WASHINGTON, Dec. 15, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for imsidolimab, a novel IgG4 IL-36 receptor antagonist, to treat generalized pustular psoriasis (GPP). Imsidolimab inhibits IL-36 receptor signaling, addressing the deficiency in the endogenous IL-36RA regulator commonly seen in GPP patients due to IL36RN gene mutations. GPP is a rare, chronic, life ...

Core Viewpoint - Hagens Berman is investigating Alvotech for potential misrepresentation regarding its lead drug candidate AVT05 and its manufacturing operations, following a significant revision to the company's financial forecasts for 2025 [1][2]. Financial Performance - Alvotech raised its 2025 revenue guidance to $600-$700 million and adjusted EBITDA to $200-$280 million on May 8, 2025, and reiterated these forecasts on August 14, 2025 [3]. - Following the FDA's issuance of a Complete Response Letter (CRL) on November 2, 2025, Alvotech revised its revenue forecast down to $570-$600 million and adjusted EBITDA to $130-$150 million, representing a 10% decrease in revenue and a 58% decrease in adjusted EBITDA from prior projections [3][4]. Market Reaction - The announcement of the CRL led to a 33% drop in Alvotech's share price on November 3, 2025, resulting in a loss of hundreds of millions of dollars in market value in a single day [4]. Investigation Focus - The investigation by Hagens Berman centers on whether Alvotech adequately disclosed the status of its Biologics License Application for AVT05 and the manufacturing practices at its Reykjavik facility, which were critical to the company's revenue projections [2][5].

Core Insights - Vera Therapeutics has submitted a Biologics License Application (BLA) to the FDA for atacicept, targeting immunoglobulin A nephropathy (IgAN) through the Accelerated Approval Program [1][3][7] - The BLA submission is supported by positive interim results from the ORIGIN 3 trial, showing a 46% reduction in proteinuria from baseline and a 42% reduction compared to placebo at week 36 [2][4][8] - Atacicept is positioned as a first-in-class dual BAFF/APRIL inhibitor, addressing a significant unmet medical need in IgAN, which can lead to end-stage kidney disease in over 50% of patients [3][9] Company Overview - Vera Therapeutics is a late clinical-stage biotechnology company focused on developing treatments for serious immunological diseases, with atacicept as its lead product candidate [11] - The company aims to change the standard of care for patients by targeting the source of diseases, particularly in autoimmune conditions like IgAN and lupus nephritis [11] - Vera Therapeutics retains global developmental and commercial rights to atacicept, along with other investigational therapies [11] Clinical Development - The ORIGIN 3 trial is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study involving 431 adults with IgAN, with a primary endpoint focused on proteinuria reduction [4][6] - Atacicept has demonstrated a favorable safety profile comparable to placebo across the ORIGIN program [2][8] - The ORIGIN Phase 2b trial also showed significant proteinuria reductions and stabilization of kidney function, reinforcing the efficacy of atacicept [6][9] Regulatory Status - Atacicept has received FDA Breakthrough Therapy Designation for IgAN, indicating its potential to offer substantial improvement over existing therapies [7][9] - The anticipated timeline for potential FDA approval of atacicept is in 2026 [7]

Financial Data and Key Metrics Changes - Revenue for Q3 2025 reached $60.1 million, reflecting a 23.5% increase compared to Q3 2024 and a 6% sequential increase from Q2 2025 [7][19] - Gross profit for the quarter was $46 million, up from $36.4 million in Q3 2024, resulting in a gross margin of 76.6%, an increase from 74.9% year-over-year [21][22] - Net income for the quarter was $0.7 million or $0.01 per share, compared to a net loss of $1.9 million or $0.04 per share in Q3 2024 [23] - Adjusted net income was $6.1 million or $0.12 per share, compared to $3.1 million or $0.07 per share in the same period last year [24] Business Line Data and Key Metrics Changes - Strong revenue growth was driven by the Avance Nerve Graft and the adoption of the nerve repair algorithm across target markets, with double-digit growth in extremities, oromaxillofacial, and breast markets [7][8] - Active high-potential accounts increased to 668, representing a 1.2% increase compared to the first three quarters of 2024 [11] - The company successfully ended its case stock sales program for Avance Nerve Graft, which positively impacted revenue by approximately $1.6 million in Q3 [20][10] Market Data and Key Metrics Changes - Coverage among commercial payers increased to over 64%, with an estimated 18.1 million additional lives covered for nerve repair using synthetic conduits or allografts year-to-date [16] - The company is actively engaging with national payers to transition from investigational to standard coverage [42] Company Strategy and Development Direction - The company remains focused on executing its strategic plan, optimizing resource allocation, and driving towards profitability [25] - Plans to continue expanding the sales force incrementally across all target markets to enhance nerve care adoption [83] - The anticipated approval of the Biologics License Application (BLA) for Avance Nerve Graft is expected to secure 12 years of market exclusivity and establish it as the only implantable biologic for peripheral nerve repair [18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the business's growth trajectory and the strategic plan, despite slight underperformance in high-potential accounts due to the transition from the case stock program [10][11] - The company is optimistic about the impact of the BLA approval on market development and coverage expansion, although it cautioned against expecting immediate results [94] Other Important Information - The FDA extended the PDUFA goal date for the BLA from September to December 5, 2025, due to a major amendment in the application [18] - The company is on track to meet its 2025 surgeon training targets, with significant progress in professional education programs [12][13] Q&A Session Summary Question: Insights on Q4 revenue expectations and case stock sales program impact - Management indicated that the $1.6 million revenue increase in Q3 was related to the transition from the case stock program and advised excluding it from Q4 models [30] Question: Comments on 2026 revenue growth expectations - Management refrained from providing specific guidance for 2026 but expressed confidence in the business's strategic plan [32] Question: Update on BLA and labeling expectations - Management confirmed that discussions regarding labeling are ongoing, with no definitive timeline for completion [38] Question: Coverage expansion trajectory and impact of BLA - Management noted that the BLA will support market development efforts but cautioned against expecting immediate widespread adoption [94] Question: Profitability across different market segments - Management stated that all segments are profitable and that progress in any segment contributes positively to the business [100]

Company Overview - Capricor Therapeutics (CAPR) shares increased by 5.3% to close at $6.52, with trading volume significantly higher than usual, contrasting with a 1% loss over the past four weeks [1][2]. Regulatory Update - The rise in CAPR shares is linked to a positive regulatory update regarding the Deramiocel program for Duchenne muscular dystrophy, following a Type A meeting with the FDA that provided guidance for a potential Biologics License Application [2]. Financial Expectations - The company is projected to report a quarterly loss of $0.54 per share, reflecting a year-over-year decline of 42.1%, while revenues are expected to reach $2.4 million, marking a 6.2% increase from the previous year [3]. Earnings Estimate Trends - The consensus EPS estimate for Capricor has remained stable over the last 30 days, indicating that stock price movements typically do not sustain upward trends without changes in earnings estimate revisions [4]. Industry Context - Capricor is part of the Zacks Medical - Products industry, where another company, biote Corp. (BTMD), experienced a 0.3% decline to $2.97, with a return of -11.3% over the past month [5].

Core Insights - Vera Therapeutics reported significant clinical results from the ORIGIN 3 trial for atacicept, showing potential as a first dual BAFF/APRIL inhibitor for IgA Nephropathy (IgAN) [2][6] - The company plans to submit a Biologics License Application (BLA) to the U.S. FDA for accelerated approval in Q4 2025, with a potential commercial launch in 2026 [2][6] Business Highlights - The ORIGIN 3 trial demonstrated a 46% reduction in proteinuria for atacicept-treated participants, achieving a statistically significant 42% reduction compared to placebo [7] - The safety profile of atacicept appears favorable and comparable to placebo, with full enrollment in the ORIGIN 3 trial completed [7] - Vera initiated the PIONEER trial to evaluate atacicept in a broader IgAN patient cohort and other autoimmune glomerular diseases [6][7] Financial Results - For Q2 2025, Vera reported a net loss of $76.5 million, compared to a net loss of $33.7 million in Q2 2024, with a net loss per diluted share of $1.20 [5] - Net cash used in operating activities for the first half of 2025 was $109.2 million, up from $58.6 million in the same period last year [5] - As of June 30, 2025, Vera had $556.8 million in cash, cash equivalents, and marketable securities, which is expected to fund operations through potential approval and commercial launch of atacicept [8] Anticipated Upcoming Milestones - BLA submission for atacicept expected in Q4 2025, with full primary endpoint results from the ORIGIN 3 trial to be presented at a medical congress in the same quarter [6][7] - Initial results from the PIONEER Phase 2 basket trial are also expected in Q4 2025, with the ORIGIN 3 study completion anticipated in 2027 [6][7]

Core Insights - Capricor Therapeutics, Inc. is under investigation for potential claims related to its common stock following a significant stock price drop after receiving a Complete Response Letter from the FDA regarding its lead product candidate, Deramiocel [1][2]. Company Developments - Capricor is a clinical-stage drug company focused on developing Deramiocel for treating cardiomyopathy in patients with Duchenne muscular dystrophy [1]. - The company completed its Phase 2 HOPE-2 clinical trial in 2021 [1]. - On September 24, 2024, Capricor filed a Biologics License Application (BLA) with the FDA for Deramiocel, which led to a stock price increase from $5.97 to $9.10 per share [2]. - On May 13, 2025, Capricor reported no significant deficiencies following a mid-cycle review meeting with the FDA [2]. - On July 11, 2025, Capricor announced it received a Complete Response Letter from the FDA, stating that the BLA did not meet the requirements for substantial evidence of effectiveness and required additional clinical data, resulting in a stock price drop of 33% to $7.64 per share [2]. Investor Information - Investors who experienced losses in Capricor's common stock are encouraged to contact Wolf Popper LLP for discussions regarding the investigation [3].

Core Viewpoint - Genmab A/S plans to submit a supplemental Biologics License Application (sBLA) for subcutaneous epcoritamab in combination with rituximab and lenalidomide for treating adult patients with relapsed or refractory follicular lymphoma in the first half of 2025 [1][3] Company Announcement - The sBLA submission is based on positive topline results from the Phase 3 EPCORE FL-1 trial, which showed that epcoritamab plus rituximab (R) met one of its dual primary endpoints of overall response rate (ORR) with a p-value of less than 0.0001 [2][7] - The safety profile of the combination therapy was consistent with known safety profiles of the individual components, and no new safety signals were observed [2][3] About Follicular Lymphoma (FL) - Follicular lymphoma is a slow-growing form of non-Hodgkin's lymphoma, accounting for 20-30% of all cases, with approximately 15,000 new cases diagnosed annually in the U.S. [4] - It is considered incurable with current standard therapies, and patients often experience shorter remission periods with each relapse [4] About the EPCORE FL-1 Trial - The EPCORE FL-1 trial is a Phase 3 open-label interventional study evaluating the safety and efficacy of epcoritamab plus rituximab and lenalidomide versus rituximab alone in patients with relapsed/refractory follicular lymphoma [5] About Epcoritamab - Epcoritamab is an IgG1-bispecific antibody developed using Genmab's DuoBody technology, designed to target CD3 on T cells and CD20 on B cells, inducing T-cell-mediated killing of CD20+ cells [6][7] - It is currently approved by the FDA as a monotherapy for adults with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy [3][7] Collaboration and Future Plans - Genmab is co-developing epcoritamab with AbbVie, sharing commercial responsibilities in the U.S. and Japan, and both companies are pursuing additional international regulatory approvals for the investigational indication [8][9] - Genmab and AbbVie are also evaluating epcoritamab in various hematologic malignancies across multiple ongoing Phase 3 trials [9]

Core Viewpoint - Regeneron Pharmaceuticals announced that the FDA issued a complete response letter (CRL) regarding the supplemental Biologics License Application (sBLA) for EYLEA HD, which proposed extended dosing intervals of up to every 24 weeks, but the FDA did not agree with this proposal [1][2]. Summary by Relevant Sections FDA Response - The CRL did not identify any safety or efficacy issues with EYLEA HD in its approved indications and dosing regimens [2]. - The FDA currently allows a maximum dosing interval of every 16 weeks for EYLEA HD, which Regeneron sought to extend [2]. Product Information - EYLEA HD is approved for dosing intervals ranging from every 8 to 16 weeks for wet age-related macular degeneration (wAMD) and diabetic macular edema (DME), and every 8 to 12 weeks for diabetic retinopathy (DR) after three initial monthly doses [3]. - EYLEA HD was developed to provide comparable efficacy and safety to EYLEA but with fewer injections [5]. - EYLEA HD is a joint development between Regeneron and Bayer AG, with Regeneron holding exclusive rights in the U.S. and Bayer having exclusive marketing rights outside the U.S. [6]. Company Overview - Regeneron is a leading biotechnology company focused on developing life-transforming medicines for serious diseases, with a strong emphasis on eye care and ongoing research in various serious eye diseases [12][7]. - The company utilizes proprietary technologies to accelerate drug development and aims to innovate new solutions for patients [13].