Drug Hunters. Exploring the Unexplored.

Delivering Novel Therapies for Cancer
and Other Serious Diseases

At Orphagen, we are pioneering targeted therapies to tackle cancer and other serious diseases. With breakthroughs emerging from our internal discovery engine, we are developing a portfolio of small-molecule therapeutics that aim precisely at “orphan” or yet-to-be-explored members of the highly successful nuclear receptor drug target superfamily.

Our company name, Orphagen, denotes our exploration of the untapped potential of orphan nuclear receptors.

Our most advanced program, soon to enter the clinic for oncology in an indication considered largely untreatable, is a first-in-class antagonist for the orphan nuclear receptor steroidogenic factor-1 (SF-1).

A second area of R&D has advanced therapeutic options for major forms of autoimmune disease based on delivery of oral, small molecule drugs to novel targets.

On a Mission to Imagine the Unimagined.

Orphagen Pharmaceuticals is developing a robust pipeline of novel therapeutics by unlocking the power of orphan nuclear receptors.

Our flagship compound, OR-449, is an industry-leading, orally administered small molecule inhibitor targeting steroidogenic factor-1 (SF-1 or NR5A1). Distinguished by its unique mechanism of action, OR-449 inhibits tumor cell proliferation via SF-1 antagonism, ensuring high selectivity without resorting to non-specific cell destruction. This targeted approach is backed by promising preclinical safety and tumor model data, underscoring potential of OR-449 for the treatment of adrenocortical carcinoma (ACC) and expansion indications in precision oncology.


orphagen pharmaceuticals

Expanding our therapeutic reach and platform approach, our second major program targets inflammatory bowel disease (IBD) and other gut inflammatory conditions, such as graft versus host disease (GVHD). This initiative reflects our commitment to addressing complex immune-related disorders.

Further, Orphagen is actively advancing its portfolio with another novel pharmaceutical class, antagonists to the orphan nuclear receptor RORβ.

Our inflammatory disease and RORβ programs are currently in the lead optimization and pre-clinical validation stages.


Orphagen Team

Our scientists and management team are committed to the best we can bring to bear in pursuit of new therapies. We are drug hunters. .



Our mission is translational drug discovery and development with novel targets leading to first in class therapeutics.