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."

On February 12, 2025 Opna Bio, a clinical-stage biopharmaceutical company focused on the discovery and development of novel oncology therapeutics, reported that the FDA has granted orphan drug designation (ODD) to one of its lead programs, OPN-6602, for the treatment of multiple myeloma (MM) (Press release, Opna Bio, FEB 12, 2025, View Source [SID1234650226]). OPN-6602 is an oral, small molecule inhibitor of the E1A binding protein (EP300) and CREB-binding protein (CBP) currently being tested in a Phase 1 trial in patients with relapsed or refractory MM.

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Multiple myeloma is a rare and aggressive cancer of the plasma cells in the bone marrow that often leads to serious complications such as bone damage, kidney failure and immune suppression. The disease is typically diagnosed in older adults, with limited treatment options available for patients with relapsed or refractory disease.

"We are pleased to have received ODD for OPN-6602 for the treatment of multiple myeloma, a further validation of the drug’s therapeutic potential in patients with this disease who have limited treatment options once they have relapsed," said Gideon Bollag, PhD, chief scientific officer.

Orphan Drug Designation is granted by the FDA to encourage the development of therapies for rare diseases, which are defined as those affecting fewer than 200,000 people in the U.S. The designation provides several benefits, including tax credits for clinical trial costs, a waiver of certain FDA fees, and eligibility for seven years of market exclusivity upon approval.

Opna recently presented data at the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December 2024 showing that in human-derived multiple myeloma models, OPN-6602 suppresses tumor growth, while downregulating key MM driving genes. Synergistic effects were observed with OPN-6602 in combination with dexamethasone, pomalidomide and mezigdomide. OPN-6602’s distinct pharmacokinetic profile allows for continuous daily dosing that potentially results in a lower incidence of toxicities and improved efficacy.

The Phase 1 study in patients with relapsed or refractory multiple myeloma (NCT06433947) is taking place at multiple sites in the U.S. Opna Bio expects to complete the single agent, dose-escalation phase of the trial in 2026. Further development of OPN-6602 in combination with other standard-of-care agents in multiple myeloma is planned.

On February 12, 2025 OnCusp Therapeutics, Inc., a clinical stage biopharmaceutical company dedicated to transforming cutting-edge preclinical innovation into clinically validated treatments for cancer patients, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to CUSP06, a Cadherin-6 targeting Antibody-Drug Conjugate (CDH6 ADC) and the company’s lead program, for the treatment of patients with platinum-resistant ovarian cancer (PROC) (Press release, OnCusp Therapeutics, FEB 12, 2025, View Source [SID1234650224]).

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"We are extremely pleased that the FDA granted Fast Track Designation to CUSP06," said Eric Slosberg, PhD, Chief Development Officer of OnCusp Therapeutics. "The early results from our Phase 1 trial have been encouraging, and this designation will expedite our efforts to bring this potentially transformative therapy to patients. Given the need for new therapeutic options in this underserved population, we are committed to working closely with the FDA to accelerate its development."

The ongoing Phase 1 multicenter study is evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of CUSP06 in adults with platinum-refractory/resistant ovarian cancer and other advanced solid tumors (CUSP06-1001). Early data from the trial have shown promising anti-tumor activity and a manageable safety profile, supporting further development of the program.

About CUSP06

CUSP06, a CDH6 ADC, is composed of a proprietary antibody with high CDH6 binding affinity, a protease-cleavable linker, and an exatecan payload (a potent and clinically validated topoisomerase-1 inhibitor). The linker is designed to complement the exatecan payload, enabling a stable and homogenous ADC. The payload is a weak substrate for BCRP/P-gp, which are drug efflux pumps that drive chemoresistance to many therapies. In preclinical data, this linker-payload has been shown to have an increased "bystander effect" compared to competitor ADCs. CUSP06 has a drug-to-antibody ratio of eight. OnCusp obtained the exclusive global rights (outside of China) to lead the development and commercialization of CUSP06 from Multitude Therapeutics in 2022. CUSP06 is being evaluated in a Phase 1 study in patients with platinum-refractory/resistant ovarian cancer and other advanced solid tumors. Additional information on the CUSP06-1001 (NCT06234423) trial can be found at clinicaltrials.gov.

About Platinum-Resistant Ovarian Cancer

Ovarian cancer is the leading cause of death from gynecologic malignancies in the United States, with approximately 20,000 new cases diagnosed annually.[1] Platinum-based chemotherapy is a standard first-line treatment, but approximately 80% of patients who respond to initial treatment will develop platinum resistance, typically within two years.[2] Platinum-resistant ovarian cancer is defined as disease progression within six months of completing platinum-based therapy and carries a particularly poor prognosis, with median survival of only 9-12 months and limited effective treatment options.[2] Despite recent therapeutic advances, the 5-year survival rate for patients with platinum-resistant disease remains around 15%, highlighting a critical unmet need for new therapeutic approaches.[1],[3]

About Fast Track Designation

Fast Track Designation is intended to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need. Programs with FTD may benefit from more frequent meetings with the FDA, eligibility for Accelerated Approval and Priority Review if relevant criteria are met, and the potential for a rolling review of the Biologics License Application (BLA).

On February 12, 2025 GRAIL, Inc. (Nasdaq: GRAL), a healthcare company whose mission is to detect cancer early when it can be cured, and Quest Diagnostics (NYSE: DGX), a leading provider of diagnostic information services, reported the initial phase of a program to improve provider access to GRAIL’s Galleri multi-cancer early detection (MCED) test (Press release, Grail, FEB 12, 2025, View Source [SID1234650223]). Providers can now order the Galleri test directly from GRAIL through the Quest Diagnostics connectivity system. The Quest Diagnostics connectivity system enables providers in the United States to order and receive reports of laboratory tests electronically through Quest’s Quanum laboratory portal and more than 900 electronic health record (EHR) systems. More than 500,000 providers used the Quest connectivity system last year.

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The integration will help streamline the process of ordering the Galleri test for physicians. It will also increase availability by allowing patients access to the test at any of Quest’s approximately 7,400 patient access points nationwide. Patients with a test order from their physician can now go directly to Quest without needing to bring a Galleri test kit to the blood draw appointment.

"Quest Diagnostics and GRAIL share a commitment to improving access to cancer screening and have worked productively together to enable patient access to GRAIL’s Galleri test via Quest’s phlebotomy network since 2021," said Mark Gardner, Senior Vice President, Molecular Genomics and Oncology for Quest Diagnostics. "Integrating GRAIL’s Galleri test into the Quest connectivity system is the next step in this collaboration. We expect it to increase patient access by giving Quest’s provider clients the ability to seamlessly order the test through Quest, same as they do for other blood work. This collaboration brings to life the tremendous value of Quest’s ability to scale diagnostic innovation to make it accessible for all."

Cancers growing in the body shed DNA into the bloodstream. These DNA fragments act like a unique "fingerprint" of cancer. With a single blood draw, the Galleri test screens for the "fingerprint" of many of the deadliest cancers before they become symptomatic, including those with no recommended screening tests today. It can also provide doctors with information on the cancer’s origin to help predict the tissue type or organ associated with the cancer signal. The Galleri test is prescription only, recommended for adults with an elevated risk for cancer, such as those age 50 or older, and is to be used in addition to recommended cancer screenings.

"We know every minute counts for busy providers, their staff and their patients, which is why we’re so pleased to work with Quest to offer a seamless experience that fits into providers’ existing ordering process," said Josh Ofman, MD, MSHS, President at GRAIL. "While today there are recommended screenings for five cancers, nearly 70% of deaths are caused by cancers with no recommended screening test. We believe this integration will help to make it easier to incorporate the Galleri test into routine exams to help screen for cancer before it becomes symptomatic when outcomes may be improved."