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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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."

On August 16, 2022 Bristol Myers Squibb (NYSE:BMY) and Turning Point Therapeutics, Inc. (NASDAQ:TPTX) ("Turning Point") reported the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended ("HSR Act"), and the receipt of merger control clearance from the Federal Cartel Office of Germany ("FCO"), in connection with the previously announced offer (the "Offer") to acquire all outstanding shares of common stock of Turning Point at a price of $76.00 per share in an all-cash transaction for total consideration of approximately $4.1 billion (Press release, Bristol-Myers Squibb, AUG 16, 2022, View Source [SID1234618422]). The expiration of the HSR Act waiting period occurred at 11:59 p.m. Eastern Time on August 15, 2022, and the FCO clearance was received on August 15, 2022. The Offer expired at 5:00 p.m. Eastern Time on August 15, 2022 (the "Expiration Time"), and the Offer was not extended.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Equiniti Trust Company, the depositary for the Offer, has advised that, as of the Expiration Time, approximately 41,896,678 shares of common stock were validly tendered, and not validly withdrawn pursuant to Offer, representing approximately 84% of the issued and outstanding shares of common stock.

The parties expect the transaction to close on August 17, 2022, promptly following the acceptance of all shares of common stock validly tendered and not validly withdrawn pursuant to the Offer.

Turning Point shareholders can direct questions regarding the Offer to MacKenzie Partners, Inc., the information agent for the Offer, toll free, at 1-800-322-2885.

On August 16, 2022 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a pre-commercial-stage biopharmaceutical company focused on oncology, reported its financial results for the second quarter ended June 30, 2022 and provides a corporate update (Press release, BioLineRx, AUG 16, 2022, View Source [SID1234618421]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Significant events and achievements during the second quarter 2022 and subsequent period:

Progressed the New Drug Application (NDA) for Motixafortide in stem cell mobilization (SCM), with submission to the FDA expected within the next 4-6 weeks;
Appointed commercial strategy and operations veteran Holly May as Chief Commercial Officer, based in the U.S.;
Continued to advance critical pre-launch activities with respect to Motixafortide commercialization in the U.S., if approved;
Entered into a development collaboration agreement with GenFleet Therapeutics to execute a randomized Phase 2b clinical trial of Motixafortide, in combination with anti-PD1 and chemotherapy, for first-line treatment in approximately 200 pancreatic cancer (PDAC) patients in China;
Ended the second quarter on solid financial footing, with cash and cash equivalents of $43.2 million, sufficient to fund operations, as currently planned, into the first half of 2024.
"Since our last quarterly update, we achieved significant progress across both our Motixafortide stem cell mobilization and pancreatic cancer (PDAC) programs," stated Philip Serlin, Chief Executive Officer of BioLineRx. "With respect to stem cell mobilization, we are in the final stages of preparing for submission of our NDA to the FDA. With Holly May on board as our new Chief Commercial Officer, we are rapidly advancing critical pre-launch activities while we continue to assess all of our options with respect to commercialization of Motixafortide in the U.S., if approved."

"The totality of data that we have compiled in stem cell mobilization, both clinical and pharmacoeconomic, make an extremely strong case for Motixafortide as the standard of care in this indication for all multiple myeloma patients undergoing autologous stem-cell transplantation, which is a highly concentrated end market estimated to be $360 million in the U.S. alone and growing consistently."

"In PDAC, the development collaboration agreement that we announced with GenFleet builds upon the positive results from our COMBAT/KEYNOTE-202 study, and we look forward to the initiation of a randomized Phase 2b PDAC trial next year. Importantly, this collaboration allows us to advance the development of Motixafortide in PDAC while retaining rights to the molecule across all indications and geographies."

"Finally, we are nearing a significant milestone for our second program, the anti-cancer vaccine AGI-134, with the upcoming release of proof-of-mechanism data from part 2 of a Phase 1/2a trial in solid tumors. If positive, we plan to initiate a randomized Phase 2 study next year."

"In summary, we believe we are well-positioned to deliver several meaningful potential regulatory, commercial and clinical catalysts over the next 12-18 months," concluded Mr. Serlin.

Upcoming Expected Milestones:

Submission of NDA to FDA for Motixafortide as novel mobilization agent for multiple myeloma patients undergoing autologous stem cell transplantation in next 4-6 weeks;
Initial results from Part 2 of Phase 1/2a trial of AGI-134 in solid tumors in H2 2022;
Potential FDA approval of Motixafortide in 2023;
Potential US launch of Motixafortide in SCM in 2023;
Initiation of randomized Phase 2b study in PDAC under collaboration with GenFleet in 2023;
Potential initiation of randomized Phase 2 study of AGI-134 in 2023.
Financial Results for the Quarter Ended June 30, 2022:

Research and development expenses for the three months ended June 30, 2022 were $5.4 million, an increase of $0.3 million, or 5.0%, compared to $5.1 million for the three months ended June 30, 2021. The increase resulted primarily from an increase in expenses associated with the AGI-134 study, offset by lower expenses associated with the completed Motixafortide GENESIS trial, as well as lower expenses related to NDA supporting activities related to Motixafortide. Research and development expenses for the six months ended June 30, 2022 were $9.8 million, an increase of $0.4 million, or 4.4%, compared to $9.4 million for the six months ended June 30, 2021. The reason for the increase is similar to the aforementioned increase in the three-month period.

Sales and marketing expenses for the three months ended June 30, 2022 were $1.2 million, an increase of $0.8 million, or 250.9% compared to $0.3 million for the three months ended June 30, 2021. The increase resulted primarily from initiation of pre-commercialization activities related to Motixafortide, as well as an increase in market research. Sales and marketing expenses for the six months ended June 30, 2022 were $1.8 million, an increase of $1.3 million, or 270.9% compared to $0.5 million for the six months ended June 30, 2021. The reason for the increase is similar to the aforementioned increase in the three-month period.

General and administrative expenses for the three months ended June 30, 2022 were $1.0 million, similar to the comparable period in 2021. General and administrative expenses for the six months ended June 30, 2022 were $2.1 million, similar to the comparable period in 2021.

The Company’s operating loss for the three months ended June 30, 2022 amounted to $7.6 million, compared to an operating loss of $6.5 million for the comparable period in 2021. The Company’s operating loss for the six months ended June 30, 2022 was $13.7 million, compared to $12.0 million for the comparable period in 2021.

Non-operating income (expenses) for the three and six months ended June 30, 2022 and for the three and six months ended June 30, 2021 primarily relate to fair-value adjustments of warrant liabilities on the Company’s balance sheet, offset by warrant offering expenses.

Net financial expenses for the three months ended June 30, 2022 amounted to $0.3 million, compared to net financial expenses of $0.1 million for the three months ended June 30, 2021. Net financial expenses for the 2022 period primarily relate to loan interest paid and losses recorded on foreign currency (primarily NIS) cash balances due to the strengthening of the US dollar during the period, offset by investment income earned on bank deposits. Net financial expenses for the 2021 period primarily relate to loan interest paid, offset by investment income earned on bank deposits. Net financial expenses for the six months ended June 30, 2022 amounted to $0.4 million, compared to net financial expenses of $0.3 million for the six months ended June 30, 2021. The composition of the expenses is similar to the aforementioned composition detailed in the three-month periods.

The Company’s net loss for the three months ended June 30, 2022 amounted to $7.4 million, compared with a net loss of $6.8 million for the comparable period in 2021. The Company’s net loss for the six months ended June 30, 2022 amounted to $12.4 million, compared with a net loss of $17.0 million for the comparable period in 2021.

The Company held $43.2 million in cash, cash equivalents and short-term bank deposits as of June 30, 2022.

Net cash used in operating activities was $11.9 million for the six months ended June 30, 2022, compared with net cash used in operating activities of $13.1 million for the six months ended June 30, 2021. The $1.2 million decrease in net cash used in operating activities between the two periods was primarily the result of changes in operating asset and liability items in the two periods, i.e., a smaller increase in prepaid expenses and other receivables in 2022 versus 2021, as well as an increase in accounts payable and accruals in 2022 versus decrease in the 2021 period.

Net cash provided by investing activities was $15.1 million for the six months ended June 30, 2022, compared to net cash used in investing activities of $42.3 million for the six months ended June 30, 2021. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits.

Net cash used in financing activities was $1.6 million for the six months ended June 30, 2022, compared to net cash provided by financing activities of $56.0 million for the six months ended June 30, 2021. The cash flows in 2022 primarily reflect the repayments of the loan from Kreos Capital. The cash flows in 2021 primarily reflect the underwritten public offering of the Company’s ADSs in January 2021, warrant exercises and net proceeds from the ATM facility, offset by repayments of the loan from Kreos Capital.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, Tuesday, August 16 at 10:00 a.m. EDT. To access the conference call, please dial +1-888-281-1167 from the US or +972-3-918-0685 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until August 18, 2022; please dial +1-888-295-2634 from the US or +972-3-925-5904 internationally.