IntraBio Reports the US FDA’s sNDA Submission of Aqneursa for Ataxia-Telangiectasia
IntraBio has filed a supplemental New Drug Application (sNDA) with the U.S. FDA seeking approval of Aqneursa (levacetylleucine) for Ataxia‑Telangiectasia (A‑T). The filing is supported by a Phase III trial that met its primary and key secondary endpoints and demonstrated a favorable safety profile in both adult and pediatric patients. Aqneursa is already approved in the United States and Europe for neurological manifestations of Niemann‑Pick disease type C. The new indication could broaden the drug’s market and address a significant unmet need in a rare neurodegenerative disorder.
HUTCHMED Initiates P-III Trial of HMPL-760 + R-GemOx for R/R Diffuse Large B-Cell Lymphoma in China
HUTCHMED has launched a Phase III trial of HMPL‑760 combined with R‑GemOx in relapsed/refractory diffuse large B‑cell lymphoma (DLBCL) patients in China, dosing the first patient on March 20, 2026. The study will enroll approximately 240 patients who have failed first‑line therapy and...
Dizal Reports the P-III (WU-KONG28) Trial Results on Zegfrovy (Sunvozertinib) in EGFRm NSCLC
Dizal announced topline results from its Phase 3 WU‑KONG28 trial, comparing oral once‑daily Zegfrovy (sunvozertinib) to platinum‑based chemotherapy as first‑line treatment for advanced NSCLC with EGFR exon 20 insertion mutations. The study met its primary endpoint, demonstrating a statistically significant improvement in...
LivaNova’s Aura6000 System Secures the US FDA Premarket Approval to Treat Obstructive Sleep Apnea (OSA)
LivaNova received FDA Premarket Approval for its aura6000 neurostimulation system, targeting adults with moderate‑to‑severe obstructive sleep apnea (OSA) who cannot use or have failed first‑line therapies. The approval is based on the OSPREY randomized controlled trial, which showed a 65%...
Nia Therapeutics’ Smart Neurostimulation System Receives FDA Breakthrough Device Designation to Treat Memory Loss
The U.S. Food and Drug Administration granted Breakthrough Device Designation to Nia Therapeutics’ Smart Neurostimulation System (SNS) for treating episodic memory loss in adults with moderate to severe traumatic brain injury. The fully implantable, closed‑loop system records neural activity from...
AL-S Pharma Reports the P-II (AP-101-02) Trial Data on AP-101 for Amyotrophic Lateral Sclerosis (ALS)
AL‑S Pharma released Phase II (AP‑101‑02) data evaluating intravenous AP‑101 every three weeks in 73 ALS patients, including 52 with sporadic disease and 21 with SOD1 mutations. The trial met its primary safety and tolerability endpoint and demonstrated disease modification,...
Myriad Genetics Receives the US FDA Approval for MyChoice CDx Test as a Companion Diagnostic for GSK’s Zejula
Myriad Genetics announced FDA approval of its MyChoice CDx test as a companion diagnostic for GSK’s Zejula (niraparib) in advanced ovarian cancer. The clearance follows the PRIMA trial, which showed that patients identified as HRD‑positive derived significant benefit from Zejula...
Samsung Bioepis Partners with Sandoz for Up to Five Biosimilar Candidates
Samsung Bioepis and Sandoz have signed a global license, development and commercialization agreement covering up to five biosimilar candidates, including the preclinical SB36 biosimilar to Entyvio (vedolizumab). Samsung Bioepis will manage development, manufacturing and regulatory submissions, while Sandoz will handle...
Cogent Biosciences Reports US FDA’s NDA Acceptance of Bezuclastinib for NonAdvanced Systemic Mastocytosis (NonAdvSM)
Cogent Biosciences announced that the U.S. FDA has accepted its new‑drug application for bezuclastinib in non‑advanced systemic mastocytosis (NonAdvSM), with a PDUFA target date of December 30 2026 and no advisory committee required. The filing is supported by the SUMMIT trial, which...
Ascendis Pharma Reports Topline P-II (New InsiGHTS) Trial Data on TransCon hGH in Turner Syndrome
Ascendis Pharma released topline results from its Phase‑II New InsiGHTS trial evaluating TransCon hGH (lonapegsomatropin) administered once‑weekly versus daily somatropin in 49 prepubertal children with Turner syndrome. At week 52, the weekly formulation achieved an LS mean annualized height velocity...
Voro Therapeutics Collaborates with Daiichi Sankyo to Develop Tumor-Activated ADCs
Voro Therapeutics has signed a research collaboration with Daiichi Sankyo’s San Diego research institute to create tumor‑activated antibody‑drug conjugates (ADCs) using Voro’s PrimeBody platform. The partnership will focus on masked ADCs that employ proprietary masking domains and protease‑cleavable linkers to achieve...
EMA Marketing Authorization of New Drugs in February 2026
In February 2026 the European Commission and the CHMP granted marketing authorisations and positive opinions for a slate of innovative therapies spanning COVID‑19, oncology, cardiology, immunology and rare diseases. Notable approvals include Moderna’s mNEXSPIKE COVID‑19 vaccine, Hansoh’s Aumseqa for EGFR‑mutated...
Pfizer Reports P-II (FOURLIGHT-1) Trial Data on Atirmociclib Combination for 2L Metastatic Breast Cancer
Pfizer disclosed topline Phase II (FOURLIGHT‑1) data showing that atirmociclib combined with fulvestrant outperformed fulvestrant alone or everolimus + exemestane in 264 HR+, HER2‑negative metastatic breast cancer patients previously treated with CDK 4/6 inhibitors. The trial met its primary endpoint, delivering a statistically significant...
AstraZeneca Secures the EC Approval of Imfinzi as a Perioperative Therapy for G/GEJ Cancers
The European Commission has granted approval for AstraZeneca’s Imfinzi (durvalumab) combined with FLOT chemotherapy as a perioperative treatment for resectable, early‑stage and locally advanced gastric and gastro‑esophageal junction (G/GEJ) cancers. The decision is based on the Phase III MATTERHORN trial, which...
Bayer Reports the P-III (FIND-CKD) Trial Data on Kerendia in Non-Diabetic Chronic Kidney Disease
Bayer disclosed results from the pivotal Phase III FIND‑CKD trial evaluating Kerendia (finerenone) in more than 1,500 adults with non‑diabetic chronic kidney disease. Patients received 10 mg or 20 mg of Kerendia alongside standard of care and were compared with placebo. The study...
Roche Receives CE Mark for Its Elecsys ApoE4 Test to Support Blood-Based Alzheimer’s Biomarker Testing
Roche has secured CE Mark approval for its Elecsys ApoE4 in‑vitro diagnostic immunoassay, a blood‑based test that detects the ApoE4 gene variant linked to Alzheimer’s disease. In a validation study of 607 patients with cognitive complaints, the assay achieved 100%...
Otsuka Pharmaceutical Reports OLE Study Data on Repinatrabit in Phenylketonuria
Otsuka Pharmaceutical disclosed early open‑label extension (OLE) data for repinatrabit (JNT‑517) in adolescents with phenylketonuria (PKU). A 75 mg twice‑daily regimen achieved a 67% mean reduction in blood phenylalanine by day 56, with responses observed across prior sapropterin responders, non‑responders, and a...
Why Revenue Performance Often Starts with Accurate Clinical Notes
Hospitals that prioritize accurate clinical notes see measurable revenue gains because documentation directly drives coding and reimbursement. Vague or incomplete entries cause denied claims, audit flags, and lost tariffs, even when billing tools are sophisticated. Solutions such as Scribe X and...
PRISM BioLab and Receptor.AI Partner to Develop a Drug Discovery Platform
PRISM BioLab has teamed with Receptor.AI to build an AI‑driven, physics‑guided platform for discovering orally available small molecules that target intracellular protein‑protein interactions, membrane proteins, and complex receptor systems. The collaboration fuses PRISM’s PepMetics technology—3‑dimensional scaffolds that mimic α‑helix and...
Ultragenyx Reports the P-III (Enh3ance) Trial for DTX301 AAV8 Gene Therapy in OTC Deficiency
Ultragenyx announced Phase 3 Enh3ance data for its DTX301 AAV8 gene therapy targeting ornithine transcarbamylase (OTC) deficiency. At week 36, the therapy lowered 24‑hour plasma ammonia by 18% versus placebo and kept levels in the normal range, even as scavenger drug use...
Natera Launches Zenith Genomics in the US to Diagnose Rare Diseases
Natera announced the commercial launch of Zenith Genomics, a next‑generation whole‑genome sequencing (WGS) assay aimed at diagnosing rare and ultra‑rare diseases in the United States. The platform pairs standard WGS with long‑read sequencing confirmation to capture complex genomic features such...
PharmaShots Magazine-March-2026 Edition
AI is reshaping clinical trial oversight by moving from periodic checks to continuous, real‑time intelligence. Advanced analytics and machine learning now power predictive, risk‑based monitoring that aggregates data from decentralized sites, wearables, ePROs and multiple clinical systems. This integration enables...
Aplagon Doses First Patient in P-IIa (HEALING) Trial of APAC for Peripheral Arterial Occlusive Disease/Chronic Limb Threatening Ischemia
Aplagon Therapeutics has administered the first dose of its intravenous APAC candidate to a patient in a Phase 2a (HEALING) trial for peripheral arterial occlusive disease leading to chronic limb‑threatening ischemia in Finland. The study will enroll approximately 42 CLTI...
Insulet Reports EVOLUTION 2 Study Results for FCL Automated Insulin Delivery System in Type 2 Diabetes
Insulet presented data from its EVOLUTION 2C feasibility study of a fully closed‑loop (FCL) automated insulin delivery system for adults with type 2 diabetes at ATTD'26. In the 24‑patient trial, the final algorithm raised time‑in‑range (TIR) to an average 68%,...
George Medicines Partners with Ahngook Pharmaceutical to Commercialize GMRx2 in Korea
George Medicines has signed an exclusive licensing and supply agreement with South Korea’s Ahngook Pharmaceutical to bring its triple‑combination antihypertensive pill, GMRx2, to the Korean market. The single‑pill formulation blends telmisartan, amlodipine and indapamide in three dose strengths, aiming for...
C2N Diagnostics Partners with BeauBrain Healthcare to Offer PrecivityAD2 Blood Test for Alzheimer’s Disease in South Korea
C2N Diagnostics has signed a partnership with BeauBrain Healthcare to introduce its PrecivityAD2 blood test for Alzheimer’s disease in South Korea, targeting patients aged 50 and older with mild cognitive impairment or dementia. Clinical studies published in JAMA and npj...
Cairn Surgical Reports the FDA De Novo 510(k) Submission for BCL System to Improve Accuracy of Lumpectomy
Cairn Surgical has filed a De Novo 510(k) application with the U.S. FDA for its Breast Cancer Locator (BCL) System, a device designed to improve the accuracy of breast‑conserving surgery. The company completed a pivotal U.S. trial that demonstrated a...
UCB Reports P-III (BE BOLD) Trial Results on Bimzelx (Bimekizumab) in Active Psoriatic Arthritis
UCB announced results from its Phase III BE BOLD trial comparing Bimzelx (bimekizumab) to risankizumab in 553 adults with active psoriatic arthritis. The study met its primary endpoint, showing Bimzelx superior in achieving ACR50 responses at week 16. Bimzelx is...
ARTHEx Biotech’s ATX-01 Secures the US FDA Fast Track Designation for Myotonic Dystrophy Type 1
ARTHEx Biotech announced that its RNA‑based drug ATX‑01 has received U.S. FDA Fast Track designation for treating Myotonic Dystrophy Type 1 (DM1). The therapy works by inhibiting miR‑23b, thereby increasing free MBNL protein, correcting splicing errors and reducing toxic DMPK mRNA...
Rapport Therapeutics Partners with Tenacia Biotechnology to Advance RAP-219 in Greater China
Rapport Therapeutics has granted Tenacia Biotechnology exclusive rights to develop and commercialize its TARPγ8‑specific AMPA receptor negative allosteric modulator RAP‑219 in Greater China, covering indications such as focal onset seizures and bipolar mania. The agreement provides Rapport with a $20 million...
Bridging Innovation and Clinical Reality: Shalabh Gupta, CEO of Unicycive Therapeutics, on the Future of Biotech
Shalabh Gupta, CEO of Unicycive Therapeutics, argues that biotech can benefit from tech‑driven design thinking, rapid prototyping, and disciplined capital allocation. He highlights how the company repurposed automotive battery‑shrinkage technology to create Oxylanthanum carbonate, a kidney‑disease candidate now under FDA...
TaiGen Completes Enrolment and Doses First Patient with Insilico Medicine’s ISM4808 in P-I Trial
Insilico Medicine and TaiGen announced that enrollment for the Phase‑I trial of ISM4808, an AI‑designed therapy for CKD‑related anemia, is now complete and the first patient has been dosed. The study comprises single‑ascending‑dose and multiple‑ascending‑dose cohorts evaluating safety and pharmacokinetics...
Johnson & Johnson Reports the US FDA Approval of Tecvayli + Darzalex Faspro for R/R Multiple Myeloma
Johnson & Johnson announced FDA approval of the Tecvayli (teclistamab) and Darzalex Faspro (subcutaneous daratumumab with hyaluronidase) combination for adults with relapsed/refractory multiple myeloma who have received at least one prior line of therapy. The approval is grounded in the Phase III...
Harrison.ai Secures the US FDA 510(k) Clearance for Acute Infarct Triage on Non-Contrast CT Brain
Harrison.ai has received U.S. FDA 510(k) clearance for its Acute Infarct Triage software, which analyzes non‑contrast CT brain scans to identify acute ischemic strokes. In validation studies the algorithm achieved roughly 89% sensitivity on thin‑slice CT and 86% on thicker...
Roche and Zealand Pharma Report P-II (ZUPREME-1) Trial Data on Petrelintide in Obesity
Roche and Zealand Pharma disclosed Phase‑II (ZUPREME‑1) results for petrelintide, an amylin‑analog injected weekly, in 493 overweight or obese adults (mean BMI 37 kg/m²) with weight‑related comorbidities. The trial evaluated five dose levels against placebo over 42 weeks and met its primary endpoint,...
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...
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...
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...
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...
Key Biosimilars Events of February 2026
February 2026 saw a surge of biosimilar activity, with key regulatory approvals for products such as Sandoz Enzeevu (Eylea), Accord Filkri (Neupogen), and STADA Gotenfia (Simponi) across the US, Canada and the EU. Companies forged new licensing and commercialization deals that extend biosimilar...
Lynk Pharmaceuticals Reports Topline P-III Trial Data on Zemprocitinib in Atopic Dermatitis
Lynk Pharmaceuticals announced topline Phase III data for its oral JAK inhibitor zemprocitinib in 356 patients with moderate‑to‑severe atopic dermatitis. Both the 12 mg and 24 mg doses met the co‑primary endpoints at week 16, delivering 38‑46 percentage‑point improvements in EASI‑75 and roughly 30 percentage‑point gains...
Innovent Receives the NMPA Approval for Jaypirca to Treat R/R Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma (CLL/SLL)
Innovent’s pirtobrutinib, marketed as Jaypirca, received Chinese NMPA approval for adults with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma who have undergone at least one prior therapy, including a BTK inhibitor. The decision follows the phase‑III BRUIN CLL‑321 trial,...
Merck and Eisai Present P-III (LITESPARK-011) Trial Data on Welireg + Lenvima in Advanced Renal Cell Carcinoma (RCC) at ASCO...
Merck and Eisai disclosed Phase III LITESPARK-011 results showing that the oral combination of Welireg (belzutifan) and Lenvima (lenvatinib) outperformed cabozantinib in 747 patients with advanced renal cell carcinoma who progressed after anti‑PD‑1/PD‑L1 therapy. At a median follow‑up of 29...
Gyre Therapeutics to Acquire Cullgen for ~$300M
Gyre Therapeutics announced an all‑stock acquisition of Cullgen valued at roughly $300 million, slated to close in Q2 2026. The deal will make Cullgen a wholly owned subsidiary and integrate its targeted protein degradation (TPD/DAC) platform into Gyro’s pipeline. Post‑close, the combined...
CPHI Middle East 2026 | 11-13 May, Riyadh
CPHI Middle East will return to Riyadh in May 2026, building on the 2024 edition that attracted over 30,000 visitors from more than 100 countries. The event promises expanded networking opportunities, direct market access, and the latest regulatory and innovation...
Vueway Expands Pediatric Use: Alberto Spinazzi Shares Bracco Group’s Vision
The European Union has granted approval for Vueway (gadopiclenol) to be used in neonates, infants, and toddlers, marking a pivotal expansion of pediatric MRI contrast agents. Vueway delivers diagnostic‑grade imaging at half the dose of conventional macrocyclic GBCAs, thanks to...
Zymeworks Raises $250M From Royalty Pharma Backed by Ziihera Royalties
Zymeworks announced a $250 million non‑recourse, royalty‑backed note from Royalty Pharma, secured against 30% of worldwide tiered royalties on its Ziihera program licensed to Jazz Pharmaceuticals and BeOne Medicines. The note will be repaid until Royalty Pharma receives 1.65 times the principal...
Ascendis’ Yuviwel (Navepegritide) Receives the US FDA Accelerated Approval for Achondroplasia
The U.S. FDA granted accelerated approval to Ascendis’ Yuviwel (navepegritide) for increasing linear growth in children aged two years and older with achondroplasia and open epiphyses. Approval rests on three placebo‑controlled trials and up to three years of open‑label extension...
X4 Pharmaceuticals’ Xolremdi (Mavorixafor) Receives the CHMP Positive Opinion for WHIM Syndrome
X4 Pharmaceuticals’ Xolremdi (mavorixafor) received a positive opinion from the European CHMP, recommending approval under exceptional circumstances with an EC decision expected in Q2 2026. The recommendation is based on the global Phase III 4WHIM trial involving 31 patients aged 12 and...
Ipsen Reports the CHMP Positive Opinion for Ojemda (Tovorafenib) for R/R BRAF-Altered Pediatric Low-Grade Glioma (pLGG)
European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending conditional approval of Ipsen’s Ojemda (tovorafenib) as monotherapy for pediatric low‑grade glioma with BRAF fusions, rearrangements or V600 mutations. The recommendation is based on...