platoseedAltay develops disease-modifying therapies targeting transcription…
Altay develops small molecule drugs to treat chronic liver diseases like liver fibrosis and liver cancer. Our co-founder Dr. Osman Ozes, led the first drug development program for Esbriet (Pirfenidone), resulting in the first FDA approved therapy for lung fibrosis. Esbriet is now a $1 billion/year drug and has already made over $6 billion in revenue. With a team that has over 70+ years of small molecule drug development experience, we intend to do the same with chronic liver diseases, which affects over 20 million Americans and represents a market size of over $30 billion. We know we are well positioned for success because we have shown excellent pre-clinical mouse data in several liver fibrosis models using our novel small molecule. We demonstrate a reduction in fibrosis with our drug, but more significantly, we show a reversal in fibrotic tissue back to normal tissue. With our current developmental strategy, we hope to have a clinic-ready compound by the end of 2021 and be in phase I clinical trials by mid of 2022.
Altay Therapeutics develops disease-modifying therapies that target transcription factors, including an oral DUX4 inhibitor for FSHD and a STAT3 inhibitor with potential in cancer and inflammatory diseases. The company emphasizes precision biology and a platform for allosteric transcription factor inhibition to create first- or best-in-class therapies.
Altay develops small-molecule inhibitors that target transcription factors through allosteric sites revealed by dynamic structural changes. Lead programs include DX5057, the first oral DUX4 inhibitor for FSHD with preclinical efficacy comparable to IV therapies and scalable oral delivery, and a STAT3 inhibitor with nanomolar potency designed to bind the activated form of STAT3 for broad therapeutic relevance and minimal off-target effects. The pipeline also includes undisclosed transcription-factor targets in oncology, with ongoing IND enabling activities and a development path from discovery to Phase 1/2a readiness.
Who it’s for: Biopharma companies, biotechnology startups, and pharmaceutical developers focusing on transcription-factor targets and disease-modifying therapies for FSHD, cancer, and inflammatory diseases.
funding mentions (investors, Golden Ticket awards, prize finalist), multiple partnerships/advisors, IND enabling and phase development timelines

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