Gossamer Nearly Halves Workforce in Savings Push After Late-Stage Hypertension Fail
Gossamer Bio announced a near‑half workforce reduction after its Phase 3 seralutinib trial failed to meet statistical significance in pulmonary arterial hypertension. The company will lay off 77 employees, about 48 % of staff, aiming to preserve cash while its future path remains uncertain. The failed PROSERA study showed only a 28.2‑meter improvement versus 13.5 meters for placebo, sending the stock down 80 % to $0.42. As of year‑end 2025, Gossamer held $136.9 million in cash, highlighting a tight financial runway.
TerraPower Commits $450M to Build Radioisotope Production Plant
TerraPower Isotopes is committing $450 million to build a cGMP‑compliant actinium‑225 manufacturing plant in Philadelphia, a move that will expand production capacity roughly twenty‑fold. The 250,000‑square‑foot facility, slated to begin output in 2029, will create 225 full‑time jobs and benefits from...
Protagonist’s First Approval Spells Trouble for Pharma’s Immunology Heavyweights
Protagonist Therapeutics received FDA approval for icotrokinra, marketed as Icotyde, becoming the first oral IL‑23 receptor blocker for plaque psoriasis. The clearance arrived ahead of schedule, unlocking a $50 million milestone from Johnson & Johnson and setting up royalty terms of 6‑10% on...
Kennedy’s Vaccine Agenda Stalled, Structure’s ‘Competitive’ Obesity Pill, Novo’s Warning Letter
Structure Therapeutics reported a 16.3% weight loss after 44 weeks in a Phase 2 trial of its oral GLP‑1 pill, positioning it as a competitive alternative to Eli Lilly’s and Novo Nordisk’s candidates. Rhythm Pharmaceuticals disclosed that its obesity drug Imcivree failed in...
Lilly-Backed China Startup Debuts With $68.7M Seed to Advance Next-Gen T Cell Engagers
Excalipoint Therapeutics, a Shanghai‑based biotech, closed a $68.7 million seed round, including a $41 million founding raise and a $27.7 million extension led by MPCi, Centurium Capital, Lilly Asia Ventures, and Eisai Innovation. The capital will fund six tri‑specific T‑cell engager candidates, notably...
Webinar: Operationalizing AI in Drug Development: Inside DIA’s Global AI Consortium
The Drug Information Association (DIA) has launched a public‑private AI Consortium that unites regulators, biopharma, academia, and technology firms to shape AI governance in drug development. The group is developing a seven‑step classification framework that aligns AI use‑cases with risk‑proportionate...
Third FDA Rejection for Aldeyra’s Dry Eye Disease Drug Sends Shares Plummeting
The FDA issued a third complete response letter rejecting Aldeyra Therapeutics' lead dry‑eye candidate, reproxalap, citing a lack of substantial evidence and inconsistent efficacy data. The agency noted the drug failed to demonstrate clear benefit in well‑controlled studies, though no...
Denali’s Hunter Syndrome Candidate in the Spotlight After REGENXBIO Rejection
REGENXBIO's gene therapy RGX‑121 for Hunter syndrome received an FDA Complete Response Letter, with the agency flagging patient‑eligibility definitions, natural‑history control comparability, and the surrogate endpoint as problematic. The rejection redirects focus to Denali Therapeutics, whose enzyme‑replacement candidate tividenofusp alfa...
Biotechs Report Regulatory Headaches, High-Stakes Catalysts During Q4 Earnings
Q4 earnings highlighted a mixed biotech landscape, with regulatory turbulence easing for some firms while high‑stakes catalysts loom. Capricor Therapeutics is preparing to resubmit its Duchenne cardiomyopathy cell therapy after the departure of FDA CBER director Vinay Prasad, and reported...
Rare Disease Sales To Soar to $400B+ By 2032 as Small Molecules Resurgent: Evaluate
Orphan‑drug sales are projected to exceed $400 billion by 2032, more than double the 2025 level. Small‑molecule therapies dominate the pipeline, accounting for 45% of the 20 most valuable orphan candidates, signaling a resurgence after years of biologic focus. The Inflation...
Novo Holdings’ Assets Fell 34% in 2025 As Its Namesake Drugmaker Struggled
Novo Holdings' assets under management plunged 34% in 2025, falling to €93 billion as its controlling stake in Novo Nordisk lost market value. Despite the valuation hit, the holdings generated €2.8 billion in income and delivered 8‑9% five‑ and ten‑year returns, outpacing...
BridgeBio Builds Case for Early 2027 Launch of Dystrophy Drug
BridgeBio Pharma reported that its small‑molecule candidate BBP‑418 produced a 1.8‑fold increase in the α‑dystroglycan biomarker in a Phase 3 FORTIFY trial for limb‑girdle muscular dystrophy type 2I/R9, with effects sustained through 12 months. The trial enrolled 81 patients and also showed...
From Two Trials to One, Sponsors Face a Higher Standard
The FDA announced that a single pivotal trial can now satisfy efficacy requirements for new drug applications, replacing the previous two‑study mandate. This change raises expectations for data depth, quality, and risk‑based management throughout the trial lifecycle. Guests Oxana Iliach...
The Next Cambridge? LA Sets Its Sights Higher
Los Angeles County now hosts more than 3,000 life‑science companies, outpacing peer regions, and generated roughly $58 billion in annual economic output. Venture‑capital inflows surged to $4.4 billion in 2024, a 119 % jump, underscoring growing investor confidence. The ecosystem benefits from a...
Hiring Outlook: February Brings First YOY Job Increase Since 2022
Biopharma job postings on BioSpace rose 5% year‑over‑year in February, the first increase since September 2022, and jumped 21% month‑over‑month. The broader U.S. labor market shed 92,000 jobs, driven by healthcare strikes and winter storms, but biopharma layoffs eased, with...
6 Companies Hiring in Cambridge
Cambridge, Massachusetts remains a premier life‑sciences hub, anchored by giants like Intellia Therapeutics and Moderna and supported by the MassBio trade group. While BioSpace job postings fell 4% year‑over‑year in February, they surged 17% month‑over‑month, reflecting renewed hiring momentum. Six...
Capricor Shares Rise as FDA Sets August Decision Date for Rejected Duchenne Therapy
Capricor Therapeutics announced that the FDA has scheduled an August 22 decision on its investigational Duchenne muscular dystrophy cell therapy, deramiocel, after lifting a prior complete response letter. The biotech resubmitted an enhanced package that includes robust Phase III HOPE‑3 data showing...
Pharma Is Ravenous for M&A Action but Late-Stage Supply Dwindles
Big pharma is intensifying its M&A hunt as the pool of late‑stage biopharma assets shrinks, turning deal processes into competitive auctions. Sellers are leveraging multiple inquiries to secure higher upfront cash and better terms, while unsolicited public bids—exemplified by the...
FDA Sets Scope for Attempt To Reduce Manufacturing-Related Approval Rejections
The FDA has defined the scope of pre‑submission facility meetings to curb manufacturing‑related complete response letters that delay drug approvals. It agreed to cover prior production‑site inspections, novel process elements and supply‑chain node strategies, while rejecting topics such as alternative...
AI-Focused Fund Breakout Raises $114M To Support Early-Stage Biotechs
Breakout Ventures announced the close of its third fund, raising $114 million to back AI‑driven biotech startups. The capital will be deployed to early‑stage companies, including a University of Chicago spin‑out focused on computational small‑molecule design and a stealth venture tackling...
Rising Amid Flurry of CAR T Deals, Stylus Proves Cell Therapy Is Not Dead
Stylus Medicine entered the cell‑therapy arena in May 2025 with an in‑vivo CAR‑T platform that delivers a lipid nanoparticle‑encapsulated recombinase to engineer T cells inside patients. The move comes after major pharma acquisitions—BMS buying Orbital Therapeutics for $1.5 billion and Gilead...
Dyne Plans Post-Prasad FDA Run as Duchenne Exon Skipper Sustains Benefit in Long Term Data
Dyne Therapeutics reported that its exon‑skipping candidate z‑rostudirsen sustained respiratory and cardiac benefits through 24 months in the Phase 1/2 DELIVER study for Duchenne muscular dystrophy. The therapy maintained forced vital capacity, circumferential strain and left‑ventricular ejection fraction improvements compared with...
Single Pivotal Trials Demand Stronger Data and Risk Strategies
Following the FDA’s recent shift to require only one pivotal trial for new drug applications, sponsors now face heightened pressure to generate robust efficacy and safety data. Regulators expect a single, bullet‑proof study rather than two less conclusive trials, mirroring...
Nonprofit Aims To Modernize Manufacturing by Clearing Capital Hurdle
A nonprofit, the API Innovation Center (APIIC), is tackling the capital barrier that prevents U.S. drug makers from adopting continuous manufacturing. By pooling state, federal, philanthropic and private funds, APIIC installs equipment at manufacturers at no upfront cost, de‑risking the...
AbbVie’s Amylin Candidate ‘Competitive’ in Early-Stage Trial
AbbVie announced top‑line Phase 1 multiple ascending‑dose data for its amylin analog ABBV‑295, showing 7.75‑9.79% weight loss after 12 weeks of treatment. The long‑acting compound was administered every other week then monthly, with a favorable tolerability profile and no serious adverse...
Regeneron’s Weight Loss Partner Hansoh Delivers Much-Needed Phase 3 Win in China
Regeneron’s Chinese partner Hansoh announced that its dual GLP‑1/GIPR agonist olatorepatide achieved a 19% mean weight loss in a Phase 3 trial of 604 obese or overweight adults, meeting both co‑primary endpoints. The study reported lower gastrointestinal adverse events compared with...
Xenon To Seek Approval of First-in-Class Epilepsy Drug After Exceeding Phase 3 Expectations
Xenon Pharmaceuticals reported that its Phase 3 X‑TOLE2 trial of azetukalner, a novel Kv7 potassium channel opener, achieved a 53.2% reduction in focal onset seizures at the 25 mg dose, far exceeding expectations and representing the highest placebo‑adjusted efficacy recorded in a...
Two AstraZeneca Drugs To Be Scrutinized in First FDA Cancer Advisory Panel in 9 Months
The FDA’s Oncologic Drugs Advisory Committee will convene on April 30 to evaluate AstraZeneca’s oral SERD camizestrant for first‑line HR‑positive, HER2‑negative breast cancer and its AKT inhibitor Truqap for metastatic hormone‑sensitive prostate cancer. Camizestrant’s Phase 3 SERENA‑6 trial reported a 56% reduction...
Incyte’s Lung Cancer Expansion Bid Thwarted by Issues at Novo’s Catalent-Acquired Site
The FDA rejected Incyte’s supplemental application to add non‑small cell lung cancer to Zynyz’s label, citing compliance failures at Novo Nordisk’s Catalent‑owned Indiana manufacturing plant. The agency’s complete response letter pinpointed inspection findings at the site as the sole approvability...
J&J Wins Third National Priority Approval for Multiple Myeloma Combo
Johnson & Johnson’s Tecvayli and Darzalex combination received FDA approval for second‑line multiple myeloma treatment, marking the third drug cleared under the Commissioner’s National Priority Voucher (CNPV) program. The decision was rendered in just 55 days after J&J’s filing, thanks...
Psychedelics Are Placeholders for More Traditional Depression Therapies: Analysts
Psychedelic antidepressants are poised for FDA review this year, driven by strong investor and patient interest. William Blair analysts note that while Johnson & Johnson’s Spravato generated $1.7 billion in 2025 sales, psychedelics are unlikely to capture the entire treatment‑resistant depression market. Companies...
Alnylam Unites With Tenaya in Potential $1B+ Pact To Find New Genetic Heart Disease Targets
Alnylam Pharmaceuticals has signed a deal with Tenaya Therapeutics, providing $10 million upfront and the potential for up to $1.13 billion in milestones to discover up to 15 new genetic targets for heart disease. Tenaya will apply its modality‑agnostic platform to validate...
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,...
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...