Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline.
The partnership with Sirius expands CRISPR Therapeutics’ modality toolkit, especially in the cardiovascular space.
The late-stage results come in advance of pivotal data that Ionis expects to provide for its antisense oligonucleotide Tryngolza in the third quarter, building up toward a regulatory submission in hypertriglyceridemia by year-end.
In an interview on Friday, FDA Commissioner Marty Makary threw his weight behind psychedelic therapies, noting that patients taking these substances experience significant benefits for various neuropsychiatric conditions.
The deal comes three months after Pfizer inked a PD-1/VEGF partnership with Summit Therapeutics, leading BMO Capital Markets to express confusion regarding the pharma’s overall strategy.
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.
FEATURED STORIES
Biotech was starting to show signs of recovery after years of investor pullback—until new tariffs and economic uncertainty sent fresh shockwaves through an already fragile market.
Alnylam and BridgeBio are competing for people who are switching from Pfizer’s blockbuster ATTR amyloidosis drug tafamidis while all three companies are fighting for new patients.
FDA Commissioner Marty Makary last week announced a directive that would limit industry participation in the agency’s advisory committees. But not only do company reps serve only as non-voting members, a 1997 law actually requires industry involvement.
FROM BIOSPACE INSIGHTS
While Quantum computing has been reported to be five years away for many years now, companies are preparing for it by setting foundations with AI in development.
LATEST PODCASTS
The Novo-Catalent deal now moving ahead highlights unprecedented investment in manufacturing, while also standing out as an exception to the unspoken rule of keeping M&As to less than $5 billion this year.
GSK, Gilead and Arcellx, Vertex and more present new data at the American Society of Hematology annual meeting just as sickle cell therapies Casgevy and Lyfgenia have a new outcomes-based payment model; Eli Lilly and Novo Nordisk pump new funds into manufacturing; and AbbVie makes a Cerevel comeback while uniQure clears a path toward accelerated approval in Huntington’s disease.
In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis, Miguel Forte and Ali Pashazadeh discuss how a slow and steady pace is a continuation of the pattern we have seen throughout the last three years.
Job Trends
Merck announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended the approval of WINREVAIR.
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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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Initial rounds of VC financing totaled $7.7 billion over 137 deals for biopharma in 2024, compared to $3.8 billion over 156 deals in 2023. -
J.P. Morgan releases its quarterly look-ahead days before the entire biopharma industry descends on San Francisco for the annual J.P. Morgan Healthcare Conference. -
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. -
Licensing deals have risen in prominence in a restrained market environment. Is it desperation, or an important part of the biotech ecosystem? Experts weigh in. -
Look for renewed investment driven by lower interest rates in the new year, and a continued focus on late-stage assets, oncology and reaping the benefits of AI.
WEIGHT LOSS
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Wegovy sales increased by more than 100% over 2024. But that wasn’t enough to satiate analysts who want to know why Novo Nordisk can’t access more patients, particularly in the U.S. -
Amgen outperformed expectations in the fourth quarter of 2024, but revealed an FDA hold on early-stage obesity asset AMG 513 and the discontinuation of other programs. -
Just over a year after striking an obesity deal with Novo Nordisk, an SEC filing shows Flagship Pioneering spinout Omega Therapeutics is days away from bankruptcy and will lay off up to 17 employees. -
Amid growing concern of the overuse and misuse of obesity drugs, the UK’s pharmacies regulator rolled out stricter guidelines for online pharmacies selling medicines including Novo Nordisk’s Wegovy and Eli Lilly’s Mounjaro. -
The U.S. Centers for Medicare and Medicaid Services has named Novo Nordisk’s Wegovy, Ozempic and Rybelsus as part of the second round of the IRA drug price negotiation program, even as the pharma challenges the program.
POLICY
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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. -
Senators Bill Cassidy and Bernie Sanders asked HHS Secretary Robert F. Kennedy, Jr. to make good on a pledge to appear before the committee regularly. It is not clear if or when the hearing, which was scheduled for Thursday, will take place. -
Under Friday’s final ruling anti-obesity medications for weight-loss will remain ineligible for Medicare coverage. -
Kennedy, a long-time opponent of vaccines, stated that the MMR vaccine is “the most effective way” to combat the measles outbreak, which has already claimed the lives of two children in the U.S. -
Pharma’s reprieve from Donald Trump’s tariffs is expected to be temporary, with Leerink analysts anticipating possible sector-specific duties “in the next month or so.”
Dry promotions include new titles and responsibilities without higher pay. What should you do if you’re offered this type of promotion?
By incorporating soft skills in your resume, you showcase your ability to work well with others, which is important to employers.
It’s important to quit your job the right way so you can exit on a positive note, which could benefit you in the future.
The FTC’s final rule banning most new noncompetes should go into effect later this year, although it faces opposition from the U.S. Chamber of Commerce.
By understanding the different types of difficult bosses, you can shift your perspective and approach the situation from a less stressful, more effective angle.
Despite the benefits of hiring foreign-born STEM employees, some companies avoid it largely due to unfamiliarity with the visa process, according to two recruitment experts.
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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Abecma made $406 million in 2024, of which BMS paid $43 million to 2seventy bio as part of their profit-sharing agreement. -
The FDA’s Oncologic Drugs Advisory Committee recently voted to narrow the label for checkpoint inhibitors Keytruda and Opdivo in stomach and esophageal cancers based on PD-L1 expression levels—but the high unmet need in these patient populations should also be considered. -
On the agenda for the FDA this month are two RNA-based treatments for rare diseases. -
Imfinzi is one of AstraZeneca’s key growth drivers for 2025, with potential approvals in stomach and bladder cancers. The PD-L1 blocker brought in over $4.7 billion in sales last year. -
The new formulation of Keytruda, currently under FDA review, is sparking conflict with Halozyme, which makes enzymes that convert intravenous drugs into injectable versions.
NEUROSCIENCE
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The monthly maintenance regimen, which offers a more convenient frequency than the initially approved treatment schedule for patients with Alzheimer’s, could help with Leqembi’s thus far disappointing uptake and sales. -
Vigil Neuroscience reported a strong safety profile and 50% sTREM2 reduction in an early-stage trial for VG-3927, potentially representing a new avenue for treating Alzheimer’s disease. -
In a good-news-bad-news week for Biogen, the company will cut an undisclosed number of employees, just as a higher dose of its Ionis-partnered therapy Spinraza for spinal muscular atrophy will be considered by the FDA and EMA. -
The drugs’ active ingredient, glatiramer acetate, has been linked to more than 80 cases of anaphylaxis worldwide since December 1996 and six patient deaths. -
The U.S. Court of Federal Claims ruled that Vanda cannot claim a breach of confidentiality for specifications that it did not develop itself.
CELL AND GENE THERAPY
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Sarepta Therapeutics’ Duchenne muscular dystrophy therapy Elevidys handily beat analysts’ expectations in the fourth quarter of 2024, reflecting the biotech’s “world-class” execution, according to BMO Capital Markets analysts. -
Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic for cardiovascular diseases including congestive heart failure, chronic refractory angina and cardiomyopathy. -
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. -
In this short teaser, BioSpace’s Head of Insights Lori Ellis talks to CBER Director Peter Marks and Tom Whitehead, Co-Founder of the Emily Whitehead Foundation about anticipated discussions at the upcoming GenScript Biotech Forum. -
In a deal expected to close in Q1 2025, Roche will gain access to Poseida’s off-the-shelf CAR T candidates.
