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Pictured: Syringe and vial on background of market stock graph/iStock, MadamLead Every year, Clarivate curates a list of drugs with the potential of becoming breakthrough and blockbuster medicines in the coming year. This year, 13 molecules made it to the 2024 Drugs to Watch list, the majority of them approved for oncology and rare diseases. Michael Ward, global head of thought leadership, life sciences and healthcare at Clarivate, told BioSpace that the practice is an opportunity to identify drugs that would be consequential to the life sciences and the health sector in the coming year. The selected drugs were either undergoing Phase II or III clinical trials, in the pre-registration or registration stage, approved early in 2023 or were deemed to have a high chance of impacting patient wellbeing. Among them is bluebird bio’s lovotibeglogene autotemcel (Lyfgenia), approved last year for treating sickle cell disease in patients aged 12 and above. According to the report, this molecule treats the underlying causes of sickle cell disease and transfusion-dependent beta-thalassemia and is said to be more effective than current treatment options. Another example is Sanofi’s efanesoctocog alfa (Altuviiio) for hemophilia A, which is expected to make sales of $1.77 billion in 2029. Some of the drugs on the list, such as AstraZeneca’s datopotamab deruxtecan, an antibody drug conjugate, and Johnson & Johnson’s talquetamab (Talvey), a bispecific antibody, represent new modalities. Ward said these drug types are gaining more approvals from the FDA than traditional drug platforms, and this pushes pharmaceutical companies to be more innovative. “We’re going to see pharmaceutical companies looking around to make sure that they’ve got access to the technology platforms that are going to be the springboard for the next generation of medicines,” said Ward. “That will be RNA technologies, it will be cell and gene therapy and it will be gene editing.” To achieve this feat, big pharmaceutical companies often collaborate with smaller innovative companies. The best-selling drugs in some of the top pharmaceutical companies are from small companies, said Ward. For instance, all five of Bristol-Myers Squibb’s best-selling drugs are from smaller companies it acquired, he noted. For example, BMS assumed ownership of its best-selling drug Revlimid after purchasing Celegene Corporation in 2019. Ward predicted that 2024 will see many such deals. Clarivate’s full 2024 Drugs to Watch List: Aflibercept (Eylea) Budesonide (Tarpeyo/Kinpeygo) Datopotamab deruxtecan (Dato-DXd) Efanesoctocog alfa (Altuviiio) Ensifentrine (RPL554) Exagamglogene autotemcel (exa-cel) Lovotibeglogene autotemcel (lovo-cel) Mirikizumab (LY-3074828) Niraparib + abiraterone acetate (Akeega) RSVpreF (Abrysvo) RSVpreF3 (Arexvy) Talquetamab (Talvey) Zolbetuximab (IMAB362) Besides ramping up partnerships in the industry, another trend is that global pharmaceutical companies are becoming very interested in the growing chronic disease market in mainland China, according to Matthew Arnold, principal analyst, life sciences and healthcare at Clarivate. Among seven drugs the report highlights that could either improve treatment outcomes or achieve $1 billion blockbuster status by 2029 is Sanofi and Regeneron Pharma Inc.’s Dupilumab, expected to make sales of $1.5 billion in 2029 in China alone. “We’ve seen in the last five to ten years significant regulatory reforms in mainland China that have really opened up that market to a lot of pharma products that were previously excluded,” Arnold said. Last year Clarivate reported that mainland China has eased its regulatory protocols for testing and approvals of drugs, allowing more drugs to be available to patients. The current report also looked at the most successful drug of 2023. Although not on the watch list for 2024, Ward said Merck & Co. was the most successful company last year, mostly because of its cancer drug Keytruda, which has continued to improve clinical outcomes in patients. First approved in 2014 for treating advanced melanoma, Keytruda has since been approved for more than 20 indications, including, recently, the early stages of non-small cell lung cancer. “It’s described as a pipeline in a drug because there are lots of opportunities for that drug to be used in lots of different cancers in combination with other drugs to be highly effective,” said Ward. Keytruda’s patent exclusivity will expire in 2028. Clarivate’s report also highlighted the impact of gene editing and artificial intelligence (AI), including machine learning (ML), citing the use of these technologies for developing personalized therapies based on an individual’s genetic makeup. Five hundred AI/ML-related clinical trials were conducted in 2022, and one of the drugs on this year’s list, Bluebird’s Lyfgenia, was developed using the technology. Arnold said using these technologies in drug development means more novel molecules will make it to the market. “Only one of ten molecules that ever actually enter into human clinical trials ever get anywhere near the regulator for approval,” said Ward. He predicted that using AI in clinical trials would eliminate redundancy in the research and development process and improve these odds, but that the technology would not necessarily reduce healthcare costs. “I don't expect to see any decline in the amount of money that is going to be put to work for R&D.” Patience Asanga is a Nigeria-based freelance science journalist who writes about the environment, biotechnology and life sciences.

Therapeutic advancements for sickle cell disease, RSV, breast cancer, Crohn's and other ailments poised to advance patient health despite urgent challenges facing life science companies LONDON, Jan. 8, 2024 /PRNewswire/ -- Clarivate Plc (NYSE:CLVT), a global leader in connecting people and organizations to intelligence they can trust to transform their world, today announced the release of its annual Drugs to Watch™ report. The report provides in-depth predictive analysis of drugs with the potential for standout commercial and/or clinical success. The report, a key industry resource in the evolving healthcare landscape, has identified over 85 Drugs to Watch over the 11-year course of its publication. This year, analysts identified 13 new-to-market therapeutics and drugs poised to launch in 2024 which Clarivate predictive analytics project will achieve blockbuster status by 2029 or deliver game-changing benefits to patients. These promising advancements include a broad spectrum of therapeutic innovation for conditions including breast cancer, hemophilia A, sickle cell disease, Crohn's disease, ulcerative colitis, respiratory syncytial virus (RSV) and multiple myeloma, among others. In addition, the report spotlights the growing chronic disease market in Mainland China, highlighting seven drugs that are likely to achieve $1 billion blockbuster status by 2029 or to have a significant impact for Chinese patients in need of better treatment options. Mike Ward, Global Head of Thought Leadership, Life Sciences and Healthcare, Clarivate said: "The fundamentals underpinning the biopharma sector have never been stronger, with new technologies fueling medical advancements and providing treatment options to patients with previously unmet needs. Leveraging deep industry expertise and comprehensive therapeutic area differentiated data, this year's Drugs to Watch report identifies innovative medicines based on recent scientific breakthroughs poised to have extraordinary impacts on patient outcomes." 2024 is anticipated to be a transformational year of innovation for the pharma industry. New modalities like antibody drug conjugates and AI/machine learning, underpinned by scientific breakthroughs in the past decade, are achieving clinical successes and providing therapies for patients with limited treatment options. However, external influences such as government initiatives to contain healthcare costs, the sustained high cost of capital and global geopolitical disputes are negatively impacting investor appetites for the sector. The 2024 edition of Drugs to Watch highlights trends that will likely be consequential to the discovery, development and delivery of new medicines, and spotlights drugs and drug candidates that are likely to achieve important milestones in the coming years as they establish themselves as either blockbuster or breakthrough medicines. The 2024 Drugs to Watch, are: Aflibercept (high dose; EYLEA® HD), developed by Bayer and Regeneron Pharmaceuticals Inc For individuals with wet age-related macular degeneration (AMD), diabetic macular edema (DME) or diabetic retinopathy (DR) whose treatment choices include invasive, burdensome administration that limits treatment uptake, high-dose aflibercept offers less-frequent administration while achieving similar efficacy and safety as the current standard of care. Budesonide (TARPEYO®/Kinpeygo®/Nefecon), developed by Calliditas Therapeutics AB, Everest Medicines and STADA Arzneimittel AG TARPEYO®/Kinpeygo® (developed under the project name Nefecon) is a second-generation, synthetic, non-halogenated form of the corticosteroid budesonide. The delayed release formulation of budesonide has shown greater efficacy for protein reduction and slowing the decline in kidney function in primary immunoglobulin A (IgA) as well as a much better safety profile than conventional corticosteroids. Datopotamab deruxtecan (Dato-DXd), developed by AstraZeneca and Daiichi Sankyo With the potential to become the best-in-class TROP2-targeted antibody drug conjugate (ADC), datopotamab deruxtecan is set to be second to market (after TRODELVY®; Gilead Sciences Inc) for both HR-positive/HER2-negative and triple-negative breast cancer, and to enter the non-small cell lung cancer (NSCLC) market. The collaboration between AstraZeneca and Daiichi Sankyo combines the former's strategic focus on NSCLC and breast cancers and investment in ADCs with the latter's proprietary DXd ADC technology. Efanesoctocog alfa (ALTUVIIIO™/BIVV001), developed by Sanofi (Bioverativ Therapeutics Inc) and Swedish Orphan Biovitrum AB (Sobi®) Efanesoctocog alfa is the first once-weekly factor VIII (FVIII) replacement intravenous infusion therapy, which will help reduce the burden associated with the injection frequency of other currently available FVIII therapies. For patients reluctant to receive novel therapies, such as mAbs or gene therapy, efanesoctocog alfa will likely be an appealing option. Clinicians also view efanesoctocog alfa favorably given the attainable FVIIII levels, injection frequency and safety profile demonstrated in clinical trials to date. Ensifentrine (RPL554), developed by Verona Pharma Ensifentrine is an inhaled dual phosphodiesterase (PDE)3 and PDE4 inhibitor that is expected to reduce exacerbations in moderate to severe chronic obstructive pulmonary disease (COPD) without the systemic side effects of current PDE inhibitors that are delivered orally. If approved, it would be the first in class as well as the first novel mechanism that has become available for maintenance COPD treatment in more than 10 years. The clinical and safety profile of ensifentrine makes it a promising addition to the limited treatment class options available for this patient population. Exagamglogene autotemcel (CASGEVY™ /exa-cel) and lovotibeglogene autotemcel (LYFGENIA™/lovo-cel/formerly LentiGlobin™), developed by CRISPR Therapeutics and Vertex Pharmaceuticals Inc (exa-cel) and Bluebird Bio (lovo-cel) All eyes are on exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel, which are set become the first disease-modifying therapies for sickle cell disease (SCD) and beta-thalassemia, a significant achievement for a patient population with debilitating, life-altering diseases that have limited symptomatic and curative treatments currently available. The excitement around exagamglogene autotemcel also stems from the landmark first approval of a CRISPR/Cas9 gene-edited therapy globally, and the approval sets the stage for upcoming approvals in other regions. Mirikizumab (Omvoh™/ LY-3074828), developed by Eli Lilly and Company Mirikizumab, an mAb targeting the p19 subunit of IL-23, was approved as first-in-class therapy for ulcerative colitis by the EMA and the U.S. FDA and will likely be the third in the class approved for Crohn's disease. Included in Drugs to Watch 2023, a delayed U.S. launch meant that it remains a drug to watch for 2024. Niraparib + abiraterone acetate (AKEEGA™), developed by Johnson & Johnson Innovative Medicine This is the first and only dual action (or fixed-dose combination [FDC]) tablet combining a PARP inhibitor (niraparib) and a next-generation hormonal therapy (abiraterone acetate). Its ability to serve as a treatment for patients with deleterious or suspected deleterious BRCA-mutated, metastatic castration-resistant prostate cancer (mCRPC) should help to fullfil the need for more effective treatments. RSVpreF (ABRYSVO™/PF-06928316) and RSVpreF3 (AREXVY/GSK-3844766A), developed by Pfizer Inc (ABRYSVO) and GSK plc (AREXVY) Respiratory syncytial virus (RSV) infections continue to be a public health concern, particularly for infants and older adults (65 years and older). A common upper respiratory infection that can result in hospitalizations in severe cases, RSV infection tends to be seasonal and present with symptoms similar to those of influenza and COVID-19. The first approvals of RSV vaccines (RSVpreF and RSVpreF3) targeted at infants and older adults mark a significant public health milestone. Talquetamab (TALVEY™), developed by Johnson & Johnson Innovative Medicine After receiving conditional and accelerated approval from the European Commission and FDA, respectively, talquetamab became the first-in-class bispecific antibody targeted to CD3 and GPRC5D to treat multiple myeloma. It was approved based on the pivotal phase 1/2 MonumenTAL-1 trial for heavily pretreated patients with relapsed or refractory (R/R) multiple myeloma. Ongoing phase 3 trials are expected to provide confirmation of clinical benefit in talquetamab's approved setting and lead to label expansions in other multiple myeloma patient populations, including in combination with other approved agents. Talquetamab is poised as an important addition to the treatment armamentarium for this incurable, often-relapsing disease. Zolbetuximab (IMAB362), developed by Astellas Pharma Inc Metastatic HER2-negative gastric and gastroesophageal junction (GEJ) adenocarcinoma is notoriously difficult to treat and has a significant unmet need for new efficacious treatments. In contrast to HER2-positive disease (for which HER2-targeted agents such as trastuzumab [Genentech] and ENHERTU [Daiichi Sankyo] are available), targeted treatment options are more limited for HER2-negative patients. Zolbetuximab would address some of that unmet need as a first-in-class claudin 18.2 (CLDN18.2) inhibitor in oncology as well as first-line metastatic HER2-negative gastric or GEJ adenocarcinoma. New technology platforms that are likely to achieve significant proof of medical use in the market in 2024 include CRISPR-Cas9 gene-editing as well as artificial intelligence (AI)/machine learning (ML) tool applications in drug discovery, clinical development and commercial launch. In the long run, the latter technologies hold enormous potential to help drugmakers cut costs and shorten innovation cycles, enabling the delivery of more innovative drugs to patients faster going forward. Access the Drugs to Watch 2024 report from Clarivate, here. For more Drugs to Watch updates and analyses throughout the year, visit the Drugs to Watch web page and follow Clarivate for Life Sciences & Healthcare on LinkedIn and X. Join the conversation, using #DrugstoWatch2024. To learn more about how Clarivate can help healthcare companies inform and shape the drug discovery, development and delivery process, visit . Methodology for the Clarivate Drugs to Watch 2024 Report To identify this year's Drugs to Watch, Clarivate drew from the expertise of over 160 analysts covering hundreds of diseases, drugs and markets, along with 11 integrated data sets that span the R&D and commercialization lifecycle, including: Cortellis Competitive Intelligence™, Disease Landscape & Forecast, BioWorld™, Drug Timeline & Success Rates, Cortellis Clinical Trials Intelligence™, Cortellis Generics Intelligence™, Cortellis Deals Intelligence™, Access & Reimbursement payer studies, Clarivate Real World Data and Analytics, Web of Science™ Derwent Innovation™, and other industry sources including biopharma company press releases, filings and peer-reviewed publications. Candidate drugs in phase 2 or phase 3 trials, at pre-registration or registration stage, or already launched in 2023 were selected for analysis, including both novel treatments and already-marketed drugs pursuing new indications that could be particularly impactful. Drugs launched prior to 2023 were excluded. The dataset was filtered for drugs that had total forecast sales of $1 billion or more by 2029. Clarivate experts and analysts evaluated each drug in its individual context, based on factors such as expected approval or launch dates, competitive landscape, regulatory status, trial results, market dynamics and other key factors, and added novel drugs that, while likely to fall short of blockbuster status, are poised to be therapeutic game-changers. Please note that Clarivate analysts generated the data shown in this report prior to December 31, 2023. The Drugs to Watch 2024 Report and the treatments referenced in this release are based on Clarivate's current expectations per existing data, but actual results derived from the drugs named in the report and here may differ significantly. Clarivate is committed to comprehensively supporting customers across the entire drug, device and medical technology lifecycles to advance human health. By combining patient journey data, therapeutic area expertise, artificial intelligence and analytics in ways that unlock hidden insights, data-driven decisions and accelerating innovation, Clarivate's end-to-end research intelligence is designed to enable customers to make informed evidence-based decisions. About Clarivate Clarivate is a leading global information services provider. We connect people and organizations to intelligence they can trust to transform their perspective, their work and our world. Our subscription and technology-based solutions are coupled with deep domain expertise and cover the areas of Academia & Government, Life Sciences & Healthcare and Intellectual Property. For more information, please visit clarivate.com. Media Contact: Catherine Daniel Director, External Communications, Life Sciences & Healthcare [email protected] SOURCE Clarivate Plc