The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access to patients two years and older in what one Stanford Medicine professor called a “game changing advance” for the field.
Novo Nordisk’s amycretin showed no weight-loss plateau over 36 weeks in patients with type 2 diabetes, suggesting its efficacy could become even stronger with longer follow-up, according to analysts at BMO Capital Markets.
Tecvayli plus Darzalex led to an 83% boost to progression-free survival versus the current standard therapy in relapsed or refractory multiple myeloma, results analysts at Guggenheim Securities called “remarkable.”
Richard Pazdur, the new director of the Center for Drug Evaluation and Research, raised concerns amid the rollout of several FDA initiatives seeking to shorten the drug review process.
Johnson & Johnson will discontinue the Phase II Auτonomy study of posdinemab after a scheduled review found the anti-tau antibody failed to slow clinical decline in patients with early Alzheimer’s disease.
Analysts agree that the failure of Novo Nordisk’s semaglutide to reduce Alzheimer’s disease progression removes a “modest” or “perceived” overhang on Biogen and the anti-amyloid antibody class in general, clearing the way for increased uptake of Leqembi and Eli Lilly’s Kisunla.
FEATURED STORIES
While a new facility setup program aimed at encouraging onshoring received a positive reception at a recent meeting, industry representatives said the current rules on existing production plants are the main regulatory issues facing manufacturing teams.
To drive true innovation in drug development, executives must not let excitement about the latest shiny object obscure ultimate outcomes.
Heading into the final quarter of a year that has seen dramatic upheaval at the FDA—from the exodus of numerous senior leaders to unclear policy changes and a safety saga that engulfed the gene therapy space—drug approvals appear roughly on par with recent years.
Therapies from industry leaders BioMarin and Ascendis Pharma supply a key hormone that promotes bone growth. In order to move the field forward, challengers are looking to address the underlying cause of the rare, genetic disease.
New analysis from Jefferies shows that rare disease and cancer drugs granted the status are especially likely to be approved.
Takeda wanted to create something new in the cell therapy world by combining the technology with T cell engagers. A series of acquisitions in 2021 started the process.
LATEST PODCASTS
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.
Sarepta’s Elevidys is back on the market for ambulatory patients with Duchenne muscular dystrophy, Health Secretary Robert F. Kennedy Jr. reportedly plans to dissolve the U.S. Preventive Services Task Force and “fix” the vaccine injury compensation program, Merck, AstraZeneca and more report Q2 earnings, Novo names a new leader and Roche’s trontinemab impresses at AAIC25.
Job Trends
Less than two months after two FDA-related setbacks, Atara Biotherapeutics is again cutting its workforce in half. This time, it’s also hitting pause on two CAR T programs, including one affected by an FDA clinical hold in January.
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SPECIAL EDITIONS
In this deep dive, BioSpace investigates China’s rise as a biotech powerhouse.
In this deep dive, BioSpace explores the next big thing in obesity.
BioSpace did a deep dive into biopharma female executives who navigated difficult markets to lead their companies to high-value exits.
DEALS
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If the trend holds, IQVIA expects 2025 deal volume between Chinese and multinational companies to easily eclipse the 100 agreements signed in 2024. -
The deal extends AbbVie’s commitment to the psychedelics space and depression, after emraclidine’s high-profile flop in schizophrenia last November. -
Patients who are prescribed Wegovy or Ozempic can now use GoodRx to access the medications at just $499 a month if they skip insurance. This is not the first time Novo has partnered with a pharmacy to offer the blockbuster drugs. -
After a slow 2024, the biotech shell company Concentra Biosciences is back, offering to buy four biotechs in the past month and seven so far this year. -
Sarepta’s troubles had nothing to do with Arrowhead’s assets, and yet both companies have seen their stock prices decline this past month. BioSpace caught up with Arrowhead’s Chris Anzalone to talk about the biotech’s role as an RNAi pipeline savior.
WEIGHT LOSS
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The sub-analysis, presented at the European Association for the Study of Diabetes congress, showed improved safety data to counteract past tolerability issues. -
Both Novo Nordisk and Eli Lilly are eyeing regulatory advancements for their obesity blockbusters as the European Association for the Study of Diabetes’ annual conference continues this week. -
While Eli Lilly’s orforglipron is top of mind heading into the European Association for the Study of Diabetes meeting this week, experts told BioSpace the conference will also provide important insights into the therapeutic benefits of incretin therapies beyond weight loss. -
As Novo Nordisk cuts 9,000 people from its organization in a restructuring effort, BioSpace looks back on the Danish pharma company’s rise. -
This week’s release of the Make America Health Again report revealed continued emphasis on vaccine safety; Health Secretary Robert F. Kennedy Jr.’s faceoff with senators last week amounted to political theater; the FDA promises complete response letters in real time and shares details on a new rare disease framework; and Summit disappoints at the World Conference on Lung Cancer in Barcelona.
POLICY
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New draft guidelines suggest the FDA is open to exercising regulatory flexibility for non-opioid drugs being developed for chronic pain. -
Former CDC director Susan Monarez and former chief medical officer Debra Houry will appear in front of the Senate HELP Committee on Sept. 17. -
In a livestreamed meeting Tuesday afternoon, Health Secretary Robert F. Kennedy Jr. drew a dark portrait of the state of America’s health while addressing the MAHA Commission’s most recent report, which includes plans to research potential links between vaccines and rising rates of chronic disease. -
Ori Biotech’s CEO said the prioritization of review by FDA, coupled to the impact of the technology, could shave up to three years off development timelines. -
While a win for consumers, the regulatory action did nothing to stem the manufacture of compounded versions of the popular obesity drugs that are made by Novo Nordisk and Eli Lilly. In fact, the FDA seems to be signaling that “some level of compounded product is acceptable,” according to BMO Capital Markets.
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Whether they’re newly minted managers or C-suite executives, effective managers often lead with empathy, intellectual curiosity and vulnerability.
Generative AI could enhance and accelerate the way people work on clinical trials. In this Q&A, a management consultant shares his insights on benefits, risks and more.
While many describe California as having a tough life sciences market, there’s some optimism that employment opportunities will improve soon, according to California Life Sciences President and CEO Mike Guerra.
Looking for a quality control job? Check out these nine companies hiring life sciences professionals like you.
Companies will look at job candidates’ LinkedIn profiles, so make sure yours is strong, from the summary of your expertise and qualifications to testimonials from colleagues.
HOTBEDS
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REPORTS
After a tumultuous 2022, life science employers are settling into their hiring goals for 2023. Though they may be hiring at lower volume, the majority of organizations are still actively recruiting.
Economic turbulence has persisted into 2023 and the life science industry is certainly not immune. How are organizations juggling business needs, budgets, recruitment and retention?
Following a tumultuous start to 2023, layoffs have cooled off though recruiting activities have been significantly impacted by the economic environment.
CANCER
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Krystal Biotech’s decision follows the FDA’s rejection last month of Replimune’s RP1, which works similarly to Krystal Biotech’s KB707. The biotech said this has introduced “heightened uncertainty” regarding a potential accelerated pathway for the candidate. -
The FDA recommends that companies use overall survival as a primary endpoint for clinical trials where feasible. The new guidance follows the surprising return of CBER Head Vinay Prasad, who has previously argued for prioritizing OS. -
The platform strategy of using one molecule to target an underlying biological pathway to address many different diseases can be a goldmine for smaller companies. But it also has a unique set of challenges. -
VantAI will use its machine learning capabilities to identify novel target-effector pairs that Halda can use in designing its bifunctional small-molecule drugs. -
The Department of Health and Human Services’ mRNA pullback only applies to their use in upper respiratory disease, according to Secretary Robert F. Kennedy Jr.
NEUROSCIENCE
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The reprioritization effort will help AC Immune extend its cash runway through the third quarter of 2027. -
Health Secretary Robert F. Kennedy Jr. will appear before the Senate Finance Committee Thursday, ahead of a vaccine advisory committee meeting later in September. Meanwhile, deal-making appetite appears healthy, and the weight loss space continues generating clinical data and other news.
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Some of the biggest SPACs from the industry’s pandemic-fueled heyday are no longer on the market. -
Novartis is licensing ARO-SNCA, a preclinical siRNA therapy for synucleinopathies, a group of neurodegenerative disorders including Parkinson’s disease. -
While the approval of Leqembi Iqlik bodes well for Biogen and Eisai’s planned application for a subcutaneous induction regimen next year, its financial impact remains “uncertain,” as potentially higher revenues from the injection could be offset by steeper costs of production, according to Jefferies.
CELL AND GENE THERAPY
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In late May, a patient died after receiving Rocket Pharmaceuticals’ investigational gene therapy for Danon disease, spurring the hold. After lowering the dose and changing the regimen of immune modulators patients receive, the company has received FDA clearance for the trial to continue. -
The FDA has postponed its decision date for Regenxbio’s Hunter syndrome gene therapy to review additional longer-term clinical data for the asset. -
Kriya is advancing a host of gene therapies for a wide variety of chronic diseases, including geographic atrophy, trigeminal neuralgia and type 1 diabetes. -
Prasad Returns, Delany Departs, Lilly’s Weight Loss Pill Disappoints and Sarepta’s Fallout ContinuesCBER 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.
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Jefferies analysts said these detailed safety outcomes confirm the gene therapy’s positive risk/benefit profile in ambulatory patients with Duchenne muscular dystrophy.
