Core Insights - Amgen has received FDA approval for UPLIZNA (inebilizumab-cdon) as the first CD19-targeted B cell therapy for treating generalized myasthenia gravis (gMG) in adults who are positive for anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibodies, offering a new treatment option with the potential for long-term disease control through biannual dosing after initial loading doses [1][7][20] Group 1: Treatment Efficacy and Approval - The approval is based on the Myasthenia Gravis Inebilizumab Trial (MINT), which is the largest Phase 3 study for gMG, demonstrating significant efficacy in reducing symptoms and steroid dependency among patients [4][5] - At Week 26, UPLIZNA showed a 1.9-point improvement in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score compared to placebo, with a statistically significant p-value of <0.0001 [6][11] - The trial included 238 adults, with 87.4% of UPLIZNA patients reducing their steroid dose to 5 mg or less per day by Week 26, indicating a reduction in steroid dependency [4][14] Group 2: Patient Impact and Market Potential - UPLIZNA offers a convenient dosing schedule of twice a year, which is expected to improve patient adherence and quality of life by providing six months of treatment-free time between doses [2][7] - The prevalence of gMG in the U.S. is estimated to be between 80,000 and 100,000 patients, with a growing global incidence, highlighting a significant market opportunity for UPLIZNA [3][17] - This approval marks the third indication for UPLIZNA, previously approved for anti-AQP4 antibody positive neuromyelitis optica spectrum disorder (NMOSD) and Immunoglobulin G4-related disease (IgG4-RD), showcasing the drug's versatility [7][20] Group 3: Safety and Adverse Reactions - Common adverse reactions reported in gMG patients include headache and infusion-related reactions, with a noted incidence of 10.1% during the clinical trials [8][30] - Amgen emphasizes its commitment to patient support and access to UPLIZNA, indicating a proactive approach to managing treatment-related challenges [8][39]

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Summary of Cardiff Oncology Conference Call Company Overview - **Company**: Cardiff Oncology (NasdaqCM: CRDF) - **Focus**: Development of onvansertib, a first-in-class PLK1 inhibitor targeting RAS-mutated metastatic colorectal cancer [2][35] Key Points and Arguments Product and Mechanism - **Onvansertib**: A highly selective PLK1 inhibitor designed for first-line treatment of RAS-mutated metastatic colorectal cancer [2][4] - **Mechanism**: PLK1 is an enzyme that drives tumor cell division and survival; onvansertib inhibits this enzyme, leading to reduced tumor growth and metastasis [3][4] Clinical Trials and Efficacy - **Clinical Trials**: Initial trials showed strong efficacy signals in RAS-mutated metastatic colorectal cancer, particularly in BEV-naive patients [3][11] - **Response Rates**: In a second-line trial, onvansertib combined with standard care showed a 73% response rate in BEV-naive patients compared to 16% in BEV-exposed patients [11] - **Progression-Free Survival (PFS)**: Nearly doubled in BEV-naive patients compared to historical controls [11] Market Opportunity - **Unmet Need**: Colorectal cancer has a low five-year survival rate of only 15%, with no new therapies introduced in over 20 years for RAS-mutated patients [5][6] - **Target Population**: Approximately 50% of newly diagnosed colorectal cancer patients have RAS mutations, indicating a significant market opportunity [6][35] Regulatory and Development Path - **FDA Alignment**: Received clear guidance from the FDA to pivot from second-line to first-line clinical development, addressing a larger patient population [4][18] - **Ongoing Trials**: The ongoing first-line trial is designed to assess the efficacy of onvansertib in combination with standard chemotherapy [19][20] Safety and Tolerability - **Safety Profile**: Onvansertib is well tolerated, with no significant increase in neutropenia compared to control groups [30][31] - **Combination Therapy**: The drug can be combined with existing chemotherapies without compromising safety, allowing for longer treatment durations [40][41] Financial and Strategic Partnerships - **Partnership with Pfizer**: Pfizer holds a 3% equity stake in Cardiff Oncology and is involved in trial execution through its Pfizer Ignite program, providing high-quality data without strings attached [33][34] Future Outlook - **Next Steps**: Plans to update on trial progress in Q1 of the following year, with a focus on durability data and potential registrational trials [36] - **Investment Opportunity**: The company is positioned to make a significant impact in the treatment of colorectal cancer, presenting a compelling investment opportunity [42][43] Additional Important Information - **Patents**: Two new patents were issued, extending the intellectual property protection for onvansertib until at least 2043 [17] - **Clinical Data Publication**: Findings have been published in the Journal of Clinical Oncology, highlighting the significance of the research [16] This summary encapsulates the critical insights from the conference call, emphasizing Cardiff Oncology's innovative approach to treating colorectal cancer and the potential for significant market impact.

Even in this difficult environment, our business fundamentals are solid and the key positive drivers of performance that we've seen recently carried through into Q2. We continue to see solid signs of underlying single-cell demand. On the consumables side, while revenue was down year-over-year, Chromium reaction volumes grew both year-over-year and sequentially, an indicator of increasing demand for our solutions and single cell more broadly. This growth was driven by robust adoption of our latest products, ...

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WALTHAM, Mass., Dec. 11, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced the closing of its previously announced upsized underwritten public offering of 21,827,549 shares of its common stock at a public offering price of $18.44 per share, which includes 2,847,071 shares issued upon the exercise in full by the underwriters of their option to p ...

WATERTOWN, Mass., Dec. 11, 2025 (GLOBE NEWSWIRE) -- Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing a new class of oral small molecule degrader medicines for immunological diseases, today announced the closing of its upsized underwritten public offering of $602.0 million of shares of its common stock. Kymera sold and issued 8,050,000 shares of its common stock, which includes 1,050,000 shares sold and issued upon the full exercise by the underwriters of their o ...

NEWARK, N.J., Dec. 11, 2025 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE: RFL) today reported its financial results for the first quarter fiscal year 2026 ended October 31, 2025. “We remain pleased with the progress of our pivotal Phase 3 TransportNPC™ study evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1, which the Data Monitoring Committee (DMC) recommended continuing after their review of prespecified safety and efficacy data at 48 weeks. We believe that Trappsol® Cyclo™ ...

MENLO PARK, Calif., Dec. 11, 2025 (GLOBE NEWSWIRE) -- Oruka Therapeutics, Inc. (“Oruka”) (Nasdaq: ORKA), a clinical-stage biotechnology company developing novel biologics designed to set a new standard for the treatment of chronic skin diseases including plaque psoriasis (PsO), today announced the appointment of Chris Martin to its Board of Directors. Additionally, Cameron Turtle, DPhil, resigned from the Board effective December 11, 2025. “As we rapidly advance our co-lead programs through development, I’m ...

SOUTH SAN FRANCISCO, Calif., Dec. 11, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced it has received official notification from the U.S. Food and Drug Administration (FDA) that the clinical hold on the MyPEAK-1™ Phase 1b/2a clinical trial of TN-201 has been removed. All concerns raised by the FDA related ...