Senior Medical Director, Clinical Development

Reporting to: Vice President, Global Head of Clinical Development and Safety

Do you have the passion and commitment to join a team whose mission is to develop treatments that have a clear and profound impact on people with rare diseases? Does it excite you to be a part of a company developing potential new treatments that could dramatically improve the lives of people with limited to no treatment options? Named one of the Top Places to Work by The Boston Globe , X4 is an exceptional place, focused on open communication and collaboration and dedicated to each other, our partners, and the medical community.

With the April 2024 U.S. approval of our first drug XOLREMDI™ (mavorixafor), we have made great progress in advancing our patient-centric mission and in evolving the company into a fully integrated pharmaceutical company. But there is so much left to do! We are continuing to explore global opportunities for mavorixafor in its first indication, while also advancing mavorixafor for people with chronic neutropenia through an ongoing pivotal, global Phase 3 clinical trial. In addition, our research team in Vienna, Austria is focusing on new drug discovery and examining the genetic causes of chronic neutropenic disorders.

We recognize that developing innovative, new medicines is challenging, but we embrace this challenge to truly make progress for patients each day. If you feel you would be a good fit for X4, we invite you to share with us your aspirations and career goals.

At X4 you will find…

• A team that values grit and passion of innovative science.
• The opportunity to leverage your knowledge of clinical development to support our innovative science and allow you to have a major impact on our mission to support our patients.
• A collegial environment with a team-based approach, where our mission drives our values, behaviors, and creativity.
  
  
  

Your Responsibilities

• Support clinical contributions to corporate objectives, including various business development activities.
• Support scientific and clinical advisory board meetings, publications, and communications strategies.
• Foster an environment that thrives on intellectual challenges and seeks opportunities to mentor others in the organization on clinical perspectives and opportunities for drug development.
• Provide medical leadership primarily for the rare disease interests of the company with the ability to collaborate with the Medical Affairs, Clinical Operations, Research & Development, and Safety groups to advance the treatment of patients in the company’s rare disease platforms.
• Drive execution of clinical development plans with an understanding of the risks and benefits of the different strategies.
• Review and interpret trial data (safety, efficacy, PK/PD, translation biomarker, HR-QOL) with the clinical study team and communicate results to internal and external stakeholders.
• Remain current with the literature and with the competitive scientific, clinical treatment, and regulatory landscape.
• Develop relationships with Key Opinion Leaders and Principal Investigators to enable open and positive communication regarding trial design, enrollment, protocol modifications, safety, and scientific exchange, among other communications.
• Collaborate with other Clinical Development colleagues and Medical Affairs in representing X4 at global medical and advisory meetings, conferences, and advisory boards; collaborate with thought leaders to ensure the integrity of information exchange and to significantly increase the X4 fingerprint in the communities.
• Provide medical oversight and clinical guidance to support development and review of clinical protocols, Investigator Brochures, Informed Consent Forms and Clinical Study Reports.
• Collaborate cross-functionally in the preparation of the CDP, briefing books for health authority interactions, clinical protocols, investigator brochures, target product profiles, annual IND reports, severe adverse advent reports, periodic safety update reports, and clinical study reports.
• Participate as medical lead in various meetings with the EMA and FDA, or other Regulatory bodies.
• Partner with Medical Affairs colleagues in activities related to collection of natural history data, patient advocacy, and medical education.
• Provide clinical input for review of safety data, protocol deviations, clinical study reports, with responsibility for the content of various clinical components to support global regulatory submissions.
• Provide clinical input to study teams on Case Report Form design, statistical analysis plans, monitoring guidelines, Data Review Plans, and Quality Management Plans as needed, and track the emerging efficacy and safety profile of the drug.
• Work with Development Operations, Clinical Research Organizations, and CRO Medical Monitors to identify and select clinical sites, investigators, patient populations, and the recruitment strategy to meet goals in a time- and cost-effective manner and to enable quality, compliance, and patient safety at the trial, site, and patient level.
• Oversee regular review and assessment of ongoing medical data.
• Partner with Safety/PV to ensure effective and timely review of SAE reports, ASEs, targeted medical events, and cumulative and aggregate safety.
• Support discovery preclinical science to assure appropriate clinical input guides pipeline advancement and translational strategies.
• Provide medical monitor oversight to address medical questions/problems which may arise (e.g., eligibility questions) to enable quality, compliance, and patient safety and to assure the integrity of trial data.
• Partner with other Clinical Development colleagues and Medical Affairs to prepare and/or deliver abstracts, posters, and slide presentations at external Conference Symposia and manuscripts for peer reviewed journals.
• Review Investigator Initiated Research and Compassionate/Extended Use proposals upon request, based on subject matter expertise.
  
  
  

What You Will Bring

• Board-certified MD required, with 5 years clinical experience preferred.
• 7-10 years of clinical development experience in the pharmaceutical/biotechnology industry, ideally in rare and/or complex diseases.
• Clinical development experience in all phases of pipeline development with an emphasis in late-stage development.
• Track record and experience designing and implementing development plans and clinical trial strategies, methods, and processes.
• Experience with US & global regulatory discussions with the agencies and submissions of INDs, NDAs BLAs including ownership of the medical sections.
• Ability to review and understand the emerging safety and efficacy profile of the drug candidate, assess risks, and provide solutions.
• Demonstrated understanding of the complexities and recent developments in the relevant therapeutic/technical area and the ability to apply such knowledge to drug development strategies and decisions.
• Proven experience presenting clinical data, study plans and tactics clearly and accurately to internal and external stakeholders.
• Working knowledge of clinical trial biostatistics.
• Ability to work efficiently in a team-oriented and interdisciplinary setting; willing and open to contribute to the team in terms of personality and competence.
• Ability to communicate clearly, effectively, and efficiently verbally and in writing by using the appropriate communication techniques and tools, and excellent presentation skills.
• Proactive approach to identify process improvement opportunities with an ability to adapt easily to a changing working environment.
• Good judgment/analytical thinking: able to differentiate and prioritize between critical and non-critical, assess implications and risks, and initiate the appropriate steps for escalation as needed.
  
  
  

This position is based in Boston, MA, and offers a flexible hybrid work model with a minimum of 2 days/week in the office.

We encourage you to learn more about X4 Pharmaceuticals. Click on the links below!

About Us

Pipeline

X4 Pharmaceuticals is an Equal Opportunity, and Affirmative Action Employer committed to a culturally diverse workforce. All qualified candidates will receive consideration without regard to race, color, creed, religion, national origin, age, ancestry, nationality, marital, domestic partnership or civil union status, sex, gender identity or expression, sexual orientation, disability, veteran status, violence victim status or any other characteristic protected by law.