The pivotal trial for Neurogene’s Rett syndrome gene therapy makes use of baseline controls and a rigorous endpoint that could help ensure a broader label for the drug product, if approved, according to analysts.
A readout from the company’s SUMMIT trial put its small molecule bezuclastinib on a collision course with rival Blueprint’s Ayvakit, which Leerink analysts said does not sufficiently treat all patients.
The partnership will give Chugai access to Gero’s artificial intelligence technology to discover novel targets in aging-related diseases. Chugai will then develop antibody-based drugs based on the findings.
After a season of regulatory upheaval, obesity and rare genetic diseases will likely remain major themes for biopharma in 2025, according to Jefferies.
Ekterly’s road to approval was not a smooth one. Last month, the FDA informed KalVista it would not meet its PDUFA date due to resource constraints and reports surfaced that Commissioner Marty Makary tried to have the application rejected.
Despite rehiring hundreds of FDA, CDC and NIH employees, the Department of Health and Human Services is still a skeleton of its former self under Health Secretary Robert F. Kennedy Jr.
After the FDA rejection of Zurzuvae in one type of depression and the triple failure of neuro asset dalzanemdor, Sage was searching for a path forward at the end of December 2024. Biogen CEO Chris Viehbacher spied a possible deal, but the smaller company wasn’t interested.
FEATURED STORIES
While the Trump administration has painted the jettisoning of staff and regulations as good for business, there are multiple reasons it’s unlikely to work out that way.
The number of employees laid off increased year over year during the first quarter. BioSpace recaps the five largest rounds of layoffs, which included BMS and Novartis cuts.
To more effectively treat neurodegenerative conditions, we first need diagnostic tools that lend a more complete picture of protein aggregates in the brain.
At the intersection of radiation and precision, Novartis, Bayer, AstraZeneca and more hope to cash in on a radiopharmaceuticals market that could top $16 billion by 2033.
As the Trump administration slashes funding for HIV-related research and infrastructure, Gilead, Immunocore and more are targeting the next goalpost: a cure.
With President Donald Trump expected to deliver a drug pricing order on Monday that Big Pharma and patient groups alike have railed against, the industry’s tumultuous ride is far from over.
FROM BIOSPACE INSIGHTS
BioSpace surveyed industry employers and professionals to understand what to expect from the recruitment market in 2022. What do professionals want? How difficult will it be to recruit new talent?
LATEST PODCASTS
In the third podcast in a special series focused on BioSpace’s NextGen Class of 2025, Senior Editor Annalee Armstrong speaks with Mark McKenna, CEO of Mirador Therapeutics.
In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis and Miruna Sasu, CEO of COTA, discuss the challenges of inclusion and exclusion criteria of clinical trial patients, and reflect on current investment approaches around women’s health.
BioSpace remembers COVID-19 five years after the pandemic was declared, Novo Nordisk’s CagriSema again misses expectations as the company joins a lawsuit filed by drug compounders against the FDA, Viking secures ample supply of its investigational obesity medication, J&J strikes out in depression, and Makary and Bhattacharya near confirmation.
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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
  1. Despite making an unsolicited bid for gene therapy maker bluebird bio, Ayrmid failed to deliver a binding offer after weeks of due diligence. Bluebird’s board recommended that it go with Carlyle and SK Capital Partner’s original offer to take the company private for $30 million.
  2. Merck has not disclosed which of its peptide therapies it plans to develop oral formulations for.
  3. The French pharma is getting Black Diamond’s de-prioritized molecule for non-small cell lung cancers with specific mutations whose development had been paused to save money.
  4. Roche’s up to $1 billion investment will provide access to Oxford BioTherapeutics’ antibody-drug conjugate platform for undisclosed cancer targets.
  5. In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis and Miruna Sasu, CEO of COTA, discuss the challenges of inclusion and exclusion criteria of clinical trial patients, and reflect on current investment approaches around women’s health.
WEIGHT LOSS
  1. Long considered resistant to economic downturns, the pharmaceutical industry may face a greater challenge this time around as GLP-1s dominate and the population grows older.
  2. 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.
  3. Stifel analysts were bullish on the data, which showed a 16.5% drop in body-mass index among patients with damage to the hypothalamus taking Rhythm Pharmaceuticals’ Imcivree.
  4. Under Friday’s final ruling anti-obesity medications for weight-loss will remain ineligible for Medicare coverage.
  5. Eli Lilly says Indianapolis-based Premier Weight Loss is cracking open auto-injector pens containing its blockbuster drug and repackaging them into separate doses.
POLICY
  1. Leerink Partners previews the drugs likely to be subject to the Inflation Reduction Act’s next price negotiations as the program expands to Medicare Part B and smaller biotechs.
  2. A February executive order on pharmaceutical price transparency does nothing to change the incentives that keep costs opaque. But drug companies and other stakeholders would reap the benefits of such disclosures.
  3. In a video posted to social media on Tuesday, HHS Secretary Robert F. Kennedy, Jr. claimed that there is a lack of clinical data to support the repeat COVID-19 booster strategy in children.
  4. Rouzbeh Haghighat was serving on the board of Chinook Therapeutics at the time of the Novartis acquisition and, according to the DOJ’s allegations, used confidential information to conduct insider trading.
  5. According to President Trump, CMS Administrator Mehmet Oz is a “tough hombre” who can bring down drug costs “like a rock.”
CAREER HUB
A recent legal decision could signal the ultimate demise of the FTC’s final rule banning most noncompete clauses. A biotech talent expert discusses how that affects biopharma job searches.
Whether they’re newly minted managers or C-suite executives, effective managers often lead with empathy, intellectual curiosity and vulnerability.
Generative AI could enhance and accelerate the way people work on clinical trials. In this Q&A, a management consultant shares his insights on benefits, risks and more.
While many describe California as having a tough life sciences market, there’s some optimism that employment opportunities will improve soon, according to California Life Sciences President and CEO Mike Guerra.
Looking for a quality control job? Check out these nine companies hiring life sciences professionals like you.
Companies will look at job candidates’ LinkedIn profiles, so make sure yours is strong, from the summary of your expertise and qualifications to testimonials from colleagues.
After more than 20 years at Eli Lilly, Leslie Sam moved into independent consulting. To prepare for the transition, she focused on becoming technically deep and earning industry recognition.
HOTBEDS
Where are the Best Places to Work in life sciences? BioSpace’s annual Best Places to Work list demonstrates a company’s desirability in the recruitment marketplace - find out who made the list this year.
IN CASE YOU MISSED IT
The molecule, developed in collaboration with Massachusetts-based Kailera Therapeutics, is headed for a new drug application in China and global clinical trials.
REPORTS
Following a tumultuous start to 2023, layoffs have cooled off though recruiting activities have been significantly impacted by the economic environment.
The 2021 Salary Report is significant in more ways than one. This year, BioSpace analyzed salary data through two key lenses: the impact of the pandemic, and for the first time, race and ethnicity.
BioSpace’s 2022 Salary Report explores the average salaries and salary trends of life sciences professionals. Though movement in the labor market slowed during the pandemic, recovery has been swift and employers are once again having to cope with a highly competitive talent market.
CANCER
  1. The cell engineering company, co-founded by oncologist and writer Siddhartha Mukherjee, does not see a path forward for its pipeline of early-stage cell therapies for two different types of cancer.
  2. The Massachusetts biotech will focus its efforts and resources into cemsidomide, an oral drug candidate being trialed for multiple myeloma and non-Hodgkin lymphoma.
  3. Keytruda is set to lose exclusivity in 2028, meaning Summit may face competition from cheaper biosimilars. Meanwhile, other branded drugmakers are also seeking to improve on the blockbuster checkpoint inhibitor.
  4. BNT327, a PD-L1/VEGF therapy, is still currently being manufactured in China, but BioNTech is working to establish a diversified supply chain, executives said during the company’s Q1 investor call.
  5. On the FDA’s docket this month are two expansion bids, one for GSK’s asthma drug Nucala into COPD and another for Merck’s oral cancer drug for a pair of rare tumors.
NEUROSCIENCE
  1. Jefferies analysts predict Annexon’s tanruprubart could be approved by mid-2026.
  2. Jazz is being accused of anti-competitive practices regarding its narcolepsy drug, as generic competitors emerge on the market.
  3. Wegovy and Zepbound are just the latest drug dyads to face-off in the competitive pharma market, continuing a legacy of rivalry that includes blockbuster drugs Keytruda, Humira and Eliquis.
  4. GSK is paying to access ABL Bio’s Grabody-B platform, which potentially enables therapies to cross the blood-brain barrier.
  5. Analysts at BMO Capital Markets said Centessa’s orexin receptor agonist has “best-in-class” potential for narcolepsy, putting the company in a strong position in the $15 billion market.
CELL AND GENE THERAPY
  1. Ayrmid’s offer is 50% higher than bluebird’s previously announced deal with Carlyle and SK Capital Partners.
  2. Industry updates and investment insights—including investor behavior and fundraising advice—were among the topics experts explored at Charles River Laboratories’ 2025 Cell & Gene Therapy Summit.
  3. Werner held roles at Bristol Myers Squibb, AstraZeneca and Novartis before landing at Alltrna, where she works to develop tRNA-based treatments for a range of diseases.
  4. Last month, Deerfield Management accused Alcon of obstructing Aurion’s IPO plans so it could acquire the startup “at a discount.”
  5. Adaptimmune is rolling out its T cell therapy Tecelra for synovial sarcoma, recording $1.2 million in sales since its approval in August 2024. Nevertheless, it is pausing development of two oncology assets to save money.