On October 13, 2021 Susan G. Komen, the world’s leading breast cancer organization, reported the awarding of $1.5 million for three new research projects that examine three unique areas focused on metastatic breast cancer (MBC) (Press release, Susan G Komen, OCT 13, 2021, View Source [SID1234591189]). The grants are part of the Susan G. Komen Metastatic Breast Cancer Collaborative Research Initiative, an innovative, first-of-its-kind collaboration between Komen, Duke Cancer Institute and the University of North Carolina Lineberger Comprehensive Cancer Center. The initiative is an effort to pair researchers from each of the organizations to work together to address significant gaps in our knowledge about MBC to advance patient care and improve patient outcomes.

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"We know that finding the cures for breast cancer will only be accomplished by working together through innovative research," said Paula Schneider, Komen’s president and CEO. "It’s this strong belief in the power of collaboration to advance discovery that led to this novel partnership between some of the leading researchers at two institutions that are known for their rivalry."

These three grants fall under Komen’s two primary research priorities, which are to find new ways to prevent, detect, and treat metastatic and aggressive breast cancers, as well as to understand and overcome the inequities that lead certain people and communities to have higher rates of mortality from breast cancer.

"This bold investment by Komen and its supporters is aimed at changing our fundamental understanding of metastatic breast cancer," said UNC Lineberger Director Shelley Earp, M.D. "Combining the creative minds of two of the country’s premier cancer centers is the way to probe the longstanding problem of breast cancer spread. Each of these approaches will yield groundbreaking knowledge to develop better, more effective treatments by harnessing a patient’s own immune system and will identify the biological and societal drivers that contribute to disparities in breast cancer outcomes among Black and white women."

"We are very grateful to Susan G. Komen for their long-term dedication to improving approaches to the prevention, diagnosis and treatment of breast cancer," said Michael B. Kastan, M.D., Ph.D., executive director of the Duke Cancer Institute. "Metastatic breast cancer remains a major challenge and this visionary funding mechanism brings together outstanding investigators and physicians from two neighboring institutions in collaborative projects that will make a difference for women in the state of North Carolina and around the country."

Thanks to a $500,000 lead gift by Blue Cross and Blue Shield of North Carolina (Blue Cross NC), we are able to award the Susan G. Komen and Blue Cross NC Metastatic Breast Cancer Disparities Collaborative Research Grant to:

A research team led by Drs. Jennifer Freedman and Steven Paterno of Duke Cancer Institute, and Dr. Katie Hoadley of the University of North Carolina Lineberger Comprehensive Cancer Center, who were awarded a grant to investigate how the ancestry of different populations impacts the immune response to metastatic breast cancer. The study leaders identified biological differences in certain genetic events (called RNA splicing) in tumors between those with African versus European ancestry. The team seeks to determine if these differences cause breast cancer cells to grow and spread more quickly in patients of African descent and contribute to higher metastasis and death rates among Black women. Improved understanding of these underlying molecular mechanisms may lead the way to better treatments and outcomes.
"We know that the health of our state depends on the health of all our communities – and to improve health equity, we must take a closer look at the factors that drive the disparities between Black women and metastatic breast cancer," said Dr. Kia Williams, associate medical director at Blue Cross NC. "We are excited to support Komen and researchers at UNC and Duke and eagerly await the invaluable scientific contributions to come from this collaborative effort."

Thanks to a $500,000 gift by Gilead Sciences, Inc., we are able to award the Susan G. Komen and Gilead Sciences, Inc. for North Carolina’s Metastatic Breast Cancer Collaborative Research Grant to:

A research team led by Dr. Melissa Troester of the University of North Carolina Lineberger Comprehensive Cancer Center and Dr. Terry Hyslop of Duke Cancer Institute for their project that will seek to use information on tumor biology and social factors in UNC’s long-standing Carolina Breast Cancer Study (CBCS) to understand racial differences in breast cancer metastasis and death. Researchers will also evaluate how life stress contributes to higher metastasis rates and worse breast cancer outcomes in Black women when compared to white women. They aim to develop specific interventions to reduce metastasis that consider multiple factors from basic biology to societal factors in order to improve outcomes for Black women.
"As metastatic breast cancer disproportionately impacts Black women, this innovative research to better understand evidence-based solutions to improve outcomes for Black women is crucial," said Bill Grossman, M.D., Ph.D., Senior Vice President, Oncology Therapeutics, Gilead Sciences. "Gilead is proud to support Susan G. Komen in these much-needed efforts."

Thanks to funds raised by individuals and organizations in North Carolina and across the country, Komen is able to award:

A research team led by Dr. Benjamin Vincent of the University of North Carolina Comprehensive Cancer Center and Dr. Zachary Hartman of Duke Cancer Institute with a $500,000 grant for their project to develop a personalized anti-tumor vaccine strategy for patients with advanced Triple Negative Breast Cancer that would mobilize the body’s immune system (T cells) to shut down tumor growth and metastasis.
Spearheaded by Komen Development Director Pam Kohl, who is living with MBC, the Komen Collaborative Metastatic Research Initiative seeks to raise funds with the hope of accelerating discovery by connecting leading researchers from these two leading institutions. These three grants are the initial awards from this initiative. Those interested in supporting additional collaborations can donate at www.komen.org/mbccollaborative.

"Currently, there is no cure for MBC and the treatments are difficult at best," said Kohl. "Research is HOPE for the far too many of us who are living with MBC. This disease is smart, and it is relentless, but I know that these brilliant researchers at UNC and Duke will work every day to help give us the gift of time."

On October 13, 2021 NeoImmuneTech, Inc. (KOSDAQ: 950220), a clinical-stage T cell-focused biopharmaceutical company, reported that the first patient has been dosed in a Phase 1b study of its lead drug candidate, NT-I7 (efineptakin alfa), a novel long-acting human interleukin-7 (IL-7), following CAR-T cell therapy tisagenlecleucel (Kymriah) in patients with relapsed/refractory (r/r) Large B-Cell Lymphoma (LBCL) (Press release, NeoImmuneTech, OCT 13, 2021, View Source [SID1234591188]).

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Diffuse Large B-Cell Lymphoma (DLBCL) is the most commonly occurring subtype of Non-Hodgkin Lymphoma (NHL), accounting for 25% to 30% of all NHL and >20,000 cases in the U.S. annually1. Despite improvement in therapeutic options, treatment is often not curative with as many as 50% of patients developing relapsed or refractory disease2.

"We have shown in multiple animal models that the addition of NT-I7 to CAR-T cells substantially increased CAR-T cell proliferation, persistence, and target-specific tumor killing, resulting in significantly prolonged survival of the treated animals," said NgocDiep Le, M.D., Ph.D., Executive VP and Chief Medical Officer of NeoImmuneTech (NIT). "Now that we have begun dosing in this study, we look forward to evaluating the potential of NT-I7 therapy to prolong clinical response and survival for patients with r/r LBCL."

Se Hwan Yang, Ph.D., President and Chief Executive Officer of NIT added, "While CAR-T cell therapies have revolutionized the way we treat multiple hematologic malignancies, relapsed or refractory illness still impacts many patients with LBCL who are in need of additional treatment options. By advancing this Phase 1b study, we hope to pave the way for a new therapeutic solution that could enhance the clinical impact of Kymriah alone and ultimately improve patient outcomes."

This multicenter Phase 1b study will evaluate the safety, tolerability, and preliminary anti-tumor activity of NT-I7 administration following standard-of-care tisagenlecleucel CAR-T cell therapy, and determine the recommended Phase 2 dose of the combination for further future clinical development.

More information on this trial can be found at www.clinicaltrials.gov, identifier: 05075603.

Kymriah is a registered trademark of Novartis AG.

About NT-I7

NT-I7 (efineptakin alfa) is the only clinical-stage long-acting human IL-7, and is being developed for oncologic and immunologic indications, in which T cell amplification and enhanced functionality may provide clinical benefit. IL-7 is a fundamental cytokine for naïve and memory T cell development and for sustaining immune response to chronic antigens (as in cancer) or foreign antigens (as in infectious diseases). In clinical trials to date, NT-I7 has exhibited favorable PK/PD and safety profiles, both as a monotherapy and in combination with other anticancer treatments. NT-I7 is being studied in multiple clinical trials in solid tumors and as a vaccine adjuvant. Studies are being planned for testing in hematologic malignancies, additional solid tumors and other immunology-focused indications.

On October 13, 2021 Veracyte, Inc., (Nasdaq: VCYT) reported the publication of new data demonstrating that the company’s Decipher Bladder genomic classifier accurately identifies bladder tumors that are most likely to respond to chemotherapy prior to radical cystectomy (Press release, Veracyte, OCT 13, 2021, View Source [SID1234591182]). These findings could ultimately help physicians optimize treatment planning for their patients with bladder cancer based on their tumor subtype biology. The peer-reviewed paper appears online today in The Journal of Urology.

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Patients diagnosed with non-metastatic muscle-invasive bladder cancer (MIBC) often undergo neoadjuvant chemotherapy (NAC) prior to standard-of-care radical cystectomy, even though the absolute survival benefit associated with the addition of NAC to radical cystectomy is 5-10%. Until recently, there was no reliable way to determine which MIBC tumors would – or would not – respond to chemotherapy. Molecular subtyping with the Decipher Bladder genomic classifier has shown that biological differences in MIBC are strongly associated with chemotherapy response.

"Our findings are among the first to show the clinical utility of implementing molecular subtyping in order to identify patients for whom NAC is most likely to confer significant benefit," said Yair Lotan, MD, professor of urology and chief of urologic oncology at UT Southwestern Medical Center, and the paper’s lead author. "This study represents meaningful progress in clinically validating biomarkers that are associated with chemotherapy response in patients with MIBC, and it offers hope that we will be able to better manage these patients in the future by accurately selecting those most likely to benefit from additional treatment."

In this multicenter retrospective study, scientists evaluated the use of the Decipher Bladder genomic classifier in a cohort of 601 patients. After three years, patients with classifier-identified luminal tumors (37% of the study population) experienced no additional benefit to overall survival (OS) from receiving NAC prior to radical cystectomy (63% OS with NAC and 65% OS without). However, those patients whose tumors were classified as non-luminal (63% of the study population) experienced a significant benefit from the addition of NAC, with 10% greater overall survival after three years as compared to patients who were treated with cystectomy alone (71% vs. 61%).

"We are pleased that our Decipher Bladder genomic classifier performed so well in this broad study," said Elai Davicioni, Ph.D., Veracyte’s senior vice president of scientific and clinical operations, Urologic Cancers. "We look forward to further validating the test to encourage its routine clinical use for patients diagnosed with MIBC."

The Decipher Bladder test is supported by multiple peer-reviewed clinical studies demonstrating its ability to identify which patients have a higher risk of upstaging to non-organ confined disease at surgery and which patients may benefit the most from neoadjuvant therapy. The test also can be used to identify neuroendocrine-like and immune-infiltrated subtypes, which may have implications for future therapeutic strategies. In June 2021, Veracyte announced that Decipher Bladder had become the first molecular subtyping test to gain Medicare coverage to inform treatment for individuals with bladder cancer.

About Decipher Bladder

Decipher Bladder is a genomic test that measures the molecular profile of bladder cancer using gene expression analysis from transurethral resected bladder tumor specimens. It was developed for bladder cancer patients with high-grade non-muscle-invasive disease who are being considered for treatment and patients with muscle-invasive disease who face the question of immediate cystectomy or systemic treatment in the neoadjuvant setting prior to cystectomy (NAC). Decipher Bladder reports the molecular subtype of the tumor specimen as Luminal or Non-Luminal (Luminal Infiltrated, Basal, Basal Claudin-Low or Neuroendocrine-like), with each subtype having distinct biological composition, clinical behavior and predicted benefit from NAC.

On October 13, 2021 LAVA Therapeutics Presented the Corporate Presentation (Presentation, Lava Therapeutics, OCT 13, 2021, View Source [SID1234591181]).

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On October 13, 2021 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, reported positive results from a pharmacoeconomic study evaluating the cost-effectiveness of using investigational drug Motixafortide as a primary stem cell mobilization (SCM) agent on top of granulocyte colony stimulating factor (G-CSF), versus G-CSF alone, in multiple myeloma patients undergoing autologous stem cell transplantation (ASCT) (Press release, BioLineRx, OCT 13, 2021, View Source [SID1234591180]). The study was performed by the Global Health Economics and Outcomes Research (HEOR) team of IQVIA, and was a pre-planned study conducted in parallel with the GENESIS Phase 3 trial. These results, together with the highly significant and clinically meaningful data from the GENESIS trial, strongly support the potential use of Motixafortide, on top of G-CSF, as the standard of care in SCM for ASCT.

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The study concluded that the addition of Motixafortide to G-CSF (the current standard of care) is associated with a statistically significant decrease in health resource utilization (HRU) during the ASCT process, compared to G-CSF alone. Based on the significantly higher number of mobilized cells and the lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus G-CSF alone. The study findings, combined with model estimates, suggest that the use of Motixafortide, on top of G-CSF, as the standard of care in mobilization for ASCT, could be a cost-effective option in the US, based on accepted willingness-to-pay (WTP) values for healthcare payers.

"The compelling cost savings identified by this rigorously designed study strongly support the Company’s view that Motixafortide, in combination with G-CSF, can become the new standard of care as an upfront, or primary, therapy for all multiple myeloma patients undergoing autologous stem cell transplantation," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Based on data from the GENESIS trial showing that nearly 90% of patients collected an optimal number of cells for transplantation following a single administration of Motixafortide and in only one apheresis session, versus less than 10% for G-CSF alone, the pharmacoeconomic study demonstrates that use of Motixafortide on top of G-CSF can save $17,000 per patient, not including the cost of Motixafortide. These cost savings should leave substantial room in the future to optimize our pricing strategy for Motixafortide at product launch and thereafter, if approved.

"It is also important to note that fewer administrations and apheresis sessions confer meaningful safety and time benefits to patients. In addition, the significantly higher median number of cells collected in one apheresis session – ~11 million using Motixafortide on top of G-CSF versus ~2 million for G-CSF alone – not only enables transplantation of an optimal number of cells, with the potential to significantly save on time to engraftment, it also permits the retention of enough cells for cryopreservation in the event that an additional transplantation is required in the future. Lastly, higher levels of certainty regarding the number of apheresis sessions required for mobilization could enable more efficient utilization of apheresis units at transplantation institutions, where there is often a shortage of available machines.

"We believe the data from the GENESIS study, together with the results from this pharmacoeconomic study, set Motixafortide apart from all other mobilization agents either currently available or in development. If approved, Motixafortide represents a significant advancement in SCM to the benefit of patients and payers alike, and, to that end, we remain on track to submit a New Drug Application (NDA) to the FDA in the first half of next year," Mr. Serlin concluded.

About the Pharmacoeconomic Study

The pharmacoeconomic study analyzed healthcare resource utilization (HRU) observed during the Phase 3 GENESIS trial, which randomized 122 patients into two arms: Motixafortide plus G-CSF (n=80) or placebo plus G-CSF (n=42). HRU data points collected include: (1) the number of Motixafortide and G-CSF doses, as well as the number of apheresis sessions performed, in primary mobilization; (2) the percentage of patients needing rescue mobilization due to poor primary mobilization, including the number of apheresis sessions needed and the number of G-CSF and plerixafor doses required; and (3) hospitalization costs related to conditioning and transplantation, including length of stay. Quality-adjusted life years gained (QALY) from published literature were also incorporated into the model. Motixafortide plus G-CSF was associated with a statistically significant HRU decrease during the autologous stem cell transplantation process compared to standard-of-care G-CSF alone. Given the higher number of mobilized cells and lower number of apheresis sessions, lifetime estimates show quality-adjusted-life-year (QALY) benefits and net cost savings of ~$17,000 (not including the cost of Motixafortide), versus the current standard of care.

About the GENESIS Phase 3 Trial

The GENESIS Phase 3 trial (NCT03246529) was initiated in December 2017. GENESIS was a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of Motixafortide and G-CSF, compared to placebo and G-CSF, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. The primary objective of the study was to demonstrate that only one dose of Motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize ≥ 6 million CD34+ cells in up to two apheresis sessions. Additional objectives included time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters. The study successfully met all primary and secondary endpoints with an exceptionally high level of statistical significance (p<0.0001), including approximately 90% of patients who mobilized the target number of cells for transplantation with only one administration of Motixafortide and in only one apheresis session.

About Stem Cell Mobilization for Autologous Stem Cell Transplantation

Autologous stem cell transplantation (ASCT) is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma, non-Hodgkin’s lymphoma and other lymphomas. In eligible patients, ASCT is performed after initial (induction) therapy, and, in most cases, requires consecutive-day clinic visits for the mobilization and apheresis (harvesting) phases, and full hospitalization for the conditioning chemotherapy and transplantation phases until engraftment. The associated burden is therefore significant – patients experience clinically relevant deteriorations in their quality of life during ASCT, and healthcare resource use throughout the ASCT phases is particularly intense. Therefore, new interventions impacting the ASCT process have the potential for relieving some of the clinical burden for transplanted patients, the logistical burden for the apheresis units, and the financial burden for healthcare providers and payers.

Described simply, ASCT consists of: (1) mobilizing the patient’s own stem cells from his/ her bone marrow to the peripheral blood for removing (harvesting) via an apheresis procedure; (2) freezing and storing the harvested cells until they are needed for transplantation; (3) providing a conditioning treatment, such as high-dose chemotherapy or radiation, to kill the remaining cancer cells the day before transplant; and (4) infusing the stored stem cells back to the patient intravenously via a catheter.

To mobilize the patient’s stem cells from the bone marrow to the peripheral blood for harvesting, the current standard of care includes the administration of 5-8 daily doses of granulocyte colony stimulating factor (G-CSF), and the performance of 1-4 apheresis sessions. For patients unable to mobilize sufficient numbers of cells for harvesting during this primary mobilization phase, rescue therapy is carried out, consisting of 1-4 doses of plerixafor on top of G-CSF, and the performance of an additional number of apheresis sessions as necessary. In light of this, an agent with superior mobilization activity may significantly reduce the mobilization and harvesting burden and associated risks of the ASCT process and lead to significant clinical and resource benefits.