News

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.

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.

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.

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.

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.

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

 

Overnight Switch from Tetrabenazine to Deutetrabenazine Safe, Trial Shows

JAMA Neurology paper published today from ARC-HD Trial

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

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

 

“This trial provides more good news for our patients who need options for medication to control their chorea,” said Samuel Frank, MD, HSG’s principal investigator of First-HD and director of the HDSA Center of Excellence at Beth Israel Deaconess Medical Center in Boston. “We are grateful to the people who volunteered in this trial and their families who supported their participation.”

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

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

Deutetrabenazine is structurally related to tetrabenazine with deuterium atoms placed at key positions in the molecule, prolonging plasma half-life and reducing metabolic variability, without changing target pharmacology.

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

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

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

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

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Help4HD Blog Radio features I am the DIFFERENCE campaign

Help4HD gave the Huntington Study Group the opportunity on its radio show to update the community on our many current projects, including the I am the DIFFERENCE campaign. Katrina Hamel, VP of Help4HD, hosted the show with guests Ann Nelson, an HD community member and participant in the campaign, and Heather Hare, HSG’s director of Communications & Outreach. Listen to the show by clicking here.

To learn more about HSG 2017: Elevating HD, click here.

To participate in the HD-HI survey mentioned on the show, click here.

Updates on the HDCIP survey are forthcoming.

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.

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