Capricor (CAPR) Final Countdown
By way of introduction, I’m a veteran in the Life Sciences industry and manage a narrow portfolio of select stocks in this sector. The investing philosophy is that there are clear winners and losers in this sector and I use my expertise to focus on select investments that are attractive based on science, valuation and positive triggers. I’ve been a frequent poster on Stocktwits (https://stocktwits.com/LineBacker04) and was invited to post here by the Kamet Resesarch team (thanks guys!). This is my first post on the Kamet Research website and serves as an update on what’s currently going on with Capricor Therapeutics.
There has been plenty of speculation lately on the Capricor board on the potential triggers related to a Type B meeting request with the FDA. What I’m putting together here is a high level discussion on what exactly is going on and what investors should potentially expect. Before I do that, let’s put some dates on the whiteboard.
- May 13th 2020 -> CAPR announces Positive TOP-LINE FINAL results from HOPE-2 (note the emphasis on “TOP LINE FINAL” – these are preliminary FINAL results)
- March 16th 2021 -> Filed an intent to raise capital in the future through an unactivated S3
- May 13th 2021 -> One year Anniversary of the HOPE-2 results $CAPR holds a earnings call
- June 15th 2021 -> Capricor activates the S3 offering
- June 23rd 2021 -> Linda presents DEMO on DMD OLE Patient after ONE infusion
To see the exact transcript of the earnings call, go here -> https://seekingalpha.com/article/4428520-capricor-therapeutics-inc-s-capr-ceo-linda-marban-on-q1-2021-results-earnings-call-transcript). In this call the following statement was made by Linda – (again no speculation here, it’s there in the transcript link I just posted)
“…Now the path forward for CAP-1002 and DMD continues to be very exciting. And I would like to update you today on several key events that have occurred recently. First, we have submitted a new data package to FDA based on the complete HOPE-2 data sets and analysis….
…We believe that based on the strength of the clinical data, an expedited path to registration might be warranted. To this end, we continue to explore ways to work with the FDA to accelerate the pathway to approval of the therapeutic for DMD…”
So, the big question here is WHAT DATA? What exactly was presented as ‘new information’ in a package to the FDA on the anniversary of the Hope 2 TOP LINE Final data? With regards to DMD, there can be only two data sets that could possibly qualify as ‘new information’
- A trial continuation that would provide more clarity on HOPE-2 data – the data has been presented at 3 months, 6 months and 12 months – is it possible that the data was allowed to mature longer between say an additional 6-12 months?
- The second possibility is that the “New Data” came from the OLE data of placebo patients crossing over to CAP-1002. This would make sense if the trial was concluded on or around May 2020 and the OLE kicked off then.
Between the two, I believe that the “New Data” set that was sent to the FDA with a request for a “Type-B” meeting was definitely the FINAL Hope-2 data and not the OLE supporting data. This is also the data that Linda and the management team have declared that they would share in a peer-reviewed scientific journal. This makes sense on many levels:
- If this is the case – the “FINAL” data would be under embargo and not be presented at the PPMD conference in June 2021
- This kind of analysis would be OK for the FDA as a ‘Maturing’ of data as opposed to post-hoc analysis that the FDA frowns upon
- OLE data while being supportive in nature is never going to be powered to be statistically significant
So, assuming that the FINAL data set was compiled and sent to FDA on or around May 13th 2021, when did the OLE infusions start? Could the FINAL data have been allowed to mature for an additional 6 months? A full year all the way to May 2021? If we look at the clinical record here -> https://clinicaltrials.gov/ct2/show/NCT04428476 the trial details were submitted on June 2020 with an anticipated completion date of October 2021. The trial also states “…Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9 and 12. Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6 and 9 trial visits, unless otherwise indicated..”. Based off this record, the last data collection should be around September 2021 (allow one month for data compilation), with infusions on – Sept 2020, Dec 2020, Mar 2021, June 2021.
That puts us right on the borderline regarding the ‘TYPE B’ meeting with the FDA and being able to compile a full data set for the FDA to review. There has been no direct communication from the Capricor management, but if the above analysis of timelines is accurate, the true guidance from the FDA can only be expected in September 2021 which would align with the guidance of Q3 that has been communicated by Capricor. Therefore, what we should expect with regard to the DMD updates is:
- The full “FINAL” data on the HOPE-2 trial that is expected to be published in a major peer-reviewed scientific journal.
- The full OLE data that will likely not be public but will be with the FDA in their review of CAP-1002 Accelerated Approval.
- A guidance from CAPR and the FDA regarding AA sometime towards the end of Q3.
There are lots of reasons to be optimistic about positive guidance from the FDA (Change in leadership at FDA, CAPR getting ready for a positive outcome with a massive S3 filing, Manufacturing contract with Lonza, Readiness to vacate existing office space, positive support from PPMD, etc.) but with anything regarding the FDA, there will be uncertainty around timelines and outcomes. So until some movement (like the data publication) the best approach is to ignore the noise and stay patient.
Disclosure: I am long CAPR. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this article.