RARE DISEASES NEWSLETTER
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01 RARE DISEASES NEWSLETTER Volume 5, January 2017 EDITION HIGHLIGHTS 01. Fourth quarter highlights and 2016 recap 02. Disease Spotlight: Primary Biliary Cholangitis and Primary Sclerosing Cholangitis 03. Where We've Been & Where We're Going WELCOME TO THE FIFTH EDITION OF THE RARE DISEASES NEWSLETTER! This quarterly publication will keep you up to date on PRA’s rare disease team, experience, achievements, and initiatives. 2016 was a fantastic year for PRA, and a year filled with significant growth and accomplishments for the Rare Disease Team. Sarah Rose and Sravan Jaggumantri joined our team as Clinical Scientists in July, and their contributions have helped us exceed our goals for 2016. Beginning with our new strategic partnership with Global Genes, and including new preferred provider relationships with Ultragenyx and LFB, we have firmly established PRA as the leader in rare disease clinical development. We continue to collaborate with our PRA colleagues, research consortia, patient advocacy groups, and other rare disease partners to create innovative solutions to executing rare disease studies. Look for several new initiatives from us in 2017, additional preferred partnership relationships, and a commitment to innovation as we strive to bring new therapies to rare disease patients faster. FOURTH QUARTER HIGHLIGHTS AND 2016 RECAP PRA’s Rare Disease Experience: Last 5 years PRA RARE DISEASE STUDY EXPERIENCE (N = 189) Oncology | 39 Immunology | 22 Hematology | 69 Ophthalmology | 3 Cardio-Metabolic Diseases | 5 Hepatology | 5 Neurology | 15 Endocrinology | 10 Musculoskeletal | 10 Respiratory | 3 Hereditary Disorders | 8 PRA’s Rare Disease Drug Approvals Our expertise in executing rare disease trials has led to the approval of 16 drugs across 5 therapeutic areas. We are proud to have supported these trials and look forward to seeing future approvals for the many therapeutic areas that encompass the unmet need of our rare disease patients.
Transcript of RARE DISEASES NEWSLETTER
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RARE DISEASES NEWSLETTERVolume 5, January 2017
EDITION HIGHLIGHTS01. Fourth quarter highlights and 2016 recap
02. Disease Spotlight: Primary Biliary Cholangitis and Primary Sclerosing Cholangitis
03. Where We've Been & Where We're Going
WELCOME TO THE FIFTH EDITION OF THE RARE DISEASES NEWSLETTER! This quarterly publication will keep you up to date on PRA’s rare disease team, experience, achievements, and initiatives.
2016 was a fantastic year for PRA, and a year filled with significant growth and accomplishments for the Rare Disease Team.
Sarah Rose and Sravan Jaggumantri joined our team as Clinical Scientists in July, and their contributions have helped us exceed our goals for 2016. Beginning with our new strategic partnership with Global Genes, and including new preferred provider relationships with Ultragenyx and LFB, we have firmly established PRA as the leader in rare disease clinical development. We continue to collaborate with our PRA colleagues, research consortia, patient advocacy groups, and other rare disease partners to create innovative solutions to executing rare disease studies.
Look for several new initiatives from us in 2017, additional preferred partnership relationships, and a commitment to innovation as we strive to bring new therapies to rare disease patients faster.
FOURTH QUARTER HIGHLIGHTS AND 2016 RECAP
PRA’s Rare Disease Experience: Last 5 years
PRA RARE DISEASE
STUDY EXPERIENCE
(N = 189)Oncology | 39
Immunology | 22
Hematology | 69
Ophthalmology | 3
Cardio-Metabolic Diseases | 5
Hepatology | 5
Neurology | 15
Endocrinology | 10
Musculoskeletal | 10
Respiratory | 3
Hereditary Disorders | 8
PRA’s Rare Disease Drug ApprovalsOur expertise in executing rare disease trials has led to the approval of 16 drugs across 5 therapeutic areas. We are proud to have supported these trials and look forward to seeing future approvals for the many therapeutic areas that encompass the unmet need of our rare disease patients.
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RARE DISEASES NEWSLETTER | Volume 5, January 2017
P R A participated in the pivotal or supportive trials and/or key NDA support services that led to approval of 16 important rare disease drugs currently on the market.
2 I M M U N O L O G YO N C O L O G Y6 H E M A T O L O G Y5
N E U R O L O G Y1C A R D I O - M E T A B O L I C2
Pediatric Collaboration Team announcement:The Rare Disease Team is pleased to partner with PRA's newly formed Pediatric Collaboration Team (PCT) led by Mark Sorrentino, VP of Scientific Affairs. The PCT will serve as an internal resource to support ongoing pediatric projects, extend relationships with existing clients, and support the acquisition of new business. As a multi-disciplinary and cross-functional team, the central goal of the group will be to maintain our internal knowledge of the current regulatory landscape while staying abreast of and anticipating relevant changes in pediatric drug development. Despite current regulatory guidelines in place, only 30% of the drugs used in children have undergone a randomized clinical trial.
We believe our teams are especially well aligned as it is estimated that approximately half of the people affected by rare diseases are children and 30% of these children will not live to see their 5th birthday (https://globalgenes.org/rare-diseases-facts-statistics/). Sarah Rose, Rare Disease Clinical Scientist, will serve as a liaison between our teams and will support the important goals of this internal group.
DISEASE SPOTLIGHT: PRIMARY BILIARY CHOLANGITIS AND PRIMARY SCLEROSING CHOLANGITISPrimary Biliary Cholangitis (PBC) and Primary Sclerosing Cholangitis (PSC) are two rare disorders affecting liver function that despite ongoing research, continue to present an area of significant unmet medical need. Both disorders affect the release of bile from the liver, which is an important component of digestion. Cholestasis, the inability of bile to flow into the small intestine, directly damages the liver and leads to inflammation, fibrosis, and eventually cirrhosis. Patients affected by these disorders may experience fatigue, abdominal pain, jaundice, pruritus, and depending on disease progression may necessitate liver transplantation. Of note, these are distinct disorders and females are 9 times more likely than men to develop PBC, which appears to have a genetic component. Males are more often affected by PSC which appears to have an autoimmune component and is often seen in individuals with inflammatory bowel disease.
Within the last 5 years, PRA has participated in over 20 hepatology studies. PRA has positioned itself as the expert in rare hepatology having conducted 2 PBC and 3 PSC studies. These studies span a variety of phases, mechanisms of actions, and countries. Sravan Jaggumantri, PRA Rare Disease Clinical Scientist, leads our rare hepatology efforts and has formed strong relationships with Key Opinion Leaders in the space. Breaking News in Rare Diseases: New SMA therapy approvedThe 2016 Q3 newsletter highlighted Spinal Muscular Atrophy, a rare, genetic neurodegenerative disease. We are proud to report the first and only approved drug for SMA in the US! The FDA approved Ionis/Biogen’s Spinraza (Nusinersen) on Dec 23, 2016!
This historic approval brings great hope to the SMA community and serves as a testimony to all the patients and families who contributed to the clinical trial process. With around 20 other drugs being developed for the treatment of SMA, this approval is a key milestone in a highly competitive landscape. The multi-system nature of this disease necessitates multidisciplinary medical care and we hope to see more drug approvals in the horizon for this devastating disease.
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FOURTH QUARTER HIGHLIGHTS
SCOTT SCHLIEBNERDirect: +1 [email protected]
SRAVAN JAGGUMANTRIDirect: +1 [email protected]
WHERE WE'VE BEEN• Scott presented at the Evolution Summit in Cologne,
Germany• Lisa attended Action Duchenne in London, UK• Lisa attended the Roche Spinal Muscular Atrophy
Investigator Meetings in Lisbon, Portugal• Scott and Lisa attended and presented at the World Orphan
Drug Congress in Brussels, Belgium• Sravan presented at MAGI in Las Vegas, NV
WHERE WE'RE GOING
January 2017:• Lisa attended JPM in San Francisco, California• Scott's interview on rare disease innovations in the January
edition of PharmaVoice
CONTACT INFO
If you need assistance with a rare disease study, have a particular personal interest in rare diseases, or would like more information, please contact us at:
LISA DILWORTHDirect: +1 [email protected]
SARAH ROSEDirect: +1 [email protected]
NEWLY AWARDED RARE DISEASE STUDIES:
• Cystic Fibrosis Phase II study• Beta Thalassemia Phase II study• Spinal Muscular Atrophy: Compassionate use award to ac-
company the 2-study program we are running• Primary Sclerosing Cholangitis• ALS• Primary Sjogren’s syndrome• Retrospective Chart Review - Fatty Acid Oxidation Disorders• Consulting agreement• Secured preferred partnership with European rare
disease-focused client
IN THE PIPELINE:
• Retinitis Pigmentosa ophthalmologic device Phase I/II program (3 studies)
• SMA program (an additional 4 studies)• Endogenous Cushing's syndrome• Ocular cystinosis• Sickle cell disease• Gene therapy for Hemophilia A• Friedreich's ataxia• Prader-Willi Syndrome• Rare disease preferred provider relationship• FOP
ACTION DUCHENNE
CONFERENCE IN LONDON IN NOVEMBER.
