On December 1, 2023 Kazia Therapeutics Limited (NASDAQ: KZIA) ("Kazia" or the "Company"), an oncology-focused drug development company, reported that it has entered into a definitive agreement for the purchase and sale of up to an aggregate of 4,444,445 of the Company’s American Depositary Shares ("ADSs") (or ADS equivalents in lieu thereof), each ADS representing ten (10) ordinary shares of the Company, at a purchase price of $0.45 per ADS (or ADS equivalent in lieu thereof), in a registered direct offering (Press release, Kazia Therapeutics, DEC 1, 2023, View Source [SID1234638098]). The Company has also agreed to issue in a concurrent private placement unregistered warrants to purchase up to an aggregate of 4,444,445 ADSs. The warrants will have an exercise price of $0.583 per ADS, will be immediately exercisable upon issuance, and will expire five and one-half years from the date of issuance. The closing of the offering is expected to occur on or about December 5, 2023, subject to the satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The gross proceeds to the Company from the offering are expected to be approximately $2 million, before deducting the placement agent’s fees and other offering expenses payable by the Company. The Company intends to use the net proceeds from this offering as working capital for general corporate purposes.

The securities described above (excluding the warrants and ADSs underlying the warrants) are being offered and sold by the Company in a registered direct offering pursuant to a "shelf" registration statement on Form F-3 (File No. 333-259224) that was originally filed with the Securities and Exchange Commission (the "SEC") on September 1, 2021, and declared effective on September 8, 2021. The offering of such securities in the registered direct offering is being made only by means of a prospectus supplement that forms a part of the effective registration statement. A final prospectus supplement and the accompanying base prospectus relating to the registered direct offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Electronic copies of the final prospectus supplement and the accompanying base prospectus may also be obtained, when available, from H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (212) 856-5711 or e-mail at [email protected].

The unregistered warrants described above are being offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act"), and Regulation D promulgated thereunder and, along with the ADSs representing ordinary shares underlying such warrants, have not been registered under the Act, or applicable state securities laws. Accordingly, the warrants and the underlying ADSs may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of the securities described herein, 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 other jurisdiction.

On December 1, 2023 280Bio, Inc., a clinical stage biotechnology company focused on the development of precision oncology medicines, reported that the first patient was treated with TEB-17231 (YL-17231), a small molecule inhibitor of RAS signaling (Press release, 280Bio, DEC 1, 2023, View Source [SID1234638097]). 280Bio is a wholly owned subsidiary of Shanghai Yingli Pharmaceuticals, Inc. (Yingli Pharma) of Shanghai, China. KRAS, NRAS and HRAS genes are frequently mutated in many tumor indications causally associated with promoting tumor cell growth. TEB-17231 demonstrated potent inhibition of tumor cell proliferation in vitro and in vivo in preclinical research, exhibiting activity with different KRAS, NRAS and HRAS alterations, including tumors that have become resistant to KRAS G12C inhibitors.

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"We are gratified that the first patient has been dosed with our novel pan-RAS inhibitor, marking a major step in TEB-17231’s clinical development, and introducing new medicines for the patients we hope to serve." said Michael Hui, Chief Executive Officer of 280Bio Inc., "This milestone highlights our commitment to addressing unmet needs in cancers through precision oncology approaches. We look forward to continued enrollment and dosing of patients in this trial."

This first US patient was enrolled at The University of Texas MD Anderson Cancer Center, Houston, Texas, by David Hong, MD, Deputy Chair of Investigational Cancer Therapeutics and lead investigator on the trial.

Reflecting on the high unmet need for new cancer agents, Dr. Zusheng Xu, Head of Research and Development of Yingli Pharma, said "TEB-17231 was developed to have a broader activity against the oncogenic RAS signaling pathways, than is currently demonstrated for many of the KRAS inhibitors. With its excellent pharmacologic and in vivo anti-tumor properties, we are hopeful that TEB-17231 will also be determined to be safe and efficacious by continuous oral dosing in patients."

On December 1, 2023 Veracyte, Inc. (Nasdaq: VCYT) reported that new data presented today at the 24th Annual Meeting of the Society of Urologic Oncology (SUO 2023) highlight clinical observations and translational research conducted by users of the company’s Decipher Prostate Genomic Classifier (Press release, Veracyte, DEC 1, 2023, View Source [SID1234638096]). These independent, real-world studies provide further evidence supporting the clinical utility of the test in the treatment and management of prostate cancer, particularly for patients considering active surveillance.

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Not all types of prostate cancer require immediate intervention; many can be monitored carefully over time with active surveillance, while more aggressive cancers have better outcomes with treatment. Identifying the appropriate candidates for active surveillance, however, is not always easy, as the clinical indicators used to assess patient risk often do not reliably distinguish between favorable and more aggressive disease.

Researchers at the Washington University School of Medicine in St. Louis presented two posters at the SUO meeting based on separate analyses of their Decipher biopsy cohort of 888 patients with prostate cancer who were seen at their institution between December 2016 and March 2023. In the first study, the researchers analyzed 235 patients who were initially managed with active surveillance, among whom 88 patients ultimately progressed to receive treatment. In evaluating clinical information available prior to treatment, including PSA levels, Gleason score, and results from magnetic resonance imaging (MRI), along with Decipher Prostate Genomic Classifier results, they found that the Decipher Prostate Genomic Classifier score was the only predictor of progression from active surveillance to treatment.

The second study focused on 104 patients who had a multiparametric MRI (mpMRI) result, Gleason Grade Groups 1-3 on biopsy, and Decipher testing prior to treatment with radical prostatectomy (surgery to remove the prostate). The researchers compared all of the available information prior to treatment to determine the best predictor of final pathology after prostatectomy. While multiparametric MRI (mpMRI) is often viewed as prognostic of cancer pathology, this study found that Decipher scores were strongly associated with final pathology, but clinical variables such as PSA levels, Gleason score and mpMRI were not. While mpMRI is often viewed as prognostic of cancer pathology, this study found that only Decipher scores were strongly associated with final pathology, while clinical variables such as PSA levels, Gleason score and mpMRI results were not.

"Accurately predicting disease progression in patients with prostate cancer is essential so they can receive the treatment that is best suited for them," said Eric Kim, M.D., associate professor of surgery at Washington University School of Medicine in St. Louis and senior author of both posters. "Our studies consistently demonstrated that data from the Decipher Prostate Genomic Classifier was more reliably predictive of prostate cancer progression and underlying pathology than any other clinical data available, including MRI."

Two other studies came from researchers at the University of Miami who used the ongoing Miami MAST prospective trial (MRI selection for active surveillance versus treatment; NCT02242773) to better understand cancer progression at the molecular level. In one study, researchers characterized 224 samples from 124 patients based on cancer progression. Assessment with the Decipher Prostate Genomic Classifier and biological insights from the Decipher Genomic Resource for Intelligent Discovery (GRID) research tool indicated that Decipher scoring was associated with grade progression, but not volume progression, of prostate cancer.

Finally, a large-scale, population-based study of over 52,000 patients tested with the Decipher Prostate biopsy test, led by investigators from the University of Michigan, found wide variation in Decipher risk distribution within and across clinical stage groups, suggesting that genomic assessment identified something that the clinical information does not. From this population-based initiative they concluded that integration of the test into clinical care can provide more precise prognosis estimates for patients and lead to better individualized risk assessment.

"We developed the Decipher Prostate Genomic Classifier to provide actionable information that helps guide patient care," said Elai Davicioni, Ph.D., Veracyte’s medical director for Urology. "The data presented at SUO 2023 show how users of our test, conducting prospective research on their own patients, affirm the utility of Decipher in real-world settings. This real-world evidence and scientific research conducted by Decipher users shows how physicians can refine their surveillance or treatment plans based on a more comprehensive view of each patient’s cancer. We are very grateful for the work conducted by these clinician-scientists on behalf of the entire prostate cancer research community."

On December 1, 2023 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported that an article describing the potential use of HyBryte in the treatment of cutaneous T-cell lymphoma (CTCL) has been published in Frontiers in Drug Discovery (Press release, Soligenix, DEC 1, 2023, View Source [SID1234638095]). The mini-review summarizes findings about the use, mechanism and effectiveness of HyBryte in the treatment of CTCL with a particular emphasis on the ability of HyBryte to address the unmet medical need in patients with early-stage disease.

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"With its chronic course and major impact on patient quality of life, CTCL is an orphan disease in urgent need of additional treatment options that are well-tolerated and safe over the long term," stated Brian Poligone, MD, PhD, Director of the Rochester Skin Lymphoma Medical Group, Fairport, NY. "Clinical studies with HyBryte have demonstrated its safety and effectiveness, with broad applicability across different lesion types, different skin tones and different disease stages. I know I can speak for my colleagues that have been involved with these studies when I say that the data generated to date has been extremely compelling."

"In treating CTCL, which is a chronic cancer with no cure, long-term safety is a strong driver of treatment choice. Most current treatment options for CTCL are associated with significant safety concerns, including black-box warnings. HyBryte treatment has demonstrated strong and rapid efficacy with a very benign safety profile," stated Richard Straube, MD, Senior Vice President and Chief Medical Officer of Soligenix. "Studies to date have indicated a substantial increase in efficacy with longer treatment with similar performance against both patch and plaque lesions. These results are derived from one of the largest studies ever conducted in CTCL and we believe HyBryte will be of significant benefit to patients living with this difficult disease."

About HyBryte

HyBryte (research name SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light approximately 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).The published Phase 3 FLASH trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a positive treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte’s mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With very limited systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc. In addition, the FDA awarded an Orphan Products Development grant to support the evaluation of HyBryte for expanded treatment in patients with early-stage CTCL, including in the home use setting. The grant, totaling $2.6 million over 4 years, was awarded to the University of Pennsylvania that was a leading enroller in the Phase 3 FLASH study.

About Cutaneous T-Cell Lymphoma (CTCL)

CTCL is a class of non-Hodgkin’s lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin where they form various lesions, typically beginning as patches and may progress to raised plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas.

CTCL constitutes a rare group of NHLs, occurring in about 4% of the approximate 700,000 individuals living with the disease. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects over 25,000 individuals in the U.S., with approximately 3,000 new cases seen annually.

On December 1, 2023 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported the publication in Blood of an abstract providing initial clinical data from the first patient treated at the lowest dose in the ongoing ARDENT Phase 1 clinical trial with SC291, a hypoimmune (HIP)-modified allogeneic CD19-directed CAR T cell therapy (Press release, Sana Biotechnology, DEC 1, 2023, View Source [SID1234638094]). SC291 appeared safe and well tolerated, evaded immune detection, and induced a partial response in a patient with chronic lymphocytic leukemia (CLL). ARDENT is a Phase 1 study evaluating safety and tolerability of SC291 in patients with CLL and non-Hodgkin lymphoma. Treatment in this dose escalation study is ongoing, and the company expects to present more data from this study at a later date in an appropriate venue.

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"These are the first clinical data demonstrating that our HIP technology can engineer allogeneic cells to evade adaptive and innate immune detection and rejection in the context of an intact immune system, overcoming the key challenge in unlocking the potential of allogeneic cells," said Gary Meininger, MD, Sana’s Chief Medical Officer. "These data suggest the potential of SC291 to persist and attack cancer cells in a manner consistent with autologous cells, which combined with scaled manufacturing, encourage us about both the opportunity for SC291 and our other HIP-modified cells to provide clinical benefit for patients. The data were published as part of an abstract submitted over the summer, and we look forward to sharing more data from this ongoing clinical trial that we expect will more clearly outline SC291’s profile."

The full abstract is available for online viewing at View Source

About SC291 in B-cell Lymphomas or Leukemias
SC291 is a hypoimmune, CD19-directed allogeneic CAR T cell therapy derived from healthy donor CD4+ and CD8+ T cells that are genetically engineered. SC291 is developed with Sana’s hypoimmune platform, which is designed to overcome the immunologic rejection of allogeneic cells, which if true for SC291 may result in longer CAR T cell persistence and a higher rate of durable complete responses for patients with B-cell lymphomas or leukemias. The hypoimmune technology includes disruption of major histocompatibility (MHC) class I and MHC class II expression to allow cells to evade the adaptive immune system, which includes antibody and T cell responses, as well as overexpression of CD47 to evade the innate immune cell system, in particular macrophages and natural killer (NK) cells. The company has presented data across multiple preclinical models highlighting the potential of this platform to cloak cells from immune recognition and the potential of SC291 as a therapeutic for patients with B-cell malignancies. SC291 is being evaluated in a Phase 1 study called ARDENT for patients with chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphoma (NHL).

About Hypoimmune Platform
Sana’s hypoimmune platform is designed to create cells ex vivo that can evade the patient’s immune system to enable the transplant of allogeneic cells without the need for immunosuppression. We are applying the hypoimmune technology to both donor-derived allogeneic T cells, with the goal of making potent and persistent CAR T cells at scale, and pluripotent stem cells, which can then be differentiated into multiple cell types at scale. Preclinical data published in peer-reviewed journals demonstrate across a variety of cell types that these transplanted allogeneic cells are able to evade both the innate and adaptive arms of the immune system while retaining their activity. Our most advanced programs utilizing this platform include an allogeneic CAR T program targeting CD19+ cancers, an allogeneic CAR T program for B-cell mediated autoimmune diseases, an allogeneic CAR T program targeting CD22+ cancers, and stem-cell derived pancreatic islet cells for patients with type 1 diabetes.