Bluejay Diagnostics, Inc. (NASDAQ: BJDX) (“Bluejay”, “the Company”), a medical diagnostics company focused on developing cost-effective, rapid, near-patient products for triage and monitoring of disease progression, today announced that the FDA has agreed to consider Bluejay’s plan to pursue a De Novo submission for the Symphony IL-6 Test.
In written feedback to the January 2022 submission of the FDA pre-submission briefing package, the FDA stated that Bluejay’s Symphony IL-6 Test could be considered for the De Novo pathway.
CVS Health (CVS) - Get CVS Health Corporation Report shared nudged higher Thursday after the group reiterated its full-year profit outlook ahead of a series of investor meetings scheduled for the remainder of June.
CVS said its early-May forecast for 2022 earnings in the region of $8.20 to $8.40 per share still stands, with revenues pegged between $304 billion and $309 billion. The group's cash flow from operators guide also remains unchanged at $12 billion to $13 billion, the company said.
CVS posted stronger-than-expected earnings of $2.22 per share for the three months ending in March, an 8.8% increase from the same period last year, as as Aetna continued to power gains in the group's healthcare benefit division and retail traffic held up well despite slowing Covid testing and vaccinations.
Group revenues, CVS said, rose 11.1% from last year to $76.8 billion, again topping analysts' estimates of a $75.4 billion tally, a same-store sales rise 10.7%. Pharmacy store sales rose 10.1%, both benefiting from the group's administering of 8 million Covid vaccinations and a further 6 million tests over the three month period.
Cemdisiran Treatment Resulted ina 37 Percent Mean Reduction in 24-Hour Urine Protein to Creatinine Ratio, Relative to Placebo – a Key Prognostic Marker of Progression to End-Stage Kidney Disease in Patients with IgA Nephropathy -
- Acceptable Safety Profile, Supporting Continued Clinical Development -
- Full Results to Be Presented at Future Medical Congress -
BioCryst Pharmaceuticals, Inc.(Nasdaq: BCRX) today announced the company has entered into an exclusive collaboration with Pint Pharma GmbH to register and promote ORLADEYO®(berotralstat) in the pan-Latin America (LATAM) region.
“We are pleased to join forces with Pint Pharma to bring our oral, once-daily therapy to hereditary angioedema patients in LATAM who are in need of a new treatment option. Pint Pharma is the ideal partner for BioCryst based on the team’s deep experience in rare diseases that spans multiple aspects of commercialization and their established network across this important region,” said Charlie Gayer, chief commercial officer of BioCryst.
“Our partnership with BioCryst is significant for Pint Pharma given the critical unmet need among HAE patients in LATAM who are seeking an innovative treatment option for this serious disease. ORLADEYO has successfully launched in multiple markets across the globe, and we are privileged to leverage our expertise to support BioCryst in introducing this prophylactic therapy to the region,” said David Munoz, chief executive officer and co-founder of Pint Pharma.
Under the terms of the agreement, Pint Pharma will be responsible for obtaining and maintaining all marketing authorizations and for commercializing ORLADEYO in the pan-LATAM region.
Tuesday was a crucial day for Novavax(NVAX 5.41%). At long last, the U.S. Food and Drug Administration (FDA) Vaccines and Related Biological Products Advisory Committee (VRBPAC) met to review the company's COVID-19 vaccine data. A negative recommendation from the FDA advisory committee held the potential to deliver a crushing blow.
But that didn't happen. Instead, the VRBPAC members voted 21-0 in favor of recommending Emergency Use Authorization (EUA) for Novavax's NVX-CoV2373, with one abstention.
Novavax's shares initially moved higher on Wednesday, starting the day up around 10%. However, the upward momentum for the vaccine stock didn't last long. By mid-morning, all of the early gains had evaporated. Here's why investors yawned at Novavax's great FDA-panel news.
Stocks move the most when there's good or bad news that's unexpected. In this case, though, many investors were looking for a positive recommendation from the FDA advisory committee.
Microbiologist Eric Rubin, who is editor-in-chief of The New England Journal of Medicine, served as a temporary voting member of the VRBPAC. He perhaps summed things up about EUA for Novavax's vaccine by stating before the committee's vote, "It's not that difficult a decision now."
Granted, there was some level of uncertainty. Last week,Novavax stock plungedafter the FDA released briefing documents that highlighted a potential link between NVX-CoV2373 and myocarditis (inflammation of the heart). Investors were concerned that the FDA advisory-committee members could be swayed against EUA because of these possible safety issues.
However, the committee determined that the benefits of Novavax's COVID-19 vaccine outweighed any risks. It's important to note, as well, that the FDA briefing documents stated that there wasn't enough data to establish a causal relationship between NVX-CoV2373 and incidents of myocarditis.
The FDA doesn't always go along with advisory-committee recommendations. In this case, though, it seems very likely that the agency will do so. Before the VRBPAC held its vote, FDA official Dr. Peter Marks stated, "We speak to making available another option for those who might not otherwise take a vaccine."
But Marks' comments underscore two major hurdles remaining for Novavax. Maybe NVX-CoV2373 will be appealing to many of the unvaccinated since it doesn't use messenger RNA (mRNA) and instead uses an approach employed in other vaccines before COVID-19 came along. However, there's no guarantee that large numbers of unvaccinated Americans even want another COVID-19 vaccine.
Also, the EUA at play for NVX-CoV2373 right now is only for the primary two-dose series in adults. Nearly 77% of the U.S. population ages 18 and older are already fully vaccinated. More than 89% have received at least one COVID-19 vaccine dose. Novavax's vaccine won't be a booster option for any of these people, even if it wins the initial EUA, as expected.
One knock against NVX-CoV2373 in the FDA advisory-committee meeting was that there wasn't much data about how the vaccine protects against newer coronavirus strains, including the omicron variant. Moderna and the Pfizer-BioNTech teams are already moving forward with versions of their respective vaccines that specifically target omicron. Novavax could be at a big disadvantage if its rivals' efforts are successful.
It's possible that Novavax could clear all these hurdles. The company has already filed for authorization in the United Kingdom for use of its vaccine as a booster. It will almost certainly try to move quickly to secure a similar EUA in the U.S.
Novavax also is arguably in the lead at this point in developing a combination COVID-19/flu vaccine. If COVID-19 transitions from pandemic to endemic, as many expect, the combo-vaccine market could be where the most money is made.
For now, Novavax has passed a crucial test with the FDA advisory committee's overwhelmingly positive recommendation. But more big tests loom ahead for the company.
bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") today announced that Nasdaq has halted trading of the company’s common stock on Thursday, June 9 and Friday, June 10, 2022. The U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) is meeting to discuss the biologics licensing applications (BLAs) for betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel (eli-cel).
Beti-cel is under review for the treatment of people with β-thalassemia who require regular red blood cell transfusions. Eli-cel is under review for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor.
Briefing materials, agendas and webcast information for the meeting can be accessed here. The Company is not responsible for the content of, nor the statements made in, the briefing materials that were prepared by the FDA.
California-basedAnnexonreleased final dataTuesday from an open-label phase II clinical trial, showing that its drug candidate ANX005 safely stabilized disease progression in patients with Huntington’s disease (HD).
The findings, which were shared by company representatives at a conference call, revealed that over a 6-month treatment period, Annexon’s drug successfully prevented significant worsening of disease symptoms and preserved motor and cognitive function, as well as functional capacity, in HD patients. These effects remained durable over the next three months despite being off-treatment.
Of note, ANX005 appeared to be more beneficial for patients with more aggressive disease at baseline, as measured by elevated levels of the C4a molecule in cerebrospinal fluid. ANX005 treatment in these patients yielded symptom improvements as soon as six weeks after the first dose, which were maintained throughout the remainder of the on-treatment and subsequent off-treatment periods.
By targeting C1q, the starting molecule of the classical component cascade, ANX005 represents Annexon’s unique approach to Huntington’s disease.
Indeed, results of this phase II trial showed that during the treatment period ANX005 was able to completely and rapidly suppress C1q levels in the patients’ serum and cerebrospinal fluid. This effect was also durable, persisting for about one month after the final ANX005 dose.
In terms of safety, ANX005 gave no new signals than what had previously been reported. Three patients discontinued the trial due to side effects potentially related to the study drug. All three cases improved or were completely resolved after treatment cessation. Nevertheless, data from prior trials, enrolling more than 170 patients, have shown that ANX005 is generally well-tolerated across multiple indications.
“We are very encouraged by these final data with ANX005, which provide significant insights into our mechanism of action for a chronic neurodegenerative disease where the role of classical complement has been well-characterized,” Douglas Love, Esq., president and chief executive officer of Annexon, said in a statement. “The totality of the data, including robust and sustained C1q inhibition, clear impact on clinical outcomes, and favorable safety results observed, strongly support the potential for ANX005 to treat patients with Huntington’s disease.”
Love added, “Based on these results, we look forward to engaging with regulatory authorities to assess the opportunity for a well-controlled trial in Huntington’s disease leveraging a mechanistically compelling precision medicine approach.”
The phase II clinical trial employed an open-label design and enrolled 28 patients from multiple medical centers. ANX005 was administered intravenously for six months, followed by a three-month, off-label follow-up period. The primary study outcome was the drug’s pharmacokinetic and pharmacodynamic profile. Meanwhile, symptom burden was measured using the Composite Unified Huntington's Disease Rating Scale and Total Functional Capacity scale.
Huntington’s disease is a progressive and fatal brain disorder, causing uncontrollable movements and cognitive decline. Around 80,000 people worldwide have Huntington’s, while some 300,000 are at risk of inheriting a disease-causing gene. Patients diagnosed with Huntington’s are usually given 15 to 20 years to live.
There are currently no approved disease-modifying treatments for Huntington’s; that is, no drug can yet alter its ultimate course. Several biopharma companies have tried to leverage cutting-edge genetic and molecular technologies against the disease, but success remains elusive. Annexon’s novel approach of inhibiting early and late players in the complement cascade is a promising development in an otherwise frustrating field.
