Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
fyi, prior to this financing there were 131mm shares outstanding on a fully diluted-basis.
Harvey always said he was gonna build a stand-alone oncology franchise...this is a pretty good down payment,lol.
Gee, Rachel McMinn's model projected 17% dilution...seems down right prescient.
yes, but, remember man who waits for roast duck to fly into mouth must wait very, very long time.
Well, your concern that the world is going to end and take ariad with it has been going on for at least 5 months:
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=65876917
Have you considered putting your money under your mattress and calling it a day? I guarantee you'll sleep better. :)
"annoying....total a-hole....f'ing BS" but how do you really feel? ;)
I guess we now know why there was such a lackluster market response to what was very good ASH data. The typical Berger, "we're not raising money" BS is irritating but I can't fault the overall strategy. I'll take the dilution hit in order to keep the upside on the pipeline.
bw, that's my take as well. plus any tax loss selling is only going to be exascberated by the low float.
It is a 130 patient trial which just started enrolling in October. All three trial centers are still recruiting. I doubt any data will be available until after ASCO.
Catalyst Pharmaceutical Partners, Inc. Announces Commencement of Phase I(a) Safety Study for CPP-115
Dec. 13, 2011 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX) today announced that it has commenced its first in man Phase I(a) safety study for its investigational drug, CPP-115, a novel GABA aminotransferase inhibitor.
"We are extremely pleased to now have two drugs, CPP-115 and CPP-109, in the clinic," said Patrick J. McEnany, Catalyst's President and Chief Executive Officer. "Our preclinical experience to date with CPP-115 has demonstrated its potential for certain addiction and epilepsy indications. We hope to offer providers and their patients safe and effective therapies for addiction, as well as safer, more effective medications for infantile spasms and other central nervous system (CNS) diseases than those currently available. We expect to report the results of this study at the end of the first quarter or the beginning of the second quarter of 2012."
The Phase I(a) study is a randomized, double-blind, single ascending dose study in six cohorts of eight normal healthy volunteers, totaling 48 healthy subjects. The study is designed to evaluate the basic human safety characteristics of CPP-115, including CNS side effects, and respiratory and cardiovascular safety.
About CPP-115
CPP-115 is a novel GABA aminotransferase inhibitor and an analogue of vigabatrin that is more potent than vigabatrin (CPP-109) and has reduced side effects (e.g., visual field defects, or VFDs and sedation) from those associated with vigabatrin in preclinical studies. Catalyst is planning to develop CPP-115 for several indications, including epilepsy (initially infantile spasms), drug addiction and for other selected CNS disease indications. CPP-115 has been granted orphan-drug designation by the FDA for the treatment of infantile spasms.
CPP-115 is the lead compound being developed by Catalyst under its license agreement with Northwestern University. Dr. Richard B. Silverman, the John Evans Professor of Chemistry at Northwestern University, led the team of scientists that invented CPP-115. Dr. Silverman holds more than 40 patents and is the inventor of Pfizer's drug, pregabalin (Lyrica®).
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc. is a development-stage biopharmaceutical company focused on the development and commercialization of prescription drugs targeting diseases of the central nervous system with a focus on the treatment of addiction and epilepsy. Catalyst has two products in development, CPP-109 and CPP-115, and is currently evaluating its lead product and first-in-class GABA aminotransferase inhibitor candidate, CPP-109 (vigabatrin), for the treatment of cocaine addiction. CPP-109 has been granted "Fast Track" status by the U.S. Food & Drug Administration (FDA) for the treatment of cocaine addiction. Catalyst also expects to evaluate CPP-109 for the treatment of other addictions. Catalyst believes that it controls all current intellectual property for drugs that have a mechanism of action related to the inhibition of GABA aminotransferase. For more information about Catalyst, go to www.catalystpharma.com.
fyi, CPRX just announced a randomized, double-blind , phase 1 trial for their second drug candidate, CPP-115. So far, they are progressing right on schedule. Given the safety profile of Sabril, a positive outcome for CPP-115 should be a significant catalyst for Catalyst.
Catalyst Pharmaceutical Partners, Inc. Announces Commencement of Phase I(a) Safety Study for CPP-115
Dec. 13, 2011 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX) today announced that it has commenced its first in man Phase I(a) safety study for its investigational drug, CPP-115, a novel GABA aminotransferase inhibitor.
"We are extremely pleased to now have two drugs, CPP-115 and CPP-109, in the clinic," said Patrick J. McEnany, Catalyst's President and Chief Executive Officer. "Our preclinical experience to date with CPP-115 has demonstrated its potential for certain addiction and epilepsy indications. We hope to offer providers and their patients safe and effective therapies for addiction, as well as safer, more effective medications for infantile spasms and other central nervous system (CNS) diseases than those currently available. We expect to report the results of this study at the end of the first quarter or the beginning of the second quarter of 2012."
The Phase I(a) study is a randomized, double-blind, single ascending dose study in six cohorts of eight normal healthy volunteers, totaling 48 healthy subjects. The study is designed to evaluate the basic human safety characteristics of CPP-115, including CNS side effects, and respiratory and cardiovascular safety.
About CPP-115
CPP-115 is a novel GABA aminotransferase inhibitor and an analogue of vigabatrin that is more potent than vigabatrin (CPP-109) and has reduced side effects (e.g., visual field defects, or VFDs and sedation) from those associated with vigabatrin in preclinical studies. Catalyst is planning to develop CPP-115 for several indications, including epilepsy (initially infantile spasms), drug addiction and for other selected CNS disease indications. CPP-115 has been granted orphan-drug designation by the FDA for the treatment of infantile spasms.
CPP-115 is the lead compound being developed by Catalyst under its license agreement with Northwestern University. Dr. Richard B. Silverman, the John Evans Professor of Chemistry at Northwestern University, led the team of scientists that invented CPP-115. Dr. Silverman holds more than 40 patents and is the inventor of Pfizer's drug, pregabalin (Lyrica®).
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc. is a development-stage biopharmaceutical company focused on the development and commercialization of prescription drugs targeting diseases of the central nervous system with a focus on the treatment of addiction and epilepsy. Catalyst has two products in development, CPP-109 and CPP-115, and is currently evaluating its lead product and first-in-class GABA aminotransferase inhibitor candidate, CPP-109 (vigabatrin), for the treatment of cocaine addiction. CPP-109 has been granted "Fast Track" status by the U.S. Food & Drug Administration (FDA) for the treatment of cocaine addiction. Catalyst also expects to evaluate CPP-109 for the treatment of other addictions. Catalyst believes that it controls all current intellectual property for drugs that have a mechanism of action related to the inhibition of GABA aminotransferase. For more information about Catalyst, go to www.catalystpharma.com.
I was glad to see this issue addressed in the Ariad QA today. The consensus being that it would be hard to design a randomized P3 trial when there is no comparator arm available for T315I pts and only a 25% MCyR achieved in 3/4 line for sprycel/tasigna.
Peter, one thing that surprised me was Talpaz' comment that the response rate achieved in T315I pts was, in part, due to the shorter duration in this patient group versus non-T315I CP patients (I believe he made reference to 3 years vs 7). The implication being the earlier a patient gets on pona better. He's certainly not in the hold pona "in reserve" camp.
yup, that's my thinking too. Other than potential partner news or a financing, this is one of those times when you just have to wait for the trial data. 6 months down the road, there will be a lot of activity around the Rida panel, AP113 first look, and of course pona. btw, do you know if the market is expecting pona news at ASCO?
Ariad hardly ever "makes news" at analyst conferences. Fortunately for investors, these days Berger is letting the data speak for itself.
For those interested, a pdf of the slides from today's presentation are available for download:
http://phx.corporate-ir.net/External.File?item=UGFyZW50SUQ9MTE4MjA5fENoaWxkSUQ9LTF8VHlwZT0z&t=1
It's hard for me to come up with a $400mm peak sales scenario that's realistic. If sprycel/tasigna continue to take 1st line market share, pona will end up capturing an even greater share of the 2nd line market which is approaching $1 billion, today. Even if gleevec (or its generic) is able to maintain its first line market share, the anticipated confirmatory study should position pona well in 1st line high-risk pts in addition to T315I which, combined with high-risk pts, could readily generate 400MM alone.
At 12 weeks, a 47% MCyR in 3rd/4th line CP pts is actually slightly better than sprycel/tasigna achieved in 2nd line. The drug is approvable based on the 65% MCyR in T315I pts alone, but the data should remove any doubt that the company is on track to file an NDA in 2012.
ARIAD Announces Preliminary Data from Pivotal Pace Trial of Ponatinib, Its Investigational pan-BCR-ABL Inhibitor
http://phx.corporate-ir.net/phoenix.zhtml?c=118422&p=irol-newsArticle&ID=1638434&highlight=
ARIAD will hold an investor meeting and webcast at 7:00 am (PST) on Monday, December 12, 2011 to review these data being presented at ASH.
Ariad mentioned in Globe article, "Push is on to stay one step ahead of cancer."
Yes, Sabril and CPP109 are both formulations of vigabatrin. CPRX acquired Brookhaven's patent portfolio for use in addiction treatment while Lundbeck has patents for use in CNS. As far as boom or bust goes, at a $25mm market cap, I'd say the "bust" happened when the P2a failed to meet its endpoint. At around the time of the NIDA partnership, I put it on my watch list and started researching the company. fwiw, I've only suggested a couple of stocks on this board (GHDX when it was around $14 and ARIA around $2) and, other than responding to your post, probably wouldn't have even mentioned CPRX.
Don't get me wrong, mgt has made some mistakes not the least of which was the poor P2a trial design but overall they've progressed the pipeline while maintaining one of the cleanest balance sheets I've seen for a bio at this stage (one of the side benefits,imo, of senior mgt maintaining such a large ownership position). In fact, until the latest financing, the comnpany managed to raise $'s without issuing warrants through direct placements with Fidelity and Federated which is not something you usually see for a company this size.
I don't find the lack of a partner for CPP115 at this stage of development all that unusual...not many drugs are partnered pre-clinical. And with NIDA picking up 75% of the CPP109 trial, they de facto partnered the trials "cost" without giving up the upside.
bottomline, it's a small market cap bio with two interesting drugs that's fully funded through the next 2 or 3 catalysts.
All the best with BIOD.
...Don
in response to several posts on the biotechvalues board regarding CPRX:
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=69170267
What I appreciate most about this board is it's focus on due diligence and the mutual respect posters show here. We all have different investment styles/risk tolerances but that doesn't mean that we can't learn from one another.
First, it's vigabatrin not vigapatin. Second, at a 25mm market cap, CPRX isn't on any ones radar screen (although Cowen recently initiated coverage with an "outperform"). The market completely discounted the stock after the company failed to meet its primary endpoint in its P2a trial of vigabatrin aka CPP109. However, the trial was poorly designed. Basically, they relied on addicted patients to "self-report". Not surprisingly, almost 2/3 didn't take the prescribed dose, go figure. As a result of the low compliance, the trial ended up underpowered.
Although post-analysis, cherry picking of the data is usually a big red flag, in this case, because of the non-compliance issue, it offers a reasonable explanation of why the trial failed. When you break out the 35% that did take the drug, the trial actually easily met its endpoint. Apparently the National Institute on Drug Abuse and VA reached the same conclusion which is why they are picking up 75% of the cost to run the P2b trial, which is currently enrolling.
Vigabatrin (brand name Sabril) is already approved for use in infant epilepsy but, as you point out, the drug has serious AE's:
Black Box Warning Label: VISION LOSS
Sabril causes progressive and permanent bilateral concentric visual field constriction in a high percentage of patients. In some cases, Sabril may also reduce visual acuity.
Risk increases with total dose and duration of use, but no exposure to Sabril is known that is free of risk of vision loss.
Risk of new and worsening vision loss continues as long as Sabril is used, and possibly after discontinuing Sabril.
Periodic vision testing is required for patients on Sabril, but cannot reliably prevent vision damage.
Because of the risk of permanent vision loss, Sabril is available only through a special restricted distribution program.
Pre-clinical trials indicate that CPRX's 2nd drug CPP115 offers a far better safety profile and efficacy then Sabril, the drug Lundbeck acquired for $900mm (600mm + 300mm in milestone payments) in 2009. Sabril is now generating ~ $100mm in revenue despite the rather dire black box warning label. Although just entering clinical trial, CPP115 makes CPRX far more interesting to me from an investment perspective.
So, relative to its current market cap I really like the company's pipeline and their competitive position. Beyond this the CEO has maintained a significant owner position and has successfully raised $'s with high quality investors such as Fidelity and Federated.
CPRX is definitely not every ones cup of tea. CPP109 failed its P2a and CPP115 is just entering trial but I wouldn't write it off the investment opportunity here so quickly. The stock is currently trading at $1.13, lets just see where things stand after the CPP115 initial results.
while retail investors are probably focused on CPP109's potential in addiction treatment, as far as take-out speculation goes, the epilepsy drug, CPP115, is what will more likely attract buyout interest. Ovation Pharma's Sabril (vigabatrin) had just received approval, when Lundbeck acquired them for $600mm+ two years ago. Sabril is now generating ~ $100mm in revenue despite a rather dire black box warning label:
Black Box Warning Label: VISION LOSS
Sabril causes progressive and permanent bilateral concentric visual field constriction in a
high percentage of patients. In some cases, Sabril may also reduce visual acuity.
Risk increases with total dose and duration of use, but no exposure to Sabril is known that
is free of risk of vision loss.
Risk of new and worsening vision loss continues as long as Sabril is used, and possibly
after discontinuing Sabril.
Periodic vision testing is required for patients on Sabril, but cannot reliably prevent vision
damage.
Because of the risk of permanent vision loss, Sabril is available only through a special
restricted distribution program.
btw, pre-clinical, CPP115 has shown greater efficacy and a far better safety profile at lower doses. I'm highly anticipating the P1 data due Q1/2 next year. If the data proves out, I would think at the very least Lundbeck would want to protect its investment in Sabril.
With CPP115 about to enter clinical trial, I'm glad to see this appointment. My guess is that any partnership deal will likely include both CPP109 and CPP115.
btw, based on today's price/volume we may have put in a bottom.
biomaven, wondering what you think about the idea the company floated last week to use the confirmatory study to run a head to head trial with gleevec? If ponatinib is able to demonstrate an improved molecular response rate over gleevec, do you think it may allow the company to make the case that high risk pts should start on pona or, at the very least, position pona more competitively in the 2nd line vis-a-vis sprycel/tasigna? Since sprycel/tasigna have both run 1st line CP trials vs gleevec, running a similar trial indirectly allows a comparison between pona and sprycel/tasigna without immediately running the head to head, mult-arm trial that will be needed down the road.
In the first line setting, 15% of gleevec pts develop resistance from mutations. Although another post recently attributed all 15% to the T315I mutation, the T315I mutation is responsible for only about half. Using a 7% incidence and gleevec's current run rate, i peg the 2nd line ponatinib T315I opportunity at around $300mm.
50% of sprycel/tasigna pts eventual progress so even in a third line setting, ponatinib has significant commercial opportunity. Based on current sprycel/tasigna sales, my "back on the envelope" has ponatinib revenue at around $900mm (to avoid double counting, I'm backing out the 300mm available from the second line).
Attach any reasonable multiple and ponatinib, alone, is worth more than ARIA current market cap. fwiw, I had a $12 year end target on the stock so based on current data I feel the stock is close to fairly valued. However for anyone with more than a short-term time horizon, there are significant catalysts (namely, rida approval, ponatinib NDA, AP113 P1/2 results) upcoming in 2012 that should push the stock higher. I don't know about Dew, but I can certainly find a better bio to short.
several slides referencing Ridaforolimus from Merck' research day presentation
http://www.sec.gov/Archives/edgar/data/310158/000095012311097292/y93336y93336l007.gif
http://www.sec.gov/Archives/edgar/data/310158/000095012311097292/y93336y93336z0252.gif
Merrill's expectation of a 70%+ MCyR for T315I pts. after only 57 days was unrealistic to begin with. In the Phase 1 trial, 82% of CML CP pts with T315I mutation had CHR but that was after 142-599 days. My expectation is that the updated data available at the presentation will confirm the response rates are moving in the right direction.
Basically, yesterdays sell off seems to be a combination of unrealistic response rate expectations and a misreading of the SAE data. Typical knee jerk reaction by short term "investors" who don't bother to read more than just the headlines.
fwiw, even if it meant additional dilution in Q1/Q2, i'd much rather own 100% of pona in US and EU and all of 113 going forward. even at today's discounted price, the company could raise almost $150mm with around 10% dilution.
The company has another potential $40mm in upcoming approval milestones associated with rida. When you take their current cash position and burn rate into account and factor in that MRK is covering the co-promote expenses, there is no immediate pressure to raise $'s - even without partnering EU or Asia. My guess is the company will look to time the next financing around the rida approval and/or run-up to the ponatinib filing (that being said, harvey has surprised me with financings before,lol)
I think the key point is the company "has many good term sheets from potential partners" which gives them some additional flexibility.
"Management reiterated that it is keeping US marketing rights and is finalizing its plans for Europe, although it appears
likely to maintain EU rights as well. It has many good term sheets from potential partners. Ariad is moving ahead with a
Japanese IND in 1H:12 independent of the deliberations on Asian strategies. Management emphasized that the company is
not out raising money and it has cash through the end of 2012 without potential milestone on ridaforolimus approval
($25M)"
http://www.healthace.com/rssfeed/Oncology_Report_101911M.pdf
For comparison, here's the first read of the phase 1 trial from ASH last year. http://ash.confex.com/ash/2010/webprogram/Paper31434.html
Initial Findings From the PACE Trial: A Pivotal Phase 2 Study of Ponatinib in Patients with CML and Ph+ ALL Resistant or Intolerant to Dasatinib or Nilotinib, or with the T315I Mutation
Program: Oral and Poster Abstracts
Type: Oral
Session: 632. Chronic Myeloid Leukemia - Therapy: Resistance to Tyrosine Kinase Inhibitors
Sunday, December 11, 2011: 4:30 PM
Ballroom 20BC (San Diego Convention Center)
Jorge E. Cortes1, Dong-Wook Kim2, Javier Pinilla-Ibarz, MD, PhD3, Philipp D. Le Coutre, Priv. Doz. Dr.4*, Charles Chuah, MBChB, FRCPE, MD5*, Franck E Nicolini, MD6, Ron Paquette, MD, PhD7*, Jane F Apperley, MBChB, MD, FRCP, FRCPath8, John F. DiPersio, MD, PhD9, H. Jean Khoury, MD, FACP10, Delphine Rea, MD, PhD11*, Moshe Talpaz, MD12*, Daniel J. DeAngelo, MD, PhD13*, Elisabetta Abruzzese, MD14, Michele Baccarani, MD15, Martin C Mueller, MD16*, Carlo Gambacorti-Passerini, MD17, Stephane Wong, PhD18, Stephanie Lustgarten19*, Christopher D. Turner, MD, FAAP19, Victor M. Rivera, PhD19*, Tim Clackson, PhD19, Frank Haluska, MD, PhD19, Hagop M. Kantarjian20 and The PACE Study Group19*
1The University of Texas MD Anderson Cancer Center, Houston, TX
2Seoul St. Mary’s Hospital, The Catholic University of Korea, Seoul, South Korea
3H. Lee Moffitt Cancer Center, Tampa, FL
4Charité - University of Medicine Berlin, Berlin, Germany
5Singapore General Hospital, Duke-NUS Graduate Medical School, Singapore, Singapore
6Hôpital Edouard Herriot, Lyon, France
7Ronald Reagan UCLA Medical Center, University of California, Los Angeles, CA
8Department of Haematology, Hammersmith Hospital, Imperial College London, London, United Kingdom
9Washington University School of Medicine, St. Louis, MO
10Emory Winship Cancer Institute, Atlanta, GA
11Service des Maladies du Sang, Hôpital Saint Louis, Paris, France
12Comprehensive Cancer Center, University of Michigan, Ann Arbor, MI
13Dana-Farber Cancer Institute, Boston, MA
14Ematologia e Oncologia, Ospedale S. Eugenio, Rome, Italy
15Department of Hematology-Oncology 'L. and A. Seragnoli,' S Orsola-Malpighi University Hospital, Bologna, Italy
16III. Med. Klinik, Universitätsmedizin Mannheim, Mannheim, Germany
17Unità di Ricerca Clinica - Ematologia, Azienda Ospedaliera San Gerardo/University of Milano Bicocca, Monza, Italy
18MolecularMD Corp, Portland, OR
19ARIAD Pharmaceuticals Inc, Cambridge, MA
20Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX
Background: Despite progress in chronic myeloid leukemia (CML) therapy with tyrosine kinase inhibitors (TKIs), patients (pts) who fail dasatinib or nilotinib or pts with T315I mutation have no treatment options. Ponatinib is a potent, oral, pan-BCR-ABL inhibitor active against the native enzyme and all tested resistant mutants, including the uniformly resistant T315I mutation. Methods: The PACE trial (Ponatinib Ph+ALL and CML Evaluation) was initiated in September 2010. The objective of this international, single-arm, open-label, phase 2 trial is to establish the efficacy and safety of ponatinib. Pts with refractory CML in chronic, accelerated or blast phase (CP, AP or BP), or Ph+ acute lymphoblastic leukemia (ALL), resistant or intolerant (R/I) to dasatinib or nilotinib or with the resistant T315I mutation received 45 mg ponatinib orally once daily in one of 6 cohorts: CP R/I; CP T315I; AP R/I; AP T315I; BP/ALL R/I; BP/ALL T315I. The primary endpoints are major cytogenetic response (MCyR) for CP and major hematologic response (MaHR) for AP, BP or ALL. The trial is ongoing; projected enrollment is approximately 450. Data as of 18 July 2011 are reported. Results: At analysis, 403 pts were enrolled; 397 were treated and eligible. The median age was 59 (range, 18-94) years, 52% were male. Diagnoses were: CP R/I, n=188; CP T315I, 48; AP R/I, 52; AP T315I, 15; BP/ALL R/I, 51; BP/ALL T315I, 43. Median time from initial diagnosis to start of ponatinib was 6.2 years. Prior TKIs included imatinib (93%), dasatinib (85%), nilotinib (66%), and bosutinib (8%); 94% failed >2 prior TKIs, and 57% failed >3 prior TKIs. Overall, 88% had a history of resistance to dasatinib or nilotinib, and 12% were purely intolerant. Mutation status was determined centrally by MolecularMD. Overall, 106 pts had the T315I mutation. Of 291 R/I pts, 110 (38%) had non-T315I BCR-ABL mutations, most frequently F317L (10%), F359V (5%), E255K (4%), and G250E (4%). To date, 343 (85%) pts remain on therapy, 60 (15%) have discontinued (42 BP/ALL): 24 (6%) progressive disease (20 BP/ALL); 11 (3%) AE (3 pain, 3 thrombocytopenia, 1 each haemorrhage, loss of consciousness, enterocolitis, cytokine release syndrome, hepatotoxicity/pleuro-pericardial effusion after overdose); 8 (2%) died (3 related; 7 BP/ALL); 17 (4%) other. The most common drug-related AEs (=10% any grade) were thrombocytopenia (19%; 15% grade 3/4), rash (18%), dry skin (13%), myalgia (12%), abdominal pain (11%; 3% grade 3/4), headache (11%), arthralgia (11%). Overall, 67 (17%) pts experienced at least 1 related SAE. The most common related SAEs (>5 cases) were pancreatitis 15 cases (3.7%), 5 cases each (1.2%) diarrhea, anemia, febrile neutropenia, and pyrexia. At the time of reporting, 159/397 eligible pts were evaluable for the primary endpoints. Median follow-up was 57 days. Of CP pts, 83 had an assessment at 3 months (10 at 6 months) or discontinued. In CP R/I, 25/60 (42%) attained MCyR (15 CCyR). In CP T315I, 13/23 (57%) had MCyR (11 CCyR). The overall CP MCyR rate was 38/83 (46%) (26 CCyR). Of AP, BP/ALL pts, 76 had an assessment at 1 month or later or discontinued. In AP, 17/23 (74%) R/I and 1/1 T315I pts achieved MaHR. In BP/ALL, 11/30 (37%) R/I and 6/22 (27%) T315I pts had MaHR. Conclusion: In this first analysis of the pivotal PACE trial, ponatinib has a favorable early safety profile, similar to that observed in phase 1, but with a lower incidence of pancreatitis. Initial response data after short follow-up indicate ponatinib has substantial anti-leukemic activity in this heavily pretreated population, and in pts with refractory T315I. These early efficacy signals replicate initial response results reported in the phase 1 setting. Updated data will be presented at the annual meeting.
http://ash.confex.com/ash/2011/webprogram/Paper41105.html
There should be a link available from the meeting home page after 10 a.m.
yes, but the heartmate II is only approved for pts ineligible for transplantation (e.g. with end-stage, class IV symptoms). the more significant opportunity for LVADs is as a potential alternative to transplant or in pts not severe enough to qualify for transplant http://circ.ahajournals.org/content/123/14/1552.extract
to that end, HTWR is just starting a new P2 trial enrolling pts with class III symptoms not yet eligible for transplant.http://clinicaltrials.gov/ct2/show/NCT01369407?spons=%22HeartWare%2C+Inc.%22&spons_ex=Y&rank=3
When one considers that 100,000 patients need transplants each year but only 2,000 get them, I believe left ventricular assist devices are one of the more interesting investment opportunities in the medical device space.
Of patients admitted to hospital with acute heart failure, the 1-year mortality is 30–50%. Currently, I believe THOR's LVAD has bridge to transplant approval but the real winner will be the device that first gets US approval for use as a destination therapy and I believe HTWR has the lead. On the other hand, both THOR and HTWR make interesting potential buy out candidates.