Know What's Happening in Pharma
Know What's Happening in Pharma
FDA greenlights KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA approved KRESLADI, a gene‑editing treatment from Rocket Pharmaceuticals for severe leukocyte adhesion deficiency‑I in children lacking a bone‑marrow donor. It is the first product directly funded by California’s Institute for Regenerative Medicine, highlighting CIRM’s role in translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma
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 such as natural immunity in cancer‑free smokers, hyper‑activating growth signals, the “dark proteome,” transient DNA damage, and brain‑body interoception. Funding will be delivered over the next five years under the joint stewardship of Cancer Research UK and the US National Cancer Institute.
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,...
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,...
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...
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, 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...
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...
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 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....
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 (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...
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...
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 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...
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...
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...
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...
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...
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 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...
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 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 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...
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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’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...
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...
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,...
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 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...
Virginia lawmakers are proposing a prescription‑drug affordability board that will use Medicare’s annually negotiated drug list as its benchmark. The board would also impose upper payment limits, creating a ceiling on what insurers pay for those medicines. This strategy would...
Novo Nordisk announced a $506 million investment to expand its manufacturing site in Ireland, aiming to boost capacity for diabetes and obesity therapies. The expansion addresses rising global demand and enhances supply‑chain resilience. The announcement coincides with industry focus on measuring...
SpotSee and Controlant have launched a strategic partnership that combines SpotSee’s WarmMark QR temperature indicators with Controlant’s real‑time IoT monitoring devices and cloud analytics. The joint solution creates a layered, end‑to‑end visibility platform that captures environmental data during transit and...
A phase‑1 pilot of low‑dose intralesional cemiplimab in early‑stage cutaneous squamous cell carcinoma (CSCC) demonstrated rapid tumor regression with visual objective response rates of 66.7%–75% and pathologic complete responses of 58.3%–66.7%. Safety was favorable, with no grade ≥ 3 events and minimal...
Tirzepatide: In the SURMOUNT-1 trial, weight reduction percentages were dose-dependent: 5 mg: ~15.0% weight reduction. 10 mg: ~19.5% weight reduction. 15 mg: ~20.9% weight reduction (Jastreboff et al., 2022). https://www.nejm.org/doi/10.1056/NEJMoa2206038 https://www.gatlan.com/ @GatlanHealth 
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.
The FDA granted accelerated approval to navepegritide (Yuviwel), a once‑weekly CNP prodrug, for children aged two years and older with achondroplasia and open growth plates. In the phase 2b ApproaCH trial, the drug raised annualized growth velocity to 5.89 cm/year, a 1.49 cm/year...
Prime Medicine $PRME to seek approval for gene editing treatment after two-patient trial https://t.co/nBnrUzqSCN via @Jasonmmast
New post‑hoc analyses of the phase 3 STOP‑HS1 and STOP‑HS2 trials show that oral povorcitinib, a selective JAK1 inhibitor, delivers rapid, high‑threshold lesion clearance in patients with severe hidradenitis suppurativa. In the 75 mg arm, up to 57% of participants achieved complete...
Pierre Fabre seeks to revive US approval chances for spurned cell therapy https://t.co/gJeklT8asd @ByJonGardner $ATRA $QURE $RGNX
Kyowa Kirin announced it will cease all clinical trials of rocatinlimab, an anti‑OX40 antibody once touted as a potential eczema blockbuster, after a safety review identified emerging malignancy signals. The review, conducted with former partner Amgen, uncovered a confirmed and...
Scientists at Ludwig Maximilian University discovered that loss of the DJ-1 protein triggers ATP deficiency in human dopaminergic neurons, leading to reduced VMAT2 levels and impaired dopamine vesicle loading. The resulting dopamine oxidation fuels accumulation of pathological α‑synuclein species, a...
Did the FDA’s individualized therapy guidance put a spike in the heart of a startup biotech? Fake news, says the HHS. Yeah, it did, says an insider. Exclusive from @RLCscienceboss and @Jared_Whitlock https://t.co/IN9o4VIp03
Real‑world evidence from Duke’s PRECISION platform shows Pluvicto (Lu‑177 vipivotide tetraxetan) delivers a median progression‑free survival of 13.5 months in PSMA‑positive metastatic castration‑resistant prostate cancer (mCRPC) after androgen‑receptor pathway inhibitor (ARPI) therapy, matching the pivotal PSMAfore trial. Patients who received Pluvicto...
The FDA granted priority review to Takeda’s rusfertide, a first‑in‑class hepcidin mimetic for polycythemia vera, after Phase III trials more than doubled response rates. A regulatory decision is expected in Q3 2026. The agency also expanded Novo Nordisk’s once‑weekly Sogroya to treat children...
The FDA’s Drug Competition Action Plan (DCAP) continues to drive generic drug competition by streamlining standards for complex products, closing loopholes that allow brand‑name companies to delay approvals, and improving the overall ANDA review process. Aligned with GDUFA III, the plan...
U.S. pharmaceutical tariffs on EU imports have risen to 15% after a brief 10% measure, reviving trade tensions despite a prior MFN deal. Europe is pursuing alternative markets, notably a new EU‑India free‑trade agreement that could eliminate up to 11%...
A new systematic review of LLMs in medicine @NatureMedicine @ekoermann https://t.co/npvDeqUvel “Despite thousands [4,609] of publications since late 2022, only a small fraction use real clinical data and just 19 randomized trials exist.”
New integrated safety data from 20 trials confirm that ruxolitinib 1.5% cream delivers a low incidence of serious infections, cardiovascular events, thromboembolic events, and malignancies across atopic dermatitis, vitiligo and other inflammatory skin conditions. A phase 3b trial in adults with...
The FDA has published a batch of newly added guidance documents, spanning drug exclusivity, post‑approval safety reporting, real‑world data studies, Bayesian trial methods, and patient‑preference research. Ten documents are highlighted, with six in draft form and four finalized, dated between...
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...
