Novavax COVID-19 shot could be cleared for U.S. use as early as May: CEO

 Novavax Inc’s chief executive said on Monday its COVID-19 vaccine could be cleared for use in the United States as soon as May if U.S. regulators authorize it based on data from the company’s British trial, which could be completed as soon as April.

Novavax can already manufacture its shots at scale and will be able to have tens of millions of doses stockpiled and ready to ship in the United States when it receives authorization, Chief Executive Stanley Erck said.

“It will be substantial - in the many tens of millions or a hundred million,” Erck said in an interview.

Novavax had promised to deliver 110 million doses to the U.S. government by the end of the third quarter. That could happen as early as July, Erck said.

The timeline for U.S. authorization would get pushed back to June or July if regulators choose to wait for Novavax to complete its U.S.-based trial before reviewing the vaccine, he said.

Novavax on Monday reported that its net loss widened to $177.6 million, or $2.70 per share, in the fourth quarter, from $31.8 million, or $1.13 per share a year earlier.

Boosted by vaccine business, revenue rose to $279.7 million from $8.8 million a year earlier.

Novavax promised to deliver doses to the United States after it was awarded $1.6 billion to help finance research, development and production of a COVID-19 vaccine.

An early data readout in January from its UK trial showed the vaccine to be around 96% effective against the original version of the coronavirus and around 86% effective against the now widely circulating variant first discovered in Britain.

Novavax completed enrollment of its 30,000-subject U.S.-based trial in February.

https://www.reuters.com/article/us-novavax-results/novavax-covid-19-shot-could-be-cleared-for-u-s-use-by-may-ceo-idUSKCN2AT3MD

Emergent Bio a Buy on Vaccine News: Cramer

 Emergent and Johnson & Johnson have a manufacturing agreement for JNJ's  (JNJ) - Get Report COVID-19 vaccine. 

The vaccine, which received an emergency use authorization from the Food & Drug Administration over the weekend, is now the third vaccine to start being distributed in the United States.

Per a press release from July 2020, "Under the agreement, Emergent will begin providing large-scale drug substance manufacturing for Johnson & Johnson’s adenovirus-based COVID-19 vaccine in 2021, upon successful completion of the activities under the previously executed Technology Transfer Agreement. For the subsequent years beginning 2023, Emergent will provide a flexible capacity deployment model to support additional drug substance batches annually."

https://www.thestreet.com/video/jim-cramer-emergent-bio-buy-jnj-vaccine

Merck Withdraws KEYTRUDA Indication in Metastatic Small Cell Lung Cancer

 Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the company is voluntarily withdrawing the U.S. indication for KEYTRUDA (pembrolizumab) for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy. The withdrawal of this indication was done in consultation with the U.S. Food and Drug Administration (FDA), and Merck is working to complete this process over the coming weeks. This decision does not affect other indications for KEYTRUDA, Merck’s anti-PD-1 therapy.

This accelerated approval for KEYTRUDA was granted in June 2019 based on tumor response rate and durability of response data from KEYNOTE-158 (cohort G) and KEYNOTE-028 (cohort C1). Continued approval for this indication was contingent upon completion of the post-marketing requirement establishing superiority of KEYTRUDA as determined by overall survival (OS). As announced in Jan. 2020, KEYNOTE-604, the confirmatory Phase 3 trial for this indication, met one of its dual primary endpoints of progression-free survival but did not reach statistical significance for the other primary endpoint of OS.

Merck’s consultation with the FDA on this withdrawal is part of an industry-wide evaluation of indications based on accelerated approvals that have not yet met their post-marketing requirements. Merck is notifying health care professionals about this withdrawal. Patients being treated with KEYTRUDA for their metastatic SCLC should discuss their care with their health care provider.

“The accelerated pathways created by the FDA have been integral to the remarkable progress in oncology care over the past five years and have helped many cancer patients with advanced disease, including small cell lung cancer, access new treatments,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. “KEYTRUDA remains a foundational treatment for certain patients with metastatic non-small cell lung cancer. We will continue to rigorously evaluate the benefits of KEYTRUDA in small cell lung cancer and other types of cancer, in pursuit of Merck’s mission to save and improve lives.”

https://www.businesswire.com/news/home/20210301005851/en/Merck-Provides-Update-on-KEYTRUDA%C2%AE-pembrolizumab-Indication-in-Metastatic-Small-Cell-Lung-Cancer-in-the-US

Beijing CRMO Further Expands UK Footprint with Abbvie’s Allergan Site

 Beijing-based CRMO Pharmaron has accomplished impressive global growth via acquisitions over the past eight months. First buying up Beijing SMO LinkStart in July, then US-based Absorption Systems, a non-clinical CRO in November.

Today Pharmaron expands its U.K. presence with the $118.7 million purchase of AbbVie’s Allergan Biologics Limited (the ABL site) in Liverpool.  

The ABL site secures certified GMP manufacturing capabilities with state-of-the-art Cell and Gene Therapy product development expertise. Already accredited by the MHRA for biologic manufacturing, the site is turnkey for manufacturing on a commercial scale. 

Pharmaron’s goals to offer global end-to-end services are becoming a reality. The purchase of Absorption Systems not only got the company facilities and labs in Philadelphia, San Diego and Boston, but also core expertise in DMPK and bioanalysis. Additionally, Absorption System brought capability to evaluate cell and gene therapy products, and “further expands and strengthens our services and global network with strategic presence in life science hubs in the U.S.,” according to the November press release.  

Dr. Boliang Lou, Chairman and CEO of Pharmaron, commented, “We have been impressed with the CGT focused process R&D and complex biologic manufacturing capabilities of the ABL site, as well as the abilities that the highly experienced scientific, technical and managerial teams have demonstrated. The addition of this platform to Pharmaron will further strengthen our CGT services platform and allow us to better serve our partners’ needs in these exciting and emerging therapies. We are committed to further expanding capabilities and capacities at the Liverpool site to meet growing unmet demand. This transaction demonstrates our commitment to becoming a global leader in drug R&D services across multiple therapeutic modalities.” 

The ABL site in Liverpool hadn’t been in the Abbvie family all that long before the giant made the decision to sell it off. The two pharma’s finally got the clear to close on the massive deal last May after initially proposing 11 months prior. The $63 billion price tag makes this merger the second largest in life sciences history, just $11 billion short of record-holder Bristol-Myers Squibb with the acquisition of Celgene.  

Pharmaron now has over 11,000 employees on staff. The ABL site adds its name to three other Pharmaron sites in the U.K., bringing the company’s location total to 16 sites across China, the U.S. and the U.K. The company has partners across North America, Europe, Japan and China.

https://www.biospace.com/article/beijing-crmo-expands-uk-foothold-with-abbvie-acquisition-/

Authorization of J&J's COVID-19 Vaccine Increases States' Supplies by 25%

 As expected, the U.S. Food and Drug Administration (FDA) granted Emergency Use Authorization (EUA) to Janssen Pharmaceutical of Johnson & Johnson’s single-shot COVID-19 vaccine. The EUA covers individuals 18 years of age and older.

Unlike the Pfizer-BioNTech and Moderna vaccines, which were authorized late in 2020 and require two doses three or four weeks apart, respectively, the J&J vaccine is a single dose. And while the Pfizer-BioNTech and Moderna vaccines require extreme cold storage, particularly the Pfizer-BioNTech product that requires temperatures as low as -94 degrees F, the J&J vaccine remains stable for two years at -4 degrees F and a maximum of three months at routine refrigerator temperatures.

“This milestone follows a year of incredible work by our dedicated teams and unprecedented collaboration with health leaders around the world—all of whom shared a goal of bringing a single-shot vaccine to the public,” stated Alex Gorsky, chairman and chief executive officer of J&J. “We will do everything we can to help bring this pandemic to an end, in the United States and throughout the world.”

The company has agreed to deliver 100 million doses of the vaccines by the end of June. It expects to begin delivering the vaccine on March 1 and can distribute more than 20 million doses in the U.S. by the end of March. The initial capacity immediately is about 3.9 million doses, according to Lori Tremmel Freeman, chief executive officer of the National Association of County and City Health Officials, which should increase vaccine capacity for states by about 25%.

Utah Gov. Spencer Cox, for example, told CNN he expects Utah will receive “several thousand doses” over the next several days. “We expect shots in arms this coming week. We know they have about 4 million doses on hand right now ready to go out. They’ll start shipping those out, we believe, on Monday. We should have those shots, we think, on Wednesday. And shots in arms on Thursday and Friday. That’s a big deal for our state and for Americans everywhere.”

Despite how fast the vaccines have been developed, the unprecedented manufacturing and distribution has been criticized for bottlenecks and slow rollout. Currently, Pfizer is ramping up the supply of the vaccine. At the beginning of this month, the company and BioNTech were delivering 4 to 5 million doses weekly. By mid-March, they project it will be 13 million doses per week, with expectations of delivering all 300 million doses it has a contract for with the U.S. government delivered by the end of July. This would allow for full vaccination of 150 million people in the U.S. Moderna said that by the end of March between it and Pfizer, they should have 220 million doses, a dramatic increase from the approximately 75 million delivered so far.

According to the U.S. Centers for Disease Control and Prevention (CDC)’s COVID Data Tracker, as of Sunday, February 28, 96,402,290 doses have been delivered with 72,806,180 having been administered. Of those, 23,698,627 have received two doses.

There has been some concern expressed by the public over the J&J vaccine’s lower efficacy rates than the Pfizer-BioNTech and Moderna vaccines. In J&J’s global clinical trial, it demonstrated 66% efficacy at preventing symptomatic COVID-19 infections. In the U.S. it was slightly higher, 72%. Both the Moderna and Pfizer-BioNTech vaccines demonstrated about 95% efficacy in trials. It’s very difficult to actually compare the results of different clinical trials, for several reasons. One, they don’t necessarily define “severe illness” or even “symptomatic illness” in exactly the same way. And two, they had different trial populations.

One of the key considerations, however, it their ability to prevent severe disease and death. All three appeared to be almost 100% effective in preventing COVID-19-related deaths. J&J’s vaccine was also tested in clinical trials in South Africa and has demonstrated protection against severe disease and death from the South African variant. Neither the Pfizer-BioNTech nor Moderna vaccines have been evaluated against the more virulent South African variant in clinical trials, although they were tested in laboratory assays and the companies have stated the vaccines continue to offer protection against the variants.

All three vaccines have similar side effects, all of which have been quickly resolved. They include fatigue, body aches, soreness at the injection site, nausea and fever. Many people have few, if any, side effects.

Amesh Adalja, senior scholar at the Johns Hopkins Center for Health Security, told Reuters, “These vaccines are the path to getting your life back. These are vaccines you should not hesitate to get when offered.”

At this time, most vaccination centers are only receiving one brand of vaccine, so few people will have a choice of vaccines, and most experts say you should take what’s offered. The more people that receive the vaccine quickly, the faster the pandemic will end and the less likelihood of more dangerous variants emerging.

Paul Offit, a member of the FDA’s vaccines advisory panel and director of the Vaccine Education Center and attending physician in the Division of Infectious Diseases at Children’s Hospital of Philadelphia, told NPR’s Scott Simon on Weekend Edition Saturday, the J&J vaccine “is very effective … at preventing severe disease after a single dose and it induces the kind of response, so-called cellular immune response, that looks like it’s going to have fairly long-lived memory, which is all good. This certainly provides protection against what you care about, which is hospitalization, ICU admission and death. It’s virtually 100% effective at doing that.”

https://www.biospace.com/article/single-shot-j-and-j-covid-19-vaccine-receives-emergency-use-authorization/

Bluebird reveals new details on cancer cases in gene therapy trial

 

• Executives at Bluebird bio on Thursday gave investors listening to a conference hosted by bank SVB Leerink an update on their investigation into two recent cancer cases in a study of the company's gene therapy for sickle cell disease. 
• The two diagnoses are a serious safety concern, recalling past worries over the potential for gene therapy to trigger genetic mutations that might lead to cancer. In response, Bluebird halted further study of the experimental gene therapy, and suspended sales of a similar product approved in Europe for the blood disease beta thalassemia. 
• New information shared by Bluebird's CEO and chief scientific officer, however, suggest other factors than the gene therapy may have led to one of cases, a diagnosis of acute myeloid leukemia in a patient treated five years ago. And the other case, of myelodysplastic syndrome in a more recently treated trial volunteer, may not be a conclusive diagnosis, the executives said. 

Whether or not Bluebird's gene therapy, known as LentiGlobin, led to cancer is the most important question facing the company, making the executives' exclusive disclosure of early findings to investors of note. Companies often release information they judge to be "material" to their business via a statement or filing with regulators. 

Bluebird's investigation remains ongoing, and the information shared Thursday is preliminary. The Leerink webcast is publicly accessible and Bluebird issued a press release on Feb. 22 confirming its participation in the conference. (Bluebird did not return a request for comment from BioPharma Dive by publication, but told a STAT News journalist the company would not issue a statement or file a report with the Securities and Exchange Commission.) 

"Results are early, our understanding of the two cases are evolving and our dialogue with the regulators is continuing," said Nick Leschly, Bluebird's CEO, at the virtual Leerink conference. "We're going to be transparent as can be with all stakeholders."

Bluebird disclosed the two cases on Feb. 16, sharing the diagnoses reported to the company and basic information about timing. On Thursday, Philip Gregory, Bluebird's chief scientific officer, added that testing had determined the patient with AML had several major "chromosomal abnormalities" and genetic mutations that are commonly observed with the blood cancer.

"These are all genes associated with AML, classical mutations associated with AML as well as genomic rearrangements associated with AML and provide an independent mechanism by which this AML occurred," Gregory told investors listening to the Leerink conference. 

Bluebird has found in the patient's leukemic cells genetic sequences for the viral vector it uses to deliver its gene therapy. The question it's now trying to answer is whether the vector is a "passenger" alongside those AML mutations or a "driver." 

Gregory noted that, to Bluebird's knowledge, there are no confirmed cases in any gene therapy study of a lentiviral vector like the one the company uses triggering cancer by integrating into patient cells. 

More information will be available in the coming weeks, Gregory said, at which point Bluebird would make a more substantive update. 

The other case, of myelodysplastic syndrome, is more complex, according to the executive, and may have not met the formal criteria for a diagnosis. No "blasts," or abnormal white blood cells, were detected in the patient's bone marrow. 

Both patients were quite sick with sickle cell disease, which could also have played a role in their developing cancer. Rates of blood cancers among people with sickle cell are anywhere from two to 10 times higher than the general population, Gregory said. 

Another case of myelodysplastic syndrome diagnosed in a study participant in 2018 was determined not to be the result of gene therapy treatment. 

The threat of cancer risk has shadowed the gene therapy field for decades, since several cases of leukemia were observed in an gene therapy study in France in the early 2000s. Researchers have refined and improved both their tools and their methods, and recent clinical trials have not shown any elevated cancer risk.

But Bluebird's February disclosure came about two months after another company, UniQure, announced a participant in one of its gene therapy studies had been diagnosed with liver cancer. Investigation of that case is still ongoing. UniQure uses a different type of vector than Bluebird. 

https://www.biopharmadive.com/news/bluebird-reveals-new-details-on-cancer-cases-in-gene-therapy-trial/595787/

Israel looking to buy 36 million booster shots of COVID-19 vaccine

  Israel is looking to buy 36 million more doses of COVID-19 vaccines, three times the number it has already bought, in case booster shots are needed later in the year, Prime Minister Benjamin Netanyahu said on Monday.

Netanyahu has made Israel’s world-leading COVID-19 vaccine roll-out, and what he has highlighted as his personal role in securing sufficient doses, a showcase of his campaign in the March 23 national election.

Scientists have raised the probability of regular boosts to deal with coronavirus variants. Netanyahu, with an eye on the ballot box, retweeted on Monday a comment from a senior official of his Likud party urging voters to turn to the prime minister’s “proven leadership” to ensure “millions” of future doses.

“We are working on bringing a further 36 million vaccines for the citizens of Israel,” Netanyahu said in public remarks. “The vaccines we have, no one knows how long they last...We need to prepare for the worst scenario. The worst scenario is that we have to vaccinate every half year.”

That would mean as many as 36 million doses in the coming year, if all 9 million Israelis require two doses every six months, he said.

“The entire world will compete for those vaccines ...I am again determined to bring Israel to the top of the list.”

More than half the Israeli population has received a single dose of the Pfizer/BionTech vaccine, and about 3.4 million of them have received two shots.

On Sunday, Israel approved plans to offer COVID-19 vaccines to Palestinians with Israeli work permits, a step which a rights group said did not go far enough to safeguard Palestinians in occupied territory. The Palestinians have received relatively few doses to date and lag far behind Israel.

https://www.reuters.com/article/us-health-coronavirus-israel-vaccine/israel-looking-to-buy-36-million-booster-shots-of-covid-19-vaccine-idUSKBN2AT2YG