Rapidly developing disruptive treatments for today’s most challenging diseases

Eikonoklastes is an early stage biopharmaceutical company focused on rapidly advancing innovative medicines for numerous areas of significant unmet medical need, starting with neurodegenerative diseases and cancers.

We’re relentlessly striving to rapidly advance a new generation of medicines where value is most efficiently generated. Most importantly, we will significantly improve the lives of patients and families suffering from the most devastating diseases.

“We’re hyperfocused on breakthrough medicines treating diseases with the highest unmet clinical need.” – Bruce Halpryn, CEO
Unlocking value in areas of significant unmet need

We are iconoclasts passionate about building a new kind of biotech company. One that breaks the mold by developing a pipeline of novel compounds initially tailored toward areas with the strongest benefit/risk (e.g., rare disease and oncology) and then collaborating with strategic partners to further explore the immense potential in larger indications.

Eikonoklastes is pushing the frontiers in multiple areas of severe unmet clinical need. Our dynamic discovery and development approach allows us to focus on accelerated approvals and outsized returns for multiple compounds. Our first two assets are on track for Phase 1/2a studies in 2023, with several additional therapies and potential partnerships on the horizon.

Three Benchmarks for Future Opportunities
Here are the three key criteria we use as a consideration set for any future development opportunities.
In-license novel compounds with platform capability
Aim for single molecules that may treat multiple billion dollar indications that are severely inadequately treated
Focus on rare indications & molecules with breakthrough potential
Target indications likely to get preferred regulatory treatment before expanding to other opportunities
Pursue accelerated clinical trial strategies
Concentrate on initial indications for each compound with Phase 1/2 trials that may morph into a registration study
“Eikonoklastes…from the Greek word for “iconoclast”; breaker of traditional concepts, principles.” –Sam Lee, CBO
At Eikonoklastes, we have licensed and are currently developing two state of the art, groundbreaking technologies. SynCav1 is a gene therapy under development for the treatment of neurodegenerative disorders such as ALS and Alzheimer’s disease. L-ICON3 is a new generation tissue factor (TF) immunotherapy–designed to target the cell surface receptor now known to be prevalent on key components of the tumor microenvironment.
First-in-class gene therapy for neurodegenerative diseases

SynCav1 is a first-in-class, non-monogenic gene therapy platform to treat a wide range of neurodegenerative diseases with precise, targeted overexpression of Caveolin-1.

Engineered for safety
Intraspinal delivery minimizes dose requirements and significantly reduces systemic exposure and thus the possibility of liver toxicity and immunogenicity.

Unparalleled benefit
Demonstrated improvement in quality of life and survival in Amyotrophic Lateral Sclerosis (ALS) models and improved memory in Alzheimer’s disease models.

LEAD INDICATION: Our lead indication is ALS, which is eligible for Orphan Drug and Breakthrough designation. ALS is a rare neurological disease with significant socioeconomic impact whose patients are desperate for novel safe and effective therapies.

Delivering additional copies of a specific gene to induce neuroprotection
SynCav1 is a gene therapy that has been engineered by Dr. Brian Head to overexpress Caveolin-1, which organizes and regulates synaptic receptors essential for neuromuscular signaling and function. Research has shown that increasing Caveolin-1 leads to a neuroprotective effect so that nerve cells can compensate for the neurodegenerative process caused by the underlying disease, allowing the cells to stay healthier and improving their functional ability. This increase leads to healthier nerve cells and better communication between them. By maintaining nerve cell health, SynCav1 could be an option for a wide range of neurodegenerative diseases.

See How SynCav1 Works
Next generation tissue factor-targeted therapy

L-ICON3 is a novel tissue factor (TF)-targeting immunoconjugate agent, with enhanced delivery and efficacy against multiple solid tumors and other pathologic conditions.

Superior technology
Proprietary, state of the art molecule discovered and engineered by scientific founder, Dr Zhiwei Hu—one of the early pioneers of TF-targeting therapies.

Optimized for efficacy
Designed for greater potency and selectivity, improved safety, and immune activation.

LEAD INDICATION: Our lead indication is Triple Negative Breast Cancer (TNBC), which accounts for 15% of all breast cancers. TNBC is an aggressive cancer and in most cases considered incurable with an 18-month life expectancy from initial diagnosis.

Launching a 3-pronged immune attack on the tumor microenvironment
L-ICON3 is a conjugate technology targeting tissue factor for the treatment of solid tumors and other pathologic conditions. The immune conjugate is engineered with a natural high affinity ligand for tissue factor, conjugated to an IgG3 Fc. This allows the compound to bind to the tumor cells, neovasculature supplying blood to the tumors, and the tumor stem cells, to trigger complement activation (CDC) and antibody dependent cellular cytotoxicity (ADCC) killing of the pathologic cells.

See How L-ICON3 Works
Traumatic Brain Injury
Multiple Sclerosis
Ovarian Cancer
Our leadership team has a sustained record of successful drug development in multibillion-dollar pharmaceutical companies and early-stage biotech ventures as well as deep expertise in Tissue Factor biology.
Bruce Halpryn, PhD image Bruce Halpryn, PhD
Chairman and Chief Executive Officer
  • Director, New Drug Development, P&G Pharmaceuticals
  • Co-founder, Myonexus Therapeutics
  • COO, N8 Medical, Kinnear Pharmaceuticals and Myonexus Therapeutics
Sam Lee, MD, MBA, MPH image Sam Lee, MD, MBA, MPH
President and Chief Business Officer
  • Venture capitalist at CincyTech, leading multiple successful life science investments
  • Chairman, Invirsa
Paulo Fontes, MD, FACS image Paulo Fontes, MD, FACS
Chief Scientific/Medical Officer
  • Extensive experience in early and late phase drug development in multiple therapeutic areas
  • 2x founder (LyGenesis and ICaPath) and Director of UPMC’s world renowned Liver Transplant Program, Starzl Transplant Institute 
Brian Head, PhD image
Brian Head, PhD
  • World leading expert in Caveolin biology
  • Inventor of Eikonoklastes gene therapy platform
Zhiwei Hu, MD, PhD image
Zhiwei Hu, MD, PhD
  • Pioneer in Tissue Factor biology
  • Inventor of Eikonoklastes immunotherapy platform
Vishal Agrawal, PhD image Vishal Agrawal, PhD
Vice President, CMC and Process Development
  • Leading expert in antibody and gene therapy manufacturing for several successful private and publicly traded biotech companies
Scientific Advisory Board – Gene Therapy
Brian Head, PhD
Research Career Scientist, VA San Diego Healthcare System
Professor of Anesthesiology, University of California – San Diego
Russell Lonser, MD
Chairman, Department of Neurological Surgery, Ohio State University Wexner Medical Center
Director, Gene Therapy Institute, Ohio State University
Professor of Neuroscience, Ohio State University College of Medicine
Michael O’Beirne
Former Executive Director, Regulatory Strategy at Sarepta Therapeutics
Vaithianathan “Palani” Palaniappan, PhD
Chief Technology Officer, Pioneering Medicines at Flagship Pioneering
Former Head of Global Technical Operations, Sarepta
Former Global Head of Biologics and New Modality Development, Takeda
Seward Rutkove, MD
Chair, Department of Neurology at Beth Israel Deaconess Medical Center
Professor of Neurology, Harvard Medical School
Dame Pamela Shaw, DBE, MD
Founder and Director, Sheffield Institute for Translational Neuroscience
Professor of Neurology, University of Sheffield
Mark Tuszynski, MD, PhD
Director, UC San Diego Translational Neuroscience Institute
Distinguished Professor of Neuroscience, University of California – San Diego
Board of directors
Bruce Halpryn, PhD
Co-founder, Chairman and
Chief Executive Officer
Sam Lee, MD, MBA
Co-founder, President and
Chief Business Officer
Tom Finn
Retired, President Global Personal Health Care & Global Pharmaceuticals, P&G
Kenneth Morand, PhD
EIR, CincyTech
Neel Patel
Managing Principal,
Elk Capital Markets
Meyer (Sandy) Frucher
Retired Vice-Chairman,
Contact Us

    News And Publications
    PRESS RELEASE: Eikonoklastes Announces Formation of Scientific Advisory Board – Gene Therapy
    Eikonoklastes Therapeutics, a biopharmaceutical company developing a novel gene therapy platform to discover and develop critical treatments for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases, today announced the formation of a Scientific Advisory Board (SAB) – Gene Therapy, comprised of key leaders in neurology and gene therapy development. The SAB – Gene Therapy will work cohesively with management to advance the company’s gene therapy pipeline. Read More >
    PRESS RELEASE: Eikonoklastes Therapeutics and Forge Biologics Announce Viral Vector Contract Development and AAV Manufacturing Partnership
    Eikonoklastes Therapeutics, a preclinical biotech company developing treatments for today’s most challenging diseases, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced a manufacturing partnership that will advance Eikonoklastes’ AAV-based gene therapy, ET-101, into clinical trials for the treatment of patients with amyotrophic lateral sclerosis (ALS). Read More >
    PRESS RELEASE: Eikonoklastes Therapeutics Receives FDA Orphan Drug Designation for 1st-in-class Treatment of Amyotrophic Lateral Sclerosis (ALS)
    Eikonoklastes Therapeutics, a preclinical biotech company developing a 1st-in-class, AAV9-based gene therapy for treatment of sporadic and familial amyotrophic lateral sclerosis (ALS), announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for the ET-101 program. Read More >
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    PRESS RELEASE: Eikonoklastes Announces Formation of Scientific Advisory Board – Gene Therapy
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    PRESS RELEASE: Eikonoklastes Therapeutics and Forge Biologics Announce Viral Vector Contract Development and AAV Manufacturing Partnership
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    PRESS RELEASE: Eikonoklastes Therapeutics Receives FDA Orphan Drug Designation for 1st-in-class Treatment of Amyotrophic Lateral Sclerosis (ALS)
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    Subpial delivery of adeno-associated virus 9-synapsincaveolin-1 (AAV9-SynCav1) preserves motor neuron and neuromuscular junction morphology, motor function, delays disease onset, and extends survival in hSOD1G93A mice
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    PRESS RELEASE: Former NASDAQ executive, Meyer ‘Sandy’ Frucher, appointed to Eikonoklastes Therapeutics Board of Directors
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    PRESS RELEASE: Eikonoklastes Recognized by Life Sciences Review as a 2021 Top Biotech Startup
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    PRESS RELEASE: Eikonoklastes Closes Oversubscribed Series A Financing, Helping to Advance Immunotherapies Toward Clinical Trial Stage
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    PRESS RELEASE: Eikonoklastes Therapeutics Appoints Tom Finn, Retired President P&G Global Personal Health Care, to Board of Directors
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    PRESS RELEASE: Eikonoklastes Therapeutics Closes Oversubscribed Seed Financing to Advance Next-Generation Tissue Factor (TF) Immunotherapies With Initial Focus on Triple-Negative Breast Cancer
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    Neuron-targeted caveolin-1 improves neuromuscular function and extends survival in SOD1G93A mice
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    Targeting Tissue Factor for Immunotherapy of Triple-Negative Breast Cancer Using a Second-Generation ICON
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    Therapeutic Antibody-Like Immunoconjugates against Tissue Factor with the Potential to Treat Angiogenesis-Dependent as Well as Macrophage-Associated Human Diseases
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