Syntimmune Announces Clinical Data from First-in-Human Trial of Lead Candidate SYNT001

NEW YORK--(BUSINESS WIRE)-- Syntimmune, Inc., a clinical-stage biotechnology company focused on breakthrough therapies based on FcRn biology, today announced robust clinical data from the Phase 1a single ascending dose, double-blind, placebo-controlled trial of Syntimmune’s lead drug candidate, SYNT001. The trial results demonstrated SYNT001 as an intravenous formulation was well tolerated with a favorable safety profile in healthy volunteers. The study also showed that administration of SYNT001 resulted in rapid, dose-dependent, durable, and clinically significant reductions of IgG and circulating immune complexes (CICs). IgG and CICs are key measures of FcRn’s biologic functions in effectuating disease activity. Disabling these highlights SYNT001’s significant promise as a treatment for IgG-mediated autoimmune diseases.

SYNT001 is a first-in-class monoclonal antibody that blocks FcRn-IgG interactions and is being developed for the treatment of a broad variety of autoimmune diseases that are dependent upon IgG. An abstract of the Phase 1a trial results was posted today on the ASH website in advance of a poster presentation of the data, which will take place during the 59th ASH Annual Meeting, to be held in Atlanta, December 9-12.

“The Phase 1a data we are presenting provide the first clinical evidence showing SYNT001 can effectively block and dismantle the FcRn-mediated processes that drive autoimmune diseases and that derive from the activities of IgG,” said Richard Blumberg, M.D., Syntimmune’s scientific founder and senior author of the ASH abstract. “SYNT001 was designed to optimally inhibit FcRn interactions with monomeric and multimeric IgG in acidic intracellular compartments where FcRn function mainly takes place. These data demonstrate significant, rapid, and dose-dependent reductions of all IgG subclasses that are durable through 28 days, following a single intravenous administration of SYNT001 to healthy volunteers.”

Dr. Blumberg added, “As predicted by results in preclinical models, we also confirmed that SYNT0001 promoted the clearance of CICs from the bloodstream in this Phase 1a study. These data demonstrate the ability of SYNT001 to block FcRn interactions with CIC. As FcRn binding to CIC is critical to IgG’s ability to enable innate and adaptive immune responses that drive disease, these observations have important therapeutic implications. We look forward to continued clinical development of this promising experimental treatment.”

Laurence Blumberg, M.D., Syntimmune’s business founder and lead author of the ASH abstract, said, “As the time to an approximately 50 percent reduction of total IgG and CIC lowering occurred within five days at a dose of 30 mg/kg and in conjunction with a favorable safety profile, we are extremely enthusiastic about our further development of SYNT001 in a broad range of IgG-mediated autoimmune diseases. Syntimmune is now advancing and enrolling patients in two US-based multi-dose Phase 1b/2a clinical trials of SYNT001 in pemphigus and warm autoimmune hemolytic anemia. Based on our progress, we believe we will be able to report topline data from these studies in the first half of 2018. We also intend to initiate additional studies in other indications for SYNT001.”

Details of the poster presentation at the ASH Annual Meeting are as follows:

Title: SYNT001: A Humanized IgG4 Monoclonal Antibody That Disrupts the Interaction of FcRn and IgG for the Treatment of IgG-Mediated Autoimmune Diseases (Abstract #3438)
Session: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Poster III
Date: Monday, December 11, 2017
Presentation Time: 6:00 PM - 8:00 PM Eastern Time
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
Authors: Laurence Blumberg, MD^a; John E. Humphries, MD^b; Kenneth C. Lasseter, MD^c; Richard S. Blumberg, MD^d; ^aSyntimmune, Inc., New York, NY; ^bBiologics Consulting, Alexandria, VA; ^cClinical Pharmacology of Miami, Inc., Miami, FL; ^dBrigham and Women’s Hospital, Boston, MA

Additional Details on the SYNT001 Clinical Development Program

SYNT001 Phase 1a Study in Healthy Volunteers

Syntimmune’s Phase 1a single ascending dose, randomized, double-blind, placebo-controlled trial enrolled 31 healthy male subjects at a single site in the US. Subjects were randomized to receive one of five dose levels of SYNT001 or placebo. The study’s primary outcome measures were safety and tolerability. Secondary measures included pharmacokinetics (PK) and pharmacodynamics (PD).

Phase 1b/2a Program

Both warm autoimmune hemolytic anemia and pemphigus provide an opportunity to address major unmet medical needs. These IgG-mediated autoimmune diseases occur in distinct tissue compartments and could serve to illustrate the broad potential utility and applicability of SYNT001 across the spectrum of IgG-mediated diseases. This highly specific and differentiated approach to blockade of IgG-mediated inflammation is expected to preserve other key aspects of the immune system and enable substantial improvement over current standards of care for these devastating diseases.

SYNT001 Phase 1b/2a Study in Warm Autoimmune Hemolytic Anemia (WAIHA)

Syntimmune is advancing a multicenter, open-label, safety, tolerability, and activity Phase 1b/2a study of SYNT001 in individuals with chronic, stable WAIHA. The study’s primary outcome measures are safety and tolerability. Secondary measures include PK, PD, immunogenicity, and effects on disease activity markers. WAIHA is a rare blood disorder in which the immune system produces antibodies that attack a patient’s own red blood cells, the destruction of which can lead to severe, potentially debilitating anemia. There are limited treatment options and other than corticosteroids, no therapies are currently approved to treat WAIHA. For more information, please visit clinicaltrials.gov.

SYNT001 Phase 1b/2a Study in Pemphigus

Syntimmune is also advancing a multicenter, open-label, safety, tolerability, and activity Phase 1b/2a study of SYNT001 in subjects with pemphigus (vulgaris or foliaceus). The study’s primary outcome measures are safety and tolerability. Secondary measures include PK, PD, immunogenicity, and effects on disease activity markers. Pemphigus is characterized by autoimmune blistering of the skin and mucous membranes; in its more severe manifestations this disease can be highly debilitating and potentially fatal. Many patients require long-term use of corticosteroids and other immune system suppressors associated with serious adverse events. For more information, please visit clinicaltrials.gov.

About Syntimmune

Founded in 2013 by Richard Blumberg, M.D., and Laurence Blumberg, M.D., Syntimmune is advancing novel therapies based on its leading position in the biology of the neonatal Fc receptor (FcRn). Based upon pre-clinical models, FcRn functions as a core part of a central common pathway that enables abnormal IgG responses, making it a well-validated target for the treatment of IgG-mediated autoimmune diseases. Syntimmune’s lead candidate, SYNT001, is a monoclonal antibody that specifically blocks FcRn-IgG interactions and is being studied in multiple Phase 1b/2a trials for the treatment of IgG-mediated autoimmune diseases. In addition to SYNT001, Syntimmune is developing SYNT002, which targets the interaction between FcRn and albumin and promotes clearance of albumin-bound endo- and exotoxins. The Syntimmune team has world-class experience in the field of FcRn biology and has successfully pioneered and advanced biologics that engage FcRn. Since its founding, the company has been funded with a total of $78 million in capital commitments from leading life sciences investors, led by Apple Tree Partners and includes Partners Innovation Fund, FMB Research, and AFB Fund. For more information on Syntimmune, please visit the company’s website at www.syntimmune.com.

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