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https://www.fightingblindness.org/news/inherited-retinal-diseases-cost-the-us-and-canadian-economies-up-to-33-billion-annually-201

This shows the potential rewards for the RP program. I expect at least 5 x the current price (and possibly more) within 12 months

I think the first we will know about it is when they announce that a US investor has bought in. There won't be a public cash raise.

Michael Hunt (CFO) admitted that they would probably need to raise funds before any partnership deals were penned in 2021 but he also stated that the amount required would be moderate and easily achievable.

The price is heavily discounted, partly because of a potential dilution and also partly because of concerns over the effect of COVID on the PH2a extension timetable. I suspect once fundraising is announced, the price will rise not fall.

https://www.choate.com/insights/routes-to-exclusivity-in-cell-based-therapy-development.html

Yes, he is correct. You should read the section on clinical superiority to understand eligibility.

https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/clinical-superiority-findings

https://www.fda.gov/industry/developing-products-rare-diseases-conditions/designating-orphan-product-drugs-and-biological-products

https://en.wikipedia.org/wiki/Orphan_drug

Founded in 1994, Shanghai Fosun Pharmaceutical Group (Fosun Pharma for short), is one of China’s most well-established pharma firms, active in both innovative and generic drugs. Fosun boasts a global footprint, manufacturing and R&D facilities, and a strong presence in medical devices and diagnostics, healthcare services, and pharmaceutical distribution and retail.

Fosun has multibillion dollar revenues and will have no problem funding the CTX stroke trial which is their primary interest, I believe. I expect them to proceed at a much faster pace than RENE have simply because of their resources.

It's true they are also working on the COVID vaccine but that is unlikely to affect the plans for CTX.

Ok - that was actually more informative than MH's presentation. There were a number of points I was really glad to hear:
1. He acknowledged that jCyte had good results in their Ph2b but he expects to beat them with the 2M cell dose.
2. He recognised that COVID could derail some of the trial sites so they are planning on more for contingency so that the trial is not delayed.
3. They expect to revisit partnering for the hRPC program next summer . The timescales make sense given that the primary endpoint in the extension study is at 6 months (see clinical trials.gov). Dosing all the patients by Q1 next year and results are evident after 30 - 60 days.
4. Exosome collaborations are ongoing but they are playing things close to their chest (they are probably under NDA anyway) but at some point news will break.

I was please MH confirmed that they are testing intravitreal as well as subretinal. I think it's key that they compete with jCyte head-to-head.

I got my question into MH about intravitreal injection and he confirmed RENE will now be testing this as well as proceeding with subretinal. It makes perfect sense for them to compete in both types of procedures. RENE's cryopreserved formula offers a real competitive advantage against jCyte's fresh formulation in widespread distribution to ophthalmologist offices.

Shame we didn't get to learn more about the number of patients treated so far in the phase 2a extension. I did ask but he didn't read out the question..

50% efficacy in the 6M cell group seems pretty impressive to me. Clearly, they will be dosing at that level in their Ph3.

The fact that intravitreal injection procedure can be performed by a regular ophthalmologist rather that a highly skilled (and expensive) retinal surgeon in a specialist centre is significant. Rene has stated that sub-retinal injection confers other benefits, new photoreceptors may develop/integrate and longevity of effect may be significantly better, but this has to be balanced with ease of administration and cost. IMHO Rene need to be agnostic to the method of treatment and play to their strengths in lower cost of goods as a result of their Cryopreservation techniques.

At the end of the day, I suspect the decision on strategy will be down to the commercialisation partner who licenses the hRPC cells for the indication.

Let's hope it was RENE management showing their confidence, in which case we'll see an RNS!

Rene made a big point about the Ph2a being an open-label study which allows them to report whenever they wish so I wouldn't take too much notice of those dates. In the Ph2a, they reported results at two months follow-up for one subject, and at 18 days for the other two. They are required to report any "material" events they are aware of to the market.

For the extension study, they announced dosing had started a month ago so It's probable that further patients are being treated by now - hence the update to the clinical trials register.

Based on the Phase 2a results, it seems that efficacy becomes evident a short time after treatment so they should have a good idea of vision improvement early on. The longevity of effect is the other concern in these studies but they now have the previous Ph2a patients results - out to 18 months in one patient in their latest update .

https://clinicaltrials.gov/ct2/show/NCT02464436?cond=Retinitis+Pigmentosa&draw=2&rank=33

Sorry if this question has already been asked, but has anyone done the math on the best case CVR values?

It does seem that way...

When you look at RENE's valuation compared to pharma/biotech on Nasdaq, I do wonder why the Company has not been taken out by an opportunist bid from a US peer, if only for their IP.

I would watch Michael Hunt's latest interview rather than Olav. Hunt was somewhat guarded regarding exosomes development prospects and also stated that RENE did not have the same level of expertise as some of the pure-play, start-ups such as Codiak. However, he did say that RENE would still have a place in the market if the platform was validated. His main emphasis was still RP and the "exciting" prospects of further positive data.

If you look back at the RNS history for the PH2a, an RNS was released regarding efficacy "at two months follow-up for one subject, and at 18 days for the other two". I would expect a similar timeline if the PH2a results are "material" because the company would be obliged to release the news for regulatory reasons. RENE recently confirmed that the first patient has been treated so we could be looking at news mid to late October.

If RENE were to "go it alone", a pivotal PH3 is likely to require a larger number of participants than the Ph2a ext and take around 2 years. Assuming it starts in 2022 and If successful, RENE could submit an NDA (or BLA) late 2024. They would need to recruit a small sales force and incur launch costs in 2025. So, that's 4-5 years from now. I can see them needing at least £50million to get to that point.

As we all know, the RENE share price moves on Newsflow so another interesting quote of interest..."There are also earlier stem cell projects (in immunotherapy and diabetes) which could generate preclinical data in 2020".

In the prelim results presentation on July 20, the CMO stated that they expect to start the ph2a extension study in August/early September. They have two sites ready (Oxford and Boston) but it sounded like they were still scoping out two additional sites (Pheonix has probably been dropped because Pravin Dugel seems to have moved on). Hopefully, Jason Commander and Robert MacLaren will move forward fairly quickly. We must also hope that RENE has secured the two new locations (Barcelona was mentioned as one) and has kicked off recruitment by now. Allowing for the DSMB delay between each treatment, quarter1 next year seems reasonable for completion. They have the PH2a data on duration of effect and if the one-month extension study results are equal or better than the original ph2a then this could be enough for a US institutional investor. It's also possible that they have partners heavily engaged (as suggested by the CMO) who are willing to cut a deal on early data. All supposition though - I'd much rather hear something from RENE's management ream!