Immorna Biotherapeutics Gets FDA Nod for Phase 1/2 Study of JCXH-211 IV in Advanced Solid Tumors

Immorna Biotherapeutics, Inc., a biotechnology firm based in Morrisville, NC, has received approval from the U.S. Food and Drug Administration (FDA) for its investigational new drug (IND) application for JCXH-211 intravenous (IV). This drug is a novel, first-in-class self-replicating mRNA encoding the engineered human interleukin (IL)-12 protein. The approval allows Immorna to commence a Phase 1/2 clinical trial aimed at evaluating the safety and effectiveness of JCXH-211 IV in patients with malignant solid tumors.

The upcoming trial will be a multi-center, open-label study that includes dose escalation and expansion phases. The objectives are to assess the safety and tolerability of the drug, determine the recommended Phase 2 dose (RP2D) when used in combination with a checkpoint inhibitor (CPI), and evaluate preliminary efficacy at the RP2D.

NgocDiep Le, M.D., Ph.D., President and Global Chief Medical Officer of Immorna, expressed enthusiasm over this milestone. Le highlighted that preclinical studies, along with clinical data from a previous JCXH-211 intratumoral administration (IT) trial, suggest that the intravenous form of JCXH-211, when combined with CPI, could enhance anti-tumor effects. The company aims to collaborate with investigators and patients to bring this innovative therapy to those in urgent need of new treatment options.

JCXH-211 stands out as a first-in-class lipid nanoparticle (LNP) encapsulated self-replicating RNA (srRNA) developed using Immorna’s proprietary technology. The drug encodes the engineered human IL-12 protein, and its combination of an anti-viral innate response triggered by RNA replicon and the anti-cancer immunity stimulated by IL-12 has shown superior tumor-eradicating potency in multiple preclinical animal and patient-derived xenograft (PDX) models. Interim data from a Phase 1 trial of JCXH-211 IT monotherapy revealed excellent safety, tolerability, and significant anti-tumor biological activities, including an abscopal effect.

IL-12 is a cytokine that naturally plays a pivotal role in the immune response against cancer. Despite its potent anti-tumor activity in preclinical studies, recombinant IL-12 protein treatments at tolerable doses have not demonstrated clinical benefits in humans. The primary challenge has been the non-overlap of tolerability and therapeutic windows. Recombinant IL-12 is unstable in vivo with a short half-life, making frequent intravenous administration challenging due to toxicities. However, JCXH-211 IV, thanks to its srRNA technology, enables prolonged IL-12 expression specifically in tumor tissues. This leads to modulation of the tumor microenvironment and activation of antitumor immune responses, while minimizing systemic toxicity. Nonclinical studies in rodents and non-human primates have shown an excellent safety profile for JCXH-211 IV. If successful, this drug could become a lifesaving and accessible therapy for cancer patients who have exhausted current treatment options.

Immorna Biotherapeutics, founded in 2019, is a rapidly growing biotechnology company focusing on RNA-based therapeutics and vaccines. Utilizing multiple RNA platforms—conventional, self-replicating, and circular RNAs—the company has built a strong CMC platform for RNA synthesis, purification, and analytical testing. Immorna also boasts advanced screening tools and a range of RNA delivery vehicles, including polymers and lipid nanoparticles designed for various delivery methods. Its diverse development pipeline covers cancer immunotherapy, infectious diseases, rare genetic diseases, and medical cosmetology, with several candidates advancing into clinical stages.