Press Release

Neurocrine Biosciences Announces Publication of Full KINECT™-HD Phase 3 Study Results of Valbenazine for the Treatment of Chorea Associated with Huntington’s Disease in The Lancet Neurology 

  • Statistically Significant Improvement in Chorea Associated with Huntington’s Disease Seen as Early as Week 2
  • Chorea Improvement Supported by Statistically Significant Clinical Global Impression of Change (CGI-C) Response Status and Patient Global Impression of Change (PGI-C) Response Status Scores at Week 12
  • First Phase 3 Study to Implement Huntington’s Disease Health Index (HD-HI), a Patient-Reported Outcome Measure, Showed Reduced Disease Burden as Reported by Patients Receiving Valbenazine versus Placebo
  • Supplemental New Drug Application (sNDA) Filed, with Prescription Drug User Fee Act (PDUFA) Target Date Set for August 20, 2023

Neurocrine Biosciences, Inc. today announced that the complete study results from its Phase 3 KINECT™-HD study investigating valbenazine for the treatment of chorea associated with Huntington’s disease (HD) has been published in The Lancet Neurology online edition and will appear in the June 2023 print issue. The study met its primary and secondary endpoints, demonstrating a reduction in chorea symptoms associated with HD and improvement of overall chorea severity as noticed by clinicians and patients, with improvement seen as early as Week 2 of the initial dose in the 12-week study.
 
The KINECT-HD randomized double-blind, placebo-controlled Phase 3 study was conducted with 128 participants at 46 Huntington Study Group (HSG)–credentialed sites in North America.

The primary endpoint was a reduction in severity of chorea, the cardinal motor feature in HD, as measured by change in the HSG-developed Unified Huntington’s Disease Rating Scale (UHDRS®) Total Maximal Chorea (TMC) score from baseline to the average score at weeks 10 and 12. The TMC score is part of the motor assessment of the UHDRS that measures chorea. 
 
To read the full press release, click here.

Huntington Study Group Achieves Last Patient Last Visit for Its Observational Study to Test Virtual Use of the Unified Huntington’s Disease Rating Scale®

Rochester, NY – May 16, 2023 – The Huntington Study Group (HSG) together with its wholly owned subsidiary, HSG Clinical Research, Inc., announces an important milestone in the novel observational study Virtual Unified Huntington’s Disease Rating Scale (vUHDRS™). The last participant completed the last visit, marking study completion.

The purpose of this study is to determine the reliability of administering all sections of the Unified Huntington’s Disease Rating Scale (UHDRS®) virtually compared to in-person for clinical and clinical trial use. The UHDRS® is the gold-standard in assessing motor function, cognitive function, behavioral abnormalities, and functional capacity in Huntington’s disease (HD). Since the tool was originally developed in 1993, the HSG-owned instrument has been used in part or in total as the primary endpoint in many HD trials. The changing face of clinical trials is prompting the need for reliable tools for assessing and interacting with patients and research participants remotely. This need became clearer and more urgent during the COVID-19 pandemic, accelerating HSG’s development of this study.

vUHDRS™ has the potential to positively impact care delivery and clinical trial operations as part of remote and hybrid trials, effectively removing some of the barriers people face when participating in research studies. This study could also increase data availability, frequency of data collection and could enhance clinical care.

Samuel Frank, MD, Associate Professor of Neurology, Director of the Huntington’s Disease Society of America Center of Excellence at Beth Israel Deaconess Medical Center, and Principal Investigator of the vUHDRS™ study, said “It’s exciting to be part of this critical study that will enhance future clinical trials in HD. We couldn’t do any of this without an incredible team and the help of our dedicated participants for whom we are so thankful.”

The vUHDRS™ study was conducted at 16 HSG sites across the US, where 60 subjects with motor manifest HD were followed over approximately six weeks through a combination of in-person and remote visits. The study team is beginning to analyze the data and hopes to announce results in third quarter 2023.

More information about this study will also be presented at HSG’s 30th Annual Meeting in November. To learn more about the meeting, including sponsorship opportunities, click here.  
 
About Huntington’s disease 
Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent.  Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered.

About Huntington Study Group / HSG Clinical Research, Inc.  
The Huntington Study Group (HSG), a not-for-profit organization founded in 1993 in Rochester, NY, and its wholly owned subsidiary, HSG Clinical Research, Inc., designs and conducts clinical trials through the world’s first and largest collaborative network of over 800 experts in Huntington’s disease at more than 130 HSG credentialed research sites worldwide. HSG is dedicated to improving the lives of people impacted by Huntington’s disease through research, education, and collaboration. For more information, visit www.huntingtonstudygroup.org. 

To read the full press release, click here.

HSG and HD-CAB Collaborate to Provide Global Patient Advisory Board

Rochester, NY — March 30, 2023 — The Huntington Study Group (HSG) together with its wholly owned subsidiary, HSG Clinical Research, Inc., a world leader in conducting clinical trials for Huntington’s disease (HD), recently collaborated with Huntington’s Disease – Community Advisory Board (HD-CAB) to provide a Global Patient Advisory Board on behalf of Annexon Biosciences, Inc. Annexon is a clinical-stage biopharmaceutical company whose focus is on developing a pipeline of novel therapies for patients with disorders of the body, brain, and eye that are caused by improper regulation of the body’s complement system1.

The Advisory Board (Ad Board), held on November 30, 2022 in Jersey City, New Jersey, USA, was a six-hour session that included eleven HD-CAB advocate members from the US, Canada, Scotland, France, Italy, Sweden, Pakistan, and India, who convened to share with Annexon their stories and viewpoints and to discuss the challenges HD trial participants and their families face. One of the goals of the Ad Board was to encourage open communication about how trial sponsors and Clinical Research Organizations (CROs) can better support HD families before, during, and after clinical trials. This conversation was led by Astri Arnesen, Chair of HD-CAB and President of the European Huntington Association. Dr. Christopher Ross, HSG’s Chief Scientific Officer and Dr. Jody Corey-Bloom, Neurologist at UC San Diego and longtime HSG member, facilitated a discussion regarding Annexon’s scientific rationale in HD and planned trial design.

“HD-CAB represents the voice of the global HD community. Consisting of HD family members from six continents and 20 countries, our aim is to ensure that critical factors aren’t overlooked in the design and implementation of clinical trials. It was a pleasure to work with HSG and Annexon as they actively listened to the real-world experience of HD-CAB advocates, adjusted course as appropriate and committed to doing so on an ongoing basis,” said Astri Arnesen.

“I would like to thank the HD-CAB advocates as well as the HSG and HD-CAB management teams for the expertise and actionable next steps shared at the Global Patient Advisory Board,” said Peter Collins, VP, Global Program Team Lead of the Neurodegeneration Franchise at Annexon Biosciences. “We look forward to continued collaborations with leading patient organizations, such as HD-CAB and HSG, to ensure Annexon consistently engages with, and incorporates feedback from, the patients and families we seek to serve.”

HD-CAB recently completed a follow-up International Ad Board on behalf of Annexon Biosciences at the HYDO International Congress in Glasgow, Scotland in March 2023. HSG, HD-CAB, and Annexon Biosciences are continuing to discuss opportunities for future collaborations.

Dr. Ross noted, “HSG is always looking for innovative ways to support and engage the HD community and to make sure their voices are heard. We found this collaborative effort to be truly meaningful and hope this opportunity is just one of many that we will have the privilege of participating in. Our team is grateful to HD-CAB for their invaluable contributions and to Annexon for enabling our organizations to make this important meeting come to fruition. It was a great success.”

If you’re interested in learning more about collaboration opportunities, please contact info@hsglimited.org.

1 The body’s complement system is part of the immune system. It is responsible for defending cells and tissues from infections and diseases.

About Huntington’s disease
Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent. Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered.

About Huntington Study Group / HSG Clinical Research, Inc.
The Huntington Study Group (HSG), a not-for-profit organization founded in 1993 in Rochester, NY, and its wholly owned subsidiary, HSG Clinical Research, Inc., designs and conducts clinical trials through the world’s first and largest collaborative network of over 800 experts in Huntington’s disease at more than 130 HSG credentialed research sites worldwide. HSG is dedicated to improving the lives of people impacted by Huntington’s disease through research, education, and collaboration. For more information, visit www.huntingtonstudygroup.org.

About HD-Community Advisory Board
HD-Community Advisory Board (HD-CAB) is a coalition of partners from the European Huntington Association, the International Huntington Association, and the Huntington’s Disease Youth Organization whose mission is to represent the voice of the global HD community and provide HD community experience to regulators, industry, researchers, and governing bodies. HD-CAB provides the unique expertise of living with HD to stakeholders relevant for therapeutic development. For more information, visit www.hd-cab.org.

Click here to view the full press release.

HSG Announces 30th Annual Meeting

Rochester, NY — March 21, 2023 — The Huntington Study Group (HSG), a world leader in conducting clinical trials and providing educational programming for Huntington’s disease (HD), recently announced that their 2023 Annual Meeting will be held November 2-4, 2023 at the Sheraton Grand at Wild Horse Pass in Chandler, AZ, a little south of Phoenix. This year is particularly special, as the HSG will be honoring its 30th year as an organization, along with the 30th anniversary of the HD gene discovery.
 
“We are proud of the work HSG has completed over the past 30 years. As Co-Chairs, we look forward to a meaningful, productive and promising future for our patients and families impacted by HD,” said Sam Frank, MD and Sandra Kostyk, MD, PhD, Co-Chairs of HSG’s Executive Membership Committee.
 
The three-day long Annual Meeting provides an excellent forum for scientists, industry partners, HSG research sites (largely from academic institutions), advocacy groups, members, staff, HD patients, families, and caregivers around the world to present and learn about new findings for Huntington’s disease treatments. Through the Annual Meeting, the HSG facilitates programs to provide the latest updates on current HD clinical trials, scientific advancements, and industry developments. Most importantly, the event provides opportunities for connection, networking, and collaboration. Attendees generally report leaving HSG’s annual meetings feeling energized, inspired, and full of hope and this year’s meeting should be equally impactful.
 
“I look forward to attending HSG’s event each year and consider it a very useful opportunity to hear from and interact with HD thought leaders, and to make valuable connections,” said Dietrich Haubenberger, MHSc, MD, Executive Medical Director for Clinical Development at Neurocrine Biosciences.
 
This year’s agenda is being planned by the newly formed Executive Membership Committee and the Family Day Planning Committee. Two full weekdays will be devoted to scientific updates and learning opportunities while Saturday, Family Day, will be dedicated to patients, families, caregivers, and the local HD community.
 
Shari Kinel, JD, CEO of Huntington Study Group noted, “We’re looking forward to everyone coming together to connect and participate in a full range of scientific programming and educational activities. We want to honor the incredible network of people who continue to support our mission of accelerating treatments that make a difference for Huntington’s disease.”

More details and registration opportunities will be coming in the next few months.
If you’d like to sponsor HSG’s 30th Annual Meeting, please click here for details.

About Huntington’s disease
Huntington’s disease (HD) is a progressive hereditary neurodegenerative disease characterized by movement disorders, psychiatric difficulties, and cognitive changes. HD symptoms usually present in middle adult life but can begin at any age. HD is a rare disorder — about 200,000 people worldwide have been diagnosed but many more are at risk for inheriting the disease from a parent. Huntington’s disease impacts people of all genders, races, and ethnicities. While symptomatic treatments are available, a cure for HD has not yet been discovered.

About Huntington Study Group / HSG Clinical Research, Inc. 
Founded in 1993 in Rochester, NY, the Huntington Study Group (HSG) is a not-for-profit organization composed of the world’s first and largest collaborative network of over 800 experts in Huntington’s disease at more than 130 HSG credentialed research sites worldwide. HSG Clinical Research, Inc., a wholly owned subsidiary of the HSG, is a full-service clinical research organization that specializes in conducting HD trials. The HSG also offers educational services for healthcare professionals and care providers on treating patients with HD. For more information, visit www.huntingtonstudygroup.org.

Click here to view the full press release.

Huntington Study Group Annual Meeting

Rochester, NY – November 29, 2022 – The Huntington Study Group (HSG), a world leader in conducting clinical trials for Huntington’s Disease (HD), recently held its 29th Annual Meeting in Tampa, FL. This renowned meeting brings together HD thought leaders, scientific experts, industry partners and sponsors, advocacy groups, and HD family and community members from around the world.

At this year’s meeting, attendees heard about exciting clinical trial advancements including gene therapies, biomarkers (e.g., PET Imaging of Synapses and Mutant Huntingtin), and updates regarding the development and progress of novel tools being used to improve research and care (e.g., HSG’s® and myHDstory™ observational studies). These presentations and other inspiring talks raised hope for the whole HD community of patients, families, clinicians, and researchers.

“The plenary sessions at the Huntington Study Group 2022 Annual Meeting in Tampa were truly some of the best I can remember! I was particularly excited by the session on imaging and tissue biomarkers and also the Innovator’s Forum, which brought together a large number of early-stage therapeutics companies focused on preclinical drug development for HD,” said Jody Corey-Bloom, MD, PhD, Neurologist at UC San Diego and member of HSG.

Christopher Ross, MD, PhD Chief Scientific Officer at HSG, said “Interestingly, small molecules (i.e., drugs that can be taken orally) seem to be undergoing a bit of resurgence. It’s great to see that there are studies of so many approaches to treating HD.”

At the meeting, HSG announced more details about their expanded clinical trial operations. The organization offers a full suite of Clinical Research Organization (CRO) services including Global Trial Operations, Advisory Boards, and Protocol Design and Review. For 29 years, the HSG has been leveraging the expertise, innovation, and commitment of their members and staff to set the gold standard for running HD clinical trials. The HSG’s dedication to its mission – seeking treatments that make a difference – has never been stronger. 

Shari Kinel, JD, CEO of Huntington Study Group noted, “After holding the meeting remotely for the past two years due to COVID, the joy and excitement we all felt from being back together in person was palpable. We are truly grateful to all of our attendees, the HD community, and to everyone who helped make this event a remarkable success.”

Next year, HSG will celebrate its 30th Anniversary at their 2023 Annual Meeting.

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. About 40,000 people in North America have HD, and another 200,000 are considered “at risk” for inheriting the illness because they have (or had) a parent with HD.
 
About Huntington Study Group / 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 whose mission is to seek treatments that make a difference for those affected by HD. HSG Clinical Research, Inc., a wholly owned subsidiary of the HSG, is a full-service clinical research organization that specializes on conducting trials in HD. There are over 800 HD experts at more than 130 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.

View full press release.

Huntington Study Group Announces Results from the HD-Net Assessment on the State of Care for Huntington’s Disease in the United States

Rochester, NY — October 25, 2022 — The Huntington Study Group (HSG), a world leader in clinical research for Huntington’s Disease (HD) for over 30 years, is pleased to announce the results from the novel HD-Net survey, which examined the United States Huntington’s disease (HD) care delivery in a variety of clinic settings by HD specialists and non-specialists.

“The importance of this research survey is the real-world sampling of HD care across all types of medical practices in the United States and how it shows us the inequality in care levels with different practice types. This is a call for us to increase outreach and education to those practices who are already seeing HD patients to improve care and access to specialty services,” said Lauren Seeberger, MD, HD-Net Principal Investigator and Associate Professor at the University of Colorado.

The HSG collaborated with the University of Colorado, University of California San Diego, Genentech, Inc., and RAND Corporation, to make this novel survey accessible for qualifying United States residents to access. The HD-Net is obtained a clearer understanding of current care structure and delivery of care through a survey of representative US physicians treating HD patients. The survey included 40 closed-ended evaluative items and one open-ended item to a sample of 339 US practices. Unique to this survey was the inclusion of non-specialists. Findings concluded that HD care was inconsistently applied across the US. Practices led by neurologists trained in movement disorders, and higher HD volume practices, tended to be better equipped to provide multi-disciplinary staffing and procedures as compared to those with fewer numbers of HD patients.

“We would like to thank Lauren Seeberger, MD; Jody Corey-Bloom, MD, PhD; Michael O’Brien; Diana Slowiejko, PharmD, PhD; Danielle Schlang, MA; Marika S. Booth, MS; Beth Ann Griffin, PhD; and Peggy G. Chen, MD, MSc, MHS for their work on HD-Net,” said Shari Kinel, JD, Chief Executive Officer of the Huntington Study Group. “The HSG would also like to extend our appreciation to Diana Slowiejko, PharmD, PhD, the Principal Med Science Director of Genentech, for her pivotal role in project development and support.

Acknowledgment
Acknowledgment to the HD-Net Steering Committee (Donald Higgins MD, Samuel S. Stratton VA Medical Center, Albany, New York; Joseph Jankovic MD, Baylor College of Medicine, Houston, Texas; Jamie Levey, MBA, CHDI Foundation; Karen Marder MD, MPH, Columbia University Medical Center, New York, New York; Julie Parsons MD, Children’s Hospital Colorado, Aurora, Colorado; Abdul R Shaikh, PhD, Guidehouse, Washington, DC; Emily Troncoso; Louise Vetter, Huntington’s Disease Society of America, New York, New York); and for the generous support from Genentech, Inc.

For more information about HD-Net, visit https://huntingtonstudygroup.org/hd-net/.

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. About 40,000 people in North America have HD, and another 200,000 are considered “at risk” for inheriting the illness because they have (or had) a parent with HD. 

About Huntington Study Group / 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 whose mission is to seek treatments that make a difference for those affected by HD. HSG Clinical Research, Inc., a wholly owned subsidiary of the HSG, is a full-service clinical research organization that specializes on conducting trials in 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 HD-Net
The HD-Net Community was formed in 2019 through funding by Genentech, managed by the Huntington Study Group (HSG) and supported by leaders of the HD community – CHDI, HDSA and others. HD-Net is committed to elevating the level of HD care and bridging the gaps that exist in the access to that care. HD-Net’s initial focus is to improve care, deepen HD knowledge, provide resources, emphasize the patient, harness technology, and support new therapies.

View full press release.

Huntington Study Group Announces myHDstory™ Pilot Study:  Making HD Voices ­­­Heard Has Reached Target Enrollment

Rochester, N.Y. — October 18, 2022 — The Huntington Study Group (HSG), a world leader in clinical research for Huntington’s Disease (HD) for over 30 years, is pleased to announce that the pilot study, Making HD Voices Heard, on the new online research platform, myHDstory™, has reached its enrollment target. 

“The HSG is beyond thrilled by the level of participation in this first-of-its-kind study. We are extremely grateful to all the participants who enrolled,” said Dr. Karen Anderson, Professor of Psychiatry and Neurology at Georgetown University Medical Center and Principal Investigator of the Making HD Voices Heard study. “We hope the data and lessons learned from this pilot will inform and drive further virtual studies that will engage larger segments of the Huntington’s Disease (HD) population, family members, and caregivers, thereby improving overall development of HD care and treatments,” said Ira Shoulson, MD, Professor of Neurology at the University of Rochester and founder of Grey Matter Technologies, now a wholly owned subsidiary of Modality.ai.

The HSG collaborated with technology companies Grey Matter Technologies and Neurotargeting LLC, to make this novel online research study accessible for qualifying United States residents to access. The myHDstory™ platform is intended to capture longitudinal data on Huntington’s disease from the patient perspective, giving voice to consenting adults to report what they feel, experience, and how they function with Huntington’s disease. This research platform is a significant opportunity for the HSG to serve the HD community because myHDstory™ enables collection of data from patients about their symptoms, without having to schedule in-office visits.
 
“We thank the Griffin Foundation, NJ Cure HD, Ira and Josie Shoulson, Elise Kayson-Rubin, and Richard Rubin for their financial support of myHDstory,” said Shari Kinel, Chief Executive Officer of the Huntington Study Group. “The HSG would also like to extend our appreciation to advocacy groups who helped raise awareness of the pilot study, including Help 4 HD, Huntington’s Disease Youth Organization (HDYO), HD Reach, and Huntington’s Disease Society of America (HDSA).” 

For more information about myHDstoryTM, visit https://huntingtonstudygroup.org/myhdstory/  

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. About 40,000 people in North America have HD, and another 200,000 are considered “at risk” for inheriting the illness because they have (or had) a parent with HD. 

About Huntington Study Group / 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 whose mission is to seek treatments that make a difference for those affected by HD. HSG Clinical Research, Inc., a wholly owned subsidiary of the HSG, is a full-service clinical research organization that specializes on conducting trials in 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 Neurotargeting

Neurotargeting was founded in 2007 by Pierre-Francois D’Haese, PhD., Benoit Dawant, PhD. and Peter Konrad, M.D PhD. Neurotargeting, LLC emerged from more than a decade of research performed at Vanderbilt University and supported by NIH funding focused on solving such issues. From this research emerged a unique system called Cranial vault, connecting some of the active groups researching the new potential of DBS such as Vanderbilt University, Ohio State University, Wake Forest University, and the VA in Richmond. With a unique research relationship with its partners, Neurotargeting has built a disruptive software platform. By linking care providers, neuroscience centers and device manufacturers, Neurotargeting aims at improving patients’ care experience and life.

About Modality.ai

Modality.ai is a digital health startup, which acquired Grey Matter Technologies in June 2022. Grey Matter was founded in January 2017 by Ira Shoulson, MD and Carol A Christopher, PhD, who had worked together for nearly three decades to advance innovative medical products for patients with neurological and developmental disorders. Grey Matter’s founders, management team and advisors have long recognized the unmet need to harness the power of the patient’s story, typically told verbatim in unstructured language. Together with the Modality.ai team, they are leading the application of data science technologies aimed at Making Patients Heard™ in healthcare and clinical research settings.

View full press release.