Sotagliflozin Program - Lexicon is on track for a 2026 resubmission of the New Drug Application (NDA) for Zynquista (sotagliflozin) for type 1 diabetes (T1D) based on STENO1 study data[12] - The SONATA-HCM Phase 3 study, evaluating approximately 500 patients with both obstructive and non-obstructive HCM, is accelerating towards 2026 enrollment completion, with topline results anticipated in Q1 2027[13] - Approximately 11 million people in the U S have either obstructive or non-obstructive HCM, but only ~1% CMI total market penetration[14, 15] Pilavapadin Program - Phase 3 development of Pilavapadin for Diabetic Peripheral Neuropathic Pain (DPNP) is planned, with potential for additional indications in Phase 2[6] - Approximately 9 million U S patients have progressive DPNP, and 60% of patients have tried multiple treatments[23] - Phase 2 studies in DPNP showed clinically meaningful efficacy of the 10mg dose, with a 2-point average daily pain score (ADPS) reduction from baseline at 12 weeks[28] LX9851 Program - IND-enabling studies for LX9851 in obesity/weight management are completed, with clinical development preparations underway[6, 38] - Lexicon achieved an initial $10 million milestone from Novo Nordisk, with the potential to achieve an additional $20 million in milestone payments in 2026, and up to $950 million in remaining potential milestones plus tiered royalties on net sales[38, 39] Financial Status - Lexicon ended 2025 with $1252 million in cash, investments, and restricted cash, sufficient to support planned operations into 2027[39]

JP Morgan Healthcare Conference January 2026 NASDAQ GS ALXO This presentation concerns product candidates that are under clinical investigation, and which have not yet been approved for marketing by the U.S. Food and Drug Administration. These product candidates are currently limited by federal law to investigational use, and no representation is made as to their safety or effectiveness for the purposes for which they are being investigated. This presentation also contains estimates and other statistical da ...

Financial Performance & Growth - Total revenue for 2025 reached $33.6 million, representing a 20% year-over-year growth[13] - Recurring revenue accounted for $17.8 million in 2025, making up 53% of the total revenue[14] - The company achieved a significant gross margin improvement in 2025[14] - Q4 2025 total revenue was $11.3 million, a 37% growth[14] Market Position & Strategy - Rapid Micro Biosystems' Growth Direct system is used by 75% of the top 20 global pharmaceutical companies[13, 34] - 86% of approved CAR-T manufacturers have placed Growth Direct systems[13, 35] - The company has a land and expand strategy, focusing on enterprise deployments and new geographies[31, 32] - The company has 190 cumulative system placements[13, 35] Technology & Innovation - The Growth Direct platform automates microbial quality control (MQC), reducing the process to 2 steps and halving the time to results[25] - The company has 155 cumulative system validations[13, 35] Partnerships - Strategic partnership with MilliporeSigma is expected to drive growth, margin expansion, and innovation[14]

Pipeline and Milestones - Sutro is developing a differentiated pipeline of single- and dual-payload ADCs, focusing on complex targets and overcoming resistance [12, 13] - Initial Phase 1 data for STRO-004, a Tissue Factor (TF)-targeting ADC, is expected in mid-2026 [12, 59] - An IND submission for STRO-006, an ITGB6-targeting ADC, is expected in 2026 [12, 59] - An IND submission for STRO-227, a PTK7-targeting dual-payload ADC, is expected in 2026-2027 [13, 50, 59] - The first immunostimulatory ADC (iADC) program is expected to enter the clinic in early 2026 [56, 59] STRO-004 (TF-Targeting ADC) - STRO-004 demonstrated promising anti-tumor activity in multiple TF-expressing cancer models, with a disease control rate (DCR) of 93% (28/30) and an overall response rate (ORR) of 73% (22/30) in PDX models [25] - STRO-004 is well-tolerated at 50 mg/kg in non-human primates (NHPs) [12] STRO-006 (ITGB6-Targeting ADC) - STRO-006 is designed for superior selectivity, safety, and stability, targeting ITGB6 [32] - In HNSCC PDX models, STRO-006 at 5 mg/kg showed superior anti-tumor activity compared to aITGB6 ADC (DAR4 MMAE) at 5 mg/kg, with an ORR of 71% (12/17) vs 47% (8/17) [37] - In HNSCC PDX models, STRO-006 showed ADC response (below baseline) at end of study 64% (7/11) vs aITGB6 DAR4 MMAE 27% (3/11) [38] Dual-Payload ADCs - Dual-payload ADCs have the potential to overcome resistance resulting from conventional ADCs and reduce toxicity compared to ADC combination approaches [44]

Market Opportunity & Growth Strategy - The US nerve care market represents a large and underserved $56 billion opportunity[9] - Axogen is pursuing a strategic plan (2025-2028) with a revenue CAGR target of 15-20%[25, 72] - The company aims to achieve operational cash flow exceeding $60 million per year by the end of 2028[73] Clinical & Technological Leadership - Avance is the first FDA-approved biologic treatment for nerve discontinuities in patients aged one month and older[18] - Axogen has treated over 200,000 patients and has over 300 clinical and scientific publications supporting its nerve repair algorithm[26] - Allograft is increasingly preferred for nerve repair, with 41% preferring it for gaps >2cm in 2024[33] Reimbursement & Coverage - Approximately 35% of commercial lives remain uncovered, indicating an opportunity for expanded coverage[24, 46] - In FY25, 198 million lives were added across ten regional BCBS plans (182 million private; 16 million Medicare Advantage)[48] - New outpatient code group and Level 3 Nerve Procedure Code increases reimbursement for hospitals and ASC's[49] Financial Performance - Axogen's revenue has grown from $599 million in 2021 to $2252 million in 2025, representing a 149% 5-year CAGR[66, 67] - The company is expanding EBITDA, with projections of $198 million and $214 million for EBITDA and Adjusted EBITDA respectively in 2025[69]

Company Overview - Molecular Partners is a clinical-stage biotech company founded in 2004, with operations and listings in Switzerland (SIX, 2014) and the US (Nasdaq, 2021)[12] - The company is financed with approximately USD 116 million / CHF 93 million to reach upcoming value inflection points[12] - Molecular Partners focuses on oncology with differentiated assets like MP0712 (targeted radiotherapy) and MP0533 (next-gen immune cell engagers)[12] Radio-DARPin Therapeutics (RDT) - The company is developing Radio-DARPin therapeutics, which are designed for precise delivery of potent radio-isotopes to tumors[26, 27] - Molecular Partners has a global partnership with Orano Med to develop 212Pb Radio-DARPin therapeutics, with a 50:50 cost and share split for 4 programs[33, 36] - MP0712, a 212Pb x DLL3 Radio-DARPin, is in Phase 1/2a clinical trials in the US for small cell lung cancer (SCLC) and other neuroendocrine cancers (NECs), with early data expected in 2026[43, 77] - Preclinical data for MP0712 shows high tumor accumulation (Tumor > Kidney) and reduction of established tumors in mice[51] - MP0726, a 212Pb x MSLN Radio-DARPin, is being developed for ovarian cancer and is progressing towards first-in-human (FIH) imaging[86, 94] Other Pipeline Programs - MP0317, a FAP-localized CD40 agonist, is in a Phase 2 combo study in advanced biliary tract cancer, with interim analysis expected by YE 2027[104, 117, 123] - MP0533, a tetra-specific T-cell engager for AML, is in Phase 1/2a, with decisional data expected in H1 2026[124] - The company is developing Switch-DARPin platform for next-generation T cell engagers, with a lead candidate selection expected in H1 2026 and an update at AACR 2026[99, 140] Financial Outlook - The company's cash of approximately USD 116 million (CHF 93 million) ensures funding until 2028[99]

Pipeline and Milestones - Korro Bio anticipates a regulatory filing for KRRO-121 in the second half of 2026 [26, 55] - The company expects to nominate a development candidate (DC) for its GalNAc-conjugated AATD program in the first half of 2026 [26, 55] - A development candidate is also expected for a third GalNAc-conjugated liver asset in the second half of 2026 [26, 55] KRRO-121 for Hyperammonemia - KRRO-121 targets hyperammonemia by stabilizing an intracellular protein in the liver to enhance ammonia clearance capacity [36, 39] - The addressable patient population for KRRO-121 includes 4,200 U S patients with Urea Cycle Disorders (UCD) and 80,000 U S patients with Hepatic Encephalopathy (HE) [37] - The market opportunity for KRRO-121 is estimated at $1 5 billion for UCD and $2 billion+ for HE [37] AATD Program - Korro Bio terminated the REWRITE clinical trial for KRRO-110 in AATD [27, 42] - The company achieved >90% editing of the SERPINA1 transcript using GalNAc delivery in vivo [43] AMPK Activation for Liver Function - In obese mice, approximately 20% editing was sufficient to normalize liver function and reduce body weight [47] Financial Runway - Korro Bio's cash runway extends into the second half of 2027 [14, 55]

Financial Performance & Growth - Veracyte achieved approximately $516 million in total revenue for 2025, with a 17% testing revenue growth[12] - The company expects to achieve greater than 25% adjusted EBITDA in 2025[14] - Veracyte anticipates revenue between $570 million and $582 million in 2026, representing a 10-13% year-over-year growth[61] - The company projects a 14-16% year-over-year testing growth in 2026[61] - The company anticipates approximately 25% adjusted EBITDA margin in 2026[61] Product & Market Expansion - Over 800,000 patients have been served with Veracyte's tests to date[12] - Decipher Prostate has achieved approximately 33% market penetration in the U S[19] - Afirma has achieved approximately 38% market penetration in the U S thyroid cancer market[31] - The company plans to launch TrueMRD for muscle-invasive bladder cancer (MIBC) and Prosigna LDT in 2026[64] - Veracyte is developing IVD versions of its tests, including Decipher qPCR and Prosigna NGS, for geographic expansion[54]

Financial Performance & Cash Position - Zevra exited Q3 2025 with a strong cash position of $230.4 million[8], and total debt of $61.3 million[44] - The company generated net revenue of $72.3 million in the 9-month period[8], and $26.1 million for Q3 2025[43] - Q3 2025 net loss was $(0.5) million, or $(0.01) per basic and diluted share[43] Product Portfolio & Pipeline - MIPLYFFA adoption achieved in approximately 40% of diagnosed Niemann-Pick Disease Type C (NPC) patients within the first year of launch[13] - Payor coverage for MIPLYFFA reached 66% of covered lives as of Q3 2025[24] - Approximately 80% of patients who participated in the Phase 2/3 clinical trial for MIPLYFFA took miglustat[20] - Celiprolol Phase 3 DiSCOVER trial is ongoing for Vascular Ehlers-Danlos Syndrome (VEDS), with 44 of 150 patients enrolled as of Q3 2025[40] - For VEDS patients on celiprolol, the annual major vascular event rate is approximately 5% compared to approximately 12% in untreated patients[40] Market & Regulatory - Approximately 900 individuals in the U S live with NPC, of which 300-350 are diagnosed or treated[27] - Approximately 1,100 individuals are living with NPC in Europe[27] - MAA for Arimoclomol is under review by the EMA[8, 13]

Lanifibranor's Potential - Lanifibranor is presented as a potential best-in-disease oral therapy for MASH (Metabolic dysfunction-Associated SteatoHepatitis), targeting progressive fibrosis in F2/F3 MASH patients[1,5] - The company anticipates regulatory filings in 2027 and a potential commercial launch in 2028, pending approval[8] - Phase 2b trial data showed a 24% effect size on the dual endpoint of fibrosis improvement and MASH resolution in just 24 weeks[70] - The Phase 3 NATiV3 clinical trial is fully recruited with 1,009 patients in the main cohort and 410 in the exploratory cohort[56] - Topline data from the Phase 3 trial is expected in the second half of 2026[8,64] MASH Market and Medical Understanding - The MASH market is expected to exceed $15 billion by 2035[15] - Approximately 19 million were diagnosed with MASH in the U S in 2025, representing ~10% of the patients with MASH[13] - Approximately 910,000 patients have clinically actionable F2/F3 disease, with about 374,000 (~40%) under treated care[14] Financial Position - The company raised $172 million from investors in November 2025[8] - The company's cash runway extends until the middle of Q3 2027, assuming full exercise of the 3rd tranche[8,67]