Here is the third and final episode of this first series of letters dedicated to Sappiens from CXS Therapeutics, the company that invented the first neuroprotective treatment for Parkinson's disease.
If you haven't read them yet, start with the 1st letter , then the 2nd
Focus on the drug candidate CXS 003, the next key steps, and medical and financial perspectives.
A more technical letter, but necessary to fully understand everything and invest, if you wish, with a clear conscience. It's gone.
(As a reminder: second fundraiser in progress, so don't miss the train!)
There's a lot more research on repositioning than you might think — it's a lesser-known but very active sector. The cost here is ridiculously low by:
It's not as massive for all repositions.
CSX 003 is a drug candidate resulting from a synergy of already known drugs that have been used in therapeutic areas for a long time.
They “went around the world.”
The combination and the specific reformulation in a new galenic form will optimize its effect in Parkinson.
CXS003 builds on existing knowledge of these molecules, but their mechanism of action in Parkinson's is new. A neuroprotective effect is expected. Several medicines/active ingredients are used, especially in diabetology.
Key point that has been verified with firms specializing in intellectual property: their use is royalty-free for Parkinson.
Intellectual property is secure, a first patent published, a second one filed to further strengthen the first.
This is one of the key points of this type of project.
Inventive activity is protected, which is always based, and regardless of the field, on elements that are already known.
You don't reinvent printing or the alphabet with every new book.
Based on existing and already marketed molecules, this is a truly inventive activity: dose/synergy/therapeutic/galenic use.
It is the core of registered patents.
Note that in the pharmaceutical industry, data obtained during clinical trials and regulatory files and advances contribute to the strengthening of intellectual property.
In addition to the filing of the various patents, which were challenged by several experts, an FTO was carried out. “Freedom to Operate”: which determines which rights/licences/patents may be an obstacle to the full exploitation of the invention.
It would have been entirely possible to negotiate an agreement for a new use, with the possible beneficiaries.
This could also be the case for other drug candidates in the future and it is a common practice.
For CXS 003, this is not the case. Inventive activity is not subject to no restrictions of use in the field in question.
A large lab can no longer do the same research, since everything is protected. Moreover, large laboratories often let startups do their thing and intervene later, when entering the clinical phase, but even more easily with phase II “it works”.
And sometimes by putting an upfront (several million) when the project is carried out - to have priority rights, once the results are known.
Will investors in this round have access to patents?
At this stage, investors will not have access to unpublished patents. It is of such a level of complexity that, unless you are a hyper-specialist, it would not provide additional information. Hyper-specialists can contact us (NDA etc).
It is not a good practice to distribute sensitive items too widely, it is only done under NDA conditions (confidentiality agreement) and when it is essential.
Wouldn't putting the drug into the public domain accelerate its dissemination?
Putting the drug into the public domain does not make it accessible to the patient: who would make the efforts of clinical studies, pharmacovigilance, production, packaging, distribution, if a competitor can copy it without participating in the investments?
CXS Therapeutics is the platform that continues to work for look for other hits in central system diseases.
Sappiens was created to support the clinical development of CXS 003 in Parkinson's.
It will be able to concentrate expertise, experience and funding around this pathology. This makes sense in the long run, when other hits mature, in other pathologies, there will be no dispersion of energy.
The team - and the capital dedicated to the clinical development of SAPPIENS - will remain focused on the clinical path in Parkinson's.
Hit's research activity and clinical development activity do not mobilize the same skills and are not in the same timeframe.
It therefore makes sense to subsidize Parkinson's activity, now that the CXS 003 program has provided the necessary guarantees to move to this stage.
All to accelerate clinical development - in 18 months phase II in patients.
Sappiens will be a subsidiary of CXS Therapeutics which will gain autonomy as investors and teams arrive, and then one day a pharmaceutical laboratory in charge of its distribution and marketing.
CXS will ensure that financial licences/patent sales agreements ensure commercialization so that Parkinson's patients can benefit from them, if the results of the clinical phases confirm the therapeutic interest.
Sappiens is intended to be acquired by a laboratory that will make the drug available to patients.
It is early to define the medical outlook, but there is still no approved neuroprotective drug for Parkinson's disease.
The hope is that this treatment can at least stop the evolution of the disease, and since its expected side effects are low, that it may be Administered early in the development of pathology.
But we are only at the stage of conjecture, clinical studies will determine which categories of patients are the ones for whom the benefit is real, and extensions can then be carried out, if justified from a medical point of view.
In an ideal world, CXS 003 is the first neuroprotector in Parkinson's disease, it makes it possible to stop the evolution of the disease, can be administered as soon as the diagnosis is made when the damage is still not very visible (although real).
In an even more ideal world, it allows restore partly, as in the laboratory, the functions of certain neurons.
In either case, it is a real therapeutic revolution.
(The CXS team has already made initial contacts and is for example going to Bio-Europe in Stockholm to make progress in these discussions now.)
Investor information is one of Valeureux's requirements.
It works very well in the laboratory on the models studied. Other studies are under way to prepare for phase II.
Know insert
The proof of concept in humans is carried out during phase II — at the end of this phase, we “know that it works” (or not) for the population of patients who participated in the study — with a higher or lower level of “it works” and we then begin a larger phase, phase III, to confirm on a larger sample and prepare for the first marketing.
So we can say that, if everything continues to go well, we will know if it works in 4 years.
The financial perspective is very important in case of the success of clinical studies, we are talking here about Multiple of times 50 and more. I'll let you calculate what would become of the €10,000 placed... €500,000 and more.
(But we're not there yet, so be careful.)
For example, we can refer to Mitokinin — which is only entering phase I — acquired by Abbvie last year.
“Under the terms of the agreement, AbbVie will pay Mitokinin shareholders $110 million at closing for the acquisition of Mitokinin. Mitokinin shareholders remain eligible for potential additional payments of up to $545 million upon the achievement of certain development and commercial milestones related to the success of the PINK1 program, plus tiered royalties based on net sales.”
But all of this is only valid if CXS 003 is medically relevant for patients. The alignment between progress and financial success couldn't be stronger
The financial benefits for CXS Therapeutics would be astronomical and would amount to billions of euros.
(The Valeureux fund would immediately become one of the most beautiful deeptech funds on the market! : D)
We can still invest in SAPPIENS — which will become a subsidiary of CXS in the coming months. To date, this new subsidiary is provided for by an investment in CSX and is specified in the partner agreement.
This also allows current investors to benefit from possible good news on another CXS Therapeutics hit (licensing, patent sale...) if the event happened before Sappiens reached maturity.
I hope you enjoyed this series. This episode was more technical than the previous ones, but necessary to understand the issues and perspectives. Can't wait to see you again soon for the rest of the adventure and other letters!
At Valeureux, knowledge and transparency are at the heart of our philosophy. If you invest with us, you need to do so at the right time and with a clear conscience.
This is probably the last opportunity to invest in this project (outside of specialized professional funds), so if you want to invest, It's over here.
Looking forward to seeing you again soon for the rest of the CXS Therapeutics adventure and other letters. Until then, get ready to discover other crazy projects.
Thanks for reading.
Philippe de Valeureux
Investing in unlisted companies presents risks of illiquidity and total loss of invested capital. Only invest what you are prepared to lose.
