On September 29, 2026 GT Medical Technologies, a company focused on improving the lives of patients with brain tumors, reported the publication of the results of the randomized, multicenter ROADS trial (NCT04365374) as a Rapid Communication in the Journal of Clinical Oncology (JCO), the official journal of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). Rapid Communication is a discretionary designation JCO’s editors may grant to manuscripts accepted, reserved for findings of exceptional significance.2 Recommended manuscripts post online within days of acceptance and are openly accessible upon publication, well ahead of standard timelines, so that practice-changing data reaches oncologists faster than standard publication timelines allow.2 The data demonstrate that patients with newly diagnosed operable brain metastases treated with tumor removal surgery and GammaTile cesium-131 collagen tile-based radiation therapy (TBRT) experienced lower local tumor recurrence and lived significantly longer compared to the standard-of-care approach of surgery followed by post-operative stereotactic radiation therapy (SRT).

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Study authors conclude GammaTile should be considered a standard-of-care option
In the manuscript’s conclusion, the study authors state that GammaTile should be considered a standard-of-care option for patients with newly diagnosed brain metastases. This conclusion is based on several compelling pieces of evidence, including:1

ROADS is a multicenter randomized phase 3 trial with longer median follow-up than the studies that established post-operative SRT as standard-of-care
GammaTile was superior on both co-primary endpoints
The reduction in surgical bed recurrence was substantial
The efficacy gain came without an increase in radiation necrosis, neurocognitive decline, or quality-of-life deterioration
"The Rapid Communication publication in the Journal of Clinical Oncology reflects how significant these findings are for patients with newly diagnosed brain metastases," said Jeffrey Weinberg, MD, professor of Neurosurgery at The University of Texas MD Anderson Cancer Center, co-lead investigator of the ROADS trial, and first author on the publication. "Standard-of-care treatment for this disease has always focused on controlling the tumor in the brain so patients can get back to fighting their primary cancer, and TBRT (GammaTile) does that more effectively than existing treatment options. By initiating radiation at the moment of surgery, TBRT closes the gap between resection and radiation treatment for the operable tumor entirely, and this randomized evidence supports it as a new standard-of-care option that gives patients that certainty from day one."

How does GammaTile address limitations of the post-operative SRT pathway?
GammaTile, an FDA-cleared, bioabsorbable collagen implant embedded with 4 cesium-131 radiation sources, is placed directly in the surgical cavity at the time of tumor removal. Radiation begins immediately when surgery ends. The current standard-of-care approach for operable brain metastases is surgery to remove the tumor, followed by post-operative SRT multiple weeks later, once the patient has recovered from surgery. That time gap is not only a source of anxiety for patients and families waiting to know if treatment for the operable brain tumor is complete; it also creates a window in which any tumor cells left behind after surgery may regrow before radiation begins.

In the ROADS trial, 17.8% of patients randomized to standard-of-care did not receive their prescribed post-operative SRT,1 consistent with published data showing that 20-27% of patients do not complete their full prescribed course of post-operative SRT,3,4 a limitation of the post-operative SRT strategy itself. Because GammaTile is placed at the time of surgery, radiation begins immediately, eliminating the risk of prescribed radiation going undelivered.1 Measured against standard-of-care, GammaTile demonstrated superior efficacy on both co-primary endpoints and significantly longer overall survival.1

While delays in or failure to receive post-operative SRT are a reality of current standard-of-care, the study authors also examined how GammaTile compared when the analysis was limited to only those patients in each arm who received their prescribed radiation. Even in this comparison, GammaTile’s superiority in local tumor control and surgical bed recurrence-free survival persisted.

"That many patients do not receive post operative SRT appears to be an underappreciated issue amongst physicians caring for these patients, but the benefit of TBRT (GammaTile) is not solely increased treatment completion," said Thomas H. Beckham, MD, PhD, Associate Professor of CNS Radiation Oncology at The University of Texas MD Anderson Cancer Center and corresponding author on the publication. "The trial was designed to compare resection and TBRT versus resection and post-operative SRT, a pathway in which some patients do not ultimately receive their planned radiation. But even when we analyzed only those patients in each arm who did receive their assigned radiation, TBRT remained superior on both co-primary endpoints. That tells us this advantage isn’t explained solely by guaranteed radiation delivery. TBRT immediately begins concentrating a higher therapeutic dose of radiation right at the resection cavity, providing a continuous dose over the next few weeks, while minimizing radiation to surrounding healthy brain tissue, a fundamentally different biological approach than a delayed course of radiation delivered from outside the body."

How did patients benefit from GammaTile in the ROADS trial?
The phase 3 trial randomized 230 patients with operable brain metastases across 32 centers to treatment with resection plus GammaTile or the standard-of-care approach of resection followed by post-operative SRT.1

GammaTile showed superior performance in the study’s co-primary endpoints1
GammaTile demonstrated a lower 12-month cumulative incidence of surgical bed recurrence (SBR) compared with standard of care (1.3% vs. 15.4%, HR: 0.07, 95% CI: 0.01–0.56; p=0.012)
This represents the lowest tumor recurrence rate reported in a published randomized controlled trial of newly diagnosed operable brain metastases
GammaTile reduced the combined risk of tumor recurrence or death by more than 50% compared to standard-of-care (HR: 0.48, 95% CI: 0.30-0.76, p=0.002)1
GammaTile provided benefit in key secondary endpoints1
The median overall survival for patients who received GammaTile was more than twice as long as that of patients who received standard-of-care: median OS was 42.5 months compared to 17.6 months with standard-of-care (HR: 0.59, 95% CI: 0.37-0.96, p=0.032)1
GammaTile demonstrated significant gains in efficacy with no increase in safety or patient reported health status concerns1
Rates of grade 3 or higher treatment-related side effects remained low and comparable between arms (20.0% GammaTile vs. 21.7% standard-of-care)1
Functional status, quality of life, time to distant brain failure, adverse events, leptomeningeal disease, and radiation necrosis were similar in both arms1
In an exploratory composite analysis, GammaTile reduced the combined risk of tumor recurrence or radiation necrosis by more than 70% compared to standard-of-care (HR: 0.28, 95% CI: 0.12-0.66, p=0.004)1
How will publication of the ROADS results change clinical practice?
"The data from this multi-institutional randomized trial provide a high level of evidence demonstrating the superiority of resection plus GammaTile over the current standard-of-care. We expect that this rigorous peer-reviewed publication will give physicians confidence in adopting GammaTile as a first-line treatment for patients with newly diagnosed surgical brain metastases," explained Dr. Michael Garcia, MD, MS, Chief Medical Officer of GT Medical Technologies. "Broader adoption of GammaTile can enable more patients to live longer without recurrence, an important advance in this indication."

"As a company founded by brain tumor specialists, GT Medical recognizes the importance of randomized, controlled studies in driving the evolution of clinical practice," said Per Langoe, Chief Executive Officer of GT Medical Technologies. "The publication of the ROADS trial results is a key milestone for the company, the clinical community, and patients with newly diagnosed brain metastases. We are committed to generating robust evidence in additional brain tumor indications, such as through the ongoing BRIDGES Phase 3 trial for newly diagnosed glioblastoma and providing additional data to inform clinical decision-making."

What is tile-based radiation therapy (TBRT) with GammaTile?
Each GammaTile is about the size of a postage stamp (2 cm x 2 cm) and contains purposefully spaced seeds containing cesium-131 that emit low-dose rate radiation over several weeks.5 GammaTile is used to line the cavity that remains after the tumor has been removed, ensuring even distribution of radiation across the surface of the cavity and precise targeting of remaining tumor cells. This highly localized approach targets remaining cancer cells when they are at their lowest levels to help prevent tumor regrowth.5 The emitted radiation only travels a short distance, which maximizes dosing to the tumor cavity while minimizing radiation dose to surrounding healthy brain tissue.

(Press release, GT Medical Technologies, SEP 29, 2026, View Source [SID1234671164])

On September 29, 2026 XYone Therapeutics, Inc. ("XYone"), a clinical-stage biotechnology company, reported that the first patient has been dosed in the first-in-human Phase 1b/2 clinical trial of XYA02(also known as AT2021), the Company’s lead antibody-drug conjugate ("ADC") targeting MUC1-C.

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The multicenter, open-label study is evaluating XYA02 in patients with advanced, relapsed and/or refractory solid tumors. The study is designed to characterize the safety and tolerability of XYA02, identify appropriate dose levels for further clinical development, and assess pharmacokinetics, pharmacodynamics and preliminary antitumor activity.

The study is registered on ClinicalTrials.gov as NCT07670312, "Evaluation of XYA02 in Patients with Advanced Solid Tumors."

"Dosing the first patient with XYA02 represents the culmination of several years of work to develop a differentiated therapeutic approach against MUC1-C," said Anshu Goyal, Chief Executive Officer and Co-Founder of XYone Therapeutics. "This marks a significant milestone in XYone’s transition to a clinical stage company."

The clinical study includes patients with advanced solid tumors in non-small cell lung, ovarian, gastric/GEJ & colorectal cancers. The study will initially assess escalating doses of XYA02, followed by further evaluation in selected tumor types based on emerging safety, pharmacokinetic, biomarker and antitumor activity data.

"MUC1 has been recognized as an important pan-cancer target for decades, but the biology of targeting the specific domain (MUC1-C) addresses several vexing issues," said Dr. Ravi Jasuja, Co-Founder and Chief Scientific Officer of XYone Therapeutics. "MUC1-C is expressed across multiple solid tumors and, unlike the shed extracellular MUC1-N domain, remains tumor anchored. Compared to MUC1-C, heterogeneous glycosylation of MUC1-N further compounds the complexity in therapeutic targeting of the extracellular domain. Accordingly, MUC1-C targeting with an antibody-drug conjugate provides a compelling rationale for an improved therapeutic index."

"We are excited that the U.S. Food and Drug Administration has granted XYA02 Orphan Drug Designation (ODD) for the treatment of both pancreatic cancer and gastric cancer, with additional applications pending in other indications," said Dr. Surender Kharbanda, Chief Operating Officer of XYone Therapeutics. "These designations recognize the potential of XYA02 to treat patients with these difficult-to-treat solid tumors."

About XYA02
XYA02 is a novel MUC1-C targeting ADC for the treatment of multiple solid tumors. It was engineered with a novel, proprietary antibody conjugated with Exatecan payload.

XYA02 is an investigational drug and has not been approved by the U.S. Food and Drug Administration, the Australian Therapeutic Goods Administration, or any other regulatory authority. Its safety and efficacy have not been established.

(Press release, XYone Therapeutics, SEP 29, 2026, View Source [SID1234671163])

On September 29, 2026 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a biopharmaceutical company dedicated to the discovery, development, manufacturing, and commercialization of innovative medicines for the treatment of oncology, autoimmune, cardiovascular and metabolic, ophthalmology, and other major diseases, reported updated Phase 1 clinical data for IBI3003, a novel trispecific antibody targeting GPRC5D, BCMA, and CD3, in patients with relapsed or refractory multiple myeloma (R/R MM) in an oral presentation at the 23rd International Myeloma Society (IMS) Annual Meeting 2026. Based on the results of the Phase 1 dose optimization study, the recommended Phase 2 dose (RP2D) of IBI3003 was determined to be 360 μg/kg. At the RP2D, IBI3003 demonstrated rapid, deep, and durable response signals, particularly in heavily pretreated, high-risk patients with extramedullary disease.

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IBI3003 is a novel trispecific antibody targeting G protein-coupled receptor C5D (GPRC5D), B-cell maturation antigen (BCMA), and CD3. Its dual-targeting design against BCMA and GPRC5D is intended to overcome tumor escape caused by the loss or downregulation of a single tumor antigen. In preclinical mouse models, IBI3003 demonstrated superior in vivo anti-tumor activity compared with marketed benchmark bispecific antibodies targeting GPRC5D/CD3 or BCMA/CD3, with particularly prominent tumor-killing activity in in vitro cell models with low expression of BCMA and GPRC5D. Currently, Innovent is conducting Phase 1/2 clinical trials of IBI3003 in China, Australia, and the United States (NCT06083207 and NCT07336472) to evaluate its safety, tolerability, and preliminary efficacy in patients with R/R MM. In parallel, Innovent has advanced the Phase 3 TriadicMM-1 study (NCT07623798) in China for IBI3003 in patients with R/R MM who have received 1 to 4 prior lines of therapy. ‌ In addition, the Phase II study of IBI3003 combined with CD38 monoclonal antibody for frontline MM is also ongoing (NCT07764861).

The updated data were from the Phase 1 clinical study NCT06083207, which enrolled eligible patients with R/R MM who had failed at least two prior lines of therapy, including at least one proteasome inhibitor (PI), one immunomodulatory drug (IMiD), and one anti-CD38-based therapy, and who had relapsed or were refractory to their last anti-myeloma regimen. Patients with prior BCMA- and/or GPRC5D-targeted therapy were also eligible for enrollment. IBI3003 was administered subcutaneously once weekly (QW). Patients who received continuous treatment for ≥6 months and achieved a partial response (PR) or better for ≥2 months could switch to once-every-two-weeks (Q2W) dosing for maintenance. To reduce the risk of cytokine release syndrome (CRS), 1 to 3 priming doses were incorporated into the study design.

A total of 102 patients were enrolled in this phase of the study in China and Australia, with doses ranging from 0.1 μg/kg to 800 μg/kg. The median patient age was 62 years (range: 40-88), 37.1% of patients were classified as high risk according to mSMART criteria, and 53.9% had ≥1 site of extramedullary disease (EMD). The median number of prior lines of therapy was 4 (range: 2-12). All patients had received at least three classes of therapy, including a PI, an IMiD, and an anti-CD38 antibody. Among them, 55.9% had received at least five classes of therapy, including at least two PIs, two IMiDs, and one anti-CD38 antibody; 33.3% had previously received anti-BCMA and/or anti-GPRC5D therapies; and 88.2% were refractory to their last treatment. As of the data cutoff date of June 30, 2026, the median follow-up duration was 6.65 months (range: 0.8-15.2).

Rapid, Deep, and Durable Responses Observed with IBI3003 at 360 μg/kg

Based on an integrated analysis of the efficacy, safety, and pharmacokinetic/pharmacodynamic (PK/PD) data from dose optimization cohorts receiving IBI3003 at 120, 360, and 540 μg/kg in the Phase 1 study, the RP2D was determined to be 360 μg/kg.

A total of 26 patients received IBI3003 at 360 μg/kg, with a median follow-up duration of 7.57 months. mPFS was not reached yet.

The overall response rate (ORR) was 84.6%, including 11 cases of stringent complete response (sCR), 4 cases of very good partial response (VGPR), and 7 cases of partial response (PR). The ORR was 94.7% among 19 patients who had received 2 to 4 prior lines of therapy, 87.5% among 8 patients with non-bone-related EMD, and 92.3% among 13 patients without EMD.
The median time to first response was 0.99 months (range: 0.9-8.3 months), the median time to first response of ≥VGPR was 1.87 months (range: 0.9-4.6 months), and the median time to first response of ≥CR was 2.79 months (range: 0.9-9.2 months).
Among patients who achieved CR or better as assessed by central laboratory next-generation sequencing (NGS) testing, the minimal residual disease (MRD) negativity rate was 100% (n=8). Three patients achieved MRD negativity after receiving only one treatment cycle.
Manageable Safety Profile of IBI3003 in Patients with R/R MM

Hematologic toxicities were the most common Grade ≥3 treatment-related adverse events (TRAEs), occurring primarily during the dose-escalation phase and being manageable and reversible.
The incidences of CRS and immune effector cell-associated neurotoxicity syndrome (ICANS) were 52% and 3.9%, respectively. All events were Grade 1-2 and resolved with treatment. Prophylactic use of tocilizumab may reduce the risk of CRS.
The incidence of infections was 42.2%, with Grade ≥3 infections reported in 26.5% of patients.
For GPRC5D target-related TEAEs involving the oral cavity, skin, and nails, no Grade ≥3 oral TEAEs were observed. Most skin and nail TEAEs were Grade 1-2, with only two patients experiencing Grade 3 rash.
Potent and Sustained Pharmacodynamic Responses Observed with IBI3003 in Patients with R/R MM

Biomarker analyses showed a significant and sustained decline in serum soluble BCMA (sBCMA) levels across the IBI3003 120 μg/kg, 360 μg/kg, and 540 μg/kg dose groups.
Deep and durable reductions in sBCMA levels were observed across all dose groups. After three treatment cycles, the median reduction from baseline in sBCMA levels was 98.38% in the IBI3003 360 μg/kg group.
IBI3003 continued to demonstrate encouraging and durable responses, together with a manageable safety profile, in heavily pretreated patients with R/R MM. These efficacy and safety data support further evaluation of the clinical value of IBI3003 in the ongoing Phase 3 clinical study.

Professor Peng Liu of Zhongshan Hospital Affiliated to Fudan University stated, "Patients with R/R MM have a poor prognosis after failing treatments including PIs, IMiDs, and anti-CD38-based therapies, with an ORR of only 29.8%, a median progression-free survival of 4.6 months, and a median overall survival of 12.4 months[1]. Although bispecific antibodies are reshaping the treatment landscape of R/R MM, trispecific antibodies targeting two antigens simultaneously may help overcome treatment resistance caused by intra-tumor and inter-tumor heterogeneity, as well as treatment-induced antigen escape. A substantial unmet clinical need remains in R/R MM, particularly among heavily pretreated and high-risk patients.

The dual-target coverage of BCMA and GPRC5D by IBI3003 addresses antigen expression heterogeneity and treatment resistance associated with single-target therapies, thereby reducing tumor escape. Its optimized CD3 affinity enables precise T-cell activation and tumor killing while improving safety. In the Phase 1 results presented at this meeting, IBI3003 demonstrated a manageable safety profile and impressive efficacy at the 360 μg/kg dose, with an ORR of 84.6%. Significant efficacy was also observed in patients with EMD or other high-risk features, fully demonstrating the potential of IBI3003 to overcome treatment resistance. The ORR reached 94.7% among patients who had received 2 to 4 prior lines of therapy. We look forward to the results of the ongoing Phase 3 clinical study of IBI3003."

About IBI3003 (Anti-GPRC5D/BCMA/CD3 Trispecific Antibody)

IBI3003 was developed using Innovent’s proprietary Sanbody platform. It is a novel trispecific antibody targeting G protein-coupled receptor C5D (GPRC5D), B-cell maturation antigen (BCMA), and CD3. The molecule was designed to overcome tumor escape caused by the loss or downregulation of a single tumor antigen. In preclinical mouse models, IBI3003 demonstrated superior in vivo anti-tumor activity compared with marketed benchmark bispecific antibodies. It also demonstrated particularly prominent tumor-killing activity in in vitro cell models with low expression of BCMA and GPRC5D.

Phase 1/2 clinical trials of IBI3003 (NCT06083207 and NCT07336472) are being conducted concurrently in China, Australia, and the United States to evaluate its safety, tolerability, and efficacy in patients with R/R MM. In China, IBI3003 has advanced into a pivotal registrational clinical stage. TriadicMM-1 (NCT07623798) is a multicenter, randomized, open-label Phase 3 study designed to compare the efficacy and safety of IBI3003 with investigator’s choice of therapy in patients with R/R MM who have received 1 to 4 prior lines of therapy. The primary endpoint is progression-free survival (PFS) assessed by an independent review committee (IRC). In addition, the Phase II study of IBI3003 combined with CD38 monoclonal antibody for frontline MM is also ongoing (NCT07764861).

(Press release, Innovent Biologics, SEP 29, 2026, View Source [SID1234671162])

On September 29, 2026 HUYABIO International, the leader in accelerating the development of innovative biopharmaceutical products discovered in China, reported that further data from its pivotal phase 3 trial of HBI-8000 in combination with nivolumab in advanced melanoma has been selected as a Late-Breaking Abstract (LBA) and will be presented in an oral Proffered Paper session at ESMO (Free ESMO Whitepaper) Congress 2026 taking place Oct. 23 – 27 in Madrid, Spain.

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HBI-8000 is a highly differentiated oral selective Class I histone deacetylase (HDAC) inhibitor designed to boost the immune response against cancer cells through epigenetic modification. By modifying the tumor microenvironment from non-responsive (‘cold’) to responsive (‘hot’), HBI-8000 has the potential to enhance the activity of anti-PD-1 checkpoint inhibitors such as nivolumab.

HUYABIO announced topline results from the global Phase 3 trial in July which showed a statistically significant improvement in median progression-free survival (mPFS) in advanced melanoma patients taking HBI-8000 and nivolumab compared with those taking nivolumab plus placebo. The randomized global Phase 3 trial enrolled 404 patients across 15 countries.
The abstract will be available on the ESMO (Free ESMO Whitepaper) website at 00:05 CET on Friday, October 23.

Details of the ESMO (Free ESMO Whitepaper) Congress 2026 presentation

Abstract Number: LBA54 – Primary Analysis of HBI-8000-303: A Randomized Phase 3 Trial of Nivolumab plus HBI-8000 or Placebo in Advanced Melanoma
Presenter: Nikhil I. Khushalani, Moffitt Cancer Center, Tampa, FL, USA
Proffered Paper Session Details: Friday, October 23 at 16:15 – 17:45 CET in Barcelona Auditorium – Hall 9

(Press release, HUYA Bioscience, SEP 29, 2026, View Source [SID1234671161])

On September 29, 2026 UTR Therapeutics Inc., a biotech company with clinical trial-ready assets defining a new category of RNA therapeutics to conquer the undruggable proteome, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to 3′UTRMYCN M1-14 (UTRxMYCN M1-14) for the treatment of soft tissue sarcoma, including rhabdomyosarcoma (RMS), and Rare Pediatric Disease Designation (RPDD) for the treatment of rhabdomyosarcoma.

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The company’s core technology of ultra-targeted 3′UTR engineering, invented by Chidiebere U. Awah, M.D., Ph.D. (CEO, UTR Therapeutics Inc.), targets the oncogenic mRNA of undruggable genes, overwrites their endogenous oncogenic mRNA messages, and triggers them for degradation by the nonsense-mediated decay pathway, thus eliminating the diseased mRNA while sparing the healthy mRNA.

MYCN (N-MYC) is a basic helix-loop-helix transcription factor belonging to the MYC superfamily. It is an intrinsically disordered transcription factor with no binding pocket, which has rendered it "undruggable" and beyond the reach of conventional methods. MYCN is a defining oncogenic driver of aggressive soft tissue sarcomas, including fusion-positive rhabdomyosarcoma, the most common soft tissue sarcoma in children. MYCN contributes to a subset of the more than 80 soft tissue sarcoma subtypes affecting children and adults. There are currently no clinically approved therapies that directly target MYCN.

In preclinical studies, UTRxMYCN M1-14, invented by Dr. Awah at UTR Therapeutics Inc., reliably targeted and degraded the oncogenic MYCN in aggressive MYCN-driven rhabdomyosarcoma, including the deadly fusion-positive rhabdomyosarcoma. The drug achieved on-target specificity and therapeutic efficacy, demonstrating dose-dependent inhibition of MYCN, tumor inhibition, and inhibition of liver and lung metastasis while remaining safe.

"The ODD and RPDD designations granted to UTRxMYCN M1-14 by the U.S. FDA validate the UTR Therapeutics platform technology and the potential of UTRxMYCN M1-14 to address several aggressive sarcomas that represent serious unmet needs in the U.S. and worldwide," said David T. Asuzu, M.D., Chief Medical Officer.

About Orphan Drug Designation and Rare Pediatric Disease Designation

The FDA grants Orphan Drug Designation to drugs and biologics intended to treat diseases affecting fewer than 200,000 people in the United States. ODD provides seven years of U.S. market exclusivity upon approval, a tax credit for qualified clinical testing costs, and an exemption from prescription drug user fees.

The FDA grants Rare Pediatric Disease Designation to drugs for serious or life-threatening diseases that primarily affect individuals under 18. RPDD is a prerequisite for a Rare Pediatric Disease Priority Review Voucher, which may be awarded upon FDA approval of the asset, subject to the FDA’s eligibility criteria at the time of approval. The Priority Review Voucher has been recorded to trade at values beyond $195M

(Press release, UTR Therapeutics, SEP 29, 2026, View Source [SID1234671160])