After revoking Sarepta’s award in July and awarding one to Krystal last month, the FDA’s platform technology designation program appears to be back on track. These six biotechs could be on the regulator’s radar.
While expressing disappointment, William Blair analysts were unsurprised by the Phase II failure, having assigned the VISTA study a high level of risk given the “mixed” performance of a similar drug in a prior multiple sclerosis study.
After GSK subsidiary Tesaro filed a lawsuit Thursday claiming that AnaptysBio breached “certain requirements” under their 2014 license agreement involving GSK’s Jemperli, Anaptys responded Friday morning.
Amid an aggressive savings push, Moderna has cut three assets and taken on a loan to increase “flexibility.”
A new analysis from Jefferies shows that drugs receiving breakthrough designations sail through the regulatory process more quickly, on top of frequently winning approval.
Aspen is now also considering the possibility of an initial public offering next year in an effort to bring its cell therapy to the market.
FEATURED STORIES
A new analysis from SRS Acquiom puts into perspective the headline values seen when a company announces a backloaded M&A deal. Biotechs have much on the line when they agree to deals with massive potential but little upfront.
Talks between pharma and successive U.K. governments have failed to deliver the market access terms that the industry wants, contributing to a pullback in investment.
Companies are moving from using AI for distinct operations to applying the technology for control and optimization of the whole production process.
Reshoring generic pharmaceutical production is essential in today’s era of geopolitical instability and heightened awareness surrounding national health security. And it is possible—if done right.
The FDA in September issued two rejections for spinal muscular atrophy therapies—both linked to manufacturing problems—and granted approvals in Barth syndrome and for a subcutaneous version of Merck’s Keytruda that could be key to the blockbuster’s future earnings.
By publishing complete response letters as soon as they are issued to drug sponsors, the FDA is expanding transparency in a way that, while positioned as a public health measure, also grants investors greater visibility into regulatory decisions. Experts question whether this is the agency’s proper remit.
LATEST PODCASTS
H2 2025 catalysts to watch, biopharma implications of President Trump’s tax law, KalVista’s new hereditary angioedema drug that Marty Makary reportedly tried to reject, another lawsuit aimed at Health Secretary Robert F. Kennedy Jr. and a plea from patients with ALS for access to BrainStorm’s NurOwn.
The high court sides with HHS on HIV PrEP drugs; Health Secretary RFK Jr.’s newly appointed CDC vaccine advisors discuss thimerosal in flu vaccines, skip vote on Moderna’s mRNA-based RSV vaccine; FDA removes CAR T guardrails; AbbVie snaps up Capstan for $1.2B to end first half; and psychedelics take off again with data from Compass and Beckley.
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the FDA’s first draft guidance for AI in drug development, published in January 2025, with Archana Hegde, senior director, pv systems and innovations at IQVIA.
Job Trends
Carisma’s second workforce reduction this year likely leaves the company with 44 full-time employees as turns its focus to developing therapies for fibrosis, oncology and autoimmune diseases.
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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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Analysts said the deal with Novo was likely giving Hims “‘credibility’ or increased consumer traffic,” adding that the “litigation risk is back on the table” now that the Danish pharma has stepped away. -
The deal marks an end for CAR T company Cargo Therapeutics, which has been slashing its workforce and cutting programs since the January decision to halt its lead candidate for a certain type of aggressive large B cell lymphoma. -
After the FDA rejection of Zurzuvae in one type of depression and the triple failure of neuro asset dalzanemdor, Sage was searching for a path forward at the end of December 2024. Biogen CEO Chris Viehbacher spied a possible deal, but the smaller company wasn’t interested. -
Why did two private equity firms with more than $460 billion under management want a little old gene therapy biotech called bluebird bio? We wanted to know. -
Big Pharmas like Eli Lilly, Sanofi and Novartis headed back to the dealmakers table multiple times, with 32 total deals counted across the industry for the first half.
WEIGHT LOSS
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In December 2024, Teva also secured FDA approval for the other liraglutide brand Victoza, indicated for type 2 diabetes. -
Novo Nordisk has brought on other cardiometabolic collaborators this year, including United Laboratories International and Deep Apple Therapeutics. -
Eli Lilly drops a second Phase III readout for orforglipron; AbbVie committed to the psychedelic therapeutics space with the $1.2 billion acquisition of Gilgamesh’s depression asset; the CDC taps vaccine skeptic Retsef Levi to lead its COVID-19 immunization working group; and the FDA prioritizes overall survival in cancer drug development. -
While Truist Securities analysts said the results from the ATTAIN-2 trial leave “room for competition,” they also pointed to a manufacturing advantage that could unlock a “double-digit billion dollar opportunity” for Eli Lilly. -
Closely watched data from Eli Lilly and Viking Therapeutics this month have reignited the discussion around oral weight-loss drugs—and their ultimate place within the anti-obesity medication market.
POLICY
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During the COVID-19 pandemic, Health Secretary Robert F. Kennedy Jr.—along with FDA Commissioner Marty Makary and CBER Director Vinay Prasad—argued against vaccine mandates, partly because they limited medical choice. This week, the FDA under their leadership approved updated COVID-19 vaccines with restrictions that do the same. -
Health Secretary Robert F. Kennedy Jr. will testify before the Senate Finance Committee on Sept. 4, following the ouster of CDC Director Susan Monarez and tapping of HHS Deputy Secretary Jim O’Neill as her interim replacement. -
The CDC director—the first to be confirmed by the Senate under new legislation—has been ousted after less than a month following internal unrest regarding new, more restrictive approvals for updated COVID-19 vaccines, according to multiple sources. -
Scott Gottlieb, who served as FDA commissioner during the first Trump administration, wrote in a JAMA editorial that China is speeding drugs to market and could potentially surpass the U.S. in the innovation game. -
The MIT professor of management, who already sits on the CDC’s revamped immunization advisory committee, is a known skeptic of vaccines, particularly mRNA technology.
A recent legal decision could signal the ultimate demise of the FTC’s final rule banning most noncompete clauses. A biotech talent expert discusses how that affects biopharma job searches.
For the second quarter of 2024, there were 25% fewer jobs posted live on BioSpace compared to the same quarter of 2023. The year-over-year job response rate rose from 14.6% to 15.3%.
When moving to a new role at the same company, it’s important to create a transition plan, understand your responsibilities and expectations and get to know your new team.
It’s important to maintain anonymity when seeking your next executive role, as your departure could impact other employees and the organization.
Interviews can be difficult in any field, but especially in a competitive industry like the life sciences. Read on to discover the best way to answer salary interview questions in our guide.
Plus, what to do if your offer is paused and how to manage work anxiety.
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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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.
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In an open letter, 22 experts who designed and ran Replimune’s Phase III IGNYTE trial answered the FDA’s issues, as outlined in the complete response letter for the melanoma candidate RP1. -
According to reporting from multiple outlets, Richard Pazdur, head of the Oncology Center of Excellence at CDER, opposed the consensus opinion of CBER staff to approve the drug. Replimune’s stock has dropped precipitously since the rejection. -
The company expects that the U.S. COVID-19 vaccination rate will be “maybe a couple of points lower” than the prior level of around 20% but that pricing and Comirnaty’s market share will hold steady. -
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.
NEUROSCIENCE
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The small molecule, vatiquinone, had already flunked a Phase III trial, but the company pushed ahead with an approval bid anyway. -
Waltham, Massachusetts–based Skyhawk Therapeutics has been collecting collaborations with larger companies in spades since launching in 2018. -
Arguably the FDA’s most anticipated decision this month is for a subcutaneous induction formulation of Biogen and Eisai’s Alzheimer’s drug Leqembi, which, according to Eisai, could “help reduce the burden on healthcare professionals and patients.” -
Praxis’ vormatrigine reduced seizures by 56.3%, an effect size that, according to analysts at Truist Securities, exceeds that of its closest competitors. -
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.
CELL AND GENE THERAPY
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The strategic reprioritization comes after the company hit two major hurdles in the past year, including a clinical hold for an investigational gene therapy and an FDA rejection for its lead asset. -
CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an anonymous senior FDA official. Given Elevidys’ full approval, however, experts told BioSpace this path would set up a length legal battle between the regulator and Sarepta Therapeutics. -
Second-quarter earnings come amid many high-level challenges for the biopharma industry. How will these five closely watched biotechs fare? -
Dispatch seeks to address two main challenges of immunotherapies in solid tumors: the lack of a target and the immunosuppressive tumor environment. -
The voluntary pauses follow two patient deaths associated with the Duchenne muscular dystrophy gene therapy.
