5 Headlines from the Pharmacy World This Week

In case you missed it …

1. A promising future for fetal and congenital genetic diseases¹

Congenital disease is estimated to claim 300,000 infant deaths within the first month of life every year worldwide. It also contributes to childhood illness and long-term disability. In the past decade, technology has rapidly advanced, allowing us to perform prenatal screening techniques to make early diagnosis with the hopes of treating the disease before birth. However, there are very few, if any, treatments available.

Earlier this week, researchers at the Rosalind Franklin University of Medicine and Science and Oregon Health and Science University developed a method to treat genetic diseases in utero via an injection into the amniotic fluid. The researchers injected short sequences of nucleic acids called antisense oligonucleotides designed to bind to specific genes. This study was performed on mice, and the mice demonstrated alterations in their gene expression for up to a month after birth.

It poses far less of a risk when a drug is delivered to the amniotic fluid instead of straight to the fetus. This also shows a promising future for gene therapy and therapeutics for fetal and congenital genetic diseases.

2. Ebola vaccine gains more funding²

Merck and NewLink Genetics secured an additional $76 million grant from the Biomedical Advanced Research and Development Authority (BARDA). The grant allocates $24.8 million toward manufacturing and clinical trials, while the other $51 million could be awarded further down the road.

Merck’s candidate vaccine, rVSV-ZEBOV (V920) won FDA and EMA milestones this summer and hopes to submit it for approval by the end of 2017. Its phase 3 ring study showed 100% protection of the subjects. It is currently a race toward the finishing line. Earlier this year, GlaxoSmithKline started its phase 3 clinical trials in Liberia with its own Ebola candidate vaccine.

3. Another essential medication sees an enormous price increase³

It seems like price hikes for essential medication are a complete norm these days. First, it was Daraprim, then the EpiPen, and now Calcium EDTA. Calcium EDTA is a drug used to treat lead poisoning. Before Valeant acquired this drug in 2013, the price for a package of vials was $950. By December 2014, this medication saw a price increase of more than 2,700% to $26,927. This has created a toxic outrage in poison control centers and hospitals.

4. Xarelto (rivaroxaban) still safe to take, says FDA⁴

Earlier this week, the FDA determined that rivaroxaban is still safe and effective for patients with non-valvular atrial fibrillation. Rivaroxaban has come under extreme scrutiny lately because in their phase 3 clinical trial, ROCKET-AF, warfarin therapy used the Alere Inc. INRatio device, which has been recalled due to its inaccurate results. The FDA concluded the faulty machine had minimal effects on strokes and bleeding data.

5. Keytruda (pembrolizumab): a potential blockbuster?^5,6,7

Merck just announced Keytruda’s results for the treatment of bladder and non—small-cell lung cancer (NSCLC) at the European Society for Medical Oncology (ESMO) 2016 Congress this past weekend.

In the KEYNOTE-052 phase 2 study, patients were treated for bladder cancer who were ineligible for cisplatin chemotherapy. 24% of patients responded to therapy and 6% of patients experienced complete remission.

In the KEYNOTE-024 phase 3 study, Keytruda evaluated squamous and non-squamous NSCLC patients whose tumors expressed high levels of PD-L1. Keytruda reduced the risk of disease progression or death by 50% and the risk of death by 40% among patients expressing PD-L1 levels of 50% or greater. Also, Keytruda demonstrated that its median progression-free survival time was 10.3 months compared to 6 months for those only on chemotherapy.

In the KEYNOTE-021 phase 3 study, it included patients with metastatic non-squamous NSCLC regardless of PD-L1 expression level. Patients on Keytruda and chemotherapy (carboplatin and pemetrexed) achieved a 55% response rate compared to 29% for chemotherapy alone and reduced the risk of disease progression or death by 47%. To date, Keytruda is the only anti-PD-1 therapy to exhibit superior efficacy in combination with chemotherapy compared to chemotherapy alone in patients receiving first-line treatment.

These NSCLC results are extremely promising and a huge win over Bristol-Myers Squibb’s Opdivo (nivolumab). Opdivo was thought to take over NSCLC market due to its efficacy and lack of PD-L1 testing. However, Opdivo failed to deliver and was unable demonstrate superiority over platinum-based doublet chemotherapy. Opdivo’s median progression-free survival (4.2 months vs 5.9 months) and overall survival (14.4 months vs 13.2 months) Opdivo’s median progression-free survival failed to beat out chemotherapy (4.2 months vs 5.9 months) and it didn’t statistically significantly beat out overall survival (14.4 months vs 13.2 months) either.

References

• Masterson J. Study shows potential disease treatment in newborns via drug delivery to amniotic fluid. eurekalert.org/pub_releases/2016-10/rfuo-ssp100516.php.
• Sagonowsky E. Merck partner NewLink grabs up to $76M in Ebola vax funding from feds. fiercepharma.com/vaccines/merck-partnered-newlink-grabs-up-to-76m-ebola-vax-assistance.
• Silverman E. Huge Valeant price hike on lead poisoning drug sparks anger. statnews.com/pharmalot/2016/10/11/valeant-drug-prices-lead-poisoning.
• Reuters. FDA clears Xarelto blood thinner despite faulty trial device. reuters.com/article/us-johnson-johnson-bayer-xarelto-idUSKCN12B2A9.
• Eisele P, et al. Two major studies to be presented at ESMO 2016 Congress Presidential Symposium Demonstrate Potential of Merck’s KEYTRUDA® (pembrolizumab) for the first-line treatment of metastatic non-small cell lung cancer in a broad range of patients. mercknewsroom.com/news-release/oncology-newsroom/two-major-studies-be-presented-esmo-2016-congress-presidential-sympos.
• Eisele P, et al. New KEYTRUDA® (pembrolizumab) data in advanced urothelial cancer demonstrate overall response rate of 24 percent in cisplatin-ineligible patients. mercknewsroom.com/news-release/oncology-newsroom/new-keytruda-pembrolizumab-data-advanced-urothelial-cancer-demonstrat.
• Helfand C. UPDATED: Still no Opdivo benefit in subgroup breakdown of Bristol’s first-line NSCLC flop. fiercepharma.com/pharma/still-no-opdivo-benefit-subgroup-breakdown-bristol-s-first-line-nsclc-flop.