Sudden sensorineural hearing loss, in which hearing in one ear suddenly disappears, often cannot be recovered if early treatment is missed, yet there is no current therapy. Patients who do not respond to steroid treatment have no choice but to rely on hearing aids or cochlear implants.
Korea-based biotech NewracleScience has thrown its hat into the ring to develop a hearing loss treatment. The company is targeting not only hearing loss but also degenerative neurological diseases such as dementia with a new approach that revives the "synapse" between nerve cells. A synapse is a structure like a "communication network" that connects nerve cells and transmits signals. When this consolidation is cut off, information transfer is blocked, leading to declines in hearing or cognitive function.
On the 1st at Korea University in Seongbuk District, Seoul, Seong Jae-young, CEO of NewracleScience (a professor at Korea University College of Medicine), told ChosunBiz, "Our main goal is to prove with clinical data an innovative principle that restores synapse consolidation, so domestic technology can lead the global market and proceed to first-in-class (the first novel drug with no marketed product) commercialization."
CEO Seong Jae-young, who founded NewracleScience in 2015, is a neuroendocrinologist and an expert in identifying drug target proteins. After earning a bachelor's in zoology and a Ph.D. in molecular biology at Seoul National University, Seong served as a postdoctoral researcher at the University Medical Center Göttingen in Germany and as a professor at Chonnam National University College of Medicine. Seong is currently a professor at Korea University College of Medicine.
◇ Discovery of a "novel target" with no papers… entering the clinic after 10-plus years of research
Seong's research accelerated after the human genome map was completed in 2003. Seong said, "There are 20,000 human genes, but only about 1,000—just 5%—were drug targets," adding, "The remaining 95% was an area no one knew."
Through bioinformatics, Seong narrowed the field to about 50 candidates, and judged FAM19A5 to be the most promising target among them. This protein suppresses the formation of synapses, the junctions between nerve cells, and promotes their degradation. NewracleScience's core pipeline, NS101, inhibits the activity of this protein to maintain and restore synapses.
The criteria for selecting the target were clear: it had to be expressed only in the nervous system (central and sensory), be secreted outside the cell, and be evolutionarily conserved. These may sound simple, but they are key conditions that determine the likelihood of success in new drug development.
To reduce drug side effects, the target protein should not exist in other organs, and for a drug to act, it is easier if the protein is outside the cell. The explanation is that the more evolutionarily conserved a protein is, the more important it is for sustaining life, and thus it rarely has mutations.
In particular, the decisive factor was that the amino acid sequences are 100% identical between humans and mammals (monkeys, mice, rats, dogs, and cats). This provides a scientific basis for a very high likelihood that the therapeutic effect seen in animal studies (synapse restoration) will be reproduced in humans.
However, choosing such an unprecedented "novel target" itself was a major challenge. Seong said, "At the time, there was not a single paper on this protein," adding, "We had to identify everything from its function and mechanism of action to experimental methods from scratch."
Seong recalled, "Typically, you form a hypothesis by referring to existing research, but we had to start from a completely blank slate," adding, "We spent a long time forming and testing hypotheses, and if they were wrong, we went back to the beginning and repeated the process."
Because of this, Seong prioritized securing patents over disclosing research results. Seong said, "Publishing a paper first could affect patents, so we delayed public disclosure for a long time," adding, "We discovered the protein in 2007, but full-fledged paper publication came much later, and the key paper was published in 2025." NewracleScience has secured 232 registered patents and 110 patent applications related to FAM19A5.
◇ "Confirmed synapse restoration effect… phase 2 is on track"
NS101 is an antibody therapy that inhibits the activity of FAM19A5, a protein involved in synapse loss. FAM19A5 binds to LRRC4B, a protein important for synapse formation, and acts to reduce consolidation between nerve cells. By blocking this binding, NS101 prevents synapse disappearance and induces the recovery of damaged consolidation.
Seong likened nerve cells to "entities with contact lists." Seong explained, "You can think of one nerve cell as having about 1,000 contacts (consolidation), but as you age or develop a disease, these connections are cut one by one, and when they fall below a certain level, function declines and eventually the cell dies," adding, "If you increase the severed consolidation again, cell function can be restored."
This hypothesis was confirmed in preclinical studies. In animal experiments, administering NS101 led to reformation of damaged synapses, recovery of neural consolidation, and significant improvement in cognitive function and hearing indicators.
Similar safety and pharmacokinetic characteristics were observed in a phase 1 study in humans. A phase 2a study is currently underway in 95 patients with sudden hearing loss, and key data are expected to be released after June.
Seong said, "Synapses are the common foundational structure of all neurological diseases," adding, "We can expand beyond hearing loss to dementia, ophthalmic diseases, and motor function disorders."
The reason for choosing "hearing loss" as the first indication was the judgment that hearing loss is the disease in which the innovative principle (mechanism) of synapse restoration can be proven most objectively and quickly.
The sound of Beethoven's Symphony No. 5 ("Fate") heard with normal hearing is very different from what a cochlear implant recipient hears. Seong said, "Sound through a cochlear implant is not grand music but more like a 'chik-chik-chik' converted signal," adding, "The information is conveyed, but its quality is completely different."
Seong continued, "Before the hair cells in the ear are completely lost, neural consolidation is cut first; if we just revive the consolidation at that point, functional recovery is possible."
Seong said, "With hearing loss, we can immediately quantify a drug's efficacy through hearing tests, so even with a small patient population we can obtain statistically meaningful data, and compared with dementia—where confirming cognitive improvement takes a long time—it is advantageous for proof of concept (PoC)," adding, "Another key reason is that it is an 'untapped market' with no clear therapy."
Global pharmaceutical companies are also jumping into hearing loss drug development, but their strategies differ from this company's. U.S. drugmakers such as Eli Lilly and Company and Regeneron are targeting specific hereditary hearing loss based on gene therapy, while NewracleScience is first developing for sudden sensorineural hearing loss and plans to expand indications to noise-induced hearing loss, ototoxic hearing loss, and presbycusis.
Seong said, "Gene therapy has limited eligible populations and a relatively small market," adding, "By contrast, we target general hearing loss caused by aging or environmental factors, so the market is much larger."
◇ Targeting "technology export" as the first step to going global
The industry expects the phase 2 results of NS101 to be a watershed for technology export (licensing out). Seong also said, "After we secure the data, we will move in earnest to collaborate with global pharmaceutical companies."
Seong described the research and development journey so far as "a series of lucky breaks." New drug development is structured so that failures can halt progress at each stage. If a toxicity test fails, development stops; if serious side effects occur in clinical trials, a retry is virtually impossible.
Seong said, "From discovering the FAM19A5 target and researching NS101 to passing gates like nonclinical toxicity testing and a phase 1 trial in patients, we've had luck five or six times," adding, "Even though it was a road no one had taken, the scientific evidence verified through multiple gates gave us the strength to keep developing the drug."
NewracleScience plans to first commercialize NS101 in sudden sensorineural hearing loss and then expand indications to neurotology disorders and neurodegenerative diseases such as dementia. That is because synapses are a structure that acts commonly across all neurological diseases. Seong said, "The frontotemporal dementia trial already enrolled its first patient in Dec. last year and is proceeding smoothly."
Financing will proceed in parallel. The company plans to raise a roughly 30 billion won pre-IPO investment this year and pursue a KOSDAQ listing next year, starting with a technology evaluation application after confirming topline hearing loss clinical data. It recently completed a 21.3 billion won rights offering.
Seong said, "In the long term, our goal is to grow into corporations that bring in external technology and build it out, like global pharmaceutical companies," adding, "With domestic technology, we will set a new standard for treating neurological diseases."