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When a small-cap turdling biotech CEO says, "they have existing cash to get them well into 2014"....expect a raise within 3 months.
I am long. But don't like (a) when companies say aren't in need of money, and (b) infer they have all these partners lined up, but never sign a deal.
These guys are going to have to raise more capital; I doubt very much (after speaking with them) that they are remotely close to signing a partner for '011.
Sounded more like all I got was excuses like, "potential partners want to see results of FDA talks" or "potential partners want to see more clarity on follow-up data". So, it's not like all of a sudden after getting clarity on those two things, a partner is going to up and run to them with the signed term-sheets. I would be very surprised, actually, to see them ever partner '011, and go it alone through all these trials, and just keep on diluting like every other JV biotech management team seems to be doing.
Having said that, IF '011 results are outstanding (i can't see how they will be MASSIVELY outstanding with the small N), they probably should keep it 100%.
True re: the historical data, but they data they use seems to be matched up well with what you would expect.
Too bad the Phase 3 is going to take forever.
CLDX
Right. If the CVR payout on just the approval with the OS label alone is around $7.40, I can't see any reason why he would take anything less than this. The $7.40 doesn't even take into consideration any of the future royalties due; and, it's pretty clear to me that this will be the new SOC - further, melanoma and other cancers will put this well over a $1 billion drug (of which CVRs payout above $1B)
They would have to negotiate with Patrick Soon who owns something like 80% of the CVR, if they wanted to buy back the CVR.
ARRY
17 million priced at 3.65
I would have to say that if you invest in ARRY, you are investing in an incompetent management team. That is just downright ugly.
BTW. 2016 or later? LOL, thats three (or more) years away. How many more dilutions before then?
Randomized Discontinuation Designs in Early Cinical Drug Screening ? The Cabozantinib Experience
Speaker: P. Lamb (USA)
Cabozantinib (cabo) is a potent, targeted therapy that inhibits MET & VEGFR2. MET & VEGF signaling are implicated in tumor angiogenesis, invasion & metastasis. In the initial Phase I clinical study, treatment with cabozantinib resulted in a high rate of stable disease (SD) across multiple tumor types. In order to broadly evaluate efficacy in a single Phase 2 study, an adaptive randomized discontinuation trial (RDT) design was implemented (Ratain et al. 2006).
Nine tumor types were selected for evaluation based on the role of MET & VEGFR in tumor biology. The trial design incorporated stopping rules based on the week-12 disease control (partial response or SD per RECIST) rates for the first 20 patients (pts) in each tumor cohort, & up to 2 cohorts could be fully expanded based upon review of the clinical activity observed. Treatment beyond wk 12 was based on wk 12 response: pts with confirmed response continued open-label cabo, pts with stable disease were randomized to cabo vs. placebo, & pts with progressive disease (PD) were discontinued. The primary endpoints were objective response rate (ORR) per RECIST in the 12 wk Lead-in Stage, & PFS in the Randomized Stage. Response per RECIST criteria was assessed every 6 wks. As originally outlined in the protocol, accrual to any cohort could be halted for either high rates of ORR or PD.
Overall, 526 pts were enrolled from September 2009 to July 2011. Pts with progressive measurable disease (RECIST) received 100mg cabo PO qd over a 12 wk Lead-in Stage. Three cohorts--small cell lung, pancreatic, & gastric cancer – were not expanded due to low rates of disease control at wk 12. The remaining 6 cohorts (prostate, ovarian, hepatocellular, breast, non-small cell lung, & melanoma) showed encouraging levels of clinical activity that warranted further expansion. The prostate, ovarian, & hepatocellular cancer cohorts showed the highest rates of disease control at wk 12. The unique & unexpected observations of bone scan improvement, pain improvement, & a narcotic-sparing effect in the prostate cancer cohort, as well as high rates of soft tissue regression in the prostate & ovarian cancer cohorts, led to the randomization in these two cohorts being halted. In addition, the protocol was amended to add non-randomized expansion cohorts for both tumor types, including incorporation of a novel primary endpoint of bone scan response in the prostate cohort.
This adaptive RDT design provided distinct advantages as a signal-search study, including determination of ORR in 9 well-defined populations. However, certain disadvantages relative to more traditional Phase 2 designs became evident early in the trial, including randomization to placebo of pts who had stable disease by RECIST but were clearly experiencing other measures of clinical benefit. Thus, the success of this trial design depends on the timely retrieval & analysis of data, allowing for the potential suspension of the randomized discontinuation component in select tumor types where a stronger than anticipated efficacy signal is observed.
Is there any data out there right now for people who have become resistant to ponatinib?
Don?
If its intended to be used in the salvage setting, wouldn't ponatinib take all those cases anyway?
It's orally available, and better efficacy.
So you seem to think Synribo is going to be a player in the CML space?
If so, I don't see this as even being remotely the case.
Teva Receives Approval For SYNRIBOTM (Omacetaxine Mepesuccinate) for Injection
**My thoughts: this should get about 5-10 patients in total
Treats Chronic Myeloid Leukemia (CML) in Patients Who Have Become
Resistant to or Intolerant of Two of More Tyrosine Kinase Inhibitors
Protein Synthesis Inhibitor Provides New Treatment Option for CML
Patients who Fail Prior TKI Therapy
Business Wire11:30 AM Eastern Daylight Time Oct 26, 2012
Teva Pharmaceutical Industries Ltd. (NYSE: TEVA) announced today that the U.S. Food and Drug Administration (FDA) approved SYNRIBO (omacetaxine mepesuccinate) for Injection to treat adult patients with chronic phase (CP) or accelerated phase (AP) chronic myeloid leukemia (CML) with resistance and/or intolerance to two or more tyrosine kinase inhibitors (TKIs). The indication is based upon response rate. There are no trials verifying an improvement in disease-related symptoms or increased survival with SYNRIBO. It will be available for prescribing shortly.
Previously, CP and AP CML patients who failed on two or more TKIs have had limited treatment options. “While the CML treatment landscape has seen advancements with available TKI treatments, there are still cases where patients may not be able to continue using TKIs due to issues such as resistance, intolerance, suboptimal response, and disease progression,” said Jorge E. Cortes, M.D., deputy chair and professor of medicine in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. “With SYNRIBO, physicians will now have access to another option, offering potential hope to patients who experience treatment failure.”
SYNRIBO received an accelerated approval that allows the FDA to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. The program is designed to provide patients with earlier access to promising new drugs. Full approval is expected following submission of more mature data from pivotal analysis.
“Teva Pharmaceuticals is pleased to bring SYNRIBO to the market for patients who need additional treatment options when others have failed,” said Michael R. Hayden, M.D., Ph.D., President of Global R&D and Chief Scientific Officer of Teva Pharmaceutical Industries Ltd. “SYNRIBO joins TREANDA and TRISENOX as important hematologic treatment options in the Teva Oncology portfolio.”
The approval is based on an analysis of combined data subsets from two Phase II, open-label, multicenter studies. The pooled analysis included patients who had received 2 or more approved TKIs and, at a minimum, had evidence of resistance or intolerance to dasatinib and/or nilotinib. 47% of CP patients and 63% of AP patients had failed treatment with imatinib, dasatinib, and nilotinib. The majority of patients had also received other treatments including hydroxyurea, interferon, and cytarabine.
For CP patients, 18% (14/76) achieved a major cytogenetic response (MCyR) with a mean time to MCyR onset of 3.5 months. The median duration of MCyR for these patients was 12.5 months (Kaplan-Meier estimate).
For AP Patients, 14% (5/35) achieved a major hematologic response (MaHR) with a mean time to response onset of 2.3 months. The median duration of MaHR for these patients was 4.7 months (Kaplan-Meier estimate).
Most common adverse reactions (frequency =20%) in chronic and accelerated phase patients: thrombocytopenia, anemia, neutropenia, diarrhea, nausea, fatigue, asthenia, injection site reaction, pyrexia, infection, and lymphopenia
Administered subcutaneously, SYNRIBO will be dosed twice daily for 14 consecutive days of a 28-day cycle at treatment induction, and twice daily for seven consecutive days of a 28-day cycle during maintenance therapy once a response is achieved.
What NYC forum are we talking about here. Need some context and who the people in the panel were.
Thanks.
I think that if you have a drug that shows clear evidence of survival (something like ponatinib, or ibrutinib type of drugs aka game changers) NICE isn't going to be a problem.
It will be a problem for drugs and those drug companies that show statistically signiifcance enough to meet an SPA but have almost little to no clinical benefit ..ie., a drug that shows a PFS benefit of 2.5 weeks, or something along those lines, in which an oncology company thinks just because it met statistical criteria and was approved, it deserves a $80,000 price tag.
Give me a break.
Institutional ownership is close to 85%...
This is what happens when everyone is "all in". When a few firms want to take profits on their 100% gains, they sell. Period.
To single out SAC is laughable. Why not Blackrock, or some other plain-vanilla mutual fund who are getting redemptions...or just want to take profits?
ARIAD downgraded to Perform from Outperform at Oppenheimer
Oppenheimer downgraded ARIAD citing valuation but raised its price target for shares to $23 from $20.
ARIAD price target raised to $30 from $25 at Stifel Nicolaus
After surveying 35 hematology/oncology specialists, Stifel Nicolaus believes the survey suggests that ARIAD's ponatinib in front-line CML has significant potential. The firm thinks the company's outlook for sales of the drug is conservative and it maintains a Buy rating.
These damn Chicago Boys will do anything.....
tivantinib
PS Interesting that ARQL popped some yesterday (before this PR)
If the company only had cash and a CEO....CYCC
No insider selling.
They likely could not sell ahead of the WMS conferences which data constituted an 8-K event.
Maybe they'll sell now? Or maybe not...if there is a partnership coming.
I wonder if they do a 10b-1 planned sale....
I have no position in the stock anymore.
But sometimes I think common sense and easy thinking is better than over-analyzing and trying to show off your skills you've learned in getting that PhD.
Exactly. Why not give it some sort of conditional approval. Whats the downside..theres no side effects. Clearly, Max is walking and running around now....and its not because the drug is an inert placebo...its clear that the drug has a big effect in some sort of patient.
Does anyone actually think there is a placebo effect here? LOL...look at the videos. Quite an amazing sugar serum!!
Yeah, I dnt understand why this is a "big red flag".
Basically, if I amputate your legs because you got gangrene, theyre not repairable.. If I break your legs and you get medical attention, they might be repairable, and you might be able to walk again.
Why is it hard to believe that if a DMD patient is so far gone, that they cant recover, regardless of how great a drug might be?
FDA APPROVES ABRAXANE FOR THE FIRST-LINE TREATMENT OF ADVANCED NON-SMALL CELL LUNG CANCER
Celgene Corporation (NASDAQ: CELG) today announced the U.S. Food and Drug Administration (FDA) has approved ABRAXANE® (paclitaxel protein-bound particles for injectable suspension) (albumin-bound) for the first-line treatment of locally advanced or metastatic non-small cell lung cancer, in combination with carboplatin, in patients who are not candidates for curative surgery or radiation therapy.
“Non-small cell is the most common type of lung cancer, the leading cause of cancer death in the United States,” said Dr. Mark A. Socinski, MD, Director, Lung Cancer Section, Division of Hematology/Oncology, University of Pittsburgh, and lead investigator of ABRAXANE phase II and phase III lung cancer trials. “The FDA approval of ABRAXANE is exciting for healthcare professionals because it offers an important new treatment option for all types of non-small cell lung cancer patients, in an area that has seen few treatment advancements in recent years.”
The ABRAXANE sNDA approval is based upon the results of CA-031, a phase III, multi-center, randomized open-label study where patients with advanced non-small cell lung cancer (NSCLC) received either ABRAXANE (100mg/m2) weekly plus carboplatin (AUC=6) every three weeks (n=521) or paclitaxel (200mg/m2) every three weeks plus carboplatin (AUC=6) (n=531). The study met its primary end-point demonstrating a statistically significantly higher overall response rate for patients in the ABRAXANE arm compared to those in the paclitaxel arm (33% vs 25%).
In the phase III study, ABRAXANE demonstrated a higher overall response rate as compared to paclitaxel for squamous cell carcinoma (41% vs. 24%) and large cell carcinoma (33% vs. 15%). ABRAXANE achieved a similar overall response rate to paclitaxel in patients with carcinoma/adenocarcinoma (26% vs. 27%).
The most common adverse reactions (=20%) of ABRAXANE in combination with carboplatin for NSCLC are anemia, neutropenia, thrombocytopenia, alopecia, peripheral neuropathy, nausea, and fatigue.
Additional regulatory submissions have been filed in Japan, Australia and New Zealand with anticipated decisions in 2013.
I truly, cannot, believe, people take this guys advice. He has over 2,000 followers on Twitter and people think this guy is legit. If he ever had an interview with a hedge fund he would be laughed out of the room.
He really thinks hes something special !
http://stockmatusow.com/?p=10
Just to review:
Was there an ODAC meeting for this?
The play-by-play on the daily ticks here is epic.
Vismodegib Breast Cancer; resistance
Tomorrow could be interesting..
Ibrutinib and 113 are two entirely different classes of drug in separate indications and markets.
But 113 is very probable, a billion dollar drug; even though its early.
FYI (I dont know if this has been posted yet)
A lot of comments on Twitter right now on Summer Street research call on SRPT.
Hey! Where's Scott Mattisow? Didn't he say about 10 points ago that "pronatrib" would fail because it was a "similar compound as 'reederforoleemus' and that it was a good short?"
Scott?
Is this a scale issue, or does it sctually take a long time to produce one dose of this drug?
What I am trying to get at is: is the drug production here extremely complicated, or, is it just scale.
LOL i thought you were serious for a second.
This is funny.
Definitely, SAC?
I think the simple takedown of this failed trial is this: if you have a highly targeted molecule, focus highly on that specific target and patients who specifically benefit from the drug - and don't dick around with all-comers or pseudo all-comers trials. This MARQUEE trial reminds me of a transitional trial from the days of those "kitchen sink" trials, into a true biomarker driven, diagnostic, new paradigm trial. MARQUEE was shortcut to something in between...."lets do a biomarker trial, sorta, but lets not truly drill down to the patients which will show the most success."