Heikki Mansikka, MD, PhD, Anesthesiologist, working in the phama industry developing new drugs for pain, respiratory and various inflammatory diseases now for over twelve years, talks about a potential new drug to treat patients with peripheral neuropathy pain.
Interview conducted by Ivanhoe Broadcast News in August 2017.
Can you tell me what your position with Chromocell Corporation is?
Dr. Mansikka: I’m a Vice President leading clinical development at Chromocell Corporation.
What can you tell us about the new drug in development? What is it called and what does it do?
Dr. Mansikka: Our drug candidate is called CC8464 and it is still in the very early stages of the clinical development process. We are in Phase I where we investigate this new drug candidate for the first time in humans, including healthy volunteers to understand the initial safety and tolerability profile of the compound. That’s the first stage of the development. If successful, we will move to the next stage of development, Phase 2. In that phase, we take this drug candidate to a small number of patients that we want to investigate and that will expand our knowledge and understanding of this compound. In Phase 2 we will learn more about the safety profile of the compound and about whether it has efficacy in the patient population that we are investigating in the trial. If CC8464 is successful in Phase 2, we will need to agree with regulatory authorities on required Phase 3 program. This could involve several hundreds up to thousands of patients. Based on those, what we call confirmative trials, we will confirm the safety and efficacy of this drug. This will form the basis for the marketing authorization and applications that we would submit to the US and other regulatory authorities who would then approve the drug to be a marketed product.
The Phase I trial – how long is that intended to run, how many people are enrolled in it, are you still enrolling?
Dr. Mansikka: Yes, the Phase I is currently ongoing. Typically, the Phase I trial include tens up to the low hundreds of people. Depending on what we find, we may need to modify the trial based on the data we generate. So, it may be too early to tell.
Tell me a little bit about this drug candidate, what is it made of?
Dr. Mansikka: There are a few exciting aspects of this candidate.
The most exciting is that it’s a non-opioid by how it works in the body. As your viewers know there is a big public health crisis in the US, especially with addiction and abuse associated with opioid drugs; that has a huge impact on the patients and their families as well as on the whole healthcare system. Another very exciting characteristic of our drug is how it works; it targets specifically the pain fibers in the periphery and it’s not expected to go to the brain or to the central nervous system where we know that opioids become addictive. With its unique profile we do not expect CC8464 the addiction problem or unpleasant central nervous system mediated side effects, unlike opioids and other drugs that are currently in the market to treat the pain.
You mentioned the way this drug works within the central nervous system and that’s one of the reasons why you don’t believe you will have these addictive properties. Can you tell me a little bit more about that?
Dr. Mansikka: If we think about opioids, why do they cause addiction? It’s because there are reward centers in our brain and when they’re activated this causes us to get this feeling of euphoria. This can occur a number of ways but opioids cause this euphoria by having this pharmacological effect on these reward centers directly on your brain. As our drug does not penetrate this area, it shouldn’t have the addictive properties of opioids. In addition, we don’t expect to have any central nervous system mediated side effects which are also a big problem for the patients with current therapies.
Tell me a little bit about some of the compound that is in this drug, Japanese blow fish toxin, is that correct?
Dr. Mansikka: The Puffer Fish is an established delicacy in Japan, but it is associated with some toxic effects if not prepared correctly. The toxic effects of the Puffer Fish are mediated by a toxin called tetrodotoxin or TTX. TTX targets proteins on the cell surface of the muscles, nerves and heart and that’s how it causes its toxic effects. It can immobilize your muscles or your heart, which may then lead to respiratory or heart failure. On the contrary, our drug only inhibits very selectively one subtype of these proteins and has a very specific effect on pain fibers and it therefore is not associated with the toxicity of tetrodotoxin.
Tell me about the best candidates for this trial. Which patients would be best to use this?
Dr. Mansikka: In our Phase II trials, we will target patients who have nerve-related pain. There is evidence that this particular protein that our drug candidate targets may be a key component mediating these types of nerve related pain. This nerve-related pain can come from different diseases, diabetes, for example, is a common disease that can damage nerves which then start to function abnormally, causing pain without good reason. It is this type of pathological pain that we believe we can best target with our first drug candidate.
I know that this is just entering Phase I, but do you have a rough timeframe should everything continue on track?
Dr. Mansikka: It will most likely be several years before the compound can make it to the market. Unfortunately, I cannot be very specific about that because in clinical development there are always surprises. Once we start to generate data, we will have negotiations with the Food and Drug Administration. What the data looks like, do they agree with our plans? Then we start the trials in the patients and depending on how the data turns out, that decide the fate of the compound for treating pain.
Is there anything that I didn’t ask you that you want to make sure is highlighted?
Dr. Mansikka: Given the current opioid-induced public health crisis the development of CC8464 is really very exciting. We believe it has the potential to be a major advancement in the field of pain management. Because of its unique mechanism of action, how it works in the body by targeting peripheral nerve fibers and not penetrating the brain and the central nervous system, CC8464 avoids addictive potential and other central nervous mediated side effects. At Chromocell our focus is on pain management and we hope that this candidate will be a product to help patients in pain management without the devastating side effects that are associated with current treatment options.
END OF INTERVIEW
This information is intended for additional research purposes only. It is not to be used as a prescription or advice from Ivanhoe Broadcast News, Inc. or any medical professional interviewed. Ivanhoe Broadcast News, Inc. assumes no responsibility for the depth or accuracy of physician statements. Procedures or medicines apply to different people and medical factors; always consult your physician on medical matters.
If you would like more information, please contact:
Heikki Mansikka, MD, PhD
Chromocell Corporation
Heikki.Mansikka@Chromocell.com
Elizabeth Dowling
elizabeth.dowling@mountsinai.org
Foundation for Peripheral Neuropathy
847-883-9942
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