Keros Therapeutics, Inc. · NASDAQ:KROS
Keros Therapeutics — Investor Presentation (March 2022)
36 slides from Keros Therapeutics's investor presentation, Mar 9, 2022
Slide 1: March 2022 Corporate Presentation
March 2022 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 • Leveraging our extensive experience in TGF-β superfamily protein structure, function and protein engineering to generate a clinical pi
Slide 4: Keros is Developing 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 product candidate designed to address diseases arising from ineffective hematopoiesis • Myelodysplastic syndromes • Myelofibrosis 5
Slide 6: KER-050: A potential treatment for ineffective hematopoiesis in MDS and MF
KER-050: A potential treatment for ineffective hematopoiesis in Myelodysplastic Syndromes (MDS) and Myelofibrosis (MF) MDS • Hematologic malignancy predominantly affecting the elderly, resulting in multiple cytopenias (anemia, thrombocytopenia, neutropenia) due to ineffective hematopoiesis • 60-170,
Slide 7: 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 8: KER-050 Increased RBC Parameters and Platelets Following Single Doses in Phase 1
KER-050 Increased RBC Parameters and Platelets Following Single Doses in a Phase 1 Clinical Trial in Healthy Volunteers • Single doses of KER-050 observed to lead to clinically meaningful changes in platelets through day 29 • Maximum changes observed between 7-15 days post- dosing • Observed increas
Slide 9: KER-050 Preclinical Data Support Potential to Promote Multiple Stages of Erythropoiesis
KER-050 Preclinical Data Support Potential to Promote Multiple Stages of Erythropoiesis Bas lin e 12 hr 24 hr 0 20 40 60 80 100 120 7d 14 d 28 d 35 d 42 d Pe rc en t o f B as el in e L ab el ed C el ls Vehicle RKER-050 RKER-050 treatment did not alter RBC life span BFU-E CFU-E Pro-E Poly-EBaso-E RET
Slide 10: RKER-050 Preclinical Data Support Potential to Promote Multiple Stages of Thrombopoiesis
RKER-050 Preclinical Data Support Potential to Promote Multiple Stages of Thrombopoiesis • Observed rapid onset of platelet increase in mouse models is consistent with terminal maturation of proplatelets • Observed increase in the number of CD41+ cells and the polyploid in mouse models is consistent
Slide 11: RKER-050 Increased Platelets Potentially Through Activin A Inhibition
RKER-050 Increased Platelets Potentially Through Activin A Inhibition • Treatment with Activin A inhibited differentiation of platelet production in a preclinical study in mice • Decreased number of polyploid CD41+ cells (megakaryocytes) • In contrast, inhibition of activin A through administration
Slide 12: KER-050 Summary
KER-050 Summary • KER-050 increased RBC parameters and platelets following single doses in a Phase 1 clinical trial in healthy volunteers • In preclinical studies, a research form of KER-050 (RKER-050) was observed to increase RBCs and platelets, potentially through promotion of multiple stages of e
Slide 13: KER050-MD-201 Phase 2 Clinical Trial in MDS
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) 13
Slide 14: Phase 2 Clinical Trial of KER-050 in MDS
Phase 2 Clinical Trial of KER-050 in MDS 0.75 mg/kg (N=6) 1.5 mg/kg (N=6) 2.5 mg/kg (N=6) 3.75 mg/kg (N=6) Part 1: Dose Escalation (Open Label) Part 2: Dose Confirmation (Open Label) Dose TBD based on Part 1 • Phase 2, multicenter, open-label clinical trial in very low-, low- and intermediate-risk M
Slide 15: Phase 2 Clinical Trial of KER-050 in MDS Key Eligibility Criteria
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 patients that did not have ring sideroblasts (non-RS) and patients that have ring siderob
Slide 16: Trial Status and Baseline Characteristics
Trial Status and Baseline Characteristics • Abstract announcing additional data from our ongoing Phase 2 clinical trial of KER-050 in MDS was presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition • Data cut-off date: October 25, 2021 • Efficacy data presented from
Slide 17: Biomarkers of Ineffective Hematopoiesis in MDS Patients by RS Status
Biomarkers of Ineffective Hematopoiesis in Very Low-, Low- and Intermediate-Risk MDS Patients by RS Status Biomarker RS Status Mean (SD) RS+ (N=13) Non-RS (N=11) EPO (IU/L) 326.0 (826.2) 490.2 (855.7) Reticulocytes (109/L) 39.1 (28.0) 29.8 (24.8) Platelets (109/L) 228.4 (67.8) 134.6 (55.5) TPO (pg/m
Slide 18: Safety Data as of the Data Cut-off Date
Safety Data as of the Data Cut-off Date (October 25, 2021) • Safety Review Committee has reviewed data from 0.75 mg/kg (Cohort 1), 1.5 mg/kg (Cohort 2), 2.5 mg/kg (Cohort 3) and Cohort 4 (3.75 mg/kg) • Summary of safety data as of the data cut-off date (Cohorts 1-4, n=24) • No drug-related serious a
Slide 19: Preliminary Results from Phase 2 Clinical Trial
Preliminary Results from Phase 2 Clinical Trial Preliminary results*: • n=8/16 (50%) of the evaluable patients met at least one of the following endpoints: • 2006 IWG HI-E criteria: n=7/16 (43.8%) • Hemoglobin increase of ≥1.5 g/dL for 8 weeks (in NT and LTB patients) • Reduction of ≥4 RBC units tra
Slide 20: KER-050 Demonstrated Improvement in Erythropoiesis and Thrombopoiesis
KER-050 Demonstrated Improvement in Erythropoiesis and Thrombopoiesis KER-050 upregulated erythropoiesis • Reticulocyte increases observed in patients achieving HI-E or TI endpoints • Increases in serum soluble transferrin receptor (sTfR) and decreases in serum ferritin observed in patients achievin
Slide 21: Summary of KER-050 Phase 2 Clinical Trial
Summary of KER-050 Phase 2 Clinical Trial • Keros believes the additional 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 trial in h
Slide 22: Updated Design of KER050-MD-201
Updated Design of KER050-MD-201 • Cohort 4 Safety Review Committee permitted dosing of participants in Cohort 5 at 5.0 mg/kg, Q4W for 24 months • Preparing to initiate Part 2 of this trial, with centers participating globally • Data from Part 2 will inform our registration plans for KER-050 • Part 2
Slide 23: KER050-MF-301 Phase 2 Trial in Myelofibrosis
KER050-MF-301 A Phase 2 Clinical Trial to Evaluate KER- 050 as a Monotherapy or in Combination With Ruxolitinib in Myelofibrosis 23
Slide 24: Phase 2 Clinical Trial of KER-050 in Patients with Myelofibrosis-Associated Cytopenias
Phase 2 Clinical Trial of KER-050 in Patients with Myelofibrosis- Associated Cytopenias • Ongoing open label, two-part, multiple ascending dose Phase 2 clinical trial to evaluate KER-050 as a monotherapy and in combination with ruxolitinib in participants with myelofibrosis-associated cytopenias • P
Slide 25: KER-047
KER-047 A novel product candidate designed to address: • Anemia resulting from iron imbalance • Iron deficiency anemia • IRIDA 25
Slide 26: 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 27: 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 28: Phase 1: KER-047 Led to Reduced Hepcidin and Increased Reticulocyte Hemoglobin
• Consistent with ALK2 inhibition, decreases in serum hepcidin were observed in Cohorts 1 through 3 of Part 2 of the expanded trial • Treatment related decreases in hepcidin resulted in increased serum iron Phase 1 Clinical Trial: KER-047 Treatment Led to Reduced Hepcidin Levels and Increased Hemogl
Slide 29: Phase 2 Trials to Provide Proof-of-Concept for Anemia from Iron Imbalance
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 H1 20
Slide 30: KER-012
KER-012 A clinical program designed to address: • Pulmonary arterial hypertension (PAH) • Bone loss disorders such as osteoporosis and osteogenesis imperfecta 30
Slide 31: KER-012: Product Candidate for Treatment of PAH and Bone Disorders
KER-012: Product Candidate for Treatment of PAH and Bone Disorders Proprietary selective activin receptor ligand trap in clinical development for the treatment of pulmonary arterial hypertension (PAH) and bone disorders In preclinical studies, KER-012: • Demonstrated effects on bone • Exhibited high
Slide 32: Phase 1 Clinical Trial of KER-012 in Healthy Volunteers
Phase 1 Clinical Trial of KER-012 in Healthy Volunteers • Ongoing randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate single and multiple ascending doses of KER-012 in healthy volunteers • Trial objectives: safety, tolerability and pharmacokinetics Phase 1 Clini
Slide 33: Keros Summary
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Slide 34: We Believe 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 35: Anticipated Key Milestones
Anticipated Key Milestones* KER-050 • Announce additional data from Phase 2 trial in MDS Mid-2022 • Announce initial data from Phase 2 trial in myelofibrosis End of 2022 KER-047 • Initiate Phase 2 trial in IDA H1 2022 (initial data end of 2022) • Initiate Phase 2 trial in IRIDA H1 2022 (initial data
Slide 36: Thank You
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