On June 3, 2025 Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us" or "IMMX"), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis and other serious diseases, reported meeting its primary endpoint of complete response (CR) rate for cell therapy NXC-201 at an oral presentation of interim results at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO 2025) in Chicago, Illinois (Press release, Immix Biopharma, JUN 3, 2025, View Source [SID1234653672]). The oral presentation summarized meeting its interim results endpoint in from the U.S. multi-site NEXICART-2 clinical trial evaluating NXC-201 in relapsed/refractory AL Amyloidosis with a data cutoff of April 11, 2025.

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A Key Opinion Leader (KOL) event to discuss the significance will be held Tuesday, June 3, 2025 3:00pm ET with live Q&A (register here to participate).

"Meeting primary endpoint at this interim data readout is a testament to the groundbreaking efficacy of NXC-201, as we work toward the first approval in the challenging indication of relapsed/refractory AL Amyloidosis," said Ilya Rachman, MD, PhD, Chief Executive Officer of Immix Biopharma. Gabriel Morris, Chief Financial Officer of Immix Biopharma, added, "This inflection point further accelerates our progress toward BLA submission for FDA approval."

Immix’s Phase 1/2 NEXICART-2 clinical trial is a U.S. multi-site, single-arm study to evaluate the efficacy and safety of NXC-201 in patients who are refractory to, or relapse on treatment for relapsed/refractory AL Amyloidosis.

ASCO Presentation Results
Prior to NXC-201 treatment, the median lines of therapy was 4 (range: 2-12). All patients had baseline relapsed/refractory AL Amyloidosis organ involvement. After NXC-201 treatment, all patients normalized pathological disease markers. Complete responses (CRs) were observed in 70% (7 out of 10) of patients treated with NXC-201. The remaining 3 patients are bone marrow minimum residual disease (MRD) negative (10-6), predicting future CR (Immix believes remaining three MRD negative (10-6) patients could be confirmed as CRs in the coming weeks and months). Downstream clinical improvement, including cardiac and renal organ responses, were recorded after CRs. There have been no relapses recorded to-date and no safety signals identified. No neurotoxicity has been observed. Only low-grade cytokine release syndrome has been observed. The ASCO (Free ASCO Whitepaper) presentation contains clinical data as of April 11, 2025.

Current treatments typically result in a lower than 10% complete response (CR) rate in relapsed/refractory AL Amyloidosis according to Zanwar, et al 2024, indicating a high unmet medical need.

KOL Event to Discuss NXC-201 ASCO (Free ASCO Whitepaper) Clinical Data Presentation
A Key Opinion Leader (KOL) event with lead investigator Heather Landau, MD, of Memorial Sloan Kettering Cancer Center, Shahzad Raza, MD of Cleveland Clinic and Jeffrey Zonder, MD of Karmanos Cancer Center will be held Tuesday, June 3, 2025 at 3:00 pm ET to discuss these results. Register here: View Source

About NEXICART-2
NEXICART-2 (NCT06097832) is an ongoing single-arm multi-site U.S. Phase 1/2 clinical trial of sterically-optimized CAR-T NXC-201 in relapsed/refractory AL Amyloidosis. NEXICART-2 is expected to enroll 40 patients with preserved heart function (excluding patients with pre-existing heart failure) who have not been exposed to prior BCMA-targeted therapy. The primary endpoint of the Phase 1 portion is safety. The primary endpoint of the Phase 2 portion is efficacy.

About NXC-201
NXC-201 is a sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy with a "digital filter" that filters out non-specific activation. Initial data from ex-U.S. study NEXICART-1 has demonstrated high complete response rates in relapsed/refractory AL Amyloidosis. U.S. Phase 1/2 study NEXICART-2 is ongoing. NXC-201 has been awarded Regenerative Medicine Advanced Therapy (RMAT) by the FDA, and Orphan Drug Designation (ODD) by the US FDA and in the EU by the EMA.

About AL Amyloidosis
AL amyloidosis is caused by abnormal plasma cells in the bone marrow, which produce misfolded amyloid proteins that circulate in the blood, then build-up in the heart, kidney, liver, and other organs. This build-up causes progressive and widespread organ damage, including heart and renal failure, leading to high mortality rates.

The U.S. observed prevalence of relapsed/refractory AL Amyloidosis is estimated to be growing at 12% per year according to Staron, et al Blood Cancer Journal, to approximately 33,277 patients in 2024.

The Amyloidosis market was $3.6 billion in 2017, and is expected to reach $6 billion in 2025, according to Grand View Research.

On June 3, 2025 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that its team will be attending and participating at the BIO 2025 International Convention taking place June 16-19, 2025 in Boston, Mass (Press release, Genprex, JUN 3, 2025, View Source [SID1234653671]).

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In attendance from Genprex’s executive leadership team will be Thomas Gallagher, Senior Vice President of Intellectual Property and Licensing. Throughout the duration of the conference, Mr. Gallagher will be available in-person to conduct one-on-one meetings with industry groups to provide an overview of the Company’s gene therapies for cancer and diabetes.

For those interested in meeting with Mr. Gallagher or other members of Genprex’s management team, please request a meeting through the conference meeting portal or by contacting Investor Relations at [email protected].

The BIO International Convention is the largest and most comprehensive event for biotechnology, representing the full ecosystem of biotech with 20,000 industry leaders from across the globe.

On June 3, 2025 Cerus Corporation (Nasdaq: CERS) reported that members of the management team are scheduled to present at the Goldman Sachs 46th Annual Global Healthcare Conference 2025 in Miami, Florida, on Wednesday, June 11, 2025, at 8:40 a.m. Eastern Time (Press release, Cerus, JUN 3, 2025, View Source [SID1234653670]).

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A live webcast of the presentation can be accessed via the Events & Presentation section of Cerus’ Investor Relations website at ir.cerus.com. A replay of the webcast will be available on Cerus Investor Relations website shortly after the event for 30 days.

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