• 100 percent of mice treated with NTX-250 saw complete regression of tumors, long-term survival, and robust immune cell infiltration
• NTX-250 demonstrated translational potential in non-human primates by inducing HPV-specific immune responses

Nutcracker Therapeutics, Inc., a biotechnology company dedicated to developing transformative RNA therapies through its proprietary technology platform, today presented the first set of preclinical data for its lead oncology mRNA therapeutic, NTX-250, at the 37th annual Society for Immunotherapy of Cancer meeting. The data demonstrate that NTX-250 has the ability to elicit strong anti-tumor responses in C3.43 murine tumor models and induces an immune response in non-human primates specific to human papillomavirus (HPV).

NTX-250 is being developed for tumors driven by HPV16, which includes cervical cancer and its precursor, cervical intraepithelial neoplasia (CIN). Key highlights of the data include:

• NTX-250 induced complete regression of large tumors – greater than 120 mm³ in volume – resulting in long-term, tumor-free survival of all 10 treated animals
  • This result was accompanied by markers of strong tumor infiltration of CD8+ T cells, CD4+ antigen-presenting cells and natural killer (NK) cells
  • Successful modulation of the tumor microenvironment (TME) was also observed through the upregulation of interferon-gamma (IFN-γ) and other key immunomodulators
• The translational potential of NTX-250 was assessed in cynomolgus monkeys (n=2), where it was seen that administration of 3 doses of NTX-250 induced strong HPV16-specific responses

“We’re excited to share Nutcracker Therapeutics’ first set of data on NTX-250 with the scientific community,” commented Chief Medical Officer Robert J. Schott, M.D. “It’s been our long-held belief that the RNA drug class presents a unique opportunity to create safe and effective immunotherapies. The preclinical data we presented today is compelling in this regard, and shows that NTX-250 is able to induce a strong immune response and completely regress tumors in a murine model.”

NTX-250 is composed of three unique and synergistic mRNA molecules in a mRNA therapeutic. This triple mechanism is designed to induce a robust antitumor T-cell response in addition to opening the tumor microenvironment to enhance immune cell activity and reduce immunosuppression. All three components have been shown to enhance long-term efficacy against HPV-driven tumors in model systems.

More than 90 percent of cervical cancers and CIN lesions, and 70 percent of head and neck cancers, are linked to infections with high-risk HPV. HPV16 is the most prevalent high-risk strain, accounting for nearly half of infections. The company believes that there is an increasing need to not just target cervical cancer itself, but also CIN, as a significant percentage of women with high-grade CIN lesions progress to invasive cancer, if left untreated.

“Developing a therapy that includes all the necessary cancer-killing components efficiently is uniquely suited to mRNA,” Dr. Schott elaborated. “Nutcracker Therapeutics’ proprietary RNA development platform was created to bring forward therapies, such as NTX-250, quickly and efficiently.

“Designing the three individual mRNAs, manufacturing the mRNA molecules, and encapsulating them within our Nutshell^® delivery vehicles was done using our end-to-end workflow within our platform,” Dr. Schott continued. “We look forward to using our in-house technology and expertise to develop much-needed therapies for other types of cancer.”

A Phase I clinical trial for NTX-250 is planned for 2023, pending the clearance of an Investigational New Drug application by the Food and Drug Administration.

About Nutcracker Therapeutics, Inc.

Nutcracker Therapeutics, Inc. is an RNA therapeutics company that has combined the power of advanced engineering with high-precision biosynthesis to deploy a complete RNA platform that encompasses the design, delivery, and manufacturing of RNA medicines. Armed with this high-tech advantage, the company has initiated multiple therapeutic programs with the support of clinical investigators at leading institutions. Nutcracker Therapeutics’ technology platform has the potential to significantly reduce costs and cycle times for RNA therapeutic development, with dramatic advantages in speed and capacity scaling over other RNA manufacturing approaches.

For more information, visit www.nutcrackerx.com.

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