On November 20, 2024 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral targeted agents to treat hematologic malignancies, reported initiation of the TUSCANY study, tuspetinib (TUS) in combination therapy with azacitidine (AZA) and venetoclax (VEN) as a frontline triplet combination therapy for patients newly diagnosed with acute myeloid leukemia, or AML (Press release, Aptose Biosciences, NOV 20, 2024, View Source [SID1234648513]). The trial is being conducted at multiple U.S. clinical sites.

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Tuspetinib is being developed for frontline AML therapy as part of the TUS+VEN+AZA triplet for newly diagnosed AML patients ineligible to receive intensive chemotherapy. Tuspetinib, a convenient once daily oral agent that potently targets SYK, FLT3, mutated KIT, JAK1/2, and RSK2 kinases, avoids many typical toxicity concerns observed with other agents and has the potential to treat the larger AML populations, not just narrow subpopulations. In the recently conducted Phase 1/2 APTIVATE trial in a very ill and heavily pre-treated relapsed or refractory (R/R) AML population, tuspetinib as a single agent (TUS) and in combination with venetoclax (TUS+VEN) safely achieved broad activity across various difficult-to-treat AML subpopulations. This included patients with prior-VEN, prior-FLT3 inhibitor (FLT3i) and prior-hematopoietic stem cell transplantation (HSCT) therapies, those with highly adverse genetics – including mutations in TP53 and RAS genes, and those with mutated or unmutated (wildtype) FLT3 genes.

"Initiation of the trial is a key milestone for Aptose. AML treatment has rapidly shifted to combination therapies, and we are pleased to include tuspetinib as part of TUS+VEN+AZA triplet combination therapy in patients with newly-diagnosed AML – representing a new patient population for TUS," said William G. Rice, Chairman, President and Chief Executive Officer. "We thank our investigators for their enthusiasm and our clinical team for activating the TUSCANY triplet study. As one of our investigators noted, if TUS brings added efficacy to frontline treatment of a broad array of AML patients without the added toxicities that are plaguing some other agents, we may have a game changer in TUS."

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study
The triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard of care dosing of azacitidine and venetoclax. TUS will be administered in 28-day cycles, beginning with 40mg, with dose escalations planned after safety review of each dose level. A planned 12 sites in the US will enroll in the TUSCANY trial with anticipated enrollment of 18-24 patients by mid-late 2025.

On November 20, 2024 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported the initiation of its ALISertib in CAncer (ALISCA-Breast1) Phase II trial (PUMA-ALI-1201; NCT06369285) of alisertib in combination with endocrine therapy for the treatment of patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-negative) recurrent or metastatic breast cancer who have been previously treated with CDK 4/6 inhibitors and received at least two prior lines of endocrine therapy in the recurrent or metastatic setting (Press release, Puma Biotechnology, NOV 20, 2024, View Source [SID1234648532]). The ALISCA-Breast1 trial will enroll up to 150 patients who will be randomized (1:1:1) to receive alisertib dosed at either 30 mg, 40 mg or 50 mg twice daily on days 1-3, 8-10 and 15-17 in a 28-day cycle in combination with the endocrine therapy of the investigator’s choice. Patients must provide blood and tissue specimens so that biomarkers can be analyzed.

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"Additional therapies are needed for patients with HER2-negative, HR+ metastatic breast cancer whose disease progresses on CDK4/6 inhibitors in the first-line setting"

The primary objective of the trial is to determine the optimal alisertib dose in combination with selected endocrine therapy. The primary endpoints of the trial include objective response rate, duration of response, disease control rate, progression-free survival, and overall survival. As a secondary endpoint, Puma will evaluate each of these efficacy endpoints within biomarker subgroups in order to determine whether any biomarker subgroup correlates with response. Puma will perform its biomarker analysis of the ALISCA-Breast1 trial in parallel with the execution of the clinical trial. Puma plans to perform an initial interim analysis for the evaluation of safety and efficacy.

Based upon the outcomes of the trial, Puma anticipates meeting with the U.S. Food and Drug Administration to explore the potential for an approval pathway for alisertib in HER2-negative, HR+ metastatic breast cancer. Once the optimal alisertib dose is identified, Puma plans to engage with global regulatory agencies regarding the design of a pivotal (Phase III) trial, which it anticipates will be a randomized trial of alisertib plus investigator’s choice endocrine therapy versus placebo plus investigator’s choice endocrine therapy in patients with HER2-negative, HR+ metastatic breast cancer.

"Additional therapies are needed for patients with HER2-negative, HR+ metastatic breast cancer whose disease progresses on CDK4/6 inhibitors in the first-line setting," said Joyce A. O’Shaughnessy, M.D., the Celebrating Women Chair in Breast Cancer Research at Baylor University Medical Center, Texas Oncology, Sarah Cannon Research Institute in Dallas, Texas. "The results from the TBCRC 041 trial indicated that alisertib has impressive clinical activity in the setting of endocrine therapy and CDK4/6 inhibitor-resistant metastatic breast cancer, with good tolerability. I look forward to the further evaluation of alisertib in the ALISCA-Breast1 trial to definitively determine the clinical impact of this treatment."

Alan H. Auerbach, Chief Executive Officer, President and Founder of Puma, stated, "We are excited to initiate this Phase II trial and to move forward with the development of alisertib in HER2-negative HR+ metastatic breast cancer. We believe that the data from the previous trial of alisertib monotherapy (published in Lancet Oncology) as well as the TBCRC 041 trial (published in JAMA Oncology), which tested alisertib alone and with fulvestrant, and the randomized trial of alisertib plus paclitaxel versus paclitaxel alone (published in JAMA Network Open) have demonstrated that alisertib is active in patients with HER2-negative, HR+ metastatic breast cancer and in biomarker focused subgroups. We look forward to enrollment in the ALISCA-Breast1 trial and anticipate that we should have initial data from this trial in 2025."

On November 20, 2024 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that it has launched fibroblast growth factor receptor (FGFR) selective tyrosine kinase inhibitor "TASFYGO Tablets 35mg" (generic name: tasurgratinib succinate) in Japan for the treatment of patients with unresectable biliary tract cancer with FGFR2 gene fusions or rearrangements that progressed after cancer chemotherapy (Press release, Eisai, NOV 20, 2024, View Source [SID1234648514]). The product received manufacturing and marketing approval in Japan on September 24, 2024, and was published in Japan’s National Health Insurance Drug Price List today.

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Discovered in-house by Eisai’s Tsukuba Research Laboratories, TASFYGO is an orally available novel tyrosine kinase inhibitor that demonstrates selective inhibitory activity against FGFR1, FGFR2 and FGFR3.

The approval of TASFYGO in Japan is based on data such as the results of a multicenter, open-label, single-arm clinical phase II trial (Study 201) conducted by Eisai in Japan and China.1 A companion diagnostic test to detect FGFR2 gene fusions or rearrangements for the use of TASFYGO in biliary tract cancer, "AmoyDx FGFR2 Break-apart FISH Probe Kit" by Nihon Stery, Inc. (Headquarters: Tokyo) was approved in August 2024.2

The estimated number of patients in Japan with biliary tract cancer is approximately 22,000,3,4 with approximately 25% of the five-year relative survival rate,3 making it an intractable cancer with the second worst prognosis following pancreatic cancer. Since drug therapy options are limited in comparison with other cancers, it is a disease with significant unmet medical needs. FGFR2 gene fusions or rearrangements are observed in approximately 5-14% of intrahepatic cholangiocarcinoma, which accounts for 15-30% of biliary tract cancers.5,6,7 FGFR genetic aberrations such as the gene fusions are known to be deeply involved in the proliferation, survival and migration of cancer cells as well as tumor angiogenesis and drug resistance. As these genetic aberrations in FGFRs have been observed in various other types of cancers as well as biliary tract cancer, there is growing interest in FGFRs as a promising target for cancer therapy.

TASFYGO is produced at the Kawashima Industrial Park (Gifu Prefecture), using innovative Continuous Manufacturing and Real Time Release Testing, a manufacturing technology that ensures product quality within production processes. Continuous Manufacturing is a production method in which processing is carried out continuously from raw material input to formulation. By incorporating real-time quality monitoring technology, multiple manufacturing processes are integrated, enabling automatic production. This method allows for higher quality control compared to conventional processes that focus on product release testing, by utilizing data within the manufacturing process and reducing human-error through automation.

Eisai aims to make continuous efforts to meet the diversified needs of and increase the benefits provided to patients with cancer, their families, and healthcare professionals, by delivering TASFYGO as a new treatment option for biliary tract cancer with FGFR2 gene fusions or rearrangements.

On November 20, 2024 Schrodinger, Inc. (Nasdaq: SDGR) reported that management will participate in a fireside chat at the Piper Sandler 36th Annual Healthcare Conference (Press release, Schrodinger, NOV 20, 2024, View Source [SID1234648533]). The live presentation will take place on Wednesday, December 4, 2024 at 8:30 a.m. ET.

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The live webcast can be accessed in the "Investors" section of Schrödinger’s website and will be archived for approximately 90 days following the event.

On November 19, 2024 Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company using its proprietary Cloudbreak platform to develop drug-Fc conjugate (DFC) immunotherapies designed to save lives and improve the standard of care for patients facing serious diseases, reported that Jeffrey Stein, Ph.D., President and Chief Executive Officer, will participate in the Evercore 7th Annual HealthCONx Conference (Press release, Cidara Therapeutics, NOV 19, 2024, https://www.cidara.com/news/cidara-therapeutics-to-participate-in-evercore-7th-annual-healthconx-conference/ [SID1234648498]).

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Details are as follows:

Event: Evercore 7th Annual HealthCONx Conference
Date: Tuesday, December 3, 2024
Time: 7:55 AM ET
Format: Fireside chat
Webcast: View Source

A replay of the presentation will be available in the Investors section on the Company’s website at www.cidara.com. The replay of the presentation will be available for 90 days.

Cidara will also participate in one-on-one investor meetings during this event. Investors interested in meeting with Cidara at the conference should contact their Evercore representative directly.