On December 9, 2020 Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a pharmaceutical company focused on the development of targeted therapies for difficult-to-treat hematological diseases, reported that the pivotal phase 2 HORIZON study evaluating intravenous melflufen (INN melphalan flufenamide) in combination with dexamethasone in relapsed refractory multiple myeloma, has been published in the peer-reviewed Journal of Clinical Oncology (Press release, Oncopeptides, DEC 9, 2020, View Source [SID1234572521]).

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The phase 2 HORIZON data are the basis for the ongoing priority review of the New Drug Application to the US Food and Drug Administration FDA, for accelerated approval of melflufen in combination with dexamethasone in triple-class refractory multiple myeloma patients, who are refractory to at least one proteasome inhibitor, one immunomodulatory drug and one anti-CD38 monoclonal antibody.

"The results from the HORIZON study demonstrate that melflufen in combination with dexamethasone, has a potential to provide a therapeutic option for patients who are difficult to treat and have a poor prognosis, including patients with triple class refractory myeloma and patients with extramedullary disease", says Klaas Bakker, MD, PhD, Chief Medical Officer, Oncopeptides AB. "These patients have limited, or no treatment options left. The introduction of a new treatment class may represent a potentially important alternative".

The phase 2 HORIZON study is a pivotal, single-arm, multicenter, phase 2 study evaluating the safety and efficacy of melflufen in combination with dexamethasone in patients with relapsed refractory multiple myeloma. The study included 157 heavily pretreated patients, who had received >2 earlier lines of therapy with immunomodulatory drugs and proteasome inhibitors and were refractory to pomalidomide and/or daratumumab. The HORIZON study population includes subgroups of patients who were triple?class refractory and/or had extramedullary disease and/or had cytogenetic high?risk features.

Summary of results:

30 Intention to Treat (n=157) Triple Class Refractory (n=119) Extra Medullary Disease (n=55)
Overall Response Rate (ORR) 29% 26% 24%
Median Progression Free Survival (PFS)) 4.2 months 3.9 months 2.9 months
Median Overall Survival (OS) 11.6 months 11.2 months 6.5 months
Responding patients n=45 n=31 n=13
Median Duration of Response (DOR) 5.5 months 4.4 months 5.5 months
Median Progression Free Survival (PFS) 8.5 months 8.5 months 17.3 months
The publication is available on; View Source

The information in this press release was submitted for publication on December 9, 2020 at 22:00 (CET).

About melflufen
Melflufen (INN melphalan flufenamide) is a first in class peptide-drug conjugate (PDC) that targets aminopeptidases and rapidly releases alkylating agents into tumor cells. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is immediately hydrolyzed by peptidases to release an entrapped hydrophilic alkylator payload. Aminopeptidases are overexpressed in tumor cells and are even more pronounced in advanced cancers and tumors with a high mutational burden. In vitro, melflufen is 50-fold more potent in myeloma cells than the alkylator payload itself due to the increased intracellular alkylator concentration. Melflufen displays cytotoxic activity against myeloma cell lines resistant to other treatments, including alkylators, and has also demonstrated inhibition of DNA repair induction and angiogenesis in preclinical studies. In the pivotal phase 2 HORIZON study melflufen plus dexamethasone demonstrated encouraging efficacy and a clinically manageable safety profile in heavily pretreated patients with relapsed refractory multiple myeloma, with primarily hematologic Adverse Events (AE) and a low incidence of non-hematologic AEs.

On December 9, 2020 Lantheus Holdings, Inc. (NASDAQ: LNTH) (the Company), the parent company of Lantheus Medical Imaging, Inc. and Progenics Pharmaceuticals, Inc., and a global leader in the development, manufacture and commercialization of innovative diagnostic and therapeutic agents and products, reported that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for PyLTM (18F-DCFPyL), a prostate specific membrane antigen (PSMA)-targeted positron emission tomography (PET) imaging agent for prostate cancer (Press release, Lantheus Medical Imaging, DEC 9, 2020, View Source [SID1234572540]).

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The FDA granted Priority Review for the PyL NDA and assigned a Prescription Drug User Fee Act (PDUFA) action date of May 28, 2021. The FDA has also indicated in the NDA filing acceptance notification that it is not currently planning to hold an advisory committee meeting to discuss the application.

"We are pleased that the FDA has accepted our PyL NDA for review and granted our application Priority Review, which is a significant milestone for Lantheus. We believe that there is a significant unmet need for reliable, targeted imaging in prostate cancer, particularly in the high risk and biochemically recurrent populations," said Istvan Molnar, MD, Chief Medical Officer of Lantheus. "We look forward to working with the FDA during the NDA review process with the goal of bringing PyL to patients and physicians who need it."

The PyL NDA is supported by data from two Company-sponsored pivotal studies (OSPREY and CONDOR) designed to establish the safety and diagnostic performance of PyL imaging across the prostate cancer disease continuum. Results from OSPREY Cohort A demonstrated improvement in specificity and positive predictive value (PPV) of PyL PET imaging over conventional imaging in men with high risk prostate cancer. OSPREY Cohort B and CONDOR studied men with prostate cancer in various disease states, including biochemical recurrent, hormone sensitive, non-metastatic castrate resistant, and metastatic castrate resistant. OSPREY Cohort B demonstrated the sensitivity of PyL PET imaging in detecting metastatic lesions, while CONDOR, in patients with biochemical recurrent prostate cancer and non-informative baseline findings, demonstrated PyL’s high correct localization rate and detection rate, including in patients with low PSA values. In the CONDOR study, 63.9% of patients had a change in intended disease management plans due to the PyL PET imaging results. We believe the results from these two studies, taken as a whole, demonstrate the ability of PyL to reliably detect and localize disease and could enable more appropriate patient management.

PyL has been administered in approximately 3,500 subjects globally, including the two Company-sponsored pivotal studies, multiple investigator sponsored studies, as well as clinical use reported in the literature. Across these studies, PyL has shown an attractive safety profile.

About PyL for PET Imaging of Prostate Cancer

PyL (also known as 18F-DCFPyL) is an investigational fluorinated PSMA-targeted PET imaging agent that enables visualization of localized prostate cancer as well as bone and soft tissue metastases to determine the presence or absence of recurrent and/or metastatic prostate cancer.

About OSPREY

The Phase 2/3 OSPREY trial assessed the diagnostic performance of PyL to detect prostate cancer in pelvic lymph nodes in subjects with high risk, locally advanced prostate cancer (Cohort A) and distant metastases in subjects with metastatic or recurrent prostate cancer (Cohort B). In the trial, the diagnostic performance of PyL in detecting disease in pelvic lymph nodes (Cohort A) showed specificity of 96-99%, sensitivity of 31-42%, and PPV of 78-91% although the trial did not meet one of its the primary endpoints. In the metastatic or recurrent prostate cancer setting (Cohort B), PyL exhibited sensitivity of 93-99% and PPV of 81-88% in detecting metastatic lesions. Overall, PyL demonstrated high diagnostic performance in reliably detecting nodal and distant metastatic prostate cancer.

About CONDOR

The Phase 3 CONDOR trial evaluated the diagnostic performance and clinical impact of PyL in men with biochemical recurrence of prostate cancer and uninformative baseline imaging based on conventional modalities. The CONDOR trial achieved its primary endpoint, with a correct localization rate (CLR) of 84.8% to 87.0% among the three blinded independent readers (the lower bound of the 95% confidence intervals ranging from 77.8% to 80.4%). CLR is based on positive predictive value, defined as the percentage of subjects with a one-to-one correspondence between localization of at least one lesion identified on PyL PET/CT and a composite truth standard comprised of histopathology, conventional imaging and/or changes in PSA levels following radiation therapy. 63.9% of subjects in the CONDOR trial had a change in intended disease management plans due to PyL imaging results, a key secondary endpoint of the trial. The changes to treatment management plans due to the PyL results included salvage local therapy to systemic therapy, observation to initiating therapy, noncurative systemic therapy to salvage local therapy, and planned treatment to observation.

About Prostate Cancer

Prostate cancer is the second most common form of cancer affecting men in the United States — an estimated one in nine men will be diagnosed with prostate cancer in his lifetime. The American Cancer Society estimates that each year 192,000 new cases of prostate cancer will be diagnosed, and 33,000 men will die of the disease. Approximately 3.2 million men in the United States currently count themselves among prostate cancer survivors.1

On December 9, 2020 Exact Sciences Corp. (NASDAQ: EXAS) reported that data from the Rx for Positive Node, Endocrine Responsive Breast Cancer, or RxPONDER, trial successfully defined the benefit of chemotherapy in early-stage, node-positive breast cancer patients with Oncotype DX Breast Recurrence Score results of 0 to 25. First results from the study, led by the independent SWOG Cancer Research Network, and sponsored by the National Cancer Institute (NCI), identified the majority of women with 1-3 positive nodes who received no benefit from chemotherapy (Press release, Exact Sciences, DEC 9, 2020, View Source [SID1234572556]).i The data will be presented on December 10 at the 2020 San Antonio Breast Cancer Symposium (SABCS).

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RxPONDER showed a different effect of chemotherapy based on Recurrence Score results for postmenopausal and premenopausal women. Postmenopausal women with Recurrence Score results 0-25 were not observed to show benefit from chemotherapy and may avoid the associated side effects of the treatment. Importantly, no chemotherapy benefit was observed regardless of the number of affected nodes, tumor grade, or size. Two-thirds of the women in the trial were postmenopausal.

The first results also demonstrated, after a median of five years of follow-up, that premenopausal women with Recurrence Score results 0-25 were observed to have a statistically significant chemotherapy benefit, with an average improvement in distant recurrence rates at 5 years of 3%.

Approximately 85% of women with node- positive disease have Recurrence Score results of 0 to 25. ii Postmenopausal and premenopausal women with Recurrence Score results 26-100 were not included in the study because investigators reviewed prior studies and determined that this patient group had chemotherapy benefit. The SWOG investigators intend to publish the detailed RxPONDER results in a peer-reviewed publication.

"Every day in clinics around the world, physicians wrestle with the question of how to best treat women with this common form of breast cancer," said study lead author Kevin Kalinsky, MD, a long-time SWOG investigator and director of the Glenn Family Breast Center at the Winship Cancer Institute of Emory University. "These results are practice changing and demonstrate that the great majority of postmenopausal women can be spared unnecessary chemotherapy and receive only hormone therapy. This should bring more clarity to physicians and some relief for patients."

Approximately 25% of patients diagnosed with hormone receptor (HR)-positive, HER2-negative early breast cancer have tumor that has spread to their lymph nodes and two out of three are postmenopausal.iii The vast majority of these patients currently receive chemotherapy.iv

"With the RxPONDER and TAILORx trials, there is now definitive and undeniable clarity on who does and who does not benefit from chemotherapy among early-stage breast cancer patients, with either node-negative or node positive disease," said Steven Shak, MD, chief medical officer at Exact Sciences. "These long-awaited results, which continue to build on the body of evidence supporting the role of the Oncotype DX test in shaping clinical practice, are estimated to impact tens of thousands of women worldwide."

One of the largest clinical trials in women with node-positive HR+, HER2- early breast cancer, RxPONDER is a prospective, randomized Phase III study conducted at 632 sites in nine countries – the United States, Canada, Mexico, Colombia, Ireland, France, Spain, South Korea, and Saudi Arabia. The study enrolled more than 5,000 women with up to three positive nodes. Women with a Recurrence Score result 0-25 were randomized for treatment with hormone therapy alone or chemotherapy followed by hormone therapy. Randomized patients were stratified based on their Recurrence Score result, menopausal status, and the type of lymph node surgery.

The use of the Oncotype DX test in early-stage breast cancer is supported by prospective outcomes from more than 17,000 patients with node-positive disease and more than 83,000 patients with node-negative disease, including the TAILORx study. Results from TAILORx, published in 2018, showed that the Oncotype DX test identifies the vast majority of women with node-negative disease who receive no substantial benefit from chemotherapy (approximately 80%), as well as the important minority for whom chemotherapy can be life-saving.

About Oncotype DX
The Oncotype DX portfolio of breast, colon and prostate cancer tests applies advanced genomic science to reveal the unique biology of a tumor in order to optimize cancer treatment decisions. In breast cancer, the Oncotype DX Breast Recurrence Score test is the only test that has been shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer. Additionally, the Oncotype DX Breast DCIS Score test predicts the likelihood of recurrence in a pre-invasive form of breast cancer called DCIS. In prostate cancer, the Oncotype DX Genomic Prostate Score test predicts disease aggressiveness and further clarifies the current and future risk of the cancer prior to treatment intervention, and the Oncotype DX AR-V7 Nucleus Detect test helps determine which patients with metastatic castration-resistant prostate cancer (mCRPC) are resistant to androgen receptor (AR)-targeted therapies. The Oncotype DX AR-V7 Nucleus Detect test is performed by Epic Sciences at its centralized, CLIA-certified laboratory in San Diego and offered exclusively by Exact Sciences. With more than 1 million patients tested in more than 90 countries, the Oncotype DX tests have redefined personalized medicine by making genomics a critical part of cancer diagnosis and treatment. To learn more about Oncotype DX tests, visit www.OncotypeIQ.com, www.MyBreastCancerTreatment.org or www.MyProstateCancerTreatment.org.

On December 9, 2020 Enzo Biochem, Inc. (NYSE:ENZ), a leading biosciences and diagnostics company, reported financial results for the first quarter ended October 31, 2020 and provided a business update on recent corporate and operational developments (Press release, Enzo Biochem, DEC 9, 2020, sec.gov/Archives/edgar/data/316253/000121390020042554/ea131605ex99-1_enzobio.htm [SID1234572813]).

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"During this quarter we exceeded guidance and achieved profitability based on strong results that reflect our strategy to both streamline our operations and expand our product and platform capabilities as an integrated solutions provider of products and services in molecular diagnostics, immunoassays, cytology, and immunohistochemistry," said Elazar Rabbani, PhD., Chairman and Chief Executive Officer of Enzo. "The rapid application of our proprietary GenFlex platform in response to the COVID-19 pandemic is the most recent reflection of the strength and flexibility of our molecular diagnostic capabilities. We have also worked aggressively to expand our operations to focus on more advanced, higher margin market opportunities that reflect the core strengths of Enzo and our team. Our portable microplate reader brings new potential capabilities in point-of-care testing to researchers and healthcare providers while our GoTestMeNow.com online portal represents a major step forward in bringing access to diagnostic testing directly to people who need it conveniently and quickly."

"During this very strong quarter for our company we also added two outstanding industry leaders to our Board of Directors. Drs. Mary Tagliaferri and Ian Walters significantly expand the operational and commercial expertise of our Company’s board, particularly concerning companion diagnostics and other therapeutic opportunities. We continue to execute on our strategy to position Enzo as a leading vertically integrated end–to-end diagnostic company capitalizing on our multi-platform technologies and products now and in a post-COVID-19 environment," said Barry Weiner, President of Enzo.

Operational Highlights:

The Company posted another successful quarter of progress in implementing its strategic plan as a fully integrated end-to-end diagnostic product and service provider while navigating through a range of operational issues during the COVID-19 pandemic. Our first responder staff’s response and commitment has been noteworthy in our achieving these goals.
Enzo rapidly expanded capacity in reagent and consumable supply manufacturing while broadening its menu of tests on its GenFlex molecular diagnostic platform.
The Company continued its strategic operational expansion program including plans to double its facility footprint in Farmingdale, NY.
Enzo completed the launch of the company’s new portable microplate reader for use with its ELISA and assay kits to simplify laboratory workflow and expand POC capabilities in molecular diagnostics.
In response to the continuing demand for improved access to molecular testing, Enzo introduced the GoTestMeNow online portal where consumers can directly order COVID-19 laboratory tests that are physician-approved, with plans to expand its use to other testing needs in 2021.
Customer interest for Enzo’s diagnostic platforms was strong following award of Emergency Use Authorization from the FDA highlighting the Company’s integrated in-house capabilities and ability to develop high throughput sensitive detection platforms. These platforms support rapid scalability of testing for COVID-19 in a model that can be applied to future testing needs in multiple areas including upper respiratory panels, STDs and expanded women’s health panels.
Corporate & Organizational Highlights:

Enzo added industry veterans Mary Tagliaferri, MD and Ian B. Walters, MD to the Company Board of Directors.
The Company planned and executed a range of operational and technological strategies to further its cost efficiencies.
The Company is in the process of evaluating various business opportunities concerning Enzo’s assets and capabilities for investment, partnerships, and commercial relationships.
Financial Highlights:

Following a slow-down of operations at the beginning of the COVID-19 pandemic as a result of a sharp industry-wide fall-off in medical visits and the closure of many customer facilities, Enzo monthly revenues have registered steady gains since April. Quarterly revenues advanced 47% sequentially and 42% year-over-year during the period.
The Company’s current annual revenue run-rate is approximately $115 million, representing more than 50% topline growth on an annualized basis.
Gross margins in the Enzo Clinical Labs division reached 39% in the first quarter.
1Q21 net income was $0.3 million compared to a net loss of $7.6 million in the previous year’s period, representing a $7.9 million improvement over the corresponding period a year ago and a $3.6 million improvement over the prior quarter.
First Quarter 2021 Financial Results

Total first quarter revenue was $28.7 million, compared to $20.2 million in the first quarter last year, an increase of 42%, reflecting continuing expansion of operations and revenue following the slow-down associated with the impact of COVID-19 on the diagnostic testing sector. Consolidated gross margin was 42% compared to 28% a year ago.
Enzo Clinical Lab revenue increased 66% to $21.2 million from $12.8 million in the first quarter 2020 and more than 55% sequentially. The year over year improvement was driven by volume growth to 300,000 accessions in the period versus approximately 200,000 in the previous year’s first quarter. Net revenue per accession increased to more than $69 per accession vs. $62 in the previous year’s period. Clinical services gross margin amounted to 39% up from 14% in the first quarter 2020, primarily due to testing mix as well as from ongoing cost-saving initiatives.
Enzo Life Sciences revenue of $7.4 million increased 27% from $5.8 million in the previous quarter reflecting the beginning of a recovery from the impact of COVID-19 pandemic globally. Gross margin was 49%, compared to 52% in the previous year’s quarter and 46% in the fourth quarter due to product mix and the impact of the COVID-19 pandemic.
Research and development expenses decreased 29% to $0.8 million (3% of total revenues) from $1.1 million, (5% of total revenues), in the year ago period. Selling, general and administrative expenses of $10.0 million (down to 35% of total revenue) declined from $11.1 million (55% of total revenue) in year ago period, 10% lower.
GAAP net income was $0.3 million or $0.01 per share versus a net loss of $3.3 million or ($0.07) per share last quarter and a loss of $7.7 million, or ($0.16) per share, in the year-ago quarter. Adjusted EBITDA in the quarter was $1.0 million compared to an adjusted EBITDA loss of $5.7 million in the previous year’s first quarter. The year-over-year improvement was driven mainly by an increase in gross margin (from COVID-19 testing and lower reagent costs) and lower SG&A expenses from headcount efficiencies, as well as reduced intangibles amortization and travel.
Cash and cash equivalents totaled $46 million at the end of the first quarter, slightly lower than the $48 at the end of the fiscal year due to increases in accounts receivable, inventory, and capital expenditures. Working capital improved to $37 million from $36 million at the end of the fiscal year. As of October 31, 2020, the Company had 47.9 million shares outstanding.
Conference Call and Webcast Information

The Company will host a conference call on Wednesday, December 9, 2020, at 4:30 pm, Eastern Standard Time, to review the operational, corporate, and financial highlights. To participate in the conference call, please dial the following numbers prior to the start of the call or click the webcast link below to participate over the internet:

Domestic: 877-407-0792
International: 201-689-8263
Conference ID: 13713236
Webcast: View Source

A replay of the call will be available via webcast for on-demand listening shortly after completion of the call on the Investor Relations section of the Company’s website, View Source, and will remain available for approximately 90 days. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.

Adjusted Financial Measures

To comply with Regulation G promulgated pursuant to the Sarbanes-Oxley Act, Enzo Biochem attached to this news release and will post to the investor relations section of the Company’s website (View Source) any reconciliation of differences between GAAP and Adjusted financial information that may be required in connection with issuing the Company’s quarterly financial results.

The Company uses EBITDA as a measure of performance to demonstrate earnings exclusive of interest, taxes, depreciation and amortization. Adjustments to EBITDA are for items of a non-recurring nature and are reconciled on the table provided. The Company manages its business based on its operating cash flows. The Company, in its daily management of its business affairs and analysis of its monthly, quarterly and annual performance, makes its decisions based on cash flows, not on the amortization of assets obtained through historical activities. The Company, in managing its current and future affairs, cannot affect the amortization of the intangible assets to any material degree, and therefore uses EBITDA as its primary management guide. Since an outside investor may base its evaluation of the Company’s performance based on the Company’s net loss not its cash flows, there is a limitation to the EBITDA measurement. EBITDA is not, and should not be considered, an alternative to net loss, loss from operations, or any other measure for determining operating performance of liquidity, as determined under accounting principles generally accepted in the United States (GAAP). The most directly comparable GAAP reference in the Company’s case is the removal of interest, taxes, depreciation and amortization.

We refer you to the tables attached to this press release, which includes reconciliation tables of GAAP to Adjusted net income (loss) and EBITDA to Adjusted EBITDA.

On December 9, 2020 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that a clinical update on DANYELZA (naxitamab-gqgk) for the treatment of Refractory/Relapsed High-Risk Neuroblastoma was given at the ESMO (Free ESMO Whitepaper) Immuno-Oncology Virtual Congress 2020, which is being held December 9 through December 12, 2020 (Press release, Y-mAbs Therapeutics, DEC 9, 2020, View Source [SID1234572522]). The DANYELZA data was presented by Dr. Jaume Mora from SJD Barcelona Children’s Hospital, Spain and Dr. Daniel A. Morgenstern from The Hospital for Sick Children, Toronto, Canada.

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In the Company’s Study 201, a total of 95% of the infusions were administered in an outpatient setting. The median infusion time was 37 minutes, and all infusions were completed in less than 2 hours. The adherence rate was deemed very satisfactory, as 98% of the infusions were completed as planned, and the treatments appeared to be generally well tolerated. By independent review, the overall response rate ("ORR") in Study 201 was 68% with 59% complete responses ("CR"). In refractory patients, the ORR was 71% with 64% CR, and in relapsed patients the ORR was 63% with 50% CR.

DANYELZA 40mg/10ml was recently approved by the U.S. FDA. DANYELZA is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. DANYELZA is a humanized, monoclonal antibody that targets the ganglioside GD2, which is highly expressed in various neuroectoderm-derived tumors and sarcomas. During the Company’s Study 201, DANYELZA was primarily administered to patients in an outpatient setting three times in a week and the treatment was repeated every four weeks.

"I am very pleased to see that 98% of the infusions were completed as planned and 95% of the infusions were outpatient. Furthermore, the response rates achieved in these patients, who were either refractory to frontline treatment or had relapsed are notable, as this is a difficult to treat patient population, and the responses are based on antibody treatment alone, and not combination treatment with chemotherapy," said Thomas Gad, founder, Chairman and President.

"We are pleased that Study 201 showed that when DANYELZA is given in an outpatient setting with a high degree of treatment adherence, it addressed a significant unmet medical need in an efficient way. In addition, in Study 201 we saw that the median infusion time was just over half an hour, and when compared to the duration of the first infusion, the subsequent infusion times were generally lower, which we believe is great news for children living with refractory/relapsed high-risk neuroblastoma," said Dr. Claus Moller, Chief Executive Officer.

Researchers at Memorial Sloan Kettering Cancer Center ("MSK") developed DANYELZA, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests related to the compound and Y-mAbs.

About DANYELZA (naxitamab-gqgk)

DANYELZA (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication was approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA includes a Boxed Warning for serious infusion-related reactions, such as cardiac arrest, anaphylaxis, hypotension, bronchospasm and stridor and neurotoxicity, such as severe neuropathic pain, transverse myelitis and reversible posterior leukoencephalopathy syndrome. See full Prescribing Information for complete Boxed Warning and other important safety information.