Press Releases

Dr. Nicolò Zarotti Named Recipient of the Peter Como Scholarship

The Huntington Study Group (HSG) is pleased to announce the that Dr. Nicolò Zarotti has been named recipient of the Peter Como Scholarship to attend the renowned annual meeting.

Dr. Zarotti obtained his Bachelor’s and Master’s degree in Psychology in Italy, and later joined Lancaster University (UK), where he completed his PhD focused on emotional processing and communication in Huntington disease (HD). Dr. Zarotti is currently part of a team collaborating with the British Psychological Society to produce new guidance on psychological approaches to the understanding and treatment of psychological difficulties in people with motor neurodegenerative conditions such as HD.

The Peter Como Scholarship, named in memory of one of HSG’s founders and biggest supporters, is awarded annually for a professional in the mental health field to attend HSG 2018: Unlocking HD. The Como Scholarship includes two nights’ lodging, travel funding, and a modest stipend.

The Huntington Study Group was formed in 1993 and is the world’s first HD cooperative therapeutic research organization. Twenty-five years later, HSG is a world leader in facilitating high quality clinical research trials and studies that work toward finding effective treatments for HD. HSG has the first and largest HD clinical research network of more than 400 active and compassionate investigators, coordinators, scientists and HD experts at more than 120 credentialed research sites around the globe. HSG members are world experts in caring for individuals and families impacted by Huntington disease. Visit https://huntingtonstudygroup.org/ for more information.

HSG Scholarship Winners Receive Funding to Attend Premier Huntington’s Disease Conference

The Huntington Study Group (HSG) announces the 2018 CME4HD Online scholarship winners who have been awarded funding to attend its renowned annual meeting, HSG 2018: Unlocking HD.

Maryluz Camargo Mendoza, Ph.D

Addie Patterson, D.O

Maryluz Camargo Mendoza, Ph.D, associate professor at the National University of Colombia, Bogota, has received a scholarship for referring 49 people who successfully completed five CME4HD Online modules. Addie Patterson, D.O., associate professor at the University of Gainesville won the random drawing of 133 participants who completed five modules successfully. Both scholarship winners receive airfare, hotel and a travel stipend to attend the conference, held at ZaZa Hotel in Houston in November.

CME4HD Online is a continuing medical education program designed to teach healthcare providers how to care for and manage individuals with Huntington disease (HD). Participants gain the knowledge, tools,and resources needed to provide quality care to families impacted by HD, while learning from worldwide HD experts who teach the course. Topics and discussion include symptoms and diagnosis, HD treatments, ethical dilemmas in genetic testing, strategies for disease management, translation of complex clinical information for families and the role of ancillary services in the management of HD.

The CME4HD referral program encourages sharing and completion of the program modules to help educate medical professionals about Huntington’s Disease in order to help patients receive better care between sub-specialist appointments.

CME4HD Online program was first shared at HSG’s annual event and is now available online free of charge. The program was planned and implemented by HSG in partnership with the North American Center for Continuing Education, LLC (NACCME). This program was made possible, in part, by an independent educational grant from TEVA Pharmaceuticals.

The Huntington Study Group was formed in 1993 and is the world’s first HD cooperative therapeutic research organization. Twenty five years later, HSG is a world leader in facilitating high quality clinical research trials and studies that work toward finding effective treatments for HD. HSG has the first and largest HD clinical research network of more than 400 active and compassionate investigators, coordinators, scientists and HD experts at more than 120 credentialed research sites around the globe. HSG members are world experts in caring for individuals and families impacted by Huntington disease. Visit https://huntingtonstudygroup.org/ for more information.

Huntington Study Group Welcomes New Leadership May 1

The Huntington Study Group (HSG) is pleased to announce that Andrew Feigin, MD, and Elise Kayson, MS, ANP, have been elected as the chair and co-chair, respectively, of HSG, a world-wide network of Huntington disease (HD) researchers. Both Feigin and Kayson have dedicated their careers to the clinical care of patients and families and research in HD.

Feigin and Kayson will begin their four-year term as chair and co-chair May 1, 2018, succeeding Ray Dorsey and Blair Leavitt, who along with Julie Stout, Joni Steinman, and Shari Kinel, expertly led HSG through the last four years. They were democratically elected by HSG’s world-wide membership of more than 500 investigators, coordinators, and other researchers and care providers.

Feigin, a Professor of Neurology at NYU Langone Health and co-director of the Marlene and Paolo Fresco Institute for Parkinson’s and Movement Disorders, has been involved in the care of HD patients and research since his participation in the Venezuela Collaborative Research Group, which isolated the HD gene 25 years ago. Kayson, Director of Clinical and Strategic Initiatives at the University of Rochester’s Center for Health + Technology (CHeT), has been involved in the care of HD patients and research since the inception of the HSG and was one of the founders of the organization.

Feigin’s independent research has focused on the development of novel imaging biomarkers that could be used as outcome measures for HD clinical trials. He has served in many HSG leadership positions over the past 20 years, including as a member of the Executive Committee, chair of the Program Committee for the HD Clinical Research Symposium for five years, and currently chairs the Clinical Research Advisory Committee. Feigin is the principal investigator (PI) of the SIGNAL trial and the co-PI of LEGATO-HD, and has served as a site PI on numerous other HD trials. “I am honored and excited to serve as the Chair of this amazing organization as the most exciting new potential therapies for HD are now entering human trials,” Feigin said.

Prior to leading CHeT’s Clinical and Strategic Initiatives, Kayson was the Director of Project Management for the Clinical Trials Coordination Center (CTCC) at the University of Rochester and previously worked in industry. In addition, Kayson’s long involvement in all aspects of more than 50 clinical trials and the FDA approval of the only two drugs for HD gives her a deep understanding of clinical trial design, organization and conduct, and insights into and appreciation of HD clinical trials from the perspective of study participants to coordinators, investigators, CRO functions, and sponsors. She has served in many leadership positions in HSG, including as a member of the Executive Committee, co-chair of the HSG Credentials Committee, and co-chair of the HSG Educational Committee. “It is exciting to be part of the momentum of research in HD. I am honored to serve as the HSG Co-Chair and look forward to reaching the goal of finding treatments that make a difference for our patients and families,” said Kayson.

HSG appreciates all the candidates who ran for these positions and salutes their enthusiasm and commitment to the entire election process. In addition, we thank each of our members who attended the webinars, viewed the webpages, and took the time to cast votes for this important election.

As HSG begins our 25th year of seeking treatments that make a difference, let’s join in congratulating our new leadership and wishing them the very best as they pursue their vision of partnerships, innovation and education to bring an exciting future to HSG.

Welcome new staff member, members of leadership team

The Huntington Study Group is pleased to announce a new staff member and new members of the leadership team. Anne Van Dusen, CHC, is HSG’s new Director of Education & Special Programs, Sarah Noonberg, MD, PhD, replaces Mike Poole, MD, on the executive committee, and Christopher Beck, PhD, replaces David Oakes, PhD, as HSG’s Director of Biostatistics/executive committee member.

“We are extremely fortunate to have Sarah, Chris, and Anne join our team,” said Ray Dorsey, MD, chair of HSG. “Sarah and Chris have a long-standing interest and record of service in HD. Anne brings much-needed expertise in training to help drive our educational efforts.”

Anne Van Dusen, CHC, Director of Education & Special Programs

Anne joined the HSG team in June of 2017 as the Director of Education & Special Programs. Anne is part of the Executive team and will develop and implement both educational and program plans that will sustain and help HSG to maintain its standing as a proven world leader in facilitating high quality clinical research trials and studies in Huntington Disease (HD). Anne holds a BS from the University of Binghamton School of Management and is certified in healthcare compliance by the Compliance Certification Board. Prior to joining HSG, Anne held several leadership roles in compliance, quality and finance, most recently at CCSI of Rochester, ACM Medical Laboratory and Lifetime Care Home Care and Hospice. Anne is passionate about trail running and is very active in the local running community. 

 

 

Christopher Beck, PhD, HSG Director of Biostatistics

Chris is an Associate Professor of Biostatistics and Orthopaedics at the University of Rochester Medical Center. As a member of several national and international collaborative groups conducting research in areas including Huntington disease, Parkinson disease, Batten disease, bone repair, rheumatoid arthritis, and cardiomyopathy, Chris has extensive experience and a great deal of expertise in the biostatistical aspects of basic and clinical research. He joined HSG in 2005 while serving as a biostatistican for the TREND-HD study, and he has continued in that role for a total of eight HSG studies. Chris is honored to become only the second Director of Biostatistics in the history of HSG.

 

 

Sarah Noonberg, MD, PhD, Executive Committee Member

Sarah is currently the Chief Medical Officer at Prothena, a biotechnology company focused on the discovery, development, and commercialization of novel immunotherapies for diseases involving protein misfolding. Prior to joining Prothena, she served as Group Vice President and Head of Global Clinical Development at BioMarin Pharmaceuticals Inc., where she was responsible for clinical development programs using gene therapies, oligonucleotides, and recombinant proteins to address a diverse range of neurodegenerative, neuromuscular, metabolic, and hematologic diseases. Earlier in her career she led clinical studies in Huntington disease in partnership with the Huntington Study Group, as well as clinical studies in Parkinson’s disease, Alzheimer’s disease, prostate cancer, and cystic fibrosis. Sarah earned her MD at the University of California, San Francisco, her PhD in Bioengineering at the University of California, Berkeley, and her BS at Dartmouth College. She is a board-certified internist and works part-time as a hospitalist physician treating a broad range of inpatient and critical care patients.

Overnight Switch from Tetrabenazine to Deutetrabenazine Safe, Trial Shows

JAMA Neurology paper published today from ARC-HD Trial

People with Huntington disease-associated chorea can safely convert overnight from tetrabenazine to deutetrabenazine (brand name: Austedo), according to the results of the Alternatives for Reducing Chorea in Huntington Disease (ARC-HD) trial published yesterday in JAMA Neurology. The Phase III open-label, single-arm switch cohort of the trial was led by the Huntington Study Group (HSG) and the University of Rochester’s Clinical Trials Coordination Center (CTCC) on behalf of Teva Pharmaceutical Industries Ltd.

Although the topline results of the trial have been released previously, the peer-reviewed publication about the switch arm of ARC-HD clinical trial is now published in a premier neurological medical journal.

 

“This trial provides more good news for our patients who need options for medication to control their chorea,” 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. “We are grateful to the people who volunteered in this trial and their families who supported their participation.”

The ARC-HD trial enrolled 37 patients who were on a stable dose of tetrabenazine for 8 weeks or longer. Participants converted from tetrabenazine to deutetrabenazine at a dose that was half of their original total daily tetrabenazine dose. After one week, investigators began weekly dose adjustments, if needed, to achieve optimal chorea control. This study was focused on safety. In addition, Total Maximal Chorea Score and Total Motor Score were evaluated as efficacy endpoints.

In April, the U.S. Food and Drug Administration (FDA) approved deutetrabenazine, the second drug approved for use in the United States to treat chorea in HD. The approval was based on positive results from the First-HD study, a Phase 3 clinical trial which was also led by HSG and CTCC on behalf of Teva Pharmaceuticals. In the double-blind, placebo controlled trial, deutetrabenazine significantly decreased chorea. The results were published in JAMA, July 2016.

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.

“The deuterium chemistry can provide effective chorea control with fewer daily doses and with lower peak doses, potentially improving medication tolerance. These are both big benefits for our patients,” said Claudia Testa, MD, PhD, HSG’s co-principal investigator for ARC-HD and director of the HDSA Center of Excellence at Virginia Commonwealth University.

“It’s gratifying to see the current progress in treatments for people with Huntington disease. In addition to being grateful to the research participants who are a major driver in that progress, we are grateful to the sites and site staff whose dedication to seeking treatments that make a difference is unparalleled,” Testa added.

Much of the work that led to the completion of the ARC-HD trial was carried out by 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 ARC-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 www.ctcc.rochester.edu.

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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:

Huntington Study Group — +1 800-487-7671 or info@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:
+1 800-487-7671 or info@hsglimited.org

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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 contact HSG at +1 800-487-7671 or info@hsglimited.org.

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