The latest Investing Matters Podcast episode featuring Jeremy Skillington, CEO of Poolbeg Pharma has just been released. Listen here.
"The Company submitted its second Orphan Drug Designation request to the FDA for treating pediatric brain tumors with GaM. "
Orphan drug developers may be able to apply to the FDA for Rare Pediatric Disease Priority Review Vouchers. These are granted on approval and can then be sold to big pharma. The vouchers typically change hands for around $100million...
https://www.londonstockexchange.com/news-article/IQAI/orphan-drug-designation-status/15855721
It's worth noting that the trial description on the MCW sites states that "The purpose of this Phase I study is to determine the dose of GaM that can be tolerated by patients when taken by mouth (orally) and to see whether gallium maltolate can shrink their tumors." So, they are undoubtedly looking for an efficacy signal (tumor shrinkage) along with safety.
Sadly, this is a very aggressive form of cancer - I believe median survival is around 14 months from detection (including surgery and chemo).
I also see that MCW has tested a combination therapy of GM and Metformin. The results were very encouraging so this might be an option if GM merely slows tumor growth.
https://www.froedtert.com/clinical-trials/cancer/phase-1-clinical-trial-oral-gallium-maltolate-treatment-relapsed-and
RP is a rare disease - I believe the stats are something like 1 in every 4000 people in the US. The FDA granting orphan status entitles RENE to tax breaks and a shortened regulatory path - for example, FDA may accept a single phase 3 instead of two as is normally required.
The value will come when Rene partner which is likely to happen middle of next year if things go to plan. The CMO stated that partners were enthusiastic about results so far but they want more objective measurements of efficacy. That's why they will be using microperimetry testing to precisely measure changes to the retina following treatment. This was used by professor MacLaren as a trial endpoint for Nightstar's gene therapy tteatment for x-linked RP. You can find a youtube vdeo of him talking about how he formed Nightstar and how he mamaged to convince the regulator to accept what were at the time novel endpoints to measure efficacy. It's worth remembering that Nightstar got bought out by Biogen for $800 million. He's now one of the investigators in Rene's RP ttial. His backing gives massive credibility to the whole program.
Based on the clinical trials.gov update, the original two sites are recruiting but Oxford has yet to start. This may.be becauae it"s an NHS hospital or maybe the professor is just a very busy man!
When you look at the RP progress, potential news of exosome colaborations, possible CTX partnering plus Fosun progress, there seems ample scope for an unexpected RNS in the lead up to xmas. This will sutely catch the market out and likely lead to a significant jump in the share price.
It's worth remembering that Michael Hunt has said they will need.to raise money at some point. AIM companies tend to do this on the back of good news that has inflated their share price. I'd suggest Rene is likely to do the same.
Awesome. Thanks for responding... Let's hope they all come in.
Rick was given 7 minutes. so it was all rather compressed. There was detail on the ph2a results along with an explanation of adverse events (which was reassuring). Ph2b sites and investigators were named but no details of progress to date. We do know that one operation was performed a month ago. If all went well, the DSMB should authorize the next tranche.
Rick Beckman is presenting on the 30th. This could be more informatuve than the normal CFO pitch..
https://www.ophthalmology-futures.com/forums/
As long as they pull in a deal for RP or exosomes, it might be preferable if they didn't partner the stroke program in a firesale. The whole program would become much more valuable if they were able to fund the remainder of the PH2b and results were positive. However, they probably made the right decision in suspending gven COVID and the cash burn. Betting the house on stroke is risky given the numerous failures in this indication that include the SanBio cell therapy phase 2 failure last year. I would prefer them to wait until they are in a positiin where a single trial failure doesn't take the company out.
I am sure the CEO and CFO are feeling the heat regarding further funding. I'm sure major shareholders wiould not be happy with a bad deal and may look to replace them.
Chester
I agree that they should be able to secure a partnership deal if the ph2a ext data is good. The jCyte deal with Santen suggests there are prospects in this space.
Michael Hunt did state that they expect exosome data read-out towards the end of the year and into 2021 with the potential for commercials agreements in H2 2021.
He also said they will need to raise further funds although only a "manageable" amount. I imagine they will attempt to lift the share price with newsflow prior to any fundraiser.
The update was very light on detail - especially with regard to the RP trial. It seems they have started in the US - almost certainly at Mass Eye & Ear but there was no mention of Oxford, a second US site or Barcelona which was previously discussed. It's important that there is clarity around the potential impact of Covid oat trial sites and what contingency planning they have in place to avoid further felays.
RENE will almost certainly need to raise cash in the next 6 months - the CFO stated that had a little under a year's cash reserves at the last corporate presentation. I suspect it will be US investors who they will turn to. Often, such investors will insist on a future NASDAQ listing as a condition of any deal.
This is very interesting piece of research but as is stated in the article, " Mitrousis and Shoichet caution that there is a very long road between these preliminary results and a trial that could eventually find its way into the clinic".
Long suffering small biotech investors know this phrase only too well!
I view the changes announced today a positive. Having a.well qualified, active strategic shareholder on the board should inject commercial realism. It's also good.to note that they will chair the renumeration commitee which presumably decides executive compensation.
My guess is that the first Joe Public will know about a fundraiser is when it completes. I imagine US institutional investor(s) under NDA are looking at the proposition right now. £10-20m is small beer for a large VC/investor.
I agree that RENE has beem around a very long time without achieving a launched product. This might be because they chose stroke for their lead indication. Unfortunately, this has a history of many failed attempts at tteatments. I think it was MH who joked that potential investors glaze over whenever he broached the subject. Olav also stated that there is at present little partnering interest outside of China for CTX/stroke. The ph2b was expensive and the delays as a result of covid-19 in the US would only have increased costs. It seems that the board, under pressure from some investors, have been forced to conserve cash and invest only in prospects that are likely to deliver near term value.
There seems to be much greater investor appetite for indications such as RP judging by recent M&A activity. RP is also an orphan indication which confers benefits in terms of reduced development time and costs. Stroke would probably reqiure two large, very expensive ph3 studies after the ph2b whereas RP should require only a single, moderate sized ph3.
Completing the ph2b puts jCyte slightly ahead of RENE but this does not mean they have a comparable or superior product. We need to wait for the readout from RENE's ph2a extension study to see if they further improve efficacy. If the results vindicate their approach then they will almost certainly partner with big pharma on attractive terms next year.
Will there be a need for additional funds before then? Edison seem to think so - but an additional £10m would see them through another year and allow an RP partner agreement to be reached. There will be some dillution but the valuations for ph3 ready treatments should mean that long term shareholders will still be rewarded. I certainly hope that's the case...