On July 7, 2023 Ichnos Sciences Inc., a global clinical-stage biotechnology company developing innovative multispecific immune cell engager antibodies in oncology, reported that it has been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for its first-in-class T-cell engaging trispecific antibody, ISB 2001, for the treatment of multiple myeloma (Press release, Ichnos Sciences, JUL 7, 2023, View Source [SID1234633109]).
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ISB 2001, Ichnos’ third clinical-stage asset to receive ODD for the treatment of multiple myeloma, is the company’s first BCMA x CD38 x CD3 TREATTM1 trispecific antibody based on the company’s proprietary BEAT platform2, which enables the development of immune cell engagers. This designation closely follows that of Ichnos’ first-in-class CD38 x CD47 biparatopic bispecific antibody innate cell modulator, ISB 1442, which was granted ODD by the FDA in March 2023.
"The FDA’s decision to grant orphan drug designation to ISB 2001 – our third clinical-stage asset to receive that designation in five years – underscores the importance of the work our team is doing to develop potentially curative therapies for the treatment of multiple myeloma," said Cyril Konto, M.D., President and Chief Executive Officer of Ichnos Sciences. "As new multiple myeloma diagnoses continue to climb, our charge of providing patients more innovative, novel therapies that can overcome the limitations of options available to them today becomes that much more urgent."
This milestone is achieved as Ichnos prepares to initiate a Phase 1 first-in-human dose-escalation dose-expansion study of ISB 2001 later this summer, after securing approval from the Human Research Ethics Committee (HREC) in Australia and IND clearance from the FDA.
"The entire team at Ichnos – across every role and function – comes to work focused on realizing our vision of a world free of multiple myeloma. This decision by the FDA reinforces that we are on the right path," said Lida Pacaud, M.D., Chief Medical Officer of Ichnos Sciences. "Securing orphan drug designation for ISB 2001 is a critical milestone as we advance the clinical development of this promising asset, and it comes at a pivotal moment as we prepare to initiate a Phase 1 clinical trial."
ISB 2001 combines three proprietary Fab arms binding to CD3 on T cells, as well asBCMA and CD38 on multiple myeloma cells. Through targeting two tumor-associated antigens, ISB 2001 has increased binding specificity to multiple myeloma cells due to enhanced avidity-based binding, and it has demonstrated increased killing of tumor cells in vitro across variable levels of expression of both BCMA and CD38 compared to teclistamab, alnuctamab and EM-801. Additionally, ISB 2001 exhibits higher potency in vitro when compared to the combination of daratumumab and teclistamab currently under clinical investigation, as demonstrated by data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2023.
The FDA grants ODD to therapies that show promise in the treatment, prevention, or diagnosis of rare disease or conditions that affect fewer than 200,000 people in the United States, such as multiple myeloma, which is a rare cancer of plasma cells. According to the National Cancer Institute, in 2019, there were an estimated 159,787 people living with myeloma in the United States with approximately 34,470 new cases estimated in 20223. While there has been headway in the treatment of multiple myeloma, there remains a significant unmet need for therapies that can overcome developed resistance and decreased effectiveness over time.