Takeda’s oveporexton improved wakefulness, attention and other key narcolepsy endpoints “with a high degree of statistical significance,” according to Jefferies analysts.
The development saga for the depression molecule has been rocky for years, unable to ease symptoms in multiple late-stage trials.
The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech noting that the regulator found its neurodevelopmental findings for the gene therapy to be “robust.”
Through its recently unveiled Priority Voucher program, the FDA seeks to accelerate the review process for companies that promise to keep prices down.
In its complete response letter, the FDA cited insufficient evidence establish deramiocel’s effectiveness for cardiomyopathy associated with Duchenne muscular dystrophy. The decision comes after CBER Director Vinay Prasad canceled an advisory committee meeting for the therapy.
The approval of Moderna’s Spikevax for kids at higher risk of contracting the disease continues the company’s regulatory winning streak, which has also included nods for a next-gen COVID-19 vaccine and an RSV shot.
FEATURED STORIES
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 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.
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.
It’s time for Congress to step up and fund America’s supply chain independence from Chinese companies by bolstering our domestic manufacturing capabilities.
LATEST PODCASTS
In this episode presented by PII, BioSpace’s head of insights discusses with guests Oliver Eden and Travis Webb how autoinjectors offer opportunities to improve delivery systems, patient compliance and clinical trial processes.
M&A headlined for a second straight week as Genmab acquired Merus for $8 billion; Pfizer strikes most-favored-nation deal with White House; CDER Director George Tidmarsh caused a stir with a now-deleted LinkedIn post; GSK CEO Emma Walmsley will step down from her role; and uniQure’s gene therapy offers new hope for patients with Huntington’s disease.
The FDA is hoping to repurpose GSK’s Wellcovorin for cerebral folate deficiency; Pfizer acquired fast-moving weight-loss startup Metsera for nearly $5 billion after suffering a hat trick of R&D failures; psychedelics are primed for M&A action and Eli Lilly may be next in line; RFK Jr.’s revamped CDC advisory committee met last week with confounding results; and Stealth secured its Barth approval.
Job Trends
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.
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SPECIAL EDITIONS
In this deep dive, BioSpace explores the next big thing in obesity.
BioSpace did a deep dive into biopharma female executives who navigated difficult markets to lead their companies to high-value exits.
BioSpace data show biopharma professionals faced increased competition for fewer employment opportunities during the second quarter of 2025, with increased pressure from further layoffs.
DEALS
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BioSpace and guests from Halia Therapeutics, Triumvira Immunologics and the Alzheimer’s Drug Discovery Foundation will discuss alternative financing strategies to consider for 2024. Watch now.
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The companies have received all required regulatory approvals to complete the deal, the largest for the sector in the past three years and the biggest for the hot antibody-drug conjugate market.
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With the acquisition, AstraZeneca will gain access to Icosavax’s investigational combination vaccine IVX-A12, which is being developed for respiratory syncytial virus and human metapneumovirus in older adults.
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Friday’s FDA approval of Vertex-CRISPR’s Casgevy and bluebird bio’s Lyfgenia has immediately revealed startling differences between these two gene therapies: price and a black-box warning.
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The deal, announced late Wednesday, will provide AbbVie with access to Cerevel Therapeutics’ pipeline of clinical-stage and preclinical candidates for psychiatric and neurological diseases.
WEIGHT LOSS
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The FDA currently does not recommend using changes in lean mass as an endpoint in clinical trials for weight loss drugs. Should it?
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Amid shortages of its Mounjaro and Zepbound blockbuster drugs, Eli Lilly on Friday said it is investing an additional $5.3 billion in its production site in Lebanon, Indiana, to boost output of their active ingredient.
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Aiming to compete in the lucrative weight-loss drug market, Zealand Pharma on Thursday posted topline results showing its dapiglutide led to only a slight weight reduction in low doses.
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Full data from the FLOW study show that Novo Nordisk’s top-selling GLP-1 receptor agonist semaglutide can significantly reduce the risk of death by 20% in diabetes patients with chronic kidney disease.
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SixPeaks Bio emerged from stealth Wednesday with up to $110 million, a deal with AstraZeneca and plans to take weight-loss candidates designed to preserve muscle mass toward the clinic.
POLICY
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While some analysts say Donald Trump is a wild card when it comes to drug pricing, many argue his presidency would be more positive for the industry overall, as Kamala Harris has her price-cutting sights squarely on Big Pharma.
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The CDC’s vaccine advisors on Thursday maintained that respiratory syncytial virus immunization is only recommended for adults aged 75 and older, and in seniors 60 to 74 years who are at risk of severe disease.
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The CDC’s Advisory Committee on Immunization Practices on Wednesday backed the use of Merck’s Capvaxive and Pfizer’s Prevnar 20 in adults between 50-64 years of age, opening a bigger market for the respective companies.
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On election day, Tuesday, November 5, Americans will choose between former President Donald Trump and current Vice President Kamala Harris for their next president. The election will also see the rearrangement of Congress.
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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.
In order to play the “cat and mouse” game with poise, we’ve provided a breakdown of the steps to take to negotiate properly. Read on for tips to help you successfully negotiate a job offer.
Once you’ve determined where you want to reside, the next step is hunting for a premier job in your field in that location.
Before you even begin researching, prioritize which aspects of the job offer are most important to you.
First, prioritize these criteria according to your own desires and needs. Which aspects of the offer do you most value?
When you’re ready for a new job, contacting employers is a no-brainer. But the same kind of clear-cut call to action doesn’t necessarily apply to contacting recruiters.
As you enter the job market (or a new industry) the salary range of positions you’re qualified for is likely top of mind. But how do you figure out what a realistic range is for what you’re after?
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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The pharma continued its clinical losing streak on Thursday with the announcement that it is discontinuing late-stage studies of the anti-PD-1 therapy in non-small cell lung cancer and cutaneous squamous cell carcinoma.
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With the FDA’s rejection of Ordspono in March, Monday’s green light from the European Commission marks the first approval worldwide and the first regulatory victory for Regeneron’s bispecific antibody platform.
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The Oncologic Drugs Advisory Committee in a Sept. 26 meeting will discuss whether the regulator should restrict approval of checkpoint inhibitors based on PD-L1 expression levels.
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The regulator’s Complete Response Letter flagged problems at a third-party fill/finish site, which Regeneron says it has already addressed. The manufacturing facility is currently awaiting reinspection.
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Johnson & Johnson announced Tuesday that the FDA has approved Rybrevant with Lazcluze as a first-line chemotherapy-free treatment for patients with EGFR-mutated non-small cell lung cancer.
NEUROSCIENCE
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An undisclosed once-daily oral dose of Neurocrine Biosciences’ Takeda-partnered investigational drug significantly reduced symptom severity in adults with major depressive disorder.
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As the Phase III amyotrophic lateral sclerosis pipeline thins out, the ALS community is placing its hopes on earlier-stage trials sponsored by Denali Therapeutics, PTC Therapeutics and more.
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As AbbVie and NeuroDerm race toward potential U.S. approvals later this year, the companies presented strong data at last week’s American Academy of Neurology 2024 annual meeting for their respective continuous subcutaneous levodopa infusions.
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A one-time treatment for Parkinson’s disease could be a ‘market changer,’ experts told BioSpace, adding that cell therapies could limit the adverse effects seen with current drugs.
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The FDA will close out April with five target action dates around indications that include pediatric seizures and a neurological cancer in children.
CELL AND GENE THERAPY
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Cell and gene therapy professionals gathered in Miami last month to discuss new manufacturing approaches for these up-and-coming treatments.
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Johnson & Johnson and Legend Biotech got a positive opinion from a European Medicines Agency panel for earlier lines of treatment, as they ready for a March FDA advisory committee meeting.
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After treatment with a CD19 CAR-T therapy, patients with systemic lupus erythematosus, idiopathic inflammatory myositis or systemic sclerosis achieved long-lasting remission, according to results published in the NEJM.
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The regulator has granted a priority review of the efficacy supplement for Sarepta Therapeutics’ gene therapy Elevidys with a target decision date of June 21, 2024.
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Kelonia Therapeutics’ in vivo gene placement system is being tapped to help Astellas Pharma expand its portfolio of in vivo CAR-T cell therapies for cancer.