FDA Allows IND Application for Investigational Duchenne MD Therapy Carmeseal-MD

FDA Allows IND Application for Investigational Duchenne MD Therapy Carmeseal-MD
0
(0)

The U.S. Food and Drug Administration (FDA) has allowed an investigational new drug (IND) application for Carmeseal-MD in Duchenne muscular dystrophy (DMD), according to Phrixus Pharmaceuticals.

Carmeseal-MD (Poloxamer-188 NF, or P-188 NF) is the first disease-modifying agent in diseases characterized by membrane instability such as Duchenne MD or limb girdle muscular dystrophy, among others.

Phrixus has a leading global patent portfolio for the use of poxamers in DMD, heart failure, and respiratory dysfunction. The pharmaceutical company is developing Carmeseal-MD for subcutaneous injection in DMD and Carmeseal-HF for intravenous administration, for acute decompensated heart failure.

DMD is estimated to affect 20,000 boys and young men in the U.S. and a similar number in Europe. Hallmarks of DMD include skeletal muscle weakness, respiratory distress, and heart failure.

Carmeseal MD has the potential to protect all three dystrophic target muscles: skeletal limb muscle, diaphragm, and heart.

In animal models of DMD and heart failure, Carmeseal-MD has been proven to improve the efficiency of damaged hearts. In the same models, the drug candidate has also been shown to improve the performance of damaged diaphragms and to protect skeletal limb muscle.

Carmeseal-MD is expected to be effective in all DMD patients, regardless of the genetic defect.

The studies that enabled the IND application were supported by the National Institutes of Health’s (NIH) National Heart, Lung and Blood Institute (NHLBI) and by Coalition Duchenne.

The basis for the IND include a two-arm, randomized, double-blind clinical trial with 120 participants at several sites. One dose of Carmeseal-MD will be evaluated against the standard of care over 48 weeks.

The study’s primary endpoint will be forced vital capacity (FVC), and a broad set of secondary endpoints will include cardiac endpoints and additional respiratory endpoints and measures of upper body strength, besides safety measures.

The principal investigator leading the study will be Dr. John L. Jeffries, the director of Advanced Heart Failure and Cardiomyopathy, and a cardiology professor at the Heart Institute and a professor of human genetics at the Cincinnati Children’s Hospital Medical Center.

“This novel therapeutic strategy offers a unique opportunity to potentially favorably impact outcomes in boys and young men with Duchenne muscular dystrophy,” Jeffries said in a press release.

Carmeseal-MD is already being sold in Europe through Ethicor Pharma as part of Phrixius’ European Access Program.

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
×
Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
Latest Posts
  • Hockey player Ryan Getzlaf
  • Sarepta partnership
  • MYO-101 for LGMD2E
  • ICER review

How useful was this post?

Click on a star to rate it!

Average rating 0 / 5. Vote count: 0

No votes so far! Be the first to rate this post.

As you found this post useful...

Follow us on social media!

We are sorry that this post was not useful for you!

Let us improve this post!

Tell us how we can improve this post?