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YMI - PGS, Any thoughts on how YMI's JAK inhibitor CYT387 compares with INCY's 18424? The safety profile appears to be better for CYT387.
Anemia Response from Phase I/II Trial of JAK1/JaK2 Inhibitor CYT387
ORLANDO, FL, Dec 06, 2010 /PRNewswire via COMTEX/ --
Results presented at the 52nd American Society of Hematology Annual Meeting
YM BioSciences Inc. (NYSE Amex: YMI; TSX: YM) today announced positive interim data from the first 60 patients enrolled in the Phase I/II trial for its JAK1/JAK2 inhibitor, CYT387, in myelofibrosis. The results were reported today in an oral presentation at the 52nd American Society of Hematology (ASH) Annual Meeting being held in Orlando, Florida.
"Anemia is the most serious symptom associated with myelofibrosis, so I am highly encouraged by the emerging activity profile of CYT387, which uniquely continues to demonstrate a substantial ability to improve anemia while producing similar results to its peers in reducing spleen size and controlling constitutional symptoms," noted Dr. Alayew Tefferi, Chair of the Study. "I also encourage YM to evaluate CYT387 in other diseases where anemia has a significant impact."
"These unprecedented results highlight the potential safety and efficacy advantages of CYT387 compared with other JAK inhibitors. If CYT387 continues to demonstrate this unique anemia benefit in larger trials, it may ultimately serve as a drug of choice for the majority of patients with myelofibrosis who find anemia problematic," said Dr. Nick Glover, President and CEO of YM BioSciences. "We look forward to updated data from the full 140 patient trial in calendar mid-2011."
Patient Characteristics :
The presentation described interim results for the first 60 patients enrolled in the dose escalation (n=21) and dose confirmation (n=39) portions of the 140 patient Phase I/II trial, for which recruitment is ongoing. In the dose-confirmation phase, subjects were started at one of two dose levels that were deemed clinically effective: 150 mg/day (n=18) and 300 mg/day (n=21). Thirty-three subjects (55%) were red cell transfusion-dependent at study entry. Forty-five subjects (75%) expressed the JAK2V617F mutation. Forty-eight (80%) had palpable splenomegaly greater than 10 cm. A number of the patients had discontinued treatment with other JAK inhibitors (INCB018424, n=11; TG101348, n=3) or pomalidomide (n=13). The median time to follow up since study start is currently 4.9 months, ranging from 0.9-12.5 months.
Efficacy Results
: - The Overall Response Rate (anemia, spleen) to date, as per the
International Working Group for Myeloproliferative Neoplasms Research
and Treatment (IWG-MRT) criteria, is 62%.
- Anemia Response: Of 42 subjects who were evaluable for anemia
response (baseline Hgb less than 10 g/dL or red cell transfusion-
dependent), 21 subjects (50%) had achieved Clinical Improvement (CI)
as per the IWG-MRT criteria. A 57% response rate was observed in
transfusion dependent patients. For the 150 mg/day dosing group, the
anemia response rate was 41% (43% for transfusion dependent
patients). For the 300 mg/day dosing group, the anemia response rate
was 58% (69% for transfusion dependent patients). Responses were
observed in five of nine (55%) patients who had discontinued
treatment with other JAK inhibitors (INCB018424, TG101348) and 5 of
12 (42%) who had discontinued pomalidomide.
- Spleen Size Reduction: Twenty-five (47%) of the 53 evaluable subjects
who had splenomegaly at baseline achieved a minimum 50% decrease in
palpable spleen size, thus qualifying them for CI per IWG-MRT
criteria. For the 150 mg/day dosing group, 53% qualified for CI. For
the 300 mg/day dosing group, 46% qualified for CI.
- Constitutional Symptoms: CYT387 controlled constitutional symptoms in
a significant percentage of patients (night sweats: 88%, bone pain
: 80%, pruritus: 92%, fever: 100%).
Safety Results :
In the dose escalation portion of the study, at the highest dose level (400 mg/day), two of six subjects experienced dose limiting toxicity (DLT) (one each of reversible asymptomatic Grade 3 hyperlipasemia and Grade 3 headache); consequently, the maximum tolerated dose (MTD) was declared at 300 mg/day.
CYT387 was well tolerated. The overall discontinuation rate was 5% and there were no patient withdrawals for drug-related adverse events. No Grade 4 non-hematological toxicities were observed. Grade 3 non-hematologic adverse events were infrequent and included increased transaminases (n=2), headache/head pressure (n=2), and increased lipase (n=3), No QTc prolongations greater than Grade 1 were observed.
Some patients experienced a "first-dose effect" characterized by Grade 1 lightheadedness (26 subjects, 43%) and hypotension (31 subjects, 52%). The phenomenon was self-limiting, generally resolved within 3-4 hours and did not recur after the first dose.
Grade 3/4 thrombocytopenia was seen in 16 (27%) subjects. It should be noted that the minimum platelet count specified in the inclusion criteria for the study is 50,000/mcl. The majority of Grade 3/4 thrombocytopenia were observed in subjects where baseline platelet counts were less than 100,000/mcl.
Treatment-emergent Grade 3 anemia was seen in four subjects (7%). None of the subjects developed new red cell transfusion requirements. Treatment-emergent Grade 3/4 neutropenia was seen in three subjects (5%).
For more information on the CYT387 Phase I/II trial, go to :
http://clinicaltrials.gov/ct2/show/NCT00935987?term=cyt387&rank=1
http://www.ymbiosciences.com/investors/press_releases_item.php?newsid=1504709
http://www.ymbiosciences.com/upload_files/CYT387_pres_ASH2010_Mayo.pdf
http://investor.incyte.com/phoenix.zhtml?c=69764&p=irol-newsArticle&ID=1471706&highlight=
Pharmacyclics Reports CLL Results from Preclinical and Clinical Studies of its Btk Inhibitor PCI-32765
Company to Host Conference Call at 4:30 p.m. ET Wednesday, December 8, 2010
Press Release Source: Pharmacyclics On Sunday December 5, 2010, 5:00 pm EST
http://files.shareholder.com/downloads/PCYC/1082503877x0x426027/10B827F5-5257-4875-8957-EA4A5CD9612A/ASH_Pharmacyclics_Investor_Presentation_12_5_10.pdf
ORLANDO, Fla. and SUNNYVALE, Calif., Dec. 5, 2010 /PRNewswire-FirstCall/ -- Pharmacyclics, Inc. (Nasdaq:PCYC - News) today announced data from three Chronic Lymphocytic Leukemia (CLL) presentations at the American Society of Hematology (ASH) Annual Meeting describing the Btk-selective inhibitor PCI-32765. Two presentations report preclinical data, and one presentation reports clinical data from a pooled analysis of 54 patients with CLL or Small Lymphocytic Lymphoma (SLL) treated with PCI-32765.
The two preclinical abstracts focus on the mechanism of action of this novel drug. An oral presentation titled "Bruton's Tyrosine Kinase Inhibitor PCI-32765 Abrogates BCR and Nurselike Cell-Derived Activation of CLL Cells In Vitro and In Vivo" is being presented by Sabine Ponader, PhD from the MD Anderson Cancer Center. The other preclinical presentation is a poster presented by Sarah Herman, PhD of the laboratory of John Byrd, MD, of The Ohio State University titled "The Kinase Inhibitor, PCI-32765, Demonstrates Activity in Chronic Lymphocytic Leukemia Cells Independent of Microenvironmental Survival Signals." Data from these two presentations together suggest a model whereby PCI-32765 directly affects CLL cells by inducing apoptosis, and also blocks the ability of CLL cells to migrate to and to adhere to lymph nodes, a protective environment where the tumors grow. The clinical results of treatment, as discussed below, appear to bear out these combined mechanisms, with evidence of impairment of both homing to lymph nodes and cell viability in the typical clinical response to PCI-32765.
Data Results from the Pooled Phase IA and IB Studies in CLL/SLL
The oral presentation, titled "The Bruton's Tyrosine Kinase Inhibitor PCI-32765 is Well Tolerated and Demonstrates Promising Clinical Activity In Chronic Lymphocytic Leukemia (CLL) and Small Lymphocytic Lymphoma (SLL): An Update on Ongoing Phase 1 Studies" is being presented by Jan Burger MD, PhD. This abstract describes clinical data from a pooled analysis of 54 CLL/SLL patients from a Phase Ia (N=16) and a Phase Ib/II (N=38) study. Patients from the Phase Ia trial have relapsed/refractory CLL/SLL and have been treated for a median of approximately 8 months, while patients on the Phase Ib/II trial include two cohorts—one with relapsed or refractory disease and one with treatment-naive disease—who were just recently enrolled.
Response data from the pooled analysis is presented below. Outcomes are described based on strict application of standard response criteria, with an additional outcome category termed "nodal response with lymphocytosis." This category describes patients with substantial lymph node (LN) shrinkage who have not yet achieved a 50% decrease of malignant B cells in the blood (lymphocytosis). The inclusion of this category is necessary because of the unique characteristics of response to this agent, especially early in treatment. From the Ia trial, with approximately 8 months median follow-up, 1 patient achieved a complete response (CR) and 8/13 evaluable patients achieved a partial response (PR) for an objective response rate of 69%. Two additional patients achieved a nodal response with lymphocytosis (15%) and 2 patients had stable disease (SD). Of note, 3 of the patients now classified as partial responses were initially nodal response with lymphocytosis earlier in treatment. In the Ib/II trial, with less than 2 months median follow-up, 8/32 evaluable patients achieved a PR (25%) by standard response criteria with an additional 17/32 (53%) achieving a nodal response with lymphocytosis. Six other patients have SD. As noted previously, study of the patients from the Ia trial suggest that blood lymphocytosis decreases over time on treatment and patients who initially are described as nodal responses with lymphocytosis may achieve a PR by standard response criteria with continued therapy.
(Photo: http://photos.prnewswire.com/prnh/20101205/SF12271)
Based on the pooled analysis from the Phase Ia study (~8 months median follow-up) and the Ib/II study (<2 months median follow-up) in this group of CLL/SLL patients, of the 45 evaluable patients, one patient achieved a CR (2%), 16 (36%) achieved a PR and an additional 19 (42%) have experienced a dramatic reduction in lymph node disease with lymphocytosis. When evaluating nodal disease alone, 87% percent (34 out of 39) of patients achieved a 50% or greater reduction in lymph node burden. In patients with symptomatic disease, this early response was often associated with improvement in symptoms.
PCI-32765 continues to be well tolerated in this patient population. Grade 3/4 events were uncommon. More common grade1/2 events were diarrhea and other GI and constitutional symptoms. No hepatic or renal laboratory adverse events were reported on this trial and significant hematologic events were infrequent. There was no evidence of cumulative toxicity.
ASH Conference Call and Webcast Details:
Date: Wednesday, December 8, 2010
Time: 4:30 pm ET
Slides used in Presentation: http://ir.pharmacyclics.com/events.cfm
Listen via Internet: http://ir.pharmacyclics.com/events.cfm
Live Participant Dial In (Toll Free): 877-407-8133
Live Participant Dial In (International): 201-689-8040
To access the live audio broadcast or the subsequent archived recording, log on to http://ir.pharmacyclics.com/events.cfm. The archived version of the webcast will be available for 30 days on the Investor Relations section of the company's Web site at www.pharmacyclics.com. To see a slide presentation to be discussed during the call, please go to the Investor Relations Section of our website, under Events & Webcasts: http://ir.pharmacyclics.com/events.cfm and click on "ASH 2010 Conference Call Slides".
About this trial
The Phase Ia is a multicenter study being conducted in collaboration with investigators at leading lymphoma centers including Stanford University, MD Anderson Cancer Center, the University of Chicago, the University of Vermont, Weill Medical College of Cornell University, and the US Oncology Group. The trial is an open-label, dose-escalation study of PCI-32765 in recurrent B cell malignancies treating a minimum of 6 patients per cohort. Fifty six patients were enrolled between March 2009 and September 2010.
In the Phase Ia five dose levels are being explored—1.25, 2.5, 5.0, 8.3 and 12.5 mg/kg/day. Each cycle of treatment consists of 28 consecutive days of once daily dosing followed by a 7-day rest period. Additionally two dose groups at 8.3 mg/kg/day and 560mg have also been explored using a 35-day cycle with no rest period ("continuous dosing" or "CD1 and CD2"). Dose limiting toxicities were evaluated at the end of the first cycle and drug efficacy is evaluated every 2 cycles. Safety is being monitored throughout the trial.
The Phase Ib/II is a multicenter study being conducted in collaboration with investigators at leading lymphoma and leukemia centers including the University of Texas, MD Anderson Cancer Center, Stanford University, Sarah Cannon Research Institute, the University of Vermont, Weill Medical College of Cornell University, the Ohio State University and the US Oncology Group. This open-label, fixed dose study was designed to evaluate safety and preliminary efficacy in patients with CLL/SLL who received a continuous dose of PCI-32765. The study is evaluating two patient populations, relapsed refractory and elderly patients (naive to therapy). The first cohort of relapsed/refractory patients have been dosed at 420 mg (27 patients, fully enrolled) and a second cohort is being dosed at 840mg (20 patients enrolled out of 24 total planned). The elderly patients, naive to therapy, are being dosed at 420 mg daily and 16 patients out of a planned 24 have been enrolled. The enrollment numbers for the Ib/II study were as of November 30, 2010 and the study continues to enroll rapidly.
About Bruton's Tyrosine Kinase Inhibitor PCI- 32765
PCI-32765 is an orally active small molecule inhibitor of Bruton's tyrosine kinase (Btk) that is being developed by Pharmacyclics for the treatment of patients with B-cell lymphoma or leukemia. Btk plays a prominent role in B-cell lymphocyte maturation by mediating B-cell receptor (BCR) signal transduction. Recent studies indicate that some B-cell lymphomas have kinases that are activated downstream of the BCR and that suppression of this signaling by a Btk inhibitor can induce apoptosis in these cells. BCR signaling is also thought to promote malignant cell expansion and survival in chronic lymphocytic leukemia (CLL).
About Chronic Lymphocytic Leukemia
CLL is the most common type of leukemia, a cancer of the white blood cells. CLL is a B cell cancer that originates in the bone marrow. Approximately 18,000 patients in the US are diagnosed each year with CLL. The prevalence of CLL is approximately 86,000 in the US. The disease is a chronic disease of the elderly with an average survival of about 5 years. Patients commonly receive multiple lines of treatment over the course of their disease. CLL and Small lymphocytic lymphoma (SLL) are considered the same underlying disease. SLL is a disease that is limited to the lymph nodes.
http://finance.yahoo.com/news/Pharmacyclics-Reports-CLL-prnews-1747660639.html?x=0&.v=1
ARRY - My understanding is that the response rate normally only includes complete + partial responses so in this case it would be 2/30. IMO even if ARRY-520 is a single agent the response rate should be comparable to combinational treatments.
ARRY is conducting a single agent PII trial so they must see something positive.
ARRY - Here's a link to the actual poster. The # of responses only differs by an additional PR from ASCO 2010 and seems a bit underwhelming but this is a single agent trial. I couldn't find any other single agent trials in MM to compare with.
http://www.arraybiopharma.com/_documents/Publication/PubAttachment428.pdf
MNTA - Regarding whether MNTA should have an idea of whether Teva did infringe, I would say that the fact that the lawsuit is limited to just two patents provides an indication that MNTA does know to some degree.
6,485 Overseas Clinical Trials and Counting
December 2, 2010, 1:49 PM
By DUFF WILSON
http://prescriptions.blogs.nytimes.com/2010/12/02/6485-overseas-clinical-trials-and-counting/?src=twt&twt=nytimeshealth
http://www.vanityfair.com/politics/features/2011/01/deadly-medicine-201101
The venerable investigative reporters Donald L. Barlett and James B. Steele turn their focus to overseas clinical trials in an article in this month’s Vanity Fair on newsstands today and online here.
With their trademark exhaustive research – did you know there were 6,485 clinical trials overseas in 2008 on drugs intended for American use, 23 times more than the 271 in 1990? – the reporters note, several times, a gap in the system.
Nobody keeps track of all those trials.
“Data is made available to the public on a purely voluntary basis,” they wrote. “Its accuracy is unknown. The oversight that does exist often is shot through with the kinds of ethical conflicts that Wall Street would admire.”
Barlett and Steele found the F.D.A. does occasional inspections, visiting 0.7 percent of trial sites outside the country in 2008, compared with 1.9 percent inside the United States.
But they seem to distrust some of the research done by overseas contractors in places with much less regulation and more willing, low-income subjects, and cite case after case. The article is titled “Deadly Medicine,” so you get the point.
Barlett and Steele wrote:
“It used to be that clinical trials were done mostly by academic researchers in universities and teaching hospitals, a system that, however imperfect, generally entailed certain minimum standards. The free market has changed all that. Today it is mainly independent contractors who recruit potential patients both in the U.S. and—increasingly—overseas. They devise the rules for the clinical trials, conduct the trials themselves, prepare reports on the results, ghostwrite technical articles for medical journals, and create promotional campaigns. The people doing the work on the front lines are not independent scientists. They are wage-earning technicians who are paid to gather a certain number of human beings; sometimes sequester and feed them; administer certain chemical inputs; and collect samples of urine and blood at regular intervals. The work looks like agribusiness, not research.”
The reporting partners, who live in Philadelphia, are contributing editors to Vanity Fair magazine. They are known for thorough research leading to sweeping conclusions about important subjects, and this article fits that billing. The pair have won two Pulitzer Prizes, two National Magazine Awards, and written seven books including the 1992 best-seller “America: What Went Wrong?”
Looks like AF has changed his mind about pereira since he wrote the following:
I'm the first one to say not to trust everything said by the CEO of a drug or biotech company. There are countless examples of investors led down the wrong path by management exaggerations or misstatements.
But in this case, I find it hard not to side with Pereira in the ferumoxytol argument. It's not that Berens doesn't present some good arguments. He does, and handicapping an FDA decision these days, especially one that seems to hinge on safety, is fraught with risk.
But when you speak with Pereira, or witness him answer questions about ferumoxytol in an investor meeting, he comes across as highly credible and assured. Moreover, this is not a guy learning the ins and outs of kidney disease and anemia on the job. Pereira is a highly trained physician and noted expert on kidney disease. He's a past president of the National Kidney Foundation and, literally, helped put together one of the seminal textbooks in the field.
So, in the Berens vs. Pereira fight, the edge, at least in my book, goes to Pereira.
The servier deal is only for the hdac inhibitor and only covers ex US. If PCYC mgmt is astute enough to get a similar deal for pci-765 then an EV of ~270 M isn't too expensive right now.
There's an ASH presentation comparing enox with cutenox.
http://ash.confex.com/ash/2010/webprogram/Paper31905.html
1086 An Open Label, Non Randomized, Propspective Phase IV Clinical Trial Evaluating the Immunogenicity of Branded Enoxaparin Versus Biosimilars In Healthy Volunteers
Oral and Poster Abstracts
Poster Session: Antithrombotic Therapy: Poster I
Saturday, December 4, 2010, 5:30 PM-7:30 PM
Hall A3/A4 (Orange County Convention Center)
Poster Board I-66
Marise Gomes, Ph.D.1*, Eduardo Ramacciotti, M.D., Ph.D.1*, Debra Hoppensteadt, Ph.D.2*, Jeanine M. Walenga, PhD3, Bruce E Lewis, MD4*, Walter Jeske, Ph.D.5* and Jawed Fareed, PhD2
1University of Michigan, Ann Arbor, MI
2Pathology and Pharmacology, Loyola University Chicago, Maywood, IL
3Cardiovascular Institute, Loyola Univ. Medical Center, Maywood, IL
4Cardiology, Loyola University Chicago, Maywood, IL
5Thoracic and Cardiovascular Surgery, Loyola University Medical Center, Maywood, IL
Biosimilar enoxaparin preparations are in use outside the U.S. Due to compositional variations, their interaction with platelet factor 4 (PF4) differs leading to differential immunogenic responses between branded and biosimilar agents. To compare their immunogenic response, branded enoxaparin (Clexane®, Sanofi-Aventis) and a biosimilar version (Cutenox®, Gland-Pharma) were administered to healthy volunteers (n=110/drug) at a dose of 40 mg SQ for10 days. Blood samples drawn on days 1 and 10 were analyzed for anti-heparin/PF4 antibody (A-HPF4-Ab) titers and subtypes by ELISA (GTI, Brookfield, WI). Treatment with each LMWH resulted in comparable A-HPF4-Ab generation as compared by using the total absorbance for each population (p<0.05). However in the thrombin generation assays clexane group showed a stronger inhibition (45+12% vs 32+9%). None of these antibodies activated platelets as determined by the serotonin release assay. The two groups alos showed comparable AXa and AIIa responses (p<0.05). Antibody subtyping demonstrated different profiles between LMWHs. For IgG (Clexane1=0.15±0.04, Clexane10=0.21±0.06, Cutenox1=0.17±0.04, Cutenox10=0.28±0.10) with a significant time effect (p<0.0001), a significant drug effect (p<0.0001), and a significant time by drug interaction (p=.0009). Post hoc comparisons showed a difference between the drugs at time 0 (p=0.03), a difference between the drugs at time 10 (p<0.0001) and a significant time effect for each drug (p<0.0001).For IgA (Clexane1=0.12±0.02, Clexane10=0.15±0.02, Cutenox1=0.12±0.03, Cutenox10=0.13±0.02) with significant effects for time (p<0.0001), drug (p=0.0078) and for the drug x time interaction (p<0.0001). The post hoc comparisons showed a significant drug effect at time 10 (p<0.0001). There was a significant time effect for Clexane (P<0.0001) but not for Cutenox. For IgM (Clexane1=0.11±0.01, Clexane10=0.13±0.02, Cutenox1=0.11±0.03, Cutenox10=0.13±0.02), there was only a time effect (p<0.0001). The post hoc comparisons showed no difference between drugs at either time, but significant time effects for each drug (p<0.0001). The immunogenic potential of LMWHs varies in terms of ability to generate A-HPF4-Ab, the antibody subtypes generated, and their cross-reactivity with pre-formed A-HPF4-Ab. Such parameters may be useful in defining the bioequivalence of generic LMWHs. Future studies evaluating the immunogenicity of different compounds in patients exposed to biosimilar drugs are warranted.
Disclosures: Jeske: PolyMedix, Inc.: Research Funding.
The biggest reason I'm considering buying is the fact that the CEO made a hefty purchase a couple of months ago at 6.51. As for its pipeline, it has a couple of candidates in PII stage. And unless I've missed something PCYC only owes CRA mid to high single digit royalties so I fail to see how PCYC will see single digit royalties as well.
PCYC - Has gone down 10% since monday on larger than average volume and I'm debating whether its worth taking a bite. The slide could be due to the ASH presentations. However the abstracts have been up for a while so its interesting why the volume increased on monday.
Two of the 4 presentations are linked below.
http://ash.confex.com/ash/2010/webprogram/Paper34320.html
http://ash.confex.com/ash/2010/webprogram/Paper34300.html
The june ASCO presentation is linked below and a comparison shows that 5 patients withdrew due to adverse events and the OR dropped from 49% to 43 %.
http://ir.pharmacyclics.com/releasedetail.cfm?ReleaseID=476851
Is there a chance Teva could prove through a different method that it is a authorized generic to Lovenox? .
IMMU/UCB uses a different metric for the PE from what HGSI/GSK used. I have to double check but I also think that the patient populations in the IMMU PII trial had patients with more severe SLE that HGSI omitted from their PIII trial. That turned out to be an issue according to the benlysta briefing docs since more patients with more severe SLE take other medication and the FDA is leery of allowing benlysta to be taken alongside those. As for valuation IMMU gets a 20% royalty and not a 50% sharing scheme like HGSI has. Plus there are other issues to think about such as how would the trial be designed if benlysta is approved.
UCB/Immunomedics’s lupus drug excites researchers
World News | November 12, 2010
http://www.pharmatimes.com/Article/10-11-12/UCB_Immunomedics_s_lupus_drug_excites_researchers.aspx
Results from a Phase IIb study involving epratuzumab, a new humanised IgG1 monoclonal antibody in development by UCB and Immunomedics for systemic lupus erythematosus, have impressed investigators.
Data from the 12-week EMBLEM study were presented at the American College of Rheumatology meeting in Atlanta by lead investigator Daniel Wallace of Cedars-Sinai Medical Center and UCLA School of Medicine, Los Angeles. “This was primarily a dose-ranging study comparing five doses and dose schedules between 200mg and 3600mg against placebo,” he told rheumatologists who crowded into an oral session to hear the data. But the study also suggested promising information on safety and efficacy from the 227 patients with active moderate and severe SLE who took part, he added.
All cumulative doses were superior to placebo, he said. “However, patients who received a cumulative dose of 2400mg either by 600mg weekly or 1200mg every other week saw a clear, clinically meaningful and statistically significant improvement in their condition by 12 weeks compared to placebo.” Response rates observed for epratuzumab were more than double those for placebo. Improvement was measured by BILAG-2004 (British Isles Lupus Assessment Group) criteria and by SLEDAI (SLE Disease Activity Index) scores.
By week 12, significantly more patients receiving epratuzumab 600mg weekly had improved their baseline BILAG A/B scores to BILAG D in all six body systems (indicating no active disease) than patients receiving placebo.
Epratuzumab, which targets CD22, also reduces B cells but only by a modest amount, said Dr Wallace. Benefit started to be apparent quickly, within four to eight weeks. This is faster-acting than drugs with a larger B-cell depleting effect, he commented.
“The degree of improvement was substantial. These were sick patients and I’m very excited about the results we saw from epratuzumab.” Most patients had high disease activity at baseline, he noted., and over 70% had more than one BILAG A score across multiple organ systems.
Adverse events including infections and injection site reactions, and serious adverse events were similar in both the treatment and placebo arms of the study. “The drug was very well tolerated and there were no new safety signals,” he added
Co-investigator Ken Kalunion of the UCSD School of Medicine, La Jolla, said: “These are small numbers but the results are very encouraging. It’s hard to say from a Phase II trial but this drug could prove to be a breakthrough in SLE”. He added: “The most dramatic improvements seen were in the cardiorespiratory and neuropsychiatric organ systems.” In the former, all patients receiving the 600mg dose improved to BILAG D by 12 weeks. And in the neuropsychiatric system, 5 out of 6 patients in the 600mg weekly group went from BILAG B to D.
Another co-investigator, Vibeke Strand of Stanford University, Palo Alto, commented: “It is terrific to see improvement within three months that is clinically meaningful. I am very excited about epratuzumab. I think it’s a great drug that may have potential to be an induction therapy. I’m eager to see where it goes in Phase III.” Trials are expected to begin early 2011, Dr Wallace said.
SLE affects about 50 per 100,000 people in the USA and the condition is currently regarded as having an unmet clinical need. Although there are several symptomatic treatments there are none that tackle the underlying pathology of SLE.
Piper Jaffray: Human Genome Sciences' Benlysta Phase III Data Supports Approval; But Not Without Controversy
Presentations of the integrated data from the Human Genome Sciences (NASDAQ: HGSI) Phase III and Phase II trials highlighted durability of response and organ domain benefits of Benlysta. However, pooled safety data could be a point of contention at the FDA panel as higher rates of opportunistic infections, neoplasms, and mortality were seen, albeit in small numbers.
Piper Jaffray continues to expect an FDA advisory panel recommendation for approval to provide 10%+ upside to shares, but the stock is likely to trade lower before the panel. The additional risk is potential lack of lupus experts on the panel, which could fail to appreciate Benlysta's efficacy on top of best available treatment.
Ultimately, Piper expects data at ACR to lead to investors questioning Benlysta's market opportunity. We however remain comfortable with the launch, with our 2011 $178mn revenue estimate based on 7% penetration of moderate-to-severe patients; well below 20-25% cited by physicians at the ACR.
http://www.benzinga.com/analyst-ratings/analyst-color/10/11/598002/piper-jaffray-human-genome-sciences-benlysta-phase-iii-da
FDA chief urges caution on budget cut
By Lisa Richwine
NEW YORK | Tue Nov 9, 2010 10:39pm GMT
(Reuters) - Lawmakers should think carefully before cutting taxpayer funding for the Food and Drug Administration in an effort to reduce government spending, the agency's chief said on Tuesday.
http://uk.reuters.com/article/idUKTRE6A84QI20101109
Republicans, who will take over the U.S. House of Representatives in January, campaigned on promises to cut funding for federal government operations, a plan that could put the FDA in the cross-hairs with other government agencies.
"Not every function of government can be cut to the same degrees using the same tools. I think we should proceed with real care," FDA Commissioner Margaret Hamburg said at the Reuters Health Summit in New York.
"It should be recognized if we can't do our job and do it well there isn't any other entity that will backstop behind us," she said.
"What we do really matters to health," Hamburg added.
The FDA oversees about 25 percent of the U.S. economy including prescription and over-the-counter drugs, medical devices such as implanted pacemakers and artificial knees, most foods, and many other consumer products. Last year, Congress also ordered the FDA to regulate tobacco.
Industry and consumer groups agree the FDA is stretched thin with its current budget of more than $3 billion despite steady increases in recent years. Advocates have lobbied Congress for the past several years to add money.
Democrats retain control of the Senate and the White House but will need to work with House Republicans who won a majority in last week's midterm elections when setting agency funding.
Hamburg said the FDA's "responsibilities outstrip our resources" and one of the growing challenges is the rapid globalization of the food and drug supply.
"It's one area where we need to strengthen despite economic pressures," she said.
Hamburg also said the agency was looking for ways to be more efficient and had taken steps to better inform the public about how the agency spends its money. (Link to Reuters Insider video: link.reuters.com/ban44q)
Part of the agency's budget comes from industry fees levied on drug and device makers that submit applications for product approval. The current fees expire in 2012, and the FDA has begun negotiating with companies on an extension.
"Industry recognizes that it will have to continue to invest to broaden and strengthen some of these programs." Hamburg said.
The former New York City health commissioner took over the FDA in May 2009 and said she has no plans to leave her current post.
"It's a difficult job, no doubt about it. Every day I'm grappling with important issues," Hamburg said.
But she said "you really do feel that what you are doing makes a difference and that is very rewarding. I have no plans to go."
(Reporting by Lisa Richwine and Susan Heavey, editing by Matthew Lewis and Carol Bishopric)
Bavarian in Talks to Sell Prostate Cancer Vaccine
By Frances Schwartzkopff - Nov 9, 2010 11:53 AM ET
http://www.bloomberg.com/news/2010-11-09/bavarian-is-in-advanced-talks-to-sell-prostate-cancer-vaccine.html?cmpid=yhoo
Bavarian Nordic A/S, the Danish drugmaker supplying the U.S. government with smallpox vaccines, gained the most in two weeks in Copenhagen trading after saying it’s in discussions to license its prostate cancer shot.
Several drugmakers, “including some of the largest,” are conducting due diligence on the product, which may extend men’s lives two times longer than Dendreon Corp.’s and Johnson & Johnson’s medicines, Bavarian Nordic said today in a statement.
“The market’s opening its eyes and seeing that Bavarian Nordic could enter into a partnership in the near future,” said Frank Hoerning Andersen, an analyst at Jyske Bank.
The medicine, Prostvac, extended men’s lives by as much as 8.5 months compared with a placebo, early trials showed. Men lived about 4.1 months longer with Dendreon’s Provenge and 3.9 months longer in a study of J&J’s experimental treatment abiraterone. Bavarian may be able to demand royalty payments of as much as 25 percent of sales, Andersen said.
Bavarian gained 12.50 kroner, or 5.7 percent, to 233 kroner as of 5:05 p.m. It was the biggest advance since Oct. 25. The shares have risen 46 percent in the past 12 months compared with an 11 percent increase in the Bloomberg Europe Pharmaceutical Index.
Bavarian Nordic plans to begin the final stage of testing needed to gain regulatory approval next year. The drugmaker received fast-track designation in April from the U.S. Food and Drug Administration for the vaccine’s use in men whose cancer is worsening despite treatment.
Larry Biegelsen, an analyst at Wells Fargo Securities in New York, estimated that Seattle-based Dendreon’s Provenge costs about $93,000 for three doses administered over the course of a month. Abiraterone, which New Brunswick, New Jersey-based Johnson & Johnson acquired last year as part of its $878 million takeover of Cougar Biotechnology, may be priced at $27,000 annually, he said.
To contact the reporter responsible for this story: Frances Schwartzkopff at fschwartzko1@bloomberg.net
I think thats the right decision. Don't look a gift horse in the mouth. I'm surprised as anyone it got back to the mid 4's.
This is amazing and confusing at the same time. Why is bill marth publicly being confident while privately complaining about FDA delays? I know I know, shlomo told him to do it but still.
No h1n1 scare this year so fewer masks were purchased. However uncertainty due to obamacare also did have an effect though the impact seems to be less compared to the reduced h1n1 mask demand.
You may want to wait till after the CMS ruling sometime later this month. If that is any way positive, the stock may jump. On the other hand it may be a negative event as well but its something to look for IMO.
OT - That was hilarious. You can also read the text at the link below so that you don't miss a word.
http://www.npr.org/blogs/money/2010/11/03/131043062/federal-reserve
RIGL does have a JAK drug though I doubt it is currently being developed for arthritis. Maybe they have plans for that indication down the road. And if AZN is a syk inhibitor then its not a late stage jak drug.
Not defending the writer:)
Pfizer Pill Leads Race to Dominate $12 Billion Arthritis Market
By Tom Randall - Nov 5, 2010 9:45 AM ET
http://www.bloomberg.com/news/2010-11-05/pfizer-pill-leads-race-to-supplant-12-billion-arthritis-market.html
Pfizer Inc., the world’s biggest drugmaker, leads a race against three rivals to sell the first new pill in a decade for rheumatoid arthritis, a joint disease treated by injected drugs with $12 billion in annual sales.
The tablet, called tasocitinib, curbed inflammation and stopped the disease from worsening, Pfizer reported last week. Pfizer may edge out shots sold by Johnson & Johnson, Abbott Laboratories, and Amgen Inc. if research to be presented Nov. 10 at the American College of Rheumatology in Atlanta suggests its tablet is also a safe alternative.
Tasocitinib is the most advanced pill in a family of experimental drugs to target a protein, called JAK, which leads to joint destruction in 1.3 million Americans with rheumatoid arthritis. Pfizer’s study is the first late-stage JAK trial, one of six the company plans to complete by the end of next year. The pill can generate peak annual sales of $2 billion, said Tim Anderson, an analyst with Sanford C. Bernstein & Co.
“Tasocitinib’s main value proposition is that it is a first-in-class novel oral therapy launching into an area filled with injectable products that collectively sell about $12 billion a year for rheumatoid arthritis alone,” Anderson said in a Nov. 1 research report.
Pfizer shares fell 2 cents to $17.36 at 9:42 a.m. in New York Stock Exchange composite trading. The New York-based drugmaker rose 2.7 percent in the 12 months before today.
Lipitor Competition
Pfizer is counting on revenue from tasocitinib to help offset losses when its top-selling Lipitor cholesterol pill, with $11.4 billion in annual sales, faces competition next year from cheaper copies in the U.S. The company gained the arthritis remedy Enbrel in last year’s $68 billion purchase of Wyeth. The anti-inflammatory medicine generated $799 million in the third quarter, making up 5 percent of Pfizer’s revenue.
Rheumatoid arthritis is a chronic disease where the immune system mistakenly attacks healthy tissue, causing inflammation in and around joints. Shots known as anti-TNF therapy are standard care for the 80 percent of patients who don’t respond to treatment with methotrexate, a generic drug that can block cell growth in people with certain cancers and immune disorders.
Three biotechnology companies are trying to catch up to Pfizer with their own JAK drugs - Rigel Pharmaceuticals Inc., Incyte Corp., and Vertex Pharmaceuticals Inc. Pfizer’s pill is the only one in the third and final stage of tests generally needed for U.S. approval.
Two-Year Lead
Tasocitinib “appears to be in the lead by at least two years in comparison to other JAK inhibitors,” said Marc Goodman, an analyst with UBS Securities, in an Oct. 27 note to clients. “We believe that the safety issues are key to gaining the appropriate label and could limit its use to patients with low cardiovascular risk.”
Investors are most concerned about whether Pfizer’s drug will raise cholesterol levels enough to limit the number of patients who might use it, Goodman said. The pill showed signs of this side effect in previous study results.
Doctors may be slow to adopt new JAK drugs for patients who are already getting relief from older treatments, said David Pisetsky, professor of rheumatology and immunology at Duke University School of Medicine. While high cholesterol linked to tasocitinib may be treatable, physicians may resist adding an additional drug to patients’ daily treatment regimens.
“Because it’s a new mode of action, there’s great interest, but what’s going to happen in terms of efficacy and safety when you really put it out there?” Pisetsky said in a telephone interview. “There’s always a difference between what happens in a trial setting and what happens in real life.”
Enbrel, Remicade
The rheumatoid arthritis market is dominated by three anti- TNF treatments: Enbrel from Pfizer and Thousand Oaks, California-based Amgen; Humira, from Abbott Park, Illinois-based Abbott; and Remicade, sold by New Brunswick, New Jersey-based J&J and Merck & Co., based in Whitehouse Station, New Jersey. The treatments cost as much as $20,000 a year and leave patients susceptible to infections.
Summary results of the first three months of Pfizer’s six- month study were released last week. The study’s 611 patients took one of two doses of the drug or a placebo pill. After three months of treatment, 66 percent of patients taking the higher dose had at least a 20 percent improvement of symptoms, compared with 27 percent in the placebo group. Side effects were reported by 330 patients, with 25 seriously affected. Thirteen patients stopped taking the drug. A 79-year-old woman with a history of heart disease and diabetes died after developing diarrhea followed by renal failure.
Complete Data
The complete six-month data from the trial will be released Nov. 7 and presented Nov. 10 at the rheumatology meeting. The presentation will include the number of patients with 70 percent improvement and will describe side effects in greater detail.
Incyte Corp., based in Wilmington, Delaware, will present the second of three stages of testing of its drug, INCB-28050, at the rheumatology meeting. Early results of the 125-patient trial show similar effectiveness to Pfizer’s drug and comparable anti-TNFs, though more studies are needed, said Goodman of UBS. Incyte is partnered with Eli Lilly & Co., of Indianapolis.
Rigel, of South San Francisco, California, in September entered late-stage research for its JAK drug, called fostamatinib, in joint development with London-based AstraZeneca Plc. A study in July 2009 failed to show statistically significant improvement for patients taking the drug.
Vertex, based in Cambridge, Massachusetts, is conducting a mid-stage trial of its JAK drug, VX-509.
Methotrexate, now generic, was approved in 1988 and sells for less than $1 a pill. Arava, a pill approved in 1998 for Paris-based Sanofi-Aventis SA, is also sold as a generic.
To contact the reporter on this story: Tom Randall in New York at trandall6@bloomberg.net.
To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net.
Teva is behaving much more stupidly than I am willing to believe, much as I'd like to.
Interesting to see that CYPB remains interested in ALXA's inhalation technology despite concerns in the CRL.
Did CYPB mention what Savella sales milestones would trigger payments? There is probably a significant amount left.
INTERVIEW: Sandoz Says Well Placed For Biotech Generics Growth
NOVEMBER 2, 2010, 12:12 P.M. ET
By Goran Mijuk
Of DOW JONES NEWSWIRES
http://online.wsj.com/article/BT-CO-20101102-713188.html
ZURICH (Dow Jones)--Sandoz AG Chief Executive Jeff George believes the Swiss generics maker is well placed to broaden the fledgling market for copies of biotech medicines, for which regulators still need to provide a clear framework.
The Swiss firm, a unit of Novartis AG (NVS), already has a leading position in this segment, wielding a market share of roughly 50%, ahead of competitors Teva Pharmaceutical Industries Ltd (TEVA.TV) of Israel and U.S.-based Hospira Inc (HSP).
"We expect to continue to be the leader in [this market] looking forward," George told Dow Jones Newswires in an interview Tuesday.
Called biosimilars, the drugs are the closest thing to a generic for a biologic therapy.
Sandoz has three such products on the market--the first was launched in 2006. That trio of drugs generated $118 million in sales last year, with growth rates of more than 70%. Since the start of 2010, sales from the three biosimilars have jumped another 59%.
The market for biotech generics is set to become red hot. According to Datamonitor, sales could reach more than $2 billion by 2014. By 2020, the market could grow "well over $20 billion", according to estimates provided by Sandoz.
But much of the expected growth will depend on how regulators will treat this new class of generic medicines.
The U.S. Food and Drug Administration is currently discussing how this new class of drugs should be handled. In Europe, regulators are more advanced but still need to decide on the regulatory framework for copies of complex biotech medicines such as antibodies.
In contrast to traditional generics, which copy chemically synthesized drugs, biotech generics attempt to impersonate proteins that are made in living cells. Due to this complicated manufacturing process, which is prone to errors, generic versions of biotech drugs are similar and not identical versions of the original medicines. Hence, they are often called "biosimilars" or "follow-on biologics."
CEO George said new regulations should guarantee that biosmilars are safe, pure and potent medicines but at the same time regulations should only allow entry barriers that help make the medicines affordable to patients.
Manufacturing biosimilars is expensive, especially when compared to simple-to-produce chemical drugs which cost around $3 million. "It costs about $75 million to $250 million to produce a biosimilar," George said, adding that the final amount "depends on the complexity of the molecule."
Strict regulations--especially extensive testing of biosimilars--could inflate these costs and turn biosimilar ventures into a less lucrative business, analysts say. Consultancy Kalorama also said in a research report that consumer resistance due to a lack of clear regulatory guidelines and uncertainty over the safety of biosimilars could potential curb the market's potential.
But should entry barriers be reasonable, bringing biosimilars to market could help substantially slash costs for patients. According to German health research consultancy IGES Institut GmbH, biosimilars can help reduce health care costs by more than 15%.
Despite the regulatory concerns and efforts from biotech drug producers to raise the bar for generics makers and patient concerns, growth could be exponential as many biological drugs will lose patent protection in a few years time, including Roche Holding AG's (ROG.VX) cancer drugs Mabthera and Herceptin and Amgen Inc's (AMGN) autoimmune disease medicine Enbrel, analysts say.
"We have about 8 to 10 molecules in our pipeline," George said. "The focus is on the big opportunity" to launch biosimilar versions of medicines known as monoclonal antibodies, declining to specify which drugs Sandoz is working on at the moment. Competitor Teva has already said it is working on biosimilars of Roche's drugs.
As the market is awaiting a quantum leap in sales for this new class of generic drugs amid partnerships and M&A transactions--recent deals include Pfizer Inc's (PFE) deal with India's Biocon Ltd (532523.BY) to produce insulin--Sandoz can go it alone, although it will continue to invest "hundreds of millions of dollars over time," George said.
George said Sandoz doesn't need any bolt-on acquisitions.
"We are well positioned to grow organically," CEO George said, adding that Sandoz's early start in this market segment--it launched research in 1996--has allowed it to create technological expertise and attract key industry talent. Synergies with parent Novartis also help its biosimilars franchise.
"An acquisition isn't critical" and "we already have big facilities," George said. While Sandoz, which had nearly $10 billion in annual sales in 2009, owns several dozen factories around the world, the company has three facilities dedicated to the production of biotech generics.
-By Goran Mijuk, Dow Jones Newswires, +41 43 443 80 47; goran.mijuk@dowjones.com
Lilly CEO Calls for a 'Wave of Invention' to Reduce the Toll of Diabetes
Wednesday, November 3, 2010
http://www.pharmpro.com/news/2011/11/pharmaceutical-companies-Reduce-the-Toll-of-Diabetes/
In a speech to the Cleveland Clinic Medical Innovation Summit, John C. Lechleiter, Ph.D., chairman, president and CEO of Eli Lilly and Company described the "paradox of progress" against diabetes, noting that "for all our progress in treating diabetes, our ultimate goal seems as far away as ever." He called for a new "wave of invention" to combat the disease.
"Breakthroughs against diabetes are just as urgently needed today as they were a century ago," Lechleiter said, adding that current trends suggest that one in every three Americans could have type 2 diabetes by 2050, up from one in 10 Americans who live with the disease today.
"As we all know, this is a health and economic time bomb," said Lechleiter, noting that diabetes is the leading cause of new cases of blindness, kidney failure, and non-traumatic amputations, as well as a leading contributor to heart disease and stroke.
Since Lilly helped mass produce insulin for patients worldwide in the 1920s, the company has remained dedicated to fighting diabetes, maintaining the goal of offering a full range of treatment options for diverse patients in the many stages of the disease. Just yesterday, the company announced a new center for basic diabetes research in China, where an estimated 92 million people - almost 10 percent of the adult population - today are afflicted with diabetes.
Lechleiter stressed the importance of pursuing many paths of research given that diabetes, obesity and metabolic syndrome are complex conditions impacting a diverse group of patients. Lechleiter outlined some of the most promising developments in biopharmaceutical labs, including:
-- How researchers are looking into the genetics that underlie susceptibility to diabetes;
-- Ways researchers are improving insulin delivery through novel injection and infusion technology, and improved glucose monitoring and data integration;
-- Progress around "glucose-plus" therapies that both better control glucose levels and address related aspects of cardiovascular risk; and
-- Other advances that could help move us from managing type 2 diabetes to truly modifying the disease and improving patients' lives.
Despite these advances, more must be done. "While the potential of research has never been greater, and the need for breakthroughs more urgent, there are serious barriers to innovation," Lechleiter said.
"We must find new approaches that reduce the cost and time of drug development and deliver more value to patients." Lechleiter gave examples of how the industry is working to "reinvent invention" to address some of these barriers. He pointed to increased collaboration across the industry in precompetitive stages of research, new approaches to clinical trials that make it possible to adjust trials as they proceed in order to capture learning and lower costs, and the growing ability to tailor medicines to meet the distinct needs of individual patients.
Lechleiter warned that "all of these new approaches, all of the potential of current medical advances, are at risk without an environment that supports medical innovation." He added, "To sustain progress against diabetes, public policies - including benefit/risk assessments, reimbursement decisions, and prescribing guidelines - must enable and foster true medical innovation." Lechleiter urged creation of a systematic and transparent regulatory approach to assessing the benefits and risks of new medicines. He noted that ongoing efforts with the FDA on the Prescription Drug User Fee Act - which is up for reauthorization in 2012 - offered an opportunity for a "real victory for innovation and for patients." These policy objectives should be part of a larger "ecosystem" that allows innovation to flourish, according to Lechleiter. Such an ecosystem, he said, must include an "atmosphere" where innovation can thrive, adequate "nutrients" in the form of monetary investments supported by sound tax policy and protection of intellectual property, and the "seeds" of human talent in science and math--areas where America's children are currently behind the curve.
Lechleiter concluded with a call to action, saying, "We must achieve continued innovation in diabetes treatment. We must aggressively pursue the fight, to alleviate the suffering brought about by the long-term consequences of this devastating disease. And, to sustain this progress against diabetes, we must all support public policies that enable and reward medical innovation."
Is the Street questioning Teva so relentlessly because they see their dominance being challenged, because of Copaxone or because of Momenta?
I thought they sounded pretty confident on "sameness" - whether that is their version of sameness or the FDA version remains to be seen. But personally I'm a little more cautious after their call.
We believe our competitors will need to do substantial work to meet the FDA's criteria for approval.
I think there's a lot of stuff that Teva claims that doesn't add up but to me the biggest question is why should Marth BS now and look like a complete idiot a couple of years down the road when tEnox's application is gathering dust at the FDA? Teva is not a small biotech that needs to pump its stock up to raise money. They can't be intentionally trying to slow down MNTA by keeping its share price depressed because MNTA has Sandoz to back it up. I think knowing Marth's motive would be reassuring.
Wheeler on M-enox in EU
Well, so, as this is a – my personal belief is it's worth taking a shot at that. We haven't announced anything that we're doing. Of course, you remember that the EU did put out guidelines which required trials for this but I think with the approval and the successful use of this in the U.S. that may open the door but we have not announced any activities there yet.
Re a lovenox authorized generic, the only instance that topic appeared to come up was at around the 28 minute mark. In response to an AG related question Viehbacher appeared to say that SNY doesn't want to tips its hand and then Spek said SNY would be ready when the right scenario occurs, whatever that scenario may be. I agree that he was probably referring to an additional generic entering the market. As long as m-enox maintains a 40-60% market share range there is no logic in introducing an AG.
http://www.thomson-webcast.net/uk/dispatching/?event_id=0c88d7ba71afe501d7b2e25c244be152&portal_id=2ba4dbdbaced91ffe0c73e301ff6ea0e
I think this quote from the article makes the distinction.
However, the government suggested such a change would have limited impact on the biotechnology industry because man-made manipulations of DNA, like methods to create genetically modified crops or gene therapies, could still be patented.
It's misleading because it didn't clearly state what the drug is approved for in NYTimes while it is in Bloomberg:
From Bloomberg:
The medicine, Nuedexta, is the first approved to treat patients with multiple sclerosis or Lou Gehrig’s disease who develop symptoms known as pseudobulbar affect that causes loss of emotional control, the Food and Drug Administration said in a statement.
It is laughter devoid of inner joy, and weeping without sorrow.
Involuntary outbursts of laughing and crying not tied to underlying emotion can afflict people with neurological ailments like multiple sclerosis, Lou Gehrig’s disease, traumatic brain injury and stroke.
Now there is a drug for it. On Friday, the Food and Drug Administration approved the first medicine to treat the condition, which is called pseudobulbar affect.
I'm not sure I understand why you think its misleading? It appears to question the market more than the drug while the bloomberg article appears to accept the analysts claims of a large market without questioning it.
Well the reference is only to the genes which I think we can all agree were not invented by drug companies.
I'm not familiar with AVNR at all but it seems to me that a decent number of the more regarded posters on this board would have concluded that this company was a sham based on this NYT article. I'm surprised, given that the condition doesn't even have a proper name and the drug appears to have safety issues, that the FDA approved it.
October 30, 2010, 10:14 AM
A Pill to Control Laughing and Weeping
By ANDREW POLLACK
http://prescriptions.blogs.nytimes.com/2010/10/30/a-pill-to-control-laughing-and-weeping/?src=twt&twt=nytimeshealth
It is laughter devoid of inner joy, and weeping without sorrow.
Involuntary outbursts of laughing and crying not tied to underlying emotion can afflict people with neurological ailments like multiple sclerosis, Lou Gehrig’s disease, traumatic brain injury and stroke.
Now there is a drug for it. On Friday, the Food and Drug Administration approved the first medicine to treat the condition, which is called pseudobulbar affect.
The drug, called Nuedexta, was developed by Avanir Pharmaceuticals of Aliso Viejo, Calif.
Keith Katkin, chief executive of Avanir, said the company estimates that 2 million Americans suffer from pseudobulbar affect.
“We like to refer to PBA as one of the most common diseases that people have never heard of,’’ he said in an interview earlier this week.
Estimates are somewhat imprecise, however. Only five years ago the company was estimating that there were fewer than 1 million people with the condition.
The company faces a marketing challenge. Many patients and doctors do not know the condition exists. And there is skepticism that PBA warrants treatment, given that people with the condition have more serious underlying diseases.
Mr. Katkin said that laughing and crying at inappropriate times can be socially debilitating. He cited the case of a grandmother who was not allowed to stay with her young grandchildren because she would suddenly burst into tears and scare them.
The difficult and unusual name of the condition is also a barrier to marketing the drug, especially since the “pseudo’’ makes it sound like a fake ailment. The company at one point tried to coin its own name – involuntary emotional expression disorder — but the F.D.A. nixed that.
Nuedexta is a combination of two existing drugs. One is dextromethorphan, an ingredient in some cough medicines. The other is quinidine, a drug used to treat malaria and heart arrhythmia.
It is believed that dextromethorphan binds to certain receptors in the brain to dampen the laughing and crying. The quinidine keeps the dextromethorphan from breaking down quickly.
The F.D.A. turned down the drug in 2006, citing concerns that the quinidine could cause heart rhythm problems. Avanir did a new clinical trial using only one third of the previous dose of quinidine.
The trial involved 326 patients with either multiple sclerosis or Lou Gehrig’s disease, known formally as amyotrophic lateral sclerosis.
Before entering the trial, the patients had 4 to 6 episodes of laughing or crying a day, with each usually lasting from 30 seconds to more than five minutes.
At the end of 12 weeks the number of incidents for those getting the high dose of the Neudexta had fallen by about 88 percent, to only about 0.6 per day. That was a significantly greater reduction than for those getting the placebo.
Neudexta will go on sale in the first quarter. While the price hasn’t been announced, Mr. Katkin said it would probably be between $3,000 and $5,000 a year. Analysts estimate sales could eventually reach a few hundred million dollars a year.
U.S. Says Genes Should Not Be Eligible for Patents
By ANDREW POLLACK
Published: October 29, 2010
Reversing a longstanding policy, the federal government said on Friday that human and other genes should not be eligible for patents because they are part of nature. The new position could have a huge impact on medicine and on the biotechnology industry.
(contd)
http://www.nytimes.com/2010/10/30/business/30drug.html?_r=2&hp