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Flashpoint Therapeutics Announces Publication Demonstrating Superior Pre-Clinical Anti-Leukemia Efficacy with its Structural Nanomedicine Platform

via Business Wire

Study published in ACS Nano shows novel structural nanomedicine-based chemotherapeutic selectively targets and eliminates acute myeloid leukemia (AML) cells in preclinical models with significantly improved efficacy and safety

Flashpoint Therapeutics, a biotechnology company pioneering a new class of structural nanomedicine, announced today the publication of foundational research demonstrating the power of its proprietary Spherical Nucleic Acid (SNA) platform to create highly potent and targeted cancer therapies. The study, published in the journal ACS Nano by a team led by Flashpoint’s scientific co-founder Professor Chad A. Mirkin at the International Institute for Nanotechnology at Northwestern University, reports results of research with a new chemotherapeutic SNA that selectively targets and eliminates acute myeloid leukemia (AML) cells in preclinical models.

The publication highlights a revolutionary approach to nanomedicine design which incorporates the chemotherapeutic agent into the shell of the nanoparticle. Unlike conventional nanocarriers that encapsulate drugs within a core, Flashpoint’s SNAs are built with oligonucleotides made from units of a chemotherapeutic drug, 5-fluorouracil (5-Fu), anchored to a nanoparticle core. This unique architecture, a key tenet of structural nanomedicine, drives the therapeutic’s biological activity, enabling preferential uptake by myeloid cells, the lineage from which AML originates.

The study reports exceptional efficacy and a promising safety profile. Key findings include:

  • Targeted Delivery: The SNAs were selectively taken up by myeloid cells, including AML cells, at rates up to 12.5 times higher than the free drug components.
  • Potent Efficacy: The SNA construct demonstrated up to a 10,000-fold enhancement in cancer cell killing in vitro compared to the free drug. In a human AML mouse model, the therapy exhibited 59-fold greater antitumor efficacy than 5-Fu.
  • Favorable Safety: The potent anti-leukemia activity was achieved without observable side effects in animal models, suggesting a wide therapeutic window and the potential to reduce the harsh toxicities associated with conventional chemotherapy.

“This groundbreaking research by Professor Mirkin’s laboratory validates the capability of Flashpoint Therapeutics’ technology platform to precisely control the structure of a medicine at the nanoscale, thereby unlocking unprecedented therapeutic properties,” said Barry Labinger, Chief Executive Officer of Flashpoint Therapeutics. “The results in AML are a powerful demonstration of our platform’s ability to create targeted, highly potent drug candidates that overcome the limitations of conventional approaches. We are excited to advance this and other programs based on our structural nanomedicine platform to bring transformative new treatments to patients.”

AML is a devastating blood cancer with low survival rates, particularly for older patients who cannot tolerate aggressive chemotherapy. Flashpoint’s approach offers the potential for a new precision medicine that can effectively eliminate cancer cells while minimizing collateral damage to the body.

“This is a new class of chemotherapeutic that is defined by its structure,” said Professor Mirkin. “Today’s chemotherapeutics kill cancer cells but also a lot of healthy cells. Our structural nanomedicine preferentially seeks out the myeloid cells, where the AML resides. Instead of overwhelming the whole body with chemotherapy, it delivers a higher, more focused dose where it is needed.”

The full article, titled “Chemotherapeutic Spherical Nucleic Acids,” can be found in ACS Nano.

About Flashpoint Therapeutics

Flashpoint Therapeutics is a clinical-stage biotechnology company developing a new class of precision-engineered structural nanomedicines. Our platform enables the creation of targeted therapies that co-deliver optimized combinations of therapeutic components directly to individual cells, enhancing drug delivery, stability, potency, and safety. This modular approach supports a broad range of therapies, encompassing modalities like mRNA, DNA, proteins, and CRISPR. The company’s proprietary discovery platform is founded on nanotechnology research developed over the past decade at the laboratory of Dr. Chad Mirkin, Director of the International Institute for Nanotechnology at Northwestern University. Through strategic licensing and acquisition transactions with Northwestern University, Holden Pharmaceuticals, and Exicure, Flashpoint has assembled more than 150 issued patents and patent applications covering nucleic acid, protein, and CRISPR gene editing therapeutics. The company is funded by key investors, including Beta Lab and CS Venture Opportunities Fund. The company has also established an important partnership with King Abdullah International Medical Research Center (KAIMRC), which will advance the technology platform via the Flashpoint-KAIMRC Center of Excellence in Structural Nanomedicine and clinical trials in Saudi research institutions.

For more information about Flashpoint Therapeutics, please visit www.flashpoint.bio.

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