S.Africa testing vaccines on variant, hopes for results within 2 weeks

 South African scientists are testing whether vaccines will be less effective against a COVID-19 variant first detected locally and hope for initial results within two weeks, a professor at the national communicable disease institute said.

The variant, known as 501Y.V2, was identified by South African genomics experts late last year and is thought to be more contagious than older variants.

Professor Penny Moore told Reuters the National Institute of Communicable Diseases had received samples from several local vaccine trials, including Oxford University and AstraZeneca's shot, and would try to find out whether antibody responses are reduced against 501Y.V2.

"The assays are now robustly set up and so we can start the vaccine studies, which are really very time-sensitive so we hope to have some preliminary results for at least two of the trials within the next two weeks," she said on Friday.

Moore said the NICD would run tests on all the vaccines being trial led in South Africa, including doses being developed by Pfizer and BioNTech , Johnson & Johnson and Novavax.

She said concerns expressed by British scientists and politicians this week that vaccines may not be as effective against the 501Y.V2 variant were not unfounded because of worrying mutations in the spike protein the virus uses to infect human cells.

"There is enough evidence to justify a concerted effort to understand whether there will be reduced protection from vaccines," Moore added.

The variant has driven a resurgence in infections in South Africa, taking total cases to more than 1.17 million - the most in Africa - and sending daily new cases to a peak of over 21,000 this week.

South Africa's health minister said on Thursday that hospitals were struggling to manage the influx of patients, as he laid out plans to vaccinate two-thirds of the population to achieve herd immunity.

https://www.marketscreener.com/quote/stock/NOVAVAX-INC-58256108/news/S-Africa-testing-vaccines-on-variant-hopes-for-results-within-2-weeks-32149861/

Denali Therapeutics Expected Key Milestones in 2021

 

• Late-breaker presentation on 12-week data from Cohort A in Phase 1/2 study of DNL310 (ETV:IDS) in Hunter syndrome (MPS II) to be held at WORLD Symposium on February 12th
  
• Following previously announced data on CSF GAG reduction with DNL310 and achievement of biomarker proof-of-concept, Phase 1/2 study is expanded with the addition of a third cohort (Cohort C) to further explore clinical endpoints
• Announcing five new enzyme replacement therapy programs in Transport Vehicle (TV) portfolio; expanding manufacturing capabilities and building out commercial capabilities
• Phase 1b study of LRRK2 inhibitor, DNL151 (BIIB122), in Parkinson’s disease is complete; target engagement and pathway engagement goals were met and data will be presented at an upcoming medical congress; late-stage clinical development to begin by year-end 2021 in collaboration with Biogen
• Phase 1 healthy subject data on EIF2B activator, DNL343, expected to be available in 1H 2021; Phase 1b study in ALS patients planned to begin in 2H 2021​
• Phase 1 study of CNS-penetrant RIPK1 inhibitor, DNL788 (SAR443820), initiated by Sanofi in Q4 2020; Phase 2 study of peripherally-restricted RIPK1 inhibitor, DNL758 (SAR443122), in cutaneous lupus erythematosus to begin in early 2021
• IND-enabling studies for PTV:PRGN and ATV:TREM2 to trigger two milestone payments from Takeda in Q1 2021

https://www.globenewswire.com/news-release/2021/01/08/2155565/0/en/Denali-Therapeutics-Announces-Significant-Program-Progress-and-Expected-Key-Milestones-in-2021-for-Its-Broad-Therapeutic-Portfolio-in-Neurodegeneration.html

U.S. to relaunch small business pandemic aid program

 The U.S. government is introducing new “robust safeguards” when the third round of the country’s main small business pandemic aid program launches on Monday after fraudsters and ineligible companies claimed cash last year, administration officials said on Friday.

The Small Business Administration (SBA) will kick off the third round of the Paycheck Protection Program (PPP) on Monday, opening initially to community financial institutions and to all lenders shortly thereafter, the officials said during a media briefing.

In contrast to the program’s previous two rounds during which loan applications were automatically approved upon submission, the SBA will vet the initial information, slightly slowing approvals. That process will involve running automated identity and data verification checks overnight, the officials said.

The additional $284 billion authorized for the program in a December relief bill is expected to be enough to meet incoming demand and will not run out, senior administration officials said.

The PPP, created by Congress to help small businesses hurt by coronavirus pandemic lockdowns keep staff on payrolls, enabled participating lenders to dish out $525 billion worth of loans during two rounds last year.

Government watchdogs and congressional investigators have warned that the program has attracted fraudsters, while many large and listed companies, as well as blacklisted companies, gamed the program’s rules to take cash.

The Department of Justice, working with other agencies, has charged more than 80 individuals with stealing more than $250 million from the program.

Congress also made several changes to the program when it reauthorized it, including allowing small companies which suffered a 25% or greater decline in 2020 revenues to apply for a second loan of up to $2 million. It also tightens language promising lenders will not be held responsible if borrowers break the rules, pledging no enforcement action may be taken against the lender if it acted in good faith and complied with relevant federal and state regulations. That tighter language had been lobbied for by lenders, who worried they would be swept up in a broader federal probe into PPP fraud, putting more onus on the SBA to vet applications.

Dan O’Malley, CEO of Numerated, a fintech company that provides software for roughly 125 banks to process PPP loans, said the program changes were positive but had caused it to become “really complicated” and warned that could create new technical hitches.

https://www.reuters.com/article/us-usa-smallbusiness-fraud/u-s-to-relaunch-small-business-pandemic-aid-program-monday-with-new-fraud-checks-idUSKBN29D1QV

EU says it has secured nearly half of Pfizer's 2021 global output of COVID-19 shots

 The European Union reached a deal with Pfizer and BioNTech for 300 million additional doses of their COVID-19 vaccine, the head of the European Commission said on Friday, in a move that would give the EU nearly half of the firms’ global output for 2021.

Pfizer has said it can produce up to 1.3 billion doses around the world this year. The new agreement with the EU comes on top of another order for 300 million doses of the vaccine, branded in the EU under the name Comirnaty, that the bloc agreed with Pfizer and German partner BioNTech in November.

“We now have agreed with BioNTech and Pfizer to extend this contract. With the new agreement we could purchase a total of up to an additional 300 million doses of the BioNTech vaccine,” Ursula von der Leyen told a news conference on Friday.

Pfizer was more cautious.

“We are in talks with the European Commission about an amendment to our existing supply agreement for Comirnaty, the Pfizer-BioNTech COVID-19 vaccine,” it said in a statement.

The move would allow EU governments to double their orders from Pfizer to 600 million doses, von der Leyen said, as the 27-nation bloc races to ramp up the vaccination of its 450 million people. Each recipient of the Pfizer vaccine need two doses to develop maximum protection.

In a separate announcement, the EU drug regulator changed its recommendation for the use of the Pfizer vaccine, allowing the extraction of six doses from a vial instead of five, confirming an earlier Reuters story.

That would increase available doses by 20%. The pricing would continue to be calculated in doses and not in vials, a spokesman for Pfizer said, meaning that there would be no discount on prices paid by the EU for new deliveries. An EU document seen by Reuters in December showed the price was 15.5 euros ($19) per dose.

Von der Leyen said 75 million of the additional doses under the new deal would be delivered in the second quarter of this year, and the rest by the end of 2021. Pfizer said that in total 500 million doses would be available to the EU by the end of this year, and an option for another 100 million could be taken up.

PRODUCTION CAPACITY

The large EU order is likely to require an expansion of Pfizer-BioNTech production capacity this year, as the companies already have deals with other wealthy nations for large supplies in 2021.

They cut by half to 50 million doses their production in 2020 after facing some snags in securing supplies of vaccine ingredients.

European officials said the companies were working to expand production capacity in Europe.

Pfizer has agreed to sell the United States up to 600 million doses, of which 200 million have been already ordered. Another 120 million doses are reserved for Japan this year, and 40 million for Britain, among disclosed deals.

Germany said on Monday it had agreed with BioNtech to supply 30 million additional doses in a side deal, although the timing of the delivery was unclear.

The German move, agreed in September but revealed only this week as the government faced pressures at home, appears to be in contrast with EU agreements that forbid members to negotiate parallel deals with vaccine makers.

“No member state on this legal binding basis is allowed to negotiate in parallel or to have a contract in parallel,” von der Leyen said. But a Commission spokesman declined to comment on whether the German deal was legal under EU agreements.

The new EU agreement includes a possible immediate order for 200 million doses and an additional option to buy another 100 million, the Commission said.

Doses are usually shared among EU states in proportion to their population, but it was unclear whether all governments would make orders based on the new contract.

https://www.reuters.com/article/us-health-coronavirus-vaccine-eu/eu-says-it-has-secured-nearly-half-of-pfizers-2021-global-output-of-covid-19-shots-idUSKBN29D113

Pfizer/BioNTech vax appears effective against mutation in new coronavirus variants

 Pfizer Inc and BioNTech’s COVID-19 vaccine appears able to protect against a key mutation in the highly transmissible new variants of the coronavirus discovered in Britain and South Africa, according to a laboratory study conducted by the U.S. drugmaker.

The study by Pfizer and scientists from the University of Texas Medical Branch, which has not yet been peer-reviewed, indicated the vaccine was effective in neutralizing virus with the so-called N501Y mutation of the spike protein.

The mutation is linked to greater transmissibility and there had been concerns it could also make the virus escape antibody neutralization elicited by the vaccine, said Phil Dormitzer, one of Pfizer’s top viral vaccine scientists.

The first results of tests on the variants offer some hope as Britain and other countries try to tame the more infectious variants which authorities believe are driving a surge in infections that could overwhelm healthcare systems.

The study was conducted on blood taken from people who had been given the vaccine. Its findings are limited because it does not look at the full set of mutations found in either of the new variants of the rapidly spreading virus.

Dormitzer said it was encouraging that the vaccine appears effective against the mutation, as well as 15 other mutations the company has previously tested against.

“So we’ve now tested 16 different mutations, and none of them have really had any significant impact. That’s the good news,” he said. “That doesn’t mean that the 17th won’t.”

Dormitzer said another mutation found in the South African variant, called the E484K mutation, was also concerning.

The U.S. researchers plan to run similar tests to see whether the vaccine protects against other mutations found in the British and South African variants and hope to have data within weeks.

The variants are said by scientists to be more transmissible than previously dominant ones, but they are not thought to cause more serious illness.

Independent experts gave a cautious welcome to the Pfizer study findings, but said the situation needs constant vigilance.

Paul Hunter, a professor in medicine at Britain’s University of East Anglia, said “the jury is still out on the impact of the South African variant on vaccine efficacy.”

Deborah Dunn-Walters, a professor of immunology at Surrey University in Britain, said it was “reassuring” that Pfizer is closely monitoring variants.

Ongoing testing will be needed, experts said, to allay concerns about whether vaccines being given to millions of people in the fight against the pandemic will protect them as the virus mutates. COVID-19 has killed more than 1.8 million people worldwide.

“The evidence is not conclusive but there is a lot to indicate that the existing MRNA vaccines do cover the new variants. That is the good news,” Andreas Bergthaler, principal investigator at the Research Center for Molecular Medicine of the Austrian Academy of Sciences in Vienna.

TESTS AND TWEAKS

AstraZeneca, Moderna and CureVac are also testing whether their shots will protect against the fast-spreading coronavirus variants. They have said they expect them to be effective, but have not said when study results will be published.

A senior British lawmaker expressed concerns in an interview on Friday that COVID-19 vaccines might not work properly against the South African variant. He was not responding to questions about Friday’s data.

The Pfizer/BioNTech COVID-19 vaccine and the one from Moderna Inc, which use synthetic messenger RNA technology, can be quickly adapted to address new mutations in the coronavirus if necessary. Scientists have suggested the changes could be made in as little as six weeks.

The variant is not the first of the pandemic to emerge and Eleanor Riley, a professor of immunology and infectious disease at the Edinburgh University, said similar studies would need to be repeated as new mutations appear. “It may be necessary to tweak the vaccine over time,” she said.

https://www.reuters.com/article/us-health-coronavirus-pfizer-vaccine/pfizer-biontech-vaccine-appears-effective-against-mutation-in-new-coronavirus-variants-study-idUSKBN29D0DX

Sarepta Crashes On Mixed Results For Gene Therapy In Muscular Dystropy

 Sarepta Therapeutics (SRPT) late Thursday announced mixed results for its gene therapy targeting a form of muscular dystrophy, leading SRPT stock to plummet.

In after-hours trading on the stock market today, SRPT stock tanked 51.9% near 81.50.

The biotech company said its gene therapy produced large amounts of a key protein needed for muscle function and strength. That protein is deficient in children born with Duchenne muscular dystrophy. But the increases didn't lead to statistically significant improvements in muscle function after one year.

Sarepta tested its gene therapy in 41 patients with Duchenne muscular dystrophy. The one-time treatment aimed to increase the amount of the missing protein, called dystrophin. The drug delivers a shortened form of the gene that codes for dystrophin.

At 12 weeks after treatment, patients showed an increased amount of dystrophin in their muscle fibers. But at 48 weeks following treatment, patients who received the gene therapy didn't show statistically significant improvements in muscle function. That led SRPT stock to crumble.

However, patients who received the gene therapy consistently outperformed those given a placebo, Sarepta said in a news release. Still, SRPT stock plunged as investors questioned the future of the one-time gene therapy.

The news also sent shares of Solid Biosciences (SLDB) tumbling 25.7% near 6.30. Solid Biosciences is also working on a gene therapy to cure Duchenne muscular dystrophy. Pfizer (PFE), which also has a competing gene therapy in the works, fell a fraction to near 37.

Shares of SRPT stock fell to their lowest point since March.

https://www.investors.com/news/technology/srpt-stock-crashes-mixed-muscular-dystrophy-gene-therapy-results/

Moderna coronavirus vaccine approved for use in the UK

 

• In a release, the country’s Department of Health said the Moderna vaccine meets the “strict standards of safety, efficacy and quality” of the Medicines and Healthcare Products Regulatory Agency.
• It added that the U.K. had ordered an extra 10 million doses of the vaccine, taking its total to 17 million. They are expected to be available from the spring.
• https://www.cnbc.com/2021/01/08/uk-regulator-approves-moderna-covid-vaccine.html