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 2,900+ scientific posters on ePosters
  • More Than 4,200+ 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

Visualizing a Cancer Drug Target at Atomic Resolution
Using cryo-electron microscopy, researchers were able to view, in atomic detail, the binding of a potential small molecule drug to a key protein in cancer cells.
Wednesday, February 10, 2016
Tick Genome Reveals Secrets of a Successful Bloodsucker
NIH-funded study could lead to new tick control methods.
Tuesday, February 09, 2016
Genomic Signature Shared by Five Types of Cancer
National Institutes of Health researchers have identified a striking signature in tumor DNA that occurs in five different types of cancer.
Monday, February 08, 2016
Natural Protein Points to New Inflammation Treatment
Findings may offer insight to effective treatments for inflammatory diseases, such as rheumatoid arthritis, psoriasis, and multiple sclerosis.
Friday, February 05, 2016
Cancer Drug Target Visualized at Atomic Resolution
New study using cryo-electron microscopy shows how potential drugs could inhibit cancer.
Thursday, February 04, 2016
Genome-Wide Study Yields Markers of Lithium Response
An international consortium of scientists has identified a stretch of chromosome that is associated with responsiveness to the mood-stabilizing medication lithium among patients with bipolar disorder.
Monday, February 01, 2016
Schizophrenia’s Strongest Known Genetic Risk Deconstructed
Suspect gene may trigger runaway synaptic pruning during adolescence – NIH-funded study.
Thursday, January 28, 2016
Experimental Combination Surprises with Anti-HIV Effectiveness
A compound developed to protect the nervous system from HIV surprised researchers by augmenting the effectiveness of an investigational antiretroviral drug beyond anything expected.
Monday, January 25, 2016
Dengue Vaccine Enters Phase 3 Trial
Investigational vaccine to prevent ‘breakbone fever’ developed at NIH.
Friday, January 15, 2016
NIH Genome Sequencing Program Targets the Genomic Bases of Common, Rare Disease
The National Institutes of Health will fund a set of genome sequencing and analysis centers whose research will focus on understanding the genomic bases of common and rare human diseases.
Friday, January 15, 2016
Trying to Conceive Soon After a Pregnancy Loss May Increase Chances of Live Birth
NIH study finds no reason for delaying pregnancy attempts after a loss without complications.
Wednesday, January 13, 2016
Three Glaucoma-Related Genes Discovered
NIH-funded genetics analysis of glaucoma is largest to date.
Tuesday, January 12, 2016
NIH-funded Memory Drug Moves into Phase 1 Clinical Study
Collaboration between NIH and Tetra Discovery Partners leads to development of treatment that may affect cognition.
Monday, January 04, 2016
International Study Reveals New Genetic Clues to AMD
NIH-funded research provides framework for future studies of AMD biology, therapy.
Tuesday, December 22, 2015
NIH Unveils FY2016–2020 Strategic Plan
Detailed plan sets course for advancing scientific discoveries and human health.
Thursday, December 17, 2015
Scientific News
Breaking Cell Barriers with Retractable Protein Nanoneedles
Adapting a bacterial structure, institute researchers have developed protein actuators that can mechanically puncture cells.
Gene Signature could Lead to a New Way of Diagnosing Lyme Disease
Lyme disease patients had distinctive gene signatures that persisted for at least three weeks, even after they had taken the antibiotics.
Retractable Protein Nanoneedles
The ability to control the transfer of molecules through cellular membranes is an important function in synthetic biology; a new study from researchers at Harvard’s Wyss Institute for Biologically Inspired Engineering and Harvard Medical School (HMS) introduces a novel mechanical method for controlling release of molecules inside cells.
Leukemia’s Surroundings Key to its Growth
Researchers at The University of Texas at Austin have discovered that a type of cancer found primarily in children can grow only when signaled to do so by other nearby cells that are noncancerous.
Common Cell Transformed into Master Heart Cell
By genetically reprogramming the most common type of cell in mammalian connective tissue, researchers at the University of Wisconsin—Madison have generated master heart cells — primitive progenitors that form the developing heart.
‘Smelling’ Prostate Cancer
A research team from the University of Liverpool and the University of the West of England (UWE Bristol) has reached an important milestone towards creating a urine diagnostic test for prostate cancer that could mean that invasive diagnostic procedures that men currently undergo eventually become a thing of the past.
Genetic Mutation that Prevents Diabetes Complications
The most significant complications of diabetes include diabetic retinal disease, or retinopathy, and diabetic kidney disease, or nephropathy. Both involve damaged capillaries.
A Crystal Clear View of Biomolecules
Fundamental discovery triggers paradigm shift in crystallography.
Could the Food we Eat Affect Our Genes?
Almost all of our genes may be influenced by the food we eat, according to new research.
NIH Seeks Research Applications to Study Zika in Pregnancy, Developing Fetus
Institute has announced that the new effort seeks to understand virus effect on reproduction and child development.
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
2,900+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,200+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!