Clinical benefit observed in multiple patients treated with single agent RORgamma Agonist

NEW YORK and ANN ARBOR, Mich., April 17, 2018 /PRNewswire/ — Lycera Corp., a privately held biopharmaceutical company developing breakthrough immune modulatory medicines, today announced new clinical findings from the Phase 1 portion of its Phase 1/2a ARGON study of the company’s novel immuno-oncology therapeutic candidate, LYC-55716, at the 2018 American Association of Cancer Research (AACR) Annual Meeting. LYC-55716 is a first-in-class oral, selective retinoic acid-related orphan receptor-gamma (RORgamma) agonist designed to reprogram the immune system in patients with solid tumors.

The results from the Phase 1 portion of the ARGON study demonstrated that LYC-55716 was well- tolerated by patients. During the study, no dose-limiting toxicities were observed in the 32 patients enrolled in 6 dosing cohorts in the Phase 1 portion of the study, and no patients withdrew from the trial due to treatment-related adverse events. The majority of adverse events (AEs) were graded 1-2. Grade 3 treatment-related AEs were reported only for single occurrences of anemia, elevated gamma-glutamine transferase, and hypophosphatemia; no Grade 4 treatment-related AEs occurred.

Of the 26 patients evaluable for response in the dose escalation portion of the study, 12 (46%) had stable disease (SD), including 7 patients (27%) with disease stabilization exceeding 4 months.  One patient has a confirmed partial response (cPR) and is 1 of 3 patients that remain on study. The cPR was observed in a patient with advanced metastatic non-small cell lung cancer with PD-L1 high expression levels who did not respond to first line pembrolizumab (KEYTRUDA®) or second line treatment with carboplatin and pemetrexed (Alimta®).  The patient remains on study through cycle 8 (28-day cycles).  Stabilization of disease with tumor regression was also observed in other patients during the study.  Importantly, a patient with advanced, refractory sarcomatoid breast cancer that failed 2 prior lines of doublet chemotherapy experienced a 19% regression in tumor volume and is currently continuing treatment in cycle 8.  The remaining patient on study with endometrial cancer has experienced stable disease through cycle 7.

“The promising safety results and early signals of efficacy with LYC-55716 as a monotherapy are very encouraging,” said Judy S. Wang, MD, Associate Director of Drug Development, Florida Cancer Specialists and Sarah Cannon Research Institute. “We are very pleased to be participating in the development of this novel immunotherapeutic, and are actively enrolling patients with advanced solid tumor cancers in the Phase 2a portion of the study.”

“We believe the growing body of evidence we are reporting with our novel RORgamma Agonist program will help drive recognition of the significance of the RORgamma target in immuno-oncology.  We are excited with our progress moving into the Phase 2a portion of the study to demonstrate the potential of LYC-55716 in addressing diverse areas of unmet need in cancer,” said Paul Sekhri, President and CEO of Lycera. “We are looking forward to fully enrolling the Phase 2a study, as well as the ongoing Phase 1b combination trial with pembrolizumab (KEYTRUDA®) in individuals with metastatic non-small cell lung cancer (NSCLC). Based on this progress, we anticipate reporting topline efficacy findings from both studies by or before mid-2019.”

In addition, presentations at AACR by Lycera and collaborators are focused on RORgamma agonist rationale and preclinical data supporting clinical combinations with established immunotherapies.  Data presented by Samir N. Khleif, MD, BioMedical Scholar and Professor of Oncology and Director, The Loop Immuno-Oncology Laboratory, Lombardi Cancer Center, Georgetown University Medical Center, demonstrated that RORgamma agonism significantly bolstered anti-PD1 anti-tumor effect and improved anti-OX40 tumor control in preclinical models. Lycera is also presenting additional preclinical and bio- informatic research which explored other potential combinations that may be synergistic with RORgamma agonists in the clinic.  These data findings support the Phase 1b combination study of LYC-55716 and pembrolizumab in advanced non-small cell lung cancer and potentially additional combination strategies.

About the ARGON Trial

The ARGON trial (Trial of RORgamma Agonist LYC-55716 in Advanced Cancer) is a Phase 1/2a study of LYC-55716 in patients with advanced, relapsed, or refractory solid tumors. The initial Phase 1 portion of the ARGON study was designed to find the biologically active or maximum tolerated dose (MTD) of LYC-55716. The study utilized a 3+3 study design, in which LYC-55716 was administered orally in subjects with relapsed or refractory solid tumors. The primary endpoints were safety and tolerability and determination of the recommended Phase 2a dose, while secondary endpoints included objective responses according to RECIST v1.1 criteria. Upon determination of the recommended Phase 2a dose, LYC-55716 initiated Phase 2a, which is expected to enroll approximately 75 patients. The primary efficacy endpoint of the Phase 2a portion of the study will be objective response rate according to response evaluation criteria in solid tumors.

About LYC-55716

LYC-55716 is a first in class oral, selective RORgamma agonist. The retinoic acid-related orphan receptor gamma (RORgamma) is a nuclear receptor transcription factor that acts as an immune cell master control switch. RORgamma agonists modulate gene expression to reprogram immune cells for improved function, as well as decrease immunosuppressive mechanisms, resulting in decreased tumor growth and enhanced survival in in vivo preclinical models of cancer. Essentially, Lycera’s RORgamma agonist approach “removes the brake” and “pushes on the accelerator” of immune function. LYC-55716 is an investigational compound that is not approved for any use in any country.

About Lycera

Lycera is a biopharmaceutical company developing novel oral immune modulators for the treatment of autoimmune diseases and cancer. Based on successful progress of its world-class R&D platform, including expertise in immune metabolism, cell signaling, and immune cell differentiation, Lycera commenced multiple clinical programs in 2016. The company is advancing a wholly owned, oral, gut- directed ATPase modulator, designated LYC-30937-EC, for the treatment of autoimmune disease, and has entered Phase 2 clinical studies in patients with ulcerative colitis and psoriasis. A second product candidate, LYC-55716, an oral RORgamma agonist, is progressing in the Phase 1/2a ARGON trial, a monotherapy study in patients with advanced solid tumors, as well as in a Phase 1b combination trial. Lycera has an exclusive strategic collaboration with Celgene Corporation to advance Lycera’s proprietary pipeline for cancer and immune-mediated diseases.

Lycera’s leadership possesses deep experience in drug discovery, development, and commercialization and has established close relationships with renowned thought leaders and clinical researchers worldwide. Lycera was founded in 2006 based on an initial scientific platform in-licensed from the University of Michigan. Lead investors in Lycera include InterWest Partners, ARCH Venture Partners, Clarus Ventures, and EDF Ventures.


KEYTRUDA® is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

Alimta® is a registered trademark of Eli Lilly and Company Indianapolis, IN, USA.

CONTACT: Justin Jackson, Burns McClellan, 212-213-0006, ext. 327,