On December 1, 2020 Dana-Farber Cancer Institute researchers reported that it will present more than 40 research studies at the virtual 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 5-8, including two studies that were selected for inclusion in the official press program (Press release, Dana-Farber Cancer Institute, DEC 1, 2020, View Source [SID1234572069]). Dana-Farber is home to one of the largest and most respected treatment centers for patients with disorders of the blood and bone marrow. ASH (Free ASH Whitepaper) is the world’s most comprehensive hematology event, attracting more than 25,000 hematology professionals from around the world.

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Scientific updates to be presented at ASH (Free ASH Whitepaper) reveal potentially practice changing findings related to stem cell transplant, lymphoma, leukemia, and multiple myeloma treatment. Notable oral presentations by Dana-Farber researchers include:

Title: A multi-center biologic assignment trial comparing reduced intensity allogeneic hematopoietic cell transplantation to hypomethylating therapy or best supportive care in patients aged 50-75 with advanced myelodysplastic syndrome: Blood and marrow transplant clinical trials network study 1102*

Abstract: 75

Presenter: Corey Cutler, MD, MPH, FRCPC

Press Program Time: Friday, Dec. 4, 9:30 a.m. PST / 12:30 p.m. EST

Session Time: Saturday, Dec. 5, 7:30 a.m. PST / 10:30 a.m. EST

Recent advances in the treatment of myelodysplastic syndrome (MDS) have improved patient survival and quality of life, while reducing transfusion burden. However, allogeneic hematopoietic cell transplantation (HCT), widely used in younger MDS patients, remains the only curative therapy for MDS. While transplantation outcomes among selected older patients with MDS are similar to younger patients with MDS, early transplantation for older patients is infrequently offered since the relative benefits of HCT over non-HCT therapy have not been well defined in this patient group. This multi-center, biologic assignment trial in older individuals with high risk MDS defines the benefit of HCT over non-HCT therapy.

Title: DNMT3A clonal hematopoiesis in older donors is associated with improved survival in recipients after allogeneic hematopoietic cell transplant

Abstract: 80

Presenter: Christopher Gibson, MD

Session Time: Saturday, Dec. 5, 8:45 a.m. PST / 11:45 a.m. EST

Clonal hematopoiesis (CH) is an age-related condition in which somatic mutations can be detected in the blood of healthy individuals. In the non-transplant setting, CH is associated with an elevated risk of developing hematologic malignancy and an increased risk of non-hematologic outcomes due to altered inflammatory signaling. During hematopoietic cell transplantation (HCT), CH in older donors can engraft in recipients and could therefore influence outcomes through effects on graft immunologic function or by causing donor cell leukemia. A definitive link between donor CH and recipient outcomes has not been established. Dana-Farber researchers evaluated the impact of CH in donors aged 40 years or older on recipient clinical outcomes in 1,727 donor-recipient pairs.

Title: Safety and efficacy of adding venetoclax to reduced intensity conditioning chemotherapy prior to allogeneic hematopoietic cell transplantation in patients with high risk myeloid malignancies

Abstract: 190

Presenter: Jacqueline S. Garcia, MD

Session Time: Saturday, Dec. 5, 12:15 p.m. PST / 3:15 p.m. EST

The outcomes for patients with myeloid malignancies who relapse after allogeneic hematopoietic cell transplantation (HCT) is poor, and relapse occurs more frequently for those with high-risk mutations or cytogenetics. The oral selective BCL-2 inhibitor and BH3 mimetic venetoclax increases mitochondrial apoptotic priming, even in chemoresistant myeloblasts. Reasoning that venetoclax would selectively increase the anti-leukemic effect of HCT conditioning chemotherapy without undue toxicity, this study evaluated the safety and efficacy of adding venetoclax to fludarabine and busulfan reduced intensity conditioning chemotherapy. Dana-Farber researchers report on the completed dose-escalation and expansion cohorts from the phase 1 trial.

Title: Prognostic value of circulating tumor DNA (ctDNA) in autologous stem cell graft and post-transplant plasma samples among patients with diffuse large B-Cell lymphoma

Abstract: 531

Presenter: Reid Merryman, MD

Session Time: Monday, Dec. 7, 7:15 a.m. PST / 10:15 a.m. EST

While autologous stem cell transplantation (ASCT) can be curative for patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), relapse remains common. With the emergence of novel effective therapies, it is even more important to identify patients at high risk of treatment failure who may not benefit from ASCT, and patients with impending post-ASCT relapse who may be candidates for pre-emptive interventions. Researchers assembled cohorts of DLBCL patients who underwent ASCT and had apheresis stem cell samples or serially collected post-ASCT peripheral blood mononuclear cell and plasma samples. Researchers hypothesized that circulating tumor DNA identified using immunoglobulin-based next generation sequencing in apheresis stem cell or peripheral blood samples could predict relapse.

Title: Safety, Efficacy, and patient-reported outcomes of venetoclax in combination with azacitidine for the treatment of patients with higher-risk myelodysplastic syndrome: A phase 1b study

Abstract: 656

Presenter: Jacqueline S. Garcia, MD

Session Time: Monday, Dec.7, 12:15 p.m. PST / 3:15 p.m. EST

Hypomethylating agents form the current standard treatment for patients with higher-risk myelodysplastic syndrome who are not eligible for allogeneic hematopoietic stem cell transplantation (HSCT). However, overall response rates remain low in patients receiving azacitidine, and median overall survival is reported as ~15 months. In addition, there are few data on patient-reported outcomes published in this population while on treatment. Venetoclax is a selective, potent, orally bioavailable BCL-2 inhibitor, which has demonstrated synergy with hypomethylating agents in preclinical studies of higher-risk myelodysplastic syndrome. From an ongoing, open-label, dose-escalation, Phase 1b study evaluating venetoclax and azacitidine for the treatment of treatment-naïve higher-risk MDS, researchers report the updated safety and efficacy in all treated patients and the exploratory analysis of key patient-reported outcomes in patients who received the recommended Phase 2 dose (RP2D).

Title: Primary analysis of ZUMA-5: A phase 2 study of axicabtagene ciloleucel (axi-cel) in patients with relapsed/refractory (R/R) indolent non-Hodgkin lymphoma (iNHL)*

Abstract: 700

Presenter: Caron Jacobson, MD, MMSc

Press Program Time: Saturday, Dec. 5, 9:30 a.m. PST / 12:30 p.m. EST

Session Time: Monday, Dec. 7, 1:30 p.m. PST / 4:30 p.m. EST

Patients with advanced-stage indolent non-Hodgkin lymphoma, including follicular lymphoma and marginal zone lymphoma, frequently relapse with standard treatment, underscoring a need for novel therapies. Axi-cel autologous anti-CD19 chimeric antigen receptor (CAR) T cell therapy is approved for the treatment of relapsed/refractory large B cell lymphoma after more than two lines of systemic therapy. Dana-Farber will present the primary analysis of ZUMA-5, a Phase 2, multicenter, single-arm study of axi-cel in patients with relapsed/refractory indolent non-Hodgkin lymphoma.

On December 1, 2020 The Case-Coulter Translational Research Partnership (CCTRP) reported that it has announced more than $1.1 million in funding and other support for six biomedical technologies (Press release, Case Western Reserve University, DEC 1, 2020, View Source [SID1234572021]).

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The six Case Western Reserve projects were selected for full program funding, which ranges from $50,000 to $200,000 each. Several additional pilot projects have or will be awarded funding by the end of the year. All projects are partnerships between a clinician and a biomedical engineer, and are focused on solving areas of unmet healthcare needs.

The 14-year-old program—a collaboration between Case Western Reserve University and the Wallace H. Coulter Foundation—provides direct funding and support services to help campus research teams advance products from their laboratories to the marketplace, where they can improve patient care.

Funding supports steps involved in preparing projects for potential commercialization, such as demonstrating technical feasibility, analyzing the business opportunity and assessing market feasibility. Projects must have the potential to advance to a commercial entity within 12 to 30 months.

More than two-dozen companies have emerged from the partnership’s support, and licensing arrangements have enabled three-dozen technologies to reach patients. For each dollar the partnership has invested, the projects have garnered an additional $25 of investment.

"The Case-Coulter Translational Research Partnership continues to be a cornerstone, filling an essential gap to transition university biomedical technologies from research to products, where they can significantly improve the health of our society," said Robert Kirsch, the Allen H. and Constance T. Ford Professor and chair of the university’s Department of Biomedical Engineering.

"As a group, the quality of the evaluated technologies continues to improve each year, demonstrating the robustness of the biomedical research-based technology pipeline," said Stephen Fening, CCTRP director. "We had many more proposals that were deserving of inclusion into the program than we were able to accommodate, making the selection process more challenging than ever."

The six projects selected and their inventors are:

10-liter Scale Production of BG34-200 Immunotherapeutic Under cGMP Guidelines
Mei Zhang, assistant professor of biomedical engineering, and Alex Huang, professor of pediatrics and pathology.

A significant portion of patients with solid tumor cancers do not respond to immunotherapies due to a lack of T-cell-inflamed tumor microenvironment. This novel plant-derived non-toxic BG34-200 molecule can be intravenously injected to modulate macrophages and create a tumor microenvironment that is vital for the generation of antitumor T-cell responses. The team is launching a clinical trial targeting canine metastatic osteosarcoma to collect key and gap data in preparation for a first human clinical trial targeting pediatric osteosarcoma.

Drug-Free Targeted Prostate Cancer Treatment with TNT – Targeted Nanobubble Therapy
Agata Exner, professor of radiology and biomedical engineering, Jim Basilion, professor of radiology and biomedical engineering, and Lee Ponsky, professor of urology

Drug-free, low-toxicity prostate cancer treatment using nanobubbles (NBs) are targeted to the prostate specific membrane antigen (PSMA) biomarker overexpressed on prostate tumor cells. The nanobubbles are injected into the bloodstream and specifically seek out only the cancer cells. Once inside the target cell, the NBs remain trapped and can be excited with an ultrasound pulse. Exposure to ultrasound results in collapse of the bubbles, leading to a highly focused mechanical disruption of the cancer cells and cell death. The approach, which we call TNT (targeted nanobubble therapy) can fit into the existing clinical work-flow and can be carried out with standard clinical ultrasound equipment. TNT can treat tumors without severe side effects, as it will be effective only when NBs are sonicated and will destroy only the cancer cells and not the surrounding healthy cells.

TraumaChek: A Field-deployable Dielectric Coagulometer for Comprehensive Assessment of Trauma-induced Coagulopathy
Anirban Sen Gupta, professor of biomedical engineering, Pedram Mohseni, professor of electrical engineering and biomedical engineering, and Sanjay Ahuja, professor of pediatrics

TraumaChek is a multichannel, handheld blood-coagulation analyzer for early, rapid and comprehensive assessment of trauma-induced coagulopathy. It is designed to guide hemorrhage control, transfusion and resuscitative management of trauma at the point-of-injury by first responders and at the point-of-care by hospital clinicians.

Minimally Invasive Interfascicular Nerve Stimulation (MiiNS) System for Chronic Pain Management
Dustin Tyler, the Kent H. Smith Professor of Biomedical Engineering, Emily Graczyk, research assistant professor of biomedical engineering, and Jennifer Sweet, professor of neurological surgery

This is a drug-free technology to provide targeted, comfortable, worry-free relief to people suffering from long-term pain. The discomfort and emotional stress from pain affects a person’s activity, sleep and ability to live a healthy life, leading to other serious health problems. Their Minimally Invasive Interfascicular Nerve Stimulation (MiiNS) technology provides a targeted, personally customized and comfortable treatment without side effects, addiction or surgical procedures. MiiNS can be implanted by a doctor during a simple office visit to provide long-lasting pain relief.

BAFF CAR-NK Cells for Therapy of B Cell Malignancies
Reshmi Parameswaran, assistant professor of medicine, and Pallavi Tiwari, assistant professor of biomedical engineering

BAFF CAR-­NK cells can specifically kill B cell cancers in a very effective manner with minimum side effects. This is a potential curative therapeutic strategy for patients who are not responding to current treatment methods.

PhotoSorb: Safe and Long-lasting Sunscreen
Vijay Krishna, assistant staff in Cleveland Clinic’s Department of Biomedical Engineering, and Edward Maytin, staff in Cleveland Clinic’s Department of Dermatology

Every year, more than a million new cases of skin cancer, including melanoma, are diagnosed in the United States. The primary cause is exposure to ultraviolet radiation (UV) from sunlight. Sunscreens can block UV, but increasing concerns about the health and environmental risks of chemical sunscreens now on the market underscores an urgent need for safer, more effective alternatives. A team from biomedical engineering and dermatology at Cleveland Clinic is developing a novel sunscreen (PhotoSorb) that appears to be safer and more stable than current sunscreens, and also has the potential to actually prevent skin cancers.

On December 1, 2020 Odonate Therapeutics, Inc. (NASDAQ: ODT), a pharmaceutical company dedicated to the development of best-in-class therapeutics that improve and extend the lives of patients with cancer, reported that it will host a virtual Investor and Analyst Event on Friday, December 11, 2020, at 1:00 p.m. CT / 2:00 p.m. ET (Press release, Odonate Therapeutics, DEC 1, 2020, View Source [SID1234572037]). The event will follow the presentation of the results of CONTESSA, a Phase 3 study of tesetaxel in patients with metastatic breast cancer, at the 2020 SABCS, which is scheduled to occur at 8:45 a.m. CT / 9:45 a.m. ET on December 11, 2020 (View Source). Featured speakers will include Lee Schwartzberg, M.D., FACP, Chief Medical Director, West Cancer Center & Research Institute, and Andrew Seidman, M.D., Medical Director, Bobst International Center, Memorial Sloan Kettering Cancer Center and Professor of Medicine, Weill Cornell Medical College.

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Event Information

About Tesetaxel

Tesetaxel is an investigational, orally administered chemotherapy agent that belongs to a class of drugs known as taxanes, which are widely used in the treatment of cancer. Tesetaxel has several pharmacologic properties that make it unique among taxanes, including: oral administration with a low pill burden; a long (~8-day) terminal plasma half-life in humans, enabling the maintenance of adequate drug levels with relatively infrequent dosing; no history of hypersensitivity (allergic) reactions; and significant activity against chemotherapy-resistant tumors. In patients with metastatic breast cancer, tesetaxel was shown to have significant, single-agent antitumor activity in two multicenter, Phase 2 studies. Tesetaxel currently is the subject of three studies in breast cancer, including a multinational, multicenter, randomized, Phase 3 study in patients with metastatic breast cancer, known as CONTESSA. Odonate recently announced positive top-line results from CONTESSA, and full results are scheduled to be presented at the San Antonio Breast Cancer Symposium in December 2020.

About CONTESSA

CONTESSA is a multinational, multicenter, randomized, Phase 3 study of tesetaxel, an investigational, orally administered taxane, in patients with metastatic breast cancer (MBC). CONTESSA is comparing tesetaxel dosed orally at 27 mg/m2 on the first day of each 21-day cycle plus a reduced dose of capecitabine (1,650 mg/m2/day dosed orally for 14 days of each 21-day cycle) to the approved dose of capecitabine alone (2,500 mg/m2/day dosed orally for 14 days of each 21-day cycle) in 685 patients randomized 1:1 with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative MBC previously treated with a taxane in the neoadjuvant or adjuvant setting. Capecitabine is an oral chemotherapy agent that is considered a standard-of-care treatment in MBC. Where indicated, patients must have received endocrine therapy with or without a cyclin-dependent kinase (CDK) 4/6 inhibitor. The primary endpoint is progression-free survival (PFS) as assessed by an Independent Radiologic Review Committee (IRC). The secondary efficacy endpoints are overall survival (OS), objective response rate (ORR) as assessed by the IRC and disease control rate (DCR) as assessed by the IRC.

On December 1, 2020 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported that the U.S. Food and Drug Administration (FDA) has approved Gavreto (pralsetinib) for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic rearranged during transfection (RET)-mutant medullary thyroid cancer (MTC) who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) (Press release, Genentech, DEC 1, 2020, View Source [SID1234572052]). These indications were approved under the FDA’s accelerated approval program based on data from the Phase I/II ARROW study. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

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"We are proud to partner with Blueprint Medicines to bring this important new option to people with certain types of RET-altered thyroid cancer," said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. "Gavreto is now approved across multiple RET-altered tumor types, underscoring our commitment to advancing personalized healthcare with treatments that target the underlying biology of each person’s cancer."

Approximately 10-20% of people with papillary thyroid cancer (the most common type of thyroid cancer) have RET fusion-positive tumors, and roughly 90% of people with advanced MTC (a rare form of thyroid cancer) carry RET mutations. Biomarker testing for RET fusions and mutations can help identify people who are eligible for treatment with Gavreto.

The approvals are based on results from the Phase I/II ARROW study, which demonstrated durable clinical activity in people with or without prior therapy and regardless of RET alteration genotypes. Treatment with Gavreto led to an overall response rate (ORR) of 60% (95% CI: 46%, 73%) in 55 people with RET-mutant metastatic MTC previously treated with cabozantinib and/or vandetanib, and the median duration of response (DoR) was not reached (95% CI: 15.1 months, not estimable). In 29 people with RET-mutant advanced MTC who were cabozantinib and vandetanib treatment-naïve, the ORR was 66% (95% CI: 46%, 82%), and the median DoR was not reached (95% CI: not estimable, not estimable). In nine people with RET fusion-positive metastatic thyroid cancer, Gavreto demonstrated an ORR of 89% (95% CI: 52%, 100%), and the median DoR was not reached (95% CI: not estimable, not estimable). In ARROW trial patients across RET-altered tumor types, the most common adverse reactions (≥25%) were constipation, increased blood pressure (hypertension), fatigue, musculoskeletal pain and diarrhea.

The FDA reviewed and approved the application under its Real-Time Oncology Review (RTOR) pilot program, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible. In September, the FDA also granted accelerated approval to Gavreto for the treatment of adults with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. In addition, the FDA granted Breakthrough Therapy Designation to Gavreto for the treatment of RET mutation-positive MTC that requires systemic treatment and for which there are no acceptable alternative treatments and for RET fusion-positive NSCLC that has progressed following platinum-based chemotherapy.

Gavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations. Gavreto is jointly commercialized by Genentech and Blueprint Medicines in the United States. For those who qualify, Blueprint Medicines offers patient assistance programs for people prescribed Gavreto by their doctor through YourBlueprint. Please visit www.yourblueprint.com or contact 1-888-BLUPRNT for more information.

About the ARROW study

ARROW (NCT03037385) is a Phase I/II, open-label, first-in-human study designed to evaluate the safety, tolerability and efficacy of Gavreto, administered orally in people with rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer (MTC), RET fusion-positive thyroid cancers and other RET-altered solid tumors. The trial consists of two parts: a dose escalation portion, which is complete, and an expansion portion in people treated with 400 mg of Gavreto, once-daily. ARROW is being conducted at multiple sites across the United States, European Union and Asia.

About RET-altered cancers

RET gene alterations, such as fusions and mutations, are key disease drivers in many types of cancer, including NSCLC and several types of thyroid cancers. Approximately 10-20% of people with papillary thyroid cancer (the most common type of thyroid cancer) have RET fusion-positive tumors, and roughly 90% of people with advanced MTC (a rare form of thyroid cancer) carry RET mutations. In NSCLC, RET fusions represent approximately 1-2% of patients. Oncogenic RET fusions also are observed at low frequencies in cholangiocarcinoma, colorectal, neuroendocrine, ovarian, pancreatic and thymus cancers.

About Gavreto

Gavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations, regardless of the tissue of origin. Preclinical data have shown that Gavreto inhibits primary RET fusions and mutations that cause cancer in subsets of patients, as well as secondary RET mutations predicted to drive resistance to treatment. Blueprint Medicines and Genentech are co-developing Gavreto for the treatment of patients with various types of RET-altered cancers.

Gavreto U.S. Indications

Gavreto (pralsetinib) is indicated for the treatment of:

Adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test.
Adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy.
Adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)
These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

Important Safety Information

Interstitial Lung Disease (ILD)/Pneumonitis occurred in 10% of patients who received Gavreto, including 2.7% with Grade 3/4, and 0.5% with fatal reactions. Monitor for pulmonary symptoms indicative of interstitial lung disease (ILD)/pneumonitis. Withhold Gavreto and promptly investigate for ILD in any patient who presents with acute or worsening of respiratory symptoms (e.g., dyspnea, cough, and fever). Withhold, reduce dose or permanently discontinue Gavreto based on severity of confirmed ILD.

Hypertension occurred in 29% of patients, including Grade 3 hypertension in 14% of patients. Overall, 7% had their dose interrupted and 3.2% had their dose reduced for hypertension. Treatment-emergent hypertension was most commonly managed with anti-hypertension medications. Do not initiate Gavreto in patients with uncontrolled hypertension. Optimize blood pressure prior to initiating Gavreto. Monitor blood pressure after 1 week, at least monthly thereafter and as clinically indicated. Initiate or adjust anti-hypertensive therapy as appropriate. Withhold, reduce dose, or permanently discontinue Gavreto based on the severity.

Hepatotoxicity: Serious hepatic adverse reactions occurred in 2.1% of patients treated with Gavreto. Increased aspartate aminotransferase (AST) occurred in 69% of patients, including Grade 3/4 in 5% and increased alanine aminotransferase (ALT) occurred in 46% of patients, including Grade 3/4 in 6%. The median time to first onset for increased AST was 15 days (range: 5 days to 1.5 years) and increased ALT was 22 days (range: 7 days to 1.7 years). Monitor AST and ALT prior to initiating Gavreto, every 2 weeks during the first 3 months, then monthly thereafter and as clinically indicated. Withhold, reduce dose or permanently discontinue Gavreto based on severity.

Grade ≥ 3 hemorrhagic events occurred in 2.5% of patients treated with Gavreto including one patient with a fatal hemorrhagic event. Permanently discontinue Gavreto in patients with severe or life-threatening hemorrhage.

Tumor Lysis Syndrome (TLS): Cases of TLS have been reported in patients with medullary thyroid carcinoma receiving Gavreto. Patients may be at risk of TLS if they have rapidly growing tumors, a high tumor burden, renal dysfunction, or dehydration. Closely monitor patients at risk, consider appropriate prophylaxis including hydration, and treat as clinically indicated.

Impaired wound healing can occur in patients who receive drugs that inhibit the vascular endothelial growth factor (VEGF) signaling pathway. Therefore, Gavreto has the potential to adversely affect wound healing. Withhold Gavreto for at least 5 days prior to elective surgery. Do not administer for at least 2 weeks following major surgery and until adequate wound healing. The safety of resumption of Gavreto after resolution of wound healing complications has not been established.

Based on findings from animal studies and its mechanism of action, Gavreto can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with Gavreto and for 2 weeks after the final dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with Gavreto and for 1 week after the final dose. Advise women not to breastfeed during treatment with Gavreto and for 1 week after the final dose.

Common adverse reactions (≥25%) were constipation, hypertension, fatigue, musculoskeletal pain and diarrhea. Common Grade 3/4 laboratory abnormalities (≥2%) were decreased lymphocytes, decreased neutrophils, decreased hemoglobin, decreased phosphate, decreased calcium (corrected), decreased sodium, increased AST, increased ALT, decreased platelets and increased alkaline phosphatase.

Avoid coadministration of Gavreto with strong CYP3A inhibitors or combined P-gp and strong CYP3A inhibitors. If coadministration cannot be avoided, reduce the Gavreto dose. Avoid coadministration of Gavreto with strong CYP3A inducers. If coadministration cannot be avoided, increase the Gavreto dose.

Please click here to see the full Prescribing Information for Gavreto.

Blueprint Medicines, Gavreto, YourBlueprint and associated logos are trademarks of Blueprint Medicines Corporation.

On December 1, 2020 Step Pharma SAS, in collaboration with Sygnature Discovery, reported that it has been selected to present a poster named "STP938, a Novel, Potent and Selective Inhibitor of CTP Synthase 1 (CTPS1) Is a Targeted Therapy Specifically Blocking De Novo Nucleotide Synthesis in Lymphomas and Leukemias" at the American Society of Hematology (ASH) (Free ASH Whitepaper) conference starting December 5th (Press release, Step Pharma, DEC 1, 2020, View Source [SID1234572070]).

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