Precision Therapeutics for Genetic Obesities

DISEASE FOCUS: MC4R HI

Our lead candidate, CTX-B, treats melanocortin-4 receptor haploinsufficiency (MC4R HI) — a well-defined obesity characterized by early onset hyperphagia and obesity with no approved targeted therapeutic options, impacting 100,000-150,000 patients in the United States (roughly half of the total genetic obesity population.)

MC4R Pathway

MC4R is the central regulator of hunger and body weight and is a major genetic driver of hyperphagia and severe obesity. MC4R pathway-driven obesity arises from mutations at multiple points — as far upstream, where defects impair leptin signaling and the production of pro-opiomelanocortin (POMC), the precursor of α-melanocyte-stimulating hormone (α-MSH), MC4R’s natural ligand; through to the MC4R itself. Mutations in between can impair the ability of POMC production.

Courage’s agonists act directly on MC4R to compensate for the lack of adequate ligand expression or can augment endogenous ligand levels to restore pathway activity and reduce the otherwise uncontrollable drive to eat.

CLINICAL CANDIDATE: CTX-B

CTX-B is a weekly injectable for the treatment of MC4R HI. While we believe our drug can address all 250K+ patients in the US that have severe obesity caused by MC4R pathway mutations, we chose MC4R HI as our initial indication:

  • High clinical need in this severely obese population;
  • The absence of any therapy targeted to this particular segment of genetic obesity;
  • The size of the unserved patient population.

EFFECTIVENESS AND SAFETY

In preclinical models of MC4R HI, CTX-B produces significant weight loss in MC4R HI models.  In non-human primates, CTX-B has shown no cardiovascular signal at supratherapeutic exposures — a liability that has caused suspension of some earlier MC4R programs. 

Our Pipeline

Courage R&D supports extension of the CTX platform into additional therapeutic areas. Our current focus, though, is the validation and application of CTX-B in the large and underserved MC4R HI population.

About Us

Courage Therapeutics is a neuroendocrine company co-founded by Dr. Roger Cone.  Dr. Cone, Director of the Life Sciences Center at the Life Sciences Center at The University of Michigan and a member of both the National Academy of Sciences and the National Academy of Medicine, is a pioneering researcher in melanocortin receptor biology and the central melanocortin system’s neural circuits that govern energy balance. We translate decades of Dr. Cone’s discoveries and novel chemistries into therapeutic candidates targeting MC4R, a proven pathway.

Leadership

Giovanni Ferrara, M.Sc., M.B.A.
CEO

Roger Cone, Ph.D.
Co-Founder, CSO

Tomi Sawyer, Ph.D.
Chief Medicinal Chemist

Sasha Blaug, Ph.D., M.B.A.
CBO-CFO

George Grass, Pharm.D., Ph.D.
Head of Non-Clinical

Board of Directors

Isaac Barchas, J.D.
Managing Director, Arsenal Bridge Ventures

Roger Cone, Ph.D.
Co-Founder, CSO Courage

Giovanni Ferrara, M.Sc., M.B.A.
CEO Courage

Dan Housman, B.S.
Co-Founder Courage

Bruce Leuchter, M.D.
CEO Neurvati

Contact

For business development inquiries please email us at BD@couragetx.com