Navigation Links
Enobia Reports Positive Results For Phase II Trial of ENB-0040 in Juvenile Hypophosphatasia
Date:9/24/2010

PRAGUE, Sept. 24 /PRNewswire/ -- Enobia Pharma today announced positive results for its Phase II juvenile clinical trial of ENB-0040 (asfotase alfa), an experimental bone-targeted enzyme replacement therapy intended for the treatment of hypophosphatasia (HPP).  Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease that affects individuals of all ages. Michael Whyte, MD, Medical/Scientific Director of the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospital for Children in St. Louis, Missouri, and a principal investigator of the Phase II study, presented the findings at the European Society for Paediatric Endocrinology (ESPE) Annual Meeting in Prague. Enobia's abstract describing these Phase II results was awarded the Henning Anderson Prize for best clinical abstract by ESPE.

In this trial, after six months of ENB-0040 treatment results include:

  • Radiographic improvement of rickets
  • Improvement in muscle strength and agility over baseline, including an average improvement of 125m on six-minute walk test
  • Amelioration of pain in 6 of 7 patients experiencing pain at baseline  

"We are encouraged by these preliminary results. Improvements were seen in objective measures of muscle strength, agility and pain. These were accompanied by histomorphometric, radiologic and biomarker improvements, suggesting an integrated improvement of this debilitating skeletal disorder," said Hal Landy, MD, Chief Medical Officer at Enobia. "With nearly 50 patients on ENB-0040 treatment, we are rapidly expanding our understanding of the potential safety and efficacy of ENB-0040."  

Dr. Michael Whyte, Medical/Scientific Director of the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospital for Children in St. Louis and a Principal Investigators said, "The findings from this study represent an important advance in our quest to find a medical treatment for hypophosphatasia."

ENB-0040 Phase II Juvenile Trial Design

This six-month, Phase II, multinational, open-label investigation of the safety, tolerability, pharmacokinetics (PK), and efficacy of ENB-0040 treatment enrolled 13 children (ages 5-12 years, Tanner Stage less than or equal to 2) with rickets and gross motor deficits from HPP. One patient discontinued the trial due to elective scoliosis surgery. Inclusion criteria included low serum ALP activity for age, plasma PLP greater than or equal to 2x normal range, normal serum vitamin D, and evidence of HPP associated rickets on radiographs. Exclusion criteria included significant co-morbid disease, nutritional rickets, bisphosphonate exposure, hypocalcemia or hypophosphatemia, or experimental treatment for HPP (e.g., bone marrow transplantation). All patients who have completed the 6 month study have been enrolled in an extension study.

ENB-0040 Phase II Juvenile Trial Results

Although radiographs have yet to be assessed by blinded radiologists and compared to historical controls, preliminary radiographic improvements in rickets were noted in all patients by investigators. Consistent with data from the infant study, radiographic improvement was noted as early as 6 weeks after starting treatment in some patients. Plasma levels of two naturally occurring substrates of alkaline phosphatase, PLP and PPI, declined following subcutaneous (SC) injection of ENB-0040. Serum ALP concentrations increased substantially and were maintained within the target range throughout the study. Positive skeletal mineral balance with healing rickets was heralded by increases in serum parathyroid hormone levels. There were no overt episodes of hypocalcemia from "hungry bones."

SC injections of ENB-0040 were generally well tolerated and no serious adverse events were reported. Injection site reactions consisting of transient skin discoloration were common. Retinal findings, renal ultrasound studies, and skin examination showed no evidence of ectopic mineralization.

Enobia recently reported that updated results from a phase I/II trial in infantile HPP will be presented at the 60th Annual American Society of Human Genetics Annual Meeting in Washington, DC on November 3. In addition, a third phase II trial of ENB-0040 in adolescents and adults with HPP is underway.  

About Hypophosphatasia

Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease. Affected individuals have low levels of the tissue non-specific form of alkaline phosphatase, an essential regulator of bone mineralization, leading to rickets in infants and children and osteomalacia ("soft bones" resulting from poor mineralization) in adults. Disease severity is inversely proportional to the age at symptom onset and includes marked skeletal hypomineralization. The marked skeletal hypomineralization causes respiratory compromise often resulting in death in infants and persistent and debilitating osteomalacia in children and adults.

In the infantile form, infants may appear normal at birth but develop serious symptoms in the first six months of life. These can include failure to thrive, respiratory failure, fractures, and seizures. Radiographic findings include generalized hypomineralization and rickets. First year mortality in these patients is estimated at 50 percent. In the childhood form, patients have varying degrees of skeletal hypomineralization and may have frank rickets, short stature, bone pain, muscle weakness, delayed motor milestones, early loss of deciduous teeth, and may experience frequent, poorly-healing fractures. In the adult form, the underlying osteomalacia causes pathological fractures that impair ambulation often with debilitating bone pain.

About ENB-0040

There are currently no therapies approved for HPP, a rare genetic disease characterized primarily by defective bone mineralization caused by a deficiency in the enzyme tissue non-specific alkaline phosphatase (TNSALP). ENB-0040 (asfotase alfa), an investigational treatment for hypophosphatasia, is a subcutaneous enzyme replacement therapy of tissue non-specific alkaline phosphatase (TNSALP) fused to a bone targeting peptide. ENB-0040 is designed to directly target TNSALP to the bone in order to correct the enzyme deficiency, which could lead to restoration of normal bone mineralization. ENB-0040, awarded orphan designation in the U.S. and EU in 2008 and Fast Track status in 2009, is currently in Phase 2b clinical development.

About Enobia Pharma Inc.

Enobia Pharma Inc. is a private Montreal based company focused on the development of therapeutics to treat serious bone disorders for which there are no drug therapies currently approved. ENB-0040, an investigational drug for the treatment of hypophosphatasia, is the Company's lead program. For more information, please visit www.enobia.com.


'/>"/>
SOURCE Enobia Pharma Inc.
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. Enobia Pharma Initiates Clinical Testing of Enzyme Replacement Therapy to Treat Hypophosphatasia
2. Enobia Pharma Receives FDA Orphan Drug Designation for Hypophosphatasia Product Candidate ENB-0040
3. Enobia Pharma Appoints Industry Veterans with Commercial and Manufacturing Expertise to Expand Senior Management Team
4. Enobia Initiates Infantile Enzyme Replacement Trial for Rare Bone Disease
5. Enobia Announces Positive Clinical Results With ENB-0040, a Bone Targeted Enzyme Replacement Therapy for Hypophosphatasia
6. EpiCept Reports Expansion of Clinical Development For Licensed Cancer Compound
7. Spherix Reports Second Quarter Earnings
8. Tapestry Reports Second Quarter 2007 Results
9. Callisto Reports on Second-Quarter 2007 Milestones
10. ImmuneRegen BioSciences Reports Material Transfer Agreement with VaxGen, Inc.
11. Vical Reports Second Quarter 2007 Financial Results, Allowance of Pandemic Influenza IND and Other Advances in Product Development Programs
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/28/2017)... Elysium Health joins major pharmaceutical companies to ... Cambridge academic scientists through ... Institute today announces Elysium Health as a partner to the ... collaborative projects with academic researchers in Cambridge ... major research investment outside the U.S. for the ...
(Date:3/27/2017)... Summary This report provides all the information ... interests and activities since 2010. Description The Partnering Deals ... the partnering activity of one of the world,s leading life ... upon purchase to ensure inclusion of the most up to ... be delivered in PDF format within 1 working day of ...
(Date:3/27/2017)... , March 27, 2017  ImMAGE Biotherapeutics (OTCMKTS: ... immunotherapy successfully passed early toxicology and efficacy studies. The ... to target a specific protein, MAGE A, in an ... breast cancer. After 4 weeks of ... to show very little toxicity in a full toxicology ...
Breaking Medicine Technology:
(Date:3/28/2017)... ... March 28, 2017 , ... Calibration, qualification, and the ... results and maintaining GMP and USP compliance. In a new webinar from METTLER ... requirements " these requirements are explained. The challenge is to determine how ...
(Date:3/28/2017)... (PRWEB) , ... March 28, 2017 , ... With less ... from acne, access to quality care can be limited while the desire to conquer ... offers customized prescription acne care for every customer online, today released its inaugural survey ...
(Date:3/28/2017)... , ... March 28, 2017 , ... Columbus OH. Dr. ... as one of few medical professionals in the country to sit on the 2017 ... Juvly Aesthetics, in just 2 years Dr. Harper helped propel the clinic from a ...
(Date:3/28/2017)... ... March 28, 2017 , ... Public relations pros work ... a variety of business channels. , While many results are clear, much of PR ... program. , When it comes to measurement, firms should always take an all-inclusive ...
(Date:3/28/2017)... ... ... skin is a common and unwelcomed occurrence in people of all ages, genders and ethnicities. ... the discussion of dealing with excess skin oil. “Oily skin is a challenge to many ... help remove the oily shine while keeping the skin fresh and clean,” says Dr. Au. ...
Breaking Medicine News(10 mins):