Business Details
Pioneering First-in-Class Therapies
Orphagen Pharmaceuticals is a pioneering biopharmaceutical company dedicated to discovering and developing first-in-class small molecule therapies targeting nuclear receptors for oncology and autoimmune diseases. Our mission centers on translational drug discovery, leveraging proprietary high-throughput assays and extensive compound libraries to identify selective chemical probes. We focus on unexplored or orphan nuclear receptors, optimizing lead compounds through iterative medicinal chemistry to improve drug-like properties. Our most advanced programs include OR-449 for adrenocortical cancer and OR-812 for inflammatory bowel disease, both supported by significant grants and partnerships. We collaborate with clinical, scientific, and industry leaders to accelerate our path from discovery to clinical development.Our team combines decades of pharmaceutical R&D expertise with a commitment to addressing significant unmet medical needs. We prioritize oral bioavailability and tissue selectivity to enhance therapeutic efficacy and safety. Our work has attracted partnerships with major pharmaceutical companies and recognition from regulatory bodies like the FDA. We maintain a robust pipeline spanning oncology and autoimmune diseases, driven by a passion for innovation and patient impact. Our scientific approach integrates genomic insights, pharmacological validation, and preclinical models to advance novel therapies.
Services
Oncology Drug Development
Autoimmune Disease Therapies
Nuclear Receptor Research
Preclinical Validation
Clinical Development Partnerships
Highlights and features
- We target unexplored nuclear receptors with proven therapeutic potential.
- Our team has 30+ years of pharmaceutical R&D expertise.
- We maintain partnerships with major pharmaceutical companies.
- Our pipeline includes FDA-designated rare disease therapies.
- We integrate genomic insights with preclinical validation.
Services
Oncology Drug Development
Orphagen Pharmaceuticals develops first-in-class oncology treatments targeting nuclear receptors. Our lead candidate OR-449 addresses adrenocortical cancer through SF-1 antagonism, with potential applications in head/neck and lung squamous cancers. The program has received FDA Rare Pediatric Disease Designation and $10.2M CPRIT grant support for clinical development.
Autoimmune Disease Therapies
Our OR-812 program targets inflammatory bowel disease through RAR antagonism. This novel oral therapy modulates gut homing receptors and T regulatory cell activity, showing promise in preclinical colitis models. Supported by $1.7M NIH funding, it offers differentiation from existing biologics and small molecules.
Nuclear Receptor Research
We specialize in orphan nuclear receptor exploration using proprietary high-throughput assays. Our platform identifies selective chemical probes, validates direct receptor binding, and optimizes lead compounds through iterative medicinal chemistry. This approach has yielded three clinical-stage programs targeting SF-1, RAR, and ROR.
Preclinical Validation
Our preclinical programs employ diverse models including patient-derived xenografts and genetically characterized cell lines. We measure tumor DNA synthesis inhibition, steroid regulation, and gut inflammation reduction to validate therapeutic mechanisms before clinical translation.
Clinical Development Partnerships
We form strategic partnerships with academic institutions, pharmaceutical companies, and funding agencies. These collaborations accelerate drug development through shared expertise, resources, and clinical trial networks, as demonstrated by our history with Merck, Pfizer, and NIH-supported programs.
People
Scott Thacher
Founder and CEO
30+ years pharmaceutical R&D experience, former Allergan director, Harvard biophysics PhD, NIH postdoc, and academic faculty member. Secured 20+ NIH grants and industry partnerships for Orphagen Pharmaceuticals's programs.
Paul Crowe
VP Drug Discovery
Led Orphagen Pharmaceuticals's discovery research since 2008. Former Neurocrine Biosciences director with expertise in CNS, autoimmunity, and metabolic disease programs. PhD in Pharmacology from University of South Florida.
Jukka Muhonen
VP Business Development
25+ years pharma BD experience, former Orion Pharma VP. Orchestrated major deals with Bayer and Merck. MBA from Henley Business School and dual law degrees from Illinois and Helsinki universities.
Andrew Dorr
Chief Medical Officer
Oncology expert with NCI and Eli Lilly experience. UNC medical graduate, former Indiana associate professor. Specializes in Phase 1 trial design and execution for novel cancer therapies.
Neil Raheja
Director of Chemistry
Designed OR-449 and OR-812 compounds. Led 10 clinical programs at Pfizer. PhD in Organic Chemistry from Wayne State University with NIH postdoctoral fellowship at UPenn.
Questions
What diseases does Orphagen focus on treating?
We specialize in oncology (adrenocortical cancer, head/neck squamous cancers) and autoimmune diseases like inflammatory bowel disease.
What makes your drug candidates unique?
Our therapies target novel nuclear receptors with first-in-class mechanisms, offering tissue-selective treatments for unmet medical needs.
Are your therapies in clinical trials?
OR-449 for ACC is entering clinical trials with FDA Rare Pediatric Disease Designation and CPRIT grant support.
How do you validate drug targets?
We use high-throughput assays, orthogonal validation, and preclinical models with genomic and pharmacological correlation.
Do you collaborate with other organizations?
Yes, we partner with academic institutions, pharmaceutical companies, and funding agencies like NIH and CPRIT.
