BioMarin Announces Two Oral and 16 Poster Presentations at Society for the Study of Inborn Errors of Metabolism 2016 Annual M...
September 08 2016 - 4:30AM
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that two
oral and 16 poster presentations related to the company’s products
and product candidates will be made at the Society for the Study of
Inborn Errors of Metabolism (SSIEM) 2016 Annual Meeting, which will
be held on September 6-9 in Rome, Italy. These 18
presentations at SSIEM cover a range of areas in rare genetic
diseases, including Phenylketonuria (PKU), Mucopolysacchardosis
(MPS) and CLN2 disease, a form of Batten disease. The
presentations focus on better understanding the burden of disease
in PKU, intracerebroventricular (ICV) delivery and the multi-
disciplinary management of CLN2 disease and the impact of treatment
on adults with Morquio A syndrome, an MPS disease.
Oral Platforms at SSIEM
Updated long-term data from an extension study of Brineura™
(cerliponase alfa), a recombinant human tripeptidyl peptidase 1
(rhTPP1) to treat children with CLN2 disease, will be presented
during an oral platform session. CLN2 disease is a rapidly
progressing, fatal neurodegenerative disease with no approved
treatments, where the majority of affected children lose their
ability to walk and talk by approximately six years of age.
Brineura is an enzyme replacement therapy designed to restore TPP1
enzyme activity and break down the storage materials that cause the
disease. Brineura is currently under review by the U.S. Food and
Drug Administration with a Prescription Drug User Fee Act goal date
of April 27, 2017. BioMarin also has submitted a Marketing
Authorization Application to the European Medicines Agency for
Brineura, and it is undergoing validation at the Agency.
In addition, updated four year results of the long-term safety
and efficacy of pegvaliase to treat adults living with
phenylketonuria (PKU) will also be presented during an oral
session. PKU is a rare genetic condition in which the body cannot
metabolize the essential amino acid phenylalanine. Pegvaliase is an
enzyme substitution therapy that is designed to substitute
phenylalanine ammonia lyase for phenylalanine hydroxylase, the
enzyme deficient in people living with PKU. BioMarin intends to
submit a marketing application by the first quarter of 2017.
Additionally, data from 16 abstracts spanning CLN2 disease,
mucopolysaccharidosis, PKU, and Pompe disease will be
presented.
Listing of Posters and Presentations at
Society for the Study of Inborn Errors of Metabolism 2016
Annual Meeting
Oral Presentations
Title |
Authors |
Intracerebroventricular cerliponase alfa (BMN 190) in children with
CLN2 disease: Results from a Phase 1/2, open-label, dose-escalation
study Presentation: Thur.,
Sept. 8, 201610:30 am – 12:00 pm |
A Schulz |
Evaluation of long-term safety and efficacy of pegvaliase treatment
for adults with phenylketonuria: updated year 4 results
Presentation: Thur., Sept. 8, 201613:15 – 14:45
pm |
N Longo |
Poster Presentations
CLN2 Disease |
Title |
Authors |
Expert opinion on the management of Intracerebroventricular (ICV)
drug delivery |
I Slavc, JL Cohen-Pfeffer, S Gururangan, E Jurecki, J Krauser, T
Lester, D Lim, M Maldaun, C Schwering, AJ Shaywitz, M Westphal |
Expert opinion on the management of CLN2 disease |
N Specchio, RE Williams, HR Adams, M Blohm, JL Cohen-Pfeffer, E de
los Reyes, J Denecke, K Drago, C Fairhurst, M Frazier, N Guelbert,
S Kiss, A Kofler, J Lawson, L Lehwald, M Leung, S Mikhailova, JW
Mink, M Nickel, R Shediac, K Sims, M Topcu, I von Löbbecke, A West,
A Schulz |
Expert recommendations for the laboratory diagnosis of neuronal
ceroid lipofuscinosis type 2 (CLN2 disease): diagnostic algorithm
and best practice guidelines for a timely diagnosis |
E Izzo, M Al-Sayed, D Burke, J Cohen-Pfeffer, JD Cooper, L
Dvořáková, M Fietz, R Giugliani, H Jahnova, Z Lukacs, S Mole, I
Noher de Halac, D Pearce, A Schulz, N Specchio, W Xin, N
Miller |
Mucopolysaccharidosis (MPS) |
Title |
Authors |
Design and rationale of the study programs for BMN 250,
a novel Enzyme Replacement Therapy (ERT) for Sanfilippo B
syndrome |
A Shaywitz, M Oh, S Kent |
Dramatic mobility improvement on galsulfase ERT: a case
report |
J Zakharchuk |
Clinical outcomes from a sub-analysis of adults with
Morquio A in a long-term extension study of elosulfase alfa
treatment |
D Hughes, R Giugliani, N Guffon, AS Jones, KE Mengel, R Parini, R
Matousek, E Jurecki, A Quartel |
Morquio A Registry Study (MARS): design and baseline
characteristics of enrolled patients |
N Guffon, G Baujat, MB Bober, BK Burton, L Clarke, P Garcia, R
Giugliani, CJ Hendriksz, C Lavery, J Raiman, E Jurecki, Z Sisic, A
Waite |
|
|
Phenylketonuria (PKU) |
Title |
Authors |
National study to assess current practices of the management of
phenylketonuria |
F Rohr, ER Jurecki, S Cederbaum, J Kopesky, A
Sanchez-Valle, K Viau, JL Cohen-Pfeffer |
Neuropsychiatric concomitant medications and comorbidities in
individuals with Phenylketonuria: findings from the PKUDOS
Registry |
S Waisbren, J Zambrano, M Grant, B Cheng, S Parker, D
White, JL Cohen-Pfeffer |
Case-control study of neuropsychological results in patients with
mild hyperphenylalaninemia (MHP) |
A Belanger-Qunitana, B Zamora |
Neuropsychological and quality of life outcomes in untreated adults
with mild hyperphenylalaninemia with phenylalanine levels between
360 and 600 µmol/L |
A Feigenbaum, A Wilson, L Nagy, K Siriwardena, E Nasr,
E Kerr |
Phase 3 PRISM-1 and PRISM-2 clinical trial results: to evaluate the
efficacy and safety of pegvaliase for the treatment of adults with
phenylketonuria (PKU) |
C Harding, S Amato, J Vockley, K Wierenga, H Li, D
Bilder, B Burton, D Dimmock, N Longo, J Posner, JA Thomas, R Zori,
O Rosen, F Greblikas, Z Gu, M Li, M Merilainen, HH Weng, H
Levy |
Phase 3 PRISM-2 long-term extension study evaluating efficacy and
safety of pegvaliase for treatment of adults with
phenylketonuria |
H Levy, C Harding, N Longo, D Bilder, B Burton, R
Zori, J Posner, JA Thomas, J Olbertz, O Rosen, Z Gu, M
Merilainen, HH Weng, D Dimmock |
The sixth interim analysis of the Kuvan® Adult Maternal Paediatric
European Registry (KAMPER): pregnancies |
F Feillet, FB Lagler, J Alm, AC Muntau, A Burlina, A
Belanger-Qunitana, I Alvarez, A Champigneulle, FK Trefz |
The sixth interim analysis of the Kuvan® Adult Maternal Paediatric
European Registry (KAMPER): interim results in PKU and BH4
deficiency patients |
AC Muntau, FB Lagler, F Feillet, J Alm, A Burlina, A
Belanger-Qunitana, I Alvarez, A Champigneulle, FK Trefz, FJ Van
Spronsen |
Pompe disease |
Title |
Authors |
Long-term efficacy and safety of reveglucosidase alfa in subjects
with late-onset Pompe Disease: 144-week follow-up of the
POM-001/002 Studies |
T Geberhiwot, B Byrne, BA Barshop, R Barohn, D Hughes, D Bratkovic,
C Desnuelle, P Laforet, E Mengel, M Roberts, P Haroldsen, L Smith,
K Yang, L Walsh |
About BioMarinBioMarin 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 five 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.
BioMarin® and Kuvan® are registered trademarks
and Brineura™ is a trademark of BioMarin Pharmaceutical Inc.
Contact:
Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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