DTx Pharma: Revolutionizing RNA Medicine Delivery - From Series A Investment to Novartis Acquisition

Investing in the Future of RNA Medicines

In May 2019, Tachyon Ventures embarked on an exciting journey by investing in DTx Pharma, a preclinical RNA startup based in San Diego. At the time, the company was less than 2 years old and had only raised $2M in a Seed Round, but already presented an ambitious vision and potentially groundbreaking approach to address a longstanding challenge in the field of RNA therapeutics: delivering small interfering RNA (siRNA) beyond the confines of the liver.

DTx Pharma's incipient platform held two provisional patents to be filed that month, which demonstrated the activity of DTx motifs with multiple siRNAs across in vitro, ex vivo & in vivo assays. This, together with a seasoned management team and a strategically timed entrance into the rapidly expanding oligo drugs market, caught our attention.  Their track record of moving fast with limited capital further solidified Tachyon's belief in the company's potential, ultimately leading us to invest in DTx Pharma's Series A funding round. 

Today, we are delighted to report that DTx Pharma has been acquired by Novartis for $500 million in up front payments, and another $500 million upon completion of milestones.

The Challenge: Delivering siRNA Beyond the Liver

RNA interference (RNAi) is a revolutionary mechanism that allows scientists to silence disease-causing genes using small interfering RNA (siRNA) molecules, presenting a vast potential for treating a wide range of debilitating illnesses. However, the practical application of siRNA-based therapeutics was hindered until recently by a significant limitation: the ability to effectively deliver these therapeutic molecules to target cells and tissues throughout the body. Traditionally, siRNA was primarily taken up by the liver and kidney, rendering it challenging to reach other crucial organs and tissues. 

Unlocking the Potential of RNA Medicines with a Game-Changing Delivery Platform

Recognizing this limitation, DTx Pharma set out to develop a delivery platform that could efficiently deliver siRNA to most cells in the body, with no overt toxicity.

DTx Pharma's approach involves using fatty acids as targeting ligands to transport siRNA molecules to their target cells and drive cellular update. These oligo-based carriers showed tremendous promise in their preliminary results, indicating a potential breakthrough in RNA medicine delivery. By enabling RNA medicines to reach beyond the liver and into various organs and tissues, DTx Pharma's platform holds immense potential to unlock a whole new class of medicines.

DTx Pharma's Lead Drug Candidate: Pioneering Hope for Charcot-Marie-Tooth (CMT) Disease

One of the key factors that has propelled DTx Pharma and attracted the attention of Novartis is its lead drug candidate, poised for Phase 1 testing in Charcot-Marie-Tooth (CMT) disease. CMT is a rare and debilitating neuromuscular disorder affecting thousands of individuals worldwide, with no FDA-approved therapies currently available. CMT disease manifests as a hereditary and degenerative condition, causing muscle weakness and loss of sensation in the extremities. Patients often experience difficulty walking, frequent tripping, muscle wasting, and nerve-related pain. With no cure in sight, the disease significantly impacts the quality of life for those affected and leaves them with limited treatment options. 

DTx’s lead program aims to target Schwann cells, which are found in the peripheral nervous system, and silence the specific disease-causing gene responsible for CMT1A, PMP22, in order to slow down or halt disease progression. In addition to preclinical data demonstrating that DTx’s CMT1A therapy was able to engage Schwann cells, key data coming from studies of DTx’s lead candidate in non-human primates further showed a promising therapeutic index – a treatment dose range balancing safety and efficacy. 

As DTx Pharma's lead drug candidate moves closer to Phase 1 clinical trials for CMT1A, the acquisition by Novartis brings with it the resources and expertise necessary to advance the program swiftly through clinical trials and regulatory processes. This acquisition has the potential to expedite the development and approval of a groundbreaking therapy for CMT, offering hope and possibility to a patient community lacking an effective treatment for their condition.

A Blossoming Market and Strategic Collaborations

With the oligo drugs market rapidly expanding and already boasting a couple of approved drugs grossing over $1 billion in annual sales at the time we invested, DTx Pharma's breakthrough technology was developed at an opportune time. Furthermore, the company has strategically established academic collaborations with prestigious institutions such as UC San Diego, the Neural Stem Cell Institute, University of Pennsylvania, and University of Wisconsin, enhancing DTx’s research capabilities and providing invaluable academic support.

A Bright Future Ahead

DTx Pharma has come a long way since Tachyon Ventures' initial investment. DTx effectively leveraged their resources to make significant strides in their preclinical research, and the results of their lead program for non-human primates proved promising enough to secure an acquisition by Novartis. Today, DTx has achieved the moonshot potential that Tachyon’s team had hoped for.

The addition of DTx Pharma's innovative RNA-based platform to Novartis' arsenal of drug development capabilities also positions the company at the forefront of precision medicine and RNA therapeutics. Novartis can further explore applications of DTx Pharma's platform in neurological disorders. Conditions like Alzheimer's, Parkinson's, and Huntington's diseases, which are characterized by abnormal gene expression, could benefit from the precision and potential disease-modifying effects of RNA-based therapeutics.

The recent acquisition by Novartis  marks a momentous event in DTx Pharma's journey, affirming the value of their pioneering approach to RNA medicines delivery. This is particularly meaningful at a time where 165 publicly traded US biotech companies are trading below cash, compared to only 2 in February 2021.

As we celebrate this achievement, the team at Tachyon looks forward to witnessing the DTx platform technology’s continued growth and contributions to biotechnology. As the company continues its development through the completion of milestones in clinical development (Phase 1-3), FDA approval, and commercialization, it will further unlock  substantial value for Novartis, the company’s original investors and, most importantly, thousands of patients around the world.

The future of biotech looks exceptionally bright, and Tachyon Ventures remains committed to supporting innovative startups that invite paradigm shifts in science and medicine, aiming to shape a healthier and more promising future for all.

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