Full Press Release Details
NOVATO, Calif. , July 28, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN ) today announced an update on the GALNS Phase 1/2 extension study (MOR-100) in which patients have continued treatment on an ongoing basis.
Patients originally enrolled in the initial Phase 1/2 study of GALNS (MOR-002) were continued on therapy in a new extension study (MOR-100). In the MOR-100 study, patients were treated at a 2.0 mg/kg/week dose and have been followed for an additional 24 weeks. Previously, these patients were dosed at 0.1 mg/kg/week, 1.0 mg/kg/week and 2.0 mg/kg/week for 12 weeks each in the Phase 1/2 study (MOR-002). Following the dose escalation phase, patients were then dosed at 1.0 mg/kg/week for an additional 36 to 48 weeks as part of the MOR-002 extension, before continuing on to MOR-100. Taken together, these patients have received GALNS for approximately two years.
"We now have data from MPS IVA patients that have been on GALNS for approximately 2 years and the data continues to look promising," said Hank Fuchs , M.D., Chief Medical Officer of BioMarin. "Looking at the Phase 1/2 data in totality, we now see that the endurance and pulmonary function improvements are durable and clinically meaningful compared to baseline, and similar in magnitude to what we have observed during the development of Naglazyme and Aldurazyme. These data are encouraging in regard to the ongoing Phase 3 study in that the benefits of GALNS appear consistently sustained, and no new risks have emerged. Enrollment in the Phase 3 study is accelerating and its execution is BioMarin's highest product development priority."
Preliminary highlights, including information on patients treated for 24 additional weeks at 2.0 mg/kg/week are provided below. Data from the extension study will be submitted for presentation at the World Lysosomal Storage Disease conference in February 2012 .
Highlights of MOR-100 Extension Study:
Simon Jones , Consultant in Paediatric Metabolic Medicine, St. Mary's Hospital, Manchester UK commented, "I'm pleased with the outcome of the GALNS program to date. The longer-term data look good with sustained improvements in endurance and pulmonary function. Patients feel stronger and function better than I expected. All in all, we look forward to the results of the Phase 3 study as it is well designed and will, if successful, support introduction of an important product to patients in need."
Hank Fuchs , M.D., Chief Medical Officer of BioMarin added, "It's remarkable that improvements in endurance are sustained in spite of the severe and progressive nature of Morquio syndrome. Patients with Morquio syndrome can miss therapy, for example, due to the need for corrective surgery. In spite of that, resumption of therapy returns patients to their original state of health or better."
| Table 1 – Sum mary of Efficacy Results Through Week 24 of MOR-100 Extension Study | |||||||
| Statistic | MOR-002 Week 24 | MOR-002 Week 36 | MOR-100 Week 0 | MOR-100 Week 12 | MOR-100 Week 24 | ||
| Dose | 1.0 mg/kg | 2.0 mg/kg | 1.0 mg/kg for previous 36-48 weeks, Begin 2.0 mg/kg | 2.0 mg/kg | 2.0 mg/kg | ||
| ENDURANCE | |||||||
| 6 Minute Walk Test – change from Baseline (meters) | N Mean (Std. Dev.) Median | 16 17.3 (74.0) 37.6 | 16 14.6 (65.2) 18.9 | 15 13.9 (98.6) 14.0 | 15 21.8 (85.4) 15.7 | 14 29.1 (79.1) 6.8 | |
| 6 Minute Walk Test – change from Baseline (meters) (for patients with baseline walk distance < 325m) | N Mean (Std. Dev.) Median | 12 38.0 (45.2) 42.4 | 12 28.2 (61.0) 24.4 | 11 37.3 (79.8) 14.0 | 11 40.0 (77.0) 15.7 | 11 38.3 (81.8) 2.6 | |
| 3 Minute Stair Climb Test – change from Baseline (steps/minute) | N Mean (Std. Dev.) Median | 15 6.9 (8.9) 7.3 | 15 8.9 (14.3) 10.2 | 14 14.5 (14.3) 13.1 | 14 13.9 (15.2) 14.6 | 12 13.6 (19.6) 10.4 | |
| PULMONARY FUNCTION | |||||||
| FVC – Percent Change from Baseline | N Mean (Std. Dev.) Median | 15 1.5 (16.2) 0.0 | 15 7.2 (16.0) 10.0 | 14 11.5 (15.5) 6.9 | N/A | 13 15.9 (16.8) 12.0 | |
| MVV – Percent Change from Baseline | N Mean (Std. Dev.) Median | 13 11.0 (21.5) 6.4 | 14 10.5 (17.4) 10.0 | 13 11.1 (16.4) 10.6 | N/A | 12 10.3 (23.2) 0.8 | |
| * After Week 36 in MOR-002, patients were dosed at 1.0 mg/kg/week for 36 to 48 weeks. At Week 0 of MOR-100, patients were returned to 2.0 mg/kg/week dosing. ** Results were impacted by several patients with extenuating circumstances (knee procedures, car acciden t, unplanned extended drug holiday) after week 24 of MOR-002. |
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme ® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme ® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan ® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany ; and Firdapse™ (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA, amifampridine phosphate (3,4-diaminopyridine phosphate), which is currently in Phase III clinical development for the treatment of LEMS in the U.S., PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, and BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers. For additional information, please visit www.BMRN.com . Information on BioMarin's website is not incorporated by reference into this press release.
Forward-Looking Statement
BioMarin ® , Naglazyme ® , Kuvan ® and Firdapse™ are registered trademarks of BioMarin Pharmaceutical Inc.
Aldurazyme ® is a registered trademark of BioMarin/Genzyme LLC.
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SOURCE BioMarin Pharmaceutical Inc.