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])

On December 8, 2025 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported the presentation of encouraging updated data of patients treated in the Phase 1 NATHALI-01 clinical trial with eti-cel, at the 67th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Orlando, FL.

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Eti-cel product candidate is the first allogeneic dual CAR-T targeting CD20 and CD22 simultaneously, being developed in Phase 1 of the NATHALI-01 clinical trial, for patients with relapsed/refractory non-Hodgkin lymphoma (r/r NHL), following at least two lines of therapy.

Cellectis presented preliminary results on eti-cel, which demonstrated an encouraging overall response rate (ORR) of 88% and a complete response (CR) rate of 63% (n=8) at the current dose level.

Additional in vivo data presented suggest that exogenous low dose Interleukin-2 (IL-2) support can significantly enhance the expansion and persistence of CAR-T cells to boost CAR-T efficacy without exacerbating toxicity.

"Cellectis believes that, with the addition of low dose IL-2 support, it is possible to further deepen the already high response rates seen with eti-cel in these patients who have relapsed following multiple prior lines of therapy including, in most cases, a CD19 CAR-T" said Adrian Kilcoyne, MD, MPH, MBA, Chief Medical Officer at Cellectis. "The trial will now investigate any potential impact of low dose IL-2 support in these difficult to treat patients. We look forward to sharing the full Phase 1 dataset expected in 2026."

Next Steps

Overall, these preliminary data underscore the potential of this innovative approach to transform outcomes for r/r NHL patients. The Company will now investigate the potential impact of low dose IL-2 support and will start recruitment of patients in the IL-2 support cohort in Q1 2026. Cellectis expects to present the full Phase 1 dataset in 2026.

(Press release, Cellectis, DEC 8, 2025, View Source [SID1234661251])

On December 8, 2025 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines in oncology and autoimmune disease, reported new clinical data from patients with relapsed or refractory Waldenström macroglobulinemia (WM) treated in the Phase 1 clinical trial of its Bruton’s tyrosine kinase (BTK) degrader bexobrutideg (NX-5948). These data will be presented by Scott Huntington M.D., MPH, Associate Professor of Internal Medicine (Hematology), Yale School of Medicine, and a clinical investigator on the trial, on December 8, 2025, at 6 p.m. ET at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held in Orlando, FL.

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"The data presented at ASH (Free ASH Whitepaper) in this older and heavily pre-treated WM population that includes patients with MYD88 and CXCR4 mutations continue to demonstrate encouraging activity of bexobrutideg with durable and deepening responses with longer time on treatment," said Paula G. O’Connor, M.D., chief medical officer of Nurix. "Bexobrutideg was well tolerated, consistent with the overall study population and previous disclosures."

"Collectively, these clinical data and recent data highlighting the unique properties of our potent and highly selective BTK degrader contribute to a growing body of evidence that support bexobrutideg’s potential to be the best-in-class and an important new therapeutic option for patients," said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. "We believe bexobrutideg is an innovative therapy with the potential to transform care in CLL, WM, and additional NHL indications, while supporting long-term value creation as its development expands into inflammatory and autoimmune settings."

The data presented at the 2025 ASH (Free ASH Whitepaper) Annual Meeting include patients with WM (n=31) treated with bexobrutideg at doses ranging from 200 mg to 600 mg once daily by oral administration from both the Phase 1a dose escalation and Phase 1b cohort expansions. Among the 31 WM patients, the median age was 71.0 years (range 49–88 years), and the median number of prior lines of therapy was 3 (range 1-7). All 31 patients previously had been treated with a BTK inhibitor (100%), 28 had received prior chemotherapy/chemo-immunotherapy (90.3%), four had received prior non-covalent BTK inhibitor (12.9%), and four patients had received prior treatment with a BCL2 inhibitor (12.9%). Twenty-four patients (77.4%) had mutations in MYD88, and six patients (19.4%) had mutations in CXCR4. Three patients (9.7%) had central nervous system (CNS) involvement at baseline.

Bexobrutideg was well tolerated in patients with WM, consistent with the overall study population and previous disclosures. Adverse events (AEs) were predominantly low grade with the most common being neutropenia (29.0%), petechiae (29.0%), diarrhea (25.8%), anemia (22.6%), purpura/contusion (22.6%), and thrombocytopenia (19.4%). There were no dose limiting toxicities observed and no grade 5 AEs. Two treatment emergent AEs led to drug discontinuation. No new onset atrial fibrillation was observed.​

As of the September 19, 2025 data cut, 28 patients were evaluable for response. Bexobrutideg demonstrated an objective response rate (ORR) of 75.0%, including very good partial responses (VGPR) in three patients (10.7%), partial responses (PR) in 14 patients (50.0%), and minor responses (MR) in four patients (14.3%). Six patients (21.4%) had a best response of stable disease (SD). In a subgroup analysis of patients with 2 or more disease assessments (n=23), ORR was 82.6% and disease control rate (DCR) was 100.0%.

Responses were observed in patients regardless of their baseline mutations in MYD88 and CXCR4. Out of three patients with CNS involvement (2 with systemic disease), two have responded and none progressed. Overall, responses were durable. With a median follow up of 8.1 months, median duration of response and median progression-free survival were not reached. As of the September 19, 2025 data cut, fourteen patients had continued on treatment for more than six months, and six patients had remained on treatment for more than one year.

Nurix Webcast Details
Date and time: Monday, December 8, 2025, 8:15 p.m. ET
Access Details: The live webcast and subsequent archived replay will be available in the Events section of the Investor page of the Nurix website at ir.nurixtx.com.

About Bexobrutideg (NX-5948)
Bexobrutideg is an investigational, orally bioavailable, brain penetrant, highly selective small molecule degrader of BTK currently being evaluated in the DAYBreak CLL-201 clinical trial (NCT07221500), a pivotal single-arm Phase 2 study of bexobrutideg in patients with relapsed or refractory chronic lymphocytic leukemia. Nurix also continues enrollment in the NX-5948-301 Phase 1a/1b clinical trial (NCT05131022) of bexobrutideg in patients with relapsed or refractory B cell malignancies.

(Press release, Nurix Therapeutics, DEC 8, 2025, View Source [SID1234661267])

On December 8, 2025 Pillar Biosciences and AstraZeneca reported an expansion of their existing laboratory access program for NGS-based kitted liquid biopsy tumor profiling to include China.

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This expanded collaboration aims to deliver rapid, cost-effective, and clinically actionable genomic insights through liquid biopsy testing. By increasing the local availability of plasma-based tumor profiling at leading clinical laboratories in China, the partnership seeks to accelerate diagnostic turnaround times and improve access to precision oncology solutions.

China faces one of the world’s highest cancer burdens, accounting for approximately 24% of new global cancer cases and 30% of cancer-related deaths, according to the World Health Organization’s GLOBOCAN 2022 report. Lung, colorectal, and liver cancers remain among the most prevalent, and access to early detection and molecular diagnostics continues to be a significant healthcare challenge. By expanding localized liquid biopsy testing, this collaboration supports China’s ongoing initiatives to enhance early cancer detection, precision diagnostics, and equitable access to targeted therapies.

As part of the initiative, AstraZeneca, Pillar Biosciences, and Shanghai Zhengu Biological Technology Co., Ltd. (Zhengu) will collaborate to support assay validation in local hospital laboratories and facilitate the implementation of Pillar’s liquid biopsy panels to enable localized tumor profiling.

"Expanding access to decentralized, high-quality molecular testing is critical to improving outcomes for cancer patients," said Dan Harma, Chief Commercial Officer, Pillar Biosciences. "By enabling local laboratories to perform in-house next-generation sequencing, we can reduce turnaround times, lower costs, and ensure that oncologists have faster access to actionable insights that guide personalized treatment decisions."

(Press release, Pillar Biosciences, DEC 8, 2025, View Source [SID1234661284])