Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 for Pompe disease.
Praxis Precision Medicines has also announced a “successful” pre-NDA meeting with the FDA for its essential tremor drug candidate ulixacaltamide, for which an approval application is slated for early 2026.
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s Phase I/II studies of AMT-130 are “unlikely” to provide the primary evidence to support a biologics license application.
Writing in separate editorials in two leading medical journals, former chiefs of federal scientific agencies issued warnings about the changes being proposed to vaccine frameworks by current officials.
The partnership will focus on Crescent’s PD-1/VEGF inhibitor CR-001 and Kelun-Biotech’s SKB105, both of which the companies plan to push into Phase I/II development for solid tumors early next year.
Companies who run their early-stage clinical development outside the U.S. would “experience higher fees” according to an FDA proposal made during the negotiation process for the eighth cycle of the user fee program.
FEATURED STORIES
Last month, “historic positive results” from uniQure’s gene therapy snapped the Huntington’s community out of years of failure. As the biotech prepares to submit for FDA approval, BioSpace looks at four more candidates on the near horizon.
Pivotal results from uniQure’s gene therapy for Huntington’s disease have brought new light to patients who have known only disappointment in recent years—but one expert worries that communication of the results is creating “false expectations.”
M&A is back, the S&P XBI is rising again, a biotech pulled off an IPO and positive data is pulling in investors again. This may just be the industry’s new normal.
AI is changing the nature of leadership in biopharma. Here’s how executives can not only adapt, but lead the way.
The company cut back in areas while investing in internal and external opportunities to offset the loss of exclusivity on a product that until recently accounted for 20% of innovative medicine sales.
To tailor cancer therapies to individual patients, Moderna, BioNTech and other companies are rethinking how they optimize manufacturing schedules and resources.
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
Although U.S. full-time employees worked an average of 42.9 hours weekly last year, according to Gallup, that wasn’t true for most BioSpace LinkedIn poll respondents. A Karius HR executive discusses a few potential reasons for the longer workweeks.
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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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The acquisition will give BioCryst an investigational injectable drug for hereditary angioedema, potentially complementing its FDA-approved oral drug Orladeyo. -
AviadoBio will have the option to exclusively license UGX-202, a vision-restoring gene therapy for the rare eye condition retinitis pigmentosa. -
Novo will add Akero’s efruxifermin to its MASH portfolio, which includes Wegovy after the GLP-1 gained an FDA nod in the indication earlier this year. -
The centerpiece of the deal is orelabrutinib, a BTK inhibitor in late-stage development for multiple sclerosis that Biogen once paid $125 million for but abandoned after less than two years of testing. -
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.
WEIGHT LOSS
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Kailera’s lead asset, KAI-9531, elicited an average weight loss of more than 17% in a Phase III study in China. The biotech expects to launch a global late-stage program for the drug this year. -
The U.S. government remains shut down, with the FDA closed for new drug applications until further notice; cell and gene therapy leaders gather for the annual meeting in Phoenix with the field in a state of flux; Pfizer and Amgen will make drugs available at a discount as President Donald Trump’s tariffs still loom; and new regulatory documents show how Pfizer beat out the competition for Metsera. -
Skye Bioscience’s nimacimab fell short of investor and company expectations, but showed encouraging weight-loss results when combined with Wegovy, according to analysts at William Blair. -
M&A headlined for a second straight week as Genmab acquired Merus for $8 billion; Pfizer strikes most-favored-nation deal with White House; CDER Director George Tidmarsh caused a stir with a now-deleted LinkedIn post; GSK CEO Emma Walmsley will step down from her role; and uniQure’s gene therapy offers new hope for patients with Huntington’s disease.
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MET-097i’s mid-stage performance “bodes well” for Pfizer’s proposed buyout of Metsera, according to BMO Capital Markets, a deal centered heavily on the investigational GLP-1 drug.
POLICY
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A complex state vs. federal regulatory scheme allows drug compounders to advertise drugs without disclosing risks like a pharma company must do. Experts say it’s time for the FDA to crack down. -
While the FDA is trumpeting this new initiative as “sweeping reforms” to the way drug companies can advertise, experts say the regulator is going after a problem that doesn’t exist. -
The FDA has vowed to fix a pharma ad loophole—but they’re targeting the wrong one. -
Like the first batch of appointees to the CDC’s vaccine advisory committee, several of the new panelists have documented histories of vaccine and COVID-19 skepticism. -
Some observers see risks to becoming over-reliant on local facilities, noting the potential need for trade partners if domestic production is disrupted.
Massachusetts’ biopharma jobs increased 2.6% in 2023, according to the MassBio Industry Snapshot. Whether the state’s jobs grow in 2024 remains to be seen based on this year’s layoffs and seemingly slowed hiring based on BioSpace data.
The federal judge’s decision Tuesday said the Federal Trade Commission exceeded its statutory authority in implementing a final rule aimed at restricting noncompete clauses.
Massachusetts’ new salary range transparency law can be a tool for companies looking to attract biopharma professionals while also helping candidates and current employees improve their job searches and salaries, according to two experts.
By building and nurturing a strong personal brand, you can benefit in multiple ways, including enhancing your credibility, attracting opportunities and inspiring investor confidence.
To avoid being laid off, a third of biopharma professionals would take a pay cut and nearly a quarter would take a demotion, according to BioSpace LinkedIn polls. We spoke to several professionals about their layoff experiences and what they would—and wouldn’t—have done to keep their jobs.
Plus, learn how to handle vacation time in a new job and navigate the stress of a recent promotion to leadership.
HOTBEDS
REPORTS
In the final instalment of our Diversity in Life Sciences series, BioSpace provides life sciences organizations with practical solutions and benchmarking data to strengthen their DEI initiatives.
BioSpace surveyed industry employers and professionals to understand what to expect from the recruitment market in 2022. What do professionals want? How difficult will it be to recruit new talent?
Get up to speed with BioSpace’s data with up-to-date info about retention, layoffs, “quiet quitting” and projections for 2023.
CANCER
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Moving forward, Innate will focus on the clinical development of its antibody-drug conjugate IPH4502, the lymphoma candidate lacutamab and the AstraZeneca-partnered monalizumab. -
The White House is clamping down on pharma’s ability to buy new molecules from Chinese biotechs; Sanofi, Merck and others abandon the U.K. after the introduction of a sizeable levy; Novo CEO Maziar Mike Doustdar lays off 9,000 while the company presents new data at EASD; Capsida loses a patient in a gene therapy trial; and CDER Director George Tidmarsh walks back comments on FDA adcomms.
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On the FDA’s docket for the back half of September is Merck’s proposed subcutaneous formulation of its blockbuster cancer drug Keytruda. -
Cullinan Therapeutics and Taiho Oncology’s zipalertinib elicited promising response rates in two mid-stage studies of non-small cell lung cancer patients with typical and uncommon EGFR mutations. -
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.
NEUROSCIENCE
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LB Pharma landed on the Nasdaq Thursday, with 3 million additional shares sold than expected. -
Capsida has yet to disclose the exact cause of death. The patient had received the gene therapy CAP-002 for a type of epilepsy. -
Presenting at the World Sleep Congress 2025, the Dublin-based company’s Phase II study bested Takeda drug in both efficacy and safety. -
Shares of Rapport Therapeutics popped Monday morning after Phase IIa data for RAP-219 exceeded analyst and Wall Street expectations, reducing seizures by almost 78% in patients with drug-resistant focal onset seizures. -
KER-0193 is a modulator of ion channels connected to autism spectrum disorder. The FDA bestowed orphan drug and rare pediatric drug designations on the candidate earlier this year.
CELL AND GENE THERAPY
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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. -
As AAV9 and CRISPR programs navigate safety, delivery and scalability hurdles, small molecules offer a deployable, scalable bridge, complementing genetic approaches and accelerating meaningful impact for patients with Duchenne muscular dystrophy. -
It can cure deadly diseases, save long-term healthcare costs and transform lives. But the U.S. insurance system still isn’t ready to pay for it. -
Vertex Pharmaceuticals commits $45 million upfront to leverage Enlaza Therapeutics’ War-Lock platform to create drug conjugates and T cell engagers for autoimmune diseases and gentler conditioning for sickle cell/beta thalassemia gene-editing therapy Casgevy. -
Appia Bio’s shutdown continues an unfortunate trend this year that has seen biotech closures nearly every month.
