Press Releases

— Wugen Earns FDA Orphan Drug Designation for WU-NK-101 for the Treatment of Patients with AML —

ST. LOUIS, MO and SAN DIEGO, CA, Aug. 15, 2023 – Wugen, Inc., a clinical-stage biotechnology company developing a pipeline of allogeneic cell therapies to treat a broad range of hematological and solid tumor malignancies, today announced that it has initiated patient dosing in the first-in-human Phase 1 clinical trial of WU-NK-101 for patients with relapsed or refractory (r/r) acute myelogenous leukemia (AML). Additionally, Wugen announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to WU-NK-101 for the treatment of AML.

WU-NK-101 is Wugen’s lead memory natural killer (NK) cell therapy product and is comprised of cells optimized for anti-cancer function. WU-NK-101 cells possess a unique cytokine-induced memory-like (CIML) phenotype that supports enhanced anti-tumor activity, robust trafficking, superior proliferation capacity, and metabolic flexibility, all of which contribute to treatment resilience in the adverse tumor microenvironment (TME). WU-NK-101 is produced with the company’s proprietary Moneta^TM manufacturing platform, which enables robust generation of off-the-shelf memory NK cell-based therapies.

“The start of this clinical trial is a significant step forward in our memory NK cell program, which we believe offers significant advantages over other NK cell therapy approaches and has the potential to benefit patients with a number of tumor types,” said Jan Davidson-Moncada, M.D., Ph.D., chief medical officer of Wugen. “Early clinical studies with memory NK cells demonstrated impressive response rates in patients with AML, even those with a high disease burden. We look forward to building on these data while advancing WU-NK-101 as a readily accessible allogeneic cell product. In tandem, we are proud to announce our Orphan Drug Designation for WU-NK-101, which will enhance our efforts to deliver this therapy to AML patients.”

“Today’s news represents a significant milestone for Wugen, as WU-NK-101 is the first therapeutic candidate from our memory NK cell platform to enter the clinic,” added Kumar Srinivasan, Ph.D., president and chief executive officer of Wugen. “Beginning in AML, where there remains a high unmet need for new therapeutic options, our goal is to deploy our memory NK cell platform to deliver next-generation, best-in-class allogeneic memory NK cell therapies to transform cancer care, including treatment of solid tumors.”

The Phase 1 study is a global, open-label, dose-escalation, and cohort expansion study designed to characterize the safety and tolerability of WU-NK-101 in patients with r/r AML and determine the recommended Phase 2 dose. It will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and preliminary anti-leukemic activity of WU-NK-101. WU-NK-101 will be administered as three weekly infusions in a 28-day cycle. Additional information is available on clinicaltrials.gov, identifier NCT05470140.

An abstract on the design of the Phase 1 trial was published in the November 2022 supplemental issue of Blood, the journal of the American Society of Hematology.

Memory NK cells have been clinically validated for safety and efficacy in AML. In addition, solid tumor preclinical studies of WU-NK-101 suggest efficacy in solid tumors alone or in combination with monoclonal antibodies.

The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics being developed to treat a rare disease or condition affecting fewer than 200,000 patients in the United States. ODD qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including potentially seven years of U.S. marketing exclusivity, tax credits for clinical research costs, clinical research trial design assistance, the ability to apply for annual grant funding and waiver of Prescription Drug User Fee Act filing fees.

About WU-NK-101

WU-NK-101 is a novel immunotherapy harnessing the power of memory natural killer (NK) cells to treat liquid and solid tumors. Memory NK cells are hyper-functional, long-lasting immune cells that exhibit enhanced anti-tumor activity and a cytokine-induced memory-like (CIML) phenotype. This rare cell population has a superior phenotype, proliferation capacity, and metabolic fitness, making it better suited for cancer therapy than other NK cell therapies. Wugen is applying its proprietary Moneta^TM platform to advance WU-NK-101 as a commercially scalable, off-the-shelf cell therapy for cancer. WU-NK-101 is currently in development for acute myelogenous leukemia (AML). Wugen is planning to initiate solid tumor studies of WU-NK-101 in combination with cetuximab. Studies of WU-NK-101 to date have shown promising robust in vivo activity in various tumor indications, retention of anti-cancer activity in TME, resistance to immune suppression, and enhanced activity with checkpoint inhibitors.

About the Moneta^TM Platform

Wugen’s proprietary Moneta^TM manufacturing platform is a robust, efficient, scalable process to generate off-the-shelf memory natural killer (NK) cell therapies with enhanced anti-tumor functionality. The Moneta^TM platform uses cytokine fusion complexes for streamlined and consistent manufacturing, is free of feeder cells for enhanced safety, and integrates cryopreservation to allow convenient dosing options for cancer patients.

About Wugen

Wugen, Inc. is a clinical-stage biotechnology company developing the next generation of off-the-shelf memory natural killer (NK) and CAR-T cell therapies for cancer. Wugen is leveraging its proprietary Moneta^TM platform and deep genomic engineering expertise to pioneer a new class of memory NK cell therapies to treat hematological and solid tumor malignancies. For more information, please visit www.wugen.com.

Contacts:

Investor Contact:
Mark Lewis
Wugen
Mlewis@wugen.com
314-501-1968

Media Contact:
Christine Fanelle
Scient PR
christine@scientpr.com
215-595-5211