Keros Therapeutics, Inc. · NASDAQ:KROS
Keros Therapeutics — Investor Presentation (January 2023)
32 slides from Keros Therapeutics's investor presentation, Jan 9, 2023
Slide 1: January 2023 Corporate Presentation
January 2023 Corporate Presentation
Slide 2: Disclaimer
Disclaimer 2 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,” “pot
Slide 3: Keros is Focused on Transforming the Lives of Patients
• Keros is a clinical-stage biopharmaceutical company developing differentiated product candidates designed to alter transforming growth factor-beta (TGF-β) signaling and target pathways critical for the growth, repair and maintenance of a number of tissue and organ systems • Targeting such pathways
Slide 4: Hematology Franchise
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Slide 5: TGF-β Superfamily Plays a Critical Role in the Bone Marrow Microenvironment
TGF-β Superfamily Plays a Critical Role in the Maintenance of the Bone Marrow Microenvironment • Hematopoiesis, the process by which blood cells are produced in the bone marrow, requires the coordinated control of cell division, differentiation and production of the specialized cellular machinery fo
Slide 6: KER-050 Investigational Treatment for Anemia and Thrombocytopenia in MDS
KER-050 Investigational Treatment for Anemia and Thrombocytopenia in Patients with Myelodysplastic Syndromes Ongoing Phase 2 Clinical Trial of KER-050 for the Treatment of Anemia in Patients with Very Low-, Low- or Intermediate-Risk Myelodysplastic Syndromes 6
Slide 7: Myelodysplastic Syndromes (MDS)
Myelodysplastic Syndromes (MDS) • MDS is a collection of bone marrow disorders characterized by ineffective hematopoiesis and peripheral cytopenias • The clinical consequences of MDS include anemia, bleeding, iron overload, cardiovascular disease and progression to acute myeloid leukemia (AML) • Med
Slide 8: Novel Treatment Options Are Needed to Address Unmet Need of Patients Living with MDS
Novel Treatment Options Are Needed to Address Unmet Need of Patients Living with MDS 8 Current Treatment Options: Treatment for symptomatic anemia includes red blood cell (RBC) transfusions, erythropoiesis-stimulating agents (ESAs) and Reblozyl® • ESAs’ benefit is limited to patients with low transf
Slide 9: Ongoing Phase 2 Clinical Trial of KER-050 in MDS
Ongoing Phase 2 Clinical Trial of KER-050 for the Treatment of Anemia in Patients with Very Low-, Low-, or Intermediate-Risk MDS 9 • KER-050 administered subcutaneously once every four weeks (Q4W) • Trial objectives in Part 1 (Dose Escalation): • Evaluate safety, tolerability and pharmacokinetics •
Slide 10: KER-050 Safety and Efficacy Update in Patients Receiving the Recommended Part 2 Dose
KER-050 Safety and Efficacy Update in Patients Receiving the Recommended Part 2 Dose* SLD: Single lineage dysplasia WHO: World Health Organization *Data cutoff date: 1-Oct-2022 10 In December 2022, we presented data from 36 patients receiving the RP2D as of the Oct 1-2022 data cut-off date • 36 pati
Slide 11: Sustained Transfusion Independence Response with Longer-Term Treatment with KER-050
Observed a Sustained Transfusion Independence Response with Longer-Term Treatment with KER-050 at the RP2D* Response Summary Response Rate, n/m (%) All evaluable patients HTB evaluable patients Overall Erythroid Response (HI-E or TI) 15/29 (51.7%) 10/16 (62.5%) IWG 2006 HI-E 15/29 (51.7%) 10/16 (62.
Slide 12: KER-050 Treatment Resulted in HI-E and TI with Sustained Increase in Platelets
KER-050 Treatment Resulted in HI-E and TI in Transfusion-Dependent Non-RS and RS+ Patients with Sustained Increase in Platelets* Altered visit schedules for patients re-baselined in Part 1 Extension following treatment gap contribute to fluctuating numbers across visits. RS+ Non-RS • KER-050 treatme
Slide 13: Long-term Transfusion Independence Achieved in a Patient Dosed at RP2D
Long-term Transfusion Independence Achieved in a Patient Dosed at RP2D 13 Study Weeks Part 1 Base Study, 1.5 mg/kg, HI-E responder Part 1 Extension, 2.5 mg/kg up to 3.75 mg/kg, TI responder -8 -4 0 4 8 12 16 20 24 28 32 36 40 44 48 1.5 mg/kg 2.5 mg/kg 3.75 mg/kg RBC Transfusion Units 1 2 3 HI-E or T
Slide 14: KER-050 Investigational Treatment in Myelofibrosis
KER-050 Investigational Treatment for Anemia and Thrombocytopenia in Patients with Myelofibrosis Ongoing Phase 2 Open-Label Clinical Trial to Evaluate the Safety and Efficacy of KER-050 as Monotherapy or in Combination with Ruxolitinib in Participants with Myelofibrosis 14
Slide 15: Myelofibrosis (MF)
Myelofibrosis (MF) • MF is a group of rare cancers of the bone marrow in which the marrow is replaced by scar tissue and is not able to produce healthy blood cells • MF is characterized by ineffective hematopoiesis, an enlarged spleen, bone marrow fibrosis and shortened survival. Patients often expe
Slide 16: Ongoing Phase 2 Clinical Trial to Evaluate KER-050 in Patients with MF
Ongoing Phase 2 Clinical Trial to Evaluate KER-050 as Monotherapy or in Combination with Ruxolitinib in Patients with MF • Primary objective: • Part 1: Assess safety and tolerability of KER-050 • Part 2: Confirm safety and tolerability of the dose(s) selected from Part 1 • Secondary objectives: • Ev
Slide 17: KER-050 Treatment Increased Reticulocytes and Platelets in MF Patients
KER-050 Treatment Increased Reticulocytes and Platelets in MF Patients* Change by Study Arm Change by Transfusion Status potential for KER-050 to promote differentiation of erythroid and megakaryocytic precursors and ameliorate anemia and thrombocytopenia wit • Although variability was observed amon
Slide 18: Increases in Red Blood Cells and Platelets in a Patient with KER-050
Observed Increases in Red Blood Cells and Platelets in a Patient with KER- 050 Treatment (Monotherapy)* Case Study of Patient on KER-050 Monotherapy Treatment at 0.75mg/kg Q4W • 60-year-old non-transfusion dependent female with primary MF • Treatment with KER-050 increased hematopoiesis • A robust i
Slide 19: KER-047 Novel Product Candidate for Anemia
KER-047 A Novel Product Candidate Designed to Address Anemia in Patients with: • Iron-Refractory Iron Deficiency Anemia (IRIDA) • Functional Iron Deficiency (FID) in MDS and MF 19
Slide 20: ALK2 Signaling Controls Hepcidin Expression
• ALK2 signaling controls hepcidin expression, a hormone that controls iron homeostasis • Hepcidin is the master regulator of iron flux into and out of storage tissues • The body exerts control and responds to demands for iron by increasing or reducing the production of hepcidin, which leads to a re
Slide 21: KER-047 Reduced Hepcidin Levels and Increased Hemoglobin Content in Reticulocytes
• Consistent with ALK2 inhibition, decreases in serum hepcidin were observed in Cohorts 1 through 3 of Part 2 of the trial • Treatment related decreases in hepcidin were associated with increased serum iron KER-047 Treatment Reduced Hepcidin Levels and Increased Hemoglobin Content in Reticulocytes i
Slide 22: KER-047 Treatment of One IRIDA Patient in a Phase 2 Clinical Trial
KER-047 Treatment of One IRIDA Patient in a Phase 2 Clinical Trial Resulted in a Decrease in Hepcidin and an Increase in Reticulocyte Hemoglobin 22 Ongoing, two-part, open-label dose-escalation and dose-expansion Phase 2 clinical trial in patients with IRIDA (an inherited form of iron deficiency ane
Slide 23: KER-012 Clinical Program for PAH and Cardiovascular Disorders
KER-012 A Clinical Program Designed to Address: • Pulmonary Arterial Hypertension (PAH) • Cardiovascular Disorders 23
Slide 24: Imbalances in TGF-β Superfamily Signaling Underlies Vascular Remodeling in PAH
Imbalances in TGF-β Superfamily Signaling Underlies Vascular Remodeling in PAH Thickened Vasculature Pulmonary Arterial Hypertension Pa PVR Myogenic & Fibrogenic Differentiation • PAH is a debilitating disorder characterized by elevated pulmonary vascular resistance due to increased vascular sm
Slide 25: RKER-012 Reduced Pulmonary Arterial Pressure and RV Hypertrophy in a Rat PAH Model
RKER-012 Reduced Pulmonary Arterial Pressure and Right Ventricle (RV) Hypertrophy in a Rat PAH Model • Hypoxic rats were dosed with vehicle, ActRIIA-Fc (10 mg/kg) or RKER-012 (10 mg/kg), twice weekly for three weeks • Normoxic rats were dosed with vehicle • Relative to vehicle-treated hypoxic rats,
Slide 26: KER-012 was Well Tolerated in a Phase 1 Trial in Healthy Volunteers
KER-012 was Well Tolerated in a Phase 1 Clinical Trial in Healthy Volunteers 26 Key Inclusion Criteria: • Postmenopausal females aged 45 to 70 years • Serum follicle stimulating hormone (FSH) levels > 40 IU/L • BMI >18.5 kg/m2 to <32.0 kg/m2 Part 1 and Part 2 Endpoints: • Safety and pharmacokinetics
Slide 27: KER-012 Indicates Maximal Target Engagement at the Highest Doses Tested
KER-012 Treatment Indicate Maximal Target Engagement at the Highest Doses Tested in Phase 1 Clinical Trial -80 -60 -40 -20 0 20 40 Study Day FS H % C ha ng e fro m B as el in e (m ea n ± SE M ) Placebo (n=6) 0.75 mg/kg (n=8) 1.5 mg/kg (n=6) 4.5 mg/kg (n=6) 1 5 7 11 15 22 293 Follicle stimulating hor
Slide 28: KER-012 Changed Pharmacodynamic Markers Consistent with Increased BMP Signaling in Bone
KER-012 Treatment Changed Pharmacodynamic Markers Consistent with Increased BMP Signaling in the Bone in Phase 1 Clinical Trial 28 Part 2 (MAD) 0 25 50 75 Mean Maximum Percent Change from Baseline in BSAP B SA P m ax im um % c ha ng e (m ea n ± SE M ) Placebo 0.75mg/kg 1.5 mg/kg 3.0 mg/kg 5.0 mg/kg
Slide 29: KER-012 Resulted in an Increase in Serum BSAP After Each Administration
In Part 2 of the Phase 1 Clinical Trial, Treatment with KER-012 Resulted in an Increase in Serum BSAP After Each Administration 1 15 29 43 57 71 -20 0 20 40 60 80 100 Study Day BS AP % c ha ng e fro m b as el in e (m ea n ± SE M ) Placebo (n=6) 0.75 mg/kg (n=8) 1.5 mg/kg (n=6) 4.5 mg/kg (n=6) = KER-
Slide 30: KER-012 Did Not Elicit Dose-Limiting Increases in Red Blood Cells
KER-012 Did Not Elicit Dose-Limiting Increases in Red Blood Cells in Phase 1 Clinical Trial 1 15 29 43 57 71 85 99 113 -2 -1 0 1 2 3 Study Day H em og lo bi n C ha ng e (g /d L, ± S EM ) Placebo (n=6) 0.75 mg/kg (n=8) 1.5 mg/kg (n=6) 4.5 mg/kg (n=6) 1 15 29 43 57 71 85 99 113 -1.0 -0.5 0.0 0.5 1.0 S
Slide 31: Keros Summary
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Slide 32: Anticipated Key Milestones
Anticipated Key Milestones* KER-050 • Complete enrollment in transfusion-dependent cohorts in Phase 2 MDS trial H2 2023 • Announce additional data from Part 2 of Phase 2 MDS trial H1 and H2 2023 • Expand Phase 2 MDS trial to MDS patients with iron overload H2 2023 • Announce dose escalation data fro
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