A2 Bio Announces First Patient Dosed in EVEREST-2 Phase 1 Clinical Trial of Novel Mesothelin Logic-Gated CAR T

On May 16, 2024 A2 Biotherapeutics, Inc. (A2 Bio), a clinical-stage cell therapy company developing first-in-class logic-gated cell therapies for solid tumors, reported dosing of the first patient in the Phase 1 clinical trial of A2B694 (Press release, A2 Biotherapeutics, MAY 16, 2024, View Source [SID1234643416]). The multi-center Phase 1 dose escalation clinical trial, EVEREST-2 (NCT06051695), will evaluate safety and determine the recommended dose of A2B694 in patients with solid tumors that express mesothelin, including ovarian, mesothelioma, pancreatic, colorectal and non-small cell lung cancer, and have lost HLA-A*02 expression.

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A2B694 is the second autologous cell therapy in clinical development by A2 Bio using its proprietary Tmod platform. The Tmod platform utilizes a dual-receptor design consisting of an activator that targets tumor cells and a blocker that protects normal cells. This dual-receptor design is intended to provide selective killing of tumor tissues that express mesothelin and have lost the HLA-A*02 gene permanently. This novel design is aimed at tackling the fundamental challenge in solid tumor cancer medicines – the ability to selectively kill tumor cells and protect normal cells.

"Dosing our first patient in this trial is a key step to provide a precise, novel CAR T therapy to patients with solid tumors that express mesothelin with no curative treatment options. Sadly, current treatment options for these patients are palliative and limited by toxicity in the recurrent, unresectable, locally advanced or metastatic setting. All of us at A2 Bio would like to thank participating patients, investigators, and clinical care providers," said Dr. William Go, chief medical officer of A2 Bio.

​​In addition to A2B694, A2 Bio continues to advance its clinical development of A2B530 as well as other preclinical programs as the company pursues additional pipeline expansion opportunities using its proprietary Tmod platform.

About EVEREST-2

EVEREST-2 (NCT06051695) is a seamless Phase 1/2 study for A2B694, an autologous logic-gated investigational cell therapy developed from A2 Bio’s proprietary Tmod platform. The Tmod platform provides selective killing of tumor cells and protection of normal cells via a dual-receptor design consisting of an activator that targets tumor cells and a blocker that protects normal cells. A2B694 consists of an activator that targets mesothelin (MSLN) and a blocker that targets HLA-A*02. HLA-A*02 is lost in tumor cells and present in normal cells in the eligible patient population. The study is recruiting patients with lung, colorectal, pancreatic, ovarian and mesothelioma cancers.

About the Tmod Platform

A2 Bio has pioneered a precision-targeting cellular system – the Tmod mechanism – that incorporates two receptors, an activator and a blocker, to aim the powerful armaments of immune cells directly at tumors to unequivocally differentiate tumors from normal tissues. The activator recognizes antigens on tumor cells that trigger their destruction, while the blocker recognizes antigens on normal cells that protect them. This novel blocker technology enables precise, personalized and effective T cell targeting. The blocker component equips Tmod cells with the capacity to identify tumors as distinct from normal cells.

CEL-SCI Corporation Reports Second Quarter Fiscal 2024 Financial Results

On May 16, 2024 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the quarter ended March 31, 2024, as well as key recent clinical and corporate developments (Press release, Cel-Sci, MAY 16, 2024, View Source [SID1234643415]).

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Clinical and Corporate Developments include:

In May 2024, CEL-SCI received the go-ahead from the U.S. Food and Drug Administration (FDA) for its confirmatory Registration Study of Multikine* in the treatment of head and neck cancer based on very strong safety and efficacy data from the completed Phase 3 study which enrolled 928 patients. The survival benefit for the target population treated with Multikine vs standard of care alone was so strong and clear that the confirmatory study only needs to enroll 212 people. The FDA agreed with the pre-surgical selection of patients most likely to benefit from Multikine—those with newly diagnosed advanced primary head and neck cancer with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy).

According to statisticians, the Registration Study is highly likely to succeed because it aims to confirm prior findings and to show a 10% absolute survival benefit vs control at 5 years following treatment. In the Phase 3 study, Multikine demonstrated the following in the target population:
28% absolute survival benefit vs control at 5 years
Risk of death cut in half compared to control at 5 years
73% survival for Multikine vs 45% in the control at 5 years
No safety signals or toxicities vs standard of care
More detailed results may be viewed here: LINK
In February 2024, CEL-SCI’s cGMP state-of-the-art dedicated manufacturing facility commissioning was completed, a significant milestone toward a regulatory approval of Multikine.
In January 2024, the European Medicines Agency’s Paediatric Committee granted CEL-SCI a product-specific waiver of strict requirements for commercialization of cancer drugs in the European Union. The waiver is a significant step forward for Multikine, as it removes a major hurdle on the path towards commercialization in Europe.
CEL-4000, CEL-SCI’s LEAPS vaccine technology, was featured in an article titled "Current status of immunological therapies for rheumatoid arthritis with a focus on antigen-specific therapeutic vaccines" published in the peer reviewed journal Frontiers in Immunology. The article articulates how CEL-4000 may deliver a safe and effective therapy for rheumatoid arthritis by rebalancing inflammatory response, without weakening important immune defense mechanisms, risking infections or cancer, as current treatments may.
"We are eager to commence our FDA Registration Study to confirm the remarkable overall survival benefits Multikine delivered for patients in our target population. The FDA has acknowledged the great unmet need in this patient population. Through the wealth of data produced in our Phase 3 trial, we were able to identify the precise selection criteria for patients and the corresponding diagnostics to identify these patients prior to surgery. This achievement paved the way for us to get FDA agreement to conduct a study on what could become a pre-surgical standard of care in head and neck cancer," stated CEL-SCI CEO, Geert Kersten. "We believe the Registration Study is de-risked, thereby building even more confidence in CEL-SCI and our prospects for Multikine in other solid-tumor cancers."

Financial Results

Research and development expenses decreased by $2.5 million, or 22%, to approximately $9 million during the six months ended March 31, 2024, compared to $11.5 million for the six months ended March 31, 2023. General and administrative expenses in the six months ended March 31, 2024 were $4.6 million compared to $4.4 million in the six months ended March 31, 2023. During the six months ended March 31, 2024, net loss narrowed by $2.2 million or 14% to $14.0 million, approximately $5.3 million of which are non-cash expenses, from $16.4 million in the prior year period. CEL-SCI raised gross proceeds of approximately $7.75 million through a public offering of common stock during the second fiscal quarter.

Bayer presents latest data from oncology portfolio at 2024 ASCO Annual Meeting

On May 16, 2024 Bayer reported that it will present new data across its oncology portfolio at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place in Chicago, from May 31 – June 4, 2024 (Press release, Bayer, MAY 16, 2024, View Source [SID1234643414]). The breadth of new data in different tumor types underscores Bayer’s ambition to deliver innovative and effective oncological therapies in areas with the highest unmet medical needs.

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Prostate cancer is a key focus for Bayer Oncology, with data to be presented on NUBEQA (darolutamide) and XOFIGO (radium Ra 223 dichloride) across multiple stages of the disease. NUBEQA data will include a post hoc analysis from the Phase III ARASENS trial, evaluating post-progression survival with NUBEQA and androgen-deprivation therapy (ADT) plus docetaxel in patients with metastatic hormone-sensitive prostate cancer (mHSPC), which will further support the established efficacy profile of NUBEQA. Additionally a follow-up analysis from the Phase III ARAMIS trial, evaluating the association between prostate-specific antigen (PSA) level and risk of radiological progression with NUBEQA and ADT in patients with non-metastatic castration-resistant prostate cancer (nmCRPC) will also be presented.

An overview of the ongoing investigational Phase III ARASTEP trial in hormone sensitive prostate cancer patients with high-risk biochemical recurrence will also be presented. Additionally, new data from the ongoing Bayer sponsored ALADDIN study, conducted by the Association Pour La Recherche des Thérapeutiques Innovantes en Cancérologie, investigating the impact of NUBEQA plus ADT and radiation therapy on failure-free survival in patients with newly diagnosed prostate cancer and pelvic lymph nodes metastases will be presented.

NUBEQA is currently indicated in the U.S. in combination with docetaxel for the treatment of adult patients with mHSPC and for the treatment of adult patients with non-metastatic castration-resistant prostate cancer (nmCRPC).

In the field of targeted radionuclide therapies, Bayer will present data from the Phase III REASSURE study, evaluating patient profiles and treatment outcomes in patients with metastatic castration resistant prostate cancer (mCRPC) with XOFIGO in the real-world setting, will be shared. XOFIGO is indicated for the treatment of patients with mCRPC, symptomatic bone metastases, and no known visceral metastatic disease.

Bayer will present a late-breaking abstract on BAY 2927088 in patients with HER2-mutant non-small cell lung cancer (NSCLC) from the Phase I/II SOHO-01 trial. BAY 2927088 is an oral, small molecule tyrosine kinase inhibitor being investigated for patients with NSCLC harboring HER2 activating mutations.

Bayer will highlight long-term efficacy and safety data from the VICTORIA study for VITRAKVI, including outcomes in patients with tropomyosin receptor kinase (TRK) fusion cancer treated with VITRAKVI in clinical trials (NCT02122913, NCT02576431, NCT02637687). Vitrakvi is approved for the treatment of adult and pediatric patients with solid tumors that have a NTRK gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. Patients should be selected for therapy based on a FDA-approved test. This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Details on selected abstracts from Bayer at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting are listed below:

Darolutamide

Abstract title: Association between prostate-specific antigen (PSA) level <0.2 ng/mL and risk of radiological progression in patients (pts) with nonmetastatic castration-resistant prostate cancer (nmCRPC): Follow-up analysis of ARAMIS
Poster: 5022; June 2, 2024. 09:00 AM – 12:00 PM CDT
Abstract title: Post-progression survival of patients with metastatic hormone-sensitive prostate cancer (mHSPC) who received darolutamide or placebo: Post hoc analysis of ARASENS
Poster: 5083; June 2, 2024. 09:00 AM – 12:00 PM CDT
Abstract title: Darolutamide plus androgen-deprivation therapy (ADT) in patients with high-risk biochemical recurrence (BCR) of prostate cancer: A phase 3, randomized, double-blind, placebo-controlled study (ARASTEP) Trial in Progress
TiP Poster: TPS5122; June 2, 2024. 09:00 AM – 12:00 PM CDT
Abstract title: ALADDIN: Evaluation of darolutamide in addition to androgen deprivation therapy and radiation therapy in newly diagnosed prostate cancer with pelvic lymph nodes metastases
TiP Poster: TPS5127; June 2, 2024. 9:00AM CDT
Radium-223 dichloride

Abstract title: Comparing patient profiles and treatment outcomes with radium-223 (223Ra) in real-world settings: Academic vs. community practice in the US—Insights from the REASSURE study
ePoster: e17040; Published J Clin Oncol 42, 2024, suppl 16
BAY 2927088

Abstract title: Safety and clinical activity of BAY 2927088 from a Phase I/II trial expansion cohort in patients with HER2-mutant non-small cell lung cancer (NSCLC)
Late Breaking Abstract: LBA8598; June 3, 2024. 1:30PM CDT
Larotrectinib

Abstract title: Long-term efficacy and safety of larotrectinib in patients (pts) with TRK fusion lung cancer
Poster: 8570; June 3, 2024. 1:30PM CDT
Abstract title: Long-term efficacy and safety of larotrectinib (laro) in patients (pts) with TRK fusion thyroid carcinoma (TC)
Poster: 6095; June 2, 2024. 9:00AM CDT
Abstract title: Outcomes of larotrectinib compared with real-world data from non-TRK inhibitor therapies in patients with TRK fusion cancer: VICTORIA study
Poster: 3105; June 1, 2024. 9:00AM CDT
About NUBEQA (darolutamide)1

NUBEQA (darolutamide) is an androgen receptor inhibitor (ARi) with a distinct chemical structure that competitively inhibits androgen binding, AR nuclear translocation, and AR-mediated transcription.

On July 30, 2019, the FDA approved NUBEQA based on the ARAMIS trial, a randomized, double-blind, placebo-controlled, multi-center Phase III study, which evaluated the safety and efficacy of oral NUBEQA in patients with non-metastatic castration-resistant prostate cancer (nmCRPC).

Based on results from the ARASENS trial, a randomized, Phase III, multi-center, double-blind, placebo-controlled trial, NUBEQA plus androgen deprivation therapy (ADT) and docetaxel was approved on August 5, 2022 for the treatment of adult patients with metastatic hormone-sensitive prostate cancer (mHSPC).

NUBEQA is also being investigated in additional studies across various stages of prostate cancer, including in the Phase III ARANOTE trial evaluating NUBEQA plus ADT versus ADT alone for mHSPC, the ARASTEP Phase III trial evaluating NUBEQA plus ADT versus ADT alone in HSPC patients with high-risk BCR and no evidence of metastatic disease, as well as in the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP) led international Phase III co-operative group DASL-HiCaP (ANZUP1801) trial evaluating NUBEQA as an adjuvant treatment for localized prostate cancer with very high risk of recurrence. Information about these trials can be found at www.clinicaltrials.gov.

Developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company, NUBEQA is indicated for the treatment of adults with nmCRPC or with mHSPC in combination with docetaxel.1 Filings in other regions are underway or planned.

INDICATIONS

NUBEQA (darolutamide) is an androgen receptor inhibitor indicated for the treatment of adult patients with:

Non-metastatic castration-resistant prostate cancer (nmCRPC)
Metastatic hormone-sensitive prostate cancer (mHSPC) in combination with docetaxel
IMPORTANT SAFETY INFORMATION

NUBEQA (darolutamide) is an androgen receptor inhibitor indicated for the treatment of adult patients with:

Non-metastatic castration-resistant prostate cancer (nmCRPC)
Metastatic hormone-sensitive prostate cancer (mHSPC) in combination with docetaxel
IMPORTANT SAFETY INFORMATION

Warnings & Precautions

Ischemic Heart Disease – In a study of patients with nmCRPC (ARAMIS), ischemic heart disease occurred in 3.2% of patients receiving NUBEQA versus 2.5% receiving placebo, including Grade 3-4 events in 1.7% vs. 0.4%, respectively. Ischemic events led to death in 0.3% of patients receiving NUBEQA vs. 0.2% receiving placebo. In a study of patients with mHSPC (ARASENS), ischemic heart disease occurred in 3.2% of patients receiving NUBEQA with docetaxel vs. 2% receiving placebo with docetaxel, including Grade 3-4 events in 1.3% vs. 1.1%, respectively. Ischemic events led to death in 0.3% of patients receiving NUBEQA with docetaxel vs. 0% receiving placebo with docetaxel. Monitor for signs and symptoms of ischemic heart disease. Optimize management of cardiovascular risk factors, such as hypertension, diabetes, or dyslipidemia. Discontinue NUBEQA for Grade 3-4 ischemic heart disease.

Seizure – In ARAMIS, Grade 1-2 seizure occurred in 0.2% of patients receiving NUBEQA vs. 0.2% receiving placebo. Seizure occurred 261 and 456 days after initiation of NUBEQA. In ARASENS, seizure occurred in 0.6% of patients receiving NUBEQA with docetaxel, including one Grade 3 event, vs. 0.2% receiving placebo with docetaxel. Seizure occurred 38 to 340 days after initiation of NUBEQA. It is unknown whether antiepileptic medications will prevent seizures with NUBEQA. Advise patients of the risk of developing a seizure while receiving NUBEQA and of engaging in any activity where sudden loss of consciousness could cause harm to themselves or others. Consider discontinuation of NUBEQA in patients who develop a seizure during treatment.

Embryo-Fetal Toxicity – Safety and efficacy of NUBEQA have not been established in females. NUBEQA can cause fetal harm and loss of pregnancy. Advise males with female partners of reproductive potential to use effective contraception during treatment with NUBEQA and for 1 week after the last dose.

Adverse Reactions

In ARAMIS, serious adverse reactions occurred in 25% of patients receiving NUBEQA vs. 20% of patients receiving placebo. Serious adverse reactions in ≥1% of patients who received NUBEQA included urinary retention, pneumonia, and hematuria. Fatal adverse reactions occurred in 3.9% of patients receiving NUBEQA vs. 3.2% of patients receiving placebo. Fatal adverse reactions in patients who received NUBEQA included death (0.4%), cardiac failure (0.3%), cardiac arrest (0.2%), general physical health deterioration (0.2%), and pulmonary embolism (0.2%). The most common adverse reactions (>2% with a ≥2% increase over placebo), including laboratory test abnormalities, were increased AST, decreased neutrophil count, fatigue, increased bilirubin, pain in extremity and rash. Clinically relevant adverse reactions occurring in ≥2% of patients treated with NUBEQA included ischemic heart disease and heart failure.

In ARASENS, serious adverse reactions occurred in 45% of patients receiving NUBEQA with docetaxel vs. 42% of patients receiving placebo with docetaxel. Serious adverse reactions in ≥2% of patients who received NUBEQA with docetaxel included febrile neutropenia (6%), decreased neutrophil count (2.8%), musculoskeletal pain (2.6%), and pneumonia (2.6%). Fatal adverse reactions occurred in 4% of patients receiving NUBEQA with docetaxel vs. 4% of patients receiving placebo with docetaxel. Fatal adverse reactions in patients who received NUBEQA included COVID-19/COVID-19 pneumonia (0.8%), myocardial infarction (0.3%), and sudden death (0.3%). The most common adverse reactions (≥10% with a ≥2% increase over placebo with docetaxel) were constipation, rash, decreased appetite, hemorrhage, increased weight, and hypertension. The most common laboratory test abnormalities (≥30%) were anemia, hyperglycemia, decreased lymphocyte count, decreased neutrophil count, increased AST, increased ALT, and hypocalcemia. Clinically relevant adverse reactions in <10% of patients who received NUBEQA with docetaxel included fractures, ischemic heart disease, seizures, and drug-induced liver injury.

Drug Interactions

Effect of Other Drugs on NUBEQA – Combined P-gp and strong or moderate CYP3A4 inducers decrease NUBEQA exposure, which may decrease NUBEQA activity. Avoid concomitant use.

Combined P-gp and strong CYP3A4 inhibitors increase NUBEQA exposure, which may increase the risk of NUBEQA adverse reactions. Monitor more frequently and modify NUBEQA dose as needed.

Effects of NUBEQA on Other Drugs – NUBEQA inhibits breast cancer resistance protein (BCRP) transporter. Concomitant use increases exposure (AUC) and maximal concentration of BCRP substrates, which may increase the risk of BCRP substrate-related toxicities. Avoid concomitant use where possible. If used together, monitor more frequently for adverse reactions, and consider dose reduction of the BCRP substrate.

NUBEQA inhibits OATP1B1 and OATP1B3 transporters. Concomitant use may increase plasma concentrations of OATP1B1 or OATP1B3 substrates. Monitor more frequently for adverse reactions and consider dose reduction of these substrates.

Review the Prescribing Information of drugs that are BCRP, OATP1B1, and OATP1B3 substrates when used concomitantly with NUBEQA.

For important risk and use information about NUBEQA, please see the accompanying full Prescribing Information.

About XOFIGO (radium Ra 223 dichloride) Injection2

Xofigo is indicated for the treatment of patients with castration-resistant prostate cancer, symptomatic bone metastases and no known visceral metastatic disease.

Important Safety Information for Xofigo (radium Ra 223 dichloride) Injection

Warnings and Precautions:

Bone Marrow Suppression: In the phase 3 ALSYMPCA trial, 2% of patients in the Xofigo arm experienced bone marrow failure or ongoing pancytopenia, compared to no patients treated with placebo. There were two deaths due to bone marrow failure. For 7 of 13 patients treated with Xofigo bone marrow failure was ongoing at the time of death. Among the 13 patients who experienced bone marrow failure, 54% required blood transfusions. Four percent (4%) of patients in the Xofigo arm and 2% in the placebo arm permanently discontinued therapy due to bone marrow suppression. In the randomized trial, deaths related to vascular hemorrhage in association with myelosuppression were observed in 1% of Xofigo-treated patients compared to 0.3% of patients treated with placebo. The incidence of infection-related deaths (2%), serious infections (10%), and febrile neutropenia (<1%) was similar for patients treated with Xofigo and placebo. Myelosuppression–notably thrombocytopenia, neutropenia, pancytopenia, and leukopenia–has been reported in patients treated with Xofigo.
Monitor patients with evidence of compromised bone marrow reserve closely and provide supportive care measures when clinically indicated. Discontinue Xofigo in patients who experience life-threatening complications despite supportive care for bone marrow failure

Hematological Evaluation: Monitor blood counts at baseline and prior to every dose of Xofigo. Prior to first administering Xofigo, the absolute neutrophil count (ANC) should be ≥1.5 × 109/L, the platelet count ≥100 × 109/L, and hemoglobin ≥10 g/dL. Prior to subsequent administrations, the ANC should be ≥1 × 109/L and the platelet count ≥50 × 109/L. Discontinue Xofigo if hematologic values do not recover within 6 to 8 weeks after the last administration despite receiving supportive care
Concomitant Use With Chemotherapy: Safety and efficacy of concomitant chemotherapy with Xofigo have not been established. Outside of a clinical trial, concomitant use of Xofigo in patients on chemotherapy is not recommended due to the potential for additive myelosuppression. If chemotherapy, other systemic radioisotopes, or hemibody external radiotherapy are administered during the treatment period, Xofigo should be discontinued
Increased Fractures and Mortality in Combination With Abiraterone Plus Prednisone/Prednisolone: Xofigo is not recommended for use in combination with abiraterone acetate plus prednisone/prednisolone outside of clinical trials. At the primary analysis of the Phase 3 ERA-223 study that evaluated concurrent initiation of Xofigo in combination with abiraterone acetate plus prednisone/prednisolone in 806 asymptomatic or mildly symptomatic mCRPC patients, an increased incidence of fractures (28.6% vs 11.4%) and deaths (38.5% vs 35.5%) have been observed in patients who received Xofigo in combination with abiraterone acetate plus prednisone/prednisolone compared to patients who received placebo in combination with abiraterone acetate plus prednisone/prednisolone. Safety and efficacy with the combination of Xofigo and agents other than gonadotropin-releasing hormone analogues have not been established
Embryo-Fetal Toxicity: The safety and efficacy of Xofigo have not been established in females. Xofigo can cause fetal harm when administered to a pregnant female. Advise pregnant females and females of reproductive potential of the potential risk to a fetus. Advise male patients to use condoms and their female partners of reproductive potential to use effective contraception during and for 6 months after completing treatment with Xofigo
Administration and Radiation Protection: Xofigo should be received, used, and administered only by authorized persons in designated clinical settings. The administration of Xofigo is associated with potential risks to other persons from radiation or contamination from spills of bodily fluids such as urine, feces, or vomit. Therefore, radiation protection precautions must be taken in accordance with national and local regulations

Fluid Status: Dehydration occurred in 3% of patients on Xofigo and 1% of patients on placebo. Xofigo increases adverse reactions such as diarrhea, nausea, and vomiting, which may result in dehydration. Monitor patients’ oral intake and fluid status carefully and promptly treat patients who display signs or symptoms of dehydration or hypovolemia

Injection Site Reactions: Erythema, pain, and edema at the injection site were reported in 1% of patients on Xofigo

Secondary Malignant Neoplasms: Xofigo contributes to a patient’s overall long-term cumulative radiation exposure. Long-term cumulative radiation exposure may be associated with an increased risk of cancer and hereditary defects. Due to its mechanism of action and neoplastic changes, including osteosarcomas, in rats following administration of radium-223 dichloride, Xofigo may increase the risk of osteosarcoma or other secondary malignant neoplasms. However, the overall incidence of new malignancies in the randomized trial was lower on the Xofigo arm compared to placebo (<1% vs 2%; respectively), but the expected latency period for the development of secondary malignancies exceeds the duration of follow-up for patients on the trial

Subsequent Treatment With Cytotoxic Chemotherapy: In the randomized clinical trial, 16% of patients in the Xofigo group and 18% of patients in the placebo group received cytotoxic chemotherapy after completion of study treatments. Adequate safety monitoring and laboratory testing was not performed to assess how patients treated with Xofigo will tolerate subsequent cytotoxic chemotherapy

Adverse Reactions: The most common adverse reactions (≥10%) in the Xofigo arm vs the placebo arm, respectively, were nausea (36% vs 35%), diarrhea (25% vs 15%), vomiting (19% vs 14%), and peripheral edema (13% vs 10%). Grade 3 and 4 adverse events were reported in 57% of Xofigo-treated patients and 63% of placebo-treated patients. The most common hematologic laboratory abnormalities in the Xofigo arm (≥10%) vs the placebo arm, respectively, were anemia (93% vs 88%), lymphocytopenia (72% vs 53%), leukopenia (35% vs 10%), thrombocytopenia (31% vs 22%), and neutropenia (18% vs 5%)

Please see the full Prescribing Information for Xofigo (radium Ra 223 dichloride).

About VITRAKVI (larotrectinib)3

VITRAKVI is indicated for the treatment of adult and pediatric patients with solid tumors that:

have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation,
are metastatic or where surgical resection is likely to result in severe morbidity, and
have no satisfactory alternative treatments or that have progressed following treatment.
Select patients for therapy based on an FDA-approved test.

This indication is 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 benefit in confirmatory trials.

IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Central Nervous System Effects: Central nervous system (CNS) adverse reactions occurred in patients receiving VITRAKVI, including dizziness, cognitive impairment, mood disorders, and sleep disturbances.

In patients who received VITRAKVI, all grades CNS effects including cognitive impairment, mood disorders, dizziness and sleep disorders were observed in 42% with Grades 3-4 in 3.9% of patients.

Cognitive impairment occurred in 11% of patients. The median time to onset of cognitive impairment was 5.6 months (range: 2 days to 41 months). Cognitive impairment occurring in ≥ 1% of patients included memory impairment (3.6%), confusional state (2.9%), disturbance in attention (2.9%), delirium (2.2%), cognitive disorders (1.4%), and Grade 3 cognitive adverse reactions occurred in 2.5% of patients. Among the 30 patients with cognitive impairment, 7% required a dose modification and 20% required dose interruption.

Mood disorders occurred in 14% of patients. The median time to onset of mood disorders was 3.9 months (range: 1 day to 40.5 months). Mood disorders occurring in ≥1% of patients included anxiety (5%), depression (3.9%), agitation (2.9%), and irritability (2.9%). Grade 3 mood disorders occurred in 0.4% of patients.

Dizziness occurred in 27% of patients, and Grade 3 dizziness occurred in 1.1% of patients. Among the 74 patients who experienced dizziness, 5% of patients required a dose modification and 5% required dose interruption.

Sleep disturbances occurred in 10% of patients. Sleep disturbances included insomnia (7%), somnolence (2.5%), and sleep disorder (0.4%). There were no Grade 3-4 sleep disturbances. Among the 28 patients who experienced sleep disturbances, 1 patient each (3.6%) required a dose modification or dose interruption.

Advise patients and caretakers of these risks with VITRAKVI. Advise patients not to drive or operate hazardous machinery if they are experiencing neurologic adverse reactions. Withhold or permanently discontinue VITRAKVI based on the severity. If withheld, modify the VITRAKVI dosage when resumed.
Skeletal Fractures: Among 187 adult patients who received VITRAKVI across clinical trials, fractures were reported in 7% and among 92 pediatric patients, fractures were reported in 9% (N=279; 8%). Median time to fracture was 11.6 months (range 0.9 to 45.8 months) in patients followed per fracture. Fractures of the femur, hip or acetabulum were reported in 4 patients (3 adult, 1 pediatric). Most fractures were associated with minimal or moderate trauma. Some fractures were associated with radiologic abnormalities suggestive of local tumor involvement. VITRAKVI treatment was interrupted due to fracture in 1.4% patients.

Promptly evaluate patients with signs or symptoms of potential fracture (e.g., pain, changes in mobility, deformity). There are no data on the effects of VITRAKVI on healing of known fractures or risk of future fractures.
Hepatotoxicity: Hepatotoxicity including drug induced liver injury (DILI) has been reported in patients taking VITRAKVI.

In patients who received VITRAKVI, increased AST of any grade occurred in 52% of patients and increased ALT of any grade occurred in 45%. Grade 3-4 increased AST or ALT occurred in 3.1% and 2.5% of patients, respectively. The median time to onset of increased AST was 2.1 months (range: 1 day to 4.3 years). The median time to onset of increased ALT was 2.3 months (range: 1 day to 4.2 years). Increased AST and ALT leading to dose modifications occurred in 1.4% and 2.2% of patients, respectively. Increased AST or ALT led to permanent discontinuation in 3 (1.1%) of patients.

There have been reports in adult patients from clinical studies and post-marketing cases of Grade ≥ 2 increases in ALT and/or AST with increases in bilirubin ≥ 2 x ULN.

Obtain liver function tests (ALT, AST, ALP and bilirubin) before initiation of VITRAKVI and monitor every 2 weeks during the first two months of treatment, then monthly thereafter, or more frequently following the occurrence of Grade 2 or greater AST or ALT elevation. Temporarily withhold, reduce the dose, or permanently discontinue VITRAKVI based on severity.
Embryo-Fetal Toxicity: VITRAKVI can cause fetal harm when administered to a pregnant woman. Larotrectinib resulted in malformations in rats and rabbits at maternal exposures that were approximately 11- and 0.7-times, respectively, those observed at the clinical dose of 100 mg twice daily. Advise women of the potential risk to a fetus. Advise females of reproductive potential to use an effective method of contraception during treatment and for 1 week after the last dose of VITRAKVI.
Adverse Reactions

The most common adverse reactions (≥20%), including laboratory abnormalities, were: increased AST (52%), increased ALT (45%), anemia (42%), musculoskeletal pain (42%), fatigue (36%), hypoalbuminemia (36%), neutropenia (36%), increased alkaline phosphatase (34%), cough (32%), leukopenia (28%), constipation (27%), diarrhea (27%), dizziness (27%), hypocalcemia (25%), nausea (25%), vomiting (25%), pyrexia (24%), lymphopenia (22%) and abdominal pain (21%).
Drug Interactions

Avoid coadministration of VITRAKVI with strong CYP3A4 inhibitors (including grapefruit or grapefruit juice), strong CYP3A4 inducers (including St. John’s wort), or sensitive CYP3A4 substrates. If coadministration of strong CYP3A4 inhibitors or inducers cannot be avoided, modify the VITRAKVI dose as recommended. If coadministration of sensitive CYP3A4 substrates cannot be avoided, monitor patients for increased adverse reactions of these drugs. For coadministration with moderate CYP3A4 inhibitors, monitor for adverse reactions more frequently and reduce the dosage based on severity. For coadministration with moderate CYP3A4 inducers, modify dose as recommended.
Use in Specific Populations

Lactation: Advise women not to breastfeed during treatment with VITRAKVI and for 1 week after the last dose.
Please see the full Prescribing Information for VITRAKVI (larotrectinib).

Accelerated Biosciences and Spanios Partner to Advance Therapeutic Design Across Solid Tumor Cancers

On May 16, 2024 Spanios, a firm that created and leverages its unique 3D tumoroid platforms to validate solid tumor therapies reported a partnership with Accelerated Biosciences, a company that licenses its commercial-grade human trophoblast stem cell (hTSC) platform for drug discovery, therapeutic development, biomanufacturing, and toxicology testing (Press release, Accelerated Biosciences, MAY 16, 2024, View Source [SID1234643413]).

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The partnership combines Spanios’ proprietary COMPASS platforms and patient-derived tumoroid models with AccelBio’s hTSC platform to advance therapeutic design across solid tumor cancers.

Accelerated Bio’s hTSC platform serves as the starting material for this collaboration. hTSCs are the earliest form of pluripotent stem cells that are ethically sourced. These cells are highly plastic and immune-privileged, making them ideal for numerous medical and biotechnological applications. Among many other functions that the hTSCs can perform, effector cells derived from the hTSCs have been shown to be successful against many cancer cells.

Spanios’ tumoroid research expertise enhanced by its ability to rapidly recapitulate preclinical therapeutic performance ex vivo will play a key role in generating clinically relevant data for the hTSCs. "We are excited about the preliminary data supporting the NK-like behaviors of these cells," said Vivek Ravi, Spanios CEO. "This opens a huge opportunity for cell-based therapies where other forms have struggled, especially for Ovarian, Colorectal and other challenging cancers, and is the driving force for the partnership."

"We are excited to collaborate with Spanios to advance the research and development of effector cells derived from hTSCs," said Yuta Lee, CEO of Accelerated Bio. "Together, we believe that we can further demonstrate the versatility of the hTSC platform for the development of the next-generation precision medicine therapies," said Ravi.

Compugen Publishes Paper in Cancer Immunology Research on Unique Biology of PVRIG and its Therapeutic Potential

On May 16, 2024 Compugen Ltd. (Nasdaq: CGEN) (TASE: CGEN) a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, reported the online publication of a peer reviewed paper titled "PVRIG is expressed on stem-like T cells in dendritic cell-rich niches in tumors and its blockade may induce immune infiltration in non-inflamed tumors" link, in Cancer Immunology Research, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (Press release, Compugen, MAY 16, 2024, View Source [SID1234643412]).

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"Increasingly, we are observing that an additional component is needed to optimize the anti-tumor response to a combination of an anti-TIGIT and anti-PD-(L)1," said Anat Cohen-Dayag, Ph.D., President, and Chief Executive Officer of Compugen. "Through our computational discovery work at Compugen we were the first to discover the novel immune checkpoint PVRIG and later presented preclinical data on the potential synergy in blocking PVRIG, TIGIT and PD-1 pathways. Subsequently, we showed in Phase 1 studies, that the triple blockade of PVRIG with our potential first-in-class antibody, COM701 in combination with an anti-TIGIT and anti-PD-1 demonstrated durable clinical responses with a favorable safety profile in indications typically not responding to immunotherapy. These responses were associated with the infiltration and proliferation of cancer-fighting T cells in the tumor microenvironment, atypical for less inflamed indications."

Eran Ophir, Ph.D., Chief Scientific Officer at Compugen added, "Our paper published online today in Cancer Immunology Research provides an advanced understanding of the unique biology of PVRIG and its role in cancer. Using single-cell RNA sequencing and spatial transcriptomic analysis, we showed the differentiated expression of PVRIG and its ligand PVRL2 on early differentiated stem like memory T cells and dendritic cells, respectively, the interaction of which plays an important role in inhibiting the recruitment and proliferation of cancer fighting T-cells in the tumor microenvironment. This unique biology of PVRIG may support a novel approach of potentially overcoming resistance to immunotherapy by sensitizing tumors to respond to other immune checkpoint inhibitors."