$CAPR resubmission of the deramiocel application accepted by the FDA. New decision date: Aug. 22. https://t.co/mWSyyXg8di
On $CAPR, the primary endpoint was not changed. Those alleging otherwise are incorrect. The mean change on the PUL 2.0 endpoint was 1.2 points favoring deramiocel over placebo. From CEO Linda Marban: "Capricor’s external statistical vendor followed the pre-specified statistical analysis for the protocol specified primary endpoint of the HOPE-3 trial. This was detailed in a statistical analysis plan that was finalized prior to unblinding."
Read our story on the $CAPR HOPE-3 results here: https://t.co/8ql95NDbaj
Deramiocel improved upper-limb function of Duchenne patients by 1.2 points on a 42-point assessment scale, translating into a 54% slowing of disease progression over one year. Deramiocel also slowed the loss of heart function by 91%. Left ventricular ejection fraction, which measures the heart’s ability to pump blood, improved by a statistically significant 2.4 percentage points relative to a placebo. $CAPR
$CAPR CEO Linda Marban told me: “We believe we can answer the issues that were raised by the FDA. We’re going to be submitting these data to the FDA and I don’t really see how they could possibly sling arrows at it.”
BREAKING: Capricor $CAPR says its cell therapy improved heart and muscle function in Duchenne patients The dual outcomes in a Phase 3 study will likely bolster the treatment’s regulatory future https://t.co/8ql95NDbaj via @Jasonmmast and me
$CAPR halt. HOPE-3 results on deck. Standby....
Next week: We will certainly see $CAPR and $NVO study readouts, right?
$CAPR no HOPE-3 data tonight with earnings release. Study results "expected in the coming weeks."
$CAPR regulatory update after FDA meeting: HOPE-3 study primary endpoint unchanged. https://t.co/26IzYAiMM1
$REPL +25% today on the WSJ editorial bashing (again) Vinay Prasad? Super interesting. Reminder, I will be interviewing Replimune's CEO on stage at the STAT Summit Oct. 15. Joining us onstage will be $CAPR CEO. Info/tickets https://t.co/Ls3j1cYQ1p
Hey folks - FDA has posted more Complete Response Letters, including those sent to $CAPR and $REPL. My quick read doesn't reveal any issues / deficiencies not previously disclosed. You can find the new CRLs here: https://t.co/n2VKUet580 Let me know if you see anything important that I missed.
The STAT Summit is Oct. 15-16 in Boston. We'd love to see you in the audience this year. To entice, we posted a preliminary agenda, with more speakers to come. https://t.co/DJjPFoIMA3 I'll be moderating a panel titled, "Biotech Faces FDA Uncertainty" featuring the CEOs of $CAPR and $REPL + one more newsworthy CEO who we hope to confirm soon. This will be a VERY topical conversation given all the FDA drama of late. My colleague @ADeAngelis_bio and I will also be onstage to chat with biotech A listers Arie Belldegrun and Noubar Afeyan about the current and future climate for biotech financing. You can register for the STAT Summit here: https://t.co/hvMIKIDdad
Are we sure Vinay Prasad’s ouster is positive for biotech? Some musings in this week's newsletter. A commonly held view of his three-month tenure running the FDA’s biologics division was that he was more obstructionist and inflexible toward cell and gene therapies than one might have predicted, even accounting for his reputation as a conservative data nitpicker. The FDA rejected $REPL $RARE and $CAPR. Prasad went to war against $SRPT His departure means the FDA will revert to its more permissive, flexible ways, the thinking goes, and that’s good for developers of cell and gene therapies, good for makers of drugs for rare diseases, and a tailwind for biotech overall. I’m not so sure. You know me by now. I have a hard time fully buying into the rosy story. What if Prasad’s replacement as CBER chief is worse? By that, I mean a person who is more politically motivated and more in the MAHA, anti-pharma and anti-vaccine orbit of Health and Human Services Secretary Robert F. Kennedy Jr. Read more at link below, plus some thoughts on the road ahead for $SRPT
