Equillium, Inc. (Nasdaq: EQ) announced on 12/19/18 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EQ001 for the treatment of acute graft-versus-host disease (aGVHD). The Company is planning to initiate a Phase 1b/2 clinical trial in early 2019, called the EQUATE trial, which will evaluate EQ001 for the treatment of patients presenting with aGVHD.
“FDA Fast Track designation of EQ001 highlights the significant need for novel approaches for the first line treatment of aGVHD in combination with corticosteroids and positions Equillium to rapidly advance a promising treatment to patients suffering with this common complication after hematopoietic stem cell transplant,” said Krishna Polu, M.D., chief medical officer of Equillium. “We are excited about this important achievement and believe the opportunity for more frequent dialog with the FDA will benefit the efficient development of EQ001 in aGVHD. Our team is working closely with investigators and trial sites to rapidly prepare for dosing of the first patients in the EQUATE trial early next year.”
The EQUATE trial is a Phase 1b/2 clinical trial that will enroll approximately 84 patients to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical activity of EQ001 for the initial treatment of aGVHD in combination with corticosteroids (NCT 03763318). The Phase 1b component is an open-label dose escalation trial and will be followed by the Phase 2 component, which is a randomized, double-blind, placebo-controlled trial in which subjects will receive either EQ001 or placebo in combination with corticosteroids over a two-month period.
Both aGVHD and chronic graft-versus-host disease (cGVHD) are multisystem disorders that are common complications of allogeneic hematopoietic stem cell transplants (HSCT). Graft-versus-host disease (GVHD) is caused by the transplanted immune system recognizing and attacking the recipient’s body. Symptoms of GVHD include rash, itching, skin discoloration, nausea, vomiting, diarrhea and jaundice, as well as eye dryness and irritation.
GVHD is the leading cause of non-relapse mortality in cancer patients receiving allogeneic HSCT, and the risk of GVHD limits the number and type of patients receiving HSCT. Approximately 50 percent of HSCT recipients develop aGVHD, resulting in very high morbidity and mortality, with five-year survival of approximately 53 percent in patients who respond to corticosteroid treatment, and mortality as high as 95 percent in patients who do not respond to corticosteroids.
GVHD is predominantly driven by T effector cells (Teff). Prior clinical studies have implicated Teff cells that express high levels of CD6 in the development of GVHD, providing evidence that CD6 is a highly relevant target in this disease.
EQ001 (itolizumab) is a clinical-stage, first-in-class monoclonal antibody that selectively targets CD6, a novel immune checkpoint pathway. CD6 plays a central role in the modulation of Teff cell activity and trafficking. Activated Teff cells drive a number of immuno-inflammatory diseases across therapeutic areas, including transplantation science, pulmonary, neurologic, gastrointestinal, renal, vascular, ophthalmic and dermatologic inflammatory disorders. Based on its broad upstream multi-modal mechanism of action, EQ001 may have potential to treat multiple severe immuno-inflammatory diseases, including those that are resistant or refractory to existing therapies.