Orphagen Pharmaceuticals

At Orphagen, we are driven by a passion to pioneer targeted therapies for cancer and other serious diseases. Our mission is to harness the untapped potential of orphan nuclear receptors to deliver innovative treatments that make a meaningful difference in patients' lives. Our team of renowned scientists and experts in the field of life sciences has spent decades researching and developing novel therapeutics for the most pressing healthcare challenges.

Our Science

Our innovative drug discovery and development pipeline is built around a proprietary target discovery and development platform. This unique approach allows us to identify and validate novel nuclear receptors that are critical to specific diseases. We then design and develop small molecule inhibitors that target these receptors with unparalleled precision. Our flagship compound, OR-449, is an industry-leading, orally administered small molecule inhibitor targeting steroidogenic factor-1 (SF-1 or NR5A1). This targeted approach has shown promising preclinical safety and has the potential to treat adrenocortical carcinoma (ACC) and other indications.

Our Programs

In addition to our most advanced program, OR-449, we are also advancing a separate program targeting inflammatory bowel disease (IBD) and other gut inflammatory conditions, such as graft versus host disease (GVHD). This program leverages our proprietary technology to develop selective antagonists to the orphan nuclear receptor ROR for precision therapy of small cell lung cancer, subtypes of leukemia, and potentially other cancers.

Our Team

At Orphagen, we are a team of dedicated professionals who are committed to pushing the boundaries of science and innovation. Our experts have a proven track record in life sciences research, pharmaceutical R&D, and entrepreneurial ventures. Our leadership team is led by Scott Thacher, Ph. D., who has over 30 years of expertise in life sciences research and pharmaceutical R&D. Alongside our scientific advisors, we have secured numerous grants and partnerships, creating a solid foundation for our innovative drug discovery and development efforts.

Our Founders and Scientific Advisors

Our founders and scientific advisors are renowned experts in their fields, with a combined total of over 100 years of research and development experience. Our leadership team includes:

• Scott Thacher, Ph. D., Founder and CEO of Orphagen Pharmaceuticals
• Paul Crowe, Ph. D., Vice President of Drug Discovery
• Neil Raheja, Ph. D., Director of Chemistry
• Michael Wood, MBA, Business Advisor
• Len Post, Ph. D., Orphagen Board Member since 2007
• Gary D. Hammer, MD, PhD, Senior Advisor

Each member of our team has a unique blend of scientific expertise, industry experience, and entrepreneurial drive, which enables us to effectively tackle even the most complex scientific challenges. We are proud to have built a team that is passionate about delivering novel therapies and making a meaningful impact on patients' lives.