Denise Myshko

Senior vice president at CVS Caremark

Researchers say that a deficiency in the brain of the hormone leptin is as much a part of the cascade that can lead to diabetic ketoacidosis as a lack of insulin is. This opens the potential for the development of new therapies for patients with Type 1 diabetes that target the brain.

Modeyso is the first drug to treat patients with diffuse midline glioma with an H3 K27M mutation, an ultra-rare and aggressive brain tumor.

Ixchiq now has a warning about its use among elderly people with multiple underlying health conditions after postmarketing reports indicated there is a risk of neurologic and cardiac events in people over the age of 60.

In a video interview with MHE editors, Nathan Downhour, Pharm.D., vice president of strategic pharmacy solutions at Prime Therapeutics, said a shift is already underway in the pharmacy benefit market in terms of becoming more transparent and sustainable across the industry.

Senior vice president of strategic pharmacy solutions, Prime Therapeutics

Founder, chairman and CEO, LucyRx

Farxiga, along with insulin and monitoring for diabetic ketoacidosis, shows promise for kidney and glucose control in youth with Type 1 diabetes.

Two presentations discussed the significant care disruptions when there are copay assistance shortages, with patients forgoing effective treatments and turning to lower-cost alternatives with worsening outcomes.

A new analysis finds that prior authorization for anti-VEGF therapies increases societal costs despite minimal insurer savings, burdening patients, workplaces, and providers.

Ixo-vec reduced anti-VEGF injection frequency and was well tolerated in patients with wet AMD. A phase 3 pivotal trial comparing it with Eylea 2 mg is under way.

IRX-101, a new antiseptic tested in patients receiving eye injections for macular degeneration, resulted in less pain and fewer adverse events than povidone-iodine.

In a study in mice, a wireless implant was able to release glucagon as an emergency treatment for hypoglycemia, a serious complication of Type 1 diabetes.

Anti-VEGF and anti-complement therapies work on distinct mechanisms in age-related macular degeneration, and both may be required to treat patients with advanced disease or complex cases.

PER-001, delivered through a slow-release, dissolvable implant in the vitreous cavity of the eye, is designed to block endothelin signaling to increase ocular blood flow and prevent vision loss.

C3 glomerulopathy (C3G) and immune-complex membranoproliferative glomerulonephritis (IC-MPGN) are two diseases that have a high risk of kidney failure. Empaveli is also approved to treat patients with paroxysmal nocturnal hemoglobinuria.

Sarepta continues to work with regulators to complete the safety label update for Elevidys, and they are discussing an approach for risk mitigation for non-ambulatory patients.

Two PBMs, True Rx Health Strategies and Capital Rx, are using pharmacogenomics — how a person’s DNA affects their response to medications — to reduce the trial-and-error of prescribing medications, saving employers and patients time and money.

Health policy researcher Geoffrey Joyce argues that only delinking compensation from the list price of a drug will lower drug spending.

Last week, an FDA advisory committee against the risk-benefit profile of Blenrep in combination with other therapies. Regulators and reviewers were concerned about the ocular side effects and dosing and tolerability. The new action date is Oct. 23, 2025.

The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene therapy for boys with blue cone monochromacy, which impairs color vision and severely affects visual function.

Sarepta officials said the temporary halt in shipments was done to maintain a productive working relationship with regulators while they address a safety labeling update about the risk of acute liver disease related to Elevidys.

Icotrokinra is a first-in-class targeted oral peptide that blocks interleukin (IL) 23 and its receptor, which plays a key role in the inflammatory response in plaque psoriasis.

Elevidys is a gene therapy approved to treat Duchenne muscular dystrophy. Last month, Sarapta halted sales of the therapy for non-ambulatory patients.

In early research, OCU410ST, a gene therapy for Stargardt disease, demonstrated slowed lesion growth by 48% at the 12-month follow-up in evaluable treated eyes.

The combination of RPI, a genetically engineered herpes simplex virus type 1, and Opdivo shrank both injected and non-injected tumors by 30%.

Following in the footsteps of other pharmaceutical companies, Bristol Myers Squibb and Pfizer are offering Eliquis for cash-paying patients at a discount of 40% off the list price.

A class of drugs, MDM2 inhibitors, being studied as cancer therapeutics may accidentally damage the protective barriers in the brain and eyes, finds new research in mice.

Despite the recent approval of lenacapavir as a twice yearly PrEP, there is still a need for choice in HIV prevention, argue speakers at a session at the International AIDS Society meeting.

Global health leaders and advocates challenged attendees of IAS to support the work of the partnerships that are creating patient-focused innovations for HIV prevention and treatment.