Histogenics Announces Top-Line Results From Phase 3 Clinical Trial of NeoCart® in Patients With Knee Cartilage Damage
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? Phase 3 Clinical Trial of NeoCart Did Not Meet Primary Endpoint of a Statistically Significant Improvement in Pain and Function in a Dual Threshold Responder Analysis One Year After Treatment as Compared to Microfracture ?
? NeoCart Demonstrated Statistically Significant and Clinically Meaningful Improvements on Dual Threshold Responder Analysis Six Months After Treatment and Nearly All Pain and Function Measures Compared to Microfracture One and Two Years After Treatment ?
? Data Compared Favorably to Other Products on the Market or in Development Per Guidance from the U. S. Food and Drug Administration ?
? Company to Discuss Plans for Submission of Biologics License Application with U.S. Food and Drug Administration ?
? Company to Host Conference Call and Webcast Today at 8:30 a.m. ET ?
Histogenics Corporation (Histogenics) (Nasdaq: HSGX), a leader in the development of restorative cell therapies that may offer rapid-onset pain relief and restored function, today announced that its Phase 3 clinical trial of NeoCart did not meet the primary endpoint of a statistically significant improvement in pain and function in a dual threshold responder analysis one year after treatment as compared to microfracture. In the modified Intent to Treat (mITT) population (which excludes those patients who were randomized but not treated with NeoCart), 74.2% of the NeoCart patients exhibited clinically meaningful improvements in pain and function compared to 62.0% of microfracture patients at one year (p=0.071). However, in this mITT population, patients treated with NeoCart achieved a statistically significant improvement in pain and function (p=0.018) six months after treatment as compared to patients treated with microfracture. Both NeoCart and microfracture were well tolerated and exhibited strong safety profiles.
“Based on the totality of the data generated in the Phase 3 clinical trial, we continue to believe in NeoCart’s potential as a treatment for knee cartilage damage. When we designed our Phase 3 clinical trial in 2009, we set a very high clinical bar for NeoCart and narrowly missed hitting the trial’s primary endpoint with statistical significance by only two microfracture responders out of the 249 patients that participated in the trial. While the NeoCart treatment group exhibited a response as early as three months after treatment that continued through two years, the microfracture response rate was better than expected, which impacted the statistics. We are encouraged by the results and believe we have a meaningfully differentiated product that, if approved, can compete effectively and provide physicians and patients with a beneficial treatment option that may grow the market,” said Adam Gridley, President and Chief Executive Officer of Histogenics. “We continue to analyze the data and are in the process of scheduling a meeting with the FDA to discuss the results and prepare for a potential submission of a biologics license application for NeoCart. We wish to acknowledge and thank the patients and investigators who participated in the trial and shared their positive experiences with NeoCart,” stated Mr. Gridley.
The NeoCart Phase 3 clinical trial is believed to be the largest and first prospectively designed, randomized clinical trial in North America evaluating the safety and efficacy of a restorative cell therapy to treat knee cartilage damage. It is also believed to be the only trial with a dual threshold responder analysis endpoint. As part of the prospective data analysis, Histogenics collected a variety of patient reported outcome endpoints, including all measures of the Knee Injury and Osteoarthritis Outcomes Score (KOOS) and the International Knee Documentation Committee (IKDC) score, which are validated, patient-centered assessments of pain and function that are commonly used in current clinical trials of cartilage therapies. On all but one of these measures, two of which are being utilized as primary endpoints in ongoing clinical trials by third parties in the U.S. for other therapies, NeoCart demonstrated statistically significant superiority versus microfracture at one and two years.
The Phase 3 clinical trial is the first study prospectively enrolled consistent with current U.S. Food and Drug Administration (FDA) guidance, which provides for the use of microfracture as a comparator treatment in trials to repair knee cartilage damage. The published FDA guidance also specifically calls for a study population that, given the clinical limitations and variable results of microfracture, we believe provides more favorable results than what is typically seen in microfracture in both the literature and a real-world setting.
“We are pleased with the overall performance of NeoCart in this Phase 3 clinical trial and the data confirm the feedback we have received from several of the investigators who participated in the trial. Most importantly, patients treated with NeoCart displayed an early and sustained recovery from pain and return to function that was clinically meaningful. The data from this trial are also consistent with results seen in prior clinical trials of NeoCart as well as the biomechanical data generated as part of our collaboration with Cornell University,” said Lynne Kelley, M.D., Chief Medical Officer of Histogenics. “While we are continuing to analyze the data, we have already seen a number of important results, including a statistically significant improvement of NeoCart compared to microfracture in lesion sizes of greater than 2 cm and patients with higher body mass index. We think that results such as these will be an important part of our planned discussions with the FDA, as well as with clinicians if NeoCart is approved,” continued Dr. Kelley.
There are approximately 1.2 million arthroscopic procedures conducted each year to treat knee cartilage defects in the U.S., with less than half of eligible patients currently electing to receive treatment. Based on the data generated to date, NeoCart may offer many of these patients a safe and effective alternative, subject to FDA approval.
“As a physician who treats patients with knee cartilage damage, I am keenly aware of the limitations of current treatment approaches for this common and underserved condition,” said David C. Flanigan, MD Associate Professor, Department of Orthopedics, Director, Cartilage Restoration Program at The Ohio State University Wexner Medical Center, and a high-enrolling investigator in the Phase 3 clinical trial. “The pain and loss of function associated with uncorrected knee cartilage lesions can significantly limit these patients’ ability to maintain their daily routines and often leads to other more serious comorbidities over time. The rapid recovery for patients who received this cartilage tissue implant compared to those who underwent microfracture indicates that implants, such as NeoCart, may be an attractive alternative for patients seeking a better quality of life and faster return to function,” continued Dr. Flanigan.
The primary endpoint for the Phase 3 clinical trial was a dual-threshold responder analysis measuring the improvement in KOOS pain and IKDC function scores for each patient treated with NeoCart compared to those treated with microfracture one year after the time of treatment. Dual-threshold responders were defined as patients who, relative to their baseline measurements, had at least a 12-point improvement in the KOOS pain sub-score assessment and a 20-point improvement in the IKDC subjective assessment. The trial also evaluated additional pain, quality of life, and function outcomes using all five measures of KOOS subscales, including Sports and Recreation. The change from baseline and the relative change between the NeoCart and microfracture arms was also measured at one year which contrasts with clinical trials of other products, either on the market or in development, that measured these changes at two years. Efficacy and safety will continue to be followed out to three years, and Histogenics expects to further track patients for future planned analyses, including patients from prior clinical trials who received a NeoCart treatment.
Demographics for both study arms were similar and represent a patient population that was intended to ensure that microfracture would respond favorably, including patients with an average age of approximately 39 years old and a Body Mass Index (BMI) of approximately 27. Furthermore, the mean lesion size was 2.1 cm in the NeoCart arm and 1.8 cm in the microfracture arm. There were no other significant differences between the treatment arms.
