when you say ten fold do you really mean a valuation of 150 a share? are you serious
not bashing just curious how you arrived at such a number...from what ive read- granted a limited sampling- 50 is the max for this co
very sad to think of the people out there with children going thru this horrible problem...really makes me happy about investing in Amicus and supporting the cause...
Questioning the the make up of the drug that FOLD is buying is one of the nuttiest theories out there but you're lucky because you are not the first one to try and play that angle. Read about QCOR or AVNR and see all of the dolts that were crying about the compound being easy to make. Then see what the stocks did.
Dude, you are way off on that one.
basically, if ph3 is as good as ph2b, the drug is dead. AF also eluded to this fact. So it better exceed ph2 and come as significant. I still say huge gamble by John and a bad timing as the focus should be on its own drug.
What treatments are available?
At the time of designation, no satisfactory methods were authorised in the EU to treat epidermolysis bullosa. A high standard of personal hygiene and skincare were recommended to help blisters heal, to avoid infections and to protect the skin from damage. Painkillers were also used. Surgery was sometimes necessary if there were complications such as deformed hands or the development of skin cancer.
How is this medicine expected to work?
Allantoin has been used for many years in the treatment of wounds and ulcers. It is thought to act by reducing inflammation and stimulating the body’s natural mechanisms for removing damaged tissue and encouraging the growth of new replacement tissue. In addition, it may act to prevent the growth of bacteria that can infect damaged areas. In this medicine, these actions could help to heal the blistering associated with epidermolysis bullosa.
What is the stage of development of this medicine?
The effects of allantoin have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with allantoin in patients with epidermolysis bullosa were ongoing.
At the time of submission, allantoin was not authorised anywhere in the EU for epidermolysis bullosa. Orphan designation of allantoin had been granted in the USA for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 December 2013 recommending the granting of this designation.
Opinions on orphan medicinal product designations are based on the following three criteria:the seriousness of the condition;
the existence of alternative methods of diagnosis, prevention or treatment;
either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.
to know more about this drug, google "EU/3/13/1232"
That is the orphan drug designation of the subject drug. The drug is basically Allantoin 3% and 6% cream. I now doubt if the drug has any other active ingredients than allantoin.
Review of designation
On 16 January 2014, orphan designation (EU/3/13/1232) was granted by the European Commission to ORS Oxford Ltd, United Kingdom, for allantoin for the treatment of epidermolysis bullosa.
The sponsorship was transferred to Scioderm Limited, Ireland, in October 2014.
What is epidermolysis bullosa?
Epidermolysis bullosa describes a group of diseases of the skin, in which the skin is very fragile and forms severe blisters upon minor mechanical friction or injury. The condition usually is present from birth, although some forms occur in adults. The diseases are caused by abnormalities in the genes responsible for the production of certain proteins that make the skin strong and elastic, such as collagen or keratins.
Epidermolysis bullosa is a long-term debilitating and life-threatening condition because the severe blistering and associated scarring and deformities result in poor quality of life and may also reduce life expectancy.
What is the estimated number of patients affected by the condition?
At the time of designation, epidermolysis bullosa affected less than 0.8 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 41,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).
the above is part of the document. google for the whole pdf if interested.
if still wondering about the active ingredient, it is allantoin, and i think Zorblisa used in Ph3 is allantoin 6% cream. What other ingredients Zorblisa has, I don't know, but its active ingredient is listed as allantoin, which is in many topicals at 0.1 to 2% levels.
I think Zorblisa is the Alwextin cream (probably bought from Northwestern univ Dr Amy Paller.) I guess the ph2b showed inadequacy at 3% levels, and thus the reason for the move up to the 6%. That's a non-issue.
In any case, the active ingredient is well known in dermatology (you may easily google). I assume Zorblisa does have a unique mechanism of action; it must. I also read from ph2b study that (as stated by CEO) the safety profiles of placebo-treated patients were indistinguishable from the Zorblisa-treated patients. Now that is comforting to know.
I'm no scientist but the pictures of the healing with Zorblisa looks pretty impressive.
KB looks for reasons to not like the deal and that's cool. Always good to not wear blinders.
That being said, I would be shocked if Mr. Crowley doesn't understand the data (if fact I have and would be that he does). After all, it's the type of science that he has been involved in for a long time. Trying to figure out the trial data is not what I do and that is why we have CEOs and CSOs, so that they do it for us and then we place our bets on them.
Yes, the wager FOLD has made is a risky one...CRIPE, IT's BIOTECH and that's how it works. It's easy to pick apart the deal. To say they over payed for a drug that's still in trials. Well guess what? For a drug in P3 with a billion dollar market, you not gonna get it for any less and that leads back to if you believe that management took a worthwhile, calculated risk.
I have said many times here that I have met Mr. Crowley and his family on several occasions. This is a sharp man who knows what he is doing and is as driven as anyone in the business world. Will the gamble pay off? I'm saying yes BUT it's far from a lock. Witness that Scioderm sold the product because not even they know for sure if it will succeed. They took they payoff, are filthy rich and will let the chips fall where they may.
google this stuff: Improving patients’ quality of life through development of innovative therapies September 20, 2014 DEBRA International Annual Congress
then click on the first pdf file...
In addition, a randomized trial found that a preparation that combines calcipotriene with betamethasone dipropionate (0.064%) was effective with once daily usage, and more effective than once daily therapy with either betamethasone or calcipotriene ; this combination preparation typically costs more than $400 for a 60 g tube. Patients who use topical corticosteroids in combination with calcipotriene must be monitored for adverse effects as with corticosteroid monotherapy. (See 'Corticosteroids' above.)
Similar to calcipotriene, calcitriol ointment is more expensive than many generic potent topical corticosteroids. The drug is applied twice daily.
• 45g of 1.2% tube of creams (Qty:1)
• appx. price $291.00
3k a month for a lotion from a natural source? is there any evidence (other things on the market) that demonstrate this will be supported and paid for?
So, you're claiming the ACTIVE ingredient in Scioderm's drug is a common component of cosmetics - do you really think JC is stupid enough to commit $1B for a drug that doesn't have a unique/patented mechanism of action?