On January 12, 2026 Oncolytics Biotech Inc. (Nasdaq: ONCY) ("Oncolytics" or the "Company"), a clinical-stage immunotherapy company developing pelareorep, reported updated clinical data from GOBLET Cohort 4 in patients with third-line metastatic squamous cell anal carcinoma ("SCAC"), a setting with no U.S. Food and Drug Administration ("FDA")-approved treatment options. Previous analysis from this cohort has focused on second-line or later SCAC patients.

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Updated GOBLET Cohort 4 Third-Line Anal Cancer Data

As of the current data cut, four of 14 evaluable third-line patients receiving pelareorep and atezolizumab achieved objective responses, resulting in an objective response rate ("ORR") of approximately 29%. These responses included two complete responses and two partial responses. The median duration of response ("DOR") is approximately 17 months (67 weeks), indicating both depth and durability of clinical benefit in a heavily pretreated population.

Patients enrolled in this cohort had progressed following multiple prior systemic therapies and represent a highly refractory disease population. In historical third-line SCAC studies, objective response rates are typically approximately 10% or less, with limited durability.1, 2 There are currently no FDA-approved therapies for patients with third-line anal cancer.

The observed response rate and emerging durability in GOBLET Cohort 4 compare favorably with historical outcomes and highlight the potential clinical relevance of pelareorep plus atezolizumab in this setting of significant unmet medical need.

"As we continue to analyze the Goblet data, we are finding important trends that are helping to shape our clinical development strategy," said Jared Kelly, Chief Executive Officer of Oncolytics. "When you isolate to anal cancer patients with two prior lines of treatment and see a strong signal like this, it points the arrow in a direct line to a registration study in an indication where there are no FDA-approved therapies. We already had good data here, but looking closer, it becomes clearer that we can make an immediate impact on patients’ lives who have no options."

In the second-line setting, pelareorep and atezolizumab achieved a 30% ORR, more than doubling the 13.8% ORR that was approved by the FDA for the current standard of care therapy. Additionally, the median duration of response is 15.5 months for pelareorep and atezolizumab compared to 9.5 months (link to the PR).

Planned Registration Strategy and Accelerated Approval Pathway

If the objective response rate and duration of response observed in GOBLET Cohort 4 are reproduced in the planned registration study, Oncolytics believes the resulting dataset would be sufficient to support accelerated approval in this indication, consistent with regulatory precedent in rare cancers with no available therapies. After initial encouraging feedback from KOLS and the FDA, Oncolytics is planning to have a Type C meeting with the FDA in Q1 2026 to discuss and receive guidance on this development plan.

(Press release, Oncolytics Biotech, JAN 12, 2026, https://oncolyticsbiotech.com/press_releases/oncolytics-biotech-announces-updated-clinical-data-from-goblet-cohort-4-demonstrating-activity-of-pelareorep-plus-atezolizumab-in-third-line-anal-cancer/ [SID1234662101])

On January 12, 2026 Pierre Fabre Pharmaceuticals, Inc. reported a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) stating that the Agency is unable to approve the tabelecleucel Biologics License Application (BLA) in its present form.

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We are surprised and deeply disappointed by the FDA’s decision, particularly given the urgent and life-threatening unmet medical need faced by patients with Epstein-Barr virus–positive post-transplant lymphoproliferative disease (EBV+ PTLD) after failure of standard-of-care therapy. These patients have no FDA-approved treatment options and a life expectancy often measured in weeks to months.

The BLA was resubmitted following clear alignment with the FDA on the acceptability of the resubmission criteria and fulfillment of the conditions outlined in the January 15, 2025, CRL, which identified a single GMP-related deficiency and raised no concerns regarding safety, efficacy, or trial design. Upon acceptance of the resubmission in July 2025, the FDA granted tabelecleucel accelerated approval status.

In the new CRL, despite acknowledging that the GMP issue had been resolved and raising no safety concerns, the FDA stated that it no longer considers the previously accepted single-arm ALLELE study to be adequate to support accelerated approval and requested a new study. This represents a significant and unexpected change in position, and one that is contrary to extensive dialogue with the Agency over more than five years.

We are concerned that this decision may have far-reaching consequences for the development of rare disease treatments, effectively creating barriers for generating clinical evidence within a unique patient population with ultra-rare conditions thereby significantly delaying—or preventing altogether— patient access to urgently needed therapies.

We firmly believe that tabelecleucel represents an important treatment advance for patients with EBV+ PTLD and that the totality of data supports its efficacy and safety. We intend to engage with the FDA to urgently pursue a path forward, in collaboration with Atara Biotherapeutics (Nasdaq: ATRA) and our clinical and patient partners, to enable timely accelerated approval of tabelecleucel. We continue to be committed to making tabelecleucel available to patients through our Expanded Access Program.

Approval and real-world use of tabelecleucel over several years in multiple countries outside the United States further support its clinical value. We remain fully committed to securing approval of this critical treatment option for U.S. patients and the physicians who care for them.

(Press release, Pierre Fabre, JAN 12, 2026, View Source [SID1234661984])

On January 12, 2026 OSR Holdings, Inc. (NASDAQ: OSRH) reported that its Swiss biotechnology subsidiary, Vaximm AG, has received a binding term sheet from BCM Europe AG confirming BCM Europe’s interest in entering a global exclusive licensing transaction for VXM01, Vaximm’s lead immunotherapy candidate.

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Under the terms of the binding term sheet, BCM Europe intends to establish and manage a dedicated investment vehicle, anchored by a strategic investor, tentatively named BCM Decentralized Science Investors I, LP (the "Fund"), which would act as the Licensee under the contemplated definitive license agreement. The proposed transaction structure includes:

the grant of global exclusive rights to develop and commercialize VXM01 to the Fund;
an upfront payment of $30 million, an increase from the previously discussed non-binding proposal, structured as 50% cash and 50% digital assets;
aggregate milestone payments of up to $815 million, with the milestone structure weighted primarily toward clinical and development achievements, rather than operational milestones; and
a royalty pass-through structure, pursuant to which the Fund would pass through royalty revenues received from a future sublicensee or commercial partner (the "Ultimate Licensee") to Vaximm, subject to a reconciliation mechanism reflecting the difference, if any, between milestone payments received from the Ultimate Licensee and the aggregate milestone payments paid by the Fund to Vaximm.
Vaximm’s Board of Directors will conduct a comprehensive review of the binding term sheet and has determined to engage an independent third-party valuation firm with recognized expertise in life-science and biotechnology licensing transactions to provide a fairness opinion. The fairness opinion will evaluate whether the proposed transaction is fair and reasonable to Vaximm and OSR Holdings’ shareholders from a financial point of view, based on the fair market value of VXM01.

"VXM01 has demonstrated encouraging clinical and safety data across multiple indications. The receipt of this binding term sheet reflects the growing recognition of VXM01’s potential value as a differentiated immunotherapy asset and underscores BCM Europe’s genuine intent and capability to close the deal," said Andreas Niethammer, CEO of Vaximm AG. "If completed, this proposed licensing framework would provide a strong foundation to advance VXM01 into later-stage development while preserving long-term upside for Vaximm."

The parties currently target, on a best-efforts basis, to negotiate and execute a definitive global exclusive license agreement by the end of May 2026, subject to customary conditions, including the completion of Vaximm’s board review, receipt of an independent third-party fairness opinion, final documentation, and required approvals.

"This binding term sheet represents a meaningful progression from earlier discussions and reflects third-party confidence in the value of VXM01," said Tim Smith, Head of Investor Relations at OSR Holdings. "While we remain mindful that a definitive agreement has not yet been executed, we believe the proposed structure, together with an independent valuation review, demonstrates a disciplined approach to unlocking long-term value for OSR Holdings’ shareholders."

(Press release, Vaximm, JAN 12, 2026, View Source [SID1234661994])

On January 12, 2026 Actuate Therapeutics, Inc. (NASDAQ: ACTU) ("Actuate" or the "Company"), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), reported that new additional and promising patient survival and biomarker data from treatment with elraglusib for metastatic pancreatic cancer were featured in oral and poster presentations at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI) on January 9, 2026. The presentations highlighted new, expanded data from the Company’s Phase 2 clinical program (Actuate 1801-Part 3B) evaluating elraglusib in combination with gemcitabine/nab-paclitaxel (GnP), a first-line chemotherapy regimen for metastatic pancreatic cancer, providing additional evidence that suggests that elraglusib combined with GnP is associated with improved survival when compared with GnP alone.

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"Pancreatic cancer continues to have a high unmet medical need, particularly at the metastatic stage," said Dr. Devalingam Mahalingam, MD, PhD. "The results from this Phase 2 study suggest that adding elraglusib to standard gemcitabine and nab-paclitaxel chemotherapy may improve survival outcomes while maintaining a manageable safety profile. The scope of this trial, which enrolled 286 patients across 60 sites in six countries, underscores the robustness of the dataset. We are grateful to the patients and their families whose participation made this research possible."

"Metastatic pancreatic cancer remains one of the most difficult cancers to treat, with few effective first-line options," said Dan Schmitt, Chief Executive Officer of Actuate Therapeutics. "In this randomized Phase 2 study, the addition of elraglusib to gemcitabine and nab-paclitaxel resulted in a meaningful improvement in overall survival compared with chemotherapy alone. As of November 2025, seventeen patients are still alive in the elraglusib/GnP arm, and three of them have remarkably passed the 24-month mark on first-line treatment, while no patients remain on GnP treatment alone. Current patient survival for metastatic pancreatic cancer is less than 12 months, so our findings are especially encouraging. We look forward to building from this data and continuing to advance elraglusib for patients with this devastating disease."

Key Findings:

Efficacy: The primary efficacy endpoints were met in this study:

· The median overall survival (OS) showed significant benefit in the elraglusib/GnP vs GnP alone arms (mOS 10.1 months vs. 7.2 months, p=0.02, HR=0.62)
· 12-month survival rate doubled to 44.4% (elraglusib/GnP) vs 22.3% (GnP)
· 24-month survival rate increased fivefold to 12.9% in the elraglusib/GnP arm compared to 2.6% in the GnP arm, emphasizing the potential for long-term clinical benefit.

Safety: The safety and tolerability profile of elraglusib was consistent with previously reported data at the 9.3 mg/kg dose, and no new safety signals were identified:

· Serious TEAE were similar between the elraglusib/GnP (56.1%) and the GnP alone arm (56.4%),
· TEAE resulting in death were similar between treatment arms: elraglusib/GnP (12.3%) and GnP alone (16.7%)
· Grade-3 or higher TEAE leading to stoppage of any study drug: elraglusib/GnP (16.8%) and GnP alone (21.8%)

Genomic biomarkers: Several mutations commonly associated with pancreatic cancer were identified as potential predictive biomarkers of overall survival in the elraglusib/GnP arm, but not in the GnP-alone arm. Actionable mutations like KRAS or ARID1A could suggest novel drug combinations for future clinical studies.

Immunological biomarkers: The percentage of CD8+ and Granzyme B+ cells, as well as NK cells, was increased in tumors obtained from elraglusib/GnP-treated patients but not in GnP-treated patients, suggesting that combination therapy may potentiate improved anti-tumor immune response.

Oral Presentation

Title: Results from the randomized Phase 2 study (1801 Part 3B) of elraglusib plus gemcitabine/nab-paclitaxel (GnP) versus GnP in previously untreated metastatic pancreatic ductal adenocarcinoma (mPDAC).
Abstract: 653
Session: Rapid Oral Abstract Session B: Cancers of the Pancreas, Small Bowel, and Hepatobiliary Tract
Date/Time: January 9, 2026, 4:15 PM-5:00 PM (PST)
First Author: Devalingam Mahalingam, MD, PhD, Gastrointestinal Oncologist and Professor of Medicine at Northwestern University Feinberg School of Medicine

Poster Presentation

Title: Mutational analysis and identification of potential biomarkers in patients with metastatic pancreatic cancer treated with the combination of the GSK-3 inhibitor elraglusib and gemcitabine/nab-paclitaxel in the 1801 Part 3β Phase 2 study.
Abstract: 761
Poster Bd: L5
Session: Poster Session B: Cancers of the Pancreas, Small Bowel, and Hepatobiliary Tract
Date/Time: January 9, 2026, 11:30 AM-1:00 PM; 5:00 PM-6:00 PM (PST)
First Author: Andrey Ugolkov, MD, PhD, Senior Director, Clinical Science at Actuate Therapeutics

About Actuate-1801 Part 3B Study

The Actuate-1801 Part 3B study (NCT03678883) is a randomized, controlled Phase 2 trial of elraglusib with GnP versus GnP alone in first-line mPDAC. The trial enrolled 286 mPDAC patients with no prior systemic treatment for metastatic disease, who were randomized 2:1 to the elraglusib treatment arm (elraglusib/GnP) or the control arm (GnP alone). Elraglusib is administered at a dose of 9.3 mg/kg by IV infusion on Day 1 of each week of a 28-day cycle. The primary endpoint for this study is median overall survival, with OS summarized throughout the study by estimates of 1-year survival. Secondary endpoints are DCR, ORR, PFS, and AE.

(Press release, Actuate Therapeutics, JAN 12, 2026, View Source [SID1234661934])

On January 12, 2026 Relmada Therapeutics, Inc. (Nasdaq: RLMD, "Relmada" or the "Company"), a clinical-stage biotechnology company advancing innovative therapies for oncology and central nervous system disorders, reported that it has received written feedback from the U.S. Food and Drug Administration (FDA) regarding the registrational development pathway for NDV-01 in 2nd-line refractory, high-grade BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) — one of the highest-risk and most treatment-resistant NMIBC populations — and in intermediate risk NMIBC in the adjuvant setting, where there are currently no approved therapies.

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In its written response to Relmada’s Type B pre-IND submission, the FDA indicated that a single-arm, open-label clinical trial in this high-grade, BCG-unresponsive with CIS population is an appropriate registrational approach for NDV-01. This feedback provides a clear and efficient development path toward a potential NDA submission for NDV-01 as a bladder-sparing therapeutic option in a patient population with significant unmet need.

The FDA also provided separate, supportive written feedback on the Company’s planned single registrational study in intermediate-risk NMIBC in the adjuvant setting, which is expected to follow an open-label, randomized-to-observation design.

Relmada continues to anticipate initiating both registrational trials in the first half of 2026.

"We are very pleased with the FDA’s alignment on the registrational design for NDV-01 in high-grade BCG-unresponsive NMIBC," said Raj S. Pruthi, MD, Chief Medical Officer – Oncology at Relmada Therapeutics. "A single-arm pivotal study in this setting represents a meaningful opportunity to advance an in-office, bladder-sparing therapy for patients who have few if any effective alternatives. This study represents the fastest path to approval for NDV-01."

Dr. Pruthi continued, "We are also encouraged by the FDA’s feedback on our intermediate-risk registration plans, where we believe NDV-01 could potentially provide meaningful clinical benefit to patients where no approved treatments currently exist."

About the Planned High-Grade Registrational Study

The planned pivotal Phase 3 study in 2nd-line, refractory, high-grade BCG-unresponsive NMIBC with carcinoma in situ (CIS) will be an open-label, single-arm trial evaluating:

Primary endpoint: Complete response (CR) rate at any time
Key secondary endpoint: Duration of response (DOR)
Assessments: Cystoscopy, cytology, and biopsy per protocol

The design reflects FDA’s written guidance on the study population, endpoint selection, and evaluation methodology and is consistent with prior FDA precedents for single-arm registrational trials in NMIBC.

About the Planned Intermediate-Risk Registrational Study

The planned pivotal Phase 3 study in intermediate-risk NMIBC in the adjuvant setting will be an open label randomized-to-observation study:

Primary endpoint: Disease Free Survival (DFS)
Key secondary endpoint: Duration of response (DOR)
Assessments: Cystoscopy, cytology, and biopsy per protocol

The design reflects FDA’s written guidance on the study population, endpoint selection, and evaluation methodology.

About NDV-01

NDV-01 is a sustained-release, intravesical formulation of gemcitabine and docetaxel (Gem/Doce), in development for the treatment of non-muscle invasive bladder cancer. It is designed to enable Gem/Doce bladder retention and gradual drug release over ten days. The formulation creates a soft matrix that enhances local exposure while minimizing systemic toxicity. The NDV-01 formulation is a ready to use, convenient to administer in-office in less than five minutes, and does not require anesthesia or specialized equipment. It is protected by patents through 2038.

About NMIBC

NMIBC represents 75-80% of all bladder cancer cases and is associated with high recurrence (50 – 80% over 5 years). With over 744,000 prevalent cases in the U.S. and limited treatment options, the market opportunity is significant. High-grade BCG-unresponsive disease represents one of the most difficult-to-treat NMIBC subtypes, with limited bladder-sparing options. Intermediate-risk NMIBC in the adjuvant setting has no currently approved therapies. NDV-01 has the potential to serve as a frontline or salvage therapy and could be applicable across multiple NMIBC subtypes.