Ann: ATL1102 for DMD EMA PIP feedback and FDA regulatory progress, page-58

Hi guys

Thanks for your Concerns I am fine just been watching from the sidelines giving others a chance to air their thoughts LOL

To be quite honest I dont really have much to add from my last post on the 10^th of this month

Since then what have we had

The Pip feedback, so what do I think of this news

what did I take from it

Well lets take a look

PDCO feedback in line with expectations

So looks like they got what they asked for in line with their trial set up and reporting etc etc sounds like a formality in answering what they have asked no major hurdles by the look of it.

I do like this Paragraph they are obviously listening in on HC

The PIP addresses the entire paediatric development program for ATL1102 in DMD (including potential ambulant DMD patient studies)

It does say potential but its an update which is good enough, and I hope readers on this thread realise what a major piece of news this is, if it actually comes to fruition, opens the market right up.
It will be a big plus in the major negotiations that must be going on behind closed doors at the moment, This I would imagine as I have said constantly would be a major requirement for any Pharma getting involved to be able to treat 100% of these boys as the need arises


in parallel the Company continues to progress its interactions with the US Food and Drug Administration (FDA) and has recently submitted a FastTrack Designation Request

Fast track designation applied for which was expected as per FDA ann, nothing new there

Except to say that it was the FdAs feedback that Instructed ANP to apply for fast track, so I think its pretty much a given we will receive Fast track designation

More importantly they go on to say

While the Company’s Phase IIb clinical trial protocol was initially developed to meet EMA’s expectations, the recent feedback from the FDA has provided the Company the opportunity to streamline the regulatory processes in Europe and the US and to the extent possible harmonize the Company’s overall global clinical development plans.

I think this shows the FDA like what they have seen result wise, as in they are trying harmonise our global development (IE FAST TRACK US )

and below highlights this fact

Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.

So once fast track is received where does this lead us

well from the above it looks like far better communication with the FDA in our efforts to move on

but fast track could also well bring about ACCELERATED APPROVAL

So what is this all about how could this benefit us see below

FDA to base accelerated approval for drugs for serious conditions that fill an unmet medical need on whether the drug has an effect on a surrogate or an intermediate clinical endpoint.
A surrogate endpoint used for accelerated approval is a marker - a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit. Likewise, an intermediate clinical endpoint is a measure of a therapeutic effect that is considered reasonably likely to predict the clinical benefit of a drug, such as an effect on irreversible morbidity and mortality (IMM).
The FDA bases its decision on whether to accept the proposed surrogate or intermediate clinical endpoint on the scientific support for that endpoint. Studies that demonstrate a drug’s effect on a surrogate or intermediate clinical endpoint must be “adequate and well controlled” as required by the FD&C Act.
Using surrogate or intermediate clinical endpoints can save valuable time in the drug approval process. For example, instead of having to wait to learn if a drug actually extends survival for cancer patients, the FDA may approve a drug based on evidence that the drug shrinks tumors, because tumor shrinkage is considered reasonably likely to predict a real clinical benefit. In this example, an approval based upon tumor shrinkage can occur far sooner than waiting to learn whether patients actually lived longer

So my interpretation of this is that we would have qualified for this in our last trial as in

Atl1102 targets • DMD patients with greater number of circulating CD4+ and CD8+ T cells with high levels of CD49d (CD49dhi) expression have both more severe & rapid progression of disease2

In our trial the outcome,

ATL1102 demonstrated positive effects modulating CD49d+lymphocytes in the blood during treatment and once the trial finished then their cd49d once more elevated

Would this be classed as a clinical endpoint even though ATL1102 hit its target and reduced the numbers,

I doubt it very much as all it has done is reduce the numbers of CD49d its just numbers

But what ATL1102 did do was under MRI

MRI data suggests stabilisation of percentage of fat in muscles and preservation of functional muscle mass

MRI data confirms the positive changes at a muscular/cellular level and supports the observed physical stabilisation/improvements in muscle strength and function.

I would say right there we have a clinical endpoint

Once more

A surrogate endpoint used for accelerated approval is a marker - a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit,

Also remember prof voits call that to stop progression of the disease is basically unheard of you could more or less guarantee increase in fat fraction and decrease in muscle mass over a 6 month period

So all being well if we can replicate or improve on our initial trial, then I believe we can safely say we can look forwards to ACCELERATED APPROVAL

Moving onto the other benefit of ACCELERATED APPROVAL

Is the issue of a priority review

Well once the trial is finalised and hoping all goes well, having the RPDD gives us the right to apply for a priority review

What I wasn't aware but have since found out, is that gives us the right to a priority review for ATL1102 plus a voucher to sell or combine with another indication that may come to market through our animal studies we have had announced today, thereby boosting the value of any uptake by a third party interested in taking on one of our other indications further

See below

Priority Voucher Program Attributes
Eligible candidates are granted two vouchers and receive priority review for each voucher: the drug winning a voucher for a neglected or rare disease,
AS in ATL1102
and the drug using a voucher for another indication.
I know the story just keeps getting better

Especially after today's Announcement, I now need to catch up lol

So that is the positive and I will end there as I have gone a little overboard with the length of the post, didnt think I had much to ADD LOL

And in light of todays Ann now brings more importance upon the FAST TRACK DESIGNATION

AS IN

A. Qualifying Criteria for Fast Track Designation Fast track designation applies to the drug (either alone or in combination with other drugs) and the specific use for which it is being studied. The term drug refers to the combination of two or more drugs if the combination is the subject of the fast track designation or request. Where appropriate, FDA may grant designation to the development of a new use of an approved drug.

Once you are over this marathon perhaps I will maybe catch up on todays Ann and maybe we can look at the negatives because for all the positives none of this is transferring into market appreciation and yes we know about the science and how hard the board are working ( not being sarcastic with this comment )

will close for now

and thank you once more for your concerns the ones that asked very much appreciated


SEE HOW WE GO