Capricor Therapeutics Inc (CAPR) Q4 2025 Earnings Call Transcript
Capricor Therapeutics reported fourth‑quarter 2025 revenue of $16 million, a modest 4% year‑over‑year increase, while gross margin rose to 86% driven by higher selling prices and manufacturing efficiencies. Net loss widened to $11.9 million as operating expenses outpaced revenue growth, but the company ended the quarter with $75.7 million in cash and a $60 million debt facility. Adoption metrics improved, with 252 active implanting centers and a 13% rise in European revenue. The firm launched the 2,500‑patient BENEFIT HF trial, which could expand its addressable market to $30 billion if successful.
Vaxart Inc (VXRT) Q4 2025 Earnings Call Transcript
Vertex Pharmaceuticals reported Q4 2025 revenue of $3.2 billion, a 10% quarterly increase, and $12 billion for the full year, up 9% year‑over‑year. Gross margin reached 85.7% and non‑GAAP net income rose 24% to $1.3 billion, underscoring strong profitability. New product...
DNA Origami Vaccine Rivals mRNA Shots While Being Easier to Store and Manufacture
Researchers at Harvard’s Wyss Institute and Dana‑Farber unveiled DoriVac, a DNA origami‑based vaccine platform that delivers antigens and adjuvants on a self‑folding nanostructure. In pre‑clinical mouse studies and a human lymph‑node‑on‑a‑chip model, DoriVac generated antibody and T‑cell responses comparable to...
Startup Vima Adds Parkinson’s to Movement Disorder Scope, Expanding Series A Round to $100M
Vima Therapeutics announced an additional $40 million raise, taking its Series A funding to $100 million. The capital will support parallel Phase 2 trials of its lead candidate VIM0423 in isolated dystonia and Parkinson’s disease, expanding the startup’s focus beyond the rare movement disorder....
Can FDA Tolerate Cancer Risk for Rare Pediatric Disease Gene Therapies?
The FDA placed a clinical hold on Regenxbio’s RGX‑111 and RGX‑121 gene‑therapy trials after a pediatric MPS I patient developed a tumor four years post‑treatment. The case marks the first documented long‑latency cancer linked to an adeno‑associated virus (AAV) vector in...
Science Spotlight: New Ways to Attack Β-Amyloid Plaques in Alzheimer’s
Two pre‑clinical studies propose active clearance of β‑amyloid as a new Alzheimer’s strategy. Researchers at Washington University engineered astrocytes with chimeric antigen receptors (CARs) that engulf plaques, while another team designed bispecific peptides that ferry amyloid into cells for lysosomal...
Hawley Unveils Bill to Ban Abortion Pill, Strip FDA Approval
Senator Josh Hawley introduced a bill to immediately withdraw the FDA's safety approval for mifepristone, the primary abortion medication. The legislation follows recent Supreme Court and Trump‑era reviews of the drug and cites a controversial conservative study alleging serious adverse...
AGENT IDE Midterm Results Still Give DCB an Edge for In-Stent Restenosis
Three‑year follow‑up of the AGENT IDE trial shows the Agent paclitaxel‑coated balloon (DCB) outperforms uncoated balloon angioplasty in treating in‑stent restenosis (ISR), with target‑lesion failure (TLF) rates of 32.7% versus 40.9% (hazard ratio 0.72). The advantage is driven mainly by...
Foghorn Therapeutics Provides Financial Update for 2025 and 2026 Strategic Outlook
Foghorn Therapeutics announced a financial update highlighting progress on its lead oncology candidate FHD-909, which remains on schedule in Phase 1 dose‑escalation for SMARCA4‑mutant NSCLC. The company also confirmed that its selective CBP and EP300 degrader programs are slated for...
Elutia Reports Fourth Quarter and Full Year 2025 Financial Results; Initiates NXT-41 Regulatory Process
Elutia Inc. reported its Q4 and full‑year 2025 results, highlighting the $88 million sale of its BioEnvelope business to Boston Scientific and a cash position of $36.4 million plus $8 million in escrow. The company submitted a 510(k) for the base biologic matrix...
Codexis Reports Fourth Quarter and Fiscal Year 2025 Financial Results
Codexis reported FY 2025 revenue of $70.4 million, a 19% increase year‑over‑year, driven largely by a $37.8 million technology‑transfer agreement with Merck. Q4 revenue jumped 81% to $38.9 million, and product gross margin improved to 64% for the year. The company ended 2025 with...
FDA Merges Adverse Event Trackers Into One Database
The FDA announced a unified adverse‑event database that will eventually incorporate all of its existing reporting systems, including the vaccine‑side‑effect tracker jointly operated with the CDC. The new platform, called the Adverse Event Monitoring System, centralizes data from drugs, biologics,...
New Chromatography Resin Developed for Secretory Antibodies
Researchers at BOKU University in Vienna have engineered a novel chromatography resin designed to capture secretory immunoglobulin A (IgA) at titers suitable for commercial manufacturing. The resin employs a reengineered bacterial surface ligand, analogous to Protein A, within a macropore...
Safer Large DNA Insertion Moves Genetic Medicine Toward Scalability
Researchers at Massachusetts General Hospital, in partnership with Full Circles Therapeutics, have introduced a circular single‑stranded DNA donor platform called INSTALL that enables kilobase‑scale gene insertion without triggering the cGAS immune sensor. The method combines a short double‑stranded DNA segment...
Simple 'Cocktail' Of Amino Acids Dramatically Boosts Power of mRNA Therapies and CRISPR Gene Editing
Researchers at Biohub identified a three‑amino‑acid cocktail—methionine, arginine and serine—that dramatically improves lipid nanoparticle (LNP) delivery of therapeutic mRNA and CRISPR components. Co‑administering the supplement boosted protein expression up to 20‑fold and raised gene‑editing rates from roughly 25% to nearly...
Biodegradable Nanoparticles Can Seek and Destroy Diseased Immune Cells
Johns Hopkins researchers have engineered a streamlined biodegradable polymeric nanoparticle that delivers mRNA to T cells, prompting them to generate CD19‑CAR receptors that target disease‑causing B cells. In mice, a single intravenous dose eliminated 95% of circulating B cells within...
VIDO – Six Years Later: How VIDO Helped Respond to the COVID-19 Pandemic
VIDO swiftly responded to COVID‑19 by designing a subunit vaccine candidate within days of the SARS‑CoV‑2 genome release, isolating the virus, and establishing animal models that enabled a Phase 1 human trial by early 2021, making it the first Canadian university...
Costco Is Slashing Fertility Drug Prices By Up To 80%
Costco announced a partnership with digital health platform Sesame and fertility network IVI RMA North America to offer members dramatically reduced prices on fertility medications, with discounts of up to 80 percent. The program guides members through a digital intake,...
FDA Drug Official Moves To Hire A Friend Who Touts Unproven Claims About Antidepressants
The FDA’s acting CDER director, Dr. Tracy Beth Hoeg, is reportedly moving to hire Dr. Adam Urato, a maternal‑fetal specialist who is petitioning the agency to add boxed warnings about alleged pregnancy risks of antidepressants. Urato’s claims rely on limited...
Biogen Phase Ib Data Positions Salanersen for SMA Treatment Sequencing
Biogen reported Phase Ib data for its antisense oligonucleotide salanersen, showing substantial neurofilament light chain reductions and new motor milestones in children with spinal muscular atrophy (SMA) who had previously received onasemnogene abeparvovec. The study of 24 participants demonstrated a roughly...
Lonza Expands Agreement to Manufacture Gene Therapy for Transfusion-Dependent Beta-Thalassemia
Lonza has broadened its agreement with Genetix Biotherapeutics to increase manufacturing capacity for ZYNTEGLO, the sole FDA‑approved gene therapy for transfusion‑dependent beta‑thalassemia. Production will continue at Lonza’s Houston, Texas, dedicated cell‑and‑gene therapy site, with provisions to scale up for future...
The FDA Approves Leucovorin for Rare Genetic Condition and Not for Autism
The U.S. Food and Drug Administration approved leucovorin, a synthetic vitamin B9, solely for cerebral folate deficiency, a rare genetic disorder. Earlier this year, President Trump and HHS Secretary Robert Kennedy Jr. promoted the drug as an autism cure, prompting a surge in...
Eli Lilly CEO David Ricks Seeks 'Exit Velocity' For Pharma's Future
Eli Lilly’s chief executive David Ricks announced a strategic push to sustain the company’s recent $1 trillion revenue milestone by accelerating AI‑driven drug discovery and manufacturing. He highlighted a new partnership with Nvidia to embed generative‑AI models across the R&D pipeline, aiming...
Eli Lilly to Invest $3bn in China in Anticipation of Orforglipron Approval
Eli Lilly announced a $3 bn, ten‑year investment to expand its Chinese supply chain for the oral GLP‑1 weight‑loss drug orforglipron. The plan adds oral solid‑dosage capacity in Beijing, expands the Suzhou injection hub, and includes a $200 m CDMO partnership with Pharmaron....
A New Antibody Approach To Preventive Treatment For HIV
Researchers have engineered an antibody‑drug conjugate that simultaneously triggers HIV’s entry‑facilitating conformational shift and binds the newly exposed epitopes. In vitro tests showed the dual‑action molecule achieved seven‑ to ten‑fold greater neutralization than the antibody alone and outperformed separate mixtures...
STAT+: FDA Approves Leucovorin for Rare Disorder without Trial Data
The FDA has granted approval for leucovorin, a folinic‑acid formulation, to treat a rare metabolic disorder despite the absence of new clinical trial data. The decision leans on decades of off‑label use and historical safety records rather than prospective studies....
Cell Rejuvenation Therapy to Hit Clinic
Life Biosciences has secured FDA IND approval for ER-100, the first partial epigenetic reprogramming therapy to enter human trials. The gene‑therapy delivers OCT‑4, SOX‑2 and KLF‑4 to retinal ganglion cells via a single intravitreal injection, aiming to reset age‑related epigenetic...
STAT+: Pharmalittle: We’re Reading About the FDA Warning Novo Nordisk, Lilly Investing in China, and More
Astellas Pharma leveraged the Trump administration’s push for lower U.S. drug prices to argue for higher reimbursement for its eye drug Izervay in Japan, winning a more generous price level. Meanwhile, a review of FDA adverse event reports found that...
FDA Adverse Event Monitoring System (AEMS) Electronic Submissions
The FDA is rolling out the Adverse Event Monitoring System (AEMS), replacing the legacy FAERS platform and consolidating reporting across drugs, biologics, devices, cosmetics, food, tobacco, and veterinary products. AEMS introduces standardized electronic submissions, AI‑driven redaction and analytics, and a...
GoodRx Partners with Viatris to Offer up to 85 Percent Savings on Established Brand Medications
GoodRx announced a partnership with Viatris to slash prices on 17 of the latter’s established brand drugs, offering up to 85% off cash prices and as little as $0‑$4 for eligible commercially insured patients. The discounts are available at more...
Boron Chemistry Breaks Protein Synthesis Barrier, May Aid Cancer Therapies
Researchers at ETH Zurich have introduced a boron‑based ligation strategy that overcomes the concentration barrier in chemical protein synthesis. By masking potassium acyltrifluoroborates (KATs) with chiral zwitterionic complexes, the team achieved efficient peptide coupling at micromolar levels, far lower than...
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...
Zinereo Pharma Set to Rollout New Probiotic Solution for Childhood Ear Infections
Zinereo Pharma is preparing to launch Otibiome, a clinically evaluated probiotic derived from *Ligilactobacillus salivarius* PS7, aimed at preventing recurrent acute otitis media (AOM) in children. A pilot study of 61 kids showed an 84% drop in AOM episodes over...
Playing Sound Waves to Cells Decreases Laryngeal Cancer Aggressiveness
An international team led by the Turku Bioscience Centre discovered that applying sound‑wave vibration to vocal‑fold cancer cells restores cellular movement and markedly reduces tumor aggressiveness. The mechanical stimulation lowered levels of the oncogenic protein YAP, both in cultured cells...
Keytruda and Padcev Could Become Cancer’s Power Couple
Oncology is moving toward combination regimens, and Merck's Keytruda paired with Pfizer/Astellas' Padcev has emerged as a leading duo. A phase 3 trial in muscle‑invasive bladder cancer showed the combo cut the risk of recurrence, progression and death roughly in half,...
Mortality Risk Similar with Tocilizumab, Rituximab in RA-ILD
An emulated target trial using the TriNetX database compared tocilizumab and rituximab in 1,194 rheumatoid arthritis‑associated interstitial lung disease (RA‑ILD) patients each over a five‑year follow‑up. The analysis found no statistically significant difference in all‑cause mortality (15.9% vs 17.7%) or...
Capricor Shares Rise as FDA Sets August Decision Date for Rejected Duchenne Therapy
Capricor Therapeutics announced that the FDA has scheduled an August 22 decision on its investigational Duchenne muscular dystrophy cell therapy, deramiocel, after lifting a prior complete response letter. The biotech resubmitted an enhanced package that includes robust Phase III HOPE‑3 data showing...
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...
UK Health Authorities Join Forces to Champion Foreign Infectious Disease Trials
UK health agencies and the Wellcome charity have launched a joint initiative to fund high‑quality infectious‑disease clinical trials in Africa, South Asia and Southeast Asia. The program, co‑led by the NIHR, the Foreign Commonwealth Development Office and Wellcome, will prioritize...
RA Capital, Forbion and Canaan Appear to Back Harbour's CTLA-4 Partner Solstice
RA Capital, Forbion, and Canaan have collectively invested in Solstice Therapeutics, the CTLA-4 antibody partner of Harbour Therapeutics. The funding round, reportedly a multi‑million dollar Series B, will support Solstice's pre‑clinical and early clinical programs. By backing Solstice, the investors...
Viagra Compound May Hold Promise for Treating Fatal Genetic Disease
Researchers have identified the erectile‑dysfunction drug sildenafil as a potential therapy for Leigh syndrome, a fatal mitochondrial disorder affecting roughly one in 40,000 births. In cell models, the compound corrected mitochondrial membrane potential and normalized gene expression, while treated mice...
Early Data Show Benefits of GLP-1s in Breast Cancer
Early data presented at the San Antonio Breast Cancer Symposium indicate that GLP‑1 receptor agonists, long used for diabetes and obesity, are linked to markedly lower breast cancer recurrence and mortality. Retrospective analyses of thousands of patients showed up to...
Idorsia and Pharmalink Sign Agreement to Distribute Quviviq
Swiss biotech Idorsia has signed an exclusive agreement with UAE‑based Pharmalink Drug Store to distribute its insomnia drug Quviviq (daridorexant) across Kuwait, Oman, Qatar, Bahrain and the United Arab Emirates. Under the deal Idorsia will receive an upfront payment, retain...
NHS to Offer Fezolinetant for Menopause‑related Hot Flushes and Night‑sweats
NICE has issued final draft guidance recommending fezolinetant 45 mg once daily for moderate to severe menopause‑related vasomotor symptoms when hormone replacement therapy is unsuitable. The recommendation enables the drug to be supplied through the NHS, providing a new non‑hormonal option...
Spinal Stimulation Above and Below Injury Restores Leg Movement and Sensory Feedback in Clinical Trial
Researchers at Brown University, Rhode Island Hospital, and VA Providence demonstrated that simultaneous electrical stimulation above and below a spinal cord lesion can restore both leg movement and spatial sensory feedback in people with complete spinal cord injuries. In a...
Kainova Reports Positive Top Line Results From Phase I EPRAD Trial
Canada‑based Kainova Therapeutics announced positive top‑line results from its Phase I EPRAD study of DT‑9081, an oral EP4 receptor antagonist, in patients with advanced, recurrent and metastatic solid tumours. The trial, conducted at four sites in Belgium and France, met all...
Intra‐Articular Injectable Hydrogel Microsphere‐Based Drug Delivery System for Osteoarthritis Treatment
Osteoarthritis incidence is climbing as populations age and obesity spreads, intensifying health and economic pressures. Conventional treatments merely decelerate disease progression and often entail systemic side effects or surgical risks. Emerging hydrogel microsphere platforms create a biomimetic joint microenvironment, offering...
MariMed Inc (MRMD) Q4 2025 Earnings Call Transcript
CorMedix reported Q4 2025 revenue of $128.6 million, propelled by DEFENCATH ($91.2 M) and the first full‑quarter contribution from the Melinta acquisition ($37.4 M). Full‑year pro forma revenue reached $401.3 million, matching prior guidance, with DEFENCATH net sales totaling $258.8 million. Adjusted EBITDA for the...
Verrica Pharmaceuticals Inc (VRCA) Q4 2025 Earnings Call Transcript
Veru Inc. reported a $23.4 million public offering that lifted cash and working capital to $33 million and $29.7 million respectively, while narrowing its quarterly net loss to $5.3 million ($0.26 per share). The company received FDA feedback confirming two regulatory pathways for its...
Viant Technology Inc (DSP) Q4 2025 Earnings Call Transcript
Vanda Pharmaceuticals reported 2025 revenue of $216.1 million, up 9% year‑over‑year, driven primarily by Fanapt’s 24% sales increase and a successful bipolar launch. The company secured FDA approval for tradipitant (Nirius) to prevent motion‑sickness vomiting, with a commercial rollout slated for...