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Friday, September 9, 2022

With 34 Vaccine Programs in Development, Moderna Eyes Global Launches

Moderna is preparing for multiple product launches over the next several years, including RSV and flu vaccines, boosters for COVID-19 and mRNA programs for rare diseases.

Between 2023 and 2026, Moderna anticipates multiple vaccine launches across the globe, beginning with boosters aimed at the various strains of COVID-19. In 2023, Moderna aims to pursue approval under the accelerated pathway for its flu vaccine mRNA-1010. As part of its launch plans, Moderna said it will continue investing in its manufacturing capabilities to support vaccine production.

On Thursday, the Cambridge, Mass.-based company hosted its annual R&D day for investors. Leadership showcased the company’s clinical programs across multiple indications, including 34 vaccine programs in development.

During its presentation, Chief Executive Officer Stéphane Bancel called that a remarkable number. He said Moderna has always been excited about the use of mRNA technology, nothing that the software-like nature of RNA has had the company excited about the mRNA modality.

“We are investing for other launches and we are investing for scale… because with the pipeline that is coming, it is important that we do that to maximize the potential for patients,” Bancel said.

Rare Diseases

Among its presentations were proof-of-concept studies on rare diseases. Because mRNA is like software for the body, Bancel stated the company would be delving into rare diseases more and more due to the potential of the technology.

These include potential therapeutics for multiple rare diseases, including propionic academia and methylmalonic acidemia, metabolic disorders that share disease pathology and glycogen storage diseases. For propionic academia (PA), the company is developing mRNA-3927, which encodes for two proteins that can help the body make the missing enzyme absent in PA patients. Moderna is enrolling patients in a Phase I/II study that will evaluate the safety and pharmacology of mRNA-3927.

In methylmalonic academia (MMA), the company has enrolled the first two groups of patients in a Phase I/II study assessing the safety and pharmacology of mRNA-3705. The mRNA-based therapy is designed to instruct the body to restore the missing or dysfunctional proteins that cause MMA.

For glycogen storage disease 1a, Moderna initiated the Phase I/II Ba1ance trial assessing a single intravenous dose of mRNA-3745. Enrollment in the trial is ongoing, and the company anticipates data in 2023. Moderna is also advancing a new development candidate, mRNA-3139, a potential drug for ornithine transcarbamylase deficiency. It uses the same lipid nanoparticle as the mRNA-3745 program for GSD1a.

Respiratory Vaccines

Moderna has already seen significant success with its COVID-19 vaccine. The company continues to develop booster doses that address new strains of the SARS-CoV-2 virus that run rampant across the globe.

Moderna has launched two vaccine boosters to meet different needs across the largest markets: mRNA-1273.214 and mRNA-1273.222. mRNA-1273.214 targets both the original strain of SARS-CoV-2 and the Omicron variant of concern (BA.1). Moderna’s mRNA-1273.222 targets the Omicron BA.4 and BA.5 variants, combined with mRNA-1273. A Phase II/III trial for this candidate is underway.

The company’s seasonal influenza vaccine candidate, mRNA-1010, is in Phase III development in the Southern hemisphere, with approximately 6,000 patients. Moderna aims to initiate a Phase III trial in the Northern Hemisphere designed to evaluate mRNA-1010 against a licensed seasonal influenza vaccine.

In RSV (respiratory syncytial virus), Moderna launched a Phase III trial that will evaluate mRNA-1345 in older adults aged 60 and above. The test has enrolled more than 24,000 patients so far. Full enrollment is expected at 34,000 patients. The study has been designed to provide an efficacy readout in the 2022-2023 winter season. In addition to older adults, RSV is also a virus of concern in young children. Moderna is also assessing mRNA-1345 in a Phase I trial in pediatric populations. There is currently no vaccine available for RSV.

In addition to single-agent vaccines, Moderna is advancing several combination respiratory vaccines, including a fully enrolled Phase I/II trial testing mRNA-1073 for both COVID-19 and influenza. The company is also conducting a study of mRNA-1230, a vaccine candidate for COVID-19, influenza and RSV. A clinical trial for this vaccine is expected to be initiated this year.

CMV and Cancer Vaccines

Moderna is developing a vaccine for cytomegalovirus (CMV), a type of latent herpes virus known to cause long-term health problems in newborns. The company is assessing mRNA-1647 in the Phase III CMVictory that is designed to prevent congenital CMV. Enrollment in this study is ongoing.

The company is also developing personalized cancer vaccines, including mRNA-4157, which is being assessed in combination with Merck’s Keytruda in patients with resected melanoma at high risk of recurrence. The combination is in Phase II, and primary data is expected by the end of the fourth quarter of this year.

https://www.biospace.com/article/with-34-vaccine-programs-in-development-moderna-eyes-multiple-global-launches/

U.S. Starts Enrollment in Trial Testing Siga's Antiviral for Monkeypox

The National Institutes of Health (NIH) said on Friday it had started enrolling monkeypox patients in a late-stage study testing Siga Technologies Inc's antiviral pill Tpoxx against the disease.

The oral and intravenous formulations of Tpoxx are approved by the U.S. Food and Drug Administration for the treatment of smallpox, but does not yet have clearance to treat monkeypox.

It is, however, currently accessible by clinicians for treating monkeypox under a compassionate use request.

The NIH aims to enroll more than 500 patients, including both adults and children, who will then be randomized to receive either Tpoxx or placebo pills for 14 days.

Investigators will evalutate if participants receiving Tpoxx heal more quickly compared to placebo, as well as provide critical data on the optimal dosing and safety of the drug in children and people who are pregnant.

The United States has recorded more than 21,000 confirmed cases of monkeypox, according to data from the Centers for Disease Control and Prevention.

https://www.usnews.com/news/top-news/articles/2022-09-09/u-s-starts-enrollment-in-trial-testing-sigas-antiviral-for-monkeypox

Galapagos cut to Equal Weight from Overweight by Morgan Stanley

Target to $61 from $80

https://finviz.com/quote.ashx?t=GLPG&ty=c&ta=1&p=d

NY declares state of emergency over polio to boost low vaccination rates

New York Gov. Kathy Hochul on Friday declared a state of emergency over polio to boost vaccination rates in the state amid further evidence that the virus is spreading in communities.

Poliovirus has now been detected in sewage samples from four counties in the New York metro area as well as in the city itself. The counties are Rockland, Orange, Sullivan, and the latest, Nassau.

The samples tested positive for poliovirus that can cause paralysis in humans, according to state health officials. Unvaccinated individuals who live, work, go to school or visit Orange, Rockland, Nassau, New York City and Sullivan are at the highest risk of paralytic disease, officials said.

New York began wastewater surveillance after an unvaccinated adult caught polio in Rockland County in July and suffered from paralysis, the first known infection in the U.S. in nearly a decade.

The emergency declaration will expand the network of vaccine administrators to include pharmacists, midwives and EMS workers in an effort to boost the immunization rate in areas where it has slipped.

New York Health Commissioner Dr. Mary Bassett called on people who are unvaccinated to get their shots immediately. Individuals and families who are unsure of their vaccination status should contact a health care provider, clinic or the county health department to make sure they are up to date on their shots.

“On polio, we simply cannot roll the dice,” Bassett said. “I urge New Yorkers to not accept any risk at all. Polio immunization is safe and effective — protecting nearly all people against disease who receive the recommended doses.”

The polio vaccination rate is alarmingly low in some New York counties. The vaccination rate is 60% in Rockland, 58% in Orange, 62% in Sullivan and 79% in Nassau, according to the health department. The statewide average for polio immunization is about 79%.

The goal of the vaccination campaign is to boost immunization rates well above 90% statewide, according to the health department.

Some New Yorkers should get boosted

Some New Yorkers who completed their vaccine series should receive a single lifetime booster shot, health officials said. These individuals include people who might have contact with someone infected or thought to be infected with poliovirus or members of the infected person’s household.

Health care workers should also get a booster if they work in areas where poliovirus has been detected and they might handle specimens or treat patients who may have polio. People who might be exposed to wastewater due to their job should also consider getting a booster, health officials said.

All children should receive four doses of the polio vaccine. The first dose is administered between 6 weeks and 2 months of age, the second dose is given at 4 months, the third at 6 months to 18 months, and the fourth dose at 4 to 6 years old.

Adults who have only received one or two doses should get the remaining one or two. Health officials said it doesn’t matter how long ago the first doses were given.

How poliovirus spreads

Polio spreads between people when the virus enters the mouth, typically through hands contaminated with the stool of an infected person. The virus often spreads unnoticed because 70% of people infected do not show symptoms. About 25% of those infected develop mild symptoms similar to the flu.

One in 100 people infected develop severe disease such as permanent paralysis. Polio is fatal in 2% to 10% of people suffering from paralysis because the muscles used to breathe are immobilized.

The chain of transmission that introduced polio to New York is thought to have originated from abroad in someone who received the oral polio vaccine. The oral vaccine uses a weakened form of the virus that still replicates. In rare cases, the virus used in the vaccine can mutate, become virulent and spread to others.

The U.S. stopped using the oral vaccine more than two decades ago. It now uses a vaccine administered as a shot in which the virus is inactivated, which means it does not replicate and mutate. While this vaccine is very effective at preventing disease, it does not block transmission of the virus.

The oral polio vaccine can block transmission of poliovirus that occurs in nature, but carries the risk that the strain used in the vaccine could mutate to become virulent and result in spread of what’s called vaccine-derived poliovirus.

https://www.cnbc.com/2022/09/09/new-york-declares-state-of-emergency-over-polio-to-boost-vaccination-rate.html

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