Biotech

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.

SAN RAFAEL, Calif., Sept. 08, 2016 (GLOBE NEWSWIRE) — 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.
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.

Oral Presentations

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, 2016
10:30 am – 12:00 pm
Authors: A Schulz
Evaluation of long-term safety and efficacy of pegvaliase treatment for adults with phenylketonuria: updated year 4 results
Presentation: Thur., Sept. 8, 2016
13:15 – 14:45 pm
Authors: N Longo

Poster presentations:

Expert opinion on the management of Intracerebroventricular (ICV) drug delivery
Authors: 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
Authors: 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
Authors: 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
Design and rationale of the study programs for BMN 250, a novel Enzyme Replacement Therapy (ERT) for Sanfilippo B syndrome
Authors: A Shaywitz, M Oh, S Kent
Dramatic mobility improvement on galsulfase ERT: a case report
Authors: J Zakharchuk
Clinical outcomes from a sub-analysis of adults with Morquio A in a long-term extension study of elosulfase alfa treatment
Authors: 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
Authors: 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
National study to assess current practices of the management of phenylketonuria
Authors: 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
Authors: 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)
Authors: 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
Authors: 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)
Authors: 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 LevyPhase
3 PRISM-2 long-term extension study evaluating efficacy and safety of pegvaliase for treatment of adults with phenylketonuria 
Authors: 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
Authors: 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
Authors: AC Muntau, FB Lagler, F Feillet, J Alm, A Burlina, A Belanger-Qunitana, I Alvarez, A Champigneulle, FK Trefz, FJ Van Spronsen
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
Authors: 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 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 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.

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