Cytora, a clinical-stage biopharmaceutical company, has announced successful results from its Phase 1/2a clinical trial evaluating hOMSC200, an allogeneic stem cell therapy for treating chronic diabetic foot ulcers (DFUs). The trial demonstrated both safety and significant efficacy advantages over standard care, potentially offering new hope for a condition that affects millions of diabetes patients worldwide.
The study enrolled 21 patients with particularly challenging cases of DFUs, with wounds that had persisted for an average of 27 months. Results showed that hOMSC200 was well-tolerated with no adverse events or immunological rejection responses reported, addressing a key safety concern for allogeneic cell therapies.
"Diabetic foot ulcers are a common complication of diabetes and are extremely difficult to treat, leading to significant morbidity," explained Yona Geffen, PhD, CEO of Cytora. "We are targeting the DFU patient population that do not respond to available treatment options."
Significant Efficacy Improvements Over Standard Care
The trial was designed as a single-center, randomized, partially blinded study with two hOMSC200 dose groups (low and high) and a placebo control group. All participants also received standard wound care treatments throughout the 18-month follow-up period.
Efficacy data revealed compelling advantages for hOMSC200:
- In the intent-to-treat analysis, the treatment group achieved a 53% wound closure rate compared to 33% in the placebo group
- In the per-protocol population (12 patients who strictly adhered to study guidelines), hOMSC200 demonstrated a remarkable 100% success rate versus only 20% in the placebo group
- No significant differences were observed between the low and high dose groups, suggesting efficacy at even the lower dose level
These results are particularly significant given the challenging nature of the enrolled patient population, whose wounds had failed to respond to conventional treatments for over two years on average.
Novel Stem Cell Platform with Unique Properties
hOMSC200 is derived from human Oral Mucosa Stem Cells (hOMSCs), a unique stem cell population discovered in the oral mucosa. These cells originate from neural crest tissue and retain properties that make them particularly effective for treating complex conditions.
"We are at the forefront of cell therapy, leveraging a proprietary unique stem cell population discovered in the human oral mucosa," stated Prof. Sandu Pitaru, Co-Founder and CTO of Cytora. "Notably, the embryonic origin of this population is a primordial tissue of the developing embryonic brain. These unique stem cells retain in the adult's oral mucosa the properties of neural and other stem cell types, making them effective in targeting chronic multifaceted diseases."
The technology offers several key advantages:
- A small biopsy (4x3x2 mm) from a healthy donor can generate thousands of treatment doses
- The cells can be propagated without losing their stem cell properties
- They do not trigger immune rejection when transplanted into allogeneic recipients
- Their wound healing capabilities remain largely unaffected by diabetes, unlike other tissues
Addressing a Significant Unmet Medical Need
Diabetic foot ulcers represent a substantial burden for patients and healthcare systems. Approximately 19-34% of individuals with diabetes develop DFUs during their lifetime, with current treatments achieving only a 75% healing rate. About 17% of cases ultimately require amputation, and patients with DFUs face significantly higher mortality rates than those with diabetes alone.
The economic impact is equally substantial, with approximately 1.2 million new DFU patients annually in the US alone. Average treatment costs reach $20,000 per patient, with severe cases requiring amputation exceeding $40,000. The global market for DFU treatments is estimated at $7.6 billion annually.
Future Development Plans
Based on these promising results, Cytora plans to apply for an Investigational New Drug (IND) application during the second half of 2025, which would enable the initiation of a larger Phase 2 trial for DFU treatment in 2026.
The company is also exploring applications of its hOMSC technology for other conditions, including neurodegenerative diseases such as Parkinson's Disease, Multiple System Atrophy (MSA), and Alzheimer's Disease. A Phase 1 study for MSA treatment is currently underway.
"The results of this trial put us on solid footing for applying for an IND during the second half of 2025 that will enable starting a large-scale Phase 2 trial for treating DFUs during 2026," said Dr. Geffen.
Founded in 2018, Cytora's technology platform is based on the discoveries of Prof. Sandu Pitaru from the Faculty of Medicine, School of Dentistry at Tel Aviv University in Israel. The company's innovative approach to stem cell therapy could potentially transform treatment options for patients with chronic, difficult-to-treat conditions.
