good write up, I used to love the peter marks trade, got a rare fatal disease state and a therapy well sir you now are able to use your p1/2 with no controls for the AA pathway. I've stopped playing the trade with pm's departure. I was long capr twice and have exited. If VP does miraculously approve this therapy I will 100% go back to investing in qure, lexo and others. I see this as kind of a litmus test. However for my risk profile, and due to the cost of shorting it I'm on the sidelines with popcorn.
Gave a hint Like in one Investor call Said Something Like
We needed to Express IT Like that because of our Partner , so we Said between 30 and 50 , you can do the math ( and Kind of winked)
So 40% IS indeed a reasonable guess
Also please be aware the company will additionaly bei compensated for Produktion costs , the ceo Said that number should BE around of 10 % of list price ( If i remember right)
Also CEO IS in Public record that Phase 3 Data IS ready in early August If needed
While I respect the analytical depth here, I believe some critical context is missing.
Capricor’s Deramiocel is not just another DMD drug it’s the first to show sustained cardiac benefit, which addresses the #1 cause of death in this population. The HOPE2 and OLE data demonstrate durable improvements in LVEF over several years. While not perfect, that level of consistency and safety profile stands out.
FDA accepted the BLA and granted Priority Review, which signals internal acknowledgment of unmet need and potential clinical benefit. While sample size and design adjustments in HOPE2 are fair to question, they were transparently handled, and the totality of data including quality of life and biomarker trends adds weight.
The absence of an AdCom isn’t inherently bearish. Many approvals, especially in rare diseases with clear unmet needs, proceed without one particularly if the FDA doesn’t need external input.
Also worth noting, Sarepta and Capricor target very different aspects of DMD. The cardiac focus of Deramiocel, supported by 4+ years of safety and efficacy, fills a gap that even gene therapies do not currently address.
Whether one is bullish or bearish, the broader picture deserves full context. Respectfully, this might be one of those cases where absence of perfection doesn’t mean absence of impact.
nice write up, thanks for your thoughts. as far as the royalty goes, it's really not uncommon to put language like that. it's most likely a tiered royalty agreement that goes up with sales although i agree this wording is super weird. they usually keep it vague because they don't want to face the public scrutiny of this arrangement when other comps come up (which will also be vague)
Great write-up, thanks!
good write up, I used to love the peter marks trade, got a rare fatal disease state and a therapy well sir you now are able to use your p1/2 with no controls for the AA pathway. I've stopped playing the trade with pm's departure. I was long capr twice and have exited. If VP does miraculously approve this therapy I will 100% go back to investing in qure, lexo and others. I see this as kind of a litmus test. However for my risk profile, and due to the cost of shorting it I'm on the sidelines with popcorn.
The capr CEO
Gave a hint Like in one Investor call Said Something Like
We needed to Express IT Like that because of our Partner , so we Said between 30 and 50 , you can do the math ( and Kind of winked)
So 40% IS indeed a reasonable guess
Also please be aware the company will additionaly bei compensated for Produktion costs , the ceo Said that number should BE around of 10 % of list price ( If i remember right)
Also CEO IS in Public record that Phase 3 Data IS ready in early August If needed
While I respect the analytical depth here, I believe some critical context is missing.
Capricor’s Deramiocel is not just another DMD drug it’s the first to show sustained cardiac benefit, which addresses the #1 cause of death in this population. The HOPE2 and OLE data demonstrate durable improvements in LVEF over several years. While not perfect, that level of consistency and safety profile stands out.
FDA accepted the BLA and granted Priority Review, which signals internal acknowledgment of unmet need and potential clinical benefit. While sample size and design adjustments in HOPE2 are fair to question, they were transparently handled, and the totality of data including quality of life and biomarker trends adds weight.
The absence of an AdCom isn’t inherently bearish. Many approvals, especially in rare diseases with clear unmet needs, proceed without one particularly if the FDA doesn’t need external input.
Also worth noting, Sarepta and Capricor target very different aspects of DMD. The cardiac focus of Deramiocel, supported by 4+ years of safety and efficacy, fills a gap that even gene therapies do not currently address.
Whether one is bullish or bearish, the broader picture deserves full context. Respectfully, this might be one of those cases where absence of perfection doesn’t mean absence of impact.
nice write up, thanks for your thoughts. as far as the royalty goes, it's really not uncommon to put language like that. it's most likely a tiered royalty agreement that goes up with sales although i agree this wording is super weird. they usually keep it vague because they don't want to face the public scrutiny of this arrangement when other comps come up (which will also be vague)
Sure, I know how tiered royalties work, this read to me as a fixed royalty that we simply cannot know, but maybe I’m misunderstanding