Biotech Autist

$REPL Quiet now.

@blindad3 i am still leaning yes but am less sure about the timing. i bought may 15 puts so i am worried about it too haha

@BiotechAutist Has exceeded the 60 day response and still waiting to hear but shake up in the fda with makary .. do you think rtf still incoming??

@BiotechAutist what are your thoughts on Immunity bio (IBRX)?

@DavidCh34622985 PUL can be meaningful and still not answer a cardiomyopathy CRL. I could be wrong, that's why I appreciate discussions like these.

@BiotechAutist You do realize that the PUL is the golden functional benchmark for DMD so it’s not only hard to hit but even more clinically meaningful. You are wrong on CAPR the same way you are wrong on DNLI, you assume zero flexibility on FDA side.

1/ FDA vs $CAPR at their words 🧵

@blindad3 I don't think it will be approved.

@BiotechAutist And thoughts on approval ?

@DavidCh34622985 harder to hit isn’t the regulatory standard. the CRL asked for cardiac primary evidence. hope 3 doesn't have it. you nailed it.

@BiotechAutist Well, neither it is for hope 3, you do realize that the hope 3’s PM is even harder to hit than the cardiac which is also hit in the 2nd endpoint. You do realize that? Do you?

@bronxb_13 no. the point is simple: you said that FDA accepted the SAP. the quote is “we received no comments.” those are not the same.

@BiotechAutist So your short premise is the fda didnt explicitly give written agreement or denial on the sap so they will be issued another crl after a successful trial on both endpoints ? The fda doesnt always give written agreement esp in rare disease

@bronxb_13 no. you’re confusing IND “may proceed” with SAP acceptance. 21 CFR 312.40 = a trial can proceed after 30d absent clinical hold. it doesnt deem a SAP adequate for approval. for endpoint/planned-analysis concurrence, FDA gives written agreement.

@BiotechAutist Yeah that’s how it works by fda statute, if they don’t respond to it within the allotted time period it’s deemed accepted

@bronxb_13 it gets way better. stay tuned

@BiotechAutist your logic is essentially that the primary endpoint wasn't cardiac so no approval for you?

@bronxb_13 watch the topline call. Linda said “We received no comments on the statistical analysis plan.” then later: “silence is assent or no news is good news.”

@BiotechAutist they wouldn't unblind hope-3 without having FDA confirmation the trial was adequate for approval. they got the confirmation they needed and then acceptance of the SAP. they hit on both primary (skeletal) and secondary (cardiac).

@DavidCh34622985 litigation is not a cardiac primary endpoint

@BiotechAutist They just filed a lawsuit trying to get ride of NS the partner. 8K released an hour ago. Why bother to fight a right for a failed asset? Read between the lines, it will be approved.

@DavidCh34622985 You'll see

@BiotechAutist Well it’s CEO’s job to spin a message however they see fit… but phase 3 reached end point. I don’t know why these back and forth even matter now

10/ CAPR, March 10: FDA “has lifted the previously issued Complete Response Letter and resumed review” Fierce asked HHS what that meant. HHS: the new review constitutes a “resubmission.” then when asked what “lifted” meant, HHS deferred to CAPR. then CAPR clarified: “there was no special agreement or unusual process involved.” so “lifted” meant: they submitted a response and FDA restarted the clock.

9/ FDA CRL, on what to do next for cardiomyopathy approval: “we recommend that you conduct adequate and well-controlled study(ies), whose primary objective is evaluation of cardiac outcomes” CAPR, July 11: “Capricor plans to submit data from the Phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness from an adequate and well-controlled study.” see the problem?