ASH 2017 Phase 2 Presentation Highlights. Pulmonary. December 11, Accelerating Drug Discovery to Transform Patients Lives

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1 ASH 2017 Phase 2 Presentation Highlights Pulmonary December 11, 2017 Accelerating Drug Discovery to Transform Patients Lives

2 Acceleron Forward-Looking Statements THIS PRESENTATION CONTAINS FORWARD-LOOKING STATEMENTS ABOUT THE COMPANY S STRATEGY, future plans and prospects, including statements regarding the development of the Company's compounds, the timeline for clinical development and regulatory approval of the Company s compounds and the expected timing for reporting of data from ongoing clinical trials. The words anticipate, believe, could, estimate, expect, goal, intend, may, plan, potential, project, should, target, will, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. ACTUAL RESULTS COULD DIFFER MATERIALLY FROM THOSE INCLUDED IN THE FORWARD-LOOKING statements due to various risks and uncertainties, including, but not limited to, that preclinical testing of the Company's compounds and data from clinical trials may not be predictive of the results or success of ongoing or later clinical trials, that the development of the Company's compounds will take longer and/or cost more than planned, that the Company or its collaboration partner, Celgene, will be unable to successfully complete the clinical development of the Company s compounds, that the Company or Celgene may be delayed in initiating, enrolling or completing any clinical trials, and that the Company's compounds will not receive regulatory approval or become commercially successful products. These and other risks and uncertainties are identified under the heading "Risk Factors" included in the Company's most recent Annual Report on Form 10-K, and other filings that the Company has made and may make with the SEC in the future. THE FORWARD-LOOKING STATEMENTS CONTAINED IN THIS PRESENTATION ARE BASED ON management s current views, plans, estimates, assumptions and projections with respect to future events, and the Company does not undertake and specifically disclaims any obligation to update any forwardlooking statements. 2

3 Habib Dable Chief Executive Officer

4 59 th American Society of Hematology Meeting (ASH) December 9-12, 2017 Myelodysplastic Syndromes Clinical Presentations Mutational Profile and Analysis of Lower-Risk Myelodysplastic Syndromes (MDS) Patients Treated with Luspatercept: Phase 2 PACE-MDS Study Myelofibrosis (Investigator Sponsored Trial) Sotatercept (ACE-011) Alone and in Combination with Ruxolitinib in Patients (pts) with Myeloproliferative Neoplasm (MPN)-Associated Myelofibrosis (MF) and Anemia 4

5 Hematology Trials: Chronic Anemia in Rare Blood Disorders Phase 1 Phase 2 Phase 3 Luspatercept Sotatercept MEDALIST Phase 3 Study COMMANDS Phase 3 Study (Planned) BELIEVE Phase 3 Study BEYOND Phase 2 Study Phase 2 Study Phase 2 Base/Extension Phase 2 Base/Extension Phase 2 IST MD Anderson Myelodysplastic Syndromes (MDS) Beta-Thalassemia Myelofibrosis (MF) MDS Beta-Thalassemia MF 5

6 Matthew Sherman, M.D. EVP and Chief Medical Officer

7 ASH 2015 MDS Presentation Informed MEDALIST Phase 3 Trial PACE-MDS Phase 2 ESA refractory or ESA ineligible/epo >500 IU/L (n=49) Response Rates IWG HI-E n/n (%) RBC-TI n/n (%) RS+ 22/40 (55%) 12/31 (39%) Non-RS 2/7 (29%) 2/7 (29%) RS Unknown 0/2 (0%) 0/2 (0%) Patients Treated at Dose Levels 0.75 mg/kg Data as of 31 Aug 2015 Lower-Risk MDS (RS+) Primary Endpoint RBC-TI during the first 24 weeks Key Secondary Endpoints IWG HI-E Duration of RBC-TI 7 EPO: erythropoietin; ESA: erythropoiesis stimulating agent, RS: ring sideroblast RBC-TI: red blood cell transfusion independence, IWG HI-E: hematologic improvement-erythroid

8 ASH 2017 Lower-Risk MDS Phase 2 Presentation PACE-MDS Phase 2 ESA refractory ESA ineligible/epo >500 IU/L (n=49) New PACE-MDS Expansion Cohorts Initiated in 1H 2016 ESA-naïve who are: RS+ and EPO 200 IU/L RS- and any EPO level ASH 2017 (n=99) Response Rates IWG HI-E n/n (%) RBC-TI n/n (%) All patients 52/99 (53%) 29/67 (43%) ESA-naïve 28/53 (53%) 17/31 (55%) Prior ESA 24/46 (52%) 12/36 (33%) Patients Treated at Dose Levels 0.75 mg/kg Data as of 08 Sept EPO: erythropoietin; ESA: erythropoiesis stimulating agent; RS: ring sideroblast RBC-TI: red blood cell transfusion independence, IWG HI-E: hematologic improvement-erythroid

9 COMMANDS Phase 3 Trial First-Line Lower-Risk MDS PHASE 3 PLANNED COMMANDS Patient Profile: Treatment naïve RS+ and RS- patients Head-to-head versus ESA

10 ASH 2017 Phase 2 Presentation: Increase in Mean Hemoglobin in LTB MDS Patients Patients treated at dose levels 0.75 mg/kg Low-transfusion-burden (LTB) patients: < 4 units/8 wk, Hb <10 g/dl Patients Treated at Dose Levels 0.75 mg/kg Data as of 08 Sept 2017

11 ASH 2017 Phase 2 Presentation: Durability of Response in RBC-TI Responders Study 03/05: High Dose, Baseline RBC >= 2 and RBC-TI Responder Months Median duration of treatment for EHA RBC-TI responders (n=23): 19.0 months for ASH 2017 versus 14.7 months at EHA Patients Treated at Doses 0.75 mg/kg with Baseline RBC 2U/8 weeks Data as of 08 Sept 2017 / 1 Data as of 13 Apr 2017

12 ASH 2017: Sotatercept MD Anderson Cancer Center Phase 2 IST in Myelofibrosis (MF) 3 cohorts of up to 20 patients each: Sotatercept 0.75 mg/kg Sotatercept 1.0 mg/kg Sotatercept + ruxolitinib combo Monotherapy: 7 of 18 (39%) evaluable patients have responded Anemia response = composite of Hb response and RBC-TI Ruxolitinib combination: 3 of 10 (30%) evaluable patients have responded Luspatercept Phase 2 company-sponsored trial initiated in MF 12

13 Habib Dable Chief Executive Officer

14 ASH 2017 Phase 2 Presentation Highlights MDS Efficacy Clinically meaningful erythroid response in over 50% of patients RBC transfusion independence achieved in over 40% of patients Duration Multiple patients out to 34 months of treatment achieving meaningful benefit Safety > 1,200 patient-months (or 100 patient-years) of Phase 2 lower-risk MDS patient experience Myelofibrosis Efficacy Anemia response in over 30% of patients Upcoming MEDALIST and BELIEVE Phase 3 trial top-line results Mid-2018 COMMANDS Phase 3 trial initiation 1H

15 Building a Potential Multi-Billion Dollar Anemia Brand ADDRESSABLE US/EU PATIENT POPULATION POTENTIAL = >115K MDS (RS+) ESA Experienced/Ineligible >40K PATIENTS TD Beta-Thalassemia >20K PATIENTS NTD Beta-Thalassemia >20K PATIENTS MDS First-line >20K PATIENTS NEW INDICATION Myelofibrosis >15K PATIENTS 15

16 ASH 2017 Results Q&A Session Habib Dable Matthew Sherman, M.D. Chris Rovaldi John Quisel Todd James, IRC Chief Executive Officer Chief Medical Officer SVP, Operations & Program Mgmt. SVP, Corporate Development VP, IR and Corp. Comm. 16

17 THANK YOU Accelerating Drug Discovery to Transform Patients Lives NASDAQ: XLRN