Press Releases

FDA Approves Second Drug for Huntington Disease Symptom

Frank

The U.S. Food and Drug Administration (FDA) today approved SD-809 (deutetrabenazine), the second drug approved for use in the United States to treat chorea in Huntington disease (HD), a rare, inherited neurodegenerative disorder.

The approval was based on positive results from the First-HD study, a Phase 3 clinical trial which was led by the Huntington Study Group (HSG) on behalf of Teva Pharmaceuticals. In the double-blind, placebo controlled trial, deutetrabenazine significantly decreased chorea, the involuntary movements that many individuals with HD experience. The results were published in JAMA, July 2016.

“We are so grateful to the patients and families who made this development possible by participating in this ground-breaking trial. Trial participants are the key to bringing new treatments to the entire HD community,” said Samuel Frank, MD, Huntington Study Group’s principal investigator for First-HD and associate professor of Neurology at Beth Israel Deaconess Medical Center/Harvard Medical School. Claudia Testa, MD, PhD, associate professor of Neurology at Virginia Commonwealth University served as the co-principal investigator.

Testa

“It’s exciting to offer a new treatment,” Testa said. “Trials like these give patients, families, and care providers more options to effectively manage HD symptoms and improve quality of life.”

Most individuals with HD experience chorea during the course of the disease. Huntington disease is an autosomal-dominant, inherited disease that usually manifests in people in their 30s and 40s, though some people are affected as early as childhood and others experience disease symptoms much later in life. The disease brings with it an array of symptoms besides chorea, including dystonia, cognitive problems, changes in personality, and psychiatric problems like depression. Because HD is autosomal dominant, each child of a person with HD has a 50 percent chance of inheriting the genetic change that causes the disease from their affected parent, whether that parent is their mother or father. For more information about HD, visit www.huntingtonstudygroup.org.

Deutetrabenazine is structurally related to tetrabenazine with deuterium atoms placed at key positions in the molecule, prolonging plasma half-life and reducing metabolic variability, without changing target pharmacology. Deutetrabenazine is the first FDA approved compound with deuterium substitution. Much of the clinical work that led to the approval of deutetrabenazine was carried out by the Huntington Study Group, a non-profit network of 400 Huntington disease experts from more than 100 medical centers throughout North America, Europe, and Australia who are dedicated to seeking treatments that make a difference for people and families affected by the disease. For more information, visit www.huntingtonstudygroup.org.

“This is a great day for the HD community,” said Ray Dorsey, MD, chair of the Huntington Study Group and director of the University of Rochester’s Center for Human Experimental Therapeutics (CHET). “The unmet need for therapeutics for individuals with HD is immense, and this approval brings us closer to making HD an increasingly treatable condition.”

First-HD was conducted at 34 Huntington Study Group sites across the United States and Canada, enrolling 90 participants over 14 months, in the 13-week double-blind, placebo-controlled trial. Scientific, technical, logistical, and analytical support for the study was provided by the University of Rochester Clinical Trials Coordination Center (CTCC). The Clinical Trials Coordination Center is part of the Center for Human Experimental Therapeutics (CHET) and is a unique academic-based organization with decades of experience working with industry, foundations, and governmental researchers in bringing new therapies to market for neurological disorders.  For more information about the Clinical Trials Coordination Center, visit www.ctcc.rochester.edu.

Teva Pharmaceuticals owns the rights to develop and sell deutetrabenazine in the United States, following its purchase of Auspex Pharmaceuticals in 2015. Deutetrabenazine is an investigational, oral, small-molecule inhibitor of vesicular monoamine 2 transporter, or VMAT2, that was granted Orphan Drug Designation for the treatment of HD by the FDA.

A second deutetrabenazine trial, ARC-HD, which has completed enrollment, is investigating the safety, efficacy, and tolerability of the drug when individuals with HD switch from tetrabenazine to deutetrabenzine and the safety of longer term exposure. This open-label trial is also being led by the Huntington Study Group and the Clinical Trials Coordination Center for Teva Pharmaceuticals. Teva is also investigating the potential of deutetrabenazine to treat tardive dyskinesia, a disorder that causes involuntary and repetitive movements, and for tics associated with Tourette syndrome.

Media inquiries:

Heather Hare

Huntington Study Group

(585) 242-0277

Heather.Hare@hsglimited.org

World Experts in HD to Convene in Nashville

HSG 2016 with dateTraining opportunities, family education available at HSG 2016

The Huntington Study Group (HSG), an international, non-profit network of more than 400 researchers and clinicians dedicated to seeking treatments that make a difference for Huntington disease (HD), is hosting its annual forum, HSG 2016: Discovering Our Future, Nov. 2-5 in Nashville for training and education and for presentation of new research findings and treatments to the worldwide community. The event, which is expected to draw over 300 attendees, is being held at the Gaylord Opryland Resort and Conference Center.

Among the highlights of the event are:

  • UHDRS Symposium, which focuses on a proposed modified Unified Huntington Disease Rating Scale and its use in clinical trials as an efficacy endpoint.
  • HD Innovators Forum, which includes presentations from leading HD industry partners working on developing new HD treatments.
  • CME4HD, a day-long, in-person continuing medical education course for healthcare providers hosted by HSG and Vanderbilt University School of Medicine. The course will teach providers how to care for and manage individuals with HD. Participants will have the opportunity to earn 8.75 AMA PRA Category 1 CME CreditsTM.
  • Keynote speech by Dr. Bob Beall, former CEO of the Cystic Fibrosis Foundation, who will talk about how CFF developed its care and research models in a non-profit setting through partnering with families, care providers, researchers, and biotech.
  • HD Research Symposium, highly acclaimed research presentations drawing worldwide recognition.
  • HD Family Education Day, designed for the local HD community and family members. The day starts with hearing about the latest in HD research, followed by interactive workshops and breakout sessions.

Registration costs vary on participation, but HD Family Education Day is free for HD community members. Please visit www.huntingtonstudygroup.org for more information and to register.

For more information:
Heather Hare
(585) 242-0277

# # #

Vaccinex Receives FDA Fast Track Designation for VX15 Antibody for the Treatment of Huntington’s Disease

vaccinex_logoVaccinex, Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for VX15 as a potential treatment for Huntington’s disease (HD).  VX15 is the Company’s novel clinical stage monoclonal antibody that blocks the activity of semaphorin 4D (SEMA4D), a molecule that is believed to promote chronic inflammatory responses in the brain.

Read the company’s press release.

Chorea Reduced by Deutetrabenazine in Study led by HSG


JAMA publishes First-HD study

People with Huntington disease experienced improvements in chorea while taking deutetrabenazine (SD-809) compared to placebo, according to a paper published today in the Journal of the American Medical Association (JAMA). Although the topline results of the trial have been released previously, the complete peer-reviewed publication about the First-HD clinical trial is now published in a premier medical journal.FirstHD_Horizontal sm screen res

Deutetrabenazine was investigated in the First-HD study, a Phase 3 clinical trial which was led by the Huntington Study Groupteva_cns_logo (HSG) on behalf of Teva Pharmaceuticals. In the double-blind, placebo-controlled trial, deutetrabenazine significantly decreased chorea, the involuntary movements that many individuals with HD experience.

“Patients’ chorea and motor scores improved compared to placebo over the course of 12 weeks,” said Samuel Frank, MD, HSG’s principal investigator of First-HD and director of the HDSA Center of Excellence at Beth Israel Deaconess Medical Center in Boston. “In addition, both the participants and their study physicians reported overall improvement.”

First-HD enrolled 90 patients at 34 HSG research sites between August 2013 and August 2014. The trial followed patients for 12 weeks on the medication and measured their chorea, as well as patients’ and clinicians’ impression of improvement.

Sam Frank resized

Frank

“As a physician who cares for people with HD, it’s gratifying to see positive results from a well-designed, fully enrolled trial. Until we find a cure, we aim to bring our patients more treatment options to relieve symptoms,” Frank said. “We are grateful to the people who participated in this trial and their families and support systems that made their participation possible. Research in the HD community depends on volunteers enrolling in trials.”

At the end of May, Teva Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) asked for more data on deutetrabenazine, which had been under review to treat chorea associated with HD. The request for more data is common when the FDA is asked to approve new medications, and this is the first deuterated compound to be reviewed by the FDA. Michael Hayden, M.D., Ph.D., Teva’s president of Global R&D and chief scientific officer said Teva plans to respond to the request in the third quarter of 2016.

There is only one drug currently approved to treat chorea associated with Huntington disease: tetrabenazine. Deutetrabenazine is structurally related to tetrabenazine with deuterium atoms placed at key positions in the molecule, prolonging plasma half-life and reducing metabolic variability, without changing target pharmacology. This can translate into effective symptom control with fewer medication doses a day, lower total daily doses, and improved tolerance. In First-HD, both patient and clinician overall assessments were significantly better in the deutetrabenazine treated group compared to placebo after 3 months. The deutetrabenazine group improved in a quality of life measure while the placebo group worsened.

testa_VCU

Testa

“Overall status and quality of life measures are especially relevant in chorea, where no single number captures what is clinically meaningful to patients themselves,” said Claudia Testa, MD, PhD, HSG’s co-principal investigator for First-HD and director of the HDSA Center of Excellence at Virginia Commonwealth University. “It’s exciting to see how treating an HD symptom can make a real-life positive impact.”

Much of the work that led to the completion of the First-HD trial was carried out by the HSG, a non-profit network of 400 Huntington disease experts from more than 100 medical centers throughout North America, Europe, Australia, New Zealand, and South America who are dedicated to seeking treatments that make a difference for people and families affected by the disease. For more information about the Huntington Study Group, visit www.huntingtonstudygroup.org.

Scientific, technical, logistical, and analytical support for First-HD was provided by the University of Rochester Clinical Trials Coordination Center (CTCC). The Clinical Trials Coordination Center is part of the Center for Human Experimental Therapeutics (CHET) and is a unique academic-based organization with decades of experience working with industry, foundations, and governmental researchers in bringing new therapies to market for neurological disorders.  For more information about the Clinical Trials Coordination Center, visit http://www.ctcc.rochester.edu.

Teva Pharmaceutical acquired deutetrabenazine through its purchase of Auspex Pharmaceuticals last year. Deutetrabenazine is an investigational, oral, small-molecule inhibitor of vesicular monoamine 2 transporter, or VMAT2, that was granted Orphan Drug Designation for the treatment of HD by the FDA.

A second deutetrabenazine trial, ARC-HD, which has completed enrollment, is investigating the safety, efficacy, and tolerability of the drug when individuals with HD switch from tetrabenazine to deutetrabenzine and the safety of longer term exposure. This trial, which includes participants who completed First-HD, is also being led by the HSG and the Clinical Trials Coordination Center for Teva Pharmaceutical Industries. Teva is also investigating the potential of deutetrabenazine to treat tardive dyskinesia, a disorder that causes involuntary and repetitive movements, and for tics associated with Tourette syndrome.

FDA Requests More Data on Potential New Treatment for Huntington Disease

tevahomeTeva Pharmaceuticals Industries announced yesterday that the U.S. Food and Drug Administration (FDA) has asked for more data on SD-809 (deutetrabenazine), which is currently under review to treat Huntington disease (HD), a rare, inherited neurodegenerative disorder.

The request for more data is common when the FDA is asked to approve new medications, and this is the first deuterated compound to be reviewed by the FDA. Michael Hayden, M.D., Ph.D., Teva’s president of Global R&D and chief scientific officer said Teva plans to respond to the request in the third quarter of 2016.

Deutetrabenazine was investigated in the First-HD study, a Phase 3 clinical trial which was led by the Huntington Study Group (HSG) on behalf of Teva Pharmaceutical Industries. In the double-blind, placebo controlled trial, deutetrabenazine significantly decreased chorea, the involuntary movements that many individuals with HD experience.

“We are grateful to the patients and families who have participated in First-HD and helped us get to this point. HSG will continue its role in the clinical development of this product with TEVA,” said Samuel Frank,Sam Frank resized M.D., Huntington Study Group’s principal investigator for First-HD and a movement disorders specialist at Beth Israel Deaconess Medical Center. Huntington Study Group’s co-principal investor is Claudia Testa, M.D., Ph.D., associate professor of Neurology at Virginia Commonwealth University.

Most individuals with HD experience chorea during the long course of the disease, which averages 15-20 years. Huntington disease is an autosomal-dominant, inherited disease that usually manifests in people in their 30s and 40s, though some people are affected as early as childhood and others don’t experience the diseases symptoms until much later in life. The disease is caused by the death of brain cells known as medium spiny neurons, which are killed off by a mutant protein. The disease brings with it an array of symptoms besides chorea, including cognitive problems, changes in personality, and psychiatric problems like depression. Because HD is autosomal dominant, each child of a person with HD has a 50 percent chance of inheriting the disease. For more information about HD, visit www.huntingtonstudygroup.org.

Deutetrabenazine is structurally related to tetrabenazine with deuterium atoms placed at key positions in the molecule, prolonging plasma half-life and reducing metabolic variability, without changing target pharmacology. Much of the work that led to the completion of the First-HD trial was carried out by the Huntington Study Group, a non-profit network of 400 Huntington disease experts from more than 100 medical centers throughout North America, Europe, and Australia who are dedicated to seeking treatments that make a difference for people and families affected by the disease. For more information about the Huntington Study Group, visit www.huntingtonstudygroup.org.

“While disappointed with the delay, we remain hopeful and optimistic that the FDA will soon approve the second treatment for HD,” said Ray Dorsey, M.D., chair of the Huntington Study Group and director of the University of Rochester’s Center for Human Experimental Therapeutics (CHET).Ray Dorsey

First-HD was conducted at 34 Huntington Study Group sites across the United States and Canada, enrolling 90 participants over 14 months, in the 13-week double-blind, placebo-controlled trial. Scientific, technical, logistical, and analytical support for the study was provided by the University of Rochester Clinical Trials Coordination Center (CTCC). The Clinical Trials Coordination Center is part of the Center for Human Experimental Therapeutics (CHET) and is a unique academic-based organization with decades of experience working with industry, foundations, and governmental researchers in bringing new therapies to market for neurological disorders. For more information about the Clinical Trials Coordination Center, visit www.ctcc.rochester.edu.

Teva Pharmaceutical owns the rights to develop and sell deutetrabenazine in the United States, following its purchase of Auspex Pharmaceuticals last year. Deutetrabenazine is an investigational, oral, small-molecule inhibitor of vesicular monoamine 2 transporter, or VMAT2, that was granted Orphan Drug Designation for the treatment of HD by the FDA.

A second deutetrabenazine trial, ARC-HD, which has completed enrollment, is investigating the safety, efficacy, and tolerability of the drug when individuals with HD switch from tetrabenazine to deutetrabenzine and the safety of longer term exposure. This trial, which includes participants from First-HD, is also being led by the Huntington Study Group and the Clinical Trials Coordination Center for Teva Pharmaceutical Industries. Teva is also investigating the potential of deutetrabenazine to treat tardive dyskinesia, a disorder that causes involuntary and repetitive movements, and for tics associated with Tourette syndrome.

For media inquiries, contact Heather Hare, director of Communications & Outreach, at heather.hare@hsglimited.org.

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.

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Kevin C. Mannix, United States, 215-591-8912
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Tomer Amitai, Israel, (972 (3) 926-7656
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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

 

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