Latest news with #BiologicsLicenseApplication
Yahoo
25-06-2025
- Business
- Yahoo
Capricor's deramiocel moves ahead without AdCom, says H.C. Wainwright
Capricor on Tuesday disclosed that the FDA has indicated that no AdCom is necessary for deramiocel's Biologics License Application, and an in-person late-cycle review meeting is scheduled for mid-July, H.C. Wainwright analyst Joseph Pantginis tells investors in a research note. The firm, which has a Buy rating and $77 price target on the shares, highlights that even in bear case scenarios with the FDA, Phase 3 HOPE-3 data readouts are 'waiting in the wings' to further strengthen deramiocel's overall clinical and regulatory package and thinks deramiocel's first-in-class therapeutic profile in tandem with strong manufacturing support will get the therapy over the finish line on expected timelines. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See Insiders' Hot Stocks on TipRanks >> Read More on CAPR: Disclaimer & DisclosureReport an Issue HHS spokesperson tells Reuters FDA 'actively re-evaluating' Capricor AdCom Capricor Therapeutics provides regulatory update on Deramiocel BLA Roth says 'excessive' Capricor selloff due to 'investor overreaction' Capricor selloff brings buying opportunity, says H.C. Wainwright Capricor Therapeutics price target lowered to $22 from $43 at Oppenheimer
Yahoo
24-06-2025
- Business
- Yahoo
Carisma Therapeutics, OrthoCellix enter definitive merger agreement
Carisma Therapeutics (CARM) and OrthoCellix, a wholly-owned subsidiary of Ocugen (OCGN), announced that they have entered into a definitive merger agreement to combine the companies in an all-stock transaction. The combined company will focus on the development of OrthoCellix's NeoCart technology for the treatment of knee articular cartilage defects and plans to initiate a U.S. Food and Drug Administration-endorsed Phase 3 clinical trial for NeoCart. OrthoCellix is developing NeoCart as an autologous cartilage implant technology utilizing patient cells to repair articular cartilage defects of the knee. The novel platform merges a fortified 3D scaffold and patented bioprocessing technology to grow chondrocytes-the cells responsible for maintaining cartilage health-to produce adolescent-like cartilage at the time of implant. NeoCart has the potential to accelerate healing and reduce pain by creating a similar, functional joint surface to help patients return to normal activities and prevent complications associated with articular cartilage damage. OrthoCellix anticipates launching its Phase 3 clinical trial by the end of 2025. Previously, NeoCart received Regenerative Medicine Advanced Therapy designation and concurrence from the FDA on a single, confirmatory Phase 3 clinical trial to enable submission of a Biologics License Application. Under the terms of the merger agreement, OrthoCellix will merge with and into a wholly-owned subsidiary of Carisma, with OrthoCellix continuing as a wholly-owned subsidiary of Carisma and the surviving company of the Merger. Carisma will issue to the pre-merger OrthoCellix stockholder shares of Carisma common stock as merger consideration in exchange for the cancellation of shares of capital stock of OrthoCellix. Carisma also expects to enter into subscription agreements for a private financing with Ocugen and other select investors, which is expected to close concurrently with the completion of the merger, to enable the combined company to complete the Phase 3 trial of NeoCart without any additional cost or investment from Ocugen. In connection with the closing of the proposed transactions, Carisma stockholders will be issued contingent value rights representing the right to receive certain payments from proceeds received by the combined company, if any, related to Carisma's pre-transaction legacy assets. Under the terms of the merger agreement, upon the closing of the proposed transactions and after giving effect to the contemplated $25M concurrent financing, OrthoCellix's stockholder and the other participants in the concurrent financing are expected to own approximately 90% of the combined company, and existing Carisma stockholders are expected to own approximately 10% of the combined company, each on a fully diluted basis. The percentage of the combined company that each company's former stockholders will own after completion of the merger is subject to adjustment based on Carisma's net cash at the closing and the proceeds from the concurrent financing, among other adjustments, in each case as described in the merger agreement. Upon the closing of the proposed transactions, 'Carisma Therapeutics Inc.' is expected to be renamed 'OrthoCellix, Inc.' and trade on the Nasdaq Capital Market under the ticker symbol 'OCLX.' The transaction has been unanimously approved by the board of directors of both companies and is expected to close in the second half of 2025, subject to customary closing conditions, including approvals by the stockholders of each company and the effectiveness of a registration statement to be filed with the Securities and Exchange Commission to register the shares of Carisma common stock to be issued in connection with the merger. In connection with the companies' entry into the merger agreement, directors and officers of Carisma and OrthoCellix's stockholder have executed support agreements, pursuant to which they have agreed to vote all of their shares of capital stock in favor of the merger or the issuance of Carisma equity in the merger, as applicable. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Disclaimer & DisclosureReport an Issue Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
24-06-2025
- Business
- Yahoo
Sangamo Therapeutics Announces Positive Topline Results From Registrational STAAR Study in Fabry Disease
STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which U.S. Food and Drug Administration (FDA) has agreed will serve as primary basis of approval Isaralgagene civaparvovec showed a favorable safety and tolerability profile Sangamo intends to submit a Biologics License Application (BLA) in 2026 RICHMOND, Calif., June 24, 2025--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced positive topline results from the registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease. Following a single dose of isaralgagene civoparvovec, a positive mean annualized eGFR slope of 1.965 mL/min/1.73m2/year (95% confidence interval (CI): -0.153, 4.083) at 52-weeks was observed across all 32 dosed patients in the study, which the FDA has agreed will serve as an intermediate clinical endpoint under the Accelerated Approval pathway. Furthermore, a mean annualized eGFR slope of 1.747 mL/min/1.73m2/year (95% CI: -0.106, 3.601) was observed for the 19 patients who have achieved 104-weeks of follow-up. As recommended by the FDA, Sangamo plans to compare the annualized mean eGFR slope of isaralgagene civaparvovec with approved treatments for Fabry disease by performing a meta-analysis of published studies. According to observational studies, estimated mean annualized eGFR slopes for other marketed treatment options range from -2.2 to -0.4 mL/min/1.73m2/year for treatments such as Replagal (agalsidase alfa)1, Fabrazyme (agalsidase beta)2 and Galafold (migalastat)3. We believe these data support the potential for isaralgagene civaparvovec as a one-time, durable treatment for Fabry disease that can improve patient outcomes and will form the basis for an anticipated BLA submission under the Accelerated Approval pathway as early as the first quarter of 2026. The STAAR study enrolled male and female patients who were either on enzyme replacement therapy (ERT), were ERT pseudo-naïve (defined as having been off ERT for six or more months), or were ERT-naïve. The median age of patients enrolled in the study was 42, with a median duration of follow-up of 24 months and the longest treated patient having achieved 4.5 years of follow-up. Key secondary endpoints in the study were also positive. Elevated expression of alpha-galactosidase A (α-Gal A) activity was maintained for up to 4.5 years for the longest treated patient. All 18 patients who began the study on ERT have been withdrawn from ERT and all remain off ERT as of today. Plasma lyso-Gb3 levels in these patients remained generally stable following ERT withdrawal. A stabilization in cardiac endpoints was also observed. Patients demonstrated a range of other clinical benefits, including improvements in disease severity reported in the Fabry Outcome Survey adaptation of the Mainz Severity Score Index (FOS-MSSI) age-adjusted score and statistically and clinically significant improvements in the short form-36 (SF-36) quality of life scores, including role-physical +14.8 (95% CI: 7.3, 22.4, p=0.0003), vitality +9.6 (95% CI: 3.9, 15.2, p=0.0017), bodily pain +9.0 (95% CI: 2.3, 15.7, p=0.0104), social functioning +7.8 (95% CI: 2.0, 13.6, p=0.0100), general health +7.4 (95% CI: 2.0, 12.8, p=0.0091), and physical component scores +4.2 (95% CI: 1.8, 6.6, p=0.0014), at week 52 compared to baseline. Statistically significant improvements in the gastrointestinal symptoms rating scale (GSRS) compared to baseline were also observed. Furthermore, following a single administration of isaralgagene civaparvovec, additional clinical benefits were observed in some patients, such as the reduction or elimination in pain medication usage and the resumption of sweating, that has enabled these patients to perform physical tasks and exercise. Isaralgagene civaparvovec demonstrated a favorable safety and tolerability profile in the study, without the requirement for preconditioning. The majority of adverse events were grade 1-2 in nature. The most common treatment-emergent adverse events (TEAEs) were pyrexia (60.6% of participants), COVID-19 (36.4%), headache (33.3%) and nasopharyngitis (33.3%). All TEAEs resolved in response to clinical management and there were no safety-related study discontinuations. "Fabry disease is a debilitating and multifaceted condition, for which there is a serious unmet medical need," said Nathalie Dubois-Stringfellow, Ph. D, Chief Development Officer at Sangamo. "We are thrilled to see these compelling topline STAAR study results, including the positive mean annualized eGFR slope at both 52 and 104 weeks, alongside notable improvements in a range of secondary endpoints. Taken together these data demonstrate the potential for a single dose of ST-920 to provide meaningful clinical benefits above current standards of care and to treat the underlying pathology of Fabry disease. We want to thank the patients and investigators who participated in this study and look forward to sharing these data with health authorities." Isaralgagene civaparvovec has been granted Orphan Drug, Fast Track and RMAT designations from the FDA, Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from U.K. Medicines and Healthcare products Regulatory Agency. Analyses of the full dataset from the STAAR study are ongoing and additional data will be presented at an upcoming medical meeting. Sangamo is advancing BLA preparation activities for isaralgagene civaparvovec, while continuing to engage in business development negotiations for a potential Fabry commercialization agreement. About the STAAR Study The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec, or ST-920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning. About Fabry Disease Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. Symptoms of Fabry disease can include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain and tingling in the extremities. About Sangamo Therapeutics Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo believes that its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders and that its capsid discovery platform can expand delivery beyond currently available intrathecal delivery capsids, including the central nervous system. Sangamo's pipeline also includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit and connect with us on LinkedIn and X. 1 Replagal (agalsidase alfa) estimated mean annualized eGFR slope: -2.2 mL/min/1.73m2/year (95% CI: -2.8, -1.7) in male patients and -0.7 mL/min/1.73m2/year (95% CI: -1.4, 0) in female patients (Source: Feriozzi, 2012: The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy)2 Fabrazyme (agalsidase beta) estimated mean annualized eGFR slope: -1.5 mL/min/1.73m2/year (Source: Fabrazyme Package Insert: Galafold (migalastat) estimated mean annualized eGFR slope: -0.4 mL/min/1.73m2/year (95% CI: -2.27, 1.48) (Source: Hughes, 2016: Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomized phase III ATTRACT study) Forward-Looking Statements This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to: the safety and efficacy and therapeutic potential of isaralgagene civaparvovec, including the potential for it to be a one-time, durable treatment option for Fabry disease that can improve patient outcomes and treat the underlying pathology of Fabry disease; the presentation of clinical data from the Phase 1/2 STAAR study; the potential for isaralgagene civaparvovec to qualify for the FDA's Accelerated Approval program, including the adequacy of data generated in the Phase 1/2 STAAR study to support any such approval; expectations concerning the availability of additional data to support a potential BLA submission for isaralgagene civaparvovec, and the timing of such submission; the potential to accelerate the expected timeline to approval of isaralgagene civaparvovec; Sangamo's plans to seek a potential collaboration partner for ST-920; and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to Sangamo's lack of capital resources to obtain regulatory approval for and commercialize its product candidates in a timely manner or at all, including the ability to secure a collaboration partner for ST-920; the uncertain timing and unpredictable nature of clinical trial results, including the risk that preliminary or topline data is not indicative of final results, that the therapeutic effects observed in the latest clinical data from the Phase 1/2 STAAR study will not be durable in patients and that final clinical trial data from the study will not validate the safety and efficacy of isaralgagene civaparvovec, including that the 52-week data from the Phase 1/2 STAAR study will not support a BLA submission and/or that the 104-week data from such study will not verify the clinical benefit of isaralgagene civaparvovec or support FDA approval, and that the patients withdrawn from ERT will remain off ERT; Sangamo's need for substantial additional funding to execute its operating plan and to continue to operate as a going concern; the effects of macroeconomic factors or financial challenges on the global business environment, healthcare systems and Sangamo's business and operations; the research and development process; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; the potential for technological developments that obviate technologies used by Sangamo; Sangamo's reliance on collaborators and the potential inability to secure additional collaborations; and Sangamo's ability to achieve expected future financial performance. All forward-looking statements about Sangamo's future plans and expectations, including Sangamo's development plans for its product candidates, are subject to Sangamo's ability to secure adequate additional funding. There can be no assurance that Sangamo and its current or potential future partners will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and its collaborators. These risks and uncertainties are described more fully in Sangamo's Securities and Exchange Commission, or SEC, filings and reports, including in Sangamo's Annual Report on Form 10-K for the year ended December 31, 2024, as supplemented by its Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, each filed with the SEC, and future filings and reports that Sangamo makes from time to time with the SEC. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law. View source version on Contacts Investor Relations Louise Wilkieir@ Media Inquiries Melinda Hutcheonmedia@
Business Wire
18-06-2025
- Business
- Business Wire
Incyte Announces FDA Approval of Monjuvi ® (tafasitamab-cxix) in Combination with Rituximab and Lenalidomide for Patients with Relapsed or Refractory Follicular Lymphoma
WILMINGTON, Del.--(BUSINESS WIRE)--Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has approved Monjuvi® (tafasitamab-cxix), a humanized Fc-modified cytolytic CD19-targeting monoclonal antibody, in combination with rituximab and lenalidomide for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL). "Patients living with relapsed or refractory FL have been waiting for new options that improve progression-free survival without substantial increase in side effects. Based on the data from the inMIND trial of Monjuvi, today's approval brings to this patient population the first CD-19 and CD20-targeted immunotherapy combination and a potential new treatment standard,' said Hervé Hoppenot, Chief Executive Officer, Incyte. 'This second U.S. approval for Monjuvi reinforces our commitment to advancing innovation for the lymphoma community.' The Priority Review and FDA approval of the supplemental Biologics License Application (sBLA) for Monjuvi was based on data from the pivotal, randomized, double-blind, placebo-controlled Phase 3 inMIND trial evaluating the efficacy and safety of Monjuvi in combination with rituximab and lenalidomide in adult patients with relapsed or refractory FL. Data from the trial was featured in the Late-breaking Session (LBA-1) at the 2024 American Society of Hematology (ASH) Annual Meeting. 1 The study met its primary endpoint demonstrating a statistically significant and clinically meaningful improvement in progression-free survival (PFS) by investigator assessment, which demonstrated 27.5% (N=273) of patients with an event in the Monjuvi group vs. 47.6% (N=275) of patients with an event in the control arm. Patients receiving Monjuvi in combination with rituximab and lenalidomide achieved a median PFS by investigator assessment of 22.4 months (95% CI, 19.2-not evaluable [NE]) compared to 13.9 months (95% CI, 11.5-16.4) in the control arm (hazard ratio [HR]: 0.43 [95% CI, 0.32-0.58]; P<0.0001). The PFS assessed by an Independent Review Committee (IRC) was consistent with investigator-based results. Median PFS by IRC was not reached (95% CI, 19.3-NE) in the Monjuvi group versus 16.0 months (95% CI, 13.9-21.1) in the control arm (HR: 0.41 [95% CI, 0.29-0.56]. The PFS benefit was consistent across prespecified patient subgroups, including number of previous lines of therapy. The safety of Monjuvi in patients with FL was evaluated in 546 patients in the inMIND trial. Serious adverse reactions occurred in 33% of patients who received Monjuvi in combination with rituximab and lenalidomide, including serious infections in 24% of patients (including pneumonia and COVID-19 infection). Other serious adverse reactions in ≥ 2% of patients included renal insufficiency (3.3%), second primary malignancies (2.9%), and febrile neutropenia (2.6%). Fatal adverse reactions occurred in 1.5% of patients, including from COVID-19, sepsis, and adenocarcinoma. The most common adverse reactions (≥ 20%) in recipients of Monjuvi, excluding laboratory abnormalities, were respiratory tract infections (including COVID-19 infection and pneumonia), diarrhea, rash, fatigue, constipation, musculoskeletal pain, and cough. The most common Grade 3 or 4 laboratory abnormalities (≥ 20%) were decreased neutrophils and decreased lymphocytes. 'Follicular lymphoma is generally an indolent yet chronic cancer that frequently recurs after treatment, making long-term disease control a critical objective,' said Christina Poh, M.D., Assistant Professor of Medicine at the University of Washington and Fred Hutchinson Cancer Center. 'The FDA approval of Monjuvi in combination with rituximab and lenalidomide marks a significant advancement, offering a chemotherapy-free option that has demonstrated a meaningful reduction in the risk of disease progression across a broad patient population, including those with high-risk disease.' FL is the second most common type of non-Hodgkin lymphoma (NHL) and represents up to 30% of NHL cases. 2 While considered an indolent, slow-growing disease with prolonged survival, FL is challenging to treat due to its tendency for frequent relapse, need for multiple lines of therapy and potential transformation into large B-cell lymphoma. 2,3 'While the initial responses to FL treatment are often positive, recurrence can become increasingly difficult for patients to manage as they navigate emotions and the next treatment steps related to relapse,' said Mitchell Smith, M.D., Ph.D., Chief Medical Officer, Follicular Lymphoma Foundation. "We are pleased that the FDA has approved tafasitamab, part of a treatment combination offering a new option for patients living with this chronic disease.' In July 2020, Monjuvi in combination with lenalidomide received FDA approval for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication was approved under accelerated approval by the U.S. FDA based on overall response rate (ORR). Continued approval of Monjuvi for this indication may be contingent on verification and description of clinical benefit in confirmatory trial(s). Tafasitamab is also being evaluated as a therapeutic option in an ongoing pivotal trial for first-line DLBCL. Incyte is committed to supporting patients and removing barriers to access medicines. Eligible patients in the U.S. who are prescribed Monjuvi have access to IncyteCARES (Connecting to Access, Reimbursement, Education and Support), a comprehensive program offering personalized patient support, including financial assistance and ongoing education and additional resources. More information about IncyteCARES is available by visiting or calling 1-855-452-5234, Monday through Friday, from 8 a.m. to 8 p.m. ET. About inMIND A global, double-blind, randomized, controlled Phase 3 study, inMIND (NCT04680052) evaluated the efficacy and safety of tafasitamab in combination with rituximab and lenalidomide compared with placebo in combination with rituximab and lenalidomide in patients with relapsed or refractory follicular lymphoma (FL) Grade 1 to 3a or relapsed or refractory nodal, splenic or extranodal marginal zone lymphoma (MZL). The study enrolled a total of 654 adults (age ≥18 years). The primary endpoint of the study is progression-free survival (PFS) by investigator assessment in the FL population, and the key secondary endpoints are PFS in the overall population as well as positron emission tomography complete response (PET-CR) and overall survival (OS) in the FL population. For more information about the study, please visit About Monjuvi ® (tafasitamab-cxix) Monjuvi ® (tafasitamab-cxix) is a humanized Fc-modified cytolytic CD19-targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb ® engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP). MorphoSys and Incyte entered into: (a) in January 2020, a collaboration and licensing agreement to develop and commercialize tafasitamab globally; and (b) in February 2024, an agreement whereby Incyte obtained exclusive rights to develop and commercialize tafasitamab globally. In the U.S., Monjuvi is approved by the U.S. Food and Drug Administration in combination with lenalidomide and rituximab for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL). MONJUVI is not indicated and is not recommended for the treatment of patients with relapsed or refractory marginal zone lymphoma outside of controlled clinical trials. Additionally, Monjuvi received accelerated approval in the United States in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). In Europe, Minjuvi ® (tafasitamab) received conditional Marketing Authorization from the European Medicines Agency in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory DLBCL who are not eligible for ASCT. XmAb® is a registered trademark of Xencor, Inc. Monjuvi, Minjuvi, the Minjuvi and Monjuvi logos and the 'triangle' design are registered trademarks of Incyte. IMPORTANT SAFETY INFORMATION What are the possible side effects of MONJUVI? MONJUVI may cause serious side effects, including: Infusion reactions. Your healthcare provider will monitor you for infusion reactions during your infusion of MONJUVI. Tell your healthcare provider right away if you get fever, chills, rash, flushing, headache, or shortness of breath during an infusion of MONJUVI Low blood cell counts (platelets, red blood cells, and white blood cells). Low blood cell counts are common with MONJUVI, but can also be serious or severe. Your healthcare provider will monitor your blood counts during treatment with MONJUVI. Tell your healthcare provider right away if you get a fever of 100.4 °F (38 °C) or above, or any bruising or bleeding Infections. Serious infections, including infections that can cause death, have happened in people during treatment with MONJUVI and after the last dose. Tell your healthcare provider right away if you get a fever of 100.4 °F (38 °C) or above, or develop any signs or symptoms of an infection The most common side effects of MONJUVI when given with lenalidomide in people with DLBCL include: respiratory tract infection feeling tired or weak diarrhea cough fever swelling of lower legs or hands decreased appetite The most common side effects of MONJUVI when given with lenalidomide and rituximab in people with FL include: respiratory tract infections diarrhea rash feeling tired or weak muscle and bone pain constipation cough These are not all the possible side effects of MONJUVI. Your healthcare provider will give you medicines before each infusion to decrease your chance of infusion reactions. If you do not have any reactions, your healthcare provider may decide that you do not need these medicines with later infusions. Your healthcare provider may need to delay or completely stop treatment with MONJUVI if you have severe side effects. Before you receive MONJUVI, tell your healthcare provider about all your medical conditions, including if you Have an active infection or have had one recently Are pregnant or plan to become pregnant. MONJUVI may harm your unborn baby. You should not become pregnant during treatment with MONJUVI. Do not receive treatment with MONJUVI in combination with lenalidomide if you are pregnant because lenalidomide can cause birth defects and death of your unborn baby You should use an effective method of birth control (contraception) during treatment and for 3 months after your last dose of MONJUVI Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with MONJUVI Are breastfeeding or plan to breastfeed. It is not known if MONJUVI passes into your breastmilk. Do not breastfeed during treatment and for at least 3 months after your last dose of MONJUVI You should also read the lenalidomide Medication Guide for important information about pregnancy, contraception, and blood and sperm donation. Tell your healthcare provider about all the medications you take, including prescription and over- the-counter medicines, vitamins, and herbal supplements. Call your doctor for medical advice about side effects. You may report side effects to the FDA at (800) FDA-1088 or You may also report side effects to Incyte Medical Information at 1-855-463-3463. Please see the full Prescribing Information including the Medication Guide for Monjuvi. About Incyte A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia. For additional information on Incyte, please visit or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube. Incyte Forward-Looking Statements Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding whether Monjuvi may provide a successful treatment option for patients with FL, contain predictions, estimates and other forward-looking statements. These forward-looking statements are based on Incyte's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other regulatory authorities outside of the United States; the efficacy or safety of Incyte and its partners' products; the acceptance of Incyte and its partners' products in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in Incyte's reports filed with the Securities and Exchange Commission, including its annual report for the year ended December 31, 2024 and its quarterly report on form 10Q for the quarter ended March 31, 2025. Incyte disclaims any intent or obligation to update these forward-looking statements.
Business Insider
14-06-2025
- Business
- Business Insider
Regenxbio's RGX-202 shows efficacy in Phase 1/2 Duchenne trial
Regenxbio (RGNX) announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial. Updates include positive functional, safety and biomarker data for RGX-202, Regenxbio's potential best-in-class, investigational gene therapy for Duchenne muscular dystrophy. The functional data demonstrate consistent benefit among dose level 2 participants at 9 and 12 months following treatment with RGX-202. The data further supports the planned Biologics License Application submission under accelerated approval in mid-2026. Regenxbio announced positive interim functional results from the first five participants, aged approximately 6 to 12 years at dosing, receiving RGX-202 at dose level 2. RGX-202 continues to demonstrate evidence of positively impacting disease trajectory. At 9 months, RGX-202 participants demonstrated improvement in function and exceeded external controls on all measures. Four out of the five participants reached 12-months post dosing. Results at 12 months are similar to those seen at 9 months. RGX-202 participants demonstrated improved performance on timed function tests and NSAA, exceeding external natural history controls at 12 months. All participants within this cohort demonstrated improvement on all timed function tests compared to baseline. RGX-202 was appropriately localized to the sarcolemma, demonstrating that the differentiated construct is appropriately targeting the muscle. RGX-202 was well tolerated with no serious adverse events and no AEs of special interest. Regenxbio is enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial of RGX-202. The pivotal trial is expected to support a Biologics License Application submission using the accelerated approval pathway in mid-2026. Regenxbio expects to share top-line data in the first half of 2026.
