eFFECTOR Therapeutics' Lead Product Candidate, eFT508, Receives Orphan Designation from FDA for Treatment of Diffuse Large B-Cell Lymphoma

Dosing commenced in Phase 1/2 clinical trial in subjects with hematological malignancies

SAN DIEGO, March 9, 2017 /PRNewswire/ -- eFFECTOR Therapeutics today announced that its lead product candidate, eFT508, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of diffuse large B-cell lymphoma (DLBCL), an aggressive form of non-Hodgkin lymphoma. eFT508 is a potent, highly selective inhibitor of MNK1 and MNK2, two enzymes that are critical for the growth and survival of tumors. eFFECTOR also announced that it has dosed the first subject in a Phase 1/2 clinical trial of eFT508 in patients with B-cell hematological malignancies.

"Diffuse large B-cell lymphoma is an aggressive disease for which patients badly need additional treatment options," said Steve Worland, Ph.D., president and chief executive officer of eFFECTOR. "We hope that eFT508, a selective translation regulator that blocks multiple oncogenic growth and proliferation pathways, as well as activates anti-tumor immunity, will prove effective in treating patients afflicted with this devastating disease."

The recently initiated trial is a Phase 1/2, open-label, sequential-group, dose-escalation and cohort-expansion study evaluating the safety, pharmacokinetics, pharmacodynamics and antitumor activity of eFT508. The primary goals will be to establish a maximum tolerated dose and recommended dose for further evaluation and to assess preliminary efficacy of the compound. eFFECTOR is also conducting a Phase 1/2 dose escalation trial of eFT508 in patients with solid tumors.

Orphan designation is granted by the FDA Office of Orphan Products Development to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the United States. The orphan designation of eFT508 for DLBCL provides increased access to FDA reviewers to discuss clinical trial designs, the ability to qualify for tax credits for certain clinical research costs, the ability to apply for annual grant funding, and a waiver of Prescription Drug User Fee Act filing fees, as well as the potential for seven years of U.S. marketing exclusivity if the product candidate is approved.

About eFT508
eFT508 is a potent, highly selective and orally bioavailable inhibitor of MNK1 and MNK2 being studied in patients with solid tumors and lymphoma. MNK1 and 2 are kinases acting as signal integrators at the convergence of multiple oncogenic pathways to regulate production of disease-driving proteins, including checkpoint proteins.

About Diffuse Large B-Cell Lymphoma
Diffuse large B-cell lymphoma is an aggressive form of non-Hodgkin lymphoma (NHL) that can begin in lymph nodes and areas of the body outside of the lymphatic system. DLBCL is the most common form of NHL, making up 30 percent of newly-diagnosed NHL cases each year, according to the Lymphoma Research Foundation. Due to the fast-acting and aggressive nature of the disease, DLBCL requires immediate treatment upon diagnosis.

About eFFECTOR Therapeutics
eFFECTOR Therapeutics is a clinical-stage biopharmaceutical company pioneering the discovery and development of selective translation regulators as a new class of small molecule therapeutics for cancer. The company's investigational compounds are designed to restore translational control to halt underlying disease mechanisms while preserving healthy physiological processes. eFFECTOR's most advanced program focuses on the development of eFT508. The company has additional selective translation regulator programs currently in discovery and development. For more information visit www.effector.com.

SOURCE eFFECTOR Therapeutics

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