The star of the acquisition is the enzyme replacement therapy INZ-701, being developed for the rare disease ENPP1 deficiency.
The FDA also approved the use of Zynyz as a monotherapy for patients with squamous cell carcinoma of the anal canal who are intolerant to platinum chemotherapy or whose disease has progressed.
Since Elevidys’ accelerated approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up.
Lilly will use Rznomics’ proprietary ribozyme technology to develop RNA editing therapies for congenital hearing loss.
Over Lars Fruergaard Jørgensen’s eight years as CEO, Novo’s sales, profits and share price have almost tripled, the company said. However, the shares have taken a turn since mid-2024, falling by half in one year.
As the FDA prepares for a busy Oncologic Drugs Advisory Committee meeting next week, an agency insider told BioSpace that volunteers with little training are scrambling to secure the required expertise after workforce cuts decimated the adcomm planning office.
FEATURED STORIES
While San Diego remains a top biotech hub behind Boston and San Francisco, the city—which hosts this week’s BIO International Convention—has seen employment drop amid economic headwinds.
Experts say approval of Lykos Therapeutics’ MDMA capsules for post-traumatic stress disorder would open the door to further research into psychedelic-assisted therapies.
AstraZeneca last week set another ambitious goal, this time with plans to nearly double its total revenue by the end of the decade. However, it’s easier said than done, according to analysts.
While NK cell therapies can potentially avoid the serious side effects sometimes seen with CAR T cell therapies, experts say durability may stall their path to the market.
As AstraZeneca looks to climb toward the top of biopharma companies by revenue by the end of the decade, smaller companies are looking to join the ranks of the unofficial Big Pharma club.
Recent M&A activity indicates a potential resurgence in the appetites of larger companies for psychiatric drug development, but experts say the space may not offer a sufficient risk-reward proposition for R&D.
FROM BIOSPACE INSIGHTS
While FDA Commissioner Marty Makary emphasizes learning and humility, the FDA has systematically removed the very experience that would make change possible.
LATEST PODCASTS
In this episode presented by Cresset, BioSpace’s head of insights Lori Ellis discusses the emerging geopolitical battle for AI supremacy and global AI governance with Mutlu Dogruel, VP of AI and Mark Mackey, CSO of Cresset.
George Tidmarsh takes over temporarily at CBER following Vinay Prasad’s abrupt departure; Replimmune trial leaders protest rejection reportedly driven by FDA’s top cancer regulator Richard Pazdur; Merck’s $3 billion savings push claims 6,000 jobs; and Pfizer CEO Albert Bourla addresses President Donald Trump’s new threats around Most Favored Nation drug pricing.
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the concerns and opportunities of patient data driving AI tasks with Louise Molloy, associate director medical information and pharmacovigilance.
Job Trends
Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Opdivo® (nivolumab), in combination with cisplatin and gemcitabine, for the first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), the most common type of bladder cancer.
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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
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Despite a sharp downturn in initial public offering activity, New York-based gene therapy company Lexeo Therapeutics and French biotech Abivax are seeking funding for their lead candidate programs. -
The Italian pharma company is acquiring what was once one of the hottest stocks in the biotech sector, just months after the FDA rejected Intercept’s non-alcoholic steatohepatitis candidate. -
After a sluggish start to the year, experts expect an uptick in IPO offerings moving into fall, although it’s unlikely 2023 will fully shrug off its slump. -
In the largest biotech Series C financing so far this year, Generate:Biomedicines raised $273 million, while Neumora and RayzeBio announced IPO pricing valued at more than $560 million combined. -
The two biotech companies announced initial public offering pricing Thursday, respectively, with shares beginning trading Friday and valued at more than $560 million cumulatively.
WEIGHT LOSS
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After stopping the study early due to strong efficacy, Novo Nordisk released data from the FLOW study showing significant benefits of semaglutide in patients with type 2 diabetes and chronic kidney disease. -
Eli Lilly is planning to launch its blockbuster diabetes drug Mounjaro next year in India, the world’s most populous nation, ahead of its main competitor Novo Nordisk which is eyeing an Indian launch of Wegovy in 2026. -
Viking Therapeutics announced Tuesday that its GLP-1/GIP receptor dual agonist helped patients lose up to nearly 15% of their weight over about three months in a Phase II study. -
Boehringer Ingelheim and Zealand Pharma’s dual glucagon/GLP-1 receptor agonist elicited significant topline Phase II results in metabolic dysfunction-associated steatohepatitis. -
Seeking a slice of the lucrative obesity market, Indian pharma companies are developing their own versions of Novo Nordisk’s blockbuster weight-loss drug Wegovy, Reuters reports.
POLICY
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This week on Denatured, Head of Insights Lori Ellis and guests discuss the implications of not addressing the DE&I data gaps for the future. -
The Department of Justice and Securities and Exchange Commission are investigating two senior employees of Cassava Sciences in connection with allegations of data manipulation for its Alzheimer’s disease candidate simufilam. -
The Federal Trade Commission has opened an investigation into Teva Pharmaceuticals after the company allegedly ignored directives to withdraw or amend contentious patent listings on the FDA’s Orange Book. -
The U.S. Supreme Court on Friday overturned the long-standing Chevron doctrine, which required courts to defer to federal agencies and their interpretation of statutes, putting potential limits on the FDA’s regulatory decisions. -
Daiichi Sankyo has secured a victory in its patent arbitration with Seagen, nabbing a $47 million award for attorneys’ fees and other costs, plus interest. However, a larger patent battle with Pfizer remains.
Consider limiting the use of “manage” on your resume, opting instead for more powerful keywords for your resume that can better indicate you’re a leader.
If you’re having trouble finding your confidence again, try these tips for regaining a (healthy) ego.
The interview process at many organizations is not limited to a single interview. Multiple interviews are common and can occur for numerous reasons.
If you’re planning on looking for a new job at the beginning of the year, now is the time to start your job search prep.
Interview practice will help you reduce interview anxiety, improve your interview skills, and in many cases, gain important feedback about how you interview.
While many job seekers are unnerved by the “Tell me about yourself” request, it’s actually an opportunity to shine and take control of the interview.
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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In 2023, the ADC market exceeded $10 billion, and this momentum is persisting into 2024, as evidenced by several strategic deals and a robust pipeline of candidate drugs.
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Gilead Sciences’ blockbuster antibody-drug conjugate Trodelvy has encountered some clinical speed bumps in 2024 in bladder and lung cancer. Still, analysts remain bullish on its prospects in the oncology space. -
The FDA on Thursday rejected Merck and Daiichi Sankyo’s HER3-targeted antibody-drug conjugate patritumab deruxtecan in a Complete Response Letter, citing problems with a third-party manufacturer. -
AbbVie and Genmab on Wednesday announced the FDA has expanded the label of their bispecific antibody Epkinly, allowing it to be used in patients with relapsed or refractory follicular lymphoma. -
One patient died of respiratory failure in a Phase I study of Lyell Immunopharma’s investigational CAR-T therapy. The company on Wednesday said it has not definitively linked the fatality to the treatment.
NEUROSCIENCE
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Novo Nordisk seems to believe it can do a better job managing troubled Catalent than the contract manufacturer. However, the Danish drugmaker has its work cut out for it. -
The French drugmaker Thursday touted trial results in The New England Journal of Medicine showing frexalimab significantly slowed disease activity in people with relapsing multiple sclerosis. -
A longer-acting formulation of Otsuka Pharmaceuticals’ aripiprazole could help soften the blow of Abilify Maintena’s loss of exclusivity coming later this year, according to a new report from GlobalData. -
In the fourth quarter of 2023, Biogen’s revenue from multiple sclerosis drugs fell 8% due to generics competition and the company paid $60 million in close out costs related to Alzheimer’s treatment Aduhelm. -
AVP-786 was unable to significantly reduce total scores in the Cohen-Mansfield Agitation Inventory versus placebo, Otsuka Pharmaceuticals reported Monday. The company is evaluating the next steps for the drug combination.
CELL AND GENE THERAPY
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Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook. -
From innovation in manufacturing to more-flexible regulation and better communication with payers, much needs to happen to make CGTs commercially viable. But it is possible, experts agreed at a recent panel. -
The primary focus in scaling up production should first be the adoption of lean manufacturing principles used in virtually every other industry. -
The pivotal Phase II trial is testing Allogene’s CAR T candidate cemacabtagene ansegedleucel for large B-cell lymphoma. ALLO-647 was being used as a preparative lymphodepletion therapy. -
The FDA greenlit multiple new drugs this month and issued some notable label expansions, including for Eli Lilly’s Kisunla. Meanwhile, the regulator turned away a cell therapy for Duchenne muscular dystrophy and a gene therapy for the rare disease Sanfilippo syndrome.
