On December 9, 2021 Ambrx Biopharma Inc., or Ambrx, (NYSE: AMAM), a clinical stage biopharmaceutical company using an expanded genetic code technology platform to discover and develop Engineered Precision Biologics (EPBs), reported that NovoCodex Pharmaceuticals Ltd. (NovoCodex), Ambrx’s partner in China, presented positive safety and efficacy data from its ongoing ACE-Breast-01 Phase 1 clinical study of ARX788 at the San Antonio Breast Cancer Symposium (SABCS) (Press release, Ambrx, DEC 9, 2021, View Source [SID1234596704]).

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ACE-Breast-01 is a Phase 1 clinical study of ARX788 in HER2-positive metastatic breast cancer patients whose disease is resistant/refractory to HER2 targeted agents including trastuzumab, ADCs (antibody drug conjugates), TKIs (tyrosine kinase inhibitors) and bispecific antibodies. The updated data, presented during a spotlight poster session (PD8-04), demonstrates ARX788’s robust anti-tumor activity.

Study Highlights

ARX788 at 1.5 mg/kg Q3W demonstrated robust treatment effect as illustrated by objective response rate (ORR) in 29 patients in all prior anti-HER2 treatments groups:
Prior Anti-HER2 Therapy

Confirmed ORR

Trastuzumab containing regimens*

19/29 (66%)

HER2 ADC regimens (T-DM1, DX126-262, A166, BAT8001, HS630)**

4/5 (80%)

HER2 TKI regimens (lapatinib, pyrotinib, neratinib, AST-1306, Hemay-022)

15/23 (65%)

Both HER2 ADC and HER2 TKI regimens

3/4 (75%)

Bispecific antibody containing regimens (KN026 and M802)

3/4 (75%)

Table represents subsets of a total of 29 evaluable patients in the 1.5 mg/kg Q3W cohort, treatment groups are not mutually exclusive

* All patients at 1.5 mg/kg Q3W received prior trastuzumab-containing regimens

** One patient who received prior pertuzumab also achieved confirmed partial response (PR)

The disease control rate among evaluable patients in the 1.5 mg/kg cohort was 100% (29/29)
ARX788 demonstrated low systemic toxicity and was generally well tolerated with most adverse events being grade 1 or 2
No dose limiting toxicity or drug-related deaths occurred
"As the ACE-Breast-01 data for ARX788 continues to develop, we are encouraged by the ADC’s anti-tumor activity and safety profile. ARX788’s ability to continually deliver anti-cancer activity in patients with prior anti-HER2 therapies including ADCs, TKIs and bispecifics, truly highlights the potential of our drug candidate," said Feng Tian, Ph.D., Chairman of the Board, President and CEO of Ambrx. "I look forward to working with NovoCodex to realize the full potential of ARX788."

The Phase 1 clinical study being conducted by our partner, NovoCodex, is a dose escalation study designed to evaluate the safety and anti-tumor activity of ARX788 administered every three weeks in heavily pretreated patients with HER2-positive metastatic breast cancer. The 29 evaluable patients in the 1.5 mg/kg Q3W dose cohort who participated in the study were heavily pretreated with, and had failed, a median of seven prior lines of therapy (median of six for the 69 patients in all cohorts) in the advanced disease setting.

Additionally, Ambrx presented a poster on ACE-Breast-03 in a SABCS ongoing trial poster session (OT1-02-02). The poster details ARX788 in its ongoing global ACE-Breast-03 Phase 2 clinical study in patients with HER2-positive metastatic breast cancer whose disease is resistant or refractory to T-DM1, and/or T-DXd, and/or tucatinib-containing regimens. The study is currently enrolling up to 200 patients and will assess the efficacy, safety and pharmacokinetics of ARX788, with confirmed objective response rate as the primary endpoint.

Dr. Tian continued, "Ambrx’s proprietary EuCODE technology enables ARX788 to maximize the delivery efficiency of the therapeutic’s cytotoxic payload AS269 into HER2-expressing tumor cells. I am looking forward to our on-going global ACE-Breast-03 phase 2 study designed for patients with HER2-positive metastatic breast cancer whose disease is resistant or refractory to T-DM1, T-DXd, and tucatinib-containing regimens."

On December 9, 2021 TYME Technologies, Inc. (Nasdaq: TYME) (the Company or TYME), an emerging biotechnology company developing cancer metabolism-based therapies (CMBTs), reported that its abstract, Oral SM-88 plus MPS, an effective yet less toxic treatment option in second-line advanced pancreatic cancer? Final phase II/III study results, has been accepted for poster presentation at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium, January 20 – 22, 2022, in San Francisco, CA (Press release, TYME, DEC 9, 2021, View Source [SID1234596703]).

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"We are delighted that our abstract was chosen from among the more than 750 submissions that were reviewed by the Symposium Program Committee, and we look forward to sharing more information about this important work at the conference," stated Richie Cunningham, Chief Executive Officer of TYME.

Additional information on the meeting can be found on the ASCO (Free ASCO Whitepaper) 2022 Gastrointestinal Cancers Symposium website View Source

Poster Presentation Details:

Abstract Number: 585

Abstract Title: Oral SM-88 plus MPS, an effective yet less toxic treatment option in second-line advanced pancreatic cancer? Final phase II/III study results.

Session Information: Poster Session B: Pancreas, Small Bowel, and Hepatobiliary Tract

Date/Time: Friday, January 21, 2022, 3:05 – 4:35 PM EST

On December 9, 2021 Agendia, Inc., a world leader in precision oncology for breast cancer, reported that data from a research collaboration with Vanderbilt University Medical Center demonstrating the capability of MammaPrint and BluePrint to identify differences in recurrence risk and tumor classification amongst different racial groups at the 2021 San Antonio Breast Cancer Symposium (SABCS 2021) (Press release, Agendia, DEC 9, 2021, View Source;Breast-Cancers-in-Black-and-White-Women-Beyond-Clinical-Factors [SID1234596702]).

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The presentation, titled MammaPrint and BluePrint identify genomic differences in HR+ HER2- breast cancers from young Black and White women, highlights new data signaling that among young women (­<50 years of age) with localized hormone receptor positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer, MammaPrint and BluePrint identified racial disparities in risk of recurrence and breast cancer subtype distribution not detected by existing clinical factors.

The analysis included 186 Black women and 186 White women under the age of 50 with stage I-III HR+ HER2- breast cancer, matched by age, tumor stage, nodal status and receptor status. Of these patients, Black women were significantly more likely to present as a MammaPrint High Risk 2, and in general had more High Risk tumors compared to White women – who were more likely to have Low Risk tumors, including more MammaPrint UltraLow Risk tumors – despite matching for clinical and pathologic features.

Additionally, the data demonstrated that estrogen-receptor-positive (ER+) tumors reclassified by BluePrint as Basal-type occur at a higher frequency in Black women compared to White women. Basal-type tumors often have worse outcomes compared to ER+, so it is essential to identify this reclassification in Black women to understand the tumor’s biology and ultimately provide better, personalized care. These data underscore the importance of MammaPrint Risk assessment and BluePrint subtyping for providing greater precision in the prediction of risk of recurrence and the selection of therapy.

"It is crucial to identify the genomic differences among young women with breast cancer that may be contributing to racial/ethnic survival disparities, especially given that Black women are more than 40% more likely to die from breast cancer compared to White women," said Sonya Reid, MD, MPH, Department of Medicine, Vanderbilt University Medical Center. "These data demonstrate the promise of personalized medicine to advance our understanding of racial differences on a genomic level to improve breast cancer outcomes across all racial and ethnic groups."

Agendia previously presented data at ASCO (Free ASCO Whitepaper) 2021 which also highlighted the importance of genomic insights that are representative of and effective for diverse patient groups, to better understand and begin to solve for disparities in outcomes.

"Black women continue to remain underrepresented in clinical trials, and this study signifies an important step in measuring the clinical and genomic differences of breast cancers in Black women, which will help to redefine the standard of care and guide treatment planning," said William Audeh, MD, Chief Medical Officer of Agendia and a study author. "These results provide novel insights about differences in tumor biology; MammaPrint and BluePrint have tremendous potential to add to our understanding of the causes of disparities in survival associated with race, and will help guide therapeutic strategies to improve outcomes. We greatly appreciate the opportunity to collaborate with Dr. Reid and her colleagues at Vanderbilt in this important research."

Agendia is dedicated to gathering data that has translational potential as well as immediate clinical impact for diverse patient populations. Providing novel insights about tumor biological differences and adding to the body of evidence of genomic profiling of breast cancer based on genetic ancestry is critical to improving patient outcomes. These data underscore the company’s mission to help guide the diagnosis and personalized treatment of breast cancer for all patients throughout their treatment journey.

On December 9, 2021 Outcomes4Me Inc., a developer of a leading free mobile app and platform to navigate cancer treatment and care, reported, at the San Antonio Breast Cancer Symposium, findings from a study done in collaboration with the Mass General Cancer Center, Boston (Press release, Outcomes4Me, DEC 9, 2021, View Source [SID1234596700]). The pilot study analyzed the concordance of patient reported disease characteristics compared to electronic medical record (EMR) data. Outcomes4Me found that the data clearly points to the need for patients to have a better understanding of their breast cancer diagnosis. Patients’ understanding of their breast cancer diagnosis is important in order to improve treatment adherence, shared decision-making, and clinical trial matching. The study shows important discrepancies between EMR and patient self-reported information, shining a light on the need for better patient education and medical records access.

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The study, which collected data via a survey and an in-app questionnaire between June 2020 to December 2020, found that 97 percent of the study cohort reported that they "somewhat" or "strongly" understood their cancer diagnosis. Yet, 24 percent of patients answered "I don’t know" or "I’m not sure" to fundamental questions about their disease characteristics including hormone receptor (HR) status, and/or HER2 status.

"There is no doubt that the inaccuracies found in self-reporting suggest a need for improved patient education regarding key cancer characteristics and a need for caution when relying on self-reported characteristics for clinical trials matching and targeted patient education," said Maya R. Said, Sc. D., founder and CEO of Outcomes4Me. "These findings underscore a problem that all stakeholders here at the SABCS – practicing providers and researchers – involved in breast cancer need to have top of mind. The burden of improving patients’ understanding of breast cancer should not be on the patient alone."

The study also found that HR and HER2 status had limited concordance with the EMR, in contrast to a high degree of accuracy in self-reported metastatic status. Receptor status is critically important to understanding breast cancer and what treatment options are available, including clinical trials. Yet, only 55 to 61 percent of patients accurately self-reported their HR and/or HER2 status. In particular, HER2 status was only reported accurately among 64 to 70 percent of the cohort, compared to 79 to 89 percent of patients accurately reporting their metastatic status.

"Patient education is critical to shared decision making," said Steven Isakoff, MD, PhD, Breast Oncologist at Mass General Cancer Center and lead author on the study. "Patients need to have a better understanding of their breast cancer diagnosis in order to have more informed decision-making conversations. Without accurately knowing critical disease characteristics, our patients are not in the best position to make informed decisions with their oncologist."

These results further highlight the need for digital platforms to integrate self-reported characteristics with EMR access to help provide accurate patient information to address these critical needs. The Outcomes4Me platform was developed to enable this integration with a goal to impact patient empowerment and care. The Outcomes4Me app is available free to users on both the App Store and Google Play.

More about the Outcomes4Me Inc. study, done in collaboration with the Mass General Cancer Center, Boston:
Data was analyzed from a single institution pilot study (NCT04262518) and eligibility included breast cancer patients with any subtype or stage of invasive cancer presenting with a new diagnosis or for follow-up on active therapy. Outcomes4Me compared patient reported characteristics within a study specific survey and/or the Outcomes4Me app for stage (metastatic or not metastatic), recurrence history, hormone receptor status, HER2, and surgery history with the data recorded in the EMR. All statistics were descriptive. The company conducted the same comparison between patient reported clinical characteristics among real world users of the Outcomes4Me app and EMR records downloaded by that cohort of patients.

Access to the SABCS poster can be found on sabcs.org.

On December 9, 2021 CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, reported that it signed the collaboration on a rare disease research agreement with the Peking Union Medical College Hospital (PUMCH), on December 9, 2021 (Press release, CANbridge Life Sciences, DEC 9, 2021, View Source [SID1234596699]). Under terms of the agreement, the two parties will collaborate on research and development in rare disease drug innovation, translational medicine and clinical verification, leveraging their respective strengths to further improve the development of innovative rare disease drugs and drive medical research and the development of industrial systems for diagnosis and treatment.

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"We are delighted and honored to enter into the collaboration agreement with PUMCH, the only national lead organization of the National Collaborative Network for the Diagnosis and Treatment of Rare Diseases with extensive experience in the diagnosis, treatment and clinical research of rare diseases," said James Xue, Ph.D., CANbridge Founder, Chairman and CEO. "Working together, I believe we’ll be able to find a characteristically Chinese path for the development and innovation of rare disease treatments and to provide hope for patients with rare diseases and their families."

About the Peking Union Medical College Hospital (PUMCH)

Founded in 1921, the Peking Union Medical College Hospital (PUMCH) is a modern AAA hospital. In addition to providing medical services, it is also a teaching and research hospital. PUMCH is a national center designated by the National Health Commission (NHC) for guiding the diagnosis and treatment of intractable diseases and critical illness. It is also one of the first hospitals designated to provide healthcare services for government officials and foreigners, one of the national demonstration centers for higher medical education and the standard training of resident doctors, and one of the key national centers for clinical medical research and innovation. The hospital is known both in China and abroad for its full range of services strong human resources, prominent specialties, and overall strength. PUMCH was ranked No.1 twice during the nationwide performance evaluation of AAA public hospitals and topped the "Ranking of Chinese Hospitals," by Fudan University Institute of Hospital Management, for 12 consecutive years.

PUMCH is a leader in rare diseases. In 2016, PUMCH led the national key research program, the Clinical Cohort Study of Intractable and Rare Diseases, and built China’s National Registration System for Intractable and Rare Diseases, which became one of the largest platforms for information about rare diseases in the world. Within the framework of the NHC’s Expert Committee on the Clinical Care and Accessibility for Rare Diseases, PUMCH led top Chinese experts to draft China’s First List of Rare Diseases and compiled and published Interpretation of China’s First List of Rare Diseases, Guidelines for the Diagnosis and Treatment of Rare Diseases (the 2019 edition) (released by the NHC), and Rare Diseases, a textbook for graduate students during the 13th Five-Year Plan period. The hospital has also been driving technical training on the diagnosis and treatment of rare diseases across the country. Currently, PUMCH is undertaking research projects of the State Key Laboratory of Rare Diseases, the National Center for Translational Medicine, the National Center of the National Collaborative Network for the Diagnosis and Treatment of Rare Diseases, and the National Center for the Quality Control of Rare Diseases. The hospital is building a national center for research and innovation with respect to the diagnosis and treatment of rare diseases with all the necessary clinical resources and expertise.