ILB

PiratesWife

Member
Joined
Jan 12, 2022
Messages
19
Reason
CALS
Diagnosis
05/2022
Country
CA
State
BC
City
Gibsons
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”.

 

Nikki J

Moderator
Joined
Mar 22, 2012
Messages
11,736
Reason
PALS
Diagnosis
04/2014
Country
US
State
MA
City
Boston

Clearwater AL

Very helpful member
Forum Supporter
Joined
Aug 28, 2013
Messages
2,293
Reason
PALS
Diagnosis
12/2016
Country
US
State
NC
City
Get Real
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

Member
Joined
Jan 12, 2022
Messages
19
Reason
CALS
Diagnosis
05/2022
Country
CA
State
BC
City
Gibsons
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

Member
Joined
Nov 21, 2021
Messages
17
Reason
PALS
Diagnosis
04/2021
Country
US
State
NC
City
Asheville
Are you going to write to TikoMed?
 

lgelb

Moderator
Forum Supporter
Joined
Nov 5, 2009
Messages
11,187
Reason
Lost a loved one
Diagnosis
00/0000
Country
US
State
WA
City
Seattle
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

Member
Joined
Jan 12, 2022
Messages
19
Reason
CALS
Diagnosis
05/2022
Country
CA
State
BC
City
Gibsons
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

New member
Joined
Sep 15, 2021
Messages
7
Reason
PALS
Diagnosis
09/2021
Country
NL
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

New member
Joined
May 4, 2020
Messages
2
Reason
PALS
Diagnosis
04/2020
Country
JA
State
MN
City
Tokyo
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

Moderator
Forum Supporter
Joined
Nov 5, 2009
Messages
11,187
Reason
Lost a loved one
Diagnosis
00/0000
Country
US
State
WA
City
Seattle
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...
 
Last edited:

Haruba

New member
Joined
May 4, 2020
Messages
2
Reason
PALS
Diagnosis
04/2020
Country
JA
State
MN
City
Tokyo
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

Moderator
Joined
Mar 22, 2012
Messages
11,736
Reason
PALS
Diagnosis
04/2014
Country
US
State
MA
City
Boston
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

Moderator
Forum Supporter
Joined
Nov 5, 2009
Messages
11,187
Reason
Lost a loved one
Diagnosis
00/0000
Country
US
State
WA
City
Seattle
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

Member
Joined
Nov 21, 2021
Messages
17
Reason
PALS
Diagnosis
04/2021
Country
US
State
NC
City
Asheville
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
 
Last edited by a moderator:

lgelb

Moderator
Forum Supporter
Joined
Nov 5, 2009
Messages
11,187
Reason
Lost a loved one
Diagnosis
00/0000
Country
US
State
WA
City
Seattle
"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.
 
Top