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Didn't see this posted here. This could have a huge impact on some companies. Amgenhas been fighting attacks on their EPO patents for 50,000 years. With all the talk of high prices one would expect legislation to easily pass.
Sorry Dew, Looked like there was a potential quiz or two in this article, but your quizes are usually a bit more difficult :)
http://biz.yahoo.com/ibd/061013/health.html?.v=1&printer=1
Generic Threat Puts Biologic Firms On The Defensive
Friday October 13, 7:00 pm ET
Peter Benesh
Technology marches on, but the law and regulations often don't keep up. That's been the case with biological drugs, which are made from proteins instead of chemicals.
Several early biologics have lost or soon will lose patent protection. If they were chemically based, they'd be vulnerable to competition from generic drugs.
But science has outrun the 22-year-old law that made conventional generic drugs possible, says attorney Marc Scheineson, a former associate commissioner of the Food and Drug Administration and head of the food and drug practice with the law firm of Alston & Bird in Washington, D.C.
"The law did not envision application to unique characteristics of biologics," Scheineson said. "It was designed for chemical drugs, not living organisms."
The FDA contends it lacks authority to approve generic biologics. On Sept. 29, Rep. Henry Waxman, D-Calif., co-author of the 1984 law that paved the way for generic drugs to compete with off-patent branded drugs, re-entered the scene.
Backed by Democratic Sens. Hillary Clinton and Charles Schumer of New York, Waxman introduced a bill to update the law with procedures to let the FDA approve generic biologics.
"This bill will use competition to make biological drugs -- which are often prohibitively expensive -- available to those who suffer from diseases like cancer, diabetes and AIDS," Waxman said in a statement.
Biologics, also called specialty drugs or biopharmaceuticals, are a huge and growing sector of the health care economy. In 2005, biologics had U.S. sales of $32.8 billion, or 14% of total drug sales, according to market analysis firm IMS Health.
While year-over-year sales of biologics rose 17% in 2005, sales of conventional prescription drugs gained only 5.4%.
Annual sales of biologics will reach $90 billion by 2009, says Mark Merritt, president and chief executive of the Pharmaceutical Care Management Association, which represents U.S. pharmacy benefit managers. Merritt's estimate was given during July testimony before a Senate panel.
States Of Panic?
One reason sales are rising is that prices of the drugs are rising. The cost of biologics increased 17.5% last year vs. 10% for conventional drugs, Merritt told the panel.
It costs $5,500 a month to treat colorectal cancer with Genentech's (NYSE:DNA - News) Avastin. The drug might cost twice as much if approved for breast cancer because patients will need double the dose. The FDA approved it on Oct. 11 for certain kinds of lung cancer.
High prices have created alarm among officials at state governments, which pay half of the cost for Medicaid patients.
Since August, governors of seven states -- Kansas, Minnesota, New Mexico, Vermont, Virginia, West Virginia and Wisconsin -- have petitioned the FDA to set out guidelines for generic insulin and human growth hormone, two biologics that are off patent.
Patents have expired on several biologics, and more will soon. Those due to come off patent include Amgen's (NASDAQ:AMGN - News) Epogen, used to treat anemia in dialysis patients, and Neu-pogen, which boosts white blood cells after cancer treatment.
The list also includes Genentech's Activase for heart attack, and Pfizer's (NYSE:PFE - News) Genotropin for children with stunted growth, which came off-patent in February.
Several generic drug makers -- including Barr Pharmaceuticals (NYSE:BRL - News), Mylan Laboratories (NYSE:MYL - News) and Teva Pharmaceutical (NASDAQ:TEVA - News) -- are gearing up to enter the lucrative market by acquiring firms equipped to do the work.
Sandoz, a unit of Novartis (NYSE:NVS - News), is ready to start making Omnitrope, a generic version of Pfizer's $800 million-a-year human growth hormone, Genotropin.
Sandoz had to sue the FDA in federal court to force it to rule on Omnitrope. The court ordered the FDA to make a decision. In May the FDA issued the approval, though on its Web site the agency said the decision didn't necessarily clear the way for other approvals. The FDA didn't return calls seeking comment.
Burden Of Proof
Good luck trying to figure out how the FDA can approve Sandoz's generic biologic without opening the floodgates to more generics.
"(The issues) are extremely legalistic and as complicated as you can get," Scheineson said.
Much of it boils down to the FDA's inability to determine whether a biological drug is identical in all respects to the original drug. Making that exact match isn't easy.
"Biological molecules are more complex (than chemical-based drugs)," said Joseph Reisman, a biotech attorney with Knobbe, Martens Olson & Bear in San Diego. "They can react differently with cells and receptors on cells. Their presence in the bloodstream can't be tracked in any linear way to their efficacy."
The European Union is satisfied that a generic biologic can be as safe and effective as the branded original. The EU approved Omnitrope in April and created procedures for other generic biologics to follow.
The U.S. Biotechnology Industry Association wants makers of generics to have to go through all the clinical trials for safety and efficacy that the original drugs faced, something generics makers see as expensive and unnecessary.
Kathleen Jaeger, CEO of the Generic Pharmaceutical Association, says her group plans to push the Waxman bill.
That would suit the FDA, Scheineson says. "The FDA wants a congressional fix."
I am with Dew my biggest positons haven't been under 3 ever (going by memory so not 100% sure). I do own a few < 1 even but I personally wish every company was run like Buffet. Few shares, don't issue, don't do splits and of course great appreciation :). Shouldn't Market Cap / Earnings be the important thing? I guess there is some glammor with these cheap stocks.
Don't know if this was posted (though it is just an update of old news). Aside from the implications on biotech/pharma stocks, it is disappointing that governments really are valuing Human Life/QOL! Not to mention that some diseases may be slowed in early stages more easily then mid-late stages!
http://tinyurl.com/s9z7y
The Association of the British Pharmaceutical Industry (ABPI) attacked the decision, saying it "disregards the basic humanity of a situation where patients who could benefit from a medicine are denied it" and would discourage further research.
"This decision makes it harder for companies to justify devoting the enormous sums of money and resource necessary to research and develop new medicines," Nigel Brooksby, president of the ABPI said.
MODERATE SEVERITY
The new guidance means that Aricept, from Pfizer Inc. (PFE.N: Quote, Profile, Research) and Eisai Co Ltd (4523.T: Quote, NEWS, Research), along with Shire Plc's (SHP.L: Quote, Profile, Research) Reminyl and Novartis AG's (NOVN.VX: Quote, Profile, Research) Exelon will be prescribed only for patients with Alzheimer's disease of moderate severity.
Critics say the move deprives the majority of early-stage Alzheimer's disease sufferers of a treatment option that is widely available in other countries.
A fourth drug, Ebixa, made by Lundbeck (LUN.CO: Quote, Profile, Research), is given to patients with more serious disease, but NICE is not convinced of its benefits and said it should only be used in clinical trials.
Charities caring for Alzheimer's patients and some medical experts condemned NICE's refusal to budge.
"This is a terrible decision based on a deeply flawed process," Dr David Anderson of the Royal College of Psychiatrists said. "Implementation of this guidance will set the treatment of Alzheimer's disease back 10 years."
But others accepted that NICE had to strike a value-for-money balance.
"I agree with this decision by NICE. The problem with these drugs is that they have a very small effect in patients with dementia, which is of uncertain value," Professor Raymond MacAllister of University College London said.
The NICE deliberations have been watched by governments around the world, who increasingly have to weigh the benefits of modern medicines against their prices. The drugs cost around 1,000 pounds ($1,860) per patient a year.
David,
I saw (heard) you don't take payment from the companies you profile (which is refereshing) but how do you determine which companies you cover?
Are you the one who follows REX? I am more optimistic then some on OvaRex but more pessimestic then a lot of REX shareholders on the outcome (very strong in the middle :)) I started going through some old studies, papers and United's R&D day info and have some thoughts (currently mostly in my head) I was hoping to engage others in some discussion on the merits. I know poorgradstudent had some opinions curious if you do as well.
I remember a post where you said there were 3 (or 4?) of you (and then later said you were kidding... I think you were kidding about the kidding or you just don't need sleep.
I'll have to "unhide" the board description (didn't realize it gets updated).
Maybe after InterMune releases their Q3, I'll try to clean up everything.
Thanks for making such a useful board!
gradstudent,
Appreciate your posts and commentary... Sometime I want to pick up the OvaRex discussion. Saw your post against Murine MAB's so not sure if you are against it more because it is that, retrospective analysis, bad data, etc. I am strongly divided on the drug and thought to bounce a few numbers but I think the conclusion is 2nd half '07 (at least) barring some DSMB intervention so there is time.
OK Thanks for the info Dew. Is the message editable? I also noticed after posting that longer links didn't turn out well. Once the 15 minute window passes can that be edited too? There is probably a few more things I missed if I reread it especially as I may have overlooked things that someone new to the company may be curious out.
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 (it appears Pirfenidone is exceeding enrollment expectations and INSPIRE enrollment for Actimmune added 225 patients in less then 6 months)
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.
4. 191 Will be partnered at some point. It seems to me 500 million is the starting point, OK 191 may be 3rd but it appears (at least preclinically) to have some advantages of Schering and Vertex compounds. See http://messages.finance.yahoo.com/Healthcare/Biotechnology_and_Drugs/threadview?m=tm&bn=9830&...
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 causes light sensitivity and may have some liver monitoring requirements
6. I believe the CEO was working (consulting?) for Warburg Pincus and they have a couple of board seats and a big position.
7. Other companies looking at IPF include Genzyme (collaboration with what was CAT) in Phase 1, Actelion (with Tracleer though Phase 2 failed in their endpoint proceed with Phase 3 different end point), Gleevec (believe it failed Phase 2 not sure going forward). CoTherix (PAH patients with IPF), others?
InterMune "ReadMe First"
InterMune http://www.intermune.com/
Pulmonology and Hepatology focus. In Pulmonology focused on Idiopathic Pulmonary Fibrosis (IPF) and in Hepatology have InterMune 191 recently submitted to European Union for clinical development (also an undisclosed preclinical compound)
IPF
• Scarring of lungs, unknown cause
• Median survival from diagnosis 2-5 years
• 83K US, 30K diagnosed each year, is what InterMune WAS using in their presentations…. “According to the new findings, pulmonary fibrosis now affects 128,000 people and more than 48,000 new cases are diagnosed each year. In 2000, the Consensus Statement of the American Thoracic Society estimated prevalence at approximately 50,000” http://biz.yahoo.com/prnews/061005/cgth065.html?.v=46&printer=1 or http://www.investorshub.com/boards/read_msg.asp?message_id=13818453 or http://ajrccm.atsjournals.org/cgi/content/abstract/174/7/810 (journal abstract)
• No approved therapy, steroid and immune suppressants used (such as Methotrexate and Prednisone) along with off-label use of Actimmune
Actimmune http://www.actimmune.com/
• Interferon Gamma 1B natural occurring protein administered by sub-q injection (200mcg, 3x week for IPF patients in protocol for INSPIRE study)
• IP Protection (http://www.corporate-ir.net/ireye/ir_site.zhtml?ticker=ITMN&script=410&layout=6&item_id=... 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.
• Being studied for 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: 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://www.corporate-ir.net/ireye/ir_site.zhtml?ticker=ITMN&script=410&layout=6&item_id=... 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.
Study ITT Patients Follow-up Duration No. of Deaths Actimmune® No. of Deaths Control Relative reduction P-value*
GIPF-0011 (NEJM, Jan 2004) 330 1.4 years 16/162
(9.9%) 28/168 (16.7%) 41% 0.08
Ziesche2 (CHEST, Nov 2002) 18 5 years 2/9
(22.2%) 7/9 (77.8%) 71% 0.01
Antoniou3 (ERJ, Apr 2006) 50 ~2 years 5/32
(15.6%) 7/18 (38.8%) 70% 0.028
INSPIRE Trial http://www.inspiretrial.com/
http://www.clinicaltrials.gov/ct/show/NCT00075998
• Randomized, Double-Blind, Placebo-Controlled
• ~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%)
• 2:1 Randomization – Actimmune: Placebo
• Treatment Period: 2 Years after randomization of 600th patient. 600th patient enrolled in November 2005. Completed enrollment April 2006 (826 patients total)
• PR for rational of increasing enrollment http://www.corporate-ir.net/ireye/ir_site.zhtml?ticker=ITMN&script=410&layout=6&item_id=...
Pirfenidone
• Orally active small molecule administered as 3x day pill
• Acquired NA and EU rights from Marnac, http://www.marnac.com/ for Fibrotic Indications. (PR - http://www.corporate-ir.net/ireye/ir_site.zhtml?ticker=ITMN&script=410&layout=6&item_id=... 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: 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.
• Being studied in a number of indications (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://www.corporate-ir.net/ireye/ir_site.zhtml?ticker=ITMN&script=410&layout=6&item_id=... 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://www.corporate-ir.net/ireye/ir_site.zhtml?ticker=ITMN&script=410&layout=6&item_id=...
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
InterMune 191
• Collaboration began with Array BioPharma http://www.corporate-ir.net/ireye/ir_site.zhtml?ticker=ITMN&script=410&layout=6&item_id=.... The royalties are described as “small”
• High affinity for and stability in the liver
• Favorable pharmacokinetic profile
• Potency of ITMN compound is maintained against mutations that show resistance to other protease inhibitors
• HCV replicon EC50 = ~2nM (VX-950=402nM, SCH-503034=200nM)
• Liver exposure in Rat/Primate (30 mg/kg) nM concentration 2,680/188 Log Drop in replicon RNA 3.23/2.08 (after 12 hours!)
• No observed toxicity in mammalian cells
• HCV protease selectivity is high
• Good safety margins for: Body weights, organ weights, Clinical chemistry, clotting parameters and hematology, Renal, neurologic, gastrointestinal, immune and cardiovascular systems
• There were no significant effects on microscopic histopathology on any organ or tissue, including heart and liver
• Potential BID dosing
• 9/26/06 CTA submitted to the French Medicinal and Biological Products Evaluation Directorate http://www.corporate-ir.net/ireye/ir_site.zhtml?ticker=ITMN&script=410&layout=6&item_id=...
Other Pipeline/Interests
• Early stage preclinical program partnered with Array (undisclosed indication in Hepatology)
• Equity interest in Targanta Therapeutics, http://www.targanta.com/ as a result of selling Oritavancin in December 2005. (http://www.corporate-ir.net/ireye/ir_site.zhtml?ticker=ITMN&script=410&layout=6&item_id=...
• Early stage work on PEGylated version of Actimmune (with Maxygen), does not appear to have much effort as this time. (http://www.corporate-ir.net/ireye/ir_site.zhtml?ticker=ITMN&script=410&layout=6&item_id=...
• PEG-Alfacon (PEGylated version of Infergen) appears unlikely to be further developed.
•
Financials
• Cash 180 million (end of Q2)
• 170 million convertible low coupon, 2011 maturity (after 2 phase 3’s)
• 33 million shares outstanding
• Guidance
o Revenue 75– 100 million.
o COGS 21-23%.
o R&D 90-105 million (48 million first 6 month expect back weighted with CAPACITY enrollment and 191 ramp-up). 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).
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/Early Q1 ’07 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
• Q1 ’08 INSPIRE data released
• Q4 ‘07/Q1 ’08 CAPACITIY (Pirfenidone) treatment period ends
• Q1 ’09 Top line results from CAPACITY
Was refering to a general "read me". As long as poorgradstudent (and others) aren't an English majors and you exuse some of the formating (I put it in word and I don't think it will cut and past well) I'll post what I put together now in one post and then some comments in the following.
I am not a big fan of Warburg Pincus either!! I was a TKT shareholder prior to their board members pressing for the sale to Shire (which the then CEO Michael Astrue was highly outspoken against and resigned over). I think that is a real possibility now with Gilead (which was looking at InterMunes PI) moving into Pulmonology and Dan Welch having sold one company to Gilead. I think there are a few other "good fits" too. The good thing is Warburg have been holders for a number of years and perhaps they wouldn't consider selling anywhere near current levels!
Dew, I am working on organizing some of my notes for a write-up on ITMN (More the pulmonology side, IPF) then the Hepatology, but give me a few days. Any good message posts to refer to as a guide for a "read me first" (sorry I don't have search I subscribe to SI and can't yet justify paying here particularly since they have the same ownership) or is it better if I e-mail you a word or acrobat document?
I have been meaning to do a nice write-up on this board on InterMune’s Pulmonology products as I am surprised at how little attention is given to InterMune in particular to their Pulmonology products. Most people think of them as just having a Protease Inhibitor that had good preclinical results and is awaiting EU authorization to start trials…
I will concede Actimmune treatment alone isn’t enough for people with Pulmonary Fibrosis but this is the most advanced drug in development AND its already approved for 2 rare indications. At 60K per year, if approved, this will not take many patients to become a huge drug. I think the company did a lot better job in the INSPIRE trial design to overcome the failed GIPF001 results (The literature would suggest being able to show a survival benefit with this design and the larger trial gives them much more power then the 001 retrospective subset analysis).
I also think Pirfenidone has a very good chance at showing efficacy (improved lung function in this case). My guess is that the company will beat enrollment too based on a few things (INSPIRE enrollment, being an oral compound, no off label use available such as is for Actimmune).
One of these days I’ll put together a better write up… unless INSPIRE Interim analysis stops for efficacy or Shionogi’s Pirfenidone trial does well and the market begins to value in the potential for a drug in Pulmonary Fibrosis.
http://biz.yahoo.com/prnews/061005/cgth065.html?.v=46&printer=1
Pulmonary Fibrosis Prevalence Increases 156 Percent in Six Years
Thursday October 5, 5:25 pm ET
Funding for Research to Find Treatment, Cure Unacceptably Low
SAN JOSE, Calif., Oct. 5 /PRNewswire/ -- Prevalence of pulmonary fibrosis has increased 156 percent in the last six years, according to a new paper published this week in the American Journal of Respiratory and Critical Care Medicine. The article, entitled "Incidence and Prevalence of IPF" appears in the Oct. 1 issue of the Journal.
ADVERTISEMENT
According to the new findings, pulmonary fibrosis now affects 128,000 people and more than 48,000 new cases are diagnosed each year(1). In 2000, the Consensus Statement of the American Thoracic Society estimated prevalence at approximately 50,000(2).
Idiopathic pulmonary fibrosis (IPF) is the deadliest form of pulmonary fibrosis and is a progressive and ultimately fatal lung disease. There is no known cause, no FDA-approved treatment and no cure.
IPF deadliest form of pulmonary fibrosis
IPF is characterized by progressive scarring in the lungs that robs patients of their ability to breathe. The disease is relentlessly progressive, with a median survival time from diagnosis of three to five years, and a five- year survival rate of approximately 20 percent.
"While this study, like all epidemiological studies, has some limitations, it reinforces a prevailing opinion in the pulmonary community that IPF prevalence and incidence is, in fact, higher than people and the medical community realize," said Ganesh Raghu, M.D., lead author of the paper and professor of medicine at University of Washington, Seattle, Wash., and director of the University of Washington Medical Center's Interstitial Lung Diseases/Sarcoidosis/Pulmonary Fibrosis program. "Based on increased awareness in the entities of idiopathic interstitial pneumonia among pulmonologists, the true incidence and prevalence of idiopathic pulmonary fibrosis and interstitial pneumonias is likely to be significantly higher than this study suggests. Increased funding for research is desperately needed to identify new approaches to understanding and treating this devastating disease."
Increases in awareness, education improve diagnosis
According to Dr. Raghu, a world-renowned IPF expert who has dedicated his career to IPF/interstitial lung diseases community, the reasons for the increase in numbers of people across the country diagnosed with disease is more than likely due to an increase in education of the disease both to the public as well as to medical professionals.
"My personal feeling is that more and more doctors are now aware of the disease entities associated with pulmonary fibrosis of unknown cause," said Dr. Raghu. "With routine access to high resolution CT scans of the chest, they are identifying the disease patterns earlier than ever before and hence able to diagnose these conditions better than a few years ago. Non profit organizations like the Coalition for Pulmonary Fibrosis (CPF) and respiratory societies dedicated to enhance education and research of pulmonary fibrosis have certainly contributed greatly in surfacing the problem and helping people to recognize it more."
To view the complete article "Incidence and Prevalence of IPF", please visit http://www.coalitionforpf.org/ipfresearch .
Funding for IPF research unacceptably low
IPF research is often delayed because researchers have difficulty obtaining the funds necessary to fuel new studies. Funding for research, including federal funding, remains a massive challenge for the IPF community, despite clear evidence that new cases are being diagnosed at an alarming rate.
"To make matters worse there is no proven cause of IPF, so research is still needed to better understand disease progression," said Mark Shreve, chief executive officer of the CPF. "Increased research funding is needed now more than ever to help improve understanding of IPF so that new approaches to treat the disease can be identified."
Federal funding for IPF is estimated at $14 million from 2006-2008, primarily through the IPF Research Network (IPFNet) program funded by the National Institutes of Health, according to Shreve. "For a disease that affects so many people, federal funding remains unacceptably low," he said.
CPF funds research to supplement federal dollars
The CPF, the nation's largest nonprofit organization representing the IPF community, is working to fund research to supplement federal dollars. To date, the CPF has awarded more than $250,000 in research gifts to leading medical centers in the U.S., and has also partnered with the American Thoracic Society to jointly fund a $100,000 research award in 2007. The CPF also advocates for improved federal funding of IPF research through its campaign ACT advocacy program in Washington, DC. To learn more about the CPF or campaign ACT, please visit http://www.coalitionforpf.org or call (888) 222-8541.
Key Facts about IPF:
-- Prevalence of pulmonary fibrosis is on the rise and expected to double
in the next 20 years as the U.S. population continues to age.
-- IPF symptoms (shortness of breath and unexplained, persistent dry
cough) are so similar to other lung ailments that as many as 50 percent
of patients are initially misdiagnosed.
-- The only treatment shown to extend survival is lung transplantation;
however 50 percent of IPF patients who are eligible for transplant pass
away before a donor lung becomes available.
About the Coalition for Pulmonary Fibrosis:
The Coalition for Pulmonary Fibrosis (CPF) is a 501(c)(3) nonprofit organization, founded in 2001 to accelerate research efforts leading to a cure for idiopathic pulmonary fibrosis (IPF), while educating, supporting, and advocating for the community of patients, families, and medical professionals fighting this disease. The CPF funds promising research into new approaches to treat and cure IPF; provides patients and families with comprehensive education materials, resources, and hope; serves as a voice for national advocacy of IPF issues; and works to improve awareness of IPF in the medical community as well as the general public. The CPF's nonprofit partners include many of the most respected medical centers and healthcare organizations in the U.S. With more than 10,000 members nationwide, the CPF is the largest nonprofit organization in the U.S. dedicated to advocating for those with pulmonary fibrosis. For more information please visit http://www.coalitionforpf.org or call (888) 222-8541.
References
(1) Ganesh Raghu, Derek Weycker, John Edelsberg, Williamson Z. Bradford,
Gerry Oster: Incidence and Prevalence of Idiopathic Pulmonary
Fibrosis: American journal of Respiratory and Crit Care Med; vol 174:
810-816,2006
(2) American Thoracic Society. Idiopathic pulmonary fibrosis: diagnosis
and treatment (international consensus statement). Am J Respir Crit
Care Med. 2000; 161:646-664.
Just a guess but I'd think Millennium is one... I am taking this is operating loss and not loss in market cap :)
UBS Conference Schedule
http://www.ibb.ubs.com/Conferences/pdfs/Final%20GLSC%20Agenda%209-21.pdf
Sorry it is too big and not in a good format to cut and paste
David,
Just heard your interview on Zanghi (?), glad to see you have BioMarin as your top pick (sorry I don't subscribe to your newsletter to have known sooner). Just a point about market for drugs I believe the numbers you mention are off unless you have insights outside of what the company has stated (but it sounded that you were speaking in generalizations). I believe I heard 300 people for enzyme product, 20k and 10k for other products. The numbers I have are (Developed World wide):
Naglazyme (MPS VI) - 1100
Aldurazyme (MPS I) - 3000
Phenoptin and Phenylase (PKU) - 50,000 30-50% treatable with Phenoptin, 100% with Phenylase (though probably only pursue ones that don’t respond to Phenoptin)
6R BH4 (Various Cardio Vascular Indications) - Millions
I believe they had in the past (year+) stated they identified OVER 300 patients for MPS VI but that is a while ago now and that was just identified not prevalence.
Personally, I think (barring major changes/investments in BH4) the company will be cash flow breakeven in 2007, especially if you factor in milestone payments but the company says 2008.
Sorry to be nit picky but I really like the prospects for the company and have been a long time shareholder (I owned in the Fred Price days). I thought the interview Allen Leon did with Mr. Bienaime near when he first joined the company was one of the best overviews of a company, in particular getting Mr. Bienaime to break down the investment potential for the company (for those that don't like to let their imaginations run wild on their own)
If I can guess at just one I'll say Transkaryotic.
mskatiescarletohara,
Thanks for the reply. PPHM is a company I wasn't familiar with. It seems like half the biotech's are working on something in Hep. C.
Which PI's are you referring too, the one's in clinical trials? ABT's Kaletra is the only other PI I'm aware of that is being testing in combo with other HCV compounds.
I was thinking a few years out if there are 3 PI's approved (or earlier in the clinic if 1 is approved and a company wants to do something with their PI). Not a scientist but curious if there may be some advantage to combining 2 PI's with different resistence against variants or if some scientific reason (at this early stage) why it wouldn't make sense to try 2 different PI's together
I also signed up late and appreciate you resending it!
Sorry if this has been mentioned before (I don't have search, subscribe to SI but not IHub)...
Any thoughts on InterMune's Protease Inhibitor Program or waiting to see how it does in Phase 1B? Their preclinical data seems very impressive even though they are behind Schering and Vertex. Wonder if anyone would consider combining PI's if ones are more resistant against certain variants then others?
ALS is a terrible disease and I hope some sort of treatments become available... Somehow I think CYTR would have went up less then it did if they just received a $24 million grant with no royalty
If we are voting for funniest call I have two candidates (going back a couple years).
1. When Genzyme was removing the tracking stock. One caller (I believe he had a fund with a lot of shares) wouldn't let up on Henri Termeer.... I believe a lawsuit followed.
2. Back when TKT was led by Richard Selden and in the race with Genzyme to get a Fabry drug to market. An analyst (Jennifer Chow??) really laid in to him accusing him of basically lying... He would resign a couple months after and IMHO one of the best BioTech CEO's would take over.
Maybe your talking funny in a different sense? I'll have to listen to the call. Any part in particular or just management in general?
Interesting article in the August 3 issue of NEJM, "Access before Approval — A Right to Take Experimental Drugs?"
http://content.nejm.org/cgi/content/full/355/5/437?query=TOC
I believe a couple years back a (rich) patient had filed a lawsuit against InterMune attempting to force them to sell him Perfinidone which was (and still is) in clinical trials to treat Idiopathic Pulmonary Fibrosis (a life threatening lung disease). I believe the suit was later drop and he may have found a source for it (I am guessing he got into the Japanese shionogi trial).
Wonder what implications this may have on some of the more lifethreating diseases without adequate treatment and if the FDA will be under more pressure to streamline some sort of limited approval.
poorgradstudent,
Thanks for the reply and link. It is interesting to note that treatment-stop indicator to be highly significant to disease progression in this trial. I would think three month of chemo would have an effect of PFS, how much??
I had come across some other front-line trials with different PFS such as SCOTROC which compared paclitaxel and docetaxel (granted it was Stages IC to Stage IV) and had about 15 month PFS in each arm (I am not sure whether this is from after surgery or after chemo).
Another other study I have heard is a good reference is the GOG-0182. I haven't seen the ASCO presentation of this. The company pointed out in a presentation that study goes from time of randomization so it would include the chemo time (6 months or so).
Have been reading posts on this board for a few weeks now and find the quality and level of discussion very high! I was wondering if anyone follows UTHR or REX and the currently running IMPACT I&II Ovarian cancer trials?
By my estimation the mean PFS is likely to be 20+ month (in each trial). If one goes by the Successful Front-Line Therapy Patients (SFLT) subset of the Phase 2B trial that had a Placebo averages 10.8 months (ITT Placebo was 10.3 months) that would put the OvaRex arm in the 24+ month range which would be HIGHLY statistically significant.
However, what is puzzling me is the Phase 2B was powered with an assumption of 18 month median TTR in the Placebo arm. I have been looking for some historical data to see which is the more reasonable number and was wondering if anyone has any insight (links would be greatly appreciated)
Basically it is for Stage III/IV patients with complete clinical response to front-line surgery and chemo (platinum based) with elevated CA-125 levels prior to surgery. There is a lot more that is brief summary
The entry criteria for the trial can be found at:
http://www.clinicaltrials.gov/ct/show/NCT00050375
Thanks in advance for any info and for making a useful discussion board!
Interesting trading in SMID today. Seems like the past couple months the stock has a day with big volume and 20 - 30 cent increase then volume dries up and goes quiet. Anyone care to speculate?
Has anyone been to one of their annual meetings? I know its probably not till August but wondering if it is worthwhile. Thanks in advance.
Not that I expect Warren to start buying our stock (though I certainly wouldn't object). Maybe this will bring some interest to our company
http://news.yahoo.com/s/nm/20060507/bs_nm/financial_israel_olmert_dc_1
JERUSALEM (Reuters) - Israeli Prime Minister Ehud Olmert on Sunday said he believed
Warren Buffett's multi-billion dollar deal to buy an Israeli company could lead to more such transactions by foreign investors.
Buffett's Berkshire Hathaway (NYSE:BRKA - news) (NYSE:BRKB - news) over the weekend bought 80 percent of privately held Iscar Metalworking Cos for $4 billion, marking one of the largest deals involving an Israeli company and Buffett's biggest investment outside the United States.
Iscar manufacturers metalworking tools used by makers of heavy equipment such as cars and planes. Reports about the deal dominated local news in
Israel.
"This is not just another deal for the Israeli economy," Olmert told ministers at the start of a meeting of his cabinet, which was sworn in by parliament last week.
"We are talking about the biggest investor in the world who never before made an investment outside of the United States," he said.
"His decision to invest in an Israeli company, in a country he has never visited, in a company whose (Israeli) factories he has never seen ... indicates first and foremost great confidence in Israel's economy, full trust in the stability of Israel's economic system and belief in its great potential," Olmert said.
Buffett, who bought the stake in Iscar from the Wertheimer family -- which will retain 20 percent -- was quoted as saying he would consider further investments in Israel.
"I plan further acquisitions of Israeli companies in the future," Israeli mass daily Maariv quoted him as saying from Omaha, Nebraska.
Buffett, the world's second richest man after Microsoft's
Bill Gates, also plans to visit Israel in September to inspect his investment and examine the local business scene, Maariv said.
He told thousands of shareholders on Saturday he seeks to shrink his cash pile to $10 billion from above $40 billion.
"When a man like Warren Buffett, in front of 25,000 of the most important economic minds in the world, that came to listen to him, says he believes in the Israeli economy, it's a signal that tens of thousands of investors around the world will do what's best for them," Olmert said.
"What's good for Warren Buffett with $4 billion will be good for many more investors and we can just be happy with that."
The deal sent Israeli shares up 2.6 percent on Sunday in anticipation of a jump in foreign investment.
Israel in 2003 shifted from a socialist economy to a free-market one and has courted foreign investors. International groups have bought stakes in two of Israel's top banks, Leumi and Discount, and main phone company Bezeq.
In 2005, when the economy grew 5.2 percent, foreigners invested some $10 billion. The economy is set to grow another 4-4.5 percent this year.