Announcements

Raptor Pharmaceutical Receives FDA Approval of Expanded Label for PROCYSBI® to Treat Children Aged 2-6 Years with Nephropathic Cystinosis

NOVATO, Calif., August 17, 2015 – Raptor Pharmaceutical Corp. (NASDAQ: RPTP) today announced that the U.S. Food and Drug Administration (FDA) approved the expanded use of PROCYSBI® (cysteamine bitartrate) delayed-release capsules to treat children two to six years of age with nephropathic cystinosis. The approved supplement was based on efficacy and safety data from an ongoing long-term extension study in which a cohort of children aged 2 to 6 years were enrolled and treated with PROCYSBI for 12 months. Additionally, data submitted as part of this supplement supported the longterm maintenance of white blood cell cystine levels and renal function in all age groups studied during extended treatment with PROCYSBI. PROCYSBI is now approved for the treatment of nephropathic cystinosis in adult and in pediatric patients 2 years of age and older in the U.S. Please see the full prescribing information at www.PROCYSBI.com.

“Strict adherence to cystine depleting therapy from as early an age as possible is critical to maintaining kidney function and leading longer, healthier lives for patients with cystinosis,” said Craig Langman, M.D., The Isaac A. Abt, M.D. Professor of Kidney Diseases, Feinberg School of Medicine and Head of Kidney Diseases at Ann & Robert H. Lurie Children's Hospital of Chicago and the lead investigator on the extension trial. “The data in the two to six year old group confirm the maintenance of stable kidney function over time. Not only will this help these young patients with cystinosis, but the 12 hour dosing schedule for PROCYSBI could be a significant benefit to the caregivers of these young children.”

“We are extremely pleased that younger patients and families living with cystinosis will now have access to PROCYSBI,” said Krishna Polu, M.D., Chief Medical Officer of Raptor. “We appreciate the support of the cystinosis community, families, caregivers, and patients who participated in the trials that led to the initial and expanded approval of PROCYSBI. We remain committed to advancing the science and understanding of cystinosis to help patients and caregivers take more control of their lives.” "Caregivers of younger individuals with cystinosis will now have another treatment option to discuss with their medical professional teams,” stated Jeff Larimore, President of the Cystinosis Research Network. “Every opportunity provided to improve treatment adherence is applauded by the cystinosis community.”

PROCYSBI was initially approved in 2013. Physicians can prescribe PROCYSBI by calling RaptorCares at 1-855-888-4004. RaptorCares provides individualized services to help patients access PROCYSBI through education, support, extensive case management and a commitment to the principle that no eligible U.S. patient with nephropathic cystinosis will be denied access to PROCYSBI based on inability to pay.

About Nephropathic Cystinosis
Nephropathic cystinosis comprises 95% of diagnosed cases of cystinosis, a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body. Elevated cystine leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death. Nephropathic cystinosis is typically diagnosed in infancy and requires lifelong therapy.

Strict adherence to therapy is crucial to improve outcomes, as even brief delays in cysteamine dosing may allow cystine to return to toxic levels. Left untreated, the disease is usually fatal by the end of the first decade of life. There are an estimated 500 patients living in the United States with cystinosis, and 2,000 worldwide.

About PROCYSBI® (cysteamine bitartrate) delayed-release capsules PROCYSBI is the first cystine depleting agent that can be given every 12 hours that is approved in the U.S. for the management of nephropathic cystinosis in adults and children 2 years of age and older. It is contraindicated in patients with a hypersensitivity to cysteamine or penicillamine. The most commonly reported side effects are vomiting, nausea, abdominal pain, breath odor, diarrhea, skin odor, fatigue, rash, and headache. For additional information on PROCYSBI, including full prescribing information, please visit www.procysbi.com.


About Raptor Pharmaceutical
Raptor Pharmaceutical Corp. is a global biopharmaceutical company focused on the development and commercialization of life-altering therapeutics that treat rare, debilitating and often fatal diseases. The company is engaged in multiple therapeutic areas such as nephropathic cystinosis, Huntington's disease (HD), pediatric nonalcoholic steatohepatitis (NASH), and mitochondrial diseases including Leigh syndrome. Raptor holds several orphan drug designations, including orphan drug exclusivity for nephropathic cystinosis in the U.S. and EU, and orphan drug designation for HD in the U.S. and EU. Raptor holds intellectual property for the use of cysteamine in HD and other neurodegenerative disorders including Parkinson's disease and Rett syndrome. For additional information, please visit www.raptorpharma.com.


COMPANY CONTACT:
Kimberly Lee, D.O.
Vice President, Corporate Strategy and Communications
(415) 408-6351
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INVESTOR CONTACT:
Robert H. Uhl
Managing Director
Westwicke Partners, LLC
(858) 356-5932
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MEDIA CONTACT:
Cammy Duong
Canale Communications
(619) 849-5382
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Rare Disease Artists’ Work to Travel Globe to Raise Awareness Art Showcase Debuts to Record Attendance

FOR IMMEDIATE RELEASE
Contact: Christy Greeley
Executive Director
Phone: 847-204-6004
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.
Website: www.cystinosis.org

Lake Forest, IL – August 11, 2015 – The Cystinosis Research Network (CRN), a 501(c)(3) charitable organization, created the first-ever “Dream, Achieve, Inspire” art exhibit prepared entirely by patients with cystinosis, a rare, genetic-metabolic disease. Paintings, poems, sculptures, dance, and photography from 106 individuals with cystinosis covering 30 countries came together under Exhibit Chairperson, Katie Larimore. Global representation was an ambitious but important goal for Mrs. Larimore. She said, “Our goal is to showcase the amazing talent in our community as well as raise awareness about cystinosis through art from around the world. In the United States, we are searching for a cure but in some countries the most current treatments are not available. Medicine accessible to U.S. patients for 25 years is unattainable. Those children are sent home to die. To me, that is unacceptable!”

art group
A group photo from the 2015 Cystinosis Research Network Family Conference in Chicago. The "Dream, Acheive, Inspire" art exhibit helped attract record-breaking attendance.

The art exhibit opened in Chicago at the 2015 CRN Family Conference July 16th – July 18th. The result was a record-breaking 340 attendees, the largest turn out in conference history. In addition to the art showcase, the CRN conference featured comprehensive discussion panels, access to ‘best in class’ medical professionals, stress-free entertainment, and a Cystinosis Teen Adventure. Group President, Jeff Larimore, categorized the event a success by saying, “…all in attendance were provided an opportunity to better capture the challenges of cystinosis through exchanges between peers living with cystinosis, dialogue between caregivers and professionals, listening to the transitioning generation and the sharing of emotions and inspirations though the art exhibit.”

The Chicago debut of the “Dream, Achieve, Inspire” collection is the beginning of a world tour for the art pieces. Mrs. Larimore has booked the exhibit for cystinosis gatherings throughout the year in Mexico, Brazil, and Europe with more destinations being added. For information on how to view the artwork, donate, volunteer, or become part of the Cystinosis Research Network, visit www.cystinosis.org.

Cystinosis is a rare, genetic, metabolic disease impacting approximately 2,000 people worldwide. Cystinosis causes an amino acid, cystine, to accumulate in the kidneys, eyes, liver, muscles, pancreas, brain, and white blood cells. Without treatment, children with cystinosis can develop end stage kidney failure at approximately age nine. This disease has devastating outcomes when untreated. The CRN is comprised of family, friends, and medical researchers dedicated to finding a cure, developing treatments, and enhancing the quality of life for those affected by cystinosis.

Media

For more information, contact Christy Greeley at This email address is being protected from spambots. You need JavaScript enabled to view it. or 847-204-6004 or Katie Larimore at This email address is being protected from spambots. You need JavaScript enabled to view it. or 803-873-2139. Follow up interviews and additional photographs available. You can find the CRN on Facebook at www.facebook.com/CystinosisResearch, Twitter @CystinosisCRN, Instagram @CystinosisResearchNetwork, and www.cystinosis.org.

“Dream, Achieve, Inspire” International Artwork Showcase Unveils Rare Disease Patients’ Creativity

Chicago Hosting Cystinosis Research Network Conference Mid-July

FOR IMMEDIATE RELEASE
Contact: Christy Greeley
Executive Director
Phone: 847-204-6004
Email: This email address is being protected from spambots. You need JavaScript enabled to view it.

Lake Forest, IL – June 11, 2015 – The Cystinosis Research Network (CRN), a Chicagoland-based 501(c)(3) charitable organization, is hosting its family conference in Chicago, Illinois from July 16 to July 18, 2015. This summer’s event features the first global gathering of art from over 100 artists from 30 countries who are affected with cystinosis, a rare, genetic-metabolic disease. Chicago is the first stop for the “Dream, Achieve, Inspire” art show with cystinosis support groups in Brazil, Mexico, Spain, and the United Kingdom lining up to share the unprecedented collection.

miriam narvaez

Miriam Narvaez from Mexico poses with her painting included in the “Dream, Achieve, Inspire” exhibit. She was diagnosed with cystinosis at 10 months old.

“The exhibit is designed to bring artists with cystinosis together to see each other as beautiful and creative. The worldwide participation has been overwhelming!” says Katie Larimore, Exhibit Chairperson.

The biennial event unites cystinosis families and leading experts, doctors, and scientists to discuss critical topics from medical research and management techniques to social challenges and insurance coverage. In addition to these essential conversations, raising cystinosis awareness and advancing access to life-changing treatment is top of mind. Mrs. Larimore said, “In countries such as Iraq, Venezuela and Thailand we hope to gain recognition for the patients there and make vital medicines available for the first time.”

lee knaus

Floridian, Lee Knaus, is an artist by trade. He is living with cystinosis and aspires to have his own art studio.

Cystinosis is a rare, genetic, metabolic disease impacting approximately 2,000 people worldwide. Cystinosis causes an amino acid, cystine, to accumulate in the kidneys, eyes, liver, muscles, pancreas, brain, and white blood cells. Without treatment, children with cystinosis can develop end stage kidney failure at approximately age nine. This disease has devastating outcomes when untreated. The CRN is comprised of family, friends, and medical researchers dedicated to finding a cure, developing treatments, and enhancing the quality of life for those affected by cystinosis.

Event Details
Who: Artists with rare disease called cystinosis
What: Travelling art exhibit, “Dream, Achieve, Inspire” debuting at the Cystinosis Research Network Family Conference
Where: Chicago, Illinois. The Magnificent Mile Doubletree by Hilton
When: July 16- 18, 2015

Media
For more information, contact Christy Greeley at This email address is being protected from spambots. You need JavaScript enabled to view it. or 847-204-6004. Media passes, follow up interviews, and photo opportunities available. You can find the CRN on Facebook at www.facebook.com/CystinosisResearch, Twitter @CystinosisCRN, Instagram @CystinosisResearchNetwork, and www.cystinosis.org.

The 2015 CRN Newsletter Has Arrived

This newsletter catches up on the latest CRN happenings, including information on the CRN Family Conference: “Winds of Change” to be held July 16 – 18 in Chicago. As our community grows, this event provides a unique opportunity to bring everyone together to discuss the current and future outlook of our organization and to address questions and concerns from all of you in person.

Also featured in the newsletter is a personal message from Jeff Larimore, president of CRN – as well as several other members of the CRN community. Comprehensive financial, fundraising, and research updates are also included.

To access the full newsletter, please visit our newsletter page or download it by clicking here.

2015 Cystinosis Research Network Call for Proposals

Applicants may now submit proposals for the 2015 CRN Call for Research Proposals.

Research proposals may be submitted to CRN for review and consideration. CRN utilizes a Scientific Review Board comprised of leading experts on the disease of cystinosis which reviews grant proposals and submits funding recommendations to the Cystinosis Research Network. More specifically, the Scientific Review Board provides independent, objective review and recommendations regarding each research proposal utilizing grant review guidelines established by CRN and in accordance with the mission of the organization. The Chairperson of the Scientific Review Board summarizes its recommendations and presents them to the Cystinosis Research Network which then votes on each proposed project.

Priority is given to interventional research, both clinical and basic, that will lead to improved treatments for cystinosis. New investigators are particularly encouraged to apply. CRN has a strong interest in funding projects related to advancing Newborn Screening for Cystinosis – applications regarding this subject will have priority.

Applicants must submit an electronic copy of their proposal to:

Christy Greeley, Vice President for Research This email address is being protected from spambots. You need JavaScript enabled to view it.

Submission deadline is May 31, 2015. Proposals will be reviewed in July and notification will be made to applicants in August.

Detailed information on the grant submission process can be found on the CRN website at: https://cystinosis.org/research/grant-guidelines

Bonner Paddock to speak at CRN 2015 Family Conference in Chicago

Bonner Paddock is the Founder of the OM Foundation (OMF), which promotes the idea that any one person can make a difference if they dedicate themselves to look beyond their own personal limits to achieve their goals. Bonner is the first person with Cerebral Palsy to ascend Mt. Kilimanjaro and to finish the infamous Ironman World Championship unassisted. He has since raised over $1 Million Dollars to help build and support early learning centers in the US & Africa, which provide therapy for special needs children.  He has also recently launched his first book,One More Step, a memoir about his life living with Cerebral Palsy, conquering the incredibly challenging physical feats described above, and starting his own foundation.  At this year’s Cystinosis Research Network Family Conference, Bonner will share his inspirational story about what held him back for 30 years and how he pushed beyond his own limits to achieve success.  His book, “One More Step”, will be available for all conference attendees.

One More Step

one-more-stepFor 29 years, Bonner Paddock was in denial of his cerebral palsy-until he met Jake Robert. Bonner befriended the young boy, who suffered from severe CP, and his family while participating in his first half-marathon to raise funds for CP research, and was incredibly moved watching Jake be carried across the finish line. That night, Jake passed away.

In the wake of his loss, Bonner and the Robert family formed an extraordinary bond, built on the same unconditional love Jake gave every day of his life. Heartened by their support, Bonner finally had the push he needed. He would live life to the fullest in Jake's honor, accomplishing astounding physical feats and starting a foundation for disabled children along the way.

 

 

Bonner-and-Ashley-in-her-Hart-Walker

Bonner and Ashley in her Hart Walker