Replies to post #221407 on Biotech Values

[jmarce]

FCSC I've look at the data but I agree the cap structure is a mess and I wouldn't consider investing at this time.

How much read through do you think you can get from a non-disease modifying treatment like SD-101 into the current gene therapies in the clinic? From my understanding temporary wound healing with non-disease modifying tx like SD-101 is possible but without the anchoring fibrils it won't be durable for any significant amount of time.

If you look at SD-101 2b 24 month extension it seems hard to call that clinically sig despite the wound closure in the 1-3month range:
BSAi of lesional skin: percentage of total body coverage of epidermolysis bullosa–related lesions
baseline: 24.4 ± 19.4
change at 24 months: -2.1 ± 9.84

And from the ABEO paper:

In this study, wound healing was accompanied by full-length type VII collagen expression in the dermal-epidermal basement membrane. Wound healing was also associated with the formation of anchoring fibrils that were not present prior to grafting.

I'd like to see what happens over a longer time frame with these initial 2 in the first cohort for KRYS but like I said in previous post the combination of anchoring fibril presence along with durable wound closure so far (longer follow up need 1yr+ to confirm) is what makes me optimistic. I'm also taking into account method of administration and cost compared to competition which seems to significantly favor KRYS even assuming all else being equal. Still a lot of risk until we get larger N and longer follow up but that's why it's a relatively smaller position for me.