Brazilian authorities said the death was unlikely to have been caused by Elevidys and was instead more in line with severe infection exacerbated by immunosuppression.
Despite the failure of its Recognify-partnered inidascamine, Jefferies analysts do not expect a definitively negative stock impact on atai, given the company’s promising psychedelic pipeline.
The Commissioner’s National Priority Vouchers aim to offer accelerated pathways to drugs that meet certain criteria, perhaps including a low price-tag. But the policy is vaguely defined and was announced without public input, going against the FDA’s own published practices, experts say.
The collaboration focuses on ‘molecular gates,’ a class of molecules that the startup company Gate Bioscience says can stop pathogenic proteins from leaving the cell.
The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne muscular dystrophy.
The strategic reprioritization comes after the company hit two major hurdles in the past year, including a clinical hold for an investigational gene therapy and an FDA rejection for its lead asset.
FEATURED STORIES
BioSpace Senior Editor Annalee Armstrong headed to the J.P. Morgan Healthcare Conference with a months-long story idea brewing. Unfortunately, it was one she’s written before.
Of the 102 company launches or series A financings since October 2023, just nine had a woman at the helm, according to a BioSpace analysis. This is happening in an era of biotech where new company founders are searching for CEOs with a track record.
At the J.P. Morgan Healthcare Conference, the Biotech CEO Sisterhood assembled in Union Square to showcase the large group of women and allies in biopharma as their authentic selves.
Faced with the encroaching threats of patent expirations and generics, biopharma companies in 2024 invested 33% more in licensing deals, on average, than in 2023 with an eye toward enriching their pipelines with novel and potentially more effective therapies.
AbbVie launched a revamped version of its Allē loyalty program, which ultimately was not adopted by providers. The marketing misstep comes as the company’s aesthetics franchise faces broader pressures.
Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio—have products in the pipeline that could improve treatment options for this rare disease.
FROM BIOSPACE INSIGHTS
Establishing trust through thought leadership is no longer optional in today’s cautious biopharma market. This webinar will show leaders how strategic insights and targeted outreach can turn awareness into high-converting leads. Watch now.
LATEST PODCASTS
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.
BioSpace’s Lori Ellis and Chantal Dresner bring live updates from the first day of #DIA2024 in San Diego.
This week, an FDA adcomm unanimously voted for the approval of Eli Lilly’s anti-amyloid antibody as thousands of layoffs continue to rattle the industry.
Job Trends
AstraZeneca and Alexion, AstraZeneca Rare Disease, will showcase 14 studies, including real-world evidence (RWE), from their portfolio and pipeline of investigational amyloidosis therapies at the International Symposium on Amyloidosis (ISA), in Rochester, MN from May 26–30, 2024.
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SPECIAL EDITIONS
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.
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.
DEALS
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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. -
Asceneuron, which develops small molecules targeting tau protein aggregation, plans to use the funds to advance its Alzheimer’s disease asset into Phase II. -
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. -
In its second antibody-drug conjugate licensing agreement this year, Ipsen has secured exclusive rights to Foreseen Biotechnology’s FS001, which targets a novel antigen expressed across a range of solid tumors. -
Eli Lilly becomes the latest to make a major investment in immunology and inflammation, while antibody-drug conjugate biopharma Myricx Bio nets a large Series A round and new research highlights the potential and possible risks of GLP-1s.
WEIGHT LOSS
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Novo Nordisk’s total revenue in the third quarter missed analysts’ expectations but sales of weight loss drug Wegovy exceeded the consensus forecast. Still, the Danish drugmaker narrowed its full-year guidance for revenue and operating profit. -
While expected and seen as largely incremental, Jefferies analyst Peter Welford in a Tuesday note to investors said the detailed data for three early-stage assets support moving them into Phase IIb studies and creates a “foothold” for AstraZeneca in the weight loss space. -
Jefferies analyst Roger Song in an investor note said that Viking Therapeutics’ readout for its investigational therapy VK2735 exceeded expectations, with “class-leading” weight loss. Patients on 100-miligram doses of the pill lost 8.2% of their body weight after 28 days. -
In part 1 of the pivotal ESSENCE trial, Novo Nordisk’s weight loss drug Wegovy demonstrated “statistically significant and superior improvement” in liver fibrosis in patients with metabolic dysfunction–associated steatohepatitis. -
Lilly CEO Dave Ricks in Wednesday’s third-quarter earnings call acknowledged that the company is at the mercy of wholesaler stocking decisions.
POLICY
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Analysts suggested that the tariffs will have little effect on reshoring manufacturing and will likely decrease patient drug access and increase costs. -
The National Security Commission on Emerging Biotechnology recommends at least a $15 billion investment into biotech over the next five years. -
Jazz is being accused of anti-competitive practices regarding its narcolepsy drug, as generic competitors emerge on the market. -
Billions in market cap are being shed as the markets reel over President Donald Trump’s escalating trade war. Eli Lilly’s value has dropped more than $95 billion in just one month. -
Last week, The Trump administration reversed a Biden-era proposal for Medicare coverage of anti-obesity treatments. But on Monday, HHS suggested it is open to future policy considerations toward this end.
Discover the most common departments that make up a BioPharma company, learn about the responsibilities of each department and figure out which role is the right fit for you.
Like any field, there are upsides and downsides to working in biotech, and educating yourself can help you make an informed decision if you’re considering a career in this industry.
Learn the best tips, tricks, and practices to help yourself to get the first job after college life sciences industry right out of college in our comprehensive guide.
Discover the most in-demand pharma qualifications and how to use them to score your dream job in our comprehensive guide.
Instead of going through pharma job search websites, you should create a powerful LinkedIn profile. Check out Biospace to find out the benefits of having a strong LinkedIn Profile.
Despite the massive financing from venture capitalists that poured into Massachusetts in 2021, Bay State biopharma companies are struggling to find enough talent to fill the number of available jobs.
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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Datroway, formerly known as Dato-DXd, significantly improved median progression-free survival in a Phase III study but failed to do so for overall survival. -
Even before the FDA’s recent approval of Dato-DXd in breast cancer, analysts predicted sales of the antibody-drug conjugate could hit $5.9 billion in 2030. However, the asset faced a series of setbacks in 2024. -
Traditionally carrying a dire prognosis, the treatment paradigm for multiple myeloma is changing, with CAR T therapies, bispecifics and more contributing to multifaceted regimens unique to each patient’s needs. -
The conversion of Calquence’s accelerated approval in mantle cell lymphoma comes a day before the drug was listed among the 15 products to be subject to IRA-prescripted price negotiations for Medicare this year. -
The Phase III CodeBreaK 300 study returned disappointing overall survival data for Lumakras plus Vectibix in metastatic colorectal cancer, but in its approval announcement, the FDA pointed to significant improvements in progression-free survival, calling it the “major efficacy outcome” of the trial.
NEUROSCIENCE
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Despite hotly debated biomarkers and failed or delayed confirmatory trials, the accelerated approval program has a track record of propelling R&D for some of medicine’s most challenging illnesses. -
Emboldened by technological advances and a deeper knowledge of glioblastoma, Merck, Kazia Therapeutics, CorriXR Therapeutics and others are targeting the often-fatal brain tumor. -
Despite the “unfortunate” failure, William Blair analysts do not believe that the utreloxastat readout will heavily affect PTC, instead postulating that the upcoming FDA decision on its phenylketonuria candidate sepiapterin will be a stronger driver of the biotech’s stock. -
Alector is kicking off a resource realignment effort that will include a workforce reduction. The biotech expects its current cash position to last it through 2026. -
While taldefgrobep alfa failed to show improved motor function in spinal muscular atrophy, treated patients saw a marked reduction in body fat. Biohaven plans to launch a Phase II trial in obesity by the end of the year.
CELL AND GENE THERAPY
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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. -
As it nears a crucial FDA action date for its transthyretin amyloid cardiomyopathy candidate, BridgeBio focuses on its late-stage pipeline. -
No patients have received Casgevy, CRISPR Therapeutics and Vertex Pharmeceuticals’ recently approved sickle cell gene therapy. Experts weigh in on the path to profit for the treatment and the therapeutic class in general. -
BioMarin executives sought to calm an anxious investor base Wednesday with a public address and pledge to achieve a nearly 50% bump in annual revenue by 2027. But analysts were left wanting. -
Proceeds from the oversubscribed financing will be used to advance ArsenalBio’s lead programs, which include a handful of solid tumor cell therapy candidates.
