Category Archives: FDA Stem Cell Trials

SEATTLE, Aug. 1, 2012 /PRNewswire/ --Cell Therapeutics, Inc. ("CTI" or the "Company") (NASDAQ and MTA: CTIC) today reported financial results and recent accomplishments for the second quarter of 2012. Recent Company Highlights "Based on in-market research among more than 250 lymphoma specialists in the five major market countries in the European Union ("EU"), we are encouraged by the interest and potential adoption of Pixuvri for treatment," stated James A. Bianco, M.D., President and CEO of CTI. "We believe we can present an argument for Pixuvri to provide fair pricing reimbursement in an effort to address the commercial potential of Pixuvri in the E.U. On the heels of our projected fourth quarter launch of Pixuvri in the E.U., we expect to start pivotal studies of our recent JAK2 product acquisition, pacritinib. In the meantime, we are also advancing tosedostat toward its phase 3 clinical trial." Financial Results For the quarter endedJune 30, 2012, total operating expenses were$49.4 millioncompared to$16.9 millionfor the same period in 2011. The increase is predominantly due to an acquired in-process research and development expense of $29.1 million relating primarily to the acquisition of pacritinib from S*BIO Pte Ltd. ("S*BIO"), in addition to equity-based compensation and expenses related … Continue reading →

Marc Buoniconti has been paralyzed from the neck down for 27 years after a college football injury at South Carolina's The Citadel. His Hall-of-Fame linebacker father, Nick Buoniconti, triumphed on the football field as a Boston Patriot and Miami Dolphin, but finding a cure for his son's paralysis has been the toughest game of all, until now. Father and son have seen the fruits of their joint venture -- the Miami Project Cure Paralysis -- to help the 300,000 Americans living with spinal-cord injuries. The Food and Drug Administration has approved a phase 1 clinical trial to evaluate the safety of transplanting human Schwann cells to treat patients with paralysis. It is the first such trial in the world. Researchers believe these cells, which are found in the peripheral nervous system and are responsible for sending electrical signals, might be the key to eventual cures. "We believe today's announcement is just as important to our field as man's first step on the moon was to the space program," said neurosurgeon Dr. Barth Green, co-founder and chairman of The Miami Project. Eight patients with acute spinal-cord injuries, or those within one month of paralysis, will be injected with their own Schwann … Continue reading →

HENDERSON, Nev.--(BUSINESS WIRE)-- Spectrum Pharmaceuticals (SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, announced today publication of positive long-term clinical data for ZEVALIN (ibritumomab tiuxetan) Injection in patients with advanced, relapsed follicular non-Hodgkins lymphoma (fNHL). The data, published in Blood, the journal of the American Society of Hematology, showed that addition of ZEVALIN following fludarabine, cyclophosphamide, rituximab (FCR) conditioning and nonmyeloablative allogeneic stem cell transplantation (NST) resulted in a significantly improved 6-year overall survival (OS) estimate of 80% (95% CI, 41%-95%) in chemotherapy refractory patients, thereby overcoming a strong negative prognostic factor that typically precludes these patients from receiving other treatment options. As reported in the literature, following three episodes of relapsed disease, patients with chemorefractory disease have a median survival of 1.5 years.1 The clinical trial data were presented most recently at the 53rd American Society of Hematology Annual Meeting in December 2011. It is very encouraging to see the publication of long-term clinical data showing that ZEVALIN's usage in this allogeneic transplantation regimen can contribute to improved overall survival in the abysmal prognostic group of patients with disease progression after multiple chemotherapy regimens and in some … Continue reading →

LOS ANGELES--(BUSINESS WIRE)-- ImmunoCellular Therapeutics (NYSE MKT: IMUC) today announced that the U.S. Food and Drug Administration (FDA) has given clearance to a physician sponsored investigative new drug (IND) application to initiate a Phase I clinical trial of ICT-121, IMUCs novel dendritic-cell-based vaccine targeting CD-133, an antigen that is highly expressed by multiple solid tumors. The trial, which will be conducted at a leading Los Angeles based medical center, will initially test the vaccine in up to 20 patients with recurrent glioblastoma multiforme (GBM), the most common and aggressive form of brain cancer. With a Phase II trial of IMUCs lead product candidate ICT-107 well under way in treating patients with newly diagnosed GBM, ICT-121 is the Companys second dendritic-cell-based vaccine to enter the clinic. Like ICT-107, ICT-121 targets the cancer stem cells (CSCs) that are widely believed to be the root cause of many cancers. Preclinical studies conducted by IMUC indicate that ICT-121 effectively targets CD-133, a protein that is over-expressed by a very wide range of solid tumors, including glioblastoma, pancreatic, breast, non-small-cell lung cancer, and several other malignancies associated with poor survival and limited treatment options, said Manish Singh, Ph.D., president and CEO of IMUC. Given the … Continue reading →

Peter Aldhous, San Francisco bureau chief It's official: stem cells are drugs. At least, that's the opinion of the US district court in Washington DC, which has ruled that the Food and Drug Administration (FDA) has the authority to regulate clinics offering controversial stem cell therapies. Treatments in which stem cells are harvested from bone marrow and injected straight back into the same patient are deemed part of routine medical practice - not regulated by the US government. But if the cells are subjected to more than "minimal manipulation", the FDA maintains that the therapy becomes a "drug", which must be specifically approved for use. It was on this basis that in 2008 the FDA began moves to shut downRegenerative Sciences, a clinic in Broomfield, Colorado, that treats orthopaedic problems using a stem cell therapy called Regenexx. Regenerative Sciences challenged the FDA's authority to regulate its activities, setting the stage for a legal fight. In 2010, the FDA sought an injunction to take Regenexx off the market. This has now been granted in the court's ruling. Christopher Centeno, medical director of Regenerative Sciences, vows to appeal. "This is really round one," he says. "Our position remains that a patient's cells … Continue reading →

Embargoed for Release Tuesday, July 24, 2012 11 a.m. EDT Seventeen National Institutes of Health grants are aimed at creating 3-D chips with living cells and tissues that accurately model the structure and function of human organs such as the lung, liver and heart. Once developed, these tissue chips will be tested with compounds known to be safe or toxic in humans to help identify the most reliable drug safety signals ultimately advancing research to help predict the safety of potential drugs in a faster, more cost-effective way. The initiative marks the first interagency collaboration launched by the NIH's recently created National Center for Advancing Translational Sciences (NCATS). Tissue chips merge techniques from the computer industry with modern tissue engineering by combining miniature models of living organ tissues on a transparent microchip. Ranging in size from a quarter to a house key, the chips are lined with living cells and contain features designed to replicate the complex biological functions of specific organs. NIH's newly funded Tissue Chip for Drug Screening initiative is the result of collaborations that focus the resources and ingenuity of the NIH, Defense Advanced Research Projects Agency (DARPA) and U.S. Food and Drug Administration. NIH's Common Fund … Continue reading →

Los Angeles, July 23, 2012 - (ACN Newswire) - Trying to find companies that represent the future of the biotech sector can be tricky for investors. The sector currently leads the market because Food and Drug Administration (FDA) regulatory guidance is changing, with restrictions easing, and newguidelines being proposed and implemented. The Obama Administration's 2011 directive towards the agency jump started aggressive investor speculation in the sector-- especially small cap developmental companies. Orphan drug designated medical devices, and subcutaneous self-injector medical devices all are benefiting from the progression of change currently occurring. The hard part is trying to find the right company-- a company that does not over dilute shareholders, spends its money wisely, and has a legitimate shot at becoming a large revenue and profit producing company. Too many small cap biotech companies show promising technology, but have bad stock and financial structures. The key in small cap biotech investing is finding companies that structure their business with the main goal of both financial growth and technological advancement. Antares Pharma (ATRS) is my largest holding because it fits the current regulatory changes occurring-- subcutaneous self-injection medical devices. I invested last year in Antares because I did my due diligence … Continue reading →

BOTHELL, Wash.--(BUSINESS WIRE)-- Seattle Genetics, Inc. (SGEN) today announced that its collaborator, Millennium: The Takeda Oncology Company, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, has received a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the conditional marketing authorization of ADCETRIS (brentuximab vedotin) for two indications: (1) the treatment of adult patients with relapsed or refractory CD30-positive Hodgkin lymphoma (HL) following autologous stem cell transplant (ASCT) or following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option, and (2) for the treatment of adult patients with relapsed or refractory systemic anaplastic large cell lymphoma (sALCL). ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30. The positive opinion from CHMP and broad label recommendation is a key step in the European regulatory process for ADCETRIS and brings us closer to our goal of making this important new therapy globally available to patients with relapsed Hodgkin lymphoma or systemic ALCL, said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. If approved in the European Union, ADCETRIS will represent the first new therapeutic advance for relapsed Hodgkin lymphoma patients in … Continue reading →

LOS ANGELES--(BUSINESS WIRE)-- Targeted Medical Pharma, Inc., a specialty pharmaceutical company that develops and distributes prescription medical foods to physicians, pharmacies and skilled nursing facilities, today issued the following letter to shareholders from its Chairman and Chief Executive Officer and Chief Scientific Officer, William Shell, M.D. Dear Fellow Shareholders: If one word can best characterize the past year for me and for Targeted Medical Pharma, that word would be change. It has been a fast-paced journey for all of us, marked by substantial growth that places our company on the cusp of formidable transformation. There was great progress positioning us for the introduction of promising new products, having filed multiple new patents, completing clinical trials of a treatment for Post-Traumatic Stress Disorder (PTSD) and developing an advanced obesity management system. As we enter the second half of 2012, our team is excited about the research that is now well underway on a technology, believed to be the first of its kind, for stem cell stimulation via an oral administration. As of this writing, our employee count has grown to 68 full time employees, with more than 40 direct and indirect sales representatives in our own sales force and those of … Continue reading →

ALLENDALE, N.J. and WILMINGTON, Del., July 16, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NYSE MKT:NBS) and its subsidiary, Progenitor Cell Therapy, LLC ("PCT"), an internationally recognized Contract Development and Manufacturing Organization (CDMO), and SOTIO, LLC, a Delaware limited liability company that is responsible for organizing the U.S. part of a global pivotal Phase 3 clinical trial of SOTIO, LLC's affiliate, SOTIO a.s. ("SOTIO"), announced today that SOTIO, LLC has retained the services of PCT to manufacture clinical products for SOTIO's U.S. part of a global pivotal Phase 3 clinical trial. SOTIO, LLC is an affiliate of a Czech Republic-based biotechnology company developing new therapies based on activated dendritic cells, focusing on the treatment of cancer and autoimmune diseases. SOTIO, LLC will use the services of PCT to transfer and qualify at PCT's Allendale, New Jersey facility, SOTIO's GMP manufacturing process for the U.S. part of a global pivotal Phase 3 clinical trial for SOTIO's autologous dendritic cell vaccine expected to launch in early 2013, subject to FDA approval. As part of this agreement, PCT will complete a technology transfer of SOTIO's current product manufacturing and analytical procedures into PCT's ongoing CDMO operations. PCT will then implement and perform process qualification … Continue reading →