Eikonoklastes completes a license to add novel gene therapy for treating neurodegenerative diseases

Reviewed by Emily Henderson, B.Sc.Jan 20 2022

Eikonoklastes Therapeutics, a preclinical stage biopharmaceutical company, today announced it has completed a license with the University of California San Diego to add a novel gene therapy for the treatment of neurodegenerative diseases.

Notably, this includes treatment for amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease. ALS is often fatal 3-5 years after diagnosis and causes an irreversible degeneration of motor neurons, which are the nerve cells in the spinal cord and brain that move the muscles needed for breathing, walking, and swallowing, among other daily activities.

We expect to see more than 29,000 ALS cases in the United States alone by 2040. Patients and their families deserve to be able to seek novel and safe therapies to treat this devastating disease. This is just the beginning as Eikonoklastes Therapeutics expands its pipeline of disruptive new therapies to treat devastating diseases that are currently severely inadequately treated. With our top-tier team, we have the unique capability to grow even more and help even more patients."

Bruce Halpryn, PhD, Chief Executive Officer, Eikonoklastes

Brian Head, Ph.D., Professor in the Department of Anesthesiology at UC San Diego and inventor of the gene therapy technology, said his team looks forward to helping develop therapies to fight catastrophic neurodegenerative diseases. "Our gene therapy is a unique, neuroprotective approach to helping patients," said Head, who is also a consultant to Eikonoklastes. "We hope to make significant progress in the treatment of ALS and give families hope for the future."

"We are very encouraged by the world-class research from Dr. Brian Head's lab at UC San Diego and its potential to treat both familial as well as the 90%-plus cases of ALS that are sporadic. This is really a step change in gene therapy as we transition from treating monogenic diseases to now polygenic diseases" said Dr. Samuel Lee, co-founder and Chief Business Officer. "The preclinical data package for Alzheimer's is just as impressive and we are excited about the future opportunities in many other diseases."

Tom Finn, Eikonoklastes Board Member and former President of P&G Global Health Care, said the novel therapy represents a continuation of the company's efforts to expand its therapeutic pipeline. "Our stated objective is to develop innovative medicines that address significant unmet clinical needs for patients. Our therapies addressing Triple Negative Breast Cancer as well as catastrophic diseases such as ALS reflect that commitment. The management team, with an impressive, long-standing experience of successful drug development in multinational pharmaceutical companies as well as early-stage biotech companies, is well-positioned to achieve these objectives."

Eikonoklastes raised a Series A financing in Q2 2021, less than a year after emerging from stealth with its seed financing. The company was founded to rapidly advance breakthrough technologies, starting with a novel fusion protein for the treatment of Triple Negative Breast Cancer. Eikonoklastes has engineered molecules which attack three key components of the tumor microenvironment (the tumor cells, the diseased neovasculature, and tumor stem cells) by activating the body's natural immune system and is therefore efficacious without harming normal cells.