Innovating therapies for treatment-refractory cancers and rare neuromuscular disease
RAMiller is a clinical-stage biotechnology company focused on developing innovative drugs for patients with unmet medical needs, including:
Our Mission: To improve patients' lives through innovation and breakthrough science.
Molecular glues degraders are small molecules that induce or stabilize interactions between a target protein and an E3 ligase, leading to selective degradation of disease-causing proteins. Unlike traditional inhibitors, they degrade the disease-causing proteins by leveraging the ubiquitin, proteasome system.
Selective elimination of disease-causing proteins through E3 ligase recruitment
Single molecule can bind and degrade target proteins, providing sustained therapeutic effect
Molecular glue degraders enable new treatments for escape variants
This approach will provide durable solution for unmet medical needs
This challenge requires new therapeutic approaches
United States (Latest Data)
Innovative Approach: Our next-generation molecular glues target androgen receptor signaling pathways with unprecedented precision, offering new hope for patients with treatment-resistant conditions.
Dual Mechanism: Our compounds simultaneously antagonize and degrade androgen receptor ((AR); wildtype and polyglutamine repeats-expanded) and AR splice variants by binding to the non-canonical N-terminus domain of the AR, addressing key resistance mechanisms in advanced prostate and triple negative breast cancers and Kennedy’s disease (Spinal and Bulbar Muscular Atrophy (SBMA).
Indication: Castration-resistant prostate cancer (CRPC)
Mechanism: Small molecule MGD binding to AR-NTD, antagonizing and degrading AR and AR-SVs
Status: Phase 1/1B Multiple Ascending Dose
Highlights: Well tolerated, showed early efficacy signals
Impact: Addresses AR- and AR-SV-positive CRPC with no current treatment options
Indication: Triple Negative Breast Cancer (TNBC)
Mechanism: Small molecule MGD binding to AR-NTD, antagonizing and degrading AR and AR-SVs
Status: Preclinical development stage
Highlights: Strong efficacy in multiple TNBC animal models
Safety: Large safety window
Impact: Addresses AR- and AR-SV-positive TNBC with no current treatment options
Indication: Spinal and Bulbar Muscular Atrophy (SBMA) or Kennedy’s Disease
Mechanism: Small molecule MGD binding to AR-NTD, antagonizing and degrading polyglutamine repeat-expanded AR
Status: Preclinical development stage
Highlights: Strong efficacy in various transgenic preclinical models with significant improvement in neuromuscular functions and survival
Safety: Large safety window
Impact:Addresses Kennedy’s disease, a rare genetic disease with no treatment options. Aim is to improve quality of life and survival of Kennedy’s disease patients
Castration-resistant prostate cancer (CRPC) is subdivided into non-metastatic (nmCRPC) and metastatic (mCRPC), with survival benchmarks, practical next-line choices, and where the real opportunities are.
Leading cause of cancer worldwide; projected 40M men living with it by 2040; >30,000 deaths annually in the U.S. alone
Aggressive form of breast cancer; currently no targeted treatments
Currently no targeted treatments
Co-founder, Chief Scientific Officer
Co-founder, Professor Emeritus, College of Pharmacy, UTHSC
Chief Executive Officer
Seasoned executive with over 25 years of experience in global business development, corporate strategy, and commercialization across biotech and pharma sectors.
Chief Medical Officer
Dr. Getzenberg is responsible for the development of a companion diagnostic that accompanied the Phase 3 metastatic breast cancer program. He oversaw major elements of the clinical program with the mission of bringing novel agents into clinical practice. Dr. Getzenberg provided oversight and data analysis of the Phase 2 program evaluating sabizabulin in patients with metastatic castrate resistant prostate cancer as well as involvement in the planning and execution of the VERACITY Phase 3 clinical trial of sabizabulin in this same population. He led efforts to clinically evaluate enobosarm as an agent to provide bipolar androgen therapy (BAT) in men with advanced prostate cancer in a Phase 2 trial. Dr. Getzenberg provided the clinical support on the approval and release of Entadfi for the treatment of BPH including developing the clinical messaging and strategy.
Scientific Advisor
E. William Radany, PhD, brings over 40 years of experience in the biotechnology industry, with a strong track record of scientific innovation, executive leadership, and company building. Prior to serving as CEO of AssayQuant, Dr. Radany founded the synthetic biology companies VertuBio and Verdezyne, and served as President and CEO of High Throughput Genomics, Inc., a pioneer in array-based gene expression assay technologies for the life sciences sector. Earlier in his career, he was President of Biacore and the founding CEO of NeoGenex. Dr. Radany has also held key executive roles at leading biotech firms including Xencor, Caliper Technologies, and Stratagene, where he was instrumental in forging strategic partnerships with major pharmaceutical and biotechnology companies. Dr. Radany earned his Bachelor of Science degree in Cell Biology from Colorado State University, and holds a Ph.D. in Biochemistry and Physiology from the University of Wyoming.