Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Aes-103 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Aes-103 300 mg to 1000 mg (Group A) | EXPERIMENTAL | Group A will consist of six subjects receiving a single dose of either a low dose of Aes-103 (300 mg) or placebo without food. After a minimum of a 1- to 2-week washout period and evaluation of blinded safety data, subjects initially randomized to Aes-103 will receive a second, higher dose of Aes-103 (1,000 mg), and subjects initially randomized to placebo will receive a second dose of placebo without food. |
| Aes-103 2000 mg to 4000 mg (Group B) | EXPERIMENTAL | Group B will consist of six subjects receiving an initial dose of either Aes 103 (2,000 mg) or placebo without food. After a minimum of a 1- to 2-week washout period and evaluation of blinded safety data, subjects initially randomized to Aes-103 will receive a second, higher dose of Aes-103 (4,000 mg), and subjects initially randomized to placebo will receive a second dose of placebo without food. |
| Top Dose Expansion (Group C) | EXPERIMENTAL | Once the top dose (i.e., highest tolerated) of Aes-103 has been determined, the size of this group will be expanded with an additional six subjects (Group C) for a total of 12 at that dose, distributed so that six subjects receiving hydroxyurea (HU) and six subjects not receiving HU (for the past 6 months) will receive study drug (five receiving Aes-103 and one receiving placebo in each of the HU and non-HU treated cohorts). This total of 12 subjects includes the initial six subjects who received the highest dose in the study plus six Group C subjects. These subjects will receive a single dose of the top dose of Aes-103 or placebo without food. After a minimum of a 1- to 2-week washout period and evaluation of blinded safety data, subjects initially randomized to Aes-103 will receive a second dose of the top dose of Aes-103 and subjects initially randomized to placebo will receive a second dose of placebo; all subjects will be administered a pre-dose, high fat, high protein meal. |
| Name | Type | Description |
|---|---|---|
| Aes-103 | DRUG | 300 mg Aes-103 powder reconstituted in orange juice to a volume of 100 mL per single dose for oral administration. |
| Placebo | DRUG | Orange juice vehicle, a solution that is highly similar in appearance to the Aes-103 orange juice solution. |
Inclusion Criteria: * Be male or female, aged 18-65 years old, inclusive * Have sickle cell disease (SCD) (hemoglobin SS) without hospitalization for pain crises in the 30 days before screening or for any SCD complications on more than two occasions in the past 12 months; subjects are allowed conco...
Top 12 of 13 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 6 | PHASE3 | Etavopivat |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab, OTQ923, ITU512 |
| Agios Pharmaceuticals, Inc. | AGIO | 3 | PHASE3 | Mitapivat, Tebapivat |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, rilzabrutinib |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Travere Therapeutics, Inc. | TVTX | 1 | PHASE3 | L-Citrulline |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470 |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
Aes-103 is an investigational small molecule being studied for sickle cell disease, a hematology condition. It has been evaluated in a Phase 1 clinical trial in adults with stable sickle cell disease. It is not an approved therapy and remains in clinical development.
Aes-103 is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. The company sponsored the clinical research conducted on the compound in sickle cell disease.
Aes-103 is in Phase 1 clinical development. Its single recorded trial, NCT01597401, was a Phase 1 study that has been completed. The drug is investigational and has not been approved for any indication.
Aes-103 has been studied in one clinical trial, NCT01597401, titled a single dose study of the safety, blood levels and biological effects of Aes-103 compared to placebo in subjects with stable sickle cell disease. The trial was completed and enrolled 19 participants in the United States.
NCT01597401 was a randomized, double-blind, controlled Phase 1 trial of a single dose of Aes-103 compared with placebo in adults with stable sickle cell disease. It enrolled 19 participants aged 18 years and older in the United States and has been completed.