RS Oncology Announces Positive Data from a Phase 1 Clinical Trial of RSO-021, a First-in-Class Therapeutic for Malignant Pleural Mesothelioma

On June 3, 2024 RS Oncology, a privately held biopharmaceutical company focusing on novel treatments for rare and aggressive cancers reported positive results from its Phase 1 study in patients with Malignant Pleural Mesothelioma (MPM) with Malignant Pleural Effusion (MPE) or MPE associated with other solid tumors at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, RS Oncology, JUN 3, 2024, View Source [SID1234644050]).

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The Phase 1 data was presented by Professor Dean Fennell, MRCP, FRCP (MD/PhD) on behalf of all the MITOPE investigators at an oral presentation session. In the 15 recruited patients safety data demonstrated weekly treatment of RSO-021 was well tolerated at 90 mg. The pharmacokinetic data showed minimal systemic exposure to RSO-021 after intrapleural administration. Efficacy data showed a long-term partial response in one patient as well as encouraging survival in 7 of the 10 evaluable patients. In addition to responses in mesothelioma, the drug showed promising responses in non-target lesions and other cancers with metastatic disease to the lung.

"The MITOPE trial would not have been possible without the support of the outstanding investigation teams throughout the UK who are dedicated to the treatment of patients with mesothelioma. We thank all of the MITOPE trial participants and their supportive families, and look forward to hearing about their continued benefit," said George Naumov, PhD, RS Oncology Chief Operations Officer.

"RSO-021 represents a new class of drugs with a first-in-class anticancer mechanism. The safety and efficacy observed in the Phase 1 trial is supported by strong pre-clinical rationale" said Brian Cunniff, PhD, Chief Science Officer for RS Oncology.

MPM is a rare and aggressive form of cancer that typically develops years after asbestos inhalation and/or exposure. Most cases (70%) originate in the pleura, but it can also be found the peritoneum and the pericardium. A mesothelioma prognosis remains poor with a shorter life expectancy and decreased quality of life.

Phase 2 exploration of this novel agent is ongoing at two doses, as a single agent and in combination with chemotherapy. Clinical trial information: NCT05278975.

About RS0-021

RSO-021 is a naturally occurring, sulfur-rich, cyclic oligopeptide of the thiopeptide class, which covalently inactivates PRX3, leading to catastrophic oxidative stress and cell death.

The oral and poster presentations will be available for viewing on RSOncology.com.

About the MITOPE study

MITOPE is an open-label, non-randomized, multicenter, translational Phase 1/2 dose-escalation and expansion study. It is designed to determine the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of RSO-021 after intrapleural (IP) administration in patients with MPE associated with either MPM or other solid tumors.

Fapon Biopharma Announces a Safer Immunotherapy for Cancers

On June 3, 2024 Fapon Biopharma, an innovator in developing therapeutic antibodies and fusion proteins, reported an immunocytokine with modified IL-10M fused to anti-PD-1 antibody, codenamed FP008 at Bio International Convention 2024, which is taking place here in San Diego during June 3-6 (Press release, Fapon Biopharma, JUN 3, 2024, View Source [SID1234644049]). This fusion protein provides a promising therapeutic strategy for solid tumors refractory to anti-PD-1 therapy. The groundbreaking announcement is a testimony to Fapon Biopharma’s strength in developing anticancer drugs.

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FP008 is a patent-pending drug candidate developed by Fapon Biopharma using cutting-edge antibody technology. It stands out as a first-in-class biologic candidate combining IL-10M (an attenuated IL-10 monomer variant) and an anti-PD-1 antibody, providing a targeted therapy with enhanced efficacy while significantly reducing the challenging toxicity associated with wtIL-10. This is a critical advancement in the field. FP008 not only reduces the apoptosis of the exhausted CD8+ T cells and restores their activity in vitro, but also significantly increases the level of CD8+ T cells within the tumor microenvironment, exhibiting a potent anti-tumor effect in vivo. It exhibited good druggability and was well tolerated in a toxicity study of up to 10 mpk in cynomolgus monkeys. As a result, FP008 is a viable drug candidate.

FP008 is poised to have a substantial impact on immunotherapy for cancer in patients who do not respond to existing treatments. The company aims to present it to a global audience and seek partners to drive its development for the benefit of cancer patients around the world eventually.

Fapon Biopharma plans to submit an IND application for FP008 to the FDA in early 2025. The company remains committed to developing more effective and more accessible therapeutics with consistent innovation and collaboration.

STORM Therapeutics Presented Interim Phase 1 Clinical Data on its METTL3 RNA Methyltransferase Inhibitor STC-15 at ASCO 2024

On June 3, 2024 STORM Therapeutics Ltd. (STORM), the clinical stage company pioneering cellular reprogramming through RNA modifications to treat disease, reported that interim Phase 1 clinical data from its first-in-class lead product, STC-15, was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, held in Chicago, US (Press release, STORM Therapeutics, JUN 3, 2024, View Source [SID1234644048]).

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The presented poster, entitled, ‘Phase 1 Dose Escalation and Cohort Expansion Study Evaluating Safety, PK, PD and Clinical Activity of STC-15, a METTL3 Inhibitor in Patients with Advanced Malignancies’, highlighted recent findings on STORM’s lead product, STC-15, a potent and selective METTL3 inhibitor and the first molecule specifically targeting an RNA methyltransferase enzyme to enter clinical development.

The study was conducted to evaluate the safety, pharmacokinetics, target modulation and clinical activity of STC-15 in patients with advanced malignancies, with the aim of establishing a recommended Phase 2 dose escalation to support future clinical development. The study enrolled 33 patients across five dose escalation cohorts ranging from 60mg to 200mg and explored daily and thrice weekly oral dosing regimens. Data presented at ASCO (Free ASCO Whitepaper) from 33 patients is outlined below:

STC-15 is well tolerated across the pharmacologically active dose range
Treatment emergent adverse events (TEAEs) were manageable: common related adverse events were thrombocytopenia, rash, and pruritus
As of April 15, 2024, 27 patients were evaluable for response assessment with at least 1 on-treatment scan. Clinical activity was observed, comprising of;
– 11% Overall Response Rate (3 partial responses)
– 63% Disease Control Rate (14 stable disease, 3 partial responses)
PK simulations and safety data support oral administration of STC-15 three times a week (TIW)
Significant reductions in methylated polyA-RNA in blood samples from all cohorts demonstrated METTL3 target engagement and rapid PD effect
Additional PD biomarker analysis indicated activation of interferon signaling and innate immune responses, as measured by RNA transcript analysis in blood cells from treated patients
Recommended Phase 2 doses were identified as between 60mg and 200mg TIW
Advancement of STC-15 into combination studies with checkpoint inhibitors is expected to commence in 2024
Jerry McMahon, Chief Executive Officer of Storm Therapeutics, said: "This Phase 1 data, to date, demonstrates the encouraging potential of STC-15 and its ability to impact a wide range of cancers. STC-15 is showing excellent safety and pharmacologic properties as it advances further into clinical development. As part of STORM’s evolving strategy, we are expanding our studies and structuring clinical trial designs to include patients with cancers such as lung, melanoma, head and neck, and endometrial cancer in the future, and we look forward to providing further updates in due course."

Josi Holz, Chief Medical Officer of STORM Therapeutics, commented: "These data signify great progress for STORM as we continue to develop our pipeline of RNA modifying enzyme inhibitors. We are pleased to be presenting data that demonstrates the potential of STC-15 and its promising signs of tolerability and clinical activity. We look forward to completing the current Phase 1 study and including the addition of checkpoint inhibitors."

Justin Moser, Principal Investigator at HonorHealth Research Institute and First Author, remarked: "These new data presented at ASCO (Free ASCO Whitepaper) on STC-15 are promising and support future clinical studies with this novel agent and new mechanism. METTL3 inhibition has demonstrated early clinical activity and represents a new way to activate the innate immune system with the potential to treat cancer in patients across a range of tumor types."

Abstracts are available on the ASCO (Free ASCO Whitepaper) program guide here, and on the STORM website here.

STC-15 is currently under evaluation for the treatment of patients with advanced solid tumors in a first-in-human, Phase 1 clinical study with ongoing with cohorts being expanded to further evaluate safety, food effect, PK/PD, clinical activity, with the goal of determining optimized active doses. Details of the study can be found on clinicaltrials.gov under the identifier NCT05584111.

NeoImmuneTech’s New Data at ASCO 2024 Highlights Enhanced Benefits of Combining NT-I7 with Checkpoint Inhibitors (CPI) in Immuno-Oncology

On June 3, 2024 NeoImmuneTech, Inc. (NIT), a T cell-focused therapeutics company, reported promising interim results of its major clinical trial NIT-110 of NT-I7 (efineptakin alfa), at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) global meeting held in Chicago, from May 31 to June 4 (Press release, NeoImmuneTech, JUN 3, 2024, View Source [SID1234644047]). The company also presented two additional posters at the conference.

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NIT-110 is a foundational clinical trial for NT-I7 that aims to confirm the safety and efficacy of combining NT-I7 with pembrolizumab (Keytruda) in solid tumors. The data presented at ASCO (Free ASCO Whitepaper) 2024 confirmed that the combination is safe and well-tolerated.

Key findings from NIT-110 include:

Pancreatic cancer – Data showed a median overall survival (mOS) of 11.1 months among the 48 pancreatic cancer patients included in the study. The mOS for pancreatic cancer patients who have received a second-line standard of care treatment is currently known to be 6.1 months[1]. This mOS improvement is particularly noteworthy considering that 93.75% of the patients are receiving the combination treatment as third-line or beyond.
MSS colorectal cancer -The mOS for the 50 microsatellite-stable (MSS) colorectal cancer patients was 13.2 months. The mOS for the current standard of care treatment is 10.8 months[2].
These results underscore the improved efficacy of the NT-I7 and Keytruda combination over existing standard of care treatments.

A separate poster from trial NIT-110 revealed a correlative analysis identifying a potentially predictive biomarker. These biomarkers may help identify patient populations more likely to benefit clinically from the NT-I7 and Keytruda combination. NeoImmuneTech is committed to further biomarker validation to enhance clinical outcomes.

Additionally, a preclinical study presented at ASCO (Free ASCO Whitepaper) 2024 highlighted the combination of NT-I7 with an oncolytic virus (ZIKV) in a glioblastoma animal model. Results demonstrated a significant increase in tumor specific CD8 T cells in the tumor microenvironment, leading to improved survival rates. Furthermore, 80% of cases in the experimental group combining NT-I7 with immune checkpoint inhibitors resulted in complete tumor eradication. NT-I7 continues to show clinical benefits driven by T cell amplification when combined with immune therapies.

NeoImmuneTech’s CEO, Luke Oh, PhD, said: "We are very encouraged by the improved clinical efficacy over standard of care in pancreatic and colorectal cancer, a notoriously difficult to treat cancer. These findings confirm our promising preclinical data and open new pathways to further enhance clinical outcomes. We are actively discussing the next steps with Merck. The results presented at ASCO (Free ASCO Whitepaper) 2024 confirm our strategy to continue to develop NT-I7 in the larger and high-potential immuno-oncology market."

Posters references:

Abstract #

Track

Poster board #

Title

2621 (Link)

Developmental Therapeutics -Immunotherapy

100

A Phase 2a study of NT-I7 (efineptakin alfa), a long-acting IL-7, and pembrolizumab to evaluate efficacy, including overall survival, in hard-to-treat MSS-CRC and PDAC gastrointestinal tumors

2563

(Link)

Developmental Therapeutics -Immunotherapy

42

Investigation of a potential protein biomarker signature that may predict clinical benefit of NT-I7 and pembrolizumab in patients with cold gastrointestinal tumors

2043

(Link)

Central Nervous System Tumors

342

Expand and pull: a new treatment paradigm for glioblastoma using a long-acting recombinant interleukin-7 and oncolytic viral therapy

About Study NIT-110
NIT-110 is an open label Phase 2a clinical trial supported by Merck that aims to confirm the efficacy of combining NT-I7 with Keytruda in two solid tumors. Early results in 2022 had confirmed the efficacy of the combination in pancreatic cancer and MSS colorectal cancer patients, who are known to be unresponsive to immune checkpoint inhibitors alone. Consequently, 24 and 25 additional patients were recruited for each group, respectively, bringing the total to 48 pancreatic cancer patients and 50 MSS colorectal cancer patients currently undergoing clinical trials. Results presented at ASCO (Free ASCO Whitepaper) 2024 provide an updated analysis including the original and expansion cohorts.

About NT-I7 (efineptakin alfa) (rhIL-7-hyFc)

NT-I7 (efineptakin alfa) is the only clinical-stage long-acting human IL-7, and is being developed in oncologic and immunologic indications, where T cell amplification and increased functionality may provide clinical benefit. IL-7 is a fundamental cytokine for naïve and memory T cell development and for sustaining immune response to chronic antigens (as in cancer) or foreign antigens (as in infectious diseases). NT-I7 exhibits favorable PK/PD and safety profiles, making it an ideal combination partner. NT-I7 is being studied in multiple clinical trials in solid tumors and as vaccine adjuvant. Studies are being planned for testing in hematologic malignancies, additional solid tumors and other immunology-focused indications.

TECVAYLI® (teclistamab-cqyv) shows sustained deep and durable responses in patients with relapsed or refractory multiple myeloma

On June 3, 2024 Johnson & Johnson reported longer-term data from the pivotal Phase 1/2 MajesTEC-1 study of TECVAYLI (teclistamab-cqyv) showing deep and durable responses in patients with relapsed or refractory multiple myeloma (RRMM) who are triple-class exposed (TCE)a and who previously received three or more prior lines of therapy, including in patients who switched to less frequent dosing (Abstract #7540) (Press release, Johnson & Johnson, JUN 3, 2024, View Source [SID1234644046]). These data were featured at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in a poster presentation.1

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Additional presentations highlight the potential for outpatient step-up administration with prophylactic tocilizumab from the MajesTEC-1 study (Abstract #7517) and the first-in-class Phase 2 OPTec study (Abstract #7528), as well as first results from the subgroup analysis of patients with high-risk (HR) features that will be presented at the 2024 European Hematology Association (EHA) (Free EHA Whitepaper) Congress (Abstract #923).2,3,4 The safety run-in MajesTEC-7 study in frontline TECVAYLI administration (Abstract #7506) will also be presented at ASCO (Free ASCO Whitepaper).4

"With the longest follow-up of any bispecific antibody, teclistamab demonstrates continued deep and durable responses observed in patients with relapsed or refractory multiple myeloma who have limited treatment options," said Niels van de Donk, M.D., Professor of Hematology at Amsterdam University Medical Centers, and principal study investigator.* "The results of the MajesTEC-1 study indicate the potential of teclistamab to transform the treatment paradigm, and clinical studies are investigating whether teclistamab may be a pivotal advancement for improved care and management in the broader patient population."

Results from the MajesTEC-1 study show that, at a median follow-up of 30.4 months, patients treated with TECVAYLI at the recommended Phase 2 dose (RP2D)b (n=165) demonstrated an overall response rate (ORR) of 63 percent, with responses continuing to deepen and 46 percent of patients achieving a complete response (CR) or better.1 For patients with a CR or better, mDOR, mPFS, and mOS were not yet reached, and estimated 30-month DOR, PFS, and OS rates were 61, 61 and 74 percent, respectively.1 Patients who achieved a partial response or better after a minimum of four cycles of therapy (Phase 1), or maintained a CR or better for a minimum of six months (Phase 2) per protocol, had the option to switch to biweekly dosing (every two weeks) (Q2W).1 Additionally, 37 out of 38 patients who switched to Q2W dosing maintained responses.1

The safety profile remained consistent, with a notable decrease in new onset of severe infections over time.1 Adverse events (AEs) included neutropenia (any grade, 72 percent; grade 3/4, 66 percent), anemia (any grade, 55 percent; grade 3/4, 38 percent), thrombocytopenia (any grade, 42 percent; grade 3/4, 23 percent), lymphopenia (any grade, 36 percent; grade 3/4, 35 percent), and infections (any grade, 79 percent; grade 3/4, 55 percent).1 Of 22 grade 5 infections, 18 were due to COVID-19.1 The decrease in new-onset grade 3 or greater infections may be due to switching to Q2W dosing or other factors such as implementing the use of intravenous immunoglobulin.1

"Over the past two years, TECVAYLI has helped over 10,000 patients with relapsed or refractory multiple myeloma," said Rachel Kobos, M.D., Vice President, Oncology Research & Development, Johnson & Johnson Innovative Medicine. "Through robust clinical data and real-world evidence, and by leveraging our team’s expertise, we’re working relentlessly to address unmet needs for patients with myeloma and drive the development of new treatment options for use across the treatment paradigm, including in the frontline setting."

TECVAYLI studies investigate outpatient administration in patients with RRMM, examining a more convenient approach to treatment, including in a community setting

Extended follow-up of patients from a MajesTEC-1 cohort, investigating the prophylactic use of tocilizumab for the reduction of cytokine release syndrome (CRS) in patients treated with TECVAYLI, were also presented at ASCO (Free ASCO Whitepaper) in an oral presentation (Abstract #7517).2 Results show a single dose of tocilizumab before TECVAYLI in patients with RRMM (n=24) reduced the incidence of CRS with a 65 percent relative reduction versus the overall MajesTEC-1 population.2 This approach is continuing to be evaluated in the first-in-class Phase 2, multicenter, prospective OPTec study of TECVAYLI in the community setting, presented as a poster presentation (Abstract #7528) at ASCO (Free ASCO Whitepaper).3 Data showed preliminary evidence that prophylactic tocilizumab potentially reduces the incidence of CRS, with no new safety concerns to date and underscores the opportunity for outpatient administration.3

Evaluation of patients with high-risk multiple myeloma from MajesTEC-1 study shows clinical benefit from treatment with TECVAYLI

Subgroup analysis from the MajesTEC-1 study of TECVAYLI investigating patients with HR RRMM will be presented at EHA (Free EHA Whitepaper) (Abstract #923).4 Results show at a median follow-up of 30 months, patients who were aged 75 years or older, patients who had HR cytogenetics and patients who were penta-drug refractory demonstrated similar efficacy as the overall RP2D population with an ORR of 54 percent, 61 percent and 60 percent and a CR or better rate of 42 percent, 42 percent and 48 percent, respectively.4 The data demonstrate the clinical benefit of TECVAYLI as an additional treatment option for some patients with HR features who typically face poor outcomes.4 The safety profile across subgroups was consistent with the RP2D population, including overall incidence and severity of TEAEs.4

Data from a single-arm run-in cohort of the Phase 3 MajesTEC-7 study shows early clinical profile of TECVAYLI-based regimen in patients with transplant ineligible/not intended newly diagnosed multiple myeloma

The results, presented in an oral presentation (Abstract #7506) at ASCO (Free ASCO Whitepaper), of the first safety run-in (SRI) from a single-arm cohort of the Phase 3 MajesTEC-7 study provide preliminary data for a TECVAYLI-based regimen in transplant- ineligible/not intended newly diagnosed multiple myeloma.5 Patients (n=26) received TECVAYLI in combination with daratumumab and lenalidomide (DR).5 At a median follow-up of 13.8 months, the ORR was 92 percent, with 23 patients remaining on treatment.5 Treatment-emergent adverse events (TEAEs) occurred in 100 percent of patients, where 61.5 percent of patients experienced grade 1/2 CRS in cycle one – all of which resolved.5

About the MajesTEC-1 Study

MajesTEC-1 (NCT03145181, NCT04557098) is a Phase 1/2 single-arm, open-label, multicohort, multicenter dose-escalation study evaluating the safety and efficacy of teclistamab in adults with RRMM who received three or more prior lines of therapy.6,7

Phase 1 of the study (NCT03145181) was conducted in two parts: dose escalation (Part 1) and dose expansion (Part 2).6 It evaluated safety, tolerability, pharmacokinetics, and preliminary efficacy of teclistamab in adult participants with RRMM.6 Phase 2 of the study (NCT04557098) evaluated the efficacy of teclistamab at the RP2D, established at subcutaneous 1.5 mg/kg weekly, as measured by ORR.

About the OPTec Study

OPTec (NCT05972135) is a Phase 2, single-arm, non-randomized, multicenter, prospective study evaluating the use of prophylactic tocilizumab in patients with RRMM to reduce the incidence and severity of CRS associated with administration of the step-up dosing regimen of teclistamab in the outpatient setting.

About the MajesTEC-7 Study

MajesTEC-7 (NCT05552222), is a Phase 3 randomized study, comparing teclistamab in combination with daratumumab SC and lenalidomide (Tec-DR) and talquetamab in combination with daratumumab SC and lenalidomide (Tal-DR) versus daratumumab SC, lenalidomide, and dexamethasone (DRd) in participants with newly diagnosed multiple myeloma who are either ineligible or not intended for autologous stem cell transplant as initial therapy.8

About TECVAYLI

TECVAYLI (teclistamab-cqyv) received approval from the U.S. FDA in October 2022 as an off-the-shelf (or ready-to-use) antibody that is administered as a subcutaneous treatment for adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody.2 The European Commission (EC) granted TECVAYLI conditional marketing authorization (CMA) in August 2022 as monotherapy for the treatment of adult patients with RRMM who have received at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody, and have demonstrated disease progression since the last therapy. In August 2023, the EC granted the approval of a Type II variation application for TECVAYLI, providing the option for a reduced dosing frequency of 1.5 mg/kg every two weeks in patients who have achieved a complete response (CR) or better for a minimum of six months. TECVAYLI is a first-in-class, bispecific T-cell engager antibody therapy that uses innovative science to activate the immune system by binding to the CD3 receptor expressed on the surface of T-cells and to the B-cell maturation antigen (BCMA) expressed on the surface of multiple myeloma cells and some healthy B-lineage cells. In February 2024, the U.S. FDA approved the supplemental Biologics License Application (sBLA) for TECVAYLI for a reduced dosing frequency of 1.5 mg/kg every two weeks (Q2W) in patients with relapsed or refractory multiple myeloma who have achieved and maintained a CR or better for a minimum of six months.

For more information, visit www.TECVAYLI.com.

About Multiple Myeloma

Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.9 In multiple myeloma, these plasma cells proliferate and spread rapidly and replace normal cells in the bone marrow with tumors.10 Multiple myeloma is the third most common blood cancer worldwide and remains an incurable disease.11 In 2024, it was estimated that more than 35,000 people will be diagnosed with multiple myeloma in the U.S. and more than 12,000 people would die from the disease.12 People living with multiple myeloma have a 5-year survival rate of 59.8 percent.13 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels and kidney problems or infections.14,15

TECVAYLI IMPORTANT SAFETY INFORMATION

WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGIC TOXICITY including IMMUNE EFFECTOR CELL-
ASSOCIATED NEUROTOXICITY SYNDROME

Cytokine release syndrome (CRS), including life-threatening or fatal reactions, can occur in patients receiving
TECVAYLI. Initiate treatment with TECVAYLI step-up dosing schedule to reduce risk of CRS. Withhold TECVAYLI
until CRS resolves or permanently discontinue based on severity.

Neurologic toxicity, including Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) and serious and life-
threatening reactions, can occur in patients receiving TECVAYLI. Monitor patients for signs or symptoms of neurologic
toxicity, including ICANS, during treatment. Withhold TECVAYLI until neurologic toxicity resolves or permanently
discontinue based on severity.

TECVAYLI is available only through a restricted program called the TECVAYLI and TALVEY Risk Evaluation and
Mitigation Strategy (REMS).

INDICATION AND USAGE

TECVAYLI (teclistamab-cqyv) is a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody.

This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

WARNINGS AND PRECAUTIONS

Cytokine Release Syndrome – TECVAYLI can cause cytokine release syndrome (CRS), including life-threatening or fatal reactions. In the clinical trial, CRS occurred in 72% of patients who received TECVAYLI at the recommended dose, with Grade 1 CRS occurring in 50% of patients, Grade 2 in 21%, and Grade 3 in 0.6%. Recurrent CRS occurred in 33% of patients. Most patients experienced CRS following step-up dose 1 (42%), step-up dose 2 (35%), or the initial treatment dose (24%). Less than 3% of patients developed first occurrence of CRS following subsequent doses of TECVAYLI. The median time to onset of CRS was 2 (range: 1 to 6) days after the most recent dose with a median duration of 2 (range: 1 to 9) days. Clinical signs and symptoms of CRS included, but were not limited to, fever, hypoxia, chills, hypotension, sinus tachycardia, headache, and elevated liver enzymes (aspartate aminotransferase and alanine aminotransferase elevation).

Initiate therapy according to TECVAYLI step-up dosing schedule to reduce risk of CRS. Administer pretreatment medications to reduce risk of CRS and monitor patients following administration of TECVAYLI accordingly. At the first sign of CRS, immediately evaluate patient for hospitalization. Administer supportive care based on severity and consider further management per current practice guidelines. Withhold or permanently discontinue TECVAYLI based on severity.

TECVAYLI is available only through a restricted program under a REMS.

Neurologic Toxicity including ICANS – TECVAYLI can cause serious or life-threatening neurologic toxicity, including Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS).

In the clinical trial, neurologic toxicity occurred in 57% of patients who received TECVAYLI at the recommended dose, with Grade 3 or 4 neurologic toxicity occurring in 2.4% of patients. The most frequent neurologic toxicities were headache (25%), motor dysfunction (16%), sensory neuropathy (15%), and encephalopathy (13%). With longer follow-up, Grade 4 seizure and fatal Guillain-Barré syndrome (one patient each) occurred in patients who received TECVAYLI.

In the clinical trial, ICANS was reported in 6% of patients who received TECVAYLI at the recommended dose. Recurrent ICANS occurred in 1.8% of patients. Most patients experienced ICANS following step-up dose 1 (1.2%), step-up dose 2 (0.6%), or the initial treatment dose (1.8%). Less than 3% of patients developed first occurrence of ICANS following subsequent doses of TECVAYLI. The median time to onset of ICANS was 4 (range: 2 to 8) days after the most recent dose with a median duration of 3 (range: 1 to 20) days. The most frequent clinical manifestations of ICANS reported were confusional state and dysgraphia. The onset of ICANS can be concurrent with CRS, following resolution of CRS, or in the absence of CRS.

Monitor patients for signs and symptoms of neurologic toxicity during treatment. At the first sign of neurologic toxicity, including ICANS, immediately evaluate patient and provide supportive therapy based on severity. Withhold or permanently discontinue TECVAYLI based on severity per recommendations and consider further management per current practice guidelines.

Due to the potential for neurologic toxicity, patients are at risk of depressed level of consciousness. Advise patients to refrain from driving or operating heavy or potentially dangerous machinery during and for 48 hours after completion of TECVAYLI step-up dosing schedule and in the event of new onset of any neurologic toxicity symptoms until neurologic toxicity resolves.

TECVAYLI is available only through a restricted program under a REMS.

TECVAYLI and TALVEY REMS – TECVAYLI is available only through a restricted program under a REMS called the TECVAYLI and TALVEY REMS because of the risks of CRS and neurologic toxicity, including ICANS.

Hepatotoxicity – TECVAYLI can cause hepatotoxicity, including fatalities. In patients who received TECVAYLI at the recommended dose in the clinical trial, there was one fatal case of hepatic failure. Elevated aspartate aminotransferase (AST) occurred in 34% of patients, with Grade 3 or 4 elevations in 1.2%. Elevated alanine aminotransferase (ALT) occurred in 28% of patients, with Grade 3 or 4 elevations in 1.8%. Elevated total bilirubin occurred in 6% of patients with Grade 3 or 4 elevations in 0.6%. Liver enzyme elevation can occur with or without concurrent CRS.

Monitor liver enzymes and bilirubin at baseline and during treatment as clinically indicated. Withhold TECVAYLI or consider permanent discontinuation of TECVAYLI based on severity.

Infections – TECVAYLI can cause severe, life-threatening, or fatal infections. In patients who received TECVAYLI at the recommended dose in the clinical trial, serious infections, including opportunistic infections, occurred in 30% of patients, with Grade 3 or 4 infections in 35%, and fatal infections in 4.2%. Monitor patients for signs and symptoms of infection prior to and during treatment with TECVAYLI and treat appropriately. Administer prophylactic antimicrobials according to guidelines. Withhold TECVAYLI or consider permanent discontinuation of TECVAYLI based on severity.

Monitor immunoglobulin levels during treatment with TECVAYLI and treat according to guidelines, including infection precautions and antibiotic or antiviral prophylaxis.

Neutropenia – TECVAYLI can cause neutropenia and febrile neutropenia. In patients who received TECVAYLI at the recommended dose in the clinical trial, decreased neutrophils occurred in 84% of patients, with Grade 3 or 4 decreased neutrophils in 56%. Febrile neutropenia occurred in 3% of patients.

Monitor complete blood cell counts at baseline and periodically during treatment and provide supportive care per local institutional guidelines. Monitor patients with neutropenia for signs of infection. Withhold TECVAYLI based on severity.

Hypersensitivity and Other Administration Reactions – TECVAYLI can cause both systemic administration-related and local injection-site reactions. Systemic Reactions – In patients who received TECVAYLI at the recommended dose in the clinical trial, 1.2% of patients experienced systemic-administration reactions, which included Grade 1 recurrent pyrexia and Grade 1 swollen tongue. Local Reactions – In patients who received TECVAYLI at the recommended dose in the clinical trial, injection-site reactions occurred in 35% of patients, with Grade 1 injection-site reactions in 30% and Grade 2 in 4.8%. Withhold TECVAYLI or consider permanent discontinuation of TECVAYLI based on severity.

Embryo-Fetal Toxicity – Based on its mechanism of action, TECVAYLI may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with TECVAYLI and for 5 months after the last dose.

ADVERSE REACTIONS

The most common adverse reactions (≥20%) were pyrexia, CRS, musculoskeletal pain, injection site reaction, fatigue, upper respiratory tract infection, nausea, headache, pneumonia, and diarrhea. The most common Grade 3 to 4 laboratory abnormalities (≥20%) were decreased lymphocytes, decreased neutrophils, decreased white blood cells, decreased hemoglobin, and decreased platelets.

Please read full Prescribing Information, including Boxed WARNING, for TECVAYLI.