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Also known as Darvadstrocel, Alofisel
Cx601 · 5 trials · 4 indications
Combined Remission of perianal fistulising Crohn's disease defined as the clinical assessment of closure of all treated external openings (EO) that were draining at baseline despite gentle finger compression at week 24, and absence of collections \> 2 cm of the treated perianal fistulas confirmed by centrally blinded MRI assessment by week 24.
Combined remission was defined as the closure of all treated external openings that were draining at baseline despite gentle finger compression and absence of collection(s) \>2 cm (in at least 2 dimensions) of the treated perianal fistula(s) confirmed by blinded central magnetic resonance imaging (MRI) assessment. Percentages are rounded off to whole number at the nearest decimal.
An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered an investigational medicinal product; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with a study intervention or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the study intervention or medicinal product.
TEAE is defined as: any adverse event emerging/manifesting at or after the initiation of treatment with a study intervention/medicinal product or any existing event that worsens in either intensity/frequency following exposure to the study intervention/medicinal product. Serious adverse event (SAE) is an untoward medical occurrence, significant hazard, contraindication, side effect/precaution that at any dose: results in death, is life-threatening, required in-patient hospitalization/prolongation of existing hospitalization, results in persistent/significant disability/incapacity, is a congenital anomaly/birth defect or is medically significant.
AESIs are AEs that are not solicited local or systemic AEs, they are predefined AEs that require close monitoring and prompt reporting to the sponsor. Protocol pre-specified AESIs included immunogenicity/allo-immunoreactions, tumorigenicity, ectopic tissue formation and fistula/abscess. In addition, ad hoc AESIs of anaphylactic reaction, hypersensitivity, and malignancy.
Combined remission of perianal fistulising CD is defined as the clinically confirmed closure of all treated external openings that were draining at Screening despite gentle finger compression, and absence of collections \>2 cm in the treated fistulas, confirmed by central magnetic resonance imaging (MRI) assessment at Week 24 visit. In case of missing values, last observation carried forward (LOCF) method was applied. Percentages are rounded off to the nearest decimal point.
| Arm | Type | Description |
|---|---|---|
| Treatment Arm | EXPERIMENTAL | Cx601 is a cell suspension in aseptic buffered solution containing human expanded adipose-derived stem cells (eASCs) of allogeneic origin in disposable vials with no preservative agents. The cells will be given at a dose of 120 million cells (5 million cells / mL) for intralesional injection. |
| Placebo-control group | PLACEBO_COMPARATOR | Placebo (saline solution) will be given also for intralesional injection at the same quantity (volume, 24 mL) and following the same schedule. |
| Placebo | PLACEBO_COMPARATOR | Placebo (saline) 24 milliliters (mL) was administered once by local injection. |
| Cx601 | EXPERIMENTAL | Cx601 expanded adipose-derived stem cells (eASCs) 120 million cells (5 million cells per mL) was administered once by local injection. |
| Darvadstrocel | EXPERIMENTAL | Participants who received a single dose of darvadstrocel, 120 million cells, intralesionally previously in the ADMIRE-CD II study were observed for efficacy and safety. No drug was administered in this study. |
| Darvadstrocel 24 mL | EXPERIMENTAL | Darvadstrocel (Cx601) 24 mL suspension of 120 million cells of expanded allogeneic adipose-derived stem cells (eASC) as an intralesional injection, once on Day 1. |
| Name | Type | Description |
|---|---|---|
| Cx601 | OTHER | 120 million cells administered by intralesional injection. |
| Saline solution | OTHER | 24 mL saline solution by intralesional injection |
| Placebo | OTHER | Cx601 placebo-matching eASCs intralesional injection. |
| Darvadstrocel | BIOLOGICAL | Allogenic expanded adipose-derived stem cells (eASCs) 5 million cells/ml - suspension for injection darvadstrocel received in previous ADMIRE-CD II study. No drug administration in this study. |
Inclusion Criteria: The reference population will consist of patients with perianal fistulising Crohn´s disease refractory to at least one of the following treatments: antibiotics, immunosuppressants or anti-tumor necrosis factor (TNF). Naïve patients are excluded, and those patients refractory to ...
Top 15 of 17 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Sanofi SA Sponsored ADR | SNY | 5 | PHASE3 | Duvakitug, balinatunfib, Brivekimig |
| AbbVie, Inc. | ABBV | 16 | PHASE3 | Upadacitinib, Risankizumab, Lutikizumab, Trosunilimab, ABBV-8736 |
| Eli Lilly and Company | LLY | 7 | PHASE3 | Mirikizumab, Tirzepatide, LY4395089, MORF-057 |
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 15 | PHASE3 | Vedolizumab, Zasocitinib, Darvadstrocel |
| Johnson & Johnson | JNJ | 24 | PHASE3 | Ustekinumab, Guselkumab, JNJ-78934804, Golimumab, Icotrokinra |
| Merck & Co., Inc. | MRK | 2 | PHASE3 | Tulisokibart |
| AstraZeneca PLC | AZN | 2 | PHASE2 | AZD7798 |
| Spyre Therapeutics, Inc | SYRE | 2 | PHASE2 | SPY001, SPY002, SPY003 |
| Disc Medicine, Inc. | IRON | 1 | PHASE2 | DISC-0974 |
| Abivax SA Sponsored ADR | ABVX | 1 | PHASE2 | Obefazimod |
| Shattuck Labs, Inc. | STTK | 1 | PHASE2 | SL-325 |
| AgomAb Therapeutics NV ADR | AGMB | 2 | PHASE2 | Ontunisertib |
| Hoffmann-La Roche | RHHBY | 1 | PHASE2 | Afimkibart |
| Pfizer Inc. | PFE | 1 | PHASE2 | Etrasimod |
| Palisade Bio, Inc. | PALI | 1 | PHASE1 | PALI-2108 |
Cx601 is being developed for the treatment of complex perianal fistulas in patients with Crohn's disease. It is an allogeneic expanded adipose-derived stem cell therapy given to adults 18 years and older. The clinical program focuses on this specific fistulizing complication of Crohn's disease rather than Crohn's disease broadly.
Cx601 is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda holds the development program for this asset in Crohn's disease associated with complex perianal fistulas.
Cx601 has completed Phase 3 clinical development. Three trials have been completed, including two Phase 3 studies and one Phase 1 study, with no trials currently active. The program has not been described as approved, so Cx601 remains an investigational therapy in clinical development.
Cx601 has been studied in three completed trials. The largest, NCT03279081, was a Phase 3 study of Cx601 in adults with complex perianal fistulas and Crohn's disease that enrolled 568 participants across the United States, Canada, Europe, Israel, and Puerto Rico. NCT01541579 was a Phase 3 study in Europe with 278 participants, and NCT01372969 was an earlier Phase 1 study in Spain with 24 participants.
The Cx601 trials were randomized, double-blind, and placebo-controlled. The Phase 3 study NCT03279081 enrolled 568 adults with complex perianal fistulas and Crohn's disease, and the Phase 3 study NCT01541579 enrolled 278 adults. Across the three completed trials, total enrollment was 870 participants, all aged 18 years or older.