PER 13.5% 6.4¢ percheron therapeutics limited

As far as half-year reports go there is normally a bit of...

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    As far as half-year reports go there is normally a bit of generic filler and reiterating.
    But of this report I find the below to be the most interesting iterations, to remind us of potential, and science.
    My focus is here because it's the pertinent aspects, and potentially the base of our future, and is controlled by science, not the market environment.
    The current share price means nothing in reality, but this below could mean everything, so (rhetorically) what is it worth us focusing on?

    A prior Type C guidance meeting held with US FDA provided the Company with clarity on the requirement for the chronic monkey study and design of a Phase IIb/III trial for the US. Given the apparent high-level alignment between EMA and FDA on Phase IIb/III study requirements, the feedback from the FDA provides the Company with the opportunity to engage with the agency to streamline the regulatory processes and to the extent possible harmonize the Company’s overall global clinical development plans. The Company considers that it has potential optionality in its actions with FDA including to take the EU Phase IIb/III data to the FDA to be assessed as supportive data for a future marketing application or should the data warrant it, possibly an approval of ATL1102 for DMD without further trials.

    Statistically significant mean modulation at 24 weeks compared to baseline in Thrombospondin-1 (TSP-1) and Latent TGF-beta-binding protein 4 (LTBP4) levels, two proteins that modify the rate of loss of ambulation in DMD. ATL1102 modulation of these two DMD disease modifier proteins known to impact TGF-β and the rate of loss of ambulation in DMD patients supports ATL1102’s potential use in ambulant patients with DMD, and as an agent to reduce fibrosis in other human diseases

    Increase at 24 weeks in plasma VCAM-1 supportive of the ATL1102 mechanism of action of reducing CD49d on the surface of cells to which soluble VCAM-1 is bound, and in CXCL16 which can promote muscle regeneration appear to align with the positive effects on muscle structure observed under MRI in the ATL1102 Phase II trial. These plasma proteins were increased such that they approached the median levels seen in an external control dataset of health adults, supporting the beneficial nature of the outcomes in ATL1102 treated DMD patients. Positive effects on LTBP4 and TSP-1 positions ATL1102 as an exciting prospect for the treatment of both non-ambulant and ambulant patients with DMD and the treatment of other muscle and fibrotic conditions. The protein changes observed in the plasma of the ATL1102 treated non ambulant DMD patients in the Phase II study is also consistent with the drug’s positive effects on muscle function and strength reported in the ATL1102 Phase II trial.
 
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