BioBlurb

Biotech analysis.

[BioBlurb @BioBlurb]

$SRPT Correction:

Casimersen path will be informed after Golodirsen CRL meeting with FDA. Accelerated approval (AA) for Casimersen was planned and may still be possible.

This won’t effect Microdystrophin timeline as AA wasn’t planned.

27:50 SoundCloud:
https://t.co/L5H1XHd9Z1

[BioBlurb @BioBlurb]

$SRPT @Sarepta Dr. Gilmore O'Neill, CMO: "There are no last thoughts���there are only future thoughts."

[BioBlurb @BioBlurb]

$SRPT CEO reiterates the CRL for Golodirsen does not change the approach or timeline for follow on exon-skipping.

Waiting on data coming early 2019 before deciding whether to dive into PMO or PPMO for the next set of pre-identified exon targets for ~40% of the DMD population.

[BioBlurb @BioBlurb]

$SRPT CEO concerned with releasing CRL before their formal communication with the FDA as issues in the CRL were issues outside of (not brought up by) the reviewing division. They want to understand why approving authority raised the issues to begin with since it was news to them.

[BioBlurb @BioBlurb]

$SRPT CEO clarifies the surprise to "the 2 safety concerns [...] were not vetted or raised by the division [that reviewed the drug]."

[BioBlurb @BioBlurb]

$SRPT Microdystrophin regulatory approval would have to go through a different division, not CDER where Eteplirsen and Golodirsen were reviewed.

[BioBlurb @BioBlurb]

$SRPT @ParentProjectMD President: "Our community isn't good at sitting around and waiting. What can we do to help–to be supportive?"

@Sarepta CEO: "The obligation is ours." Reiterates it's the company's job and they will not abandon their responsibility.

[BioBlurb @BioBlurb]

$SRPT "We have no plan on abandoning Vyondys, or Casimersen, or PPMO [...] we stand resolute."

[BioBlurb @BioBlurb]

$SRPT EMA progress: Accelerated approval with a biomarker doesn't exists in the EU like it does in the US. Therefore, ESSENCE would be need to finish before seeking EMA approval.

Conditional approval can only happen with functional data.

[BioBlurb @BioBlurb]

$SRPT Sarepta will obtain formal FDA communication within 60 days.

[BioBlurb @BioBlurb]

$SRPT Sarepta is committed to continuing the ESSENCE study. No comment about PPMO specifically but reaffirmed commitment to pace.

[BioBlurb @BioBlurb]

$SRPT Sarepta replies to compassionate use question for Golodirsen since patients have been waiting so long already and many won't be able to wait until 2024:

"For compassionate use we’d have to consider over time. Right now there’s not an active compassionate use policy."

[BioBlurb @BioBlurb]

$SRPT All data, including the home videos, will be collected in the ESSENCE study and included in the full package of data in the future.

[BioBlurb @BioBlurb]

$SRPT Cannot do an interim readout of ESSENCE because then they'd have to enroll more patients and push the end date late later than it already is. Notes it wouldn't be the best solution.

[BioBlurb @BioBlurb]

$SRPT CEO: "I am not comfortable making the CRL public because it may be disrespectful to the agency as we work through the issues."

[BioBlurb @BioBlurb]

$SRPT The study submitted to FDA had mainly safety and tolerability. If the FDA now requires functional data it'll require the readout of the ESSENSE study. The functional data in ESSENCE won't read out until 2023, or possibly 2024.

[BioBlurb @BioBlurb]

$SRPT CEO @douglas_ingram "I want to tell all the families how deeply sorry I am for not receiving an approval on Monday."

[BioBlurb @BioBlurb]

$SRPT Dr. Jerry Mendell will provide follow up functional data on 2 or 3 LGMD patients at the WMS (1-5 October 2019).

[BioBlurb @BioBlurb]

$SRPT CEO notes that $AVXS $NVS has gone back to Paragon to support their commercial GTx manufacturing.

Sarepta has long-term arrangements for large dedicated commercial manufacturing facilities through Paragon.

[BioBlurb @BioBlurb]

$SRPT The study 2 expansion from 24 to 40 patients will include the same type of patients (~7 age and ambulatory) as planned in the original cohort.