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May 16

MADISON, Wis., May 15, 2012 /PRNewswire/ — Novelos Therapeutics, Inc. (NVLT.OB), a pharmaceutical company developing novel drugs for the treatment and diagnosis of cancer, today announced that it has successfully completed the first cohort in a U.S. multi-center Phase 1b dose-escalation trial of its cancer-targeted molecular radiotherapeutic compound I-131-CLR1404 (HOT) in cancer patients with advanced solid tumors. The first two-patient cohort was successfully dosed with approximately 20 mCi of HOT, triggering enrollment into the second cohort at approximately 40 mCi. Details of the trial design are available on www.clinicaltrials.gov ID: NCT01495663, or at www.novelos.com in the ‘Clinical Trials’ section. Glenn Liu, M.D., University of Wisconsin Carbone Cancer Center, is the trial’s principal investigator. Detailed trial results are expected to be presented at a scientific venue at a later date. “Patients with advanced solid tumors need safer and more effective therapies,” said Dr. Liu. “Based on animal data, results from a completed Phase 1a dosimetry trial, and now initial data from this Phase 1b trial, HOT appears to deliver radiation directly and selectively to cancerous tumors. Data from the first cohort indicates HOT was well-tolerated, without any grade 3 or 4 toxicities, enabling enrollment of the first patient in the second cohort. HOT uptake in cancerous tumors persisted for at least 21 days. One patient with advanced prostate cancer remains on trial at two months following treatment with HOT, while another patient with advanced colorectal cancer has completed the trial.” “We are pleased with HOT’s safety profile and selective cancerous tumor uptake and retention in this first cohort at a dose of approximately 20 mCi,” said Kim Hawkins, Vice President of Clinical Development of Novelos. “We now look forward to evaluating HOT at approximately 40 mCi in cancer patients with advanced solid tumors, as per the trial protocol. We also look forward to adding two additional clinical investigators to the trial, Joanne Mortimer, M.D., at the City of Hope and Michael Pishvaian, M.D., Ph.D., at Georgetown University.” “We intend to combine the data from this trial with calculation of effective doses for HOT based on quantitative positron emission tomography (PET) tumor imaging data using I-124-CLR1404 (LIGHT), our small-molecule cancer-targeted PET imaging agent,” said Harry Palmin, President and CEO of Novelos. “Together, we believe these data will enable us to commence HOT Phase 2 proof-of-concept trials in the first quarter of 2013 in cancer patients that have significant unmet medical need.” About HOTHOT (iodine-131 radiolabeled CLR1404) is a small-molecule, broad-spectrum, cancer-targeted molecular radiotherapeutic that we believe has first-in-class potential. HOT is comprised of a small, non-pharmacological quantity of CLR1404 (COLD) acting as a cancer-targeted delivery and retention vehicle and incorporating a cytotoxic (cell-killing) dose of radiotherapy (in the form of iodine-131, a radioisotope that is already in common use to treat thyroid and other cancer types). The ongoing Phase 1b dose-escalation trial is aimed at determining the Maximum Tolerated Dose of HOT. We expect to initiate HOT Phase 2 efficacy trials as a monotherapy for solid tumors with significant unmet medical need as soon as a minimal efficacious dose is established, subject to additional funding. We may determine such an effective dose upon calculating it from ongoing PET imaging trials in cancer patients with LIGHT (since PET imaging is quantitative, enabling determination of tumor radiation exposure at a given dose level) or seeing a tumor response in the Phase 1b trial. Preclinical experiments in vitro (in cell culture) and in more than a dozen in vivo (in animals) models have demonstrated selective killing of cancer cells along with a benign safety profile. In view of HOT’s selective uptake and retention in a wide range of solid tumors and in cancer stem cells, its single-agent efficacy in animal models and its non-specific mechanism of cancer-killing (radiation), we are first developing HOT as a monotherapy for solid tumors with significant unmet medical need. About Novelos Therapeutics, Inc.We are a pharmaceutical company developing novel drugs for the treatment and diagnosis of cancer. Our three cancer-targeted compounds are selectively taken up and retained in cancer cells, including cancer stem cells, versus normal cells. Thus, our therapeutic compounds appear to directly kill cancer cells while minimizing harm to normal cells. This offers the potential for a paradigm shift in cancer therapy by providing efficacy versus all three major drivers of mortality in cancer: primary tumors, metastases and stem cell-based relapse. I-124-CLR1404 (LIGHT) is a small-molecule cancer-targeted PET imaging agent. We believe LIGHT has first-in-class potential and Phase 1-2 clinical trials are ongoing. I-131-CLR1404 (HOT) is a small-molecule, broad-spectrum, cancer-targeted molecular radiotherapeutic that delivers cytotoxic radiation directly and selectively to cancer cells and cancer stem cells. We believe HOT also has first-in-class potential. HOT Phase 1b dose-escalation trial is ongoing and we expect HOT to enter Phase 2 trials in the first quarter of 2013 as a monotherapy for solid tumors with significant unmet medical need, subject to additional funding. CLR1404 (COLD), a pre-clinical cancer-targeted non-radioactive chemotherapy, works primarily through Akt inhibition. Together, we believe our compounds are able to “find, treat and follow” cancer anywhere in the body in a novel, effective and highly selective way. For additional information please visit www.novelos.com INVESTOR CONTACTSJ. Patrick Genn, Vice President of IR Anne Marie Fields, Senior Vice President Novelos Therapeutics, Inc. LHA


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May 16

SEBASTIAN Toby, a 6-year-old golden retriever, loves to run and play catch. And Oreo, a 12-year-old border collie mix, also is a bundle of energy. Movement for both dogs got easier about a month ago when they received a revolutionary stem cell treatment at the Highlands Animal Hospital. Veterinarian Marcus Kramer performed the successful transplant procedures, which were developed by Kentucky-based MediVet-America. Both dogs had been in significant pain with a restricted range of motion, as shown on X-rays. “It’s made a big difference,” said Kramer. “The really amazing thing is that they both healed so quickly. Both dogs had problems with their hips and were suffering from osteoarthritis. Just 30-days later, they are able to walk and run again.” Adult animal stem cell technology uses the pet’s own regenerative healing power to treat dogs, cats and horses suffering from arthritis, hip dysplasia and tendon, ligament and cartilage injuries. Under anesthesia, Kramer removed about 40 grams of fat from each dog and separated the stem cells from the fat. He then activated the stem cells under an LED light, and injected them back into the dogs. Stem cell therapy allows an animal to get off pain and anti-inflammatory drugs, Kramer said. MediVet-America’s therapy is done entirely at the animal hospital in about three hours, and costs about $1,800 for dogs and $2,400 for horses. That compares to thousands of dollars that pet owners could expect to pay for medication over a pet’s lifetime. Erica Kent, a spokesman for MediVet-America, said using the LED light is integral to the patented-process, because the light helps to awaken stem cells and makes them more active. The three-color light stimulates millions of dormant cells to initiate repair from the moment the cells are injected into the animal’s body, according to the MediVet-America website. The company is also offering a program that allows pet owners to bank stem cells when animals are younger to use if their pet develops illnesses like arthritis in old age. STEM CELL THERAPY


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May 16

CAMBRIDGE, Mass.–(BUSINESS WIRE)– Foundation Medicine, Inc., a molecular information company that brings comprehensive cancer genomic analysis to routine clinical care, today announced that new clinical data highlighting the companys comprehensive cancer genomic profile and next-generation sequencing approach in clinical oncology will be presented at the 2012 Annual Meeting of the American Society for Clinical Oncology (ASCO) being held June 1-5, 2012 in Chicago. The data to be presented at ASCO support Foundation Medicines deep sequencing approach to simultaneously detect all classes of genomic alterations across hundreds of genes known to be related to cancer, said Michael J. Pellini, M.D., president and chief executive officer, Foundation Medicine. In our clinical experience abstract, this approach detected actionable alterations those associated with available targeted treatments or ongoing clinical trials for 74% of tumor samples in the study. Foundation Medicines test has also been shown to identify novel genomic alterations in multiple tumor types, including potentially druggable gene fusions. The combined evidence presented in these studies suggests that fully informative genomic profiling can now become a routine component of cancer patient care. The schedule for Foundation Medicines oral presentation is as follows: Date & Time: Session: Abstract Number: Title: Discovery of recurrent KIF5B-RET fusions and other targetable alterations from clinical NSCLC specimens. Location:


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May 16

Featured Article Main Category: Melanoma / Skin Cancer Also Included In: Dermatology;Cancer / Oncology Article Date: 16 May 2012 – 10:00 PDT Current ratings for: ‘Cell Signaling Breakthrough May Help Melanoma Treatment’ A key discovery made by scientists from the Texas University Health Science Center at Houston (UTHealth) Medical School reveals that cell signaling plays an important role in the fight against melanoma and various other fast-spreading tumors. The study is published online ahead of the June 5 edition of Current Biology. About 9,000 people die each year from melanoma (skin cancer), according to the American Cancer Society. The researchers have now discovered the reason as to why BRaf inhibitors, which are frequently used for the treatment of skin cancers do not always work, and most significantly, how these drugs can possibly speed up the growth of cancer in certain patient populations. Senior author John Hancock, M.B, B.Chir, Ph.D., a John S. Dunn Distinguished University Chair in Physiology and Medicine and chairman of the Department of Integrative Biology and Pharmacology, who is also interim director of the Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases at the UTHealth Medical School declared: A chain of proteins that forms a signaling pathway transmits growth signals from a cell’s surface to the nucleus, whilst the command for dividing cells in order generate new cells is relayed by a chain of four proteins, namely RAS, BRaf, MEK and ERK. This pathway is shared by all cells and is generally very effective, yet difficulties occur when one of the first proteins in the chain is mutated, as both proteins lock the pathway in the ‘on’ position. BRaf inhibitors are drugs that block the signaling from the second protein, and are successful in treating melanomas with mutant BRaf proteins. However, so far, there are no inhibitors available that can block the first protein (RAS). The team conducted in vivo studies to explore what happens when BRaf inhibitors are applied to human cancer tissues with Ras mutations. Kwang-jin Cho, Ph.D., the study’s lead author and research fellow at the UTHealth Medical School declared: The majority of melanomas either have a mutation of the BRaf protein or of the Ras protein, yet they seldom feature both. Mutations of the RAS protein cause an otherwise normal BRaf protein to remain switched ‘on’.


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May 16

LEUVEN, BELGIUM–(Marketwire -05/15/12)- TiGenix NV (TIG) a leader in the field of cell therapy, today gave a business update and announced the financial results for the first quarter ending March 31, 2012. Business highlights Financial highlights “In the first quarter 2012 we continued to aggressively push our commercial efforts forward,” said Eduardo Bravo, CEO of TiGenix. “As a result sales of ChondroCelect are developing in line with the improved traction we observed in the second part of last year. At the same time we are moving ahead of schedule with most of our clinical adipose stem cell programs. We closed the quarter with almost EUR 17 million cash on hand, which is sufficient to execute on our business plan and reach key inflection points.” Business update ChondroCelect sales increase continues apaceThe Company reports net sales growth for the quarter of 123% compared with the same period of last year, and of 62% compared to Q4, 2011, a positive trend reflecting the uptake in Belgium, where we benefit from national reimbursement. In the Netherlands one of the leading private healthcare insurance companies has made treatment with ChondroCelect compulsory for its insured, and no longer reimburses non-ATMP treatments. Similarly, one of the large private insurers in the UK has expressed its intention to routinely reimburse ChondroCelect going forward. Discussions to obtain full national reimbursement keep advancing in the Netherlands, France, Spain and Germany. Positive outcome of ChondroCelect compassionate use program published in leading journalPositive outcome data from the ChondroCelect compassionate use program (CUP), involving 43 orthopedic centers in 7 European countries, treating 370 patients with ChondroCelect over the span of four years, were published in advance online in Cartilage, the official journal of the International Cartilage Repair Society. The data show that the implantation of ChondroCelect results in a positive benefit/risk ratio when used in an unselected, heterogeneous population, irrespective of the follow-up period, lesion size and type of lesion treated. In addition, the CUP study significantly expands the data set used to obtain approval for ChondroCelect from the European Medicines Agency in 2009, increasing eight-fold, from 43 to 334, the number of patients with long-term follow up data. To date almost 700 patients have been treated with ChondroCelect. ADMIRE-CD Phase III trial (Cx601) in complex perianal fistula on schedule The ADMIRE-CD (Adipose Derived Mesenchymal stem cells for Induction of REmission in perianal fistulizing Crohn’s Disease) Phase III protocol was submitted to Ethics Committees or Health Authorities in all 8 participating countries, and to date approvals have been received in four of those countries already. Cx611 Phase IIa in RA passes last safety hurdleOn April 17, upon review of the safety data of the first three patients of the third cohort of the company’s Phase IIa clinical trial in rheumatoid arthritis (Cx611), TiGenix received the go-ahead from the independent Safety Monitoring Board to recruit and dose the remaining patients of this cohort. This fact is of major importance. In RA it ensures that the product will not be held back by any dose-limiting factors and that we will be able to move forward with the optimal treatment dose. Of almost equal importance is that, if required, we can expand the dosing range in other indications that we are exploring as well. With 6 months of follow-up, the current RA trial in 53 patients is expected to report meaningful results in H1 2013. Last patient treated in Cx621 Phase I clinical trialAll 10 healthy volunteers have been recruited and treated in the Phase I study of Cx621. Cx621 investigates the safety and feasibility of intra-lymphatic administration of stem cells. Intra-lymphatic administration of (all) stem cells is patented by TiGenix. The final report of this trial will be available at the end of June.


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May 16

JERUSALEM–(BUSINESS WIRE)– Gamida Cell announced today that it has closed an internal E financing round of $10 million. All major shareholders participated. The financing will be used to support the global commercialization of the companys lead cell therapy product, StemEx, in development as an alternative therapeutic treatment for patients with blood cancers, such as leukemia and lymphoma, who can be cured by bone marrow transplantation but do not have a matched bone marrow donor. The company is currently seeking a strategic partner to join in the global commercialization of StemEx. The financing will also support the continued development of the companys pipeline of products, primarily the NiCord clinical trial for sickle cell disease and thalassemia. Mr. Reuven Krupik, chairman of the board of Gamida Cell said, The investors were unanimous in their decision to reinvest, understanding the importance of bringing StemEx to market as well as maintaining the companys leadership role in the stem cell industry. Gamida Cell is a game changer. The international, multi-center, pivotal registration, Phase III clinical trial of StemEx completed enrollment in February 2012. Clinical outcome is expected in Q4/2012. The market launch of StemEx is planned for 2013. StemEx is likely to be the first allogeneic stem cell product in the market. StemEx is being developed by the Gamida Cell-TEVA joint venture. Dr. Yael Margolin, president and chief executive officer of Gamida Cell said, With the continued support of our shareholders and the analysis of the clinical results of the StemEx trial just around the corner, we are now focused on submitting the BLA. StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining, non-manipulated cells from the same unit. Competing products in development use two units. As the average cost of a cord blood unit in the U.S. is $40K, StemEx is expected to be a significantly less expensive treatment option. StemEx is also expected to be available in the market several years before any of the competing products. About Gamida Cell Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The companys pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, neutropenia and acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cells therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, bone marrow and peripheral blood, which are expanded in culture. Gamida Cells current shareholders include: Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: www.gamida-cell.com.


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May 16

ALACHUA, Fla.–(BUSINESS WIRE)– AxoGen, Inc. (AXGN.OB), a leading regenerative medicine company focused on the commercialization of proprietary products and technologies for peripheral nerve reconstruction and regeneration, today announced revenues for the first quarter ended March 31, 2012 of $1.65 million, a 47% increase over 2011 first quarter revenues of $1.12 million. This quarters record performance has been the direct result of our increase in sales and marketing activity, commented Karen Zaderej, Chief Executive Officer of AxoGen, Inc. During the first quarter we continued to expand our sales force, while continuing to get hospital approval for AxoGen products and training and developing the sales team. Our growing base of sales representatives, combined with increasing surgeon awareness of our technologies and clinical data, creates a strong environment for our continued growth. Revenues Revenues for the period increased to a record $1.65 million, or 47%, compared to $1.12 million in 2011. The improved results were primarily due to an increase in new accounts as well as stronger sales penetration into key accounts. Revenues increased 21% over fourth quarter revenues of $1.36 million. Gross Profit Gross profit reached $1.21 million, a 55% increase, for first quarter 2012 up from $0.78 million reported for the same period 2011. The higher gross profit reflects lower manufacturing and labor cost and the absence of one-time manufacturing startup expenses reported during the first quarter of 2011. The gross profit margin increased to 73% compared to 70% for the same quarter last year. Sales and Marketing Expenses As a result of the Company’s investment in additional sales and marketing resources, sales and marketing expenses during the first quarter of 2011 increased to $1.63 million, compared to $0.86 million reported during the same period last year. As of the end of the period, the Company reported 16 direct and 21 independent sales representatives and distributors. Research and Development Expenses Research and development expenses increased to $0.30 million during the first quarter of 2012. Substantially all of the research and development expenses relate to expenditures for clinical activity. General and Administrative Expenses General and administrative expenses increased to $1.23 million for the quarter, compared to $0.72 million reported last year. This increase was largely driven by payroll and benefit increases and expenses associated with being a public company. Operating Loss The Company reported a net loss of $2.11 million, or $0.19 per common share, compared to a net loss of $2.3 million, or $2.21 per common share, reported during the same period in 2011.


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May 16

NORTHRIDGE, Calif. & CAMBRIDGE, England–(BUSINESS WIRE)– Avita Medical Ltd. (ASX: AVH), (OTC: AVMXF), (OTCQX: AVMXY),the regenerative medicine company, today announced that it has commenced enrolment in the US FDA-approved feasibility study for the use of ReCell Spray-On-Skin in the treatment of hypertrophic dyspigmented scars (raised and/or discoloured scars). The initial three patients were treated by Dr Rajiv Sood, at the Richard M. Fairbanks Burn Center of Wishard Hospital, Indiana University, Indianapolis, Indiana, for scarring resulting from previous grafting due to burn injuries. The approved FDA protocol permits the Company to treat 20 patients with scars at up to four U.S. study sites; patients will be assessed for healing and pain on a weekly basis during the initial four weeks post-treatment; at weeks 12 and 24 the treatment site will be assessed for healing and aesthetic outcomes by both the patient and an independent observer. “Commencement of the FDA scar study is an important milestone for Avita,” said Dr William Dolphin, Avita Medicals CEO. “ReCell has shown the potential to provide significant benefits over current options in the treatment of acute and chronic wounds and for a wide range of skin defects. We are confident that this study will demonstrate the effectiveness of ReCell in the corrective treatment of scars, making ReCell directly applicable and immediately relevant to the very large aesthetic markets. The feasibility study is primarily designed to confirm the effectiveness of ReCell for the treatment of scars in a single session in comparison to the current standard of care involving dermabrasion of the scar and often requiring multiple treatment sessions; study endpoints are time-to-healing and aesthetic outcomes. Following completion of the study, Avita will submit the feasibility data and seek FDA approval for a statistically powered, pivotal clinical trial. The study is funded by the US Department of Defense in partnership with the OSD Manufacturing Technology Program and Rapid Fielding Directorate for the Limb Salvage and Regenerative Medicine Initiative. The contract is a Technology Investment Agreement that is focused on the transition of the capability to meet DoD needs. ReCell was selected as it has the potential to be a quantum advance over the existing ability to treat and re-grow tissue and to substantially reduce the effects and appearance of scarring and thereby profoundly assist in the treatment and rehabilitation of wounded warriors suffering from disfigurement and impeded function due to combat injuries. An interview with Dr Sood regarding the use of ReCell in treatment of scars and acute wounds is available at http://soundmedicine.iu.edu/segment/3245/Spray-on-Skin. ABOUT AVITA MEDICAL LTD. Avita Medical (www.avitamedical.com) develops and distributes regenerative and tissue-engineered products for the treatment of a broad range of wounds, scars and skin defects. The companys lead product, ReCell Spray-On-SkinTM, is used in a wide variety of burns, plastic, reconstructive and cosmetic procedures. ReCell is patented, CE-marked for Europe, TGA-registered in Australia, and SFDA-cleared in China. ReCell is not available for sale in the United States; in the U.S. ReCell is an investigational device limited to investigational use.


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May 16

Regenerative medicine startup Juventas Therapeutics has begun enrollment in a phase 2a trial of critical limb ischemia patients. The Cleveland-based company, which recently secured an important investment from Takeda Pharmaceuticals, is planning to enroll 48 patients and complete enrollment early next year, CEO Rahul Aras said. Juventas technology, called JVS-100, works by recruiting stem cells from the bone marrow to create new blood vessels. Its based on Stromal Cell-Derived Factor-1 (SDF-1), a naturally produced molecule that attempts to repair the heart immediately following a heart attack. Critical limb ischemia (CLI) patients are enrolling at several U.S. hospitals, as well as three in India. CLI is a severe obstruction of the arteries that greatly decreases blood flow to the extremities. Advertisement CLI has become a very exciting clinical opportunity, Aras said. Its becoming a growing area of interest for a number of biotech and pharma companies. Other companies pursuing CLI treatment include Aastrom Biosciences, Arteriocyte and Biomet. Among the top advantages of Juventas CLI therapy is its simplicity and cost-effectiveness, Aras said. Patients can be injected with the companys therapeutic in an easy procedure at a physician office, and the approach doesnt require bone marrow aspiration to obtain patients own stem cells or complex cell processing as some competing therapeutics do. Juventas also has a phase 2 trial underway to investigate its therapy with heart failure patients. The company is expected to shortly announce a series B round of investment, which includes the funding from Takeda, that totals around $20 million or $25 million.


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May 16

Researchers in Spain may have discovered the secret to a longer life, a new study says.


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