MEDIPAL HOLDINGS Corporation and JCR Pharmaceuticals Co., Ltd. announced that the Board of Directors of both companies conclude two agreements on JR-446, a blood-brain barrier (BBB) penetrating form of a-N-acetylglucosaminidase that was developed using JCR's proprietary J-Brain Cargo®? technology, for the treatment of Mucopolysaccharidosis Type IIIB (MPS IIIB or Sanfilippo syndrome type B). In October 2022, both companies entered into a partnership for the development of certain drugs for the treatment of ultra-rare diseases.

JR-446 is an additional program to this partnership. Under the ex-Japan licensing agreement, MEDIPAL will acquire an exclusive commercialization right of JR-446 with sublicensing rights, including clinical development, manufacturing, and marketing. As regards to the Japan collaboration agreement, MEDIPAL will support JCR in the clinical development of JR-446 in Japan, including distribution of investigational drugs, disease awareness and conducting clinical trials.

MEDIPAL will receive a certain amount of profit under a profit-sharing scheme based on the sales in Japan. In accordance with both agreements, JCR is scheduled to receive upfront payments. In addition, JCR will receive royalties dependent on the sales outside Japan and milestone payments when relevant milestone events are achieved in the development progress in Japan.

JCR is responsible for sales in Japan and will book sales. JR-446 is a novel drug developed based on the J-Brain Cargo® technology, which has been clinically validated through the approval of IZCARGO® for the treatment of MPS II in Japan in 2021. Animal studies have previously confirmed delivery of JR-446 into the CNS(Central Nervous System) and somatic organs and significant reduction of substrate caused by the deficiency in the enzyme that causative for the onset of MPS IIIB.

As there is no established standard of care for the treatment of MPS IIIB, JR-446 is believed to have the potential to become a transformative treatment of MPS IIIB. MEDIPAL and JCR recognize the urgency to develop treatment for MPS IIIB for which there is no established treatment. It is also noted that there are potentially undiagnosed and misdiagnosed MPS IIIB patients in Japan and overseas due to the nature of the disease being confused with different diseases.