InGeneron Announces Encouraging Results from Emory University’s 480-Patient Phase III Knee Osteoarthritis Trial

The MILES study, published in Nature Medicine, showed clinical effectiveness in patients and “confirms an exceptional safety profile” of InGeneron’s cell therapy.

HOUSTON--(BUSINESS WIRE)--InGeneron, Inc., a clinical stage biotechnology company, announces the publication of a Phase III 480-patient clinical trial sponsored by Emory University (Atlanta, GA, USA). Published in the Journal Nature Medicine on November 2^nd, 2023, the study, entitled “Cell based versus corticosteroid injections for knee pain in osteoarthritis: a randomized phase 3 trial”, investigated the efficacy of InGeneron’s regenerative cell therapy in comparison to two other orthobiologic treatments and the conventional corticosteroid injection (CSI) for knee osteoarthritis (OA). 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. Although the primary findings indicated similar efficacy among all treatments, InGeneron reiterates the sentiment expressed in Emory University’s recent press release (Emory News Center), that additional analyses are needed to determine if certain subgroups of patients might preferentially benefit from one of these treatments more than another.

At InGeneron, we are dedicated to evidence-based medicine and deeply value our partnerships with respected institutions like Emory University. These collaborations reflect our commitment to advancing innovative treatments through rigorous clinical research.

"Additional information is necessary to fully interpret the MILES study's outcomes," says Dr. Christoph Schmitz, Head of the Department of Anatomy II at Ludwig-Maximilians University of Munich (Munich, Germany) and Advisory Medical Director of InGeneron. "Specifically, the publication does not clarify if the reported primary outcome measures, including the Visual Analog Scale (VAS) for Pain and the Knee Injury and Osteoarthritis Outcome Score (KOOS) 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 (e.g., https://pubmed.ncbi.nlm.nih.gov/36728848/). 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, like those provided by InGeneron, patients with severe pain might experience significant pain relief and improvement of joint function 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 we feel more information and further analysis is needed, InGeneron is encouraged by the MILES study’s finding that InGeneron’s cell 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 FDA-approved clinical trials for the treatment of partial-thickness rotator cuff tears, wrist osteoarthritis and facet joint syndrome.

Publication Details

DOI: https://doi.org/10.1038/s41591-023-02632-w
Citation: Mautner, K., Gottschalk, M., Boden, S.D. et al. Cell-based versus corticosteroid injections for knee pain in osteoarthritis: a randomized phase 3 trial. Nat Med (2023). https://doi.org/10.1038/s41591-023-02632-w

About the Transpose^® RT System and Current Clinical Trials

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 being investigated in several FDA-approved clinical trials and is currently available in the U.S. for research use only. More information on InGeneron’s actively enrolling clinical trials can be found at www.clinicaltrials.gov under the identifiers NCT03752827, NCT03513731, and NCT03503305.

About InGeneron

InGeneron is a clinical-stage biotechnology company developing novel, safe, and evidence-based cell therapies. We are setting new therapeutic standards by enabling minimally invasive treatments that unlock the healing potential of each patient’s own regenerative cells processed at the point of care for same-day treatment. We currently focus on helping patients impacted by orthopedic conditions and are conducting several clinical trials to validate our 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.

www.ingeneron.com

InGeneron, Inc.
Eckhard Alt, MD, PhD
Executive Chairman of InGeneron
+1 (713) 440 9900
press@ingeneron.com

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