Navigation Links
BioMarin Initiates Phase 2 Clinical Study of PEG-PAL in PKU
Date:9/22/2009

NOVATO, Calif., Sept. 22 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the first patient has initiated treatment in the Phase 2 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). Initial top-line results are expected in mid-2010.

"We remain optimistic on this program and believe that, if approved, PEG-PAL may offer a significant benefit for many PKU patients, especially those who do not respond adequately to Kuvan," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "In our view, the flexible design of the Phase 2 protocol provides multiple opportunities to arrive at an optimal dose and dosing frequency that is tolerable in at least in a sub-segment of the PKU population. We are encouraged by the results of the Phase 1 study, which showed a substantial blood Phe level reduction across all patients in the fifth cohort at a dose of 0.1mg/kg, no serious adverse events, and reactions as expected with a protein of this nature.

The Phase 2 clinical trial is an open-label, multi-center study to be conducted in up to 35 patients in a series of dose-escalating cohorts from 0.001 mg/kg. The primary treatment period of eight once weekly injections at a fixed dose will be followed by eight weeks of dose and frequency optimization and an extension period where doses can be increased up to 2.0 mg/kg/week.

The primary objective is to evaluate the effect of PEG-PAL on blood Phe concentrations in subjects with PKU. The secondary objectives are to evaluate the safety and tolerability, immune response and steady state pharmacokinetics of subcutaneous injections of multiple dose levels of PEG-PAL.

Abou
'/>"/>

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2009 PR Newswire.
All rights reserved

Page: 1 2 3

Related medicine technology :

1. BioMarin Initiates Phase 1/2 Clinical Trial for GALNS for Morquio A Syndrome
2. BioMarin Announces Positive Results From Phase 2A Clinical Study of 6R-BH4 in Sickle Cell Disease
3. BioMarin Initiates Phase 1 Clinical Study of PEG-PAL in PKU
4. BioMarin Announces FDA Approval for Kuvan
5. BioMarin Partner AnGes MG, Inc. Submits BLA to Japanese Ministry of Health
6. Lexicon Completes Phase 1 Clinical Trial and Initiates Phase 2 Clinical Trial of LX4211 in Patients With Type 2 Diabetes
7. LigoCyte Pharmaceuticals Initiates a Randomized, Double Blind, Placebo Controlled Norovirus Vaccine Study Including Live Virus Challenge
8. Takeda Initiates Cardiovascular Outcomes Trial for Alogliptin, An Investigational Treatment for Type 2 Diabetes
9. Terumo Cardiovascular Systems Initiates U.S. Clinical Trial for Anaconda(TM) AAA Stent Graft System
10. Cytokine PharmaSciences Initiates Phase I Study of Oral Anti-Inflammatory Agent, CPSI-2364
11. Conatus Pharmaceuticals Initiates a Second Phase 2 Clinical Trial for the Treatment of Hepatitis
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/24/2014)... , Dec. 24, 2014  Actavis plc (NYSE: ... Company has received a complete response letter from ... its New Drug Application (NDA) for the fixed-dose ... treatment of hypertension.   Photo - ... disappointed in the receipt of a complete response ...
(Date:12/24/2014)... CHENGDU, China, Dec. 24, 2014 /PRNewswire/  -- Tianyin ... a pharmaceutical company that specializes in the patented ... and active pharmaceutical ingredients (API) released unaudited preliminary ... year 2015. Fiscal Year 2015 Ended ... was $9.7 million compared with $14.7 million in ...
(Date:12/24/2014)... Soligenix, Inc. (OTCQB: SNGX) (Soligenix or ... that address unmet medical needs in the areas ... on December 24, 2014 it closed its previously ... raised approximately $2.3 million in gross proceeds from ... proceeds from the offering to fund its research ...
Breaking Medicine Technology:Actavis Confirms Complete Response Letter for Nebivolol/Valsartan Fixed-Dose Combination for Hypertension 2Actavis Confirms Complete Response Letter for Nebivolol/Valsartan Fixed-Dose Combination for Hypertension 3Actavis Confirms Complete Response Letter for Nebivolol/Valsartan Fixed-Dose Combination for Hypertension 4TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 2TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 3TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 4TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 5TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 6TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 7TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 8TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 9TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 10TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 11TPI Presents Unaudited Preliminary First Quarter Fiscal Year 2015 Financial Results 12Soligenix Announces Closing of Public Offering of Units 2Soligenix Announces Closing of Public Offering of Units 3
... Company (Nasdaq: PRGO;TASE) today announced that its Board ... per share, payable on March 20, 2012 to ... Perrigo Company is a leading global healthcare ... generic prescription (Rx) pharmaceuticals, infant formulas, nutritional products, ...
... Corporation (NASDAQ: LIFE ) today announced ... Leader, GE Molecular Diagnostics, has joined the company as ... of experience in the diagnostics industry, including leadership positions ... As President, Medical Sciences at Life Technologies, ...
Cached Medicine Technology:Ronnie Andrews Joins Life Technologies as President, Medical Sciences 2Ronnie Andrews Joins Life Technologies as President, Medical Sciences 3Ronnie Andrews Joins Life Technologies as President, Medical Sciences 4
(Date:12/25/2014)... News) -- Among early stage breast cancer patients who ... one percent will eventually develop leukemia as a result ... finding comes from a review of more than 20,000 ... it suggests that the risk for developing treatment-related leukemia, ... thought. "The frequency of bone marrow cancers such ...
(Date:12/25/2014)... (PRWEB) December 25, 2014 AngelWeddingDress.com tries ... to the online realm. Today, the business has introduced ... a site-wide prom dress promotion. , The new prom ... each and every dress lover. They are specially designed ... 2015 prom dresses should visit its website before ...
(Date:12/25/2014)... (PRWEB) December 25, 2014 “ SmileStix ” ... which features the latest and coolest mobile applications on the ... expert and host of NewsWatch, conducted the app review and ... to create personal stickers. , The old saying goes, “A ... lot of words that can describe a single image, because ...
(Date:12/25/2014)... North Carolina (PRWEB) December 25, 2014 ... North Carolina Retirement Community, recently showed their generous ... girls of the Catawba County Parenting Network- Grandparents ... the 10 children gathered around the largest Christmas ... to open presents purchased by residents of the ...
(Date:12/25/2014)... Parker & Sons, Inc. an industry leader when ... regarding heating, cooling and plumbing services in the greater ... “Queen of Clean” for impressive contractor services in Arizona. ... the years for providing outstanding quality customer service and ... for both homeowners and businesses. Linda Cobb, known as ...
Breaking Medicine News(10 mins):Health News:Breast Cancer Chemo Tied to Small But Significant Leukemia Risk 2Health News:Breast Cancer Chemo Tied to Small But Significant Leukemia Risk 3Health News:Angelweddingdress 2015 Prom Dresses Online Now 2Health News:An Application to Create Custom Stickers was Featured on NewsWatch Television on December 19, 2014 2Health News:Retirement Community Residents Make Impact During Holiday Season 2Health News:Parker & Sons Announces Recognition In 2014 By The “Queen Of Clean” For Impressive Contractor Services In Arizona 2Health News:Parker & Sons Announces Recognition In 2014 By The “Queen Of Clean” For Impressive Contractor Services In Arizona 3
... ... an all-new chapter, Dating Games Men Play: 16 Men,s Deadly Dating Patterns that will help ... book also includes specific dating advice for single mothers, women over 45, successful professionals and ... (PRWEB) January 28, ...
... ... Sunquest Collection Manager™ automated specimen collection software , ... (PRWEB) January 28, 2010 -- ... announced that Valley Medical Center, in Renton, Washington, has licensed Sunquest ...
... Bella’s House Community ... in and around the Houston, TX area. We have added two new ways parents can ... ... Community News is dedicated to supporting parents with special needs children by acting as a ...
... identified several cytokines, cytokine-related factors, and chemokines that increase ... These findings confirm those of earlier studies which suggest ... and disease progression may be prevented. Complete findings of ... Arthritis & Rheumatism , a journal of the ...
... new study led by Mount Sinai researchers in collaboration ... for Disease Control and Prevention, has found higher prenatal ... be connected with disruptive and problem behaviors in children ... study, which is the first to examine the effects ...
... 30 Groups Emphasize The Urgency Of Repealing Medicare ... Americans , WASHINGTON , Jan. 27 ... professionals and providers recently sent a letter to Congressional ... process for Medicare Part B outpatient therapy services. Without ...
Cached Medicine News:Health News:New Paperback Dating Advice Book, Love in 90 Days, Reveals 16 Men's Dead-End Dating Patterns and What To Do About Them 2Health News:New Paperback Dating Advice Book, Love in 90 Days, Reveals 16 Men's Dead-End Dating Patterns and What To Do About Them 3Health News:Sunquest Information Systems Enters Into Business Relationship with Valley Medical Center of Washington 2Health News:Announcing the Launch of a New Workshop and Forum for Parents of Disabled Children 2Health News:Announcing the Launch of a New Workshop and Forum for Parents of Disabled Children 3Health News:Blood test can predict rheumatoid arthritis before symptoms arise 2Health News:Mount Sinai finds prenatal exposure to certain chemicals affects childhood neurodevelopment 2Health News:Congress Urged to Take Immediate Action to Ensure Frail Patients Continue to Receive Critical Therapy Services 2Health News:Congress Urged to Take Immediate Action to Ensure Frail Patients Continue to Receive Critical Therapy Services 3