With the Simpliciti-T1 phase II trial testing new-mechanism TTP-399 as adjunct therapy in type 1 diabetes (T1D), High Point, N.C.-based Vtv Therapeutics Inc. nailed the HbA1c endpoint with none of the safety concerns foiling developers of type 2 diabetes (T2D) drugs who sought to broaden their labels. Read More
A high-profile test of two experimental medicines in people with autosomal dominant Alzheimer’s disease (ADAD), a rare inherited form of the disease, found that neither drug significantly slowed rates of cognitive decline vs. placebo, the primary endpoint. Read More
BEIJING – Zhongshan-based Akeso Inc., which focuses on bispecific antibodies, is again seeking pre-revenue listing on the Hong Kong Stock Exchange (HKEX), after its first IPO attempt failed to go through in December. The move comes at a time when analysts believe the coronavirus outbreak is weighing on the city’s IPO market. Read More
LONDON – Oculis SA reported positive results for its topical nanoparticle formulation of dexamethasone in the treatment of diabetic macular edema (DME), showing the eye drops reduced central macular thickness (CMT) in the phase II proof-of-concept trial. Read More
For depression, and other mental health disorders, the era of precision medicine has yet to arrive.
Symptoms are “very poorly reflective of the underlying biology,” Amit Etkin told BioWorld. Depression can manifest through multiple different symptoms that differ both between and within cultures. Read More
Top-line results from Biohaven Pharmaceutical Holding Co.’s phase III trial of troriluzole against placebo in treating generalized anxiety disorder (GAD) failed to hit its primary endpoint, prompting the company CEO to say the poor results support halting development plans for the glutamate modulator as a monotherapy in GAD. Read More
In a budget proposal sure to spark opposition, the Trump administration has proposed to provide the National Institutes of Health with only $38 billion in fiscal 2021, several billion dollars short of the agency’s funding for the current fiscal year. The proposal also calls for funding of less than $95 billion for the Department of Health and Human Services, a 10% reduction in funding that includes an adjustment for drug pricing proposals and a $5 million cut to FDA monies for the 21st Century Cures Act. Read More
Crowned by a potential cure for severe hemophilia A, that could become the most expensive drug ever, a new list of 11 medicines expected to generate $1 billion-plus in annual sales by the end of 2024 or earlier throws into stark relief the growing tension between medical innovation and society's ability to pay for it. The 2020 Cortellis Drugs to Watch list, including medicines both approved and likely to be, points to a future of ongoing conflict between payers and industry spurred by fundamental disagreements. Read More
New analysis from Clarivate Analytics' Cortellis Forecast Team predicts 11 medicines set to enter the market in 2020 will reach more than $1 billion in sales by 2024. Read More
Data for this report were compiled from Cortellis, the suite of life sciences intelligence solutions from Clarivate Analytics. Cortellis includes the broadest and deepest range of sources of intelligence across the R&D lifecycle, including annual filings, drug pipelines, clinical trials, patents, chemistry, deals, conferences and company announcements. Read More
No matter how effective it is, a drug is worthless if the people who need it can’t afford it. That’s been almost an anthem for patients and policy wonks testifying before U.S. Congress on drug prices. Read More
While the efficacy of three central nervous system (CNS) drugs awaiting regulatory approvals is not vastly different from currently marketed products, their formulations and methods of delivery, combined with what payers will support, make them formidable players in the multiple sclerosis (MS) and migraine markets. Read More
Jaime Sanders was just a child, barely 8 years old, when a debilitating condition kept her inside from recess and home from school. “I would get these intense headaches focused on the left side like a sledgehammer was banging on my head,” she said. Read More
At 26 years old, Karen Jury stood before a class of elementary students as her right arm tingled before falling completely numb. That led to a conversation with her doctor. Years of migraine headaches and a recurring sensation of shock waves throughout her body, simply from the turn of her head, resulted in a scheduled spinal tap and an MRI. She received a diagnosis of Arnold-Chiari malformation, a structural defect in the base of the skull and cerebellum. Read More
Millions of people are affected by blood disorders, and the prevalence is expected to grow as our population ages. It is not surprising that, according to the American Society of Hematology, the FDA approved several new therapies – or new indications for previously approved therapies – in 2019 for people living with non-malignant blood disorders. Those included two disease-modifying treatments for sickle cell disease and the first anticoagulant for venous thromboembolism management in children. Read More
“Lack of knowledge is the true bottleneck to clinical translation. We need to stop telling basic scientists, especially trainees, that their work’s value lies in its translatability.” That is the unexpected advice of none other than William Kaelin Jr., whose scientific discoveries have proved to be both top-rate science and very translatable indeed. His work, for which Kaelin has won the 2019 Nobel Prize in Physiology or Medicine and a host of other awards, has enabled the development of multiple therapies targeting anemia and cancer, including vadadustat. Read More
Filgotinib (GLPG-0634) has the potential to be a blockbuster drug, according to Cortellis Drugs to Watch analysis, but it may take a few approvals in various indications to reach the coveted mark of $1 billion in annual sales. Read More
The less-frequent dosing regimen of Basel, Switzerland-based Novartis AG’s cholesterol therapy, inclisiran, under development in the hands of subsidiary The Medicines Co., positions the small interfering RNA (siRNA) drug to take on marketed proprotein convertase subtilisin/kexin type 9 (PCSK9)-targeting antibodies as well as statins in the busy therapeutic space. Statins are the gold standard now, but about 80% of patients don’t reach their lipid goals.
Inclisiran’s inclusion on the 2020 Cortellis Drugs to Watch list is an example of target discovery possibilities hiding in plain sight – if companies and institutions are willing to put effort into increasing sample diversity in genomic research. Read More
Two of three oncology drugs selected for blockbuster status in the Cortellis Drugs to Watch analysis are antibody-drug conjugates (ADCs), highly targeted cancer therapies designed to leave the healthy cells be and zap the bad ones. What once was a dead end for development has morphed into a competitive space with 57 ADC candidates for cancer indications in phase I or later trials, according to Cortellis. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Acasti, Adverum, Aerpio, Alkahest, Beyondspring, Biohaven, Eli Lilly, Ico, Kodiak, Myovant, Oculis, Oxurion, Regeneron, Rezolute, Roche, Sernova, Vtv. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Angiogenex, Arbutus, Axim, Blue Water Vaccines, Clover, Gibson Oncology, Inmunebio, Intellia, Koutif, KSQ, Novozymes, Oncosec, Phio, Recce, Seurat, Synthetic Biologics, Zealand. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Aptorum, Aquestive, Eagle, Inovio, Kite, Nanobiotix, Trevena, Zai Lab. Read More
