Press Releases

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.

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Clinical Research Organization, Huntington Study Group “Firsts” Continue

Rochester, NY December 5, 2022 – The Huntington Study Group (HSG), a world leader in conducting clinical trials for Huntington’s Disease (HD), through its Clinical Research Organization (CRO), HSG Clinical Research, Inc. (HSGCR), continues making groundbreaking progress in the HD world. At their Annual Meeting in November, the HSG team presented exciting updates about their Clinical Research Organization’s achievements.

There were many notable highlights, including information about two current HSG clinical trials. The KINECT-HD/KINECT-HD2 trials, sponsored by Neurocrine Biosciences, Inc. (NBI), met enrollment goals and continued study activities during COVID-19. It was announced during the meeting that the KINECT-HD trial met its primary endpoint of change in chorea severity using the Total Maximal Chorea (TMC) score of the UHDRS® from screening period baseline to maintenance period. Improvement in the TMC score was significantly greater with Valbenazine versus placebo. KINECT-HD2 is an open-label trial that is currently enrolling subjects.

Erin Furr-Stimming, MD, the Principal Investigator of the KINECT studies, said,  “The strong partnership between HSG and NBI was instrumental in facilitating a successful symptomatic trial in HD, an important milestone advancing HD care and awareness. We are incredibly grateful to the study participants and their care partners who participated in KINECT-HD and continue to participate in KINECT-HD2.”

The Phase 3 global PROOF-HD trial, sponsored by Prilenia Therapeutics, is assessing the effect of pridopidine on function in early-stage HD subjects. The trial launched early during COVID-19 and enrolled 499 subjects over 1 year during the height of the pandemic. Andrew Feigin, MD, the North American Principal Investigator (PI) of the study and HSG’s Chief Medical Officer, said “The HSG is proud to be working with an amazing team to conduct this international multicenter clinical trial. The rapid rate of recruitment in this study during the most challenging of times speaks to the inspiring dedication, commitment, and enthusiasm of the HD community.”

The SIGNAL trial, sponsored by Vaccinex, Inc., is the first IV Infusion trial in HD and detailed results were recently published in Nature Medicine1. This 301-subject study met enrollment goals and completed closeout ahead of schedule during COVID-19.  

Elise Kayson, MS, ANP, the VP of Clinical Operations, said, “HSGCR is committed to designing and conducting innovative HD clinical trials. Our dedicated and experienced HD centric team works closely with HSG sites to be efficient and trial ready to meet the needs of our sponsors and patients and families. HSGCR’s recent successful accomplishments speak multitudes about our capabilities as a CRO.” 

Since the HSG was founded in 1993, the organization has continually proven that they are a leader in HD clinical research. The HSG continues to innovate in clinical trial design, biomarkers, and in the operational side of HD clinical trials. Notably, the HSG conducted the clinical trials for the first and only two FDA approved drugs for HD, the first clinical trial in HD, and the first longitudinal study in HD.

“As the year comes to a close, our team is reflecting on the impact our collective efforts have made, and we’re looking forward to continuing to do everything we can to make a difference in 2023 and beyond. We would also like to extend our gratitude to all of our partners, sponsors, and collaborators who make what we do every day a possibility and a reality,” said Shari Kinel, JD, CEO.

1Feigin, A., Evans, E.E., Fisher, T.L. et al. Pepinemab antibody blockade of SEMA4D in early Huntington’s disease: a randomized, placebo-controlled, phase 2 trial. Nat Med 28, 2183–2193 (2022). https://doi.org/10.1038/s41591-022-01919-8

HSG offers a full suite of CRO services. To learn more, please visit: https://huntingtonstudygroup.org/wp-content/uploads/HSG_Trial_Services_Sheet.pdf

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.

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Huntington Study Group Names First Chief Innovation Officer

Rochester, NY  January 10, 2023 — The Huntington Study Group (HSG), a world leader in clinical research for Huntington’s Disease (HD) for over 30 years, today announced Brett Kinsler has been named to the newly created position of Chief Innovation Officer (CINO). Kinsler will build on HSG’s remarkable history of mission-focused, transformative advances in clinical trial work related to HD. “We created this role knowing our organization would benefit greatly from a champion who could generate and cultivate ideas and guide their successful implementation,” said Shari Kinel, Chief Executive Officer of HSG. “We know Brett is the right person, in part due to his previous experience with HSG.”

Last year, Kinsler served as a consultant to develop HSG’s myHDstory™, an online platform that allows the HD community to contribute to research remotely. “The development and implementation of this platform were key to the success of our pilot observational study, ‘Making HD Voices Heard.’ We owe the ease of participation in our study in large part to Brett and the structured thinking he brought to the team,” said Dr. Karen Anderson, MD, Professor of Psychiatry and Neurology at Georgetown University Medical Center and Principal Investigator of the study. Brett will also be an instrumental part of the organization’s plans to create a comprehensive education center dedicated to enhancing the knowledge and capabilities of HSG members, families, and sponsors to improve research and care. The formation of this center is another example of how HSG continues to lead, innovate, and grow.

Kinsler’s broad background, which includes experience in clinical trials, clinical practice, entrepreneurialism, post-graduate teaching, and strategic advisory for hospitals, health systems, payers, collaboratives, federal and state governments are a natural fit for HSG and its wholly owned clinical research subsidiary, HSG Clinical Research, Inc. 

“I’m so excited to join the amazing HSG team in their mission to discover treatments that matter to people affected by HD,” Kinsler said. “I love applying strategic thinking, technology, and innovation to develop new solutions and processes that drive better outcomes. HSG is at the forefront of HD research and is making history with its many firsts. I cannot wait to be a part of that and help HSG continue to grow as a leader in HD research, education, and care.”

Kinsler began as the new CINO on January 9, 2023.

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 30,000 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 comprising 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 in conducting HD trials.  There are 700 credentialed HD experts at more than 120 HSG credentialed research sites worldwide.  The HSG also offers educational services such as CME4HD™ for healthcare professionals and care providers treating patients with HD. For more information, visit www.huntingtonstudygroup.org.

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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.

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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.

Huntington Study Group Enrolls First Participant for Its Observational Study to Test the Reliability of the Virtual Use of the  Unified Huntington’s Disease Rating Scale® in Clinical Trials for Huntington’s Disease

Rochester, N.Y.   October 12, 2022 — The Huntington Study Group (HSG), a world leader in conducting clinical trials for Huntington’s Disease (HD) for over 30 years, together with its Clinical Research Organization (“CRO”), the HSG Clinical Research, Inc., today announces the enrollment of the first participant of its novel and innovative observational study, Virtual Unified Huntington’s Disease Rating Scale (vUHDRS®), assessing the reliability of a virtual use of the HSG’s standard assessment tool, the Unified Huntington’s Disease Rating Scale (UHDRS®). The first participant was enrolled at Hereditary Neurological Disease Centre in Wichita, KS.

View full press release.

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.

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.

SIGNAL completes enrollment in cohort A

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

Auspex Press Release: December 16, 2014

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

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

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

NDA Submission Planned by Mid-2015

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

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

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

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

First-HD Topline Results

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

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

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

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

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

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

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

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

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

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

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

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

ARC-HD Switch Study

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

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

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

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

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

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

Upcoming SD-809 Clinical Data and Other Milestones

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

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

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

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

Conference Call Today

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

About the First-HD Trial

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

About the ARC-HD Switch Trial

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

About Huntington’s Disease

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

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

About the Huntington Study Group (HSG)

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

About Auspex Pharmaceuticals

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

Forward-Looking Statements

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

CONTACT: Corporate Communications Contacts:

         

         For Media:

         Dan Budwick, Pure Communications, Inc.

         dan@purecommunicationsinc.com

         (973) 271-6085

         

         For Investors:

         Monique Allaire Lyons, Pure Communications, Inc.

         monique@purecommunicationsinc.com

         (617) 895-9511

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

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