Ewing Sarcoma

Dr. Yael Mossé receives recognition for project dedicated to researching and potentially developing new therapies for undruggable solid tumors PHILADELPHIA, March 6, 2024 /PRNewswire/ -- An interdisciplinary, global team of scientists led by Yael Mossé, MD, Professor of Pediatrics in the Cancer Center at Children's Hospital of Philadelphia (CHOP), has been selected as one of five research teams from all over the world to receive funds from Cancer Grand Challenges, a funding initiative co-founded by the National Cancer Institute and Cancer Research UK. Mossé's international team will receive up to $25 million to develop transformative new therapies for previously undruggable forms of childhood cancer. Continue Reading International team led by Dr. Yael Mossé, Professor of Pediatrics in the Cancer Center at Children's Hospital of Philadelphia, will receive up to $25 million to develop transformative new therapies for previously undruggable forms of childhood cancer. Last year, 176 global teams submitted bold ideas for tackling some of the most pressing challenges in cancer research and care. Twelve teams were shortlisted, and the project "Developing a suite of oncoprotein degraders for childhood solid cancers," nicknamed Team KOODAC3, emerged as one of five teams to receive funding after a year-long rigorous selection process. The team unites the expertise of researchers from the United States, Austria, France, Germany and the United Kingdom to tackle the challenge of oncogene-addicted solid tumors in children. While survival rates for many childhood cancers have improved dramatically through the combination of standard chemotherapy and radiation therapy, survivors are often burdened with life threatening toxicities related to those therapies. Genetic discoveries related to cancer have transformed how many types of tumors are treated. Cancer is caused by genetic mutations that transform normal genes into oncogenes. These oncogenes are then translated into proteins that are not typically found in the cell, known as oncoproteins. Several drugs have been developed to treat different tumors by targeting oncogenes and their associated oncoproteins. Despite advances in this field, a critical challenge has been the difficulty of targeting proteins responsible for causing solid tumors related to childhood cancers. As a result, cure rates for most solid tumors have not improved at the rate of other types of cancer. KOODAC3 aims to develop drugs that break down or 'degrade' five of the most significant oncoproteins in children with high-risk oncogene-driven cancers, including neuroblastoma, medulloblastoma, Ewing sarcoma, fibrolamellar hepatocellular carcinoma, rhabdomyosarcoma and other cancers that deregulate these essential oncoproteins. The team is addressing this challenge by extending an ongoing collaboration with Nurix Therapeutics, a leading biopharmaceutical company focused on degrader drugs, providing a streamlined route for clinical translation of discoveries made by Team KOODAC3. Collectively, this consortium has the ambitious goal of developing oral drugs that will be deployed world-wide in partnership with a robust international patient advocacy committee that will inform outreach to childhood cancer communities globally. "Our team is deeply honored to have been selected as one of the five to receive funding from the Cancer Grand Challenges initiative," said Mossé. "Team KOODAC3 was brought together with the goal of identifying new strategies for treating high-risk pediatric cancers with a high unmet need. Each of the projects that applied for this funding were worthy, so we do not take this award lightly and we are committed to bringing our expertise together with the goal of developing paradigm-altering safe and effective new therapies for currently lethal childhood solid cancers." Additional CHOP co-investigators who are part of Team KOODAC3 include John Maris, MD, and Adam Wolpaw, MD, PhD. "At CHOP, our researchers are committed to creating life-changing solutions for children around the world, and improving the care for some of the most difficult to treat childhood cancers is what drives so much of what we do," said Susan L. Furth, MD, PhD, Executive Vice President and Chief Scientific Officer at CHOP. "We are thrilled that this prestigious organization recognized the importance of what this project is trying to achieve, and we expect the funding to help us make important strides toward our understanding of childhood cancer." The National Cancer Institute and Cancer Research UK, the world's leading funders of cancer research, co-founded and launched the Cancer Grand Challenges initiative in 2020. Cancer Grand Challenges aims to provide funding for interdisciplinary research teams from around the world whose novel ideas offer the greatest potential to advance bold cancer research and improve outcomes for people affected by cancer. The team is co-led by Martin Eilers at the University of Würzburg in Germany and is funded by Cancer Research UK, Institut National Du Cancer and KiKa (Children Cancer Free Foundation) through Cancer Grand Challenges. Learn more about the Cancer Grand Challenges at . In addition to the Team KOODAC3 award, Team MATCHMAKERS, which includes Dr. Nikolaos G. Sgourakis from CHOP, also received a Cancer Grand Challenges award of up to $25 million. About Children's Hospital of Philadelphia: A non-profit, charitable organization, Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. The institution has a well-established history of providing advanced pediatric care close to home through its CHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout Pennsylvania and New Jersey, as well as the Middleman Family Pavilion and its dedicated pediatric emergency department in King of Prussia. In addition, its unique family-centered care and public service programs have brought Children's Hospital of Philadelphia recognition as a leading advocate for children and adolescents. For more information, visit . Contact: Jennifer Lee Children's Hospital of Philadelphia (610)800-6592 [email protected] SOURCE Children's Hospital of Philadelphia

Company implementing a series of cost-savings measures designed to extend Salarius’ expected cash runway into the first half of 2025 Extended runway supports the generation of additional data from the ongoing Phase 1/2 clinical trials in hematologic cancers and Ewing sarcoma HOUSTON, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biopharmaceutical company developing therapies for patients with cancer in need of new treatment options, today announced that its Board of Directors is implementing a series of additional cost-savings measures designed to extend Salarius’ expected cash runway into the first half of 2025. These measures will allow Salarius to support the generation of additional clinical data for seclidemstat in the ongoing MD Anderson Cancer Center (MDACC) investigator-initiated Phase 1/2 clinical trial in hematologic cancers and Salarius’ Phase 1/2 trial in Ewing sarcoma. Earlier this year Salarius announced that MDACC had resumed enrollment in the hematologic cancer trial, and based on the available data Salarius is encouraged by the 50% objective response rate (ORR) previously reported by MDACC researchers. Salarius also reported earlier this year that an additional Ewing sarcoma patient treated with a combination of seclidemstat, topotecan and cyclophosphamide (TC) achieved a partial response, increasing the ORR among first-relapse Ewing sarcoma patients to 60%. In connection with the cost-savings measures, David Arthur, the Company’s President and Chief Executive Officer, has ended his full-time employment and transitioned to a part-time consultant role, effective February 20, 2024. He will continue to serve as Chief Executive Officer and support Salarius’ ongoing activities. The cost-savings measures also include reducing operating expenses and reducing the cash compensation payable to the Company’s non-employee directors beginning in the second quarter of 2024. In August 2023 Salarius announced that it had retained Canaccord Genuity, LLC to lead a comprehensive review of strategic alternatives focusing on maximizing shareholder value. While these efforts are ongoing, the Company continues to support its clinical programs, as appropriate, and the cost-savings measures approved by the Board of Directors are designed to enable the Company to continue supporting such activities. “With these additional expense reductions, we are able to extend our cash runway to allow for the generation of additional clinical data in both seclidemstat clinical trials. The Board of Directors believes this decision is in the best interest of shareholders, and the additional data may enhance our opportunities to maximize shareholder value,” said Dr. William McVicar, Chair of the Board. “By further reducing expenses, we are able to support the ongoing clinical development of seclidemstat into the first half of 2025. We look forward to reviewing the updated clinical data from both trials later this year and to sharing those data with prospective strategic partners and other interested parties.” About SeclidemstatSeclidemstat is a novel oral reversible inhibitor of the LSD1 enzyme and has received fast track, orphan drug and rare pediatric disease designations for Ewing sarcoma from the FDA. In addition to the MDACC investigator-initiated trial, seclidemstat has been studied in a company-sponsored Phase 1/2 trial evaluating its use in combination with TC for the treatment of relapsed/refractory Ewing sarcoma. Researchers at MDACC previously reported interim clinical trial results evaluating seclidemstat in combination with azacitidine for the treatment of myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML) patients who relapsed or progressed after hypomethylating agent therapy. Of eight evaluable patients, four (50%) had an objective response. These researchers reported a 90% probability of survival for 11 months in patients receiving seclidemstat plus azacitidine. Typically, overall survival is four to six months after failing therapy with hypomethylating agents. The hematologic cancer Phase 1/2 clinical trial being conducted at the University of Texas MD Anderson Cancer Center is now listed as active and recruiting on clinical trials.gov – trial NCT04734990. The Company-sponsored Ewing sarcoma clinical trial focuses on seclidemstat in combination with topotecan and cyclophosphamide as a treatment for relapsed and refractory Ewing sarcoma. To date, a total of 13 relapsed Ewing sarcoma patients, including five patients with first relapse and eight patients with second relapse, have been enrolled at seclidemstat doses of 600 mg or 900 mg twice daily in combination with TC. The five first-relapse patients demonstrated a 60% ORR and a 60% disease control rate (DCR) including one complete response and two partial responses. Among the three patients achieving OR, the median progression-free survival (mPFS) has not been reached with these patients still alive and have disease control and objectives responses at 17.4, 25.7 and 27.2 months, and increasing, after starting seclidemstat + TC combination treatment.The eight second-relapse patients demonstrated a 13% ORR, a 25% DCR and a mPFS of 1.6 months (range: 0.0 months to 10.7 months).Together the 13 first- and second-relapse patients demonstrated a mPFS of 8.1 months (range: 2.0 months to 27.2 months). Five patients, or 38%, achieved confirmed disease control and progression has not been observed in any of these patients while on study. Salarius has completed FDA Type B End of Phase 2 (EOP2) meeting process for the Seclidemstat Ewing sarcoma development program and has amended the current clinical trial protocol to reflect guidance agreed to with FDA. There is currently one patient enrolled in the Ewing sarcoma clinical trial, who recently achieved a partial response defined by a 30% or greater reduction in their target lesions, and this patient is continuing treatment with seclidemstat plus TC therapy. The Ewing sarcoma trial is currently active but is not currently enrolling additional patients. About Salarius PharmaceuticalsSalarius Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing therapies for patients with cancer in need of new treatment options. Salarius’ product portfolio includes seclidemstat, Salarius’ lead candidate, which is being studied as a potential treatment for pediatric cancers, sarcomas and other cancers with limited treatment options, and SP-3164, an oral small molecule protein degrader being developed for the treatment of non-Hodgkin’s lymphoma. Salarius has received financial support from the National Pediatric Cancer Foundation to advance the Ewing sarcoma program and was a recipient of a Product Development Award from the Cancer Prevention and Research Institute of Texas (CPRIT). For more information, please visit salariuspharma.com or follow Salarius on Twitter and LinkedIn. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. These forward-looking statements may be identified by terms such as “will,” “believe,” “developing,” “expect,” “may,” “progress,” “potential,” “could,” “look forward,” “encouraging,” “might,” “should,” and similar terms or expressions or the negative thereof. Examples of such statements include, but are not limited to, statements relating to the following: Salarius’ expectations regarding the exploration of strategic alternatives, opportunities to extend Salarius’ resources, the Company’s expected cash runway, the Company’s expectations that the cost-savings measures will support the generation of additional data from the ongoing Phase 1/2 clinical trials in hematologic cancers and Ewing sarcoma; the future of the Company’s operations and product candidates; the future of the Company’s preclinical studies and clinical trials and development activities; the advantages of protein degraders including the value of SP-3164 as a cancer treatment; the value of seclidemstat as a treatment for Ewing sarcoma, Ewing-related sarcomas, and other cancers and its ability to improve the life of patients. Salarius may not actually achieve the plans, carry out the intentions or meet the expectations or objectives disclosed in these forward-looking statements. You should not place undue reliance on these forward-looking statements. These statements are subject to risks and uncertainties which could cause actual results and performance to differ materially from those discussed in the forward-looking statements. These risks and uncertainties include, but are not limited to, the following: the risk that exploration of strategic alternatives may not result in any definitive transaction or enhance stockholder value and may create a distraction or uncertainty that may adversely affect our operating results, business, or investor perceptions; the potential for the Company to seek other alternatives for restructuring and resolving its liabilities, including bankruptcy proceedings, a dissolution and orderly wind-down of operations; expectations regarding future costs and expenses; our product candidates being in early stages of development; the uncertainty about the paths of our programs and our ability to evaluate and identify a path forward for those programs, particularly given the constraints we have as a small company with limited financial, personnel and other operating resources (including with respect to the allocation of our limited capital and the sufficiency of our capital in the near term for any path we do select); Salarius’ ability to continue as a going concern; the sufficiency of Salarius’ capital resources; availability of suitable third parties with which to conduct contemplated strategic transactions; whether the Company will be able to pursue a strategic transaction, or whether any transaction, if pursued, will be completed successfully and on attractive terms or at all; whether our cash resources will be sufficient to fund the Company’s foreseeable and unforeseeable operating expenses and capital requirements; changes in the Company’s operating plans that may impact its cash expenditures; the uncertainties inherent in research and development, future clinical data and analysis; the risks associated with reductions in workforce, including reduced morale and attrition of additional employees necessary for the strategic reprioritization; the risk of not having a full-time chief executive officer; future clinical trial results and the impact of such results on Salarius; that the results of studies and clinical trials may not be predictive of future clinical trial results; the competitive landscape and other industry-related risks; and other risks described in Salarius’ filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2022, as revised or supplemented by its Quarterly Reports on Form 10-Q and other documents filed with the SEC. The forward-looking statements contained in this press release speak only as of the date of this press release and are based on management’s assumptions and estimates as of such date. Salarius disclaims any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. Contact: LHA Investor RelationsKim Sutton Golodetzkgolodetz@lhai.com212-838-3777

MELBOURNE, Australia, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) today announces it has entered into an agreement to acquire QSAM Biosciences, Inc. (U.S. OTC: QSAM) and its lead investigational drug Samarium-153-DOTMP (153Sm-DOTMP). QSAM is a United States (U.S.) based company developing therapeutic radiopharmaceuticals for primary and metastatic bone cancer. 153Sm-DOTMP is a novel kit-based bone-seeking targeted radiopharmaceutical candidate that uses a “next generation” chelating agent to deliver a proprietary formulation of Samarium-153 radioisotope. With two major potential applications – pain management of bone metastases and osteosarcoma therapy, including in paediatric patients – 153Sm-DOTMP is highly aligned with Telix’s existing therapeutic focus areas in urologic oncology (prostate cancer), neuro-oncology (glioma), and musculoskeletal oncology (sarcoma). 153Sm-DOTMP has shown in pre-clinical and early clinical trials evidence of safety, efficacy and future commercial utility, and has the potential to deliver significant improvements on prior bone-seeking agents in the treatment and management of late-stage metastatic disease. This includes a much-improved safety profile (myelotoxicity and renal toxicity), more targeted delivery (reduced off-target irradiation) and a streamlined production system (flexible supply chain and lower COGS).1 The potential commercialisation of this asset would build on a well-established reimbursement pathway for this class of drugs, offering a potential cost-effective and quality of life-enhancing alternative to standard-of-care (bisphosphonates, steroids, opioids). Recent pre-clinical and clinical data have yielded encouraging evidence for 153Sm-DOTMP to enable the pain management of prostate cancer bone metastases,2 where there remains a significant unmet patient need particularly after progression from other forms of radionuclide and radiation therapy. Telix believes that 153Sm-DOTMP may benefit patients with metastatic lung and breast cancer, where many patients develop brain and bone metastases, and disease management often focusses on quality-of-life palliative care. 153Sm-DOTMP has also been granted Orphan Drug3 and Rare Pediatric Disease4 Designations (ODD/RPDD) by the U.S. Food and Drug Administration (FDA) for the treatment of osteosarcoma. This designation broadens Telix’s portfolio to include potential treatment for a disease that mostly affects children and young adults, building upon Telix’s established focus in musculoskeletal oncology (the targeted alpha program – TLX300 / olaratumab). The RPDD designation may enable 153Sm-DOTMP to be brought to market more rapidly through regulatory incentives, including eligibility for a paediatric rare disease Priority Review Voucher (PRV) that may be applied to this or other Telix programs. Dr Christian Behrenbruch, Managing Director and Group CEO of Telix said, “The acquisition of QSAM provides Telix with an additional near-term therapeutic pipeline asset, further differentiating our innovation position in radiopharmaceuticals and building depth in Telix’s key disease focus areas of urological and musculoskeletal oncology. Samarium is a highly optimal radionuclide for treating bone metastases, and the combination of ODD and PRDD status with Telix’s demonstrated experience in pharmacy-based cold-kit distribution has strong potential for a rapid pathway to commercialisation of this asset.” Deal terms and conditions The purchase price comprises: US$33.1 million (AU$50.8 million5) upfront, which is payable in the form of 4,369,914 Telix ordinary shares to be issued at closing, subject to certain cash adjustments (at or after closing) which would be deducted off the upfront value, and Contingent Value Rights, or performance rights, that represent the right of the holders to receive contingent payments up to US$90 million (AU$138 million5) in the aggregate, which are payable in cash and/or in ordinary shares, upon achievement of certain clinical and commercial milestones. Telix will issue ordinary shares to the stockholders of QSAM at closing within its Listing Rule 7.1 placement capacity as consideration for the acquisition. The ordinary shares issued upfront will be subject to escrow conditions.6 The number of ordinary shares to be issued at closing will be determined based on a price per share, which represents the volume weighted average price at which the ordinary shares traded on the ASX over the 10 trading day period prior to signing. Execution of the acquisition agreement concludes an option period of exclusivity and to complete diligence following the signing of a Term Sheet for the proposed acquisition in November 2023.7 Completion of the transaction is subject to customary conditions, including approval of QSAM’s stockholders and regulatory approvals. Refer to Telix disclosures and Appendix 3B lodged with the ASX today for further information. About bone cancer In the U.S., there are over 400,000 new patients diagnosed each year with metastatic bone cancer and 350,000 patient deaths.8 The incidence of advanced malignant tumours with bone metastasis can be up to 70%, especially common in patients with advanced prostate and breast cancer.9 Osteosarcoma and Ewing’s sarcoma are the most common malignancies of the bones in children. About QSAM Biosciences, Inc. QSAM Biosciences, Inc. is developing next-generation nuclear medicines for the treatment of cancer and other diseases. QSAM’s initial technology, 153Sm-DOTMP, is a clinical-stage bone-targeting radiopharmaceutical originally developed by IsoTherapeutics Group LLC and now owned by IGL Pharma Inc. About Telix Pharmaceuticals Limited Telix is a biopharmaceutical company focused on the development and commercialisation of diagnostic and therapeutic radiopharmaceuticals and associated medical devices. Telix is headquartered in Melbourne, Australia with international operations in the United States, Europe (Belgium and Switzerland), and Japan. Telix is developing a portfolio of clinical-stage products that aims to address significant unmet medical needs in oncology and rare diseases. Telix is listed on the Australian Securities Exchange (ASX: TLX). Visit for further information about Telix, including details of the latest share price, announcements made to the ASX, investor and analyst presentations, news releases, event details and other publications that may be of interest. You can also follow Telix on X and LinkedIn. Telix’s lead imaging product, gallium-68 (68Ga) gozetotide injection (also known as 68Ga PSMA-11 and marketed under the brand name Illuccix®), has been approved by the FDA,10 by the Australian Therapeutic Goods Administration (TGA),11 and by Health Canada.12 Telix Investor Relations Ms. Kyahn Williamson Telix Pharmaceuticals Limited SVP Investor Relations and Corporate Communications Email: kyahn.williamson@telixpharma.com This announcement has been authorised for release by the Telix Pharmaceuticals Limited Board. Legal Notices This announcement is not intended as promotion or advertising directed to any healthcare professional or other audience in any country worldwide (including Australia, United States and the United Kingdom). This announcement may include forward-looking statements that relate to anticipated future events, financial performance, plans, strategies or business developments. Forward-looking statements can generally be identified by the use of words such as “may”, “expect”, “intend”, “plan”, “estimate”, “anticipate”, “outlook”, “forecast” and “guidance”, or other similar words. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. Forward-looking statements are based on the Company’s good-faith assumptions as to the financial, market, regulatory and other risks and considerations that exist and affect the Company’s business and operations in the future and there can be no assurance that any of the assumptions will prove to be correct. In the context of Telix’s business, forward-looking statements may include, but are not limited to, statements about: the initiation, timing, progress and results of Telix’s preclinical and clinical studies, and Telix’s research and development programs; Telix’s ability to advance product candidates into, enrol and successfully complete, clinical studies, including multi-national clinical trials; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities; the commercialisation of Telix’s product candidates, if or when they have been approved; estimates of Telix’s expenses, future revenues and capital requirements; Telix’s financial performance; developments relating to Telix’s competitors and industry; and the pricing and reimbursement of Telix’s product candidates, if and after they have been approved. Telix’s actual results, performance or achievements may be materially different from those which may be expressed or implied by such statements, and the differences may be adverse. Accordingly, you should not place undue reliance on these forward-looking statements. Except as required by applicable laws or regulations, Telix does not undertake to publicly update or review any forward-looking statements. Past performance cannot be relied on as a guide to future performance. Readers should read this announcement together with our material risks, as disclosed in our most recently filed reports with the ASX and on our website. ©2024 Telix Pharmaceuticals Limited and QSAM Biosciences, Inc.. The Telix Pharmaceuticals and Illuccix name and logo are trademarks of Telix Pharmaceuticals Limited and its affiliates. The QSAM Biosciences name and logo are trademarks of QSAM Biosciences, Inc. All rights reserved. ________________________ 1 QSAM corporate presentation Q2 2023. 2 QSAM media release 16 August 2023. 3 QSAM media release 18 August 2021. 4 QSAM media release 2 February 2022. 5 AUD/USD exchange rate of 0.6522 6 Refer to Telix disclosures and Appendix 3B lodged with the ASX today for further details. 7 Telix ASX disclosure 14 November 2023. 8 Huang, J., et al., (2020). Incidence Of Patients with Bone Metastases At Diagnosis Of Solid Tumors In Adults: A Large Population-Based Study. Doi: 10.21037/atm.2020.03.55. 9 Colsia, et al., (2022). The Burden of Metastatic Cancer-Induced Bone Pain: A Narrative Review. Doi:10.2147/JPR.S371337. 10 Telix ASX disclosure 20 December 2021. 11 Telix ASX disclosure 2 November 2021. 12 Telix ASX disclosure 14 October 2022.