ISA Pharmaceuticals and Cancer Focus Fund Announce $5 Million Investment to Support First-in-Human Trial of ISA’s Novel Immunotherapy ISA103 in Uveal Melanoma

Clinical Trial of PRAME-Targeting Immunotherapy ISA103 in Rare Ocular Cancer to be Conducted at MD Anderson Cancer Center

ISA Pharmaceuticals, B.V., a clinical-stage biotech company developing immunotherapies to treat cancers and infectious diseases, and Cancer Focus Fund, LP, a unique investment fund established in collaboration with The University of Texas MD Anderson Cancer Center to provide funding and clinical expertise to advance promising cancer therapies, today announced that Cancer Focus Fund is investing $5 million in funding to support ISA103, ISA’s PRAME-targeting immunotherapy, in a first-in-human study for the treatment of uveal melanoma, a rare ocular cancer.

ISA103 immunotherapy targets PRAME (PReferentially expressed Antigen in MElanoma), a cancer testis antigen that is overexpressed in various solid and liquid cancers. It plays a crucial role in tumor survival and spread. ISA103 was developed using the company’s proprietary Synthetic Long Peptide (SLP®) technology, designed to fully harness and direct the body’s defense mechanisms towards fighting the disease. It contains multiple long peptides spanning the most immunogenic regions of the PRAME protein.

“At ISA we believe that our rationally designed immunotherapies have the potential to produce improved outcomes for cancer patients,” said Gerben Moolhuizen, Chief Executive Officer of ISA Pharmaceuticals. “We have already achieved proof-of-concept successes with our lead product ISA101b in HPV-induced cancers, and we view PRAME-positive tumors as another promising target for our unique approach. We believe this investment from the Cancer Focus Fund and the clinical expertise provided by MD Anderson will significantly advance ISA103 as an immunotherapy with the potential to combat this rare but debilitating malignancy.”


“Cancer Focus Fund is committed to using its investments to support the clinical development of truly innovative cancer therapies,” said Ross Barrett, a founder and Managing Partner of Cancer Focus Fund. “ISA’s multi-pronged approach to the design and development of fully synthetic novel immunotherapeutics has great potential and importantly, has already demonstrated early success in its initial programs. Our distinctive investment model is continuing to resonate globally, with another of our portfolio programs now based outside the U.S., and we are pleased to be collaborating in this effort with ISA and MD Anderson.”


PRAME expression is common in uveal melanoma and is associated with poor disease outcome. In the two-part open label Phase 1b/2 trial, ISA103 will be tested in combination with standard of care checkpoint blocker immunotherapies. An initial dose-finding phase will be followed by an expansion cohort using the optimal dose. Study endpoints will include safety, immunogenicity and signs of efficacy (response rates and survival indicators). A total of 90 patients will be enrolled in the trial. The trial will be conducted at MD Anderson under the direction of Principal Investigator Sapna Patel, M.D., Associate Professor of Melanoma Medical Oncology and Director of MD Anderson’s Uveal Melanoma Program.

About ISA103 and PRAME: PRAME (PReferentially expressed Antigen in MElanoma) is a protein that is expressed on various types of tumors, including lung, breast, head & neck and kidney cancers, as well as melanoma and neuroblastoma, while there is minimal expression in heathy tissue. There appears to be a functional association of PRAME expression and tumorigenesis. Overexpression of PRAME is associated with poor disease prognosis. ISA103 is designed to create a strong and specific T cell immune response against PRAME. It consists of multiple Synthetic Long Peptides (SLP®) spanning the most immunogenic regions of the PRAME protein. ISA103 is under investigation as a potential treatment for PRAME expressing cancers. 

About ISA’s SLP® Technology: ISA’s SLP® immunotherapeutics are easy-to-manufacture synthetic peptides. They cover the most immunogenic regions of the therapeutic target and contain epitopes for the efficient induction of both CD4 and CD8 T cell responses by the patient, regardless of the individual’s HLA type. SLP®-based immunotherapeutics overcome central tolerance, cover a broad range of HLA restricted epitopes to induce strong and lasting specific T cell responses, and are complementary to standard of care therapy, including, in the case of cancer, chemotherapy and immunomodulators like anti-PD1 antibodies.

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