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FDA Grants Orphan Drug Designation to TG Therapeutics’ Umbralisib for the Treatment of Marginal Zone Lymphoma

NEW YORK, April 15, 2019 (GLOBE NEWSWIRE) — TG Therapeutics, Inc. (NASDAQ: TGTX) today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation to its phosphoinositide-3-kinase (PI3K) delta inhibitor, umbralisib (TGR-1202), for the treatment of patients with all three types of marginal zone lymphoma (MZL): nodal, extranodal, and splenic MZL.

Umbralisib monotherapy is being evaluated in the UNITY-NHL Phase 2b registration directed clinical trial. The MZL cohort of the UNITY-NHL trial is currently evaluating the safety and efficacy of single agent umbralisib in patients with MZL who have received at least one prior anti-CD20 regimen, the same indication for which the FDA recently granted breakthrough therapy designation for umbralisib.

Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics, stated “Receiving orphan drug designation for umbralisib in patients with MZL is another important milestone in our commitment to developing novel treatment options for patients with B-cell malignancies, including orphan diseases such as MZL.” Mr. Weiss continued, “We are highly encouraged by the interim results presented thus far for the MZL cohort of the UNITY-NHL trial and we look forward to presenting final data from this cohort later this year and to discussing the results with the FDA with the goal of filing for accelerated approval of umbralisib by year-end.”


Orphan drug designation is granted by the U.S. FDA to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan product exclusivity.


Marginal zone lymphoma (MZL) comprises a group of indolent (slow growing) B-cell non-Hodgkin lymphomas (NHLs) that begin forming in the marginal zone of lymphoid tissue. With an annual incidence of approximately 7,500 newly diagnosed patients, MZL is the third most common B-cell NHL accounting for approximately eight percent of all NHL cases. MZL consists of three different subtypes: extranodal MZL, nodal marginal zone lymphoma (NMZL), and splenic marginal zone lymphoma (SMZL).

ABOUT THE UNITY-NHL PHASE 2b STUDY—Marginal Zone Lymphoma Cohort

The multicenter, open-label, UNITY-NHL Phase 2b study – Marginal Zone Lymphoma cohort was designed to evaluate the safety and efficacy of single agent umbralisib in patients with MZL who have received at least one prior anti-CD20 regimen. The primary endpoint is overall response rate (ORR) as determined by Independent Review Committee (IRC) assessment. Secondary endpoints include safety, duration of response, and progression-free survival (PFS).

The MZL cohort completed enrollment in August 2018, and in February of 2019 the Company announced that the primary endpoint of ORR as determined by central IRC was met for all treated patients (n=69).

Earlier this month at the American Association of Cancer Research (AACR) annual meeting, interim data from the UNITY-NHL MZL cohort were presented on the first 42 patients enrolled, demonstrating an ORR of 52%, with a Complete Response (CR) rate of 19%, and a tolerable safety profile amongst all patients treated to date (n=69).


TG Therapeutics is a biopharmaceutical company focused on the acquisition, development and commercialization of novel treatments for B-cell malignancies and autoimmune diseases. Currently, the company is developing multiple therapies targeting hematological malignancies and autoimmune diseases. Ublituximab (TG-1101) is a novel, glycoengineered monoclonal antibody that targets a specific and unique epitope on the CD20 antigen found on mature B-lymphocytes. TG Therapeutics is also developing umbralisib (TGR-1202), an oral, once-daily inhibitor of PI3K-delta. Umbralisib uniquely inhibits CK1-epsilon, which may allow it to overcome certain tolerability issues associated with first generation PI3K-delta inhibitors. Both ublituximab and umbralisib, or the combination of which is referred to as “U2”, are in Phase 3 clinical development for patients with hematologic malignancies, with ublituximab also in Phase 3 clinical development for Multiple Sclerosis. Additionally, the Company has recently brought into Phase 1 clinical development, TG-1501, its anti-PD-L1 monoclonal antibody, TG-1701, its covalently-bound Bruton’s Tyrosine Kinase (BTK) inhibitor and TG-1801, its anti-CD47/CD19 bispecific antibody. TG Therapeutics is headquartered in New York City.

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