Rapidly developing disruptive treatments for today’s most challenging diseases
Eikonoklastes Therapeutics™ is an early stage biopharmaceutical company focused on rapidly advancing innovative medicines for numerous areas of significant unmet medical need, starting with neurodegenerative diseases.
We are relentlessly striving to rapidly advance a new generation of medicines that have potential to significantly improve the lives and lifespans of patients and families suffering from the most devastating diseases, while creating significant value for all stakeholders.
We are iconoclasts who are passionate about finding new and better ways to treat disease. We are developing a pipeline focused on neurodegenerative diseases with the strongest benefit vs. risk.
Our initial focus is on Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig’s Disease), a rare, yet debilitating nerve disease that causes progressive muscle degeneration and eventual death. An estimated 5,000 new patients in the U.S. are diagnosed each year with ALS, and there are few treatment options beyond supportive care. Our dynamic discovery and development approach allows us to focus on accelerated approvals and outsized returns for ALS and potentially other neurodegenerative diseases.
Caveolin-1 in neurons. This equips those neurons to compensate for the damage caused by various neurodegenerative diseases such as ALS, Huntington’s Alzheimer’s, Parkinson’s, Multiple Sclerosis, and more.
Engineered for safety
Intraspinal delivery minimizes the dose to only ~1% of what systemic delivery would require, reducing potential for liver toxicity and immunogenicity. Synapsin promoter prevents gene from expressing Caveolin-1 in non-neuronal tissue. AAV9 capsid provides well characterized neuronal delivery and other actions.
Unparalleled benefit
Significant, unparalleled improvement in functional capability, quality of life and survival in ALS and other neurodegenerative disease models.
LEAD INDICATION: Our lead indication is ALS, which received Orphan Drug Designation from the FDA, and could be eligible for Breakthrough Designation. ALS is a rare, neurodegenerative 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
ET-101/SynCav1 is a gene therapy that was originally engineered in the lab of Dr. Brian Head at UC-San Diego 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/neuroplasticity 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.
| INDICATION | DISCOVERY | IND-ENABLING | FIRST IN HUMAN | PIVOTAL | |
|---|---|---|---|---|---|
| ALS | |||||
| Alzheimer’s | |||||
| Parkinson’s, Huntington’s, MS, TBI, and more | |||||
Bruce Halpryn, PhD
- Director, New Drug Development, P&G Pharmaceuticals
- Co-founder, Myonexus Therapeutics
- COO, N8 Medical, Kinnear Pharmaceuticals and Myonexus Therapeutics
Dr. Bruce Halpryn co-founded Eikonoklastes in 2019 and has served as Chairman of our Board of Directors and Chief Executive Officer since 2019. Dr. Halpryn has more than 30 years of experience in the industry with involvement in the formation and the leadership of four early stage biotechnology companies as well as global biopharmaceutical companies. Prior to Eikonoklastes, Bruce co-founded Myonexus Therapeutics (acquired by Sarepta in 2018), and served as Chief Operating Officer and as a member of its Board of Directors. Dr. Halpryn has extensive experience in designing and leading biologics and small molecule drug development programs and companies across a broad range of therapeutic areas.
Earlier in his career, Dr. Halpryn led the Congestive Heart Failure Research Lab at Norwich Eaton Pharmaceuticals before its acquisition by the Proctor & Gamble Company and subsequently renamed P&G Pharmaceuticals. He then led drug discovery, development, and commercialization efforts for P&G Pharmaceuticals, playing a key role in the strategic design and program leadership of numerous successful approvals.
Bruce is a cardiovascular physiologist by education and conducted his post-doctoral fellowship as a National Research Council Fellow at NASA/Ames Research Center. He holds a Ph.D. in Biology/Physiology from SUNY Binghamton.
Sam Lee, MD, MBA, MPH
- Venture capitalist at CincyTech, leading multiple successful life science investments
- Chairman, Invirsa
Dr. Samuel Lee co-founded Eikonoklastes in 2019 and has served as a member of our Board of Directors and Chief Business Officer since 2019. Dr. Lee brings a multidisciplinary convergence of experiences in the life sciences, physical sciences, and computer sciences to successfully develop breakthroughs in healthcare. Prior to Eikonoklastes, Sam was a Director at CincyTech, a venture capital firm, where he led investments and actively supported operations in biotech and medical device portfolio companies such as Myonexus (acquired by Sarepta), Standard Bariatrics (acquired by Teleflex), and Blue Water Vaccines (NASDAQ: BWV).
Dr. Lee completed his medical training at the University of Cincinnati, formal business and public health education at the Stanford Graduate School of Business and Johns Hopkins, and post-graduate research fellowship at Northwestern University and Cincinnati Children’s Hospital Medical Center. Dr. Lee also serves as Chairman of the Board of Directors at Invirsa and is an Executive-in-Residence at CincyTech.
Hernán Picard, MD, PhD
- Physician-Scientist with extensive experience developing therapeutics in neuroscience, inflammation, and rare diseases from IND-enabling and first-in-human through registration and post-approval studies.
- Led and developed high-performing teams at global pharmaceutical and small biotech companies to support innovation for patients and stakeholder value.
Dr. Hernán Picard is the Chief Scientific/Medical Officer at Eikonoklastes Therapeutics, where he leads translational research and clinical development of our first-in-class gene therapy programs. Hernán is a physician-scientist with extensive experience developing small molecules and biologics in neuroscience, inflammation, and rare diseases from IND-enabling and first-in-human through registration and post-approval studies. Before joining Eikonoklastes, Hernán was the Head of Global Medical Science at Mitsubishi Tanabe Pharma America overseeing a pipeline of 10 assets in 13 different rare disease programs in neuroscience and inflammation.
Previously, Hernán served as Vice President of Clinical Science at Alector, a company specialized in immunological pathways in neurodegenerative diseases. Prior to that, Hernán spent 9 years at Amgen, where he held positions of increasing responsibility in clinical development, from European to Global leadership roles and then Executive Medical Director of several key clinical-stage and blockbuster assets in neuroscience and inflammation. Hernán began his career in industry as a Clinical Research Physician at Eli Lilly supporting research in Alzheimer’s disease and psychiatry/neuropathic pain.
Dr. Picard earned his medical degree from the University of Buenos Aires. He also earned a Masters in Cognitive Sciences and a PhD in Cognitive Neuroscience from Pierre & Marie Curie University in Paris, France.
Vishal Agrawal, PhD
- Leading expert in antibody and gene therapy manufacturing for several successful private and publicly traded biotech companies
Dr. Vishal Agrawal joined Eikonoklastes in 2022 as our Vice President of CMC and Process Development. Prior to joining Eikonoklastes, Vishal worked at a number of successful private and publicly-traded biotech companies with increasing responsibility in both antibody and gene therapy manufacturing. Dr. Agrawal completed his Bachelors and Masters in Biochemical Engineering at The Indian Institutes of Technology Delhi and his PhD in Cell Biology at Max-Delbruck Centre.
- World leading expert in Caveolin biology
- Inventor of Eikonoklastes gene therapy platform
Professor of Anesthesiology, University of California – San Diego
Director, Gene Therapy Institute, Ohio State University
Professor of Neuroscience, Ohio State University College of Medicine
Professor of Neurology, Harvard Medical School
Professor of Neurology, University of Sheffield
Distinguished Professor of Neuroscience, University of California – San Diego
Chief Executive Officer
Chief Business Officer
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