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
Summit Therapeutics’ ivonescimab has the potential to challenge Merck’s blockbuster checkpoint inhibitor in non-small cell lung cancer, but experts stress the need for diverse and overall survival data.
The release of early-stage data on three oral weight loss drug candidates hints at which companies have the strongest hand, but the comparisons are rife with confounding variables, leaving analysts unsure about where to place their bets.
Last month, Vertex said sickle cell patients had not yet received infusions of its gene therapy Casgevy. That’s now changed, as the company races with bluebird bio’s Lyfgenia.
Launched in 2020 to more quickly bring to market an effective medicine for amyotrophic lateral sclerosis, the HEALEY Platform Trial has generated disappointing results for many but also continuing programs from Clene and Prilenia.
The FDA has six target action dates ahead to round out September as drugs for gastroparesis, Niemann-Pick disease type C and more await decisions.
The potential of mRNA vaccines was established during the COVID-19 pandemic. Now, a new wave of candidates could soon hit the market for cancer, influenza and more.
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
On this episode of the Weekly: Biden administration puts pressure on the biopharma industry; renewed interest in psychedelics after MindMed announces LSD-based candidate meets primary endpoint; bluebird changes its tune.
How can startup leaders support long-term sustainable growth and investment? BioSpace’s Lori Ellis speaks with venture capital guests Ansbert Gadicke, Martin Gershon and Mike Goguen for their advice and recommendations on how biopharma startups should approach funding.
What needs to happen for funding in biopharma to bounce back? BioSpace’s Lori Ellis discusses the macroeconomic environment and biopharma funding outlook with venture capital guests Ansbert Gadicke, Martin Gershon and Mike Goguen.
Job Trends
Positive high-level results from an interim analysis of the ECHO Phase III trial showed AstraZeneca’s CALQUENCE® in combination with standard-of-care chemoimmunotherapy, bendamustine and rituximab, demonstrated a statistically significant and clinically meaningful improvement in progression-free survival versus standard of care in previously untreated adult patients with mantle cell lymphoma.
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SPECIAL EDITIONS
BioSpace did a deep dive into executive pay, examining the highest compensation packages, pay ratios and golden parachutes—what a CEO would get paid to leave.
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.
DEALS
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Monday’s announced buyout of Virginia-based Landos Biopharma adds a mid-stage, oral NLRX1 agonist for ulcerative colitis and Crohn’s disease to AbbVie’s growing portfolio. -
Just weeks after Wegovy won FDA approval for cardiovascular disease, Novo Nordisk has bought mid-stage biotech Cardior Pharmaceuticals and its miRNA-targeting candidate for heart failure. -
The contract manufacturer plans to expand its U.S. footprint with a former Roche facility in Vacaville, California, which Lonza contends is one of the largest biologics manufacturing facilities in the world by volume. -
Following in the footsteps of Bristol Myers Squibb and Eli Lilly, AstraZeneca on Tuesday jumped into the radiopharmaceuticals space by acquiring Fusion Pharmaceuticals in a deal worth $2.4 billion. -
Contineum Therapeutics joined the 2024 initial public offering class on Friday with an SEC filing. The biotech will use the IPO proceeds to complete a Phase II trial for its most mature candidate targeting multiple sclerosis.
WEIGHT LOSS
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Roche’s oral GLP-1 receptor agonist CT-996—obtained in the $2.7 billion acquisition of Carmot Therapeutics—reduced body weight by more than 6% at four weeks versus placebo in a Phase I trial. -
Pfizer selects its candidate for the oral GLP-1 race as Eli Lilly strives to overtake Novo Nordisk in the injectable weight-loss drug space. Meanwhile, pressure builds to reduce drug prices in the U.S. -
To help keep pace with the demand for GLP-1 therapies, CordenPharma has announced a sizeable $980 million investment in its U.S. and European sites. -
Some analysts say so, and a recent study suggested Lilly’s tirzepatide beat Novo’s semaglutide at inducing weight loss, but there are other factors in the market race. -
Despite recent concerns about suicidality and other neuropsychiatric issues, a recent study has found that Novo Nordisk’s Ozempic (semaglutide) is associated with lower risks of dementia, cognitive deficit and nicotine misuse.
POLICY
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While unlikely to pass this year, given the Democrats’ control over the White House and Senate, the proposed legislation might be a harbinger of the Republicans’ agenda next year for the Inflation Reduction Act should they win the November elections. -
In the battle over drug prices, one sector of the healthcare industry has risen above all the players as the boogeyman: pharmacy benefit managers. In this special edition of BioPharm Executive, BioSpace takes a deep dive into the lens now focused on PBMs’ business practices. -
Ahead of a Senate health committee hearing next week with Novo Nordisk CEO Lars Jørgensen, Sen. Bernie Sanders claims he has secured assurances from generics developers that they will charge a significantly lower monthly price than the $969 Americans currently pay for the Danish drugmaker’s diabetes blockbuster. -
If Johnson & Johnson refuses to scrap its proposed changes to the 340B drug pricing for hospitals, it risks the termination of participation in the program and monetary fines, the Health Resources and Services Administration warned. -
It’s time for Congress to step up and fund America’s supply chain independence from Chinese companies by bolstering our domestic manufacturing capabilities.
Discover the pros and cons before you hire an independent contractor (IC). These include freelancers, consultants, seasonal workers, and other temporary professionals.
Getting job-hunting and career advice is hard, especially when you get it from those who have worked for decades. Some advice is still helpful today, like making a good impression, but others are considered counter-productive.
The salary expectation question is one of the most difficult questions asked during a job interview. Here’s how you can answer the question perfectly.
The coronavirus disease (COVID-19) pandemic that has ravaged across the globe has demanded more experts in microbiology, particularly jobs in virology.
BioSpace’s Pharm Country Hotbed ranges from Connecticut, New York, New Jersey, Pennsylvania and Rhode Island.
“The Great Resignation” is a term that’s been coined to describe the wave of people leaving their jobs for other opportunities or exiting the workforce altogether.
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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Six months after treatment with the radiopharmaceutical therapy, 77.8% of patients with meningioma were alive and had not experienced further disease progression, beating the 26% benchmark established in earlier studies. -
Johnson & Johnson linked Carvykti to a 45% reduction in risk of death and Darzalex to a 61% improvement in minimal residual disease-negativity, boosting the prospects of two key growth drivers for the company. -
One upcoming decision—on a perioperative PD-1 regimen for lung cancer—comes as the FDA considers an overhaul of trial designs in this treatment setting. -
The FDA’s Oncology Drugs Advisory Committee voted near-unanimously that the benefits of PD-1 inhibitors like Keytruda and Opdivo in PD-L1 low patients do not outweigh the risks. -
Merck follows in the footsteps of Bristol Myers Squibb, which in December 2023 also failed to secure a late-stage victory for its combo regimen of a PD-1 blocker and an anti-LAG-3 antibody.
NEUROSCIENCE
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A new observational study from the University of Iowa points to the potential of alpha-blocker drugs, commonly used to treat enlarged prostates, as a preventive therapeutic option for dementia with Lewy bodies. -
The next six months for the FDA are primed to be as groundbreaking as the first six, with Eli Lilly’s donanemab and Lykos Therapeutics’ MDMA-assisted PTSD therapy on the docket, among others. -
Accurately diagnosing Alzheimer’s disease pathologies is becoming increasingly important, but the U.S. is facing imaging resource constraints. -
While Sage Therapeutics’ drug candidate showed a slight difference compared to placebo in a mid-stage Huntington’s disease trial, William Blair analysts in a Tuesday note to investors said they “remain cautious” on dalzanemdor and “do not view the small numerical changes as definitive.” -
Despite concerns raised in FDA briefing documents about Eli Lilly’s Alzheimer’s treatment, donanemab, the committee concluded that the benefits outweighed the risks.
CELL AND GENE THERAPY
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Jeffrey Chamberlain, president of the American Society of Gene & Cell Therapy, spoke with BioSpace about what we can expect to learn about in Baltimore this week. -
Adeno-associated viruses have long been go-to vectors for gene therapies. How AAVs are improving will be among the cell and gene therapy topics to be covered in Baltimore this week. -
Follow News Editor Greg Slabodkin and Managing Editor Jef Akst as they travel with some 8,000 others for discussions of cell and gene therapy advances, challenges, regulations and more. -
Ahead of the FDA’s June target action date for Sarepta’s Duchenne muscular dystrophy gene therapy, ICER Chief Medical Officer David Rind blasted the regulator’s accelerated pathway in a JAMA viewpoint article. -
Astellas Pharma and Poseida Therapeutics have entered into a second CAR-T contract to develop novel and flexible allogeneic cell therapies in oncology.
