Gordon Stein, CFO of CleanTech Lithium, explains why CTL acquired the 23 Laguna Verde licenses. Watch the video here.
Thanks for highlighting. Very good news for patients. Interesting that the clinical trial was open label. If MCW run a similar trial for Glioblastoma, it would make recruitment potentially easier as all participants would be on drug.
Interesting that there is no mentiin of working capital. I was under the impression they were just about out of cash based on thw run rate of the previous few years. Maybe the imaging business is now at least covering its costs?
So looks like the foubdation is working on a fund to cover the full cost of the EAP. They must have real faith in the science. Offering the drug free on an EAP in the US (with its high drug cost) would, I suspect, raise the profile of GaM massively.
I would suggest it is basically saying the maps provide a more accurate assesment of the extent of tumour than a radiologist would otherwise determine by looking at the same MRI images (that are used as input for the map generation software). This improved accuracy helps the surgeon remove maximal tumour (interoperative MRI) which I think is called resection. The maps are also seem to help predict the prognosis for the patient.
But I am no expert :-)
It's unfortunate that the latest video was taken down by IB after only 1 day. I watched the presentation during the brief time it was available. Two points were standout. Firstly, they have now started recruitmemt at the 2000mg dose.. This is important because, as I understand it, the dosing used so far is way below that used in the pre-clinical animal models which demonstrated increases in PFS. If efficacy is dose dependent, then it would seem the 2000mg and 2500mg patients are likely to derive most benefit. Given the terrible prognosis for recurring disease, it should be possible to detect an efficacy signal in the near term. Secondly, Dr Connerley stated that the team were going to be analysing samples of tumour taken from trialists (post-treatment) as well as measure drig levels in blood. These actions give the investigators a better understanding of how much drug is in the body and its effect on glioblastoma at a cellular level. This is all part of the "discovery" process of first-in-man use (for this indication). I would expect 2024 to be a year of potentially very meaningfull news. So far, it seems GaM has been very well tolerated with little or no side effects. Given the serious side effects of existing standard of care (Chemo & Radiotherapy), I imagine the potential for a low/no side effects treatment that extends survival, by even a few months, is high. TTF, (Optune) which is approved sadly extends survival by only a relatively short period and requires using the appliance for long periods.
As you said, it seems like a basic mistake, probably by management. I'm sure they have learned from this and don't imagine a short delay will have any real implications. The FDA are engaged and now expecting a revised application so it's not like starting from scratch.
I find it hard to believe anyone from the clinical team actually submitted the original request. According to the FDA ODD database, GaM has been designated for (1) glioblastoma and (2) atypical teratoid/rhabdoid tumours. Indeed, in all of the phase 1 presentations, by Dr. Connelly, it is explained that the WHO has classified brain tumours based on certain genetic characteristics and makes a point of differentiating between glioblastima and lower grade(s) glioma.
Thanks for this. I've watched a few of these now and learn snippets of new information every time. It's great to see really thorough clinical testing taking place while also looking to accelerate avaiability through an EAP.
The results presented are interesting but I'm not sure you can read too much into the efficacy results. its worth bearing in mind that tbey are only now reaching ph1 doses somewhere closer to that used in the preclinical animal studies (50mg/kg/day). Given that GaM is thoughr to effectively "crowd out" iron in the cancer cells, I would have thought that efficacy would be highly dose dependant. In which case, we may see a stronger efficacy signal in the two additional cohorts (2000mg and 2500mg) recently added. Encouraging to hear they are well on the way to closing out recruitment given the 6 month timescale quoted.
A very sizeable grant given the total of 2023 awrds. The foundation is also actively promoting the Promising Pathways act in the US. This act could significantly reduce time to market for promising treatments that have been through a phase 2 trial.. It also ties in nicely with expanded access as this could provide a demonstration of real world efficacy.
This has probably already been discussed but for those who missed it (like myself), the animal trial publication (Jan.2024) is here:
https://www.frontiersin.org/journals/oncology/articles/10.3389/fonc.2023.1278157/full
Its worth a read to get some understanding of the science. Interesting to see the dosage level used (50mg/kg/day) which resulted in slowing of disease progression. This would suggest an equivalence in humans of 3750mg/day (assuming 75kg) which is significantly higher than the doses in the phase 1 used to date. It seems that the team were conservative in their choice of starting dosage (maube for very good reasons). It does beg the question as to whether the lower doses are likely to have an effect. Perhaps, they will only start to see a.signal at the higher doses now being tested - 2000mg and.above. I do recall that it has been stated upwards of 3000mg was tolerated by some patients in the original Titan Pharma trial of GaM (according to Dr..Chitambar).
This probably explains the sharp drop.
https://www.biospace.com/article/kazia-s-paxalisib-disappoints-in-global-glioblastoma-study/
I imagine it will be extended again at the appropriate time. I did read that there was some debate about how effective the program is but nobody has come up with a better idea.
A similar voucher program exists to encourage the development of treatments for agents that present national security threats (thinl chemical, biological agents that can be weaponised). The bill to extend that program was renewed recently (google H.R.5708 if you want more info)
So, if the 2000mg is tolerated, that's 3 more patients, plus potentially 3(+3) at 2500mg (RP2D). I imagine that recriitment won't take too long now they have administered to 18(?) without any real safety issues.
Latest with more info on dosing schedule. Good to see they are recruiting at 2000mg now. I believe its 3 more patients at this dose plus six at 2500mg if tolerable.
https://youtu.be/67D6MwSQc7U?si=FyoqNYKYyLzJJTE7