U.S. Food and Drug Administration Issues Complete Response Letter for Cosibelimab Solely Due to Inspection Findings at Third-Party Manufacturer

On December 18, 2023 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), reported that the U.S. Food and Drug Administration ("FDA") has issued a complete response letter ("CRL") for the cosibelimab biologic license application ("BLA") for the treatment of patients with metastatic or locally advanced cutaneous squamous cell carcinoma ("cSCC") who are not candidates for curative surgery or radiation (Press release, Checkpoint Therapeutics, DEC 18, 2023, View Source [SID1234638640]). The CRL only cites findings that arose during a multi-sponsor inspection of Checkpoint’s third-party contract manufacturing organization as approvability issues to address in a resubmission.

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The CRL did not state any concerns about the clinical data package, safety, or labeling for the approvability of cosibelimab.

"As the only deficiencies relate to the FDA’s inspection of our third-party contract manufacturing organization, we believe we can address the feedback in a resubmission to enable marketing approval in 2024," said James Oliviero, President and Chief Executive Officer of Checkpoint. "We are committed to working closely with our third-party manufacturer and the FDA on our resubmission in order to make cosibelimab available to patients living with cSCC."

bluebird bio, Inc. Announces Proposed Public Offering of Common Stock

On December 18, 2023 bluebird bio, Inc. (Nasdaq: BLUE) ("bluebird") reported that it has commenced an underwritten public offering of $150,000,000 of shares of its common stock (Press release, bluebird bio, DEC 18, 2023, View Source [SID1234638638]). bluebird also intends to grant the underwriters a 30-day option to purchase up to an additional $22,500,000 of shares of its common stock to be sold in the offering. The offering, actual size and terms are subject to market conditions, and there can be no assurance as to whether or when the offering may be completed. All shares in the offering are to be sold by bluebird.

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Goldman Sachs & Co. LLC and J.P. Morgan Securities LLC are acting as joint book running managers for the offering. Raymond James & Associates, Inc. is acting as co-manager for the offering.

bluebird intends to use the net proceeds of the offering (i) to support commercialization and manufacturing for its three approved gene therapies, ZYNTEGLO, SKYSONA and LYFGENIA; and (ii) to fund working capital and other general corporate purposes.

The offering is being made pursuant to an effective shelf registration statement on Form S-3, including a prospectus, that was filed with the U.S. Securities and Exchange Commission (the "SEC") on May 9, 2023 and was declared effective by the SEC on May 19, 2023. A preliminary prospectus supplement describing the terms of the offering will be filed with the SEC and will form a part of the effective registration statement. Copies of the preliminary prospectus supplement and accompanying prospectus relating to the public offering may be obtained, when available, by contacting Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by telephone at (866) 471-2526 or by email at prospectus-ny@ny.email.gs.com; or J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204 or by email at prospectus-eq_fi@jpmorganchase.com . Before you invest, you should read the preliminary prospectus supplement and accompanying prospectus and the other documents that bluebird has filed with the SEC for more complete information about bluebird and the proposed offering.

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 the registration or qualification under the securities laws of any such state or jurisdiction.

Aulos Bioscience Advances AU-007 to Phase 2 Portion of Phase 1/2 Study Evaluating Computationally Designed IL-2 Antibody for Treatment of Solid Tumors

On December 18, 2023 Aulos Bioscience, an immuno-oncology company working to revolutionize cancer care through the development of best-in-class IL-2 therapeutics, reported plans to initiate the Phase 2 portion of its Phase 1/2 clinical trial of AU-007 in patients with unresectable locally advanced or metastatic solid tumor cancers of the skin and kidney (Press release, Aulos Bioscience, DEC 18, 2023, View Source [SID1234638637]). AU-007 is a human IgG1 monoclonal antibody designed using artificial intelligence to harness the power of interleukin-2 (IL-2) to eradicate solid tumors.

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"We are very pleased with the safety profile of AU-007 when paired with low-dose Proleukin based on clinical results to date, and we are thrilled to take the AU-007 program to the next stage in its clinical development," said Aron Knickerbocker, Aulos Bioscience’s chief executive officer. "Since our last data cutoff date of October 13, 2023 (data presented at SITC (Free SITC Whitepaper)), new data indicate clinical activity in several patients, including disease control and objective response results. This is encouraging as the anti-tumor activity has been observed in heavily pre-treated patients whose tumors had progressed through checkpoint inhibitors. We look forward to the Phase 2 portion of this study and presenting clinical data from the expansion cohorts next year."

Aulos’ Phase 1/2 clinical trial is a two-part, open label, first-in-human study evaluating the safety, tolerability, immunogenicity and clinical activity of AU-007 in patients with unresectable locally advanced or metastatic cancer. The trial is currently enrolling patients at multiple locations in the United States and Australia.

The first Phase 2 expansion cohorts will focus on melanoma and renal cell carcinoma (RCC). These Phase 2 cohorts will evaluate AU-007 in combination with a single loading dose of low-dose, subcutaneous Proleukin (aldesleukin; recombinant human IL-2), or with AU-007 and low-dose, subcutaneous Proleukin administered every two weeks.

Clinical data from the Phase 1/2 study were presented at the 38th Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November. The Phase 1 dose escalation cohort data indicated that AU-007 is well tolerated as a monotherapy treatment or in a combination therapy regimen with low-dose, subcutaneous Proleukin, with no pulmonary or generalized edema, vasculature leakage or dose-limiting toxicity to date. Additionally, tumor shrinkage was observed in patients with melanoma, bladder, kidney and lung cancers.

Created by Biolojic Design, AU-007 is the first computationally designed monoclonal antibody to be tested in a clinical trial. The antibody was designed using artificial intelligence and has a mechanism of action that is completely unique in the IL-2 therapeutic space. AU-007 has the ability to bind precisely to the portion of IL-2 that binds to CD25, which prevents IL-2 from binding to high-affinity IL-2 receptors on regulatory T cells (Tregs), vasculature, pulmonary tissue and eosinophils. This allows IL-2 to redirect to medium-affinity receptors on effector T cells (Teffs) and natural killer (NK) cells, which expand and kill tumor cells.

To learn more about the AU-007 clinical trial program, please visit ClinicalTrials.gov (identifier: NCT05267626). For patients and providers in the U.S., please visit www.solidtumorstudy.com. For patients and health professionals in Australia, please visit www.solidtumourstudy.com.

About AU-007
AU-007 is a computationally designed, human IgG1 monoclonal antibody that is highly selective to the CD25-binding portion of IL-2. With a mechanism of action unlike any other IL-2 therapeutic in development, AU-007 leverages IL-2 to reinforce anti-tumor immune effects. This is achieved by preventing IL-2, either exogenous or secreted by effector T cells, from binding to trimeric receptors on regulatory T cells while still allowing IL-2 to bind and expand effector T cells and NK cells. This prevents the negative feedback loop caused by other IL-2-based treatments and biases the immune system toward activation over suppression. AU-007 also prevents IL-2 from binding to CD25-containing receptors on eosinophils, as well as vasculature and pulmonary endothelium, which may significantly reduce the vascular leak syndrome and pulmonary edema associated with high-dose IL-2 therapy.

Abbott to Present at J.P. Morgan Healthcare Conference

On December 18, 2023 Abbott (NYSE: ABT) reported that it will present at the 42nd annual J.P. Morgan Healthcare Conference on Tuesday, Jan. 9, 2024 (Press release, Abbott, DEC 18, 2023, View Source [SID1234638636]). Robert B. Ford, chairman and chief executive officer, will present at 11 a.m. Central time.

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A live webcast of the presentation will be accessible through Abbott’s Investor Relations website at www.abbottinvestor.com.

EISAI SUBMITS MARKETING AUTHORIZATION APPLICATION IN JAPAN FOR ANTICANCER AGENT TASURGRATINIB FOR BILIARY TRACT CANCER WITH FGFR2 GENE FUSION

On December 18, 2023 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that it has submitted a marketing authorization application in Japan for its in-house discovered fibroblast growth factor (FGF) receptor (FGFR1, FGFR2, FGFR3) selective tyrosine kinase inhibitor tasurgratinib succinate (generic name, development code: E7090, "tasurgratinib") for biliary tract cancer with FGFR2 gene fusion (Press release, Eisai, DEC 18, 2023, View Source [SID1234638620]). In Japan, tasurgratinib has received orphan drug designation for a prospective indication for unresectable biliary tract cancer with FGFR2 gene fusion by the Ministry of Health, Labour and Welfare, (MHLW). Under this system, this application will be subject to priority review.

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This application is based on the results of a multicenter, open-label, single-arm clinical phase II trial (Study 201) in Japan and China conducted by Eisai. Study 201 enrolled patients with unresectable biliary tract cancer with FGFR2 gene fusion previously treated with gemcitabine-based combination chemotherapy. The primary endpoint of this study was objective response rate, and secondary endpoints included safety. Detailed results of the study will be presented at upcoming academic conferences.

The estimated number of patients with biliary tract cancer is approximately 25,000 in Japan1, 2 and the five-year survival rate for the cancer is approximately 25%, which makes it an intractable cancer with the second worst prognosis following pancreatic cancer.1 Drug therapy options are limited in comparison with other cancers, and as such it is a disease with significant unmet medical needs. FGFR2 gene fusion is observed in approximately 14% of intrahepatic cholangiocarcinoma, which account for 15-30% of biliary tract cancers.3 FGFR genetic aberrations such as the gene fusions are known to be deeply involved in the proliferation, survival and migration of cancer cells as well as tumor angiogenesis and drug resistance. As these genetic aberrations in FGFRs have been observed in various other types of cancers including biliary tract cancer, there is growing interest in FGFRs as a promising target for cancer therapy. By selectively inhibiting FGFR1, 2 and 3, and blocking those signals, tasurgratinib has been expected to become a new molecular targeted therapy for cancers with FGFR genetic aberrations.4

Eisai acknowledges "Oncology" as one of its key strategic areas, and will continue to focus on the discovery and development of anti-cancer drugs within drug discovery domains including "tumor microenvironment", "proteostasis disruption", "cell linage and cell differentiation", and "inflammation, hypoxia, oxidative stress and cell senescence" under the Deep Human Biology Learning (DHBL) drug discovery and development organization. Eisai aspires to discover innovative new drugs with new targets and mechanisms of action from these domains with the aim of contributing to the cure of cancers.