Precision Therapeutics for Obesity

Leveraging the hypothalamus and brainstem to achieve metabolic homeostasis

About Us

Courage Therapeutics is a leading neuroendocrine company co-founded by Dr. Roger Cone, 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 into therapeutic candidates targeting MC4R, the master regulator of appetite and body weight.

Our melanocortin agonists show single-agent activity across multiple forms of genetic obesity — populations that today have no or limited effective targeted therapy.

Our Focus

Our lead candidate, CTX-B, treats genetic obesity driven by deficiencies in the MC4R pathway, beginning with MC4R haploinsufficiency (MC4R HI) — a well-defined obesity characterized by early onset hyperphagia and obesity with no approved targeted therapeutic options.

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.

Our agonists act directly on MC4R to compensate for the lack of adequate ligand expression or augmenting 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 haploinsufficiency (MC4R HI). While we believe our drug can address the 250K+ patients in the US that have severe obesity caused by MC4R pathway mutations, we chose MC4R HI as our initial indication because of:

  • the clinical need in this severely obese population;
  • the absence of any therapy targeted to this particular segment of genetic obesity;
  • and the size of the population - an estimated 100K–150K patients, roughly half of the genetic obesity population

In preclinical models of MC4R HI, CTX-B produces significant weight loss. In preliminary non-human primate studies, it showed no cardiovascular signal at supratherapeutic exposures — a liability that has caused suspension of certain earlier programs.

Beyond Genetic Obesity

The MC4R pathway also opens a path into common obesity. In animal models, our compounds combine with various incretin therapies to deepen weight loss while sparing lean muscle and delaying weight regain after discontinuation, thereby addressing key limitations of today’s GLP-1 medicines. Our compounds also show single agent activity in animal models of dietary obesity, offering an alternative therapy for the 25% of the general population that are unresponsive to incretins or cannot tolerate them. We view general obesity as a meaningful expansion opportunity beyond our core focus on genetic obesity.

Our Pipeline

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pipeline graphic

Leadership

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

Roger Cone, Ph.D.
Co-Founder

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.
GP 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