Last reviewed · How we verify
IB1001
IB1001 is an intrabone injection therapy designed to deliver therapeutic agents directly into the bone marrow microenvironment.
IB1001 is an intrabone injection therapy designed to deliver therapeutic agents directly into the bone marrow microenvironment. Used for Multiple myeloma (in development).
At a glance
| Generic name | IB1001 |
|---|---|
| Also known as | N-Acetyl-L-Leucine, Recombinant factor IX (rFIX), IXINITY, Recombinant factor IX |
| Sponsor | IntraBio Inc |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
IB1001 utilizes IntraBio's proprietary intrabone delivery platform to administer biologics or small molecules directly into bone tissue, potentially bypassing systemic circulation and achieving high local concentrations. This approach may enhance therapeutic efficacy while reducing systemic exposure and associated toxicities for bone-related or hematologic conditions.
Approved indications
- Multiple myeloma (in development)
Common side effects
Key clinical trials
- Pivotal Study of N-acetyl-L-leucine for CACNA1A (PHASE3)
- N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) (PHASE2)
- A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) (PHASE3)
- A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (PHASE3)
- Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B (PHASE3)
- N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) (PHASE2)
- N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) (PHASE2)
- Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B (PHASE2, PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- IB1001 CI brief — competitive landscape report
- IB1001 updates RSS · CI watch RSS
- IntraBio Inc portfolio CI