Keros Therapeutics, Inc. · NASDAQ:KROS
Keros Therapeutics — Investor Presentation (August 2023)
33 slides from Keros Therapeutics's investor presentation, Aug 7, 2023
Slide 1: August 2023 Corporate Presentation
August 2023 Corporate Presentation
Slide 2: Forward-looking statements
Corporate Presentation 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," “
Slide 3: Focused on Disorders Linked to Dysfunctional TGF-β Superfamily Signaling
KER-065 therapeutic protein P R E C L I N I C A L Corporate Presentation 3 Focused on Transforming the Lives of Wide Range of Patients with Disorders Linked to Dysfunctional TGF-β Superfamily Signaling We believe our product candidates have the potential to unlock the full therapeutic benefits of mo
Slide 4: Hematology Franchise
B Hematology Franchise Corporate Presentation 4
Slide 5: TGF-β Superfamily Plays a Critical Role in the Bone Marrow Microenvironment
5 TGF-b 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
B KER-050 6 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 Corporate Presentation
Slide 7: Myelodysplastic Syndromes (MDS)
BCorporate Presentation 7 MDS MDS is a collection of bone marrow disorders characterized by ineffective hematopoiesis and peripheral cytopenias. Clinical Consequences The clinical consequences of MDS include anemia, bleeding, iron overload, cardiovascular disease and progression to acute myeloid leu
Slide 8: Current treatment options for symptomatic anemia
8Corporate Presentation Current treatment options for symptomatic anemia includes red blood cell (RBC) transfusions, erythropoiesis-stimulating agents (ESAs) and Reblozyl® ▸ RBC transfusions provide symptomatic relief of anemia, but are also associated with iron overload which can increase risk of A
Slide 9: Amended Phase 2 Clinical Trial Design and Dose Levels
B ▸ KER-050 administered subcutaneously once every four weeks (Q4W) Primary Objective: ▸ Assess safety and tolerability of KER-050 Select Efficacy Endpoints: ▸ IWG 2006 Hematological improvement-erythroid (HI-E): ▸ Hemoglobin increase of ≥1.5 g/dL for 8 weeks (in NT and LTB patients) ▸ Reduction of
Slide 10: Baseline Characteristics
10Corporate Presentation Baseline Characteristics RP2D dataset (n=59) Age, years, median (range) 74.0 (53-89) Male, n (%) 34 (57.6) RBC transfusion status, units per 8 weeks, n (%) Non-transfused (NT), 0 units 12 (20.3) Low Transfusion Burden (LTB), <4 units 16 (27.1) High Transfusion Burden (HTB),
Slide 11: KER-050 generally well-tolerated at RP2D
11Corporate Presentation Category RP2D dataset (n=59) Any TEAE, n (%) 53 (89.8) Any treatment related TEAE 19 (32.2) Any TE serious AE (TESAE) 20 (33.9) Any treatment-related TESAE 1 (1.7) Any TEAE leading to death 2 (3.4) Any TEAE leading to IMP Discontinuation1 6 (10.2) Data are presented as of a
Slide 12: Response Summary
12Corporate Presentation Data are presented as of a data cutoff date of April 3, 2023. *Percentage based on 34 patients who had at least 24 weeks of treatment or discontinued AND had both baseline and 8weeks of post-baseline platelet data. Response Summary RP2D Patients1 All evaluable patients HTB e
Slide 13: KER-050 reduced transfusion burden and increased hemoglobin
13Corporate Presentation Data are presented as of a data cutoff date of April 3, 2023. 1. 2 patients discontinued with insufficient data to determine 8-week transfusion reduction, and are not included in this plot; 2. Baseline hemoglobin calculated as average over 8-week pre-treatment period. Hemogl
Slide 14: Data Suggest KER-050 Elicited a Durable Response
14Corporate Presentation Data are presented as of a data cutoff date of April 3, 2023. 19 patients achieved HI-E or TI ▸During weeks 0-24 in RP2D patients with ≥24wk of treatment or who discontinued Median duration of response was 42.4 weeks 10/19 patients (52.6%) had ongoing response at time of dat
Slide 15: KER-050 Investigational Treatment in Patients with Myelofibrosis
B KER-050 15 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 Corporate Presentation
Slide 16: Myelofibrosis (MF)
BCorporate Presentation 16 MF MF is a rare cancer of the bone marrow in which the marrow is replaced by scar tissue and is not able to produce healthy blood cells Clinical Consequences MF is characterized by ineffective hematopoiesis, an enlarged spleen, bone marrow fibrosis and shortened survival.
Slide 17: Ongoing Phase 2 Clinical Trial in Patients with MF
BCorporate Presentation 17 Data are presented as of a data cutoff date of October 1, 2022. 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 Endpoints include: ▸ Evaluate the pharmacokinetics
Slide 18: KER-050 Treatment Increased Reticulocytes and Platelets in Patients with MF
BCorporate Presentation 18 Change by Study Arm Change by Transfusion Status NTD: Non–transfusion dependent Although variability was observed amongst the patients, treatment with KER-050 at the lowest dose in this trial (0.75 mg/kg) resulted in increased reticulocytes and platelets on aggregate, both
Slide 19: Case Study of Patient on KER-050 Monotherapy
BCorporate Presentation 19 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 increase in reticulocytes observed after a single dose of KER-050 was followed
Slide 20: KER-047 Designed to Treat Functional Iron Deficiency
B KER-047 20 A Novel Product Candidate Designed to Treat Functional Iron Deficiency That is a Consequence of Elevated ALK2 Signaling Corporate Presentation
Slide 21: ALK2 signaling controls hepcidin expression
BCorporate Presentation 21 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 responds to demands for iron by increasing or reducing the production of hepcidin, which leads to
Slide 22: KER-047 Oral Small Molecule Inhibitor of ALK2
BCorporate Presentation 22 KER-047 is a novel, oral, investigational small molecule inhibitor of ALK2 with low nanomolar IC50 PK/ADME: Suitable for 1x daily oral dosing There were no serious adverse events reported in the randomized, double-blind, placebo-controlled two-part Phase 1 clinical trial o
Slide 23: KER-047 Treatment of One IRIDA Patient
BCorporate Presentation 23 KER-047 Treatment of One IRIDA Patient Resulted in a Decrease in Hepcidin and an Increase in Reticulocyte Hemoglobin Laboratory Results Before, During, and After Administration of KER-047 for the First Low-Dose Cohort (n=1) In December 2022, we presented data from one pati
Slide 24: Pulmonary and Cardiovascular Franchise
Pulmonary and Cardiovascular Franchise Corporate Presentation 24
Slide 25: KER-012 Investigational Treatment for PAH and Cardiovascular Disorders
KER-012 Corporate Presentation 25 Investigational Treatment for Pulmonary Arterial Hypertension (PAH) and for Cardiovascular Disorders Ongoing Randomized, Phase 2, Double-blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of KER-012 in Combination with Background Therapy in
Slide 26: Pulmonary Arterial Hypertension and KER-012 Mechanism
Corporate Presentation 26 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 smooth muscle cell proliferation and inflammation ▸ This
Slide 27: RKER-012 Reduced Pulmonary Arterial Pressure, Right Ventricle Hypertrophy and Cardiac Fibrosis
1. K. Babbs, et al. Am J Respir Crit Care Med 2022;205:A5776; 2. Babbs K, et al. Am Heart Association Scientific Sessions 2021; RKER-012 = Research KER-012 fused with Fc region of murine IgG1 0.0 0.2 0.4 0.6 0.8 Fulton Index R V /( L V + S ) -15.7% -28.0% ✱ ✱ ✱ ✱ ns ✱ ✱ ns 0 20 40 60 80 100 Pulmonar
Slide 28: KER-012 Preclinical and Phase 1 Data Summary
PAH Domain Preclinical Data Phase 1 Clinical Trial1,2 MOA & Ligand Specificity: • Strong activin/GDF binding observed • Observed to be BMP-sparing vs. ActRIIA-Fc • We believe PD data support potential for maximal target engagement with doses in Phase 2 Fibrosis & Inflammation: Inflammation Fibro
Slide 29: Phase 2 Trial Design of KER-012 in PAH
Corporate Presentation 29 Primary Objective: ▸ To evaluate the effect of KER-012 on hemodynamics compared to placebo in participants on background PAH therapy Primary Endpoint: ▸ Change from baseline in pulmonary vascular resistance (PVR) at Week 24 Key Secondary Objective: ▸ To evaluate the effect
Slide 30: Neuromuscular Franchise
Neuromuscular Franchise Corporate Presentation 30
Slide 31: KER-065 for Neuromuscular Diseases, Initially Duchenne Muscular Dystrophy
KER-065 Corporate Presentation 31 Designed to Address Neuromuscular Diseases, with an initial focus on Duchenne Muscular Dystrophy (DMD)
Slide 32: KER-065 Selective Activin Receptor Ligand Trap
Corporate Presentation 32 KER-065 is a selective activin receptor ligand trap ▸Designed to inhibit the biological effects of myostatin and activin to increase skeletal muscle and bone mass, increase fat metabolism and reduce fibrosis ▸ Being developed for the treatment of neuromuscular diseases, wit
Slide 33: Anticipated Key Milestones
Corporate Presentation 33 ▸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 H2 2023 ▸Announce dose escalation data from Phase 2 MF trial H2 2023 ▸Initiate Part 2 of Phase 2 MF trial H2 2023 ▸KER-047
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