Ependymoma

SAN FRANCISCO, Feb. 05, 2024 (GLOBE NEWSWIRE) -- ProLynx Inc. announced today that the first patient was treated with PLX038 (PEGylated SN-38) in a Phase I/II clinical trial for primary CNS tumors driven by MYC or MYCN amplifications. National Institutes of Health’s NCI investigators Dr. Marta Penas-Prado and Dr. Mark Gilbert are conducting the trial. MYC genes regulate expression of genes involved in cell division. High levels of MYC drive oncogenesis in many cancers and induce DNA changes leading to the formation of “topoisome complexes”. MYC and MYCN amplifications are seen in multiple primary tumors of the central nervous system (CNS). This includes 30% of relapsed medulloblastomas, and MYCN-amplified ependymoma, characterized by aggressive clinical behavior and treatment resistance. The MYC or MYCN-induced topoisome contains high levels of topoisomerases that should be susceptible to topoisomerase inhibitors. PLX038 is a long-acting prodrug of the topoisomerase 1 inhibitor, SN-38 – the active metabolite of irinotecan and sacituzumab govitecan. PLX038 is unique as the SN-38 is covalently bound to a circulating nanomolecule and is slowly released to provide free SN-38 with a long half-life, low Cmax and very high exposure – important for optimal safety and efficacy. Importantly, in preclinical studies, PLX038 was shown to accumulate in CNS tumors, where it slowly releases SN-38. The NCI trial will assess whether PLX038 is safe and efficacious in primary CNS tumors driven by MYC or MYCN amplifications. The Phase I trial will confirm the recommended Phase II dose. The Phase II trial will test PLX038 in 3 independent cohorts: 1) newly diagnosed MYCN-amplified ependymoma, 2) recurrent ependymoma or medulloblastoma with MYCN or MYC amplification, and 3) other recurrent primary CNS tumors with MYC or MYCN amplifications. Notably, the Phase II trial will incorporate on-treatment tumor biopsies to investigate biomarkers of response and resistance, including SLFN11, MYC, MYCN and Top1, 2a, and 2b expression. This data could prospectively identify patients most responsive to the drug. Additionally, working collaboratively with The Office of Patient-Centered Outcomes (OPCORe), researchers will collect patient-reported outcomes (PROs) to measure the impact of therapy, including toxicity, symptom severity and interference with daily activities, and physical functioning. ProLynx co-founder, Daniel V. Santi, stated, “We are excited to work with NCI investigators to bring novel treatments to patients with rare CNS cancers, in hope of improving their outcomes.” He added, “Conventional short-acting topoisomerase 1 inhibitors have clinical activity in certain CNS tumors. Accordingly, we are optimistic that a long-acting SN-38 will result in increased efficacy and safety.” Additional information on this clinical trial is available at clinicaltrials.gov, through identifier number NCT06161519. For patients interested in enrolling in this clinical trial, please call NCI’s toll-free number: 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615); visit the website: and/or email: NCIMO_referrals@mail.nih.gov. ProLynx is a San Francisco biotechnology company developing proprietary systems to improve the pharmacokinetics/efficacy of important drugs ( ).

New survival data from 15 patients in the Phase 2 ReSPECT-GBM trial of rhenium (186Re) obisbemeda in recurrent glioblastoma (rGBM) will be presented at SNO Annual Meeting on November 17th; Company will also host a Key Opinion Leader (KOL) event following the meeting to discuss the results Completed dosing in Cohort 4 of the Phase 1 ReSPECT-LM dose escalation trial with rhenium (186Re) obisbemeda for leptomeningeal metastases, the first of four additional planned cohorts in Part B (Cohorts 4-7); an update will also be provided at the SNO meeting in November Current cash of $11 million, plus an additional $10.2 million expected through the end of 2024 from the Cancer Prevention & Research Institute of Texas (CPRIT) grant, should provide sufficient runway to fund operations into 2025 Management to Host Conference Call Today at 5:00 p.m. ET AUSTIN, Texas, Oct. 31, 2023 (GLOBE NEWSWIRE) -- Plus Therapeutics, Inc. (Nasdaq: PSTV) (the “Company”), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system cancers, today announced financial results for the third quarter ended September 30, 2023, and provided an overview of recent and upcoming business highlights. “Our active trials of Rhenium-186 Obisbemeda continue to show encouraging results in treating both recurrent glioblastoma and leptomeningeal metastases and we will provide significant updates on both trials at the Society for Neuro-Oncology (SNO) Annual Meeting and at our KOL event following the meeting in November,” said Marc H. Hedrick M.D., President and Chief Executive Officer of Plus Therapeutics. “Furthermore, our current balance sheet and forecasted grant payments from CPRIT, which will continue through August 2025, should be sufficient to provide a cash runway into 2025.” UPCOMING EVENTS AND MILESTONES Through the remainder of 2023 and 2024, the Company plans to accomplish the following key business objectives: Present the latest safety and efficacy data from the Phase 2 ReSPECT-GBM trial at the annual SNO meeting in Vancouver on November 15-19, 2023.Present the latest safety and efficacy data from the Phase 1 ReSPECT-LM trial at the annual SNO meeting in Vancouver on November 15-19, 2023.Participate in virtual KOL webinar following SNO meeting to discuss GBM data presented at the SNO meeting.Complete enrollment in the Phase 2 ReSPECT-GBM trial and finalize pivotal design with FDA.Complete enrollment in the Phase 1 ReSPECT-LM trial and begin phase 2 trial.Complete internal implementation of the CNSide™ cerebrospinal fluid (CSF)-based biomarker and tumor cell capture and enumeration assay being utilized in the ReSPECT-LM clinical trial.Obtain FDA IND approval and initiate the Phase 1 ReSPECT-PBC trial for pediatric patients with ependymoma and high-grade glioma at the Lurie Children’s Hospital in Chicago.Complete key development milestones for the company’s next generation radioembolic device 188RNL-BAM.Add key second source GMP supply chain partners to support late-stage clinical trials and commercial supply.Publish ReSPECT-GBM Phase 1 data in a peer-reviewed publication. Q3 HIGHLIGHTS AND MILESTONE ACHIEVEMENTS Completed dosing in Cohort 4 of the ReSPECT-LM Phase 1/2a dose escalation trial of rhenium (186Re) obisbemeda for the treatment of LM. Cohort 4 is the first of 4 planned cohorts in Part B.Presented preliminary safety and efficacy results from Phase 1/Part A of the ReSPECT-LM clinical trial at the SNO/ASCO CNS Cancer Conference. Following the presentation, the Company hosted a key opinion leader roundtable on data presented at the conference.Received advance payment of grant funds of approximately $1.9 million from CPRIT, as planned, as part of its overall $17.6 million award contract.Completed transfer of proprietary materials, protocols, and equipment from Biocept for CNSide, a cerebrospinal fluid (CSF)-based biomarker and tumor cell capture and enumeration assay being utilized in the ReSPECT-LM clinical trial.Strengthened clinical development leadership with the appointment of Pius Maliakal, M. Pharm., Ph.D., as Vice President of Clinical Operations. THIRD QUARTER 2023 FINANCIAL RESULTS The Company’s cash balance was $11.0 million at September 30, 2023, compared to $18.1 million at December 31, 2022.The Company recognized $1.2 million in grant revenue in the third quarter of 2023, which represents CPRIT’s share of the costs incurred for our rhenium (186Re) obisbemeda development for the treatment of patients with LM.Total operating expenses for the third quarter of 2023 were $4.5 million, compared to total operating expenses of $5.2 million for the same period the prior year. The decrease is due primarily to a decrease in research and development expenses from completion of the initial cGMP development work on rhenium (186Re) obisbemeda.In addition to current cash on hand, the Company benefits from grant awards of $3 million from the National Institutes of Health and $17.6 million from CPRIT.Net loss for the third quarter of 2023 was $(3.2) million, or $(1.00) per share, compared to a net loss of $(5.2) million, or $(2.85) per share, for the same period the prior year. THIRD QUARTER RESULTS CONFERENCE CALL The Company will hold a conference call and live audio webcast at 5:00 pm Eastern Time today to discuss its financial results and provide a general business update. A live webcast will be available at ir.plustherapeutics.com/events. Participants may also pre-register any time before the call here. Once registration is completed, participants will be provided a dial-in number with a personalized conference code to access the call. Please dial in 15 minutes prior to the start time. Following the live call, a replay will be available on the Company’s website under the ‘For Investors’ section. The webcast will be available on the Company’s website for 90 days following the live call. About Plus TherapeuticsPlus Therapeutics, Inc. is a clinical-stage pharmaceutical company developing targeted radiotherapeutics for difficult-to-treat cancers of the central nervous system with the potential to enhance clinical outcomes for patients. Combining image-guided local beta radiation and targeted drug delivery approaches, the Company is advancing a pipeline of product candidates with lead programs in recurrent glioblastoma (GBM) and leptomeningeal metastases (LM). The Company has built a robust supply chain through strategic partnerships that enable the development, manufacturing and future potential commercialization of its products. Plus Therapeutics is led by an experienced and dedicated leadership team and has operations in key cancer clinical development hubs including Austin and San Antonio, Texas. For more information, visit https://plustherapeutics.com/. Cautionary Statement Regarding Forward-Looking StatementsThis press release contains statements that may be deemed “forward-looking statements” within the meaning of U.S. securities laws. All statements in this press release other than statements of historical fact are forward-looking statements. These forward-looking statements may be identified by future verbs, as well as terms such as “designed to,” “will,” “can,” “potential,” “focus,” “preparing,” “next steps,” “possibly,” and similar expressions or the negatives thereof. Such statements are based upon certain assumptions and assessments made by management in light of their experience and their perception of historical trends, current conditions, expected future developments and other factors they believe to be appropriate. These statements include, without limitation, statements regarding the following: the potential promise of 186Re including the ability of 186Re to safely and effectively deliver radiation directly to the tumor at high doses; expectations as to the Company’s future performance including the next steps in developing the Company’s current assets; the Company’s clinical trials including statements regarding the timing and characteristics of the ReSPECT-GBM and ReSPECT-LM clinical trials; possible negative effects of 186Re; the continued evaluation of 186Re including through evaluations in additional patient cohorts; and the intended functions of the Company’s platform and expected benefits from such functions. The forward-looking statements included in this press release are subject to a number of risks and uncertainties that may cause actual results to differ materially from those discussed in such forward-looking statements. These risks and uncertainties include, but are not limited to: the Company’s actual results may differ, including materially, from those anticipated in these forward-looking statements as a result of various factors, including, but not limited to, the following: the early stage of the Company’s product candidates and therapies, the results of the Company’s research and development activities, including uncertainties relating to the clinical trials of its product candidates and therapies; the Company’s liquidity and capital resources and its ability to raise additional cash, the outcome of the Company’s partnering/licensing efforts, risks associated with laws or regulatory requirements applicable to it, market conditions, product performance, litigation or potential litigation, and competition within the cancer diagnostics and therapeutics field, among others; and additional risks described under the heading “Risk Factors” in the Company’s Securities and Exchange Commission filings, including in the Company’s annual and quarterly reports. There may be events in the future that the Company is unable to predict, or over which it has no control, and its business, financial condition, results of operations and prospects may change in the future. The Company assumes no responsibility to update or revise any forward-looking statements to reflect events, trends or circumstances after the date they are made unless the Company has an obligation under U.S. federal securities laws to do so. Investor ContactCorey Davis, Ph.D.LifeSci Advisors(212) 215-2577cdavis@lifesciadvisors.com PLUS THERAPEUTICS, INC.CONDENSED BALANCE SHEETS(UNAUDITED)(in thousands, except share and par value data) September 30, 2023 December 31,2022 Assets Current assets: Cash and cash equivalents $11,006 $18,120 Grant receivable 91 — Other current assets 487 3,697 Total current assets 11,584 21,817 Property and equipment, net 1,009 1,324 Operating lease right-of-use assets 232 248 Goodwill 372 372 Intangible assets, net 49 94 Other assets 32 12 Total assets $13,278 $23,867 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable and accrued expenses $6,073 $10,134 Operating lease liability 117 110 Term loan obligation 4,348 1,608 Total current liabilities 10,538 11,852 Term loan obligation — 3,786 Noncurrent operating lease liability 118 141 Deferred grant liability — 1,643 Total liabilities 10,656 17,422 Stockholders’ equity: Preferred stock, $0.001 par value; 5,000,000 shares authorized; 1,952 shares issued and outstanding at September 30, 2023 and December 31, 2022, respectively — — Common stock, $0.001 par value; 100,000,000 shares authorized; 4,522,656 and 2,240,092 shares issued and outstanding at September 30, 2023 and December 31, 2022, respectively 5 2 Additional paid-in capital 479,308 473,628 Accumulated deficit (476,691) (467,185)Total stockholders’ equity 2,622 6,445 Total liabilities and stockholders’ equity $13,278 $23,867 PLUS THERAPEUTICS, INC.CONDENSED STATEMENTS OF OPERATIONS(UNAUDITED)(in thousands, except share and per share data) For the Three Months Ended September 30, For the Nine Months Ended September 30, 2023 2022 2023 2022 Grant Revenue $1,240 $73 $3,600 $73 Operating expenses: Research and development 2,493 2,945 6,896 7,560 General and administrative 1,998 2,222 6,165 6,653 Total operating expenses 4,491 5,167 13,061 14,213 Loss from operations (3,251) (5,094) (9,461) (14,140) Other income (expense): Interest income 119 48 290 74 Interest expense (87) (173) (333) (552)Loss on disposal of property and equipment — — (2) — Change in fair value of liability instruments — — — 1 Total other income (expense) 32 (125) (45) (477)Net loss $(3,219) $(5,219) $(9,506) $(14,617) Net loss per share, basic and diluted $(1.00) $(2.85) $(3.54) $(9.22)Basic and diluted weighted average shares used in calculating net loss per share attributable to common stockholders 3,225,351 1,829,444 2,688,232 1,585,946 PLUS THERAPEUTICS, INC.CONDENSED STATEMENTS OF CASH FLOWS(UNAUDITED)(In thousands) For the Nine Months Ended September 30, 2023 2022 Cash flows used in operating activities: Net loss $(9,506) $(14,617)Adjustments to reconcile net loss to net cash used in operating activities: Depreciation and amortization 477 460 Amortization of deferred financing costs and debt discount 160 309 Stock issued for research and development 75 — Loss on disposal of property and equipment 2 — Stock-based compensation expense 428 476 Change in fair value of derivative instruments — (1)Amortization of operating lease right-of-use assets 86 66 Increases (decreases) in cash caused by changes in operating assets and liabilities: Grant receivable (91) 73 Other assets 3,190 642 Accounts payable and accrued expenses (4,061) 1,955 Change in operating lease liabilities (87) (101)Deferred grant liability (1,643) — Net cash used in operating activities (10,970) (10,738) Cash flows used in investing activities: Purchases of property and equipment (118) (381)Purchase of intangible assets — (117)In process research and development acquired — (250)Net cash used in investing activities (118) (748) Cash flows from financing activities: Principal payments of term loan obligation (1,206) (1,206)Proceeds from sale of common stock, net of offering cost of $0.2 million 5,180 14,558 Net cash provided by financing activities 3,974 13,352 Net increase (decrease) in cash and cash equivalents (7,114) 1,866 Cash and cash equivalents at beginning of period 18,120 18,400 Cash and cash equivalents at end of period $11,006 $20,266 Supplemental disclosure of cash flows information: Cash paid during period for: Interest $186 $248 Supplemental schedule of non-cash investing and financing activities: Unpaid offering cost $1 $68 Right-of-use assets acquired by assuming operating lease liabilities $71 $— Common stock issued in payment for in process research and development $75 $—

ROOT, Switzerland--(BUSINESS WIRE)-- NovoCure Ltd. (NASDAQ: NVCR) today announced it has entered into an agreement with Stanford University to establish the Stanford School of Medicine Tumor Treating Fields (TTFields) Research Program. The program is intended to support both preclinical studies and clinical trials with TTFields, electric fields that exert physical forces to kill cancer cells via a variety of mechanisms. “We look forward to collaborating with Stanford Medicine to establish a research framework for preclinical and clinical development of TTFields,” said Moshe Giladi, Ph.D., Novocure’s Chief Science Officer. “Together, we will enable new discoveries into the mechanisms and treatment possibilities of TTFields, including new indications and uses of TTFields along with other therapies. We will pursue our mission to extend survival in some of the most aggressive forms of cancer while enhancing the education and scholarship of future clinicians and scientists.” To enable novel preclinical research, Novocure will provide Stanford Medicine investigators with its portfolio of proprietary inovitro™, inovitro Live™ and inovivo™ TTFields preclinical systems which allows for exploring a vast range of research questions, starting from the fundamental physical mode of action all the way to most complex systemic biological outcomes. In addition, Novocure will provide devices for clinical studies. At present, Stanford Medicine is a site for four clinical studies of TTFields therapy. This includes TRIDENT, a Novocure-sponsored multi-center study of TTFields therapy given concomitantly with radiation therapy and temozolomide in newly diagnosed glioblastoma (GBM) patients, in which Stanford University Cancer Institute is one of 132 participating study centers. Investigator-sponsored trials at Stanford include multiple investigational uses, including a study of TTFields therapy for children with high-grade glioma or ependymoma and TTFields therapy for children with diffuse intrinsic pontine glioma, a study of TTFields therapy with hypofractionated chemoradiotherapy in newly diagnosed GBM, and a study of TTFields therapy with bevacizumab in treating patients with recurrent or progressive Grade 2 or 3 meningioma. In addition, Stanford is home to a preclinical study of the efficacy of TTFields in medulloblastoma. “With this initiative, we will establish a research infrastructure for preclinical and clinical development of TTFields and of potential new indications and combination therapies using TTFields,” said Michael Lim, M.D., Chair of the Department of Neurosurgery at Stanford Medicine. “We hope that this collaboration will educate, bring together, and inspire a diverse group of leaders in medicine and science who will help further our mission to deliver outstanding patient-centered care and improve human health through discovery, innovation, and education.” About Tumor Treating Fields Therapy Tumor Treating Fields (TTFields) are electric fields that exert physical forces to kill cancer cells via a variety of mechanisms. TTFields do not significantly affect healthy cells because they have different properties (including division rate, morphology, and electrical properties) than cancer cells. The multiple, distinct mechanisms of TTFields therapy work together to selectively target and kill cancer cells. Due to its multimechanistic actions, TTFields therapy can be added to cancer treatment modalities in approved indications and demonstrates enhanced effects across solid tumor types when used with chemotherapy, radiotherapy, immune checkpoint inhibition, or targeted therapies in preclinical models. TTFields therapy provides clinical versatility that has the potential to help address treatment challenges across a range of solid tumors. To learn more about Tumor Treating Fields therapy and its multifaceted effect on cancer cells, visit tumortreatingfields.com. About Novocure Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer through the development and commercialization of its innovative therapy, Tumor Treating Fields. Novocure’s commercialized products are approved in certain countries for the treatment of adult patients with glioblastoma, malignant pleural mesothelioma and pleural mesothelioma. Novocure has ongoing or completed clinical studies investigating Tumor Treating Fields in brain metastases, gastric cancer, glioblastoma, liver cancer, non-small cell lung cancer, pancreatic cancer and ovarian cancer. Headquartered in Root, Switzerland and with a growing global footprint, Novocure has regional operating centers in Portsmouth, New Hampshire and Tokyo, as well as a research center in Haifa, Israel. For additional information about the company, please visit Novocure.com and follow @Novocure on LinkedIn and Twitter. Forward-Looking Statements In addition to historical facts or statements of current condition, this press release may contain forward-looking statements. Forward-looking statements provide Novocure’s current expectations or forecasts of future events. These may include statements regarding anticipated scientific progress on its research programs, clinical study progress, development of potential products, interpretation of clinical results, prospects for regulatory approval, manufacturing development and capabilities, market prospects for its products, coverage, collections from third-party payers and other statements regarding matters that are not historical facts. You may identify some of these forward-looking statements by the use of words in the statements such as “anticipate,” “estimate,” “expect,” “project,” “intend,” “plan,” “believe” or other words and terms of similar meaning. Novocure’s performance and financial results could differ materially from those reflected in these forward-looking statements due to general financial, economic, environmental, regulatory and political conditions as well as issues arising from the COVID-19 pandemic and other more specific risks and uncertainties facing Novocure such as those set forth in its Annual Report on Form 10-K filed on February 23, 2023, and subsequent flings with the U.S. Securities and Exchange Commission. Given these risks and uncertainties, any or all of these forward-looking statements may prove to be incorrect. Therefore, you should not rely on any such factors or forward-looking statements. Furthermore, Novocure does not intend to update publicly any forward-looking statement, except as required by law. Any forward-looking statements herein speak only as of the date hereof. The Private Securities Litigation Reform Act of 1995 permits this discussion.