Primary mitochondrial myopathy as a group affects 1 in 5000. It’s clear Elamipretide is increases exercises tolerance and perhaps can enhance performance in healthy individuals. Despite not meeting the end point of recent phase 3 (which basically only measured one performance metric of walking and was way underpowered when considering all covariates) there was clearly a signal of better physical performance. It’s demonstrated to be safe and no serious AEs whether as IV and SQ. And for Barth Syndrome, it’s essentially FDA approved pending an unexpected catastrophe. Awaiting official announcement and SP will take off. I think Mito has just started to tap into the potential of this preFDA approved drug. And they are working with highly respected clinicians from MGH, UCSD etc.. expect already a BO target from BP.
jpjazzkat
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I get the challenge and approach these guys face/are taking coming from both academia translational/clinical trials background and a SGmO investor myself (20+ yrs background). In many ways, they are speaking the language of scientists & clinicians. The reality is, this technology is well grounded and they’ve developed a solid platform that will be the gold standard in this industry soon and years to come. They aspire to meticulously fine tune it even more, tho it comes across as R&D without deliverables. From clinical trial design, publications, abstracts, prior announcements n today’s RND talk, I respect the focus on making sure this works in patients (which it does despite the downplay of recent ASH data - seriously AE from DSMO is unrelated) for long term viability and value. Reading between the lines this company
Their Pfizer/Sanofi alliances are smart and translates to immediate & long term sustainable revenue. And makes a very appetizing gem for potential M&A (wink).
This will be at $18-24 in a year. It’s basically a question of when folks will jump in and how much they wanna profit.
MITO
SGMO