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GB2064

Phase 2

Myelofibrosis | Small molecule | Oncology |Damora Therapeutics, Inc.|Trials Updated: Apr 3, 2024

GB2064 development status

Highest phase Phase 2
Registered trials 1 across 1 sponsor since Dec 2020

GB2064 target and mechanism

Molecular targetLOXL2
Target classEnzyme
ModalitySmall molecule

Also known as PAT-1251

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment21

FDA Designations

No designations recorded

GB2064 clinical trials

GB2064 · 1 trial · 1 indication

Phase 2 1
NCT04679870A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral GB2064 in Participants With MyelofibrosisMyelofibrosis
ACTIVE NOT_RECRUITING21 Analytics
PHASE2ACTIVE NOT_RECRUITING
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral GB2064 in Participants With Myelofibrosis
MyelofibrosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Safety and tolerability of GB2064: AE
9 Months

Incidence and severity of adverse events as reported by investigators

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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
GB2064EXPERIMENTALGB2064 will be administered orally as 4 x 250 mg tablets twice a day.

Interventions

NameTypeDescription
GB2064DRUGGB2064 (formerly PAT-1251) is a high-affinity, selective, mechanism-based, small molecule inhibitor of LOXL2, administered twice a day
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites11

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...

Countries:United StatesAustraliaGermanyItaly
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Frequently asked questions about GB2064

What is GB2064 used for?

GB2064 is an investigational small molecule being studied for the treatment of myelofibrosis, a bone marrow cancer. It is in Phase 2 clinical development and is not approved for any indication. Development is led by Damora Therapeutics, Inc.

What does GB2064 target?

GB2064 targets LOXL2, an enzyme. By inhibiting LOXL2, the drug is designed to act on the lysyl oxidase family of enzymes, which are involved in remodeling of the bone marrow environment. This mechanism is being explored in the myelofibrosis setting.

Who is developing GB2064?

GB2064 is being developed by Damora Therapeutics, Inc., which trades under the ticker DMRA. The company is running the clinical program for the asset in myelofibrosis.

What phase is GB2064 in?

GB2064 is in Phase 2 clinical development. It is an investigational agent and has not been approved by the FDA or any other regulatory authority. The ongoing study is active but no longer recruiting participants.

What clinical trials is GB2064 in?

GB2064 is being evaluated in one clinical trial, NCT04679870, titled a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of oral GB2064 in participants with myelofibrosis. The Phase 2 trial is active and not recruiting, with 21 participants enrolled across the United States, Australia, Germany, and Italy.

Is GB2064 the same as PAT-1251?

Yes, GB2064 is also known as PAT-1251. Both names refer to the same investigational LOXL2 inhibitor being developed by Damora Therapeutics, Inc. for myelofibrosis, so readers searching for either name are looking at the same drug asset.