Novavax reports promising early trial results of COVID-19 vaccine

Novavax reports promising early trial results of COVID-19 vaccine – World

  • August 5, 2020

US biotech company Novavax on Tuesday announced its experimental COVID-19 vaccine elicited a robust immune response, producing more antibodies than are present in recovered patients, and with generally tolerable side-effects in its early-stage trial.

The company was given $1.6 billion by the US government last month to develop and fund the drug under Operation Warp Speed — but in terms of timeline it is behind other firms including Moderna and AstraZeneca which have entered the final stages of their trials.

It reported in a press release that the phase one stage of its placebo-controlled trial involved 131 healthy adults aged 18-59 and two dose groups of five and 25 micrograms.

Side effects included soreness at the site of injection, headache, fatigue and muscle pain. These were classified as mild to moderate, and none were severe.

After the first dose, all subjects who got the vaccine developed antibodies that block SARS-CoV-2’s “spike protein,” which it uses to latch on to human cells.

Most also developed “neutralizing antibodies” which are more potent and prevent viruses from infecting cells.

After a second dose given 28 days later, all participants had the more powerful neutralizing antibodies.

Novavax reported that the lower dose performed comparably with the higher dose, which is important when it comes to mass production and because lower doses generally elicit fewer side effects.

Antibodies are infection-fighting proteins made by the immune system. 

Another part of the immune response comes in the form of T cells, types of white blood cells that have the capability to kill infected cells and which are increasingly thought to play an important role against COVID-19.

Novavax said it looked for these cells in a subset of participants and found they were present.

The trial was supported by funding from the Coalition for Epidemic Preparedness Innovations (CEPI) and was conducted at two sites in  Australia.

Novavax has not yet shared the detailed findings but said it was submitting the research for publication in a peer-reviewed journal and to medical preprint site medrxiv.org. 

The final stage Phase 3 trial of its vaccine, called NVX-CoV2373, is set to take place this fall.

The Maryland-based company grows synthesized pieces of the SARS-CoV-2’s “spike protein,” which triggers an immune response, inside insect cells in order to help scale up production.

It also uses an “adjuvant,” a compound that boosts the production of neutralizing antibodies.

The company says the drug, which is a liquid formulation, can be stored at two degrees celsius to eight degrees celsius, refrigerator temperature.

In the spring, the company said it had proven the efficacy of a seasonal flu vaccine it had developed using the same technology.

AstraZeneca; covid-19

AstraZeneca successfully clears out the first trial for the Covid-19 vaccine

  • July 22, 2020

The Covid-19 vaccine being developed by AstraZeneca and Oxford University has been successful in the first phase of its clinical trials.

The AZD1222 vaccine has generated concrete results from the first clinical trials, involving 1,077 healthy adults aged between 18 and 55. The vaccine is proven to be able to generate antibodies and T-cells for at least 56 days, creating a protective shield for the immune system against the coronavirus.

However, this doesn’t mean the vaccine will be overall successful, it means it is ready for the second stage. People and overall investors placing their hopes on AstraZeneca and Oxford University have to keep in mind that the AZD1222 vaccine it is not ready yet and it has to go through intense testing before commercial use. This is only an indication that based on the data, the vaccine could be successful.

And this is what investors believe as well, placing their trust in AstraZeneca even more than before.

AstraZeneca share price has been on the rise due to recent news of the Covid-19 vaccine being successful

AstraZeneca’s share price traded on the London Stock Exchange has experienced an almost 10% increase under 6 days. Investors reaction to the successful trial announcement of the AZD1222 vaccine raised AstraZeneca’s price to £9,320 from £8,549, under a six-day period.

AstraZeneca has been one of the top 5 performers in FTSE 100, demonstrating the levels of trust investors have placed on the pharmaceutical giant to deliver a Covid-19 vaccine. AstraZeneca’s share price has been on the rise since last April, reflecting early news of the company being on the right course of producing a successful coronavirus vaccine, and how investors react to that news, by buying even more company shares.

However, investors reacting only to news could raise long-term issues if the AZD1222 vaccine turns out to be unsuccessful.

AstraZeneca’s future is placed on the AZD1222 vaccine

As long as the AZD1222 vaccine is successful at the final stage and the use of the vaccine doesn’t have major after effects, then the company will be successful, experiencing the biggest share price increase it has ever.

On the other hand, if the AZD1222 vaccine turns out to be unsuccessful or it would have after-effects placing human life at risk, then it is most certain investors will dump AstraZeneca’s share price by the hundreds.

When a company of that calibre takes a big bet as producing a Covid-19 vaccine, then at the end it could either be very successful or doomed. AstraZeneca seems to be in the right track at the moment, with investors boosting its share price, thus being unsuccessful or not, in the long run, is something that only time and concrete data will tell.

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Coronavirus treatment reduces number of intensive care patients in clinical trial, biotech firm says

Coronavirus treatment reduces number of intensive care patients in clinical trial, biotech firm says

  • July 20, 2020

Alex Trebek is emotionally opening up, and sharing some more good news with fans, as he continues to fight pancreatic cancer.

The Jeopardy! host, who in 2019 was diagnosed with Stage 4 pancreatic cancer, spoke with Good Morning America on Monday about the “good days” and “bad days” he has experienced while undergoing treatment, describing recently feeling “like I want to die” and worrying that he’s a burden to his wife, Jean.

“I feel like I’m a terrible burden to her, and that bothers me tremendously,” Trebek said.

Trebek went on to choke up while discussing the support he’s received from his wife throughout his cancer battle, explaining that she has told him he’s not a burden and that she’s been a “saint.”

“She has so much goodness in her that she is always giving out, always putting out to help me get over difficult moments, and there have been some difficult moments,” Trebek said. “And I’m just in awe of the way she handles it.”

Trebek says that, should the current treatment he’s undergoing not succeed, he won’t go to any “extraordinary measures to ensure my survival,” as “if the quality of life is not there, it’s hard sometimes to push” and keep going. But after recently sharing with viewers that his treatment is “paying off,” Trebek told GMA that “I expect to be around” in February to mark two years since he was diagnosed, and his results show that he’s “going in the right direction.” Brendan Morrow


Oxford researchers report positive early trial results for COVID-19 vaccine candidate

Oxford researchers report positive early trial results for COVID-19 vaccine candidate

  • July 20, 2020
COVID-19 Coronavirus

[Image from Unsplash]

Researchers at Oxford University say early-stage results from a Phase I/II clinical trial of a SARS-CoV-2 vaccine candidate display safety and strong immune responses.

Results, published in The Lancet, revealed that the vaccine is safe, causes few side effects and induces strong immune responses in both parts of the immune system in the fight against COVID-19, which is caused by SARS-CoV-2.

A T cell response (a cellular immune response) was provoked within 14 days of vaccination and an antibody response within 28 days. Researchers say that an ideal SARS-CoV-2 vaccine should be effective after one or two vaccinations, confer protection for a minimum of six months and reduce onward transmission of the virus to contacts.

“The immune system has two ways of finding and attacking pathogens – antibody and T cell responses,” lead author and Oxford professor Andrew Pollard said in a news release. “This vaccine is intended to induce both, so it can attack the virus when it’s circulating in the body, as well as attacking infected cells. We hope this means the immune system will remember the virus, so that our vaccine will protect people for an extended period. However, we need more research before we can confirm the vaccine effectively protects against SARS-CoV-2 infection, and for how long any protection lasts.”

Currently, the results from the trial are too preliminary to confirm whether the new vaccine meets those requirements, but Phase 2 (United Kingdom) and Phase 3 trials (UK, Brazil, South Africa) are taking place to confirm how effective the candidate is in protecting against infection.

Oxford’s new trial included 1,077 healthy adults between 18 years old and 55 years old with no history of COVID-19 infection across five UK hospitals between April 23 and May 21. The data reported in The Lancet covered the first 56 days of the ongoing trial.

The participants either received the new COVID-19 vaccine candidate (543 total recipients) or the meningococcal conjugate vaccine (534 people). Additionally, 113 participants (among which 56 received the COVID-19 vaccine and 57 were in the control) were asked to take paracetamol before and for 24 hours after their vaccination to help reduce vaccine-associated reactions.

Participants were split into four groups, with 88 people in “Group 1” receiving additional safety monitoring with assessment of antibody and T cell responses. Group 2 (412 people) had extra blood taken to assess for antibody and T cell responses, while Group 4 (567 people) had serum taken for antibody response observation only. Among those groups, half the participants received the COVID-19 vaccine and half received the control.

Group 3 (10 people) received only the COVID-19 vaccine and were given an extra dose 28 days after the first dose to determine safety and whether it boosted antibody and T cell responses.

After observing no serious adverse events, the researches deemed the vaccine to have an acceptable safety profile. The most commonly reported reactions were fatigue and headache, with 70% of all participants given the COVID-19 vaccine only reporting fatigue and 68% reporting headache. Other side effects included pain at the injection site, muscle ache, malaise, chills, feeling feverish and high temperature.

Participants who took paracetamol around their vaccination had reduced pain, chills, feeling feverish, muscle ache, headache and malaise in the two days after vaccination, while side effects were less common after the second dose in the 10 people who received the additional one. Taking paracetamol did not affect the immunogenicity of the vaccine candidate.

The researchers found that the vaccine produced strong antibody and T cell responses, with T cell responses targeting the SARS-CoV-2 spike protein markedly increased, although the level slightly declined to the median by day 56. The T cell response did not increase with the second dose in those 10 patients, which researchers say is consistent with other vaccines of this kind.

Antibody responses peaked by day 28 and remained high until day 56 in those given a single vaccine, with a boost in those who received a second dose.

At day 28, in those with a single dose of the vaccine candidate, 32 of 35 participants had neutralising antibody responses when measured in MNA80 neutralization assay, while 35 out of 35 had those responses when measured in PRNT50 neutralization assay.

The study is being done in collaboration with AstraZeneca (NYSE:AZN), the pharmaceutical company working with Oxford to develop and manufacture the vaccine candidate.

“We are encouraged by the Phase I/II interim data showing AZD1222 was capable of generating a rapid antibody and T-cell response against SARS-CoV-2,” AstraZeneca executive VP of biopharmaceuticals R&D Mene Pangalos said in a release. “While there is more work to be done, today’s data increases our confidence that the vaccine will work and allows us to continue our plans to manufacture the vaccine at scale for broad and equitable access around the world.”

Coronavirus vaccine trial at Oxford University shows 'robust' immune system response

Coronavirus vaccine trial at Oxford University shows ‘robust’ immune system response

  • July 20, 2020

The early results of Oxford University and AstraZeneca’s coronavirus vaccine trial showed “robust” immune system responses, according to the pharmaceutical firm.

“COVID-19 vaccine AZD1222 showed robust immune responses in all participants in phase I/II trial,” said AstraZeneca in an emailed statement.

Some 1,077 people took part in the study of AZD1222, which is also known as ChAdOx1 nCoV-19. About half of the participants received the experimental vaccine.

UK CORONAVIRUS VACCINE TRIAL IS ‘PROGRESSING VERY WELL,’ RECRUITING CHILDREN AND OLDER ADULTS

In the research, scientists said that they found their experimental COVID-19 vaccine produced a dual immune response in people aged 18 to 55. Dr. Adrian Hill, director of the Jenner Institute at Oxford University, said neutralizing antibodies were produced by the vaccine candidate.

In this handout photo released by the University of Oxford a doctor takes blood samples for use in a coronavirus vaccine trial in Oxford, England, Thursday June 25, 2020 - file photo.

In this handout photo released by the University of Oxford a doctor takes blood samples for use in a coronavirus vaccine trial in Oxford, England, Thursday June 25, 2020 – file photo.
(John Cairns, University of Oxford via AP)

In addition, the vaccine also causes a reaction in the body’s T-cells, which help to fight off the coronavirus.

“We are seeing good immune response in almost everybody,” said Dr. Hill. “What this vaccine does particularly well is trigger both arms of the immune system.”

Reported side effects from the trial include feeling tired, headaches, muscle aches, chills and fever. No serious side effects were noted.

MORE THAN 100 YEARS BEFORE CORONAVIRUS, THE SPANISH FLU PANDEMIC RAVAGED THE GLOBE

Further evaluation of the vaccine is being undertaken. “ChAdOx1 nCoV-19 showed an acceptable safety profile, and homologous boosting increased antibody responses,” the researchers wrote, in the study. “These results, together with the induction of both humoral and cellular immune responses, support largescale evaluation of this candidate vaccine in an ongoing phase 3 program.”

The results of the research are published in The Lancet medical journal.

With 296,364 cases and 45,385 deaths, the U.K. is one of the most impacted countries by the coronavirus pandemic, according to data compiled by Johns Hopkins University.

Shares of AstraZeneca were slightly lower in early Monday trading, changing hands at $61.02, down 0.15 percent.

INVESTIGATIONAL VACCINE PROTECTS MONKEYS AGAINST COVID-19 PNEUMONIA

A number of efforts to develop a coronavirus vaccine are underway around the world. Scientists at Israel’s Tel Aviv University and biopharmaceutical company Neovii, for example, recently announced a project to develop a COVID-19 vaccine.

Experts involved in the effort say that they are targeting the “Achilles heel” of coronavirus.

As of Monday morning, more than 14.5 million coronavirus cases have been diagnosed worldwide, at least 3.7 million of which are in the U.S., according to Johns Hopkins University. The disease has accounted for at least 606,741 deaths around the world, including at least 140,541 people in the U.S.

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Fox News’ Christopher Carbone and the Associated Press contributed to this article. Follow James Rogers on Twitter @jamesjrogers

Major coronavirus vaccine boost as trial suggests jab 'induces immune reaction'

Major coronavirus vaccine boost as trial suggests jab ‘induces immune reaction’

  • July 20, 2020

Trials on a potential vaccine for coronavirus being worked on in England have shown it can induce a response from the body’s immune system, a study suggests.

Scientists at the University of Oxford have been developing the jab for several months as part of efforts to find a way of preventing COVID-19 from spreading.

The findings of the team’s first phase of trials, which have involved around 1,077 people, have been published in The Lancet medical journal.

The study suggests the vaccine being developed is safe and “stimulated strong immune responses” with the bodies of people given the jab producing antibodies and white blood cells which can tackle the virus.

However, the team at the university have said there is still work to be done on the vaccine.

Professor Sarah Gilbert, of the University of Oxford, said: “There is still much work to be done before we can confirm if our vaccine will help manage the COVID-19 pandemic, but these early results hold promise.

“As well as continuing to test our vaccine in phase-three trials, we need to learn more about the virus – for example, we still do not know how strong an immune response we need to provoke to effectively protect against Sars-Cov-2 infection.

“If our vaccine is effective, it is a promising option as these types of vaccine can be manufactured at large scale.

“A successful vaccine against Sars-Cov-2 could be used to prevent infection, disease and death in the whole population, with high-risk populations such as hospital workers and older adults prioritised to receive vaccination.”

Following the news from the study being announced, Prime Minister Boris Johnson said the development was good to hear.

Mr Johnson tweeted: “This is very positive news. A huge well done to our brilliant, world-leading scientists & researchers at @UniofOxford.

“There are no guarantees, we’re not there yet & further trials will be necessary – but this is an important step in the right direction.”

Meanwhile, Health Secretary Matt Hancock also took to Twitter saying the news was “very encouraging”.

“We have already ordered 100 million doses of this vaccine, should it succeed,” Mr Hancock added.

“Congratulations to the scientists at @UniofOxford & @OxfordVacGroup and leadership of @AstraZeneca.”

Labour leader Sir Keir Starmer was also quick to praise the developed, tweeting: “This is encouraging news.”

Follow updates from the coronavirus pandemic via our live blog here.

Ottawa-led clinical trial seeks to protect cancer patients from COVID-19

Ottawa-led clinical trial seeks to protect cancer patients from COVID-19

  • July 18, 2020


OTTAWA – July 16, 2020 – Dr. Rebecca Auer, surgical oncologist and director of cancer research at The Ottawa Hospital, will lead a $2.8-million clinical trial that seeks to protect cancer patients from COVID-19 by strengthening their depleted immune systems. If it proves successful, the immunotherapy approach could help many of the people most vulnerable to COVID-19. Photo courtesy of The Ottawa Hospital.

Postmedia

A surgical oncologist at The Ottawa Hospital will lead a $2.8 million Canada-wide clinical trial that seeks to protect cancer patients from COVID-19 by strengthening their depleted immune systems.

If it proves successful, the approach could help not only cancer patients, but anyone vulnerable to COVID-19 because of immune systems weakened by age or disease.

“We think harnessing innate immunity could be one of our best weapons for fighting COVID-19 — and could easily be adapted to tackle future pandemics,” said Dr. Rebecca Auer, director of cancer research at The Ottawa Hospital, and the study lead.

The clinical trial involves IMM-101, an immune-boosting biotherapeutic made with a heat-killed bacterium, Mycobacterium obuense, found in soil and water.

Developed as an immunotherapy cancer treatment, IMM-101 has demonstrated the ability to activate the body’s first line of defence, including the natural killer (NK) cells responsible for guarding against viral and bacterial infections. It has been safely used in other clinical trials with cancer patients.

“An effective vaccine against COVID-19 could take another year or more to develop, test, and implement,” said Auer, an associate professor at the University of Ottawa. “In the meantime, there is an urgent need to protect people with cancer from severe COVID-19 infection, and we think this immune stimulator, IMM-101, may be able to do this.”

The researchers hope that boosting cancer patients’ immune systems with IMM-101 will protect them from developing COVID-19 or other respiratory illnesses, such as the flu, that can force delays in their treatment.

Cancer patients are often vulnerable to infection because chemotherapy indiscriminately targets fast-dividing cells, whether cancer cells or immune cells. The cancer itself can also attack a patient’s bone marrow, where most immune cells are made.

More than 90,000 people in Ontario received chemotherapy or radiation treatments last year alone, and COVID-19 has greatly complicated their care.

The clinical trial, approved by Health Canada, is expected to launch later this summer. It will enrol 1,500 patients — including about 250 in Ottawa — at nine cancer centres across the country.

Patients in the study will be randomly assigned to two groups: One that receives regular care, and one that has IMM-101 added to it.

Those in the latter group will be given three doses of IMM-101 over the course of 45 days then carefully observed for COVID-19, other flu-like illnesses, and respiratory infections.

Researchers want to understand if patients who receive IMM-101 are less likely to develop such illnesses.

There’s considerable evidence to support the scientific theory on which the trial is built. Immune boosters have been successfully used for years in veterinary medicine to prevent the transmission of respiratory viruses in cattle, horses and other animals.

What’s more, clinical trials in Europe are now testing the ability of the BCG vaccine — a tuberculosis vaccine that contains a live, weakened strain of Mycobacterium bovis — to prime the immune system against COVID-19. Research has shown BCG can train the immune system to better fight all kinds of respiratory infections.

“It’s like sending your innate immune system to the gym for a while,” Auer explained. “So when it comes back to attack the next pathogen, it’s much stronger and better.”

Researchers believe that an individual’s innate immune system response helps to explain why some people get severely ill with COVID-19 while others have only mild symptoms.

Innate immune cells, such as NK cells, recognize and attack a broad spectrum of infectious agents based on their general characteristics. (Our second line of defence, known as the adaptive immune system, recognizes specific features of a bacterium or virus, and those that it has previously encountered.)

But BCG and other live vaccines can’t be given to cancer patients, so Auer and her colleagues looked for an alternative and found IMM-101. Even though it’s not a live vaccine, IMM-101 creates a response because the innate immune system recognizes it as a foreign invader.

Researchers from The Ottawa Hospital worked with the Canadian Cancer Trials Group at Queen’s University to design the clinical trial. 

Funding and support has also come from the Canadian Cancer Society, BioCanRx, the Ontario Institute for Cancer Research, The Ottawa Hospital Foundation, The Ottawa Hospital Academic Medical Organization, ATGen Canada/NKMax, a biotechnology company, and Immodulon Therapeutics, the manufacturer of IMM-101.

The clinical trial is expected to completed by the end of the year.

Auer warned that COVID-19 will not be the last pandemic.

“I imagine that if this trial was positive, this would be something you could have in your back pocket when the next pandemic came,” she said. “You’d be able to say, ‘We have something that works and can protect high risk people.’”

OTTAWA - July 16, 2020 - Dr. Rebecca Auer, surgical oncologist and director of cancer research at The Ottawa Hospital, will lead a $2.8-million clinical trial that seeks to protect cancer patients from COVID-19 by strengthening their depleted immune systems. If it proves successful, the immunotherapy approach could help many of the people most vulnerable to COVID-19. Photo courtesy of The Ottawa Hospital.

Ottawa-led clinical trial seeks to protect cancer patients from COVID-19 | News

  • July 17, 2020

A surgical oncologist at The Ottawa Hospital will lead a $2.8 million Canada-wide clinical trial that seeks to protect cancer patients from COVID-19 by strengthening their depleted immune systems.

If it proves successful, the approach could help not only cancer patients, but anyone vulnerable to COVID-19 because of immune systems weakened by age or disease.

“We think harnessing innate immunity could be one of our best weapons for fighting COVID-19 — and could easily be adapted to tackle future pandemics,” said Dr. Rebecca Auer, director of cancer research at The Ottawa Hospital, and the study lead.


The clinical trial involves IMM-101, an immune-boosting biotherapeutic made with a heat-killed bacterium, Mycobacterium obuense, found in soil and water.


Developed as an immunotherapy cancer treatment, IMM-101 has demonstrated the ability to


activate the body’s first line of defence, including the natural killer (NK) cells responsible for guarding against viral and bacterial infections. It h

as been safely used in other clinical trials with cancer patients.


“An effective vaccine against COVID-19 could take another year or more to develop, test, and implement,” said Auer, an associate professor at the University of Ottawa. “In the meantime, there is an urgent need to protect people with cancer from severe COVID-19 infection, and we think this immune stimulator, IMM-101, may be able to do this.”


The researchers hope that boosting cancer patients’ immune systems with IMM-101 will protect them from developing COVID-19 or other respiratory illnesses, such as the flu, that can force delays in their treatment.

Cancer patients are often vulnerable to infection because chemotherapy indiscriminately targets fast-dividing cells, whether cancer cells or immune cells. The cancer itself can also attack a patient’s bone marrow, where most immune cells are made.

More than 90,000 people in Ontario received chemotherapy or radiation treatments last year alone, and COVID-19 has greatly complicated their care.

The clinical trial, approved by Health Canada, is expected to launch later this summer. It will enrol 1,500 patients — including about 250 in Ottawa — at nine cancer centres across the country.

Patients in the study will be randomly assigned to two groups: One that receives regular care, and one that has IMM-101 added to it.

Those in the latter group will be given three doses of IMM-101 over the course of 45 days then carefully observed for COVID-19, other flu-like illnesses, and respiratory infections.

Researchers want to understand if patients who receive IMM-101 are less likely to develop such illnesses.

There’s considerable evidence to support the scientific theory on which the trial is built.

Immune boosters have been


successfully used for years in veterinary medicine


to prevent the transmission of respiratory viruses in cattle, horses and other animals.

What’s more,

clinical trials in Europe

are now testing the ability of the BCG vaccine — a tuberculosis vaccine that contains a live, weakened strain of

Mycobacterium bovis —

to prime the immune system against COVID-19. Research has shown BCG can train the immune system to better fight all kinds of respiratory infections.

“It’s like sending your innate immune system to the gym for a while,” Auer explained. “So when it comes back to attack the next pathogen, it’s much stronger and better.”

Researchers believe that an individual’s innate immune system response helps to explain why some people get severely ill with COVID-19 while others have only mild symptoms.

Innate immune cells, such as NK cells, recognize and attack a broad spectrum of infectious agents based on their general characteristics. (Our second line of defence, known as the adaptive immune system, recognizes specific features of a bacterium or virus, and those that it has previously encountered.)

But BCG and other live vaccines can’t be given to cancer patients, so Auer and her colleagues looked for an alternative and found IMM-101. Even though it’s not a live vaccine, IMM-101 creates a response because the innate immune system recognizes it as a foreign invader.


Researchers from The Ottawa Hospital worked with the Canadian Cancer Trials Group at Queen’s University to design the clinical trial.


Funding and support has also come from the Canadian Cancer Society, BioCanRx, the Ontario Institute for Cancer Research, The Ottawa Hospital Foundation, The Ottawa Hospital Academic Medical Organization, ATGen Canada/NKMax, a biotechnology company, and Immodulon Therapeutics, the manufacturer of IMM-101.

The clinical trial is expected to completed by the end of the year.

Auer warned that COVID-19 will not be the last pandemic.

“I imagine that if this trial was positive, this would be something you could have in your back pocket when the next pandemic came,” she said. “You’d be able to say, ‘We have something that works and can protect high risk people.’”

Copyright Postmedia Network Inc., 2020

Umbilical Cord Blood Transplant Treats Children With SCD in Phase 2 Trial

Umbilical Cord Blood Transplant Treats Children With SCD in Phase 2 Trial

  • July 17, 2020

An umbilical cord blood transplant, coupled with a reduced intensity conditioning (RIC) regimen, can safely and effectively treat children with genetic disorders that include sickle cell disease (SCD) and thalassemia, data from a Phase 2 trial show.

These findings are detailed in the study, “Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders,” published in the journal Blood Advances.

An umbilical cord blood transplant, known as UCBT, is a procedure in which blood isolated from the umbilical cord and placenta of healthy babies at birth — a readily available source of stem cells— is infused to a patient with the goal of resetting and restoring the production of healthy blood cells.

Compared to a blood marrow transplant, UCBT is more easily accessible and less likely to lead to rejection or other transplant complications when a donor and recipient are not a perfect match.

These features make UCBT particularly suitable as a universal treatment for children with non-cancerous genetic disorders, bringing another alternative to more specific therapies directed to each disorder.

“There has been a lot of emphasis placed on cool new technologies that might address these diseases, but — even if they prove effective — those aren’t available to most centers,” Paul Szabolcs, MD, division director of bone marrow transplantation and cellular therapies at UPMC Children’s Hospital of Pittsburgh, and the study’s senior author, said in a press release.

“The regimen we developed is more robust, readily applicable and will remain significantly less expensive,” Szabolcs said.

This regimen consisted not only on UCBT itself, but also a new conditioning regimen that involved a combination of low-dose chemotherapy and immunosuppressant treatments. This regimen is given to patients prior to the transplant to reduce a risk of rejection.

Rather than administer all umbilical cord blood at once, the investigators also reserved 5% to be given at a later stage. This was done to spur a patient’s immune system into action as fast as possible.

The safety and efficacy of this new regimen was tested in a Phase 2 trial (NCT01962415), the largest of its kind to date, that enrolled 44 children (median age, 1.7; maximum age, 16) with different metabolic, immune-related, or blood disorders.

The study, which also includes adults up to age 55 and is set to end in November 2022, is still recruiting patients at its single UMPC site. More information is available here.

Transplanted cells were successfully implanted in all children at a median of 15 days following the transplant. One year later, more than 90% of blood cells in most of these children were derived from the donor, confirming the procedure’s effectiveness. The transplant failed in one child, the researchers reported.

Complications following the umbilical cord blood infusion were relatively mild. The incidence of graft-versus-host disease (GVHD) — a potential life-threatening condition that occurs when the recipient’s immune system perceives the donor’s cells as a threat — was 27% at six months. No child developed severe GVHD.

Nearly all children were alive at one (95%) and five years (85%) post-transplant. High survival rates (around 90%) were also observed in a subgroup of children with rare childhood disorders known as leukodystrophies, where three-year survival rates typically do not surpass 60% even with treatment.

“This RIC transplant regimen using single-unit UCB graft resulted in outstanding survival and remarkably low rates of graft failure,” the investigators wrote.

In a subgroup of 30 children with metabolic disorders caused by the lack of specific enzymes, the transplant not only restored the production of the missing enzymes after one year, but also halted the progression of neurological decline.

“We designed an approach now proven to be efficacious for at least 20 diseases. And we believe it might be effective for many, many more,” Szabolcs said.

Since submitting their study for publication, Szabolcs and his colleagues report having successfully used this treatment regimen in adults and in children with other diseases.


Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.

Total Posts: 15


José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.

Dynavax Announces First Participants Dosed in Phase 1 Clinical Trial Evaluating Medicago’s COVID-19 Vaccine Candidate with Dynavax’s CpG 1018 Adjuvant

Dynavax Announces First Participants Dosed in Phase 1 Clinical Trial Evaluating Medicago’s COVID-19 Vaccine Candidate with Dynavax’s CpG 1018 Adjuvant

  • July 14, 2020
  • Medicago expects to enroll 180 healthy adult participants
  • Preliminary safety and immunogenicity results are expected in October 2020
  • Dynavax is providing CpG 1018, the adjuvant contained in its U.S. FDA-approved adult hepatitis B vaccine, to enhance the immune response of Medicago’s COVID-19 vaccine candidate

EMERYVILLE, Calif., July 14, 2020 (GLOBE NEWSWIRE) — Dynavax Technologies Corporation (Nasdaq: DVAX), a biopharmaceutical company focused on developing and commercializing novel vaccines, announced today that the first participants have been dosed in the Phase 1 clinical trial evaluating Medicago’s plant-derived vaccine candidate adjuvanted with CpG 1018 to prevent COVID-19.

The previously announced collaboration is evaluating the combination of Medicago’s Coronavirus Virus-Like Particle (CoVLP) with Dynavax’s CpG 1018, the adjuvant contained in Dynavax’s U.S. FDA-approved adult hepatitis B vaccine.  Adding CpG 1018 is intended to enhance the immune response of Medicago’s COVID-19 vaccine which may reduce the amount of antigen required per dose, providing more doses to help protect a greater number of people.

“We are committed to supporting the development of an adjuvanted vaccine to prevent COVID-19,” commented Ryan Spencer, Chief Executive Officer of Dynavax. “CpG 1018’s ability to enhance the immune response, as successfully demonstrated in HEPLISAV-B, is expected to reduce the dose of antigen needed, helping ensure broader availability to patients. Additionally, the unique mechanism of action of CpG 1018 may provide important enhancements including increased protection in populations traditionally less responsive to vaccination such as older adults who are at greatest risk for severe disease from COVID-19.”

The study is a Phase 1 randomized, partially-blinded, prime-boost, staggered dose-escalation study intended to assess the safety, tolerability, and immunogenicity of the Coronavirus-Like Particle COVID-19 Vaccine at three dose levels (3.75 µg, 7.5 µg, and 15 µg VLP) unadjuvanted or adjuvanted with either CpG 1018, or another company’s adjuvant, in approximately 180 healthy subjects 18 to 55 years of age, who have been tested for the absence of SARS-CoV-2 antibodies.  Preliminary safety and immunogenicity results are expected in October 2020.  Medicago is also planning a Phase 2/3 trial to be initiated this October.

Medicago’s innovative plant-based production platform will be used to manufacture the COVID-19 vaccine antigen. This platform uses plants as mini-factories which create proteins that self-assemble into the virus-like particles that are used in the CoVLP vaccine candidate. Combining Medicago’s technology with Dynavax’s CpG 1018 adjuvant, the companies expect to be able to deliver up to 100 million doses by the end of 2021. By the end of 2023, Medicago expects to complete the construction of a large-scale manufacturing facility in Quebec City, Canada that Medicago anticipates will have the capacity to produce up to 1 billion vaccine doses annually.
 
About Vaccine Adjuvants
An adjuvant is a pharmacological or immunological agent that modifies the effect of other agents. Adjuvants are added to a vaccine to boost the immune response to produce more antibodies and longer-lasting immunity, thus reducing the dose of antigen needed. Adjuvants may also be used to enhance the efficacy of a vaccine by helping to modify the immune response by particular types of immune system cells.

About CpG 1018 Adjuvant
CpG 1018 is the adjuvant used in HEPLISAV-B® [Hepatitis B Vaccine (Recombinant), Adjuvanted], an adult hepatitis B vaccine approved by the U.S. Food and Drug Administration (FDA). Dynavax developed CpG 1018 to provide an increased vaccine immune response, which has been demonstrated in HEPLISAV-B. In pre-clinical and clinical studies, results demonstrated that the addition of CpG 1018 increases antibody concentrations, stimulates helper (CD4+) and cytotoxic (CD8+) T cell populations and generates robust T and B cell memory responses.  Additionally, CpG 1018 strongly favors development of the Th1 subset of helper T cells, the type of helper T cell that is essential for protection from infections with viruses and intracellular bacteria. CpG 1018 targets a single, well defined receptor (TLR9) expressed on only a few key cell types and the mechanisms of action as an adjuvant are quite well understood.  CpG 1018 provides a well- developed technology and a significant safety database, potentially accelerating the development and large-scale manufacturing of a COVID-19 vaccine.  Upon completion of on-going scale up activities, the existing equipment capacity for CpG 1018 will be 600 million to 1.2 billion adjuvant doses annually, depending on final dose selected.

About Dynavax
Dynavax is a commercial stage biopharmaceutical company developing and commercializing novel vaccines. The Company launched its first commercial product, HEPLISAV-B® [Hepatitis B Vaccine (Recombinant), Adjuvanted], in February 2018, following U.S. FDA approval for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older.  Dynavax is also further developing CpG 1018 as an advanced vaccine adjuvant through research collaborations and partnerships.   Current collaborations are focused on adjuvanted vaccines for COVID-19 and pertussis. For more information, visit www.dynavax.com and follow the company on LinkedIn.

About Medicago
Medicago is a biopharmaceutical company headquartered in Quebec City with production sites in Quebec, Canada and Durham, North Carolina, USA. Medicago’s mission is to improve global health outcomes by leveraging innovative plant-based technologies for rapid responses to emerging global health challenges. Medicago is committed to advancing therapeutics against life-threatening diseases worldwide. For more information please visit www.medicago.com.

About the Novel Coronavirus SARS-CoV-2 (and COVID-19 Disease)
SARS-CoV-2 is a new coronavirus identified in late 2019 which belongs to a family of enveloped RNA viruses that include MERS and SARS, both of which caused serious human infections of the respiratory system.  The virus causes a disease named COVID-19. Since this outbreak was first reported in late 2019, the virus has infected over 12.7 million people and has caused over 566,000 reported deaths (as of July 13, 2020). It has been declared a pandemic by the World Health Organization (WHO).  Currently there is no vaccine available for COVID-19.

Forward-Looking Statements
This press release contains “forward-looking” statements, including statements regarding the potential to develop a COVID-19 vaccine containing CpG 1018, and to do so on an accelerated basis.  Actual results may differ materially from those set forth in this press release due to the risks and uncertainties inherent in vaccine research and development, including the timing of completing development, the timing and results of clinical trials, whether CpG 1018 is proven to be beneficial when combined with Medicago’s CoVLP,  whether and when the vaccine will be approved for use, and whether sufficient quantities of CpG 1018 and of vaccine timely will be able to be manufactured, as well as other risks detailed in the “Risk Factors” section of our Annual Report on Form 10-K for the fiscal year ended December 31, 2019, as well as discussions of potential risks, uncertainties and other important factors in our other filings with the U.S. Securities and Exchange Commission. We undertake no obligation to revise or update information herein to reflect events or circumstances in the future, even if new information becomes available. Information on Dynavax’s website at www.dynavax.com is not incorporated by reference in our current periodic reports with the SEC.

Contacts – Dynavax
Nicole Arndt, Senior Manager, Investor Relations
narndt@dynavax.com
510-665-7264

Derek Cole, President
Investor Relations Advisory Solutions
derek.cole@IRadvisory.com 

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