ILB

[PiratesWife]

What do we think of this? Could be years before it’s available and looks like it would be a monthly injection for life, but still hopeful?

“In a Phase II study of ILB® administered to patients with ALS for five weeks, the progression of ALS was halted and/or reversed for all study participants assessed using the validated ALSFRS-R and Norris clincal rating scales that score a broad range of patient relevant functions”.

TikoMed AB reports positive phase II clinical data for the treatment of Amyothropic Lateral Sclerosis in data published by PLOS ONE | BioSpace

TikoMed AB reports positive phase II clinical data for the treatment of Amyothropic Lateral Sclerosis in data published by PLOS ONE - read this article along with other careers information, tips and advice on BioSpace

www.biospace.com

 

[Nikki J]

The biomarker data are encouraging but this was a 13 person trial and the treatment was weekly for five weeks with a 70 day follow up which is too small and too short to really tell us much. Hopefully a larger and longer trial will validate this. The treatment burden is negligible I think. Here is the paper A phase II open label clinical study of the safety, tolerability and efficacy of ILB® for Amyotrophic Lateral Sclerosis

 

[Clearwater AL]

Thanks Nikki for the link. Took a while to read it over and a lot terminology was over my head
but what I did digest is encouraging.

Reading this JAMA link over in a segment of slower progression I found some encouraging news…
the second sentence below . (Well, maybe “encouraging” isn’t the right word here but…)

A couple of key sentences…

Conclusions: Contemporary patients had significantly prolonged survival and slower disease
progression compared with patients from the historical group

*Our observations suggest the possibility that disease course has changed and that ALS is
becoming less aggressive over time.

The improved outcome seemed independent of specific ALS outcome-modifying therapies,
but have influenced medical treatment and survival.

 

[PiratesWife]

One would hope that results like this would garner expedited process and trial completion. I know we shouldn’t get our hopes up but I personally need hope like this to keep me going. One could go crazy knowing that the best potential treatment that could make ALS livable is in Sweden right now as we speak. We would travel to be a part of future larger trials if it was an option.

 

[GreenCat]

Are you going to write to TikoMed?

 

[lgelb]

They are already in the orphan drug pathway for both the US and EMEA. That gets them some monetary and commercial benefits. They haven't yet done a blinded [people do not know which arm they are in] trial to compare TikoMed recipients with those who don't get it (standard care).

Probably looking for a partner -- usually why you do a cheap one to start with, to try to get data to snare a partner with. However, that does not mean that the partner will choose to have US sites for the next trial, and it can be faster not to have them.

The company that ends up spearheading the program, whether TikoMed or someone else, can apply to the FDA for the various fast-track designations if/as they have the data that will justify that. But with no blinded trial data as yet, ILB is pretty far from any kind of compassionate use program, if that's what you're talking about.

 

[PiratesWife]

Yes I did write them and recieved this auto generated response:
“Many thanks for your interest in Tikomed and your interest in our drug candidate ILB®.

Tikomed is committed to developing safe and efficacious therapies for neurodegenerative diseases. We understand the huge impact these diseases can have on the patients and their families' lives. We are committed to providing these therapies to the broadest group of patients as quickly as possible. As part of this commitment, we conduct clinical trials to determine if our investigational medicine is safe and effective for patients with a particular disease, disorder or condition which may allow us to seek the necessary regulatory approvals to provide wide access to this medicine.

To date, our drug candidate ILB® has not been approved in any country. Currently our drug candidate ILB® is not available for use outside clinical trials. TikoMed does not provide early access to our drug candidate ILB® in this stage of development outside our clinical trials.

Please refer to www.clinicaltrials.gov for updates and news about our clinical trials.”

Best regards,
TikoMed AB

 

[smarinkov]

It appears that at least two other parties are pursuing the hepatocyte growth factor (HGF)-based solutions (such as ILB). Both placebo-controlled:

• Helixmith: drug Engensis / VM202, plasmid that carries instructions for HGF protein, intramuscular injection, phase 2 (active), results in summer 2022
• Tohoku University Hospital, drug KP-100-IT, recombinant 5-residue-deleted HGF, intrathecal administration, phase 2 (active), results in summer 2022

Hopefully, they confirm the encouraging findings from Tikomed.

 

[Haruba]

They have apparently finished a similar (small no placebo) trial in the UK, but have not announced any results. That trial doses participants weekly for 48 weeks.

Wonder why they are moving so slow. They started with IV then switched to subcutaneous, so maybe that delayed initial development, but not publishing results from a long completed trial is curious. This trial in Sweden has also been complete for a long time. TikoMed doesn't seem to be acting with urgency.

Best hope is to lobby the company to apply for the Healy Platform Trial. Best case could be available in two years.

 

[lgelb]

Remember publication lag from database lock, and they don't have to publish at all at this stage. But, the longer the trial, the more data cleaning and stat procedures to account for dropouts, deaths, and other missing data. And everything will match up with the volumes submitted to regulators down the road.

Again, if they don't have the money to run a larger / controlled trial, which by my count, they don't, there's not much that they can do until they get some, either through M&A, partnership, or other strategic investors.

There is no way that a therapy that has no controlled trials as yet will be marketed in 2y. But when you see a trial listing...

 

[Haruba]

By "no controlled trials" are you referring to the lack of placebos? I don't believe the Healy Trial requires placebo trials to consider a drug for inclusion. They require proof of safety in humans and multiple animal models.
If included in 2023, it would be possible to conclude by end of 2023 for approval in 2024.I contend this is feasible, and the most cost effective path for small pharma companies like TikoMed.

 

[Nikki J]

The Healey trial selection is very competitive. I don’t know that they require a placebo trial but they certainly look at the totality of the evidence to determine which applications show the most promise. None of us ( unless someone here is on the selection committee) can know how this might compare. It would be up to the company to apply if they have not already done so. I believe at least the next 2 treatments have been chosen and are in the process to be rolled out. The Healey platform is wonderful but their resources are not limitless. It is also true that they carefully designed the trial so that a really excellent result might go for approval but in this case the lack of an earlier placebo controlled trial might make the FDA more skeptical. We can’t know any of these things but For Healey participation the first step would be the company’s

 

[lgelb]

Nikki is right --the Healey platform wouldn't be enough to get regulatory approval in this case. I don't see that ILB would meet their criteria as yet anyway. They just need to keep going, is all. If the UK results are promising and the trial was conducted appropriately, I would think they could attract a partner, licensee, or buyer.

And no, not all controlled trials are placebo-controlled.

 

[GreenCat]

Dear All. Do you happen to know about the "Right to Try" law? ILB is dextran sulphate, It's a patented, low-mol. weight form.of DSS and the clinical dosage is LMW-DS, approx. 20 % sulfate, mean MW 5 kDa [edited for accuracy; the trial you linked to is complete but is now linked to its NIH page]

Do you know what the rules are about compounding under the Right to Try law a drug like this?

Thanks,
Liz

 

[lgelb]

"Right to Try" is a pathway for compounds that are further along (remember, no controlled trials for ILB yet), and was really just a packaging of packaging existing early approval pathways that the FDA already has the discretion to use. Anyone can try to get anything compounded, but doing so for ILB would not be legal under RTT or any other regulation.