Financial Position and Runway - Beam had $1.25 billion in cash as of December 31, 2025, inclusive of $255.1 million proceeds from the Orbital acquisition[16] - The company's operating runway is expected into 2029, funding through risto-cel launch and execution of BEAM-302 pivotal development plan[16,60] Program Advancements and Regulatory Alignment - Beam achieved first human proof of concept for in vivo gene correction with BEAM-302 in 2025[17] - Alignment was reached with the FDA on a potential accelerated approval pathway for BEAM-302 in AATD, with primary endpoint expected to be based on AAT biomarkers evaluated over 12 months[27] - The company plans to submit the risto-cel BLA package as early as YE 2026[45,60] Clinical Data and Trial Updates - BEAM-302 Phase 1/2 trial has dosed >25 patients across cohorts to date, with updated clinical data expected by the end of first quarter 2026[25,60] - Initial clinical data for BEAM-301 is planned to be reported by YE 2026[31,60] - BEACON results for risto-cel in SCD show HbF levels >60% and HbS levels <40%, comparable to sickle trait, with resolution of anemia and normalized or improved markers of hemolysis and oxygen delivery[37] - At Day 28, BEAM-302 showed mean total AAT in circulation at 12.4µM, mean % change in Z-AAT at -78%, and corrected M-AAT reached >90% of total circulating AAT[24] Strategic Initiatives - Beam completed a $500 million financing and runway extension into 2029[17] - The company intends to pursue an accelerated approval pathway for BEAM-302 based on FDA feedback to date[26]

Zorevunersen for Dravet Syndrome - Stoke Therapeutics is advancing zorevunersen as a potential disease-modifying therapy for Dravet Syndrome[9] - Phase 3 study (EMPEROR) enrollment is expected to complete in Q2 2026 with data readout in mid-2027 to support NDA[12, 38] - Approximately 330 patients have been identified globally for the EMPEROR study, with ~60 in screening and ~60 randomized[12, 39] - Data from ongoing OLE studies show continuing improvements in cognition and behavior over 36 months[24] - Zorevunersen demonstrated significant reduction in major motor seizure frequency at 6 months compared to natural history data, with an 8248% reduction compared to a 2043% reduction in the natural history group (P=00181)[26] - In OLE studies, 86% of patients experienced CSF protein elevation, classified as a TEAE in 45%[31] STK-002 for Autosomal Dominant Optic Atrophy (ADOA) - A Phase 1 study of STK-002 for ADOA has been initiated in the UK and Europe[11] Financial Status and Collaboration - Stoke Therapeutics had approximately $3917 million in cash, cash equivalents, and marketable securities as of December 31, 2025, with funding anticipated into 2028[12] - A strategic collaboration with Biogen exists for the development and commercialization of zorevunersen, with Biogen holding exclusive commercialization rights outside of the US, Canada, and Mexico[48] - The Biogen collaboration includes $165 million upfront, shared development costs, and potential milestone payments of $385 million[49] Market Opportunity - There are approximately 38,000 patients with Dravet syndrome across 7 major markets[40, 44]

Financial Performance and Growth - Harmony Biosciences anticipates net revenue between $1 billion and $1.04 billion for 2026[16] - The company's revenue has grown consistently year-over-year, with $714.7 million in 2024, $868 million in 2025, and projected growth into 2026[52] - WAKIX net revenue has shown significant growth from $160 million in 2020 to over $1 billion projected for 2026[15] Pitolisant Franchise Strategy - The company is focused on growing the WAKIX franchise, extending it with Pitolisant GR, and expanding it with Pitolisant HD[8] - Pitolisant GR has the potential to extend the pitolisant franchise with utility patents filed to 2044[9] - Pitolisant HD has the potential to expand the pitolisant patient base into new indications[9] Pipeline Development - A Phase 1 clinical PK data for BP1.15205, a potential best-in-class Orexin 2 Receptor (OX2R) Agonist, is anticipated in mid-2026[42] - Topline data from ongoing global Phase 3 trials for EPX-100 in Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS) are anticipated in 1H 2027[45, 47] - The company submitted an NDA for Pitolisant GR in Q2 2026, with an anticipated PDUFA date in Q1 2027[31] Market Opportunity - There are approximately 170,000 people living with narcolepsy in the U S, with over 50% undiagnosed[22]

Business Overview - The company has nine medicines on the market driving revenue growth[4] - Two co-owned medicines are TIVDAK® (tisotumab vedotin) and EPKINLY® /TEPKINLY® (epcoritamab)[4] - The company is targeting <3x gross leverage by 2027E[6] Pipeline and Potential Launches - Three late-stage assets are positioned for potential 2027 launches[4] - Epkinly® (Lymphoma) has a peak annual sales potential of >$3 Billion[8] - Rina-S® (Gyn-Onc) has a peak annual sales potential of >$2 Billion[8] - Petosemtamab (HNSCC) has a multi-$Billion peak annual sales potential[8] - Up to six registrational readouts are expected in 2026, enabling 2027 launches[13] Financial Performance and Projections - In 2024, revenue was $3121 million[28] - In 2024, net profit was $1133 million[28] - Darzalex sales in 2024 were $1167 billion and are projected to reach $203 billion by 2030[15]

Company Strategy & Pipeline - Zai Lab is executing a dual-engine growth strategy, leveraging a commercially profitable China platform to power global innovation, with the first U S approval expected by 2028[6] - The company aims to expand its global pipeline to over 9 indications in development by 2030[10, 12] - Zai Lab is focused on oncology and immunology, utilizing next-generation biologic modalities like ADCs and BsAbs, targeting validated targets with proven biology[16] Zoci (ZL-1310) Development - Zoci, a DLL3 ADC, is in a pivotal study and shows data supportive of potential best-in-class status[10] - In 2L SCLC, Zoci demonstrated a 68 4% ORR (Objective Response Rate)[32] - Grade ≥3 TRAEs (Treatment-Related Adverse Events) were 13% with Zoci at the selected Phase 3 dose[32] - Zai Lab anticipates a potential first global approval for Zoci in 2028, targeting a $9+ billion total addressable market across SCLC & NEC[40] Financial Position - The company reported a robust cash position of $817 2 million as of September 30, 2025[10, 20] - 48% of revenue was invested in R&D in 9M 2025[70] ZL-1503 (IL13/IL31R) Development - ZL-1503, a dual IL-13 / IL-31R bispecific antibody, is in a global Phase 1/1b study for Mod-to-Sev AD (Atopic Dermatitis)[19, 49] - Preclinical data showed rapid itch suppression sustained for up to 133 days in non-human primates[50]

Key Objectives & Platform - Denali aims to transform lives by delivering biotherapeutics to the whole body, including the brain[11] - The company's TransportVehicle (TV) platform enables systemic delivery of biologics to the brain and other hard-to-target tissues[12, 24] - Denali's TransportVehicle has demonstrated best-in-class properties for brain delivery, modularity, and safety[50] Near-Term Commercial Opportunities - Denali anticipates launching tividenofusp alfa (DNL310) in 2026 and DNL126 in 2027, establishing a commercial foundation for the Enzyme TransportVehicle (ETV) franchise[13] - Tividenofusp alfa has a PDUFA target action date of April 5, 2026, for accelerated approval[61, 81] - The ETV franchise targets lysosomal storage disorders (LSDs), with a combined market opportunity exceeding $1 billion for MPS II and MPS IIIA[13, 107] Pipeline & Milestones - Denali has a broad clinical-stage pipeline, including programs for Alzheimer's disease, with several near-term milestones expected[14] - The company anticipates an approval decision for tividenofusp alfa, ETV:SGSH Phase 1/2 data, and initiation of Phase 1 studies for OTV:MAPT, ATV:Abeta, and ETV:GAA in the first half of 2026[16] - Denali is developing the next generation of enzyme replacement therapies designed to treat brain and body manifestations of serious genetic diseases[61, 64] Financial Position - Denali has a strong financial foundation with approximately $873 million in cash and investments as of Q3 2025, plus $488 million from royalty financing and an equity capital raise in December 2025[146]

PresentationOn stage, we have the team from Coherus Oncology. I will now pass the mic to Dennis Lanfear, who is our CEO, for a short presentation, followed by a live audience Q&A. Denny, the stage is yours.Good morning everyone. It's still the morning. Thanks for joining us for another session at the 44th JPMorgan Healthcare Conference. I'm Brian Cheng. I'm one of the senior biotech analysts here at the firm.Dennis LanfearChairman, President & CEO Thank you, Brian, and thank you to the organizers for having ...

- Duchenne: Dosed 33 participants in the Phase 1/2 INSPIRE DUCHENNE clinical trial as of January 9, 2026; SGT-003 continues to be generally well tolerated using a steroid-only prophylactic immunomodulation regimen - - Duchenne: First participant enrolled in IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled, ex-U.S. clinical trial, with dosing expected in Q1 2026 - - FA: First participant dosed in Phase 1b FALCON clinical trial; FDA Orphan Drug designation granted to SGT-212, the only d ...

PresentationGreat. Good morning. Welcome. My name is Jess Fye. I'm a biotech analyst at JPMorgan, and we're continuing the 44th Annual Healthcare Conference today with Crinetics. You're going to hear a presentation from the management team, and then we're going to go into some Q&A. [Operator Instructions] So with that out of the way, I'm excited to turn it over to Crinetics CEO, Scott Struthers.R. StruthersFounder, President, CEO & Director Thanks, Jess, and thank you all for coming. It's great to see you. ...

PresentationGood morning, everybody. Thank you so much for joining us here on Day 2 of the JPMorgan Healthcare Conference. My name is Bhavana Balakrishnan. I am an associate with the Healthcare Investment Banking team. Thank you for joining us here for the presentation of Sionna Therapeutics. With us, we have Mike Cloonan, President and Chief Executive Officer; Elena Ridloff, Chief Financial Officer; and Charlotte McKee, Chief Medical Officer. Over to you, Mike.Michael CloonanPresident, CEO & Director Thank ...