UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms
UniQure’s one‑time gene therapy AMT‑130 showed a 75% slowdown in Huntington’s disease progression in its Phase 1/2 trial, prompting expectations for a BLA filing in early 2026. The FDA, however, reversed its earlier stance and now requires a sham‑controlled Phase 3 study, citing insufficient primary evidence. UniQure intends to request a Type B meeting to explore feasible, ethical trial designs while analysts argue a path without a full Phase 3 may exist. The company’s next steps hinge on 48‑month data and stakeholder consensus on risk‑benefit balance.
FDA’s Hoeg Reportedly Trying To Hire Friend, Fellow Antidepressant Skeptic
The FDA’s acting CDER director Tracy Beth Høeg is reportedly moving to hire Adam Urato, a maternal‑fetal specialist known for his skepticism of antidepressants during pregnancy. Urato has publicly called for stronger warnings on SSRIs and has filed a citizen...
PepGen’s Mid-Stage Myotonic Dystrophy Study Hit With ‘Surprise’ Pause
PepGen’s Phase 2 FREEDOM2 trial in myotonic dystrophy type 1 received a partial FDA clinical hold due to concerns over a sub‑chronic mouse study that showed blood‑pressure changes. The agency did not question the Phase 1 human data, and the company continues dosing...
Roche Broadens Global Clinical Trial Footprint With $480M+ South Korea Pledge
Roche announced a $480 million, five‑year investment in South Korea to build a national clinical‑trial ecosystem, fund R&D infrastructure, and train specialized personnel. The pledge also includes support for domestic biotech startups aiming for global markets. This move aligns with a...
Keeping the Door Open: Many Would Return to Companies That Laid Them Off
A BioSpace LinkedIn poll of 1,267 biopharma professionals revealed that 51% would consider returning to the employer that laid them off. Tight labor markets, limited local opportunities, and the appeal of consulting contracts drive this willingness. Respondents cite generous severance...
UniQure’s Delay, REGENXBIO’s Rejection Explained, Sarepta’s Ingram Steps Down, More
The FDA issued a complete‑response letter to UniQure, requiring a randomized, double‑blind, sham‑surgery Phase 3 trial for its Huntington’s disease gene therapy, and similarly rejected REGENXBIO’s Hunter syndrome candidate over study design flaws. Regulatory experts warned that the agency’s decision‑making appears...
Teva Bags $400M Backing From Blackstone To Advance Sanofi-Partnered Drug
Teva Pharmaceutical has secured a $400 million financing commitment from Blackstone Life Sciences to advance duvakitug, a TL1A‑blocking monoclonal antibody co‑developed with Sanofi for inflammatory bowel disease. The capital will be disbursed over four years, granting Blackstone low‑single‑digit royalties and eligibility...
Bespoke Startup EveryONE Folds 1 Week After Plausible Mechanism Guidance
EveryONE Medicines announced its shutdown just a week after the FDA released draft guidance for its new bespoke pathway targeting personalized genetic therapies. The guidance requires each individualized drug to be submitted as a separate application, a burden that the...
Rethinking Biotech’s Valley of Death as Federal Grants and Family Offices Step In
Federal budget approvals for 2026 dramatically increase nondilutive capital for biotech, raising NIH funding to $49 billion and expanding programs like CDMRP and ARPA‑H. Seed‑stage companies are responding, with 38 % planning to rely on grants and other non‑equity sources. Simultaneously, mission‑driven...
Keytruda Hangs On to Best Seller Crown as GLP-1s Gain Ground
Merck’s Keytruda remained pharma’s top‑selling drug in 2025, generating $31.7 billion and a 7 % year‑on‑year increase. However, the combined sales of GLP‑1 franchises—Eli Lilly’s tirzepatide line and Novo Nordisk’s semaglutide portfolio—totaled over $36 billion, overtaking Keytruda for the first time. The GLP‑1 market is...
After Exiting 2seventy, Chip Baird Took a 180 to Food Allergy With Newly Launched Poplar
Chip Baird, after selling 2seventy bio to Bristol Myers Squibb, launched Poplar Therapeutics as CEO in early 2025. The company secured a $50 million Series A and an additional $45 million extension to fund Phase 1 trials of its anti‑IgE antibody PHB‑050. PHB‑050 aims to...
Webinar: The Future Is Collaborative: Transforming Clinical Trials
A MEDSIR‑hosted webinar titled “The Future is Collaborative: Transforming Clinical Trials” examined how collaborative‑initiated trials and investigator‑initiated trials (IITs) are reshaping oncology research. Speakers Dr. Javier Cortés and Dr. Antonio Llombart‑Cussac, both leading breast‑cancer oncologists, discussed their roles in recent...
Boehringer Ingelheim Axes MASH Pact With OSE After Disappointing Mid-Stage Data
Boehringer Ingelheim has terminated its metabolic dysfunction‑associated steatohepatitis (MASH) collaboration with OSE Immunotherapeutics after an exploratory Phase 2 trial failed to meet its primary endpoint. The original 2018 deal included a €15 million upfront payment and up to €1.1 billion in milestone potential,...
How North Carolina Attracted Amgen and Roche To Become a Next-Gen Obesity Drug Production Hotspot
North Carolina’s Holly Springs is becoming a hub for next‑generation obesity drugs as Amgen and Roche commit more than $3.5 billion to build manufacturing capacity. Amgen’s $1.55 billion rollout includes a new drug‑substance plant and a second facility slated for 2032, while Roche’s...
Navigating the FDA After the Storm To Advance Drug Candidates
After a wave of senior departures and a 3,500‑position cut, the FDA entered 2026 with a largely inexperienced workforce, raising uncertainty for drug developers. The agency’s new AI guidance, released in January, outlines best practices but emphasizes human oversight and...
BioAtla Axes 70% of Staff, Explores Strategic Options
BioAtla announced it is slashing roughly 70% of its staff as it evaluates strategic alternatives, leaving only essential personnel for the review. The move follows a 30% reduction last year and leaves the company with about $7.1 million in cash, down...
Merck, Pfizer’s Drug Combo ‘Rewrites the Standard of Care’ in Bladder Cancer
Merck and Pfizer’s Keytruda‑Padcev doublet delivered a dramatic survival advantage in the Phase 3 EV‑304 study of muscle‑invasive bladder cancer. The regimen cut the risk of disease progression, recurrence or death by 47% compared with gemcitabine‑cisplatin and lowered overall‑mortality risk by...
FDA Action Alert: BMS, GSK, Aldeyra and More
The FDA will render decisions this March on five high‑profile drug applications, including two delayed from last year. Bristol Myers Squibb seeks a psoriatic arthritis label for Sotyktu, backed by Phase 3 data showing a 54.2% ACR20 response. Aldeyra, Rhythm, GSK...
Boehringer Wins Speedy Lung Cancer Approval Under Commissioner’s Priority Program
The FDA granted accelerated approval to Boehringer Ingelheim’s oral HER2 kinase inhibitor Hernexeos six weeks after the company filed its application, marking the second approval under the new Commissioner’s National Priority Voucher (CNPV) program. The decision expands Hernexeos use to...
After Sarepta’s Annus Horribilis, Elevidys Sales Expected To Continue Downward Spiral
Elevidys, Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, posted $110 million in fourth‑quarter sales, bringing full‑year revenue to $898.7 million—well below the $1 billion benchmark. Safety incidents in 2025 and a severe flu season have eroded confidence, prompting analysts to project 2026...
FDA’s One Trial Policy Not a Revolution but a Potentially Risky Evolution
The FDA announced it will default to a single pivotal trial for new drug applications, extending a practice long used in oncology and rare‑disease approvals. In 2024, 66% of new molecular entities were cleared based on one trial, signaling a...
ALS Advances Unite Patients and Pharma on Novel Targets, Biomarker Breakthroughs
The latest episode of BioSpace’s Denatured podcast spotlights a growing partnership between ALS patients and pharmaceutical developers, featuring insights from EverythingALS founder Indu Navar and VectorY Therapeutics CMO Dr. Olga Uspenskaya. The discussion highlights how patient‑driven collaborations are accelerating trial timelines...
Atara Climbs Amid Report of FDA Inconsistency Leading to Cell Therapy’s Rejection
Atara Biotherapeutics’ stock jumped 20% after STAT reported that internal FDA inconsistencies may have driven the rejection of its EBV‑positive PTLD cell therapy, Ebvallo. The FDA denied the U.S. application citing insufficient evidence of effectiveness, yet former reviewers said data...
Pfizer Advances Beam’s Gene Editor After Pulling Hemophilia Gene Therapy
Pfizer has signed a global gene‑editing partnership with Beam Therapeutics, reviving its genetics ambitions after withdrawing the hemophilia B therapy Beqvez a year earlier. The agreement includes a $300 million upfront payment and up to $1.05 billion in milestones, granting Pfizer exclusive worldwide...
The Right Fit: Contract Work Finds Increasing Favor Among Employers, Professionals
BioSpace data shows contract biopharma job postings jumped 36.7% from Jan 2025 to Jan 2026 while full‑time listings fell 11.3%. A 2026 employment outlook survey found contract workers now represent 14.3% of respondents, up from 10.6% in 2023. Recruiters say the shift...
10 Companies Hiring in San Francisco and South San Francisco
San Francisco and South San Francisco remain biotech hubs, employing over 113,700 workers according to a Cushman & Wakefield report. Although BioSpace job postings fell sharply in January, listings are now climbing, with ten companies actively hiring. Companies such as AbbVie, Amgen, Eli Lilly and Ideaya...
Patient Death Forces Partial Freeze on MacroGenics’ Gynecologic Cancer Study
A patient in MacroGenics' Phase 2 LINNET trial of the bispecific antibody lorigerlimab suffered grade 4 neutropenia and septic shock, leading to a fatality and prompting the FDA to place a partial clinical hold on the study. The company also reported three...
Lilly Bests Novo Again, Rare Disease Week Goes Regulatory, More CDC Leadership Upheaval
Eli Lilly’s obesity drug Zepbound achieved a 25.5% weight loss in a head‑to‑head Phase 3 trial, outpacing Novo Nordisk’s CagriSema and sending Novo’s shares down 20%. The FDA launched Rare Disease Week, issuing draft guidance for a new Plausible Mechanism Pathway that...
Opinion: Balancing Safety Access in Rare Disease—Lessons From Sarepta
In July 2025 the FDA ordered a complete halt of Sarepta’s gene‑therapy Elevidys after two non‑ambulatory Duchenne muscular dystrophy (DMD) patients died, sparking a market plunge and patient access concerns. Sarepta argues that ambulatory patients, who had been receiving Elevidys...
How LB Rode the Brutal Capital Markets to One of 2025’s Most Successful IPOs
LB Pharma went public in September 2024, pricing its shares at $15 and raising $285 million despite a severely constrained capital market. The IPO followed a Phase 2 trial that showed LB‑102 markedly reduced schizophrenia symptoms, sparking investor demand even as venture...
Cashing In on ‘Creative Fundamentals’ To Fund Rare Disease R&D
The FDA reauthorized its rare pediatric disease priority review voucher program, bolstering a wave of regulatory incentives that include new guidance on plausible mechanisms and a Rare Disease Evidence Principles framework. Yet ultra‑rare indications still struggle to attract traditional venture...
Cut Down on Oncology Drug Waste With Better Forecasting
Oncology drug waste reaches up to 30%, costing over $2 billion annually in the U.S. A 2023 CMS rule now forces manufacturers to refund Medicare for discarded volumes beyond a 10% threshold, adding financial pressure. Traditional forecasting relies on static sales...
Radiopharmaceutical Sector Shapes Supply Chain Around Fast-Decaying Products
Radiopharmaceutical manufacturers must design ultra‑fast supply chains because isotopes decay rapidly, turning each dose into a ticking clock. Companies such as NorthStar and Nucleus mitigate this by locating production near major airports, using private air freight for longer‑half‑life isotopes, and...
Merck Reorganizes Human Health Work Into Two New Units
Merck is reorganizing its Human Health division into two distinct units—Oncology and Specialty, Pharma & Infectious Diseases—to sharpen focus as its flagship immuno‑oncology drug Keytruda approaches patent expiry. Jannie Oosthuizen, formerly head of U.S. Human Health, will lead the Oncology...
REGENXBIO Secures Legal Win in Gene Therapy Patent Battle Vs. Sarepta
An appellate court reversed a lower‑court ruling, siding with REGENXBIO in its patent dispute against Sarepta Therapeutics over an adeno‑associated virus (AAV) vector used in Duchenne muscular dystrophy gene therapy. The judge held that the patented composition, as a whole,...
Novo’s CagriSema Falls to Lilly’s Zepbound in Daring Head-to-Head Test
Novo Nordisk’s experimental obesity drug CagriSema delivered a 23% average weight loss in the 84‑week REDEFINE 4 Phase 3 trial, but fell short of Eli Lilly’s tirzepatide (Zepbound) which achieved 25.5%. The head‑to‑head study failed the primary non‑inferiority endpoint, prompting a 15% drop...
Sickle Cell Gene Therapies Casgevy and Lyfgenia Still Lacking Traction 2 Years In
In February 2024 Vertex and CRISPR Therapeutics launched Casgevy, and bluebird bio (now Genetix Biotherapeutics) launched Lyfgenia as the first FDA‑approved gene therapies for sickle cell disease. Two years later, uptake remains modest—only 64 patients received Casgevy and just over 100...
Beyond Rezdiffra: The 4 Most Promising MASH Pipelines in Play
Madrigal’s Rezdiffra became the first approved therapy for metabolic dysfunction‑associated steatohepatitis (MASH), generating $817 million in revenue within six quarters and prompting a wave of big‑pharma acquisitions focused on FGF21 analogs. In 2025, GSK, Roche and Novo Nordisk spent nearly $10 billion acquiring...
Redefining Obesity Treatment Beyond GLP-1 Limits
BioSpace’s Denatured podcast featured Verdiva Bio’s R&D head Jane Hughes and MitoRx CEO Jon Rees discussing next‑generation obesity therapies that move beyond the limitations of GLP‑1 agonists. They highlighted how GLP‑1 treatments can cause muscle loss and suffer from poor...
9 Companies Hiring Now in Pennsylvania
Pennsylvania’s Greater Philadelphia corridor remains a biotech hotbed, housing over 1,200 life‑science firms including AstraZeneca, GSK and Johnson & Johnson. Eli Lilly announced a new injectable weight‑loss manufacturing plant in the Lehigh Valley, projected to create roughly 850 jobs by 2031....
Extending Scientific Rigor From Bench to Boardroom
Researchers who launch biotech startups often abandon evidence‑based decision‑making once they enter the boardroom, falling prey to technology myopia, base‑rate neglect, and confirmation bias. Their lack of formal training in commercialization, financial modeling, and competitive analysis amplifies these pitfalls, leading...
Korsana Joins Alzheimer's Amyloid Arena With $150M Debut, Next-Gen Antibody
When Senior Leaders Are Asked To Carry Too Much
Biopharma senior leaders are increasingly shouldering operational work left behind by downsizing, stretching their capacity beyond strategic duties. The overload fragments focus, slows decisions, and erodes communication, while burnout remains hidden behind continued presence. As pressure builds, the organization’s decision‑making...
Early-Stage Biotechs Suffer in 2025 As VC Shuns Risk: PitchBook
Venture capital in biopharma rebounded in 2025, delivering $33.8 billion across 1,171 deals, but the capital is increasingly concentrated in late‑stage companies. Early‑stage startups received only 32% of the funding, down from over 40% during the pandemic surge, while median deal...
Ongoing Regulatory Uncertainty Means Innovation Is Now Higher Risk
Regulatory uncertainty is rising as the FDA shifts leadership, adopts a single‑trial approval pathway, and tightens its benefit‑risk framework. While the one‑trial standard promises faster, cheaper market entry, recent surprise complete response letters (CRLs) show that even successful Phase 3 results...
Ionis Paves Its Own Path as Initial Tryngolza Launch Defies Expectations
Ionis Therapeutics announced that its GSK‑partnered antisense drug bepirovirsen achieved a statistically significant functional cure rate of roughly 15‑20% in a Phase 3 trial, far surpassing the 1‑3% rate of existing hepatitis B therapies. The company also reported a strong first‑year launch...
Proliferating Patents, Lawsuits Stave Off Pharmas’ Generic Competitors
A new analysis in Health Affairs Scholar reveals that brand‑name drugmakers are exploiting the Hatch‑Waxman framework through serial patent litigation and continuation patents to extend market exclusivity. By filing multiple overlapping patents, companies can repeatedly sue generic challengers, triggering 30‑month...
Does Vaxart Hold the Key to the Elusive Norovirus Vaccine?
Vaxart is advancing an oral, bivalent norovirus vaccine pill (VXA‑G1.1‑NN) that demonstrated a 30% reduction in infection in a Phase 2b challenge study. The vaccine uses a non‑replicating adenovirus vector loaded with a dsRNA adjuvant to stimulate gut immunity, and a...
As FDA Moves Away From Animal Testing, AI and Organoids Can Shine
The FDA is actively phasing out animal‑testing requirements for certain therapies, a move echoed by the NIH and codified in the pending FDA Modernization Act. This regulatory shift opens the door for artificial intelligence and human‑derived organoid models to become...
Biopharma Must Cope With The Fetal Bovine Serum Squeeze
Biopharma faces a tightening fetal bovine serum (FBS) market as U.S. cattle inventories hit record lows and European disease outbreaks curb supply. Prices have surged more than 300% over five years and release timelines have lengthened from weeks to months...