I have had discussions with the CEO Rohan Hocking and he answered some pertinent questions that HC members would be interested in. He has also given me permission to share them with you here. “You are welcome to share the content of the email with whomever you like - the more people who are interested and understand what is happening, the better”, he said. My heartfelt thanks to Rohan.
How would PYC select patient cohorts to maximise efficacy in Clinical Trial ?Thanks for your questions on the RP11 program - these touch on some very relevant points for us right now as we consider how to maximise the prospects of gaining insight on the efficacy of VP-001 in the initial clinical trials bearing in mind that these are primarily safety studies.
We are currently working through the question of maximising the insights on efficacy with clinicians from around the world who have a special interest in RP11. The first consideration here relates to patient stratification - there are multiple different 'stages' of RP progression (you can think of these as 'early', 'middle', 'late' in the disease course) and the impact on patient's vision is different for each stage. This then brings in the related question of which endpoints are most important for which subset of patients and whether we try to bias towards a particular subset of the patient population in order to maximise our prospects of seeing the effect of VP-001. It is quite a complex area and our thinking here is still evolving as we go deeper on this issue with the subject matter experts.
The upside of the slow disease progression, however, is that if we do identify a favourable risk/benefit ratio in VP-001, the conversion from disease prevalence to addressable patient population is likely to be very high. This touches on your question regarding patients with little visual function left at the time of starting treatment - because vision loss follows an 'S-curve', even those patients with very little visual function left at the start of treatment may benefit from VP-001 because they place a very high value on the independence associated with the last 5-10% of visual function.
Could VP-001 offer RP-11 patients disease reversal or just stopping its progression ?On your question on disease reversal versus stopping disease progression, the primary objective of VP-001 is to stop further progression of the RP11 disease course. Having said that, there is a theory that recovery of non-functional but still viable cells at the outset of treatment may confer an improvement of visual function in the initial stages of therapy (this seems to have been observed in the Luxturna clinical trial in RPE65 patients) and so we could hope that this phenomenon also applies to RP11.
Would PYC consider applying for the Australian Government's new "Clinical Trials Activity initiative" ? Hence eliminating the dilution caused by capital raising, or huge reduction in future revenues through licensing deals.On the funding side, we have been applying for non-dilutive funding and will continue to do so. Frustratingly, one of the critical elements of a lot of grant-funding criteria is that the work would not be funded absent the grant and that is not the case for PYC. This has been a consistent piece of feedback on the grants that we have been applying for recently. Somewhat perversely, the fact that the trials will occur irrespective of the outcome of the grant is actually counting against us.
This is an ongoing discussion and more insights from Rohan to come
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