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INO - Insider buying http://www.biotechinvestorsnetwork.com/#!insider-buying/c1l5u
CLSN - Griffin Securities research report - $18 target maintained...
https://skydrive.live.com/?cid=d5139e805024fd7c&id=D5139E805024FD7C%211104&authkey=!AA79ZJluHFOP7GQ
'NAVB' - (Navidea completes Alzheimer's disease detection study)
Jan 17, 2013 (Datamonitor via COMTEX) -- Navidea Biopharmaceuticals, Inc., a
biopharmaceutical company focused on precision diagnostic radiopharmaceuticals,
has announced the completion of a study of its novel radiopharmaceutical NAV4694
as a biomarker for visual detection and quantification of cerebral beta-amyloid
in diagnosing Alzheimer's disease, or AD.
The study was designed and conducted by Navidea's partner, AstraZeneca, to
assess the effects of various mass amounts of AZD4694 (NAV4694) on safety and
the efficacy of Positron Emission Tomography (PET) scanning in subjects with AD
and in healthy volunteers (HVs).
Evaluations were completed on the effects of two mass doses of the radioligand
on binding parameters and overall image quality. These endpoints are typical and
important requirements of drug registration dossiers filed with regulatory
authorities for approval of diagnostic agents.
The completed trial was an open-label, non-randomized, multi-center, PET study
in a total of sixteen individuals (8 with mild to moderate AD and 8 elderly HVs)
each imaged on two PET systems at Karolinska Institutet sites in Stockholm,
Sweden.
The study included elderly HVs to demonstrate that no unexpected t racer mass
effects of AZD4694 (NAV4694) in subjects with low or no cerebral beta-amyloid
occur; to compare imaging parameters from healthy, non-AD subjects with those
from subjects with AD; and to extend the safety database.
Navidea is also announcing that it has expanded its relationship with
AstraZeneca and obtained rights to two additional beta-amyloid imaging agents.
AZD2184 and AZD2995 expand Navidea's intellectual property portfolio and are
positioned for research use.
AZD2184 and AZD2995 are 11C Positron Emission Tomography (PET) radioligand
tracers discovered by AstraZeneca in collaboration with Karolinska Institutet in
Stockholm. AstraZeneca entered the agreement to ensure that the compounds are
available to KOLs and academic centers. Both parties believe that these agents
may serve as useful laboratory tools to support additional investigation of
Alzheimer's disease. Financial terms related to the granting of the additional
rights were not disclosed as they were not material.
NAV4694 is a Fluorine-18 labeled precision radiopharmaceutical candidate for use
in the imaging and evaluation of patients with signs or symptoms of cognitive
impairment such as AD. It binds to beta-amyloid deposits in the brain that can
then be imaged in positron emission tomography (PET) scans. Amyloid plaque
pathology is a required feature of AD diagnosis and the presence of amyloid
pathology is a supportive feature for diagnosis of probable AD. Patients who are
negative for amyloid pathology do not have AD.
"We are very pleased to have completed this study addressing some of the
fundamental requirements of diagnostic imaging agents seeking approval. We
continue to make exciting progress with NAV4694, which has demonstrated
important performance characteristics that we believe position it as a true
'best-in-class' second generation agent to aid in the diagnosis of Alzheimer's
disease," said Cornelia Reininger, Navidea's chief medica l officer.
"As previously reported in other Phase II studies, NAV4694 exhibits the
strengths of 11C PIB, the benchmark amyloid imaging agent, but, as the agent is
radio-labeled with 18F, has distribution characteristics that make it more
practical to use. The data from these studies suggest that NAV4694 shows
favorable sensitivity, specificity, rapid brain uptake yet decreased
white-matter uptake which affords improved image clarity."
NAVB to Run into Anticipated Lymphoseek Approval
Jake King January 17, 2013
Shares of Navidea Biopharmaceuticals (NASDAQ:NAVB) are ready to climb as the PDUFA (FDA action) date for the company’s lead product, Lymphoseek, is approaching. This is the second PDUFA date for Lymphoseek, as the FDA delayed its initial decision, previously issuing a complete response letter (CRL) due to questions regarding the manufacture of the diagnostic agent. Impressively, the company was able to respondquickly to the FDA and resubmit its application for U.S. approval less than 2 months after FDA’s request for information, and the current PDUFA date, when the FDA could grant approval, is set for April 30th...
http://propthink.com/navb-to-run-into-anticipated-lymphoseek-approval/4732
$AVEO unusual volume http://www.biotechinvestorsnetwork.com/#!unusual-volume/c20kt TheBIN
$AVEO unusual volume http://www.biotechinvestorsnetwork.com/#!unusual-volume/c20kt
CLSN - Griffin Securities research report...
https://skydrive.live.com/?cid=d5139e805024fd7c&id=D5139E805024FD7C%211104&authkey=!AA79ZJluHFOP7GQ
Meet the Biotech CEO With a $30 Billion Breakthrough
By MICHAEL A. ROBINSON, Defense and Technology Specialist, Money Morning January 17, 2013
Don't worry if you never heard of Dr. J. Joseph Kim. Most investors haven't-at least not yet.
He's the CEO of an early-stage biotech firm that has developed a truly novel product for one of the nation's major medical markets -- vaccines...
http://moneymorning.com/2013/01/17/meet-the-biotech-ceo-with-a-30-billion-breakthrough/
Apps Aim to Detect Skin Cancer
By CHRISTOPHER WEAVER
Smartphone apps are inching onto the turf of doctors and medical-device makers, promising to measure heart rates, display X-ray images and detect skin cancer. They also are prompting concerns about how well they work and whether consumers may rely on their smartphones and skip seeing a doctor...
http://online.wsj.com/article/SB10001424127887323783704578245973988828066.html?mod=rss_Health
Retrophin - An Upstart With Huge Potential
January 17, 2013 | about: RTRX.OB
Disclosure: I am long RTRX.OB, ARNA. (More...)
Retrophin (RTRX.OB) is a thinly covered pharmaceutical company that carries, in my opinion, tremendous upside potential. The company has 4 drugs in the pipeline with one, RE-021, being in phase II trials. The differences between Retrophin and other pharma companies is not only in the types of drugs the company is developing, but in the business model and management style as well. What sets Retrophin apart?...
http://seekingalpha.com/article/1117081-retrophin-an-upstart-with-huge-potential?source=feed
Nuvo research is definitely one of the most undervalued stock in the Biotech Sector .
I won't say lotto. solid one but inept management
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=83500175
Taking Some Profits In Celsion...
http://seekingalpha.com/article/1115281-taking-some-profits-in-celsion?source=feed
old PR but relevant...
Neuralstem Cells Induce Significant Functional Improvement In Permanent Rat Spinal Cord Injury, Cell Study Reports
Paralyzed Rats Regain Use of Lower Limbs
Sept. 13, 2012, 12:01 p.m. EDT
ROCKVILLE, Md., Sept. 13, 2012 /PRNewswire via COMTEX/ -- Neuralstem, Inc. (nyse mkt:CUR) announced that its neural stem cells were part of a study, "Long-Distance Growth and Connectivity of Neural Stem Cells After Severe Spinal Cord Injury: Cell-Intrinsic Mechanisms Overcome Spinal Inhibition," published online today in a leading scientific journal CELL ( http://www.cell.com/current ). In the study, rats with surgically transected spinal cords, which rendered them permanently and completely paraplegic, were transplanted with Neuralstem's spinal cord stem cells (NSI-566). The study reports that the animals recovered significant locomotor function, regaining movement in all lower extremity joints, and that the transplanted neural stem cells turned into neurons which grew a "remarkable" number of axons that extended for "very long distances" over 17 spinal segments, making connections both above and below the point of severance. These axons reached up to the cervical region (C4) and down to the lumbar region (L1). They also appeared to make reciprocal synaptic connectivity with the host rat spinal cord neurons in the gray matter for several segments below the injury.
Further study showed that re-transecting the spinal cord immediately above the graft abolished the functional gain, indicating that the regeneration of host axons into the human stem cell graft was responsible for the functional recovery. The cells that Neuralstem contributed to the study, NSI-566, are the same cells used in the recently completed Phase 1 clinical trial for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). Neuralstem has also submitted an application to the FDA for a trial to treat chronic spinal cord injury with these cells.
"This study demonstrates that our neural stem cells can induce regeneration of injured spinal cord axons into the graft and serve as a bridge to reconnect to gray matter motor neurons for many spinal cord segments below the injury," said Karl Johe, PhD, Chairman of Neuralstem's Board of Directors and Chief Scientific Officer. "This is important in spinal cord injury because the nerve connections below the point of injury die, causing paralysis. Our cells built a bridge that received inputs from regenerating rat axons above the injury. They also sent out new human axons which made new synaptic connections with the host motor neurons in the gray matter below the injury. The fact that these cells induce regeneration of axons and partial recovery of motor function makes them relevant for testing for the treatment of human spinal cord injury."
About the Study
In a study of 12 rats, all 12 underwent complete spinal transections at vertebrae, T3. Six of these were subsequently transplanted with Neuralstem spinal cord stem cells (NSI-566) seven days after the injury. This group was assessed over the next seven weeks and compared to the control group, which had not received transplants. The transplanted rats exhibited significant locomotor recovery, regaining movement in all lower extremity joints. A majority of the grafted cells (57%) turned into neurons. From these, the study reported, a remarkable number of axons emerged, extending both above and below the point of spinal cord lesion. These axons expressed synaptic proteins in the host gray matter, which suggests they made synaptic contact with the host spinal neurons.
EU signs off on Lilly's Amyvid, its diagnostic test for Alzheimer's
January 16, 2013 | By Eric Palmer
While Eli Lilly struggles with development of its therapy for treating Alzheimer's disease, its diagnostic imaging agent for helping diagnose, or rule out, the condition has gotten another approval, this time in Europe.
Lilly ($LLY) announced today that EU regulators have approved Amyvid with PET imaging for use in adult patients who are being evaluated for Alzheimer's disease and other cognitive conditions. The tool measures the level of plaque in the brain, which is associated with Alzheimer's. Before, the tests were only evident during an autopsy. Lilly points out that one in 5 patients who receive a diagnosis of probable Alzheimer's are not found to have the plaques during an autopsy. The FDA approved the test last April.
Press release...
http://www.fiercepharma.com/press-releases/amyvid-florbetapir-f-18-injection-approved-european-union-use-patients-cogn
previous post on GE's flutemetamol...
GE Healthcare ($GE) moved ever-so-close this week to the regulatory finish line for flutemetamol, a PET imaging agent designed to help diagnose Alzheimer's disease.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=83212266
Big Pharma drives R&D push for new drugs to treat neglected diseases
January 16, 2013 | By John Carroll
The campaign to develop new drugs for traditionally neglected diseases like malaria has been gaining steam. An industry organization surveyed the field and found 132 R&D programs under way in 2012, a 40% increase over the year before, as billions of dollars are being pumped into a fresh set of collaborations.
The vast majority of those programs--112 to be exact--are being conducted in partnerships with key pharma players, according to the International Federation of Pharmaceutical Manufacturers & Associations. And 15%, or 20 programs, are being driven entirely by companies. In stark contrast, just 7 years ago the industry association could find only about 30 R&D programs in progress for neglected diseases, a roster of ailments that afflict poor populations unable to afford the medicines that drive profitability in the industry.
The spike in research activity, though, has a long way to go before it can translate into newly approved drugs. And that's been a source of real frustration for some researchers in the field. A new study out late last year concluded that heightened activities in the field over the past decade had not produced any significant increase in new drug approvals for neglected conditions. Only 37 of the 850 new drugs approved between 2000 and 2011 were for neglected diseases, Nature reported, and only four of those treatments were actually new chemical entities--a woeful track record.
The lion's share of the cash needed to do the new work is coming from public sources, says the IFPMA. The new analysis finds that the public sector provided close to $2 billion of the $3.05 billion spent in 2011. Philanthropic groups contributed another $570 million, with pharma companies coming in third with $525 million.
Some pharma giants do the work themselves, or simply finance outside groups that do the work. Lilly ($LLY), for example, helps fund the Infectious Disease Research Institute in Seattle, which is doing early-stage work on new TB drugs. Sanofi ($SNY), GlaxoSmithKline ($GSK) and AstraZeneca ($AZN) are among the most active companies, according to the report, which breaks down each of the 132 research programs it was able to track.
http://www.fiercebiotech.com/story/big-pharma-drives-rd-push-new-drugs-treat-neglected-diseases/2013-01-16#ixzz2I9q1Z9bX
ITMN - InterMune Flush With Cash And ASCEND Positioned To Succeed..
http://www.biotechinvestorsnetwork.com/#!featured-article/c17ob
ITMN - InterMune Flush With Cash And ASCEND Positioned To Succeed..
http://www.biotechinvestorsnetwork.com/#!featured-article/c17ob
ITMN - InterMune Flush With Cash And ASCEND Positioned To Succeed..
http://www.biotechinvestorsnetwork.com/#!featured-article/c17ob
InterMune Flush With Cash And ASCEND Positioned To Succeed
Rajesh Patel, Ph.D.January 16, 2013
InterMune (NASDAQ:ITMN) just announced very favorable pricing on their latest round of financing. The company sold $105 million aggregate principal amount of its 2.5% senior convertible notes as well as 13.5 million shares of common stock priced at $9.90 per share (the stock closed yesterday at $10.04). The financings netted the company $228.1 million, not counting any over-allotment options granted to the underwriters (up to $35.79 million additional gross proceeds). We believe that InterMune is attractively priced given fundamental developments at the company as well as technical positioning of the stock.
In their press release, the company indicated that they have commitments to repurchase $50 million of their 5.00% senior convertible notes due 2015. The 2015 notes had an aggregate principal amount outstanding of approximately $85 million. We believe that the company will eventually be able to repurchase the entire tranche of 2015 notes. It’s encouraging to see that the 2017 notes will bear interest at half the rate of the 2015 senior notes. The market is acknowledging the progress the company has made in gaining European approval of Esbriet and commencing sales. Furthermore, the new notes have an effective conversion price of $12.87 which is 30% above the current stock price. Interestingly, the 424(b)(5) prospectus filed with the SEC indicated that the company expected to raise about $82.1 million through the notes offering. Instead, they have been able to raise $105 million, offering ITMN additional cash runway.
Prior to this stock offering, ITMN had 65.9 million shares of common stock outstanding. After the offering, not counting over-allotments, the company will have 79.7 million shares outstanding. As of September 30, 2012 the company had 351.4 million in cash and cash equivalents. Since ITMN has been using approximately $42 million in cash per quarter, after the offering the company’s current cash position should be approximately $513 million. The company recently guided that total operating expenses for 2013 should be in the range of $245 to $285 million; therefore, the company should be able to end 2013 with an additional year’s worth of cash on-hand at 2013 expense levels.
Upcoming Catalysts
EU Rollout
InterMune will be expanding their EU rollout of Esbriet to the remaining 6 of the top 15 EU markets this year. While EU sales are a focal point of the story, management’s wide guidance range for these sales sets the tone for conservatism. The company expects EU Esbriet sales to fall in the range of $40M to $70M this year, versus the Wall Street Consensus estimate of approximately $65M. While estimates are currently towards the high end of the range, analysts are likely to tweak their estimates down given the company’s detailed guidance, which reminds investors that certain territories in the EU still need to come on-line in terms of final drug approval and reimbursement. As a result, ITMN should be positioned to meet expectations as the year progresses, removing any prior overhang on the stock (see company’s guidance below). Most importantly, the majority of value that analysts are ascribing to the stock relate to the results of the ASCEND trial and the subsequent U.S. sales opportunity.
“Esbriet revenue: currently projected to be in a range of $40 to $70 million. This includes projected revenue in a range of $40 to $55 million in countries where Esbriet is currently launched (Germany, France, Canada and seven mid-sized European countries), and projected revenue in a range of $0 to $15 million in countries where Esbriet pricing and reimbursement approval and launch is not yet concluded but is currently anticipated during 2013 (Italy, UK, Spain and three mid-sized European countries). The guidance also accounts for the projected time needed to address regional and provincial reimbursement procedures in Italy, Spain and Canada before meaningful Esbriet revenues can be achieved in all regions or provinces in these countries.”
UK Reimbursement
In the UK the National Institute for Clinical Excellence (NICE) issued a preliminary assessment of Esbriet in November 2012 that did not support reimbursement for the drug. The company is addressing issues raised in the NICE preliminary assessment and expects a final assessment in March 2013. The market currently appears to be pricing in a negative decision by NICE. The march NICE final assessment for Esbriet is a binary event for ITMN stock because if the drug receives reimbursement in England, 2013 Esbriet revenues are likely to come in towards the middle to upper end of the guided range of $40 – $70 million.
ASCEND Trial
The key catalyst for ITMN’s stock is actually in 2104 with the outcome of it’s ASCEND phase III trial to support US approval. Recall that in 2010, ITMN received a 9-3 positive advisory committee vote supporting approval of Esbriet (pirfenidone) for treatment of idiopathic pulmonary fibrosis (IPF). Two months later, the FDA issued a complete response letter requiring another clinical trial to demonstrate further efficacy of the drug. The ASCEND trial reached full enrollment towards the end of 2012, and the company has guided that top-line results from the study will be available in Q2 2014.
We believe that the ASCEND trial is well positioned to show a treatment benefit for pirfenidone due to changes in study enrollment criteria compared to previous trials. According to the company:
Relative to InterMune’s previous studies of pirfenidone in IPF, the entry criteria for ASCEND have been refined to enrich the study population for patients who are more likely to experience decline in lung function and disease progression during the study. InterMune believes that enrolling patients whose disease is more likely to progress will provide greater opportunity to demonstrate a treatment effect in a one-year study.While the entry criteria have been refined, the study is conservatively powered based on the intent-to-treat analyses from the previous CAPACITY Phase 3 program.
Indeed, in 2013 the company intends to “begin building its U.S. commercial infrastructure and begin pre-launch preparations for Esbriet.” We take this as a bullish indication that the company expects a positive outcome in the trial based on the enriched study population.
Conclusion
With the overhang of financing removed at very favorable terms, as well as full EU roll out and initiation of Esbriet sales in Canada, our view is that ITMN stock is positioned to move higher. This past August, ITMN’s stock price put in a 5-year low of $7.21. Since then the stock has been moving in an ascending triangle and the latest financing should serve as a support level. The 200-day Exponential moving average stands at $11. Once this price level is broken to the upside, the stock has room to move back to the $12 – $13 range in the short term. As outlined above, should the UK NICE agree to reimburse for Esbriet in March, this will serve as a near-term catalyst and will position the firm to achieve the middle to upper range of their 2013 revenue guidance.
Another potential positive note for the stock is the possibility of in-licensing or acquisitions. As mentioned in the prospectus for the latest financing round, after refinancing the 2015 debt, the company plans to use proceeds to fund the EU roll out of Esbriet, for working capital, possible capital expenditures, and “acquisitions or investments in complementary businesses, products and technologies.” If the company does in-license or make an acquisition, we believe that this will be viewed favorably by the market as it diversifies their future revenue stream.
FDA Considering Alternative Approval Pathway
By Alexander J. Varond –
January 15, 2013
FDA announced earlier this week that it will hold a public hearing to obtain input on a potential new pathway to expedite the development of drugs and biological products for serious or life-threatening conditions that would address an unmet medical need. The new pathway would be used to approve drugs studied in well-defined subpopulations of patients with more serious manifestations of a condition. By focusing on patients with more serious manifestations of a condition, the pathway opens the door to the use of smaller and faster clinical trials. This approach was recently outlined in FDA’s draft guidance entitled “Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products.” We wrote about that draft guidance here. FDA notes that by focusing on obtaining approval in well-defined, sicker subpopulations, the risk-benefit considerations may favor approval compared with trials that aim for approval in broader groups of patients with wide ranges of clinical manifestations. The potential new approval pathway could also help reduce the development of resistance to antibacterial drugs by limiting their use to certain patients.
The public hearing is a follow-up to a recommendation from the President’s Council of Advisors on Science and Technology’s (PCAST’s) September 2012 report entitled “Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation.” We blogged on that report here. Among the eight specific recommendations made in the report, PCAST recommended a new, optional pathway for initial approval of drugs shown to be safe and effective in a specific subgroup of patients. The report also discussed a “Special Medical Use” designation which would strongly signal to prescribers and payors that the population that should be prescribed the drug is limited.
The issues to be addressed at the hearing include:
The effectiveness of adding an additional pathway to expedite drug development (on top of accelerated approval and fast-track designation);
Which serious or life-threatening conditions the new approval pathway might benefit;
What approaches can be used to prevent, manage, and monitor use of a drug approved under this pathway in a broader population where safety and efficacy has not been demonstrated;
Whether this pathway could be particularly useful in the development of antibacterial drugs;
What benefit-risk considerations should to be taken into account regarding the smaller population of patients with more serious manifestations of a condition;
and
Would the use of a logo or formal designation that reflected approval under the new pathway be effective in limiting use to the indicated subpopulation?
FDA’s proposal for a special logo or formal designation reflects PCAST’s and the Enrichment guidance’s concerns that drugs approved under the new pathway would be used off-label in larger populations, effectively side-stepping the normal, more rigorous approval pathway FDA typically requires. Importantly, FDA’s and PCAST’s proposals both note that a drug initially approved using the new pathway could be later approved for a broader population if long-term studies demonstrate safety and effectiveness for broader use.
The public hearing is scheduled for February 4 and 5, 2013.
http://www.fdalawblog.net/fda_law_blog_hyman_phelps/2013/01/fda-considering-alternative-approval-pathway.html
GenMark Diagnostics target raised to $14 at Canaccord Genuity; on to the next leg of growth (11.14)
Canaccord raises their GNMK tgt to $14 from $12. Given mgmt's recent track record of executing, coupled with its long-term growth trajectory, firm believes GNMK merits trading at the upper end of the MDx peer group P/S multiple (5x-7x). Given the strong start to the flu season, GNMK should see good results from its RVP test. CDC indicated that 7.3% of all deaths resulted from pneumonia and influenza, crossing the "epidemic" threshold of 7.2%.
Read more: http://www.briefing.com/InPlayEq/InPlay/InPlayDual.htm#ixzz2I3SHr07T
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Early Stage Biotech Showing Positive Signs of Scaling Its Wall of Worry
PHARMA & HEALTHCARE | 1/15/2013 @ 1:04PM |138 views
Bruce Booth, Contributor
Early stage life science VC
As 2013 begins, and the JPM conference is behind us, it’s a great time to reflect on the state of the biotech industry and the sentiments around early stage investing in particular. While pessimism abounds in many corners about the current and future health of emerging biotech, the reality is that the sector has been quietly scaling its wall of worry.
For years we’ve complained about a triple threat of worries: FDA obstacles, poor public markets, and a perceived lack of funding in biotech. Some of this we’ve done to ourselves (and deserved), as I’ve noted before (here), but 2012 in particular has challenged these concerns with positive data which should manifest into real improvements in the early stage climate. I’ve been accused of being a Pollyanna about early stage by some, but I think the data support a more optimistic view than is widely held by many stakeholders and observers. Unfortunately even with good data it will take time to turn bearish sentiments into bullish ones, but I think the fundamentals are pointing in the right direction. Let me take on each of the big three “worries” that has plagued our sector of late.
FDA and the regulatory landscape. Last year we witnessed 39 new molecular entity drug approvals by the FDA, one of the three best years in the past half-a-century (yes, 50 years!). 2012 was only bested by the outlier year of 1996, which at 53 included a big “drug lag” cohort pushed through in the wake of PDUFA I (passed in 1992). Coupled with a big year in 2011, this is a great sign that the decline in approvals may be reversing. According to FierceBiotech, McKinsey forecasts 36 or so approvals each year through 2016. This is great news, and would make for one of the best five years in the history of the Agency.
Lots of others have already written about the positive and negative attributes of these 39 approvals (here, here, here). It’s worth noting that most of the critique is that many of the drugs last year were either approved for orphan diseases or were me-too incremental innovations. These criticisms may well be true, but this isn’t much different than others years. Taking the outlier 1996 cohort of approvals as an example, how many readers of heard of blockbusters like Albenza, Cerebyx, ProAmatine, Elmeron, and Zanaflex? All of those were orphan drug approvals. And what about the incremental me-too innovations approved in 1996: Lipitor, Crixivan, Humalog, Zyprexa, and Diovan. They all did reasonably well. I have no reason to believe the Class of 2012 drug approvals won’t have some strong performers.
Irrespective of whether these drugs help drive big economic gains for Pharma, they certainly reflect an FDA that is increasingly open to approving new medicines, especially those that are real, innovative products. Many of the first-in-class agents approved last year were reviewed faster then their full PDUFA-directed review time. This and other efforts, like the Breakthrough medicine concept, reflect an FDA that is trying, and succeeding at least in part, to adopt a more innovation-friendly posture, and this bodes well for early stage biotech as we need clear paths to regulatory approval.
IPOs and the public market for biotech. While I’m often a critic of the “go-public” path for aspiring biotechs, its fair to say that post-IPO performance of the class of 2011-2012 has done exceptionally well. Of the 20 biotechs that priced IPOs in that period, 75% are above their trading price, with an average and median gain of 27% and 17% respectively. Intercept and Pacira have more than doubled, Kythera is up 80%, and even preclinical stories like Regulus and Verastem have held their own. This is much better than in many prior biotech vintages of the past decade where two-thirds traded down in their first 12-24 months under the pressure of future fundraising expectations. However, it remains a real issue that the pricing of IPOs is a massive challenge (and I still won’t advocate for the IPO path for most biotechs), but for much of the 2000s both pricing and after-market performance were issues. At least we’re trending well on the latter and hopefully the former will follow suite. We obviously need both to create a healthy public market for new biotech offerings.
A related point to the improving IPO landscape is that many of the buysiders that would buy into these offerings are recognizing that great companies are getting plucked off by Pharma before they get to the public markets. This has led to a influx of buyside interest in late stage stories again. Take Agios and Foundation Medicine – both have raised considerable amounts of capital from buyside investors at very high valuations. In fact, Agios may be the highest valued preclinical stage company I can think of, reflecting the confidence the market has in David Schenkein, his team, and the cancer metabolism field. Seeing some of these emerging success stories come to fruition with solid IPOs or M&A deals will be further catalysts for addressing the public market worry that many early stage investors currently have.
Funding. The quarterly vacillations in venture funding usually create lots of fanfare, especially amongst the Chicken Little’s of the sector, but the aggregate annual funding numbers for the past few years are surprisingly strong. Based on BioCentury data, $5.5 Billion was invested in venture-backed biotechs in 2012, up from 2008-2011, and it includes ~$1 Billion for Seed/Series A investments. Given the outcry about funding drying up, this is pretty robust.
At the New Paradigms meeting (a satellite of JPM Conference), a panel I was involved with discussed the perceived funding gap and whether great companies were still getting financing. The unanimous view was that innovative new startups were continuing to attract capital. I do share the concern of many about the number of new startups being formed in biotech; its down from its historic range of 80-100 per year, and is worth watching closely. These new startups will be what feeds the later stage funding cycle and future IPOs. That said, a flip side to this is that as an early stage investor involved in creating these new startups, the supply-and-demand dynamic reinforces my belief that this a great time for early stage venture creation. Many entrepreneurs I know feel the same way.
Although the aggregate funding numbers remain strong, its very clear that the mix of investors in these financings has changed. As noted above, the buyside and in particular hedge fund investors are supporting some of the later rounds. In addition, the early stage rounds have seen a significant increase in corporate venture capital involvement (discussed here, here). As I’ve commented previously, some 80%+ of our new deals are now syndicated with Pharma VCs. These corporate players are also actively filing the coffers of standalone venture firms through an increasing number of LP relationships. Lastly, a new crop of life science VCs have emerged. Beyond big new players, like Third Rock Ventures and VenBio, there are a number of smaller and/or regionally focused LS investors including Remeditex Ventures (coinvestor with me at Miragen), Allied Bridge Gap Ventures in Boston, City Hill Ventures, NaviMed Capital in the device space, and a number of others.
I’d be remiss if I didn’t acknowledge that there’s a huge bias against Life Sciences in the current LP environment; LS is clearly a contrarian bet today, and its not easy to raise funds from traditional LPs for either hybrid funds (Tech + LS) or LS-only vehicles. This negativity is a major concern, but hopefully continued solid data on LS returns, coupled with an embrace of data-driven decision-making by LP’s, will change this sentiment for the better.
Beyond the directional improvement around these big three worries, I am encouraged to see that innovation is continuing to be rewarded, through both earlier stage M&A deals with great venture returns (like Phase 1-2 exits at Calistoga, Amira, Avila, Stromedix, Enobia), as well as in the public markets, as I’ve written about recently (here). Further, Pharma’s posture has changed for the better towards early stage innovaiton, and it appears keen on helping facilitate but not suffocate translational efforts beyond their walls (here). These trends also bode well for early stage biotech.
We live in an era of big biology: we’ve taken ten years, but the real fruits of genomics are finally ripening and exciting new medicines are being developed. This is a great time to be in early stage translational research, and I’m hopeful the FDA, the public markets, and the funding environment continue to trend favorably. As a sector, we’re scaling the wall of worry and I look forward to a great run of successful investing in innovation over the next few years.
New article... Sangamo BioSciences: A Potentially Revolutionary 2013
http://propthink.com/sangamo-biosciences-a-potentially-revolutionary-year/4628
New article... Sangamo BioSciences: A Potentially Revolutionary 2013
http://propthink.com/sangamo-biosciences-a-potentially-revolutionary-year/4628
Stop your complaining! :)
The rumor is there were customers who bought in 4Q because of the flu epidemic, that were expected to wait until 1Q 2013.
The Race to Cure Spinal Cord Injury...
http://www.biotechinvestorsnetwork.com/#!featured-article/c17ob
The Race to Cure Spinal Cord Injury...
http://www.biotechinvestorsnetwork.com/#!featured-article/c17ob
The Race to Cure Spinal Cord Injury...
http://www.biotechinvestorsnetwork.com/#!featured-article/c17ob
The Race to Cure Spinal Cord Injury...
http://www.biotechinvestorsnetwork.com/#!featured-article/c17ob
Re: Sorrento....the only clue as to the source of the acquired rights was this...
"Partnering our rIVIG invention with STI's unique G-MAB® technology may produce novel and highly differentiated IVIG products," adds Dr. Tien Lee, M.D., one of the co-inventors of the rIVIG technology.
The mysterious Dr. Tien Lee is a no-show when I searched on google...unless he's a GP practicing in San Francisco...
Searching on these keywords also came up zilch...
>Dr. Tien Lee recombinant Intravenous immunoglobulins<
http://goo.gl/Qcvvk
Canaccord ups TearLab price target to $8
January 14, 2013 by Jennifer Poland · Leave a Comment
Canaccord Genuity has raised its price target on “buy-rated” TearLab (NASDAQ:TEAR; TSX:TLB) to $8 from $6, saying the growth profile of the stock justifies a higher multiple than its peer group. The stock closed at $5.31 on Friday.
“In our opinion, TearLab should start benefitting from physician referrals (>500 IB), expanding sales force (12 reps and four managers), and robust lead activity” from the American Academy of Ophthalmology conference in November,” writes analyst Jeffrey Frelick.
Citing rising awareness among doctors, Mr. Frelick estimates TearLab collected orders on 80-90 units during the one-week conference, compared with 134 placements in the third quarter.
He is maintaining his fourth quarter revenue estimate of $1.6-million, a 223% year-over-year increase, and 115 instrument placements, which compares to only 28 in the same period last year. “As awareness grows, we expect placements to range from 130 to 160 per quarter in 2013,” he added.
TearLab has first-mover advantage with its point-of-care diagnostic platform for the diagnosis of dry eye disease using tear film Osmolarity.
“Greater awareness of TearLab’s Osmolarity test should lead to additional referrals among the U.S. ophthalmologist community,” Mr. Frelick said. “We believe improved patient workflow and high mix of dry eye patients will support adoption of the TearLab Osmolarity System.”
http://biotuesdays.com/2013/01/14/canaccord-ups-tearlab-price-target-to-8/?utm_source=feedburner&utm_medium=feed&utm_campaign=Feed%3A+Biotuesday+%28BioTuesday%29
Thanks for the insight. The depth of knowledge on this board is humbling and the willingness to share is appreciated.
We provide evidence that MYC gene expression can be potently and reversibly abrogated by BET bromodomains inhibitors. Transcriptional silencing of MYC is coincident with release of BET proteins from the MYC locus, indicating that BET proteins directly regulate MYC gene expression. Although BET inhibitors influence the expression of an assortment of genes, the MYC transcriptome heavily dominates the gene expression changes observed upon BET-bromodomain inhibition. The MYC gene is translocated in all patients with BL and a significant fraction of patients with multiple myeloma or diffuse large B-cell lymphoma (19). BL is well established to depend on the functions of MYC (15, 19). We observed that several cell lines harboring MYC translocations are sensitive to BET inhibitors as measured by both transcriptional effects and apoptotic responses (Fig. 1, Fig. S2, and Fig. S3), perhaps reflecting their degree of MYC addiction (20). Apart from the special case of midline carcinoma, we have yet to observe a cell type that is sensitive to BET inhibitors without down-regulation of MYC (Fig. S6A). However, MYC reduction did not always correlate with a cell proliferation phenotype in vitro. For example, MYC protein was significantly reduced in the breast cancer cell line MDA-MB-231, which nevertheless grew normally upon administration of (+)-JQ1 (Fig. 1A and Fig. S3B). Previous studies have indicated that the in vitro proliferation of the MDA-MB-231 cell line is only mildly affected by a severe reduction of MYC protein by shRNA (21). To corroborate this observation, we infected MDA-MB-231 cells with four different shRNAs that knockdown MYC mRNA and assessed their effect on proliferation. As shown in Fig. S6B, MDA-MB-231 cells are not phenotypically sensitive to MYC knockdown by shRNA at comparable mRNA knockdown levels to (+)-JQ1–mediated suppression of MYC. No gross toxicity was observed upon high-dose treatment of BET inhibitors in this MYC-insensitive cell line, revealing no marked, general MYC-independent effects on cellular viability in this setting. Suppression of MYC by BET inhibitors was not observed in every cell line tested, and the degree of MYC silencing also varied at the time points tested. It is possible that variation in the permeability and efflux of BET inhibitors across cell lines contributes to some of these observations. Conceptually, it will be important to elucidate in detail the chromatin or regulatory contexts that define MYC sensitivity to BET inhibitors. These regulatory features may ultimately identify the patients who will respond best to this class of small molecule inhibitors.
http://www.pnas.org/content/108/40/16669.full
Interesting. So are there no companies out there that are currently targeting Myc, even at an early research stage?
DNA pioneer James Watson takes aim at "cancer establishments"
By Sharon BegleyPosted 2013/01/09 at 6:34 am EST
NEW YORK, Jan. 9, 2013 (Reuters) — A day after an exhaustive national report on cancer found the United States is making only slow progress against the disease, one of the country's most iconic - and iconoclastic - scientists weighed in on "the war against cancer." And he does not like what he sees.
James Watson, co-discoverer of the double helix structure of DNA, lit into targets large and small. On government officials who oversee cancer research, he wrote in a paper published on Tuesday in the journal Open Biology, "We now have no general of influence, much less power ... leading our country's War on Cancer."
On the $100 million U.S. project to determine the DNA changes that drive nine forms of cancer: It is "not likely to produce the truly breakthrough drugs that we now so desperately need," Watson argued. On the idea that antioxidants such as those in colorful berries fight cancer: "The time has come to seriously ask whether antioxidant use much more likely causes than prevents cancer."
That Watson's impassioned plea came on the heels of the annual cancer report was coincidental. He worked on the paper for months, and it represents the culmination of decades of thinking about the subject. Watson, 84, taught a course on cancer at Harvard University in 1959, three years before he shared the Nobel Prize in medicine for his role in discovering the double helix, which opened the door to understanding the role of genetics in disease.
Other cancer luminaries gave Watson's paper mixed reviews.
"There are a lot of interesting ideas in it, some of them sustainable by existing evidence, others that simply conflict with well-documented findings," said one eminent cancer biologist who asked not to be identified so as not to offend Watson. "As is often the case, he's stirring the pot, most likely in a very productive way."
There is wide agreement, however, that current approaches are not yielding the progress they promised. Much of the decline in cancer mortality in the United States, for instance, reflects the fact that fewer people are smoking, not the benefits of clever new therapies.
GENETIC HOPES
"The great hope of the modern targeted approach was that with DNA sequencing we would be able to find what specific genes, when mutated, caused each cancer," said molecular biologist Mark Ptashne of Memorial Sloan-Kettering Cancer Center in New York. The next step was to design a drug to block the runaway proliferation the mutation caused.
But almost none of the resulting treatments cures cancer. "These new therapies work for just a few months," Watson told Reuters in a rare interview. "And we have nothing for major cancers such as the lung, colon and breast that have become metastatic."
The main reason drugs that target genetic glitches are not cures is that cancer cells have a work-around. If one biochemical pathway to growth and proliferation is blocked by a drug such as AstraZeneca's Iressa or Genentech's Tarceva for non-small-cell lung cancer, said cancer biologist Robert Weinberg of MIT, the cancer cells activate a different, equally effective pathway.
That is why Watson advocates a different approach: targeting features that all cancer cells, especially those in metastatic cancers, have in common.
One such commonality is oxygen radicals. Those forms of oxygen rip apart other components of cells, such as DNA. That is why antioxidants, which have become near-ubiquitous additives in grocery foods from snack bars to soda, are thought to be healthful: they mop up damaging oxygen radicals.
That simple picture becomes more complicated, however, once cancer is present. Radiation therapy and many chemotherapies kill cancer cells by generating oxygen radicals, which trigger cell suicide. If a cancer patient is binging on berries and other antioxidants, it can actually keep therapies from working, Watson proposed.
"Everyone thought antioxidants were great," he said. "But I'm saying they can prevent us from killing cancer cells."
'ANTI-ANTIOXIDANTS'
Research backs him up. A number of studies have shown that taking antioxidants such as vitamin E do not reduce the risk of cancer but can actually increase it, and can even shorten life. But drugs that block antioxidants - "anti-antioxidants" - might make even existing cancer drugs more effective.
Anything that keeps cancer cells full of oxygen radicals "is likely an important component of any effective treatment," said cancer biologist Robert Benezra of Sloan-Kettering.
Watson's anti-antioxidant stance includes one historical irony. The first high-profile proponent of eating lots of antioxidants (specifically, vitamin C) was biochemist Linus Pauling, who died in 1994 at age 93. Watson and his lab mate, Francis Crick, famously beat Pauling to the discovery of the double helix in 1953.
One elusive but promising target, Watson said, is a protein in cells called Myc. It controls more than 1,000 other molecules inside cells, including many involved in cancer. Studies suggest that turning off Myc causes cancer cells to self-destruct in a process called apoptosis.
"The notion that targeting Myc will cure cancer has been around for a long time," said cancer biologist Hans-Guido Wendel of Sloan-Kettering. "Blocking production of Myc is an interesting line of investigation. I think there's promise in that."
Targeting Myc, however, has been a backwater of drug development. "Personalized medicine" that targets a patient's specific cancer-causing mutation attracts the lion's share of research dollars.
"The biggest obstacle" to a true war against cancer, Watson wrote, may be "the inherently conservative nature of today's cancer research establishments." As long as that's so, "curing cancer will always be 10 or 20 years away."
(Reporting by Sharon Begley; Editing by Jilian Mincer and Peter Cooney)
Copyright Reuters 2008. See Restrictions for more
Genmark (GNMK) has been a beneficiary of the flu hysteria... their RVP (respiratory virus panel) which detects 14 viruses that cause flu like symptoms was FDA approved at the beginning of the 4Q... http://investorshub.advfn.com/boards/read_msg.aspx?message_id=79473271
GNMK broke out to 52 wk highs on Fri.
BioTime Picks Up Geron’s Stem Cell Assets
Newly formed public company will be co-owned by Geron, BioTime, and private investor.
MICHAEL FITZHUGH
The Burrill Report
Following the shutdown of what was once the biotech industry’s leading embryonic stem cell therapeutics program, BioTime and former Geron CEO Tom Okarma are spearheading a revival of programs to develop Geron’s stem cell assets.
Geron shelved its pioneering stem cell therapeutics programs to focus on cancer drugs in November 2011 as a faster path to revenues. That's been a bumpy course for the company with imetelstat, its first-in-class telomerase inhibitor, yielding poor results against breast cancer but positive results in treating essential thrombocythemia, a chronic blood disorder.
In a complex agreement between a subsidiary of BioTime, Geron, and a private investor, Geron will transfer to BioTime Acquisition Corp. its intellectual property, cell lines, and other assets related to its discontinued human embryonic stem cell programs, including the early-stage clinical trial in patients with acute spinal cord injury, as well as its autologous cellular immunotherapy program.
Following a series of share and warrant transactions, the parties anticipate that Geron stockholders will own approximately 21 percent of BioTime Acquisition Corp., BioTime will own approximately 72 percent, and a private investor will own about 7 percent after an additional $5 million investment in the acquisition entity.
BioTime Acquisition Corp. will pay royalties to Geron on the sale of commercialized products that rely on patents acquired by Geron.
The closing of the transaction is subject to certain approvals by BioTime's shareholders, the completion of registrations with the Securities and Exchange Commission, and other negotiated but non-disclosed closing conditions. The transaction is expected to close no later than the end of September.
What Homer Simpson Can Teach Oncologists About Math
Let’s warm up with a quick arithmetic problem, which I want you to do in your head.
What is one thousand plus forty?
Now add another thousand
And thirty more
Plus twenty
Plus another thousand
And finally, add an additional ten.
What’s the answer? According to Dean Buonomano, in his excellent book Brain Bugs, the majority of people give an answer of four thousand, instead of the correct answer of 3100.
“Most of us can find a face in the crowd faster than we can come up with the answer to eight times seven. The truth is—to put it bluntly—we suck at numerical calculations.”
I discuss this problem of innumeracy in my book Critical Decisions: “Throw enough numbers at people and many will experience negative emotions, emotions that often interfere with their ability to make good choices.”
I focus on these math problems because, for some very important medical decisions, the right choice depends on grasping some basic mathematics. And perhaps nowhere in medical care is there more math—more explicit discussion of numerical probabilities and five-year survival rates—than in the care of cancer patients. Consider this snippet of a hematologist trying to explain that there is a good chance a patient’s leukemia will respond to chemotherapy:
“So if you look at complete cytogenetic response rates in the chronic phase,” the hematologist explains, “it’s about 80%, and if you look at the accelerated phase, it’s about 15%. So, the drug doesn’t work in advanced disease very well. If you look at patients who get a complete cytogenetic response as their best response in the Iris trial, their risk of ever progressing in the next 4 years, so about 48 months roughly, is about 8% overall.”
I re-enact a bit more of this conversation in one of my book-related videos, which you can find here. But let it suffice to say for now that this visit goes on for quite a while, with a staggering number of numbers being thrown at this patient. But even in this short paragraph, the hematologist moves rapidly from response rate percentages to progression percentages—in the former, high numbers being good, in the latter, high numbers being bad. A very confusing switch around for patients to follow, in the context of what is no doubt an emotional clinical encounter.
Which raises a question: Why all this talk about math with patients? Shouldn’t all this number stuff be handled by physicians? Shouldn’t chemotherapy decisions be made by medical experts?
As it turns out, many such decisions depend strongly on patient preferences. Suppose that chemotherapy increases a patient’s odds of five-year survival from 30% to 35%, but that same chemotherapy also increases the chance that person will die in the next six months, from treatment complications. Should the patient take this chemotherapy? There is no simple medical answer to this question. The answer depends on this individual patient’s preferences for long-term versus short-term survival, with additional consideration for the burdens of chemotherapy. Making the right decision often means coming to grips with some math.
In future posts, I will show you some of the work I have done to make this math easier to understand. But meanwhile, let me leave you with another quote from Brain Bugs:
“We may have an inherent sense of the quantities one through three, but beyond that things get hazy—you may be able to tell at a glance whether Homer Simpson has two or three strands of hair, but you’ll probably have to count to find out whether he has four or five fingers.”
Only one adequate response I can give to that, as a physician who no doubt has overwhelmed too many of my patients with too much complicated math:
“D’OH!”
http://www.forbes.com/sites/peterubel/2013/01/11/what-homer-simpson-can-teach-oncologists-about-math/
New and improved Biotech Investors Network... check out new charts and stock data sections...everything needed for biotech investing...
http://www.biotechinvestorsnetwork.com/
Pfizer May Push $5 Billion Breast Cancer Hope for Ruling
By Drew Armstrong - Jan 10, 2013 4:05 PM ET
Pfizer Inc. (PFE), the world’s largest drugmaker, is weighing whether to ask U.S. regulators to expedite their review of a potential $5 billion-a-year treatment to slow a type of incurable breast cancer.
The therapy, called PD 0332991, generated excitement among oncologists in December after data showed the drug stopped disease progression for more than two years in 165 patients. That trial was done in the second of three stages of research regularly required for Food and Drug administration approval.
The FDA has cleared cancer drugs for sale before final trial results are completed, including New York-based Pfizer’s Xalkori for lung tumors in 2011 and Novartis AG (NOVN)’s Gleevec for leukemia a decade earlier. While no company decision has been made to push for early clearance, “There are some precedents for approval after Phase 2,” said Mikael Dolsten, Pfizer’s head of worldwide research.
“We’ll see whether there are ways to accelerate availability of the drug for patients,” Dolsten said in an interview at the JPMorgan Chase & Co. health conference in San Francisco. “We’re obviously open to dialogues and advice from FDA and the European agencies.”
Pfizer climbed 1.1 percent to $26.76 at the close of New York trading. The shares have increased 22 percent in the last 12 months.
The drugmaker hopes to start the final-stage trial by the end of March, said Geno Germano, who heads the company’s oncology and specialty care businesses. The medicine is the first in a new class of agents that works by blocking a protein critical in the cancer cell cycle, lead researcher Richard Finn said in December.
Metastatic Disease
The study unveiled in December included women with tumors fueled by the hormone estrogen, the most common type of breast cancer. All had metastatic disease, which had spread to other parts of the body and is no longer considered curable. Almost 230,000 women in the U.S. will be diagnosed with breast cancer this year, and more than 39,900 will die from it, according to the American Cancer Society.
If cleared, Pfizer’s drug may generate as much as $5 billion in yearly sales, wrote Andrew Baum, an analyst at Citigroup Inc., in a Nov. 29 note to investors. That comes as Pfizer is seeking new treatments to replace the $10 billion in losses from its cholesterol medicine Lipitor, the world’s best- selling pill with $13 billion in sales before losing market exclusivity.
“By the end of this year, we’re going to have the Phase 3 enrolling, we’re going to have two Phase 2s done, and we’re going to be talking to the agency about getting the product to patients,” Germano said in an interview at the conference.
Data Available
The positive Phase 2 results, which showed the drug was safe, may give Pfizer much of the data it could use to justify seeking an early ruling, even without the larger number of patients usually tested in a late-stage study, Dolsten said.
“The patients tolerate it very, very well, and that brings the two components of benefit and risk to the table,” he said. The agency has been “very thoughtful when it comes to making those judgments on when they can accelerate access of drugs and approve them on a smaller dataset than a traditional approval.”
Baum, the Citigroup analyst, said in his note that Pfizer is already at least two years ahead of its closest competitors, Eli Lilly & Co. (LLY) and Novartis, with similar cancer drugs in development.
To contact the reporter on this story: Drew Armstrong in New York at darmstrong17@bloomberg.net;