Ferring Pharmaceuticals, Inc.
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Subsidiary
- Employees
- -
- Market Cap
- -
- Website
- http://www.ferringusa.com
Phase 3b Trial Shows Colonoscopic Administration of Rebyota Effective for Preventing Recurrent C. difficile Infection
• A phase 3b clinical trial demonstrated that Rebyota (fecal microbiota, live-jslm) administered via colonoscopy achieved a 95.1% treatment success rate in preventing recurrent C. difficile infection.
• Only 9.8% of participants experienced Rebyota-related adverse events, all of which were mild and gastrointestinal in nature, confirming the safety profile of this administration method.
• Patients reported significant quality of life improvements after treatment, with 89.7% experiencing symptom relief within the first month and substantial reductions in diarrhea, abdominal pain, and fatigue.
FDA Approves Ferring Pharmaceuticals' New Manufacturing Facility for Bladder Cancer Gene Therapy ADSTILADRIN
• The FDA has approved Ferring Pharmaceuticals' new drug product manufacturing hub in Parsippany, NJ, for production of ADSTILADRIN, the first FDA-approved intravesical gene therapy for high-risk BCG-unresponsive bladder cancer.
• The approval expands Ferring's manufacturing capabilities to three sites dedicated to ADSTILADRIN production, triggering a final $200 million payment from Royalty Pharma as part of a financing agreement announced in 2023.
• The 12,000 square foot state-of-the-art facility features renewable energy solutions and specialized technology to ensure stable supply for growing global demand, reinforcing Ferring's commitment to uro-oncology treatment innovation.
Nadofaragene Firadenovec Shows 75% Complete Response Rate in Japanese Phase 3 Trial for BCG-Unresponsive Bladder Cancer
• Nadofaragene firadenovec demonstrated a 75% complete response rate at 3 months in Japanese patients with high-risk BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, according to new Phase 3 trial data.
• The gene therapy showed a favorable safety profile with 84.2% of treatment-related adverse events being Grade 1 and 15.8% Grade 2, with no Grade 3 or higher events reported in the Japanese patient population.
• These results appear higher than previously reported in US trials and align with recent Mayo Clinic real-world data showing a 79% complete response rate, potentially establishing nadofaragene as a new standard of care for patients who would otherwise face bladder removal.
DelveInsight Report: Over 50 Pipeline Drugs in Development for Urothelial Carcinoma Treatment
• DelveInsight's latest pipeline analysis reveals 40+ pharmaceutical companies actively developing 50+ therapeutic candidates for urothelial carcinoma treatment, indicating robust research activity in this space.
• Major pharmaceutical companies including AstraZeneca, Seagen, and Janssen are advancing promising therapies, with notable candidates like disitamab vedotin and cetrelimab in late-stage development.
• Recent clinical trials include Seagen's Phase 2 study of disitamab vedotin with pembrolizumab, and Roche's Phase 1b/II umbrella study investigating multiple combination therapies for advanced urothelial carcinoma.
Endometriosis Clinical Trial Pipeline Shows Promise with 20+ Therapies in Development
• The endometriosis clinical trial landscape is robust, featuring over 15 companies developing more than 20 potential therapies.
• Recent clinical trial activity includes positive Phase II results for Hope Medicine's HMI-115 and TiumBio's Merigolix, alongside Lisata Therapeutics' preclinical research.
• Key companies like Kissei Pharmaceutical, Mithra Pharmaceuticals, and Organon are advancing novel treatments, including Linzagolix and Estelle, to address unmet needs.
• These emerging therapies aim to improve pain management and overall outcomes for the estimated 190 million women affected by endometriosis worldwide.
Advancements in Clostridioides difficile Treatment and Prevention Highlighted in 2024
• Real-world data presented at ID Week 2024 confirms the continued efficacy and safety of Rebyota, a live biotherapeutic product, for recurrent Clostridioides difficile infection (rCDI) two years post-approval.
• Research indicates reduced vancomycin susceptibility in Clostridioides difficile isolates correlates with diminished sustained clinical response, emphasizing the need for alternative treatment strategies.
• Vedanta Biosciences has commenced a Phase 3 clinical trial (RESTORATiVE303) evaluating VE303, a live biotherapeutic product, for preventing recurrent Clostridioides difficile infection (rCDI).
• A study showed that oral vancomycin prophylaxis significantly reduces Clostridioides difficile infection incidence during hospitalization for autologous stem cell transplant recipients.
Real-World Data and Ongoing Trials Highlight Efficacy of Nadofaragene Firadenovec in Bladder Cancer
• Real-world data presented at the 2025 ASCO Genitourinary Cancers Symposium indicates nadofaragene firadenovec demonstrates positive efficacy and safety outcomes in treating NMIBC.
• The ABLE-32 phase 3B trial is underway to assess nadofaragene firadenovec in intermediate-risk NMIBC patients, addressing the unmet need for FDA-approved treatments.
• Additional trials, including ABLE-22 and LUNAR, are exploring nadofaragene firadenovec as a monotherapy, in combination with other treatments, and in low-grade upper tract urothelial carcinoma.
• Nadofaragene firadenovec, the first FDA-approved intravesical gene therapy, offers a novel approach by turning bladder wall cells into interferon microfactories to fight cancer.
Ferring Advances Nadofaragene Firadenovec Clinical Program in Urothelial Cancers
• Ferring Pharmaceuticals is expanding the clinical trial program for nadofaragene firadenovec, an intravesical gene therapy, to include intermediate-risk NMIBC and low-grade UTUC.
• A Phase 2 trial (ABLE-22) will evaluate nadofaragene firadenovec alone or with chemotherapy/immunotherapy in high-risk BCG-unresponsive NMIBC, including re-induction for non-responders.
• The Phase 3B ABLE-32 trial will assess nadofaragene firadenovec in intermediate-risk NMIBC, an area with no FDA-approved treatments, using recurrence-free survival as the primary endpoint.
• The LUNAR trial is initiated to study nadofaragene firadenovec in patients with low-grade upper tract urothelial cancer (UTUC), addressing a significant unmet need.
Advancements in Clinical Trials for Fabry Disease, C. difficile Infections, and Mydriasis
• Clinical trials are actively progressing for Fabry Disease, with companies like Idorsia and Sanofi Genzyme developing novel therapies.
• Emerging treatments like Venglustat and Pegunigalsidase alfa are expected to significantly impact the Fabry Disease market in the coming years.
• Research and development efforts are also focused on Clostridium Difficile Infections, with Vedanta Biosciences leading Phase 3 trials for VE303.
• Mydriasis treatments are advancing, with companies like Eyenovia exploring solutions like MYDCOMBI for pupil dilation.
REBYOTA Shows Sustained Efficacy in Preventing C. difficile Infection Recurrence
• REBYOTA, a live biotherapeutic product, continues to demonstrate efficacy in preventing recurrent Clostridioides difficile infection (rCDI) two years post-FDA approval.
• Real-world data from a study of 64 adults showed that 75% of patients treated with REBYOTA experienced no CDI recurrence after eight weeks.
• An open-label study with 697 patients, expanding on the PUNCH CD3 trial, found a 73.8% efficacy rate with 91% of responders remaining responsive at six months.
• REBYOTA, administered rectally, delivers a diverse microbiota to restore gut health and prevent CDI recurrence following antibiotic treatment.
Seltorexant Shows Promise as First-in-Class Treatment for Depression with Insomnia in Phase III Trial
• Phase III trial demonstrates seltorexant's efficacy as an add-on therapy, showing significant improvement in depression scores with a -2.6 point difference on the MADRS scale compared to placebo.
• As the first selective orexin-2 receptor antagonist for depression, seltorexant significantly improved both depression and sleep disturbance symptoms in patients with inadequate response to conventional antidepressants.
• The drug demonstrated a favorable safety profile with fewer adverse events than placebo (36% vs 40.3%), positioning it as a potential first-in-class treatment for concurrent depression and insomnia.
Condoliase (SI-6603) Shows Promise in Phase 3 Trials for Lumbar Disc Herniation
• SI-6603, an investigational treatment, significantly improved leg pain compared to sham in U.S. and placebo in Japan at Week 13 in Phase 3 trials.
• The studies involved intradiscal injections of SI-6603 and demonstrated statistically significant changes from baseline in worst leg pain.
• Common adverse events included spinal MRI abnormalities and back pain, with no treatment-related serious adverse events reported in the U.S. study.
• These findings support SI-6603's potential as a therapeutic option for radicular leg pain associated with lumbar disc herniation.
Vironexis' VNX-101 Cleared for Phase 1/2 Trial in CD19+ Acute Lymphoblastic Leukemia
• Vironexis Biotherapeutics received FDA clearance for its IND application for VNX-101, an AAV-based gene therapy, enabling a Phase 1/2 trial for CD19+ acute lymphoblastic leukemia.
• VNX-101 uses the TransJoin platform to express a bispecific T-cell engager, targeting CD19 on tumor cells and CD3 on T-cells to facilitate cancer cell killing.
• The Phase 1/2 trial is anticipated to begin in Q4 2024, marking the first clinical trial of an AAV-delivered cancer immunotherapy, with Fast Track and Rare Pediatric Disease designations.
• Vironexis' TransJoin technology aims to improve safety, efficacy, and durability compared to CAR-T therapy and bispecific antibodies, while streamlining manufacturing and reducing treatment burden.
Phase 3 Trial Shows Low-Dose Carbetocin Effective for Hyperphagia in Prader-Willi Syndrome
• Levo Therapeutics' Phase 3 CARE-PWS trial found that the lower 3.2mg dose of intranasal carbetocin (LV-101) significantly reduced hyperphagia and anxiety symptoms in patients with Prader-Willi syndrome, while the higher 9.6mg dose did not meet primary endpoints.
• The oxytocin-receptor agonist was generally well-tolerated, with mild to moderate side effects including headache, flushing, and diarrhea at the effective dose, offering potential relief for a condition with no currently approved treatments for its most challenging symptoms.
• Over 98% of trial participants elected to continue receiving the treatment in the long-term follow-up phase, with maintained benefits or further improvements observed, demonstrating sustained clinical value for this rare neurodevelopmental disorder affecting approximately 1 in 16,000 births.
© Copyright 2025. All Rights Reserved by MedPath