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Why is this significant? While it certainly could be a scheduling conflict I don't think so! First this is during the day and not at the close. Since JJ Bienaime took the helm I believe all quarterly calls have been at 5PM, the only ones during the day have been announcements. The company is due to announce results from their 2A study in poorly controlled hypertension (6R-BH4) any day now so my guess is that is what will happen then. So lets see according to Dew's logic if there is negative news you want to do it on a Friday night, so positive news would be done when? While everyone is expecting the company to be sold this year, I am not convinced of them being sold soon (just my opinion).
[Edit] Mistaken the first couple of calls when JJ took over were at 12:00 but none since.
http://biz.yahoo.com/prnews/070216/sff023.html?.v=81
BioMarin Announces Date and Time Change of Fourth Quarter and Full Year 2006 Financial Results Conference Call and Webcast to Tuesday, February 20 at 11:00 a.m. ET (17:00 CET)
Friday February 16, 4:00 pm ET
NOVATO, Calif., Feb. 16 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN - News News) today announced that it has changed the date and time of its fourth quarter and full year 2006 financial results conference call and webcast from Thursday, February 22 at 5:00 p.m. ET (23:00 CET) to Tuesday, February 20, at 11:00 a.m. ET (17:00 CET).
What: BioMarin Fourth Quarter and Full Year 2006 Earnings Call and Webcast
When: Tuesday, February 20, 2007 at 11:00 a.m. ET (17:00 CET)
U.S. / Canada Dial-in Number: 800.901.5217
International Dial-in Number: 617.786.2964
Participant Code: 35915200
Replay Dial-in Number: 888.286.8010
Replay International Dial-in Number: 617.801.6888
Replay Code: 94899251
Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.BMRN.com. A replay of the call will be archived on the site for at least one week following the call.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio is comprised of two approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, and Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include Phenoptin(TM) (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of poorly controlled hypertension and peripheral arterial disease. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
Naglazyme® is a registered trademark of BioMarin Pharmaceutical Inc.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.
Contacts:
Investors and Media
Eugenia Shen
BioMarin Pharmaceutical Inc.
415-506-6570
Why would sppi settle for not very much?
I don't have any special insight and I know everyone thinks this is a bitter feud between the two companies but my reasoning is as follows:
From listening to calls my impression on how Dr. Raj operates is to try to get something and limit risk (obviously arbitration is risky). An example is rather then get involved in a messy situation with GSK over the paragraph IV challenge for Imitrex the company first did a deal with Par then second settled (OK granted a lot of these get settled before litigation). Spectrum got 5 million and I believe about half of the net profit when its launched.
I think its in everybody's interest to kiss and make up. OK GPC shareholders may think we don't owe them a dime but I know all things being equal I prefer not to have a cloud hanging over me. Further more I think it could be done in a mutually beneficial way.
1-Give Spectrum Shares of GPC stock. Say 100,000 now and 150,000 that vest based on "milestones" being reached. This would be about 7 - 8 million and I don't think it would have any negative impact on GPC the dilution would be almost unnoticed and I think having the cloud removed would probably make it a net neutral situation. Having the stock vest gives GPC some leverage to keep SPPI from complaining (too much anyway).
2-OK everyone will think I am crazy but I really think licensing another of Spectrum's compounds could give GPC a bit more of a pipeline and SPPI certainly isn't in a position to do a big Phase 3 and launch. I think again issuing GPC shares would be a good way, perhaps Spectrum would want some cash too.
I won't ramble. Your posts are pretty knowledgeable so I'd be interested in your view on this.
True to form, SPPI “scooped” the story by submitting its own PR seven minutes before GPC’s.
I think it is all part of Dr. Raj's big strategy to convince the arbitrator that satraplatin is really Spectrum's and not GPC's :). Maybe GPC will require certain wording for any future press releases on satraplatin as part of any settlement. I still think GPC settles before any arbitration decision as I don't think Spectrum would want very much and I don't think GPC wants to be distracted with Spectrum every few months complaining about some aspect of the agreement.
Excel, how about setting up a survey on if/when Barry breaks "The Record"?
Suggestions
A. Before All-star break
B. July 2007
C. August 2007
D. September 2007
E. 2008
F. Go directly to jail do not pass go do not collect 756 Home run ball. OK that sounds a little harsh, perhaps a simple Doesn't do it is better
From a quick look it seems there are a little about 6.8 million Warrants with an Exercise price of .74. Are they all exercisable today? Anyone have a breakdown of the share count and the dates/prices for options and warrants? TIA
I am surprised the union is fighting this. :)
http://sports.yahoo.com/top/news?slug=ap-athletes-steroids&prov=ap&type=lgns
Baseball players move to overturn steroid decision
By DAVID KRAVETS, Associated Press Writer
February 12, 2007
SAN FRANCISCO (AP) -- The Major League Baseball Players Association asked a federal appeals court late Monday to revisit its December decision to allow investigators probing steroids in sports to use the names and urine samples of more than 100 players who tested positive for performance enhancing drugs.
The 2-1 decision in December by the 9th U.S. Circuit Court of Appeals in San Francisco overturned three lower court decisions and could help authorities pinpoint the source of steroids in baseball.
Investigators seized computer files containing the test results in 2004 during raids of labs involved in MLB's testing program. The samples were collected at baseball's direction the previous year as part of a survey to gauge the prevalence of steroid use. Players and owners agreed in their labor contract that the results would be confidential, and each player was assigned a code number to be matched with his name.
Quest Diagnostics of Teterboro, N.J., one of the largest drug-testing firms in the nation, analyzed more than 1,400 urine samples from players that season. Comprehensive Drug Testing of Long Beach, Calif., coordinated the collection of specimens and compiled the data. Comprehensive joined the players in their petition to the appeals court to rehear the case with 15 judges.
If the December decision survives, the players who tested positive could be called before a grand jury and asked how they obtained their steroids.
Federal investigators originally demanded to see the 2003 results for Barry Bonds, Gary Sheffield, who was recently traded by the New York Yankees to the Detroit Tigers, the Yankees' Jason Giambi and seven other players.
When they raided the testing labs for those 10 results, investigators also seized computer files containing the test results of nearly 100 other players not named in the government's subpoena and warrants.
"If the majority's decision is allowed to stand, it will create circuit law giving the government carte blanche to use a warrant for some piece of data on a computer as the pretext for seizing the entire computer and perusing its contents," attorneys for the union and lab wrote.
The testing was part of baseball's effort to determine whether a stricter drug-testing policy was needed. Because 5 percent or more of the tests for steroids came back positive, it automatically triggered the start of testing with penalties in 2004.
The lower courts had declared the use of the data beyond the original 10 names harassment and unreasonable.
There is no timeline for the court to decide whether to rehear the case.
The case is United States v. Comprehensive Drug Testing Inc., 05-10067.
Thanks. That explains some things!
I had thought Biomarin-Genzyme-TKT and the like took a long time to get their drugs in Japan because of difficulty getting enough patients for bridging studies... I guess not!!
Would guess that probably means clinical trials enroll faster in Japan too.
Edit:
And I hate to say it but the FDA doesn't look so bad (on a relative basis) as I thought.
Is my searching accurate and they were granted fast-track in 9/05 and the approval was 1/07. It seems the bridging trial (not sure if that is what they call it ) in Japanese patients was already completed then too so Fast-track = 15 months in Japan?
Appreciate the information. By chance would you happen to know the criteria for fast-track? I'm searching but I'm not as good as Biowatch :).
I did find this don't know if it was posted but amusing
http://seattletimes.nwsource.com/html/nationworld/2003547747_webbirth30.html
Japan's health minister rebuked for calling women "birth machines"
By Kozo Mizoguchi
The Associated Press
TOKYO — Prime Minister Shinzo Abe rebuked Japan's health minister Monday for calling women "birth machines," a remark that caused an outcry in the country.
But Abe dismissed calls for the embattled minister to resign over the comment.
"I reprimanded him severely," Abe said, adding that he saw no reason for Health Minister Hakuo Yanagisawa to step down. "From now on, I hope he will remain fully devoted in his job and obtain the people's understanding."
Abe's comments came just hours after a group of female lawmakers called for Yanagisawa's removal.
The health minister triggered outrage over the weekend by describing women as "birth machines" in a speech on the country's falling birthrate. He later retracted the remarks.
"The number of women between the ages of 15 and 50 is fixed. The number of birth machines (and) devices is fixed, so all we can ask is that they do their best per head," Yanagisawa was reported as saying in the speech Saturday.
The government has been scrambling to implement measures to persuade couples to have more children amid concerns about the country's shrinking population. Japan's birth rate was 1.26 babies per woman in her lifetime in 2005, a record low and far below the level needed to keep the country's population steady.
A proposal adopted in June calls for increasing child care, promoting greater gender equality, and encouraging companies to be more flexible in allowing staff to take care of family responsibilities.
But the high cost of raising children, as well as the lingering notion that women should quit their jobs after giving birth, has meant many opt to have few or no children.
Any body know anything about the Japanese regulatory process? Are they like the US with a (more or less) fixed time frame for review, if so how long is the process from submission or is it open-ended? Also do they have an expedited review process of some sort?
TIA
They also said that treatments customized to each patient, (e.g., an anti-idiotypic antibody treatment for B cell lymphoma that targets the specific B cell causing a patient's lymphoma,) tends to work better, but is much more expensive and technically difficult to accomplish than a one-size fits all treatment (like the anti-CD20 antibody Rituxan.)
I don't follow them closely but isn't that what Favrille is doing? Not sure where they stand now but a couple months ago they had a big setback. I seem to recall another company in a similar stage (Phase 2 or 3) working on something similar too but can't recall who.
If you follow Dew's advice on DNDN, you will have lost on a huge upcoming opportunity.
After reading the board for a couple months now I think I get the general strategy that Dew takes. I can't speak for him but from my perspective it is almost like a Warren Buffet Biotech strategy (if he invested in Biotech which he doesn't). Buy value, Don't swing for the fences, know what you invest in well a corollary may be when you are sure invest heavily (OK this one he has pretty much stated) and buy for the longer term.
I can't say I am innocent of not taking risks but I learned from some past mistakes. I think even if Provenge gets approved without needing additional data it fails as a "Value Biotech" at this stage so would be speculative. I for one am staying away. If it succeeds and pays off big congrats, I'll wait for the next pitch since no strike gets called for taking a pitch even if it is right down the middle (DNDN at best is on the corner) :).
Updates: IPF competitive landscape, Shionogi Pirfenidone, Actimmune-BI relationship, financials, milestones, and misc. cleanup, corrections (IPF vs. PF prevalence information)
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 (for PF) 128,000, Incidence 48,000 each year http://tinyurl.com/u6kgo, journal abstract http://tinyurl.com/tqd6m IPF prevalence incidence 83K US and 30K incidence.
• 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).
Competitive Landscape for IPF
1. Genzyme (collaboration with what was CAT) in Phase 1
2. Bosentan (Tracleer) from Actelion, Phase 2 failed in their endpoint proceeding with Phase 3 different end point expected to initiate in early 2007
3. Imatinib (Gleevec), I believe it failed Phase 2 not sure of status going forward.
4. Sildenafil (Phase 2) http://www.clinicaltrials.gov/ct/show/NCT00352482
5. Pipex (Tetrathiomolybdate) Phase I-II completed http://clinicaltrials.gov/ct/show/NCT00189176 Company filings say plans to initiate Phase 3
6. FivePrime Collaboration http://tinyurl.com/yz444l
7. Others?
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) In Q4 2006 Earnings Call said increased price 6.8% effective 1/07 to 206 for ½ ml vile. Presentation said almost 65,000/yr for fully complaint patient. 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) With 83K prevalence and 30K incidence in US company estimates that 50K would be mild to moderate and 80% of newly diagnosed patients would fall into that category. Company is targeting the “mild-to-moderate” 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.
• Boehringer Ingelheim relationship re Actimmune (Imukin) http://tinyurl.com/yh9pez
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. Q4 2006 company continues to state will only report if interim analysis causes trial to stop for efficacy or futility.
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. Company states on Q4 2006 call that it is working on expanding IP and have certain activities and progress (very vague could be anything from follow-on compound to exploring new patents
• 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
• Shionogi Phase 3 Top Line PR http://www.shionogi.co.jp/ir_en/news/detail/e_061222.pdf
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 585 patients
• Randomized first patient April 27, 2006
• Expect enrollment to conclude Q4 of 2007, with top-line data expected in early 2009
• Exploring European partnering opportunities
• Enrollment proceeding “Very Well” (according to Q4 call) company still maintains enrollment expected to complete in Q4 2007.
• Differences between CAPACITY and Shionogi Phase 3 (Company presentations) Shionogi 250 patients, ours I-260, II-325, roughly same. Length Shionogi-52 ours 60 weeks. Dosing in Shionogi low dose 400mg TID high 600 TID, CAPACITY I-800mg TID, CAPACITY II 800mg and 400mg both TID, if account for avg. body weight of US/EU vs. Japan about 30mg/kg in each. Endpoint Shionogi change in Vital Capacity ours Change in Percent Predicted Forced Vital Capacity. Comparable measures of lung volume. Other difference is the patient population, Shionogi is Japanese ours is mainly NA and EU do not know of any metabolism difference in population or Pirfenidone between two groups.
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
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
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
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.
• Phase 1A Trial Information http://tinyurl.com/32lvtz
1. One clinical trial site in Europe and will enroll approximately 74 healthy volunteers.
2. First patient dosed early January 2007.
3. Single-dose ascending dose in healthy volunteers.
4. Expects to complete the currently running Phase 1a study of ITMN-191 in healthy subjects in the first half of 2007.
5. Schering and Vertex held 1A until 1B that could be something we’ll do and have not disclosed plans yet.
• Phase 1B Trial Information (Press release/call) “InterMune plans to evaluate ITMN-191 in a Phase 1b randomized, double- blind, placebo controlled, multiple ascending dose study in patients infected with chronic hepatitis C virus. ITMN-191 will be administered to treatment naïve patients for 14 days and the study will include a cohort of non- responder patients. InterMune and its partner Roche expect to announce initial viral kinetic results from the Phase 1b trial in the second half of 2007.”
1. Not disclosing number of patients at this point
2. A specific cohort of non-responders in our mono-therapy studies so we can understand is there any difference in kinetics between non-responders and naïve patients. Ours will be the first in systematic fashion look at those directly.
3. We have very clear definition of what it means to be non-responder as operational defined in protocol. Will be a homogeneous group. (in response to question about type of non-responders)
• Other 191 Information
1. Looking at addition of ribavirin toxicology and PK (preclinical).
2. Plan to move quickly into Phase 2 of a design and duration of what would want to do in Phase 3 which you may imagine would include PEG-Interferon and Ribavirin.
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/securities 214.5 million (end of Q4 2006)
• 170 million convertible. Coupon rate .25%. 2011 maturity. Conversion Price $21.63.
• Shelf registration (http://tinyurl.com/29rsqc) for up to 175 million filed 12/28/2006
• 33 million shares outstanding
• 2007 Guidance
o Revenue 70– 90 million.
o COGS 21-23%.
o R&D 110-125 million. Including 5-10 million for est. FAS 123R.
o SG&A 35-45 million including 5-10 million for FAS 123R.
• 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/Medical Presentations
• Interim look in INSPIRE. Company said likely would not disclose unless it was material (i.e. trial stopped for efficacy or futility) 2 in protocol not stated if any have occurred.
• Present 8 abstracts from Pulmonology research at ATS in May
• Present 3 abstracts of preclinical experience of 191 at EASL in April
• Submitted several abstracts on preclinical data for 191 for DDW in May
• 2H ’07 Phase 1B data
• 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 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 (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)
b. Pirfenidone:
i. Price 30K (estimate based on discount of Tracleer pricing in PAH)
ii. Using incidence figure 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.
I'd recommend listening to the Merrill Lynch conference to anyone who hasn't already and who follows BMRN.
JJ spent a lot of the time on BH4 Cardio Vascular and there was a Q&A period at the end.
I got the impression Phenoptin may be priced higher then, at least I, was expecting and perhaps even some analysts.
Everyone loves to speculate but my guess (no more) is BioMarin doesn't get bought out this year. Also keep in mind while the Uncontrolled Hypertension study results are do this month this is just a 2A study and the company has already indicated (assuming positive results) they'd do a 2B (dose ranging) study after. So my guess is the 2A would offer an idea at efficacy but not the clear picture on it.
Before this financing I believe InterMune's stake in Targanta was about 20%. They may have an additional interest outside of the equity too (can't recall from memory).
I expected there to be some disappointment with the Actimmune number yesterday and the lowered guidance (especially when you factor the price hike in Actimmune and they lumped in some of the amortization of the Roche milestone in the revenue range). Its good investors aren't focusing on the (declining) revenue number it says something to Mr. Welch convincing investors that InterMune is a pipeline story as opposed to revenue [I am looking forward to meaningful revenues].
http://biz.yahoo.com/prnews/070209/nyf023.html?.v=83
Targanta Announces $70 Million Series C Financing
Friday February 9, 7:00 am ET
Capital to Fund Antibacterial Oritavancin NDA and Advance Clinical Trials for Multiple Indications
CAMBRIDGE, Mass., Feb. 9 /PRNewswire/ -- Targanta Therapeutics, a privately held biopharmaceutical company developing antibacterial drugs, announced today that it has raised $70 million in a Series C venture round. The financing was led by Brookside Capital, Skyline Ventures, Radius Ventures, and OrbiMed Advisors, and included existing investors Seaflower Ventures, VenGrowth Advanced Life Sciences Fund, and the Canadian Medical Discoveries Fund, among others.
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The company intends to utilize this capital to prepare and submit the New Drug Application (NDA) for its lead product, oritavancin, for the treatment of complicated skin and skin structure infections (cSSSI). Targanta expects to file the NDA in the second half of 2007. In addition, the Series C will help fund new oritavancin clinical trials in pneumonia, bacteremia and single dose treatment for cSSSI.
"We are pleased that the new and existing investors participating in this round recognize the significant opportunity to develop and commercialize novel antibiotics, like oritavancin, to address the critical unmet need to fight the growing prevalence of serious gram-positive infections, including those caused by multi-resistant strains like MRSA (Methicillin Resistant Staphylococcus aureus)," said Mark Leuchtenberger, President and CEO. "With two successfully completed Phase 3 trials in cSSSI and a pre-NDA meeting with FDA behind us, this financing should greatly enhance our ability to file an NDA for oritavancin this year and prepare for a U.S. launch, if approval is received. Additionally, this funding allows us to leverage oritavancin's broad gram- positive spectrum activity and superior in vitro potency to pursue its development in other indications, thus providing Targanta with multiple opportunities to build a substantial franchise around this product."
In relation to the Series C financing, Jay Venkatesan, Director, of Brookside, and Eric M. Gordon, Ph.D., Partner, Skyline, have joined Targanta's Board of Directors.
"The gram-positive bacterial market could approach several billion dollars in the U.S. over the next 10 years," said Dr. Gordon, who also founded Vicuron, an anti-infectives company acquired by Pfizer in 2005, "and we believe that oritavancin will compete extremely well within that market. Skyline invested in Targanta because we believe oritavancin is the gram- positive antibiotic with the broadest spectrum against resistant organisms and the highest potency. In addition, because of oritavancin's mechanism of action, we believe that gram positive bacteria are less likely to develop resistance to oritavancin than to other antibiotics."
About Oritavancin
Oritavancin, Targanta's lead product candidate, is a once-daily, semi- synthetic glycopeptide antibiotic with rapid bactericidal activity against all clinically relevant serious gram-positive infections, including multi- resistant strains. Oritavancin's multiple targets and mechanisms of action work against the development of resistant strains, which is important when treating serious gram-positive infections. To date, over 1,500 individuals have received oritavancin in clinical trials, including two large multicenter Phase III studies performed by former developers Eli Lilly and Intermune to demonstrate oritavancin as an effective and safe therapy for complicated skin and skin structure infections. Targanta expects to file a New Drug Application (NDA) for oritavancin with the U.S. Food and Drug Administration in the second half of 2007.
About Targanta Therapeutics
Targanta Therapeutics is a privately held biopharmaceutical company developing and commercializing antibacterial drugs to treat serious infections in the hospital setting. Its pipeline includes an array of antibacterial agents in various stages of development. The company has operations in Cambridge, MA, Indianapolis, IN, and Montreal, Canada. For further information about Targanta, visit the company website, www.targanta.com.
At the risk of being too basic here is a Street.com article with basic terminology
http://www.thestreet.com/pf/funds/investing/10337421.html
Investor Biotech Primer
By Marc Lichtenfeld
Senior Columnist
2/8/2007 12:07 PM EST
URL: http://www.thestreet.com/funds/investing/10337421.html
Like all industries, biotech has its own jargon. Newcomers to the sector can find themselves struggling to get through just one paragraph of a press release without having to look up several terms.
Here is a primer on some of the key phrases, acronyms or statistical concepts that you'll need to know when investigating a biotech stock idea.
ANDA -- Abbreviated New Drug Application. This is an application to the Food and Drug Administration (FDA) to seek approval of a generic drug.
For example, last week Mylan Laboratories (MYL) announced that its ANDA for generic Valtrex received tentative approval from the FDA. Valtrex, for the treatment of herpes, is made by GlaxoSmithKline (GSK) .
NDA -- New Drug Application. This application seeks approval for a new drug before commercialization. Included in the NDA is safety and efficacy data, proposed labeling and manufacturing methods.
IND -- Investigational New Drug. This is the process in which companies apply to the FDA for permission to begin trials of a new drug in humans. Pharmion (PHRM) announced last week that the FDA accepted the company's IND for an oral version of Vidaza, a treatment for patients with myelodysplastic syndromes. The new drug will enter phase I trials shortly.
Phase I Studies -- The first study of a drug in humans. The trial is usually conducted in a small number (20-80) of healthy people. According to the FDA, sufficient information about the drug's pharmacokinetics and pharmacological effects should be obtained in order to design phase II studies.
Phase II Studies -- These studies are used to test the effectiveness of the drug and include patients with the disease that the drug is intended to treat. Phase II trials usually include several hundred patients or fewer. In rare disorders, the number may be less than one hundred.
Phase III Studies -- Larger studies with hundreds or thousands of patients. Safety and efficacy from these trials are what the FDA uses to determine whether a drug will be approved or rejected. Occasionally, the FDA can order more studies be conducted before reaching a decision.
It's important to keep in mind that drugs can be in different phases for different diseases. For example, Genentech's (DNA) Avastin is approved for colorectal and non-small-cell lung cancer. However, the drug is in or about to begin phase III trials for a host of other cancers, including first-line metastatic breast cancer and first- and second-line ovarian cancers. Avastin is being prepared to enter phase II for extensive small-cell lung cancer.
P-value -- A measure of how statistically significant a trial's results are. In other words, it's the percentage chance that a result is not true. The standard "line in the sand" p-value is 0.05, meaning 5%. Results with a p-value of greater than 0.05 are considered statistically insignificant. The results have a greater than 5% chance of being false. Let's take a look at a real-world example.
Last year, Celgene (CELG) compared a combination of its thalidomide with melphalan and prednisone vs. just melphalan and prednisone in patients newly diagnosed with multiple myeloma. The company reported that the combination that included thalidomide led to improvement in overall survival. The p-value was 0.001 -- meaning the results had only a 1/10 of one percent chance of being false.
Contrast those results with Amgen's (AMGN) trial for Aranesp for cancer-caused anemia. Amgen released the results in its fourth-quarter earnings report. In the trial, 18% of the patients taking Aranesp needed red blood cell transfusions vs. 24% in the placebo group. Sounds promising, right? But the p-value was 0.15, indicating there was only an 85% certainty that the results were accurate. There were some other problems in the study, but this example shows that you can have positive results, but without a low p-value, they're meaningless.
Hazard Ratio -- The relative risk of a patient in the experimental group compared to the control arm. If a drug increased survival by 100%, the hazard ratio would be 0.01. If there was no increase in survival, the hazard ratio would be 1. So clearly, a lower number is better. Returning to our Aranesp example -- after 16 weeks the hazard ratio was 0.89, indicating only a 12% increase in survival.
These are just a few of the basic terms you should know when investing in biotech stocks. I'll cover others as time goes by. In the meantime, feel free to email me with your questions or write Adam Feuerstein, who fields questions each week in Feuerstein's Biotech Mailbag.
Here are some notes from the call. Excuse any misspellings some of the terminology was above my level of knowledge
. Encouraged by anti-viral activity even at lowest dose significant anti-viral activity. Validates unique mechanism, inhibiting protein NS4A.
. Does have dose limiting toxicity that would limit dose escalation.
. Early signs of elevation of serum creatinine, reversable after dosing completed.
. Next generation some in preclinical development. No date set on when next would move into the clinic. Think can get IND filing in Q1/Q2 2008 if get lead candidate soon.
. Talked about other programs, broad pipeline, etc.
[JS-Cowen] What promotion increased creatinine? See it preclinically? Route of elimination for drug (through Kidney?)
Roughly 6 of 8.
Did not see any sign in preclinical tox. Difference in that there is multiple dosing. Elevation about .4 - .5 mg / deciliter.
Primarily metabolized in liver, exact answer not 100% known how much through urine appears most through liver.
[BH-CIBC] 1095 and backup hemotypes, how close? Efficacy of 1095 vs. 806?
Different pharmacore. EC-50 of 9132 14 nM (Genotype 1) vs. 1095 and related compounds EC-50 20-30 nM
[JC-Susq] Mechanism of Action for elevated creatinine level? How proceed with GILD? Lost about a year?
Point to it being causing some damage to renal tubule. Don't see signs of interstitial nephritises. Trying to elicit exact mechanism still. Most likely not related to target of mechanism of action. Believe effect most related to structure of 9132 then mechanism. [JC]as passing through kidney believe some binding their [ACHN] we believe so.
No substantive change. Achieve proof of concept with backup compound, defined by clearing 3 hurdles (>= 1 log Viral log reduction, acceptable safety profile, movement into Phase 2).
Think so maybe conservative since learned things and may do things different such as go directly into patients. No one else as far as we know with advanced program against this target.
[GF-Wachovia] Why tox not observed in Phase 1A. Talk about doses examined? Backup compounds Achillion through Phase 1, Gilead Phase 2 on?
Phase 1A 10mg up to 2400 mg as single dose. Didn’t see tubular issue that saw in multiple dose. In multiple dose 300mg BID.
Not quite we will do whats best for drug. Far more collaborative effort “more then likely with Gilead in the drivers seat”
[JF-Morgan Stanley] Any other side effects that saw to see mechanism of toxicity outside kidney? Anything that can do differently?
No didn’t see anything else that would go along with this.
Data is pretty recent. Starting to look in other models to see what is predictive of what we saw. Not mechanism related but more hematite related.
[TW-Piper] Viral load on trajectory comparable efficacy to class? Any non-responders? Did meet the first criteria (positive efficacy data)?
806 clearly demonstrated anti-viral activity. Probably more so then expected at low dose activity. Didn’t see signs of viral rebound.
Depends on how you define it. All patients who received drug had greater viral load drop then 2 who received placebo. 4 above 1 log, 4 below but higher then placebo. Believe higher dosing would see much stronger activity.
Till finish analysis no answer.
[MS-R Capital] Novel mechanism of action, NS3? telaprevir also 4a? Financials Shares/Cash?
GS-9132 binds non structural protein NS4A co factor for NS3 protease. Shown after our compound binds to it, disrupt formation of replicate complex.
No active site inhibitor of NS3 protease.
15.5 million [preferred convertible soon?] converted at IPO. Cash low 60 million range.
[DK-Credit Swiss] Gilead experience, did they see why and how to fix it?
No net yet. Working quite closely with them reviewing data.
> MAXY – I still think he (Russell Howard) is on the arrogant side though...<
I respectfully disagree on this. Perhaps you are interpreting as arrogance what is simply confidence and skilled public speaking.
I'll give you both (good speaker and highly confident). My concern is more is he overly promoting. I haven't listened to as many of their calls lately but about a year or so ago when I was listening to more of them he would be critical of wall st.'s valuation of the company (I don't have a problem with that I like all my CEOs to think they are under valued) just his tone/manner seemed to talk down to the people who end up recommending (or not) his company. Kind-a like Fred Price did (at BioMarin).
>Maybe because he criticized my InterMune a year or two ago :) <
What did he say about ITMN?
I believe he was talking about the PEG-Actimmune program Maxygen was working on. Wish I could remember the exact wording the tone sticks out though it was to the effect of "Get off their *ss*s and move the program forward" [my wording not implying he said that] Of course InterMune has no interest in burning (more) cash unless Actimmune is successful in some indication and with patent life till like they year 3000 (actually 202? or other) I don't think they will move it forward without good reason.
I actually heard that and thought it was pretty good too. I would have recommended it but thought only HCV knowledge naive (like me) could benefit from it. I thought very little of HGS's program until hearing him talk a bit more about it which bumped up my opinion of it a few notches (I thought their program was low on efficacy didn't think too much about the improved compliance/convenience it would give).
I still think he (Russell Howard) is on the arrogant side though... Maybe because he criticized my InterMune a year or two ago :).
OK I was wrong I saw this and thought to see if anyone heard the call and posted anything. (I had other priorities besides just saw it now). Achillion is down big in after hours!
http://biz.yahoo.com/prnews/070208/nyth185.html?.v=46&printer=1
Gilead and Achillion Announce Positive Antiviral Activity of NS4A Antagonist in HCV, But Discontinue GS 9132 (ACH-806) Development
Thursday February 8, 4:24 pm ET
- HCV Collaboration to Continue With Potential New Development Candidates -
FOSTER CITY, Calif. and NEW HAVEN, Conn., Feb. 8 /PRNewswire-FirstCall/ -- Gilead Sciences (Nasdaq: GILD - News) and Achillion Pharmaceuticals (Nasdaq: ACHN - News) today announced their decision to discontinue the development of GS 9132, also known as ACH-806, for the treatment of hepatitis C viral (HCV) infection, based upon preliminary data from a Phase 1b/2 trial. Preliminary data from the first cohort of the Phase 1b/2 trial indicated that the compound demonstrated antiviral activity, validating the novel anti-HCV mechanism that involves inhibition of a viral protein called NS4A, which binds to a portion of HCV protease. However, based on small elevations of serum creatinine (a marker of kidney function), which were reversible after completion of dosing, Gilead and Achillion have elected to shift their focus to the evaluation of other NS4A antagonists developed by Achillion to identify a lead candidate for development.
"GS 9132 has demonstrated antiviral activity in patients with genotype 1 HCV infection. Even at the low dose studied, we observed significant reductions in hepatitis C viral load. This validation of the mechanism of action is encouraging as we evaluate next-generation compounds for potential development," said Norbert Bischofberger, Ph.D., Executive Vice President, Research and Development, Gilead Sciences. "We look forward to our continued collaboration with Achillion."
"As part of our collaboration with Gilead, we have worked diligently to generate a number of compounds belonging to a different chemical class that demonstrate the same mechanism of action and similar in vitro potency to GS 9132. One of the most promising of these has been designated by Achillion as ACH-1095, and we are evaluating this and other compounds in preclinical studies to determine if one has the right profile to advance into clinical development," stated Milind Deshpande, Ph.D., Chief Scientific Officer of Achillion. "Our goal is to develop a novel, efficacious and safe therapeutic for HCV, and the data indicate that candidates with this mechanism may be complementary to both protease and polymerase inhibitors, as well as interferon therapies."
The GS 9132 Phase 1b/2 trial was a double-blind, randomized, placebo-controlled dose-escalation study initiated in 2006. The goal of the trial was to evaluate the antiviral activity, safety and pharmacokinetics of GS 9132 in patients with HCV genotype 1 infection. Gilead and Achillion are continuing to analyze the data from this trial. Following completion of analysis, these data will be submitted for possible presentation at an upcoming scientific conference.
In November 2004, Gilead and Achillion established an agreement granting Gilead worldwide rights for the research, development and commercialization of certain Achillion compounds for the treatment of hepatitis C. GS 9132 and ACH-1095 are small molecule inhibitors of HCV replication, which target a viral protein called NS4A. NS4A antagonism is a novel mechanism of action for HCV treatment distinct from that of protease or polymerase inhibitors currently in development. GS 9132 and ACH-1095 were discovered by Achillion, and the company completed the initial work necessary to move GS 9132 into clinical development.
Conference Call Access Information
Achillion will hold a conference call and simultaneous webcast today, Thursday, February 8, 2007 at 4:45 p.m. Eastern time. To participate in the conference call, please dial (877) 707-9632 in the U.S. or (785) 830-7982 for international callers. A live audio webcast of the call will be accessible at http://www.achillion.com, under the Investor Relations section of the website. Please connect to Achillion's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.
A replay of the webcast will be available on http://www.achillion.com through March 8, 2007. Alternatively, the replay will be available starting at 8:00 p.m. Eastern time today through 11:59 p.m. eastern time Thursday, February 15, 2007 by dialing (888) 203-1112 or (719) 457-0820. The replay passcode is 4731223.
About Hepatitis C
Hepatitis C is a viral liver disease, caused by infection with the hepatitis C virus. Globally, it is estimated that more than 170 million people have chronic hepatitis C, including about three million in the United States. Chronic hepatitis C is a leading cause of cirrhosis, a common cause of hepatocellular carcinoma, and is the leading cause of liver transplantation in the United States.
About Gilead Sciences
Gilead Sciences is a biopharmaceutical company that discovers, develops and commercializes innovative therapeutics in areas of unmet medical need. The company's mission is to advance the care of patients suffering from life-threatening diseases worldwide. Headquartered in Foster City, California, Gilead has operations in North America, Europe and Australia.
About Achillion
Achillion is a biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for infectious diseases. Achillion is currently developing treatments for HIV infection, chronic hepatitis C infection and serious hospital-based bacterial infections.
As an investigational compound, ACH-1095 has not yet been determined safe or efficacious in humans for its ultimate intended use.
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including statements with respect to completion and success of Achillion's preclinical studies and clinical trials of Achillion's drug candidates. Among the factors that could cause actual results to differ materially from those indicated by such forward-looking statements are: unexpected regulatory actions or delays; uncertainties relating to results of clinical trials, including additional data relating to ongoing clinical trials; Achillion's ability to obtain additional funding required to conduct its research, development and commercialization activities and Achillion's dependence on its collaboration with Gilead Sciences. These and other risks are described in the reports filed by Achillion and Gilead with the U.S. Securities and Exchange Commission, including their Quarterly Reports on Form 10-Q for the quarter ended September 30, 2006.
All forward-looking statements reflect Gilead's and Achillion's expectations only as of the date of this release and should not be relied upon as reflecting the parties' views, expectations or beliefs at any date subsequent to the date of this release. Gilead and Achillion anticipate that subsequent events and developments may cause these views, expectations and beliefs to change. However, while Gilead and Achillion may elect to update these forward-looking statements at some point in the future, they specifically disclaim any obligation to do so.
For more information on Gilead Sciences, please visit the company's web site at http://www.gilead.com or call the Gilead Public Affairs Department at 1-800-GILEAD-5 or 1-650-574-3000.
For more information on Achillion Pharmaceuticals, please visit the
company's web site at http://www.achillion.com or call Achillion at
1-203-624-7000. ACHN-G
Gilead Contacts: Achillion Contacts:
Amy Flood, Media Kari Watson, Media
(650) 522-5643 (508) 647-0209
Susan Hubbard, Investors Mary Kay Fenton, Investors
(650) 522-5715 (203) 624-7000
The Actimmune number is a bit disappointing (they tried to explain it a bit) lets see how much the market cares about this.
They (as usually) gave some but not too much detail and wide ranges.
Here is the pertinent 191 info from the PR
-- InterMune expects to complete the currently running Phase 1a study of ITMN-191 in healthy subjects in the first half of 2007.
-- InterMune plans to evaluate ITMN-191 in a Phase 1b randomized, double- blind, placebo controlled, multiple ascending dose study in patients infected with chronic hepatitis C virus. ITMN-191 will be administered to treatment naïve patients for 14 days and the study will include a cohort of non- responder patients. InterMune and its partner Roche expect to announce initial viral kinetic results from the Phase 1b trial in the second half of 2007.
Thats probably it for me for today. I'll try to update the readme sometime after the call hopefully within the next day or two.
Just a joke. Didn't mean to imply anything by it.
Got sense not many follow InterMune on this board (some follow the PI program but not so much the company) so posted there.
Appreciate all the info iwfal and Biowatch!
From another board I won't name so I don't get banned :)
So tomorrow we should (hopefully) get updates on a lot of things. I don't expect anything earth shocking (but they tend to some how happen to announce news the day of calls both good and bad).
Here are some things I am looking forward to being updated in the PR or the call
1-191 status and time table and hopefully design of trial.
a. I then the market expects that 191 is in Phase 1A doubt if we'll here anything about side-effects/safety etc. At best an "in general" type of response.
b. The dosing (amount and intervals being examined).
c. As far as time table I think Phase 1B data in Q3 will be announced.
2-Pirfenidone
a. I am hoping we get a formal wording of trial enrollment ahead of expectations Q2/Q3 completion. Actually I hope it is end of Q1 but I don't think they would say that even if it is. I guess my aggressive enrollment expectation may be off.
b. Maybe we can get some comment on "general conduct of the trial". Dan Welch has in the past made a general conduct of trial comment on the INSPIRE trial so perhaps he'll do one hear or they will PR a comment.
c. The big one (for me) hopefully someone will ask it. What are the thoughts on partnering outside of US, given the good top-line Shionogi results? It is conceivable the company takes advantages and announces a partnership deal (but I highly doubt it) and at the other extreme (which I'd love to hear) the company could say something to the effect of "given the added funding with the Roche collaboration, our stronger financial picture, the rapid and advanced nature of the CAPACITY trials and most importantly the exceptional results Shionogi had in their Phase 3 we have elected to pursue developing Pirfenidone on our own in Europe. We believe with a small infrastructure and a modest sales force this presents a significant revenue opportunity in the not to distant future. We have seen Genzyme, Actelion and other companies develop therapies for rare diseases with modest invest and achieve highly profitable returns in a short period and are excited at the potential for Pirfenidone to do the same. Having said that should someone overwhelm us with an offer to market it in Europe we would certainly listen" (OK, OK, I can be somewhat of an optimist can't I?)
3-Actimmune
a. I don't really expect much if anything to be said new here. Other then sales which I would guess at 22 million for the quarter. Maybe a "general conduct of the trial" update.
4-Early Stage pipeline
a. While it is possible, I think it unlikely that InterMune reveal the nature of their preclinical targets.
5-Financials
a. We should get a very vague revenue number for Actimmune for '07. If I were to guess I would say 60 - 90 million.
b. We should get forecasts for R&D, G&A and general financial picture.
c. What I am hoping is someone pins down the company on the shelf and their plans. Specifically I'd like to know if they can retire any of the existing convertible at a discount to current share price, and if they plan to do so. I'd also be interested if this means they are looking to do a deal more near term or as mentioned earlier pursue Pirfenidone development in Europe alone, etc.
Thanks Biowatch! I'm sure I can find the schedule for upcoming meetings somewhere from one of those links if not I know who to ask :)
OT: Hiding security warnings. I think this will work if you don't like getting those balloon pop-ups and security shield with an x through if you don't have the firewall and anti-virus in what windows thinks is an "up-to-date" state.
Over an empty area or the clock on the task bar right click and select properties. Then on the property sheet at the bottom where it has "Hide inactive icons" their is a "Customize..." button. If you press it'll show a bunch of icons (presumably recently used ones) and what you want to have done with them. For the windows security shield I changed it to "Always hide" and I haven't noticed getting any of those pop-up warnings since!
Are ODAC transcripts publicly available, for that matter advisory committee's in general? If so, can someone point out a link? TIA
I was wondering the same thing about Dew. I guess it is better his computer has troubles then him.
Speaking of computer troubles... After seeing my (granted older) computer slow down I went through the obligatory reformat and reinstall a bit ago. I came to a big realization, that XP Service Pack 2 does something really bad to performance! I think it is something related to svchost and how it does auto-updates. I figured a work-around that helps in that I turned off auto-updates and manually do them when I want my PC to slow to a crawl :).
I am not a big conspiracy theorist but if I were in Microsoft's shoes and I was coming out with a new OS and wanted everyone to upgrade what better way then to make them dissatisfied with their current environment... Since SP2 was an update most people did what better way. Of course I am not a conspiracy theorist but perhaps Microsoft had some of their 2nd tier programs work on this aspect.
Yeah cyberonics did get the device approved on appeal (the panel had recommended approval before the non-approvable I believe). I only remember it because it ranks high in my list of entertaining conference calls:
Some of the 8k
http://yahoo.brand.edgar-online.com/EFX_dll/EDGARpro.dll?FetchFilingHTML1?SessionID=Z1NsjDYNNDh4Mrg&...
We are shocked and bewildered by FDA’s decision to ignore its expert Advisory Panel’s recommendation,” commented Robert P. (“Skip”) Cummins, Cyberonics’ Chairman of the Board and Chief Executive Officer. “FDA’s Center for Neurological and Restorative Devices had no prior depression experience before the VNS submission and as a result, FDA deputized four of the seven voting members of its expert Advisory Panel. All four of these deputized members were psychiatrists, including three who also serve on the antidepressant drug Advisory Panel. All of the reasons cited in yesterday’s not-approvable letter were addressed at the Panel meeting on June 15 prior to the Panel’s vote recommending approval. FDA has now chosen inexplicably to ignore not only the recommendation of its panel of experts, but also the strong recommendations of numerous psychiatric thought leaders and the compelling testimony and needs of people with treatment-resistant depression who today have no long-term treatment for their lifelong and life-threatening illness.”
Drugmaker Roche Raising R&D Spending
This can't be a bad thing for InterMune :)
http://biz.yahoo.com/ap/070205/switzerland_roche.html?.v=1&printer=1
Monday February 5, 7:28 am ET
Drugmaker Roche Boosting Spending on Research and Development, Forming 2 New U.S. Units
BASEL, Switzerland (AP) -- Drugmaker Roche Holding AG said Monday it would increase its spending on research and development this year, partly through the creation of two new units based in the United States.
The Swiss-based pharmaceutical company, whose products include the flu treatment Tamiflu, said it would increase its total research spending this year from the 6 billion Swiss francs (about $4.8 billion) it spent in 2006. It said spending would rise even further in the next years. It did not provide precise figures.
Roche said the new research model would be organized around five centers combining development, regulatory approval and marketing for each of its drug lines.
The cancer drugs unit will be based in Nutley, New Jersey, the headquarters of the company's U.S. subsidiary, Hoffmann-La Roche Inc.
Research focused on virology and inflammation will be done in Palo Alto, California, home of U.S. biotechnology company Genentech Inc., which is majority-owned by Roche.
The development of drugs for metabolic diseases, like diabetes, and central nervous systems, will be located in Basel.
Roche shares gained 0.6 percent to 238.30 francs ($191.02) in Zurich trading.
bladerunner,
I posted the royalty info in my reply to onco. I also reference the Altarex filing (thats who United made the deal with and they later where acquired by ViRexx) where you can see the terms.
I posted earlier to someone that I though REX is diluting too much.
BTW, I am trying to build a position in SPPI so I'd appreciate if you don't post good news about it for a while :) (just kidding of course)
I follow United pretty closely and so REX indirectly (I have a small position). If you follow REX for things other then OvaRex I can't help you too much as far as talking about OvaRex I would be interested in discussing the merits (even hearings PGS arguments against it).
As far as royalties go:
http://messages.finance.yahoo.com/Stocks_%28A_to_Z%29/Stocks_V/threadview?m=tm&bn=26969&tid=...
... basically 7% < 500M, 8% to 1B and 9% over 1B. Note also that UTHR does not have world wide rights...
As far as merits of IMPACT trials reaching success. I've come to the conclusion its a toss up :) (so I am still on the fence). My dilema is basically the data (from the Analyst Data) would suggest a mean well above 20 months so if Placebo comes in around 11 months (heck even 15 months) then I think BOTH trials succeed. The problems (I have) are the following:
1-Phase 2B was powered for a much higher TTR then was observed and from what I can tell other trials have reported (but its hard to know the exact patient populations). This one scares me the most.
2-Very small subset made up the SFLT group in the Phase 2b study which is the basis for the IMPACT trials.
3-ViRexx in their PR says about the success of MAB's when they reach Phase 3 (citing a Journal of Nature article I believe) however I believe the same article notes that murine have very poor success (I believe Zevilin is a murine MAB).
4-A paper by MarkMan published early last year I believe reports the correlation between CA-125 level and TTR that would seem to give some explanation for the improved results in what comprises SFLT subset. However the trials referenced have additional therapy so it isn't clear how much benefit patients receive from the other therapy.
Anyway I am long United for the Remodulin franchise. I would think United is a better way to play OvaRex given (minimal) royalties REX gets and it will be some time until meaningful revenue come ViRexx's way but what do I know REX certainly could have much more speculative increase on success in IMPACT trials. I just don't expect them to see meaningful revenue anytime soon unless the deal they did fell through and they still have the EU (and other) territories to license.
Saw this posted (I don't agree with everything but it sounds competent)
http://www.stockhouse.ca/bullboards/viewmessage.asp?no=14113197&t=0&all=0&TableID=0
Would be interested in any other opinions on OvaRex.
Regards
PS
Sorry for any typos grammar mistakes not my strength and more evident longer I write.
Curious if anyone who follows Allos Therapeutics (ALTH) knows why the jump today. I don't see a news release wondering if it is some analyst upgrade or anything published on one of their compounds. TIA
I was a little surprised by that I thought it would be much later on before we see Polymerase-protease combo trials. It seems to me dosing/combo of protease and PEG-Ribo is still not clearly defined so adding another component would be much further off?
Curious as to anyone's opinion since a polymerase would obviously not be approved (and if they are as ambitious as they said on the CC telaprevir wouldn't be approved either) if this signals Vertex is looking to make another deal of some sort and what form it would take (they do have quite a lot of cash on the balance sheet and seem more then willing to issue shares).
Along the same lines of Ted Johnson although not as fortunate. I wonder if the NFL starts to get some lawsuits from x-Players.
http://sports.aol.com/nfl/story/_a/report-waters-suicide-tied-to-brain/n20070119031809990004
Report: Waters' suicide tied to brain damage
AP Sports
NEW YORK (AP) -Brain damage caused on the football field ultimately led to the suicide of former NFL defensive back Andre Waters, according to a forensic pathologist who studied Waters' brain tissue.
Bennet Omalu of the University of Pittsburgh told The New York Times that Waters' brain tissue resembled that of an 85-year-old man and that there were characteristics of early stage Alzheimer's. Omalu told the newspaper he believed the damage was related to multiple concussions Waters sustained during his 12-year NFL career with the Philadelphia Eagles and Arizona Cardinals.
Waters was 44 when he committed suicide last November.
Omalu said trauma was a significant factor in Waters' brain damage, "no matter how you look at it, distort it, bend it."
The pathologist also told the newspaper the signs of depression that family members described Waters as exhibiting in his final years likely were caused by the brain trauma. Had he lived, Omalu said, the former player would have been fully incapacitated within 10 years.
The Alzheimer's Association Web site reports "there appears to be a strong link between serious head injury and future risk of Alzheimer's." The statement did not distinguish between a single catastrophic trauma and lesser repetitive injuries.
"Whatever its cause, Andre Waters' suicide is a tragic incident and our hearts go out to his family," NFL spokesman Greg Aiello said Thursday.
"The subject of concussions is complex. We are devoting substantial resources to independent medical research of current and retired players, strict enforcement of enhanced player safety rules, development and testing of better equipment, and comprehensive medical management of this injury. This work over the past decade has contributed significantly to the understanding of concussions and the advancement of player safety."
Omalu began his research at the request of Chris Nowinski, a former Harvard football player and professional wrestler whose career was ended by multiple concussions. After hearing of Waters' suicide, Nowinski called Waters' sister, Sandra Pinkney, and asked permission to do further investigation on her brother's remains.
Pinkney agreed. In mid-December, Nowinski shipped four pieces of Waters' brain from Florida to Omalu in Pennsylvania.
Nowinski chose Omalu because he had examined the brains of two former Pittsburgh Steelers players who were discovered to have brain trauma after sustaining concussions - Mike Webster, who suffered brain damage and became homeless before dying of heart failure in 2002, and Terry Long, who killed himself in 2005.
Waters' relatives said they hope further research will change the way the NFL - and all athletic organizations - treat concussions.
"I just want there to be more teaching and for them to take the proper steps as far as treating them," Kwana Pittman, Waters' niece, told the newspaper. "Don't send them back out on those fields. They boost it up in their heads that, you know, 'You tough, you tough."
Thanks!
Even links to the webcasts!
Your pretty good at this if you can figure out how to google tomorrows news today I think I know how to make some money.
Anyone have a link of who is appearing at the Merrill Lynch Global Pharmaceutical, Biotechnology and Medical Device Conference?
Biowatch since you are so good at googling maybe you can find it? I tried and didn't have much success (actually I use yahoo search more then google).
TIA
I caught some of it. What I was most interested in was the finances/future direction. Maybe someone who follows it more closely could comment more?
They will have about 80 million in cash (Canadian) / 2 years burn. Nothing decided yet about what (if anything) to do in terms of costs going forward. Said didn't have much new to comment on and will try to present the data.
Commented about the trial being well run personally I would prefer a poorly run trial with great results. At least you have a chance at a positive outcome :).
Good to see there is at least one more ITMN long here :)
Dew already caught me on one mistake but I am pretty sure I didn't write 191 is complete [in phase 1 studies]. If I gave that impress, certainly didn't mean to!! The notes are written more for myself and so something may have been abbreviated. To clarify things:
191 in Phase 1 studies as we speak, proof of concept data in 2007,
Enrollment "is" complete? That didn't didn't take long.
It is still in Phase 1. The way Tom Kassberg talked about it the Phase 1A and B trials are combined (that is my impression he didn't come out and say so). The proof of concept would be the 1B results.
Was the 175 million shelf filed recently?
The shelf was filed in late December. There is a separate 170 million convertible already on the books (conversion price of 21.63 which gives about 8 million shares combined with the 33 gives the fully diluted number)
I have a couple theories on this shelf. If I am right in my interpretation of the 170 million if holders elect to redeem early the company can pay in cash so they'd need the cash to do so. The other theories revolve around what the company may be planning with extra cash.
What we said is there are 2 interim looks (for efficacy). Very specific about not saying when/if they occurred just saying they have very specific efficacy stopping boundaries.
Is this in reference to the HCV study? I thought ITMN was testing healthy patients in Phase I? Has that changed?
2 Interim looks are for Actimmune (the INSPIRE trial).
You had me second guessing myself, I'll admit I hear what I want sometimes :). I went back and its around the 16:20 mark. I did get it wrong sorry to mispost that information! It was in talking about an all oral regimine and combining telaprevir or [I must of recalled and] another protease inhibitor with a polymerase inhibitor.