A non-interventional study of melphalan flufenamide (melflufen, Pepaxti) in combination with dexamethasone in patients with relapsed and/or refractory multiple myeloma (RRMM) according to the approved label. - OP-113 / HARBOUR

Start Date16 Jul 2024

Sponsor / Collaborator

Oncopeptides AB

EUCTR2020-004349-35-DK

/ Not yet recruitingPhase 2IIT

Melphalan flufenamide (melflufen) and Dexamethasone re-induction in Daratumumab-refractory Multiple Myeloma as an Adjunct to Continued Daratumumab (MERMAID) - MERMAID

Start Date02 Feb 2021

Sponsor / Collaborator-

NCT04649060

/ TerminatedPhase 3

A Randomized, Controlled, Open-Label Phase 3 Study of Melflufen in Combination With Daratumumab Compared With Daratumumab in Patients With Relapsed or Relapsed-Refractory Multiple Myeloma

This was a randomized, controlled, open-label, Phase 3 multicenter study which enrolled patients with Relapsed-Refractory Multiple Myeloma (RRMM) who were either double refractory to an Immunomodulatory Drug (IMiD) and a Proteasome Inhibitor (PI) (regardless of the number of prior lines of therapy), or had received at least 3 prior lines of therapy including an IMiD and a PI.
Patients received treatment with melflufen+dexamethasone+daratumumab or daratumumab until documented progressive disease, unacceptable toxicity, or patient/treating physician decision. Patients in the daratumumab treatment arm had the option to receive treatment with melflufen+dexamethasone+daratumumab after confirmed progressive disease.

Start Date21 Dec 2020

Sponsor / Collaborator

Oncopeptides AB

100 Clinical Results associated with Melphalan flufenamide hydrochloride

100 Translational Medicine associated with Melphalan flufenamide hydrochloride

100 Patents (Medical) associated with Melphalan flufenamide hydrochloride

Literatures (Medical) associated with Melphalan flufenamide hydrochloride

01 Jun 2025·EUROPEAN JOURNAL OF HAEMATOLOGY

Outcomes of Melflufen Treatment in Patients With Relapsed/Refractory Multiple Myeloma

Article

Author: O'Neill, Kathleen ; Richardson, Paul G. ; Patches, Sarah ; Goodrich, Kathryn ; Laubach, Jacob ; Leblebjian, Houry ; Regan, Eileen ; Hossain, Shahrier ; Noonan, Kim ; Mo, Clifton

ABSTRACT:

Objective:

Melphalan flufenamide (melflufen) plus dexamethasone is fully approved in Europe for patients with relapsed/refractory multiple myeloma (RRMM) with ≥ 3 prior lines of therapy. We analyzed the efficacy and safety of melflufen in the real‐world setting.

Methods:

In this retrospective analysis, we examined baseline features, efficacy, and safety outcomes with melflufen plus dexamethasone in a cohort of 12 patients with heavily pre‐treated RRMM at the Dana‐Farber Cancer Institute, USA.

Results:

Patients had received a median of 5.5 prior lines of therapy. Three patients (25%) had extramedullary disease, three (25%) cytogenetically high‐risk features, and five (42%) had received prior autologous stem cell transplantation. The overall response rate was 55% (complete response: three [27%], very good partial response: one [9%], partial response: two [18%] patients). Five patients (42%) had stable disease; one was non‐evaluable. Adverse events (AEs) were mostly hematologic and proved manageable; two patients had Grade 2 infections. Reasons for melflufen discontinuation were progressive disease (42%), drug withdrawal from the United States market (33%), AEs (17%), and sudden death (8%) unrelated to treatment.

Conclusions:

Consistent with clinical trial data, melflufen had an expected safety profile with manageable toxicity and clinically meaningful efficacy in patients with RRMM treated in the real‐world setting.

12 Dec 2024·NEW ENGLAND JOURNAL OF MEDICINE

Autopsy of a Drug Withdrawal — The Case of Melphalan Flufenamide

Article

Author: Gormley, Nicole ; Carioti, Theresa ; Pazdur, Richard ; Kanapuru, Bindu

The accelerated approval (AA) pathway, which was established in 1992, has expedited approvals of drugs for serious and life-threatening diseases, with decisions being made on the basis of an intermediate clin. end point or a surrogate end point that is reasonably likely to predict clin. benefit.When confirmatory trials don't verify a drug's clin. benefit or there are other legal grounds for withdrawal, the Food and Drug Administration (FDA) may withdraw the approval.

01 Feb 2024·Journal of clinical pharmacology

Pharmacokinetics and Metabolism of Melflufen, an Alkylating Peptide–Drug Conjugate, in Patients with Relapsed Refractory Multiple Myeloma

Article

Author: Gullbo, Joachim ; Pekar, David ; Sjöberg, Per ; Huledal, Gunilla ; Kawakatsu, Sonoko ; Jerling, Markus ; Norin, Stefan ; Ruiz-Garcia, Ana ; Wang, Xiaoning

Abstract:

Melphalan flufenamide (melflufen) is a novel lipophilic peptide–drug conjugate recently approved in the European Union and the United Kingdom for the treatment of relapsed refractory multiple myeloma. Melflufen rapidly crosses the cell membrane, and inside tumor cells, melflufen utilizes peptidases and esterases to release entrapped hydrophilic metabolites with alkylating activity. In vitro, in whole blood, melflufen was rapidly distributed into blood cells and quickly converted to its main metabolite melphalan, with maximum cellular concentrations of noncovalently bound melflufen and melphalan after 1 and 6 minutes, respectively. Melphalan outflow from blood cells was slow, with peak concentrations in plasma after 25 minutes. The pharmacokinetics of melflufen was best described by a 2‐compartment model. Following a 30‐minutes intravenous infusion of 40 mg in 27 patients with relapsed refactory multiple myeloma, mean half‐life in the α phase of the curve was 1.24 minutes, half‐life in the β phase of the curve 26.7 minutes, and clearance 13.4 L/min. Desethyl‐melflufen exposure was below 20% compared to melflufen. Based on population analysis (298 patients with relapsed refactory multiple myeloma), the melphalan pharmacokinetics were well characterized by a 3‐compartment model with melflufen dosing into a peripheral compartment, assuming instantaneous distribution of melflufen into cells and subsequent rapid metabolism to melphalan. Mean clearance and central and deep peripheral volumes of distribution were 22.4 L/h, 2.70 L, and 51.3 L, respectively. Clearance increased and maximum concentration decreased with increasing body weight and estimated glomerular filtration rate. In conclusion, melflufen administration differs from melphalan administration by a more rapid distribution into cells, which, in conjunction with a rapid intracellular metabolism, allows for higher maximum concentrations of alkylating agents, and by a more extensive distribution of melphalan to peripheral tissues.

News (Medical) associated with Melphalan flufenamide hydrochloride

04 Feb 2025

·www.prnewswire.com

Global Peptide-Drug Conjugate Market to Show Enormous Growth at a CAGR of ~29% by 2032 | DelveInsight

The increasing global prevalence of cancer is fueling the demand for peptide-drug conjugates as targeted therapies, providing improved effectiveness and lower systemic toxicity than conventional treatments. Additionally, the expanding use of peptide-based therapeutics, driven by their enhanced treatment outcomes, biocompatibility, and precise tumor-targeting capabilities, is further propelling market growth. LAS VEGAS , Feb. 4, 2025 /PRNewswire/ -- DelveInsight's Peptide-drug Conjugate Market Insights report provides the current and forecast market analysis, individual leading peptide-drug conjugate companies' market shares, challenges, peptide-drug conjugate market drivers, barriers, trends, and key market peptide-drug conjugate companies in the market. Key Takeaways from the Peptide-drug Conjugate Market Report As per DelveInsight estimates, North America is anticipated to dominate the global peptide-drug conjugate market during the forecast period. The LUTATHERA (lutetium Lu 177 dotatate) category in the peptide drug conjugates market dominated with a market share of 37% in 2024. Notable peptide-drug conjugate companies such as Novartis AG, Oncopeptides AB, Cybrexa Therapeutics, Avacta Therapeutics, ProteinQure, Theratechnologies Inc., CBP, Soricimed Biopharma, NMS Group S.p.A., Mainline Scientific LLC., Bicycle Therapeutics, and several others, are currently operating in the peptide-drug conjugate market. In April 2024, PeptiDream Inc. announced the expansion of its peptide discovery collaboration with Novartis Pharma AG. Using its proprietary Peptide Discovery Platform System (PDPS®), PeptiDream identified and optimized macrocyclic peptides for conjugation with radionuclides for therapeutic and diagnostic applications. In March 2023, Ono Pharmaceutical Co., Ltd. announced that it entered into a drug discovery collaboration agreement with PeptiDream Inc., to discover and develop novel macrocyclic constrained peptide drugs against multiple targets of Ono's interest. To read more about the latest highlights related to the PDC market, get a snapshot of the key highlights entailed in the Global Peptide-drug Conjugate Market Report Peptide-drug Conjugate Overview Peptide-drug conjugates (PDCs) are an emerging class of targeted therapeutics that combine the specificity of peptides with the potent efficacy of cytotoxic drugs. These conjugates leverage peptides as targeting ligands to deliver drugs selectively to diseased cells, such as cancerous or infected tissues, thereby minimizing off-target toxicity. The design of PDCs involves three key components: a targeting peptide, a linker, and a payload (therapeutic drug). The peptide is typically selected based on its ability to recognize and bind to overexpressed receptors on diseased cells, ensuring precise drug delivery. The linker plays a crucial role in controlling drug release, which can be triggered by specific conditions like enzymatic cleavage or pH changes in the tumor microenvironment. Compared to antibody-drug conjugates (ADCs), PDCs offer advantages such as smaller size, better tissue penetration, and lower immunogenicity. They are particularly promising for targeting intracellular pathways, where antibodies may struggle to enter. Advances in peptide engineering, including modifications to enhance stability and binding affinity, have further improved the efficacy of PDCs in preclinical and clinical settings. PDCs are being explored in various therapeutic areas, including oncology, infectious diseases, and metabolic disorders. As research progresses, optimizing peptide selection, linker chemistry, and payload potency will be critical in developing next-generation PDCs with superior safety and efficacy profiles. Peptide-drug Conjugate Market Insights North America dominated the global peptide drug conjugates market in 2024, accounting for the largest share of 56.70%. The market is expected to grow at a CAGR of 28.47% from 2025 to 2032, primarily driven by the increasing incidence of cancer in the U.S. Additionally, the region's market growth is fueled by rising collaborations among key industry players for PDC development and increasing investments in this sector. Furthermore, major companies in North America are emphasizing research and development to create innovative peptide-drug conjugates. For instance, in February 2021, Theratechnologies Inc. announced that the U.S. FDA granted fast-track designation to TH1902, a docetaxel-based peptide-drug conjugate, for treating patients with sortilin-positive recurrent advanced solid tumors that are resistant to standard therapies. In addition, in April 2024, Novartis, which already has two FDA-approved PDC drugs, expanded its collaboration with PeptiDream to further develop peptide-drug conjugates in a deal exceeding USD 2.8 billion. These factors collectively contribute to the anticipated growth of the peptide drug conjugates market in the region. To know more about why North America is leading the market growth in the PDC market, get a snapshot of the Peptide-drug Conjugate Market Outlook Peptide-drug Conjugate Market Dynamics The peptide-drug conjugate market is witnessing significant growth, driven by advancements in targeted therapies and the increasing prevalence of cancer and other chronic diseases. PDCs combine the specificity of peptides with the potency of cytotoxic drugs, offering an improved therapeutic index compared to traditional chemotherapy. With growing interest in precision medicine, pharmaceutical companies are heavily investing in PDC research and development, leading to an expanding pipeline of clinical candidates. One of the key market drivers is the rising demand for novel drug delivery systems that enhance efficacy while minimizing off-target effects. Peptides, due to their high specificity and low immunogenicity, serve as excellent carriers for cytotoxic payloads. Additionally, advancements in peptide synthesis and linker technologies have improved the stability and half-life of PDCs, making them more viable for clinical applications. Companies such as Bicycle Therapeutics and PeptiDream are leading the innovation in this space. Despite the promising outlook, the PDC market faces challenges such as manufacturing complexities, high development costs, and regulatory hurdles. Peptide synthesis and conjugation processes require stringent quality control, and scalability remains a concern. Furthermore, competition from other targeted therapies, such as ADCs and small-molecule inhibitors, poses a challenge for widespread adoption. Looking ahead, growth opportunities exist in expanding indications beyond oncology, such as antimicrobial, autoimmune, and neurodegenerative diseases. Strategic collaborations between biotech firms and large pharmaceutical companies are accelerating clinical development, while advancements in artificial intelligence and computational modeling are further refining peptide design and optimization. With ongoing innovations and increasing investments, the PDC market is poised for substantial expansion in the coming years. Get a sneak peek at the PDC market dynamics @ Peptide-drug Conjugate Market Dynamics Analysis Peptide-drug Conjugate Market Assessment Peptide-drug Conjugate Market Segmentation Peptide-drug Conjugate Market Segmentation By Product: LUTATHERA (lutetium Lu 177 dotatate), PEPAXTI (Melphalan flufenamide), and PLUVICTO (lutetium Lu 177 vipivotide tetraxetan) Peptide-drug Conjugate Market Segmentation By Cancer Type: Prostate Cancer, Gastrointestinal Cancer, Multiple Myeloma Peptide-drug Conjugate Market Segmentation By Geography: North America, Europe, Asia-Pacific, and Rest of World Porter's Five Forces Analysis, Product Profiles, Case Studies, KOL's Views, Analyst's View Which MedTech key players in the PDC market are set to emerge as the trendsetter explore @ Peptide-drug Conjugate Companies Table of Contents Interested in knowing the PDC market by 2032? Click to get a snapshot of the Peptide-drug Conjugate Market Trends Related Reports Antibody-drug Conjugates in Oncology Competitive Landscape Antibody-drug Conjugates in Oncology Competitive Landscape – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ADC in oncology companies, including Jiangsu Hengrui Medicine Co., Bio-Thera Solutions, MediLink Therapeutics, Byondis, AbbVie, Oxford BioTherapeutics, Iksuda Therapeutics, Novelty Nobility, LegoChem Biosciences, DualityBio, Lepu Biopharma, Ambrx, CSPC ZhongQi Pharmaceutical Technology, MacroGenics, ADC Therapeutics, Genor Biopharma, CStone Pharmaceuticals, AstraZeneca, Alteogen, Biocity Biopharmaceutics, among others. Antibody-drug Conjugates Competitive Landscape Antibody-drug Conjugates Competitive Landscape – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ADC companies, including Sorrento Therapeutics, Ambrx, MacroGenics, SOTIO Biotech, Klus Pharma, BioAtla, BiOneCure Therapeutics, Shanghai Miracogen, ProfoundBio, ImmunoGen, Arcus Biosciences, CytomX Therapeutics, RemeGen, Bliss Biopharmaceutical, Aivita Biomedical, CoImmune, Zenith Epigenetics, Sutro Biopharma, Ambrx, Mythic Therapeutics, Shanghai Fudan-Zhangjiang Bio-Pharmaceutical, Byondis, MediLink Therapeutics (Suzhou), Oncomatryx Biopharma, MacroGenics, Shanghai Miracogen, Mirati Therapeutics, Ambrx, Orum Therapeutics, Silverback Therapeutics, Shanghai Fudan-Zhangjiang Bio-Pharmaceutical, Aivita Biomedical, Northwest Biotherapeutics, Daiichi Sankyo, Merck, Pfizer, GSK, AstraZeneca, Eisai, FOSUN PHARMA, LaNova, Mabwell Therapeutics, MBRACE THERAPEUTICS, MINGHUSI PHARMACEUTICALS, BioNTech , Bio-Thera, Corbus Pharmaceuticals, AbbVie, Multitude Therapeutics, Innovent, OnCusp Therapeutics, Simcere, Ymmunobio, Jiangsu Hengrui Medicine, among others. Antibody-drug Conjugates Market Antibody Drug Conjugates Market Size, Target Population, Competitive Landscape & Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key ADC companies, including Sorrento Therapeutics, Ambrx, MacroGenics, SOTIO Biotech, Klus Pharma, BioAtla, BiOneCure Therapeutics, Shanghai Miracogen, ProfoundBio, ImmunoGen, Arcus Biosciences, CytomX Therapeutics, RemeGen, Bliss Biopharmaceutical, Aivita Biomedical, CoImmune, Zenith Epigenetics, Sutro Biopharma, Ambrx, Mythic Therapeutics, Shanghai Fudan-Zhangjiang Bio-Pharmaceutical, Byondis, MediLink Therapeutics (Suzhou), Oncomatryx Biopharma, MacroGenics, Shanghai Miracogen, Mirati Therapeutics, Ambrx, Orum Therapeutics, Silverback Therapeutics, Shanghai Fudan-Zhangjiang Bio-Pharmaceutical, Aivita Biomedical, Northwest Biotherapeutics, Daiichi Sankyo, Merck, Pfizer, GSK, AstraZeneca, Eisai, FOSUN PHARMA, LaNova, Mabwell Therapeutics, MBRACE THERAPEUTICS, MINGHUSI PHARMACEUTICALS, BioNTech , Bio-Thera, Corbus Pharmaceuticals, AbbVie, Multitude Therapeutics, Innovent, OnCusp Therapeutics, Simcere, Ymmunobio, Jiangsu Hengrui Medicine, among others. Bispecific Antibody Competitive Landscape Bispecific Antibody Competitive Landscape – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key bispecific antibodies companies, including Janssen, Amgen, Akeso, Zymeworks, Roche, IGM Biosciences, MacroGenics, Provention Bio, Jiangsu Alphamab Biopharmaceuticals, Sichuan Baili Pharmaceutical, Regeneron Pharmaceuticals, Boehringer Ingelheim, among others. About DelveInsight DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Fast TrackPDC

09 Aug 2024

·www.fda.gov

WITHDRAWN: FDA grants accelerated approval to melphalan flufenamide for relapsed or refractory multiple myeloma

UPDATE: On February 23, 2024, the FDA announced its final decision to withdraw approval of melphalan flufenamide (Pepaxto) with dexamethasone to treat certain patients with multiple myeloma. The agency determined the grounds for withdrawal were met because: (1) the confirmatory study conducted as a condition of accelerated approval did not confirm Pepaxto’s clinical benefit, and (2) the available evidence demonstrates that Pepaxto is not shown to be safe or effective under its conditions of use. For further information, see the CDER Alert. On February 26, 2021, the Food and Drug Administration granted accelerated approval to melphalan flufenamide (Pepaxto, Oncopeptides AB) in combination with dexamethasone for adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD-38 directed monoclonal antibody. Efficacy was evaluated in HORIZON (NCT02963493), a multicenter, single-arm trial. Eligible patients were required to have relapsed refractory multiple myeloma. Patients received melphalan flufenamide 40 mg intravenously on day 1 and dexamethasone 40 mg orally (20 mg for patients ≥75 years of age) on day 1, 8, 15 and 22 of each 28-day cycle until disease progression or unacceptable toxicity. Efficacy was evaluated in a subpopulation of 97 patients who received four or more prior lines of therapy and were refractory to at least one proteasome inhibitor, one immunomodulatory agent, and a CD38-directed antibody. The main efficacy outcome measure was overall response rate (ORR) and duration of response (DOR) assessed by investigators according to the International Myeloma Working Group (IMWG) Criteria. The ORR was 23.7% (95% CI: 15.7, 33.4) and median DOR 4.2 months (95% CI: 3.2, 7.6). Safety was evaluated in the 157 patients enrolled in HORIZON. Most common adverse reactions (> 20%) are fatigue, nausea, diarrhea, pyrexia and respiratory tract infection. Most common laboratory abnormalities (≥50%) are decreased leukocytes, platelets, lymphocytes, neutrophils, and hemoglobin, and increased creatinine. The safety and efficacy of melphalan flufenamide has not been established for use as a conditioning regimen in patients receiving transplant. The USPI includes Limitations of Use statement that melphalan flufenamide is not indicated and is not recommended for use as a conditioning regimen for transplant outside of controlled clinical trials. The recommended dose of melphalan flufenamide is 40 mg intravenously over 30 minutes on day 1 of each 28-day treatment cycle, in combination with dexamethasone. View full prescribing information for Pepaxto. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). This application was granted priority review and orphan drug designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088. For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov. For information on the COVID-19 pandemic, see the following resources: FDA: Coronavirus Disease 2019 (COVID-19) NCI: Coronavirus: What People With Cancer Should Know CDC: Coronavirus (COVID-19) Follow the Oncology Center of Excellence on Twitter @FDAOncologyExternal Link Disclaimer. Content current as of: 02/23/2024 Regulated Product(s) Drugs Prescription Drugs 02/23/2024 Drugs Prescription Drugs Resources for Information | Approved Drugs Oncology (Cancer)/Hematologic Malignancies Approval Notifications Ongoing | Cancer Accelerated Approvals Verified Clinical Benefit | Cancer Accelerated Approvals Withdrawn | Cancer Accelerated Approvals Other | Cancer Accelerated Approvals Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) Short Description Oncology (Cancer)/Hematologic Malignancies Approval Notifications Ongoing | Cancer Accelerated Approvals Verified Clinical Benefit | Cancer Accelerated Approvals Withdrawn | Cancer Accelerated Approvals Other | Cancer Accelerated Approvals Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) Short Description

Accelerated ApprovalOrphan DrugPriority ReviewDrug Approval

27 Jun 2024

·pipelinereview.com

Oncopeptides selects first candidate drug from its SPiKE platform

STOCKHOLM, Sweden I June 27, 2024 I Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a biotech company focused on difficult-to-treat cancers, today announces that the first candidate drug based on the company´s unique platform for Small Polypeptide based innate Killer Engagers (SPiKE) has been selected. The SPiKE platform uses multi-specific constructs, able to bind to multiple targets simultaneously. The first drug candidate, OPSP1, is a bi-specific construct designed to both engage natural killer cells, a type of immune cell, and target cancer cells. The goal of OPSP1 is to prove the ability of the SPiKE platform to activate these natural killer cells. To do this, OPSP1 targets a specific protein called BCMA that is expressed in some cancers including multiple myeloma. By targeting this protein, Oncopeptides will be able to assess how well the SPiKE platform can activate natural killer cells to fight cancer, a crucial step before continued clinical development of the SPiKE platform. “There is significant potential in NK cell-mediated therapy for difficult-to-treat cancers,” says Dr. Karl-Johan Malmberg, professor at Oslo University and Karolinska Institutet. ”Despite the substantial advancements in current treatments, the reality is that resistance to these therapies often develops. This underscores the urgent need for new and innovative treatment options to address this unmet medical need.” NK cell-mediated therapy represents a promising avenue in cancer immunotherapy, with ongoing research aimed at overcoming existing challenges and enhancing its therapeutic potential. As a next step for Oncopeptides, a first-in-human clinical trial will be designed to evaluate the safety, efficacy, and overall therapeutic potential of OPSP1. “The ability to show a promising product pipeline behind its flagship product is important for a growing biotech company. Following the accomplishments we have seen with our first technology platform PDC, where we have an approved product, we are excited to also announce progress with SPiKE, our second technology platform,” says Sofia Heigis, CEO of Oncopeptides. “This milestone is a major step forward in our ambition to ensure that Oncopeptides can continue to provide hope for patients suffering from difficult-to-treat cancers, and value to shareholders investing in our science.” A pre-clinical project for the SPiKE platform has received a financial grant from the Eurostars 3-program, co-financed by the European Union research and innovation program “Horizon Europe” and is driven by an international research consortium that includes world-leading expertise from the department of Cancer Immunology at Oslo University Hospital, Pharmatest Services Ltd in Turku, Finland and the Royal Institute of Technology in Stockholm (KTH), from where the technology originally stems. With this grant, Sweden’s Innovation Agency, Vinnova, has provided Oncopeptides resources to develop pre-clinical proof of concept for a novel synthetic small polypeptide for the treatment of multiple myeloma. For more information, including questions and answers for investors and additional details on the SPiKE platform, please visit our website . About Oncopeptides Oncopeptides is a biotech company focusing on research, development and commercialization of targeted therapies for difficult-to-treat cancers. The company uses its proprietary Peptide Drug Candidate platform (PDC) to develop compounds that rapidly and selectively deliver cytotoxic agents into cancer cells. Pepaxti® (melphalan flufenamide, also called melflufen) has been granted Marketing Authorization, in the European Union, the EEA-countries Iceland, Lichtenstein and Norway, as well as in the UK. Pepaxti is indicated in combination with dexamethasone for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation. Oncopeptides is developing several new compounds based on its proprietary technology platforms and is listed on Nasdaq Stockholm with the ticker ONCO. For more information see: www.oncopeptides.com SOURCE: Oncopeptides

Clinical StudyDrug ApprovalImmunotherapyPDC

100 Deals associated with Melphalan flufenamide hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D11865 D11870	-	L01XX	-

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Multiple Myeloma	United States	Oncopeptides AB	26 Feb 2021

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Relapse multiple myeloma	Phase 2	United States	Oncopeptides AB	12 Apr 2018
Relapse multiple myeloma	Phase 2	Czechia	Oncopeptides AB	12 Apr 2018
Relapse multiple myeloma	Phase 2	France	Oncopeptides AB	12 Apr 2018
Relapse multiple myeloma	Phase 2	Spain	Oncopeptides AB	12 Apr 2018
Refractory Multiple Myeloma	Phase 2	United States	Oncopeptides AB	28 Dec 2016
Refractory Multiple Myeloma	Phase 2	France	Oncopeptides AB	28 Dec 2016
Refractory Multiple Myeloma	Phase 2	Italy	Oncopeptides AB	28 Dec 2016
Refractory Multiple Myeloma	Phase 2	Spain	Oncopeptides AB	28 Dec 2016
Immunoglobulin Light-Chain Amyloidosis	Phase 1	United States	Oncopeptides AB	06 Aug 2020
Immunoglobulin Light-Chain Amyloidosis	Phase 1	Czechia	Oncopeptides AB	06 Aug 2020

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03151811 (OCEAN, EHA2024)	Phase 3	Multiple Myeloma del(17p) \| TP53 mutation	-	Melflufen plus dexamethasone	vnszzjmion(vbeshrrwhx) = txedfzhxld qfmjorducl (rnhadjwjjl ) View more	Positive	14 May 2024
				Pomalidomide plus dexamethasone	vnszzjmion(vbeshrrwhx) = yzjlrcxuts qfmjorducl (rnhadjwjjl ) View more
OCEAN (OP-103) (ASH2023)	Phase 3	Refractory Multiple Myeloma	495	Melflufen 40 mg	hlmcxznnjn(acdokvmxgr) = opqmswfqxm ukpaukibxw (qogkgdtser )	-	09 Dec 2023
				Pomalidomide 4 mg	hlmcxznnjn(acdokvmxgr) = jvhhqemohd ukpaukibxw (qogkgdtser )
NCT04649060 (LIGHTHOUSE, IMS2023)	Phase 3	Multiple Myeloma	54	Melflufen + Daratumumab + Dexamethasone	zghheawzav(hkktpyshdb) = vocjysrzpf mwegsurdkd (swcjkmdtkt ) View more	Positive	26 Sep 2023
				Daratumumab	zghheawzav(hkktpyshdb) = xplncladhz mwegsurdkd (swcjkmdtkt ) View more
Anchor (OP?104) (IMS2023)	Not Applicable	Refractory Multiple Myeloma	-	Melflufen + Vd	pjajoxruzr(fjlpcnazga) = kxwjhilenv pzmotnfwxl (kyrwlcuvyy ) View more	-	26 Sep 2023
				Melflufen + Dd	pjajoxruzr(fjlpcnazga) = qxkgdxayfs pzmotnfwxl (kyrwlcuvyy ) View more
NCT03151811 (OCEAN, Pubmed \| Clin Lymphoma Myeloma Leuk)	Phase 3	Multiple Myeloma age	495	Melflufen and Dexamethasone	oenwvdksyb(ceobinicvf) = sgivotjznr mecvwbtuea (tlqnfaxiwt )	Positive	01 Sep 2023
				Pomalidomide	oenwvdksyb(ceobinicvf) = zqdqltnnrk mecvwbtuea (tlqnfaxiwt )
NCT03481556 (ANCHOR, EHA2023)	Phase 1/2	Relapse multiple myeloma	56	Melflufen + Bortezomib + Dex	rdijmcwkhd(jtyefrlhhc) = onflernktz shrxplqncs (qmisjmwtuf ) View more	-	08 Jun 2023
				Melflufen + Daratumumab + Dex	zqvquttzou(alvapsbvky) = yrvuwcjksn bsjzjoubqt (olevhhoegu, 16.4 - not estimable) View more
NCT03481556 (ANCHOR \| ANCHOR (OP-104), CTgov)	Phase 1/2	Relapse multiple myeloma	56	Melflufen+Dexamethasone+Bortezomib (Regimen A - Melflufen 30mg + Bortezomib + Dexamethasone)	gvwbnbkoic = gezvvivskw wrroxabrma (hzyttftzke, stpyjzwjvf - grwwbxnode) View more	-	19 Dec 2022
				Melflufen+Dexamethasone+Bortezomib (Regimen A - Melflufen 40mg + Bortezomib + Dexamethasone)	gvwbnbkoic = dwteqatdua wrroxabrma (hzyttftzke, ewzmyatoaf - xfsbzbjlkw) View more
NCT03151811 (OCEAN, CTgov)	Phase 3	Multiple Myeloma	495	Melflufen+Dexamethasone (Arm A: Melflufen+Dexamethasone)	xtjmhehnjf(lwhjzmfgyt) = ydeebogdzs rzfufpsaos (xwebueszji, wlflaewcpu - ndemsacbsa) View more	-	27 Jul 2022
				Dexamethasone+pomalidomide (Arm B: Pomalidomide+Dexamethasone)	xtjmhehnjf(lwhjzmfgyt) = uzpebwwpty rzfufpsaos (xwebueszji, uevixorrfw - encqwgihhs) View more
NCT03151811 (OCEAN, Pubmed \| Lancet Haematol) Manual	Phase 3	Multiple Myeloma	495	Dexamethasone+Melflufen	hqrzartrun(wfzularaml) = pijpqrinoh sxoyxrqwku (lwvrkikxdc ) View more	Superior	01 Feb 2022
				Dexamethasone+Pomalidomide	hqrzartrun(wfzularaml) = xunxrmrxdp sxoyxrqwku (lwvrkikxdc ) View more

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