$HSGX news, Histogenics Announces Agreement With the

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Histogenics Announces Agreement With the Japan Pharmaceuticals and Medical Devices Agency Regarding Japanese Regulatory Pathway for NeoCart(R)
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Histogenics Announces Agreement With the Japan Pharmaceuticals and Medical Devices Agency Regarding Japanese Regulatory Pathway for NeoCart(R)

-- Long history and significant US data package may enable full Marketing and Manufacturing Authorization of NeoCart subject to successful completion of small confirmatory clinical trial in Japan --

-- PMDA Consultations concluded in first quarter of 2017 encompass all parts of potential regulatory submission including: Clinical Trial, Quality/Manufacturing and Non-Clinical Safety --

-- Japanese cartilage repair market estimated to be second largest in the world with established reimbursement, and physicians and patients seeking treatments for cartilage defects that may prevent osteoarthritis in the knee --

WALTHAM, Mass., May 08, 2017 (GLOBE NEWSWIRE) -- Histogenics Corporation (Histogenics) (Nasdaq:HSGX), a regenerative medicine company focused on developing and commercializing products in the musculoskeletal space, today announced that it has successfully completed all formal consultations with the Office of Cellular and Tissue-based Products of the Japan Pharmaceuticals and Medical Devices Agency (the PMDA) regarding the Marketing and Manufacturing Authorization pathway for Histogenics' NeoCart (autologous cell therapy designed to treat cartilage defects in the knee) in the Japanese market. Histogenics is currently nearing the completion of enrollment in a 245 patient Phase 3 clinical trial of NeoCart that is being conducted under a Special Protocol Assessment (the SPA) with the U.S. Food and Drug Administration (the FDA).

"We are very pleased with the formal feedback we received from the PMDA regarding the relatively expeditious approval pathway for NeoCart in Japan, and appreciate the thoughtful, rapid and collaborative approach provided by Japan regulators," stated Adam Gridley, President and Chief Executive Officer of Histogenics. "These successful consultations and meetings are evidence of our execution of a broadening global regulatory strategy launched only seven months ago where we initiated efforts to leverage the robust U.S. clinical, preclinical and cGMP data for NeoCart into international markets. We believe the clarity on this efficient regulatory pathway for full Marketing Authorization will be important to our efforts to seek a development and commercialization partner for NeoCart in Japan and other markets in Asia."

Various aspects of the Japanese regenerative medicine laws were updated in 2014 to potentially expedite the clinical development and commercialization pathways for innovative, qualified regenerative cell-based medicines that have demonstrated safety and probable efficacy. Shortly after reacquiring Japanese rights for NeoCart from Purpose Co. in May 2016, Histogenics initiated preparatory pre-consultations (Jizen-Mendan) with the PMDA for the three discreet modules of the regulatory process: Clinical Trial, Non-clinical Safety and Quality/Manufacturing.

During the first quarter of 2017, Histogenics held a series of consultations with the PMDA, including face-to-face (Taimen-Jogen) meetings. In these consultations, the PMDA agreed that Histogenics' ongoing Phase 3 clinical trial with its one-year primary endpoint could be appropriate and provide sufficient evidence of safety and effectiveness for the full Marketing and Manufacturing Authorization in Japan. Furthermore, the PMDA agreed that a 30-patient, one-year confirmatory clinical trial in Japanese patients, comparing NeoCart to microfracture, would be sufficient for applying for full Marketing and Manufacturing Authorization in Japan. This Japanese study will utilize the same protocol as the ongoing Phase 3 clinical trial of NeoCart. Histogenics also confirmed that NeoCart would be regulated as a Regenerative Medical Product, as covered by the recently enacted laws in Japan, and that Histogenics' current good manufacturing process (cGMP) in the U.S. could be utilized to manufacture and supply the confirmatory clinical study. These consultations with the PMDA were attended by representatives of Histogenics, including co-founder Dr. Shuichi Mizuno, regulatory and regenerative medicine experts, and two esteemed orthopedic surgeons from Japan, Dr. Akihiro Tsuchiya and Dr. Takumi Nakagawa, both of whom have trained on NeoCart procedures with U.S. surgeons.

In addition to the confirmatory clinical trial, Histogenics agreed with the PMDA to conduct additional minor, non-clinical safety studies, including a short-term (three-month) general toxicity study and in vitro tumorigenicity studies to reflect current standards and to augment the significant amount of pre-clinical data previously generated by Histogenics. These additional study protocols will be reviewed with the PMDA and will be conducted prior to the Marketing and Manufacturing Authorization. With regards to Histogenics' Quality/Manufacturing applications, the PMDA and Histogenics agreed to several updates for certain procedures and testing methodology, all of which are planned as part of Histogenics' continuous improvement activities for the biologics license application (BLA) submission planned with the FDA. Histogenics believes that the PMDA's conclusions are based on the strength of the NeoCart clinical and non-clinical data package and the long 15+ year cGMP manufacturing history of NeoCart.

"We are thankful for the leadership and partnership with the PMDA," commented Dr. Shuichi Mizuno, Ph.D., Co-founder of Histogenics, and Assistant Professor, Orthopedic Surgery, Brigham and Women's Hospital, and Harvard Medical School. "We have been working collaboratively for nearly 20 years to provide substantial supportive basic science and non-clinical data as well as novel cell culture technology to guide the development of this important innovative therapy for cartilage defects."

In conjunction with the launch of its commercial partnering efforts, Histogenics commissioned through Locust Walk's Japan team a quantitative market research study with approximately 80 orthopedic surgeons including a mix of hospital physicians and general practitioners in Japan regarding the market opportunity for NeoCart for knee cartilage defects in the Japanese market. The findings paralleled what is observed from surgeons and patients in the United States -- a market with a high unmet need characterized by long recoveries and variable clinical outcomes from current therapies, leaving many patients choosing to avoid having unsatisfactory treatments. These data may reflect the opportunity for innovative regenerative medicine products such as NeoCart to grow the market substantially by providing satisfactory outcomes and better quality of life, while avoiding progression to osteoarthritis (OA) and unnecessary surgeries.

The findings from the Japan survey include:

-- Cartilage defects account for approximately 40% of total knee trauma

cases causing pain and loss of function in the knee (with or without

other injuries).

-- Approximately 60% of patients with knee cartilage defects are either not

treated at all, or treated only with conservative therapies (such as

debridement) to temporarily treat pain.

-- Approximately 60-70% of patients with knee cartilage defects that are

left untreated will likely progress to OA, and 15-20% of their patients

currently suffering from knee OA have such OA likely due to cartilage

defects.

-- More than 85% of orthopedic surgeons are not satisfied with the currently

available treatment options for pain and loss of function due to knee

cartilage defects.

-- Approximately 80% of the surgeons consider early improvement in pain and

function to be important and over half of the surgeons feel they would

prescribe NeoCart based on the data already published.

"As is the case in the United States, we believe Japanese surgeons and patients are seeking new regenerative medicine options to repair cartilage defects that may offer both a more rapid recovery in terms of pain and function, as well as a more durable response over time with fewer repeat surgeries, which improves overall quality of life," commented Dr. Tsuchiya, Vice President, Funabashi Orthopaedic Hospital, Chiba, Japan, and Chairman, Tokyo Women's Medical College, Tokyo, Japan, and Dr. Nakagawa, Professor of Department of Orthopaedic Surgery, School of Medicine, Teikyo University, Tokyo, Japan. "We have followed the development of NeoCart for many years and have trained with surgeons in the U.S., and along with our medical colleagues, look forward to potentially introducing this novel non-NSAID and non-opioid cell-based therapy product to our patients in Japan that currently do not have good options to treat the pain and loss of function due to knee cartilage defects."

Histogenics estimates the Japanese market represents a significant opportunity, with an estimated 200,000 procedures annually in Japan for patients suffering from pain associated with cartilage defects in the knee and limited options to treat the defect or related pain. If left untreated, cartilage defects may result in OA and ultimately, total knee replacements as patients age, with a substantial economic impact on patients and insurance companies. Market forecasts predict that the number of OA patients in Japan aged 40 and older amounts to more than 25 million and is expected to accelerate with the aging population. Once a development and commercial partner is identified, Histogenics intends to file with such partner the Clinical Trial Notification (CTN) to the PMDA to begin the proposed clinical trial.

About Histogenics Corporation

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May 08, 2017 06:30 ET (10:30 GMT)