InGeneron Shares Results From Phase III Knee Osteoarthritis Study

InGeneron Inc. is sharing results of an Emory University-sponsored study that found no serious adverse events from its regenerative cell therapy treatment for knee osteoarthritis (OA) pain.

Initial study findings, published in Nature Medicine, investigated the efficacy of InGeneron’s regenerative cell therapy in comparison to two other orthobiologic treatments—autologous bone marrow aspirate concentrate (BMAC) and autologous stromal vascular fraction (SVF)—and the conventional corticosteroid injection (CSI) for knee osteoarthritis. Importantly, the study reported no serious treatment-related adverse events from any of the 109 patients treated with InGeneron’s Transpose RT cell therapy system. The primary outcome measures were the visual analog scale (VAS) and the Knee Injury and Osteoarthritis Outcome Score (KOOS) for pain from baseline to one year. The question driving the research was whether cell therapies could outperform corticosteroids in treating knee osteoarthritis pain at the one-year mark.

While each group in the Phase III 480-patient study had a measurable improvement in pain and function, the data showed there was no significant advantage to using any of the tested cell products compared to anti-inflammatory CSI treatment at 12 months in relieving knee OA pain. Similarly, the KOOS pain score analysis produced consistent results, with no significant differences between groups at the 12-month mark in the change in score from baseline.  
 
“Additional information is necessary to fully interpret the MILES study’s outcomes,” said Dr. Christoph Schmitz, head of the Department of Anatomy II at Ludwig-Maximilians University of Munich (Germany) and Advisory Medical Director at InGeneron. “Specifically, the [study] does not clarify if the reported primary outcome measures, including the Visual Analog Scale for Pain and the Knee Injury and Osteoarthritis Outcome Score Total scores, are absolute or relative changes. Also, the publication does not provide the baseline data of these scores, and without access to these baseline data it is challenging to gauge the true impact of the treatments. Moreover, the study presents an unexpectedly positive one-year outcome for corticosteroid treatment, which deviates from prior research. A discussion on this contrast would have been beneficial. Nonetheless, we welcome the pursuit of additional analyses, ensuring we do not overlook any meaningful treatment effects.”
 
Specifically, InGeneron proposes a subgroup analysis focusing on patients who entered the trial with moderate VAS pain scores, determined by the literature to be between 30 and 60 (on a scale of 0 to 100, where 0 indicated no pain and 100 indicates maximum pain). In clinical research, especially for conditions with variable symptoms like knee OA, selecting a patient population that best represents those who would benefit from the treatment in a real-world setting is crucial for interpreting the effectiveness of a therapeutic intervention. The reasons for conducting a subgroup analysis of patients with moderate pain scores at baseline are as follows:
 

• Sensitivity to Change: Patients with moderate pain scores are in the mid-range of the VAS Pain score and thus may have a greater sensitivity to change. This means that improvements or deteriorations in pain and function can be more readily detected than in patients with mild or severe pain, where a floor or ceiling effect can mask changes. 
• Optimization of Treatment Effects: For regenerative therapies, patients with severe pain might experience significant pain relief and joint function improvement by repeated application of the therapy rather than a single application. Conversely, patients with mild pain may not show dramatic improvements simply because their baseline pain is already low. Moderate pain patients stand to show a more pronounced response, thus providing a clearer measure of treatment efficacy.
• Real-world Applicability: Targeting the subgroup of patients with moderate pain scores for analysis mirrors the likely real-world use of the therapy. It is this group of patients that clinicians are often deciding whether to treat with conservative management or advance to more interventional therapies like InGeneron’s regenerative cell therapy.
• Guideline and Policy Influence: Focusing on this subgroup could influence clinical guidelines and insurance policies by providing strong evidence for when and for whom these treatments are most effective, thereby affecting treatment protocols and access to care. 

Although more information and further analysis is needed, InGeneron executives said they are encouraged by the MILES study’s finding that Transpose RT therapy surpassed the threshold for clinical efficacy while maintaining an “exceptional safety profile.” These findings emphasize the significant potential of InGeneron’s technology, which is currently being evaluated in three ongoing U.S. Food and Drug Administration (FDA)-approved clinical trials for treating partial-thickness rotator cuff tears, wrist osteoarthritis, and facet joint syndrome.
 
InGeneron’s Transpose RT cell therapy platform consists of a processing unit, a set of disposables, and Matrase, a proprietary enzyme mixture. The platform allows the isolation of regenerative cells from the patients’ own adipose tissue at point-of-care for same-day treatment. The cells are re-administered into the patient’s damaged tissue by injection under ultrasound or fluoroscopic guidance. The Transpose RT System is available in the United States for research purposes only. 
 
InGeneron is a clinical-stage biotechnology company developing novel evidence-based cell therapies. The company is focused on helping patients impacted by orthopedic conditions and is conducting several clinical trials to validate its technology as a disease-modifying treatment. Based on more than 20 years of research, InGeneron is dedicated to developing therapies supported by clinical evidence and approved by the FDA.