Closing out our Precision Medicine in Neurosciences discussion, Nik and Jason discuss how various partnerships can impact the success of Neuroscience drugs through precision medicine.

Moderator:

  • Jason Kralic, Operating Executive, Kineticos

Panlist:

  • Nik Tezapsidis, President and CEO, Neurotez

Kineticos: How does precision medicine play a role in supporting investment and partnering? There are companies that have tools that companies like yours and others may need to access in order to bring their products to market. Or are these tools being developed in large pharma that give them an advantage over startups that have a compound in development that could benefit from a precision medicine tool.

NT: Certainly, the more resources you have as a group of highly qualified and motivated individuals, as Neurotez does, the more advantageous it would be for the programs that you are developing for any indication whatsoever. Even more so for Alzheimer’s, an indication that involves long and expensive trials, that has been hampered by a history of failures. The more that can be shared that will facilitate the highly innovative, unconventional, and novel approaches, usually developed by smaller biotech companies, the better. The easier it would be for us, as a whole, to test those novel compounds. The small companies traditionally would bring an opportunity up to a certain point beyond which it becomes financially challenging to deliver later stage (phase 3) clinical trials. By that point, they are good candidates for partnerships with larger corporations, with the resources necessary and needed for late stage clinical trials. By that point, the small biotechs have created enough value to justify whatever sacrifices they have made throughout the time of taking the drug to that point. There is going to be more than enough reward for any innovators and founders of companies, to simply bring a compound to delivery of phase 2 clinical trial. By that point, usually big pharma has means to evaluate data and the compound has been reasonably de-risked, there is some signal that the compound has studied efficiently enough for big pharma to make a more concise decision on whether they can carry on to the next phase.

That’s roughly where we are headed too. If we can secure enough financing to do up until and deliver a phase 2, that would be optimal for us. That said, if we do get the financial backing, we will not back down on the possibility of executing a phase 3. By that point, you can always man the company with efficient executives and experienced personnel in the field that you can deliver phase 3. Particularly if all goes well, based on the approval of surrogate biomarker endpoints, we can be in the market in 4 years. That would include manufacturing the protein, doing the IND enabling studies, phase 1 and 2, and a short phase 3 could get us to the market in 4 years. Of course, there is a lot of things that have to be done properly for that to become a reality.

The small biotechs, can coordinate the studies and monitor progress. CROs become a major part of strategizing and executing the clinical trials. Small companies through those contractual arrangements can deliver compounds all the way to phase 3 clinical trials and beyond. It is all a matter of how able they are to convince investors to back this. In most cases, it’s the combination of investors and strategic partners that become the necessary partners/collaborators of the small biotechs’ drug development programs.

Whether there is anyone else ready to deliver a disease modifying drug in the near future, I don’t know the answer to that. Everyone was expecting, particularly those close to the beta-amyloid hypothesis, that one of these antibodies directed against those plaques would be a success. That wasn’t the case.

I am particularly curious as to how antibodies can be used as a therapeutic in Alzheimer’s disease targeting Abeta in the brain. I am puzzled at how that can be and there has been billions of dollars spent on this and there is still a big question mark there. Antibodies are huge molecules, only .01% of what is injected actually makes it into the brain. You need 1 antibody per ABeta molecule. It is a very inefficient process.

Kineticos: For the beta-amyloid story, the antibodies were shown to be effective at what they targeted them to do. But it appears that clearing beta-amyloid wasn’t sufficient to improve the course of disease.

NT: I don’t know how much antibody they had to inject and how cheap that therapy would have been. It’s not a practical or cost-effective means to treat 50 million people around the globe requiring frequent outpatient visits for hours of infusion. Even if it was doing its mission, which was removing Abeta. However, as you pointed out, they were able to do that, but it was ineffective in improving cognition. However, at least these very expensive trials provided a clear answer to the question: Is Abeta the culprit of the disease?  In our case, even though our drug is a protein (usually more expensive and complicated to manufacture than chemically synthesized small molecules) it does not require post-translational modifications for activity and as such can be produced by prokaryotes considerably cheaper than proteins needing mammalian cell systems.


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