On July 8, 2025 Norgine, a leading European specialty pharmaceutical company, reported that Swissmedic has approved the registration of IFINWIL (eflornithine) as monotherapy for the treatment of paediatric patients from one year with high-risk neuroblastoma (HRNB) (Press release, Norgine, JUL 8, 2025, View Source [SID1234654290]).

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"This milestone marks a further regulatory approval of IFINWIL for high-risk neuroblastoma, following approvals in the US, Israel and Australia," said Jörg Plessl, VP and Head of Global Regulatory Affairs at Norgine. "We deeply appreciate Swissmedic and the other Project Orbis partners for their keen awareness of the distinct challenges associated with paediatric oncology. Their agility in utilizing adaptive regulatory pathways is crucial because the stakes are incredibly high when it comes to children’s lives. In these situations, speed and flexibility are not just advantageous, they can make all the difference."

"By collaborating with international regulatory partners through Project Orbis, we are advancing our strategy to broaden access to innovative therapies for paediatric patients in Europe and ANZ." said Dr David Gillen, Chief Medical Officer at Norgine. "At Norgine, we’re proud to take on some of the most complex disease areas, driven by our mission to deliver meaningful treatments where they’re needed most."

HRNB is a rare but aggressive form of cancer, predominantly affecting children and most commonly presenting in the first 5 years of life.2 Globally, neuroblastoma affects approximately 10.2 children per million annually under age 15, with around 50% of cases classified as high-risk at diagnosis.3,4 Neuroblastoma originates in the body’s nerve cells (neuroblasts) and typically presents as a primary tumour in the adrenal glands.5 It is considered an aggressive tumour because it often spreads to other parts of the body (metastasises). In most cases, it has spread by the time it is diagnosed.5

Despite intensive multimodal therapy, the 5-year survival rate for HRNB remains below 50%, compared to up over 95% for infants with low-risk disease.2,6 Neuroblastoma accounts for approximately 15% of all paediatric cancer-related deaths, underscoring the urgent need for more effective treatment options.7

About IFINWIL
IFINWIL has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy.8 IFINWIL is a therapy that blocks an enzyme called ornithine decarboxylase (ODC) responsible for producing polyamines, which are important to tumour growth and development.9

High-Risk Neuroblastoma (HRNB) Treatment Background
Children diagnosed with HRNB undergo an intense treatment regimen that still leaves them vulnerable to relapse and death.10 Although there have been some improvements in survival, the prognosis for children with high risk neuroblastoma is still poor with around 80% of high risk relapses occuring within 2 years from diagnosis, and long-term survival remains low, with only about 15% surviving more than 5 years post-relapse.11,12,13 Avoiding relapse is key to long-term survival, and until now, there have been no approved therapies for the post maintenance treatment period in major markets outside of the United States.14

About Project Orbis
Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health).

In April 2024, Norgine submitted an application for approval of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. In April 2025, the Australian Therapeutic Goods Administration (TGA) approved the registration of IFINWIL for the treatment of adults and paediatric patients with HRNB, who have responded to prior multiagent, multimodality therapy.

These regulatory milestones support Norgine’s efforts to deliver patient access to IFINWIL and bring a further treatment option in the field of paediatric oncology.

On July 8, 2025 IRIC and IRICoR reported the start of a Phase 1 clinical trial of its small molecule therapy for solid tumors licensed to Ipsen (Press release, Université de Montréal, JUL 8, 2025, View Source [SID1234654289]). This marks a major milestone in the strategic collaboration between the partners, which began in May 2020 with a research and option agreement followed by a licensing agreement in February 2023.

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The small molecule clinical candidate, now known as IPN01195, is a RAF inhibitor, an important target in a signaling pathway involved in cancer cell growth and proliferation. It was originally discovered and developed by the teams of Professors Marc Therrien and Anne Marinier at the Université de Montréal’s Institute for Research in Immunology and Cancer (IRIC) and has shown promising activity in preclinical models of various solid tumors.

"We are extremely proud to see our novel small molecule therapy reaching this important clinical milestone. This is another example of the efficiency and innovative approach of IRIC’s collaborative drug discovery research model," said Dr. Anne Marinier, Director of the Drug Discovery Unit at IRIC. "This marks a major step forward for our multidisciplinary teams of biologists and chemists, but more importantly, it brings us closer to delivering a potential new treatment option for patients facing advanced cancers," said Dr. Marc Therrien, Principal Investigator, Intracellular Signalling Research Unit and Chief Executive Officer, IRIC. "We are delighted by the commitment and progress of our partner Ipsen, and we look forward to the continued development of this promising therapy," concluded Dr. Marinier.

"The initiation of this clinical trial marks a major validation of our business development efforts," said Elizabeth Douville, CEO, IRICoR. "It demonstrates our ability to translate novel science into programs that attract world-class partners and progress into the clinic. With additional first-in-class and best-in-class molecules targeting key cancer pathways in our pipeline, we are actively exploring new strategic collaborations and raising capital to accelerate development. This is an exciting inflection point for IRICoR as we continue to deliver both scientific and commercial value," added Elizabeth Douville.

Under the terms of the agreement, the Université de Montréal granted Ipsen exclusive rights to develop and commercialize the compound globally. The University and IRICoR are eligible to receive development and commercial milestone payments, as well as royalties on net sales.

On July 8, 2025 LakeShore Biopharma Co., Ltd (Nasdaq: LSB) (the "Company"), a global biopharmaceutical company dedicated to discovering, developing, manufacturing, and delivering new generations of vaccines and therapeutic biologics for infectious diseases and cancer, reported that it has entered into a share and warrant purchase agreement (the "Purchase Agreement") with an institutional investor for the issuance and sale of 16,987,542 ordinary shares of the Company, par value US$0.0002 per share, at US$0.883 per share, and 16,987,542 warrants, each entitling the institutional investor to purchase one ordinary share at an exercise price of US$1.079 at any time and from time to time during a 36-month period, in a private placement of US$15 million exempt from registration pursuant to Regulation S under the Securities Act of 1933, as amended (Press release, LakeShore Biopharma, JUL 8, 2025, View Source [SID1234654288]). The closing of the private placement is expected to take place as soon as possible upon agreement by the parties to the Purchase Agreement, subject to customary conditions precedent stipulated thereunder.

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This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities, in any state or jurisdiction in which such offer, solicitation, or sale of these securities would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On July 8, 2025 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system (CNS) cancers, reported the treatment of its initial patients in the Company’s ReSPECT-LM dose optimization trial for REYOBIQTM (rhenium Re186 obisbemeda) for the treatment of leptomeningeal metastases (LM) (Press release, Plus Therapeutics, JUL 8, 2025, View Source [SID1234654287]). The dose optimization trial builds on promising results from the Company’s completed Phase 1 single-dose escalation study, which demonstrated the feasibility of REYOBIQ for treating LM. The trial is designed in alignment with the FDA’s Project Optimus to identify the optimal dosing regimen that maximizes efficacy and safety.

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"Based on the promising, recently completed Phase 1 single-dose escalation study and lack of FDA approved therapies for LM, we expect trial enrollment to proceed rapidly in accordance with the trial protocol," said Andrew Brenner, M.D., Ph.D., Professor-Research, Departments of Medicine, Neurology, and Neurosurgery & S & B Kolitz/CTRC-Zachry Endowed Chair Neuro-Oncology Research, Mays Cancer Center at UT Health San Antonio. "Furthermore, assuming REYOBIQ continues its attractive safety profile, we expect that we will enroll all required patients and doses in Cohort 1 by the end of this year."

Patients and family members interested in enrolling in the ReSPECT-LM Dose Optimization Trial can learn more here.

ReSPECT-LM Dose Optimization Trial
The study aims to optimize treatment dosing for maximum efficacy and safety, with the primary objectives of determining the safety and tolerability of multiple REYOBIQ doses administered via intraventricular catheter at defined intervals in patients with LM from any primary solid tumor cancer, and to identify both the maximum tolerated dose and minimum effective dose. Previously announced details of the study, including primary, secondary, and exploratory endpoints, can be found here.

The dose optimization study builds on encouraging results from the Company’s previously announced Phase 1 trial, which showed a single dose of REYOBIQ delivered up to an average absorbed dose of >250 Gy to the cranial subarachnoid space. It also demonstrated that 5 of 7 patients achieving an over 80% reduction in LM tumor cells in the cerebrospinal fluid survived at least one year post-treatment. Additional details can be found here.

The Company anticipates presenting these data and additional information from the completed single-dose escalation trial at the upcoming SNO/ASCO CNS Metastases Conference on August 14-16, 2025, in Baltimore, MD. The company will also request an End of Phase 1 Type B meeting with the FDA to align on the clinical development plan and the design of a potential registrational trial.

The ReSPECT-LM dose optimization trial benefits from a $17.6 million grant from the Cancer Prevention & Research Institute of Texas (CPRIT), the second largest public funding source for cancer research in the world.

About LM
Leptomeningeal metastases (LM) are a rare but severe complication of advanced cancer, affecting the fluid-lined structures of the central nervous system. LM occurs in approximately 5% of patients with metastatic cancer, with breast cancer, lung cancer, and melanoma being the most common sources. Median survival is typically 2-6 months, and effective treatment options are limited, highlighting the urgent need for novel therapies.

About REYOBIQ (rhenium Re186 obisbemeda)
REYOBIQ (rhenium Re186 obisbemeda) is a novel injectable radiotherapy specifically formulated to deliver direct targeted high dose radiation in CNS tumors in a safe, effective, and convenient manner to optimize patient outcomes. REYOBIQ has the potential to reduce off target risks and improve outcomes for CNS cancer patients, versus currently approved therapies, with a more targeted and potent radiation dose. Rhenium-186 is an ideal radioisotope for CNS therapeutic applications due to its short half-life, beta energy for destroying cancerous tissue, and gamma energy for real-time imaging. REYOBIQ is being evaluated for the treatment of recurrent glioblastoma, leptomeningeal metastases, and pediatric brain cancer in the ReSPECT-GBM, ReSPECT-LM, and ReSPECT-PBC clinical trials. ReSPECT-GBM is supported by an award from the National Cancer Institute (NCI), part of the U.S. National Institutes of Health (NIH), and ReSPECT-LM is funded by a three-year $17.6M grant by the Cancer Prevention & Research Institute of Texas (CPRIT). The Company’s ReSPECT-PBC clinical trial for pediatric brain cancer is supported by a $3 million grant from the U.S. Department of Defense’s Peer Reviewed Cancer Research Program.

About Cancer Prevention & Research Institute of Texas (CPRIT)
The Cancer Prevention & Research Institute of Texas (CPRIT) is a $6 billion cancer research and prevention initiative legislated in Texas. CPRIT accepts applications and award grants for a wide variety of innovative cancer-related research and product development and for the delivery of evidence-based cancer prevention programs and services by public and private entities located in Texas. CPRIT is the largest state cancer research investment in the history of the United States and the second largest cancer research and prevention program in the world with a three-part mission, established by the Texas Legislature, to invest in the research prowess of Texas universities and research organizations; create and expand life science infrastructure across the state; and expedite innovation in research and enhance the potential of breakthroughs in prevention and cures.

On July 8, 2025 Lixte Biotechnology Holdings, Inc. (NASDAQ: LIXT) (the "Company"), a clinical stage pharmaceutical company, reported the closing of a registered direct offering with accredited investors for the purchase and sale of approximately $1.5 million of shares of Common Stock (or Pre-Funded Warrants) (Press release, Lixte Biotechnology, JUL 8, 2025, View Source [SID1234654286]).

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The offering consisted of the sale of an aggregate of 974,026 shares of Common Stock (or Pre-funded Warrants in lieu thereof). The public offering price per share of Common Stock was $1.54 (or $1.53999 for each Pre-Funded Warrant, which is equal to the offering price per share of Common Stock sold in the offering minus an exercise price of $0.00001). The Pre-Funded Warrants are immediately exercisable and may be exercised at any time until exercised in full.

Aggregate gross proceeds to the Company were approximately $1.5 million. The transaction closed on July 8, 2025. The Company expects to use the net proceeds from the offering, together with its existing cash, for general corporate purposes and working capital.

Spartan Capital Securities, LLC acted as exclusive placement agent for the offering. TroyGould PC acted as counsel to the Company. Kaufman & Canoles, P.C. acted as counsel to Spartan Capital Securities, LLC.

The registered direct offering was made pursuant to an effective shelf registration statement on Form S-3 (No. 333-278874) previously filed with the U.S. Securities and Exchange Commission (SEC) and declared effective by the SEC on May 2, 2024. A final prospectus supplement and accompanying prospectus describing the terms of the proposed offering will be filed with the SEC and will be available on the SEC’s website located at www.sec.gov. Electronic copies of the final prospectus supplement and the accompanying prospectus may be obtained, when available, by contacting Spartan Capital Securities, LLC, Attention: Prospectus Department, 45 Broadway, 19th Floor, New York, NY 10006, by e-mail at [email protected]. Interested parties should read in their entirety the prospectus supplement and the accompanying prospectus and the other documents that the Company has filed with the SEC that are incorporated by reference in such prospectus supplement and the accompanying prospectus, which provide more information about the Company and such offering.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.