Poloxamer 188 NF

Questions and Answers

What stage is this research?

In the US: Our research is at the clinical stage, our product has an open IND and approval for an initial clinical trial.

In Europe: While we are developing Carmeseal-MD in the US under FDA supervision, it is already available to patients in Europe, Argentina, and New Zealand as an unlicensed medicinal product (“Special”) when prescribed by a specialist.

Where is this research being done and who is funding this research?

Phrixus studies were supported financially by the SMARTT (Science Moving towards Research Translation and Therapy) program at NHLBI. Technical work is being conducted at SRI International. Our work to date has been funded by the National Institutes of Health (NIH) through SBIR grants, the Biosciences Research and Commercialization Center as well as Coalition Duchenne and Duchenne Alliance.

What is the goal or purpose of this research?

Our goal is to demonstrate that Carmeseal-MD has beneficial effects in patients with both Duchenne and Becker muscular dystrophy: An improvement in cardiac and respiratory function via protection of heart muscle and diaphragm as well as an improvement in upper body strength. Carmeseal-MD acts as a molecular band-aid by binding to and then sealing microscopic tears in muscle cells caused by the lack of functional dystrophin. This prevents the uncontrolled leakage of calcium which in turn increases the performance of heart muscle and diaphragm and prevents their degeneration.

What is the current state of this research?

Carmeseal-MD has been shown to be effective in three dystrophic animal models (mdx and mdx/utr double-knock out mice, GRMD dogs) and two models of heart failure (rats with surgically induced heart failure, micro-embolism induced dog heart failure model). All pre-clinical studies are complete and we are starting to collect data from our first patients. Specifically, we have not observed any adverse events, however, data from biomarkers is favorable (such as a 60% reduction in CPK).

What steps need to be completed before moving into a clinical trial?

We have raised approximately $1 million to conduct P-004, a 10 patient open label trial at Cincinnati Children’s Hospital with Dr. John Lynn Jefferies as Principal Investigator. All regulatory activities have been completed.

What is your best estimate for the length of time it will take to move this research into clinical trials?

We expect the trial at CCHMC to begin in May or June 2018.

Where would a clinical trial take place?

We are already collaborating with leading research clinicians and have verbal commitments from several to conduct the first trials. High likelihood centers include Cincinnati Childrens Hospital (John Jefferies, supported our pre-IND meeting), and the University of Tennessee Health Science Center (Jeff Towbin, also supported our pre-IND meeting). We are also in discussions with Cedar-Sinai in Los Angeles.

Who would be eligible to participate in a clinical trial?

Carmeseal-MD is expected to be useful for all patients with Duchenne or Becker, regardless of genetic mutation. We expect enrollment of non-ambulatory patients with early cardiac and respiratory dysfunction. This approach maximizes our chances of seeing a positive effect and provides a clinical trial opportunity for patients who cannot perform the six- minute walk test.

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