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.
'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 young as two, delivered via intrathecal infusion. Families like Freddie Truelove’s describe a transformation from hundreds of daily seizures to a few weekly events, enabling normal activities. Phase III studies are now planned to validate these findings for broader use.
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...
Cell‑free DNA Test Predicts Liver Disease and Mortality
We've used a blood test—cell free DNA—for detecting cancer or prenatal fetal abnormalities. It turns out it can be used to detect liver diseases and all-cause mortality from other conditions @ScienceTM https://t.co/IJxPMq7hGU
A Fair Amount of ARPA-H Funding Is Being Used for Clinical Trials Relevant to Aging
ARPA‑H is committing up to $144 million to healthspan‑focused human trials through its PROSPR program, funding seven teams to develop early biomarkers and surrogate endpoints for aging interventions. The agency has awarded Cambrian Bio $30.8 million for an oral rapamycin analog targeting...
FDA COO Butler Retires After One Year; Deputy Assumes Role
after just one year in the position, FDA COO Butler will retire, and his deputy will take over in April - https://t.co/9qmmIiuWX2
Oncolytic Virotherapies in Ovarian Cancer and NSCLC with Genelux CEO Thomas Zindrick — Episode 245
Genelux Corporation’s President and CEO Thomas Zindrick discussed the company’s oncolytic virotherapy programs targeting ovarian cancer and non‑small cell lung cancer on the Xtalks Life Science Podcast. The interview highlighted the therapeutic potential of engineered viruses to stimulate anti‑tumor immunity...
All Family Weekly Health Briefing
The FDA is reportedly reconsidering its stance on peptide compounds, a shift potentially driven by officials linked to Robert F. Kennedy Jr., which could broaden research and bio‑hacking applications. Meanwhile, ivermectin has re‑entered public discourse after high‑profile media mentions, prompting...
Blackstone Invests $400M in Teva, Sanofi Gut Drug
Blackstone puts $400M into Teva, Sanofi gut disease drug https://t.co/CkxPaI6CPr @ByJonGardner $TEVA $SNY $MRK $RHHBY
CTMX Soars 660% Ahead of Masked ADC Catalyst
$CTMX, +660% in 12 months, approaches its masked ADC catalyst. Via @ApexOnco -> https://t.co/oQI1iQiV4N ( $JANX -58% in the same timeframe)
Antengene and UCB Enter $1.18 Billion Global License Agreement for Bispecific T-Cell Engager
Antengene and UCB have signed a global license agreement to develop ATG‑201, a CD19/CD3 bispecific T‑cell engager targeting B‑cell‑driven autoimmune diseases. The deal delivers an $80 million upfront payment and unlocks more than $1.1 billion in development and commercial milestones, plus tiered...
Autonomous Labs vs Traditional Automation: Key Differences Explained
I get asked a lot how Autonomous Labs are different than traditional lab automation, here’s how 👇 Happy to take Qs in comments too https://t.co/AdGMiQWHHT
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...
Teva and Blackstone Life Sciences Enter $400 Million Strategic Growth Capital Agreement
Teva Pharmaceuticals has secured a $400 million strategic growth capital agreement with Blackstone Life Sciences to fund the Phase III development of duvakitug, a TL1A‑targeting monoclonal antibody for ulcerative colitis and Crohn’s disease. The four‑year deal grants Blackstone milestone payments and low‑single‑digit...
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...
Ethics Corner: “As a Statistical Consultant, if You’re a Co-Author on a Substantive Paper, Is It Your Duty to...
Statistical consultants who become co‑authors must balance honesty with scope. They should fully disclose the analyses they performed and any limitations, but are not obligated to fix every statistical flaw beyond their contract. If a manuscript contains questionable methods, the...
Future Cancer Blood Tests May Accurately Screen High‑Risk Groups
Cancer blood tests for screening? Not the way they've been studied to date (age 50+) A new feature @Nature “I’m confident we’re going to see more accurate tests going forward. In high-risk groups, such as those with a genetic predisposition,...
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...
2025 Non-US Novel Large Molecule Drug Approvals
In 2025, Europe’s EMA, China’s NMPA, and Japan’s PMDA each granted first‑time approvals for novel large‑molecule therapeutics. Oncology accounted for the largest share of these approvals, while endocrinology—driven largely by GLP‑1‑based agents—was the second biggest category. The approvals span a...
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....
FDA's History with Complex Plants
The FDA issued its first botanical drug guidance in 2004 and updated it in 2016, yet only four complex plant‑derived drugs have been approved to date. Ajna Biosciences, led by Joel Stanley, has secured clearance to begin a Phase 2 trial...
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...
Why Asia Is the Emerging Epicenter for Global Biopharmaceutical Progress
In this episode, Daniel Levine talks with Feng Nying Zhang, a partner at McKinsey Shanghai, about Asia’s rapid rise as a global hub for biopharmaceutical innovation, highlighted by a McKinsey report showing the region’s share of innovative drug pipelines jumping...
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...
Enhancing Cold Chain Visibility
SpotSee introduced WarmMark QR, a battery‑free, QR‑enabled temperature sensor designed for cold‑chain logistics. The device delivers affordable, real‑time, package‑level temperature data, replacing error‑prone visual indicators. By allowing remote data access, it improves recipient adoption and streamlines monitoring for shippers. The...
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...
EyePoint Inc (EYPT) Q4 2025 Earnings Call Transcript
EyePoint reported Q4 2025 results, highlighting rapid enrollment of over 900 patients in two pivotal wet AMD Phase 3 trials (Lugano and LUCHIA) with top‑line data expected mid‑2026. The company also initiated Phase 3 DME studies (COMO and CAPREIT) slated for first...
Keros Therapeutics Inc (KROS) Q4 2025 Earnings Call Transcript
Karyopharm Therapeutics reported Q4 2025 total revenue of $34.1 million, an 11.8% year‑over‑year increase, driven primarily by XPOVIO net product revenue of $32.1 million. The company trimmed R&D and SG&A expenses, improving operating performance by roughly 43% quarter‑over‑quarter, yet posted a GAAP...
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...
Niagen Bioscience Inc (NAGE) Q4 2025 Earnings Call Transcript
Niagen Bioscience reported Q2 2025 revenue of $31.1 million, a 37% year‑over‑year increase, and lifted full‑year revenue guidance to 22‑27%. Gross margin improved to 65% driven by a higher‑margin sales mix and inventory efficiencies. The company expanded its Niagen Plus clinic...
![[Comment] Considerations for Improving Non-Inferiority Trials](https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/0f1a72bb75d13d20256f6082d5abd222.webp)
[Comment] Considerations for Improving Non-Inferiority Trials
Non‑inferiority trials are increasingly employed to evaluate new therapies that may offer advantages beyond efficacy, such as safety or convenience. However, their design and analysis present unique methodological challenges that can compromise study validity. The commentary outlines essential considerations, including...
Sino Biopharm Unit Licenses Blood Cancer Drug to Sanofi for up to $1.53 Bln
Sino Biopharm’s subsidiary Chia Tai Tianqing Pharmaceutical Group has signed an exclusive global licensing deal with Sanofi for its blood‑cancer drug rovadicitinib. The agreement provides Sanofi with worldwide rights to develop, manufacture and commercialize the oral JAK/ROCK inhibitor, and includes...
TGA Seeks Public Feedback on New Manufacturing Rules for Medicines, Medicinal Gases
The Therapeutic Goods Administration (TGA) has launched two concept papers for public consultation, proposing revisions to PIC/S GMP Annex 6 for medicinal gases and Annex 15 for qualification and validation processes. The Annex 6 update seeks to reflect modern manufacturing technologies and computerized...
Moderna Won’t Risk a Public Jury Verdict Today
If you were Moderna, would you have gambled going in front of a jury made up of the general public these days? I don’t think so.
GU26 Prostate Cancer Strategic Intelligence Report
Following strong interest in the ASH25 hematologic malignancy intelligence report, Biotech Strategy Blog released a new strategic intelligence series covering the ASCO GU meeting data on prostate, bladder, and renal cell cancers. The first installment focuses on advanced prostate cancer,...
FDA Approves First Generic of Flovent HFA for Treatment of Asthma
The FDA has approved the first generic version of Flovent HFA, a fluticasone propionate inhalation aerosol delivering 44 µg per actuation for asthma maintenance in patients aged four and older. The generic, produced by Glenmark Specialty SA, matches the brand’s safety and efficacy...
Oculis Reports Q4 and Full Year 2025 Financial Results and Provides Company Update
Oculis Holding AG reported a transformative 2025, highlighted by a $268.7 million cash position that extends its runway to 2029 and a $210 million equity raise. The company secured FDA Breakthrough Therapy designation for Privosegtor, its neuro‑protective candidate for optic neuritis, and...