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Thursday, October 10, 2019

Business Leaders Should Crunch the Numbers On Medicare for All

Big business appears to be getting behind Medicare for All.
That’s one way to read a new report from the National Business Group on Health. The organization recently asked 147 large employers that provide coverage to over 15.6 million workers and their dependents for their opinions of Medicare for All.
Some employers expressed concern about the quality of care under Medicare for All, or how much it would cost. Others embraced it or had little idea what it would entail.
This lack of awareness about the details of Medicare for All is problematic. Even its proponents acknowledge that it would raise taxes or introduce new taxes, particularly on businesses. Evidence from other countries with similar systems, meanwhile, shows that Medicare for All would deliver poor care to patients.
Not every employer realizes what the transition to Medicare for All would entail. Four in five respondents to the National Business Group on Health’s survey correctly said the government would have to raise taxes to get the program off the ground. But around one in five did not know whether taxes would go up or down.
That’s alarming. Medicare for All could cost as much as $40 trillion in its first decade, according to one of its own architects, Senator Bernie Sanders. He’d raise a big chunk of that money by taxing employers.
Chief among the levies he has in mind is a 7.5 percent payroll tax. He expects this will extract $3.9 trillion from employers over 10 years. Sanders has also proposed a tax hike on capital gains for those earning above $250,000 annually and a 4 percent tax on every American household.
Even that wouldn’t be enough to finance the plan. So employers are likely to face even higher taxes down the line.
Employers are equally unclear about how Medicare for All would affect their workers. Less than half the respondents in the National Business Group on Health survey believed their employees’ health costs would go up under Medicare for All. Some said Medicare for All would drive down their workers’ health costs.
But that’s not the case. More than 70 percent of working, privately insured households would pay more for health care under a fully-funded single-payer plan than they currently do, according to an analysis from Emory University professor Ken Thorpe. Another study found that Medicare for All would cause the average American’s post-tax income to shrink by more than 10 percent.
Employers seem equally misinformed about how Medicare for All would transform the U.S. healthcare system. Only 56 percent of those polled by the National Business Group on Health said the plan would decrease the quality of patient care; a mere 40 percent said it would reduce healthcare access.
By making care free to patients at the point of access, Medicare for All could induce effectively unlimited demand for care. That surge in demand would encounter comparatively limited supply. The country is already projected to be short 122,000 physicians by 2032. Long waits would be the result.
For proof, look to Canada, whose healthcare system is the model for Medicare for All. Last year, Canadian patients waited a median of 19.8 weeks to receive treatment from a specialist after receiving a referral from a general practitioner, according to the Fraser Institute. For some specialties, the wait is even worse. Patients waited a median of 39 weeks for orthopedic surgery.
Medicare for All may seem tempting to businesses struggling to absorb the steadily rising cost of health benefits. But it would deliver a major hit to their bottom lines — and consign their workers to long waits for substandard care.
https://www.realclearmarkets.com/articles/2019/10/09/business_leaders_should_crunch_the_numbers_on_medicare_for_all_103939.html

Hexo gives prelim Q4 revenue, pulls 2020 guidance

HEXO Corp (“HEXO” or the “Company”) (TSX: HEXO; NYSE: HEXO) is providing preliminary revenue for its fiscal fourth quarter and year ended July 31, 2019 and is also withdrawing its previously issued financial outlook for fiscal 2020.
Based on preliminary financial information and subject to year-end closing adjustments, HEXO expects net revenue for the fourth quarter to be approximately $14.5 million to $16.5 million and net revenue for the year to be approximately $46.5 million to $48.5 million.
“Fourth quarter revenue is below our expectation and guidance, primarily due to lower than expected product sell through,” commented Sebastien St-Louis, CEO and co-founder of HEXO Corp. “While we are disappointed with these results, we are making significant changes to our sales and operations strategy to drive future results. Over the past quarter, we began re-configuring our operations to focus on high-selling strains and initiated a new sales strategy that we believe will meaningfully improve performance.  We plan to discuss these in more detail on our upcoming earnings call.”
Slower than expected store rollouts, a delay in government approval for cannabis derivative products and early signs of pricing pressure are being felt nationally. The delay in retail store openings in our major markets has meant that the access to a majority of the target customers has been limited.  Additionally, regulatory uncertainty across the pan-Canadian system and jurisdictional decisions to limit the availability and types of cannabis derivative products have contributed to an increased level of unpredictability. As a result, HEXO is withdrawing its previously issued financial outlook for fiscal year 2020.
“Withdrawing our outlook for fiscal year 2020 has been a difficult decision,” added St-Louis. “However, given the uncertainties in the marketplace, we have determined that it is the appropriate course of action. We are also placing a greater focus on profitability. We are evaluating our plans and operations to see where we can be even more efficient. We are at our best when we are highly focused on our strategic priorities, always with a view to drive long-term value for shareholders. Growing low-cost, quality cannabis and developing innovative products is our priority and we are renewing our commitment to do so.”
The Company plans to release its complete financial results for the year ended July 31, 2019 on Thursday, October 24, 2019, before markets open, as well as host a webcast for investors and analysts at 8:30 a.m. EDT that same day.
Webcast Details
Date: October 24, 2019
Time: 8:30 a.m. EDT
Webcast: https://event.on24.com/wcc/r/2112048/C13DC676CE53A5FD2CEB4095D0FF4A3A
Replay information: A replay of the call will be accessible by telephone until 11:59 a.m. EDT on November 7, 2019.
Toll Free Dial-In Number: 1-888-390-0541.
Replay Password: 531469#
https://finance.yahoo.com/news/hexo-corp-provides-preliminary-fourth-103000615.html

European biotech firm BioNtech jumps 10% in U.S. market debut

Shares of BioNTech SE jumped on their market debut on Thursday, after the German biotech firm downsized its initial public offering (IPO) and priced its stock below an earlier expected range.

The shares, whose launch was the latest test of fragile sentiment this year for primary stock market offerings, opened up 10% at $16.50 and rose as high as $16.69 in early trading on Nasdaq, giving the company a market valuation of $3.78 billion (3.08 billion pounds).
The company had originally aimed to sell 13.2 million American Depository Share (ADS) at between $18 and $20 apiece, but on Wednesday cut the deal size to 10 million ADS at $15 to $16 per share.
The final pricing of its public flotation at $15 per ADS helped BioNtech raise about $150 million less than a month after the dramatic collapse of another major IPO for WeWork owner The We Company.
The Mainz, Germany-based company specializes in messenger RNA (mRNA) molecules that prompt human cells to produce therapeutic proteins, triggering an immune response against cancer or infectious diseases.
Its closest rivals are Massachusetts-based Moderna and Belgium’s eTheRNA, which are currently locked in the race to develop mRNA therapies.
BioNtech said it expects to use proceeds from the public share sale for general corporate purposes and to acquire or invest in complementary technologies and products.
J.P. Morgan, BofA Securities and UBS were the lead underwriters of the IPO.

https://www.marketscreener.com/news/European-biotech-firm-BioNtech-jumps-10-in-U-S-market-debut–29362460/

Americans Increasingly View People With Mental Illness as a Threat

Despite an absence of supporting evidence, Americans increasingly view people with mental illness as a threat, and many support involuntary hospitalization for such individuals, new research shows.
The increasing stigma toward people with mental illness is “concerning, as it can lead to increasing discrimination,” lead author Bernice Pescosolido, PhD, distinguished professor of sociology at Indiana University in Bloomington, told Medscape Medical News.
“People with mental health problems have to face the challenges of having a major illness. The increasing stigma that we document translates into these people and their families also having to fight the rejection, isolation, and denial of civil rights that may come with an increasing negative climate of fear,” said Pescosolido.
The study was published online October 7 in Health Affairs.

“Mentally Ill Monsters”

Political rhetoric regarding mass shootings that wrongly links violence to mental illness likely plays a role in the evolving public view, the researchers say.
Following the August mass shootings in El Paso, Texas, and Dayton, Ohio, President Trump said publicly that mental illness is linked to gun violence and characterized psychiatric patients as “mentally ill monsters.”
In response, the American Psychiatric Association quickly issued a statement saying that the “overwhelming majority” of people with mental illness are more likely to be victims of violent crime rather than the perpetrators, as reported by Medscape Medical News.
Pescosolido and colleagues examined trends in public perceptions of violence regarding people with mental illness and in support for coerced treatment of these individuals over a 22-year period using data from three National Stigma Studies conducted in 1996, 2006, and 2018.
As part of the studies, respondents were given one of three vignettes describing people who meet clinical criteria for mental disorders or one describing a person with nonclinical “daily troubles.”
Results showed a general increase over time in people’s perceptions that individuals with mental disorders are dangerous. Results also indicated a general increase in support for stripping these individuals of civil rights, significantly so for individuals with schizophrenia.
By 2018, more than 60% of respondents regarded people who met criteria for schizophrenia as being dangerous to others, and 44% to 59% supported coercive treatment (either medication, forced physician visit, or hospitalization), the researchers found.
In addition, 68% of respondents in 2018 viewed people who had alcohol dependence as being dangerous to others, and 26% to 38% supported coercion.
“Lower but substantial percentages” were reported for people with depression; 30% thought that people with major depression were likely to be dangerous to others.
And “remarkably,” 20% of respondents felt that individuals with nonclinical “daily troubles” were likely to pose a threat to others, they report.

Troubling Trends

Pescosolido said these results are troubling because little evidence supports the hypothesis that individuals with mental illness are more likely to commit violence.
Even more troubling, she said, is the increasing risk of using legal means to force individuals who only have problems of daily living, not mental illness, into treatment.
“Given the current state of affairs, it is critical to continue to monitor cultural attitudes, beliefs, and predispositions that link mental illness to violent behavior. The continued existence of such beliefs and their connection in the public mind to forced treatment may drive the adoption of formal public health laws that restrict the lives of people diagnosed with mental illness,” the authors write.
Undertaking efforts to counteract sources of stigma is “daunting but indispensable to public health. We caution against policies based on erroneously linking mental illness and violence. Public policies stoked by political rhetoric will not improve the lives of any Americans,” they conclude.
The research was supported by grants from the National Science Foundation, the Brain and Behavior Research Foundation, the Indiana Consortium for Mental Health Services Research, and the Indiana University Network Science Institute. The authors have disclosed no relevant financial relationships.
Health Aff. Published online October 7, 2019. Abstract
https://www.medscape.com/viewarticle/919700

Menarini Silicon Biosystems banks on cells for its liquid biopsy

The group has the only approved cell-based cancer blood test in the world, but competing in an increasingly cutthroat market will be tough.
Most companies exploring liquid biopsies use technologies that search for and sequence DNA strands shed by tumours into a patient’s blood. Not so Menarini Silicon Biosystems: the company offers Cellsearch, the only FDA-cleared product for the enumeration of entire circulating tumour cells (CTCs) in blood samples.
“With CTCs we can offer a wide range of information compared to circulating tumour DNA [ctDNA],” Fabio Piazzalunga, president of MSB, tells Vantage. “We believe that the future will be linked to single-cell technologies.”
Single cell analysis is a technique that can aid the understanding of metastasis and therapy resistance with more precision than bulk tumour measurements, as well as tease out the role of rare cells in tumour progression. MSB’s Cellsearch technology can perform this testing; the company, a subsidiary of the private Italian pharma company Menarini, bought Cellsearch from Janssen in 2017.
Cellsearch can obtain clinically useful information in a number of ways. Most obvious is simply counting the cells, which can be used as a prognostic marker in metastatic disease, and it is this use for which Cellsearch has FDA clearance, as a way to monitor patients with metastatic breast, colorectal or prostate cancer.
It also has other potential applications for which it is not yet approved. These include measuring tumour antigens such as Her2 and PD-L1, useful for selecting targeted drugs or immunotherapy and monitoring the effectiveness of the treatment, and, based on the analysis of a single cell, acquire “information on tumour heterogeneity and chronicle how its evolution responds to therapy”, Mr Piazzalunga says. The CTCs can be isolated as live cells, providing information on gene expression.
Complementary
The group is not entirely eschewing the other liquid biopsy approach, however. It has a distribution agreement with the Taiwanese company Plexbio under which it distributes the latter’s IntelliPlex circulating tumour DNA analysis technology.
“We believe that the two technologies, CTCs and ctDNA, provide complementary rather than mutually exclusive information,” Mr Piazzalunga says.
MSB is not pursuing new regulatory clearances at the moment. Instead it is developing new uses for its systems; Mr Piazzalunga says it is working towards a new application of the Cellsearch technology related to multiple myeloma.
The system can run a full battery of tests, including the in-depth analyses that are not yet approved by the regulators, on around five patient samples per day, Mr Piazzalunga says. He adds that the company is working towards upgrading the system to allow a shorter turnaround time.
MSB is also moving into another fast-growing area: prenatal testing. Remarkably, cells can pass from a foetus into the mother’s bloodstream, and MSB is working towards developing tests for foetal genetic disorders using a blood draw taken from the mother. The technology will be able to detect several prenatal abnormalities, though Mr Piazzalunga declined to specify exactly what these might be.
The prenatal diagnostic will be a laboratory-developed test sold under a Clia waiver “at the beginning”, Mr Piazzalunga says. “Once we get the necessary clinical evaluation data, we will submit it.”
Too many companies
But liquid biopsy is the big prize. As targeted therapies and immuno-oncology continue their meteoric rise, competition to develop a successful blood-based test to aid patient selection is fierce, and companies are increasingly vying to have their tests put on an equal footing with tissue biopsy (Esmo 2019 – Foundation eyes liquid biopsy rule change, September 30, 2019).
“There are many, many companies in the liquid biopsy space – we believe there are too many,” says Mr Piazzalunga, not unreasonably. He believes that the market will come to a crunch point after which the less successful cancer blood test developers fall by the wayside. After that, he says, a couple of dozen test makers will remain, “and we count on being one of those”.
Having FDA clearance for a cell-counting test in three metastatic, solid tumour indications is a commercial advantage, Mr Piazzalunga says. Tests such as Foundation Medicine’s FoundationOne Liquid and Guardant Health’s Guardant360, both of which use circulating DNA rather than CTCs, are on sale in the US as lab-developed tests but are not approved or cleared by regulators.
At least one other company is also looking at CTCs: the UK-based group Angle (Interview – Aiming for a new Angle on liquid biopsy, July 25, 2017).
MSB will not disclose its sales, though Mr Piazzalunga says it is “by far the market leader” in CTC, with more 45,000 CTC tests run per year. By contrast Guardant360 is forecast to have sales of $185m in 2019 – and should Guardant’s test gain the FDA’s imprimatur too, it will likely see a bit of a sales boost.
Competition is only getting more intense, and doctors will only be convinced of the utility of cell-based tests by positive clinical data. It is data that will determine whether MSB ends up as one of the last groups standing.
https://www.evaluate.com/vantage/articles/interviews/menarini-silicon-biosystems-banks-cells-its-liquid-biopsy

Novo goes early to leapfrog haemophilia gene therapies

Novo Nordisk took an early step towards genome editing with yesterday’s Bluebird tie-up, and isn’t ruling out a move into conventional gene therapies.
Novo Nordisk claims to be a leader in haemophilia, but looks to have missed the boat with the first wave of gene therapies for the disorder.
The group acknowledged this yesterday when it struck a deal with Bluebird Bio covering in vivo genome editing, which it hopes will become the next big thing. But, like many Novo collaborations, the project is extremely early, and the haemophilia space could look very different in several years’ time.
In haemophilia A, the initial focus of the Novo-Bluebird project, several gene therapies are approaching the market. The most advanced, Biomarin’s valoctocogene roxaparvovec (valrox), is set to be filed by the end of this year, and is expected to become the third-biggest seller in the disease by 2024, according to EvaluatePharma sellside consensus.
Meanwhile, Novo’s next haemophilia hope, the tissue factor pathway inhibitor concizumab, is forecast to bring in just $47m that year. That asset is due to begin phase III development this year.
Top five haemophilia A therapies in 2024




Annual sales ($m)
Product Company Description Status 2018 2024e
Hemlibra Roche/Chugai Anti-factor IXa/X bispecific MAb Marketed 230 3,979
Eloctate Sanofi/Swedish Orphan Biovitrum Factor VIII Marketed 1,239 1,234
Valoctocogene roxaparvovec (valrox) Biomarin AAV-factor VIII gene therapy Filing imminent 1,212
Advate/Adynovate Takeda Factor VIII Marketed 2,860 1,173
NovoSeven Novo Nordisk Factor VII Marketed 1,033 595
Source: EvaluatePharma.
Valrox and other conventional gene therapies are based around a viral vector that delivers the gene for a missing clotting factor to patients’ livers – factor VIII in the case of haemophilia A.
Meanwhile, Novo and Bluebird hope to elicit a genetic change directly in patients using the latter’s megaTAL editing technology.
Daniel Timmerman, vice-president of Novo’s biopharm transformational research unit, admitted that were the company to start developing a traditional haemophilia gene therapy now, its chances of being competitive would be “relatively slim”.
However, he stopped short of ruling out such a move. “Classic gene therapies are still attractive for Novo. We’re interested in new opportunities close to where we’re already working,” he told Vantage, citing rare genetic diseases as an area of interest for Novo and also a sector that “lends itself well to gene therapies”.
Given Novo’s reputation as a builder rather than a buyer, this seems unlikely to translate into a big acquisition. But perhaps this possibility should not be completely dismissed given the Danish group’s strategy shift last year, not long after it tried and failed to buy Ablynx (Novo casts its net wider but shrinks in the process, September 19, 2018).
MegaTALented?
Various methods are currently being explored for in vivo genome editing. Bluebird’s megaTAL platform essentially combines aspects of two better-known technologies: Talens and meganucleases.
The TAL motif is responsible for DNA binding, while the meganuclease part provides the DNA strandbreak, Mr Timmerman explained. He highlighted megaTALs’ specificity and the possibility of limiting off-target effects.
When asked why Novo had chosen not to work with traditional Talens – a space dominated by the French Car-T player Cellectis – Mr Timmerman said intellectual property had been a consideration, but that the main attraction was Bluebird’s technology.
He added that, while other genome editing modalities, including Talens, required two proteins to carry out DNA cleavage, megaTALs only needed a single molecule. This simplicity could turn out to be an advantage in a sector where caution around safety is high, he said, but conceded that this needed to be proven.
It will be a while before it becomes clear whether Novo has made a good move. The groups hope to have a candidate to take into preclinical testing by the end of the three-year collaboration.
No financial details of the deal were given, and Bluebird will continue to develop megaTALs for other applications.
Rivals include Casebia Therapeutics, a joint venture between Bayer and Crispr Therapeutics that is investigating Crispr/Cas9 in haemophilia, among other diseases. Sangamo Therapeutics has an in vivo editing candidate for haemophilia B based on its zinc finger technology, as well as its conventional AAV-vector based haemophilia A gene therapy.
Mr Timmerman noted that genome editing held the promise of a truly lifelong correction of a faulty gene, which might not be the case with gene therapies. He conceded, however, that the approach could also raise the risk of a lifelong genetic error being inadvertently introduced. No wonder Novo is taking it slowly.
https://www.evaluate.com/vantage/articles/news/deals/novo-goes-early-leapfrog-haemophilia-gene-therapies

UCB goes to Ra for a complementary approach

The $2bn takeout of Ra Pharmaceuticals provides solace for investors bemoaning the biotech market’s poor performance.
With biotech indices in the doldrums some assets are affordable again. Step forward Ra Pharmaceuticals, a group whose stock had come off 30% in the past 10 weeks, making it a perfect target for the acquisition-hungry UCB of Belgium.
The rationale for the deal is zilucoplan, Ra’s sole asset of note, which UCB hopes will boost its presence in complement-mediated diseases, most importantly myasthenia gravis. And more broadly, fuelled by optimism over the takeout’s 110% premium, many investors will look to the move as a sign that the biotech market’s pitiful performance is bottoming out.
Surprisingly, however, UCB already has a project that targets complement-mediated diseases, and does so in a way that differs from many competitors: rozanolixizumab is an inhibitor of the neonatal FcRn receptor, which is thought to control the recycling of IgG and thus reduce the activation of complement that is thought to cause autoimmune diseases like myasthenia gravis.
Ra’s zilucoplan, meanwhile, works by binding complement C5, and inhibiting its cleavage into C5a and C5b. This strategy is being pursued by several companies, including Apellis, Roche and Regeneron, which are trying to challenge the leading C5 inhibitor, Alexion’s Soliris (Ra joins the challenge to Alexion’s Soliris stranglehold, December 12, 2018).
Thus UCB’s acquisition can be read in one of two ways. Either the group is concerned that the FcRn approach is not all it was cracked up to be, and needs something better to maintain its challenge, or it wants to give itself multiple shots on goal. On a call today the Belgian group insisted that the deal was complementary, and cited the possibility of a combinatorial approach.
Alexion monopoly
Certainly, the monopoly of the mighty Alexion will prove hard to overcome. Soliris is notorious for being one of the world’s most expensive drugs, and Ra had indicated that competing on price was one possibility; UCB says it aims to price zilucoplan “in a very competitive way”.
But Alexion has no fewer than four fingers in the complement-mediated disease pie: Soliris, the Soliris follow-on Ultomiris, and two FcRn projects: ALXN1830, acquired through the takeover of Syntimmune, and ABY-039, a subcutaneous asset licensed earlier this year from Affibody.
While myasthenia gravis is UCB’s initial focus the group played up zilucoplan’s potential in other diseases, including amyotrophic lateral sclerosis. Last month the project became one of 30 selected for an academia-sponsored study called Healy ALS, based on the theory that complement-related proteins are implicated in this CNS disease.
Selected players in complement-mediated diseases
Project Company Mechanism Delivery Status
Soliris Alexion Anti-complement factor C5 MAb IV Sold for PNH, HUS & MG
Ultomiris Alexion Anti-complement factor C5 MAb IV, working on SC Sold for PNH, filed for HUS, ph3 MG
Rozanolixizumab UCB Anti-FcRn MAb IV & SC Ph3 MG
Zilucoplan UCB (ex Ra) Complement factor C5 inhibitor SC Ph 3 MG; ph2 PNH
Efgartigimod Argenx Anti-FcRn Ab fragment IV, working on SC Ph3 MG
APL-2 Apellis Complement factor C3 inhibitor SC Ph3 PHN
RG6107/SKY059 Roche Anti-complement factor C5 MAb SC Ph2 PNH
Pozelimab Regeneron Anti-complement factor C5 MAb IV & SC Ph2 PNH
ACH-4471 Achillion Anti-complement factor D Oral Ph2 PNH
M281 Momenta Anti-FcRn MAb IV, SC planned Ph2 MG
IMVT-1401 Roivant Anti-FcRn MAb SC Ph2 MG
Synt001/ALXN1830 Alexion (ex Syntimmune) Anti-FcRn MAb IV, SC planned Ph2 MG
ABY-039 Alexion/Affibody Anti-FcRn bivalent Ab mimetic SC Ph1
Source: EvaluatePharma. PNH=paroxysmal nocturnal hemoglobinuria; HUS=haemolytic uremic syndrome; MG=myasthenia gravis.
Apart from the competition in the market, another concern is anticompetitive authorities – particularly at a time when Bristol-Myers Squibb’s takeover of Celgene and Roche’s purchase of Spark have been delayed by an unusually strict stance by the US FTC.
Indeed, it is worth considering whether UCB/Ra’s putative position in complement-mediated diseases might also be construed as anticompetitive. Perhaps Alexion’s position dispels such fears, however, and UCB today said it was confident that the Ra deal did not raise anticompetitive concerns, without elaborating further.
The all-cash transaction will see UCB pay $48 for each Ra share – the stock had previously maxed out at $36 in July – amounting to $2.5bn in total, or $2.1bn net of Ra’s cash balance.
The proof of UCB’s wisdom will be seen in early 2021, when zilucoplan’s pivotal myasthenia gravis study, Raise, reads out; a phase III trial of rozanolixizumab should yield data at roughly the same time, and the two projects are expected to generate 2024 sales of $406m and $136m respectively, according to EvaluatePharma’s sellside consensus.
Stifel analysts today opined that the UCB/Ra deal was exactly what small/mid-cap cap biotech needed right now. It is probably too soon to gauge the move’s effect on the overall space, but this morning Apellis and Momenta crept up 4% and 1%.
https://www.evaluate.com/vantage/articles/news/deals/ucb-goes-ra-complementary-approach