Remix Therapeutics, Inc.

REM-422, a First-in-Class Small Molecule MYB mRNA Degrader, Demonstrates Anti-Leukemic Activity as Monotherapy and in Combination in Preclinical Models of AML WATERTOWN, Mass., Nov. 20, 2024 /PRNewswire/ -- Remix Therapeutics (Remix), a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address the underlying drivers of disease, today announced an upcoming oral presentation at the 66th ASH Annual Meeting taking place from December 7-10, 2024, in San Diego, CA. Results demonstrate oral dosing of REM-422, a selective mRNA degrader of the MYB oncogene, leads to robust anti-leukemic activity observed both as a monotherapy and in combination across a genetically diverse set of preclinical models of acute myeloid leukemia (AML), including eradication of AML blasts in engrafted patient-derived xenograft (PDX) models of AML. The presentation also highlights the differentiated mechanism of action of REM-422 as it can be combined effectively with other agents used in the treatment of AML/MDS and retains activity in cell models engineered with mutations known to confer resistance to other targeted agents. "The preclinical data for REM-422 provide strong therapeutic rationale for our ongoing Phase I study in AML and High-Risk MDS," said Peter Smith, Ph.D., President and Chief Executive Officer of Remix Therapeutics. "REM-422 is the first compound from our REMaster™ platform to enter clinical development. Its unique mechanism of action, coupled with the robust anti-leukemic activity observed both as a monotherapy and in combination, positions REM-422 as a promising candidate to address the unmet needs in these patient populations." The MYB oncogenic transcription factor plays a crucial role in hematopoietic cell differentiation and proliferation. Its dysregulation and aberrant activity have been identified in various cancers, including adenoid cystic carcinoma (ACC), AML, acute lymphoblastic leukemia (ALL), and lymphoma. In AML, functional genomics studies have demonstrated a lineage-wide dependency on MYB, consistent with its involvement in disease driven by multiple oncogenic abnormalities (e.g. MLLr, NPM1, FLT3, p53, etc.). REM-422 is a first-in-class, potent, selective, oral small molecule degrader of MYB mRNA currently in clinical development for AML/HR-MDS (NCT06297941) and ACC (NCT06118086). It functions by inducing the inclusion of a normally unused 'poison exon' (PE) in the MYB pre-mRNA transcript, activating the nonsense-mediated decay pathway and preventing MYB protein expression. Details for the oral presentation are as follows: Title: REM-422, a Small Molecule MYB mRNA Degrader, Demonstrates Anti-Leukemic Activity As Monotherapy and in Combination with Standards of Care in Preclinical Models of AML Speaker: Samantha Levin-Furtney, Scientist, Remix Therapeutics Session: 604- Molecular Pharmacology and Drug Resistance: Myeloid Neoplasms: New Targets and Drugs Date: Monday, December 9, 2024 Time: 3:30 PM (Session time: 2:45-4:15 PM) Location: Manchester Grand Hyatt San Diego, Seaport Ballroom ABCD About REM-422 REM-422 is a first-in-class, potent, selective, and oral small molecule mRNA degrader that induces the reduction of MYB mRNA and subsequent protein expression. REM-422 functions by facilitating the incorporation of poison exons within the mRNA transcript, leading to nonsense-mediated decay (NMD) of the transcript. REM-422 addresses MYB dysregulation, a driver of oncogenesis, upstream of protein expression. About AML/HR-MDS Acute myeloid leukemia (AML), a rare cancer of the blood and bone marrow, is the most common type of acute leukemia in adults. AML is caused by genetic mutations within bone marrow cells, which in turn causes the production of leukemic white blood cells that crowd out healthy blood cells. This may cause problems with bleeding, infection, and anemia. Myelodysplastic Syndromes (MDS) are disorders of blood-forming units in the bone marrow. High-risk (HR)-MDS patients have a higher percentage of blast cells in the bone marrow and that, in many cases, progresses to AML. There are several approved agents to treat AML, however, many patients relapse after achieving a response, underscoring the need for new therapies. About Remix Therapeutics Remix Therapeutics is a clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and treat diseases. The REMaster™ technology platform facilitates RNA processing pattern identification, leveraging these learnings to modulate gene expression. Remix's innovative therapeutic approach has the potential to alter the way genes are read from the genome, to correct, enhance, or eliminate the gene message, thereby addressing disease drivers at their origin. For more information visit . Media Contact: Peg Rusconi Deerfield Group [email protected] Investor Contact: Will O-Connor Precision AQ [email protected] SOURCE Remix Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

WATERTOWN, Mass., Oct. 31, 2024 /PRNewswire/ -- Remix Therapeutics (Remix), a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address underlying drivers of disease, today announced the publication of a collaborative research study with Prof. Juan Valcárcel at the Center for Genomic Regulation in Barcelona, Spain in the prestigious journal Science. The research article, titled "Transcriptome-wide splicing network reveals specialized regulatory functions of the core spliceosome," represents a significant advancement in understanding the complexities of RNA splicing regulation. The study, conducted by the Valcárcel lab in collaboration with Remix Therapeutics, unveils distinct regulatory networks within the spliceosome and sheds light on how its intricate structural and conformational complexity modulates splice site selection. By knocking down the expression of 305 spliceosome components and regulators in human cancer cells and employing deep RNA sequencing, the researchers monitored over 250,000 alternative splicing events across all classes of splice site selection modes. "This groundbreaking research provides mechanistic insights into the intricate workings of the spliceosome and its regulatory functions," said Dom Reynolds, CSO at Remix Therapeutics. "The findings not only advance our understanding of RNA processing but also open new avenues for therapeutic interventions in diseases associated with splicing dysregulation." This collaborative effort led to several significant discoveries, including the unexpected finding that different protein components of the U1 snRNP complex, a well-studied ribonucleoprotein complex of the spliceosome, are differentially required for two classes of alternative splicing decisions, revealing an unprecedented division of labor in a core spliceosome component. This publication serves as a valuable resource for exploring targets of spliceosome components and associated regulatory factors, identifying modulators of alternative splicing events, and unraveling the complex regulatory relationships within one of the most intricate molecular machineries in eukaryotic cells. "Our study uncovered complex networks within the spliceosome that control how genes are spliced. Unexpectedly, we found that different parts of a key spliceosome component, the U1 snRNP complex, have distinct roles in alternative splicing decisions. The precise topological arrangement of snRNP complexes involved in spliceosome assembly occurring after initial splice site recognition, determines their activities in splice site selection, uncovering the extensive regulatory potential of this complex machinery" said Prof. Juan Valcárcel. "Our research with Remix substantially advances our understanding of the space and provides the foundation for leveraging RNA processing to develop novel therapeutics across a wide spectrum of human diseases." The full article, "Transcriptome-wide splicing network reveals specialized regulatory functions of the core spliceosome" can be found here: . Additionally, Remix Therapeutics, in collaboration with Prof. Aaron Hoskins at the University of Wisconsin-Madison, recently published a mechanistic study on human U1 snRNP in Nature Communications. This publication sheds light on the binding mechanism of U1snRNP to 5'-splice sites and how small molecules can influence alternative splicing. The full article "A sequential binding mechanism for 5' splice site recognition and modulation for the human U1 snRNP" can be found here: . About Remix Therapeutics Remix Therapeutics is a clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and treat disease. The REMaster™ technology platform facilitates RNA processing pattern identification, leveraging these learnings to modulate gene expression. Remix's innovative therapeutic approach has the potential to alter the way genes are read from the genome, to correct, enhance, or eliminate the gene message, thereby addressing disease drivers at their origin. For more information visit . Contacts: Media Contact: Peg Rusconi [email protected] 202-930-4762 ext. 409 Investor Contact: Will O'Connor Stern Investor Relations [email protected] 212-362-1200 SOURCE Remix Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

CAMBRIDGE, Mass., Oct. 29, 2024 /PRNewswire/ -- Remix Therapeutics (Remix), a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address the underlying drivers of disease, today announced that its management team will host one-on-one meetings with investors at the Jefferies London Healthcare Conference on Tuesday, November 19, 2024 in London, UK. For those investors interested in scheduling a one-on-one meeting with the Remix management team, please contact your Jefferies representative. About Remix Therapeutics Remix Therapeutics is a clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and treat disease. The REMaster™ technology platform facilitates RNA processing pattern identification, leveraging these learnings to modulate gene expression. Remix's innovative therapeutic approach has the potential to alter the way genes are read from the genome, to correct, enhance, or eliminate the gene message, thereby addressing disease drivers at their origin. For more information visit . Contacts: Media Contact: Peg Rusconi Deerfield Group [email protected] Investor Contact: Will O'Connor Precision AQ [email protected] SOURCE Remix Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED