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SPPI - I don't follow SPPI nor have I listened to the call. However if nothing was mentioned about future/ongoing trials during the call then its probably safe to say the trials below will be terminated.
http://clinicaltrials.gov/ct2/results?term=apaziquone&recr=Open
Also interestingly while SPPI has updated the product page with the latest trial results the bottom of the page still mentions the above mentioned additional P3 trials.
http://sppirx.com/eoquin.html
Incremental positive in the low NMIBC indication? I presume you are referring to competition. There are additional multi-instillation apaziquone trials and if pooled data was positive perhaps that bodes well for those trials?
I believe OGXI is currently running a P1 for the stage of bladder cancer that Apaziquone failed in. The 427 P2 trial is for more advanced bladder cancer.
I need to go to the SI site more often:) Is there a separate list of the stocks that were picked most often? A cursory glance indicates ARRY and MNTA were the most popular.
Yes. I was clearly careless in skimming through most of the article even though one of the lines i quoted also indicated he was only considering cancer drugs. However my point still holds. IMO the marketcap is indicative of how valuable the drug is to the company and a 300 M limit cannot be used to predict whether a trial would be successful. Consider CRIS which was less than 200M before the BCC results. Even EXEL may not have had a 300M MC before the MTC results if the market hadn't known that cabo was active in PC.
BMTI - Their quarterly CC was very downbeat. The processing of the EU application had been suspended indefinitely and they pushed announcing the canadian augment injectable trial results to the 2nd half of 2012. The company also seemed very uncertain that reanalysis of the augment trial data would be good enough to even submit to the FDA. They will end the year with about 30-40 million. I think this would have been a decent bet with an impending EU decision but since the application has been suspended indefinitely and its trading above cash its a lot more risky to hold.
The CEO also bought about 100K of shares in may of 2011 when the share price was ~8.
AVII - I think a homerun PII was what the market was hoping for. The barrons article indicated they may have filed for approval with significant improvements in clinical outcome. Its now unlikely that they will file for approval and there is the possibility that the Prosensa/GSK trial will be +ve. If that happens AVII will have a higher hurdle to overcome. There are just a limited number of DMD patients so AVII will have to prove that their drug is much better. So the next catalyst may be negative results from the Prosensa trial.
BTW regarding ANTH do you really feel that the lupus trial will be positive with the pooled data? Don't you think the lower dose groups will hamper the chances of the pooled data being stat sig?
Adam F seems to be contradicting himself somewhat. He writes
Many investors ridiculed our theory last fall because they mistakenly thought we were simply predicting the outcome of phase III cancer drug trials based on market cap. That's not true.
Market cap, therefore, becomes a reliable and accurate proxy for predicting cancer drug trial outcomes.
AVI BioPharma Announces Eteplirsen Meets Primary Endpoint, Demonstrating a Significant Increase in Dystrophin at 24 Weeks Compared to Placebo in Phase IIb Trial for the Treatment of Duchenne Muscular Dystrophy
From a layman's point of view, the results seem underwhelming. Plus with patent issues and Prosensa/GSK being way ahead in their DMD program, probably a risky investment at this point
BOTHELL, WA, Apr 02, 2012 (MARKETWIRE via COMTEX) --AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced that treatment with eteplirsen met the primary efficacy endpoint in a randomized, double-blind, placebo-controlled Phase IIb study in boys with Duchenne muscular dystrophy (DMD). Eteplirsen administered once weekly at 30mg/kg over 24 weeks resulted in a statistically significant (p = 0.002) increase in novel dystrophin (22.5% dystrophin-positive fibers as a percentage of normal) compared to no increase in the placebo group.
"This study represents a major advance in the field of DMD research as the results indicate that eteplirsen is producing consistent levels of dystrophin, which is the essential protein that these patients need," said Jerry Mendell, M.D., Director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital and principal investigator of the Phase IIb study. Dr. Mendell added, "We anticipate that these levels of dystrophin could lead to significant clinical benefit if maintained over a longer course of treatment."
In the study, a shorter duration of eteplirsen treatment, 12 weeks, did not show a significant increase in novel dystrophin (0.79% dystrophin-positive fibers as a percentage of normal; p-value NS), despite administration of the drug at a higher dose (50mg/kg once weekly). This finding suggests that a longer duration of dosing is required before meaningful levels of dystrophin are produced. There were no significant improvements in clinical outcomes in the treated groups compared to placebo. Performance on the 6-minute walk test and other outcome measures were generally stable across most of the patients, including the placebo patients, suggesting that a longer period of observation will be required to demonstrate clinical effects of eteplirsen versus a placebo control.
Eteplirsen was well tolerated at both dose levels through 24 weeks of treatment. There were no treatment-related adverse events, no serious adverse events, and no treatment discontinuations related to eteplirsen. Furthermore, no treatment related changes were detected on any safety laboratory parameters, including several biomarkers for renal function.
"We are very encouraged by the results of this first placebo-controlled study investigating exon-skipping technology in DMD," said Chris Garabedian, President and CEO of AVI BioPharma. "Eteplirsen represents the first drug candidate for DMD to demonstrate the production of novel dystrophin in a robust and consistent manner and these study results support advancing eteplirsen into a pivotal study."
Conference Call AVI BioPharma, Inc. will hold a conference call to discuss these results today at 8:00 a.m. EDT (5:00 a.m. PDT). The conference call may be accessed by dialing 800.561.2718 for domestic callers and 617.614.3525 for international callers. The passcode for the call is 99858553. Please specify to the operator that you would like to join the "AVI BioPharma Phase IIb Top-Line Data Results Call." The conference call will be webcast live under the events section of AVI's website at www.avibio.com and will be archived there following the call for 90 days. Please connect to AVI's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. An audio replay will be available through April 9, 2012 by calling 888.286.8010 or 617.801.6888 and entering access code 16040637.
About Study 201 (Eteplirsen Phase IIb Study) Study 4658-US-201 was conducted at Nationwide Children's Hospital in Columbus, Ohio. Twelve boys meeting the inclusion criteria being between 7 and 13 years of age with appropriate deletions of the dystrophin gene that confirm eligibility for treatment with an exon-51 skipping drug received double-blind IV infusions of placebo (n=4), 30 mg/kg of eteplirsen (n=4), or 50 mg/kg of eteplirsen once weekly for 24 weeks (n=4). Muscle biopsies for evaluation of dystrophin were obtained at baseline for all subjects and after 12 weeks for patients in the 50 mg/kg cohort and after 24 weeks for patients in the 30 mg/kg cohort. Two placebo patients were randomized to the 30 mg/kg cohort and two placebo patients were randomized to the 50 mg/kg cohort. This study design allowed AVI to investigate the relationship of dose and duration of eteplirsen treatment on the production of dystrophin over the course of the 24 week study.
About Eteplirsen Eteplirsen is AVI's lead drug candidate that is systemically delivered for the treatment of a substantial subgroup of patients with DMD. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.
Eteplirsen uses AVI's novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene. By skipping exon 51, eteplirsen may restore the gene's ability to make a shorter, but still functional, form of dystrophin from mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to improve, stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD.
AVI is also developing other PMO-based exon-skipping drug candidates intended to treat additional patients with DMD.
About AVI BioPharma AVI BioPharma is focused on the discovery and development of novel RNA-based therapeutics for rare and infectious diseases, as well as other select disease targets. Applying pioneering technologies developed and optimized by AVI, the Company is able to target a broad range of diseases and disorders through distinct RNA-based mechanisms of action. Unlike other RNA-based approaches, AVI's technologies can be used to directly target both messenger RNA (mRNA) and precursor messenger RNA (pre-mRNA) to either down-regulate (inhibit) or up-regulate (promote) the expression of targeted genes or proteins. By leveraging its highly differentiated RNA-based technology platform, AVI has built a pipeline of potentially transformative therapeutic agents, including eteplirsen, which is in clinical development for the treatment of Duchenne muscular dystrophy, and multiple drug candidates that are in clinical development for the treatment of infectious diseases. For more information, visit www.avibio.com.
http://investorrelations.avibio.com/phoenix.zhtml?c=64231&p=RssLanding&cat=news&id=1678924
AVI BioPharma Announces Conference Call and Webcast on Monday, April 2, 2012, to Discuss Top-Line Data Results From the Phase IIb DMD Study
BOTHELL, WA, Mar 30, 2012 (MARKETWIRE via COMTEX) --AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced it will hold a conference call at 8:00 a.m. EDT (5:00 a.m. PDT) on Monday, April 2, 2012 to discuss top-line results from its Phase IIb study evaluating eteplirsen for the treatment of Duchenne Muscular Dystrophy (DMD).
The conference call may be accessed by dialing 800.561.2718 for domestic callers and 617.614.3525 for international callers. The passcode for the call is 99858553. Please specify to the operator that you would like to join the "AVI BioPharma Phase IIb Top-Line Data Results Call." The conference call will be webcast live under the events section of AVI's website at www.avibio.com and will be archived there following the call for 90 days. Please connect to AVI's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.
http://investorrelations.avibio.com/phoenix.zhtml?c=64231&p=RssLanding&cat=news&id=1678725
Do you know approximately when the OS data would be mature? I presume if Roche submits for approval without it then they would be hoping for accelerated approval?
THLD - So do all these questions marks present the possibility that Merck KGaA will not consider the pancreatic trial positive?
The Shire trial for Midodrine doesn't appear to be rigorous at all in terms of duration of treatment effect. Based on that perhaps the FDA may consider the ongoing trial. If the FDA agreed on the shire design they appear to be inconsistent.
http://clinicaltrials.gov/ct2/show/NCT01515865
Very mixed results and AZN passed on the drug.
http://www.targacept.com/wt/page/pr_1295392049
The ADHD trial results for 5619 were worse and another ADHD trial reports in Q3 12. I think this trial is unlikely to be positive. I agree with you. Nothing compelling in the pipeline to consider TRGT right now.
BTW I should add that the schizo trial was partly conducted in India. One of your two favorite countries to conduct clinical trials in:)
I don't disagree. The schizophrenia trial that reports in early 2013 is possibly the next catalyst and by that time TRGT will probably have less cash than the current marketcap.
TC5214 is quietly killed.
http://phx.corporate-ir.net/phoenix.zhtml?c=178332&p=irol-SECText&TEXT=aHR0cDovL2lyLmludC53ZXN0bGF3YnVzaW5lc3MuY29tL2RvY3VtZW50L3YxLzAwMDExOTMxMjUtMTItMTMzMTQwL3htbA%3d%3d
And other TRGT clinical news
Targacept, Inc. (NASDAQ: TRGT), a clinical-stage biopharmaceutical company developing novel NNR Therapeutics™, today announced top-line results from two separate exploratory Phase 2 studies of its product candidate TC-6987 conducted in the United States, one in asthma and one in type 2 diabetes. In the asthma study, oral TC-6987 met protocol-defined success criteria (one-sided p < 0.1) on both co-primary outcome measures, change from baseline in forced expiratory volume for adjunct TC-6987 compared to adjunct placebo measured at two time points on day 28 (51ml and 58ml). In the type 2 diabetes study, the primary outcome measure, change in fasting plasma glucose, was not met, and Targacept will not pursue further development of TC-6987 as a treatment for diabetes. TC-6987 is a modulator of the alpha7 neuronal nicotinic receptor (NNR) discovered by Targacept scientists using Pentad™, the company’s proprietary drug discovery platform.
http://www.targacept.com/wt/page/pr_1332791451
On a more serious note, are there any publicly-traded biotechs working on CD47 antibodies?
One Drug to Shrink All Tumors
by Sarah C. P. Williams on 26 March 2012, 3:05 PM |
A decade ago, biologist Irving Weissman of the Stanford University School of Medicine in Palo Alto, California, discovered that leukemia cells produce higher levels of a protein called CD47 than do healthy cells. CD47, he and other scientists found, is also displayed on healthy blood cells; it's a marker that blocks the immune system from destroying them as they circulate. Cancers take advantage of this flag to trick the immune system into ignoring them. In the past few years, Weissman's lab showed that blocking CD47 with an antibody cured some cases of lymphomas and leukemias in mice by stimulating the immune system to recognize the cancer cells as invaders. Now, he and colleagues have shown that the CD47-blocking antibody may have a far wider impact than just blood cancers.
http://news.sciencemag.org/sciencenow/2012/03/one-drug-to-shrink-all-tumors.html
AEs were almost double in drug group at 50mg but no SAEs. Guess it depends on how unmet of a medical need this indication is. If this is an indication with a clear need
Complete NBIX PR below. Doesn't seem that positive. Mgmt says they expected 12.5 mg dose to be ineffective so why not try a higher dose. Also TEAE's were almost double that of placebo for the 50mg dose.
Neurocrine Announces Phase II Results of VMAT2 Inhibitor NBI-98854 for Treatment of Tardive Dyskinesia
COMPANY TO HOST CONFERENCE CALL AND WEBCAST TUESDAY, MARCH 27TH AT 8:00AM ET / 5:00AM PT
SAN DIEGO, March 26, 2012 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ: NBIX) today announced efficacy and safety results from a Phase II trial of NBI-98854 in 37 tardive dyskinesia patients. For the final analysis, data from one site was removed due to the inconsistent and incorrect application of the efficacy assessment protocol. With this site removed, the results showed a significant reduction in tardive dyskinesia symptoms at end of two weeks of active treatment with 50mg once-daily doses of NBI-98854.
"This Phase II trial was extremely informative. NBI-98854 displayed the efficacy and safety data we expected to see in both the 12.5mg and 50mg doses," said Chris O'Brien, Chief Medical Officer of Neurocrine Biosciences. "While we had data inconsistencies at one site, this study has provided us with the necessary information and confidence to move forward into the larger Phase IIb trials as planned."
After database lock and unblinding of study data, the pre-specified statistical assessment was conducted and the normal quality control evaluation of the output performed. An inconsistent pattern of Abnormal Involuntary Movement Scale (AIMS) scores emerged at one of the eight sites that was not evident during the blinded data review. Potential errors in randomization and drug exposure as causes for this data inconsistency were ruled out. However, a review of videotaped AIMS assessments at this single site noted discrepancies between the clinical score and the video record which were well outside of the variability associated with the AIMS. Additionally, videotaped AIMS assessments were reviewed at other sites and found to be administered appropriately.
Based on these findings, the AIMS data from this single site was removed and the statistical assessment was repeated. This post-hoc analysis demonstrated a clinically meaningful and statistically significant improvement in tardive dyskinesia symptoms for the subjects while receiving the 50mg once-daily dose. These subjects had a significant reduction in tardive dyskinesia symptoms at the end of two weeks of active treatment vs. the end of two weeks of placebo (difference in LS mean of 4.2 for the 50mg period vs. the placebo period, p-value=0.002). As expected, the 12.5mg dosing group was not statistically better during the active treatment period than during the placebo period (difference in LS mean of 0.4 for the 12.5mg period vs. placebo period, p-value=0.68).
The improvement in symptomology is also evidenced by the significant improvement in AIMS scores over baseline levels relative to placebo, excluding the one site. NBI-98854 reduced the average baseline AIMS score by 9.2 points in the 50mg period (p-value=0.0004) vs. a reduction of 4.9 points in the 12.5mg period and 4.7 for the placebo periods. A responder analysis also showed improvement in both the investigator reported Clinical Global Impression-Tardive Dyskinesia and the patient reported Patient Global Impression of Change.
When including the data from the site in question, this study did not meet the pre-specified primary endpoint of reducing the AIMS scores during active treatment periods. The efficacy results from the entire study population showed a non-significant reduction in tardive dyskinesia at the end of two weeks of active treatment vs. the end of two weeks of placebo (difference in LS mean of 1.1 for the 50mg period vs. the placebo period (n=15), p-value=0.42) (difference in LS mean of 0.7 for the 12.5mg period vs. placebo period (n=17), p-value=0.59).
The tables below summarize the primary endpoint as well as the responder analyses for all clinical sites as well as the post-hoc analysis.
All Clinical Trial Sites
Baseline
(mean)
Placebo
12.5mg
50mg
Abnormal Involuntary Movement Scale (LS Means)
14.7
9.9
9.1
8.8
p=0.59
p=0.42
Responder Analysis
"Much Improved or Very Much Improved"
Clinical Global Impression-Tardive Dyskinesia
n/a
52%
65%
60%
Patient Global Impression of Change
n/a
39%
53%
60%
Excluding Single Site
Baseline
(mean)
Placebo
12.5mg
50mg
Abnormal Involuntary Movement Scale (LS Means)
14.9
10.3
9.9
6.1
p=0.68
p=0.002
Responder Analysis end of Treatment
"Much Improved or Very Much Improved"
Clinical Global Impression-Tardive Dyskinesia
n/a
46%
67%
80%
Patient Global Impression of Change
n/a
38%
62%
80%
Safety Profile
NBI-98854 was generally safe and well tolerated; the frequency of treatment-emergent adverse events was 17% during the placebo period and 24% and 32% in the 12.5mg and 50mg treatment periods, respectively. There were no serious adverse events during the treatment period. The most common adverse event was headache and one subject in the 50mg group discontinued due to akathisia. The underlying psychiatric state of subjects was monitored using the Brief Psychiatric Ratings Scale (BPRS) and shown to be stable or improved across study groups declining from 32 at baseline to 28 at the end of the study. There were no drug-drug interactions identified in subjects who were utilizing a range of psychotropic and other concomitant medications.
"While not ideal, this study served its primary purpose of informing the larger Phase IIb studies. The 50mg once-daily dose of NBI-98854 provided tardive dyskinesia sufferers with a remarkable improvement of symptoms, coupled with an excellent safety and tolerability profile," said Kevin C. Gorman President and Chief Executive Officer of Neurocrine Biosciences. "We will apply additional controls in future studies to ensure appropriate scoring of AIMS."
Trial Design
This trial was a randomized, double-blind, placebo controlled, cross-over, Phase II clinical trial utilizing NBI-98854 in tardive dyskinesia patients at eight investigator sites. This 37 subject study assessed once-daily NBI-98854 (12.5mg and 50mg) over a two week dosing period. The primary endpoint of the study was a comparison of placebo vs. active scores utilizing the Abnormal Involuntary Movement Scale (AIMS).
Next Steps for NBI-98854
A placebo controlled, double-blind, parallel design, multiple dose, twelve week Phase IIb study is planned to assess six-week dosing of NBI-98854 against placebo, followed by six weeks of active treatment with NBI-98854. The study will incorporate a capsule formulation of NBI-98854 and will be initiated in mid-2012, with top-line data anticipated by year-end.
http://phx.corporate-ir.net/phoenix.zhtml?c=68817&p=irol-newsArticle&highlight=&ID=1676713
That shouldn't be a huge black mark provided the fundamentals are sound. After all, if i remember correctly, BMR pumped MNTA pretty hard as well. Unfortunately biotech companies can't prevent being pumped by sources like BMR. It looks like the CEO was merely responding to questions emailed to him rather than sitting down for an interview.
That said i'm not a huge bull of POZN. It seems a bit overbought now so there may be a small pullback.
POZN - I see that biopete already posted on this. Anyone interested in hearing what Plachetka said on this topic can go to the 9.50 mark.
http://edge.media-server.com/m/p/ic9vvaqq/lan/en
POZN - Agree that the risk reward is good. BTW the 400M revenue for 540 quoted from the presentation is only for the US. And their P3 product is for OA that they think could garner peak sales > 500M WW.
http://wsw.com/webcast/roth26/pozn/
POZN - The EV is about 30 M. Management cited concern by investors in marketing the product as the reason for them looking for a partner for the PA32540. However JAZZ was a small company and it has been very successful with Xyrem and since POZN has cash perhaps they should try to go it alone. They may sell the product better than a larger company like AZN and they get the whole pie instead of a small piece. And its not like they have a blockbuster waiting in the wings to spend money on.
XNPT - The PHN indication is based on a single PII trial. That appears to satisfy the 505b2 pathway to some respect. The only question I have is about the dosage. The recommended dosage for Horizant is 600 mg daily. The PHN approval is for 600mg BID. Will this be an issue especially with the cancer issue they had earlier.
TRGT - AstraZeneca and Targacept Announce Remaining TC-5214 Phase 3 Efficacy Studies Do Not Meet Primary Endpoint, Regulatory Filing Will Not Be Pursued
LONDON & WINSTON-SALEM, N.C.--(BUSINESS WIRE)--Mar. 20, 2012-- AstraZeneca and Targacept, Inc. today announced top-line results from the remaining Phase 3 studies investigating efficacy, tolerability and safety of TC-5214 as an adjunct therapy to an antidepressant in patients with major depressive disorder (MDD) who did not respond adequately to initial antidepressant treatment. RENAISSANCE 4 and RENAISSANCE 5, both efficacy and tolerability studies, did not meet the primary endpoint of change on the Montgomery-Asberg Depression Rating Scale (MADRS) total score after eight weeks of adjunct treatment with TC-5214 as compared to placebo.
http://phoenix.corporate-ir.net/phoenix.zhtml?c=178332&p=irol-newsArticle&ID=1674213&highlight=
CRME - Here is a link to the FDA's warning about multaq.
http://www.fda.gov/Drugs/DrugSafety/ucm264059.htm
I wonder if any of the larger pharma's with a new oral anticoagulant would be interested. From what i've read they would be somewhat complementary with Vernakalent so perhaps Pfizer or Bayer may be interested. Amioderone has its own safety issues so there is probably a market for a safer product. But it will be expensive to prove that Vernakalant has a good risk/benefit profile.
OT - Amazon.com to buy Kiva Systems for $775 million
(Reuters) - Amazon.com Inc said on Monday it agreed to buy Kiva Systems Inc for $775 million in cash, a deal that will bring more robotic technology to the e-commerce company's giant network of warehouses.
I bought some May calls today but chances are the trials would be negative. They do have some asthma and diabetes pII trials reporting in the 1st half as well so may calls may not go entirely worthless if the depression trials fail.
Merck is meeting with FDA later this year so the trial may not restart until that meeting.
TRGT - The fixed dose trials appear to have completed on clintrials so results should be out within the next few days/weeks.
CRME - Here's an older article about the potential of oral vernakalent. Obviously since then multaq has thrown a spanner in the works . I'm not sure what the current terms are but if Merck's not interested perhaps CRME has the right to partner with someone else albeit not for such great terms. They don't need cash immediately and another potential catalyst could be the restart of the IV program here in the US.
Kynapid
Cardiome's Kynapid (also known as vernakalant hydrochloride) could be the first drug on our list approved by the FDA.
In fact, if the intravenous medication receives FDA approval by Jan. 19, it could be the first new drug for atrial fibrillation since 2000.
Atrial fibrillation strikes 2.5 million Americans, and is caused when the heart's upper chambers beat too quickly.
The heart irregularity causes blood clots, chest pains and palpitations, and is considered a leading cause of stroke.
Blood thinners prevent that. Drugs called beta blockers can slow the heartbeat. And surgery is sometimes an option.
An oral version is being studied. Meanwhile, annual sales could hit $1.4 billion by 2015.
"The oral drug is a clear blockbuster," says Mike King, an analyst with Rodman & Renshaw, an investment bank.
ECYT - I think it's still undervalued. Perhaps some are taking profits. The EV is about 60M when debt is taken into account. I suppose when you compare to similar companies like THLD, SNTA or INFI it's cheap. It's a wholly owned program and they have a pipeline of linkers and payloads. So they have the opportunity to partner and I hope they do that. Also the assumption is that there is a lack of catalysts until late 2013 but they are presenting at AACR in a few weeks and probably the most interesting presentation could be the late breaker.
http://www.abstractsonline.com/Plan/ViewAbstract.aspx?sKey=783edb67-9fbe-4b38-81c5-4b7f74b27171&cKey=8d83b352-380b-4cd5-880f-5ece93aa8c02&mKey=%7b2D8C569E-B72C-4E7D-AB3B-070BEC7EB280%7d
ECYT - The point about bias is well taken. In ECYT's case when an IRC did its own analyses, while the FR++ PFS data remained statistically significant, the overall PFS data did not indicating possible bias. I think confirmation by the IRC for the FR++ population augurs well for the PIII trial.
Thanks Zipjet. At the 49 minute mark Dan Welch mentioned that the rating of 1,2 or 3 is used to figure out a premium to a comparator. However the GBA determined that there is no comparator so ITMN can likely go for the price they want. Also if one uses tracleer worldwide pricing to get an idea of what the prices would be in the other European nations, surprisingly, the price in Italy could be higher. Tracleer pricing is about 30% higher in Italy, closer to the US price (~68K), than to the price in Germany. In the other major markets however tracleer pricing is significantly less than German pricing.
ITMN - I listened to the call and it seemed like ITMN would be able to avoid any type of price reduction? Would you agree? The muted share price reaction appears to be due to the Jefferies report citing the lower than expected number of eligible IPF patients in the GBA report.
ITMN - pirfenidone
Order of 15 March 2012
Entered into force on:
Federal Gazette [] No [..], dd.mm.yyyy, p [..]
Approved Application:
Esbriet is used in adults for the treatment of mild to
moderate idiopathic pulmonary fibrosis (IPF).
First Extent of the additional benefits of the drug
Pirfenidone has been approved as drugs for treatment of a rare
Suffering under Regulation (EC) No 141/2000 of the European Parliament
and of the Council of 16 December 1999 on orphan medicinal products.
According to § 35a paragraph 1, sentence 10 of the additional medical benefits is through the
Admission as evidence.
The Federal Joint Committee (G-BA) determined according to 5 Section § 12
Paragraph 1 sentence 1 No. 2 of the Rules of the G-BA (Rules of Procedure) the extent
value-added to the number of patients and patient groups, for
a therapeutically significant additional benefit. this quantification
the value added is the measure of in 5th Section § 5, paragraph 7, Nos. 1 to 4
Rules of Procedure established criteria.
Extent of the additional benefits:
not quantifiable
http://www.g-ba.de/downloads/39-261-1456/2012-03-15_AM-RL-XII_Pirfenidon.pdf
ITMN -
German pharma criticizes new AMNOG vetting procedure
Article | 13 March 2012 Print This ShareThis
Until recently, pharmaceutical companies could set prices for new drugs in Germany. But now their “monopoly is broken,” says the journal Pharmazeutische Zeitung. The government Act on the Reform of the Market for Medicinal Products (AMNOG) has erected a series of hurdles for drugmakers hoping to bolster revenues with new drugs and thus made the German regulatory environment a great deal more arduous and uncertain for the industry.
Under AMNOG, which came into effect last year, pharma firms introducing a new drug must submit a dossier showing that it constitutes an improvement on existing treatments. A Federal Joint Committee (GBA) delivers a verdict after the Institute for Quality and Efficiency in Healthcare (IQWiG) has evaluated the drug. This takes up to three months.
Drugs are graded on their added therapeutic benefit using a six-stage scale ranging from “considerable added therapeutic benefit” to “no added benefit.” If the drug adds no value, it is assigned a price under Germany’s reference-pricing system. If it is deemed an improvement, the pharma company and state health insurers negotiate the price and the rebate for the health system. An arbitration board can step in if the two parties cannot agree. The whole process is supposed to be completed within 15 months.
Mixed results so far
Over 20 drugs are undergoing vetting under AMNOG and while several have been deemed to add some value, the industry has criticized the IQWiG’s methodology and assumptions in several cases where it feels the assessment is unfair. It noted, for instance, that the IQWiG decided that the orphan drug pirfenidone (Esbriet, from US biotech firm InterMune) provided no additional benefit (The Pharma Letter December 18, 2011). Yet the industry points out that orphan drugs are supposed to be exempt from the vetting procedure as their added value is legally endorsed by their successful completion of the European approval process.
Another example is the breast cancer drug eribulin (Eisai’s Halaven; TPL February 17)). This is supposed to be administered when initial therapy no longer works, but the drug it was compared to is used in the first phase of the illness, which is, unsurprisingly, cheaper. This is a case of comparing apples and pears, according to the director-general of the German pharma association VFA. AstraZeneca’s Brilique (ticagrelor, also marketed as Brilinta), fared rather better, becoming the first drug to be cleared under the AMNOG procedure (TPL December 16, 2011). It received a favorable opinion in one indication, allowing AstraZeneca to negotiate a price with the country’s public healthcare organization rather than be subjected to reference pricing.
Price referencing key area of conflict
Recurrent procedural disputes are, however, being overshadowed by the question of establishing a framework for negotiating prices for drugs that add value. A study conducted for the VFA by health economist Juergen Wasem of the University of Duisburg-Essen notes that Germany has a huge influence on international drug reference prices. Germany directly or indirectly influences drug prices in 31 countries, his study shows. So the fear is that lower prices in Germany are likely to undermine revenues across Europe.
So when it comes to establishing a framework for determining drug pricing under AMNOG, Pharma is clearly hoping that the arbitration board, which is soon to finalize the criteria for assessing European prices in order to create reference prices for AMNOG drugs, leans towards countries where prices would tend to be comparatively high.
But this issue does not seem to be going Pharma’s way either. The arbitration board is putting too much emphasis on population size, notes a joint statement by various German industry associations, and not enough on whether the countries whose prices are set to be included in the benchmark are economically comparable to Germany: they include Greece, Portugal, Slovakia and the Czech Republic.
The full extent to which AMNOG is set to clip German pharma’s wings will only begin to emerge over the next few months. But with bureaucratic hurdles mounting and prices under constant pressure, the industry, as Pharmazeutische Zeitung puts it, is being squeezed into “an unusually tight corset.”
http://www.thepharmaletter.com/file/111771/german-pharma-criticizes-new-amnog-vetting-procedure.html