Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech rounds
Spanish biotech firm Ona announced an $86.6 million Series B financing, ranking among the largest venture rounds in Spain’s life‑science sector this year. The round was led by Seventure Partners with participation from existing backers and new strategic investors. The funding will be used to scale Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical programs.
Strategic Intelligence Report on Bladder Cancer
At the recent ASCO‑GU meeting, industry leaders highlighted that the greatest threat to emerging bladder‑cancer programs is strategic, not clinical. Phase‑2 candidates are poised to enter Phase‑3 trials, but shifting control arms, evolving endpoints, and changing patient demographics risk rendering these studies irrelevant. The report warns that without agile trial design, novel therapies may lose competitive footing before market entry. Stakeholders must anticipate regulatory and market dynamics to preserve development momentum.
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 Veteran Decries Huntington Therapy Refusal as “Evil”
Amazing quote in this NYT story on the FDA from @By_CJewett. Janet Woodcock, a top drug regulator for decades, said reversals for a promising therapy send shock waves through the industry. “The Huntington’s refusal I thought was truly evil,” she told NYT....
Cryo-EM Drug Discovery Center in Bay Area Opened by Thermo Fisher Scientific
Thermo Fisher Scientific has opened a Cryo‑Electron Microscopy Drug Discovery Center in South San Francisco, offering pharmaceutical and biotech firms direct, hands‑on access to cutting‑edge cryo‑EM instrumentation. The facility is designed to accelerate structural insight generation, enabling faster, more cost‑effective...
At CNBC Cures, Becky Quick Leads Clarion Call for Rare Disease Research
The CNBC Cures Summit opened with Becky Quick urging families and innovators to accelerate rare‑disease research. Speakers highlighted a widening gap between rapid scientific breakthroughs—gene therapies, AI‑driven diagnostics, and modular “nodal biology”—and an aging regulatory framework. Leaders from Biogen, the...
Call for a Balanced, Transparent FDA Approach
My friend @adamfeuerstein delivers some great reporting and smart commentary on the FDA's direction that $QURE conduct a new randomized trial for its Huntington's drug. A senior FDA official and the company disagree on the basic question of whether the...
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...
Using Real World Data From the Patient Experience to Improve Drug Development
Randomized trials for non‑small cell lung cancer often miss real‑world nuances that affect patients' quality of life. Real‑world data (RWD) and social determinants of health (SDOH) expose hidden barriers such as transportation gaps and limited molecular testing access. Pharma can...
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...
Can GLP-1 Drugs Help Tackle Addiction?
A new BMJ meta‑analysis of over 600,000 U.S. veterans with type‑2 diabetes found that patients prescribed GLP‑1 agonists such as semaglutide or tirzepatide were 14% less likely to develop substance‑use disorders (SUD) than those on SGLT2 inhibitors. Over a three‑year...
Pirtobrutinib
Late 2025 saw the FDA grant traditional approval to pirtobrutinib, an oral, reversible BTK inhibitor targeting multiple B‑cell malignancies. The drug demonstrated robust efficacy in BTK‑resistant chronic lymphocytic leukemia and small lymphocytic lymphoma, backed by positive Phase 3 data and early...
FDA's Extremist Shifts Harm Rare Disease Stakeholders
In this week's Biotech Scorecard newsletter: The extremism of the FDA’s Peter Marks and Vinay Prasad has come with costs Two regulators, two extreme regulatory philosophies, one replacing the other. The rare disease community is suffering whiplash. Drugmakers ( $QURE...
Bladder Cancer Landscape Shifts, Opening New Strategic Opportunities
When cancer niches are rapidly changing they create both a vacuum of indecision and fresh opportunities for smart players. Here we explore bladder cancer and look at where the field is headed... https://t.co/cf93Qb8dhe https://t.co/fx5V1MVwYN
Airiver Medical Receives FDA Breakthrough Device Designation for Airiver DCB and Treats First Patient with Central Airway Stenosis
Airiver Medical announced that the U.S. Food and Drug Administration granted Breakthrough Device Designation to its pulmonary drug‑coated balloon (DCB) for treating central airway stenosis. The company also enrolled and treated the first patient in a pivotal trial that will...
Eli Lilly Courts Employers to Expand Obesity Drug Access
Lilly targets employers in new bid to broaden access to obesity drugs https://t.co/Y6fmcmTcf1 @ByJonGardner $LLY $NVO #obesity
China's Rise Threatens US Gene Therapy Market
China competition is coming for the US's multimillion-dollar gene therapies -- a must-read from @Jared_Whitlock about what's happening there and how it will affect access around the world: https://t.co/F7KaMv3Kkp
Cancer-Eating Bacteria Engineered to Consume Tumors From the Inside Out
University of Waterloo researchers have engineered the anaerobic bacterium Clostridium sporogenes to consume solid tumors from the inside out. The spores infiltrate the oxygen‑free tumor core, proliferate and break down cancerous tissue. By inserting an oxygen‑resistant gene and a quorum‑sensing...
Yamanaka Stem Cells Earn Conditional Approval for Heart and Parkinson's
20 years of Yamanaka stem cell factor research culminates in 2 conditional approvals for heart disease and Parkinson's disease in Japan https://t.co/8oxKxXYafu by Shinya Yamanaka @CellStemCell https://t.co/U878IxSfSi @ScienceMagazine https://t.co/HCCPaTEP7e
HMPL‑A580 Confirmed as Anti‑EGFR ADC, Impacts ALXO
I speculated last month that HMPL-A580 was an anti-EGFR ADC https://t.co/XFDtZfWWkH and $HCM just confirmed that this is the case. Relevant for $ALXO
Government Throws Weight Behind Space-Manufactured Drugs
The UK government announced a new package of measures to accelerate space‑based pharmaceutical manufacturing, offering regulatory clarity and a sandbox for companies developing drugs in microgravity. The initiative, led by the Department for Science, Innovation and Technology, brings together the...
Theravance Biopharma Faces Second Layoff Wave After Phase III Flop
5March: Back in 2021, I wrote about a significant round of layoffs at Theravance Biopharma ( $TBPH). The company is cutting jobs again, and has begun a strategic review, after a Phase III failure. Details in my latest for @GENbio:...
Red Blood Cell–PD1 Conjugates Show Promise in Resistant Tumors
Erythrocyte–anti-PD1 conjugates in persons with advanced solid tumors resistant to anti-PD1/PDL1: preclinical characterization and results of a phase 1 trial https://t.co/1GQMlUHn4Y https://t.co/0it5nTvUm0
Targeted Therapies to Join Chemo as Oncology Treatment Backbone
Targeted therapies are reshaping oncology, yet chemotherapy remains essential. Immune checkpoint inhibitors like Keytruda generate $31.7 bn sales in 2025 and cover more than 40 indications. The FDA granted 63 antibody‑drug conjugate review designations in 2024, nearly double the previous peak....
Hansa Closes on FDA Verdict for Transplant Drug Imlifidase
The FDA has begun reviewing Hansa Biopharma’s imlifidase, a drug that desensitises highly sensitised kidney‑transplant patients, with a decision expected by December 19, 2024. If approved, it would be the first U.S. therapy to improve transplant odds for the 10‑15 % of...
Xtalks Featured Member: Giorgia Palano, Life Science Consultant, Knightec Group
Xtalks has spotlighted Giorgia Palano, PhD, a Life Science Consultant at Knightec Group, highlighting her expertise in validation strategies and regulatory compliance. Palano works with cross‑functional teams to ensure quality documentation and continuous improvement across complex life‑science operations. She emphasizes...
STAT+: Eli Lilly Launches Program for Employers to Subsidize Cost of Obesity Drug Outside Insurance
Eli Lilly introduced a new employer‑focused program allowing companies to subsidize the cash price of its obesity medication Zepbound. The drug can be purchased directly from LillyDirect for $449 per month, and employers can contribute a fixed amount, such as $50...
The Ongoing Impact of MFN Pricing
The pharmaceutical industry is grappling with evolving Most‑Favored‑Nation (MFN) pricing rules as the TrumpRx initiative clarifies administration expectations. Recent Supreme Court rulings on IEEPA tariffs, distinct from Section 232 tariffs used in MFN negotiations, add uncertainty to pricing strategies. Manufacturers must...
Akeso Presents P-II (COMPASSION-03) Trial Data on Cadonilimab for R/M Cervical Cancer at ESGO 2026
Akeso presented Phase‑II COMPASSION‑03 data for cadonilimab in patients with recurrent or metastatic cervical cancer who progressed after platinum chemotherapy. The trial reported a median overall survival of 17.5 months across the cohort, with 24‑month OS rates of 40.9% irrespective...
Next-Gen Malaria Vaccine Overcomes Major Hurdle
Researchers at WEHI and the Burnet Institute have mapped the human immune response to Plasmodium vivax, revealing how antibodies neutralize the parasite. The study, published in Immunity, shows that protective immunity depends on antibody function and simultaneous targeting of multiple...
GAIA and Daiichi Sankyo Europe Enter Exclusive Partnership to Launch Next-Generation Digital Therapeutic for Cardiovascular Care in Europe.
GAIA and Daiichi Sankyo Europe announced an exclusive partnership to commercialize lipodia, a next‑generation digital therapeutic for hypercholesterolemia, pending regulatory approval. The deal grants Daiichi exclusive rights in Germany with expansion mechanisms for other European markets, leveraging GAIA’s validated behavioral...
HUTCHMED Begins Phase I/IIa Trial of HMPL-A580 for Solid Tumours
HUTCHMED has launched a first‑in‑human Phase I/IIa trial of HMPL‑A580, its second antibody‑targeted therapy conjugate, in the United States and China. The multi‑centre, open‑label study will evaluate safety, tolerability, immunogenicity, pharmacokinetics and early efficacy across dose‑escalation and expansion cohorts. HMPL‑A580...
Affinia Receives FDA Fast Track Designation for AFTX-201
Affinia Therapeutics announced that the FDA has granted fast‑track designation to its gene‑therapy candidate AFTX‑201 for BAG3‑associated dilated cardiomyopathy. The one‑time intravenous treatment delivers a full‑length BAG3 transgene via an engineered capsid that requires doses five to ten times lower...
Guiding Nano Assembly for Drug Delivery with Machine Learning
Researchers repurposed the FAP inhibitor SP‑13786 as a co‑assembly excipient to create SP co‑assembled nanoparticles (SCAN) that encapsulate hydrophobic drugs. Using molecular dynamics and a random‑forest machine‑learning model, they identified 228 physicochemical descriptors that predict successful nano‑co‑assembly, highlighting aromaticity and...
Opinion: AI Could Revolutionize Antibiotics — but the Market Is Standing in the Way
Antibiotic resistance now kills roughly 4.9 million people each year, and the last truly novel class of antibiotics was introduced over three decades ago. Artificial intelligence promises to accelerate the discovery of new antimicrobial agents by scanning vast chemical spaces and...
STAT+: Gilead and South Africa Are Negotiating a License for Local Production of New HIV Drug
Gilead Sciences is in talks with the South African government to issue a voluntary licence for the local manufacture of lenacapavir, a novel HIV‑prevention drug. The agreement would enable South African firms, identified with help from Unitaid and the US...
Moderna Settles LNP Patent Dispute with Genevant Sciences and Arbutus Biopharma for ~$2.25B
Moderna agreed to a roughly $2.25 billion settlement with Genevant Sciences and Arbutus Biopharma, ending disputes over lipid nanoparticle (LNP) technology used in its COVID‑19 vaccine. The deal requires $950 million payable in July 2026 and an additional $1.3 billion contingent on an appellate...
Atlas Insights: Single-Cell Data Point to Druggable Nodes in Down Syndrome
Researchers have generated five single‑cell atlases of human brains affected by Down syndrome, providing unprecedented cell‑type and temporal resolution. The atlases pinpoint molecular signatures that correspond to distinct neuronal and glial populations across developmental stages. By integrating these data, scientists...
New HIV Cure Approach Forces Hidden Virus Into Tripping Immune Sensor
Researchers have unveiled a novel HIV‑cure strategy that forces dormant virus particles to reveal themselves to the body’s innate immune system. The method employs a STING‑pathway agonist to coax latent proviruses into producing viral RNA, which then triggers a potent...
New Clinical Investigation Exclusivity (3-Year Exclusivity) for Drug Products: Questions and Answers
The FDA released a draft Level 1 guidance titled “New Clinical Investigation Exclusivity (3‑Year Exclusivity) for Drug Products: Questions and Answers.” The document clarifies statutory and regulatory criteria for obtaining three‑year market exclusivity on new drug applications or supplements. It outlines...
Menopause Hormone Therapy: The Myths, the Medicine, and the “Why Didn’t Anyone Tell Me?” Moment
The article debunks long‑standing myths about menopausal hormone therapy (MHT) by highlighting how the 2002 Women’s Health Initiative (WHI) trial was misapplied to all women. It explains that the WHI tested an older cohort using a specific estrogen‑progestin combo, leading...
Kura Oncology Inc (KURA) Q4 2025 Earnings Call Transcript
Syndax Pharmaceuticals reported $172 million total revenue for 2025, driven by $125 million from its menin inhibitor RevuForge and $152 million from chronic GVHD therapy Nictimvo. RevuForge saw 38% quarter‑over‑quarter revenue growth and a 35% rise in prescriptions, bolstered by an expanded NPM1...
TriSalus Life Sciences Inc (TLSI) Q4 2025 Earnings Call Transcript
TriSalus Life Sciences reported Q3 2025 revenue of $11.6 million, a 57% year‑over‑year increase, while adjusted EBITDA loss narrowed to $5.4 million despite one‑time study charges. Gross margin slipped to 84% as new product launches strained manufacturing efficiency, but cash burn improved,...
Atea Pharmaceuticals Inc (AVIR) Q4 2025 Earnings Call Transcript
Atea Pharmaceuticals reported a $329.3 million cash position that funds its Phase III hepatitis C program through 2027 and supports a new hepatitis E pipeline. The CBEYOND and C FORWARD trials are on track, with enrollment completing next month and mid‑2026 respectively, and top‑line data...
Contineum Therapeutics Inc (CTNM) Q4 2025 Earnings Call Transcript
Compugen Ltd. reported a dramatic financial turnaround in its Q4 2025 earnings, posting a $56.8 million quarterly profit and $35.3 million annual profit after a year of losses. A $65 million upfront royalty monetization from AstraZeneca boosted cash to $145.6 million, extending the cash runway...
Regenxbio Inc (RGNX) Q4 2025 Earnings Call Transcript
Regenxbio reported that enrollment for its Duchenne gene therapy RGX-202 was completed ahead of guidance, with top‑line data slated for early Q2 2026 and a BLA submission planned for mid‑2026. The company confirmed manufacturing capacity of 2,500 annual doses and highlighted...
GLP‑1 Drugs Cut Substance‑abuse Risk in Veterans
Best evidence yet that GLP-1 drugs reduce the risk of substance abuse, from >600,00 US Veterans across alcohol, nicotine, opioid, cocaine, cannabis @bmj_latest @zalaly @Miao_Cai_SLU https://t.co/rcqUt0kY77
GDUFA Type II API DMF Payment Receipts Report
The FDA’s GDUFA Type II API DMF payment report shows 385 payments in FY 2023, a dip to 264 in FY 2024, a rebound to 374 in FY 2025, and 90 early‑year payments in FY 2026. Monthly counts reveal a pronounced September 2023 spike of 103...
Fiscal Year 2026 Generic Drug Science and Research Initiatives Public Workshop - 06/08/2026
The FDA is holding its Fiscal Year 2026 Generic Drug Science and Research Initiatives public workshop on June 8‑9, offering both in‑person and virtual attendance. The two‑day event will review current research gaps and solicit stakeholder input to shape the FY 2027...