Nogapendekin alfa inbakicept
- Generic Name
- Nogapendekin alfa inbakicept
FDA Rejects ImmunityBio's Expanded Anktiva Application While Lifting Hold on Atara's Ebvallo Trials
• ImmunityBio received an FDA "refusal to file" letter for its application to expand Anktiva's use in bladder cancer patients, causing a 25% stock plunge and prompting calls for agency clarification.
• The rejection comes despite Anktiva's approval last year for a related subset of bladder cancer patients, with ImmunityBio's founder Patrick Soon-Shiong citing "confounding inconsistency" in the FDA's decision-making process.
• In contrast, Atara Biotherapeutics received positive news as the FDA lifted its clinical hold on blood cancer drug Ebvallo and scheduled discussions for resubmission, driving a 6% stock increase.
ImmunityBio Secures $75 Million in Equity Financing to Advance Immunotherapy Pipeline
• ImmunityBio has executed a $75 million registered direct offering with a single institutional investor, providing crucial working capital for its ongoing operations.
• The financing includes warrants that could generate an additional $90 million in gross proceeds if fully exercised, potentially bringing total funding to $165 million.
• The company plans to use the capital to support its immunotherapy platforms, including further development of ANKTIVA, the FDA-approved therapy for non-muscle invasive bladder cancer.
Novel BCMA-Directed CAR T-Cell Therapy Shows Promising Efficacy in Relapsed/Refractory Multiple Myeloma and AL Amyloidosis
• A second-generation BCMA-directed CAR T-cell therapy, MDC-CAR-BCMA001, demonstrated remarkable efficacy in patients with relapsed/refractory multiple myeloma and AL amyloidosis, achieving a 5/6 overall response rate with 4 complete responses.
• The novel therapy showed a favorable safety profile with manageable toxicity, including low incidence of severe cytokine release syndrome and no neurotoxicity, even in patients with significant organ dysfunction.
• These promising results have prompted further investigation through ongoing clinical trials, including CARLOTTA001 (NCT05836896) and the CLEAR AL trial, potentially offering new hope for difficult-to-treat hematologic malignancies.
ImmunityBio Achieves Multiple Milestones in Q1 2024, Advances ANKTIVA Development with RMAT Designation
• ImmunityBio reports significant progress with ANKTIVA's approval and permanent J-code assignment for treating BCG-unresponsive bladder cancer CIS, marking a major advancement in treatment options.
• The company receives RMAT designation from FDA for ANKTIVA + PD-L1 t-haNK combination therapy for lymphopenia reversal, potentially transforming immunotherapy treatment approaches.
• Strategic developments include EMA and MHRA submission acceptance, BeOne collaboration for checkpoint inhibitor supply, and expanded access to recombinant BCG amid TICE BCG shortage.
Verastem Oncology to Present New LGSOC Treatment Data at SGO 2025 Annual Meeting
• Verastem Oncology will present additional analyses from the Phase 2 RAMP 201 trial evaluating avutometinib plus defactinib in recurrent low-grade serous ovarian cancer at SGO 2025, building on their FDA Priority Review submission.
• The presentations include new subgroup analyses by KRAS mutational status, supporting the company's ongoing efforts to advance treatment options for RAS/MAPK pathway-driven cancers.
• The company's NDA for the avutometinib-defactinib combination therapy has received Priority Review from the FDA with a PDUFA date of June 30, 2025, for KRAS mutant LGSOC patients.
FDA Grants ImmunityBio Authorization to Supply Recombinant BCG Amid TICE BCG Shortage
• ImmunityBio receives FDA authorization to provide recombinant BCG (rBCG) to urologists, addressing the critical TICE BCG shortage for bladder cancer treatment.
• Phase 1/2 clinical studies demonstrate rBCG's favorable safety profile compared to earlier BCG strains, showing tolerability similar to placebo when administered intravesically.
• The modified rBCG features two genetic enhancements designed to improve immunogenicity and safety, supported by data from multiple clinical trials including studies in bladder cancer patients.
BCG Shortage Continues to Shape Bladder Cancer Treatment Landscape Despite New Therapy Approvals
• The recent FDA approval of nogapendekin alfa inbakicept plus BCG for BCG-unresponsive bladder cancer faces implementation challenges due to ongoing BCG supply shortages across U.S. healthcare facilities.
• Healthcare providers are adapting treatment strategies through split-dose BCG regimens and exploring alternative therapies, including nadofaragene firadenovec which offers convenient quarterly dosing schedules.
• Novel diagnostic tools, including AI-powered pathology analysis and urinary cell-free DNA testing, are emerging as promising approaches for improving treatment selection and monitoring in bladder cancer.
ImmunityBio and BeiGene Collaborate on Phase 3 Trial of ANKTIVA and Tislelizumab in NSCLC
• ImmunityBio and BeiGene are collaborating on a Phase 3 trial (ResQ201A-NSCLC) to evaluate ANKTIVA plus BeiGene's tislelizumab in advanced NSCLC patients who have progressed on CPI therapy.
• The trial aims to confirm the efficacy and safety of the ANKTIVA and CPI combination, building on Phase 2 results showing prolonged overall survival in CPI-resistant NSCLC patients.
• The global Phase 3 trial will enroll 462 participants, with overall survival as the primary endpoint and secondary endpoints including disease control rate and safety.
• ImmunityBio plans to submit a BLA in 2025 for this indication based on the QUILT 3.055 data, with the goal of addressing unmet needs in second- and third-line NSCLC treatment.
ImmunityBio's Anktiva Pursues European and UK Approval for BCG-Unresponsive Bladder Cancer
• ImmunityBio's Anktiva, combined with BCG, seeks approval in the EU and UK for treating BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS).
• The MAAs submitted to the EMA and MHRA are supported by Phase 2/3 QUILT-3.032 trial data, demonstrating a 71% complete response rate in patients with NMIBC with CIS.
• Regulatory assessments by both agencies are anticipated to conclude by Q4 2025, potentially leading to approval in Europe and the UK by 2026, expanding global access.
• ImmunityBio also plans to submit a supplemental BLA to the FDA in 2025, seeking approval for Anktiva in patients with BCG-unresponsive NMIBC in the papillary indication.
J&J Seeks FDA Approval for TAR-200 in BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
• Johnson & Johnson has initiated a New Drug Application (NDA) submission to the FDA for TAR-200.
• TAR-200 is intended for patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC).
• Phase 2b SunRISe-1 study data showed an 83.5% complete response rate with TAR-200 monotherapy.
• The FDA is reviewing the application under the Real-Time Oncology Review (RTOR) program to expedite potential approval.
FDA Approves ImmunityBio's Bladder Cancer Drug After Second Review
The FDA has approved ImmunityBio's Anktiva, a treatment for non-muscle invasive bladder cancer (NMIBC) that did not respond to BCG vaccine alone, marking the company's first commercial-stage product. This decision comes after initial rejection due to manufacturing quality issues.
ANKTIVA Receives Permanent J-Code from CMS, Streamlining Reimbursement for Bladder Cancer Treatment
• The Centers for Medicare & Medicaid Services (CMS) has assigned a permanent J-code (J9028) for ANKTIVA, effective January 1, 2025, to streamline billing.
• ANKTIVA, in combination with BCG, is FDA-approved for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS).
• Clinical trial data shows ANKTIVA achieved a 71% complete response rate in patients with BCG-unresponsive NMIBC CIS, with durable responses up to 54 months.
FDA Approves Subcutaneous Opdivo Qvantig for Multiple Solid Tumors
• The FDA approved Opdivo Qvantig, a subcutaneous injection of nivolumab, for adult solid tumors previously approved for intravenous Opdivo.
• CheckMate-67T trial data showed non-inferior pharmacokinetic exposures, similar efficacy, and comparable safety to intravenous Opdivo.
• Opdivo Qvantig offers a faster 3-5 minute administration time, providing convenience and flexibility for patients.
• The approval helps Bristol Myers Squibb maintain market share amid patent expirations and increasing biosimilar competition.
Camrelizumab and Apatinib Show Promise in Neoadjuvant Treatment of TNBC
• Camrelizumab plus chemotherapy significantly improved pathologic complete response (pCR) rates in early or locally advanced triple-negative breast cancer (TNBC).
• Apatinib combined with sintilimab and chemotherapy demonstrated a high pCR rate of 70.6% in early TNBC, suggesting synergistic effects.
• Both camrelizumab and apatinib regimens exhibited manageable safety profiles, supporting their potential as new neoadjuvant therapeutic options.
• Biomarker analysis in the apatinib study identified correlations between immune response and pCR, offering insights for predicting treatment efficacy.
Durvalumab Gains Priority Review for Muscle-Invasive Bladder Cancer Treatment
• The FDA has granted priority review to durvalumab (Imfinzi) for muscle-invasive bladder cancer (MIBC), potentially expediting its approval.
• Phase 3 NIAGARA trial data supports the application, showing a 32% reduction in disease progression or recurrence with perioperative durvalumab.
• The Imfinzi regimen also demonstrated a 25% reduction in the risk of death, offering a significant survival benefit.
• Regulatory decisions are anticipated in the second quarter of 2025, with potential implications for MIBC treatment standards.
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.
ImmunityBio's Anktiva Receives EMA Review for Bladder Cancer Treatment
• The European Medicines Agency (EMA) has accepted ImmunityBio's marketing authorization application for ANKTIVA in combination with BCG for BCG-unresponsive NMIBC.
• The application is supported by the QUILT 3.032 study, which demonstrated a 71% complete response rate in patients treated with ANKTIVA and BCG.
• Durable responses in the study extended up to 54 months, exceeding the benchmark for clinically meaningful results in this indication.
• ANKTIVA, an IL-15 agonist, has already been approved by the FDA for use in the United States for the same indication.
Novel Agents Show Promise in BCG-Unresponsive NMIBC Treatment
• TAR-200 monotherapy demonstrated an 83.5% complete response rate in the SunRISe-1 trial, with an estimated 12-month duration of response rate of 65.7%.
• Pembrolizumab monotherapy in the KEYNOTE-057 trial achieved a 41% complete response rate at 3 months in high-risk NMIBC patients without carcinoma in situ.
• Nadofaragene firadenovec-vncg showed a 53.4% complete response rate at 3 months and 45.5% at 1 year in a phase 3 trial for BCG-unresponsive NMIBC.
• Nogapendekin alfa inbakicept-pmln plus BCG achieved a 71% complete response rate at any time, with a median follow-up of 23.9 months in the QUILT-3.032 trial.
Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025
• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD.
• EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration.
• Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option.
• Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.
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