Overview

The ED/VP of Medical Science serves as the Global Medical Leader, overseeing compounds targeting neurodevelopmental (FXS, Jordan’s, etc) and CNS diseases and assuming the crucial responsibility of Medical Monitor for clinical studies. Responsibilities include providing comprehensive medical and scientific guidance, ensuring alignment of company clinical programs with the overarching Global Clinical Development Plan for assigned assets. Extensive interactions with Regulatory Authorities, including the FDA, as well as engagement with clinical experts, investigators and patient organizations, are integral to the role. Collaboration with functions such as Project Management, Regulatory Affairs, biometrics and Clinical Operations is essential to drive seamless integration of development plans, medical strategy, and operational execution. This role is both tactical and strategic, requiring hands-on involvement while shaping long-term vision and strategy.

Responsibilities

• Leading the design and execution of clinical trials specific to neurodevelopmental (FXS, Jordan’s, etc) and CNS diseases, ensuring adherence to regulatory standards and ethical guidelines.
• Developing comprehensive medical strategies tailored to address the unique challenges and considerations of rare neurodevelopmental diseases, including patient recruitment, endpoint selection, and data analysis.
• Overseeing safety monitoring activities, including the review and analysis of adverse events, safety signals, and risk management strategies specific to these diseases.
• Providing medical oversight throughout all phases of clinical development, including protocol development, site selection, patient monitoring, and data interpretation.
• Coordinating the preparation of clinical study reports, investigator brochures, and other regulatory documents relevant to clinical trials in this disease space.
• Overseeing the medical and clinical aspects of New Drug Applications and regulatory interactions globally.
• Leading the dissemination of study results through publications in peer-reviewed journals, presentations at scientific conferences, and engagement with medical and patient advocacy communities.
• Collaborating with cross-functional teams including Clinical Operations, Regulatory Affairs, Pharmacovigilance, and Medical Affairs to ensure alignment of medical objectives with broader organizational goals.
• Coordinating the preparation of clinical study reports, investigator brochures, and other regulatory documents relevant to rare disease clinical trials.
• Establishing and maintaining relationships with Key Opinion Leaders (KOLs), external experts in the field of rare diseases to gather insights, facilitate investigator-initiated studies, and advance scientific knowledge.
• Staying abreast of advancements in the field of neurodevelopmental diseases and other CNS conditions including cognitive disorders, through ongoing education, participation in scientific meetings, and engagement with professional networks to ensure the application of best practices and innovative approaches in clinical development.
• Developing and delivering educational initiatives for internal stakeholders, investigators, and patient communities to raise awareness and understanding of neurodevelopmental diseases and ongoing clinical research efforts.
• Evaluate external assets for potential in-licensing or acquisition, considering medical value, strategic alignment, and therapeutic relevance. Collaborate with business development teams to provide medical insights and support due diligence assessments.
• Conduct comprehensive evaluations of internal pipeline products, analyzing clinical data, market potential, and competitive landscape to inform strategic decisions. Provide recommendations for resource allocation, prioritization, and portfolio management to maximize asset value and strategic alignment.
• Other duties as assigned.

Minimum Job Requirements

Qualifications

• Medical degree (MD or equivalent) with board certification preferred.
• Minimum of 12 years of experience in clinical practice/clinical research with at least 5 years in pharmaceutical industry required; 5 years of continuous clinical development experience in neurodevelopmental diseases, serving as a medical expert/medical monitor for Phase 2 and/or Phase 3 studies, or as a clinical leader for a product in Phase 2 and/or Phase 3.
• Substantial experience in clinical development within the pharmaceutical or biotechnology industry, with a focus on neurodevelopmental diseases.
• Proven track record of successful leadership in designing and executing clinical trials for neurodevelopmental diseases, resulting in regulatory submissions and approvals.
• Strong understanding of regulat