BioMarin to Advance BMN-701 for Pompe Disease; US Patent Office Grants Patent for TECFIDERA™ Print E-mail
By Staff and Wire Reports   
Tuesday, 19 March 2013 18:56
Below is a look at some of the headlines for companies that made news in the healthcare sector on March 19, 2013.

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN)
announced results from POM-001, the Phase 1/2 trial for BMN-701, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) for the treatment of late-onset Pompe disease. The results exceeded the company's pre-specified requirements for proceeding to the next phase of development by showing that in the 20 mg/kg every other week dose cohort, three out of 16 patients, or 19 percent, had a greater than 75 meter improvement in 6-minute walk distance, and that there was a 14.1 percent relative improvement in Maximal Expiratory Pressure (MEP) and a 27.0 percent relative improvement in Maximal Inspiratory Pressure (MIP) from pre-treatment baseline to week 24, two important measures of overall respiratory muscle function and strength. Pending a review with regulatory authorities, the company expects to continue development of BMN-701 by initiating a Phase 2/3 switching trial by the end of 2013 in late-onset Pompe patients who have previously been treated with alglucosidase alfa (Myozyme®/Lumizyme®).

"More than half of late-onset Pompe patients require ventilatory assistance, and many more patients have impairments directly related to weakness of breathing muscles. This means that a therapy that improves respiratory muscle function substantially would be important and welcome and could help delay premature death in Pompe disease," said Professor Benedikt Schoser of the Friederich-Baur Institute and speaker of the German working group for Pompe disease.

"The findings from this study point the way forward for a potentially meaningful advance in the management of late-onset Pompe patients. Pompe is a disease that destroys muscle tissue throughout the body, including the muscles of respiration. Maximal Expiratory Pressure (MEP) and Maximal Inspiratory Pressure (MIP) are direct measures of respiratory muscle function and likely an important indicator of a drug's effectiveness in this setting. The improvements in MEP and MIP that BMN-701 patients demonstrated is a unique and important finding," said Barry Byrne, M.D., Ph.D., Professor, Pediatrics and Molecular Genetics & Microbiology and Director, University of Florida Powell Center, and lead investigator for POM-001. "If treatment with BMN-701 results in improvements in these important respiratory functional measures in patients who have already experienced a maximal benefit from current therapy, BMN-701 could become an important treatment option," Byrne said.

Phase 1/2 Study: Efficacy and Safety -- The mean improvement in 6-minute walk distance was approximately 22 meters for the 16 patients treated in the 20 mg/kg cohort. In addition, there were three super-responders, or 19 percent of patients, who experienced greater than a 75 meter improvement in 6-minute walk distance from baseline to week 24. For pulmonary function, mean improvement in percent predicted forced vital capacity (FVC) was 1.2 percent in absolute terms, or a 2.0 percent relative improvement from pre-treatment baseline to week 24. Mean improvement in maximum voluntary ventilation (MVV) was 2.9 L/min in absolute terms, or a 4.3 percent relative improvement from pre-treatment baseline to week 24. Mean improvement in percent predicted maximal expiratory pressure (MEP) was 5.1 percent in absolute terms, or a 14.1 percent relative improvement from pre-treatment baseline to week 24. Mean improvement in percent predicted maximal inspiratory pressure (MIP) was 11.0 percent in absolute terms, or a 27.0 percent relative improvement from pre-treatment baseline to week 24. The company conducted a responder analysis in which each patient was assigned a score of plus one for improvement of more than 10 percent and minus one for a decline of 10 percent in the domains of 6-minute walk test, MEP and MIP. Each patient's scores were aggregated by summing the individual domain scores for that patient. Thirteen of the 16 patients scored a plus one or greater; two patients had a score of 0, consistent with stabilization, and one patient declined in one domain.

Side effects for BMN-701 were generally consistent with those seen for other enzyme replacement therapies. The principal clinical adverse event was infusion-associated reaction in two patients, resulting in temporary drug interruption in one patient and drug withdrawal in one patient. Infusion-associated hypoglycemia, an expected pharmacologic effect of BMN-701, occurred in 13 patients in the 20 mg/kg cohort. All hypoglycemia events occurred during or within one hour of infusion, were transient and readily manageable through diet and predominantly asymptomatic.


Biogen Idec (NASDAQ: BIIB)
announced the U.S. Patent and Trademark Office (USPTO) has granted U.S. Patent No. 8,399,514, which offers additional protection for TECFIDERA™ (dimethyl fumarate), the company's oral therapeutic candidate for the treatment of multiple sclerosis (MS). The patent, which will expire in 2028, covers the dosing regimen of daily administration of 480 mg of TECFIDERA. This regimen is included in the proposed marketing application for TECFIDERA, which is currently under review by the U.S. FDA.

“The patent for this dosing regimen is recognition of the remarkable innovation TECFIDERA represents for the MS community,” said George A. Scangos, Ph.D., chief executive officer of Biogen Idec. “The tremendous research investment required to study and validate the patented dosing regimen is an example of innovation that leads to meaningful benefits to patients.”

The European Patent Office also recently determined that Biogen Idec's application for a patent covering the same dosing regimen of TECFIDERA is allowable. Once granted, this patent would also expire in 2028.

The TECFIDERA dose regimen patents add to the growing portfolio of granted patents covering TECFIDERA.

Also Tuesday:

Accelrys, Inc. (NASDAQ: ACCL)
, a leading provider of scientific innovation lifecycle management software, announced at Pittcon 2013 a strategic partnership with U.S. Data Management (USDM), a global regulatory compliance and risk management consulting firm focused exclusively on the life sciences industry.

AmerisourceBergen (NYSE: ABC)
today announced that it is entering into a strategic, long-term relationship with Walgreen Co. and Alliance Boots GmbH, which will streamline the distribution of pharmaceuticals to Walgreens’ stores and leverage global supply chain efficiencies while improving patient access to affordable pharmaceuticals to increase the efficiency of the healthcare system.

Aqua Gen, a member of the Erich Wesjohann (EW) Group GmBH and Center for Integrative Genomics (CIGENE) at the Norwegian University of Life Sciences (UMB) in collaboration with Affymetrix, Inc. (NASDAQ: AFFX) announced today that they are the first to genotype more than 900,000 markers per sample from the Atlantic salmon (Salmo salar), thereby achieving the capability to implement genomic selection and improve their salmon breeding program at Aqua Gen.

Bioheart, Inc. (OTCQB: BHRT)
announced today that it has expanded its Center of Excellence program to include a clinical site in Jamaica with the American Stem Cell and Anti-Aging Center.

is proud to announce an exciting line of new products that will be showcased at this year's American Academy of Orthopaedic Surgeons Annual Meeting in Chicago.

eHealth, Inc. (NASDAQ: EHTH)
, parent company of, America's first and largest private online health insurance exchange, released a 'Cost of Comprehensive Health Benefits' report as an addendum to its seventh consecutive 'Cost and Benefits' report, released in November 2012.

announced today that its poster presentation entitled, “GTx-758, an ERα agonist, reduces serum free testosterone lower than can be achieved by leuprolide with a significantly lower rate of hot flashes in men with advanced prostate cancer”, was selected as one of the best posters at the 28th Annual European Association of Urology Congress held in Milan, Italy, which commenced March 15, 2013.

Impax Pharmaceuticals, a division of Impax Laboratories, Inc. (NASDAQ: IPXL), today announced the presentation of results from its RYTARYTM (IPX066) Phase III and open-label extension trials at the 65th Annual Meeting of the American Academy of Neurology in San Diego, CA on March 18, 2013.

Mast Therapeutics, Inc. (NYSE MKT: MSTX)
today reported financial results for the fourth quarter and year ended December 31, 2012.

Milestone Scientific Inc. (OTCQB: MLSS)
, the recognized leader in advanced, computer-controlled injection technologies, today announced that Valencia College will be implementing Milestone's STA Single Tooth Anesthesia System® (STA System) in their Dental Hygiene program.

MMRGlobal, Inc. (OTCQB: MMRF)
and Healthcare Merchant Solutions ("HMS") today jointly announced that they have entered into an agreement to begin offering MMR's patented MyMedicalRecords Personal Health Record ("PHR") to HMS clients currently not doing business with MMR, including physician offices, surgery centers and other healthcare professionals, starting immediately.

Northwest Biotherapeutics (NASDAQ: NWBO)
, a biotechnology company developing DCVax® personalized immune therapies for solid tumor cancers, announced today that Linda F. Powers, the Company's Chairman and Chief Executive Officer, will be presenting at the 25th Annual ROTH Conference at 8:30 a.m. PDT on Wednesday, March 20, 2013, at the Ritz-Carlton, Laguna Niguel, Dana Point, California.

NuVasive, Inc. (NASDAQ: NUVA)
, a medical device company focused on developing minimally disruptive surgical products and procedures for the spine, is excited to sponsor the 5th Annual San Diego Festival of Science and Engineering Expo Day on Saturday, March 23, 2013 from 10:00 a.m. to 5:00 p.m. at Petco Park.

Pressure BioSciences, Inc. (OTCQB: PBIO) today announced that scientists from three separate research groups presented data at the recent US HUPO 9th Annual Conference indicating that sample preparation methods based on the Company's patented pressure cycling technology ("PCT") platform resulted in improved quality and/or efficiency of test results.

Stellar Biotechnologies, Inc. (OTCQB: SBOTF) (TSX VENTURE: KLH)
, the world leader in sustainable manufacture of Keyhole Limpet Hemocyanin (KLH), today announced the Company has submitted a Type IV Biologics Master File (BB-MF) to the U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) for its subunit KLH.

Loopback Analytics, a care transitions management platform provider, has been selected to provide the technology platform for Walgreens WellTransitionsSM, a coordinated care program that brings hospitals and health systems together with Walgreens (NYSE: WAG) (Nasdaq: WAG) to reduce readmission rates and health care costs, while improving patient health outcomes and medication adherence.

"Featured Content" profiles are meant to provide awareness of these companies to investors in the small-cap and growth equity community and should not in any way come across as a recommendation to buy, sell or hold these securities. BiomedReports is not paid or compensated by newswires to disseminate or report news and developments about publicly traded companies, but may from time to time receive compensation for advertising, data, analytics and investor relation services from various entities and firms. Full disclosures should be read in the 'About Us Section'.

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