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@waynesworld

One thing ANP have done right is locking in those patent applications, it's quite a portfolio now covering DMD and LGMD.

You are right in that Sarepta could just wait for a suitable combo drug to come onto the market...but time is against them.

Sarepta are yet to have a successful P3 program approved by FDA.

Exondys was fast tracked based on surrogate endpoint of 'increased dystrophin' - NO real functional results, no PUL2, no plasma protein data, no Fat fraction %...

We know that increased dystrophin alone won't help the boys once the cycle of fibrosis sets in by the age of 5 or 6.

Exondys brought in over AU$1B of revenue for Sarepta in the last year.

Will FDA approve SRP-9001 yet again on the surrogate endpoint?

It appears the FDA staffers in the story this week thought SRP-9001 should be rejected based on poor functional data...before senior FDA management intervened with an Advisory Committee meeting for May 12?

It's unprecedented - if the FDA approves two drugs based on surrogate endpoints without functional data, there is going to be some serious noise from the industry.

On the other hand, FDA wants to encourage Gene Therapy investment, particularly for unmet orphan indications...this is a strategically important decision for them.

Adding an ATL1102 combo arm to their SRP-9001 Endevour program might just help both Sarepta and the FDA with their respective goals.

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