Y-mAbs Announces Data to be Presented at SIOP 2021

On September 16, 2021 Y-mAbs Therapeutics, Inc. ("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 the acceptance of two presentations at the International Society of Pediatric Oncology ("SIOP") Virtual Annual Congress held October 21 through October 24, 2021 (Press release, Y-mAbs Therapeutics, SEP 16, 2021, View Source [SID1234587834]).

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DANYELZA (naxitamab-gqgk)

The abstracts include the following oral presentation of DANYELZA, the Company’s approved therapy for the treatment of pediatric patients with relapsed or refractory high-risk neuroblastoma, which is currently also being evaluated for the treatment of osteosarcoma and other GD2-positive tumors:

"Naxitamab treatment of refractory/relapsed high-risk neuroblastoma (R/R HR NB); subgroup analysis of updated efficacy and safety data for the registrational Phase II trial", submitted by SJD Barcelona Children’s Hospital in Barcelona, Spain
Omburtamab

The abstracts also include the following poster presentation of omburtamab, the Company’s lead product candidate, which is currently being evaluated for the treatment of patients with CNS/Leptomeningeal metastasis from neuroblastoma, diffuse intrinsic pontine glioma ("DIPG"), and desmoplastic small round cell tumors ("DSRCT"):

"Outcomes of intraventricular 131-I-8H9 and external beam radiotherapy in patients with medulloblastoma, ependymoma, and pineoblastoma," submitted by Memorial Sloan Kettering Cancer Center ("MSK") in New York
Researchers at MSK developed DANYELZA and omburtamab, which are exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests in the compounds.

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 and anaphylaxis, and neurotoxicity, such as severe neuropathic pain and transverse myelitis. See full Prescribing Information for complete Boxed Warning and other important safety information.

PharmaCyte Biotech Announces Positive Results of Biocompatibility Study for FDA

On September 16, 2021 PharmaCyte Biotech, Inc. (NASDAQ: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported the first test results of the biocompatibility studies of its CypCaps clinical trial product candidate (Press release, PharmaCyte Biotech, SEP 16, 2021, View Source [SID1234587833]). These results were from an "In Vitro Complement Activation Study of Empty Cellulose Sulphate Capsules," the same capsules PharmaCyte uses in its treatment for pancreatic cancer.

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PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said, "A number of studies are currently ongoing by independent, third-party CROs on the biocompatibility of our CypCaps pancreatic cancer product candidate and its components. We are pleased to report that we have received the results from the first of these studies showing that, as expected, the capsule material does not activate a major line of the human body’s innate defense – the complement system. This is just one of the biocompatibility studies that are being performed and, in the coming weeks, we expect that the results of other studies will become available."

The complement system consists of multiple proteins, approximately 30 of which are circulating blood proteins that work together to promote immune and inflammatory responses. The complement system’s principal role is to help identify, destroy, and remove foreign pathogens such as bacteria and viruses, as well as damaged cell materials (for example, cells and proteins).

The objective of the ISO 10993-4: 2017 (E) compliant study was to evaluate the complement activation potential of empty cellulose sulphate capsules when mixed with normal human serum complement under in vitro test conditions. The study was performed by a third-party GLP laboratory. That laboratory concluded that empty cellulose sulphate capsules did not activate the complement system under defined experimental conditions based on the results of the study. These results were also supported by statistical comparisons.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, we encourage you to watch the company’s documentary video complete with medical animations at: View Source

New Data Demonstrate Trodelvy® Survival Benefit in Metastatic Triple-Negative Breast Cancer Patients Regardless of Initial HR/HER2 Status

On September 16, 2021 Gilead Sciences, Inc. (Nasdaq: GILD) reported new data from the Phase 3 ASCENT study evaluating Trodelvy (sacituzumab govitecan-hziy) in patients with relapsed or refractory metastatic triple-negative breast cancer (TNBC) who received two or more prior systemic therapies, at least one of them for metastatic disease (Press release, Gilead Sciences, SEP 16, 2021, View Source [SID1234587831]). In a retrospective subgroup analysis, Trodelvy improved progression-free survival (PFS), overall survival (OS) and objective response rate (ORR) compared with chemotherapy chosen by the patients’ physicians in patients who were not initially diagnosed with TNBC. The results were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 from September 16-21, 2021 (Poster #258P).

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"In the metastatic stage of breast cancer, it is not uncommon for people to change from one subtype to another," said Javier Cortés, MD, Head of the International Breast Cancer Center (IBCC), Madrid and Barcelona, Spain. "Roughly one-third of patients with TNBC in the ASCENT study were not originally diagnosed with TNBC, and they still experienced a survival benefit with Trodelvy compared with chemotherapy. For treating physicians, this reinforces Trodelvy’s efficacy in more complex patients."

This analysis included 146 chemotherapy-eligible brain metastasis-negative patients with an original breast cancer diagnosis that was not TNBC, of which 70 received Trodelvy and 76 received physician’s choice of chemotherapy. Among these patients, Trodelvy improved median PFS compared with chemotherapy (4.6 months vs. 2.3 months; HR: 0.48; P=0.0004), median OS (12.4 months vs. 6.7 months; HR: 0.44; P<0.0001) and ORR (31% vs. 4%). Outcomes were similar to those of the overall ASCENT trial population.

The safety profile of Trodelvy in this subgroup was consistent with prior reports from the ASCENT study. Key treatment-related grade ≥3 adverse events for Trodelvy compared to chemotherapy were neutropenia (59% vs. 40%), leukopenia (12% vs. 9%), anemia (8% vs. 7%) and diarrhea (7% vs. 0%). There were no treatment-related deaths with Trodelvy. The Trodelvy U.S. Prescribing Information has a BOXED WARNING for severe or life-threatening neutropenia and severe diarrhea; see below for Important Safety Information.

"Trodelvy is already transforming outcomes for patients with second-line or later metastatic TNBC," said Bill Grossman, MD, PhD, Senior Vice President, Oncology Clinical Research, Gilead Sciences. "Gilead is committed to continued research that further defines the clinical profile of Trodelvy. As such, we are pleased that this sub-analysis in patients not initially diagnosed with TNBC demonstrated similarly strong overall survival, progression-free survival and response rates as were observed in the overall ASCENT study population."

About Triple-Negative Breast Cancer

TNBC is the most aggressive type of breast cancer and accounts for approximately 15% of all breast cancers. TNBC is diagnosed more frequently in younger and premenopausal women and is more prevalent in Black and Hispanic women. TNBC cells do not have estrogen and progesterone receptors and have limited HER2. Due to the nature of TNBC, treatment options are extremely limited compared with other breast cancer types. TNBC has a higher chance of recurrence and metastases than other breast cancer types. The average time to metastatic recurrence for TNBC is approximately 2.6 years compared with 5 years for other breast cancers, and the relative five-year survival rate is much lower. Among women with metastatic TNBC, the five-year survival rate is 12%, compared with 28% for those with other types of metastatic breast cancer.

About the ASCENT Study

The ASCENT study is a global, open-label, randomized Phase 3 study that enrolled more than 500 patients across 230 study locations. The study evaluated the efficacy and safety of Trodelvy compared with a single-agent chemotherapy of the physician’s choice in patients with unresectable, locally advanced or metastatic TNBC who had received at least two prior systemic treatments. Patients were randomized to receive either Trodelvy or a chemotherapy chosen by the patients’ treating physicians. The primary endpoint was PFS (as determined by blinded independent central review) in patients without brain metastases. Secondary endpoints included: PFS for full study population or intention-to-treat (ITT) population, OS in both the ITT population and in the subgroup without brain metastasis, independently determined ORR, duration of response, time to onset of response according to Response Evaluation Criteria in Solid Tumors (RECIST 1.1), quality of life and safety. More information about ASCENT is available at View Source

About Trodelvy

Trodelvy (sacituzumab govitecan-hziy) is a first-in-class antibody and topoisomerase inhibitor conjugate directed to the Trop-2 receptor, a protein overexpressed in multiple types of epithelial tumors, including metastatic TNBC and metastatic UC, where high expression is associated with poor survival and relapse. Beyond the approvals of Trodelvy in the United States, it is also approved in Australia, Switzerland, and the UK for adults with metastatic TNBC. Trodelvy is also under regulatory review in the EU and Canada, as well as in Singapore through our partner Everest Medicines. Trodelvy is also being investigated as a potential treatment for hormone receptor-positive/human epidermal growth factor receptor 2-negative metastatic breast cancer and metastatic non-small cell lung cancer. Additional evaluation across multiple solid tumors is also underway.

In the United States, Trodelvy is indicated for the treatment of:

Adult patients with unresectable locally advanced or metastatic TNBC who have received two or more prior systemic therapies, at least one of them for metastatic disease.
Adult patients with locally advanced or metastatic UC who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor.
U.S. Important Safety Information for Trodelvy

BOXED WARNING: NEUTROPENIA AND DIARRHEA

Severe or life-threatening neutropenia may occur. Withhold Trodelvy for absolute neutrophil count below 1500/mm3 or neutropenic fever. Monitor blood cell counts periodically during treatment. Consider G-CSF for secondary prophylaxis. Initiate anti-infective treatment in patients with febrile neutropenia without delay.
Severe diarrhea may occur. Monitor patients with diarrhea and give fluid and electrolytes as needed. Administer atropine, if not contraindicated, for early diarrhea of any severity. At the onset of late diarrhea, evaluate for infectious causes and, if negative, promptly initiate loperamide. If severe diarrhea occurs, withhold Trodelvy until resolved to ≤Grade 1 and reduce subsequent doses.
CONTRAINDICATIONS

Severe hypersensitivity reaction to Trodelvy.
WARNINGS AND PRECAUTIONS

Neutropenia: Severe, life-threatening, or fatal neutropenia can occur and may require dose modification. Neutropenia occurred in 61% of patients treated with Trodelvy. Grade 3-4 neutropenia occurred in 47% of patients. Febrile neutropenia occurred in 7%. Withhold Trodelvy for absolute neutrophil count below 1500/mm3 on Day 1 of any cycle or neutrophil count below 1000/mm3 on Day 8 of any cycle. Withhold Trodelvy for neutropenic fever.

Diarrhea: Diarrhea occurred in 65% of all patients treated with Trodelvy. Grade 3-4 diarrhea occurred in 12% of patients. One patient had intestinal perforation following diarrhea. Neutropenic colitis occurred in 0.5% of patients. Withhold Trodelvy for Grade 3-4 diarrhea and resume when resolved to ≤Grade 1. At onset, evaluate for infectious causes and if negative, promptly initiate loperamide, 4 mg initially followed by 2 mg with every episode of diarrhea for a maximum of 16 mg daily. Discontinue loperamide 12 hours after diarrhea resolves. Additional supportive measures (e.g., fluid and electrolyte substitution) may also be employed as clinically indicated. Patients who exhibit an excessive cholinergic response to treatment can receive appropriate premedication (e.g., atropine) for subsequent treatments.

Hypersensitivity and Infusion-Related Reactions: Serious hypersensitivity reactions including life-threatening anaphylactic reactions have occurred with Trodelvy. Severe signs and symptoms included cardiac arrest, hypotension, wheezing, angioedema, swelling, pneumonitis, and skin reactions. Hypersensitivity reactions within 24 hours of dosing occurred in 37% of patients. Grade 3-4 hypersensitivity occurred in 2% of patients. The incidence of hypersensitivity reactions leading to permanent discontinuation of Trodelvy was 0.3%. The incidence of anaphylactic reactions was 0.3%. Pre-infusion medication is recommended. Observe patients closely for hypersensitivity and infusion-related reactions during each infusion and for at least 30 minutes after completion of each infusion. Medication to treat such reactions, as well as emergency equipment, should be available for immediate use. Permanently discontinue Trodelvy for Grade 4 infusion-related reactions.

Nausea and Vomiting: Nausea occurred in 66% of all patients treated with Trodelvy and Grade 3 nausea occurred in 4% of these patients. Vomiting occurred in 39% of patients and Grade 3-4 vomiting occurred in 3% of these patients. Premedicate with a two or three drug combination regimen (e.g., dexamethasone with either a 5-HT3 receptor antagonist or an NK1 receptor antagonist as well as other drugs as indicated) for prevention of chemotherapy-induced nausea and vomiting (CINV). Withhold Trodelvy doses for Grade 3 nausea or Grade 3-4 vomiting and resume with additional supportive measures when resolved to Grade ≤1. Additional antiemetics and other supportive measures may also be employed as clinically indicated. All patients should be given take-home medications with clear instructions for prevention and treatment of nausea and vomiting.

Increased Risk of Adverse Reactions in Patients with Reduced UGT1A1 Activity: Patients homozygous for the uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1)*28 allele are at increased risk for neutropenia, febrile neutropenia, and anemia and may be at increased risk for other adverse reactions with Trodelvy. The incidence of Grade 3-4 neutropenia was 67% in patients homozygous for the UGT1A1*28, 46% in patients heterozygous for the UGT1A1*28 allele and 46% in patients homozygous for the wild-type allele. The incidence of Grade 3-4 anemia was 25% in patients homozygous for the UGT1A1*28 allele, 10% in patients heterozygous for the UGT1A1*28 allele, and 11% in patients homozygous for the wild-type allele. Closely monitor patients with known reduced UGT1A1 activity for adverse reactions. Withhold or permanently discontinue Trodelvy based on clinical assessment of the onset, duration and severity of the observed adverse reactions in patients with evidence of acute early-onset or unusually severe adverse reactions, which may indicate reduced UGT1A1 function.

Embryo-Fetal Toxicity: Based on its mechanism of action, Trodelvy can cause teratogenicity and/or embryo-fetal lethality when administered to a pregnant woman. Trodelvy contains a genotoxic component, SN-38, and targets rapidly dividing cells. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with Trodelvy and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with Trodelvy and for 3 months after the last dose.

ADVERSE REACTIONS

In the ASCENT study (IMMU-132-05), the most common adverse reactions (incidence ≥25%) were fatigue, neutropenia, diarrhea, nausea, alopecia, anemia, constipation, vomiting, abdominal pain, and decreased appetite. The most frequent serious adverse reactions (SAR) (>1%) were neutropenia (7%), diarrhea (4%), and pneumonia (3%). SAR were reported in 27% of patients, and 5% discontinued therapy due to adverse reactions. The most common Grade 3-4 lab abnormalities (incidence ≥25%) in the ASCENT study were reduced neutrophils, leukocytes, and lymphocytes.

In the TROPHY study (IMMU-132-06), the most common adverse reactions (incidence ≥25%) were diarrhea, fatigue, neutropenia, nausea, any infection, alopecia, anemia, decreased appetite, constipation, vomiting, abdominal pain, and rash. The most frequent serious adverse reactions (SAR) (≥5%) were infection (18%), neutropenia (12%, including febrile neutropenia in 10%), acute kidney injury (6%), urinary tract infection (6%), and sepsis or bacteremia (5%). SAR were reported in 44% of patients, and 10% discontinued due to adverse reactions. The most common Grade 3-4 lab abnormalities (incidence ≥25%) in the TROPHY study were reduced neutrophils, leukocytes, and lymphocytes.

DRUG INTERACTIONS

UGT1A1 Inhibitors: Concomitant administration of Trodelvy with inhibitors of UGT1A1 may increase the incidence of adverse reactions due to potential increase in systemic exposure to SN-38. Avoid administering UGT1A1 inhibitors with Trodelvy.

UGT1A1 Inducers: Exposure to SN-38 may be substantially reduced in patients concomitantly receiving UGT1A1 enzyme inducers. Avoid administering UGT1A1 inducers with Trodelvy.

Please see full Prescribing Information, including BOXED WARNING.

Allarity Therapeutics Presents Dovitinib Survival Data from DRP® Screened RCC Patients at ESMO 2021 Virtual Congress

On September 16, 2021 Allarity Therapeutics A/S ("Allarity" or the "Company") reported validation results for its Dovitinib DRP companion diagnostic utilizing data from Novartis’ prior Phase III trial of dovitinib in renal cell carcinoma (RCC), which will be included in a poster presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2021 Virtual Congress taking place from September 16 until September 21, 2021 (Press release, Allarity Therapeutics, SEP 16, 2021, View Source [SID1234587830]).

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The poster displays how the RCC patients selected with the Dovitinib DRP companion diagnostic (i.e., those who had a DRP score above 50%) had a median survival of 15.0 months (N=49), compared to a median survival of 11.2 months in the comparator sorafenib arm (N=286, Hazard Ratio: 0.69; 95% Confidence Interval 0.48-0.99) of the clinical trial. When the DRP-Dovitinib score was increased to a score above 67%, the survival in the DRP-selected group increased to a median of 20.6 months (95% Confidence Interval 9.53-35.6, N=15). These results validate that the Dovitinib DRP companion diagnostic can identify RCC patients that benefit from treatment with dovitinib when compared to alternative treatment with sorafenib. The benefit of dovitinib therapy was also evident in progression-free survival data.

Dovitinib, Allarity’s lead clinical-stage asset, is a small molecule, pan-tyrosine kinase inhibitor in-licensed from Novartis. Allarity plans to file a new drug application ("NDA") with the U.S. Food and Drug Administration ("FDA") for the approval of dovitinib for the treatment of RCC during Q4 2021. Allarity has previously filed a pre-market approval (PMA) application for the Dovitinib-DRP. If the FDA provides the anticipated PMA for the Dovitinib-DRP as a companion diagnostic, as well as an NDA approval for dovitinib, Allarity will be able to commercialize dovitinib for DRP-selected RCC patients as an effective new therapy to treat their disease.

Allarity’s CEO Steve Carchedi noted, "The DRP validation data we have been able to publish today further establish the value of our DRP platform in advancing true personalized cancer care, and builds our confidence in a successful road ahead for our planned filing of an NDA for dovitinib. We remain committed to bringing novel oncology therapeutics to market, and to patients, together with their DRP companion diagnostics to improve patient outcomes."

Medigene’s PD1-41BB switch receptor improves TCR-T cell functionality against solid tumors

On September 16, 2021 Medigene AG (Medigene, FSE: MDG1, Prime Standard), a clinical stage immuno-oncology company focusing on the development of T cell immunotherapies, reported that compelling new pre-clinical data on the cellular functionality of its lead T cell receptor-modified T cell therapy (TCR-T) candidate containing the PRAME-specific T cell receptor (TCR) "TCR-4" combined with Medigene’s PD1-41BB switch receptor (Press release, MediGene, SEP 16, 2021, https://www.pressetext.com/news/20210916008 [SID1234587829]).

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In vitro and in vivo, the co-expression of the PD1-41BB switch receptor and TCR-4 on TCR-T cells led to an enhanced response to antigen. In vitro, co-expression enhanced TCR-T proliferation as well as tumor cell killing and increased the release of cytokine messengers, particularly of effector, stimulatory and chemo-attractive cytokines. In an in vivo model, expression of PD1-41BB together with TCR-4 showed the same effects as during the in vitro experiments and promoted tumor clearance substantially. Importantly, co-expression of TCR-4 and the PD1-41BB switch receptor did not impede the favorable preclinical safety profile.

The ePoster 1007P is entitled "T cells transgenic for a highly potent PRAME-specific TCR and a chimeric PD1-41BB co-stimulatory receptor represent a promising approach for the treatment of solid tumors." It will be presented as part of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress being held virtually on 16-21 September 2021. The poster can be found on Medigene’s website: View Source

Prof. Dolores Schendel, Chief Executive Officer and Chief Scientific Officer at Medigene: "The PD1-41BB switch receptor clearly improves TCR-T performance. With PD1-41BB, TCR-4 T cells showed a higher percentage of poly-functional T cells than without. With the PD1-41BB switch receptor co-expressed, a higher proportion of single cells secreted 4-10 cytokines simultaneously. Furthermore, with PD1-41BB co-expression we saw a high number of effector and stimulatory cytokines, and raised levels of chemo-attractive cytokines, which help T cells to migrate towards their target tissue in the body. These data underline our decision to advance the development of the PD1-41BB switch receptor in tandem with TCR-4 in PRAME-positive solid tumors."