Avidity has been given the go-ahead by the FDA to request accelerated approval of delpacibart braxlosiran—potentially the first disease-modifying treatment for facioscapulohumeral muscular dystrophy—with an application in the second half of 2026.
The loss of special government employee status for four members of the CDC’s Advisory Committee on Immunization Practices follows the resignation of ACIP co-lead Lakshmi Panagiotakopoulos, who left in protest of a controversial change in COVID-19 recommendations for healthy children and pregnant women.
After a major shareholder pushed back, Keros is returning half of its capital to investors in a move that Guggenheim analysts called “a positive step forward.”
Merck’s enlicitide decanoate significantly lowered low-density lipoprotein cholesterol in two Phase III trials, unlocking what BMO Capital Markets called a “multi-billion dollar opportunity” for the pharma.
In what Guggenheim Partners called one of Metsera’s “critical program milestones” this year, its ultra-long-acting amylin injection MET-233i showed promising weight-loss after a little more than a month of treatment.
Empaveli reduced proteinuria by 68% versus placebo in glomerulopathy and glomerulonephritis, an effect that was sustained through one year of follow-up.
FEATURED STORIES
The intellectual property landscape for newer gene-editing technologies, like that for CRISPR-Cas9, remains unclear and hard to navigate.
Analysts expect the companies’ Vabysmo and Eylea HD to generate a combined $13.2 billion by 2030 in the vascular endothelial growth A therapy market, as healthcare providers and patients switch from older products.
With promising early results, cell and gene therapies are making headway against both rare and common ocular and auditory diseases.
The FDA has three regulatory milestones in the next two weeks, including a decision on a subcutaneous formulation of an effective multiple sclerosis therapy.
With gene therapies by REGENXBIO and AbbVie, Adverum and others in mid- or late-stage trials, this therapeutic class could soon be an option for this common cause of blindness in the elderly.
Eli Lilly this week announced plans to sell single-dose vials of its weight loss drug Zepbound directly to consumers. Novo Nordisk could adopt a similar strategy for Wegovy as its CEO is set to testify Sept. 24 before the Senate health committee.
FROM BIOSPACE INSIGHTS
Establishing trust through thought leadership is no longer optional in today’s cautious biopharma market. Learn how strategic insights and targeted outreach can turn awareness into high-converting leads.
LATEST PODCASTS
A draft copy of an upcoming MAHA report reveals a strategy in lockstep with recent HHS actions such as reviving the Task Force on Safer Childhood Vaccines; Viking Therapeutics reports robust efficacy from mid-stage oral obesity candidate but is tripped up by tolerability concerns; Novo Nordisk wins approval for Wegovy in MASH; and Lilly takes a pricing stand.
In this episode of Denatured, BioSpace’s head of insights Lori Ellis discusses the ‘enormous implications’ of patent policy changes with Aaron Cummings and Anne Li of Brownstein Hyatt Farber Schreck.
Prasad Returns, Delany Departs, Lilly’s Weight Loss Pill Disappoints and Sarepta’s Fallout Continues
CBER Chief Vinay Prasad reclaimed his job less than two weeks after his mysterious exit; MAHA implementor Gray Delany is out after reportedly sparring with other agency officials over communications strategy; Eli Lilly’s first Phase III readout for oral obesity drug orforglipron missed analyst expectations; and Arrowhead Pharmaceuticals addresses the recent woes of its of partner Sarepta.
Job Trends
Daiichi Sankyo (TSE: 4568) and Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced today that the first patient has been dosed in the REJOICE-Ovarian01 phase 2/3 trial evaluating the efficacy and safety of investigational raludotatug deruxtecan (R-DXd) in patients with platinum-resistant ovarian cancer.
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SPECIAL EDITIONS
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.
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.
DEALS
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Panelists at JPM’s Biotech Showcase gave positive projections as the year begins and offered advice to those in the space. -
The BioSpace team is recording from San Francisco as they bring you the the latest highlights from Day 3 at JPM2024. -
All assets of the regenerative medicine and cell therapy company are being divested to its research partner to the tune of $2 million in the form of a credit bid. -
Following in the footsteps of Novartis, J&J and Merck, GSK jumped into the J.P. Morgan dealmaking frenzy Tuesday, picking up respiratory and inflammation-focused Aiolos Bio. -
From the rising trend of royalty financing to other adaptive funding strategies, firms think outside the box in responding to unprecedented challenges.
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? -
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. -
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. -
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. -
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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As technology continues to evolve, companies should have strategies that incorporate an understand of where they are now, where they want to be, and do they have the talent to get there. -
In a high-profile showdown Tuesday with Sen. Bernie Sanders’ Senate health committee, Novo Nordisk CEO Lars Fruergaard Jørgensen will be asked to defend the drugs’ U.S. monthly list prices of $969 and $1,349, respectively. -
Regulators, policymakers and others can more effectively battle the disease by creating incentives to make mpox a more attractive investment opportunity. -
A lawsuit filed by the Pharmaceutical Research and Manufacturers of America, which claims the Inflation Reduction Act’s Medicare Drug Price Negotiation Program is unconstitutional, now goes back to a lower Texas court. -
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.
CAREER HUB
Results from a recent BioSpace Community Survey indicated that 67% of life science respondents are likely to look for a job in the next 12 months. With an influx of talent in the market, how can life science professionals set themselves apart to make a positive impression and land the job?
Whether you’re an experienced professional in the field or just embarking on your new career, it’s time to look at your qualifications and career plans from an entirely different perspective.
Maryland has become a key part of the strategy for Kite, a Gilead company, and its mission to find a cure for cancer.
As your organization continues with hiring efforts, interviewers will face new questions from candidates that they’ve never been asked before.
If you are looking for a new job, it is crucial to have an ATS friendly resume. Let’s discuss how to do this and why they’re so popular with hiring managers.
Do you have soft skills but don’t know how to show them on your resume? Here are 6 of the most popular soft skills for resume and tips on how to make sure they get noticed.
Here are some of the top questions you should consider asking during an interview regarding COVID-19.
HOTBEDS
IN CASE YOU MISSED IT
The rollercoaster week for Sarepta Therapeutics continued, with shares of the embattled gene therapy-turned-siRNA biotech down 37% Friday afternoon as media outlets reported FDA plans to request a stop to all shipments of the Duchenne muscular dystrophy therapy Elevidys following a third patient death linked to the underlying platform.
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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BioMarin’s new business strategy leaves investors with questions; Lykos CEO steps down; Terns releases compelling data on oral weight loss candidate; and more. -
Despite the disappointing late-stage results in non-small cell lung cancer, Jefferies analyst Stephen Barker contends the likelihood of FDA approval “remains high” for the experimental antibody-drug conjugate, though the regulator is now more likely to convene an advisory committee. -
Set to start in 2025, Relay Therapeutics is moving toward a pivotal study of experimental treatment RLY-2608 in heavily pretreated locally advanced or metastatic breast cancer. -
In advanced non-small cell lung cancer, Summit Therapeutics’ ivonescimab appears to be 49% more effective at reducing the risk of disease progression or death versus Merck’s Keytruda in a late-stage study. However, analysts contend the caveat is that the trial was conducted in an entirely Chinese patient population. -
Follow-up data from the MARIPOSA study show a favorable overall survival trend versus Tagrisso in EGFR-mutated advanced non-small cell lung cancer. The findings come just weeks after the Rybrevant-Lazcluze combination was approved for the first-line treatment.
NEUROSCIENCE
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Amylyx’s recent decision to withdraw its ALS drug Relyvrio from the market highlights an important business decision for companies: when to continue marketing or investigating a drug that has failed a pivotal or confirmatory study. -
Neurocrine Biosciences on Tuesday won the FDA’s approval for a sprinkle capsule formulation of Ingrezza, providing a more convenient route of treatment for Huntington’s disease patients with trouble swallowing. -
On June 10, the FDA will convene its Peripheral and Central Nervous System Drugs Advisory Committee to discuss the New Drug Application for Lilly’s Alzheimer’s drug. -
After a record low in 2022, the pharma industry in 2023 saw a surprise rebound in productivity following one of its slowest years on record, according to a report from audit firm Deloitte. -
Ketabon is working to develop a medication that harnesses ketamine’s antidepressant effects without the safety issues that currently prohibit its at-home use.
CELL AND GENE THERAPY
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To help support the launch of Lyfgenia, bluebird bio on Monday entered into a five-year term loan deal with Hercules Capital that will extend the biotech’s cash runway through the first quarter of 2026. -
Orchard Therapeutics on Monday secured the FDA’s first approval for an autologous gene therapy to treat the rare metabolic disease metachromatic leukodystrophy in children. -
By votes of 11-0 and 8-3, respectively, an FDA advisory committee Friday deemed the risks of early death for both Johnson & Johnson’s Carvykti and Bristol Myers Squibb’s Abecma acceptable.
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Asgard Therapeutics, a Swedish gene therapy biotech, has closed a $32 million Series A round with help from prominent pharma players as it prepares for a 2026 IND. -
The FDA approved Bristol Myers Squibb’s Breyanzi for chronic lymphocytic leukemia and small lymphocytic leukemia prior to Friday’s adcomm for the company’s other CAR-T therapy, Abecma.
