On September 21, 2023 Dizal reported that the China National Medical Products Administration (NMPA) has granted priority review status to golidocitinib, Dizal’s investigational JAK1-only inhibitor, for the treatment of relapsed or refractory peripheral T-cell lymphoma (r/r PTCL) (Press release, Dizal Pharma, SEP 21, 2023, View Source [SID1234635321]). In February 2022, golidocitinib received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for the same indication, making it the first and only innovative drug in China to receive this recognition for PTCL.

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"Patients with relapsed or refractory PTCL worldwide face limited treatment options and a poor prognosis. We are pleased with the CDE’s decision to grant priority review status to golidocitinib, as it recognizes the potential of this groundbreaking drug to improve survival benefits for this underserved patient population. We are fully committed to expediting the NDA filing process and ensuring that golidocitinib becomes available to Chinese patients as quickly as possible." said Xiaolin Zhang, PhD, Chairman and CEO of Dizal.

Golidocitinib, a JAK1-only inhibitor, aimed to address the r/r PTCL dilemma

PTCL is an aggressive non-Hodgkin lymphoma (NHL) that represents approximately 7% to 10% of NHLs worldwide. Patients with r/r PTCL face a poor prognosis, with a 3-year survival rate ranging from 21% to 28%. Currently, there is no established consensus on the standard approach for treating r/r PTCL. Therefore, there is an urgent need for novel and innovative treatment options to improve survival in this patient population.

The Priority Review designation for golidocitinib’s application was supported by data from the JACKPOT8 PARTB study, a multinational, pivotal study to evaluate the efficacy and safety of golidocitinib in patients with r/r PTCL. The primary endpoint of the study, objective response rate (ORR) assessed by an independent review committee (IRC), reached 44.3%, with a complete response rate (CRR) of 23.9%. Anti-tumor efficacy was observed across different PTCL subtypes and irrespective of the patients’ prior treatment history. The majority of treatment-related adverse events (TRAEs) could be monitored and well managed in the clinic. These findings, highlighting the superior efficacy and safety of golidocitinib, have been widely acknowledged at prestigious conferences such as ASCO (Free ASCO Whitepaper), EHA (Free EHA Whitepaper), ICML, and ASH (Free ASH Whitepaper) with five oral presentations for four consecutive years. Recently, the Phase I clinical data of golidocitinib for the treatment of r/rPTCL (JACKPOT8 PARTA) was published in the esteemed peer-reviewed journal, Annals of Oncology (Impact Factor: 51.8).

Driving innovation through the fundamentals of translational science

Dizal, a spinoff of AstraZeneca’s oncology translational science center, has established a portfolio of five clinical-stage assets with global competitiveness. Among these, golidocitinib represents a major research achievement deeply rooted in the principles of translational science. Dizal was the first to identify and validate the JAK/STAT pathway as a highly promising therapeutic approach for PTCL, leading to the development of golidocitinib as the world’s first and currently the only JAK1-only inhibitor in pivotal trial for T cell lymphoma.

About golidocitinib (DZD4205)

Golidocitinib is the first-in-class Janus kinase 1 (JAK1) only inhibitor currently being evaluated in a global, multicenter pivotal study (JACKPOT8 PARTB) in r/r PTCL. At the data cut-off date of February 16, 2023, Golidocitinib has demonstrated robust and durable anti-tumor activity, with an ORR of 44.3% and a CRR of 23.9%. More than 50% of the patients with tumor remission achieved a complete response. The median relative dose intensity was 100%. Golidocitinib was granted Fast Track Designation by the U.S. FDA for the treatment of r/r PTCL in February 2022. In September 2023, the CDE accepted the NDA and granted the Priority Review status for the treatment of r/r PTCL. And the Phase I clinical data of golidocitinib for the treatment of r/r PTCL (JACKPOT8 PARTA) was published in Annals of Oncology (Impact Factor: 51.8).

On September 21, 2023 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the European Commission (EC) has granted marketing authorization for Enrylaze (JZP458; a recombinant Erwinia asparaginase or crisantaspase) for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients (1 month and older) who developed hypersensitivity or silent inactivation to E. coli-derived asparaginase (Press release, Jazz Pharmaceuticals, SEP 21, 2023, View Source [SID1234635320]). Enrylaze, approved as Rylaze in the United States and Canada, is a new Erwinia-derived asparaginase developed using a next-generation recombinant technology with a safety profile consistent with that of other asparaginase preparations.1,2,3,4,5

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"Asparaginase is a core component of multi-agent chemotherapeutic regimens for the treatment of ALL, however, up to 30% of patients develop hypersensitivity to E. coli-derived asparaginase, resulting in a delay or disruption in treatment," said Professor Carmelo Rizzari, Department of Pediatrics, University of Milano-Bicocca, Head of the Pediatric Hematology Oncology Unit, Foundation IRCCS San Gerardo dei Tintori, Monza, Italy. "The ability to complete a full course of asparaginase treatment is of critical importance when treating ALL and LBL, as it is strongly linked to improved outcomes for patients. The approval of Enrylaze now provides an important option to support patients in completing their planned asparaginase treatment regimen."

Enrylaze may be given by both intravenous infusion (IV) and intramuscular injection (IM) and is dosed on either alternate days (every 48 hours) or via a Monday/Wednesday/Friday (MWF) dosing schedule.1 The use of recombinant technology to manufacture Enrylaze delivers a scalable supply – able to meet global demand, and a ready-to-use solution that avoids the need for reconstitution in the clinic.2

"This approval is a testament to Jazz’s commitment to developing an Erwinia-derived asparaginase using innovative recombinant technology to deliver a scalable supply, and we look forward to making Enrylaze available to those who need it," said Robert Iannone, MD., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "Healthcare professionals in the European Union will now have access to a new, recombinant Erwinia-derived asparaginase with multiple dosing and administration options to address their patients’ individual needs, which allows them to complete their treatment program as prescribed."

The EC approval is based on data from a Phase 2/3 trial conducted in collaboration with the Children’s Oncology Group (COG) in a cohort of 228 pediatric and adult patients with ALL and LBL who have developed hypersensitivity or silent inactivation to E. coli-derived asparaginase. The study was conducted in two parts to assess the IV and IM routes of administration.1,3 The determination of efficacy was based on demonstration of the achievement and maintenance of nadir serum asparaginase activity (NSAA) levels ≥ 0.1 U/mL.1

The study showed that for the IV administration of JZP458 (a recombinant Erwinia asparaginase or crisantaspase) (25/25/50 mg/m2 MWF), the proportion of patients maintaining NSAA ≥ 0.1 U/mL at 48 hours after a dose was 89.8% (95% CI: 82.1%, 97.5%) and 40% at 72 hours post-dose (95% CI: 26.4%, 53.6%). The IM administration of JZP458 (25/25/50 mg/m2 MWF) achieved sustained asparagine activity in 95.9% of patients at 48 hours after a dose (95% CI: 90.4%, 100.0%) and 89.8% of patients at 72 hours post-dose (95% CI: 81.3%, 98.3%). The other dosing schedules were based on interpolation from pharmacokinetic (PK) and response rates observed with the very similar investigated regimens.1

Overall, the safety profile of JZP458 was consistent with the reported safety information for patients with ALL/LBL receiving asparaginase with combination chemotherapy.1,3 The most common adverse reactions were anemia, vomiting, thrombocytopenia, neutropenia, nausea, febrile neutropenia, fatigue, pyrexia, decreased appetite, transaminase increased, abdominal pain, white blood cell count decreased, headache, diarrhea, and lymphocyte count decreased. The most frequent serious adverse reactions were febrile neutropenia, pyrexia, vomiting, sepsis, medicinal product hypersensitivity, nausea, and pancreatitis.1

The European Commission approval extends to all European Union Member States, as well as Iceland, Norway, and Liechtenstein.

For a full list of side effects and information on dosage and administration, contraindications, and other precautions when using Enrylaze, please refer to the Summary of Product Characteristics for further information.

About Enrylaze (JZP458) Enrylaze, also known as JZP458 and approved as Rylaze in the United States and Canada, is the only recombinant Erwinia asparaginase or crisantaspase that is derived from a Pseudomonas fluorescens expression platform.2,4,5 It is approved for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients (1 month and older) who developed hypersensitivity or silent inactivation to E. coli-derived asparaginase products. JZP458 was approved by the U.S. Food and Drug Administration (FDA) in June 2021 for the treatment of this patient population and became commercially available in July of the same year in the U.S.4

About Study JZP458-201

The EC approval of Enrylaze is based on clinical data from the pivotal Phase 2/3 single-arm, open-label, multicenter, dose confirmation study evaluating 228 pediatric and adult patients with ALL or LBL who have developed hypersensitivity or silent inactivation to E. coli-derived asparaginases and have not previously received Erwinia-derived asparaginase. The study was designed to assess the safety, tolerability, and efficacy of JZP458. The determination of efficacy was measured by serum asparaginase activity (SAA) levels. The Phase 2/3 study was conducted in two parts. The first part investigated the intramuscular (IM) route of administration, including a Monday-Wednesday-Friday dosing schedule. The second investigated the dose and schedule for the intravenous (IV) route of administration.1,3

About Acute Lymphoblastic Leukemia (ALL)
Acute lymphoblastic leukemia (ALL) is a cancer of the blood and bone marrow that can progress quickly if not treated.6,7 ALL is the most common childhood malignancy, accounting for 80% of leukemia diagnoses in children, compared to 20% of adults.8 Long-term survival rates for pediatric patients have improved significantly over the last few decades, which is in part a result of crafting effective combinations of multi-agent chemotherapeutics with an asparaginase backbone.9 The estimated overall incidence of ALL and lymphoblastic lymphoma (LBL) in Europe is 1.28 per 100,000.10 The number of ALL global cases in children was 59,100 in 2017.11

Asparaginase is a core component of multi-agent chemotherapeutic regimens in ALL,12 however, up to 30% of patients develop hypersensitivity to E. coli-derived asparaginase,13 necessitating treatment discontinuation or a switch to a non-E. coli-derived asparaginase preparation.14 Patients not receiving asparaginase due to hypersensitivities and those not receiving all prescribed doses have been shown to have poor outcomes.2,15

About Lymphoblastic Lymphoma (LBL)

Lymphoblastic Lymphoma (LBL) is a rare, fast-growing, aggressive subtype of non-Hodgkin’s lymphoma (NHL), which is very rare in adults and is most often seen in teenagers and young adults under the age of 35.16,17 LBL is a type of high-grade lymphoma – which means the lymphoma grows quickly with early spread to different parts of the body.17 LBL is the second most common type of NHL in childhood and adolescence, accounting for 25-35% of cases.

On September 21, 2023 Affimed N.V. (Nasdaq: AFMD) ("Affimed", or the "Company"), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, reported that its Chief Financial Officer, Angus Smith, will participate in a fireside chat at the Cantor Global Healthcare Conference 2023 on Thursday, September 28, 2023 at 1:15 p.m. Eastern Daylight Time / 19:15 Central European Time (Press release, Affimed, SEP 21, 2023, View Source [SID1234635318]).

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A live webcast of the presentation will be accessible on Affimed’s website at View Source A replay of the call will be archived on Affimed’s website for 30 days after the call.

For more information on the conference or to schedule a one-on-one meeting with Affimed’s management, please contact your Cantor representative or Alex Fudukidis via email at [email protected] or phone at +1 (917) 436-8102.

On September 21, 2023 Veracyte, Inc. (Nasdaq: VCYT) reported that three posters showcasing new data from the company’s Afirma Genomic Sequencing Classifier (GSC) will be presented at the 2023 American Thyroid Association (ATA) Annual Meeting being held in Washington, DC from September 27 to October 1 (Press release, Veracyte, SEP 21, 2023, View Source [SID1234635317]). Together, these abstracts offer novel insights into the molecular underpinnings of thyroid nodules and tumors based on whole-transcriptome RNA sequencing data from thyroid nodules analyzed with the Afirma GSC.

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"As we enter a new era in the personalized diagnosis and management of thyroid nodules and cancer, it is more important than ever to advance the science around this cancer so we can ultimately characterize each patient’s individual molecular profile," said Joshua Klopper, MD, Veracyte’s medical director for Endocrinology. "The abstracts to be presented by our clinical research colleagues at the ATA meeting show that the Afirma GSC platform can be used to glean new insights about thyroid nodules and thyroid cancer preoperatively. We believe that Afirma-based whole-transcriptome analysis will help drive cancer research and unlock findings that may ultimately be used in clinical care."

The following late-breaking posters will be presented at the 2023 ATA Annual Meeting (all times ET):

Title: Leveraging RNA Sequencing for Pre-Operative Immunophenotyping of BRAFV600E+ Thyroid Nodules
Presenter: Jarod Olay, M.S., UCLA David Geffen School of Medicine
Poster #: 502
Date/Time: September 28; 10:00 a.m. – 10:30 a.m. and 11:35 a.m. – 12:45 p.m.
Title: Molecular Assessment of Isthmus Thyroid Carcinomas

Presenter: Sina Jasim, M.D., Washington University in St. Louis
Poster #: 501
Date/Time: September 28; 10:00 a.m. – 10:30 a.m. and 11:35 a.m. – 12:45 p.m.
Title: Sodium Iodide Symporter (NIS) Expression in Cytologically Indeterminate and Malignant Thyroid Nodules

Presenter: Prasana Santhanam, M.B.B.S., M.D., The Johns Hopkins University School of Medicine
Poster #: 515
Date/Time: September 28; 10:00 a.m. – 10:30 a.m. and 11:35 a.m. – 12:45 p.m.

These abstracts will later be published as an online supplement to Thyroid, the official journal of the ATA.

On September 21, 2023 Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), a clinical stage immuno-oncology company focused on the discovery and development of next-generation therapeutics for cancer patients, reported that it will present new preclinical data from SNS-101, a clinical-stage conditionally active, human monoclonal antibody targeting the immune checkpoint VISTA (V-domain Ig suppressor of T cell activation), at the Seventh Annual CRI-ENCI-AACR International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper): Translating Science into Survival, held on September 20 – 23, 2023 in Milan, Italy (Press release, Sensei Biotherapeutics, SEP 21, 2023, View Source [SID1234635316]).

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"We believe these data further support the potential best-in-class PK and safety profile of SNS-101 and its ability to effectively target VISTA, a promising, but difficult-to-drug immune checkpoint," said Edward van der Horst, Ph.D., Chief Scientific Officer of Sensei Biotherapeutics. "Restricting drug activity to low pH conditions, such as those found in the acidic tumor microenvironment, allowed us to safely dose SNS-101 in our multi-dose NHP study at increasing levels while maintaining linear elimination kinetics and displaying no target-mediated drug disposition or adverse effects. Paired with new mechanistic data showing SNS-101 can shift immunosuppressive macrophages towards an anti-tumor phenotype, we believe SNS-101 could provide a transformative treatment option for patients. We look forward to sharing data from our ongoing clinical trial later this year."

Presentation Highlights:

SNS-101 displayed linear elimination kinetics and no target-mediated drug disposition or adverse effects in a multi-dose GLP toxicology study in cynomolgus monkeys evaluating SNS-101 doses of 1, 10 and 100 mg/kg.
In MC38 tumor-bearing mice, SNS-101 alone and in combination with an anti-PD-1 antibody shifted the cytokine expression profile of immunosuppressive myeloid cells in the tumor microenvironment to an anti-tumor, M1 phenotype. The expression of anti-tumor cytokines was correlated with a reduction in tumor size.
Sensei is evaluating SNS-101 in a Phase 1/2 clinical trial in patients with advanced solid tumors, and expects to report initial monotherapy pharmacokinetic and safety data by year-end 2023. Topline monotherapy and initial combination data are anticipated in 2024.

The full poster is available for viewing on Sensei’s website and will be on display at the conference during Poster Session A, being held today, September 21, 2023, from 12:30 pm to 2:00 pm CET.