Goldman Sachs bought – then sold – a noteworthy stake in a tiny Danish biotech firm that became a meme stock last week.
Orphazyme, a Copenhagen, Denmark-based biotech firm, said the bank’s holdings increased to 5.58% of the company’s shares as of June 16. Then, just a day later, the bank’s stake fell below the 5% threshold that triggers a filing.
Orphazyme’s American Depository Shares rallied 61% on June 16 and then fell the following three days. The bank didn’t respond to Insider’s request for comment on the matter.
The stock has been on a wild ride since June 10 when it surged as much as 1,387% in a single trading day. The company rose to popularity on Reddit’s Wall Street Bets that day, though some retail traders questioned if the trade was a hedge-fund pump-and-dump.
In a statement about the share surge, the company said it wasn’t aware of any material changes to the business that would explain the stock move and warned investors they could lose money if the price dropped.
The price of the company’s American Depository Shares has surged then plummeted twice this month. Wednesday, the price was on the rise again, gaining as much as 53%.
The company, founded in Denmark in 2009, is researching and developing heat-shock proteins to treat people living with rare neurodegenerative diseases, according to its website. It went public in 2017. Orphazyme did not immediately respond to Insider’s request for comment.
Orphazyme isn’t the only small firm to be added to the meme-stock basket recently. This week, another small biotech firm, Atossa Therapeutics, saw a huge rally when retail traders poured into the stock, while a tiny Texas oil driller that’s previously warned it could go out of business also surged.
The meme-stock frenzy began earlier this year when investors mobilized on social media like Reddit caused GameStop’s share price to soar. Since then, many other stocks have been pulled into the frenzy.
A COVID-19 vaccine candidate developed by the German biotech CureVac failed in a critical late-stage study, the company said Wednesday.
It’s the first failure of a major vaccine candidate in a final-stage trial. CureVac said an interim analysis showed the shot was 47% effective, falling short of the study’s goals and the minimum bar for what US regulators find approvable.
The development is a setback to the world’s immunization efforts, as European officials had previously reached deals to acquire up to 405 million doses of the shot.
Despite the disappointing result, CureVac CEO Franz-Werner Haas said the company plans to go “full speed for the final readout.” The trial is still ongoing and the final vaccine efficacy figure may vary as more COVID-19 cases are tallied.
CureVac, which is backed by The Bill & Melinda Gates Foundation, saw its stock price plummet following Wednesday’s announcement. Shares were down more than 50% in post-market trading. The foundation owns about 3.1 million shares of CureVac, or 1.7% of the company, according to data compiled by Bloomberg.
An independent biostatistics expert said it will be nearly impossible for CureVac’s study to still produce success. The 47% estimate of efficacy is based on 134 COVID-19 cases among study participants. “It’s not going to change dramatically,” Natalie Dean, a University of Florida biostatistician, told The Times.
CureVac blames variants, even as other vaccines hold up against different strains
In a statement, CureVac leaders said that the abundance of virus variants played a role in the result. Only one of the 134 analyzed cases resulted from the original strain of the SARS-CoV-2 virus, the company said.
“While we were hoping for a stronger interim outcome, we recognize that demonstrating high efficacy in this unprecedented broad diversity of variants is challenging,” CureVac CEO Franz-Werner Haas said in a statement.
The disappointing result is surprising given some of the similarities CureVac’s experimental vaccine candidate had with other, highly effective immunizations. CureVac’s shot is a messenger RNA vaccine, a new technology platform that’s also used by Moderna and Pfizer-BioNTech.
Those shots proved to be more than 90% effective in late-stage trials last year. They also appear to protect people against some major virus variants.
Other vaccines have also shown success against variants. Novavax, for instance, announced earlier this week its two-dose shot was 90% effective in a late-stage study. What’s more, Novavax’s vaccine was about 93% effective in preventing illnesses caused by variants of concern or variants of interest, the company said.
A quiet existence, until the pandemic
Since its founding in 2000, CureVac had a largely quiet existence until the pandemic. As one of the first companies trying to develop a COVID-19 vaccine, CureVac’s CEO was invited to the White House in March 2020. Shortly after, reports circulated that the US had offered a “large sum” for access to its vaccine program. CureVac disputed the reports. The company also cycled through three CEOs in the span of a week.
Tesla CEO Elon Musk has also drawn attention to CureVac with his tweets. A Tesla subsidiary is working with CureVac in building a “prototype of an automated manufacturing unit,” Insider reported in July.
In its Wednesday press release, CureVac focused attention on its second-generation coronavirus vaccine. Pharma giant GlaxoSmithKline has partnered with CureVac on that research, paying the company roughly $235 million upfront and investing an additional $180 million in multipledeals over the past year.
CureVac said it hopes this next-generation program could start human testing by the end of September, with the goal of launching in 2022.
Dr. Catherine Schuster-Bruce contributed reporting.
Young people who received the Pfizer-BioNTech or Moderna coronavirus vaccines have reported higher-than-usual rates of heart inflammation and swelling, US health officials said on Thursday.
The findings are preliminary and come from a self-reported database of side effects. The Centers for Disease Control and Prevention hasn’t confirmed a link to the vaccines, though researchers are investigating these incidents of heart inflammation, known as myocarditis, and heart swelling, known as pericarditis.
In interviews with Insider on Thursday, cardiologists and infectious-disease specialists said the risk from the vaccine is still tiny compared with the potential damage from getting infected with the coronavirus. Not all experts are convinced there’s a link between the events and the shots.
“It’s not a no-brainer that there’s an association, because the season for getting myocarditis is around now,” Dr. Lorry Rubin, the director of pediatric infectious diseases at Northwell Health’s Cohen Children’s Medical Center, told Insider.
A group of viruses called enteroviruses is the most common cause of myocarditis, Rubin said. Those viruses typically circulate in the summer.
“That’s not the sole cause, but that’s the most common infectious trigger for myocarditis, and that’s more of a seasonal infection,” he said.
All three experts agreed that getting vaccinated, even for young and healthy men and boys, is still the best option.
“You definitely should choose vaccination in that age group because it’s safer than wild-type virus infection,” said Dr. Leslie Cooper, a cardiologist who specializes in myocarditis at the Mayo Clinic.
Early reporting shows more heart side effects than expected
The preliminary data suggests elevated rates of the heart issues among younger vaccinated people, ages 16 to 24. At a Food and Drug Administration advisory meeting on Thursday, CDC officials shared data that included about 12 million doses given to people in this age range. CDC officials said that they would typically expect to see 10 to 102 cases of these heart events in this group but that 275 confirmed cases had been reported.
Most of these cases came after the second dose and were commonly reported in men, according to the preliminary data. The CDC said it was still confirming and investigating these self-reported cases.
The most common symptoms were chest pain and elevated levels of cardiac enzymes. Most of these cases were short-lived; at least 81% of people had already fully recovered from their symptoms, according to the data.
Even if the cases are linked to the vaccine, experts said, the greater risk, by far, is COVID-19.
“We’re talking about an incidence of myocarditis from the vaccine that is 100 times lower than the incidence when you actually get the infection,” Dr. Eliot Peyster, a cardiologist at the University of Pennsylvania, told Insider. “So, yes, it’s probably twice as likely as people who don’t get the vaccine, in a vacuum. But we aren’t in a vacuum. We’re in a pandemic where young people who get the virus get myocarditis in about one in 300 cases.”
The incidence rate among younger people who got the COVID-19 vaccine is still being determined, CDC officials said on Thursday. The agency said it would have more data to present at another meeting next Friday.
A strong immune response to the vaccine could be the culprit
The Pfizer and Moderna shots both use messenger RNA, a new technology that had not been used in a federally approved medicine before the pandemic. Despite its newness, experts said they didn’t think these cases stemmed from something unique to the technology.
Peyster said he would guess it was from a robust inflammatory response to the vaccine, particularly after the second dose.
“Any systemic inflammatory condition – any kind – can generate enough inflammatory stuff where you can actually get a little bit of heart-muscle damage just through systemic inflammation,” he said.
While more than 140 million Americans have been fully vaccinated, the concerns about younger people come as the vaccines start to be used in younger populations. Pfizer’s vaccine was OK’d last month for children as young as 12 years old. Moderna applied for a similar authorization on Thursday.
Not enough shots have been given and tracked in the 12-to-15-year-old population to know whether these heart side effects could be a concern for that age group. Both vaccine developers have said they’re testing their shots in even younger children and infants.
Experts guess that heart side effects will be less common in younger children
Rubin said he didn’t think there was reason for concern among parents deciding whether to get their children vaccinated.
“We don’t know if there’s a causal relationship, and it appears that the rate is low given the millions of people in the 12-to-25-year-old age group who have gotten this vaccine,” he said.
Peyster said he expected that these heart side effects would decrease among the younger ages. Given the history of myocarditis, he said, it’s likely the incidence rate will peak with young adults and adolescents.
The medical experts were unequivocal that the vaccine is clearly the best option overall, for all age groups.
“The evidence says you are much more likely to have heart inflammation from the virus than from the vaccine, even in young, healthy people,” Peyster said. “Therefore, the clear recommendation is vaccine is better than no vaccine.”
Moderna’s coronavirus vaccine appears to be highly protective in 12-to-17-year-olds, the Massachusetts biotech said Tuesday in a press release, paving the way for the shot to be given widely to adolescents as soon as next month.
If regulators give the green light, Moderna’s vaccine would be the second COVID-19 shot to be authorized for people as young as 12, after the Pfizer-BioNTech vaccine. Both vaccine developers are studying their shots in even younger populations.
Moderna enrolled more than 3,700 people between the ages of 12 and 17 in a clinical study. Two-thirds of the volunteer received two doses of Moderna’s vaccine. The other third received placebo injections.
Adults and children had similar immune responses to the shot, Moderna says
The main goal of the study was to measure the immune responses of participants, comparing their reactions to what was seen in adult volunteers. Moderna said Tuesday the study was successful in showing those immune responses were comparable between age groups, and that it plans to submit that information to regulators early next month.
Pfizer and BioNTech used a similar study to win authorization from the US Food and Drug Administration to provide their shot to adolescents.
The Moderna study counted how many people got sick with symptomatic cases of COVID-19, starting two weeks after the second vaccine dose. Four adolescents had COVID-19 cases in that period, and all of them received the placebo.
The study’s results have not yet been published in a peer-reviewed medical journal.
Moderna described the safety and tolerability of the vaccine as “generally consistent” with the adult trial. The most common side effects after the second shot were headache, fatigue, muscle pain, and chills.
Teenagers in the US are now eligible for a leading coronavirus vaccine, as the Food and Drug Administration on Monday expanded the authorization for Pfizer-BioNTech’s COVID-19 shot to include 12- to 15-year-olds.
Expanding eligibility to include the younger age group could give the immunization campaign a boost. The US has seen the daily rate of people getting their first dose plummet since early April, from a peak of 1.9 million people a day to about 830,000 a day, according to data from the Centers for Disease Control and Prevention.
A major driver for that decline has been that a majority of adults in the US have already received at least one dose. About 58% of US adults have received at least the first dose of a vaccine, according to CDC data.
“Today’s action allows for a younger population to be protected from COVID-19, bringing us closer to returning to a sense of normalcy and to ending the pandemic,” Dr. Janet Woodcock, the acting commissioner of the FDA, said in a statement. “Parents and guardians can rest assured that the agency undertook a rigorous and thorough review of all available data, as we have with all of our COVID-19 vaccine emergency use authorizations.”
Pfizer tested the vaccine in a clinical study that enrolled 2,260 children ages 12 to 15. Half of the volunteers received Pfizer’s shot, while the other half got a placebo injection. Overall, the trial recorded 18 COVID-19 cases, all in the placebo group. Side effects of the shot were in line with those observed in people 16 to 25 years old, Pfizer said.
Pfizer and other vaccine developers are also testing COVID-19 shots in younger populations down to 6-month-olds
In September, Pfizer anticipates study results showing whether the shot works in children ages 2 to 11.
The final step down for kids – from 6-month-old babies to 2-year-olds – should produce data in November, according to Pfizer’s projections.
Other leading vaccine developers are also testing their shots in younger populations.
On May 6, Moderna said its vaccine was highly effective in an initial review of a clinical trial enrolling more than 3,000 teenagers. The Massachusetts biotech said it was “in discussions with regulators about a potential amendment to its regulatory filings” after those early results. Moderna is also enrolling volunteers for a study testing the vaccine in kids 11 years old all the way down to 6 months old.
Moderna is voicing confidence in a new version of its coronavirus vaccine tailored to fight variants of the virus. The biotech on Wednesday released data from a clinical trial testing its booster doses.
While no one knows exactly when booster shots would be needed, experts generally think the presence of variants that can partially degrade the efficacy of vaccines will speed up the need to give additional shots, particularly for the most vulnerable populations.
Researchers tried 3 different kinds of boosters in the clinical trial
In this new clinical trial, Moderna tested a third dose on people who had previously gotten the two-dose vaccine six to eight months ago.
These study volunteers received one of three options: another dose of Moderna’s original vaccine, this new version tailored to the B.1.351 variant, or a multivalent version that contains a 50-50 mix of the original and B.1.351 vaccine versions.
In a Wednesday press release, Moderna described initial results from the original vaccine and the B.1.351 shot. The company plans to later report on the multivalent version, as well as testing an even lower dose strength of 30 micrograms. The research has yet to be published in a peer-reviewed medical journal, and Moderna said it has submitted the paper to a preprint server.
Before getting the third dose, Moderna tested volunteer blood samples to see how durable the vaccine response has been through six to eight months. While 37 of the 40 people had detectable levels of antibodies, the virus-fighting proteins that are the backbone of our immune response, only about half of the participants had detectable level of antibodies against B.1.351 and P.1, the variant first identified in Brazil.
One booster shot was most effective and led to fewer reactions
The study found those who were given the B.1.351-specific booster had the most effective immune response. Antibody levels were about 62% higher in that group, compared to those given another dose of Moderna’s original shot.
“We are encouraged by these new data, which reinforce our confidence that our booster strategy should be protective against these newly detected variants,” Moderna CEO Stéphane Bancel said in a statement. “The strong and rapid boost in titers to levels above primary vaccination also clearly demonstrates the ability of mRNA-1273 to induce immune memory.”
Moderna said the booster shots were well tolerated overall, with people having reactions similar to those seen after the second dose.
The B.1.351 booster shot appeared to produce slightly less severe reactions overall, Moderna said. About 11% getting the 351-specific booster reported a grade-three adverse event, which is severe enough to impact daily living activities, compared to 15% who got the a third dose of the original shot.
The most common side effects were fatigue, headache, muscle pain, and joint pain.
Invitae stock surged as much as 7% on Monday after a report out of the Wall Street Journal said Softbank plans to lead a $1.2 billion investment in the San Francisco-based genetic testing company.
The investment will come in the form of convertible debt and allow Invitae to expand its current genetic testing operations, per WSJ.
The convertible debt notes have an initial conversion price of $43.18 per share. Invitae shares closed at $39.19 on Friday.
The Inviate stake is the second sizeable investment from Softbank into a medical sciences company in the past few months.
The Japanese conglomerate also invested $900 million into the convertible debt of Pacific Biosciences, a next-generation DNA-sequencing systems firm, back in February.
Invitae announced plans to acquire the genomics company Genosity on Monday as well. The genetic testing company plans to use Genosity’s holdings to enhance its personalized oncology offerings.
Under the terms of the deal, Invitae will spend $200 million on the acquisition, consisting of approximately $120 million in cash and $80 million in Invitae shares. The deal is expected to close in the second quarter.
Softbank is the second big name to come out in support of Invitae.
In a CNBC interview in March, Cathie Wood of Ark Invest said Invitae “is probably one of the most important companies in the genomic revolution.”
Wood argued Inviate is “investing aggressively to be the leader in the diagnostics testing space” and that a “move towards personalized testing is going to give a few companies the lion’s share of the market.”
Despite the recent support from big names like Wood and Softbank, Invitae continues to struggle with profitability. The company turned in revenues of roughly $280 million in 2020 while posting a $602 million net loss, according to the annual 10-K SEC filing.
Invitae traded up 5.62%, at $41.36 per share, as of 10:56 a.m. ET on Monday.
We asked the investors to name companies from their firms’ portfolios, and ones they haven’t invested in. We noted when investors have stakes in the companies they nominated.
For some, that meant identifying startups that have seen their businesses take off amid the pandemic, like Everlywell, and others that have had to adapt as more care moved online, like Cityblock Health. Others still picked startups finding new ways to tackle conditions like ADHD, as Akili Interactive is doing with its FDA-approved video game.
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3:00 PM: Could Moderna’s shot provide some protection after a single dose? It’s possible, FDA says
Moderna’s COVID-19 vaccine trial was designed to measure how well two shots, given 28 days apart, protect people from infections. And it showed the vaccination works very well at that: it is 94% effective at preventing symptomatic COVID-19, 14 days after a second shot.
But, it’s possible that good protection from infection starts much sooner than that.
Preliminary trial data suggests the first dose of Moderna’s vaccine likely provides a decent measure of protection from infection all on its own.
Researchers found fewer COVID-19 cases among people who got Moderna’s shot compared to the placebo group after volunteers got their first injection, but before they got the second booster dose. That protection appeared to ramp up over time, generally kicking in a couple weeks after the initial shot.
“This could suggest some protection after the first dose, but data is limited by the very short follow-up, around 28 days, as the majority of the study subjects received a second dose,” Dr. Rachel Zhang, an FDA medical officer said.
Overall, the single shot appeared to be about 70% effective at preventing symptomatic infections. That looks better than Pfizer’s potential one shot protection, which appeared to be about 54% effective in the 21-day period before participants in that trial got a second injection.
Evercore ISI biotech analyst Umer Raffat also pointed out how well Moderna’s first dose might work on its own in a recent research note to investors. Data suggests the first dose was about 92% effective compared to a placebo, when looking at first COVID-19 occurrences starting two weeks after the first dose, Raffat said.
It’s still unclear how protective one dose would be in the long run, without a second shot. But Raffat, along with a handful of thought leaders in the drug industry like Pfizer board member and former FDA head Scott Gottlieb and University of Pittsburgh researcher Walid Gellad, have advocated the potential benefits after one dose should be considered, especially given the state of the pandemic right now.
Dr. James Hildreth, an immunologist on the committee, and an esteemed HIV researcher, also suggested that since so many people in the US have already had COVID-19, they might receive ample benefit from just one dose of Moderna’s vaccine.
“There are probably tens of millions of Americans who’ve already been infected,” Hildreth said Thursday during the meeting. “What I’m asking is, if you took the ones who have been infected, and gave them an injection of your vaccine, could that possibly serve as a boost?”
Moderna’s Dr. Jaqueline Miller said that as more study data becomes available in the weeks and months ahead, that could be something to look into.
Operation Warp Speed is holding back half of the available doses to ensure each vaccine recipient gets their booster dose at the right time — after three weeks for Pfizer’s shot, and after four weeks for Moderna’s.
Gottlieb and Gellad have argued it would be better to distribute all the doses right away, getting a first dose to twice as many people. While this opens up a risk that there may not be enough supply for everyone to get their booster shot on time, they say this could be an acceptable risk to take during the most deadly period of the pandemic.
2:45 PM: One panelist inquires about above-average Bell’s palsy rates, but the numbers are too small to draw conclusions
Dr. Paul Offit, a committee member and vaccine expert at the University of Pennsylvania, highlighted that both Moderna’s and Pfizer’s vaccine studies appeared to have higher rates than usual of Bell’s palsy.
Bell’s palsy is a typically temporary condition marked by weakness or paralysis in facial muscles. FDA scientists have acknowledged more people getting the coronavirus vaccines developed this condition than those getting placebo.
But the number of cases — seven among more than 30,000 vaccinated volunteers — is too small to say if the vaccine caused these cases. Agency reviewers said the Bell’s palsy cases in the studies were in line with typical incidence rates.
Offit also said it’s important to not fall victim to “the tyranny of small numbers” and conclude a relationship just yet, saying he is pleased the agency will closely monitor these vaccines going forward.
But Offit questioned the notion that the studies had typical rates of Bell’s palsy cases. “I’m just not quite sure how we are so comfortable that this was a background rate,” he said.
“If you look at the Pfizer trial, there were four cases of Bell’s palsy in a group of 22,000 [vaccinated people] per three months, which works out to about 8 cases per 10,000 per year,” Offit said. “If you look at the Moderna trial, it’s 3 cases for 15,000 for three months, which also works out to about 8 cases per 10,000 per year.”
Combining the two placebo groups together, Offit said those volunteers had about 1.2 cases per 10,000 per year. He said that’s roughly in line with the background rate.
FDA medical officer Dr. Rachel Zhang said the agency is looking into this.
“There is still no clear basis upon which to conclude a causal relationship, but we definitely see your point with the two studies combined,” she said.
She did not dispute Offit’s point that the vaccinated groups seemed to have higher-than-normal rates.
11:00 AM: Moderna pitches plan to immunize placebo volunteers without ‘jumping the line’
Moderna has a plan to offer trial volunteers who received placebo shots the actual vaccine, while side-stepping some of the key ethical concerns.
What to do with ongoing placebo-controlled trials during the pandemic has become a top conundrum facing vaccine developers like Pfizer and Moderna. On one hand, researchers want to keep following the placebo group to have high-quality, long-term data to see how these shots work. But ethicists also wonder if studies can, or should, withhold an effective vaccine from volunteers.
Moderna’s proposal is to ask participants whether or not they want to know if they got the real vaccine or a placebo. They can choose to remain blinded and in the study, or they can decide to know which arm they were in. Placebo participants who choose to become unblinded are then offered the real vaccine from clinical trial stocks, if they want it. All of those participants would be followed for long-term data.
Tal Zaks, Moderna’s chief medical officer, said this would not take doses away from priority groups. Instead, placebo recipients would get doses already produced for the sole purpose of clinical trial use that can’t be dispatched to the general public.
“None of our vaccine trial participants would be ‘jumping the line’ ahead of others, because we have clinical trial supplies available that would in fact expire and go to waste if we don’t use them,” Zaks told the panel.
Dr. Lindsey Baden, an infectious-disease expert at Brigham and Women’s Hospital, also spoke as part of Moderna’s presentation as one of the study’s investigators.
Baden said there have been a “substantial” number of volunteers dropping out of the trial in the last week since Pfizer’s vaccine secured an emergency OK last Friday.
“This dropout undermines the data integrity and what can be learned,” Baden said. “We must be proactive to ensure that the best choice is for our participants to remain in the study.”
Giving placebo recipients the chance to be vaccinated is important, because Zaks said many of the volunteers are at higher risk of infection and also have risk factors for severe disease.
A 54-year-old man with diabetes who got placebo shots in Moderna’s trial ultimately died from COVID-19, Zaks said, highlighting the risk in not immunizing these volunteers.
9:50 AM: FDA and CDC are investigating allergic reactions to Pfizer’s vaccine, but have no answers or recommendations yet
Dr. Stanley Perlman, a University of Iowa immunologist, asked about possible explanations for allergic reactions to COVID-19 vaccines, after two healthcare workers in the UK, and then two more in Alaska, had anaphylactic reactions after receiving Pfizer’s vaccine.
“At this point we and CDC are continuing to investigate cases and consider data,” said Doran Fink, an FDA vaccine expert. “At this point, we don’t have enough information to make definitive recommendations one way or another, and as we continue to investigate and evaluate the data we will consider whether additional recommendations need to be made.”
We are off and running for a marathon meeting expected to wrap up at 5:15 p.m.
On Tuesday, the FDA released the most detailed results yet from Moderna’s late-stage study in preparation for this meeting. The agency’s summary showed the two-dose vaccine to be overwhelmingly protective at preventing symptomatic COVID-19 with no major safety concerns.