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forodesine

Phase 2

Cutaneous T-cell Lymphoma (CTCL), | Small molecule | Oncology |BioCryst Pharmaceuticals, Inc.|Trials Updated: Jul 10, 2013

forodesine target and mechanism

Molecular targetPNP
Target classInhibitor
ModalitySmall molecule

Also known as forodesine hydrochloride, forodesine hydrochloride (BCX-1777), forodesine HCl

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials8
Total Enrollment215

FDA Designations

No designations recorded

forodesine clinical trials

forodesine · 8 trials · 7 indications

Phase 2 4Phase 1 4
NCT00640523Phase II Study of Forodesine in Subjects With Chronic Lymphocytic Leukemia (CLL)Chronic Lymphocytic Leukemia (CLL)
COMPLETED23 Analytics
NCT00501735Forodesine in the Treatment of Cutaneous T-Cell LymphomaCutaneous T-cell Lymphoma (CTCL),
COMPLETED144 Analytics
NCT00289549Study of Forodesine Hydrochloride in Patients With Advanced, Fludarabine-refractory Chronic Lymphocytic Leukemia (CLL)Leukemia, Lymphocytic, Chronic
COMPLETED8 Analytics
NCT00095381Repeat-Dose of Forodesine Hydrochloride (BCX-1777) Infusion in Patients With Advanced T-Cell LeukemiaLeukemia, T-Cell
COMPLETED20 Analytics
PHASE2COMPLETED
Phase II Study of Forodesine in Subjects With Chronic Lymphocytic Leukemia (CLL)
Chronic Lymphocytic Leukemia (CLL)Unlock trial analytics
PHASE2COMPLETED
Forodesine in the Treatment of Cutaneous T-Cell Lymphoma
Cutaneous T-cell Lymphoma (CTCL),Unlock trial analytics
PHASE2COMPLETED
Study of Forodesine Hydrochloride in Patients With Advanced, Fludarabine-refractory Chronic Lymphocytic Leukemia (CLL)
Leukemia, Lymphocytic, ChronicUnlock trial analytics
PHASE2COMPLETED
Repeat-Dose of Forodesine Hydrochloride (BCX-1777) Infusion in Patients With Advanced T-Cell Leukemia
Leukemia, T-CellUnlock trial analytics

Study Endpoints

Primary Endpoints

Overall response rate
Up to 6 cycles of therapy
The primary objective of this study is to determine the objective response rate to treatment with oral forodesine in subjects with cutaneous manifestations of CTCL subjects, stages IIB, III, and IVA.
Duration of Study
Response rate after 2 cyclesof forodesine therapy.
8 weeks
Determine the sustained effectiveness of IV forodesine hydrochloride infusions in patients with advanced T-cell leukemia (T-ALL or T-PLL)

Secondary Endpoints

Safety and tolerability
Duration of Study
Time to and duration of objective response in cutaneous manifestations
Duration of Study
Time to loss of objective response
Duration of Study
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Interventions

NameTypeDescription
forodesine HClDRUG2 x 100mg capsules daily
Forodesine 200 mgDRUG2 x 100mg tablets once daily
forodesine hydrochloride (BCX-1777)DRUGexperimental, forodesine oral dose 200 mg administered daily.
forodesine hydrochlorideDRUG -
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Eligibility Criteria

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

Inclusion Criteria: 1. Age 18 years and older 2. Diagnosis of CLL established by peripheral blood and/or bone marrow examination and using the standard diagnostic criteria. 3. Subjects with Rai stage III or IV, or earlier stage requiring therapy as determined by the 1996 National Cancer Institute -...

Countries:United StatesAustraliaAustriaFinlandFranceGermanyItalySpainSwitzerlandUnited KingdomGeorgia
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Competitive Landscape -Cutaneous T-Cell Lymphoma 7 trials (matched to "Cutaneous T-cell Lymphoma (CTCL),")

Top 1 of 2 competitors

CompanyTickerTrialsLead PhaseDrugs
Incyte CorporationINCY2PHASE2Ruxolitinib
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Frequently asked questions about forodesine

What is forodesine?

Forodesine is an investigational small molecule drug developed by BioCryst Pharmaceuticals, Inc. (ticker BCRX) for certain blood cancers. It has been studied in B-cell acute lymphoblastic leukemia, chronic lymphocytic leukemia, cutaneous T-cell lymphoma, and other leukemias and lymphomas. It is not approved and remains in clinical development.

What is forodesine used for?

Forodesine has been studied in several hematologic cancers, including B-cell acute lymphoblastic leukemia, chronic lymphocytic leukemia, cutaneous T-cell lymphoma, and other leukemia and lymphoma indications. These studies were conducted in adults aged 18 years and older. Forodesine is investigational and is not an approved treatment for any indication.

What does forodesine target?

Forodesine targets purine nucleoside phosphorylase, or PNP, and acts as an inhibitor of that enzyme. PNP is involved in purine metabolism, and inhibiting it is the intended mechanism of action for this small molecule in lymphoid malignancies. The drug was designed to interfere with PNP activity in cancer cells.

Who makes forodesine?

Forodesine is developed by BioCryst Pharmaceuticals, Inc., which trades under the ticker BCRX. BioCryst is the company associated with the forodesine hydrochloride compound, also referred to as BCX-1777, across its clinical development programs in leukemia and lymphoma.

What phase is forodesine in?

Forodesine is in Phase 1 development and is investigational. It has not been approved by the FDA for any indication. Clinical work has included Phase 1 and Phase 2 studies, and no trials are currently active. The completed studies enrolled a total of 144 participants across the programs.

What clinical trials is forodesine in?

Completed forodesine trials include NCT00640523 in chronic lymphocytic leukemia, NCT00501735 in cutaneous T-cell lymphoma, NCT00289562 in B-cell acute lymphoblastic leukemia, and NCT00289549 in fludarabine-refractory chronic lymphocytic leukemia. These studies were conducted across the United States, Australia, and several European countries. All are listed as completed.

Is forodesine the same as forodesine hydrochloride?

Yes, forodesine hydrochloride is the salt form of forodesine and is also known as BCX-1777. Other names used for this asset include forodesine HCl and Forodesine 200 mg. These names refer to the same BioCryst Pharmaceuticals compound studied in leukemia and lymphoma trials.