Today is a special day – the type that reminds you why you chose a career in making medicines that change people’s lives.
At #ESMO25, Parabilis Medicines shared the first-ever clinical evidence that our lead investigational therapy, FOG-001, can directly & beneficially inhibit the elusive β-catenin:TCF complex, the key node in the Wnt/β-catenin pathway, implicated in millions of cancer cases every year.
𝗪𝗵𝘆 𝗧𝗵𝗶𝘀 𝗠𝗮𝘁𝘁𝗲𝗿𝘀: For 30+ years, the Wnt/β-catenin pathway has been recognized as one of the most important drivers of a range of rare & common cancers. But its flat, intracellular protein interfaces have resisted every attempt at therapeutic modulation, remaining out of reach for traditional small molecules & antibodies despite heroic efforts across industry.
Desmoid tumors – which can cause tremendous pain, immobility & suffering — arise from activating β-catenin mutations or APC loss. This floods cells with β-catenin & drives transcription via TCF, making the β-catenin:TCF interface an ideal intervention point, yet it's never been drugged—until now.
𝗢𝘂𝗿 𝗔𝗽𝗽𝗿𝗼𝗮𝗰𝗵: Helicons™, our class of stabilized α-helical peptides, were built to overcome those barriers—guided by a decade of know-how and customized AI algorithms & physics-based computational models. Their astronomical diversity, derived from thousands of non-canonical amino acid side chains, allows us to design potent & highly selective compounds that bind “flat” surfaces after gently entering cells—enabling us to engage targets once thought beyond reach.
𝗪𝗵𝗮𝘁 𝗪𝗲 𝗦𝗮𝘄: Our first preliminary data from our FOG-001 Phase 1/2 study across tumor types showed:
· Tumor reductions in every desmoid patient evaluated
· 80% objective response rate in patients with multiple post-baseline scans
· Acceptable safety and tolerability to date
This moment is a testament to the vision & perseverance of Greg Verdine—the founder of Parabilis (then FOG Pharma)—whose foundational work at Harvard in constrained peptides, unwillingness to accept industry convention, & conviction that these targets could be drugged made this milestone possible.
It’s also a testament to the Parabilis team’s persistence, creativity & conviction in bringing this science to life—to our many partners, and to the physicians, nurses & especially the patients who are participating in our trials. I am so proud of our amazing team.
Breakthroughs like this are exceedingly rare. They’re exhilarating & humbling. Most importantly, they have the potential to change the lives of patients, their families & their communities.
We’re just getting started. Read our announcement at the link in comments & look for additional data across tumors at AACR-NCI-EORTC #Targets25 & #PCFRetreat next week & in the weeks ahead at #CTOS2025 & #SNO2025.
ESMO - European Society for Medical Oncology
#Oncology #CancerResearch #Biotech #DrugDiscovery #WntPathway #PeptideTherapeutics #CreatingExtraordinaryMedicines
