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Definitely, sure look at the market for Botox.
It probably one I missed.
Couldn't put the trigger now.
Jim
Yes the CF trial results must be hotly anticipated by CF sufferers - let's hope the outcome is just as positive as for EB. IMHO, the real money-spinner could be Jeune Aesthetics. Krystal's aesthetics subsidiary is due to announce phase 1 data in early 2022 for "KB303, an innovative, investigational gene-delivery therapy designed to enable local, full-length elastin production in the skin". Ask yourself how much is spent worldwide on skin improvement products (that probably don't work). If KB303 really does rejuvenate the skin through gene therapy, it will be an undoubted winner!
Sorry Jayne.
Well done once.I was looking at Krystal but got greedy.
Also because of their cystic fibrosis candidate as Ireland have the highest levels in the world of it.
The Krystal Phase 3 results are certainly grounds for optimism in the EB community. However, bringing novel gene therapy to market is likely to be significantly more fraught than an ointment made of birch bark and sunflower oil. I would expect the regulators to be heavily focused on safety and it's worth noting that the sample sizes in the Ph2 and 3 trials are relatively small. According to clinicaltrial.gov, Krystal is running a study entitled "A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa" which won't read out until Dec 2022. This is probably an insurance policy against a safety knock-back from the regulators.
I did buy a few krystal last week and sold out on US market open this morning. It's almost covered my Amryt loss!
Before you dive into Krystal, it's worth noting that they are being sued (and countersuing) regarding the use of the core technology (the Herpes Simplex Virus) in their platform which further complicates the outlook. https://seekingalpha.com/article/4468950-krystal-biotechs-legal-risk-view
Jim, I've haven't heard about Vyjuvek from the contacts I have within the UK EB community, but then I've not been as involved since my family member passed on. Dystrophic EB sufferers already carry a high risk of developing cancer and my understanding is that gene therapy itself carries an enhanced risk, which is why my money was (literally) on Filsuvez.
However... Vyjuvek doesn't seem to be an insertional gene therapy so I'm wondering whether the risk has been deemed to be much lower with topical application? It's a fascinating area and thanks for the prompt as it's something I should really research further.
From my own perspective I can't see how the FDA would compare one against the other simply because of the difference in approach/risk.
Krystal's NDA won't be submitted until next year and likely well after Amryt's revised PDUFA date. FDA will only get to review the data upon submission.
I know at 1 stage a few years ago AP103 was only a few months behind and it still hasn't entered the clinic.
Krystal might get approval before Filsuvez its share price is up over 100% today.
Not sure if the FDA would be comparing the results of the 2 trial against each other.
Jayne what do you think?
https://finance.yahoo.com/news/krystal-biotech-announces-positive-topline-120000999.html
Naw I wouldn't think Krystal and Amryt have enough synergies,other than Filsuvez.It doesn't fit in with Krystal's business model imho.
+
I don't think Krystal can afford Amryt.
Probably more to do with a bad trial or maybe Krystal might be getting acquired and its a tree shake knowing how corupt these markets are.
Are you thinking what I’m thinking?
I see Krystal Biotech is down almost 20%.
Jim, Bermuda & Propinquity, thank you for the positive feedback, it means a lot. Thanks too for the link to the stem cell research Bermuda, which I hadn't heard about. Although treatments like this and AP103 are at a very early stage of development, there's at least hope of alleviation ahead for sufferers.
Interesting news today. It's probably a good decision for the company, not so sure whether it's a good deal for us as holders, but hoping it means it's looking good for Filsuvez. Good luck all.
Jayne, Thank you for your heartfelt, yet objective, view on your experience as a family-carer to a sufferer - it reveals the human aspect behind the corporate-profit motive; and, of course, how much treatments like Filsuvez mean to those who see at first-hand the distress it inflicts - hopefully not long before it reaches the market to diminish some of the pain, and make a difference to sufferers’ lives.
Some interesting posts here, thanks to all and Jayne, thanks for the insight into the issues facing those living with EB.
Worth having a read of an article published in The Times today which features 9 year old EB sufferer, Gabrielius who is taking part in a clinical trial at Great Ormond Street. The treatment involves infusions of stem cells taken from umbilical cord tissue and the hope is that these infusions can reduce pain and inflammation for a period. Although Filsuvez will hopefully become the first ever treatment approved for EB, Amryt have said that they see it sitting alongside other therapies as and when they come along. You can see how that would work in this case and let's hope that children like Gabrielius won't have long to wait before these new therapies make a real difference to everyday life.
https://www.thetimes.co.uk/article/football-dream-of-boy-with-rare-butterfly-skin-disease-dwnw0pkj2?utm_medium=Social&utm_source=Twitter#Echobox=1637252115
I'll return the compliment (:
Candid & Jim, sorry for the late response and I very much hope you're right on the FDA decision Candid. A you said it's a treatment not a cure, but the effect of any beneficial treatment can make a lot of difference. As well as being invested here, I have direct experience of Dystrophic EB as family-carer to a sufferer for decades. We were also involved in the formation of the main charity, so I've done a lot of research and know/n other sufferers personally.
It's from that perspective I know anything beneficial to prevent infection spread is important. Pain for sufferers is constant with degrees of severity, however I emphasise the infection risk in particular because alongside the additional pain caused, it also ups the mortality risk over time. As a side-note the only pain-management available for severe EB sufferers is via drugs like Fentanyl which bring along their own terrible problems and mortality risk (as detailed in the US recently).
JimBeann, there are some things we can definitely agree on, you'll be surprised to hear I even upticked your reply on OO to Elrico's bizarre Greta rant. :-) I agree with Mark Summeray, there would be benefit in dressings being changed less frequently (it's often a traumatic and painful procedure), though because of the infection risk, they'd still need changing daily. It's terrible when they're kids, but the suffering can be even worse for those who do reach adulthood. :-(
I'm an LTH here, but as well as a good investment case, it's also my way of supporting Amryt in bringing treatments to a cause that's very special to me. GLALTH.
Didn’t mean to rock the boat regarding efficacy. It’s a treatment to help mitigate the symptoms rather than a cure, so it can get misinterpreted. However the trial proved that patients did benefit from faster wound closure and that in itself is a significant help.
My view is that approval will be achieved from both FDA and EMA. Remember that an EMA approval already exists for partial thickness wounds.
As it’s on a priority review I think this will come in the coming days, there’s a chance the date can get pushed out but I think if that was the case we would have been notified already about a date extension.
https://www.biopharmcatalyst.com/calendars/pdufa-calendar
I wont disagree with you Jane we're on on the same page here.
I did listen to the Conference Call prior to FDA submission and Mark Summeray did emphasise that pain management and the fact that dressings had to be changed less frequently was very beneficial to the patients. (especially as we're dealing with poor kids here)
So sad:(
It is good from the trials. Statistically significant increase in speed of wound healing in EB between Filsuvez (formerly Oleogel) and placebo: https://epidermolysisbullosanews.com/2020/09/23/amryt-plans-regulatory-submissions-for-filsuvez-following-positive-phase-3-data/
It's not so much about pain relief. The main importance of Filsuvez to my mind is that quicker wound healing will help reduce the risk of infection. Those with Dystrophic EB in particular often suffer terrible skin infections because wounds from the blisters can remain open for weeks and become antibiotic resistant over time.
where is the efficacy talk coming from, I thought it was good from the trials?
also what's going on the with SP today!?
Candid I think the efficacy of Filsuvez is marginal but I think it's been proven there is significant reduction in the pain and suffering of the patients.
So I think it will get over the line.
Only a few weeks to go before the the approval - AMRYT’s already positioning its worldwide sales force to sell it.
I wouldn't go making assumptions like that, as most people will confirm who were in imm prior to their FDA decision.
The important point is, if it isn't approved there's still a revenue that's growing, so wouldn't be as bad as something like that. Therefore, risk vs reward is there for me personally
Anyone seen any broker research about the chances of a successful approval? Got to be quite high as the safety record is good. Could FDA still approve if the efficacy is low to moderate? I’m guessing yes as something is better than nothing treatment wise.