23 Nov

Preliminary Results from Phase 2 MPS I Study

ASX RELEASE 2 3 rd November 20 2 1 Positive interim data from p hase 2 rare disease trial presented at i nternational medical congress . KEY HIGHLIGHTS • Preliminary MPS - I p hase 2 data presented at the 14th International Congress of Inborn Errors of Metabolism in Sydney . • PPS was well tolerated with no serious adverse events reported over a 24 - week period. • Meaningful improvements in pain, function, and activities of daily living and an overall improvement in quality of life was observed in all patients. • Administration of PPS resulted in improvements in walk tests, range of motion and other standa rd tests of activities important to daily function of the patients . • Changes in the profile of biomarkers suggest PPS has the potential to modulate the inflammatory and joint degenerating biomarkers associated with arthralgia in MPS I patients. Paradigm Biopharmaceuticals Ltd (ASX: PAR) (“ Paradigm ” or “the Company”), a clinical stage biopharmaceutical company focus ed on repurposing existing molecules for new indications with unmet clinical needs, is pleased to report preliminary data on the p hase 2 pilot study of pentosan polysulfate sodium (PPS) for the treatment of mucopolysaccharidosis type I (MPS - I ) that will be presented at the 14th International Congress of Inborn Errors of Metabolism in Sydney (ICIEM 2021) . During the conference a poster detailing the promising da ta will be presented along with a discussion with the Head of the Metabolic Unit at the Adelaide Women and Children’s Hospital, Dr Drago Bratkovic, and Paradigm Global Head of Safety and MPS, Dr Michael Imperiale . MPS - I is a rare disease caused by reduced levels, or the complete lack of, an enzyme responsible for the catabolism (break down) of glycosaminoglycans (GAG) resulting in the progressive accumulation of GAG in the tissues. Th e disorder causes problems with neurological, skeletal and cardiovascular developme nt . There is no cure and c hildren born w ith the most severe form of MPS - I d o not typically survive beyond 10 years of age , without treatment . Current standard treatments includ e bone marrow transplant and enzyme replacement therapy to address the underlying cause of the disease . T he study at the Adelaide Women’s and Children’s Hospital, South Australia has enrolled three patients who are over halfway through the 48 - week treatment regime . The encouraging data from these patients was presented at ICIEM 2021 by Dr Drago Bratkovic, H ea d of the M etabolic C linic at the h ospital . The poster provides evidence PPS could help address the unmet medical needs of MPS - I patients and supported further studies. Summary h ighlights of the poster presentation included: • PPS may address the unmet medical needs for patients who continue to experience pain and symptoms that affect function following treatment with the best current standard of care. • PPS was well tolerated with no serious adverse events reported over a 24 - week period. • Meaningful improvements in pain, function, and activities of daily living and an overall improvement in quality of life was observed in all patients. • Administration of PPS resulted in improvements in 2 and 6 - minute walk tests, range of motion and other standa rd tests of activities important to daily function of the patients . • Pharmacokinetic results demonstrated consistency in serum concentrations that were dose dependent . • Changes in the profile of biomarkers suggest PPS has the potential to modulate the inflammatory and joint degenerating biomarkers associated with arthralgia in MPS - I patients. Paradigm inter im Chief Executive Officer , Dr Donna Ske rrett said , “ We are very encouraged by the overall preli minary study data which supports our clinical development strategy for PPS as a viable treatment for children with residual musculoskeletal symptoms despite standard of care therapy in this difficult to treat disease .” A p hase 2 open - label study in up to 10 patients with MPS - I was initiated in Se ptember 2020. The primary aim of the study is to evaluate safety and tolerability of PPS over a n initial 48 - week period , with a 6 - month treatment extension available, in patients treated with the current standard of care . Secondary and exploratory objectives include examining the effects of PPS on pain, function, and quality of life, pharmacokinetics, biomarkers, and inflammatory processes. The open - label study remai ns ongoing with additio nal patient recruitment exp ected in CY22. Paradigm is currently exploring strategic partnerships to progress current and future clinical studies to further evaluate PPS as a treatment to address the critical unmet need of ongoing musculoskeletal symptoms in this very rare patient population. Registered attende es can view the poster presentation via the ICIEM ( https://www.iciem2021.com.au/ ) , and after conclusion of the conference (23 rd November) it will be available on the Paradigm website . About injectable PPS Pentosan polysulfate sodium (PPS) is a medication that has been used in humans for over 60 years. Injectable PPS has previously been approved in European markets, where it is registered as an antithrombotic agent. In Australia, injectable PPS for human use is not currently available for sale. Injectable PPS is available via a Paradigm sponsored clinical trial or under the TGA Special Ac cess Scheme to physicians for individual patients who satisfy strict criteria and is subject to approval from the TGA. Elmiron (the oral formulation utilised for interstitial cystitis) is the only PPS product approved in the US . A subcutaneous injectable f orm ulation of PPS is currently being evaluated by Paradigm for the treatment of osteoarthritis and other inflammatory diseases in the US and other major global markets. About Paradigm Biopharmaceuticals Paradigm Biopharmaceuticals LTD (ASX: PAR) is a late - stage drug development company with the mission to develop and commercialise pentosan polysulfate sodium for the treatment of pain associated with musculoskeletal disorders driven by injury, inflammation, ageing, degenerative disease, infection or genetic predisposition. Paradigm is also investigating p roof - of - concept for the use of PPS in respiratory and heart failure indications. Forward Looking Statements This Company announcement contains forward - looking statements, including statements regarding anticipated commencement dates or completions dat es of preclinical or clinical trials, regulatory developments and regulatory approval. These forward - looking statements are not guarantees or predictions of future performance, and involve known and unknown risks, uncertainties and other factors, many of which are beyond our control, and which may cause actual results to differ materially from those expressed in the statements contained in this presentation. Readers are cautioned not to put undue reliance on forward - looking statements. Authorised for release by the Paradigm Board of Directors. Zilosul® is a registered Trademark of Paradigm Biopharmaceuticals Ltd (ASX: PAR). To learn more please visit: www.paradigmbiopharma.com FOR FURTHER INFORMATION PLEASE CONTACT: Simon White Director of Investor Relations Tel: +61 404 216 467 Paradigm Biopharmaceuticals Ltd ABN: 94 169 346 963 Level 15, 500 Collins St, Melbourne, VIC, 3000, AUSTRALIA Email: investorrelations@paradigmbiopharma.com
Information on this Website is provided for general information purposes only and is not a substitute for professional advice. ASX Information (including company announcements and prices) is delayed by at least 20 minutes. JSE Information (including company announcements and prices) is delayed by at least 15 minutes. Reliance on the information you access on or from this Website is solely at your own risk. We make no representation or warranty in relation to the future performance of the companies that appear on this Website. Investment in securities involves risk and you should obtain independent professional legal, financial, investment or company advice before acting on any of the information you access on this Website. Using, browsing or otherwise accessing this Website is subject to our Terms and Conditions and our Privacy Policy.

© 2021 Listcorp. ABN 60 166 140 307

Never miss news from Paradigm Biopharmaceuticals Limited (ASX:PAR) when you join Listcorp.