On June 3, 2025 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer and obesity drugs, reported highlights from the recent clinical development and operational update conference call and webcast held May 26, 2025 (Press release, Bio-Path Holdings, JUN 3, 2025, View Source [SID1234653669]). An archived webcast of the event can be accessed here.

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"Our business model centers around generating new drug candidates from our DNAbilize platform and licensing them for final development and commercialization with partners that have expertise and scale to successfully bring them to market," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. "The clinical progress we are making across our pipeline is bringing us one step closer to our goal of delivering a better path for oncologic and cardiometabolic patients."

Clinical Program Overview

Prexigebersen Phase 2 Clinical Trial – Bio-Path’s Phase 2 clinical trial for the treatment of AML is comprised of three cohorts of patients and treatments, each separately approvable by the FDA as a new indication. The first two cohorts are treating patients with the triple combination of prexigebersen, decitabine and venetoclax. The first cohort includes untreated AML patients, and the second cohort includes relapsed/refractory AML patients. Finally, the third cohort is treating relapsed/refractory AML patients who are venetoclax-resistant or intolerant with the two-drug combination of prexigebersen and decitabine. Outcomes for these older patients who are unable to receive intensive chemotherapy due to the challenging side effect profile, remain suboptimal with a median survival of only 5 to 10 months. As previously reported, Bio-Path identified two patients who have demonstrated continued treatment durability. These patients have each received over 15 treatment cycles and remain in complete remission.

Bio-Path expects to utilize an advisory panel of AML experts to assist in the design of the final clinical development plans through potential FDA approval. Other significant milestones expected during 2025 include the completion of Cohort 2 and an interim analysis for Cohort 3.

Phase 1/1b Clinical Trial in BP1001-A in Advanced Solid Tumors – A Phase 1/1b clinical trial of BP1001-A in patients with advanced or recurrent solid tumors, including ovarian and uterine, pancreatic and breast cancer, is ongoing. BP1001-A is a modified product candidate that incorporates the same drug substance as prexigebersen but has a slightly modified formulation designed to enhance nanoparticle properties. The Phase 1 study has advanced to the second, higher dose level and the first patient in the second dose cohort continued experiencing a positive response which may signal that this analog of prexigebersen has potential as a new treatment for advanced solid tumors. The patient continues to be doing well after failing extensive chemotherapy and surgical treatment for gynecologic cancer, demonstrating a 15% reduction in her primary tumor through ten cycles of treatment. Moreover, it appears that these positive outcomes may have contributed to allowing her to continue with rigorous exercise and improved quality of life. Completion of the second and third dosing cohorts are expected later this year.

The Phase 1b portion of the study is expected to commence after successful completion of the three BP1001-A monotherapy dose level cohorts and is intended to assess the safety and efficacy of BP1001-A in combination with paclitaxel in patients with recurrent ovarian or endometrial tumors. Phase 1b studies are also expected to be initiated in combination with gemcitabine in Stage 4 pancreatic cancer and combination therapy in breast cancer.

Phase 1/1b Clinical Trial in BP1002 in Relapsed/Refractory AML – A Phase 1/1b clinical trial for BP1002 to treat relapsed/refractory AML patients, including venetoclax-resistant patients, is ongoing. BP1002 targets the protein Bcl-2, which is responsible for driving cell survival in up to 60% of all cancers. Venetoclax treats AML patients by blocking the activity of the Bcl-2 protein in AML patients. However, over time patients become resistant to venetoclax. BP1002 treats the Bcl-2 target by blocking the cell’s ability to produce Bcl-2 and could provide benefit for these venetoclax resistant patients. AML patients that fail frontline venetoclax-based therapy have very poor prognosis with median overall survival of less than three months. The first dose cohort consisted of a starting dose of 20 mg/m2, the second dose cohort of 40 mg/m2 and there were no dose limiting toxicities. The third dosing cohort of 60 mg/m2 has been completed and the fourth dosing cohort of 90 mg/m2 is open for enrollment. Enrollment in the third dosing cohort closed faster than expected which Bio-Path believes reflects the need for additional treatment options.

Prexigebersen as Potential Treatment for Obesity in Type 2 Diabetes Patients – BP1001-A downregulates Grb2 expression to increase insulin sensitivity and helps lower blood glucose level in Type 2 diabetes patients. Scientific evidence suggests that by downregulating Grb2 expression, BP1001-A could help lower blood glucose levels by affecting insulin signaling. Bio-Path conducted preclinical studies that confirmed the effectiveness of BP1001-A in affecting insulin signaling and its potential efficacy as a therapeutic treatment for obese patients who have Type 2 diabetes. In May, the Company reported the achievement of a third milestone from preclinical studies of BP1001-A that provide additional support for its potential as a treatment for obesity. These studies showed BP1001-A rescues the decrease in AKT activity in liver cells and prevents cells from becoming insulin resistant, confirming its potential as a treatment for obesity and related metabolic diseases in Type 2 diabetes patients. In 2025, Bio-Path expects to complete preclinical testing and to file an Investigational New Drug (IND) application.

Intellectual Property Protection

Bio-Path’s composition of matter patents are designed to protect encroachment from third parties on its proprietary products. These composition patents allow the Company to apply its core technology to new protein targets and receive new 20-year patents. Bio-Path’s patent portfolio is as follows:

Composition and methods of use patents issued cover DNAbilize technology, solely owned by Bio-Path.
Seven patents issued in the U.S. with one additional application allowed; 61 foreign patents issued across 26 countries; five additional foreign patent applications allowed; three applications pending in the U.S. along with more than 30 applications pending in foreign jurisdictions.

On June 3, 2025 BeyondSpring Inc. (NASDAQ: BYSI) ("BeyondSpring" or the "Company"), a clinical-stage global biopharmaceutical company focused on developing cancer therapies, reported that it presented interim phase 2 data on the 303 Study, a study in 2L/3L non-small cell lung cancer (NSCLC) after disease progression on 1L PD-1/L1 inhibitors with and without chemotherapy (NCT05599789), with financial support from Merck’s (Rahway, NJ USA) Investigator Studies Program and provision of study drug, at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, on May 31st, 2025 in Chicago, IL (Press release, BeyondSpring Pharmaceuticals, JUN 3, 2025, View Source;utm_medium=rss&utm_campaign=beyondspring-presents-efficacy-safety-data-from-a-phase-2-study-of-pembrolizumab-plus-plinabulin-docetaxel-in-metastatic-nsclc-after-progressing-on-first-line-immune-checkpoint-inhibitors-at-2025 [SID1234653667]).

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The single study finished enrollment of 47 patients with patients treated with Plinabulin, pembrolizumab and docetaxel on day 1 of each cycle until disease progression or intolerable severe adverse events. All patients progressed on PD-1/L1 inhibitors, including 78% who received pembrolizumab. Of the 47 patients, the median age was at 67 ranging from 44 to 83; 80.9% were male and 19.1% were female; 72.3% were current or former smokers. Histology included 63.8% patients with non-squamous cell carcinoma and 36.2% with squamous cell carcinoma. The median follow-up was 12.7 months. As of the data cut-off date of May 16, 2025, there are patients still responding to the therapy; therefore, the final data will be updated. The key results at the database lock are summarized below.

Median Progression-Free Survival (PFS):8 months — nearly double the 3.7 months seen with current standard of care (SOC) docetaxel1
Confirmed Objective Response Rate (ORR):2% — higher than the 12.8% seen with SOC docetaxel1
Disease Control Rate (DCR: PR + SD > 4 months):3% — indicating clinical benefit majority of patients who progressed on prior PD-1/L1 inhibitors
Median Duration of Response (DoR):2 months
6-Month PFS Rate:56%
15-Month Overall Survival (OS) Rate:78% (median OS not yet reached) – longer than median OS of 11.8 months for SOC docetaxel1
The combination was well tolerated. 1% of patients experienced grade 3 or higher treatment-related adverse effects, including GI side effects of 14.9% and transient hypertension of 14.9%. There were no treatment-related deaths.
"These promising Phase 2 results reinforce Plinabulin’s potential as a first-in-class therapy that addresses one of the most urgent challenges in oncology—acquired resistance to checkpoint inhibitors," said Dr. Lan Huang, PhD, Co-Founder, Chairman and CEO of BeyondSpring.

"By restoring immune sensitivity and improving progression-free survival, disease control rate and overall survival, Plinabulin opens a new therapeutic path for the more than 60% of patients who stop responding to PD-1/L1 therapies. We are encouraged by these data and committed to advancing Plinabulin in combination strategies to meet critical unmet needs in lung cancer and beyond."

A New Potential Pathway to Re-Sensitize Tumors to Immunotherapy
Immune checkpoint inhibitor (ICI)-based regimens have remained as the standard of care for first-line treatment of NSCLC, but over 60% patients could progress from ICI2. "Acquired resistance" to ICI could be caused by "T cell exhaustion" or "antigen presenting cell pathway mutation"2. Once progressed, it is not recommended to continue using ICI monotherapy. This underscores a substantial unmet need for more effective treatment options.

With over 700 cancer patients treated with good tolerability, Plinabulin is a first-in-class, late-stage, differentiated tubulin binder that activates GEF-H1, triggering dendritic cell (DC) maturation and T cell activation, and reduce chemotherapy induced neutropenia3,4,5. This dual innate and adaptive immune mechanism has demonstrated Plinabulin’s strong potential to reverse "acquired resistance to ICI" and to enhance the efficacy and tolerability of both PD-1/L1 inhibitors and chemotherapy-based regimens.

"This patient population—those who relapse after checkpoint inhibitors—faces a grim outlook with limited options," said Dr. Mengzhao Wang, Principal Investigator and Chief of Respiratory and Critical Care Medicine at Peking Union Medical College Hospital. "Second- and third-line NSCLC with no actionable driver mutation after progression of ICIs is a critical unmet medical need, with no new agent approval in the last decade. The current SOC docetaxel, approved 25 years ago, has limited efficacy and over 40% severe neutropenia. With Plinabulin’s benefit in significantly reducing severe neutropenia of docetaxel in a number of studies, this promising Phase 2 efficacy data suggests the triple combination may provide both efficacy and safety benefit in a meaningful way. We are encouraged and look forward to further study."

2025 ASCO (Free ASCO Whitepaper) Poster Presentation: Phase 2 Study of Pembrolizumab (Pembro) plus Plinabulin (Plin) and Docetaxel (Doc) for Patients (Pts) with Metastatic NSCLC after Progression on First-line Immune Checkpoint Inhibitor Alone or Combination Therapy: Initial Efficacy and Safety Results on Immune Re-sensitization

Presenter / Author: Yan Xu, Minjiang Chen, Xiaoxing Gao, Xiaoyan Liu, Jing Zhao, Wei Zhong, Ruili Pan, Mengzhao Wang
Poster Session: Lung Cancer – Non-Small Cell Metastatic
Abstract Number: 8560

On June 3, 2025 Avenzo Therapeutics, Inc. ("Avenzo"), a clinical-stage biotechnology company developing next-generation oncology therapies, reported initiation of a Phase 1/2 clinical study of its potential best-in-class EGFR/HER3 bispecific antibody-drug conjugate, AVZO-1418, in patients with advanced solid tumors (Press release, Avenzo Therapeutics, JUN 3, 2025, View Source [SID1234653666]).

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"HER3 is a key dimerization partner of EGFR, and their interaction is known to drive oncogenic signaling across a range of solid tumors, including non-small cell lung cancer, breast cancer, and head and neck cancer," said Anthony Tolcher, M.D., FRCPC, CEO and Founder, NEXT Oncology. "Based on its preclinical data and differentiated design targeting this pathway, AVZO-1418 offers a promising approach for patients with advanced solid tumors."

The Phase 1/2 first-in-human, open-label clinical study is designed to assess the safety, tolerability, and preliminary clinical activity of AVZO-1418 as a single agent and in combination therapy in patients with advanced solid tumors.

"We are excited to have initiated our company’s second clinical trial within 2 weeks of receiving IND clearance," said Mohammad Hirmand, M.D., Co-founder and Chief Medical Officer of Avenzo Therapeutics. "This is another important milestone in our journey to advance potentially differentiated drug candidates that help fight cancers with high unmet medical needs."

On June 3, 2025 Alector, Inc. (Nasdaq: ALEC), a late-stage clinical biotechnology company focused on developing therapies to counteract the devastating progression of neurodegeneration, reported that management will participate in a fireside chat at the Goldman Sachs 46th Annual Global Healthcare Conference on Tuesday, June 10, 2024, at 8:40 am ET (Press release, Alector, JUN 3, 2025, View Source [SID1234653665]).

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A live webcast of the fireside chat will be available on the "Events & Presentations" page within the Investors section of the Alector website at View Source A replay will be available on the Alector website for 90 days following the event.

On June 3, 2025 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that biomarker data from its OPTIMIZE-1 clinical trial were presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Alligator Bioscience, JUN 3, 2025, View Source [SID1234653629]).

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OPTIMIZE-1 is a Phase 1b/2 trial evaluating mitazalimab in combination with mFOLFIRINOX in first-line metastatic pancreatic cancer (mPDAC). The study has demonstrated strong clinical outcomes to date, including a ~30% survival rate at 24 months and a median overall survival of 14.9 months.

The biomarker analyses highlighted mitazalimab’s immunological mechanism of action, with immune activation in responding patients and gene signatures associated with clinical benefit and improved survival. These results support continued development of mitazalimab, including a planned confirmatory trial in mPDAC.

"ASCO continues to be a cornerstone event for clinical oncology, and it was a privilege to share our OPTIMIZE-1 data with the international community," said Søren Bregenholt, CEO of Alligator Bioscience. "The high level of engagement and many productive meetings we’ve had during the conference further validate the growing interest in mitazalimab and its potential in pancreatic cancer."
The presentation poster is available on Alligator’s website. Alligator’s participation at ASCO (Free ASCO Whitepaper) is part of its broader commitment to scientific collaboration and strategic business development.