Analysts at Jefferies see blockbuster potential in zorevunersen in Dravet syndrome, with sales potentially reaching $1 billion to $4 billion.
Commissioner Marty Makary said that the FDA will soon start requiring only one pivotal trial, instead of two, for companies seeking approval for new drugs.
Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 for Pompe disease.
Praxis Precision Medicines has also announced a “successful” pre-NDA meeting with the FDA for its essential tremor drug candidate ulixacaltamide, for which an approval application is slated for early 2026.
The ACIP voted 8-3 to recommend delaying the hepatitis B vaccine, commonly given just after birth. The CDC itself has said the shot is safe and effective.
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s Phase I/II studies of AMT-130 are “unlikely” to provide the primary evidence to support a biologics license application.
FEATURED STORIES
Novo Nordisk goes “on the offensive” following Trump deal that also included rival Eli Lilly, putting an exclamation point on rapidly declining GLP-1 drug prices. Experts say the unusual situation makes it hard to predict what’s next.
Drug candidates don’t usually move among Big Pharma, but these five biotechs helped facilitate such hand-offs, scooping up assets from one pharma on the cheap before being bought out for billions by another.
A week into his tenure as head of the FDA’s Center for Drug Evaluation and Research, experts agree that Rick Pazdur is the “ideal fit” to stabilize the agency. And, according to one ex-FDA official, if his CBER counterpart Vinay Prasad tries to supersede Pazdur’s authority, “there will be hell to pay.”
The introduction of AbbVie’s hepatitis C drugs in 2014 forced Gilead’s hand in the fight for market dominance in hepatitis C. A similar dynamic is now playing out between Eli Lilly and Novo Nordisk in the obesity space, with some key differences.
The industry’s ability to generate a return on billions of dollars of investment rests on a heavily regulated supply chain defined by time-pressured logistics.
Priority voucher awardees and regulators could feel pressure to “meet the moment” as FDA watchers question the intent and feasibility of the Commissioner’s National Priority Voucher program.
LATEST PODCASTS
Johnson & Johnson has yet to make a drug pricing deal with Trump; Novo makes more moves under new CEO; more than 1,000 laid off from CDC, though many immediately hired back; the BIOSECURE Act is back and more.
In this bonus episode, BioSpace’s Vice President of Marketing Chantal Dresner and Careers Editor Angela Gabriel take a look at Q3 job market performance, layoffs and wider employment trends and policies impacting the biopharma workforce.
The U.S. government remains shut down, with the FDA closed for new drug applications until further notice; cell and gene therapy leaders gather for the annual meeting in Phoenix with the field in a state of flux; Pfizer and Amgen will make drugs available at a discount as President Donald Trump’s tariffs still loom; and new regulatory documents show how Pfizer beat out the competition for Metsera.
Job Trends
Looking for a biopharma job in Illinois? Check out the BioSpace list of six companies hiring life sciences professionals like you.
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SPECIAL EDITIONS
In this deep dive BioSpace dissects the global obesity and diabetes markets along with the growing pipelines that aim to serve them.
For the second quarter of 2024, there were 25% fewer jobs posted live on BioSpace compared to the same quarter of 2023. The year-over-year job response rate rose from 14.6% to 15.3%.
The pace of mergers and acquisitions has accelerated. In this deep dive, BioSpace takes a closer look at the nature of recent deals and the players involved.
DEALS
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Gilead is actively looking for late-stage and de-risked assets for potential deals across various therapeutic spaces, including liver disease, cancer and immunology. -
Pfizer called Novo’s offer “reckless and unprecedented,” in a statement issued Thursday morning. -
Mergers and acquisitions are not just for Big Pharma. A new report from Leerink Partners takes a stab at identifying the small- to mid-cap pharmas best prepared to bolster their pipelines with a buyout. -
Vas Narasimhan confirmed that Novartis is having weekly discussions with the Trump administration on drug pricing, but a deal has not yet been reached. -
The deal, announced early Sunday afternoon, will see Novartis gain access to Avidity’s neuroscience assets, while the San Diego biotech spins out a new company to shepherd its early-stage precision cardiology programs.
WEIGHT LOSS
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The agreement will also secure a $150 price for future weight loss pills from Novo Nordisk and Eli Lilly—at least initially. -
The highest dose of Eli Lilly’s eloralintide led to 20.1% weight loss after 48 weeks in a Phase II trial, exceeding analyst expectations and highlighting a “potentially best in class profile,” according to BMO Capital Markets. -
In an investor call Thursday, AstraZeneca CEO Pascal Soriot outlined his company’s approach to obesity drug development based on “the medical aspect” of weight loss, including driving down visceral fat. -
The court’s decision allows Metsera to walk away from its previous acquisition agreement with Pfizer in favor of Novo Nordisk’s higher bid. -
Pressure from the FTC and Pfizer is building around Novo Nordisk’s bid to snatch obesity biotech Metsera. The antitrust watchdog has spotted issues with the structure of Novo’s bid, while its New York-based rival has put more money on the table.
POLICY
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The CDC’s Advisory Committee on Immunization Practices was scheduled to convene Oct. 22 to 23, but this meeting has been postponed, with no new date specified. The delay comes as the VA published new research showing that COVID-19 shots prevented hospitalizations and death. -
The $48 million award, granted through the Advanced Research Projects Agency for Health, will help Kernal take its in vivo mRNA-encoded CAR T therapy forward. -
Cell and gene therapy experts question where the FDA designation fits in an environment that features a range of intersecting regulatory perks. -
A new group of CDC advisors voted last month to separate the chickenpox vaccine from the measles, mumps, rubella components of the MMRV shot due to concerns over febrile seizures, while recommending a more risk-based approach to COVID-19 immunizations that mirrors recent FDA approvals. -
Jeanne Marrazzo, former director of the National Institutes of Allergy and Infectious Disease, was formally terminated Thursday after months on administrative leave, after filing a whistleblower report.
As market values increase for computational biology and data science, biopharma companies are looking to hire R&D professionals in those areas. A biotech talent acquisition expert shares his insights on these in-demand roles.
Gratitude, a key part of stoicism, can benefit those working in—and being served by—the pharmaceutical industry.
Businessman and entrepreneur Mark Cuban recently discussed leadership with Leadership Lab columnist Michael Pietrack. The three lessons that came out of that conversation start with one word: caring.
When hiring job candidates to work on cell and gene therapies, companies look for more than just technical skills. Talent acquisition executives from Bristol Myers Squibb and Intellia Therapeutics offer an inside look at what they want in an employee.
Learn how to leverage your end-of-year downtime to document achievements, update your professional presence and prepare for a successful 2025.
Based on how President-elect Donald Trump’s first administration handled immigration, experts are concerned about how his second term will impact foreign-born biopharma professionals. Two immigration attorneys discuss what may be ahead, including increased difficulty getting work visas.
HOTBEDS
REPORTS
BioSpace’s Employment Outlook report investigates anticipated job search activity and hiring outlook in 2024 as well as how the current workforce is currently faring
If it feels like there has never been a tougher time to look for work, you’re not alone—and you’re likely not wrong.
In this job market report we’re reviewing life sciences job market movement in Q3 and what to expect for Q4 and beyond.
CANCER
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For $1.2 billion upfront and up to $10.2 billion in milestones, Takeda will gain access to a bispecific antibody fusion protein targeting both the PD-1 and IL-2 pathways, among other assets. -
Novo Nordisk’s leadership gets another shake-up as President Trump promises to significantly slash prices for its GLP-1 drugs; Summit/Akeso, Exelixis and more present new data at ESMO 2025; Replimune pops as FDA accepts resubmitted BLA; FDA names first winners of Commissioner’s National Priority Voucher program; and more. -
With data from the Phase III STELLAR-303 study in the books, Exelixis is plotting a 2025 regulatory application for zanzalintinib. -
In May, Summit released early data from the Phase III HARMONi study showing that while the PD-1/VEGF inhibitor resulted in significant progression-free survival improvements, it fell short of the overall survival bar. -
To tailor cancer therapies to individual patients, Moderna, BioNTech and other companies are rethinking how they optimize manufacturing schedules and resources.
NEUROSCIENCE
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Manifold will use its tissue-targeting shuttle technology to help Roche develop new therapeutics for diseases of the central nervous system. -
A month after sparking optimism for patients with Huntington’s disease with highly positive data for AMT-130, uniQure revealed Monday that the FDA may be changing its tune on the evidence required for an approval application, a change of direction Stifel called “very challenging” for uniQure. -
Bristol Myers Squibb beat analyst and consensus estimates for the third quarter with $12.2 billion in sales, but executives on the company’s investor call faced questions about a sluggish uptake for schizophrenia drug Cobenfy as well as a highly anticipated Alzheimer’s psychosis readout for the product. -
To expand the population for the anti-amyloid Alzheimer’s drugs, Lilly and Biogen are testing presymptomatic patients. Will doctors be open to this paradigm-shifting change? -
New interim data from a Phase III trial puts the company on track to file for FDA approval next year in an indication that not only lacks a disease-modifying treatment but suffered significant setbacks after a patient died in a clinical trial for Sarepta’s investigational gene therapy.
CELL AND GENE THERAPY
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Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene therapy: its limb-girdle muscular dystrophy candidate. But the treatment’s path forward, analysts say, is highly uncertain. -
After the FDA revoked Sarepta Therapeutics’ platform designation in July, Krystal Biotech is “poised to be the first major beneficiary” of this program, according to analysts at William Blair. -
Johnson & Johnson has yet to make a drug pricing deal with Trump; Novo makes more moves under new CEO; more than 1,000 laid off from CDC, though many immediately hired back; the BIOSECURE Act is back and more.
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The startup, launched out of CEO Kevin Parker’s grad school idyll during the COVID lockdowns, is primed to find new targets where Big Pharmas won’t dare. -
Regeneron is aiming to file a regulatory application for DB-OTO by the end of the year.
