WALTHAM, Mass. – June 6, 2012 – Regenerative medicine company Histogenics Corporation, announced today publication in the Journal of Bone and Joint Surgery (JBJS) of two year results from a Phase 2 randomized clinical trial of the Company’s lead product candidate, the NeoCart® Autologous Cartilage Tissue Implant (ACTI) for patients with grade III chondral injury to the femur (cartilage damage in the knee). The paper concludes that NeoCart:
• has a comparable safety profile to microfracture surgery,
• significantly improves pain and function within six months of treatment,
• provides significantly greater improvements in a greater proportion of patients than microfracture, and
• is associated with greater clinical efficacy two years after treatment than microfracture.
NeoCart is an autologous bioengineered neocartilage grown outside the body using the patient’s own cells for the repair of full thickness cartilage lesions. A multi-center, randomized Phase 3 study of the product candidate is underway comparing treatment of articular cartilage defects of the knee with NeoCart versus current standard of care, microfracture surgery. In current clinical practice, microfracture surgery, which works by creating tiny fractures in the underlying bone, is widely recommended as a primary treatment for chondral injury to the femur, although outcome measures have been reported to plateau between 12 to 24 months. Prior to the NeoCart study featured in the JBJS, no studies of microfracture alternatives had shown a significant clinical improvement when directly compared to microfracture before and up to two years of treatment.
“There is a clinical need for a primary surgical treatment option for cartilage repair that improves on the historical outcomes of microfracture and the results detailed in our recent analysis and publication strongly suggest that NeoCart may meet this need as a first-line therapeutic alternative to microfracture procedures,” said Dennis Crawford, M.D., Ph.D., Assistant Professor, Orthopedics Oregon Health Science University and the lead author of the paper. “Preliminary findings strongly suggest that autologous cartilage tissue implant using NeoCart significantly improved knee pain and function within six months and provided significantly greater improvements, in a greater proportion of patients than microfracture. This includes, importantly, greater clinical efficacy two years after treatment in contrast to those treated with microfracture surgery.”
“Histogenics is committed to improving the quality of life for active adults and elite athletes by developing innovative solutions upstream to the current standard of less efficacious or more invasive treatment modalities,” said Patrick O’Donnell, President and Chief Executive Officer of Histogenics. “The analysis in JBJS adds to a growing body of evidence that NeoCart has the potential to be a longer-term, effective solution for cartilage injury—which is especially encouraging for young, active adults who are eager to return to their pre-injury activities and want to avoid more invasive, bridge-burning treatments during these prime years of their lives. Based on these continued positive findings, we are hopeful that NeoCart could become a valuable addition to the treatment armamentarium for cartilage damage and look forward to completing our ongoing NeoCart Phase 3 clinical study.”
In the JBJS paper, three, six, twelve, and twenty-four month data were reported with a mean of 26 months for all patients (21 NeoCart; 9 microfracture). Mean age, body mass index, injury acuity and lesion size were similar across both arms. Adverse event rates did not differ between treatment arms. Short Form (36) Health Survey, Knee injury and Osteoarthritis Outcomes Score (KOOS) activity of daily living, KOOS quality of life and International Knee Documentation Committee (IKDC) score improved from baseline (p<0.05) at two years for both treatments. Improvement for NeoCart versus baseline was significant (p<0.05) for all measures at 6, 12 and 24 months. NeoCart treatment improvement was statistically greater (p<0.05) than microfracture for KOOS pain at 6, 12 and 24 months, KOOS symptoms at 6 months, IKDC, KOOS sports and Visual Analog Scale pain at 12 and 24 months, and KOOS quality of life at 24 months. Analysis of covariance at one year indicated that KOOS pain (p=0.016) and IKDC (p=0.028) change from pre-treatment Ievels favored NeoCart. Significantly more NeoCart treated patients (p=0.0125) were therapeutic responders at 6 (43% v. 25%) and 12 (76% v. 22%) months. This trend continued as a greater proportion of NeoCart treated patients (15/19) were therapeutic responders at 24 months than microfracture treated participants (4/9). In the responder analysis, a patient was classified as a responder if they achieved at least a 12-point improvement in the pain score of the KOOS assessment and a 20-point improvement in the IKDC subjective score.
The paper, titled “NeoCart®, an Autologous Cartilage Tissue Implant, Compared to Microfracture for Treatment of Distal Femoral Cartilage Lesions. An FDA Phase II Prospective, Randomized Clinical Trial after Two Years,” was authored by Dennis Crawford, M.D., Ph.D., Assistant Professor, Orthopedics Oregon Health Science University, Thomas DeBerardino, M.D., Associate Professor, Orthopaedic Surgery, New England Musculosketal Institute at University of Connecticut Health Center and Riley Williams, III, M.D., Associate Professor, Orthopaedic Surgery, Weill Medical College of Cornell University, Hospital for Special Surgery.
In addition to the results reported today in JBJS, data were also presented last month at the International Cartilage Repair Society Annual Meeting from previously completed Phase 1 and 2 clinical trials that demonstrated that NeoCart efficacy was sustained throughout a median study period of 48 months and, in the first patients treated with NeoCart, for up to 5 years.
NeoCart® is an autologous bioengineered neocartilage grown outside the body using the patient’s own cells for the regeneration of cartilage lesions. NeoCart recently entered a Phase 3 clinical trial after reporting positive Phase 2 data, in which all primary endpoints were met and a favorable safety profile was demonstrated.
Histogenics is a leading regenerative medicine company that combines cell therapy and tissue engineering technologies to develop highly innovative products for tissue repair and regeneration. In May of 2011, Histogenics acquired Israeli cell-therapy company Prochon BioTech. Histogenics’ flagship products focus on the treatment of active patients suffering from articular cartilage derived pain and immobility. The Company takes an interdisciplinary approach to engineering neocartilage that looks, acts, and lasts like hyaline cartilage. It is developing new treatments for sports injuries and other orthopedic conditions, where demand is growing for long-term alternatives to joint replacement, including lead candidates NeoCart®, an autologous bioengineered neocartilage grown outside the body using the patient’s own cells for the regeneration of cartilage lesions, and VeriCart™, a three-dimensional cartilage matrix designed to stimulate cartilage repair in a simple, one-step procedure. Histogenics has successfully completed Phase 1 and Phase 2 clinical trials in which the NeoCart autologous tissue implant’s effectiveness is compared to that of standard microfracture surgery. Based in Waltham, Massachusetts, the company is privately held. For more information, visit www.histogenics.com.