MacroGenics Provides Update on Corporate Progress and 2024 Financial Results

On March 20, 2025 MacroGenics, Inc. (NASDAQ: MGNX), a biopharmaceutical company focused on discovering, developing, manufacturing and commercializing innovative antibody-based therapeutics for the treatment of cancer, reported an update on its recent corporate progress and reported financial results for the year ended December 31, 2024 (Press release, MacroGenics, MAR 20, 2025, View Source [SID1234651310]).

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"We concluded 2024 with the achievement of multiple clinical development milestones, including the completion of enrollment in the LORIKEET Phase 2 study evaluating lorigerlimab in combination with docetaxel in patients with mCRPC. We look forward to building upon this momentum in 2025 as we work to advance our novel pipeline of clinical product candidates, including lorigerlimab, MGC026 and MGC028," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "Finally, we have determined that the results of the TAMARACK Phase 2 study of vobra duo in mCRPC do not support additional financial investment by MacroGenics. We believe the B7-H3 target continues to have potential and are pleased with the progress being made with our alternate anti-B7-H3 ADC, MGC026."

Updates on Proprietary Investigational Programs

Lorigerlimab is a bispecific, tetravalent PD-1 × CTLA-4 DART molecule designed to enhance CTLA-4 blockade on dual-expressing, tumor-infiltrating lymphocytes compared to a PD-1/CTLA-4 monoclonal antibody (mAb) combination therapy, while maintaining maximal PD-1 blockade on all PD-1-expressing cells.

Enrollment is now complete in the ongoing LORIKEET Phase 2 trial, a 150 patient randomized study of lorigerlimab in combination with docetaxel vs. docetaxel alone in second-line, chemotherapy-naïve patients with metastatic castration-resistant prostate cancer (mCRPC). The Company expects to provide a clinical update in the second half of 2025.
Based on MacroGenics’ cumulative experience to date from its Phase 1 and Phase 2 studies of lorigerlimab, including in mCRPC – a tumor setting historically insensitive to checkpoint inhibition – the Company plans to conduct the LINNET Phase 2 study. This clinical trial will evaluate lorigerlimab as monotherapy in patients with either platinum-resistant ovarian cancer (PROC) or clear cell gynecologic cancer (CCGC); both represent areas of unmet need and historically have been relatively insensitive to checkpoint inhibitor therapy. The study’s primary endpoint is ORR, with multiple secondary endpoints to be explored. The Company anticipates enrolling up to 40 patients with PROC and up to 20 patients with CCGC in LINNET, which is expected to commence by mid-2025.
Emerging ADC Pipeline. MacroGenics is developing two clinical and one preclinical antibody-drug conjugate (ADC) molecules that each incorporate a novel, glycan-linked topoisomerase I inhibitor (TOP1i)-based payload developed by the Company’s collaboration partner, Synaffix (a Lonza company). These three candidates are described below.

MGC026 is a TOP1i-based ADC that targets B7-H3, an antigen with broad expression across multiple solid tumors and a member of the B7 family of molecules involved in immune regulation. MGC026 shares the same variable domain as that of vobra duo. MGC026 is currently being evaluated in a Phase 1 dose escalation study in patients with advanced solid tumors, with dose expansion in selected indications expected to initiate in 2025.
MGC028 is a TOP1i-based ADC that targets ADAM9, a member of the ADAM family of multifunctional type 1 transmembrane proteins that play a role in tumorigenesis and cancer progression and is overexpressed in multiple cancers. The Company previously presented preclinical data showing antitumor activity of MGC028 in in vivo models. Also, in a non-human primate study, MGC028 was well tolerated at high dose levels with mild, reversible side effects and no ocular toxicity, which is often a concern with tubulin-inhibitor-based ADCs. The first patient was recently dosed in a Phase 1 study of MGC028 in patients with advanced solid tumors.
MGC030 is a preclinical TOP1i-based ADC that targets an undisclosed antigen expressed across several solid tumors. There are currently no approved therapeutics to this target. An Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for MGC030 is planned for 2026.
Updates on Selected Partnered Programs

MGD024 is a next-generation CD123 × CD3 DART molecule. Under an October 2022 exclusive option and collaboration agreement with Gilead Sciences, Inc. (Gilead), MacroGenics continues to enroll patients in a Phase 1 dose escalation study of MGD024 in patients with CD123-positive neoplasms, including acute myeloid leukemia and myelodysplastic syndromes. Gilead has the option to license MGD024 at predefined decision points during the Phase 1 study.
ZYNYZ (retifanlimab-dlwr) is a monoclonal antibody targeting PD-1 that the Company licensed to Incyte Corporation (Incyte) in 2017. In July 2024, Incyte announced positive Phase 3 top-line results for its registrational studies of retifanlimab in squamous cell carcinoma of the anal canal (SCAC) and non-small cell lung cancer (NSCLC) and continues to conduct global studies of retifanlimab across multiple indications. In February 2025, Incyte disclosed that its supplemental Biologics License Application (sBLA) for retifanlimab in advanced/metastatic SCAC was filed with the FDA in December 2024, with approval anticipated in the second half of 2025. To date, MacroGenics has received $365.0 million in upfront and milestone payments from Incyte under the agreement and remains eligible for up to $540.0 million in additional development, regulatory and commercial milestones.
MARGENZA (margetuximab-cmkb) global rights were sold to TerSera Therapeutics LLC (TerSera), a privately-held biopharmaceutical company with a focus on oncology and non-opioid pain management, pursuant to an agreement previously announced. TerSera made a payment of $40.0 million to MacroGenics at closing in November 2024 and MacroGenics may receive additional sales milestone payments of up to an aggregate of $35.0 million. MacroGenics subsequently paid an $8.0 million amendment fee to its former commercialization partner during the fourth quarter of 2024. MacroGenics will manufacture MARGENZA drug substance on behalf of TerSera going forward.
Update on Vobramitamab Duocarmazine

Vobramitamab duocarmazine (vobra duo) is an ADC with a cleavable peptide linker designed to deliver a DNA-alkylating duocarmycin payload to solid tumors that express B7-H3.

Results for the concluded TAMARACK Phase 2 study included, based on a February 21, 2025 data cut-off, mature median radiographic progression-free survival (rPFS) of 9.5 months for the 2.0 mg/kg cohort (95% CI, 8.5-11.2) and 10.0 months for the 2.7 mg/kg cohort (95% CI, 7.4-11.4) in patients with mCRPC. Safety data from the study remained consistent with prior data disclosures.
Based on its assessment of the vobra duo safety and efficacy profile and an internal resource and portfolio review, MacroGenics has decided not to pursue further internal development of vobra duo and will instead explore potential alternatives for partnering this program.
2024 Financial Results

Cash Position: Cash, cash equivalents and marketable securities balance as of December 31, 2024, was $201.7 million, compared to $229.8 million as of December 31, 2023.
Revenue: Total revenue was $150.0 million for the year ended December 31, 2024, compared to total revenue of $58.7 million for the year ended December 31, 2023. The increase was primarily due to a net increase of $85.0 million in revenue recognized from milestones achieved under the Incyte License Agreement.
R&D Expenses: Research and development expenses were $177.2 million for the year ended December 31, 2024, compared to $166.6 million for the year ended December 31, 2023. The increase was primarily due to increased research, development, manufacturing and clinical costs related to MGC028, the Company’s preclinical ADC pipeline and lorigerlimab, offset by decreased development and clinical trial costs related to the Company’s discontinued projects and margetuximab.
SG&A Expenses: Selling, general and administrative expenses were $71.0 million for the year ended December 31, 2024, compared to $52.2 million for the year ended December 31, 2023. The increase was due to an amendment fee paid by MacroGenics to its former commercial partner pursuant to the sale of MARGENZA and increased non-cash stock-based compensation and accrued severance expenses related to the separation agreement with the Company’s Chief Executive Officer.
Other Income: Other income for the year ended December 31, 2024, reflected a $36.3 million gain recognized on the sale of MARGENZA.
Net Loss: Net loss was $67.0 million for the year ended December 31, 2024, compared to net loss of $9.1 million for the year ended December 31, 2023.
Shares Outstanding: Shares of common stock outstanding as of December 31, 2024 were 62,819,857.
Cash Runway Guidance: MacroGenics anticipates that its cash, cash equivalents and marketable securities balance of $201.7 million as of December 31, 2024, in addition to projected and anticipated future payments from partners should extend its cash runway into the second half of 2026. The Company’s expected funding requirements reflect anticipated expenditures related to the ongoing Phase 2 LORIKEET study of lorigerlimab in mCRPC as well as MacroGenics’ other clinical and preclinical studies currently ongoing.
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Linnaeus Therapeutics Announces Publication of Seminal Paper on LNS8801 in Cancer Research Communications

On March 20, 2025 Linnaeus Therapeutics, Inc. ("Linnaeus"), a privately held biopharmaceutical company focused on the development and commercialization of novel, small molecule oncology therapeutics, reported that seminal data describing Linnaeus’s clinical candidate, LNS8801, was published in Cancer Research Communications (Press release, Linnaeus Therapeutics, MAR 20, 2025, View Source [SID1234651326]). This paper characterizes LNS8801 as a developable pharmaceutical drug candidate, demonstrating its activity in cancer and highlighting a predictive biomarker.

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The manuscript, entitled "LNS8801: An enantiomerically pure agonist of the G protein-coupled estrogen receptor suitable for clinical development," was authored by Natale et al. The publication can be viewed at: View Source

This new study highlights the potential of LNS8801, a first-in-class, oral compound targeting the G protein estrogen receptor (GPER), to be used as a novel cancer therapy. Linnaeus researchers and collaborators discovered that LNS8801 is the active component of the widely studied GPER agonist, G-1. In preclinical models, LNS8801 demonstrated potent, oral, GPER-dependent anticancer effects. This study also demonstrates that a common genetic variant of GPER may influence patient response, offering a potential mechanism-linked biomarker for patient selection and personalized therapy. These findings complement the data generated in ongoing clinical trials of LNS8801 for cancer.

"The validation of our science by the acceptance of this paper in Cancer Research Communications underscores the importance of GPER as a therapeutic target," said Patrick Mooney, MD, Chief Executive Officer of Linnaeus. "This data demonstrates that using LNS8801 to target GPER has therapeutic effects in melanoma and other GPER-positive cancers and is the appropriate molecule for clinical development. We are excited by the data we have seen in our clinical phase 1/2 study, and we are poised to open enrollment in a randomized, controlled study in the near future."

Linnaeus anticipates initiating a randomized controlled clinical trial testing LNS8801 in unresectable, treatment-refractory, cutaneous melanoma patients this year. This study will randomize 135 biomarker-positive patients to receive either LNS8801 monotherapy, LNS8801 in combination with pembrolizumab, or physician’s choice therapy. The study will assess progression-free survival and overall survival between the groups.

About LNS8801

LNS8801 is an orally bioavailable and highly specific and potent agonist of GPER whose activity is dependent on the expression of GPER. GPER activation by LNS8801 rapidly and durably depletes c-Myc protein levels. In preclinical cancer models, LNS8801 displays potent antitumor activities across a wide range of tumor types, rapidly shrinking tumors and inducing immune memory.

In the ongoing clinical study in humans, LNS8801 has been safe and well tolerated. Additionally, LNS8801 has demonstrated target engagement, c-Myc protein depletion, and clinical benefit in patients with advanced cancers, and a predictive biomarker has been identified.

Monte Rosa Therapeutics Announces Fourth Quarter 2024 Financial Results and Provides Corporate Update Including New Clinical Results from MRT-6160 and MRT-2359 Programs

On March 20, 2025 Monte Rosa Therapeutics, Inc. (Nasdaq: GLUE), a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, reported a clinical update, business highlights, and financial results for the fourth quarter ended December 31, 2024 (Press release, Monte Rosa Therapeutics, MAR 20, 2025, View Source [SID1234651311]).

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"We continue to make excellent progress with our clinical and preclinical molecular glue degrader programs, targeting areas poorly addressed by conventional pharmaceutical approaches and with expansive therapeutic potential," said Markus Warmuth, M.D., Chief Executive Officer of Monte Rosa Therapeutics. "Today, we are pleased to report new clinical results from our two clinical programs. We believe the current results from our Phase 1 study of MRT-6160 are highly encouraging and demonstrate deep VAV1 degradation, biologically meaningful levels of ex vivo T and B cell functional inhibition, including inhibition of secretion of various cytokines following ex-vivo stimulation, and a highly favorable safety/tolerability profile. These data strengthen our conviction in the broad potential application of MRT-6160 as a novel treatment approach for immune-mediated diseases, and we look forward to rapidly advancing this program alongside our collaborators at Novartis."

Dr. Warmuth continued: "Our updated MRT-2359 results deepen our understanding of the drug’s clinical profile across several challenging-to-treat solid tumors. We have observed encouraging early signals of clinical response in heavily pretreated castration-resistant prostate cancer (CRPC), including a confirmed partial response and two patients with stable disease within the first three patients treated with MRT-2359/enzalutamide combination therapy. In light of these data, we plan to focus our ongoing MRT-2359 development efforts in CRPC, with the potential to expand this cohort to 20-30 patients, while deprioritizing other expansion arms except ER-positive breast cancer. We see CRPC as a hugely exciting opportunity for MRT-2359 with the added potential advantage for us of not having to identify biomarker positive patients, which would simplify our further clinical development. We expect to present further data from this program in the second half of the year. Furthermore, our early-stage pipeline is also making significant strides. Our NEK7 program is on track for an IND submission for MRT-8102 in the first half of this year, supported by GLP toxicology findings that demonstrate a considerable safety margin, with a more than 200-fold exposure margin over human efficacious doses in rats and non-human primates. We’re progressing our second generation, CNS-penetrant NEK7 program towards a 2026 IND submission and have released preclinical data demonstrating impressive levels of CNS activity in multiple species. Our ‘only-in-class’ CCNE1-directed MGD program represents a unique opportunity to directly target a previously undruggable but highly validated driver oncogene. Our AI/ML-powered QuEEN discovery engine has been highly productive, and we are advancing several programs as we seek to expand our portfolio of oral I&I drugs targeting pathways currently served only by injectable biologics or cell therapies."

RECENT HIGHLIGHTS

MRT-6160, VAV1-directed MGD for immune-mediated conditions

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Today, Monte Rosa announced clinical results from its MRT-6160 Phase 1, single ascending dose / multiple ascending dose (SAD/MAD) study. Details about the study, First-in-human Study of MRT-6160 in Healthy Subjects, can be found at clinicaltrials.gov under the identifier NCT06597799. The data presented includes the SAD portion of the study, which evaluated 5 dose cohorts, and the MAD portion of the study, which evaluated 3 dose cohorts. All cohorts were randomized and placebo controlled, and the study enrolled over 70 subjects in total. The study objectives were to evaluate safety and tolerability, pharmacokinetics, and pharmacodynamics, including VAV1 degradation and its impact on T and B cell function following ex vivo stimulation.
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Results demonstrated sustained, dose-dependent VAV1 degradation of greater than 90% in peripheral blood T cells after single and multiple dose administration. Similar results were observed in peripheral blood B cells. Sustained suppression of TCR-mediated T and B cell activation measured by CD69 was observed following single and multiple dose administration and ex vivo activation of whole blood. MRT-6160 also inhibited secretion of inflammatory cytokines, including IL-2, IFN-γ and IL-17A, by up to 99% from whole blood-derived T cells following ex vivo activation of TCR and demonstrated significant and sustained attenuation of IL-6 production across dose levels following B cell stimulation. The cytokine modulation profile observed in the clinical study closely aligned with preclinical studies that demonstrated the activity of MRT-6160 in various models of immune-mediated diseases.
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The administration of MRT-6160 was generally well-tolerated and there were no serious adverse effects observed. Treatment emergent adverse events were in general mild (82%) or moderate (18%) and self-limiting, and the overall frequency of treatment-related adverse effects was similar in the MRT-6160 and placebo groups.
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In summary, the current Phase 1 data and chronic toxicology package support a clear path into anticipated Phase 2 studies and broad potential applications in multiple immune-mediated diseases. Further development of MRT-6160 toward Phase 2 studies is ongoing, in collaboration with Novartis.
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In October, the Company announced a global exclusive development and commercialization license agreement with Novartis to advance VAV1 MGDs including MRT-6160. Under the terms of the agreement, Monte Rosa received a $150 million upfront payment. Monte Rosa is eligible to receive up to $2.1 billion in development, regulatory, and sales milestones, beginning upon initiation of Phase 2 studies, as well as tiered royalties on ex-U.S. net sales. Monte Rosa will co-fund any Phase 3 clinical development and will share any profits and losses associated with the manufacturing and commercialization of MRT-6160 in the U.S.

MRT-2359, GSPT1-directed MGD for MYC-driven solid tumors

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Today, Monte Rosa provided updated clinical results in its evaluation of safety, pharmacodynamics and clinical activity of MRT-2359 in various tumor types. Details about the study, Study of Oral MRT-2359 in Selected Cancer Patients, can be found at clinicaltrials.gov under the identifier NCT05546268.
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A total of 59 patients were dosed with MRT-2359 monotherapy at 6 dose levels across two dose schedules, 5 days on / 9 days off drug and 21 days on / 7 days off drug. Using the 5/9 dosing schedule, doses of 0.5 mg and 1 mg per day were generally well tolerated with mostly low-grade adverse events (AEs), while doses of 1.5 mg or higher were above the maximum tolerated dose (MTD) with thrombocytopenia being a dose-limiting toxicity (DLT). Using the 21/7 schedule, both the 0.5 and 0.75 mg doses were generally well tolerated with mostly low-grade AEs observed. The 0.5 mg dose using the 21/7 dose schedule was selected as the recommended phase 2 dose (RP2D). Optimal degradation (based on optimal PD modulation in preclinical studies) of approximately 60% was achieved in biomarker L-/N-MYC high tumor samples, as assessed by targeted mass spectrometry.
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While MRT-2359 demonstrated signals of activity in the dose escalation cohorts, the frequency of tumors expressing high levels of L-MYC or N-MYC in study patients was lower than expected compared to preclinical data across target populations, including non-small cell lung cancer (NCSLC), small cell lung cancer (SCLC), and high grade neuroendocrine (HG NE) tumors, resulting in lower than expected identification of biomarker-positive patients. Monte Rosa has determined this lower-than-expected biomarker positivity rate to be insufficient to support further development in lung cancers and neuroendocrine tumors through expansions cohorts. As such, the Company does not plan to conduct further studies in these indications and open the respective expansion cohorts. In summary, L-MYC and N-MYC expression in tumor tissue obtained at baseline or known L-MYC or N-MYC amplification in the absence of tissue was available in 48 patients. Of these, 37 patients were evaluable for response per RECIST 1.1. Of 13 patients determined to be biomarker positive, there was 1 confirmed partial response (PR), and 4 patients with stable disease (SD), for a disease control rate (DCR) of 38%. Of 24 biomarker negative patients (low L-MYC and N-MYC expression), there was 1 unconfirmed PR and 3 patients with SD, for a DCR of 17%.
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Evaluation of MRT-2359 activity in castration-resistant prostate cancer (CRPC) patients resistant to AR therapy, a patient group characterized by widespread expression of c-MYC, demonstrated encouraging early signals of clinical response, including a confirmed RECIST response, in the ongoing Phase 1/2 study. MRT-2359 is being dosed at 0.5 mg per day with a 21 days on drug, 7 days off drug dosing schedule, in combination with enzalutamide, a standard of care therapy for CRPC. As of a data cutoff of March 10, 2025, three CRPC patients were evaluable per RECIST 1.1 criteria. One patient had a confirmed partial response (tumor shrinkage of -57%) and two patients had stable disease. All 3 patients had mutations typically associated with resistance to AR antagonists including enzalutamide. PSA response was available for 2 patients showing 1 PSA response (-90%) in the patient with a confirmed PR. The safety profile observed has been generally favorable, with one case of grade 3 stomatitis being the only AE over grade 2. The Company is continuing to enroll and evaluate patients with CRPC, with the potential to expand enrollment to 20-30 patients if a positive efficacy signal continues to be observed, and expects to present additional results in H2 2025. The Company believes that the lack of need for biomarker-based patient selection in CRPC due to the widespread expression of c-MYC in this tumor type will facilitate future clinical development.
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The Company is continuing to enroll and evaluate patients with HR+ breast cancer and expects to present additional results for this cohort in H2 2025.

NEK7-directed MGDs for inflammatory and CNS diseases driven by IL-1β and the NLRP3 inflammasome

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Monte Rosa has successfully completed GLP tox studies for MRT-8102, a first-in-class NEK7-directed MGD for the treatment of inflammatory diseases driven by interleukin-1β (IL-1β) and the NLRP3 inflammasome, supporting a considerable safety margin. The no observed adverse effect level (NOAEL) was the highest dose tested, specifically 150 mg/kg/day in rats and 100 mg/kg/day in cynomolgus monkeys (cynos). The study demonstrated a greater than 200-fold exposure margin over the projected human efficacious dose in both species. No MRT-8102 related clinical signs, no changes in immunophenotyping (studied in cynos only), and no gross or clinical pathology findings were observed at any dose​ level.
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The Company is on track to submit an IND application for MRT-8102 in H1 2025. Following the IND submission, Monte Rosa plans to initiate a Phase 1 healthy volunteer study, and Phase 1 proof-of-concept studies in individuals with high levels of C-reactive protein (CRP) and in individuals with pericarditis. The Company is also evaluating future Phase 2 proof-of-concept studies in gout, pseudogout (calcium pyrophosphate deposition disease), and osteoarthritis.
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Monte Rosa has generated preclinical data with a second-generation NEK7-directed MGD optimized for CNS penetration. The compound has demonstrated potent systemic and CNS NEK7 degradation in cynos, and substantial reductions of inflammatory cytokines in an LPS-induced murine model of neuroinflammation. IND submission is expected in 2026.

Cyclin E1 and CDK2-directed MGD programs for treatment of solid tumors

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Monte Rosa continues to advance its cyclin E1 (CCNE1)- and CDK2-directed MGD programs for the treatment of CCNE1-amplified solid tumors and ER+ breast cancer. The company is benchmarking MGD molecules for both programs against each other in multiple preclinical models to determine the optimal MGD to advance to IND submission, which is expected in 2026.
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In December, at the 2024 San Antonio Breast Cancer Symposium, the Company presented preclinical data on the potential of its highly selective cyclin-dependent kinase 2 (CDK2)-directed molecular glue degrader to treat HR-positive/HER2-negative breast cancer. Data demonstrated deep tumor regression in preclinical models of HR-positive/HER2-negative breast cancer when combined with either a CDK4/6 inhibitor or a CDK4/6 inhibitor and endocrine therapy.
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In October, the Company presented preclinical data at the 36th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on the potential of its cyclin E1 (CCNE1)-directed MGDs, demonstrating that Monte Rosa MGDs degrade cyclin E1 with a high level of selectivity and induced tumor growth suppression and regression preferentially in CCNE1-amplified and over-expressing tumor cell lines and xenograft models. Cyclin E1 MGDs may represent a potential novel therapeutic approach by directly and selectively targeting a frequently amplified non-enzymatic driver oncogene relevant in multiple solid tumors.

QuEEN (Quantitative and Engineered Elimination of Neosubstrates) discovery engine

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Monte Rosa is advancing novel discovery programs for immunology and inflammation targets that the Company believes have the potential for highly differentiated, oral MGDs degrading undruggable targets in critical I&I pathways. These may include programs with the potential to improve upon the clinical profile of cell therapies such as CAR-T or biologics such as FcRn inhibitors.

ANTICIPATED UPCOMING MILESTONES AND DEVELOPMENT PRIORITIES

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Continue advancement of MRT-6160 through Phase 2 initiation, in collaboration with Novartis.
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Share additional MRT-2359 Phase 1/2 study data in CRPC patients resistant to AR therapy in H2 2025.
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Submit an IND application for MRT-8102 in H1 2025.
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Submit an IND application for the second generation NEK7-directed MGD with enhanced CNS penetration in 2026.
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Submit an IND application for a CDK2 and/or cyclin E1-directed MGD in 2026.

FOURTH QUARTER AND FULL YEAR 2024 FINANCIAL RESULTS

Collaboration revenue: Collaboration revenue for the fourth quarter of 2024 was $60.6 million and $75.6 million for the year ended December 31, 2024. The Company did not record collaboration revenue in 2023. Collaboration revenue represents amounts earned from our collaboration and license agreements with Roche and Novartis.

Research and Development (R&D) Expenses: R&D expenses for the fourth quarter of 2024 were $38.9 million, compared to $27.1 million for the fourth quarter of 2023, and $121.6 million for the year ended December 31, 2024, compared to $111.3 million for the year ended December 31, 2023. These increases were driven by the successful achievement of key milestones in our R&D organization, including the continuation of the MRT-2359 clinical study, the progression and growth of our preclinical pipeline, including research performed in connection with our collaboration with Roche, the advancement of MRT-6160 to enter the clinic, and the continued development of the Company’s QuEEN discovery engine. Non-cash stock-based compensation constituted $2.7 million of R&D expenses for Q4 2024, compared to $2.2 million in the same period in 2023, and $10.6 million and $8.9 million for the years ended December 31, 2024 and 2023, respectively.

General and Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2024 were $8.8 million compared to $7.7 million for the fourth quarter of 2023, and $35.2 million for the year ended December 31, 2024, compared to $32.0 million for the year ended December 31, 2023. The increase in G&A expenses was a result of increased headcount and expenses in support of the Company’s growth and operations. G&A expenses included non-cash stock-based compensation of $1.8 million for the fourth quarter of 2024 and 2023, and $7.5 million and $7.7 million for the years ended December 31, 2024 and 2023, respectively.

Net Income (Loss): Net income for the fourth quarter of 2024 was $13.4 million, compared to a net loss of $33.3 million for the fourth quarter of 2023, and net losses of $72.7 million for the year ended December 31, 2024, compared to $135.4 million for the year ended December 31, 2023.

Cash Position and Financial Guidance: Cash, cash equivalents, restricted cash, and marketable securities as of December 31, 2024, were $377 million, compared to cash, cash equivalents, restricted cash, and marketable securities of $247.1 million as of September 30, 2024. The increase of $129.9 million was primarily related to upfront payment received from Novartis in connection with our license agreement.

Based on current cash, cash equivalents, restricted cash, marketable securities, the Company expects its cash and cash equivalents to be sufficient to fund planned operations and capital expenditures into 2028.

RYBREVANT® (amivantamab-vmjw) plus LAZCLUZE™ (lazertinib) significantly outperforms standard of care in first-line EGFR-mutated lung cancer with compelling new data at ELCC 2025

On March 20, 2025 Johnson & Johnson (NYSE:JNJ) reported that new data from its industry-leading oncology pipeline will be presented at the 2025 European Lung Cancer Congress (ELCC), including overall survival (OS) results from the Phase 3 MARIPOSA study evaluating RYBREVANT (amivantamab-vmjw) plus LAZCLUZE (lazertinib) versus osimertinib in the first-line treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or L858R substitution mutations (Press release, Johnson & Johnson, MAR 20, 2025, View Source [SID1234651327]).

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"Patients with EGFR-mutated non-small cell lung cancer deserve to live longer and with more hope than current treatments provide," said Yusri Elsayed, M.D., M.H.Sc., Ph.D., Global Therapeutic Area Head, Oncology, Johnson & Johnson Innovative Medicine. "These newest and compelling overall survival data for first-line treatment with RYBREVANT and LAZCLUZE are fundamentally changing treatment discussions and transforming expectations for how long patients can live."

Additional presentations will feature the first data from the Phase 2 COCOON study, evaluating a simple to use and widely accessible dermatologic regimen given prophylactically to patients receiving the RYBREVANT combination, and new results from the Phase 2 PALOMA-2 study evaluating the feasibility of switching to subcutaneous (SC) amivantamab.

"We are redefining the way EGFR-mutated non-small cell lung cancer is treated with therapies that improve survival and give patients more time," said Henar Hevia, Ph.D., Senior Director, EMEA Therapeutic Area Lead, Oncology, Johnson & Johnson Innovative Medicine. "The chemotherapy-free regimen of RYBREVANT plus LAZCLUZE represents a major breakthrough, offering an innovative and life-extending option that can meaningfully impact lives."

ELCC 2025 Presentation Highlights:

OS results from the Phase 3 MARIPOSA study comparing RYBREVANT plus LAZCLUZE versus osimertinib in first-line EGFR-mutant advanced NSCLC (Proffered Paper Abstract #4O).
First presentation of data from the Phase 2 COCOON study evaluating an easy-to-use, readily available prophylactic regimen for the prevention of dermatologic reactions in the first-line treatment of EGFR-mutant advanced NSCLC in patients receiving RYBREVANT plus LAZCLUZE (Mini Oral Abstract #10MO).
Initial results from the Phase 2 PALOMA-2 study evaluating switching to SC amivantamab among patients benefitting from IV delivery in advanced EGFR-mutated NSCLC (Poster Abstract #58P).
Insights from the Phase 1/1b CHRYSALIS-2 study comparing RYBREVANT plus LAZCLUZE to EGFR tyrosine kinase inhibitor (TKI) monotherapy in a matched real-world cohort of atypical EGFR-mutated advanced NSCLC (Poster Abstract #59P).
Preliminary results from a Phase 1 study evaluating the safety and feasibility of JNJ-1900 (NBTXR3), a novel radioenhancer activated by radiation therapy, in combination with anti-PD1 treatment for patients with advanced solid tumors and lung metastases (Poster Abstract #255P).
A complete list of Johnson & Johnson-sponsored abstracts is available on JNJ.com.

About RYBREVANT

RYBREVANT (amivantamab-vmjw), a fully-human bispecific antibody targeting EGFR and MET with immune cell-directing activity, is approved in the U.S., Europe and other markets around the world as monotherapy for the treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.1 In the subcutaneous formulation, amivantamab is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE drug delivery technology.

RYBREVANT is approved in the U.S., Europe and other markets around the world in combination with chemotherapy (carboplatin and pemetrexed) for the first-line treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations, as detected by an FDA-approved test.

RYBREVANT is approved in the U.S., Europe and other markets around the world in combination with LAZCLUZE (lazertinib) for the first-line treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test.

RYBREVANT is approved in the U.S., Europe and other markets around the world in combination with chemotherapy (carboplatin-pemetrexed) for the treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or L858R substitution mutations, whose disease has progressed on or after treatment with an EGFR TKI.

In February 2025, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended the approval of SC amivantamab and LAZCLUZE in Europe for the first-line treatment of adult patients with advanced NSCLC with EGFR exon 19 deletions or exon 21 L858R substitution mutations, and as a monotherapy for the treatment of adult patients with advanced NSCLC with activating EGFR exon 20 insertion mutations after failure of platinum-based therapy.

The National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines) for NSCLC§ prefer next-generation sequencing–based strategies over polymerase chain reaction–based approaches for the detection of EGFR exon 20 insertion variants. The NCCN Guidelines include:

Amivantamab-vmjw (RYBREVANT) plus lazertinib (LAZCLUZE) as a Category 1 recommendation for first-line therapy in patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R mutations.2 †‡
Amivantamab-vmjw (RYBREVANT) plus chemotherapy as a Category 1 recommendation for patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R mutations who experienced disease progression after treatment with osimertinib.2 †‡
Amivantamab-vmjw (RYBREVANT) plus chemotherapy as a Category 1 recommendation for first-line therapy in treatment-naive patients with newly diagnosed advanced or metastatic EGFR exon 20 insertion mutation-positive advanced NSCLC.2 †‡
Amivantamab-vmjw (RYBREVANT) as a Category 2A recommendation for patients that have progressed on or after platinum-based chemotherapy with or without an immunotherapy and have EGFR exon 20 insertion mutation-positive NSCLC.2 †‡
RYBREVANT is being studied in multiple clinical trials in NSCLC, including:

The Phase 3 MARIPOSA (NCT04487080) study assessing RYBREVANT in combination with LAZCLUZE versus osimertinib and versus LAZCLUZE alone in the first-line treatment of patients with locally advanced or metastatic NSCLC with EGFR ex19del or substitution mutations.3
The Phase 3 MARIPOSA-2 (NCT04988295) study assessing the efficacy of RYBREVANT (with or without LAZCLUZE) and carboplatin-pemetrexed versus carboplatin-pemetrexed alone in patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or L858R substitution mutations after disease progression on or after osimertinib.4
The Phase 3 PAPILLON (NCT04538664) study assessing RYBREVANT in combination with carboplatin-pemetrexed versus chemotherapy alone in the first-line treatment of patients with advanced or metastatic NSCLC with EGFR exon 20 insertion mutations.5
The Phase 3 PALOMA-3 (NCT05388669) study assessing LAZCLUZE with subcutaneous (SC) amivantamab compared to RYBREVANT in patients with EGFR-mutated advanced or metastatic NSCLC.6
The Phase 2 PALOMA-2 (NCT05498428) study assessing SC amivantamab in patients with advanced or metastatic solid tumors including EGFR-mutated NSCLC.7
The Phase 1 PALOMA (NCT04606381) study assessing the feasibility of SC amivantamab based on safety and pharmacokinetics and to determine a dose, dose regimen and formulation for SC amivantamab delivery.8
The Phase 1 CHRYSALIS (NCT02609776) study evaluating RYBREVANT in patients with advanced NSCLC.9
The Phase 1/1b CHRYSALIS-2 (NCT04077463) study evaluating RYBREVANT in combination with LAZCLUZE and LAZCLUZE as a monotherapy in patients with advanced NSCLC with EGFR mutations.10
The Phase 1/2 METalmark (NCT05488314) study assessing RYBREVANT and capmatinib combination therapy in locally advanced or metastatic NSCLC.11
The Phase 1/2 swalloWTail (NCT06532032) study assessing RYBREVANT and docetaxel combination therapy in patients with metastatic NSCLC.12
The Phase 1/2 PolyDamas (NCT05908734) study assessing RYBREVANT and cetrelimab combination therapy in locally advanced or metastatic NSCLC.13
The Phase 2 SKIPPirr study (NCT05663866) exploring how to decrease the incidence and/or severity of first-dose infusion-related reactions with RYBREVANT in combination with LAZCLUZE in relapsed or refractory EGFR-mutated advanced or metastatic NSCLC.14
The Phase 2 COPERNICUS (NCT06667076) study combining developments in treatment administration and prophylactic supportive care in representative US patients with common EGFR-mutated NSCLC treated with SC amivantamab in combination with LAZCLUZE or chemotherapy.15
The Phase 2 COCOON (NCT06120140) study assessing the effectiveness of a proactive dermatologic management regimen given with first-line RYBREVANT and LAZCLUZE in patients with EGFR-mutated advanced NSCLC.16
For more information, visit: View Source

About LAZCLUZE

In 2018, Janssen Biotech, Inc., entered into a license and collaboration agreement with Yuhan Corporation for the development of LAZCLUZE (marketed as LECLAZA in South Korea). LAZCLUZE is an oral, third-generation, brain-penetrant EGFR TKI that targets both the T790M mutation and activating EGFR mutations while sparing wild-type EGFR. An analysis of the efficacy and safety of LAZCLUZE from the Phase 3 LASER301 study was published in The Journal of Clinical Oncology in 2023.17

About Non-Small Cell Lung Cancer

Worldwide, lung cancer is one of the most common cancers, with NSCLC making up 80 to 85 percent of all lung cancer cases.18,19 The main subtypes of NSCLC are adenocarcinoma, squamous cell carcinoma, and large cell carcinoma.20 Among the most common driver mutations in NSCLC are alterations in EGFR, which is a receptor tyrosine kinase controlling cell growth and division.21 EGFR mutations are present in 10 to 15 percent of Western patients with NSCLC with adenocarcinoma histology and occur in 40 to 50 percent of Asian patients.18,19,22,23,24,25 EGFR ex19del or EGFR L858R mutations are the most common EGFR mutations.26 The five-year survival rate for all people with advanced NSCLC and EGFR mutations treated with EGFR tyrosine kinase inhibitors (TKIs) is less than 20 percent.27,28 EGFR exon 20 insertion mutations are the third most prevalent activating EGFR mutation.29 Patients with EGFR exon 20 insertion mutations have a real-world five-year overall survival (OS) of eight percent in the frontline setting, which is worse than patients with EGFR ex19del or L858R mutations, who have a real-world five-year OS of 19 percent.30

IMPORTANT SAFETY INFORMATION1,31

WARNINGS AND PRECAUTIONS

Infusion-Related Reactions

RYBREVANT can cause infusion-related reactions (IRR) including anaphylaxis; signs and symptoms of IRR include dyspnea, flushing, fever, chills, nausea, chest discomfort, hypotension, and vomiting. The median time to IRR onset is approximately 1 hour.

RYBREVANT with LAZCLUZE

RYBREVANT in combination with LAZCLUZE can cause infusion-related reactions. In MARIPOSA (n=421), IRRs occurred in 63% of patients treated with RYBREVANT in combination with LAZCLUZE, including Grade 3 in 5% and Grade 4 in 1% of patients. The incidence of infusion modifications due to IRR was 54% of patients, and IRRs leading to dose reduction of RYBREVANT occurred in 0.7% of patients. Infusion-related reactions leading to permanent discontinuation of RYBREVANT occurred in 4.5% of patients receiving RYBREVANT in combination with LAZCLUZE.

RYBREVANT with Carboplatin and Pemetrexed

Based on the pooled safety population (n=281), IRR occurred in 50% of patients treated with RYBREVANT in combination with carboplatin and pemetrexed, including Grade 3 (3.2%) adverse reactions. The incidence of infusion modifications due to IRR was 46%, and 2.8% of patients permanently discontinued RYBREVANT due to IRR.

RYBREVANT as a Single Agent

In CHRYSALIS (n=302), IRR occurred in 66% of patients treated with RYBREVANT. Among patients receiving treatment on Week 1 Day 1, 65% experienced an IRR, while the incidence of IRR was 3.4% with the Day 2 infusion, 0.4% with the Week 2 infusion, and cumulatively 1.1% with subsequent infusions. Of the reported IRRs, 97% were Grade 1-2, 2.2% were Grade 3, and 0.4% were Grade 4. The median time to onset was 1 hour (range 0.1 to 18 hours) after start of infusion. The incidence of infusion modifications due to IRR was 62% and 1.3% of patients permanently discontinued RYBREVANT due to IRR.

Premedicate with antihistamines, antipyretics, and glucocorticoids and infuse RYBREVANT as recommended. Administer RYBREVANT via a peripheral line on Week 1 and Week 2 to reduce the risk of infusion-related reactions. Monitor patients for signs and symptoms of infusion reactions during RYBREVANT infusion in a setting where cardiopulmonary resuscitation medication and equipment are available. Interrupt infusion if IRR is suspected. Reduce the infusion rate or permanently discontinue RYBREVANT based on severity. If an anaphylactic reaction occurs, permanently discontinue RYBREVANT.

Interstitial Lung Disease/Pneumonitis

RYBREVANT can cause severe and fatal interstitial lung disease (ILD)/pneumonitis.

RYBREVANT with LAZCLUZE

In MARIPOSA, ILD/pneumonitis occurred in 3.1% of patients treated with RYBREVANT in combination with LAZCLUZE, including Grade 3 in 1.0% and Grade 4 in 0.2% of patients. There was one fatal case (0.2%) of ILD/pneumonitis and 2.9% of patients permanently discontinued RYBREVANT and LAZCLUZE due to ILD/pneumonitis.

RYBREVANT with Carboplatin and Pemetrexed

Based on the pooled safety population, ILD/pneumonitis occurred in 2.1% treated with RYBREVANT in combination with carboplatin and pemetrexed with 1.8% of patients experiencing Grade 3 ILD/pneumonitis. 2.1% discontinued RYBREVANT due to ILD/pneumonitis.

RYBREVANT as a Single Agent

In CHRYSALIS, ILD/pneumonitis occurred in 3.3% of patients treated with RYBREVANT, with 0.7% of patients experiencing Grade 3 ILD/pneumonitis. Three patients (1%) permanently discontinued RYBREVANT due to ILD/pneumonitis.

Monitor patients for new or worsening symptoms indicative of ILD/pneumonitis (e.g., dyspnea, cough, fever). For patients receiving RYBREVANT in combination with LAZCLUZE, immediately withhold both drugs in patients with suspected ILD/pneumonitis and permanently discontinue if ILD/pneumonitis is confirmed. For patients receiving RYBREVANT as a single agent or in combination with carboplatin and pemetrexed, immediately withhold RYBREVANT in patients with suspected ILD/pneumonitis and permanently discontinue if ILD/pneumonitis is confirmed.

Venous Thromboembolic (VTE) Events with Concomitant Use of RYBREVANT and LAZCLUZE

RYBREVANT in combination with LAZCLUZE can cause serious and fatal venous thromboembolic (VTE) events, including deep vein thrombosis and pulmonary embolism. The majority of these events occurred during the first four months of therapy.

In MARIPOSA, VTEs occurred in 36% of patients receiving RYBREVANT in combination with LAZCLUZE, including Grade 3 in 10% and Grade 4 in 0.5% of patients. On-study VTEs occurred in 1.2% of patients (n=5) while receiving anticoagulation therapy. There were two fatal cases of VTE (0.5%), 9% of patients had VTE leading to dose interruptions of RYBREVANT, and 7% of patients had VTE leading to dose interruptions of LAZCLUZE; 1% of patients had VTE leading to dose reductions of RYBREVANT, and 0.5% of patients had VTE leading to dose reductions of LAZCLUZE; 3.1% of patients had VTE leading to permanent discontinuation of RYBREVANT, and 1.9% of patients had VTE leading to permanent discontinuation of LAZCLUZE. The median time to onset of VTEs was 84 days (range: 6 to 777).

Administer prophylactic anticoagulation for the first four months of treatment. The use of Vitamin K antagonists is not recommended. Monitor for signs and symptoms of VTE events and treat as medically appropriate.

Withhold RYBREVANT and LAZCLUZE based on severity. Once anticoagulant treatment has been initiated, resume RYBREVANT and LAZCLUZE at the same dose level at the discretion of the healthcare provider. In the event of VTE recurrence despite therapeutic anticoagulation, permanently discontinue RYBREVANT and continue treatment with LAZCLUZE at the same dose level at the discretion of the healthcare provider.

Dermatologic Adverse Reactions

RYBREVANT can cause severe rash including toxic epidermal necrolysis (TEN), dermatitis acneiform, pruritus, and dry skin.

RYBREVANT with LAZCLUZE

In MARIPOSA, rash occurred in 86% of patients treated with RYBREVANT in combination with LAZCLUZE, including Grade 3 in 26% of patients. The median time to onset of rash was 14 days (range: 1 to 556 days). Rash leading to dose interruptions occurred in 37% of patients for RYBREVANT and 30% for LAZCLUZE, rash leading to dose reductions occurred in 23% of patients for RYBREVANT and 19% for LAZCLUZE, and rash leading to permanent discontinuation occurred in 5% of patients for RYBREVANT and 1.7% for LAZCLUZE.

RYBREVANT with Carboplatin and Pemetrexed

Based on the pooled safety population, rash occurred in 82% of patients treated with RYBREVANT in combination with carboplatin and pemetrexed, including Grade 3 (15%) adverse reactions. Rash leading to dose reductions occurred in 14% of patients, and 2.5% permanently discontinued RYBREVANT and 3.1% discontinued pemetrexed.

RYBREVANT as a Single Agent

In CHRYSALIS, rash occurred in 74% of patients treated with RYBREVANT as a single agent, including Grade 3 rash in 3.3% of patients. The median time to onset of rash was 14 days (range: 1 to 276 days). Rash leading to dose reduction occurred in 5% of patients, and RYBREVANT was permanently discontinued due to rash in 0.7% of patients.

Toxic epidermal necrolysis occurred in one patient (0.3%) treated with RYBREVANT as a single agent.

Instruct patients to limit sun exposure during and for 2 months after treatment with RYBREVANT or LAZCLUZE in combination with RYBREVANT. Advise patients to wear protective clothing and use broad-spectrum UVA/UVB sunscreen. Alcohol-free (e.g., isopropanol-free, ethanol-free) emollient cream is recommended for dry skin.

When initiating RYBREVANT treatment with or without LAZCLUZE, administer alcohol-free emollient cream to reduce the risk of dermatologic adverse reactions. Consider prophylactic measures (e.g. use of oral antibiotics) to reduce the risk of dermatologic reactions. If skin reactions develop, start topical corticosteroids and topical and/or oral antibiotics. For Grade 3 reactions, add oral steroids and consider dermatologic consultation. Promptly refer patients presenting with severe rash, atypical appearance or distribution, or lack of improvement within 2 weeks to a dermatologist. For patients receiving RYBREVANT in combination with LAZCLUZE, withhold, reduce the dose, or permanently discontinue both drugs based on severity. For patients receiving RYBREVANT as a single agent or in combination with carboplatin and pemetrexed, withhold, dose reduce or permanently discontinue RYBREVANT based on severity.

Ocular Toxicity

RYBREVANT can cause ocular toxicity including keratitis, blepharitis, dry eye symptoms, conjunctival redness, blurred vision, visual impairment, ocular itching, eye pruritus, and uveitis.

RYBREVANT with LAZCLUZE

In MARIPOSA, ocular toxicity occurred in 16% of patients treated with RYBREVANT in combination with LAZCLUZE, including Grade 3 or 4 ocular toxicity in 0.7% of patients. Withhold, reduce the dose, or permanently discontinue RYBREVANT and continue LAZCLUZE based on severity.

RYBREVANT with Carboplatin and Pemetrexed

Based on the pooled safety population, ocular toxicity occurred in 16% of patients treated with RYBREVANT in combination with carboplatin and pemetrexed. All events were Grade 1 or 2.

RYBREVANT as a Single Agent

In CHRYSALIS, keratitis occurred in 0.7% and uveitis occurred in 0.3% of patients treated with RYBREVANT. All events were Grade 1-2.

Promptly refer patients with new or worsening eye symptoms to an ophthalmologist. Withhold, reduce the dose, or permanently discontinue RYBREVANT based on severity.

Embryo-Fetal Toxicity

Based on its mechanism of action and findings from animal models, RYBREVANT and LAZCLUZE can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential of the potential risk to the fetus.

Advise female patients of reproductive potential to use effective contraception during treatment and for 3 months after the last dose of RYBREVANT.

Advise females of reproductive potential to use effective contraception during treatment with LAZCLUZE and for 3 weeks after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with LAZCLUZE and for 3 weeks after the last dose.

Adverse Reactions

RYBREVANT with LAZCLUZE

For the 421 patients in the MARIPOSA clinical trial who received RYBREVANT in combination with LAZCLUZE, the most common adverse reactions (≥20%) were rash (86%), nail toxicity (71%), infusion-related reactions (RYBREVANT, 63%), musculoskeletal pain (47%), stomatitis (43%), edema (43%), VTE (36%), paresthesia (35%), fatigue (32%), diarrhea (31%), constipation (29%), COVID-19 (26%), hemorrhage (25%), dry skin (25%), decreased appetite (24%), pruritus (24%), nausea (21%), and ocular toxicity (16%). The most common Grade 3 or 4 laboratory abnormalities (≥2%) were decreased albumin (8%), decreased sodium (7%), increased ALT (7%), decreased potassium (5%), decreased hemoglobin (3.8%), increased AST (3.8%), increased GGT (2.6%), and increased magnesium (2.6%).

Serious adverse reactions occurred in 49% of patients who received RYBREVANT in combination with LAZCLUZE. Serious adverse reactions occurring in ≥2% of patients included VTE (11%), pneumonia (4%), ILD/pneumonitis and rash (2.9% each), COVID-19 (2.4%), and pleural effusion and infusion-related reaction (RYBREVANT) (2.1% each). Fatal adverse reactions occurred in 7% of patients who received RYBREVANT in combination with LAZCLUZE due to death not otherwise specified (1.2%); sepsis and respiratory failure (1% each); pneumonia, myocardial infarction, and sudden death (0.7% each); cerebral infarction, pulmonary embolism (PE), and COVID-19 infection (0.5% each); and ILD/pneumonitis, acute respiratory distress syndrome (ARDS), and cardiopulmonary arrest (0.2% each).

RYBREVANT with Carboplatin and Pemetrexed

For the 130 patients in the MARIPOSA-2 clinical trial who received RYBREVANT in combination with carboplatin and pemetrexed, the most common adverse reactions (≥20%) were rash (72%), infusion-related reactions (59%), fatigue (51%), nail toxicity (45%), nausea (45%), constipation (39%), edema (36%), stomatitis (35%), decreased appetite (31%), musculoskeletal pain (30%), vomiting (25%), and COVID-19 (21%). The most common Grade 3 to 4 laboratory abnormalities (≥2%) were decreased neutrophils (49%), decreased white blood cells (42%), decreased lymphocytes (28%), decreased platelets (17%), decreased hemoglobin (12%), decreased potassium (11%), decreased sodium (11%), increased alanine aminotransferase (3.9%), decreased albumin (3.8%), and increased gamma-glutamyl transferase (3.1%).

In MARIPOSA-2, serious adverse reactions occurred in 32% of patients who received RYBREVANT in combination with carboplatin and pemetrexed. Serious adverse reactions in >2% of patients included dyspnea (3.1%), thrombocytopenia (3.1%), sepsis (2.3%), and pulmonary embolism (2.3%). Fatal adverse reactions occurred in 2.3% of patients who received RYBREVANT in combination with carboplatin and pemetrexed; these included respiratory failure, sepsis, and ventricular fibrillation (0.8% each).

For the 151 patients in the PAPILLON clinical trial who received RYBREVANT in combination with carboplatin and pemetrexed, the most common adverse reactions (≥20%) were rash (90%), nail toxicity (62%), stomatitis (43%), infusion-related reaction (42%), fatigue (42%), edema (40%), constipation (40%), decreased appetite (36%), nausea (36%), COVID-19 (24%), diarrhea (21%), and vomiting (21%). The most common Grade 3 to 4 laboratory abnormalities (≥2%) were decreased albumin (7%), increased alanine aminotransferase (4%), increased gamma-glutamyl transferase (4%), decreased sodium (7%), decreased potassium (11%), decreased magnesium (2%), and decreases in white blood cells (17%), hemoglobin (11%), neutrophils (36%), platelets (10%), and lymphocytes (11%).

In PAPILLON, serious adverse reactions occurred in 37% of patients who received RYBREVANT in combination with carboplatin and pemetrexed. Serious adverse reactions in ≥2% of patients included rash, pneumonia, ILD, pulmonary embolism, vomiting, and COVID-19. Fatal adverse reactions occurred in 7 patients (4.6%) due to pneumonia, cerebrovascular accident, cardio-respiratory arrest, COVID-19, sepsis, and death not otherwise specified.

RYBREVANT as a Single Agent

For the 129 patients in the CHRYSALIS clinical trial who received RYBREVANT as a single agent, the most common adverse reactions (≥20%) were rash (84%), IRR (64%), paronychia (50%), musculoskeletal pain (47%), dyspnea (37%), nausea (36%), fatigue (33%), edema (27%), stomatitis (26%), cough (25%), constipation (23%), and vomiting (22%). The most common Grade 3 to 4 laboratory abnormalities (≥2%) were decreased lymphocytes (8%), decreased albumin (8%), decreased phosphate (8%), decreased potassium (6%), increased alkaline phosphatase (4.8%), increased glucose (4%), increased gamma-glutamyl transferase (4%), and decreased sodium (4%).

Serious adverse reactions occurred in 30% of patients who received RYBREVANT. Serious adverse reactions in ≥2% of patients included pulmonary embolism, pneumonitis/ILD, dyspnea, musculoskeletal pain, pneumonia, and muscular weakness. Fatal adverse reactions occurred in 2 patients (1.5%) due to pneumonia and 1 patient (0.8%) due to sudden death.

LAZCLUZE Drug Interactions

Avoid concomitant use of LAZCLUZE with strong and moderate CYP3A4 inducers. Consider an alternate concomitant medication with no potential to induce CYP3A4.

Monitor for adverse reactions associated with a CYP3A4 or BCRP substrate where minimal concentration changes may lead to serious adverse reactions, as recommended in the approved product labeling for the CYP3A4 or BCRP substrate.

Santo Therapeutics Begins Clinical Trial Targeting NF2-Related Tumours

On March 20, 2025 Santo Therapeutics, a biotechnology company focused on advancing gene therapies, reported the initiation of its first Investigator-Initiated Clinical Trial (IIT) in China for ST002. Santo’s ST002 is an innovative in vivo lentiviral gene therapy designed to treat tumours with NF2 gene mutations. This marks a major milestone as ST002 is the world’s first gene therapy targeting NF2 mutation-driven tumours to enter clinical trials.

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"We are excited to start this groundbreaking clinical trial," said Dr. Luke Zhou, CEO of Santo Therapeutics. "ST002 is the first gene therapy specifically targeting NF2-related schwannomatosis (NF2-SWN), and with our proprietary platform, we are introducing a mechanism that precisely targets both the tumour and its environment, providing a more effective solution for NF2-related tumours."

Nick Rodgers, Chairman of the Advisory Board of Santo, commented, "Currently, there is no cure for NF2 disease. Gene therapy is the only promising solution to offer a cure, and we are eager to see the potential of ST002. Having spent months in pre-clinical testing we are pleased to be working with The Cancer Institute and Hospital, Chinese Academy of Medical Sciences to validate our therapy in humans."

The first-in-human IIT for ST002 is now underway, with initial clinical data expected to be available in the second half of 2025. In preclinical studies, ST002 demonstrated notable anti-tumour effects in NF2 patient-derived-xenograft (PDX) models, significantly inhibiting tumour growth and showing high specificity and durability.

"We are excited to advance ST002 to the clinical stage and bring hope to patients suffering from tumours with NF2 mutations," Dr. Zhou added. "This therapy holds the potential to not only treat NF2-related tumours but also open new avenues for treating other rare genetic conditions. We look forward to sharing our progress and early results in the coming months."

About ST002
ST002 is an innovative lentiviral gene therapy that delivers precise genetic material directly to the tumour site. By targeting the tumour micro-environment and restoring NF2 gene function, ST002 inhibits tumour growth by blocking abnormal Schwann cell. As an off-the-shelf product, ST002 offers a cost-effective, scalable treatment option for patients with schwannoma and other NF2 related tumours.

(Press release, Santo Therapeutics, MAR 20, 2025, View Source [SID1234663856])