Pfizer, BioNTech launch COVID-19 vaccine trial in kids under 12

 Pfizer Inc and German partner BioNTech SE began testing their COVID-19 vaccine in children under 12, with hopes of expanding vaccination to that age range by early 2022, the U.S. drugmaker said on Thursday.

The first volunteers in the early-stage trial were given their first injections on Wednesday, Pfizer spokesperson Sharon Castillo said.

The Pfizer/BioNTech vaccine was authorized by U.S. regulators in late December for people age 16 and older. Nearly 66 million doses of the vaccine had been administered in the United States as of Wednesday morning, according to data from the U.S. Centers for Disease Control and Prevention.

The pediatric trial, which will include children as young as 6 months, follows a similar one launched by Moderna Inc last week.

Only the Pfizer/BioNTech vaccine is being used in 16- and 17-year-olds in the United States. Moderna’s shot was cleared for those age 18 and older, and no COVID-19 vaccine has been authorized in younger kids yet.

Pfizer and BioNTech plan to initially test the safety of their two-shot vaccine at three different dosages - 10, 20 and 30 micrograms - in a 144-participant Phase I/II trial.

They plan to later expand to a 4,500-participant late-stage trial in which they will test the safety, tolerability and immune response generated by the vaccine, likely by measuring antibody levels in the young subjects.

Castillo said the companies hope to have data from the trial in the second half of 2021.

Meanwhile, Pfizer has been testing the vaccine in children from age 12 to 15. The company expects to have data from that trial in the coming weeks, Castillo said.

https://www.reuters.com/article/us-health-coronavirus-pfizer-children/pfizer-biontech-launch-covid-19-vaccine-trial-in-kids-under-12-idUSKBN2BH2LM

Novavax delays EU vaccine supply deal amid production problems

 Novavax is delaying signing a contract to supply its COVID-19 vaccine to the European Union, an EU official involved in the talks told Reuters, as the U.S. biotech company warned it was struggling to source some raw materials.

Prolonging the talks might further complicate the EU’s vaccination plans as the bloc had planned to sign a deal early this year for at least 100 million doses of Novavax’s vaccine, with an option for another 100 million.

The EU official, who asked not to named as the talks are confidential, said the company had postponed signing a deal for weeks, citing legal issues in meetings with the bloc’s vaccine negotiators.

“They are slowing down the process of finishing the contract,” the official, who attended the meetings, told Reuters.

Informally, a Novavax executive had said a slow pace in negotiations was warranted because the company was having production problems, the EU official said.

A Novavax spokeswoman said in an email that talks with the EU over a supply deal continue, but declined to say more about the discussions.

She said the company was working “through some pandemic-related raw materials supply shortages”, without giving details.

The European Commission, which coordinates talks with vaccine makers, declined to comment.

Facing supply problems from other COVID-19 vaccine makers, notably AstraZeneca, the EU’s immunisation campaign is lagging far behind those in the United States and Britain, a situation that has become more critical as a third wave of infections sweeps the bloc.

Novavax is planning to produce key components of its two-dose vaccines for the EU in several of its factories, it said, including one in the Czech Republic, which makes antigens, the inactivated organisms that trigger an immune response.

The EU official said production capacity at the plant, the only one in the EU, was too small the meet the needs of the 27-nation bloc.

A spokesman for the plant declined to comment.

Novavax has eight manufacturing locations, including the Serum Institute of India, the world’s biggest vaccine maker.

Serum warned earlier this month that a temporary U.S. ban on exports of critical raw materials could hurt its production of vaccines, including Novavax’s.

Serum is also making AstraZeneca’s COVID-19 vaccine and has suspended major exports of the shot.

It’s not clear if Serum’s issues are affecting Novavax’s potential supplies to the EU.

Novavax executives said in an investor call earlier this month its vaccine production plants should all be fully functional by April.

CEO Stanley Erck told Reuters on March 1 the company expected to have tens of millions of doses stockpiled and ready to ship in the United States when it receives regulatory approval there, possibly in May.

PRELIMINARY ACCORD

Novavax concluded exploratory talks with the EU in mid-December, a step that has usually been followed by the signature of a contract within two or three months, according to EU deals with other COVID-19 vaccine makers.

At a meeting in mid-February, the Commission told EU ambassadors a deal with Novavax was likely to be finalised in a week, an official who attended that meeting said. A second EU official, involved in talks with the firm, also said in February that a deal was close.

But the same official told Reuters on Thursday that talks were ongoing and the company was now saying it aimed for a deal by June, although the official added the timeline was not clear.

Novavax says it is working towards submitting an application for regulatory approval in the EU in the second quarter.

Regulatory approval is a precondition for a vaccine to be rolled out in the bloc, but its absence has not prevented the EU from signing vaccine supply deals.

All EU supply contracts have so far been signed before regulatory approval, with the EU usually making upfront payments to companies and agreeing the full price for vaccines would only be paid after regulatory authorisation.

Novavax’s vaccine has been assessed by the EU drugs regulator under a rolling review since early February.

Earlier in March, Novavax said its vaccine was highly effective in a late-stage trial in the United Kingdom.

https://www.reuters.com/article/us-health-coronavirus-eu-novavax-exclusi/exclusive-novavax-delays-eu-vaccine-supply-deal-amid-production-problems-source-idUSKBN2BH2GY

Amazon starts on-site COVID-19 vaccination for U.S. employees

 Amazon.com Inc is rolling out on-site COVID-19 vaccination for its U.S. front-line employees, it said on Thursday, as companies step up efforts to get their workers immunized against the coronavirus.

The e-commerce company, which has also been testing employees for COVID-19, said that on-site vaccination programs would first start in Missouri, Nevada, and Kansas and then expand across the country as more vaccines become available.

Amazon will give up to $80 to front-line workers with no access to the on-site clinics, mirroring a move by companies such as hotel operator Marriott International Inc and retailer Kroger Co to provide employees incentives to get COVID-19 vaccines.

Earlier in December, Amazon had asked the U.S. government to prioritize essential workers, including its warehouse, grocery store and data center staff, for receipt of the COVID-19 vaccine.

https://www.reuters.com/article/us-health-coronavirus-amazon-com-vaccine/amazon-starts-on-site-covid-19-vaccination-for-u-s-employees-idUSKBN2BH317

Why ProQR Therapeutics Is Popping

 Shares of ProQR Therapeutics (NASDAQ:PRQR) were trading higher after the company released positive results from a clinical trial for one of its leading pipeline candidates, QR-421a. The biotech is also benefiting from bullish commentary from a Wall Street analyst. 

QR-421a is an experimental treatment for Usher syndrome, a rare disorder that, among other things, can cause loss of vision. In a phase 1/2 clinical trial for QR-421a, the treatment was well tolerated by patients and produced improvement in several measures of vision, including visual acuity, visual field, and more.

Aniz Girach, chief medical officer of ProQR Therapeutics, said, "With just a single dose, QR-421a demonstrated clinical proof of concept with benefit observed in treated eyes compared to the untreated eyes in multiple concordant measures of vision."

Meanwhile, MP Securities analyst Liisa Bayko raised her price target on ProQR Therapeutics' stock to $35, up from $19. She also kept an outperform rating on the stock. According to the analyst, QR-421a seems increasingly promising after the clinical trial results that ProQR Therapeutics released. Bayko raised the probability of success of the QR-421a program on patients with Usher syndrome in her model to 60%, up from 20%. Note that ProQR Therapeutics is trading for $5.79 per share as of this writing, which means the $35 price target represents a significant upside. 

ProQR Therapeutics plans to start two phase 2/3 studies for QR-421a by the end of the year. As is usual with biotech companies, ProQR could see its shares plunge if these clinical trials don't yield positive results, while the stock could skyrocket if the data indicates that QR-421a is safe and effective. Is ProQR Therapeutics' risk-reward profile attractive? In my view, the answer is no, but investors comfortable with above-average risk may reach a different conclusion.

https://www.fool.com/investing/2021/03/24/why-proqr-therapeutics-nv-stock-is-popping-today/

FDA 2nd-in-command Abernethy leaving as Biden commissioner yet to be named

 After just two years at the FDA, principal deputy chief Amy Abernethy, M.D., Ph.D., is leaving the agency. As second-in-command and acting chief information officer, Abernethy oversaw tech and data modernization at the FDA and launched its COVID-19 accelerator project to boost real-world data gathering.

Her unexpected departure comes as the search for a permanent FDA commissioner continues two months into the Biden administration. Abernethy was considered as a contender for the job earlier in the process.

Abernethy plans to take time off with her family, but promised to be "back at it soon,” she wrote in a Twitter post Tuesday. She expressed her thanks to the staff at FDA and added some thoughts in a series of tweets.“If COVID has taught us anything, it’s that we need to rethink US digital health infrastructure. This transformation will require new ways of working across traditional silos in government & business/tech, ensuring we always put patients first,” she said.

Acting FDA Commissioner Janet Woodcock praised Abernethy in an internal memo to staff, noting her “remarkable modernization of the agency’s technology and data practices.” Woodcock pointed out Abernethy's initiative in volunteering to take on the acting CIO job, and said Abernethy again stepped up when the pandemic struck to assemble teams to support the FDA’s response.

The acting FDA chief has not yet named Abernethy’s successor.

Abernethy joined the FDA in December 2018 from Flatiron Health, where she was chief medical and scientific officer and also senior VP of oncology. Abernethy, who was hired by former FDA chief Scott Gottlieb, M.D., is a hematologist-oncologist and a well-regarded leader in the industry.

In December, Fierce Pharma voters chose Abernethy in an informal poll over 15 other potential candidates as the best person for the job. “Outstanding,” “great energy,” “nurturing leader” and “sensible and will not be politically swayed” were some descriptions about Abernethy from voters.

Her departure comes as pressure mounts on President Joe Biden to nominate a permanent FDA head. Six former FDA commissioners wrote the White House recently urging the president to step up the pace in naming a new leader. It's not clear who is currently the front runner for the Biden FDA commissioner job, but acting chief Woodcock is reported to still be in contention.

But with the Senate's approval last week of U.S. Department of Health and Human Services Secretary Xavier Becerra, a nominee may be forthcoming. Some industry insiders have suggested a nominee wouldn’t be advanced until a new HHS secretary, who will be the new FDA commissioner’s boss, was confirmed.

https://www.fiercepharma.com/pharma/fda-second-command-abernethy-to-depart-agency-as-biden-permanent-commissioner-still-yet-to

FDA slaps new warning on Glaxo Shingrix

 GlaxoSmithKline’s vaccine cash cow Shingrix has a new safety warning on its label, although regulators still maintain the shot’s benefits outweigh risks.

Wednesday, the FDA alerted the public that it has added to the “warnings and precautions” section of the shingles vaccine’s label language about an increased risk for a rare neuro autoimmune disorder called Guillain-Barré Syndrome (GBS).

The update came after a post-marketing observational study by federal health agencies noted the risk during the 42 days following vaccination with Shingrix. The FDA stressed that “available evidence is insufficient to establish a causal relationship,” and that the “benefits of vaccination with Shingrix continue to outweigh its risks.”

In short, the association of the rare disorder doesn’t change the FDA’s view that Shingrix is safe, the agency said.

In a statement, GSK said it “remains confident in the favorable benefit-risk profile of Shingrix for the prevention of shingles.” The company will continue to work with the FDA and the CDC on post-marketing safety monitoring for Shingrix, the statement said.

The FDA based its analysis on GBS cases reported from Medicare beneficiaries in the CDC’s vaccine safety monitoring system, Vaccine Safety Datalink. The team used data from nearly 3.73 million vaccinations of Shingrix from October 2017 to February 2020.

Compared with historical data for Merck’s older shingles vaccine Zostavax, the FDA found about 3 additional cases of the syndrome per million doses of Shingrix administered to adults aged 65 years or older during the 42 days following vaccination. Shingrix is given in two doses two to six months apart.

The increase seemed to be driven by the first dose. The FDA said it found an estimated 6 excess cases per million doses in the 42 days following the first dose, while it observed no increased risk of GBS after the second dose.

Before the postmarketing study, GBS wasn’t identified as a potential problem for Shingrix in the clinical trials that led to its approval in 2017.

GBS is a rare but potentially life-threatening disorder in which the body’s immune system attacks nerve cells. It most commonly occurs after infection with a virus or bacteria. Herpes zoster, the viral infection that Shingrix is designed to prevent, has itself been associated with multiple cases of subsequential GBS. Each year, about 3,000 to 6,000 people develop the syndrome, according to the CDC.

It’s also been associated with other vaccines, including the H1N1 pandemic flu vaccine. But a 2013 study looking at cases at Kaiser Permanente Northern California from 1995 to 2006 found the link was weak between GBS and common vaccinations.

In its statement, GSK also pointed to an analysis (PDF) presented at the February meeting of the CDC’s Advisory Committee on Immunization Practices, which offers vaccination recommendations. In backing routine vaccination of Shingrix in people 50 years of age and older, the agency’s herpes zoster work group said that available data, including from observational studies, were limited on the risk of GBS following herpes zoster infection and vaccination.

https://www.fiercepharma.com/vaccines/fda-slaps-new-warning-glaxosmithkline-s-vaccine-shingrix-based-postmarketing-findings

Epigenomics runs out of road

 Precisely seven years ago it was possible to consider Epigenomics and Exact Sciences alongside one another, as two companies heading to the US FDA with colorectal cancer tests whose clinical data showed them to be less accurate than the cheaper standard of care. Over the following years, however, their fortunes have diverged sharply. Aided by progressively larger acquisitions, Exact is now a top-10 diagnostics company making strides in everything from precision medicine to pan-cancer liquid biopsy, while Epigenomics today admitted that it had come to the end of the road and put itself up for sale. The German group blamed the Centers for Medicare & Medicaid Services’ refusal, in October 2020, to reimburse its Epi proColon blood test. An earlier attempt by the Chinese private equity firm Cathay Fortune to take the group private floundered in August 2017 after failing to gain enough support from Epigenomics’ shareholders. Perhaps this time those investors will admit that the company has no real future as an independent concern.

The contrasting fates of two colorectal cancer test developers
Company	Exact Sciences	Epigenomics
Test name	Cologuard	Epi ProColon
Pivotal trial sensitivity	92%	71%
Pivotal trial specificity	87%	81%
Adcom date	March 27, 2014	March 26, 2014
Adcom risk-benefit vote	10 to 0 in favour	5 to 4 in favour
US approval date	August 11, 2014	April 12, 2016; had been rejected twice
Current market cap	$22.0bn	$17.6m
Forecast 2026 test revenues	$3.5bn	$58m
Source: EvaluateMedTech, FDA & company releases.

 Epigenomics: 2

https://www.evaluate.com/vantage/articles/news/snippets/epigenomics-runs-out-road