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NK Cell Therapy CYNK-001 for the Treatment of Acute Myeloid Leukemia

The FDA has granted Fast Track Designation for its non-genetically modified cryopreserved human placental hematopoietic stem cell-derived natural killer (NK) cell therapy in development for the treatment of acute myeloid leukemia (AML). The therapeutic program based on placental-derived unmodified NK cell type is CYNK-001, an allogeneic unmodified NK cell being developed as a treatment for hematologic malignancies, solid tumors, and infectious diseases.

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CYNK-001 is a cryopreserved allogeneic off-the-shelf cell therapy enriched for CD56+/CD3- NK cells expanded from human placental CD34+ cells. The safety and efficacy of CYNK-001 have not been established, and CYNK-001 has not been approved for any use by the FDA or any other analogous regulatory authority.


Rare Pediatric Disease Designation for IMX-110 for Life-Threatening Cancer

The FDA has granted IMX-110 Rare Pediatric Disease designation for the treatment of rhabdomyosarcoma (RMS), a life-threatening form of pediatric cancer. IMX-110 is an investigational product currently being evaluated in a Phase Ib/IIa clinical trial. This FDA designation covers serious and life-threatening diseases that primarily affect children aged 18 years or younger and impact fewer than 200,000 people in the United States.


RMS is a high-grade, malignant neoplasm, the most common soft tissue sarcoma in pediatric and adolescent populations that rarely occurs in adults. The prevalence of RMS in the United States is approximately 20,000 children of all ages. The 5-year survival rate ranges from 20 percent to 30 percent for children in the high-risk group where cancer spreads widely in the body.


IMX-110 is the first clinical-stage product of the SMARxT Tissue-Specific Platform, which produces Tissue-Specific Therapeutics that accumulate at intended therapeutic sites at 3-5 times the rate of conventional medicines. The FDA has already granted Orphan Drug Designation to IMX-110 for the treatment of soft tissue sarcoma.


Researchers recently shared clinical data across multiple soft tissue sarcoma subtypes in several heavily pretreated patients demonstrating median progression-free survival of 4 months with zero drug-related severe adverse events and zero dose interruptions due to toxicity.


CLN-081 for EGFR-Mutated Non-Small Cell Lung Cancer

The FDA granted Breakthrough Therapy Designation for CLN-081 to treat patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring epidermal growth factor (EGFR) exon 20 insertion mutations who have previously received platinum-based systemic chemotherapy.


CLN-081 is an orally available, irreversible EGFR inhibitor that selectively targets cells expressing EGFR exon 20 insertion mutations while sparing cells expressing wild-type EGFR. Researchers are evaluating various doses of CLN-081 in a Phase I/IIa trial in patients with NSCLC harboring exon 20 mutations whose disease has progressed on or after prior therapy.


Breakthrough Therapy Designation to Telisotuzumab Vedotin for NSCLC

Telisotuzumab vedotin has been granted FDA Breakthrough Therapy Designation for the treatment of patients with advanced/metastatic epidermal growth factor receptor (EGFR) wild-type (WT), nonsquamous non-small cell lung cancer (NSCLC) with high levels of c-Met overexpression whose disease has progressed on or after platinum-based therapy.


This designation is supported by data from LUMINOSITY (Study M14-239), an ongoing Phase II study designed to identify the target NSCLC populations that overexpress c-Met best suited for telisotuzumab vedotin monotherapy in the second-line or third-line setting, and then to expand the groups to further evaluate efficacy in the selected populations. The primary endpoint is overall response rate (ORR) per central review in patients with >=12 weeks follow-up. Among patients with EGFR WT nonsquamous NSCLC, ORR was 53.8 percent in the c-Met high group and 25.0 percent in the c-Met intermediate group at a previously reported interim analysis.


Telisotuzumab vedotin is also being evaluated in combination with osimertinib in the ongoing Phase I study M14-237 in patients with previously treated c-Met overexpressing NSCLC. In addition, it will be further evaluated as monotherapy in patients with previously treated c-Met overexpressing NSCLC in the randomized Phase III study TeliMET NSCLC-01 (Study M18-868).


Telisotuzumab vedotin is an investigational antibody-drug conjugate targeting c-Met, a receptor tyrosine kinase that is overexpressed in tumors including NSCLC. Telisotuzumab vedotin is not approved by any regulatory authority and its safety and efficacy have not been established. Currently, there are no approved cancer therapies specifically for patients with c-Met overexpressing NSCLC.