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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FDA Writes Back

Voices from all across the United States are being heard by the Food and Drug Administration (FDA) in Silver Spring, Maryland.

Help 4 HD International issued a call for action to the Huntington’s disease community on January 28 of this year. We asked as many people as possible to send a letter to the FDA addressing three big questions:

  • Why is there only one treatment approved for Huntington’s disease?
  • Why are clinical trials moving to other countries?
  • Does the FDA understand that time is something we simply don’t have?

We provided a pre-written letter on our web site and invited people to download the letter, add to it or change it or leave it as is, and mail it to the FDA.

Hundreds of people responded to our call, and the FDA is listening. Those of us who sent letters began receiving responses this past week, and the responses are promising. Though the letters appear to be computer-generated, they speak directly to our concerns. In part, the letter I received reads:

Your letter serves as a powerful reminder of the kind of strength it takes patients and their families to battle Huntington’s disease every day.

We understand that Huntington’s disease is a hereditary, debilitating disease with many physical and emotional symptoms that have devastating impacts on patients and their families. We also recognize the critical unmet medical need for new safe and effective treatments for Huntington’s disease.

FDA is committed to working with drug companies and the Huntington’s disease community to facilitate the development of new drugs to treat this devastating disease. We are prepared to use all expedited development and approval pathways available to us to further this mutual goal.

Thank you to everyone who sent letters, and a special thank you to WeHaveaFace.org for partnering with us in furthering our mission and to CIRM (California Institute of Regenerative Medicine) for helping publicize it. We are hopeful that this marks the beginning of a new chapter in the relationship between the Huntington’s/Juvenile Huntington’s disease community and the FDA. We’ll be keeping a close watch to see what happens with current and new clinical trials! For now, it’s important for all of us to realize that our voices CAN make a difference!

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The Patient Advocate: Celebrating the Season of Giving

The Patient Advocate: Celebrating the Season of Giving

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by Katie Jackson, President/CEO, Help 4 HD

I would like to begin this article with some of my favorite quotes about being a patient advocate:

  • “Advocacy: To change ‘what is’ to ‘what should be’”
  • “Strong people stand up for themselves. But the strongest people stand up for others.”
  • My final quote is one that was introduced to me by one of the strongest patient advocates I know, Judy Roberson. This quote is used by Judy every time she speaks publicly, and because of her, it is now used by many HD advocacy groups. “Never doubt that a small group of thoughtful, committed people can change the world. Indeed, it is the only thing that ever has.”—Margaret Mead

Whenever I think of the power of the patient advocate, I think about the polio epidemic. This is an event that I have talked about for years when I speak about what a group of patient advocates can accomplish. What Franklin D. Roosevelt, Basil O’Connor, and Eddie Cantor, along with all the powerful advocates, did to combat the polio virus was astounding. A vaccination for the polio virus was the result of their efforts. The world was forever changed because of the March of Dimes advocates.

I started my journey as a patient advocate over ten years ago. I quickly learned that to be a patient advocate, you have to find a cause you are deeply passionate about. One that you can feel deep in your soul that brings out a power in you that you didn’t know you had. You must be passionate, compassionate, persistent, patient, resilient, and strong. You will endure disappointment and pain when your countless hours of work don’t go the way you planned.

The patient advocate knows what I am talking about, but the passion and strength we have make it so that we “won’t back down,” and we continue to fight and push on, even when things don’t go our way. We don’t have time to cry over a blow; there is too much work that needs to be done and too many people that are counting on us, so we push on.

There are those brilliant moments when you work so hard on a project, and it succeeds, and the change happens right before your eyes. These moments are what it is all about—to know all the work, blood, sweat, and tears have made a difference for the families you are serving. There is no better feeling in the world for a patient advocate than when you get to see the world change a little because of your efforts.

The patient advocacy spirit within the Huntington’s disease community is strong. This is a family disease, and families have been advocating for years and years and generation after generation. I have had the honor of meeting HD patient advocates who have been fighting for over 30 years. Being a patient advocate myself, I am here to tell you that at times it is exhausting. That is why I am always in awe when I get to meet the “pioneer” patient advocates. Their persistence and strength to keep going after so many years is truly incredible. Although there are still no therapies and no cure for our loved ones living with HD/JHD, can you imagine where we would be if it weren’t for those early advocates who fought so hard for our families to get support, care, and to find the gene that has affected families for hundreds of years?

Without the patient advocate, change wouldn’t happen.

I am so excited that we are using the month of December for “Help 4 HD Live!” to highlight the patient advocate. They call this holiday season the “season of giving,” and I can’t imagine a person in this world who is more giving than a patient advocate. They are truly the most selfless people walking this earth, fighting hard, not for themselves, but for those who can no longer fight for themselves.

We have decided to name the series of shows we’re doing in December “Celebrating the Season of Giving.” I would like to share with you a little about the four advocates we will be interviewing and a little about what they have done and are currently doing to advocate for our HD community.

 

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Lauren Holder, North Carolina

Lauren is an incredible advocate and person. Lauren has been advocating for our Huntington’s disease community for 10 years now. She has hosted many Huntington’s disease fundraisers. She started her advocacy journey by going to Washington, DC, twice a year to speak on behalf of the Huntington’s Disease Parity Act. She started educating law enforcement after her father went missing for 12 hours, and she was unable to get any help, due to the lack of knowledge about HD.

She has worked with NAMI (National Alliance for Mental Illness) to get CIT (Crisis Intervention Training) in North Carolina for law enforcement. Lauren was the HDSA North Carolina president for many years before she had to step down to help care for her father. In 2004, she was honored with the HDSA Person of the Year award. She has written a book about living with Huntington’s disease and is in the process of composing her second novel.

Recently, Lauren and her father’s story was featured in an article in Good Housekeeping. Lauren is a powerhouse, and we are lucky to have her voice telling her story and educating people about what it is like to live with Huntington’s disease.

 

havanna-lowes

Havanna Lowes, Missouri

 Havanna is one of the most brilliant 17-year-olds I have ever met. Havanna visited Washington, DC, for the first time in June of last year. There, she was personally able to speak to four Congressional staff members about HD and the parity act. Havanna spoke to her senator from Missouri, and he signed the parity act in July.

Havanna has spoken at many education days. Just recently, she spoke at the Huntington Study Group meeting in Nashville in November. She is passionate about Huntington’s disease youth programs like NYA (National Youth Alliance) and HDYO (Huntington’s Disease Youth Organization). She has held fundraisers and has given talks about HD to her community.

Havanna says, “Advocacy is about telling your story to your community and getting people involved and informed.” Havanna is an amazing young lady who I believe will go so far in this world and will continue to be a champion for HD.

 

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Marie Clay, New York

Marie Clay has been a Huntington’s disease advocate for 20 years. Marie has educated many people about HD over the years. She has done law enforcement HD education, CIT, firefighter and first responder training, hospital training, and much more. She has spoken to many community organizations, like the Rotary Club, about HD.

Marie has been on Capitol Hill many times to speak up for the parity act, and recently, in September 2015, Marie spoke on a panel at the FDA (Food and Drug Administration) Patient Focus Meeting. Marie was able to tell members of the FDA about the challenges living with Huntington’s disease.

In 2008, Marie started a support group in Virginia Beach and has held many fundraising events.  Marie worked very closely with Sitrin, a care facility in New York state that now has a unit dedicated to HD/ALS. She has introduced many families to Sitrin, and everyone’s hope is that one day we will have more options like Sitrin available to our HD community.

Marie is one of only two employees that have been honored twice with the Volunteer of the Year award by GEICO Insurance. Marie has been fighting for HD families for many years, and we are thankful for all she has done for the HD/JHD community.

 

kinser-cancelmo

Kinser Cancelmo, Massachusetts

Kinser became more involved with both HD and JHD after her daughter Meg passed from JHD after spending four months in the hospital, with doctors unsure about how to help her. Kinser lost her husband shortly after she lost her daughter.

While Meaghan was in the hospital, Kinser enlisted local news teams and radio stations to do programs to raise awareness for JHD.  The local radio station had Kinser on their morning talk show to speak about JHD.

When Kinser lost her daughter and her husband just a couple of months apart from each other, she decided it was time to work on starting a nonprofit business in memory of her daughter, “Meg’s Fight 4 a Cure, Juvenile Huntington’s Disease Foundation, Inc.”  Kinser says, “I was appalled at the lack of medical services and places available for children/teens her age that had illnesses that the medical community couldn’t handle.  Especially JHD.  I wanted to raise money for researchers to continue working on medicines and hopefully a cure for this horrible disease.  A group of my friends, who are now Board members of my business, began working on a fundraiser to raise these funds to send out to UC Davis in California.  The fundraiser was held in September 2016, raising more than $20,000.”

Kinser is a board member of her local Massachusetts Chapter of HDSA.  She is involved in attending meetings quarterly and helping with the HD walks around the state, Education Days, as well as many other events that the chapter sponsors.

Kinser recently took Meaghan’s service dog, Dixie, and finished putting her through a pediatric training therapy class. Dixie passed the test so that she is now an official K-9’s for Kids therapy dog.

Kinser is an incredible woman. One can only imagine how immensely hard it would be to lose your husband and your little girl only two months apart from each other. Kinser is a survivor and is taking what she has learned to help others. We can’t wait to see what the future holds and the changes that will be made because of the “Meg’s Fight 4 a Cure, Juvenile Huntington’s Disease Foundation.”

Celebrating All the Patient Advocates

Help 4 HD International would like to thank all the Huntington’s disease patient advocates out there. Without you, change will not happen. So keep fighting, my friends, for the next generation and all the generations to come. Let’s hope that one day our efforts become a paragraph in a history book of “how they used to live before they found the cure for Huntington’s disease.”

 

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