The condition, recessive dystrophic epidermolysis bullosa, causes chronic wounds and has an 84% mortality rate by age 40.
Vas Narasimhan said on Tuesday that if the U.S. adopts international drug pricing, all companies would have to “relook at their medium- to long-term outlook.”
Following the recent discontinuations of assets in Alzheimer’s and migraine, AstraZeneca is stepping away from neuro altogether.
While Vaxart can proceed with its study, fellow COVID-19 vaccine developers GeoVax and Novavax continue to be hamstrung by various regulatory roadblocks.
Biohaven will use the money to bankroll commercial preparations for the spinocerebellar ataxia drug candidate troriluzole, which is currently under FDA review with a decision expected in the third quarter.
The company’s $3 billion in earnings for the first quarter missed on both top and bottom line, according to BMO Capital Markets. Adding insult to injury, the FDA declined to approve a prefilled syringe of its ophthalmology cornerstone.
FEATURED STORIES
Konstantina Katcheves, Senior VP of Innovative Global Business Development at Teva Pharmaceuticals brings insights from the World Economic Forum to SCOPE 2025.
Merck’s Keytruda holds on to the top spot while AbbVie’s Humira—once the world’s top-selling drug—continues to cede its market share to biosimilar competitors.
Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple effect is already being felt across biopharma.
FROM BIOSPACE INSIGHTS
In a year when eradicated diseases are on the uptick in America, how will American children survive RFK Jr.’s vaccine scrutiny and inconsistency? Two experts call on pharma and regulatory bodies to rebuild trust.
LATEST PODCASTS
BioSpace’s Lori Ellis and Chantal Dresner bring live updates from day three of #DIA2024 in San Diego.
This week on Denatured, Head of Insights Lori Ellis and guests discuss the persisting challenges of diversity, equity and inclusion when designing clinical trials.
This week’s news ranged from BioSpace’s on-the-ground updates from DIA to safety concerns in clinical trials to BIOSECURE Act updates to new projections that the GLP-1 market could top $100 billion within 10 years.
Job Trends
Merck KGaA, Darmstadt, Germany, a leading science and technology company that operates its Healthcare business as EMD Serono in the US and Canada, today shared updates on the company’s oncology pipeline and focused approach to the research and development of potential new medicines designed to improve the futures of people with cancer.
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SPECIAL EDITIONS
A new generation of checkpoint inhibitors is emerging, with some showing more promise than others. From recent TIGIT failures to high-potential targets like VEGF, BioSpace explores what’s on the horizon in immuno-oncology.
Peter Marks, the venerable head of the FDA’s Center for Biologics Evaluation and Research, has been forced out. In this special edition of BioPharm Executive, BioSpace takes a deep dive into the instability of the HHS.
Year-over-year BioSpace data show biopharma professionals faced increased competition for fewer employment opportunities during the first quarter of 2025.
DEALS
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The acquisition of the contract development and manufacturing organization will allow Agilent Technologies to provide a one-stop source for gene-editing services for its customers.
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Oral doses of SIGA Technologies’ antiviral drug Tpoxx will help the U.S. maintain its reserves of the vaccine in preparation for future potential outbreaks, according to the company.
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After completing a buyout transaction with The Column Group to remove it from the stock exchange, NGM Bio has raised a $122M Series A to fund a registrational study for a rare liver disease drug and a Phase II trial in hyperemesis gravidarum.
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Asceneuron, which develops small molecules targeting tau protein aggregation, plans to use the funds to advance its Alzheimer’s disease asset into Phase II.
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Akebia Therapeutics on Thursday said it regained full U.S. rights to its chronic kidney disease anemia drug Vafseo, which the biotech has priced at around $15,500 per year.
WEIGHT LOSS
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Monday’s lawsuits from Eli Lilly are the first to be filed by the pharma since the regulator officially removed tirzepatide from its drug shortage database earlier this month.
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This week marked the start of the third-quarter earnings season, with Johnson & Johnson exceeding Wall Street’s expectations. Pfizer is projected to have a strong quarter, while Eli Lilly could pull ahead of Novo Nordisk in the obesity space. Moderna, by contrast, has a decidedly negative outlook.
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Pressure has been mounting for the Federal Trade Commission to take action, with Senator Elizabeth Warren last week urging FTC Chair Lina Khan to block the merger if it violates antitrust laws.
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While the regulator conducts another review into the supply of Eli Lilly’s tirzepatide, compounders will be able to continue selling their own remixed versions of the blockbuster drug.
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Senator Elizabeth Warren told the Federal Trade Commission that the acquisition of contract manufacturer Catalent could increase Novo’s dominance over the hot GLP-1 market, reducing competition and increasing prices.
POLICY
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Drugmakers will have until the end of February to decide whether they want to participate in the second round of Medicare negotiations or not. CMS has until June 1 to send an initial offer for the adjusted prices.
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Novartis is locked in a legal back-and-forth with MSN Pharma over alleged patent infringement of its heart failure drug Entresto.
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Along with its gene editing therapy Casgevy, Vertex is offering fertility preservation support for its patients—a program that the HHS claims violates anti-kickback statutes.
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A report published Tuesday shows hundreds and thousands of percent markups on HIV, hypertension and cancer drugs for Medicare and commercial claims alike.
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Concurrently, a preprint from the industry-backed Vital Transformation found a 50% drop in company investments into small-molecule drug development.
Beginning the job search is the first step toward starting your career after graduating with a Bachelor’s Degree in Chemistry. To help, we’ve compiled seven of the most common chemistry jobs for new grads.
BioSpace spoke with Saundra Pelletier, CEO of Evofem Biosciences, to discuss the realities of being a woman in the life sciences and find out the career moves that propelled her forward.
Becoming a chemist in the biopharma industry takes hard work, dedication and a willingness to continuously learn. Here’s what you need to know to become a biochemist.
As automation ramps up in the coming years, many jobs will be lost to machines. Here are a few life science sectors that are expected to maintain steady growth in the coming years.
Landing your first internship can be a major milestone in your career journey. Here’s what you can do to set yourself up for success and make the most of your life science internship.
The best thing a professional can do when considering a position at the FDA is weigh the pros and cons. To help, we’ve created a guide to working at the FDA, with benefits, challenges and tips.
HOTBEDS
REPORTS
In this Employment Outlook report, BioSpace explores current workforce sentiment, job activity trends and the prospective job and hiring outlook for 2025, particularly as it compares to the previous year.
BioSpace’s third report on diversity, equity, inclusion and belonging in life sciences examines dramatic shifts in attitude around diversity initiatives.
CANCER
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First approved in 2021, Jemperli has now become a cornerstone of GSK’s cancer business, earning more than $160 million in the third quarter.
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Both vibostolimab and favezelimab have had disappointing runs leading up to their termination, sustaining several late-stage failures.
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The FDA cited issues with a manufacturing facility as the reason for the rejection. J&J is currently “working closely” with the regulator to resolve these problems.
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Bristol Myers Squibb aims to generate around $1.5 billion in savings through 2025—a goal that it hopes to reach by lowering third-party expenditures, focusing only on key growth brands and cutting some 2,200 jobs by year-end.
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Some 90% of investigational drugs fail—and success rates are even more dire in the neuro space. Here, BioSpace looks at five clinical trial flops that stole headlines over the past 12 months.
NEUROSCIENCE
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This year has seen several biopharma companies drop Alzheimer’s and Parkinson’s disease programs, but experts say plenty are still chasing these multi-billion-dollar markets.
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Bristol Myers Squibb’s third-quarter results benefited from sales of its legacy brands Eliquis and Revlimid, as well as growth portfolio products such as Abecma, Breyanzi and Reblozyl.
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In another delay for the psychedelic treatment space, Compass Pathways announced adjusted timelines for two Phase III trials of its psilocybin to address treatment-resistant depression.
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Biogen touted strong Q3 sales of its Alzheimer’s drug Leqembi a day after announcing a deal worth up to $1.45 billion with Neomorph to discover and develop molecular glue degraders.
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On its third-quarter earnings call Tuesday, Sage highlighted the launch of its Biogen-partnered postpartum depression drug Zurzuvae but said it will stop pursuing approval for major depressive disorder, which the FDA previously denied.
CELL AND GENE THERAPY
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The intellectual property landscape for newer gene-editing technologies, like that for CRISPR-Cas9, remains unclear and hard to navigate.
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With promising early results, cell and gene therapies are making headway against both rare and common ocular and auditory diseases.
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With gene therapies by REGENXBIO and AbbVie, Adverum and others in mid- or late-stage trials, this therapeutic class could soon be an option for this common cause of blindness in the elderly.
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Quotient Therapeutics’ platform targets somatic mutations, which the startup contends can help identify a broader scope of genes potentially associated with disease phenotypes. Wednesday’s agreement is part of an existing partnership between Pfizer and Flagship Pioneering.
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As the biotech implements a more focused strategy for its Roctavian hemophilia A gene therapy, BioMarin has recruited two seasoned pharma executives to bolster its C-suite.