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 most advanced program, soon to enter the clinic in oncology, is a first-in-class antagonist for the orphan nuclear receptor steroidogenic factor-1 (SF-1). Major indications include the rare and largely untreatable cancer, adrenocortical cancer (ACC), where SF-1 is universally expressed, and significant subsets of head and neck and lung squamous cancer where SF-1 is highly elevated.

Orphagen is an IND-ready oncology therapeutics company supported by a novel target discovery and development pipeline.

On a Mission to Imagine the Unimagined.

Orphagen has developed a robust pipeline of novel therapeutics by unlocking the power of orphan nuclear receptors. Our company name, Orphagen, denotes our exploration of the untapped potential 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 blocks cell proliferation in sensitive tumors that express high levels of the SF-1 target. This targeted approach is backed by promising preclinical safety, underscoring the potential of OR-449 for the treatment of adrenocortical carcinoma (ACC) and expansion opportunities in head and neck and lung squamous cancers.

orphagen pharmaceuticals

Orphagen is advancing its oncology portfolio with a separate, novel pharmaceutical class, antagonists to the orphan nuclear receptor RORβ, for precision therapy of small cell lung cancer, subtypes of leukemia, and potentially other cancers.

Expanding our therapeutic reach and platform approach, a third program targets inflammatory bowel disease (IBD) and other gut inflammatory conditions, such as graft versus host disease (GVHD).

The RORβ and inflammatory disease programs are currently at 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.