On February 12, 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 an update from the Company’s ongoing Phase 1/1b clinical trial evaluating BP1002 for the treatment of refractory/relapsed acute myeloid leukemia (AML), including venetoclax-resistant patients (Press release, Bio-Path Holdings, FEB 12, 2025, View Source [SID1234650242]). The Company announced a meaningful patient response to treatment and the clinical trial has progressed to the fourth, higher dose cohort of 90 mg/m2.

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"We were excited to learn that one patient in the third cohort had a meaningful response to just one treatment cycle, experiencing stable disease and a significant reduction in blast count, which we believe offers promise for venetoclax-resistant AML patients with limited treatment options," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. "AML patients that fail or relapse from frontline venetoclax-based therapy have very poor prognosis with a median overall survival of less than three months. The third dosing cohort completed enrollment faster than expected, which we believe reflects the urgent need for additional treatment options. We look forward to quickly advancing this study through the fourth dosing cohort and into the combination therapy segment of this Phase 1/1b study with increased levels of BP1002 for the treatment of these vulnerable patients."

The current standard of care for patients with AML not eligible for intensive chemotherapy is venetoclax, an oral Bcl-2 inhibitor that targets the BH3 domain of the Bcl-2 protein, in combination with a hypomethylating agent or with low-dose cytarabine. However, many patients become resistant to venetoclax treatment. A published study found that AML patients who had relapsed from frontline venetoclax-based treatment were refractory to salvage therapy and had a median survival of less than 3 months. By targeting Bcl-2 at the mRNA level rather than the protein, BP1002 may overcome and prevent some of the mechanisms of resistance that affect venetoclax treatment.

The Phase 1/1b clinical trial is being conducted at several leading cancer centers in the United States, including the Weill Medical College of Cornell University, The University of Texas MD Anderson Cancer Center, Scripps Health, and The University of California at Los Angeles Cancer Center. The approved treatment cycle is two doses per week over four weeks, resulting in eight doses administered over twenty-eight days. The Phase 1b portion of the study is expected to commence after completion of BP1002 monotherapy cohorts and will assess the safety and efficacy of BP1002 in combination with decitabine in refractory/relapsed AML patients.

Gail J. Roboz, M.D., is the National Principal Investigator for the Phase 1/1b trial. Dr. Roboz is a Professor of Medicine and Director of the Clinical and Translational Leukemia Program at the Weill Medical College of Cornell University and the New York-Presbyterian Hospital in New York City. Gary Schiller, M.D., The University of California at Los Angeles Cancer Center, Maro Ohanian, D.O., Department of Leukemia, University of Texas MD Anderson Cancer Center, and David Hermel, M.D., Scripps Health, are each serving as principal investigators.

On February 12, 2025 Terrain Biosciences reported the company launches from stealth as the world’s first RNA design-build company, using advanced AI and its proprietary manufacturing platform to accelerate development of the next generation of programmable medicines (Press release, Terrain Biosciences, FEB 12, 2025, View Source [SID1234650230]). RNA-based modalities, including mRNA, cell & gene therapies, and emerging personalized cancer vaccines are promising and rapidly advancing, with hundreds of drug candidates in development for indications ranging from oncology to infectious disease to neurodegenerative illnesses. Despite incredible progress, innovators in the space continue to face challenges in designing, selecting, manufacturing, and delivering optimal customized RNA sequences for therapeutic benefit.

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Founded by Professors Omar Abudayyeh and Jonathan Gootenberg of Harvard Medical School and Professor Patrick Hsu of the Arc Institute and University of California, Berkeley, the company is led by industry veterans including CSO Aaron Larsen (formerly at Beam & Moderna), COO Ted Tisch (formerly at Mammoth Biosciences and Synthego), and founding CEO Chetan Tadvalkar (veteran GM/COO of pharma services companies, including former head of business operations at Ginkgo Bioworks). Terrain has raised $9M in seed funding to bring cutting-edge research in RNA design and manufacturing to innovators building RNA-based therapies and vaccines, and support development of personalized cancer vaccines. Over a dozen biotechs already trust Terrain Bio to help them identify and produce sequences with better expression, greater durability, and optimal manufacturability, accelerating their path to the clinic.

"AI is poised to dramatically accelerate how we design and develop drugs, but the reality is that in silico models can only get us so far in RNA — bringing a drug from the lab to the clinic requires bringing advanced science into the real world. Our partners need excellent machine learning tools combined with a real manufacturing platform and data to enable and accelerate their path to the clinic," said Terrain co-founder Omar Abudayyeh.

"Our partners are brilliant scientists on an incredibly challenging journey to develop new medicines and ultimately help patients," said Chetan Tadvalkar, CEO of Terrain. "Our team has been part of similar journeys before, and today our purpose is to help scientists navigate the complexity of designing the right RNA sequence and guide them towards their goal while ensuring they hit key preclinical milestones."

RNA optimization is critical for therapeutic efficacy and safety — sequence and purity reaches beyond the mechanism of action to have broad impacts across expression, stability, durability, immunogenicity, and manufacturability. "The search space for designing RNA is nearly infinite," said co-founder Jonathan Gootenberg. "Navigating that space computationally and experimentally will unlock enormous potential in medicine, but remains incredibly challenging given the lack of top-quality data for training better models."

"Bridging design and manufacturing in a tight feedback loop means higher quality preclinical data, which means stronger generative models for design, which means better drug candidates," said co-founder Patrick Hsu. "Terrain is all about making the tools for this kind of rapid iteration possible for delivering better RNA."

On February 12, 2025 HAYA Therapeutics, SA, a biotechnology company pioneering precision RNA-guided regulatory genome targeting therapeutics for rare, common, chronic and age-associated diseases, reported that it has been awarded the prestigious Innosuisse Certificate for Sustainable Growth from Innosuisse, the Swiss Innovation Agency, who also provided funding to support HAYA’s research into using its technology to address cancer (Press release, Haya Therapeutics, FEB 12, 2025, View Source [SID1234650229]).

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In addition to Innosuisse’s award for HAYA’s potential for growth and success, it is providing 1.5M CHF (approximately $1.64M USD) in non-dilutive funding for HAYA through Innosuisse’s Start-up Innovation Projects, recognizing HAYA’s cutting-edge approach and broad applicability of their long non-coding RNA (lncRNA) platform in developing disease-modifying therapeutics beyond the heart and into oncology.

The funds will be used over the next two years for translational studies to further develop an innovative therapy targeting cancer-associated fibroblast-specific lncRNA for aggressive solid tumors.

"Solid tumors, which make up approximately 90% of all cancers, require innovative therapeutic strategies due to their complexity and resistance to traditional treatments," said Samir Ounzain, Ph.D., Co-founder and CEO of HAYA Therapeutics. "Fibrosis plays a critical role in shaping the tumor microenvironment, creating a rigid and immunosuppressive barrier that fuels cancer progression and limits treatment efficacy. At HAYA, we are pioneering a novel RNA-guided approach to precisely reprogram cancer-associated fibroblasts, disrupting the fibrotic niche and unlocking new possibilities for more effective and durable cancer therapies."

"By leveraging the exquisite cell specificity of lncRNAs coupled with antisense oligonucleotide’s broad distribution to extra hepatic tissues including tumors, HAYA is able to drug disease modifying targets within the tumor microenvironment. This novel paradigm holds tremendous potential as a next generation treatment strategy for immunotherapy resistant solid tumors such as for pancreatic cancer and triple negative breast cancer," said Sudhir Agrawal, DPhil, FSRC, a member of HAYA’s scientific advisory board and Founder of Arnay Sciences. "HAYA’s strategy could be transformational and offer disease-modifying therapy options to patients in need."

HAYA’s unique RNA-guided regulatory genome technology enables the discovery and development of innovative RNA-based therapeutics for a wide range of diseases. HAYA’s lead candidate, HTX-001, is being developed for the treatment of heart failure, with other preclinical therapeutics in development for other indications.

On February 12, 2025 Ferring Pharmaceuticals reported three abstracts featuring ADSTILADRIN (nadofaragene firadenovec-vncg) will be presented at the 2025 American Society of Clinical Oncologists Genitourinary Cancers (ASCO GU) Symposium held February 13-15 in San Francisco (Press release, Ferring Pharmaceuticals, FEB 12, 2025, View Source [SID1234650228]). ADSTILADRIN is the first and only intravesical non-replicating gene therapy approved by the U.S. Food and Drug Administration (FDA) for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1).

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Mayo Clinic will present additional updated early results in a poster presentation of an independent, retrospective real-world outcomes study conducted at three of their sites. The study is evaluating complete response rates, high-grade recurrence-free survival, cystectomy-free survival, overall survival, and adverse events among patients treated with ADSTILADRIN for high-risk BCG-unresponsive NMIBC.

"Understanding real-world outcomes in patients with BCG-unresponsive NMIBC is essential for informing how we approach treatment and deliver care," said Mark D. Tyson, M.D., urologic oncologist and associate professor, Mayo Clinic, Scottsdale, Ariz., and principal investigator of this independent study. "These initial encouraging post-marketing results provide valuable insights into the benefits of using ADSTILADRIN in everyday clinical practice, allowing providers to make more informed decisions and deliver the best possible care for patients."

Additional abstracts to be presented include the study protocols for two studies in the ABLE (ADSTILADRIN in BLadder CancEr) clinical trial program – the three-arm ABLE-22 study and the two-arm ABLE-32 study. Clinical sites were recently activated for both studies which are investigating the efficacy and safety of ADSTILADRIN in high-risk and intermediate-risk NMIBC.

"These real-world data extend the body of evidence for efficacy, safety, and low treatment burden of ADSTILADRIN in NMIBC patients who no longer respond to BCG, building on the positive five-year results from our pivotal Phase 3 trial announced last year," said Bipin Dalmia, Global Head of Uro-Oncology & Urology Franchise, Ferring Pharmaceuticals. "As the first and only FDA approved intravesical bladder-sparing monotherapy for BCG-unresponsive NMIBC patients, ADSTILADRIN is fully available throughout the United States, offering a convenient quarterly dosing schedule that may help encourage treatment adherence. The continuation of our clinical trial program further highlights our commitment to advancing the science in our journey to establish ADSTILADRIN as the new standard of care and backbone therapy across the urothelial cancer disease spectrum."

About ADSTILADRIN Presentations at ASCO (Free ASCO Whitepaper) GU
ADSTILADRIN poster titles and presentation times at ASCO (Free ASCO Whitepaper) GU, February 13-15, 2025, are:

Real-world outcomes of nadofaragene firadenovec in BCG-unresponsive non-muscle invasive bladder cancer. Abstract #716 (Poster #D13), Friday, February 14, 11:30 a.m. PST
ABLE-22: Safety and efficacy evaluation of nadofaragene firadenovec alone or in combination with chemotherapy or immunotherapy—a randomized, open-label, phase 2 study. Abstract #TPS891 (Poster #J19), Friday, February 14, 11:30 a.m. PST
ABLE-32: A randomized, controlled, phase 3B clinical trial of nadofaragene firadenovec-vncg versus observation in patients with intermediate-risk non–muscle-invasive bladder cancer. Abstract #TPS888 (Poster #J16), Friday, February 14, 11:30 a.m. PST
About ABLE-22 and ABLE-32
ABLE-22 (NCT06545955) is a three-arm, Phase 2 trial evaluating ADSTILADRIN as a monotherapy and as part of combination with chemotherapy or an immune checkpoint inhibitor in patients with high-risk BCG-unresponsive NMIBC. Investigators in all three arms of the ABLE-22 study will have the option to re-induce appropriate patients who do not achieve a complete response to the initial single dose or combination regimen, an option that was not included in the original Phase 3 study.

The Phase 3B ABLE-32 (NCT06510374) trial is assessing ADSTILADRIN in patients with intermediate-risk NMIBC (IR NMIBC), for which there are no U.S. FDA-approved treatment options.

About ADSTILADRIN
ADSTILADRIN (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer.

ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-risk, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).1

Ferring is leading the future in uro-oncology treatment with ADSTILADRIN at the center, while expanding access with the support of new, state-of-the-art manufacturing facilities. As announced in January 2024, ADSTILADRIN is fully available and accessible in the U.S. ADSTILADRIN has confirmed 99 percent coverage for commercial and government-insured patients. As of April 1, 2024, in accordance with the Centers for Medicare and Medicaid Services (CMS), ADSTILADRIN established an Average Sales Price (ASP). Since the establishment of ASP, all covered claims submitted for reimbursement have received payment within an average of 25 days.2

Ferring is committed to investing in novel therapies, developing life-changing solutions that address unmet medical needs, and aiding the uro-oncology community in helping patients live better lives. More information is available in the U.S. at FerringUroOncology.com and on the dedicated Ferring Uro-Oncology channels on LinkedIn and X.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
NMIBC is a form of bladder cancer that is found in the inner layer cells of the bladder and does not invade into or beyond the muscle wall.3 In the United States, bladder cancer is the sixth most common cancer,4 fourth among men,5 and it is estimated that there will be approximately 84,870 new cases of bladder cancer in the U.S. in 2025.5 Historically, 75% of bladder cancer presents as NMIBC.6 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care. However, approximately one third of patients with NMIBC will not respond to BCG therapy and 50% of those with an initial response will experience recurrence or progression of their disease.7 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder).8

INDICATION
ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product.

WARNINGS AND PRECAUTIONS:

Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration.

USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose.

ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination).

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.

Please click to see the full Prescribing Information.

On February 12, 2025 Artera, the developer of multimodal artificial intelligence (MMAI)-based prognostic and predictive cancer tests, and Tempus, a technology company leading the adoption of AI to advance precision medicine and patient care, reported that they will collaborate to expand access to the ArteraAI Prostate Test (Press release, Tempus, FEB 12, 2025, View Source [SID1234650227]). Through the collaboration, Tempus and Artera will work together in an exclusive manner to commercialize Artera’s prostate cancer risk stratification test. Tempus is currently connected to more than 50% of all oncologists practicing in the United States.

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"We are looking forward to collaborating with Artera to expand access to Artera’s current prostate cancer test," said Ezra Cohen, MD, Chief Medical Officer at Tempus. "Management of localized prostate cancer has always been a balance between doing too much and not enough. Now, by combining our efforts, we can empower clinicians and patients with critical insights to allow an informed decision at this crucial point in their treatment journey, ensuring that each patient receives the care that is right for them."

The ArteraAI Prostate Test is an AI-enabled test that provides predictive and prognostic results for patients with localized prostate cancer. The first of its kind, the test provides actionable insights to empower shared decision-making between the patient and clinician to help personalize treatment plans. Test results can be received in 1-2 days after receiving the patient’s specimen. The test is the first AI risk stratification tool for patients with localized prostate cancer recommended by the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Prostate Cancer, and the only test included for its predictive performance regarding the use of short-term androgen deprivation therapy (ADT). The company will roll out new tests for additional indications in the future.

"Collaborating with Tempus is a powerful validation of the hard work and dedication our team at Artera has put into advancing precision medicine," said Andre Esteva, CEO and co-founder of Artera. "With Tempus’ unmatched expertise, depth, and broad reach, this collaboration strengthens our shared mission to revolutionize patient care and drive breakthroughs in the field of oncology."