Muscular Disorders, Atrophic

- BLA submitted to FDA for Upstaza™ for the treatment of AADC deficiency - - NDA to be submitted mid-year for Translarna™ based on FDA feedback - SOUTH PLAINFIELD, N.J., March 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the submission of a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. In addition, based on recent feedback from the FDA, PTC plans to re-submit an NDA for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). PTC expects to make the NDA resubmission by mid-year. "We are excited to bring Upstaza one step closer to children in the United States with the highly mortal and fatal disease of AADC deficiency. The data collected to date continue to demonstrate the transformative clinical benefits the gene therapy provides to patients," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "In addition, we look forward to the Translarna NDA resubmission this summer. We know U.S. patients and families have been waiting for an approved therapy that specially targets nmDMD." In addition, the sepiapterin MAA for PKU remains on schedule for submission to the EMA by the end of this month. About Upstaza™ (eladocagene exuparvovec) Upstaza is a one-time gene replacement therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L–amino acid decarboxylase (AADC) deficiency with a severe phenotype. It is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy, containing the human DDC gene.1 It is designed to correct the underlying genetic defect, by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production.2,3 The efficacy and safety profile of Upstaza has been demonstrated across clinical trials and compassionate use programs.1 The first patient was dosed in 2010. In clinical trials, Upstaza demonstrated transformational neurological improvements. The most common side effects were initial insomnia, irritability and dyskinesia. Administration of Upstaza occurs through a stereotactic surgical procedure, a minimally invasive neurosurgical procedure used for the treatment of a number of pediatric and adult neurological disorders. The Upstaza administration procedure is performed by a qualified neurosurgeon in centers specialized in stereotactic neurosurgery. Upstaza has received marketing authorization in Europe, Great Britain and Israel. About aromatic L-amino acid decarboxylase (AADC) deficiency AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life – physical, mental and behavioral. The suffering of children with AADC deficiency may be exacerbated by: episodes of distressing seizure-like oculogyric crises causing the eyes to roll up in the head, frequent vomiting, behavioral problems, and difficulty sleeping. The lives of affected children are severely impacted and shortened. Ongoing physical, occupational and speech therapy, and interventions, including surgery, also are often required to manage potentially life-threatening complications such as infections, severe feeding and breathing problems. About Translarna™ (ataluren) Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the tradename of ataluren, is licensed in multiple countries for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States. About Duchenne Muscular Dystrophy (Duchenne) Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20's due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at and follow us on Facebook, Instagram, LinkedIn and Twitter at @PTCBio. For More Information: Investors: Kylie O'Keefe +1 (908) 300-0691 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statement This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; PTC's ability to use the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from our trials in Translarna; expectations with respect to Upstaza, including any regulatory submissions and potential approvals, commercialization, manufacturing capabilities, the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna and Upstaza. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. Acronyms: BLA: Biologics License Application DMD: Duchenne Muscular Dystrophy EMA: European Medicines Agency FDA: U.S. Food and Drug Administration MAA: Marketing Authorization Application NDA: New Drug Application PKU: Phenylketonuria References: Tai CH, et al. Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency. Mol Ther. 2022;30(2):509-518. Chien et al. AGIL-AADC gene therapy results in sustained improvements in motor and developmental milestones through 5 years in children with AADC deficiency. Poster presented at the 48th Annual Meeting of the Child Neurology Society, Charlotte, NC, USA, Oct 23-26, 2019. Chien YH, et al. Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial. Lancet Child Adolesc Health. 2017;1(4):265-273. SOURCE PTC Therapeutics, Inc.

NEW YORK, March 18, 2024 /PRNewswire/ -- Technavio categorizes the global gene therapy market size as estimated to increase by USD 4,613.9 million between 2022 and 2027. The market's growth momentum will be progressing at a CAGR of 19.91% during the forecast period. During the forecast period, North America is anticipated to drive 39% of global market growth, as per Technavio's analysts. The US, particularly, emerges as a promising investment hub within the region, supported by substantial funding from entities like the National Institutes of Health (NIH) and the Biomedical Advanced Research and Development Authority (BARDA) to emerging players in the market. This influx of government and private sector investment is propelling the development of innovative gene therapies, with 18 clinical trials and 17 biotech startups underway. Such financial backing is poised to significantly stimulate market demand in the coming years. Continue Reading Technavio has announced its latest market research report titled Global Gene Therapy Market 2023-2027 Discover some insights on market size historic data (2017 to 2021) and Forecast (2023 to 2027) before buying the full report - Request a sample report Technavio has segmented the market based on therapy areas (oncology, CNS, ophthalmology, rare diseases, and others), and delivery mode (in vivo and ex vivo). The market growth in the oncology segment will be significant during the forecast period. The global oncology drugs market has a huge unmet demand for the treatment of various indications. Also, the increasing prevalence of cancer indications has led pharmaceutical companies and research institutions to conduct extensive research to develop drugs for these indications. The lack of efficacy of small molecules and biomolecules has led companies to move towards effective therapeutic options such as gene therapy. Such factors will increase the segment growth during the forecast period. For insights on global, regional, and country-level parameters with growth opportunities from 2017 to 2027 - Download a Sample Report Key Companies The global gene therapy market is fragmented, with the presence of several global as well as regional vendors. A few prominent vendors that offer gene therapy in the market are Abeona Therapeutics Inc., Adaptimmune Therapeutics plc, Adverum Biotechnologies Inc., Amgen Inc., Astellas Pharma Inc., Biogen Inc., bluebird bio Inc., Editas Medicine Inc., ElevateBio, F. Hoffmann La Roche Ltd., Generation Bio Co., Gilead Sciences Inc., Novartis AG, Orchard Therapeutics Plc, Poseida Therapeutics Inc., Sangamo Therapeutics Inc., Sibiono GeneTech Co. Ltd., Syncona Ltd., uniQure NV, and Voyager Therapeutics Inc. and others. Abeona Therapeutics Inc.: The company offers gene therapy solutions such as Phase 3 VIITAL Clinical Trial of EB 101. Amgen Inc.: The company offers gene therapy solutions such as CAR T cell immunotherapies. Astellas Pharma Inc.: The company offers gene therapy solutions such as AT132, AT845, AT466. Biogen Inc.: The company offers gene therapy solutions such as GTxAU. The growth of the market is driven by the increase in special drug designations. Most gene therapy programs have been granted orphan drug designation, fast track designation, or breakthrough designation by the U.S. FDA, EMA, and other regulatory bodies for the treatment of certain diseases. In addition, orphan drug status will also help the company with tax incentives in its next research. Such advantages of specialty drug designations will drive the growth of the market during the forecast period. Growing research in gene therapy for CVDs and orphan diseases is one of the key market trends that is contributing to the market growth. High treatment cost is one of the factors hindering the gene therapy market growth. Driver, Trend & Challenges are the factor of market dynamics which states about consequences & sustainability of the businesses, find some insights from a sample report! Analyst Review Gene therapy stands at the forefront of medical innovation, offering promising solutions for a myriad of genetic disorders ranging from atrophy to muscular dystrophy. As the world grapples with health challenges like the COVID-19 pandemic, companies like Applied Genetic Technologies Corporation and Benitec Biopharma in countries like Australia and Belgium are actively engaged in pioneering gene therapy research to combat such conditions. Beta thalassemia, sickle cell disease, and spinal muscular atrophy represent just a fraction of the genetic issues that gene therapy aims to address. With the advent of breakthrough treatments like betibeglogene autotemcel and onasemnogene abeparvovec, patients suffering from these debilitating conditions now have newfound hope. The gene therapy market, bolstered by advancements in CRISPR Therapeutics and lentivirus technology, is witnessing exponential growth. As companies like Pfizer, Cellectis, and Novartis Gene Therapies invest in research and development, the market is projected to expand further, especially in regions like California and Massachusetts, known for their biotechnology hubs. However, this growth isn't without challenges. Regulatory bodies like ClinicalTrials.gov, the FDA, and the International Organization for Standardization (ISO 9000) play pivotal roles in ensuring the safety and efficacy of gene therapies. Striking a balance between innovation and compliance with regulations such as the California Consumer Privacy Act and the General Data Protection Regulation remains paramount. One of the critical factors driving the gene therapy market is the increasing prevalence of genetic disorders worldwide. From hereditary illnesses to congenital diseases, the demand for innovative treatments continues to rise. Moreover, with advancements in gene editing and therapeutic gene delivery systems, the potential to target specific cells and protein molecules offers new avenues for treatment. Venture capital financing and partnerships between academia and industry players further fuel research efforts. Companies like Lonza Group and Oxford Biomedica contribute to the manufacturing and logistics aspects of gene therapy, ensuring efficient drug delivery and scalability. In conclusion, the gene therapy market holds immense promise in addressing genetic disorders and improving patient outcomes. As the field continues to evolve, collaboration, adherence to regulations, and a focus on patient-centric approaches will be essential in unlocking the full potential of gene therapy to tackle the most challenging medical conditions of our time. Download a Sample Report Market Overview The Gene Therapy Market is witnessing exponential growth globally, with companies like Applied Genetic Technologies Corporation and Benitec Biopharma leading the charge. This revolutionary approach offers hope for conditions such as atrophy and beta thalassemia, providing targeted solutions to genetic disorders. Recent advancements include Betibeglogene autotemcel, a promising treatment for beta-thalassemia pioneered by CRISPR Therapeutics. Biomedicine is at the forefront of this transformative field, driving innovation and clinical trials. Amidst the COVID-19 pandemic, gene therapy research continues unabated, with countries like Australia and Belgium actively contributing to the global effort. As the market expands, gene therapy emerges as a beacon of hope for patients worldwide, offering personalized treatments and potential cures. Download a Sample Report Related Reports: The cell and gene therapy market size is expected to increase by USD 9.97 billion from 2021 to 2026, and the market's growth momentum will accelerate at a CAGR of 20.15%. The cell therapy market size is expected to increase by USD 21.061 billion from 2021 to 2026, and the market's growth momentum will accelerate at a CAGR of 56.79%. TOC Executive Summary Market Landscape Market Sizing Historic Market Size Five Forces Analysis Market Segmentation by Therapy Areas market Segmentation by Delivery Mode Customer Landscape Geographic Landscape Drivers, Challenges, and Trends Company Landscape Company Analysis Appendix About US Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavio's report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio's comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios. Contact Technavio Research Jesse Maida Media & Marketing Executive US: +1 844 364 1100 UK: +44 203 893 3200 Email: [email protected] Website: SOURCE Technavio

Pictured: FDA headquarters surrounded by pill bottles/Taylor Tieden for BioSpace The FDA’s busy March continues with six target action dates remaining on the calendar. Over the next two weeks, the regulator will decide on investigational therapies for Duchenne muscular dystrophy, chronic kidney disease anemia, a rare metabolic disorder and more. Read below for more. Orchard/Kyowa Kirin Eye First FDA Approval for Metachromatic Leukodystrophy Orchard Therapeutics is proposing Libmeldy (atidarsagene autotemcel) for the treatment of metachromatic leukodystrophy (MLD), a rare metabolic disease. The FDA’s target action date is March 18, 2024. If approved, Libmeldy would become the first treatment for early-onset MLD in the U.S. The approval would also mark the first major regulatory victory for Kyowa Kirin since it acquired Orchard in October 2023 for approximately $477.6 million. Affecting one in every 100,000 live births, MLD is a heritable and life-threatening disease characterized by the toxic build-up of sulfatides in the brain, liver, kidneys and other organs. This accumulation can lead to organ damage and neurological issues such as cognitive, behavioral and motor decline. MLD patients also suffer from severe seizures and over time lose their ability to talk, swallow, eat, move and see. MLD is caused by a pathological mutation in the arylsulfatase-A (ARSA) gene. Libmeldy, an autologous gene therapy, addresses the underlying mechanism of MLD by using a patient’s own stem cells to deliver functioning copy of the ARSA gene. The European Union approved Libmeldy for the treatment of MLD in December 2020. In the U.S., Libmeldy, also known as OTL-200, has been granted the FDA’s Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations. Its Biologics License Application (BLA), which the regulator accepted in September 2023, has been given Priority Review. Libmeldy’s regulatory bid is backed by data from the OTL-200 clinical development program, which enrolled a total of 39 pediatric patients and used data from 49 untreated natural history controls. Results showed that Libmeldy could preserve motor function and cognitive development in treated patients. ITF Awaits Verdict for DMD Candidate ITF, the U.S. subsidiary of Italian pharma Italfarmaco Group, is proposing its investigational HDAC blocker givinostat for the treatment of Duchenne muscular dystrophy (DMD). The FDA’s verdict is due on March 21, 2024. Designed to be orally available, givinostat works by blocking histone deacetylases (HDACs), a group of enzymes that affect gene expression in muscles. The “deregulation of HDACs is a major consequence of the lack of dystrophin associated with DMD,” ITF head Matt Trudeau told BioSpace in a previous interview. Givinostat’s mechanism of action can potentially “inhibit HDAC pathological overactivity and thereby impact the cascade of events leading to muscle damage which, in turn, may counteract disease pathology and slow muscle deterioration,” Trudeau explained. ITF’s New Drug Application (NDA), which was submitted in June 2023, includes data from the Phase III EPIDYS trial, which enrolled 179 ambulant boys aged six years and older. Givinostat was given on top of steroid treatment. Patients who received the HDAC blocker showed a significantly slower decline than placebo recipients in the time it took to climb four stairs, according to a June 2022 readout. Givinostat also aced its secondary endpoints, including the time to rise test and the North Star Ambulatory Assessment, which measures patient function. Givinostat was initially granted Priority Review and was due for an FDA decision in December 2023. However, the regulator informed Italfarmaco weeks before its target action date that it would extend the review period by three months to allow additional review time. Merck Seeks to Open New Treatment Approach for PAH By March 26, the FDA will release its decision on Merck’s investigational activin signaling inhibitor sotatercept, which the company is proposing for the treatment of pulmonary arterial hypertension (PAH). In a statement alongside the BLA acceptance, Joerg Koglin, senior vice president of global clinical development at Merck Research Laboratories, said sotatercept “has the potential to transform the treatment of patients with PAH.” Sotatercept is a fusion protein therapeutic composed of the extracellular domain of the human activin receptor and the Fc domain of an IgG1 antibody. This structure allows sotatercept to trap TGF-β ligands, thereby helping to restore balance between growth-promoting and growth-limiting pathways. Preclinical models have demonstrated that sotatercept’s mechanism of action can modulate vascular cell proliferation and reverse vascular and ventricle remodeling. Merck is supporting sotatercept’s regulatory bid with data from the pivotal Phase III STELLAR study, a double-blinded and placebo-controlled trial that enrolled more than 320 patients. The results, published in The New England Journal of Medicine, showed that sotatercept could significantly increase six-minute walk distance after 24 weeks. Sotatercept likewise outperformed placebo in several secondary metrics, including pulmonary vascular resistance, improvement in WHO functional class and time to death or clinical worsening. Akebia Anticipates Long-Awaited Decision for CKD Anemia Drug After a long back and forth, the FDA is set to announce its verdict on Akebia’s NDA seeking approval for vadadustat in anemia due to chronic kidney disease (CKD) in adult patients on dialysis. Vadadustat is an investigational inhibitor of the hypoxia-inducible factor prolyl hydroxylase (HIF-PH) enzyme, which is involved in the cellular response to low oxygen levels. Vadadustat mimics the physiological effects of altitude on oxygen availability and can promote the production of red blood cells and the delivery of oxygen to tissues. In March 2022, the FDA rejected vadadustat, citing safety concerns. Phase III data for the candidate, released in September 2020, showed that while vadadustat aced its primary and key secondary endpoints, it failed its chief safety outcomes. Compared with darbepoetin alfa, major adverse cardiovascular events occurred more readily in the vadadustat arm. To demonstrate a more favorable risk-benefit profile, the FDA suggested that Akebia conduct new clinical trials to support vadadustat. Following the rejection, Akebia in November 2022 filed a Formal Dispute Resolution Request with the FDA, insisting on the “favorable balance of the benefits and risks of vadadustat,” CEO John Butler said in an investor call at the time. The FDA then requested additional information to support Akebia’s appeal, but in February 2023 ultimately decided to defer the decision to the Office of New Drugs. In May 2023, the FDA handed Akebia a victory when it decided the biotech no longer needed to conduct additional studies for a resubmission. Instead, it could re NDA to target CKD patients on dialysis and include post-approval data from Japan. The new application would also include the risk of heart- or liver-related side effects in the proposed label. Esperion Seeks Expanded Label for Cholesterol Drugs Esperion is proposing to expand the labels for its bempedoic acid drugs Nexletol and Nexlizet, allowing their use to lower cardiovascular risk in patients with or at risk of atherosclerotic cardiovascular disease. The FDA’s decision is due on March 31. Bempedoic acid is a non-statin oral drug that lowers low-density lipoprotein cholesterol (LDL-C) via the inhibition of the ATP citrate lyase enzyme. The drug was first approved in February 2020 as Nexletol, becoming the first FDA-approved non-statin therapy to lower LDL-C since 2002. In March 2023, Esperion presented full data from the Phase III CLEAR study demonstrating that Nexletol cut the risk of major adverse cardiovascular events (MACE) by 13% to 15%. The drug also lowered the risk of heart attack and coronary revascularization by 23% and 19%, respectively, Esperion reported. In an accompanying statement, Esperion CEO Sheldon Koenig called the results “practice changing.” In an investor call, he added that if the label expansion is approved, Esperion could see major growth in sales and Nexletol could reach blockbuster status. Investors were not as impressed, however, and the biotech’s shares dropped around 20% in reaction to the readout. Esperion’s partner Daiichi Sankyo was also unimpressed with the data, refusing to pay a $300 million milestone that Esperion insists it will be owed once cardiovascular risk reduction data are included in Nexletol’s European label. According to an SEC filing from Esperion, it was entitled to a milestone payment if Nexletol could show at least a 20% cardiovascular risk reduction. The 23% decrease in heart attack risk, according to the biotech, qualified it for the milestone. Daiichi Sankyo disagreed, insisting that the analysis should focus on the 13% drop in MACE risk, which fell short of this milestone. In January 2024, the partners reached an amicable end to the payment spat, with Daiichi Sankyo agreeing to pay Esperion $125 million in two separate tranches. Regeneron Eyes Follicular Lymphoma, DLBCL Approval for Bispecific Antibody Capping off the FDA’s month is a target action date for Regeneron’s BLA proposing its investigational bispecific antibody odronextamab for relapsed or refractory follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL). The regulator is set to release its verdict on March 31. Regeneron is supporting its application with data from Phase I and pivotal Phase II trials, which were presented at the 64th American Society of Hematology meeting in December 2022. One study evaluated odronextamab as a treatment for grade I to IIIa relapsed or refractory FL and found it elicited an 82% objective response rate (ORR), with 75% seeing a complete response. The median response time was 20.5 months. The trial also found that median progression-free survival was 20 months, while median overall survival at the time had not been reached. The second study tested odronextamab in relapsed or refractor DLBCL, for which the investigational antibody demonstrated a 49% objective response rate in CAR-T-naïve patients. In this subgroup, 31% achieved complete response. In those who had previously been exposed to CAR-T therapy, the ORR was 48%, with a 32% complete response rate. Odronextamab is an investigational bispecific antibody designed to target CD20, which is typically expressed on cancer cells, and CD3, which is found on T cells. This mechanism of action allows odronextamab to bring the immune cell closer to the tumor cell, facilitating the body’s cancer-killing activity. If approved, odronextamab would become the first bispecific antibody authorized to treat both FL and DLBCL. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.