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alexa
I never trusted the Alexa management. I went to a group meeting a few weeks after they went public. After looking at their prospectus I thought they were a home mortgage company because all of the exectutives took out multimillion dollar mortgages from the company to buy homes.
didn't biovail give them 40 million up front to lisence this. Wouldn't they have been concerned about the pulmonary function problem?
can you give me some background on what you are saying about access being a fraud.
and yes, i have invested in private placements and then gave positive comments on boards. If I didn't think positively about the company I wouldn't have invested in the private placement.
the oral mucositis product is approved, and it is approved for treatment prior to getting oral mucositis. I have seen the label, that is not a fraud.
I have invested in private placements but I typically buy on the open market more than do private placements.
Please do not mention me and rodman in the same sentence. They do not invest, they simply market the pipes
Allos Therapeutics Announces Presentation of Favorable Survival Data from Randomized Phase 2b Study of FOLOTYN® in Patients with Advanced Non-Small Cell Lung Cancer
Results demonstrate clinical activity of FOLOTYN in advanced NSCLC
Data presented at 35th European Society of Medical Oncology Congress
overall survival was higher.. Data at six and twelve months was very good... median survival of folotyn was lower. this means the failures came early.
Press Release Source: Allos Therapeutics, Inc. On Monday October 11, 2010, 7:15 am EDT
MILAN--(BUSINESS WIRE)-- Allos Therapeutics, Inc. (NASDAQ:ALTH - News) today announced the presentation of favorable survival data from the Company’s international, randomized Phase 2b investigational study of FOLOTYN® (pralatrexate injection) relative to erlotinib in patients with Stage IIIB/IV (advanced) non-small cell lung cancer (NSCLC). In this study, patients receiving FOLOTYN had a 16 percent reduction in the risk of death compared to erlotinib in the overall (intent-to-treat) population (n=201; hazard ratio (HR)=0.84) and a 13 percent reduction in the risk of death in the primary efficacy analysis population (n=166; HR=0.87). At six months, 56 percent of patients treated with FOLOTYN were alive and 51 percent of patients treated with erlotinib were alive; at one year, 28 percent of patients treated with FOLOTYN were alive and 18 percent of patients treated with erlotinib were alive. The median overall survival (OS) time was 6.7 months for patients who received FOLOTYN and 7.0 months for patients who received erlotinib. The safety profile of FOLOTYN was consistent with that observed and reported in previous FOLOTYN solid tumor studies.
“Overall survival remains the most clinically relevant endpoint in assessing the benefit of an investigational therapy to treat cancer,” said Karen Kelly, M.D., an investigator of the study, The University of Kansas Medical Center. “I believe the survival data from this randomized Phase 2b study are compelling and warrant exploring Phase 3 options in order to fully understand the potential role of FOLOTYN to treat patients with advanced non-small cell lung cancer.”
The objective of this randomized, international, multi-center Phase 2b study (PDX-012) was to estimate the efficacy of FOLOTYN relative to that of erlotinib as assessed by OS, the primary endpoint of the trial. All patients had received one or two prior systemic treatments including at least one prior platinum-based regimen. Secondary endpoints included progression-free survival (PFS) (HR=0.91; median PFS=3.4 months and 2.8 months for FOLOTYN and erlotinib, respectively) and objective response rate (RR) (2 percent and 7 percent, respectively). Disease control rate (DCR), which includes patients with complete responses, partial responses or stable disease, was 36 percent for FOLOTYN and 43 percent for erlotinib.
Analyses were also performed according to the statistical analysis plan to assess the activity of FOLOTYN and erlotinib in predefined patient cohorts. Most notably, patients with non-squamous cell carcinoma (n=107) who received FOLOTYN experienced a 35 percent reduction in the risk of death (OS HR=0.65) and 42 percent reduction in the risk of disease progression (PFS HR=0.58) relative to erlotinib. In patients with squamous cell carcinoma, a hazard ratio for OS of 1.06 was observed, which suggests activity of FOLOTYN given that erlotinib has historically shown a survival benefit in these patients. In the small subset of patients who received prior pemetrexed (n=30), a hazard ratio for OS of 1.15 was observed.
The most common Grade 3-4 adverse event observed in patients treated with FOLOTYN was mucositis (23 percent). Other Grade 3-4 adverse events occurring in more than five percent (but less than 10 percent) of patients were fatigue (9 percent), dyspnea (6 percent), neutropenia (6 percent), thrombocytopenia (5 percent) and anemia (5 percent) in patients treated with FOLOTYN, and rash (8 percent), dyspnea (8 percent), anemia (8 percent) and fatigue (5 percent) in patients treated with erlotinib.
“Advanced non-small cell lung cancer is an aggressive disease. Unfortunately, nearly all patients will progress after their initial treatment,” said Charles Morris, MB ChB, MRCP, chief medical officer at Allos Therapeutics. “This study is providing Allos and the lung cancer community with important data regarding the efficacy and safety profile for FOLOTYN in advanced NSCLC, where there remains a high unmet medical need. We believe these data demonstrate the clinical activity of FOLOTYN in patients with advanced NSCLC, and we are in the process of exploring potential Phase 3 development options for FOLOTYN in this indication.”
These data were presented in an oral presentation at the 35th European Society of Medical Oncology (ESMO) Congress in Milan, Italy. The company had previously announced the topline results from this trial in July 2010.
Study Design
The objective of this randomized, open-label, international, multi-center Phase 2b study was to estimate the efficacy of FOLOTYN relative to that of erlotinib, marketed as TARCEVA®, as assessed by overall survival. The trial enrolled 201 current or former smokers with Stage IIIB/IV (advanced) NSCLC who had received one or two previous treatments including at least one prior platinum-based chemotherapy regimen. The primary endpoint of the trial was overall survival. Secondary endpoints included progression-free survival and response rate as compared with erlotinib, and the safety and tolerability of FOLOTYN. The study was not designed to show a statistically significant difference between the two treatment arms. No definition of statistical significance was included in the statistical analysis plan.
The trial – which enrolled patients across 43 locations worldwide (15 U.S. sites and 28 ex-U.S. sites) – was initiated by Allos in January 2008 and completed enrollment in July 2009. The first 35 patients enrolled in the study were randomly assigned one-to-one to receive FOLOTYN (intravenous push on days 1 and 15 of a 28-day cycle; initial dose of 230 mg/m2) or erlotinib (oral, 150 mg daily in a 28-day cycle). Following a protocol amendment, 166 patients were randomly assigned one-to-one to receive either FOLOTYN at 190 mg/m2 or erlotinib at 150 mg/day and then further stratified by light versus heavy smokers. All patients received concurrent vitamin therapy of B12 and folic acid. According to the statistical analysis plan, analyses were conducted to assess the activity of FOLOTYN and erlotinib in predefined patient cohorts, which included light smokers (n=37) and heavy smokers (n=164), current smokers (n=50) and former smokers (n=151), squamous (n=76) and non-squamous histology (n=107), and patients who received prior pemetrexed (n=30) and those who had not (n=171).
About Lung Cancer
More people die each year from lung cancer than any other type of cancer – including breast, prostate, and colorectal cancers combined. In 2010, it is estimated that there will be more than 200,000 new cases of lung cancer diagnosed in the United States and over 150,000 deaths. There are primarily two types of lung cancer: NSCLC and small cell lung cancer (SCLC). NSCLC accounts for the majority of lung cancers (about 87 percent) and develops slowly – often causing few or no symptoms until very late stages. The most common subtypes of NSCLC are squamous cell carcinoma and non-squamous carcinomas such as adenocarcinoma and large-cell undifferentiated carcinoma. Squamous cell carcinomas account for 25-30 percent of all lung cancers while adenocarcinoma and large-cell undifferentiated carcinoma account for 40 percent and 10-15 percent of lung cancers, respectively. The majority of people are diagnosed with advanced stage disease and only one to five percent of people with advanced stage (IIIB/IV) NSCLC survive to five years. The most widely used therapies to date remain surgery, chemotherapy, and radiation therapy.
About Allos Therapeutics
Allos Therapeutics, Inc. (Nasdaq:ALTH - News) is a biopharmaceutical company committed to the development and commercialization of innovative anti-cancer therapeutics. Allos is currently focused on the development and commercialization of FOLOTYN® (pralatrexate injection), a folate analogue metabolic inhibitor. FOLOTYN is the first and only drug approved in the U.S. for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma. Allos is also developing FOLOTYN in other hematologic malignancies and solid tumors. Allos retains exclusive worldwide rights to FOLOTYN for all indications. Allos is headquartered in Westminster, CO. For additional information, please visit www.allos.com.
IMPORTANT SAFETY INFORMATION
Warnings and Precautions:
FOLOTYN may suppress bone marrow function, manifested by thrombocytopenia, neutropenia, and anemia. Monitor blood counts and omit or modify dose for hematologic toxicities.
Mucositis may occur. If = Grade 2 mucositis is observed, omit or modify dose.
Patients should be instructed to take folic acid and receive vitamin B12 to potentially reduce treatment-related hematological toxicity and mucositis.
FOLOTYN can cause fetal harm. Women should avoid becoming pregnant while being treated with FOLOTYN, and pregnant women should be informed of the potential harm to the fetus.
Use caution and monitor patients when administering FOLOTYN to patients with moderate to severe renal function impairment.
Elevated liver function test abnormalities may occur and require monitoring. If liver function test abnormalities are = Grade 3, omit or modify dose.
Dermatologic reactions may occur. Patients with dermatologic reactions should be monitored closely.
Adverse Reactions:
The most common adverse reactions observed were mucositis (70%), thrombocytopenia (41%), nausea (40%), and fatigue (36%). The most common serious adverse events were pyrexia, mucositis, sepsis, febrile neutropenia, dehydration, dyspnea and thrombocytopenia.
Use in Specific Patient Population:
Nursing mothers should be advised to discontinue nursing or the drug, taking into consideration the importance of the drug to the mother.
Drug Interactions:
Co-administration of drugs subject to renal clearance (e.g., probenecid, NSAIDs, and trimethoprim/sulfamethaxazole) may result in delayed renal clearance.
For additional important safety information, please see the full prescribing information for FOLOTYN at www.allos.com.
Safe Harbor Statement
This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding the potential safety and efficacy of FOLOTYN for the treatment of patients with advanced non-small cell lung cancer, the potential future development of FOLOTYN for the treatment of advanced non-small cell lung cancer; and other statements that are other than statements of historical facts. In some cases, you can identify forward-looking statements by terminology such as “may,” “will,” “should,” “expects,” “intends,” “plans,” anticipates,” “believes,” “estimates,” “predicts,” “projects,” “potential,” “continue,” and other similar terminology or the negative of these terms, but their absence does not mean that a particular statement is not forward-looking. Such forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that may cause actual results to differ materially from those anticipated by the forward-looking statements. These risks and uncertainties include, among others: that data from preclinical studies and clinical trials may not necessarily be indicative of future clinical trial results; that the safety and/or efficacy profile for FOLOTYN may not support further clinical development in advanced non-small cell lung cancer; and the risk that the Company may lack the financial resources and access to capital to fund future clinical trials for FOLOTYN. Additional information concerning these and other factors that may cause actual results to differ materially from those anticipated in the forward-looking statements is contained in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2010, and in the Company's other periodic reports and filings with the Securities and Exchange Commission. The Company cautions investors not to place undue reliance on the forward-looking statements contained in this press release. All forward-looking statements are based on information currently available to the Company on the date hereof, and the Company undertakes no obligation to revise or update these forward-looking statements to reflect events or circumstances after the date of this presentation, except as required by law.
Note: The Allos logo and FOLOTYN name are trademarks of Allos Therapeutics, Inc.
Access Pharmaceuticals
MuGard Provides Significant Improvement or Stabilization of Oral Mucositis in Over 75% of Patients in European Clinical Experience
Patients Experienced 52% Reduction in Pain and Oral Discomfort; MuGard's Ready-to-Use Formulation Well Accepted By 86% of Patients
ACCP.OB 2.63 0.00
Press Release Source: Access Pharmaceuticals, Inc. On Monday October 11, 2010, 8:00 am EDT
DALLAS and NEW YORK, Oct. 11 /PRNewswire-FirstCall/ -- Access Pharmaceuticals, Inc. (OTC Bulletin Board:ACCP.ob - News), a biopharmaceutical company specializing in products for cancer and supportive care, announced the highlights from a poster presentation on European clinical experience of MuGard at this year's European Society of Medical Oncology Conference in Milan, Italy. Some of the findings reported in the ESMO presentation included:
MuGard provided a significant improvement and/or stabilization of oral mucositis lesions and symptoms of oral mucositis (OM) in more than 75% of cancer patients treated with radiotherapy/chemotherapy;
Patients reported a 52% significant reduction in oral discomfort (oral pain and swallowing ability) in patients with pre-existing lesions of OM, and reported a substantial reduction in the use of pain medication; MuGard has a ready-to-use formulation that is well accepted by 86% of patients, as reported in this assessment program; No MuGard-related adverse reactions have been identified in clinical practice; MuGard represents a valuable therapeutic option in the treatment and prevention of OM in cancer patients undergoing radio/chemotherapy.
"These additional findings out of Europe are very exciting and strengthen our belief in the clinical benefits that MuGard can provide patients undergoing radiation and chemotherapy," said Jeffrey Davis, CEO of Access Pharmaceuticals. He continued, "This data confirms the competitive advantages of MuGard over the current treatment options on the market and we will use this data to educate patients, clinicians and the medical industry on the superior attributes of our product."
Earlier this year, Access presented MuGard data and clinical experience at the MASCC Conference (Multinational Association of Supportive Care in Cancer). At that conference, Access presented data from a US clinical trial in patients undergoing radiation and/or chemotherapy for treatment of head and neck cancer showing that 42% of patients using MuGard preventatively never exceeded an Oral Mucositis Assessment Scale (OMAS) score of 0.5. This data underscored MuGard's potential for oral mucositis prevention. The overall comparison showed that patients treated with MuGard had significantly lower OMAS scores when compared to the three control groups. The largest benefit was observed when patients used MuGard on the first day they started cancer therapy and continued using MuGard during the entire duration of treatment. Additionally, MuGard lowered erythema (a precursor inflammatory condition to OM) scores in subjects with various oral inflammatory conditions, including patients with xerostomia, cheilitis and stomatitis. The presentation of clinical experience at the ESMO conference is the culmination of all previously announced European clinical programs.
For additional information on MuGard, including information for clinicians, nurses and patients, please visit www.MuGard.com.
About MuGard:
MuGard is a novel, ready-to-use, easy-to-swallow, mucoadhesive oral wound liquid that provides a protective polymer coating over the mucosa. MuGard received 510(k) marketing allowance in the US with a broad indication for treating lesions of the mouth including the management of oral mucositis. In Europe, MuGard is a class IIa medical device which has been granted CE mark certification with the following indication: prevention and management of the lesions and symptoms of oral mucositis Up to 40% of all patients receiving chemotherapy and radiotherapy develop moderate to severe mucositis, and almost all patients receiving radiotherapy for head and neck cancer and those undergoing stem cell transplantation develop mucositis. Updated clinical practice guidelines for the prevention and treatment of mucositis recommend the use of a preventive oral care regimen as part of routine supportive care along with a therapeutic oral care regimen if mucositis develops. The market for the treatment of oral mucositis is estimated to be in excess of $1 billion world-wide.
About Access:
Access Pharmaceuticals, Inc. is an emerging biopharmaceutical company that develops and commercializes proprietary products for the treatment and supportive care of cancer patients. Access' products include ProLindac™, currently in Phase II clinical testing of patients with ovarian cancer, and MuGard™ for the management of patients with mucositis. The company also has other advanced drug delivery technologies including CobaCyte™-mediated targeted delivery and Cobalamin™ oral drug delivery, its proprietary nanopolymer delivery technology based on the natural vitamin B12 uptake mechanism and Thiarabine, a new generation nucleoside analog which has demonstrated both pre-clinical and clinical activity in certain cancers. For additional information on Access Pharmaceuticals, please visit www.accesspharma.com.
This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include those relating to: our cash burn rate, clinical trial plans and timelines and clinical results for ProLindac, MuGard, Thiarabine and Cobalamin and other product candidates, our ability to achieve clinical and commercial success and our ability to successfully develop marketed products. These statements are subject to numerous risks, including but not limited Access' need to obtain additional financing in order to continue the clinical trial and operations and to the risks detailed in Access' Annual Reports on Form 10-K and other reports filed by Access with the Securities and Exchange Commission.
Contact: Company
Contact: Investor Relations
Christine Berni
Donald C. Weinberger/Diana Bittner (media)
Director of Investor Relations
Wolfe Axelrod Weinberger Assoc. LLC
Access Pharmaceuticals, Inc.
(212) 370-4500
(212) 786-6208
how can they go into a phase 3 combination trial without doing a combination phase 2 trial.
what if there are drug interactions?
Amgen drug extends survival in ovarian cancer study
I would say they should charge a minimum of 250k a year with benefit like this. why are they even going into phase 3?
* AMG 386 extends survival 1.6 mos vs chemotherapy alone
* Amgen has launched Phase 3 trial
LOS ANGELES Oct 9 (Reuters) - A mid-stage trial of an experimental drug being developed by Amgen Inc (AMGN.O) found that it can extend survival by several weeks for women with advanced ovarian cancer when combined with chemotherapy.
The 161-patient Phase 2 study found that patients treated with the higher dose of the drug, called AMG 386, lived for a median of 22.5 months, compared with 20.4 months for those given a lower dose of the drug, and 20.9 months for patients who received only the chemotherapy drug paclitaxel.
Amgen said it has begun a Phase 3 of the drug, in combination with paclitaxel, as a treatment for ovarian, primary peritoneal, and fallopian tube cancers.
The main goal of the Phase 2 study was the time it took for the disease to worsen in half the patients in each treatment group -- a measure known as median progression-free survival.
Patients who got the higher dose of AMG 386 had median progression-free survival of 7.3 months, compared with 7.4 months in those who received a lower dose and 5 months for those who received only paclitaxel.
Earlier results reported in June had shown progression-free survival of 7.2 months for the high dose, 5.7 months for the lower dose and 4.6 months for chemotherapy alone.
The updated results were reported at a medical meeting in Milan, Italy on Saturday.
AMG 386 is an anti-angiogenesis drug, which means it inhibits the growth of blood vessels needed to feed a tumor.
Adverse side effects seen with the drug included peripheral neuropathy, low levels of potassium in the blood and blood clots. (Reporting by Deena Beasley; editing by Carol Bishopric)
anth
is this based on MoA of ANTH's drug theoretically being superior in some way
yes
but early, it could be better, or just as good. or could have a safety issue. But I would prefer to have all my eggs in that basket than have them spend money on an acs trial that I think has a 95 percent chance of failing
anthera
I sat down with the ceo and cfo at a deutche bank conference a few months ago. They had the drug for acs and another one for lupus.
I told them if they only did the trial for lupus and dropped acs I would buy the stock.
they have absolutely no data that tells me that their acs trial has a chance.
the lupus drug looks like a better version of the hgsi compound
Update for Soligenix from Jason Kolbert of National Securities
Investment Thesis
Soligenix provided a corporate update earlier this week, announcing a delay in the timing of results of the pivotal trial of orBec® in treatment of acute GvHD. This is due to a setback in the enrollment of subjects outside the United States. Investors are now focused on results for the prevention study by year end. We have a high confidence in the results for the acute study BUT the prevention study is more of an unknown.Nevertheless at this valuation our investment thesis is unchanged for Soligneix, as the delay in the completion of the orBec® pivotal study is just that, a slight delay.
Highlights
Delay in completion of Phase III study of orBec® in treatment of acute GVHD, previously anticipated at 1H-2011, we now expect completion by 2H-2011. It is still possible for the study to be finished on-time by 1H-2011, but there were delays in the initiation of study site outside the United States for patient enrollment. Soligenix with ~$10 million in capital should have enough to fund the pivotal trial to completion.
orBec® Prevention is the Next Driver: Results for the Phase II GvHD prevention study should arrive no later than year end. The trial is randomized 2:1 across 140 patients. The primary endpoint of the trial is the proportion of subjects who develop acute GVHD with severity sufficient to require systemic immunosuppressive treatment on or before day 90 after transplantation. Subjects in the orBec® group began study drug at the start of their conditioning regimen and continued through day 75 following hematopoietic cell transplantation (HCT). Other endpoints will include the severity curve (we expect to see it shift from Stage III & IV’s to I & II’s), the amount of organ involvement and, of course, survival. A 15% or greater difference would be clinically “a home run” in our opinion.
Shareholders approve the newly elected Tamar D. Howson and Virgil D. Thompson to join the Board of Directors, while reinstating the previous directors. The two bring on in-depth business development and financial expertise as well as added value to the continual development of Soligenix’s main driver, orBec® in preventing and treating GVHD. Tamar Howson, an alumna of Columbia University Business School, has 20 years of experience in licensing, collaborations, and M&A transactions as a corporate and business development executive at a number of pharmaceutical and biotechnology companies. She currently is a partner with JSB-Partners and held senior positions in industry at SmithKline Beecham and Bristol-Meyers Squibb as well as conducted venture capital investments at Johnston Associates. She currently is on the board of directors at three biopharmaceutical companies. Virgil Thompson, a BS degree in pharmacy from Kansas University and George Washington University Law school graduate, brings 40 years of pharmaceutical as well as biopharmaceutical management and financial oversight experience. He has served as president and CEO of numerous companies and currently is CEO of a privately-held ophthalmic company, Spinnaker Biosciences. Certainly, Tamar and Virgil are not new to the life sciences industry (refer to exhibit 2 for past and current directorship positions).
Valuation: Our valuation for Soligenix is based on several metrics, including FCFF, DCF EPS and Sum of the Parts models. These metrics all suggest a target north of $1.00 versus the current $0.18. The key assumption we make is that orBec® reports positive data in the current Phase III confirmatory pivotal trial in acute GvHD (2H-2011). The next major event that will impact the valuation of the Company will be the results from the Phase 2 prevention study, expected by year end. Expectations are mixed to positive for the data near term. Our Buy rating is based on the longer term view around acute GvHD trial..
Fundamental Research:
Biotechnology
October 1, 2010
Bull Case: Soligenix is undervalued as the potential for orBec® is underappreciated. The prior trials (Phases 2 & 3) and the current confirmatory Phase 3 trial have effectively de-risked the outcome in acute GvHD. The scientific rational behind prevention is also strong and will be validated when results are reported before year’s end (2010). The current confirmatory (acute) trial is highly powered (90%) and of the same design (drug, dose, patient population and expected control group outcomes) as the prior pivotal trial (powered at 80%) The primary endpoint: Treatment failure rate at Study Day 80, was highly statistically significant in the previous Phase 3 trial with p-value of 0.005. Market factors suggest orBec® can acquire significant penetration in the GvHD marketplace as the first approved drug for GI GVHD. The GvHD market is concentrated and closely-knit and information is disseminated rapidly among the Key Opinion Leaders (KOL’s). Beyond GvHD (Acute, Prevention and Chronic) a wide range of other indications, such as Radiation Enteritis and Crohn’s Disease exists. North American rights are partnered with Sigma-Tau EU and the rest of the world, but however, are open for partnership. Soligenix also has an attractive drug delivery platform with Lipid Polymer Micelles (LPM) platform technology and a very active BioDefense program led by RiVax™ (Ricin Toxin Vaccine). All this, with a market capitalization of under $50 million.
Bear Case: orBec® is similar to generic enteric-coated budesonide and other steroids which are cheaply available and used today off-label to treat GvHD. orBec® will not be able to achieve the price we project even with the pharmacoeconomic justifications, as hospitals will continue to use cheaper steroids to treat GvHD. Bears may also argue that the clinical risk is being underestimated in both the current orBec® confirmatory trial (pivotal – acute trial) and especially in the prevention trial. Performance in the acute setting does not ensure good results that orBec® will be effective in prevention and that data is reported before year end. Bears will also argue that Soligenix has given away too much of the economics in the initial license deal with Sigma-Tau. The GvHD marketplace is small and treatment paradigm is shifting away from BMT toward new biologic therapies; as such, the market is becoming smaller, not larger, over time. The treatment paradigm in GvHD is not likely to shift towards prevention, and that market opportunity is over-estimated. The market opportunity in chronic is unique in that these patients have a different set of immunological problems. As such, as stated previously, the clinical risk that a true benefit can be demonstrated in these patients is high (and is not implied from prior results in acute patients). The remaining opportunities at the Company include the drug delivery platform (Lipid Polymer Micelle -LPM™) which is unproven, and the Company lacks the resources to continue to develop this platform. Lastly, the biodefense platform deserves minimal value as the Company is totally dependent on the US Government as a client. Prior experiences with other companies suggest that the road to generate meaningful profit is an arduous and difficult one.
Our Take: We know that while cheaper steroids are a potential commercial threat, multiple examples of therapeutics that were re-tasked exist in other marketplaces, and once on label, off-label use of cheap generics was eliminated. In addition, the two tablet system of immediate and time release makes orBec® unique in treating this disease. We see orBec® as the key driver for Soligenix. Based on our analysis of the Phase 2 and Phase 3 trials, we see low clinical risk in the current Phase 3 (acute) confirmatory trial and a very strong rationale to expect good results in prevention. The current pivotal trial uses a favorable endpoint and greater statistical powering, (versus the prior trial) while keeping all other variables the same. As such, we see a high likelihood of success. The best case scenario for Soligenix is a positive outcome in the NIH sponsored prevention trial followed by good results in the confirmatory Phase 3, Acute GI GvHD trial. Mechanistically, we believe a good outcome in acute GvHD does set the stage for off- label use in the prevention and chronic marketplaces. Beyond orBec® in GvHD we see tremendous potential in other GI diseases (Crohn’s), Radiation Enteritis and on the BioDefense side, Radiation Injury (orBec® as a counter-measure). We view the Biodefense and LPM platforms as creating a positive upside option for investors. On the BioDefense side, little value is ascribed for RiVaxTM and/or the vaccine development platform (vaccines stable at room temperature), but investors should note that Soligenix has recently received a $9.4 million development award to support the heat stabilization platform. With cash to see the Company through to pivotal results, we believe the valuation is attractive.
Valuation: Our valuation for Soligenix is based on several metrics, including FCFF, DCF EPS and Sum of the Parts models. These metrics all suggest a target north of $1.00 versus the current $0.24. The key assumption we make is that orBec® reports positive data in the current Phase III confirmatory pivotal trial in acute GvHD (1H-2011). Positive data from the Phase 2 prevention study (2H-2010) will be important as well. Investors should pay close attention to the catalysts presented in time sequence. Each catalyst’s outcome should impact valuation. For example, should Soligenix announce favorable terms for a partnership for orBec® European rights, it should drive valuation higher.
I was speaking with a fund manager at the ubs conference.
He said he is close to Teva and if Teva was told that their approval would be coming soon that is what he expected to happen
This shows how naive some people that run money are. The FDA doesn't tell you when an approval is coming when you have a pdufa date, there is much less chance of that when you have an anda
I was speaking with a person from Momenta that said for the last two years Momenta employees were carrying around conference call speaches and other data because the approval could have come at anytime and without warning and they didn't want to be unprepared if it happened when they were on the road.
Teva has put the lid on the Momneta stock price with their statements after the approval. It has now been two months. Every week that goes by should start removing some of the confidence in the Teva statements, and that should be reflected over time in the MNTA stock price.
As discussed previously, in the FDA response to Sanafi's citizens petition the fda outline several steps that momenta was able to show the fda to convince the fda to approve the drug and each of these steps was a neccessary component. The odds that Teva had the technical knowlege to do each of these steps when they designed their drug is unlikely.
you are right, their entire premium is based on their ADC technology. If this fails they are in trouble.
I think it will work but that doesn't mean anything. I am not in the stock
there is a guy in a white thinly striped shirt, in the lower right center of the photo putting in a buy order through his iphone.
is that legal
this happens whenever Piper leads a financing
anthera stock
Sep 20, 2010 2.85 3.05 2.85 2.93 59,500 2.93
Sep 13, 2010 3.41 3.42 2.82 2.85 28,300 2.85
Sep 7, 2010 3.80 3.82 3.28 3.32 16,800 3.32
Aug 30, 2010 3.81 4.01 3.67 3.95 9,500 3.95
Aug 23, 2010 4.00 4.03 3.81 3.89 14,500 3.89
Aug 16, 2010 4.58 4.80 3.83 3.95 20,800 3.95
Aug 9, 2010 5.72 5.85 4.58 4.58 11,900 4.58
Aug 2, 2010 5.52 5.99 5.40 5.69 12,800 5.69
Jul 26, 2010 4.46 5.54 4.13 5.38 20,400 5.38
Jul 19, 2010 4.16 4.58 3.76 4.36 16,100 4.36
Jul 12, 2010 5.17 5.40 4.00 4.11 20,400 4.11
Jul 6, 2010 5.71 5.80 5.00 5.17 14,400 5.17
Jun 28, 2010 6.56 6.56 5.15 5.50 24,700 5.50
Jun 21, 2010 5.99 7.00 5.81 6.56 129,400 6.56
FDA calls for hearing on biosimilars legislation
By Julian Pecquet - 09/20/10 11:50 AM ET
This could be big for mnta
The Food and Drug Administration will hold a two-day public hearing as it prepares to put into place regulations for the approval of generic versions of biological products, according to a draft document circulating among healthcare lobbyists.
The healthcare reform law allows for the approval of generic versions of biologics — also known as biosimilars — after 12 years of patent exclusivity. Biologics refers to medications and other medical products created through biological processes rather than chemistry.
The two-day hearing would take place on Nov. 2 and 3. Since it's a draft the document could change before publication in the Federal Register, but it provides insight into the issues on which the FDA is seeking comment from industry.
An FDA spokeswoman refused to comment on the draft.
"The purpose of this public hearing," the document states, "is to create a forum for interested stakeholders to provide input regarding the agency’s implementation of the statute concerning the following issues, among others:
— Scientific and technical factors related to a determination of biosimilarity or interchangeability;
— The type of information that may be used to support a determination of biosimilarity or interchangeability;
— development of a framework for optimal pharmacovigilance for biosimilar and interchangeable biological products;
— scope of the revised definition of a “biological product”;
— priorities for guidance development;
— scientific and technical factors related to reference product exclusivity;
— scientific and technical factors that may inform the agency’s interpretation of “product class” as it relates to available regulatory pathways for certain protein products during the 10-year transition period following enactment of the BPCI Act; and
— the establishment of a user fee program for biosimilar and interchangeable biological products."
Source:
http://thehill.com/blogs/healthwatch/prescription-drug-policy/119735-fda-calls-for-hearing-on-biosimilars-legislation
The doctor, who wishes to remain anonymous, was approached in the hallway of the hotel where the AdCom was held by a Caucasian man, approximately 5’10”, mid-40’s, balding, dressed in an “expensive suit”, when the man began to use obscenities. He said, “You are a pompous asshole…you are wrong…you better get the hell out of here fast..” he repeated the comments.
sounds like david axelrod
I was at the breakout.
the way I see it other companies will have to do biosimilars (run trials to get their products approved) whereas they can probably get away with biogeneric because they may not need trials.
that would be a huge differenciation.
we will see
When a company has a garbage drug but raises over a hundred million dollars a year they get analyst coverage
Bank of America Merrill Lynch Steve Byrne, CFA
Barclays Capital Jim Birchenough, M.D.
BMO Capital Markets Jason Zhang, Ph.D.
Canaccord Adams Adam Cutler
Cowen & Co. Phil Nadeau, Ph.D.
CRT Capital Group Leah A. Hartman
Davenport & Company Joel M. Ray, CFA
Hapoalim Securities Jon LeCroy, M.D.
Jefferies & Company Thomas Wei
JMP Securities Jason N. Butler, Ph.D.
JPMorgan Cory Kasimov
Lazard Capital Markets William Tanner, Ph.D.
Leerink Swann and Co. Steve Y. Yoo
Needham & Company Alan Carr
Oppenheimer & Co. Bret Holley, Ph.D.
Piper Jaffray Edward A. Tenthoff
Rodman and Renshaw Elemer Piros, Ph.D.
Summer Street Research Partners Carol Werther
UBS Jeff Elliott, Ph.D.
Wells Fargo Securities Aaron S. Reames
pretty unbelieveble
I think Geneara's obesity drug is going to be a homerun
Gene Therapy Appears to Help Patient With Anemia
By Maggie Fox, Health and Science Editor
Reuters
WASHINGTON
A patient with a rare genetic form of anemia is getting by without blood transfusions after experimental gene therapy, French and U.S. researchers reported on Wednesday.
The case, reported in the journal Nature, is a rare success for the troubled field of gene therapy, although the researchers and other experts said it still needs fine-tuning.
The patient has beta-thalassemia, a group of conditions caused by genetic defects in the production of hemoglobin. Researchers used gene therapy to fix the faulty gene responsible for the condition in some of the patient's own bone marrow stem cells, and re-infused them.
Nearly three years later, the patient has been healthy without the need for the usual blood transfusions, the team at Brigham & Women's Hospital and Harvard Medical School in Boston and University of Paris reported.
"For the first time, a patient with severe beta-thalassemia is living without the need for transfusions over a sustained period of time," Dr. Marina Cavazzana-Calvo of the University of Paris and the Necker Hospital said in a statement.
Gene therapy is a promising but difficult approach to medicine, based on the idea that faulty genes that underlie many diseases can be corrected.
Researchers have struggled to find good ways to get the repaired genes back into the body safely. Viruses are usually used to carry the genes into the body and they can themselves be dangerous.
One experiment cured two French boys with a rare immune disorder but gave them leukemia in 2002, and an Arizona teenager died in a 1999 gene therapy experiment, evidently from an immune system overreaction to the virus that was used.
But researchers at Children's Hospital of Philadelphia have used gene therapy to help improve worsening eyesight in 12 patients with a rare blinding disease called Leber congenital amaurosis, or LCA.
HELP FROM AIDS VIRUSFor the beta-thalassemia study, the researchers used a modified AIDS virus to repair the DNA in the patient's bone marrow stem cells.
A small, Massachusetts-based biotech company called Bluebird Bio is developing the approach as a product called LentiGlobin.
In an unusual step, they first destroyed the patient's bone marrow -- a method used before giving patients bone marrow transplants for cancer and other conditions.
The patient got the first transfusion in 2007, when he was 18, and again in 2008. "At present, approximately 3 years post-transplantation, the biological and clinical evolution is remarkable, and the patient's quality of life is good," the researchers wrote in Nature.
Dr. Derek Persons of St. Jude Children's Research Hospital in Memphis, Tennessee, said destroying the patient's bone marrow appears to have been crucial to making the treatment work.
"Although, three years on, he remains mildly anemic and shows signs of compensatory expansion of red-blood cell precursors in his bone marrow, absence of the need for blood transfusions means that this case can be viewed as a clinical success," Parsons, who was not involved in the research, wrote in a commentary.
Beta-thalassemia is diagnosed in about 60,000 children globally each year.
It is caused by defects in a gene involved in making hemoglobin, the component that carries iron in red blood cells, and patients generally need monthly blood transfusions to treat severe anemia and then special therapy called chelation to clear out the extra iron buildup caused by the transfusions.
Copyright 2010 Reuters News Service. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
Copyright © 2010 ABC News Internet Ventures
-FDA mulls label changes for some osteoporosis drugs
3:28pm EDT
* FDA advises doctors: be aware of unusual femur fractures
* Unclear if bisphosphonates are cause of fractures
* Reviewing all long-term data on bisphosphonates
* Drug class includes Fosamax, Boniva, Reclast, Actonel (Updates with comment from Roche, background, adds byline)
By Toni Clarke
BOSTON, Sept 14 (Reuters) - The U.S. Food and Drug Administration said on Tuesday it may require a class of osteoporosis drugs known as bisphosphonates to carry new information related to unusual femur fractures.
The bisphosphonate class includes Fosamax, made by Merck & Co Inc (MRK.N: Quote, Profile, Research, Stock Buzz) and sold generically under the chemical name alendronate; Roche Holding AG's (ROG.VX: Quote, Profile, Research, Stock Buzz) Boniva; Novartis AG's (NOVN.VX: Quote, Profile, Research, Stock Buzz) Reclast; and Warner Chilcott Plc's (WCRX.O: Quote, Profile, Research, Stock Buzz) Actonel.
"Although it is not clear if bisphonsphonates are the cause, these unusual femur fractures have been identified in patients taking these drugs," the FDA said. "FDA recommends that healthcare professionals be aware of the possible risk of unusual femur fractures in patients taking bisphosphonates."
The FDA's statement follows the release of a report from the American Society of Bone and Mineral Research (ASBMR), which the agency said provides "important perspectives" on the potential association between long-term treatment with bisphosphonates and a rare but serious type of fracture of the thigh bone, or femur.
Ronald Rogers, a spokesman for Merck, said that in clinical studies involving more than 28,000 patients, Fosamax has not been associated with increased fracture risk at any skeletal site.
"Since bisphosphonates were first approved for the treatment of osteoporosis in 1995, millions of people, primarily women, have been treated," he said in an email.
Terence Hurley, a spokesman for Roche, said no causal link has been established for Boniva and atypical fractures.
Officials at Novartis and Warner Chilcott were not immediately available for a comment.
The FDA said the report from the ASBMR includes a case definition that describes the atypical features of these unusual femur fractures, which it believes will help in identifying cases and reporting on them.
The FDA said recommendations from the ASBMR's Task Force include changes to product labels alerting healthcare professionals and patients to the possibility of unusual femur fractures with long-term use of the drugs. The agency said it is thoroughly reviewing all long-term data available on the products, as well as safety reports, and is considering label revisions.
Bisphosphonates, which have been on the market for roughly a decade, have raised safety concerns in the past, including heart risks.
A report in the New England Journal of Medicine in 2007 had raised concerns about serious atrial fibrillation, a type of abnormal heart beat. In 2008, the FDA said the drugs showed no overall risk of heart problems.
But it requested information from all bisphosphonate drug makers related to atypical femur fractures. In March, the agency said it was working with the ASBMR to help assess the risk of such fractures.
Now that the report is final, the agency will decide on its next step.
The FDAs statement was made on its website at: here. (Additional reporting by Susan Heavey and Ransdell Pierson; editing by Andre Grenon, Gary Hill)
OLGX Announces Solid Ex-U.S. Licensing Agreement with Servier –
This is from Matt Kaplan from Ladenburg
We Reiterate Our Buy Rating and $4 Price Target on OLGX.
Highlights
Osteologix announces signing of a licensing agreement with Servier for ex-U.S.
rights to NBS-101. Based on the terms of the agreement OLGX will receive: up to
12 million Euros ($15.6 million) in upfront and milestone payments; 30 million Euros
($39 million) of guaranteed minimum royalty payments based on mid- to low- single
digit royalties on product sales. Furthermore, OLGX will receive additional milestone
payments and royalties based on development milestones and sales in Japan.
Importantly, Servier will be responsible for all ex-U.S. expenses associated with the
development, regulatory approval processes and commercialization of the product.
We are impressed with the terms of the deal and by the company, Servier, OLGX
established the agreement with. As the innovator of Strontium for the treatment of
osteoporosis with the introduction of Protelos to ex-U.S. markets, we believe Servier
represent the ideal partner for NBS-101. Additionally, we believe NBS-101
represents an important asset for Servier as it will now allow them to maintain their
Strontium franchise well beyond the anticipated expiration of the Protelos patents
expected in the late 2014 to August 2015 time frame. In our view, this licensing deal
indicates Servier has a high level of commitment to Strontium as a treatment for
osteoporosis. Consequently, we feel the Servier deal reduces the risk NBS-101 will
fail to become a significant product for the treatment of osteoporosis in ex-U.S.
markets.
? Osteologix maintains ownership of U.S. rights to NBS-101. OLGX met with the
FDA in 2009 and based on these discussions, the Company needs to complete an
additional Phase II study examining bone mineral density (BMD) as well as a single
Phase III study to file an NDA in the U.S., which will likely take two years to complete.
If the results of the Phase II BMD clinical trials are positive, we expect OLGX to work
in collaboration with a larger pharmaceutical company to execute a Phase III program
in an effort to seek approval for NBS-101 in the U.S.
? Servier deal reduces risk of dilution. Prior to the Servier transaction we believed
OLGX had cash to fund operations to 4Q 2010. The $15.6 million in upfront and
milestone payments strengthen OLGX’s cash position. However, depending on the
breakdown and timing of the upfront and milestone payments, OLGX may need to
further augment the Company’s cash position to continue U.S. development efforts to
maximize the value of their asset in the U.S. .
? We reiterate our BUY rating. We do not believe OLGX’s current market cap of
$14 million captures the value of either the Servier ex-U.S. partnership or the
potential for the product in the U.S. We continue to believe Osteologix represents a
unique opportunity to invest in a late-stage/Phase III-ready compound which
addresses a large indication at a very attractive valuation. We view the Servier ex-
U.S. partnership agreement as a major value driver for the Company as it
significantly de-risks the product’s potential in these markets. Further, we do not
believe the value of the strong intellectual property surrounding NBS-101 in U.S. is
reflected in the current stock price.
Nordic Biotech K/S remains the largest shareholder. Nordic, a private venture capital firm based in Denmark,
owns approximately 68% OLGX common stock. Additionally, as long as Nordic owns over 20% of the company,
OLGX is only allowed 7 board members, with 3 of the 7 members appointed by Nordic. As the majority
shareholder, all transactions (including, but not limited to: M&A, going private, financings, and other significant
transactions) are subject to Nordic approval (in other words, Nordic has strong veto power).
Protelos (strontium ranelate) sales remain strong and continue to grow. Strontium ranelate, a similar
osteoperosis drug marketed by privately held Servier continues to show sales growth in Europe being very strong
in new markets, with moderated growth in certain countries. As a reminder, Protelos is not approved in the U.S.
Protelos ex-U.S. sales were $205 million in 2007, $248 million in 2008, and $290 million in 2009. The growth in the
revenue from 2007 supports our belief physicians view the potential for anaphylactic reactions essentially as a nonissue
which must be monitored for and is addressable by discontinuation of therapy. The rate of observed
anaphylactic reactions is lower than that seen with several other approved drugs. We continue to believe the
occurrence of DRESS (Drug Rash with Eosinophilia and Systemic Symptoms) associated with strontium ranelate
may be associated with the synthetic ranelate salt (which is not used in any other therapeutic agent). Investors
should keep in mind NBS-101 is strontium malonate which is a naturally occurring salt used in other approved
drugs and has demonstrated an improved efficacy and safety profile over Protelos in clinical trials.
Osteologix appears to have a strong patent estate for NBS-101. NBS-101 fully issued patents in the
European Union, the U.S., Australia and Japan provide coverage through 2024 (with extensions possible
through 2026). The USPTO issued a U.S. Patent titled “Water-Soluable Strontium Salts for Use in the Treatment of
Cartilage and/or Bone Conditions” with specific claims covering NBS-101 (strontium malonate) for the treatment of
osteoporosis and related bone conditions. We believe the U.S. patent covering NBS-101 through 2024 solidifies the
U.S. IP position and will allow the Company to consummate a partnership for the product in 2010. Additionally, OLGX
won its First Opposition in the E.U. when the European Patent Office upheld the Company’s NBS-101 patent in Europe in
April 2009, which we believe validates the findings of enhanced bioavailability (BA) with malonate salt were not “obvious”
and increases the potential value of the product for a partnership in Europe. We believe the upholding of the patent by
the EPO represented an important victory for Osteologix for a number of reasons including: First, it validated the
potential value of the product for a partnership in Europe. Second, it validated the findings of enhanced
bioavailability (BA) with the malonate salt were not “obvious”. In other words, the EPO found, since the malonate
salt is not widely used or a well know salt in drug formulation and it has not already been shown to demonstrate an
increase in BA for a wide range of compounds, it was not “obvious” malonate would increase BA. Third, the EPO
agreed malonate salt definitely increased BA. Importantly, OLGX also filed IP surrounding combination use of
strontium with other therapies, and have applications related to manufacturing, osteonecrosis, and arthritis.
100 million ED visits could be handled elsewhere
Its hard to believe that old farts wanting viagra would tie up hospitals like this
By Sandra Yin Comment | Forward | Twitter | Facebook | LinkedIn
An estimated 17 to 27 percent of all emergency department visits for non-emergency care--or, about 104 million visits--could have occurred elsewhere, according to an article recently published in Health Affairs. Such a change could result in savings of roughly $4.4 billion each year.
The more conservative estimate represents the share of all ED visits that could take place during the limited hours when retail clinics and urgent care centers are open. The larger estimates equals the total share of ED visits that could be managed at the same sites.
The main conditions that could be treated at alternative sites are minor acute illness, strains and fractures.
For the study, researchers compared the share of specific diagnoses treated at retail clinics, urgent care centers and emergency departments. Among the findings, 61 percent of retail clinic visits were for upper respiratory infections compared with 33 percent of urgent care center visits and 10 percent of ED visits. Strains and fractures were seen slightly more often at urgent care centers than EDs (22 percent of visits vs. 19 percent).
The analysis was based on the 2006 National Hospital Ambulatory Medicare Care Survey, a nationally representative survey.
Diverting the patients who were headed to EDs to urgent care sites or retail clinics could cut the time spent waiting to see a clinician, the authors write. Diversion could also lead to savings of $279 to $460 for retail clinic visits and $228 to $414 for urgent care center visits, according to earlier research.
why would Goddard, the ceo of osip, care if eyetech was bought out by pfizer
Somaxon seeks to wake up Silenor launch with P&G alarm clock
Source EP Vantage
http://www.evaluatepharma.com/Universal/View.aspx?type=Story&id=222099
This is a good article. If the drug is a failure somx gets hurt by the fact that they didn't get an upfront payment with a 20 percent royalty, but the company wouldn't have been worth anything anyway. If the drug is successful the company will keep 85 percent of the economics and will be very successful. At this price there isn't much priced in for a successful launch so it is a good risk/reward, I believe
Company Somaxon Pharmaceuticals
Related: Procter & Gamble
Date August 26, 2010
Somaxon Pharmaceuticals has finally delivered a deal on Silenor, and it is an unusual one. For a fixed fee and royalties, Somaxon has hired the OTC sales force of consumer products giant Procter & Gamble for a September launch of the insomnia medication, a deal that leaves the smaller partner with nearly all of the potential revenue but also nearly all of the risk.
The deal points up the need for brand awareness in the highly competitive and genericised insomnia space as Somaxon executives focused on the reach P&G’s sales force will provide with primary care physicians, pharmacies and managed care plans. Somaxon shares jumped 29% higher Wednesday to reach $4.34 on news that a long-sought deal had finally be secured, a healthy gain but still less than half the $9.21 they closed at on March 18, following FDA approval of Silenor (Somaxon wins approval for Silenor and hunt for partner begins, March 19, 2010). Investors clearly remain sceptical about the commercial potential of this product, even with P&G on board.
Somaxon now plans a September launch of Silenor, a low-dose version of the antidepressant doxepin hydrochloride. The California company’s pitch on the sleeping pill is that it is not a controlled substance like so many other sleep-assistance drugs and therefore carries reduced risk of dependence, addiction or withdrawal.
Launch sequence commencing
On word of the deal, analysts from Jefferies increased their 12-month price target on Somaxon to $10 from $9 along with their 2015 sales estimates for Silenor to $322m from $257m.
After spending $108m in R&D in the last six years to develop Silenor, Somaxon’s bank accounts were nearly empty following FDA approval, with just $4m in cash left. With its stock riding high in March, the firm raised a timely $52.8m by selling 6.9 million shares at $8.25, a discount from the $9.51 they closed on March 25, the day the offer was priced.
Although the buyers of that offer are probably smarting at today's share price, the new cash did allow Somaxon to hire the French contract sales organisation Publicis Touchpoint Services to give it 110 dedicated sales representatives to target the top 30% of insomnia drug prescribers.
The partnership with P&G nearly doubles that sales force to 215, with the reps from the Cincinnati firm focusing on their strengths in primary care physicians along with pharmacies and managed care plans, Somaxon executives said in an investor conference call Wednesday. Since Warner Chilcott bought P&G's prescription drugs unit the reps have largely been pitching the few remaining P&G OTC products, including Prilosec OTC.
With intense competition in the insomnia market, ensuring that Silenor is side-by-side with Lunesta and Ambien on pharmacy shelves will be critical to Silenor’s commercial success, as the launch could run aground if patients, once prescribed, cannot get the drug.
Likewise, getting on managed-care formularies will also be important, although Jeffrey Raser, Somaxon’s senior vice president and chief commercial officer, said Silenor will be priced at below the average of the three branded products on the market.
No check collector
Under the deal, Somaxon executives estimate that they will pay P&G no more than 15% of net sales. Each party will be responsible for paying for their own sales force, and in a sign that the risk is still firmly in Somaxon’s hands, it will also pay for all other commercialisation costs.
Presumably, all costs related to development of line extensions or OTC formulations would belong to Somaxon also. But significantly, P&G would have right of first refusal on an OTC product, where the consumer products giant retains a significant presence. Somaxon executives highlighted this part of the deal as a wise lifecycle management strategy.
In its unusual deal with P&G, Somaxon has structured a partnership that will allow it to be more than just a check collector. However, should the team stumble out of the blocks, it looks like only Somaxon will be the partner to take a fall.
Somaxon seeks to wake up Silenor launch with P&G alarm clock
Source EP Vantage
http://www.evaluatepharma.com/Universal/View.aspx?type=Story&id=222099
This is a good article. If the drug is a failure somx gets hurt by the fact that they didn't get an upfront payment with a 20 percent royalty, but the company wouldn't have been worth anything anyway. If the drug is successful the company will keep 85 percent of the economics and will be very successful. At this price there isn't much priced in for a successful launch so it is a good risk/reward, I believe
Company Somaxon Pharmaceuticals
Related: Procter & Gamble
Date August 26, 2010
Somaxon Pharmaceuticals has finally delivered a deal on Silenor, and it is an unusual one. For a fixed fee and royalties, Somaxon has hired the OTC sales force of consumer products giant Procter & Gamble for a September launch of the insomnia medication, a deal that leaves the smaller partner with nearly all of the potential revenue but also nearly all of the risk.
The deal points up the need for brand awareness in the highly competitive and genericised insomnia space as Somaxon executives focused on the reach P&G’s sales force will provide with primary care physicians, pharmacies and managed care plans. Somaxon shares jumped 29% higher Wednesday to reach $4.34 on news that a long-sought deal had finally be secured, a healthy gain but still less than half the $9.21 they closed at on March 18, following FDA approval of Silenor (Somaxon wins approval for Silenor and hunt for partner begins, March 19, 2010). Investors clearly remain sceptical about the commercial potential of this product, even with P&G on board.
Somaxon now plans a September launch of Silenor, a low-dose version of the antidepressant doxepin hydrochloride. The California company’s pitch on the sleeping pill is that it is not a controlled substance like so many other sleep-assistance drugs and therefore carries reduced risk of dependence, addiction or withdrawal.
Launch sequence commencing
On word of the deal, analysts from Jefferies increased their 12-month price target on Somaxon to $10 from $9 along with their 2015 sales estimates for Silenor to $322m from $257m.
After spending $108m in R&D in the last six years to develop Silenor, Somaxon’s bank accounts were nearly empty following FDA approval, with just $4m in cash left. With its stock riding high in March, the firm raised a timely $52.8m by selling 6.9 million shares at $8.25, a discount from the $9.51 they closed on March 25, the day the offer was priced.
Although the buyers of that offer are probably smarting at today's share price, the new cash did allow Somaxon to hire the French contract sales organisation Publicis Touchpoint Services to give it 110 dedicated sales representatives to target the top 30% of insomnia drug prescribers.
The partnership with P&G nearly doubles that sales force to 215, with the reps from the Cincinnati firm focusing on their strengths in primary care physicians along with pharmacies and managed care plans, Somaxon executives said in an investor conference call Wednesday. Since Warner Chilcott bought P&G's prescription drugs unit the reps have largely been pitching the few remaining P&G OTC products, including Prilosec OTC.
With intense competition in the insomnia market, ensuring that Silenor is side-by-side with Lunesta and Ambien on pharmacy shelves will be critical to Silenor’s commercial success, as the launch could run aground if patients, once prescribed, cannot get the drug.
Likewise, getting on managed-care formularies will also be important, although Jeffrey Raser, Somaxon’s senior vice president and chief commercial officer, said Silenor will be priced at below the average of the three branded products on the market.
No check collector
Under the deal, Somaxon executives estimate that they will pay P&G no more than 15% of net sales. Each party will be responsible for paying for their own sales force, and in a sign that the risk is still firmly in Somaxon’s hands, it will also pay for all other commercialisation costs.
Presumably, all costs related to development of line extensions or OTC formulations would belong to Somaxon also. But significantly, P&G would have right of first refusal on an OTC product, where the consumer products giant retains a significant presence. Somaxon executives highlighted this part of the deal as a wise lifecycle management strategy.
In its unusual deal with P&G, Somaxon has structured a partnership that will allow it to be more than just a check collector. However, should the team stumble out of the blocks, it looks like only Somaxon will be the partner to take a fall.
Somaxon seeks to wake up Silenor launch with P&G alarm clock
Source EP Vantage
http://www.evaluatepharma.com/Universal/View.aspx?type=Story&id=222099
This is a good article. If the drug is a failure somx gets hurt by the fact that they didn't get an upfront payment with a 20 percent royalty, but the company wouldn't have been worth anything anyway. If the drug is successful the company will keep 85 percent of the economics and will be very successful. At this price there isn't much priced in for a successful launch so it is a good risk/reward, I believe
Company Somaxon Pharmaceuticals
Related: Procter & Gamble
Date August 26, 2010
Somaxon Pharmaceuticals has finally delivered a deal on Silenor, and it is an unusual one. For a fixed fee and royalties, Somaxon has hired the OTC sales force of consumer products giant Procter & Gamble for a September launch of the insomnia medication, a deal that leaves the smaller partner with nearly all of the potential revenue but also nearly all of the risk.
The deal points up the need for brand awareness in the highly competitive and genericised insomnia space as Somaxon executives focused on the reach P&G’s sales force will provide with primary care physicians, pharmacies and managed care plans. Somaxon shares jumped 29% higher Wednesday to reach $4.34 on news that a long-sought deal had finally be secured, a healthy gain but still less than half the $9.21 they closed at on March 18, following FDA approval of Silenor (Somaxon wins approval for Silenor and hunt for partner begins, March 19, 2010). Investors clearly remain sceptical about the commercial potential of this product, even with P&G on board.
Somaxon now plans a September launch of Silenor, a low-dose version of the antidepressant doxepin hydrochloride. The California company’s pitch on the sleeping pill is that it is not a controlled substance like so many other sleep-assistance drugs and therefore carries reduced risk of dependence, addiction or withdrawal.
Launch sequence commencing
On word of the deal, analysts from Jefferies increased their 12-month price target on Somaxon to $10 from $9 along with their 2015 sales estimates for Silenor to $322m from $257m.
After spending $108m in R&D in the last six years to develop Silenor, Somaxon’s bank accounts were nearly empty following FDA approval, with just $4m in cash left. With its stock riding high in March, the firm raised a timely $52.8m by selling 6.9 million shares at $8.25, a discount from the $9.51 they closed on March 25, the day the offer was priced.
Although the buyers of that offer are probably smarting at today's share price, the new cash did allow Somaxon to hire the French contract sales organisation Publicis Touchpoint Services to give it 110 dedicated sales representatives to target the top 30% of insomnia drug prescribers.
The partnership with P&G nearly doubles that sales force to 215, with the reps from the Cincinnati firm focusing on their strengths in primary care physicians along with pharmacies and managed care plans, Somaxon executives said in an investor conference call Wednesday. Since Warner Chilcott bought P&G's prescription drugs unit the reps have largely been pitching the few remaining P&G OTC products, including Prilosec OTC.
With intense competition in the insomnia market, ensuring that Silenor is side-by-side with Lunesta and Ambien on pharmacy shelves will be critical to Silenor’s commercial success, as the launch could run aground if patients, once prescribed, cannot get the drug.
Likewise, getting on managed-care formularies will also be important, although Jeffrey Raser, Somaxon’s senior vice president and chief commercial officer, said Silenor will be priced at below the average of the three branded products on the market.
No check collector
Under the deal, Somaxon executives estimate that they will pay P&G no more than 15% of net sales. Each party will be responsible for paying for their own sales force, and in a sign that the risk is still firmly in Somaxon’s hands, it will also pay for all other commercialisation costs.
Presumably, all costs related to development of line extensions or OTC formulations would belong to Somaxon also. But significantly, P&G would have right of first refusal on an OTC product, where the consumer products giant retains a significant presence. Somaxon executives highlighted this part of the deal as a wise lifecycle management strategy.
In its unusual deal with P&G, Somaxon has structured a partnership that will allow it to be more than just a check collector. However, should the team stumble out of the blocks, it looks like only Somaxon will be the partner to take a fall.
The Mayo Clinic just sent out Jewish New Years cards. I am glad I wasn't on their list.
Mayo tells more than 3,000 patients of possible Hepatitis C infection
By Alicia Caramenico
The Mayo Clinic in Jacksonville, Fla., will send out letters to 3,209 patients who may have been infected with Hepatitis C by a former employee, said the hospital's CEO at a news conference Thursday.
Mayo fired the former employee--later identified by the Jacksonville Sheriff's Office as Steven Beumel--last week for stealing pain medication intended for patients and putting them at risk. Patients who were in contact with Beumel since his hiring in 2004 will receive regular and certified letters stating that they may have been exposed to Hepatitis C and explaining how to get tested for infection, Mayo Clinic Florida CEO Dr. William Rupp told reporters.
The hospital system will offer three free-of-charge testing options for the at-risk patients. This is nice of them
They can be tested on Mayo premises, at their own residences, or at other testing sites.
Mayo also will ask those patients to take HIV and Hepatitis B tests as protocol, even though Beumel has tested negative for the two viruses.
Patients who don't respond to the letters will receive a follow-up phone call, noted Rupp. "Mayo Clinic will do whatever is necessary to support the needs of our patients."
According to the CEO, there have been five cases where patients arrived at Mayo Clinic not having hepatitis C, but were infected with it when they left, notes First Coast News. Three of those cases have been linked to Beumel.
The actions of the former radiology technologist are now under comprehensive criminal investigation, the hospital said in statement. He has been charged with fraudulently obtaining a controlled substance. Mayo has been "cooperating completely" with authorities, noted Rupp.
The GM-CSF situation that Dr. Garren had speculated about was never mentioned.
specifically it wasn't mentioned, but it was answered when they said they have already manufactured the phase 3 material.
Dew is working on it. He is a programmer, you know
what is your email address
I would like to send you info on a company that may be of interest to you. they have some data coming out in september and people aren't following it
UPDATE 1-Alimera Sciences eye therapy gets FDA priority review
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Alimera Sciences Inc
ALIM.O
$8.10
-0.07-0.86%12:00am EDT
Mon Aug 30, 2010 6:15pm EDT
* Alimera expects FDA response in Q4
* Shares up 5 pct in extended trade
Aug 30 (Reuters) - Alimera Sciences Inc (ALIM.O) said its experimental eye drug to treat retinal swelling got priority review status from U.S. health regulators, sending its shares up 5 percent.
The late-stage drug, Iluvien, is being developed for the treatment of diabetic macular edema -- the most prevalent cause of moderate vision loss in patients with diabetes.
The Alpharetta, Georgia-based company said with the priority review status, it could receive a response from the U.S. Food and Drug Administration in the fourth quarter regarding its new drug application.
Priority review status implies the agency would aim to complete its analysis of the application within six months, rather than within the standard 10-month review period.
The FDA reserves priority review designation for drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists.
Alimera said it expects to file for Canadian marketing approval in September.
The company's shares, which debuted on Nasdaq in April, were up 5 percent at $8.50 after the bell. They closed at $8.10 Monday. (Reporting by Krishnakali S
boy
that bernstein does research like no other healthcare analysts that I have ever seen.
Has their chatter affected any other companies in that location or is Momenta in the eye of the storm
MNTA: Perhaps it was just today's general "market malaise" but whether one is a trader or an investor a 9% drop in 3 hours with no apparent news is a tad troubling.
not if you are waiting to buy
Cleveland BioLabs well positioned as a government award candidate Written by M.E.Garza
Friday, 27 August 2010 02:45
It doesn't appear that Garza has changed his writing technique.
it certainly isn't DARPA that he is hearing this from. It may be his meds
We are hearing some very loud and credible chatter that long-awaited good news may be coming for Cleveland BioLabs (NasdaqCM: CBLI), the company that engages in the discovery, development, and commercialization of products for human protection from radiation.
In the first quarter of this year, the company submitted additional paperwork about Protectan CBLB502 (a radioprotectant molecule with multiple medical and defense applications for reducing injury from acute stresses, such as radiation and chemotherapy) to a potential customer- the U.S. Government's Department of Defense. Many on Wall Street believe that the company has the best chance of not only getting significant funding from the DoD, but also a potentially bigger order from the Biomedical Advanced Research and Development Authority (BARDA) who would acquire doses of the drug for the Strategic National Stockpile.
Share prices began to see some appreciation yesterday afternoon after we broke this news to our subscribers, but could rise significantly higher in anticipation of news and subsequent developments involving these government contracts.
We reached out to the CEO & President of Cleveland BioLabs, Michael Fonstein for his thoughts on these and other issues. The following is an unedited transcript of our Q&A:
BioMedReports: Have there been any important business developments for CBLI since we last spoke to you? If so, what are they?
Cleveland BioLabs' (CBLI) CBLB502 Granted Fast Track Status
BARDA Exercises Options on Cleveland BioLabs $15.6 Million Contract to Develop Radiation Countermeasure
Cleveland BioLabs Submits Response to Department of Defense Request for Proposal
Cure for Radiation Sickness Found?
Marked Up Technical Analysis Chart (Subscribers Only)
CBLI's CEO & President, Michael Fonstein: There have been a few notable advances at CBLI over the past 9 months or so, since we last spoke.
First, is a highly significant and more recent development – the FDA granted fast track status to CBLB502 for Reducing the Risk of Death Following Total Body Irradiation During or After a Radiation Disaster. This achievement is important from a development and potential licensure standpoint, as it provides the opportunity for expedited review of a BLA for CBLB502 and allows for a rolling submission of data (which we have planned). In addition, the label attached to this application is incredibly broad and outlines a clear path to move forward with final development steps.
While we are on the topic of development, we have made significant progress in moving CBLB502 forward for the Acute Radiation Syndrome indication over the past few months. In May, we completed dosing of our second healthy volunteer safety trail for CBLB502 in 100 people. We are still finalizing biomarker data analysis, etc., but on an observational level, we were pleased to see consistency in overall side effect profile, as in our first, 50 person trial. The primary objectives of this study were to gather additional data on safety, pharmacokinetics, and cytokine biomarkers in a larger and broader subject population in order to finalize an appropriate dose to take forward for the large, definitive human volunteer safety study.
The other major development I’d like to highlight is probably the most significant in terms of business. In February, we submitted a response to the Department of Defense’s Request For Proposal (RFP) for development and acquisition of a radiation countermeasure. We and the rest of the world are currently awaiting the final contract award to be made public, and we believe CBLB502 is well positioned as a potential award candidate. A potential contract to come from this award is likely to have two primary components: a budget for advanced development of a radiation countermeasure through FDA licensure, and conditional commitments to purchase up to 37,500 doses, upon licensure. While we wouldn’t expect the total dollar value of such a contract to exceed a few tens of millions, it would be highly significant in terms of validation, as the DoD is globally recognized as a leader in radiation research. It also potentially sets up a successful program for potential purchases from the even bigger potential customer in the United States, the Biomedical Advanced Research and Development Authority (BARDA) of the Department of Health and Human Services (HHS), whose mandate is to develop and acquire countermeasures for the Strategic National Stockpile. According to publicly released reports from January, such acquisitions of radiation countermeasures are slated for 2011. Based on similar types of acquisitions for other countermeasures that have taken place over the last 2 years, one would expect this type of contract to be valued somewhere in the range of $100-300 million. Then there are other potential foreign buyers, such as Israel, or other countries with a seemingly imminent nuclear threat. We’ll see what happens, but the next year or so should prove very exciting for CBLI.
BioMedReports: What are the next important milestones for the company? How soon do you expect to reach them?
CBLI's CEO & President, Michael Fonstein: Our primary focus for the next 12 months or so is execution of the remaining development steps for CBLB502 and submission of a BLA to the FDA, as well as securing any potential contracts for both development funds and potential purchases from the US and/or other friendly foreign governments.
As far as CBLB502 development goes, we have a few clear milestones to achieve, which are of course supported by dozens of daily tasks. There are three primary aspects to development under the Animal Efficacy Rule, by which CBLB502 is being developed. One is of course manufacturing and controls, another is animal efficacy, and the third is human safety and biomarkers. We have a few critical final steps among each of these areas to complete in order to finish a BLA filing. We intend to run our final consistency batches of cGMP product in the coming months and begin our BLA submission with the manufacturing portion of the package in early 2011. For the animal efficacy portion, we have done studies in more than 800 non-human primates to date, looking at multiple aspects of potential efficacy, but we will still need to perform a pivotal animal efficacy study under GLP conditions, with supportive care measures. We are currently conducting supporting studies and preparing to finalize a protocol for this study with the FDA towards the end of the year. We would expect the pivotal animal study to be run in 2011. Finally, we are finishing analysis on the second human safety study we just completed, and will need to make a public top line summary announcement regarding the outcome in the coming weeks or month. We will still need to nail down a final protocol with the FDA for a definitive human safety study in a larger group of volunteers; which is also slated to happen towards the end of this year, with the study planned for 2011. Overall, our goal is to finalize all data and submission for our BLA filing in 2011.
On the contract side, there are some potentially closer milestones, including the DoD contract I mentioned earlier, as well as some additional development contracts we have applied for with other federal agencies. A final award decision on the DoD contract is literally expected any day or week now, and there may some other nice chunks of development funds from other agencies coming before year end. Any of these would certainly demonstrate levels of commitment to the CBLB502 program.
BioMedReports: Financially, how is the company doing? Are you looking to raise money in the near future?
CBLI's CEO & President, Michael Fonstein: Based on cash and receivables on our balance sheet at June 30, 2010, we have $7.2 million in CBLI corporate cash, with a burn rate of only $200,000 per month, and another $3.2 million in cash for our joint venture, Incuron, which is developing our Curaxin family of anti-cancer molecules. The burn on the Incuron money is also approximately $200,000 per month at this point. Remember, any government grants or development contracts are not reflected on our balance sheet. These are recognized as revenues as we draw upon them to pay receivables. Also remember that government funded work is 100% cost reimbursed, plus a small allowed overhead. We currently have about $5.2 million in funds remaining under current government contracts to develop CBLB502, and have applied for additional monies.
Given this position, we are feeling pretty good and have no overwhelming need to raise money, unless there are opportunistic circumstances dictated by a nice movement in the share price on the back of some potential contracts or other benchmarks in the coming months.
CBLB502 is funded for acute radiation syndrome, as well as for its first medical trial for supportive care in head and neck cancer patients, which we hope will open enrollment at the end of this year. Our Curaxin program is also funded under the joint venture, which has milestone commitments of up to $18 million over the next 2.5 to 3 years. What is not currently funded are additional medical trials for CBLB502 for supportive care in cancer treatment, or development of CBLB612, our hematopoietic stem cell inducer and mobilizer. So depending on what happens in the coming months, it may be something we look at.
BioMedReports: From a science and technology perspective, have you continued work in the lab on the Protectans and Curaxins? Any new developments there?
CBLI's CEO & President, Michael Fonstein: We have certainly been active on both these fronts. I’ve already spoken a bit about CBLB502’s development progress towards potential licensure for Acute Radiation Syndrome. While this is more clinical research than lab work, I want to just remind folks that we are in fact also prepping for our first medical trial of CBLB502 as a supportive care agent in head and neck cancer patients undergoing radio and chemotherapy. We have been working closely with Roswell Park Cancer Institute to finalize the protocol, and update our investigational brochure for additional data gained in healthy volunteer trials. The head and neck study is to be a Phase I/II trial, looking to determine safety and tolerability of CBLB502 in cancer patients, as well as looking for trends of efficacy in reduction of severity and occurrence of side effects of therapy, such as mucositis.
As far as mechanistic studies of CBLB502, we continue to find new twists that add additional insights into CBLB502’s mechanism of action, which is always helpful as you progress in development.
CBLI's CEO & President, Michael Fonstein: On the Curaxin front, we’ve published a few peer review papers on mechanistic discoveries of our first generation compound, CBLC102 or quinacrine. One published study in Cell Cycle form December 2009, examined the ability of our prototype Curaxin to inhibit heat shock response in tumors, making them selectively susceptible to heat shock inhibitors, especially if applied in combination with certain cancer therapies provoking proteotoxic stress. This opens a whole new avenue of potential treatment options that may broaden the spectrum of tumors responding to therapy by cutting off an escape mechanism.
Another study, published in Oncogene in January of 2009, indicated that treatment of cancer cells with CBLC102 resulted in the inhibition of an additional molecular pathway (PI3K/Akt/mTOR) that is considered to be a highly relevant anticancer treatment target. Simultaneous targeting of several molecular pathways is a unique property of Curaxins. This attribute makes them very promising drug candidates that may be effective against a wide spectrum of human malignancies and reduces the risk of development of tumor resistance to this class of compounds.
This spring, our Chief Scientific Officer, Dr. Andrei Gudkov presented some exciting early discoveries about our proprietary next generation Curaxin molecules at the American Association for Cancer Research (AACR) Annual Meeting in Washington, DC.
More recently, we announced that a discovery regarding potential antiviral applications for our Curaxin family of molecules was pre-published online in the Journal of Virology. The published study, conducted by Cleveland BioLabs scientists in cooperation with investigators from Roswell Park Cancer Institute and Cleveland State University, examined the ability of CBLC102 and other similar compounds to inhibit a mechanism used by picornaviruses to synthesize their proteins that is essential for their viability. This group of viruses includes important human pathogens such as poliovirus. In particular, Curaxins’ specific interaction with double-stranded RNA effectively blocks synthesis of viral, but not cellular proteins. This study provides proof of principle for prospective extension of Curaxins’ applications from anticancer to antiviral applications.
All of this is still early research, but quite exciting.
BioMedReports: What are your short and long term plans moving forward?
CBLI's CEO & President, Michael Fonstein: Well, our most important short term goal is to complete development of CBLB502 for the acute radiation syndrome indication and to secure contracts for additional development funding and more importantly, procurement. Accomplishing these goals would change the entire essence of CBLI, in terms of valuation and credibility. For the longer-term, we are eager to move our medical applications forward and get some real clinical evidence of efficacy for CBLB502 in supportive care, and for our Curaxin anti-cancer molecules. We have a pipeline of other exciting compounds that we’d like to see brought forth, as well, either under our own hand, or through strategic partnership or collaboration.
BioMedReports: What are your biggest challenges as a company at this point?
CBLI's CEO & President, Michael Fonstein: At this point, the single biggest challenge for us is execution. There are a myriad of tasks involved in prepping for and conducting the remaining development steps for CBLB502 for its first indication in defense. We are focused on keeping things moving forward in a timely fashion, while being ever mindful of the importance of our working relationships with the FDA and the federal agencies partnering with us on the CBLB502 program. We feel that many of the typical drug development risks regarding efficacy or safety have been mitigated at this point, given all of the work done so far, but execution remains paramount. To this end, we have been proactive about augmenting our regulatory affairs and clinical development teams, so we ensure that the right expertise is within our grasp. I think our recent accomplishment of securing fast track designation is evidence that we have the right people in place to guide us through this next critical year.
BioMedReports: Your stock price seems undervalued right now. Would you agree?
CBLI's CEO & President, Michael Fonstein: Like many CEOs of microcap or smallcap biotech companies, I do believe our stock is undervalued. Based on many conversations with investors, I think there are a few reasons in our case. First, is the oddity of CBLI as both a biodefense and medical play. The biodefense space experienced some early, highly publicized failures, which have largely overshadowed the ensuing success stories. Many traditional biotech investors are, as a result of this, leary of the government as a potential customer, and are willing to wait for concrete successes before participating. At the same time, our medical developments are preclinical, and this too carries a discount in the eyes of traditional biotech investors. It is more the retail and smaller healthcare or generalist funds that have been excited by the early prospects for CBLI’s success through biodefense and see the credibility of our science and broad range of applications as long term upside. At the moment, we are certainly only valued for the CBLB502 defense program, and even that is discounted until we are able to deliver concrete purchase commitments. I have to believe that CBLI’s valuation will improve over time, with delivery of milestones and achievement of initial revenues. Only then can we start to gain more credibility regarding our potential to deliver similar achievements on the medical or commercial side of our business.
Disclosure: Long CBLI
I am not an expert on the drug, I just know the data looked great and it is targeted to braf.
just because a drug targets a protein doesn't mean they all work the same way or are effective. this drug worked, I don't know why it worked better than nexavar
are you saying that the two trials that genentech ran were not in indications driven by the hedgehog mutation, even though the drug targets the hedgehog pathway.
why would Genentech pursue this? I thought they loved targeted therapies? They orignially tried Herceptin in all breast cancer patients and then found that it only works in breast cancer that overexpressed her2.
New drug shrinks many advanced melanomas: study
(AFP) – 20 hours ago
This data looks great. I figured I would try to turn it into a biotech board instead of a mnta board.
WASHINGTON — An experimental therapy that targets the protein that feeds certain types of advanced skin cancer has successfully shrunk tumors in up to 80 percent of test patients, a study has indicated.
The orally-administered medication, called PLX4032, "shuts off" tumors by neutralizing a mutated gene called "BRAF" that feeds the cancerous growths.
"We have never seen an 80 percent response rate in melanoma, or in any other solid tumor for that matter, so this is remarkable," said Paul Chapman, senior author of the study and a doctor at Memorial Sloan-Kettering Cancer Center.
"Metastatic melanoma has a devastating prognosis and is one of the top causes of cancer death in young patients," said Keith Flaherty of the Massachusetts General Hospital Cancer Center and a lead author.
"Until now, available therapies were few and unreliable, so these findings can really change the outlook for patients whose tumors are fueled by this mutation."
The study, published in the August 26 edition of the New England Journal of Medicine, grew out of the discovery that BRAF mutations, which occur in roughly half of patients with melanomas, effectively feed and grow the tumors.
PLX4032 blocks the BRAF protein at the cellular level, allowing researchers to test whether starving the melanomas of the gene would shrink patients' tumors.
The research is potentially exciting news for the treatment of patients with advanced skin cancer. Early-stage melanomas can usually be successfully removed surgically, but few treatment options currently exist once the cancer has spread.
The only two drugs currently available on the US market help only between 10 to 20 percent of patients, and for those with advanced melanomas, the prognosis for survival is usually nine months or less.
The study released this week documents the results of two phases of tests involving PLX4032, the first to determine the optimum dose and the second to examine the drug's effectiveness.
During the first phase, 55 patients received gradually escalating doses that allowed scientists to determine that a twice-daily dose of 960 milligrams would be optimal.
The second stage involved 32 patients with BRAF-mutated melanomas, 26 of whom saw their tumors shrink more than 30 percent, including two whose tumors disappeared altogether.
The drug proved capable of shrinking metastatic tumors in the liver, small bowel, bone and thyroid and produced minor side-effects, the researchers said.
"One of the things that make these results truly remarkable is that this drug works so reliably," Flaherty said.
"And patients who have been experiencing symptoms like pain and fatigue begin to feel better within a week of starting treatment, giving them a much better quality of life."
The study acknowledged that many patients eventually developed a resistance to the therapy, with tumor suppression lasting anywhere from three months to two years.
They said they would examine long-term prospects for the therapy, including how it might be combined with other drugs to extend its capabilities against tumors, during a final, phase III trial.
"We don't know if treatment really improves overall survival of melanoma patients," said Chapman.
"That is what we are trying to find out in the ongoing phase III trial. In the future, we hope to combine PLX4032 with other anti-melanoma drugs currently being developed."
Copyright © 2010 AFP. All rights reserved. More »
From Fish Oil to Snake Oil
Sirtris Scandal Aside, Drugs Face No Supplemental Threat
By Trista Morrison
BioWorld Insight Editor
The biotech industry was abuzz last week with the news that Christoph Westphal and Michelle Dipp – who sold resveratrol-based Sirtris Pharmaceuticals Inc. to GlaxoSmithKline plc for $720 million – were exposed for selling resveratrol online.
In a nutshell, resveratrol is a natural substance found in red wine that's thought to positively impact caloric intake, insulin sensitivity and other factors by activating the sirtuin SIRT1. It's widely sold as a nutritional supplement, but Sirtris had developed a formulation with improved bioavailability, known as SRT501, and moved it into clinical trials for diabetes and MELAS syndrome. The biotech also was developing a handful of more potent, small-molecule SIRT1 activators when it was acquired by GSK. (See BioWorld Today, April 24, 2008.)
After the acquisition, Sirtris CEO Westphal and senior director of corporate development Dipp joined the GSK ranks. The two later started a side project together: an anti-aging-focused nonprofit called the Healthy Lifespan Institute. GSK spokeswoman Louise Dunn told BioWorld Insight that the big pharma firm was aware of the nonprofit, but it wasn't aware until Xconomy broke the story earlier this month that the Healthy Lifespan Institute was selling resveratrol online.
The situation spotlights the sometimes-nebulous dividing line between the worlds of nutritional supplements and FDA-approved prescription drugs.
Competitive Considerations
According to the Food, Drug and Cosmetic Act, dietary supplements are labeled as such, intended for ingestion but not a conventional food, and contain a vitamin, mineral, herb/botanical, amino acid and/or other dietary substance, or some concentrate, metabolite, constituent, extract or combination thereof.
A supplement can be an experimental or approved new drug as long as it was marketed as a supplement first, according to the FDA. The only differences between the nutritional version and the drug version are that the latter has a rigorously identified and controlled composition, has been proven safe and effective in clinical trials, and has passed the FDA's regulatory process.
Hence resveratrol could be marketed as a nutritional supplement, while Sirtris's resveratrol formulation, SRT501, could conceivably be marketed as a drug.
But that was not GSK's intent. Dunn said the company is not initiating any additional trials of SRT501 and is instead focusing on its second-generation SIRT1 activators, which are chemically unrelated to resveratrol and said to be 1,000 times more potent than the original.
Even though GSK isn't advancing SRT501, the Healthy Lifespan Institute has stopped selling resveratrol online, and Westphal and Dipp have resigned their positions with the nonprofit at GSK's request.
Of course, that doesn't stop other nutritional supplement companies from selling resveratrol. But Dunn said GSK doesn't see the resveratrol supplement makers as any commercial threat to GSK's SIRT1 activators because the compounds are completely different.
"Prescription medicines and nutraceuticals don't compete," Dunn said.
GSK should know. A few years ago, the big pharma paid $1.65 billion to acquire Reliant Pharmaceuticals Inc., gaining the rights to several drugs including Lovaza (omega-3-acid ethyl esters), a fish oil-based treatment for high triglycerides. (See BioWorld Today, Nov. 26, 2007.)
Unlike the SIRT1 activators, Lovaza is not a synthetic chemical designed to mimic the activity of the natural product – it is actually FDA-approved fish oil. It costs, on average, $28.70 for insured patients and $180 for uninsured patients for a 30-day supply. And although patients could buy a variety of unapproved fish oil nutritional supplements online or from stores like GNC, Lovaza enjoys 83 percent coverage through managed care formularies and generated some $700 million in revenue for GSK last year.
Declan Doogan, chief medical officer of Amarin Corp. plc, explained that fish oil nutritional supplements are "fine as a general tonic," but once a patient has been diagnosed with a medical condition, like elevated triglycerides, both doctor and patient tend to want a drug.
"For high cholesterol, does your doctor tell you to go buy garlic oil? No, he tells you to change your diet and exercise, and if that doesn't work, he gives you statins," Doogan said.
Since the FDA does not regulate nutritional products, their consistency of dosing and quality of manufacture often cannot be verified. But there are other factors that deter nutritionals from competing with drugs as well.
Doogan explained that patients with high triglycerides need 2 g of the omega-3 EPA daily. Supplements usually contain much less than that. To fit such a high concentration into one pill, they would need increased purity, which Amarin holds patents on. Additionally, increasing the dosage and the manufacturing purity significantly increases the cost to make the pills. Once you head in that direction, "you need a system to support the cost of the product, and that system is reimbursement," Doogan said.
Additionally, the marketability of nutritional supplements and foods is limited by the fact that such products can't be promoted with medical claims. Last year, the FDA slammed General Mills for illegally marketing Cheerios as a drug, citing advertising claims that the cereal is clinically proven to lower cholesterol.
Amarin is in Phase III trials for hypertriglyceridemia and mixed dyslipidemia with AMR101, a prescription grade, ultra-pure ethyl-EPA. Doogan said Amarin expects the pure EPA to offer benefits over Lovaza's mixed omega-3 formulation, such as potentially decreased risk of elevated LDL cholesterol.
Safety? Or Just Sketchy?
Interestingly, while a product sold as a nutritional supplement can be purified, tested and approved as a drug, the process does not work in reverse. The FDA told BioWorld Insight that if a drug failed in clinical trials or failed to gain FDA approval, the sponsor could not try to salvage the compound by selling it as a supplement "if it has never been marketed as a dietary supplement and the [investigational new drug] information has been made public."
However, the FDA added that if the IND was never made public, the product could potentially be marketed as a dietary supplement.
That's the path Neurochem Inc. sought to take after Alzheimer's disease drug Alzhemed (tramiprosate) failed a Phase III trial. The company rebranded itself as Bellus Health Ltd. and said it would commercialize tramiprosate as a branded nutraceutical. (See BioWorld Today, Aug. 28, 2007.)
Tramiprosate, also known as homotaurine, is a naturally occurring amino acid found in certain types of seaweed, according to the Bellus Health website. Scientific literature describes it as a synthetic gamma-aminobutyric acid analogue similar to the sulfonic acid taurine. Bellus sells the product on its website as Vivimind, but has temporarily stopped marketing the nutraceutical in Canada after a government directive requested that pharmacists not sell natural health products without a Health Canada Natural Health Product Number.
Bellus reported C$37,000 (US$35,587) in net sales during the three months ended June 30, 2010.
So if drug makers don't necessarily view nutritional supplements as an economic threat, why was GSK concerned with Westphal and Dipp's extracurricular resveratrol activities?
There's the safety consideration. Clinical trials conducted in a limited number of people sometimes fail to detect adverse events. If a supplement were to become hugely popular and taken by a lot of people, new safety issues could crop up.
"Any kind of safety signal is going to be taken very seriously," GSK's Dunn said. But since GSK's SIRT1 activators are completely different compounds from resveratrol, Dunn said the pharma firm isn't terribly concerned that a safety issue with one would translate to the other.
Amarin's Doogan, whose AMR101 actually is EPA, was similarly unconcerned about safety issues with EPA supplements. "We are not entering into the unknown . . . omega-3s enjoy a long safety record," he said.
In the end, GSK's primary concern was not competition or safety, but conflict of interest, Dunn said. "The specific issue was the involvement of GSK employees . . . contributing to our work to generate sirtuin-based prescription medicines" while being affiliated with a company that sold resveratrol, she said.
It was that conflict of interest, especially involving two extremely seasoned biotech veterans, that caught everyone's attention and caused such a scandal.
Published: August 23, 2010
I do not think you are hyping but when you right an article you need to get your facts better if you want to be taken seriously on this board.
for the idiots that read biomed reports your reports are fine, but we are a bit more serious than most biotech boards.
You also have an uphill road to climb because even though Garza has treated you well, he is about the worst pump and dump artist we have ever seen and you work for him, so expect his sh=t to stick to you until you write some worthwhile articles.
Take a look at the garbage he has written and you will see why you can't be taken seriously here right now.
Then again I think they have actually deleted many of his worst articles from their website so you won't even be able to find them.
ctic, cvm, nwci, and there was another company that was mentioned earlier that had russian breast cancer data that failed miserably but I can't remember the symbol right now.