The drug, a small molecule protein inhibitor, brought in $132 million in the first quarter, missing consensus estimates by 17%.
As Q1 2025 earnings season continues, tariffs remain top of mind for pharma CEOs and investors. Meanwhile, the American Association for Cancer Research’s annual event kicks off this year’s oncology conference season. Plus, will the FDA become politicized under HHS Secretary RFK Jr.?
Nevertheless, Albert Bourla has been lobbying the U.S. government—as high as President Donald Trump himself—to skip the pharmaceutical industry tariffs, which Trump has threatened, in part as a way to shore up U.S. national security.
Despite executing perfectly, Octagon confronted a “scientific no-go,” CEO Isaac Stoner said in his LinkedIn post announcing the company’s impending closure.
The condition, recessive dystrophic epidermolysis bullosa, causes chronic wounds and has an 84% mortality rate by age 40.
Vas Narasimhan said on Tuesday that if the U.S. adopts international drug pricing, all companies would have to “relook at their medium- to long-term outlook.”
FEATURED STORIES
AbbVie’s Humira was the top-selling drug in the world for many years. Now, its sales are eroding as doctors switch to biosimilars and new options enter the market.
Biosimilars are essential healthcare equalizers, but their regulation is overly complicated due to lobbying by makers of branded biologics looking to maintain blockbuster revenue.
Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several companies—including Novartis, Scholar Rock and Biogen—progressing novel candidates through clinical trials.
FROM BIOSPACE INSIGHTS
In a year when eradicated diseases are on the uptick in America, how will American children survive RFK Jr.’s vaccine scrutiny and inconsistency? Two experts call on pharma and regulatory bodies to rebuild trust.
LATEST PODCASTS
In this episode of Denatured, Lori and guests from Teva Pharmaceuticals and TOWER Capital review the investment landscape this year and the policies influencing investment heading into 2025.
Yet another therapy with FDA accelerated approval suffers a setback; Sage’s tough year continues; Sanofi drops $326 million in radiopharma while selling its consumer health unit; Novo Nordisk’s positive Rybelsus results in cardiovascular disease; and more.
J&J beat expectations this week to launch the Q3 earnings season; a study about children treated with bluebird bio’s Skysona comes at a bad time for the company; Sen. Warren calls for scrutiny of Novo’s purchase of Catalent; and other news.
Job Trends
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced positive topline results from its HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).
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SPECIAL EDITIONS
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.
Year-over-year BioSpace data show biopharma professionals faced increased competition for fewer employment opportunities during the first quarter of 2025.
DEALS
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BridgeBio’s Attruby wins approval for transthyretin amyloid cardiomyopathy while the FDA accepts Alnylam’s application for Amvuttra in the indication; Cassava’s controversial Alzheimer’s drug flunks Phase III; Amgen’s MariTide fails to impress investors, Donald Trump’s controversial nominations continue. -
With Elevidys expansion in hand, Sarepta commits up to $10 billion to develop short interfering RNA–based drugs to build out its pipeline. -
Novartis has disclosed roughly $19.4 billion in deals in the past five years. CEO Vas Narasimhan says there’s more to come. -
The deal has secured Novartis the chance to work with Ratio Therapeutics on a novel drug candidate that could fortify the Big Pharma against competition from would-be radiopharmaceutical rivals such as BMS and Lilly. -
The acquisition will give BioNTech full ownership of an investigational bispecific antibody targeting the PD-L1/VEGF-A pathways, a hot area in oncology that could potentially replace standard checkpoint inhibitors for cancer treatment.
WEIGHT LOSS
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Metsera will use its IPO proceeds to fund the Phase III development of its injectable, ultra-long-acting GLP-1 therapy MET-097i, which last week achieved 11.3% weight loss in a Phase IIa study. -
The company’s lead asset is a potentially first-in-class oral GLP-1 receptor agonist that has the potential to be dosed weekly, which according to CEO Khurem Farooq can help improve accessibility and affordability. -
According to BMO Capital Markets, Medicare coverage of Lilly’s Zepbound opens the door to using secondary indications to secure CMS coverage for obesity drugs. -
The FDA recommended maintaining a minimum of 5% weight-loss for drug developers seeking to establish the efficacy of their investigational obesity candidates. -
BioSpace presents 25 noteworthy biopharma startups in ’25; analysts forecast stronger M&A as the J.P. Morgan Healthcare Conference kicks off next week; GLP-1s continue to expand their reach as Novo, Lilly fight against compounders; and a look ahead to five key FDA decisions in Q1.
POLICY
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The last few years have been tough for the insulin market, with recent policies and high-level pressure forcing companies to lower drug prices. -
Konstantina Katcheves, Senior VP of Innovative Global Business Development at Teva Pharmaceuticals brings insights from the World Economic Forum to SCOPE 2025. -
Pfizer reacts to Donald Trump’s tariff threats on big pharma, another regulatory meeting is canceled under RFK Jr., AbbVie and Eli Lilly strike mid-sized deals in obesity and molecular glues, priority review vouchers set to take a hit and immuno-oncology matures.
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Leqembi’s application now moves forward to the European Commission, which will issue a formal verdict for the injection that will apply to all EU member states as well as Norway, Liechtenstein and Iceland. -
In the current legal and political landscape, it is all about survival for DEI initiatives.
BioSpace spoke to HR leaders about how they have been supporting companies navigating a challenging economy while meeting the needs of the workforces they support.
Politics can be a touchy subject, especially during a presidential election year. How should you engage in political discussions at work?
A string of rejections prompt some to look beyond research roles in the biopharma industry as they seek to launch a career.
The past year saw the slowest year-over-year growth in biopharma salaries in the past five years, according to the BioSpace 2024 Life Sciences Salary Report.
Staffing agencies say contract work is a great way to break into an industry and avoid a resume gap.
Plus, tips on applying to multiple jobs at the same company, making new work friends, and how to ask for more time at the offer stage.
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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Despite significant dips in its vaccines sales, the British pharma narrowly beat consensus estimates for Q4 2024 and raised 2031 sales projections to just over $50 billion. -
Faced with the encroaching threats of patent expirations and generics, biopharma companies in 2024 invested 33% more in licensing deals, on average, than in 2023 with an eye toward enriching their pipelines with novel and potentially more effective therapies. -
Topline data on a combo including Pfizer’s kinase inhibitor Braftovi point to improved progression-free survival and pave the way for its full approval for the treatment of certain colorectal cancers, according to the company. -
Novartis was among the most prolific pharma dealmakers in 2024, a trend that it expects to continue with more bolt-on deals this year to set up for sustainable long-term growth. -
In this episode, presented by the Genscript Biotech Global Forum 2025, BioSpace’s Head of Insights Lori Ellis talks to Tom Whitehead, co-founder of the Emily Whitehead Foundation, about how standard care, cell and gene therapies and their impact on patients.
NEUROSCIENCE
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Emraclidine was the centerpiece of AbbVie’s $8.7 billion acquisition of Cerevel in December 2023 but failed two mid-stage trials. Tavapadon, meanwhile, has been a more rewarding asset for the pharma, clearing three Phase III Parkinson’s studies in 2024. -
Among the 55 novel drugs that crossed the regulatory finish line last year were notable new mechanisms of action, coming particularly in the oncology and neurosciences spaces. -
Misses from amyotrophic lateral sclerosis hopefuls Denali Therapeutics and partners AbbVie and Calico Life Sciences mark the latest setbacks for the controversial platform trial, the results from which have largely mirrored the dismal success rate in ALS overall. -
Backed by ARCH Venture Partners, F-Prime Capital and Mubadala Capital, the new company will develop a pipeline of brain-penetrant small molecules to address inflammation, metabolic dysfunction and restoring lysosomal function. -
Denali’s failure on Monday continues biopharma’s losing streak against amyotrophic lateral sclerosis. PTC Therapeutics and Amylyx have seen similarly disappointing results.
CELL AND GENE THERAPY
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Not exactly known for its dealmaking, Sarepta Therapeutics has thrown down a massive wad of cash to work with Arrowhead Pharmaceuticals on RNAi-based medicines. -
The cancers were diagnosed 19 to 92 months after Skysona treatment. -
Emboldened by technological advances and a deeper knowledge of glioblastoma, Merck, Kazia Therapeutics, CorriXR Therapeutics and others are targeting the often-fatal brain tumor. -
Despite the death, the FDA has allowed Neurogene to forge ahead with the Phase I/II Rett syndrome trial, but using only the lower 1E15 vg dose of its investigational gene therapy NGN-401. -
Novartis’ up to $1.1 billion acquisition of gene therapy specialist Kate Therapeutics fits with the pharma’s plan to expand its new modality pipeline to ensure long-term business sustainability.
