Thursday, September 13, 2018
ArQule announces FDA Fast Track Designation for rare disease med
ArQule announced that the FDA has granted Fast Track Designation to miransertib for the treatment of PIK3CA-Related Overgrowth Spectrum, or PROS, a group of related, ultra-rare genetic disorders characterized by excessive tissue growth in various parts of the body. The FDA’s Fast Track program aims to expedite the development and review of drugs which treat serious or life-threatening conditions and have demonstrated the potential to address unmet clinical needs. Miransertib has already been granted Rare Pediatric Disease Designation by the FDA and Orphan Designation by the FDA and European Medicines Agency in the rare overgrowth disease, Proteus syndrome. During the past three years, miransertib has been tested in a Phase 1 NIH/NHGRI-sponsored trial in Proteus Syndrome. More recently, we have been conducting a Phase 1/2 company-sponsored trial in PROS and a single patient use program with select physicians.
https://thefly.com/landingPageNews.php?id=2789809
Humana, Cerner initiated at Barclays
Barclays sees favorable Medicare Advantage outlook, starts Humana at Overweight. Barclays analyst Steve Valiquette initiated coverage of Humana (HUM) with an Overweight rating and $370 price target. The fundamental Medicare Advantage outlook is the strongest it has been since pre-Affordable Care Act levels “thanks to a number of industry tailwinds brought about by the Republican administration,” Valiquette tells investors in a research note. These include higher Medicare reimbursement, corporate tax reform, and 2019 health insurance fund suspension, the analyst says. He believes Humana will be able to deliver on its low-to-mid-teens annual adjusted earnings target through at least 2021. Valiquette this morning also initiated coverage of Cerner with an Equal Weight rating and $70 price target. The company’s 7%-12% revenue growth outlook relies on the ramp-up of newer products, the analyst contends.
https://thefly.com/landingPageNews.php?id=2789829
Incyte announces eczema med Phase 2b trial met primary endpoint
Incyte Corporation announced positive results from its randomized, dose-ranging, vehicle- and active-controlled Phase 2b study evaluating ruxolitinib cream in patients with atopic dermatitis who are candidates for topical therapy. The study, part of the True-AD clinical trial program, met its primary endpoint, demonstrating that ruxolitinib cream 1.5% administered twice daily significantly improved Eczema Area and Severity Index scores – a measurement of the extent and severity of AD – from baseline versus vehicle control at Week 4. Additionally, treatment with ruxolitinib cream 1.5% BID resulted in a rapid and sustained reduction in itch versus vehicle, a key secondary endpoint. These results were shared in an oral presentation today at the 27th European Academy of Dermatology and Venerology Congress in Paris, France. Key study results included: Significantly improved EASI score in the ruxolitinib cream 1.5% BID arm versus vehicle at Week 4, the primary endpoint, and improvement in EASI score versus the active control, triamcinolone 0.1% cream, at Week 4, a secondary endpoint. Significantly improved EASI scores in the ruxolitinib cream 1.5% BID arm versus vehicle at Weeks 2 and 8. Significantly greater changes in EASI score in the once daily ruxolitinib cream 1.5% and 0.5% arms versus vehicle at Week 4. Significantly more Investigator’s Global Assessment responders – a measure of disease severity – in the ruxolitinib cream 1.5% BID arm versus vehicle at Week 4, and greater IGA response rates across other ruxolitinib arms versus vehicle. Rapid and sustained reductions in itch numerical rating scale score observed as early as within two days from the initiation of therapy, and a more pronounced reduction in itch with ruxolitinib cream 1.5% BID and QD than with triamcinolone cream 0.1% BID. Ruxolitinib cream was well-tolerated at all dosage strengths and was not associated with clinically-significant application site reactions. All treatment-related adverse events were Grade 1 or Grade 2 in severity. Ruxolitinib cream is the first JAK1/JAK2 inhibitor to exhibit positive results as a topical monotherapy in the AD patient population. Over-activity of the JAK signaling pathway has been shown to drive inflammation involved in the pathogenesis of AD. These data support the planned initiation of a global, pivotal Phase 3 program, for which preparations are already underway.
https://thefly.com/landingPageNews.php?id=2789857
Gilead Sciences, Precision partner on genome-editing therapies for hep B
Gilead Sciences Inc. (NASDAQ:GILD) and Precision BioSciences Inc. (Durham, N.C.) partnered to develop therapies that eliminate the HBV virus in vivo, using Precision’s genome editing platform ARCUS.
Current HBV treatments suppress HBV viral replication without clearing the virus, according to the partners.
ARCUS generates synthetic nucleases that can insert, edit or remove DNA from genomes. ARCUS nucleases that target HBV covalently closed circular DNA (cccDNA) — DNA that enables HBV replication when patients stop treatment — have shown promising activity in human liver cells in preclinical studies at Gilead, the partners said.
Precision VP of Business Development Michael Dombeck told BioCentury Gilead’s previous “hands-on experience” with ARCUS was not part of a formal collaboration, and none of those data have been published.
Under the deal, Precision will be eligible to receive milestone payments of up to $445 million, plus tiered royalties up to the mid-teens. Gilead will fund all R&D, with Precision leading preclinical studies and Gilead taking on clinical development and commercialization. Dombeck declined to disclose any further financial terms or when it expects the lead partnered program to begin clinical development.
Takeda weighs sale of Shire’s eye care business to cut debt
Japan’s Takeda Pharmaceutical is considering selling Shire Plc eye care business once it closes its $62 billion (47 billion pounds) purchase of the London-listed drugmaker, as it looks to cut the debt raised to fund the deal, Bloomberg reported on Thursday.
Takeda is weighing the sale of Shire’s Xiidra drug which treats dry eye, as well as its Natpara medicine used to control low blood calcium levels, the report said https://www.bloomberg.com/news/articles/2018-09-13/takeda-is-said-to-weigh-sale-of-shire-eye-care-drug-to-cut-debt, citing people familiar with the matter.
The sale could help Takeda raise between $4 billion and $5 billion, Bloomberg reported.
Takeda agreed to buy Shire in May, in what would be the largest overseas purchase by a Japanese company.
New Flu Season, New Flu Drug from Genentech
Last year’s influenza season in the U.S. had record-breaking hospitalization rates. The Centers for Disease Control and Prevention (CDC) reported 30,429 laboratory-confirmed flu-associated hospitalizations from October 1, 2018 through April 30, 2018. With any luck, this year’s flu season won’t be as bad.
A lot of industry observers are looking at a new flu drug, baloxavir marboxil, for which clinical trial data was just published. STAT News notes, “The drug … cut the time people were sick with flu symptoms by a little over a day. And it dramatically reduced the amount of viruses that people with infections had in their upper respiratory tracts, suggesting they might be less likely to infect others through coughs and sneezes.”
But one drawback was baloxavir marboxil didn’t make patients feel better faster than Tamiflu (oseltamivir). And the articles, published in the New England Journal of Medicine, indicated that viruses seemed to develop resistance to it quickly. It was of enough concern that the New England Journal of Medicine published an editorial by Timothy M. Yueki, associate clinical professor of Pediatrics with the University of California, San Francisco (UCSF), emphasizing the problem.
Baloxavir was developed by Japan’s Shionogi & Co., which is sold in Japan as Xofuza. Roche,which markets Tamiflu, acquired most of the global rights to baloxavir, and Genentech, Roche’s subsidiary, is developing it for the U.S. market. Shionogi still holds the rights to the drug in Japan and Taiwan.
Baloxavir is taken orally in one or two pills, depending on body weight. In Japan, STAT reports, it costs about $43.50 for the one-pill dose. It is the first drug in a new class called endonuclease inhibitors. They prevent viruses from replication.
“It looks like a very exciting drug,” Trish Perl, chief of infectious diseases at the University of Texas Southwestern Medical Center in Dallas, told STAT. “It’s a new [drug] class. Looks like it’s very efficacious. And it’s interesting because it has implications for patient care as well as for pandemic planning.”
The other two classes of flu drugs on the market are adamantine, which hasn’t been recommended for use in a long time because viruses have developed resistance, and endonuclease inhibitors. Despite the apparent enthusiasm, the fact that the patients didn’t feel better sooner than on Tamiflu is unexpected. Frederick Hayden of the University of VirginiaSchool of Medicine, who was the lead author of the studies, told STAT he was surprised by that fact, suggesting those symptoms, fever, aches, malaise, may be a result of the immune system response rather than the infection itself.
STAT writes, “The drug acts quickly, but must be taken quickly as well for maximum effect. The studies — a Phase II and a Phase III, published together — found that people who took the drug within 24 hours of the start of symptoms felt better, on average, 33 hours sooner than people whose flu was untreated. But for those who started the drug later, their recovery was sped up by only about 13 hours.”
That can be tough, particularly if it’s used in response to a global pandemic, such as was observed last season.
In general, it is probably better to get a flu vaccine to prevent the flu, rather than try to deal with it after you’ve already caught it. Particularly since baloxavir is not yet available in the U.S.
An article published in USA Today on September 5 reported that the American Academy of Pediatrics was recommending that children ages 6 months and older should receive a flu vaccine as soon as possible—no later than the end of October. And the CDC on September 7 recommended everyone six months and older receive a seasonal flu vaccine by the end of October. It takes about two weeks to develop antibodies against the flu after receiving the vaccine, so the earlier the better.
Wednesday, September 12, 2018
Mich. threatens to repeal Medicaid expansion if work requirements not OKd
- Michigan will roll back expansion of its Medicaid program unless it is allowed to institute controversial work requirements for the program, according to an application Gov. Rick Snyder, a Republican, sent to HHS this week.
- Under the proposal, beneficiaries will be required to either work, volunteer, attend job training or engage in other health-promoting activities for a minimum of 80 hours a month in order to keep their coverage, along with tracking and logging their progress in monthly verifications to prove they are complying with the requirements. The proposal includes 12 exemptions for populations such as pregnant women and those who are caregivers for children under 6.
- Approval for the extension request is being sought for early 2019, with a slated program introduction between six months to a year later. To date, four states (Arkansas, Indiana, Kentucky and New Hampshire) are imposing work requirements for Medicaid and seven (Maine, Kansas, Arizona, Mississippi, Wisconsin, Utah and Ohio) have submitted waiver applications.
The application is likely to be approved, as CMS has made work requirement implementation a priority since the beginning of this year and the policy has quickly become a linchpin for the current administration. In a 10-page letter to state Medicaid directors, the agency urged them to test programs that include work requirements as a condition for eligibility and, so far, has approved them in four states.
Michigan’s proposal impacts beneficiaries with income between 100% and 133% of the federal poverty level who have been eligible for or enrolled in Medicaid for four years. Those who do not meet the program’s healthy behavior, work or cost-sharing requirements will be notified 60 days before the end of their fourth year on Medicaid that their coverage will be terminated until they comply with the new strictures.
The state expanded Medicaid in 2014 under the Healthy Michigan Plan (HMP), which granted coverage to more than 1 million low-income citizens and currently enrolls roughly 655,000 people. The introduction of work requirements is meant to “empower individuals” to improve their health by promoting “accountability, self-sufficiency, and independence from public assistance,” according to the application, which also touts the positive correlation between income and health.
The petition similarly posits the new stipulations will help beneficiaries realize the mental and physical health benefits associated with gainful employment, along with providing future opportunities to obtain insurance through an employer or the federal marketplace.
Yet the many critics of work requirements point out that most Medicaid recipients who can work are already doing so, and that instituting and tracking work requirements will heap additional administrative burden onto an already-taxed system. Two recent JAMA studies found evidence backing up their opposition.
The first found that, among the 11 states with submitted waiver applications, only 3.9% to 29.2% of Medicaid-eligible individuals were subject to proposed work requirements. In that subset, only 0.3% to 5.4% were not meeting those requirements, bringing up the question of whether the cost of implementation is justified by the work requirements’ “narrow projected reach.”
The second study, also published in JAMA and building off the former’s findings, estimated changes in Medicaid enrollment and spending if work requirements were applied on a country-wide scale. If that were to happen, 2.1 million beneficiaries would be at risk of losing coverage, representing 2.8% of current Medicaid enrollees and accounting for a mere 0.7% of Medicaid spending. Yet, the study’s authors pointed out, these minimal savings would “likely come at substantial cost in terms of human health” due to a potential spillover effect, wherein exempt Medicaid enrollees lose their coverage because they’re unable to comply with stringent documentation requirements for reporting their work hours or reason for exemption as frequently as every month.
The murky outcomes of the policies caused a federal judge to invalidate Kentucky’s work requirements in late June, calling their institution “arbitrary and capricious.” Kentucky was the first state to receive approval to test work requirements. U.S. District Judge James Boasberg wrote in his ruling that the government never adequately considered whether the requirements “would in fact help the state furnish medical assistance to its citizens,” a setback to the administration’s aim to link benefits to employment.
In response, Kentucky halted non-emergency medical transportation, along with dental and vision coverage for Medicaid patients, claiming it could not afford to pay for those programs if its waiver was not approved. Following pushback from social advocacy groups, the state reluctantly reinstituted the benefits.
Arkansas, which started kicking thousands of people off Medicaid for failing to comply in July, is currently undergoing similar litigation. Eight more states are waiting on the outcome of their waiver application, including new entrant Michigan.
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