On December 9, 2025 Dizal (SSE:688192), a biopharmaceutical company committed to developing novel medicines for cancer and immunological diseases, reported new data from its hematology portfolio at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Highlights include golidocitinib, a Janus kinase 1 (JAK1) only inhibitor, in T-cell lymphoma, and birelentinib, a non-covalent LYN/BTK dual inhibitor, in B-cell lymphoma.

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Golidocitinib

Newly diagnosed PTCL
Two dose regimes of golidocitinib combined with CHOP have been explored for the treatment of newly diagnosed PTCL. Both demonstrated promising antitumor activities and a manageable safety profiles.

Golidocitinib 75mg daily with CHOP, followed by 150mg maintenance after CHOP, showed an ORR of 94.1% and a CR rate of 64.7%. By the data cutoff date, 85% of patients remained on treatment.
Golidocitinib 150mg daily with CHOP, followed by 150mg maintenance after CHOP, showed an ORR of 88.9% and a CR rate of 61.1%

R/R PTCL
An updated 2-year follow-up from the MD Anderson Cancer Center cohort of the multinational pivotal trial JACKPOT8 Part B showed that golidocitinib monotherapy in patients with relapsed or refractory peripheral T-cell lymphoma (r/r PTCL) achieved an objective response rate (ORR) of 53.8% and a complete response (CR) rate of 46.1%. Median progression-free survival (PFS) was 37.9 months and the 2-year PFS rate was 58.3%. The research findings validated golidocitinib’s long-lasting efficacy and tolerability in the U.S. patient population.

Rare subtypes of PTCL
Golidocitinib monotherapy demonstrated compelling clinical activity with a favorable safety profile in heavily pretreated relapsed or refractory T-cell and NK-cell large granular lymphocyte leukemia (r/r T-LGLL) patients. Results from a prospective study showed an ORR of 92.3% and a CR rate of 61.15%. Additionally, the study reported a 100% response among STAT3-wildtype patients.

A Phase II clinical study showed that golidocitinib in combination with CHOP demonstrated profound antitumor activity in treatment-naïve monomorphic epitheliotropic intestinal T-cell lymphoma (MEITL). The ORR was 85.7% and the CR rate was 71.4%, demonstrating a significant therapeutic advance over conventional chemotherapy.

PTCL-associated HLH
In r/r PTCL-associated hemophagocytic lymphohistiocytosis (HLH), golidocitinib-based regimens demonstrated dual anti-HLH and antitumor efficacy, with rapid clinical improvement and an ORR of 46.7%. Most patients achieved systemic and hematologic recovery with a manageable safety profile. These findings highlight the potential of JAK1 inhibition in this high-risk disease.

Birelentinib

Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) is a malignancy originating from mature B-cell non-Hodgkin lymphoma (B-NHL). While Bruton’s tyrosine kinase (BTK) inhibitors have transformed the treatment of B-cell lymphomas, resistance remains a significant challenge. Two primary types of resistance mutations have been identified after BTK inhibitor treatment for B-NHL: BTK-dependent and non-BTK pathway-mediated resistance. Although the BTK-dependent resistance is well-characterized, the emergence of kinase-impaired BTK mutations underscores the increasingly recognized role of non-BTK pathways.

Birelentinib is designed to block both BTK-dependent and BTK independent BCR signaling. Building upon promising data presented orally at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting and the 18th International Conference on Malignant Lymphoma (ICML), updated follow-up data of birelentinib reported at this ASH (Free ASH Whitepaper) Annual Meeting demonstrated potent anti-tumor efficacy with a manageable safety profile in heavily pre-treated CLL/SLL patients.

At 50 mg QD (RP3D), birelentinib achieved an ORR of 84.2%. Tumor responses were observed irrespective of prior BTK inhibitor, BCL-2 inhibitor or BTK degrader treatment, and in patients with kinase-proficient or kinase-impaired BTK mutation. Antitumor efficacy proved durable, with no new safety concerns identified during follow-up.

Based on the encouraging results, birelentinib has received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA). The global multicenter Phase III study in r/r CLL/SLL is currently ongoing.

About Golidocitinib (DZD4205)

Golidocitinib is currently the first and only Janus kinase 1 (JAK1) inhibitor being evaluated for the treatment of r/r PTCL. In June 2024, golidocitinib was approved by the National Medical Products Administration (NMPA) of China for the treatment of adult patients with relapsed or refractory peripheral T-cell lymphoma (r/r PTCL).

At the data cut-off date of August 31, 2023, golidocitinib has demonstrated robust and durable anti-tumor efficacy, with an ORR of 44.3%. All subtypes benefited well, and the ORR of common subtypes exceeded 40%. More than 50% of the patients with tumor remission achieved a complete response with a CRR of 23.9%. Per IRC assessment, the median duration of response (mDoR) reached 20.7 months. As of February 2024, golidocitinib showed a median overall survival (mOS) of 24.3 months.

Golidocitinib was granted Fast Track Designation by the U.S. FDA for the treatment of r/r PTCL in February 2022. In September 2023, the CDE accepted its NDA and granted Priority Review for the treatment of r/r PTCL. The Phase I clinical data of golidocitinib (JACKPOT8 PART A) were published in Annals of Oncology (Impact Factor: 51.8), and global pivotal trial data of golidocitinib for the treatment of r/r PTCL (JACKPOT PART B) were published in The Lancet Oncology (Impact Factor: 54.4).

About Birelentinib (DZD8586)

Two resistance mechanisms have been found in patients whose diseases have progressed on a BTK inhibitor treatment: the BTK C481X mutation and BTK-independent BCR signaling pathway activation. Birelentinib is a first-in-class, non-covalent, LYN/BTK dual inhibitor with full blood-brain barrier (BBB) penetration, designed to treat both BTK-dependent and BTK-independent B-cell non-Hodgkin lymphoma (B-NHL).

In August 2025, birelentinib was granted Fast Track Designation by the U.S. FDA for the treatment of adult patients with relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have received at least two prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor.

(Press release, Dizal Pharma, DEC 9, 2025, View Source [SID1234661329])

On December 9, 2025 Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us" or "IMMX"), a global leader in relapsed/refractory AL Amyloidosis, reported the closing of its previously announced underwritten registered offering of 19,117,646 shares of its common stock at a price to the public of $5.10 per share, and to certain investors in lieu of common stock, pre-funded warrants to purchase 490,196 shares of common stock at a price to the public of $5.09 per pre-funded warrant, which represents the per share public offering price for the common stock, less the $0.01 per share exercise price for each such pre-funded warrant. The net proceeds to Immix from the offering, after deducting the underwriting discounts, commissions and other offering expenses, were approximately $93.7 million.

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The financing includes leading U.S. biotechnology institutional investors and mutual funds.

Morgan Stanley acted as the sole book-running manager for the offering. Citizens Capital Markets and Mizuho acted as co-managers for the offering.

The securities in the registered offering were offered and sold pursuant to a "shelf" registration statement on Form S-3 (File No. 333-269100), including a base prospectus, filed with the U.S. Securities and Exchange Commission (the "SEC") on January 3, 2023, and declared effective on January 11, 2023. A prospectus supplement and accompanying prospectus describing the terms of the registered offering was filed with the SEC and is available on its website at www.sec.gov. Copies of the prospectus supplement and the accompanying prospectus relating to the offering may also be obtained from: Morgan Stanley & Co. LLC, attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014, by phone: 1-866-718-1649 or by email: [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, 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.

(Press release, Immix Biopharma, DEC 9, 2025, View Source [SID1234661314])

On December 9, 2025 GC Genome, a leading clinical genomics and liquid biopsy company, reported that its study analyzing cell-free DNA (cfDNA) fragmentation patterns in 1,154 healthy individuals has been published in Clinical Chemistry (Impact Factor 6.3, 2025). The findings reveal key physiological factors that can interfere with cancer-associated cfDNA signals, offering a foundation for improving the accuracy of liquid biopsy tests.

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The study, conducted in collaboration with Professor Min-Jung Kwon and her team at Kangbuk Samsung Medical Center, examined correlations between cfDNA fragmentomic profiles and 65 clinical variables, including age and liver function markers. The goal was to identify potential confounders that could influence cfDNA-based cancer detection in individuals without cancer.

Study Overview

Healthy cohort: 1,154 noncancerous individuals who underwent routine health checkups
Clinical variables included: 65 demographic, hematologic, and biochemical parameters
Three fragmentomic features were derived: cfDNA concentration, short-fragment ratio (SFR), and frequency of cancer-enriched motifs(CEMs)
Key Findings

Liver enzymes(including AST, ALP, γ-GTP) and age were identified as major factors altering cfDNA fragmentation patterns.
Elevated AST or age closely resembled cancer-like fragmentomic signatures, blurring the distinction between noncancer and cancer profiles.
AST showed high similarity to fragmentation size patterns seen in lung cancer patients (cosine similarity = 0.98).
Age showed the highest similarity to cancer-like profiles among clinical variables (cosine similarity = 0.52).
Receiver Operating Characteristic (ROC) analysis confirmed that these physiological variables can act as confounders by reducing the specificity of cfDNA-based detection, potentially leading to false-positive results.
These findings demonstrate that non-cancer physiological factors can influence cfDNA signals, underscoring the need for confounder-aware modeling approaches in liquid biopsy development.

A GC Genome spokesperson stated:

"This study is significant because it uses large-scale data from healthy individuals to identify key confounders that influence cfDNA fragmentation patterns. These insights will play an important role in refining our Multi-Cancer Early Detection (MCED) test, ai-CANCERCH, particularly in reducing false-positive rates and improving test specificity."

About ai-CANCERCH

Launched in September 2023, ai-CANCERCH is an AI-based multi-cancer early detection(MCED) test powered by Lc-WGS. Using just 10 mL of blood, the test detects signals associated with multiple cancers. A major upgrade—expanding from 6 detectable cancers to 10 cancers (colorectal, lung, esophageal, liver, ovarian, pancreatic, biliary, breast, gastric, and head-and-neck)—is planned for January 2026.

(Press release, GC Genome, DEC 9, 2025, View Source [SID1234661330])

On December 9, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported an update on enrollment from its November report with an increase to 78% of the target number of subjects for the first planned interim unblinding of data having consented to its pivotal Phase 2B/3 "MIRACLE" study of Annamycin in combination with cytarabine for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML). This is up from 60% in its report in November. The targeted number for the first unblinding of data is 45 subjects. Additional subjects beyond the 78% mark continue to be identified by site investigators. This update is as of December 3, 2025, as identification and recruitment are ongoing. The Company expects to complete treatment of the first 45 subjects in the first quarter of 2026.

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Walter Klemp, Chairman and CEO of Moleculin, commented, "We continue to see blinded clinical activity tracking within our expected range, based on historical responses of the trial arm equivalents. With this update, we are quickly approaching the 45th subject, which we expect to be in the first quarter of 2026, to be treated and support the first unblinding in Part A of the MIRACLE trial shortly thereafter. This increase in subjects consented in one month is highly encouraging and demonstrates the enthusiasm of investigators around Europe and the US. Although we saw early enrollment at some sites in Europe being impacted by bed shortages, we are now seeing this situation improve in certain EU countries. Moving toward our first unblinding milestone, we are excited about Annamycin’s potential to fill a major gap in AML treatment. We believe we’re well on our way to determining if Annamycin has the potential to offer a much-needed, safer, and more effective option for patients facing this devastating disease."

Mr. Klemp continued, "While the 45 subject data is not designed to hold statistical significance, we expect to see that at least one of the two arms of Annamycin (at two different dosages) plus cytarabine outperforms cytarabine plus placebo, the control arm. That could be a strong indicator that the MIRACLE trial is on track to support approval of Annamycin. We will continue to recruit the remaining 45 subjects for the full 90 subjects of Part A while we are unblinding the first 45 subjects. The second unblinding should provide enough data to decide on which dose of Annamycin to proceed with into Part B of the MIRACLE trial."

MIRACLE Trial Progress and Next Steps

The MIRACLE study (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a Phase 2B/3, global multi-center, randomized, double-blind, placebo-controlled, adaptive designed clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) portion for purposes of measuring its primary efficacy endpoint. The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (90 subjects). The first early unblinding will yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo. Before each unblinding, trial data will be subject to audit, database lock and review.

The currently consented subjects are from sites across seven countries, providing a diverse base of patient population. Subjects are identified, consented, screened, enrolled, treated and then evaluated in that order with subjects possibly withdrawing from the trial at any point of this process. The Company expects to reach the recruitment and treatment of the first 45 subjects in the first quarter of 2026 and to complete Part A of the MIRACLE trial with up to 90 patients within the first half of 2026.

For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

(Press release, Moleculin, DEC 9, 2025, View Source [SID1234661315])

On December 9, 2025 Akeso, Inc. (9926.HK) ("Akeso" or the "Company") reported that data from the Phase II study (COMPASSION-25) for its first-in-class PD-1/CTLA-4 bispecific antibody, cadonilimab, in combination with SOX regimen (oxaliplatin + tegafur/gimeracil/oteracil) as neoadjuvant therapy for resectable gastric or gastroesophageal junction (G/GEJ) adenocarcinoma, was presented at the 2025 ESMO (Free ESMO Whitepaper) Asia Congress.

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Currently, Akeso is running the pivotal Phase III study (AK104-310/COMPASSION-33) investigating cadonilimab combined with the SOX regimen for perioperative treatment of resectable G/GEJ adenocarcinoma. This regimen is expected to further push the efficacy boundaries beyond existing single-target immunotherapies and establish a new standard for perioperative immunotherapy in gastric cancer.

Promising Pathologic Complete Response (pCR) Rate

Among all evaluable patients, the overall pCR rate was 28.6%. Notably, in patients receiving the cadonilimab Q3W dosing regimen, the pCR rate reached 50.0%. pCR, defined as the absence of viable tumor cells in both the primary tumor site and regional lymph nodes upon surgical resection, is considered the "gold standard" surrogate endpoint for evaluating neoadjuvant treatment efficacy and predicting long-term survival benefits.

High Rate of Major Pathologic Response (MPR)

The overall MPR rate (defined as ≤10% residual viable tumor cells) across all evaluable patients was 71.4%. For the cadonilimab Q3W regimen, the MPR rate was as high as 85.7%. This suggests that the cadonilimab-based regimen induces substantial tumor regression in the majority of patients.

100% R0 Resection Rate

All patients who underwent surgery achieved an R0 resection (microscopically margin-negative resection), providing a solid foundation for curative intent and potentially reducing the risk of recurrence.

Significant Tumor Downstaging

Among all evaluable patients, 85.7% achieved downstaging of the primary tumor (ypT), and 75.0% achieved nodal downstaging (ypN). These results confirm the efficacy of the cadonilimab regimen in reducing tumor burden and lowering the pathological stage, thereby improving the conditions for successful surgical intervention.

Manageable Safety Profile with Good Tolerability

Treatment-related adverse events were consistent with the known safety profiles of the SOX regimen and immune checkpoint inhibitors. No new or unexpected safety signals were observed, indicating an overall manageable and favorable safety profile.

In perioperative treatment of resectable G/GEJ adenocarcinoma, chemotherapy remains the standard therapy for locally advanced gastric cancer. However, chemotherapy has limited efficacy. Cadonilimab, the first PD-1/CTLA-4 bispecific antibody, works by synergistically activating the immune system, achieving a dual blockade of the tumor immune suppressive microenvironment. This mechanism provides a stronger anti-tumor effect compared to PD-1/L1 monotherapies.

Currently, cadonilimab’s clinical value in gastric cancer is scientifically well-established. Beyond its ongoing phase III clinical trial in the perioperative setting, cadonilimab combined with chemotherapy as a first-line treatment for advanced gastric cancer (with survival benefits across the PD-L1 expression levels) has been approved for commercialization in China. Additionally, a pivotal phase III trial exploring cadonilimab in combination with pulocimab (VEGFR-2) for immune therapy-resistant advanced gastric cancer is currently ongoing and is expected to offer a new therapeutic option for later-line gastric cancer. Collectively, these pivotal phase III studies will expand the use of cadonilimab, paving the way for a comprehensive gastric cancer treatment options that spans from advanced, unresectable gastric cancer to early-stage, resectable disease.

(Press release, Akeso Biopharma, DEC 9, 2025, View Source [SID1234661331])