Reports Q2 revenue $33.9M, consensus $26.74M. “STAAR generated record quarterly sales of $33.9 Million, a 55% increase from prior year, driven by the continuing expansive growth of our EVO Visian ICL(TM) family of lenses,” said Caren Mason, President and CEO. “ICL unit growth highlights for the quarter included Japan up 131%, China up 127%, Canada up 64% and India up 61% with solid 30% unit growth in Germany and 20% growth from our European distributors. We continue to see a high level of momentum in our key international markets and therefore believe our second half sales growth may exceed 20% even taking into account our strong finish to 2017. In addition, we believe our full year fiscal 2018 sales growth may now exceed 25% compared with our prior target for sales growth closer to 20% over 2017, based on current market conditions.”
Wednesday, August 1, 2018
Discovery of unknown lung cell points to new target for cystic fibrosis meds
The researchers set out to take a census of all the cells that line our airway, a taxonomy of the tissue made possible by new technology. Perhaps, they thought, they would find different subtypes of cells they already knew existed or would come to better understand the cells’ functions.
As the data came in, though, they pointed to a more intriguing finding: a previously unknown cell, one that was similar to a cell found in the gills of fish and skin of frogs.
Senior researchers were initially dubious. “I thought, ‘It’s great to have graduate students who are so bold,’ but in the back of my head, I was thinking there was no way this was right,” said Dr. Jay Rajagopal, a developmental biologist and pulmonologist at Massachusetts General Hospital.
But in a pair of papers published Wednesday in the journal Nature, two independent research teams reported that they didn’t just unearth this new type of cell, but that the cell appears to play an outsize role in cystic fibrosis.
Taken together, the discoveries could shift the understanding of the basic biology of the devastating lung disease, signal potential targets for new forms of therapies, and one day shake up the market for cystic fibrosis medications.
“They have discovered a previously unknown cell type that could hold the key to understanding the disease cystic fibrosis,” scientists Kyle Travaglini and Mark Krasnow of Stanford University, who were not involved in the research, wrote in an editorial also published Wednesday.
Other outside scientists praised the research but said they had questions about how much influence other types of lung cells still had over cystic fibrosis. The studies in part focused on the trachea, or windpipe, so researchers said they wondered if the same cellular story would hold deep at the ends of the airway, which is where cystic fibrosis first emerges, or if other cell types still had some involvement in the disease.
“That’s predominantly where the CF lesions are,” said Dr. Tien Peng, a CF expert at the University of California, San Francisco, who was not involved with the new studies.
Rajagopal was a co-senior author of one of the papers, along with Aviv Regev, a computational and systems biologist at the Broad Institute. Allon Klein, a systems biologist at Harvard Medical School, and Aron Jaffe, co-leader of respiratory disease research at the Novartis Institutes for BioMedical Research, were the senior authors of the other study.
The two Boston-area teams learned of each other’s work as it was ongoing, wound up coordinating their papers’ submission, and together landed on a name for the newly revealed cell: a pulmonary ionocyte.
For patients with cystic fibrosis, goopy mucus builds up in their lungs and can clog their airways. The mucus also acts like fly paper and ensnares bacteria, leading to regular infections and lung damage. About 30,000 people have the disease in the U.S.
Scientists have known that the disease is caused by mutations in the CFTR gene, but the working theory was that the gene was active in cells all over the airway, including ones with tiny hair-like structures called cilia that sweep out mucus and nasty invaders.
The new studies turn that thinking on its head. Instead of humming along in a variety of cells, the gene really only gets going in pulmonary ionocytes, the scientists reported. While ionocytes accounted for at most 2 percent of all cells in the tissue the researchers studied, they were responsible for the vast majority of CFTR activity.
That means when there are disease-causing mutations in the CFTR gene, it is these cells that treatments should target, the studies suggest. If researchers develop a CF gene therapy, for example, they could direct it to ionocytes or the stem cells that produce them.
Still, the research left open the idea that other airway cells are home to some CFTR activity, and outside experts said that future studies should aim to calculate how much CFTR activity throughout the lungs occurs in these ionocytes. Even if there were only a little CFTR expression in other types of cells, they noted, there are enough of them to contribute a sizable amount to the overall activity.
“The challenge now for all of us is to try to fit this information into the whole animal, i.e. the whole lung,” said Dr. Richard Boucher, a CF expert at the University of North Carolina, who was not involved in the studies. “Is [the ionocyte] the only actor in town? I suspect it’s not.”
CFTR encodes an ion channel (also called CFTR) that helps regulate the chemical and acid balance of the epithelium (the layer of tissue that lines the airway). Experts aren’t sure exactly why the cystic fibrosis mutations cause the problems they do — perhaps the channel can no longer control the acid levels of the mucus, which makes it thicker and harder to expel — but “it’s clear that breaking CFTR makes things go really wrong,” said Klein.
As part of their studies, the researchers identified a transcription factor — a molecule that can turn other genes on or off — called FOXI1 that drives CFTR activity. It might be possible then to use FOXI1 to promote the production of healthy pulmonary ionocytes — another potential therapeutic route beyond gene therapy.
“If we knew how to generate the cell, we could try to make more,” Jaffe said. “And by making more, you would hope that would increase the activity of the channel across the epithelium.”
For now, three existing medications — all made by Boston-based Vertex Pharmaceuticals — aim to improve the function of CFTR, but a gene therapy (if one were ever developed) could provide more lasting benefits for patients. Other drugs on the market help patients clear their mucus or stave off infections.
To create their census of airway cell types, the researchers used a technique called single-cell RNA sequencing. First, they broke apart their tissue samples into individual cells, then merged those with a few other key ingredients, leaving each cell ensconced in a tiny droplet of oil.
“Everything has to happen really, really, really quickly,” Regev said. “Otherwise the cells start dying and the RNA that’s in them starts degrading.”
Joining the cells inside their oily nooks is a particle that performs like a pricing gun, labeling each cell’s genetic details with a unique barcode. Thanks to the barcodes, researchers can track which gene came from which cell and can measure the gene expression of each cell.
As the researchers started sorting the data and identifying cell types, there was one pile that stood out as a big unknown. They noticed those cells shared features with cells called ionocytes that are found in fish gills and frog skin and that feature ion channels.
But despite their identification, pulmonary ionocytes still maintain some mystery. The papers’ authors said they’re not sure, for example, why so much CFTR activity appears concentrated in so few cells. As Rajagopal said, “Maybe they’re just super hardworking.”
EMA: More Staff Than Anticipated Quit Ahead of Brexit Relocation
The European Medicines Agency (EMA) says it will further scale back operations as staff who do not intend to relocate to Amsterdam next year begin to depart the agency.
“The temporary cuts in activities are required because it has also become clear that the agency will lose more staff than initially anticipated,” EMA says, noting that it expects staff departures to accelerate as the move to Amsterdam in March 2019 approaches.
EMA says it now expects to lose 30% of its staff ahead of the move and is uncertain about mid-term staff retention once it begins operations in Amsterdam. A survey of EMA staff conducted ahead of the European Council’s vote to determine the agency’s new home found that Amsterdam was the most favored location by staff, with only 19% reporting they would not relocate with the agency.
Due to employment rules in the Netherlands, EMA says it will not be able to bring some 135 short-term contract employees with it in the move.
Bracing for the departures, EMA says it has launched a comprehensive staff recruitment program, but in the near term will have to “reprioritize its resources to fully maintain its core activities,” such as reviewing marketing authorization applications and overseeing safety surveillance.
By 1 October 2018, EMA says it will have scaled back additional non-core activities.
On an international level, the agency will continue to focus on product specific issues and will still carry out Article 58 procedures, but will reduce its role in harmonization efforts and take on other global public health issues “on a case-by-case basis.”
EMA will also halt the publication and revision of guidelines except those that address an urgent public or animal health need, or that directly relate to Brexit.
Similarly, the agency will limit itself from organizing and attending stakeholder meetings that are not related to Brexit or its relocation.
EMA also says it will stop launching new procedures related to clinical data publication as of August, but will process all data packages submitted through July 2018.
‘Superbugs now also becoming resistant to alcohol disinfectants’
Multidrug-resistant “superbugs” that can cause dangerous infections in hospitals are becoming increasingly resistant to alcohol-based hand sanitizers and disinfectants designed to hold them at bay, scientists said.
In a study of what the researchers described as a “new wave of superbugs”, the team also found specific genetic changes over 20 years in vancomycin-resistant Enterococcus, or VRE – and were able to track and show its growing resistance.
Their findings were published on Wednesday in the journal Science Translational Medicine.
VRE bugs can cause urinary tract, wound and bloodstream infections that are notoriously difficult to treat, mainly because they are resistant to several classes of antibiotics.
In efforts to tackle the rise of hospital superbugs such as VRE and MRSA, or methicillin-resistant Staphylococcus aureus, institutions worldwide have adopted stringent hygiene steps – often involving hand rubs and washes that contain alcohol.
Tim Stinear, a microbiologist at Australia’s Doherty Institute who co-led the study, said that in Australia alone, use of the alcohol-based hand hygiene has increased tenfold over the past 20 years. “So we are using a lot and the environment is changing,” he said.
Yet while rates of MRSA and other infections have stabilised due to heightened hygiene, Stinear said, VRE infection rates have not. This prompted his team to investigate the VRE bug for potential resistance to disinfectant alcohols.
They screened 139 isolated bacterial samples collected between 1997 and 2015 from two hospitals in Melbourne and studied how well each one survived when exposed to diluted isopropyl alcohol.
They found that samples collected after 2009 were on average more resistant to the alcohol compared with bacteria taken from before 2004.
The scientists then spread the bacteria onto the floors of mouse cages and found that the alcohol-resistant samples were more likely to get into, and grow in the guts of the mice after the cages were cleaned with isopropyl alcohol wipes.
Paul Johnson, a professor of infectious diseases at Austin Health in Australia who also co-led the study, said the findings should not prompt any dramatic change in the use of alcohol-based disinfectants.
“Alcohol-based hand rubs are international pillars of hospital infection control and remain highly effective in reducing transmission of other hospital superbugs, particularly MRSA,” he said.
Stinear said health authorities should try higher-alcohol concentrate products and renew efforts to ensure hospitals are deep cleaned and patients found to be carrying VRE infections are isolated.
Cheaper Health Plans With Less Coverage Move Forward
The Trump administration, in one of its most significant efforts to roll back the Affordable Care Act, on Wednesday released a rule that will allow for the proliferation of cheaper, less-comprehensive health plans that have been restricted by the former Obama administration.
The rule would loosen restrictions on a type of coverage known as short-term medical insurance—low-cost plans that cover a limited period with less-expansive benefit offerings, which are subject to fewer consumer protection regulations.
Such plans now can only be carried up to 90 days. The new rule would allow the plans to last for a year and be renewed for a total coverage period of 36 months.
The plans are often sold to people who qualify as healthy and may have annual limits on the amount of care the policies will cover. The plans don’t have to cover people with pre-existing conditions, and insurers can charge higher premiums based on a consumer’s health status.
They also don’t have to include the specific benefits mandated by the ACA, such as prescription-drug coverage.
A proposed version of the rule was released in February, and the new action makes the rule final. It goes into effect in 60 days.
Health and Human Services Secretary Alex Azar said the step “will help increase choices for Americans faced with escalating premiums and dwindling options in the individual market.” The plans originally were designed for limited coverage when people were between jobs, for up to 90 days. Now, though dubbed “short-term,” the plans can be extended for a total coverage period of three years.
The administration said the average monthly premium for such a plan has been about $124, less than a third the cost of an unsubsidized plan in the ACA markets.
Democrats and some outside health-care groups, however, characterized the plans as “junk health insurance” that shortchanges consumers, is subject to fraud and falls short of the plans available under the Affordable Care Act.
“By giving the green light to junk plans, Trump and his administration are once again siding with fraudsters, unscrupulous brokers and insurance companies over unsuspecting Americans that simply want affordable health care,” said Sen. Ron Wyden (D., Ore.), the top Democrat on the Senate Finance Committee, which oversees many health-care issues.
Some insurers were also critical. The plans have “the potential to harm consumers, both by making comprehensive coverage more expensive and by leaving some consumers unaware of the risks of these policies,” said Justine Handelman, senior vice president of the Blue Cross Blue Shield Association.
The rule marks the latest in a series of Republican-led efforts to roll back the ACA after GOP lawmakers in Congress failed to repeal it last year. Congressional Republicans ended the ACA’s penalty on people who don’t have health coverage, a change that takes effect next year.
The ACA, also known as Obamacare, sought to make insurance more readily available to people who aren’t covered by a large employer, and to regulate that insurance more tightly.
President Barack Obama and fellow Democrats saw the ACA as a way to guarantee that people with prior health conditions could buy coverage, for example. They also required health plans on the individual market to cover a broad set of benefits, including maternity care and hospitalization, with the goal of protecting consumers from unwittingly buying shoddy products.
But Republicans say these insurance rules have driven up premiums for healthier Americans, especially the roughly seven million people in the individual market who don’t get tax credits that offset premium costs. The ACA also limits the ability of Americans to buy insurance policies that fit their circumstances, critics say.
Health analysts say the short-term plans that would be allowed under the new proposal would likely appeal to healthier customers seeking inexpensive alternatives to ACA plans, while higher-risk consumers would remain in traditional insurance coverage.
That could cause their premiums to rise, because healthier consumers are needed on the ACA’s individual market exchanges to offset costs of older and sicker people, analysts say.
Faster, cheaper DNA sequencing gets Omniome $60M led by Chinese investors
A San Diego company making new DNA sequencing tech just brought home $60 million in a fundraising round chock full of Chinese investors.
The company is called Omniome — first founded by an ex-Illumina employee Kandaswamy (Swamy) Vijayan in 2013 — and it says its tech can provide highly accurate and fast sequencing results at a low cost. Vijayan has since left Omniome, and the company is now run by CEO Dave Mullarkey.
This new Series B round was led by new investors Decheng Capital (Shanghai) and Hillhouse Capital (Beijing), joined by Lam Research Capital (US) and Nan Fung Life Sciences (Hong Kong). The round is the latest in an avalanche of deals involving US-based biotech companies teaming up with investors overseas.
The company’s tech is called “Sequencing by Binding” (SBB), which it says can give an “enhanced precision of nucleotide and DNA matching by leveraging the natural matching ability of the polymerase.” This decreases runtimes and boosts the number of samples per run.
First incubated in the San Diego tech accelerator EvoNexus, Omniome plans to use the cash to expand the company’s team — which currently stands at 40 people, according to the company’s website. They’re looking to add staff in several areas, including engineering, and to increase its research and production capacity. Omniome also plans to optimize its technology.
“We have been able to validate the tremendous power of our proprietary sequencing biochemistry,” Mullarkey said in a statement. “Now we are directing our efforts on product development to rapidly advance our first commercial instrument prototypes.”
Bar lowers yet again for Priority Review Voucher prices
Are we back where we started with the price tag of priority review vouchers? Their dwindling value slid further south this morning, with news that Kyowa Hakko Kirin and its partner Ultragenyx sold off their rare disease PRV for $80.6 million. That’s a far cry from the go-go days of AbbVie’s historic $350 million PRV purchase in 2015.
Kyowa revealed Wednesday morning that the duo sold its PRV for a rare pediatric disease drug to an undisclosed buyer during Q2, according to a BioCentury report. The companies received the voucher in April when the FDA approved Crysvita to treat abnormally low levels of phosphate in the blood.
PRV prices have been falling ever since that major AbbVie deal, with Spark and Jazz announcing a PRV sale of $110 million this April. And before that, Sarepta auctioned its PRV to Gilead for $125 million. Ultragenyx itself sold a PRV to Novartis last December for $130 million.
The shrinking deal size may simply reflect supply and demand, with the FDA handing out more PRVs today than it used to. These PRVs have had a controversial history. Regulators have made it clear that they don’t like being forced to give the inside track at the FDA to any company that can afford to pay the price to hurry along a therapy that’s not so urgently needed. But lawmakers like the added incentive, claiming that it encourages innovation where it’s needed most.
As a result, the number of PRVs on the market has multiplied. Perhaps the most notable aspect of the vouchers has been the price they’ve commanded. The first voucher ever sold (by BioMarin) was purchased for $67 million in 2014. Months later, Gilead bought Knight’s voucher for $125 million, and the price climbed to an eye-popping $350 million in 2015, when AbbVie bought United’s voucher. But today’s price of $80.6 million falls awfully close to the beginning point in PRV sales. It will be interesting to see where this market goes.
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