CHDI FOUNDATIONCHDI

Accelerating Therapeutic Development for Huntington's Disease

  • Preclinical research
  • Scientific Publications
  • Community Resources
    • Materials
      • Research tools & reagents
      • Mouse Models
      • Clinical biorepository
    • Data sharing & infomatics
      • Preclinical informatics: HDinHD
    • Funding & partnerships
      • Academic Proposals
      • External Partnership Proposals
    • HD expertise & guidance
      • Independent Statistical Standing Committee
      • Clinical outcomes: Rating scales
      • Field guide to HD mouse models
  • News & Video
    • News
    • HD Therapeutics Conference
      • 2022 Presentations
      • 2021 Presentations
      • 2020 Presentations
      • 2019 Presentations
      • 2018 Presentations
      • 2017 Presentations
      • 2016 Presentations
      • 2015 Presentations
      • 2014 Presentations
      • 2013 Presentations
      • 2012 Presentations
      • 2011 Presentations
    • Postcards
    • Events
  • About Us
    • The Foundation
    • People
    • Policies
      • Data, Reagents, and Biomaterials Sharing Policy
      • Publication Policy
      • Reimbursement of Publication Costs
      • Financial Support for Conferences, Meetings, or Workshops
      • Academic Overhead
      • Academic Salary Limits
      • Leave of Absence Policy
      • Eligible Personnel
  • Preclinical research
  • Scientific Publications
  • Community Resources
    • Materials
      • Research tools & reagents
      • Mouse Models
      • Clinical biorepository
    • Data sharing & infomatics
      • Preclinical informatics: HDinHD
    • Funding & partnerships
      • Academic Proposals
      • External Partnership Proposals
    • HD expertise & guidance
      • Independent Statistical Standing Committee
      • Clinical outcomes: Rating scales
      • Field guide to HD mouse models
  • News & Video
    • News
    • HD Therapeutics Conference
      • 2022 Presentations
      • 2021 Presentations
      • 2020 Presentations
      • 2019 Presentations
      • 2018 Presentations
      • 2017 Presentations
      • 2016 Presentations
      • 2015 Presentations
      • 2014 Presentations
      • 2013 Presentations
      • 2012 Presentations
      • 2011 Presentations
    • Postcards
    • Events
  • About Us
    • The Foundation
    • People
    • Policies
      • Data, Reagents, and Biomaterials Sharing Policy
      • Publication Policy
      • Reimbursement of Publication Costs
      • Financial Support for Conferences, Meetings, or Workshops
      • Academic Overhead
      • Academic Salary Limits
      • Leave of Absence Policy
      • Eligible Personnel

July 21, 2015

Isis Pharmaceuticals initiates clinical study of ISIS-HTTRx in patients with Huntington’s disease

First therapy designed to directly target the cause of disease

Isis earns $22 million milestone payment from Roche

Carlsbad, Calif., July 21, 2015 –Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today announced that it has initiated a Phase 1/2a clinical study of ISIS-HTTRx in patients with Huntington’s disease (HD). ISIS-HTTRx is the first therapy to enter clinical development that is designed to directly target the cause of the disease by reducing the production of the protein responsible for HD. HD is a rare genetic neurological disease in which patients experience deterioration of both mental abilities and physical control. Presently, there are no disease-modifying treatments for HD, with current therapies focused only on treating disease symptoms. ISIS-HTTRx has been granted orphan drug designation by the European Medicines Agency for the treatment of patients with HD. Orphan drug designation is granted to products designed to diagnose, prevent or treat life-threatening or very serious conditions that affect not more than five in 10,000 persons in the European Union.

“Although the toxic protein produced from the huntingtin (HTT) gene in HD patients has been a target of interest for many years, no therapies have advanced to clinical trials to treat the underlying cause of the disease. Our antisense technology has enabled us to discover and develop ISIS-HTTRx, the first therapeutic approach designed to treat the genetic cause of HD. Together with Roche, we are committed to investigating this approach to treat patients with HD, a devastating disease that typically affects generations of families,” said C. Frank Bennett, Ph.D., senior vice president of research at Isis Pharmaceuticals.

“Initiating the clinical study of ISIS-HTTRx in patients with HD is the first step in developing a treatment that could significantly impact a patient’s disease. It is also an important milestone in our collaboration with Roche. As we advance this program, we will continue to benefit from Roche’s scientific expertise in developing therapeutics for neurodegenerative conditions,” said B. Lynne Parshall, chief operating officer of Isis Pharmaceuticals.

The randomized, placebo-controlled, dose escalation Phase 1/2a clinical study will evaluate the safety and activity of ISIS-HTTRx in patients with early stage HD. In this study, ISIS-HTTRx will be administered intrathecally as an injection directly into the cerebral spinal fluid. Intrathecal administration of antisense drugs has been shown to be well tolerated in multiple clinical studies in patients.

“The initial development of this antisense drug for Huntington’s disease came out of a longstanding productive partnership between Isis and CHDI, and its advancement now to clinical trial is testament to Isis’ perseverance and scientific expertise,” said Robi Blumenstein, president of CHDI Management, which oversees the activities of CHDI Foundation, a nonprofit research organization exclusively dedicated to the development of therapies that will slow the progression of HD. “It’s exciting that therapeutic candidates grounded in the biology of Huntington’s disease are finally making their way to clinical trial.”

ABOUT ISIS and ROCHE

Roche and Isis are collaborating to develop antisense drugs to treat HD. The alliance combines Isis’ antisense expertise with Roche’s scientific knowledge in developing neurodegenerative therapeutics. With the initiation of the Phase 1/2a study for ISIS-HTTRx, Isis earned a $22 million milestone payment from Roche. To date, Isis has earned $52 million in upfront and milestone payments from its relationship with Roche and is eligible to earn additional milestone payments as the drug progresses in development, as well as royalties on sales of ISIS-HTTRx if it is commercialized. Roche has the option to license ISIS- HTTRx from Isis through the completion of the Phase 1/2a study. Prior to option exercise, Isis is responsible for the discovery and development of ISIS-HTTRx. If Roche exercises its option, it will assume responsibility for global development, regulatory and commercialization activities for the drug.

CHDI Foundation, Inc. provided financial and scientific support to Isis’ HD drug discovery program through a development collaboration with Isis. Over time, CHDI will be reimbursed for its support of Isis’ program out of milestone payments received by Isis.

ABOUT ISIS-HTTRx and Huntington’s Disease

ISIS-HTTRx is a Gen. 2.0+ antisense drug in development for the treatment of Huntington’s disease. ISIS-HTTRx is designed to reduce the production of all forms of the huntingtin (HTT) protein, which is the protein responsible for HD. As such, ISIS-HTTRx offers a unique approach to treat all patients with HD. HD is a rare genetic, progressive neurological disease resulting in deterioration in mental abilities and physical control. HD is referred to as a triplet repeat disorder, and is one of a large family of genetic diseases in which certain gene sequences are mistakenly repeated. In HD, the gene that encodes for the HTT protein contains a trinucleotide sequence that is repeated in the gene more than 36 times. The resulting HTT protein is toxic and gradually damages neurons in the brain. Symptoms of HD usually appear between the ages of 30 to 50 years, and continually worsen over a 10 to 25 year period.

Ultimately, the weakened individual succumbs to pneumonia, heart failure or other complications. Presently, there is no effective disease modifying treatment, and current approaches only focus on managing the severity of some disease symptoms.

ABOUT ISIS PHARMACEUTICALS, INC.

Isis is exploiting its leadership position in RNA-targeted technology to discover and develop novel drugs for its product pipeline and for its partners. Isis’ broad pipeline consists of 38 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer. Isis’ partner, Genzyme, is commercializing Isis’ lead product, KYNAMRO®, in the United States and other countries for the treatment of patients with homozygous FH. Isis has numerous drugs in Phase 3 development in severe/rare diseases and cardiovascular diseases.

These include volanesorsen, a drug Isis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with familial chylomicronemia syndrome and familial partial lipodystrophy; ISIS-TTRRx, a drug Isis is developing with GSK to treat patients with the polyneuropathy and cardiomyopathy forms of TTR amyloidosis; and ISIS-SMNRx, a drug Isis is developing with Biogen to treat infants and children with spinal muscular atrophy, a severe and rare neuromuscular disease. Isis’ patents provide strong and extensive protection for its drugs and technology. Additional information about Isis is available at www.isispharm.com.

ISIS PHARMACEUTICALS’ FORWARD-LOOKING STATEMENT

This press release includes forward-looking statements regarding Isis’ alliance with Roche, the development, activity, therapeutic potential, commercial potential and safety of ISIS-HTTRx. Any statement describing Isis’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Isis’ forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Isis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis’ programs are described in additional detail in Isis’ annual report on Form 10-K for the year ended December 31, 2014, and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.

In this press release, unless the context requires otherwise, “Isis,” “Company,” “we,” “our,” and “us” refers to Isis Pharmaceuticals and its subsidiaries.

Isis Pharmaceuticals® is a registered trademark of Isis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark of Isis Pharmaceuticals, Inc. KYNAMRO® is a registered trademark of Genzyme Corporation.

Isis Pharmaceuticals’ Contacts:

D. Wade Walke, Ph.D.
Vice President, Corporate Communications and Investor Relations
760-603-2741

Amy Williford, Ph.D.
Associate Director, Corporate Communications
760-603-2772

Post navigation

← 11th Annual CHDI HD Therapeutics Conference 2016 Video Postcard from 2015 HD Therapeutics Conference →
Connect with CHDI
Provide Feedback on this Page

Send Feedback

We appreciate any comments or questions on this page—our goal is to make this concise and helpful.

Join Our Mailing List
Terms of Use
Privacy Policy
Creative Commons License
Except where otherwise noted, content on this site is licensed under a Creative Commons Attribution 3.0 Unported License.
×

Join Our Mailing List

Receive spam-free updates from the Huntington's disease research community.
×

Terms of Use

  • These Terms of Use apply to your use of CHDI Foundation.org, all its sub-domains and all associated services including, but not limited to, e-mail received from CHDIFoundation.org, any of our computer systems, databases or networks connected to CHDIFoundation.org and all its sub-domains (collectively, the "Website"). The Website is being made available for your use by CHDI Foundation, Inc.

    If you browse or otherwise use the Website, you accept these Terms of Use. We reserve the right to modify these Terms of Use at any time by posting such change here. Your continued use of the Website after such modification is made constitutes your acceptance of, and agreement to be bound by, the Terms of Use as modified. If you do not agree with these Terms of Use, please do not access or use the Website.

    Limitations on Use
    We reserve the right, in our sole discretion, without any obligation or notice requirement, to suspend or deny your access to the Website for any reason, including for scheduled or unscheduled maintenance, upgrades, improvements or corrections.

    You may not attempt to gain unauthorized access to the Website, through hacking, password mining or any other means to circumvent the Website's security procedures. You may not use the Website in any manner that could damage, disable, overburden or impair the Website or any service provided through the Website, or interfere with any other user's use and enjoyment of the Website or any service provided through the Website. You may not make use of the Website or any service provided through the Website to forge e-mail headers or send bulk unsolicited e-mail messages.

    Privacy Policy
    These Terms of Use include our Privacy Policy, which describes how we use your personal information.

    Accuracy of Content
    Any content we provide or post on the Website may contain errors or inaccuracies, including both typographical and substantive errors. We reserve the right, in our sole discretion and for any reason, without any obligation or notice requirement, to discontinue, change, improve or correct the content we provide or post on the Website. Any dated content we provide or post on the Website is published as of its date only and we have no responsibility to update or amend any such content.

    It is your sole responsibility to evaluate the accuracy, completeness or usefulness of any content provided or posted on the Website.

    Links to Other Websites
    These Terms of Use apply only to the Website. We provide links to other websites, but we do not control the content on those websites or their practices. We are not responsible for other websites' content, information collection practices or use of any information they collect. Links to other websites do not constitute an endorsement by us of those websites or their content, owners or posters.

    Your Use of Content Contained on the Website
    The entire Website is copyrighted. Certain content, such as articles, you may find within the Website may also be separately copyrighted by us or by others.

    Except where otherwise expressly noted or contemplated, all content provided or posted on the Website is being made available to you for the purpose of Huntington's disease related research activities.

    Except where otherwise expressly noted or contemplated, we grant you a license to use any content we have provided or posted on the Website under a Creative Commons Attribution 3.0 License. Certain content we have provided or posted on the Website may also be subject to you agreeing to further terms of use specifically covering such content.

    You acknowledge and agree that the permission granted in this section does not constitute an endorsement by us of you or your use of any such content. Please contact us directly for copyright permissions other than those expressly granted in these Terms of Use.

    Disclaimer of Warranties, Not a Substitute for Medical Advice and Limitation of Liability?
    THE WEBSITE IS PROVIDED ON AN "AS IS" AND "AS AVAILABLE" BASIS. WE AND OUR AFFILIATES AND AGENTS MAKE NO REPRESENTATIONS, WARRANTIES OR CONDITIONS OF ANY KIND, EXPRESS OR IMPLIED, INCLUDING WARRANTIES OF MERCHANTABILITY, FITNESS FOR A PARTICULAR PURPOSE, NON-INFRINGEMENT OR OTHERWISE, AS TO THE OPERATION OF THE WEBSITE, OR THE ACCURACY, TIMELINESS OR COMPLETENESS OF CONTENT OR SERVICES INCLUDED ON THE WEBSITE. YOU EXPRESSLY AGREE THAT YOUR USE OF THE WEBSITE AND ANY CONTENT OR SERVICES INCLUDED ON THE WEBSITE IS AT YOUR SOLE RISK.

    ALL CONTENT INCLUDED ON THE WEBSITE, SUCH AS TEXT, TREATMENTS, DATA, DOSAGES, OUTCOMES, CHARTS, PATIENT PROFILES, GRAPHICS, PHOTOGRAPHS, IMAGES, ADVICE, MESSAGES, FORUM POSTINGS AND ANY OTHER CONTENT PROVIDED ON THE WEBSITE ARE FOR INFORMATIONAL PURPOSES ONLY AND ARE NOT A SUBSTITUTE FOR PROFESSIONAL MEDICAL ADVICE OR TREATMENT. YOU SHOULD ALWAYS SEEK THE ADVICE OF YOUR PHYSICIAN OR OTHER QUALIFIED HEALTH PROVIDER WITH ANY QUESTIONS YOU MAY HAVE REGARDING YOUR HEALTH. NEVER DISREGARD PROFESSIONAL MEDICAL ADVICE OR DELAY IN SEEKING IT BECAUSE OF SOMETHING YOU HAVE READ ON THIS WEBSITE. IF YOU THINK YOU MAY HAVE A MEDICAL EMERGENCY, CALL YOUR DOCTOR OR 911 IMMEDIATELY. WE DO NOT RECOMMEND OR ENDORSE ANY SPECIFIC TESTS, PHYSICIANS, PRODUCTS, PROCEDURES, OPINIONS, OR OTHER CONTENT THAT MAY BE MENTIONED ON THE WEBSITE.

    YOU EXPRESSLY AGREE THAT NEITHER WE NOR OUR AFFILIATES AND AGENTS ARE LIABLE FOR ANY DAMAGES OF ANY KIND ARISING OR RESULTING FROM YOUR USE OF THE WEBSITE OR ANY CONTENT OR SERVICES INCLUDED ON THE WEBSITE, INCLUDING, BUT NOT LIMITED TO, DIRECT, INDIRECT, INCIDENTAL, PUNITIVE, AND CONSEQUENTIAL DAMAGES.

    Copyright Policy
    We respect the intellectual property of others and we ask users of the Website to do the same. In accordance with the Digital Millennium Copyright Act ("DMCA") and other applicable law, we have adopted a policy of, in appropriate circumstances and at our sole discretion, terminating users of the Website who are deemed to be repeat infringers. We may also, at our sole discretion, limit access to the Website of any user of the Website who infringes any intellectual property rights of others, whether or not there is any repeat infringement.

    Procedure for Notifying Us of Claims of Copyright Infringement
    If you believe that any content provided or posted on the Website infringes upon any copyright which you own or control, or that any link on this Website directs users to another website that contains content or descriptions that infringes upon any copyright which you own or control, you may file a notification of such infringement with us as set forth below. Notifications of claimed copyright infringement must be sent to the attention of: CHDI Foundation, Inc., c/o CHDI Management, 350 Seventh Avenue, Suite 200, New York, NY 10001, Attention: David P. Rankin, Chief Legal Officer.

    Governing Law
    These Terms of Use shall be governed by and construed in accordance with the domestic laws of the State of New York, USA without giving effect to any choice or conflict of law provision or rule (whether of the State of New York, USA or any other jurisdiction) that would cause the application of the laws of any jurisdiction other than the State of New York, USA.

    CHDI Foundation, Inc. – www.CHDIFoundation.org – Terms of Use Revision No. 003 (Effective 11/01/2015)

    Download Terms of Use

  • ×

    Privacy Policy

  • This Privacy Policy applies to your use of CHDIFoundation.org, all its sub-domains and all associated services including, but not limited to, e-mail received from CHDIFoundation.org, any of our computer systems, databases or networks connected to CHDIFoundation.org and all its sub-domains (collectively, the "Website"). The Website is being made available for your use by CHDI Foundation, Inc.

    We are committed to protecting the privacy and security of your visits to the Website. This is our online Privacy Policy. If you have questions about this Privacy Policy, please let us know. If you do not agree with this Privacy Policy, please do not access or use the Website.

    We reserve the right to modify this Privacy Policy at any time by posting such change here. We encourage you to refer back to this page and review this Privacy Policy often for the latest information and the effective date of any modifications. If we decide to change this Privacy Policy, we will post a new policy on the Website and change the revision number and effective date at the bottom. Changes to this Privacy Policy will not apply retroactively. Your continued use of the Website after any such modifications are made constitutes your acknowledgement of, and agreement with, the Privacy Policy, as modified.

    Collection of Information and Use
    If you join our mailing list, we collect some information that can be directly associated with you. We call this information "Personal Information" and it includes your name, title and email address and the name of the company or research institution with which you are affiliated. You may modify your Personal Information at any time by following the update profile/email address links at the end of any email we send you.

    By joining our mailing list, you have granted us permission to use your Personal Information to send you emails ("opt-in") relating to our activities and Huntington's disease related research and information. If you have received an email from us, our records indicate that you joined our mailing list. Because we respect your time and attention, we make every effort to control the frequency of our emails.

    You may revoke the permission you have given us to use your Personal Information to send you emails ("opt-out") by following the instructions set out in our e-mails.

    Anti-Spam
    If you believe you have received unwanted, unsolicited email from us sent via the Website or purporting to be sent via the Website, please forward a copy of that email to info@CHDIFoundation.org.

    Privacy and Sharing of Your Personal Information
    It is our general policy not to make Personal Information available to anyone other than our personnel, website administrators, service providers and agents. We may share your Personal Information with our personnel, website administrators, service providers and agents that carry out certain functions on our behalf, such as website hosting, data processing and order fulfillment. Some of these personnel, website administrators, service providers and agents may be located in jurisdictions (including the United States) that may not have the same or as strict privacy laws as your country of residence. We require that our personnel, website administrators, service providers and agents comply with this Privacy Policy when processing or handling Personal Information on our behalf.

    We will not disclose your Personal Information for purposes other than those described herein without your prior consent except as permitted or required by law. In the event of a sale, amalgamation, re-organization, transfer or financing of some or all of our operations, your Personal Information may be disclosed to an acquiring organization, either as part of due diligence or on completion of the transaction. If Personal Information is disclosed in this context, we will require the acquiring organization to comply with this Privacy Policy in its processing and handling of such Personal Information.

    Security
    We maintain a variety of physical, electronic and procedural safeguards to protect your Personal Information. As mentioned above, it is our general policy to restrict access to Personal Information to our employees and agents.

    The Website may have links to other websites that we do not control. You should know that we have no control over the content, privacy policies or security of any of these websites you elect to visit or interact with. Furthermore, we are not responsible for the content, privacy policies or security of any of these websites you elect to visit or interact with and you should check those policies on such websites.

    Browser Information Collected on the Website

    We log IP addresses, which are the locations of computers or networks on the Internet, and analyze them in order to improve the utility of the Website. We also collect aggregate numbers of page hits in order to track the popularity of certain pages and improve the utility of the Website. We do not gather, request, record, require, collect or track any users' Personal Information through these processes.

    We use cookies on the Website. A "cookie" is a tiny text file that we store on your computer to customize your experience and support some necessary functions. We also use cookies to better understand how users use the Website. Our cookies contain no Personal Information and are neither shared nor revealed to other websites. We do not look for or at other websites' cookies on your computer.

    You also have choices with respect to cookies. By modifying your browser preferences, you can accept all cookies, be notified when a cookie is set, or reject all cookies (for more information on how to block or filter cookies, see http://www.cookiecentral.com/faq). However, if you reject some or all cookies, your experience at the Website may not be complete.

    Use of Web Beacons

    When we send emails to users of the Website, we may include a web beacon to allow us to determine the number of users who open our emails. When you click on a link in an email we have sent to you, we may record this individual response to allow us to customize our offerings to you. Web beacons collect only limited information, such as a cookie identifier, time and date of a page being viewed, and a description of the page on which the Web Beacon resides (the URL).

    Web Beacons can be refused when delivered via email. If you do not wish to receive Web Beacons via email, you will need to disable HTML images or refuse HTML (select Text only) emails via your email software.

    Contacting Us

    You have a right to request access to, and rectification of, your Personal Information by contacting us at info@CHDIFoundation.org. If you have any questions about this privacy policy or our Personal Information practices, you may likewise contact us at info@CHDIFoundation.org.

    CHDI Foundation, Inc. – www.CHDIFoundation.org – Privacy Policy Revision No. 003 (Effective 11/01/2015)

  • Click to read a letter from Robert Pacifici, CHDI CSO, regarding COVID-19