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For what its worth I too thought they'ld want to see if different ratios would show one drug needed to be higher then another. If 1000mg is the optimal polymerase and they intended to dose at least 200mg q8 for the protease then perhaps studying 500mg 200mg or 1000mg 100mg while it could provide some additional information may not be of much benefit.
The 1000mg dose on the Polymerase I believe was found to be the optimal dose (I believe 1500mg didn't seem to be statistically better) and the 200mg q8 protease was the maximum in treatment naive that was tested (and reported/presented) so perhaps that is the max they could do for now. Intermune has stated they have tested higher doses in the triple combo (PEG-Riba) study.
Dan Welch did say the 1 is to be followed by multiple other INFORM studies. I wonder if they are trying to gain preliminary information in a quick fashion. Also I would think that 1000mg / 200mg is the minimum necessary to even bother with a 2 drug combo so if they can't reach that then additional drugs are needed in combos to go further.
Welch has always struck me as a straight shooter. Was he crossed up by Roche, or did he simply misinterpret the question about the INFORM-1 design on the recent webcast?
But he also is very careful in disclosing information (as Adam Feurestein can attest to in his prejudging mean vs. median data). On roche matters he's often spoken in generalizations giving an idea but not specifics. What I do recall is him saying at the Deutche Bank Confab is to do kind of a matrix lo/lo, lo/hi, hi/lo, hi/hi which i don't see in this design. But he may have been trying to limit what was disclosed as he was not giving specific trial design.
Nice find thanks! I don't see what is being done in cohort C (vs b & d).
to be off topic a little I saw a poster about Naglazyme A Follow-Up of Enzyme Replacement Therapy in Two MPS VI Patients with Poorly Engrafted Bone Marrow
Transplantation. I thought BMT was not an option for MPS VI. A little more research and I found it is an option in some do you know how common? Am I right in thinking BMT is not an option in Morquio too. TIA!
ZYXI:
I think the insurance risk to zyxi is mitigated somewhat in that they are now being reimbursed for alternate products. The risk of having to settle past payments may still exist but I would put that at minimal. This market seems easily scared though so the threat seems enough to depress the stock.
OT:
olddog,
Sorry I am not a paying member so couldn't reply. Altus has a board if you want to discuss it there since that is the company with a non-pig PEP I had followed. I think they blew their trial but there are a number of other PEP competitors to Zentase (Solvay just had positive panel vote, Axcan, think J&J still pursuing and also couple new entrants I came across). The altus board is
http://investorshub.advfn.com/boards/board.aspx?board_id=10029
(its unmoderator so I doubt anything would be off topic :) or you could post on biotech values too it has a lot of knoweldgeable posters the moderator is quite long IDIX the link is:
http://investorshub.advfn.com/boards/board.aspx?board_id=1418
OT:
EURX?
What do you like about them? I used to follow a company with a competing drug and only know about that one (Zentase) but for my money Eurand was and continues feeding investors a lot of BS on its timeline on when it may be approved (I posted as such on the Yahoo boards I believe;maybe here on the ALTU board too it was months ago when they first announced the approvable letter). With Solvay likely to get approval after positive panel vote, I would hope the value you see in the company is elsewhere even then I don't trust management after their BS on Zentase.
HGSI:
I don't follow it closely but was surprised to see it in the 1's. This was a 100 dollar stock in the boom days. I thought they had some interesting compounds (HCV, Lupus, antibodies)/technology but haven't kept up with them. Anyone follow/own, is it the debt that has killed the stock or clinical development?
BMRN:
Some of us think it is pretty cheap even with mediocre Kuvan sales. Baring a major change to the company (deal, big trial for BH4 indication, etc.) I would not be shocked if EPS comes in near a buck next year, OK that may be optimistic but I wouldn't be shocked by it at all. Now this time next year if things go well PEG-PAL and Morquio will be nearing to start pivotal trials. It sure sounded to me like Morquio trial will be a Phase 1-2 (in patients) followed by a 2-3. I think Morquio could be better then Nagalzyme in terms of market size (assuming the drug works well). Since I lost faith in Altus developing anything meaningful PEG-PAL looks to be a real block buster. I posted on the BMRN board I think (again if it works as good as forecast) could be 100-125k annual but I think that may be on the low side and unlike Kuvan I would think patients would be very anxious to get on it and not have to worry at all about diet/medical foods/etc.
I think JJ & Aselage have done great though it is disappointing to lose Emil. I can live with the slow and stead winning in the end especially in this market.
Perhaps Adam read about this on i-hub :)
#msg-33834866
Sorry BR I have been busy a bit lately. I did catch part of the SPPI call. I don't intend to buy any soon is the short answer. Raj ducked the question about EU Zevalin with less restrictions. From (long time ago) seeing Zevalin data thought it would be a good NHL drug. I think they twisted some things. Yes recurrences are common especially in indolent but CR's are too so I take what was said with grain of salt. There are more (granted some are little) details I don't like about SPPI spin on deals (e.g. CTIC has a put option to dump Zevalin for 18 million so if it really flops Spectrum is short more cash; also they will be dishing out more cash for milestones to BIIB (& Bayer?) plus the costs till the JV is profitable). Unfortunately (fortunately for buyers) with this market I don't see the stock taking off. I hope Zevalin does well but I will wait to see it ramping before I'ld even consider. Leucovorin doing 2 million in 2 months would be great (without colorectal) sounds very impressive but the fact that Raj didn't boost much leads me to think perhaps they have distributors stocking initial inventory or something else that I wouldn't count on maintaing that much less improving on it in the near term.
This seems quite relevant since John talks a lot about personalized medicine (granted we are focusing on very rare diseases). Since our CEO has political ambitions I wonder if we could somehow benefit with any contacts he may have made. If nothing else maybe we could get a handout to reduce our burn :) though I would settle for getting rid of Canaan Partners.
Obama to broaden role of genetics in medical care
http://news.yahoo.com/s/ap/20081128/ap_on_he_me/transition_genetic_medicine_1
By RICARDO ALONSO-ZALDIVAR, Associated Press Writer Ricardo Alonso-zaldivar, Associated Press Writer – Fri Nov 28, 1:57 pm ET
WASHINGTON – For years, scientists have held out hope that the rapidly evolving field of genetics could transform medical diagnosis and treatment, moving beyond a trial-and-error approach as old as the Hippocratic Oath.
But the vision of individualized treatment based on a patient's genetic makeup and other biological markers has yet to materialize, even if better use of genetic information has led to advances in cancer care and other areas.
Now the pursuit of "personalized medicine" is expected to get a major push from the incoming administration of President-elect Barack Obama. As a senator, Obama introduced legislation to coordinate the sometimes conflicting policies of government agencies and provide more support for private research. He remains keen on the idea.
"The president-elect has indicated his support for both advancing personalized medicine and increasing (research) funding," said Rep. Patrick J. Kennedy, D-R.I., who has introduced legislation in the House that builds on Obama's.
Obama is also interested in the role that personalized medicine could play as an element of changes in the broader health care system.
"The issue of getting the right treatment to the right person goes with his whole emphasis on health reform," said Mark McClellan, a noted Republican health care expert who served President George W. Bush as Medicare director and head of the Food and Drug Administration. "If we're thinking about reforming the health care system, we should be thinking about what medicine will be like down the road when health care reform is fully implemented," McClellan said.
Although medical science is more technologically advanced than at any time in history, in some ways it is still strikingly old-fashioned. For example, most prescription drugs are effective only in about 60 percent of treated patients, leading to a trial-and-error approach to treatment that not only may be more costly, but can put some patients at risk.
Among patients, the varying responses to medications may be linked to differences in genetic makeup that affect how the body processes a drug. For example, the FDA recently warned that certain drugs for epileptic seizures may prompt a severe skin reaction in Asian patients because of a genetic trait. The practice of medicine could be streamlined if doctors had reliable ways of predicting which drugs would work on which individuals.
Government funding for research helped make possible many of the scientific gains in genetics, and Congress has passed landmark legislation outlawing discrimination against patients on the basis of genetic information.
But the mundane decisions, such as whether or not to pay for some genetic tests, have not progressed that smoothly.
For example, the FDA supported research that found certain genes can make some patients taking the blood thinner Coumadin susceptible to potentially fatal bleeding. The agency has been pushing for some time to make doctors aware of genetic tests that could help their patients.
But Medicare has not yet set a national policy on paying for the tests. Problems with Coumadin, also known as warfarin, are a major cause of emergency room visits for seniors.
"It would be very helpful if you could get (Medicare) and FDA talking to each other," said Edward Abrahams, executive director of the Personalized Medicine Coalition. "Right now they really don't communicate very well, and they have different agendas. The federal government is not coordinated around removing the barriers to personalized medicine." His group represents university research centers, industry and patients.
Obama's legislation would create an interagency group to coordinate the policies of federal agencies whose decisions have an impact on the issue. Kennedy also would direct Medicare to set a fixed policy for coverage of genetic tests and treatments.
Both bills would establish a national 'Biobank' to pool data that could serve as a resource for scientific researchers, and the legislation also calls for increases in federal funding. Obama has proposed a 100 percent tax credit for private research to develop diagnostic tests that can predict the safety and effectiveness of certain high profile drugs.
"So far, we haven't really seen any transformation of medicine," said McClellan. "There are some real questions to deal with to see if there is some policy that could speed it up.
Patient-led drug trials defy medical establishment
I found this article interesting reminded me of that movie Lorenzo's Oil? I went to the website they referenced ( http://www.patientslikeme.com/ ) seems to be still in the early stages (judging by # of diseases). It was interesting to see the website has venture capital behind it. I wonder if they run into some legal/regulatory troubles at some point. No truth to the rumor that Raj is going to run his next trial through this network :)
http://news.yahoo.com/s/ap/20081126/ap_on_he_me/med_patients_take_charge_3
By MARCUS WOHLSEN, Associated Press Writer Marcus Wohlsen, Associated Press Writer – Wed Nov 26, 5:02 pm ET
Alan Felzer, right, and his daughter Karen Felzer, holding her son 11-month-old AP – Alan Felzer, right, and his daughter Karen Felzer, holding her son 11-month-old Emmet Harrington, look …
CLAREMONT, Calif. – Until last year, Alan Felzer was an energetic engineering professor who took the stairs to his classes two steps at a time. Now the 64-year-old grandfather sits strapped to a wheelchair, able to move little but his left hand, his voice a near-whisper.
Felzer suffers from ALS, also known as Lou Gehrig's disease. The fatal neurological disorder steals the body's ability to move, speak and ultimately to breathe. But rather than succumb to despair along with his illness, Felzer turned to the Web to become his own medical researcher — and his own guinea pig.
Dozens of ALS patients are testing treatments on their own without waiting on the slow pace of medical research. They are part of an emerging group of patients willing to share intimate health details on the Web in hopes of making their own medical discoveries.
Some doctors caution that such patient-led research lacks rigor and may lead to unreliable results, false hopes and harm to patients.
"The Internet is a wonderful tool, but you know, it's buyer beware," said Dr. Edward Langston, immediate past chairman of the American Medical Association's board.
In Felzer's case, the experiment's results illustrate the obstacles that stand between patients and self-discovered breakthroughs. The drug he tried did no good. But he and his family felt they had little time and little to lose in trying.
"ALS is such a short illness," said Felzer's wife, Laura. She helps her husband communicate using sign language with his one good hand when his slow, halting words become difficult to understand. "You want to do what you can as fast as you can."
The U.S. Food and Drug Administration has approved only one drug to treat ALS symptoms. It only works for some patients, and its effects are limited. As a result, Internet forums for ALS patients brim with links to the latest research offering any hint of promise. After Alan Felzer was diagnosed last year, his 33-year-old daughter, Karen, dived into the forums and found new hope.
In a recent small study, Italian scientists reported that every ALS patient given the drug lithium, commonly used to treat bipolar disorder, saw the disease's progress slow substantially.
Many ALS patients began trying lithium on their own. They persuaded their doctors to prescribe it "off-label" — a use not approved by federal drug regulators. Off-label prescribing is a common practice, researchers say, when patients are facing a terminal illness.
Despite the risks, Langston of the AMA pointed out that doctors often stumble upon treatments, and patients could possibly do the same. "If patients are willing to share their experiences, that may in fact occur," he said.
Felzer began taking lithium in January, and his scientifically minded family reached out to other ALS patients. "All those people are taking it anyway," said Alan Felzer, whose smile remains bright and his gaze sharp even as the rest of his body fails him. "So it only made sense to keep track of what was happening."
The task of leading the ALS-lithium project fell to Felzer's daughter, Karen, a U.S. Geological Survey earthquake researcher. Her partner in the effort was Humberto Macedo, a 42-year-old computer systems analyst, father of six and ALS patient in Brasilia, Brazil.
The study grew naturally out of the strong reliance of ALS patients on one another for information, Macedo said.
Working online, Karen Felzer and Macedo recruited nearly 200 patients worldwide to take a specific lithium dosage and answer standard surveys to gauge their symptoms. They began running their study through a Web site called PatientsLikeMe.com, using it to attract volunteers and track their progress.
On the site, patients share detailed information about their symptoms and the drugs they are taking. The site focuses on conditions that have stubbornly resisted medical science, such as ALS, Parkinson's and multiple sclerosis.
The site's founders hope professional and amateur researchers alike will dip into the resulting pool of data and emerge with insights that lead to better treatments.
"My ultimate frustration that drove this site into existence was an overall feeling that there was a lack of transparency and speed or urgency" by the medical system, said Jamie Heywood, who co-founded PatientsLikeMe months before his own brother died of ALS.
Heywood too hoped that lithium was the breakthrough he and others had been seeking.
But after six months, none of the 87 people who stuck with the study showed any letup in the disease's progress, said Karen Felzer. She now doubts the Italian study's results.
"It's obvious to everyone it's not the miracle drug we thought at first," she said. She also thinks other tests of lithium for ALS should be halted to spare patients the drug's possible side effects, such as tremors, weakness and difficulty breathing. Her father stopped taking the drug, though Macedo is continuing.
However, other reseachers say professional lithium studies should go forward. Dr. Merit Cudkowicz, a Harvard Medical School professor, is set to begin one in December with 84 patients. Her study will stick to the so-called gold standard of research, in which each patient will be randomly chosen to take the drug in question or a placebo. Neither patients or researchers will know who got the drug to avoid introducing bias.
Because the patient-led lithium study lacked those tight controls, it is unreliable as a measure of safety and effectiveness, Cudkowicz said. With an incurable disease, she said, "You don't want to be throwing something away that works because of a bad study."
I believe Dew and some of the other posters who commented on the commercial viability of the drug. I believe that was a major reason but I'll let them speak for themselves.
I was just of the belief that Zevalin could be beneficial to a significant number of NHL patients. As a few years ago anyway NHL was on in the increase. I believe Rituxan hit a billion in sales long before it was used in other indications.
I'ld say its good the Doc isn't taking CTIC at their word.
Its been a long time since I looked at NHL therapies and I doubt that is something I would have remember but here is a link (I posted part of the article in case you don't have a subscription):
http://www.medscape.com/viewarticle/440526
Experience With Zevalin (Ibritumomab Tiuxetan) MoAB Therapy
Zevalin, the only radioimmunotherapy approved for use by the United States Food and Drug Administration (FDA), is a murine radiolabeled anti-CD20 MoAB that is particularly appropriate for stage III and IV disease as a systemic treatment. Zevalin's therapeutic radioisotope is Y-90 with a half-life of 64 hours, which emits a beta particle with a rather large 5-mm range and no imageable gamma rays. Treatment regimens follow a 250-mg/m2 predosing with cold antibody followed by a diagnostic dose of 5 mCi of In-111-labeled antibody. Therapy regimen doses are 0.3 mCi/kg for patients with a platelet count between 100,000 and 149,000 per microliter (mcL) and 0.4 mCi/kg for patients with platelets > 150,000/ mcL. In the phase 1/2 trial of patients with low- or intermediate-grade and mantle cell NHL, the overall response rate using Zevalin was 67% in 51 patients.[5] In a phase 3 randomized trial, patients with relapsed or refractory low-grade, follicular, or transformed CD20(+) B-cell NHL and < 25% bone marrow involvement received either 375 mg/m2 weekly x 4 of Rituximab or 0.3/0.4 mCi/kg of Zevalin. The overall response rate in the Zevalin group was 73%, whereas the response in the cold antibody group was 47%. The incident of human to mouse antibody reaction (HAMA) was 1.4%. Overall, for 179 patients the mean red marrow dose was 53 cGy, spleen 667 cGy, liver 341 cGy, lungs 255 cGy, and kidney 38 cGy. The conclusions of the initial trials were that toxicities with Zevalin are primarily hematologic, transient, and reversible. There was a high response rate in relapsed low-grade NHL, with a short treatment course, which did not require hospitalization.
ALTU:
The (major) problem I see is with their stubbornness to reduce cash burn they've quickly removed their odds of getting to approval with the drug on their own. Don't forget the safety study will only conclude late q1/early q2 of next year so they won't file till mid-late '09 (I doubt mid with their history but I think that was their claim). If I heard right on the quarterly call they are basically looking to sell some/all of Trizytek anyway.
No posts today! Well here is an interesting article if you believe Boras is a puppeteer. It makes some sense but I don't think things will play out this way.
http://msn.foxsports.com/mlb/story/8854244/Boras-holds-the-cards-in-this-free-agent-market
Boras holds the cards in this free-agent market
by Ken Rosenthal
Ken Rosenthal has been the senior baseball writer for FOXSports.com since Aug. 2005. He appears weekly on the FSN Baseball Report and MLB on FOX.
Here's my theory: Scott Boras wants Mark Teixeira in Boston.
Teixeira, a native of Maryland, would be happy to go back East, happy to accept whatever ridiculous deal the Red Sox gave him.
That way, both the Angels and Yankees would remain options for Manny Ramirez, Boras' other major free-agent slugger.
The Angels could sign free-agent left-hander CC Sabathia with the money they had slotted for Teixeira. They also could add Ramirez to fill Garret Anderson's slot and protect them against the possible departure of Vladimir Guerrero as a free agent after next season.
Sabathia, who is represented by the Legacy Sports Group, might cost $150 million over six years, Ramirez $90 million over three. But the additions of both would give the Angels utter control of the Los Angeles market, and make them the favorites to win the World Series.
Teixeria to Boston, Sabathia and Ramirez to the Angels. The Yankees would freak out if all that transpired, and yet another Boras client — free-agent right-hander Derek Lowe — would be the immediate beneficiary.
Oh, and don't forget Boras' free-agent catcher, Jason Varitek, who could return to Boston to join Teixeira, his friend and fellow Georgia Tech alum, with Boras posing between them at the news conference.
See how all this works?
If you think Boras lacks the vision to formulate such a grandiose plan, then you haven't been paying attention for oh, about the past 20 years.
Boras not only reads the market better than most agents, he reads it better than most clubs.
And when he holds this many prominent pieces, he is fully capable of manipulating the process to his advantage.
Obviously, things could go awry, particularly with Ramirez, whose market remains unclear.
The Yankees simply might decide to sign Sabathia at any cost, particularly when they figure out that the Angels' interest is sincere and that the Giants might be serious players for CC, too.
If the Yankees indeed land Sabathia, they could add Lowe or A.J. Burnett as their second major free-agent piece and address their offense through lesser signings or trades.
The Angels seem hotter for Ramirez, but owner Arte Moreno might have just wanted to tweak the Dodgers when he said, "At the end of the day, you want people who can hit, and he may be one of top right-handed hitters ever."
Ramirez sure doesn't seem like Mike Scioscia's type of player; Raul Ibanez or Adam Dunn might be a better fit. But if Moreno wants Ramirez, Scioscia and the rest of the Angels' baseball people will have no choice but to fall in line.
One thing is certain: Ramirez would be easier to counsel (read: babysit) if he remained with a team close to Boras' headquarters in Newport Beach, Ca. Teixeira, on the other hand, requires no adult supervision, which is yet another reason why Boras might prefer him in Boston.
The Red Sox clearly want Teixeira, but the Angels will wait on him for only so long. At some point, the Angels might simply determine that their chances are better with Sabathia, whose preference is to be on the West Coast.
You'll notice I haven't mentioned the Dodgers, the only team that is known to have made a contract proposal offer to Ramirez. The Dodgers also should be a favorite for Sabathia, offering him — like the Giants — the chance not only to play on the West Coast, but also the National League.
Well, in an interview with the Los Angeles Times on Tuesday, Dodgers president Jamie McCourt wondered aloud about whether it would be "a little weird" to sign big-money free agents while the nation's economy is reeling.
McCourt also said that the free-agent game would be more tolerable if the players' contracts were not guaranteed — an antiquated point of view that is certain to draw ridicule, no matter how much clubs wish it were true.
McCourt's husband, owner Frank McCourt, indicated that the club will not raise its offer to Ramirez. The Dodgers have given no indication they will be a serious player for Sabathia. No wonder agents are again questioning the Dodgers' willingness to spend big.
The Dodgers still could sign a lesser free-agent bat, trade for Yankees second baseman Robinson Cano and piece together their pitching. But now is not the time for a high-revenue team to fret over baseball economics, not when Sabathia is there for the taking and Boras is ready to play ball.
The game is about to start.
The dominos are about to fall.
OT:
I haven't seen him post in a while I guess he is busy with his day job....Follow the link to see who :) (just kidding I think anyway)
http://www.newswire.ca/en/releases/archive/November2008/28/c5150.html
ALTU:
Interesting, thanks for posting. If only they could have produced descent data they could have been in great position. I was surprised they did not report any significant findings to account for the data disparity.
I sold a fair amount and will probably do some tax loss selling on the rest this year. Gemayel speaks at the Piper conference on the 3rd for what its worth. They really should have cut their burn (really since DNA bailed).
Feel free to add them to the list #msg-33753805
I don't know a who lot about Fusilev or colerectal. I thought they were doing an Oral version of the drug too? I thought if they could generate 100-150 million peak sales its pretty good for a company SPPI's size at least pay the bills and maybe support Raj's inlicensing habit :).
The failed Phase 2 study combined with both AEZS and SPPI unable to partner their BPH drugs lead me to think that either the data is not as good as we are lead to believe and/or there are more obstacles/competitors. I would personally wait to see what happens with AEZS compound and not spend anything on it until then. Maybe if there was something to be gained from doing a small Phase 2 I wouldn't object. I agree about Raj doing things on the cheap which really bugs me that they claim to save money by doing things in-house as opposed to contracting well he can't have it both ways when the trial is botched its not saving shareholds money!
Eoquin is the type of drug I like. Limited competition, small directed sales force, descent price and a lot of repeat business (unfortunately for patients). The down side is I think they will need a few more trials though to get approval in 2nd line/maintenance (and other tumor types?). It seemed like in the US it may also be a tougher sell to use the drug but it sounds a whole lot more convenient (same day verse subsequent visit) and safer then using bcg and perhaps longer time to retreatment. I don't know much about allergan but I'ld want someone who'll spend the bucks. I think it is a 2 year followup so by the time they get it fully enrolled and followup SPPI could be a completely different company or part of someone else :). I doubt I will be an SPPI shareholder again under Raj anyway (he is in his 60's right :)).
There are a number of Biotechs I follow well under cash. On SI their is a board an rkrw periodically lists a bunch. Unfortunately everyone thinks their Biotech is unduly undervalued time will tell who was right. I think at least 3/4 of the managements are self serving. If you are well under cash you probably should be sold/liquidated or taken private IMO.
VIFL:
I am not paying so excuse the reply to a post on another stock. I looked at the stock a while back and found it attractive as it has a lot of assets to go with EPS and interesting business but I am holding out as its been pointed out on their board that a big holder is slowly unloading and they have a ton of shares and the stock is extremely thin for such a big holder to keep selling.
Board -
http://investorshub.advfn.com/boards/board.aspx?board_id=1735
#msg-32379236 Last posting on sale by MDS Canada
br,
I actually hope Raj proves me wrong as I would like to see Zevalin do well, I am just skeptical. I may even reconsider SPPI as an investment as I like Eoquin. I think Raj was wrong/deceived about Ozarelix. I suspected after a while Phase 3 wouldn't start till they had further data but they should not have spent anything on it until AEZS burned theirs to see what Cetrorelix did (since presumably Ozarelix is more potent). I also think SPPI-1620? had some potential but was early.
Now as far as the cash and cash burn. I haven't kept up with it but going by PR and general knowledge they will be down to 60 million after paying CTIC in January. They claimed to be at a 5 million/quarter burn. My thesis is they will be at least 10 million going forward. Even with Allergan paying 65% to fund a big Phase 3 will costs them more then the pennies they spend now on developing their pipeline. Plus you can add more into SG&A for ramping up and promoting 2 oncology compounds. I think if they are at 10 million burn a quarter it'ld be lucky (unless Zevalin and/or fusilev really ramp). That would be 6 quarters of cash and I think they would raise cash well before that (I think Raj would want 4 quarters of cash). OK say they get a little for the Asia deal if they only got 40 million from Allergan I would say 10 million up-front max which is 1 quarter of burn by my estimation. If you want to argue Raj will keep the burn at 5 million then I want to argue Zevalin and Fusilev never generate meaningful revenue :).
UTHR:
I went back over the presentation a couple times and actually think FREEDOM-M has a pretty good shot (> 50%) to get a P value < .05. I still need to review some of the literature I can find on dose response with the sub-q and IV treprostinil. What we don't know yet is how high an average dose patients got and I am estimating going by memory about the dose response. Its not unlike UTHR to mess up trials before getting a drug approved and actually have it work out to their advantage. The sub-q was delayed because of that (they enrolled too mild of patients and endup with a broad label class II-IV though I doubt there are many if any class II's actually using the sub-q or IV).
So if things stick to UTHR history. They could actually turn this into a positive (for long term investors) if they end up generating a couple years of sales with the inhaled and get it going before the Oral comes plus they have Cials too. This would keep them at the 40% growth rate for some time.
Now my (and others) pet peeve with the company is their options rewards are very excessive (but this is nothing new). What is annoying is they reprice them and try to bury it on a low news day.
http://www.sec.gov/Archives/edgar/data/1082554/000110465908073618/a08-29017_28k.htm
and
http://www.sec.gov/Archives/edgar/data/1082554/000104746908012620/a2189373zscto-i.htm
Anyway at 50 the stock got incredibly cheap (though I find it difficult to value stocks in this market). It was getting a PE in the 20 range (much lower if you take out options/rewards) with a 40% growth rate and 2 drugs with PDUFA's 1H '09 both of which have a good chance of approval.
If anyone is interested perhaps in the next couple months I will review some data and can come up with a better guestimate on the odds for FREEDOM-M (I also have a table of periodic enrollment stats so could theories on how many got lower dose tablets though the company gave some rough data in the call too).
I also thought Zevalin was a good therapy for NHL patients. I was very disappointed to see Biogen give it away to CTIC as I tend to agree with those who are critical of CTIC. To me it ment that Biogen just saw it wasn't going to ever be significant.
I don't really understand what Raj is thinking though. Its not like Spectrum will have a big sales force and if he wanted another drug to sell why not do a deal where they get a much smaller cut but don't have to put up a lot of cash (I would guess CTIC wanted cash now). With Spectrum's share price below cash and them burning cash I don't think it was the best use of funds in the current environment. If the drug starts producing significant profits for the JV and gets SPPI to near breakeven then I'll think he is a genius and new something that both Biogen and CTIC didn't know. But he also will have to get this to happen in the next year or so to save diluting current shareholders even more.
Disclosure: x-BIIB shareholder (long time ago) and burned by Raj before :).
OrbiMed has been buying. I used to think Isaly was pretty good (he had some of my stocks :)) but after I saw him on NBR pumping NPS (quite some time ago) I lost some respect for him. I don't know how his fund has done and I don't follow aryx just an FYI post.
http://www.sec.gov/Archives/edgar/data/1055949/000094787108000620/ss51720_sc13d.htm
Pirates gamble on raw talents with million-dollar arms
I had heard about the two pitchers throwing for scouts but I didn't realize they were part of a reality show.
http://sports.espn.go.com/mlb/news/story?id=3724303
PITTSBURGH -- The Pittsburgh Pirates hope Rinku Singh and Dinesh Patel really do have million-dollar arms.
SportsNation
What do you think of the Pirates signing reality show winners Rinku Singh and Dinesh Patel to minor league contracts?
Legitimate risk
Publicity stunt
The two 20-year-old pitchers, neither of whom had picked up a baseball until earlier this year, signed free-agent contracts Monday with the Pirates. They are believed to be the first athletes from India to sign professional baseball contracts outside their country.
Singh and Patel came to the United States six months ago after being the top finishers in an Indian reality TV show called the "Million Dollar Arm" that drew about 30,000 contestants. The show sought to find athletes who could throw strikes at 85 miles per hour or faster.
While neither pitcher threw hard enough to earn the $1 million prize, Singh made $100,000 from the contest and Patel made $2,500, plus his trip to the United States.
The contest was sponsored by a California sports management company that believed it could locate major league-worthy arms in a country of more than 1 billion. After working extensively with Southern California pitching coach Tom House since May, the pitchers staged a tryout in Tempe, Ariz., on Nov. 6 that was attended by 30 major league scouts.
"The Pirates are committed to creatively adding talent to our organization," Pirates general manager Neal Huntington said Monday. "By adding these two young men, the Pirates are pleased to not only add two prospects to our system but also hope to open a pathway to an untapped market. We are intrigued by Patel's arm strength and Singh's frame and potential."
Neither pitcher has taken the mound in a game situation, no doubt a first for a Pirates prospect. They have pitched in scrimmages against junior college competition.
Both threw the javelin in India, a country best known for producing cricket players, and neither the right-hander Patel nor the left-hander Singh had left his small village before coming to the United States. Singh was born in Bhadoni, Uttar Pradesh, and is the youngest of nine children. Patel is from Varanasi, Uttar Pradesh, and has four brothers and sisters.
The 5-foot-11, 185-pound Patel hit 90 mph on the radar gun during his tryout, and the 6-2, 195-pound Singh topped out at 84 mph. Each has thrown harder during workout sessions that weren't attended by scouts.
"Think of them as two Dominican kids," House told the scouts. "They're very raw. But I think this has a huge upside."
When they first came to the United States and began playing catch, the pitchers were mystified by the concept of gloves and had to be taught not to try to catch the ball with their bare hands.
Despite being more than raw, the pitchers were signed by well-known agent Jeff Borris, who was attracted by their potential after watching them work out at Southern Cal.
Borris estimates they will need three to four years of minor league experience before becoming major league ready.
Patel and Singh are learning English, most of which they have picked up from watching ESPN's Baseball Tonight and by taking online classes.
"These young men have improved a tremendous amount in their six-month exposure to baseball and we look forward to helping them continue to fulfill their promise," Huntington said.
The signings represent a shift in policy for the Pirates, who have mostly ignored nontraditional markets such as Asia for players.
Copyright 2008 by The Associated Press
They didn't have Q&A at the end of each section and I skimmed through the ending Q&A so could have missed it but didn't hear it questioned.
In one of the prior presentations (Deutsche Bank?) they were asked about other compounds including this. Response is basically not seen as direct competitors since different MOA. Though if one is far superior obviously it becomes the prefered initial treatment and I am not sure how willing payers would be to support two high priced therapies.
I don't know much about the Centocor compound. The death rate of IPF/PF compounds is very high not too many that I am aware of have made it to Phase 3. The most advanced at this stage are Bosentan (Actellion) and Ambrisentan (Gilead) both in Phase 3 (Bosentan is fully enrolled, Amberisentan recently initiated). Both are ERA's approved for PAH.
I have not updated the competitive space in some time here is the old msg for those interested: #msg-26702328
SGP:
FYI the portion of the talk on their PI's starts at about 2:14. They don't go into too much detail. They review development of Boceprevir. Currently treatment experienced trial fully enrolled, naive screened 1200 patients. Will be studying with Pegasys. Rash similar to placebo.
SGH-900518 just said QD dosing, favorable resistance profile, phase 2, referenced slides but I only had the audio.
EDIT:
Link for slide presentation http://media.corporate-ir.net/media_files/irol/89/89839/SP_RandD_WEB_Archive_11_24_08.pdf
I don't believe they have PR'd it in the past but they have talked about it in interviews and the like (after the fact).
The past year or two they have cut back on PR's in general though they do have a lot more news content on their various websites.
This is what Dan Welch has said other companies have reported and so we can expect to see reported data using similar metrics in the triple combo study when they report it this quarter. Preliminary safety data may also be reported (depending on if more cohorts are being done or not). The fact that he is giving these metrics leads me to think that the current completed cohorts are at least as good.
ITMN/INFORM Study:
On a closer rehear Dan Welch indicated there are 4 planned cohorts 56 patients total.
Given the PR on the 10th said patients were dosed (I believe in one of the presentations the 7th was said as the first day) we should now have some who have completed cohort 1.
If the second cohort is dosing lo/high for the Protease and Polymerase is there any reason why they wouldn't do 2 cohorts simultaneously (i.e. lo/high Protease-Polymerase and lo-high for Polymerase and Protease).
I got the sense INFORM-1 won't be adding cohorts and will be more to get some data for the follow-on INFORM studies which there may be several of likely international studies (given that the two agents should be in Phase 2 by that time).
SGP:
Did anyone listen?
This is from todays PR
http://biz.yahoo.com/prnews/081124/ny48186.html?.v=1
Boceprevir, an oral protease inhibitor for treating hepatitis C with peginterferon and ribavirin combination therapy (in Phase III); 24-week sustained viral response (SVR) data were presented showing that 75 percent of patients achieved SVR; in addition, a next-generation protease inhibitor in Phase II for treating hepatitis C was unveiled, with the potential to offer once-daily dosing and having 10 times the in vitro potency of current protease inhibitors in late-phase development;
Late-Stage Products Looking for a Home
Criteria for inclusion:
a) Program is in phase-3 or beyond and is non-partnered.
b) Company does not have enough cash to bring the product to market.
Edits: LJPC
Stock Product Stage Comments
----- --------------- --------------------- -----------------------------
AUXL Xiaflex Phase 3 Dupuytren's Contracture (and PH2 for peyronie's and frozen shoulder syndrome)
BPAX Libigel Phase 3 ~2010 Submit Adequate Safety?
CHTP Droxidopa Pivotal Phase III Orthostatic hypotension. Results expected 02/09.
DYAX DX-88 for HAE BLA filed 9/24/08 (CABG indication partnered w CBST)
EPIX Vasovist PDUFA 12/30/08 FDA initially rejected saying 2 more studies needed. Looking to sell outright on approval.
GNVC TNFerade Pivotal Phase III Pancreatic cancer
JAV Dyloject Phase III Approved in UK. Post operative Pain (injectable)
JAV Ketamine Phase III Acute moderate-to-severe pain (intranasal)
LJPC Riquent Phase 3 lupus (reduce renal flare)
NTII Viprinex Phase 3 Interim analysis January '09. Stroke Drug, high chance of failure.
OXGN Zybrestat Phase 3 Anaplastic thyroid cancer
Pharming Rhucin Pre-BLA Rejected in EU
RPRX Proellex Phase 3 Uterine fibroids
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TARG:
Down 83% on the news about a million shares traded (which is a lot for this stock). I didn't listen to the call and don't follow them too close but I was surprised that the panel voted against. I would think the need for new antibiotics would more then out-way the fact that one of the studies is old. Any think there is a chance the FDA approves with a thinner label and or post approval study requirement?
FDA Advisory Committee Provides Opinion on Oritavancin for the Treatment of Complicated Skin and Skin Structure Infections
Haven't followed Oritavancin closely but I am surprised by the need for new antibiotics that the vote would come out negative.
http://biz.yahoo.com/bw/081119/20081119006496.html?.v=1
Wednesday November 19, 9:17 pm ET
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Targanta Therapeutics Corporation (Nasdaq: TARG - News) today announced that oritavancin, the Company’s investigational antibiotic therapy for the treatment of complicated skin and skin structure infections (cSSSI) caused by gram-positive pathogens, including methicillin resistant Staphylococcus aureus (MRSA), received a mixed review from the United States Food and Drug Administration’s (FDA) Anti-Infective Drugs Advisory Committee.
During today's meeting, the Advisory Committee voted 11-6 (with one abstaining) that clinical study ARRI independently provides evidence of the effectiveness of oritavancin for cSSSI. The Advisory Committee voted 8 to 10 (against) that clinical study ARRD independently provides evidence of the effectiveness of oritavancin for cSSSI. Finally, the Advisory Committee voted by a narrow margin of 8-10 (against) that the data presented demonstrate the safety and effectiveness of oritavancin for the treatment of cSSSI.
Mark Leuchtenberger, President and CEO of Targanta, commented, "We obviously are disappointed with the recommendation of the Advisory Committee, but we firmly believe that oritavancin’s safety profile to-date and its unique pharmacokinetic profile confirm its potential to change treatment paradigms in cSSSI and other serious bacterial infections. We will continue our discussions with the FDA as it completes its review of oritavancin.”
While the Advisory Committee's recommendation is not binding, the Advisory Committee’s guidance will be considered by the FDA in its review of the New Drug Application (NDA) that Targanta submitted for oritavancin in February 2008. The target PDUFA date for oritavancin is December 8, 2008.
****************CONFERENCE CALL & WEBCAST INFORMATION******************
Targanta will host a conference call and live audio webcast to discuss the recommendation of the Advisory Committee
WHEN: November 20, 2008 at 8:00 a.m. ET
LIVE DOMESTIC & CANADA CALL-IN: 877-407-9210
LIVE INTERNATIONAL CALL-IN: 201-689-8049
24-HOUR REPLAY DOMESTIC & CANADA: 877-660-6853
24-HOUR REPLAY INTERNATIONAL: 201-612-7415
REPLAY PASSCODES (BOTH REQUIRED FOR PLAYBACK):
ACCOUNT #: 266, CONFERENCE ID #: 303975
The call will also be webcast live, listen only, via the internet at: www.targanta.com.
Replay will be available on Targanta’s website for 30 days.
Thanks for clarifying that!
Another Director buying. This is a nice chunck of shares!
http://www.sec.gov/Archives/edgar/data/900393/000134652908000002/xslF345X03/edgar.xml
Your welcome, you've saved me from listening to quite a few calls!
Other then the starting dose and what if (which was obvious) nothing notable.
Still waiting for you to say if Novartis had anything noteworthy (pertinent to MNTA) :)
I thought too what little I've read their technology seems interesting and Genzyme backing certainly gives credability. But I would think they will have to do studies in each specific indication especially since their are different end-points. My understanding is they are initially targeting a subset of MD. I also think Genzyme is in no hurry to advance a competing therapy so I wouldn't expect too rapid of development in Pompe especially.
VRTX:
I just heard the Vertex LCM presentation today and caught something I missed before if it was disclosed. They said the non-responder results (which I admit look very impressive) are patients who were in the control arm of prior studies. I am not a science guy and wanted to through this out to anyone who has more of a science background who cares to respond. Could this subpopulation have done considerably better then general non-responder population because:
1-Their initial viral load was lower because they had received interferon for some time (granted they were off of it for a while).
2-This group may have been more motivated and thus more compliant and less likely to drop out (they are volunteering for a second round of Interferon so soon)
3-Since they were in the prior study they may have likely been receiving better care in general then the general population and thus in better health (then other HCV non-responders not the general population as a whole)
4-Open label.
I hadn't seen news of FoldRx partnering maybe I missed that or do you mean PTC?