NeoStem And Neuralstem: 2 Early Regenerative Medicine Winners

Back in April in my article titled, "Regenerative Medicine's Time Has Come", I covered two very interesting companies focused on this field: NeoStem Inc. (NBS) and Neuralstem Inc. (CUR).

Since the writing of the article, these stocks have gained the following:

CUR $1.20 to a high of $1.79 for a 49% gain

NBS $6.40 to a high of $8.90 for a 55% gain

As you can see, these stocks have outperformed the market in a big way. Since the writing of the article, there have been even more positive developments for both stocks, so today I will go over them in this update. NBS has four distinct divisions: Amorcyte, Athelos, PCT, and VSEL Technology. The focus is cardiovascular, autoimmune disorders, and regenerative medicine. There is also a new focus for NBS, intellectual property. Patents have become the buzzword in investments over the last several months so it is very good to see NBS is pursuing this possible revenue source.

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On July 16 the company performed a 1 for 10 reverse stock split. This helped to dramatically reduce the outstanding shares of NBS and has, in part, helped to bring the stock to the attention of both investors and institutions. Also helping was the move from the NYSE to the Nasdaq market place. Average dollar trading volume has picked up nicely since the move, making it more appealing to investors that look for liquidity in stocks they own and trade.

In August NBS also hired an investment banker to be its new CFO. Robert Dickey IV has 15 years of management experience at life sciences companies. He has specific expertise in financing, M&A, partnering/licensing transactions and project management, as well as international experience. The hiring of Dickey could signal the beginning of a new stage in the evaluation of NBS and also bring much needed institutional attention to the company.

Since my last article there has also been some key updates from NBS. First up, we have the earnings report from August:

Revenues were $4.4 millionNet losses from continuing operations were $8.6 million$14.7 million in cashSecured $4.6 million in grantsProgenitor Cell Therapy generated a 73% increase in revenues from Q1 2013Market cap of $150mFloat 17.1m sharesInsiders own 15.8%

PCT offers its clients and NeoStem cell processing and development capabilities on both the East and West Coasts of the U.S. and is pursuing plans to expand internationally. Per the last report, NBS also signed a contract with " a large pharmaceutical company that is entering the cell therapy sector." Potential news on what company this is could positively impact the stock.

Most investors are unaware that NBS has a partnership with the Vatican that was made back in 2011. The goal of this deal is to promote the use of adult stem cells for treating disease rather than focusing research on embryonic stem cells. Back in August, NBS stated that its chief financial officer was named to the new position of vice president of strategic initiatives and mentioned this Vatican deal, perhaps signaling some progress or potential news on this in the near future.

Amorcyte is the lead product of NBS and the one I focused on the most last time. In the last few months, we have had some updates. Enrollment has continued in its PreSERVE Phase 2 clinical trial with 120 patients infused as of August 8, 2013. The trial is on track to complete patient enrollment for this trial in 2013 with data read out 6-8 months after the last patient is infused.

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The leading cause of death continues to be heart disease, as you can see from the chart below. The market potential for NBS just on this indication could be huge.

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NBS is presenting at many conferences in the month of September, which should bring more investor and institutional interest to the stock. The company may also release news too. The new CFO is definitely putting his investment banker talents to work getting interest in the company. Here is the list of the eight conferences NBS is attending.

CUR is a company focused on the development of treatments for central nervous system diseases based on human neural stem cells. Its stem cell technology enables the isolation and expansion of human neural stem cells from various areas of the developing human brain and spinal cord. The key update for CUR over the last several months has been its spinal cord research and this has driven the stock up nicely during that time.

CUR is expected to begin its NSI-566 ALS Phase II trial in September at both Emory and the University of Michigan. The trial, with generous funding from the National Institutes of Health and the ALS Association, will focus on cervical area injections and an increase in both the number of injections each patient receives, as well as the number of cells in each injection. The ALS trial will treat eight patients with T2-T12 complete paralysis. The primary endpoints of the trial are to demonstrate safety and toxicity of NSI-566 to treat chronic spinal cord injury. The other key development in the last quarter was NSI-189 that also advanced in the second quarter. The FDA approved the third and final cohort in the major depressive disorder Phase Ib trial, and dosing has nearly been completed. The company expects data from the trial to be available in the second half of the year so news could come at any time sending shares higher, if results are positive.

Recently the company announced that it is working with the National Football League Alumni Association (NFLAA) to develop a trial for treating NFL alumni members suffering from traumatic brain injuries (TBI), with NSI-189, the lead compound in the company's neurogenic drug platform. NSI-189 is currently in a Phase Ib clinical trial to treat major depressive disorder (MDD). This news from a few weeks ago should get more attention given the news last week that the NFL settled with the NFL players for $765m, with some of that money being set aside for research. Given the earlier partnership announced by CUR, it is reasonable to assume CUR should see some potential funding for this trial in the near future. Also, the high profile nature of news like this also helps to bring more institutional interest into small cap stocks like CUR.

On Thursday CUR raised $4m in financing at $1.60 per share. The risk on CUR, aside from bad trial results, is of course the constant financing the company does. However, on a positive note the recent financing actually saw shares of CUR increase so that can be viewed as a positive.

CUR continues to roll out positive news on its spinal cord pipeline. It also has a possible funding deal coming funded by the NFL. Developments on these could help the stock move nicely higher in the coming year.

In June, NBS was given a price target of $25 per share. That is 300% higher than the current price of the stock, giving potential investors here tremendous upside potential. NBS is still an unknown stock to much of the investing world. The new CFO has a strong background in the investment-banking world and is starting to put that knowledge to work. As more and more institutions and investors discover NBS, the stock price will respond. Fund ownership is only 6.6% of NBS. With a very small float now of only 17m, once the stock gains attention, the accumulation should push shares much higher. Investors taking a serious look at NBS should also view its investor presentation PowerPoint slide for more information.

I will look back again in a few months to see how NBS and CUR have performed. Perhaps both stocks will see a similar 50% move.

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article. (More...)

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NICE does not recommend Xalkoriq (crizotinib) - a new advance in 'personalised medicine' for lung cancer patients

Main Category: Lung Cancer
Also Included In: Regulatory Affairs / Drug Approvals
Article Date: 16 Aug 2013 - 2:00 PDT Current ratings for:
NICE does not recommend Xalkoriq (crizotinib) - a new advance in 'personalised medicine' for lung cancer patients

Pfizer Ltd has expressed concern and disappointment that the final appraisal determination (FAD) from the National Institute of Health and Care Excellence (NICE) does not recommend Xalkori (crizotinib) for previously treated, ALK positive advanced non small cell lung cancer (NSCLC). [1] Whilst NICE has acknowledged that crizotinib may offer eligible patients better outcomes compared to standard chemotherapy, it has not been recommended for use within the NHS because NICE does not consider it to be cost- effective.[1]

Pfizer is concerned about the impact this decision will have on eligible patients with previously treated NSCLC, whose tumours have been identified as ALK positive. As a personalised medicine, crizotinib allows targeted treatment of a specific group of patients who are most likely to benefit. In reality, the UK's limited and slow-paced adoption of innovative medicines such as crizotinib poses a real threat to both the Government's goal to have UK cancer outcomes among the highest in Europe and its vision to make the UK a world leader in life sciences.

Dr Michael Peake, Clinical Lead, National Cancer Intelligence Network and Consultant Physician, University Hospitals of Leicester, commented: "As someone who cares for lung cancer patients on a regular basis, I am personally very saddened by this decision. Advanced lung cancer is an aggressive disease with very poor outcomes for many patients. Clinicians recognise the urgent need for personalised medicines which target the specific drivers of an individual patient's tumour. However, if patients are unable to routinely access such therapies, it leaves them at risk of potentially poorer outcomes than patients in other countries where they have access to personalised medicine."

Pfizer believes that the health technology assessment system makes it increasingly difficult for innovative medicines to be accepted for use within the NHS and available for the benefit of patients. During the appraisal process, NICE accepted that crizotinib offered patients a 'noteworthy' improvement in progression free survival and the number of patients responding to treatment was 'very high' for a second or later line therapy in this difficult-to-treat cancer. [4,1] Furthermore, NICE acknowledged that crizotinib was likely to extend patients' lives, but it was uncertain for how long.[4,1] This was due, in part, to uncertainty in estimating the magnitude of overall survival benefit attributed to crizotinib.[4,1]

Like many clinical trials for oncology medicines, the crizotinib trial was designed, for ethical reasons, to give patients allocated chemotherapy the opportunity to receive crizotinib once their cancer had progressed. This factor makes it difficult to compare the differences in overall survival between the two arms of the trial, because the majority of patients will have received crizotinib.

Following NICE's decision not to recommend crizotinib the only other route for patients with previously treated, ALK positive advanced NSCLC to access the treatment is through the National Cancer Drugs Fund (CDF).[5] However the CDF is only available to patients in England, treatments are not guaranteed to stay on it and the future of its existence remains unknown. As such, a NICE recommendation still remains the optimal way to ensure all eligible patients in England and Wales have routine access to a second line therapy. In addition, approval by NICE may improve the uptake of diagnostic testing, a necessary step in the treatment pathway for many targeted medicines. Dr David Montgomery, Medical Director, Pfizer Oncology UK commented: "The Government's strategy for personalised care in cancer includes treating people with medicines targeted at the specific characteristics of their cancer. Yet crizotinib has not been well served by the current assessment models employed by NICE. Today's decision is another example of NICE declining a medicine which we strongly believe is a clinically and cost effective treatment. If this trend of negative decisions continues, we could see the UK fall even further behind other European countries for cancer survival rates".

Dr Jesme Fox, medical director of Roy Castle Lung Cancer Foundation, said: "We are very disappointed that lung cancer patients in the UK will be denied a new therapy, which is routinely available in other parts of the world. [...] It is clear that clinically this is a good drug, which would benefit some lung cancer patients. I would urge the drug's manufacturer and NICE to urgently come together to discuss the price issue and ensure this is routinely available to all lung cancer patients who would benefit."

Lung cancer is the most common cause of cancer death in the UK, accounting for more than a fifth of all cancer deaths.[6] Around 41,500 new cases of lung cancer are diagnosed in the UK every year.[7] NSCLC accounts for about 85 percent of lung cancer cases and remains difficult-to-treat, particularly in the metastatic (or advanced) disease setting.[8] Approximately 70 percent of NSCLC patients are diagnosed late with metastatic disease,[3] where the five year survival rate is only one to five percent.[9] Around 1 in 20 people (5 percent) with NSCLC have the genetic alteration that results in production of the ALK fusion protein.[3] Approximately 550 patients would be eligible for crizotinib treatment each year.[10]

Crizotinib is an oral therapy, and the first of a new class of treatments known as anaplastic lymphoma kinase (ALK) inhibitors. [11] The ALK fusion protein has been identified as a therapeutic target in lung cancer. It is a protein expressed by cancer cells and a direct and potent driver of the ALK-positive variant of this disease. By inhibiting the ALK fusion protein, crizotinib blocks signalling in a number of cell pathways that are believed to be critical for the growth and survival of cancer cells. This can lead to growth inhibition or regression of cancers.[12,13] Crizotinib is licensed for the treatment of patients with previously treated ALK-positive advanced non-small cell lung cancer (NSCLC).[11]

Crizotinib was granted a conditional licence in December 2012 based on data from two multi-centre, single-arm studies, including a Phase 2 study and a Part 2 expansion cohort of a Phase I study. The primary endpoint in both studies was Objective Response Rate (ORR) (defined as complete response plus partial response), and ORRs of 53 percent and 60 percent were seen in the Phase 2 and Phase 1 studies respectively.

Pfizer is in the process of submitting the results of a confirmatory phase 3 trial to regulators with a view to gaining a normal licence for the medicine.

The most common adverse reactions observed in crizotinib Phase I and II studies were vision disorders (visual impairment, vision blurred, vitreous floaters and visual field defect) and gastrointestinal disorders (nausea, diarrhoea, vomiting and constipation). The most frequently reported Grade 3-4 adverse reactions included increased liver enzyme (ALT). Liver enzyme elevations were frequently reported and led to permanent discontinuation of the study drug. Additional cases of liver disorders have been reported from studies 1005 and 1007, including Hy's law cases and death. As expected with some tyrosine kinase targeting drugs, crizotinib has been associated with study-drug related pneumonitis/ILD (including pneumonitis with fatal outcome). Cardiac disorders including QT prolongation, bradycardia, syncope, dizziness, and sudden death have also been observed during the conducted studies. Most of the decrease in neutrophil count was mild in severity, however, grade 3 and grade 4 neutropenia were common.

It is only possible to detect whether a tumour is ALK-positive by performing laboratory tests on a sample of a patient's tumour tissue. For a patient to be eligible for crizotinib, their tumour must have tested positive for an ALK re-arrangement using an accurate and validated test, undertaken by appropriately trained technicians with access to the required technology.

Current ALK testing methods in NSCLC include the following:

Fluorescence In-Situ Hybridization (FISH) FISH provides researchers with a way to visualise and map the genetic material in individual cells, including specific genes or portions of genes. This is important for understanding a variety of chromosomal abnormalities and other genetic mutations. Immunohistochemistry (IHC) IHC uses antibodies that are specifically directed against particular protein structures. An enzyme coupled to the antibodies catalyzes a colour reaction which facilitates the detection of the particular protein under the light microscope. Polymerase Chain Reaction (PCR) PCR is a technique widely used in molecular biology. It derives its name from one of its key components, a DNA polymerase used to amplify a piece of DNA by in vitro enzymatic replication. Article adapted by Medical News Today from original press release. Click 'references' tab above for source.
Visit our lung cancer section for the latest news on this subject.

1 National Institute of Health and Care Excellence (NICE) Crizotinib for the treatment of previously treated non-small-cell lung cancer associated with an anaplastic lymphoma kinase fusion gene Final Appraisal Determination. Available at http://www.nice.org.uk

2 National Institute of Health and Care Excellence (NICE) Pathways. Treatment for non small cell lung cancer. Available at: http://pathways.nice.org.uk - treatment-for-non-small-cell-lung-cancer.xml Last accessed August 2013

3 National Institute of Health and Care Excellence (NICE) Crizotinib for the treatment of previously treated non-small-cell lung cancer associated with an anaplastic lymphoma kinase fusion gene Final scope. Available at http://www.nice.org.uk/nicemedia/live/13639/60925/60925.pdf Last accessed August 2013

4 National Institute of Health and Care Excellence (NICE) Crizotinib ACD, available at: http://guidance.nice.org.uk/TA/Wave28/3/Consultation/DraftGuidance Last accessed August 2013

5 NHS England. The Cancer Drugs Fund Available at http://www.england.nhs.uk/wp-content/uploads/2013/07/ncdf-list.pdf Last accessed August 2013

6 Cancer Research UK. Lung cancer mortality statistics. http://www.cancerresearchuk.org/cancer- info/cancerstats/types/lung/mortality/uk-lung-cancer-mortality-statistics Last accessed August 2013

7 Roy Castle Lung Cancer Foundation. Lung Cancer Facts and Figures. http://www.roycastle.org/lung-cancer/Lung-Cancer- Facts-and-Figures Last accessed August 2013

8 Reade CA, Ganti AK. EGFR targeted therapy in non-small cell lung cancer: potential role of cetuximab. Biologics 2009;3:215- 224

9 American Cancer Society. Lung cancer (non-small cell): Non-small cell lung cancer survival rates by stage. http://www.cancer.org/cancer/lungcancer-non-smallcell/detailedguide/non-small-cell-lung-cancer-survival-rates Last accessed August 2013

10 National Institute of Health and Care Excellence (NICE) Crizotinib for the second line treatment of ALK positive non-small cell lung cancer. STA submission. Available at http://guidance.nice.org.uk/TA/Wave28/3/Consultation/EvaluationReport/ManufacturerSubmissions/Pfizer/pdf/English Last accessed August 2013

11 Xalkori SPC

12 Chiarle R, Voena C, Ambrogio C, et al. The anaplastic lymphoma kinase in the pathogenesis of cancer. Nat Rev Cancer 2008;8(1):11-23

13 Zou HY, Li Q, Lee JH, et al. An orally available small-molecule inhinitor of cMET, PF2341066, exhibits cytoreductive antitumor efficacy through antiproliferative and antiangiogenic mechanisms. Cancer Res 2007;67:4408-4417

Pfizer Ltd

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Greater success in personalized medicine class by students who underwent genetic testing

Main Category: Medical Students / Training
Also Included In: Genetics
Article Date: 25 Jul 2013 - 0:00 PDT Current ratings for:
Greater success in personalized medicine class by students who underwent genetic testing

Students who had their genome tested as part of a groundbreaking medical school course on personalized medicine improved their knowledge of the class materials by an average of 31 percent compared with those who didn't undergo the testing, according to a study by researchers at the Stanford University School of Medicine.

While the sample size was small - 23 students sent their saliva to a commercial genetics testing company; eight did not - the results may encourage educators to consider this approach in the future, the authors said.

"These results indicate that learning principles of human genetics is more powerful, and learning is more sustained, when exploring your own data," said Keyan Salari, MD, PhD, a former Stanford student who initially proposed the course, called "Genomics and Personalized Medicine." Salari, who is the lead author of the study, is now a urology resident at the Massachusetts General Hospital in Boston. The study was published in PLOS ONE.

The eight-week elective course was the first in the country to give students in advanced-degree programs the option of personal genotyping as part of the curriculum. It was designed to teach them how the explosion of knowledge about genetics over the past 10 years could affect the treatment of patients. Since the course was first offered in 2010, the use of genetic testing in clinical care has grown.

The course, which is still being taught, was designed as a way to train future doctors and scientists in the skills necessary to use this new tool. The study, which was based on a pre- and post-course survey taken voluntarily by the majority of the students in the class, also showed that personal testing and the use of personal genotype data in the classroom did not appear to cause significant anxiety.

"This was a novel teaching approach," said Kelly Ormond, co-author of the study and associate professor of genetics. "There is always a lot of interest in whether personalized learning can influence education. ... What our study shows is that it might have benefits for some self-selected students, and is worthy of cautious consideration."

Initially controversial, the course was only approved after a campus task force met regularly for a year to debate the pros and cons of students undergoing genetic testing as part of a class. A number of concerns were raised, including the possible anxiety of learning they could be more susceptible to certain diseases, such as diabetes or Parkinson's. A number of safeguards were subsequently included as part of the course plan, including complete anonymity as to which students chose to undergo testing.

Salari conceived of the idea for the course in 2009 as a PhD student in genetics. He was working as a teaching assistant in the first-year human genetics course for medical students. At the time, the course curriculum consisted primarily of traditional genetics and didn't reflect the genomics revolution of the past 10 years. Salari had also recently undergone his own genetic testing, and saw the educational benefits.

"I was curious about what stories were hidden in my genome, what health risks, what responses to drugs that might be predicted," Salari said. "For instance, I learned I might have a higher risk for age-related macular degeneration. That led me to read and learn a lot more about the genetics of that disease than I probably would have otherwise."

He added: "I wanted to find a way to translate my passion for genomics to all these medical students."

Study results also showed that 83 percent of students who chose to undergo testing were pleased with their decision. Seventy percent of those who underwent the testing reported a better understanding of human genetics on the basis of having undergone testing. The post-course survey also asked students who underwent the testing whether they made any behavioral changes based on the results, such as lifestyle changes or making appointments with doctors. Some initial behavior changes were reported. Yet in a previous study involving face-to-face interviews with the same students, no behavior changes were reported six months after the end of the course.

Article adapted by Medical News Today from original press release. Click 'references' tab above for source.
Visit our medical students / training section for the latest news on this subject.

Other Stanford authors included Konrad Karczewski, a bioinformatics PhD student, and Louanne Hudgins, MD, professor of pediatrics and of medical genetics.

Stanford University Medical Center

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