On September 19, 2025 Johnson & Johnson (NYSE:JNJ) reported that an investigational immune-based induction regimen with TECVAYLI (teclistamab-cqyv) and DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) demonstrated meaningful clinical efficacy in transplant-eligible (TE) patients with newly diagnosed multiple myeloma (NDMM) (Press release, Johnson & Johnson, SEP 19, 2025, View Source [SID1234656115]).

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!

Forty-nine patients were treated across three treatment cohorts, with a steroid-sparing approach, including regimens of TECVAYLI with DARZALEX FASPRO and lenalidomide, with and without bortezomib.1 Overall response (≥partial response) was achieved by 100% of patients in all treatment arms following induction therapy.1 Of 46 MRD-evaluable patients with available samples after Cycle 3 and/or Cycle 6, 100% achieved MRD negativity by next-generation flow at 10-5 sensitivity threshold.1 By next-generation sequencing, all were MRD-negative at 10-6 after Cycle 6.1 Overall, 85.7% (42/49) of patients achieved a complete response or better (≥CR) and were MRD-negative at Cycle 6 (≤10⁻⁵).1 Additionally, 96% of patients successfully completed stem cell mobilization with a median total stem cell yield of 8.1×106/kg.1

"Multiple myeloma weakens the immune system over time, making it difficult to eliminate cancerous plasma cells," said Marc S. Raab, M.D., Heidelberg University Hospital, Germany.* "It’s critical to treat patients with the most effective regimens in the newly diagnosed setting. TECVAYLI and DARZALEX FASPRO work synergistically to significantly reduce cancer cells – resulting in deep responses with potential for long term benefit."

Data from the safety analysis reinforced the tolerability of the approach.1 The most common treatment-emergent adverse events (TEAEs) were hematologic, and Grade 3/4 infections occurred in 36.7% of patients.1 Serious TEAEs occurred in 53% (n=26) of patients.1 No TEAEs led to full study treatment discontinuation and no Grade 5 adverse events were observed.1 Additionally, no patients experienced immune effector cell-associated neurotoxicity syndrome (ICANS).1 Cytokine release syndrome (CRS) was observed in 65% of patients (n=32) and were all Grade 1/2.1

"Exploring TECVAYLI, with its established use in later lines of treatment, and DARZALEX FASPRO, a foundational therapy across all lines, together helps us understand how this doublet immunotherapy can support patients across different stages of disease," said Jordan Schecter, M.D., Vice President, Disease Area Leader, Multiple Myeloma, Johnson & Johnson Innovative Medicine. "Importantly, immune-based therapies like TECVAYLI have the potential to improve patient outcomes with a manageable safety profile, particularly when used earlier in the treatment journey, with 100 percent of evaluable patients achieving MRD negativity. We continue to explore TECVAYLI in earlier lines of therapy and across difficult-to-treat populations."

About MajesTEC-5 Study
MajesTEC-5 (NCT05695508) is an ongoing, Phase 2 study evaluating the safety and efficacy of combination regimens including teclistamab and daratumumab in participants with newly diagnosed transplant eligible multiple myeloma. The MajesTEC-5 clinical trial is sponsored by the German-Speaking Myeloma Multicenter Group (GMMG) and Deutsche Studiengruppe Multiples Myelom (DSMM), and conducted in collaboration with Johnson & Johnson.2

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.3 Since FDA approval, more than 15,900 patients have been treated worldwide with TECVAYLI. 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 (Q2W) 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 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 DARZALEX FASPRO and DARZALEX
DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) received U.S. FDA approval in May 2020 and is approved for ten indications in multiple myeloma, three of which are for frontline treatment in newly diagnosed patients who are transplant eligible or ineligible. It is the only subcutaneous CD38-directed antibody approved to treat patients with MM. DARZALEX FASPRO is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE drug delivery technology.

DARZALEX (daratumumab) received U.S. FDA approval in November 2015 and is approved in ten indications, three of which are in the frontline setting, including newly diagnosed patients who are transplant eligible and ineligible.

DARZALEX is the first CD38-directed antibody approved to treat multiple myeloma. DARZALEX-based regimens have been used in the treatment of more than 618,000 patients worldwide.

In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialize daratumumab.

Since 2020, the National Comprehensive Cancer Network (NCCN) has recommended daratumumab-based combination regimens for the treatment of newly diagnosed multiple myeloma and relapsed and refractory multiple myeloma.† For newly diagnosed multiple myeloma in non-transplant candidates, the NCCN guidelines recommend daratumumab in combination with lenalidomide and dexamethasone as a Category 1 preferred regimen; daratumumab in combination with bortezomib, melphalan, and prednisone as another recommended Category 1 regimen; and daratumumab in combination with bortezomib, cyclophosphamide, and prednisone as another recommended Category 2A regimen. For newly diagnosed multiple myeloma in transplant candidates, the NCCN guidelines recommend daratumumab in combination with bortezomib, lenalidomide and dexamethasone as another recommended Category 2A regimen; daratumumab in combination with bortezomib, thalidomide and dexamethasone as a Category 2A regimen useful in certain circumstances; daratumumab in combination with carfilzomib, lenalidomide and dexamethasone as a Category 2A regimen useful in certain circumstances; and daratumumab in combination with cyclophosphamide, bortezomib and dexamethasone as a Category 2A regimen useful in certain circumstances. For maintenance in transplant candidates, the NCCN guidelines recommend daratumumab in combination with lenalidomide as useful in certain circumstances. In relapsed/refractory myeloma, four daratumumab regimens are listed as Category 1 preferred regimens for early relapses (1-3 prior therapies): daratumumab in combination with lenalidomide and dexamethasone; daratumumab in combination with bortezomib and dexamethasone; daratumumab in combination with carfilzomib and dexamethasone; and daratumumab in combination with pomalidomide and dexamethasone [after one prior therapy including lenalidomide and a proteasome inhibitor (PI)]. The NCCN also recommends daratumumab in combination with cyclophosphamide, bortezomib and dexamethasone as another Category 2A regimen for early relapses (1-3 prior therapies) and as monotherapy as a Category 2A regimen useful in certain circumstances for early relapse patients after at least three prior therapies, including a PI and an immunomodulatory agent, or for patients who are double refractory to a PI and an immunomodulatory agent.

For more information, visit www.DARZALEX.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.4 In multiple myeloma, these plasma cells proliferate and spread rapidly and replace normal cells in the bone marrow with tumors.5 Multiple myeloma is the third most common blood cancer worldwide and remains an incurable disease.6 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.7 People living with multiple myeloma have a 5-year survival rate of 59.8 percent.8 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.9,10

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.

DARZALEX INDICATIONS AND IMPORTANT SAFETY INFORMATION

INDICATIONS
DARZALEX (daratumumab) is indicated for the treatment of adult patients with multiple myeloma:

In combination with bortezomib, melphalan, and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant
In combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy
In combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant
In combination with pomalidomide and dexamethasone in patients who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor
In combination with carfilzomib and dexamethasone in patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy
In combination with bortezomib and dexamethasone in patients who have received at least one prior therapy
As monotherapy in patients who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent
CONTRAINDICATIONS

DARZALEX is contraindicated in patients with a history of severe hypersensitivity (eg, anaphylactic reactions) to daratumumab or any of the components of the formulation.

WARNINGS AND PRECAUTIONS

Infusion-Related Reactions

DARZALEX can cause severe and/or serious infusion-related reactions including anaphylactic reactions. These reactions can be life threatening, and fatal outcomes have been reported. In clinical trials (monotherapy and combination: N=2066), infusion-related reactions occurred in 37% of patients with the Week 1 (16 mg/kg) infusion, 2% with the Week 2 infusion, and cumulatively 6% with subsequent infusions. Less than 1% of patients had a Grade 3/4 infusion-related reaction at Week 2 or subsequent infusions. The median time to onset was 1.5 hours (range: 0 to 73 hours). Nearly all reactions occurred during infusion or within 4 hours of completing DARZALEX. Severe reactions have occurred, including bronchospasm, hypoxia, dyspnea, hypertension, tachycardia, headache, laryngeal edema, pulmonary edema, and ocular adverse reactions, including choroidal effusion, acute myopia, and acute angle closure glaucoma. Signs and symptoms may include respiratory symptoms, such as nasal congestion, cough, throat irritation, as well as chills, vomiting, and nausea. Less common signs and symptoms were wheezing, allergic rhinitis, pyrexia, chest discomfort, pruritus, hypotension and blurred vision.

When DARZALEX dosing was interrupted in the setting of ASCT (CASSIOPEIA) for a median of 3.75 months (range: 2.4 to 6.9 months), upon re-initiation of DARZALEX, the incidence of infusion-related reactions was 11% for the first infusion following ASCT. Infusion-related reactions occurring at re-initiation of DARZALEX following ASCT were consistent in terms of symptoms and severity (Grade 3 or 4: <1%) with those reported in previous studies at Week 2 or subsequent infusions. In EQUULEUS, patients receiving combination treatment (n=97) were administered the first 16 mg/kg dose at Week 1 split over two days, ie, 8 mg/kg on Day 1 and Day 2, respectively. The incidence of any grade infusion-related reactions was 42%, with 36% of patients experiencing infusion-related reactions on Day 1 of Week 1, 4% on Day 2 of Week 1, and 8% with subsequent infusions.

Pre-medicate patients with antihistamines, antipyretics, and corticosteroids. Frequently monitor patients during the entire infusion. Interrupt DARZALEX infusion for reactions of any severity and institute medical management as needed. Permanently discontinue DARZALEX therapy if an anaphylactic reaction or life-threatening (Grade 4) reaction occurs and institute appropriate emergency care. For patients with Grade 1, 2, or 3 reactions, reduce the infusion rate when re-starting the infusion.

To reduce the risk of delayed infusion-related reactions, administer oral corticosteroids to all patients following DARZALEX infusions. Patients with a history of chronic obstructive pulmonary disease may require additional post-infusion medications to manage respiratory complications. Consider prescribing short- and long-acting bronchodilators and inhaled corticosteroids for patients with chronic obstructive pulmonary disease.

Ocular adverse reactions, including acute myopia and narrowing of the anterior chamber angle due to ciliochoroidal effusions with potential for increased intraocular pressure or glaucoma, have occurred with DARZALEX infusion. If ocular symptoms occur, interrupt DARZALEX infusion and seek immediate ophthalmologic evaluation prior to restarting DARZALEX.

Interference With Serological Testing

Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive indirect antiglobulin test (indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6 months after the last daratumumab infusion. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient’s serum. The determination of a patient’s ABO and Rh blood type is not impacted. Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX. Type and screen patients prior to starting DARZALEX.

Neutropenia and Thrombocytopenia

DARZALEX may increase neutropenia and thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer’s prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX until recovery of neutrophils or for recovery of platelets.

Interference With Determination of Complete Response

Daratumumab is a human immunoglobulin G (IgG) kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some patients with IgG kappa myeloma protein.

Embryo-Fetal Toxicity

Based on the mechanism of action, DARZALEX can cause fetal harm when administered to a pregnant woman. DARZALEX may cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX and for 3 months after the last dose.

The combination of DARZALEX with lenalidomide, pomalidomide, or thalidomide is contraindicated in pregnant women because lenalidomide, pomalidomide, and thalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide, pomalidomide, or thalidomide prescribing information on use during pregnancy.

ADVERSE REACTIONS

The most frequently reported adverse reactions (incidence ≥20%) were: upper respiratory infection, neutropenia, infusion related reactions, thrombocytopenia, diarrhea, constipation, anemia, peripheral sensory neuropathy, fatigue, peripheral edema, nausea, cough, pyrexia, dyspnea, and asthenia. The most common hematologic laboratory abnormalities (≥40%) with DARZALEX are: neutropenia, lymphopenia, thrombocytopenia, leukopenia, and anemia.

Please click here to see the full Prescribing Information.

On September 19. 2025 Precision Biologics, Inc. reported that in vitro and in vivo efficacy of the novel ADC, using its anti-core 2 O-glycans anti-human carcinoma mAb PB-223 (PB-vcMMAE-5), against human ovarian cancer expressing truncated core 2 O-glycans will be presented in a poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference in Cancer Research: Advances in Ovarian Cancer Research on September 20th, 2025, Grand Hyatt Denver, Denver, Colorado, USA (Press release, Precision Biologics, SEP 19, 2025, View Source [SID1234656116]).

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!

Poster title: In vitro and in vivo efficacy of the antibody-drug-conjugate (ADC) PB-vcMMAE-5 against human ovarian cancer expressing truncated core 2 O-glycans

The presentation of the poster will be made in person on the following date and location:

Saturday, September 20, 6:30 p.m. – 8:00 p.m.

Grand Hyatt Denver, Denver, Colorado, USA

Poster Section: B

Abstract Control Number: B068

BACKGROUND:

Ovarian cancers remain largely unresponsive to immune checkpoint inhibitors, in part due to their ability to suppress the cytotoxic activity of immune cells infiltrating the tumor microenvironment. One of the disrupted pathways in these cancers is O-glycosylation, a feature particularly associated with ovarian cancer progression, metastasis and poor prognosis. This underscores the urgent need for alternative therapeutic strategies. We developed an ADC, designated PB-vcMMAE-5, composed of the following:

The mAb: We used PB-223, an innovative mAb developed through affinity maturation of mAb NEO-102 (Ensituximab), a chimeric human IgG1 mAb that targets truncated core 2 O-glycans, specifically expressed by cancer cells and not by healthy tissues. The binding affinity of PB-223 for its target was improved, compared to NEO-102, by optimizing its VH and VL sequences through Fast Screening for Expression Biophysical Properties and Affinity. PB-223 demonstrated a binding affinity (KD) at least 4-fold lower than NEO-102, indicating stronger tumor binding. PB-223 does not bind to normal tissues and it can be internalized into human cancer cell lines expressing its target.

The payload: Monomethyl auristatin E (MMAE) was used as payload. MMAE is a potent antimitotic agent that inhibits cell division by blocking the polymerization of tubulin and is the most common ADC payload used to be linked to antibodies in clinical development for oncologic applications.

The linker: mc-vc-PABc was used as a cleavable linker. PB-223 was conjugated to the linker-payload through a cysteine-based conjugation method.
STUDY PRESENTED AT AACR (Free AACR Whitepaper) Special Conference in Cancer Research: Advances in Ovarian Cancer Research, 2025

After development of the ADC PB-vcMMAE-5, we evaluated its efficacy in vitro and in vivo

In vitro efficacy: The in vitro cytotoxicity of PB-vc-MMAE-5 was tested in four human cancer cell lines: triple-negative breast cancer (HCC1937, MDA-MB-231), ER+/PR+/HER2+ breast cancer (BT474), and ovarian cancer (OV-90). Cells were treated with varying ADC concentrations for 5 days.
This study shows that PB-vcMMAE-5 effectively killed all cell lines tested.
At the highest concentration, the percentage of cell killing for PB-vcMMAE-5 was 52.72% in HCC1937 and 88.36% in MDA-MB-231.
At the highest concentration, the percentage of cell killing for PB-vcMMAE-5 was 92.51% in OV-90 and 83.22% in BT474.
In contrast, naked PB-223 mAb showed no killing in all cell lines tested

In vivo safety: In a study presented in April 2025, at AACR (Free AACR Whitepaper) Annual Meeting 2025, we showed that PB-vcMMAE-5 was well tolerated in rats. No sign of distress nor loss of body weight were observed after administration. In this study we confirm the same pattern in mice. NOD-SCID mice bearing OV-90 xenografts were treated with weekly doses of PBS, MMAE alone, or PB-vc-MMAE-5 (1, 3, 6, or 9 mg/kg) for five weeks. Animal body weight was monitored regularly, twice a week, as an indirect measure of toxicity. The ADC PB-vcMMAE-5 was well tolerated in mice. No sign of distress and loss of body weight were observed

No significant hematological or pathological changes were detected in the liver, spleen, brain, or heart of mice treated with efficacious doses of the ADC compared with controls.

In vivo efficacy: The efficacy of the ADC PB-vcMMAE-5 was assessed in OV-90 subcutaneous xenograft model established in NOD-SCID mice. The ADC PB-vcMMAE-5 was administered intravenously at doses 1, 3, 6 or 9 mg/kg, once per week for five weeks.

On day 31 from first ADC infusion, most alive mice were sacrificed, and tumors were excised for histological analysis using Ki-67 staining to assess proliferating viable tumor cells. To further assess systemic toxicity and prolonged efficacy, three mice each from the 6 and 9 mg/kg groups were followed to day 45.

Data presented in this study show that PB-vcMMAE-5 at 1 mg/kg did not significantly reduce tumor volume compared with PBS, whereas PB-vcMMAE-5 at 6 and 9 mg/kg induced robust reduction of tumor volume.

In addition, analysis of tumor viability at day 45 (Ki67 H-score) showed no tumor activity in mice treated with PB-vcMMAE-5 at 9 mg/kg
Findings from this study showed that PB-vcMMAE-5 can kill human cancer cells expressing PB-223’s target, is not toxic in vivo in mice and is highly effective in vivo at 9 mg/kg in NOD-SCID mice bearing human ovarian cancer. In addition, in a poster presented at AACR (Free AACR Whitepaper) Annual Meeting 2025 we reported that PB-vcMMAE-5 is stable in human plasma.

All these data suggest that PB-vcMMAE-5 has promising potential as a therapeutic option for human ovarian tumors and for a range of human malignancies expressing core 2 O-glycans.

On September 19, 2025 Nuvation Bio Inc. (NYSE: NUVB), a global oncology company focused on tackling some of the toughest challenges in cancer treatment, reported that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved IBTROZITM (taletrectinib) for the treatment of adult patients with ROS1-positive (ROS1+) unresectable, advanced and/or recurrent non-small cell lung cancer (NSCLC) (Press release, Nuvation Bio, SEP 19, 2025, View Source [SID1234656117]). As part of an exclusive license agreement entered in 2023, Nippon Kayaku will commercialize IBTROZI in Japan.

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!

The approval by Japan’s MHLW was based on data from the pivotal Phase 2 TRUST clinical program evaluating taletrectinib in patients globally, including Japan. Results from a pooled analysis of the TRUST clinical program were published in the Journal of Clinical Oncology in April 2025.

In parallel, the MHLW also approved the AmoyDx PLC Panel as a companion diagnostic to identify patients with locally advanced or metastatic ROS1+ NSCLC who may benefit from treatment with IBTROZI.

"Building on the regulatory approvals for IBTROZI in the U.S. and China, this additional approval by the MHLW further underscores the best-in-class potential and promise that IBTROZI holds for patients living with advanced ROS1-positive non-small cell lung cancer around the globe," said David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio. "We remain steadfastly committed to bringing forward innovative cancer treatments that can help patients stay ahead of their disease."

On June 11, 2025, the U.S. Food and Drug Administration (FDA) granted full approval to IBTROZI for the treatment of locally advanced or metastatic ROS1+ NSCLC across lines of therapy, following a Priority Review and double Breakthrough Therapy designations. On June 20, the National Comprehensive Cancer Network added taletrectinib (IBTROZI) as a Preferred Agent in the National Comprehensive Cancer Network Clinical Practice Guidelines (NCCN Guidelines) in Oncology for Non-Small Cell Lung Cancers (NSCLC). Specifically, the NCCN Guidelines now include taletrectinib (IBTROZI) as a Preferred Agent for both first-line and subsequent therapy for ROS1+ NSCLC, including specific recommendations for those with brain metastases and resistance mutations.

Prior to this, Nuvation Bio announced on January 6, 2025, that China’s National Medical Products Administration (NMPA) had approved taletrectinib for the treatment of adult patients with locally advanced or metastatic ROS1+ NSCLC who either have or have not been previously treated with ROS1 tyrosine kinase inhibitors (TKI). Taletrectinib is marketed in China by Innovent Biologics under the brand name DOVBLERON.

Upon the first establishment of the reimbursement price in Japan, which is anticipated in the fourth quarter of 2025, Nuvation Bio will receive a USD $25 million milestone payment to go towards continued progression of our pipeline and portfolio.

About ROS1+ NSCLC
Each year, more than one million people globally are diagnosed with non-small cell lung cancer (NSCLC), the most common form of lung cancer. It is estimated that approximately 2% of patients with NSCLC have ROS1+ disease. About 35% of patients newly diagnosed with metastatic ROS1+ NSCLC have tumors that have spread to their brain. The brain is also the most common site of disease progression, with about 50% of previously treated patients developing central nervous system (CNS) metastases.

About IBTROZI
IBTROZI is an oral, potent, CNS-active, selective, next-generation ROS1 inhibitor therapy. On June 11, following Priority Review and Breakthrough Therapy Designations for both first- and second-line or later, the U.S. Food and Drug Administration (FDA) approved IBTROZI for the treatment of adult patients with locally advanced or metastatic ROS1+ NSCLC. Learn more at IBTROZI.com.

About the TRUST Clinical Program
The TRUST clinical program evaluating IBTROZI for the treatment of adult patients with advanced ROS1+ NSCLC included two Phase 2 single-arm pivotal studies: TRUST-I (NCT04395677) in China, which enrolled 173 patients, and TRUST-II (NCT04919811), a global study, which enrolled 189 patients. The primary endpoint of these registrational studies is confirmed objective response rate (cORR) as assessed by an independent review committee (IRC). Secondary endpoints include intracranial cORR, duration of response, progression-free survival, and safety.

Indication
IBTROZI is indicated for the treatment of adult patients with locally advanced or metastatic ROS1+ non-small cell lung cancer (NSCLC).

IMPORTANT SAFETY INFORMATION FOR IBTROZITM (taletrectinib)

WARNINGS AND PRECAUTIONS

Hepatotoxicity: Hepatotoxicity, including drug-induced liver injury and fatal adverse reactions, can occur. 88% of patients experienced increased AST, including 10% Grade 3/4. 85% of patients experienced increased ALT, including 13% Grade 3/4. Fatal liver events occurred in 0.6% of patients. Median time to first onset of AST or ALT elevation was 15 days (range: 3 days to 20.8 months).

Increased AST or ALT each led to dose interruption in 7% of patients and dose reduction in 5% and 9% of patients, respectively. Permanent discontinuation was caused by increased AST, ALT, or bilirubin each in 0.3% and by hepatotoxicity in 0.6% of patients.

Concurrent elevations in AST or ALT ≥3 times the ULN and total bilirubin ≥2 times the ULN, with normal alkaline phosphatase, occurred in 0.6% of patients.

Interstitial Lung Disease (ILD)/Pneumonitis: Severe, life-threatening, or fatal ILD or pneumonitis can occur. ILD/pneumonitis occurred in 2.3% of patients, including 1.1% Grade 3/4. One fatal ILD case occurred at the 400 mg daily dose. Median time to first onset of ILD/pneumonitis was 3.8 months (range: 12 days to 11.8 months).

ILD/pneumonitis led to dose interruption in 1.1% of patients, dose reduction in 0.6% of patients, and permanent discontinuation in 0.6% of patients.

QTc Interval Prolongation: QTc interval prolongation can occur, which can increase the risk for ventricular tachyarrhythmias (e.g., torsades de pointes) or sudden death. IBTROZI prolongs the QTc interval in a concentration-dependent manner.

In patients who received IBTROZI and underwent at least one post baseline ECG, QTcF increase of >60 msec compared to baseline and QTcF >500 msec occurred in 13% and 2.6% of patients, respectively. 3.4% of patients experienced Grade ≥3. Median time from first dose of IBTROZI to onset of ECG QT prolongation was 22 days (range: 1 day to 38.7 months). Dose interruption and dose reduction each occurred in 2.8% of patients.

Significant QTc interval prolongation may occur when IBTROZI is taken with food, strong and moderate CYP3A inhibitors, and/or drugs with a known potential to prolong QTc. Administer IBTROZI on an empty stomach. Avoid concomitant use with strong and moderate CYP3A inhibitors and/or drugs with a known potential to prolong QTc.​

Hyperuricemia: Hyperuricemia can occur and was reported in 14% of patients, with 16% of these requiring urate-lowering medication without pre-existing gout or hyperuricemia. 0.3% of patients experienced Grade ≥3. Median time to first onset was 2.1 months (range: 7 days to 35.8 months). Dose interruption occurred in 0.3% of patients.

Myalgia with Creatine Phosphokinase (CPK) Elevation: Myalgia with or without CPK elevation can occur. Myalgia occurred in 10% of patients. Median time to first onset was 11 days (range: 2 days to 10 months).

Concurrent myalgia with increased CPK within a 7-day time period occurred in 0.9% of patients. Dose interruption occurred in 0.3% of patients with myalgia and concurrent CPK elevation.

Skeletal Fractures: IBTROZI can increase the risk of fractures. ROS1 inhibitors as a class have been associated with skeletal fractures. 3.4% of patients experienced fractures, including 1.4% Grade 3. Some fractures occurred in the setting of a fall or other predisposing factors. Median time to first onset of fracture was 10.7 months (range: 26 days to 29.1 months). Dose interruption occurred in 0.3% of patients.

Embryo-Fetal Toxicity: Based on literature, animal studies, and its mechanism of action, IBTROZI can cause fetal harm when administered to a pregnant woman.

ADVERSE REACTIONS
Among patients who received IBTROZI, the most frequently reported adverse reactions (≥20%) were diarrhea (64%), nausea (47%), vomiting (43%), dizziness (22%), rash (22%), constipation (21%), and fatigue (20%). ​

The most frequently reported Grade 3/4 laboratory abnormalities (≥5%) were increased ALT (13%), increased AST (10%), decreased neutrophils (5%), and increased creatine phosphokinase (5%). ​

DRUG INTERACTIONS

Strong and Moderate CYP3A Inhibitors/CYP3A Inducers and Drugs that Prolong the QTc Interval: Avoid concomitant use.
Gastric Acid Reducing Agents: Avoid concomitant use with PPIs and H2 receptor antagonists. If an acid-reducing agent cannot be avoided, administer locally acting antacids at least 2 hours before or 2 hours after taking IBTROZI.
OTHER CONSIDERATIONS

Pregnancy: Please see important information in Warnings and Precautions under Embryo-Fetal Toxicity. ​
Lactation: Advise women not to breastfeed during treatment and for 3 weeks after the last dose.
Effect on Fertility: Based on findings in animals, IBTROZI may impair fertility in males and females. The effects on animal fertility were reversible.
Pediatric Use: The safety and effectiveness of IBTROZI in pediatric patients has not been established.
Photosensitivity: IBTROZI can cause photosensitivity. Advise patients to minimize sun exposure and to use sun protection, including broad-spectrum sunscreen, during treatment and for at least 5 days after discontinuation.
Please see accompanying full Prescribing Information.

On September 19, 2025 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it obtained regulatory approval from the Ministry of Health, Labour and Welfare for the anti-cancer agent/humanized anti-PD-L1 monoclonal antibody Tecentriq Intravenous Infusion [generic name: atezolizumab (genetical recombination)] for an additional indication of relapsed or refractory extranodal natural killer/T-cell lymphoma, nasal type (ENKL) (Press release, Chugai, SEP 19, 2025, View Source;category= [SID1234656076]). Tecentriq is the first immune-checkpoint inhibitor approved in Japan for this disease.

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 are very pleased that we can offer Tecentriq as a new treatment option for ENKL in adults and children 12 years and older. ENKL is a rare type of malignant lymphoma that primarily develops in the nasal cavity. In advanced stages, approximately 60% of patients relapse after initial treatment, and there is no established standard therapy after relapse. We will continue our efforts to provide information on the proper use of Tecentriq in order to contribute to the patients with ENKL," said Chugai’s President and CEO, Dr. Osamu Okuda.

This approval is based on the results from the phase II ATTACK study initiated by investigators in Japan including National Cancer Center Hospital, which evaluated the efficacy and safety of Tecentriq in patients with R/R ENKL.

In addition to the approval of this expanded indication, we have also received additional approval for a 4-weekly dosing regimen in the "dosage and administration" for existing indications in lung cancer and breast cancer. The additional options in dosing intervals will enable more flexible treatment planning. This is expected to improve convenience for patients and healthcare professionals by reducing the number of hospital visits and lessening the burden on both.

Chugai Pharmaceutical, a leading company in the oncology field, remains committed to addressing unmet medical needs in cancer treatment with innovative medicines, supporting patients and healthcare professionals.

Approval Information *Newly added or revised description (underlined)

All indications: The initial dose of Tecentriq must be administered over 60 minutes. If the first infusion is tolerated, all subsequent infusions may be administered over 30 minutes.

Indications Dosage and administration
Unresectable, advanced or recurrent non-small cell lung cancer
For patients with chemotherapy-naïve unresectable advanced or recurrent non-squamous non-small cell lung cancer
The usual adult dosage is 1200 mg atezolizumab (genetical recombination) administered by intravenous infusion once every 3 weeks.
Subsequently, when administered as monotherapy, atezolizumab (genetical recombination) is administered as an intravenous infusion either 1200 mg once every 3 weeks or 1680 mg once every 4 weeks.
For patients with chemotherapy-naïve PD-L1 positve unresectable advanced or recurrent non-small cell lung cancer
The usual adult dosage of atezolizumab (genetical recombination) is either 1200 mg once every 3 weeks or 1680 mg once every 4 weeks administered by intravenous infusion.

For patients with unresectable advanced or recurrent non-small cell lung cancer previously treated with chemotherapy
The usual adult dosage of atezolizumab (genetical recombination) is either 1200 mg once every 3 weeks or 1680 mg once every 4 weeks administered by intravenous infusion.

PD-L1-positive adjuvant non-small cell lung cancer The usual adult dosage of atezolizumab (genetical recombination) is either 1200 mg once every 3 weeks or 1680 mg once every 4 weeks administered by intravenous infusion. The dosage period is up to 12 months.
Extensive-stage small cell lung cancer The usual adult dosage is 1200 mg atezolizumab (genetical recombination) in combination with carboplatin and etoposide by intravenous infusion once every 3 weeks for 4 doses.
This is followed by atezolizumab (genetical recombination) 1200 mg administered as an intravenous infusion once every 3 weeks or 1680 mg once every 4 weeks.
Unresectable hepatocellular carcinoma The usual adult dosage is 1200 mg atezolizumab (genetical recombination) in combination with bevacizumab administered by intravenous infusion once every 3 weeks.
PD-L1-positive, hormone receptor-negative and HER2-negative inoperable or recurrent breast cancer The usual adult dosage of atezolizumab (genetical recombination) in combination with nab-paclitaxel (albumin-bound) is either 840 mg once every 2 weeks or 1680 mg once every 4 weeks administered by intravenous infusion.
Unresectable alveolar soft part sarcoma The usual adult dosage is 1200 mg atezolizumab (genetical recombination) administered by intravenous infusion once every 3 weeks. The usual dose for children over 2 years old is 15 mg/kg (weight) (max 1200 mg) atezolizumab (genetical recombination) administered by intravenous infusion once every 3 weeks.
Relapsed or refractory extranodal natural killer/T-cell lymphoma, nasal type The usual adult dosage of 1200 mg atezolizumab (genetical recombination) administered by intravenous infusion once every 3 weeks. The usual dose for patients aged 12 to < 18 years is 15 mg/kg (body weight) atezolizumab (genetical recombination) (max 1200 mg) administered by intravenous infusion once every 3 weeks.
About the ATTACK study1
ATTACK study (NCCH1903, jRCT2031190177) is a Japanese Phase II, multicenter, open-label, single-arm study led by physicians including National Cancer Center Hospital to evaluate the efficacy and safety of Tecentriq in patients with relapsed or refractory extranodal natural killer/T-cell lymphoma, nasal type. The study enrolled 14 patients to investigate safety and efficacy. The primary endpoint is the independent review committee (IRC)-assessed overall response rate. Key secondary endpoints include progression-free survival, and overall survival.
The ATTACK study is being conducted as a substudy of the MASTER KEY project, which promotes the development of treatments for rare cancers through industry-academia collaboration with the National Cancer Center Hospital.

About extranodal natural killer/T-cell lymphoma, nasal type (ENKL)
ENKL is a form of malignant lymphoma that primarily affects the nasal cavity. It can occur in individuals of all ages, from children to adults.2,3,4 ENKL is rare, accounting for approximately 0.68% of all malignant lymphoma cases (annual incidence: about 36,000 cases) in Japan.5 For patients with advanced ENKL, about 60% experience relapse following initial treatment.6,7 Relapsed or refractory ENKL has a poor prognosis, and there is currently no established standard treatment.

About Tecentriq8
Tecentriq is a cancer immune-checkpoint inhibitor targeting PD-L1, which is a protein expressed on tumor and tumor-infiltrating immune cells. PD-L1 blocks T cell activity by binding with PD-1 and B7.1 receptors on the T cell surface. By inhibiting PD-L1, Tecentriq may enable the activation of T cells and boost immune response against cancer cells. In Japan, Tecentriq was launched in April 2018 and has obtained approval for 5 indications (non-small cell lung cancer, extensive-stage small cell lung cancer, breast cancer, hepatocellular carcinoma and alveolar soft part sarcoma).

Trademarks used or mentioned in this release are protected by law.

On September 19, 2025 IMUNON, Inc. (Nasdaq: IMNN), a clinical-stage company in Phase 3 development with its DNA-mediated immunotherapy, reported that new translational data from the Phase 2 OVATION 2 Study of IMNN-001, its investigational therapy for the treatment of women with newly diagnosed advanced ovarian cancer, will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference in Cancer Research: Advances in Ovarian Cancer Research, being held September 19-21, 2025, in Denver, Colorado (Press release, IMUNON, SEP 19, 2025, View Source [SID1234656102]).

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!

IMNN-001, based on IMUNON’s proprietary TheraPlas technology platform, is an interleukin-12 (IL-12) DNA plasmid vector incorporated into a novel nanoparticle delivery system, enabling cell transfection followed by persistent, local production and secretion of the IL-12 protein in the tumor microenvironment. IL-12 is a powerful pluripotent cytokine known for inducing strong anti-cancer immunity by promoting T-lymphocyte and natural killer cell proliferation while inhibiting tumor-mediated immune suppression. IMNN-001 is the first therapy to achieve a clinically effective response in advanced (stage IIIC/IV) ovarian cancer including benefits in both progression-free survival and overall survival in a first-line treatment setting when used with standard of care chemotherapy.

Details of the AACR (Free AACR Whitepaper) presentation:

Abstract Title: IMNN-001, an IL-12 gene therapy, added to Neo/Adjuvant chemotherapy safely turns the tumor microenvironment cold-to-hot in newly diagnosed epithelial ovarian cancer (EOC)
Presenting Author: Douglas V. Faller, M.D., Ph.D., Chief Medical Officer, IMUNON
Date: Saturday, September 20, 2025
Time: 6:35-8:05 p.m. ET
Abstract Number: B050

About the OVATION 3 Study

OVATION 3 is IMUNON’s pivotal Phase 3 study of IMMN-001, an IL-12 gene-mediated immunotherapy, in women with advanced epithelial ovarian cancer. The study is supported with unprecedented overall survival (OS) data from a large, 112-patient, randomized Phase 2 study showing the following:

Median 13-month increase in OS (HR 0.70) and median 3-month increase in PFS (HR 0.79) in IMNN-001 treatment arm compared to standard of care alone.
Better therapeutic effect observed with IMNN-001 treatment compared to the control arm (p=0.0375), as shown by mean 6.5-month extension of time free of progression or death (PFS + OS) captured in totality of treatment effect.
Use of poly ADP-ribose polymerase (PARP) inhibitors as part of maintenance therapy further enhanced outcomes, with median OS not yet reached in IMNN-001 treatment arm as patients surpass >5 years since randomization in the trial compared to 37 months on standard of care (HR 0.42).
While not powered for significance, the results from the OVATION 2 Study nonetheless have resulted in invitations to present at both ASCO (Free ASCO Whitepaper) and ESMO (Free ESMO Whitepaper) annual meetings and publication in the peer-reviewed journal Gynecologic Oncology.

OVATION 3 is currently enrolling patients at four trial sites with up to 46 additional sites being considered for activation.