Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech financings
Spanish biotech Ona announced a $86.6 million Series B round, ranking among the largest venture financings 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 funds will expand Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical pipeline.
Emergent BioSolutions Inc (EBS) Q4 2025 Earnings Call Transcript
Emergent BioSolutions reported a successful completion of its stabilization phase, cutting net debt by $156 million and improving leverage to 3.3x adjusted EBITDA. Adjusted EBITDA surged to $183 million, a $205 million swing from the prior year, while operating cash flow rose to $59 million. The company highlighted strong NARCAN nasal spray sales—22 million doses—and secured $550 million in medical countermeasure contracts. For 2025, Emergent projects revenue of $750‑$850 million with adjusted EBITDA between $150 million and $200 million, emphasizing margin expansion and continued growth in its core product lines.
Syndax Pharmaceuticals Inc (SNDX) Q4 2025 Earnings Call Transcript
Ascendis (SNDX) reported €720 million total 2025 revenue, driven by €477 million from Yorvipath and €206 million from Skytrofa. Q4 operating profit hit €10 million and cash flow reached €73 million, leaving €616 million in cash at year‑end. Management guided 2026 operating cash flow of roughly...
This Compound Enhances Long-Term Memory of Mice — but only in Females
Researchers injected acetate, a common metabolic by‑product, into mice and tested long‑term memory using spatial and object‑recognition tasks. Female mice that received acetate showed significantly better performance 24 hours later, while male mice displayed little to no improvement. The memory...
Amphastar Pharmaceuticals Inc (AMPH) Q4 2025 Earnings Call Transcript
Amphastar Pharmaceuticals reported full‑year 2025 net revenues of $719.9 million, a modest 2% decline, while its flagship product Baqsimi grew 12% to $185.4 million. The company secured FDA approval for ipratropium bromide HFA (AMP‑007), gaining 180‑day generic exclusivity, and announced a major...
Akebia Therapeutics Inc (AKBA) Q4 2025 Earnings Call Transcript
Akebia Therapeutics reported 2025 total revenue of $236.2 million, a 47% increase driven by the commercial launch of Vafseo and higher Auryxia sales. Net product revenue reached $227 million, with Vafseo contributing $45.8 million and gaining prescribing access for 290,000 dialysis patients. The...
Synaptic Correlates of Benzodiazepine Tolerance
Recent pre‑clinical work has mapped the synaptic changes that underlie benzodiazepine tolerance. Chronic exposure uncouples GABA(A) receptors from their benzodiazepine binding site, down‑regulates α1‑subunit mRNA, and reshapes receptor trafficking through auxiliary proteins such as Shisa7. These alterations weaken inhibitory signalling,...
Anika Therapeutics Inc (ANIK) Q4 2025 Earnings Call Transcript
Anika Therapeutics reported total revenue of $27.8 million for the quarter, a 6% year‑over‑year decline driven primarily by lower pricing in its OEM osteoarthritis pain‑management channel. Commercial channel revenue rose 22% to $12 million, powered by a 25% surge in...
Ligand Pharmaceuticals Inc (LGND) Q4 2025 Earnings Call Transcript
Ligand Pharmaceuticals reported a breakout 2025, delivering $268 million total GAAP revenue and $240 million core revenue, driven by a 48% surge in royalty income. Adjusted earnings per share rose 42% to $8.13, surpassing guidance by more than 30%, while cash and...
A Nuclei-Specific Fronto-Amygdala Pathway and Its Neurotransmitter Receptor Distribution: Implications for Antidepressant Selection
Recent neuroimaging and tract‑tracing studies have identified a nuclei‑specific fronto‑amygdala pathway that links distinct amygdala subregions with ventromedial prefrontal cortex. The work maps serotonin, norepinephrine and dopamine receptor densities across these nuclei, revealing divergent pharmacological profiles for SSRIs and SNRIs....
Organogenesis Holdings Inc (ORGO) Q4 2025 Earnings Call Transcript
Organogenesis Holdings posted a record fourth‑quarter 2025, delivering $225.1 million in net product revenue, a 78% year‑over‑year increase driven largely by an 83% surge in advanced wound‑care sales. Gross profit rose to 78% of revenue and GAAP operating income jumped 519%...
Relay Therapeutics Inc (RLAY) Q4 2025 Earnings Call Transcript
Heron Therapeutics reported full‑year 2025 net product sales of $155 million and adjusted EBITDA of $14.7 million, surpassing its guidance. Acute Care drove growth, with ZYNRELEF revenue up 48% YoY and the franchise expanding 57% overall, helped by the CrossLink IGNITE incentive program,...
Radiopharmaceutical Targeted Toward Treatment-Resistant Cancer Put on FDA's Fast Track
Aktis Oncology announced that its investigational radiopharmaceutical AKY-1189 has earned FDA Fast Track designation. The drug targets Nectin‑4, present in 80‑90 % of urothelial cancers, and delivers the alpha‑emitter actinium‑225 directly to tumors. AKY-1189 is in a Phase 1b trial covering urothelial,...
11th Annual Rare Disease Day | Advancing a Divalent siRNA for Prion Disease: An Investigator-Initiated Program
Rare Disease Day marked its 11th anniversary, highlighting the stark disparity between the 8,000 known rare‑disease genes and the under 500 approved therapies. Hosted by the Broad Institute’s Ladders to Cures Accelerator and the Termeer Institute, the event featured leading...
An Endangered Natural Pharmacy Hidden in Coral: Hundreds of Reef-Dwelling Microbes Reveal Untapped Potential
A new Nature study led by ETH Zurich researchers sequenced the genomes of 645 bacteria and archaea from over 800 coral samples, revealing that more than 99% of these reef‑dwelling microbes were previously unknown. The analysis showed that each coral...
CAB+RPV LA Is Versatile, Preferred in Treatment-Naive Patients
Long‑acting cabotegravir/rilpivirine (CAB+RPV LA) demonstrated strong virologic control and high patient preference in both treatment‑naïve and treatment‑experienced cohorts presented at CROI 2026. In the VOLITION trial, 85% of ART‑naïve adults switched early from DTG/3TC to a q2‑month injectable, achieving 95% overall suppression...
Iron Nanoparticle Eliminates Tuberculosis in Mice and May Pave the Way for New Treatments
Brazilian researchers have shown that an iron‑based compound, ferroin, encapsulated in lipid nanoparticles, completely eradicated Mycobacterium tuberculosis from mouse lungs after a 30‑day course. The formulation, LNP@FEP, stabilizes the drug, enhances the activity of existing antibiotics, and targets bacterial cell‑wall synthesis....
Leading Women CEOs Across Pharma and Biotech: A 2026 Snapshot
2026’s leading women CEOs in pharma and biotech are steering some of the industry’s biggest strategic moves, from multi‑billion‑dollar investments to landmark FDA approvals. The list includes Julie Kim’s upcoming Takeda leadership, Emma Walmsley’s transition at GSK, Reshma Kewalramani’s growth...
Withdrawn | Non-Malignant Hematological, Neurological, and Other Disorder Indications Accelerated Approvals
The FDA has removed several accelerated approvals for non‑malignant hematological, neurological, and other disorder indications after post‑marketing requirements were withdrawn. These withdrawn indications are not reflected in official listings until a Federal Register notice or label update is issued. The...
Dotinurad (FYU-981)
Dotinurad (FYU‑981), marketed as Urece®, is a URAT1 inhibitor approved for gout and hyperuricemia in Japan and China. The drug was chemically refined from the older uricosuric benzbromarone to retain potency while eliminating rare hepatotoxic events. Crystalys Therapeutics is now...
Ongoing | Non-Malignant Hematological, Neurological, and Other Disorder Indications Accelerated Approvals
The FDA’s ongoing accelerated‑approval list details 30+ drugs for non‑malignant hematologic, neurologic and other rare disorders that remain contingent on post‑marketing requirements (PMRs). Each entry cites the indication, approval date, specific confirmatory trial design, and a projected completion date ranging...
Accelerated Approval Program
The FDA’s Accelerated Approval Program permits earlier market entry for drugs treating serious conditions by relying on surrogate endpoints that predict clinical benefit. Companies must later complete confirmatory trials; positive results convert the approval to traditional status, while negative outcomes...
ADMET Predictions Get AI Boost, Federated Data Network Unites Pharma
Apheris has launched the ADMET Network, a federated learning platform that lets pharmaceutical companies jointly train ADMET prediction models without exposing raw data. Five founding members—Lundbeck, Orion Pharma, Recursion, Servier and an undisclosed partner—have each contributed roughly 80% of their...
Psychedelic Stocks Overhyped as Nominee Warns Caution
Psychedelic tape still trades on vibes > policy. Surgeon General nominee Casey Means says she “would not recommend” psilocybin to the American people today. Yes, she nods to “exciting work” in PTSD and veterans. But that is a far cry from...
Rare Disease Day Insight: Epilepsy Care Gaps in Pregnancy with UCB’s Andrea Wilkinson — Episode 244
The Xtalks Life Science Podcast’s Episode 244 spotlights a persistent gap in epilepsy care for pregnant women, noting that fewer than 5% of clinical trials include this population. Andrea Wilkinson, UCB’s Global Head of Patient Engagement & Advocacy, discusses new...
STAT+: A Rare Disease Drug Was Approvable, Then It Wasn’t. Inside a Surprise Rejection by the FDA
An experimental cell therapy for a rare post‑transplant blood cancer, developed by Atara Biotherapeutics and Pierre Fabre, was initially deemed approvable by FDA reviewers but was abruptly rejected in February 2026 over alleged clinical data deficiencies. The disease affects roughly...
Pharmaceutical Executive Daily: FDA Approves Braftovi
The FDA has approved Braftovi for patients with BRAF V600E‑mutant metastatic colorectal cancer, expanding targeted options in a genetically defined subset. The clearance underscores the momentum of biomarker‑driven oncology and combination‑therapy strategies. In parallel, Novo Nordisk announced a $2 billion partnership with Vivtex...
AI‑Engineered AAV Capsid Makes Gene Delivery Programmable
One company is unlocking gene delivery to overcome the challenges that held back the promise of genetic medicine. If you want to learn more, they’ll be hosting an exclusive fireside chat at 9am Thursday, May 7th at SynBioBeta. Last year at...
GLP‑1 Drugs Plus Healthy Habits Cut Heart Events
The combination of GLP-1 drugs (such as Ozempic) and healthy lifestyle factors was associated with less major adverse cardiovascular events in ~100,000 people with T2D @TheLancetEndo https://t.co/phNzEBy7zY https://t.co/DDVi8uedRP
Menopausal Hormone Therapy Does Not Increase Mortality
A Danish register study of over 800,000 women examined the long‑term safety of menopausal hormone therapy (MHT). About 12% of participants used MHT for a median of 1.7 years, and the analysis found no association between MHT use and all‑cause...
FDA Rejects Experimental Rare Blood Cancer Therapy
An experimental therapy for a rare blood cancer was on the path toward approval by the Food and Drug Administration last year — with internal reviewers recommending it be cleared — before the agency rejected the drug last month, @adamfeuerstein...
FDA Rejects Europe‑approved Drug: What Shifted?
Interesting read: the thread and the story. A drug that was approved in Europe in 2022, which looked like it was on track for #FDA approval, instead is rejected. What changed at FDA? 🤔
Bridging the Translation Gap for Regenerative Tissues
Muvon Therapeutics, a clinical‑stage company developing regenerative muscle treatments, is confronting the translation gap between academic discovery and commercial manufacturing. The firm highlights three core hurdles: co‑developing evolving regulatory frameworks, automating novel manufacturing processes, and recruiting personnel with GMP expertise....
ACIP Meeting Notice Raises Questions on Vaccine Injury Role
The Federal Register notice for the (now) March #ACIP meeting is now up & it's ... puzzling. Possible vote on Covid vaccine injury? ACIP can recommend changes to the way a vaccine is used, for safety reasons. But vaccine injuries aren't...
AI Should Boost Human Response Rates, Not Invent New Drugs
$LTRN is a live experiment in our thesis: AI doesn’t need to invent new drugs. It needs to improve probability of success in humans. If RADR consistently enriches responders, that’s durable value. If not, platform premium disappears. Listen to the podcast here: https://t.co/ZAg5btpcIK
Samsung Joins CEPI Vaccine Network to Prepare for Next Pandemic
Samsung Biologics has joined the Coalition for Epidemic Preparedness Innovations (CEPI) Vaccine Manufacturing Facility Network, committing to develop a ready‑to‑activate recombinant protein vaccine platform. Backed by a $20 million budget, Samsung will pre‑agree manufacturing processes, run simulated outbreak drills, and prepare...
Dual BCMA‑CD19 Targeting Shows Promise in ASyS
interesting and different efficacy outcomes in ASyS and SSc autoimmune patients treated with blinatumomab (CD3/CD19) and teclistamab (CD3/BCMA), respectively, + Rituximab maintenance. Potential for targeting both BCMA and CD19, especially in ASys. https://t.co/Dyq7h9EeGo
United Unveils Newly Partnered Soft Mist Inhaler; Mannkind Sinks
United Therapeutics announced a partnership to launch a soft‑mist inhaler, targeting more efficient pulmonary drug delivery for its pipeline. The collaboration marks a strategic pivot away from traditional propellant‑based devices and positions United in the growing soft‑mist market. Investors reacted...
Medicines and Healthcare Products Regulatory Agency Confiscates 2,000 Doses of Illegal Weight-Loss Drugs: Report
Britain's Medicines and Healthcare products Regulatory Agency (MHRA) seized nearly 2,000 doses of illegal weight‑loss drugs in raids on sites in Lincolnshire and Nottinghamshire, marking its second major operation in four months. The confiscated products were labelled as containing tirzepatide...
Alkermes’ Richard Pops to Step Down After Three-Decade Run as CEO
Richard Pops will step down as Alkermes CEO on July 31 after a 32‑year tenure, remaining as board chair. COO Blair Jackson will succeed him and join the board during the transition. Under Pops, Alkermes shifted from drug‑delivery to commercial...
Stem Cell Therapy Boosts Frailty Walk Test Performance
A randomized trial of a stem cell therapy vs placebo for aging frailty showed durable and dose-dependent improvement in the 6-minute walk test @CellStemCell https://t.co/2jOYuIgUrY https://t.co/04mEV7OdLo
Online Communities Bridge
Online Patient Communities for the E-Patient “Betwixt & Between” a New Pt & an Expert [Aug 29, 2013] @mtmdphd #ASCOConnection https://t.co/IxinXTK715 #mmsm
New Insights Into Hypertension and MACE Reduction in HIV: Steven Grinspoon, MD
A recent analysis of the REPRIEVE randomized trial shows that pitavastatin reduces incident hypertension by 17 % and cuts major adverse cardiovascular events (MACE) by 36 % in adults living with HIV. The findings extend statins’ benefit beyond LDL‑cholesterol lowering, highlighting a...
Identifying Key Barriers to Boost Myeloma Trial Enrollment
Identification of Significant Barriers to Accrual (BtA) to @theNCI Sponsored Multiple Myeloma – Clinical Trials (MM-CT): A Step towards Improving Accrual to #ClinicalTrials - @mweissmdphd et al. #ASH12 Abstract 3165 https://t.co/i4zO7ktqHJ #mmsm #caxtx #ctsm
Quadruplet Therapy Effective without Early Transplant in NDMM
#EAOnc EQUATE EAA181 Effective Quadruplet Utilization After Tx Evaluation: Ph3 RCT NDMM Not Intended for Early ASCT [Activated: 10/27/20] PI= @myelomaMD https://t.co/qD26HMx3J8 #NCT04566328 #mmMRD #mmsm @eaonc @mweissmdphd @VincentRK @LynneWagnerPhD Wei Snyder Kostakoglu @mtmdphd
Learning and Education to ADvance and Empower Rare Disease Drug Developers (LEADER 3D)
The FDA’s Center for Drug Evaluation and Research has launched the Learning and Education to ADvance and Empower Rare Disease Drug Developers (LEADER 3D) initiative under its Accelerating Rare disease Cures (ARC) program. LEADER 3D provides a suite of educational videos, downloadable...
Phase 3 Trial Tests Daratumumab Regimens for New AL Amylo
.@SWOG S2213 Ph3 RCT Dara-VC Induction Followed by ASCT or Dara-VCD Consolidation & Daratumumab Maintenance in Pts w/ Newly Diagnosed AL Amyloidosis [Activated: 12/1/23] https://t.co/OizUfJCc2c #mmsm #bmtsm https://t.co/uikUnnJd3n
New Guidelines Define Prior Therapy Lines in Multiple Myeloma
Guidelines for determination of the number of prior lines of therapy in multiple myeloma [Aug 13, 2015] @VincentRK , Richardson, San Miguel @BloodJournal https://t.co/Tdmr2gTUi6 #mmsm #ctsm #ClinicalTrials https://t.co/ZOobout75Q
Gibson Oncology and NIH Launch Phase 2 Trials of LMP744 Targeting First-Time Recurrent Glioblastoma
Gibson Oncology has launched a Phase 2 trial of its dual‑action drug LMP744 in patients with first‑time recurrent glioblastoma. The compound simultaneously inhibits topoisomerase 1 and down‑regulates the cMYC oncogene, aiming to disrupt tumor proliferation. The study will enroll about 40 patients,...
PI‑Rd Triplets Show Superior Efficacy And
Comparative Effectiveness and Safety of PI-Rd Triplets in Relapsed/Refractory Multiple Myeloma: INSIGHT-MM Data Analysis [Jan 13, 2026] @NoemiPuigM et al. European Journal of Haematology https://t.co/Vf7USecLlR #NCT02761187 #mmsm #openaccess https://t.co/C5Ya6TTQfo
Combining EMR and Multi‑omics Sharpens Biological Age Predictions
Assessment of biological age integrating electronic medical records and multi-omics increases accuracy for predicting outcomes @NatureAging https://t.co/7y60SSPTQf https://t.co/u02OlWMdQz