Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy

TOKYO -- Businesswire -- Neu REFIX Beta glucan, made in Japan has received both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (US FDA) for treatment of Duchenne muscular Dystrophy (DMD). These designations would help take the progress in pre-clinical and clinical studies conducted in Japan and India to clinical trials in the USA, for approval by FDA.

The Neu-REFIX Beta 1,3-1,6 glucan, is an exo-polysaccharide produced by N-163 strain of Aureobasidium Pullulans, available as an orally consumable, allergen-free, food supplement, that has proven to be safe, with the following potentials in DMD:

· Stand-alone efficacy in reducing skeletal muscle inflammation and fibrosis in mdx mice (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.nature.com%2Farticles%2Fs41598-023-44330-0&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Stand-alone+efficacy+in+reducing+skeletal+muscle+inflammation+and+fibrosis+in+mdx+mice&index=1&md5=18a1f9530aed0700e2a656ecb0fe78f9) · Enhancement of muscle regeneration in mdx mice in a duration of 45-days (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.mdaconference.org%2Fabstract-library%2Fenhanced-muscle-regeneration-in-mdx-duchenne-muscular-dystrophy-mice-proven-by-cd44-myh3-on-consumption-of-a-pullulans-produced-neu-refix-s-glucan%2F&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Enhancement+of+muscle+regeneration+in+mdx+mice+in+a+duration+of+45-days&index=2&md5=4b1385b65baad9f453b8ca5cb983bc28) · Decline in fatigue score in pre-clinical study (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.mdaconference.org%2Fabstract-library%2Fdecline-in-muscle-fatigue-score-of-mdx-mice-after-oral-administration-of-neu-refix-beta-13-16-glucans-in-a-short-duration-study-of-45-days%2F&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Decline+in+fatigue+score+in+pre-clinical+study&index=3&md5=35357aef217d883976211861c7e342e3) · Clinically safe as an adjuvant along with varying regimens of standard-of-care treatment (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.sciencedirect.com%2Fscience%2Farticle%2Fpii%2FS2667242123000556&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Clinically+safe+as+an+adjuvant+along+with+varying+regimens+of+standard-of-care+treatment&index=4&md5=561874c3a26dfd734ce02ea30d6cbc80) · Efficacy of improvement in plasma dystrophin in a clinical study of 45-days duration (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.ncbi.nlm.nih.gov%2Fpmc%2Farticles%2FPMC10482562%2F&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Efficacy+of+improvement+in+plasma+dystrophin+in+a+clinical+study+of+45-days+duration&index=5&md5=f5a58b854b91886327d5330919dcc5e9) · Halting of disease progress in a clinical trial of six months, safely (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.mdaconference.org%2Fabstract-library%2Fefficacy-of-a-safe-s-glucan-in-improving-muscle-function-in-duchenne-muscular-dystrophy-results-of-a-6-month-nonrandomized-open-label-extension-trial%2F&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Halting+of+disease+progress+in+a+clinical+trial+of+six+months%2C+safely&index=6&md5=2b62115b84c5f899b10e485ba993c20f) · Beneficial reconstitution of gut microbiome in patients with DMD (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fdoi.org%2F10.1101%2F2022.12.09.22283273&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Beneficial+reconstitution+of+gut+microbiome+in+patients+with+DMD&index=7&md5=6da8fc16dafdab806d1dc6672a6c168a) · Clinical improvement in patients with LGMD (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.actamyologica.it%2Farticle%2Fview%2F312&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Clinical+improvement+in+patients+with+LGMD&index=8&md5=1d9c824e25e82a3f72b59e18c635053d) · Reduction of myocardial fibrosis in mdx mice (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fdoi.org%2F10.1101%2F2023.09.25.559276&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Reduction+of+myocardial+fibrosis+in+mdx+mice&index=9&md5=cb387f74bad727701506522fd670d314)

The research findings of the journey started in 2009 (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fcommunities.springernature.com%2Fposts%2Fdiscovery-of-a-safe-adjuvant-to-duchenne-muscular-dystrophy-a-journey-of-14-years-of-passion-pursuits-perseverance&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=the+journey+started+in+2009&index=10&md5=a373310a803e62cd7d81f75fd82e2464) have been published in peer-reviewed journals and conferences including the Asian and Oceanian Myology Center- Japan Muscle Society joint conference, AOMC-JMS 2024 (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.youtube.com%2Fwatch%3Fv%3D8WAKMpAePiU&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Asian+and+Oceanian+Myology+Center-+Japan+Muscle+Society+joint+conference%2C+AOMC-JMS+2024&index=11&md5=93f5b3ecfe8dea1f5c62660ea85ba3ad).

DMD is a rare genetic disease affecting 1 in 5000 male births with close to 50000 patients in the USA. The anti-inflammatory potentials of Neu-REFIX documented in the rare disease of DMD have paved ways to expand further research to include auto-immune diseases, rare disease like Multiple Sclerosis (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fdoi.org%2F10.1101%2F2023.09.07.23295172&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Multiple+Sclerosis&index=12&md5=74d24ca80270c34d6422f471bb81f8db) and also common skin diseases like Psoriasis Vulgaris (https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.thehindu.com%2Fnews%2Fcities%2FMadurai%2Fsignificant-breakthrough-achieved-in-treating-symptoms-of-psoriasis-vulgaris%2Farticle68348220.ece&esheet=54124892&newsitemid=20240920153092&lan=en-US&anchor=Psoriasis+Vulgaris&index=13&md5=da0836293271e2162607112d4c3d6c0f) as a disease modifying adjuvant.

Neu-REFIX is a food supplement produced in Japan and available commercially since 2018; A food additive as per Japanese standards. It’s not a drug or remedy to any illness; nor a diagnostic or disease preventing agent. Research outputs are not to be construed as medical advice. The information is propagated to gain relevant support from organizations with synergy to undertake clinical trials and obtain approvals from regulatory agencies. The RPDD and ODD designations are not approvals but are considered as a validation of our approach that have several advantages of market exclusivity, access to grants and research support as well as a fast-track progress.

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