announced initial findings from an investigator-sponsored study evaluating vadadustat for Acute Respiratory Distress Syndrome (ARDS) with COVID-19 and hypoxemia.
Akebia said the drug failed the primary endpoint, as measured by the National Institute of Allergy and Infectious Disease Ordinal Scale (NIAID-OS).
While the primary superiority threshold over placebo was 95%, researchers calculated a 94% probability that their drug was better than the placebo.
The University of Texas Health Science Center conducted the trial and was partially funded by Akebia. It enrolled 449 patients.
“While the trial missed its prespecified primary endpoint at Day14, we are extremely encouraged by the data and believe they support further developing vadadustat as a treatment for ARDS due to COVID-19 or other causes,” CEO John Butler said in a statement. “We will now work to review the full data set more thoroughly, consult with experts in the field and ultimately consult FDA on a potential path forward.”
The medicineSiga Technologies(SIGA) developed more than a decade ago to combat biowarfare is now being used in the ongoing viral outbreak, and Siga stock has exploded.
On Thursday, nearly three months after discovering the first U.S. case, the White House declared monkeypox a public health emergency. The declaration sent Siga stock to a record high on Friday — a repeat performance from May 18, the day after health officials identified the first case. Since mid-May, Siga stock has continued along the same trajectory, more than tripling.
Siga Tech makes the only medication allowed by the Food and Drug Administration to conditionally treat monkeypox, a viral disease characterized by fever symptoms as well as lesions or rash. The drug, dubbed Tpoxx, has full approval in the U.S. for smallpox treatment.
Smallpox and monkeypox both belong to the orthopoxvirus family. So, when monkeypox began to spread globally this spring, Siga was already in place. In fact, the U.S. government has a stockpile of 1.7 million courses of Tpoxx, the company says. Canada also has a stockpile. Siga stock has surged as orders have piled on — $56 million in requests to date.
Now, the rest of the world is trying to catch up, Siga Chief Executive Phillip Gomez told Investor's Business Daily. When Covid became a pandemic, epidemiologists warned that it wouldn't be the last viral outbreak. But no one expected monkeypox to arrive on its heels. This outbreak, says Gomez, will test the resolve of governments worldwide to prepare for what follows monkeypox.
"The real market for us in the long run is preparedness for orthopoxviruses," he said. "There are strains of monkeypox that are much more deadly. Part of the ultimate messaging around this outbreak is governments need to prepare in advance and have these products stockpiled."
Siga Stock Rockets Amid Outbreak
Public health officials declared smallpox eradicated in 1980. Only two labs are approved to have the virus for research: the Centers for Disease Control and Prevention and the Russian State Center for Research on Virology and Biotechnology.
But it's possible to engineer the virus from scratch. That's why the company received a grant from the National Institutes for Health in response to growing worries that laboratories abroad could engineer and weaponize smallpox. It's part of Project Bioshield, which was established in 2004 in light of the Sept. 11, 2001, terror attacks.
The CDC considers a single case of smallpox an emergency. The consequences of any spread would be "devastating," says Siga Chief Scientific Officer Dr. Dennis Hruby. The U.S. stopped routinely vaccinating against smallpox in 1972. So, most people in the U.S. born after that year would be vulnerable.
"It's probably the most serious bioweapon that could be deployed," he told IBD. "And, up until we developed a drug, there was no effective antiviral that could be used. If you got smallpox, you were either going to die and/or suffer the consequences of the disease."
Today, though, Siga stock is soaring on the use of Tpoxx in monkeypox patients. Tpoxx is available in the U.S. as a pill or intravenously. This year, Siga has received $60 million international order for Tpoxx from 10 countries. In the second quarter, the company reported $16.7 million in sales, surging about 92% year over year.
Tpoxx Orders Continue To Mount
The European Medicines Agency has fully approved Tpoxx for patients with smallpox, monkeypox, cowpox and complications of the smallpox vaccine. But U.S. and Canadian officials have approved Tpoxx only for smallpox treatment.
Amid the current outbreak, the FDA and CDC are allowing "compassionate use" of Tpoxx for patients with monkeypox. This means monkeypox patients can take Tpoxx even though it's not officially approved as a monkeypox treatment. But its use is limited due to a lengthy request process and specific criteria for prescriptions. As of July 23, just 215 patients had received Tpoxx, according to a CNN report.
Siga stock soared almost 25% on July 28 after the company announced an additional $28 million in Tpoxx orders from Canada and another $2 million from new jurisdictions in Europe and Asia-Pacific regions.
Since then, Siga has received "dozens of requests," CEO Gomez told IBD.
The market for the monkeypox treatment could be huge. But monkeypox, so far, appears to be slow to spread. There were just north of 7,100 cases in the U.S. as of Aug. 5, according to the CDC. Unlike Covid, which spreads through the air, monkeypox tends to spread through bodily fluids, requiring prolonged contact with an infected person, animal or contaminated surface.
But it could still go wider.
"We should nip this in the bud, so to speak," says Hruby, Siga's lead scientist. "We're all worried about it getting into animal reservoirs."
Two Smallpox Vaccines Available
The market could be massive. Some estimates suggest the smallpox vaccine offers about 80%-90% protection against monkeypox. But most people born in the U.S. after 1972 probably haven't received a smallpox vaccine.
There are two smallpox vaccines in the U.S. today. Sanofi (SNY) makes one called ACAM2000 for people at high risk of contracting smallpox. It contains a live vaccinia virus. Vaccinia belongs to the poxvirus family.
And Bavarian Nordic makes Jynneos, approved in the U.S. to prevent smallpox and monkeypox. It also contains a live virus that doesn't replicate. Hruby says Jynneos is safer but not as protective.
Moderna (MRNA) said in May it's looking at a monkeypox vaccine in laboratory testing. But as of early August, the company says it doesn't have an update on that effort.
That puts investors' focus in monkeypox almost exclusively on Siga stock.
Gomez, the CEO, says Siga could soon seek full approval for Tpoxx in monkeypox treatment. The current outbreak is giving Siga the chance to run clinical studies in monkeypox patients. Tpoxx's current suite of approvals is based on smallpox testing in animals because human testing would be unethical.
Siga Stock Gets High Ratings
The outbreak is also putting Siga stock on the map.
Shares went public in 2018, but long traded under 10. Prior to that, Siga was embroiled in a legal battle with PharmAthene over the medicine that became Tpoxx. A court ordered Siga to pay PharmAthene $113 million plus damages for failing to execute on a license agreement. Around that time, Siga filed for bankruptcy. It emerged from bankruptcy in 2016.
The biotech stock has surged since May and now has a perfect Relative Strength Rating of 99, according to IBD Digital. This puts shares in the top 1% of all stocks in terms of 12-month performance. Shares also have a strong Composite Rating of 97. This means Siga ranks in the leading 3% of all stocks in fundamentals and technicals.
Siga stock broke out of a consolidation with a buy point at 14.90 on July 13, the day after it signed an agreement for $28 million international orders of oral Tpoxx, according to MarketSmith.com.
Investors now are watching what's next for the biotech company. CEO Gomez says the monkeypox outbreak stands as an example of "imperfect incrementalism" in terms of improvement. The quick development of vaccine and treatments marked the Covid pandemic. In the monkeypox outbreak, there are already vaccines and a treatment. But "we kind of stumbled out of the gate."
"I think there will be some learnings out of this outbreak that are really around that last mile of preparedness," he said. "We should have countermeasures — vaccines, drugs and diagnostics — available for virus families to make sure we're prepared in an outbreak."
Shares of BeiGene Ltd. gained Friday in Hong Kong after the China-focused biotechnology company beat expectations with a doubling of revenue in the second quarter.
BeiGene's stock closed almost 12% higher at 116.10 Hong Kong dollars (US$14.79), paring year-to-date losses to less than 28%. The rise tracked the company's American Depositary Shares, which advanced 13% overnight.
BeiGene, which is based in Cambridge, Mass., and has offices in China and Switzerland, late Thursday posted second-quarter revenue of US$341.6 million, more than double the US$150.0 million it booked in the year-earlier period. That result, helped by starkly higher sales of its Brukinsa and tislelizumab cancer medicines, beat the consensus estimate of US$313.2 million, according to a FactSet poll of analysts.
The company's net loss widened to US$571.4 million from US$480.3 million a year ago. It attributed that to nonoperating expenses primarily related to foreign-exchange losses due to a stronger U.S. dollar.
Chairman and Chief Executive John V. Oyler said the company had expanded its drug approvals to more than 50 markets globally by the second quarter.It plans to share final analysis data for a global Phase 3 trial of Brukinsa as well as topline data for tislelizumab "as a first-line treatment for patients with unresectable hepatocellular cancer," during the second half of the year, he said.
Nomura analysts Jialin Zhang and John Nie said in a research note that the company's topline results came in 42% below their estimates, but that its gross profit margin improved slightly from the first quarter while its operating loss narrowed. BeiGene remains their preferred name in the Chinese biotech space.
AstraZeneca PLC said Friday that its lymphoma treatment Calquence's tablet formulation has been approved by the U.S. Food and Drug Administration for all current indications.
The Anglo-Swedish pharmaceutical giant said the approval was based on results from the Elevate-Plus trials where it was shown that Calquence's capsule and tablet formulations are bioequivalent, indicating the same efficacy and safety profile can be expected with the same dosing strength and schedule.
The company said Calquence, or acalabrutinib, was also approved as a capsule formulation for the same indications as the tablet in the U.S. and in many other countries around the world.
"Today's approval of the new Calquence tablet formulation will offer physicians and patients increased flexibility when devising treatment plans for chronic lymphocytic leukaemia and mantle cell lymphoma. This new option is a result of our focus on understanding the wants and needs of this community and providing patient-focused solutions for their treatment," the company said.
The newest health care and climate spending bill from Democrats includes an $80 billion boost to theInternal Revenue Servicethat is intended to help the agency crack down on wealthy tax cheats. However, Republican critics say that a bigger IRS could ultimately hurt lower-income Americans.
Providing the IRS with an influx of funding has been a top priority for President Biden. It has emerged as one of the most prominent financiers of the Inflation Reduction Act that Senate Majority Leader Chuck Schumer, D-N.Y., and Sen. Joe Manchin, D-W.Va., unveiled last week.
The Democrats projected that enhancing IRS funding could add an extra $124 billion in federal revenue over the next decade by hiring more tax enforcers who can limit tax evasion by rich individuals and corporations. Roughly $1 trillion in federal taxes goes unpaid yearly because of errors, fraud and a lack of resources to adequately enforce collections, according to an estimate from IRS Commissioner Chuck Rettig last year.
But GOP lawmakers have sounded the alarm over the proposal, warning that it could have serious ramifications for lower-income workers.
That's because the IRS disproportionately targets low-income Americans when it conducts tax audits each year. In fact, households with less than $25,000 in earnings are five times as likely to be audited by the agency than everyone else, according to a recent analysis of tax data from fiscal year 2021 by the Transactional Records Access Clearinghouse (TRAC) at Syracuse University.
The reason for that is a rise in what is known as "correspondence audits," meaning the IRS conducts reviews of tax returns via letters or phone calls rather than more complex face-to-face audits. Just a fraction — 100,000 of the 659,000 audits in 2021 – were conducted in person.
According to the Syracuse study, more than half of the correspondence audits initiated by the IRS last year — 54% — involved low-income workers with gross receipts of less than $25,000 who claimed the earned income tax credit, an anti-poverty measure.
Even taxpayers with a total positive income that ranged from $200,000 to $1 million had one-third the odds of being audited by the IRS compared to the lowest-income wage earners. About 9 million taxpayers reported these high-income levels in 2021, but fewer than 40,000 of their returns were audited, or roughly 4.5 out of every 1,000. That contrasts sharply with lower-income Americans, who faced an audit rate of 13 out of every 1,000.
The discrepancy is primarily due to high-income taxpayers having complex investments that can easily shroud the gaps between taxes owed and paid vs. taxes reported and paid.
"Barring an unlikely significant change in the composition of IRS enforcement, the stepped-up IRS enforcement would subject taxpayers across the income spectrum to more scrutiny and greater audit risk," the right-wing Heritage Foundation said in a recent blog post.
The Heritage Foundation noted that most IRS individual audit examinations target taxpayers reporting less than $50,000 of adjusted gross income. Although that group earns considerably less income than others, it faced recommended tax adjustments from the IRS of about $3.4 billion in fiscal year 2010. That compares to about $3.7 billion for those Americans reporting more than $50,000.
The IRS has maintained that it will not increase audits on households earning less than $400,000 if the $80 billion in funding is approved.
"These resources are absolutely not about increasing audit scrutiny on small businesses or middle-income Americans," Retting, the IRS commissioner, wrote in a letter to lawmakers on Thursday. "As we have been planning, our investment of these enforcement resources is designed around Treasury’s directive that audit rates will not rise relative to recent years for households making under $400,000."
President Biden tested positive for COVID-19 yet again Friday ashis “rebound” infectionpersisted for a seventh day — meaning he must remain quarantined at the White House.
Biden’s physician Dr. Kevin O’Connor wrote in a public memo that “[t]his morning, his SARS-CoV-2 antigen testing remained positive” and that the president “will continue his strict isolation measures as previously described.”
The White House has not let Dr. O’Connor take questions from reporters since Biden first tested positive on July 21, arguing he isn’t sick enough to justify such transparency.
The continued illness threatens Biden’s plan to resume in-person events next week — including a planned Monday trip to tour storm-damaged areas of Kentucky and a planned Rose Garden bill-signing event Wednesday to increase funding for veteran disability benefits and medical care linked to toxic smoke inhaled in war zones.
Biden completed a five-day regimen of antiviral Paxlovid last week and tested negative last Tuesday and Wednesday, but the virus resurfaced in daily testing Saturday after the president had for three days resumed in-person White House engagements.
O’Connor wrote Friday that Biden has minimal symptoms, despite testing positive.
The president, 79, “continues to feel very well. His cough has almost completely resolved,” O’Connor wrote. “His temperature, pulse, blood pressure, respiratory rate and oxygen saturation remain entirely normal. His lungs remain clear.”
Biden, the oldest-ever US president, began to experience symptoms on the evening of Wednesday July 20 and tested positive the following morning, according to prior disclosures from O’Connor.
The president fell ill less than four days after he returned to the White House from a trip to Saudi Arabia to make peace with killer crown prince Mohammed bin Salman. The White House has sought to tamp down speculation about how Biden contracted the virus.
Biden managed to avoid the virus for more than two years in part because White House aides and reporters who came in close contact with him had to get tested beforehand.
Biden’s advanced age and history of asthma put him at elevated risk of serious illness, though the fact that he received four doses of Pfizer’s coronavirus vaccine reduced that risk.
AstraZeneca and Daiichi Sankyo on Friday secured U.S. approval that broadens the use of their therapy Enhertu, for patients with breast cancer, and paves the way for billions in sales.
This is the first-ever U.S. Food and Drug Administration (FDA) approval for a medicine specifically targeting patients with so-called HER2-low breast cancer, the agency said.
Breast cancer can express, at varying levels, a protein called HER2 that contributes to its growth and spread. HER2-low tumours are defined as those whose cells contain lower levels of the HER2 protein on their surface.
The U.S. approval is based on data from a late-stage trial involving over 550 patients with HER2-low breast cancer - most with tumours that were hormone-sensitive - whose disease had spread and who had undergone at least one round of chemotherapy.
The data, unveiled at a scientific conference in June that prompted a standing ovation from the audience, showed Enhertu prolonged survival by an additional 6.4 months in patients with hormone-sensitive tumours.
In the small group of patients with hormone-insensitive tumours, patients on Enhertu lived 6.3 months longer.
The therapy also nearly doubled the time before patients with hormone-sensitive disease began to worsen - a measure known as progression free survival (PFS).
However, Enhertu carries some significant safety concerns.
Enhertu should carry a boxed warning - the FDA's most serious - to highlight the the risk of a type of lung scarring and fetal toxicity, the regulator said on Friday, adding that the therapy is not recommended for pregnant women.
The drug belongs to class of therapies called antibody drug conjugates (ADC). Enhertu comprises a monoclonal antibody - in this case trastuzumab (also known as Herceptin) - chemically linked to a cell-killing chemotherapy drug.
Enhertu originally won U.S. approval in late 2019 as a third-line treatment for the 15% of breast cancer patients with HER2-positive disease.