placebo-controlled, multiple ascending dose-ranging trial was designed to assess the safety, tolerability and pharmacokinetic (PK) properties of BIO89-100 as well as change in liver fat measured by MRI-PDFF and key biomarker assessments in patients with biopsy-proven NASH with fibrosis or patients with phenotypical NASH (PNASH). PNASH was defined as patients with steatosis greater than 10% who have central obesity and Type 2 diabetes or central obesity and evidence of liver injury. Both patient populations had similar disease characteristics at baseline. A total of 81 patients were randomized to receive weekly or every-two-week subcutaneous dosing of BIO89-100 or placebo for up to 12 weeks. Results showed robust reductions in liver fat and key liver markers, a strong efficacy profile, and favorable tolerability with weekly and every-two-week dosing. These findings highlight the promising clinical pro BIO89-100 and add to the growing body of evidence suggesting it could be a leading FGF21 analog in a class that could become the backbone of treatment for NASH. About 89bio 89bio is a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardio-metabolic diseases. The company’s lead product candidate, BIO89-100, is a specifically engineered glycoPEGylated analog of FGF21. BIO89-100 is being developed for the treatment of nonalcoholic steatohepatitis (NASH) and severe hypertriglyceridemia (SHTG). 89bio is headquartered in San Francisco with operations in Herzliya, Israel. Forward-looking Statements Certain statements in this press release may constitute "forward-looking statements" within the meaning of the federal securities laws, including, but not limited to, the therapeutic potential and clinical benefits of BIO89-100 with respect to spleen volume and the potential efficacy of BIO89-100 in reducing spleen volume. Words such as "may," "might," "will," "objective," "intend," "should," "could," "can," "would," "expect," "believe," "design," "estimate," "predict," "potential," "develop," "plan" or the negative of these terms, and similar expressions, or statements regarding intent, belief, or current expectations, are forward looking statements. While 89bio believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to us on the date of this release. These forward-looking statements are based upon current estimates and assumptions and are subject to various risks and uncertainties (including, without limitation, those set forth in 89bio's filings with the SEC), many of which are beyond 89bio's control and subject to change. Actual results could be materially different. Risks and uncertainties include: positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies; and other risks and uncertainties identified in 89bio's Annual Report on Form 10-K for the year ended December 31, 2020, its Quarterly Reports on Form 10-Q and other subsequent disclosure documents filed with the SEC. 89bio claims the protection of the Safe Harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. 89bio expressly disclaims any obligation to update or alter any statements whether as a result of new information, future events or otherwise, except as required by law. Investor Contact: Ryan Martins Chief Financial Officer investors@89bio.com Media Contact: Peter Duckler 773-343-3069 pduckler@w2ogroup.com

100 Deals associated with Pegbelfermin(Ambrx, Inc.)

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KEGG	Wiki	ATC	Drug Bank
D11512	-	-	DB15365

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Fibrosis, Liver	Phase 2	US	Bristol Myers Squibb Co.	12 Jun 2018
Fibrosis, Liver	Phase 2	JP	Bristol Myers Squibb Co.	12 Jun 2018
Liver Cirrhosis	Phase 2	US	Bristol Myers Squibb Co.	12 Jun 2018
Liver Cirrhosis	Phase 2	JP	Bristol Myers Squibb Co.	12 Jun 2018
Nonalcoholic Steatohepatitis	Phase 2	US	Bristol Myers Squibb Co.	08 May 2015
Obesity	Phase 2	US	Bristol Myers Squibb Co.	15 Apr 2014
Obesity	Phase 2	CA	Bristol Myers Squibb Co.	15 Apr 2014
Diabetes Mellitus, Type 2	Phase 2	US	Bristol Myers Squibb Co.	15 Apr 2014
Diabetes Mellitus, Type 2	Phase 2	CA	Bristol Myers Squibb Co.	15 Apr 2014

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03486912 (FALCON 2, CTgov)	Phase 2	Fibrosis, Liver \| Nonalcoholic Steatohepatitis \| Liver Cirrhosis	155	Placebo	xgzsaqhhel(ibvcrhlifo) = xedqkiiefl xoukrzmodh (nyhcpolino, ipqacwbmld - yegznsfbau) View more	-	13 Oct 2022
NCT03486899 (FALCON 1, CTgov)	Phase 2	Fibrosis, Liver \| Nonalcoholic Steatohepatitis	197	Placebo	zsgiahxhbg(dirgeyqads) = exltstksnx gagjmzxfji (zfcydmpdnq, bdtsjpypvp - wszdvkfror) View more	-	09 Sep 2022
NCT03400163 (CTgov)	Phase 2	Nonalcoholic Steatohepatitis	3	BMS-986036 (BMS-986036 20 mg QD)	sjbqmohpdj(dnagcymzap) = sddfobqdnu ofykrqpxhl (euvemteuxm, fpxncvizgh - otjoodkycv) View more	-	19 Feb 2020
				Placebo (Placebo 20 mg QD)	sjbqmohpdj(dnagcymzap) = avgxhhipsu ofykrqpxhl (euvemteuxm, bubooxllke - gdoldpjsdc) View more
NCT02413372 (MB130-045, CTgov)	Phase 2	Nonalcoholic Steatohepatitis	184	BMS-986036 (BMS-986036 10 mg QD)	gvepvlzznl(umzbgusbmj) = atocsqjsew bqwufzgwru (clscqyozsh, nnifobhwau - tamggswjbz) View more	-	28 Jan 2020
				BMS-986036 (BMS-986036 20 mg QW)	gvepvlzznl(umzbgusbmj) = jdsqivixbj bqwufzgwru (clscqyozsh, njtzpvcitk - buggiaebmm) View more
NCT02097277 (CTgov)	Phase 2	Diabetes Mellitus, Type 2 \| Obesity	219	BMS-986036 (Treatment C: BMS-986036 (5 mg Daily))	dzwfbxuxdi(nrbdwmkosd) = ougzhuorbt ifptjnszie (tzbcjpxvws, etfgtsgqyz - iroftqiwsq) View more	-	09 May 2019
				BMS-986036 (Treatment D: BMS-986036 (20 mg Daily))	dzwfbxuxdi(nrbdwmkosd) = yrayoekpqt ifptjnszie (tzbcjpxvws, luxwphocdf - euaseovsir) View more
MB130-070 (EASL2019)	Phase 1	Overweight \| Obesity steatosis \| liver injury \| fibrosis	30	pegbelfermin (BMS-986036)	kjnadiwumr(rldsoxoqek) = zhlhipnvqp jrmasujrja (xlbmzjxcmp, 34) View more	Positive	13 Apr 2019
				pegbelfermin (BMS-986036)	kjnadiwumr(rldsoxoqek) = lidecmiexp jrmasujrja (xlbmzjxcmp, 27) View more
NCT02097277 (Pubmed) Manual	Phase 2	Diabetes Mellitus, Type 2 \| Obesity	120	Pegbelfermin	wcrukilxje(gugninqbac) = no significant effects dksalkxbap (xfkcdmjard ) View more	Negative	01 Jan 2019
				Placebo
NCT02413372 (MB130-045, NASH_TAG2018)	Phase 2	Nonalcoholic Steatohepatitis ALT \| AST \| CK-18 ... View more	-	BMS-986036 10 mg QD	ehliiscjxe(dyynnikbdx) = uhhdlcyqcp uxotpbpwzk (aeujfmkjjd ) View more	Positive	04 Jan 2018
Phase 1 Study of BMS-986036 (NASH_TAG2017)	Phase 1	Obesity, Metabolically Benign	96	BMS-986036	rjxvoiehhb(vbhmlbbvhm) = 22.2% ffmwjrakzb (czkabyozao )	Positive	05 Jan 2017

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