Low grade glioma

Achieved $20.1 million in OJEMDATM (tovorafenib) net product revenue Ended the third quarter with $558.4 million in cash, cash equivalents and short-term investments Company to host conference call and webcast today, October 30, 4:30 p.m. Eastern Time BRISBANE, Calif., Oct. 30, 2024 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN) (“Day One” or the “Company”), a biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced its third quarter 2024 financial results and highlighted recent corporate achievements. “Our third quarter results demonstrate continued patient demand for OJEMDA, driven by the need for new therapies for children living with pediatric low-grade glioma,” said Jeremy Bender, Ph.D., chief executive officer of Day One. “Looking ahead to 2025, we plan to continue to drive growth by advancing our programs and pipeline, including DAY301, a potential first-in-class ADC targeting PTK7 that we expect to be in the clinic in the coming months.” Program Highlights Strong growth in OJEMDA net revenue with $20.1M in the third quarter of 2024, representing a 145% increase over the second quarter of 2024.Quarterly prescriptions (TRx) grew to 619 in the third quarter of 2024, representing a 159% increase over the second quarter of 2024.Day One expects to dose the first patient in the Phase 1a portion of the Phase 1a/b clinical trial of DAY301 by the end of 2024 or in the first quarter of 2025.Day One provided updated duration of response data from the registrational Phase 2 FIREFLY-1 trial investigating tovorafenib in patients with BRAF-altered, relapsed or progressive pLGG. For the 77 patients enrolled on Arm 1, which was the dataset used to assess OJEMDA’s efficacy, the median duration of response is 18 months.The pivotal Phase 3 FIREFLY-2/LOGGIC clinical trial evaluating tovorafenib as a front-line therapy in patients aged 6 months to 25 years with pLGG continues to enroll patients in the United States, Canada, Europe, Australia and Asia, with more than 100 sites activated. Corporate Highlights and Upcoming Milestones Day One and Ipsen entered into an exclusive licensing agreement to commercialize tovorafenib outside of the U.S. in July 2024. Under the agreement, Day One received approximately $111 million upfront in cash and equity investment at a premium with up to approximately $350 million in additional launch and sales milestone payments as well as tiered double-digit royalties starting at mid-teens percentage on net sales.Day One entered into a definitive agreement for an oversubscribed private placement of its securities for total gross proceeds of approximately $175 million in July 2024. Third Quarter 2024 Financial Highlights Product Revenue, Net: OJEMDA net product revenues were $20.1 million for the third quarter of 2024, the first full quarter of the U.S. launch.License Revenue: License revenue from the sale of ex-U.S. commercial rights for tovorafenib was $73.7 million for the third quarter of 2024.R&D Expenses: Research and development expenses were $33.6 million for the third quarter of 2024 compared to $33.2 million for the third quarter of 2023. The increase was primarily due to the clinical trial activities related to tovorafenib and additional employee compensation costs.SG&A Expenses: Selling, general and administrative expenses were $29.0 million for the third quarter of 2024 compared to $18.3 million for the third quarter of 2023. The increase was primarily due to employee compensation costs, commercial launch activities, and professional service expenses to support the launch of OJEMDA.Net Income/Loss: Net income totaled $37.0 million for the third quarter of 2024 with non-cash stock-based compensation expense of $11.6 million, compared to a net loss of $46.2 million for the third quarter of 2023, with non-cash stock-based compensation expense of $9.6 million.Cash Position: The Company’s cash, cash equivalents and short-term investments totaled $558.4 million as of September 30, 2024. Upcoming Events Two posters on health-related quality of life and drug holiday from the registrational Phase 2 FIREFLY-1 trial investigating tovorafenib in patients with BRAF-altered, relapsed or progressive pLGG will be presented at the Society for Neuro-Oncology Annual Meeting on November 22, 2024.Piper Sandler 36th Annual Healthcare Conference, December 3-5, 2024. Conference Call Day One will host a conference call and webcast today, October 30 at 4:30 p.m. Eastern Time. To access the live conference call by phone, dial 877-704-4453 (domestic) or 201-389-0920 (international), and provide the access code 13745150. Live audio webcast will be accessible from the Day One Investors & Media page. To ensure a timely connection to the webcast, it is recommended that participants register at least 15 minutes prior to the scheduled start time. An archived version of the webcast will be available for replay on the Events & Presentations section of the Day One Investors & Media page for 30 days following the event. About OJEMDA™OJEMDA (tovorafenib) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. OJEMDA is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma. For more information, please visit www.ojemda.com. About Day One Biopharmaceuticals Day One Biopharmaceuticals believes when it comes to pediatric cancer, we can do better. The Company was founded to address a critical unmet need: the dire lack of therapeutic development in pediatric cancer. Inspired by “The Day One Talk” that physicians have with patients and their families about an initial cancer diagnosis and treatment plan, Day One aims to re-envision cancer drug development and redefine what’s possible for all people living with cancer—regardless of age—starting from Day One. Day One partners with leading clinical oncologists, families, and scientists to identify, acquire, and develop important targeted cancer treatments. The Company’s pipeline includes tovorafenib (OJEMDA™), DAY301 and a VRK1 inhibitor program. Day One is based in Brisbane, California. For more information, please visit www.dayonebio.com or find the Company on LinkedIn or X. Day One uses its Investor Relations website (ir.dayonebio.com), its X handle (x.com/DayOneBio), and LinkedIn Home Page (linkedin.com/company/dayonebio) as a means of disseminating or providing notification of, among other things, news or announcements regarding its business or financial performance, investor events, press releases, and earnings releases, and as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Cautionary Note Regarding Forward-Looking StatementsThis press release contains “forward-looking” statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: Day One’s plans to develop and commercialize cancer therapies, expectations from current and planned clinical trials, the execution of the Phase 2 and Phase 3 clinical trial for tovorafenib as designed, expectations with respect to the timing of Day One’s Phase 1a/b clinical trial of DAY301, any expectations about safety, efficacy, timing and ability to complete clinical trials, release data results and to obtain regulatory approvals for tovorafenib and other candidates in development, and the ability of tovorafenib to treat pLGG or related indications. Statements including words such as “believe,” “plan,” “continue,” “expect,” “will,” “develop,” “signal,” “potential,” or “ongoing” and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. Forward-looking statements are subject to risks and uncertainties that may cause Day One’s actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties in this press release and other risks set forth in our filings with the Securities and Exchange Commission, including Day One’s ability to develop, obtain and retain regulatory approval for or commercialize any product candidate, Day One’s ability to protect intellectual property, the potential impact of global business or macroeconomic conditions, including as a result of inflation, rising interest rates, instability in the global banking system, geopolitical conflicts and the sufficiency of Day One’s cash, cash equivalents and investments to fund its operations. These forward-looking statements speak only as of the date hereof and Day One specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law. Day One Biopharmaceuticals, Inc.Condensed Statements of Operations(in thousands, except share and per share amounts)(unaudited) Three Months EndedSeptember 30, Nine Months EndedSeptember 30, 2024 2023 2024 2023 Revenue: Product revenue, net$20,070 $— $28,262 $— License revenue 73,691 — 73,691 — Total revenues 93,761 — 101,953 — Cost and operating expenses: Cost of product revenue 1,590 — 2,297 — Research and development 33,563 33,163 165,879 93,173 Selling, general and administrative 28,972 18,275 85,715 53,374 Total cost and operating expenses 64,125 51,438 253,891 146,547 Income (loss) from operations 29,636 (51,438) (151,938) (146,547)Non-operating income: Gain from sale of priority review voucher — — 108,000 — Investment income, net 5,322 5,291 13,649 12,163 Other income (expense), net 1,197 (3) 1,177 (22)Total non-operating income, net 6,519 5,288 122,826 12,141 Income (loss) before income taxes 36,155 (46,150) (29,112) (134,406)Income tax benefit (expense) 882 — (670) — Net income (loss) 37,037 (46,150) (29,782) (134,406)Net income (loss) per share - basic$0.38 $(0.54) $(0.33) $(1.73)Net income (loss) per share - diluted$0.38 $(0.54) $(0.33) $(1.73)Weighted-average number of common shares used in net income (loss) per share - basic 96,623,123 85,952,501 90,164,895 77,682,237 Weighted-average number of common shares used in net income (loss) per share - diluted 96,937,759 85,952,501 90,164,895 77,682,237 Day One Biopharmaceuticals, Inc.Selected Condensed Balance Sheet Data(in thousands)(unaudited) September 30,2024 December 31,2023Cash, cash equivalents and short-term investments$558,383 $366,347 Total assets 600,807 376,048 Total liabilities 45,344 29,508 Accumulated deficit (488,367) (458,585)Total stockholders’ equity 555,463 346,540 DAY ONE MEDIALaura Cooper, Head of Communications media@dayonebio.com DAY ONE INVESTORSLifeSci Advisors, PJ Kelleherpkelleher@lifesciadvisors.com

AGOURA HILLS, Calif., Oct. 28, 2024 (GLOBE NEWSWIRE) -- Oncotelic Therapeutics, Inc (OTCQB:OTLC) announced today its fourth publication of its TGFB2 series. Clinical data demonstrated that pancreatic cancer patients with high expression levels of either TGFB2, IRF9, or IFI27 showed improvement of median overall survival from 16-20 months to 72 months for patients with low levels of expression for both TGFB2 and either IRF9 or IFI27 validating that knockdown of IRF9 and IFI27 as therapeutic targets for TGFB2 therapeutics. https://www.mdpi.com/3003472 The publications of this series are available at Pubmed as follow: 1) TGFB2 mRNA Levels Prognostically Interact with Interferon-Alpha Receptor Activation of IRF9 and IFI27, and an Immune Checkpoint LGALS9 to Impact Overall Survival in Pancreatic Ductal Adenocarcinoma. Qazi S, Trieu V. Int J Mol Sci. 2024 Oct 18;25(20):11221. doi: 10.3390/ijms252011221. PMID: 39457004 2) Transforming Growth Factor Beta 2 (TGFB2) mRNA Levels, in Conjunction with Interferon-Gamma Receptor Activation of Interferon Regulatory Factor 5 (IRF5) and Expression of CD276/B7-H3, Are Therapeutically Targetable Negative Prognostic Markers in Low-Grade Gliomas. Trieu V, Maida AE, Qazi S. Cancers (Basel). 2024 Mar 19;16(6):1202. doi: 10.3390/cancers16061202. PMID: 38539537 Free PMC article. 3) Transforming Growth Factor Beta 2 (TGFB2) and Interferon Gamma Receptor 2 (IFNGR2) mRNA Levels in the Brainstem Tumor Microenvironment (TME) Significantly Impact Overall Survival in Pediatric DMG Patients. Qazi S, Talebi Z, Trieu V. Biomedicines. 2024 Jan 15;12(1):191. doi: 10.3390/biomedicines12010191. PMID: 38255296 Free PMC article. 4) High Intra-Tumor Transforming Growth Factor Beta 2 Level as a Predictor of Poor Treatment Outcomes in Pediatric Diffuse Intrinsic Pontine Glioma. Uckun FM, Qazi S, Trieu V. Cancers (Basel). 2023 Mar 9;15(6):1676. doi: 10.3390/cancers15061676. PMID: 36980562 Free PMC article. Additional information on our current phase 3 can be found at: https://www.youtube.com/watch?v=5KqdbySDRKE About Oncotelic Oncotelic (f/k/a Mateon Therapeutics, Inc.), was formed in the State of New York in 1988 as OXiGENE, Inc., was reincorporated in the State of Delaware in 1992, and changed its name to Mateon Therapeutics, Inc. in 2016, and Oncotelic Therapeutics, Inc. in November 2020. Oncotelic is seeking to leverage its deep expertise in oncology drug development to improve treatment outcomes and survival of cancer patients with a special emphasis on rare pediatric cancers. Oncotelic has rare pediatric designation for Diffuse Intrinsic Pontine Glioma (“DIPG” through OT-101) through its 45% joint venture, GMP Biotechnology Limited, melanoma (through CA4P), and Acute Myeloid Leukemia (through OXi 4503). Oncotelic acquired PointR Data Inc. in November 2019 to build an AI driven biotechnology company. Further, Oncotelic acquired AL-101, during the 4th quarter of 2021, for the intranasal delivery of apomorphine. We intend to develop AL-101 for the treatment of Parkinson Disease, erectile dysfunction, female sexual disorder and hypoactive sexual desire disorder. All these ailments have a very large population suffering from them and there is a need for treatments for each. For more information on AL-101, refer to our Annual Report on Form 10-K/A filed with the SEC on April 19, 2023. Oncotelic's Cautionary Note on 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 communication regarding strategy, future operations, future financial position, prospects, plans and objectives of management are forward-looking statements. Words such as "may", "expect", "anticipate" "hope", "vision", "optimism", "design", "exciting", "promising", "will", "conviction", "estimate," "intend," "believe", "quest for a cure of cancer", "innovation-driven", "paradigm-shift", "high scientific merit", "impact potential" and similar expressions are intended to identify forward-looking statements. Forward looking statements contained in this press release include, but are not limited to, statements about future plans related to the operations of the JV, taking the JV into an initial public offering or the success thereof, the progress, timing of clinical development, scope and success of future clinical trials, the reporting of clinical data for the Company’s product candidates and the potential use of the Company's product candidates to treat various cancer indications as well as obtaining required regulatory approval to conduct clinical trials and upon granting of approval by the regulatory agencies, the successful marketing of the products; building and the success of our nanoparticle platform and the related success of launching the platform, the development of the Company’s AI suite including but not limited to AI Chatbots, the public access to the Company’s AI suite, the success of the development of the AI suite or any other AI technologies and whether if any or portion thereof the Company’s AI suite or technologies will be commercialized and launched for sale. Each of these forward-looking statements involves risks and uncertainties, and actual results may differ materially from these forward-looking statements or may not occur at all. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes, taking the Company or its affiliates through initial public offerings. These risks are not exhaustive, the company faces known and unknown risks, including the risk factors described in the Company's Annual Report on Form 10-K/A filed with the SEC on April 19, 2023 and in the company's other periodic filings. Forward-looking statements are based on expectations and assumptions as of the date of this press release. Except as required by law, the company does not assume any obligation to update forward-looking statements contained herein to reflect any change in expectations, whether because of new information, future events, or otherwise. Contact Information: For Oncotelic Therapeutics, Inc.:Investor Relationsir@oncotelic.com

LOUISVILLE, Ky., Aug. 12, 2024 (GLOBE NEWSWIRE) -- Onco360®, the nation’s leading independent Specialty Pharmacy, has been selected by Servier Pharmaceuticals, as a specialty pharmacy partner for VORANIGO® (vorasidenib). VORANIGO® is approved for the treatment of adults and pediatric patients 12 years of age and older with grade 2 astrocytoma or oligodendroglioma and a susceptible isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation following surgery including biopsy, sub-total resection, or gross total resection1. This indication was approved based on the Phase III INDIGO (NCT04164901) trial, which showed vorasidenib significantly extended progression free survival (PFS) when compared to placebo. Vorasidenib is a small molecule inhibitor that targets IDH1 and IDH2 enzymes. “Onco360 is grateful for the opportunity to become a specialty pharmacy provider for VORANIGO®,” said Benito Fernandez, Chief Commercial Officer. “We are proud to add the first FDA approved targeted therapy for Grade 2 IDH-mutant glioma to our portfolio.” The FDA approval of vorasidenib is based on the results of the Phase III INDIGO clinical trial, which evaluated 331 patients with grade 2 astrocytoma or oligodendroglioma with IDH1 or IDH2 mutations. Patients received either vorasidenib 40mg daily or placebo until disease progression or unacceptable toxicity. The INDIGO trial met its primary endpoint of PFS and key secondary endpoint of time to next intervention (TTNI). The median PFS was 27.7 months in the vorasidenib group compared to 11.1 months in the placebo arm, HR = 0.39 (95% CI 0.27-0.56). Vorasidenib was well tolerated by patients as part of the INDIGO trial. The most common adverse reactions (≥15%) were fatigue, COVID-19, musculoskeletal pain, diarrhea, and seizure.2 Please see the full Prescribing Information for VORANIGO®. About Onco360 Oncology Pharmacy:Onco360 is the nation’s largest independent Oncology Pharmacy and clinical support services company. Onco360 was founded in 2003 to bring together the stakeholders involved in the cancer treatment process and serve the specialized needs of oncologists, patients, hospitals, cancer centers of excellence, manufacturers, health plans, and payers. It dispenses nationally through its network of URAC-, and ACHC-accredited Specialty Pharmacies. Onco360 is headquartered in Louisville, Kentucky, and is a flagship specialty pharmacy brand of PharMerica Corporation, a leading institutional pharmacy, specialty infusion, and hospital services company servicing healthcare facilities in the United States. For more information about Onco360, please visit Onco360.com. Media Contact: Benito Fernandez, Chief Commercial OfficerBenito.Fernandez@Onco360.com516-640-1332 References: VORANIGO (vorasidenib) Prescribing Information. Accessed August 2024. https://d11vmvycldsjdg.cloudfront.net/7fffe4c3-9746-4e82-9582-df9879a712d5/5939acdd-1c44-41f5-b2f6-260ab3860288/5939acdd-1c44-41f5-b2f6-260ab3860288_web_ready__c.pdfIngo K. Mellinghoff et al. INDIGO: Vorasidenib in IDH1 or IDH2 mutant low grade glioma. NEJM 2023; 389: 589-601. DOI: 10.1056/NEJMoa2304194.