Latest news with #BLA
Hans India
a day ago
- Politics
- Hans India
Pakistan: 13 soldiers killed, 29 injured in North Waziristan suicide bombing
Islamabad: As many as 13 Pakistani soldiers were killed and 29 others injured, including 19 civilians, in a suicide attack early Saturday morning in Khadi area of North Waziristan district of Khyber Pakhtunkhwa. Citing security agencies, Pakistani media outlet Mashriq TV stated that a suicide bomber crashed an explosive-laden vehicle into a mine-resistant Improvised Explosive Device (IED) vehicle belonging to the Bomb Disposal Unit amid a curfew imposed due to military movement in the area. The powerful blast resulted in the killing of 13 military personnel and injuring 24 people, including 14 civilians. There were reports of indiscriminate firing also which caused injuries to 19 civilians, including women and children, in the area. Reports suggest that the rescue operations were launched immediately following the blast. According to local authorities, the condition of several injured, including women and children, remains critical and the number of casualties might rise significantly. The Usud al-Harab organisation, a sub-faction of the Hafiz Gul Bahadur group affiliated with Tehreek-e-Taliban (TTP) Pakistan, has reportedly taken responsibility for the suicide attack. The incident is termed as the deadliest attack in North Waziristan in recent months, raising serious concerns over the deteriorating security conditions in the area. The Inter Services Public Relations (ISPR), the media wing of the Pakistan Army, has not yet commented on the attack. According to reports, the security forces have cordoned off the area and launched a search operation in the region. Last month, a car bomb explosion in Gulistan Area of Qila Abdullah, a border area of Pakistan's Balochistan province with Afghanistan, killed at least four people and injured more than 20 others, eight of them critically injured. The explosion occurred in Jabbar commercial market situated on the Quetta–Chaman National Highway in Gulistan town, adjacent to the fort of Pakistan's Frontier Corps (FC). As per details, the massive explosion destroyed many shops and vehicles parked in the area and walls of the FC Fort were also damaged. Locals said that many shops in the market also caught fire. Tehreek-e-Taliban Pakistan had claimed responsibility for the attack with Pakistani analysts suggesting a clear coordination between the Baloch Liberation Army (BLA) and the TTP, who have intensified their attacks in the recent past. At the same time, the BLA has also intensified its attacks on security forces in the province.
Business Wire
2 days ago
- Business
- Business Wire
Omeros Submits Narsoplimab Marketing Authorization Application to the European Medicines Agency for the Treatment of TA-TMA
SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today announced the recent submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). The MAA includes response-based analyses in narsoplimab-treated TA-TMA patients as well as analyses comparing overall survival between narsoplimab-treated patients and a well-matched external control group. Collectively, the results demonstrate a 61% response rate and, compared to the matched external control, a three-fold improvement in overall survival. The submission also includes outcomes in over 130 TA-TMA patients treated with narsoplimab under Omeros' expanded access program. Narsoplimab has been granted orphan drug designation by the EMA for treatment in hematopoietic stem cell transplant, enabling review of the MAA through the centralized procedure. This allows for a single marketing authorization to cover all EU member states and the European Economic Area countries of Iceland, Liechtenstein and Norway. The review procedure begins in mid-July and will follow a standard review timeline. The Committee for Medicinal Products for Human Use (CHMP) will conduct the scientific assessment and will issue an opinion at the end of the review. This opinion is typically adopted by the European Commission, with a final decision expected in mid-2026. The MAA submission follows the acceptance for review by the U.S. Food and Drug Administration (FDA) of the resubmission of the Biologics License Application (BLA) for narsoplimab for the treatment of TA-TMA. The resubmission was assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 25, 2025. About Narsoplimab Narsoplimab, also known as 'OMS721,' is an investigational fully human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 has been demonstrated to leave intact the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. A biologics license application (BLA) for use of narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) is under review by the U.S. Food and Drug Administration (FDA) and Omeros has submitted the corresponding European MAA. FDA has granted narsoplimab breakthrough therapy and orphan drug designations for TA-TMA and orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in hematopoietic stem-cell transplant. About Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA) is a significant and often lethal complication of stem cell transplantation. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, graft-versus-host disease, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of TA-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an approximately 40-percent incidence of TA-TMA, and high-risk features may be present in up to 80 percent of these patients. In severe cases of TA-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae (e.g., dialysis) are common. There is no approved therapy or standard of care for TA-TMA. About Omeros Corporation Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing first-in-class small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders, including complement-mediated diseases and cancers, as well as addictive and compulsive disorders. Omeros' lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application under review by FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy. Omeros' long-acting MASP-2 inhibitor OMS1029 has successfully completed Phase 1 single- and multiple-ascending dose clinical studies. OMS906, Omeros' inhibitor of MASP-3, the key activator of the alternative pathway of complement, is in clinical development for paroxysmal nocturnal hemoglobinuria and complement 3 glomerulopathy. Funded by the National Institute on Drug Abuse, Omeros' lead phosphodiesterase 7 inhibitor OMS527 is in clinical development for the treatment of cocaine use disorder. Omeros also is advancing a broad portfolio of novel cellular and molecular immuno-oncology programs. For more information about Omeros and its programs, visit Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, which are subject to the 'safe harbor' created by those sections for such statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as 'aim,' 'anticipate,' 'believe,' 'could,' 'estimate,' 'expect,' 'goal,' 'intend,' 'likely,' 'look forward to,' 'may,' 'objective,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'slate,' 'target,' 'will,' 'would' and similar expressions and variations thereof. Forward-looking statements, including statements regarding the anticipated review process and timing of FDA action on the resubmitted BLA for narsoplimab in the United States, the anticipated review process and timing of EMA action on the MAA submission, the prospects for obtaining FDA or EMA approval of narsoplimab in any indication, and expectations regarding the sufficiency and availability of our capital resources to fund current and planned operations, including the potential commercialization of narsoplimab if it is approved by FDA or the EMA, are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Omeros' actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, unfavorable or unexpected regulatory conclusions or interpretations related to the clinical data, external registry data, statistical analyses or other information and data included in the narsoplimab BLA or narsoplimab MAA, inability to respond satisfactorily to information requests during regulatory review of the narsoplimab BLA or MAA, potential differences between the diagnostic criteria used in our pivotal trial and in the external registry, and whether FDA and the EMA determine the registry used in our statistical analysis is sufficiently representative of TA-TMA patients, unanticipated or unexpected outcomes or requirements of regulatory processes in relevant jurisdictions, our financial condition and results of operations, including our ability to raise additional capital for our operations on favorable terms or at all, regulatory processes and oversight, challenges associated with manufacture or supply of our products to support clinical trials, regulatory inspections and/or commercial sale following any marketing approval, changes in reimbursement and payment policies by government and commercial payers or the application of such policies, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading 'Risk Factors' in our Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 31, 2025 and in subsequent reports filed with the Securities and Exchange Commission. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law.
Yahoo
5 days ago
- 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@
Newsweek
5 days ago
- Business
- Newsweek
Padres' Fernando Tatis Jr. Involved in Lawsuit Against Big League Advance Fund
Based on facts, either observed and verified firsthand by the reporter, or reported and verified from knowledgeable sources. Newsweek AI is in beta. Translations may contain inaccuracies—please refer to the original content. Fernando Tatis Jr. signed a 14-year, $340 million deal with the San Diego Padres in 2021. He had agreed to a deal with Big League Advance Fund and Big League Advantage, LLC to receive an upfront payment in exchange for a percentage of his future earnings in 2017, according to The Athletic's Brittany Ghiroli and Dennis Lin. Now that Tatis is a highly paid player, he owes Big League Advance millions of dollars. Tatis is suing the company and seeks to hold Big League Advance accountable for "exploitative, predatory business practices, which shamelessly push illegal loans on young, vulnerable athletes — most from economically disadvantaged Latin American countries," read a press release by his legal team, according to The Athletic. At the time of the deal, Tatis told The Athletic's Ken Rosenthal that he needed an advance to hire a trainer, rent a better apartment and buy food. He received a $700,000 signing bonus when he signed with the Chicago White Sox in 2015. SEOUL, SOUTH KOREA - MARCH 21: Fernando Tatis Jr. #23 of the San Diego Padres celebrates the team's 15-11 victory in the 2024 Seoul Series game between San Diego Padres and Los Angeles Dodgers at... SEOUL, SOUTH KOREA - MARCH 21: Fernando Tatis Jr. #23 of the San Diego Padres celebrates the team's 15-11 victory in the 2024 Seoul Series game between San Diego Padres and Los Angeles Dodgers at Gocheok Sky Dome on March 21, 2024 in Seoul, South press release by his legal team claims that BLA has been operating an "unlicensed lending business that evades legal oversight and siphons millions in earnings from California workers," per The Athletic. Francisco Mejía filed a similar suit in 2018, but eventually dropped the case. According to The Athletic, Tatis refused to make any further comments when he arrived at Petco Park on Monday. However, it's clear that a deal he thought was favorable in 2017 is no longer viewed as such. "The money BLA gives players is not a loan, per se," wrote The Athletic. "If a player doesn't reach the big leagues, he owes nothing. If he does reach the major leagues, however, the price — a portion of pre-tax major-league earnings — can be hefty. Michael Schwimer, a 39-year-old former pitcher for the Philadelphia Phillies who is the president and CEO of BLA, told The Athletic in 2018 that his company's goal is to help minor leaguers guard against uncertain futures." Tatis is one of the biggest stars in the game, and this lawsuit could end up being a very important development for many young players around the league. More MLB: Mets Insider Shares Concern For 25-Year-Old's Shocking 'Sudden Downfall'
Washington Post
5 days ago
- Business
- Washington Post
Padres star Tatis sues Big League Advance in attempt to get out of future earnings deal
SAN DIEGO — San Diego Padres star Fernando Tatis Jr. filed a lawsuit Monday against Big League Advance in an attempt to void the future earnings contract he signed as a 17-year-old minor leaguer that could cost him about $34 million. The lawsuit, filed in San Diego County Superior Court, accuses BLA of using predatory tactics to lure him into an 'investment deal' that was actually an illegal loan. BLA misrepresented itself to Tatis, hiding its unlicensed status and pushing him into loan terms banned by California's consumer protection laws, the suit alleges.
