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Monday, July 8, 2019

Bayer’s Xarelto successful in pediatric study

Results from a Phase 3 clinical trial, EINSTEIN-Jr., evaluating a body weight-adjusted 20 mg equivalent dose of Bayer’s (OTCPK:BAYRY) blood thinner XARELTO (rivaroxaban) in children from birth to age 17 for the prevention of recurrent venous thromboembolism (VTE) showed comparable efficacy and safety to studies in adults. The data were presented at the International Society on Thrombosis and Hemostasis Congress in Melbourne.
Only 1.2% (n=4/335) of treated children experienced recurrent VTE compared to 3.0% (n=5/165) in those receiving standard-of-care (SOC) treatment (heparin alone or with a vitamin K antagonist).
The rate of clinically relevant bleeding in the treatment group was 3.0% (n=10/335) versus 1.9% (n=3/158) in the SOC arm.
A marketing application in Europe for the expanded use is next up.

ViiV launches U.S. study on implementing monthly HIV treatment

ViiV Healthcare commences a U.S.-based study, CUSTOMIZE, aimed at identifying and assessing the best ways to implement a once-monthly injectable treatment for HIV infection into clinical practice. The goal is to find the most practical and efficient ways to manage monthly treatment.
In April, ViiV submitted its marketing application to the FDA seeking approval for its once-monthly two-drug HIV regimen of cabotegravir and Janssen’s rilpivirine. The agency’s action date under Priority Review status is December 29.
ViiV Healthcare is the HIV-focused joint venture between GlaxoSmithKline (GSK -0.5%), Pfizer (PFE -1.3%) and Shionogi (OTCPK:SGIOY -3.1%).

NICE shuts down AZ’s Tagrisso—and backs Pfizer rival Vizimpro instead

England’s cost watchdogs won’t get behind AstraZeneca blockbuster Tagrisso in previously untreated patients with EGFR-mutated lung cancer. But in a change of events, they’re instead backing a Pfizer challenger.
Friday, the National Institute for Health and Care Excellence (NICE) deemed Tagrisso too expensive for routine NHS use, dealing a blow to AZ’s sales ambitions. NICE already endorses Boehringer Ingelheim’s Gilotrif, Roche’s Tarceva and AstraZeneca’s own Iressa, it said (PDF), and while Tagrisso has topped both Tarceva and Iressa in trials, there’s no evidence that it can beat out Gilotrif.
While the body sometimes covers a drug under its Cancer Drugs Fund while companies collect more data, NICE shut down that option for the British drugmaker. “It does not have the potential to be cost effective at the price offered,” it said. Tagrisso runs at £5,770 for a pack of 30 tablets, and the confidential discount AZ served up wasn’t enough to sway the gatekeeper.
Meanwhile, though, Pfizer can’t say the same. After an initial rejection, NICE came around to support its Vizimpro on Friday, and it made clear that Pfizer’s price break on the drug was responsible for the about-face.

Vizimpro, new on the scene after picking up its European green light for first-line patients in early April, has only topped Iressa in the clinic, and “an indirect comparison suggests there is no difference between” it and Gilotrif “in terms of how long people live or how long it is before their disease gets worse,” NICE said (PDF) in final draft guidance.
Still, though, “the most plausible cost-effectiveness estimate is within what NICE normally considers an acceptable use of NHS resources,” it wrote.
Pfizer, for its part, was pleased with the decision. “We welcome NICE’s decision,” Olivia Ashman, oncology medical director at Pfizer UK, said in a statement, adding that “Lung cancer remains a challenging condition to treat and we are pleased that clinicians managing patients with EGFR-mutated NSCLC will now have access to this important medicine.”

AstraZeneca, though, likely isn’t too happy with the latest developments—and it wouldn’t be the first time it ran into frustrations with NICE. Back in 2016, it faced a NICE delay on Tagrisso in previously treated patients, and then-U.K. head Lisa Anson said the company was “very disappointed.”
“it takes longer for NICE to agree on the same data set that other markets have had and granted access to,” she griped in an interview with The Telegraph.

Soliton to Announce Cellulite Trial Data for 26-Week Period on July 15

Soliton, Inc., (SOLY) (“Soliton” or the “Company”), a medical device company with a novel and proprietary platform technology licensed from The University of Texas on behalf of the MD Anderson Cancer Center (“MD Anderson”), today announced that it will release the cumulative 26-week patient data from its cellulite proof of concept clinical trial on Monday, July 15, 2019.  As the Company has previously announced, the proof-of-concept clinical trial studied the safety and efficacy of a new version of its acoustic shockwave device for the treatment of moderate to severe cellulite.  The results from the 26-week assessment are being presented at The Aesthetics Show in Las Vegas on July 12, 2019.

Biomarin on with marketing applications for hemophilia A gene therapy

BioMarin Pharmaceutical (NASDAQ:BMRN) expects to file marketing applications in the U.S. and Europe in Q4 seeking approval for gene therapy valoctocogene roxaparvovec (VR) for the treatment of adults with severe hemophilia A.
VR has Breakthrough Therapy status in the U.S. and PRIME status in Europe.
Shares are off 1% premarket, albeit on only 100 shares.

UnitedHealthcare to cover Inspire Medical’s sleep apnea therapy

UnitedHealthcare will provide coverage for Inspire Medical Systems’ (NYSE:INSP), Inspire therapy effective August 1, 2019.
Under its policy, UnitedHealthcare considers Inspire’s FDA approved hypoglossal nerve neurostimulation device to be medically necessary for the treatment of moderate to severe obstructive sleep apnea (OSA) when specified criteria are met.
INSP now have 35 coverage policies, representing approx. 125M members.
Shares are up 7% premarket.

FDA Fast-Tracks Prevail’s PROO1 for Parkinson’s

Prevail Therapeutics (NASDAQ:PRVL) is up 12% premarket on light volume on the heels of the FDA designating gene therapy PR001 for Fast Track review for the treatment of GBA1 mutation-positive Parkinson’s disease patients.
Dosing in a Phase 1/2 study should commence in H2.