First HD patients enrolled in Roche/Genentech studies

Roche/Genentech announced today that they have enrolled the first patients in the HD Natural History and Phase III GENERATION HD1 clinical studies. The following press release gives an update on the RG6042 Huntington’s disease (HD) program.

28 January 2019

Update on RG6042 Huntington’s disease (HD) programme: First patients enrolled in HD Natural History and Phase III GENERATION HD1 clinical studies

Dear Global Huntington’s Community,

Just over one year ago, we and our partner Ionis Pharmaceuticals announced the results of thefirst ever study that tested the huntingtin-lowering scientific hypothesis. Today, we are pleased to announce that the first patient has entered into the GENERATION HD1 clinical study – a pivotal, global Phase III study to investigate the efficacy and safety of RG6042 (formerly known as IONIS-HTTRx). If this clinical study is successful, it is our hope that RG6042 will be approved by health authorities and made available for the treatment of manifest Huntington’s disease.

We are grateful to the patients and family members participating in clinical research, as well as everyone supporting them in the broader HD community. Scientific progress is only possible with your collaboration and participation.

Recent updates

  • Two global clinical studies have started, and first patients are enrolled – an important achievement for the research programme; study status is available on ClinicalTrials.gov.
  • HD Natural History study (NCT03664804)
    • This observational study for early manifest HD will run in the USA, Canada, Germany and the UK; planned study sites were announced in late 2018.
    • Initial sites have opened, and the first patients have enrolled; our team continues to work to open recruitment at all study sites as quickly as possible.
  • Phase III GENERATION HD1 study (NCT03761849)
    • We received health authority approvals in the USA and Canada to start this pivotal study for manifest HD. Planned study sites for those countries were announced at the end of 2018 and the first patient has now enrolled.This study will run in approximately 15 countries; we are diligently working to set up study infrastructure and receive approvals in the remaining countries.
    • Ongoing open-label extension of the Phase I/IIa study: sponsorship has been transferred from Ionis to Roche.
    • Patients who completed the Phase I/IIa study have been participating in an open-label extension study sponsored by Ionis (NCT03342053). Responsibility of this study has now transferred to Roche. Moving forward these patients will roll into a new Roche-sponsored, open-label extension study called GENEXTEND. GENEXTEND will allow us to continue to study longer-term effects of RG6042 in those participants who have previously completed a clinical trial for the investigational molecule.

Important to note

  • At this time, access to RG6042 is only through clinical studies because the benefits and risks of RG6042 are not yet fully understood.
  • Additional countries/sites for the Phase III study: Information about additional countries/sites will be announced on a progressive basis, once sites are nearly ready to enroll participants; information will be posted on ClinicalTrials.gov.
  • Communications about study data: Roche is committed to transparent and timely communications, as well as ensuring the integrity of ongoing clinical trial operations and data collection. In line with our Global Policy on Sharing of Clinical Study Information, we will share overall programme updates and relevant data from completed and ongoing clinical studies with the scientific community via appropriate channels (e.g., scientific meetings, peer-reviewed journals, etc.).

Our team recognises that the need in HD is greater than the capacity of the RG6042 development programme, and that not every person nor every capable HD centre interested in these clinical studies will be able to participate. We can assure you that the studies are designed to provide health authorities with the required data so that the benefit-risk of RG6042 can be determined as quickly as possible. The ultimate goal is that this investigational medicine can be approved by health authorities and made accessible to the broader HD community.

Our team continues to engage with health authorities and HD communities around the world on the RG6042 research programme. We look forward to providing you with further updates.

Sincerely,

Mai-Lise Nguyen, on behalf of the Roche & Genentech HD team

Patient Partnership Director, Rare Diseases

Roche Pharma Research & Early Development / Roche Innovation Centre Basel, Switzerland

Frequently asked questions and answers

What is the HD Natural History study?

This 15-month observational study aims to further understand the role of mutant huntingtin protein in disease progression, including how levels of mHTT change over time in the absence of any drug treatment. There is no drug treatment in this study. This study will include up to 100 participants with early manifest (Stage I and II) HD. For all patients who complete the HD Natural History study, an open-label extension study with the option of receiving RG6042 (no placebo control) is planned, pending eligibility, approval by Authorities and Ethics Committees/Institutional Review Boards and if data support the continued development of RG6042.

The HD Natural History Study will run at up to 17 sites in Canada, Germany, the United Kingdom and the United States. For more information about the study/trial sites visit ClinicalTrials.gov or contact the local Roche Medical Information team:

● Germany: (+49) 07624-14-2015

● UK: (+44) 0800 3281629 or medinfo.uk@roche.com

● Canada & United States of America: (+1) 888-662-6728

What is the Phase III GENERATION HD1 study?

The GENERATION HD1 study will evaluate the efficacy and safety of RG6042 treatment for manifest HD. The study will run over a period of 25 months (approx. two years). GENERATION HD1 is designed to determine whether RG6042 is safe and effective, and therefore includes a comparison to placebo. Participants will be randomised to one of three treatment study arms: RG6042 monthly, RG6042 once every two months (bi-monthly) or placebo monthly. This means for every two participants randomised to RG6042, one will receive placebo. The study is “double-blinded,” meaning neither the participant nor his/her investigator or site staff will know which study arm the participant is assigned.

For all patients who complete the GENERATION HD1 study, an open-label extension study with the option of receiving RG6042 (no placebo control) is planned, pending eligibility, approval by Authorities and Ethics Committees/Institutional Review Boards and if data support the continued development of RG6042.

The GENERATION HD1 study will enroll up to 660 patients with manifest HD at 80-90 sites in approximately 15 countries around the world. Planned sites have been announced for Canada and the United States of America. For more information about the study/trial sites, visit ClinicalTrials.gov or contact our Clinical Trial Information Support Line for the USA and Canada at (+1) 888-662-6728. Information about additional countries/sites involved in the study will be announced, as those details are finalised.

How are the clinical study sites selected?

A variety of factors influence site selection, including assessments on experience with HD studies, clinic infrastructure capacity to run the study as well as usual site activities, ability to operationalise the study as quickly and completely as possible, patient population, and geographic location.

What if there is not a study site near where I live? Can I relocate to participate in a study?

Clinical studies are subject to international, national and local laws and regulations. Additionally, factors such as institutional site policies, health insurance and travel burden may impact your ability to relocate and be accepted into one of the study sites. Eligibility and enrollment are decided by the study investigator at each site, who takes into account all these factors and may also wish to speak to you or your local HD specialist for more information. Whether your HD centre is selected for participation or not, this is no reflection on the quality of the many outstanding HD clinics and dedicated care providers around the world. The need in HD is greater than the capacity of our development programme. We have designed the programme to provide the required data to health authorities so that the benefits and risks of RG6042 can be determined as quickly as possible. Our ultimate goal is that this investigational medicine can be approved by health authorities, and made accessible to the broader HD community.

Can I access RG6042 outside of clinical studies?

Currently, access to RG6042 is only through clinical study participation because the benefits and risks of RG6042 are not yet fully understood. This means that we are not able to grant pre-approval, compassionate use or “right-to-try” requests at this time. As our understanding of the benefits and risks of RG6042 grows, we will regularly evaluate this position.

Your clinical studies are in early manifest and manifest HD. Will you study RG6042 in other patient populations (e.g., juvenile onset HD or prodromal HD)?

We recognise the critical medical need for a treatment for HD, especially for people living with severe forms like juvenile onset HD. Once there is sufficient scientific and safety rationale, our team will consult with HD community experts and explore the potential use of RG6042 in populations beyond manifest HD.

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uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-130 in Huntington’s Disease

uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-130 in Huntington’s Disease

~ AMT-130 Poised to Become First AAV Gene Therapy to Enter the Clinic for Huntington’s Disease ~

~ Lead Product Candidate from Company’s Proprietary miQURE™ Gene Silencing Platform   ~

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Jan. 22, 2019 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the U.S. Food and Drug Administration (FDA) has completed its review of the Company’s Investigational New Drug (IND) application for AMT-130, and the IND is now effective, allowing uniQure to begin its planned Phase I/II study. AMT-130 comprises a recombinant AAV5 vector carrying a DNA cassette encoding a microRNA that non-selectively lowers or knocks-down human huntingtin protein in Huntington’s disease patients.

“The FDA’s clearance of our IND for AMT-130 is a significant milestone for Huntington’s disease patients and an important event in the field of gene therapy,” said Matt Kapusta, chief executive officer at uniQure. “We expect that AMT-130 will be the first one-time administered AAV gene therapy to enter clinical testing for the treatment of Huntington’s disease, a devastating neurodegenerative disorder for which there is no approved disease-modifying treatment.

“AMT-130 also represents the first clinical-stage AAV-based therapy specifically designed to silence an abnormal gene in the brain with a single administration, and we believe our proprietary miQURE™ gene silencing platform has the potential to be applied to many other diseases, such as spinocerebellar ataxia type 3 (SCA3),” continued Mr. Kapusta. “This achievement is a major milestone for uniQure’s research organization, who have dedicated years of effort with the hope we can one day offer treatment for the many patients waiting generations for an effective therapy.”

FDA clearance of the IND enables uniQure to initiate its planned dose-escalating, randomized and controlled Phase I/II clinical trial to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington’s disease. uniQure expects to open several clinical sites in the United States and begin dosing patients in the second half of this year.  

About Huntington’s Disease
Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline, resulting in complete physical and mental deterioration. The disease is caused by an autosomal dominant mutation, CAG repeat expansion in the first exon of the huntingtin gene, that leads to the production of a mutated protein that aggregates in the brain. Despite the clear etiology of HD, there are no therapies available to treat the disease, delay onset, or slow progression of a patient’s decline.

About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and other severe genetic diseases. www.uniQure.com

uniQure Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, the achievement of any of our planned near term or other milestones, our ability to become the first AAV Gene therapy for Huntington’s Disease to begin clinical trials, our ability to initiate our planned dose-escalating, randomized and controlled Phase I/II clinical trial, our ability to open several clinical sites in the United States and begin enrolling patients in the second half of this year or ever, the development of our gene therapy product candidates, the ability to achieve therapeutic or curative effects in human patients in any of our product candidates, whether our proprietary miQURE™ gene silencing platform can be applied to any other diseases, such as spinocerebellar ataxia type 3 (SCA3), the ability to produce a product candidate that is safe and effective, the ability to obtain regulatory approval for any of our product candidates, and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaboration activities, product development activities, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading “Risk Factors” in uniQure’s Annual Report on Form 10-K filed on March 14, 2018 and Quarterly Report on Form 10-Q filed on November 6, 2018. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

uniQure Contacts:

FOR INVESTORS:       FOR MEDIA:
         
Maria E. Cantor   Eva M. Mulder   Tom Malone
Direct: 339-970-7536   Direct: +31 20 240 6103   Direct: 339-970-7558
Mobile:  617-680-9452   Mobile: +31 6 52 33 15 79    Mobile:339-223-8541
m.cantor@uniQure.com    e.mulder@uniQure.com    t.malone@uniQure.com
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You Pay the Higher Cost

You Pay the Higher Cost

by Sharon Quigley

Caregivers of people with Huntington’s disease often feel lonely, even when they’re with their loved ones. Sharon Quigley uses poetry to express the loneliness she feels even when she’s with her husband, Ken. While recognizing that the disease is much more costly for the person with HD, she knows that Huntington’s affects both the caregiver and the person with HD.

Ken and Sharon Quigley, attending a wedding last summer

I wish that there was more of you
Just like there used to be,
But now I feel so lonely
Even when you’re next to me.
 
I look so deep into your eyes
And it looks like you are lost,
I know between the two of us, 
You pay the higher cost.
 
Remember times we’d sit and talk
Of some current world affair?
Now when I try to speak to you
All I get is your despair.
 
We used to sit along the beach
And watch the world go by
We’d live forever, you and I,
Never thinking we would die.
 
Many years have come and gone
And still our love is strong
But I don’t feel you here with me,
Something is very wrong.
 
Now when we sit and watch tv
I feel a chill and frost
I know between the two of us, 
You pay the higher cost.
 
So many times, I feel alone
And yet, there you sit.
I wish that there was more of you
I miss your charm and wit.
 
The disease, you see, affects us both.
At times, we are both lost,
But I know between the two of us, 
You pay the higher cost.

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