• HOME
  • NEWS
  • EXPLORE
    • CAREER
      • Companies
      • Jobs
    • EVENTS
    • iGEM
      • News
      • Team
    • PHOTOS
    • VIDEO
    • WIKI
  • BLOG
  • COMMUNITY
    • FACEBOOK
    • INSTAGRAM
    • TWITTER
Tuesday, October 7, 2025
BIOENGINEER.ORG
No Result
View All Result
  • Login
  • HOME
  • NEWS
  • EXPLORE
    • CAREER
      • Companies
      • Jobs
        • Lecturer
        • PhD Studentship
        • Postdoc
        • Research Assistant
    • EVENTS
    • iGEM
      • News
      • Team
    • PHOTOS
    • VIDEO
    • WIKI
  • BLOG
  • COMMUNITY
    • FACEBOOK
    • INSTAGRAM
    • TWITTER
  • HOME
  • NEWS
  • EXPLORE
    • CAREER
      • Companies
      • Jobs
        • Lecturer
        • PhD Studentship
        • Postdoc
        • Research Assistant
    • EVENTS
    • iGEM
      • News
      • Team
    • PHOTOS
    • VIDEO
    • WIKI
  • BLOG
  • COMMUNITY
    • FACEBOOK
    • INSTAGRAM
    • TWITTER
No Result
View All Result
Bioengineer.org
No Result
View All Result
Home NEWS Science News

Neuren’s Phase 2 trofinetide trial shows significant benefit in pediatric Rett syndrome

Bioengineer by Bioengineer
March 22, 2017
in Science News
Reading Time: 2 mins read
0
Share on FacebookShare on TwitterShare on LinkedinShare on RedditShare on Telegram

(Cincinnati, OH) The International Rett Syndrome Foundation, now doing business as Rettsyndrome.org, announces today that Neuren Pharmaceuticals reported that trofinetide had significant clinical benefit in their Phase 2 clinical trial in girls with Rett syndrome aged 5 to 15. Rettsyndrome.org (a sponsor of this trial) is enthusiastic about the results and proud to partner with Neuren to move trofinetide forward toward the Phase 3 clinical trial.

Neuren's trial was a double-blind, randomized, placebo controlled study with 82 subjects. Neuren tested three doses of trofinetide compared with placebo in the subjects. The highest dose of trofinetide achieved statistically significant clinical benefit compared with placebo for each of three syndrome-specific efficacy measures; the Rett Syndrome Behavior Questionnaire, the Clinical Global Impression of Improvement and the Rett Syndrome Domain Specific Concerns. These measures included assessments by both clinicians and caregivers. Clinical improvements above baseline were determined as clinically meaningful by leading Rett syndrome physicians. To read the full study results, please go to https://www.rettsyndrome.org/document.doc?id=574

These results provide strong evidence of biological activity of the high dose across multiple symptom areas. This indicates the potential for disease modification rather than simply addressing isolated symptoms. In addition, trofinetide was well tolerated and had a good safety profile in these younger subjects, with no dose-limiting effects observed.

Neuren now intends to discuss with the US Food and Drug Administration (FDA) plans for a pivotal Phase 3 trial commencing in 2018. Neuren will be using the Rett Syndrome Behavior Questionnaire (RSBQ) as a primary efficacy measure, supported by the Clinical Global Impression of Improvement (CGI-I) as a key secondary efficacy measure. In parallel, Neuren will now move to complete the necessary chronic toxicity studies and manufacturing scale-up.

Rettsyndrome.org's Chief Science Officer, Steve Kaminsky, shares, "These pediatric study results are very exciting. The data suggest that trofinetide is having a positive change on a number of challenges of Rett syndrome. We at Rettsyndrome.org are very proud to have supported this game-changing study, believing that the best is yet to come."

###

About Rettsyndrome.org

Rettsyndrome.org is the most comprehensive nonprofit organization dedicated to accelerating research of treatments and a cure for Rett syndrome to accelerate full spectrum research to treat and cure Rett syndrome while empowering the community through knowledge and connectivity.

As the world's leading private funder of Rett syndrome research, Rettsyndrome.org has funded over $40M in high-quality, peer-reviewed research grants and programs to date. The organization hosts the largest global gathering of Rett researchers and clinicians to establish research direction for the future. Rettsyndrome.org, a 501(c)3 organization, has earned Charity Navigator's prestigious 3 star rating year after year. To learn more about our work and Rett syndrome, visit http://www.rettsyndrome.org or call (800) 818-7388 (RETT).

Media Contact

Steven Kaminsky
[email protected]
301-514-4068

Homepage

############

Story Source: Materials provided by Scienmag

Share12Tweet8Share2ShareShareShare2

Related Posts

Enzalutamide vs. Abiraterone: A Taiwan Study Insight

October 7, 2025

Study Reveals GLP-1 Agonists Present New Challenges for PET-CT Imaging

October 7, 2025

Linkage: Connect DNA Regulatory Peaks to Genes

October 7, 2025

Understanding Cassava Mosaic Disease in Burkina Faso

October 7, 2025
Please login to join discussion

POPULAR NEWS

  • Sperm MicroRNAs: Crucial Mediators of Paternal Exercise Capacity Transmission

    955 shares
    Share 382 Tweet 239
  • New Study Reveals the Science Behind Exercise and Weight Loss

    99 shares
    Share 40 Tweet 25
  • New Study Indicates Children’s Risk of Long COVID Could Double Following a Second Infection – The Lancet Infectious Diseases

    95 shares
    Share 38 Tweet 24
  • Ohio State Study Reveals Protein Quality Control Breakdown as Key Factor in Cancer Immunotherapy Failure

    77 shares
    Share 31 Tweet 19

About

We bring you the latest biotechnology news from best research centers and universities around the world. Check our website.

Follow us

Recent News

Enzalutamide vs. Abiraterone: A Taiwan Study Insight

Study Reveals GLP-1 Agonists Present New Challenges for PET-CT Imaging

Linkage: Connect DNA Regulatory Peaks to Genes

Subscribe to Blog via Email

Enter your email address to subscribe to this blog and receive notifications of new posts by email.

Join 63 other subscribers
  • Contact Us

Bioengineer.org © Copyright 2023 All Rights Reserved.

Welcome Back!

Login to your account below

Forgotten Password?

Retrieve your password

Please enter your username or email address to reset your password.

Log In
No Result
View All Result
  • Homepages
    • Home Page 1
    • Home Page 2
  • News
  • National
  • Business
  • Health
  • Lifestyle
  • Science

Bioengineer.org © Copyright 2023 All Rights Reserved.