Pulmonary Interstitial Fibrosis(Lung Disease)

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from a preliminary analysis of data from Part 2 of the Phase 2a clinical trial of the investigational oral drug VX-770 in cystic fibrosis (CF) patients who carry the G551DCFTR mutation. VX-770, an investigational CFTR potentiator, was well-tolerated when dosed orally as 150 mg or 250 mg twice daily for 28 days. In this analysis, no patients discontinued treatment and no serious adverse events were reported. At both the 150 mg and 250 mg doses, significant improvements in lung function, as measured by an increase in FEV1, and significant improvements in the function of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, as measured by changes from baseline in sweat chloride levels and changes in nasal potential difference (NPD), were observed. In patients receiving placebo, a smaller increase in FEV1 was observed at 28 days that was not statistically significant, and no significant changes from baseline in sweat chloride levels or NPD were observed. Based on these results, Vertex intends to work with global regulatory authorities to finalize the design of a registration program for VX-770 targeted to begin in 2009. VX-770 was developed with support from Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation.

From: http://news.morningstar.com/newsnet/ViewNews.aspx?article=/BW/20081020005727_univ.xml

 

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Here is yesterday's addition to my intermune file:

3Feb09: The primary endpoint Forced Vital Capacity at Week 72 was met (p=0.001) in CAPACITY 2, along with the secondary endpoints PFS. Pirfenidone was safe and well tolerated. Now preparing NDA and soon MAA to EU’s EMEA.

Read more at:
http://quicktake.morningstar.com/stocknet/san.aspx?id=274278
and:
"log on to the investor relations page of the company's website at http://www.intermune.com. ... A replay of the webcast and teleconference ... will be available for 10 business days following the call of 3Feb09

 

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As I understand Pirfenidone is not very effective and may not get approval
See the article below.
P.J.LAKHAPATE
plakhapate@gmail.com

American Thoracic Society: Pirfenidone Slows IPF Lung Declines
By Michael Smith, North American Correspondent, MedPage Today
Published: May 20, 2008
Reviewed by Zalman S. Agus, MD; Emeritus Professor
University of Pennsylvania School of Medicine. Earn CME/CE credit
for reading medical news
TORONTO, May 20 -- An investigational anti-fibrotic agent is showing promise in the treatment of idiopathic pulmonary fibrosis (IPF), a researcher said here.
Action Points

* Explain to interested patients that there are no approved treatments for idiopathic pulmonary fibrosis.

* Note that this clinical study suggests a new investigational agent may reduce the rate of decline in lung function in patients with the disease.

* Note that this study was published as an abstract and presented orally at a conference. These data and conclusions should be considered to be preliminary until published in a peer-reviewed journal.

In a randomized, double-blind phase III study, pirfenidone, at either of two doses, significantly reduced (at P<0.05) the loss of lung capacity compared with placebo, according to Takashi Ogura, M.D., of the Kanagawa Cardiovascular and Respiratory Center in Yokohama, Japan.

"I expect our study will guide new therapies for IPF in the future," Dr. Ogura said at a press conference at the American Thoracic Society meeting.

The drug, licensed in the U.S. to InterMune Inc., got fast-track status from the FDA yesterday.

Dr. Ogura said the drug is an anti-inflammatory and an anti-oxidant, but that its key mechanism of action in IPF is anti-fibrotic.

After promising earlier studies, he and colleagues enrolled 267 patients and randomized them to placebo, or to one of two doses of the drug -- 1,200 and 1,800 milligrams a day.

The primary endpoint of the 52-week study was change from baseline in lung vital capacity, while secondary endpoints included progression-free survival, Dr. Ogura said.

For this study, progression-free survival was defined as a period without either death or a decrease of more than 10% in vital capacity, Dr. Ogura said.

The researchers found that the drug significantly affected vital capacity:

* Patients on placebo lost 70 milliliters more vital capacity on average than did patients getting the higher dose, a difference that was significant at P<0.05.
* Patients on placebo lost 80 milliliters more vital capacity on average than did patients getting the lower dose, also significant at P=0.05.

Also, Dr. Ogura said, progression-free survival was significantly better (at P<0.05) for both treatment groups than for placebo.

There was no significant difference in oxygen saturation at the end of a six-minute walk test nor in the risk of acute exacerbation, he noted.

The most common adverse events were photosensitivity, loss of appetite, dizziness, and elevated gamma-glutamyl transpeptidase, a marker of poor liver function.

While both doses appeared to improve outcomes, it's not clear that the lower dose is as effective as the higher dose, because of the smaller sample size, Dr. Ogura said.

The researchers made the high-dose cohort larger because of a relatively high dropout rate in earlier studies, Dr. Ogura said.

In this study, however, there was a 32% overall dropout rate, broken down into 37% in the high-dose group, 28% in the low-dose group, and 27% in the placebo group.

He said drop-outs in the treatment groups were mainly due to adverse effects, while the patients in the placebo groups tended to stop because of lack of benefit.

The results of the trial, "done in Japanese patients, may represent a sub-population of patients with IPF," said Ganesh Raghu, M.D., of the University of Washington Medical Center in Seattle, who led one of the early studies on the drug.

Among other things, he said, the study allowed patients to use medications such as prednisone as well, rather than "true placebo controls."

He called for further trials "in a well-defined larger patient population with IPF at different clinical stages."

The study was supported by Shionogi & Co. of Osaka, Japan. Dr. Ogura said he had no disclosures.

 

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Biology forum?

 

Except for the not well defined “yesterday” above your source is accurate and informative. Fast track was announced on 19May08. Here from my ITMN file is more information:

"19May08 FDA fast-track on idiopathic pulmonary fibrosis. Has orphan status in US & EU.
Orphan drug gives exclusivity on Pirfenidone for IPF. (7 years in US & 10 in EU from first sale.) Expecting late 2009/early 2010 US & EU approvals."


As I said in June 08 post, IPF is a terrible disease, leading to certain, slow, unpleasant death in a few years. (Slowly you lose the ability to get the oxygen you need.) There is now, in Japan, a marketed drug available (same as ITMN's drug, which was licensed to the Japanese company by ITMN, in part to get it in use quicker than possible via FDA in US.) Unfortunately, this drug is still far from a "cure." – It only slows the disease progress. (Unfortunately it does not reduce the rate of loss of capacity by even 50%.)

------
*Main, and quite insignificant difference, between the Japanese and ITMN studies is ITMN's primary goal measured FVC (Forced VC) not VC.

**The current ITMN’s P3 (CAPACITY 2) is getting the same results, but with better statistics etc., as the Japanese did. It is also experiencing a much lower "dropout rate" as now the patients in the trial now know (from the Japanese studies) that it does extend their lives and slow their loss of ability to get oxygen.

***True, but the fact that the “placebo patients” did get other drugs also tends to make the improvement wrt placebo all the more impressive / real.

To mods:
I tend to also think this whole thread should be moved to another forum.

PS: It is a terrible thing to say and admit, but true that drugs that do not cure are better for investors than those that do. My father was an MD, when he told me: "Become a dermatologist, not a GP like me. Your patients will never get cured and you will not cause any to die." About a year later I decided I liked physic better and when I did, he started to study for Board Certification Exams as a Psychiatrist - He barely passed, but did on his first try, which few do. I have always been thankful he was just a GP when raising me (prior to my leaving home for college). - I am crazy enough as it is.

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This thread and
http://www.sciforums.com/showpost.php?p=2058478&postcount=39
should be merged with this thread being the one that moves. (does not belong in this forum)

 

"... The Coalition for Pulmonary Fibrosis (CPF) is calling this a historic time in the research and treatment of PF, as a pivotal Phase III clinical trial is completed for Pirfenidone and data released. The positive data could pave the way for the first FDA-approved therapy to treat PF - a relentlessly progressive and ultimately fatal lung disease affecting more than 128,000 people and claiming 40,000 lives each year. The CPF urges the FDA to review this study as soon as possible, given that there are no current FDA-approved treatments for PF. ...
Unfortunately due to the regulatory process, another 40,000 patients will die this year from PF while this application is submitted and reviewed by the FDA. We call on the FDA and the manufacturer to make every possible effort to expedite this process, so that more lives may possibly be saved, ..."

More at: http://pharmalive.com/news/index.cfm?articleID=602600&categoryid=48&newsletter=1

PS I agree with the CPF's urgent POV, especially as THE VERY SAME DRUG IS NOW BEING SOLD IN JAPAN; however last part I made bold is not true. Drug only will extend and make less painful their lives but those now suffering will all die before a real cure is found.

 

To all concerned,

I like to inform you all that after two years of rigorous treatment, controlled diet and exercise now PFT test was carried out for my wife.

According to doctor this test report is similar to the test report for a normal person.

Although her health is improved a lot I can not say she is 100 % cured.

Even though she can walk 5-6 km at a stretch, a little more exercise, change in diet change in climate etc disturbs her immunity level.

Again proper rest , diet etc brings her to normal level after one or two days.

If anybody wants any information , pls do not hesitate to contact me.

P.J.LAKHAPATE
plakhapate@gmail.com
91-9867069587
91-22-27702655

 

To plakhapate:

Glad to hear your wife is doing so well. If her prior PFT data is well documented, then perhaps you should send the "before & after" documantion to one of the companies I have mentioned or some PFT organization. Such recovery from PFT is quite remarkable.

 

Updating posts 43 & 48:

"InterMune, Inc. today announced that it has submitted an electronic NDA with the FDA seeking approval to market pirfenidone for the treatment of patients with idiopathic pulmonary fibrosis. ..."

Read Full Article at: http://www.therapeuticsdaily.com/ne...943008&contenttype=sentryarticle&channelID=26

PS reason why I keep up with their progress is I have had small position (100 sh) in ITMN since Oct2006.

 

To plakhapate: Can you give us an update on how your wife is doing? I hope well, but if so I suspect she did not really have IPF.

Updating post 51 and others:

" InterMune, Inc. (NASDAQ:ITMN) today announced that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) seeking approval to market pirfenidone for the treatment of patients with idiopathic pulmonary fibrosis (IPF) in the European Union (EU). Currently, there are no EMA-approved treatments for IPF in the EU.

IPF is a debilitating and universally fatal disease that affects as many Europeans as Americans, ... Pirfenidone has been granted Orphan Drug designation in Europe. ..."

Details at: http://www.therapeuticsdaily.com/ne...e=688384&contentType=newsarchive&channelID=34

 

Hi
I was diagnosed with IPF last DEC 1st. I signed up to be on InterMunes email fanouts and I received that email yesterday as well and that all looks and sounds promiseing, but what I am wondering is, has anyone gone to Japan to try and get Pirfenidone? I live near Hamilton, Ontario and my Dr said if I could get it he would administer it. So I am curiose about the how to get it.

 

Sorry that is your case. I have owned ITMN since Oct 2006 so have long file on it. Here is part that may interest you from about two years ago:

Shionogi has Japanese, Korean & Taiwan rights. Filed in Japan in March07.* (RoW owned by ITMN)* 18March Shionogi** reported:
• Good Clinical Practice inspections passed at both the clinical sites and at the company.
• Shionogi presented P2&3on 368 cases total at 52 wk “pos effect” (p= ~0.035) at Am. Thoracic Soc. 20May08 and:
• Abstract available at http://www.thoracic.org (Session C95, Publication Page A768).
• Shionogi expects a pirfenidone NDA approval in Japan and launch in their FY2008, (April 2008 til March 2009).
• Top-line results of 72 wk P3 CAPACITY in IPF in January of 2009.
• InterMune to make long term safety study with patients who have completed one of CAPACITY1or2, P3s which have 779 patients total. (95% detect prob. If 50% better; 85% detect p. if 40% better) Forced Vital Capacity is end point (but 2nd is P.Free.Survial)
In may08 in US Shionogi presented good P3 results (first drug to beat placebo on IPF!)

*Purchased all + some IP for $13.7e6 + delayed cost of $53e6 if drug gets to market, but will not pay any royalties, etc. ever.
**Shionogi expects to launch (in Japan) during their FY which ends in March 2009. (Filed the Japanese “NDA” in March08)

BTW, the name of the drug in commercial market in Japan is: Pirespa.

----------------
*I think the March 08 is the correct file date and other is error in my notes.

PS: There is a possibility of getting drugs before approved in cases like IPF with no approved drug. I think it is called "compassionate use." Not sure how it works, but suspect that the drug company will provide the drug, perhaps for free - they get at least some safety data.

 

Thanks I'll look into that. Getting this drug seems the best bet right now while I wait for something better.
Flemming

 

Visit www.intermune.com - you probably have already. I think that the FDA should be acting on their NDA about now. Let me know what you find. - You may be able to buy soon in the USA, but not already.

 

I have tried to get the drug from Intermune but at this time you can't. Intermune did say that they were trying to start a "named patient program" which would allow certain people in Canada to get the drug if they fall into the perameters of the test. There is no time line on that yet but I am on there email list so hope to hear soon. But in the mean time I still want to figure out if I can just go to Japan for it.
Flemming

 

Good news (for us both - you as a patient me as share holder):

"InterMune shares rocketed after the Food and Drug Administration released documents on its Website that included favorable reviews of InterMune's new drug Esbriet. … InterMune is seeking to have Esbriet approved for the treatment of idiopathic pulmonary fibrosis, a deadly lung disease. A final agency decision is expected by May 4. The FDA advisory panel meeting is slated for March 9, … While the FDA is not bound to the decisions of its advisory panels, it generally follows them. Shares of InterMune shot up almost 90% to $27.14 in pre-market trading...." (Quote is combined from two differrent financial reports)

I suspect that the review will be unanimous and strongly recommend approval because the need is so great and idential drug is sold in Japanese market for more than a year.

Now the bad news:
It will be expensive. The current stock price is only a few dollars higher than when I bought more than two years ago, because they spent much more than planned to develop the drug to this nearly approved stage.

 

That is good news.. I have really good drug coverage through work so should be able to get it covered either through our government plan or my work coverage. Even if I have to pay out of pocket at first it will still be cheaper than going to Japan
thanks
Flemming

 

I sold a call open until Ap 17 with strike price of $30 on Itmn today. It will expire after the scientific review report but before FDA acts. just now ITMN traded at $23.36 and is dropping.

 

In a few years, perhaps a competitor to ITLM:

NeoPharm said today it has filed an orphan drug application with the FDA for IL13-PE38QQR for the treatment of Idiopathic Pulmonary Fibrosis. ... IL13-PE exclusively binds to the pulmonary fibroblasts, which express IL13 receptors for selective cytotoxicity, thereby ameliorating all the clinical and histopathological evidence of IPF.

Full article at: http://www.therapeuticsdaily.com/ne...e=690477&contentType=newsarchive&channelID=26