BridgeBio Pharma
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BridgeBio Pharma (BBIO) investor relations material

BridgeBio Pharma 44th Annual J.P. Morgan Healthcare Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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44th Annual J.P. Morgan Healthcare Conference summary12 Jan, 2026

Strategic vision and leadership

  • Focus on becoming a fully integrated biopharma company, advancing from discovery to commercialization.

  • Deep expertise in CNS ion channel drug development underpins the portfolio strategy.

  • Major emphasis on azetukalner, with expansion into psychiatry and early-stage pipeline development.

  • Recent hires in commercial and financial leadership to support forward integration.

  • Commitment to building a world-class team with deep epilepsy experience for launch success.

Clinical pipeline and trial updates

  • Six ongoing phase III trials for azetukalner: three in epilepsy, three in neuropsychiatry.

  • X-TOLE2 phase III in focal onset seizures completed randomization; top-line data expected March 2024.

  • X-TOLE3 includes Japanese subjects, streamlining global regulatory strategy.

  • Phase III trial in primary generalized tonic-clonic seizures running in parallel.

  • Early-stage pipeline includes Nav1.7 and Kv7 programs, with Nav1.7 showing promising phase I data.

Azetukalner efficacy and safety data

  • Phase II-B X-TOLE study showed best-in-class seizure reduction in a highly refractory population.

  • Rapid onset of efficacy observed, with significant seizure reduction within the first week.

  • Long-term open-label extension data show over 90% reduction in seizure burden and sustained seizure freedom for many.

  • Safety profile consistent with other CNS-active drugs; no new adverse events in long-term use.

  • High physician anticipation due to novel mechanism, ease of use, and lack of titration or drug-drug interactions.

Depleter program's clinical differentiation
Encaleret's CHP market expansion strategy
PORT-77's best-in-disease profile validation
Attruby's $1B+ 2026 revenue target. How?
LGMD2I & ADH1 "therapeutic cure" basis
EPP PORT-77's best-in-class potential. Why?
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Frequently asked questions

BridgeBio Pharma, Inc. engages in the discovery, development, and delivery of various medicines for genetic diseases. The company's lead candidates for the treatment of Fabry disease comprise a pipeline of recombinant enzyme-replacement therapies comprising elaprase and lysosomal-phospholipid-glycoprotein (PLG) analogs. It focuses on developing therapeutics for treating Fabry disease, a rare genetic disorder characterized by progressive loss of kidney function and the buildup of fatty substances that damage blood vessels and organs. BridgeBio Pharma, Inc.i s based in Waltham, Massachusetts.

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