Hyogo, Japan - JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; 'JCR') today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).
In a series of oral and poster presentations at the 20th Annual WORLDSymposium in San Diego, Calif., JCR highlighted several programs that rely on J-Brain Cargo, a proprietary technology developed by JCR, to deliver medicines across the blood-brain barrier (BBB).
'Lysosomal storage disorders present a lifelong burden for patients because of the somatic, and most importantly, the progressive neurological signs and symptoms associated with these devastating and life-threatening diseases for which there are inadequate treatment options or no approved therapies available. JCR is dedicated to address the unmet medical needs for this community,' said Shin Ashida, President and CEO of JCR Pharmaceuticals. 'The clinical data presented at the WORLDSymposium demonstrate the safety and efficacy of JR-141 in individuals with MPS II and the potential of our proprietary J-Brain Cargo technology across multiple lysosomal storage disorders. In addition, we're excited to present preclinical data focused on retinal function and bone abnormalities.'
There are three JR-141 presentations, including one presentation focused on the long-term safety and efficacy and behavioral effects of JR-141 (pabinafusp alfa) for mucopolysaccharidosis type II (MPS II, or Hunter syndrome). JR-141 is a recombinant iduronate-2-sulfatase (I2S) enzyme replacement therapy (ERT) that was approved in March 2021 by the Ministry of Health, Labour and Welfare (MHLW) in Japan, where it is marketed as IZCARGO for the treatment of patients with MPS II. The second JR-141 presentation details the global phase III clinical trial study. Finally, the third JR-141 presentation is pre-clinical and highlights the recovery of retinal function in mice with MPS II.
'We are pleased to be able to offer IZCARGO to patients with MPS II in Japan,' said Yoshikatsu Eto, M.D., Ph.D., Institute of Neurological Disorders, Advanced Clinical Research Center, Kanagawa, Japan. 'As we continue gathering new data from the Japanese and Brazilian long-term efficacy study and from the ongoing phase III global JR-141 clinical study, currently in recruitment, we look forward to providing updates as appropriate. These data sets will make it more certain that JR-141 can treat not only the somatic symptoms of MPS II, but also the neurocognitive symptoms of this devastating and life-threatening disease.'
In addition to the three JR-141 presentations, the WORLDSymposium 2024 scientific program featured presentations focusing on: 52-week interim data from the JR-171 phase I/II clinical trial (Hurler syndrome, Hurler-Scheie syndrome or Scheie syndrome); pre-clinical data on an ERT with JR-171 preventing bone deformities in a mouse model of MPS I; JR-441 global clinical trial study design (Sanfilippo syndrome type A).
JR-141 MPS II Datasets
The following poster presentation provides additional evidence and context for the use of JR-141 in the treatment of MPS II: Integrated long-term efficacy and safety data on enzyme replacement therapy with pabinafusp alfa for neuronopathic mucopolysaccharidosis II (MPS-II): updated clinical data from Japan and Brazil (Abstract 81)
Presenter: Yoshikatsu Eto, M.D., Ph.D. (Institute of Neurological Disorders, Advanced Clinical Research Center, Kanagawa, Japan)
This abstract analyzes the long-term efficacy and safety data on pabinafusp alfa for the treatment of individuals with MPS II. This study analyzed 104 patients over 260 weeks. In terms of safety, the data include four pabinafusp alfa-related serious adverse events (consciousness disturbance, hypotension, pyrexia and facial palour) in one individual, but all four were managed and resolved. Infusion-associated reactions were noted to gradually decrease in frequency as the length of exposure to pabinafusp alfa increased. The Kyoto Scale of Psychological Development, Bayley Scale for Infant Development, Kaufman Assessment Battery for Children, and Vineland Adaptive Behavioral Scale were used to evaluate the efficacy of pabinafusp alfa against neurocognitive impairment. A developmental quotient (DQ) of >70 was identified as the best predictor of treatment response in achieving age-equivalent neurodevelopment. Another predictor was age at initiation of treatment, with initiation before 36 months of age associated with the most favorable response in subjects with severe MPS II. Analysis of adaptive behavior showed a trend to overall improvement in most individuals with the attenuated subtype, and some improvement in those with the severe subtype and a DQ of >70, while changes, as expected, were limited in those with a DQ of
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