New PKU Program (BEAM-304) Initiated and Rapidly Advanced
Beam announced BEAM-304, a liver-targeted base editing program for phenylketonuria (PKU). The company identified two development candidates (including R408W) that together could treat nearly half (~50%) of PKU patients in the U.S. Initial IND-enabling work was advanced in under 2 years with an IND planned to be filed in 2026.
Strong Preclinical Efficacy for BEAM-304
Preclinical data in mouse models showed rapid and robust plasma phenylalanine (Phe) reductions after a single dose (0.3 mg/kg). In animal studies Phe was reduced below the therapeutic threshold by day 7 (and in a separate humanized model normalized by 48 hours), with a predictable dose–response between on-target liver editing and Phe reduction.
Regulatory Engagement and Innovative Trial Design Support
Beam reported productive pre-IND interactions with FDA and substantive discussions supporting a platform/umbrella approach (multiple mutation-specific editors in a single IND) and the ability to add new variants via rapid IND amendments using in vitro data, potentially enabling faster addition of mutation-specific editors.
LNP Delivery Expertise and Manufacturing Capability
Beam emphasized internally developed LNP delivery and optimization expertise, including proprietary ionizable lipids and internal GMP manufacturing capacity in North Carolina, enabling outpatient IV dosing, titratability and redoseability for liver-directed programs.
Strategic Non-Dilutive Financing to Strengthen Balance Sheet
Beam entered a long-term financing facility with Sixth Street providing up to $500 million: $100M funded at close, up to $300M tied to milestones, and an additional $100M subject to agreement. Repayment of principal is due in early 2033.
Healthy Cash Balance and Extended Runway
Beam ended 2025 with $1.25 billion in cash, cash equivalents and marketable securities. With the anticipated minimum draw of $200M from the Sixth Street facility, management expects runway to extend into mid-2029 to support pipeline execution and anticipated commercialization activities.
Clear Development Milestones and Pipeline Momentum
Near-term expectations include BEAM-302 pivotal planning update (alpha-1 antitrypsin deficiency) this quarter, potential risto-cel BLA submission as early as year-end, IND filing for BEAM-304 in 2026, initial BEAM-301 data in GSDIa, completion of BEAM-103 healthy volunteer study, and continued in vivo HSC editing work.
Clinical Need and Market Opportunity Quantified
PKU: ~20,000 individuals in the U.S.; regulatory precedent accepts blood Phe reduction as an endpoint for full approval in U.S./EU. Clinical data cited: ~30% of R408W patients had lifetime average Phe above recommended maximum; only ~25% of adult PKU patients maintain Phe <360 µM; enzyme replacement therapy reaches target in ~60% of patients after 1–2 years.