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There is some very encouraging news in the Independent amd Telegraph regarding an immutherapy treatment for Glioblastoma. It looks like the NHS will be sponsoring further trials.

I am hoping this will highlight the disease more generally. GaM anti-cancer properties may not be limited to brain cancers.

This happens all the time with the FDA. Companies go in too early, with insufficient data, and hope they can bluff their way through. They haven't completed the PH1 and haven't (yet) done the imaging work (using their own software!). But that's what happens when you limp along on a shoestring budget, relying mostly on grants and academic support. Dr. Connely has a day job! If they do produce compelling data, the doorbell will start ringing, I'm sure.

The patient in the presentation is on the 1500mg dose (below the max dose of 2500mg), has NO SIDE EFFECTS, is celebrating TWO YEARS on the trial, all with STABLE DISEASE? I hope others are responding similarly to the treatment because it may offer a ray of hope that the disease can be slowed significantly.

Dr. Connelly has not said much about the trial since her "disappearing spoon". That could all be about to change now that the last few patients are being treated. We could get some positive news on the FTB mapping results that radically change the PFS. I am waiting with great anticipation for an update from the MCW team.

Just in case anyone missed the link

https://www.mcw.edu/mcwknowledge/mcw-stories/innovative-glioblastoma-treatment-advances-thanks-to-groundbreaking-research-and-community-support

Very informative presentation. Highly encouraging that the brave patient who participated in the video and who is in the GaM trial, is doing so well. His scans out to 679 days with stable disease, and the fact that he is "doing well", are a testament to the level of care being provided. His oncologist is Dr. Connelly, lead investigator in the GaM phase 1.

149 bipartisan sponsors! - That seems like a good start!

Sorry, I got the wrong trial. FIREFLY-1 (a phase 2) was the basis for approval. This ran at 35 sites and started in April '21. Still an amazing timeline.

Tyke

The phase 1 has constraints that limit eligibility, namely being single site and limited to refactory/relapsed glioblastoma, which partially explains the length of time it has taken. A phase 1 may also be viewed as highly experimental by those looking at clinical trial options. A phase 2 will have the benefit of the (limited) efficacy readout from Ph 1. Also, the clinical team have previously stated that they wish to trial GaM at an earlier disease state in the phase 2, which would increase eligibility. This, and the use of multiple sites in a phase 2, should lead to much faster recruitment. The Firefly-2 trial, used as the basis for OJEMDA approval, ran in a huge number of sites across multiple countries. Clinical trials.gov shows the start date as Feb '23 and the drug was awarded accelerated approval in April '24. That is an amazingly fast timeline but it shows what is possible if funding is available. It would be nice to think GaM has similar potential.

Tyke

Hopefully, IB will also be granted accelerated approval which could further speed things up. I did check the list of drugs that had received this approval over the last 12 months and came across OJEMDA. This drug was approved to treat Pediatric low-grade glioma. Approval was based on an open label phase 2 enrolling a total of 137 patients. Arm 1 (77 patients) was used for the efficacy analysis and arm 2 (60 patients) provided asditional safety data.

...seeks to renew the voucher scheme for an additional 5 years... It has already been passed bybthe house and is now with the senate...

Web search for more detailed info.

Apolgies that document defines proposed development paths, not definitive ones that the FDA uses. Still, the paper might give us a clue as to what the promising pathways act would look like, if implemented.

I found this document, which compares the various approval paths. Worth reading to gain an understanding of the possible approval paths.

https://pmc.ncbi.nlm.nih.gov/articles/PMC3745545/

Look at table 2 "Potential Breakthrough Categories, Criteria, and Development Paths"

For drug categorization (cat/row 1) "Drug addresses serious condition with poor outcomes for which there is no SoC" the Potential Development Path is:"Phase I B expansion or single arm pivotal trial could lead to full or accelerated approval in single arm study"

then look down at what FDA consider a Cat 1 drug and you find:

b. Drugs that demonstrate substantial efficacy in refractory populations.

What is IB trial called? "A Phase 1 Clinical Trial of Oral Gallium Maltolate for the Treatment of Relapsed and Refractory Glioblastoma"

Wow! Imagine if they only needed a phase 1B to get this over the line!

The notes

It looks like FDA offers discounted rates for small companies - less than 50 employees or low $ turnover.

BB

Could yiu share where you got that figure. Is that the cost the FDA charges an applicant or a more general figure relating to how much it costs to prepare such an application.

Many thanks

So, a total of 500 over the lifetime of the program. Very low when you consider the overall number of therapies in development.

Or possibly the broker (paid in shares?) or a placee from the last fundraiser.

It really says something about the quality of the clinical team in that tbey have got so far on a shoestring budget. Breakthrough is rarely granted by the FDA If they do obtain it, then it's a real wake up call and should unlock further funding.

I suspect the truth was that there were 40 ish platform installations being evaluated within which IB software was bundled as one of the many available packages. He could therefore just about get away with saying the software was being evaluated by all the sites. The truth was probably that most had no interest. It's an extremely specialised product relevant to advanced diagnostic cancer centres.

No problem panman. Its also good to know they are treating the final 3 patients. Although they can go.to the FDA with the 1 month safetu data, that now seems.oretty much a given in terms if approval to proceed to ph2. What is more interesting is the efficacy. I am guessing they will have mote xompelling data in the later half of the year as they inckude more patients who took the higher doses - hopefully, they are responding well to treatment.

Ah no.. it doesn't say a meeting in q2. It says they will submit a request for a meeting. The FDA has a gormal process that involves making a submission first. There is then an SLA which determines how long the wait is before the company meets with the FDA. I can't recall the delay period but you can find it on the FDA website.