On August 16, 2022 Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer, and Invivoscribe, a global provider of diagnostic kits and services for oncology, reported their agreement to develop a companion diagnostic (CDx) for use with Kronos Bio’s investigational therapy, entospletinib (Press release, Invivoscribe Technologies, AUG 16, 2022, View Source [SID1234618428]). Entospletinib is Kronos Bio’s lead clinical compound, currently in the ongoing Phase 3 registrational AGILITY study for the treatment of newly diagnosed NPM1-mutated acute myeloid leukemia (AML).

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The diagnostic will screen for the NPM1 mutation, which is present in approximately one-third of all patients with AML.

Over the past year, the two companies have worked together to develop and advance the diagnostic and prepare to submit the Premarket Approval (PMA) application to the U.S. Food and Drug Administration (FDA) at the same time as the submission of the entospletinib New Drug Application (NDA). The FDA requires the validation and approval of companion diagnostics used to select patients for treatment with a specific therapeutic agent.

The agreement builds on Invivoscribe’s experience in developing and obtaining approval for diagnostics used for identification of patients with genetically mutated AML. Invivoscribe markets an FDA-approved CDx for FLT3-mutated AML.

"This companion diagnostic NPM1 mutation assay development work with Kronos Bio represents a significant milestone for our company," said Jeffrey Miller, Ph.D., chief scientific officer and chief executive officer of Invivoscribe. "Companion diagnostics play a key role in the development and approval of targeted drug therapies and these kinds of partnerships are critical to improving care for patients with cancer."

Kronos Bio’s AGILITY trial is designed to assess the efficacy and safety of entospletinib in approximately 180 adults who have been newly diagnosed with NPM1-mutated AML. In the trial, patients are being randomized to receive entospletinib or placebo, in combination with standard induction and consolidation chemotherapy. The primary endpoint of the trial is measurable residual disease (MRD) negative complete response. Event-free survival (EFS) is a key secondary endpoint, and mature EFS data are anticipated to be used to support potential full approval.

"The development of the NPM1 mutation companion diagnostic is a critical step in our efforts to rapidly advance entospletinib," said Jorge DiMartino, M.D., Ph.D., chief medical officer and executive vice president of Clinical Development at Kronos Bio. "We are fortunate to benefit from Invivoscribe’s prior experience in bringing to market companion diagnostics for patients with AML."

On August 16, 2022 I-Mab (the "Company") (Nasdaq: IMAB) reported that the Company and AbbVie Global Enterprises Ltd. (as assignee of AbbVie Ireland Unlimited Company) ("AbbVie") have entered into an amendment to the original license and collaboration agreement dated September 3, 2020 among I-Mab Biopharma (Shanghai) Co., Ltd. and I-Mab Biopharma US Limited, each a wholly-owned subsidiary of the Company, and AbbVie (as amended, the "Agreement") (Press release, I-Mab Biopharma, AUG 16, 2022, View Source [SID1234618427]).

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The parties will continue to collaborate on the global development of anti-CD47 antibody therapy under the Agreement. The Company will be eligible to receive, and AbbVie will pay, up to US$1.295 billion in the development, regulatory and sales milestone payments, and the tiered royalties at rates from mid-to-high single digit percentages on global net sales outside of Greater China for certain new anti-CD47 antibodies currently in development, or the original milestone payments and tiered royalties previously disclosed in the Company’s Form 20-F for the fiscal year 2021 for other licensed products. The Company has the exclusive right to develop and commercialize all licensed products under the Agreement in Greater China.

AbbVie will discontinue the global Phase 1b study of lemzoparlimab combination therapy with azacitidine ("AZA") and venetoclax, in patients with myelodysplastic syndrome ("MDS") and acute myelocytic leukemia ("AML"). This decision was not based on any specific or unexpected safety concerns.

The Company continues its commitment on lemzoparlimab development with a near-term focus on the initiation of a Phase 3 clinical trial in patients with MDS in China, which is supported by the safety and efficacy data from its Phase 2 study of combination therapy of lemzoparlimab and AZA in patients with higher risk MDS. The detailed data will be presented in a proffered paper at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in September 2022. To date, Phase 1 and Phase 2 clinical studies of lemzoparlimab in the U.S. and China with nearly 200 patients enrolled have shown a good safety profile without the need for a priming dosing regimen.

The Company has a strong cash position (US$671 million in cash, cash equivalents and short-term investments as of December 31, 2021) to support the ongoing and planned clinical development of lemzoparlimab, in addition to other critical late-stage clinical assets.

On August 16, 2022 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that the United States Patent and Trademark Office (USPTO) has granted Genprex U.S. Patent No: 11,278,592 B2 (Press release, Genprex, AUG 16, 2022, View Source [SID1234618426]). The patent covers methods of using REQORSATM Immunogene Therapy in conjunction with immune checkpoint inhibitors, through 2038.

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"This new patent provides critical protection for our REQORSA gene therapy in combination with a checkpoint inhibitor, for example an anti-PD1 antibody, to treat cancer. This is important as our ongoing Acclaim-2 Phase 1/2 clinical trial combines REQORSA with Keytruda, an anti-PD1 antibody, to treat non-small cell lung cancer," noted Thomas Gallagher, Esq., Senior Vice President of Intellectual Property and Licensing at Genprex. "We continue to strengthen our intellectual property portfolio and continue to build protection around our technology as it safeguards our gene therapy with target-specific combination therapy, is a deterrent to would-be competitors and creates value around our core competencies."

Acclaim-2 is an open-label, multi-center Phase 1/2 clinical trial evaluating the Company’s lead drug candidate, REQORSA Immunogene Therapy, in combination with Keytruda (pembrolizumab) in patients with late-stage non-small cell lung cancer (NSCLC) whose disease progressed after treatment with Keytruda. More information on Acclaim-2 can be found at www.clinicaltrials.gov Identifier NCT05062980.

In 2021, Genprex received U.S. Food and Drug Administration’s (FDA) Fast Track Designation for treatment of the Acclaim-2 patient population.

Keytruda is a registered trademark of Merck & Co. and is its largest selling drug with 2021 sales of more than $17 billion.

About Acclaim-2

The Acclaim-2 trial is a Phase 1/2 open-label, dose-escalation and clinical response study of REQORSA in combination with Keytruda in patients with advanced, metastatic non-small-cell lung cancer who have progressed after treatment with Keytruda. The Company anticipates enrolling patients at approximately 15 clinical sites and estimates that the Phase 1 portion of the Acclaim-2 trial will enroll up to 30 patients and the Phase 2 portion will enroll approximately 126 patients. Patients enrolled in the Phase 2 portion of the study will be randomized 2:1 to either REQORSA and Keytruda combination therapy or to chemotherapy (docetaxel with or without ramucirumab). Patients will be treated until disease progression or unacceptable toxicity is experienced. Patients must have histologically confirmed unresectable stage III or IV NSCLC (any histology) with radiological progression on Keytruda and an ECOG performance status of 0 to 1. Genprex expects to complete the Phase 1 portion of Acclaim-2 by mid-2023.

About REQORSA

REQORSA Immunogene Therapy (quaratusugene ozeplasmid) for non-small cell lung cancer (NSCLC) uses Genprex’s unique, proprietary ONCOPREX Nanoparticle Delivery System, which is the first systemic gene therapy delivery platform used for cancer in human clinical trials. The active ingredient in REQORSA is the TUSC2 gene, a tumor suppressor gene. REQORSA consists of the TUSC2 gene encapsulated in a nanoparticle made from lipid molecules with a net positive electrical charge. REQORSA is injected intravenously and can specifically target cancer cells, which generally have a negative electrical charge. Once REQORSA is taken up into a cancer cell, the TUSC2 gene is expressed, and the TUSC2 protein is capable of restoring certain defective functions arising in the cancer cell. REQORSA has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for programmed cell death, or apoptosis, in cancer cells, and modulates the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance.

Tagrisso is a registered trademark of AstraZeneca plc and its largest selling drug with 2021 sales of over $5 billion.

On August 16, 2022 Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Orna Therapeutics, a biotechnology company pioneering a new investigational class of engineered circular RNA (oRNA) therapies, reported a collaboration agreement to discover, develop, and commercialize multiple programs, including vaccines and therapeutics in the areas of infectious disease and oncology (Press release, Merck & Co, AUG 16, 2022, View Source [SID1234618425]).

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Under the terms of the agreement, Merck will make an upfront payment to Orna of $150 million, which will be expensed by Merck in the third quarter of 2022 and included in non-GAAP results. In addition, Orna will be eligible to receive up to $3.5 billion in development, regulatory, and sales milestones associated with the progress of the multiple vaccine and therapeutic programs, as well as royalties on any approved products derived from the collaboration. Orna will retain rights to its oRNA-LNP technology platform and will continue to advance other wholly owned programs in areas such as oncology and genetic disease. Merck will also invest $100 million of equity in Orna’s recently completed Series B financing round.

Orna’s proprietary oRNA technology creates circular RNAs (oRNAs) from linear RNAs by self-circularization. oRNA molecules have been shown to have greater stability in vivo than linear mRNA and have the potential to produce larger quantities of therapeutic proteins inside the body. Newly synthesized oRNA molecules are more compactly packaged into custom lipid nanoparticles (LNPs), which Orna has engineered to target key tissues in the body. Preclinical data, including presentations at the 2022 American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, have demonstrated the potential of oRNA expression and delivery as an approach for further development in multiple areas, including vaccines and oncology therapeutics.

"This broad strategic collaboration brings together Merck’s significant expertise in nucleic acid biology, clinical development, and manufacturing with Orna’s compelling circular RNA technology to explore the opportunity to develop a new generation of potential vaccines and therapeutics," said Fiona Marshall, senior vice president and head of discovery, preclinical and translational medicine at Merck Research Laboratories. "We look forward to working with the talented scientific and technical teams at Orna."

"We are thrilled to collaborate with Merck, a company committed to breakthrough science, which has recognized the potential our platform can bring to patients. Our oRNA technology plus novel delivery solutions are designed to unlock the full potential of RNA in therapeutics and vaccines," said Tom Barnes, Chief Executive Officer of Orna. "The combined expertise of Orna and Merck aims to accelerate the development of RNA therapeutics for patients in need of better treatment options."

On August 16, 2022 Senda Biosciences, Inc., a company that is harnessing nature to program targeted, potent, and tunable medicines, reported the completion of a $123 million Series C financing, bringing its total funding raised to date to $266 million (Press release, Senda Biosciences, AUG 16, 2022, View Source [SID1234618424]). Flagship Pioneering, which founded Senda, participated along with new investors, including the Samsung Life Science Fund, Qatar Investment Authority (QIA), Bluwave Capital, and Stage 1 Ventures. Also participating in the financing are current investors, including Alexandria Venture Investments, Longevity Vision Fund, Mayo Clinic, Partners Investment, and State of Michigan Retirement System.

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"Senda is pioneering the development of comprehensively programmable medicines with the potential to reach previously inaccessible cells, tissues, and organs," said Guillaume Pfefer, Ph.D., Chief Executive Officer of Senda Biosciences and Partner at Flagship Pioneering. "We believe our approach could transform the lives of patients in need of novel treatments, and so we’re delighted to have attracted support from investors who recognize this potential and its significance."

The proceeds of the financing will be used to drive further development of Senda’s proprietary programmable medicines platform and advance its first programs into clinical testing. Senda’s platform deconstructs the chemical-addressing codes of natural nanoparticles from species across all kingdoms of life that have evolved to precisely shuttle biomolecules into human cells. By cataloging these chemical-addressing codes into a large, diverse atlas, Senda is programming nanoparticles with key bioproperties, including specific cell and tissue targeting and repeatable dosing. Combining the atlas with an mRNA engine, Senda’s platform fully unlocks the potential to generate a new class of comprehensively programmed medicines. The unique properties of these medicines create new frontiers for therapeutics and vaccines with further possible applications in the gene-editing and protein-based therapy landscapes.

Dr. Pfefer continued: "The enormous therapeutic promise of information molecules, such as mRNA, siRNA, and gene editors—which enable programming within cells of interest—has yet to be realized, owing in part to an inability to program to cells of interest. Senda’s extensive body of preclinical data in small and large animals, and across a range of disease models, shows that by combining these programmed nanoparticles with information molecules, we can program within and to cells. It’s quite exciting to know that this round of funding will help accelerate our platform expansion and refinement as we move our first programs toward the clinic."

"Senda’s unique approach of programming specifically to cells of interest could be the key to unleashing the revolutionary therapeutic potential of current and future information molecules," said Ignacio Martinez, Chairperson of the Board, cofounder of Senda Biosciences, and General Partner, Flagship Pioneering. "We’re thrilled that this group of co-investors has joined us to propel Senda’s pursuit of life-changing medicines."