Bioverativ Therapeutics, Inc.

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

Appointments strengthen the leadership team and deepen expertise in drug discovery, oncology research, and strategic partnerships Dr. Brian Jones appointed to Scientific Advisory Board LEXINGTON, MA / ACCESSWIRE / September 12, 2023 / Triana Biomedicines, Inc. (TRIANA), a leading biopharmaceutical company focused on advancing a target-first and rational molecular glue discovery platform to address difficult to drug disease targets, today announced the appointments of Dr. Vito Palombella as Chief Scientific Officer and Dr. Reza Mazhari as Chief Business Officer. Additionally, Dr. Brian Jones is appointed to the growing Scientific Advisory Board (SAB). "I am thrilled to have Vito and Reza join the team at such a pivotal time for the company. As we rapidly advance our discovery pipeline currently focused on targeted protein degradation, Vito's oncology and drug development expertise and Reza's prowess in business development and portfolio strategy complement and strengthen our growing team. Both share a depth in translational sciences that will play a central role in the development of TRIANA's future molecular glue products," said Dr. Patrick Trojer, President and CEO of TRIANA. "Also, I am delighted to welcome Dr. Brian Jones to our Scientific Advisory Board. We are privileged to have the opportunity to work with a premier group of molecular glue thought leaders on drug development initiatives from discovery through drug commercialization. Their insights will help advance our efforts to build our world class molecular glue discovery platform and a robust pipeline of innovative molecular glue therapeutics." "I joined TRIANA because of its high powered scientific team and its product-focused approach to exploit this exciting new modality to tackle undrugged cancer targets," said Dr. Vito Palombella. "We are at the cusp of creating transformative medicines for patients." "I am very pleased to join the TRIANA team as we explore opportunities which leverage the company's proprietary scientific platform and discovery pipeline," said Dr. Reza Mazhari. "There is a growing interest in unlocking the full potential of molecular glues to develop precision medicines for a broad range of indications, allowing TRIANA to consider various value-creating asset- and/or platform-focused business development opportunities in the months and years ahead." "This is an exciting time for TRIANA as they deliver on the potential of molecular glue therapies. I look forward to working with the company and the other members of the SAB in guiding the company to address the undruggable dilemma facing many patients with first-in-class therapies," said Dr. Brian Jones. Vito J. Palombella, PhD, joins TRIANA with 30 years of scientific leadership and experience advancing first-in-class therapeutic programs, as well as a successful track record of building drug discovery and development organizations. Prior to joining TRIANA, he was the Chief Scientific Officer (CSO) at Surface Oncology, where he led the non-clinical research and development organization. Prior to that, Dr. Palombella was EVP and CSO at Infinity Pharmaceuticals, where he was responsible for drug discovery and preclinical development. He held roles of increasing scientific responsibility at Syntonix Pharmaceuticals, Millennium Pharmaceuticals, LeukoSite Inc, and ProScript. Dr. Palombella was involved in the discovery and development of bortezomib and duvelisib, both for cancer therapy. Dr. Palombella holds a PhD in Viral Oncology and Immunology from the New York University Medical Center and completed his postdoctoral fellowship at Harvard University. Reza Mazhari, PhD, brings to TRIANA more than twenty years of business development as well as drug discovery and development experience. Prior to joining TRIANA, he was the Head of Search and Evaluation for Oncology at Novartis, where he led numerous successful in-licensing deals that contributed to the early-stage oncology pipeline and expanded the portfolio of enabling technologies. Dr. Mazhari was also responsible for out-licensing and divestment efforts at Novartis Institutes for BioMedical Research (NIBR). Previously, Dr. Mazhari was the Vice President of Translational Medicine at Rexahn Pharmaceuticals. Prior to that, he held dual responsibilities for leading drug discovery and early development in addition to leading the company's business development efforts at Cerecor. He was the co-founder of Cardioxyl Pharmaceuticals which was acquired by Bristol-Myers Squibb for $1.8 billion. He began his career as a faculty member at the School of Medicine in The Johns Hopkins University. Dr. Mazhari holds a PhD in Bioengineering from University of California at San Diego and a post-doctoral research fellowship in Cardiovascular Medicine from The Johns Hopkins School of Medicine. Brian Jones, PhD, is a seasoned drug discovery leader with over 30 years of industry experience. He is responsible for over 20 INDs across a broad spectrum of therapeutic indications. Most recently, he incubated, launched and was the CSO at Cedilla Therapeutics, which successfully discovered distinctive conditional modulators of targeted cancer biology. He was Entrepreneur in Residence at Third Rock Ventures, with active involvement in a wide variety of therapeutic areas and responsible for the incubation and launch of several private biotech companies. Previously, he was Head of Discovery Chemistry and Chemical Biology at Novartis Institute for Biomedical Research. Dr. Jones holds a PhD in Organic Chemistry from Imperial College of Science at University of London and a NATO post-doctoral research fellowship from Yale University. About TRIANA Biomedicines, Inc. TRIANA Biomedicines is a private biotechnology company, headquartered in Lexington, Massachusetts and focused on building the leading molecular glue discovery platform to regulate disease targets that are difficult to address with any other modality. TRIANA's drug discovery engine is powered by high-resolution structural insights, state-of-the-art AI and computational tools, and bespoke chemical libraries. TRIANA's target first and rational approach to molecular glue discovery is currently focused on inducing or enhancing the degradation of high-pro targets. The therapeutic approach pioneered by TRIANA has the potential to fundamentally change the paradigm of small molecule drug discovery and bring significant therapeutic benefits to patients. Contact IR@trianabio.com SOURCE: TRIANA Biomedicines, Inc. View source version on accesswire.com:

TIRAT HACARMEL, Israel, Jan. 05, 2023 (GLOBE NEWSWIRE) -- Minovia Therapeutics (Minovia), a clinical-stage company focused on developing novel Mitochondrial Augmentation Technology (MAT) to fight mitochondrial diseases, has announced a Board appointment. Maha Radhakrishnan, M.D., joins the board at a time in which the company advances clinical programs in primary mitochondrial diseases and hematological disorders associated with mitochondrial dysfunction. As a trained physician, Maha is a senior leader with extensive pharmaceutical and biotech experience covering clinical development, post-marketing regulatory activities, medical affairs, safety, and reimbursement requirements. Maha is the Chief Medical Officer of Biogen, and in her previous roles, she served as Senior Vice President and Global Head of Medical, Primary Care Business Unit at Sanofi, Senior Vice President and Head of Worldwide Medical at Bioverativ Therapeutics Inc., and Head of Europe & Canada Medical and US Medical at Biogen. She has also worked at Bristol Myers Squibb, Cephalon, and United Health Group before first joining Biogen in early 2013. Maha has significant experience across a broad range of therapeutic areas and disciplines, including the central nervous system (neurology and psychiatry), rheumatology and immunology, rare blood disorders, ophthalmology, cardiology, diabetes, and internal medicine. Natalie Yivgi Ohana, Co-Founder and CEO Minovia Therapeutics, said, “Mitochondrial diseases are often fatal and currently viewed as untreatable, something we are working relentlessly to change. So far twelve patients suffering Primary Mitochondrial Diseases were dosed with our Mitochondrial Augmentation Technology, both through compassionate use program and a Phase 1/2 trial in Israel. Minovia Invested greatly in research and development in the last two years, establishing strong scientific and GMP foundations, which will enable us to seek approval for Pearson Syndrome and expand to other disease areas. As board member at Minovia, we will be able to draw on Maha’s considerable medical, regulatory and drug development expertise and experience in bringing successful treatments to patients with primary and secondary mitochondrial diseases.” Maha added: “Minovia is changing the way we look at mitochondrial therapies. They are on the precipice of meaningfully and measurably improving the lives of patients in need. I look forward to providing oversight and helping Minovia achieve its goals to approve their novel mitochondrial cell therapies in multiple disease areas.” The appointment of Maha as a board member follows on from Minovia’s recent partnership with Astellas Pharma to develop novel Mitochondrial Cell Therapies, as well as the initial data publication in Science Translational Medicine (https://www.science.org/doi/10.1126/scitranslmed.abo3724). Contact Info@minoviatx.com About Minovia Therapeutics Minovia Therapeutics is a clinical stage company and the first to use a cell therapy approach to treat patients affected with mitochondrial diseases through its Mitochondrial Augmentation Technology (MAT) platform. MAT is being developed as a robust and scalable therapeutic platform targeting the root cause of diseases generated by mitochondrial dysfunction. The lead product, MNV-201 is composed of autologous hematopoietic stem cells enriched with healthy allogeneic mitochondria. Minovia’s initial clinical focus is on rare mitochondrial diseases for which there are no approved treatments in the United States and the unmet medical need is immense. After completing an IND enabled phase I/II clinical trial in Pearson Syndrome, a fatal pediatric disease, Minovia is planning to accelerate the clinical development for Pearson Syndrome and expand the pipeline to other Hematological/Mitochondrial-related diseases. Harnessing the power of mitochondria, Minovia is committed to exploring the full potential of its proprietary platform to address mitochondrial diseases ranging from orphan indications to more common and age-related diseases. For more information, please visit http://minoviatx.com/.