Expanding the Druggable Space

Remix™ discovers and develops small molecules that modulate RNA processing to control gene and protein expression, allowing us to advance novel therapies. Remix’s proprietary drug discovery platform, REMaster™, identifies druggable target sites and active chemical matter bringing the promise of new medicines to patients.

Our Unique Rhythm

Deep expertise in data science and bioinformatics has identified novel target sites that are druggable via modulation of RNA processing.
Multiplexed high-throughput screening technologies that simultaneously screen dozens of novel target sites at an unprecedented scale.
A purpose-built chemical library enriched for compounds that can interact with RNA processing mechanisms.
Clinical development programs designed by industry experts and informed by patients and clinicians to advance relevant and impactful therapies.

RNA Processing

Altering Gene Expression

RNA processing is a tightly regulated mechanism of gene transcription, 5’ end capping, polyadenylation, alternative splicing and protein expression. In disease states, this machinery can become dysregulated through genetic and non-genetic means. Remix's proprietary REMaster™ platform has been built to have visibility into multiple steps of the RNA processing cascade for target discovery and modulation.

The Platform

Our Three Scientific Pillars

Data Science

Identifying druggable high value targets

Our teams have built an extensive transcriptome database that we leverage using machine learning to identify modulatable sites across diverse target areas. To date, the Remix™ team has identified more than 1.6M novel exon targets in the mRNA degradation space alone - 25-fold more than the largest public database. These exon targets are present in dozens of previously undrugged high value targets.

Biology & Biomolecular Sciences

Functional target validation and multiplexed high-throughput screens

Remix™ has developed a proprietary genetic toolkit to validate that novel exon targets can be modulated in cells and lead to functional outcomes on gene and protein expression. The validation process ensures biologically meaningful targets are selected for progression into high-throughput screening campaigns. Multiplexed cell-based screening assays are deployed against dozens of targets in parallel, identifying novel compounds with high specificity and selectivity.

Medicinal & Computational Chemistry

Proprietary libraries and computational approaches

Our extensive proprietary chemical library samples distinct regions of chemical space and continues to grow over time. Cutting-edge computational chemistry analysis has ensured the library diversity is sufficient for identification of lead chemical matter for drug discovery programs.

Turning Targets into Therapies

Designing Molecules for Unmet Patient Needs

Determined to deliver novel and impactful treatments, Remix™ sets about advancing our breakthrough discoveries into innovative and relevant medicines. Our first act is tackling previously undrugged oncogenic transcription factors to eradicate certain solid tumors and hematological malignancies. Next up, breakthrough science to deliver for patients with other malignancies and neurodegenerative diseases.

Pipeline

Broad pipeline of mRNA degrader small molecules
across Oncology and Neurodegeneration

Target/Compound

Mechanism

Indications

Pre-Clinical

Phase I

Phase II

Phase III

Rights

Anticipated Milestones

MYB/REM-422

phase ii

Mechanism: mRNA degrader

mRNA
degrader

Indication: Adenoid Cystic Carcinoma

Anticipated Milestone: ORR/DoR Data ~Mid ‘27

Anticipated Milestone: ORR/DoR
Data ~Mid ‘27

Indication: AML/MDS

Anticipated Milestone: RP2D Data ~Mid ‘27

Anticipated Milestone: RP2D Data
~Mid ‘27

Indication: Others (e.g. BRCA, lymphoma, CRC)

MYB is an undruggable transcription factor that is known to be an oncogenic driver of multiple solid tumors and hematological malignancies, such as adenoid cystic carcinoma (ACC) and acute myeloid leukemia/myelodysplastic syndromes (AML/MDS). REM-422 is a potent, selective, and oral small molecule mRNA degrader that induces the reduction of MYB mRNA and protein expression resulting in antitumor activity in MYB-dependent human tumor models. REM-422 is currently in Phase 1 clinical trials (NCT06297941).

Oncology
Program

Oncology Program

phase i

Mechanism: mRNA degrader

mRNA
degrader

Indication: MYC-dependent cancers (~25% of all cancers)

Additional
Targets

Additional Targets

phase i

Mechanism: Degradation

Degradation

Indication: Oncology and CNS