captisol-enabled melphalan / Generic mfg. - LARVOL DELTA

Evaluation of Melphalan Population Pharmacokinetic Models to Predict Propylene Glycol-Free Melphalan (Evomela) Clearance (TCT-ASTCT-CIBMTR 2026) - "Our evaluation of population PK models shows that the Shah model more accurately predicts Evomela clearance than the Nath model. However, fewer than 60% of patients had CL within the ±20% prediction range. The extended stability of reconstituted Evomela may influence CL, contributing to lower model concordance."

Clinical • PK/PD data • Transplantation

Evaluate the Safety and Efficacy of Short Versus Long Infusion Schedules of Evomela As Conditioning for Autologous Stem Cell Transplantation in Multiple Myeloma.Interpret Bayesian Modeling Results Assessing the Relationship between Melphalan Exposure (AUC) and Depth of Response or Progression-Free Survival.Identify Potential Pharmacokinetic (AUC) Targets That May Guide Individualized Evomela... (TCT-ASTCT-CIBMTR 2026) - "Evomela 225 mg/m 2 given over either 30 min or 8–9 h was safe and effective. Compared with MEL200, evomela showed a higher probability of achieving MRD-negative CR after ASCT. PK modeling identified AUC ≈ 17,800 µg·h/L as a potential target goal for L-IV in high-risk patients, supporting further evaluation of PK-guided evomela dosing as a conditioning regimen for ASCT in MM."

Clinical • PK/PD data • Febrile Neutropenia • Hematological Malignancies • Multiple Myeloma • Neutropenia • Transplantation

Propylene Glycol-Free Melphalan HCl (EVOMELA®) in Combination With Fludarabine and Total Body Irradiation Based Reduced Intensity Conditioning for Haploidentical Transplantation (clinicaltrials.gov) - P2 | N=43 | Completed | Sponsor: Medical College of Wisconsin | Active, not recruiting ➔ Completed

Trial completion • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation • HLA-B • HLA-C • HLA-DRB1

CASI Pharmaceuticals, Inc. reported that China’s National Medical Products Administration formally rejected its renewal application for the Import Drug Registration License for FOLOTYN in China. (StockTitan.net) - " The company had already stopped selling FOLOTYN in China after the prior license expired, in line with applicable regulations, so sales had ceased before this formal rejection....CASI highlights its focus on developing CID-103 and notes ongoing disputes and legal proceedings related to EVOMELA and CNCT-19, suggesting a portfolio in transition toward pipeline assets."

Pipeline update • Regulatory • Immunology • Oncology

Phase 1/2 Trial to Jointly Optimize Dose and Administration Schedule of Evomela in Newly Diagnosed Multiple Myeloma Patients Undergoing Autologous Hematopoietic Cell Transplantation (ASH 2023) - "Dose escalation of evomela to 225 mg/m2 is safe and associated with an acceptable toxicity profile and a high response rate. Short and extended infusions of evomela are well-tolerated and associated with high response rates. The PFS is longer with the short infusion schedule, however, to affirm these preliminary observations, additional follow-up is needed."

Clinical • P1/2 data • Atrial Fibrillation • Cardiovascular • Gastrointestinal Disorder • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

Study of Captisol Enabled Melphalan and Pharmacokinetics for Patients With Multiple Myeloma or Light Chain Amyloidosis That Are Receiving an Autologous Transplant. (clinicaltrials.gov) - P1 | N=46 | Active, not recruiting | Sponsor: Memorial Sloan Kettering Cancer Center | Trial completion date: Sep 2025 ➔ Sep 2026 | Trial primary completion date: Sep 2025 ➔ Sep 2026

Trial completion date • Trial primary completion date • Amyloidosis • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation • CD34

A real-world data study of the effect of co-solvent differences on the clinical safety of melphalan for injection. (PubMed, Front Med (Lausanne)) - "This study aimed to compare the efficacy and safety of myeloablative conditioning with high-dose propylene glycol-free melphalan (PGF-Mel, EVOMELA®) versus propylene glycol melphalan (PG-Mel) in Chinese patients with multiple myeloma (MM) undergoing autologous stem cell transplantation (ASCT) in the real world. The EVOMELA® group had a higher rate of minimal residual disease (MRD) negativity after ASCT (73.6% vs. 48.1%, p = 0.007) than the PG-Mel group. EVOMELA® appears to demonstrate better efficacy and safety compared to PG-Mel; nonetheless, considering the study's limitations, these observations warrant further rigorous investigation to confirm their validity."

Journal • Real-world evidence • Anorexia • Endocrine Disorders • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P3 | N=750 | Recruiting | Sponsor: Children's Oncology Group | Trial completion date: Sep 2026 ➔ Sep 2030 | Trial primary completion date: Sep 2026 ➔ Sep 2030

Trial completion date • Trial primary completion date • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor • MYCN

Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) (clinicaltrials.gov) - P3 | N=750 | Recruiting | Sponsor: Children's Oncology Group | Active, not recruiting ➔ Recruiting

Enrollment open • Ganglioneuroblastoma • Neuroblastoma • Oncology • Solid Tumor • MYCN

CASI Pharmaceuticals Enters Into Definitive Agreement for the Divestiture of Assets in China (ACCESSWIRE) - "CASI Pharmaceuticals...announced that it has entered into a definitive Equity and Assets Transfer Agreement...with Kaixin Pharmaceuticals Inc...and CEO of the Company and two direct wholly-owned subsidiaries of the Company in China...pursuant to which the Company shall sell and transfer, and Kaixin Pharmaceuticals shall purchase and acquire, 100% equity interests in both Target Companies...and all licensing rights, distribution rights, supply arrangements and related rights related to BI-1206 (in China), CID-103 (in Asia excluding Japan) and Thiotepa (in China excluding Hong Kong, Macau and Taiwan)...for an aggregate purchase price of $20.0 million, which shall include assumption of up to $20.0 million of indebtedness of the Company....After the closing of the Transaction, the Company expects to retain the rights related to CID-103 (in Japan and non-Asian regions), EVOMELA, FOLOTYN, CNCT19 and CB-5339, and remain firmly committed to progressing CID-103 at an accelerated pace."

Commercial • Hematological Malignancies • Immunology • Solid Tumor

Intra-arterial Chemotherapy for Retinoblastoma (clinicaltrials.gov) - P1 | N=5 | Completed | Sponsor: Washington University School of Medicine | Active, not recruiting ➔ Completed | Trial completion date: Jan 2026 ➔ Mar 2025

Trial completion • Trial completion date • Eye Cancer • Oncology • Retinal Disorders • Retinoblastoma • Solid Tumor

Safety, effectiveness and pharmacokinetics of high-dose propylene glycol-free melphalan (EVOMELA) with a prolonged infusion as myeloablative conditioning in Chinese multiple myeloma patients undergoing autologous stem cell transplantation: A prospective phase iv study. (PubMed, J Oncol Pharm Pract) - "Prolonged infusion resulted in a lower Cmax but comparable AUCs.ConclusionsHigh-dose BSES-melphalan as a conditioning medicine is effective and safe in Chinese patients with MM before ASCT. Prolonging infusion duration may improve the safety without compromising efficacy."

Journal • P4 data • PK/PD data • Bone Marrow Transplantation • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

Propylene Glycol-Free Melphalan HCl (EVOMELA®) in Combination with Fludarabine and Total Body Irradiation Based Reduced Intensity Conditioning for Haploidentical Transplantation (clinicaltrials.gov) - P2 | N=43 | Active, not recruiting | Sponsor: Medical College of Wisconsin | Recruiting ➔ Active, not recruiting | Trial primary completion date: Nov 2025 ➔ Nov 2024

Enrollment closed • Trial primary completion date • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation • HLA-B • HLA-C • HLA-DRB1

Intra-arterial Chemotherapy for Retinoblastoma (clinicaltrials.gov) - P1 | N=5 | Active, not recruiting | Sponsor: Washington University School of Medicine | Trial completion date: Jan 2025 ➔ Jan 2026

Trial completion date • Eye Cancer • Oncology • Retinal Disorders • Retinoblastoma • Solid Tumor

Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation (clinicaltrials.gov) - P2 | N=43 | Active, not recruiting | Sponsor: Emory University | Recruiting ➔ Active, not recruiting

Enrollment closed • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Transplantation

Study of Captisol Enabled Melphalan and Pharmacokinetics for Patients With Multiple Myeloma or Light Chain Amyloidosis That Are Receiving an Autologous Transplant. (clinicaltrials.gov) - P1 | N=46 | Active, not recruiting | Sponsor: Memorial Sloan Kettering Cancer Center | Trial completion date: Sep 2024 ➔ Sep 2025 | Trial primary completion date: Sep 2024 ➔ Sep 2025

Trial completion date • Trial primary completion date • Amyloidosis • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation • CD34

Pre-transplant Purging and Post-transplant MRD-guided Maintenance Therapy With Elranatamab in Patients With High-risk Multiple Myeloma (clinicaltrials.gov) - P2 | N=40 | Recruiting | Sponsor: M.D. Anderson Cancer Center | Not yet recruiting ➔ Recruiting

Enrollment open • Post-transplantation • Pre-transplantation • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

NCI-2018-00906: Melphalan Hydrochloride in Treating Participants With Newly-Diagnosed Multiple Myeloma Undergoing Donor Stem Cell Transplantation (clinicaltrials.gov) - P1/2 | N=62 | Completed | Sponsor: M.D. Anderson Cancer Center | Active, not recruiting ➔ Completed | Trial completion date: Dec 2024 ➔ Jul 2024 | Trial primary completion date: Dec 2024 ➔ Jul 2024

Trial completion • Trial completion date • Trial primary completion date • Bone Marrow Transplantation • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

Thiotepa Plus Fludarabine+ Melphalan as the Preparative Regime for Alternative Donor Transplantation (clinicaltrials.gov) - P2 | N=40 | Completed | Sponsor: Case Comprehensive Cancer Center | Trial completion date: Jun 2025 ➔ Jun 2024 | Active, not recruiting ➔ Completed

Trial completion • Trial completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Lymphocytic Leukemia • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • FLT3

KANGFUXIN-BASED CRYOTHERAPY DEMONSTRATED A SIGNIFICANT PROTECTIVE EFFECT ON ORAL MUCOSITIS IN MYELOMA PATIENTS UNDERGOING ASCT WITH MELPHALAN HYDROCHLORIDE AS CONDITIONING REGIMEN (EHA 2024) - "Melphalan Hydrochloride (trade name Evomela),utilizing specially modified cyclodextrin as a solvent, has significantly improved the drug's solubility andstability while preventing adverse reactions associated with propylene glycol. Kangfuxin-base cryotherapy represents an effective and inexpensive supportive measure to prevent OMinduced by HDmel-based regimens in ASCT."

Clinical • Bone Marrow Transplantation • Hematological Malignancies • Mucositis • Multiple Myeloma • Oncology • Pain • Stomatitis • Transplantation

PHASE 1/2 TRIAL TO JOINTLY OPTIMIZE DOSE AND ADMINISTRATION SCHEDULE OF EVOMELA IN NEWLY DIAGNOSED MULTIPLE MYELOMA PATIENTS UNDERGOING AUTOLOGOUS HEMATOPOIETIC CELL TRANSPLANTATION (EHA 2024) - "The dose escalation of evomela to 225 mg/m2 is safe and associated with an acceptable toxicity profile and ahigh response rate. Achieving a higher AUC, a lower evomela clearance, and the extended evomela infusion isassociated with a substantially higher probability of achieving MRD-negative CR at day-100 and 1-year afterauto-HCT. However, longer follow-up is required to discern any survival difference."

Clinical • P1/2 data • Atrial Fibrillation • Cardiovascular • Gastrointestinal Disorder • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

NCI-2017-01069: Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant (clinicaltrials.gov) - P1/2 | N=35 | Active, not recruiting | Sponsor: Roswell Park Cancer Institute | Trial completion date: Feb 2024 ➔ May 2027 | Trial primary completion date: Feb 2024 ➔ Aug 2023

Post-transplantation • Trial completion date • Trial primary completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Lymphocytic Leukemia • Chronic Myeloid Leukemia • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Lymphoma • Lymphoplasmacytic Lymphoma • Multiple Myeloma • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Non-Hodgkin’s Lymphoma • Oncology • T Acute Lymphoblastic Leukemia • Transplantation • Waldenstrom Macroglobulinemia • HLA-DRB1

Evaluate Safety and Efficacy of High-dose Melphalan HCL for Injection in MM Patients With Auto-HSC Transplantation (clinicaltrials.gov) - P4 | N=67 | Completed | Sponsor: CASI Pharmaceuticals (China) Co., Ltd.

New P4 trial • Bone Marrow Transplantation • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

CASI PHARMACEUTICALS ANNOUNCES FIRST QUARTER 2024 BUSINESS AND FINANCIAL RESULTS (PRNewswire) - "In the first quarter of 2024, CASI's total revenue was $3.4 million. Total revenue decreased by 59% compared to the same period in 2023. This decline can be attributed to several factors: increasing competition from a generic melphalan product with lower cost and undifferentiated formulation, implementation of an inventory management strategy by the distributor of EVOMELA....With respect to Inaticabtagene Autoleucel (CNCT-19 CAR-T cell therapy), we have received an emergency injunctive relief that prohibits Juventas from commercializing CNCT-19 by itself or through another third party, and while continuing to vigorously assert and enforce our rights with respect to the commercial launch of CNCT-19, we intend to negotiate with Juventas in good faith the temporary arrangement for new patients' access to CNCT-19 treatment. We will continue to drive our portfolio forward by executing on several milestones in the quarters ahead."

Commercial • Licensing / partnership • Hematological Malignancies • Immunology • Oncology • Solid Tumor

Melphalan on Disease Burden Measured by Next Generation Sequencing Before AHCT (Autologous Hematopoietic Cell Transplant) for Multiple Myeloma (clinicaltrials.gov) - P1 | N=0 | Withdrawn | Sponsor: Koen van Besien | N=20 ➔ 0 | Trial completion date: Sep 2023 ➔ Mar 2024 | Recruiting ➔ Withdrawn | Trial primary completion date: Jun 2023 ➔ Dec 2023

Biomarker • Enrollment change • Trial completion date • Trial primary completion date • Trial withdrawal • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation • CD34