On July 24, 2023 AvenCell Therapeutics, Inc., a leading clinical-stage cell therapy company focused on advancing both switchable and allogeneic engineered CAR-T cell therapies, reported that the European Medicines Agency (EMA) has approved the company’s Clinical Trial Application (CTA) application for AVC-201 for the treatment of relapsed/refractory Acute Myeloid Leukemia (AML) and other selected hematologic malignancies positive for CD123 (NCT05949125) (Press release, AvenCell Therapeutics, JUL 24, 2023, View Source [SID1234633395]). AVC-201 is a CRISPR-engineered allogeneic switchable CAR-T candidate designed to target and eliminate cells expressing receptor CD123, which is known to be overexpressed in nearly all acute myeloid leukemias, and several other hematological malignancies.

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The phase 1 study, which includes up to 35 patients, will be conducted at multiple sites in Germany. The primary objective of the trial is to assess the safety profile of AVC-201 and to determine the maximum tolerated dose. Secondary measures will include efficacy, safety, and CAR-T persistence.

"We are excited to build on the early success and promising activity and safety of our ongoing switchable autologous clinical program in AML (AVC-101) by now including what we believe is the most scientifically-compelling allogeneic technology in the industry," said Andrew Schiermeier, AvenCell’s President & CEO. "With the application of both platform technologies, we are the first company in this space to completely separate the manufacture of cells from the ultimate patients and cancer indications they will be targeted to. This modular approach allows for unparalleled future flexibility and reduction in cycle times, massive scaling of supply, and meaningful reductions in cost of goods, all of which will dramatically advance the field of cell therapy for patients."

About AVC-201
AVC-201 is a CRISPR-edited Chimeric Antigen Receptor ("CAR")-T Cell therapy that embodies two discrete technology platforms. The first leverages AvenCell’s "UniCAR" universal/switchable technology which is comprised of a two-component system. Engineered T Cells are transduced with a "universal" receptor that is completely biologically inert (expressing human La peptide), and are only activated when bound to a second biologic molecule ("targeting module") which directs the T cells to a cancer antigen of interest (in this case, CD123). The presence or absence of the targeting module in circulation allows for exquisite "on" and "off" control, respectively, of the therapeutic activity. The second technology platform consists of an in-licensed allogeneic cell engineering technology developed by Intellia Therapeutics which allows for unrelated donors to provide cells for patients. These cells are uniquely engineered via CRISPR/Cas9 to avoid GvHD and rejection via the host/patient immune system by either innate or adaptive mechanisms.

About AVC-201 Clinical Program
AvenCell’s Phase I study (NCT05949125) is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of AVC-201 in adults with relapsed or refractory AML and other CD123 positive hematological malignancies. The study is an open-label, single-ascending dose design used to identify two dose levels of AVC-201 that will be further evaluated in a subsequent Phase 2 study.

About Acute Myeloid Leukemia (AML)
AML typically develops from mutations in the DNA of early blood-forming cells, leading to the disruption of normal cell maturation and proliferation. This results in a buildup of immature cells in the bone marrow, crowding out healthy cells and impairing their ability to function properly. AML accounts for a significant proportion of all leukemia cases. In the United States, it is estimated that approximately 21,000 new cases of AML will be diagnosed in 2023, while on a global scale, the incidence of AML is estimated to be around 2-8 cases per 100,000 people annually. Treatment for AML usually involves chemotherapy to destroy cancer cells, and more recently available targeted therapies directed against specific mutations (e.g. IDH, FLT3.) While a majority of AML cells express the receptor CD123, several previous attempts to target this receptor therapeutically have failed due to the difficulty in managing toxicity. Stem cell transplant continues to be considered the only curative option. The survival rate of AML can vary depending on several factors, including age, overall health, specific genetic mutations, response to treatment, and other individual characteristics. However, the overall five-year survival rate for AML is around 25-30%.

On July 24, 2023 HDT Bio Corp., a clinical stage private company developing advanced RNA vaccine products to treat and prevent infectious diseases and cancer, reported the publication of preclinical data demonstrating that the company’s AMPLIFY vaccine platform, comprised of self-replicating RNA (repRNA) and LION formulation technology, holds a more favorable safety profile and increased durability and localization compared to repRNA vaccines delivered by lipid nanoparticles (LNPs), similar to those used to deliver currently-available RNA vaccines for COVID-19 (Press release, HDT Bio, JUL 24, 2023, View Source [SID1234633394]). The data were published in the journal Molecular Therapy, in an article entitled, "A localizing nanocarrier formulation enables multi-target immune responses to multivalent replicating RNA with limited systemic inflammation."

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"While RNA vaccines have greatly curtailed the COVID-19 pandemic, legitimate safety concerns have arisen that must be taken into consideration," said Steve Reed, Ph.D., Chief Executive Officer of HDT Bio. "The findings of this study demonstrate that our LION delivery system offers unique properties that greatly improve upon the safety, immunogenicity and efficacy of the type of lipid nanoparticles that are currently in use. This is a clear demonstration that AMPLIFY is a next-generation platform with the potential to transform immunizations worldwide."

The key findings of the publication, conducted in a mouse model, are summarized below.

Intramuscular administration of repRNA with LION technology (repRNA/LION) restricted RNA expression to the muscle, whereas repRNA with LNPs (repRNA/LNP) was observed broadly throughout the body;
repRNA/LNP triggered both local and systemic innate immune/inflammatory responses, whereas repRNA/LION restricted innate immune activity to the local injection site, but did not trigger a systemic inflammatory response;
repRNA/LION induced a comparable antibody and T cell response to repRNA/LNP, despite the absence of a systemic response;
In a multivalent vaccine design repRNA/LION induced potent neutralizing antibody responses to each antigen.
The publication can be found on the Molecular Therapy website and on the Publications page of the HDT Bio website.

On July 24, 2023 argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported the closing of its previously announced global offering of an aggregate of 2,581,633 ordinary shares, which may be represented by American Depositary Shares ("ADSs") and which includes full exercise of the underwriters’ option to purchase 336,734 additional ordinary shares in the form of ADSs (Press release, argenx, JUL 24, 2023, View Source [SID1234633393]). The gross proceeds from the global offering were approximately $1.27 billion (approximately €1.13 billion).

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J.P. Morgan, Morgan Stanley, Goldman Sachs & Co. LLC, BofA Securities and TD Cowen acted as joint bookrunning managers for the offering. Van Lanschot Kempen acted as manager for the offering.

The securities were offered in the United States pursuant to an automatically effective shelf registration statement that was previously filed with the Securities and Exchange Commission ("SEC"). A preliminary prospectus supplement relating to the securities, filed with the SEC on July 17, 2023, and a final prospectus supplement relating to the securities, filed with the SEC on July 20, 2023, are both available on the SEC’s website at www.sec.gov.

Copies of the final prospectus supplement and the accompanying prospectus relating to the U.S. offering may be obtained for free from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by email at [email protected], or by telephone at (866) 803-9204; from Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, NY 10014, Attn: Prospectus Department, or by email at [email protected]; from Goldman Sachs & Co. LLC, 200 West Street, New York, NY 10282, Attn: Prospectus Department, by email at [email protected], or by telephone at 866-471- 2526; from BofA Securities, NC1-022-02-25, 201 North Tryon Street, Charlotte, North Carolina 28255-0001, Attn: Prospectus Department, or by email at [email protected]; or from Cowen and Company, LLC, 599 Lexington Avenue, New York, NY 10022, by email at [email protected], or by telephone at (833) 297- 2926.

In addition, argenx announces the listing of and the commencement of dealings in its 2,581,633 new ordinary shares on the regulated market of Euronext Brussels.

This press release is for information purposes only and does not constitute, and should not be construed as, an offer to sell or the solicitation of an offer to buy or subscribe to any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale is not permitted or to any person or entity to whom it is

unlawful to make such offer, solicitation or sale. Reference is also made to the restrictions set out in "Important information" below. This press release is not for publication or distribution, directly or indirectly, in or into any state or jurisdiction into which doing so would be unlawful or where a prior registration or approval is required for such purpose.

On July 24, 2023-Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE:4151, President and CEO: Masashi Miyamoto) reported that the company has filed an application for partial change of approved indication of the sustained duration form of G-CSF(Granulocyte-Colony Stimulating Factor)*1 product G-Lasta [KRN125, generic name: pegfilgrastim (genetical recombination)] for the mobilization of hematopoietic stem cells into peripheral blood in autologous blood stem cell transplantation*2 (the "indication") in Japan today (Press release, Kyowa Hakko Kirin, JUL 24, 2023, View Source [SID1234633392]).

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This application is based on the results of a clinical trial conducted by Kyowa Kirin to evaluate the effect of G-Lasta on the mobilization of hematopoietic stem cells into peripheral blood in patients with multiple myeloma and malignant lymphoma.

G-Lasta is a long-acting G-CSF preparation licensed from Amgen K-A and has been marketed in Japan since 2014 for decreasing the incidence of febrile neutropenia*3 in patients receiving cancer chemotherapy. In February 2022, Kyowa Kirin received an approval for the indication of G-Lasta for the mobilization of hematopoietic stem cells into peripheral blood in allogeneic blood stem cell transplantation*4. By adding the indication of this sustained duration preparation to autologous blood stem cell transplantation, Kyowa Kirin expects to contribute to reducing burden on patients in hematopoietic stem cell transplantation therapy.

"We are very delighted with this application to add the indication to autologous blood stem cell transplantation therapy," said Yuichi Kawasaki, Executive Officer, Director of Product Strategy Department of Strategy Division at Kyowa Kirin. "

We will continue to strive to deliver this product to patients and provide new value to the blood stem cell transplantation therapy area." The Kyowa Kirin Group companies strive to contribute to the health and well-being of people around the world by creating new value through the pursuit of advances in life sciences and technologies.

On July 24, 2023 VBI Vaccines Inc. (NASDAQ: VBIV) (VBI or the Company), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that the underwriters of its recent underwritten public offering of common shares and accompanying common warrants to purchase common shares partially exercised their option to purchase an additional 1,536,363 common shares at a public offering price of $1.64 per common share (Press release, VBI Vaccines, JUL 24, 2023, View Source [SID1234633391]). The aggregate gross proceeds from this exercise were approximately $2.5 million, resulting in total gross proceeds of $23.5 million from the underwritten public offering and the previously completed concurrent registered direct offering, before deducting the underwriting discounts, commissions, and estimated offering expenses. The partial option exercise closed on July 21, 2023.

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Immediately following the closing of the partial option exercise, the number of outstanding common shares of the Company is 22,872,175.

Raymond James & Associates, Inc. acted as the sole book-running manager for the underwritten public offering. Newbridge Securities Corporation acted as the lead manager for the underwritten public offering. The registered direct offering was made without an underwriter or a placement agent.

VBI intends to use the net proceeds from the underwritten offering, including the partial option exercise, for the commercialization activities for PreHevbrio [Hepatitis B Vaccine (Recombinant)] in the United States, Europe, and Canada; manufacturing of PreHevbrio and clinical materials for its pipeline programs; and ongoing activities related to its development stage candidates, including VBI-1901 (glioblastoma) and VBI-2901 (coronaviruses). The net proceeds will also be used for general corporate purposes, including working capital and capital expenditures.

A shelf registration statement on Form S-3 (File No. 333-267109) relating to these securities was previously filed with the Securities and Exchange Commission ("SEC") on August 26, 2022 and declared effective on September 6, 2022. A final prospectus supplement and accompanying prospectus relating to the underwritten offering were filed with the SEC and available on the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and accompanying prospectus may be obtained from Raymond James & Associates, Inc., Attention: Equity Syndicate, 880 Carillon Parkway, St. Petersburg, Florida 33716, by telephone at (800) 248-8863, or by e-mail at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction. Any offer, if at all, will be made only by means of the prospectus supplement and accompanying prospectus forming a part of the effective registration statement.