Thursday, November 5, 2026
Tea Plants' Secret Weapon Against Disease: The Power of Sakuranetin
Tea plants use sakuranetin as a natural antifungal shield against anthracnose, offering hope for greener farming Tea, one of the world's most cherished beverages, may have a powerful natural defense system hidden within its leaves
Wednesday, November 4, 2026
FDA Approves Baxfendy (baxdrostat) as the First Aldosterone Synthase Inhibitor Treatment for Adults with Hypertension
AstraZeneca’s Baxfendy (baxdrostat) has
been approved in the US as a first-in-class aldosterone synthase inhibitor
(ASI) for the treatment of hypertension in combination with other
antihypertensive medications, to lower blood pressure in adults who are not
adequately controlled.
- Approval based on BaxHTN Phase III results
showing statistically significant and clinically meaningful reduction in
systolic blood pressure in patients with uncontrolled or resistant
hypertension
- Baxfendy 2mg lowered systolic blood pressure by
15.7 mmHg (9.8 mmHg placebo- adjusted) from baseline in BaxHTN trial
There are 1.4 billion people
worldwide living with hypertension.1 In the US, approximately
50% of patients living with hypertension who are already taking multiple
antihypertensive medications still struggle with persistently elevated blood
pressure,2 which is a leading risk factor for cardiovascular
disease and premature death.3,4 Hypertension is the most
prevalent and significant modifiable cardiovascular risk factor worldwide,
accounting for more deaths and disability than any other modifiable risk.5-7
Baxfendy is a
first-in-class, highly selective and potent ASI designed to lower blood
pressure in a new way by specifically inhibiting the production of aldosterone,8 a
hormone that raises blood pressure to unhealthy levels and increases the risk
of heart and kidney problems.9-11
The approval by the US Food and
Drug Administration (FDA) was based on positive results from the BaxHTN Phase
III trial,12 with Baxfendy demonstrating statistically
significant and clinically meaningful seated systolic blood pressure reduction
at both 2mg and 1mg doses in patients with uncontrolled and resistant
hypertension on two or more medications. Baxfendy was generally well-tolerated
with no unanticipated safety findings.
Dr. Bryan Williams, Chair of
Medicine at University College London, and BaxHTN primary investigator, said:
“We have been waiting for an innovative medication like Baxfendy for
hypertension for many years. Its novel way of lowering blood pressure has the potential
to transform clinical practice by targeting a root cause of persistently
uncontrolled hypertension. In addition, the nearly double-digit
placebo-adjusted systolic blood pressure reduction achieved with Baxfendy is
exciting and clinically meaningful for clinicians and patients. Epidemiological
data indicate that a 10 mmHg decrease in systolic blood pressure is associated
with a roughly 20% lower risk of serious cardiovascular events.”
John M. Clymer, Executive
Director, National Forum for Heart Disease & Stroke Prevention, said:
“Hypertension remains a staggeringly widespread silent killer and a leading
risk factor for stroke, heart attack, kidney damage and dementia. Tens of millions
of people struggle to control their blood pressure despite lifestyle changes
and currently available treatments. Innovative, new treatments could help
millions protect their heart, kidney and brain health.”
Ruud Dobber, Executive Vice
President, BioPharmaceuticals Business Unit, AstraZeneca, said: “The approval
of Baxfendy offers a much‑needed, first-in-class innovation for people living
with persistently uncontrolled hypertension who have not responded to or
tolerated existing medicines. In the US, about 23 million patients are
uncontrolled despite being on two or more medicines for hypertension, which is
a disease that has seen little therapeutic progress for the past two decades.”
In the BaxHTN Phase III trial,13 published
in the New England Journal of Medicine,12 Baxfendy
(baxdrostat) demonstrated statistically significant and clinically meaningful
efficacy for the treatment of patients with hypertension on top of standard of
care. At week 12, the absolute reduction from baseline in mean seated SBP was
15.7 mmHg (95% confidence interval [CI], -17.6 to -13.7) and placebo-adjusted
reduction was 9.8 mmHg (95% CI, -12.6 to -7.0; p<0.001) for the 2mg dose.
For the 1mg dose, the absolute reduction from baseline was 14.5 mmHg (95% CI,
-16.5 to -12.5) and placebo-adjusted reduction was 8.7 mmHg (95% CI, -11.5 to
-5.8; p<0.001). The reduction in mean seated SBP with placebo was 5.8 mmHg
(95% CI, -7.9 to -3.8). Results were consistent across both uncontrolled and
treatment-resistant subgroups.
Tuesday, November 3, 2026
FDA Approves Differin Epiduo Acne Gel (adapalene/benzoyl peroxide) for Nonprescription Use in the Treatment of Acne
In continuation of my update on Adapalene
Galderma (SIX: GALD), the
pure-play dermatology category leader, announced the United States
(U.S.) Food and Drug Administration (FDA) approval of Differin Epiduo Acne
Gel (Adapalene 0.1% and Benzoyl Peroxide 2.5% Acne Treatment) for
over-the-counter (OTC) use in ages 12 years and older, marking a significant
Prescription-to-OTC transition in acne care.
- unique Prescription-to-OTC switch in acne care,
this approval expands access to a dermatologist-trusted,
prescription-strength treatment for millions of acne sufferers ages 12
years and older
- Backed by more than 15 years of real-world
dermatologist use and a robust clinical research program, this milestone
demonstrates the depth of science behind the Differin and Epiduo heritage
- Adapalene plus benzoyl peroxide (0.1/2.5%) was
the first FDA-approved, stable, fixed- dose prescription acne treatment to
combine of benzoyl peroxide with a retinoid, and is now available
over-the-counter
- The formulation is engineered to target multiple
causes of acne more effectively than either of its individual active
ingredients alone
This milestone represents yet
another example of Galderma’s unique Integrated Dermatology strategy,
demonstrating how proven innovations from its Therapeutic Dermatology portfolio
can further strengthen its Dermatological Skincare offerings. The Prescription‑to‑OTC
transition highlights Galderma’s scale and expertise in successfully
commercializing dermatology innovations across the full spectrum of acne care.
With the approval of Differin
Epiduo OTC, Galderma is bringing its proven acne science to a broader
population of acne sufferers through wider access to a
dermatologist-recommended, prescription-strength treatment, while further
strengthening its global leadership in acne care.
Delivering continued
innovation in acne prevention and care
Differin Epiduo is a topical acne
treatment that combines adapalene, a third-generation retinoid originally
discovered and developed by Galderma to address the stability and tolerability
challenges of earlier retinoids, with benzoyl peroxide (0.1/2.5%). This
adapalene molecule reflects Galderma’s long-standing leadership in retinoid
science, delivering an innovation-led approach that goes beyond traditional OTC
acne actives. Together, these two dermatologist-trusted ingredients
address key biological drivers of acne – clearing clogged pores, killing
acne-causing bacteria, and reducing acne inflammation – to help clear acne,
prevent future breakouts and improve skin tone, texture, and overall
appearance.1,2
The formulation is further
powered by its Simulgel™ Gel Base, which stabilizes and evenly distributes both
active ingredients while maintaining benzoyl peroxide’s efficacy and improving
overall tolerability. This enables a simple, one-pump, once-daily regimen – a
level of ease that is critical to building consistent routines and achieving
clearer skin over time.
“This approval marks an important
milestone in acne care and reflects Galderma’s longstanding leadership in acne
and retinoid science. Differin Epiduo pairs two complementary, evidence‑based
actives: adapalene, a highly stable and well‑tolerated retinoid developed by
Galderma, and benzoyl peroxide, a proven antimicrobial agent. This
retinoid-benzoyl peroxide combination aligns with dermatology guidelines, which
strongly recommend using both together as a first‑line, multimodal approach to
acne treatment. Bringing this scientifically grounded combination to the OTC
setting allows us to extend that legacy to patients across the U.S. while
preserving the clinical rigor that underpins its effectiveness.” BILL
ANDRIOPOULOS, PH.D., HEAD OF GLOBAL MEDICAL AFFAIRS, GALDERMA
Clinical data supporting the
approval demonstrated:
- Proven efficacy across multiple randomized,
controlled Phase 3 studies, with adapalene/benzoyl peroxide gel
consistently outperforming adapalene alone, benzoyl peroxide alone, and
vehicle gel across inflammatory, non-inflammatory, and total acne lesions,
as well as Investigator Global Assessment (IGA) success rates (“clear” or
“almost clear”).3-5
- Clinically meaningful improvement in lesion counts
seen as early as Week 1, with antimicrobial activity beginning on Day 1
and up to 70.3% reduction in inflammatory acne lesions at Week
12.3
- Sustained efficacy with long‑term use, maintaining
~65% total lesion reduction in a single arm study through 12 months, which
supports once‑daily, ongoing acne control.6
- Proven efficacy across diverse patient populations,
with consistent results and favorable tolerability across skin tones.
“This milestone reflects a
meaningful advancement for patients, as it expands access to a clinically
proven, dual-active acne solution without the need for a prescription. In my
practice, the combination of easy access and a simple, once-daily application
can help patients use treatment more consistently in their treatment journey
for the best possible outcomes.” HEATHER WOOLERY-LLOYD, MD, BOARD-CERTIFIED
DERMATOLOGIST, UNITED STATES
First approved as a prescription
treatment, Epiduo® (Adapalene 0.1% and Benzoyl Peroxide 2.5%)
Gel has been trusted by dermatologists for more than 15 years, with millions of
prescriptions written worldwide. Its global clinical legacy includes more than
ten Galderma studies involving over 3,200 patients, along with extensive
third-party studies, across a wide range of ages, skin tones and acne severity.
With this Prescription-to-OTC switch, Galderma continues to play a defining
role in shaping modern acne care, leveraging its own proprietary science to advance
dermatology, and expanding access to effective treatments.
Differin Epiduo will be available
over-the-counter at most major retailers, including Walmart, Ulta, Target and
Amazon, beginning in the summer of 2026. With a commitment to providing
effective solutions across consumers’ acne journey, the Differin portfolio also
includes cleansers, treatments, and moisturizers to support and provide
wholistic solutions. For more information on Differin Epiduo and other Differin
products, please visit www.differin.com and join the Differin community on
Instagram, Facebook and YouTube to stay up to date on the latest innovations.
https://en.wikipedia.org/wiki/Adapalene
Monday, November 2, 2026
FDA Grants Accelerated Approval to Hepcludex for HDV
Gilead Sciences, Inc. (Nasdaq:
GILD) announced the U.S. Food and Drug Administration (FDA) granting accelerated approval for Hepcludex (bulevirtide-gmod)
8.5 mg for the treatment of adults living with chronic hepatitis delta virus
(HDV) infection, making it the first and only approved treatment for HDV in the
United States.
- Chronic HDV is considered the most severe form of
viral hepatitis due to rapid disease progression towards liver failure and
liver-related death
- Approval introduces the first FDA-approved therapy
for this disease with limited treatment options and high unmet need
The FDA granted accelerated
approval to Hepcludex based on reductions in HDV RNA and normalization of
alanine aminotransferase (ALT), supported primarily by data from the pivotal,
controlled Phase 3 MYR301 study. At Week 48, the study demonstrated a statistically
significant improvement versus the control (delayed treatment) group in a
combined virologic and biochemical response. Improvement in disease-related
clinical outcomes has not been established. Continued approval for the approved
indication may be contingent on verification and description of clinical
benefit in a confirmatory trial.
Chronic HDV is considered the
most severe form of viral hepatitis and is associated with a markedly higher
risk of rapid disease progression, liver failure, and mortality compared with
HBV alone. In the United States, studies in general populations have estimated
that HDV affects between 2% and 4% of individuals who have chronic hepatitis B
virus (HBV), representing ~40,000-80,000 people.
“Hepatitis delta virus is
associated with rapid progression of liver disease and a high risk of serious
or even life-threatening liver-related complications,” said Dr. Ira Jacobson,
MD, Department of Medicine at NYU Grossman School of Medicine. “For patients,
an HDV diagnosis means managing two distinct viral liver diseases—hepatitis B
and hepatitis D—each contributing to disease progression, monitoring demands,
and treatment complexities. The approval of Hepcludex for chronic HDV
represents a critical advancement, introducing a long-awaited option that
begins to address a significant unmet medical need and has the potential to
meaningfully alter the course of this devastating disease for people living
with HDV in the United States.”
MYR301 (NCT03852719) evaluated
the efficacy and safety of Hepcludex in adults with chronic HDV, with treatment
administered for up to 144 weeks followed by 96 weeks of off-treatment
follow-up. Hepcludex met its primary endpoint at Week 48, with continued
treatment, demonstrated sustained efficacy and was generally well tolerated
through up to 144 weeks of on-treatment exposure.
“The approval of Hepcludex
represents a historic milestone for people living with HDV in the United
States, marking the first FDA-approved treatment for HDV,” said Dietmar Berger,
MD, PhD, Chief Medical Officer at Gilead Sciences. “This reflects years of
close engagement with the FDA and the application of rigorous science to
address a serious disease with long-standing unmet need. With Hepcludex, we now
have the opportunity to deliver a meaningful clinical advancement that has the
potential to change the trajectory of HDV for patients in the U.S.”
U.S. Access and Hepcludex
Approval Across Markets
The Gilead Support Path® Program
offers information and resources to help patients diagnosed with chronic HBV,
HDV and hepatitis C virus (HCV) and primary biliary cholangitis (PBC), as well
as healthcare professionals, understand coverage and financial options for
prescribed Gilead treatments.
Bulevirtide 2 mg is also approved
for use in the European Economic Area (EEA) and other countries globally to
treat people living with chronic HDV.
Please see below for U.S.
Indication and Important Safety Information for Hepcludex.
U.S. Indication for Hepcludex
Hepcludex (bulevirtide-gmod) 8.5
mg for injection is indicated for the treatment of chronic hepatitis delta
virus infection in adults without cirrhosis or with compensated cirrhosis.
This indication is approved under
accelerated approval based on a decrease in HDV RNA and alanine
aminotransferase (ALT) normalization. An improvement in disease-related
clinical outcomes has not been established. Continued approval for this
indication may be contingent upon verification and description of clinical
benefit in a confirmatory trial(s).
Saturday, October 31, 2026
FDA Approves Zaynich (cefepime and zidebactam) for the Treatment of Complicated Urinary Tract Infections
In continuation of my update on cefepime
FDA approved Zaynich (cefepime and zidebactam), a novel intravenous antibiotic for the treatment of adults with complicated urinary tract infections (cUTI), including pyelonephritis, caused by susceptible Gram-negative pathogens. Zaynich previously received Qualified Infectious Disease Product (QIDP) and Fast Track designations from the FDA.
- Zaynich is a multi- penicillin-binding protein targeting combination of the 4 th generation cephalosporin cefepime and zidebactam working synergistically against the most challenging multidrug-resistant Gram-negative bacteria
- In the Phase 3 ENHANCE-1 clinical trial, Zaynich demonstrated higher combined clinical cure and microbiologic response (composite endpoint) at the test of cure (TOC) visit versus meropenem, 89.0% vs 68.4% respectively and was generally well tolerated
- More than 2.8 million antimicrobial-resistant infections occur each year in the U.S., resulting in more than 35,000 deaths1
“The threat of drug-resistant infections is an escalating crisis, leaving clinicians with fewer tools to treat patients facing these aggressive pathogens. The FDA approval of Zaynich is a monumental step forward in validating a new option for these underserved populations,” said Dennis Deruelle, MD, FHM, Chief Medical Officer at Wockhardt. “This milestone underscores our commitment to addressing critical unmet needs and offers a profound sense of hope to the families we are working to serve.”
“This approval is a significant realization of our mission to provide patients with novel antibiotics that help to address one of the most urgent global health threats—antimicrobial resistance,”2 said Dr. Habil F. Khorakiwala, Founder and Chairman of Wockhardt Group. “Furthermore, Zaynich is the first New Chemical Entity fully developed and commercialized by an Indian pharmaceutical company to receive an FDA approval, representing a historic milestone not only for Wockhardt, but for the Indian pharmaceutical industry.”
Zaynich, unlike most other beta-lactam combinations, targets multiple penicillin binding proteins (PBP 1a/b, 2 and 3) simultaneously. This unique, multi-target synergy provides bactericidal activity against the most challenging drug-resistant Gram-negative bacteria for which there are currently very limited treatment options.
“Multidrug-resistant bacterial infections are a substantial burden for patients and the healthcare system, as patients with these infections typically require longer, more intensive care, and are at increased risk of lifethreatening complications,” explained Keith Kaye, MD, MPH, Professor of Medicine and Division Chief for Infectious Diseases at Rutgers Robert Wood Johnson Medical School. “There is an ongoing need for new antibiotics to combat these drug-resistant pathogens, and we are pleased that this approval means patients will soon have an exciting unique option that is urgently needed in the US and worldwide.”
cUTI is responsible for over 600,000 hospitalizations in the U.S. annually. A growing number of cUTIs are caused by antimicrobial resistant bacteria, including multidrug-resistant bacteria, a leading cause of bacteremia and associated with significant morbidity and mortality and a burden on the health care system.3
The FDA approval was based, in part, on the results from ENHANCE-1, a Phase 3, randomized, double-blind, multicenter study which evaluated the efficacy, safety and tolerability of Zaynich compared with meropenem in the treatment of hospitalized adults with cUTI or acute pyelonephritis (AP). Zaynich demonstrated efficacy at the primary endpoint, achieving a composite clinical cure and microbiological response rate of 89.0% versus 68.4% for meropenem Treatment difference 20.6% (95% CI; 12.3, 29.5). Zaynich was generally well tolerated in the phase 3 study. The study enrolled 530 patients from the U.S., Europe, LATAM, China and India, and spanned across 64 sites.
Zaynich® was approved by the Drugs Controller General of India (DCGI) on May 27, 2026. Wockhardt has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency.
Friday, October 30, 2026
FDA Approves Xocova (ensitrelvir), the First and Only Oral Option to Help Prevent COVID-19 Following Exposure
In continuation of my update on ensitrelvir
Shionogi & Co., Ltd. (Head
Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, PhD; hereinafter
“Shionogi”) announced that the U.S. Food and Drug Administration (FDA) has
approved Xocova (ensitrelvir),
an oral antiviral, for post-exposure prophylaxis (PEP) of COVID‑19 in adults
and adolescents 12 years of age and older following contact with an individual
who has COVID-19.3 This approval introduces the first and only
oral option to help prevent COVID-19 after exposure in the current therapeutic
landscape, addressing a critical gap in prevention. Xocova is a five-day oral
regimen with three tablets taken on day one and one tablet taken on days two
through five.3 The approval occurred ahead of the Prescription
Drug User Fee Act (PDUFA) action date of June 16, 2026.
- Approval is based on SCORPIO-PEP, the only Phase
3 study of an oral antiviral to meet the primary endpoint of preventing
symptomatic COVID-19 following exposure to an infected individual*
- Xocova offers a new approach to help prevent
COVID-19 by blocking viral replication during the critical window between
exposure and COVID-19 symptom onset1,2
“The FDA approval of Xocova
provides an important new approach to preventing COVID-19, which continues to
impact lives. COVID-19 can become severe and even when mild or moderate, it can
worsen or exacerbate chronic conditions or trigger new ones, including long
COVID,” said Frederick Hayden, MD, Richardson Professor Emeritus of Clinical
Virology and Professor Emeritus of Medicine, University of Virginia School of
Medicine. “Ensitrelvir inhibits viral replication, helping protect people who
have been exposed to COVID-19 from developing illness. The PEP strategy has the
potential to benefit anyone who does not want to get COVID-19. It could be
useful not only in household settings but also in other exposure circumstances,
such as outbreaks in nursing homes, chronic or acute care facilities and
following travel-related exposures.”
The approval is based on positive
results from SCORPIO-PEP, the only Phase 3 study of an oral antiviral to meet
the primary endpoint of preventing symptomatic COVID-19 following exposure to
an infected individual.* Xocova significantly reduced the risk of symptomatic
COVID-19 by 67% in uninfected individuals following exposure to an infected
individual through Day 10 compared with placebo (ensitrelvir
n=1,030; placebo n=1,011). 3,4 Overall, Xocova was
generally well tolerated, with similar rates of adverse events across groups
(15.1% in the Xocova group (n=1,190) and 15.5% in the placebo group (n=1,187)).4 The
most common adverse events (regardless of causality) occurring in greater than
or equal to 1% of the Xocova group and at a greater frequency compared to
placebo were headache, diarrhea, and cough. 3 There were
no reports of altered taste (dysgeusia) attributed to Xocova in the trial.4,5 Results
from the SCORPIO-PEP trial were published in the New England Journal of
Medicine on May 14, 2026.
“The FDA approval of Xocova is an
exciting new chapter in the Shionogi antiviral story, which includes innovative
medicines that have changed the way we manage other viruses including HIV and
influenza,” said Nathan McCutcheon, President and CEO, Shionogi Inc. “Xocova is
the first and only oral option clinically proven to help prevent symptomatic
COVID-19 after exposure among study participants regardless of vaccination
status or baseline immunity from prior infection. With Xocova, people who are
exposed to COVID-19 can act early to help protect themselves.”
The impact of this matter on the
consolidated financial results for the fiscal year ending March 2027 has
already been reflected in the earnings forecast announced on May 12, 2026.
The Ongoing Impact of
COVID-19
COVID-19 remains highly
transmissible, driven by Omicron and its subvariants, and up to 47% of people
living with an infected individual may develop COVID-19.6 The
U.S. Centers for Disease and Control estimates that between October 1, 2025 and
May 23, 2026 there were 3.8-12.4 million new cases in the U.S., resulting in
800,000 – 2.3 million outpatient visits, 120,000 – 240,000 hospitalizations and
13,000 - 42,000 deaths.7
Beyond acute infection, COVID-19
can have lasting impacts. People diagnosed with COVID-19 had increased rates of
both new and worsening neurologic, cardiovascular, respiratory, and renal
conditions during the year following infection.8-17 COVID-19
also disproportionately affects older adults with greater risk for severe
illness and death in close-community settings, such as long-term care
facilities.18
About SCORPIO-PEP
The global, double-blind,
randomized, placebo-controlled Phase 3 study, SCORPIO-PEP, assessed the safety
and efficacy of Xocova as post-exposure prophylaxis for COVID-19. The study
included 2,387 study participants aged 12 years and older with a negative local
screening test for SARS-CoV-2 infection and no symptoms at the time of
enrollment, who were exposed to a person living in their household with
symptomatic COVID-19. The primary analysis included 2,041 household contact
participants with a central laboratory-confirmed negative SARS-CoV-2 test at
baseline.
The trial was conducted from June
2023–September 2024. More than 99% of household contacts tested positive for
antibodies against SARS-CoV-2 N (nucleocapsid) or S (spike) proteins,
indicating that nearly all had evidence of previous SARS-CoV-2 infection or
vaccination, or both.
Study participants were randomly
assigned at a 1:1 ratio to receive Xocova (375 mg on day 1 and 125 mg on days
2-5) or placebo, once daily, and began treatment within 72 hours of when the
household member with COVID-19 began showing symptoms. Participants then
continued Xocova (125 mg) or placebo for five days. SCORPIO-PEP is the first
and only Phase 3 study of an oral antiviral to meet the primary endpoint of
preventing COVID-19 following exposure to an infected individual.
https://en.wikipedia.org/wiki/Ensitrelvir
Thursday, October 29, 2026
Psilocybin to target persistent concussion symptoms in Australia's first clinical trial
Monash University
researchers have launched Australia's first clinical trial to test whether the
psychedelic drug psilocybin is an effective treatment for persistent
post-concussion symptoms.
There are few effective
therapies for the debilitating long-term effects of mild traumatic brain
injury, more commonly known as concussion.
But researchers now think
psilocybin, a psychedelic compound found in some mushrooms, could be the
answer.
A recent preclinical
study from Monash University indicates that a single macrodose can reduce
the symptoms that often appear after brain injury. The research is published in
the journal Cell Reports Medicine.
The project will leverage
leading-edge capability and a track record in neuroimaging and biomarkers of
psychedelics within the Monash Trauma Group, and clinical implementation and
assessment within the Clinical Psychedelic Lab.
Why researchers are
hopeful
Lead researcher on the
trial, Professor Terence O'Brien, head of Monash University's School of
Translational Medicine, program director Alfred Brain, and deputy director of
research at Bayside Health Alfred, said the clinical trial is a huge step
forward and will provide hope for many patients.
"Given the lack of
effective treatment options for persistent and debilitating concussion
symptoms, we are excited to be studying a promising new approach," O'Brien
said.
"In addition to
alleviating symptoms, research suggests it can also target the biological
effects concussion has on the brain."
Up to 50% of
people who sustain a concussion will experience persistent post-concussion
symptoms.
These commonly include
cognitive and emotional issues, plus other debilitating symptoms such as
headache, dizziness, irritability and sleep disturbances.
How the trial will work
The
Monash-led trial will take place in Melbourne over three years, beginning in
2026.
It is
Australia's first randomized, double-blind, active placebo-controlled clinical trial investigating the efficacy of
psilocybin-assisted therapy for persistent post-concussion symptoms in humans.
It will
study the effect of the drug on people who have had persistent post-concussion
symptoms for at least six months, regardless of the cause of the original
concussion.
Professor
Sandy Shultz, from the Monash Trauma Group in the School of Translational
Medicine, said researchers expect psilocybin will reduce neuroinflammation and enhance the brain's
ability to form new neural connections.
"We
will also be looking at blood and neuroimaging biomarkers in the brain so we
can understand exactly what the drug is doing in the body and how it is helping
reduce symptoms," Shultz said.
"These
benefits could last for at least six months post-treatment, which would be a
game changer, and we will test this in the trial."
Support and broader collaboration
Dr. Josh
Allen, a research fellow in the Clinical Psychedelic Lab at Monash's School of
Clinical Sciences, said while many think this kind of trial isn't for them,
it's worth reaching out if you suffer from persistent post-concussion symptoms.
"Participants
are supported every step of the way," he said.
"This
includes preparation sessions before, guided care during the psychedelic
experience, and integration therapy afterward.
"Our
approach will promote safety and maximize benefits for all trial
participants."
Dr. Paul
Liknaitzky, head of the Clinical Psychedelic Lab, said the research will
address a major unmet need for people with persistent post-concussion symptoms.
"We're
testing a combined treatment approach that targets both the neurological and
psychological drivers of patients' symptoms and functionality," Liknaitzky
said.
The project
is a collaboration between the university's Department of Neuroscience and
Department of Psychiatry. Also part of the research team are Dr. Georgia
Symons, Dr. Jennifer Makovec Knight, Dr. Zhibin Chen and Dr. Andrew Gibbons,
all from Monash University, as well as Professor Karen Caeyenberghs from Deakin
University, Professor Joshua Byrnes from the University of Queensland,
Professor Karen Barlow from the University of Queensland, and Naomi Fuller from
Connectivity Traumatic Brain Injury Australia.
Wednesday, October 28, 2026
Baricitinib Exhibits Steroid-Sparing Effect in PMR Trial
In coninuation of my update on Baricitinib
Baricitinib had a significant glucocorticoid (GC)-sparing effect in people with newly diagnosed polymyalgia rheumatica (PMR), who participated in the phase 3 JAK-Spare trial, was according to results reported at the European Alliance of Associations for Rheumatology (EULAR) 2026 Annual Meeting.
GC-free remission at week 16 was achieved in nearly two thirds (65.2%) of the 23 people who had received the drug in the trial, as compared to 17.4% of the 23 people who had been treated with placebo (P < .01).
And when participants who had initially been treated with placebo crossed over to treatment with baricitinib, their GC-free remission rate at week 28 increased to 65.2%. The GC-free remission rate in the baricitinib arm also increased to 78.3%.
Baricitinib is a drug that is “efficacious and safe in many of our [rheumatic and musculoskeletal] diseases. We know the mechanism, we know the safety profile, and therefore, it also makes sense to also test this drug in this patient population,” Lechner-Radner added.
The JAK-Spare Study
The JAK-Spare study involved 46 people with recently diagnosed PMR who were recruited between July 2022 and May 2024 across five European centers. The mean age of the recruited participants was 65 years, just over half were men, and all were White.
Participants were randomly allocated to receive baricitinib 4 mg/d or to a placebo. A rapid GC-tapering regimen was used in both arms, which aimed to reduce the dose of GCs to zero within 11 weeks rather than the usual 52 weeks. After the initial 16-week study period, patients in the placebo group crossed over to treatment with baricitinib, while those in the baricitinib arm continued treatment. Then at 28 weeks, participants in GC-free remission were randomly assigned again either to continue baricitinib 4 mg/d through week 44 or lower their dose of baricitinib to 2 mg/d, stopping it altogether from 36 to 44 weeks.
Lechner-Radner said in an interview that GCs were given from the outset in the trial in both treatment arms because the investigators felt that it would not have been ethical to give patients newly diagnosed with PMR no treatment at all.
The time to relapse was significantly longer with baricitinib than placebo (P = .008). Participants in the baricitinib arm also took a lower median cumulative GC dose than those in the placebo group, at 736 mg vs 1065 mg at 16 weeks (P = .015) and 736 mg vs 1086 mg at 28 weeks (P = .013).
After re-randomization of the 33 patients who were in GC-free remission at 28 weeks, GC-free remission continued out to 44 weeks in 93.3% of those still taking baricitinib 4 mg/d and in 72.2% of those who tapered to 2 mg/d and then 0 mg/d (P = .186). Flare-free survival was higher among people who continued to take baricitinib 4 mg/d but was not statistically significant from those who tapered and then stopped baricitinib.
In each phase of the study, the percentages of participants with at least one adverse event were similar between the groups, at 87% in both in the first phase, 60.9% with baricitinib 4 mg/d vs 43.5% in the placebo crossover in the second phase, and 80% with baricitinib 4 mg/d vs 66.7% in those who tapered baricitinib in the third phase.
New Steroid-Sparing Option in PMR?
“With PMR, one wants to get patients off steroids as quickly as possible. Steroid therapy is quite toxic, and this is minimized by short duration of use and at a low dose,” Jonathan Kay, MD, professor of medicine and population and quantitative health sciences and the Timothy S. and Elaine L. Peterson Chair in Rheumatology at the UMass Chan Medical School in Worcester, Massachusetts, told Medscape Medical News. He was not involved in the trial.
But “if you can get someone on a medication that has less likelihood of causing toxicity than the steroid, the better that is going to be for the patient,” he added.
While these were positive data for a JAK inhibitor, Kay wondered whether such a drug would be likely to gain approval for use in this indication from regulatory authorities.
Kay pointed out that the interleukin 6 inhibitor sarilumab, which is given by subcutaneous injection every 2 weeks, is already approved to treat PMR.
Tuesday, October 27, 2026
Psilocybin linked to reduction in depression
A study of 19 patients with treatment-resistant depression treated with psilocybin in Switzerland found psilocybin was associated with a significant reduction in depression symptoms. The study is published in The Lancet Regional Health—Europe.
Psilocybin—a naturally occurring psychedelic compound found in some species of mushrooms—has demonstrated promising antidepressant effects in depression and treatment-resistant depression in controlled clinical trials. However, its effectiveness and safety in real-world treatment settings remain largely unknown.
This study analyzed the medical records of 19 patients who received one to four clinical dosing sessions of psilocybin at the Psychiatric University Hospital Zurich as treatment for treatment-resistant mild to severe depression.
On average, the clinician-reported depression rating scores decreased from approximately 31/60 before psilocybin treatment to 20/60 within 42 days after the final dosing session. The patient-reported depression scores decreased from an average of 32/63 to 23/63. Side effects were reported in 30/40 psilocybin dosing sessions, with the most common being fatigue, headache, and tearfulness, but no serious or sustained side effects were documented.
The authors say their findings suggest that results from clinical trials may translate into routine clinical practice, although outcomes appear smaller in scale. They highlight that the small sample size of this study limits generalizability and, as there was no longer-term follow-up, it is not known if the improvements in depression symptoms were maintained. They call for larger real-world studies.
Saturday, October 24, 2026
Drug duo synergy may help cystic fibrosis patients clear lung mucus, animal tests suggest
Two existing types of drugs synergize to improve the clearing of mucus from the lungs of animals with cystic fibrosis, a Stanford Medicine research team has found. The findings are published in the June 2026 issue of the Journal of Clinical Investigation. If replicated in humans, the discovery may provide a way to reduce chronic lung infections in people with cystic fibrosis.
"I really hope this discovery can help CF patients," said lead author Nam Soo Joo, Ph.D., a senior research scientist at Stanford Medicine. "If these drugs improve mucociliary clearance in people as much as they do in our animal models, we expect this to help remove pathogens from the lungs and reduce patients' airway infections."
The study's senior author is Carlos Milla, MD, a professor of pediatrics at Stanford Medicine and a pulmonologist who treats children with CF at Stanford Medicine Children's Health.
The research team showed that the drug combination—a beta adrenergic agonist, formoterol, and a low-dose cholinergic agonist, methacholine—was safe and well-tolerated in a small group of people, including individuals with CF and healthy volunteers. The drugs are already approved by the U.S. Food and Drug Administration for other uses, and the scientists are now planning a trial of the combination's effectiveness in CF patients.
The finding could be especially beneficial for the 10% to 20% of CF patients who do not respond to targeted medications for cystic fibrosis known as CFTR (cystic fibrosis transmembrane conductance regulator) modulators, Joo noted.
The synergistic medications may also help people with other respiratory conditions, such as chronic obstructive pulmonary disease or bronchiectasis, a chronic lung disease characterized by severe airway scarring, he added.
Cystic fibrosis is a relatively common genetic disease, affecting 1 in every 3,000 to 4,000 babies born in the United States. Patients have mutations in the cystic fibrosis transmembrane conductance regulator gene. In healthy people, the CFTR gene encodes protein channels that let chloride and bicarbonate ions cross cell membranes. But in CF patients, the channels malfunction, causing the lungs and digestive system to produce thick mucus. This sticky mucus harbors bacteria and makes CF patients vulnerable to chronic lung infections.
Drugs called CFTR modulators that restore the chloride channels' function have improved the lives of many CF patients by allowing their bodies to manufacture normal mucus. But up to 20% of patients' gene mutations are incompatible with CFTR modulator drugs, meaning the medications don't help.
That's why Joo's team was excited to find a different approach to the mucus problem.
A fortuitous finding
The researchers' discovery was an accident. They were studying whether various drugs could speed mucociliary clearance, in which the cilia, hairlike projections that line the passages into the lungs, beat in a coordinated way to move mucus and bits of foreign matter, such as bacteria or dust, out of the airways. The process keeps the lungs healthy.
During drug tests on tissue in a dish, the researchers used a combination of a beta adrenergic agonist and a cholinergic agonist to check that the tissues were viable. (Each of the two medication categories includes several drugs; at first, the researchers were not using drugs approved for use in people.) To the scientists' surprise, the drug combination—intended only as an experimental control—sped up mucociliary clearance.
Joo discussed this unexpected observation with study co-author Jeffrey Wine, Ph.D., an emeritus professor of psychology and of human biology.
"Jeff said, 'Do you think this may work in CF?'" Joo recalled. "I said, 'Well, probably not.'"
The combination seemed unpromising because people with CF are already known to have an abnormal response to beta adrenergic agonists. Meanwhile, cholinergic agonists administered alone cause people's airways to constrict in an asthma-like manner, which presents a safety concern.
A counterintuitive result
Even though they suspected the drug combination would fail, Joo's team tested it, first in airway tissue samples from a ferret model of CF, then in tissue and live animals including ferrets, rats, sheep, and pigs with pharmacologically induced or genetic CF.
In the new study, the researchers used formoterol and methacholine, which are a beta adrenergic agonist and a cholinergic agonist, respectively. The scientists chose these specific drugs because they are already approved by the FDA for use as inhaled medications.
Each drug caused a small increase in mucociliary clearance when given alone, but together their effects were far more pronounced. For instance, in rats with CF, the drug combination increased the animals' mucociliary clearance velocity to about 80% of that seen in healthy rats.
Follow-up experiments on tracheal tissue showed that the drugs given together increase fluid secretion, inhibit absorption of fluid, and raise the pH of the secreted fluid. These changes make the mucus less viscous and more mobile.
Furthermore, the researchers showed that when the drug combination was given to ferrets with CF that were also receiving a CFTR modulator drug, the animals could clear mucus more easily than when they received only the CFTR modulator, suggesting that the drug combination may benefit all CF patients.
In a small safety test in humans, as expected, methacholine caused airway constriction when given alone. But the two drugs in combination did not.
The results are exciting both for their implications for cystic fibrosis and for patients with other lung conditions, Joo said.
"Patients with other respiratory disorders like chronic obstructive pulmonary disease or bronchiectasis also have mucociliary clearance impairment, but compared with CF, it's mild," he said. "If we use this drug in other airway diseases, it may improve those patients as well, but we still require large-scale clinical trials to know the optimal combination and concentrations of these drugs."
