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Research by ClinMet Scientific Founder and Colleagues Point to Enzyme that Restores Function in Diabetic Kidney Disease

ClinMet recently announced that researchers from The University of California, San Diego School of Medicine and colleagues have published new findings that could fundamentally change understanding of how diabetes-related diseases develop – and how they might be better treated.

Sanofi Announces Upcoming Launch of MyStar Extra, the First Self-Monitoring Blood Glucose Meter With Estimated A1c

At the annual meeting of the European Association for the Study of Diabetes (EASD) in Barcelona, Spain, Sanofi (EURONEXT : SAN and NYSE : SNY) recently presented the innovative blood glucose meter MyStar Extra®, the first self-monitoring device that provides robust estimates of the A1c value, a key indicator for long-term glucose control.[3],[4] The hemoglobin A1C (HbA1C) assay has become the cornerstone for the assessment of diabetes control and A1c test results are widely used to guide treatment decisions.[5],[6] Especially convenient for people starting on insulin or using insulin, MyStar Extra® is a supportive meter, designed to help people with diabetes be engaged in their insulin management and treatment plan.[7],[8],[9]

University Of California, San Diego School Of Medicine To Investigate Risk Factors For Parkinson’s Disease Via Parkinson’s Progression Markers Initiative (PPMI)

University of California, San Diego School of Medicine will be one of 23 official clinical sites of the Parkinson’s Progression Markers Initiative’s (PPMI) new arm to study at-risk populations for Parkinson’s disease (PD). The $55 million landmark observational clinical study launched in 2010 to define one or more biomarkers of PD and now seeks to better understand potential risk factors of the disease. The University of California, San Diego School of Medicine has been a part of PPMI for three years and is currently enrolling for the new, pre-motor arm of the study.

The pre-motor arm of PPMI will enroll participants who do not have Parkinson’s disease and are living with one of three potential risk factors for PD: a reduced sense of smell (hyposmia); rapid eye movement sleep behavior disorder (RBD); or a mutation in the LRRK2 gene (the single greatest genetic contributor to PD known to date). Validating these risk factors could not only enable earlier detection of the disease, but open new avenues in the quest for therapies that could slow or stop disease progression.

“Understanding risk factors for Parkinson’s disease could help to identify therapies that may prevent the onset of motor symptoms in future generations of PD patients,” said Douglas Galasko, MD, the principal investigator for the study at the University of California, San Diego School of Medicine.

Local residents can easily get involved in this research by being one of 10,000 individuals needed to complete a brief online survey (www.michaeljfox.org/takethesmellsurvey) about their sense of smell. People over the age of 60 who do not have Parkinson’s disease are needed to take the survey. Most respondents will be sent a scratch-and-sniff smell test and brief questionnaire in the mail to be completed at home. Some individuals may also be asked to undergo more extensive testing.

“In the third year of PPMI, it is evident that a large-scale biomarker study is not only possible in Parkinson’s disease, but is already yielding scientific insights that could help transform the field of Parkinson’s research,” said Todd Sherer, PhD, CEO of The Michael J. Fox Foundation for Parkinson’s Research. “None of this progress would be possible without the willing volunteers who donate their time and energy to the pursuit of a cure.”

Source: PR Newswire

Roche and Isis Pharmaceuticals Form Alliance for Huntington’s Disease

Roche (SIX: RO, ROG; OTCQX: RHHBY) and Isis Pharmaceuticals, Inc (NASDAQ: ISIS) recently announced that they have formed an alliance to develop treatments for Huntington’s disease (HD) based on Isis’ antisense oligonucleotide (ASO) technology. This alliance combines Isis’ antisense expertise with Roche’s scientific expertise in developing neurodegenerative therapeutics. In addition, Isis and Roche will be collaborating to combine Isis’ ASOs and Roche’s proprietary “brain shuttle” program with the objective of increasing the brain penetration of ASOs with systemic administration.

Huntington’s disease is an inherited genetic brain disorder that results in the progressive loss of both mental faculties and physical control. Symptoms usually appear between the ages of 30 to 50, and worsen over a 10 to 25 year period. Ultimately, the weakened individual succumbs to pneumonia, heart failure or other complications. Presently, there is no effective treatment or cure for the disease, and current treatments focus on reducing the severity of some disease symptoms.

Initially, research will focus on Isis’ lead drug candidate that blocks production of all forms of the huntingtin (HTT) protein, the protein responsible for HD and thus has the potential to treat all HD patients. Isis is also conducting research into treatments that specifically block production of the disease-causing forms of the HTT protein which has the potential to treat subsets of HD patients. In parallel, Roche will combine its proprietary brain shuttle technology with Isis ASO technology that, if successful, will also allow systemic administration of antisense drugs to treat asymptomatic patients.

Under the terms of the agreement, Roche will make an upfront payment of $30 million to Isis, with total payments related to license fee and pre- and post-licensing milestone payments reaching potentially $362 million, including up to $80 million in potential commercial milestone payments. In addition, Isis will receive tiered royalties on sales of the drugs. Roche has the option to license the drugs from Isis through the completion of the first Phase 1 trial. Prior to option exercise, Isis is responsible for the discovery and development of an antisense drug targeting HTT protein. Roche and Isis will work collaboratively on the discovery of an antisense drug utilizing Roche’s “brain shuttle” program. If Roche exercises its option, it will be responsible for global development, regulatory and commercialization activities for all drugs arising out of the collaboration.

Commenting on the deal, Luca Santarelli, Head of Neuroscience and Small Molecules Research at Roche, said: “Huntington’s is a severely debilitating neurodegenerative disease and a large unmet medical need. Patients experience gradually worsening motor function and psychological disturbances, with a significant shortening of life expectancy after the disease is diagnosed. Treatments are urgently needed, and we believe that the Isis approach in combination with Roche’s brain shuttle represent one of the most advanced programs targeting the cause of HD with the aim of slowing down or halting the progression of this disease.”

Shafique Virani, Global Head Neuroscience, Cardiovascular & Metabolism at Roche Partnering, added: “Central to the partnership is Roche’s brain shuttle program, which we see as highly complementary to Isis’ drug development work. This dual track development program ensures whichever candidate compound proves to be most promising — Isis’ lead target or Roche’s brain shuttle version — can be taken forward to pivotal clinical trials.”

“We are pleased to be working with Roche, a global leader in drug development with significant experience in developing and commercializing drugs to treat neurological diseases. We believe that Roche’s expertise in developing CNS drugs, along with their clinical development experience, will greatly enhance our development efforts for this program and allow us to move forward more rapidly. In addition, by utilizing Roche’s brain shuttle technology with our antisense drug discovery capabilities, we have the potential to significantly improve the therapeutic potential for this program,” said B. Lynne Parshall, Chief Operating Officer of Isis. “By partnering our more complex and nuanced research and development programs earlier in development, like our Huntington’s disease CNS program, we add value and resources with partners that bring unique benefits.”

“We are excited to be working with Roche,” said Frank Bennett, Senior Vice President of Research at Isis. “We believe our mature antisense drug discovery platform is a perfect fit for Roche’s neuroscience franchise, and we anticipate a fruitful collaboration to advance our pre-clinical compounds.”

CHDI Foundation, a non-profit foundation exclusively dedicated to the development of therapies that slow the progression of HD, provided financial and scientific support to Isis’ HD drug discovery program through a development collaboration with Isis. CHDI’s support has enabled Isis to make significant progress in discovering a drug to treat HD. Together Isis and CHDI demonstrated that antisense compounds can be used to inhibit the production of HTT protein in both brain and peripheral tissues, and that the inhibition of normal HTT protein was well tolerated. Over time, CHDI will be reimbursed for its support of Isis’ program out of the milestone payments received by Isis. CHDI will receive $1.5 million associated with the signing of the Roche agreement. CHDI will continue to provide advice to Isis and Roche on the development of antisense drugs to treat patients with HD.

Isis also recognizes the tremendous benefit provided to its HD program by its academic collaborators, Drs. Don Cleveland at the Ludwig Institute, University of California San Diego and David Corey at University of Texas Southwestern. These collaborators have been instrumental in Isis’ early preclinical work demonstrating that antisense drugs can inhibit the HTT protein and produce activity in animal models of disease.

Source: Isis Pharmaceuticals

Pilot Study Demonstrates Home BNP Testing Is Feasible & Suggests Home Monitoring May Have Value in Guiding Therapy for High-Risk Patients

Alere Inc. (NYSE: ALR), a leading provider of near-patient diagnostics and health information solutions, recently announced the final results of the HABIT pilot study, which were recently published in the Journal of the American College of Cardiology. Led by Dr. Alan Maisel , Professor of Medicine at the University of California, San Diego, the study is the first to capture serial data from patients at high risk for recurrent acute clinical heart failure decompensation (ADHF), who performed a fingerstick BNP self-test from home for a period of 60 days. Results not only demonstrate that home BNP testing is safe and feasible for heart failure patients, but also indicate that BNP patterns following treatment for ADHF provide a wealth of information that may facilitate more personalized treatment leading to significant outcomes benefits.

In the United States, nearly one out of every four patients hospitalized for ADHF is rehospitalized within 30 days of discharge from the hospital, and the bulk of the costs associated with managing these patients derives from rehospitalization. Consensus among clinical experts is that more than 50% of hospital readmissions can be prevented through increased attention to modifiable factors that affect pulmonary congestion. But, while shortness of breath, edema, and weight gain can often signal pulmonary congestion, these symptoms may not appear in up to one-third of all heart failure patients. Changes in natriuretic peptide (NP) levels, along with monitoring for symptoms, signs, and weight changes, have been shown to improve the certainty of predicting heart failure decompensation. Up until now, however, NP measurements have been excluded from home heart failure monitoring programs because of the need for phlebotomy.

The HABIT study, a multi-center, single-arm, double-blinded observational prospective clinical trial, was designed to monitor daily concentrations of B-type natriuretic peptide (BNP) and determine the extent to which they correlate with ADHF and related adverse outcomes. A total of 163 patients with ADHF who were discharged from the hospital or being treated in an outpatient setting measured their weight and BNP levels daily for a period of 60 days using a fingerstick test run on the Alere™ Heart Check. Adverse outcomes for ADHF were measured as a composite of events that included cardiovascular death, admission for decompensated heart failure, or clinical heart failure decompensation requiring either parenteral therapy or adjustments to oral medications.

A total of 6,934 daily BNP values were recorded, with a median of 46 measures per patient over the course of the monitoring period. 40 patients had 56 events. Correlations between BNP measures weakened over time, and the dispersion between measures grew. When the monitoring period for each subject was broken into intervals based on ADHF events, there were 39 (18.4%) intervals of upward-trending BNP corresponding to a risk increase of 59.8% and 64 (30.2%) downward-trending intervals corresponding to a risk decline of 39.0%. There were also 94 (44.3%) intervals with one or more days of weight gain corresponding to an increased risk of 26.1%. Investigators concluded that daily weight monitoring is complementary to BNP measurement, but changes in BNP levels ultimately signaled larger shifts in risk, both upward and downward.

“Our results demonstrate that it is feasible and safe for heart failure patients to measure their results at home on a daily basis,” said Alan Maisel . “The incremental data from serial home measurements following an episode of ADHF appear to provide a novel means with which to identify those patients who are at highest risk of recurrent decompensation. The pattern or trend in BNP values may be used to identify when to correlate with a face-to-face clinical assessment or therapeutic intervention, or to guide therapy with specific medication changes without additional office visits.”

Investigators have determined that the results of the HABIT pilot study warrant an interventional trial to measure the extent to which home BNP testing impacts formal outcomes measures that include hospital readmissions. A study comparing today’s standard of care, which involves monitoring symptoms of heart failure and body weight changes following hospital discharge under the management of hospital-based outpatient heart failure clinics, to an algorithm that incorporates routine, home BNP measurements is set to commence before the end of 2013.

Study: Primary Results of the HABIT Trial (Heart Failure Assessment With BNP in the Home)

Source: PR Newswire