Sm04554

[JayM]

To be fair though it was a good spot with that they include patient experience data. It just adds to the fact this might be what they are after.

[xyz123]

This is true. It is known that the 21st Century Cures act is supposed to add patient experience data as a valid metric for considering approval.

What I want to know is why they are doing it for SM04554. This tells me one of two things: The numerical data may be insufficient and they want to supplement it with patient data to ensure approval. Or that they may be able to get some sort of accelerated approval for SM04554 before or during Phase 3. Their Phase 2 had an unusually large number of patients. The 21st century cures act hints at accelerated approval without a phase 3, but mostly for treatments of life threatening diseases.

I really don't think they are missing their numbers. This second study with scalp biopsies has more ambitious endpoints than the first trial (and correct me if I'm wrong - but the hair growth questionnaire was filled out in the first trial - not the second? ).

These more ambitious endpoints are with just 50 patients - so ~ 17 in each group. They know how the drug is going to perform in this second Phase 2 based on the first Phase 2 - and I'm sure they based their sample size in the second study based on the results of the first. A smaller study indicates that they anticipate hitting these endpoints without needing as many participants - ie. the drug has a large effect size so fewer participants required to show statistical significance. If the drug didn't work, they would be setting themselves up for failure in the second Phase 2 study - and no company does that - they would either go back to the drawing board or move on.

Everything points to SM04554 being a success.

[nameless]

I really don't think they are missing their numbers. This second study with scalp biopsies has more ambitious endpoints than the first trial (and correct me if I'm wrong - but the hair growth questionnaire was filled out in the first trial - not the second? ).

These more ambitious endpoints are with just 50 patients - so ~ 17 in each group. They know how the drug is going to perform in this second Phase 2 based on the first Phase 2 - and I'm sure they based their sample size in the second study based on the results of the first. A smaller study indicates that they anticipate hitting these endpoints without needing as many participants - ie. the drug has a large effect size so fewer participants required to show statistical significance. If the drug didn't work, they would be setting themselves up for failure in the second Phase 2 study - and no company does that - they would either go back to the drawing board or move on.

Everything points to SM04554 being a success.

You could be wrong but if you are then so am I because I concur with you. I also think that the fact that they have only 50 people in their additional phase 2 indicates that they expect a high rate of success or else they would have involved more subjects.

[lacazette]

What about the asian market? Maybe Samumed is planning something there also, if they have to do a phase 3

And in japan the approval process is also quicker for new drugs

"Japan’s Pharmaceuticals and Medical Devices Agency was quicker to approve more new drugs in 2014 than its U.S. and EU counterparts, according to a new study released by the Centre for Innovation in Regulatory Science."

Maybe this second phase2 with biomarkers could be sufficient to ask fir approval procedure in japan, or maybe just the need of a little phase 3 with more biomarkers.
I think there's a possibility that samumed will hit the asian market sooner than in US/Europe

[failly]

What about the asian market? Maybe Samumed is planning something there also, if they have to do a phase 3

And in japan the approval process is also quicker for new drugs

"Japan’s Pharmaceuticals and Medical Devices Agency was quicker to approve more new drugs in 2014 than its U.S. and EU counterparts, according to a new study released by the Centre for Innovation in Regulatory Science."

Maybe this second phase2 with biomarkers could be sufficient to ask fir approval procedure in japan, or maybe just the need of a little phase 3 with more biomarkers.
I think there's a possibility that samumed will hit the asian market sooner than in US/Europe

Why would a company want a faster approval by going to Japan? Even though it may be approved, the FDA will still do their own work, which takes some years.

Even if a treatment(speaking of all, not only hairloss) gets approved by the FDA, people in Europe will still be waiting some years till they get it. I think every big country or continent does it's own testing, even though it is approved in other countries.

[SriHanuman]

Why would a company want a faster approval by going to Japan? Even though it may be approved, the FDA will still do their own work, which takes some years.

Even if a treatment(speaking of all, not only hairloss) gets approved by the FDA, people in Europe will still be waiting some years till they get it. I think every big country or continent does it's own testing, even though it is approved in other countries.

That is not true, they take around 6 months to approve drugs already approved by other regulatory bodies. There is a drug lag, but not in years. Read this:

The Speeding Access to Already Approved Pharmaceutical Actwould accelerate the review process by requiring FDA to review any drug approved for use in the EU in just 90 days—far more quickly than FDA's current fastest assessment, which takes six months (priority review designation). - See more at: http://www.raps.org/Regulatory-Focus....yTErdaPY.dpuf

[failly]

That is not true, they take around 6 months to approve drugs already approved by other regulatory bodies. There is a drug lag, but not in years. Read this:

The Speeding Access to Already Approved Pharmaceutical Actwould accelerate the review process by requiring FDA to review any drug approved for use in the EU in just 90 days—far more quickly than FDA's current fastest assessment, which takes six months (priority review designation). - See more at: http://www.raps.org/Regulatory-Focus....yTErdaPY.dpuf

Good to hear it's faster the other way. However, it states "for use in the EU". I was talking about a drug that has been approved in the US coming to the EU will take years. I don't know if it's true though, I heard it on the news some days ago about another drug.

[SriHanuman]

Yes, it is true you were talking about US -> EU but I thought that it is the same timeline, or at least I hoped so. Then I found this: http://www.ncbi.nlm.nih.gov/pubmed/26013294

[dus]

If needed us Europeans will arrange a ****ing submarine and stuff it with Sm04554. Or I'll just do a couple of plane trips .

[lacazette]

Why would a company want a faster approval by going to Japan? Even though it may be approved, the FDA will still do their own work, which takes some years.

Even if a treatment(speaking of all, not only hairloss) gets approved by the FDA, people in Europe will still be waiting some years till they get it. I think every big country or continent does it's own testing, even though it is approved in other countries.

What I meant is that they could maybe have enough data to have approval in japan and hit the asian market. But of course they would still continue the FDA process normally for US approval. But like that we could buy by internet some SM from asia before US/Europe approval. They just need a partnership with a japan pharma company, and commercialisation would not take that long there

If they can have japan's approval sooner, I don't see why they would wait the fda approval to hit the asian market

[failly]

What I meant is that they could maybe have enough data to have approval in japan and hit the asian market. But of course they would still continue the FDA process normally for US approval. But like that we could buy by internet some SM from asia before US/Europe approval. They just need a partnership with a japan pharma company, and commercialisation would not take that long there

If they can have japan's approval sooner, I don't see why they would wait the fda approval to hit the asian market

Didn't thought about that. That is indeed true. But the price will skyrocket, since the demand is so high from both the US and the EU. Atleast it's something!

[rdawg]

This is getting close to completion isn't it? I thought it was done by around November?

will be very interesting to see the results.

[Tomtom21]

I really feel that samumed has got a product that at the very least will be an additional to add to our arsenal. I was looking at their additional round of phase 2 study with the punch biopsy. I was curious to see what exactly they were looking to evaluate from the tissue biopsy in terms of biomarkers. straight from the clinical trials page they are aiming to evaluate the following:

Change in nuclear expression of beta-catenin
Change in nuclear expression of Ki-67 in epidermis and hair follicles.
Change in Ki-67 index in epidermis and hair follicles

First off beta catenin, I believe we all understand at this point if we follow the thread that it has direct affect on wnt pathway and therefore blah blah blah. Basically it can be very important in reactivating our dead beat hair follicle cells that don't want to do their job anymore.
However, I was curious to look into the Ki-67 biomarker they were interested in. I looked at what exactly Ki-67 protein is for and straight from wikipedia:

"The Ki-67 protein (also known as MKI67) is a cellular marker for proliferation.[5] It is strictly associated with cell proliferation. During interphase, the Ki-67 antigen can be exclusively detected within the cell nucleus, whereas in mitosis most of the protein is relocated to the surface of the chromosomes. Ki-67 protein is present during all active phases of the cell cycle (G1, S, G2, and mitosis), but is absent from resting cells (G0)"

(G0) is = senescent phase where nothing is happening. Whereas previous studies have shown that our hair follicle cells experience longer and longer phases of G0 and shorter phases of interphase and mitosis, until ultimately permanently senescent and unfortunately slick bald.

All this coupled with the fact they recruited high norwoods, had a huge first phase 2 study, decided to run another smaller phase 2 study with biomarker biospy evaluation simultaneously with their first phase 2 study, and are evaluating these biomarkers which are directly linked to cell proliferation seem to all point to a positive opposed to a negative. Of course this is all speculative and we will have to wait a few more months to see just exactly the results are.

[JayM]

I really feel that samumed has got a product that at the very least will be an additional to add to our arsenal. I was looking at their additional round of phase 2 study with the punch biopsy. I was curious to see what exactly they were looking to evaluate from the tissue biopsy in terms of biomarkers. straight from the clinical trials page they are aiming to evaluate the following:

Change in nuclear expression of beta-catenin
Change in nuclear expression of Ki-67 in epidermis and hair follicles.
Change in Ki-67 index in epidermis and hair follicles

First off beta catenin, I believe we all understand at this point if we follow the thread that it has direct affect on wnt pathway and therefore blah blah blah. Basically it can be very important in reactivating our dead beat hair follicle cells that don't want to do their job anymore.
However, I was curious to look into the Ki-67 biomarker they were interested in. I looked at what exactly Ki-67 protein is for and straight from wikipedia:

"The Ki-67 protein (also known as MKI67) is a cellular marker for proliferation.[5] It is strictly associated with cell proliferation. During interphase, the Ki-67 antigen can be exclusively detected within the cell nucleus, whereas in mitosis most of the protein is relocated to the surface of the chromosomes. Ki-67 protein is present during all active phases of the cell cycle (G1, S, G2, and mitosis), but is absent from resting cells (G0)"

(G0) is = senescent phase where nothing is happening. Whereas previous studies have shown that our hair follicle cells experience longer and longer phases of G0 and shorter phases of interphase and mitosis, until ultimately permanently senescent and unfortunately slick bald.

All this coupled with the fact they recruited high norwoods, had a huge first phase 2 study, decided to run another smaller phase 2 study with biomarker biospy evaluation simultaneously with their first phase 2 study, and are evaluating these biomarkers which are directly linked to cell proliferation seem to all point to a positive opposed to a negative. Of course this is all speculative and we will have to wait a few more months to see just exactly the results are.

I'm excited by it as well. I guess the thing is though they could just be doing this because they saw sweet F all. Like see if the drug is actually doing anything haha.

I dunno. I'm not so strong on scientific methods but maybe with such a huge group for phase 2 it was easier to do a separate phase 2 for biopsies if it actually did work. To be fair maybe you can't even go to phase 3 without this information even If you see hair growth.

They better say something in November!

[champpy]

that's one thing that makes me really nervous, them not saying anything in November and we have to wait again just like we are waiting for bimatoprost