Last update 01 Jul 2024

Belimumab

Last update 01 Jul 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Monoclonal antibody

Synonyms

Anti-BLyS monoclonal antibody, Belimumab (Genetical Recombination), Belimumab (genetical recombination) (JAN)

+ [12]

Target

BAFF

Mechanism

BAFF inhibitors(B-cell-activating factor/B lymphocyte stimulator inhibitors)

Therapeutic Areas

Immune System DiseasesSkin and Musculoskeletal DiseasesUrogenital Diseases+ [5]

Active Indication

Lupus NephritisSystemic Lupus ErythematosusInterstitial lung disease due to systemic disease+ [9]

Inactive Indication

Allograft RejectionEmphysemaGranulomatosis With Polyangiitis+ [9]

Originator Organization

GSK Plc

Active Organization

GSK PlcGlaxoSmithKline LLC

Pharmaceutical Product Development LLC

+ [2]

Inactive Organization

Human Genome Sciences, Inc.

Glaxo Group Ltd.

Hospira, Inc.

+ [1]

Drug Highest PhaseApproved

First Approval Date

US (09 Mar 2011),

Systemic Lupus Erythematosus

RegulationBreakthrough Therapy (US), Orphan Drug (US)

Gene Sequence

Sequence Code 192048L

Source: *****

Sequence Code 192132H

Source: *****

137

Clinical Trials associated with Belimumab

NCT06411249 / RecruitingPhase 4

A Phase 4, Multicenter, Prospective, Open-Label Study Describing the Efficacy and Safety of Belimumab Administered Subcutaneously in Adult Participants With Early Systemic Lupus Erythematosus

This is a prospective, open-label, single arm 3-year clinical study to describe the short-term and long-term efficacy and safety of belimumab in participants with autoantibody positive early SLE with ongoing disease activity despite stable initial SLE therapy.

Start Date04 Jun 2024

Sponsor / Collaborator

GSK Plc

NCT04893161 / Not yet recruitingPhase 4IIT

A Model to Early Predict the Response of Belimumab Treatment in the Patients With Systemic Lupus Erythematosus

A prospective, single-center cohort study aims to determine a predictive model of the response of belimumab at Week 48.

Start Date01 Jun 2024

Sponsor / Collaborator

First Affiliated Hospital of Xi'an Jiaotong University

NCT06381453 / Not yet recruitingPhase 2

Belimumab in the Management of Autoimmune Hepatitis: A Multi-centre, Open-label Trial of add-on Belimumab Therapy to Standard of Care

Background: Autoimmune hepatitis (AIH) is a rare chronic and lifelong liver disease. Untreated, disease progresses to end-stage cirrhosis and the focus of therapy is with immunosuppression. Current therapies are limited, not targeted, and associated with side effects that patients report reduce quality of life. AIH is believed to arise as a consequence of genetic & environmental risks. Disease is characterised by impaired immunoregulation, that favours a chronic and relapsing hepatitis. As well as recognising an important role for cytotoxic T cells and regulatory T cells, it has become apparent that in AIH, as well as other related autoimmune conditions, that B-cells are important. AIH is characterised by a plasma cell rich interface hepatitis and elevated IgG concentrations. Furthermore B-cell lineages interact with regulatory T-cells. Off-label use of Rituximab, an anti-CD20 agent, has been described for patients with AIH. A number of other ways of effectively targeting B-cells in the treatment of related autoimmune diseases have also been developed, but there have been limited studies in people living with autoimmune hepatitis. Belimumab is a human monoclonal antibody that inhibits B-cell activating factor (BAFF), also known as B-lymphocyte stimulator. It is approved in the Canada to treat systemic lupus erythematosus and lupus nephritis. It has not been studied before in AIH, but off-label reports are published. In an open-label clinical trial of people living with autoimmune hepatitis, the investigator will now formally study the effect of adding Belimumab to existing standard of care, with the goal being to evaluate treatment efficacy, the ability to reduce the burden of existing therapies whilst still controlling AIH disease, and to describe the tolerability & safety of Belimumab in people with AIH. Study Design: Open label, multi-centre, Canadian clinical trial. Patient population: Patients with autoimmune hepatitis, excluding patients with decompensated liver disease, who either have active disease despite standard of care (Group A), or who are maintained with disease remission using standard of care therapy (Group B). 48 patients will be recruited. Intervention: Weekly sub-cutaneous Belimumab. Duration: 72 weeks with interim analysis after 24 patients have been treated for 24 weeks; target recruitment 48 patients. Evaluation: Safety, Serum liver tests, quality of life, exploratory immunologic biomarkers, optional liver biopsy or fine needle liver aspirate. Primary end-point: Group A: 50% or more of subjects have an ALT<2x ULN & corticosteroids at a dose of

Start Date30 Apr 2024

Sponsor / Collaborator

University Health Network

[+1]

100 Clinical Results associated with Belimumab

100 Translational Medicine associated with Belimumab

100 Patents (Medical) associated with Belimumab

1,075

Literatures (Medical) associated with Belimumab

01 May 2024·Current opinion in rheumatology

Emerging biologic therapies for systemic lupus erythematosus

Review

Author: Kato, Hiroshi ; Kahlenberg, J. Michelle

Purpose of review:

The approval of belimumab and anifrolumab has expanded the scope of treatment for systemic lupus erythematosus (SLE) patients. However, many patients remain refractory to currently available therapies and suffer from drug toxicities. This review will discuss approved and target-specific therapeutics in development that bring hope for better SLE treatments.

Recent findings:

Since the last review on this subject in the journal, the FDA has approved anifrolumab and belimumab for SLE and lupus nephritis (LN), respectively. A fully humanized anti-CD20, obinutuzumab, met the primary end point in a phase II trial in LN. A Tyk2 inhibitor, deucravacitinib, and an antibody targeting plasmacytoid dendritic cells, litifilimab, met the primary end point in phase II trials in SLE and cutaneous lupus erythematosus (CLE). Ustekinumab and baricitinib met the primary end point in phase II but not in phase III trials.

Summary:

While many drug candidates which met the end points in phase II trials have failed phase III trials, the number of target-specific therapies for SLE has continued to expand.

01 Feb 2024·Rheumatology and therapy

Correction: Real-World Clinical Outcomes in Belimumab-Treated US African American and Hispanic Patients with Systemic Lupus Erythematosus: A Retrospective, Observational Study

Author: Chung, Jake ; Rubin, Bernard ; Bell, Christopher F

01 Feb 2024·CNS drugs

The Efficacy and Safety of Different Targeted Drugs for the Treatment of Generalized Myasthenia Gravis: A Systematic Review and Bayesian Network Meta-analysis

Review

Author: Nie, Xiangtao ; Ma, Yongbo ; Zhu, Geke ; Hao, Lei ; Guo, Xiuming ; Qi, Wenjing

BACKGROUND:

The treatment of generalized myasthenia gravis (gMG) has been transformed by the development and approval of new targeted therapies. This analysis aimed to rank and compare the new therapies for gMG using efficacy and safety data from randomized controlled trials (RCTs).

METHODS:

We searched PubMed, Embase, the Cochrane Central Register of Controlled Trials (CENTRAL), and ClinicalTrials.gov (up to November 2022) for RCTs of targeted drugs for gMG. We used a Bayesian random-effects network meta-analysis (NMA) model and a Markov chain Monte Carlo (MCMC) model for statistical analysis. The primary outcome was the change in quantitative myasthenia gravis score (QMGS) from baseline, while the secondary outcome was the risk ratio (RR) of adverse events (AEs) during treatment. The surface under the cumulative ranking curve (SUCRA) was used to rank these targeted drugs, with higher SUCRA values indicating better efficacy or lower likelihood of AEs.

RESULTS:

In total, 13 studies (872 subjects) were included in this analysis evaluating 10 targeted drugs (batoclimab, belimumab, CFZ533, eculizumab, efgartigimod, nipocalimab, rituximab, ravulizumab, rozanolixizumab, and zilucoplan). With regards to the primary outcome, batoclimab [standardized mean difference (SMD), - 1.61; 95% credible interval (CrI), - 2.78, - 0.43] significantly reduced QMGS in patients with gMG when compared with placebo and was ranked as the most efficacious drug. Ranked second and third were eculizumab (SMD, - 0.67; 95% CrI, 1.43, 0.01) and zilucoplan (SMD, - 0.54; 95% CrI, - 1.56, 0.46), respectively. Nipoclimab (SMD, - 0.02; 95% CrI, - 1.04, 1.00) had the worst efficacy and ranked last among all targeted drugs. In our study, except for batoclimab, there was no statistically significant difference in the reduction of patient QMGS for the remaining targeted agents compared with placebo. With regards to the secondary outcomes, only batoclimab (RR, 0.19; 95% CrI, 0, 0.97) led to a significant reduction in the incidence of AEs when compared with the placebo. Belimumab (RR, 0.85; 95% CrI, 0.57, 1.19), CFZ533 (RR, 0.95; 95% CrI, 0.72, 1.25), eculizumab (RR, 0.99; 95% CrI, 0.85, 1.21), and efgartigimod (RR, 0.93; 95% CrI, 0.76, 1.15) also led to a lower incidence of AEs, although these effects were not significantly different from the placebo.

CONCLUSIONS:

Batoclimab had the best efficacy and safety for the treatment of gMG and was ranked first out of the 10 targeted drugs included in this study. Eculizumab was ranked second, and nipocalimab had the worst efficacy. With the exception of batoclimab, the incidence of AEs for the remaining drugs was not statistically significantly different from placebo. We note, however, that wide CrIs reflect the uncertainty in this analysis owing to the small number of available studies and low numbers of study participants; moreover, batoclimab had the widest CrI of all drugs in this analysis. More well-designed studies with long-term follow-up are needed to further evaluate and compare the efficacy and safety of these drugs in the future.

News (Medical) associated with Belimumab

14 Jun 2024

·www.biospace.com

Cabaletta Bio Reports Positive Initial Clinical Data from Phase 1/2 RESET-Myositis™ and RESET-SLE™ Trials of CABA-201

No CRS, ICANS, infections or serious adverse events observed in either of the first two patients through data cut-off of May 28, 2024 CABA-201 exhibited anticipated pro CAR T cell expansion and contraction with complete B cell depletion observed in both patients by day 15 post-infusion Improvements in both patients’ specific disease measures, consistent with academic experience of a similar 4-1BB CD19-CAR T, suggest emerging clinical benefit with CABA-201 while discontinuing all disease-specific therapies other than a planned steroid taper in one patient Immature, naïve B cell repopulation in first IMNM patient observed at week 8 consistent with a potential immune system reset 18 sites open and recruiting across four Phase 1/2 RESET™ trials with 5 patients enrolled as of June 12, 2024; initial clinical and translational data support continued development of CABA-201 at the current dose Company to host live investor conference call and webcast today at 8:00 a.m. ET PHILADELPHIA, June 14, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases, today reported positive initial clinical data from each of the first two patients dosed with CABA-201 in the Phase 1/2 RESET-Myositis and RESET-SLE trials. These data will be presented today at 8:15 a.m. CEST (2:15 a.m. ET) at a EULAR European Congress of Rheumatology 2024 Industry Symposia session titled “Immune Reset: The Potential of CAR T Cell Therapy to Transform the Treatment of Patients with Autoimmune Disease” in Vienna, Austria. Slides from the presentation can be found on the company’s website here. “We are encouraged by the initial safety, clinical and translational data from the RESET-Myositis and RESET-SLE trials which we believe provide important early validation regarding the potential of the selected clinical dose of CABA-201 to enable an immune system reset for patients with autoimmune diseases. By demonstrating a potentially well-tolerated safety pro with initial clinical and translational data consistent with the academic experience of a similar 4-1BB CD19-CAR T construct, we believe CABA-201 may be uniquely positioned to fulfill unmet patient needs across a broad range of autoimmune diseases,” said David J. Chang, M.D., Chief Medical Officer of Cabaletta. “With the RESET-SSc™ and RESET-MG™ trials recently opening for enrollment, an additional cohort evaluating patients with juvenile myositis incorporated into the RESET-Myositis trial and the momentum provided by the promising early clinical data, we are looking forward to accelerating clinical trial enrollment in the RESET clinical program. We continue to expect to report initial clinical data from the Phase 1/2 RESET-SSc and RESET-MG trials as well as additional data from the RESET-Myositis and RESET-SLE trials in the second half of this year.” Cabaletta designed CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, to deeply and transiently deplete CD19-positive B cells following a one-time infusion that may enable a reset of the immune system with the potential for durable remission without chronic therapy in patients with autoimmune diseases. Cabaletta is advancing four Phase 1/2 RESET trials evaluating CABA-201 within a total of ten cohorts with six patients in each cohort. All cohorts are evaluating the same single, weight-based dose of 1 x 106 cells/kg, following a preconditioning regimen of fludarabine and cyclophosphamide consistent with the dosing regimen used in the academic experience, without a dose escalation requirement. As of May 28, 2024, the data cut-off date, one patient treated in the immune-mediated necrotizing myopathy (IMNM) cohort in the RESET-Myositis trial had completed three months of follow-up and one patient enrolled in the systemic lupus erythematosus (SLE) non-renal cohort in the RESET-SLE trial had completed one month of follow-up. The patient with IMNM is a 33-year-old male with a two-year history of disease, positive for anti-SRP antibody and who had prior disease-specific therapy that included IVIg, rituximab, methotrexate and glucocorticoids. The patient with SLE is a 26-year-old male with a six-year history of disease, positive for anti-dsDNA antibody and who had prior disease specific therapy that included cyclophosphamide, voclosporin, belimumab, tacrolimus, mycophenolate mofetil, hydroxychloroquine and glucocorticoids. Both patients were administered a one-time infusion of CABA-201 at 1 x 106 cells/kg, following a preconditioning regimen of fludarabine and cyclophosphamide. The primary endpoint of each trial is safety and tolerability within 28 days of infusion. Secondary endpoints include translational assessments and clinical outcomes. Initial Clinical Data Summary Safety and Tolerability CABA-201 was administered during a four-day hospital stay, as currently required by the protocol, and was generally well-tolerated with no serious adverse events reported for either patient through the follow-up period. No evidence of cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) of any grade was observed for either patient through the follow-up period. Tocilizumab was not administered for either patient. No infections were observed for either patient through the follow-up period. All chronic maintenance therapy or concomitant medications were discontinued for both patients through the follow-up period, other than a planned prednisone taper for the SLE patient. Clinical and Translational Pro > Complete B cell depletion was observed within 15 days post-infusion with CABA-201 in both patients. Both patients had early, transient leukopenia, as expected with the preconditioning regimen. CAR T cell expansion associated with CABA-201 reached its peak magnitude at day 15 post-infusion in both patients and the magnitude of expansion was consistent with the academic experience with a similar 4-1BB CD19-CAR T construct. At week 12 of follow-up for the IMNM patient, the data show a decline in creatinine kinase from 617 at infusion to 308 and a total improvement score (TIS) of 30, which is consistent with the clinically meaningful improvement seen in the academic experience of a similar 4-1BB CD19-CAR T construct that also recently reported data from an IMNM patient. At week 4 of follow-up for the SLE patient, the data demonstrated an improvement in the SLEDAI-2K (systemic lupus erythematosus disease activity index) score from 26 at baseline to 10. B cell repopulation was observed in the IMNM patient at week 8 with immature, naïve B cell phenotypes as demonstrated by flow cytometry, suggesting potential immune system reset with confirmatory analyses ongoing. Investor Conference Call and Webcast Information Cabaletta will host a conference call and webcast today, June 14, 2024, at 8:00 a.m. ET to review the initial clinical data presented at the satellite symposium at the EULAR 2024 Congress and provide an update on the RESET clinical development program. A webcast of the live call can be accessed on the News and Events section of the Company’s website at . An archived replay will be available on the Company’s website. About the RESET-Myositis™ Trial The RESET-Myositis™ trial is a Phase 1/2 open-label study of CABA-201 in subjects with active idiopathic inflammatory myopathy (IIM, or myositis), including the subtypes of dermatomyositis (DM), anti-synthetase syndrome (ASyS), immune-mediated necrotizing myopathy (IMNM) and juvenile myositis (JM), each evaluated in individual cohorts. Subjects will receive a one-time infusion of CABA-201 at a dose of 1 x 106 cells/kg, following a preconditioning regimen of fludarabine and cyclophosphamide. Key inclusion criteria for the DM, ASyS and IMNM cohorts include patients between ages 18 to 75 (inclusive), evidence of active disease and disease activity despite prior or current treatment with standard of care treatments. Key exclusion criteria for the DM, ASyS and IMNM cohorts include cancer-associated myositis, significant lung or cardiac impairment, treatment with a B cell depleting agent within the prior approximately six months or treatment with a biologic agent within the prior approximately three months. About the RESET-SLE™ Trial The RESET-SLE™ trial is a Phase 1/2 open-label study of CABA-201 in subjects with systemic lupus erythematosus (SLE) and lupus nephritis (LN), each evaluated in individual cohorts. Subjects will receive a one-time infusion of CABA-201 at a dose of 1 x 106 cells/kg, following a preconditioning regimen of fludarabine and cyclophosphamide. Key inclusion criteria include patients between ages 18 to 65 (inclusive), evidence of active disease and disease activity despite prior or current treatment with standard of care treatments. Key exclusion criteria include treatment with a B cell depleting agent within the prior approximately six months or treatment with a biologic agent within the prior approximately three months. About CABA-201 CABA-201 is designed to deeply and transiently deplete CD19-positive cells following a one-time infusion, which may enable an “immune system reset” with the potential for durable remission without chronic therapy in patients with autoimmune diseases. Cabaletta is evaluating CABA-201 in multiple autoimmune conditions within five disease-specific company sponsored INDs including myositis (idiopathic inflammatory myopathy, or IIM), systemic lupus erythematosus (SLE), systemic sclerosis (SSc), generalized myasthenia gravis (gMG) and pemphigus vulgaris (PV; a sub-study to evaluate CABA-201 without preconditioning). About Cabaletta Bio Cabaletta Bio (Nasdaq: CABA) is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies that have the potential to provide a deep and durable, perhaps curative, treatment for patients with autoimmune diseases. The CABA™ platform encompasses two strategies: the CARTA (chimeric antigen receptor T cells for autoimmunity) strategy, with CABA-201, a 4-1BB-containing fully human CD19-CAR T, as the lead product candidate being evaluated in the RESET™ (REstoring SElf-Tolerance) clinical trials in systemic lupus erythematosus, myositis, systemic sclerosis and generalized myasthenia gravis and in the RESET-PV™ sub-study within the DesCAARTes™ clinical trial in pemphigus vulgaris, along with the CAART (chimeric autoantibody receptor T cells) strategy, with multiple clinical-stage candidates, including DSG3-CAART for mucosal pemphigus vulgaris and MuSK-CAART for MuSK-associated myasthenia gravis. The expanding CABA™ platform is designed to develop potentially curative therapies that offer deep and durable responses for patients with a broad range of autoimmune diseases. Cabaletta Bio’s headquarters and labs are located in Philadelphia, PA. Forward-Looking Statements This press release contains “forward-looking statements” of Cabaletta Bio within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including without limitation, express or implied statements regarding: Cabaletta’s ability to grow its autoimmune pipeline; Cabaletta’s future plans and strategies for its CAAR T and CARTA technologies and the company’s business plans and objectives as a whole; Cabaletta’s expectations around the potential safety and therapeutic benefits of CABA-201, including its belief that CABA-201 may enable an “immune system reset” with the potential for durable remission without chronic therapy in patients with autoimmune diseases; the Company’s advancement of separate Phase 1/2 clinical trials of CABA-201 in patients with SLE, myositis, SSc and gMG and advancement of a RESET-PV sub-study within the ongoing DesCAARTes trial in PV, including the Company’s expectations for the efficiency of the trial designs and updates related to status, safety data, or otherwise and the expected timing of the related data read-outs; Cabaletta’s ability to accelerate its pipeline, develop meaningful therapies for patients and leverage its research and translational insights; the clinical significance of the initial clinical data read-out at the EULAR 2024 Congress in June 2024 for patients with myositis and SLE treated with CABA-201; Cabaletta’s additional planned initial clinical data read-outs for patients with SSc and gMG treated with CABA-201 or otherwise; Cabaletta’s advancement of the process to activate clinical trial sites and pursue patient enrollment; and Cabaletta’s planned assessment of its DesCAARTes™ and MusCAARTes™ trials. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to regulatory filings and potential clearance; the risk that signs of biologic activity or persistence may not inform long-term results; Cabaletta’s ability to demonstrate sufficient evidence of safety, efficacy and tolerability in its preclinical studies and clinical trials of CABA-201; the risk that the results observed with the similarly-designed construct employed in academic publications, including due to the dosing regimen, are not indicative of the results we seek to achieve with CABA-201; risks related to clinical trial site activation, delays in enrollment generally or enrollment rates that are lower than expected; delays related to assessment of clinical trial results; risks related to unexpected safety or efficacy data observed during clinical studies; risks related to volatile market and economic conditions and public health crises; Cabaletta’s ability to retain and recognize the intended incentives conferred by Orphan Drug Designation and Fast Track Designation or other designations for its product candidates, as applicable; risks related to Cabaletta’s ability to protect and maintain its intellectual property position; risks related to fostering and maintaining successful relationships with Cabaletta’s collaboration and manufacturing partners, including in light of recent legislation; uncertainties related to the initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Cabaletta’s product candidates will not be successfully developed and/or commercialized; and the risk that the initial or interim results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Cabaletta’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Cabaletta’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Cabaletta’s other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Cabaletta undertakes no duty to update this information unless required by law. Contacts: Anup Marda Chief Financial Officer investors@cabalettabio.com William Gramig Precision AQ william.gramig@precisionaq.com

Clinical ResultImmunotherapyCell TherapyOrphan DrugFast Track

27 Mar 2024

·www.prnewswire.com

AMPEL BioSolutions' Lupus Test Launches to Predict Flares & Drug Targets

CHARLOTTESVILLE, Va., March 27, 2024 /PRNewswire/ -- AMPEL BioSolutions announces a breakthrough in personalized precision medicine that will revolutionize the way doctors manage the autoimmune disease Lupus. Revealed in the peer-reviewed journal Genome Medicine and being made available to patients through a US follow-on study, the LuGENE® blood test forecasts patient flares and drug targets from gene expression utilizing predictive AI. The LuGENE® lab test is now launching at several Lupus practices around the country including those in medical centers, concierge practices and healthcare systems as a practical decision support biomarker test that could greatly impact day-to-day healthcare for patients with Lupus. The blood test predicts flares before they happen and provides specific information about a patient's disease abnormalities leading to symptoms, allowing physicians to make evidence-based decisions regarding therapy including whether a patient is stable on their current therapeutic regimen. There are a million and a half lupus patients in the US that could benefit from LuGENE® by preventing unexpected flares and shortening the time to disease-controlling therapy. From RNA gene expression in cells circulating in the blood, LuGENE® identifies real-time abnormalities leading to inflammation and immune system dysfunction that are targetable by medication. Some abnormalities are well-known and others are novel. Until now, therapeutic intervention is a trial-and-error endeavor that often takes years until a patient is stabilized without further threat of organ-damaging flares. LuGENE® clarifies that there are eight subgroups that make up the universe of Lupus patients, the reason why getting the right drug to the right patient at the right time has been so challenging, as until now physicians did not have a tool to know what drug targets are abnormal in their patient. AMPEL's approach pinpoints the patient subgroup(s) that express the target of one or both of the FDA-approved drugs for lupus, Benlysta and Anifrolumab, providing decision support for physicians to potentially shorten the time to effective therapy and potentially improve quality of life. Diversity across ancestries is a hallmark of Lupus and the AMPEL R&D team included patients from all ethnicities to ensure that LuGENE® is predictive for all patients in the United States living with Lupus. This is especially important as Lupus emerges earlier and with greater severity in African-American, Asian, Native-American and Hispanic/Latinx communities shortening life-span. Lupus has been called the "cruel mystery" because until now doctors have few objective metrics to assess whether their patient is stable with low risk of flare. Patients with Lupus experience inflammation and pain from their immune system attacking their own organs and tissues with the same vigor as fighting off an infection. Ninety-five percent of Lupus patients have experienced a flare in the last year. Symptoms including extreme fatigue and "brain fog" that impact daily activities, including work and family life, has led to Lupus being referred to as an "invisible disability". A Lupus Foundation of America survey found that fifty-five percent of patients with Lupus reported a complete or partial loss of their income because they no longer are able to work full time due to complications of lupus; one in three have been temporarily disabled by the disease, and one in four currently receive disability payments. "AMPEL is an industry leader in lupus disease management, and we are excited to unveil our latest advancement in the field of precision medicine," said Dr. Amrie Grammer, Co-Founder and President/CSO of AMPEL BioSolutions. "With so many patients experiencing a flare every year, and a high percentage of those cases so severe that they are hospitalized, this is a crucial tool in our fight against Lupus. With LuGENE®, healthcare providers will be able to stabilize lupus patients faster than ever before, minimize the occurrence of disruptive flares and hopefully transform Lupus to a chronic condition with increased quality of life." "Currently, there is no way to predict flares or determine which treatment is most appropriate for each patient, and in many instances it can take many years for some patients to find the most effective one to manage their disease," said Dr. Peter Lipsky, Co-Founder and CEO/CMO of AMPEL BioSolutions. "Healthcare professionals can now know whether a flare in disease activity in imminent and if the disease is not controlled, LuGENE® can identify the specific targets of therapies that are more likely to provide relief. The LuGENE® blood test is a monitoring biomarker that provides essential information to support personalized management for each individual lupus patient." "As a clinician, I am tired of wasting my patients' valuable time and money by trying different therapies for their Lupus using the 'trial and error' methodology, which is our only option now," said Dr. Don Thomas, Rheumatologist at Arthritis & Pain Associates of Prince George's County in Maryland, author of the Lupus Encyclopedia & Former Chair Advisory council Lupus Foundation of America DC/MD/VA, "This approach to taking care of Lupus patients ends up causing so much misery as they deal with life-altering symptoms and complications when therapies do not work or cause them side effects. The dream of using personalized medicine to assist me in choosing therapies that have a greater chance of working, based on their endotype and actual immunologic abnormalities, is closer to becoming a reality with the LuGENE® blood test." "Lupus is a very complicated disease that is pleomorphic in nature and very difficult to treat," said Dr. Daniel Wallace, author of Dubois SLE and The Lupus Book & Rheumatologist at Cedars-Sinai, UCLA, Wallace-Lee Center and BoD member of the Lupus Research Alliance affiliate Lupus Therapeutics and LupusLA, "The LuGENE® blood test is a novel and innovative approach that has the potential to revolutionize the way heterogenous Lupus patients are managed by identifying their genomic biomarker fingerprint and predict if the disease is stable (or not) and what drug targets are abnormal indicating therapeutic options to consider." "I want to express my excitement to be one of the first practices to utilize the LuGENE® genomic biomarker test for Lupus," Dr. John Tesser, Arizona Arthritis and Rheumatology Associates, "to potentially manage patients more specifically by being informed if a patient is stable on their current medication or if particular drug targets are abnormal. AMPEL's Genomic Platform with RNA analytics and machine learning is highly unique and is poised to provide true personalized medicine for patients living with Lupus. I am most enthusiastic to participate in the LuGENE® study called ReLATE that will examine the relationship between standard clinical & lab parameters with prediction of flare & abnormal drug targets." "The LuGENE® blood test is incredibly exciting as it represents an important advance towards achieving personalized medicine for Lupus patients by using novel technology of explainable predictive AI and analytics that group all known gene expression by drug target," Dr. Cindy Aranow, Feinstein Institute at Northwell Health. "A key part of the AMPEL Genomic approach is subsetting/endotyping patients by their RNA biomarker fingerprint that predict a patient's disease status and potential to flare. I am delighted to be one of practices that is launching LuGENE® through the ReLATE study." "I am excited about the publication describing LuGENE® as a blood test that offers rheumatologists clinically relevant information in real-time predicted by AMPEL's explainable-AI powered RNA analytic platform, that should be highly informative about an individual lupus patient's status," Dr. Orrin Troum, The Doctors of Saint John's Medical Center, Providence Healthcare "This approach could provide a real advance in personalized medicine for Lupus patients. Additionally, I am thrilled to be one of the first practices participating in the LuGENE® study called ReLATE, whose clinical report I anticipate will help me tailor Lupus management." During the interview with Scripps News Network, Dr. Grammer explains how there is currently no way to predict flares or determine which is the most appropriate treatment for an individual lupus patient. Identifying a therapeutic regimen that stabilizes a patient's disease course often takes several years of trial and error. LuGENE® is currently available through the ReLATE study at Cedars-Sinai in LA and Arthritis & Osteoporosis Consultants of the Carolinas (AOCC) in Charlotte NC. The study is expanding to sites across the US including Arizona Arthritis & Rheumatology, Feinstein at Northwell Health, Mayo Clinic, Providence Healthcare, Rush in Chicago, Hospital for Special Surgery, Cleveland Clinic, Univ of Maryland, Yale, MetroHealth at Case Western Reserve, U Mass, Duke and others. Physicians and patients enrolled at these centers will experience how the LuGENE® blood test informs lupus patient management. Kathleen Arntsen, Lupus and Allied Diseases Association (LADA) President & CEO, "For people struggling daily to live with complex autoimmune diseases like Lupus, what motivates us to face another day is the promise and progress of research that will improve our quality of life. Our community has eagerly awaited the development of more effective therapeutic approaches that can be tailored to each individual. This groundbreaking LuGENE® blood test has the potential to achieve this goal and transform patient care not only for lupus but for other autoimmune conditions." Emmitt Thompson III, Male Lupus Warriors, "Being able to predict a flare before it happens and get on stabilizing medications for my Lupus is a game changer! As patient who ends up in the ER frequently with organ damaging autoimmunity, I am encouraged by the possibility of getting ahead of Lupus inflammation by monitoring with the LuGENE® blood test." Deidre Gee Baptista and Kirsten Gee Maeda, GEE Twins for Lupus and Lupus Foundation of Northern California Board Members: "With two decades of experiencing the ups and downs of chronic Lupus, we are thrilled for a truly more personalized approach to managing and treating this chronic autoimmune disease. The LuGENE® blood test provides a much needed, personalized approach to reducing inflammation, pain and what are now 'unpredictable flares'. Families navigating their lupus journeys look forward to getting ahead of autoimmune organ damage by getting to an effective treatment more quickly, helping patient's lead productive lives." Jay Remley, Inaugural Google Cloud Global Dir & Vice-Chair Lupus Foundation NC, "Lupus is a complex autoimmune disease that is often mis-treated for years, causing pain and anxiety for people living with this lifelong condition. The LuGENE® blood test may address this critical care need. One of the most challenging times for a Lupus warrior is the time to establish an initial therapy. The second most difficult time is the 24x7 anxiety that a patient endures waiting for the inevitable next flare to arrive. LuGENE® will provide decision support to physicians re whether a patient is stable on the current drug regimen and specifics about what drug targets are abnormal right now, both of which are monumental breakthroughs for the Lupus community." McKenna Bowes, Molecular Biology Student, "As an undergraduate student in the Sawalha lab at the Univ. of Pittsburgh (BS, May 2025) and an aspiring medical student, understanding the science behind LuGENE® makes it even more exciting! Even after almost five years of living with lupus, I still find that managing symptoms and trying different medications can be a frustrating and lengthy process. The ability of the LuGENE® blood test to predict flares and identify different lupus endotypes with the LuGENE® score offers a more personalized and targeted approach to my lupus care, ultimately alleviating some of these challenges for patients overall . I believe that this test can significantly improve the lives of those living with lupus." Sobia Saleem MA LMFT, Psychotherapist and Lupus Advocate, "I am beyond thrilled at the launch of the LuGENE®. As a Lupus patient 10+ years, a blood test that can both predict flares and help my medical team prescribe me targeted therapies that work best with my body is an unprecedented leap. One of my greatest struggles is that Lupus is unpredictable and can rob you of important moments decreasing quality of life due to volatility in disease activity. To understand a flare is approaching in addition to what pathways are abnormal would be life-changing! I have wasted literal years of my life in pain because the current model of lupus treatment is a trial-and-error approach. The LuGENE® blood test solves two of the most unknown variables in a patient's journey," Estela & Juana Mata, Looms For Lupus, "As advocates for those living with lupus, and as people who live with lupus, we are thrilled about the LuGENE® blood test. Innovative approaches and precision medicine are crucial new ways to predict flares and manage care. More than exciting scientific advancements, personalized precision medicine is our hope for reducing irreversible damage and complications from unexpected flares. This LuGENE® personalized blood test represents a transformative impact on patient care, giving those living with lupus, and potentially others living with autoimmune conditions, the knowledge of what drug targets are abnormal so they can discuss early treatment opportunities with their doctors to potentially improve quality of life." Christine von Raesfeld, NIH All of Us Ambassador and People with Empathy Founder: "As a Lupus patient of mixed ancestry, I've experience long-term damage from standard treatments. I believe LuGENE® offers immense promise for our autoimmune community. By analyzing our gene expression profile, LuGENE® empowers patients to tailor their treatments, especially crucial for minority populations often underserved by current therapeutic approaches. Instead of "trial and error" prevalent in today's standard-of-care, LuGENE® provides a path to targeted treatment that minimizes flares and bridges the gap between science and personalized precision care." About AMPEL BioSolutions AMPEL is a Precision Medicine company commercializing a development pipeline of blood tests that measure dynamic RNA gene expression providing a real-time CLIA-certified report of inflammation status including drugs targets that are abnormal. Unlike DNA tests which only need to be done once, AMPEL's RNA blood or biopsy tests are utilized on demand with the goal of "treat to target" and optimal disease management for Lupus (LuGENE®) or Dermatology Conditions such as Psoriasis or Ezcema (DermaGENE®). For an otherwise healthy individual, AMPEL's prediction of inflammation with WellGENE® may be an early indicator of changes in the immune system that may be worth investigating further by a trained healthcare provider. In addition, AMPEL works with Pharma/Biotech customers for target identification, pre-clinical models, post-hoc analysis and real-time enrichment during clinical trial enrollment. The company's portfolio of tests are supported by the AMPEL Genomic Platform of RNA analytic tools and explainable predictive AI. SOURCE AMPEL BioSolutions

Clinical Study

15 Feb 2024

·www.biopharmadive.com

Aurinia to cut research after sale process fails to find a buyer

Dive Brief:Aurinia Pharmaceuticals will restructure its operations and give money back to shareholders after failing to find a buyer willing to take over the company.Investors can expect a buyback of as much as $150 million worth of stock, Aurinia said Thursday. The company is also planning to downsize, resulting in what it expects will be annual savings of as much as $55 million.Aurinias first move is the discontinuation of research around two experimental drug programs, AUR200 and AUR300. By the end of the first quarter, the company also plans to cut at least 25% of its workforce.Dive Insight:Aurinias actions are the result of a strategic review it began in June last year under pressure from shareholder MKT Capital. While seeking buyers or other alternatives, the company reshuffled its board and reached a cooperation agreement with MKT in September.During the review, company officials and financial adviser J.P. Morgan engaged with more than 60 parties, Aurinia said. But the effort resulted in only one expression of interest that didnt lead to a formal offer. The company also disclosed that a similar, confidential strategic review in 2018 likewise resulted in only one expression of interest and no formal offer.Other options such as licensing assets from other companies also didnt bear fruit during the latest review, and the board decided to wrap up and instead narrow the companys focus to its sole marketed medicine, Lupkynis, for a form of lupus that affects the kidneys. Employees involved in marketing or supporting commercialization of Lupkynis will be spared from layoffs.Lupkynis in early 2021 became the first oral treatment approved in the U.S. for lupus nephritis. It competes with GSKs Benlysta, an older injectable lupus medicine that won an expanded approval in late 2020 to treat lupus nephritis. Aurinia said it expects its product to yield revenue of $200 million to $220 million this year.Even as it switches gears, Aurinia made clear that its still open to any bona fide offers. The company had about $350 million in cash and equivalents as of Dec. 31. '

Drug ApprovalAccelerated Approval

100 Deals associated with Belimumab

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KEGG	Wiki	ATC	Drug Bank
D03068	Belimumab	L04AA26	DB08879

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Lupus Nephritis	EU	GlaxoSmithKline (Ireland) Ltd.	13 Jul 2011
Lupus Nephritis	IS	GlaxoSmithKline (Ireland) Ltd.	13 Jul 2011
Lupus Nephritis	LI	GlaxoSmithKline (Ireland) Ltd.	13 Jul 2011
Lupus Nephritis	NO	GlaxoSmithKline (Ireland) Ltd.	13 Jul 2011
Systemic Lupus Erythematosus	US	GlaxoSmithKline LLC	09 Mar 2011

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Interstitial lung disease due to systemic disease	Phase 3	US	GSK Plc	13 Sep 2023
Interstitial lung disease due to systemic disease	Phase 3	CN	GSK Plc	13 Sep 2023
Interstitial lung disease due to systemic disease	Phase 3	JP	GSK Plc	13 Sep 2023
Interstitial lung disease due to systemic disease	Phase 3	AR	GSK Plc	13 Sep 2023
Interstitial lung disease due to systemic disease	Phase 3	AU	GSK Plc	13 Sep 2023
Interstitial lung disease due to systemic disease	Phase 3	BE	GSK Plc	13 Sep 2023
Interstitial lung disease due to systemic disease	Phase 3	BR	GSK Plc	13 Sep 2023
Interstitial lung disease due to systemic disease	Phase 3	CA	GSK Plc	13 Sep 2023
Interstitial lung disease due to systemic disease	Phase 3	DK	GSK Plc	13 Sep 2023
Interstitial lung disease due to systemic disease	Phase 3	FI	GSK Plc	13 Sep 2023

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02347891 (CTgov)	Phase 2/3	Myositis	17	Belimumab (Arm 1 (Belimumab + SoC (Randomized Phase and Open Label Phase))	miwywsvbbo(eebggohqyt) = djeyuxtvby agjinepnua (gemmcanzhu, lqwriwnbyw - krrrrzehwt) View more	-	09 Feb 2024
				Placebo+Belimumab (Arm 2 (Placebo + SoC (Randomized Phase) Followed by Belimumab + SoC (Open Label Phase))	miwywsvbbo(eebggohqyt) = ciuegiylpj agjinepnua (gemmcanzhu, klnookihpn - dneguibxsl) View more
NCT00424476 \| NCT01345253 \| NCT01484496 \| NCT00410384 (BLISS-NEA \| BLISS-52 \| BLISS-76 \| BLISS-SC, Pubmed)	Phase 3	Lupus Nephritis	3,225	Intravenous belimumab 1 mg/kg	-	Positive	01 Feb 2024
				Intravenous belimumab 10 mg/kg
NCT01705977 (BASE, ACR2023)	Phase 4	Systemic Lupus Erythematosus autoantibody-positive	4,003	Belimumab (BEL)	yvlbmdncgw(ppkdpalboe) = cxexghwech mrzrbrxeyq (tywunzcblx ) View more	Positive	13 Nov 2023
				Placebo (PBO)	yvlbmdncgw(ppkdpalboe) = fbnuoamtxj mrzrbrxeyq (tywunzcblx ) View more
ACR2023	Not Applicable	Systemic Lupus Erythematosus SARS-CoV-2 antibody titers	21	Belimumab	edmstftudv(nqenmdckqp) = mild myalgia civtmuqwkc (qlrcdbarlc )	Positive	13 Nov 2023
ACR2023	Not Applicable	Systemic Lupus Erythematosus	-	Belimumab optimized dose	sdgocchqel(nohbxgukyp) = fbhnuuagxb qfmqxgnres (yeictjsxnt ) View more	-	12 Nov 2023
BEAT-lupus, CALIBRATE (ACR2023)	Not Applicable	Systemic Lupus Erythematosus IgA2 anti-dsDNA antibodies	43	Belimumab after rituximab and cyclophosphamide (RCB)	zucgydihsf(lgwqihvwep) = tjygorziqx tfgvbtotmp (dzugczwmcm )	Positive	12 Nov 2023
				Rituximab and cyclophosphamide (RC)	zucgydihsf(lgwqihvwep) = tealsxwezo tfgvbtotmp (dzugczwmcm )
NCT03244059 (CTgov)	Phase 2	Emphysema \| Pulmonary Emphysema	17	Belimumab (Belimumab)	xxpjqhouyd(oiprllgkgn) = jsaspmdebh ynsrhuuelo (nleecqmcwz, iodselubqu - uyivvdmasj) View more	-	18 Jul 2023
				Placebo (Placebo)	xxpjqhouyd(oiprllgkgn) = msyszmkqec ynsrhuuelo (nleecqmcwz, dvltgdvdos - fglauxvrox) View more
EULAR2023	Not Applicable	Systemic Lupus Erythematosus Maintenance	110	Belimumab+Standard-of-care	ttnmnbbxns(tskgosfzwc) = avazamlebm euooxyppya (xyjersimre ) View more	-	31 May 2023
				Standard-of-care	ttnmnbbxns(tskgosfzwc) = hjfgkgvgjm euooxyppya (xyjersimre ) View more
EULAR2023	Not Applicable	Systemic Lupus Erythematosus	27	Intravenous Belimumab	kksvkubgna(dffrlbsqqb) = lwhxwmueyc lcwazuudsd (mnenqzgtlh ) View more	-	31 May 2023
EULAR2023	Not Applicable	Systemic Lupus Erythematosus	15	Belimumab	rtgxgufetk(hscehtilqi) = None of the patients experienced a serious adverse event vksetywrly (ayhjtobulq )	-	31 May 2023

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