Know What's Happening in Pharma
Know What's Happening in Pharma
Eli Lilly’s oral GLP‑1 pill and triple‑agonist trial reshape obesity drug race
The FDA approved Lilly’s oral GLP‑1 weight‑loss tablet Foundayo (orforglipron), which can be taken without food or water restrictions. In a Phase 3 trial, Lilly’s experimental injectable triple‑hormone agonist retatrutide achieved a 28.7% average body‑weight reduction, outpacing Novo Nordisk’s next‑generation candidates.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma
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 six months and a marked reduction in antibiotic use. The product, already sold in Spain as Elebiotic, can be taken alongside antibiotics with a two‑hour gap. Otibiome will debut at Vitafoods Europe and will be rolled out globally thereafter.
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...
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,...
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 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 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 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 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...
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 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...
The FDA granted its first approval for a therapy targeting cerebral folate transport deficiency, an ultra‑rare neurological disorder, with Wellcovorin (leucovorin calcium) showing meaningful neurological improvements in 89% of patients. Meanwhile, over‑the‑counter naloxone prices have slipped by roughly $0.49 each...
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...
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...
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...
ICON Biotech’s 2025 survey of 163 global biotech leaders, plus 100 Chinese executives, reveals shifting dynamics as China overtakes the United States in innovative trial volume and is projected to contribute 35% of FDA approvals by 2040. Funding remains scarce,...
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...
Curatis Holding and Japan’s Neupharma have signed an exclusive licensing deal granting Neupharma rights to develop and market corticorelin (C‑PTBE‑01) for peritumoral brain edema (PTBE) in Japan. The agreement includes up to CHF 83.5 million in upfront and milestone payments and royalties...
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...
Out‑Of‑Pocket’s 2025 outlook forecasts a turning point for several health‑care segments. Obesity drugs such as GLP‑1s are expected to become cost‑effective as pricing pressure and outcomes‑based contracts expand access. AI models will split, with healthcare‑specific versions emphasizing explainability, security and...
In this episode, Daniel Levine interviews Richard Freed, CEO of Rheumagen, about the pivotal role of HLA genes in autoimmune diseases and the company’s innovative gene‑editing approach to cure them. Freed explains how a single amino‑acid change at a conserved...
The Role of Cellular Senescence and SASP in the Pathogenesis of Atherosclerosis and the Therapeutic Potential of Senolytic Strategies in Cardiovascular Diseases "Cellular senescence and SASP drive chronic inflammation and vascular dysfunction central to atherosclerosis pathogenesis, with senescent endothelial cells, VSMCs,...
Correct. There will likely be no RCTs on BPC. Unless a new variant can perform better and be patented and Rx’d… and THATs happening.
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...
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...
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...
The FDA granted approval for leucovorin calcium tablets to treat a rare genetic disorder that impairs folate transport to the brain. Unlike typical approvals, the agency relied primarily on a handful of patient case reports rather than a sponsor‑run clinical...
Sen. Ron Johnson (R‑Wis.) announced an investigation into the FDA’s rejections of rare‑disease treatments, requesting the agency’s complete response letters for drugs targeting ataxia, Sanfilippo syndrome and similar conditions. Johnson argues the FDA’s cited deficiencies are often “nitpicky,” suggesting an...
The FDA approved 18 biosimilars in 2025, spanning six therapeutic areas and marking a surge in interchangeable designations to over 20. A wave of denosumab biosimilars and first‑in‑kind interchangeable products such as Poherdy® and Omlyclo® highlighted market diversification. BPCIA litigation...
Public Citizen announced it will encourage compulsory licensing of generic drugs if Pfizer follows through on its January threat to withhold medicines from France unless the country raises drug prices. The consumer group’s stance turns a pricing dispute into a...
Researchers at the University of Toronto have engineered synthetic long non‑coding RNA (lncRNA) molecules that can dampen inflammatory responses. The study, led by PhD student Janice Pang and biologist Omar Khan, demonstrates that these engineered lncRNAs reduce cytokine release in...
Neuland Laboratories announced that the first module of its new commercial peptide manufacturing facility will be operational by summer on its 17‑acre Bonthapally campus in India. The module adds 6,370 L of solid‑phase and liquid‑phase peptide synthesis capacity, ranging from 250 L...
Lundbeck chief executive Charl van Zyl warned that Europe’s fragmented drug‑pricing regime threatens biotech sustainability and patient access. He cited recent remarks by HHS Secretary Robert F. Kennedy Jr. suggesting Europeans already pay higher prices than Americans. Van Zyl called...
The FDA has cleared Pylarify TruVu, a reformulated version of Lantheus’ PSMA‑targeting radiopharmaceutical piflufolastat F‑18. The new formulation improves stability at higher radioactive concentrations, enabling larger batch production and broader distribution. Lantheus plans a Q4 2026 commercial launch with a rolling geographic...
Johnson & Johnson has filed a Type II variation with the European Medicines Agency to add teclistamab, a bispecific T‑cell‑redirecting antibody, as a second‑line monotherapy for relapsed/refractory multiple myeloma. The Phase III MajesTEC‑9 trial, enrolling 614 patients, demonstrated a 71 % reduction in...
The FDA has expanded approval of leucovorin calcium tablets as the first therapy for cerebral folate transport deficiency linked to FOLR1 gene variants. The decision relied on published case literature, showing 87‑89% of patients achieved clinical improvement. In oncology, AstraZeneca...
Study shows nearly half of weight loss participants reduced alcohol use after starting anti-obesity medication (GLP-1 agonists, metformin, naltrexone). https://jamanetwork.com/journals/jamanetworkopen/fullarticle/2827069 https://www.gatlan.com/ @GatlanHealth
Melatonin as a Guardian of Mitochondria: Mechanisms and Therapeutic Potential in Neurodegenerative Diseases https://t.co/kp4LZEbZzN https://t.co/boWzW7fcjM
Several U.S. universities have launched two‑year medication safety fellowships that rotate participants through academic training, a major pharmaceutical company, and the FDA. Butler University offers two tracks with Regeneron and Eli Lilly, Purdue partners with AbbVie, and Rutgers collaborates with Pfizer....
Last fall the administration was talking about leucovorin as a potential treatment for large numbers of people with #autism. Today's approval suggests the evidence supports its use in a much smaller group of people, @rosebroderick_ reports. https://t.co/NlJR8KlaqP
Peptide therapeutics are booming, with more than 2,000 candidates in discovery and pre‑clinical stages and projected global sales of $91.4 bn by 2025. Companies like Orocidin faced severe synthesis and purification hurdles for their QR‑01 peptide, but Syngene’s custom low‑loading resin...
Johns Hopkins Medicine is heading a five‑year, $24 million NIH‑funded Hepatitis B and HIV Cure Consortium that brings together research teams from the United States, Brazil, India, Senegal and Uganda. The first year will enroll 450 participants co‑infected with HIV and chronic...
A multicenter retrospective study of 17 Chinese patients with juvenile myasthenia gravis found that weekly efgartigimod 10 mg/kg for four weeks produced rapid and substantial clinical improvement. Clinically meaningful improvement was observed in 70.6% of patients by week 1 and 91.7% by...
BioNTech announced it will create an independent biotech company dedicated to next‑generation mRNA technologies. The spin‑out will be led by co‑founders Ugur Sahin and Özlem Türeci, who will assume management by the end of 2026. BioNTech will contribute core mRNA assets in...
CSL‑Behring broke ground on a $1.5 billion expansion of its Kankakee, Illinois manufacturing complex, slated to be operational by 2031. The project will add at least 300 pharmaceutical positions and roughly 800 construction jobs, with the state offering more than $200 million...
BioNTech founders to step down and helm new mRNA startup https://t.co/yjvsVRQH2D by @Lilah_Alvarado $BNTX - 20%
Xenon hits a ‘home run’ in epilepsy; FDA restarts review of Duchenne cell therapy https://t.co/l40xYeW2Xh $XENE $CAPR + 15% $REGN $PFE $ABBV $GSK #biotech
Agomab Therapeutics NV closed its Nasdaq IPO, raising roughly $200 million by selling 12.5 million American Depositary Shares at $16 each. The capital will fuel its fibrosis‑focused pipeline, beginning with ontunisertib, an oral TGFβ‑ALK5 inhibitor for fibrostenosing Crohn’s disease that achieved its...
$BBIO $ALNY moving higher on apparently good news re: Pfizer's tafamidis patent defense. If you have access to Umer Raffat's research, he has all the details.
🚨 @NPR spotlights our new JAMA Network Open study: One psilocybin dose + therapy gives 6X better odds of quitting smoking vs. nicotine patch + therapy. Game-changer for tobacco smoking addiction?
Predicting Alzheimer's disease up to 25 years in advance of any symptoms with the p-tau217 biomarker blood test, among 2,766 women mean age 70 https://t.co/aOActAUN27 https://t.co/Bxl2YZxnlf
