Melphalan flufenamide

also known as Melflufen · CK 1535 · J 1 (PRODRUG) · J-1 HYDROCHLORIDE · Melfalan flufenamida · Melflufen hydrochloride · MELPHALAN FLUFENAMIDE HYDROCHLORIDE · Pepaxti · +2 more

Peptide

Developed for: plasma cell myeloma · refractory plasma cell neoplasm · AL amyloidosis · indolent plasma cell myeloma
Investigated by: Oncopeptides AB · Fondazione EMN Italy Onlus · PRA Health Sciences · Precision For Medicine

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

Milestone	Jurisdiction	Brand	Indication	Date	Sentence it was read from
Approved	EU (EMA)	Pepaxti	—	2022-08-17	europa.eu ↗
Clinical hold	US (FDA)	—	—	2021-07-08	“In July 2021, the US Food and Drug Administration (FDA) requested a partial clinical hold of all studies with melflufen.” oncopeptides.com ↗ “the FDA has requested a partial clinical hold of all clinical studies with melflufen, pending further investigation.” oncopeptides.com ↗

Trials 9

today

EU approved 2022

NCT06682637 · Ph 2 NCT04649060 · Ph 3 NCT04115956 · Ph 1 NCT03639610 · Ph 2 NCT03481556 · Ph 1/2 NCT03151811 · Ph 3 NCT02963493 · Ph 2 NCT01897714 · Ph 1/2

20142015201620172018201920202021202220232024202520262027

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 11 trial
Phase 1	NCT04115956	Aug 2020 → Jan 2022	AL amyloidosis	Oncopeptides AB	Terminated	No outcome recorded
Phase 1/22 trials
Phase 1/2	NCT03481556	Apr 2018 → Feb 2022	plasma cell myeloma	Oncopeptides AB	Terminated	No outcome recorded
Phase 1/2	NCT01897714	Jul 2013 → Dec 2017	indolent plasma cell myeloma	Oncopeptides AB	Terminated	No outcome recorded
Phase 23 trials
Phase 2	NCT06682637	Mar 2025	—	Fondazione EMN Italy Onlus	Withdrawn	No outcome recorded
Phase 2	NCT03639610	Aug 2018 → Dec 2021	impaired renal function disease, plasma cell myeloma	Oncopeptides AB	Terminated	No outcome recorded
Phase 2	NCT02963493	Dec 2016 → Oct 2020	plasma cell myeloma	Oncopeptides AB	Completed	No outcome recorded
Phase 32 trials · 2 met primary
Phase 3	NCT04649060	Dec 2020 → Feb 2022	plasma cell myeloma, refractory plasma cell neoplasm	Oncopeptides AB	Terminated	Met primary Stop: Business
Phase 3	NCT03151811	Jun 2017 → Feb 2021	plasma cell myeloma	Oncopeptides AB	Terminated	Met primary
Phase not stated1 trial
—	NCT04534322	—	refractory plasma cell neoplasm	Oncopeptides AB	Approved for marketing	No outcome recorded

News releases announcing trial results or a regulatory action · 65

Date	Issuer	Release
2026-10-09	Oncopeptides AB	Results Oncopeptides confirms blood-brain-barrier penetration based on data from first patients in glioblastoma Window-of-Opportunity study oncopeptides.com ↗ Analysis of resected tumor tissue from three evaluated patients showed clear and consistent presence of active compound (the primary endpoint in this part of the study), indicating efficient penetration across the human blood-brain barrier.
2026-09-22	Oncopeptides AB	Results Preclinical and translational glioblastoma data to be presented at EANO 2026, Oncopeptides nominated for prestigious EANO 2026 Poster award oncopeptides.com ↗ Studies in animal models confirmed that the drugs successfully reach high concentrations in the brain relative to the bloodstream.
2026-09-17	Oncopeptides AB	Regulatory CHMP issues positive opinion of label expansion of Pepaxti to third-line treatment oncopeptides.com ↗ Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a biotech company focused on difficult-to-treat cancers, today announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending an extension of the therapeutic indication for Pepaxti (melflufen) to earlier lines of therapy.
2026-08-17	Oncopeptides AB	Regulatory Oncopeptides receives list price for Pepaxti in Slovenia, marking first expansion milestone in Central and Eastern Europe oncopeptides.com ↗ Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a biotech company focused on difficult-to-treat cancers, today announces that the Public Agency of the Republic of Slovenia for Medicinal Products and Medical Devices (JAZMP) has formally endorsed the Maximum Allowed Price (MAP) for Pepaxti (melflufen) in Slovenia.
2026-06-12	Oncopeptides AB	Regulatory Oncopeptides submits Type II variation application to EMA for Pepaxti label expansion into third line treatment oncopeptides.com ↗ STOCKHOLM — June 12, 2026 — Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a biotech company focused on difficult-to-treat cancers, today announces that it has formally submitted its Type II variation application to the European Medicines Agency (EMA).
2026-05-27	Oncopeptides AB	Results Oncopeptides to present clinical and translational data on Pepaxti at EHA 2026 in Stockholm oncopeptides.com ↗ New translational data demonstrates that melflufen induces damage to both nuclear and mitochondrial DNA.
2026-05-20	Oncopeptides AB	Results Study confirms use of Pepaxti in a broader population, with strong data in the 50% of RRMM patients that suffer from reduced kidney function oncopeptides.com ↗ Study confirms use of Pepaxti in a broader population, with strong data in the 50% of RRMM patients that suffer from reduced kidney function
2026-05-13	Oncopeptides AB	Results COMy: New clinical and real-world evidence for Pepaxti demonstrating strong data and expanded positioning oncopeptides.com ↗ Three e-poster presentations of data related to Pepaxti will be held during the conference.
2026-03-13	Oncopeptides AB	Results European Journal of Haematology: Real-World Data reinforces Pepaxti’s role in treatment sequencing for multiple myeloma oncopeptides.com ↗ Despite a highly refractory population—where 88% were triple-class refractory and 41% were penta-drug refractory—Pepaxti demonstrated a clinically meaningful overall response rate (ORR) of 41%.
2025-12-29	Oncopeptides AB	Results Research shows that Pepaxti is effective in high-risk myeloma oncopeptides.com ↗ Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a biotech company focused on difficult-to-treat cancers, today announces new research demonstrating that Pepaxti (melflufen) maintains its anti-myeloma activity in patients with high-risk genetic features, including deletion 17p (del(17p)) and TP53 mutations.
2025-10-16	Oncopeptides AB	Results Journal of Cancer Research and Clinical Oncology: Exceptional long-term responses to Pepaxti oncopeptides.com ↗
2025-04-16	Oncopeptides AB	Results New Real-World Data support effectiveness and tolerability of Pepaxti in heavily pretreated Multiple Myeloma patients oncopeptides.com ↗ Despite this, treatment with Pepaxti in combination with dexamethasone showed an overall response rate (ORR) of 37.5%, with partial responses seen even among patients with advanced disease manifestations.
2025-02-25	Oncopeptides AB	Results Strong real-world efficacy and safety data with Pepaxti published in the European Journal of Haematology oncopeptides.com ↗ The study, authored by researchers at the Dana-Farber Cancer Institute in Boston, Massachusetts, USA found that melflufen demonstrated clinically meaningful efficacy, with an overall response rate (ORR) of 55%, including complete responses in 27% of patients.
2025-01-27	Oncopeptides AB	Regulatory Oncopeptides’ Pepaxti formally approved for full reimbursement in Italy oncopeptides.com ↗ Stockholm, January 27, 2025 – Oncopeptides AB (publ), a biotech company focused on difficult-to-treat cancers, today announces that the positive reimbursement decision for its drug Pepaxti (melflufen) has been published in the Italian Official Journal (“IOJ”, It.: Gazetta Ufficiale).
2024-11-27	Oncopeptides AB	Regulatory Oncopeptides obtains reimbursement for Pepaxti in Italy oncopeptides.com ↗ Oncopeptides AB (publ), a biotech company focused on difficult-to-treat cancers, today announces that it has come to an agreement with the Italian Medicines Agency (Agenzia Italiana del Farmaco, AIFA) on the pricing and reimbursement of melflufen, branded in Europe as Pepaxti.
2024-05-16	Oncopeptides AB	Results Oncopeptides presents new data highlighting treatment benefits of Pepaxti in high-risk multiple myeloma patients at the COMy Congress oncopeptides.com ↗ The research findings indicate that melflufen is more effective than traditional alkylating agents causing DNA damage and promoting the death of cancer cells lacking a functional so-called p53 protein, which is a common issue in high-risk multiple myeloma cases.
2024-03-01	Oncopeptides AB	Results Oncopeptides’ PORT study shows peripheral administration of Pepaxti being equally safe as central venous administration oncopeptides.com ↗ The results provide further evidence that administration via peripheral venous catheter (PVC) is well tolerated with no local infusion-related reactions or new safety signals and may represent an alternative route of administration.
2024-02-29	Oncopeptides AB	Results Pepaxti provides sustained health related Quality of Life OCEAN study shows, article published in Haematologica oncopeptides.com ↗ The article, titled “Health-related quality of life in relapsed/refractory multiple myeloma treated with melflufen and dexamethasone: analyses from the phase 3 OCEAN study” concludes that melflufen plus dexamethasone is shown to be safe and effective with sustained health-related quality of life also in later lines of therapy.
2024-02-23	Oncopeptides AB	Regulatory Oncopeptides receives positive recommendation for Pepaxti by Spanish Pricing Authority oncopeptides.com ↗ Oncopeptides AB (publ), a biotech company focused on difficult-to-treat cancers, today announces that the Spanish Drug Pricing Authority (CIPM) has published a positive recommendation for the pricing of melflufen, branded in Europe as Pepaxti.
2023-11-17	Oncopeptides AB	Results Article with exploratory alkylator-refractory subgroup analysis from Oncopeptides’ OCEAN study observing longer PFS and OS in melflufen vs. pomalidomide published in European Journal of Haematology oncopeptides.com ↗ The results suggest that melflufen is safe and effective in patients with alkylator-refractory disease, suggesting differentiated activity from other alkylators.
2023-11-13	Oncopeptides AB	Results Oncopeptides selected to present additional data from OCEAN study at the American Society of Hematology meeting oncopeptides.com ↗ The abstract including key data has been published and is available through this link.
2023-09-04	Oncopeptides AB	Results Oncopeptides publishes two articles with results from ANCHOR and LIGHTHOUSE studies in Haematologica oncopeptides.com ↗ It concludes that melflufen plus daratumumab and dexamethasone demonstrated superior progression free survival (HR 0.18 [0.05-0.65], p=0.0032).
2023-05-12	Oncopeptides AB	Results Correction: Oncopeptides presents new data at the European Haematology Association meeting oncopeptides.com ↗
2023-05-11	Oncopeptides AB	Results Oncopeptides presents new data at the European Haematology Association meeting oncopeptides.com ↗
2023-04-20	Oncopeptides AB	Results Oncopeptides presents new scientific data at European Myeloma Network Meeting oncopeptides.com ↗ The results from OCEAN are important since HRQoL was maintained throughout the treatment with melflufen plus dexamethasone and was similar to the outcome from the treatment with pomalidomide plus dexamethasone.
2022-12-12	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides presents new scientific data at the Annual American Society of Hematology Meeting ASH oncopeptides.com ↗ Data shows that melflufen is a safe and effective therapy in patients who are alkylator refractory, regardless of whether they received a prior autologous stem cell transplant or not.
2022-12-07	Oncopeptides AB	Regulatory Oncopeptides AB: Oncopeptides provides update on Pepaxto US marketing authorization oncopeptides.com ↗ today announces that the US Food and Drug Administration, FDA, has requested a withdrawal of the US marketing authorization for Pepaxto[® ](melphalan flufenamide, also called melflufen).
2022-11-11	Oncopeptides AB	Regulatory Oncopeptides AB: Oncopeptides’ Pepaxti has been granted marketing authorization in the UK oncopeptides.com ↗
2022-10-26	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides phase 3 LIGHTHOUSE study further confirms clinical benefit of melflufen oncopeptides.com ↗
2022-09-23	Oncopeptides AB	Regulatory Oncopeptides AB: Oncopeptides provides an update from the meeting with FDAs advisory committee ODAC oncopeptides.com ↗ In a 14 to 2 vote, the ODAC answered no to the question.
2022-08-18	Oncopeptides AB	Regulatory Oncopeptides AB: European Commission approves Oncopeptides’ Pepaxti for the treatment of patients with relapsed refractory multiple myeloma oncopeptides.com ↗
2022-06-23	Oncopeptides AB	Regulatory Oncopeptides AB: CHMP issues a positive opinion recommending full approval of Oncopeptides Pepaxti in EU for patients with triple class refractory multiple myeloma oncopeptides.com ↗ today announces that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), has unanimously adopted a positive opinion recommending a full marketing authorization approval (MAA) of Pepaxti[®] (melphalan flufenamide, also called melflufen) in EU.
2022-01-21	Oncopeptides AB	Regulatory Oncopeptides AB: Oncopeptides rescinds voluntary withdrawal of Pepaxto in the US – no intent to market in the US at this time oncopeptides.com ↗ Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a biotech company focused on research and development of therapies for difficult-to-treat hematological diseases, today announces that the Company has contacted the US Food and Drug Administration and rescinded the October 22, 2021, letter requesting voluntary withdrawal of the NDA of Pepaxto[® ](INN melphalan flufenamide, also called melflufen) in the US.
2022-01-13	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides phase 3 OCEAN study published in the Lancet Haematology oncopeptides.com ↗ “The OCEAN results demonstrates that melflufen improves PFS for lenalidomide refractory RRMM patients who have received two to four prior lines of therapy,”
2021-10-22	Oncopeptides AB	Regulatory Oncopeptides AB: Oncopeptides withdraws Pepaxto®[ ]in US, scale down organization and focus on R&D oncopeptides.com ↗
2021-09-11	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides presents phase 3 OCEAN study results at the IMW meeting oncopeptides.com ↗ The data was presented today by Fredrik Schjesvold, Head of Oslo Myeloma Center, Norway, at the 18[th] International Myeloma Workshop (IMW) in Vienna.
2021-08-27	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides presents new data from OCEAN and PORT study – abstracts online oncopeptides.com ↗ The HR for OS was 1.10 in favor of pomalidomide in the Intention to Treat (ITT) population.
2021-07-08	Oncopeptides AB	Results Oncopeptides AB: Updated results from phase 3 OCEAN study shows melflufen met primary endpoint of superior PFS – Overall Survival data lead to partial clinical hold oncopeptides.com ↗ In the final analysis melflufen met the primary endpoint of superior Progression Free Survival (PFS) compared to pomalidomide with a Hazard Ratio (HR) of 0.792 (95% CI 0.640-0.979, p-value 0.0311) as determined by the IRC.
2021-05-25	Oncopeptides AB	Results Oncopeptides AB: Phase 3 OCEAN study demonstrates that melflufen is at least as efficacious as pomalidomide, the most used medicine in relapsed refractory multiple myeloma oncopeptides.com ↗ today announces positive topline results from the head-to-head phase 3 OCEAN study evaluating the efficacy and safety of melflufen (INN melphalan flufenamide) versus pomalidomide in patients with relapsed refractory multiple myeloma (RRMM).
2021-05-19	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides presents clinical abstracts on melflufen at the 2021 American Society of Clinical Oncology oncopeptides.com ↗ The data presented here at ASCO data clearly support further development of melflufen in triplet regimens and reinforce the rational for the ongoing phase 3 LIGHTHOUSE study, comparing melflufen plus dexamethasone in combination with subcutaneous daratumumab with daratumumab alone.
2021-05-12	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides presents new clinical and preclinical melflufen data at the upcoming European Hematology Association meeting oncopeptides.com ↗ The clinical data presentation contains interim results from the phase 2 BRIDGE study supporting the use of melflufen in relapsed refractory multiple myeloma patients with moderately impaired renal function.
2021-04-16	Oncopeptides AB	Regulatory Oncopeptides AB: Oncopeptides submits application for conditional marketing authorization of melflufen in the EU oncopeptides.com ↗
2021-02-26	Oncopeptides AB	Regulatory Oncopeptides AB: FDA approves Oncopeptides’ PEPAXTO® ( melphalan flufenamide) for patients with relapsed or refractory multiple myeloma oncopeptides.com ↗ PEPAXTO is the first anticancer peptide-drug conjugate approved by the FDA.
2020-12-09	Oncopeptides AB	Results Oncopeptides AB: Full data set of Oncopeptides phase 2 HORIZON study in multiple myeloma published in the Journal of Clinical Oncology oncopeptides.com ↗
2020-12-06	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides presents new data from phase 2 ANCHOR combination study in multiple myeloma at American Society of Hematology meeting ASH oncopeptides.com ↗ The overall response rate for melflufen plus dexamethasone was 73% in combination with daratumumab and 62% in combination with bortezomib.
2020-11-04	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides Announces that New Data for Melflufen is Accepted for Presentation at the Annual American Society of Hematology Meeting oncopeptides.com ↗ The updated analysis of the ongoing phase 1/2 ANCHOR study confirms the initial findings of encouraging activity as a triplet regimen with melflufen plus dexamethasone and either daratumumab or bortezomib
2020-08-29	Oncopeptides AB	Regulatory Oncopeptides AB: FDA grants Priority Review of melflufen for patients with triple-class refractory multiple myeloma oncopeptides.com ↗ has granted priority review for Oncopeptides’ New Drug Application seeking approval of melflufen
2020-06-30	Oncopeptides AB	Regulatory Oncopeptides AB: Oncopeptides submits a New Drug Application to the FDA for accelerated approval of melflufen in triple-class refractory multiple myeloma patients oncopeptides.com ↗ today announces that the Company submits a New Drug Application (NDA) to the U.S. Food and Drug Administration, FDA, for accelerated approval of melflufen (INN melphalan flufenamide) in combination with dexamethasone for the treatment of adult patients with multiple myeloma whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent and one anti-CD38 monoclonal antibody (i.e., triple-class refractory multiple myeloma patients).
2020-06-15	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides reports positive results from full data set of Phase 2 HORIZON trial in triple-class refractory multiple myeloma patients oncopeptides.com ↗ Oncopeptides AB (Nasdaq Stockholm: ONCO) announces today that the full top-line results from the pivotal phase 2 HORIZON study, evaluating intravenous melflufen in combination with dexamethasone in patients with relapsed refractory multiple myeloma (RRMM), have been presented at the ongoing 25[th] European Hematology Association meeting, EHA.
2020-05-14	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides announces new clinical and preclinical melflufen data at the upcoming European Hematology Association meeting oncopeptides.com ↗ The primary read-out of the HORIZON-data represents an important milestone for Oncopeptides.
2020-03-26	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides announces 26% Overall Response Rate of melflufen in triple-class refractory multiple myeloma patients from the pivotal HORIZON study oncopeptides.com ↗ Oncopeptides AB (Nasdaq Stockholm: ONCO) announces today the final topline results from the pivotal phase 2 HORIZON study evaluating melflufen in relapsed refractory multiple myeloma (RRMM) patients.
2020-03-23	Oncopeptides AB	Results Oncopeptides AB: The Lancet Haematology Publishes Results from Oncopeptides’ Multicenter, International Phase 1/2 Study (O-12-M1) oncopeptides.com ↗ Patients receiving melflufen plus dexamethasone experienced an overall response rate (ORR) of 31%, a median duration of response (DOR) of 8.4 months, and median overall survival (OS) of 20.7 months.
2019-12-08	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides presents promising data from the Phase 2 ANCHOR combination study in patients with RRMM at ASH Annual Meeting 2019 oncopeptides.com ↗ In the data presented, melflufen and dexamethasone demonstrated positive efficacy in combination with daratumumab or bortezomib in patients with relapsed/refractory multiple myeloma (RRMM).
2019-12-07	Oncopeptides AB	Results Oncopeptides AB: Oncopeptides presents updated efficacy and safety data from pivotal Phase 2 HORIZON study in patients with RRMM at ASH Annual Meeting 2019 oncopeptides.com ↗ Melflufen showed an overall response rate (ORR) of 29% and clinical benefit rate (CBR) of 37% in patients with late-stage relapsed/refractory multiple myeloma (RRMM).
2019-09-15	Oncopeptides AB	Results Oncopeptides AB: New Interim Data in RRMM Patients with Extramedullary Disease from the Pivotal Phase 2 Horizon-study presented at International Myeloma Workshop oncopeptides.com ↗ In the HORIZON study, an overall response rate of 23% was achieved with treatment with melflfufen and dexamethasone in RRMM patients with EM
2019-09-11	Oncopeptides AB	Results Oncopeptides AB: Data from Oncopeptides Melflufen Clinical Development Program Presented at SOHO 2019 Annual Meeting oncopeptides.com ↗ In the study, patients demonstrated an overall response rate (ORR) of 28% and a clinical benefit rate (CBR) of 40%.
2019-08-30	Oncopeptides AB	Results Oncopeptides AB: New updated data will be presented from Oncopeptides’ Pivotal Phase 2 HORIZON study in patients with extra medullary disease (EMD) on September 15th oncopeptides.com ↗ Patients without extra-medullary disease showed a response rate of 30% with a median duration of response of 7.5 months and in patients with EMD the response rate was 24% with a median duration of response of 3.1 months.
2019-06-16	Oncopeptides AB	Results Oncopeptides AB: New Data Presented from Oncopeptides’ Pivotal Phase 2 HORIZON Trial Evaluating Melflufen in Relapsed/Refractory Multiple Myeloma at 24th EHA Congress oncopeptides.com ↗ Oncopeptides AB (Nasdaq Stockholm: ONCO) announced today updated interim efficacy and safety data from the ongoing, pivotal Phase 2 HORIZON trial.
2019-06-14	Oncopeptides AB	Results Oncopeptides AB: New Data Presented at 24th EHA Congress from Oncopeptides’ Phase 1/2 ANCHOR Trial Evaluating Melflufen in Relapsed/Refractory Multiple Myeloma (RRMM) oncopeptides.com ↗ For patients that had completed two or more cycles of therapy the overall response rate (ORR) was 100% for the bortezomib combination and 82% for the daratumumab combination.
2019-06-03	Oncopeptides AB	Results Oncopeptides AB: New Data from Oncopeptides Phase 1/2 O-12-M1 Trial Evaluating Melflufen in RRMM Presented at 2019 ASCO Annual Meeting oncopeptides.com ↗ The data demonstrate that melflufen may offer positive disease stabilization and favorable time to next treatment (TTNT) outcomes in heavily-pretreated patients with relapsed/refractory multiple myeloma (RRMM).
2018-12-03	Oncopeptides AB	Results Interim data from the ongoing HORIZON trial with melflufen presented in an oral presentation at the 60th American Society of Hematology Meeting oncopeptides.com ↗ The first evaluation of Progression Free Survival (PFS) shows a median of 4.0 months in the ongoing trial with a median of 6.4 months for responding patients
2018-12-01	Oncopeptides AB	Results Oncopeptides presents first interim data from the ongoing combination trial ANCHOR with melflufen at the 60th American Society of Hematology Meeting oncopeptides.com ↗ An Overall Response Rate (ORR) of 86% was observed with melflufen and dexamethasone in combination with daratumumab (CD38-directed monoclonal antibody)
2018-06-15	Oncopeptides AB	Results Oncopeptides present updated interim data from the ongoing HORIZON trial with Ygalo® at the 23rd Congress of The European Hematology Association (EHA) oncopeptides.com ↗ Despite all this, we see a tumor response in 32% of patients, disease stabilization in 84% of patients, positive initial indication of the duration of the treatment effect as well as a manageable safety profile for Ygalo®.
2017-12-10	Oncopeptides AB	Results Oncopeptides presented survival data from the phase II-study O-12-M1 of Ygalo® (melflufen) at the 59th Annual Meeting of ASH today oncopeptides.com ↗ The results show a median progression-free survival (PFS) of 5.7 months, a median overall survival (OS) of 20.7 months and an overall response rate of 31%.
2017-11-01	Oncopeptides AB	Results Oncopeptides to present data from two clinical studies of Ygalo® (melflufen) at American Society of Hematology (ASH), December 9-12. oncopeptides.com ↗ In the abstract, the median overall survival (OS) of 20.7 months and the median progression-free survival (PFS) of 5.1 months are improvements compared with the previously reported figures of 18.2 months and 4.3 months respectively.

All press releases naming this drug 106 releases

Date	Issuer	Release
Show all 106 releases opens the full record — a long page

Evidence & citations 13 cited values

Every value below carries the sentence it was read from. 11 sources stand behind the page.

Field	Value	Cited text
Known as	Melphalan-flufenamide	“An Open-Label Phase I/IIa Study of the Safety and Efficacy of Melphalan-flufenamide (Melflufen) and Dexamethasone Combination for Patients With Relapsed and/or...” NCT01897714 ↗ 4 NCT04534322 ↗ NCT04115956 ↗ NCT03481556 ↗ NCT02963493 ↗
Known as	CK 1535	ChEMBL registry synonym — accepted as the source's own label CHEMBL4303060 ↗
Known as	J 1 (PRODRUG)	ChEMBL registry synonym — accepted as the source's own label CHEMBL4303060 ↗
Known as	J-1 HYDROCHLORIDE	ChEMBL registry synonym — accepted as the source's own label CHEMBL4303060 ↗
Known as	Melfalan flufenamida	ChEMBL registry synonym — accepted as the source's own label CHEMBL4303060 ↗
Known as	Melflufen	“Melphalan flufenamide (melflufen) is a peptide-drug conjugate that rapidly delivers an alkylating payload into tumor cells.” NCT02963493 ↗ 7 “Melphalan flufenamide (Melflufen)” NCT03481556 ↗ “Melphalan-Flufenamide (Melflufen)” NCT04115956 ↗ NCT01897714 ↗ NCT03639610 ↗ NCT06682637 ↗ NCT04649060 ↗ NCT03151811 ↗
Known as	Melflufen hydrochloride	ChEMBL registry synonym — accepted as the source's own label CHEMBL4303060 ↗
Known as	MELPHALAN FLUFENAMIDE HYDROCHLORIDE	ChEMBL registry synonym — accepted as the source's own label CHEMBL4303060 ↗
Known as	Pepaxti	ChEMBL registry synonym — accepted as the source's own label CHEMBL4303060 ↗
Known as	Pepaxto	ChEMBL registry synonym — accepted as the source's own label CHEMBL4303060 ↗
Known as	PRODRUG J 1	ChEMBL registry synonym — accepted as the source's own label CHEMBL4303060 ↗
Modality	Peptide	“Melphalan flufenamide (melflufen) is a peptide-drug conjugate” NCT02963493 ↗
Route	Intravenous	“patients received an intravenous infusion of melflufen at 15 mg, 25 mg, 40 mg, or 55 mg” PMID 32213344 ↗ Mar 2020