Sharon Worcester, MA

The United States Food and Drug Administration (FDA) has approved bevacizumab-nwgd (Jobevne, Biocon Biologics Ltd), a biosimilar to bevacizumab (Avastin, Genentech), for intravenous use across multiple cancer types.

Approval was based on “a comprehensive package of comparative pharmacokinetic, safety, efficacy, nonclinical, structural, analytical and functional data, which confirmed the Jobevne is highly similar to Avastin,” according to a Biocon Biologics Ltd press release. 

“The data demonstrated that there were no clinically meaningful differences between Jobevne and Avastin in terms of pharmacokinetics, safety, efficacy, and immunogenicity,” the company stated.

The biosimilar agent is indicated as part of various combinations for the treatment of metastatic colorectal cancer, certain types of non-squamous non–small cell lung cancer, recurrent glioblastoma, metastatic renal cell carcinoma, certain advanced cervical cancers, and epithelial ovarian, fallopian tube, or primary peritoneal cancers, the company noted.

The agent is not indicated for adjuvant treatment of colon cancer, according to the press release, which includes detailed information about the indications, as well as a list of warnings and precautions.

A version of this article first appeared on Medscape.com.

The United States Food and Drug Administration (FDA) has approved bevacizumab-nwgd (Jobevne, Biocon Biologics Ltd), a biosimilar to bevacizumab (Avastin, Genentech), for intravenous use across multiple cancer types.

Approval was based on “a comprehensive package of comparative pharmacokinetic, safety, efficacy, nonclinical, structural, analytical and functional data, which confirmed the Jobevne is highly similar to Avastin,” according to a Biocon Biologics Ltd press release. 

“The data demonstrated that there were no clinically meaningful differences between Jobevne and Avastin in terms of pharmacokinetics, safety, efficacy, and immunogenicity,” the company stated.

The biosimilar agent is indicated as part of various combinations for the treatment of metastatic colorectal cancer, certain types of non-squamous non–small cell lung cancer, recurrent glioblastoma, metastatic renal cell carcinoma, certain advanced cervical cancers, and epithelial ovarian, fallopian tube, or primary peritoneal cancers, the company noted.

The agent is not indicated for adjuvant treatment of colon cancer, according to the press release, which includes detailed information about the indications, as well as a list of warnings and precautions.

A version of this article first appeared on Medscape.com.

The United States Food and Drug Administration (FDA) has approved bevacizumab-nwgd (Jobevne, Biocon Biologics Ltd), a biosimilar to bevacizumab (Avastin, Genentech), for intravenous use across multiple cancer types.

Approval was based on “a comprehensive package of comparative pharmacokinetic, safety, efficacy, nonclinical, structural, analytical and functional data, which confirmed the Jobevne is highly similar to Avastin,” according to a Biocon Biologics Ltd press release. 

“The data demonstrated that there were no clinically meaningful differences between Jobevne and Avastin in terms of pharmacokinetics, safety, efficacy, and immunogenicity,” the company stated.

The biosimilar agent is indicated as part of various combinations for the treatment of metastatic colorectal cancer, certain types of non-squamous non–small cell lung cancer, recurrent glioblastoma, metastatic renal cell carcinoma, certain advanced cervical cancers, and epithelial ovarian, fallopian tube, or primary peritoneal cancers, the company noted.

The agent is not indicated for adjuvant treatment of colon cancer, according to the press release, which includes detailed information about the indications, as well as a list of warnings and precautions.

A version of this article first appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved nivolumab (Opdivo, Bristol Myers Squibb) with ipilimumab (Yervoy, Bristol Myers Squibb) as a first-line treatment for adults with unresectable or metastatic hepatocellular carcinoma (HCC). 

The decision, which follows the FDA’s 2020 accelerated approval in the second-line setting for advanced HCC and adds to the list of other indications for the combination therapy, was based on efficacy demonstrated in the randomized, open-label CHECKMATE-9DW trial. The trial enrolled 668 patients with unresectable or metastatic HCC and no prior systemic therapy for advanced disease, according to the FDA approval notice. 

Median overall survival, the primary outcome measure, was 23.7 months in those randomized to receive nivolumab + ipilimumab, compared with 20.6 months in those randomized to receive investigators’ choice of lenvatinib or sorafenib (hazard ratio, 0.79). The overall response rate was 36.1% vs 13.2% in the arms, respectively.

Those in the treatment arm received 1 mg/kg intravenous (IV) nivolumab with 3 mg/kg IV ipilimumab every 3 weeks for up to four doses, followed by single-agent IV nivolumab at 480 mg every 4 weeks. Those in the control arm received either 8 or 12 mg lenvatinib daily or 400 mg sorafenib twice daily until disease progression or unacceptable toxicity, according to early results from the trial, which were presented at the 2024 American Society of Clinical Oncology meeting. 

Adverse reactions occurring in more than 20% of patients included rash, pruritus, fatigue, and diarrhea.

The recommended nivolumab dose for this indication is 1 mg/kg with 3 mg/kg ipilimumab given intravenously every 3 weeks for up to four doses, followed by 240 mg nivolumab every 2 weeks or 480 mg nivolumab every 4 weeks. Full prescribing information will be available at Drugs@FDA.

A version of this article first appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved nivolumab (Opdivo, Bristol Myers Squibb) with ipilimumab (Yervoy, Bristol Myers Squibb) as a first-line treatment for adults with unresectable or metastatic hepatocellular carcinoma (HCC). 

The decision, which follows the FDA’s 2020 accelerated approval in the second-line setting for advanced HCC and adds to the list of other indications for the combination therapy, was based on efficacy demonstrated in the randomized, open-label CHECKMATE-9DW trial. The trial enrolled 668 patients with unresectable or metastatic HCC and no prior systemic therapy for advanced disease, according to the FDA approval notice. 

Median overall survival, the primary outcome measure, was 23.7 months in those randomized to receive nivolumab + ipilimumab, compared with 20.6 months in those randomized to receive investigators’ choice of lenvatinib or sorafenib (hazard ratio, 0.79). The overall response rate was 36.1% vs 13.2% in the arms, respectively.

Those in the treatment arm received 1 mg/kg intravenous (IV) nivolumab with 3 mg/kg IV ipilimumab every 3 weeks for up to four doses, followed by single-agent IV nivolumab at 480 mg every 4 weeks. Those in the control arm received either 8 or 12 mg lenvatinib daily or 400 mg sorafenib twice daily until disease progression or unacceptable toxicity, according to early results from the trial, which were presented at the 2024 American Society of Clinical Oncology meeting. 

Adverse reactions occurring in more than 20% of patients included rash, pruritus, fatigue, and diarrhea.

The recommended nivolumab dose for this indication is 1 mg/kg with 3 mg/kg ipilimumab given intravenously every 3 weeks for up to four doses, followed by 240 mg nivolumab every 2 weeks or 480 mg nivolumab every 4 weeks. Full prescribing information will be available at Drugs@FDA.

A version of this article first appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved nivolumab (Opdivo, Bristol Myers Squibb) with ipilimumab (Yervoy, Bristol Myers Squibb) as a first-line treatment for adults with unresectable or metastatic hepatocellular carcinoma (HCC). 

The decision, which follows the FDA’s 2020 accelerated approval in the second-line setting for advanced HCC and adds to the list of other indications for the combination therapy, was based on efficacy demonstrated in the randomized, open-label CHECKMATE-9DW trial. The trial enrolled 668 patients with unresectable or metastatic HCC and no prior systemic therapy for advanced disease, according to the FDA approval notice. 

Median overall survival, the primary outcome measure, was 23.7 months in those randomized to receive nivolumab + ipilimumab, compared with 20.6 months in those randomized to receive investigators’ choice of lenvatinib or sorafenib (hazard ratio, 0.79). The overall response rate was 36.1% vs 13.2% in the arms, respectively.

Those in the treatment arm received 1 mg/kg intravenous (IV) nivolumab with 3 mg/kg IV ipilimumab every 3 weeks for up to four doses, followed by single-agent IV nivolumab at 480 mg every 4 weeks. Those in the control arm received either 8 or 12 mg lenvatinib daily or 400 mg sorafenib twice daily until disease progression or unacceptable toxicity, according to early results from the trial, which were presented at the 2024 American Society of Clinical Oncology meeting. 

Adverse reactions occurring in more than 20% of patients included rash, pruritus, fatigue, and diarrhea.

The recommended nivolumab dose for this indication is 1 mg/kg with 3 mg/kg ipilimumab given intravenously every 3 weeks for up to four doses, followed by 240 mg nivolumab every 2 weeks or 480 mg nivolumab every 4 weeks. Full prescribing information will be available at Drugs@FDA.

A version of this article first appeared on Medscape.com.

A noninvasive clinicopathologic and gene expression profiling (CP-GEP)–based tool, the Merlin assay, shows promise for identifying recurrence risks in patients with early-stage melanoma who do not undergo sentinel lymph node biopsy (SNLB).

This was the conclusion of a retrospective analysis of a large cohort of patients with stage I/II disease, reported by Teresa Amaral, MD, PhD, at the 11th World Congress of Melanoma and 21st European Association of Dermato-Oncology Congress 2025.

Of 930 patients included in the study, the assay identified 879 as having a low risk for recurrence and 51 as having high risk for recurrence. The overall 5-year recurrence-free survival (RFS), distant metastasis-free survival (DMFS) and melanoma-specific survival (MSS) rates were 90.9%, 96.9%, and 97.5%, respectively.

The corresponding rates among those stratified by the assay as having low vs high recurrence risk, respectively, were 94.6% and 26.6% for RFS (hazard ratio [HR], 25.08), 98.6% vs 62.1% for DMFS (HR, 35.39), and 99.4% vs 61.7% for MSS (HR, 71.05), said Amaral, during her presentation at the meeting.

Of 16 melanoma-specific deaths, 12 were stratified as high risk for recurrence by the CP-GEP assay, said Amaral, head of the Skin Cancer Clinical Trials Center at the University of Tübingen, Tübingen, Germany, and first author of the study.

Study participants had stages IA-IIC melanoma, 41% were women, and median age was 64 years. Median melanoma thickness was 0.5 mm, and 94% were not ulcerated. 

No systemic treatment options currently exist for patients with this early-stage disease, the author said.

The CP-GEP model, initially developed by the Mayo Clinic and SkylineDx BV to predict the positivity of SNLB, has been validated in multiple studies.

Amaral and her colleagues previously demonstrated the ability of the CP-GEP model to stratify patients with stages I-II disease as having low or high risk for recurrence — including in a small number of patients without SNLB. Those findings are confirmed in this larger population of patients who did not undergo SNLB, she said.

SkylineDx announced the findings in a press release stating the results validate the prognostic power of the Merlin assay for “identifying tumors at high risk for relapse that would otherwise be missed by traditional clinical and pathological evaluation.”

SNLB is the gold standard for nodal assessment for staging cutaneous melanoma. More than 80% of patients who undergo SNLB are negative for nodal metastases, but most patients who relapse or die from their melanoma are initially stratified by SNLB as having low-risk early-stage disease, Amaral explained. This suggests “SNLB is not enough,” she noted.

The findings suggest that CP-GEP has the potential to risk stratify patients with early-stage melanoma that did not undergo SLNB and help select those who can forgo SLNB, she said.

Without another means of assessing risk, patients considered low risk based on SNLB are closely followed, the author explained.

“If we could identify the very low-risk patient and then allocate the resources to the very high-risk patients who really need a more detailed and more tailored approach…we would be doing a favor to our patients,” the author concluded.

Amaral disclosed personal financial relationships with Delcath, Philogen, Bristol Myers Squibb, NeraCare, Novartis, Pierre Fabre, CeCaVa, and MedTrix.

A version of this article first appeared on Medscape.com.

A noninvasive clinicopathologic and gene expression profiling (CP-GEP)–based tool, the Merlin assay, shows promise for identifying recurrence risks in patients with early-stage melanoma who do not undergo sentinel lymph node biopsy (SNLB).

This was the conclusion of a retrospective analysis of a large cohort of patients with stage I/II disease, reported by Teresa Amaral, MD, PhD, at the 11th World Congress of Melanoma and 21st European Association of Dermato-Oncology Congress 2025.

Of 930 patients included in the study, the assay identified 879 as having a low risk for recurrence and 51 as having high risk for recurrence. The overall 5-year recurrence-free survival (RFS), distant metastasis-free survival (DMFS) and melanoma-specific survival (MSS) rates were 90.9%, 96.9%, and 97.5%, respectively.

The corresponding rates among those stratified by the assay as having low vs high recurrence risk, respectively, were 94.6% and 26.6% for RFS (hazard ratio [HR], 25.08), 98.6% vs 62.1% for DMFS (HR, 35.39), and 99.4% vs 61.7% for MSS (HR, 71.05), said Amaral, during her presentation at the meeting.

Of 16 melanoma-specific deaths, 12 were stratified as high risk for recurrence by the CP-GEP assay, said Amaral, head of the Skin Cancer Clinical Trials Center at the University of Tübingen, Tübingen, Germany, and first author of the study.

Study participants had stages IA-IIC melanoma, 41% were women, and median age was 64 years. Median melanoma thickness was 0.5 mm, and 94% were not ulcerated. 

No systemic treatment options currently exist for patients with this early-stage disease, the author said.

The CP-GEP model, initially developed by the Mayo Clinic and SkylineDx BV to predict the positivity of SNLB, has been validated in multiple studies.

Amaral and her colleagues previously demonstrated the ability of the CP-GEP model to stratify patients with stages I-II disease as having low or high risk for recurrence — including in a small number of patients without SNLB. Those findings are confirmed in this larger population of patients who did not undergo SNLB, she said.

SkylineDx announced the findings in a press release stating the results validate the prognostic power of the Merlin assay for “identifying tumors at high risk for relapse that would otherwise be missed by traditional clinical and pathological evaluation.”

SNLB is the gold standard for nodal assessment for staging cutaneous melanoma. More than 80% of patients who undergo SNLB are negative for nodal metastases, but most patients who relapse or die from their melanoma are initially stratified by SNLB as having low-risk early-stage disease, Amaral explained. This suggests “SNLB is not enough,” she noted.

The findings suggest that CP-GEP has the potential to risk stratify patients with early-stage melanoma that did not undergo SLNB and help select those who can forgo SLNB, she said.

Without another means of assessing risk, patients considered low risk based on SNLB are closely followed, the author explained.

“If we could identify the very low-risk patient and then allocate the resources to the very high-risk patients who really need a more detailed and more tailored approach…we would be doing a favor to our patients,” the author concluded.

Amaral disclosed personal financial relationships with Delcath, Philogen, Bristol Myers Squibb, NeraCare, Novartis, Pierre Fabre, CeCaVa, and MedTrix.

A version of this article first appeared on Medscape.com.

A noninvasive clinicopathologic and gene expression profiling (CP-GEP)–based tool, the Merlin assay, shows promise for identifying recurrence risks in patients with early-stage melanoma who do not undergo sentinel lymph node biopsy (SNLB).

This was the conclusion of a retrospective analysis of a large cohort of patients with stage I/II disease, reported by Teresa Amaral, MD, PhD, at the 11th World Congress of Melanoma and 21st European Association of Dermato-Oncology Congress 2025.

Of 930 patients included in the study, the assay identified 879 as having a low risk for recurrence and 51 as having high risk for recurrence. The overall 5-year recurrence-free survival (RFS), distant metastasis-free survival (DMFS) and melanoma-specific survival (MSS) rates were 90.9%, 96.9%, and 97.5%, respectively.

The corresponding rates among those stratified by the assay as having low vs high recurrence risk, respectively, were 94.6% and 26.6% for RFS (hazard ratio [HR], 25.08), 98.6% vs 62.1% for DMFS (HR, 35.39), and 99.4% vs 61.7% for MSS (HR, 71.05), said Amaral, during her presentation at the meeting.

Of 16 melanoma-specific deaths, 12 were stratified as high risk for recurrence by the CP-GEP assay, said Amaral, head of the Skin Cancer Clinical Trials Center at the University of Tübingen, Tübingen, Germany, and first author of the study.

Study participants had stages IA-IIC melanoma, 41% were women, and median age was 64 years. Median melanoma thickness was 0.5 mm, and 94% were not ulcerated. 

No systemic treatment options currently exist for patients with this early-stage disease, the author said.

The CP-GEP model, initially developed by the Mayo Clinic and SkylineDx BV to predict the positivity of SNLB, has been validated in multiple studies.

Amaral and her colleagues previously demonstrated the ability of the CP-GEP model to stratify patients with stages I-II disease as having low or high risk for recurrence — including in a small number of patients without SNLB. Those findings are confirmed in this larger population of patients who did not undergo SNLB, she said.

SkylineDx announced the findings in a press release stating the results validate the prognostic power of the Merlin assay for “identifying tumors at high risk for relapse that would otherwise be missed by traditional clinical and pathological evaluation.”

SNLB is the gold standard for nodal assessment for staging cutaneous melanoma. More than 80% of patients who undergo SNLB are negative for nodal metastases, but most patients who relapse or die from their melanoma are initially stratified by SNLB as having low-risk early-stage disease, Amaral explained. This suggests “SNLB is not enough,” she noted.

The findings suggest that CP-GEP has the potential to risk stratify patients with early-stage melanoma that did not undergo SLNB and help select those who can forgo SLNB, she said.

Without another means of assessing risk, patients considered low risk based on SNLB are closely followed, the author explained.

“If we could identify the very low-risk patient and then allocate the resources to the very high-risk patients who really need a more detailed and more tailored approach…we would be doing a favor to our patients,” the author concluded.

Amaral disclosed personal financial relationships with Delcath, Philogen, Bristol Myers Squibb, NeraCare, Novartis, Pierre Fabre, CeCaVa, and MedTrix.

A version of this article first appeared on Medscape.com.

The United States Food and Drug Administration (FDA) has expanded the approval for lutetium Lu 177 vipivotide tetraxetan (Pluvicto, Novartis) to include adults with prostate-specific membrane antigen (PSMA)–positive metastatic castration-resistant prostate cancer (mCRPC), who have received androgen receptor pathway inhibitor (ARPI) therapy and are considered appropriate to delay taxane-based chemotherapy.

The radioligand therapeutic agent was previously approved for the treatment of PSMA-positive mCRPC in patients who have already received ARPI therapy and taxane-based chemotherapy. Approval for the expanded indication was based on efficacy demonstrated in the randomized, open-label, phase 3 PSMAfore trial.

Treatment in 468 patients who progressed on one ARPI and who were deemed appropriate for delay of taxane-based chemotherapy was associated with improved radiographic progression-free survival (rPFS) and overall survival (OS) vs a different ARPI.

Patients were randomized 1:1 to receive lutetium Lu 177 vipivotide tetraxetan (7.4 GBq [200 mCi] every 6 weeks for six doses) or to receive a different ARPI, according to a statement from the FDA. Those who progressed on the new ARPI were allowed to cross over to the experimental therapy arm after progression, and 60% did so.

Median rPFS was 9.3 vs 5.6 months in the experimental and control arms, respectively (hazard ratio [HR], 0.41). Median OS durations were 24.5 and 23.1 months, respectively (HR, 0.91), but the difference in OS did not reach statistical significance.

Adverse reactions were consistent with the known safety profile of lutetium Lu 177 vipivotide tetraxetan, which includes possible radiation exposure, myelosuppression, and renal toxicity.

The recommended dose, according to prescribing information, is 7.4 GBq (200 mCi) administered intravenously every 6 weeks for six doses or until disease progression or unacceptable toxicity.

“The earlier indication for Pluvicto could really change our treatment paradigms for patients with mCRPC. It offers a targeted therapy that better delays disease progression compared to a second ARPI,” Michael Morris, MD, of Memorial Sloan Kettering Cancer Center, New York, and the principal investigator of the study in the United States stated in a Novartis press release. “This approval is a significant step forward and should open the doorway to a therapy that has clear clinical advantages for the patient with mCRPC who has progressed on one ARPI and has not received chemotherapy.”

A version of this article first appeared on Medscape.com.

The United States Food and Drug Administration (FDA) has expanded the approval for lutetium Lu 177 vipivotide tetraxetan (Pluvicto, Novartis) to include adults with prostate-specific membrane antigen (PSMA)–positive metastatic castration-resistant prostate cancer (mCRPC), who have received androgen receptor pathway inhibitor (ARPI) therapy and are considered appropriate to delay taxane-based chemotherapy.

The radioligand therapeutic agent was previously approved for the treatment of PSMA-positive mCRPC in patients who have already received ARPI therapy and taxane-based chemotherapy. Approval for the expanded indication was based on efficacy demonstrated in the randomized, open-label, phase 3 PSMAfore trial.

Treatment in 468 patients who progressed on one ARPI and who were deemed appropriate for delay of taxane-based chemotherapy was associated with improved radiographic progression-free survival (rPFS) and overall survival (OS) vs a different ARPI.

Patients were randomized 1:1 to receive lutetium Lu 177 vipivotide tetraxetan (7.4 GBq [200 mCi] every 6 weeks for six doses) or to receive a different ARPI, according to a statement from the FDA. Those who progressed on the new ARPI were allowed to cross over to the experimental therapy arm after progression, and 60% did so.

Median rPFS was 9.3 vs 5.6 months in the experimental and control arms, respectively (hazard ratio [HR], 0.41). Median OS durations were 24.5 and 23.1 months, respectively (HR, 0.91), but the difference in OS did not reach statistical significance.

Adverse reactions were consistent with the known safety profile of lutetium Lu 177 vipivotide tetraxetan, which includes possible radiation exposure, myelosuppression, and renal toxicity.

The recommended dose, according to prescribing information, is 7.4 GBq (200 mCi) administered intravenously every 6 weeks for six doses or until disease progression or unacceptable toxicity.

“The earlier indication for Pluvicto could really change our treatment paradigms for patients with mCRPC. It offers a targeted therapy that better delays disease progression compared to a second ARPI,” Michael Morris, MD, of Memorial Sloan Kettering Cancer Center, New York, and the principal investigator of the study in the United States stated in a Novartis press release. “This approval is a significant step forward and should open the doorway to a therapy that has clear clinical advantages for the patient with mCRPC who has progressed on one ARPI and has not received chemotherapy.”

A version of this article first appeared on Medscape.com.

The United States Food and Drug Administration (FDA) has expanded the approval for lutetium Lu 177 vipivotide tetraxetan (Pluvicto, Novartis) to include adults with prostate-specific membrane antigen (PSMA)–positive metastatic castration-resistant prostate cancer (mCRPC), who have received androgen receptor pathway inhibitor (ARPI) therapy and are considered appropriate to delay taxane-based chemotherapy.

The radioligand therapeutic agent was previously approved for the treatment of PSMA-positive mCRPC in patients who have already received ARPI therapy and taxane-based chemotherapy. Approval for the expanded indication was based on efficacy demonstrated in the randomized, open-label, phase 3 PSMAfore trial.

Treatment in 468 patients who progressed on one ARPI and who were deemed appropriate for delay of taxane-based chemotherapy was associated with improved radiographic progression-free survival (rPFS) and overall survival (OS) vs a different ARPI.

Patients were randomized 1:1 to receive lutetium Lu 177 vipivotide tetraxetan (7.4 GBq [200 mCi] every 6 weeks for six doses) or to receive a different ARPI, according to a statement from the FDA. Those who progressed on the new ARPI were allowed to cross over to the experimental therapy arm after progression, and 60% did so.

Median rPFS was 9.3 vs 5.6 months in the experimental and control arms, respectively (hazard ratio [HR], 0.41). Median OS durations were 24.5 and 23.1 months, respectively (HR, 0.91), but the difference in OS did not reach statistical significance.

Adverse reactions were consistent with the known safety profile of lutetium Lu 177 vipivotide tetraxetan, which includes possible radiation exposure, myelosuppression, and renal toxicity.

The recommended dose, according to prescribing information, is 7.4 GBq (200 mCi) administered intravenously every 6 weeks for six doses or until disease progression or unacceptable toxicity.

“The earlier indication for Pluvicto could really change our treatment paradigms for patients with mCRPC. It offers a targeted therapy that better delays disease progression compared to a second ARPI,” Michael Morris, MD, of Memorial Sloan Kettering Cancer Center, New York, and the principal investigator of the study in the United States stated in a Novartis press release. “This approval is a significant step forward and should open the doorway to a therapy that has clear clinical advantages for the patient with mCRPC who has progressed on one ARPI and has not received chemotherapy.”

A version of this article first appeared on Medscape.com.

Vaccines for treating and preventing cancer have long been considered a holy grail in oncology.

But aside from a few notable exceptions — including the human papillomavirus (HPV) vaccine, which has dramatically reduced the incidence of HPV-related cancers, and a Bacillus Calmette-Guerin vaccine, which helps prevent early-stage bladder cancer recurrence — most have failed to deliver.

Following a string of disappointments over the past decade, recent advances in the immunotherapy space are bringing renewed hope for progress.

In an American Association for Cancer Research (AACR) series earlier in 2024, Catherine J. Wu, MD, predicted big strides for cancer vaccines, especially for personalized vaccines that target patient-specific neoantigens — the proteins that form on cancer cells — as well as vaccines that can treat diverse tumor types.

“A focus on neoantigens that arise from driver mutations in different tumor types could allow us to make progress in creating off-the-shelf vaccines,” said Wu, the Lavine Family Chair of Preventative Cancer Therapies at Dana-Farber Cancer Institute and a professor of medicine at Harvard Medical School, both in Boston, Massachusetts.

A prime example is a personalized, messenger RNA (mRNA)–based vaccine designed to prevent melanoma recurrence. The mRNA-4157 vaccine encodes up to 34 different patient-specific neoantigens.

“This is one of the most exciting developments in modern cancer therapy,” said Lawrence Young, a virologist and professor of molecular oncology at the University of Warwick, Coventry, England, who commented on the investigational vaccine via the UK-based Science Media Centre.

Other promising options are on the horizon as well. In August, BioNTech announced a phase 1 global trial to study BNT116 — a vaccine to treat non–small cell lung cancer (NSCLC). BNT116, like mRNA-4157, targets specific antigens in the lung cancer cells.

“This technology is the next big phase of cancer treatment,” Siow Ming Lee, MD, a consultant medical oncologist at University College London Hospitals in England, which is leading the UK trial for the lung cancer and melanoma vaccines, told The Guardian. “We are now entering this very exciting new era of mRNA-based immunotherapy clinical trials to investigate the treatment of lung cancer.”

Still, these predictions have a familiar ring. While the prospects are exciting, delivering on them is another story. There are simply no guarantees these strategies will work as hoped.

 

Then: Where We Were

Cancer vaccine research began to ramp up in the 2000s, and in 2006, the first-generation HPV vaccine, Gardasil, was approved. Gardasil prevents infection from four strains of HPV that cause about 80% of cervical cancer cases.

In 2010, the Food and Drug Administration approved sipuleucel-T, the first therapeutic cancer vaccine, which improved overall survival in patients with hormone-refractory prostate cancer.

Researchers predicted this approval would “pave the way for developing innovative, next generation of vaccines with enhanced antitumor potency.”

In a 2015 AACR research forecast report, Drew Pardoll, MD, PhD, co-director of the Cancer Immunology and Hematopoiesis Program at Johns Hopkins University, Baltimore, Maryland, said that “we can expect to see encouraging results from studies using cancer vaccines.”

Despite the excitement surrounding cancer vaccines alongside a few successes, the next decade brought a longer string of late-phase disappointments.

In 2016, the phase 3 ACT IV trial of a therapeutic vaccine to treat glioblastoma multiforme (CDX-110) was terminated after it failed to demonstrate improved survival.

In 2017, a phase 3 trial of the therapeutic pancreatic cancer vaccine, GVAX, was stopped early for lack of efficacy.

That year, an attenuated Listeria monocytogenes vaccine to treat pancreatic cancer and mesothelioma also failed to come to fruition. In late 2017, concerns over listeria infections prompted Aduro Biotech to cancel its listeria-based cancer treatment program.

In 2018, a phase 3 trial of belagenpumatucel-L, a therapeutic NSCLC vaccine, failed to demonstrate a significant improvement in survival and further study was discontinued.

And in 2019, a vaccine targeting MAGE-A3, a cancer-testis antigen present in multiple tumor types, failed to meet endpoints for improved survival in a phase 3 trial, leading to discontinuation of the vaccine program.

But these disappointments and failures are normal parts of medical research and drug development and have allowed for incremental advances that helped fuel renewed interest and hope for cancer vaccines, when the timing was right, explained vaccine pioneer Larry W. Kwak, MD, PhD, deputy director of the Comprehensive Cancer Center at City of Hope, Duarte, California.

When it comes to vaccine progress, timing makes a difference. In 2011, Kwak and colleagues published promising phase 3 trial results on a personalized vaccine. The vaccine was a patient-specific tumor-derived antigen for patients with follicular lymphoma in their first remission following chemotherapy. Patients who received the vaccine demonstrated significantly longer disease-free survival.

But, at the time, personalized vaccines faced strong headwinds due, largely, to high costs, and commercial interest failed to materialize. “That’s been the major hurdle for a long time,” said Kwak.

Now, however, interest has returned alongside advances in technology and research. The big shift has been the emergence of lower-cost rapid-production mRNA and DNA platforms and a better understanding of how vaccines and potent immune stimulants, like checkpoint inhibitors, can work together to improve outcomes, he explained.

“The timing wasn’t right” back then, Kwak noted. “Now, it’s a different environment and a different time.”

 

A Turning Point?

Indeed, a decade later, cancer vaccine development appears to be headed in a more promising direction.

Among key cancer vaccines to watch is the mRNA-4157 vaccine, developed by Merck and Moderna, designed to prevent melanoma recurrence. In a recent phase 2 study, patients receiving the mRNA-4157 vaccine alongside pembrolizumab had nearly half the risk for melanoma recurrence or death at 3 years compared with those receiving pembrolizumab alone. Investigators are now evaluating the vaccine in a global phase 3 study in patients with high-risk, stage IIB to IV melanoma following surgery.

Another one to watch is the BNT116 NSCLC vaccine from BioNTech. This vaccine presents the immune system with NSCLC tumor markers to encourage the body to fight cancer cells expressing those markers while ignoring healthy cells. BioNTech also launched a global clinical trial for its vaccine this year.

Other notables include a pancreatic cancer mRNA vaccine, which has shown promising early results in a small trial of 16 patients. Of 16 patients who received the vaccine alongside chemotherapy and after surgery and immunotherapy, 8 responded. Of these eight, six remained recurrence free at 3 years. Investigators noted that the vaccine appeared to stimulate a durable T-cell response in patients who responded.

Kwak has also continued his work on lymphoma vaccines. In August, his team published promising first-in-human data on the use of personalized neoantigen vaccines as an early intervention in untreated patients with lymphoplasmacytic lymphoma. Among nine asymptomatic patients who received the vaccine, all achieved stable disease or better, with no dose-limiting toxicities. One patient had a minor response, and the median time to progression was greater than 72 months.

“The current setting is more for advanced disease,” Kwak explained. “It’s a tougher task, but combined with checkpoint blockade, it may be potent enough to work.” 

Still, caution is important. Despite early promise, it’s too soon to tell which, if any, of these investigational vaccines will pan out in the long run. Like investigational drugs, cancer vaccines may show big promising initially but then fail in larger trials.

One key to success, according to Kwak, is to design trials so that even negative results will inform next steps.

But, he noted, failures in large clinical trials will “put a chilling effect on cancer vaccine research again.”

“That’s what keeps me up at night,” he said. “We know the science is fundamentally sound and we have seen glimpses over decades of research that cancer vaccines can work, so it’s really just a matter of tweaking things to optimize trial design.”

Companies tend to design trials to test if a vaccine works or not, without trying to understand why, he said.

“What we need to do is design those so that we can learn from negative results,” he said. That’s what he and his colleagues attempted to do in their recent trial. “We didn’t just look at clinical results; we’re interrogating the actual tumor environment to understand what worked and didn’t and how to tweak that for the next trial.”

Kwak and his colleagues found, for instance, that the vaccine had a greater effect on B cell–derived tumor cells than on cells of plasma origin, so “the most rational design for the next iteration is to combine the vaccine with agents that work directly against plasma cells,” he explained.

As for what’s next, Kwak said: “We’re just focused on trying to do good science and understand. We’ve seen glimpses of success. That’s where we are.”

A version of this article first appeared on Medscape.com.

Vaccines for treating and preventing cancer have long been considered a holy grail in oncology.

But aside from a few notable exceptions — including the human papillomavirus (HPV) vaccine, which has dramatically reduced the incidence of HPV-related cancers, and a Bacillus Calmette-Guerin vaccine, which helps prevent early-stage bladder cancer recurrence — most have failed to deliver.

Following a string of disappointments over the past decade, recent advances in the immunotherapy space are bringing renewed hope for progress.

In an American Association for Cancer Research (AACR) series earlier in 2024, Catherine J. Wu, MD, predicted big strides for cancer vaccines, especially for personalized vaccines that target patient-specific neoantigens — the proteins that form on cancer cells — as well as vaccines that can treat diverse tumor types.

“A focus on neoantigens that arise from driver mutations in different tumor types could allow us to make progress in creating off-the-shelf vaccines,” said Wu, the Lavine Family Chair of Preventative Cancer Therapies at Dana-Farber Cancer Institute and a professor of medicine at Harvard Medical School, both in Boston, Massachusetts.

A prime example is a personalized, messenger RNA (mRNA)–based vaccine designed to prevent melanoma recurrence. The mRNA-4157 vaccine encodes up to 34 different patient-specific neoantigens.

“This is one of the most exciting developments in modern cancer therapy,” said Lawrence Young, a virologist and professor of molecular oncology at the University of Warwick, Coventry, England, who commented on the investigational vaccine via the UK-based Science Media Centre.

Other promising options are on the horizon as well. In August, BioNTech announced a phase 1 global trial to study BNT116 — a vaccine to treat non–small cell lung cancer (NSCLC). BNT116, like mRNA-4157, targets specific antigens in the lung cancer cells.

“This technology is the next big phase of cancer treatment,” Siow Ming Lee, MD, a consultant medical oncologist at University College London Hospitals in England, which is leading the UK trial for the lung cancer and melanoma vaccines, told The Guardian. “We are now entering this very exciting new era of mRNA-based immunotherapy clinical trials to investigate the treatment of lung cancer.”

Still, these predictions have a familiar ring. While the prospects are exciting, delivering on them is another story. There are simply no guarantees these strategies will work as hoped.

 

Then: Where We Were

Cancer vaccine research began to ramp up in the 2000s, and in 2006, the first-generation HPV vaccine, Gardasil, was approved. Gardasil prevents infection from four strains of HPV that cause about 80% of cervical cancer cases.

In 2010, the Food and Drug Administration approved sipuleucel-T, the first therapeutic cancer vaccine, which improved overall survival in patients with hormone-refractory prostate cancer.

Researchers predicted this approval would “pave the way for developing innovative, next generation of vaccines with enhanced antitumor potency.”

In a 2015 AACR research forecast report, Drew Pardoll, MD, PhD, co-director of the Cancer Immunology and Hematopoiesis Program at Johns Hopkins University, Baltimore, Maryland, said that “we can expect to see encouraging results from studies using cancer vaccines.”

Despite the excitement surrounding cancer vaccines alongside a few successes, the next decade brought a longer string of late-phase disappointments.

In 2016, the phase 3 ACT IV trial of a therapeutic vaccine to treat glioblastoma multiforme (CDX-110) was terminated after it failed to demonstrate improved survival.

In 2017, a phase 3 trial of the therapeutic pancreatic cancer vaccine, GVAX, was stopped early for lack of efficacy.

That year, an attenuated Listeria monocytogenes vaccine to treat pancreatic cancer and mesothelioma also failed to come to fruition. In late 2017, concerns over listeria infections prompted Aduro Biotech to cancel its listeria-based cancer treatment program.

In 2018, a phase 3 trial of belagenpumatucel-L, a therapeutic NSCLC vaccine, failed to demonstrate a significant improvement in survival and further study was discontinued.

And in 2019, a vaccine targeting MAGE-A3, a cancer-testis antigen present in multiple tumor types, failed to meet endpoints for improved survival in a phase 3 trial, leading to discontinuation of the vaccine program.

But these disappointments and failures are normal parts of medical research and drug development and have allowed for incremental advances that helped fuel renewed interest and hope for cancer vaccines, when the timing was right, explained vaccine pioneer Larry W. Kwak, MD, PhD, deputy director of the Comprehensive Cancer Center at City of Hope, Duarte, California.

When it comes to vaccine progress, timing makes a difference. In 2011, Kwak and colleagues published promising phase 3 trial results on a personalized vaccine. The vaccine was a patient-specific tumor-derived antigen for patients with follicular lymphoma in their first remission following chemotherapy. Patients who received the vaccine demonstrated significantly longer disease-free survival.

But, at the time, personalized vaccines faced strong headwinds due, largely, to high costs, and commercial interest failed to materialize. “That’s been the major hurdle for a long time,” said Kwak.

Now, however, interest has returned alongside advances in technology and research. The big shift has been the emergence of lower-cost rapid-production mRNA and DNA platforms and a better understanding of how vaccines and potent immune stimulants, like checkpoint inhibitors, can work together to improve outcomes, he explained.

“The timing wasn’t right” back then, Kwak noted. “Now, it’s a different environment and a different time.”

 

A Turning Point?

Indeed, a decade later, cancer vaccine development appears to be headed in a more promising direction.

Among key cancer vaccines to watch is the mRNA-4157 vaccine, developed by Merck and Moderna, designed to prevent melanoma recurrence. In a recent phase 2 study, patients receiving the mRNA-4157 vaccine alongside pembrolizumab had nearly half the risk for melanoma recurrence or death at 3 years compared with those receiving pembrolizumab alone. Investigators are now evaluating the vaccine in a global phase 3 study in patients with high-risk, stage IIB to IV melanoma following surgery.

Another one to watch is the BNT116 NSCLC vaccine from BioNTech. This vaccine presents the immune system with NSCLC tumor markers to encourage the body to fight cancer cells expressing those markers while ignoring healthy cells. BioNTech also launched a global clinical trial for its vaccine this year.

Other notables include a pancreatic cancer mRNA vaccine, which has shown promising early results in a small trial of 16 patients. Of 16 patients who received the vaccine alongside chemotherapy and after surgery and immunotherapy, 8 responded. Of these eight, six remained recurrence free at 3 years. Investigators noted that the vaccine appeared to stimulate a durable T-cell response in patients who responded.

Kwak has also continued his work on lymphoma vaccines. In August, his team published promising first-in-human data on the use of personalized neoantigen vaccines as an early intervention in untreated patients with lymphoplasmacytic lymphoma. Among nine asymptomatic patients who received the vaccine, all achieved stable disease or better, with no dose-limiting toxicities. One patient had a minor response, and the median time to progression was greater than 72 months.

“The current setting is more for advanced disease,” Kwak explained. “It’s a tougher task, but combined with checkpoint blockade, it may be potent enough to work.” 

Still, caution is important. Despite early promise, it’s too soon to tell which, if any, of these investigational vaccines will pan out in the long run. Like investigational drugs, cancer vaccines may show big promising initially but then fail in larger trials.

One key to success, according to Kwak, is to design trials so that even negative results will inform next steps.

But, he noted, failures in large clinical trials will “put a chilling effect on cancer vaccine research again.”

“That’s what keeps me up at night,” he said. “We know the science is fundamentally sound and we have seen glimpses over decades of research that cancer vaccines can work, so it’s really just a matter of tweaking things to optimize trial design.”

Companies tend to design trials to test if a vaccine works or not, without trying to understand why, he said.

“What we need to do is design those so that we can learn from negative results,” he said. That’s what he and his colleagues attempted to do in their recent trial. “We didn’t just look at clinical results; we’re interrogating the actual tumor environment to understand what worked and didn’t and how to tweak that for the next trial.”

Kwak and his colleagues found, for instance, that the vaccine had a greater effect on B cell–derived tumor cells than on cells of plasma origin, so “the most rational design for the next iteration is to combine the vaccine with agents that work directly against plasma cells,” he explained.

As for what’s next, Kwak said: “We’re just focused on trying to do good science and understand. We’ve seen glimpses of success. That’s where we are.”

A version of this article first appeared on Medscape.com.

Vaccines for treating and preventing cancer have long been considered a holy grail in oncology.

But aside from a few notable exceptions — including the human papillomavirus (HPV) vaccine, which has dramatically reduced the incidence of HPV-related cancers, and a Bacillus Calmette-Guerin vaccine, which helps prevent early-stage bladder cancer recurrence — most have failed to deliver.

Following a string of disappointments over the past decade, recent advances in the immunotherapy space are bringing renewed hope for progress.

In an American Association for Cancer Research (AACR) series earlier in 2024, Catherine J. Wu, MD, predicted big strides for cancer vaccines, especially for personalized vaccines that target patient-specific neoantigens — the proteins that form on cancer cells — as well as vaccines that can treat diverse tumor types.

“A focus on neoantigens that arise from driver mutations in different tumor types could allow us to make progress in creating off-the-shelf vaccines,” said Wu, the Lavine Family Chair of Preventative Cancer Therapies at Dana-Farber Cancer Institute and a professor of medicine at Harvard Medical School, both in Boston, Massachusetts.

A prime example is a personalized, messenger RNA (mRNA)–based vaccine designed to prevent melanoma recurrence. The mRNA-4157 vaccine encodes up to 34 different patient-specific neoantigens.

“This is one of the most exciting developments in modern cancer therapy,” said Lawrence Young, a virologist and professor of molecular oncology at the University of Warwick, Coventry, England, who commented on the investigational vaccine via the UK-based Science Media Centre.

Other promising options are on the horizon as well. In August, BioNTech announced a phase 1 global trial to study BNT116 — a vaccine to treat non–small cell lung cancer (NSCLC). BNT116, like mRNA-4157, targets specific antigens in the lung cancer cells.

“This technology is the next big phase of cancer treatment,” Siow Ming Lee, MD, a consultant medical oncologist at University College London Hospitals in England, which is leading the UK trial for the lung cancer and melanoma vaccines, told The Guardian. “We are now entering this very exciting new era of mRNA-based immunotherapy clinical trials to investigate the treatment of lung cancer.”

Still, these predictions have a familiar ring. While the prospects are exciting, delivering on them is another story. There are simply no guarantees these strategies will work as hoped.

 

Then: Where We Were

Cancer vaccine research began to ramp up in the 2000s, and in 2006, the first-generation HPV vaccine, Gardasil, was approved. Gardasil prevents infection from four strains of HPV that cause about 80% of cervical cancer cases.

In 2010, the Food and Drug Administration approved sipuleucel-T, the first therapeutic cancer vaccine, which improved overall survival in patients with hormone-refractory prostate cancer.

Researchers predicted this approval would “pave the way for developing innovative, next generation of vaccines with enhanced antitumor potency.”

In a 2015 AACR research forecast report, Drew Pardoll, MD, PhD, co-director of the Cancer Immunology and Hematopoiesis Program at Johns Hopkins University, Baltimore, Maryland, said that “we can expect to see encouraging results from studies using cancer vaccines.”

Despite the excitement surrounding cancer vaccines alongside a few successes, the next decade brought a longer string of late-phase disappointments.

In 2016, the phase 3 ACT IV trial of a therapeutic vaccine to treat glioblastoma multiforme (CDX-110) was terminated after it failed to demonstrate improved survival.

In 2017, a phase 3 trial of the therapeutic pancreatic cancer vaccine, GVAX, was stopped early for lack of efficacy.

That year, an attenuated Listeria monocytogenes vaccine to treat pancreatic cancer and mesothelioma also failed to come to fruition. In late 2017, concerns over listeria infections prompted Aduro Biotech to cancel its listeria-based cancer treatment program.

In 2018, a phase 3 trial of belagenpumatucel-L, a therapeutic NSCLC vaccine, failed to demonstrate a significant improvement in survival and further study was discontinued.

And in 2019, a vaccine targeting MAGE-A3, a cancer-testis antigen present in multiple tumor types, failed to meet endpoints for improved survival in a phase 3 trial, leading to discontinuation of the vaccine program.

But these disappointments and failures are normal parts of medical research and drug development and have allowed for incremental advances that helped fuel renewed interest and hope for cancer vaccines, when the timing was right, explained vaccine pioneer Larry W. Kwak, MD, PhD, deputy director of the Comprehensive Cancer Center at City of Hope, Duarte, California.

When it comes to vaccine progress, timing makes a difference. In 2011, Kwak and colleagues published promising phase 3 trial results on a personalized vaccine. The vaccine was a patient-specific tumor-derived antigen for patients with follicular lymphoma in their first remission following chemotherapy. Patients who received the vaccine demonstrated significantly longer disease-free survival.

But, at the time, personalized vaccines faced strong headwinds due, largely, to high costs, and commercial interest failed to materialize. “That’s been the major hurdle for a long time,” said Kwak.

Now, however, interest has returned alongside advances in technology and research. The big shift has been the emergence of lower-cost rapid-production mRNA and DNA platforms and a better understanding of how vaccines and potent immune stimulants, like checkpoint inhibitors, can work together to improve outcomes, he explained.

“The timing wasn’t right” back then, Kwak noted. “Now, it’s a different environment and a different time.”

 

A Turning Point?

Indeed, a decade later, cancer vaccine development appears to be headed in a more promising direction.

Among key cancer vaccines to watch is the mRNA-4157 vaccine, developed by Merck and Moderna, designed to prevent melanoma recurrence. In a recent phase 2 study, patients receiving the mRNA-4157 vaccine alongside pembrolizumab had nearly half the risk for melanoma recurrence or death at 3 years compared with those receiving pembrolizumab alone. Investigators are now evaluating the vaccine in a global phase 3 study in patients with high-risk, stage IIB to IV melanoma following surgery.

Another one to watch is the BNT116 NSCLC vaccine from BioNTech. This vaccine presents the immune system with NSCLC tumor markers to encourage the body to fight cancer cells expressing those markers while ignoring healthy cells. BioNTech also launched a global clinical trial for its vaccine this year.

Other notables include a pancreatic cancer mRNA vaccine, which has shown promising early results in a small trial of 16 patients. Of 16 patients who received the vaccine alongside chemotherapy and after surgery and immunotherapy, 8 responded. Of these eight, six remained recurrence free at 3 years. Investigators noted that the vaccine appeared to stimulate a durable T-cell response in patients who responded.

Kwak has also continued his work on lymphoma vaccines. In August, his team published promising first-in-human data on the use of personalized neoantigen vaccines as an early intervention in untreated patients with lymphoplasmacytic lymphoma. Among nine asymptomatic patients who received the vaccine, all achieved stable disease or better, with no dose-limiting toxicities. One patient had a minor response, and the median time to progression was greater than 72 months.

“The current setting is more for advanced disease,” Kwak explained. “It’s a tougher task, but combined with checkpoint blockade, it may be potent enough to work.” 

Still, caution is important. Despite early promise, it’s too soon to tell which, if any, of these investigational vaccines will pan out in the long run. Like investigational drugs, cancer vaccines may show big promising initially but then fail in larger trials.

One key to success, according to Kwak, is to design trials so that even negative results will inform next steps.

But, he noted, failures in large clinical trials will “put a chilling effect on cancer vaccine research again.”

“That’s what keeps me up at night,” he said. “We know the science is fundamentally sound and we have seen glimpses over decades of research that cancer vaccines can work, so it’s really just a matter of tweaking things to optimize trial design.”

Companies tend to design trials to test if a vaccine works or not, without trying to understand why, he said.

“What we need to do is design those so that we can learn from negative results,” he said. That’s what he and his colleagues attempted to do in their recent trial. “We didn’t just look at clinical results; we’re interrogating the actual tumor environment to understand what worked and didn’t and how to tweak that for the next trial.”

Kwak and his colleagues found, for instance, that the vaccine had a greater effect on B cell–derived tumor cells than on cells of plasma origin, so “the most rational design for the next iteration is to combine the vaccine with agents that work directly against plasma cells,” he explained.

As for what’s next, Kwak said: “We’re just focused on trying to do good science and understand. We’ve seen glimpses of success. That’s where we are.”

A version of this article first appeared on Medscape.com.

The Food and Drug Administration (FDA) has granted accelerated approval to zenocutuzumab-zbco (Bizengri, Merus) as an intravenous infusion for the treatment of certain adults with non–small cell lung cancer (NSCLC) or pancreatic adenocarcinoma.

Specifically, the systemic agent was approved for those with advanced, unresectable, or metastatic NSCLC or pancreatic adenocarcinoma harboring a neuregulin 1 (NRG1) gene fusion who progress on or after prior systemic therapy, according to the FDA.

The approval, based on findings from the multicenter, open-label eNRGy study, is the first from the FDA for a systemic therapy in this setting. In the multicohort study, treatment was associated with an overall response rate of 33% and 40% in 64 patients with NSCLC and 40 patients with pancreatic adenocarcinoma, respectively. Median duration of response was 7.4 months in the NSCLC patients and ranged from 3.7 to 16.6 months in those with pancreatic adenocarcinoma.

Adverse reactions occurring in at least 10% of patients included diarrhea, musculoskeletal pain, fatigue, nausea, infusion-related reactions, dyspnea, rash, constipation, vomiting, abdominal pain, and edema. Grade 3 or 4 laboratory abnormalities occurring in at least 10% of patients included increased gamma-glutamyl transferase and decreased hemoglobin, sodium, and platelets.

“The Personalized Medicine Coalition applauds the approval of BIZENGRI®,” Edward Abrahams, president of the Personalized Medicine Coalition, a Washington-based education and advocacy organization, stated in a press release from Merus. “In keeping with the growing number of personalized medicines on the market today, BIZENGRI® offers the only approved NRG1+ therapy for patients with these difficult-to-treat cancers.”

The agent is expected to be available for use in the “coming weeks,” according to Merus.

“The FDA approval of BIZENGRI® marks an important milestone for patients with pancreatic adenocarcinoma or NSCLC that is advanced unresectable or metastatic and harbors the NRG1 gene fusion,” noted Alison Schram, MD, an attending medical oncologist in the Early Drug Development Service at Memorial Sloan Kettering Cancer Center, New York City, and a principal investigator for the ongoing eNRGy trial. “I have seen firsthand how treatment with BIZENGRI® can deliver clinically meaningful outcomes for patients.”

Prescribing information for zenocutuzumab-zbco includes a Boxed Warning for embryo-fetal toxicity. The recommended treatment dose is 750 mg every 2 weeks until disease progression or unacceptable toxicity.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration (FDA) has granted accelerated approval to zenocutuzumab-zbco (Bizengri, Merus) as an intravenous infusion for the treatment of certain adults with non–small cell lung cancer (NSCLC) or pancreatic adenocarcinoma.

Specifically, the systemic agent was approved for those with advanced, unresectable, or metastatic NSCLC or pancreatic adenocarcinoma harboring a neuregulin 1 (NRG1) gene fusion who progress on or after prior systemic therapy, according to the FDA.

The approval, based on findings from the multicenter, open-label eNRGy study, is the first from the FDA for a systemic therapy in this setting. In the multicohort study, treatment was associated with an overall response rate of 33% and 40% in 64 patients with NSCLC and 40 patients with pancreatic adenocarcinoma, respectively. Median duration of response was 7.4 months in the NSCLC patients and ranged from 3.7 to 16.6 months in those with pancreatic adenocarcinoma.

Adverse reactions occurring in at least 10% of patients included diarrhea, musculoskeletal pain, fatigue, nausea, infusion-related reactions, dyspnea, rash, constipation, vomiting, abdominal pain, and edema. Grade 3 or 4 laboratory abnormalities occurring in at least 10% of patients included increased gamma-glutamyl transferase and decreased hemoglobin, sodium, and platelets.

“The Personalized Medicine Coalition applauds the approval of BIZENGRI®,” Edward Abrahams, president of the Personalized Medicine Coalition, a Washington-based education and advocacy organization, stated in a press release from Merus. “In keeping with the growing number of personalized medicines on the market today, BIZENGRI® offers the only approved NRG1+ therapy for patients with these difficult-to-treat cancers.”

The agent is expected to be available for use in the “coming weeks,” according to Merus.

“The FDA approval of BIZENGRI® marks an important milestone for patients with pancreatic adenocarcinoma or NSCLC that is advanced unresectable or metastatic and harbors the NRG1 gene fusion,” noted Alison Schram, MD, an attending medical oncologist in the Early Drug Development Service at Memorial Sloan Kettering Cancer Center, New York City, and a principal investigator for the ongoing eNRGy trial. “I have seen firsthand how treatment with BIZENGRI® can deliver clinically meaningful outcomes for patients.”

Prescribing information for zenocutuzumab-zbco includes a Boxed Warning for embryo-fetal toxicity. The recommended treatment dose is 750 mg every 2 weeks until disease progression or unacceptable toxicity.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration (FDA) has granted accelerated approval to zenocutuzumab-zbco (Bizengri, Merus) as an intravenous infusion for the treatment of certain adults with non–small cell lung cancer (NSCLC) or pancreatic adenocarcinoma.

Specifically, the systemic agent was approved for those with advanced, unresectable, or metastatic NSCLC or pancreatic adenocarcinoma harboring a neuregulin 1 (NRG1) gene fusion who progress on or after prior systemic therapy, according to the FDA.

The approval, based on findings from the multicenter, open-label eNRGy study, is the first from the FDA for a systemic therapy in this setting. In the multicohort study, treatment was associated with an overall response rate of 33% and 40% in 64 patients with NSCLC and 40 patients with pancreatic adenocarcinoma, respectively. Median duration of response was 7.4 months in the NSCLC patients and ranged from 3.7 to 16.6 months in those with pancreatic adenocarcinoma.

Adverse reactions occurring in at least 10% of patients included diarrhea, musculoskeletal pain, fatigue, nausea, infusion-related reactions, dyspnea, rash, constipation, vomiting, abdominal pain, and edema. Grade 3 or 4 laboratory abnormalities occurring in at least 10% of patients included increased gamma-glutamyl transferase and decreased hemoglobin, sodium, and platelets.

“The Personalized Medicine Coalition applauds the approval of BIZENGRI®,” Edward Abrahams, president of the Personalized Medicine Coalition, a Washington-based education and advocacy organization, stated in a press release from Merus. “In keeping with the growing number of personalized medicines on the market today, BIZENGRI® offers the only approved NRG1+ therapy for patients with these difficult-to-treat cancers.”

The agent is expected to be available for use in the “coming weeks,” according to Merus.

“The FDA approval of BIZENGRI® marks an important milestone for patients with pancreatic adenocarcinoma or NSCLC that is advanced unresectable or metastatic and harbors the NRG1 gene fusion,” noted Alison Schram, MD, an attending medical oncologist in the Early Drug Development Service at Memorial Sloan Kettering Cancer Center, New York City, and a principal investigator for the ongoing eNRGy trial. “I have seen firsthand how treatment with BIZENGRI® can deliver clinically meaningful outcomes for patients.”

Prescribing information for zenocutuzumab-zbco includes a Boxed Warning for embryo-fetal toxicity. The recommended treatment dose is 750 mg every 2 weeks until disease progression or unacceptable toxicity.

A version of this article first appeared on Medscape.com.