Press Releases

Huntington Study Group® and Roche Collaborate on Generation HD2 Study

To read the full press release, click here.

Huntington Study Group® Holds 30th Annual Meeting 

To read the full press release, click here.

HSG UPDATES – September 2023

September 2023
MAKING AN IMPACT

  1. CEO’s Insights
  2. New Options for Patients: FDA Approval of Valbenazine
  3. Lowering Research Participation Barriers: vUHDRS® Topline Results
  4. Facilitating HD Research Collaboration: CHDI
  5. Forging More Diversity and Representation in Research: myHDstory® Results
  6. Creating Opportunities for Learning, Engaging, and Connecting: HSG 2023 Annual Meeting
  7. Building a Stronger HSG: ENGAGE-HD + New Members + Process for New Ideas and Projects
  8. Reflections on an Impactful Life: Remembering Christine Hunter
  9. Catalysts for the Future: HSG Education and Training
  10. In the Know

1.   CEO’s Insights

In memory of Christine Hunter, someone who was devoted to making an impact on the Huntington’s disease community with her skills, talent, and heart, we are dedicating this issue of HSG Updates to the theme of impact. While we have had some big wins, we recognize these are some battles won while a war rages on. No one is giving up. In this issue we explore some of the areas in which HSG has recently driven progress, spurred potential, and helped positively impact the acceleration of treatments for HD.

– Shari Kinel, JD


2.   New Options for Patients: FDA Approval of Valbenazine 

We are thrilled with the recent approval of valbenazine for chorea in adults with HD. This approval was based off the positive, highly statistically significant results from the KINECT-HD trial and the ongoing KINECT-HD2 trial led by the HSG and NBI. While this is not the first VMAT2 inhibitor approved for chorea associated with HD, this is still a welcome and important win for those living with or at-risk for HD.

Evaluation of the ENROLL-HD database demonstrates that chorea may be undertreated. The reasons for this finding are multifactorial but this reminds us that we still have work to do in the realm of symptomatic treatment for HD. Chorea is certainly not the only symptom our patients struggle with; however, it is an important symptom that can negatively impact quality of life, functional independence and increase caregiver burden.

A positive trial not only gives our patients another symptomatic treatment option, but also provides opportunities to raise awareness and in doing so, hopefully indirectly improves care and access to HD patients and families. In addition, the availability of multiple treatment options encourages ongoing research and innovation in the field of HD.

We remain optimistic about and focused on an approved disease modifying therapy for HD hopefully sooner rather than later, but in the interim, we hope that additional effective symptomatic treatments can help our HD patients and families throughout their journey.

– Erin Furr Stimming, Study Principal Investigator of KINECT®-HD/HD-2 Studies


3.   Lowering Research Participation Barriers: vUHDRS® Topline Results

The Huntington Study Group (HSG) has revolutionized research methodologies by introducing innovative approaches. Our participant-centric strategies, coupled with advancements in technology, aim to deliver clinical trials to participants, eliminating the need for participants to travel to trial sites. This approach mitigates recruitment and participation barriers, including unforeseen circumstances, geographical constraints, evolving participant preferences, and time constraints. HSG has a longstanding commitment to addressing these challenges, consistently achieving study enrollment and retention objectives while conducting high-quality clinical trials.

In 2010, HSG’s Project AWARE was a comprehensive survey of over 600 individuals and families impacted by Huntington’s disease (HD). This observational study aimed to enhance awareness, willingness, and ability to participate in clinical trials and observational studies, contributing to the design of future trials. HSG has diligently applied the insights gained from Project AWARE to initiatives aimed at facilitating access to research centers and formulating study protocols that incorporate the input of HD patients, thereby enhancing the quality of clinical trials.

In keeping with the HSG’s philosophy of bringing innovative methods to clinical research, the HSG successfully embarked on developing a virtual version of the gold standard assessment tool, the Unified Huntington’s Disease Rating Scale (UHDRS®) developed in 1996 (HSG et al 1996). The results of the virtual Unified Huntington’s Disease Rating Scale (vUHDRS®) showed that virtual implementation of the vUHDRS® was reliable and feasible including assessment of motor, Total Functional Capacity and all other functional sections, cognitive and behavioral sections. The HSG is looking to the vUHDRS® as the next research study tool to assist with the development of hybrid clinical trials combining remote and in-person visits to significantly reduce participant burden and increase research.

The vUHDRS® results were presented at the International Congress of Parkinson’s Disease and Movement Disorders® in Copenhagen, Denmark in August and will be presented at HSG’s annual meeting in Phoenix.

– Samuel Frank, MD, vUHDRS® Study Principal Investigator
– Jody Goldstein, vUHDRS® Study Co-Principal Investigator


4.   Facilitating HD Research Collaboration: CHDI

For 30 years, HSG has been working to improve the lives of people impacted by Huntington’s disease through research, education, and collaboration. By building collaborative relationships and credibility with key HD stakeholders, we are better positioned to understand perspectives and fulfill the needs of research site personnel, sponsors, and people impacted by HD.

In July of this year, we announced that our clinical research organization (CRO), HSG Clinical Research, Inc. (HSGCR), began providing services for CHDI Foundation’s Enroll-HD observational study and clinical research platform in the US and Canada. Enroll-HD is currently the world’s largest observational study for Huntington’s disease families, providing an important resource for HD families, clinicians, researchers, advocates, pharma, and biotech companies.

The melding of the operational and clinical experience and knowledge of CHDI and HSG will enable the teams to mold the next generation of Enroll-HD by enhancing operational efficiency and quality and potentially creating new ways to recruit study participants, collect study data, and ensure data integrity.

Combining the strengths of both organizations allows us to more collaboratively accelerate treatments that make a difference and make a greater impact for the entire HD community. 

– Elise Kayson, HSG VP of Clinical Operations 

If you’re interested in collaborating with HSG, send us a note at info@hsglimited.org


5.   Forging More Diversity and Representation in Research: myHDstory® Results

One of the main goals at HSG is to improve diversity in research by reaching underrepresented populations. We know from the HSG Care Improvement Project surveys and other work that 70% of people with HD do not access specialty care. Because the US is such a large country, and specialty care and research are concentrated in big cities, many groups, particularly those in rural and less populous areas, are not able to join clinical or observational studies.  It can also be burdensome to travel to a site for research visits. Those with fewer economic advantages are therefore underrepresented in many studies.

Most clinical trials are 90% or more Caucasian, but the overall population is much more racially diverse, and HD affects all races and ethnic groups. At HSG, we have several initiatives to make research more accessible and inclusive. This includes the myHDstory® online, direct-to-participant research platform. We recognize that research cannot be representative of the entire HD population if it does not include perspectives from people of all backgrounds.  With the myHDstory® platform, people with HD can participate in an observational study from the comfort of their own home or other private location.

HD presents many challenges to patients and their families. HSG offers online participation through the myHDstory® platform to lessen this burden with respect to research participation. It is our hope that these efforts will make research more representative of the HD population as a whole and help us to understand how people from differing economic and racial/ethnic backgrounds experience HD symptoms.

More studies are currently in the works for the myHDstory® platform, including new PROs, longitudinal capabilities, and reaching even more participants. We hope to have some exciting announcements

– Karen Anderson, MD, Study Principal Investigator of ‘Making HD Voices Heard’


6.   Creating Opportunities for Learning, Engaging, and Connecting:
HSG 2023 Annual Meeting

Among the not-so-hidden benefits of the HSG meeting is the opportunity to meet, re-meet, learn from, and connect with colleagues who share a passion for care of HD families and research in HD. For a young clinician, investigator, or coordinator, this is the best opportunity you will have all year to discuss difficult cases, think about different ways of managing patients, learn about new and upcoming research studies, and share HD-related experiences. You can join a working group, volunteer for a committee, learn from a seasoned investigator how to conduct a study more efficiently, and test out your bright idea on an interested audience. Submit a poster describing your work! New in 2023 is a Saturday training program called ENGAGE-HD, specifically directed towards the HD experts of the future.   

Even if your hair has begun to turn gray, you will still benefit from hearing about the successes and challenges that your colleagues have faced, while inspiring and encouraging the younger attendees. Become an HSG leader! Give a presentation, create a new working group, lead a clinical trial!   

The relationships you develop and nurture at the HSG 2023 meeting will last throughout your professional lifetime—and beyond! 

– Martha Nance, MD, HSG Executive Membership Committee and Chair of HSG Family Education Committee

If you’re planning to attend this year’s annual meeting, here are a few useful links:


7.   Building a Stronger HSG: ENGAGE-HD + New Members + Process for New Ideas & Projects

ENGAGE-HD
In the realm of scientific progress, one fundamental truth stands out: progress requires people. The pursuit of groundbreaking research is inherently dependent on the collaborative efforts of dedicated individuals. But despite the increasing prevalence of progressive neurodegenerative diseases, the relatively stagnant numbers of medical students opting to specialize in neurology creates a concerning gap. The future of HD research and patient care is at risk. HD is a scientifically fascinating and clinically significant disease that has the potential to attract the intellectual curiosity of the next generation of medical practitioners and researchers. To galvanize this interest and nurture a fascination with HD as a career path, we need to offer HD-specific education created and curated by international experts.

The acronym ENGAGE-HD means to Educate, Nurture, Gather, Acquire knowledge, Grow, and Excite professionals, both experienced and new, about HD. The HSG ENGAGE-HD program is an initiative to attract, foster, and educate new members and researchers and to create educational programs and events exclusively centered on Huntington’s disease for those with an interest in neurology and movement disorders. Through this initiative, we hope to strengthen the impact HSG has on the world of research and treatment. 

Our overarching goal is to orchestrate a transformation: expand the pool of professionals dedicated to the pursuit of movement disorders (particularly HD), bolster the knowledge of people committed to research and treatment of HD, and add meaningfully to the body of HD-related publications. By cultivating the growth of researchers and practitioners passionate about Huntington’s disease, HSG becomes a stronger organization, better poised to enhance patient care and uncover scientific breakthroughs.

– Jee Bang, MD, MPH, HSG Executive Membership Committee and Chair, HSG Provider Education Committee

Over the past few months, HSG’s membership has continued to grow. There are several new faces as well as some people with whom HSG has been working for quite some time and are now official. We welcome all of you to our network and look forward to everything we will accomplish together. You are the future of HSG.

If you are already an HSG member, please login here to ensure all of your information is up to date.

Process for New Ideas and Projects
At HSG, we’re continually impressed by our members and the education, research, and care initiatives you develop. As our network continues to grow and thrive, we want to ensure that we have the proper support in place to help ensure efficiency, effectiveness, and collaborative success. Please take the time to familiarize yourselves with the related policies and processes below, and don’t hesitate to reach out with any questions.

Preliminary Proposal Form
Are you interested in starting an education or care initiative or a research project under HSG? If so, start by filling out our Preliminary Proposal Form. This is a short form intended to provide leadership with high-level details so they can ensure it is in line with HSG’s mission and is feasible from a resource perspective. If your preliminary form is approved, you will be asked to submit a more detailed proposal through either the HSG Education or Care Initiative Formal Proposal Form or the HSG Investigator-Initiated Clinical Research Formal Proposal Form. If your preliminary form is not approved, you will be provided with feedback and suggestions for a one-time resubmission.

Policy for Proposals Utilizing HSG Resources
If you are working on an HSG-approved project and are seeking funding from an external company (i.e., not HSG), please complete the Policy for Proposals Utilizing HSG Resources form prior to contacting any potential funders.


8.   Reflections on an Impactful Life: Remembering Christine Hunter

When we think of dedicated professionals, few names shine as brightly as Christine Hunter’s. With a heavy heart, we reflect on her life, one that left an indelible mark on our hearts and the world of medical research. Christine’s journey was nothing short of extraordinary, and her legacy continues to resonate within the Huntington’s and Parkinson’s Disease and communities.

A Passionate Advocate
Christine’s involvement in the Parkinson’s community was profound. She served as a coordinator representative on the Executive Committee at PSG (Parkinson Study Group) and, beyond retirement, remained actively engaged with the Parkinson’s Foundation and the Michael J. Fox Foundation. Her dedication was unwavering, a testament to her heart of gold and a smile that radiated love and kindness to all fortunate enough to know her.

Karen Williams, from Northwestern, eloquently captured Christine’s essence when she said, “Christine always made time for everyone, especially her fellow coordinators. She served as a mentor (officially and unofficially) to so many people over the years and was always available to lend an ear or give her opinion, she was a wealth of knowledge.”

Patients Were Her Priority
Throughout her illustrious career, Christine never lost sight of her patients. Whether speaking at support groups, volunteering at Tourette’s camps, or offering one-on-one support to patients and their families, she embodied the true spirit of compassionate healthcare. ZsaZsa Brown, also from Northwestern, aptly noted, “She was an advocate for the HD community as well as a great role model for us Coordinators. She will truly be missed!” 

A Remarkable Leader and Friend
Joseph Jankovic, MD, a close colleague and friend, beautifully encapsulated Christine’s remarkable contributions. He described her as “a beacon of hope for countless patients with Parkinson’s disease, Huntington’s disease, Tourette syndrome, and many other movement disorders.” Christine’s dedication went beyond borders, as she volunteered on medical missions in underserved areas and touched lives worldwide.

In a poignant personal note, Dr. Jankovic shared, “Christine was my most trusting professional partner, advisor, and confidant as well as a close friend. Because of her unique combination of warm empathy and inspiring enforcer of discipline, I often referred to her as a hybrid between Mother Theresa and General Patton.”

Christine’s sudden and tragic passing on July 26, 2023, during a family vacation in Branson, Missouri, has left a void in our lives and the broader medical community. She is survived by her loving husband, two sons, and four sisters.

As we reflect on Christine’s impactful life, let us honor her memory by carrying forward her passion for our work and striving to make our patients’ lives better. Her kindness, generosity, and wisdom will continue to inspire us and future generations, ensuring her legacy lives on.


9.   Catalysts for the Future: HSG Education and Training

Preparing investigators and coordinators for clinical trial conduct is at the core of the HSG Site Readiness Training Program. The HSG training and development programs serve as a catalyst to ensure ample support and clear guidance to all HSG members. For years, our Site Readiness Training Program (formerly known as “Member Education”) has served as the foundation for advancing clinical trial knowledge and developing what we refer to as “Tiger Teams” – an elite set of sites that can rapidly deploy a clinical study.

Our training programs include a comprehensive collection of tools and resources that enables HSG members to execute clinical trials in rapid time to provide consistent, timely, high-quality data. Providing education and training on innovative assessment tools, new methodologies, and changes in established techniques are key to preparing the next generation of researchers, clinical trial success, and ultimately our ability to make an impact for the HD community.

– Samuel Frank, MD, Co-Chair of HSG Executive Membership Committee

– Jody Goldstein, HSG Director of Clinical Operations


10.   IN THE KNOW

Grant Opportunity
The Hereditary Disease Foundation provides Postdoctoral Fellowships and Grants to researchers around the world to advance the discovery and development of treatments for Huntington’s disease and other brain disorders. They began accepting Letters of Intent for Postdoctoral Fellowships and Grants on September 15, 2023 with a due date of November 1, 2023. Click here to learn more.

Upcoming Events
Help4HD International Symposium – October 13-14 in San Diego, CA
If you’re attending this meeting and would like to connect with HSG’s Director of Clinical Operations, Jody Goldstein, send us a note at info@hsglimited.org.

European Huntington’s Association Conference – October 19-22 in Belgium


Quick Links
HSG Website
HD Trial Finder – US & Canada
HD Trial Finder – Europe
clinicaltrials.gov

If you would like to write an article for the HSG Updates newsletter or have your meeting or event featured, contact kristin.keyes@hsglimited.org 

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.

# # #

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

 

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.

HSG Announces Launch of CME4HD 2019 – Accredited Online Education Platform

Beginning February 1, 2019, the Huntington Study Group (HSG) will offer the latest update to its popular online learning program, CME4HD. Once again, CME4HD courses will be offered free of charge thanks in part to an educational grant from Teva Pharmaceuticals.

CME4HD originally began as in-person training on Huntington’s disease (HD) at the HSG’s annual meeting, before launching in 2018 in an online, self-paced format. Learners can earn up to 3.25 total CME, CNE or IPCE credits by completing all courses in the program. Interested learners can register for a user account on the HSG’s learning platform, and learn more about the CME4HD program on our website: https://huntingtonstudygroup.org/cme4hd-online/

The CME4HD program presents evidence driven data and examples about Huntington’s disease by stage and disease burden. CME4HD 2019 content is designed for healthcare professionals, including social workers, nurses, and neurologists, who provide care to patients with HD. Family members serving as caregivers will also find the content extremely valuable.The HSG is extremely grateful to Bridget Lyon and Jeff McDonald for allowing their documentary, The Inheritance, to be used as a case study and for these courses. The Inheritance honors the efforts of Bridget’s mother to strengthen public awareness of Huntington’s disease. She wanted people to know what it was and how it affected entire families, generation after generation. All users that register for CME4HD 2019 will have access to the full-length documentary.

CME4HD has been planned and implemented by North American Center for Continuing Medical Education, LLC (NACCME) and the Huntington Study group. NACCME is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC) to provide continuing education for the healthcare team.

Huntington Study Group Launches New CME Accredited Interactive Case Study Courses

The Huntington Study Group (HSG), a world leader in spearheading research and conducting clinical trials in Huntington disease (HD), has launched a new offering to its acclaimed CME4HDTM online learning curriculum. The 2020 edition of CME4HDTM features seven interactive case studies that build upon the faculty lecture content launched last year (2019). Learners are presented with real world scenarios that healthcare professionals may encounter working with patients or families at various stages of Huntington disease, and answer questions to determine the best course of action for the scenario. 

“The vision of CME4HD is to show healthcare professionals and providers a way to care for HD patients and families using currently available interventions,” said Dr. Mary Edmondson, Chair of the HSG Provider Education Committee. “Through this program, we hope to inspire them to learn more about this fascinating and challenging patient population and their remarkable families, and to “spotlight” clinical problems that, if managed well, have a great impact in terms of the quality of life for patients and a lasting effect within the extended family.”

Learners can earn up to 1.75 total credits at no cost (CME, CNE, or IPCE). Users with existing HSG Litmos accounts can login and take the new courses at their leisure. New users can sign up for a complimentary HSG Litmos learning account by following the steps on the HSG website (https://huntingtonstudygroup.org/cme4hd-online/). This activity has been supported through an independent educational grant from Genentech. 

“The Huntington Study Group has long been seen as a leader in education on Huntington disease for healthcare professionals. We are proud to continue offering a high quality program like CME4HD to the provider community,” said Dr. Andrew Feigin, HSG Chair. “The efforts of Dr. Edmondson and the Provider Education Committee are reflective of the passion we all have to improve the quality of care for HD patients and families.”

About HSG

Founded in 1993 in Rochester, NY, the Huntington Study Group (HSG) is a not-for-profit organization comprised of the world’s first and largest collaborative network of experts in Huntington disease. The mission of the HSG is seeking treatments that make a difference for those affected by HD. With more than 700 credentialed HD experts at over 125 HSG credentialed research sites worldwide, the HSG is a leader in conducting clinical trials for HD. The HSG also offers educational services like CME4HD ™ for healthcare professionals and care providers on treating patients with HD. For more information, visit our website www.huntingtonstudygroup.org.

About CME4HDTM

CME4HD has been planned and implemented by North American Center for Continuing Medical Education, LLC (NACCME) and the Huntington Study group. NACCME is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC) to provide continuing education for the healthcare team.

Huntington Study Group Announces A Partnership with Prilenia Therapeutics to Conduct A Global Phase 3 Clinical Study of Pridopidine in Huntington’s Disease

Rochester, N.Y.  — September 17, 2020 —The Huntington Study Group (“HSG”), a world leader in clinical research for Huntington’s Disease (HD), announces a partnership with Prilenia Therapeutics, a clinical stage biotech company led by Michael R. Hayden, MD, PhD, to conduct PRidopidine Outcome On Function in Huntington’s Disease (PROOF-HD) clinical study.  PROOF-HD is a global Phase 3, randomized, double-blind, placebo-controlled, parallel arm, multicenter study evaluating the efficacy and safety of pridopidine in patients with early stage HD.

Pridopidine is a first in class small molecule which  is a highly selective Sigma-1 receptor (S1R) agonist. Prior trials of pridopidine in HD have demonstrated good safety and tolerability. The PROOF-HD study seeks to demonstrate that pridopidine slows functional decline over 65 weeks of treatment.

In a prior Phase 2 clinical study, PRIDE-HD, pridopidine administered orally twice a day, was associated with maintenance of functional capacity from baseline compared to placebo at 52 weeks in patients with early HD, as measured by Total Functional Capacity (TFC). Extensive safety data from over 1300 subjects exposed to various oral daily doses of pridopidine demonstrates pridopidine has a favorable safety profile and is well tolerated.

“Slowing the decline in functional capacity in HD patients would be a significant advance in HD care,” said Andrew Feigin, MD, North American Principal Investigator of PROOF-HD. “We are excited to partner with Prilenia to study pridopidine – this study directly aligns with HSG’s mission of seeking treatments that make a difference for those affected by  HD.”

HSG is also partnering with TFS International AB, a clinical research organization (CRO), to conduct this trial in Europe, and with the Clinical Trials Coordination Center at the University of Rochester to assist with efforts in North America.  PROOF-HD will be conducted through HSG Clinical Research, Inc.  

“This is an exciting time in Huntington’s Disease research,” added Ralf Reilmann, MD, PhD, the European Principal Investigator of the study. “Prior trials of pridopidine have suggested that it may maintain functional capacity in early HD patients, as measured by Total Functional Capacity (TFC). PROOF-HD will focus on this potential effect.”

The PROOF-HD study plans to enroll 480 participants aged 25 or older with a clinical diagnosis of adult-onset HD in approximately 30 study centers across the U.S. and Canada, and another 30 study centers across Europe. The study will include a screening period, a double-blind placebo-controlled treatment period up to 78 weeks and optional open-label extension. “There is extensive preclinical evidence that further supports pridopidine’s potential beneficial effect in HD,” said Michael R. Hayden, MD, PhD, Chief Executive Officer at Prilenia. “It is a first-in-class drug candidate, with promising previous clinical results and an established safety profile. We are pleased to be partnering with HSG to explore its impact through the PROOF-HD study.”

About Huntington’s Disease

Huntington’s Disease (HD) is a hereditary neurodegenerative disease characterized by a movement disorder, psychiatric difficulties, and cognitive changes, usually beginning in middle adult life. Additional characteristics of HD include weight loss (probably from a combination of difficulty eating, and calories burned by the involuntary movements), difficulty swallowing, and hard-to-understand speech. About 30,000 people in North America have HD, and another 150,000 are considered “at risk” for inheriting the illness because they have (or had) a parent with HD.

About HSG Clinical Research

Founded in 1993 in Rochester, NY, the Huntington Study Group (HSG) is a not-for-profit organization comprised of the world’s first and largest collaborative network of experts in Huntington’s Disease. HSG Clinical Research, Inc. is a wholly-owned for-profit subsidiary of the HSG, conducts clinical trials to benefit the HSG and its mission of seeking treatments that make a difference for those affected by HD. There are 700 credentialed HD experts at more than 120 HSG credentialed research sites worldwide.  The HSG also offers educational services like CME4HD ™ for healthcare professionals and care providers on treating patients with HD. For more information, visit www.huntingtonstudygroup.org.

About Prilenia

Prilenia is a clinical stage biotech startup founded in 2018 with the purpose of improving the lives of patients and their families by developing treatments for neurodegenerative and neurodevelopmental disorders. Prilenia raised $ 82.5 million thus far and is backed by a group of well-respected investors: Talisman, Forbion, Morningside and Sectoral. The Company is based in Naarden, the Netherlands, Herzliya, Israel and Boston, MA in the U.S. For more information about Prilenia and Pridopidine, please email info@prilenia and visit Prilenia’s website: www.prilenia.com.

About TFS International

TFS was founded in 1996 and has grown to become the leading global mid-size clinical CRO focusing on small and mid-size Biotech customers. TFS employs nearly 700 professionals throughout 21 countries and currently delivers clinical research services in more than 40 countries. TFS provides end-to-end solutions including full clinical development services, strategic resourcing, and flexible single services. TFS partnering approach with customers is based on our four business principles – commitment, flexibility, value creation and global reach. Our core therapeutic specialties are Dermatology, Hematology and Oncology, Ophthalmology as well as Internal Medicine and Neurology. Detailed information about TFS, and its business offerings can be obtained through www.tfscro.com.

About the CTCC

The Clinical Trials Coordination Center (CTCC) is part of the University of Rochester Medical Center’s Center for Health + Technology (CHeT). The CTCC specializes in the development, management, and conduct of clinical research studies. Over the past 25 years, the CTCC has managed the conduct of over 100 clinical research studies with 45 sponsors (government, industry, and private) that enrolled over 38,000 research participants in US, Canada, Europe, New Zealand, and Australia. Visit the CTCC website https://www.urmc.rochester.edu/health-technology/our-expertise/clinical-trials-coordination.aspx.

HSG Announces Virtual Annual Meeting in 2020

The Huntington Study Group announced that it is taking the early and proactive step of moving this year’s annual meeting, HSG 2020: HD IN FOCUS, to a virtual experience during October 29-31, 2020. “While we will not gather together this year in-person, we have developed an interesting, novel, and innovative program that will be brought to you anywhere you are located,” said Huntington Study Group Chair Andrew Feigin, MD in an email to HSG members and the HD community.

The Huntington Study Group will hold a highly engaging and interactive virtual online meeting. HSG 2020 will still provide many of the same experiences attendees are used to — but from the safety and comfort of their home or office, which includes opportunities to network and interactive poster sessions, and much more, such as:

  • INTERACTIVE SESSIONS – plenary presentations, panel discussions, with live Q&A sessions sharing the latest in research and treatments.
  • An exciting VIRTUAL FAMILY DAY symposium for HD families to hear from HD experts and community advocates.
  • BREAKOUT SESSIONS and WORKING GROUPS.
  • CONTINUING EDUCATION (CE) credits for attending sessions.
  • Virtual NETWORKING LOUNGES to meet up with, engage and collaborate with colleagues, friends, and experts.
  • A simulated EXHIBIT HALL to engage with representatives from industry and advocacy.
  • An interactive POSTER PAVILION to browse accepted research abstract posters, and engage with poster presenters at scheduled times.

Additional details, registration information, and call for abstracts will be made available in the coming weeks. You can also bookmark our HSG 2020 web page for the latest upddates (https://huntingtonstudygroup.org/hsg-2020).

Huntington Study Group will be updating sponsor packages with some exciting opportunities to maintain a consistent experience as our usual in-person event. Organizations interested in sponsorship or exhibitor opportunities are requested to contact Kristin Strazdins (kristin@hsglimited.org or 585.242.0967) for more information on becoming a sponsor and/or exhibitor.

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