Thanks for your post & in regards to funding a Ph3, there are large benefits for Biotechs/Pharma for Orphan status trials, which makes them attractive to Big Pharma albeit it rare disease, so just wanted to add to you comments. These include tax concessions and Orphan status grants.
Also the trial cohort is not expected to be as large in rare disease, as we have discussed previously. I know as a LTH you know all of this, so the below link on Orphan Status with the FDA is for the benefit of newer investors here:
The main takeout/s I got from the article posted by @Thuynh, thank you , is that Dimerix have a choice of whether to run their own Ph3 for FSGS or to partner. This puts us in a better position (that CR was necessary, much to some disappointment at the time).
They ran through the pros and cons, and it looks like they want to start on a Ph3 ASAP for this indication, which we already knew from preparation commencing over a year ago now. What we do know is that Dimerix have ensured we are ready to go on good results from Ph2A in this indication.
While there has been a major focus on the larger DKD trial & while this indication showed excellent efficacy in responders in Ph2A, I don’t think we should discount the possibility that FSGS could also be highly successful and I thought it was interesting regarding the Ibersartan dose being standardised to 300mg, this actually could make a difference to numbers in the combined treatment (extensive global patents for combination treatment with CCR2 & ARB).
We are already aware that there were no SAEs, and as you rightly said, the primary endpoint was safety & tolerability, it looks like a tick for that from recent ANNs. The interesting thing will be the secondary endpoint of a reduction in proteinuria & symptoms of disease.
Patients were asked to keep a diary during the trial which are usually a standardised set of questions to be answered on each trial visit. These are usually an E-diary (an app, or a mobile phone like device, where the data goes directly to IQVIA) or a paper diary, where the data is entered by the trial site to the CRO (IQVIA). QOL is also important for a chronic incurable disease.
While our study was in adults >18 years old, FSGS affects children also and this was mentioned by Dr Nina Webster in the article. If safety, tolerability and efficacy were good in adults and remains that way through Ph3, it would be great to see some option for treatment in the paediatric population.
High dose steroids are an awful thing and have long-term serious side affects, especially for growing children. These kids are put on dialysis as well & on the transplant list. I think this also really highlights what Dr Brad was saying about anything above 15% improvement is clinically significant and also the ethical considerations of starting a Ph3 ASAP.
From recent reading, and data from the National Kidney Foundation in the USA, patients are skipping haemodialysis appointments, Drs appointments & hold-ups on kidney donation and transplant wait lists due to COVID-19. So the situation will only worsen & similar in the UK, both for DKD & CKD.
The Dimerix article appears to confirm that FSGS patients are accessing long term compassionate treatment with DMX-200 post trial, which was included in an ANN recently in regards to the FSGS trial.
In regards to a TO, partnering or Dimerix going forward with FSGS alone on positive trial results, it’s anyone’s guess. I think the DKD trial may have some impact on what happens here, and also COVID-19.
We really don’t know much about DMX-700 yet, which I think is to do with pending patents, but being for COPD, which is an indication that is a common disease state already, but could become far more common post COVID-19 recovery. The Receptor-HIT platform also part of the pipeline.
Our pipeline in relation to COVID-19 is looking really relevant to this global pandemic. If you look at primary & secondary outcomes in the REMAP-CAP protocol for ARDS, a secondary outcome is for Renal, which we know is a major risk factor for mortality for Diabetic & CKD patients (articles posted previously from the UK & USA in mortality rates).
Unfortunately what is happening in VIC and NSW, we may have quite a large cohort of patients now to include for DMX-200 trials. When I say unfortunately it’s not the drug, just that people may be needing it with infection rates.
Our trial will be to focus on ACE receptors in the lungs & most likely where they are more prolific in the kidneys, potentially as a secondary endpoint & dampening down the inflammatory viral affect of COVID-19. It’s interesting because ACE-receptors have long been identified as a viral receptor for COVID-19. The RAAS system, which regulates blood pressure from the kidneys, is also a factor in mortality from COVID-19 (stroke etc from hypertension).
GLTAH
DXB Price at posting:
38.0¢ Sentiment: Buy Disclosure: Held
, is that Dimerix have a choice of whether to run their own Ph3 for FSGS or to partner. This puts us in a better position (that CR was necessary, much to some disappointment at the time).
(20min delay)
