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 leverage demographic and geographic insights to locate trial sites where target patients live, making studies more inclusive. MedCity News and PurpleLab’s new eBook details how this data‑driven approach can broaden trial participation and improve drug uptake across diverse populations.
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...
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...
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...
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...
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...
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...
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...
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...
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...
'My Son Can Now Enjoy Life': Children with Severe Form of Epilepsy Helped by New Drug
A new gene‑targeted drug, zorevunersen, is showing dramatic seizure reductions in children with Dravet syndrome, a rare but severe epilepsy. Early trial data published in the New England Journal of Medicine report up to 90% fewer seizures for participants as...
Scientists Laud Potentially Life-Changing Drug for Children with Resistant Form of Epilepsy
Preliminary trials of Zorevunersen, an experimental therapy for Dravet syndrome, showed it is safe and well tolerated in 81 children. A single 70 mg dose reduced seizures by about 50%, and three doses cut seizures roughly 80% compared with baseline. The...
Endothelin Receptor Antagonist REMS Information
The FDA has eliminated risk evaluation and mitigation strategy (REMS) requirements for embryofetal toxicity (EFT) across all endothelin receptor antagonist (ERA) medicines, concluding that labeling alone sufficiently communicates the risk. The change, effective April 2025, applies to ambrisentan, macitentan‑containing products, and...
Drug Trials Snapshots: MYQORZO
MYQORZO (aficamten), a cardiac myosin inhibitor, received FDA approval on Dec 19 2025 for adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Approval is based on the SEQUOIA‑HCM phase III trial, a randomized, double‑blind, placebo‑controlled study of 282 patients across 14 countries. At 24 weeks,...
Orai1 Switches Ca2+ Signals, Balances Erythropoiesis
Researchers have identified the Orai1 calcium channel as a pivotal regulator of intracellular Ca2+ signals that govern erythropoiesis. Genetic ablation of Orai1 in mouse models leads to impaired red‑cell maturation and anemia, while pharmacologic activation rescues normal blood counts. The...
October 5, 2023: Meeting of the Oncologic Drugs Advisory Committee Meeting Announcement - 10/05/2023
The FDA’s Oncologic Drugs Advisory Committee will meet virtually on October 5, 2023 to evaluate Amgen’s supplemental NDA for LUMAKRAS (sotorasib) in KRAS G12C‑mutated non‑small cell lung cancer. The committee will review data from the confirmatory CodeBreaK 200 trial, which could convert the existing...
Upcoming EL-PFDD Meetings
The FDA has posted a public list of disease areas where external organizations have submitted letters of intent for future Externally‑Led Patient‑Focused Drug Development (EL‑PFDD) meetings. Ten topics ranging from infertility to rare neurological disorders are scheduled between March and...
Leading HIV Researchers Reflect on Breakthroughs, Challenges at CROI 2026
The 2026 Conference on Retroviruses and Opportunistic Infections (CROI) highlighted major HIV research breakthroughs while underscoring persistent funding shortfalls. Experts praised advances in antiretroviral therapy, emerging adjunct treatments, and global advocacy, yet warned that reduced U.S. support could limit access...
Nanoparticle Vaccine Approach Takes on a New Target: Hepatitis C Virus
Scientists at Scripps Research have engineered a native‑like, stabilized version of the hepatitis C virus E1E2 glycoprotein complex and displayed it on self‑assembling protein nanoparticles (SApNPs). The nanoparticle vaccine candidate elicited strong, virus‑specific antibody responses in animal models. This breakthrough overcomes...
Five Projects Share £100m UK-US Cancer Grand Challenges Fund
Five interdisciplinary teams will receive up to £20 million each from the UK‑US Cancer Grand Challenges, adding £100 million to the programme and raising total investment to £465 million since 2016. The five projects, spanning 34 institutions in nine countries, target unconventional angles...
STAT+: Pharmalittle: We’re Reading About Moderna’s $2.25 Billion Settlement, FDA Warning Letters, and More
Moderna agreed to settle Roivant's patent claims for up to $2.25 billion, paying $950 million upfront and a contingent $1.3 billion if its liability‑shifting strategy fails. The deal averts a Delaware jury trial that many analysts feared would be costly and damaging. Simultaneously,...
National Drug Code Format
The FDA issued a final rule establishing a uniform 12‑digit National Drug Code (NDC) format effective March 7, 2033, replacing the current 10‑digit variants. A seven‑year window (2026‑2033) gives manufacturers, distributors, pharmacies and payors time to upgrade systems and labeling, followed by...
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...
Theolytics Awarded €8 Million Horizon Europe Grant to Fund Ovarian Cancer Study
Theolytics, an Oxford‑based biotech, secured an €8 million non‑dilutive Horizon Europe grant to fund its phase 2 OCTOPOD‑IV expansion trial of THEO‑260, a novel oncolytic therapy for advanced ovarian cancer. The grant, awarded after a rigorous review, will finance the phase 2a portion...
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...
Aspire Biopharma and Microsize Collaborate for Alprazolam Powder Formulation
Aspire Biopharma has teamed with particle‑engineering specialist Microsize to develop a sublingual powder formulation of alprazolam, aiming for faster anxiety relief than traditional tablets. The partnership leverages Microsize’s micronisation expertise and Pace’s excipient compatibility and stability services to accelerate development....
Extracellular Vesicles: The Next Frontier in Regenerative Medicine and Drug Delivery?
Extracellular vesicles (EVs) are emerging as a versatile platform for regenerative medicine and targeted drug delivery. Researchers highlight their ability to transfer proteins, RNA, and lipids between cells, mimicking natural signaling pathways. Recent advances in isolation, engineering, and loading techniques...
UK Puts £50m Behind Expanded Clinical Trials Drive
The UK government announced nearly £48 million in equipment funding to accelerate commercial clinical trials across the NHS. The money will equip 51 NHS trusts and 79 primary‑care organisations with diagnostic tools, scanners and mobile research vans, with 60 % directed to...
Navigating Uncertainty and Building Resilient Supply Chains: The Case for US-Based CDMO Partnerships
Pharmaceutical firms are accelerating onshoring efforts, with more than $270 billion pledged for U.S. manufacturing since early 2025 and 80% of global producers weighing domestic or near‑shore options. Legislative moves such as the BIOSECURE Act and incentives like BARDA Project NextGen are...
Hua Medicine Announces the Approval of Dorzagliatin for Marketing in Hong Kong SAR, China
Hua Medicine announced that its glucokinase activator dorzagliatin, marketed as MYHOMSIS®, received marketing approval from Hong Kong’s Department of Health under the “1+” regulatory pathway. The drug, the world’s first‑in‑class GKA, targets the root cause of glucose dysregulation in Type 2 diabetes...
GLP-1 Drugs Linked to Lower Addiction Rates in Large Study of Veterans
A large retrospective study of over 600,000 U.S. veterans found that patients prescribed GLP-1 receptor agonists for diabetes were 14% less likely to develop new substance-use disorders compared with those on SGLT-2 inhibitors. The analysis also showed 30% fewer drug-related...
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...
Whole-Genome Sequencing Meets Real-World Outcomes: What 1,364 Breast Cancer Genomes Reveal About Treatment Response
A new Nature study sequenced the whole genomes of 1,364 breast cancers and linked the data to transcriptomics and real‑world treatment outcomes. The analysis showed that genome‑wide signatures such as homologous recombination deficiency, intratumoral heterogeneity, and copy‑number instability correlate with...
Verastem Inc (VSTM) Q4 2025 Earnings Call Transcript
Verastem reported $1.7 million in COPIKTRA net revenue for Q1 2019, a 38% increase over the prior quarter, and achieved reimbursement coverage with more than 92% of targeted health plans. Clinical updates highlighted the Phase 3 DUO trial, which showed...