In the Appendix 4C of October management were quite clear:
"Outcomes from the study are anticipated to be released 4Q CY2023 and will provide pivotal data for an application to the Therapeutics Goods Administration (TGA) for inclusion on the Australian Register of Therapeutic Goods (ARTG). The Company has been progressing its US commercialisation plans including investigations into technology scale up, FDA engagement and commercial preparation activities being to support a Phase 2b randomised controlled study for FDA submission."
TGA approval: ARTGA inclusion
So unless the strategic plans have changed in the last few weeks, this evidence of this phase II trial of ATI vs surgery in 48 patients and the phase II trial of ATI vs Corticosteroid will presumably be sufficient to go to the TGA for inclusion on the ARTG as they quite clearly stated above only 4 weeks ago - the omission of that statement being repeated four weeks later more likely poor communication than changed pathway strategy. The ATI vs Corticosteroid was a smaller population size given drop outs on the control cohort. But the subsequent cross-over treatment of the control cohort with ATI adds a little extra data to the mix. The two existing AT trial results were enough for TGA ARTG application in the release less than a month ago - they also must have done around 1,000 ATI injections in Australia via the SAS without incident now.
FDA: Phase 2b
They've had lengthy discussions with both the TGA and FDA, so I am presuming they are not taking leaps of faith in their regulatory applications and whilst a 200+ phase III trial not normally an unreasonable expectation pre-COVID. No longer. It depends on the reg. application and nature of the treatment and other factors, but the FDA has made serious changes to biologics application processes, with a new dedicated office and whilst a trial involving critical organs and stem cells would might have in the past demanded a trial of that size for FDA approval, tendons would not.
Peter Marks of the FDA (Centre for Biologics Evaluation and Research) has also been very vocal about making trial and regulatory protocols for novel therapies in ailments with unmet needs more flexible. Also the use of confirmatory trials in accelerated approvals. The FDA recognising big Pharma is being tight on R&D in the last 20 years and patients with unmet needs, relying on small companies who often can't afford large regulatory trials to get these products through FDA approval requirements.
So you know I am not talking out of my backside, the FDA recently approved a gene therapy by Sarepta in muscular dystrophy in a trial with just 126 patients!:
So I am afraid I disagree on the long 200+ trial. I suspect OCC is looking for a partner in the US to do a 50-100 similar confirmatory phase 2b patient trial via an ambulatory (out-patient) network/chain in the US (Jeez SurgCenter so should have looked at OCC instead of MSB - such a perfect fit). Suspect its got a few potential candidates lined up given the results to date and lower barriers to approval, probably still very much including J&J,
(20min delay)
