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Message: The Complement System - Beacon Nov 17 2017

I just updated myself on the current posts.

The following is not news but it is a new emphasis for me in a time when we wait for the SSRA.

I’ve been sorting RVX files and came across the Beacon report from Nov 17th, 2017 and re-read it.

 

What struck me in this read is Dr. Kideckel’s statement on page 16 (see below) that states that they (Beacon) are most excited about the potential for apabetalone in complement biology (he states this even though RVX is focused primarily on CVD and CKD at this stage as is the $8.55 NPV target in the Beacon coverage).

 

I had assumed that all of the various indications outside of CVD and CKD were just side issues (wonderful surprises within the multimodal MOA of apabetalone but…) to be pursued when and if the financing and time allows for experiments in these areas. Now, if Dr Kideckel is correct, it seems to me this would be a primary area for Don make progress via licensing deals with other biotechs to move these trials forward.

 

Perhaps Dr. Kideckel’s experience at Alexion Pharmaceuticals has influenced his enthusiasm (bias???) (expressed on page 16 below) for the potential of apabetalone in complement related diseases.

 

Some posters have expressed frustration at the lack of progress by RVX on other trials such as Fabry disease, etc and now I understand their frustration better. However, the reality is that the big players did not step in to take this trial (BoM) to the finish line so Don fought hard for the $30 million loan and resolved both the financing (allowing the trial to continue hopefully until a positive SSRA) and the TSX listing issue (thus retaining liquidity for our shares). Great work Don and congratulations.

 

Obviously none of this is new information. The reason I posted it is because my thinking had been focused on Beacon’s NPV of $8.55 and a peak sales year in 2025 at 2% market share. As has been discussed by others many times given that apabetalone is directed at the 70% of the CVD patients with unmet needs the 2% market share seems absurdly low.

 

But that is not my point. Even if I accept the $8.55 NPV with all of the (possibly) extreme highs and lows on the journey there the re-read of Beacon based on Dr. Kideckel’s experience at Alexion Pharma I am now hopeful that there may be far greater potential for apabetalone (and possibly other RVX compounds) beyond the CVD with DM and low HDL markets. Previously I had thought the other indications were just nice extras as opposed to being central priority markets.

 

The vote of confidence by the TSX that RVX is a viable ongoing company is a big vote of confidence. I am now hoping for smooth sailing through the SSRA and that it is an SSRA as opposed to a bSSRA (blind SSRA - which from the BDAZ post at 11:04 of May 11 I have interpreted an analysis that looks for nuisance factors such as patient discontinuation rates, statistical variance, etc but does not examine treatment effects). The reason I hope it is an SSRA is because this approach examines the treatment effect against pre-specified criteria. As has been stated many times, if clear sailing at n= 188, then it implies (in my interpretation that there is a reasonable probability (not a guarantee) of hitting the BoM of 3 point MACE target at 250 events. Thus it removes a degree of uncertainty and thus could move perceptions on the odds up and could possibly improve the share price.

 

From what I recall during the time of all of the discussions about when the FA will happen Don always referred to a SSRA and not a bSSRA so I’ll assume it will be an SSRA.

 

Uncertain times but that is always to be expected.

GLTA – we are getting close.

Toinv

 

 

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Source - Beacon Nov 17th, 2017

Page 3

New Analyst joins Beacon SecuritiesLimited: Dr. David M. Kideckel, BA, BSc, MSc, PhD, MBA

…………..Most recently he served on the North American and European Medical Affairs team for Alexion Pharmaceuticals (ALXN-Q), the world leader in rare and ultra-rare diseases, with a focus in Complement-mediated diseases. His industry experience spans nephrology, neuroscience, general and pain medicine, specialty pharma, hematology, otolaryngology and ophthalmology.

Dr. Kideckel completed his Doctorate in neurosciences at the University of Toronto’s Institute of Medical Science (IMS), spanning cross-departmental synergies in brain imaging, behavioral and cognitive neuroscience as well as movement disorders

 

Page 15

While these therapeutic areas (CVD, CKD) hold great market potential in and of themselves, Resverlogix is also positioning itself to become a leading Complement-mediated Disease with an extensive R&D program focused on this.

 

Given my previous experience at Alexion Pharmaceuticals, presently the world’s most profitable “Complement” company and the fact that Forbes has consistently named Alexion “one of the world’s most innovative companies,” I see a huge opportunity here (more on this below). Resverlogix is also presently conducting pre-clinical research occurring in rare diseases, known to be caused by Complement, with Phase 2 studies starting in early-mid 2018 (eg Paroxysmal Nocturnal Hemoglobinuria [PNH] and atypical Hemolytic Uremic Syndrome [aHUS]).

 

Page 16

What is Complement Biology?

Despite clinical trials in CVD (Phase 3) and CKD (Phase 2a) being further along the clinical pathway, we in fact, are most excited about Apabetalone’s potential in Complement biology. The Complement system is part of humans’ innate immune system and when it doesn’t function properly, the consequences to humans’ health can be impacted severely, often resulting in morbidity and death.

 

The Complement system is comprised of more than 30 proteins and can be activated by any one of three separate pathways: classical, lectin and alternative – all three of which lead to a three of which lead to a common part of the terminal pathway at the level of C5.

GLTA

Toinv

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