Diffuse Midline Glioma

SYDNEY, March 13, 2024 /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA), a biotechnology company specialising in oncology, is pleased to announce the presentation of new data for both its pipeline molecules, paxalisib and EVT801, at the upcoming Annual Meeting of the American Association of Cancer Research (AACR) in San Diego, California, from 5-10 April 2024. There will be three presentations in total at AACR, including data from the phase 1 study of EVT801 in advanced solid cancers. The data being presented will outline initial clinical data from the phase 1 study and provides support and direction for the next stage of the study. In addition, data will be presented on the results of the combination therapy of paxalisib and gemcitabine for patients with relapsed/recurrent atypical teratoid/rhabdoid tumors AT/RT. Based on these findings, the Pacific Pediatric Neuro-Oncology Consortium (PNOC) is planning to include this therapy in its next AT/RT international clinical trial. Summary of Abstracts Session PO.CL01.15 - Early Detection Biomarkers 1 April 7, 2024 – 1:30pm-5:00pm Abstract 1059 / 7: Biomarkers analysis on samples from patients in EVT801 clinical trial: Patient characterization and immunomonitoring L. Davenne, M. Fitzgerald, P.-B. Ancey, O. Delpuech, C. Poussereau-Pomié, M. Esquerre, M. R. Paillasse, M. Mandron, P. Rochaix, M. Ayyoub, C. Scarlata, C. Caux, P. Cassier, C. Gomez-Roca, J.-P. Delord, J. Friend, P. Fons Evotec International GmbH, Toulouse, France, Kazia Therapeutics, Sydney, Australia, Institut Universitaire du Cancer Toulouse-Oncopole, Toulouse, France, Centre Léon Bérard, Lyon, France Session PO.CTP01.01 - Phase I Clinical Trials in Progress 1 April 8, 2024 – 9:00am-12:30pm Abstract CT088 / 15: EVT801, a novel selective VEGFR-3 inhibitor targeting tumor angiogenesis, is pursuing dose escalation stage of phase I first-in-human study C. Gomez-Roca, P. Cassier, M. Fitzgerald, L. Davenne, C. Costantin, P. Rochaix, J.-P. Delord, J. Friend, A. Nizzardo, A. Tagliavini, M. Pergher, P. Fons, M. Mandron Institut Universitaire du Cancer Toulouse-Oncopole, Toulouse, France, Centre Léon Bérard, Lyon, France, Kazia Therapeutics, Sydney, Australia, Evotec International GmbH, Toulouse, France, Evotec International GmbH, Verona, Italy Session MS.CL08.01 - Novel Approaches for Targeted Therapies April 9, 2024 – 2:50pm-3:05pm Abstract 6565 - Improving survival of atypical teratoid/rhabdoid tumor orthotopic xenografts through the combination of PI3K inhibitor paxalisib and nucleoside analog gemcitabine T. Findlay, K. Malebranche, A. Geethadevi, C. Eberhart, J. Rubens, E. Raabe Johns Hopkins University School of Medicine, Baltimore, MD About Kazia Therapeutics Limited Kazia Therapeutics Limited (NASDAQ: KZIA) is an oncology-focused drug development company, based in Sydney, Australia. Our lead program is paxalisib, an investigational brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway, which is being developed to treat multiple forms of brain cancer. Licensed from Genentech in late 2016, paxalisib is or has been the subject of ten clinical trials in this disease. A completed Phase 2 study in glioblastoma reported early signals of clinical activity in 2021, and a pivotal study in glioblastoma, GBM AGILE, is ongoing, with final data expected in 1H2024. Other clinical trials are ongoing in brain metastases, diffuse midline gliomas, and primary CNS lymphoma, with several of these having reported encouraging interim data. Paxalisib was granted Orphan Drug Designation for glioblastoma by the FDA in February 2018, and FTD for glioblastoma by the FDA in August 2020. Paxalisib was also granted FTD in July 2023 for the treatment of solid tumour brain metastases harboring PI3K pathway mutations in combination with radiation therapy. In addition, paxalisib was granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA for diffuse intrinsic pontine glioma in August 2020, and for atypical teratoid / rhabdoid tumours in June 2022 and July 2022, respectively. Kazia is also developing EVT801, a small-molecule inhibitor of VEGFR3, which was licensed from Evotec SE in April 2021. Preclinical data has shown EVT801 to be active against a broad range of tumour types and has provided evidence of synergy with immuno-oncology agents. A Phase I study is ongoing and preliminary data is anticipated in CY2024. For more information, please visit or follow us on Twitter @KaziaTx. Forward-Looking Statements This announcement may contain forward-looking statements, which can generally be identified as such by the use of words such as "may," "will," "estimate," "future," "forward," "anticipate," or other similar words. Any statement describing Kazia's future plans, strategies, intentions, expectations, objectives, goals or prospects, and other statements that are not historical facts, are also forward-looking statements, including, but not limited to, statements regarding: the timing for results and data related to Kazia's clinical and preclinical trials and investigator-initiated trials of Kazia's product candidates, and Kazia's strategy and plans with respect to its programs, including paxalisib and EVT801. Such statements are based on Kazia's current expectations and projections about future events and future trends affecting its business and are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements, including risks and uncertainties: associated with clinical and preclinical trials and product development, related to regulatory approvals, and related to the impact of global economic conditions. These and other risks and uncertainties are described more fully in Kazia's Annual Report, filed on form 20-F with the United States Securities and Exchange Commission (SEC), and in subsequent filings with the SEC. Kazia undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required under applicable law. You should not place undue reliance on these forward-looking statements, which apply only as of the date of this announcement. This announcement was authorized for release by Dr John Friend, CEO. Company Codes: NASDAQ-SMALL:KZIA

– ONC201 ACTION Study Progressing; Reiterate Interim OS Data Expected in 2025, Final OS Data Expected in 2026 – – Phase 2 ONC201 Data Published in Peer-Reviewed Journal of Clinical Oncology – – $204 Million in Cash and Equivalents at December 31, 2023 – – Conference Call at 8:30 a.m. ET Today – DURHAM, N.C., Feb. 29, 2024 (GLOBE NEWSWIRE) -- Chimerix (NASDAQ:CMRX), a biopharmaceutical company whose mission it is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases, today reported financial results for the fourth quarter and full-year ended December 31, 2023 and provided an operational update. “Following strong clinical development in 2023, we remain very focused on advancing the ONC201 ACTION study, completing ONC206 dose escalation this year and strengthening our executive team as we prepare for potential commercialization of ONC201,” said Mike Andriole, Chief Executive Officer of Chimerix. “We are making good progress enrolling our global Phase 3 ACTION study and are excited about the prospect of having interim overall survival data next year. In addition, we are pleased to share that the ONC201 Phase 2 data was recently published in the Journal of Clinical Oncology which further elucidates key characteristics of response and detailed patient-level data.” “During the fourth quarter, we were delighted to strengthen our Board of Directors with the addition of Lisa Decker, Ph.D., as well as strengthen the management team with the promotion of Michelle LaSpaluto to Chief Financial Officer and the additions of Tom Riga as Chief Operating and Commercial Officer and Pablo Lee, MD, as Vice President of Medical Affairs. We are confident their collective expertise will be invaluable assets to Chimerix as we seek to maximize our future growth potential for patients and shareholders,” added Mr. Andriole. ONC201 Journal of Clinical Oncology Publication In February 2024, “ONC201 (dordaviprone) in Recurrent H3 K27M-mutant Diffuse Midline Glioma,” was published in the Journal of Clinical Oncology (JCO), a peer reviewed journal of the American Society of Clinical Oncology (ASCO). The manuscript reports in detail the results of 50 patients with recurrent H3 K27M-DMG treated with monotherapy ONC201 who were evaluable for objective response by Response Assessment in Neuro-Oncology (RANO) high grade glioma (HGG) criteria. ONC201 demonstrated a median overall survival (mOS) of 13.7 months (95% CI: 8.0 - 20.3), with an overall two-year rate of survival of 35% (95% CI: 21-49) from the start of ONC201 treatment post-recurrence. Chimerix previously conducted a natural disease history study (n=43) in the recurrent setting evaluating patients who did not receive ONC201 which showed a mOS of 5.1 months (95% CI: 3.9 - 7.7) with an overall two-year survival rate of 11% (95% CI:3.3-24.2).The top-line data from this JCO publication were previously disclosed by Chimerix. The journal can be accessed here. The Phase 3 ACTION trial is currently enrolling patients at over 130 sites in 13 countries. The trial enrolls patients shortly after completion of front-line radiation therapy that is the standard of care. The study is designed to enroll 450 patients randomized 1:1:1 to receive ONC201 at one of two dosing frequencies or placebo. Participants are randomized to receive 625mg of ONC201 once per week (the Phase 2 dosing regimen), 625mg twice per week on two consecutive days or placebo. The dose will be scaled by body weight for patients <52.5kg. For more information, please visit clinicaltrials.gov ONC206 ONC206 is a second generation ClpP agonist and DRD2 antagonist that has demonstrated monotherapy anti-cancer activity in pre-clinical models in primary CNS tumors and solid tumors outside of the CNS. Phase I dose escalation trials continue at the National Institutes of Health (NIH) and the Pacific Pediatric Neuro-Oncology Consortium (PNOC) in adult and pediatric CNS tumor patients, respectively. To date, ONC206 has been generally well tolerated with no dose limiting toxicities. The dose escalation trials are currently dosing at more frequent dose schedules, which are expected to increase the duration of therapeutic exposure. Chimerix expects to report preliminary safety and pharmacokinetic data from these trials beginning in mid-2024. Fourth Quarter 2023 Financial Results Chimerix’s balance sheet at December 31, 2023 included $204.5 million of capital available to fund operations, no debt, and approximately 88.9 million outstanding shares of common stock. Chimerix reported a net loss of $18.2 million, or $0.20 per basic and diluted share, for the fourth quarter of 2023, compared to a net loss of $21.0 million, or $0.24 per basic and diluted share for the fourth quarter of 2022. Research and development expenses decreased to $15.6 million for the three-month period ended December 31, 2023, compared to $19.3 million for the same period in 2022. This decrease was primarily driven by one-time costs associated with a reduction in force related to the TEMBEXA divestiture in the comparable 2022 period. General and administrative expenses decreased to $5.2 million for the fourth quarter of 2023, compared to $5.3 million for the same period in 2022. Full Year 2023 Financial Results Chimerix reported a net loss of $82.1 million, or $0.93 per basic and diluted share, for the year ended December 31, 2023. For the year ended December 31, 2022, Chimerix recorded net income of $172.2 million, or $1.97 per basic and $1.94 per diluted share. The decrease was primarily driven by the gain on sale of TEMBEXA to Emergent BioSolutions in 2022. Revenues for 2023 decreased to $0.3 million, compared to $33.8 million in 2022. The decrease was primarily related to deliveries under international TEMBEXA procurement agreements in the comparable 2022 period. Research and development expenses decreased to $68.8 million for the year ended December 31, 2023, compared to $71.6 million for the year ended December 31, 2022. General and administrative expenses increased to $24.6 million for the year ended December 31, 2023, compared to $22.1 million for the year ended December 31, 2022. Conference Call and Webcast Chimerix will host a conference call and live audio webcast to discuss fourth quarter and full-year 2023 financial results and provide a business update today at 8:30 a.m. ET. To access the live conference call, please dial 646-307-1963 (domestic) or 800-715-9871 (international) at least five minutes prior to the start time and refer to conference ID 6933453. A live audio webcast of the call will also be available on the Investors section of Chimerix’s website, www.chimerix.com. An archived webcast will be available on the Chimerix website approximately two hours after the event. About Chimerix Chimerix is a biopharmaceutical company with a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. The Company’s most advanced clinical-stage development program, ONC201, is in development for H3 K27M-mutant glioma. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include those relating to, among other things, enrollment and timing of data for the Phase 3 ACTION study, the results of dose escalation trials of ONC206, and the impact of recent changes to the Board of Directors and management team. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are risks related to the timing, completion and outcome of the Phase 3 ACTION study of ONC201; risks associated with repeating positive results obtained in prior preclinical or clinical studies in future studies; risks related to the clinical development of ONC206; and additional risks set forth in the Company's filings with the Securities and Exchange Commission. These forward-looking statements represent the Company's judgment as of the date of this release. The Company disclaims, however, any intent or obligation to update these forward-looking statements. CONTACT: Will O’ConnorStern Investor Relations212-362-1200will@sternir.com CHIMERIX, INC.CONSOLIDATED BALANCE SHEETS(in thousands, except share and per share data) December 31, December 31, 2023 2022 ASSETS Current assets: Cash and cash equivalents$27,661 $25,842 Short-term investments, available-for-sale 155,174 191,492 Accounts receivable 4 1,040 Prepaid expenses and other current assets 6,271 9,764 Total current assets 189,110 228,138 Long-term investments 21,657 48,626 Property and equipment, net of accumulated depreciation 224 227 Operating lease right-of-use assets 1,482 1,964 Other long-term assets 301 386 Total assets$212,774 $279,341 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities: Accounts payable$2,851 $3,034 Accrued liabilities 15,592 17,381 Total current liabilities 18,443 20,415 Loan Fees 125 250 Lease-related obligations 1,177 1,819 Total liabilities 19,745 22,484 Stockholders’ equity: Preferred stock, $0.001 par value, 10,000,000 shares authorized at December 31, 2023 and 2022; no shares issued and outstanding as of December 31, 2023 and 2022 - - Common stock, $0.001 par value, 200,000,000 shares authorized at December 31, 2023 and 2022; 88,929,300 and 88,054,127 shares issued and outstanding as of December 31, 2023 and 2022, respectively 89 88 Additional paid-in capital 988,457 970,535 Accumulated other comprehensive gain (loss), net 7 (337) Accumulated deficit (795,524) (713,429) Total stockholders’ equity 193,029 256,857 Total liabilities and stockholders’ equity$212,774 $279,341 CHIMERIX, INC.CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE (LOSS) INCOME(in thousands, except share and per share data) Three Months Ended December 31, Years Ended December 31, 2023 2022 2023 2022 Revenues: Procurement revenue$- $- $- $31,971 Contract and grant revenue 4 439 275 942 Licensing revenue - - 49 536 Royalty revenue - 375 - 375 Total revenues 4 814 324 33,824 Cost of goods sold - - - 447 Gross Profit 4 814 324 33,377 Operating expenses: Research and development 15,644 19,281 68,788 71,631 General and administrative 5,170 5,347 24,601 22,132 Total operating expenses 20,814 24,628 93,389 93,763 Loss from operations (20,810) (23,814) (93,065) (60,386)Other income: Interest income and other, net 2,649 2,737 10,970 2,919 Gain on sale of business, net - - - 229,670 (Loss) income before income taxes (18,161) (21,077) (82,095) 172,203 Income tax expense - (117) - 36 Net (loss) income (18,161) (20,960) (82,095) 172,167 Other comprehensive income (loss): Unrealized income (loss) on investments, net 632 (300) 344 (316) Comprehensive (loss) income$(17,529) $(21,260) $(81,751) $171,851 Per share information: Net (loss) income, basic$(0.20) $(0.24) $(0.93) $1.97 Net (loss) income, diluted$(0.20) $(0.24) $(0.93) $1.94 Weighted-average shares outstanding, basic 88,910,300 88,049,138 88,604,026 87,555,110 Weighted-average shares outstanding, diluted 88,910,300 88,049,138 88,604,026 88,776,147

DURHAM, N.C., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Chimerix (NASDAQ:CMRX), a biopharmaceutical company whose mission is to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases, today announced that Mike Andriole, Chief Executive Officer, will participate in a Targeted Oncology Panel at the TD Cowen 44th Annual Health Care Conference on Tuesday, March 5, 2024 at 12:50 p.m. ET. An audio webcast of the panel discussion will be available on the Investor Relations section of Chimerix's website at ir.chimerix.com, where it will be archived for approximately 90 days. About Chimerix Chimerix is a biopharmaceutical company with a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. The Company’s most advanced clinical-stage development program, ONC201, is in development for H3 K27M-mutant glioma. CONTACT: Will O’ConnorStern Investor Relations212-362-1200will@sternir.com