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Looking at the upcoming RBC Capital Markets conference looks like the format is all panel type discussions. Good to see something different then other Conferences. Don't get me wrong I like to see presentations of companies I follow but for those that attend/listen to all the conferences of a particular company the scripted presentations offer little new insights. This seems to have some potential for more insights. I also like when there aren't scripted presentations and its more Q&A (particularly when the analysts asking questions delve deeper then what would be in a scripted presentation).
Anyway thought I would list to the panel discussions listed on the website:
Navigating New Developments in Neurology
Close To The Heart: New Technology Solutions
New Targets For Lipid Control
G.I.
New Agents In Cardiovascular Disease
Dx: What Your Central Lab Can Do For You
Cancer Antibodies: Developing A Pipeline
Clear Skin, Fat Wallets: Aesthetic Medicine
Hot New Targets In Cancer
Away From The Heart: Expanding The Role Of Interventional Medicine
Skin Deep: The Future Of Clinical Dermatology
Motion & Fusion - The Future of Spinal Surgery
New Therapies In Oncology
Lifestyle Choices: Elective Procedures
Delivering Profits From New Formulations
Tissue, Blood & Bone: Advanced Implants & Surgical Tools
Enhancing Cancer Therapy: Safer & Better
The Cath Lab
Unique Commercial Strategies
Pipelines & Partners: Building Through Acquisition & Licensing
Better Chemistry = Better Drugs
All This Technology Going To Our Heads? Neurology/Neurointerventions
House Of Pain Relief
Cancer Horoscope: Future Treatments
Anti-Infective: Bugs Beware
Eyeing The Future Of AMD
Satisfying The Search For Sleep
Image Is Everything
New Antibody Technology
Breathing Easier: Innovative Approaches To Asthma
Cutting The Fat: New Approaches For Obesity
Dx: From Discovery to Point-of-Care
Webcast Calendar
[Please see updating procedure at the end of this post. Events listed here are regular quarterly conference calls unless indicated otherwise. All times are U.S. ET. unless indicated otherwise.]
NOTE: ANYONE MAY UPDATE THIS FILE
Edits: Added RBC Capital Markets Healthcare Conference
BMO Capital Markets Focus on Healthcare Conference
December 6-7
New York, NY
http://www.bmocm.com/conferences/healthcare2006/webcast/default.aspx
#msg-15138298
RBC Capital Markets Healthcare Conference
December 13-14, 2006
The Westin Times Square, New York
http://www.wsw.com/webcast/rbc62/
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Scientific-Meeting Calendar
[Please see updating procedure at the end of this post.]
NOTE: ANYONE MAY UPDATE THIS FILE
Edits: Add ATS 2007, AHA 2007
American Society of Nephrology, Renal Week
Nov 14 - 19, 2006
San Diego
(SCCM) Society of Critical Care Medicine
February 17-21, 2007
Orlando, Florida
(ASH) American Society of Hematology
48th Annual Meeting and Exposition
Dec 9 - 12, 2006
Orlando, FL
28th Annual San Antonio Breast Cancer Symposium
Dec 14 - 17, 2006
San Antonio, TX
(ACC) American Collage of Cardiology
March 24-27, 2007
New Orleans
(ATS) American Thoracic Society 2007 International Conference
May 18-23, 2007
San Francisco, California
http://www.thoracic.org/sections/meetings-and-courses/international-conference/2007/index.html
(AHA) American Heart Association, 2007 Scientific Sessions
November 4-7, 2007
Orlando, Florida
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Thanks for that (sorry I should have known by the MD ending you were speaking from medical profession).
I thought I read it is not recommended for women wanting to get pregnant are there risks associated with it?
Wow 20k! Out of curiosity did you ever look at the BSMD procedure they have an embolization procedure that seems to have been on the market for a while. Curious to know the opinion of a potential patient. TIA
Dewophile,
Thanks for the info. From what I saw around 40 location and a bit over 2000 people treated in 2 years doesn't seem like patients are demanding it.
Sorry about size being the main factor for seeking treatment was my speculating. One of the papers did allude to location being a factor in the efficacy of this treatment (not sure if it is one that I included).
I guess you know of no good pill (or even an injection) in development for this? I'd really be interested in knowing of anything showing promise in the clinic.
OK Dew I took the challenge rather then file it in my nice to do "one day" list. I’ll caveat this with its 4-6AM and I don’t do my best thinking then and am on the tired (and lazy) side to clean this up but here are some notes.
My (early) conclusion is this has some improvements over surgical options but I don’t see it as gaining wide adoption. It has some efficacy but leaves a lot of the fibroids (both treated and the ones that aren’t targeted) and does not seem to do well against larger fibroids (which are probably the patients most likely to seek treatment sooner). There is an article (link below) which gave some optimism for improving outcome on larger fibroids. Not clear to me but seems there is some subset of patients (not sure how large) where this procedure will not work because of various reasons (see feasibility Study below). I would also speculate this requires pretty specialized centers and the more highly qualified the doc (or technician/specialist) the better the outcome.
Company Website http://www.insightec.com/
ExAblate® 2000 http://www.insightec.com/447-2278-en-r10/ExAblate-2000.aspx
MR Imaging–guided Focused Ultrasound Surgery of Uterine Leiomyomas: A Feasibility Study[\b]
Good Journal Article (Early study of patients who had the treatment and then a hysterectomy they were going to have one and agreed to this treatment[\i]):
http://radiology.rsnajnls.org/cgi/content/full/226/3/897?maxtoshow=&HITS=10&hits=10&RESU...
Positives
Company Listed Benefits:
* Non-invasive outpatient procedure.
* No hospitalization
* Next day return to normal activity
* Limited conscious sedation.
* No ionizing radiation
* Low rate of complications
Well tolerated:
http://www.sciencedirect.com/science?_ob=ArticleURL&_udi=B6W9P-493NYSR-C&_coverDate=07%2F31%...
Potential to improve:
Pretreatment of leiomyomata with gonadotrophin-releasing hormone (GnRH)
http://www.greenjournal.org/cgi/content/abstract/108/1/49
Seems to have better outcome treated 49 women with leiomyomata in excess of 10 cm 3-month course of GnRH agonists followed by the ultrasound.
Results: “45% reduction in median symptom severity score at 6 months and 48% at 12 months posttreatment, with 83% of women achieving at least a 10-point reduction in symptom scoring at 6 months and 89% at 12 months (P < .001). There was an average reduction in target leiomyoma volume of 21% overall at 6 months (P < .001) and 37% at 12 months (P < .001). No serious infective complications or emergency operative interventions were recorded.”
Negatives
Limited Availability: http://www.insightec.com/146-en-r10/Fibroid-TreatmentLocations.aspx
Limited Adoption: (2200 patients since approval through November 2006) http://www.insightec.com/15-2434-en-r10/NewsInsightec.aspx
Limited to smaller sized fibroids:
http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&dopt=AbstractPlus&l...
Limited reduction in fibroid size:
http://www.ajronline.org/cgi/content/abstract/183/6/1713
Heck, even the President of the United States plays this game. Do you remember Nixon’s Saturday Night Massacre?
I was a little too young to remember that. I just remember Nixon from TV... sitting in the stands at Mets games of course :).
So since I didn't listen to the Pfizer Analyst webcast yet will Pfizer do a favor to those like me and edit that portion out for us :). I guess with everyone up at 4AM on this board I can't accuse them of trying to hide it here anyway.
PFE may need to buy or partner other companies to get more in the pipeline.
We must be on the same wavelength lately (didn't see your post till after I posted, really!)
I guess I was naive but I thought only smaller company's hid their bad news on odd times :).
Does this mean we can all go back to speculating on who/what Pfizer will buy next?
Thanks had never come across this. Will have to try to find more about it wonder how good long term success is and how widely accepted it is.
If you can look at the chemical structure and determine in advance whether it will fail or not, you could make a fortune by advising chemical companies
I was thinking that very thing! Well up to the "make a fortune" part :).
Uterine Fibroids
poorgradstudent / rfj1862 / daved / dewophile (think I got everyone). Appreciate the rprx discussion.
I'll admit to being naive about this a couple years ago then personal and investments have led me to slowly become more aware of how big an unmet medical need this is. I was very surprised by the prevalence and lack of (non-invasive and effective) treatment options! It doesn't seem like hormone therapies are the answer.
I was curious to any other treatment options (particularly drugs) with potential good efficacy and what others thought of them.
Here is a link to active studies from the clinical trials website:
http://tinyurl.com/ybw4dr
The Pirfenidone study by the NIH is one I was aware of but I have not inquired to InterMune if this is considered a fibrotic indication covered by their license agreement (from Marnac). From what I've read of Pirfenidone in IPF I would speculate (I don't claim to be a science guy) while it may have some potential I would not see this end-all treatment option.
Appreciate anyones thoughts on the subject. TIA
My guess is the Braves will make a lower 1 year offer with an option for a second in the next couple days (didn't want to give up that draft pick).
Webcast Calendar
[Please see updating procedure at the end of this post. Events listed here are regular quarterly conference calls unless indicated otherwise. All times are U.S. ET. unless indicated otherwise.]
NOTE: ANYONE MAY UPDATE THIS FILE
Edits: Links for Piper Jaffray and BMO Capital Markets Healthcare Conferences
BMO Capital Markets Focus on Healthcare Conference
December 6-7
New York, NY
http://www.bmocm.com/conferences/healthcare2006/webcast/default.aspx
Lazard conference
Nov 28-29, New York:
#msg-14718610
Piper Jaffray 18th Annual Healthcare Conference
Nov. 29–Dec. 1, 2006
New York, New York
http://phx.corporate-ir.net/phoenix.zhtml?c=128690&p=conferenceAgenda&id=1173562&day=1
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ANDS 11.29 3pm (Lazard); 12.01 2pm (PJ)
DVAX 11.29 9am (HBV vaccine results)
EXEL 12.04 (R&D Day)
GILD 11.29 11:30 am (Lazard); 11.30 1:30 pm (PJ)
GTCB 11.29 1:30 pm (Lazard)
HGSI 11.29 2pm (Lazard)
PFE 11.30 10am-4pm (R&D Day)
SGMO 12.01 1:00pm (PJ)
VRTX 11.29 1:30 pm (PJ)
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For the scientist who has everything...
http://www.brain-map.org/
Just saw a story on this on PBS. Interesting and free! I am not that into science but if you want to see the make-up of the mouse brain this is the place to go :)
Didn't see the RNAi USA Today story posted. Thought it would be here while more of a human interest piece (story on the ups-downs of a biotech).
Susquehanna also started coverage of VRTX with Negative rating, FWIW.
FWIW they started coverage on ITMN with a Positive.
I don't know who the analyst is that does their coverage. Usually by the questions you can tell the better analysts from the ones just trying to get the companies to do their models for them. There are some on companies I follow that I put almost no value on their ratings/comments.
As a fan and not an owner I would be thrilled by my team spending whatever is necessary to win :).
I can live with going to minor league games and watching my team on TV or following on radio/internet.
I'll miss the entertaining conference calls especially when the FDA goes against him.
Boy they really hid that to the end of the new release.
I guess that is what I get for sleeping!
Not a surprise at all that Actelion would do a deal, nor CoTherix. If I am not mistaken Schering AG has x-US Ventavis (Ilhoprost) rights and also has a right to match any bid for CoTherix (if it is a competitor). I guess Actelion doesn't qualify [don't know much about SAG]. I thought something they (Actelion) could market world-wide in PAH would be a better fit. Unless they see a lot of value in the rho-kinase inhibitor that CoTherix recently inlicensed.
With GILD buying MYOG and now this the PAH field is getting less crowded. I am still looking for United to do something at least x-US with their Inhaled/Oral if not also their sub-q and IV.
PS
I like the quizes though I often don't have a clue. Don't mind the aesthetic medicine posts (don't always read them) as follow MRX a little bit.
rfj,
THanks for the reply and comments. I finally heard all of the Analyst event. They are a bit earlier stage then I would like. But I like to keep an eye on these as sometimes you can get them near cash. They'll need a lot more cash too (unless they partner) so I am leary without having a good idea on future share count (I learned my lesson here and that is one reason I agree with those who would avoid DNDN).
One thing I liked is the potential for combo therapy (with I believe all of their compounds). I got the impression the organic arsenic is much safer, I'll have to recheck. Also the ifosfamide compound seemed interesting I have to see besides ICE what other chemo treatments its in. I believe they are working on preclinical oral formulations for the other compounds. Another thing is they mentioned having backup compounds of at least 101.
Thanks again for the comments.
Anyone follow ZioPharm or know much about the potential for a "safe" Arsenic (Trisenox)?
They are an (early stage) Oncology company with a few compounds. An organic arsenic that is supposedly less toxic is one of the more developed compounds.
They have an Analyst Investor Day today (on going)
http://www.wsw.com/webcast/ziopharm2/
am just catching the tail end of it.
Have been noticing them at a lot of Investor conferences lately and was curious if they are worth a further look. TIA
f VEC-162 makes it to market, I will definitely try it.
Why not try to get in the trial they are doing next year? :)
>I recall poorgradstudent had some thoughts on it<
PGS thinks OvaRex in particular and REX in general are losers.
Don't recall the exact wording of "losers" but I do recall him not liking it. If memory serves a couple of his points where on track but not 100% so.
One thing is PFS time needs to take into account that OvaRex trials often give time AFTER debulking and chemo so one needs to be careful with that. There are some things from the United Investor/Analyst presentation to consider too.
On the negative I think of more concern is the MarkMan paper on CA-125 level indicative of PFS (though the analysis of the paper is in trials with maintenance chemo) and the fact that the OvaRex Phase 2B was powered for a much higher Placebo then observed.
Do you mean a specific disease indication such as HCV or diabetes or AMD? If so, that’s a fine idea. All we need is some dedicated people who will expend the effort to compile the contents
Actually I tried to be deliberately vague :). I like both ideas. To know the competitive landscape of a disease would be nice. I don't follow HCV anywhere near some do on this board and it would save me a lot of effort :). I was going to do more research on OvaRex for my own purposes. I am not sold on it BTW, UTHR is first and foremost a PAH company but I wouldn't right off their mab program just yet and had that as one possibility.
I recall poorgradstudent had some thoughts on it and seems to know a lot (more then I) on mab's in general. I think there are some aspects the casual observerer may have overlooked when judging it. Thought maybe I could talk him/others into a deeper look if I presented some info.
I guess I could do a UTHR write up but time and it appears no one follows it here.
Dew,
Thanks for updating the Readme link (you are quick!) In the future should I post to let you or whoever else know it is an update (I don't have PM)?
The Pirfenidone post is a lot of opinion don't know if you want to minimize (was just going to post it on another board). If so let me know and I will refrain. If you don't mind it I would enjoy seeing debates on specific therapies. Any chance of a "ReadMe" first for a therapy?
If you have pull with Ihub the Rich-text editor on another board which I will keep nameless makes for a very nice formating of text (and tables) any chance of using your pull and getting that capability here :)
Some more thoughts on Pirfenidone
I know everyone is eagerly awaiting news on 191 (me too I wish it was someone other then the French, I haven’t had too good of success with them in past investments but maybe the focus on Pirfenidone should be moving up a bit more in peoples minds. I have been reviewing some of the calls and journal articles and have some thoughts….
On Enrollment
1. I am thinking there is a good chance of beating even my mid year early enrollment estimate. Here is the thinking… The Shionogi Phase 2 http://ajrccm.atsjournals.org/cgi/content/full/171/9/1040 enrolled 105 patients between November 2000 and January 2001 that with only 25 sights. That would be more then 1.4 patients per sight per month (given that the time is < 3 months unless enrollment started 11/1 and ended 1/31) and probably the 25 sights didn’t all start recruiting on day 1.
2. If the Capacity trials sight is up-to-date and accurate http://www.capacitytrials.com/wt/page/index we have 60 NA sights accepting patients and 14 still awaiting and there are 28 sights outside NA accepting and 20 awaiting. Granted Japan has very good healthcare system so just to be conservative making a rough guess at how the sights may have ramped along with using 1 patient/sight/month (much less then Shionogi Phase 2) month by month could be…
Month Est. # Sites Patients Enrolled In Month Total Enrollment
May ‘06 15 15 15
June ‘06 30 30 45
July ‘06 45 45 90
August ‘06 60 60 150
September ‘06 75 75 225
November ‘06 90 90 315
December ‘06 100 100 415
January ‘07 100 100 515
February ‘07 100 100 615
*Trials started enrolling patients April 27 with expected enrollment of 585 (325 in 3-arm with up to 70 centers and 260 in single dose vs. placebo)
3. As early as the June Needham call the company referred to Investigator interest being very high and patient interest being high (at first I took this as normal PR but now I think it could be enrollment). A month later at the Jefferies conference InterMune said (paraphrased) Investigator interest very, very high and wouldn’t be surprised if beat that enrollment. Though already optimistic. Speaking to excitement shooting to enroll sooner. OK, one more reference in the October CE Unterberg call perhaps a Freudian slip but said CAPACITY should finish enrolling by end of next QUARTER (I think he meant to say next year)
I don’t think enrolling at a fast rate is material information to disclose and probably since the Shionogi results are just around the corner its in InterMune’s interest to wait till then just in case they decide to alter the study (e.g. they decide to dramatically increase enrollment for the power but can then claim interest is extremely high having already enrolled over X amount so we can do it in the same time frame).
On The CAPACITY Trials
1. The Shionogi Phase 2 was stopped by the DSMB early (6 months stopped but data collected for 9 months) due to acute exacerbation in one arm which turned out to be the Placebo arm. OK, many were critical of the trial stopping early and also argued that the 6 and 9 month data were not adequate.
2. Less known is the fact that an NIH study of Pirfenidone in patients with Pulmonary Fibrosis resulting from Hermansky-Pudlak Syndrome (a very rare genetic disease one type has a high incidence in Puerto Rico) was also stopped early for (positive) efficacy
3. The CAPACITY trials have an arm with HIGHER dose (2400 mg as 3 800mg doses) then the Shionogi Phase 2 (1800mg max dosage as 3 600mg doses -- I don’t know the Phase 3 dose but would think it is comparable to the Phase 2). The 800mg dose has been tested before in other Pirfenidone trials. While Pirfenidone does have some side effects (most notably a high incidence of photosensitivity) compared to other treatment the number discontinuing treatment was very low. One would speculate that a higher dose should be at least as efficacious as the Shionogi dose.
Am I saying that the capacity trial could be stopped early??? Yes and No, it is a long shot but there is DSMB monitoring and if one arm is having serious adverse events in a meaningful way it seems ethical to do so. The main thing I am concluding is that with much more rapid enrollment the time frame for outcome/potential to market is much closer then announced even without DSMB stopping.
With the Roche cash and funding what I am wondering more about is why partner Pirfenidone? If it fails (Shionogi Phase 3 results poor) then no one will give you anything anyway. If the results are successful I think the uptake of this drug will be extremely rapid judging not only by unmet medical need, but also what appears to be very high patient interest and rapid trial enrollment. So unless the company does not see premium pricing potential (20K+/yr) then it makes a lot of sense (too me) to set up EU commercial operations with a small (50-100) targeted sales force. What will be the cost 10-20 million for the sales force and even if double that for infrastructure and marketing it will be recouped quickly! I think Encysive with Thelin (though we don’t know yet how they are doing) and BioMarin with Naglazyme are very good recent examples
Updates: IPF competitive landscape (Ventavis failed to show efficacy in IPF associated with PAH, Sildenafil trial), Roche transaction more info, Links for some Journal articles added and misc. cleanup
InterMune http://www.intermune.com/
Focus on Pulmonology and Hepatology. In Pulmonology focused on Idiopathic Pulmonary Fibrosis (IPF) and in Hepatology Protease Inhibitor recent collaboration with Roche and an undisclosed preclinical compound (in collaboration with Array).
IPF
• Scarring of lungs, unknown cause
• Median survival from diagnosis 2-5 years
• US Prevalence 128,000, Incidence 48,000 each year http://tinyurl.com/u6kgo, journal abstract http://tinyurl.com/tqd6m (old prevalence incidence 83K US and 30K incidence; In 2000 it was believed prevalence was 50K US).
• No approved therapy steroid and immune suppressants used with little effect. Drugs used include: Imuran (Azathioprine), Cellcept (Mycophenolate mofetil), Rheumatrex (Methotrexate), Mucomyst/Parvolex (N-acetylcysteine [NAC]) and Prednisone along with off-label use of Actimmune. Other off-label use include: Remicade (Infliximab), Enbrel (Etanercept) and Gleevec (Imatinib).
• Actimmune http://www.actimmune.com/
• Interferon Gamma 1B naturally occurring protein administered by sub-q injection (200mcg, 3x week for IPF patients in protocol for INSPIRE study)
• IP Protection (http://tinyurl.com/yawz9q) announced today that it has been issued two composition of matter patents that together cover the manufacture, use and sale of Actimmune(R) (interferon gamma-1b) in the United States. These patents, USPN 6,936,694 and USPN 6,936,695, expire in 2022 and extend a portfolio of intellectual property rights relating to Actimmune(R), which includes another composition of matter patent that expires in 2014.
• Investigational Studies being conducted in Idiopathic Pulmonary Fibrosis. Had failed prior phase 3 trial with composite endpoint and subject to controversial debate on benefits of interferon gamma results and letter in NEJM. INSPIRE Trial is current Phase 3 trial aimed at showing survival benefit in IPF patients. January 8, 2005 NEJM “A Placebo-Controlled Trial of Interferon Gamma-1b in Patients with Idiopathic Pulmonary Fibrosis” http://content.nejm.org/cgi/content/full/350/2/125. October 21, 1999 NEJM “A Preliminary Study of Long-Term Treatment with Interferon Gamma-1b and Low-Dose Prednisolone in Patients with Idiopathic Pulmonary Fibrosis” http://content.nejm.org/cgi/content/full/341/17/1264 criticism of the study http://content.nejm.org/cgi/content/extract/342/13/974
• Approved usage (Past 10-K’s) ACTIMMUNE has proven to be safe for patients since its approval in 1990 for the treatment of chronic granulomatous disease. There are an estimated 400 patients with chronic granulomatous disease in the United States, and there is no FDA-approved treatment for these patients other than ACTIMMUNE. Based on the indicated dosage levels, the annual cost per patient is approximately $25,000. There are approximately 400 patients with osteopetrosis in the United States. The FDA approved Actimmune for the treatment of this disease in February 2002. Based on expected dosing levels for osteopetrosis patients, we expect the annual cost per patient would be approximately $25,000.
• Method of Action (Company presentations) Immunomodulatory, Anti-fibrotic, Anti-infective – Antiviral, Anti-proliferative
• Pricing (Company presentations) based on dosing in INSPIRE for fully compliant IPF patient would equate to $60,000/patient for year of therapy. Medicare Part D would be operative because 70 percent of patients are over 65.
• Partner (http://tinyurl.com/y4m3gz) outside US, Canada and Japan, Boehringer Ingelheim with InterMune option to promote where BI does not.
• Patient Population (Company presentations) When numbers were reported as 83K prevalence and 30K incidence in US company estimated that 50K would be mild to moderate and 80% of newly diagnosed patients would fall into that category. This is significant because the “mild-to-moderate” is what the company is targeting in the INSPIRE study.
• Prior Studies supporting INSPIRE GIPF-0011 (NEJM, Jan 2004) 330 ITT, follow-up 1.4 years Deaths in Actimmune 16/162 (9.9%), Deaths in Control 28/168 (16.7%), relative reduction 41% p=.08. Ziesche2 (CHEST, Nov 2002), 18 ITT, 5 years follow-up, Deaths in Actimmune 2/9 (22%), Deaths in Control 7/9 (77.8%), relative reduction 71% p=.01. Antoniou3 (ERJ, April 2006), 50 ITT, follow-up ~2 years, Deaths in Actimmune 5/31, Deaths in Control 7/18 (38.8%), relative reduction 70% p=.028.
INSPIRE Trial http://www.inspiretrial.com/
http://www.clinicaltrials.gov/ct/show/NCT00075998
• Trial Randomized, Double-Blind, Placebo-Controlled, 2:1 Randomization – Actimmune: Placebo
• Administration Subcutaneous 3x week injections, 200 micrograms
• ~81 Sites – U.S., Canada and Europe
• Primary Endpoint Survival Time
o Greater then 90% power to detect 50% difference of mortality over 3 years
o Greater then 80% power to detect 40% difference in mortality over 3 years
• Patients – Mild to Moderate IPF (forced vital capacity ≥ 55% and carbon monoxide diffusing capacity ≥ 35%)
• Treatment Period 2 Years after randomization of 600th patient. 600th patient enrolled in November 2005. Completed enrollment April 2006 (826 patients total). Rational for increasing enrollment http://tinyurl.com/y53xcb
• INSPIRE Trial Update (Company Presentations, Q3 2006 Call recent update). 2 Interim efficacy analysis in protocol (not clear if any have yet occurred). When asked about “drop-out” company did not specifically address but stated extremely pleased with “study conduct” which includes dropout, staying on medication, etc.
Pirfenidone
• Administration Orally active small molecule 3x day pill of either 267 mg capsules (CAPACITY 1) or 267 mg or 133 mg (CAPACITY 2)
• Acquired NA and EU rights from Marnac, http://www.marnac.com/ for Fibrotic Indications. (PR http://tinyurl.com/yy2qao and 10K’s) In March 2002, we licensed from Marnac, Inc., a privately held biopharmaceutical company, and its co-licensor, KDL GmbH, their worldwide rights, excluding Japan, Korea and Taiwan, to develop and commercialize pirfenidone for all fibrotic diseases, including pulmonary, liver and renal fibrosis. Under the terms of the agreement, we received an exclusive license from Marnac and KDL in exchange for an up-front cash payment of $18.8 million and future milestone and royalty payments.
• Method Of Action (Company presentations) Preferentially binds to and disables the kinase p38-gamma. Significantly inhibits TGF-beta synthesis (fibrosis), Also inhibits TNF-alpha synthesis (inflammation)
• IP Protection (Conference call) Older compound patent from Marnac licensed covering anti-fibrotic uses 2011 expires. Basis of exclusivity more on Orphan drug. Granted in US (7 years) and Europe (10 years) after approval.
• Other Indications being studied in a number of trials (not all by InterMune). InterMune is seeking approval in Idiopathic Pulmonary Fibrosis. It is also conceivable that they later seek a broader approval for Fibrotic disease of the lung. Most notably an NIH study in Hermansky Pudlak Syndrome.
• Shionogi Phase 2 Results PR http://tinyurl.com/y5nwel Stopped after 6 months (efficacy). Treatment of 9 month. Observation/ Placebo/ Pirfenidone: Improved Minimal O2 Oximetry/6.1%/24.2% Declined Saturation/33.3/18.2% P=.016 Vital Capacity Improved/0%/9% Vital Capacity Declined/36.4%/13.4% P=.003
• InterMune Phase 2 Results PR http://tinyurl.com/y49zp7
• Journal Articles http://ajrccm.atsjournals.org/cgi/content/full/171/9/1040 (Shionogi Phase 2), http://ajrccm.atsjournals.org/cgi/content/full/159/4/1061
CAPACITY Trials, http://www.capacitytrials.com/
http://www.clinicaltrials.gov/ct/show/NCT00287729
http://www.clinicaltrials.gov/ct/show/NCT00287716 - Three Arm Study
• Primary endpoint is change in forced vital capacity (FVC) after 60 weeks of treatment
• Two concurrent, multi-national trials CAPACITY 1 and CAPACITY 2
• Approximately 580 patients
• Randomized first patient April 27, 2006
• Expect enrollment to conclude around the end of 2007, with top-line data expected in early 2009
• Exploring European partnering opportunities
• CAPACITY Trials Update (Company Presentations, Q3 2006 Call recent update). Enrollment proceeding at a brisk pace. Hinted could complete before end ’07 target.
InterMune 191 / PI Program
• Collaboration began with Array BioPharma http://tinyurl.com/y75d42
• Royalties due Described as “mid-single digits”. Array describes their royalties as 3% (generally speaking about royalty deals they have in past not just 191). As pointed out by rkrw (SI) and Dew other 3rd party royalties Chiron/others (~2%?)
• 191 General Characteristics[\b]
1. Active Sight NS3/4 PI
2. High affinity for and stability in the liver
3. Favorable pharmacokinetic profile potential Q12/BID dosing.
4. Good safety margins for: Body weights, organ weights, Clinical chemistry, clotting parameters and hematology, Renal, neurologic, gastrointestinal, immune and cardiovascular systems
5. Potency of ITMN compound is maintained against mutations that show resistance to other protease inhibitors
6. HCV protease selectivity is high
7. There were no significant effects on microscopic histopathology on any organ or tissue, including heart and liver
• 191 more specifics[\b]
1. Replicon Potency (genotype 1) EC-50 1.6nM, EC-90 14.1nM, EC-99 155.3, EC-999 1567.3 nM
2. 28 day preclinical tox suggests safe over wide dose range
3. Mutants D168A primary driver of 191 sensitivity. Hypersensitive mutation to Pegasys shift by about order may suggest suggests combo will result in protection against mutation.
4. Rat studies (looking at livers) various dose concentrations including 16 hours above EC 99.9 (3 log drop) 30 mg/kg equiv about 300mg in human to get similar human concentration.
• Some comparisons to VX-950/other PI’s[\b]
1. HCV replicon EC50 = ~2nM (VX-950=402nM, SCH-503034=200nM)
2. Mutations resistant to other PI primarily A156T 191 is highly active against mutation indicating complimentary profile with other PI’s in development
• 9/26/06 CTA submitted to the French Medicinal and Biological Products Evaluation Directorate http://tinyurl.com/yevhgh
• For technically oriented suggest listening to Think Equity “Think Tank” presentation by Dr. Blatt on 11/10/06 most technical info in company presentation to date.
Roche Collaboration http://tinyurl.com/y2e87x
• 60 million on closing, assuming successful develop in US and other countries will receive up to 470 million in milestones. 530 million total. Potentially 35 million (of 470 million) in next 12 months.
• Cost sharing – Roche responsible for 67% global development costs including all related mfg costs.
• Royalties outside US Roche commercializes all products from collaboration InterMune Royalties “mid-high teens %” depending on net sales level
• In US commercialize and share profits on 50/50 basis.
• Same economic terms would cover any 2nd generation PI’s developed and commercialized, roughly doubling value. Note this was clarified to mean excluding the up-front payment.
• Roche responsible for World-wide formulation development and manufacturing any compound.
• InterMune has 3rd party royalty obligations that total in mid single digits. Split 50/50 with Roche in US, InterMune responsible for them outside US
• Development self funding through development. Beyond significant post development milestones.
• Can opt out of either co-development or com-commercialization. If do so would receive higher x-US royalties and same royalties replace profit share in US.
• Expect to close pending Hart-Scott-Rodino around mid November
Other Pipeline/Interests
• Early stage preclinical program partnered with Array (undisclosed indication in Hepatology). Royalty described by Array as high single digits. Array Responsible for creating clinical trial and all synthetic process. InterMune take into clinical development through commercialization.
• Equity interest in Targanta Therapeutics, http://www.targanta.com/ as a result of selling Oritavancin in December 2005. (http://tinyurl.com/yznhod)
• Early stage work on PEGylated version of Actimmune (with Maxygen), does not appear to have much effort as this time. (http://tinyurl.com/yn4e4z)
• PEG-Alfacon (PEGylated version of Infergen) appears unlikely to be further developed.
• Research work in both Hepatology and Pulmonology
Financials
• Cash 169.2 million (end of Q3 2006)
• 170 million convertible. Coupon rate .25%. 2011 maturity. Conversion Price $21.63.
• 33 million shares outstanding
• 2006 Guidance
o Revenue 85– 95 million.
o COGS 21-23%.
o R&D 90-105 million (Does not factor any savings by Roche cost share, if closes in mid November). Including 5-10 million for est. FAS 123R.
o SG&A 30-45 million including 5-10 million for FAS 123R (not including 30 million for settlement).
• Government settlement for past Actimmune promotion (http://tinyurl.com/y3wa8a) . The company will pay $36.9 million plus 5% interest over five years. The first annual installment is $5 million and will be paid this year. Note some acceleration terms exist should InterMune receive 150 million in partnering/licensing (capped at $10 million/year).
Time-Line
• Late Q4 ‘06/Early Q1 ’07 Shionogi Pirfenidone Phase 3 trial top-line results (InterMune has data sharing agreement but it is not clear if the results will be released to the public… the market will know!)
• Mid Q4 ’06 Hear back from EU about 191 submission.
• Late Q4 ’06 Start dosing patients with 191
• ???? ’07 Interim look in INSPIRE. Company said likely would not disclose unless it was material (i.e. trial stopped)
• November ’07 INSPIRE trial ends (2 years after 600th patient enrolled)
• Q4 ’07 CAPACITY Trials enrollment targeted completion [I think it will be MUCH earlier]
• Q1 ’08 INSPIRE data released
• Q4 ‘07/Q1 ’08 CAPACITIY (Pirfenidone) treatment period ends
• Q1 ’09 Top line results from CAPACITY
Some Arguments For Investing in InterMune
1. It is my impression that some of the criticisms of Actimmune are side effects and limited efficacy. The fact is IPF is a disease with no good therapy and Actimmune has the potential to lengthen lives. While it does not appear to reverse the disease for some patients (treated early) it helps to keep it in check longer. To me a comparison should be more akin to some of the harder to treat cancers (The company also uses PAH as a model I think PAH therapies are much further ahead then IPF treatment options). With no treatment available one would hope the FDA does not place an extremely high hurdle and one would think patients would be very receptive.
2. Both Actimmune and Pirfenidone are potential Billion dollar drugs.
a. Actimmune:
i. Price for fully compliant patient 60K (lets use 50K)
ii. Using OLD incidence of 30K (25K mild-to-moderate) and say 25% penetration
iii. Say average patient stays on therapy 3 years
iv. The math then: 50K * 6250 * 3 = 937,500,000
v. This is VERY CONSERATIVE and VERY doable
1. If approved reimbursement by Medicare for large percent of patients (2/3 > 65)
2. Does not take into consideration incidence (OLD numbers of 50K mild-to-moderate)
3. Feel free to use
a. Higher penetration rates
b. Account for some of the 83K (50K mild-to-moderate) prevalence
c. Longer time on therapy (after all if successful Actimmune will extend life expectancy)
d. NEW prevalence and incidence numbers of 128,000 and 48,000
b. Pirfenidone:
i. Price 30K (estimate based on discount of Tracleer pricing in PAH)
ii. Again using OLD incidence but say 33% penetration (as patients may be more willing to take this as it is an Oral therapy)
iii. Again using 3 years average on therapy
iv. The math: 30K * 8333 * 749,970,000 + EU royalty? Or if market themselves
c. Some preclinical work done and it appears the compounds do not antagonize each other. In fact suggests the compounds may be complimentary if not synergistic.
d. The company has < 35 million shares and even if you include the convertible (due 2011) that adds about 8 million more shares. I’d work out the EPS numbers for you but you wouldn’t believe me, so you can plug in the numbers yourself. The COGS for Actimmune have been in the 21-23%. Sales force would be targeted (50 – 100 people say 200K each). SG&A 30-45 ’06 guidance (feel free to add a fair increase). R&D 90 -105 in ’06. Oh and the company has over 450 million in NOL’s
3. If Actimmune is successful one would think the PEGylated version becomes more of a priority to extend the product and gain patients with an easier to tolerate treatment. Also this may open the door to pursuing some other indications.
and Some Risks…
1. Actimmune failed in its previous Phase 3 and INSPIRE is based in part on retrospective analysis (everybody loves those!)
2. Actimmune has been tested and failed to progress in a number of other indications, most recently a Phase 3 trial in Ovarian Cancer was stopped (company states combo with chemo too toxic and Actimmune group did not receive Full Chemo to account for poorer results)
3. Actimmune is an injectable and has some side effects, though it is approved and has been used safely for a number of years.
4. If the dropout rate is high or compliance to dosing in INSPIRE is low (if one is injecting themselves thinking they are doing so with a Placebo it may be hard to keep on schedule)
5. Pirfenidone in trials has had high incidence of photosensitivity and some GI side effects.
6. I believe the CEO was working (consulting?) for Warburg Pincus and they have a couple of board seats and a big position.
7. Competitive Landscape for IPF Other companies looking at IPF include Genzyme (collaboration with what was CAT) in Phase 1, Bosentan (Tracleer) from Actelion, Phase 2 failed in their endpoint proceeding with Phase 3 different end point, Imatinib (Gleevec), I believe it failed Phase 2 not sure of status going forward. Sildenafil (Phase 2) http://www.clinicaltrials.gov/ct/show/NCT00352482 Others?
Don't know how appropriate to post about Acambis on a Biotech board with talk of vaccines a while back thought it may be OK.
The London shares are taking a huge hit today (about 40%). It was widely believed they would get some of the contract 0 is a shock. A while back there was speculation they would be bought out by Chiron or someone else interested in vaccines. I think they still have a nice little business, wish I would have kept up more with them may be an opportunity to get them on the cheap today
http://yahoo.reuters.com/news/articlehybrid.aspx?type=comktNews&storyID=urn:newsml:reuters.com:2...
UPDATE 3-Acambis loses U.S. smallpox deal, shares slump
Tue Nov 14, 2006 6:21 AM ET
(Adds comment on potential deals, Baxter role, latest shares)
By Mark Potter and Ben Hirschler
LONDON, Nov 14 (Reuters) - British vaccine maker Acambis Plc <ACM.L> has been unexpectedly dropped from consideration by the U.S. government for a new smallpox vaccine contract that analysts believe could be worth up to $1 billion.
Shares in Acambis plunged 39 percent to a six-year low of 95 pence on Tuesday.
Acambis had been competing with Denmark's Bavarian Nordic <BAVA.CO> to supply the U.S. government with a weakened version of the smallpox vaccine suitable for vulnerable members of the population such as the elderly.
At 1100 GMT, Bavarian Nordic shares were up 27 percent at 521 Danish crowns.
Bavarian Nordic Chairman Asger Aamund told Reuters he was surprised Acambis had been eliminated and said he expected his firm would receive news from the U.S. government on its vaccine order by the end of the year.
"We've been sure all along that we'd win an order and I don't think it will take much longer," he said.
Acambis Chief Executive Gordon Cameron said he was perplexed by the U.S. government decision to eliminate his group from the process.
"We believe that our proposal would have met the requirements of the U.S. government, especially given Acambis' track record in the biodefence field," he said in a statement.
PRICE NOT THE ISSUE
A company spokesman said technical issues, rather than price, lay behind the move and Acambis was seeking a meeting with the U.S. Department of Health and Human Services.
"We have not received the specifics as to why our proposal is being excluded on technical grounds. We have requested a meeting with the HHS to get further clarity," he said.
"The response we have received relates only to technical issues," he added.
Acambis had already supplied the U.S. government with more than $500 million worth of standard smallpox vaccine as the country moved to build up stocks amid fears of bio-terrorist attack.
Analysts had assumed Acambis, which has Baxter International Inc <BAX.N> as a manufacturing partner, would win at least half of the new contract. "This is obviously a significant disappointment," Goldman Sachs analysts wrote in a research note. "In our view, management credibility has suffered a significant setback and highlights the imperative of strategic alternatives."
Analysts at UBS said the setback left Acambis with a maximum of two years' worth of cash and put it under more pressure to find a partner for its most advanced pipeline product, a vaccine against Japanese encephalitis, in the course of 2007.
The slump in the company's market value also leaves in doubt its potential to act as a consolidator in the European vaccine and biotech. Acambis appointed veteran dealmaker Peter Fellner as its new chairman earlier this year and Fellner told Reuters in February he intended to help Acambis make acquisitions.
Gudme Rasschou analyst Annette Rye Larsen said the setback for the British company played into the hands of its Danish rival.
"I believe more and more that Bavarian is the only company left that can supply the contract," she said. (additional reporting by Mette Fraende in Copenhagen and Michael Flaherty in New York)
11/23/06 8:17 pm
FYI
Its Phase 1 I believe not sure on the merits of it
http://www.virexx.com/product/hepavaxb.asp
Hepavaxx B
Product Overview
HepaVaxx B is a Chimigen™ therapeutic vaccine developed at the ViRexx research labs for the treatment of chronic hepatitis B viral infections.
HepaVaxx B consists of a recombinant chimeric molecule containing the elements of both a hepatitis B viral antigen and a xenotypic antibody. The molecule is designed to target a particular set of cells that play a dominant role in the body's immune system. Validation of the uptake, processing and activation of the cells responsible for modulating the immune response was conducted by ViRexx using specialized assay systems developed by ViRexx. The selected Chimigen™ vaccine is expressed in insect cells which produce the desired product in large quantities under appropriate culture conditions.
This surprised me. I thought giving him a big multi-year contract was risky when the Dodgers did it. Don't know if he wanted out of LA (maybe he wants to go to Philly finally :)) or thinks he can get more... Hmmm would his agent be S.B. :)
http://sports.yahoo.com/mlb/news?slug=ap-dodgers-drew&prov=ap&type=lgns
J.D. Drew opts for free agency
By JOHN NADEL, AP Sports Writer
November 9, 2006
LOS ANGELES (AP) -- Los Angeles Dodgers outfielder J.D. Drew opted out of the final three years of his contract Thursday, making him an unrestricted free agent.
Drew, who turns 31 on Saturday, hit .283 with 20 homers and 100 RBIs last season -- his second with the Dodgers. He signed a five-year, $55 million contract on Dec. 23, 2004, and had been guaranteed $33 million over the next three years with Los Angeles.
Drew was limited to 72 games in his first year with Los Angeles by injuries, hitting .286 with 15 homers and 36 RBIs. Previously, he played with St. Louis from 1998-03 and with Atlanta in 2004, where he batted a career-best .305 with 31 homers and 93 RBIs.
I am not a shareholder but have listened to several of their calls. I think Paul is VERY direct and though I have never called him think he would be very open to talking to shareholders (from comments on calls).
My concern is that he seems like a gambler and I still think stroke is risky especially their trial since their window is longer (believe it is 6 hours) so I wonder if they will get patients that are more advanced?
I liked the company for the (small) Nemanda royalty, had another small drug for brain edema that they licensed out (to find their stroke trial) and they have a MUCH bigger royalty for Nemanda if it is developed in I believe a couple other indications.
Well it never got cheap enough for me so I am not holding. I would prefer some more risk off the table before investing here.
Biowatch,
Thanks for your suggestions. A used book is more then adequate for my purposes.
I went with a used copy of the Daniel book, also saw he had a couple other similar books. Also found a few other titles and got a couple at used http://www.abebooks.com/ instead of Amazon.
Thanks again for the suggestions. Sorry don't have private msg capability.
Thanks for the suggestion. I'll check out the specific book you mentioned.
We think alike, I was searching at Amazon for Biostatistics and found a few but thought to ask here to see if someone might know of the "Bible" on the subject.
I'm not looking to do the calculations (I could always get software for that) just to get a better understanding of various things.
Dew,
I'd be careful with the CIBC link as I believe people will first have to register to use it. They probably use a cookie or something to make sure users have first entered the source code.
Anyone have a recommendation for a good book on biostatistics? The level could be moderate to advanced. TIA
its underneath in the message (healthcare2006) it took me some searching to find it!
The ThinkEquity will probably turnout to be http://www.wsw.com/webcast/tep12/
but that wasn't working now so not sure and I only posted the referring page
For those who like to listen to more then one presentation here are some links to get the links for the webcasts (you may have to register to view):
11/6-11/8 Rodman & Renshaw 8th Annual Healthcare Conference
http:wsw.com/webcast/rrshq10/
11/6-11/8 CIBC World Markets 17th Annual Healthcare Conference
http://www.veracast.com/cibcwm/healthcare06/index.cfm
Source Code: healthcare2006
11/7-11/8
Cowen and Company 7th annual global health care conference in London
http://phx.corporate-ir.net/phoenix.zhtml?c=128610&p=conferenceAgenda&id=1230988&day=1
11/10?
ThinkEquity Partners LLC ThinkTank
http://www.thinkequity.com/about/conf/ThinkTank_06.html#webcast
Thanks.