Compassionate Use of Pluristem's PLX Cells Saves the Life of a Child After Bone Marrow Transplantation Failure (PSTI)

Pluristem Therapeutics Inc. (NasdaqCM: PSTI; TASE: PLTR) is a leading developer of placenta-based cell therapies. The company's patented PLX (PLacental eXpanded) cells drug delivery platform releases a cocktail of therapeutic proteins in response to a variety of local and systemic inflammatory diseases. PLX cells are grown using the company’s proprietary 3D micro-environmental technology and are an off-the-shelf product that requires no tissue matching or immune-suppression treatment prior to administration. PLX-PAD comprehensive clinical development plan has been recognized by both the EMA and FDA, targeting a sub-population of 20-million patients of Peripheral Artery Disease (PAD) market. 

The Company has announced that "a seven year-old girl suffering from an aplastic bone marrow whose condition was rapidly deteriorating is now experiencing a reversal of her condition with a significant increase in her red cells, white cells and platelets following the intramuscular injection of the company's PLacental eXpanded (PLX) cells."

Patients with Aplastic bone marrow have no blood-forming hematopoietic stem cells in their bone marrow.

Professor Reuven Or, Director of Bone Marrow Transplantation, Cell Therapy and Transplantation Research Center at Hadassah Medical Center and the child's physician, explained, 

"With her body rejecting all possible treatment -- and with no other options -- we finally turned to Pluristem's PLX cells, which literally saved her life. The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow."

Zami Aberman, Chairman and CEO of Pluristem, commented,

"Pluristem is extremely happy that our PLX cells have helped this little girl. Remarkably, these beneficial effects were seen in the patient after our PLX cells were administered intramuscularly and correlates with the positive effects on the bone marrow when we administered our PLX cells intramuscularly (IM) in animals exposed to toxic levels of radiation. Pluristem now has several data points to indicate that our PLX cells may work for systemic diseases when given locally, away from the target organ, and without a need to give cells intravenously."

In February 2012, Pluristem announced the results of animal studies which suggest that "PLX cells can be potentially effective in treating the life threatening hematopoietic complications associated with Acute Radiation Syndrome (ARS)." In the experiments in question, animals were given PLX cells IM up to 24 hours post irradiation. They demonstrated a recovery of their red cells, white cells, platelets and bone marrow to almost normal levels. This announcement, along with his patient's significant deterioration following two bone marrow transplants, that led Professor Reuven Or to contact Pluristem about the possible compassionate use of PLX cells to treat his young patient.

Pluristem recently received U.S. FDA Clearance "to begin a Phase II clinical trial using the company's proprietary PLX-PAD cell product candidate intramuscularly for the treatment of Intermittent Claudication (IC), a subset of peripheral artery disease (PAD). In April, the Company was awarded a $3.1 Million grant by the Israeli Government, which will be used to help fund R&D and clinical trials."

This news could have positive implications for Athersys who is planning to pursue a phase II registrational style trial in GvHD prevent. There are also implications for Osiris. 

Read the full press release at Yahoo Finance. 

Islet Sciences Announces Exclusive License Agreement with Yale University to Commercialize an Early Beta Cell Destruction and Diabetes Diagnostic ($ISLT)

Islet Sciences, Inc. (OTCBB: ISLT) is a development-stage biotechnology company with patented technologies focused on transplantation therapy for people with insulin-dependent diabetes. The Company's transplantation technology includes methods for the culturing, isolation, maturation, and immuno-protection (microencapsulation) of islet cells. Islet Sciences’ mission includes the introduction of commercial products with applications to cell-based replacement therapy in the healthcare marketplace.

 The Company announced that it has exclusively licensed technology from Yale University.

Chairman and CEO of Islet Sciences John Steel stated, 

"In the course of research conducted under Yale auspices, Dr. Kevan Herold and his colleagues at Yale University School of Medicine have developed a technology entitled 'Circulating hypomethylated B cell-derived DNA' as a biomarker of B cell destruction. We are excited to utilize this groundbreaking invention as we look to commercialize it to benefit the growing worldwide diabetic community. This test has the potential to identify beta cell death and emerging diabetes far earlier than clinical presentation."

Dr. Kevan Herold added, 

"The opportunity to identify diabetes and islet dysfunction prior to clinical presentation could be beneficial therapeutically. I am looking forward to working with Islet Sciences to commercialize this technology that clearly holds so much potential."

Clusters of beta cells form islets within the pancreas. Diminished beta/islet cell function is one of the primary causes of insulin dependent diabetes and can be caused by numerous factors. Insulin dependent diabetes is the more difficult aspect of the disease.

Jonathan Soderstrom, director of the Office of Cooperative Research at Yale commented,

"We are pleased that Islet Sciences has made the decision to invest in this discovery and look forward to developing it into a valuable new tool for treating this debilitating disease."

Read more at Yahoo Finance. 

3-D printing, bioprinting pushing the edges of regenerative medicine ($ONVO)

Organovo Holdings, Inc. (OTC: ONVO) is focused on delivering breakthrough three-dimensional biology capabilities to create tissue on demand for research and surgical applications. As the first company with a three-dimensional tissue technology that works across tissue types, we are meeting the promise of regenerative medicine to fill unmet medical needs. Organovo's powerful NovoGen Bioprinting platform creates human tissues starting with any cell source. From disease models to tissue creation, bioprinting solves urgent needs in biological research. 

On Cleveland.com, contributor Brie Zeltner wrote an article about the idea behind bioprinting, its applications, and some the companies and researchers working on it. She writes,

"Imagine a custom-made knee replacement modeled to fit exactly from scans of your own bone, built one infinitesimal layer at a time by a printer that uses 'ink' made from living cells. Or a replacement heart valve, seeded with human stem cells, printed in a single functioning piece. It may sound like science fiction, but these feats of bioengineering are not far from being realized."

She continues,

"Organovo Inc., a San Diego-based tissue engineering company, is using printers to make small amounts of human tissue, including muscle and blood vessels. With the printer precisely placing cells, the team is able to create tissue that is almost indistinguishable from the real thing under a microscope. And pharmaceutical companies are using the printed tissue to test their drugs, saving money by identifying ones that would fail early in clinical trials. The collaboration will fund their continued research."

Read the full article at Cleveland.com. 

@ BioWorld, Islet Sciences' subsidiary receives $2.1 million in grant funding (ISLT)

Islet Sciences, Inc. (OTCBB: ISLT) is a development-stage biotechnology company with patented technologies focused on transplantation therapy for people with insulin-dependent diabetes. The Company's transplantation technology includes methods for the culturing, isolation, maturation, and immuno-protection (microencapsulation) of islet cells. Islet Sciences’ mission includes the introduction of commercial products with applications to cell-based replacement therapy in the healthcare marketplace.

Islet Sciences was mentioned in the "Other News to Note" section of BioWorld Today, the daily biopharmaceutical news source:

"Islet Sciences Inc., of New York, said subsidiary DiaKine Therapeutics Inc. received a $2. 1 million in grant funding, comprising about $1 .83 million from the National Institutes of Health and $250,000 from the Iacocca Foundation to support its work in diabetes. Islet acquired DiaKine in a stock deal last month."

About BioWorld

BioWorld Today delivers actionable intelligence on the biotech science that drives the business, which funds the means to heal disease. With writers and editors stationed around the globe, BioWorld Today reports the breaking news -- and provides key perspective -- on hundreds of medicines in development, the companies behind those therapeutic candidates, the business development transactions that evolve the market, and the regulatory hurdles that both challenge and guard the process. Read more at BioWorld.com.

Pluristem Awarded a $3.1 Million Grant by Israeli Government (PSTI)

Pluristem Therapeutics Inc. (NasdaqCM: PSTI; TASE: PLTR) is a leading developer of placenta-based cell therapies. The company's patented PLX (PLacental eXpanded) cells drug delivery platform releases a cocktail of therapeutic proteins in response to a variety of local and systemic inflammatory diseases. PLX cells are grown using the company’s proprietary 3D micro-environmental technology and are an off-the-shelf product that requires no tissue matching or immune-suppression treatment prior to administration. PLX-PAD comprehensive clinical development plan has been recognized by both the EMA and FDA, targeting a sub-population of 20-million patients of Peripheral Artery Disease (PAD) market. 

The company announced that its wholly owned subsidiary, Pluristem Ltd., has been approved for a 11.8 million New Israeli Shekels (approximately $3.1 million) grant from the Office of the Chief Scientist (OCS) within the Israeli Ministry of Industry, Trade and Labor. The grant, once received, "will be used to cover R&D expenses for the period March to December 2012. According to the OCS grant terms, Pluristem Ltd. is required to pay royalties in the rate of 3% - 5% on sales of products and services derived from technology developed using this and other OCS grants until 100% of the dollar-linked grants amount plus interest are repaid. In the absence of such sales, no payment is required."

The OCS was empowered by the Law for the Encouragement of Industrial Research & Development -- 1984. It "oversees all Government sponsored support of R&D in the Israeli hi-tech and bio-tech industries. This broad-spectrum support stimulates the development of innovative state-of-the-art technologies, enhances the competitive power of the industry in the global hi-tech market, creates employment opportunities and assists in improving Israel's balance of payments."

Chairman and CEO of Pluristem Zami Aberman stated,

"We are pleased Pluristem's PLX cells were recognized as an innovative state-of-the-art technology with a potential to create long term sustainable competitive advantage in the cell therapy industry. This grant will assist the company in enhancing its R&D plans and clinical trials, helping us bring the PLX product candidates to market for the treatment of millions of patients around the world." 

Read more at Yahoo Finance. 

Gilead Sciences Announces First Quarter 2012 Financial Results ($GILD)

Gilead Sciences, Inc. (Nasdaq: GILD) is a research-based biopharmaceutical company that discovers, develops and commercializes innovative medicines in areas of unmet medical need. With each new discovery and experimental drug candidate,  they seek to improve the care of patients suffering from life-threatening diseases around the world. Gilead's therapeutic areas of focus include HIV/AIDS, hepatitis, serious respiratory, cardiovascular, and metabolic conditions, cancer and inflammation. Their portfolio of 14 marketed products includes a number of category firsts, including the only complete treatment regimens for HIV infection available in a once-daily single pill – Atripla® (efavirenz 600 mg/emtricitabine 200 mg/tenofovir disoproxil fumarate 300 mg), approved in 2006, and Complera® (emtricitabine/rilpivirine/tenofovir disoproxil fumarate), approved in 2011.

Gilead Sciences has announced their first quarter financial results for 2012.  Total revenues for the first quarter of 2012 increased 19 percent to $2.28 billion,  with a net income of $442.0 million.  The increased revenue is attributed to product sales driven by Gilead's antiviral franchise, including sales of Atripla^(R), Truvada^(R), and the launch of Complera^(R)/Eviplera^(R) in 2011.

Read the full press release here.

Market watchers have commented on this announcement and the success of The Company's antiviral sales. Brean Murray Carret & Co. commented,

"Gilead’s HIV franchise had a strong quarter, with newly launched Complera

contributing to the strength of core products Atripla and Truvada. However, investor

focus remains on the Hepatitis C opportunity and the development plans for GS‐7977.

We continue to believe Gilead represents a compelling investment opportunity and

see the strong quarter as a reaffirmation of the value of the company’s current suite of

commercial products. We believe further upside will be derived from the approval and

commercialization of GS‐7977 for Hepatitis C next year.  Gilead’s HIV franchise had a strong quarter, with newly launched Compleracontributing to the strength of core products Atripla and Truvada. However, investorfocus remains on the Hepatitis C opportunity and the development plans for GS‐7977.We continue to believe Gilead represents a compelling investment opportunity andsee the strong quarter as a reaffirmation of the value of the company’s current suite ofcommercial products. We believe further upside will be derived from the approval andcommercialization of GS‐7977 for Hepatitis C next year."

Read the Brean Murray full report here.  

Vertex Reports First Quarter 2012 Financial Results and Provides Update on Launch of KALYDECO™ ($VRTX)

Vertex (Nasdaq: VRTX) creates new possibilities in medicine. Their team discovers, develops and commercializes innovative therapies so people with serious diseases can lead better lives. Vertex scientists and collaborators are working on new medicines to advance the treatment of hepatitis C, cystic fibrosis, epilepsy and other life-threatening diseases. Vertex isn’t a conventional pharmaceutical company. Each and every person at Vertex is driven by a passion for fearless innovation and a bold desire to transform the lives of millions with new medicines. Vertex has ongoing worldwide research programs and sites in the U.S., U.K. and Canada and employs over 1800 people.

Vertex has announced their first quarter financial results for 2012.  The Company released that it earned revenues of approximately $439 million, including net product revenues of approximately $357 million from INCIVEK^® (telaprevir) and approximately $18 million from KALYDECO^TM (ivacaftor).  KALYDECO^TM and INCIVEK^®, The Company reports continued investment which will allow them to continue multiple clinical studies including Phase 2b studies of all-oral, short-duration treatment regimens for hepatitis C and JAK3 inhibitor VX-509 for rheumatoid arthritis, as well of KALYDECO in people with types of cystic fibrosis. 

Jeffrey Leiden, M.D., Ph.D., President and Chief Executive Officer of Vertex commented,  

"Since January, approximately 600 people with cystic fibrosis have started treatment with KALYDECO in the U.S., underscoring the importance of this new medicine to the CF community and further demonstrating our ability to develop and launch transformative new medicines. With INCIVEK, we continue to treat thousands of people with hepatitis C, and we are highly encouraged by the early success seen with this medicine outside the U.S., where our collaborator Janssen has attained a market-leading position in Europe and availability of INCIVO in more than 15 countries around the world."

The full press release can be viewed here.  

Market watchers have also commented about these results.  Brean Murray Carret & Co. stated, 

"Vertex launched Kalydeco in 1Q12 and in a little more than two months captured a

large portion of G551D patients.    This is a strong indication that the closely knit CF

community was anxious for a new treatment option and reinforces the potential for a

blockbuster market opportunity if Vertex can expand beyond the G551D patient

population.   Incivek sales were disappointing coming in below our expectations. We

continue to see declining Incivek sales as a drag on the stock despite the positive early

signals from the company’s CF franchise.  Vertex launched Kalydeco in 1Q12 and in a little more than two months captured alarge portion of G551D patients.    This is a strong indication that the closely knit CFcommunity was anxious for a new treatment option and reinforces the potential for ablockbuster market opportunity if Vertex can expand beyond the G551D patientpopulation.   Incivek sales were disappointing coming in below our expectations. Wecontinue to see declining Incivek sales as a drag on the stock despite the positive earlysignals from the company’s CF franchise."

 

Read the Brean Murray full report here.  

Athersys ($ATHX) holds meeting with FDA to discuss MultiStem

Athersys announced that a scheduled meeting with the FDA was held to discuss the results of its recently completed clinical trial involving the administration of MultiStem to patients being treated for leukemia or other conditions that place them at risk of Graft versus Host Disease (GvHD).

• Additionally, the company discussed with the FDA its proposed plans for the next phase of clinical development, answered questions and obtained feedback on a number of specific issues related to the potential study parameters and proposed trial design.
• The company intends to provide further information about its plans as it finalizes development of a detailed trial and statistical plan and receives additional FDA feedback.