On May 20, 2021 Tarveda Therapeutics, Inc., a clinical stage biopharmaceutical company developing a new class of potent and selective precision oncology medicines, which it refers to as Pentarin miniature drug conjugates, reported that the company will present data from its Phase 2 clinical trial of PEN-221 at the 2021 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting occurring virtually June 4-8, 2021 (Press release, Tarveda Therapeutics, MAY 20, 2021, View Source [SID1234580393]).

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PEN-221 is a miniature drug conjugate consisting of a peptide ligand that is highly selective in targeting the somatostatin receptor 2 (SSTR2), joined through a cleavable linker to the potent cytotoxic payload DM1. The Phase 2 trial assessed the safety, tolerability, pharmacokinetics, and preliminary efficacy of PEN-221 in well differentiated neuroendocrine tumors (NETs) and small cell lung cancer. The data being presented include safety and efficacy outcomes for patients enrolled in the gastrointestinal mid-gut NETs cohort.

"We are encouraged by the safety and efficacy results of our Phase 2 trial, which showed that PEN-221 was well tolerated and exceeded its clinical efficacy goals in patients with GI mid-gut neuroendocrine tumors," said Brian Roberts, President and Chief Executive Officer of Tarveda. "We look forward to presenting the results of the study at this year’s ASCO (Free ASCO Whitepaper) Annual meeting and to further evaluating PEN-221 in GI mid-gut neuroendocrine tumors."

Details of the poster presentation are as follows:

Title: The safety and efficacy of PEN-221 somatostatin analog (SSA)-DM1 conjugate in patients (PTS) with advanced GI mid-gut neuroendocrine tumor (NET): Phase 2 results.
Abstract Number: 4110
Date: Poster presentation available on demand beginning on June 4, 2021 at 9:00 AM ET

On May 20, 2021 Everest Medicines (HKEX 1952.HK), a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products in Greater China and other parts of Asia, reported that sacituzumab govitecan-hziy (SG) was granted Priority Review by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) (Press release, Everest Medicines, MAY 20, 2021, View Source [SID1234580409]).

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This marks another progress following the Company’s announcement published on 17 May 2021 that the NMPA accepted its Biologics License Application (BLA) for SG for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease.

"We are very pleased by the NMPA’s decision to grant Priority Review for SG, reinforcing its potential to serve as a novel and important treatment option to people in China suffering the devastating effects of metastatic triple-negative breast cancer," said Yang Shi, Chief Medical Officer for Oncology at Everest Medicines. "We look forward to working closely with local regulatory bodies to bring this innovative treatment to patients in China as quickly as possible."

"This exciting milestone is one of many recent global clinical and regulatory advances that further enables us to accelerate the pace at which we work to bring this first-of-its-kind therapy to patients in need," said Kerry Blanchard, MD, PhD, CEO of Everest Medicines.

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.

Under the trade name Trodelvy, the U.S. Food and Drug Administration (FDA) previously granted accelerated approval to SG in April 2020 and full approval to SG in April 2021 for adult patients with unresectable locally advanced or metastatic triple-negative breast cancer who have received two or more prior systemic therapies, at least one of them for metastatic disease.

About Triple-Negative Breast Cancer

Triple-negative breast cancer (TNBC) is a highly aggressive disease and accounts for approximately 15% of all breast cancer types worldwide. 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 lack sufficient estrogen, progesterone or HER2 receptor expression to benefit from the use of hormonal or HER2-directed therapy. Overall survival among patients with this form of breast cancer has not changed in the past 20 years, which highlights the need for advances in therapeutic options for these patients.

About Sacituzumab Govitecan-hziy

Sacituzumab govitecan-hziy (SG) is a first-in-class, antibody and topoisomerase inhibitor conjugate directed at TROP-2, a protein frequently expressed in multiple types of epithelial cancers. SG is approved in the United States under the trade name Trodelvy.

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.

The Ministry of Food and Drug Safety (MFDS) in South Korea has granted Fast Track Designation and Orphan Drug Designation (ODD) to SG for the treatment of metastatic TNBC. In addition, Everest announced in January 2021 that it submitted a New Drug Application (NDA) to the Health Sciences Authority (HSA) of Singapore for SG for the treatment of patients with metastatic TNBC who have received at least two prior therapies for metastatic disease. That application is currently under review.

On May 20, 2021 ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported an upcoming poster presentation highlighting its chemotherapy free regimen of interleukin-15 (IL-15) superagonist Anktiva (also called N-803) in combination with checkpoint therapy in patients who had relapsed from checkpoint inhibitors, at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place virtually this year from June 4 through June 8, 2021 (Press release, ImmunityBio, MAY 20, 2021, View Source [SID1234580344]).

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The Phase 2 study, titled "Preliminary data from QUILT 3.055: A Phase 2 multi-cohort study of N-803 (IL-15 superagonist) in combination with Checkpoint Inhibitors (CPI)", details data highlighting the safety and clinical benefit of adding Anktiva to checkpoint inhibitor therapy in second-line or greater treatment regimens in multiple cancer types, a basket trial.

Anktiva is designed to activate natural killer cells and CD8+ T cells, without the activation of T-reg cells that can suppress anti-tumor activity. In this Phase 2 study, Anktiva was administered to each patient in combination with a checkpoint inhibitor that had previously yielded a complete response, partial response, or six months of stable disease in that patient in the setting of first-, second- and third-line therapy before disease progression resumed.

"We are encouraged by the trend to date toward Anktiva’s safety, tolerability and clinical benefit that is robust across several types of historically difficult-to-treat cancers, which aligns with Anktiva’s mechanism of action being agnostic with respect to cancer type," said Patrick Soon-Shiong, M.D., Founder and Executive Chairman of ImmunityBio. "We hope to see durable benefit as the study progresses, which would suggest that Anktiva can confer long-term re-sensitization of tumor tissue to checkpoint inhibitor therapy across several cancer types and patterns of patient history."

Human solid tumors are made of multiple clones of tumor cells, some of which harbor genomic alterations that make them invisible to T cells. These resistant clones accomplish this "cloaking ability" by preventing the presentation of the tumor antigens on MHC-I receptors, thus "hiding" from killer T cells. For these patients, maximum activation of T cells with immunotherapy is unlikely to lead to durable tumor control or a cure. However, when NK cells are activated, tumor recognition and targeting is restored. Anktiva activates both NK and T cells and a potential mechanism of rescuing patients from checkpoint relapse is the administration of Anktiva together with the same checkpoint therapy.

"We hypothesize that checkpoint therapy alone is insufficient and that the combination of Anktiva with or without PD-L1 t-haNK may advance the strategy of developing a chemotherapy free immunotherapy protocol for the treatment of multiple tumors. We have previously demonstrated that PD-L1 t-haNK plays an important role in checkpoint failures. The encouraging data from this Phase 2 exploratory trial has formed the basis of our randomized Phase 3 clinical trials in lung cancer (QUILT 2.023, NCT03520686)" said Dr. Soon-Shiong.

Study highlights to date include:

140 patients with checkpoint relapse accrued across multiple tumor types: NSCLC, Small Cell, Urothelial, Head & Neck, Melanoma, Renal, Gastric, and Cervical cancer
121 evaluable patients to date with preliminary data demonstrating 68% (82 out of 121) disease control (partial response and stable disease >6 weeks)
Anktiva exhibits a favorable toxicity profile in combination with several different checkpoint inhibitors in second-line or greater settings, across a variety of tumor types
Adverse events, 12% of which were grade 3 or above, that were related to the chemotherapy-free combination regimen were favorable to the historical standard of care comprising combination therapies that include chemotherapy
Treatment-related serious adverse events (SAEs) were seen in 8% of study participants
Combination regimens that included Anktiva demonstrated clinical benefit in the majority of subjects, with cessation of progression, prolonged stable disease, and occasional partial responses per RECIST were observed in different tumor types

On May 20, 2021 H3 Biomedicine Inc. (H3), a U.S.-based precision medicine research & development subsidiary of Eisai Co., Ltd., reported the presentation of four posters providing updated investigational data on its H3B-6545 clinical program for breast cancer and its H3B-6527 clinical program for hepatocellular carcinoma at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, being held virtually June 4–8, 2021 (Press release, H3 Biomedicine, MAY 20, 2021, View Source [SID1234580361]).

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"We continue to be encouraged by the insights gained through our evaluation of genomically and clinically defined patient populations in these two oncology programs in areas of tremendous unmet medical need," said Antonio Gualberto, MD, PhD, Chief Medical Officer of H3. "We look forward to further highlighting our innovative, data-driven approach to precision oncology research and to showcasing the potential clinical benefit of our product candidates for patients with cancer at ASCO (Free ASCO Whitepaper) 2021."

The investigational clinical data abstracts published online today for the H3B-6545 and H3B-6527 studies reflect data as of March 31, 2021 and Jan 04, 2021, respectively. Updated results from both studies will be presented at ASCO (Free ASCO Whitepaper).

H3’s ASCO (Free ASCO Whitepaper) abstract titles are as follows:

H3B-6545
Abstract Number: 1018
Title: Phase I/II Study of H3B-6545, a Novel Selective Estrogen Receptor Covalent Antagonist (SERCA), in Estrogen Receptor Positive (ER+), Human Epidermal Growth Factor Receptor 2 Negative (HER2-) Advanced Breast Cancer
Poster Discussion Session: Breast Cancer – Metastatic
Presenter: Erika P. Hamilton, Sarah Cannon Research Institute, Tennessee Oncology

Abstract Number: e13025
Title: Phase 1b Study of H3B-6545 in Combination with Palbociclib in Women with Metastatic Estrogen Receptor–positive (ER+), Human Epidermal Growth Factor Receptor 2 (HER2)-negative Breast Cancer
Poster Session: Breast Cancer – Metastatic (Online Publication Only)
Presenter: Stephen R.D. Johnston

Abstract Number: e13022
Title: Relative Bioavailability of H3B-6545 Tablets vs Capsules and Drug-Drug Interaction between H3B-6545 and Pantoprazole
Poster Session: Breast Cancer – Metastatic (Online Publication Only)
Presenter: Alan Xiao

H3B-6527
Abstract Number: 4090
Title: Phase 1 Study of H3B-6527 in Hepatocellular Carcinoma (HCC) or Intrahepatic Cholangiocarcinoma (ICC)
Poster Session: Gastrointestinal Cancer – Gastroesophageal, Pancreatic and Hepatobiliary
Presenter: Teresa Macarulla

ABOUT H3B-6545
Estrogen receptor alpha (ERα) plays an important oncogenic role in the genesis and progression of luminal breast cancers and historically has been a target of endocrine therapies. However, recently, hotspot mutations in ERα have been detected in nearly 30% of endocrine-therapy resistant metastases. Functional studies have shown that these ERα mutations can confer ligand-independent activation of the ERα pathway and can promote partial resistance to existing classes of ER-directed therapies. H3B-6545, a first-in-class small molecule selective estrogen receptor covalent antagonist (SERCA) demonstrates activity in tumor models that harbor wild-type or mutant ERα. H3B-6545 activity against ERα mutants resistant to standard therapy provides an opportunity to target a currently unmet medical need both as a single agent and in combination with other breast cancer therapies.

ABOUT H3B-6527
Receptor tyrosine kinases (RTKs) can be dysregulated in cancer cells and can frequently promote abnormally rapid tumor growth and development. Hepatocellular carcinoma (HCC) can be driven in this way by hyperactivation of the fibroblast growth factor 19 (FGF19)/fibroblast growth factor receptor 4 (FGFR4) pathway. H3B-6527 is a selective, orally bioavailable, and covalent inhibitor of FGFR4 that has demonstrated tumor regression in several preclinical models of HCC. H3B-6527 is being tested specifically in patients with FGFR4-dysregulated advanced HCC.

The abstracts discuss investigational uses of agents in development and are not intended to convey conclusions about efficacy or safety. There is no guarantee that such investigational agents will successfully complete clinical development or gain health authority approval.

On May 20, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of precision genetic medicines, reported the appointment of Sandra Rojas-Caro, M.D., as Chief Medical Officer, effective May 24, 2021 (Press release, NeuBase Therapeutics, MAY 20, 2021, View Source [SID1234580377]). With more than 20 years of drug development experience across all phases of drug development, Dr. Rojas-Caro will oversee the preclinical and clinical development, medical, and regulatory strategy of NeuBase’s pipeline to address disease at its base cause.

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"Sandra is a dynamic and experienced R&D leader, whose expertise covers all aspects of clinical development, including the successful advancement of a novel therapeutic from IND to FDA approval," said Dietrich A. Stephan, Ph.D., Founder, CEO and Chairman of NeuBase. "Furthermore, she’s been responsible for dozens of clinical studies in multiple therapeutic modalities and indications from rare to common diseases. Now is an important time to welcome Sandra to the executive leadership team as we look to advance our first drug candidate into clinical trials next year and expand our pipeline to address historically undruggable oncogenic driver mutations."

"I’m impressed by NeuBase’s unique, targeted therapeutic approach to precision genetic medicines, which is designed to fundamentally address disease-causing mutations at the DNA or RNA level," said Dr. Rojas-Caro. "I am excited to join NeuBase’s exceptional leadership and scientific team, which is comprised of a number of founders and leading innovators in precision medicine. I look forward to applying NeuBase’s PATrOLTM platform, which has broad potential to address a wide range of rare and common genetically defined diseases."

Dr. Rojas-Caro has broad R&D leadership, executive management and team-building experience in private and public biotech companies and large pharma. She has been directly involved in successful global regulatory submissions, including an FDA and EMA approval and more than 10 investigational new drug (IND) applications. Most recently, she was Chief Medical Officer for Gemini Therapeutics, a company focused on redefining age-related macular degeneration (AMD) and linked disorders with precision medicine. At Gemini, she led development through several milestones, including the company’s first IND and the first cohorts of genetically selected patients dosed with the company’s leading biologic therapeutic. Prior to Gemini, Sandra served as Chief Medical Officer for Aeglea BioTherapeutics, a biotechnology company developing a new generation of enzyme-based therapeutics to treat inborn errors of metabolism (IEM). Prior to Aeglea, she served as Group Vice President of Clinical Research and Development at Synageva BioPharma where she was instrumental in leading the clinical development team that secured the FDA and EMA approval of Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency, as well as advancing the clinical development of other IEM programs. Following the acquisition of Synageva by Alexion, Dr. Rojas-Caro served as Vice President and R&D Project Team Leader for the Metabolic Rare Diseases Unit, and she supported the post-merger integration. Earlier in her career, she held roles in clinical and translational research with increasing levels of responsibility at Roche, Array BioPharma and Pfizer, where she was responsible for the design and implementation of early development clinical strategy across a broad range of indications.