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Umbralisib

Phase 2

Marginal Zone Lymphoma | Small molecule | Oncology |TG Therapeutics, Inc.|Last Updated: Jun 23, 2023

Target and mechanism

Molecular targetCSNK1E, PIK3CD, ABL1
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment21

FDA Designations

No designations recorded

Clinical trial landscape

Umbralisib · 2 trials · 5 indications

Phase 2 1Phase 1 1
NCT03364231Study to Assess the Efficacy and Safety of Umbralisib in Participants With Non-Follicular Indolent Non-Hodgkin's LymphomaMarginal Zone Lymphoma
COMPLETED21 Analytics
PHASE2COMPLETED
Study to Assess the Efficacy and Safety of Umbralisib in Participants With Non-Follicular Indolent Non-Hodgkin's Lymphoma
Marginal Zone LymphomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Overall Response Rate (ORR) as Assessed by Revised Response Criteria for Non- Hodgkin's Lymphoma (Lugano Classification) and Consensus-Based 6th International Workshop on Waldenstrom's Macroglobulinemia (IWWM)
Every 3 cycles (1 Cycle = 28 days) from Day 1 Cycle 1 up to approximately 4.2 years

ORR for MZL=percentage of participants with complete response (CR)/partial response (PR). ORR for WM=CR/PR/very good partial response (VGPR)/minor response (MR). Response assessed per revised Lugano Classification for MZL \&per IWWM for WM participants. Per Lugano criteria CR=complete disappearance of all evidence of disease \& disease-related symptoms. PR=regression of measurable disease \& no new disease sites. Regression=≥50% decrease in the sum of the products of the diameters (SPD) of index lesions, with no increase in size of other lymph nodes/liver/spleen.Per IWWM criteria CR=disappearance of serum monoclonal immunoglobulin M (IgM) protein by immunofixation with a normal serum IgM level. VGPR=reduction of monoclonal IgM protein \>90% from baseline. PR=reduction of monoclonal IgM protein between 50-90% from baseline with regression of measurable disease. Regression defined in similar manner as Lugano Classification. MR=reduction of monoclonal IgM protein \>25% but \<50% from baseline.

Duration of Response (DOR)
From the first demonstration of response to umbralisib till disease progression/death (up to approximately 4.2 years)

DOR is defined as the time from documentation of a response to treatment to the first documentation of tumor progression or death due to any cause, whichever comes first.

Determine Acceptable Adverse Events That Are Related to Treatment
6 months of therapy

To determine the incidence of adverse events, any potential abnormal laboratory results and any dose-limiting toxicities

Secondary Endpoints

Complete Response (CR) Rate
Every 3 cycles (1 Cycle = 28 days) from Day 1 Cycle 1 up to approximately 4.2 years
Progression-Free Survival (PFS)
From date of randomization until the date of first documented progression (up to approximately 4.2 years)
Time to Treatment Failure (TTF)
From first dose on Day 1 of Cycle 1 (28 days = 1 cycle) up to discontinuation of treatment (up to approximately 4.2 years)
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Marginal Zone Lymphoma (MZL): UmbralisibEXPERIMENTALParticipants with non-follicular indolent non-Hodgkin's lymphoma (iNHL) with MZL as the histology type received umbralisib, 800 milligrams (mg), orally, once daily (QD), until disease progression, unacceptable toxicity, or withdrawal from the study whichever occurred first.
Waldenstrom's Macroglobulinemia (WM): UmbralisibEXPERIMENTALParticipants with non-follicular iNHL with WM as the histology type received umbralisib, 800 mg, orally, QD, until disease progression, unacceptable toxicity, or withdrawal from the study whichever occurred first.
TG-1501 + Ublituximab + UmbralisibEXPERIMENTALUblituximab at a fixed IV infusion dose Days 1, 8 and 15 followed by maintenance infusions Umbralisib oral daily dose TG-1501 IV infusion at scheduled intervals

Interventions

NameTypeDescription
UmbralisibDRUGOral Daily Dose
ublituximabBIOLOGICALIV anti-CD20 monoclonal antibody
TG-1501BIOLOGICALIV immunotherapy for cancer
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Confirmed diagnosis of Waldenstroms Macroglobulinemia * Relapsed or refractory after at least one prior treatment regimen * Eastern Cooperative Oncology Group (ECOG) score of 0 to 2 Exclusion Criteria: * Any major surgery, chemotherapy or immunotherapy within the last 21 day...

Countries:United States
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Frequently asked questions about Umbralisib

What is Umbralisib used for?

Umbralisib is an investigational small molecule being studied for the treatment of Chronic Lymphocytic Leukemia and Marginal Zone Lymphoma. It is also being evaluated in Richter Syndrome and other non-follicular indolent non-Hodgkin lymphomas. The drug is in clinical development and is not yet approved by the FDA.

What does Umbralisib target?

Umbralisib is a PI3K inhibitor, as indicated by its '-lisib' suffix, which denotes its target class. It is being studied in oncology for its potential to treat certain blood cancers, including Chronic Lymphocytic Leukemia and Marginal Zone Lymphoma.

Who makes Umbralisib?

Umbralisib is being developed by TG Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol TGTX. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with certain hematologic malignancies.

What phase is Umbralisib in?

Umbralisib has completed Phase 1 and Phase 2 clinical trials. A Phase 1 study evaluated the drug in combination with ublituximab in patients with relapsed or refractory Chronic Lymphocytic Leukemia or Richter's Transformation. A separate Phase 2 study assessed its efficacy and safety in Marginal Zone Lymphoma and other indolent non-Hodgkin lymphomas.

What clinical trials is Umbralisib in?

Umbralisib has been studied in two completed clinical trials. NCT02535286 was a Phase 1 study of immunotherapy combined with ublituximab and umbralisib in patients with relapsed or refractory CLL or Richter's Transformation. NCT03364231 was a Phase 2 study evaluating umbralisib in participants with Marginal Zone Lymphoma and other non-follicular indolent non-Hodgkin lymphomas.

Is Umbralisib the same as umbralisib tosylate?

Umbralisib is the drug name used in clinical trials, and it may also be referred to by its chemical salt form, umbralisib tosylate. The clinical studies NCT02535286 and NCT03364231 use the name umbralisib, and the drug is being developed by TG Therapeutics for the treatment of certain B-cell malignancies.