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Last update 17 Jan 2025

Bortezomib

Last update 17 Jan 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

[(1R)-3-methyl-1-({(2S)-3-phenyl-2-[(pyrazin-2-ylcarbonyl)amino]propanoyl}amino)butyl]boronic acid, Bortezomib (JAN/USAN/INN), N-[(1R)-1-(DIHYDROXYBORYL)-3-methylbutyl]-N-(pyrazin-2-ylcarbonyl)-L-phenylalaninamide

+ [16]

Target

Proteasome

Mechanism

Proteasome inhibitors

Therapeutic Areas

NeoplasmsImmune System DiseasesCardiovascular Diseases+ [4]

Active Indication

Immunoglobulin Light-Chain AmyloidosisWaldenstrom MacroglobulinemiaMantle-Cell Lymphoma+ [14]

Inactive Indication

Acute Graft Versus Host DiseaseAcute Lymphoblastic LeukemiaAcute myeloid leukaemia with 11q23 abnormality+ [102]

Originator Organization

Millennium Pharmaceuticals, Inc.

Active Organization

Sun Pharmaceutical Industries (Europe) BV

Celgene Corp.Janssen Research & Development LLC

+ [20]

Inactive Organization

Johnson & Johnson Pharmaceutical Research & Development LLC

Spectrum Pharmaceuticals, Inc.

Genentech, Inc.

+ [33]

Drug Highest PhaseApproved

First Approval Date

US (13 May 2003),

Multiple Myeloma

RegulationAccelerated Approval (US), Orphan Drug (US), Orphan Drug (EU), Orphan Drug (JP)

Structure/Sequence

Molecular FormulaC19H25BN4O4

InChIKeyGXJABQQUPOEUTA-RDJZCZTQSA-N

CAS Registry179324-69-7

1,083

Clinical Trials associated with Bortezomib

NCT06348147

/ Not yet recruitingPhase 2IIT

An Attenuated Schedule Dara-RVd Induction for Patients With Newly Diagnosed Multiple Myeloma Who Are Eligible for Autologous Stem Cell Transplantation

This Phase II hybrid decentralized trial will examine the effect of daratumumab-based quadruplet induction therapy administered at an attenuated schedule in subjects with newly diagnosed multiple myeloma (NDMM) who are eligible for standard-of-care autologous stem cell transplantation (ASCT). Daratumumab, lenalidomide, bortezomib, and dexamethasone (Dara-RVd) have recently become a standard induction regimen for patients with NDMM who are eligible for ASCT in the United States. As implemented in clinical trials, Dara-RVd involves twice weekly bortezomib administration, which is inconvenient for patients and may result in increased rates of limiting toxicity, such as peripheral neuropathy. Adoption of alternate schedules involving once-weekly bortezomib is common in real-world practice, however a paucity of prospective data supporting this practice exists.
This study examines the efficacy of an attenuated Dara-RVd schedule involving once-weekly bortezomib dosing.

Start Date01 Mar 2025

Sponsor / Collaborator

UNC Lineberger Comprehensive Cancer Center

NCT06015750

/ Not yet recruitingPhase 4

An Interventional, Prospective Open-Label Study of Immunosuppressive Therapies to Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia (RESTORE)

The primary objective of this study is to evaluate the effect of immunosuppressive therapy (IST) in participants treated with asfotase alfa who demonstrate immune-mediated loss of effectiveness (LoE).

Start Date15 Jan 2025

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

NCT05272826

/ RecruitingPhase 2IIT

A Single Arm, Response-adapted, Open Label Study of Iberdomide, Weekly Bortezomib and Dexamethasone (IberBd) With Isatuximab Added on Demand for Transplant-ineligible, Newly Diagnosed Multiple Myeloma Patients: the BOREALIS Trial

This study will evaluate efficacy and tolerability of iberdomide, bortezomib, dexamethasone and isatuximab on demand administered in combination.

Start Date01 Jan 2025

Sponsor / Collaborator-

100 Clinical Results associated with Bortezomib

100 Translational Medicine associated with Bortezomib

100 Patents (Medical) associated with Bortezomib

10,608

Literatures (Medical) associated with Bortezomib

01 Dec 2025·Blood Research

Efficacy of bortezomib combined with Hyper-CVAD in adults with relapsed acute lymphoblastic leukemia or positive measurable residual disease; effect of bortezomib in leukemia

Article

Author: Pérez Sámano, Daniela ; Gallardo Rodríguez, Adán Germán ; Martínez Tovar, Adolfo ; Cabrera García, Álvaro ; Olarte Carrillo, Irma ; Barranco Lampón, Gilberto ; Ramos Peñafiel, Christian Omar ; Terreros Palacio, Camila ; Martínez Murillo, Carlos

Abstract:

Purpose:

Despite advances in the treatment of adult acute lymphoblastic leukemia (ALL), relapse remains the most significant challenge in improving prognosis. Measurable residual disease (MRD) assessment can predict bone marrow relapse based on MRD positivity. As access to innovative therapies remains limited because of the high cost, chemotherapy is the widely utilized treatment option. The efficacy of a combination of bortezomib and Hyper-CVAD has been reported in patients with multiple myeloma; however, its efficacy has not yet been confirmed in patients with ALL.

Methods:

This prospective cohort study involved patients with ALL who presented with MRD-positive results or relapse and received treatment with a combination of bortezomib and Hyper-CVAD at two reference centers in Mexico City.

Results:

Of the 20 patients with positive MRD included in this study, 60% (n = 12) exhibited MRD negative results after combination treatment, 30% (n = 6) persisted positive MRD results, and 10% (n = 2) passed away. Of the 23 patients with bone marrow relapse, 43.5% (n = 10) achieved a second complete remission (2CR), 34.8% (n = 6) exhibited refractory status, and 21.7% (n = 5) passed away. To achieve a 2CR, 20% (n = 2) patients required less than four cycles of treatment, 50% (n = 5) required four cycles (two A and B cycles each), and 30% (n = 3) required six cycles.

Conclusion:

The combination of bortezomib and Hyper-CVAD treatment exhibited better results in achieving MRD negative results, indicating its potential as a promising first-line treatment strategy for ALL.

01 Mar 2025·CELLULAR SIGNALLING

tRNA methyltransferase DNMT2 promotes hepatocellular carcinoma progression and enhances Bortezomib resistance through inhibiting TNFSF10

Article

Author: Lai, Junzhong ; Li, Xinxin ; Chen, Zhirong ; Liang, Jiadi ; Chen, Xiaolan ; Lin, Jizhen ; Zhang, Yong ; Zhao, Heng ; Tian, Ling ; Chen, Qi ; Li, Qiumei ; Chen, Linqin ; Guo, Dong ; Zhao, Guangjian ; Fan, Jiqiang

The tRNA methyltransferase DNMT2 (TRDMT1) plays a crucial role in various biological functions; however, its role in cancer, particularly in liver cancer, remains incompletely understood. In this study, we demonstrate that high DNMT2 expression is negatively correlated with prognosis in clinical liver cancer patients. A series of in vitro and in vivo experiments showed that DNMT2 promotes the proliferation, colony formation, and metastasis of hepatocellular carcinoma cells. We identified the pro-apoptotic gene TNFSF10 (TRAIL) as a downstream target of DNMT2, regulated by the N6-methyladenosine (m6A) demethylase FTO. Epigenetically, DNMT2 deletion increased FTO expression, leading to a reduction in m6A methylation levels. FTO upregulated TNFSF10 expression, significantly reducing the proliferation and metastasis of DNMT2-deficient hepatocellular carcinoma cells. Furthermore, DNMT2 deletion was shown to significantly upregulate chemokine expression in tumors. Finally, we demonstrated that the NF-κB inhibitor Bortezomib further enhances DNMT2 deletion-induced apoptosis in hepatocellular carcinoma cells. This study reveals DNMT2's role in liver cancer and presents a new therapeutic target for future treatments.

01 Mar 2025·PRESSE MEDICALE

Kidney disease in multiple myeloma

Article

Author: Royal, Virginie ; Bridoux, Frank ; Nasr, Samih H ; Jaccard, Arnaud ; Leung, Nelson

Renal disease is a frequent complication of symptomatic multiple myeloma, that increases morbidity and reduces quality of life and overall survival. It may result from various lesions, the most frequent being light chain cast nephropathy (LCCN), related to precipitation of monoclonal free light chains (FLC) with uromodulin in distal tubules. Rapid identification of the type of kidney disease with appropriate management is key. LCCN typically reveals the underlying myeloma and manifests with severe acute kidney injury, high serum FLC level (>500 mg/l) and predominant light chain proteinuria (urine albumin/creatinine ratio <10 %). Urgent therapy is required, based on vigorous fluid expansion, correction of precipitating factors and introduction of efficient anti-myeloma therapy which choice should consider renal elimination of each agent and patient frailty. Early and deep reduction in serum FLC level conditions renal recovery, warranting assessment of efficacy by serial serum FLC level monitoring. In newly diagnosed patients, the combination of bortezomib, high-dose dexamethasone and an anti-CD38 monoclonal antibody is commonly used. The benefit to risk balance of quadruplets incorporating cyclophosphamide or an immunodulatory agent requires to be evaluated in prospective studies. In patients with severe acute kidney injury, reinforcing chemotherapy with FLC removal through plasma exchange or high-cutoff hemodialysis may increase the probability of renal response, despite controversial data from randomized trials. Histological assessment of the extent of cast formation and interstitial fibrosis/tubular atrophy may help evaluating renal prognosis and refining therapy. Thanks to improved overall survival, renal transplantation may be considered in selected candidates with end-stage kidney disease.

363

News (Medical) associated with Bortezomib

13 Jan 2025

·www.globenewswire.com

Press Release: Sarclisa obtains first approval in China for the treatment of adult patients with relapsed or refractory multiple myeloma

Sarclisa obtains first approval in China for the treatment of adult patients with relapsed or refractory multiple myeloma Approval based on positive pivotal ICARIA-MM phase 3 study using the China-based IsaFiRsT real-world study as bridging data, which demonstrated Sarclisa and the standard treatment Pd, improved responses and long-term outcomes compared to Pd alone in R/R MM patients Sarclisa-Pd is currently recommended by the Chinese Society of Clinical Oncology (CCSCO) and Chinese Anti-Cancer Association (CACA) treatment guidelines for this patient population Paris, January 13, 2025. The National Medical Products Administration (NMPA) in China has approved Sarclisa, an anti-CD38 medicine, in combination with pomalidomide and dexamethasone (Pd) for the treatment of adult patients with multiple myeloma (MM) who have received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor. This approval is based on results from the pivotal ICARIA-MM phase 3 study, using the China-based IsaFiRsT real-world study as bridging data. The ICARIA-MM study demonstrated Sarclisa in combination with Pd significantly reduced the risk of disease progression or death by 40% (HR 0.596, 95% CI 0.44-0.81, p=0.001), and resulted in a clinically meaningful, 6.9-month improvement in overall survival (OS) (HR=0.78; log-rank 1-sided P=0.0319), compared to Pd alone. Additionally, the IsaFiRsT study, which is the first real-world study for the registration of an anti-CD38 therapy in combination with Pd in China, showed an overall response rate (ORR) of 82.6% among relapsed or refractory multiple myeloma (R/R MM) adult patients. Olivier NatafGlobal Head, Oncology “This approval marks an important milestone for Sanofi in China. The results of the ICARIA-MM phase 3 study, coupled with the real-world IsaFiRsT study, highlight the benefit of Sarclisa for patients living with multiple myeloma and the importance of innovative regulatory pathways for timely access to different treatments. We look forward to continuing to build strong partnerships with the medical community, local companies, and authorities in China as we work to bring more innovative treatments to patients.” Through the Lecheng Pilot for real-world data application, the NMPA has increasingly used real-world evidence (RWE) to help accelerate the review and approval of innovative therapies and medical devices in China. Sarclisa was one of the first three treatments authorized for real-world studies as part of the pilot program and is the first blood cancer treatment approved based on RWE, in addition to clinical data. In addition to the NMPA approval, the Chinese Society of Clinical Oncology (CSCO) and Chinese Anti-Cancer Association (CACA) guidelines include Sarclisa-Pd as a "Category I Recommendation" and the "Preferred Option" for the treatment of patients with first-relapsed MM. Beyond R/R MM, a regulatory submission for Sarclisa in combination with bortezomib, lenalidomide and dexamethasone (VRd) for newly diagnosed multiple myeloma (NDMM) in adult patients not eligible for autologous stem cell transplant, is also under review in China with a final decision expected in the coming months. As one of the first multinational companies to enter China in 1982, Sanofi is committed to accelerating the introduction of innovative medicines and vaccines into China, aiming to transform the practice of medicine for the benefit of more Chinese people. About the ICARIA-MM studyICARIA-MM was a pivotal phase 3 randomized, open-label, multi-center trial evaluating Sarclisa in combination with Pd versus Pd alone in patients with R/R MM. The study enrolled 307 patients with R/R MM across 96 centers spanning 24 countries. Overall, patients had received a median of three prior lines of anti-myeloma therapies, including at least two consecutive cycles of lenalidomide and a proteasome inhibitor given alone or in combination. During the trial, Sarclisa was administered by intravenous infusion at a dose of 10mg/kg once weekly for four weeks, then every other week for 28-day cycles in combination with standard doses of Pd for the duration of treatment. The primary endpoint of ICARIA-MM was progression-free survival (PFS). Key secondary endpoints included ORR and OS. About the IsaFiRsT studyThe IsaFiRsT study was a single-arm, observational, prospective, real-world study evaluating Sarclisa in combination with Pd in patients with R/R MM. The study enrolled 24 patients with R/R MM at one site in China. Overall, patients received a median of three prior lines of therapy, including lenalidomide and a proteasome inhibitor, and had measurable serum or urine M-protein. During the trial, Sarclisa was administered through an intravenous infusion at a dose of 10mg/kg once weekly for four weeks, then every other week for 28-day cycles in combination with standard doses of Pd for the duration of treatment. Treatment continued until disease progression or unacceptable toxicity. The primary endpoint of IsaFiRsT was ORR. Key secondary endpoints included PFS, OS, duration of response (DOR) and safety. About SarclisaSarclisa (isatuximab) is a CD38 monoclonal antibody that binds to a specific epitope on the CD38 receptor on MM cells, inducing distinct antitumor activity. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a target for antibody-based therapeutics such as Sarclisa. In the US, the non-proprietary name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with nonproprietary naming of biological products guidance for industry issued by the US FDA. Currently, Sarclisa is approved in more than 50 countries, including the US and EU, across two indications; Sarclisa is approved under an additional indication in the US. Based on the ICARIA-MM phase 3 study, Sarclisa is approved in combination with Pd for the treatment of patients with R/R MM who have received ≥2 prior therapies, including lenalidomide and a proteasome inhibitor, and who progressed on last therapy. Based on the IKEMA phase 3 study, Sarclisa is also approved in 50 countries in combination with carfilzomib and dexamethasone, including in the US for the treatment of patients with R/R MM who have received 1–3 prior lines of therapy and in the EU for patients with MM who have received at least 1 prior therapy. In the US, Sarclisa is approved in combination with VRd as a front-line treatment option for adult patients with NDMM not eligible for transplant based on the IMROZ phase 3 study. On November 14, 2024, the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of Sarclisa-VRd in this patient population. A final decision is expected in the coming months. Sanofi continues to advance Sarclisa as part of a patient-centric clinical development program, which includes several phase 2 and phase 3 studies across the MM treatment continuum spanning six potential indications. In addition, the company is evaluating a subcutaneous administration method for Sarclisa in clinical studies. The safety and efficacy of Sarclisa has not been evaluated by any regulatory authority outside of its approved indications and methods of delivery. In striving to become the number one immunoscience company globally, Sanofi remains committed to advancing oncology innovation. Through focused strategic decisions the company has reshaped and prioritized its pipeline, leveraging its expertise in immunoscience to drive progress. Efforts are centered on difficult-to-treat often rare cancers such as select hematologic malignancies and solid tumors with critical unmet needs, including multiple myeloma, acute myeloid leukemia, certain types of lymphomas, as well as gastrointestinal and lung cancers. For more information on Sarclisa clinical studies, please visit www.clinicaltrials.gov. About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media RelationsSandrine Guendoul | +33 6 25 09 14 25 | sandrine.guendoul@sanofi.com Evan Berland | +1 215 432 0234 | evan.berland@sanofi.com Nicolas Obrist | +33 6 77 21 27 55 | nicolas.obrist@sanofi.com Léo Le Bourhis | +33 6 75 06 43 81 | leo.lebourhis@sanofi.com Victor Rouault | +33 6 70 93 71 40 | victor.rouault@sanofi.comTimothy Gilbert | +1 516 521 2929 | timothy.gilbert@sanofi.com Investor RelationsThomas Kudsk Larsen |+44 7545 513 693 | thomas.larsen@sanofi.com Alizé Kaisserian | +33 6 47 04 12 11 | alize.kaisserian@sanofi.comFelix Lauscher | +1 908 612 7239 | felix.lauscher@sanofi.com Keita Browne | +1 781 249 1766 | keita.browne@sanofi.comNathalie Pham | +33 7 85 93 30 17 | nathalie.pham@sanofi.comTarik Elgoutni | +1 617 710 3587 | tarik.elgoutni@sanofi.com Thibaud Châtelet | +33 6 80 80 89 90 | thibaud.chatelet@sanofi.com Sanofi forward-looking statementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates regarding the marketing and other potential of the product, or regarding potential future revenues from the product. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of the product, the fact that product may not be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related future litigation and the ultimate outcome of such litigation, and volatile economic and market conditions, and the impact that pandemics or other global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2023. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. All trademarks mentioned in this press release are the property of the Sanofi group. Attachment Press Release

Phase 3Clinical ResultDrug ApprovalImmunotherapyVaccine

13 Jan 2025

·www.globenewswire.com

New Sarclisa subcutaneous formulation met co-primary endpoints in the IRAKLIA phase 3 study in multiple myeloma

New Sarclisa subcutaneous formulation met co-primary endpoints in the IRAKLIA phase 3 study in multiple myeloma Sarclisa SC formulation added to Pd for the treatment of R/R MM met the co-primary endpoints in the IRAKLIA phase 3 study, demonstrating non-inferiority compared to Sarclisa IV IRAKLIA is the first global phase 3 study to evaluate the SC administration of a cancer treatment via an OBDS OBDS is an alternative delivery method designed to improve the patient experience and currently available SC administration January 9, 2025. Results from the investigational, randomized, open-label IRAKLIA phase 3 study demonstrated that Sarclisa administered at a fixed dose subcutaneously (SC) via an on-body delivery system (OBDS) in combination with pomalidomide and dexamethasone (Pd) met its co-primary endpoints of non-inferior objective response rate (ORR) and observed concentration before dosing (C trough) at steady state compared to intravenous (IV) Sarclisa administered at a weight-based dose in combination with Pd in patients with relapsed or refractory multiple myeloma (R/R MM). Key secondary endpoints, including very good partial response (VGPR), incidence rate of infusion reactions and C trough at cycle 2 were also achieved. The study is ongoing, and the full results will be presented at a forthcoming medical meeting. Sikander Ailawadhi, MD Professor of Medicine, Division of Hematology/Oncology at Mayo Clinic Florida and principal investigator of the study “The consistent overall response rate and comparable efficacy and safety profile observed in the IRAKLIA study for subcutaneous Sarclisa represent an exciting advancement, offering insight into a potential new administration option for patients. The results from IRAKLIA, in patients with relapsed or refractory multiple myeloma, support the potential of an on-body delivery system to help ease the delivery of a new formulation without impacting patient outcomes.” The IRAKLIA study was conducted using Enable Injections’ enFuse® hands-free OBDS, which was designed to administer high-volume medicines subcutaneously through an automated drug delivery technology. The enFuse device leverages a hidden and retractable needle that is thinner compared to commonly used SC injection needles. Houman Ashrafian, MD, PhD Executive Vice President, Head of Research and Development at Sanofi “We are fueled by our focus on innovation and finding best-in-class solutions to help ease the burden of disease for patients. The IRAKLIA study results are a prime example of what’s driving our scientific engine. Being able to possibly bring a novel option that helps reduce time in a healthcare facility is driven by our patient and provider-centric mindset. We look forward to sharing full results and working to bring this new advancement to the multiple myeloma community.” Additional studies evaluating Sarclisa SC formulations across different combinations and lines of therapy are ongoing. The safety and efficacy of Sarclisa SC and the enFuse device have not been evaluated by any regulatory authority outside of their approved indications. Regulatory submissions in the US and in the EU are planned during the first half of 2025. IRAKLIA is a randomized, open-label, pivotal phase 3 study evaluating the non-inferiority of Sarclisa SC formulation administered at a fixed dose subcutaneously via an OBDS versus weight-based dosed Sarclisa IV in combination with Pd in adult patients with R/R MM. The study enrolled 531 patients across 252 global sites, who were equally randomized to receive Sarclisa SC or IV in combination with Pd for 28-day cycles until disease progression, unacceptable adverse events (AEs), participant request to discontinue therapy or any other reason, whichever came first. In the SC arm, Sarclisa was administered at a fixed dose SC weekly for four weeks during the first cycle and every two weeks for subsequent cycles. In the IV arm, Sarclisa was administered at a weight-based dose via IV infusion weekly for four weeks during the first cycle and every two weeks for subsequent cycles. The study enrolled adult patients with MM who have received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor. The co-primary outcomes being assessed are ORR, defined as the proportion of patients with stringent complete response, complete response, VGPR, and partial response (PR) according to the 2016 IMWG criteria assessed by Independent Review Committee (IRC), and observed C trough at steady state, defined as observed Sarclisa plasma concentrations. Based in the US (Cincinnati, Ohio), Enable Injections is a global healthcare innovation company committed to improving the patient treatment experience through the development and manufacturing of enFuse. enFuse is an innovative wearable drug delivery platform that is designed to deliver large volumes of pharmaceutical and biologic therapeutics via subcutaneous administration, with the aim of improving convenience, supporting superior outcomes, and advancing healthcare system economics. For more information, visit https://enableinjections.com. Sarclisa (isatuximab) is a CD38 monoclonal antibody that binds to a specific epitope on the CD38 receptor on MM cells, inducing distinct antitumor activity. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a target for antibody-based therapeutics such as Sarclisa. In the US, the non-proprietary name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with nonproprietary naming of biological products guidance for industry issued by the US FDA. Currently, Sarclisa is approved in more than 50 countries, including the US and EU, across two indications; Sarclisa is approved under an additional indication in the US. Based on the ICARIA-MM phase 3 study, Sarclisa is approved in combination with Pd for the treatment of patients with R/R MM who have received ≥2 prior therapies, including lenalidomide and a proteasome inhibitor, and who progressed on last therapy. Based on the IKEMA phase 3 study, Sarclisa is also approved in 50 countries in combination with carfilzomib and dexamethasone, including in the US for the treatment of patients with R/R MM who have received 1–3 prior lines of therapy and in the EU for patients with MM who have received at least 1 prior therapy. In the US, Sarclisa is approved in combination with bortezomib, lenalidomide, and dexamethasone (VRd) as a front-line treatment option for adult patients with newly diagnosed multiple myeloma (NDMM) who are not eligible for autologous stem cell transplant (ASCT), based on the IMROZ phase 3 study. On November 14, 2024, the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of Sarclisa-VRd in this patient population. A final decision is expected in the coming months. Sanofi continues to advance Sarclisa as part of a patient-centric clinical development program, which includes several phase 2 and phase 3 studies across the MM treatment continuum spanning six potential indications. Further clinical studies evaluating a subcutaneous administration method for Sarclisa are ongoing. In striving to become the number one immunoscience company globally, Sanofi remains committed to advancing oncology innovation. Through focused strategic decisions the company has reshaped and prioritized its pipeline, leveraging its expertise in immunoscience to drive progress. Efforts are centered on difficult-to-treat often rare cancers such as select hematologic malignancies and solid tumors with critical unmet needs, including multiple myeloma, acute myeloid leukemia, certain types of lymphomas, as well as gastrointestinal and lung cancers. We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultDrug ApprovalImmunotherapy

09 Jan 2025

·pipelinereview.com

New Sarclisa subcutaneous formulation met co-primary endpoints in the IRAKLIA phase 3 study in multiple myeloma

PARIS, France I January 9, 2025 I Results from the investigational, randomized, open-label IRAKLIA phase 3 study demonstrated that Sarclisa administered at a fixed dose subcutaneously (SC) via an on-body delivery system (OBDS) in combination with pomalidomide and dexamethasone (Pd) met its co-primary endpoints of non-inferior objective response rate (ORR) and observed concentration before dosing (C trough) at steady state compared to intravenous (IV) Sarclisa administered at a weight-based dose in combination with Pd in patients with relapsed or refractory multiple myeloma (R/R MM). Key secondary endpoints, including very good partial response (VGPR), incidence rate of infusion reactions and C trough at cycle 2 were also achieved. The study is ongoing, and the full results will be presented at a forthcoming medical meeting. Sikander Ailawadhi, MD Professor of Medicine, Division of Hematology/Oncology at Mayo Clinic Florida and principal investigator of the study “The consistent overall response rate and comparable efficacy and safety profile observed in the IRAKLIA study for subcutaneous Sarclisa represent an exciting advancement, offering insight into a potential new administration option for patients. The results from IRAKLIA, in patients with relapsed or refractory multiple myeloma, support the potential of an on-body delivery system to help ease the delivery of a new formulation without impacting patient outcomes.” The IRAKLIA study was conducted using Enable Injections’ enFuse ® hands-free OBDS, which was designed to administer high-volume medicines subcutaneously through an automated drug delivery technology. The enFuse device leverages a hidden and retractable needle that is thinner compared to commonly used SC injection needles. Houman Ashrafian, MD, PhD Executive Vice President, Head of Research and Development at Sanofi “We are fueled by our focus on innovation and finding best-in-class solutions to help ease the burden of disease for patients. The IRAKLIA study results are a prime example of what’s driving our scientific engine. Being able to possibly bring a novel option that helps reduce time in a healthcare facility is driven by our patient and provider-centric mindset. We look forward to sharing full results and working to bring this new advancement to the multiple myeloma community.” Additional studies evaluating Sarclisa SC formulations across different combinations and lines of therapy are ongoing. The safety and efficacy of Sarclisa SC and the enFuse device have not been evaluated by any regulatory authority outside of their approved indications. Regulatory submissions in the US and in the EU are planned during the first half of 2025. About the IRAKLIA study IRAKLIA is a randomized, open-label, pivotal phase 3 study evaluating the non-inferiority of Sarclisa SC formulation administered at a fixed dose subcutaneously via an OBDS versus weight-based dosed Sarclisa IV in combination with Pd in adult patients with R/R MM. The study enrolled 531 patients across 252 global sites, who were equally randomized to receive Sarclisa SC or IV in combination with Pd for 28-day cycles until disease progression, unacceptable adverse events (AEs), participant request to discontinue therapy or any other reason, whichever came first. In the SC arm, Sarclisa was administered at a fixed dose SC weekly for four weeks during the first cycle and every two weeks for subsequent cycles. In the IV arm, Sarclisa was administered at a weight-based dose via IV infusion weekly for four weeks during the first cycle and every two weeks for subsequent cycles. The study enrolled adult patients with MM who have received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor. The co-primary outcomes being assessed are ORR, defined as the proportion of patients with stringent complete response, complete response, VGPR, and partial response (PR) according to the 2016 IMWG criteria assessed by Independent Review Committee (IRC), and observed C trough at steady state, defined as observed Sarclisa plasma concentrations. About Enable Injections Based in the US (Cincinnati, Ohio), Enable Injections is a global healthcare innovation company committed to improving the patient treatment experience through the development and manufacturing of enFuse. enFuse is an innovative wearable drug delivery platform that is designed to deliver large volumes of pharmaceutical and biologic therapeutics via subcutaneous administration, with the aim of improving convenience, supporting superior outcomes, and advancing healthcare system economics. For more information, visit https://enableinjections.com . About Sarclisa Sarclisa (isatuximab) is a CD38 monoclonal antibody that binds to a specific epitope on the CD38 receptor on MM cells, inducing distinct antitumor activity. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a target for antibody-based therapeutics such as Sarclisa. In the US, the non-proprietary name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with nonproprietary naming of biological products guidance for industry issued by the US FDA. Currently, Sarclisa is approved in more than 50 countries, including the US and EU, across two indications; Sarclisa is approved under an additional indication in the US. Based on the ICARIA-MM phase 3 study, Sarclisa is approved in combination with Pd for the treatment of patients with R/R MM who have received ≥2 prior therapies, including lenalidomide and a proteasome inhibitor, and who progressed on last therapy. Based on the IKEMA phase 3 study, Sarclisa is also approved in 50 countries in combination with carfilzomib and dexamethasone, including in the US for the treatment of patients with R/R MM who have received 1–3 prior lines of therapy and in the EU for patients with MM who have received at least 1 prior therapy. In the US, Sarclisa is approved in combination with bortezomib, lenalidomide, and dexamethasone (VRd) as a front-line treatment option for adult patients with newly diagnosed multiple myeloma (NDMM) who are not eligible for autologous stem cell transplant (ASCT), based on the IMROZ phase 3 study. On November 14, 2024, the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of Sarclisa-VRd in this patient population. A final decision is expected in the coming months. Sanofi continues to advance Sarclisa as part of a patient-centric clinical development program, which includes several phase 2 and phase 3 studies across the MM treatment continuum spanning six potential indications. Further clinical studies evaluating a subcutaneous administration method for Sarclisa are ongoing. In striving to become the number one immunoscience company globally, Sanofi remains committed to advancing oncology innovation. Through focused strategic decisions the company has reshaped and prioritized its pipeline, leveraging its expertise in immunoscience to drive progress. Efforts are centered on difficult-to-treat often rare cancers such as select hematologic malignancies and solid tumors with critical unmet needs, including multiple myeloma, acute myeloid leukemia, certain types of lymphomas, as well as gastrointestinal and lung cancers. For more information on Sarclisa clinical studies, please visit www.clinicaltrials.gov . About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY SOURCE: Sanofi

Phase 3Clinical ResultDrug ApprovalImmunotherapy

100 Deals associated with Bortezomib

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KEGG	Wiki	ATC	Drug Bank
D03150	Bortezomib	L01XX32	DB00188

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Immunoglobulin Light-Chain Amyloidosis	JP	Janssen Pharmaceutical KK	20 Oct 2006
Waldenstrom Macroglobulinemia	JP	Janssen Pharmaceutical KK	20 Oct 2006
Mantle-Cell Lymphoma	EU	Janssen-Cilag International NV	26 Apr 2004
Mantle-Cell Lymphoma	IS	Janssen-Cilag International NV	26 Apr 2004
Mantle-Cell Lymphoma	LI	Janssen-Cilag International NV	26 Apr 2004
Mantle-Cell Lymphoma	NO	Janssen-Cilag International NV	26 Apr 2004
Multiple Myeloma	US	Takeda Pharmaceuticals U.S.A., Inc.	13 May 2003

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Diffuse large B-cell lymphoma recurrent	Phase 3	IT	Janssen-Cilag Ltd.	04 Feb 2013
Diffuse large B-cell lymphoma recurrent	Phase 3	IT	Janssen Pharmaceutica NV	04 Feb 2013
Diffuse large B-cell lymphoma refractory	Phase 3	IT	Janssen Pharmaceutica NV	04 Feb 2013
Diffuse large B-cell lymphoma refractory	Phase 3	IT	Janssen-Cilag Ltd.	04 Feb 2013
Plasma Cell Leukemia	Phase 3	CN	Janssen Scientific Affairs LLC	13 Jan 2012
Plasma Cell Leukemia	Phase 3	AU	Janssen Scientific Affairs LLC	13 Jan 2012
Plasma Cell Leukemia	Phase 3	KR	Janssen Scientific Affairs LLC	13 Jan 2012
Diffuse Large B-Cell Lymphoma	Phase 3	CH	Janssen-Cilag Ltd.	01 Apr 2011
Diffuse Large B-Cell Lymphoma	Phase 3	GB	Janssen-Cilag Ltd.	01 Apr 2011
B-Cell Lymphoma	Phase 3	US	Millennium Pharmaceuticals, Inc.	01 Mar 2006

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05564052 (VEGA, CTgov)	Phase 2	Mantle-Cell Lymphoma	36	Ibrutinib+Rituximab (Arm A1: Ibrutinib 560 mg + Rituximab 375 mg/m^2)	uzdvlowwem(wrmrohugtz) = mhcywypnvk btdppuzldb (baucbzhcyg, xokgynvash - oegvcxyntk) View more	-	13 Jan 2025
				Ibrutinib+Rituximab (Arm A2: Ibrutinib 420 mg + Rituximab 375 mg/m^2)	uzdvlowwem(wrmrohugtz) = bdpziihwrf btdppuzldb (baucbzhcyg, pgjgfuggvl - fkahqnduka) View more
NCT04246047 (DREAMM-7, ASH2024) Manual	Phase 3	Multiple Myeloma	494	Belantamab mafodotin, bortezomib, and dexamethasone (BVd)	smatwegbhi(fsrtoikvrq) = uwlnokhnzq yyasytmyqu (eemzhwsarw, 28.4 - NR) Met View more	Positive	09 Dec 2024
				Daratumumab, bortezomib, and dexamethasone (DVd)	smatwegbhi(fsrtoikvrq) = lizbuuwggm yyasytmyqu (eemzhwsarw, 11.1 - 17.5) Met View more
NCT03319667 (IMROZ, ASH2024) Manual	Phase 3	Multiple Myeloma First line	446	Isatuximab, Bortezomib, Lenalidomide, and Dexamethasone (Isa-VRd)	ymibcrwqnl(paooartuyo) = wqiqowiqjo bqdvbmkaix (cacjzdyfvn ) View more	Positive	09 Dec 2024
				Bortezomib, Lenalidomide, and Dexamethasone (VRd)	ymibcrwqnl(paooartuyo) = oviczssxdm bqdvbmkaix (cacjzdyfvn ) View more
NCT03620903 (ECWM-2, ASH2024) Manual	Phase 2	Waldenstrom Macroglobulinemia First line	53	Bortezomib+Ibrutinib+Rituximab	qvloutscwk(yceqwmyqip) = adomjkarwv sfscvfyuyf (glrqdxuops ) View more	Positive	09 Dec 2024
ASH2024	Not Applicable	Multiple Myeloma	-	Pomalidomide, Bortezomib, Dexamethasone	tflavslcbq(njnehxpqrs) = vgvclpwakn fkitjwtqfq (sfaogmobyc ) View more	-	09 Dec 2024
ASH2024	Not Applicable	Multiple Myeloma	-	Ixazomib-lenalidomide-dexamethasone (IRd)	ywykytdril(asnterweqt) = Any grade TEAEs reported in ≥25% of pts were diarrhea (26%) in the 1−3 cycles group; PN not elsewhere classified (NEC; 34%), fatigue (34%), diarrhea (34%), and edema NEC (26%) in the 4−9 cycles group; and diarrhea (68%), PN NEC (53%), fatigue (42%), edema NEC (38%), arthralgia (35%), nausea (33%), and back pain (29%) in the >9 cycles group. pctyratnud (glzeewzoes ) View more	-	09 Dec 2024
NCT03617731 (GMMG-HD7, ASH2024)	Phase 3	Multiple Myeloma CD38	662	Isatuximab, Lenalidomide, Bortezomib, Dexamethasone	mxxnygtmdo(nupwrpriiy) = cyhculfxrb cuqtnfdbik (oganvxdpuh, 95% CI 0.52 - 0.94) View more	Positive	09 Dec 2024
				Lenalidomide, Bortezomib, Dexamethasone	mxxnygtmdo(nupwrpriiy) = erzpjrmhvm cuqtnfdbik (oganvxdpuh, 95% CI 46 - 48) View more
CASTOR (ASH2024)	Phase 3	Multiple Myeloma	-	Daratumumab/bortezomib/dexamethasone (DVd)	dwhlcnmfxo(jflzrfzslu) = yursymmoyz kqgjadreuo (nqacsunaas, 48.5 - 56.2) View more	Positive	08 Dec 2024
ASH2024	Not Applicable	Multiple Myeloma	-	Daratumumab, Lenalidomide, Bortezomib, Dexamethasone (D-RVd)	rwbcmdbraw(yjjelvnxbo) = 82% lwuurlbbud (ntxcpesmss ) View more	-	08 Dec 2024
ASH2024	Not Applicable	Multiple Myeloma	-	Daratumumab, Bortezomib, Lenalidomide, and Dexamethasone (DVRd)	mkkgcqyzje(xbgjdrkddv) = trygzuzktl tcwuswlcxa (dbyxsntbeo ) View more	-	08 Dec 2024
				DVRd-DR	mkkgcqyzje(xbgjdrkddv) = ddamneykus tcwuswlcxa (dbyxsntbeo ) View more

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