Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ITU512 · 1 trial · 1 indication
Number of participants with adverse events (AEs) and serious adverse events (SAEs), including changes in vital signs, electrocardiograms (ECGs) and laboratory values qualifying and reported as AEs.
Number of participants with dose discontinuation due to AEs
Number of participants with adverse events (AEs) and serious adverse events (SAEs), including changes in vital signs, electrocardiograms (ECGs) and laboratory values qualifying and reported as AEs.
Number of participants with dose interruptions or reductions of ITU512
Dose intensity of ITU512 is computed as the ratio of actual cumulative dose received and actual duration of exposure
Assessment of fetal hemoglobin expression by measuring fetal hemoglobin (HbF)% by high-performance liquid chromatography (HPLC) assay
| Arm | Type | Description |
|---|---|---|
| Part 1A | EXPERIMENTAL | Part 1A in healthy participants |
| Part 1B | EXPERIMENTAL | Part 1B in healthy participants |
| Part 1C | EXPERIMENTAL | Part 1C in healthy participants |
| Part 2A | EXPERIMENTAL | Part 2A in patients with sickle cell disease |
| Part 2B | EXPERIMENTAL | Part 2B in patients with sickle cell disease |
| Part 2C | EXPERIMENTAL | Optional extension in patients with sickle cell disease |
| Name | Type | Description |
|---|---|---|
| ITU512 | DRUG | ITU512 is an investigational, oral, low molecular weight (LMW) compound. |
| Placebo | DRUG | An inactive substance that looks like and is given the same way as ITU512. The effect(s) of ITU512 will be evaluated against the placebo. Placebos are designed as a control and to have no real effect. |
Key Inclusion Criteria: Part 1 (Healthy participants) * Healthy male participants and female participants of non-childbearing potential between 18-55 years of age * In good health as determined by the investigator's assessment of medical history, physical examination, vital signs, ECG, and laborat...
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 |
ITU512 is an investigational small molecule drug being developed by Novartis AG for sickle cell disease. It is in Phase 1 clinical development and has not been approved by the FDA. The program includes a combined Phase I/II study that is currently recruiting participants.
ITU512 is being studied for the treatment of sickle cell disease, a hematologic disorder. The clinical program is evaluating the drug in both healthy participants and patients with sickle cell disease. It remains investigational and is not approved for any indication.
ITU512 is being developed by Novartis AG, which trades on the New York Stock Exchange under the ticker NVS. Novartis is the sponsor of the clinical program evaluating the drug in sickle cell disease.
ITU512 is in Phase 1 clinical development. The ongoing study is a combined Phase I/II trial, and no Phase 3 or registrational studies have been initiated. The drug is investigational and has not received FDA approval.
ITU512 is being evaluated in the Phase I/II study NCT06546670, titled A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease. The trial is recruiting, with an enrollment of 161 participants across the United States, Brazil, and Turkey, and includes participants aged 12 years and older.
Yes. The Phase I/II study of ITU512, NCT06546670, is a randomized, double-blind, controlled trial. It enrolls 161 participants, including healthy volunteers and patients with sickle cell disease, across sites in the United States, Brazil, and Turkey.