Everest Medicines Submits New Drug Application in Hong Kong for Sacituzumab Govitecan in Second-Line Metastatic Triple-Negative Breast Cancer

On March 30, 2022 Everest Medicines (HKEX 1952.HK), a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products to address critical unmet needs in Asia Pacific markets, reported that it has submitted a New Drug Application (NDA) to the Department of Health, the Hong Kong Special Administrative Region, China for sacituzumab govitecan (SG) for the treatment of unresectable locally advanced or metastatic triple-negative breast cancer (mTNBC) in adult patients who have received two or more prior systemic therapies, at least one of them for metastatic disease (Press release, Everest Medicines, MAR 30, 2022, View Source [SID1234611209]).

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"This submission reinforces the ongoing momentum in the development of SG throughout Asia, bringing this medicine one step closer to patients in yet another important region in Asia, and adding to the growing number of regulatory applications under active review," said Yang Shi, Chief Medical Officer for Oncology at Everest Medicines. "As women diagnosed with mTNBC have historically had minimal treatment options available to them and breast cancer has become the most common cause of death in women in Hong Kong over the past few decades, this application is especially timely. We look forward to moving closer to our goal of making SG an accessible treatment option for women throughout Greater China."

Everest is also closely coordinating with regulatory bodies in mainland China, South Korea and Taiwan to review its applications for SG for adult patients with unresectable locally advanced or mTNBC who have received two or more prior systemic therapies, at least one of them for metastatic disease. In February 2022, Everest announced the approval of Trodelvy (trade name for SG) in Singapore for second-line mTNBC.

In November 2021, Everest announced topline results for its Phase 2b EVER-132-001 study of SG, which met its primary endpoint with a 38.8% overall response rate (ORR). This study evaluated 80 people in China, and the results were consistent with those from the global Phase 3 ASCENT study, thus showing similar efficacy in the Chinese population.

About Triple-Negative Breast Cancer (TNBC)

TNBC is the most aggressive type of breast cancer and accounts for approximately 15% of all breast cancers. The median age of breast cancer diagnoses tends to be younger in Asian than western countries, and the percentage of the TNBC molecular subtype has been increasing in the past 10 years. TNBC cells do not have estrogen and progesterone receptors and have limited human epidermal growth factor receptor 2 (HER2). Due to the nature of TNBC, effective treatment options are extremely limited compared with other breast cancer types. TNBC has a higher chance of recurrence and metastases than other breast cancer types. The average time to metastatic recurrence for TNBC is approximately 2.6 years compared with 5 years for other breast cancers, and the relative five-year survival rate is much lower. Among women with metastatic TNBC, the five-year survival rate is 12%, compared with 28% for those with other types of metastatic breast cancer.

About Sacituzumab Govitecan (SG)

Sacituzumab govitecan (SG) is a first-in-class Trop-2 directed antibody-drug conjugate. Trop-2 is a cell surface antigen highly expressed in multiple tumor types, including in more than 90% of breast and bladder cancers. SG is intentionally designed with a proprietary hydrolyzable linker attached to SN-38, a topoisomerase I inhibitor payload. This unique combination delivers potent activity to both Trop-2 expressing cells and the microenvironment.

SG is approved in more than 35 countries, with additional regulatory reviews underway worldwide, for the treatment of adult patients with unresectable locally advanced or metastatic TNBC, under the trade name Trodelvy. SG is also approved in the U.S. under the accelerated approval pathway for the treatment of adult patients with locally advanced or metastatic urothelial cancer (UC) who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor.

SG is also being developed for potential investigational use in other TNBC and metastatic UC populations, as well as a range of tumor types where Trop-2 is highly expressed, including hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) metastatic breast cancer, metastatic non-small cell lung cancer (NSCLC), metastatic small cell lung cancer (SCLC), head and neck cancer, and endometrial cancer.

Under a licensing agreement with Gilead Sciences, Inc., Everest Medicines has exclusive rights to develop, register, and commercialize SG for all cancer indications in Greater China, South Korea, and certain Southeast Asian countries. In October 2020, SG was included in the updated 2020 China Guidelines for the Standardized Diagnosis and Treatment of Advanced Breast Cancer, compiled by the Breast Cancer Expert Committee of the National Cancer Control Center, the Breast Cancer Professional Committee of the Chinese Anti-Cancer Association, and the Cancer Drug Clinical Research Professional Committee of the Chinese Anti-Cancer Association.

CStone announces first patient enrollment in the U.S. in the Phase 1 clinical trial of CS5001, a potential global best-in-class ROR1-targeting ADC

On March 30, 2022 CStone Pharmaceuticals ("CStone", HKEX: 2616), a leading biopharmaceutical company focused on the research, development, and commercialization of innovative immuno-oncology therapies and precision medicines, reported that the first patient has been enrolled in the U.S. in the Phase 1 clinical trial for CS5001 (Press release, CStone Pharmaceauticals, MAR 30, 2022, View Source [SID1234611208]). This is a remarkable milestone for CStone’s Pipeline 2.0 strategy.

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CS5001 is a potential global best-in-class antibody-drug conjugate (ADC), targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1). As one of the three most advanced ROR1 ADCs worldwide, CS5001 has been approved for the initiation of a multi-regional clinical trial in the US and Australia. The China National Medical Products Administration (NMPA) has accepted the investigational new drug (IND) application of CS 5001. This first-in-human Phase 1 study aims to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of CS5001 in advanced B cell lymphomas and solid tumors.

ROR1 is an oncofetal protein with low or no expression in adult tissues but high expression in a variety of cancers including various forms of leukemia and non-Hodgkin lymphoma, breast, lung, and ovarian cancers, making it an ideal ADC target. Results from pre-clinical studies showed that CS5001 exhibited potent and selective cytotoxicity to a variety of ROR1-expressing cancer cell lines and demonstrated remarkable in vivo antitumor activity in both hematological and solid tumor xenograft models. The preclinical data were presented as a late-breaking abstract at the 33rd AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) 2021.

Dr. Archie Tse, Chief Scientific Officer of CStone, said: "We are very glad to have the first patient enrolled in the first-in-human study of CS5001. This potentially best-in-class ROR1 ADC contains a number of differentiated features which may translate into a wider therapeutic window against a variety of cancer types — a fully human antibody backbone, proprietary site-specific conjugation, and tumor-cleavable linker and prodrug technology. Results from the preclinical studies of CS5001 already showed its therapeutic potential in ROR1-expressing hematological and solid malignancies. We will swiftly execute the global development program of CS5001, starting with this Phase 1 study to characterize its safety and preliminary efficacy in the treatment of advanced B-cell lymphoma and selected solid tumors."

About CS5001(ROR1 ADC)

CS5001 is a clinical-stage antibody-drug conjugate (ADC) targeting ROR1 (receptor tyrosine kinase-like orphan receptor 1). CS5001 has uniquely designed and LCB’s proprietary tumor-cleavable linker and pyrrolobenzodiazepine (PBD) prodrug. Only after reaching the tumor, the linker and prodrug are cleaved to release the PBD toxin, resulting in lethal DNA cross-links in cancer cells. The use of the linker plus PBD prodrug effectively helps addressing the toxicity problem associated with traditional PBD payloads, leading to a better safety profile. Additionally, CS5001 utilizes site-specific conjugation for a precise drug antibody ratio of 2 which enables homogeneous production and large-scale manufacturing.

In October 2020, CStone signed a licensing agreement with LegoChem Biosciences, Inc. (LCB) for the development and commercialization of CS5001 which was originally generated by collaboration of LCB and ABL Bio, both South Korea-based leading biotech companies. Under the agreement, CStone obtains the exclusive global right to lead development and commercialization of CS5001 outside the Republic of Korea.

Lucid Diagnostics to Participate in the Needham 21st Annual Healthcare Conference

On March 30, 2022 Lucid Diagnostics Inc. (Nasdaq: LUCD) ("Lucid"), a commercial-stage, cancer prevention medical diagnostics company, and majority-owned subsidiary of PAVmed Inc. (Nasdaq: PAVM, PAVMZ) ("PAVmed"), reported that Dr. Lishan Aklog, Chairman and CEO, has been invited to present at the 21st Annual Needham Virtual Healthcare Conference taking place from April 11 – 14 (Press release, Lucid Diagnostics, MAR 30, 2022, View Source [SID1234611207]). Dr. Aklog will participate in a Fireside chat on Tuesday, April 12, from 1:30 – 2:10 pm ET. Register to listen to his presentation at View Source In addition, Lucid will host 1×1 investor meetings during the conference. Please contact your Needham representative to schedule a 1×1 meeting.

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RenovoRx Reports Full Year 2021 Financial Results

On March 30, 2022 RenovoRx, Inc. ("RenovoRx" or the "Company") (Nasdaq: RNXT), a biopharmaceutical company and innovator in targeted cancer therapy, reported its financial results for the year ended December 31, 2021 (Press release, Renovorx, MAR 30, 2022, View Source [SID1234611206]).

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"2021 was a transformative year for RenovoRx. We successfully closed on our initial public offering and listed on the NASDAQ under ‘RNXT,’" said Shaun Bagai, CEO of RenovoRx. "Importantly, despite challenges created by the COVID-19 pandemic, we maintained steady enrollment in our Phase 3 clinical trial in locally advanced pancreatic cancer. We recently achieved approximately 50% of the target enrollment under the current statistical analysis plan. One of the issues with systemic chemotherapy is the significant side effects to the entire body. RenovoRx is addressing this problem by localizing therapy to treat tumors via our proprietary RenovoTAMP (RenovoRx Trans-Arterial Micro-Perfusion) therapeutic platform. Today, we have the strategy and team to build on our progress in treating pancreatic cancer with the opportunity to extend the RenovoTAMP platform to other cancers. We are challenging the bounds typically associated with treating solid tumors to improve survival and quality of life for patients undergoing chemotherapy."

2021 Operational Highlights:

Issuance of seventh U.S. patent extending the intellectual property coverage of the RenovoTAMP therapy platform.
Closed on IPO and listed on the Nasdaq Capital Market under the ticker symbol, "RNXT."
Received new 510(k) clearance for the RenovoCath delivery system (the device component of the Company’s initial product, RenovoGem). RenovoRx received its initial 510(k) for the RenovoCath delivery system in 2014.
Announced final data from RR2 observational registry study. Phase 1/2 trials demonstrated that prior radiation treatment, together with targeted chemotherapy delivered via RenovoTAMP, reduced the tolerability issues typically associated with systemic chemotherapy and improved survival.
2022 Operational Highlights and Upcoming Targeted Milestones:

Began enrolling patients at Columbia University’s New York-Presbyterian Hospital Irving Medical Center in ongoing TIGeR-PaC Phase 3 Clinical Trial.
TIGeR-PaC achieved approximately 50 percent of the target enrollment under the current statistical analysis plan.
Plans to meet with the FDA to discuss trial design for a second indication, extrahepatic (or outside the liver) cholangiocarcinoma (bile duct cancer), or eCCA. Pending the outcome of that meeting and protocol submission, Phase 2 trial in eCCA may be launched in 2H 2022.
Expects to conduct TIGeR-PaC interim analysis.
Financial Highlights for the Fiscal Year Ended December 31, 2021

Cash and cash equivalents as of December 31, 2021, were $15.2 million.
Research and development expenses were $3.0 million for the year ended December 31, 2021, compared to $2.4 million for the year ended December 31, 2020. The increase was primarily due to higher clinical development employee-related costs.
General and administrative expenses were $2.6 million for the year ended December 31, 2021, compared to $0.8 million for the year ended December 31, 2020. The increase was primarily due to higher professional and consulting expenses related to preparing for our IPO in August 2021, including employee-related costs and insurance costs for Directors and Officers Liability Insurance.
Net loss was $6.3 million for the ended December 31, 2021, compared to net loss of $3.8 million for year ended December 31, 2020.
As of March 25, 2022 the Company had 9,029,305 common shares outstanding.
About the Phase 3 TIGeR-PaC Clinical Trial

TIGeR-PaC is a randomized multi-center Phase 3 study using RenovoRx’s innovative therapy platform, RenovoTAMP (RenovoRx Trans-arterial Micro-perfusion). The study is evaluating the Company’s first product candidate, RenovoGem, to treat locally advanced pancreatic cancer (LAPC) through the intra-arterial delivery of gemcitabine (an FDA-approved chemotherapy). The study has a primary endpoint of overall survival and several secondary endpoints, including quality of life.

TIGeR-PaC is currently enrolling unresectable LAPC patients at several sites across the U.S. To learn more about the study and the participating clinical trial sites, visit View Source

Accutar Biotechnology Announces First Patient Dosed with AC0176 in Phase 1 Study in Patients with Metastatic Castration-Resistant Prostate Cancer (mCRPC)

On March 30, 2022 Accutar Biotechnology, Inc., a clinical stage biotechnology company focusing on artificial intelligence (AI)-enabled drug discovery, reported the dosing of the first patient in a Phase 1 study of AC0176, an orally bioavailable, chimeric degrader molecule designed to target the androgen receptor (AR) with high potency and selectivity (Press release, Accutar Biotechnology, MAR 30, 2022, View Source [SID1234611205]).

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"The initiation of this study marks the second program from our AI-enabled drug discovery platform and our chimeric degrader portfolio to enter the clinic," said Jie Fan, Ph.D., Chief Executive Officer of Accutar Biotechnology, Inc. "AC0176 was designed to potently degrade both AR wildtype and prevalent AR mutants which confer drug resistance to current AR-targeted therapies, including but not limited to L702H, T878A, H875Y, W742C. We are excited about the differentiated therapeutic profile of AC0176 and its broad potential to treat prostate cancer patients."

The purpose of the Phase 1 multi-center, open-label study is to assess the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of AC0176 treatment in patients with mCRPC. Additional information on this clinical trial can be found on www.clinicaltrials.gov (NCT05241613).

About AC0176

AC0176 is an investigational orally bioavailable, chimeric degrader of AR for the potential treatment of prostate cancers. In preclinical studies, AC0176 demonstrated potent and selective AR protein degradation with broad coverage of AR mutants, favorable pharmacological properties, as well as promising anti-tumor activity in animal models.