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CCCC • CIK 0001662579
Overview We are a clinical-stage biopharmaceutical company dedicated to delivering on the promise of targeted protein degradation, or TPD, science to create a new generation of small-molecule medicines that transform patients’ lives. Leveraging our proprietary TORPEDO platform, we efficiently design and optimize small molecule protein degraders that are highly active against their desired targets by harnessing the body’s natural process for destroying unwanted proteins. Our strategy is to develop degraders that modulate clinically validated disease pathways with best-in-class or first-in-class potential to address significant unmet patient needs. To date, our degraders have demonstrated oral bioavailability and catalytic activity, and we have also leveraged our capability to design compounds that are brain penetrant. Our Clinical and Preclinical Pipeline Our clinical pipeline has two oncology degraders, cemsidomide, an IKZF1 and IKZF3 degrader, for multiple myeloma, or MM, and CFT8919, an EGFR L858R degrader for non-small-cell lung cancer, or NSCLC. Our discovery strategy is focused on selecting targets where we believe TPD offers a compelling mechanistic rationale and first-in-class potential in inflammation, neuroinflammation and neurodegeneration diseases. Our solely-owned pipeline is reflected below. Our most advanced product candidate, cemsidomide, is an orally bioavailable MonoDAC (molecular glue type) degrader targeting IKZF1 and IKZF3, or IKZF1/3. Cemsidomide has the opportunity to address a significant unmet need across multiple lines of therapy in multiple myeloma supported by a strong mechanistic rationale, well-defined biology, and a differentiated safety profile with compelling anti-myeloma activity demonstrated in our Phase 1 trial in combination with dexamethasone. Our other clinical-stage oncology product candidate, CFT8919, is an orally bioavailable, allosteric, mutant-selective BiDAC degrader of epidermal growth factor receptor, or EGFR, with an L858R mutation in non-small cell lung cancer, or NSCLC. Our collaboration partner, Betta Pharma, is conducting clinical development of CFT8919 in Greater China. In preclinical studies, CFT8919 demonstrated equivalent anti-proliferation activity against the major EGFR-inhibitor resistance mutations, including L858R-C797S, L858R-T790M, and L858R-T790M-C797S compared to L858R single mutation in Ba/F3 cell models in vitro. We are further diversifying our internal pipeline by developing new degraders focused on inflammation, neuroinflammation, and neurodegeneration. We have engineered degraders that have successfully achieved blood-brain barrier penetration in preclinical studies, which is a key step in developing medicines with the potential to treat brain metastases in central nervous system diseases. This element is a key part of our strategy is selecting targets where there is a strong degrader rationale over other therapeutic modalities. We are also advancing a discovery pipeline in collaboration with Merck KGaA, and Roche. Our partnership strategy allows us to further expand our reach in both oncology and non-oncology indications. 1 Table of Contents Our Strategy We are committed to improving outcomes for patients through the discovery, development, and commercialization of novel therapies in oncology as well as inflammation, neuroinflammation, and neurodegeneration. Key elements of our strategy are to: • Advance our clinical oral oncology degrader programs. We currently are advancing two clinical programs that were developed from our proprietary TORPEDO platform and have the potential to improve patients’ outcomes. ◦ Develop cemsidomide as a potential best-in-class IKZF1/3 degrader: The Phase 1 trial of cemsidomide in combination with dexamethasone demonstrated a differentiated safety profile with compelling anti-myeloma activity. Cemsidomide is advancing to the next phase of development with an efficient regulatory strategy, which includes the potential for two accelerated approvals. Our development strategy includes a Phase 2 trial evaluating cemsidomide in combination with dexamethasone as well as a Phase 1b evaluating cemsidomide in combination with elranatamab, pursuant to the Pfizer Agreement (defined below). • Progress new internal discovery strategy focused on inflammation, neuroinflammation, and neurodegeneration, which are areas that could benefit from a TPD approach.…
From recent Form 4 filings
Directors who filed Form 4s in the last 2 years (not a full DEF 14A board roster)