On August 28, 2025 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) reported that the European Medicines Agency (EMA) has granted Orphan Designation for ADX-2191 (methotrexate intravitreal injection, USP) for the treatment of primary large B-Cell lymphomas of immune-privileged sites, including primary vitreoretinal lymphoma (Press release, Aldeyra Therapeutics, AUG 28, 2025, View Source [SID1234655573]). There is currently no approved treatment for patients with primary vitreoretinal lymphoma, a rare, aggressive, high-grade cancer that affects approximately 100 to 200 people per year in the European Union.

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"Primary vitreoretinal lymphoma is a potentially fatal cancer that today is treated with off-label compounded formulations of methotrexate that are injected into the eye," stated Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. "The receipt of Orphan Designation from the EMA, in conjunction with the previously announced Orphan Drug Designation from the U.S. Food and Drug Administration, is another important step forward for our retinal disease program, complementing our ongoing clinical development of ADX-2191, a novel, vitreous-compatible formulation of methotrexate that is specifically designed for intraocular injection."

A proposed clinical trial, which received Special Protocol Assessment agreement from the U.S. Food and Drug Administration, is designed to compare cancer cell clearance after 30 days of therapy in up to 20 patients following 1:1 randomization to receive either a single intraocular injection or eight intraocular injections of ADX-2191. The frequency of methotrexate injections has been linked to cancer cell clearance in patients with primary vitreoretinal lymphoma,1 and approximately five injections on average has been observed to achieve cancer cell clearance.2 The clinical trial is expected to begin in the second half of 2025 and conclude in 2026.

The EMA grants orphan designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare, life-threatening, or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity in the European Union.

About ADX-2191

ADX-2191 (methotrexate intravitreal injection, USP) is a sterile, non-compounded intravitreal formulation of methotrexate for the potential treatment of specific rare retinal diseases, including primary vitreoretinal lymphoma and retinitis pigmentosa. The ADX-2191 intravitreal formulation is preservative-free, is designed to be vitreous-compatible, and is optimized for excipient composition, viscosity, density, tonicity, pH, concentration, and volume of administration. ADX-2191 received FDA Orphan Drug Designation for the treatment of primary vitreoretinal lymphoma and retinitis pigmentosa, and EMA Orphan Designation for the treatment of inherited retinal dystrophies of the rod-dominant phenotype, including retinitis pigmentosa, and the treatment of primary large B-cell lymphomas of immune-privileged sites, including primary vitreoretinal lymphoma.

About Primary Vitreoretinal Lymphoma

Primary vitreoretinal lymphoma is a rare, aggressive, and potentially fatal retinal cancer that is diagnosed in approximately 200 to 600 patients in the United States and 100 to 200 patients in the European Union per year. The median survival for newly diagnosed patients is less than five years. No approved treatments are currently available, though intravitreal injection of compounded methotrexate represents the current standard of care.

On August 28, 2025 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported that members of the Company’s senior management team will participate in the following investor conferences in September 2025 (Press release, Zai Laboratory, AUG 28, 2025, View Source [SID1234655572]):

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Cantor Global Healthcare Conference
Presentation: Thursday, September 4, 2025, 10:20 a.m. ET
Location: New York, NY

Morgan Stanley 23rd Annual Global Healthcare Conference
Fireside Chat: Tuesday, September 9, 2025, 2:35 p.m. ET
Location: New York, NY

Live webcasts will be available on the Investor Relations page of Zai Lab’s website at ir.zailaboratory.com/webcasts-presentations and archived replays will be available for up to 90 days following the completion of the events.

On August 28, 2025 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and precision medicine, reported the activation of the NRG Oncology trial, ARCHER (NRG-GU015), a randomized, phase III study in muscle-invasive bladder cancer (MIBC) (Press release, Natera, AUG 28, 2025, View Source [SID1234655571]). The study is evaluating whether a shorter course of radiation can achieve outcomes comparable to the current standard of care. It prospectively incorporates Signatera, Natera’s personalized, tumor-informed molecular residual disease (MRD) test, as a pre-specified secondary endpoint.

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MIBC accounts for approximately one-quarter of all bladder cancer cases in the United States and typically requires aggressive treatment.1 Bladder-sparing treatment options are increasing in patient care, and there is a high interest in identifying therapeutic approaches that can achieve promising clinical outcomes while maximizing patient quality of life.2

The ARCHER trial is sponsored by NRG Oncology through the National Cancer Institute’s National Clinical Trials Network. It is expected to have enrollment from more than 100 sites across the U.S. and Canada. Signatera will be collected and reported to investigators during treatment and follow-up at defined timepoints. The test will enable investigators to evaluate real-time circulating-tumor DNA (ctDNA) clearance patterns in each treatment arm as a predictive marker of treatment response and recurrence. Natera will also assess urine tumor DNA as an exploratory endpoint. Signatera has been clinically validated in MIBC to detect recurrence months before standard imaging and to independently predict recurrence risk following curative-intent therapy.3

"With Signatera as a key assessment in the ARCHER trial, we aim to evaluate ctDNA dynamics to detect early molecular signs of disease recurrence in real time and refine our clinical surveillance toolbox beyond imaging and cystoscopy," said Catherine Spina, M.D., Ph.D., co-chair of translational science for the study. "By utilizing ctDNA to monitor treatment response and recurrence, we hope to improve clinical outcomes for patients with MIBC."

"By exploring whether we can safely reduce the intensity and duration of therapy without compromising outcomes, ARCHER has the potential to ease patient burden and improve quality of life," said Minetta Liu, M.D., chief medical officer of oncology at Natera. "The use of serial Signatera testing in ARCHER represents an important step forward in determining how MRD insights can guide more precise patient management."

The launch of ARCHER will expand Natera’s breadth of MIBC clinical evidence, where data has proven how ctDNA testing can benefit patients. That includes the recent read-out of positive topline results from the randomized phase III IMVigor011 trial, which demonstrated Signatera’s ability to predict adjuvant immunotherapy benefit in patients post-cystectomy.

On August 28, 2025 TriSalus Life Sciences Inc. (Nasdaq: TLSI) ("TriSalus" or the "Company"), an oncology company integrating novel delivery technology with standard of care therapies to transform treatment for patients with solid tumors, and its investigational immunotherapeutic to transform treatment for patients with solid tumors, reported that Mary Szela, Chief Executive Officer and President, and David Patience, Chief Financial Officer, will participate in the following investor conferences in September (Press release, TriSalus Life Sciences, AUG 28, 2025, View Source [SID1234655570]):

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Cantor Global Healthcare Conference 2025 in New York, NY
Management will participate in one-on-one meetings and participate in a fireside chat on Friday, September 5, at 9:35 a.m. ET. Click here for a link to the live webcast.

Lake Street Capital Markets 9th Annual Best Ideas Growth Conference in New York, NY
Management will participate in one-on-one meetings on Thursday, September 11.

A webcast replay of the fireside chat will be available for 90 days following the presentation in the Events section of the TriSalus Investor website at www.investors.trisaluslifesci.com.

On August 28, 2025 Novotech, a globally recognized full-service clinical research organization (CRO), reported to have partnered with Chia Tai Tianqing Pharmaceutical Group Co., Ltd. (CTTQ Pharma) in achieving a significant regulatory milestone: the approval of Anlotinib Hydrochloride Capsules for a new indication as a first-line treatment for unresectable locally advanced or metastatic soft tissue sarcoma (STS) in combination with chemotherapy (Press release, Jiangsu Chia-tai Tianqing, AUG 28, 2025, View Source [SID1234655569]). This approval marks the ninth indication for Anlotinib in China and represents the world’s first official approval of an Anlotinib-chemotherapy combination for first-line treatment of advanced or metastatic STS.

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Previously recommended as a second-line monotherapy by the Chinese Society of Clinical Oncology (CSCO) guidelines, Anlotinib was the only Grade I targeted therapy for STS. This new indication represents a significant advancement for patients who have not previously received systemic treatment, expanding access to more effective and targeted first-line therapeutic options.

Novotech provided a comprehensive suite of regulatory-compliant biostatistics services in support of the development program, including protocol review and design, statistical analysis plans, CDISC datasets generation, and post-database lock outputs of TFL (Tables, Figures, and Listings). These contributions enhanced data transparency, improved result interpretability, and played a critical role in the successful NDA submission.

The Phase III study demonstrated compelling results:

Anlotinib combination therapy improved median progression-free survival by 5.6 months.
Delivered an objective response rate of 17.8% compared to 2.9% in the control group.
The disease control rate climbed to 79.3%, underscoring the clinical potential of the new regimen.
Novotech’s expert biostatisticians and programmers provide end-to-end services, from protocol design and statistical consultation to comprehensive reporting, supporting clinical programs from first-in-human through Phase IV.

Our collaboration with CTTQ Pharma reflects a shared commitment to advancing outcomes for patients facing difficult-to-treat cancers. The expanded use of Anlotinib in combination with chemotherapy opens new possibilities for advancing care in soft tissue sarcoma and highlights the role Novotech’s partnership model plays in shaping the future of oncology research.