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.
FDA Accepts Opus Genetics’ OPGx‑LCA5 Gene Therapy Into Rare Disease Evidence Program
Opus Genetics announced that its investigational LCA5 gene therapy, OPGx‑LCA5, has been accepted into the FDA’s Rare Disease Evidence Principles (RDEP) program. The acceptance aligns the company with early‑stage regulatory collaboration for a Phase 3 trial targeting Leber congenital amaurosis type 5, an ultra‑rare inherited retinal disease. The move could accelerate evidence generation for a condition with no approved treatments.
Top Biotech Deals in April 2026
April 2026 marked a flurry of blockbuster biotech mergers, highlighted by Sun Pharma’s $11.75 billion acquisition of Organon, giving it a foothold in women’s health across 140 countries. Eli Lilly emerged as the most active buyer, spending roughly $11.5 billion on four...
BriaCell Therapeutics Reports US FDA IND Clearance to Initiate P-I/II Trial of Bria-BRES+ in Breast Cancer
BriaCell Therapeutics announced that the U.S. Food and Drug Administration has granted IND clearance to launch a combined Phase I/II study of its Bria‑BRES+ therapy in patients with metastatic breast cancer. Preclinical results presented at AACR indicate Bria‑BRES+ stimulates both...
Single 25 Mg Psilocybin Dose Triggers Lasting Brain Entropy and Boosts Well‑Being
Researchers at UCSF and Imperial College London gave 28 psychedelic‑naïve volunteers a 25 mg psilocybin dose, finding increased brain entropy and structural changes that correlated with higher psychological insight and well‑being a month later. The findings fuel the debate over whether...
Switzerland’s Biotech Sector Defies Tough Markets with Record Revenue
Switzerland’s biotech sector posted a record CHF 7.5 billion ($8.3 billion) in revenue for 2025, up from CHF 7.2 billion the year before, driven by more market‑ready products and strong CDMO demand. Capital inflows rose 2.1% to CHF 2.6 billion ($2.9 billion), with privately financed firms leading the...
FDA Approves Selpercatinib for Medullary Thyroid Cancer with a RET Mutation
The FDA granted traditional approval on September 27, 2024, for Eli Lilly’s selpercatinib (Retevmo) to treat adult and pediatric patients two years and older with advanced or metastatic medullary thyroid cancer (MTC) harboring a RET mutation. The decision follows accelerated approvals...
SCIENTURE Announces Third Patent Grant for ARBLI™ (Losartan Potassium) Oral Suspension, Strengthening Long-Term Market Exclusivity Through 2041
Scienture Holdings announced that the U.S. Patent and Trademark Office granted a third patent (U.S. Patent No. 12,605,365) for its ARBLI™ losartan potassium oral suspension. The new patent, issued April 21, 2026, extends exclusive protection through October 7, 2041, reinforcing the company’s IP moat. ARBLI™...
Novelty Nobility Expands AGC Biologics Deal to Take Product Candidate Through GMP Manufacturing
Korea‑based Novelty Nobility has expanded its contract with CDMO AGC Biologics to move its bispecific antibody NN4101 through process development and GMP manufacturing at AGC’s Chiba, Japan facility. Cell‑line development was completed in Copenhagen and will be transferred to Chiba...
Macrocycles: Big Is the New Beautiful
Macrocycles are gaining traction as a hybrid drug modality that combines the high specificity of biologics with the oral accessibility of small molecules. Advances in synthetic chemistry, AI‑driven design, and DNA‑encoded libraries now enable rapid exploration of billions of candidates,...
FDA Panel Backs AstraZeneca’s Truqap for PTEN-Deficient Prostate Cancer
The FDA’s Oncologic Drugs Advisory Committee voted 7‑1 to endorse AstraZeneca’s Truqap (capivasertib) with abiraterone and ADT for PTEN‑deficient metastatic hormone‑sensitive prostate cancer, citing a 19% reduction in radiographic progression or death. The recommendation could pave the way for the...
Alebund Pharmaceuticals Completes Patient Enrollment in P-III (RESPOND-2) Trial of AP301 for Hyperphosphatemia
Alebund Pharmaceuticals announced that patient enrollment is complete for its global Phase III RESPOND‑2 trial of AP301, a novel therapy for hyperphosphatemia in dialysis patients. The study enrolled 282 participants—138 in the United States and 144 in China—exceeding the planned 264....
4basebio Announces Lease of an Innovation Hub and Manufacturing Facility in Cambridge, UK
4basebio PLC announced a lease for a 26,500 sq ft innovation hub and manufacturing facility in Cambridge, UK, slated to open in late summer 2026. The site will feature 7,500 sq ft of specialized laboratory space with 15 labs, advanced biosafety equipment, and a...
Calla Lily Clinical Care Doses First Patients in Clinical Trial for Intravaginal Drug Delivery Platform for Threatened Miscarriage
Calla Lily Clinical Care has begun dosing the first participants in the FREEDOM clinical trial, testing its 400 mg intravaginal progesterone product Callavid. The NIHR‑funded study targets women with luteal phase insufficiency, a condition linked to threatened miscarriage and infertility. Callavid’s...
CellCentric Raises $220M to Get Multiple Myeloma Pill to Market
CellCentric, a 22‑year‑old transatlantic biotech, closed a $220 million Series D round to accelerate development of its oral multiple myeloma drug. The capital will fund the completion of Phase III trials, scale manufacturing, and prepare regulatory filings. The therapy is designed for patients...
Pfizer, Lilly, More Report Q1, FDA Names Acting CBER Director and an ALS Awakening
Pfizer posted first‑quarter earnings that topped consensus estimates, yet analysts deemed the beat insufficient given higher expectations for the New York‑based giant. Eli Lilly delivered a striking 56% revenue surge, though the company noted that price reductions on several products capped its...
Bayer to Buy Perfuse Therapeutics for $300M Upfront
Bayer announced a $300 million upfront payment to acquire ophthalmology biotech Perfuse Therapeutics, securing its mid‑stage program aimed at the leading causes of blindness. The transaction represents Bayer’s first pharmaceutical merger‑and‑acquisition in several years, signaling a renewed focus on specialty eye...
Capturing True Single-Cell Resolution with Your Spatial Data
Spatial biology has transformed life‑science research, yet imaging and sequencing platforms still grapple with cell‑boundary segmentation and grid‑based spot limitations. Linda Orzolek of OMAPiX explains how Takara Bio’s Trekker technology delivers true single‑cell spatial resolution by isolating nuclei and pairing...
DDW Highlights: 6 May 2026
In this episode, Bruno Quinney highlights a surge in UK biotech financing, noting £552 million ($746 million) in Q1 2026 equity funding and the UK's capture of 57% of European biotech venture capital. He also covers the UK’s sweeping clinical‑trial reforms aimed at...
Signadori Bio Raises €11M to Advance Tumour Platform
Signadori Bio announced an €11 million ($11.9 million) seed extension to fund its in‑vivo monocyte engineering platform for solid tumours. The Series A round, led by Taiho Ventures with Sofinnova and Invivo Partners, will support lead candidate nomination, platform refinement, and team expansion...
Novo Nordisk's Q1 in Four Words: Pills, Pricing, Payments and Pressure
Novo Nordisk’s first‑quarter performance centered on its obesity drug Wegovy, which now serves roughly one million patients in the United States. The company is navigating heightened pricing pressure as insurers push for larger rebates while regulators scrutinize the drug’s cost....
FDA Approves Incyte’s Once‑Daily Jakafi XR for Myelofibrosis, Polycythemia Vera and GVHD
The U.S. FDA has approved Incyte’s Jakafi XR, a once‑daily extended‑release ruxolitinib tablet, for adults with intermediate‑ or high‑risk myelofibrosis, polycythemia vera patients who cannot use hydroxyurea, and patients 12 years and older with certain graft‑versus‑host disease forms. The decision follows...
From Insight to Impact: Making Real-World Evidence Actionable in Urology
Real‑world evidence (RWE) is reshaping urology, but fragmented, unstructured data limits its impact on everyday clinical decisions. In non‑muscle invasive bladder cancer, real‑world datasets reveal wide variation in BCG maintenance and treatment sequencing, while prostate cancer diagnostics suffer from inconsistent...
Can Mammals Regrow Lost Limbs? This New Treatment Could Be the First Step
Researchers at Texas A&M have demonstrated that a two‑step treatment using growth factors FGF2 and BMP2 can trigger partial digit regeneration in mice. The protocol first applies FGF2 to create a blastema‑like cell mass, then adds BMP2 to drive bone...
Sickle Cell Disease After Casgevy: Seven Companies to Watch in 2026
Casgevy’s $2.2 million, ex vivo CRISPR cure proved sickle cell disease can be edited at its genetic root, but its complex manufacturing and conditioning limit broad access. In response, a wave of innovators is targeting simpler, safer, and more scalable solutions—from Beam’s...
Cytospire Therapeutics Secures £61m to Advance Cancer Treatment
Cytospire Therapeutics, a UK biotech, closed a £61 million (≈$77 million) Series A round to accelerate its multispecific engager antibody platform. The capital will fund the development of its lead candidate, CYT X300, aimed at EGFR‑positive solid tumours such as colorectal, head‑and‑neck and non‑small‑cell...
Overcoming Material Constraints: Aseptic Filling Solutions for Rare or Expensive APIs
Aseptic fill‑finish lines can waste more than a liter of drug product per batch, translating to losses exceeding $1 million for high‑value APIs. The bulk of this loss occurs during sterile filtration and filling, where product remains trapped in vessels, tubing,...
Furin‐Mediated Intracellular Aggregation of Radioactive Molecules for Enhanced Radionuclide Imaging and Tumor Therapy
Researchers have engineered a furin‑responsive radioactive probe, RVRR‑TPE, that self‑assembles into nanoparticles inside furin‑positive cancer cells. The molecule couples a furin‑cleavable Arg‑Val‑Arg‑Arg peptide, an aggregation‑induced emission fluorophore (tetraphenylethene), and a phenol group for iodine‑125/131 labeling. In mouse models, the 125I/131I‑labeled...
STAT+: Even at a Meeting in Rome, FDA Shifts Are Top of Mind for Gene Therapy Field
At a gene‑therapy summit in Rome, Tim Hunt highlighted recent FDA approvals of rare‑disease treatments from Rocket Pharmaceuticals and Regeneron as signs of progress. He also flagged the departure of Vinay Prasad, the FDA’s top regulator for cell and gene therapies,...
Caris Launches Caris MI Clarity for AI-Powered Breast Cancer Recurrence Risk Assessment
Caris Life Sciences has introduced Caris MI Clarity, an AI‑driven prognostic test that evaluates both early (0‑5 years) and late (5‑15 years) distant recurrence risk for postmenopausal patients with HR‑positive/HER2‑negative, node‑negative early‑stage breast cancer. The assay analyzes digitized H&E pathology...
With Large DNA Fragment Assembly, Scientists Can Design Microbes that Produce Countless Complex Products
A new review in Quantitative Biology shows scientists can now reliably assemble very large DNA fragments, enabling the construction of whole metabolic pathways and even extra chromosomes inside microbes. This capability turns yeast and bacteria into efficient cell factories that...
Addressing Treatment Gaps in Gout
In this episode, Crystallis Therapeutics CEO James McKay explains the biology of gout, why existing urate‑lowering drugs often fail, and how the company’s next‑generation URAT1 inhibitor, detenurad, aims to close the large treatment gap for moderate‑to‑severe patients. He highlights that...
Popular GLP-1 Drugs Significantly Reduce Major Cardiovascular Events,
A systematic review and meta‑analysis of eleven cardiovascular outcome trials involving 91,490 high‑risk patients found that GLP‑1 receptor agonists reduce major adverse cardiovascular events (MACE) by 14% compared with placebo. The therapy also lowered cardiovascular mortality by 13% and improved...
Aurobindo Pharma Unit-VII Receives FDA VAI Classification
Aurobindo Pharma Unit-VII Gets US FDA VAI Classification Aurobindo Pharma's Unit-VII oral solid dosage facility in Telangana receives Voluntary Action Indicated (VAI) classification from US FDA, closing the inspection with 9 Form 483 observations. AUROPHARMA 1,471.60 43.50 (3.05%)
FDA Clears IND for Cellenkos' CK0802, First‑in‑Class Treg Therapy for Steroid‑Refractory GVHD
Cellenkos announced that the U.S. FDA has cleared its Investigational New Drug application for CK0802, enabling a multicenter Phase 1b/2a study in steroid‑refractory graft‑versus‑host disease. The trial, set to begin in the second half of 2026, aims to assess safety...
Lupin Secures FDA Approval for Generic Glycerol Phenylbutyrate Oral Liquid for UCDs
Lupin Limited won U.S. FDA clearance for its glycerol phenylbutyrate oral liquid, a generic version of Horizon Therapeutics' Ravicti, to treat chronic urea cycle disorders. The approval gives patients a new, potentially lower‑cost option and marks Lupin's expanding footprint in...
FDA Grants First Oral SERD Approval to Arvinas' Vepdegestrant for ESR1‑Mutant Breast Cancer
The FDA approved Arvinas' oral selective estrogen receptor degrader (SERD) vepdegestrant for ESR1‑mutant advanced breast cancer, citing a hazard ratio of 0.57 and a median progression‑free survival of 5.0 months versus 2.1 months on fulvestrant. The decision came five weeks...
“Sounds Great. Do You Want FDA Approval?” A Regulatory Analysis of Psychedelics
On April 18, 2026 President Trump signed an executive order compelling the FDA to prioritize review of psychedelic drugs, issuing priority‑review vouchers for three compounds already holding Breakthrough Therapy designation. The order also creates a Right‑to‑Try pathway for ibogaine, directs...
World's First Vaccine for Lyme Disease Could Be Available in 2027
Pfizer and French partner Valneva announced that their Lyme disease vaccine candidate PF‑07307405 (LB6V) achieved 73.2% efficacy in Phase III trials involving more than 9,000 participants. Although the study missed its primary statistical endpoint due to a low incidence of cases,...
FDA Expands Compassionate Use of Daraxonrasib for Metastatic Pancreatic Cancer
The U.S. FDA has approved an expanded‑access protocol for daraxonrasib, Revolution Medicines' KRAS‑G12D inhibitor, allowing patients with pretreated metastatic pancreatic cancer to receive the drug. The decision follows phase‑3 data that doubled survival versus standard second‑line chemotherapy, addressing a disease...
Converge Bio’s AI Platform Doubles Cetuximab Affinity in Eight Hours
Converge Bio announced that its generative‑AI platform ConvergeAB produced a cetuximab antibody with more than double the binding strength to EGFR in an eight‑hour, zero‑shot experiment. The result, filed as a provisional patent, highlights AI’s capacity to accelerate and refine...
Janus Nanomotors Offer Active Delivery for Radiation‑Induced Dermatitis
A team of nanotech researchers has introduced Janus nanomotors that autonomously move through irradiated skin to deliver anti‑inflammatory drugs, markedly reducing radiation‑induced dermatitis in animal studies. The technology converts skin‑generated hydrogen peroxide into propulsion, enabling targeted therapy with minimal systemic...
Hemophilia A Therapy by Expression Gets FDA Fast-Track, Pediatric Designation
The U.S. FDA awarded fast‑track and rare pediatric disease designations to Expression Therapeutics' investigational stem‑cell therapy for hemophilia A. The designations promise more frequent regulator interaction, rolling review, and eligibility for a priority‑review voucher, accelerating a potentially curative, one‑time treatment.
Vanda Pharmaceuticals Inc (VNDA) Q1 2026 Earnings Call Transcript
Vanda Pharmaceuticals reported Q1 2026 net product sales of $51.7 million, a modest 3% year‑over‑year increase driven primarily by a 26% surge in Fanapt revenue and a 32% rise in prescriptions. Hetlioz sales continued to decline, falling 24% YoY amid generic...
Taysha Gene Therapies Inc (TSHA) Q1 2026 Earnings Call Transcript
Taysha Gene Therapies announced that its Rett syndrome gene therapy TSHA-102 has moved into pivotal development, securing FDA Breakthrough Therapy designation and written alignment on both the REVEAL pivotal and ASPIRE trial designs. Early Phase 1/2 data showed a 100%...
Urogen Pharma Ltd (URGN) Q1 2026 Earnings Call Transcript
UroGen Pharma reported a $15.8 million 2025 revenue for its newly launched Zasturi, with the permanent J code effective Jan. 1 2026 spurring a sharp uptick in prescriber and site adoption. The company now has 838 activated sites, over 95% payer access, and...
Sarepta Therapeutics Inc (SRPT) Q1 2026 Earnings Call Transcript
Sarepta Therapeutics reported $1.86 billion net product revenue for 2025, a 16% increase, and ended the year with $954 million in cash and investments. Management guided 2026 net product revenue to $1.2‑$1.4 billion, emphasizing a low‑end outlook while targeting operating profit and positive...
Novavax Inc (NVAX) Q1 2026 Earnings Call Transcript
Novavax reported a Q1 2019 net loss of $43.2 million, improving from the prior year, while revenue fell 59% to $4 million as the Bill & Melinda Gates Foundation grant wound down. The company highlighted clinical milestones for its RSV vaccine ResVax,...
Quanterix Corp (QTRX) Q1 2026 Earnings Call Transcript
Quanterix Corp reported fourth‑quarter revenue of $43.9 million, a 25% year‑over‑year increase and 7% sequential growth, though organic revenue fell 22% as both Simoa and Spatial platforms declined over 20%. The company highlighted a $897 CMS reimbursement rate for its Lucent...
Zevra Therapeutics Inc (ZVRA) Q1 2026 Earnings Call Transcript
Zevra Therapeutics reported Q1 2026 net revenue of $36.2 million, a 78% year‑over‑year increase, driven primarily by MyPlifer sales of $24.6 million and a $10.2 million expanded‑access program. The company completed the $50 million divestiture of its SDX portfolio, recorded a $43.3 million one‑time gain...
Mineralys Therapeutics Inc (MLYS) Q1 2026 Earnings Call Transcript
Mineralys Therapeutics reported Q1 2026 results highlighting FDA acceptance of lorundrostat’s NDA with a PDUFA target of Dec 22, 2026. The company completed five trials showing durable blood‑pressure reductions, though the EXPLORER‑OSA study missed its primary AHI endpoint, it still demonstrated a...