captisol-enabled melphalan / Generic mfg. - LARVOL DELTA

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

CASI PHARMACEUTICALS ANNOUNCES FOURTH QUARTER AND FULL-YEAR 2023 BUSINESS AND FINANCIAL RESULTS (PRNewswire) - "Revenues consist of product sales of EVOMELA. Revenue was $34.0 million for the year ended December 31, 2023 compared to $38 million for the year ended December 31, 2022....Costs of revenues were $13.8 million for the year ended December 31, 2023 compared to $15.8 million for the year ended December 31, 2022. Costs of revenues as a percentage of EVOMELA sales for 2023 and 2022 were 41% and 42%, respectively."

Sales • Hematological Malignancies • Multiple Myeloma • Oncology

Lower PG-Free Mel (Evomela) Clearance and Higher Exposure Are Associated with Better Treatment Response in Newly Diagnosed Multiple Myeloma Patients Undergoing Autologous Hematopoietic Cell Transplantation (TCT-ASTCT-CIBMTR 2024) - "Our analyses of Evomela PK suggested higher Cmax, lower CL, and higher exposure in S-IV compared to L-IV schedules. Patients with lower CL and therefore higher exposure had greater probabilities of achieving sCR/CR and achieving MRD negativity. Further analyses in a larger sample population is needed to determine the target exposure range to achieve optimal treatment response."

Clinical • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

Reduced Intensity Conditioning (RIC) Transplant with Very Low Dose Evomela Is Safe and Effective in Patients with Hematologic Malignancies Undergoing Haploidentical Donor Transplant. (TCT-ASTCT-CIBMTR 2024) - "We enrolled to three cohorts: 1 - Fludarabine (Flu) 40mg/m2 x4, Mel140mg/m2, TBI200cGy, 2 - FluMel100TBI400, and to an amended cohort 3 - FluMel70TBI200. In younger pts, 140mg/m2 was tolerable with excellent disease control. In older pts, 70mg/m2 enabled reliable engraftment, low rates of severe GVHD, low NRM, and favorable disease control."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Lymphoma • Oncology • Transplantation

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

New P2 trial • Post-transplantation • Pre-transplantation • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation