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Taking RNA Therapeutics to New Heights: DTx Pharma’s Approach for Targeting Underutilized Cells and Organ Systems




If you were to ask Arthur T. Suckow, PhD, Co-founder and CEO of DTx Pharma, what is one piece of advice that has stuck with him throughout his career, you may be surprised to hear it is to “stay in school.”

With an extensive background spanning prominent pharmacology companies, Dr. Suckow has spent his career advancing small molecules targeting fatty acids and fatty-acid conjugated peptides.

When it became apparent that current limitations of drug delivery hindered the widespread use of RNA therapeutics, he was inspired: what if fatty acid receptors could address these challenges and provide potential solutions for diseases that had been traditionally underserved with treatment options such as retinitis pigmentosa or Charcot-Marie Tooth? Seizing the opportunity to be an innovator in this space, Dr. Suckow took his knowledge of fatty acids, his entrepreneurial passion, and the little bit of money he had saved in his 401k to launch DTx Pharma. Now, the company is projected to begin its clinical trials within the next two years.

In our interview with Dr. Suckow, we had the chance to catch up on what DTx Pharma has been up to since joining the alumni network of Johnson & Johnson Innovation– JLABS (JLABS) and how it aims to unlock the potential power of RNA therapeutics.

Where did the idea behind DTx Pharma come from?

When I was at the University of California, San Diego for my PhD, I first had the inclination to start my own company. I had a “minor proposition,” which is essentially an opportunity for you to create your own idea and pitch it to a committee as part of your qualifying exam.

After the pitch, a member of the committee approached me and said that they would fund the idea and provide the needed lab space. I thought it over and discussed the idea with my mentors. Everyone encouraged me to finish school first. While I was completing my courses, I was simultaneously reading entrepreneurial books and finishing my business plan to try and gain the exposure and experience needed to build a company.

While I worked at a few different pharma companies before starting DTx, including the Johnson & Johnson Family of Companies, it was the experience working closely with fatty acids and fatty-acid conjugated peptides that led me to the idea that they could be used to create new RNA therapies with the potential to address challenges of drug delivery.

Can you tell us about those current limitations of RNA therapeutics?

One of the most significant challenges with RNA therapeutics is delivery, which can be explained in two parts. First, the cells do not take these medicines up, so there is a need to improve cellular uptake. Second, the kidney rapidly absorbs these medicines, so there is less likelihood that they will be delivered to the needed tissues throughout the body like the heart, muscles, or the central or peripheral nervous systems.

The greatest potential for improving the delivery of RNA therapeutics lies within the minds of innovators and scientists. In the future, I anticipate we will see a cross-fertilization of experts who have worked across other therapeutic area modalities and who can utilize their knowledge to address these challenges cohesively. At DTx, we already have this advantage as we have all worked on other therapeutic modalities.

There are a number of learnings that have not yet been deployed across the industry which could be potentially meaningful for RNA therapeutics, and we are only scratching the surface in how you could use fatty acids to overcome these challenges.

How does DTx aim to leverage these learnings to help deliver better potential solutions for patients?

The reason that we like fatty acids is that they help address both limitations of drug delivery – essentially “killing two birds with one stone.” They have the ability to help your body take up the cells and distribute them to areas beyond the liver and the kidney. I compare it to a rideshare service: fatty acids pick you up, open the door to the cells, and deliver you to your ideal destination.

Many difficult-to-treat diseases are driven by genetic mutations, which lead to proteins that are dysfunctional or prone to aggregation. RNA therapeutics can help eliminate or reduce the expression of these aberrant protein products. Through our proprietary FALCON™ (Fatty Acid Ligand Conjugated OligoNucleotide) platform, our focus right now is to develop therapies for these genetic diseases, by leveraging its ability to promote better cellular uptake and biodistribution of RNA therapeutics. We’re currently prioritizing eye and neuromuscular diseases, though we also plan to leverage FALCON in the future to address other therapeutic areas including CNS and cardiovascular diseases.

With the support of your recent $100 million Series B Financing, how do you see the company growing?

Our immediate goal is to hire talented experts in RNA therapeutics to help us execute on our current programs and identify other areas to deploy this technology.

Our next step towards clinical development is to determine that our technology works in rodent models and then non-human primate studies, which we anticipate conducting across three therapeutic areas over the course of the next year. In parallel, we will likely move to safety studies for our ocular and neuromuscular programs which will help support the clinical dosing of these applications in patients. And in the next two years, it is very likely that we will have our first human clinical trial for our ocular program in retinitis pigmentosa.

Along the way, we hope to be able to announce a potential partnership with a leading pharmaceutical company, and to be in a position where both parties bring their expertise to add value.

How do you think being located in San Diego’s robust innovation ecosystem helped contribute to the company’s success?

There is something special about collaboration in this area, and I often wonder if I chose San Diego or if San Diego chose me. Every person here is always willing to share information and make introductions, which can help make all the difference. I would not have wanted to do this journey anywhere else, and our $100 million raise likely would have not happened anywhere else.

JLABS was critical to our success. We had everything we could have needed including benches, dedicated cell culture and chemistry spaces, and access to upwards of $1 million in equipment. On top of that, the ecosystem within is a great source for networking, creating opportunities to not only showcase our company but to practice pitching and landing investor meetings.

With the network I had established throughout my studies and career, along with the resources and opportunities that JLABS provided, it created the framework for launching a successful company.

DTx is a unique story because it all started with an idea and the little money I had saved in my 401k. To me, my life savings were not the ‘safety net’ behind this idea but rather the people around me who believed in me and saw something that I could not always see.

My hope is that there are other scientists out there with the idea and heart to take the leap toward advancing their science, and that JLABS continues to nurture their growth and support the DTx Pharmas of the world.

For more on DTx Pharma, please visit their website at https://dtxpharma.com/.


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