On December 18, 2023 Clarity Pharmaceuticals (ASX: CU6) ("Clarity", "the Company"), a clinical stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for children and adults with cancer, reported the early recruitment closure for its Phase II diagnostic 64Cu-SARTATE trial, DISCO (NCT04438304)1, for patients with known or suspected neuroendocrine tumours (NETs) (Press release, Clarity Pharmaceuticals, DEC 18, 2023, View Source [SID1234638642]). A total of 45 patients have been enrolled in the trial and all of the participants have now been administered and imaged with 64Cu-SARTATE. All trial participants have now progressed to or have completed the follow-up stage.

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The trial was originally planned for up to 63 patients based on an expected discordance level between imaging with Clarity’s 64Cu-SARTATE and the current standard of care, 68Ga-DOTATATE. The sample size was adjusted to 45 patients based on the pre-planned assessment of the images to generate sufficient evidence to plan for a Phase III trial in this indication, enabling recruitment to successfully close early.

DISCO, which derives from "Diagnostic Imaging Study of 64COpper-SARTATE Using PET on Patients with Known or Suspected Neuroendocrine Tumours", is assessing the performance of Clarity’s SARTATE imaging product as a potential new way to help diagnose and manage NETs. The DISCO trial recruited participants with Gastroenteropancreatic NETs (GEP-NETs) across four sites in Australia, comparing the diagnostic performance of 64Cu-SARTATE at 4 and 20 hours post-administration to 68Ga-DOTATATE at one hour.

The trial aims to build on earlier work with SARTATE2 which demonstrated that imaging at later time points, enabled by a longer half-life of 64Cu in comparison to 68Ga, may lead to better identification of disease.

Clarity’s Executive Chairperson, Dr Alan Taylor, commented, "We are very pleased with the progress of our SARTATE product and the DISCO trial. Whilst the main focus for this product is on neuroblastoma in children, for both therapy and diagnosis, which includes two Rare Pediatric Disease Designations and two Orphan Drug Designations in this indication, the initial imaging data looks highly encouraging in NETs. We are optimistic the final trial results will underscore the diagnostic capability of 64Cu-SARTATE for patients with NETs, and we eagerly anticipate the comprehensive analysis from the DISCO trial.

"Unfortunately, misdiagnosis and delays in diagnosis are prevalent in this patient population, but we believe that SARTATE has the potential to play a vital role in improving NETs diagnosis and treatment outcomes for these patients. We are confident the clinical advantages of SARTATE, combined with the logistical benefits of Targeted Copper Theranostics (TCTs), will ensure timely and convenient diagnosis and access to critical treatments for cancer patients.

"The Clarity TCT platform comprises of products not only focused on NETs, but also on other malignancies with high unmet need. Regarding our diagnostic portfolio, we were pleased to recently announce the recruitment closure of one of our studies in Gastrin Releasing Peptide Receptor (GRPR)-positive biochemically recurrent prostate cancer using SAR-Bombesin (SABRE, NCT05407311)3,4 and the activation of the first site for our Phase III registrational study in pre-prostatectomy patients with SAR-bisPSMA (CLARIFY, NCT06056830)5,6. We are also eagerly awaiting to receive the data and initial analysis of the results from the COBRA study (biochemically recurrent prostate cancer using 64Cu-SAR-bisPSMA, NCT05249127)7 from our partnering contract research organisation, and at this stage we expect to share these results in early 2024".

About SARTATE
SARTATE is a next generation, highly targeted theranostic radiopharmaceutical. It is being developed for diagnosing, staging and subsequently treating cancers that express somatostatin receptor 2 (SSTR2), including neuroblastoma and neuroendocrine tumours (NETs). Like all Clarity products, the SARTATE product can be used with copper-64 (64Cu) for imaging (64Cu-SARTATE) or copper-67 (67Cu) for therapy (67Cu-SARTATE).

64Cu-SARTATE and 67Cu-SARTATE are unregistered products. Individual results may not represent the overall safety and efficacy of the products. The data outlined in this announcement has not been assessed by health authorities such as the US Food and Drug Administration (FDA). A clinical development program is currently underway to assess the efficacy and safety of these products. There is no guarantee that these products will become commercially available.

About NETs
NETs, also known as well-differentiated neuroendocrine neoplasms or carcinoids, represent a heterogeneous group of malignant transformations of cells of the diffuse neuroendocrine system8. They most commonly occur in the gastrointestinal tract (48%), lung (25%), and pancreas (9%), but may also originate in other areas, including the breast, prostate, thymus and skin9. NETs can either be benign or malignant, as well as non-functional and functional10. NETs traditionally have been considered uncommon; however, the incidence has been increasing as a worldwide phenomenon11. This increase is thought to be mostly related to improvements in the way NETs are diagnosed, including better imaging tests and endoscopy, and increased awareness of these tumours12.

Overall, it is estimated that more than 12,000 people in the United States are diagnosed with a NET each year, and approximately 175,000 people are living with this diagnosis12. Patients with GEP-NETs present with subtle clinical symptoms, which can lead to a delay in diagnosis of up to 5–7 years or result in inappropriate management13. As such, about 30-75% of NET patients have distant metastases at the time of diagnosis14. A 10-year relative survival rate for patients with metastatic GEP-NETs is 3–36%.

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."

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 [email protected]; 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 [email protected] . 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.

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.

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.