Simply going from the FDA definition of BTD (does not state a specific number of patients), our TNBC trial protocol / design, I believe there is a good chance of BTD approval. For starters, I don't believe CYDY filed based on only two patients in attempt to challenge the FDA....or in other words I think they have had some discussion about what is acceptable criteria for BTD filing and approval.....I think the FDA knows leronlimab well enough by now to have some respect for it and its potential for many indications, but I don't know how the FDA will decide on this and I have not compared to previous BTD approvals to determine a baseline for a number of patients.....I intentionally have not done so because I don't think there are other drugs out there that would be an apples-to-apples comparison given the extensive proven safety record that is shown such amazing results for an extremely deadly condition which it has not been in testing for until very recently. I would guess most previous BTD approvals are for drugs designed for their indication, limited proven safety profile on a much smaller sample size than 840, and granted simply based on early P2 efficacy / results for a serious condition. Some of these may only temporarily help before resistance builds, toxicity, may only work on 20% of patients, etc. but there isn't a better options currently available.
Maybe I am biased or naive, but with the proven safety of leronlimab, surrogate endpoints of CTCs, reduced tumor sizes proven with MRI and CT scans, and many other conditions that have stabilized, TNBC (cancer in general) being such a deadly disease and all the data thus far aligns with progression free survival (PFS)......plus I think the bar is set pretty low for a safe and effective drug such as leronlimab. If this works in 100% of patients who screen positive for CCR5 on the tumor itself or on the macrophages surrounding the tumor, whether this be 4 patients, 7 patients, or 100 patients these results in cancer are very rare!
I know this has been discussed here before, but IMO if they get BTD approval, Accelerated Approval will also be granted. I don't have other examples to compare, but again I don't think there is a comparable situation to provide precedence to our situation.
But to me, isn't necessarily if/when we get BTD or TNBC/MBC cancer approved, but completing the BLA, getting combo HIV approved and continue showing impressive cancer results. If these happen, it will not matter if we received BTD or cancer is approved, it will be used extensively off-label for cancer/s that are proven or showing promise based on early trial and emergency patient results.
Maybe I am biased or naive, but with the proven safety of leronlimab, surrogate endpoints of CTCs, reduced tumor sizes proven with MRI and CT scans, and many other conditions that have stabilized, TNBC (cancer in general) being such a deadly disease and all the data thus far aligns with progression free survival (PFS)......plus I think the bar is set pretty low for a safe and effective drug such as leronlimab. If this works in 100% of patients who screen positive for CCR5 on the tumor itself or on the macrophages surrounding the tumor, whether this be 4 patients, 7 patients, or 100 patients these results in cancer are very rare!
I know this has been discussed here before, but IMO if they get BTD approval, Accelerated Approval will also be granted. I don't have other examples to compare, but again I don't think there is a comparable situation to provide precedence to our situation.
But to me, isn't necessarily if/when we get BTD or TNBC/MBC cancer approved, but completing the BLA, getting combo HIV approved and continue showing impressive cancer results. If these happen, it will not matter if we received BTD or cancer is approved, it will be used extensively off-label for cancer/s that are proven or showing promise based on early trial and emergency patient results.
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