Small molecule could slow or stop progress of Parkinson's disease and related brain disorders, not just treat symptoms

A team of researchers from NYU Abu Dhabi and the University of Denver has identified a promising small molecule that could help slow or halt the progression of serious brain diseases such as Parkinson's disease, offering new hope for treatments that go beyond managing symptoms. These diseases, which also include Lewy body dementia and multiple system atrophy, are caused by aggregation and spread of a neuronal protein, which damages brain cells over time. Currently, there are no approved treatments that can stop or slow this process.

More: 

https://medicalxpress.com/news/2026-03-small-molecule-parkinson-disease-brain.html

Small molecule could slow or stop progress of Parkinson's disease and related brain disorders, not just treat symptoms

Unicycive Therapeutics Announces Receipt of Complete Response Letter for Oxylanthanum Carbonate for the Treatment of Hyperphosphatemia in Patients with Chronic Kidney Disease on Dialysis

Unicycive Therapeutics, Inc. (“Unicycive” or the “Company”) (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease,  announced  the U.S. Food and Drug Administration (FDA)  issuing  a CRL for its New Drug Application (NDA) for OLC to treat hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis.

“We plan to immediately seek a Type A meeting with the Agency to gain alignment on the best strategy to ensure rapid resolution of the CRL,” said Shalabh Gupta, M.D., Chief Executive Officer of Unicycive. “With a second manufacturing vendor identified that has produced OLC drug product, we remain optimistic about our ability to bring this promising new treatment option to patients with CKD on dialysis who are managing hyperphosphatemia, and we plan to provide an update as soon as we have additional clarity on next steps from the FDA.”

After submitting the NDA, and as a part of the application review and routine information requests, the FDA notified Unicycive that a third-party manufacturing vendor of its main contract development and manufacturing organization (CDMO) was cited for deficiencies following a cGMP inspection. This citation is unrelated to OLC. Unicycive also notes that as part of the NDA review, the Agency has not highlighted any other technical concerns related to the submitted CMC documentation or testing of OLC itself.

As part of its overall manufacturing strategy, the Company had previously identified a back-up third-party manufacturing vendor to build redundancy into its supply chain. The second vendor has a long history of successful FDA and international regulatory inspections and has already produced OLC drug product, which could also be used to support the resolution of the CMC issues identified in the CRL.

About Oxylanthanum Carbonate (OLC)

OLC is an investigational oral phosphate binder that leverages proprietary nanoparticle technology to deliver high phosphate binding potency, reducing the number and size of pills that patients must take to treat hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis. Its potential best-in-class profile may have meaningful patient adherence benefits over currently available treatment options as it requires a lower pill burden.

Unicycive is seeking FDA approval of OLC via the 505(b)(2) regulatory pathway. The NDA submission package is based on data from three clinical studies (a Phase 1 study in healthy volunteers, a bioequivalence study in healthy volunteers, and a tolerability study of OLC in CKD patients on dialysis), multiple preclinical studies, and the chemistry, manufacturing and controls (CMC) data. OLC is protected by a strong global patent portfolio including issued patents on composition of matter with exclusivity until 2031, and with the potential for patent term extension until 2035.

About Hyperphosphatemia

Hyperphosphatemia is a serious medical condition that occurs in nearly all patients with End Stage Renal Disease (ESRD). Annually there are over 450,000 individuals in the U.S. that require medication to control their phosphate levels.1 Uncontrolled hyperphosphatemia is strongly associated with increased death and hospitalization for CKD patients on dialysis. Treatment of hyperphosphatemia is aimed at lowering serum phosphate levels via two means: (1) restricting dietary phosphorus intake; and (2) using, on a daily basis, and with each meal, oral phosphate binding drugs that facilitate fecal elimination of dietary phosphate rather than its absorption from the gastrointestinal tract into the bloodstream.

Unicycive Therapeutics Announces Receipt of Complete Response Letter for Oxylanthanum Carbonate for the Treatment of Hyperphosphatemia in Patients with Chronic Kidney Disease on Dialysis

Aldeyra Therapeutics Announces FDA Acceptance for Review of Reproxalap New Drug Application for the Treatment of Dry Eye Disease

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra)  announced   the U.S. Food and Drug Administration (FDA)   acceptance the  review of the resubmitted New Drug Application (NDA) for topical ocular reproxalap, a first-in-class investigational new drug candidate, for the treatment of the signs and symptoms of dry eye disease. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 16, 2025.

“Based on the FDA’s requirement for an additional clinical trial demonstrating the efficacy of reproxalap in treating the symptoms of dry eye disease, and per agreement with the FDA, the NDA resubmission contained a single clinical trial that achieved the primary endpoint of reducing ocular discomfort relative to the vehicle control,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra. “We look forward to a productive dialog with the FDA during the NDA review of reproxalap, which, to our knowledge, remains the only dry eye disease investigational therapy to have demonstrated acute activity in reducing ocular discomfort and redness in pivotal trials simulating the disease flares that are likely the most bothersome aspects of dry eye disease.”

About Reproxalap

Reproxalap is an investigational new drug candidate in development for the treatment of dry eye disease and allergic conjunctivitis, two of the largest markets in ophthalmology. Reproxalap is a first-in-class small-molecule modulator of RASP, which are elevated in ocular and systemic inflammatory diseases. The mechanism of action of reproxalap has been supported by the demonstration of statistically significant and clinically relevant activity in multiple physiologically distinct late-phase clinical indications. Reproxalap has been studied in more than 2,900 patients with no observed safety concerns; mild and transient instillation site irritation is the most commonly reported adverse event in clinical trials.

https://go.drugbank.com/drugs/DB16688

FDA grants speedy approval to Eli Lilly's weight-loss pill for obesity

Federal regulators on Wednesday approved Eli Lilly's new weight-loss pill, a second daily oral medication to treat obesity and other weight-related conditions.

The Food and Drug Administration granted expedited approval to orforglipron, a GLP-1 drug that works like widely used injectable medications to mimic a natural hormone that controls appetite and feelings of fullness.

The drug, which will be branded as Foundayo, is expected to begin shipping Monday. The company said people with insurance may be able to get the drug starting at $25 per month with a Lilly discount card. Prices for people paying cash will range between $149 per month to $349 per month, depending on the dose.

The new pill joins drugmaker Novo Nordisk's oral Wegovy pill, which has spurred more than 600,000 prescriptions in the United States since it was approved in December.

The FDA authorized Eli Lilly's drug as part of a new program aimed at cutting drug approval times. The agency said it reviewed the company's application in 50 days.

More;

FDA grants speedy approval to Eli Lilly's weight-loss pill for obesity

https://en.wikipedia.org/wiki/Orforglipron

Study Details | NCT07506044 | Dexmedetomidine as an Adjuvant During Dural Puncture Epidural

In continuation of my update on Dexmedetomidine

Study Overview

Brief Summary:

The study aimed to determine the effects of using varied doses of DEX on the concentration of bupivacaine injected for epidural labor analgesia, and to assess the benefits of the dural puncture epidural.

Detailed Description

The primary efficacy point is the total dose of bupivacaine administered during labor. The secondary endpoints include the VAS pain scores, number of administered additional doses, incidence of motor block, delivery type (spontaneous vaginal or cesarean), maternal satisfaction, and complications such as hypotension, fetal bradycardia, and epidural-related maternal fever.

Official Title

The Effect of Different Doses of Dexmedetomidine as an Adjuvant to Bupivacaine for Parturients Having Dural Puncture Epidural Technique to Elaborate Labor Pain.

https://en.wikipedia.org/wiki/Dexmedetomidine

Read more :

Study Details | NCT07506044 | Dexmedetomidine as an Adjuvant During Dural Puncture Epidural | ClinicalTrials.gov

Study Details | NCT07506395 | Group Psilocybin-Assisted Therapy for Post-Traumatic Stress Disorder

In continuation of my update on Psilocybin

This study is a community-informed, pragmatic, open-label, phase 1 clinical trial of group-format psilocybin-assisted therapy (GPAT) for individuals with post-traumatic stress disorder (PTSD). The primary objectives of this phase 1 study are to assess the safety and feasibility of (GPAT) for individuals with (PTSD) and to evaluate preliminary effects on PTSD severity.

Detailed Description

This study is a community-informed, pragmatic, open-label, phase 1 clinical trial of group-format psilocybin-assisted therapy (GPAT) for individuals with post-traumatic stress disorder (PTSD). The primary objectives of this phase 1 study are to assess the safety and feasibility of (GPAT) for individuals with (PTSD) and to evaluate preliminary effects on PTSD severity.

These will be assessed by the following outcome measures:

• Proportion of participants completing the study protocol
• Incidence of adverse events (AEs), serious adverse events (SAEs) and AEs of special interest using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
• Mean change in the Clinician-Administered PTSD Scale for DSM-5 (CAPS-5) and PTSD checklist for DSM-5 (PCL-5). The CAPS-5 and PCL-5 are based on the DSM-5 not the DSM-5-TR.

https://en.wikipedia.org/wiki/Psilocybin

More at : 

Study Details | NCT07506395 | Group Psilocybin-Assisted Therapy for Post-Traumatic Stress Disorder | ClinicalTrials.gov

Finerenone shows superior survival and kidney protection over spironolactone in diabetic kidney disease

In continuation of my update on Finerenone

Researchers from National Taiwan University Hospital and collaborating institutions have demonstrated that finerenone, a new-generation nonsteroidal mineralocorticoid receptor antagonist (MRA), significantly reduces the risk of death and major heart and kidney events compared with spironolactone in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D).

The study, published in Nature Communications, analyzed real-world clinical data from over 2,200 patients across global health databases, using an advanced "target trial emulation" framework to mimic randomized clinical trials.

A safer and more effective alternative

Among adults with CKD and T2D, treatment with finerenone led to:

• 69% lower all-cause mortality (adjusted hazard ratio 0.31)
• 53% lower risk of kidney failure or rapid kidney decline (MAKE)
• 26% fewer cardiovascular complications (MACE)
  compared to spironolactone.

Moreover, finerenone users experienced fewer episodes of hyperkalemia (high blood potassium), a common side effect that often limits spironolactone use.

"Finerenone appears not only safer but also more effective in protecting both the heart and kidneys," said Professor Vin-Cent Wu, senior author and nephrologist at National Taiwan University Hospital. "Our findings provide real-world confirmation that this drug may transform care for diabetic kidney disease."

Advanced analytics with real-world evidence

Using the Global Health Network, encompassing over 146 health care systems worldwide, the team applied target trial emulation—a novel data-science approach—to overcome the limitations of traditional observational studies.

This method enabled researchers to simulate the design of a randomized clinical trial using real-world data, yielding robust comparative results that align with previous landmark studies (FIDELIO-DKD and FIGARO-DKD).

"This is the first real-world head-to-head comparison of finerenone and spironolactone," noted Dr. Chung-An Wang, first author. "The results show that finerenone provides meaningful survival benefits even over a relatively short 1.3-year follow-up

Clinical implications

Both finerenone and spironolactone block the effects of the hormone aldosterone, which contributes to inflammation and fibrosis in the kidneys and heart.

However, finerenone's more selective mechanism reduces the risk of electrolyte disturbances while maintaining strong protective effects on organ health.

This study suggests that finerenone could become a preferred treatment for patients with CKD and T2D, particularly those at higher risk of cardiovascular or renal complications.

"These results could help refine international treatment guidelines and improve outcomes for millions of people living with diabetic kidney disease," said Professor Wu.

The study applied a rigorous target trial emulation framework using global real-world data from over 2,000 matched patients across 21 countries, closely mirroring a randomized clinical trial.

By employing propensity score matching to balance key clinical variables, the study minimized bias and produced consistent, statistically robust results showing finerenone's lower risks of death, kidney failure, and cardiovascular events compared with spironolactone.

Supported by strong biological rationale, transparent methodology, and public data sharing, the findings are credible, reproducible, and clinically meaningful for improving outcomes in diabetic kidney disease.

https://en.wikipedia.org/wiki/Finerenone

Chung-An Wang et al, Finerenone versus spironolactone in patients with chronic kidney disease and type 2 diabetes: a target trial emulation, Nature Communications (2025). DOI: 10.1038/s41467-025-64640-3

Finerenone shows superior survival and kidney protection over spironolactone in diabetic kidney disease

Daily coffee drinking may slow biological aging of people with major mental illness

In continuation of my update on coffee

Drinking a maximum of 3–4 cups of coffee a day may slow the "biological" aging of people with severe mental illness, by lengthening their telomeres—indicators of cellular aging—and giving them the equivalent of 5 extra biological years, compared with non-coffee drinkers, finds research published in BMJ Mental Health.

But no such effects were observed beyond this quota, which is the maximum daily intake recommended by several international health authorities, including the NHS and the US Food and Drug Administration.

Telomeres sit at the end of chromosomes and perform a role similar to the plastic tips on the end of shoelaces. While telomere shortening is a natural part of the aging process, it seems to be accelerated in those with major psychiatric disorders, such as psychosis, schizophrenia, and bipolar disorder, note the researchers.

Study details and participant information

Telomeres are sensitive to environmental factors, including, possibly, diet. And coffee, when drunk in moderation, has been associated with various health benefits, prompting the researchers to explore whether it might influence the rate at which telomeres shorten in people with major mental ill health.

They included 436 adult participants from the Norwegian Thematically Organized Psychosis (TOP) study, recruited between 2007 and 2018: 259 had schizophrenia; the rest (177) had affective disorders, including bipolar disorder and major depressive disorder with psychosis.

Participants were asked how much coffee they drank every day and were grouped into four categories: zero (44); 1–2 cups; 3–4 cups (110); and 5 or more cups. And they were asked whether they smoked, and if so, for how long they had done so.

Participants who drank 5+ cups a day were significantly older than those who drank none or 1–2 cups a day. And those with schizophrenia drank significantly more coffee than those with an affective disorder.

Coffee intake is associated with telomere length in severe mental disorders, BMJ Mental Health (2025). DOI: 10.1136/bmjment-2025-301700

Daily coffee drinking may slow biological aging of people with major mental illness

Johnson & Johnson Seeks First Icotrokinra U.S. FDA Approval Aiming to Revolutionize Treatment Paradigm for Adults and Adolescents with Plaque Psoriasis

Johnson & Johnson (NYSE: JNJ) today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking the first approval of icotrokinra, a first-in-class investigational targeted oral peptide that selectively blocks the IL-23 receptor for the treatment of adults and pediatric patients 12 years of age and older with moderate to severe plaque psoriasis (PsO). Icotrokinra is uniquely designed to block the IL-23 receptor, which underpins the inflammatory response in plaque PsO and offers potential in other IL-23-mediated diseases.1,2,3

The application included data from four pivotal Phase 3 studies conducted as part of the ICONIC clinical development program, including ICONIC-LEADa, ICONIC-TOTALb and ICONIC-ADVANCE 1 & ICONIC-ADVANCE 2c. Treatment with icotrokinra met all primary and co-primary endpoints across the development program among adults and pediatric patients 12 years of age and older with moderate-to-severe plaque PsO, demonstrating significant skin clearance and a favorable safety profile in a once-daily pill. Results from the ICONIC-ADVANCE 1 & 2 studies show icotrokinra achieved co-primary endpoints and showed superiority to deucravacitinib in moderate-to-severe plaque PsO. Across all studies, pooled safety data showed a similar proportion of patients experienced adverse events (AEs) between icotrokinra (49.1%) and placebo (51.9%) groups, with no new safety signals identified to date.4,5,6,7,8

“The rapid patient enrollment across our ICONIC clinical program underscores the unmet need for an advanced plaque psoriasis treatment that meaningfully addresses their needs and preferences,” said Liza O’Dowd, MD, Vice President, Immunodermatology and Respiratory Disease Area Lead, Johnson & Johnson Innovative Medicine. “Given the breadth and depth of our studies, along with the robust clinical results reported to date, we are confident that icotrokinra has the potential to transform how physicians and patients think about plaque psoriasis care, establishing a new standard in the treatment of this immune-mediated disease.”

Data submitted to the FDA as part of the NDA include:

• Results from the Phase 3 ICONIC-LEAD study, presented as a late-breaking abstract at the 2025 American Academy of Dermatology (AAD) Annual Meeting, that showed icotrokinra successfully met the co-primary endpoints of Investigator’s Global Assessment (IGA)d score of 0/1 (clear or almost clear skin) and Psoriasis Area and Severity Index (PASI)e 90 compared to placebo at Week 16.4
• A subgroup analysis of ICONIC-LEAD, presented at the 2025 World Congress of Pediatric Dermatology (WCPD), which demonstrated pediatric patients 12 years of age and older treated with once daily icotrokinra achieved higher rates of clear or almost clear skin at Week 16 compared to patients receiving placebo with no new safety signals identified.5
• Data from the Phase 3 ICONIC-TOTAL study, presented at the 2025 Society for Investigative Dermatology (SID) Annual Meeting, that highlighted the potential of icotrokinra in patients with difficult-to-treat scalp and genital psoriasis.6
• Results from the Phase 3 ICONIC-ADVANCE 1 & ICONIC-ADVANCE 2 studies, that further reinforced the overall efficacy profile met co-primary endpoints of IGA 0/1 and PASI 90 versus placebo at Week 16. Icotrokinra also met all key secondary endpoints at Weeks 16 and 24 that measured superiority to deucravacitinib in patients with moderate-to-severe plaque PsO.7,8 Comprehensive results are being prepared for presentation at a future medical meeting.
• Long-term data from the ICONIC development program, including at least 52-weeks of treatment for ICONIC-LEAD and ICONIC-TOTAL, and results from a randomized withdrawal analysis evaluating the durability of response, are being prepared for presentation at a future medical meeting.

Johnson & Johnson has also initiated the Phase 3 ICONIC-ASCENDf study, the first-ever head-to-head study seeking to demonstrate the superiority of an oral pill, icotrokinra, compared to an injectable biologic, ustekinumab, representing an important step forward in psoriasis research.9

• ICONIC-LEAD is a Phase 3 randomized controlled trial (RCT) evaluating the efficacy and safety of icotrokinra compared with placebo in 684 participants (icotrokinra=456; placebo=228) 12 years of age or older with moderate-to-severe plaque PsO, with the higher efficacy bar of PASI 90 and IGA score of 0/1 with at least a 2-grade improvement as co-primary endpoints. ICONIC-LEAD enrolled 66 adolescent patients.
• ICONIC-TOTAL is a Phase 3 RCT evaluating the efficacy and safety of icotrokinra compared with placebo for the treatment of plaque PsO in 311 participants (icotrokinra=208; placebo=103) with at least moderate severity affecting special areas (e.g., scalp, genital and/or hands and feet) with overall IGA score of 0 or 1 with at least a 2-grade improvement as the primary endpoint.
• ICONIC- ADVANCE 1 & 2 are Phase 3 RCTs evaluating the efficacy and safety of icotrokinra compared with placebo and deucravacitinib in participants with moderate-to-severe plaque PsO with PASI 90 and IGA score of 0/1 with at least a 2-grade improvement as co-primary endpoints.
• The IGA is a five-point scale with a severity score ranging from 0 to 4, where 0 indicates clear, 1 is minimal, 2 is mild, 3 is moderate and 4 indicates severe disease.10
• The PASI score grades the amount of surface area on each body region that is covered by psoriasis plaques and the severity of plaques for their redness, thickness and scaliness.11 PASI 90 corresponds to an improvement of >=90% in PASI score from baseline.11
• ICONIC-ASCEND is a Phase 3 RCT and the first-ever head-to-head study seeking to demonstrate the superiority of an oral pill, icotrokinra, compared to an injectable biologic, ustekinumab in moderate-to-severe plaque PsO

https://en.wikipedia.org/wiki/Icotrokinra

Johnson & Johnson Seeks First Icotrokinra U.S. FDA Approval Aiming to Revolutionize Treatment Paradigm for Adults and Adolescents with Plaque Psoriasis

ACC: Mavacamten Efficacious for Adolescents With Obstructive Hypertrophic Cardiomyopathy

In continuation of my update on Mavacamten

Mavacamten, the first-in-class cardiac myosin inhibitor approved for treatment of symptomatic adults with obstructive hypertrophic cardiomyopathy (HCM), is efficacious for adolescents with obstructive HCM, according to a study published online March 29 in the New England Journal of Medicine to coincide with the annual meeting of the American College of Cardiology, held from March 28 to 30 in New Orleans.

Joseph W. Rossano, M.D., from the Children's Hospital of Philadelphia, and colleagues conducted a randomized international study assessing the efficacy and safety of mavacamten in adolescents (aged 12 to <18 years) with obstructive HCM. After five weeks of screening, 44 participants were randomly assigned to either mavacamten (2.5 or 5 mg/day based on body weight) or placebo for 28 weeks.

The researchers found that participants receiving mavacamten had a substantial improvement in terms of the change in Valsalva left ventricular outflow tract (LVOT) gradient from baseline to week 28, with average decreases of 48.5 and 0.5 mmHg in the mavacamten and placebo groups, respectively. Significant improvements in favor of mavacamten were also seen in secondary end points, including change in resting LVOT gradient, maximal left ventricular wall thickness, peak oxygen consumption, and symptom measures such as fatigue and shortness of breath. Troponin and peptide levels decreased in those taking mavacamten and increased in those taking placebo.

"Beyond symptom relief, there's a signal that this may be favorably remodeling the heart, which could improve the natural history of the disease," Rossano said in a statement. "This suggests that it could be important to start children on this therapy when they’re young, before they've had many decades of ongoing injury to the heart from the obstruction."

https://en.wikipedia.org/wiki/Mavacamten

AAD: Significant Benefits Seen for Brepocitinib 30 mg in Dermatomyositis -

For adults with dermatomyositis, brepocitinib, an oral tyrosine kinase 2/Janus kinase 1 inhibitor, demonstrates significant benefits at a 30-mg dose compared with placebo, according to a study published online March 28 in the New England Journal of Medicine to coincide with the annual meeting of the American Academy of Dermatology, held from March 27 to 31 in Denver.

Ruth Ann Vleugels, M.D., M.P.H., from Harvard Medical School in Boston, and colleagues conducted a phase 3, double-blind, randomized trial involving adults with dermatomyositis. Participants were randomly assigned to oral brepocitinib 30 mg, brepocitinib 15 mg, or placebo once daily for 52 weeks (81, 81, and 79 participants, respectively).

The researchers found that the mean Total Improvement Scores (TIS) at week 52 were 46.5, 37.5, and 31.2, respectively (brepocitinib 30 mg versus placebo difference: 15.3). On all nine key secondary end points, brepocitinib 30 mg was superior to placebo, including Cutaneous Dermatomyositis Disease Severity Index-Activity at week 4, moderate TIS response at week 52 with ≤2.5 mg/day of prednisone equivalent, and Health Assessment Questionnaire-Disability Index at week 52. The brepocitinib 30-mg arm had an increase in serious infections (9.9 percent versus 1.3 percent in placebo), but malignancies, thromboembolic events, and cardiovascular events were more frequent with placebo. There were no deaths reported.

https://en.wikipedia.org/wiki/Brepocitinib

Arvinas Announces FDA Acceptance of the New Drug Application for Vepdegestrant for the Treatment of ESR1m, ER+/HER2- Advanced Breast Cancer

Arvinas, Inc. (Nasdaq: ARVN), today with its partner Pfizer Inc. (NYSE: PFE), announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vepdegestrant for the treatment of patients with estrogen receptor-positive (ER+)/human epidermal growth factor receptor 2-negative (HER2-), ESR1-mutated advanced or metastatic breast cancer who have previously received endocrine-based therapy. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of June 5, 2026.

“Patients often face limited treatment options after first-line treatment and vepdegestrant demonstrated improved progression-free survival in patients with ESR1-mutated ER+/HER2- advanced breast cancer,” said John Houston, Ph.D., Chairperson, Chief Executive Officer, and President at Arvinas. “With the efficacy and favorable tolerability seen in VERITAC-2, we believe vepdegestrant, if approved, has potential to be a best-in-class treatment option for patients in the second-line ESR1-mutant setting. We look forward to working alongside Pfizer and with the FDA to pursue vepdegestrant’s approval and to ensure this important treatment option is made available to patients as rapidly as possible.”

Vepdegestrant, an investigational oral PROTAC ER degrader, is being jointly developed by Arvinas and Pfizer. The NDA submission was based on data from VERITAC-2 (NCT05654623), a global, randomized Phase 3 clinical trial evaluating vepdegestrant versus fulvestrant. These data were recently presented at the American Society of Clinical Oncology (ASCO) 2025 Annual Meeting and were simultaneously published in The New England Journal of Medicine.

About Vepdegestrant

Vepdegestrant is an investigational, orally bioavailable PROteolysis TArgeting Chimera (PROTAC) estrogen receptor degrader. Vepdegestrant is being developed as a potential monotherapy for ER+/HER2- advanced or metastatic breast cancer with estrogen receptor 1 (ESR1) mutations in the second line-plus setting.

In July 2021, Arvinas announced a global collaboration with Pfizer for the co-development and co-commercialization of vepdegestrant; Arvinas and Pfizer will share worldwide development costs, commercialization expenses, and profits.

The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vepdegestrant for its use as a monotherapy in the treatment of adults with estrogen receptor–positive (ER+), human epidermal growth factor receptor 2–negative (HER2-), ESR1-mutated advanced or metastatic breast cancer previously treated with endocrine-based therapy. Vepdegestrant has also been granted Fast Track designation by the FDA, underscoring the significant unmet need in this patient population and the potential for vepdegestrant to offer a meaningful new treatment option.

About the VERITAC-2 Clinical Trial

The Phase 3 VERITAC-2 clinical trial (NCT05654623) is a global, randomized trial evaluating the efficacy and safety of vepdegestrant (ARV-471) as a monotherapy compared to fulvestrant in patients with ER+/HER2- advanced or metastatic breast cancer previously treated with a CDK4/6 inhibitor plus endocrine therapy. The trial enrolled 624 patients, 270 of whom had ESR1m positive disease, at 213 sites in 25 countries.

Patients were randomized 1:1 to receive either vepdegestrant once daily, orally on a 28-day continuous dosing schedule, or fulvestrant, administered intramuscularly on Days 1 and 15 of Cycle 1 and then on Day 1 of each 28-day cycle starting from Day 1 of Cycle 2. In the trial, 43% of patients (n=270) had ESR1 mutations detected. The primary endpoint was progression-free survival (PFS) in the ESR1-mutation and intent-to-treat populations as determined by blinded independent central review. Overall survival is the key secondary endpoint.

https://en.wikipedia.org/wiki/Vepdegestrant

Arvinas Announces FDA Acceptance of the New Drug Application for Vepdegestrant for the Treatment of ESR1m, ER+/HER2- Advanced Breast Cancer

Much-needed new drug approved for deadliest blood cancer (ziftomenib)

Biomedical research that began at the University of Virginia School of Medicine has yielded a much-needed new treatment for patients with the deadliest form of blood cancer.

The federal Food and Drug Administration has approved the drug ziftomenib for patients with recurring or treatment-resistant acute myeloid leukemia who have a mutation in the NPM1 .. gene. The new medication, taken by mouth once daily, offers a potential treatment for patients who otherwise have no good options.

The drug arose from many years of research by Jolanta Grembecka, Ph.D., and Tomasz Cierpicki, Ph.D., who began the work in 2007 as research assistant professors in UVA's Department of Molecular Physiology and Biological Physics, working closely with John Bushweller, Ph.D., their former postdoctoral mentor. In 2009, both Grembecka and Cierpicki moved to the University of Michigan, where they are professors in the Department of Pathology.

"Ziftomenib is a long-awaited and desperately needed new option for patients for whom other treatments have failed—for patients left with no hope. It's a wonderful achievement by Drs. Grembecka and Cierpicki," said Mark Esser, Ph.D., the head and chief scientific officer of UVA's Paul and Diane Manning Institute of Biotechnology.

"UVA has founded the Manning Institute specifically to advance exactly this type of important research. We are accelerating the development of new treatment and cures for the most complex and difficult diseases to benefit patients everywhere."

About acute myeloid leukemia

Acute myeloid leukemia is a particularly deadly blood cancer primarily seen in people over the age of 68. More than 22,000 Americans develop the condition each year, and more than 11,000 die, according to the American Cancer Society. Overall, the disease accounts for about 1 in 3 cases of blood cancer.

https://en.wikipedia.org/wiki/Ziftomenib

Much-needed new drug approved for deadliest blood cancer

PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia.

"We are of course disappointed by the FDA's decision to not approve vatiquinone," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for both children and adults living with Friedreich's ataxia. We plan to meet with the FDA to discuss potential steps to address the issues raised in the CRL."

The FDA stated in the CRL that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support NDA resubmission.

About Vatiquinone

Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich's ataxia. Inhibition of 15-LO helps to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately decreasing inflammation and oxidative stress and promoting neuronal survival.1,2,3 Vatiquinone has been evaluated in a number of clinical studies, many focused on pediatric patients, and has demonstrated an impact on mortality risk, and a number of neurological and neuromuscular disease symptoms.

About Friedreich's Ataxia

Friedreich's ataxia (FA) is a rare, physically debilitating, life-shortening, neuromuscular disorder that mainly affects the central nervous system and the heart.4 It is the most common hereditary ataxia (abnormal, uncoordinated movements) and is usually caused by a single genetic defect in the frataxin (FXN) gene that leads to reduced production of frataxin, a mitochondrial protein that is important for cellular metabolism and energy production.4,5 Decreased frataxin levels are associated with mitochondrial iron accumulation and increased oxidative stress, which can lead to cell death through ferroptosis.6,7,8

Symptoms include progressive loss of coordination and muscle strength leading to poor balance and coordination, difficulty speaking, swallowing, and breathing, curvature of the spine, serious heart conditions, diabetes, and hearing and vision impairment.9,10 The severity of symptoms and speed of progression varies between people and some symptoms may not be evident in all. Friedreich's ataxia is usually diagnosed in childhood or adolescence.5,11 Approximately 25,000 people have Friedreich's ataxia globally.

https://pubchem.ncbi.nlm.nih.gov/compound/Vatiquinone

PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA

FDA Accepts Shionogi’s Ensitrelvir NDA for Review as the First Oral Therapy for the Prevention of COVID-19 Following Exposure

Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter “Shionogi”) announced the U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) from Shionogi Inc., a New Jersey-based subsidiary of Shionogi, for ensitrelvir (Generic name: ensitrelvir fumaric acid, Code No.: S-217622), an investigational oral antiviral for the prevention of COVID-19 following exposure to an infected individual. The FDA has set an action date of June 16, 2026 under the Prescription Drug User Fee Act (PDUFA).

The NDA is supported by results from the global, double-blind, randomized, placebo-controlled Phase 3 study, SCORPIO-PEP, which studied ensitrelvir as post-exposure prophylaxis (PEP). If approved, ensitrelvir would be the first and only oral therapy for the prevention of COVID-19 following exposure to an infected individual.1,2

SARS-CoV-2 remains highly transmissible and up to half of people living with an infected individual may develop COVID-19.3,4 The virus is constantly evolving, with novel symptoms reported.5,6 COVID-19 continues to impact daily life and may lead to absences from school and work, may cause long COVID, and in some cases, may progress to severe disease.7-12 Even patients with mild COVID-19 may experience worsening of preexisting chronic conditions.13-17

Following exposure to COVID-19, the best way to avoid its potentially serious and long-term complications is to stop viral replication, which prevents the development of the disease.1,2 There are currently no approved antiviral therapies proven to prevent COVID-19 following exposure.1,2,18 COVID-19 vaccines are received before exposure, and they do not stop viral replication.19,20 Other COVID-19 antivirals are taken following exposure and diagnosis, after viral replication has occurred and disease is already established.21

“Shionogi has a long history of innovation in infectious disease treatment and prevention. Our dedication to this field has led to significant breakthroughs in the development of novel antimicrobials and antivirals for HIV/AIDS, influenza and COVID-19. If approved, ensitrelvir will be the first and only oral therapy to help protect people in the U.S. from COVID-19 following exposure," said Nathan McCutcheon, MBA, President and CEO, Shionogi Inc.

About ensitrelvir

Ensitrelvir is a SARS-CoV-2 main protease inhibitor created through joint research between Hokkaido University and Shionogi. SARS-CoV-2 has an enzyme called the main protease (3-CL), which is essential for the replication of the virus. Ensitrelvir suppresses the replication of SARS-CoV-2 by selectively inhibiting the main protease.

Ensitrelvir, known as Xocova® in countries where it is approved, received emergency regulatory approval in Japan in November 2022 and full approval in March 2024 for the treatment of COVID-19 based on results from SCORPIO-SR, a Phase 3 study conducted in Asia, during the Omicron-dominant phase of the pandemic.

In SCORPIO-SR, ensitrelvir showed both clinical symptomatic efficacy (symptom resolution sustained for at least 24 hours) for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in a predominantly vaccinated population of patients with mild-to-moderate SARS-CoV-2 infection, regardless of risk factors. Most adverse events were mild in severity and no deaths were seen in the study. Among the most common treatment-related adverse events were temporary decreases in high-density lipoprotein and increased blood triglycerides, as observed in previous studies. Results from this study were published in JAMA Network Open.

In 2025, Shionogi submitted two new drug applications in Japan for post-exposure prophylaxis of COVID-19 and for COVID-19 treatment in pediatric patients aged six to under 12 years. The pediatric submission is based on a multicenter, randomized, double-blind, placebo-controlled trial of ensitrelvir in mild-to-moderate COVID-19 patients aged 6 to 12 years in Japan. The study confirmed safety and tolerability and found the pharmacokinetics of ensitrelvir in this age group to be similar to that in adults.

Ensitrelvir became available in Singapore for the treatment of COVID-19 via a Special Access Route application in 2023, and it is currently under regulatory review in Taiwan for the treatment of COVID-19. Ensitrelvir is also under regulatory review with the European Medicines Agency for COVID-19 post-exposure prophylaxis and treatment.22

Ensitrelvir is an investigational drug outside of Japan and Singapore. In addition, the brand name Xocova® has not been approved for use outside of Japan and Singapore and pertains only to the approved drug in Japan and Singapore.

FDA Accepts Shionogi’s Ensitrelvir NDA for Review as the First Oral Therapy for the Prevention of COVID-19 Following Exposure