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UT Southwestern develops nanotherapeutic to ward off liver cancer

Physician researchers from UT Southwestern Medical Center have developed an innovative nanotherapeutic drug that prevents cancer from spreading to the liver in mice. Credit: UT Southwestern Medical Center Physician researchers from UT Southwestern Medical

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Physician researchers from UT Southwestern Medical Center have developed an innovative nanotherapeutic drug that prevents cancer from spreading to the liver in mice.

Credit: UT Southwestern Medical Center

Physician researchers from UT Southwestern Medical Center have developed an innovative nanotherapeutic drug that prevents cancer from spreading to the liver in mice.

The new liver-specific microRNA drug, developed by a team led by Andrew Wang, M.D., is a promising candidate for drug companies that developed messenger RNA (mRNA) vaccines for COVID-19, because of similarities in these RNA agents.

“This might be one ray of hope that comes out of the pandemic,” said Dr. Wang, Professor, Radiation Oncology, and author of a rodent-based study published in the journal Cancer Research.

“It takes major funding and resources to develop nanoparticles that can deliver nucleic acids such as mRNA and miRNA. Before the development of COVID-19 vaccines, the cost was prohibitive. But now that several platforms have been developed and approved, these platforms/nanoparticles can be utilized for other applications such as what we developed in in mice models in my lab,” added Dr. Wang, Radiation Oncology’s Associate Vice Chair for Research, and member of the Harold C. Simmons Comprehensive Cancer Center.

The study drug’s core was made by complexing miR-122 with calcium phosphate, and lipids were wrapped around the core to make the nanoparticle. The drug delivers the miR-122 into hepatocytes, which make them “healthier” by helping prevent cancer cells from establishing themselves in the liver. Although the drug has only been tested in mice, it is a valued advance in the fight against cancer, as up to 70 percent of people with conditions like colorectal cancer eventually develop liver metastases.

“Liver metastases are second only to lung metastases, so new therapeutics in this area are an urgent need in oncology. Dr. Wang’s study is promising because it showed minimal toxicity,” said Carlos L. Arteaga, M.D., Director of the Harold C. Simmons Comprehensive Cancer Center, who holds The Lisa K. Simmons Distinguished Chair in Comprehensive Oncology.

Funding for the study and initial drug development was provided by National Cancer Institute/National Institutes of Health U54CA198999, NIH T32CA196589 and the University of North Carolina Research Opportunity Initiative.  

About UT Southwestern Medical Center

UT Southwestern, one of the nation’s premier academic medical centers, integrates pioneering biomedical research with exceptional clinical care and education. The institution’s faculty has received six Nobel Prizes, and includes 25 members of the National Academy of Sciences, 16 members of the National Academy of Medicine, and 14 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,800 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UT Southwestern physicians provide care in about 80 specialties to more than 117,000 hospitalized patients, more than 360,000 emergency room cases, and oversee nearly 3 million outpatient visits a year.


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As Omicron-specific boosters are in the news, BioNTech to expand German site workforce by 200 employees

BioNTech has added about 200 jobs to its Marburg, Germany manufacturing site since it took it over from Novartis in 2020. This year, it will add another 250, as demand for Covid-19 boosters has led to calls for an omicron-specific vaccine in adults.
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BioNTech has added about 200 jobs to its Marburg, Germany manufacturing site since it took it over from Novartis in 2020. This year, it will add another 250, as demand for Covid-19 boosters has led to calls for an omicron-specific vaccine in adults.

Currently, there are around 500 employees at the site, which is north of Frankfurt. The boost is expected to cost around €50 million, or $56.5 million, and will include the addition of office spaces in anticipation of future growth, the company said. The Marburg site has supplied 1.2 billion Covid-19 vaccine doses to the world so far, and a total of 4 billion doses are expected to be doled out by the end of this year.

The news came out of an interview with Bloomberg, a spokesperson for the company confirmed. The $56 million expansion is planned for just the Marburg site alone, the spokesperson confirmed. Before the sale to BioNTech, Novartis invested in the site heavily since 2016, equipping it with capabilities for cell and gene therapy, recombinant proteins, cell culture and viral vector production. In the future, once the need for Covid-19 boosters dies down, BioNTech has said it will other mRNA vaccines, antibody and C&G therapy candidates, as well as its cancer and infectious disease pipeline.

Testing for the vaccine tailored to the pesky variant — which has been deemed more transmissible with less severe symptoms for those that are vaccinated — began this week, BioNTech said along with Pfizer. The trial will test 1,420 healthy adults under the age of 55. Three groups of people, representing different levels of immunity,

In March 2021, Marburg began producing Covid-19 vaccines. It started with the production of mRNA, the active pharmaceutical ingredient for the vaccine. The first batch of doses shipped out in April.

Uğur Şahin

The site has become an example for further growth. BioNTech made waves in the summer of 2021 when it announced plans to collaborate with the African Union to manufacture doses for the continent amid a severe shortage, and in October, the company said that its Rwandan site, which construction is set to begin in the middle of this year, will be modeled after Marburg. Capacity for that one will start at 50 million doses a year, then increase with added manufacturing lines and sites as the project progresses.

CEO Uğur Şahin has already said that the Rwandan site will spin its production into malaria and tuberculosis vaccines once there is a less desperate need for Covid-19 manufacturing.

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Roche nabs a priority review for Evrysdi, hoping to become the first oral treatment for infants with SMA

Roche has been duking it out with Biogen and Novartis ever since bursting into the spinal muscular atrophy space about a year and a half ago with its competitively priced oral treatment Evrysdi. Now the pharma giant is looking to stake its claim in a…

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Roche has been duking it out with Biogen and Novartis ever since bursting into the spinal muscular atrophy space about a year and a half ago with its competitively priced oral treatment Evrysdi. Now the pharma giant is looking to stake its claim in a younger subset of patients — and the FDA has agreed to give it a speedy review.

Regulators have granted priority review to a supplemental NDA for Evrysdi (risdiplam) to treat pre-symptomatic babies under 2 months old with SMA. While the drug is currently approved for adults, children and babies older than 2 months, a new approval here would make it the first at-home treatment available for younger infants. A decision is expected by May 30, according to Roche.

“Treating very young babies with Evrysdi before SMA symptoms arise may help them to achieve milestones such as standing and walking within timeframes typical of healthy infants,” CMO Levi Garraway said in a statement.

Evrysdi — licensed by Roche from PTC Therapeutics — is designed to treat SMA by modifying how the SMN2 gene is spliced, thus increasing functional SMN protein levels in both the central nervous system and peripheral tissues. SMN protein is critical for maintaining healthy motor neurons and movement.

The drug got a quick OK back in 2020, becoming the first oral treatment for SMA. Analysts penciled in a peak sales estimate of around $2 billion. Its competitors, Biogen’s Spinraza and Novartis’ Zolgensma, are administered by injection to the spinal cord and IV infusion, respectively.

Matthew Klein

“I think that that’s really important. One, just in general, saving people the inconvenience of going to a hospital. I think most people would much rather take something by mouth than have a procedure done, and probably even more important in COVID,” PTC COO Matthew Klein told Endpoints News. “I think people are really not wanting to… leave home, let alone go to medical facilities.”

At an interim analysis in the RAINBOWFISH study, all five patients under 2 months old maintained the ability to swallow and were able to feed exclusively by mouth after 12 months of treatment, according to Roche. Four of those five patients achieved milestones such as standing and walking independently, and all met the HINE-2 motor milestones of head control, sitting upright, rolling and crawling after 12 months.

The drug appeared safe, with no treatment-related serious events reported in any of the babies. Four treatment-emergent side effects were reported out of 12 babies, all of which were resolved or resolving with ongoing Evrysdi treatment. The most common side effects were nasal congestion (33%), cough (25%), teething (25%), vomiting (25%), eczema (17%), abdominal pain (17%), diarrhea (17%), gastroenteritis (17%), papule (17%) and pyrexia (17%).

“The AEs were reflective of the age of the babies rather than the underlying SMA,” Roche said.

From the start, Roche promised to “underwhelm” with Evrysdi’s price compared to competitors. The pharma giant set the price on a scale based on a child’s weight, maxing out when they reach 44 lbs at about 6 years of age. For example, for an infant who weighs 15 lbs and is less than 2 years old, the annual price of Evrysdi would be less than $100,000, a spokesperson told Endpoints.

Meanwhile, Zolgensma has earned the notorious title of the most expensive drug in the world at $2.1 million per patient, and back in 2020, Spinraza cost between $625,000 and $750,000 for the first year and around $375,000 every year after.

Evrysdi’s lower price hasn’t stopped it from reeling in mammoth sales. In Q3 of 2021, the drug netted around $429 million, compared to Zolgensma’s $375 million and Spinraza’s $$444 million. Spinraza’s Q3 sales decreased from $494 million in Q3 of 2020, which Biogen attributed to growing competition and the impacts of Covid-19.

Roche shelled out $30 million upfront for the rights to Evrysdi back in 2011, promising up to $460 million in biobucks and up to double-digit royalties on commercial sales. PTC raked in $35 million in milestone payments in Q3 of 2020 and $7.5 million in Q4, according to the company’s year-end report. The drug is currently approved in 70 countries and has been submitted for approval in 31 more.

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Sanofi claims high-rises neighboring its Toronto facility would pose ‘national security’ threat

Sanofi isn’t so happy with one of its neighbors. The French pharma giant is going to battle against a Canadian developer’s proposal to build two, 30-plus-story residential towers less than half a mile away from its Toronto facility, citing “national…

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Sanofi isn’t so happy with one of its neighbors. The French pharma giant is going to battle against a Canadian developer’s proposal to build two, 30-plus-story residential towers less than half a mile away from its Toronto facility, citing “national security concerns.”

The news comes just under a year after Sanofi announced it’s dumping more than $676 million into a new vaccine manufacturing facility at the Toronto site, which will be responsible for additional antigen and filling capacity for its Fluzone high-dose quadrivalent vaccine. The new facility should be up and running in 2026, the company said back in March.

In a letter to city planners, a lawyer representing Sanofi detailed its concerns about plans to redevelop a four-story housing unit just a few blocks down the road, which would add 31- and 37-story towers, and a 12-story mid-rise.

“The location of hundreds of new residential units with a 24/7 overlook of its sensitive facilities undermines Sanofi’s ability to ensure its ongoing and expanding vaccine research and manufacturing facilities are secure, which represents national security concerns given the strategic importance of the site for vaccine manufacturing and future pandemic readiness,” Sanofi’s lawyer stated in the letter, dated in November.

The developer, Tenblock, submitted an application for the redevelopment back in October 2020. The plans include tearing down the existing four-story building at 1875 Steeles West — which was built in 1973 and currently houses 120 units — and constructing three new buildings containing 866 units total.

While the original plan was for two 38- and 39-story towers, Tenblock reduced the plans to 37 and 31 stories, respectively, in response to “well-received City and community feedback,” it explained in a resubmitted proposal.

Stephen Job

The proposal also includes the restoration and expansion of the existing park and public trail space adjacent to the site.

“We have a longstanding coexistence with our valued neighbours Sanofi Pasteur. Our redevelopment proposal is a continuation of our site’s 50-year history as a residential apartment building,” VP Stephen Job wrote in an email to Endpoints News. 

Sanofi’s facility, which currently comprises more than 40 buildings, has been at the site for over 100 years. In addition to its fears over espionage, Sanofi noted that it’s investing in significant upgrades to ensure it has access to sufficient and reliable water services and electricity for its operations and wants to ensure that it “continues to have unrestricted access to the upgraded infrastructure that it is paying to have installed.” The company declined to comment further.

James Pasternak

City councilor James Pasternak, who represents Ward 10 where Sanofi’s facility is located, is siding with the pharma on this one.

“The industry has been there for 100 years, so it’s prudent not to build residential right next door. It’s just not compatible,” he said in a phone interview.

Other local ratepayers expressed concerns at a recent community meeting that the project would create an inappropriate density in the area, Pasternak said, adding that the area consists mostly of what the city calls “employment lands.” There are no high-level transit or subway stations, he said, and the towers could pose electrical and water pressures on other community members.

As for Tenblock? “I don’t think they’re willing to budge,” Pasternak said, adding that this will likely be an ongoing discussion for at least another year. “No one is telling these landowners they cannot build. What we were telling them is they should be building something much smaller. We’re suggesting either townhomes or mid rises. So far, they haven’t really come back with a plan that we can accept.”

According to CBC, which broke the news, the city is hoping to convince Tenblock to lower the towers to about 10 stories.

However, Job maintained that the current proposal meets all local guidelines.

“Our expert consultants have recently completed technical compatibility studies as required by the City of Toronto’s planning review process. These studies confirm that our redevelopment proposal complies with all regulatory and policy requirements,” he said. “We will continue to collaborate with all stakeholders including Sanofi Pasteur to achieve a high-quality and appropriate outcome.”

The news comes as a growing number of biopharma companies fight to protect trade secrets. Earlier this month, a third former GlaxoSmithKline scientist pleaded guilty to conspiracy after being caught in an alleged plot to steal trade secrets and sell the work in China. Last January, immuno-oncologist Shafat Quadri was charged with stealing “thousands” of top-secret research documents from Merck before moving to a new job at a rival company. That March, AbbVie filed suit against Alvotech, accusing the Icelandic biotech of embarking on an “unlawful plot to surreptitiously take AbbVie’s confidential and proprietary trade secrets.” A federal judge dismissed that lawsuit in October.

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