Corporate Banner
Satellite Banner
Technology
Networks
Scientific Communities
 
Become a Member | Sign in
Home>News>This Article
  News
Return

First Drug Candidate from NIH Program Acquired by Baxter

Published: Tuesday, July 15, 2014
Last Updated: Tuesday, July 15, 2014
Bookmark and Share
Potential treatment targets sickle cell disease.

A drug candidate developed by researchers at the NIH's National Center for Advancing Translational Sciences (NCATS) and its collaborators to treat sickle cell disease has been acquired by Baxter International's BioScience business.

The drug candidate, Aes-103, is the first specifically developed to target the underlying molecular mechanism of sickle cell disease. Baxter now will advance the clinical development activities required for regulatory approval and commercialization.

Sickle cell disease is a genetic blood disorder that affects millions worldwide, including approximately 100,000 people in the United States - among them, 1 in 500 African-Americans.

This is the first time a company has acquired a drug candidate developed with NCATS' Therapeutics for Rare and Neglected Diseases (TRND) program resources. Baxter International recently acquired AesRx, LLC, Newton, Massachusetts - the TRND program collaborator - including Aes-103. TRND and AesRx researchers worked together to develop Aes-103 through a Phase II clinical trial to evaluate safety and effectiveness. The trial data indicated that Aes-103 significantly reduced patients' pain.

"This is a wonderful example of why NCATS was created," said NIH Director Francis S. Collins, M.D., Ph.D. "The progress made thus far in the development of Aes-103 demonstrates NCATS' catalytic role in bringing together the necessary players, whether academic, nonprofit or industry, to overcome obstacles to translation and advance badly needed treatments to patients."

Individuals living with sickle cell disease have defective hemoglobin, the protein in red blood cells that carries oxygen. This defect causes their cells to become rigid and crescent-shaped, blocking small blood vessels and causing inflammation, pain and strokes, and decreased blood flow.

Aes-103 works by binding directly to hemoglobin and changing its structure, thereby reducing the sickling of red blood cells. This structural change may lessen sickling-related complications in patients.

Sickle cell disease disproportionately affects African-Americans and is considered both rare and neglected in the United States. African-Americans with sickle cell often face significant health disparities in clinical care. Life expectancy for people with sickle cell disease is only to mid- to late 40s.

Prior to AesRx's collaboration with TRND researchers, and despite promising data on Aes-103, the company had difficulty securing private financing because potential investors lacked interest in funding an early-stage project that was considered too risky. AesRx did not have the resources to complete preclinical and early clinical development.

Currently, the only drug approved by the U.S. Food and Drug Administration (FDA) to treat sickle cell disease is hydroxyurea, a drug initially developed to treat cancer. However, the clinical utility of hydroxyurea is limited. Many individuals with sickle cell disease either do not respond to the drug, or they may experience undesirable side effects.

"Sickle cell was the first disease to ever have its molecular cause discovered - more than 65 years ago - and now a potential treatment based on that discovery has at last been developed," said NCATS Director Christopher P. Austin, M.D. "This success validates the NCATS model, which is based on a novel collaborative approach that de-risks intervention development programs to enable private-sector investment. We look forward to applying this model to the thousands of rare diseases that are currently untreatable so that we realize the NCATS mission of getting more treatments to more patients more quickly."

TRND researchers signed a collaborative agreement with AesRx in 2010 and established a project team made up of NCATS and AesRx scientists as well as a leading sickle cell disease clinical researcher at the National Heart, Lung, and Blood Institute (NHLBI). Other key project collaborators received support through NHLBI grants, the NIH Clinical Center and its pharmacy, and NCATS' Bridging Interventional Development Gaps program. Aes-103 was licensed by AesRx from Virginia Commonwealth University, Richmond, where the compound was discovered.

"This is an important milestone for the development of this potential sickle cell disease therapeutic, and we are pleased that NHLBI researchers were able to play a role in advancing this project from the beginning," said NHLBI Director Gary H. Gibbons, M.D. "NHLBI is dedicated to advancing sickle cell disease research as a strategic priority in an effort to improve the quality of care received by patients."

In less than one year, the team completed the preclinical toxicology, chemistry, manufacturing, controls and regulatory studies necessary to support an investigational new drug (IND) application, which AesRx filed with the FDA. After IND clearance, Aes-103 moved into Phase I clinical trials in healthy volunteers and sickle cell disease patients in 2011 and into a Phase II trial in patients in 2013. The project results also helped AesRx obtain a Massachusetts Life Science Accelerator loan to support development of Aes-103.

"This project may never have reached clinical trials if not for the TRND program and its preclinical drug development expertise and novel approaches," said Stephen Seiler, AesRx's founder and former CEO. "We believe Aes-103 has the potential to be a breakthrough in the treatment of sickle cell disease. TRND's support for AesRx has enabled us to bring that potential closer to realization."

"Acquiring AesRx and this clinical development program is an important opportunity, as it complements Baxter's established relationships and expertise in treating rare and challenging blood disorders," said Ludwig Hantson, Ph.D., president of Baxter BioScience. "This investment reflects our continued focus on addressing high unmet clinical needs for patients with inadequate treatment options and no recent major clinical developments."


Further Information

Join For Free

Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 3,000+ scientific posters on ePosters
  • More Than 4,500+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

Ketamine Metabolism Lifts Depression
NIH-funded team finds rapid-acting, non-addicting agent in mouse study.
Thursday, May 05, 2016
Finding Factors That Protect Against Flu
A clinical trial examining the body’s response to seasonal flu suggests new approaches for evaluating the effectiveness of seasonal flu vaccines.
Wednesday, April 27, 2016
Factors Influencing Influenza Vaccine Effectiveness Uncovered
The long-held approach to predicting seasonal influenza vaccine effectiveness may need to be revisited, new research suggests.
Thursday, April 21, 2016
Study Finds Factors That May Influence Influenza Vaccine Effectiveness
Researchers at NIH have suggested that the long-held approach to predicting seasonal influenza vaccine effectiveness may need to be revisited.
Wednesday, April 20, 2016
Serotonin Transporter Structure Revealed
Researchers determined the 3-D structure of the serotonin transporter and visualized how two common antidepressants interact with the protein.
Wednesday, April 20, 2016
Improving Flu Vaccine Effectiveness
NIH study finds factors that may influence influenza vaccine effectiveness.
Wednesday, April 20, 2016
Submissions Open for the Cancer Moonshot Program
NCI opens online platform to submit ideas about research for Cancer Moonshot.
Tuesday, April 19, 2016
Migration Creates Cancer Cell Vulnerabilities
Scientists found that migration can damage cancer cells’ nuclei and DNA, requiring repairs for their survival. The results may open new avenues for targeting metastatic cancer.
Wednesday, April 13, 2016
NIH Sequences Genome of a Fungus
Researchers at the Institute have sequenced genome of human, mouse and rat Pneumocystis that cause life-threatening Pneumonia in immunosuppressed hosts.
Tuesday, April 12, 2016
NIH Awards Grants to Explore Vaccine Adjuvants
NIH awards six grants to explore how combination adjuvants improve vaccines.
Wednesday, April 06, 2016
Children With Cushing Syndrome May Have Higher Suicide Risk
Researchers at NIH have found that depression, anxiety and suicidal thoughts increase after treatment.
Wednesday, March 30, 2016
Experimental Vaccine Protects Against Dengue Virus
An experimental dengue vaccine protected all the volunteers who received it from infection with a live dengue virus.
Wednesday, March 30, 2016
Couples’ Pre-Pregnancy Caffeine Consumption Linked to Miscarriage Risk
Researchers at NIH have found daily multivitamin before and after conception greatly reduces miscarriage risk.
Friday, March 25, 2016
Study Finds Mindfulness Meditation Offers Relief For Low-Back Pain
Researchers at NIH have found that the MBSR and CBT may prove more effective than usual treatment in alleviating chronic low-back pain.
Wednesday, March 23, 2016
3-D Technology Enriches Human Nerve Cells For Transplant to Brain
This platform is expected to make transplantation of neurons a viable treatment for a broad range of human neurodegenerative disorders.
Friday, March 18, 2016
Scientific News
AACR 2016: Cancer Immunotherapy and Beyond
At this year's meeting there was a palpable buzz around subjects ranging from microbiomics to the tumor microenvironment and cancer vaccines, big data to in vitro and in vivo modeling and drug delivery (to name just a few).
How Skeletal Stem Cells Form The Blueprint Of The Face
USC researchers discover that two types of molecular signals work to control where and when stem cells turn into facial cartilage.
Intestinal Worms Boost Immune System In A Surprising Way
EPFL researchers find that intestinal worm infections cause lymph nodes to produce more immune cells as well as grow in size.
Measuring The Airborne Toxicants Urban Bicyclists Inhale
Researchers analyze breath biomarkers to measure uptake of volatile organic compounds by bicyclists.
Breast Milk Hormones Impact Bacteria In Infants’ Guts
Intestinal microbiome of children born to obese mothers significantly different from those born to mothers of healthy weight, CU Anschutz researchers find.
Newborn Screening Test Developed For Rare, Deadly Neurological Disorder
Scientists have developed a new dried blood spot screening test for Niemann-Pick type C, with goal to speed diagnosis and treatment.
'Kidney on a Chip' Facilitates Safer Drug Dosing
University of Michigan researchers have used a "kidney on a chip" device to mimic the flow of medication through human kidneys and measure its effect on kidney cells.
New Autism Blood Biomarker Identified
Researchers at UT Southwestern Medical Center have identified a blood biomarker that may aid in earlier diagnosis of children with autism spectrum disorder, or ASD.
New Method Allows First Look At Embryo Implantation
Researchers at The Rockefeller University develop a method that shows the molecular and cellular processes that occur up to day 14 after fertilization.
Shining A Light On Bladder Cancer
Researchers scrutinize patterns of mutations in bladder tumor genomes, gleaning insights into the roles of DNA repair and tobacco-related DNA damage.
Scroll Up
Scroll Down
Skyscraper Banner

Skyscraper Banner
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
3,000+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,500+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!