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Maverick - don't be a stranger.
Your thoughts are appreciated.
Protexia Alzheimer's proof of concept
On the milestones page they list "Protexia Alzheimer's proof of concept" as an event to be completed later this year. Anyone ever heard of using hBChE as an Alzheimer's therapy?
thanks,
vH
'belief' versus 'opinion'
Many people of science are uncomfortable using the term 'belief', and with good reason. Also, the term 'opinion' need not imply 'non-objectivity'... see the following definition (from http://www.sussex.ac.uk/languages/1-6-8-2-2.html ):
"Opinion, in academic terms, is the judgement or viewpoint reached after analysing, assessing and evaluating arguments, claims and evidence. Academic opinion is objective, like that of a judge who weighs the evidence (for and against) and judges each case on its merits. (Personal opinion which is based on beliefs or codes of ethics, rather than evidence, is not acceptable in academic terms as it cannot be tested in the same way.)"
Also, remember that Cox is British - so his choice of words is probably more reserved in general due to cultural influences. So he will say 'opinion' where GW Bush would say that you either believe GTC is the low cost provider or you're against us.
To be funny, one could write a thesis on the choice between those words, but my point is that it's ridiculous to put so much thought into the exact word that was used. This isn't a Bernanke testimony or FOMC minutes after all. Put down the tea leaves and pull out a calculator.
vH
Re: ATryn approval in US ... on a scale of 1 - 10,
One important difference is that US approval of ATryn (to which Dew attributes an 85% probability by end-of-2008 - #msg-11410659) will likely include approval for use in child birth.
What is the procedure for ammending the EU approval of ATryn in surgery to include child birth? Is this something that will be pursued once the additional data from the US study is available?
vH
ATryn US DIC study/ies
Dew, re. "U.S. ATryn time line" I believe the FDA trial would start in PIII.
Are you referring to studies for FDA approval in the DIC indication? What are people's thoughts? With data from the Phase II studies conducted by LEO in Europe, how much addtional work will the FDA require of GTC(+partner) in the US?
vH
Bio Investor conference
I just wanted to share some thoughts after listening to the presentation. There were no surprises. I do enjoy Cox's talks - it's an exciting time!
Cox specifically noted that they are following the current discussion (in the US congress) on FDA approval of follow-on biologics very closely. It is nice to see they would like to take advantage of any opportunity there.
In the Q&A after the talk, Cox mentioned the possibility of building a small (12-15 person) sales force internally that would be capable of handling US sales of ATryn for hereditary deficiency (while remaining open to the possibility of partnering with a large company for expansion into acquired deficiency markets such as DIC).
I am pleased with the careful and deliberate approach they are taking in building this company. Obviously, with so many exciting programs, one would like to move forward as quickly as possible, and it could be tempting to dilute the crap out of the stock to raise funds to do so. However, I am now fully of the impression that they have the goal of building a very significant company through organic growth, providing maximum return to current shareholders.
This is definitely an opportunity for the patient investor. Which is a polite way of saying that full-realization of the company's value could still take some time. Cox presented the timeline for likely news over the next two years (which Dew posted earlier). I think significant near-term upside in the stock price will require surprises on the news front. Possible surprises include:
1) better-than-expected efficacy observed in Merrimack's phase II rheumatoid arthritis studies for Alphafetoprotein, and a high profile partner for that program (I think the news on this will come in the June time-frame, if I remember right).
2) GTC partnering either the Alpha-1 Antitrypsin (rAAT) or CD137 programs, allowing those programs to progress to the clinic more quickly than expected.
3) GTC partnership on another external program (like the Merrimack partnership).
4) upside surprise in ATryn sales in Europe.
I listed these possible surprises in what I think is the order of likelihood.
Good luck to all. Remember, Cox said that their patents with the broadest allowed claims sat in the patent office for about 20 years before approval. We need to try to be at least 1/10th as patient as the folks who are building this company.
vH
Cell culture
What's the current state of GTC's capabilities in protein production with cultured goat cells. Can they quickly provide a partnering company with enough material to do pre-clinical studies while they develop the herd? I'm thinking especially of those partners concerned with studying the goat-glycosylated proteins from the earliest stages.
Thanks,
vH
Re: New forecasting tool could reduce drug development costs
Biowatch - thanks again. The NRDD article looks interesting. The Children's press release links to the website of the company these guys founded:
http://phorecaster.com/
vH
Re: Putting odds on FDA approval
Biowatch - thank you! How's that for timing!
Asher Schachter, MD of Children's Hospital in Boston was interviewed by Marketplace regarding their work in forecasting clinical program outcomes. They have two papers in press:
Schachter AD, and Ramoni RF. Clinical Forecasting in Drug Development. Nature Reviews Drug Discovery, in press.
Schachter AD, Ramoni RF, Baio G, Roberts TG, and Finkelstein SF. Economic evaluation of a Bayesian model to predict late phase success of new chemical entities. Value Health, in press.
I've asked Asher to send me copies of the papers. I'll let you know if that happens. Thank you once again for the information!
vH
Expert prediction of clinical program outcomes
Hello! I'm interested in hearing everyone's thoughts and starting a conversation to investigate the following question: To what extent can a clinical program's success or failure be predicted using knowledge of drug mode-of-action, published research, clinical endpoints, etc.etc. I've gone back through these boards and elsewhere after some high-profile misses (Nuvelo with Alfimeprase, Pfizer with Torcetrapib) and found some skepticism about those programs. I know I may be asking a biased crowd (I don't think we'd be here if we thought it was a crapshoot). Does anyone have insights they'd like to share about the process? What are the limitations? How do you maximize predictive power? Any tips for doing research?
What does it take to beat Joe Baggadonuts?
Re: quiz
Does it have anything to do with placing the 2nd and 3rd tranches in different calendar years?
Hey, You Got Something To Eat?
enjoy:
http://www.theonion.com/content/node/40091?issue=4228&special=2005
Thanks, Dew -
I certainly didn't intend to imply that the approval of Kineret was based on less robust clinical data. I was only trying to reconcile Amgen's statement in Q2 '99 that they would submit the application after phase II based on FDA guidance, and my memory that approval didn't happen until late 2001. I dug a little deeper - it turns out they did submit the application after phase II, but were rejected. They went on to complete the additional studies you described and resubmitted.
Anyway, this is all a big boondoggle motivated by my interest in understanding Merrimack's delay in reporting results. I am not assuming the worst like some.
Merrimack RA Phase II (from ClinicalTrials.gov)
I was looking back on the details of the study (listed as 'no longer recruiting', http://tinyurl.com/y7lrz6 ). It was a reasonably large Phase II study (260 patients) performed in almost 40 centers. The study monitored patients on one of 3 doses (2.5, 7.5 and 20 Mg) of MM-093, and did not include a placebo arm. I know this is wild, biased speculation - but I think there is some non-negligible probability that they could demonstrate clear efficacy in such a study. While a placebo arm was not included, patients were required to be on a stable regime of methotrexate and folate (so before and after comparisons are possible). Looking back, I found a reference ( http://tinyurl.com/yxlhzn ; Motley Fool) describing Amgen's intention (based on FDA guidance) to submit their application for IL-1ra (Kineret) following phase II studies of RA patients. Does anyone remember (before I dig too much further) - did they indeed submit after phase II? The Motley Fool referenced their intention to do so in Q2 1999, but I didn't think Kineret was approved until late 2001 (am I wrong on that?).
As an aside - does anyone know the half-life of MM-093 in serum and the proposed dosing frequency? If you do know a number, please indicate if the half-life is for a native AFP isoform or MM-093. I haven't searched the literature yet, but I found a reference in the clinicaltrial.gov summary that one of the exclusion criteria was women planning to become pregnant within 4 weeks after the last study dose.
Thanks for your help.
VH
Probability of Merrimack filing after Phase II
Does anyone assign any real chance of Merrimack bypassing phase III? Just trying to make sense of the delay in announcing results...
Simply - LFB is French - they're lovers, not fighters! <eom>
Cash position help in Merrimack negotiations?
I may be reaching here - but is there any immediate benefit from the improved cash position that they might realize in their negotiations with Merrimack? What terms need to be finalized as Merrimack goes into Phase III?
Thanks.
Additional comment based on CC:
While this may not be too substantial - based on my listening of Cox's comments during the CC, it sounds like there might be a small slip in the ATryn hereditary deficiency US FDA BLA timeline. Cox guided for completion of patient enrollment in H1 2007 (previous Q1 2007 ?) and submission of the BLA in H2 2007 (previously Q3 2007 ?). Let me know if I'm mistaken on this point. He made a comment about patient recruitment being difficult (which I'm sure is true for HD) - so I think my reading of the situation is correct.
I do want to say that I'm extremely excited about the possibilities for GTC - and enjoy seeing them expand the number of products in the pipeline. I will be watching the relative emphasis on internal versus external programs very carefully. I originally became interested in the company because of the albumin program and the possibility of simultanesouly developing several external programs for monoclonal antibody production - and these possibiilties seem to have taken a back seat in the unfolding vision for the new GTC. What are other people's thoughts on the future of Merrimack-style deals for GTC?
Thoughts based on CC:
From Cox's early comments in the CC, it sounds like the alpha-1 anti-trypsin program is going to be rolled into this collaboration with LFB. He briefly mentioned that the direction of that program (product development and commercialization) will be decided by a joint (GTC/LFB) steering committee yet to be constructed. I would be interested to learn more about this decision. More generally - of the four pillars of the new GTCB that Cox described (ATryn w Leo, broad recombinant plasma protein and mAB partnership with LFB, external programs i.e. Merrimack, and internal independent programs) - I would like to know more about the amount of attention to be given to each pillar.
Does someone have time to look into the global population distribution of genetic mutations that cause hemophilia (Factor VIII for hemophilia A and Factor IX for hemophilia B). I'm curious about any nonlinear benefit arising from the expansion into other markets that might be enabled by more efficient production. That said, I'm assuming that these other markets would be less lucrative than expansion within the 1st world of the use of Factor VIIa in other indications (I was happy to hear about the agressive off-label use of Novo's rFVIIa - thanks, urche, for post #920).
As an aside - it's pretty clear that Navdeep Jaikaria from Rodman & Renshaw doesn't understand/remember the terms of the LEO partnership agreement - in regards to the sharing of clinical data. I encourage the members of this community to participate in more of these calls so that we can get answers to meaningful questions (thanks for your participation on the call, urche!).
Re: Coag. and Sepsis paper request
Dew -
I can get that paper. I don't have the premium membership - so I couldn't do a private reply. If you respond with your contact information, I will send you the files.
Re: Option backdating graphic
Wow - thanks for the link. Nothing like the power of properly visualized data! That is a beautifully disturbing chart.
GTCB: Does approval change short term burn?
Hey - just wondering what people's thoughts are on GTC's likely strategy going forward and the associated capital requirements? Guidance prior to approval was that current available funds will keep the juice flowing through the year. Any chance that the CHMP ruling will affect strategy in a way that will increase burn? I assume LEO is handling costs associated with market launch in Europe. Any chance they'll decide to accelerate other programs and need to raise money to do so (including accelerating the ATryn program in Japan)?
Thanks for your thoughts,
Mike.
I agree with 10nis. On a risk-adjusted basis, this is the cheapest price I've seen in a long time. In retrospect, I probably should have kept this issue on the watchlist until today and saved myself years of indigestion.
Mike.