Ferring Pharmaceuticals SA

PARSIPPANY, N.J.--(BUSINESS WIRE)-- Ferring Pharmaceuticals today announced it will present analyses of data for REBYOTA® (fecal microbiota, live – jslm), the first and only single-dose, FDA approved microbiome-based treatment to prevent recurrent Clostridioides difficile (C. diff) infection after antibiotic treatment, as part of Infectious Disease Week (IDWeek) 2023. The congress will take place in Boston, Mass., from October 11 to 15. This press release features multimedia. View the full release here: Data presented at IDWeek will evaluate initial real-world use of REBYOTA in gastroenterology and infectious disease clinical practices, efficacy and safety of REBYOTA in patients with renal comorbidities, and efficacy of REBYOTA in patients who received antibiotics for another illness after treatment with REBYOTA. An additional analysis will focus on associations between a healthier gut microbiome and health-related quality of life. Descriptions of the abstracts accepted for presentation are as follows: Poster 685 Title – Early Experience With a Simple Administration of a Novel Fecal Microbiome Replacement for Prevention of Recurrent Clostridioides difficile Presenting Author: Richard L. Hengel, M.D., Partner, Atlanta ID Group, Atlanta, Ga. EMBARGOED UNTIL: Wednesday, October 11, 2023, at 12:01 a.m. ET Poster 706 Title – An Ad Hoc Subgroup Analysis of a Phase 3, Open-Label Study Indicates Efficacy and Safety of Fecal Microbiota, Live – jslm in Participants With Recurrent Clostridioides difficile Infection and Renal Impairment Presenting Author: Glenn Tillotson, Ph.D., F.R.S.M., F.C.C.P., GST Micro, North, Va. EMBARGOED UNTIL: Wednesday, October 11, 2023, at 12:01 a.m. ET Poster 702 Title – Efficacy of Fecal Microbiota, Live – jslm After Receipt of Non-Clostridioides difficile Infection Antibiotics: Post Hoc Subgroup Analysis of a Phase 2 Open-Label Trial Presenting Author: Andrew Skinner, M.D., University of Utah School of Medicine and George E. Wahlen VA Hospital, Salt Lake City, Utah EMBARGOED UNTIL: Wednesday, October 11, 2023, at 12:01 a.m. ET Poster 366 Title – Association Between Gut Microbiota and Health-Related Quality of Life in Patients with Recurrent Clostridioides difficile Infection: Results from the PUNCH CD3 Clinical Trial Presenting Author: Paul Feuerstadt, M.D., F.A.C.G., A.G.A.F., PACT Gastroenterology, Hamden, Conn.; Assistant Clinical Professor of Medicine, Yale University School of Medicine, New Haven, Conn. EMBARGOED UNTIL: Wednesday, October 11, 2023, at 12:01 a.m. ET IDWeek 2023 has made abstracts available on its website. About C. diff infection C. diff infection is a serious and potentially deadly infection that impacts people across the globe. The C. diff bacterium causes debilitating symptoms, such as severe diarrhea, fever, stomach tenderness or pain, loss of appetite, nausea and colitis (an inflammation of the colon).1 C. diff infection can be the start of a vicious cycle of recurrence, causing a significant burden for patients and the healthcare system.2,3 It has been estimated that up to 35% of C. diff infection cases recur after initial diagnosis and people who have had a recurrence are at significantly higher risk of further infections.4,5,6,7 After the first recurrence, it has been estimated that up to 65% of patients may develop a subsequent recurrence.6,7 Antibiotics – the current standard of care for treatment of C. diff infection – treat the disease but can also be a contributing factor to the cycle of recurrence.1 About REBYOTA REBYOTA is a pre-packaged, single-dose 150 mL microbiota suspension for rectal administration consisting of a liquid mix of up to trillions of live microbes – including Bacteroides. REBYOTA is delivered directly to the gut microbiome and is administered by a healthcare professional in one visit. INDICATION REBYOTA (fecal microbiota, live – jslm) is indicated for the prevention of recurrence of Clostridioides difficile (C. diff) infection in individuals 18 years of age and older, following antibiotic treatment for recurrent C. diff infection. Limitation of Use REBYOTA is not indicated for the treatment of C. diff infection. IMPORTANT SAFETY INFORMATION You should not receive REBYOTA if you have a history of a severe allergic reaction (e.g., anaphylaxis) to REBYOTA or any of its components. You should report to your doctor any infection you think you may have acquired after administration. REBYOTA may contain food allergens. Most common side effects may include stomach pain (8.9%), diarrhea (7.2%), bloating (3.9%), gas (3.3%), and nausea (3.3%). REBYOTA has not been studied in patients below 18 years of age. Clinical studies did not determine if adults 65 years of age and older responded differently than younger adults. You are encouraged to report negative side effects of prescription drugs to FDA. Visit or call 1-800-332-1088. Please click to see the full Prescribing Information. About Ferring Pharmaceuticals Ferring Pharmaceuticals is a research-driven, specialty biopharmaceutical group committed to helping people around the world build families and live better lives. In the United States, Ferring is a leader in reproductive medicine and maternal health, uro-oncology and in specialty areas within gastroenterology, including microbiome therapeutics, and orthopaedics. For more information, call 1-888-FERRING (1-888-337-7464) or visit . Connect with us on our dedicated microbiome therapeutics development channels on Twitter and LinkedIn. About IDWeek IDWeek is the joint annual meeting of the Infectious Diseases Society of America (IDSA), Society for Healthcare Epidemiology of America (SHEA), the HIV Medicine Association (HIVMA), the Pediatric Infectious Diseases Society (PIDS) and the Society of Infectious Diseases Pharmacists (SIDP). IDWeek is a recognized forum for peer-reviewed presentations of new research on scientific advances and bench-to-bedside approaches in prevention, diagnosis, treatment and epidemiology of infectious diseases, including HIV, across the lifespan. For more information, visit . References: Centers for Disease Control and Prevention. What is C. diff? 7 Sep. 2022. Available at: . Centers for Disease Control and Prevention. 2019 Antibiotic Resistance Threats Report: Clostridioides difficile. 23 Nov. 2021. Available at: . Feuerstadt P, et al. Healthcare resource utilization and direct medical costs associated with index and recurrent Clostridioides difficile infection: a real-world data analysis. J Med Econ. 2020;23(6):603-609. Riddle DJ, Dubberke ER. Clostridium difficile infection in the intensive care unit. Infect Dis Clin North Am. 2009;23(3):727-743. Nelson WW, et al. Health care resource utilization and costs of recurrent Clostridioides difficile infection in the elderly: a real-world claims analysis. J Manag Care Spec Pharm. 2021 Jul;27(7):828-838. doi: 10.18553/jmcp.2021.20395. Epub 2021 Mar 11. Kelly, CP. Can we identify patients at high risk of recurrent Clostridium difficile infection? Clin Microbiol Infect. 2012;18(Suppl. 6):21-27. Smits WK, et al. Clostridium difficile infection. Nat Rev Dis Primers. 2016;2:16020. doi: 10.1038/nrdp.2016.20.

While Ferring is looking at several different models, manufacturing of the gene therapy Adstiladrin is expected to hit full capacity by 2025. After securing approval at the end of last year for a potential alternative to bladder removal in certain bladder cancer patients, Ferring Pharmaceuticals has given the gene therapy Adstiladrin to its first commercial patient.   The patient, a 78-year-old man in the Midwest, is the first of many to receive treatment through Ferring’s early experience program. The program is meant to ensure patients can access the drug during a major manufacturing scale-up. Because the adenovirus vector-based therapy is complicated to make, the company knew it had to be “very, very responsible” to ensure it was getting the med to patients while ramping up manufacturing, Ferring’s chief scientific officer Elizabeth Garner, M.D., MPH, said in an interview.   Two new sites were cleared to specifically produce Adstiladrin earlier this summer, one in Finland and the other in New Jersey. Manufacturing should hit full capacity by 2025 when Ferring expects to receive $200 million in milestone payments from Royalty Pharma through a royalty deal worth up to $500 million. For now, investigators who studied Adstiladrin in its phase 3 trial and community clinics with the highest number of eligible patients can participate in the early experience program.  More clinics will be invited to join the program as manufacturing output increases. The program will continue to expand in the next year or so, Garner said. Importantly, the framework makes sure that any patient dosed during the first round can come back for their entire treatment regimen of every three months stretched out over a minimum of one year, despite the tight supply. The recently treated patient scheduled his next appointment for December. Adstiladrin was approved last December to treat high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer with carcinoma in situ (CIS) with or without papillary tumors. It’s the first gene therapy to target high-risk and non-muscle invasive bladder cancer in patients who no longer respond to standard therapy and is administered directly into the bladder from a catheter. The patient was also enrolled in Ferring’s real-world evidence study, called ABLE-41. The study looks to enroll up to 800 patients.  Through the study, the company will analyze data evaluating not only the drug’s effectiveness through measures such as durability, survival and complete response rates but also patient, caregiver and physician experience. Those outcomes will be determined through several questionnaires that can give the company a better picture of what the quality of life looks like for patients treated and the impact on their caregivers, Garner said. ABLE-41 is expected to last up to two years.

Urologists treating eligible high-risk bladder cancer patients at participating early experience program clinics in the U.S. can now prescribe the first and only FDA-approved intravesical gene therapy Ferring launches ABLE-41 Real World Evidence Study, a patient registry to explore early use, experiences and outcomes of ADSTILADRIN in a real-world setting Ferring on track to increase manufacturing supplies of new gene therapy ADSTILADRIN PARSIPPANY, N.J.--(BUSINESS WIRE)-- Ferring Pharmaceuticals today announced the first patient in the United States with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) was dosed with the commercially available intravesical gene therapy ADSTILADRIN® (nadofaragene firadenovec-vncg) as part of the ADSTILADRIN Early Experience Program announced earlier this year. ADSTILADRIN was approved by the U.S. Food & Drug Administration (FDA) in December 2022 for the treatment of adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors. It is the first and only FDA-approved intravesical gene therapy for adults with NMIBC who no longer respond to standard therapy. “The first patient dosed with ADSTILADRIN marks an incredible milestone for Ferring, the bladder cancer community and the patients we aim to serve,” said Shetal Vyas, Vice President, General Manager, Uro-Oncology at Ferring Pharmaceuticals. “Making this novel and efficacious intravesical gene therapy commercially available for patients underscores our commitment to changing the trajectory of NMIBC. We will continue bringing available doses of ADSTILADRIN forward responsibly as we scale up manufacturing.” The patient, a 78-year-old male treated at a clinic within the mid-west area also was enrolled in the non-interventional study, the “ADSTILADRIN in BLadder CancEr” (ABLE-41) U.S. Real World Evidence (RWE) Study. The research study, launched in tandem with participating Early Experience Program clinics, will explore early utilization, experiences, and outcomes of ADSTILADRIN in the routine care setting. Learn more at . “ADSTILADRIN represents a major advancement in the current treatment landscape for people living with high-risk NMIBC who may be facing removal of the bladder,” said Elizabeth Garner, MD, MPH, Chief Scientific Officer at Ferring Pharmaceuticals. “The ADSTILADRIN Early Experience Program and ABLE-41 US RWE Study allow us to address pressing patient needs while collecting data on its use in a real-world setting, further expanding on what we’ve learned in our Phase 3 clinical program.” Earlier this month, Ferring made doses of ADSTILADRIN commercially available through an Early Experience Program to urologists at the clinical trial sites that participated in the Phase 3 study1 and a mix of community clinics with the highest number of eligible high-risk patients with NMIBC. The Early Experience Program is designed to ensure every patient who starts on therapy will continue to receive future doses of ADSTILADRIN for the duration of their treatment. The ADSTILADRIN Early Experience Program is temporary, and more clinics will be offered the opportunity to participate as manufacturing volumes steadily increase over time. About the ABLE-41 (Adstiladrin in BLadder CancEr) US RWE Study The ABLE-41 US RWE Study is a real-world observational study of ADSTILADRIN focused on patient treatment outcomes and early use experiences in U.S. routine care settings. Up to 800 patients enrolled in the ADSTILADRIN Early Experience Program who previously have not received ADSTILADRIN will be followed for a minimum of 24 months. The effectiveness of ADSTILADRIN will be measured as complete response rates. Data analyzed will examine patterns of ADSTILADRIN use, and overall experience among patients, caregivers and healthcare providers. About ADSTILADRIN ADSTILADRIN® (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical 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 encoding interferon alfa-2b protein, administered 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 quantities 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-grade, 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 About Non-Muscle Invasive Bladder Cancer (NMIBC) NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.2 Bladder cancer is the sixth most common cancer in the U.S., and it is estimated that there were approximately 82,290 new cases of bladder cancer in the U.S. in 2022,3 75% of which present as NMIBC.4 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care. However, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease.2 Current treatment options for BCG-unresponsive patients are very limited, and often result in a highly invasive life-changing procedure of radical cystectomy (complete removal of the bladder).5 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 hypersensitivity to interferon alfa or 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 or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING. About Ferring Pharmaceuticals Ferring Pharmaceuticals is a research-driven, specialty biopharmaceutical group committed to helping people around the world build families and live better lives. In the United States, Ferring is a leader in reproductive medicine and maternal health, uro-oncology and in specialty areas within orthopaedics and gastroenterology, including microbiome therapeutics. For more information, call 1-888-FERRING (1-888-337-7464) or visit . References: ADSTILADRIN in Patients With High Grade, Bacillus Calmette-Guérin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC). Gov Identifier: NCT02773849. . Boorjian SA, Alemozaffar M, Konety BR, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet Oncol 2021; 22: 107–17. American Cancer Society. Key Statistics for Bladder Cancer. . Updated January 12,2022. Accessed November 22, 2022. Burger M, Catto JW, Dalbagni G, et al. Epidemiology and risk factors of urothelial bladder cancer. Eur Urol. 2013;63(2):234-41. NCCN Guideline Insights. Bladder Cancer, Version 2.2022. J Natl Compr Canc Netw. 2022;20(8):866–878. View source version on businesswire.com: Contacts More information is available at the following: Health Care Providers: If you are interested in becoming part of the ADSTILADRIN Early Experience Program, please sign up for updates at . Patients and Consumers: For more information about ADSTILADRIN, please call 1-888-FERRING (888-337-7464), and select option number one. Media: Members of the press can contact Carol Ready, Director, Brand Communications by phone at (973) 765-7307, or email at carol.ready@ferring.com. Source: Ferring Pharmaceuticals View this news release online at: