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 https://clarivate.com/industries/life-sciences-and-healthcare.
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.
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