Stephanie M. Kapsalis, OTR
Carlisle
Summary
Clinical science leader with expertise in late-stage development and regulatory strategy. Directs cross-functional teams to execute complex submissions and facilitate critical decision-making for hematology therapies. Ensures alignment with industry standards and regulatory requirements, driving successful outcomes.
Overview
27
27
years of professional experience
Work History
Senior Director, Clinical Science
Servier Pharmaceuticals
Boston
05.2021 - Current
- Clinical Science Lead for MDS sub-study in R/R MDS and frontline MDS Phase 3 trial (Pyramidh); responsibilities include directing study and cross-functional teams in the planning and execution of clinical strategy for late-stage and frontline MDS program, helping lead study design and execution and protocol concepts for MDS program in conjunction with Medical.
- Clinical Scientist Lead for Menin Inhibitor AML program.
- Liaised with external providers, including steering committees and hematology KOLs, to enhance collaboration and align clinical strategies.
- Oversaw clinical publication strategy for IDH1 hematology projects, improving dissemination of research findings and increasing visibility within the scientific community.
Senior Director, Clinical Science
Agios Pharmaceuticals
Cambridge
10.2020 - 05.2021
- Clinical Lead on the EU MAA (EMA) and other international regulatory submissions for Tibsovo in R/R AML (Singapore HSA and mainland China NMPA submissions) in preparing all clinical content associated with submissions and drafting and final SmPC.
- Clinical Lead for two expanded IDH1 Heme trial concepts in both frontline AML Maintenance and Frontline treated MDS for potential label expansions of Tibsovo; developing and leading content and questions in two key advisory boards in 2021.
- Clinical Lead on all Tibsovo IDH1 Heme publication in addition was key reviewer for regulatory strategy efforts.
- Led clinical content development for updated Tibsovo Orphan Maintenance Report and negotiated clinical questions with EUOMO reviewers.
- Attended key regulatory meetings with CHMP to address requirements of the MAA procedure.
- Assisted the team in the content and strategy for the Oral Explanation (OE) meeting with CHMP and member states prior to the procedural vote.
- Reviewed key strategies for Late Stage Clinical Team on Tibsovo IDH1 Heme IIT, collaborating with Global Medical Affairs.
- Developed Clinical Science onboarding training module, improving new hire integration process.
- Functional Line Manager for clinical scientists in the hematology department at Agios, assisting in building out roles and responsibilities as well as training for late-stage clinical development activities.
Director, Clinical Science
Agios Pharmaceuticals
Cambridge
10.2018 - 10.2020
- Led clinical science support for five studies in hematology development program.
- Co-led Phase III team that developed and submitted breakthrough designation application.
- Presented innovative study designs, securing leadership endorsement for strategic initiatives.
- Authored and reviewed FDA information requests related to clinical data.
- Conducted training sessions for investigator meetings and organized presentations at advisory boards.
- Trained new medical directors on regulatory submissions and medical data review.
Associate Director, Clinical Science
Agios Pharmaceuticals
Cambridge
03.2015 - 09.2018
- Clinical Scientist Lead on the Ivosidenib (Tibsovo) clinical trials for AML and hematological malignancies.
- Clinical Scientist Lead on cross functional NDA team responsible for authoring and reviewing all NDA clinical module documents in eCTD (2.5, 2.7.2, 2.7.3, 2.7.4 in addition to CSRs and authoring and review of clinical narratives, FDA briefing packages, and regulatory responses to FDA.
- Clinical Scientist Lead on Label Management Team in development of Ivosidenib (Tibsovo) draft and final USPI label and Medication Guide.
- Authored and reviewed clinical sections of study specific documents including protocols, ICF, protocol amendments, and annual IB and DSUR updates.
- Led site initiation visits and investigator trainings on protocol and safety data outlined in IB, ensuring compliance and understanding among all stakeholders.
- Conducted training sessions for Medical Affairs on key safety and efficacy data of Ivosidenib (Tibsovo), facilitating informed discussions with KOLs and treating physicians for successful launch preparation.
- Approved patient eligibility during study enrollment and represented clinical scientist function in data review and study management meetings, contributing to informed decision-making and study oversight.
Clinical Scientist Independent Consultant
Agios Pharmaceuticals
Cambridge
09.2014 - 03.2015
- Led clinical science consulting for pivotal Phase I/II trial of Idhifa in IDH2 mutated relapsed or refractory AML, ensuring adherence to regulatory standards.
- Collaborated with cross-functional teams to design and execute clinical studies.
- Analyzed clinical data to evaluate safety and efficacy of investigational therapies.
- Performed data cleaning for pre-NDA activities to ensure accurate submissions.
- Conducted research on metabolic diseases to support drug development initiatives.
Clinical Scientist Consultant
Genentech
South San Francisco
02.2014 - 02.2015
- Lead Clinical Science Consultant for a pivotal Phase II clinical trial for atezolizumab (Tecentriq) in addition to supporting data review for several pivotal trials in the lung cancer clinical development program for metastatic and previously treated squamous and non-squamous NSCLC.
- Represented clinical science at phase I/II study management team meetings, contributing to study development discussions for new combinations with atezolizumab.
- Supported study MDs in pivotal phase 1/II trials by reviewing medical data and assessing protocol deviations.
Clinical Science Specialist
Hoffmann-La Roche Inc.
Nutley
01.2007 - 12.2012
- Clinical Scientist Lead for development of vemurafenib’s (Zelboraf) Clinical Development Program; responsible for collaborating and supporting Medical Director Lead in planning clinical development activities for the novel first in class BRAF inhibitor for metastatic melanoma including development of key regulatory strategy, pivotal study designs, program lifecycle planning, and overall analysis of clinical and scientific data from all aspects of the phase II/III pivotal studies.
- Co-led companion diagnostic strategy for mV600E melanoma, collaborating with Biomarker expert and MD Lead at Roche Diagnostics to integrate FDA-approved test into clinical development and marketing application for vemurafenib.
- Authored and reviewed the clinical sections of the phase II and III study CSRs, led writing activities for CSR clinical narratives in addition to clinical module writing of Module 2 and 4 (eCTD) documents required for regulatory submission.
- Prepared clinical and scientific background sections for key documents including risk-benefit assessments, ICFs, IBs, dose selection memos and safety reports in addition to supporting Regulatory Lead on authoring clinical sections of regulatory applications for Fast Track and Orphan Drug Designation for vemurafenib.
- Organized and led global advisory board meetings to gather KOL feedback on clinical development planning and study designs for vemurafenib.
- Led protocol, IB, and ICF model document writing activities for several early development phase I trials, including early development of an IGF1R inhibitor for the Ewing’s sarcoma program, anti-VEGF clinical program in solid tumors, and CD40 hematological malignancy development programs.
- Co-led dose escalation meetings with lead study MD, presenting dose escalation and safety teleconference agenda to investigators and study management team, facilitating informed dosing decisions and review of dose limiting toxicities.
Country Clinical Operations Analyst
Hoffmann-La Roche Inc.
Nutley
03.2004 - 01.2007
- Lead Clinical Operations Associate for large phase III study supporting the development program for methoxy polyethylene glycol-Epoetin Beta (Mircera) in patients with chronic kidney disease associated with renal anemia.
- Oversaw study conduct activities, ensuring seamless transitions from site initiation to closure, enhancing operational efficiency.
- Negotiated and managed clinical study budgets with sites, coordinated drug forecasting with supply depot, and selected study vendors while maintaining study trial master file.
- Planned and executed investigator meetings and site monitor training to ensure compliance and alignment with study objectives.
Local Trial Manager
Johnson and Johnson Pharmaceutical Research and Development
Raritan
08.2003 - 02.2004
- Managed two US-based Phase I and II trials for Procrit®, Topamax®, and other compounds targeting type II diabetes and obesity.
- Supported clinical scientist, safety lead, and pharmacologist in drafting Phase I and II protocols, ensuring regulatory compliance and scientific rigor.
- Ensured adherence to study-driven timelines for all assigned studies.
- Facilitated overall patient enrollment management across all participating sites.
- Developed comprehensive study budgets, managed investigational product planning, and forecasted site study contracts.
- Partnered with CROs and vendors to develop and implement IVRS systems for randomized studies, improving data accuracy and participant tracking.
- Monitored system performance to ensure investigational product delivery from depot to study sites.
- Optimized vendor relationships to facilitate smoother study execution and improve communication.
Clinical Research Assistant
R.W. Johnson Pharmaceutical Research Institute
Raritan
10.2000 - 08.2003
- Coordinated participant recruitment and informed consent processes.
- Assisted in designing and implementing clinical trial protocols.
- Coordinated planning and development of in-house phase I trials, enhancing trial readiness across various therapeutic areas.
- Prepared study sites for startup activities, ensuring seamless transitions and operational efficiency.
- Maintained accurate and organized trial documentation and databases.
Occupational Therapist
St. David’s Medical Center
Austin
08.1999 - 10.2000
- Conducted comprehensive assessments to evaluate patient needs effectively.
- Designed individualized treatment plans aligned with patient goals and clinical guidelines.
- Developed tailored treatment plans focused on individual needs and objectives.
- Implemented therapeutic interventions to enhance physical, emotional, and cognitive abilities.
- Developed and implemented therapeutic activities targeting physical strength, coordination, balance, posture, and sensory integration.
- Collaborated with multidisciplinary teams to coordinate patient care, ensuring comprehensive treatment and support.
- Educated patients and families on specific treatment plans and rehabilitation techniques to facilitate understanding and engagement.
Education
Bachelor of Science - Clinical Fieldwork in Occupational Therapy
Memorial Southwest Hospital
Houston08-1999
Bachelor of Science - Clinical Fieldwork in Occupational Therapy
Heights Hospital
Houston, TX04-1999
Bachelor of Science in Occupational Therapy (BSOT) -
Texas Woman’s University
Denton, TX01-1998
Bachelor of Fine Arts (BFA) - Painting and Studio Art
Oklahoma City University
Oklahoma City, OK01-1994
Skills
- Clinical trial design
- Regulatory submissions
- Safety and efficacy analysis
- Cross-functional leadership
- Business development
- Partnership management
- Process improvement
- Employee development and mentoring
- Medical writing
- Clinical strategy
- Stakeholder engagement
- Effective communication
- Team collaboration
Publications
- Phase 3 study of either ivosidenib monotherapy or azacitidine monotherapy in patients with IDH1-mutant myelodysplastic syndromes who are hypomethylating agent naive (PyramIDH). Santini V, Sebert M, Valcarcel D, Borate U, Weishi Y, Kapsalis SM, Simonot L, Patel PA, Garcia-Manero G. J Clin Oncol Vol. 43 (16 suppl), TPS6584(2025) 2024 Blood Advances
- PyramIDH: A phase 3 study of ivosidenib monotherapy or azacitidine monotherapy in patients with mutant isocitrate dehydrogenase 1 myelodysplastic syndromes who have not received prior hypomethylating agent therapy. Sebert M, Santini V, Platzbecker U, Valcarcel D, Borate U, Weishi Y, Kapsalis S, Simonot L, Panzica M, Patel P, Garcia-Manero G. Blood 2025; 146 (Supplement 1): 8254.
- Updated phase I substudy results of ivosidenib in patients with IDH1-mutant relapsed/refractory myelodysplastic syndrome. DiNardo C, Roboz G, Watts JM, Madanat Y, Prince GT, Baratam P, De Botton S, Stein A, Foran JM, Arellano ML, Sallman D, Marchione DM, Bai X, Patel PA, Kapsalis SM, Garcia-Manero G, Fathi A. 2023 European Hematology Association (EHA) Annual Meeting, June 8-15, Frankfurt, Germany, Poster: 724
- Ivosidenib in patients with IDH1 -mutant relapsed/refractory myelodysplastic syndrome (R/R MDS): Updated enrollment and results of a phase 1 dose-escalation and expansion substudy. Sallman D, Foran J, Watts J, Stein E, De Botton, S, Fathi A, Prince G, Stone R, Patel P, Roboz G, Arellano M, Erba H, Pigneux A, Baratam P, Thomas X, Bai X, Kapsalis S, Garcia-Manero G, Dinardo, C. (2022). Journal of Clinical Oncology. 40. 7053-7053. 10.1200/JCO.2022.40.16_suppl.7053.
- Population pharmacokinetic and exposure-response analyses of ivosidenib in patients with IDH1-mutant advanced hematologic malignancies. Jiang X, Wada R, Poland B, Kleijn HJ, Fan B, Liu G, Liu H, Kapsalis S, Yang H, Le K. Clin Transl Sci. 2021 May;14(3):942-953.
- Outcomes of patients with IDH1-mutant relapsed or refractory acute myeloid leukemia receiving ivosidenib who proceeded to hematopoietic stem cell transplant. DiNardo CD, Stein EM, Pigneux A, Altman JK, Collins R, Erba HP, Watts JM, Uy GL, Winkler T, Wang H, Choe S, Liu H, Wu B, Kapsalis SM, Roboz GJ, de Botton S. Leukemia. 2021 Mar 26:10.1038/s41375-021-01229-x.
- Ivosidenib improves overall survival relative to standard therapies in relapsed or refractory mutant IDH1 AML: Results from matched comparisons to historical controls. Paschka P, Dombret H, Thomas X, Recher C, Chantepie S, Montesinos P, Acuña Cruz E, Vyas P, Kreuzer K-A, Heuser M, Metzeler KH, Dennis M, Quesnel B, Hunault-Berger M, Mohty M, Pigneux A, de Botton S, Weber D, Döhner K, Milkovich G, Reitan J, MacDonald SC, Casso D, Storm M, Liu H, Kapsalis SM, Attar EC, Winkler T, Döhner H. Presented (Oral Presentation) at the 62nd American Society of Hematology Annual Meeting and Exposition, December 6, 2020 (Virtual Meeting Only)
- Ivosidenib (IVO) prior to hematopoietic cell transplant for patients with IDH1-mutant relapsed or refractory acute myeloid leukemia (R/R AML). DiNardo CD, Stein EM, Pigneux A, Altman JK, Collins R, Erba HP, Watts JM, Uy GL, Kapsalis SM, Liu H, Winkler T, Roboz GJ, de Botton S. Presented (Poster Presentation) at the European Hematology Association - 25th Congress, June 11, 2020, (Virtual Meeting Only)
- Ivosidenib (IVO) prior to hematopoietic cell transplant for patients with IDH1-mutant relapsed or refractory acute myeloid leukemia (R/R AML). DiNardo CD, Stein E, Pigneux A, Altman JK, Collins R, Erba HP, Watts JM, Uy GL, Kapsalis SM, Liu H, Winkler T, Roboz GJ, de Botton S. Presented (Poster Presentation) at the American Society of Clinical Oncology - 56th Annual Meeting, May 13, 2020
- Ivosidenib induces deep durable remissions in patients with newly diagnosed IDH1-mutant acute myeloid leukemia. Roboz GJ, DiNardo CD, Stein EM, de Botton S, Mims AS, Prince GT, Altman JK, Arellano ML, Donnellan W, Erba HP, Mannis GN, Pollyea DA, Stein AS, Uy GL, Watts JM, Fathi AT, Kantarjian HM, Tallman MS, Choe S, Dai D, Fan B, Wang H, Zhang V, Yen KE, Kapsalis SM, Hickman D, Liu H, Agresta SV, Wu B, Attar EC, Stone RM. Blood. 2020 Feb 13;135(7):463-471.
- Ivosidenib (AG-120) in IDH1-mutant newly diagnosed acute myeloid leukemia: Updated results from a phase 1 study. DiNardo CD, Roboz GJ, Stein EM, de Botton S, Mims AS, Prince GT, Altman JK, Arellano ML, Donnellan W, Erba HP, Mannis GN, Pollyea DA, Stein AS, Uy GL, Watts JM, Fathi AT, Kantarjian HM, Tallman MS, Choe S, Dai D, Fan B, Wang H, Zhang V, Yen KE, Kapsalis SM, Hickman D, Liu H, Agresta S, Wu B, Attar EC, Stone RM. Presented at the Society of Hematologic Oncology 7th Annual Meeting; 11–14 Sep 2019; Houston, TX, USA. Poster AML-208.
- Ivosidenib (AG-120) induces durable remissions and transfusion independence in patients with IDH1-mutant relapsed/refractory myelodysplastic syndrome in a phase 1 dose escalation and expansion study. Foran JM, DiNardo CD, Watts JM, Stein EM, de Botton S, Fathi AT, Prince GT, Stein AS, Stone RM, Patel PA, Tallman MS, Choe S, Wang H, Zhang V, Dai D, Fan B, Yen KE, Kapsalis SM, Hickman D, Agresta SV, Liu H, Wu B, Attar EC, Kantarjian HM. Presented at the 24th Congress of the European Hematology Association; 13-16 Jun 2019; Amsterdam, the Netherlands. Poster PS1337.
- Ivosidenib (AG-120) induces durable remissions and transfusion independence in patients with IDH1-mutant newly diagnosed AML: Updated results from a phase 1 dose escalation and expansion study. Roboz GJ, DiNardo CD, Stein EM, de Botton S, Mims AS, Prince GT, Altman JK, Arellano ML, Donnellan W, Erba HP, Mannis GN, Pollyea DA, Stein AS, Uy GL, Watts JM, Fathi AT, Kantarjian HM, Tallman MS, Choe S, Dai D, Fan B, Wang H, Zhang V, Yen KE, Kapsalis SM, Hickman D, Liu H, Agresta S, Wu B, Attar EC, Stone RM. Presented at the 24th Congress of the European Hematology Association; 13-16 Jun 2019; Amsterdam, the Netherlands. Poster PS1025.
- Safety and clinical activity of mutant IDH1 inhibitor ivosidenib (AG-120) in combination with azacitidine for newly diagnosed acute myeloid leukemia (ND-AML) DiNardo CD, Stein AS, Stein EM, Fathi AT, Frankfurt O, Schuh AC, Döhner H, Martinelli G, Patel PA, Raffoux E, Tan P, Zeidan A, de Botton S, Kantarjian HM, Stone RM, Lam D, Wang X, Gong J, Kapsalis SM, Denice Hickman, Zhang V, Winkler T, Wu B, Vyas P. Presented at ACUTE LEUKEMIAS XVII Biology and Treatment Strategies, 24–27 February 2019, Munich, Germany Poster 40a
- Ivosidenib (AG-120) in mutant IDH1 relapsed/refractory acute myeloid leukemia: Results of a phase 1 study. DiNardo CD, Stein EM, de Botton S, Roboz GJ, Altman JK, Mims AS, Swords R, Collins RH, Mannis GN, Pollyea DA, Donnellan W, Fathi AT, Pigneux A, Erba HP, Prince GT, Stein AS, Uy GL, Foran JM, Traer E, Stuart RK, Arellano ML, Slack JL, Sekeres MA, Willekens C, Choe S, Wang H, Zhang V, Yen KE, Kapsalis SM, Yang H, Dai D, Fan B, Goldwasser M, Liu H, Agresta S, Wu B, Attar EC, Tallman MS, Stone RM, Kantarjian HM. Clin Lymphoma Myeloma Leuk. 2018;18 (suppl 1):S204–S205, AML-221.
- Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory AML. DiNardo CD, Stein EM, de Botton S, Roboz GJ, Altman JK, Mims AS, Swords R, Collins RH, Mannis GN, Pollyea DA, Donnellan W, Fathi AT, Pigneux A, Erba HP, Prince GT, Stein AS, Uy GL, Foran JM, Traer E, Stuart RK, Arellano ML, Slack JL, Sekeres MA, Willekens C, Choe S, Wang H, Zhang V, Yen KE, Kapsalis SM, Yang H, Dai D, Fan B, Goldwasser M, Liu H, Agresta S, Wu B, Attar EC, Tallman MS, Stone RM, Kantarjian HM. N Engl J Med. 2018; 378(25): 2386-2398.
- Ivosidenib (AG-120) induced durable remissions and transfusion independence in patients with IDH1-mutant relapsed or refractory myelodysplastic syndrome: results from a phase 1 dose escalation and expansion study. DiNardo CD, Watts JM, Stein EM, de Botton S, Fathi AT, Prince GT, Stein AS, Foran JM, Stone RM, Patel PA, Tallman MS, Choe S, Wang H, Zhang V, Dai D, Fan B, Yen KE, Kapsalis SM, Hickman D, Agresta SV, Liu H, Wu B, Attar EC, Kantarjian HM. Presented at the 60th American Society of Hematology (ASH) Annual Meeting, December 1–4, 2018, San Diego, CA, USA
- Ivosidenib (AG-120) induces durable remissions and transfusion independence in patients with IDH1-mutant untreated AML: Results from a phase 1 dose escalation and expansion study. Roboz GJ, DiNardo CD, Stein EM, de Botton S, Mims AS, Prince GT, Altman JK, Arellano ML, Donnellan W, Erba HP, Mannis GN, Pollyea DA, Stein AS, Uy GL, Watts JM, Fathi AT, Kantarjian HM, Tallman MS, Choe S, Dai D, Fan B, Wang H, Zhang V, Yen KE, Kapsalis SM,Hickman D, Liu H, Agresta SV, Wu B, Attar EC, Stone RM. Presented at the 60th American Society of Hematology (ASH) Annual Meeting, December 1–4, 2018, San Diego, CA, USA
- Ivosidenib (AG-120) in mutant IDH1 relapsed/refractory acute myeloid leukemia: Results of a phase 1 study. DiNardo CD, Stein EM, de Botton S, Roboz GJ, Altman JK, Mims AS, Swords R, Collins RH, Mannis GN, Pollyea DA, Donnellan W, Fathi AT, Pigneux A, Erba HP, Prince GT, Stein A, Uy GL, Foran JM, Traer E, Stuart RK, Arellano ML, Sekeres MA, Willekens C, Choe S, Wang H, Zhang V, Yen KE, Kapsalis SM, Hickman D, Yang H, Dai D, Fan B, Goldwasser M, Liu H, Agresta S, Wu B, Attar EC, Tallman MS, Stone RM, Kantajian HM. Presented at the Society of Hematologic Oncology 6th Annual Meeting; 12–15 Sep 2018; Houston, TX, USA. Poster AML-221.
- Ivosidenib (AG-120) in Mutant IDH1 AML and Advanced Hematologic Malignancies: Results of a Phase 1 Dose Escalation and Expansion Study. DiNardo CD, De Botton S, Stein EM, Roboz GJ, Mims AS, Pollyea DA, Swords R, Altman JK, Collins RH, Mannis GN, Uy GL, Donnellan W, Pigneux A, Fathi AT, Stein AS, Erba HP, Prince GT, Foran JM, Traer E, Stuart RK, Arellano ML, Slack JL, Sekeres MA, Yen K, Kapsalis SM, Liu H, Goldwasser M, Agresta S, Attar EC, Tallman MS, Stone RM, Kantarjian HM. Blood. 2017;130 (suppl 1):A725.
Timeline
Senior Director, Clinical Science
Servier Pharmaceuticals
05.2021 - Current
Senior Director, Clinical Science
Agios Pharmaceuticals
10.2020 - 05.2021
Director, Clinical Science
Agios Pharmaceuticals
10.2018 - 10.2020
Associate Director, Clinical Science
Agios Pharmaceuticals
03.2015 - 09.2018
Clinical Scientist Independent Consultant
Agios Pharmaceuticals
09.2014 - 03.2015
Clinical Scientist Consultant
Genentech
02.2014 - 02.2015
Clinical Science Specialist
Hoffmann-La Roche Inc.
01.2007 - 12.2012
Country Clinical Operations Analyst
Hoffmann-La Roche Inc.
03.2004 - 01.2007
Local Trial Manager
Johnson and Johnson Pharmaceutical Research and Development
08.2003 - 02.2004
Clinical Research Assistant
R.W. Johnson Pharmaceutical Research Institute
10.2000 - 08.2003
Occupational Therapist
St. David’s Medical Center
08.1999 - 10.2000
Bachelor of Science - Clinical Fieldwork in Occupational Therapy
Memorial Southwest Hospital
Bachelor of Science - Clinical Fieldwork in Occupational Therapy
Heights Hospital
Bachelor of Science in Occupational Therapy (BSOT) -
Texas Woman’s University
Bachelor of Fine Arts (BFA) - Painting and Studio Art
Oklahoma City University