Beam Therapeutics (BEAM) Jefferies 2024 Global Healthcare Conference summary

Sickle cell program progress

• Enrollment ongoing in a pivotal sickle cell trial, with 45 patients targeted and expansion cohort underway after positive safety review of the initial three patients.

• Clinical data on a meaningful number of patients, including follow-up, expected later this year.

• Trial design aligns with industry standards, aiming for sufficient data to support a BLA filing, with 20–30 patients likely needed for regulatory submission.

• First patient dosed early this year, approaching 12 months of follow-up by year-end.

AAT in vivo gene editing program

• Open CTA in the UK, with first patient dosing planned before the end of the current month.

• Program aims to correct the disease-causing mutation in situ, restoring both liver and lung function by preserving natural gene regulation.

• Preclinical models (mouse and bespoke rat) show 0.25 mg/kg dose achieves ~30% editing, sufficient for therapeutic AAT levels.

• Clinical trial uses a single ascending dose design, with flexibility to adjust dosing and potential for redosing if needed.

• Initial clinical data expected in 2025, with the most aggressive timeline being early 2025.

Safety and platform considerations

• Transient elevation of liver function tests (LFTs) seen with LNPs is expected and not considered dose-limiting if resolved within two weeks.

• Differences in LNP formulations and patient receptor profiles can impact safety and efficacy; Beam’s LNP is similar but not identical to others.

• Preclinical and PK/PD modeling support confidence in dose selection and translatability to humans.

• Opportunity exists to redose if initial editing is insufficient, as edits are cumulative.