BioMarin Announces 3 Platform and 15 Poster Presentations at 13th International Congress of Inborn Errors of Metabolism 2017
SAN RAFAEL, Calif., Sept. 5, 2017 /PRNewswire/ -- BioMarin (Nasdaq:BMRN) announced that the company will present data in three platform presentations and fifteen poster presentations at the 13th International Congress of Inborn Errors of Metabolism (ICIEM) being held September 5-8, 2017 in Rio de Janeiro, Brazil. Presentations span a range of areas in rare genetic diseases, including neuronal ceroid lipofuscinosis type 2 (CLN2) disease, phenylketonuria (PKU) and mucopolysacchardosis (MPS).
Platform presentations include preliminary safety and pharmacodynamic response data from a Phase 1/2 study of ICV BMN 250, a novel enzyme replacement therapy for the treatment of Sanfilippo B (MPS IIIB), a long-term safety and efficacy analysis of cerliponase alfa to treat children with CLN2 disease, as well as updated five-year safety and efficacy data evaluating pegvaliase to treat adults with PKU.
Listing of Posters and Presentations Related to BioMarin Products and Programs at the 13th International Congress of Inborn Errors of Metabolism (All times are local)
Platform Presentations
Title Authors
----- -------
Preliminary safety and Steve Maricich, MD, PhD, BioMarin
pharmacodynamics response Pharmaceutical Inc. Novato, CA, USA
data from a phase 1/2 study
of ICV BMN 250, a novel
enzyme replacement therapy
for the treatment of
Sanfilippo syndrome type B
(MPS IIIB)
Parallel Session 1: Wednesday,
September 6 from 12:00-12:15
------------------------------
Long-term safety and efficacy Marina Trivisano, MD, Bambino Gesú
of intracerebroventricular Children's Hospital, IRCCS, Rome,
enzyme replacement therapy Italy
with cerliponase alfa in
children with CLN2 disease:
interim results from an
ongoing multicenter extension
study
Parallel Session 1: Wednesday,
September 6 from 12:00-12:15
------------------------------
Phase 2 long-term pegvaliase Jerry Vockley, MD, PhD, Children's
treatment for adults with Hospital of Pittsburgh and University
phenylketonuria: updated year of Pittsburgh, Pittsburgh, PA, USA
5 safety and efficacy data
from the PAL-003 extension
Parallel Session 7: Wednesday,
September 6 from 14:45-15:00
------------------------------
Poster Presentations
MPS
---
Title Authors
----- -------
Design and rationale of ongoing Shaywitz A, Maricich S, Yu H,
observational and treatment Kent S
studies for BMN 250, a novel
enzyme replacement therapy for
Sanfilippo syndrome type B (MPS
IIIB)
Presentation: Wednesday, September
6 from 17:30-20:00
Poster/Presentation: 597
------------------------
Presenting signs and symptoms of Clarke L, Ellaway C, Foster H,
MPS: results of an international Giugliani R, Goizet C, Goring S,
physician survey Hawley S, Jurecki E, Khan Z,
Lampe C, Martin K, McMullen S,
Mitchell J, Mubarack F, Muenzer
J, Sivri S, Stewart F, Tylki-
Szymanska A, White K, Wijburg F
Presentation: Wednesday, September
6 from 17:30-20:00
Poster/Presentation: 649
------------------------
Presenting signs and symptoms of Mubarack F, Clarke L, Ellaway C,
MPS: results of systematic Foster H, Giugliani R, Goizet C,
literature analysis Goring S, Hawley S, Jurecki E,
Khan Z, Lampe C, Martin K,
McMullen S, Mitchell J, Muenzer
J, Sivri S, Stewart FJ, Tylki-
Szymanska A, White K, Wijburg F
Presentation: Wednesday, September
6 from 17:30-20:00
Poster/Presentation: 662
------------------------
Mucopolysaccharidosis type VI (MPS Tomanin R, Karageorgos L, AlSayed
VI) and molecular analysis: a M, Bailey M, Izzo E, Miller N,
review of published classified Sakuraba H, Zanetti A, Hopwood
variants in the ARSB gene JJ
Presentation: Wednesday, September
6 from 17:30-20:00
Poster/Presentation: 674
------------------------
Impact of elosulfase alfa Lavery C, Jones SA, Hughes D,
treatment on patient-reported Murphy E, Jovanovic A, Hendriksz
outcomes in Morquio A syndrome: C, Cleary M, Hiwot T, Vijay S
results from the first year of an
English managed access agreement
Presentation: Wednesday, September
6 from 17:30-20:00
Poster/Presentation: 807
------------------------
CLN2
----
Title Authors
----- -------
Long-term safety and efficacy of Trivisano M, Schulz A,
intracerebroventricular enzyme replacement Specchio N, Gissen P, de
therapy with cerliponase alfa in children los Reyes E, Cahan H,
with CLN2 disease: interim results from an Slasor P, Ajayi T,
ongoing multicenter extension study Jacoby D
Presentation: Wednesday, September 6 from
17:30-20:00
Poster/Presentation: 787
------------------------
PKU
---
Title Authors
----- -------
Phase 3 PRISM-2 long-term extension Vockley J, Levy H, Amato S,
study evaluating efficacy and safety of Zori R, Thomas J, Burton
pegvaliase for treatment of adults with B, Harding C, Longo N,
phenylketonuria Posner J, Bilder D,
Olbertz J, Gu Z, Lau K,
Lin M, Larimore K, Dimmock
D
Presentation: Wednesday, September 6
from 17:30-20:00
Poster/Presentation: 285
------------------------
Phase 2 long-term pegvaliase treatment Zori R, Levy H, Longo N,
for adults with phenylketonuria: Lau K, Rosen O, Li M,
updated year 5 safety and efficacy data Larimore K, Decker C, Weng
from the PAL-003 extension H, Vockley J
Presentation: Wednesday, September 6
from 17:30-20:00
Poster/Presentation: 288
------------------------
Seventh interim analysis of the Kuvan(R) Van Spronsen FJ, Burlina A,
Adult Maternal Paediatric European Alm J, Belanger-Quintana
Registry (KAMPER): interim results in A, Feillet F, Lagler FB,
BH4 deficiency patients Muntau AC, Trefz FK,
Jurecki ER, Kittus R,
Alvarez I
Presentation: Wednesday, September 6
from 17:30-20:00
Poster/Presentation: 295
------------------------
Seventh interim analysis of the Kuvan(R) Van Spronsen FJ, Muntau AC,
Adult Maternal Paediatric European Lagler FB, Feillet F, Alm
Registry (KAMPER): interim results in J, Burlina A, Belanger-
phenylketonuria patients Quintana A, Alvarez I,
Lilienstein J, Jurecki E,
Trefz FK
Presentation: Wednesday, September 6
from 17:30-20:00
Poster/Presentation:296
-----------------------
Interim analysis of the phenylketonuria Lilienstein J, Burton B,
(PKU) patients enrolled in the PKUDOS Grant M, Ficicioglu C,
registry Kopesky J, Nguyen-Driver
MD, Moore C, Jurecki E,
Longo N
Presentation: Wednesday, September 6
from 17:30-20:00
Poster/Presentation:297
-----------------------
An interim analysis of the Kuvan(R)Adult Feillet F, Ficicioglu C,
Maternal Paediatric European Registry Lagler FB, Longo N, Alm J,
(KAMPER) and phenylketonuria Muntau AC, Burlina A,
developmental outcomes and safety Belnager-Quintana A,
(PKUDOS) registries: pregnancies Trefz FK, Kittus R,
Jurecki E, Alvarez I,
Lilienstein J, Burton B
Presentation: Wednesday, September 6
from 17:30-20:00
Poster/Presentation: 298
------------------------
Estimation of resource use and quality Pastores G, Bracken J,
of life in phenylketonuria (PKU) Hughes J, Rogers Y,
patients in Ireland Stenson C, Clark A, Lloyd
A, Alvarez I, Jain M, Kha
A, Monavari A
Presentation: Wednesday, September 6
from 17:30-20:00
Poster/Presentation: 262
------------------------
Short-term biological variance of PHE Potter M, Pendar A, Langley
in patients with phenylketonuria E, Geraghty M
Presentation: Wednesday, September 6
from 17:30-20:00
Poster/Presentation: 250
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Amino acid level correlations between Potter M, Pendar A, Langley
tandem mass spectrometry and ultra- E, Geraghty M
performance liquid chromatography and
the clinical relevance for
phenylketonuria management
Presentation: Wednesday, September 6
from 17:30-20:00
Poster/Presentation: 251
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About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.
For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
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Contact:
Investors: Media:
Traci McCarty Debra Charlesworth
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.
(415) 455-7558 (415) 455-7451
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