Neuralstem Announces ALS Long-Term Follow-Up Phase I Data; Tekmira Establishes Regulatory Framework For Emergency Use Of TKM-Ebola Print E-mail
By William Kent   
Monday, 22 September 2014 20:01
Below is a look at some of the headlines for companies that made news in the healthcare sector on September 22, 2014.
Neuralstem, Inc. (NYSE MKT: CUR) announced Jonathan D. Glass, MD, site investigator at Emory University, presented long-term follow up data on the Phase I trial testing NSI-566 human neural stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).  The presentation, which occurred at the Annual Symposium on ALS of the Foundation Andre-Delambre, in Montreal, Canada, on Friday, and was not open to the public, covered data up to approximately 1200 days post the stem cell treatment.

Dr. Glass reported that patients in the last safety cohort (n=3), who received treatments in both the lumbar and the cervical region with the highest number of cells per injection, all showed significant slowing of the progression of the disease. One patient showed functional improvement from pre-treatment baseline, which is maintained to present day.  The other two patients are maintaining the same level of functionality as they had at the baseline for over three years since the stem cell treatment.

"The long-term follow up data is very encouraging," said Karl Johe, PhD, Neuralstem's Chairman and Chief Scientific Officer. "In Phase I, patients 10, 11, and 12 each received 10 lumbar and five cervical injections, of 100,000 cells each, which was far below the safe maximal dose. Even so, the data shows a significant slowing of the disease progression for over three years.  If replicated on a larger scale, this could represent meaningful improvement in quality of life, and lifespan, compared to untreated patients.  In our Phase II dose escalation trial, we successfully reached the maximal dose planned, which consisted of  20 lumbar and 20 cervical injections of 400,000 cells each, more than ten times the number of stem cells delivered in the highest dose cohort of the Phase I trial."

"The progress in this trial is truly groundbreaking," said Dr. Glass, who is Director of the Emory ALS Center at Emory University, the first site in the trial. "It has provided data on the safety of multiple injections and multiple transplantation surgeries in ALS patients, as well as the long- term survival of the transplanted cells in the human spinal cord.  This provides a strong foundation for moving ahead with more definitive trials focused on the potential therapeutic efficacy of spinal cord transplantation of neural stem cells for ALS."


 
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Tekmira Pharmaceuticals Corporation (Nasdaq: TKMR) (TSX:TKM)
, a leading developer of RNA interference (RNAi) therapeutics, today announced that the FDA has authorized Tekmira to provide TKM-Ebola for treatment under expanded access protocols to subjects with confirmed or suspected Ebola virus infections.

"Tekmira is reporting that an appropriate regulatory and clinical framework is now in place to allow the use of TKM-Ebola in patients. We have worked with the FDA and Health Canada to establish this framework and a treatment protocol allowing us to do what we can to help these patients," said Dr. Mark J. Murray Tekmira's President and CEO.

"We have insisted on acting responsibly in the interest of patients and our stakeholders," added Dr. Murray. "Today we are reporting that, working closely with regulators in the United States and Canada, we have established a framework for TKM-Ebola use in multiple patients. In the US, the FDA has granted expanded access use of TKM-Ebola under our Investigational New Drug application (IND) and Health Canada has established a similar framework, both of which allow the use of our investigational therapeutic in more patients."

"We have already responded to requests for the use of our investigational agent in several patients under emergency protocols, in an effort to help these patients, a goal we share with the FDA and Health Canada. TKM-Ebola has been administered to a number of patients and the repeat infusions have been well tolerated. However, it must be kept in mind that any uses of the product under expanded access, does not constitute controlled clinical trials. These patients may be infected with a strain of Ebola virus which has emerged subsequent to the strain that our product is directed against, and physicians treating these patients may use more than one therapeutic intervention in an effort to achieve the best outcome," said Dr. Murray. "Our TKM-Ebola drug supplies are limited, but we will continue to help where we can, as we continue to focus on the other important objectives we have to advance therapies to meet the unmet needs of patients."

TKM-Ebola is an investigational therapeutic, being developed under an FDA approved IND, which is currently the subject of a partial clinical hold under which the FDA has allowed the potential use of TKM-Ebola in individuals with a confirmed or suspected Ebola virus infection



Also Monday:


 
Alphatec Holdings, Inc. (Nasdaq:ATEC), the parent company of Alphatec Spine, Inc., a global provider of spinal fusion technologies, announced today that it will showcase its product portfolio at the upcoming EuroSpine 2014 meeting, which is being held in Lyon, France, from October 1st to October 3rd, 2014.

Analogic Corporation (Nasdaq:ALOG), enabling the world's medical imaging and aviation security technology, announced today that the Company will hold its Annual Investor Day on Wednesday, October 1, beginning at 10:00 a.m. (ET), at Analogic's Peabody, Massachusetts, headquarters.

athenahealth, Inc.(Nasdaq:ATHN), a leading provider of cloud-based services and mobile tools for medical groups and health systems, today announced new office openings in Atlanta, Georgia, and San Francisco, California.

Avanir Pharmaceuticals, Inc. (NASDAQ: AVNR) today announced that it is offering to sell $200 million of its common stock in an underwritten public offering. 

Bio Blast Pharma Ltd (Nasdaq:ORPN)
, a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today it has received Health Canada approval to begin enrolling patients at the Montreal Neurological Institute at McGill University for its HOPEMD Phase 2/3 study investigating Cabaletta in patients with Occulopharyngeal Muscular Dystrophy (OPMD).

Celladon Corporation (Nasdaq:CLDN)
, a clinical-stage biotechnology company applying its leadership position in the field of gene therapy and calcium dysregulation, today announced it has appointed Scott Garrett as Vice President, Commercial Planning, a newly created position.

ChemoCentryx, Inc., (Nasdaq:CCXI), a clinical-stage biopharmaceutical company focused on autoimmune diseases, inflammatory disorders and cancer, announced today that it has appointed Israel F. Charo, M.D., Ph.D., Senior Vice President of Research, effective immediately.

Codexis, Inc. (Nasdaq:CDXS), a leading developer of biocatalysts for the pharmaceutical and fine chemical industries, today announced the appointment of Kathleen Sereda Glaub to its board of directors.

EDAP TMS SA (Nasdaq:EDAP)
, the global leader in therapeutic ultrasound, announced that the growing interest in and acceptance of its Ablatherm®-HIFU technology was once again highlighted as a valuable treatment alternative for prostate cancer patients in a presentation at the World Congress of Endourology (WCE) in Taipei, Taiwan from September 3-7, 2014.

GlycoMimetics, Inc. (Nasdaq:GLYC) today announced that Rachel King, Chief Executive Officer, will present an overview of the company at BioCentury's 21st Annual Newsmakers in the Biotech Industry Conference on September 26, 2014, at 9:30 a.m. Eastern Time in New York City.

Hemispherx Biopharma, Inc. (NYSE MKT:HEB)
, announced today a peer-reviewed publication entitled "Protection from Pulmonary Tissue Damage Associated with Infection of Cynomolgus Macaques by Highly Pathogenic Avian Influenza Virus (H5N1) by Low Dose Natural Human IFN-α Administered to the Buccal Mucosa" in the current issue of Antiviral Research 2014.

India Globalization Capital, Inc. (NYSE MKT: IGC),
announced that all proposals were passed at its 2014 Annual Meeting of Stockholders reconvened on September 12, 2014.

Intersect ENT, Inc. (Nasdaq:XENT), a company dedicated to improving the quality of life for patients with ear, nose and throat conditions, today announced initial results from RESOLVE, a clinical study of the company's investigational steroid eluting implant designed to treat patients with recurrent sinus obstruction in the office setting.

Materialise NV (Nasdaq:MTLS)
announced that orthopedic surgeon Roger Jaeken, of the AZ Heilige Familie (Holy Family General Hospital) in Reet, Belgium, has successfully performed the first total knee surgery that was preplanned by means of the Materialise X-ray knee guide solution.

MedAssets (Nasdaq:MDAS) today announced that it completed its previously announced acquisition of SG-2, LLC (Sg2), a leading provider of healthcare business intelligence, market analytics and clinical consulting services.

Nuvilex, Inc. (OTCQB:NVLX)
, a clinical-stage biotechnology company providing cell and gene therapy solutions for the treatment of diseases, announced today that its partner, Austrianova, published a scientific review in the journal Diabetes Research and Treatment: Open Access.

QLT Inc. (Nasdaq:QLTI) (TSX:QLT)
announces that it has been advised by Auxilium Pharmaceuticals, Inc. ("Auxilium") that Auxilium's board of directors (the "Auxilium Board") has reviewed the unsolicited offer from Endo International plc ("Endo") to acquire all of the issued and outstanding shares of Auxilium (the "Endo Proposal") and, after consulting with its independent financial and legal advisors, has unanimously determined that the Endo Proposal is not a superior proposal under the terms of the Agreement and Plan of Merger (the "Merger Agreement") among QLT, Auxilium, QLT Holding Corp., and QLT Acquisition Corp., dated June 25, 2014.

Revance Therapeutics, Inc. (Nasdaq:RVNC)
, a biopharmaceutical company developing botulinum toxin products for use in aesthetic and therapeutic indications, today announced Azita Nejad has been named its Vice President of Quality.

T2 Biosystems (Nasdaq:TTOO), a company developing innovative diagnostic products to improve patient health, today announced that it has received market authorization from the U.S. Food and Drug Administration (FDA) for its first two products, the T2Candida® Panel and the T2Dx® Instrument for the direct detection of Candida species in human whole blood specimens from patients with symptoms of, or medical conditions predisposing the patient to, invasive fungal infections.

Tengion, Inc. (OTCQB:TNGN)
, a leader in regenerative medicine, today announced that the Company, with the support of its Board of Directors, is exploring all strategic options to maximize the value of its assets and has retained Jefferies LLC as exclusive financial advisor to assist it with reviewing and structuring potential strategic transactions.

TESARO, Inc. (Nasdaq:TSRO)
announced today that, subject to market and other conditions, it intends to offer $165 million aggregate principal amount of convertible senior notes due 2021 (the "notes") in an underwritten public offering.

Vision-Sciences, Inc. (Nasdaq:VSCI)
, a leading provider of unique flexible endoscopic products utilizing its proprietary sterile disposable EndoSheath® technology, today announced the introduction of the company's next generation video laryngoscope.



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