Thanks RYNZN, and then there is the question of the strategic value of the patent application. There is no doubt that PER is aware of the critical importance of combination therapy for therapeutic intervention in DMD. To gain an understanding of management thinking, reference is made to the interview given by the Lead Scientist for PER at the recent 2024 Muscular Dystrophy Association Clinical and Scientific Conference.
In his discussion of immune mediated damage i.e. the secondary cause of damage resulting from the loss of the dystrophin protein, the comment was made that by keeping muscle intact...there's more places for where dystrophin restoration agents can actually create dystrophin. The comment provides clarity in relation to the outcomes from the study in mice models. How does ATL1102 and dystrophin restoration agents work together to enhance outcomes? The take home message appears to be that the amelioration of immune mediated muscle damage increases the amount of functional muscle for the production of dystrophin by dystrophin restoration agents. I think we understood that but I hadn't heard it expressed in such an insightful way.
Lets return to the question of the strategic value of the patent but in the context of the out licencing of ATL1102 or a partnership agreement. Its hard to conceptualise holding discussions with prospective partners for the use of ATL1102 as a monotherapy without addressing the use of ATL1102 in combination therapy.
One can only speculate but it is somewhat tantalising. We all appreciate the perceived importance of the patent in relation to the gorilla in the room. However, what if the partnership is with a big outside player who wants to move into the DMD space? Perhaps, a partner with global reach, commercial presence and regulatory expertise.
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