On December 1, 2021 Zetagen Therapeutics, a private, clinical-stage, biopharmaceutical company dedicated to driving breakthrough innovation in the treatment of metastatic bone cancers and osteologic interventions, reported it has received Breakthrough Device designation from the Centers for Devices and Radiological Health (CDRH) of the U.S. Food and Drug Administration (FDA) for its ZetaMet (Zeta-BC-003) technology (Press release, Zetagen Therapeutics, DEC 1, 2021, View Source [SID1234643705]). Previously known as ZetaFuse, ZetaMet (Zeta-BC-003) is a synthetic, small-molecule, inductive biologic technology being developed to target and resolve metastatic bone lesions while inhibiting future tumor growth and regenerating bone.

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"We are pleased to receive this important designation from the Agency and look forward to partnering with them," said Joe C. Loy, CEO of Zetagen Therapeutics. "Our researchers have discovered an entirely new pathway for an established molecule which, if proven successful in human clinical trials, could create a new treatment paradigm for the hundreds of thousands of patients living with cancers that involve metastatic bone lesions."

ZetaMet (Zeta-BC-003) works through a mechanism of action (MOA) which is a novel and patented molecular pathway. The small molecule, precisely-dosed, delivered to the affected area through a proprietary drug-eluting carrier, stimulates stem cells, activating cells to grow healthy bone known as "osteoblasts", and inhibits cells associated with bone degradation called "osteoclasts". The combination technology has, thus far, in preclinical studies, demonstrated its ability to resolve existing metastatic bone lesions, inhibit pain and stimulate targeted bone regeneration.

Bone metastases are common among cancer patients and occur when cells from the primary cancerous tumor relocate to the bone. When these cancers relocate, they can cause changes to the bone, damaging it in a process called osteolysis. Osteolysis can cause small holes within the bone, weakening it and increasing the risk of breakage. These holes are called "lytic lesions." Among cancers which metastasize to bone, Breast and Prostate are most prevalent, amounting to approximately 70-percent of cases.[1]

ZetaMet (Zeta-BC-003) has successfully passed its preclinical trials and is being prepared for its first human clinical trial in early 2022.

On December 1, 2021 BeyondSpring Pharmaceuticals (the "Company" or "BeyondSpring") (NASDAQ: BYSI), a global pharmaceutical company focused on the development of cancer therapeutics, reported it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) seeking approval of plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia (CIN) (Press release, BeyondSpring Pharmaceuticals, DEC 1, 2021, View Source;utm_medium=rss&utm_campaign=beyondspring-pharmaceuticals-receives-complete-response-letter-from-the-fda-for-plinabulin-new-drug-application-for-prevention-of-chemotherapy-induced-neutropenia-cin [SID1234596319]). The FDA issued the CRL to indicate that they have completed their review of the application and have determined that it cannot be approved in its present form.

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The FDA’s CRL indicated that the results of the single registrational trial (106 Phase 3) was not sufficiently robust to demonstrate benefit and that a second well controlled trial would be required to satisfy the substantial evidence requirement to support the CIN indication.

"BeyondSpring strongly believes that plinabulin in combination with G-CSF has significant potential to raise the standard of care in CIN, a devastating side effect of chemotherapy," said Dr. Lan Huang, BeyondSpring’s co-founder, chief executive officer and chairwoman. "The Company plans to request a meeting with the FDA and remains committed to its goal of bringing plinabulin to cancer patients in need globally."

BeyondSpring remains confident in the efficacy and safety data for plinabulin in combination with G-CSF for the prevention of CIN. The Company expects to work closely with the FDA to consider the possible future clinical pathway for CIN, which may include a second study.

Plinabulin is the first drug candidate submitted for FDA approval that has the potential to work in the critical first week of chemotherapy treatment before G-CSF is effective, to prevent the onset and improve clinical outcomes of CIN.

About Plinabulin
Plinabulin, BeyondSpring’s lead asset, is a selective immunomodulating microtubule-binding agent (SIMBA), which is a potent antigen presenting cell (APC) inducer. It is a novel, intravenous infused, patent-protected, NDA-stage asset for CIN prevention and a Phase 3 anti-cancer candidate for non-small cell lung cancer (NSCLC) with recently released positive topline data. Plinabulin triggers the release of the immune defense protein, GEF-H1, which leads to two distinct effects: first is a durable anticancer benefit due to the maturation of dendritic cells resulting in the activation of tumor antigen-specific T-cells to target cancer cells, and the second is early-onset of action in CIN prevention after chemotherapy by boosting the number of hematopoietic stem/progenitor cells (HSPCs). Plinabulin received Breakthrough Therapy designation and priority review from both U.S. and China FDA for the CIN prevention indication. As a "pipeline in a drug," plinabulin is being broadly studied in combination with various immuno-oncology agents that could boost the effects of the PD-1/PD-L1 antibodies and re-sensitize PD-1/PD-L1 antibody-resistant patients.

On December 1, 2021 Melanie Comito, MD, division chief of hematology and oncology at Upstate Golisano Children’s Hospital, reported that it has been awarded a $50,000 infrastructure grant by the St. Baldrick’s Foundation (Press release, SUNY Upstate, DEC 1, 2021, View Source [SID1234596336]).

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These one-year grant provides Upstate the staffing to open, coordinate, and treat more children on clinical trials, making it possible for more children to access these trials close to home.

Upstate was on of 24 institutions to receive an infrastructure grant, totaling more than $1.1 million.

The award supports the work at the Dr William J. Waters Center for Children’s Cancer and Blood Disorders at Upstate Golisano Children’s Hospital, which provides oncology care to children, adolescents, and young adults.

"To be able to offer clinical trials for children with cancers is one of the most important things we can do at our center," Comito said. "As a smaller center, we still see a variety of types of cancer, so we have to be prepared by having as many trials open as possible.

"We currently have 47 open clinical trials available for pediatric cancer patients which allows children in our 17-county area to have access to clinical trials closer to home, Comito added. "St. Baldrick’s infrastructure support is essential for supporting our clinical research staff so that we can meet this goal of curing as many children as possible."

"St. Baldrick’s Infrastructure Grants are designed for one reason, to treat more children on clinical trials, often their best hope for a cure," said Kathleen Ruddy, St. Baldrick’s CEO. "Thanks to donors, volunteers, advocates, and all those who are fighting every day for kids with cancer, from making these $1.1 million in grants possible. These grants are particularly critical because they often help children who are treated at smaller hospitals, or those where resources are scarce, but needs are high."

St. Baldrick’s is a volunteer-driven charity committed to funding the most promising research to find cures for childhood cancers. St. Baldrick’s coordinates its signature head-shaving events worldwide where participants collect pledges to shave their heads in solidarity with kids with cancer, raising money to fund research.

On December 1, 2021 McKesson Corporation (NYSE: MCK) reported that it will host an Investor Day on Wednesday, December 8, 2021 from 1:00 PM to 4:00 PM ET in New York City (Press release, McKesson, DEC 1, 2021, View Source [SID1234596355]). The meeting will feature presentations by McKesson’s leadership team and a live Q&A session with chief executive officer Brian Tyler and chief financial officer Britt Vitalone . Management will provide an overview of the company’s progress towards its goal of delivering sustainable growth and details around the company’s long-term financial outlook.

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Webcast and Presentations

The video webcast will be available live and archived on McKesson’s Investor Relations website, along with the company’s slide presentation, at investor.mckesson.com.

On December 1, 2021 Australian clinical-stage drug development company Noxopharm (ASX:NOX) reported that it has in-licensed novel RNA technology developed by Hudson Institute of Medical Research (Press release, Noxopharm, DEC 1, 2021, View Source [SID1234596378]). The exclusive global contract aims to maximize opportunities in RNA drug discovery and mRNA vaccine manufacture through Noxopharm’s wholly owned subsidiary, Pharmorage.

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According to Noxopharm CEO and Managing Director, Graham Kelly, "Pharmorage already had a strong business relationship with Hudson with a major initiative in anti-inflammatory drug development. The RNA technology and its anti-inflammatory functions is an obvious fit, and with mRNA vaccine technology looking increasingly likely to extend eventually to most if not all viral diseases, this is an extraordinarily timely development." Noxopharm has previous experience in developing anti-inflammatory therapeutics, which led to their current lead drug candidate, Veyonda.

As a first step, Pharmorage will develop Hudson’s mRNA vaccine enhancement opportunities, as the mRNA vaccine market is predicted to reach $23 billion USD by 2035. Though mRNA vaccine technology has had a strong debut, unwanted side effects remain common, including fatigue, severe headache, chills, and injection-site pain. These are directly related to the body recognizing mRNA therapeutics through the immune sensor Toll-like Receptor 7 (TLR7). However, according to Associate Professor Michael Gantier of Hudson, "We have discovered a new class of TLR7 inhibitors that can outcompete immune sensing of therapeutic RNAs, such as those used in mRNA vaccines; we propose that these inhibitors could be used in conjunction with therapeutic RNAs to limit their side effects in patients and maximize their therapeutic potential."

Pharmorage will also focus on advancing Hudson’s several lead RNA drugs through development as treatments for autoimmune and inflammatory diseases. These RNA drugs target the root of the inflammatory response — key immune sensors. Pharmorage has experience in this area, evidenced by their work on the development of a first-in-class tank-binding kinase 1 (TBK1) inhibitor, to be used in similar conditions.