Executive Summary / Key Takeaways

• Legend Biotech is solidifying its leadership in cell therapy, driven by the exceptional clinical profile and commercial success of CARVYKTI, which achieved record CAR-T sales of $439 million in Q2 2025.
• CARVYKTI demonstrates superior efficacy, including a 45% reduction in the risk of death in second-line multiple myeloma patients and unprecedented 5-year progression-free survival in heavily pretreated patients, establishing a new benchmark in the field.
• The company is on a clear trajectory towards profitability, anticipating CARVYKTI operational breakeven by the end of 2025 and company-wide profitability in 2026, supported by robust manufacturing expansion and disciplined expense management.
• Strategic initiatives, including expanding market access through outpatient administration and community penetration, coupled with a diversified pipeline in solid tumors and autoimmune diseases, position Legend for durable long-term growth.
• Key factors for investors to monitor include continued manufacturing scale-up, successful integration of CARVYKTI's overall survival data into the U.S. label, and the advancement of its innovative next-generation cell therapy platforms.

A New Era in Multiple Myeloma: Legend Biotech's Ascendancy

Legend Biotech Corporation, founded in 2014, has rapidly emerged as a pivotal player in the biopharmaceutical landscape, particularly in the transformative field of cell therapy. Following its spin-off and IPO in 2020, the company's trajectory was fundamentally reshaped by the February 2022 FDA approval of CARVYKTI (ciltacabtagene autoleucel), its flagship BCMA-directed autologous CAR-T therapy for multiple myeloma. This marked Legend's definitive entry into the commercial stage, setting the foundation for its current strategic focus on maximizing CARVYKTI's potential while cultivating a robust pipeline of next-generation cell therapies.

The company's overarching strategy is to leverage CARVYKTI's differentiated clinical profile to capture significant market share in multiple myeloma, expand manufacturing capacity to meet surging demand, and drive operational efficiencies to achieve profitability. This commercial success is intended to fuel strategic investments in innovative research and development, positioning Legend Biotech as a leader in addressing intractable diseases beyond multiple myeloma. The broader industry landscape for CAR-T therapies is characterized by rapid innovation, evolving regulatory frameworks, and a growing emphasis on real-world evidence and patient access.

Technological Edge: CARVYKTI's Differentiated Profile

At the heart of Legend Biotech's investment thesis lies CARVYKTI, a best-in-class BCMA-directed autologous CAR-T cell therapy. Its core technology is designed to deliver a single, potent infusion that offers profound and durable responses in multiple myeloma patients. The tangible benefits of CARVYKTI are substantial and quantitatively supported by clinical data.

In the CARTITUDE-4 study, CARVYKTI demonstrated a remarkable 45% reduction in the risk of death compared to standard therapies in second-line multiple myeloma patients after a three-year follow-up. This overall survival benefit is a "holy grail" in oncology and a significant differentiator. Furthermore, in the CARTITUDE-1 study, which involved heavily pretreated relapsed/refractory multiple myeloma patients, an unprecedented one-third of patients remained alive and progression-free for five years or more after a single infusion, without further myeloma treatment. The median progression-free survival (PFS) in CARTITUDE-1 was 35 months, and in CARTITUDE-4, the median PFS for patients with high-risk cytogenetics was 37 months, significantly outperforming the 10 months seen with standard of care. The therapy also achieved an 89% minimal residual disease (MRD) negativity rate compared to 38% for standard of care in CARTITUDE-4 after three years.

A critical technological differentiator for CARVYKTI is its unique safety profile, specifically the delayed onset of Cytokine Release Syndrome (CRS), with a median onset of seven days. This characteristic enables extensive outpatient administration, with over half of CARVYKTI patients currently receiving treatment in an outpatient setting. This not only enhances the patient experience but also significantly expands treatment capacity for hospitals, a crucial advantage in a resource-constrained healthcare environment. The manufacturing process for CARVYKTI boasts a 97% success rate and a median turnaround time of 30 days, which is considered highly efficient within the CAR-T industry.

Expanding Horizons: R&D and Next-Generation Cell Therapies

Beyond CARVYKTI, Legend Biotech is actively investing in a diversified pipeline, leveraging its expertise to address other high-unmet-need areas. The company is building a new research facility in Philadelphia, with a key focus on in vivo CAR-T delivery. This next-generation approach aims to reprogram immune cells directly within the body, eliminating the need for complex ex-vivo cell engineering, manufacturing, and lymphodepletion. The first patient dosing in an investigator-initiated study for this platform is anticipated in June or July 2025, with preliminary clinical results expected by the end of 2025.

In solid tumors, Legend is advancing LB1908, a Claudin 18.2 targeted CAR-T, which has shown encouraging antitumor activity and manageable safety in a Phase I study. Dose escalation is expected to conclude by the end of summer 2025, with dose expansions to follow. Similarly, LB2102, a DLL3 targeting CAR-T candidate, has reported no dose-limiting toxicities and preliminary efficacy signals in its Phase I study. Legend holds an exclusive global licensing agreement with Novartis for DLL3-targeting CAR-T therapies. The company is also exploring allogeneic cell therapies and has initiated a first-in-human study for a trispecific CD19, CD20, CD22 targeting CAR-T for autoimmune indications, with enrollment ongoing and initial clinical data expected in 2025. These R&D initiatives are critical for establishing Legend's long-term growth beyond CARVYKTI, broadening its therapeutic reach, and solidifying its position as an innovation leader in cell therapy.

Financial Performance and Operational Momentum

Legend Biotech's financial performance reflects the accelerating commercial success of CARVYKTI and a disciplined approach to operational efficiency. In the second quarter of 2025, CARVYKTI net trade sales reached approximately $439 million, marking a 136% increase year-over-year and a 19% increase quarter-over-quarter. This achievement set a new industry record for CAR-T sales in a single quarter. U.S. net trade sales contributed $358 million, growing 114% year-over-year, while international sales surged to $81 million, a four-fold increase over the prior year. Total revenues for Q2 2025 were $255 million, driven by a 136% year-over-year growth in collaboration revenue. While the company reported a GAAP net loss of $125 million, it achieved an adjusted net income of $10 million, excluding a significant $111 million unrealized foreign exchange loss. Operating loss was nearly halved to $22 million in Q2 2025 from $41 million in the prior year, demonstrating strong progress in operational efficiency and expense management.

The gross margin on net product sales stood at a healthy 57% in Q2 2025. R&D expenses saw a slight decline to $98 million (39% of revenue), while SG&A grew 23% to $81 million (32% of revenue), reflecting ongoing investment in commercialization.

Legend Biotech maintains a robust liquidity position, with approximately $1 billion in cash and equivalents as of Q2 2025. This strong balance sheet is crucial for funding ongoing operations, capital expenditures, and future innovation, as the company anticipates achieving CARVYKTI operational breakeven by the end of 2025 and company-wide profitability in 2026, excluding unrealized foreign exchange fluctuations. This guidance is supported by a joint investment with Johnson & Johnson of $150 million in CapEx for the Tech Lane Phase 2 expansion, projected through 2028, underscoring confidence in long-term demand.

Competitive Landscape and Strategic Positioning

Legend Biotech operates within a highly competitive cell therapy market, facing established players like Gilead Sciences (GILD), Bristol-Myers Squibb (BMY), and Novartis , as well as its collaboration partner, Johnson & Johnson , which also has a broader oncology portfolio. While these competitors have established CAR-T therapies for various indications, CARVYKTI has carved out a distinct and leading position in multiple myeloma.

CARVYKTI's competitive advantages are rooted in its superior clinical profile. The International Myeloma Working Group (IMWG) recommends pursuing CAR-T therapy first for patients who are reasonable candidates for both BCMA CAR-T and bispecific T-cell engagers, a testament to CARVYKTI's efficacy. Legend Biotech has achieved nearly 90% market share in the BCMA CAR-T class in the U.S. and Germany, demonstrating strong market penetration. The company is strategically "owning" the second through fourth-line opportunity within the J&J portfolio, with nearly 60% of CARVYKTI orders now coming from these earlier line settings. This focus is critical as competitors' bispecifics are largely in the BCMA-naive population, which is becoming increasingly difficult to find as CARVYKTI moves into earlier treatment lines.

Compared to other CAR-T therapies, CARVYKTI's delayed CRS onset (median 7 days) is a significant differentiator, enabling over half of treatments to occur in an outpatient setting. This contrasts with competitors whose acute CRS onset often necessitates inpatient administration, limiting capacity and increasing costs. Furthermore, payers currently deny payment for transitioning from one CAR-T drug to another, reinforcing the importance of being the first and most effective option. As a later entrant, any new competitor would face increased hurdles in launching due to established hospital formularies and preferred provider networks.

Operationally, Legend Biotech is expanding its global footprint, with 123 authorized treatment centers in the U.S. and a total of 213 global sites activated with Johnson & Johnson (JNJ). The FDA's recent removal of Risk Evaluation and Mitigation Strategies (REMS) for CAR-T therapies, including CARVYKTI, is expected to further improve patient access by reducing monitoring requirements. The company is also actively working to expand access in the community setting, with Virginia Oncology Associates as a first partner, and notes that Texas has already removed FACT accreditation requirements for reimbursement, a trend Legend expects other states to follow.

Outlook and Risks

Legend Biotech's outlook is characterized by continued strong growth for CARVYKTI and strategic pipeline advancement. The company anticipates receiving approval for its physical expansion in Raritan and initiating commercial production at its Tech Lane facility in Ghent, Belgium, by the end of 2025. These expansions, along with Novartis's (NVS) facility reaching full capacity, are expected to bring Legend's combined in-network capacity to over 10,000 doses per year by the end of 2025. Management projects sequential growth for CARVYKTI sales throughout 2025, with accelerated growth in the second half of the year. The overall survival benefit from CARTITUDE-4 is expected to be included in the U.S. label by the fall of 2025, further strengthening CARVYKTI's market position. By the end of 2025, Legend expects two-thirds to three-quarters of its CARVYKTI revenue to originate from the second-to-fourth line indication.

Despite the positive outlook, investors should consider several risks. Foreign exchange volatility has significantly impacted reported net income, with unrealized losses of $111 million in Q2 2025 and $52 million in Q1 2025. While the company believes its exposure to pharma-related tariffs is immaterial due to U.S.-based production, global trade policies remain a dynamic factor. The management of neurotoxicity, particularly delayed events like Parkinsonism, remains a focus, though the incidence has decreased significantly in earlier lines (from 6% in CARTITUDE-1 to 1% in CARTITUDE-4) due to improved patient management and bridging therapies. The company is actively implementing ALC monitoring and dexamethasone intervention strategies in ongoing trials to further mitigate these risks. Competition from emerging bispecifics and other CAR-T therapies, while currently less differentiated, could intensify, necessitating continuous innovation and market education.

Conclusion

Legend Biotech Corporation stands at a pivotal juncture, having successfully commercialized a groundbreaking cell therapy in CARVYKTI that is redefining the treatment paradigm for multiple myeloma. The company's relentless focus on clinical differentiation, evidenced by unprecedented overall survival and durability data, has propelled CARVYKTI to record sales and a dominant market share in its class. With a clear path to profitability by 2026, underpinned by strategic manufacturing expansion and disciplined financial management, Legend is transforming its operational efficiency into a sustainable growth engine. The ongoing investment in a diversified, next-generation pipeline, including in vivo CAR-T and autoimmune programs, further strengthens its long-term potential. Legend Biotech's technological leadership and strategic market positioning against competitors, particularly through its unique outpatient administration capabilities, underscore a compelling investment thesis for those seeking exposure to the forefront of cell therapy innovation.