A Cincinnati-based biotech startup developing a first-in-class treatment for ALS is celebrating a key regulatory milestone, roughly two years after landing its first tranche of funding from Uptown-based investment firm CincyTech.
Eikonoklastes Therapeutics, a preclinical biotech company developing new drugs for neurodegenerative diseases and cancers, this week received U.S. Food and Drug Administration orphan drug designation for its ET-101 program. According to the FDA, orphan drug designation supports drugs that aim to prevent, diagnose or treat a rare diseases or conditions.
The designation, Eikonoklastes said, reflects “compelling data” for ET-101 and its potential as a treatment option for both familial and sporadic ALS. It qualifies sponsors for tax credits for clinical trials, among other incentives.
ALS, also known as Lou Gehrig’s Disease, is a rare, progressive, debilitating – and universally fatal – neurodegenerative disease that affects the motor neurons connecting the brain and spinal cord. Degeneration of these neurons leads to paralysis as the brain loses its ability to control muscle movement.
Most people with ALS succumb to respiratory failure, usually within three to five years from when symptoms first appear.
An estimated 10% of ALS is familial and caused by inherited genetic mutations. Around 90% of sporadic ALS may be due to a combination of environment and genetic risk factors. There is currently no cure for ALS.
“The ODD (orphan drug designation) program is critical for incentivizing the development of new treatments for rare diseases, especially those like ALS with severe morbidity and mortality,” Dr. Sam Lee, president and chief business officer of Eikonoklastes, said in a release. “Achieving orphan drug designation is an important regulatory milestone that further validates our efforts. We look forward to rapidly advancing this novel technology toward our first-in-human clinical trial.”
Lee told me Eikonoklastes expects to begin its phase 1/2 study in the next 12-18 months. The company has not finalized a site or sites but has been talking to the leading U.S. academic centers, he said.
Eikonoklastes licenses ET-101, which was pioneered by Brian Head, a researcher at the University of California-San Diego.
ET-101 has been engineered to overexpress caveolin-1, which organizes and regulates synaptic receptors essential for neuromuscular signaling and function. While the company's first target is ALS, ET-101 could also have applications for Alzheimer’s, multiple sclerosis, traumatic brain injury, Huntington’s and Parkinson’s diseases.
Eikonoklastes also is looking to develop a new generation of therapies that target triple negative breast cancer, an aggressive and often lethal form of breast cancer.
The company added a Series A round in June 2021, backed by JobsOhio, Columbus-based Rev1 and others. It did not disclose the total amount of either raise, but Lee put the company's total funding at $14.5 million.
Besides Lee, a Miami University grad who earned his medical degree from the University of Cincinnati, the Eikonoklastes leadership team includes co-founder Bruce Halpryn, a former director at P&G Pharmaceuticals; and Dr. Mark Dato, chief scientific/medical officer, who worked with Halpryn at P&G.
Overall, the company has a full-time staff of four and works with a handful of consultants.
“Our goal is to significantly increase survival and improve quality of life for patients,” Halpryn, chairman and CEO of Eikonoklastes, said in the release.
