On August 1, 2022 Gracell Biotechnologies Inc. ("Gracell" or the "Company",NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, reported the appointment of Wendy Li, M.D., as its Chief Medical Officer (CMO) (Press release, Gracell Biotechnologies, AUG 1, 2022, View Source [SID1234617227]). In this role, Dr. Li will oversee Gracell’s clinical development activities, including the advancement of its pipeline of autologous and allogeneic product candidates across the Company’s multiple proprietary technology platforms.

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Dr. Li brings to Gracell over 20 years of experience leading all critical aspects of clinical and medical operations at early-stage and large pharmaceutical organizations in the U.S. and China. Her expertise spans both clinical development and medical affairs, including leading early- and late-stage clinical trials for several therapeutic candidates for the treatment of hematological malignancies and solid tumors, and overseeing over 30 successful Investigational New Drug applications (INDs), New Drug Applications (NDAs) and Biologics License Applications (BLAs) that led to several multi-billion-dollar blockbuster drugs.

"Dr. Li is a distinguished and enthusiastic clinical leader and shares our commitment to discovering and developing breakthrough cell therapies. We are excited to add her experience to Gracell’s global leadership team," said Dr. William (Wei) Cao, Founder, Chairman and CEO of Gracell. "Dr. Li’s decades of experience in leading clinical development and strategy across many types of cancers and treatment modalities, as well as expertise in working with regulatory agencies, will be invaluable as we continue to advance our pipeline of breakthrough cell therapies and approach the filing of our U.S. IND application for GC012F later this year."

Prior to joining Gracell, Dr. Li served as CMO of EXUMA Biotech, where she provided strategic medical and clinical leadership for the advancement of its cell therapy pipeline in the U.S. and Asia. She has also held clinical development and medical affairs leadership positions at Pfizer, Sanofi, Genentech, and Sihuan Pharmaceutical. Dr. Li holds an M.D. from Sun Yat-Sen University of Medical Sciences.

"With years of experience in CAR-T, I recognize this therapy’s potential to fulfill patients’ unmet needs and am eager to join a company with a pipeline as promising as Gracell’s," Dr. Li said. "I believe the company’s FasTCAR and TruUCAR platforms have vast potential to solve some of the greatest challenges facing CAR-T therapy. The lead candidate, GC012F, is a highly differentiated CAR-T therapy that potentially affords competitive efficacy, combined with a favorable safety profile and faster delivery to patients. I look forward to joining Gracell’s leadership team and bringing the company to its next stage of growth."

On August 1, 2022 Biosight Ltd., a pharmaceutical development company focused on the development of innovative therapeutics for hematological malignancies and disorder, reported that the United States Food & Drug Administration (FDA) has granted Orphan Drug Designation to aspacytarabine (BST-236), an investigational novel antimetabolite, for the treatment of myelodysplastic syndromes, in addition to the Orphan Drug Designation granted in 2019 for aspacytarabine for the treatment of acute myeloid leukemia (AML) (Press release, BioSight, AUG 1, 2022, View Source [SID1234617226]).

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Following the encouraging single-agent efficacy and safety profile of aspacytarabine in the recently completed Phase 2b trial in front-line treatment of AML patients unfit for standard induction chemotherapy, aspacytarabine development program is being expanded to second-line treatment of AML and MDS in two ongoing Phase 2 clinical trials, one in the US and Israel and one in France in collaboration with the European cooperative group, GFM. In addition to the monotherapy development programs, a Phase 1/2 trial of aspacytarabine in combination with the Bcl2 inhibitor, venetoclax, for the treatment of AML patients unfit for standard chemotherapy will be launched in the coming weeks.

"We are very pleased to have received from the FDA the Orphan Drug Designation for aspacytarabine for the treatment of MDS, which adds to the designation granted already for the treatment of AML" said Dr. Ruth Ben Yakar, CEO of Biosight. "The accumulating clinical data from more than a hundred patients treated to date with aspacytarabine monotherapy in four clinical trials, suggest that aspacytarabine, with a differentiated mechanism that enables high-dose chemotherapy with reduced toxicity, has the potential to transform standard of care for AML and MDS patients".

Orphan Drug Designation by the FDA entitles Biosight to seven years of market exclusivity for the use of aspacytarabine for the treatment of MDS, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.

About Aspacytarabine (BST-236)

Aspacytarabine is a novel proprietary anti-metabolite. It is composed of cytarabine covalently bound to asparagine, acting as a pro-drug of cytarabine. Cytarabine serves as the backbone of AML and MDS therapy for over 45 years due to its superior efficacy, however, it is associated with severe bone marrow, gastrointestinal, and neurological toxicities, which significantly limit its use, especially in older and medically compromised patients. Due to its unique pharmacokinetics and metabolism, aspacytarabine enables high-dose therapy with lower systemic exposure to free cytarabine and relative sparing of normal tissues. As such, aspacytarabine may serve as a superior therapy for AML, MDS, and other hematological malignancies and disorders, including for older adults who are unfit for intensive therapy.

Aspacytarabine was granted FDA Fast Track Designation for the treatment of AML in adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy, and Orphan Drug designations from the FDA and EMA, which entitles Biosight to seven and ten years of market exclusivity upon aspacytarabine marketing approval for the treatment of AML in the US and Europe, respectively.

Results from the recently completed Phase 2b study evaluating aspacytarabine as a single-agent first-line AML therapy demonstrate safety and single-agent activity, and additional studies are ongoing to evaluate aspacytarabine as a second line treatment for patients with relapsed or refractory MDS or AML. For more information regarding the Phase 2b clinical study of aspacytarabine, please visit www.clinicaltrials.gov.

On August 1, 2022 Tavanta Therapeutics, a clinical-stage specialty pharmaceutical company developing a diverse pipeline of specialty drugs that bring clinically meaningful benefits to patients with serious or debilitating diseases, reported the completion of patient enrollment in the Company’s pivotal Phase 3 study for TAVT-45 (abiraterone acetate) Granules for Oral Suspension ("TAVT-45") (Press release, Tavanta Therapeutics, AUG 1, 2022, View Source [SID1234617225]). TAVT45CO2 is a global Phase 3 clinical trial evaluating TAVT-45 in patients with metastatic castrate-sensitive prostate cancer (mCSPC) and metastatic castrate-resistant prostate cancer (mCRPC). The primary objective of the study is to establish therapeutic equivalence between TAVT-45 and Zytiga tablets.

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TAVT-45 is designed to provide an easy-to-consume oral suspension of abiraterone acetate, reconstituted in water or juice, for patients with difficulty taking large tablets. Approximately 20 to 30 percent of cancer patients have difficulty swallowing pills and capsules (dysphagia) and may benefit from an alternate formulation. The prevalence of dysphagia also increases with age and is an issue for many patients with prostate cancer, whom have a median age of over 65 years old at time of diagnosis.

"The current standard of care treatment for metastatic prostate cancer requires patients to ingest multiple large tablets, and under strict fasting conditions due to the risk of increased and highly variable abiraterone exposures with food," said Kenneth M. Kernen, M.D., study investigator and partner in the Michigan Institute of Urology. "Dysphagia is an issue for many – and it occurs more frequently in elderly patients. If TAVT-45 proves successful in this trial, clinicians may soon have access to an alternative, easy-to-drink formulation of abiraterone acetate with improved bioavailability and reduced systemic variability, which may ultimately help patients achieve better clinical outcomes."

This Phase 3 registrational trial (NCT04887506) is a global, randomized, multi-center, open-label trial designed to evaluate the pharmacodynamic effect and safety profile of TAVT-45 Granules compared to Zytiga tablets, in combination with prednisone, in patients with mCSPC and mCRPC. In addition to establishing therapeutic equivalence between TAVT-45 Granules and Zytiga tablets, the study aims to characterize the multiple-dose pharmacokinetic profile of TAVT-45 Granules. Topline results for the trial are expected by the end of this year.

"On behalf of Tavanta, we would like to thank the clinical study site investigators and the patients who are participating in and supporting this pivotal trial, especially in light of the ongoing challenges of the COVID-19 pandemic. We look forward to completing the dosing and follow-up phases of the study," said Lynne Powell, chief executive officer of Tavanta Therapeutics. "As we work to complete this registrational trial for TAVT-45, we will begin preparing for the submission of our New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the third quarter of 2023, while also evaluating strategic options for commercialization of TAVT-45."

About TAVT-45 Granules
TAVT-45 Granules is an enhanced formulation of abiraterone acetate for the treatment of metastatic prostate cancer. When reconstituted with water or juice to yield an oral suspension, TAVT-45 Granules may provide an alternative for the 20 to 30 percent of patients who suffer from dysphagia or have difficulty swallowing tablets and may increase the bioavailability of abiraterone and therefore allow a lower dose to be administered. In addition to the multiple large tablets required daily, other limitations of Zytiga include the requirement to be taken on an empty stomach and the high variability in systemic exposure. This high variability in systemic exposure has been shown to result in patients with low abiraterone plasma concentrations and exposure, which can lead to suboptimal clinical outcomes.i,ii It is anticipated that TAVT-45 Granules may be given regardless of food consumption and may result in fewer patients having sub-optimal abiraterone trough plasma concentrations.

On August 1, 2022 Novavax, Inc. (Nasdaq: NVAX), a biotechnology company dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, reported it will report its second quarter 2022 financial results and operational highlights on Monday, August 8, 2022, following the close of U.S. financial markets (Press release, Novavax, AUG 1, 2022, https://www.prnewswire.com/news-releases/novavax-to-host-conference-call-to-discuss-second-quarter-2022-financial-results-and-operational-highlights-on-august-8-2022-301596821.html [SID1234617224]). Details of the event and replay are as follows:

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On August 1, 2022 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported the Company will release second quarter and first half 2022 financial results after the market closes on Monday, August 8, 2022 (Press release, Precigen, AUG 1, 2022, View Source [SID1234617223]). The Company will host a conference call that day at 4:30 PM ET to discuss financial results and provide a general business update.

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The conference call may be accessed by dialing 1-888-317-6003 (Domestic US), 1-866-284-3684 (Canada) or 1-412-317-6061 (International) and providing the participant access code 10169605. Participants are asked to dial in 10-15 minutes in advance of the scheduled call time to facilitate timely connection to the call.

Event details can be found on Precigen’s website in the Events & Presentations section at investors.precigen.com/events-presentations.