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Bronxville - let me try to explain why Amryt paid 300 million for 1.8 million in revenue. Firstly, MYCAPSSA was only launched recently in USA, about 6 months ago. Hence, the USD 1.8 million revenue likely represents 100 acromegaly patients already on the drug.
By the end of this year, I would expect 300 patients on the drug, which would represent an annual revenue of up to USD 18 million. At the moment, it looks like they may be getting 10% market penetration, as 3000 new acromegaly patients are diagnosed in USA/year. In addition, there are 25,000 existing acromegaly patients in USA at present, who may switch from injectable octreotide/Lanreotide to MYCAPSSA.
Within 2-3 years, I believe that a target of 1000 patients on MYCAPSSA in USA could easily be achieved. With EU approval, the number could be easily doubled to 2000 patients (which would represent USD 120 million annual revenue). Within 3-5 years, I believe annual sales of USD 200-300 million is achievable.
Further uplift in this figure could be achieved if 1) The market penetration in the newly diagnosed acromegaly population is higher eg 20% 2) More patients decide to convert from injectable to oral somatostatin therapy 3) Amryt achieves approval in GEP-NET indication (whereby revenues could easily be doubled).
Main difference between Krystal’s product (KB-103) and Amryt’s AP-103 is that Krystal’s product is viral-based, whereas Amryt’s is synthetic. KB-103 is a Herpes Simplex Virus -1 (HSV-1) based gene therapy; it therefore carries a higher risk of immune response. AP-103 is a more novel technology; if it succeeds, it could be a disruptive technology. Both are applied directly on the patients skin, and requires repeat application.
Yes, I agree that it would be difficult to grow into a company like Shire, which was sold to Takeda recently, and had a market cap of around USD 40 billion. But what I was talking about was a more strategic viewpoint. There are biotech companies in USA focusing in Orphan Diseases, currently worth USD 2 - 4 billion in market cap (like Amicus). The strategic decision here is whether to go for quick sale circa £200 - 400 million, or try to grow Amryt into a similar company like Amicus. The hiring decisions that Amryt have made e.g. VP in Regulatory/Licensing, etc, suggests that they are going for latter. Whereas if they were going for quick sale, they would tend to keep staffing minimal (as suggested in your previous posts). But it looks like their long term growth strategy has brought benefits - eg, funding from the Irish government for the AP103 program.
Possible Candid, but there must be a catalyst for the SP spike. Example, if Amicus have made them an offer for a licensing deal, subject to FDA approval, which they have accepted. But I think they year ahead looks bright for Amryt. Filing in early 2020 with FDA & EMA + FDA piority voucher worth 80-200 million usd.
Ajok - As per definition in MacMillan Dictionary:
Game-changers - something that completely changes the way something is done, thought about, or made.
Because there are currently no approved therapies for EB, in theory, if Episalvan does get approved, it could be a 'game changer' as it could change the way dermatologists treat their EB patients.
However, I agree that the efficacy seen with Episalvan is modest - therefore when you say it is not a 'game changer' - you are implying that it does not have BIG efficacy.
A Ph III trial would usually assess these aspects including pain, etc, using a Quality of Life (QoL) questionnaires throughout the study. However, for Regulatory drug approval, a pivotal study uses data such as time for wound healing as Primary Endpoint. For reimbursement discussions, I am sure that Amryt will be using Primary/Secondary Endpoint data as well as QoL.
Ok.... if you had a child with EB, and the doctor offered a treatment that made the fragile wounds heal faster by 1-2 days. Not only that, but helped your child sleep better, reduce the wound pain, redness, swelling, etc. No doc, don’t want any of that!
Hi Bronxville,
You are right that it looks like Episalvan is anticipated to improve wound healing in EB by about 10%, and there could be perhaps a 1 - 2 day improvement in wound healing vs. placebo. Firstly, this must be taken into context that a lot of pharmaceutical agents improve a certain parameter by 10% (or even less). Biological agents or gene therapies usually have a greater efficacy. For example, when you take two paracetamol (acetaminophen) tablets, an average person's body temperature will decrease 0.3C. In terms of total body temperature, this might be only be a decrease in body temp by 1% - but the person taking paracetamol with a temperature will likely feel a lot better! Likewise, there are other dimensions of wound healing that needs to be taken into consideration - example, pain, discomfort, and overall quality of life. More importantly, my strong feeling is that Episalvan is likely to represent the first drug to be approved for EB, which is a big step for EB patients. Later on, when there are other agents approved (say in 5-10 years time), it would be possible that 2 or 3 therapeutic agents may be used in combination, to provide even better efficacy. The key is to provide as many treatment options for EB patients. But do not simply state 'does wound healing by 1 day or even 2 days as compared to the placebo really make that much of a difference' - without looking at the bigger picture.
Hi Cookie45, As with most Interim results, you will not be provided with the granular results i.e. Wound Closure at D45 - as the knowledge of the efficacy results can affect the integrity of the trial. For example, if the results show a 20% improvement in wound healing, then existing patients may decide to abandon treatment if they do not see the marked improvement. Hence, the main goal of the interims is to inform of 3 possible outcomes 1) Continue study 'as is' 2) Increase study sample size 3) Discontinue study due to futility. This stop/continue decision will be made by the Data Safety Monitoring Board (DSBM) and will be based both on efficacy and safety data from half of the study population (n=96).
One thing you have to distinguish from Bronxville is the terminology, ‘Primary Completion’ and ‘Completion of Recruitment for Interims’. At the moment, you think it is interchangeable. Completion of Recruitment for Interims (96 patients) should have taken place at end Sept 2018. Primary Completion = Recruitment for whole study (192 patients). Therefore, Primary Completion date is being overly enthusiastic with a date of Dec 2018. I agree with Cookie that this is very likely a mistake on Amryts behalf, as it should be in summer 2019. Clinicaltrial.gov website probably was probably updated by an Amryt administrative staff.
Amryt announced on 26th Sept 2018 that they were expected to recruit the last patient for the EASE interim in late Sept. As Cookie mentioned, the primary endpoint is wound healing @ D45. Hence, that all data will be collected for Primary Endpoint by mid-November. Add another 4 weeks for inputting data, data crunching, stats analysis - that takes us to mid-December (+/- 2 weeks).
Hence, Boronxville- there will not be a delay in the release of the interims. For there to be a significant delay - there must have been something catastrophic that occurred that prevented them from recruiting the last single patient from their numerous global sites for the interims. Example, World War III, global flu epidemic, etc.
About using a single site in USA vs. multiple sites.
The FDA do not have any regulation with regards to the percentage of patients that come from USA. But for common diseases e.g. obesity, diabetes, cancer, it is common practise to include 10-15% of patient from USA, so that the sub-analysis of patients in USA is meaningful.
However, for Orphan Diseases such as EB, the FDA will likely be much less strict. It will be important to have sufficient patients in different racial categories e.g. Caucasian vs other races.
Amryt have likely chosen an easy path in for study start up in USA. By using a private site in USA, then can get the study up and running in the country very quickly. I personally would not be surprised if they added a few more sites in USA, after the release of EASE interims.
This is my analysis on your question on study timelines from Clinicaltrials.gov data:-
Firstly, I believe that the dates provided on clinicaltrials.gov are currently wrong (they probably represent the older timelines). If there are actually any Amryt staff reading these boards, please take note.
Primary completion date is the date at which all patients are observed for Primary Endpoint i.e. would healing at Day 45. At the present time, they have only recruited about half of patients for the study (as the interim is based on 50% patients). For completion of the study and recruitment of further 50% patients, it will take at least a further 6 months. Therefore, Primary completion date should be around April-May 2019, in my estimation. They will then follow up all patients for a further 2 years, therefore study completion date will be ~May 2021.
Bronxville - I actually did think of you positively in the past. You asked intelligent questions. I was happy to help you answer those questions. But constantly trawling the internet to find negative information on Amryt and then coming up your conspiracy theories, not backed up by true facts, is a different matter altogether.
To summarize what jimbobtechstock said about you a short while ago (please kindly do not misquote me again saying these are my words): "Derampers are biased by potential financial gain seeing the SP fall, period. They set a mood that suits their agenda".
The likely reason why they used the Private Clinic (run by Dr Browning) vs. UT Health Hospital is likely a contracts and start up time issue. Large hospital like UT Health, Santonio, is that contract negotiation would have been more complex.
Whereas, if you look at the thread of postings that everyone can see - nobody had mentioned your name after you said, “OK, I’ll tune out till Amryt post the interim results...”
There was only 1 posting after this - by Candid - after your posting. Therefore, unfortunately, you appear to contradict yourself again, by mentioning that insults had brought you out of 'retirement'.
The site in San Antonio, Texas, is run by Dr John Browning, Consultant Paediatric dermatologist and Key Opinion Leader in Epidemolysis Bullosa. He has published widely on EB and has been involved in other trials for EB. The site clearly has access to EB patients, and will be able to recruit the patients. Dr Browning is also Chief of Peadiatric Dermatology at UT Health, San Antonio.
Bronxville - if I recall correctly, you posted on 5 Nov 2018: “OK, I’ll tune out till Amryt post the interim results...” Interesting that you do not follow the contents of your own posting. Selective memory loss or early onset dementia?
Yes, Bronx, I am a shareholder, like jimbobtechstock.
And I happy and fully transparent to state, with my own analysis, that the probability of the upcoming EASE interims being positive is about 70%. I am not telling people to buy, buy, buy Amryt stock! There is certainly high risk involved, and buying Amryt shares is not for the faint hearted. But I am certainly not a PAID HACK.
Whereas for you....I can only echo what jimbobtechstock has stated... I know from my 10years+ trading AIM that no-one puts in the effort to deramp without having a position to make it worth their while. No-one.
Bronx... we are really tired of your highly biased ramblings.... example, your article related to Knowledge Ecology International which was published many months ago. Why don’t you mention that the same group have also flagged similar concerns about many other biotech and Pharma companies? Like Gilead, and their multi-billion dollar Hepatitis C drug? So, regarding your conspiracy that I know Mark Sumeray...I can state that I’ve never met the man.<br However, with your continuous negative postings.... the more likely conspiracy is that you have negativity towards Amryt/Aegerion Senior Management! Reasons... redundancy....insulted in the past...personal gain????<br /><br Hopefully, no one is being mislead by your stupidity!
Why should I do a history check on Mark, Bronx? Clearly, your statements have no substance, and are not based on knowledge, but are just based on fantasy or personal prerogatives! Why state that Episalvan is a non-curative treatment and imply that it is not worth developing? Imagine if your relative or even child had EB. Surely, you would want any form of treatment that could help treat the disease?
"He was responsible for the Lujuxta trials at Aegerion,got it thru with a big warning label and then imho misled the sales reps which almost bankrupt the company"
Sounds like you are terribly biased against Amryt Bronx. If what you have said above about Mark is true... then Amryt would not have employed him as CMO. Reputation is extremely important in the Pharma Industry
OK... last one Bronx... please....I don't want to spend my spare time explaining the role of each of Amryt's 35 employees!
Below is the roles/activities I would expect Dr Mark Sumeray to be performing as Chief Medical Officer (CMO), which is based on my personal experience and knowledge. Biotech companies are unique, therefore the role of CMO may differ from company to company, but here are a few key responsibilities:-
As CMO, Mark would be have the overall responsibility for the design and conduct of clinical trials, for the products in both in development and also marketed products.
For example, Mark would have overall responsibility for the design of the EASE study, the preclinical study designs for AP103 (results due in Q4 2018) and also any clinical trials which may be post-marketing requirements or commitments. For example, he would be responsible for the design of the paediatric study for Lojuxta, to support use in children.
Meanwhile, Mark would also be involved in direct interaction with Drug Regulators e.g. FDA and EMA, or other specialist regulatory bodies e.g. PDCO (EMA Paediatric Committee) for drug submissions or obtaining Regulatory feedback on clinical study designs. I expect that he will also be extremely busy in the coming months (following the EASE interims) working on the submission dossier with the Head of Regulatory - preparing for the FDA and EMA submission for Episalvan for EB. The submission dossier is a several hundred page document, summarising efficacy/safety of the product, including pertinent preclinical and clinical trial data.
Mark has also overall medical/scientific responsibility for Amryt products. For example, he would carry responsibility - together with Head of Pharmacovigilance - to assess the benefit/risk of Amryt products, and to make important medical decisions e.g. product withdrawals or even termination of products in clinical development. He would also be responsible for ensuring patient safety for patients using Amryt products in clinical trials or for marketed products. For marketed products such as Lojuxta, or drugs in clinical development (Episalvan) it is essential to provide regulators with updates on the risk/benefit profile of drugs, by submitting PBRERs/DSURs to regulators.
Mark would be involved in interaction with the media e.g. public disclosure of Study Results. For example, I would expect him to be the one disclosing the upcoming EASE results.
Clearly, his responsibility covers a wide range of areas including Strategic decision/planning (e.g. developing the Clinical Development Plan /Clinical Development Strategy for each drug product), overall responsibility for Risk/benefit of Amryt products, regulatory input, pharmacovigilance input, and media interactions. Therefore, other medics (e.g. EU Medical Director) represents an extra pair of hands to help him with the day-to-day running of clinical trials and to help monitor the safety of patients involved Amryt clinic