Merck and Orna Therapeutics Collaborate to Advance Orna’s Next Generation of RNA Technology

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."

Senda Biosciences Announces Close of $123 Million Series C Financing

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."

Bristol Myers Squibb and Turning Point Therapeutics Announce Expiration of HSR Act Waiting Period and Clearance from Federal Cartel Office of Germany Related to Pending Acquisition of Turning Point Therapeutics

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.

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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.

BioLineRx Reports Second Quarter 2022 Financial Results and Provides Corporate Update

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]).

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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.

QSAM Biosciences Reports Second Quarter 2022 Financial Results and Provides Corporate Update

On August 16, 2022 QSAM Biosciences Inc. (OTCQB: QSAM), a company developing next-generation therapeutic radiopharmaceuticals, including Samarium-153-DOTMP (CycloSam), for the treatment of bone cancer and related diseases, reported that financial results for the second quarter ended June 30, 2022, as filed with the SEC on August 15, 2022 in the Company’s Form 10-Q, and provides a corporate update (Press release, QSAM Biosciences, AUG 16, 2022, View Source [SID1234618416]).

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Recent Corporate Highlights:

Initial, preliminary evaluation of the first patient dosed in Phase 1 clinical trial for CycloSam for the treatment of metastatic bone cancer suggests promising results.
Second patient dosing and completion of the first cohort of patients in the trial is expected in the third calendar quarter.
Additional clinical trial sites for CycloSam and additional nuclear reactor sites expected to be added in the third calendar quarter.
Third key patent received in the United States covering the production and delivery of CycloSam, strengthening Company’s extensive patent portfolio.
"We are encouraged by the initial, preliminary data from the first patient dosed with CycloSam. While it is too early for formal conclusions to be made of these early results, we did see positive signals in terms of safety and efficacy. CycloSam performed how we expected it to perform, even given the lowest dosage in our dose-escalating study. Over the next few months, we expect to make steady progress in our first clinical trial, including establishing additional trial sites that will help us treat more patients, and qualifying additional nuclear reactors to build redundancy and efficiencies in our supply chain," stated Douglas R. Baum, CEO and co-founder of the Company.

"Our decision to postpone our planned equity raise and NASDAQ uplisting in May was based on market conditions at that time. We are now seeing the beginnings of a turn-around in the broader biotech markets. If these continue, and we achieve the progress we expect in our clinical trials over the next six months, we intend to revisit the uplisting process in early 2023. We are actively working towards creating shareholder value in conjunction with our most important mission to help adult and pediatric patients suffering from bone cancer," added Baum.

Summary Financial Results for the Quarter Ended June 30, 2022:
(All numbers except shares are approximated)

Net loss attributable to common stockholders for the quarter ended June 30, 2022, was $1.33 million, compared to $3.07 million for the quarter ended June 30, 2021, a decrease of 57%.

Operating expenses were $1.26 million for the three months ended June 30, 2022, as compared to $2.90 million for the three months ended June 30, 2021. The $1.64 million decrease in operating expenses was largely due to a decrease in compensation and related expenses of $1.76 million in the three months ended June 30, 2022, which is comprised of $2.44 million of expenses from the vesting of Series E-1 Stock in the 2021 period as compared to $0.22 million of expenses from Series E-1 Stock vesting in the 2022 period, plus stock option compensation of $0.21 million and deferred management compensation of $0.24 million from an agreement with management to take reduced salaries in the 2022 period. In the second quarter of 2022, we also had an increase in professional fees of $0.07 million related to our terminated public offering, and an increase in research and development expense of $0.06 million related to our clinical trials, over the previous year period.

As of June 30, 2022, the Company had cash of approximately $0.43 million. The Company believes this is sufficient capital to support operations at the current pace into the third quarter of 2022. The Company expects to raise additional capital through equity or debt offerings in 2022 to support its clinical trials and other operating expenses.

As of June 30, 2022, the Company had 1,686,587 common shares outstanding. The Company did not issue any shares or options in the quarter. At the end of the second quarter of 2022 there were approximately 497,594 shares of common stock that could be issued upon the conversion of preferred stock, warrants and convertible debt, excluding employee stock options.

The following tables summarize our results of operations for the periods indicated, and are qualified in their entirety by the Company’s Form 10-Q for the period ended June 30, 2022, filed with the SEC on August 15, 2022, including the financial footnotes contained therein