Author Topic: Joint NAC/GSK response to patient inquiries  (Read 3158 times)

Miriam Vered

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Joint NAC/GSK response to patient inquiries
« on: January 18, 2016, 12:03:48 PM »
I've attached the official NAC/GSK response to patient inquiries regarding the CPHPC plus anti SAP trials.

tchilds

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Re: Joint NAC/GSK response to patient inquiries
« Reply #1 on: May 22, 2019, 08:09:10 PM »
How is this trial progressing? I've recently read a document under the 'Trials' section of this website which suggests that GSK terminated this trial. Is this correct?

Miriam Vered

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Re: Joint NAC/GSK response to patient inquiries
« Reply #2 on: May 22, 2019, 08:57:21 PM »
Yes, this is correct- see the explanation on page 2 of the last NAC newsletter.
Also, this is Professor Sir Mark Pepys' reply to your post:


Sadly the information you have seen is true.  I am very sorry indeed about your disappointment.  I share it intensely myself.  I first suggested the targeting of SAP as a possible treatment for amyloidosis in 1984 and have been working ever since to invent and develop treatments to achieve it.

GSK’s decision to terminate the programme was based on their risk/benefit analysis of results in patients with cardiac amyloidosis in the phase 2 clinical trial that they devised and ran.  GSK returned to me my patents on the invention that they had licensed and I am currently considering whether it may be possible to restart a development programme.  We have a much more optimistic view of the treatment but there is no doubt that, even if I am successful in finding the necessary investment, development will have been delayed for at least some years.

The unfortunate situation we have encountered is not only not unusual in drug development, it is actually what usually happens.  About 95% of all drug discovery and development programmes fail or are terminated.  There are many factors, not least the enormous cost of bring a new medicine all the way from its original invention to its commercial market.  This is about $1 billion.  The pharma companies thus continually have to make very challenging commercial decisions in which the medical need is not the only, or indeed, necessarily the major factor.  For example, at more or less the same time that GSK terminated our programme, they also divested their entire rare disease portfolio, choosing to focus their resources on other areas of R&D.

It will be good if I can get traction towards restarting our anti-SAP programme.  Meanwhile I have a different immunotherapeutic approach to amyloid removal that we have shown to work well in the same mouse model that so perfectly predicted the efficacy of anti-SAP in patients.  I am now trying to assemble a development programme to take this towards clinical trials.  I do not know whether I will be successful, nor whether my new drug will eventually work in patients, but my goal from the outset has been to benefit patients with amyloidosis, improve their quality of life and survival.  I do not intend to abandon this aim now.

Kindest regards

Mark Pepys

Professor Sir Mark Pepys MA MD FRCP FRCPath FRS FMedSci


tchilds

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Re: Joint NAC/GSK response to patient inquiries
« Reply #3 on: May 22, 2019, 10:38:46 PM »
Thank you, Miriam and thank you for Sir Mark's response. I didn't see the last newsletter.

I don't really know what to say other than how desperately disappointing and frustrating for all concerned. I feel very deflated right now.

Miriam Vered

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Re: Joint NAC/GSK response to patient inquiries
« Reply #4 on: May 24, 2019, 08:38:28 AM »
Thank you, Miriam and thank you for Sir Mark's response. I didn't see the last newsletter.



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