Baird analyst Madhu Kumar raised the price target on Rocket Pharmaceuticals (NASDAQ: RCKT) to $41.00 (from $30.00).
Friday, December 6, 2019
UCB’s bimekizumab tops Humira in late-stage psoriasis study
UCB SA (OTCPK:UCBJF) announces positive results from a Phase 3 clinical trial, BE SURE, comparing bimekizumab, an IL-17A and IL-17F inhibitor, to AbbVie’s (ABBV +0.5%) TNF inhibitor Humira (adalimumab) in adults with moderate-to-severe plaque psoriasis.
The study met the co-primary endpoints at week 16, demonstrating bimekizumab’s superiority as measured by the proportion of patients achieving PASI 90 (90% improvement in psoriasis index score) and IGA 0/1 (clear or almost clear skin).
All key secondary endpoints were also met.
Detailed data will be submitted for presentation at a medical conference in 2020.
Topline results from studies in psoriatic arthritis, ankylosing spondylitis and non-radiographic axial spondyloarthritis should be available by late 2021.
FDA OKs Amgen’s Remicade biosimilar
No FDA panel review for Bristol-Myers’ luspatercept
Bristol-Myers Squibb (BMY +2.2%) and collaboration partner Acceleron Pharma (XLRN -1.3%) announce that, following a meeting with the FDA two days ago, there will be no advisory committee review of Celgene’s marketing application seeking approval of Reblozyl (luspatercept-aamt) for the treatment of adult patients with very low-to-intermediate-risk myelodysplastic syndromes-associated anemia who have ring sideroblasts and require red blood cell transfusions. The review was scheduled for Wednesday, December 18.
The agency’s April 4, 2020 action date remains as is.
FerGene’s Bladder Cancer Phase III Trial Hits Its Mark
FerGene, a gene therapy company formed by Ferring Pharmaceuticals and Blackstone Life Sciences, reported that its Phase III trial of nadofaragene firadenovec (rAd-IFN/Syn3) met its primary endpoint. The trial is evaluating the gene therapy for the treatment of high-grade, Bacillus Calmette-Guerin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC).
FKD Therapies Oy (FKD) is a specialist gene therapy company located in Kuopio, Finland. The company was originally founded to develop adenovirus-mediated interferon alfa-2b. It has led the overall development of nadofaragene firadenovec via manufacturing at FinVector Oy, late-stage clinical trials, and the current Biologics License Application (BLA).
FerGene was created to possibly commercialize the therapy in the U.S. and to advance global clinical development. New York-based investment firm Blackstone Life Sciences is investing $400 million in the company and Ferring, a Swiss company, is investing up to $170 million. Ferring will launch and commercialize the therapy outside of the U.S.
NMIBC is an early type of bladder cancer. It is present in the outside layer of the bladder, meaning it hasn’t penetrated deeper into the bladder or spread to other parts of the body. There are about 80,000 new cases of bladder cancer in the U.S. annually, with about 70% being NMIBC. The recommended treatment for NMIBC is intravesical BCG—meaning the therapy is placed directly into the bladder–but between 30% and 50% of those high-grade cases will recur. The outcome for BCG unresponsive patients is poor, with removal of the bladder often the next treatment.
The Phase III trial of 157 patients in the U.S. hit the primary endpoint, with 53% of patients whose bladder cancer was confined to the superficial layer achieving a clinical response (CR) at three months and 24% continuing to show a CR at 12 months. It also showed broad efficacy in the population with a 73% high-grade recurrence-free (HGRF) survival in patients with papillary disease at three months and 44% HGRF survival as 12 months.
“Currently, patients living with high-grade NMIBC who are unresponsive to BCG have few treatment options and often face bleak outcomes, including complete bladder removal, known as cystectomy,” said Colin PN Dinney, professor and chairman of the Department of Urology at The University of Texas MD Anderson Cancer Center. “Cystectomy is a complex and life-altering surgical procedure for patients, so these positive results from the Phase III trial of nadofaragene firadenovec are highly promising for patients. It would be gratifying to provide an alternative that addresses the critical unmet need for effective second-line therapy for patients facing radical cystectomy.”
The most common adverse events were fatigue, bladder spasm and discharge around the catheter, urinary urgency, blood in the urine, chills, fever, headache, painful urination, urinary tract infection, and diarrhea.
“As a practicing urologist and trial investigator, it’s encouraging to see these types of efficacy and safety results in patients with high-grade NMIBC, an area that’s been in need of new innovative treatment options for more than 20 years,” said Neal Shore, medical director, Carolina Urologic Research Center. “These robust clinical results further demonstrate the potential of nadofaragene firadenovec as a valuable treatment option for NMIBC patients.”
The FDA has already validated FKD’s BLA and granted it Priority Review.
Prescription Drug Prices Dip Slightly in the U.S., the First Time in 45 Years
Prescription drug prices dropped 1% last year, the first time such a drop has occurred in the United States in 45 years. The drop was driven primarily by a greater reliance on generic drugs and much slower increases in branded drugs, according to a government study.
The pricing study was conducted by the U.S. Centers for Medicare and Medicare Services. It showed the decline in retail prices in prescription drugs, a feat that has not been seen since 1973 when prices dropped .2%, The New York Times reported. Despite the 1% decrease in retail prices, total expenditures on prescription drugs rose 2.5% to $355 billion in 2018, the Washington Post added. In all, health spending climbed to $3.6 trillion in 2018, an increase of 4.6%. That spending accounts for nearly 18% of the U.S. economy, the CMS report showed. In its analysis, a breakdown of those healthcare expenses amounts to $11,172 per each person in the United States, the Post noted.
In its analysis, the Times said spending on prescription drugs at pharmacies (not including the costs of medications dispensed in hospitals) accounted for 9% of that $3.6 trillion in health spending.
The CMS report was released as lawmakers on Capitol Hill and the White House have been developing different approaches to curb the price of prescription drugs in the United States. The bulk of the legislation pending in the House and Senate, is aimed at drugs covered by government-funded programs such as Medicare. Meanwhile, the CMS report notes that more people are going without insurance. The report said there were about 1 million more additional people without health insurance in the United States in 2018, about the same increase as in 2017, the Times reported. The cost per person for private health insurance rose last year by an average of 6.7 percent, the most rapid increase since 2004, the Post added.
House Speaker Nancy Pelosi plans to move forward with her drug pricing plan for Medicare next week. The Speaker’s proposal would tie prices that Medicare pays to an international pricing index and would also penalize drugmakers for not participating in the program. Pelosi’s plan would also allow private insurers to participate in the Medicare pricing structure. It is expected that a floor vote could come as early as next week on the Speaker’s plan.
As could be expected, the pharmaceutical industry and the White House have come out in opposition to the House-led plan. A new report from STAT News also shows that more than 100 chief executive officers of emerging biotech companies are also opposed to the Speaker’s plan. The White House and the pharmaceutical industry have warned of dire consequences for the industry if this legislation is passed. Both have suggested that the Speaker’s plan would stifle innovation and cause an overall loss of new drugs being developed by companies due to the lack of available funding for R&D that results from profits off of drug sales. Instead, the White House, which at one time supported tying the prices for Medicare drugs to an international pricing index through a “favored nations clause,” is now backing a more bipartisan plan in the U.S. Senate that calls for drug companies to pay rebates to Medicare if they raise prices higher than the inflation rate.
Bayer agrees to postpone more glyphosate lawsuits for settlement talks
Germany’s Bayer has agreed with plaintiffs to postpone its next two U.S. lawsuits over the alleged cancer-causing effects of its glyphosate-based weed killers to allow more time for talks on a settlement.
The company, which is facing 42,700 U.S. plaintiffs, is widely expected to eventually buy itself out of the litigation, with analysts currently estimating the size of a future settlement at $8-$12 billion.
Bayer agreed with the plaintiff to delay for about six months a case in the California Superior Court for Lake County scheduled for Jan. 15, a company spokesman said in a written statement.
A second case due to begin on Jan. 21 in the California Superior Court for Alameda County would also be postponed until a date yet to be determined.
Bayer said the postponements would provide more time for it and representatives of the plaintiffs to “engage constructively in the mediation process”.
The company, which acquired the weed killer brands last year as part of a $63 billion takeover of Monsanto, has ruled out pulling the products from the U.S. market, citing regulators and extensive research which have found glyphosate to be safe.
It will early next year appeal to reverse the first three U.S. court rulings that awarded tens of millions of dollars to each plaintiff.
Other trials initially scheduled for this year have already been postponed.
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