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
Experts say Novo Nordisk and Eli Lilly’s GLP-1 drugs are unlikely to reach more countries in the near term, but Sanofi’s diabetes treatment has gained ground globally.
The recent invalidation of an AAV gene therapy patent overlooks the complexity of innovation in biotechnology and could put a broad swath of intellectual property at risk.
Psychedelic drug developers are undeterred by the FDA’s Complete Response Letter for the company’s MDMA therapy for PTSD, and experts expect Lykos will ultimately obtain approval.
Regeneron, Akouos and Mass Eye and Ear are testing therapies that can reverse genetic protein deficiency to restore hearing, with promising early results.
The entry of new players and new approaches into the ATTR-CM space could help bring down the cost of treatment, experts say.
For the Biden-Harris administration to compare the newly announced negotiated Medicare prices to the list prices for these drugs is, at best, not very meaningful. At worst, it’s disingenuous.
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
In this episode presented by Cresset, BioSpace’s head of insights Lori Ellis discusses clinical trial fail rates and AI’s potential to reduce preclinical costs with Mutlu Dogruel, VP of AI and Mark Mackey, CSO of Cresset.
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.
Job Trends
The Kinetix Group and Pfizer Inflammation and Immunology received a PM360 Pharma Choice Award for their recent health equity initiative.
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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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BioSpace and guests from Halia Therapeutics, Triumvira Immunologics and the Alzheimer’s Drug Discovery Foundation discuss alternative financing strategies to consider for 2024. Listen now. -
Tome Biosciences has only been on the scene for a short time but on Tuesday notched an acquisition of CRISPR-based biotech Replace Therapeutics for $65 million upfront. -
All three companies closed out the year, which saw significant growth in mergers and acquisitions, with high-value deals that could potentially set the tone for M&A in 2024. -
The acquisition announced on Friday will give Bristol Myers Squibb ownership of Karuna Therapeutics’ investigational antipsychotic KarXT, which is being tested for schizophrenia and Alzheimer’s disease psychosis. -
Continued fallout from the IRA, heightened antitrust enforcement and the rise of AI will all shape the first wave of biotech mergers and acquisitions coming this year, experts tell BioSpace.
WEIGHT LOSS
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The weight-loss drug bonanza continued in the first quarter of 2024 for Novo Nordisk and Eli Lilly, as Amgen also posted strong results, while Biogen and BMS struggled early in the financial year. -
While the FTC continues to review its $16.5 billion buy of Catalent, Novo Holdings announced Wednesday it has acquired a majority stake in Single Use Support, an Austrian life sciences tools company. -
Presented at this week’s European Congress on Obesity, the two studies also demonstrate that Novo Nordisk’s Wegovy (semaglutide) provides cardiovascular benefits irrespective of starting weight and the amount of weight lost. -
Crackdowns on drug pricing have forced one major insulin out of the market. Are more to come? -
As competition with Eli Lilly heats up, Novo Nordisk has partnered with Flagship’s Metaphore Biotechnologies to take a biomimicry approach to GLP-1s.
POLICY
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Healthcare players are pointing fingers amid regulatory crackdowns on pharmacy benefit managers, but proposed reforms wouldn’t address a dearth of competition in the larger market. -
A recent study estimated that Wegovy’s label expansion beyond obesity could push Medicare spending to $145 billion annually, but analysts remain dubious of the estimate. -
The U.S. House of Representatives on Monday overwhelmingly passed the bipartisan bill, which targets WuXi AppTec, WuXi Biologics and other Chinese biotech companies as potential national security risks. -
As Congress considers a bill that aims to distance U.S. biopharma from five Chinese companies, the industry must emphasize the importance of prioritizing patient care over power plays. -
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.
If you’ve been furloughed, it’s normal to feel some apprehension, fear and/or doubt about your employment. These are four ways to stay productive if you’ve been furloughed.
Most job applicants don’t know about questions that they should ask at the end of an interview. Here are some not-so-common interview questions that you must remember to ask.
Once you do land your next job interview, you’ll want to address your layoff in the right way. Here’s how.
Finding common ground and making small talk are both skills that help you build that rapport with someone.
Of the resumes submitted to employers that use Applicant Tracking Systems (ATS), 75% are never seen by human eyes.
The global COVID-19 pandemic may not seem like the best time to accelerate your career, but some characteristics of this unusual time actually lend themselves to career-boosting activities. Whether you are still working from home or are back in your workplace, this article offers suggestions for pumping up your career.
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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Proceeds from the oversubscribed financing will be used to advance ArsenalBio’s lead programs, which include a handful of solid tumor cell therapy candidates. -
NuCana’s chemotherapy replacement has failed to improve progression-free survival in a Phase II test, sending the biotech’s shares down by 50%. -
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. -
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. -
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.
NEUROSCIENCE
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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. -
Data from the Phase III OCARINA II study shows the subcutaneous version of Ocrevus achieved near-complete suppression of relapses and brain lesions in relapsing or primary progressive multiple sclerosis. -
Cerevel Therapeutics on Thursday reported positive data from its Phase III TEMPO-3 trial, showing that tavapadon can significantly improve symptom control in patients with Parkinson’s disease. -
Sage Therapeutics announced Wednesday it is scrapping its Parkinson’s disease program after the company’s investigational drug showed no benefit over placebo. Phase II studies of the oral treatment will continue in Huntington’s and Alzheimer’s diseases. -
After withdrawing ALS drug Relyvrio from the U.S. and Canadian markets and laying off 70% of its workforce, the Cambridge, Mass.–based biopharma got a much-needed win in Wolfram syndrome.
CELL AND GENE THERAPY
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The FDA’s busy week ahead involves three decision dates for potential industry firsts and a highly anticipated advisory committee meeting for two CAR-T therapies. -
Separate challenges exist for companies developing gene therapies for rare and common cardiovascular conditions, experts told BioSpace. -
Formerly known as Ryne Bio, Kenai Therapeutics emerged on Thursday with backing from several groups and has a cell therapy candidate going after Parkinson’s disease. -
Sarepta Therapeutics on Wednesday called the launch of the gene therapy for Duchenne muscular dystrophy “exceptional” but the company does not expect to see significant growth in the first half of 2024 due to its currently limited patient pool. -
Cell and gene therapy professionals gathered in Miami last month to discuss new manufacturing approaches for these up-and-coming treatments.
