This is progress but also highlights the slow progress the business is making. They would have been disappointed to have had to go back and provide this evidence to the FDA but having got it together is obviously good. Unfortunately it's still a way from phase 3 trial results which is the endgame here. I'm confident from the evidence already released that the drug works but the cashburn is unbelievable and they JUST NEED TO GET THEIR PHASE 3 TRIAL DONE! argh. So frustrated.
ASX RELEASE 19 April 2024
PARADIGM CONFIRMS SUBMISSION TO THE US FDA
TO PROGRESS THE PHASE 3 OA PROGRAM
Paradigm Biopharmaceuticals Ltd (ASX:PAR) (“Paradigm” or “the Company”), a
late-stage drug development company focused on delivering new therapies to address
unmet medical needs, is pleased to announce the submission of key documents to the
US Food and Drug Agency (FDA, the Agency) for review and agreement on the
progression of the phase 3 clinical program in osteoarthritis.
Paradigm has submitted to the US FDA a response to the Type D meeting held on 10
January 2024.
Paradigm’s response sets out the results of five nonclinical studies and data from the
successful phase 2 clinical trial, PARA_OA_008, as well as available clinical data from
600 participants dosed in stage 1 of PARA_OA_002. These additional data were collected
following Paradigm’s Investigational New Drug (IND) submission to the Agency in March
2021 (see ASX release), most of which have not previously been submitted nor reviewed
by the FDA. These data provide a detailed justification for the clinical development for
iPPS with the dosing regimen of 2 mg/kg twice weekly for 6 weeks of initial therapy.
Paradigm also submitted a draft of the phase 3 pivotal clinical trial protocol for agency
review and comment.
The response package to the US FDA has been submitted as directed by the Agency,
through a request for review pathway. The request for review pathway does not have
strict Prescription Drug User Fee Act (PDUFA) Agency response timelines, although
feedback is typically received within three months.
The key items addressed in the Type D response submission to the FDA include:
• The minimal effective dose justification.
• Additional nonclinical studies completed to GLP standards to address previously
noted adrenal findings.
• Draft clinical trial protocol.
• Revised safety monitoring and mitigation plan.
Subject to FDA clearance, Paradigm intends to promptly move forward with subject
enrolment into the phase 3 clinical trial (PARA_OA_012) in 2H CY2024. Clinical trial sites
in Australia and the US are planned to commence preparation activities during this quarter
(Q2 CY2024) to move the phase 3 program forward as quickly and efficiently as possible.
Paradigm Managing Director, Mr Paul Rennie commented: “This is important progress
for Paradigm as we deliver a significant amount of new data to the US FDA for review to
progress to the next stage of the phase 3 OA program. Since Paradigm’s IND submission
in March 2021, we have treated close to 700 participants under a clinical trial setting with
various iPPS doses, which provides strong justification that 2 mg/kg twice weekly is the
lowest effective dose with which to move forward. We look forward to receiving the FDA’s
feedback and remain ready to answer any further questions. We look forward to updating
all our investors in the coming months on the outcome of the agency's review and planned
execution of the phase 3 clinical program. I would like to thank our investors for their
ongoing support as we strive to bring the exciting potential of iPPS to market for those
suffering with osteoarthritis.”
Next Steps
Paradigm will now finalise the TGA provisional approval determination application for
submission. The determination application will include information from a manuscript
detailing the outcomes from the PARA_OA_008 phase 2 clinical trial and a manuscript
providing a comparison of iPPS clinical data with other available treatments for
osteoarthritis. Should the determination application decision be positive, Paradigm will
prepare a full dossier submission for TGA provisional approval marketing authorisation.
Provisional approval offers significant benefits to both patients and manufacturers.
Patients gain access to potential life-saving treatments for life-threatening and seriously
debilitating conditions sooner, particularly for those lacking satisfactory alternatives. For
manufacturers, it provides an opportunity to bring innovative therapies to market faster,
while gathering additional data to support full approval. TGA provisional marketing
approval for iPPS in Australia would expedite the pathway to revenues. The completed
manuscripts are being prepared and will be submitted to separate journals for peer-review
and publication.
-Ends-
About Paradigm Biopharmaceuticals Ltd.
Paradigm Biopharmaceuticals Ltd. (ASX:PAR) is a late-stage drug development
company driven by a purpose to improve patients’ health and quality of life by discovering,
developing, and delivering pharmaceutical therapies. Paradigm’s current focus is
developing injectable (subcutaneous) pentosan polysulfate sodium (iPPS) for the
treatment of diseases where inflammation plays a major pathogenic role, indicating a
need for the anti-inflammatory and tissue regenerative properties of iPPS, such as in
osteoarthritis (phase 3) and mucopolysaccharidosis (phase 2).
Forward Looking Statements
This Company announcement contains forward-looking statements, including statements
regarding anticipated commencement dates or completions dates of nonclinical 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.
To learn more please visit: https://paradigmbiopharma.com
Approved for release by the Paradigm Board of Directors.
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