GlyTR Therapeutics announces landmark study in Cell on their ‘Velcro-like’ Pan-Cancer Immunotherapy Platform

On October 9, 2025 GlyTR Therapeutics, a biotech company pioneering development of glycan-targeting cancer immunotherapies, reported the publication of its foundational technology in the journal, Cell (Press release, GlyTR Therapeutics, OCT 9, 2025, View Source [SID1234656544]).

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"With the publication in Cell, GlyTR is entering the global spotlight," said Bob Genthert, Co-Founder and Interim CEO. "This is a critical milestone as we advance toward clinical trials and expand partnerships with investors, academic collaborators, and pharmaceutical companies."

The study details Glycan-Dependent T cell Recruiter (GlyTR1 & GlyTR2) therapeutics that have pan-cancer activity by targeting tumor-associated carbohydrate antigens (TACAs). The TACA’s targeted by GlyTR are among the most abundant and widespread cancer antigens known but are largely inert to the immune system and previously un-targetable.

Conventional antigen-targeting immunotherapies like Chimeric Antigen Receptor T cell (CAR-T cell) and bispecific antibodies require cancer-specific antigen expression to avoid "on-target, off-cancer" toxicity to normal tissue. In contrast, GlyTR’s innovative "Velcro-like" density-dependent binding enables discrimination between tumor and normal tissue based on target density, potentially offering unprecedented safety and efficacy in oncology treatment.

GlyTR1 also overcomes a second major roadblock to development of CAR T cells and bi-specific antibodies: immune-suppression driven by the tumor. By binding to an immune-suppressive TACA, GlyTR1 defeats these mechanisms to break the shield of immunosuppression.

"This publication validates more than a decade of research and represents a major step toward making multiple TACAs druggable targets for the first time," said Dr. Michael Demetriou, M.D., Ph.D., Co-Founder and Professor of Neurology and Molecular Genetics at University of California, Irvine. "We believe GlyTR technology could fundamentally reshape the landscape of immuno-oncology by providing a safe pan-cancer therapeutic option in a single drug."

"This work represents a potential paradigm shift from the current antibody-centric approach to cancer cell targeting," said Dr. Raymond Zhou, Ph.D., co-founder and president of GlyTR Therapeutics.

Highlights from the Cell publication:

Demonstrates potent, selective killing of multiple highly diverse tumors in preclinical models, including breast, ovarian, colon, pancreatic, lung, prostate and leukemia.
Shows no toxicity in mice with human-like glycan expression.
Details the unique ability to overcome immunosuppression in hostile tumor microenvironments.
GlyTR technology has been developed in collaboration with the UC Irvine from grants totaling ~$30 million. This includes a Cancer Moonshot award from the National Cancer Institute (NCI), multiple small business grants from NCI, several awards form the California Institute for Regenerative Medicine (CIRM) and a NCI Experimental Therapeutics program award for clinical-grade manufacturing of GlyTR1 for human trials.

Here is a link to the article in Cell: View Source

Adcendo ApS Announces FDA Fast Track Designation Granted to ADCE-D01 for the Treatment of Soft Tissue Sarcoma

On October 9, 2025 Adcendo ApS ("Adcendo"), a biotech company focused on the development of first and best-in-class antibody-drug conjugates (ADCs) for the treatment of cancers with high unmet medical need, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ADCE-D01 for the treatment of soft tissue sarcoma (STS) (Press release, ADCendo, OCT 9, 2025, View Source [SID1234656545]).

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ADCE-D01 is a first-in-class ADC targeting urokinase plasminogen activator receptor-associated protein (uPARAP) conjugated to the Topoisomerase I inhibitor payload P1021. uPARAP is a novel endocytic ADC target that is overexpressed in tumors of mesenchymal origin, such as sarcomas. Preclinically, ADCE-D01 shows strong anti-tumor activity in a range of mesenchymal tumor models including STS and is well tolerated in non-human primate toxicology studies with a favorable safety profile and no evidence of target-specific toxicity.

ADCE-D01 is currently being evaluated in the ADCElerate1 clinical trial, a first-in-human Phase I/II multicenter, open-label, dose escalation and expansion study evaluating ADCE-D01 as a monotherapy in patients with metastatic and/or unresectable STS. The primary objective of the study is to evaluate the safety and tolerability of ADCE-D01. The secondary objectives are to characterize the pharmacokinetics and to evaluate the preliminary efficacy of ADCE-D01. The study is recruiting in the US (NCT06797999) and in Europe. (EUCT number: 2024-516900-41-00).

Dr. Lone Ottesen, Chief Medical Officer of Adcendo, said: "This Fast Track designation is an important recognition of the potential of our uPARAP-targeting drug candidate and marks another meaningful milestone for Adcendo. We are committed to further advancing ADCE-D01 and believe that our uPARAP-targeting approach has the potential to transform the sarcoma treatment landscape and overcome the limitations experienced with existing therapies."

Dr. Victoria Marsh, Global Head of Regulatory at Adcendo, said: "With this Fast Track designation the development of ADCE-D01 will now benefit from more frequent interactions with the FDA. Increased FDA engagement will support and expedite the future regulatory review of ADCE-D01 with the aim of making ADCE-D01 available to patients sooner".

GT Biopharma Provides Enrollment Update on GTB-3650 Phase 1 Trial in Patients with Relapsed or Refractory (r/r) CD33 Expressing Hematologic Malignancies

On October 8, 2025 GT Biopharma, Inc. (the "Company") (NASDAQ: GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company’s proprietary TriKE natural killer (NK) cell engager platform, reported that enrollment in the dose escalation cohorts of the Phase 1 trial, evaluating GTB-3650 for the treatment of relapsed or refractory (r/r) CD33 expressing hematologic malignancies, is well on track (Press release, GT Biopharma, OCT 8, 2025, View Source [SID1234656509]).

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Enrollment in Cohorts 1 and 2 were successfully completed; both patients in Cohort 3 have now initiated treatment with no evidence of dose-limiting toxicities or safety concerns to date. The level of immune activation observed from multiple biomarkers in the first patient of Cohort 3 is consistent with the evidence of heightened immune activity in the first four patients from Cohorts 1 and 2. Assuming Cohort 3 is completed with no new safety findings, the trial will continue to dose-escalate into the higher ranges of GTB-3650 anticipated to be necessary to translate heightened immune activation into clinically meaningful evidence of therapeutic activity. Initiation of dosing in Cohort 4 is planned by year-end 2025 and additional data updates are anticipated in Q1 2026.

The Phase 1 protocol allows evaluation of GTB-3650 in up to approximately 14 patients (two patients in each of seven cohorts), with doses ranging from 1.25ug/kg/day in Cohort 1 to 100ug/kg/day in Cohort 7. GTB-3650 will be dosed in two-week blocks, two weeks on and two weeks off (defining a treatment cycle), for up to four months based on clinical benefit. The trial will assess safety, pharmacokinetics, pharmacodynamics, in vivo expansion of endogenous patient NK cells and clinical activity. More details can be found on clinicaltrials.gov with the identifier: NCT06594445.

Vivace Therapeutics’ VT3989 Granted Fast Track Designation by the U.S. Food and Drug Administration for the Treatment of Mesothelioma

On October 8, 2025 Vivace Therapeutics, Inc., a small molecule discovery and development company developing first-in-class cancer therapies targeting the Hippo pathway, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to VT3989, the company’s first-in-class and best-in-class transcriptional enhanced associate domain (TEAD) autopalmitoylation inhibitor (Press release, Vivace Therapeutics, OCT 8, 2025, View Source [SID1234656525]). The designation pertains to VT3989’s use as a treatment for patients with unresectable malignant nonpleural or pleural mesothelioma whose disease has progressed on prior immune checkpoint inhibitor therapy and platinum-based chemotherapy.

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FDA’s Fast Track Designation is granted to drug candidates that are being developed for the treatment of serious or life-threatening conditions and have the potential to fill unmet medical needs. The program is designed to ensure that these important new treatments can reach patients as quickly as possible. A company that receives Fast Track Designation is eligible for more frequent meetings and written interactions with the FDA to discuss the drug candidate’s clinical development plan, including possible eligibility for accelerated approval and priority review.

"We are pleased to receive Fast Track Designation from the FDA for VT3989 in this patient population, which is in desperate need of new and effective therapeutic options. This designation represents another important step in our ongoing development of VT3989 and will offer key advantages as we continue on our path toward potential commercialization of this first-in-class and best-in-class therapy," said Sofie Qiao, Ph.D., president and chief executive officer of Vivace Therapeutics.

VT3989 is a novel investigational small molecule cancer therapeutic that is designed to target the Hippo pathway by inhibiting palmitoylation of members of the TEAD protein family. The compound has been evaluated in more than 200 patients to date in an ongoing, open-label Phase 1 clinical study and, to the company’s knowledge, is the first and only member of the TEAD autopalmitoylation inhibitor class for which compelling clinical efficacy data have been publicly reported. In addition to the promising data to date, VT3989 has demonstrated a positive safety profile in the Phase 1 trial, which supports its best-in-class potential.

About Phase 1 study of VT3989
The Phase 1 study of VT3989 (View Source) is a multi-center, open label trial designed to evaluate the safety, tolerability, pharmacokinetics (PK) and biological activity of VT3989 in patients with refractory metastatic solid tumors, including refractory pleural and non-pleural malignant mesothelioma.

Karyopharm Announces Strategic Financing Transactions to Support Growth; Extends Cash Runway Into Second Quarter of 2026, Beyond Expected Top-Line Readout of Phase 3 SENTRY Trial in Myelofibrosis

On October 8, 2025 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that it has entered into comprehensive financing and capital structure transactions expected to provide the Company with $100 million of financial flexibility and additional capital, extending the Company’s cash runway into the second quarter of 2026 based on the Company’s current operating plans (Press release, Karyopharm, OCT 8, 2025, View Source,-Beyond-Expected-Top-Line-Readout-of-Phase-3-SENTRY-Trial-in-Myelofibrosis [SID1234656510]).

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"Following the recent completion of enrollment of our Phase 3 SENTRY trial in myelofibrosis, we are excited to announce this strategic financing which is expected to provide us with the resources needed to deliver top-line data from this pivotal trial," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "I would like to thank our lenders, noteholders and equity investors for their ongoing support to strengthen our balance sheet, equitize near-term debt maturities and support our potentially transformational Phase 3 myelofibrosis program. We believe that selinexor plus ruxolitinib has the potential to be the first combination therapy approved for the treatment of myelofibrosis. By combining selinexor with the current standard of care, we have the potential to redefine the way people living with myelofibrosis are treated."

Each of the financing and capital structure transactions is expected to close on or around October 10, 2025 (Closing Date), subject to the satisfaction of customary closing conditions. The Company’s existing senior lenders have agreed to multi-faceted financing transactions with the following key components:

$67.5 million in financial flexibility and new capital consisting of $27.5 million in new term loan borrowings and new convertible notes, $25 million of near-term deferrals of interest and royalty payments, and a $15 million temporary reduction in the Company’s minimum liquidity covenant. In addition, holders of the Company’s convertible notes due 2029 have agreed to exchange $15 million of their notes for newly issued shares of common stock or pre-funded warrants in lieu thereof.
Holders of approximately $24.25 million aggregate principal amount of the Company’s senior unsecured convertible notes due October 15, 2025 have agreed to exchange their notes and accrued interest due thereon at a discount to par value for newly issued shares of common stock, or pre-funded warrants in lieu thereof, plus warrants to purchase shares of common stock.
In addition, the Company has entered into a securities purchase agreement for a private placement in which the Company agreed to sell 1,487,917 shares of common stock and accompanying warrants to purchase 1,317,771 shares of common stock with an exercise price of $6.64 per share. The warrants issued in the private placement will be exercisable on or before the 30th day following the public announcement of top-line results from the Company’s XPORT-EC-042 trial in endometrial cancer, which is anticipated in mid-2026. The private placement is expected to result in gross proceeds of approximately $8.75 million before deducting any offering expenses.

The Company intends to use the proceeds of the financing transactions to pay transaction expenses and for general corporate purposes, including to support the Company’s ongoing and planned clinical trial activities.

At closing of the financing transactions, the Company will issue an aggregate of 7,223,982 newly issued shares of common stock, newly issued pre-funded warrants to purchase an aggregate of 2,913,136 shares of common stock, and newly issued warrants to purchase an aggregate of 5,918,358 shares of common stock with an exercise price of $6.64 per share, a 15% premium to the Nasdaq minimum price. In addition, the Company will reduce the exercise price of outstanding warrants to purchase 3,068,417 shares from $16.50 per share to $6.64 per share. The financing transactions are intended to comply with Nasdaq rules, including being priced at the "minimum price" (as defined in the Nasdaq rules). Following consummation of the transactions, the Company expects to have an aggregate of 15,926,939 shares of common stock outstanding (assuming no exercise of any pre-funded warrants or warrants or conversions of any outstanding convertible notes).

Additional details on the financing transactions will be available in a Form 8-K that the Company will file with the United States Securities and Exchange Commission.

Preliminary Third Quarter 2025 Financial Results

Based on preliminary financial information, the Company expects total revenue, which includes license and royalty revenue from partners, to be in the range of $42 to $44 million and U.S. XPOVIO net product revenue to be approximately $32 million for the three months ended September 30, 2025.

Prior to the receipt of approximately $36 million of gross proceeds from the financing transactions, the Company expects to report that it had cash, cash equivalents, restricted cash and investments as of September 30, 2025 of approximately $46 million.

The financial information presented in this press release reflects the Company’s estimates with respect to total revenue, U.S. XPOVIO net product revenue and its cash balance and is based on currently available information, which is preliminary. The Company’s final results may vary from these preliminary estimates as a result of the completion of customary quarterly review procedures, including those conducted by the Company’s external auditors.

Endometrial Cancer Program

Enrollment continues in the Phase 3 XPORT-EC-042 (NCT05611931) trial evaluating selinexor as a maintenance-only therapy following systemic therapy versus placebo in patients with TP53 wild-type advanced or recurrent endometrial cancer. The Company continues to expect to report top-line data from this event-driven trial in mid-2026.

Ongoing Efforts to Further Enhance Liquidity and Maximize Value

The Company expects to continue exploring potential financing and strategic alternatives to enhance liquidity and maximize value with the assistance of its advisors, including its financial advisor Centerview Partners LLC.

Centerview Partners LLC and J. Wood Capital Advisors LLC acted as financial advisors to Karyopharm and Sidley Austin LLP acted as legal counsel to Karyopharm in connection with these transactions.

The offer and sale of the shares of common stock, pre-funded warrants, warrants, the New 2028 Notes, the New 2029 Notes or any other securities (including the shares of common stock issuable upon conversion of the New 2028 Notes, the New 2029 Notes and the shares of common stock issuable upon exercise of the warrants and pre-funded warrants issued in the financing transactions) are not being registered under the Securities Act, or any state securities laws. The shares of common stock, pre-funded warrants, warrants, the New 2028 Notes, the New 2029 Notes or any other securities (including the shares of common stock issuable upon conversion of the New 2028 Notes, the New 2029 Notes and the shares of common stock issuable upon exercise of the warrants and pre-funded warrants issued in the financing transactions) may not be offered or sold in the United States except pursuant to an exemption from the registration requirements of the Securities Act and any applicable state securities laws.

This press release does not constitute an offer to sell or the solicitation of an offer to buy shares of common stock, pre-funded warrants, warrants, the New 2028 Notes, the New 2029 Notes or any other securities, nor shall there be any offer, solicitation or sale of shares of common stock, pre-funded warrants, warrants, the New 2028 Notes, the New 2029 Notes or any other securities (including the shares of common stock issuable upon conversion of the New 2028 Notes, the New 2029 Notes and the shares of common stock issuable upon exercise of the warrants and pre-funded warrants issued in the financing transactions) in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful.

About the Phase 3 SENTRY Trial

SENTRY (XPORT-MF-034; NCT04562389) is a Phase 3 clinical trial evaluating a once-weekly dose of 60 mg of selinexor in combination with ruxolitinib compared to placebo plus ruxolitinib in JAKi-naïve myelofibrosis patients with platelet counts >100 x 109/L. Patients are randomized 2-to-1 to the selinexor arm. The co-primary endpoints for this trial are spleen volume reduction ≥ 35% (SVR35) at week 24 and the average change in absolute total symptom score (Abs-TSS) over 24 weeks relative to baseline.

About Myelofibrosis

Myelofibrosis is a rare blood cancer that affects approximately 20,000 patients in the United States and 17,000 patients in the European Union1. The disease causes bone marrow fibrosis (scarring in the bone marrow), which makes it difficult for the bone marrow to make healthy blood cells, splenomegaly (enlarged spleen), progressive anemia which often leads to symptoms like fatigue and weakness, and other disease associated symptoms including abdominal discomfort, pain under the left ribs, early satiety, night sweats and bone pain. The only approved class of therapies to treat myelofibrosis are JAK inhibitors, including ruxolitinib. Patients treated with the most commonly prescribed JAK inhibitor often require blood transfusions, and more than 30% will discontinue treatment due to anemia.2 Anemia and transfusion dependence are strongly correlated with poor prognosis and shortened survival.3

1. Clarivate/DRG (2023)
2.. Palandri, F., Palumbo, G.A., Elli, E.M. et al. Ruxolitinib discontinuation syndrome: incidence, risk factors, and management in 251 patients with myelofibrosis. Blood Cancer J. 11, 4 (2021).
3. Pardanani, A., & Tefferi, A. (2011). Prognostic relevance of anemia and transfusion dependency in myelodysplastic syndromes and primary myelofibrosis. Haematologica, 96(1), 8–10.

About the Phase 3 XPORT-EC-042 Trial

EC-042 (XPORT-EC-042; NCT05611931) is a global, Phase 3, randomized, double-blind clinical trial evaluating selinexor as a maintenance therapy following systemic therapy in patients with TP53 wild-type advanced or recurrent endometrial cancer. The EC-042 trial is expected to enroll approximately 276 patients who will be randomized 1:1 to receive either a 60 mg, once-weekly, administration of oral selinexor or placebo until disease progression. The trial includes two patient populations, for which, the primary endpoint of progression free survival will be tested sequentially and the key secondary endpoint of overall survival will be evaluated: 1) a modified intent to treat population (mITT) that will include patients with either, a) TP53 wild-type tumors with proficient mismatch repair status (pMMR); or, b) TP53 wild-type tumors with deficient mismatch repair status (dMMR), who are medically ineligible to receive checkpoint inhibitors; and, 2) the trial’s original intent to treat (ITT) population, which will include all patients enrolled in the trial whose tumors are TP53 wild-type, regardless of MMR status. The mITT population is expected to include approximately 220 patients. In connection with the EC-042 trial, Karyopharm entered into a global collaboration with Foundation Medicine, Inc. to develop FoundationOneCDx, a tissue-based comprehensive genomic profiling test to identify and enroll patients whose tumors are TP53 wild-type.