Late-stage drug development company Paradigm Biopharmaceuticals (ASX:PAR) has submitted 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 as well as available clinical data from 600 participants dosed in stage 1. These additional data were collected following Paradigm’s Investigational New Drug (IND) submission to the Agency in March 2021.
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. Clinical trial sites in Australia and the US are planned to commence preparation activities during this quarter (Q2 CY2024) to move the phase 3 programme forward as quickly and efficiently as possible.
Managing Director, Paul Rennie, said 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
“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 programme,” he said.
“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.”
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.
