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Amryt to add another 48 patients to Phase III trial of skin disorder cream

“The IDMC's recommendation is good news for Amryt as it allows us to continue the trial with only a modest increase in the size of the study”
skin
AP101 is a potential treatment for epidermolysis bullosa, a condition which makes skin fragile to the faintest touch

Amryt Pharma PLC (LON:AMYT) is to enrol another 48 patients into the Phase III trial of its AP101 skin disorder treatment.

The cream is being developed to treat epidermolysis bullosa, a rare, inherited condition which occurs predominantly in children and makes skin fragile to even the faintest touch.

It is chronic, potentially disfiguring and in some cases fatal. There are around 500,000 people living with EB worldwide and there are currently no approved treatments.

READ: Amryt opens first US site for phase III skin disorder trial

After analysing interim efficacy data, the Independent Data Monitoring Committee (IDMC) has recommended Amryt continues the study but with the addition of 48 new patients.

That will take the total up to 230 and the larger pool should yield better, more reliable data further down the line.

The additions do mean that the read-out date has been pushed back to the second half of this year. Amryt was previously on track to publish the results in the second quarter.

“The IDMC's recommendation is good news for Amryt as it allows us to continue the trial with only a modest increase in the size of the study,” said chief executive Joe Wiley.

“This brings us closer to potentially delivering a treatment for patients with EB. Today's news is a significant milestone for Amryt and our shareholders in building towards our vision of becoming a global leader in rare and orphan diseases.”

Why is this good news?

The company and analysts on the afternoon conference call are taking today’s outcome as positive news. But why?

Well, there were three options available to the IDMC after it analysed the data. It could have:

  1. Told Amryt to carry on with the trial as is
  2. Told the company to stop the trial as it was clear the cream wasn’t working
  3. Or done what it did and tell Amryt to carry on but increase the sample size

Option two would have been the worst possible outcome, but that was unlikely as AP101 had already showed efficacy in an earlier trial.

Option one would have been the optimum outcome, but analysts say that was also unlikely as the lack of historical data surrounding EB treatments and patients meant there were lots of variables surrounding the study.

In other words, Amryt would have to have been really lucky and/ or psychic to design the trial perfectly without any tweaks needed to be made as it went along.

Analysts agree that option three was the most likely option. The fact that the IDMC went with it is good for Amryt because it suggests they are encouraged by what they have seen, whilst acknowledging that some improvements can be made to boost the chances of success at the end.

After initially spiking higher, shares eased 1% lower to sit at 16.9p.

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