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Navigating the Job Interview: How to Answer the Question, “Why Are You Changing Jobs?”

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Navigating the Job Interview: How to Answer the Question, “Why Are You Changing Jobs?”

Job interviews can be nerve-wracking, but one question that you can almost guarantee will come up is, “Why are you changing jobs?” While it may seem like a straightforward query, crafting a thoughtful and convincing response can significantly impact the outcome of your interview. In this blog post, we’ll explore how to answer this common interview question effectively.

1. Emphasize Career Growth and Learning Opportunities

One of the most positive reasons for changing jobs is to seek new challenges and opportunities for professional development. When an interviewer asks about your reasons for changing jobs, highlighting your desire for growth can be a compelling response.

Here’s a sample answer:

“I’m seeking a new opportunity that will allow me to further develop my skills and take on more challenging responsibilities. While my current job has provided a strong foundation, I believe that a change will expose me to a wider range of experiences and help me grow both personally and professionally. I’m excited about the prospect of learning new things and contributing my expertise to a new team.”

This response not only demonstrates your commitment to self-improvement but also shows that you are proactive in shaping your career.

2. Address Issues Respectfully and Positively

Sometimes, the reasons for changing jobs are less about seeking opportunities and more about addressing challenges or issues in your current position. Whether it’s a toxic work environment, lack of growth prospects, or a company’s values not aligning with yours, it’s crucial to address these concerns in a way that reflects positively on you.

For example:

“I’ve had a great experience with my current employer, but I’ve reached a point where I believe my skills and values could be better utilized elsewhere. I’m looking for a work environment that aligns more closely with my personal values and offers a stronger platform for my professional growth. I believe this change is a natural progression in my career, and I’m excited about the possibilities it presents.”

This response acknowledges the positive aspects of your current job while emphasizing your desire for a better fit elsewhere.

3. Showcase Alignment with the Company’s Values and Goals

Employers appreciate candidates who have thoroughly researched their organization and can demonstrate alignment with the company’s mission, values, and goals. When you’re asked why you’re changing jobs, take the opportunity to show that you’re not just looking for any job but one that aligns with your aspirations and the company’s objectives.

Here’s how you can do it:

“I’ve been impressed by [Company’s Name]’s commitment to innovation and its dedication to making a positive impact in the industry. I’m excited about the opportunity to contribute my skills and experiences to a team that shares my values and vision. This role seems like the perfect fit for my career goals, and I believe it’s a step forward in the right direction for both of us.”

This response illustrates that you’ve done your homework and genuinely believe in the company’s mission, making you a more attractive candidate in the eyes of the interviewer.

In conclusion, the question, “Why are you changing jobs?” is an opportunity to showcase your professionalism, commitment to growth, and alignment with the organization’s values. Crafting a well-thought-out response that focuses on these aspects can help you make a strong impression during your job interview and increase your chances of landing the position you desire.

J&J announced Icotrokinra results show 75% of adolescents with plaque psoriasis achieved completely clear skin

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J&J announced Icotrokinra results show 75% of adolescents with plaque psoriasis achieved completely clear skin

April 10, 2025: “Johnson & Johnson announced new icotrokinra (JNJ-2113) data from a subgroup analysis of ICONIC-LEAD, the first ever Phase 3 registrational study in moderate-to-severe plaque psoriasis (PsO) to assess efficacy and safety of a systemic therapy in adolescents and adults simultaneously.

These data, presented at the 2025 World Congress of Pediatric Dermatology (WCPD) Annual Meeting, show adolescents 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.

Icotrokinra is a first-in-class investigational targeted oral peptide that selectively blocks the IL-23 receptor and is being studied in adults and adolescents 12 years of age and older with moderate-to-severe plaque PsO.

In the study, 84.1% of adolescent patients treated with once daily icotrokinra achieved an Investigator’s Global Assessment (IGA) score of 0/1 (clear or almost clear skin) and 70.5% achieved a Psoriasis Area and Severity Index (PASI) 90 response, compared to 27.3% and 13.6% receiving placebo, respectively, at Week 16.

Response rates continued to improve through Week 24 where 86.4% of adolescents achieved IGA 0/1 (clear or almost clear skin) and 88.6% achieved PASI 90. Further, at Week 24, 75% of adolescents achieved IGA 0 (completely clear skin) and 63.6% achieved PASI 100.

“Data from the Phase 3 ICONIC LEAD subgroup analysis demonstrate impressive efficacy rates, showing the promise of this novel therapeutic option in the treatment of adolescents with moderate-to-severe plaque psoriasis who’ve often not yet received an advanced therapy,” said Lawrence Eichenfield, M.D., Chief of Pediatric and Adolescent Dermatology at Rady Children‘s Hospital-San Diego, and Professor of Pediatrics and Medicine (Dermatology), at the University of California, San Diego (UCSD) School of Medicine, ICONIC-LEAD presenter.

“Young patients with plaque psoriasis face unique challenges due to the visible and uncomfortable nature of the disease, making effective treatment options that align with their needs and preferences all the more important.”

Icotrokinra demonstrated a favorable safety profile. At Week 16, 50% of adolescents treated with icotrokinra experienced ≥1 adverse event (AE), compared to 73% of adolescents receiving placebo, with no new safety signals identified. 

“Adolescents living with moderate to severe plaque psoriasis shouldn’t have to wait for effective treatments options that have the potential to deliver completely clear skin, which is the driving force for studying this younger population as part of the pivotal ICONIC program,” said Liza O’Dowd, Vice President, Immunodermatology Disease Area Lead, Johnson & Johnson Innovative Medicine.

“These data underscore the promise of next-generation therapies and the potential for icotrokinra to offer adolescents with moderate-to-severe plaque psoriasis the unique combination of a favorable safety profile and complete skin clearance in a once-daily pill.”

Icotrokinra results show 75% of adolescents with plaque psoriasis achieved completely clear skin and demonstrate favorable safety profile in a once daily pill

Sanofi’s Tolebrutinib phase 3 data published in NEJM demonstrate benefit on disability progression in multiple sclerosis

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Sanofi’s Tolebrutinib phase 3 data published in NEJM demonstrate benefit on disability progression in multiple sclerosis

April 8, 2025: “The New England Journal of Medicine (NEJM) published positive results from the HERCULES phase 3 study demonstrating that tolebrutinib delayed disability progression in people with non-relapsing secondary progressive multiple sclerosis (nrSPMS), where there are currently no treatment options approved.

These findings further support the differentiated mechanism of oral, brain-penetrant tolebrutinib, targeting disability progression independent of relapse activity.

These results were first presented at the ECTRIMS conference on September 20, 2024 in Copenhagen, Denmark and further analyses were also presented today during the Clinical Trials Plenary Session at the 2025 Annual Meeting for the American Academy of Neurology (AAN) in San Diego, California.

Robert Fox, MD
Vice Chair of Research at Cleveland Clinic’s Neurological Institute, Cleveland, Ohio, US, and chair of the HERCULES global steering committee
“Tolebrutinib represents a new class of therapy for the treatment of multiple sclerosis. In this large phase 3 study, tolebrutinib was found to slow the progression of disability in a subset of multiple sclerosis for which we have no approved therapies – non-relapsing secondary progressive disease. The results of this study signal a new chapter in multiple sclerosis because we finally found a potential way to treat non-relapsing secondary progressive forms.”
Dr Fox is a paid advisor to Sanofi for the HERCULES study.

Erik Wallström, MD, PhD
Global Head of Neurology Development
“By targeting disability progression mechanisms behind the blood-brain barrier, tolebrutinib has the potential to be a practice-changing therapeutic option for people living with multiple sclerosis. The data published in NEJM support our larger commitment to the multiple sclerosis patient community, transforming the treatment paradigm to defy disability across the disease spectrum.”

The HERCULES data demonstrated that tolebrutinib delayed the time to onset of 6-month confirmed disability progression (CDP) by 31% compared to placebo (hazard ratio [HR] 0.69; 95% confidence interval [CI] 0.55-0.88; p=0.003). Additionally, results from the GEMINI 1 and 2 phase 3 studies, evaluating tolebrutinib in people with relapsing multiple sclerosis (RMS) were also published in NEJM and presented today during the Clinical Trials Plenary Session at the AAN 2025 Annual Meeting.

The GEMINI 1 and 2 results did not show superiority on the primary endpoint of reducing annualized relapse rate (ARR) over teriflunomide. The ARR during the study period was 0.13 in the tolebrutinib group and 0.12 in the teriflunomide group in GEMINI 1 (adjusted rate ratio, 1.06; 95% CI 0.81-1.39; p=0.67) and was 0.11 in both groups in GEMINI 2 (adjusted rate ratio, 1.00; 95% CI 0.75-1.32; p=0.98). A pooled analysis for a key secondary endpoint, not controlled for multiplicity, showed that tolebrutinib delayed the time to onset of 6-month confirmed disability worsening by 29% versus teriflunomide (HR 0.71; 95% CI 0.53 to 0.95).

Tolebrutinib was generally well-tolerated across all arms of the studies for all participants. In HERCULES, liver enzyme elevations (>3xULN) were observed in 4.0% of participants receiving tolebrutinib compared with 1.6% in the placebo group. A small (0.5%) proportion of participants in the tolebrutinib group experienced peak ALT increases of >20xULN, all occurring within the first 90 days of treatment. All but one case of liver enzyme elevations resolved without further medical intervention. Prior to the implementation of more frequent liver enzyme monitoring following treatment initiation, one participant in the tolebrutinib arm received a liver transplant and died due to post-operative complications. The implementation of more frequent liver monitoring may help to mitigate serious liver sequelae. Other deaths in the study were assessed as unrelated to treatment by investigators; deaths were even across the placebo and tolebrutinib arms at 0.3%.

In an analysis of the GEMINI 1 and 2 pooled safety data, adverse events observed between the tolebrutinib and teriflunomide arms were generally balanced. Liver enzyme elevations (>3xULN) were observed in 5.6% of participants receiving tolebrutinib compared to 6.3% in the teriflunomide arms. A small (0.5%) proportion of participants in the tolebrutinib group experienced peak ALT increases of >20xULN, all occurring within the first 90 days of treatment. Deaths were balanced across the teriflunomide and tolebrutinib arms, at 0.2% and 0.1% respectively, and were assessed as unrelated to treatment by the investigators.

The safety and efficacy of tolebrutinib have not been determined by any regulatory authority.
The regulatory submission for tolebrutinib to treat nrSPMS and to slow disability accumulation independent of relapse activity in adult patients is being evaluated under priority review by the US Food and Drug Administration with a target action date of September 28, 2025. A regulatory submission is also under review in the EU.

About multiple sclerosis 
Multiple sclerosis is a chronic, immune-mediated disease of the central nervous system that may result in accumulation of irreversible disabilities over time.

The physical and cognitive disability impairments translate into gradual deterioration of health status, impacting patients’ quality of life. Disability accumulation remains the significant unmet medical need in MS. To date, the primary target of currently approved medicines has been peripheral B and T cells, while innate immunity within the CNS, which is believed to drive disability accumulation, remains largely unaddressed.

Currently approved or late-stage medicines being tested for MS mainly target the adaptive immune system and/or do not act directly within the central nervous system to drive clinical benefit.

Living with nrSPMS refers to people with MS who have stopped experiencing relapses but continue to accumulate disability, experienced as symptoms such as fatigue, cognitive impairment, balance and gait impairment, loss of bowel and/or bladder function, sexual disfunction, amongst others.

About tolebrutinib 
Tolebrutinib is an investigational, oral, brain-penetrant and bioactive Bruton’s tyrosine kinase (BTK) inhibitor specifically designed to target smoldering neuroinflammation, a key driver of disability progression in multiple sclerosis. Unlike conventional MS therapies that primarily address peripheral inflammation, tolebrutinib crosses the blood-brain barrier to achieve therapeutic cerebrospinal fluid concentrations, allowing it to modulate both B-lymphocytes and disease-associated microglia within the central nervous system. This mechanism is thought to directly address the underlying pathology of progressive MS by targeting the inflammatory processes that contribute to neurodegeneration and disability accumulation.

Tolebrutinib was previously granted breakthrough therapy designation by the FDA, based on positive results from the HERCULES phase 3 study in adults with non-relapsing secondary progressive MS. Tolebrutinib is being evaluated in a phase 3 clinical study for the treatment of primary progressive multiple sclerosis and its safety and efficacy have not been determined by any regulatory authority worldwide. For more information on tolebrutinib clinical studies, please visit www.clinicaltrials.gov.

Tolebrutinib represents Sanofi’s commitment to developing innovative treatments that address the underlying causes of neurological diseases and potentially transform the treatment landscape.

Standing at the intersection of neurology and immunoscience, Sanofi is focused on improving the lives of those living with serious neuro-inflammatory and neuro-degenerative conditions including MS, chronic inflammatory demyelinating polyneuropathy, Alzheimer’s disease, Parkinson’s disease, age-related macular degeneration, and other neurological diseases. The neurology pipeline currently has several projects in phase 3 studies across 7 indications.”

Press Release: Tolebrutinib phase 3 data published in NEJM demonstrate benefit on disability progression in multiple sclerosis

Novartis receives FDA accelerated approval for Vanrafia for proteinuria reduction in primary IgA nephropathy

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Novartis receives FDA accelerated approval for Vanrafia for proteinuria reduction in primary IgA nephropathy

April 3, 2025 : “Novartis announced the US FDA has granted accelerated approval for Vanrafia® (atrasentan), a potent and selective endothelin A (ETA) receptor antagonist, for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression.

This is generally defined as a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g. Vanrafia is a once-daily, non-steroidal, oral treatment that can be added onto supportive care, including a renin-angiotensin system (RAS) inhibitor with or without a sodium-glucose co-transporter-2 (SGLT2) inhibitor.

Vanrafia was granted accelerated approval based on a prespecified interim analysis of the Phase III ALIGN study measuring the reduction of proteinuria at 36 weeks compared to placebo. It has not been established whether Vanrafia slows kidney function decline in patients with IgAN.

The continued approval of Vanrafia may be contingent upon the verification of clinical benefit from the ongoing Phase III ALIGN study evaluating whether Vanrafia slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline at week 136.

The eGFR data are expected in 2026 and intended to support traditional FDA approval.

“Today’s approval marks an important milestone for people living with IgA nephropathy, offering a new option that can be seamlessly integrated into their existing treatment plan, with no REMS requirement,” said Richard Lafayette, M.D., F.A.C.P., Professor of Medicine, Nephrology and Director of the Glomerular Disease Center at Stanford University Medical Center, and Vanrafia ALIGN Study Investigator and Steering Committee Member.

“Vanrafia is a selective ETA receptor antagonist that effectively reduces proteinuria, a major risk factor in IgAN. T

aking early, decisive action is critical to help improve outcomes for these patients who too often progress toward kidney failure.”

IgAN is a progressive, rare kidney disease in which the immune system attacks the kidneys, often causing glomerular inflammation and proteinuria.

With almost 13 out of every million people in the US diagnosed per year, it is one of the most common autoimmune kidney diseases, and each person’s journey is unique. Up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring maintenance dialysis and/or kidney transplantation, and response to treatment can vary.

Effective, targeted therapies with different mechanisms of action can help physicians select the most appropriate treatment for patient.

“My son was diagnosed with IgA nephropathy long before there were any medicines approved to treat this condition, so the availability of multiple treatment options is incredibly meaningful for this community,” said Bonnie Schneider, Director and Co-Founder, IgA Nephropathy Foundation.

“The approval of Vanrafia broadens the treatment landscape and expands the opportunity to tailor care in a disease that can impact each patient so differently.”

Data supporting approval
In the ongoing Phase III ALIGN study, patients receiving Vanrafia in combination with a RAS inhibitor achieved clinically meaningful and statistically significant proteinuria reduction of 36.1% (P<0.0001) compared to placebo, with results seen as early as week 6 and sustained through week 36.

The effect of Vanrafia on UPCR was consistent across subgroups, including age, sex, race, and baseline disease characteristics, such as eGFR and proteinuria levels, in the main study cohort.

A similar treatment effect of Vanrafia was seen in an additional group of patients treated with both a RAS inhibitor and an SGLT2 inhibitor (37.4% reduction in UPCR vs. placebo)1.

The ALIGN study showed that Vanrafia has a favorable safety profile consistent with previously reported data. Adverse events reported in ≥2% of patients treated with Vanrafia, and more frequently than placebo, include peripheral edema, anemia, and liver transaminase elevation.

Because some endothelin receptor antagonists have caused elevations of aminotransferases, hepatotoxicity, and liver failure, clinicians should obtain liver enzyme testing before initiating Vanrafia and during treatment when clinically indicated.

Vanrafia may cause serious birth defects. Vanrafia does not require a REMS program.

Transforming care in kidney disease
“We are proud to expand the treatment landscape in IgA nephropathy with today’s FDA accelerated approval of Vanrafia.

IgAN is a heterogenous condition that requires differentiated therapies with unique mechanisms of action, and with our multi-asset kidney disease portfolio, we are well positioned to support a broad patient population and advance care for this disease,” said Victor Bultó, President, US, Novartis.

“Building on our longstanding legacy in nephrology, we continue to rapidly grow our capabilities in this space. Each launch enables us to more effectively reach patients with the most suitable treatment option and deliver on our promise to transform kidney disease care.”

This is the third US approval received by Novartis for its kidney disease portfolio in the last year, with Fabhalta® having been granted FDA approval in C3 glomerulopathy (C3G) in March 2025 and accelerated approval in IgAN in August 2024.

Fabhalta is also being studied in a broad range of rare kidney diseases, including atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN).

Studies are ongoing to evaluate the safety and efficacy profiles in these investigational indications and support potential regulatory submissions.

An investigational subcutaneously administered anti-APRIL monoclonal antibody, zigakibart, is currently in Phase III development in IgAN, with results expected in 2026.”

Novartis receives FDA accelerated approval for Vanrafia® (atrasentan), the first and only selective endothelin A receptor antagonist for proteinuria reduction in primary IgA nephropathy (IgAN) | Novartis

AstraZeneca showcases innovation in infectious disease protection at ESCMID Global 2025

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AstraZeneca showcases innovation in infectious disease protection at ESCMID Global 2025

April 8, 2025: “AstraZeneca will share new data across its Vaccines & Immune Therapies portfolio at the 2025 Congress of the European Society of Clinical Microbiology and Infectious Diseases (ESCMID Global 2025) in Vienna, Austria, from 11-15 April 2025.

With 13 abstracts, including two oral presentations and two late-breaking poster presentations, the Company will highlight progress in advancing novel immunisations against bacterial and viral infectious diseases and share real-world evidence showing the burden of respiratory viral infections and the continued need for protection.

Benjamin Moutier, Senior Vice President, Vaccines & Immune Therapies, AstraZeneca said: “AstraZeneca is building an innovative portfolio of antibodies and vaccines to tackle two of the top global public health threats – serious bacterial infections and respiratory viral infections.

At ESCMID, we’ll share data on three novel monoclonal antibodies targeting high-priority pathogens, highlighting our ambition to redefine care for serious bacterial infections.

In addition, we will present 20 years of effectiveness data for Fluenz/FluMist, as well as real-world data highlighting the ongoing burden of RSV and hMPV, two leading causes of respiratory illnesses.”

Advancing potential new options for serious bacterial infection prevention and treatment

Serious bacterial infections pose significant and growing threats to global health, linked to an estimated 7.7 million deaths a year globally, leading to increasing morbidity and mortality for patients and a substantial burden on health systems.

At ESCMID, the Company will share preclinical data on investigational monoclonal antibodies (mAbs) targeting three of the top five pathogens identified as urgent risks by the Centers for Disease Control and Prevention:

  • Staphylococcus aureus (S. aureus): Oral presentation on in vivo expression of an mRNA-encoded multi-mechanistic mAb combination against S. aureus. There are millions of methicillin-resistant S. aureus (MRSA) infections annually worldwide, and MRSA is resistant to many types of antibiotics, making treatment highly complex. A pathogen-specific treatment strategy with mAbs targeting some S. aureus virulence factors represents an alternative to broad spectrum antibiotics.
  • Pseudomonas aeruginosa (P. aeruginosa): Preclinical data showing that gremubamab, an anti-P. aeruginosa bispecific human mAb, may enhance neutrophil-mediated killing of P. aeruginosa in bronchiectasis patients. P. aeruginosa is a pathogen that colonizes one-third of patients with bronchiectasis, a chronic lung condition impacting about one million people globally and drives significant disease morbidity and mortality.
  • Klebsiella pneumoniae (K. pneumoniae): Research on the development and characterisation of bispecific antibodies that protect against bacteraemia and pneumonia from the major serotypes of antibiotic-resistant K. pneumoniae in mouse models. Infections caused by K. pneumoniae are responsible for more than 700,000 yearly deaths worldwide.4

Real-world data examining the impact of respiratory infections

AstraZeneca is presenting 20 years of real-world data on Fluenz/FluMist (live attenuated influenza vaccines, LAIV), recently approved in the U.S. as the only vaccine for self- or caregiver administration for the prevention of influenza, demonstrating long-term effectiveness comparable to that of inactivated (IIV) influenza vaccines in children.

The Company will also present two late-breaking posters featuring real-world studies in patients with haematological malignancies who are highly susceptible to severe respiratory infections.

The studies show that outcomes from human metapneumovirus (hMPV) and respiratory syncytial virus (RSV) were similar to those from influenza and COVID-19, highlighting the need for development of novel options for preventing disease caused by hMPV and RSV.

Retrospective analyses from Valencia, Spain, where there is systematic testing for multiple respiratory viruses, highlight the impact of RSV and influenza on severe disease and mortality in older adults throughout the COVID-19 pandemic, and suggest a potentially greater proportion of mechanical ventilation use and in-hospital death from RSV than influenza in adolescents and adults.

Additional real-world data from France highlight the extensive healthcare resource utilisation due to hMPV and RSV and underline the need for routine testing for hMPV to accurately assess its true burden.

Additionally, data being presented by our partner, Sanofi, confirm the significant public health impact and real-world effectiveness of Beyfortus in reducing RSV disease and hospitalisations in infants.”

AstraZeneca showcases innovation in infectious disease protection at ESCMID Global 2025

FDA Alerts Patients of Potential to Miss Critical Safety Alerts Due to Phone Settings When Using Smartphone-Compatible Diabetes Devices

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FDA Alerts Patients of Potential to Miss Critical Safety Alerts Due to Phone Settings When Using Smartphone-Compatible Diabetes Devices

February 05, 2025: “The U.S. Food and Drug Administration is alerting patients of a safety concern regarding diabetes devices, such as continuous glucose monitors (CGMs), insulin pumps and automated insulin dosing systems, that rely on a smartphone to deliver critical safety alerts.

Users of these smartphone-compatible diabetes devices can configure alert settings, such as which alerts to receive, how often and how the alerts are delivered (e.g. audible, vibration, text only) through the app on their phone.

The FDA has received medical device reports in which users report these alerts are not being delivered or not being heard, in cases where the users thought they had configured the alerts to be delivered.

In some cases, missing these alerts may have contributed to serious harm, including severe hypoglycemia (low blood sugar), severe hyperglycemia (high blood sugar), diabetic ketoacidosis (when the body does not have enough insulin to use blood sugar for energy) and death.

“Modern medical devices, such as diabetes devices that connect to a smartphone, can provide users with the convenience and flexibility to configure alerts that are personalized to them.

However, users should stay aware of alert settings and monitor these devices to ensure they continue to receive critical alerts as expected,” said Courtney Lias, director of the Office of In Vitro Diagnostic Products in the FDA’s Center for Devices and Radiological Health.

“Even if configured correctly, certain hardware or software changes can interrupt the expected operation of these critical devices, which can lead to patient harm if undetected.”

The FDA has identified, among others, the following hardware and software changes, updates and configurations that may lead to critical alerts not being received as expected:

  • software configuration issues, such as app notification permissions, using “do not disturb” or “focus mode” or the app entering “deep sleep” after a period of not being used;
  • connecting new hardware to the smartphone, such as connecting to car audio or using wireless earphones, that can change the default volume of alerts or prevent delivery of alerts; and
  • smartphone operating system updates that are not supported by the medical device application.

The FDA’s safety communication provides recommendations, such as the following, for users of these devices:

  • Carefully follow the instructions provided by diabetes device manufacturers when installing, setting up or updating mobile medical apps on the smartphone;
  • turn off automatic operating system (OS) updates to the smartphone and do not update the phone’s OS until confirming the diabetes device app is compatible with the new OS version;
  • after updating the phone’s OS or adding a new accessory, such as wireless headphones, confirm alert settings then carefully monitor the medical device app to make sure alerts are received and can be heard as expected;
  • at least once a month, check that the smartphone alerts are configured as expected;
  • if alerts are not being received as expected from the mobile medical app, or cannot be heard, call the technical support number for the medical device for assistance; and
  • report any problems with the diabetes device to the FDA.

The FDA is working with diabetes-related medical device manufacturers to ensure that smartphone alert configurations of their devices are carefully evaluated prior to use by patients.

The agency is also working with manufacturers to ensure that settings in smartphones and mobile medical apps that may impact safety alerts are continuously tested and any updates to recommended configurations are communicated quickly and clearly to users.”

https://www.fda.gov/news-events/press-announcements/fda-alerts-patients-potential-miss-critical-safety-alerts-due-phone-settings-when-using-smartphone

Pfizer to Showcase Advancements Across Genitourinary Cancers at ASCO GU Cancers Symposium

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Pfizer to Showcase Advancements Across Genitourinary Cancers at ASCO GU Cancers Symposium

January 28, 2025: “Pfizer will present the latest results from its leading genitourinary (GU) portfolio at the American Society of Clinical Oncology Genitourinary (ASCO GU) Cancers Symposium taking place February 13-15 in San Francisco, California.

Data from more than 20 company-sponsored, investigator-sponsored, and collaborative research abstracts, including five oral presentations, highlight advancements in developing new standards of care within prostate and bladder cancer across the company’s core scientific modalities, including small molecules and antibody-drug conjugates.

“Our long-standing commitment to the genitourinary cancer community has been foundational in our mission to transform the treatment landscapes for patients with bladder and prostate cancers,” said Karin Tollefson, Chief Oncology Medical Officer, Pfizer.

“Our strong presence at this year’s ASCO GU highlights the longer-term impacts of our approved leading medicines for patients in their respective indications. We are also looking forward to sharing updates from our rapidly growing pipeline of novel targets and combination approaches, which have the potential to help address the diverse needs of patients across various stages of disease.”

Pfizer’s GU portfolio includes seven approved medicines across bladder, prostate, and kidney cancers, as well as a growing late-stage pipeline of scientific modalities and combination approaches.

Key Pfizer presentations at ASCO GU include the detailed overall survival (OS) results from the Phase 3 TALAPRO-2 trial with TALZENNA® (talazoparib) plus XTANDI® (enzalutamide) in patients with metastatic castration-resistant prostate cancer (mCRPC), which will be featured in ASCO GU’s official Press Program.

In addition, updated analysis from the Phase 3 global EV-302 study of PADCEV® (enfortumab vedotin-ejfv) in combination with pembrolizumab in locally advanced or metastatic urothelial cancer (la/mUC) will be presented, highlighting the long-term efficacy benefits of the combination.

From the pipeline, the first randomized progression-free survival (PFS) data from the ongoing Phase 1 dose-escalation study for mevrometostat plus XTANDI reinforce the potential of this investigational combination for patients with mCRPC.

Additional pipeline presentations include updated data and real-world evidence for different types of bladder cancer, supporting the development of two potential transformative treatments, disitamab vedotin, an investigational antibody-drug conjugate (ADC), and sasanlimab, an investigational subcutaneous PD-1 blocker.

Key ASCO GU Presentations

Prostate Cancer

  • TALZENNA plus XTANDI:Oral and poster presentations from the pivotal Phase 3 TALAPRO-2 trial of TALZENNA in combination with XTANDI will provide detailed results on the statistically significant and clinically meaningful improvement in OS in all-comers (cohort 1) as well as in those patients with homologous recombination repair (HRR) gene-mutated mCRPC (cohort 2), compared to XTANDI alone.

    TALZENNA in combination with XTANDI has been approved for use in over 35 countries globally for patients with certain types of mCRPC*. The OS results will be shared with global health authorities to potentially update the TALZENNA label.
  • XTANDI: Six abstracts continue to underscore the benefit of XTANDI across its approved indications, including two posters highlighting follow-up analysis from the EMBARK trial of XTANDI in combination with leuprolide in patients with non-metastatic castration-sensitive prostate cancer with biochemical recurrence at high-risk for metastasis.
  • Mevrometostat: The first randomized PFS results from the ongoing Phase 1 dose-expansion study examine the potential of mevrometostat (PF-06821497), an investigational selective inhibitor of enhancer of zeste homolog 2 (EZH2), in combination with XTANDI in patients with mCRPC, compared to XTANDI alone.
  • Pfizer initiated two pivotal Phase 3 trials for mevrometostat plus XTANDI in 2024 and expects to start a Phase 3 study of mevrometostat plus XTANDI in first-line mCSPC during the first half of 2025.

Bladder Cancer

  • PADCEV: Long-term follow-up data from the groundbreaking Phase 3 EV-302 study of PADCEV in combination with pembrolizumab, including OS and safety data, continue to demonstrate consistent efficacy versus chemotherapy in a broad population, reinforcing the combination as standard of care in first-line treatment of la/muC.
  • Disitamab vedotin: Updated efficacy and safety data from an ongoing Phase 2 study (sponsored by Remegen) evaluating the HER2-targeting ADC disitamab vedotin plus toripalimab show encouraging results as a perioperative regimen in HER2-expressing muscle-invasive bladder cancer (MIBC).
  • These data add to the growing body of evidence supporting the continued development of disitamab vedotin across stages of bladder cancer.
  • Sasanlimab: Three real-world evidence poster presentations highlight the need for advanced treatment options for patients with non-muscle invasive bladder cancer (NMIBC), including presentations on Bacillus Calmette-Guérin (BCG) treatment patterns, impact of BCG shortages, and outcomes and treatment patterns in patients with high-risk NMIBC.

    Pfizer recently reported positive topline results for sasanlimab in combination with BCG as induction therapy with or without maintenance in patients with BCG- naïve, high-risk NMIBC. Detailed results from the Phase 3 CREST trial will be presented at an upcoming congress.

Additional information on key Pfizer-sponsored abstracts at ASCO GU 2025, including date and time of presentation, follow in the chart below.

*TALZENNA in combination with XTANDI was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with HRR gene-mutated mCRPC in June 2023.”

https://www.pfizer.com/news/press-release/press-release-detail/pfizer-showcase-advancements-across-genitourinary-cancers

FDA Approves Novel Non-Opioid Treatment for Moderate to Severe Acute Pain

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FDA Approves Novel Non-Opioid Treatment for Moderate to Severe Acute Pain

January 30, 2025: “The U.S. Food and Drug Administration approved Journavx (suzetrigine) 50 milligram oral tablets, a first-in-class non-opioid analgesic, to treat moderate to severe acute pain in adults.

Journavx reduces pain by targeting a pain-signaling pathway involving sodium channels in the peripheral nervous system, before pain signals reach the brain.  

Journavx is the first drug to be approved in this new class of pain management medicines.

Pain is a common medical problem and relief of pain is an important therapeutic goal. Acute pain is short-term pain that is typically in response to some form of tissue injury, such as trauma or surgery.

Acute pain is often treated with analgesics that may or may not contain opioids.

The FDA has long supported development of non-opioid pain treatment. As part of the FDA Overdose Prevention Framework, the agency has issued draft guidance aimed at encouraging development of non-opioid analgesics for acute pain and awarded cooperative grants to support the development and dissemination of clinical practice guidelines for the management of acute pain conditions.  

“Today’s approval is an important public health milestone in acute pain management,” said Jacqueline Corrigan-Curay, J.D., M.D., acting director of the FDA’s Center for Drug Evaluation and Research.

“A new non-opioid analgesic therapeutic class for acute pain offers an opportunity to mitigate certain risks associated with using an opioid for pain and provides patients with another treatment option.

This action and the agency’s designations to expedite the drug’s development and review underscore FDA’s commitment to approving safe and effective alternatives to opioids for pain management.”

The efficacy of Journavx was evaluated in two randomized, double-blind, placebo- and active-controlled trials of acute surgical pain, one following abdominoplasty and the other following bunionectomy.

In addition to receiving the randomized treatment, all participants in the trials with inadequate pain control were permitted to use ibuprofen as needed for “rescue” pain medication.

Both trials demonstrated a statistically significant superior reduction in pain with Journavx compared to placebo.

The safety profile of Journavx is primarily based on data from the pooled, double-blind, placebo- and active-controlled trials in 874 participants with moderate to severe acute pain following abdominoplasty and bunionectomy, with supportive safety data from one single-arm, open-label study in 256 participants with moderate to severe acute pain in a range of acute pain conditions.

The most common adverse reactions in study participants who received Journavx were itching, muscle spasms, increased blood level of creatine phosphokinase, and rash. Journavx is contraindicated for concomitant use with strong CYP3A inhibitors.

Additionally, patients should avoid food or drink containing grapefruit when taking Journavx.

The application received Breakthrough Therapy, Fast Track and Priority Review designations by the FDA.  

The FDA granted approval of Journavx to Vertex Pharmaceuticals Incorporated.”

https://www.fda.gov/news-events/press-announcements/fda-approves-novel-non-opioid-treatment-moderate-severe-acute-pain

Johnson & Johnson investigational therapy was recently granted U.S. FDA Priority Review for the treatment of gMG

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Johnson & Johnson investigational therapy was recently granted U.S. FDA Priority Review for the treatment of gMG

January 23, 2025: “Johnson & Johnson announced The Lancet Neurology has published results from the pivotal Phase 3 study of nipocalimab, an investigational FcRn blocker, evaluated in a broad population of antibody positive (anti-AChR+, anti-MuSK+, anti-LRP4+) adults with generalized myasthenia gravis (gMG).

The Vivacity-MG3 study met its primary endpoint demonstrating statistically significant and clinically meaningful improvement over 24 weeks in the MG-ADL score.

 Nipocalimab had a tolerable safety profile, with adverse events leading to discontinuation rates similar to placebo (5.1% with nipocalimab vs. 7.1% with placebo).

“Nipocalimab has been shown in multiple clinical studies to help reduce IgG, including autoantibodies, among this broad population of antibody positive adults with gMG.

The positive results from the Vivacity-MG3 study further support the potential of nipocalimab to address the underlying cause of this debilitating autoantibody disease,” said Carlo Antozzi, M.D., Neuroimmunology and Muscle Pathology Unit of the Neurological Institute Foundation C. Besta of Milan, Italy.

“It’s promising to see this positive data published in The Lancet Neurology as there is a continued need for additional approved targeted therapies with demonstrated safety profiles that offer sustained disease control for a broad range of antibody positive patients living with gMG.”

gMG is a chronic, life-long, rare, autoantibody-driven disease, for which there currently is no cure. gMG impacts an estimated 700,000 people worldwide.

Nipocalimab, a fully human IgG1 antibody, is an immunoselective investigational therapy that has been shown in clinical trials to lower immunoglobulin G (IgG), including pathogenic IgG, one of the root causes of autoantibody diseases.

Data from the Phase 3 study showed up to a 75% reduction in the median pre-dose total IgG from baseline. Additionally, reduction in levels of common pathogenic IgG subclasses, including AChR antibody and MuSK antibody, was observed over 24 weeks of the study.

No changes were observed in total IgE, IgA, and IgM, highlighting the potential ability to maintain a protective immune system even after reduction of pathogenic IgG autoantibodies is observed.

Nipocalimab plus SOC demonstrated a significantly greater reduction in MG-ADL response (≥2-point improvement from baseline) compared with placebo plus SOC (p=0.0213).

For someone living with gMG, a 1- to 2-point change on MG-ADL may be the difference between normal eating and frequent choking on food, or shortness of breath at rest and requiring the assistance of a ventilator.

“The Phase 3 Vivacity-MG3 data demonstrates our steadfast pursuit of researching and developing potential innovative and transformational approaches for autoantibody-driven diseases, such as gMG,” said Sindhu Ramchandren, M.D., Executive Medical Director, Neuroscience, Johnson & Johnson Innovative Medicine.

“We are delighted by the publication of this robust Phase 3 data in The Lancet Neurology as well as the Priority Review granted by the FDA.

People living with gMG require additional effective immunoselective therapeutic options that can potentially preserve the ability to maintain a protective immune response even after reduction of IgG.”

Johnson & Johnson submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of nipocalimab in gMG on August 29 and September 11, 2024, respectively.

Nipocalimab was granted Priority Review by the FDA which was supported by findings from the Phase 3 Vivacity-MG3 study.

In addition, nipocalimab recently received U.S. FDA Breakthrough Therapy Designation for the treatment of adults with moderate-to-severe Sjögren’s disease as supported by results from the Phase 2 DAHLIAS study.

Bayer and Pula Foundation partner up to insure 10 million smallholder farmers in Sub-Saharan Africa and South Asia

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Bayer and Pula Foundation partner up to insure 10 million smallholder farmers in Sub-Saharan Africa and South Asia

January 23,2025: “Bayer Foundation and the Pula Foundation announced at World Economic Forum that they are planning to provide insurance coverage for 10 million smallholder farmers by 2030.

The collaboration aims to enhance climate resilience among smallholder farmers, protecting them against the growing impacts of droughts and floods, which threaten harvests, livelihoods, and global food security.

It will help build private-public collaborations and shape the insurance market for agriculture in Africa and Asia.

The Pula Foundation has developed scalable and data-driven agricultural insurance solutions designed to safeguard smallholder farmers’ investments in their farms.

By mitigating risks associated with extreme weather events such as droughts and floods, the Pula Foundation ensures that farmers receive financial compensation for yield losses, enabling them to recover, reinvest, and build long-term resilience in the face of climate uncertainty.

By 2030, Bayer Foundation’s grant for insurance premium support in the amount of 10 million euros – supported by a donation from Bayer’s Crop Science Division – will unlock a potential insurance coverage of 127 million U.S. dollars for 10 million farmers working with national governments in Bangladesh, Pakistan, Malawi, Ghana, Nigeria, Kenya and Mali.

The grant originates from Bayer Foundation’s Social Innovation Ecosystem Fund which targets mature and high-impact solutions for underserved communities.

“Smallholder farmers are already affected heavily by the impacts of climate change, and this will get worse going forward.

It is crucial that we enable them to feed their communities and contribute to global food security,” said Matthias Berninger, Executive Vice President Public Affairs, Sustainability and Safety at Bayer and Member of the Board of Trustees at Bayer Foundation.

“Our Crop Science division is committed to deliver innovative, farmer-focused solutions that drive sustainable growth and regenerative agriculture.

By unlocking climate finance and collaborating with partners like the Pula Foundation, we aim to deliver ecosystem-based approaches that empower smallholder farmers and their communities to overcome challenges and thrive,” added Rodrigo Santos, President of Bayer’s Crop Science Division, Member of the Board of Management of Bayer AG and Executive Director Bayer Foundation.

Pula Foundation and Bayer Foundation already demonstrated in 2021 that building such public-private partnerships to offer insurance protection at scale works: Together with the Zimbabwean Government and as part of the Zimbabwean Conservation Agriculture Program, they developed an insurance solution that protects the input investments farmers made.

With the catalytic support of Bayer Foundation for this pilot, 31,000 farmers were insured against climate risks such as droughts in the first year, which the public-private partnership network of Pula Foundation scaled to more than 1 million farmers after three years.

“Climate resilience is not just about recovery but also about dignity and empowerment. We believe that insurance enables smallholder farmers to prepare themselves for an increasingly volatile climate, rather than waiting for handouts. We have witnessed firsthand how farmers bounce back stronger after climate shocks when they have the right tools and support,” said Rose Goslinga, Director of Pula Foundation.

“This partnership with Bayer Foundation will enable us to expand our reach and ensure that millions more farmers can secure their livelihoods and build resilience against climate risks.”

According to FAO, in 2023, around 2.33 billion people globally faced moderate or severe food insecurity.

Smallholder farmers make a significant contribution to food security and supply in their countries: more than half of the food consumed in low- and middle-income countries (LMICs) is produced by smallholders.

Yet, they face heavy challenges that pose severe constraints to their ability to serve their communities. Examples are lack of climate change adaptation tools, lack of access to agricultural inputs and inadequate credit and insurance services.

Bayer aims to support a total of 100 million smallholder farmers in LMICs by 2030 by improving their access to agricultural products and services. This also includes collaborations with partners.

To achieve this, Bayer strives to create market models that generate benefits and reduce business risks for all partners in the value chain, including smallholder farmers.

This is implemented by helping smallholder farmers gain access to the agricultural value chain and increase their productivity and income, as well as by creating resilience to ensure the long-term food security of smallholder farmers, their families and rural regions in LMICs.”

https://www.bayer.com/media/en-us/bayer-and-pula-foundation-partner-up-to-insure-10-million-smallholder-farmers-in-sub-saharan-africa-and-south-asia

First participants randomized in AskBio Phase II gene therapy trial for Parkinson’s disease

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First participants randomized in AskBio Phase II gene therapy trial for Parkinson’s disease

January 14, 2025: “AskBio Inc, a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that the first participants have been randomized in its Phase II clinical trial in patients with Parkinson’s disease (PD).

“The randomization of the first participants in REGENERATE-PD is positive news for people living with Parkinson’s disease and the physicians treating them,” said Dr Rajesh Pahwa, MD, Director, Parkinson’s Disease and Movement Disorder Center, University of Kansas Medical Center, USA, and REGENERATE-PD Principal Investigator.

“There is a significant need for neurorestorative therapies in Parkinson’s and seeing the advancement of an important investigational gene therapy in a Phase II clinical trial gives hope to patients and the medical community.”

In 2024, AskBio initiated recruitment in the United States arm of REGENERATE-PD. The objective of this randomized, double-blind, Phase II clinical trial is to evaluate the safety and efficacy of AB-1005 delivered to the putamen in adult participants aged 45–75 years with moderate-stage PD.

REGENERATE-PD is estimated to enroll approximately 87 participants across clinical centers that are being opened in the United States, Germany, Poland, and the United Kingdom.

AskBio also presented 36-month Phase Ib data at the International Congress of Parkinson’s Disease and Movement Disorders, which was held in Philadelphia, Pennsylvania, from September 27 to October 1, 2024.

The data demonstrated that administration of AB-1005 was well tolerated with no attributed serious adverse events.

The Moderate PD cohort showed trends for improvement or stability on several motor scales at 36 months, including Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and motor diaries, and trends in reductions in Parkinson’s medications (levodopa-equivalent daily dose [LEDD]).

Most participants in the Mild PD cohort showed an overall stable clinical status with little change in MDS-UPDRS, the self-reported PD motor diary, or LEDD.

“AskBio continues to mark significant milestones in the clinical development of the investigational gene therapy AB-1005 as we strive to bring a safe and effective gene therapy to patients with moderate stage Parkinson’s disease,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio.

“With REGENERATE-PD participants now being randomized, we are excited by our progress with AB-1005 and look forward to sharing further updates at an appropriate scientific forum as the program advances through next year and beyond.”

“The advancement of AskBio’s Parkinson’s program is a significant milestone that highlights our commitment to exploring the potential of gene therapy in an area of significant unmet medical need,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division, and Global Head of Research and Development.

“We believe that therapeutic modalities like gene therapy can change the treatment landscape for people living with the debilitating effects of Parkinson’s disease and look forward to seeing what we will learn in this next phase of research and development for AB-1005.”

AskBio is also exploring AB-1005 beyond Parkinson’s disease and is currently enrolling participants in the United States with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase I clinical trial to assess the preliminary safety, tolerability, and efficacy for this rapidly progressing condition.

AB-1005 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.

About Parkinson’s disease
Parkinson’s disease (PD) is a progressive neurodegenerative disease. It has a significant impact on a person’s daily life. In PD the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function.

Symptoms include tremors, muscle rigidity, and slowness of movement. Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, congestive issues, and depression.

Symptoms typically intensify over time and make everyday tasks increasingly demanding. The prevalence of PD has doubled over the past 25 years. More than 10 million people worldwide are estimated to be living with PD.

This makes it the world’s second most prevalent neurodegenerative disease. It is also the most frequent movement disorder. At present there is no cure, and current treatment options lack the holistic management of symptoms, so new therapies are needed.

About REGENERATE-PD
REGENERATE-PD is a Phase II, randomized, double-blind, sham-controlled trial of the efficacy and safety of intraputaminal AB-1005 in the treatment of adults (45-75 years) with moderate-stage Parkinson’s disease.

The trial will include an estimated 87 subjects with trial sites located in the United States, Germany, Poland, and the United Kingdom. For more information about the REGENERATE-PD clinical trial, visit clinicaltrials.gov (NCT06285643), or visit askbio.com.

About AB-1005
AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, which allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with MRI-monitored convection enhanced delivery.

In nonclinical studies, GDNF has been shown to promote the survival and morphological differentiation of dopaminergic neurons. Recombinant GDNF has long been evaluated as a potential treatment for diseases, such as Parkinson’s disease (PD), marked by progressive degeneration of midbrain dopaminergic neurons. 

Through a combination of an investigational gene therapy and innovative neurosurgical delivery approach, we can now test the GDNF hypothesis in PD by getting this neurotrophic factor to these degenerating nigrostriatal neurons in a potentially more clinically relevant fashion.

https://www.bayer.com/media/en-us/first-participants-randomized-in-askbio-phase-ii-gene-therapy-trial-for-parkinsons-disease

Johnson & Johnson initiated submission of NDA application with U.S. FDA for TAR-200 for BCG-unresponsive high-risk non-muscle-invasive bladder cancer

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Johnson & Johnson initiated submission of NDA application with U.S. FDA for TAR-200 for BCG-unresponsive high-risk non-muscle-invasive bladder cancer

 January 15, 2025: “Johnson & Johnson announced it has initiated the submission of an original New Drug Application with the U.S. Food and Drug Administration for TAR-200 for the treatment of patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC) with carcinoma in situ (CIS), with or without papillary tumors.

This submission is being reviewed by the FDA through the Real-Time Oncology Review (RTOR) program, which allows the FDA to review data before the complete application is formally submitted and helps ensure treatments are available for patients as soon as possible.

“Upon approval, TAR-200 promises to be a meaningful additional treatment option for certain patients with NMIBC, addressing a critical need for people who have had relatively limited therapeutic alternatives.

Many patients face life-altering surgical options such as radical cystectomy, which is complete bladder removal,” said Yusri Elsayed, M.D., M.H.Sc., Ph.D., Global Therapeutic Head, Oncology, Johnson & Johnson Innovative Medicine.

“By combining our expertise in innovative medicine and medical devices, Johnson & Johnson is uniquely positioned to transform how we treat certain types of bladder cancer through the first and only intravesical drug releasing system for this disease. We look forward to working with the FDA in review of this application.”

The submission of this innovative intravesical drug releasing system is supported by data from the Phase 2b SunRISe-1 registration study.

Data collected through the second quarter of 2024 and presented at the European Society for Medical Oncology (ESMO) 2024 Congress as a late-breaking oral presentation showed an 83.5 percent complete response (CR) rate and highly durable CRs without the need for reinduction – at a median follow-up of nine months, 82 percent of responders maintained response.

At data cutoff in May 2024, safety and tolerability data presented at ESMO demonstrated a low occurrence of Grade 3 or higher treatment-related adverse events (TRAEs) (9 percent); five patients had TRAEs leading to discontinuation (6 percent) and no treatment-related deaths were reported.

TAR-200 is an investigational intravesical drug releasing system designed to provide sustained local delivery of gemcitabine into the bladder. It is placed into the bladder by a healthcare professional using a co-packaged urinary placement catheter in an outpatient setting in under five minutes and without the need for anesthesia.

In December 2023, the FDA granted Breakthrough Therapy Designation (BTD) to TAR-200 for the treatment of adult patients with BCG-unresponsive HR-NMIBC with CIS who are ineligible for or have elected not to undergo radical cystectomy.

About SunRISe-1, Cohort 2
SunRISe-1 (NCT04640623), Cohort 2, is a randomized, parallel-assignment, open-label Phase 2b clinical study evaluating the safety and efficacy of TAR-200 monotherapy alone for BCG-unresponsive HR-NMIBC carcinoma in situ (CIS) patients who are ineligible for, or elected not to undergo, radical cystectomy.

The primary endpoint for Cohort 2 is CR rate at any time point. Secondary endpoints include duration of response (DOR), overall survival (OS), pharmacokinetics, quality of life, safety and tolerability.

About High-Risk Non-Muscle-Invasive Bladder Cancer
High-risk non-muscle-invasive bladder cancer (HR-NMIBC) is a type of non-invasive bladder cancer that is more likely to recur or spread beyond the lining of the bladder, called the urothelium, and progress to invasive bladder cancer compared to low-risk NMIBC.

HR-NMIBC makes up 15-44 percent of patients with NMIBC and is characterized by a high-grade, large tumor size, presence of multiple tumors, and CIS.

Radical cystectomy is currently recommended for NMIBC patients who fail BCG therapy, with over 90 percent cancer-specific survival if performed before muscle-invasive progression.

Given that NMIBC typically affects older patients, many may be unwilling or unfit to undergo radical cystectomy.

The high rates of recurrence and progression can pose significant morbidity and distress for these patients.”

https://www.jnj.com/media-center/press-releases/new-drug-application-initiated-with-u-s-fda-for-tar-200-the-first-and-only-intravesical-drug-releasing-system-for-patients-with-bcg-unresponsive-high-risk-non-muscle-invasive-bladder-cancer

What is Decentralized clinical trials?:Benefits and challenges of implementing DCT

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What is Decentralized clinical trials?:Benefits and challenges of implementing DCT

Decentralized clinical trials (DCTs) are an innovative approach to conducting clinical research, where some or all trial-related activities occur at locations other than traditional clinical trial sites. These locations can include participants’ homes, local healthcare facilities, or nearby laboratories.

Key Features of DCTs:

Benefits of DCT:

DCTs are gaining momentum, especially after the COVID-19 pandemic highlighted the need for flexible and remote healthcare solutions. They represent a significant shift towards more patient-centric and efficient clinical research.

Example of a successful decentralized clinical trial

One notable example of a successful decentralized clinical trial is the ACTIV-6 trial, which was conducted to test outpatient treatments for COVID-19. This trial was part of the National Center for Advancing Translational Sciences (NCATS) program and utilized decentralized methods to reach a broader participant base.

The ACTIV-6 trial leveraged digital technologies and community partnerships to conduct the study beyond traditional clinical sites. This approach allowed for more efficient and inclusive participant recruitment, especially from underrepresented communities. 

The trial demonstrated that decentralized clinical trials (DCTs) could be more efficient, effective, and equitable, bringing treatments to a wider population more quickly.

Lets discuss few more examples of successful decentralized clinical trials (DCTs):

  1. The REMOTE Trial: This was one of the first fully decentralized trials conducted by Pfizer. It aimed to evaluate the efficacy and safety of a drug for overactive bladder. The trial utilized digital tools for patient recruitment, consent, and data collection, significantly reducing the need for physical site visits.
  2. The CHIEF-HF Trial: Conducted by Bristol Myers Squibb, this trial focused on heart failure patients. It used a decentralized approach to monitor patients remotely through wearable devices and mobile apps, allowing for continuous data collection and improved patient engagement.
  3. The VERKKO Study: This study, conducted by Sanofi, aimed to evaluate a new diabetes treatment. It was notable for its use of telemedicine and home health visits, which enabled participation from a diverse patient population across different geographic locations3.

These examples highlight the potential of DCTs to enhance patient recruitment, improve data quality, and make clinical trials more accessible and efficient.

What are the challenges of implementing DCTs?

Implementing decentralized clinical trials (DCTs) comes with several challenges:

  1. Technology Barriers:
    • Access and Literacy: Not all participants have access to the necessary technology or the digital literacy to use it effectively.
    • Data Security: Ensuring the security and privacy of patient data collected remotely is crucial and can be complex.
  2. Regulatory and Compliance Issues:
    • Regulatory Variability: Different regions have varying regulations, making it challenging to standardize DCTs globally.
    • Compliance: Ensuring that all aspects of the trial comply with regulatory requirements can be more difficult when activities are decentralized.
  3. Data Management:
    • Data Integration: Combining data from various sources and ensuring its accuracy and consistency can be challenging.
  4. Participant Engagement:
    • Retention: Keeping participants engaged and motivated throughout the trial can be harder without face-to-face interactions.
    • Support: Providing adequate support to participants remotely, especially for those with complex needs, can be difficult.
  5. Operational Challenges:
    • Logistics: Coordinating the delivery of trial materials and medications to participants’ homes can be logistically complex.
    • Training: Ensuring that both participants and local healthcare providers are adequately trained to perform trial-related activities.Despite these challenges, the benefits of DCTs, such as increased accessibility and convenience, make them a promising approach for the future of clinical research. Continuous advancements in technology and regulatory frameworks are helping to address these challenges.
  1. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/conducting-clinical-trials-decentralized-elements
  2. https://www.fda.gov/drugs/cder-conversations/evolving-role-decentralized-clinical-trials-and-digital-health-technologies
  3. https://ncats.nih.gov/news-events/news/ncats-report-highlights-decentralized-clinical-trials-challenges-opportunities
  4. https://www.obviohealth.com/resources/decentralized-clinical-trials-a-comprehensive-synopsis
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