Roche receives FDA clearance for next generation cobas MRSA/SA Test

PLEASANTON, Calif. – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the US Food and Drug Administration (FDA) has provided 510(k) clearance for the cobas® MRSA/SA Test for the early, simultaneous detection of methicillin-resistant Staphylococcus aureus (MRSA) and methicillin-sensitive Staphylococcus aureus (SA) directly from nasal specimens. The cobas® MRSA/SA Test detects both organisms from a single specimen, providing accurate and reliable results for effective prevention and control of MRSA/SA infections.

“Numerous successful surveillance programs have led to a significant decrease in the rate of MRSA clinical infection in many organizations, and a dramatic reduction in postoperative surgical infections when screening for SA is done.  Importantly, the fight against healthcare-associated infections continues to advance, as evidenced by this new test that can rapidly detect both MRSA and SA in a single assay,” said Lance R. Peterson, MD, Director of Microbiology and Infectious Diseases Research at NorthShore University HealthSystem and Clinical Professor of Pathology and Medicine at the University of Chicago, Pritzker School of Medicine. “The cobas® MRSA/SA Test demonstrated excellent performance in detecting both MRSA and SA strains in samples collected throughout the US. Compared to culture testing, the cobas® MRSA/SA Test offers confidence in identifying colonized patients the first time they are evaluated, aiding in the prevention of MRSA disease and post-operative SA surgical infections.”

“Healthcare-associated infections continue to be a leading cause of mortality in US medical settings,” said Paul Brown, Head of Roche Molecular Diagnostics. “With the addition of the cobas® MRSA/SA Test to our expanding menu of tests for the cobas® 4800 System,  Roche offers laboratories and clinicians a highly efficient molecular solution to aid in the overall management and prevention of healthcare-associated infections, leading to lower costs for hospitals and optimal patient care.”

The cobas® MRSA/SA Test, a polymerase chain reaction (PCR)-based assay that runs on the automated cobas® 4800 System, offers labs the most simplified workflow available with a simple de-cap and loading of the primary sample vial onto the cobas® 4800 System. This approach requires less hands-on-time, enabling laboratory staff to spend time on other critical tasks. In addition, this streamlined workflow can help labs reduce costs and improve turnaround time.

About methicillin-resistant Staphylococcus aureus (MRSA) and Staphylococcus aureus (SA)
Approximately one third of the population carries Staphylococcus aureus as normal flora in the anterior nares, which can lead to opportunistic infections. Staphylococcus aureus has evolved resistance mechanisms due to frequent exposure to antibiotics in health care settings. Recent reports show up to 85% of invasive MRSA infections identified as healthcare-associated, resulting in over 90,000 infections and 18,000 deaths in 2005 alone1. Surgical site infections, ventilator assisted pneumonia, and blood stream infections attributed to colonized central lines are the most frequent manifestations of disease. Active surveillance to identify carriers is helping to mitigate the potential consequences of disease, providing relief to patients and healthcare institutions facing the challenges of escalating costs.

About the cobas 4800 System

The cobas 4800 System offers true walk-away automation of nucleic acid purification, PCR  set-up and real-time PCR amplification and detection to help laboratories achieve maximum efficiency. The expanding system menu in the U.S. currently includes the cobas® CT/NG Test (chlamydia/gonorrhea), cobas HPV Test, cobas BRAF V600 Mutation Test and cobas EGFR Mutation Test.

About Roche

Headquartered in Basel, Switzerland, Roche is a leader in research-focused healthcare with combined strengths in pharmaceuticals and diagnostics. Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, infectious diseases, inflammation, metabolism and neuroscience. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. Roche’s personalised healthcare strategy aims at providing medicines and diagnostic tools that enable tangible improvements in the health, quality of life and survival of patients. In 2012, Roche had over 82,000 employees worldwide and invested over 8 billion Swiss francs in R&D. The Group posted sales of 45.5 billion Swiss francs. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com.

1. Klevens et al. Invasive Methicillin-Resistant Staphylococcus aureus Infections in the United States. JAMA. 2007;298:63–1771

Source: Roche

FDA Approves New Glucose Monitoring App for Data-Sharing Among Diabetic Patients and Caregivers

The US Food and Drug Administration (FDA) allowed marketing of the first set of mobile medical apps that allow diabetics to automatically and securely share data from a continuous glucose monitor (CGM) with other people in real-time.

The Dexcom Share Direct Secondary Displays system’s data-sharing capability allows diabetes patient caregivers to monitor an individual’s blood sugar levels remotely. Other CGMs are also available on the market, however Dexcom Share is the first to receive the FDA’s approval.

The app allows caregivers and families to keep an eye on a diabetes patient’s glucose levels remotely, in order to avoid complications, such as hyperglycemia. The Dexcom Share involves two apps, one which would be downloaded by the patient, who will be able to set up “followers” that can view their information and the second would be downloaded by the caregiver to view the CGM data in real time. The patient can share data with up to five followers.

The system includes a small, wire-like sensor which a patient would insert just under the skin. The device continuously transmits data to a monitor that is worn externally. When used along with a blood glucose meter, CGM information can help diabetes patients detect when blood glucose values are approaching dangerously high and dangerously low levels.

“This innovative technology has been eagerly awaited by the diabetes community, especially caregivers of children with diabetes who want to monitor their glucose levels remotely,” said Alberto Gutierrez, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health. “Today’s marketing permission paves the way for similar technologies to be marketed in the United States.”

Dexcom said that it anticipates to ship the Share receiver to new patients in March. This is the first Dexcom system that is mobile compatible without the use of a docking system.

The app was approved under the FDA’s de novo process, which was created for low- to moderate-risk devices, which doesn’t require the same review process as more complicated products. It is the first to receive regulatory approval for US sales since the FDA began regulating mobile medical apps as devices in 2013.

“The Dexcom Share receiver represents a significant step forward for our company and our mobile strategy, but more importantly, it will provide a huge improvement for people managing their diabetes and for those parents and caregivers who help them each and every day,” said Kevin Sayer, President and Chief Executive Officer of Dexcom. “The FDA understands the importance of this type of innovation and the need to regulate it appropriately, and we could not be more pleased with the speed at which they reviewed and approved this important innovation.”

Sources: US Food and Drug Administration; DexCom, Inc.; Specialty Pharma Journal

Protein-based Therapy Shows Promise against Resistant Leukemia

Binding of the protein (green) to the surface of leukemia cells results in cell death via destruction of the cell’s nucleus (dark red) into pieces

LOS ANGELES (January 26, 2015) – Resistance of leukemia cells to contemporary chemotherapy is one of the most formidable obstacles to treating acute lymphoblastic leukemia (ALL), the most common form of childhood cancer.  Now researchers at Children’s Hospital Los Angeles (CHLA) have designed and developed a new protein-based therapy they believe will prove highly effective against drug-resistant leukemia cells.  It may also amplify the potency of standard treatment options such as chemotherapy and radiation therapy.

They successfully accomplished this unique assembly of the two proteins in a single “fusion protein” and named this protein therapeutic candidate “CD19L-sTRAIL.”  In their study, Uckun and collaborators have demonstrated that their engineering converted sTRAIL into a much more potent “membrane-anchored” form that is capable of triggering apoptosis, even in the most aggressive and therapy-resistant form of human leukemia cells.Their work, published online January 26 by the Journal of Clinical Investigation, demonstrated the efficacy and safety of the new fusion protein in mouse models of aggressive human leukemia using leukemia cells taken directly from patients with ALL.

Occurring at an annual rate of 35 to 40 cases per million people in the U.S., ALL represents approximately 25 percent of cancer diagnoses among children under the age of 15. Historically, ALL had a high mortality rate; nearly 80 percent of children who developed the disease did not survive long term. Today, those numbers have been reversed, with almost 80 percent of children affected by ALL achieving long- term survival.

“That’s great news, unless your child is one of the 20 percent,” said the study’s principal investigator Fatih M. Uckun, MD, PhD, of the Children’s Center for Cancer and Blood Diseases at CHLA and the Norris Comprehensive Cancer Center of the University of Southern California (USC). “Despite advances in available therapies, unmet and urgent needs remain in the fight against leukemia.  We still have children with disease that our drugs can’t help enough. And for patients who relapse, their chances of long-term survival are less than 20 percent. We’ve got to do better.”

TNF-related apoptosis-inducing ligand (TRAIL) is a protein functioning as a ligand that induces apoptosis, or cell death. Produced by the immune system cells, it has the potential to cause apoptosis in tumor cells by binding to two so-called “death receptors”, also known as TRAIL-receptor 1 and TRAIL-receptor 2.

“TRAIL is a naturally occurring part of the body’s immune system that kills cancer cells without toxicity to normal cells.  However, earlier clinical trials using TRAIL as a potential anti-cancer medicine candidate have not been successful, largely because of its propensity to bind, not only to cancer cells, but also to ‘decoy’ receptors,” explained Uckun, who is also a professor of pediatrics at Keck School of Medicine of USC.

Uckun and collaborators discovered a previously unknown protein – CD19-Ligand – a natural ligand of human CD19, which is expressed by almost all ALL cells.  They hypothesized that fusing it by genetic bioengineering to the portion of TRAIL (known as sTRAIL) that can kill cancer cells, would produce a powerful weapon against leukemia cells.  Unlike chemotherapy drugs, this precision medicine candidate would seek out, bind and destroy only leukemia cells carrying CD19 as the target docking site.
“Due to its ability to anchor to the surface of cancer cells via CD19, CD19L-sTRAIL was 100,000-fold more potent than sTRAIL, and consistently killed more than  99 percent of aggressive leukemia cells taken directly from children with ALL –  not only in the test tube, but also in mice,” said Uckun. Administering only two or three doses of CD19L-sTRAIL significantly improved the survival outcome of mice challenged with an otherwise invariably fatal dose of human leukemia cells, without side effects.  Its therapeutic potency in mice was superior to that of standard chemotherapy combinations as well as radiation therapy.

“The biggest challenge is to cure patients who experience a recurrence of their cancer, despite intensive chemotherapy,” Uckun concluded. “We are hopeful that the knowledge gained from this study will open a new range of effective treatment opportunities for children with recurrent leukemia.”

Additional contributors include Dorothea E. Myers, Zahide Ozer, Osmond J. D’Cruz and Hong Ma of the Children’s Center for Cancer and Blood Diseases and Children’s Hospital Los Angeles; Sanjive Qazi of CHLA and Gustavus Adolphus College, St. Paul, MN; and Rebecca Rose of Rose Pathology Services, St. Paul. The project was supported by a 2011 V-Foundation Translational Research Award and in part by DHHS grants R21-CA-164098, U01-CA-151837 and R01-CA-154471 from the National Cancer Institute, part of the National Institutes of Health.

About Children’s Hospital Los Angeles
Children’s Hospital Los Angeles has been named the best children’s hospital on the West Coast and among the top five in the nation for clinical excellence with its selection to the prestigious U.S. News & World Report Honor Roll. Children’s Hospital is home to The Saban Research Institute, one of the largest and most productive pediatric research facilities in the United States. Children’s Hospital is also one of America’s premier teaching hospitals through its affiliation since 1932 with the Keck School of Medicine of the University of Southern California.

Source: All Children’s Hospital Los Angeles

Shire to Acquire NPS Pharma as Further Step in Building a Leading Biotech

Dublin-based Shire has agreed to use its AbbVie breakup fee to acquire rare disease drugmaker NPS Pharmaceuticals, Inc.

The companies announced that they have entered into a merger agreement, under which Shire will acquire all the outstanding shares of NPS Pharma for $46 per share in cash, for a total consideration of approximately $5.2 billion. The deal strengthens Shires business in rare diseases.

Shire was first linked to NPS last May, however in July, Shire agreed to be acquired by US-based AbbVie, in a deal worth $55 billion. Last fall, the deal fell through after the Treasury Department announced new rules targeting companies trying to strike inversions deals, allowing US companies to reincorporate their businesses overseas. Since AbbVie decided to break the deal, it was required to pay Shire $1.6 billion in cash.

Shire’s acquisition of NPS will provide the company with two new drugs: Gattex, a treatment for short-bowel syndrome (SBS), and Natpara, a drug awaiting FDA-approval for treatment of hypoparathyroidism. The FDA is expected to make a decision on whether to approve Natpara by January 24.

“The acquisition of NPS Pharma is a significant step in advancing Shire’s strategy to become a leading biotechnology company. With our global strength and expertise in both rare diseases and GI, Shire is uniquely positioned to drive the continued success of Gattex/Revestive, and, if approved, commercialize NPS Pharma’s pipeline compound Natpara/Natpar,” said Shire’s Chief Executive Officer, Flemming Ornskov, MD, MPH.

Francois Nadar, MD, President, Chief Executive Officer and Director of NPS Pharma, said, “We look forward to accelerating the growth of the NPS Pharma portfolio based on our proven track record of maximizing value from acquired assets and commercial execution. The NPS Pharma organization will be a welcome addition to Shire will be a welcome addition to Shire as we continue to help transform the lives of patients with rare diseases.”

Source: Shire

Obama Announces Precision Medicine Initiative at State of the Union

During the State of the Union address, President Barack Obama announced his new “Precision Medicine” initiative to deliver new and more effective treatments for diseases, such as cancer and diabetes.

In his address, Obama urged Congress to increase research funding to support investments in precision medicine, a growing field of care in which treatments are tailored to an individual patient.

Precision medicine, also known as personalized medicine, gets rid of the “one-size-fits-all” approach to medicine. Precision medicine targets individuals who will benefit most from certain treatments by identifying the genetic cause for a disease in a specific group of people. He said that he wants the country to lead a new era of medicine that provides the right treatment at the right time.

Precision medicine includes sequencing the genes of patients to help doctors choose the appropriate drug to fight it, as well as sequencing genes to discover the root causes of other diseases. This strategy has helped reverse cystic fibrosis (CF) in some patients, including Bill Elder, who attended the address seated next to First Lady Michelle Obama. Elder was diagnosed with CF when he was eight, when patients with the disease commonly only lived to early adulthood. Elder, who is now 27 and is in his third-year of medical school, is among patients who have been successfully treated with Kalydeco (ivacaftor), which is approved in the US for patients with specific gene mutations.

Obama said that he is launching a new Precision Medicine Initiative in order to bring us closer to curing diseases like cancer and diabetes.

According to the National Institutes of Health (NIH), more than 1,800 genetic links to disease have been identified since the Human Genome Project, and there are more than 2,000 genetic tests for disease conditions. Researchers have been developing and looking into large databases of detailed patient records to understand the association between genetic variation and disease.

The President did not provide details of the initiative and how much it would cost, but expects these to be outlined in his fiscal 2016 budget, which will be released February 2.

Source: Specialty Pharma Journal

PBM Sees New Cancer and Cholesterol Drugs as Cost Savings Opportunity

Express Scripts, the nation’s largest pharmacy benefit manager (PBM), is looking for savings from new treatments for cancer and high cholesterol.

Now that Express Scripts and CVS Health, the nation’s second largest PBM, have negotiated discounts for new hepatitis C drugs, Express Scripts’ CEO said that new cholesterol and cancer drugs may be its next focus for negotiating discounts.

In December, Express Scripts announced that it would stop covering Gilead Sciences’ hepatitis C treatment Harvoni. The PBM struck a deal making AbbVie’s newly-approved Viekira Pak the exclusive option for its members with genotype 1 hepatitis C, in exchange for a discounted price. According to the company’s Chief Executive Officer George Paz, Express Scripts’ next cost-cutting targets include expensive new treatments for cancer and high cholesterol.

At the J.P. Morgan Healthcare Conference, Paz noted that the new cholesterol-lowering drugs, known as PCSK9 inhibitors, which significantly reduce bad cholesterol to low levels but are expensive, is an area of opportunity for cost savings. Like the new hepatitis C drugs, the PCSK9 medications are likely to enter the market around the same time, with Amgen and Regeneron in the lead and Pfizer not far behind. With high competition between these companies, PBMs will be in a position to impose pricing pressure to reduce costs.

However, Paz said that the big opportunity for cutting treatment costs is in cancer. He said that cost savings on cancer treatments could be achieved if the PBM were involved earlier in the decision making process. Currently, physicians run a patient’s cancer treatment regimen through a major medical benefits instead of through the PBM. However, according to Paz, if they did go through the PBM first, Express Scripts would get its doctors and pharmacists involved to ensure the prescribed regimens are appropriate. Although the physician makes the final decision, the PBM would advise on the most cost-effective, best regimen for the individual.

“The big opportunity out there is really in cancer,” said Paz. “If we can get out in front of that, that is a huge opportunity.”

Source: Specialty Pharma Journal

Roche acquires Trophos to expand portfolio in neuromuscular disease with high medical need

Swiss drugmaker Roche has signed an agreement to acquire privately-held Trophos for up to $545 million.

The acquisition will provide Roche with Trophos’ candidate for the rare and debilitating spinal muscular atrophy (SMA). Trophos’ proprietary screening platform generated olesoxime (TRO19622), which is in mid-stage development for SMA. SMA is a life-limiting and highly disabling genetic disease characterized by progressive muscle weakness and loss of motor function. It affects the motor neurons of the voluntary muscles used for activities like crawling, walking, head and neck control and swallowing. Typically the disease presents in early childhood, and is the most common genetic cause of infant mortality. SMA affects one in 6,000 to one in 10,000 children.

Results from a Phase II study of Trophos’ olesoxime in SMA showed a beneficial effect on the maintenance of neuromuscular function in individuals with Type II and non-ambulatory Type III SMA, as well as a reduction in medical complications associated with the disease.

“SMA is a grievous disease with a huge impact on the daily life of patients and their families, who are currently left only with supportive care. We are proud to see the development of this medicine evolving, with the ultimate goal of a potential first medicine for SMA,” said Christine Placet, Chief Executive Officer at Trophos. “This is a tremendous recognition of the work done by Trophos’ teams and supporters over the past 16 years.”

Under the agreement, Roche will pay Trophos’ shareholders roughly $139 million upfront and contingent payments of up to $406 million based on achievement of certain milestones.

“This acquisition highlights Roche’s commitment to developing medicines for spinal muscular atrophy, a serious disease with no effective treatment,” said Sandra Horning, MD, Chief Medical Officer and Head of Global Product Development at Roche. “We will build on the work done by Trophos and the French Muscular Dystrophy Association to advance the development of olesoxime and to bring it to people who live with this devastating condition as quickly as possible.”

The acquisition marks Roche’s fourth deal announced this week. In addition Roche’s deal with Trophos, the company acquired a majority stake in Foundation Medicine for $1.2 billion, and obtained rights from Meji Seika Pharma and Fedora Pharmaceuticals to develop an early-stage infection disease compound for up to $750 million.

Source: Roche