Biotech week ahead, May 6

After eking out a modest gain in the week ended April 27, the iShares NASDAQ Biotechnology Index (ETF) (NASDAQ: IBB) pulled back this week amid some not-so-encouraging earnings reports and negative regulatory decisions.

Will the lean trot end? Stay tuned to the upcoming week’s developments on the biotech front.

Medical, Health Care And Biotech Conferences

• 2018 Disruptive Growth and Healthcare Conference: May 8-9 in New York City.
• Deutsche Bank 43rd Annual Health Care Conference: May 8-9 at the InterContinental Boston Hotel.

PDUFA Dates

The FDA is set to rule on Lipocine Inc (NASDAQ: LPCN)’s NDA for tlando, which is tested for hypogonadism. The PDUFA action date is May 8.

Adcom Schedule

The Endocrinologic and Metabolic Drugs Advisory Committee is scheduled to meet May 10 to discuss the safety and efficacy of the Akcea Therapeutics Inc (NASDAQ: AKCA)-Ionis Pharmaceuticals Inc (NASDAQ: IONS) combine’s subcutaneously injected volanesoren solution for patients with familial chylomicronemia syndrome.

Earnings

Monday, May 7

• ArQule, Inc. (NASDAQ: ARQL)
• Fulgent Genetics Inc (NASDAQ: FLGT)
• MacroGenics Inc (NASDAQ: MGNX)
• Ultragenyx Pharmaceutical Inc (NASDAQ: RARE)

Tuesday, May 8

• Arena Pharmaceuticals, Inc. (NASDAQ: ARNA)
• Opko Health Inc. (NASDAQ: OPK)
• Aclaris Therapeutics Inc (NASDAQ: ACRS)
• Aerie Pharmaceuticals Inc (NASDAQ: AERI)
• Alder Biopharmaceuticals Inc (NASDAQ: ALDR)
• Amicus Therapeutics, Inc. (NASDAQ: FOLD)
• ANI Pharmaceuticals Inc Common Stock (NASDAQ: ANIP)
• Applied Genetic Technologies Corp (NASDAQ: AGTC)
• Arrowhead Pharmaceuticals Inc (NASDAQ: ARWR)
• BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX)
• Clovis Oncology Inc (NASDAQ: CLVS)
• Enanta Pharmaceuticals Inc (NASDAQ: ENTA)
• Epizyme Inc (NASDAQ: EPZM)
• Eyepoint Pharmaceuticals Inc (NASDAQ: EYPT)
• Flexion Therapeutics Inc (NASDAQ: FLXN)
• Haemonetics Corporation (NYSE: HAE)
• Infinity Pharmaceuticals Inc. (NASDAQ: INFI)
• Jazz Pharmaceuticals PLC (NASDAQ: JAZZ)
• Kindred Biosciences Inc (NASDAQ: KIN)
• Ligand Pharmaceuticals Inc. (NASDAQ: LGND)
• Loxo Oncology Inc (NASDAQ: LOXO)
• Melinta Therapeutics, Inc. (NASDAQ: MLNT)
• Merrimack Pharmaceuticals Inc (NASDAQ: MAC)
• Momenta Pharmaceuticals, Inc. (NASDAQ: MNTA)
• Ocular Therapeutix Inc (NASDAQ: OCUL)
• Oncomed Pharmaceuticals Inc (NASDAQ: OMED)
• Prothena Corporation PLC (NASDAQ: PRTA)
• Regenxbio Inc (NASDAQ: RGNX)
• Revance Therapeutics Inc (NASDAQ: RVNC)
• Sangamo Therapeutics Inc (NASDAQ: SGMO)
• Supernus Pharmaceuticals Inc (NASDAQ: SUPN)
• Syndax Pharmaceuticals Inc (NASDAQ: SNDX)
• T2 Biosystems Inc (NASDAQ: TTOO)
• Vital Therapies Inc (NASDAQ: VTL)
• Xenon Pharmaceuticals Inc (NASDAQ: XENE)

Wednesday, May 9

• Endocyte, Inc. (NASDAQ: ECYT)
• FibroGen Inc (NASDAQ: FGEN)
• Jounce Therapeutics Inc (NASDAQ: JNCE)
• Neos Therapeutics Inc (NASDAQ: NEOS)
• Mylan NV (NASDAQ: MYL)
• AcelRx Pharmaceuticals Inc (NASDAQ: ACRX)
• Adaptimmune Therapeutics PLC – ADR (NASDAQ: ADAP)
• Arcadia Biosciences Inc (NASDAQ: RKDA)
• Array Biopharma Inc (NASDAQ: ARRY)
• Audentes Therapeutics Inc (NASDAQ: BOLD)
• Collegium Pharmaceutical Inc (NASDAQ: COLL)
• Foamix Pharmaceuticals Ltd (NASDAQ: FOMX)
• Glaukos Corp (NYSE: GKOS)
• Horizon Pharma PLC (NASDAQ: HZNP)
• Inovio Pharmaceuticals Inc (NASDAQ: INO)
• Portola Pharmaceuticals Inc (NASDAQ: PTLA)
• PTC Therapeutics, Inc. (NASDAQ: PTCT)
• Puma Biotechnology Inc (NASDAQ: PBYI)
• Viking Therapeutics Inc (NASDAQ: VKTX)

Thursday, May 10

• Depomed Inc (NASDAQ: DEPO)
• Karyopharm Therapeutics Inc (NASDAQ: KPTI)
• Nektar Therapeutics (NASDAQ: NKTR)
• Athersys, Inc. (NASDAQ: ATHX)
• BioDelivery Sciences International, Inc (NASDAQ: BDSI)
• BioTime, Inc. (NYSE: BTX)
• CareDx Inc (NASDAQ: CDNA)
• Halozyme Therapeutics, Inc. (NASDAQ: HALO)
• Strongbridge Biopharma plc (NASDAQ: SBBP)
• Synergy Pharmaceuticals Inc (NASDAQ: SGYP)
• Catabasis Pharmaceuticals Inc (NASDAQ: CATB)
• RXi Pharmaceuticals Corp (NASDAQ: RXII)
• Catalyst Pharmaceuticals Inc (NASDAQ: CPRX)
• Fibrocell Science Inc (NASDAQ: FCSC)

IPO

Immuno-oncology company Abpro is set to offer 4 million shares. The shares are to be listed on the Nasdaq under the ticker symbol ABP.

Evelo Biosciences, which focuses on therapies that act on the gut-body network, is offering 5.3 million shares. Shares are to be listed on the Nasdaq under the ticker symbol EVLO.

https://bit.ly/2IpLbVA

Fed’s Quarles: Paying ‘a lot’ of attention to spread of machine learning in finance

Federal Reserve vice chair for supervision Randal Quarles said the central bank is in the early stages of studying how the expanding use of machine learning in the financial sector may change its regulatory approach, but that so far it fits within the existing regulatory framework.

“We are paying a lot of attention … within our existing framework. To the extent you have a machine learning tool that is interacting with customers we want to make sure that the traditional protections are being complied with,” Quarles said.

While the Fed may need “more specialised responses” to the use of artificial intelligence or computer algorithms in assessing risk and making credit decisions, he said he was hesitant to impose “bounds” on what institutions can do, rather than develop “milestones” or circuit breakers that would pause any process that was producing bad results.

https://bit.ly/2HWGM9g

Dentsply Sirona Q1 Results Top Estimates; But Cuts 2018 Outlook

Dentsply Sirona Inc (XRAY) reported that its net income attributable to the company for the first quarter of 2018 rose to $81.2 million or $0.35 per share, from $59.8 million or $0.26 per share in the first quarter of 2017.

“Despite a solid performance in many businesses and regions, overall results for the quarter were disappointing due headwinds in the U.S. Technologies & Equipment business,” said Donald M. Casey, Jr., Chief Executive Officer of Dentsply Sirona.

“Our revised guidance reflects our expectations of continued headwinds in that important business and more importantly the steps we are taking to return the company to growth. Our priority for the remainder of 2018 is to invest in comprehensive strategies designed to help accelerate demand in this market,” said Donald Case.

On an adjusted basis, excluding certain items, non-GAAP net earnings per share were $0.45 compared to $0.49 in the first quarter of 2017. Analysts polled by Thomson Reuters expected the company to report earnings of $0.42 per share for the quarter. Analysts’ estimates typically exclude special items.

Net sales were $956.1 million up 6.2% from $900.5 million last year. Wall Street expected revenues of $940.8 million for the quarter. Sales growth was negative 1.1% on a constant currency basis.

For 2018, the company now expects adjusted earnings per share to be in the range of $2.55 to $2.65 per share. Analysts expect annual earnings of $2.70 per share. 2018 guidance assumes about 2% constant currency revenue growth for the year. Previously, the company expected adjusted earnings per share for 2018 in the range of $2.70 to $2.80 per share. The 2018 guidance assumed about 3% constant currency revenue growth for the year.

On April 25th, 2018, Dentsply Sirona’s Board of Directors authorized an increase of $500 million of common stock to its share repurchase program, reflecting the company’s strong balance sheet and free cash flow generation. This authorization is in addition to the $500 million previously authorized by the Board of Directors on February 14, 2018, bringing the total share repurchase authorization up to $1 billion of common stock.

https://bit.ly/2HZSu2Q

Quiet rest after learning helps us to remember the fine details

Most of us know that without sleep, we are unable to create new memories. But is simply resting — without falling into the dreamy state — for only 10 minutes after learning something enough for us to memorize it in fine detail? Recent research suggests so.

Struggling to remember the details? Try resting quietly after learning, a new study suggests.

Sleep and memory are loving bedfellows. Sleep “blocks” our brain’s mechanisms of forgetting, lowering the neurotransmitter dopamine, and therefore facilitating memory formation.

Furthermore, recent studies have revealed that sleep is key for consolidating memories that we made while awake, as well as for preserving the brain’s ability to learn new things in the future.

For instance, a study revealed that during sleep, our synapses relax, staying supple and flexible, which maintains our brain’s neuroplasticity and ability to learn.

On the other hand, poor sleep leads to rigid synapses and an impaired ability to learn new things in the long run.

Perhaps even more surprisingly, researchers have recently been able to interfere with the memory consolidation process that takes place during sleep by scanning people’s brains, selectively choosing certain memories, and reinforcing them.

But could a state of simple, restful wakefulness be just as beneficial for new memory formation? A new study — jointly conducted by Michael Craig, a research fellow at the Heriot-Watt University in Edinburgh, United Kingdom, and Michaela Dewar, a research leader and assistant professor at the same university — suggests that it can.

“Recent research,” says Craig, “suggests that the memory system strengthens weak new memories by ‘reactivating’ them, where brain activity first observed during learning automatically reappears in the minutes that follow.”

Based on the findings of their own research, the scientists say, “This appears especially true during sleep and quiet resting, when we’re not busy taking in any new sensory information.”

What’s more, the new research suggests not only that a period of quiet restfulness helps us to remember new things, but that such a rest is crucial for retaining the fine details.

The new findings were published in the journal Nature Scientific Reports.

Studying fine memories

Craig and Dewar designed a memory test to assess the ability to retain finely grained information. They asked 60 young male and female participants — aged 21, on average — to look at a set of photos.

They were asked to discern between “old” photos and “similar” ones. If the participants’ ability to retain fine nuances was good, they would say the photos were “similar.”

“However,” explains Craig, “if not-so-detailed memories are stored, people should miss the subtle differences in similar photos, and mistake them for ‘old’ photos.”

He goes on to summarize these “interesting” findings, saying, “Younger adults who quietly rested in the minutes that followed the photo presentation were better at noticing subtle differences in similar photos.”

This, he explains, suggests “that these individuals stored more detailed memories, compared to those who did not rest.”

“This new finding provides the first evidence that a brief period of quiet rest can help us to retain more detailed memories.”

Michael Craig

He adds, “We think that quiet resting is beneficial because it is conducive to the strengthening of new memories in the brain, possibly by supporting their automatic reactivation.”

However, Craig admits that the mechanisms behind this surprising phenomenon remain a mystery.

“[W]e don’t know exactly,” he goes on to conclude, “how this rest-related memory strengthening works. Specifically, it remained unknown whether quiet resting only allows us to retain more information, or whether it also helps us to retain more detailed memories.”

https://bit.ly/2wiCpE8

Medicare’s Readmissions Penalty Draws More Fire

Hospital readmissions are not monolithic, and Medicare should change its readmissions penalty program time frame from 30 days to 7 days, researchers said.

The researchers from multiple U.S. institutions stated that the Medicare Hospital Readmissions Reduction Program (HRRP) often penalizes hospitals for patient outcomes that are out of their control.

“We found that readmissions within the first 7 days after hospital discharge were more likely to be preventable than those within a late period of 8 to 30 days,” they wrote in the Annals of Internal Medicine. “Early readmissions were more likely to be amenable to interventions within the hospital and to be caused by factors for which the hospital is directly accountable, such as problems with physician decision making.”

Outpatient facilities and home caregivers were more likely to be accountable for readmissions from 8 to 30 days, the researchers wrote.

“Late readmissions were more likely to be amenable to interventions outside the hospital and to be caused by factors over which the hospital has less direct control, such as appropriate monitoring and managing of symptoms after discharge by the primary care team,” they stated.

The study covered 10 academic medical centers from April 201 2 through March 2013 and included 822 adult patients. Of those, 301 patients (36.6%) were readmitted within 7 days after discharge;521 (63.4%) were readmitted eight to 30 days after discharge; and 36.2% of early readmissions versus 23.0% of late readmissions were deemed preventable.

The researchers found that faulty physician decision-making was the number one cause of early readmissions, associated with 28.9% of the cases.

Three primary variants of errant decision making were identified:

• Premature discharge: 16.3% of cases
• Inadequate treatment during hospital stay: 14.3%
• Missed diagnoses: 10.6%

Difficulty monitoring and managing symptoms was the number one cause of late readmissions, associated with 33.2% of cases. The researchers identified three primary variants of monitoring and managing difficulties:

• Lack of disease monitoring: 12.7% of cases
• Overly long wait times for follow-up appointments: 10.0%
• Inability to make follow-up appointments: 10.9%

The researchers said their data indicate several reasons why the HRRP time-frame should be switched from 30 days to 7 days.

First, they found a significant difference in the preventability of early and late readmissions in the 30-day time-frame after discharge. “Early readmissions were associated with double the odds of preventability compared with late readmissions,” they noted.

Second, a pair of physician adjudicators who reviewed the readmissions cases found hospitals were the best site to intervene and prevent early readmissions. The physician educators found outpatient clinics and home were the best settings to prevent late readmissions.

Third, the researchers found that erroneous physician decision-making and premature discharge were leading causes of early readmissions.

“Taken together, these findings suggest that readmissions in the week after discharge are more preventable and more likely to be caused by factors over which the hospital has direct control than those later in the 30-day window,” they wrote.

Beyond narrowing HRRP’s 30-day readmissions window to 7 days, the researchers also offer five recommendations to promote readmissions prevention:

• Hospitals should try to decrease cognitive errors that impact diagnosis and treatment
• The impact of hospital efforts to increase throughput on premature discharge should be examined
• Outpatient facilities should boost multidisciplinary care management for post-discharge monitoring of patients after discharge
• Access to primary care clinicians should be expanded
• Accountability for readmissions 30 days after discharge should be shared between outpatient and inpatient facilities

“Shared accountability over the 30 days, possibly with weighted penalties by readmission timing, would engage outpatient practices in readmission reduction efforts and reduce unfair financial penalties on hospitals.”

The study was funded by the American Association of Medical Colleges. Harvard Catalyst, The Harvard Clinical and Translational Science Center, and the NIH.

• Primary Source

  Annals of Internal Medicine

  Source Reference: Graham KL, et al “Preventability of early versus late hospital readmissions in a national cohort of general medicine patients” Ann Intern Med 2018; DOI:10.7326/M17-1724.

https://bit.ly/2rqq1Mv

Privacy is dead: Here's what comes next

Short of living in a remote hut while forsaking cellphones, the internet and credit cards, there is no longer any way that you, as an individual, can prevent marketers, governments or malicious actors from gathering and using comprehensive, personally identifying information about you.

There are things you can do to reduce the amount of information you leak. You could, for example, ask Facebook to delete your browsing history, or perhaps one day you’ll be able to pay the company to not track you. But keeping up requires more time, sophistication and paranoia than most of us can muster. And it still isn’t 100% effective.

There has been a sea change in how data about all of us is gathered and distributed. Those who want information about us no longer have to observe us directly. They can now collect our data from our friends, contacts — even people we don’t know. Preserving privacy used to be about protecting ourselves and our devices. Now, the information is outside of our control, stored in address books of friends and latent in our social networks and family ties.

As in cybersecurity, protection of some of our most important personal data now depends on protecting the weakest link in the systems of which we are a part.

Genuine privacy or anonymity is over, if we ever had it, says Paul Francis, a researcher at the Max Planck Institute for Software Systems in Germany. “All we can really hope to do is, piece by piece, get better at protecting privacy,” he adds.

Those pieces might come from unexpected places. The very companies currently taking fire for collecting and disseminating our personal information — Google and Facebook — could someday be stewards of it, or else be disrupted by those who are willing to.

Why our data isn’t safe

The Cambridge Analytica scandal — where 270,000 people who downloaded an app led to a data breach for 87 million Facebook users — is the first large-scale example of the importance of maintaining “group privacy,” says Yves-Alexandre de Montjoye, head of the computational privacy group at Imperial College London.

In a hypothetical example, Prof. de Montjoye’s group reported that if just 1% of cellphones in London were compromised with malware, an attacker would be able to continuously track the location of more than half the city’s population.

Our vulnerability to such attacks is compounded by another phenomenon: It’s easy to identify us with just a tiny amount of information, making it impossible to render any pool of data about a population anonymous.

Facebook, Google and others in the ad-tech space say they take pains to “anonymize” the data they collect on us. This anonymization consists of mathematical tricks allowing them to market to us while assuring that they can’t identify us for other purposes — and no one else can either.

But time and again, researchers with access to pools of anonymized data have found ways to identify individuals within it, Prof. de Montjoye says.

The Max Planck Institute’s Dr. Francis co-founded a company, Aircloak, to develop software to protect data. Diffix, as it’s called, sits between a database and its owners, allowing them to make specific queries but never revealing the whole database. It should allow firms like banks to protect user data internally, in a way that makes them compliant with sweeping new privacy rules under Europe’s General Data Protection Regulation, according to Dr. Francis and Sebastian Probst Eide, Aircloak’s chief technical officer.

But even special software can’t help online advertising companies get fully compliant with the European regulations — at least not yet. Early on, the Aircloak team abandoned an attempt to anonymize targeted advertising, because there are so many transactions that can identify a person, Dr. Francis says. For example, a company advertising medication for certain conditions could inadvertently identify people who click on the ad and then potentially share that information with others in the chain of custody of personal data.

Big Tech: From villain to savior?

If technology can’t keep personal info out of the hands of the tech giants, the seemingly paradoxical alternative is to collect all of that personal info in one place, so that a central authority can handle it.

That central authority could be a government. Estonia, for one, has created a cryptographically secure universal ID to which any kind of personal data can be attached, from taxes and financial records to health data. As a result, Estonians can e-file their taxes in about 5 minutes, patients can view a digital paper trail of everyone who has ever accessed or altered their medical records, and even non-Estonian residents can become “e-residents” who gain many of the online rights and privileges afforded to Estonia’s citizens.

Such an authority could be granted to a tech giant like Facebook, Google, Apple or Amazon.

Giving companies like Facebook and Google even more of our data might seem like the opposite of protecting it. But both companies already have the start of the infrastructure required to support such a massive undertaking: It’s the identity systems that allow us to log into other sites and apps using our Facebook, Google or Amazon credentials.

This could be an opening for Apple, Amazon or some new entrant to become a personal-data custodian. The idea of a centralized repository (a.k.a. personal-data store), which marketers would have to seek permission to access, has been proposed before. But these projects — which depend on some companies having our data, and others not — haven’t taken off, since gathering and using our data is both legal and lucrative.

With GDPR, Europe has an opening for such a service, and if any of the privacy regulations proposed in the U.S. gain traction, conditions could ripen here as well. It’s also possible people could experience a change of mind-set — realizing some data is fair game but some tracking goes too far — to create the kind of demand for privacy-protecting products and services that is currently scarce.

https://bit.ly/2HW94k5

J&J Janssen has positive Phase 3 results for depression nasal spray

The Janssen Pharmaceutical Companies of Johnson & Johnson announced the results from two Phase 3 clinical studies of the investigational compound esketamine nasal spray in patients with treatment-resistant depression. Data from a study in adults with treatment-resistant depression showed that flexibly dosed esketamine nasal spray plus a newly initiated oral antidepressant demonstrated a statistically significant, clinically meaningful rapid reduction of depressive symptoms as compared to placebo nasal spray plus a newly initiated oral antidepressant. The study defined treatment-resistant as patients who had not responded to two or more currently available antidepressants of adequate dose and duration in the current episode of depression. Data from a second study, in elderly patients aged 65 and older with treatment-resistant depression, which is the first study of its kind, showed treatment with flexibly dosed esketamine plus a newly initiated oral antidepressant demonstrated clinically meaningful effects compared to placebo nasal spray plus a newly initiated oral antidepressant. However, the study narrowly missed statistical significance for its primary efficacy endpoint. If approved by the U.S. Food and Drug Administration, esketamine would be one of the first new approaches to treat refractory major depressive disorder available to patients in the last 50 years. These findings represent two of the five Phase 3 studies that comprise Janssen’s treatment-resistant depression program with esketamine nasal spray. The results from these studies will inform regulatory filings for esketamine nasal spray in treatment-resistant depression, for which Janssen has received Breakthrough Therapy Designations from the U.S. FDA. Data from other Phase 3 studies will be presented later in 2018.

https://bit.ly/2weqffz