Financial Performance & Growth - Total revenue for Q1 2025 reached $125 million, representing a 15% growth [6] - Galafold revenue in Q1 2025 was $1042 million, with a 14% patient demand growth [23] - Pombiliti + Opfolda revenue in Q1 2025 was $21 million, showing a 92% growth at CER [50] - The company expects to surpass $1 billion in total revenue in FY 2028 [6] Strategic Priorities & Guidance - The company aims for a total revenue growth of 15-22% at CER in FY 2025 [7, 77] - Galafold revenue is projected to grow by 10-15% at CER in FY 2025 [6, 7] - Pombiliti + Opfolda revenue is expected to increase by 50-65% at CER in FY 2025 [6, 7, 53, 77] - The company anticipates positive GAAP net income during H2 2025 [7, 77] Product & Market Overview - The global Fabry market is projected to reach approximately $3 billion by 2029 [14, 15] - The global Pompe market was approximately $15 billion in 2024 and is expected to exceed $2 billion by 2029 [42, 43] - DMX-200, a Phase 3 program for FSGS, addresses a market affecting over 40000 people in the US [67]

Clinical Programs & Data - CT-0508 monotherapy showed stable disease (SD) in 29% (4/14) of patients, with 44% SD in HER2 3+ patients [13] - 75% (6/8) of HER2 3+ patients exhibited a decrease in ctDNA, with reductions up to 93% [17] - CT-0525, a CAR-Monocyte therapy, has a ~2,000-fold increased exposure compared to CT-0508 in preclinical models [44] - The first patient was treated with CT-0525 in 2Q 2024, with initial data expected in 4Q 2024 [43] In Vivo CAR-M Collaboration with Moderna - The collaboration includes up to 12 targets, with 7 nominated [64] - Carisma received an $80 million upfront payment and is eligible for over $3 billion in potential milestones and royalties [64] - Nomination of the first development candidate targeting GPC3 for HCC triggered a $2 million milestone payment [6, 65] Financial Status - As of June 30, 2024, Carisma had $40.4 million in cash and cash equivalents [100] - The company's cash runway is expected to last into 3Q 2025 [6, 100] Fibrosis Program - Preclinical data demonstrates that engineered macrophages can reduce liver fibrosis, with Relaxin-IL10 macrophages showing >100% reduction in collagen in one model [84] - Nomination of a development candidate for liver fibrosis is expected in 1Q 2025 [8, 93]

BDTX-1535 Clinical Development - BDTX-1535 is a MasterKey therapy targeting families of oncogenic mutations, designed to expand the addressable patient population [4,5,8] - BDTX-1535 is in Phase 2 clinical trials for 2L/3L NSCLC with final data expected in H1 2026, and 1L NSCLC with initial data expected in Q4 2025 [9] - BDTX-1535 is also in Phase 0/1 clinical trials for GBM with initial data in newly diagnosed patients expected in H1 2026 [9] - Preliminary Phase 2 data shows a 42% ORR in recurrent NSCLC patients with PACC-NCM and/or C797S mutations [58] - The company anticipates FDA feedback on the approval pathway for BDTX-1535 in Q4 2025 [84] EGFR Mutations and Treatment Landscape - 23-30% of newly diagnosed EGFRm NSCLC patients carry non-classical mutations (NCMs) [14,17,28] - Real-world data shows that approximately 60% of 1L patients with EGFR-NCM NSCLC receive chemotherapy [21,22,28] - Median time on treatment for 1L NCM NSCLC is less than 8 months [23,28] - BDTX-1535 demonstrates high potency across the spectrum of NCMs and maintains activity where osimertinib loses potency [26,28,32,34] Financial Status - The company ended Q1 2025 with $152.4 million, providing a cash runway into Q4 2027 [5]

Acrivon's AP3 Platform and Pipeline - Acrivon utilizes its Acrivon Predictive Precision Proteomics (AP3) platform to overcome limitations of genetics-based precision medicine [2, 7] - The AP3 platform enables exact matching of disease-driving dysregulated pathways with a drug's mechanism of action [8] - Acrivon's pipeline includes ACR-368 (CHK1/CHK2 inhibitor) in Phase 2 trials for endometrial cancer and ACR-2316 (WEE1/PKMYT1 inhibitor) in Phase 1 for AP3-identified tumor types [18] - A novel cell cycle program with an undisclosed target is anticipated to have a development candidate nomination in 2025 [18, 98] - Additional AP3-driven programs are in early discovery for autoimmune/inflammatory candidates [18] ACR-368 and Endometrial Cancer - ACR-368 is being evaluated in a registrational intent Phase 2 single-arm trial based on predicted sensitivity in OncoSignature-positive endometrial cancer patients [18] - In endometrial cancer patients, ACR-368 monotherapy showed a confirmed ORR of 35% in OncoSignature-positive patients [53] - ACR-368 is also being studied with ultra-low dose gemcitabine (ULDG) as a sensitizer in OncoSignature-negative patients [18] - Preclinical data suggests AP3-predicted sensitization to ACR-368 by LDG correlates with OncoSignature upregulation [64] ACR-2316 and WEE1/PKMYT1 Inhibition - ACR-2316 is a novel dual WEE1/PKMYT1 inhibitor designed using the AP3 platform to overcome limitations of benchmark inhibitors [97, 104] - ACR-2316 demonstrated superior preclinical potency versus benchmark WEE1/PKMYT1 inhibitors [111] - Initial clinical activity was observed with ACR-2316 at Dose Level 3, showing approximately 25% tumor shrinkage in a patient with prior chemotherapy and anti-PD-1 therapy [138] Financial Status - As of March 31, 2025, Acrivon had $1648 million in cash and investments, projecting a runway into Q2 2027 [182]

Pipeline and Clinical Trials - Cartesian Therapeutics is pioneering mRNA cell therapies for autoimmunity, with multiple anticipated near-term catalysts[5] - Phase 3 AURORA study of Descartes-08 for Myasthenia Gravis (MG) is expected to commence in 1H25[8] - An open-label Phase 2 trial of Descartes-08 in Systemic Lupus Erythematosus (SLE) is ongoing, with data readout expected in 2H25[8] - A Phase 2 pediatric basket trial, including juvenile SLE, juvenile MG, and other conditions, is expected to initiate in 2H25[8, 12] - Dosing is underway in a first-in-human Phase 1 dose escalation trial for Descartes-15, a next-generation mRNA CAR-T candidate[8] Descartes-08 Efficacy and Safety - In a Phase 2b trial, participants treated with Descartes-08 maintained deep and durable responses over 12 months[16] - At Month 4, participants in the primary efficacy dataset experienced an average MG-ADL reduction of 5.5 points[23] - 33% of participants achieved minimum symptom expression at Month 6[23] - 80% of participants reaching Month 12 maintained a clinically meaningful response[23] - In participants with no prior exposure to biologics, the average MG-ADL reduction was 6.6 points at Month 4[26] - 57% of participants with no prior exposure to biologics achieved minimum symptom expression at Month 6[26] - 100% of participants with no prior exposure to biologics reaching Month 12 maintained a clinically meaningful response[26] - The safety profile of Descartes-08 supports outpatient administration, with no new types of adverse events reported[16, 28] Financial Position - Cartesian Therapeutics has a strong balance sheet with approximately $220.9 million as of September 30, 2024[9] - This is expected to support planned operations, including completion of the planned Phase 3 trial of Descartes-08 for MG, into mid-2027[9]

Type 1 Diabetes (T1D) Program - Sana's hypoimmune platform (HIP) overcomes allogeneic rejection in people, which is confirmed by 4-week and 12-week data[4] - Type 1 diabetes affects 94 million children and adults, and is projected to affect 164 million by 2040[12, 13] - Type 1 diabetes leads to 201600 deaths per year and costs $81 billion worldwide annually[17] - SC451, a HIP-modified stem cell-derived pancreatic islet therapy, is advancing toward the clinic with an expected IND filing as early as 2026[114] - HIP-modified PSC differentiated islet cells transplanted into muscle persist & control blood glucose in mice for >15 months[64] Autoimmune Disease Program - B-cell mediated autoimmune diseases affect >5 million patients[68] - SC291, a HIP-modified CD19 CAR T, leads to deep B-cell depletion and has significant potential in B-cell mediated autoimmune diseases, with an ongoing GLEAM study[114] - Sana's T cell manufacturing process provides ~85% full knock-out of MHC class I and II, >995% TCR negative cells[79] - Fusogen platform offers the potential to treat B-cell mediated autoimmune diseases and B-cell cancers with NO lymphodepletion with an expected IND filing as early as 2026[114] Oncology Program - SC262, a HIP-modified CD22 CAR T, has meaningful potential in treating CD19 CAR T relapsed patients, with an ongoing VIVID study[114] - Estimated ~12000 B cell malignancy patients treated with CD19 CAR T in 2027, with ~35-40% durable complete responses, leading to ~7500 CAR T failures annually[106]

Company Vision and Strategy - Metagenomi's vision is to harness its metagenomics platform to create curative genetic medicines for patients[8] - The company's strategy involves using a diverse and modular genome editing toolbox to precisely target any site in the human genome, addressing the full spectrum of genetic diseases[9] - Metagenomi focuses on investments in development and manufacturing across gene editing technologies to enable rapid advancement to the clinic[11] Disease Targets and Pipeline - Hundreds of millions of people worldwide have diseases potentially treatable with gene editing, including over 180 million with rare diseases of genetic origin and over 200 million with cardiovascular disease tied to genetic risk factors[14] - The company's pipeline includes programs targeting Hemophilia A (affecting more than 500,000 patients globally), Transthyretin Amyloidosis (TTR) (affecting 300,000–500,000 patients globally), and Refractory Hypertension (affecting 900,000 patients in the US)[14, 19] - Metagenomi's Hemophilia A program, MGX-001, has demonstrated sustained Factor VIII activity in non-human primate (NHP) studies for more than 16 months[53] Technology and Platform - Metagenomi's metagenomics platform is the foundation of its gene editing toolbox, involving proprietary sampling, AI-powered screening, and engineering & optimization[20, 22, 24, 25] - The company has a proprietary library of highly precise and efficient nucleases, including ultra-small systems (SMARTs), providing a programmable chassis for other gene editing tools[31] - Metagenomi's base editing platform achieves efficient multiplex editing for cell engineering, with reprogrammed chassis increasing genome targetability[112, 113] Partnerships and Corporate Overview - Metagenomi has strategic collaborations, including one with Ionis focusing on cardiometabolic diseases, with up to 8 targets and potential milestone payments and royalties up to $2.9 billion[50] - The company is well-capitalized with a cash runway into 2027[135]

Plinabulin's Mechanism and Preclinical Evidence - Plinabulin is a unique tubulin binder with a distinct binding site compared to other tubulin-binding agents[16, 21] - Preclinical studies show that Plinabulin, combined with radiation and anti-PD-1, activates dendritic cells (DCs), stimulates T-cell proliferation, and achieves abscopal effects[17, 24] - Plinabulin induces DC maturation, especially when administered after irradiation (IR), and Plinabulin-treated DCs stimulate T cell proliferation, enhanced by combining with IR[25] Clinical Efficacy and Immune Activation - In a Phase 1 study, the Plinabulin triple combination (with radiation and PD-1/PD-L1 inhibitors) led to an 80% disease control rate (DCR) in 10 IO-refractory patients[18, 32] - Durable responses were observed in 2 Hodgkin lymphoma patients who progressed after 12 or 16 prior lines of therapy[33] - Responding patients exhibited early immune activation with DC maturation and proinflammatory monocytes in the peripheral blood across six different cancer types[19, 35] Summary and Future Directions - Plinabulin demonstrates induction of DC maturation and re-sensitization to anti-PD(L)1 in IO-refractory tumors[37] - The impressive overall results, showing a high 80% disease control rate in ICI-refractory and heavily pre-treated patients, warrant further clinical studies of Plinabulin/IO combinations in IO-refractory settings[37, 38]

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CARVYKTI® Performance and Market Position - CARVYKTI® demonstrates superior Overall Survival (OS) compared to Standard of Care (SoC) in Multiple Myeloma[15, 25] - CARVYKTI® has a strong launch with a 79% Net Trade Sales Compound Annual Growth Rate (CAGR) since launch, leading the industry[15] - CARVYKTI® Net Trade Sales grew by 135% in Q1 2025 compared to Q1 2024[34] - Outpatient treatment represents up to approximately 48% of CARVYKTI® volume in the U S [44] Manufacturing and Supply - The company aims to achieve 10,000 annualized doses of CARVYKTI® exiting 2025 and 20,000 annualized doses exiting 2027[58] - Commercial production at the Tech Lane facility in Belgium is targeted to initiate in the second half of 2025[58] Pipeline and R&D - The company has 11 pipeline programs in Hematologic Malignancies, Solid Tumors, and Autoimmune Diseases[11] - Legend Biotech and Novartis have an exclusive agreement to advance DLL3-targeted CAR-T therapies, including LB2102 for small cell lung cancer[74] - LB2102 targets DLL-3, which is approximately 80% positive in Small Cell Lung Cancer (SCLC)[80] Multiple Myeloma Market - Multiple Myeloma accounts for 10% of all hematologic cancers[17] - There were 187,952 new cases of Multiple Myeloma worldwide in 2022[18]