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 Clears Oral Decitabine/Cedazuridine + Venetoclax for Elderly AML Patients
The FDA approved the oral combination of decitabine/cedazuridine (Inqovi) and venetoclax (Venclexta) for newly diagnosed acute myeloid leukemia patients who are 75 years or older or otherwise ineligible for intensive chemotherapy. The decision rests on phase‑2 ASCERTAIN‑V data showing a 46.5% complete response rate and a median overall survival of 15.5 months.
Dare Bioscience Inc (DARE) Q1 2026 Earnings Call Transcript
Daré Bioscience announced the imminent commercial launch of its DARE to PLAY sildenafil cream through a 503B outsourcing facility in December 2025, providing a near‑term revenue stream while full FDA approval is pursued. The company highlighted a strong cash position...
Pelthos Therapeutics Inc (PTHS) Q1 2026 Earnings Call Transcript
Pelthos Therapeutics reported a record Q1 2026, with U.S. BRIUMVI net product revenue of $195 million, 63% year‑over‑year growth, and total revenue of $205 million. The company raised its full‑year U.S. revenue outlook to $885‑$900 million and global guidance to about $925 million, nearing...
Jasper Therapeutics Inc (JSPR) Q1 2026 Earnings Call Transcript
Agios Therapeutics reported first‑quarter 2026 net revenues of $20.7 million, a 138% year‑over‑year increase driven by the U.S. launch of AQVESME for thalassemia. The company recorded 242 prescriptions by REMS‑certified physicians, indicating rapid early adoption. Management announced plans to file an...
Atrium Therapeutics Inc (RNA) Q1 2026 Earnings Call Transcript
Sarepta Therapeutics reported Q1 2026 revenue of $731 million, a modest 2% decline, while posting GAAP operating profit of $358 million and ending the quarter with $748 million in cash. The company reiterated full‑year revenue guidance of $1.2‑$1.4 billion and highlighted non‑cash collaboration income...
Navigating the Clinical Progress of Antibody-Drug Conjugates: Emerging Opportunities and Remaining Challenges
Antibody‑drug conjugates (ADCs) have become one of the fastest‑growing anticancer modalities, reaching 19 FDA‑approved products worldwide by the third quarter of 2025. The portfolio has expanded from hematologic indications to a broad array of solid tumors, driven by successful HER2‑targeted...
Sangamo Therapeutics Inc (SGMO) Q1 2026 Earnings Call Transcript
Sangamo Therapeutics announced that the FDA reaffirmed the use of eGFR slope as an endpoint for an accelerated Biologics License Application for its Fabry gene therapy ST-920, positioning a filing as early as Q1 2026. The STAR study showed a mean...
Pharma Leaders Hope Next FDA Head Calms Waters
PhRMA chief Steve Ubl urged the incoming FDA commissioner to restore certainty after a turbulent period marked by Marty Makary’s resignation and staff cuts. Executives from Bristol Myers Squibb and other majors echoed the need for a stable regulator to...
Recurrent Strokes Less Severe on Asundexian: OCEANIC-STROKE
The OCEANIC‑STROKE trial of 12,327 patients showed that the factor XIa inhibitor asundexian reduced recurrent ischemic strokes by 26% versus placebo in secondary prevention. Additional analyses revealed that strokes occurring on asundexian were less severe, with fewer NIHSS scores ≥8 and...
FDA Launches Public Call to Accelerate Drug Repurposing for Rare and Chronic Diseases
The U.S. Food and Drug Administration announced a formal request for public input on drug‑repurposing, aiming to speed new indications for approved medicines that address unmet medical needs. Commissioner Marty Makary highlighted the shortage of treatment options and the potential...
FDA Approves Rocket's Kresladi, First Gene Therapy for Ultra‑Rare LAD‑1
Rocket Pharmaceuticals announced that the U.S. Food and Drug Administration has approved Kresladi, its inaugural gene‑therapy product, to treat leukocyte‑adhesion deficiency type 1 (LAD‑1). The approval marks the first gene‑editing treatment for this ultra‑rare pediatric immunodeficiency and signals a new commercial...
New Drug Candidate that Reprograms the Immune System Shows Promise as a Brain Cancer Treatment
McMaster University researchers have engineered a uPAR‑targeted CAR‑T cell that reprograms the immune system to attack glioblastoma. In preclinical mouse studies, a single infusion of 1 × 10⁶ engineered T cells eradicated established brain tumors and prevented recurrence, outperforming unmodified T cells....
Sonrotoclax Granted Accelerated Approval for R/R Mantle Cell Lymphoma
The FDA granted accelerated approval to BeOne Medicine’s sonrotoclax, marketed as Beqalzi, for adults with relapsed or refractory mantle cell lymphoma (MCL) who have received at least two prior therapies, including a BTK inhibitor. In the single‑arm BGB‑11417‑201 trial of...
5 Key Regulatory Shifts From Makary's Era at the FDA
Former FDA commissioner Marty Makary reshaped U.S. drug regulation with five sweeping changes: the agency dropped the default two‑trial requirement, making a single pivotal study the norm for most new drugs; a new biosimilar guidance eliminates routine comparative efficacy trials,...
Adenine Base Editing Reverses Dravet Syndrome in Mice
Today in @ScienceTM, we report the use of in vivo adenine base editing to correct a variant causing Dravet syndrome, a severe childhood epilepsy and neurodevelopmental disorder, substantially ameliorating disease symptoms and extending lifespan in an animal model. 1/13 https://t.co/uQEwWvj94y
How Biologic Developers Are Using Optimized Platforms and Operations to Reach First-in-Human Trials
Catalent’s Emily Schirmer explains that early chemistry, manufacturing and controls (CMC) decisions and early engagement with a capable CDMO can dramatically shorten the path to first‑in‑human (FIH) trials. By evaluating the full supply chain—drug substance, product, fill‑finish and distribution—developers avoid...
Development of Local Anesthetic Drug Products With Prolonged Duration of Effect
The FDA released a draft Level 2 guidance (Docket FDA‑2023‑D‑0608) on developing local anesthetic products with prolonged duration of effect. The document outlines recommended development pathways, trial designs, and data requirements for future new‑drug applications. It aims to help sponsors generate evidence...
Cellares and ProTgen Automate Manufacturing of Progenitor T-Cell Therapy for Blood Cancer
Cellares, an integrated development and manufacturing organization, has partnered with ProTgen to automate the production and quality control of ProT-096, a personalized progenitor T‑cell therapy for refractory leukemia and other blood cancers. Cellares will deploy its Cell Shuttle and Cell...
Rohto Pharmaceutical Posts 11% Revenue Rise and 11% Profit Jump in FY 2025
Rohto Pharmaceutical Co Ltd announced full‑year earnings of ¥34.247 bn, up from ¥30.841 bn a year earlier, while revenue climbed 11.4% to ¥343.725 bn. The results signal robust domestic demand and set the stage for the company’s next product launches.
Current Clinical Trials of Alzheimer’s Drugs
The 2026 annual report on Alzheimer’s drug development lists 158 therapies across 192 active clinical trials, enrolling 54,728 participants. Over the past decade the number of trials and tested agents grew by roughly 35% and 40%, respectively, with 59 new...
Adopting Creative Chemistry to Optimize Bioprocessing Workflow
Professor Sunny Zhou of Northeastern University argues that creative chemistry can mitigate unique bioprocessing challenges of antibody‑drug conjugates (ADCs). He highlights two major vulnerabilities: light‑sensitive payloads that cause aggregation and linker cleavage by host‑cell enzymes, both of which can compromise...
Lilly Data Point to ‘Maintenance’ Strategies for GLP-1 Weight Loss
Eli Lilly presented new data showing its GLP‑1 therapies can help patients maintain weight loss after injectable treatment. In the Surmount‑Maintain trial, patients who stayed on the highest Zepbound dose lost an additional two pounds over a year, while a reduced...
AMX-883
Amphista Therapeutics announced AMX-883, an oral degrader that recruits the DCAF16 E3 ligase to eliminate the epigenetic reader BRD9. The preclinical program demonstrates selective BRD9 degradation in acute myeloid leukemia (AML) cell models, leveraging a previously identified BRD9 binder for...
Enveric Biosciences Expands IP Portfolio with New U.S. Patent Issued for Methods of Treating Psychiatric Disorders
Enveric Biosciences announced the issuance of U.S. Patent No. 12,605,361 covering its carboxylated psilocybin‑derived EVM301 series for treating psychiatric disorders. The new patent expands the company’s intellectual‑property moat by protecting method claims that build on its earlier patent (No. 11,752,130). Enveric is...
Catalent and Elpida Therapeutics Enter Strategic Partnership for Late-Phase AAV Manufacturing
Elpida Therapeutics and contract manufacturer Catalent have entered a strategic partnership to handle late‑phase manufacturing of Elpida’s lead AAV9 gene therapy for Spastic Paraplegia Type 50 (SPG50). Catalent will also receive exclusive rights to produce Elpida’s other adeno‑associated virus (AAV) programs,...
Cipla Bets on Fast-Growing Biosimilars Market, to Steadily Add Products: CEO
Cipla is accelerating its entry into the fast‑growing biosimilars space, planning to launch 1‑2 new products each year over the next five to six years. The strategy is anchored by a joint venture with contract manufacturer Kemwell Biopharma, which will...
AI Generates First Complete Models of Proteins in Motion
Scientists at EPFL have unveiled LD‑FPG, an AI‑driven generative framework that creates full‑atom, dynamic models of proteins, moving beyond static predictions like AlphaFold. The system leverages graph neural networks and latent diffusion to generate ensembles that capture side‑chain motions and...
Standard-Dose Ivermectin Superior to High Dose for Severe Scabies
A randomized, double‑blind trial published in the New England Journal of Medicine compared standard‑dose ivermectin (200 µg/kg) with a higher dose (400 µg/kg), each combined with 5 % permethrin, in 132 adults suffering from severe scabies. The standard‑dose regimen achieved an 82 % cure...
AstraZeneca Reports Positive Phase 3 Data for Eneboparatide in Chronic Hypoparathyroidism
AstraZeneca announced that its investigational PTH‑1 receptor agonist eneboparatide met the primary endpoint in the Phase 3 CALYPSO trial, with 31.1% of patients achieving normalized serum calcium and independence from active vitamin D and high‑dose calcium supplements at 24 weeks. The placebo...
How Pharma Can Adjust to Global Market Uncertainty
In the closing segment of Pharmaceutical Commerce’s interview series at Asembia AXS26, Jessica Lovett, VP of commercial strategy at Innomar Strategies, examined how pharma manufacturers are reshaping market‑access and commercialization plans amid heightened political, regulatory and reimbursement uncertainty. She illustrated the growing...
STAT+: Gene Therapy Viruses Linked to a Boy’s Tumor
A recent investigation has linked a viral vector used in a gene‑therapy trial to a tumor in a young boy, marking the first documented case of a potential therapy‑induced cancer. The tumor was identified as a sarcoma arising at the...
Trump Announces FDA Commissioner Marty Makary’s Resignation Amid Pharma and Political Pressure
President Donald Trump announced on May 12 that FDA Commissioner Marty Makary will step down after a tumultuous year marked by conflict with pharmaceutical executives, vaping interests and anti‑abortion activists. The move clears the way for acting head Kyle Diamantas...
The Changing Landscape of New Approach Methodologies
New Approach Methodologies (NAMs) have shifted from experimental curiosities to mainstream tools in pre‑clinical drug development, driven by a growing evidence base and sweeping regulatory reforms. In 2025, FDA and UK initiatives accelerated adoption, with organ‑on‑chip technologies seeing growth rates...
Molecular Grappling Hooks Improve Cancer Drug Targeting and Effectiveness
Scientists have engineered restricted interaction peptides (RIPs) that act as molecular grappling hooks, anchoring anticancer drugs to tumor cell membranes after activation by fibroblast activation protein. In mouse models, a RIP‑linked monomethyl auristatin E payload shrank tumors more effectively and with...
Millions for Preclinical Immunology: Boehringer Teams Up with Immunitas
Boehringer Ingelheim has signed a licensing deal with US‑based Immunitas Therapeutics that could be worth up to €407.5 million (approximately $445 million). The agreement grants Boehringer global rights to a preclinical antibody designed to selectively eliminate disease‑driving immune cells in chronic inflammatory...
FDA Commissioner Marty Makary Resigns, Kyle Diamantas Named Acting Chief
President Donald Trump announced the resignation of FDA Commissioner Dr. Marty Makary after a tumultuous 13‑month tenure. Kyle Diamantas, the agency’s chief for foods, will serve as acting commissioner. The change comes as Makary faced criticism from pharma CEOs, vaping...
Taiwan Renews Healthcare Innovation Partnership with U.K. Agency
Taiwan’s National Health Insurance Administration and the UK’s National Institute for Health and Care Excellence have renewed a two‑year partnership covering 2026‑2028. The agreement expands collaboration on health‑technology assessment, digital transformation, and talent development. It targets high‑cost innovations such as...
New mRNA‑nanoparticle Vaccine Boosts Cancer Potency, Durability
An innovation taking mRNA-nanoparticle cancer vaccines to the next level for potency and durability @NatureBiotech https://t.co/6wBToMGgEA
Novo Nordisk Data Shows Oral Semaglutide Delivers Significant Weight Loss in Phase 3 Trial
Novo Nordisk presented sub‑analyses of the phase 3 OASIS 4 trial at ECO2026, showing that oral semaglutide 25 mg tablets deliver substantial weight loss and functional gains for adults with obesity. Early responders—28.8% of participants—lost at least 10% of body weight by week 16...
Isomorphic Labs Raises $2.1 B Series B to Scale AI Drug‑Design Engine
Isomorphic Labs closed a $2.1 billion Series B financing round led by Thrive Capital, with participation from Alphabet, GV, Temasek, CapitalG and the UK Sovereign AI Fund. The capital will be used to scale its IsoDDE AI engine, expand hiring worldwide,...
SELLAS Life Sciences Posts $8.4M Q1 Loss, Nears Pivotal AML Trial Readout
SELLAS Life Sciences Group posted a wider first‑quarter loss of $8.4 million as it closed in on the final readout of its REGAL Phase 3 trial in acute myeloid leukemia (AML). The company said 78 of the 80 required events have occurred,...
A Plan to Make Drugs in Orbit Is Going Commercial
Varda Space Industries has secured United Therapeutics as its first commercial partner to test drug manufacturing in orbit. The collaboration will send United's pulmonary‑arterial hypertension medicines to microgravity to grow novel crystal forms that could improve stability and efficacy. Varda...
STAT+: Drug Meant to Make Gene Therapy Safer May Also Make It Less Effective
A trial by Encoded Therapeutics investigating a gene‑therapy for Dravet syndrome found that adding the immune‑suppressant sirolimus may blunt therapeutic benefit. The study enrolled 21 children, most of whom received standard steroids, while a high‑dose subgroup also received sirolimus. Researchers...
Alkermes Reports Encouraging Data From Phase III Study of Lumryz
Alkermes announced positive top‑line results from its Phase III REVITALYZ trial of extended‑release Lumryz (sodium oxybate) in adults with idiopathic hypersomnia. In a double‑blind withdrawal phase, participants who continued Lumryz maintained significantly lower Epworth Sleepiness Scale scores than those switched to...
TIL Therapies: The Impact on Cancer Research
In 2024 the FDA cleared Iovance Biotherapeutics’ TIL therapy Amtagvi (lifileucel), the first autologous, non‑CAR‑T cell treatment approved for a solid tumor—advanced melanoma. The therapy expands a patient’s own tumor‑infiltrating lymphocytes in the lab and reinfuses them, often with high‑dose...
What Success Can Look Like, Darn It
Vepdegestrant, marketed as Veppanu, became the first FDA‑approved bifunctional degrader, a new PROTAC‑type therapy that eliminates target proteins. A Phase III trial showed it works but delivered no clear efficacy advantage over the existing degrader fulvestrant. Pfizer and Arvinas have now...
ATyr Pharma Charts New Phase 3 Path for Efzofitimod After FDA Type C Meeting
aTyr Pharma announced it will submit an IND in June 2026 for a fresh Phase 3 study of efzofitimod in pulmonary sarcoidosis, prioritizing forced vital capacity as the primary endpoint. The move follows FDA Type C meeting feedback and includes...
Actuate Therapeutics Secures FDA IND Clearance for First Oral Elraglusib Tablet
Actuate Therapeutics announced FDA clearance of an Investigational New Drug application for an oral tablet of elraglusib, paving the way for a Phase 1/2 study in solid‑tumor patients. The move follows positive Phase 2 data in metastatic pancreatic cancer and...
Coya Therapeutics Secures FDA Fast Track for ALS Drug COYA 302
Coya Therapeutics announced that the U.S. Food and Drug Administration granted Fast Track status to its experimental ALS therapy, COYA 302. The designation promises accelerated review and closer agency interaction, underscoring the urgent need for new treatments in amyotrophic lateral sclerosis.
Servier's Emi-Le Secures FDA Breakthrough Therapy Designation for Rare Salivary‑Gland Cancer
Servier announced that the U.S. FDA granted breakthrough therapy designation to its investigational antibody‑drug conjugate Emi-Le for locally advanced, recurrent or metastatic adenoid cystic carcinoma. The designation accelerates the Phase 1 program and underscores the promise of B7‑H4‑directed ADCs in...