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

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

Aethlon Medical Prepares for Potentially Transformative Phase 1 Cancer Treatment Studies

On June 3, 2024 Aethlon Medical, Inc. (Nasdaq: AEMD), a medical therapeutic company focused on developing products to treat cancer and life-threatening infectious diseases, reported the following update on its planned phase 1 safety, feasibility and dose-finding clinical trials of its Hemopurifier in patients with solid tumors who have stable or progressive disease during anti-PD-1 monotherapy treatment, such as Keytruda or Opdivo (Press release, Aethlon Medical, JUN 3, 2024, View Source [SID1234644045]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to make progress preparing for our planned, safety, feasibility and "dose finding" oncology trials in Australia and India, and want to provide our shareholders and other constituents with an update, stated Steven LaRosa, MD, Chief Medical Officer of Aethlon Medical. In early May, we announced new data showing the in vitro removal of exosomes from cancer patient plasma using a miniature version of our Hemopurifier. This data has been quickly integrated into the required documentation for Ethics Committees at our potential clinical sites. On May 17, 2024, we provided these documents to the Contract Research Organizations for these planned clinical trials. Subsequently, on May 24, 2024, one potential site submitted the documents to its Ethics Board. An additional site in Australia and another in India are currently assembling the packages for submission to their Ethics Committees.

Once we receive the expected Ethics Committee approvals, we will finalize the Clinical Trial Agreements. After this, hospitals can begin recruiting patients for the trials.

As a reminder, the target patient population for these safety, feasibility, and dose finding trials is oncology patients with solid tumors who are failing their anti-PD-1 monotherapy treatment, such as Keytruda or Opdivo.

Sengenics Corporation Announces New Collaboration to Advance Cancer Treatment

On June 3, 2024 Sengenics Corporation LLC and Duke Health reported an ambitious collaboration to redefine metastatic colorectal cancer (mCRC) treatment (Press release, Sengenics, JUN 3, 2024, View Source [SID1234644044]). This venture aims to explore the immune system’s response to immunotherapy, leveraging Sengenics’s state-of-the-art KREX technology to discover autoantibody biomarkers related to drug efficacy, resistance, and toxicity, thereby improving survival rates and quality of life for patients.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

mCRC remains a significant challenge in oncology, with a 5-year survival rate of 14% (Rumpold, 2020). Historically, the development of cancer immunotherapies has focused primarily on cellular immunity while neglecting the crucial functions of the humoral immune system. This joint effort will measure the humoral immune response to immunotherapy drugs, atezolizumab and bevacizumab, which have shown promise in clinical trials for mCRC treatment.

The project’s objective is to identify autoantibody biomarkers that can help stratify patients, ensuring they receive personalized and effective treatment regimens. It may also provide further insights into disease progression and mCRC subtypes.

"This research represents a significant step forward in the fight against metastatic colorectal cancer," said Professor Jonathan Blackburn, Chief Scientific Officer at Sengenics. "Autoantibodies not only offer a fresh perspective on treatment outcomes but can also provide biologically relevant and unique insights into disease that are not revealed by other ‘omics’ approaches."

Dr. Andrew Nixon, Professor of Medicine at Duke Health, added, "This collaboration underscores our commitment to enhancing patient care and health worldwide through innovative approaches. Our lab has focused on the development of circulating biomarkers for years, and we’re excited to better understand the roles that autoantibodies play in the development of metastatic colorectal cancer and therapeutic resistance."

The findings from this collaboration could herald a new era in cancer treatment, where precision medicine enables more accurate predictions of treatment outcomes, leading to more successful management of mCRC and potentially other cancers.

Bold Therapeutics Announces Positive Phase 2 Safety and Efficacy Results for BOLD-100 in Advanced Metastatic Biliary Tract and Gastric Cancers at ASCO 2024

On June 3, 2024 Bold Therapeutics, the world leader in the development of novel metallotherapeutics, reported positive Phase 2 safety and efficacy results for the company’s lead asset BOLD-100 in the treatment of advanced metastatic biliary tract cancer (BTC) and gastric cancer (GC) at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in Chicago, IL (Press release, Bold Therapeutics, JUN 3, 2024, View Source [SID1234644043]). This follows positive Phase 2 results in the treatment of advanced colorectal cancer presented at the 2024 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium in January.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

BTC and GC data from Bold Therapeutics’ ongoing multinational Phase 2 clinical trial (NCT04421820) of BOLD-100 in combination with standard-of-care FOLFOX in the treatment of patients with advanced gastrointestinal cancers at sites in Canada, the United States, Ireland, and South Korea was presented as Abstract #4115 (Poster Board #95) "A Phase 2 Study of BOLD-100 in combination with FOLFOX chemotherapy in patients with pretreated advanced biliary tract cancer: efficacy and safety analysis" and Abstract #4059 (Poster Board #39) "A Phase 2 Study of BOLD-100 in combination with FOLFOX chemotherapy in patients with advanced gastric cancer: efficacy and safety analysis", respectively, in the Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary session on June 1, 2024, from 1:30 – 4:30PM CDT.

BTC Efficacy and Safety:

18 out of 22 patients treated were eligible for efficacy evaluation;
Patients had received a median of 2 prior therapies;
Median progression-free survival (PFS) of 6.0 months [95% CI: 3.8 – 10.0] and median OS of 7.3 months [95% CI: 4.5 – 13.0], which compares extremely favorably with standard-of-care outcomes;
One patient (n=1) achieving a partial response (PR) for an objective response rate (ORR) of 6% [95% CI: 1.0, 23.0], fourteen (n=14) patients with stable disease (SD), and disease control rate (DCR) of 83% [95% CI: 62.0, 95.0];
Patients received a median of 4 cycles (range: 1 – 41) of therapy; and
For all treated patients (n=21), 21 had ≥ 1 TRAEs, most commonly neutrophil count decrease (n=10, 46%), nausea (n=8, 36%), fatigue (n=7, 32%), peripheral-sensory neuropathy (n=6, 27%), and pyrexia (n=6, 27%).
GC Efficacy and Safety:

18 out of 21 patients treated were eligible for efficacy evaluation;
Patients had received a median of 4 prior therapies;
Median progression-free survival (PFS) of 4.2 months [95% CI: 2.8 – 7.1] and median OS of 7.9 months [95% CI: 4.8 – 15.0], which compares extremely favorably with standard-of-care outcomes;
Two patients (n=2) achieved a partial response (PR) for an objective response rate (ORR) of 11% [95% CI: 2.0, 31.0], eleven (n=11) patients with stable disease (SD), and disease control rate (DCR) of 72% [95% CI: 49.0, 89.0];
Patients received a median of 6 cycles (range: 1 – 27) of therapy; and
For all treated patients (n=21), 19 patients had ≥ 1 TRAEs, most commonly neutrophil count decrease (n=7, 33%), nausea (n=6, 29%), and peripheral-sensory neuropathy (n=4, 19%), with most AEs being grade 1-2.
"Driven by our dedicated and talented employees and supported by generous grants and subsidies from the government of Canada, BOLD-100 continues to exceed expectations," added E. Russell McAllister, CEO. "On top of the robustly positive Phase 2 clinical data presented at both ASCO (Free ASCO Whitepaper) and ASCO (Free ASCO Whitepaper) GI, Bold Therapeutics expects to announce intriguing nonclinical data later this year, specifically biomarker data that we think will generate significant excitement amongst both clinicians and scientists as we disrupt commonly held perceptions about how to safely and effectively treat some of the most difficult-to-treat solid tumor indications where existing therapies are largely ineffective."

Later this month, Bold Therapeutics expects to begin enrolling patients into a multinational Phase 2 randomized clinical trial comparing two different doses of BOLD-100 in combination with standard-of-care FOLFOX against standard-of-care FOLFOX in patients with second-line FOLFOX-naïve colorectal cancer. Based on prior results, we expect to see significant improvements in both safety and efficacy outcomes. In addition, Bold Therapeutics is currently manufacturing drug product to support a pivotal Phase 3 trial in advanced colorectal cancer.

Minghui Pharmaceutical Presents Phase 1/2 Clinical Data of MHB088C (B7-H3 ADC) as Monotherapy for the Treatment of Patients with Recurrent or Metastatic Solid Tumors at the 2024 ASCO Annual Meeting

On June 3, 2024 Minghui Pharmaceutical, Inc., a late-stage clinical biopharmaceutical company, reported that the preliminary Phase 1/2 clinical data of MHB088C (B7-H3 ADC) was presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in an oral presentation (Press release, Minghui Pharmaceutical, JUN 3, 2024, View Source [SID1234644042]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Abstract #: 3012

Results of a Phase 1/2 Study of MHB088C: a Novel B7-H3 Antibody-Drug Conjugate (ADC) Incorporating a Potent DNA Topoisomerase I Inhibitor in Recurrent or Metastatic Solid Tumors

In this Phase 1/2 study, the safety/tolerability, pharmacokinetics, and efficacy of MHB088C in patients (pts) with recurrent or metastatic solid tumors were evaluated. The study results were as follows:

At data cutoff, MHB088C was well tolerated. The most common TRAEs were hematological toxicities. 4 mg/kg Q3W was the DLT dose and 3 mg/kg Q3W was defined as the MTD. 1.6 mg/kg Q2W, 2.0 mg/kg Q3W and 2.4 mg/kg Q3W demonstrated favorable safety profiles with low single-digit Grade ≥3 hematological AEs for the two lower doses. No interstitial lung disease (ILD) was reported as of the data cutoff.
­ Of 98 efficacy evaluable pts with different tumor types at doses ranging from 0.8 mg/kg to 4.0mg/kg, ORR was 33.7% (33/98) and DCR was 83.7% (82/98), with DoR not reached yet. The majority of pts remain on the treatment.
­ Of 31 efficacy evaluable pts with small cell lung cancer (SCLC), ORR was 61.3% (19/31) and DCR was 93.5% (29/31). 24 pts (77.4%) remain on the treatment, and 6 pts (19.4%) achieved 60% or more tumor reduction, with 2 CRs of target lesion at 3.0 mg/kg. Among 10 pts at 1.6 mg/kg Q2W, ORR was 80% and DCR was 90%.
­ Of 7 efficacy evaluable pts with esophageal squamous cell carcinoma (ESCC), ORR was 42.9% and DCR was 85.7%. The median follow-up was over 3.0 months.
Professor Lin Shen from the Beijing Cancer Hospital stated: "MHB088C represents an innovative advancement in cancer treatment as a novel B7-H3 ADC equipped with a potent DNA Topo I inhibitor. The preliminary data are highly encouraging, showcasing clinically meaningful and durable antitumor activities at very safe doses across multiple cancer types. We are optimistic about the ongoing study and anticipate further positive clinical outcomes."

Dr. Guoqing Cao, CEO of Minghui Pharmaceutical, stated: "We are excited to share the results of Phase 1/2 study of MHB088C at the 2024 ASCO (Free ASCO Whitepaper). MHB088C is a well-differentiated B7-H3 ADC, conjugating an optimal anti-human B7-H3 antibody with our Proprietary SuperTopoiTM payload. We are seeing robust efficacy at very safe doses without major hematological toxicity or ILD. Particularly, in SCLC, 9 out of 10 patients experienced tumor shrinkage at 1.6 mg/kg Q2W, with an ORR of 80%. The durability of responses was also notable in ESCC, with an ORR of 43% and median follow-up over 3 months.

The safety profile of MHB088C is consistent with that of our other program MHB036C, a TROP-2 ADC, without major hematological toxicity or ILD either. Between the two clinical programs, with data from more than 250 patients, we are confident that we now have one of the best ADC platforms in the industry. Efficacy in other cancer types has also been observed and will be reported in future conferences. We look forward to initiating registrational trials of the monotherapy for selected tumor types by the end of 2024 and exploring the IO combination in earlier line settings for both ADC assets in the near future."

About MHB088C
MHB088C is a novel B7-H3 ADC generated through Minghui’s SuperTopoiTM ADC platform. Minghui’s proprietary payload is 5 to 10 times more potent than DXd, retaining key advantages such as bystander effect while eliminating the risk of interstitial lung disease. Conjugated with Minghui’s proprietary B7-H3 antibody, which has superior binding and internalization compared to the competitor’s antibodies, MHB088C has demonstrated remarkable anti-tumor efficacy across various cancer types. It was 3 to 10 times more potent in killing tumor cells than the competitor’s compound in xenograft models.