Press Releases

SIGNAL completes enrollment in cohort A

vaccinex_logoVaccinex Inc. completed enrollment of cohort A in its SIGNAL Clinical Trial, a randomized Phase 2 study to assess the safety, tolerability, pharmacokinetics, and efficacy of VX15/2503 in subjects at risk for or with early signs of Huntington’s Disease. Read the press release.

Announcing HSG’s 2015 Award Winners

The Huntington Study Group (HSG) is proud to announce this year’s award winners, including the inaugural Shoulson and New Member Scholars. Through these awards, HSG recognizes the hard work of veteran investigatos and coordinators and encourages individuals wishing to pursue a career in HD research.

Anne Young receives the Lifetime Achievement award from Ira Shoulson.

Anne Young receives the Lifetime Achievement award from Ira Shoulson.

Lifetime Achievement

Anne Young, M.D., Ph.D., a co-founder of the Huntington Study Group in 1993, has dedicated her life’s work to finding treatments for neurodegenerative diseases. Her research has shed light on how HD damages the basal ganglia in the brain and has been a springboard for testing novel treatment options.

Investigator of the Year

Andrew Feigin, M.D., director of Neuroscience Experimental Therapeutics at the Feinstein Institute for Medical Research, has been an invaluable member of HSG for many years. In recent years, he has served as a principal investigator, including for the recently launched SIGNAL trial.

Coordinator Mentor

Jody Goldstein, Senior Project Manager at the University of Rochester’s Clinical Trials Coordination

Jody Goldstein receives the Coordinator Mentor award from Jody Corey Bloom.

Jody Goldstein receives the Coordinator Mentor award from Jody Corey Bloom.

Center, has long been dedicated to Huntington disease since her time as a coordinator at the HSG Site at the University of California, San Diego. She forged strong bonds with HD families while developing top-notch skills as a coordinator. At the University of Rochester, she now mentors coordinators across HSG’s network to be more efficient, more engaged and more passionate about finding treatments that make a difference for HD.

Shoulson Scholars
In honor of HSG’s founder, Dr. Ira Shoulson, the HSG has established the Shoulson Scholar Fund. This fund recognizes outstanding junior investigators for promising research in Huntington disease. The annual fund is awarded in support of recipient travel and attendance of the annual HSG meeting.

This year’s recipients are:

  • Kelly Andrejewski, University of Rochester
  • Nancy Downing, University of Iowa
  • George McNally, University of Birmingham

The Shoulson Scholarships have been provided through the generosity of the Fox Family Foundation and the Griffin Foundation.

New Member
Like the Shoulson Scholarship, the New Member Scholarship program supports travel to and attendance of our annual meeting for individuals interested in furthering their training and education. This year’s recipients are:certificate

Jamie Adams, University of Rochester
Jamie Archer, Washington University School of Medicine
Setareh Ashtiani, University of Alberta Hospital
Karen Ayala, University of Texas, Austin
Fang Ba, University of Alberta
Miriam Batule Dominguez, Acta Medica del Centro
Julie Bonano, Virginia Commonwealth University
Katherine Eder McDonell, Vanderbilt University
Hanne Files, Calvary Health Care Bethlehem
Joshua Grill, University of California, Irvine
Andrea Higgins, Bloomfield Hospital
Jennifer Hoblyn, Bloomfield Hospital
Jared Husketh, HD Reach
Melinda Kavanaugh, Medical College of Wisconsin
Chris Lamb, Baptist Medical Center of Jacksonville
Guillaume Lamotte, Georgetown University
Terrence McGill, Westmead Hospital
Jennifer Nichols, University of Pittsburgh Medical Center
Noberta Robertson, Hereditary Neurological Disease Center
Behrang Saminejad, University of Utah
Madeline Sharp, Columbia University

The New Member scholarships have been provided through the generosity of following sponsors:
Teva Pharmaceuticals
Valeant Pharmaceuticals
Lundbeck, Inc.
Raptor Pharmaceuticals
Prana Biotechnology
Vaccinex, Inc.
CHDI Foundation
Pfizer
LifeMax Laboratories
Roche

Both groups of scholars represent the next generation of investigators, coordinators, social workers, genetic counselors, who are joining the effort to seek treatments that make a difference for HD.

HSG is also proud to welcome the recipients of HD Insights’ Insights of the Year award winners:
Janelle Drouin-Ouelette, Lund University
Stephen Smith, Seattle Children’s Hospital
Edward Wild, University College London
William Yang, University of California, Los Angeles

And HDSA’s Human Biology Scholars:
Helen Budworth, Lawrence Berkeley National Laboratory
Tanya Garcia, University of Texas, Houston
Jun Hua, Johns Hopkins University

Regional Health Care Practitioners Invited to Learn More about Huntington Disease Oct. 23

Day-long course offering continuing education credits through USF

The Huntington Study Group (HSG), in support of the goal to make care for Huntington disease (HD) patients more available, is holding a day-long education opportunity for health care providers in Tampa on Friday, Oct. 23. “Practical Pointers and Perspectives on Huntington Disease for Local Practitioners” is part of HSG’s annual research and educational event, which, this year, is called HSG 2015: Building Our Future.

HD is uncommon, but not all that rare. Clinicians, nurses, social workers, etc., specializing in neurology, movement disorders, mental health, genetics and palliative care, as well as primary care providers, are likely to see HD patients and families who need their guidance through the complex and progressive course of the disease. This course seeks to empower providers with the knowledge and resources they need to care for HD patients.

The course, provided jointly by USF Health and HSG, is designed for health care providers in neurology and general clinicians, which has been approved for continuing education credits. The fee is $100 per registrant and includes CE credits.

Attendees will leave with:

  • An up-to-date understanding of the diagnosis, staging and typical symptoms of HD
  • Skills for expert approaches to treatment
  • Understand the role of allied health in the management of HD
  • Awareness of the available medical, education and support resources

Register here!

More details can be found here.

For more information:

Heather Hare

(585) 242-0277

Heather.Hare@hsglimited.org

 

Teva Press Release: April 24, 2014

PRIDE-HD Study Enrolling Patients Globally to Further Evaluate Pridopidine for the Symptomatic Treatment of Huntington’s Disease

– Study initiation represents significant milestone for patients with a disease with limited effective treatment options –

JERUSALEM–(BUSINESS WIRE)–Apr. 24, 2014– Teva Pharmaceutical Industries Ltd. (NYSE: TEVA) announced today enrollment of the first patient in The Pride-HD study, a phase II, randomized, double-blind, placebo-controlled global study designed to evaluate the impact of pridopidine, an investigational medication, on motor impairment in patients with Huntington’s disease (HD).

“Huntington’s disease represents a significant unmet medical need as there are currently no treatments that improve the motor movements that are crucial for gait, balance and coordination –things that greatly impact a patient,” said Professor G. Bernhard Landwehrmeyer, M.D., Ph.D, FRCP, lead study investigator and professor of neurology, Ulm University Hospital, Germany. “Based on previous observations using the compound, we believe pridopidine holds promise for symptomatic relief with an acceptable safety profile.”

“Existing treatments aren’t appropriate for some patients due to side effects and the predominant effect on involuntary movements,” said Karl Kieburtz, M.D., study investigator and director of the Clinical & Translational Science Institute, University of Rochester Medical Center. “Based on the preliminary clinical evidence to date, we believe pridopidine has the potential to make a real difference in the lives of HD patients and families.”

The start of patient enrollment in The Pride-HD Study represents the latest milestone in Teva’s commitment to developing medicines to improve the quality of life for patients suffering from devastating CNS diseases, such as Huntington’s disease.

“People with HD are in urgent need of new treatments and we are committed to investigating the potential benefit of pridopidine as quickly as possible,” said Michael Hayden, M.D., a leading expert in the study of Huntington’s disease and President of Global R&D and Chief Scientific Officer at Teva.

“Pridopidine has shown promising results in previous advanced-stage clinical trials and merits additional study, as it has the potential to have a significant effect on Total Motor Score (TMS) – the endpoint most commonly used in the assessment of treatment efficacy in HD,” said Dr. Ralf Reilmann, study investigator and founding director and C.E.O., George-Huntington-Institute, Münster, Germany.

ABOUT THE PRIDE-HD STUDY

The Pride-HD Study, a phase II, dose-finding, randomized, parallel-group, double-blind, placebo-controlled study, that aims to enroll approximately 400 patients at 30 sites across the globe and evaluate the safety and efficacy of pridopidine 45 mg, 67.5 mg, 90 mg, and 112.5 mg twice daily (bid) versus placebo for symptomatic treatment in patients with HD.

The primary objective will be to assess the efficacy of pridopidine on motor impairment after 26 weeks of treatment using the Unified Huntington’s Disease Rating Scale (UHDRS) Total Motor Score (TMS). The study will also examine the effect of treatment with pridopidine on the Physical Performance Test (PPT), as well as the safety and tolerability across the range of pridopidine doses in patients with HD during the 26 weeks of treatment.

Qualifying patients for The Pride-HD study must be 21 years of age or older, with an onset of HD after age 18 and must have a diagnosis of HD based on clinical features and the presence of ≥36 cytosine-adenosine-guanine (CAG) repeats in the HTT gene. More information about the study can be found athttp://clinicaltrials.gov/ct2/show/NCT02006472 or by calling 1-800-896-5855.

ABOUT PRIDOPIDINE

Pridopidine is an investigational, oral, small molecule being developed for the symptomatic treatment of Huntington’s disease (HD). Teva intends to design and complete new clinical studies of pridopidine to assess its potential for symptomatic relief of HD. Earlier clinical studies of pridopidine conducted in the U.S., EU and Canada in patients with HD indicate a significant treatment effect on an important secondary endpoint, Total Motor Score (TMS).

In previous studies, where doses up to 45 mg bid were tested, pridopidine was well tolerated with an adverse event profile similar to placebo, and treatment with pridopidine was not associated with worsening of disease signs and symptoms.

ABOUT HUNTINGTON’S DISEASE

Huntington’s disease (HD) is a fatal neurodegenerative disease characterized by uncoordinated and uncontrollable movements, cognitive deterioration and behavioral and/or psychological problems. The classic onset of HD symptoms typically occurs in middle age, but the disease also manifests in children and the elderly. HD is the most common genetic cause of abnormal involuntary writhing movements called chorea. Disease progression is characterized by a gradual decline in motor control, cognition and mental stability and generally results in death within 15‐25 years of clinical diagnosis.

HD is a genetic disease, passed from parent to child through a gene mutation. Each child of an HD parent has a 50-50 chance of inheriting the HD gene. If a child does not inherit the HD gene, he or she will not develop the disease and cannot pass it to subsequent generations. A person who inherits the HD gene will sooner or later develop the disease. According to the World Health Organization, Huntington’s disease affects about five to seven people per 100,000 in Western countries.

ABOUT TEVA

Teva Pharmaceutical Industries Ltd. is a leading global pharmaceutical company, committed to increasing access to high-quality healthcare by developing, producing and marketing affordable generic drugs as well as innovative and specialty pharmaceuticals and active pharmaceutical ingredients. Headquartered in Israel, Teva is the world’s leading generic drug maker, with a global product portfolio of more than 1,000 molecules and a direct presence in approximately 60 countries. Teva’s branded businesses focus on CNS, oncology, pain, respiratory and women’s health therapeutic areas as well as biologics. Teva currently employs approximately 45,000 people around the world and reached $20.3 billion in net revenues in 2013.

Safe Harbor Statement under the U.S. Private Securities Litigation Reform Act of 1995:

This release contains forward-looking statements, which are based on management’s current beliefs and expectations and involve a number of known and unknown risks and uncertainties that could cause our future results, performance or achievements to differ significantly from the results, performance or achievements expressed or implied by such forward-looking statements. Important factors that could cause or contribute to such differences include risks relating to: our ability to develop and commercialize additional pharmaceutical products; competition for our innovative products, especially COPAXONE® (including competition from orally-administered alternatives, as well as from potential purported generic equivalents); the possibility of material fines, penalties and other sanctions and other adverse consequences arising out of our ongoing FCPA investigations and related matters; our ability to achieve expected results from the research and development efforts invested in our pipeline of specialty and other products; our ability to reduce operating expenses to the extent and during the timeframe intended by our cost reduction program; our ability to identify and successfully bid for suitable acquisition targets or licensing opportunities, or to consummate and integrate acquisitions; the extent to which any manufacturing or quality control problems damage our reputation for quality production and require costly remediation; our potential exposure to product liability claims that are not covered by insurance; increased government scrutiny in both the U.S. and Europe of our patent settlement agreements; our exposure to currency fluctuations and restrictions as well as credit risks; the effectiveness of our patents, confidentiality agreements and other measures to protect the intellectual property rights of our specialty medicines; the effects of reforms in healthcare regulation and pharmaceutical pricing, reimbursement and coverage; governmental investigations into sales and marketing practices, particularly for our specialty pharmaceutical products; uncertainties related to our recent management changes; the effects of increased leverage and our resulting reliance on access to the capital markets; any failure to recruit or retain key personnel, or to attract additional executive and managerial talent; adverse effects of political or economical instability, major hostilities or acts of terrorism on our significant worldwide operations; interruptions in our supply chain or problems with internal or third-party information technology systems that adversely affect our complex manufacturing processes; significant disruptions of our information technology systems or breaches of our data security; competition for our generic products, both from other pharmaceutical companies and as a result of increased governmental pricing pressures; competition for our specialty pharmaceutical businesses from companies with greater resources and capabilities; decreased opportunities to obtain U.S. market exclusivity for significant new generic products; potential liability in the U.S., Europe and other markets for sales of generic products prior to a final resolution of outstanding patent litigation; any failures to comply with complex Medicare and Medicaid reporting and payment obligations; the impact of continuing consolidation of our distributors and customers; significant impairment charges relating to intangible assets and goodwill; potentially significant increases in tax liabilities; the effect on our overall effective tax rate of the termination or expiration of governmental programs or tax benefits, or of a change in our business; variations in patent laws that may adversely affect our ability to manufacture our products in the most efficient manner; environmental risks; and other factors that are discussed in our Annual Report on Form 20-F for the year ended December 31, 2013 and in our other filings with the U.S. Securities and Exchange Commission. Forward-looking statements speak only as of the date on which they are made and we assume no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Source: Teva Pharmaceutical Industries Ltd.

IR:
Kevin C. Mannix, United States, 215-591-8912
Ran Meir, United States, 215-591-3033
Tomer Amitai, Israel, (972 (3) 926-7656
or
PR:
Iris Beck Codner, Israel, 972 (3) 926-7687
Denise Bradley, United States, 215-591-8974
Nancy Leone, United States, 215-284-0213

This article originally appeared on tevapharm.com, April 24, 2014: http://ir.tevapharm.com/phoenix.zhtml?c=73925&p=irol-newsArticle_Print&ID=1921917

Teva & Active Biotech Press Release: November 4, 2014

Teva and Active Biotech Announce Expansion of Laquinimod Clinical Development Program with New Trial in Primary Progressive Multiple Sclerosis and First Patient Screened in Huntington’s Disease Trial

2014-11-04

ARPEGGIO and LEGATO-HD trials will further evaluate the effect of laquinimod in neurodegenerative diseases

Jerusalem & Lund, Sweden – November 4, 2014 – Teva Pharmaceutical Industries Ltd. (NYSE:TEVA) and Active Biotech (NASDAQ OMX NORDIC:ACTI) today announced the expansion of the laquinimod clinical development program with the initiation of the ARPEGGIO trial, which will evaluate the potential of laquinimod to treat primary progressive multiple sclerosis (PPMS). Additionally, Teva has screened the first patient in the LEGATO-HD trial, which will evaluate laquinimod in Huntington’s disease. Currently, there are no approved therapies available for the treatment of PPMS or the treatment of Huntington’s disease, beyond symptom management.

“Teva prides itself in striving to help patients with neurodegenerative diseases through research and innovation,” said Michael Hayden, M.D., Ph.D., President of Global R&D and Chief Scientific Officer at Teva Pharmaceutical Industries, Ltd. “Laquinimod has been shown to modulate several significant pathways common to key neurodegenerative disease. More specifically, it modulates the immune cell lineages in the periphery and in the CNS. We look forward to the results from both of these studies.”

The ARPEGGIO study will evaluate the efficacy, safety and tolerability of laquinimod in patients with PPMS with a primary endpoint of percent brain volume change (PBVC) through MRI analysis. PPMS is characterized by the worsening of neurologic function without distinct relapses (also called attacks or exacerbations). Approximately 15 percent of MS patients fall into the PPMS category.

The LEGATO-HD study will evaluate the efficacy, safety and tolerability of once-daily oral laquinimod as a potential treatment for adult patients with Huntington’s disease. The primary endpoint for LEGATO-HD is change from baseline in the Unified Huntington’s Disease Rating Scale-Total Motor Scale (UHDRS-TMS) as defined by the sum of the scores of all UHDRS-TMS sub-items after 12 months of treatment. Huntington’s disease is caused by a genetically-programmed degeneration of brain cells in select areas of the brain, which results in uncontrolled movements, loss of intellectual faculties and personality and emotional disturbances. Huntington’s disease affects about five to seven people per 100,000 in Western countries.

For further details on the Phase II ARPEGGIO and LEGATO-HD studies, please search laquinimod at ClinicalTrials.gov.

About ARPEGGIO

The study, A Randomized Placebo-controlled Trial Evaluating Laquinimod in PPMS, Gauging Gradations In MRI and Clinical Outcomes (ARPEGGIO) is a multinational, multicenter, randomized, double-blind, parallel-group, placebo-controlled, Phase II clinical trial.

ARPEGGIO is intended to serve as a proof-of-concept study for potential treatment with laquinimod in PPMS. The trial will evaluate two doses of laquinimod (0.6 and 1.5mg/day) in PPMS compared to placebo. The primary endpoint of the study is brain atrophy as defined by PBVC from baseline to week 48. Secondary endpoints include time to confirmed disability progression, the number of new T2 lesions and change in the Brief International Cognitive Assessment for Multiple Sclerosis (BICAMS) score. ARPEGGIO has an estimated completion date of H2 2017.

About LEGATO-HD

LEGATO-HD (Laquinimod Efficacy and Safety in a GlobAl Trial Of HD) is a multicenter, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of laquinimod treatment at doses of 0.5, 1.0, and 1.5 mg/day in 400 adult Huntington’s disease patients between the ages of 21-55. Ancillary studies will evaluate microglia activation, neuronal integrity, and peripheral inflammatory biomarkers. LEGATO-HD has an estimated completion date of H1 2017.

The primary endpoint of LEGATO-HD is change from baseline in the Unified Huntington’s Disease Rating Scale-Total Motor Scale (UHDRS-TMS) as defined by the sum of the scores of all UHDRS-TMS sub-items after 12 months of treatment. Secondary endpoints will measure brain atrophy, cognition, clinical global impression and functional capacity.

About Laquinimod

Laquinimod is a once-daily oral, investigational, CNS-active immunomodulator with a novel mechanism of action being developed for the treatment of relapsing-remitting MS (RRMS), progressive MS and Huntington’s disease. The global, Phase III, clinical development program evaluating laquinimod in MS includes two completed pivotal studies, ALLEGRO and BRAVO (both 0.6mg/day). A third Phase III trial, CONCERTO, is currently ongoing and evaluating two doses of laquinimod (0.6mg and 1.2mg/day) in approximately 2,100 patients for up to 24 months. The primary outcome measure is time to three-month confirmed-disability progression as measured by the Expanded Disability Status Scale (EDSS).

In the ALLEGRO and BRAVO trials, adverse reactions observed included headache, abdominal pain, back and neck pain, appendicitis, and mild, asymptomatic laboratory abnormalities, including liver enzyme elevations, hematological changes and elevation of CRP or fibrinogen levels.

About Teva

Teva Pharmaceutical Industries Ltd. (NYSE:TEVA) is a leading global pharmaceutical company, committed to increasing access to high-quality healthcare by developing, producing and marketing affordable generic drugs as well as innovative and specialty pharmaceuticals and active pharmaceutical ingredients. Headquartered in Israel, Teva is the world’s leading generic drug maker, with a global product portfolio of more than 1,000 molecules, sold in more than 100 countries, and with a direct presence in about 60 countries. Teva’s specialty medicine businesses focus on CNS, including pain, respiratory, oncology, and women’s health therapeutic areas as well as biologics. Teva currently employs approximately 45,000 people around the world and reached $20.3 billion in net revenues in 2013.

About Active Biotech

Active Biotech AB (NASDAQ OMX NORDIC:ACTI) is a biotechnology company with focus on autoimmune/inflammatory diseases and cancer. In pivotal phase is laquinimod, an orally administered small molecule with unique immunomodulatory properties for the treatment of multiple sclerosis. Also tasquinimod for the treatment of prostate cancer, with a unique mode of action, is in pivotal phase. In addition, laquinimod has concluded Phase II development for Crohn’s and Lupus. The company has two additional projects in clinical development, ANYARA primarily for the treatment of renal cell cancer and the orally administered compound paquinimod (57-57) for systemic sclerosis. Please visit www.activebiotech.com for more information.

For further information, please contact:

Tomas Leanderson, CEO

Tel: +46 (0)46 19 20 95

E-mail: tomas.leanderson@activebiotech.com

 

Hans Kolam, CFO

Tel: +46 (0)46 19 20 44

E-mail: hans.kolam@activebiotech.com

 

Active Biotech AB

Box 724, SE-220 07 Lund, Sweden

Phone +46 (0)46 19 20 00

Fax +46 (0)46 19 11 00

Safe Harbor Statement under the U.S. Private Securities Litigation Reform Act of 1995:

This release contains forward-looking statements, which are based on management’s current beliefs and expectations. Such statements involve a number of known and unknown risks and uncertainties that could cause our future results, performance or achievements to differ significantly from the results, performance or achievements expressed or implied by such forward-looking statements. Important factors that could cause or contribute to such differences include risks relating to: our ability to develop and commercialize additional pharmaceutical products; competition for our innovative products, especially Copaxone® (including competition from orally-administered alternatives, as well as from potential generic versions); the possibility of material fines, penalties and other sanctions and other adverse consequences arising out of our ongoing FCPA investigations and related matters; our ability to achieve expected results from the research and development efforts invested in our pipeline of specialty and other products; our ability to reduce operating expenses to the extent and during the timeframe intended by our cost reduction program; our ability to successfully pursue and consummate suitable acquisitions or licensing opportunities; the extent to which any manufacturing or quality control problems damage our reputation for quality production and require costly remediation; our potential exposure to product liability claims that are not covered by insurance; increased government scrutiny in both the U.S. and Europe of our patent settlement agreements; our exposure to currency fluctuations and restrictions as well as credit risks; the effectiveness of our patents and other measures to protect the intellectual property rights of our specialty medicines; the effects of reforms in healthcare regulation and pharmaceutical pricing, reimbursement and coverage; governmental investigations into sales and marketing practices, particularly for our specialty pharmaceutical products; uncertainties related to our recent management changes; the effects of increased leverage and our resulting reliance on access to the capital markets; any failure to recruit or retain executives or other key personnel; adverse effects of political or economical instability, major hostilities or acts of terrorism on our significant worldwide operations; interruptions in our supply chain or problems with internal or third-party information technology systems that adversely affect our complex manufacturing processes; significant disruptions of our information technology systems or breaches of our data security; competition for our generic products, both from other pharmaceutical companies and as a result of increased governmental pricing pressures; competition for our specialty pharmaceutical businesses from companies with greater resources and capabilities; decreased opportunities to obtain U.S. market exclusivity for significant new generic products; potential liability for sales of generic products prior to a final resolution of outstanding patent litigation; any failures to comply with complex Medicare and Medicaid reporting and payment obligations; the impact of continuing consolidation of our distributors and customers; significant impairment charges relating to intangible assets and goodwill; the potential for significant tax liabilities; the effect on our overall effective tax rate of the termination or expiration of governmental programs or tax benefits, or of a change in our business; variations in patent laws that may adversely affect our ability to manufacture our products in the most efficient manner; environmental risks; and other factors that are discussed in our Annual Report on Form 20-F for the year ended December 31, 2013 and in our other filings with the U.S. Securities and Exchange Commission. Forward-looking statements speak only as of the date on which they are made and we assume no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Active Biotech’s Safe Harbor Statement in Accordance with the Swedish Securities Market Act

This press release contains certain forward-looking statements. Such forward-looking statements involve known and unknown risks, uncertainties and other important factors that could cause the actual results, performance or achievements of the company, or industry results, to differ materially from any future results, performance or achievement implied by the forward-looking statements. The company does not undertake any obligation to update or publicly release any revisions to forward-looking statements to reflect events, circumstances or changes in expectations after the date of this press release.

Active Biotech is obligated to publish the information contained in this press release in accordance with the Swedish Securities Market Act. This information was provided to the media for publication 02:00 pm CET on November 4, 2014.

This article originally appeared on www.activebiotech.com, December, 16, 2014: http://www.activebiotech.com/press-releases-1?pressid=1868301

 

Auspex Press Release: December 16, 2014

Auspex Announces Positive Topline Results From Registration Trial of SD-809 for Chorea Associated With Huntington’s Disease

Pivotal Phase 3 Trial Met Primary and Multiple Key Secondary Efficacy Endpoints; Favorable Safety and Tolerability Demonstrated

ARC-HD Trial Shows Successful Maintenance of Chorea Control After Switch to SD-809

NDA Submission Planned by Mid-2015

Conference Call Scheduled for 4:30 PM ET / 1:30 PM PT Today

LA JOLLA, Calif., Dec. 16, 2014 (GLOBE NEWSWIRE) — Auspex Pharmaceuticals, Inc. (Nasdaq:ASPX), a biopharmaceutical company dedicated to developing innovative medicines for people with movement disorders and other rare diseases, today announced positive topline efficacy and safety results from its Phase 3 registration trial evaluating SD-809 for the treatment of chorea associated with Huntington’s disease (HD), called First-HD. In addition to meeting the primary efficacy endpoint, significant improvements in both patient and clinical global impressions of change and quality of life were observed. Importantly, the study showed a favorable safety and tolerability profile, including low rates of depression, somnolence, akathisia/restlessness and anxiety. In addition, Auspex announced results from an analysis of the completed four-week Switch portion of the ARC-HD study, which also has an ongoing long-term safety component. Data from the study show that patients who switched from the current standard of care, tetrabenazine, to SD-809 maintained chorea control at both week one and week four.

Huntington’s disease is a genetic disorder that causes a wide variety of symptoms including involuntary movements, problems with emotion, behavior, thinking, processing information, and ultimately leads to death. Approximately 90 percent of patients with Huntington’s disease will develop chorea, which is characterized by involuntary, excessive movements that can impact all parts of the body and interferes with motor functions. As a result, chorea associated with Huntington’s disease can be severely debilitating.

“For many individuals with Huntington’s disease, chorea is a key symptom impacting safety, function and quality of life. New, safe and tolerable therapies for chorea treatment are clearly needed to make this disease an increasingly treatable condition,” said Samuel A. Frank, M.D., associate professor of neurology, Boston University School of Medicine and principal investigator for First-HD. “The primary and secondary efficacy results from this study were confirmed by the Huntington Study Group independent analysis. These clear and unequivocal results are clinically meaningful and suggest that SD-809 may play an important role in the treatment of Huntington’s disease symptoms.”

First-HD Topline Results

First-HD was a 1:1 randomized, double-blind, placebo-controlled, parallel-group trial evaluating the efficacy, safety and tolerability of SD-809 in the management of chorea associated with Huntington’s disease. The primary efficacy endpoint for the study was the change from baseline to maintenance therapy in the Total Maximal Chorea (TMC) score of the Unified Huntington’s Disease Rating Scale (UHDRS). There were four pre-specified key secondary endpoints that were tested on a hierarchical basis: treatment success based on patient global impression of change (PGIC) and clinical global impression of change (CGIC), quality of life and balance. Other pre-specified motor endpoints were also analyzed. A total of 90 patients (45 in each group) were enrolled for evaluation over 13 weeks: patients were titrated weekly to an optimal dose up to week eight; were on maintenance therapy for four weeks, and; were taken off study medication in the final week of the study. A total of 87 patients completed the study; one patient in the SD-809 group and two in the placebo group discontinued.

SD-809 met the pre-specified primary efficacy endpoint. Patients taking SD-809 achieved a meaningful improvement of 2.5 points on the TMC score from baseline to maintenance therapy compared to placebo (p < 0.0001). Additional results from First-HD are as follows:

Pre-Specified Motor Endpoints SD-809 Placebo Treatment Effect Favors P-Value
Change in TMC Score1 from
Baseline to Maintenance Therapy*
4.4 Point
Improvement
1.9 Point
Improvement
Improvement of
2.5 Points over Placebo
SD-809 p < 0.0001
Percent Change in TMC Score
from Baseline to Maintenance Therapy
37 Percentage Points
Improvement
16 Percentage
Points Improvement
Improvement of
21 Percentage Points
over Placebo
SD-809 p < 0.0001
Change in Total Motor Score1 (TMS)
from Baseline to Maintenance Therapy
7.4 Point
Improvement
3.4 Point
Improvement
Improvement of
4.0 Points over Placebo
SD-809 p = 0.002
1TMC and TMS are subscales of the Unified Huntington’s Disease Rating Scale (UHDRS)
*Primary efficacy endpoint

The clinical relevance of the change in chorea was assessed by four pre-specified secondary endpoints. As summarized in the following table, the first three key secondary endpoints, including two patient-rated measures of benefit, showed statistically significant superiority of SD-809 over placebo.

Pre-specified Key Secondary Endpoints2 Favors P-Value
1. Patient Global Impression of Change (PGIC)3 SD-809 p = 0.002
2. Clinical Global Impression of Change (CGIC)3 SD-809 p = 0.002
3. SF-36 Physical Functioning Score (a Quality of Life measure) from Baseline to Week 12 SD-809 p = 0.03
4. Berg Balance Test SD-809 p = 0.14
2 Analyzed using a pre-specified hierarchical testing procedure
3 Success defined as much improved or very much improved

In general, SD-809 was well tolerated; there was no difference in the rate of dose reduction between SD-809 and placebo (6.7% in each group). Results from First-HD show a favorable safety and tolerability profile of SD-809; following are the number of patients reporting adverse events by system organ class:

System Organ Class SD-809
n = 45
Placebo
n = 45
Psychiatric Disorders 8 (17.8%) 8 (17.8%)
Nervous System Disorders 8 (17.8%) 10 (22.2%)
All Other Body Systems
Cardiac Disorders 0 (0.0%) 3 (6.7%)
Ear & Labyrinth 1 (2.2%) 1 (2.2%)
Eye Disorders 1 (2.2%) 1 (2.2%)
General Disorders 7 (15.6%) 8 (17.8%)
Gastrointestinal Disorders 9 (20%) 9 (20%)
Hepatobiliary Disorders 1 (2.2%) 0 (0.0%)
Infections and Infestations 5 (11.1%) 5 (11.1%)
Injury, Poisoning and Procedural Complications 4 (8.9%) 6 (13.3%)
Investigations 6 (13.3%) 3 (6.7%)
Musculoskeletal and Connective Tissue Disorders 2 (4.4%) 3 (6.7%)
Renal and Urinary Disorders  2 (4.4%) 1 (2.2%)
Reproductive Systems and Breast Disorders 1 (2.2%) 0 (0.0%)
Respiratory, Thoracic and Mediastinal Disorders 1 (2.2%) 3 (6.7%)
Skin and Subcutaneous Tissue Disorders 2 (4.4%) 1 (2.2%)
Vascular Disorders 2 (4.4%) 0 (0.0%)

The number of patients reporting adverse events in the system organ classes of psychiatric, nervous system, gastrointestinal and other general disorders are listed below:

System Organ Class Adverse Event Term SD-809
n = 45
Placebo
n = 45
PSYCHIATRIC Insomnia 3 (6.7%) 2 (4.4%)
DISORDERS Depression/Agitated Depression 2 (4.4%) 3 (6.7%)
Abnormal Dreams 1 (2.2%) 1 (2.2%)
Agitation 1 (2.2%) 0 (0.0%)
Anxiety 1 (2.2%) 1 (2.2%)
Suicidal Ideation 1 (2.2%) 0 (0.0%)
Compulsions 0 (0.0%) 1 (2.2%)
Impulsive Behavior 0 (0.0%) 1 (2.2%)
Sleep Disorder 0 (0.0%) 3 (6.7%)
NERVOUS Somnolence 5 (11.1%) 2 (4.4%)
SYSTEM Dizziness 2 (4.4%) 4 (8.9%)
DISORDERS Akathisia/Restlessness 1 (2.2%) 1 (2.2%)
Cognitive Disorder 1 (2.2%) 0 (0.0%)
Drooling 1 (2.2%) 0 (0.0%)
Dyskinesia 1 (2.2%) 0 (0.0%)
Migraine 1 (2.2%) 0 (0.0%)
Headache 0 (0.0%) 3 (6.7%)
Loss of Consciousness 0 (0.0%) 1 (2.2%)
Syncope 0 (0.0%) 1 (2.2%)
GENERAL Irritability 3 (6.7%) 6 (13.3%)
DISORDERS Fatigue 3 (6.7%) 2 (4.4%)
Gait disturbance 1 (2.2%) 0 (0.0%)
Chest pain 1 (2.2%) 0 (0.0%)
Hangover 1 (2.2%) 0 (0.0%)
GASTRO- Diarrhea 4 (8.9%) 0 (0.0%)
INTESTINAL Dry mouth 4 (8.9%) 3 (6.7%)
DISORDERS Constipation 2 (4.4%) 1 (2.2%)
Nausea 1 (2.2%) 2 (4.4%)
Abdominal pain upper 1 (2.2%) 0 (0.0%)
Dyspepsia 1 (2.2%) 0 (0.0%)
Frequent bowel movements 1 (2.2%) 0 (0.0%)
Gastrointestinal pain 1 (2.2%) 0 (0.0%)
Vomiting 0 (0.0%) 3 (6.7%)
Dysphagia 0 (0.0%) 1 (2.2%)
Flatulence 0 (0.0%) 1 (2.2%)
Salivary hypersecretion 0 (0.0%) 1 (2.2%)

There was one patient with two serious adverse events (cholecystitis and agitated depression) in the SD-809 group, and one patient with one serious adverse event (exacerbation of COPD) in the placebo group. The same patient experiencing the serious adverse events in the SD-809 group also reported suicidal ideation, which was not considered a serious adverse event.

“The data from both the First-HD and ARC-HD studies suggest clear efficacy and an excellent safety and tolerability profile for SD-809,” said Joseph Jankovic, MD, professor of neurology, Distinguished Chair in Movement Disorders and director, Parkinson’s Disease Center and Movement Disorders Clinic, Baylor College of Medicine and a treating investigator in the studies. “The results are very exciting and represent terrific news for patients living with Huntington’s disease and for the physicians who treat them. Any drug that suppresses chorea associated with Huntington’s disease, with such a low rate of somnolence and depression, as suggested by these findings, would be a welcome addition to the treatment options available for my patients. I am especially grateful to my patients who volunteered to participate in these studies.”

More than 90 percent of patients who completed First-HD enrolled into Auspex’s long-term safety study of SD-809. Complete results of the First-HD study will be reported in 2015.

ARC-HD Switch Study

In parallel to First-HD, Auspex completed the four-week Switch portion of the ARC-HD study, which has an ongoing long-term safety component. Although designed primarily as a safety study, maintenance of chorea control was assessed after switching patients overnight from tetrabenazine to SD-809 (at approximately half the dose of tetrabenazine).

All available data through eight weeks following the switch were included in the analysis. After switching from tetrabenazine to SD-809, chorea was assessed at one and four weeks. Dose adjustments were permitted after week one. The mean total chorea score decreased by approximately one point from baseline on the TMC score.

  • Week one change for 36 patients was -0.8 ± 0.4 (mean ± standard error)
  • Week four change for 35 patients was -0.8 ± 0.5

In addition, there were 21 patients for whom data were available at week eight; these data demonstrated an improvement of 1.9 (± 0.8) points on the TMC score. Data for the remaining 15 patients will be available at a future date.

The safety and tolerability experience observed in ARC-HD Switch over the four-week period was consistent with the experience observed in First-HD. The most commonly reported adverse events in ARC-HD Switch patients were somnolence, fall, and nasopharyngitis.

“The strong efficacy and safety results seen in both the First-HD and ARC-HD Switch studies confirm our belief in SD-809 as a highly promising new medicine for the treatment of chorea associated with Huntington’s disease,” said Pratik Shah, Ph.D., president and chief executive officer at Auspex Pharmaceuticals. “Patients with chorea associated with Huntington’s disease have been waiting for too long for a new therapeutic option. We are committed to rapidly advancing SD-809 along its regulatory pathway and submitting an NDA by mid-2015. In addition, we remain fully committed to exploring the therapeutic promise of SD-809 in other movement disorders, including tardive dyskinesia and Tourette syndrome.”

Upcoming SD-809 Clinical Data and Other Milestones

SD-809 continues to be evaluated in other clinical studies. Auspex anticipates reporting the following data from various studies in 2015:

  • Thorough QT study in the first quarter of 2015;
  • ARM-TD pivotal study for the treatment of tardive dyskinesia in mid-2015, and;
  • Phase 1b study in Tourette syndrome in mid-2015.

Based on the data reported today, Auspex expects to file a New Drug Application (NDA) for SD-809 for the treatment of chorea associated with Huntington’s disease by mid-2015.

Auspex also expects to report Phase 1 data for SD-560, a deuterated form of pirfenidone, by mid-2015.

Conference Call Today

Auspex will host a conference call and live audio webcast with slides today at 4:30 PM ET, 1:30 PM PT. To participate in the conference call, please dial 1-844-834-1429 (domestic) or 1-484-653-6711 (international) and refer to conference ID 54690193. A live webcast and slides can be accessed under “Events & Presentations” in the Investor Relations section of the company’s website at www.auspexpharma.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

About the First-HD Trial

First-HD is a randomized, double-blind, placebo-controlled, parallel-group trial of SD-809 in 90 patients with chorea associated with Huntington’s disease. The 12-week trial was designed to evaluate and generate label information for the safety, tolerability and efficacy of SD-809 for treating chorea associated with Huntington’s disease. The primary endpoint was a change in Total Maximum Chorea (TMC), a standardized score from baseline to maintenance therapy defined as the average of values from Week 9 and Week 12 visits. Patients were treated with SD-809 or placebo starting at 6 mg once per day to up to 24 mg twice per day (48 mg total maximum daily dose). This study was conducted at centers in the United States and Canada, in collaboration with the Huntington Study Group.

About the ARC-HD Switch Trial

ARC-HD Switch is an open-label clinical trial in 37 patients with chorea associated with Huntington’s disease in which safety, tolerability and chorea control are assessed in patients switching from stable doses of tetrabenazine to SD-809, which is administered in lower doses and with a simplified dosing regimen. At the time of this analysis, data were available from 36 patients at week one, 35 patients at week four and 21 at week eight. The study was designed to provide guidance to physicians on how to switch patients’ treatment from tetrabenazine to SD-809. The analysis was not designed to compare the safety and tolerability of SD-809 to tetrabenazine. Participating patients are eligible to receive open-label SD-809 treatment in a one-year long-term safety study. The study is being conducted at centers in the United States, Canada and Australia.

About Huntington’s Disease

Huntington’s disease is a genetic disorder that causes certain cells in the brain to die over time. This causes a wide variety of symptoms including involuntary movements, problems with emotion and behavior, and problems with thinking and processing information. Depression and suicide is common within this patient population, with a lifetime prevalence of depression of around 40 percent; more than 25 percent of patients attempt suicide at some point in their lives. More than 30,000 people in the US and Canada are symptomatic for Huntington’s disease and many more are genetically at risk of developing it.

The most visually prominent symptom of Huntington’s disease is chorea, which can appear as involuntary, excess movements that can impair a patient’s quality of life and be incredibly debilitating. At first, the movements may be subtle and be mistaken as twitching, but may develop into more pronounced movements over time. Approximately 90 percent of people with Huntington’s disease will experience chorea at some point. Currently, there is only one FDA approved treatment for a symptom of Huntington’s disease, giving physicians limited approved options to care for these patients.

About the Huntington Study Group (HSG)

HSG is an independent not-for-profit network of more than 400 clinical investigators, coordinators and scientists from 100 participating universities and clinics in the United States, Canada, Europe, Australia, New Zealand and South America who provide comprehensive care for Huntington disease (HD) patients and families and carry out multi-center clinical research including observational studies and controlled clinical trials. The mission of the HSG is to seek treatments that make a difference to those affected by HD.

About Auspex Pharmaceuticals

Auspex Pharmaceuticals is a biopharmaceutical company dedicated to developing innovative medicines for hyperkinetic movement disorders and other rare diseases. Auspex employs its proprietary technology to create patent-protected, new chemical entities from known, clinically proven pharmacologics. The company’s lead product SD-809 is in final stages of development for the treatment of chorea associated with Huntington’s disease, a neurodegenerative movement disorder that impacts cognition, behavior and movements. In addition, Auspex is investing in the broad potential of SD-809 for the treatment of other movement disorders, including tardive dyskinesia and tics associated with Tourette syndrome. The company’s pipeline also includes SD-560, being developed for fibrotic conditions. For further information, please visit the company’s website www.auspexpharma.com.

Forward-Looking Statements

Statements made in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding Auspex’s ability to successfully complete its ongoing clinical trials and development programs, Auspex’s ability to obtain regulatory approval for its product candidates and market penetration and acceptance of its product candidates. Risks that contribute to the uncertain nature of the forward-looking statements include: Auspex’s future preclinical studies and clinical trials may not be successful; changes in regulatory requirements in the United States and foreign countries may prevent or significantly delay regulatory approval of Auspex’s product candidates; Auspex may change its plans to develop and commercialize its product candidates; the U.S. Food and Drug Administration (FDA) may not agree with Auspex’s interpretation of the data from clinical trials of its product candidates; Auspex may decide, or the FDA may require Auspex, to conduct additional clinical trials or to modify Auspex’s ongoing clinical trials; Auspex may experience delays in the commencement, enrollment, completion or analysis of clinical testing for its product candidates, or significant issues regarding the adequacy of its clinical trial designs or the execution of its clinical trials, which could result in increased costs and delays, or limit Auspex’s ability to obtain regulatory approval; the third parties with whom Auspex has partnered with for the development of its product candidates and upon whom Auspex relies to conduct its clinical trials and manufacture its product candidates may not perform as expected; Auspex’s product candidates may not receive regulatory approval or be successfully commercialized; unexpected adverse side effects or inadequate therapeutic efficacy of Auspex’s product candidates could delay or prevent regulatory approval or commercialization; Auspex may be unable to obtain and maintain intellectual property protection for its product candidates; the loss of key scientific or management personnel; Auspex’s ability to obtain additional financing; and the accuracy of Auspex’s estimates regarding expenses, future revenues and capital requirements. All forward-looking statements contained in this press release speak only as of the date on which they were made. Other risks and uncertainties affecting Auspex are described more fully in Auspex’s filings with the Securities and Exchange Commission. Auspex undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

CONTACT: Corporate Communications Contacts:

         

         For Media:

         Dan Budwick, Pure Communications, Inc.

         dan@purecommunicationsinc.com

         (973) 271-6085

         

         For Investors:

         Monique Allaire Lyons, Pure Communications, Inc.

         monique@purecommunicationsinc.com

         (617) 895-9511

Original article appeared www.auspexpharma.com, December, 16, 2014: http://investor.auspexpharma.com/releasedetail.cfm?ReleaseID=887958

HSG Press Release: October, 2014

Announcement of CREST-E Early Study Closure

October 2014

The National Center for Complementary and Alternative Medicine (NCCAM), part of the National Institutes of Health (NIH), has discontinued its study of creatine for treating the progressive functional decline that occurs in Huntington’s Disease (HD). The study, known as the Creatine Safety, Tolerability and Efficacy study (CREST-E), was being conducted by the Huntington Study Group (HSG) under the leadership of Dr. Steven Hersch of Massachusetts General Hospital and Dr. Giovanni Schifitto of the University of Rochester School of Medicine.. The CREST-E Data and Safety Monitoring Board (DSMB) recently reviewed the existing study data from a planned interim analysis. The DSMB reported that there were no safety concerns. However the analysis also showed with high confidence that it was unlikely that the study would be able to show that creatine was effective in slowing loss of function in early symptomatic Huntington’s Disease.  Site investigators and coordinators have informed participants of the study closure and have encouraged each participant to schedule a final visit to their research center.

The CREST-E study enrolled 551 research participants with early Huntington’s Disease from 46 sites throughout the United States, Canada, Australia, and New Zealand. Participants were randomized to receive up to 40 grams per day of active creatine or matching placebo for a treatment period up to four years in duration. The Principal Investigators and the Huntington Study Group are committed to conducting a detailed analysis of the complete data set from the CREST-E study and to disseminating the results through the scientific review process. Even though creatine was not found to slow the progression of HD, the study amassed a large amount of invaluable longitudinal clinical and biological data, which will provide considerable information to facilitate future clinical studies.  It has also produced a unique safety database for creatine and has demonstrated the feasibility of conducting such a large controlled clinical trial in Huntington’s Disease. The research team is indebted to the participants for their time and dedication to this study.

Any questions or comments may be directed to the Huntington Study Group – 800-487-7671 (Toll Free – North America).

HSG Press Release: August 8, 2014

Announcement of 2CARE Early Study Closure

August 2014

 The National Institute of Neurological Disorders and Stroke (NINDS) stopped its study of coenzyme Q10 for the treatment of Huntington’s disease (HD) on July 14, 2014. The study (2CARE), conducted by the Huntington Study Group (HSG), was stopped for futility. The NINDS and the HSG acted on the recommendation of the study’s independent Data and Safety Monitoring Board (DSMB). Following the most recent DSMB review of the study data, an interim analysis was conducted that showed that, given the current data, it would be very unlikely (less than a 5 in 100 chance) to see a statistically significant benefit of active treatment (coenzyme Q10, 2400 mg/day) over placebo at the scheduled end of the trial. The DSMB also noted a higher number of deaths in the coenzyme Q10 group (7%) in comparison to the placebo group (4%); most of these deaths appeared to be related to HD, which is a severe, progressive neurological disorder. Although this number may have been due to chance, and was not statistically significant, the DSMB noted it in their decision. Site investigators and coordinators have informed participants of the study closure and have encouraged each participant to schedule a final visit to the clinic.

The 2CARE study enrolled 609 research participants with early Huntington’s disease from 48 sites throughout North America and Australia. Participants were randomized to receive either 2400 mg per day of active coenzyme Q10 or matching placebo. The Principal Investigators and the Huntington Study Group are committed to conducting a detailed analysis of the complete data set from the 2CARE study and to disseminating the results through the scientific review process. Even though coenzyme Q10 was not found to be helpful, the study amassed a large amount of longitudinal clinical data which will provide useful information on HD for clinical studies. The study also demonstrated the feasibility of conducting such a large controlled clinical trial in Huntington’s disease. The research team is indebted to the participants for their time and dedication to this study.

S:\RSRB\Watermarking\August 11, 2014\RSRB 13052.Public release early study closure 8 August 2014 FINAL.doc

HSG Press Release: September 2012

2CARE (Coenzyme Q10 in Huntington’s Disease) Study Update

September, 2012

Enrollment for the 2CARE study was closed on 25 July 2012, at which time the 609th participant joined the study. The study team has met the challenge of completing enrollment for the largest and longest clinical trial in Huntington disease (HD) to date. Forty-eight sites in North America and Australia contributed to the enrollment of these participants. The study will continue until the last participant completes the study, projected in mid-2017.

The 2CARE study involves collaboration of individuals with HD, caregivers or ‘study buddies’, along with the investigators, coordinators and research staff at the participating sites.

We have come a long way since the first enrollment in April of 2008, and none of our success would have been possible without the commitment and participation of those enrolled in 2CARE. We realize that not everyone can find time to be involved in clinical trials, but thanks to the willingness of those who can, we will be able to learn whether a high dosage of coenzyme Q10 (CoQ) is effective in treating HD.

A very important requirement of being able to learn whether CoQ is effective in slowing the progression of functional decline is that we have enough people remaining in the study through the entire five years. We encourage all enrolled participants to stay committed to the study as much as possible. Remember, HD is a slowly progressive disease, so it will take time to know if there are effects of the study drug.

Beginning in the spring of 2013, we will begin to see some of the earlier enrolled participants complete their five years of participation. We deeply thank all the patients and families who are part of the study, as well as those who have made it possible in so many ways– the National Institute of Neurological Disorders and Stroke, patient advocacy organizations, site investigators, coordinators, and members of the project team.

2CARE Principal Investigators and Study Team of the Huntington Study Group

HSG Press Release: May 14, 2009

FOR IMMEDIATE RELEASE

Huntington Study Group to Conduct Therapeutic Research Study in Pre-manifest Huntington’s Disease

May 14, 2009 – Physicians at 10 clinical sites are taking part in a nationwide study investigating coenzyme Q10 in gene positive, premanifest Huntington’s disease. This study is known as PREQUEL (Study in PRE-manifest Huntington’s disease of coenzyme Q10 (UbiquinonE) Leading to preventive trials).

Coenzyme Q10 is a nutritional supplement. The objective of the PREQUEL study is to evaluate the safety and tolerability of coenzyme Q10. A secondary objective is to assess the change from baseline to 20 weeks on biomarkers and DNA repair mechanisms in pre-manifest participants treated with coenzyme Q10.

Research participants who are 18 years of age and older and meet all eligibility criteria will be randomly assigned (like the flip of a coin) to receive 600, 1200 or 2400 mg/day of coenzyme Q10 capsules in a blinded fashion. Participants will be seen over 20 weeks. All participants will initiate treatment at 600 mg/day of coenzyme Q10 and then titrate to their assigned dosage over 4 weeks. Following the 4 week titration all participants will be maintained on their assigned study dosage for an additional 16 weeks of follow-up. Researchers at 10 clinical sites in the United States will enroll a total of 90 research participants who have tested positive for the Huntington’s disease (HD) gene expansion and not have features on a physical exam that would suggest a diagnosis of Huntington’s disease (pre-manifest). Each center will enroll approximately 9 participants.

Huntington’s disease (HD) is an inherited disease of the brain that usually begins between the ages of 30 to 50, and includes motor, cognitive and behavioral signs and symptoms. While there are medications to help relieve some of the disease symptoms, there is no known treatment to slow the progression of HD, which affects about 30,000 people in North America.

The HSG is a worldwide, not-for-profit group of physicians and other clinical researchers who are experienced in the care of Huntington’s disease patients and are dedicated to clinical research of Huntington’s disease. The study is sponsored by the National Institute of Neurological Disorders and Stroke (NINDS) and will be conducted under the direction of Principal Investigator Christopher Ross, MD PhD (Johns Hopkins University) and Co-Principal Investigator Kevin Biglan, MD MPH (University of Rochester) and Michael McDermott, PhD (University of Rochester).

Drs. Christopher Ross and Kevin Biglan state, “This will be the first therapeutic research study in pre-manifest HD and will hopefully lead to larger trials designed to delay the onset of HD.”

There is no cost to participate in the study. Participants will be followed for 20 weeks with participation being evaluated monthly. Persons who have tested positive for Huntington’s disease who are interested in participating in this study should visit the Huntington Study Group website at: www.HuntingtonStudyGroup.org.

press release final version 2.25.2009

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