Experimental Treatments for Multiple Myeloma

Unless otherwise indicated, these treatments are being evaluated in relapsed and/or relapsed-refractory myeloma.

Name

Company

Description

Phase III 

Aplidin® (plitidepsin)

PharmaMar, SA

Marine-derived antitumor agent being evaluated in combination with dexamethasone (dex) in a trial being conducted in the US, Europe, and Australia, which has completed enrollment LEARN MORE

Denosumab (AMG 162)

Amgen

Human monoclonal antibody that targets the receptor activator of nuclear factor kappa B ligand (RANKL) and inhibits bone destruction; approved as Xgeva® for use in bone disease in solid tumors and as Prolia® for osteoporosis in women after menopause

Elotuzumab (HuLuc63; humanized anti-CS1 monoclonal antibody)

Bristol-Myers Squibb/Abbott

Monoclonal antibody that binds to CS1, a cell surface protein that is highly expressed on myeloma cells; being evaluated in combination with Revlimid-dex in phase III studies in relapsed or refractory myeloma (enrollment complete) and in previously untreated disease, as well as alone and with other agents in early-stage trials LEARN MORE

Ixazomib citrate (MLN9078)

Millennium: The Takeda Oncology Company

Oral second-generation proteasome inhibitor; being evaluated combination with Revlimid-dex in phase III studies in relapsed and/or refractory myeloma and in previously untreated patients, as well as alone and with other agents in early-stage trials. LEARN MORE

Masitinib (AB1010)

AB Science

Oral anticancer agent (tyrosine kinase inhibitor) being evaluated in combination with Velcade-dex in a phase III study being conducted in the US and Europe

Panobinostat (LBH589)

Novartis

Deacetylase (DAC) inhibitor; being evaluated in combination with Velcade-dex in a phase III study in relapsed myeloma (enrollment complete), and alone and with other agents in early-stage trials LEARN MORE

Zolinza® (vorinostat, SAHA [suberoylanilide hydroxamic acid])

Merck

Histone deacetylase (HDAC) inhibitor, FDA-approved for cutaneous T-cell lymphoma; although Merck is not continuing development of Zolinza in myeloma, it is still being evaluated in combination with other agents in early-stage clinical trials, and could potentially be used off-label. LEARN MORE

Phase II

Alisertib (MLN8237)

Millennium: The Takeda Oncology Company

Novel agent (Aurora A kinase inhibitor) being evaluated in combination with Velcade in a study being facilitated by the MMRC; Phase I results show the combination is active despite the lack of a steroid

AT7519 (AT7519M)

Astex Therapeutics

Cyclin-dependent kinase (CDK) inhibitor being evaluated alone and combination with Velcade in a study being facilitated by the MMRC and MMRF Biotech Investment Award program (completed enrollment)

ARRY-520

Array BioPharma

Agent that inhibits kinesin spindle protein (KSP); being evaluated alone (enrollment complete), as well as in combination with Kyprolis, and with Velcade-dex in a study being facilitated by the MMRC; initial results with Kyprolis show a manageable side effect profile and early signs of activity in patients no longer responding to Velcade

BHQ880

Novartis

Monoclonal antibody (anti-DKK1) being evaluated in patients with untreated myeloma and impaired kidney function, as well as patients with high-risk smoldering myeloma (SMM; the agent increased bone strength in 60% of patients); enrollment completed in both trials

BT-062

Biotest AG

Anti-CD138 monoclonal antibody/cytotoxic agent compound that has demonstrated activity in early testing; being evaluated alone (enrollment complete) and in combination with Revlimid-dex

Daratumumab (HuMax-CD38)

Genmab

Human monoclonal antibody that targets the CD38 molecule, which is highly expressed on the surface of myeloma cells; being evaluated alone and in combination with Revlimid-dex; preliminary results show some evidence of single-agent activity

Dinaciclib (SCH 727965, MK-7965)

Merck/Ligand Pharmaceuticals

Cyclin-dependent kinase (CDK) inhibitor; being evaluated in combination with Velcade-dex in a Phase I trial; anti-myeloma activity observed in Phase II testing

Ibrutinib (PCI-32765)

Pharmacyclics

Anticancer agent (Bruton’s tyrosine kinase inhibitor) that targets B cells

Imetelstat (GRN163L)

Geron Corporation

Telomerase inhibitor that induces apoptosis and inhibits myeloma cell growth; enrollment completed

IPH2101 (IPH21, IPH2102)

Innate Pharma/Bristol-Myers Squibb

Monoclonal antibody (anti-KIR) that enhances killing of myeloma cells by natural killer (NK) cells; being evaluated in combination with other agents, as well as in smoldering myeloma; studies have completed enrollment. Interim results of a Phase I trial in combination with Revlimid are promising

KW-2478

Kyowa Hakko Kirin Co., Ltd.

Heat-shock protein (HSP) 90 inhibitor being evaluated in combination with Velcade (completed enrollment)

Melflufen

Oncopeptides AB

A novel, more potent form of melphalan that is preferentially targeted to tumor cells

Siltuximab (CNTO-328)

Janssen Biotech, Inc.

Anti-IL-6 monoclonal antibody being evaluated in patients with high-risk smoldering myeloma in a phase II trial, and in combination with Revlimid-Velcade-dex in transplant-eligible newly diagnosed myeloma in a phase I trial (enrollment completed)

Tabalumab (LY2127399)

Eli Lilly

Antibody directed against a protein that activates B cells; being tested in combination with Velcade-dex; results of a Phase I study show the combination to be safe, with efficacy demonstrated

TBL-12

Unicorn Pacific Corporation

Sea cucumber extract that displays immunomodulatory properties; being evaluated in untreated, asymptomatic myeloma (enrollment completed)

Tivantinib (ARQ 197)

ArQule/National Cancer Institute

Anti-cancer agent that inhibits an enzyme (c-Met) involved in many cancer cell activities

Treanda® (bendamustine hydrochloride; known as Ribomustin® in Germany)

Cephalon, a subsidiary of Teva Pharmaceuticals

Alkylating agent FDA-approved for use in treating leukemia and lymphoma; approved in Germany for use in myeloma; being evaluated as part of various combination therapies for use in newly diagnosed and relapsed and/or refractory disease LEARN MORE

Vidaza® (azacitidine)

Celgene Corporation

Cytotoxic drug FDA-approved for the treatment of myelodysplastic syndromes (MDS) being evaluated in combination with Revlimid-dex in relapsed or refractory myeloma and in combination with Revlimid prior to stem cell transplant

Phase I

ABT-199

AbbVie (formerly Abbott) and Genentech

Oral agent (Bcl-2 inhibitor) that has shown activity against myeloma cells with the t(11;14) translocation ; being evaluated in combination with Velcade-dex in an MMRC-facilitated study, as well as a single agent

ALT-801

Altor Bioscience Corporation

Anti-tumor immunotherapeutic protein (T-cell receptor/IL-2 fusion protein)

Amrubicin

Celgene Corporation

Chemotherapy drug approved in Japan for small-cell lung cancer; being evaluated in combination with Revlimid-dex; initial results show no unexpected toxicities

BMS-936564 (MDX-1338)

Bristol-Myers Squibb

Monoclonal antibody directed against a cell marker (CXCR4) normally found on immune cells and in increased levels in various cancer cells; induces tumor cell apoptosis; being evaluated alone and in combination with Revlimid-dex or Velcade-dex

Cometriq (cabozantanib, XL184)

Exelixis, Inc

Novel agent (tyrosine kinase inhibitor) that reduces tumor growth, metastasis, and growth of blood vessels; FDA approved to treat a form of thyroid cancer; being evaluated in patients with and without myeloma bone disease

CUDC-907

Curis, Inc.

Novel agent (phosphoinositide 3-kinase [PI3K] and histone deacetylase [HDAC] inhibitor) that inhibits cancer cell survival and growth

CX-4945

Cylene Pharmaceuticals

Oral protein kinase (CK2) inhibitor

DFRF4539A

Genentech

Monoclonal antibody

DKN-01

Dekkun Corporation

Monoclonal antibody (anti-DKK1); being evaluated in combination with Revlimid-dex

Erivedge® (vismodegib, GDC-0449)

Genentech

Synthetic Hedgehog pathway inhibitor being evaluated in patients with high-risk myeloma following stem cell transplant (enrollment completed); FDA approved for use in treating a type of skin cancer (basal cell carcinoma)

Folotyn® (pralatrexate)

Allos Therapeutics, a subsidiary of Spectrum Pharmaceuticals

Chemotherapy drug approved for use in peripheral T-cell lymphoma that is being evaluated in combination with Velcade

Ganetespib (STA-9090)

Synta Pharmaceuticals

Synthetic, small molecule inhibitor of heat shock protein 90 (Hsp90); being evaluated with and without Velcade in a trial being facilitated by the MMRC and the MMRF Biotech Investment Award program

GSK2110183

GlaxoSmithKline

Oral anticancer agent (AKT inhibitor) being evaluated in combination with Velcade-dex in a trial being facilitated by the MMRC

LGH447

Novartis

Oral anti-cancer agent

Linsitinib (ASP7487, OSI-906)

Astellas Pharma Inc.

Agent that triggers cancer cell death (insulin-like growth factor-1 [IGF-1] receptor inhibitor); MMRC and MMRF Clinical Fund-facilitated trial is evaluating the agent in combination with Velcade-dex

Lorvotuzumab mertansine (IMGN901, BB-10901)

ImmunoGen

Monoclonal antibody directed against the CD56 marker found on myeloma cells, coupled with a cancer drug; being evaluated in combination with Revlimid-dex (completed enrollment); activity demonstrated, including in patients previously treated with IMiDs +/- Velcade

Marizomib (NPI-0052)

Nereus Pharmaceuticals

Novel proteasome inhibitor being evaluated in an MMRC-facilitated study that has shown promising early safety data LEARN MORE

MEDI-551

MedImmune LLC

Monoclonal antibody (anti-CD19) that will be evaluated in combination with Revlimid-dex in a trial set to begin enrollment.

Nivolumab (BMS-936558)

Bristol-Myers Squibb

Monoclonal antibody (anti-programmed death-1 [PD-1])

Oncolytic measles virus (MV-NIS)

Mayo Clinic

Genetically engineered measles virus being administered with or without cyclophosphamide

Oprozomib (ONX 0912)

Onyx Pharmaceuticals, Inc.

Oral proteasome inhibitor that is similar to Kyprolis; being evaluated in combination with dex in relapsed and/or refractory myeloma; trial evaluating oprozomib in combination with Revlimid-dex in newly diagnosed disease is set to begin

PRLX 93936

Prolexys Pharmaceuticals

Novel agent that targets the RAS protein

PVX-410

OncoPep, Inc.

Therapeutic cancer vaccine targeted against four proteins found on the surface of myeloma cells; being tested in asymptomatic (smoldering) myeloma

Rocilinostat (ACY-1215)

Acetylon Pharmaceuticals, Inc.

Oral histone deacetylase (HDAC) inhibitor being evaluated alone and in combination with Velcade-dex and Revlimid-dex

SAR650984

Sanofi

Antibody directed against the CD38 marker on certain cancer cells; being evaluated as a single agent, and in combination with Revlimid in a study being facilitated by the MMRC

SNS01-T

Senesco Technologies, Inc.

Anticancer agent targeted to B cells that induces apoptosis

TH-302

Threshold Pharmaceuticals

Anticancer drug that is converted to its active form in areas where there is low oxygen (hypoxia), such as within tumors or in the bone marrow where myeloma cells are found; being evaluated alone and in combination with Velcade