Pipeline ++ Our Pipeline

Modalities
Programs (Targets)
Discovery
Lead Optimization
IND Enabling Studies
Phase 1
Phase 2
Phase 3
Molecular Glues
BTX-1188 (GSPT1 + IKZF1/3)
Hematological Malignancies, Solid Tumors

BTX-1188, our lead product candidate, is a rationally designed dual-targeting molecular glue degrader of GSPT1 and IKZF1/3 that we are developing for the treatment of hematologic malignancies and solid tumors. We believe that the combination of degrading GSPT1, a promising oncology target, and IKZF1/3, clinically validated immunomodulatory targets, provides advantages relative to GSPT1-only degradation. We are currently enrolling patients in a Phase 1/2 clinical trial of BTX-1188 with preliminary results expected in mid-2023.

Undisclosed glue discovery
Various

We have a broad discovery program leveraging parallel in silico and in vitro approaches to identify novel neosubstrates that could be degraded by Cereblon-based molecular glues. This effort has already identified several potential new targets previously considered undruggable and with relevance across a range of cancers. We look forward to translating these efforts into novel molecular glue programs in the future.

Bifunctional Degraders
SOS1
KRAS Mutant Cancers

We are developing degraders of Son of Sevenless 1 (SOS1), an important upstream target in the Kirsten rat sarcoma virus (KRAS) pathway. We are also in early stages of discovery in developing degraders directly targeting KRAS. KRAS is the most frequently mutated cancer-causing oncogene and is mutated in over 20% of all human cancers, including an estimated 90% of pancreatic tumors, 45% of colorectal tumors, and 30% of lung tumors. In preclinical models, we have demonstrated that our lead candidates rapidly and potently degraded SOS1 by up to 90% with inhibition of pan-KRAS mutant cancer cell lines.

CDK2/4/6
Solid Tumors

We are developing degraders of cyclin-dependent kinase 2 (CDK2), cyclin-dependent kinase 4 (CDK4) and cyclin-dependent kinase 6 (CDK6), important targets for a range of cancers including 30% of breast and esophageal cancers, 50% of pancreatic and 40% of bladder cancers. CDK4 and CDK6 are clinically validated targets in the context of breast cancers. In preclinical breast cancer models, our lead candidates potently degrade CDK4 and CDK6 resulting in robust inhibition of phosphorylated Rb and cell cycle arrest.

PDE4
Inflammatory Diseases

We are developing degraders of Phosphodiesterase 4 (PDE4), a validated target for auto-immune and inflammatory diseases such as psoriasis. Our first-in-class degraders are highly selective for PDE4 degradation and have shown greater potency than PDE4 inhibitors in terms of inhibition of key cytokines such as TNF-α, IFN-γand IL-1β in preclinical models.

Programs (Targets): BTX-1188 (GSPT1 + IKZF1/3)
Modalities: Molecular Glues
Hematological Malignancies, Solid Tumors
Phase 1

BTX-1188, our lead product candidate, is a rationally designed dual-targeting molecular glue degrader of GSPT1 and IKZF1/3 that we are developing for the treatment of hematologic malignancies and solid tumors. We believe that the combination of degrading GSPT1, a promising oncology target, and IKZF1/3, clinically validated immunomodulatory targets, provides advantages relative to GSPT1-only degradation. We are currently enrolling patients in a Phase 1/2 clinical trial of BTX-1188 with preliminary results expected in mid-2023.

Programs (Targets): Undisclosed glue discovery
Modalities: Molecular Glues
Various
Discovery

We have a broad discovery program leveraging parallel in silico and in vitro approaches to identify novel neosubstrates that could be degraded by Cereblon-based molecular glues. This effort has already identified several potential new targets previously considered undruggable and with relevance across a range of cancers. We look forward to translating these efforts into novel molecular glue programs in the future.

Programs (Targets): SOS1
Modalities: Bifunctional Degraders
KRAS Mutant Cancers
Lead Optimization

We are developing degraders of Son of Sevenless 1 (SOS1), an important upstream target in the Kirsten rat sarcoma virus (KRAS) pathway. We are also in early stages of discovery in developing degraders directly targeting KRAS. KRAS is the most frequently mutated cancer-causing oncogene and is mutated in over 20% of all human cancers, including an estimated 90% of pancreatic tumors, 45% of colorectal tumors, and 30% of lung tumors. In preclinical models, we have demonstrated that our lead candidates rapidly and potently degraded SOS1 by up to 90% with inhibition of pan-KRAS mutant cancer cell lines.

Programs (Targets): CDK2/4/6
Modalities: Bifunctional Degraders
Solid Tumors
Lead Optimization

We are developing degraders of cyclin-dependent kinase 2 (CDK2), cyclin-dependent kinase 4 (CDK4) and cyclin-dependent kinase 6 (CDK6), important targets for a range of cancers including 30% of breast and esophageal cancers, 50% of pancreatic and 40% of bladder cancers. CDK4 and CDK6 are clinically validated targets in the context of breast cancers. In preclinical breast cancer models, our lead candidates potently degrade CDK4 and CDK6 resulting in robust inhibition of phosphorylated Rb and cell cycle arrest.

Programs (Targets): PDE4
Modalities: Bifunctional Degraders
Inflammatory Diseases
Lead Optimization

We are developing degraders of Phosphodiesterase 4 (PDE4), a validated target for auto-immune and inflammatory diseases such as psoriasis. Our first-in-class degraders are highly selective for PDE4 degradation and have shown greater potency than PDE4 inhibitors in terms of inhibition of key cytokines such as TNF-α, IFN-γand IL-1β in preclinical models.

*Full pipeline is wholly owned by Biotheryx.

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Pipeline ++ Partner With Us

We are open to collaboration opportunities that fit with our mission and pipeline. Contact us at [email protected].