Senior Director – Biostatistics, Neurology

This is a fully remote position, open to applicants in California.

📋 Description

• Provide statistical leadership for ASO programs (Phase 1/2 to PoC readiness): Act as the lead statistician for GondolaBio's ASO assets, overseeing statistical strategy from initial first-in-human trials through proof-of-concept.

• Create and justify fit-for-purpose statistical analysis plans (SAPs) that consider small sample sizes, placebo and open-label designs, as well as significant inter-patient variability.

• Develop a seizure endpoint strategy: Formulate robust statistical methods for analyzing seizure frequency and responder rates in Developmental and Epileptic Encephalopathy populations, where within-patient variability, seizure clustering, and non-normality necessitate non-standard techniques.

• Collaborate with clinical teams to establish scientifically valid and regulatorily acceptable primary and secondary endpoints.

• Implement trial designs adapted for rare diseases: Utilize specialized methods suitable for small N studies, including Bayesian frameworks, adaptive designs, n-of-1 elements, and natural history benchmarking.

• Proactively identify and address risks associated with high variability, missing data, and limited statistical power, clearly communicating trade-offs to program leadership.

• Provide regulatory statistical support: Prepare and contribute to statistical sections of INDs, briefing documents, and FDA/EMA meeting packages. Assist with pre-IND and Type B/C meetings, and address agency inquiries regarding statistical methodologies.

• Conduct biomarker and exploratory analyses: Work with Pharmacology, Translational, and Clinical teams to develop analysis plans for pharmacodynamics biomarkers, target engagement endpoints, and patient stratification. Implement appropriate multiplicity controls and exploratory frameworks to maintain interpretability.

• Engage in cross-functional collaboration: Work closely with Clinical Development, Regulatory, Clinical Operations, and external CROs/vendors to ensure statistical considerations are incorporated early in protocol development, CRF design, data management, and study execution. Represent the Statistics team in cross-functional program team meetings.

⛳️ Requirements

• M.S. or Ph.D. in Biostatistics, Statistics, or a related field.

• 8+ years of experience in the pharmaceutical/biotech industry, with significant exposure to early-phase (Phase 1/2) rare disease studies.

• Proven experience in designing and analyzing seizure trials (e.g., DEE, TSC, Dravet, LGS) or other high-variability neurological endpoints.

• Extensive knowledge of statistical challenges unique to rare diseases: small sample sizes, open-label designs, natural history controls, and high inter-patient variability.

• Proficient in SAS and/or R; capable of independently programming and reviewing analysis datasets and outputs.

• Experience in authoring and defending SAPs, study reports, and regulatory statistical documents.

• Comfortable operating in dynamic, fast-paced biotech environments with a high level of autonomy and accountability.

• Nice-to-Haves

• Familiarity with Bayesian or adaptive design methodologies in trials for rare or neurological diseases.

• Knowledge of PRO/ClinRO endpoint development and validation in pediatric or rare disease populations.

• Experience with responder analyses, seizure diary data, and mixed-effects models for repeated measures (MMRM) in epilepsy studies.

• Exposure to seamless Phase 1/2 designs or platform trials.

• Direct experience interfacing with FDA or EMA regarding statistical methodologies for rare diseases.

🏝️ Benefits

• Patient Days, where we are privileged to hear directly from individuals living with the conditions we aim to influence throughout the year and learn how we can enhance our efforts.

• A culture driven by our values: prioritize patients, think independently, maintain radical transparency, value every minute, and let the science guide us.

• An unwavering commitment to always prioritizing patients.

• A decentralized model that empowers our program teams to focus on advancing science and assisting patients. Our affiliate structure is designed to reduce bureaucracy and place decision-making power in the hands of those closest to the science.

• An environment where you take ownership of the vision – both for your program and your own career trajectory.

• A collaborative, fast-paced, data-driven atmosphere that encourages us to inspire one another to consistently perform at our best.

• Access to learning and development resources to help you reach your peak professional potential.

• A comprehensive and competitive compensation and benefits package (Base, Performance Bonus, Equity, health, welfare & retirement programs).

• Flexible Paid Time Off (PTO).

• Opportunities for rapid career advancement for high performers.

• Potential to work on multiple GondolaBio Pharma programs across various therapeutic areas over time.

• Commitment to Diversity, Equity & Inclusion.