Histogenics Completes Enrollment for Phase 3 Clinical Trial of NeoCart

Histogenics Corporation, a regenerative medicine company focused on developing and commercializing products in the musculoskeletal space, has completed patient enrollment in its NeoCart Phase 3 clinical trial. Histogenics expects to report top-line one-year superiority data in the third quarter of 2018, followed by a potential Biologics License Application (BLA) filing.
 
“We are proud to have completed enrollment in the largest prospectively designed, randomized clinical trial evaluating the safety and efficacy of a cellular therapy to treat knee cartilage defects,” said Gloria Matthews, chief medical officer of Histogenics. “There is a substantial need for novel treatment options for knee cartilage damage, such as NeoCart, that have the potential to provide effective pain relief and restore function.”
 
The randomized Phase 3 clinical trial is designed to evaluate the safety and efficacy of NeoCart in 245 patients compared to microfracture, the current standard of care for the treatment of articular cartilage defects. The primary endpoint of the trial is a dual-threshold responder analysis measuring the improvement in the pain and function of each patient treated with NeoCart compared to those treated with microfracture one year after treatment. The Phase 3 clinical trial design and primary endpoint is based on Histogenics’ 30-patient Phase 2 clinical trial that demonstrated highly statistically significant superiority results of NeoCart compared to microfracture at one year. 
 
“Our recent market research confirmed that this market is largely underserved due to the lack of rapid, yet durable therapies that treat the underlying problem with one-third of surgeons unsatisfied with the current treatment options. As a result, two-thirds or more of the patients who suffer from knee cartilage injuries remain untreated, due to the variable outcomes and lengthy rehabilitation associated with existing treatments, often leading to debilitating osteoarthritis. Specifically, patients with defects that are smaller than four centimeters in size, which we believe represent the large majority of the market, are less likely to receive surgical treatment in part due to the low satisfaction with and poor outcomes of existing treatments. Against this background, we believe NeoCart could expand the market by meeting the needs of both patients and surgeons with a product that can potentially deliver rapid pain relief and superior one year clinical outcomes utilizing a quick and simple surgical procedure,” stated Adam Gridley, president and CEO of Histogenics.  “We are now focused on delivering our one-year superiority data by the end of the third quarter of 2018, submitting our BLA to the FDA shortly thereafter and, if approved, launching NeoCart in the second half of 2019.”
 
Histogenics recently conducted market research surveys in Japan and the United States to better characterize the target market for NeoCart based on the expected features and benefits of NeoCart, if approved following the Phase 3 clinical trial. Specifically, there were two market research studies recently performed to further define the United States market opportunity for knee cartilage repair.  One study was conducted by a third party and the second by Histogenics. In total, almost 100 orthopedic and sports medicine surgeons were interviewed. The findings from both studies were consistent and are summarized below:

• Sixty percent to 80 percent of patients with symptomatic knee cartilage defects are not treated at all, with a large gap between diagnosed incidence and actual treatment. Of those patients that are treated, most receive debridement or microfracture.
• The average surgeon sees approximately 75-100 patients per month with cartilage damage, and performs 15-20 surgical procedures per month to treat patients with knee cartilage defects.
• Approximately one-third of surgeons are not satisfied with the currently available treatment options for pain and loss of function due to knee cartilage defects.
• Less than one-quarter of surgeons are satisfied with the currently available treatment options for pain and loss of function due to knee cartilage defects.
• The most important factors for surgeons in choosing a course of treatment for patients are:  longevity/clinical outcomes, rapid patient recovery, and a quick/easy procedure.
• Almost 90 percent of the physicians would be extremely likely or likely to use NeoCart, if approved, based on the target product profile, including the data already published by Histogenics.

 
NeoCart is a cartilage-like, tissue engineered implant created from a patient’s own cartilage cells. The patient’s cells are multiplied in Histogenics’ laboratory and then infused into a proprietary scaffold to allow them to organize and function like cartilage cells. Before NeoCart is shipped to the surgeon for implantation, the cell and scaffold construct undergoes a bioengineering process that is designed to mimic a joint so that the implant, upon placement in the knee with a proprietary bioadhesive, is primed to begin functioning like healthy cartilage. NeoCart is currently in a Phase 3 clinical trial that is designed to evaluate the safety and efficacy of NeoCart as a first-line therapy for full thickness knee cartilage defects in skeletally mature adults ages 18 to 59 and to evaluate the superiority of NeoCart against the current standard of care, microfracture, at one year. NeoCart is not approved for sale in any jurisdiction.  
 
Histogenics is developing and commercializing tissue therapies that may offer more rapid and durable recoveries for patients with pain and loss of function due to musculoskeletal conditions. Histogenics’ regenerative medicine platform combines expertise in cell processing, scaffolding, tissue engineering and bioadhesives to create tissue ex-vivo. Histogenics’ first investigational product candidate, NeoCart, is designed to treat cartilage defects in the knee and is currently in Phase 3 clinical development.  NeoCart is designed to exhibit characteristics of articular, hyaline cartilage prior to and upon implantation into the knee and therefore does not rely on the body to make new cartilage. As a result, NeoCart is the only product in development or on the market with a one-year primary superiority endpoint as compared to the standard of care. There are more than 500,000 or more knee cartilage procedures in the United States each year, with many healthy active adults avoiding treatment as they seek other alternatives. Left untreated, even a small cartilage defect can expand in size and progress to debilitating osteoarthritis, ultimately necessitating a joint replacement procedure. Osteoarthritis is more common in adults over the age of 50, but the condition and precursors of the condition can be observed much earlier, and cartilage damage is believed to be one of the leading contributors of this disease. The company is based in Waltham, Mass.