Paradigm submits key documents to US FDA to advance its pivotal Phase 3 trial of iPPS/Zilosul
Biotech also submits a draft of protocol for the Phase 3 pivotal clinical trial to FDA for review and comment
Pending FDA clearance, PAR aims to kick off enrolment into the Phase 3 clinical trial in the H2 CY24.
Special Report: Paradigm Biopharmaceuticals has submitted key documents to the US Food and Drug Administration (FDA) for review, aiming to advance its pivotal Phase 3 clinical trial of lead drug injectable pentosan polysulfate sodium (iPPS/Zilosul) to treat osteoarthritis (OA).
Often referred to as degenerative joint disease or ‘wear and tear’ arthritis, osteoarthritis (OA) stands out as the most common form of this condition.
However, PAR aims to minimize its impact by improving the functional mobility and preserving the joint structure and reducing pain of affected individuals.
The company is developing injectable pentosan polysulfate sodium (iPPS/Zilosul) for the treatment of OA and Mucopolysaccharidosis, where inflammation significantly contributes to the disease process.
A Phase 2 trial demonstrated one course of subcutaneous iPPS can provide 12-month pain relief and improved function in knee OA sufferers. The same clinical trial also demonstrated improvement in cartilage volume and thickness at six months following treatment compared to placebo.
Furthermore, PAR announced in February its Phase 2 clinical trial, PARA_MPSVI_001, had successfully met its primary endpoint.
The trial was designed to evaluate the use of iPPS compared to placebo in 13 participants with Mucopolysarcharidosis Type VI (MPS VI).
Key documents submitted to US FDA for pivotal Phase 3 trial
Phase I trials are concerned primarily with establishing a new drug’s safety and dose range, while Phase II studies determine the effectiveness of an experimental drug on a particular disease or condition.
The most advanced trials are Phase III, in which massive testing provides a huge amount of data on a drug’s safety and efficacy. Once a Phase III study is completed, a pharmaceutical company can request FDA approval to market the drug.
Paradigm Biopharmaceuticals (ASX:PAR) says the submission of docs to the US FDA to move to Phase III includes a response to the Type D meeting held in January encompassing results from five nonclinical studies, data from the successful phase 2 clinical trial, PARA_OA_008, and clinical data from 600 participants in stage 1 of PARA_OA_002.
PAR says the additional data was collected post the Investigational New Drug (IND) submission to the FDA in March 2021, with most not previously submitted nor reviewed by the agency.
The company says the data provides comprehensive justification for the clinical development of iPPS with a dosing regimen of 2mg/kg twice weekly for six weeks as initial therapy.
Additionally, PAR has submitted a draft of the protocol for the Phase 3 pivotal clinical trial (PARA_OA_012) to the FDA review and comment.
The response package has been submitted through a request for review pathway as directed by the regulatory agency, with anticipated feedback within three months.
Key aspects addressed in the Type D FDA response submission include:
Additional nonclinical studies completed to GLP standards to address previously noted adrenal findings
Draft clinical trial protocol
Revised safety monitoring and mitigation plan
Next steps for PAR
Pending FDA clearance, PAR aims to kick off enrolment into the Phase 3 clinical trial in the H2 CY24.
Preparation activities for clinical trial sites in Australia and the US are scheduled to start in Q2 CY24 to expedite the progress of the phase 3 trial.
PAR will also finalise a Therapeutic Goods Administration (TGA) provisional approval determination application for submission.
PAR says application will include information from a manuscript detailing outcomes from its PARA_OA_008 Phase 2 clinical trial and a manuscript providing comparison of iPPS clinical data with other available treatments for OA.
Should the determination application decision be positive, the biotech will prepare a full dossier submission for TGA provisional approval marketing authorisation.
The company says provisional approval presents notable advantages for both patients and manufacturers. Patients can access potentially life-saving treatments for critical conditions sooner, especially when satisfactory alternatives are lacking.
For manufacturers, it offers a chance to introduce innovative therapies to market more swiftly, while collecting additional data to bolster full approval.
PAR says TGA provisional marketing approval for iPPS in Australia would accelerate the path to revenues.
Completed manuscripts are currently being readied for submission to various journals for peer-review and publication.
Treated close to 700 patients
PAR managing director Paul Rennie says delivering a significant amount of new data to the FDA for review to progress to the next stage of the Phase 3 OA trial is significant.
“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 2mg/kg twice weekly is the lowest effective dose with which to move forward,” he says.
“We look forward to receiving the FDA’s feedback and remain ready to answer any further questions.”
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