Approval Probability
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Also known as PAT-1251
GB2064 · 2 trials · 2 indications
Incidence and severity of adverse events as reported by investigators
Determination of adverse events will include assessment of vital signs, ECG, clinical laboratory and physical examination
| Arm | Type | Description |
|---|---|---|
| GB2064 | EXPERIMENTAL | GB2064 will be administered orally as 4 x 250 mg tablets twice a day. |
| PAT-1251 Single Dose | EXPERIMENTAL | Oral solution of PAT-1251, 150 - 4000 mg administered once |
| Placebo Single Dose | PLACEBO_COMPARATOR | Matching placebo solution administered once |
| PAT-1251 Multiple Dose | EXPERIMENTAL | Oral tablet(s) of PAT-1251 up to 2000 mg administered daily for 7 days |
| Placebo Multiple Dose | EXPERIMENTAL | Matching placebo tablets administered daily for 7 days |
| Name | Type | Description |
|---|---|---|
| GB2064 | DRUG | GB2064 (formerly PAT-1251) is a high-affinity, selective, mechanism-based, small molecule inhibitor of LOXL2, administered twice a day |
| PAT-1251 | DRUG | - |
| Placebo | DRUG | - |
Inclusion Criteria: Participants must satisfy all of the following criteria at the Screening visit: 1. Adult male or female participants ≥ 18 years of age at enrolment: 1. Female participants may be of non-childbearing potential defined as permanently sterile or postmenopausal, or female parti...
GB2064 is an oral small molecule drug candidate being developed by Damora Therapeutics, Inc. for myelofibrosis, a bone marrow cancer. It is also known as PAT-1251. The drug is in clinical development and has been studied in both healthy adult volunteers and participants with myelofibrosis.
GB2064 is being evaluated as a treatment for myelofibrosis, a condition in which scar tissue builds up in the bone marrow. It is an investigational therapy and is not yet approved for any indication. Clinical testing has focused on its safety, tolerability, pharmacokinetics and pharmacodynamics in this patient population.
GB2064 targets LOXL2, an enzyme. LOXL2 belongs to the lysyl oxidase family and is involved in crosslinking of the extracellular matrix. By inhibiting this enzyme, GB2064 is designed to interfere with the fibrotic process that characterizes myelofibrosis, where excess scar tissue accumulates in the bone marrow.
GB2064 is being developed by Damora Therapeutics, Inc., which trades under the ticker DMRA. The company is the sponsor of the clinical program evaluating the drug in healthy volunteers and in patients with myelofibrosis.
GB2064 is in Phase 1 development. A Phase 1 study in healthy adult subjects has been completed, and a Phase 2 study in participants with myelofibrosis is active but not recruiting. The drug remains investigational and has not been approved by the FDA for any indication.
GB2064 has been studied in two clinical trials. NCT02852551 was a completed Phase 1 study of single and multiple doses in 78 healthy adults in the United Kingdom. NCT04679870 is an active Phase 2 study evaluating oral GB2064 in 21 participants with myelofibrosis across the United States, Australia, Germany and Italy.
Yes, GB2064 is also known as PAT-1251. Both names refer to the same oral small molecule LOXL2 inhibitor being developed by Damora Therapeutics, Inc. The earlier Phase 1 healthy volunteer study used the PAT-1251 designation, while the myelofibrosis study uses the GB2064 name.