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Cision Canada
8 hours ago
- Health
- Cision Canada
Biogen Disappointed by INESSS Recommendation on SKYCLARYS™ and its Impact on Quebec Patients with Friedreich Ataxia Français
TORONTO, June 5, 2025 /CNW/ - Biogen Canada Inc. is disappointed by INESSS's recommendation against listing SKYCLARYS™ (omaveloxolone), despite its therapeutic potential for individuals living with Friedreich ataxia (FA) — a community in urgent need of treatment options. A decision to not reimburse SKYCLARYS would leave individuals living with FA in Quebec without public access to the only approved treatment for this rare, progressive, and life-shortening neurodegenerative disease – despite a clear and ongoing medical need. 1 As the only approved treatment targeting disease progression, SKYCLARYS addresses a long-standing gap in care for Canadians living with FA, particularly in Quebec where a notable concentration of cases highlights the importance of equitable access. 2 "After years of research and failed trials with no approved treatments until now, the availability of a therapy with a demonstrated ability to slow disease progression and preserve mobility is profoundly meaningful for the Friedreich ataxia community, offering renewed hope and the possibility of managing the disease and care in FA. We remain committed to working with INESSS, government authorities, and clinicians to help ensure access to SKYCLARYS for eligible FA patients in Quebec and the rest of Canada," said Eric Tse, General Manager, Biogen Canada. SKYCLARYS was approved in March 2025 under Health Canada's Priority Review process as the first and only treatment in Canada to target the underlying mechanisms of FA in patients aged 16 and older. Canada's Drug Agency (CDA-AMC), which advises on drug reimbursement across Canada's public drug programs outside of Quebec, has issued a positive draft recommendation recognizing SKYCLARYS as an important therapeutic option, valued by patients, caregivers, and clinicians for its potential to improve quality of life and preserve function. 3 About SKYCLARYS™ (omaveloxolone) SKYCLARYS™ (omaveloxolone) is an oral, once-daily medication indicated for the treatment of Friedreich ataxia (FA) in adults and adolescents aged 16 years and older in the United States (U.S.), European Union and Canada. 4,5,6 SKYCLARYS received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). 5 The European Commission granted Orphan Drug designation in Europe to SKYCLARYS for the treatment of FA. 6 In Canada, marketing authorization for SKYCLARYS was granted under the Health Canada Priority Review process. 7 Biogen continues to be dedicated to advancing research and development efforts to enhance the understanding of FA and improve patient outcomes. This includes an ongoing, open-label, Phase 1 study to evaluate the use of SKYCLARYS in in pediatric patients aged 2 to 15 years. More details can be found at About Biogen Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment, to deliver long-term growth. Biogen has been proudly serving Canadian patients for more than 25 years. For information about Biogen Canada, please visit
Yahoo
28-05-2025
- Business
- Yahoo
Biogen strikes RNAi deal with City; Aurion withdraws IPO
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Today, a brief rundown of news involving City Therapeutics and Grin Therapeutics, as well as updates from Savara Pharma, Aurion Biotech and Prothena that you may have missed. Biogen is partnering with RNA drug developer City Therapeutics to develop a better way of reaching a unspecified target that 'mediates key central nervous system diseases.' Biogen will pay City, a startup that launched publicly late last year, $16 million in upfront fees and invest another $30 million in convertible notes that could later become a minority stake. 'With this effort, we are further expanding the modalities in our R&D toolbox to potentially reach our targets of interest more precisely by adding an RNAi-based approach," Biogen research head Jane Grogan said in a statement. — Ned Pagliarulo Biotechnology startup Grin Therapeutics will get $50 million in a deal that hands Angelini Pharma rights outside of North America to an experimental drug Grin has developed for genetic epilepsies and other neurological conditions. On Tuesday, Grin also announced the closing of a $140 million Series D round that involved Angelini and Blackstone Life Sciences. Grin's drug, radiprodil, targets a receptor that is thought to contribute to epilepsy when overactivated. The company plans to start a Phase 3 trial in the third quarter that will test radiprodil in a neurodevelopmental condition that causes seizures. — Gwendolyn Wu The Food and Drug Administration declined to review a drug Savara is developing for a rare lung condition called pulmonary alveolar proteinosis. According to Savara, the agency deemed the company's application incomplete and asked for more information about how the treatment, Molbreevi, is made. The FDA didn't identify any safety issues or request more efficacy data, Savara said. The company will request a meeting with the FDA within the next month. Its share price fell by more than 20% Tuesday.— Ben Fidler Aurion Biotech has withdrawn plans to go public, according to a regulatory filing. The cell therapy developer filed for an initial public offering in January, but did so despite opposition from shareholder Alcon, leading to a legal spat between the firm and another equity holder, Deerfield Management, over Aurion's future. Alcon bought a majority stake in Aurion in late March, however, and replaced the biotech's CEO. Aurion said in a Friday filing that an IPO is no longer "in the best interests of the company." — Ben Fidler Shares in Prothena fell by more than 30% to open Tuesday trading following the company's disclosure Friday that a late-stage trial of a drug called birtamimab failed to reach its main goal. As a result, Prothena has decided to shelve further development of birtamimab, which it had been studying as a treatment for the disease AL amyloidosis. 'This is not the outcome that we expected, and we are surprised and disappointed by these results,' said Prothena CEO Gene Kinney, in a statement. The company is now considering ways to reduce expenses, among them an expected substantial workforce reduction.' — Ned Pagliarulo Recommended Reading Regeneron defeats Amgen in PCSK9 case; 2 cell therapy biotechs cut staff 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
27-05-2025
- Business
- Yahoo
Biogen and City Therapeutics Announce Strategic Research Collaboration to Develop Select Novel RNAi-based Therapies
Collaboration leverages City Therapeutics' next-generation RNAi engineering technologies and Biogen's extensive drug development expertise CAMBRIDGE, Mass., May 27, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and City Therapeutics, Inc., a privately held biopharmaceutical company leading the future of RNA interference (RNAi)-based medicine, today announced a strategic collaboration to develop select novel RNAi therapies. Through the collaboration, City Therapeutics will leverage its next-generation RNAi engineering technologies to develop an RNAi trigger molecule combined with proprietary drug delivery technology from Biogen. The collaboration will initially focus on a single target that mediates key central nervous system diseases, utilizing tissue enhanced delivery technologies with the aim of allowing for systemic administration of medicines. Biogen will be responsible for IND-enabling studies and global clinical development along with any regulatory submissions and all activities related to commercialization. "This collaboration underscores Biogen's new strategic research approach of balancing our differentiated internal capabilities with external investments in cutting-edge science. With this effort, we are further expanding the modalities in our R&D toolbox to potentially reach our targets of interest more precisely by adding an RNAi-based approach," said Jane Grogan, Ph.D., Head of Research at Biogen. "We are excited to collaborate with City Therapeutics and their world-class scientists on key programs, as well as to invest in their company as part of this innovative effort to develop new approaches to treating disease.' 'Partnering with Biogen represents a meaningful milestone in our mission to expand the therapeutic reach of RNAi, as we pioneer the next generation of RNAi technology for breakthrough medicines,' said Andy Orth, Chief Executive Officer of City Therapeutics. 'By combining our novel RNAi platform with Biogen's industry-leading capabilities in global drug development, we aim to accelerate the advancement of therapies for serious diseases. We look forward to demonstrating the potential of our RNAi platform in addressing this area of significant unmet need.' Under the terms of the agreement, City Therapeutics will receive a total of $46 million in payments including a $16 million upfront payment and an investment of $30 million in exchange for a City Therapeutics convertible note, representing a minority equity interest in the company if converted. The upfront payment will be recorded by Biogen as an Acquired In-Process Research and Development expense in the second quarter of 2025. Should the initial program achieve certain development and commercial milestones, City Therapeutics is eligible to receive up to approximately $1 billion in potential milestone payments plus tiered royalties in the high single-digit to low double-digit range based on net sales. Biogen will have the option to select one additional target in the collaboration, subject to an additional payment and availability of the target. About BiogenFounded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients' lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at Follow us on social media – Facebook, LinkedIn, X, YouTube. About City TherapeuticsCity Therapeutics is a biopharmaceutical company harnessing next-generation engineering of RNAi trigger molecules to improve and expand the reach of RNAi-based medicines. The company is building a pipeline of innovative RNAi therapeutics to make a significant impact for patients across multiple therapeutic areas. Co-founded by pioneering executives and scientists in RNAi, City Therapeutics is based in Cambridge, MA, and has raised $135 million from leading life sciences investors. For more information, please visit us at and follow us on LinkedIn. BIOGEN SAFE HARBOR This press release contains forward-looking statements, relating to: our strategy and plans; potential of, and expectations for, our commercial business and pipeline programs, including in connection with RNAi-based medicines, the potential benefits, safety, and efficacy of our and our collaboration partners' products and investigational therapies; the anticipated benefits and potential of investments, actions to improve risk profile and productivity of R&D pipeline, collaborations, and business development activities; and our future financial and operating results. These forward-looking statements may be accompanied by such words as 'aim,' 'anticipate,' 'assume,' 'believe,' 'contemplate,' 'continue,' 'could,' 'estimate,' 'expect,' 'forecast,' 'goal,' 'guidance,' 'hope,' 'intend,' 'may,' 'objective,' 'outlook,' 'plan,' 'possible,' 'potential,' 'predict,' 'project,' 'prospect,' 'should,' 'target,' 'will,' 'would,' and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. This press release includes, among others, forward-looking statements including: that Biogen is building on a new foundation with the goal of long-term sustainable growth in its commercial portfolio; the multi-billion dollar potential of its late-stage pipeline; that we believe there remains a significant long-term opportunity for our ongoing product launches including LEQEMBI; that we believe that continued execution against these key strategic elements, as well as a disciplined approach to business development, will allow us to generate long-term value for our shareholders by bringing innovative medicines to patients; and all statements and information under the heading "Full Year 2025 Financial Guidance". These forward-looking statements are based on management's current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward looking statements will be realized in whole or in part. We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to be materially different from those stated or implied in this document, including, among others, factors relating to: our substantial dependence on revenue from our products and other payments under licensing, collaboration, acquisition or divestiture agreements; uncertainty of long-term success in developing, licensing, or acquiring other product candidates or additional indications for existing products; expectations, plans and prospects relating to product approvals, approvals of additional indications for our existing products, sales, pricing, growth, reimbursement and launch of our marketed and pipeline products; the potential impact of increased product competition in the biopharmaceutical and healthcare industry, as well as any other markets in which we compete, including increased competition from new originator therapies, generics, prodrugs and biosimilars of existing products and products approved under abbreviated regulatory pathways; our ability to effectively implement our corporate strategy; the successful execution of our strategic and growth initiatives, including acquisitions; the drivers for growing our business; difficulties in obtaining and maintaining adequate coverage, pricing, and reimbursement for our products; the drivers for growing our business, including our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential future healthcare reforms; risks related to commercialization of biosimilars, which is subject to such risks related to our reliance on thirdparties, intellectual property, competitive and market challenges and regulatory compliance; failure to obtain, protect, and enforce our data, intellectual property, and other proprietary rights and the risks and uncertainties relating to intellectual property claims and challenges; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in early stage clinical trials may not be predictive of results in later stage or large scale clinical trials or trials in other potential indications; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events, restrictions on use with our products, or product liability claims; risks relating to technology, including our incorporation of new technologies such as artificial intelligence into some of our processes; risks related to use of information technology systems and potential impacts of any breakdowns, interruptions, invasions, corruptions, data breaches, destructions and/or other cybersecurity incidents of our systems or those of connected and/or third-party systems; problems with our manufacturing capacity, including our ability to manufacture products efficiently or adequately address global bulk supply risks; risks relating to management, personnel and other organizational changes, including our ability to attracting, retaining and motivating qualified individuals; risks related to the failure to comply with current and new legal and regulatory requirements, including judicial decisions, accounting standards, and tariff or trade restrictions; the risks of doing business internationally, including geopolitical tensions, acts of war and largescale crises; risks relating to investment in our manufacturing capacity; risks relating to the distribution and sale by third parties of counterfeit or unfit versions of our products; risks relating to the use of social media for our business, results of operations and financial condition; fluctuations in our operating results; risks related to investment in properties; risks relating to access to capital and credit markets to finance our present and future operations and business initiatives and obtain funding for such activities on favorable terms; risks related to indebtedness; the market, interest, and credit risks associated with our investment portfolio; risks relating to share repurchase programs; change in control provisions in certain of our collaboration agreements; fluctuations in our effective tax rate and obligations in various jurisdictions in which we are subject to taxation; environmental risks; and any other risks and and uncertainties that are described in other reports we have filed with the U.S. Securities and Exchange Commission. These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q and Form 10-K, in each case including in the sections thereof captioned 'Note Regarding Forward-Looking Statements' and 'Item 1A. Risk Factors,' and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise. Media Contacts: BiogenJack CoxHead of Media City TherapeuticsKatie Engleman 1AB katie@ Investor Contacts: BiogenTim PowerHead of Investor RelationsIR@ City Therapeutics Stephanie Ascher Precision
Yahoo
22-05-2025
- Health
- Yahoo
CD40 Targeted Therapies Market Research Report 2025: Clinical Trials, Therapeutic Approaches & Market Opportunities to 2027 - Competition Heats Up with 20+ Therapies in Advanced Stage
CD40 targeted therapies are at the forefront of immunotherapy research, focusing on leveraging the CD40 receptor to treat diverse diseases. With over 20 therapies in trials, the first CD40 therapy is anticipated to receive approval by 2027. These therapies primarily target autoimmune, inflammatory, and cancer conditions, with increasing exploration into neurodegenerative and infectious diseases. Major pharmaceuticals like Sanofi and Biogen are pivotal players, enhancing their pipeline through innovations like GenMab's DuoBody platform. As the field grows, CD40 therapies are poised to become crucial in treating prevalent diseases. Dublin, May 22, 2025 (GLOBE NEWSWIRE) -- The "CD40 Targeted Therapies Clinical Trials, Therapeutic Approaches & Market Opportunity Insight 2025" has been added to offering. CD40 targeted therapies are gaining traction in immunotherapy research, offering potential treatment solutions for various diseases. Currently, no CD40 targeted therapies have been approved for clinical use, but several promising candidates are in late-stage clinical development. With more therapies set to enter late-stage trials next year, the potential of CD40 targeted therapies is expanding rapidly, indicating a possible pivotal role in the treatment of prevalent diseases in the near future. The strategic focus of CD40 targeted therapies has expanded from autoimmune and inflammatory diseases to include cancer, infectious diseases such as HIV and COVID-19, and neurodegenerative disorders like Alzheimer's and Parkinson's. Emerging research suggests potential advantages in cardiovascular diseases, where the CD40 signaling pathway contributes to inflammation and disease progression. Traditional monoclonal antibodies and next-generation molecular therapeutics, such as small interfering RNA (siRNA), are being employed to target the CD40 receptor. While antibodies may function as either agonists or antagonists, CD40 targeted siRNAs target CD40 protein expression by degrading mRNA. These approaches are undergoing rigorous trials to determine safety and efficacy. The antibody-based approach is predominant in CD40 research, with the most advanced antibodies geared towards autoimmune and inflammatory conditions entering phase 3 trials. For example, Sanofi's Frexalimab, targeting autoimmune diseases, is in phase 3 trials. In oncology, Alligator Bioscience is preparing phase 3 trials for mitazalimab, targeting pancreatic cancer. Technological advancements, notably GenMab's DuoBody platform, have played a significant role, enabling the development of bispecific antibodies targeting two antigens. The platform fosters innovations like GEN1042/BNT312, currently in phase 2 trials for malignant solid tumors. The competitive landscape for CD40 therapies is intensifying, with significant investments from major pharmaceutical firms like Amgen, Sanofi, GenMab, BioNTech, and Biogen. Collaborations between these companies and academic institutions are crucial, combining research innovation with clinical expertise to accelerate these therapies to the market. While clinical approval is pending, CD40 targeted therapies remain a promising research avenue across diverse disease areas. With more therapies advancing through trials, their emergence as a critical treatment cornerstone for cancer, autoimmune, and infectious diseases appears imminent. Global CD40 Targeted Therapies Clinical Trials, Therapeutic Approaches & Market Opportunity Insight 2025 Report Highlights: First CD40 Targeted Therapy Approval Expected By 2027 CD40 Targeted Therapies In Clinical Trials: > 20 Therapies CD40 Inhibitors Clinical Trials Insight By Company, Country, Indication & Phase CD40 Targeted Therapy Research & Development Trends By Indication CD40 Targeted Therapy Market Trend Analysis By Region CD40 Targeted Therapies Proprietary Technologies By Company Key Topics Covered: 1. Introduction To CD40 Targeted Therapies1.1 Overview1.2 Considering CD40 As Drug Target Over Other CD Markers1.3 History & Evolution Of CD40 Targeting Therapies 2. CD40 Targeted Therapies - Mechanism Of Action2.1 Agonist Mediated Activation Of CD40 Signaling2.2 Antagonist-Mediated Inhibition Of CD40 Signaling 3. CD40 Targeted Therapeutic Approaches3.1 Antibody Formats3.2 Fusion Proteins3.3 Peptides3.4 Nucleic Acids 4. CD40 Targeted Therapy Research & Development Trends By Indication4.1 Cancer4.2 Neurological Diseases4.3 Autoimmune & Inflammatory Disorders4.4 Transplant Rejection4.5 Microbial Infections 5. Global CD40 Targeted Therapy Market Outlook5.1 Current Market Trends & Developments5.2 Future Market Opportunities 6. CD40 Targeted Therapy Market Trend Analysis By Region6.1 US6.2 Europe6.3 China6.4 Japan6.5 Australia 7. CD40 Targeted Therapies Clinical Trials Insight By Company, Country, Indication & Phase7.1 Research7.2 Preclinical7.3 Phase I7.4 Phase I/II7.5 Phase II7.6 Phase III 8. CD40 Targeted Therapies - Proprietary Technologies By Company8.1 Alligator Bioscience - FIND Technology & Neo-X-Prime8.2 Aprilbio - SAFA Platform8.3 Biocytogen - RenMice8.4 EnnoDC - Unnamed Platform8.5 Genmab - DuoBody8.6 Harbour BioMed - Harbour Mice & HBICE8.7 Kyowa Kirin - REGULGENT8.8 Strike Pharma - ADAC Technology Platform 9. Global CD40 Targeted Therapy Market Dynamics9.1 Drivers & Opportunities9.2 Challenges & Restraints 10. Competitive Landscape10.1 Alligator Bioscience10.2 Amgen10.3 Biogen10.4 BioNTech10.5 Eledon Pharmaceuticals10.6 EnnoDC10.7 Genmab10.8 Innovent Bio10.9 Novartis10.10 Sanofi10.11 Tonix Pharmaceuticals10.12 UCB For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900Error 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
Business Wire
20-05-2025
- Business
- Business Wire
PepGen Appoints Kasra Kasraian, PhD, as Chief Technology Officer
BOSTON--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced the appointment of Kasra Kasraian, PhD, as Chief Technology Officer (CTO). Dr. Kasraian brings over 25 years of experience in product and process development, CMC strategy, and technical operations, spanning small and large molecules, as well as cell and gene therapies. 'We are pleased to welcome Kasra to our executive leadership team,' said James McArthur, PhD, President and Chief Executive Officer of PepGen. 'He brings a rare depth of expertise in product and process development, coupled with a proven track record of CMC leadership extending across early-stage development through multiple successful global regulatory approvals. Kasra's background advancing therapies for rare genetic disorders, along with his ability to navigate manufacturing and regulatory landscapes, will be instrumental as we progress our clinical programs through mid-stage development and beyond. We look forward to his contributions as we work to deliver potentially transformative therapies for people living with severe neuromuscular conditions.' Dr. Kasraian joins PepGen from bluebird bio, where he held various roles of increasing responsibility. Most recently, he served as Senior Vice President, Quality, Regulatory Affairs, and CMC Sciences. Prior to this role, he served as Senior Vice President, Technical Development and Operations, where he played a central role in the approval and launch of three gene therapy products—SKYSONA™, ZYNTEGLO™, and LYFGENIA™. Prior to bluebird bio, Dr. Kasraian led the Technical Operation, Quality, and Regulatory CMC functions at Zafgen, Inc. Previously, he held scientific and leadership roles at Biogen, Wyeth Biotech, Pfizer Inc., and Genetics Institute in the areas of formulation development, process development, technical services, manufacturing, and CMC management encompassing early phase development through commercialization for both small molecules and biologics. During his tenure at Biogen, the CMC team he led guided the submission and approval of 3 BLAs/MAAs. Dr. Kasraian earned his PhD in Pharmaceutical Sciences and his BS in Chemical Engineering from the University of Kentucky. About PepGen PepGen is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen's EDO platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, we are generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases. For more information, please visit Follow PepGen on LinkedIn and X.
