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Adjusted LOA
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Motixafortide · 1 trial · 1 indication
* All toxicities will be graded using NCI-CTCAE Version 5.0 * A DLT is an event occurring during the DLT period of 28 days following administration of either motixafortide and/or natalizumab that is considered to be at least possibly, probably or definitely related to study treatment by the investigator, and that meets the criteria below: * Hematologic criteria * Any Grade 5 adverse event * Any Grade 4 adverse event, excluding Grade 4 hemolysis, bilirubin increase, leukocytosis, erythrocytosis, thrombocytosis, anemia, leukopenia, or febrile neutropenia * Non-hematologic criteria * Any Grade 4 or 5 adverse event. * Any Grade 3 or higher arterial or venous thromboembolic event * Any Grade 2 or 3 adverse event that does not resolve within 4 weeks; with the exception of \<grade 2 injection site reactions.
| Arm | Type | Description |
|---|---|---|
| Motixafortide followed by Motixafortide + Natalizumab | EXPERIMENTAL | * Consenting and eligible patients will receive a single subcutaneous injection of motixafortide, followed by leukapheresis. Patient will then be followed for 8 weeks for adverse event monitoring. * Following the 8-week monitoring period, patients will receive a single IV infusion natalizumab, then approximately 32 hours later, a single subcutaneous injection of motixafortide, followed by leukapheresis. Patients will then be followed for 8 weeks for adverse event monitoring. |
| Name | Type | Description |
|---|---|---|
| Motixafortide | DRUG | Motixafortide is to be administered as a subcutaneous injection at a dose of 1.25 mg/kg |
| Natalizumab | DRUG | Natalizumab will be administered as an IV infusion at a flat dose of 300 mg |
| Leukapheresis | PROCEDURE | Leukapheresis consisting of a 1 Blood Volume procedure |
Inclusion Criteria: * Adult patients at least 18 years old * Diagnosis of sickle cell disease (hemoglobin SS or Sβ0 genotype) * Receiving automated RBC exchanges via apheresis-capable central venous access or willing to have apheresis-capable venous access placed. * Able to hold hydroxyurea, voxelo...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 5 | PHASE3 | Etavopivat Low dose |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, 20vPCV |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Agios Pharmaceuticals, Inc. | AGIO | 2 | PHASE2 | Mitapivat |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470, Famotidine, Pantoprazole |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
Motixafortide is an investigational small molecule being studied for use in Sickle Cell Disease. It is being evaluated in combination with natalizumab to mobilize CD34+ hematopoietic stem cells for gene therapies in sickle cell disease. The drug is currently in Phase 1 clinical development and is not yet approved.
Motixafortide is being developed by Biogen Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BIIB. The drug is currently in Phase 1 clinical development for Sickle Cell Disease.
Motixafortide is in Phase 1 clinical development. It has completed one Phase 1 trial for Sickle Cell Disease, and the drug remains investigational. It has not been approved by regulatory authorities.
Motixafortide has been studied in one completed Phase 1 clinical trial, identified as NCT05618301. The trial evaluated Motixafortide and natalizumab to mobilize CD34+ hematopoietic stem cells for gene therapies in Sickle Cell Disease. The study enrolled 10 participants in the United States.
No alternative names for Motixafortide have been reported. The drug is known by its generic name Motixafortide and is being developed by Biogen Inc. for Sickle Cell Disease.