Keros Therapeutics, Inc. · NASDAQ:KROS
Keros Therapeutics — Investor Presentation (June 2021)
32 slides from Keros Therapeutics's investor presentation, Jun 22, 2021
Slide 1: June 2021 Corporate Presentation
June 2021 Corporate Presentation
Slide 2: Disclaimer
Disclaimer Statements contained in this presentation regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Words such as "anticipates," "believes," "expects," "intends," “plans,” “poten
Slide 3: Harnessing the Powerful Biology of the TGF-β Superfamily
Harnessing the Powerful Biology of the TGF-β Superfamily • Clinical-stage biopharmaceutical company developing novel therapeutics that target the TGF-β superfamily • Approach validated by marketed products, InfuseTM (BMP2) for spinal fusion and Reblozyl® (modified activin receptor IIB) for treatment
Slide 4: Differentiated Clinical Assets in Hematological and Musculoskeletal Disorders
Keros is Developing Differentiated Clinical Assets in Hematological and Musculoskeletal Disorders 4* Anticipated clinical milestones are subject to the impact of COVID-19 on our business.
Slide 5: KER-050
KER-050 A novel treatment designed to address diseases arising from ineffective hematopoiesis • Myelodysplastic syndromes • Myelofibrosis 5
Slide 6: Myelodysplastic Syndromes (MDS) Overview
Myelodysplastic Syndromes (MDS) Overview *MDS Foundation • Hematologic malignancy predominantly affecting the elderly, resulting in multiple cytopenias (anemia, thrombocytopenia, neutropenia) due to ineffective hematopoiesis • 60,000-170,000 MDS patients in U.S.* • 15,000-20,000 newly diagnosed MDS
Slide 7: Myelofibrosis (MF) is Characterized by Ineffective Hematopoiesis
Myelofibrosis (MF) is Characterized by Ineffective Hematopoiesis *Gangat 2011;**Srour 2016; ***Naymagon 2017 16,000-18,500 Prevalence of MF patients in US* >3,000 New MF patients diagnosed each year** ~100 % Nearly all MF patients will become transfusion- dependent*** 51% Anemia 38% Transfusion Depe
Slide 8: KER-050 is a Modified ActRII Fusion Protein
KER-050 is a Modified ActRII Fusion Protein • Activin receptors are expressed on hematopoietic cells and modulate differentiation of precursor cells • KER-050 is a ligand trap composed of a modified extracellular domain of activin receptor IIA (ActRIIA) fused to the Fc region of human IgG • KER-050
Slide 9: RKER-050 Increased Erythropoiesis and Serum Erythropoietin in Preclinical Study
Treatment with RKER-050 Increased Erythropoiesis by Potentially Promoting Maturation at Multiple Stages and Increased Serum Erythropoietin • In a preclinical study conducted in mice, a single, subcutaneous 10 mg/kg dose of a mouse version of KER-050 (RKER-050) resulted in: • Rapid increase in RBCs •
Slide 10: KER-050 Completed First-in-Human Trial
KER-050 Completed First-in-Human Trial • First-in-human trial was designed to explore the safety, tolerability and PK in healthy volunteers with a secondary objective to evaluate changes in PD (hematology and bone biomarkers) • Observed that KER-050 drug levels were dose proportional in Part 1 of th
Slide 11: KER-050 Phase 1 Trial Recapitulated Learnings from Preclinical Studies
KER-050 Phase 1 Clinical Trial Recapitulated Learnings from Preclinical Studies • Single, subcutaneous administration of KER-050 in healthy volunteers • Observed rapid increase in red blood cell parameters is supportive of acceleration of maturation of late-stage precursors • Reticulocytes, red bloo
Slide 12: KER-050 Led to Clinically Meaningful Changes in Platelets after a Single Dose
KER-050 Treatment was Observed to Lead to Clinically Meaningful Changes in Platelets after a Single Dose Mean Change from Baseline in Platelets at Each Dose -1 2 4 7 15 21 29 -40 -20 0 20 40 60 Days Post-Dose Placebo 0.05 mg/kg 0.5 mg/kg 1.5 mg/kg 4.5 mg/kg 30 x 109 cells/L 20 x 109 cells/L Ch an ge
Slide 13: KER050-MD-201 Phase 2 Trial in MDS Anemia
KER050-MD-201 A Phase 2 Clinical Trial Of KER-050 For The Treatment Of Anemia In Patients With Very Low, Low Or Intermediate Risk Myelodysplastic Syndromes (MDS)
Slide 14: Phase 2 Clinical Trial of KER-050 in MDS
Phase 2 Clinical Trial of KER-050 in MDS • Phase 2, multicenter, open-label clinical trial in very low-, low- and intermediate-risk MDS patients • KER-050 administered once every four weeks (Q4W) for 12 weeks • Trial objectives: • Safety, tolerability and pharmacokinetics • Evaluate pharmacodynamic
Slide 15: Phase 2 Trial in MDS Key Eligibility Criteria and Endpoints
Phase 2 Clinical Trial of KER-050 in MDS Key Eligibility Criteria: • MDS with very low-, low-, or intermediate-risk disease, as classified by the International Prognostic Scoring System- Revised, including both RS positive and non-RS • ESA naïve and experienced patients are eligible • No prior treat
Slide 16: Trial Status and Baseline Characteristics
Trial Status and Baseline Characteristics • Data cut-off date: May 14, 2021 • Preliminary data presented from two lowest dose cohorts: • Cohort 1: 0.75 mg/kg Q4W for 12 weeks • Cohort 2: 1.5 mg/kg Q4W for 12 weeks • 12 patients received at least one dose of KER-050 as of the data cut-off date • 9 pa
Slide 17: Safety Profile as of May 14, 2021
Safety Profile as of May 14, 2021 • Safety Review Committee has reviewed preliminary 0.75 mg/kg (Cohort 1) and 1.5 mg/kg (Cohort 2) data • Summary of safety profile* (Cohorts 1 and 2; n=12): • No drug related serious adverse events (SAEs) • 4 treatment-emergent SAEs deemed unrelated to study drug (a
Slide 18: Preliminary Results from Phase 2 Clinical Trial
Preliminary Results from Phase 2 Clinical Trial Preliminary results*: • Consistent with the data from the Phase 1 clinical trial, increases in reticulocytes, Hgb and platelets were observed in MDS patients • 5 patients that completed 8 weeks of treatment with KER-050 as of the data cut-off date met
Slide 19: Reduction in Transfusion Burden Observed
Reduction in Transfusion Burden Observed+ +Data cut-off date: May 14, 2021 *Baseline transfusion burden over 8 weeks **Percent transfusion reduction over 8 weeks on treatment compared to baseline transfusion burden • Patients requiring transfusions at baseline (≥2 RBC units/8 weeks) that completed 8
Slide 20: Summary of KER-050 Phase 2 Clinical Trial
Summary of KER-050 Phase 2 Clinical Trial • Keros believes the initial preliminary data from this 12-week treatment Phase 2 clinical trial demonstrate proof-of-concept of KER-050 in patients with very low-, low- or intermediate-risk MDS • Data consistent with observations from the Phase 1 clinical t
Slide 21: KER-047
KER-047 A novel treatment designed to address: • Anemia resulting from iron imbalance • Iron deficiency anemia • IRIDA • Fibrodysplasia ossificans progressiva (FOP) 21
Slide 22: ALK2 Regulates Hepcidin and Iron Homeostasis
• ALK2 signaling in the liver controls hepcidin expression, a hormone that controls iron homeostasis • Excessive ALK2 signaling results in high hepcidin and a shortage of iron availability for RBC production • ALK2 signaling requires BMP ligand and the co-receptor hemojuvelin • Hepcidin expression i
Slide 23: Inhibition of ALK2 Demonstrated Activity in Rodent Models of Iron Imbalance
Inhibition of ALK2 Demonstrated Activity in Rodent Models of Iron Imbalance • ALK2 inhibition decreased hepcidin and increased serum iron in mice • In a mouse model of IRIDA, treatment with ALK2 inhibitors reduced hepcidin and ameliorated anemia • In a mouse model of chronic kidney disease, chronic
Slide 24: Phase 1 Clinical Trial of KER-047 in Healthy Volunteers
Phase 1 Clinical Trial of KER-047 in Healthy Volunteers • KER-047 is a small molecule inhibitor of the ALK2 kinase domain with low nanomolar IC50 • PK/ADME: Suitable for 1x daily oral dosing • In November 2020, Keros completed a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical
Slide 25: KER-047 Led to Reduced Hepcidin Levels and Increased Reticulocyte Hemoglobin
• Consistent with ALK2 inhibition, decreases in serum hepcidin were observed in Cohorts 1 through 3 of Part of the expanded trial Phase 1 Clinical Trial: KER-047 Treatment Led to Reduced Hepcidin Levels and Increased Hemoglobin Content in Reticulocytes • An increase in reticulocyte hemoglobin was ob
Slide 26: Phase 2 Trials to Provide Proof-of-Concept in IDA and IRIDA
Phase 2 Trials to Provide Proof-of-Concept for Treatment of Anemia Resulting from Iron Imbalance, Including IDA and IRIDA • KER-047 is designed to normalize high hepcidin levels, restore serum iron and ameliorate anemia • We expect to initiate a Phase 2 clinical trial in patients with IRIDA in H2 20
Slide 27: KER-012
KER-012 A preclinical program designed to address • Bone loss disorders such as osteoporosis and osteogenesis imperfecta • Pulmonary arterial hypertension (PAH) 27
Slide 28: KER-012: Preclinical Product Candidate
KER-012: Preclinical Product Candidate • Proprietary selective activin receptor ligand trap in preclinical development for the treatment of pulmonary arterial hypertension (PAH) and bone disorders • In preclinical studies, KER-012: • Demonstrated high affinity for, and potent inhibition of, ligands
Slide 29: Keros Summary
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Slide 30: Keros is Positioned for Clinical and Commercial Success
We Believe Keros is Positioned for Clinical and Commercial Success • Keros is focused on the development of novel TGF-β therapeutics • Robust biology that has been validated in the clinic • Keros is well-positioned to harness the potential of the TGF-β superfamily • ActRII program (KER-050) is in a
Slide 31: Anticipated Key Milestones
Anticipated Key Milestones* KER-050 • Announce additional data from Part 1 of Phase 2 trial in MDS End of 2021 • Initiate Phase 2 trial in myelofibrosis Q3 2021 (initial data 2022) KER-047 • Initiate Phase 2 trial in IDA H2 2021 (initial data 2022) • Initiate Phase 2 trial in IRIDA H2 2021 (initial
Slide 32: Thank You
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