After launching in April, Hunter syndrome drug Avlayah surprised analysts with a strong first quarter, as patients rush to get access to Denali Therapeutics’ new option.
Follow along as BioSpace tracks job cuts and restructuring initiatives.
Analysts expect Replimune’s newly approved Tudriqev to reach $1.3 billion in peak sales in 2035.
Job postings increase and competition dips, according to BioSpace data.
In November 2025, one patient died after receiving Intellia Therapeutics’ investigational gene silencer for transthyretin amyloidosis.
The Phase 2 study was small and was not designed to establish the statistical difference between Vistagen’s drug candidate and placebo, according to Vistagen. The biotech plans to meet with the FDA to determine a registrational path for the asset.
FEATURED STORIES
Three years after Qalsody was approved by the FDA as the first drug to target a genetic cause of ALS, some patients are reporting not only disease stabilization but also improvement of symptoms.
Despite the recent emergence of a new drug class, schizophrenia remains one of the most challenging neuropsychiatric diseases to treat—and Cobenfy’s first steps into the market indicate there is still room to grow.
The shift from ex vivo to in vivo gene delivery is rewriting the rules of viral vector manufacturing, forcing developers to rethink scale, purity, cell lines and analytics as vectors themselves become the drug. AGC Biologics is betting on stable producer lines, next‑gen purification and deeper characterization to meet the far stricter demands of in vivo therapies.
While there are several FDA-approved psychiatric medicines on the market, many patients still struggle to achieve lasting symptom relief. Psychedelics could help fill this gap.
The FDA has been trying to mend fences with rare disease drug developers and patient groups since the departure of former Commissioner Marty Makary three months ago. But advisory committee meetings last week for Replimune and Capricor Therapeutics only created more confusion and frustration.
Pharma is definitely interested in making more deals. But what biotechs will come under the magnifying glass? BioSpace takes a look at companies that analysts see as near-term targets for buyouts.
UPCOMING EVENTS
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Wednesday, August 26, 2026 11am EDT·Virtual
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Tuesday, September 1, 2026 8am–5:30pm PDT·San Francisco, California
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Wednesday, September 2, 2026 8am–6:10pm PDT·San Francisco, California
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Thursday, September 3, 2026 8am–6:10pm PDT·San Francisco, California
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Tuesday, September 8, 2026 8am–6:15pm PDT·San Francisco, California
LATEST PODCASTS
In this episode of Denatured, you’ll hear from Hans Eriksson, chief medical officer at FundaMental Pharma and Steve Levine, chief patient officer at Compass Pathways. We explore why treatment-resistant depression remains so difficult to address, and how new approaches—from rapid-acting antidepressants to psychedelic therapy—may reshape the treatment paradigm.
The industry has been abuzz since the weekend when talks of a potential mega-merger between Bristol Myers Squibb and AstraZeneca were reported. Meanwhile, Pfizer, Merck and others release second quarter earnings, with some disclosing pipeline discontinuations and cost-cutting measures. Finally, all eyes turn to the FDA in the wake of heated advisory committee meetings for Capricor Therapeutics and Replimune.
In this episode of Denatured presented by Arvinas, Jennifer Smith-Parker speaks to Arvinas President and CEO Randy Teel about the strategic discipline and leadership required to successfully refocus a company on an early-stage pipeline after a lead asset is outlicensed, discontinued or otherwise falls away.
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SPECIAL EDITIONS
Who is the highest paid CEO in all of pharma? In this special edition, BioSpace examines top paid CEOs and their pay packages.
BioSpace examines how the FDA approval of Eli Lilly’s oral obesity drug Foundayo has ignited a key race with Novo Nordisk.
Opportunities increased by the end of the first quarter, according to BioSpace data.
DEALS
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Braveheart Bio has emerged on the Nasdaq with an upsized expected initial public offering—marking the public entrance of the twenty-second biotech this year. -
Reuters has deflated rumors that AstraZeneca and Bristol Myers Squibb are discussing a possible merger, letting the air out of industry-wide speculation over what could have been the largest deal in pharma history. -
BioSpace is launching an IPO dashboard to help the industry keep tabs on U.S. biotechs that have taken the public plunge in 2026. -
Asked on a second quarter earnings call if Pfizer could direct more money to M&A amid a feeding frenzy across the pharma industry, CEO Albert Bourla defended his strategy. -
While both drugmakers face looming patent cliffs, analysts say joining forces could help Bristol Myers Squibb replenish its eroding portfolio—while the strategic case for AstraZeneca seems to be more mixed.
WEIGHT LOSS
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Foundayo clocked $98 million in sales in Q2, the first quarter it was available, compared to Novo’s $355 million for the same sales period. -
A legal dispute between the FDA and Eli Lilly over whether the highly anticipated obesity candidate retatrutide is indeed a biologic has come to a head as the company prepares to submit the medicine for approval. -
Eli Lilly’s shares rose nearly 5% to $1,170 apiece in premarket trading as the company reported $23 billion in revenue for the second quarter. The company’s market cap is $1.02 trillion as of the end of July. -
Amgen has discontinued Phase 1 obesity candidate AMG 513, leaving MariTide as its sole weight loss asset. Meanwhile, executives said the company’s late-stage pipeline is largely full and future business development will focus on earlier-stage opportunities. -
Novo Nordisk’s shares fell 6% on Tuesday afternoon after its second quarter earnings report showed a slight miss for the Wegovy pill vs. consensus.
POLICY
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A Congressional advisory panel recommends the U.S. government put supportive policies in place for rare disease drug development, such as a finalized platform designation from the FDA and the allowance of novel trial designs for small patient populations. -
Having started to publish CRLs without consulting the public, the FDA is under pressure to change course amid concerns it failed to openly and fully evaluate a complex issue. -
The FDAs enforcement action against Purolea Cosmetics Lab offers an early look at how the agency expects AI-generated work to fit within existing quality systems, with experts saying human oversight and quality controls remain essential. -
The agency’s finalized recommendations for clinical testing of psychedelic compounds for mental health could pave the way for companies like Compass Pathways, whose psilocybin-based therapy for treatment-resistant depression could win approval as early as this year. -
Through the proposal, the FDA could clear barriers to distributed manufacturing approaches intended to enhance emergency preparedness and supply chain resilience.
Working at a Big Pharma has its benefits, but there are limitations too. SERB Pharmaceuticals’ Thomas Kolaras discusses how moving to a midsize company creates new opportunities for sharpened thinking and leadership.
AI tools can help job seekers write resumes and mass apply to open positions, but those aren’t the only, or best, ways to use them. A career coach shares how she advises clients to make the most of AI during the application and interview process.
The choices that change a career often look like extra work at the time. Executive coach Angela Justice examines why the projects, questions, relationships and conversations people almost dismiss can become the ones they later recognize as turning points.
Controlling CEOs, manipulative middle managers and high-performing jerks can damage employees’ trust in employers and motivate them to hit the job market. Kaye/Bassman’s Michael Pietrack discusses the problematic behaviors executives must watch for in the workplace.
Even something as simple as “Tell me about yourself” can trip up biopharma professionals during job interviews. Two recruiting experts discuss what candidates should and shouldn’t say when answering five specific questions.
Rather than getting hung up on what to call DEI in the workplace, leaders should take three specific actions to help their employees embrace and engage with it. Companies and the patients they serve will benefit.
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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The industry has been abuzz since the weekend when talks of a potential mega-merger between Bristol Myers Squibb and AstraZeneca were reported. Meanwhile, Pfizer, Merck and others release second quarter earnings, with some disclosing pipeline discontinuations and cost-cutting measures. Finally, all eyes turn to the FDA in the wake of heated advisory committee meetings for Capricor Therapeutics and Replimune. -
Pathos AI could pay more than $2 billion to a Jiangsu Alphamab Biopharmaceuticals subsidiary for a late-stage bispecific antibody-drug conjugate for breast cancer, while the financial details of the AstraZeneca agreement were not disclosed. -
If AstraZeneca and Bristol Myers Squibb were to successfully become one company, analysts say it could reset the current deal environment, which has picked up greatly in the first half of the year. -
UniQure is ready to put its Huntington’s disease gene therapy through an advisory committee meeting—even as the broader rare disease space has been put on edge by recent FDA scrutiny of assets by Replimune and Capricor. -
The FDA’s Cellular, Tissue and Gene Therapies advisory committee voted 10 to 3 in favor of Replimune’s immunotherapy for advanced melanoma in combination with Bristol Myers Squibb’s Opdivo, but trial design was a major sticking point as the panel deliberated.
NEUROSCIENCE
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In this episode of Denatured, you’ll hear from Hans Eriksson, chief medical officer at FundaMental Pharma and Steve Levine, chief patient officer at Compass Pathways. We explore why treatment-resistant depression remains so difficult to address, and how new approaches—from rapid-acting antidepressants to psychedelic therapy—may reshape the treatment paradigm.
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Supernus Pharmaceuticals and Indivior Pharmaceuticals are outlining a combined expected $2.2 billion in annual revenue, plus $125 million in cost savings that will include “natural redundancies.” -
The Phase 3 failure of AstraZeneca and Ionis Pharmaceuticals’ antisense therapy in transthyretin amyloid cardiomyopathy last month left the space reeling—and readjusting. BioSpace looks at five contenders and where they currently stand. -
AbbVie investors’ sky-high expectations led to a 4% share decline on Friday as the company reported more modest consensus beats than expected. -
Strong quarterly results are prompting Bristol Myers Squibb to lift its 2026 outlook, though the company is facing another delay for key Cobenfy Alzheimer’s data and a pushed-back readout for milvexian.
CELL AND GENE THERAPY
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As part of the collaboration, Johnson & Johnson will have an exclusive option to acquire Sail Biomedicines for an additional $2.58 billion. -
ElevateBio will be the primary source of Kyverna’s autologous CAR T cell therapy for anticipated U.S. and global launches in autoimmune indications. -
In briefing documents published Monday ahead of an advisory committee meeting for deramiocel, the FDA disagreed with Capricor’s assertion that a Phase 3 trial met its primary outcome measures. -
Safety will be the key determinant of in vivo CAR T therapies, according to H.C. Wainwright’s Mitchel Kapoor, who noted that convenience and efficacy won’t be enough to impress investors in this space. Eli Lilly, AbbVie, AstraZeneca and more are up for the challenge. -
Poised to become the first company to achieve a regulatory filing for a cell therapy in autoimmune disease, Kyverna Therapeutics is building a pipeline-in-a-product to change the treatment paradigm.
