Recent Updates
Recently added Catalysts

ION251

Phase 1

Relapsed Multiple Myeloma | Small molecule | Oncology |Ionis Pharmaceuticals, Inc.|Last Updated: Oct 31, 2024

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment23

FDA Designations

No designations recorded

Clinical trial landscape

ION251 · 1 trial · 2 indications

Phase 1 1
NCT04398485A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple MyelomaRelapsed Multiple Myeloma
COMPLETED23 Analytics
PHASE1COMPLETED
A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma
Relapsed Multiple MyelomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Maximum-Tolerated Dose (MTD)
Up to 28 days from the last dose of study drug in Cycle 1 (each cycle is 28 days)

MTD is defined as the maximum dose at which ≤ 1 of 3 evaluable participants experiences a dose-limiting toxicity (DLT) within Cycle 1 and there are 2 of 3 or 2 of 6 evaluable participants in the next higher-dose level experiencing a DLT within Cycle 1. If no dose in the dose-escalation has 2 of 3 or 2 of 6 evaluable participants experiencing a DLT, the highest dose level is considered the MTD

Recommended Phase 2 Dose (PR2D)
Up to 28 days from the last dose of study drug

RP2D is chosen based on the dose response and exposure-response analyses of the pooled clinical PK, PD, safety results, and anti-myeloma activity from both Part 1 and Part 2

Secondary Endpoints

Safety and Tolerability as Measured by the Incidence of TEAEs
Up to 28 days from the last dose of study drug
Incidence of Abnormal Laboratory Values and Vital Signs
Up to 28 days from the last dose of study drug
Cmax: Maximum Observed Concentration ION251
From Baseline up to 28 days from the last dose of study drug
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ION251EXPERIMENTALIn Part 1, the dose escalation phase, increased amounts of ION251 will be administered at multiple time points by intravenous (IV) infusion during 28-day cycles. In Part 2, the determined RP2D of ION251 will be administered at multiple time points by IV infusion.

Interventions

NameTypeDescription
ION251DRUGION251 administered by IV infusion
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: 1. Aged ≥ 18 years at the time of informed consent 2. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 3. Measurable multiple myeloma (MM) 4. In need of systemic treatment for MM and either is refractory to or has failed treatment with, is intolerant to or ...

Countries:United States
Unlock Eligibility Criteria

Competitive Landscape -Multiple Myeloma 221 trials (matched to "Relapsed Multiple Myeloma")

Top 20 of 25 competitors

CompanyTickerTrialsLead PhaseDrugs
AbbVie, Inc.ABBV16PHASE3Pomalidomide, Dexamethasone, Venetoclax
Bristol-Myers Squibb CompanyBMY18PHASE3Iberdomide, Lenalidomide
Takeda Pharmaceutical Co. Ltd. Sponsored ADRTAK5PHASE3IGI, 10%
GSK plc Sponsored ADRGSK17PHASE3Belantamab mafodotin, Pomalidomide, Dexamethasone, Bortezomib
Johnson & JohnsonJNJ28PHASE3Talquetamab, Pomalidomide, Teclistamab, Elotuzumab, Dexamethasone
Regeneron Pharmaceuticals, Inc.REGN11PHASE3Linvoseltamab, Carfilzomib, Daratumumab, Dexamethasone, Pomalidomide
Pfizer Inc.PFE11PHASE3Elranatamab, Lenalidomide
Sanofi SA Sponsored ADRSNY17PHASE3Isatuximab, Dexamethasone, Pomalidomide, Montelukast, Paracetamol/ Acetaminophen
AstraZeneca PLCAZN5PHASE3AZD0120, Daratumumab, Carfilzomib, Dexamethasone, Bortezomib
Gilead Sciences, Inc.GILD3PHASE3Anitocabtagene Autoleucel, Cyclophosphamide, Fludarabine, Pomalidomide, Bortezomib
Karyopharm Therapeutics, Inc.KPTI6PHASE3Selinexor, Elotuzumab, Pomalidomide, Dexamethasone
Grifols, S.A. Sponsored ADR Class BGRFS1PHASE3Xembify
BioLineRX Ltd. Sponsored ADRBLRX1PHASE3BL-8040/kg, G-CSF
C4 Therapeutics, Inc.CCCC3PHASE2Cemsidomide, Dexamethasone
Cellectar Biosciences, Inc.CLRB1PHASE2Iopofosine I 131 single dose, Iopofosine I 131 fractionated dose
GeoVax Labs, Inc.GOVX1PHASE2COVID-19 Vaccine, Synthetic MVA-based SARS-CoV-2 Vaccine GEO-CM04S1
Autolus Therapeutics Plc Sponsored ADRAUTL1PHASE2AUTO CAR T cell therapy
Incyte CorporationINCY2PHASE1Ruxolitinib, Lenalidomide, Methylprednisolone
Moderna, Inc.MRNA2PHASE1mRNA-2808
BeOne Medicines Ltd. Sponsored ADRONC1PHASE1Sonrotoclax, Dexamethasone, Carfilzomib, Daratumumab, Pomalidomide
Unlock Competitive Intelligence

Frequently asked questions about ION251

What is ION251 used for?

ION251 is an investigational drug being studied for the treatment of relapsed or refractory multiple myeloma, a type of blood cancer. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes ION251?

ION251 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS.

What phase is ION251 in?

ION251 is in Phase 1 clinical development. A Phase 1 study has been completed, and the drug remains investigational, meaning it is not yet approved for commercial use.

What clinical trials is ION251 in?

ION251 has been studied in a Phase 1 clinical trial with the identifier NCT04398485, titled 'A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma.' This trial enrolled 23 participants in the United States and has been completed.

Is ION251 a small molecule?

Yes, ION251 is classified as a small molecule therapeutic. It is being investigated for its potential role in treating relapsed or refractory multiple myeloma.