In June 2023, LLS made an equity investment in Rgenta Therapeutics to "Support development of RNA-targeting molecules for blood cancers."
Rgenta Therapeutics is developing a pipeline of oral, small-molecule RNA-targeting medicines with an initial focus on oncology and neurological disorders. Rgenta has a proprietary platform to mine the massive genomics data to identify targetable RNA processing events and to design small-molecule glues to modulate the interactions among the spliceosome, regulatory proteins, and RNAs.
Rgenta is working closely with LLS TAP to further develop RNA-targeting molecules by supporting preclinical studies with the goal of moving towards clinical development in hematological malignancies.Program: Therapy Acceleration Program
Project Term: Start Date: June 30, 2023 End Date: September 19, 2023
In May 2023, LLS made an equity investment in Dimericon to "Support development of dimericons (crosslinked helix dimers) for blood cancers."
Dimericon is a private biotech company focused on exploring crosslinked helix dimers (Dimericons) as therapeutics and templates for small molecule development. Dimericon’s technology targets hard-to-drug intracellular protein-protein interactions using rationally designed mimetics of helix dimers. The Seed round of financing will support preclinical studies to further develop the current cFLIP inhibitor lead compound, DMRX1004, to be an IND ready clinical candidate in hematological malignancies.Program: Therapy Acceleration Program
Project Term: Start Date: May 24, 2023 End Date: September 19, 2023
A phase 1/2 study of Bexmarilimab, an anti-Clever1 monoclonal antibody, in combination with azacitidine or azacitidine/venetoclax in patients with AML, MDS or CMML
In June 2022, LLS made an equity investment in Faron Pharmaceuticals to "Support Clinical Development of the Bexmarilimab Program for Leukemia Indications."
Faron is a clinical stage biopharmaceutical company developing novel treatments for medical conditions with significant unmet needs caused by dysfunction of our immune system. The Company currently has a pipeline based on the receptors involved in regulation of immune response in oncology, organ damage and bone marrow regeneration.
Bexmarilimab, a novel anti-Clever-1 humanized antibody, is its investigative precision immunotherapy with the potential to provide permanent immune stimulation for difficult-to-treat cancers through targeting myeloid function. A Phase 1 study (BEXMAB) of bexmarilimab in combination with azacitidine or azacitidine/venetoclax is currently enrolling AML, MDS or CMML patients in the US and Finland (NCT05428969).Program: Therapy Acceleration Program
Project Term: Start Date: June 30, 2022 End Date: September 19, 2023
A phase 1 expansion study of ICT01, an anti-BTN3A monoclonal antibody, in combination with azacitidine and venetoclax in patients with AML
In June 2022, LLS made an equity investment in ImCheck Therapeutics to "Support Clinical Development of the ICT01 Program for Blood Cancer Indications."
ImCheck Therapeutics is designing and developing a new generation of immunotherapeutic antibodies targeting butyrophilins, a novel super-family of immunomodulators.
ICT01 is a humanized, anti-BTN3A (also known as CD277) monoclonal antibody that selectively activates γ9δ2 T cells, which are part of the innate immune system that is responsible for immunosurveillance of malignancy and infections. A clinical trial (EVICTION) is currently enrolling a cohort expansion of ICT01 in combination with azacitidine and venetoclax in patients with acute myeloid leukemia (NCT04243499).Program: Therapy Acceleration Program
Project Term: Start Date: June 13, 2022 End Date: September 19, 2023
In November 2010, LLS made an equity investment in BioTheryX originally to support the development of a promising LLS-funded project with Toronto-based University Health Network and is currently supporting "An Open Label, Escalating Multiple Dose Study to Evaluate the Safety, Toxicity, Pharmacokinetics, and Preliminary Activity of BTX-1188 in Subjects With Advanced Malignancies."
BioTheryX has a technology platform in the field of targeted protein degradation. This technology utilizes the body's own protein disposal system to selectively degrade and remove disease-causing proteins. It has potential applicability to a very broad range of disease targets, including a wide range of targets that have to date been considered "undruggable."
BTX-1188 is a dual target protein degrader specifically engineered to degrade GSPT1 and IKZF1/3. A clinical study of BTX-1188 in patients with advanced hematologic (AML or NHL) and solid tumor malignancies is currently enrolling (NCT05144334).Program: Therapy Acceleration Program
Project Term: Start Date: November 16, 2010 End Date: September 19, 2023
In February 2021, LLS made an equity investment in Caribou Biosciences to "Support allogeneic CD371 (CLL-1) CAR development for acute myeloid leukemia."
Caribou is a clinical-stage biotechnology company, co-founded by CRISPR pioneer and Nobel Prize winner Jennifer Doudna, Ph.D., using next-generation CRISPR genome-editing technology to develop “off-the-shelf” (allogeneic) CAR therapies for hard-to-treat blood cancers.
CB-012, Caribou’s third allogeneic CAR-T cell therapy, an allogeneic anti-CD371 CAR-T cell therapy for the treatment of relapsed or refractory acute myeloid leukemia (AML) is in preclinical development for the treatment of acute myeloid leukemia with a projected IND filing in the second half of 2023. CD371 is expressed on the surface of AML tumor cells and leukemic stem cells, but it is not expressed on normal hematopoietic stem cells, which makes it a compelling target for the treatment of AML. Caribou is applying their genome editing expertise to armor the CB-012 CAR-T product in order to drive persistence and seek maximum patient benefit.
Program: Therapy Acceleration Program
Project Term: Start Date: February 28, 2021 End Date: September 19, 2023
A phase 2 registration-directed clinical study of ziftomenib (KO-539), a menin inhibitor, in patients with NPM1-mutant relapsed or refractory AML
Starting in July 2010, LLS TAP supported a promising University of Michigan research project led by Jolanta Grembecka, PhD, to develop new treatments for patients with a rare and lethal subtype of leukemia. Through TAP, LLS engaged chemists to improve the properties that produced lead compounds that exhibited potent anti-leukemic activity. In 2014, LLS introduced Kura Oncology to the project that ultimately led to Kura Oncology completing a licensing agreement with the University of Michigan to continue to develop these molecules.
Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer with a pipeline that consists of small molecule drug candidates that target cancer signaling pathways.
Ziftomenib (KO-539) is selective small molecule inhibitor of menin. Ziftomenib is currently in a Phase 2 registration-directed clinical trial in patients with NPM1-mutant relapsed or refractory AML (NCT04067336).Program: Therapy Acceleration Program
Project Term: Start Date: December 22, 2014 End Date: September 19, 2023
A phase 1 expansion study of IO-202, an antibody targeting LILRB4, in combination with azacitidine/venetoclax in patients with monocytic differentiation AML and CMML
In March 2021, LLS made an equity investment in Immune-Onc Therapeutics to support the "Phase 1 Clinical Development of IO-202, An Antibody Targeting LILRB4, for the Treatment of AML with Monocytic Differentiation and CMML."
Immune-Onc is a private, clinical-stage cancer immunotherapy company dedicated to the discovery and development of novel myeloid checkpoint inhibitors for cancer patients. The company aims to translate unique scientific insights in myeloid cell biology and immune inhibitory receptors to discover and develop first-in-class biotherapeutics that reverse immune suppression in the tumor microenvironment. Immune-Onc has a differentiated pipeline with a current focus on targeting the Leukocyte Immunoglobulin-Like Receptor subfamily B (LILRB) of myeloid checkpoints. The company’s work builds on early research by Chengcheng (Alec) Zhang, Ph.D. at the University of Texas Southwestern Medical Center that was also funded by LLS grants.
IO-202 is a first-in-class antibody targeting the LILRB4 and has entered a phase 1 cohort expansion clinical trial (NCT0437243) for the treatment of AML (IO-202 in combination with azacitidine and venetoclax) and CMML (IO-202 in combination with azacitidine).Program: Therapy Acceleration Program
Project Term: Start Date: March 5, 2021 End Date: September 19, 2023
In August 2017, LLS TAP partnered with Ryvu Therapeutics (formerly known as Selvita) to support "A Phase Ib Study of SEL120 in Patients With Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome."
Ryvu Therapeutics is a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology using a proprietary discovery engine platform.
RVU120 (SEL120) is a highly selective first-in-class CDK8/CDK19 small molecule inhibitor. RVU120 is currently in a Phase I clinical trial in patients with acute myeloid leukemia or high-risk myelodysplastic syndrome (NCT04021368), enrolling in the US and Poland.Program: Therapy Acceleration Program
Project Term: Start Date: August 7, 2017 End Date: December 31, 2023