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Monday, April 30, 2018

Pill for breast cancer diagnosis may outperform mammograms

As many as one in three women treated for breast cancer undergo unnecessary procedures, but a new method for diagnosing it could do a better job distinguishing between benign and aggressive tumors.
Researchers at the University of Michigan are developing a pill that makes tumors light up when exposed to infrared light, and they have demonstrated that the concept works in mice.
Mammography is an imprecise tool. About a third of  treated with surgery or chemotherapy have tumors that are benign or so slow-growing that they would never have become life-threatening, according to a study out of Denmark last year. In other women, dense breast tissue hides the presence of lumps and results in deaths from treatable cancers. All that, and mammograms are notoriously uncomfortable.
“We overspend $4 billion per year on the diagnosis and treatment of cancers that women would never die from,” said Greg Thurber, U-M assistant professor of chemical engineering and biomedical engineering, who led the team. “If we go to , we can see which tumors need to be treated.”
The move could also catch cancers that would have gone undetected. Thurber’s team uses a dye that responds to infrared light to tag a molecule commonly found on tumor cells, in the blood vessels that feed tumors and in inflamed tissue. By providing specific information on the types of molecules on the surface of the , physicians can better distinguish a malignant  from a benign tumor.
Compared to visible light, infrared light penetrates the body easily—it can get to all depths of the breast without an X-ray’s tiny risk of disrupting DNA and seeding a new tumor. Using a dye delivered orally rather than directly into a vein also improves the safety of screening, as a few patients in 10,000 can have severe reactions to intravenous dyes. These small risks turn out to be significant when tens of millions of women are screened every year in the U.S. alone.
But it’s not easy to design a pill that can carry the dye to the tumor.
“To get a molecule absorbed into the bloodstream, it needs to be small and greasy. But an imaging agent needs to be larger and water-soluble. So you need exact opposite properties,” Thurber said.
Fortunately, they weren’t the only people looking for a molecule that could get from the digestive system to a . The pharmaceutical company Merck was working on a new treatment for cancer and related diseases. They got as far as phase II clinical trials demonstrating its safety, but unfortunately, it wasn’t effective.
“It’s actually based on a failed drug,” Thurber said. “It binds to the target, but it doesn’t do anything, which makes it perfect for imaging.”
The targeting molecule has already been shown to make it through the stomach unscathed, and the liver also gives it a pass, so it can travel through the bloodstream. The team attached a molecule that fluoresces when it is struck with  to this drug. Then, they gave the drug to mice that had breast cancer, and they saw the tumors  up.
The research is described in a study in the journal Molecular Pharmaceutics, titled, “Oral administration and detection of a near-infrared molecular  in an orthotopic mouse model for  screening.”
More information: Sumit Bhatnagar et al, Oral Administration and Detection of a Near-Infrared Molecular Imaging Agent in an Orthotopic Mouse Model for Breast Cancer Screening, Molecular Pharmaceutics (2018). DOI: 10.1021/acs.molpharmaceut.7b00994

American Well Moves Into Hospital-Based Telehealth With Avizia Deal

April 30, 2018
American Well, a national provider of telehealth services, said it has agreed to buy Avizia , which provides virtual access to doctors inside of hospitals and health systems across the country.
The deal, announced at the American Telemedicine Association meeting in Chicago, will help American Well move from treating routine maladies like pink eye and conditions that help patients avoid a doctor’s office visit to more tertiary services, offering a menu of services from partner physicians and hospitals around the world. Financial terms of the deal weren’t disclosed, but the privately held companies said it should close late in the second quarter of this year.
The companies said the Avizia acquisition will “bring American Well a comprehensive acute care capability , including the best in class hospital-based cart lineup and custom software workflows for more than 40 clinical specialties, including telestroke and tele-behavioral health.”
American Well’s proposed acquisition comes as telehealth services are poised to take off in coming years thanks to the approval this year by Congress of a federal budget that includes expanded access to virtual doctor visits for Americans covered by private Medicare Advantage plans. It could be a huge boon to American Well and rivals like MDLive and Teladoc and an array of startups getting into the business of offering access to physicians and patients via smart phone, tablet or computer. Employers and private insurers like UnitedHealth Group, Anthem and Cigna are already embracing the trend as a way to make healthcare more convenient and avoid costly and unnecessary trips to the emergency room or a more expensive physician’s office.
By adding Avizia, American Well hopes to set itself apart from rivals. American Well said its telehealth options will be further “enhanced” for its clients that include health systems, health insurers, allowing them to “choose one comprehensive single platform solution.”
“American Well helps providers treat patients who are not in the room,” Dr. Ido Schoenberg, chairman and CEO of American Well. “We do that by effectively connecting the four key players in the ecosystem: consumers, providers, payers and innovators. “We share Avizia’s passion and relentless commitment to enabling better care anywhere.”
Avizia’s telehealth platform brings doctors from around the world inside hospitals for consultations with more than “1,300 hospital deployments.” “The company has a significant global presence in over 38 countries, with strong clinical use cases across behavioral health, chronic care, stroke, pediatrics and urgent care at over 70 health systems,” Avizia and American Well said in their joint statement.

Molina cost-cutting offsets first-quarter premium revenue dip

Molina Healthcare’s aggressive restructuring might be paying off: The insurer reported modest earnings in the first quarter of 2018 following a tough close to 2017.
The Long Beach, Calif.-based company reported first-quarter net income of $107 million, compared with $77 million for 2017’s first quarter. The company said that’s partly due to lowered medical and administrative costs, in addition to scaled-back restructuring expenses.
“The financial results that we announced today reflect the progress we are making towards our goal of sustainable margin recovery,” Molina CEO Joe Zubretsky said on a conference call Monday.
Premium revenue dropped 7% in the first quarter of 2018 year-over-year, which Zubretsky said was the direct result of a nearly 60% average price hike across its Affordable Care Act policies. Despite that premium increase, Zubretsky said Molina’s marketplace policies remain competitive; the company still has more than 450,000 marketplace members, a number that exceeds its own forecasts. More members are choosing bronze plans over silver ones, which Zubretsky said is a margin-neutral change.
Cost-cutting efforts spurred a modest drop in Molina’s general and administrative expense ratio, to 7.6% in the first quarter of 2018 from 8.9% at the same time in 2017. The company’s medical-care ratio fell to 86.1% in that time, down from 88.4% in the first quarter of 2017.
Overall expenses dropped 8.2% in the first quarter of 2018 to $4.4 billion, down from $4.8 billion in the first quarter of 2017.
Molina recently eliminated about 100 positions, a move it expects will save more than $10 million per year, Zubretsky said.
“We will continue to restructure inefficient processes and find opportunities for head count reductions, as doing so has a very short payback,” he said.
About a year ago, Molina laid off 10% of its workforce, or about 1,500 employees.
Molina took on $25 million in restructuring costs in the first quarter, far lower than in previous quarters.
The company’s total revenue was down 5.2%, ending the quarter at $4.6 billion, compared with $4.9 billion in the first quarter of 2017.
Medicare and Medicaid revenue were essentially flat during the quarter.
Molina failed to secure Medicaid contracts in Florida this year, and Zubretsky said the insurer plans to challenge that decision.
“While we are somewhat disappointed with this outcome, it does not alter our course,” he said. “We will continue to pursue a major entrenchment in Florida.”
Molina has also submitted Medicaid bids in Texas, Washington and Puerto Rico. Puerto Rico is overhauling its Medicaid model to an island-wide plan instead of multiple plans that compete, a change that will feature minimum medical loss ratios.
“These changes require intense scrutiny on our part,” Zubretsky said.

CMS gives post-acute providers a pay bump, plus a new value-based payment

Post-acute providers could see a boost to Medicare payments starting next year, and even a new value-based payment model, as part of a set of new federal proposals.
In a late-Friday rule dump, the Centers for Medicare & Medicaid Services (CMS) proposed both statutory payment bumps as well as some policy changes that are likely to make providers very happy.
For fiscal 2019, the agency is proposing a $340 million, or 1.8% Medicare reimbursement boost to hospice providers, up from a 1% boost from the previous year.
Skilled nursing facilities will also see an $850 million increase in Medicare payments thanks to a mandated increase by the 2018 Bipartisan Budget Act.
Additionally, inpatient psychiatric facilities and rehabilitation facilities are likely to see similar payment increases from the previous year, with a $50 million and $75 million, or a 0.98% and 0.9% respective increases for fiscal year 2019.
Similar to its hospital payment rule, CMS issued a request for information regarding possible revisions to Conditions of Participation to include electronic data sharing requirements.

Other policy changes proposed by CMS include allowing rehabilitation physicians to conduct meetings remotely and loosening of prescriptive documentation requirements. The agency also proposed reducing quality measure reporting for rehab and psychiatric facilities.

CMS takes another leap into value

In yet another move to reduce provider burden and lower costs, the agency is also planning a new value-based payment arrangement for skilled nursing facilities. The agency proposed a Patient Driven Payment Model, a type of value-based payment arrangement that would tie reimbursements to patient outcomes while also reduce reporting burdens.
Providers and insurers have been asking the agency to make a bigger commitment to value-based arrangements. Lately, private insurers have been launched their own programs as a way to lower costs.
Under the new CMS model, patients will have a greater opportunity to select facilities with services tailored to their needs, giving patients more choice, a major goal of the Trump-era department. The model, slated for October 2019, is intended to replace the Resident Classification System, a case-mix model, which has had a rocky reputation (PDF) with both providers (PDF) and policy advisers (PDF).

“As people face rising healthcare costs in other clinical settings, we need to leverage advances in technology that help to modernize our programs in a way that benefits patients,” CMS administrator Seema Verma said in an accompanying statement.
CMS said the model would reduce administrative spending for nursing facilities by $2 billion over 10 years and would go into effect Oct. 19 if the rule is finalized.
Administrator Seema Verma
We’re taking action in proposed rules to reduce paperwork, while maintaining our focus on patient safety and program integrity by focusing on . See how: 
According to the American Hospital Association (PDF), providers spend $39 billion on administrative costs related to regulatory compliance.
The agency said that when taken together with the recent proposed overhaul of the Meaningful Measures initiative, the proposed policy changes should save providers about four million hours in 2019 and 2020.
The comment period for the proposals ends June 26 and a final decision on the rules is expected by July.

Why insurers are spending billions to acquire physician practices

A couple of giant proposed healthcare mergers have garnered a lot of attention in recent months. People want to know what CVS’ $69 billion deal for Aetna or Cigna’s $52 billion agreement to buy Express Scripts can tell us about the future of U.S. healthcare.
But a clearer picture of how the industry is changing might actually come from a string of smaller, less heralded deals.
Insurers are snapping up physician practices. UnitedHealth Group, the largest U.S. health insurance company, agreed in December to pay $4.9 billion for DaVita Medical Group, whose physicians serve some 1.7 million people a year in nearly 300 clinics. That’s the largest recent deal, but it was at least the fourth acquisition of a medical provider group by the company in 2017.
Overall, 147 transactions last year involved physician practices, according to Bloomberg BNA. More multibillion-dollar acquisitions are to be expected this year.
Why? Insurers, employers and the government—the “payers” in the American health system—realize they need to pay for outcomes rather than procedures. In other words, they’ve come to understand that the fee-for-service model is broken, even if it still accounts for the bulk of the money flowing through the system.
More and more, insurers want to compensate doctors based on what they achieve rather than how many procedures they do. No wonder insurers want to own medical practices. It’s easier to improve healthcare services and rein in costs if you control what gets done in doctors’ offices, not insurance company cubicles. As much as an insurance organization wants to encourage getting a flu shot, for example, it’s doctors and nurses who actually talk to patients and administer shots.

Does this mean that your doctor is soon going to be an insurance company employee? Not necessarily. Most medical offices are too small to be swept up by insurers, which are looking for specific characteristics.
The most attractive targets for acquisition will be:
  1. Physician-led. Cost-control and quality improvement are more likely to happen when practitioners themselves have a stake in the outcome. Groups in which physicians are no longer in control of the operations and outcomes of medical care tend to be less effective. And hospital-led medical groups tend to focus on filling beds—the most expensive way to provide medical care.
  2. Strong in primary care. Primary care is the entry point for most patients and where doctors are focused on the whole patient, not just one condition. It’s a strong platform for any practice’s future growth—and the key to preventing illness rather than just treating it.
  3. Diversified. Covering enough specialties to provide a broad spectrum of patient care is important for patient retention and satisfaction, with access to strong cardiology, endocrinology and gastroenterology being key.
  4. Wired. A medical group must have up-to-date technology to collect and analyze patient data. Higher quality outcomes and lower costs come hand in hand when better data and information are available. Real-time analytics drive better prevention and enable strong physician groups to achieve better population health outcomes.
Driving these transactions isn’t just a sense among insurers that the only way to grow their business is through owning medical practices. There’s also the influence of public policy and government programs—specifically, the growth of Medicare Advantage. MA is an option for Medicare beneficiaries in which an individual signs up for a health plan instead of receiving their care through the fee-for-service delivery system. Medicare Advantage, also known as Medicare Part C, has grown steadily and now accounts for over a third of all enrollees in the government’s retiree healthcare program.

A key provision in MA, introduced in the Affordable Care Act, pays plans on the basis of quality measures. In fact, it’s impossible to run a profitable Part C plan without continually improving population cholesterol levels, glycemic control for diabetic patients, and immunization rates. These are all outcomes driven by clinical care, so a focus on physicians is central to such improvement.
What do patients ultimately want? Lower cost, higher quality and more transparency around their choices. By aligning more directly with physician-directed care, through acquisitions as well as contracts, plans are able to provide just that.
These acquisitions also show the power of partnership between the federal government and private sector in healthcare.
Healthcare always entails a balancing act between public and private interests. In this case, Medicare is on the right track by making it more lucrative for health plans to serve patients’ interests.
Dan Mendelson is president and founder of Avalere Health and a former healthcare policy adviser in the White House Office of Management and Budget.

Gilead, Alphabet's Verily to collaborate

Gilead Sciences (GILD) and Verily Life Sciences, an Alphabet company (GOOG), announced a scientific collaboration using Verily’s Immunoscape platform to identify and better understand the immunological basis of three common and serious inflammatory diseases: rheumatoid arthritis, inflammatory bowel disease and lupus-related diseases. In this first large-scale deployment of Immunoscape, a unique platform for generating immunological data and insights, Verily will analyze biological samples and clinical disease and treatment response data from patients participating in current and future Gilead clinical trials. This three-year collaboration represents the broadest effort to date to interrogate the activity of specific subtypes of immune cells to better understand disease signatures and treatment response, and has the potential to guide future drug discovery and development with the goal of improving outcomes for people living with these diseases. Verily’s state-of-the-art Immunoscape platform combines immunogenomic phenotyping and advanced computational analysis techniques to profile the molecular characteristics of inflammatory diseases at high resolution. Through the collaboration, Gilead will provide clinical data and thousands of immune cell samples from participants before, during and after administration of novel drugs in the company’s ongoing Phase 2 and Phase 3 clinical studies. This effort may lead to important new insights into these inflammatory diseases, including identifying molecular signatures that can help physicians to select a therapy or dosing tailored to a specific subgroup of patients, which could improve treatment results and avoid side effects. The data generated in this collaboration may also enable better characterization of subtypes of inflammatory diseases to help scientists identify new molecular targets leading to new therapies.

Tenet beats, ups outlook

Shares of Tenet Healthcare Corp. THC, -1.36% rose more than 5% late Monday after the company reported first-quarter earnings and sales above Wall Street expectations and increased its outlook for the year. Tenet said it earned $99 million, or 96 cents a share, in the first quarter, versus a net loss of $53 million, or 53 cents a share, in the first quarter of 2017. Revenue reached $4.7 billion, compared with $4.8 billion a year ago. Analysts polled by FactSet had expected earnings of 25 cents a share on sales of $4.57 billion for the quarter. Tenet said it expects revenue between $17.9 billion and $18.3 billion for 2018. Analysts surveyed by FactSet expect 2018 sales around $18.05 billion. Tenet shares ended the regular session down 1.4%.

Nutrisystem beats, ups outlook

Nutrisystem (NASDAQ: NTRI) reported Q1 EPS of $0.09, $0.03 better than the analyst estimate of $0.06. Revenue for the quarter came in at $211 million versus the consensus estimate of $207.3 million.
Nutrisystem sees Q2 2018 revenue of $186-191 million, versus the consensus of $190.5 million.
Nutrisystem raises FY2018 EPS to $2.04-$2.14, versus the consensus of $2.04. Nutrisystem raises FY2018 revenue of $693-708 million, versus the consensus of $697.43 million.

Inogen target upped to $190 by Piper

Inogen price target raised to $190 from $145 at Piper Jaffray. Piper Jaffray analyst JP McKim raised his price target on Inogen to $190 after the company’s Q1 earnings beat and raised FY18 revenue guidance after market close today. The analyst notes that most of the beat came from the BTB segment, though DTC and international business units were also “strong”. McKim is particularly impressed with the company’s 77% volume growth, adding that while the stock trades at a high multiple, its accelerating growth and low market penetration justify the Overweight rating.

Sermo’s ‘Yelp for drugs’ platform chalks up 665K doctor reviews in just 1 year

Sermo’s drug-rating system—by doctors for doctors—is soaring. A little less than a year after its launch, the Yelp-like platform now has 655,000 ratings on more than 4,000 medications, CEO Peter Kirk tells us.
More than half those drugs have at least 100 reviews—and 18 of them have more than 1,000.
For pharma companies, the database has come to serve as real-time feedback on physicans’ experiences with their particular drugs. It’s also a way to evaluate brand equity, check reactions to promo and educational messaging, and get competitive intelligence.
The system isn’t meant as a pharma tool, Kirk said, but drugmakers can certainly glean constructive info from it.
“Sermo should not be thought of as a toy for pharma, right? It is really for doctors and to democratize medical knowledge and how medicine works in the real world,” Kirk said. “That being said, there are a lot of potential valued insights for pharma … where the doctors are engaging with each other peer to peer. That could be from joining a conversation to trying to understand physician perceptions and experiences and what they’re saying about different therapies and issues.”

In its inaugural year, the private and anonymous drug ratings database has been used by 70,000 doctors, whether they’re posting or reading reviews or both. Sermo’s own research found that half of doctors changed their perceptions or opinions about a drug after reading the ratings. Eighty-three percent said they believe the ratings will help improve outcomes and 74% said they’ll use the ratings again when doing research for treatments.
One of the things that surprised Sermo in the first year is how much time and effort physicians are willing to put in to share their opinions, Kirk said.

Next up for the system is working on placement into electronic health records systems.
“We’re in early talks about getting drug ratings onto EMR systems, and we hope to do that by the end of the year so doctors would have access to them at point of care in the exam room,” Kirk said.

For Merck Gardasil 9, China hands out landmark nod with lightning speed

That was remarkably fast: Just nine days into its Gardasil 9 review, China’s revamped drug regulator handed Merck’s HPV vaccine a conditional green light—a new approval tool the agency is testing in its quest to speed new drugs to market.
It’s a move that could further compress the living space of GlaxoSmithKline’s Cervarix, already pressed by Merck’s previous version of Gardasil, which covered four HPV strains instead of the latest product’s nine.
China’s Food and Drug Administration based its decision on Gardasil data that previously led to the quadrivalent shot’s Chinese approval last May, and for the latest approval, considered foreign clinical trial data specifically on Gardasil 9, the agency said in a Sunday announcement (Chinese). The conditional approval comes with requirements for additional studies and postmarketing surveillance.
Even in the U.S., conditional approval is a fairly new tool that has only gained popularity at the FDA in recent years. China’s former CFDA largely followed its U.S. counterpart’s “accelerated approval” standards when the agency proposed its own conditional approval pathway in May 2017. The top government finalized that plan in principle last October—along with a provision that allows foreign clinical data into drug applications. But detailed guidance for those approvals just wrapped up public comment mid-January.

Under those new rules, Gardasil 9 took a smooth regulatory road toward its approval. After accepting Merck’s new drug application on April 20, the agency put the shot on its proposed expedited review list on April 23. A five-day window for comment followed, and the vaccine apparently immediately moved to its approval afterward. CFDA announced on Sunday that it had granted the conditional approval the day before.
GSK’s Cervarix and Merck’s previous version of Gardasil weren’t so lucky. At a time when the Chinese regulatory process was widely considered too slow, both vaccines took about 10 years to reach the Chinese market. To hasten the launch of novel drugs and to work through the notoriously large backlog of applications for clinical trial and new drug approvals, the Chinese agency made scores of game-changing new rules in recent months. Ruyi He, M.D., chief scientist of its Center for Drug Evaluation, has said on several occasions that by 2020 the agency plans to double the number of drug reviewers to 1,600 from 800—and that’s up from just about 170 in 2015.
Zooming into the HPV arena now, Gardasil 9 could pose an additional threat to Cervarix, which has already lost out to the Merck duo in major markets in the U.S. and Europe. China might be its last chance to garner a meaningful amount of sales.

Cervarix was officially launched in China late last July, a year after it became the first HPV shot approved in the country. For the fourth quarter of 2017 and first quarter of 2018, Cervarix enjoyed sales of £62 million ($85 million) and £52 million, respectively, with both quarters’ results more than double those of the same period of the previous year. The British pharma made it clear in its reports that the vaccine’s overall growth was “driven by its recent launch in China.”
Gardasil, which won its Chinese nod last May, was officially launched in November through local distributor Zhifei Biological Products. Given that Merck reported $2.31 billion in sales from the Gardasil family last year—up from $2.17 billion in 2016—China’s early contribution wasn’t singled out. But in revising an exclusive supply contract with Merck last September, Zhifei disclosed that it bought 542 million Chinese yuan ($85.6 million) worth of Gardasil in 2017. Another 1.37 billion yuan ($217 million) was planned for 2018, and 1.78 billion yuan and 2.23 billion yuan for the following two years.
It’s not clear whether Merck will tap Zhifei’s help again to market Gardasil 9 in China. But chances are high, given that the Chinese company also takes care of Merck’s hepatitis A vaccine Vaqta, pneumococcal vaccine Pneumovax 23, and pentavalent rotavirus vaccine RotaTeq, which was just greenlighted in China on April 12.

CMS Chief Slams Administration Critics

Seema Verma, the administrator of the Centers for Medicare & Medicaid Services, lashed out Monday at critics of the Trump administration’s changes to the Medicaid program and the Affordable Care Act (ACA), also known as Obamacare.
“I take exception to those out there who have made claims that we have tried to sabotage the healthcare of the American people, particularly when it comes to the healthcare exchanges,” she said here at the World Health Care Congress. “Obamacare was failing long before Donald Trump became president and I became CMS administrator.”
The reality, said Verma, is that health insurers have fled the exchange markets “after losing millions of dollars,” adding that with only one insurer offering policies, “half the counties in America, and 10 states in our country, don’t even have a choice of health insurer.”
Verma went on to detail some of the problems with the ACA’s insurance marketplaces. “We were promised that Obamacare would lower premiums by up to $2,500 for a typical family, but the reality is that premiums more than doubled since its inception,” she said, noting that in states such as Arizona, premiums rose by an average of 190%, and in Oklahoma, they rose 201%.
“These are plain, clear facts,” said Verma. “The stark reality was that when we came into the market, we were faced with health exchanges that were pricing Americans out of the system … and punishing them with penalties for being unable to afford government-mandated coverage.” In December, Congress repealed the individual mandate, which required all Americans to acquire health insurance or pay a penalty.
Supporters of the ACA have blamed Republicans in Congress for the exchanges’ increasing premiums, citing Congress’s failure to continue funding the “risk corridors” that would have helped health insurers pay for higher-cost patients; providing that funding, they say, would have encouraged more insurers to offer policies on the exchanges and made the marketplaces more competitive. They also are critical of the Trump administration’s canceling of the cost-sharing reduction payments the federal government had been making to help lower-income enrollees with their copays and deductibles.
Verma said the administration would “refuse to stand idly by while Americans are suffering,” so officials are “cleaning up regulations to provide states with more flexibility … to create more choice and competition to help drive down costs.”
“We have also proposed to expand the use of short-term insurance to now be used as an affordable option for people caught between individual market premiums they can’t afford and no coverage at all,” she said. These short-term plans are not required to include all of the benefits mandated by the ACA and can therefore be sold more cheaply than plans on the exchanges.
In addition, “it’s impossible to address Obamacare without addressing the strain it put on the Medicaid program,” Verma said. For patients who are severely disabled, and for their families, “Medicaid is more than a safety net — it’s a lifeline, one that needs to be preserved and protected for those who truly need it.”
However, the ACA’s Medicaid expansion has resulted in the addition of able-bodied adults to the Medicaid rolls, and with increased reimbursement rates for this population. “That stretches the safety net for fragile populations who are still on the waiting lists for [services like] home care,” argued Verma, and puts millions of people “into a program that wasn’t designed to meet their needs.”
The Obama administration was resistant to efforts aimed at allowing states to tailor their programs to better serve this population, said Verma. The Trump administration is remedying this problem by allowing states to require able-bodied Medicaid recipients to either work, take classes, or volunteer — a mandate known as “community engagement.” Three waivers for community engagement have already been approved, she said, “and we have 11 more we should be making decisions on pretty soon.”
Currently, eight in 10 adults who are receiving Medicaid are in families in which at least one family member is working, according to a report from the Kaiser Family Foundation. In total, 60% of Medicaid recipients are working themselves; of those who aren’t working, most cite impediments such as a disability, illness, or caregiving responsibilities as the reason, the report found.
“I hope it’s clear to everyone in this room that through all our actions we start with the goal of putting patients first,” Verma concluded. “We need to work together to create a healthcare system that pays for value, not merely volume.”

Karyopharm to pursue FDA OK for multiple myeloma drug based on study

A typically reliable method of securing U.S. approval for a cancer drug these days is to run a small, single-arm clinical trial in patients no longer responsive to all approved therapies. Eke out a modest benefit for patients from the study, and FDA is often willing to greenlight approval.
This is the path Karyopharm Therapeutics is pursuing with its experimental pill selinexor. The small biotech announced Monday evening that 25 percent of multiple myeloma patients responded to treatment with selinexor in a single-arm clinical trial. The median duration of response was 4.4 months.
Karyopharm enrolled into the clinical trial 122 patients with multiple myeloma whose disease continued to advance despite previous treatment with a minimum of five currently available drugs, including the recently approved monoclonal antibody therapy Darzalex.
The most common side effects reported in the study were nausea, vomiting, and reduced appetite, although more specific details were not disclosed by Karyopharm Monday night.
Based on these study results, Karyopharm said it will seek approval of selinexor with the FDA in the second half of the year.
If approved, selinexor sales potentially could top $500 million, Jefferies analyst Maury Raycroft forecast in a research note ahead of Monday’s Karyopharma announcement.
Karyopharm shares closed Monday at $13.08 but were trading up by 27 percent in the after-hours session.

Regeneron, Sanofi Eye October for FDA Review of Skin Cancer Drug Cemiplimab

April 30, 2018
Regeneron and its longtime development partner Sanofi are keeping their proverbial fingers crossed that their profitable collaboration will yield yet another approved treatment this year.
This morning the companies announced the U.S. Food and Drug Administration (FDA) has set a PDUFA date of Oct. 28 to determine the fate of its anti-PD-1 skin cancer treatment, cemiplimab. The two companies firmly believe there is still room in the crowded checkpoint inhibitor field dominated by MerckAstraZeneca and Bristol-Myers Squibb. Merck’s Keytruda or Bristol-Myers Opdivo are responsible for generating billions of dollars in revenue for both companies. And that is something that Regeneron and Sanofi are hoping will occur with cemiplimab as well. In January Regeneron and Sanofi demonstrated their commitment to the development of the drug with an additional $1 billion for research and development. That additional billion dollars means the investment in cemiplimab will be increased to a minimum of $1.64 billion. If the FDA approved cemiplimab in October, it’s quite likely that the two companies will immediately begin planning for approval of the checkpoint inhibitor in other indications.
In the announcement today Regeneron and Sanofi said the FDA granted a priority review for the Biologics License Application for cemiplimab based on impressive topline results from its pivotal Phase II study that continued to show a high response rate in patients. Those results, which were released in December, spurred the companies to begin seeking approval for the checkpoint inhibitor. Additionally, the FDA will consider Phase I data from two advanced CSCC expansion cohorts. Updated results from both clinical trials will be presented at the 2018 ASCO Annual Meeting, the two companies said this morning.
Sanofi and Regeneron are developing the PD-1 inhibitor cemiplimab to treat adult patients with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC, the second deadliest skin cancer after melanoma. CSCC is the second most common type of skin cancer in the United States. It can be fatal to non-melanoma skin cancer patients, Although CSCC has a good prognosis when caught early, and it can prove especially difficult to treat when it progresses to advanced stages. Patients at this stage can be disfigured due to multiple surgeries to remove CSCC tumors on the head, neck and other parts of the body.
The Phase II topline results showed that of the 82 patients treated with cemiplimab 46.3 percent demonstrated an overall response rate. Last year the FDA granted the drug Breakthrough Therapy status following the Phase I results.
In addition to submitting cemiplimab for review in the United States, the two companies are also seeking approval in Europe. The European Medicines Agency accepted the Marketing Authorization Application earlier this month.
The Sanofi and Regeneron developmental partnership is proving to be a valuable one. In 2017 the two companies snagged FDA approval for two jointly-developed drugs. In May the companies gained approval for rheumatoid arthritis drug Kevzara (sarilumab). Data from a Phase III trial released in March showed that sarilumab outperformed AbbVie’s HumiraIn March 2017 the FDA approved Dupixent (dupilumab) for the treatment of adults with moderate-to-severe atopic dermatitis.

FDA Clears Pluristem Application for Emergency Radiation Treatment

The U.S. Food and Drug Administration (FDA) cleared Haifa, Israel-based Pluristem Therapeutics Investigational New Drug (IND) application for PLX-R18 to treat acute radiation syndrome (ARS).
The U.S. National Institutes of Health’s National Institute of Allergy and Infectious Diseases (NIAID) sponsored and wrapped a successful Phase II-equivalent trial of PLX-R18 to treat ARS using the FDA’s animal rule pathway. The FDA Animal Efficacy Rule applies to developing and testing of drugs to reduce or prevent serious and life-threatening conditions caused by exposure to lethal or permanently disabling toxic agents where human testing it’s feasible or ethical.
It is also being evaluated by the U.S. Department of Defense (DOD) in supported of the military for treatment before or within the first 24 hours of radiation exposure. The company is currently in talks with the FDA and several U.S. governmental agencies for proposed pivotal study of PLX-R18 in ARS.
The traditional emergency treatment for radiation poisoning is potassium iodide (KI). This compound helps block radioactive iodine from being absorbed by the thyroid gland, which is the part of the body most sensitive to radioactive iodine.
Just last week, Canadian residents in Amherstburg, Ontario, directly across Lake Erie from the Fermi nuclear power plant in Monroe, Michigan, were offered potassium iodide pills as part of a plan to educate people in the “primary zone” of the Enrico Fermi nuclear power plant.
PLX-R18 is a cell therapy designed to treat bone marrow that can’t produce enough blood cells because of ARS, as well as certain cancers, cancer treatments, or immune-mediated bone marrow failure. Early animal studies were conducted in collaboration with the Hadassah Medical Center, and additional preclinical trials were performed with the NIH, Hadassah, the Charite in Berlin, and other research institutions.
“We are proud to have PLX-R18 join the exclusive club of IND approved medical countermeasures for the treatment of ARS,” said Zami Aberman, chairman and co-chief executive officer of Pluristem, in a statement. “This FDA clearance is one of the most significant milestones in the development of PLX-R18 to date and should provide Pluristem with significant support in advancing its off-the-shelf cell therapy into a pivotal trial. The fact that we are now able to treat human casualties in the case of a nuclear event provides us with the ability to protect from severe health consequences, savings lives of population in need.”
The drug is injected into muscle two times, a week apart. It can be used up to 96 hours after exposure. Arik Eisenkraft, the company’s director of homeland defense projects, told Reuters, “We are increasing the survival rate following high-level radiation exposure. We see improvement in all three blood lineages—red cells, white cells and platelets. We think that the everyday use of the compound will be for bone marrow failure of any cause.”
It also has an advantage in that it doesn’t require tissue matching prior to administration. The company is also conducting Phase III trials in the U.S. and Europe of PLX-PAD, to treat patients with the end-stage of peripheral artery disease, to prevent amputation.
The company indicates it will begin to prepare an emergency stock of PLX-R18 for emergency use. Full approval will depend on the Phase III clinical trial results.
Pluristem’s technology uses placental cells and a proprietary 3D technology platform to develop cell therapies for a variety of conditions, including inflammation, ischemia, muscle injuries, hematological disorders and radiation exposure.

iRadimed Q1 revenues up 37%; raises fiscal guidance

  • iRadimed (IRMD) Q1 results: Revenues: $7.1M (+36.5%); Operating Income: $1.1M; Net Income: $0.8M; Non-GAAP Net Income: $1.2M; EPS: $0.07; Non-GAAP EPS: $0.10; CF Ops: $1.6M.
  • 2018 Guidance: Revenues: $29.3M – 30.0M; EPS: $0.30 – 0.33 from $0.22 – 0.27; Non-GAAP EPS: $0.40 – 0.43 from $0.33 – 0.38.
  • Q2 Guidance: Revenues: $7.2M- 7.3M; EPS: $0.07 – 0.08; Non-GAAP EPS: $0.10 – 0.11.

Spark Therapeutics sells priority review voucher for $110M

Spark Therapeutics (ONCE) announced it has entered into an agreement to sell its rare pediatric disease priority review voucher, or PRV. The PRV was received when LUXTURNA was approved by the FDA. Under the PRV program, a sponsor who receives FDA approval for a rare pediatric disease drug or biologic may qualify for a voucher to be redeemed at a future time for priority review of a subsequent marketing application for a different product. Per the terms of the agreement with Jazz Pharmaceuticals (JAZZ), Spark Therapeutics will receive $110M upon closing of the transaction, which is subject to customary closing conditions

United Therapeutics To Acquire SteadyMed

 United Therapeutics Corporation (UTHR) and SteadyMed Ltd. (STDY) announced today the signing of a definitive merger agreement under which United Therapeutics will acquire SteadyMed for $4.46 per share in cash at closing and an additional $2.63 per share in cash upon the achievement of a milestone related to the commercialization of Trevyent®. The transaction, including the $75 million in contingent consideration, is valued at $216 million.
SteadyMed is a specialty pharmaceutical company focused on the development and commercialization of drug product candidates to treat orphan and high-value diseases with unmet parenteral delivery needs. SteadyMed’s product portfolio includes Trevyent, a development-stage drug-device combination product that combines SteadyMed’s two day, single use, disposable PatchPump® technology with treprostinil, a vasodilatory prostacyclin analogue, for the subcutaneous treatment of pulmonary arterial hypertension (PAH). United Therapeutics is a leading biotechnology company focused on the development and commercialization of therapies for the treatment of PAH and other orphan diseases.
“We are optimistic about acquiring SteadyMed and adding Trevyent to our pipeline of products to treat PAH,” said Martine Rothblatt, Ph.D., Chairman and Chief Executive Officer of United Therapeutics. “We are especially impressed with SteadyMed’s management team and global supply chain. Trevyent fits in well with our mission, and we look forward to bringing the product to the maximum number of patients as soon as possible.”
“United Therapeutics has always been at the forefront of developing therapies to treat PAH, and we are delighted at the prospect of our companies coming together, as one, to continue that mission,” said Jonathan M.N. Rigby, President and Chief Executive Officer of SteadyMed. “We believe that this proposed acquisition will help us realize our commitment to bring Trevyent to market to improve the lives of patients with PAH.”
The Board of Directors of SteadyMed has unanimously approved the merger agreement and unanimously recommends that SteadyMed shareholders adopt the merger agreement. SteadyMed shareholders owning approximately 43.3 percent of the ordinary shares of SteadyMed have entered into an agreement to vote their shares in favor of the transaction.
The transaction is subject to customary closing conditions, including approval by SteadyMed’s shareholders and the expiration or termination of the required waiting period under the Hart-Scott-Rodino Antitrust Improvements Act, and is expected to be completed in the third quarter of this year.

Allergan CEO: Big deals ‘unlikely’ from review

Says smaller deals more likely on the table. Says a fundamental shift in business strategy “unlikely.”

STAAR upped to buy by Blair

STAAR Surgical upgraded to Outperform from Market Perform at William Blair. William Blair analyst Brian Weinstein upgraded STAAR Surgical to Outperform saying he’s grown more comfortable with the company’s ability to forecast demand trends and drive a baseline case for mid- to high-teens revenue growth through 2020. STAAR has a number of potential positive catalysts over the next 12-18 months, including possible opening of the U.S. market again, which could boost growth above these levels and provide upside from current stock levels, Weinstein tells investors in a research note