Japanese Development and Sales License for NeoCart

「OrthoCellix will continue development in the United States, but preparations for the U.S. Phase III trial have been delayed because the merger agreement was terminated, making it difficult to begin the U.S. clinical trial in 2025. The Company had planned to determine its domestic development policy during the current fiscal year, but that decision has also been delayed because preparations for the clinical trial were delayed following changes to the U.S. development structure.」

「The Company held a preliminary meeting with the PMDA on the domestic trial design, using as a reference the additional U.S. Phase III trial protocol agreed between Ocugen and the FDA, and is currently preparing briefing materials for an in-person consultation. The Company plans to determine its development policy for the autologous cell-cultured cartilage product MDNT-01 in Japan during the current fiscal year, taking into account the results of discussions with the PMDA on the domestic trial design and the status of Ocugen's establishment of a manufacturing system for the investigational product.」

「The Company is currently preparing a domestic trial design with reference to the Phase III trial protocol agreed between Ocugen and the U.S. FDA and has begun discussions with the PMDA on the domestic trial design. The Company plans to determine its domestic development policy and other matters in the fiscal year ending September 2024, based on the results of discussions with the PMDA and Ocugen's preparations.」

「The Company is currently preparing a domestic trial protocol with reference to the Phase III trial protocol agreed between Ocugen and the FDA, and plans to determine its domestic development policy and other matters after consulting with the PMDA.」

「However, because finalization of the Phase III trial protocol required for Ocugen's marketing application was delayed, the Company was unable to determine its development policy and other matters for the autologous cell-cultured cartilage product MDNT-01 in Japan during the fiscal year.」

「Ocugen, which currently holds the NeoCart® assets (located in Malvern, Pennsylvania, USA), announced on 16 December 2022 that it had agreed with the FDA on the Phase III trial design required for a marketing application, with the aim of resuming development in the United States. After analyzing and reviewing the details of the Phase III trial design agreed with the FDA, the Company plans to determine its development policy and other matters for the autologous cell-cultured cartilage product MDNT-01 in Japan.」

「Regarding development of the autologous cell-cultured cartilage product MDNT-01 (U.S. product name NeoCart®), Ocugen, which currently holds the NeoCart® assets (located in Malvern, Pennsylvania, USA), continues discussions with the FDA on the Phase III trial protocol required for a marketing application, with the aim of resuming development in the United States, but no agreement was reached during the fiscal year. Ocugen announced that NeoCart® had received RMAT (Regenerative Medicine Advanced Therapy) designation from the U.S. FDA for treatment to repair adult knee cartilage. The Company plans to decide its development policy for MDNT-01 in Japan after the U.S. development policy has been determined.」

「After completion of the asset transfer, the Company will discuss the development of the autologous cell-cultured cartilage product MDNT01 in Japan with Medavate.」

「With respect to development of the autologous cell-cultured cartilage product MDNT01, completion of the asset transfer under the asset purchase agreement for the autologous cell-cultured cartilage product NeoCart® entered into between Histogenics Corporation and Medavate in the previous fiscal year means that the license agreement between the Company and Histogenics Corporation for NeoCart® will also be transferred to Medavate. Accordingly, after completion of the asset transfer, the Company will discuss the development of MDNT01 in Japan with Medavate.」

「The Company announced that it entered into a merger agreement with Ocugen, which is developing innovative clinical-stage biopharmaceuticals in the United States, in April 2019 and merged with Ocugen at the end of September 2019. In May of the same year, Histogenics entered into an asset purchase agreement with Medavate for the autologous cell-cultured cartilage product NeoCart®. As the license agreement between the Company and Histogenics for NeoCart® will also be assigned to Medavate, the Company will discuss the development of the autologous cell-cultured cartilage product MDNT01 in Japan with Medavate after completion of the asset transfer.」

「In May of the same year, Histogenics entered into an asset purchase agreement with Medavate (Fort Collins, Colorado, USA) for the autologous cell-cultured cartilage product NeoCart®. As the license agreement between the Company and Histogenics for NeoCart® will also be assigned to Medavate, the Company will discuss the development of the autologous cell-cultured cartilage product MDNT01 in Japan with Medavate, which is pursuing marketing approval for NeoCart®.」

「In response, the Company proceeded with its review of the development policy for the autologous cell-cultured cartilage product MDNT01 in Japan.」

「The Company announced that it had received a response from the FDA stating that additional clinical trials were required for the BLA. In response, Histogenics announced in December 2018 that it would temporarily suspend development in the United States of the autologous cell-cultured cartilage product "NeoCart®." Following the results of Histogenics' discussions with the FDA, the Company plans to discuss future action with Histogenics and promptly finalize its development policy for the autologous cell-cultured cartilage product "MDNT01" in Japan.」

「On 21 December 2017, the Company entered into a license agreement with Histogenics Corporation (located in Waltham, Massachusetts, USA, hereinafter "Histogenics") for the development and commercialization in Japan of the autologous cultured cartilage product "regenerative medicine product MDNT01."」

「Going forward, under this agreement, the Company will begin clinical trials using the technology for knee joint cartilage injuries and aims to obtain manufacturing and marketing approval as a regenerative medicine product in 2021.」

「On 21 December 2017, the Company entered into a license agreement with Histogenics for the development and commercialization in Japan of the autologous cultured cartilage product NeoCart. Under the agreement, the Company aims to begin a clinical trial for knee cartilage injuries and obtain marketing approval as a regenerative medicine product in 2021.」

「For NeoCart, an autologous cultured cartilage product undergoing a Phase III clinical trial in the United States by Histogenics Corporation, we plan to receive quality, preclinical and clinical trial data and investigational products from Histogenics, use them to initiate a Phase III clinical trial in Japan for knee cartilage injuries, and obtain marketing approval as a regenerative medical product.」