Company Overview 

Fulcrum Therapeutics, Inc. (“Fulcrum”) [NASDAQ: FULC] is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s lead program in clinical development is Pociredir, a small molecule designed to increase the expression of fetal hemoglobin for the treatment of sickle cell disease (SCD).

Description

Reporting to VP of Biometrics, the Director of biostatistics within the Biometrics Department is a hands-on role and is expected to be the statistics subject matter expert for clinical development programs and studies, including oversight of statistical activities in outsourced clinical trials, while ensuring adherence to all regulatory requirements. This individual is expected to have an in-depth understanding of innovative study designs and statistical methodologies, including estimand strategies, endpoint selection, missing data and multiplicity handling. In addition, this individual will have the capacity to understand the relevant multi-disciplinary knowledge and interact effectively within the biometrics department and with clinical development, clinical operations, pharmacovigilance, medical writing, and regulatory affairs.

Responsibilities

• Function as a lead statistician for internal studies.
• Contribute to enterprise strategy through proactive cross-functional collaborations and influence through decision making.
• Provide statistical input to strategic planning, study design, protocol development, sample size calculation, CRF, Results Interpretation, clinical study report, and address statistical questions from regulatory agencies.
• Conduct ad-hoc analyses as needed.
• Provide oversight of biostatistics activities in outsourced clinical trials.
• Ensure high quality, timely delivery of statistical analysis plans and statistical outputs.
• Execute quality assurance procedures on work produced by others, ensuring statistical analysis is being done according to specifications.
• Provide support for publications as needed.
• Author or review statistical SOPs and Working Instructions.

Qualifications

• A Ph.D. (or equivalent degree) in Biostatistics or Statistics with a minimum of 8 years of relevant clinical biostatistics experience in pharma, biotech or CRO industry -OR- a Master's degree and a minimum of 10 years of relevant industry experience is required.
• Comprehensive knowledge of innovative study design and statistical methodologies.
• Extensive knowledge of relevant clinical and statistical regulatory requirements (e.g., GCP, ICH, CDISC, etc.).
• Regulatory interaction and submission experience is highly preferred.
• Experience in oversight of biostatistics activities in outsourced clinical trials is highly preferred.
• Proficiency in statistical software (e.g., SAS, R) and simulation.
• Excellent verbal and written communication skills.
• Ability to work collaboratively in a multidisciplinary team environment.

Company Overview 

Fulcrum Therapeutics, Inc. (“Fulcrum”) [NASDAQ: FULC] is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases. Fulcrum is deeply committed to bringing hope and new options to patients suffering from rare hematologic disorders and is developing a pipeline of clinical and preclinical assets. Fulcrum’s lead clinical asset is pociredir, a first-in-class small molecule designed to increase the expression of fetal hemoglobin for the treatment of sickle cell disease and other hemoglobinopathies. We are working to expand our portfolio of clinical-stage assets and seek a driven and strategic leader to head our Pharmaceutical Development function.

Description

Reporting to the Head of Technical Operations, the Director of Pharmaceutical Development will lead all drug product development activities including technical, managerial, and budgetary aspects. This includes formulation development, manufacture of clinical trial material, process optimization, scale-up, and validation. The successful candidate will lead, plan and manage the technical and business-related activities associated with drug product development and manufacturing at contract development and manufacturing vendors for all dosage form programs.

Key Responsibilities

• Lead all strategic and tactical aspects of drug product development at Fulcrum
• Design and execute pharmaceutical product development plans for all development programs in accordance with corporate goals
• Collaborate with colleagues in Drug Substance, Analytical, ClinOps, QA, and Reg CMC to ensure plans are cross-functional, phase-appropriate, and on time
• Lead pharmaceutical development for the pociredir program, including CTM for pivotal clinical studies, pre-validation studies, validation plans, and commercial launch supply
• Lead and manage product development, technology transfer, and clinical manufacturing activities at contract manufacturing organizations (CMOs), assuring compliance with Regulatory and Quality requirements
• Lead pediatric formulation development efforts, as required
• Review and approve a broad spectrum of documents (MBRs, Specifications, Protocols, Reports, etc.) at CMOs
• Generate, review and approve documents related to Drug Product (formulation, process development, manufacturing, etc.) for Regulatory submissions (IND/IMPD and NDA/MAA)

Qualifications

• D with 10 years or MS with equivalent years of experience. Professional background in Pharmaceutical Sciences (preferred) or Chemical Engineering
• Proven track record and leadership in phase-appropriate drug development through pivotal studies (required) and approval (preferred)
• SME in material science aspects of dosage forms and drug delivery, with emphasis on solid oral dosage forms; strong knowledge in Dissolution, Biopharmaceutics and PK
• Well versed in Regulatory CMC and ICH guidance, including exposure to FDA interactions (e.g. via EOP2 or preNDA meetings)
• Strong written and oral communication skills; highly effective collaborator with “customers” in ClinOps and Commercial
• Ability to hire and recruit staff, including key contractors and consultants
• Positive work presence; responds to stress and ambiguity with humor

Company Overview 

Fulcrum Therapeutics, Inc. (“Fulcrum”) [NASDAQ: FULC] is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases. Fulcrum is deeply committed to bringing hope and new options to patients suffering from rare hematologic disorders and is developing a pipeline of clinical and preclinical assets. Fulcrum’s lead clinical asset is pociredir, a first-in-class small molecule designed to increase the expression of fetal hemoglobin for the treatment of sickle cell disease and other hemoglobinopathies. We are working to expand our portfolio of clinical-stage assets and seek a driven and strategic leader to head our Analytical Chemistry function.

Description

Reporting to the Head of Technical Operations, the Director of Analytical Chemistry is responsible for the design and execution of the control strategies for the manufacture of drug substances and drug products for Fulcrum developmental programs.  In this position, the successful candidate will provide technical oversight of all Quality Control (QC) activities at DS and DP contract manufacturers. Additional responsibilities include collaborating with pharmaceutics on pre-formulation activities, collaborating with QA on OOS and OOT investigations and CMC risk management, and helping Reg-CMC author relevant sections of regulatory filings.

Key Responsibilities

• Provide technical leadership to CMOs and CROs for drug substance and drug product process development, including cGMP method development, validation, method transfer, method remediation, data analysis and reporting to ensure compliance with GCP, GLP and cGMP standards
• Collaborate with colleagues in Drug Substance, Drug Product, QA, and Reg CMC to ensure plans are cross-functional, phase-appropriate, and on time
• Responsible for design and management of stability studies, including shelf-life and retest date management, to ensure seamless clinical supply
• Lead and manage reference standard, in-process control, and specification strategies across the pipeline
• Review and approve a broad spectrum of documents (Test Methods, Qualification / Validation Protocols, Specifications, Reports, etc.) at CMOs
• Generate, review and approve documents related to the control of DS and DP for Regulatory submissions (IND/IMPD and NDA/MAA)
• Visit contract manufacturing sites both domestically and internationally to support key production milestones and build excellent business relationships

Qualifications

• MS with 15+ or PhD with 10+ years of relevant experience in the development and application of analytical control strategies for both drug substance and drug product
• Expert in the development of test methods that employ advanced analytical techniques for Quality Control (UPLC, GC, MS, PSD, etc.)
• Proficient at troubleshooting analytical methods at CMOs
• Proven track record and leadership in phase-appropriate analytical control strategies from early development through pivotal studies
• Well versed in Regulatory CMC and ICH guidance
• Highly effective collaborator with “customers” in DS and DP, QA and RA
• Positive work presence; responds to stress and ambiguity with humor