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There may be interest in the dosing and safety data from the Ph1. I believe all trial data is owned by IB. Presumably, if another company/researcher were considering a trial in other cancers, having this might allow them to justify a move straight into a phase 2?

Https://www.sciencedirect.com/science/article/abs/pii/S0041008X25002364

The vouchers are for pediatric conditions. It will be interesting to see what IB do if the scheme is reinstated as they suggest might happen. The adult safety data would probably allow the FDA to authorize a trial in kids. Gemini AI suggests a phase 1/2 would be appropriate - dose escalation followed by a larger cohort at the RP2 dose. Both of the rare pediatric conditions for which IB has designation are rare so perhaps a combined trial. The possibility of a $200m pot at the end of the rainbow may tempt a suitor.

I watched a video a while back where they interviewed one of the medics who had gone through the EAP start-up process. The doctor had managed to scrape together enough from departmental budgets to fund the onboarding. However, from what she said, it is clear that funds are not easily available, even in larger US health systems. Glioblastoma is also quite niche - it's not one of the cancers that big pharma invests heavily in. Funds will always be tight - this is the problem they are facing. In an ideal world, any new treatment that offered hope would be funded through the development lifecycle - but the reality is that funding is always a commercial decision. If IB can show efficacy through extended survival as time progresses, they may attract some interest. It will all depend on what is published by the MCW team.

There would need to be an end-of-phase meeting with the FDA once the trial results have been analysed. In addition to considering the results, this enables the FDA to guide on the future development path. I would suggest that holding out for more data is a wise move. Pharma's often try to accelerate development due to investor pressure - but FDA doesn't care about that. They have a process and want to see results at each stage of the development.

It is possible, under the existing regulatory framework, to gain approval earlier in the development cycle. Cancer drugs that show efficacy in fatal conditions that have no existing therapy can be granted accelerated approval. It's provisional on phase 3 confirmation and can be withdrawn, but it does allow for provisional approval after phase 2.

Looking at the clinical trials record...

Primary Completion (Actual) *

2025-02-13

Study Completion (Estimated)

2025-10-10

*The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "estimated" primary completion date is the date that the researchers think will be the primary completion date for the study.

So, given that the primary outcome was safety, it looks like they finshed collecting the safety data in Feb. The efficacy element was secondary and, since it rekates to how long a patent survives, takes longer to measure - but I believe it was supposed to be at 6 months. That would tie in with the completion date of October. Obviously, for people who are responding well to the drug, I would expect them to continue on the drug.. So the true survivorship figures would improve..

It's not an oversight. The timing is because the people are in the USA.

I suspect that is a sale to an end customer.. especially given they were granted to third parties...

Same reaction :-) Its one of two scenarios - he's either been TOLD to buy some, or he thinks something good is around the corner! I hope it's the later!

Thanks, Panman - the interview was interesting (if short). I wonder if this is the Promising Pathways Act that Dr Musella has been campaigning for over the last several years. There is also an existing pathway for breakthrough therapies (see below). A lot depends on the efficacy in the Ph1.

https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy

Another area of research by Dr Al Gazawi in the lab is use of GaM in combination treatment with radiation. They did some initial studies that showed that GaM appears to act as a radiosensitiser, leading to much greater tumour cell damage by radiation. If proven, this might mean GaM could be used across multiple cancers and would be an absolute game changer. Fingers crossed on that one!

I think the GE Sales Training is really the stand-out point. Having no dedicated salespeople is not exactly a winning strategy!

Running clinical trials in people with terminal illness is hard. Patients can enroll but then fail to complete the treatment because they are too ill, or even die. I would wait for the data to be presented by Dr. Connelley before reaching any conclusions. Whatever you think of IQ AI, the trial itself is run by the medical team out of the Medical College of Wisconsin. They are clinicians with a reputation to uphold.

Https://youtu.be/qEyrNvnYZcM?si=eClF2HJ5VvgwD_P1

It was probably an oversight. If you compare the clinical trial records (see the history), you will see that the completion date status has changed from "estimated" to "actual." Assuming the last two patients have been treated then I assume they will need to wait 6 months for an indication of any positive effect.

Agreed with your comment regarding the trustees' obligations but it's worth reading Note 33 in the 2023 annual report. This caught my eye...

"The rules governing these schemes provide an unconditional right to a refund assuming the gradual settlement of the scheme’s liabilities over time until all members have left the schemes."

"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.