U.S. expects first shipments of J&J vaccine to be delivered Tuesday

 Initial deliveries of the newly approved Johnson & Johnson COVID-19 vaccine should start on Tuesday, helping to boost vaccination rates across the country, senior administration officials said on Sunday.

The officials acknowledged that vaccination rates among minorities were “not where we ultimately want them to be”, but said federal officials were closely monitoring distribution to ensure it was equitable.

They urged everyone in the United States to get a vaccination as soon as it was their turn, and said Black and brown Americans should understand that safeguards had been put in place after past cases of discrimination in the medical field.

https://www.reuters.com/article/us-health-coronavirus-vaccine-usa/u-s-expects-first-shipments-of-jj-vaccine-to-be-delivered-tuesday-idUSKCN2AS0SE

Sarepta FDA OK for another Duchenne drug may lift sales post-earnings

 

• Sarepta Therapeutics announced Thursday that another one of its medicines for a rare muscular illness has received approval from the Food and Drug Administration.
• The medicine, which will be sold as Amondys 45, is now cleared for use in patients with Duchenne muscular dystrophy who have specific mutations in the gene responsible for making dystrophin, a key muscle-building protein. Sarepta estimates 8% of Duchenne patients have mutations in exon 45, the part of the gene targeted by Amondys 45.
• Like its two other approved products, Sarepta's latest offering is priced by weight, with treatment for a 20 kilogram child costing $300,000 per year. The addition of Amondys 45 means there are now medicines available to treat about 30% of Duchenne patients, according to data from CureDuchenne, a patient advocacy group sponsored in part by drugmakers like Sarept

The FDA's review for Amondys 45 was relatively quiet compared to Sarepta's two earlier products, known as Exondys 51 and Vyondys 53.

Exondys 51, in particular, stands as one of the most controversial approval decisions in the agency's recent history. Clinical testing showed the drug provided small increases in dystrophin, while leaving its purported benefits on patients' lives unclear. Several high-ranking officials within the FDA pushed for its rejection. But they were overruled by Janet Woodcock, the longtime head of the agency's drug review division and, currently, its acting commissioner.

Woodcock's decision not only cleared the path for Exondys 51, which became the first marketed drug specifically for Duchenne in 2016, but also set a precedent for subsequent medicines that work similarly.

Vyondys 53 followed, securing approval in 2019 after testing demonstrated similarly modest effects on dystrophin levels. That review also proved dramatic, as the FDA initially rejected Vyondys 53 because of safety concerns, only to about-face four months later. The agency then cleared Viltepso, a competing drug from Japan's NS Pharma, one year later.

Amondys 45 appeared to simulate more dystrophin production than Exondys 51 and nearly as much as Vyondys 53 had in their respective clinical trials. In testing, patients' dystrophin levels went from less than 1% at the start to about 2% after treatment with Amondys 45.

But it's still unclear, as with Sarepta's other drugs, whether Amondys 45 improves patients' health outcomes. The FDA notes that while dystrophin production is "reasonably likely" to predict a clinical benefit like improved motor function, research has yet to prove Amondys 45 does so. A placebo-controlled, confirmatory trial known as ESSENCE is ongoing, but won't produce results until 2024.

Each of Sarepta's drugs is supposed to bind to the gene that encodes for dystrophin, but only at specific points that have been altered because of mutations. For Amondys, that point is exon 45. For the other two drugs, it's exons 51 and 53, respectively. CureDuchenne estimates that 13% of patients are eligible for exon 51-skipping therapies, while exon 53- and 45-skipping therapies could each treat another 8% of the population.

Exondys 51 and Vyondys 53 helped to support Sarepta as the company built a lengthy list of drug programs, many of which focus on gene therapy research. Over the first nine months of 2020, Sarepta recorded $333 million in net product sales, a nearly 20% year-over-year increase, but posted a $365 million net loss. The company is scheduled to deliver its full 2020 earnings report next week.

Amondys 45 could lift Sarepta sales even further. Piper Sandler analyst Tyler Van Buren, for instance, expects the drug to bring in roughly $300 million in sales by 2025.

Yet the drug's approval also comes at a potentially challenging time for the company. The August approval of Viltepso provided the first direct source of competition to a Sarepta drug. And more recently, Sarepta's high-profile gene therapy program hit a major setback when treatment failed to significantly improve motor function in Duchenne patients.

The disappointing outcome was made all the more stinging as, the very same day, Pfizer disclosed that the first patient had been dosed in a large study of its competing Duchenne gene therapy. Sarepta shares fell more than 50% on the news, erasing about $6.5 billion from the company's market value.

Following the Amondys 45 approval announcement, Sarepta stock was up a little more than 1% at market's close Thursday, to trade a $85.25.

https://www.biopharmadive.com/news/sarepta-amondys-45-approved-fda-duchenne/595754/

2 Most Important Things to Look for in Novavax's Earnings

I'll be looking at Novavax's (NASDAQ:NVAX) earnings report on March 1. But my focus won't be on the financial aspects. Novavax doesn't have products on the market yet, which means the biotech company is yet to report an annual profit. At the same time, research and development expenses have generally increased throughout the years as programs advance through the pipeline. I don't expect a different story for 2020.

But what Novavax has to say in this report may change the story for 2021. That's because the company's coronavirus vaccine candidate is approaching the finish line. Novavax recently reported U.K. phase 3 trial results and just completed enrollment in a U.S. phase 3 trial. Possible regulatory authorizations sit on the horizon. Considering all of this, here are two key items to watch for when you read through Novavax's earnings report next week.

1. Potential timing of authorizations

Earlier this month, Novavax said regulatory agencies in the U.S., U.K., Europe, and Canada have begun the rolling review process. That means the agencies have started looking at Novavax's trial data so far -- and Novavax will submit additional results as they become available. Regulators can get a head start rather than waiting until all trials are complete to begin considering a potential product. And this speeds up the review process.

In the meantime, these agencies could decide to offer the investigational vaccine emergency authorization. So far, Novavax hasn't specifically spoken of the potential timing of such authorizations. But CEO Stanley Erck has said he's hoping the U.S. Food and Drug Administration (FDA) may consider the U.K. trial data as a basis for its Emergency Use Authorization (EUA). That would be instead of waiting for the U.S. trial results later next month. I'll be looking to see if Novavax speaks in more detail about this possibility during the earnings report.

And any other details about authorization timing are welcome, too. They would offer us clues about when to expect product launch -- and revenue.

2. Progress on a booster shot

Novavax is working on a solution to the problem of new coronavirus strains. In January, the company started studying a potential booster or combination vaccine to handle new strains. Novavax said earlier this month that it would choose candidates "in the coming days." The company plans to start clinical studies of a potential candidate in the second quarter. It's very possible Novavax will present us with more news on this project during its earnings report.

Here's why this element is so important: Today, new coronavirus strains present one of the biggest challenges for vaccine makers. Pfizer (NYSE:PFE) and Moderna (NASDAQ:MRNA) brought their vaccines to market under EUAs in December -- before the strains became prevalent. Since, in vitro tests have shown their vaccines work against the latest variants. But experts -- such as White House health advisor Dr. Anthony Fauci -- have said efficacy is lower when faced with these new strains.

The company that produces a vaccine or booster that effectively conquers new strains is sure to gain market share. Pfizer and Modera technically "won" the coronavirus vaccine race by finishing first and second, respectively. But Novavax could be the winner of the next stage of this race if its booster or combined vaccine is successful. Moderna, too, is investigating a booster to address new strains. It will be important to watch both companies as the programs move forward.

Any details regarding Novavax's coronavirus vaccine candidate are worth your attention during the upcoming earnings report. But the above two are crucial as they will determine when Novavax will start generating revenue and its chances of gaining a large share of the vaccine market. And all of this will define Novavax's next annual earnings report -- and its share price performance ahead.

https://www.fool.com/investing/2021/02/24/here-are-the-2-most-important-things-to-look-for-i/

Are Short-Sellers Right About Inovio?

 Inovio Pharmaceuticals (NASDAQ:INO) started the coronavirus vaccine race on the right foot. Investors cheered last spring when the company became one of the first to bring a coronavirus vaccine candidate into human trials. And then Inovio lost steam. Some investors were disappointed by a lack of detail in the phase 1 clinical trial report. Later in the year, the U.S. Food and Drug Administration (FDA) placed a partial clinical hold on its phase 2/3 trial.

Inovio is no longer a leader from a timeline perspective. Pfizer (NYSE:PFE) and Moderna (NASDAQ:MRNA) have brought vaccines to market. And Johnson & Johnson (NYSE:JNJ) and Novavax (NASDAQ:NVAX) are requesting regulatory authorization. Meanwhile, short-sellers are betting that shares of Inovio -- up more than 56% since the start of the year -- are going to fall. Are they right?

Let's take a look at the timeline first. Inovio launched its phase 2 study in early December 2020. At that time, the FDA lifted the partial clinical hold on phase 2 but maintained it on the phase 3 portion of the trial.

The FDA originally initiated a partial hold because it had questions about the phase 2/3 study and Inovio's administration device, which is called Cellectra. The handheld device uses electrical pulses to deliver Inovio's vaccine candidate into skin cells. Inovio has said it will answer all of the FDA's questions during the phase 2 portion of the trial. The FDA has 30 days to issue a response from that point. Even if both parties make an effort here, the big risk is that the hold will delay the start of phase 3.

Inovio hasn't provided an update. But we may expect more news when the company reports earnings on March 1.

Now, let's turn to the data. In December, Inovio said it had published phase 1 data in The Lancet's EClinicalMedicine journal. Investors' earlier concern had been a lack of detail regarding the production of neutralizing antibodies. In the report, Inovio said 84% of participants given the higher dose used in the trial developed neutralizing antibodies. On this metric, Inovio still lags behind rivals like Moderna and Pfizer. Those vaccines produced neutralizing antibodies in all participants. And antibody levels were higher than those of recovered coronavirus patients.

On a brighter note, Inovio reported T-cell responses in 100% of volunteers in the higher dose group. T-cells, known for killing infected cells, recognized various parts of the spike protein. The spike protein is what the coronavirus uses to infect healthy cells. Safety data was another positive. Inovio reported no serious adverse events.

Inovio versus rivals

At this point, Inovio's results don't look as strong as those of its more advanced competitors. Inovio is lagging behind from a timeline and data perspective. But new data could still save the day, in two ways: If the T-cell responses prove to be as important as neutralizing antibody levels, and if the vaccine can handle new strains. Phase 2 information should offer us a clearer picture.

Finally, one other feature may help bring Inovio's vaccine to the forefront. It may be kept at room temperature for more than a year. This is a big plus when it comes to storage and transportation. By comparison, Pfizer's vaccine must be kept at freezing temperatures and Moderna's requires refrigeration.

The ride has been bumpy for Inovio so far. And risk remains high. But if the FDA lifts the partial clinical hold and phase 2/3 data are strong, Inovio still may have a chance to carve out a spot in the coronavirus vaccine market.

So, what are the short-sellers saying? Short positions make up about 21% of Inovio's float. That's a decent percentage of investors betting on the stock's decline. But that's actually down from a peak of nearly 36% a few months ago. (In short selling, investors borrow shares to sell at the market price. They then must buy them back later -- hopefully at a lower price -- to return to the original lender.)

INO PERCENT OF FLOAT SHORT DATA BY YCHARTS

Investors may be regaining confidence in Inovio. Ultimately, though, short-sellers won't determine what happens with Inovio's stock. The clinical trial results will. Until Inovio reports more data, it's best to be cautious. But, considering the positive points I mentioned above, we might do so with a bit more optimism than several months ago.

https://www.fool.com/investing/2021/02/21/are-short-sellers-right-about-inovio/

Healthcare Technology's Digital Dilemma

 In the space of 1 year, the healthcare sector has undergone a veritable transformation in how care is delivered. The impetus for such a swift change was, of course, COVID-19. In the thrall of the pandemic, clinicians quickly embraced digital tools and technologies in order to avoid the health risks of face-to-face encounters. This will have broad implications across multiple sectors and populations.

The impact of the pandemic on the healthcare technology sector has been, and will continue to be, substantial. Recently, a colleague at Credit Suisse Securities forwarded me a copy of its publication projecting digital growth and innovations for 2021.

The surveys of investors and industry stakeholders on their expectations around various developments in digital health revealed that digital health adoption was "one of the silver linings in 2020," the report found. With the healthcare system under extreme stress during the pandemic, there has been a pressing need for technology and processes to boost capacity. This has led to what researchers described as a huge spike in adoption and use of digital health platforms, with once-underutilized digital tools and technologies now used to their full potential as staples in providing care.

In the coming year, the analysts predict a realignment in digital health as interrelated trends drive digital health growth and innovation. Their list of potential positive trends is lengthy and well within the realm of probability – e.g., continued adoption of virtual care; acceleration in employer activism; increased focus on addressing the mental health crisis (one that has been compounded by the pandemic); a continued shift to home-based care; continued progress on addressing waste, fraud, and abuse in the healthcare system; a reinvention of primary care; a shift to value-based care; and a recognition of the importance of cybersecurity.

While the preceding report shows industry stakeholders to be very bullish on virtual care, telemedicine, and healthcare information technology in general, a broader context takes into account the effects of these changes on various patient populations. The "great digital divide" is a serious ongoing issue for many in this country, and particularly for the elderly population. Herein lies the dilemma.

For instance, data from the National Poll on Healthy Aging conducted at the University of Michigan Institute for Healthcare Policy suggest that, although most major health systems are using patient portals (e.g., MyChart) to schedule COVID-19 vaccinations, many older adults are unable to set up these accounts without assistance. The poll found that 45% of adults ages 65 to 80 (and 42% of all adults ages 50 to 80) reported they had not set up an account with their provider's portal system. Although the percentages represent a small improvement over time, it is clear that more encouragement and assistance is needed. For example, a trusted adult might be appointed as a proxy to access accounts of those without computers, or of those who need help navigating technology.

Of particular concern, wide gaps in patient portal use were observed among various subsets of older adults who have the highest risk for severe cases of COVID-19 if unvaccinated. Almost 50% of Black older adults and 53% of Hispanic older adults lacked patient portal accounts as of June 2020, compared with 39% of White older adults.

The most significant gap in patient portal use was related to income. Approximately 54% of older adults with income less than $60,000/year did not have a patient portal account, whereas only 35% percent of older adults with income over $60,000 lacked an account. A similar gap was noted with regard to the level of education: 53% of those with less than a high school education lacked a patient portal, compared with 31% of those with college degrees.

In polls prior to the pandemic, older adults reported unawareness, lack of comfort using technology, and dislike of communicating about their health online as reasons for not setting up a patient portal account. Although the recent poll shows an increase in the percentage of older adults reporting participation in telehealth visits (4% in 2019 versus 26% in August 2020), the digital divide persists.

From my perspective, it seems an ideal time to pause and take stock. Is the plethora of "MyChart" accounts making things even more complicated than having no patient portals at all? Does the continued proliferation of patient portal platforms without interconnectivity make sense? To date, there is no coherent policy on electronic medical records. Shouldn't we be working on one?

Perhaps connectivity should be considered a "vital sign" -- something we routinely ask about, and check on, at every patient visit. Without connectivity, or even intermittent connectivity, we won't be able to maintain health in the digital world of the future.

https://www.medpagetoday.com/columns/focusonpolicy/91392

States Need Vaccination Results, Not Headlines

 On the day that New York governor Andrew Cuomo won his Emmy, the governor of West Virginia announced a plan to vaccinate his state. Since then, Governor Jim Justice’s state has boasted one of the nation’s highest vaccination rates, striking a critical blow to Covid-19 and displaying a distinct heartland model of leadership. While leaders in coastal hubs drew headlines for their press conferences and top-down leadership styles, a number of heartland governors paired efficient government with trust in their communities and people—both to vaccinate and to educate.

Gretchen Garofoli, a local pharmacist in Morgantown, West Virginia, administered one of her state’s first Covid-19 vaccinations—a jab in the arm of a nursing-home patient— on December 15, weeks before any other state could do the same. “It was a beacon of hope,” Garofoli later told NPR. West Virginia is the only state in the country to use independent pharmacies to deliver shots; pharmacists in these communities often know their patients by name. “The more the state collaborates with those at the local, community level, the greater the vaccine uptake,” concluded Christopher Martin, a professor at the West Virginia University School of Public Health. As of mid-February, West Virginians have received more than 400,000 doses of the coronavirus vaccine, and some 8 percent of the population has received at least two jabs (a rate second only to Alaska’s 9 percent).

Smaller, redder states have been more successful than media coverage would suggest. Look at the rankings for Covid-19 vaccine doses given and you’ll see West Virginia, Alaska, North and South Dakota, and Utah ranking high. These states benefit from having fewer people to vaccinate—and only a few large health systems operate in North and South Dakota, allowing for easier logistics and nimbler vaccination plans— but they aren’t without geographical difficulties. Yet many of their governors have been pilloried in the press. South Dakota governor Kristi Noem, a common target, began planning her state’s vaccine rollout in mid-summer, well before the federal government gave its own guidance. Noem’s team held daily coordination meetings with local partners, who, in turn, led the distribution and shared lessons learned along the way.

The success of heartland states compares favorably with the performance of New York and California. A large hospital system or pharmacy network is by itself no guarantee of success in public health, as NYC Health + Hospitals likely knows. Meantime, California—whose governor, Gavin Newsom, was celebrated as “#PresidentNewsom” on Twitter last April—has consistently lagged the nation in vaccinations, at one point ranking dead last. In early January, Newsom promised 1 million doses in ten days; two weeks later, the governor acknowledged that he had no idea if the state had met its goal. “High-tech California can’t seem to efficiently administer the vaccine at scale,” summarized NPR. The Golden State had prided itself on stringent lockdowns and mask mandates, yet it experienced a winter surge on the way to becoming the first state to surpass 3 million cases. Things got so bad in January that Southern California’s air regulator suspended cremation limits to deal with the backlog of bodies from Covid-19.

Blue states and counties also have failed to reopen their public schools. As of December, according to one survey, some 42 percent of school-age children nationwide were still learning entirely remotely, while 17 percent were learning in-person only part-time. Data from the Centers for Disease Control found little evidence that schools contribute meaningfully to viral outbreaks. Yet school reopenings are driven more by politics than by science: the likelihood of in-person teaching falls dramatically in counties that voted for Hillary Clinton in 2016 or have strong teachers’ unions. The California Teachers Association refused to let its members return to the classroom until they were vaccinated. What the science does tell us is that closing school buildings has substantially harmed academic performance among children. In Montgomery County, Maryland, where schools remain shuttered, failure rates for African-American and Latino children from low-income families are more than six times higher than they were a year ago; white students have seen minimal declines. This week, the county’s teachers’ union voted to keep classrooms closed.

Other states have taken a different tack. “You can’t say ‘follow the science’ and keep schools closed,” Tennessee governor Bill Lee said last month. “You can’t say ‘I believe in public education’ and keep schools closed.” Lee had called a special session of the state’s General Assembly to help students and to give teachers the resources they need to reopen classrooms. Some cities resisted at first: in Memphis, the Shelby County Schools superintendent said that state lawmakers were “hurting my children,” while metro Nashville’s school district claimed that it needed more resources before committing to reopening. Lee’s office responded by asking what had happened to Nashville’s $110 million in unspent federal education grants. Ultimately, the state approved $160 million in aid and enacted a phonics-based program to boost literacy, among other measures.

Now that the pandemic has dealt severely with every state in the country, it’s fair to evaluate individual states’ performance—and it’s clearer than ever which ones are responding effectively. “West Virginia has been the diamond in the rough that a lot of people have missed,” said Governor Justice. He’s right. Justice and other leaders in heartland states are vaccinating and educating like their residents’ lives depend on it, which they do.

Michael Hendrix is director of state and local policy at the Manhattan Institute.

https://www.city-journal.org/many-heartland-states-outperforming-coastal-hubs-in-covid-fight

Advantages of the J&J vaccine

 Chalk up another victory for America’s innovative pharmaceutical sector in the battle against Covid-19. An FDA analysis has found that Johnson and Johnson’s vaccine is both safe and effective, setting up a likely emergency-use authorization for the product. This would add a third vaccine to the American market, following the introduction of vaccines from Pfizer and Moderna in December. The J&J vaccine will not only add much needed supply but also offer some significant advantages over the other two shots.

The top-line number for the J&J vaccine—67 percent effectiveness in decreasing moderate to severe Covid-19 illness—might seem to suggest that it is inferior to the Pfizer and Moderna vaccines, which boast more than 90 percent effectiveness. But the vaccine trials are not directly comparable. The Pfizer and Moderna trials were conducted earlier, when fewer and probably less dangerous virus variants were extant. More importantly, J&J’s vaccine is very effective at preventing severe illness: 77 percent effective in reducing cases occurring 14 days after vaccination and 85 percent for cases 28 days after vaccination. Notably, no deaths or hospitalizations occurred among the trial’s vaccine recipients. As with the Pfizer and Moderna vaccines, side effects were mostly minor and brief.

The J&J vaccine also offers important advantages. First, it is a single dose rather than the currently required two doses for Pfizer and Moderna. This makes it far more likely people will sign up for it, since a single dose is less of a time commitment and inconvenience than a two-dose regimen. A single dose also eliminates the logistical difficulties of scheduling second doses and ensuring that they are given.

The J&J vaccine can also be transported and stored for up to three months using regular refrigeration, unlike the freezers needed for the other two vaccines. This will make it much easier to get doses to rural areas and countries with less well-developed distribution systems.

Finally, the J&J vaccine is significantly less expensive than its competitors. Based on U.S. government purchases, it will cost $10 per dose, while Pfizer comes in at $19.50 per dose and Moderna at $25 to $37 per dose. And since the latter two require two doses, the relative product cost of vaccination is $10 for J&J, $39 for Pfizer, and $50–$74 for Moderna, plus the added labor costs of administering second doses.

J&J’s vaccine uses a relatively new technology: an adenovirus vector, which cannot replicate or cause illness, inserts genetic material (DNA) into patients’ cells, instructing them to manufacture the coronavirus spike protein, which then elicits an immune response. This differs from the Pfizer and Moderna vaccines, which both rely on a new mRNA technology. That all three could be developed, tested, and made available in hundreds of millions of doses within a year of the pandemic’s onset is a testament to the power of the free market to find innovative solutions to pressing human problems.

Joel M. Zinberg is a senior fellow with the Competitive Enterprise Institute and an associate clinical professor of surgery at the Mount Sinai Icahn School of Medicine. He was a senior economist and general counsel at the White House Council of Economic Advisors, 2017-2019.

https://www.city-journal.org/advantages-of-the-johnson-and-johnson-covid-vaccine