The Big Red Biotech Blog

Biogen Reports Good News on Oral MS Drug

Biogen's sales were up 9% to $1.2 billion for the quarter.  It reiterated that its oral drug for MS, BG-12, met its primary and secondary endpoints in a pivotal phase III trial and gave analysts a peak at some of the data.  The drug reduced relapses by 49% over two years and reduced disease progression by 38% in a study of 1200 MS patients.

Biogen Shares Jump on Good News on Revenue, Oral MS Drug.

Coupling BG-12's performance with sales growth in Biogen's other MS drugs Tysabri and Avonex (interferon beta 1a) resulted in investor happiness and Biogen's stock surging by nearly 22%.  BG-12 looks to extend Biogen's MS franchise lead with the convenience of a pill.

 

 

Posted by Bruce Lehr April 22nd 2011.

Amgen Passes Key Trial With Son of Dmab For Osteoporosis

Amgen along with its partner UCB revealed today that their next generation bone treatment, AMG 785/CDP 7851, had met its phase II primary endpoints by significantly increasing bone density at 12 months in 400 postmenupausal women.

Amgen Passes Key Trial With Son of Dmab; For Osteoporosis.

AMG 785 is unlike Amgen's current bone drug, denosumab, which blocks bone erosion in patients to help prevent osteoporosis and some cancer-related bone fractures.  Denosumab, quite new itself, is expected to generate $3-4 billion.  AMG 785 meanwhile blocks sclerosin action and helps a patient build new bone.  This is a new approach that may aid patients who have already experienced bone loss. 

AMG 785 is now poised to move on to phase III trials.  When approved, it would provide Amgen with two options for treatng osteoporosis patients -- an estimated $9 billion market.  It is estimated that 20% of women over age 50 in the US have osteoporosis.

 

 

Posted by Bruce Lehr April 22nd 2011.

Nothing Fishy about Amarin Stock Price Rise on Positive Ph 3 Data

Amarin stock doubled today on its second round of positive phase III results with its triglyceride drug, AMR101.  The drug met primary and secondary endpoints in its second phase III trial. Patients showed a significant drop in triglycerides with no rise in LDL-C - so-called "bad cholesterol".  It also caused a reduction in significant markers of cardiovascular risk like Apo B and non-HDL-C. AMR-101 is a modified form of omega-3 fish oil.

"The design and execution of the Anchor trial were robust and the trial results were very clearly positive," said Christie M Ballantyne, MD, principle investigator. Amarin may submit its NDA for AMR101 in Q3 this year and seek approval by Q2 2012.  See Fierce Biotech.

 

Posted by Bruce Lehr April 18th 2011.

Pfizer Releases Top Line Data on Novel RA Drug

Pfizer got some good news from the clinic on its novel rheumatoid arthritis drug, tofacitinib,which met its primary endpoints in a phase III study. Patients symptoms improved signifcantly and structural joint damage was slowed.

Pfizer Releases Top Line Data on Novel RA Drug.

The novel drug, a JAK inhibitor, will move into an area currently dominated by TNF inhibitors like Enbrel (Amgen), Humira (Abbott) and Remicade (J&J/Merck).  Pfizer will release more data at an upcoming meeting. It will be interesting to see how analysts rate this drugs revenue chances versus formidable competition in the RA arena.  Regardless it is good news for Pfizer I'm sure to have one perform well in phase III.

 

 

Posted by Bruce Lehr April 15th 2011.

 

Biogen Idec MS Drug Passes Clinical Test

Biogen reported that its oral MS drug, BG-12 (dimethyl fumarate), performed well in its latest pivotal trial and met its goal in reducing MS flare-ups over two years.  The drug also met secondary goals of reducing the number of new or enlargin brain lesions and slowing the rate of disability. 

BG-12 is on the cusp of joining Biogen's other MS stable-mates, Avonex (interferon B) and Tysabri (natalizumab), to stregthen its industry leading MS franchise. In addition to the benefits cited above, BG-12 was also said to offer a good safety profile and the convenience of taking it orally is considered big plus to patients. Biogen shares were up 5% on the news. See Xconomy.

 

 

Posted by Bruce Lehr April 11th 2011.

 

Medivation, Pfizer Fail in Huntington's Trial

Medivationa and Pfizer announced today that their drug, dimebon, failed to meet its end points in a pivotal trial for Huntington's disease.  As a result, development on the drug has been stopped.

Medivation, Pfizer Fail Huntington's Test.

A phase III trial utilizing the drug against Alzheimer's in patients with mild-to-moderate symptoms will continue.

Posted by Bruce Lehr April 11th 2011.

mAb Development and FDA-Approval Facts

Deloitte Recap recently analyzed the 29 mAb approvals that have thus far passed the FDA. These drugs generated an aggregate $24 B in revenues in 2009.  In looking at the length of time the mAbs were in development, Deloitte found that their mean time to market was 7.6 years - but this time frame is getting longer.  For the period, 1994-2003, the mean time was 6.7 years and that has increased to 8.3 years for the time span 2004-2011. This could possibly be due to the increased complexity of diseases that developers are now trying to attack - most of this time is from additional time in the clinic.

Surprisingly, and unfortunately, Deloitte also discovered that the regulatory programs geared at speeding up a drug's time through the approval process - like fast track, accelerated approval, priority review or orphan status - did not seem to help mAbs arrive any faster.  In fact, fast track status was associated with a longer mean time in development. 

In looking at the data for mAbs, particularly as compare to small molecules, Deloitte also concluded that mAbs approval rates are higher (23 percent), and given some are blockbusters to boot, we're likely to see heavy investment in mAbs for the foreseeable future.  At present, 27 key companies followed by Deloitte have 130 mAbs in their pipelines aimed at 211 indications.

Posted by Bruce Lehr April 5th 2011.

 

Amgen cancer drug fails in PH III

Another one bites the dust!

Amgen and Takeda's late-stage trial of motesanib failed to improve the overall survival rate of patients with late stage lung cancer (specifically advanced non-squamous non-small cell).  Side effects in the study were also reported to be most severe with patients receiving the drug.

Takeda paid $100 M upfront to partner with Amgen on the drug only 2 years ago.  It was part of a $1 billion dollar deal with Amgen to allow Takeda to get more deeply involved in the biologics sector, and to expand its cancer portfolio in the US. See Fierce Biotech.

PharmaGossip: Amgen cancer drug fails in late-stage study - Yahoo! Finance.

"We are disappointed with the results from this trial, but look forward to further analysis [ed. note, always kiss of death when you read this] of the data which may ultimately help inform future research in this area," said Roger Perlmutter, Amgen executive VP of R&D.  See Reuters.

 

 

 

Posted by Bruce Lehr March 30th 2011.

 

 

Another Vertex Positive CF Phase III Result

Vertex announced its drug VX-770 for cystic fibrosis (CF) had done well in another phase III trial with 52 patients.  The drug targets CF patients with a specific genetic defect that represents about 4% of the total CF population. VX-770 improved these patients lung function by a significant 12.5 percent.  Vertex says the drug remains on track for an anticipated FDA filing in late 2011.

"We are committed to making VX-770 available as soon as possible and are moving ahead quickly with our US and European applications for regulatory approval...", said Robert Kaufman, the CMO at Vertex. Vertex stock increased by 2.2 percent on the news of the positive trial result. See Fierce Biotech.

Posted by Bruce Lehr March 29th 2011.

EU Register Of Clinical Trials Is Launched Online

The European Medicines Agency (EMA) yesterday launched on online register for clinical trials for the first time.  The register provides public access to clinical trial information in the 27 EU member states, Iceland, Liechtenstein and Norway.  The information posted is provided by the trial sponsor and is a component of its application to conduct a trial.

PharmaGossip: EU Register Of Clinical Trials Is Launched Online Today.

The EU Clinical Trials Register increases the transparency of medical research and will make it easier for pateints to find information about clinical trials in progress in Europe. The EMA says it will continue to work to improve the register by enhancng the quality and completeness of data, and improving the search functionality.  Plans for the future include the publication of summaries of clinical trial results.

 

 

 

 

Posted by Bruce Lehr March 26th 2011.

GSK/Impax Parkinson's Drug Hits Endpoints in Ph III

GSK and its parnet Impax Pharmaceuticals reported positive results with its drug IPX066 in a phase III trial with Parkinson's disease patients.  The extended-release version of the drug induced a 37% drop in off-time (when a medication's affect has worn off) compared to a 17% decrease in patients taking an immediate release version of the drug.

Article GSK/Impax drug impresses in Ph III Parkinson's trial.

Under the terms of the companies' development and commercialization agreement, Impax wil complete the current phase III program for IPX066 and will file the drug in the US later in 2011. GSK has an exclusive license to commercialize the drug worldwide, except in the US and Taiwan. Impax shares jumped more than 10% on the clinical results news.

 

 

Posted by Bruce Lehr March 16th 2011.

Novartis Jacked Up about Jak Inhibitor Results

As reported in Fierce Biotech, Novartis and its partner Incyte have reported the second set of positive phase III results with its Jak Inhibitor drug, INC424 or ruxolitinib, for the treatment of myelofibrosis.  The companies plan to file a 2nd Qtr NDA with the idea of launching later in 2011.

Novartis paid Incyte $150 million upfront with an agreement for milestones that could reach as much as $1 billion. Novartis would like to get the Jak Inhibitor licensed for this rare blood cancer -- but its real goal would then be able to achieve licenses for additional applications, including rheumatoid arthritis. Pfizer has a similar drug, tofacitinib, aimed at the RA target.

 

Posted by Bruce Lehr March 15th 2011.

VEGF Trap Clinical Failure for Sanofi & Regeneron

Fierce Biotech reports that Sanofi and Regeneron's drug aflibercept (VEGF trap) for thetreatment of non-small cell lung cancer, used in combination with docataxel, failed to meet its endpoint, extending overall survival, in its latest phase III trial.  It did improve progression-free survival though.  Side effects were significant and 27.2 percent of the treatment group had to leave the trial.  Like most of these failures, the sponsors say they will take a closer look at the data to determine if they can proceed or will stop.

 

 

Posted by Bruce Lehr March 13th 2011.

Pharmasset's Hepatitis C Results Rocket Stock

Pharmasset reported results on a [very] small trial with its hepatitis C oral combination therapy with lucrative results to its stock value.  In a test with 16 hepatitis C patients, 15 had their viral load eliminated after only 14 days.

The drugs used in development are called PSI-938 and PSI-7977.  Both are administered via an oral route.  But the really potential excitement for the hepatitis C treatment and patient community is that the combination does NOT require the use of interferon, with its plethora of unpleasant side effects. 

Pharmasset's stock rose by more than 32 percent in a couple of days despite a rather ineffective embargo on the trial result imposed by the European Association for the Study of Liver.  Investors somehow got the news anyhow --- hmmm - I wonder how.

Seeking Alpha points out that this unbridled enthusiasm may be just a tad premature given this result is tantamount to a phase I study with its 16 person patient size.  Investors adopted a "damn the torpedos" attitude without regard to science and reality of larger phase II and III trials (see previous post on Tolerx otelixizumab for example).  The post further points out that previous hepatitis C trials have been afflicted with a phenomenon known as "viral breakthrough" -- when the virus stages a comeback from low-level dormancy as drug resistance develops. 

Thus, while the initial results are promising and surely this therapy should be pursued through the clinic, the jury remains out until larger patient populations are tested.  You may not want to plunk your cash down so early at this stage -- unless you can also afford to lose it.  See Fierce Biotech.

Posted by Bruce Lehr March 12th 2011.

Biogen Branches Out to Blood Disorders

Biogen plans to expand its therapeutic portfolio to include drugs for non cancerous blood disorders according to Reuters.  Acording to CEO George Scangos, the company wants to become less dependent on its two lead products for multiple sclerosis Tysabri and Avonex in the future.  It sees that it has a position to exploit with disorders like thalassemia and hemophilia. 

Biogen currently has two products for hemophilia in phase III.  It's Factor IX product is designed to stay in the body three times longer than current products on the market and thus can be given in a one-dose weekly regimen versus every other day.  Biogen feels the reduced dose regimen will be very attractive to patients who must endure multiple infusions per week now. It's Factor VIII product is similarly targeted to allow for fewer doses per week. Currently the Factor IX market is about $1 B and Factor XIII is about $5 B in size.

Posted by Bruce Lehr March 4th 2011.

Pfizer RA Drug Gives Good News from the Clinic

Pfizer reports good late-stage clinical results with its new rheumatoid arthritis (RA) treatment -- now called tofacitinib.  The trial results showed the drug reduced symptoms of RA as compared to placebo at 3 and 6 months, and exhibited signifcantly greater disease remission at 6 months.

The drug from a new class of oral JAK inhibitors is considered to one of the most important in the Pfizer pipeline - particularly as Lipitor reaches its patent expiration.  JAK inhibitors act by targeting intracellular signalling pathways rather than extracellular targets like other RA drugs. The drugs have great potential in treating not only RA but a range of other inflammatory conditions. 

When approved, Pfizer's drug will compete with well known anti-inflammatory drugs like Abbott's  Humira (slated to move into the world's top seller spot), Amgen's Enbrel and J&J's Remicade.  See Fierce Biotech and Reuters.

 

 

Posted by Bruce Lehr March 4th 2011.

Vertex Scores Again with Telaprevir

As reported in Fierce Biotech, Vertex Pharmaceuticals reported yet another successful trial result with is potential blockbuster telaprevir for the treatment of hepatitis C.  This time telaprevir was tested for its ability to treat hepatitis C infection in HIV patients.  After 4 weeks of therapy, telaprevir was able to eliminate signs of the hepatitis C virus in 70% of HIV patients as compared to only 5% in the placebo arm. Wow - 70 to 5! 

This treatment could potentially be applied to a large number of patients as about 30% of HIV patients also have hepatitis C infections. Vertex, still in Phase II, will push for a phase III trial with the FDA. A Vertex spokesman said "These interim results are encouraging because they showed a high proportion of people in the study had a rapid viral response to telaprevir." This may bode well as rapid response correlates well with the liklihood of acheiving a viral cure. Score another for Vertex.

 

 

Posted by Bruce Lehr March 3rd 2011.

 

 

Bydureon Stubs Toe Versus Victoza in Head to Head Trial

Amylin, Alkermes and Lilly's Bydureon was supposed to show superiority to Novo's Victoza for the treatment of Type 2 diabetes in a head to head trial with 900 patients followed for 26 weeks. Instead the challenger GLP-1 receptor agonist, with its once-weekly dosing regime, tripped up.  Novo's drug proved to be superior in lowering AC1 in comparison (1.5 percent vs 1.3 percent).  Thus, Bydureon failed to meets its primary end pont for this study.

Will this signal Bydureon's death knell with regard to FDA approval? No.  Will if make it harder to get third party payers on board? Probably.  Could physician's reasonably conclude Bydureon is less effective than Victoza? Yes.  Could this result in a less effective commercial case for a drug that is already 2 years behind its competitor reaching the market?  Hell yes.

Results to date - Almyulin stock drops 25%, Alkermes stock drops 12%. Lilly stock flat to slightly down (dependng when you took its pulse).  Big toe needs a band aid.

See Pharmalot, Xconomy-Boston, Forbes, Fierce Biotech and Reuters.

 

 

 

 

Posted by Bruce Lehr March 3rd 2011.

 

Vertex Nails Pivotal Study for Cystic Fibrosis Drug

Vertex announced its latest phase III trial results with a cystic fibrosis (CF) drug, VX-770, that improved patients lung performance by 17 percent versus a placebo - a remarkable result. The effect lasted over the entire 48 week course of the study.

Vertex Nails Pivotal Study for Cystic Fibrosis, Racing Toward Market With Second Drug.

The drug is targeted at a subpopulation of CF suffers (approx 4-5%) who have a particular mutation known as G551D.  In these patients, the drug is the first to actually treat the abnormal CFTR protein underlying the disease.  Vertex will apply for approval with the FDA and EMEA before the end of 2011. The company also plans to test whether the drug will have any benefits for patients with the F508 mutation - the most common among CF patients.

Vertex is also working on a complementary drug, VX-809, and data on the performance of the two drugs in combination is expected later this year. The positive news increased Vertex's stock price by more than 15% to 44.03 per share earlier today. This is more welcome news for Vertex which also has the promising hepatitis C drug, telaprevir, wending its way through clinical trials to approval. 

Posted by Bruce Lehr Feb 25th 2011.

Baxter's Flu Vaccine at Least as Good as Egg-produced

After analyzing the results of patients who received Baxter's seasonal flu vaccine prepared in cell culture -- the company has concluded that it was at least as effective in preventing disease as alternative vaccines produced in the more traditional eggs.  Baxter's vaccine prevented flu in 78% of patients who were vaccinated and the historic figure for egg-based vaccines is about 73%.

Cell-based vaccines offer a faster, more reliable alternative to vaccines grown in fertilized chicken eggs, a 60-year-old method that can take up to six months to complete and is prone to manufacturing problems that have led to vaccine shortages.  In particular, cell-based methods can skip the 8 to 10 week step where the vaccine virus needs to be adapted to growth in eggs.  This time savings can be a critically important factor in times of flu pandemic when vaccine stocks need to be raised quickly.

Baxter is in talks with the FDA about whether the data will be adequate to support licensure in the United States. It hopes within the next month that should be clearer.  Baxter's cell culture vaccine is approved for sale in Austria and the Czech Republic, and Baxter expects broader licensure in most European countries by the end of March.

Let's hope they can add the USA to their list of approvals.

 

Posted by Bruce Lehr Feb 16th 2011.

Drug Approvals Drop Like Stone

Fierce Biotech reports on new study from BIO and BioMedTracker that shows the overall success rate for drugs moving through clinical trials to FDA approval dropped to one in ten, from a previous rate of one in five or six.  The period covered by the study is 2003-2010.

Oncology drugs were hit hardest (no surprise if you watch clinical trial results).  Surprisingly (to me), large molecule drugs are twice as likely to be approved as small molecules (more me-too's I guess). 

"This groundbreaking study highlights the depth and breadth of risk inherent in the drug development process more comprehensively than any other previous study," said Alan Eisenberg, executive vice president of emerging companies and business development at BIO.

Insert your own lobbying effort [here] calling for more R&D grants and supports for the biotech industry.  I may make fun - but do think support announced by Obama administration for increased biotech investment is good idea. The trend described here does need to be reversed - policies that boost innovation and support greater R&D should help.

Posted by Bruce Lehr Feb 15th 2011.

Amgen Pushes Ahead With Son of Dmab

Xconomy reports on Amgen's next pipeline drug for bone treatments.  Its current drug denosumab (or Dmab) is sold under the name Prolia for osteoporosis treatment and Xgeva when used to treat bone fractures in cancer patients. Now it is working on AMG785.

Amgen Pushes Ahead With Son of Dmab; For Treating Broken, Frail Bones.

It is a misnomer to call it Son of Dmab in my mind -- more like Cousin to Dmab as the drug is different and has a completely different mechanism of action. However, it may be very complementary in use for the treatment of patients with bone ailments - like osteoporosis or fractures.

AMG785 is early in the pipeline but does offer interesting possibilities if it reachesd approval. Dmab blocks osteoclast function and prevents normal bone breakdown.  AMG785 conversely can be used to enhance osteoblast function (by inhibiting action of sclerotin) and stimulate bone build up. Thus, Amgen will be able to attack bone healing problems from two perspectives and has the beginnings of a portfolio to sell to bone treatment specialists.

These drugs could help Amgen grab a good portion of the $9 B per year market for the treatment of osteoporosis.  That's a chunk of change Cuz.

 

 

Posted by Bruce Lehr Feb 9th 2011.

FDA Says Six Drug Makers Didn't Finish Follow-Ups

The WSJ reports this afternoon that the FDA said six drug makers, who received accelerated approval for their cancer treatments, did not complete follow-up studies that were promised in exchange for that approval. Among the six are Amgen, GSK and Eli Lilly. 

Richard Pazdur, head of the FDA's cancer division, said, "These confirmatory trials are as important -- if not more important -- than the initial trials leading to the accelerated approval."  Some of the companies have been delinquent for more than six years.

The FDA has the power to fine companies millions of dollars or to even withdraw approval.  My question, "Have they ever done so?"  If not, I wonder what the Company's incentive for doing the follow up studies might be? I know their disincentive. 

For example, Roche's Avastin and AZ's Iressa are two drugs that failed to show benefit in follow up studies for certain approved applications.  The result was the drugs lost their approval - a powerful disincentive to report I'd say.  To date, five drugs failed to confirm benefit in follow up studies.

Would the number have been higher if all the drugs receiving accelerated approval had met their post-market reporting requirments?  We may never know.

 

 

Posted by Bruce Lehr Feb 8th 2011.

 

 

More Late Stage Woe for AZ's Zibotentan

Fierce Biotech reports that AZ's zibotentan failed to meet its endpoints in another late stage clinical trial - this time for prostate cancer.  The plug was pulled on the trial when researchers' concluded the drug would not increase survival rates beyond standard therapy in this patient class. 

AZ is taking one more (and its last) bite at the apple in testign zibotentan plus chemotherapy in patients with metastatic prostate cancer.  This is still considered a long shot - and all hopes of this drug competing head to head with Dendreon's Provenge are gone.

AZ looks like it can add zibotentan to the list of late stage failures to accompany Recentin (colon cancer) and Vandetanib (NSCLC).  Cancer treatments failures continue to take a heavy toll in the industry's R&D pipeline - especially in phase III vs standard therapies.

 

 

Posted by Bruce Lehr Feb 7th 2011.

Merck Drops Earnings Estimate -- Saves R&D -- for Now

Merck's CEO Kenneth Frazier advised investors that Merck had reduced its EPS estimate for 2011 to $3.64 to $3.76 per share versus analysts expectatins for $3.82 per share. The revised forecast came with news that Merck had stopped development of vorapaxar and took a $1.7 B write down of intangible assets related to the drug.

Vorapaxar was one of the prime reasons Merck acquired Schering-Plough as analysts had pegged it as a potential megablockbuster with estimated peak revenues in the $3-$5 B per year range.  Now it is dead. Merck shares are down 6% YTD and fell 3.3% on the revised forecast news.

Merck also faces the prospect of losing perhaps more revenue relative to the S-P deal as it is currently embroiled in an arbitration dispute with J&J over the fate of its Remicade and Simponi drugs. These represent multi-billion dollar sales for Merck and are growing in low double digits.  One consequence of the arbitration decision could be that J&J gains all rights to these drugs.  A decision is expected this year.

Despite all the gloomy news, Merck also announced that it will not cut its R&D staff further -- unlike its rival Pfizer did in order to maintain its EPS forecast for 2012.   Merck's Frazier said deeper R&D cuts would result in "significant underinvestment" for the company's future.  Frazier intends to keep spending on the company's late stage pipeline -- including its potential blockbuster hepatitis C drug boceprevir which is receiving expedited FDA review right now. Merck is willing to take the EPS hit now for the potential gain in the future.  See WSJ.

 

 

Posted by Bruce Lehr Feb 3rd 2011.