StemCells Presents Two-Year Pelizaeus-Merzbacher Disease (PMD) Data; India Revokes GSK Cancer Drug Patent Print E-mail
By Staff and Wire Reports   
Friday, 02 August 2013 19:34

Below is a look at some of the headlines for companies that made news in the healthcare sector on August 2, 2013. StemCells, Inc. (Nasdaq:STEM) today presented data which show that two years after transplantation of the Company's proprietary HuCNS-SC® cells (purified human neural stem cells) into patients with Pelizaeus-Merzbacher disease (PMD), the evidence of myelination, by magnetic resonance imaging (MRI), is more pronounced compared to one year post-transplantation, the gains in neurological function reported after one year were maintained, and there were no safety concerns. Patients with PMD have a defective gene which leads to insufficient myelin in the brain, resulting in progressive loss of neurological function and death.  The neurological and MRI changes suggest a departure from the natural history of the disease and may represent signals of a clinical effect. The data was presented today by Stephen Huhn, MD, FACS, FAAP, Vice President, CNS Clinical Research at StemCells, Inc., at the 2013 Pelizaeus-Merzbacher Disease Symposium and Health Fair being held at Nemours/Alfred I. duPont Children's Hospital in Wilmington, Del.


"We are encouraged that the MRI data continue to indicate new and durable myelination related to the transplanted cells and that the data is even stronger after two years compared to one year," said Dr. Huhn.  "Even in the context of a small open-label study, these MRI results, measured at time points long after transplantation, make an even more convincing case that the HuCNS-SC cells are biologically active and that their effect is measureable, sustainable and progressive.  Our challenge now is to reach agreement with the FDA on how best to correlate changes in MRI with meaningful clinical benefit, as this will be a critical step in determining a viable registration pathway for PMD."

In the Company's Phase I trial, which was conducted at the University of California, San Francisco, four patients with connatal PMD, the most severe form of the disease, were enrolled and transplanted with HuCNS-SC cells. The patients were followed for twelve months after transplantation, during which time they underwent intensive neurological assessments and magnetic resonance imaging at regular intervals. The Phase I trial results indicate a favorable safety profile for the HuCNS-SC cells and the transplantation procedure. Analysis of the MRI data showed changes consistent with increased myelination in the region of the transplantation, which progressed over time and persisted after the withdrawal of immunosuppression at nine months. The results support the conclusion of durable cell engraftment and donor cell-derived myelin in the transplanted patients' brains. In addition, clinical assessment revealed small but measureable gains in motor and/or cognitive function in three of the four patients; the fourth patient remained clinically stable. The Phase I trial results were published in October 2012 in Science Translational Medicine, the peer review journal of the American Association for the Advancement of Science. Upon completion of the Phase I trial, all four patients were enrolled into a long-term follow up study, which is designed to follow the patients for four more years.

The Pelizaeus-Merzbacher Disease Symposium and Health Fair is intended to help educate families and healthcare professionals about PMD and related disorders. Sponsors include The PMD Foundation, a non-profit organization formed to raise awareness of the disease, create support for people with PMD, reduce misdiagnosis of PMD, and fund scientific research, and Nemours Biomedical Research, a branch of the Nemours Foundation, which is dedicated to improving the health and health care of all children.


India has revoked a patent granted to GlaxoSmithKline Plc (NYSE:GSK) for breast cancer drug Tykerb, a decision that follows a landmark India court ruling disallowing patents for incremental innovations that was a blow to global pharmaceutical firms.

However, India's Intellectual Property Appellate Board (IPAB) upheld a patent granted on the original compound, or active pharmaceutical ingredient, lapatinib, citing innovative merit.

As a result, a GSK spokesman said its medicine would remain subject to patent protection until 2019. The additional patent on the particular salt of lapatinib used in Tykerb, which has now been rejected, would have extended that protection to 2021.

India's Supreme Court in April rejected a patent for Novartis AG's cancer drug Glivec, saying it was an amended version of a known molecule called imatinib, setting the precedent for more such cases in the country.

GSK had cut prices of Tykerb by about a third in India as part of a flexible pricing programme designed to make important drugs more affordable in certain emerging markets.

Western drugmakers who covet a bigger share of India's fast-growing $13 billion drugs market have been frustrated by a series of decisions on intellectual property and pricing.

Last year, India revoked patents granted to Pfizer Inc's cancer drug Sutent, Roche Holding AG's hepatitis C drug Pegasys, and Merck & Co's asthma treatment aerosol suspension formulation. All were revoked on grounds that included lack of innovation.

Fresenius Kabi Oncology, the Indian unit of German healthcare group Fresenius SE, had challenged patents granted for both the original molecule and its marketed salt version, saying both molecules lacked innovation.

"This decision is just like the landmark Glivec ruling. The IPAB has said that the salt version of lapatinib cannot hold a patent," said lawyer Dominic Alvares of S. Majumdar & Co, which represented Fresenius Kabi in the case.

The IPAB rulings for both the patent disputes were uploaded on its website late on Thursday.

Shares in GlaxoSmithKline Pharmaceuticals, the UK company's Indian unit, fell 2.8 percent on Friday.

The company can make an appeal on the decision to India's Supreme Court.

"We are studying the IPAB's decision but maintain our belief in the inventiveness of the lapatinib ditosylate salt and will consider the possibility of taking further steps before the appropriate authorities to validate this," the Indian GSK unit said in an email to Reuters on Friday.

"We are pleased that the IPAB in India has upheld our basic patent for the lapatinib compound, the active ingredient in Tykerb." GSK said. The patent expires in January 2019.

A strip of 10 Tykerb tablets costs about 4,160 rupees ($69) in India and a patient is expected to take five tablets a day for 21 days if the cancer is in an advanced stage.

Western pharmaceutical companies, looking to emerging markets such as India to help drive growth, have run into various obstacles recently, ranging from corruption and pricing probes in China to stock management problems in Brazil.


Also Friday:

Amarin Corporation plc (Nasdaq:AMRN)
, a biopharmaceutical company focused on the commercialization and development of therapeutics to improve cardiovascular health, today announced that it will host a conference call with members of Amarin senior management to discuss the company's second quarter 2013 financial results and provide an operational update on Thursday, August 8, 2013, at 4:30 p.m. EDT.

Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX)
, a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neuromuscular and neurological diseases, announced today that it has received notice from the NASDAQ Stock Market ("NASDAQ") on August 2, 2013 confirming that the Company has regained compliance with the $1.00 per share minimum bid price requirement for continued listing on the NASDAQ Capital Market.

Cytomedix, Inc. (OTCQX: CMXI)
, a regenerative therapies company commercializing and developing innovative platelet and adult stem cell technologies, announced today that it will release financial results for the three- and six-month period ended June 30, 2013, after the close of the market on Wednesday, August 7, 2013.

Digirad Corporation (NASDAQ: DRAD)
today announced that Matthew G. Molchan, President and Chief Executive Officer, is scheduled to make an investor presentation at the 2013 Southern California Investor Conference on Thursday, August 8, at 12:00 pm Pacific Time.

Hologic, Inc. (NASDAQ: HOLX)
, a leading developer, manufacturer and supplier of premium diagnostics products, medical imaging systems and surgical products, with an emphasis on serving the healthcare needs of women, today announced it received U.S. Food and Drug Administration (FDA) clearance for the Company's Single Energy (SE) Femur Exam on a dual energy x-ray absorptiometry (DXA) platform for the assessment of features associated with atypical femur fractures (AFF). This first-of-its-kind capability assists clinicians in their assessment of potential AFF in patients who have been on anti-resorptive treatments such as bisphosphonates.

MediSwipe Inc. (OTCQB: MWIP), a data management solutions company for the medicinal marijuana and health care industry, today announced that the Company has recently filed for a registered trademark for its new secure medical packaging concept "Mello Meal"™ to be used at medicinal dispensaries and on behalf of patients across the United States.

Organovo Holdings, Inc. (NYSE MKT: ONVO) today announced the sale of 9,000,000 shares of its common stock in an underwritten public offering at a price to the public of $4.50 per share.

The success of Rafarma Pharmaceuticals, Inc. (OTC: RAFA) in choosing Terbuny for its facility has attracted more biotech companies to the area.

Sunshine Heart, Inc. (Nasdaq:SSH)
today announced that its second quarter results will be released on Wednesday, August 7, 2013, before the open of the stock market., Inc. (PINKSHEETS: SEEK)
, an emerging leader in the Local search space, today announced that its CEO Scott Gallagher will be interviewed on the small cap stock market program Traders Nation Tuesday August 6th, 2013.

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