Business Details
18 Years
experience
Innovative Oncology & Autoimmune Disease Therapies
Orphagen Pharmaceuticals is a pioneering pharmaceutical company dedicated to discovering and developing first-in-class small molecule therapies for cancer and serious diseases. Our research focuses on targeting novel members of the nuclear receptor superfamily, a proven class of therapeutic targets with applications in oncology, inflammation, and metabolic diseases. With a team of seasoned scientists and industry leaders, we have advanced multiple programs into preclinical and clinical development, including OR-449 for oncology and OR-812 for inflammatory bowel disease. Our commitment to innovation is underscored by strategic collaborations, significant grant funding, and a robust pipeline driven by scientific excellence. We actively engage with clinical, scientific, and patient communities to translate groundbreaking research into transformative therapies.Our leadership team brings decades of pharmaceutical R&D expertise, including prior roles at leading institutions and biopharma companies. We prioritize objective research practices and maintain transparency through publicly available policies, including our NIH-compliant Financial Conflict of Interest policy. Our work is supported by prestigious grants from organizations like the National Institutes of Health and the Cancer Prevention and Research Institute of Texas. By focusing on unmet medical needs, we aim to deliver novel oral therapies that address significant patient care gaps in both rare and common diseases.
Services
Oncology drug development
Autoimmune disease therapies
Nuclear receptor targeting
Preclinical research programs
Clinical trial execution
Details
- Year established: 2008
Highlights and features
- We have 30+ years of pharmaceutical R&D expertise from Harvard-trained scientists
- Our pipeline includes first-in-class therapies with FDA Rare Pediatric Disease Designation
- We've secured $11.9M+ in competitive NIH and CPRIT grants for clinical development
- Our team has advanced 10+ compounds to clinical development at major pharma companies
- We maintain strategic collaborations with leading academic institutions and research organizations
Services
OR-449 Oncology Program
OR-449 is a first-in-class orally-bioavailable antagonist targeting steroidogenic factor-1 (SF-1) for adrenocortical cancer treatment. This precision therapy shows promise for both pediatric and adult populations, with FDA Rare Pediatric Disease Designation and $10.2M CPRIT grant support. Clinical development includes Phase 1 trials for adult ACC.
OR-812 Autoimmune Program
OR-812 is a retinoic acid receptor antagonist developed for inflammatory bowel disease, including Crohn's disease and ulcerative colitis. This novel oral therapy offers a differentiated mechanism of action compared to existing treatments, supported by $1.7M NIH funding for further development.
ROR Antagonist Research
Our ROR antagonist programs focus on small cell lung carcinoma and leukemia treatments. These potent ligands demonstrate broad gene expression modulation, with preclinical evidence supporting their therapeutic potential in hematological cancers and solid tumors.
Nuclear Receptor Targeting
We specialize in developing therapies targeting nuclear receptors, leveraging high-throughput assays and compound libraries to identify selective chemical probes. Our expertise spans agonist and antagonist development for precision medicine applications in multiple disease areas.
Clinical Development Programs
Our clinical development capabilities include IND-enabling studies, Phase 1 trials, and preclinical pharmacology. Supported by NIH grants and industry partnerships, we advance compounds from discovery through early clinical testing with a focus on safety and efficacy.
People
Scott Thacher
Founder and Chief Executive Officer
30+ years pharmaceutical R&D experience, former Allergan director, Harvard PhD, NIH fellow, and author of 30+ publications. Co-founded San Diego Entrepreneurs Exchange and secured 20+ NIH grants for drug discovery programs.
Paul Crowe
Vice President of Drug Discovery
15+ years leading preclinical programs at Orphagen Pharmaceuticals, former Senior Director at Neurocrine Biosciences. PhD in Pharmacology with expertise in CNS, autoimmunity, and metabolic disease programs, including industry collaborations with Janssen Pharmaceuticals.
Jukka Muhonen
Vice President of Business Development
25+ years pharma business development experience, former Orion Pharma VP. Orchestrated major industry deals with Bayer and Merck, including darolutamide and opevesostat partnerships. Holds MBA and dual law degrees from top international institutions.
Andrew Dorr
Chief Medical Officer
Board-certified oncologist with NCI and Eli Lilly experience. Specializes in Phase 1 trial design, with academic appointments at University of Texas and University of Indiana. Provides clinical leadership for our oncology development programs.
Neil Raheja
Director of Chemistry
Organic chemistry expert with 20+ years pharmaceutical experience. Led 10+ clinical programs at Pfizer, Celgene, and Ionis. PhD from Wayne State University, NIH postdoctoral fellow, and co-founder of two biotech startups.
Questions
What makes Orphagen's approach to drug discovery unique?
We focus on unexplored nuclear receptor targets using high-throughput assays and compound libraries to identify selective chemical probes that become first-in-class therapeutics.
What diseases does Orphagen currently target?
Our primary focus areas are oncology (particularly adrenocortical cancer) and autoimmune diseases like inflammatory bowel disease, with exploratory programs in hematological cancers.
How does OR-449 differ from existing cancer treatments?
OR-449 targets SF-1 with tissue selectivity for adrenal gland, offering improved safety and efficacy compared to traditional chemotherapy, immunotherapy, and tyrosine kinase inhibitors.
What clinical development support does Orphagen have?
We've received $11.9M+ in grants from NIH and CPRIT, with FDA Rare Pediatric Disease Designation for OR-449 and established partnerships for clinical trial execution.
How does Orphagen ensure research integrity?
We maintain transparent research practices with publicly available Financial Conflict of Interest policies and adhere to NIH grant compliance requirements for all funded programs.
What collaboration opportunities exist with Orphagen?
We seek partnerships for clinical development, particularly for OR-449 and our ROR antagonist programs, leveraging our proprietary nuclear receptor targeting technology for mutual therapeutic advancement.
