On January 10, 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 a clinical development and operational update for 2025 (Press release, Bio-Path Holdings, JAN 10, 2025, View Source [SID1234649588]).

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"The important work we conducted throughout 2024 has led us into what we believe is an exciting 2025 as we build off positive data generated in oncology and the addition of a new application for BP1001-A in the treatment of obesity for Type 2 Diabetes. This new application compels us to advance these studies as quickly as possible and to file for regulatory designations that could accelerate our path to approval," said Peter H. Nielsen, President and Chief Executive Officer of Bio-Path. "There is no greater challenge than the battle against cancer, and developing effective new medicines for patients suffering with few treatment options is what drives us every day. The addition of development of a potential treatment for obesity in Type 2 Diabetes patients follows this pathway as these patients need treatment beyond current weight loss drugs to support needed therapy for reducing glucose levels, which has positive impact across a number of different health-related conditions. The substantial progress we have made gives us further confidence that our DNAbilize platform is ushering in a new path of DNA-powered medicines that can make a difference in the lives of these patients."

Clinical Program Overview

Bio-Path’s clinical development program consists of one Phase 2 clinical trial, two Phase 1 or 1/1b clinical trials, and two preclinical programs. Bio-Path has developed a molecular biomarker package for its Phase 2 clinical trial in acute myeloid leukemia (AML) that was developed to identify patients that potentially have a higher propensity to respond to prexigebersen treatment. Bio-Path expects to utilize the biomarker package to accompany prexigebersen treatment in 2025 and expects to evaluate prexigebersen for the treatment of obesity. In addition, BP1001-A is in preclinical development for treatment of obesity in Type 2 Diabetes patients, which may be submitted to the U.S. Food and Drug Administration (FDA) later in the year in an Investigational New Drug (IND) application.

Development of Molecular Biomarkers – Bio-Path has developed a molecular biomarker package to accompany prexigebersen treatment, the goal of which is to identify patients with a genetic profile more likely to respond to treatment thereby improving probability of success for this program. The emerging role of biomarkers has been enhancing cancer development over the past decade and has become a more common companion to many cancer development programs. Bio-Path expects to utilize the prexigebersen biomarkers in 2025 in the Phase 2 AML clinical trial and to develop additional molecular biomarker packages to accompany its new programs.

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. Bio-Path also 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 ten months on study after failing extensive chemotherapy and surgical treatment for gynecologic cancer, demonstrating a 15% reduction in her primary tumor through nine 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 in 2025.

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 is open and enrollment 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 2025, Bio-Path expects to complete preclinical testing and to file an IND.

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 24 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 January 10, 2025 Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, reported that the U.S. Food and Drug Administration (FDA) cleared its Investigational New Drug (IND) application for TYRA-300 allowing the company to proceed with a Phase 2 clinical trial of TYRA-300 in low-grade, intermediate risk non-muscle invasive bladder cancer (IR NMIBC) (Press release, Tyra Biosciences, JAN 10, 2025, View Source [SID1234649605]). This program will be led by newly appointed Dr. Erik Goluboff, SVP, Clinical Development of TYRA, who brings more than thirty years of experience as an academic urologic oncologist, principal investigator, practicing urologist and most recently Principal Medical Lead for GU/GI cancers at Genentech/Roche.

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TYRA-300 is a potential first-in-class, investigational, oral, FGFR3-selective inhibitor designed to avoid the toxicities associated with inhibition of FGFR1, FGFR2 and FGFR4, while being agnostic for the FGFR3 gatekeeper mutations. FGFR3 is the most frequently altered gene in NMIBC, with 60-80% of IR NMIBC showing alterations. TYRA-300 is expected to be evaluated in three Phase 2 studies: SURF302 for IR NMIBC, BEACH301 for pediatric achondroplasia (ACH) and SURF301 for metastatic urothelial carcinoma (mUC).

SURF302 will be an open-label Phase 2 clinical study evaluating the efficacy and safety of TYRA-300 in participants with FGFR3-altered low-grade, IR NMIBC. The study will enroll up to 90 participants at multiple sites primarily in the United States. Participants will be randomized initially to treatment with TYRA-300 at 50 mg once-daily (QD) (Cohort 1) or treatment with TYRA-300 at 60 mg QD (Cohort 2). Following a review of efficacy and safety, an additional dosing cohort may be evaluated. The primary endpoint is complete response (CR) rate at three months. Secondary endpoints include time to recurrence, the median duration of response, recurrence free survival (RFS), progression free survival (PFS), safety and tolerability.

"Receiving FDA IND clearance is an important milestone in the advancement of TYRA-300 and for patients with NMIBC who urgently need better tolerated therapeutic options," commented Doug Warner, Chief Medical Officer of TYRA. "We look forward to leveraging Erik’s impressive background to guide our development plans in NMIBC. We expect to initiate patient dosing in the second quarter of this year, with initial three-month CR data to follow."

Dr. Goluboff joins TYRA from Genentech/Roche, where he was Principal Medical Lead for GU/GI cancers and was responsible for driving business and pipeline opportunities in those indications. Prior to Genentech/Roche, Dr. Goluboff held positions of increasing responsibility at AstraZeneca, including most recently as Global Clinical Head for IMFINZI (durvalumab) and tremelimumab for GU, GYN and tumor agnostic. Before joining industry, he held urology professorships at Columbia and Mount Sinai and managed thousands of patients from diagnosis to late line disease, medically and surgically, with bladder, prostate, and kidney cancers. He was a principal investigator for multiple clinical trials and has published over 100 peer-reviewed papers. He received his B.A. from Columbia University, an M.D. from Johns Hopkins, and an M.B.A. from NYU’s Stern School of Business. Dr. Goluboff completed his surgical internship at Johns Hopkins Hospital and his urology residency and urologic oncology fellowship at Columbia-Presbyterian Medical Center.

"For the last thirty years, I have dedicated my career to helping patients with bladder cancer as a urologic oncologist, a principal investigator running clinical trials, and as a drug developer seeking new and more effective therapies for patients with urologic cancers," added Dr. Goluboff. "I believe that TYRA-300 is the most compelling agent in development for the treatment of IR NMIBC, with a proven mechanism of action and more attractive tolerability profile than pan-FGFR inhibitors, which made joining TYRA a very exciting opportunity. I look forward to advancing TYRA-300 through the Phase 2 SURF302 study and delivering benefit to patients in need."

About Non-Muscle Invasive Bladder Cancer

In the United States, it is estimated that there are more than 730,000 people living with bladder cancer. Many of these patients have intermediate risk non-muscle invasive bladder cancer (IR NMIBC) and experience recurrence episodes throughout the course of their disease. Treatment for IR NMIBC and disease recurrence is a surgical procedure called transurethral resection of bladder tumor (TURBT) combined with intravesical-administered chemotherapy. Repeat TURBT procedures and intravesical-administered chemotherapy can impact patients’ quality of life and overall health, leading to a significant unmet medical need for better tolerated therapeutic options. TYRA-300 is the only orally administered investigational agent in clinical development for IR NMIBC.

About TYRA-300

TYRA-300 is the Company’s lead precision medicine program stemming from its in-house SNÅP platform. TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasia, including achondroplasia and hypochondroplasia. In oncology, TYRA-300 is being evaluated in metastatic urothelial cancer (mUC) and intermediate risk non-muscle invasive bladder cancer (IR NMIBC). In mUC, TYRA-300 is being evaluated in a multi-center, open label Phase 1/2 clinical study, SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors) (NCT05544552). The study is designed to determine the optimal and the recommended Phase 2 dose (RP2D) of TYRA-300, as well as to evaluate the preliminary antitumor activity of TYRA-300. In October 2024, TYRA reported interim clinical proof-of-concept data in mUC from SURF301. TYRA has received IND clearance from the U.S. FDA to proceed with its SURF302 clinical trial in patients with IR NMIBC. In skeletal dysplasia, TYRA-300 has demonstrated positive preclinical results in achondroplasia and hypochondroplasia, and TYRA has received IND clearance from the U.S FDA to proceed with its BEACH301 clinical trial in children with achondroplasia.

On January 10, 2025 Bio-Techne (NASDAQ: TECH), a global leader in life science tools and reagents, reported the launch of new designer proteins engineered using advanced Artificial Intelligence (AI) based design platforms and protein evolutionary workflows (Press release, Bio-Techne, JAN 10, 2025, View Source [SID1234649589]). This expanded portfolio from Bio-Techne’s R&D Systems brand includes IL-2 Heat Stable Agonist, Activin A Hyperactive, FGF basic Heat Stable (available both for research use only (RUO) and made under good manufacturing processes (GMP) for therapeutic development), as well as Wnt/RSPO1 Agonist, Wnt/RSPO2 Agonist, and Wnt/RSPO3 Agonist proteins.

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These innovative recombinant proteins are designed and engineered to address critical needs in cellular therapy workflows and research applications, including improved cell culture performance and optimized cell expansion. Leveraging generative AI trained on data developed over almost five decades of proteomic leadership, protein library evolution, and rational design, Bio-Techne’s expanded portfolio delivers next-generation cytokines and growth factors with tailored functionalities such as hyperactivity, enhanced receptor affinities, and improved heat stability.

"Our growing portfolio of designer proteins combine cutting-edge AI technology and innovative protein engineering," said Will Geist, President, Protein Sciences Segment. "This expanded portfolio empowers our customers with versatile, high-performance solutions to boost the production of immune cells and enhance regenerative medicine cell therapies. Bio-Techne remains committed to developing the tools and workflow solutions our customers need to advance innovative cell therapies in both clinical and research settings."

The new products include:

IL-2 Heat Stable Agonist: Engineered with enhanced thermal stability and altered receptor affinity for improved biological activity, ideal for cellular therapy applications such as CAR T-cell or Tumor-infiltrating lymphocytes (TIL) expansion.

Activin A Hyperactive: Optimized for superior performance in regenerative medicine cell differentiation applications.

FGF basic Heat Stable: A robust and versatile growth factor designed for improved protein stability and performance for stem cell culture applications, available in both RUO and GMP formats to support cell therapy manufacturing and advanced research.

Wnt/RSPO1, Wnt/RSPO2, and Wnt/RSPO3 Agonists: Key reagents for advancing stem cell research, organoid development, and regenerative medicine.

On January 10, 2025 Harbour BioMed (HKEX: 02142, the "Company"), a global biopharmaceutical company committed to the discovery, development and commercialization of novel antibody therapeutics focusing on immune-oncology and immunology, reported that the Company and Sichuan Kelun Biotech BioPharmaceutical (HKEX: 06990, "Kelun-Biotech") have entered into a license agreement with Windward Bio AG ("Windward Bio") for HBM9378/SKB378, an anti-thymic stromal lymphopoietin (TSLP) fully human monoclonal antibody co-developed by Harbour BioMed and Kelun-Biotech (Press release, Harbour BioMed, JAN 10, 2025, View Source [SID1234649606]).

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Under the terms of the agreement, Windward Bio is granted an exclusive license to research, develop, manufacture, and commercialize HBM9378/SKB378 globally, excluding Greater China and several Southeast and West Asian countries. In return, Harbour BioMed and Kelun-Biotech are eligible to receive a total of up to $970 million upfront and milestone payments, as well as tiered royalties ranging from single to double digits on net sales. The $45 million upfront and near-term milestone payments include both cash payment and an equity interest in Windward Bio’s parent company. Furthermore, Harbour BioMed and Kelun-Biotech are eligible to receive additional payments if Windward Bio undergoes a near-term change of control or enters into a sublicense agreement with a third party. All payments under the license agreement shall be paid in equal amounts to Harbour BioMed and Kelun-Biotech.

In connection with the license agreement, Windward Bio announced a $200 million Series A financing round led by OrbiMed, Novo Holdings, and Blue Owl Healthcare Opportunities, along with co-investors SR One, Omega Funds, RTW Investments, Qiming Venture Partners, Quan Capital, and Pivotal bioVenture Partners.

"We are delighted to partner with Windward Bio to advance the development of HBM9378/SKB378, a promising TSLP-targeted fully human antibody with significant potential to address immunological diseases," said Dr. Jingsong Wang, Founder, Chairman & CEO of Harbour BioMed. "This collaboration demonstrates the value of our Harbour Mice technology platform and reflects our commitment to bring transformative treatments to patients worldwide."

About HBM9378/SKB378

HBM9378/SKB378 (now also known as WIN378) is a co-development project jointly conducted by Harbour BioMed and Kelun-Biotech, with both parties equally sharing the global rights. It is a fully human monoclonal antibody targeting TSLP, generated from the two heavy chains and two light chains (H2L2) Harbour Mice platform. It inhibits the TSLP-mediated signaling pathway by blocking the interaction between TSLP and its receptor, which is a well-validated cytokine plays a key role in the development and progression of various immunological conditions, including asthma and chronic obstructive pulmonary disease (COPD). Inhibition of this pathway has shown benefits across multiple inflammatory phenotypes. The antibody’s long half-life optimization and outstanding biophysical properties provide a favorable dosing advantage.

Prior to the execution of the license agreement, Harbour BioMed submitted an Investigational New Drug (IND) application to the Centre for Drug Evaluation of the National Medical Products Administration (NMPA) in China for HBM9378/SKB378 for the treatment of COPD in November 2024. The company has also completed a phase I clinical trial in China under an IND for the treatment of moderate-to-severe asthma.

On January 10, 2025 TJ Biopharma ("TJ Bio" or "Company"), a fully integrated biotech company in China that develops, manufactures, and commercializes through partnership on innovative biologics for autoimmune diseases, oncology and metabolic diseases, reported that the Biologics License Application (BLA) submission for felzartamab, an investigational differentiated CD38 antibody, has been accepted for review by China’s National Medical Products Administration (NMPA) for the treatment of multiple myeloma (MM) (Press release, TJ Bio, JAN 10, 2025, View Source [SID1234654001]). This milestone, following the recent BLA submission for eftansomatropin alfa, demonstrates TJ Bio’s proven capability to advance late-stage pipeline products through its full-fledged R&D and manufacturing infrastructure.

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In China, where multiple myeloma affects over 160,000 patients, there remains a significant unmet need for more accessible treatment options. Felzartamab’s Phase 3 registration studies have demonstrated a differentiated profile with comparable efficacy to existing therapies, while offering advantages in safety through lower and less severe infusion-related reactions and improved convenience with shorter infusion time from 6 hours to 1.5 hours, particularly benefiting elderly patients who constitute a large portion of the multiple myeloma population.

With TJ Bio’s state-of-the-art GMP facility in Hangzhou receiving the Drug Manufacturing Permit (License A) in August 2024, felzartamab is on track to become China’s first domestically manufactured CD38 therapy. This manufacturing capability ensures reliable domestic supply to meet the growing demand for innovative biologics while providing Chinese patients with more accessible treatment options.

"This BLA acceptance for felzartamab, coming shortly after eftansomatropin alfa, demonstrates TJ Bio’s strong execution capabilities and our commitment to advancing innovative therapies," said Dr. Jingwu Zang, Founder and Chairman of TJ Bio. "While this first regulatory milestone positions us to potentially deliver China’s first domestically manufactured CD38 therapy, we are particularly excited about felzartamab’s broader potential in autoimmune diseases. As we advance its development as a novel plasma cell depletion therapy across multiple autoimmune indications, these consecutive BLA submissions and our newly licensed manufacturing facility exemplify our ability to execute on our vision of bringing innovative biologics to patients in need."

About Felzartamab
Felzartamab is an investigational therapeutic human monoclonal antibody directed against CD38, a protein expressed on mature plasma cells. Scientific research suggests that anti-CD38 antibodies have therapeutic potential in multiple disease areas across immuno-oncology and autoimmune diseases. Beyond its initial indication in multiple myeloma, felzartamab has been shown in clinical studies to selectively deplete CD38+ plasma cells, which may allow applications that ultimately improve clinical outcomes in a broad range of diseases driven by pathogenic antibodies.

Felzartamab was originally developed by MorphoSys AG (now MorphoSys GmbH, a Novartis company). TJ Bio owns the exclusive rights for development and commercialization of felzartamab for all indications in Greater China, which encompasses Mainland China, Hong Kong, Macao, and Taiwan. Human Immunology Biosciences (HI-Bio) exclusively licensed the rights to develop and commercialize felzartamab across all indications in all countries and territories excluding Greater China. Biogen acquired HI-Bio in July 2024.

About Multiple Myeloma

Multiple myeloma is a hematological malignancy that affects plasma cells, with over 160,000 patients in China requiring treatment. The disease primarily affects elderly patients, who are particularly vulnerable to treatment-related complications. Despite recent therapeutic advances, there remains a significant need for treatments that combine efficacy with improved safety profiles and greater convenience.

The current treatment landscape in China faces challenges including high rates of infusion-related reactions with existing therapies, lengthy infusion times that burden both patients and healthcare systems, and limited access to innovative treatments due to cost considerations. These factors underscore the urgent need for domestically manufactured therapies that can provide effective, safer, and more accessible treatment options.