Keros Therapeutics, Inc. · NASDAQ:KROS
Keros Therapeutics — Investor Presentation (November 2023)
33 slides from Keros Therapeutics's investor presentation, Nov 6, 2023
Slide 1: November 2023 Corporate Presentation
November 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 Transforming the Lives of Patients with Dysfunctional TGF-β 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 a 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
Slide 4: Hematology Franchise
B Hematology Franchise Corporate Presentation 4
Slide 5: KER-050 Investigational Treatment for Anemia and Thrombocytopenia in MDS
B KER-050 5 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 6: Myelodysplastic Syndromes (MDS)
BCorporate Presentation 6 MDS MDS is a collection of bone marrow disorders characterized by ineffective hematopoiesis and development of peripheral cytopenias Clinical Consequences The clinical consequences of MDS include anemia, bleeding, iron overload, cardiovascular disease and progression to acu
Slide 7: TGF-β Superfamily Plays a Critical Role in Hematopoiesis
7 TGF-b Superfamily Plays a Critical Role in Hematopoiesis Hematopoiesis, the process by which blood cells are produced in the bone marrow, requires the coordinated control of cell division and differentiation TGF-β superfamily signaling regulates differentiation and maturation of hematopoietic prec
Slide 8: KER-050 Phase 2 MDS Trial Design
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 ≥4
Slide 9: Baseline Characteristics
9Corporate 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 10: KER-050 Safety and Tolerability
10Corporate 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) 1 Related TEAEs leading to
Slide 11: Response Summary
11Corporate 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 8-weeks of post-baseline platelet data. Response Summary RP2D Patients1 All evaluable patients HTB
Slide 12: Reductions in Transfusion Burden and Sustained Hemoglobin Increases
12Corporate 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 13: Data Suggest KER-050 Elicited a Durable Response
13Corporate 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 14: KER-050 Investigational Treatment in Patients with Myelofibrosis
B KER-050 14 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 15: Myelofibrosis (MF)
BCorporate Presentation 15 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 16: Ongoing Phase 2 Trial of KER-050 in MF
BCorporate Presentation 16 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 17: KER-050 Treatment Increased Reticulocytes and Platelets in Patients with MF
BCorporate Presentation 17 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 18: Potential Opportunities Beyond the Treatment of Cytopenias in MDS and MF
18 Potential Opportunities Beyond the Treatment of Cytopenias in MDS and MF Clinical manifestations in MDS and MF extend beyond cytopenias and can involve disease- and treatment-related iron overload and progression to AML TGF-β superfamily signaling regulates many processes in the bone marrow micro
Slide 19: Pulmonary and Cardiovascular Franchise
Pulmonary and Cardiovascular Franchise Corporate Presentation 19
Slide 20: KER-012 Investigational Treatment for PAH and Cardiovascular Disorders
KER-012 Corporate Presentation 20 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 21: KER-012 Modified Activin Receptor IIB Ligand Trap and PAH
Corporate Presentation 21 KER-012 is a modified activin receptor IIB ligand trap: ▸Designed to rebalance TGF-β superfamily signaling ▸ Being developed for the treatment of pulmonary and cardiovascular disorders, including PAH ▸Designed to preferentially inhibit select ligands (activin A, activin B,
Slide 22: RKER-012 Reduced Pulmonary Arterial Pressure and Cardiac Fibrosis in Rodent PAH Models
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 23: KER-012 Preclinical and Phase 1 Clinical Trial Data
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 24: KER-012 Altered Expression of Serum Proteins and Lowered NT-proBNP
Natarajan H, et al Am J Respir Crit Care Med 2023;207:A1187 Additional exploratory biomarker data demonstrated initial target engagement at 0.75 mg/kg and maximum target engagement at 4.5 mg/kg Corporate Presentation 24 KER-012 Altered Expression of Serum Proteins Associated with Inflammation and Ex
Slide 25: TROPOS Trial: Global Phase 2 Trial of KER-012 in PAH
Corporate Presentation 25 TROPOS Trial: Global Phase 2 Clinical Trial of KER-012 in Patients with PAH In order to advance the understanding of a potential KER-012 benefit in heart disease, we plan to initiate an open-label Phase 2 biomarker clinical trial of KER-012 in patients with chronic heart fa
Slide 26: Neuromuscular Franchise
Neuromuscular Franchise Corporate Presentation 26
Slide 27: Muscle Loss and the TGF-β Pathway
Corporate Presentation 27 Muscle loss can occur as a consequence of many factors, including neuromuscular disease, disuse, aging and as a side effect of some therapies In neuromuscular diseases, muscle loss can result in muscle weakness and, with increased severity, can lead to loss of ambulation, r
Slide 28: KER-065 Designed to Address Neuromuscular Diseases, Initial Focus on DMD
KER-065 Corporate Presentation 28 Designed to Address Neuromuscular Diseases, with an initial focus on Duchenne Muscular Dystrophy (DMD)
Slide 29: KER-065 Overview
Corporate Presentation 29 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, wi
Slide 30: Treatment with RKER-065 Led to Higher Utrophin Levels in Mouse Model of DMD
Corporate Presentation 30 W T -v e h D 2 .m d x- v e h D 2 .m d -0 6 5 Treatment with RKER-065 Led to Higher Utrophin Levels in Mouse Model of DMD Muscle lacking dystrophin is easily damaged during the process of contraction Many approaches have been utilized to stabilize the muscle and provide resi
Slide 31: Proprietary Discovery Approach
Corporate Presentation 31 Proprietary Discovery Approach
Slide 32: Proprietary Discovery Approach
Corporate Presentation 32 We have developed a proprietary library of ActRII ligand traps by combining sequences from ActRIIA and ActRIIB ▸ We have engineered molecules that are designed to have the therapeutic properties of either or both parent molecules ▸ Our ActRII program has produced a broader
Slide 33: Anticipated Key Milestones
Corporate Presentation 33 ▸KER-050 ▸Complete enrollment in transfusion-dependent cohorts in Phase 2 MDS trial H1 2024 ▸Announce additional data from Part 2 of Phase 2 MDS trial ASH 2023 ▸Announce dose escalation data from Phase 2 MF trial ASH 2023 ▸KER-012 ▸Initiate Phase 2 open-label biomarker tria
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