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Last update 02 Apr 2026

Daplusiran/Tomligisiran

Last update 02 Apr 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

siRNA

Synonyms

DAP/TOM, ARO-HBV, GSK-5637608

+ [4]

Target

cccDNA

Action

inhibitors

Mechanism

cccDNA inhibitors(covalently closed circular DNA inhibitors), RNA interference

Therapeutic Areas

Infectious DiseasesDigestive System Disorders

Active Indication

Hepatitis B, Chronic

Inactive Indication

Hepatitis D, Chronic

Originator Organization

Arrowhead Pharmaceuticals, Inc.

Active Organization

Janssen Research & Development LLC

GSK Plc

Arrowhead Pharmaceuticals, Inc.

Inactive Organization

Johnson & Johnson (China) Investment Ltd.Janssen Sciences Ireland Unlimited Co.

Xian-Janssen Pharmaceutical Ltd.

License Organization

Janssen Pharmaceuticals, Inc.

Alnylam Pharmaceuticals, Inc.

GSK Plc

+ [1]

Drug Highest PhasePhase 2

First Approval Date-

Regulation-

Structure/Sequence

Boost your research with our RNA technology data.

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Sequence Code 316576694

The sequence is quoted from: *****

Sequence Code 316576738

The sequence is quoted from: *****

Clinical Trials associated with Daplusiran/Tomligisiran

NCT06537414

/ Active, not recruitingPhase 2

A Phase 2b, Multi-centre, Randomized, Partially Placebo-controlled, Double-blind Study to Investigate the Safety and Efficacy of Sequential Therapy With Daplusiran/Tomligisiran Followed by Bepirovirsen in Participants With Chronic Hepatitis B Virus on Background Nucleos(t)Ide Analogue Therapy (B-United)

The study is intended to evaluate the efficacy and safety of 2 different doses of DAP/TOM followed by bepirovirsen in participants living with CHB on standard of care nucleos(t)ide analogue (NA) therapy. The study also aims to identify an optimal dose of DAP/TOM for sequenced therapy with bepirovirsen for further clinical development and to assess the contribution of DAP/TOM to the sequential regimen.

Start Date11 Nov 2024

Sponsor / Collaborator

GSK Plc

NCT05275023

/ CompletedPhase 2

A Phase 2 Open-label Trial to Evaluate Safety, Efficacy, Tolerability, and Pharmacodynamics of a Combination of JNJ-73763989, Nucleos(t)Ide Analogs, and a PD-1 Inhibitor in Chronic Hepatitis B Patients

The purpose of this study is to evaluate efficacy of the study intervention, based on hepatitis B surface antigen (HBsAg) levels at follow-up (FU) Week 24.

Start Date30 Jun 2022

Sponsor / Collaborator

Janssen Research & Development LLC

NCT05123599

/ CompletedPhase 1

A Phase 1b, Open-label, Single-arm, Multicenter Study to Assess Efficacy, Safety, and Tolerability of Treatment With JNJ-73763989, JNJ-64300535, and Nucleos(t)Ide Analogs in Virologically Suppressed, HBeAg-negative Participants With Chronic Hepatitis B Virus Infection

The purpose of this study is to evaluate the efficacy of the study intervention based on hepatitis B surface antigen (HBsAg) levels.

Start Date06 Dec 2021

Sponsor / Collaborator

Janssen Research & Development LLC

100 Clinical Results associated with Daplusiran/Tomligisiran

100 Translational Medicine associated with Daplusiran/Tomligisiran

100 Patents (Medical) associated with Daplusiran/Tomligisiran

Literatures (Medical) associated with Daplusiran/Tomligisiran

01 Dec 2025·JHEP Reports

Corrigendum to “Peginterferon-alpha-2a add-on to treatment with siRNA JNJ-73763989 in virologically suppressed chronic hepatitis B: The phase II PENGUIN study” [JHEP Reports 2025 7(10) 101516]

Author: Kim, Gloria ; Hlebowicz, Maria ; Jezorwski, John ; Chang, Ting-Tsung ; Chuang, Wan-Long ; Bakala, Adam ; Gane, Edward ; Peng, Cheng-Yuan ; Pehlivanov, Nonko ; Biermer, Michael ; Lenz, Oliver ; Asahina, Yasuhiro ; Takehara, Tetsuo ; Verbinnen, Thierry ; Anastasiou, Zacharias ; Kalmeijer, Ronald ; Janczewska, Ewa ; Kakuda, Thomas N

[This corrects the article DOI: 10.1016/j.jhepr.2025.101516.].

01 Dec 2025·JHEP Reports

Viral sequence analysis of chronic hepatitis B patients treated with the siRNA JNJ-73763989 in phase II clinical trials

Article

Author: De Meyer, Sandra ; Augustyns, Ilse ; Jezorwski, John ; Grant, Craig ; Verbinnen, Thierry ; Yuen, Man-Fung ; Lenz, Oliver ; Agarwal, Kosh ; Lathouwers, Erkki ; Biermer, Michael

Background & Aims:

JNJ-73763989 (JNJ-3989) is a small-interfering RNA composed of two triggers targeting the HBsAg and HBx protein open reading frame, designed to target all HBV RNAs for degradation. JNJ-3989 + nucleos(t)ide analogue (NA) treatment dose-dependently reduced chronic hepatitis B (CHB) viral antigens. Viral sequence changes in the JNJ-3989 S-/X-trigger target regions were evaluated at baseline, on-treatment, and in patients with virologic relapse (VR) after discontinuation of all treatment in REEF-1 (NCT03982186) and REEF-2 (NCT0412954) studies.

Methods:

HBV DNA/RNA was extracted from plasma samples, and the HBV genome was sequenced using next-generation sequencing. Nucleotide variants were defined as changes vs. the universal HBV reference sequence (read frequency >15%).

Results:

Baseline polymorphisms in the JNJ-3989 target region complementary to positions 2-18 of the S-/X-trigger were present in 10.1% and 2.4% of not currently treated patients, respectively, with no relevant impact on JNJ-3989-induced HBsAg decline. Variants at X-trigger target region positions of interest (POI) were more frequently observed off treatment in JNJ-3989-treated virologically suppressed (VS) patients with VR vs. NA-control arm VS patients with VR (55.8% vs. 5.7%, respectively). Variants at S-trigger POI were observed off treatment in 32.1% and 19.4% of JNJ-3989- and NA-control treated VS patients with VR, respectively. Off-treatment HBsAg kinetics did not differ between JNJ-3989-treated patients with and without variants in S-/X-trigger target POI during VR.

Conclusion:

Baseline sequence polymorphisms did not impact JNJ-3989 treatment response. JNJ-3989-treated patients who experienced VR post-treatment had variants in the X-trigger target region during VR, but not at baseline or on-treatment, suggesting that X-trigger variants developed off treatment in JNJ-3989-treated patients. The presence of these variants did not impact off-treatment HBsAg kinetics.

ClinicalTrialgov Identifiers:

NCT03982186 and NCT0412954.

Impact and implications:

Small-interfering RNA (siRNA) therapy with JNJ-3989 in patients with chronic hepatitis B induces potent, dose-dependent reductions in viral antigens, though the magnitude of decline varies among individuals. Baseline nucleotide polymorphisms in JNJ-3989 trigger target regions did not account for variability in HBsAg or HBeAg responses. Substitutions in siRNA trigger target regions were frequently detected during and after virologic relapse in JNJ-3989-treated patients, confirming antiviral target engagement. However, the emergence of these variants did not affect off-treatment HBsAg or HBV DNA kinetics. This study provides the first comprehensive clinical virology analysis of siRNA-based therapy in HBV infection, offering insights relevant to the broader development of antiviral siRNA therapeutics. The clinical significance of X-trigger substitutions for potential re-treatment with JNJ-3989 or other HBV-targeting siRNAs remains to be determined.ClinicalTrial.gov Identifiers: NCT03982186 and NCT0412954.

01 Oct 2025·JHEP Reports

Peginterferon-alpha-2a add-on to treatment with siRNA JNJ-73763989 in virologically suppressed chronic hepatitis B: The phase II PENGUIN study

Article

Author: Kim, Gloria ; Hlebowicz, Maria ; Jezorwski, John ; Chang, Ting-Tsung ; Bakala, Adam ; Chuang, Wan-Long ; Gane, Edward ; Peng, Cheng-Yuan ; Pehlivanov, Nonko ; Biermer, Michael ; Lenz, Oliver ; Takehara, Tetsuo ; Asahina, Yasuhiro ; Anastasiou, Zacharias ; Verbinnen, Thierry ; Kalmeijer, Ronald ; Janczewska, Ewa ; Kakuda, Thomas N

Background & Aims:

Previous studies showed that combination treatment with short interfering RNA JNJ-73763989 (JNJ-3989) ± capsid assembly modulator bersacapavir (JNJ-56136379) and nucleos(t)ide analogs (NAs) was well tolerated by patients with chronic HBV (CHB), with JNJ-3989 dose-dependent reductions in viral markers, including HBsAg. The open-label, single-arm phase IIa PENGUIN study (NCT04667104) evaluated this regimen plus pegylated interferon alpha-2a (PegIFN-α2a) in patients with virologically suppressed CHB.

Methods:

Patients who were either HBeAg-positive or -negative virologically suppressed and taking NAs were included; all received JNJ-3989 ± bersacapavir for 24 weeks (some either did not start or discontinued bersacapavir as a result of protocol amendment) with PegIFN-α2a added during the final 12 weeks of treatment. The primary endpoint was the proportion of patients with ≥2 log₁₀ IU/ml HBsAg reduction from baseline at week 24 (W24). Safety, tolerability, and proportion of patients meeting predefined NA stopping criteria were assessed. After 24 weeks of treatment, NA was stopped when stopping criteria were met; all patients were followed for 48 weeks. Efficacy and safety endpoints were summarized using descriptive statistics.

Results:

In total, 48 patients (HBeAg positive, n = 11; HBeAg negative, n = 37) were enrolled; 47 reached follow-up week 48 (FUW48). Of the 48 patients, 31 (64.6%) achieved the primary endpoint; one achieved HBsAg seroclearance (<0.05 IU/ml) at both W24 and FUW48 after stopping NA. Mean HBsAg changes from baseline were -1.43, -2.18, and -0.71 log₁₀IU/ml at W12, W24, and FUW48, respectively. At W24, 15/48 (31.3%) patients met NA stopping criteria; 11/15 (73.3%) remained off NA by FUW48. One patient achieved functional cure at FUW48. Adverse events were consistent with known PegIFN-α2a safety profiles.

Conclusions:

Treatment with JNJ-3989 ± bersacapavir + NA and PegIFN-α2a was generally well tolerated. Most patients had pronounced HBsAg reductions from baseline, no additional benefit from PegIFN-α2a, and did not achieve functional cure.

Clinical Trials registration:

The study is registered at ClinicalTrials.gov (NCT04667104).

Impact and implications:

Previous studies with JNJ-73763989 (JNJ-3989) ± bersacapavir (JNJ-56136379) and nucleos(t)ide analogs (NAs) in patients with chronic hepatitis B (CHB) have demonstrated JNJ-3989 dose-dependent and robust reductions in viral markers; however, functional cure was only rarely achieved. The current study assessed whether the addition of pegylated interferon alpha-2a (PegIFN-α2a) to a JNJ-3989-based regimen after HBsAg levels were reduced would lead to further HBsAg declines and improved outcomes during off-treatment follow-up. Study results confirmed pronounced HBsAg reductions from baseline in virologically suppressed patients with HBeAg-positive or -negative CHB. However, the addition of PegIFN-α2a to JNJ-3989 ± bersacapavir and NA for the final 12 weeks of a 24-week treatment period did not appear to improve antiviral activity in the studied population. Studies in other CHB groups, including treatment-naïve, HBeAg-positive patients, could help elucidate whether this or other treatment regimens including JNJ-3989 and NA can lead to functional cure in a larger proportion of patients.

News (Medical) associated with Daplusiran/Tomligisiran

17 Jun 2025

·www.prnewswire.com

Chronic Hepatitis B Market--The US to Have the Lion's Share Among the 7MM, Predicts DelveInsight

The chronic hepatitis B market size is predicted to surge owing to the increasing prevalence of the disease globally and rising awareness about early diagnosis and treatment. Advances in antiviral therapies and the development of novel treatment options are fueling market expansion. LAS VEGAS, June 17, 2025 /PRNewswire/ -- Hepatitis B is the most widespread serious liver infection globally, caused by the hepatitis B virus (HBV), which targets and damages the liver. Millions of individuals worldwide are affected by chronic hepatitis B. The virus spreads through contact with infected blood and bodily fluids, including transmission via unprotected sex, sharing of contaminated needles, use of illegal drugs, and exposure to unsterilized medical equipment. In most cases, hepatitis B resolves on its own within 1 to 2 months. However, if the infection persists beyond six months, it can become chronic, potentially leading to ongoing liver inflammation, cirrhosis (liver scarring), liver cancer, or liver failure. As of 2024, around 5 million people across the 7MM were living with chronic hepatitis B. Among these, 85% had compensated liver function, while the remaining 15% had progressed to decompensated liver disease. Currently, there is no definitive cure for hepatitis B. Antiviral medications and interferon injections can help manage the disease by reducing viral activity and easing symptoms, but they do not eliminate the virus entirely. If the infection persists beyond six months, it is classified as chronic, making patients eligible for drug therapy, particularly those with active liver disease. Learn more about the chronic hepatitis B virus market @ New Treatment for Chronic Hepatitis B The US FDA has approved seven medications for treatment: two injectable interferons and five oral antiviral drugs. These treatments must be taken daily and work by suppressing viral replication, thereby reducing liver inflammation and potential damage. VEMLIDY (tenofovir alafenamide), developed by Gilead Sciences, is an innovative, targeted prodrug of tenofovir formulated as an oral tablet. In March 2024, Gilead announced that the FDA had approved a supplemental New Drug Application (sNDA) for VEMLIDY 25 mg, allowing its once-daily use in pediatric patients aged 6 years and older and weighing at least 25 kg who have chronic hepatitis B (CHB) infection with compensated liver disease. Earlier, in November 2022, the FDA approved the same 25 mg dose for once-daily use in children aged 12 and above with CHB and compensated liver disease. The initial FDA approval came in November 2016 for adult patients with CHB and compensated liver function. In January 2017, Gilead announced that the European Commission (EC) had granted marketing authorization for VEMLIDY 25 mg to treat CHB in adults and adolescents aged 12 and older who weigh at least 35 kg. Additionally, in December 2016, Japan's Ministry of Health, Labour and Welfare (MHLW) approved VEMLIDY for the treatment of chronic hepatitis B in patients showing active viral replication and abnormal liver function. Find out more on FDA-approved chronic hepatitis B drugs @ Chronic Hepatitis B (CHB) Market The chronic hepatitis B treatment drug market is continuously evolving. Several pharma companies are evaluating their lead assets in different phases of development. Many potential therapies are being investigated to manage chronic hepatitis B. Some of the drugs in the pipeline include Imdusiran (AB-729) (Arbutus Biopharma), GSK3228836 (bepirovirsen) (GlaxoSmithKline), Tobevibart (VIR-3434) + elebsiran (VIR-2218) ± PEG-IFN-a (Vir Biotechnology), and others. Even though it is too soon to comment on the above-mentioned promising candidate to enter the market during the forecast period (2025–2034), it is safe to assume that the future of this market is bright. Discover which therapies are expected to grab major chronic hepatitis B treatment drug market @ Chronic Hepatitis B Market Report Daplusiran (also known as tomligisiran, formerly JNJ-3989/GSK5637608) is an experimental siRNA-based therapy targeting hepatitis B virus (HBV). It is being studied in combination with bepirovirsen in a sequential regimen for adult patients with chronic hepatitis B who are non-cirrhotic and receiving nucleos(t)ide analogue (NA) therapy. In October 2023, GSK and Arrowhead Pharmaceuticals announced an agreement with Janssen Pharmaceuticals to acquire the exclusive global rights to further develop and commercialize JNJ-3989. Janssen had originally licensed the drug (then called ARO-HBV) from Arrowhead in 2018. Imdusiran is an RNA interference (RNAi) therapy designed to suppress all HBV viral proteins and antigens, including HBsAg, which is believed to be critical for restoring the immune system's ability to fight the virus. It uses Arbutus' proprietary GalNAc-conjugated delivery platform to specifically target liver cells (hepatocytes), allowing for subcutaneous administration. Arbutus plans to launch a Phase IIb clinical trial in the first half of 2025 to evaluate imdusiran in combination with interferon (IFN) and NA therapy. Discover more about drugs for chronic hepatitis B in development @ Chronic Hepatitis B Clinical Trials The anticipated launch of these emerging therapies for chronic hepatitis B are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the chronic hepatitis B market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for chronic hepatitis B is expected to grow at a significant CAGR by 2034. In 2024, the United States dominated the CHB market among the 7MM, capturing approximately 72% of the total market share. The market size of chronic hepatitis B is expected to grow due to several factors, including rising global prevalence of hepatitis B virus infection and increasing awareness about the disease. Advances in antiviral therapies and growing screening initiatives also boost market growth by enabling early diagnosis and improved disease management. DelveInsight's latest published market report, titled as Chronic Hepatitis B Market Insight, Epidemiology, and Market Forecast – 2034 , will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the chronic hepatitis B country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The chronic hepatitis B market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Chronic Hepatitis B Total Diagnosed Cases of Chronic Hepatitis B Chronic Hepatitis B Cases by Age Group Chronic Hepatitis B Cases by Gender Treated Cases of Chronic Hepatitis B Chronic Hepatitis B Cases by Impact on Liver The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM chronic hepatitis B market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this chronic hepatitis B market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the chronic hepatitis B market. Also, stay abreast of the mitigating factors to improve your market position in the chronic hepatitis B therapeutic space. Related Reports Chronic Hepatitis B Epidemiology Chronic Hepatitis B Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and the CHB epidemiology trends. Chronic Hepatitis B Pipeline Chronic Hepatitis B Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key CHB companies, including Vedanta Biosciences, Gilead Sciences, Dong-A ST Co, Assembly Biosciences, Arbutus Biopharma, Vir Biotechnology, Antios Therapeutics, Ascletis Pharmaceuticals, Shanghai HEP Pharmaceutical, Romark Laboratories, Qilu Pharmaceutical, Golden Biotechnology, Sunshine Lake Pharma, Ascentage Pharma, GlaxoSmithKline, Janssen Sciences, Henlix, Enyo Pharma, Tasly Tianjin Biopharmaceutical, Brii Biosciences, Vaxine Pty Ltd, Vaccitech Limited, Zhejiang Palo Alto Pharmaceuticals, Suzhou Ribo Life Science, PharmaEssentia, Nucorion Pharmaceuticals, Jiangsu HengRui Medicine, Enanta Pharmaceuticals, Chong Kun Dang Pharmaceutical, Guangzhou Lupeng Pharmaceutical, VenatoRx Pharmaceuticals, Zhimeng Biopharm, among others. Hepatitis A Market Hepatitis A Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hepatitis A companies, including Boryung Pharmaceutical, Cadila Healthcare, Indian Immunologicals, Biological E Limited, among others. Hepatitis C Market Hepatitis C Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hepatitis C companies, including AbbVie, Gilead Sciences, Atea 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

Drug ApprovalLicense out/inPhase 2

05 Feb 2025

·www.fiercebiotech.com

GSK drops one potential \'functional cure\' for hep B as it goes all-in on bepirovirsen

Hopes for GSK3528869 appeared to be dashed in December when GSK terminated a phase 1/2 chronic hepatitis B study.\n GSK is prioritizing development of its hepatitis B treatment bepirovirsen as the pharma ends work on another potential functional cure for the infectious disease.On a media call Wednesday morning to discuss her company\'s fourth-quarter and full-year earnings, CEO Emma Walmsley said that when it came to the British pharma’s infectious disease pipeline, “we\'re prioritizing development of bepirovirsen, our potential functional cure for hepatitis B.”The pharma licensed the antisense oligonucleotide from Ionis Pharmaceuticals for $25 million upfront in 2019 and is currently aiming for an approval decision by regulators for chronic hepatitis B next year. The reemphasis on bepirovirsen came as GSK disclosed this morning that it has discontinued work on GSK3528869. The company had been holding out hopes that the candidate could be another functional cure for the infection—meaning it reduces hepatitis B virus DNA and viral proteins to undetectable levels. This stands in contrast to currently available oral antiviral therapies, which only suppress the virus.But those hopes for GSK3528869 appeared to be dashed in December when GSK terminated a phase 1/2 chronic hepatitis B study after the therapeutic vaccine regimen failed to meet the efficacy endpoint. The study tested different dose levels and sequences—and included placebo—but the idea was to prime the immune system with a viral vector encoding hepatitis B protein antigens, boost the immune system with a different vaccine and administer adjuvants to enhance the response.At the time, the pharma still had another study of GSK3528869 listed as active on the federal trial database. That study was evaluating the effect of giving vaccines to prime and boost the immune system—and adjuvants to enhance the response—after patients have received GSK’s bepirovirsen. But GSK confirmed in its earnings documents (PDF) today that it won’t be taking GSK3528869 forward.GSK isn’t the only pharma to have struggled with hepatitis B candidates in the clinic. Roche axed phase 1 and 2 trials in a recent pipeline cull, while Altimmune dropped a drug candidate after phase 2 data disappointed, and Ascletis Pharma stopped a hepatitis B program after the asset flopped in another indication.While bepirovirsen is taking priority, GSK still has a number of other assets in clinical development for chronic hepatitis B, including the hepatitis B virus-targeted siRNA GSK5637608, the PAPD5/PAPD7 inhibitor GSK3965193 and the TLR8 agonist GSK5251738. GSK also disclosed this morning that it had removed a capsid protein inhibitor for HIV called VH4004280 from its phase 2 pipeline. The company still has another HIV capsid protein inhibitor in multiple clinical trials in the form of VH3739937.The final drug to be pulled is the phase 1-stage GSK2556286, which is designed to inhibit the intracellular growth of Mycobacterium tuberculosis. The pharma has other tuberculosis candidates in the clinic in the form of the leucyl t-RNA synthetase inhibitor ganfeborole, the ethionamide booster alpibectir and the serine beta lactamase inhibitor sanfetrinem cilexetil. When it came to M&A, Walmsley told journalists on the media call that the company “continues to add new opportunities through targeted business development.” She pointed to the acquisition of Boston-based cancer-focused biotech IDRx last month as “a good example of what we want to do here.”

$GSK drops one potential \'functional cure\' for hep B as it goes all-in on bepirovirsen$

VaccinePhase 2AcquisitionOligonucleotidesiRNA

29 Nov 2023

·www.biospace.com

Arrowhead Pharmaceuticals Reports Fiscal 2023 Year End Results

- Conference Call and Webcast Today, November 29, 2023, at 4:30 p.m. ET PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals, Inc., (NASDAQ: ARWR) today announced financial results for its fiscal year ended September 30, 2023. The company is hosting a conference call today, November 29, 2023, at 4:30 p.m. ET to discuss the results. Webcast and Conference Call and Details Investors may access a live audio webcast on the Company's website at . A replay of the webcast will be available approximately two hours after the conclusion of the call. For analysts that wish to participate in the conference call, please register at . Once registered, you will receive the dial-in number and a personalized PIN code that will be required to access the call. Selected Recent Events Presented new Phase 2 clinical data from the ongoing SHASTA-2 and MUIR studies of plozasiran (ARO-APOC3) and the ARCHES-2 study of zodasiran (ARO-ANG3) at the American Heart Association (AHA) Scientific Sessions. Key updates included: Plozasiran achieved mean max reductions of up to 90% in APOC3, 87% in triglycerides, and 63% in remnant cholesterol in patients with severe hypertriglyceridemia. In patients with mixed dyslipidemia, plozasiran achieved a mean max reduction in APOC3 of up to 89%, leading to robust and durable improvements in multiple atherogenic lipoproteins Zodasiran treatment resulted in substantial reductions of ANGPTL3, triglycerides, and LDL-cholesterol in patients with mixed dyslipidemia Plozasiran and zodasiran continue to show favorable safety pro Treatment emergent adverse events reported to date reflect the comorbidities and underlying conditions of the study populations Hosted a webinar featuring experts in the treatment and management of lipid and lipoprotein disorders to discuss plozasiran Daniel Gaudet, MD, PhD, Professor of Medicine at Université de Montréal, discussed plozasiran in the context of the current treatment landscape for severe hypertriglyceridemia Børge Nordestgaard, MD, Professor & Chief Physician, Copenhagen University Hospital, University of Copenhagen, Denmark, discussed the emergent role of remnant cholesterol in cardiovascular disease Steven Nissen, MD, Chief Academic Officer for the Heart and Vascular Institute at the Cleveland Clinic, discussed why the decrease in atherogenic lipoproteins observed with plozasiran has the potential to prevent cardiovascular outcomes Completed chronic GLP toxicology for pulmonary candidates ARO-RAGE, designed to reduce expression of the receptor for advanced glycation end products as a potential treatment for inflammatory pulmonary diseases, and ARO-MMP7, designed to reduce expression of matrix metalloproteinase 7 as a potential treatment for idiopathic pulmonary fibrosis Results were highly encouraging and suggest sufficient safety margins to proceed to Phase 2 studies Presented data on the company’s pipeline of pulmonary targeted RNAi therapeutic candidates, including clinical results for ARO-RAGE, at the European Respiratory Society (ERS) International Congress 2023. Key results included: Late-breaking clinical data for ARO-RAGE demonstrated deep and durable gene silencing in healthy volunteers and in patients with asthma Promising preclinical data across multiple pulmonary programs suggested broad potential applications for Arrowhead’s TRiM™ platform Filed for regulatory clearance to initiate a Phase 1/2a study of Arrowhead’s second RNAi therapeutic candidate targeting skeletal muscle, ARO-DM1, for patients with type 1 myotonic dystrophy Announced that GSK plc reached an agreement with Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company, to transfer exclusive worldwide rights to further develop and commercialize JNJ-3989. JNJ-3989 (formerly ARO-HBV) was initially in-licensed by Janssen from Arrowhead in 2018 Selected Fiscal 2023 Year End Financial Results ARROWHEAD PHARMACEUTICALS, INC. CONSOLIDATED CONDENSED FINANCIAL INFORMATION (in thousands, except per share amounts) OPERATING SUMMARY Year Ended September 30, 2023 2022 Revenue $ 240,735 $ 243,231 Operating Expenses: Research and development 353,188 297,307 General and administrative expenses 92,549 124,431 Total Operating Expenses 445,737 421,738 Operating loss (205,002 ) (178,507 ) Interest income 15,299 5,033 Interest expense (18,326 ) - Other, net 1,538 765 Loss before income tax expense and noncontrolling interest (206,491 ) (172,709 ) Income tax expense 2,784 3,785 Net loss including noncontrolling interest (209,275 ) (176,494 ) Net loss attributable to noncontrolling interest, net of tax (4,000 ) (431 ) Net loss attributable to Arrowhead Pharmaceuticals, Inc. $ (205,275 ) $ (176,063 ) Net loss per share attributable to Arrowhead Pharmaceuticals, Inc. - Diluted $ (1.92 ) $ (1.67 ) Weighted-average shares used in calculating - Diluted 106,750 105,426 FINANCIAL POSITION SUMMARY September 30, 2023 2022 Cash, cash equivalents and restricted cash $ 110,891 $ 108,005 Investments 292,735 374,263 Total cash resources (cash and investments) 403,626 482,268 Other assets 361,926 209,671 Total Assets $ 765,552 $ 691,939 Current deferred revenue $ 866 $ 74,099 Long-term deferred revenue - 55,950 Other liabilities 477,524 143,551 Total Liabilities $ 478,390 $ 273,600 Total Arrowhead Pharmaceuticals, Inc. Stockholders' Equity $ 271,343 $ 398,520 Noncontrolling Interest 15,819 19,819 Total Noncontrolling Interest and Stockholders' Equity $ 287,162 $ 418,339 Total Liabilities, Noncontrolling Interest and Stockholders' Equity $ 765,552 $ 691,939 Shares Outstanding 107,312 105,960 About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit , or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit . Safe Harbor Statement under the Private Securities Litigation Reform Act: This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the impact of the ongoing COVID-19 pandemic on our business, the safety and efficacy of our product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc.

Financial StatementClinical ResultAHA

100 Deals associated with Daplusiran/Tomligisiran

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Hepatitis D, Chronic	Phase 2	United States	Janssen Research & Development LLC	28 Sep 2020
Hepatitis D, Chronic	Phase 2	United States	Johnson & Johnson (China) Investment Ltd.	28 Sep 2020
Hepatitis D, Chronic	Phase 2	Japan	Janssen Research & Development LLC	28 Sep 2020
Hepatitis D, Chronic	Phase 2	Japan	Johnson & Johnson (China) Investment Ltd.	28 Sep 2020
Hepatitis D, Chronic	Phase 2	Australia	Johnson & Johnson (China) Investment Ltd.	28 Sep 2020
Hepatitis D, Chronic	Phase 2	Australia	Janssen Research & Development LLC	28 Sep 2020
Hepatitis D, Chronic	Phase 2	Brazil	Janssen Research & Development LLC	28 Sep 2020
Hepatitis D, Chronic	Phase 2	Brazil	Johnson & Johnson (China) Investment Ltd.	28 Sep 2020
Hepatitis D, Chronic	Phase 2	France	Janssen Research & Development LLC	28 Sep 2020
Hepatitis D, Chronic	Phase 2	France	Johnson & Johnson (China) Investment Ltd.	28 Sep 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03982186 (REEF-1, Pubmed \| JHEP Rep)	Phase 2	Hepatitis B, Chronic HBsAg \| HBx protein open reading frame \| HBV RNAs	479	JNJ-3989 + nucleos(t)ide analogue (NA)	jhgjcfeecp(ghsdknnqdu) = duyroziyah gjbhlaiybq (imfgpmiass ) View more	Positive	01 Dec 2025
NCT04439539 (REEF-IT, Pubmed \| Gut)	Phase 2	Hepatitis B, Chronic	54	JNJ-3989+NA±bersacapavir+PegIFN-α2a	smlyipepkh(vkajelkjvb) = owqarsarcu dbjkfhqrvn (pjeounupke ) View more	Positive	04 Nov 2025
NCT04667104 (PENGUIN, Pubmed \| JHEP Rep)	Phase 2	Hepatitis B, Chronic	48	JNJ-3989 ± bersacapavir + NA + PegIFN-α2a (HBeAg-positive + HBeAg-negative)	cbztsxgksv(ouxnkpsfhe) = dlhudqephe vmbtzowaom (gfqrjencdt )	Negative	01 Oct 2025
NCT05275023 (OCTOPUS-1, CTgov)	Phase 2	Hepatitis B, Chronic	37	JNJ-73763989 (JNJ-3989)+Nivolumab (JNJ-73763989 (JNJ-3989) + Nivolumab (1 Infusion) + NA)	sskcusolsx = ieuexbgpwa efmthmruco (qhorjuhvcy, xfcyfcisyb - mrasxtxirj) View more	-	10 Feb 2025
				Nivolumab+JNJ-3989 (JNJ-3989 + Nivolumab (3 Infusions) + NA)	sskcusolsx = hjwmpmedxm efmthmruco (qhorjuhvcy, agnenmlkqx - xzttpuvdvh) View more
NCT04129554 (REEF-2, CTgov)	Phase 2	Hepatitis B, Chronic	130	JNJ-73763989+JNJ-56136379 (Arm 1: JNJ-73763989 (200 Milligrams [mg])+JNJ-56136379 (250 mg)+NA)	efkupvnhzn = atgzencxcn ikwvoayrgh (nhkuxuhjdc, vayakjrcjs - ymacpgrajf) View more	-	31 Jul 2024
				Nucleos(t)Ide Analog (Arm 2: Nucleos(t)Ide Analog (NA))	efkupvnhzn = vrespzzbwb ikwvoayrgh (nhkuxuhjdc, pghbchwbsf - iweqllamtu) View more
NCT04667104 (PENGUIN, CTgov)	Phase 2	Hepatitis B, Chronic	48	JNJ-3989+PegIFN-alpha2a+JNJ-6379 (TP 2:JNJ-3989 200 mg+JNJ-6379 250 mg+PegIFN-alpha2a 180 mcg+NA)	mochsgglku = mlekimlgdj ktqvxfobdp (olhckzefmj, xktjxwozet - zkcwinbesa) View more	-	03 Jul 2024
				JNJ-73763989+JNJ-56136379 (TP 1:JNJ-73763989 200 mg+JNJ-56136379 250 mg+NA)	qblylhnnfn = kpoubmwhqi dzjlkdhsuk (phdsrlcorc, veuxctvfiy - ajogxyznyk) View more
NCT04129554 (REEF-2, Pubmed) Manual	Phase 2	Hepatitis B, Chronic Hepatitis B e-antigen (HBeAg)-negative \| HBsAg	130	JNJ-73763989-6JNJ-56136379	lurfvoafti(ircbvttqko) = keyzegwlup cnlroffxiz (fufpzvjttg ) View more	Negative	05 Apr 2024
				Placebos for JNJ-3989 and JNJ-6379 + active NA	lurfvoafti(ircbvttqko) = sjhygfobda cnlroffxiz (fufpzvjttg ) View more
NCT05005507 (PENGUIN-2, CTgov)	Phase 2	Hepatitis B, Chronic	1	Nucleos(t)Ide Analog (NA)+JNJ-73763989+PegIFN-alpha-2a	ljkyxnaojo = fhbvhidafy gqijgzgmyb (hbowseuspq, xlaxckyeka - xovzcihxeq) View more	-	06 Mar 2024
NCT04585789 (INSIGHT, CTgov)	Phase 2	Hepatitis B, Chronic	24	NA+TAF+JNJ-3989+PegIFN-alpha-2a+JNJ-6379+ETV (Panel 1)	kaojbycrvf(ruubccccyq) = uvbyhowoeh ftcljjxihl (tmpkzbotxk, kffrtmrilo - kwirwncbld) View more	-	05 Mar 2024
				PegIN-alpha2a+TAF+JNJ-3989+PegIFN-alpha-2a+JNJ-6379+ETV (Panel 2)	kaojbycrvf(ruubccccyq) = ifkbkqbiii ftcljjxihl (tmpkzbotxk, bztirjuefj - nlrmtkzser) View more
NCT03982186 (REEF-1, Pubmed) Manual	Phase 2	Hepatitis B, Chronic First line	470	JNJ-6379+JNJ-3989 (JNJ-3989 dual 40 mg group)	izvagwcfrh(rdbrimivta) = mgwmdpxuwy fygwemhrbv (bxpdcswamr, 2 - 11)	Positive	10 Jul 2023
				JNJ-6379+JNJ-3989 (JNJ-3989 dual 100 mg group)	izvagwcfrh(rdbrimivta) = bznnqafdcm fygwemhrbv (bxpdcswamr, 10 - 24)

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