Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome

Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis

TSHA-102 anticipated to submit Investigational New Drug application in 2021

Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-102, an AAV9-based gene therapy in development for the treatment of Rett syndrome. Taysha anticipates that it will submit an Investigational New Drug (IND) application for TSHA-102 to the FDA in 2021.

Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of over 25,000 cases in the U.S. and European Union (EU). It is an X-linked disease that primarily occurs in females, but it can be seen very rarely in males. It is usually recognized in children between six to 18 months of age as they begin to miss developmental milestones or lose abilities they had developed. Individuals with Rett syndrome also show symptoms that include loss of speech, loss of purposeful use of hands, loss of mobility, seizures, cardiac impairments, breathing issues and sleep disturbances.

“Patients with Rett syndrome are currently managed with symptomatic treatments as there are no therapies approved to treat the underlying cause of disease,” said Berge Minassian, M.D., Chief Medical Advisor of Taysha and Chief of Pediatric Neurology at the University of Texas Southwestern Medical Center (UT Southwestern). Dr. Minassian is credited with describing the CNS isoform of the MECP2 gene which is responsible for neuronal and synaptic function throughout the brain. “Gene therapy offers a potentially curative option for patients suffering with Rett syndrome.”

Rett syndrome is caused by mutations in the MECP2 gene. TSHA-102 is designed to deliver a healthy version of the MECP2 gene as well as the miRNA-Responsive Auto-Regulatory Element, miRARE, platform technology to control the level of MECP2 expression. “TSHA-102 represents an important step forward in the field of gene therapy, where we are leveraging a novel regulatory platform called miRARE to prevent the overexpression of MECP2,” said Steven Gray, Ph.D., Chief Scientific Advisor of Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. “In collaboration with Sarah Sinnett, Ph.D. to develop miRARE, our goal was to design a regulated construct that allowed us to control MECP2 expression to potentially avoid adverse events that are typically seen with unregulated gene therapies.”

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect under 200,000 people in the U.S. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are

Advocates Stand Up Against AstraZeneca to Save Drug Discount Program for Vulnerable Populations

Wilmington Protest, Wed., Oct. 14th – 12 noon – 1:00 pm ET

Dozens of concerned healthcare advocates from across the northeast region protest AstraZeneca, one of five U.S. based pharmaceutical companies that have cut back on the number of drugs they provide through the 340B federal drug discount program

Please replace the release with the following updated version due to multiple revisions.

The updated release reads:

DELAWARE PROTEST: ADVOCATES STAND UP AGAINST ASTRAZENECA TO SAVE DRUG DISCOUNT PROGRAM FOR VULNERABLE POPULATIONS

Wilmington Protest, Wed., Oct. 14th – 12 noon – 1:00 pm ET

Dozens of concerned healthcare advocates from across the northeast region protest AstraZeneca, one of five U.S. based pharmaceutical companies that have cut back on the number of drugs they provide through the 340B federal drug discount program.

Healthcare advocates from across the northeast region will protest the recent actions of AstraZeneca, in cutting back the number of critical life-saving drugs provided at discounted rates to non-profit healthcare providers, through the federal 340B drug discount program.

WHAT:

Protest against AstraZeneca

 

 

WHERE:

AstraZeneca’s Corporate Office

 

1800 Concord Pike, Wilmington, DE 19803

 

NOTE: The protest will take place at the intersection of Powder Mill Road & Route 22

 

 

WHEN:

Tomorrow, Wednesday, October 14, 2020

 

12noon – 1pm (EDT)

 

 

WHO:

Healthcare advocates from across the Northeast Region

The federal 340B Drug Discount Program is a lifeline that allows safety net providers, including HIV/AIDS clinics receiving funding through the Ryan White program, to obtain prescription drugs at below-retail prices. The program was established with bipartisan support as part of the Veterans Health Care Act of 1992. With 340B savings, Ryan White clinics are able to stretch their grant funds, offer a wider range of services, and improve the quality of care persons living with HIV/AIDS receive. The program also benefits qualified 340B covered entities such as non-profit rural health facilities, community clinics and children’s hospitals that serve vulnerable populations.

Tomorrow’s protest, led by healthcare advocates from AIDS Healthcare Foundation (AHF), follows a lawsuit filed by Ryan White Clinics for 340B Access (RWC-340B) against the U.S. Department of Health and Human Services (HHS), in U.S. District Court for the District of Columbia, to require the HHS secretary to take action against AstraZeneca and three other pharmaceutical companies, including Eli Lilly, Novartis and the U.S. division of Sanofi-Aventis which are illegally withholding drugs they are required to sell through the 340B program. With tomorrow’s protest, AHF is demanding that these greedy pharmaceutical companies stop their bullying tactics that will have a devastating impact on the healthcare and well-being of our most vulnerable populations (see LITIGATION PRESS RELEASE). RWC340B also recently released a study on the potential adverse impact of policies reducing resources to Ryan White clinics, see WHITE PAPER, PRESS RELEASE, and FACT SHEET.

“AstraZeneca has launched an assault on a federal drug discount program essential to the safety net of our nation’s health care,” stated John Hassell, AHF’s national director of advocacy. “They are messing with the numerous health care centers

South Korea’s Celltrion gets approval for Phase 3 trials of COVID-19 antibody drug

By Sangmi Cha

SEOUL (Reuters) – South Korean drugmaker Celltrion Inc <068270.KS> said on Monday it has received regulatory approval for Phase 3 clinical trials of an experimental COVID-19 treatment.

The approval comes as the company plans to seek conditional approval for its antibody drug, CT-P59, for emergency use by the end of this year.

The treatment, the most advanced antibody drug in terms of research in South Korea, is directed against the surface of the virus and designed to block it from locking on to human cells.

The third stage trials will be conducted on some 1,000 asymptomatic coronavirus patients and those who have come into close contact with COVID-19 patients in Korea, Celltrion said in a statement.

The Ministry of Food and Drug Safety recently approved a Phase 2/3 study on patients with mild and moderate cases of COVID-19, Lee Sang-joon, Celltrion’s senior executive vice president, told Reuters.

Celltrion began commercial production of the drug in September – likely to amount to around 1 million doses – in anticipation of demand in both domestic and overseas markets.

In July, Celltrion separately launched overseas human trials of its treatment in Britain.

(Reporting by Sangmi Cha; Editing by Kenneth Maxwell)

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Hong Kong scientists say anti-microbe drug successful against coronavirus

An affordable anti-microbial drug used to treat stomach ulcers and bacterial infections has shown promise in combatting the coronavirus in animals, scientists in Hong Kong announced Monday.

Researchers set out to explore whether metallodrugs — compounds containing metal that are more commonly used against bacteria — might also have anti-viral properties that could fight the SARS-CoV-2 coronavirus.

Using Syrian hamsters as tests subjects, they found that one of the drugs, ranitidine bismuth citrate (RBC), was “a potent anti-SARS-CoV-2 agent”.

“RBC is able to lower the viral load in the lung of the infected hamster by tenfold,” Hong Kong University researcher Runming Wang told reporters on Monday as the team presented their study.

“Our findings demonstrate that RBC is a potential anti-viral agent for Covid-19.”

The coronavirus has killed more than a million people since it first emerged in China last December and then spread across the globe.

As scientists scramble to find a vaccine, they have also been scouring readily available drugs that might alleviate symptoms caused by the Covid-19 disease or help the body fight infection.

Remdesivir, a broad-spectrum antiviral drug, and dexamethasone, a type of corticosteroid, have both been identified as having some success against the virus. Both were used by doctors to treat US President Donald Trump after he contracted Covid-19.

But they have drawbacks.

Remdesivir is expensive and there is a global shortage while dexamethasone has immunosuppression effects that are risky for all but the most ill patients. Other drug cocktails have shown liver damage can be a risk.

The Hong Kong scientists said RBC was a commonly available drug used against stomach ulcers with a safe and comprehensive pharmacological profile.

“It’s been used for decades so it’s pretty safe,” Wang said.

They added that their research, which has been published in the journal Nature Microbiology, suggested other metallodrugs might also have success against the virus and should be further explored.

jta/qan

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Trump’s COVID drug was developed using aborted fetal tissue

The antibody cocktail that President Trump received for his COVID-19 infection and touted on Wednesday evening as a “cure” for the deadly virus was developed using cells derived from aborted fetal tissue, a practice the White House and anti-abortion rights groups oppose.



Amanda Banks, Alveda King, Donald Trump standing in front of a crowd holding a sign: Anti-Abortion Activists Demonstrate In D.C. During Annual March For Life


© Mark Wilson / Getty Images
Anti-Abortion Activists Demonstrate In D.C. During Annual March For Life

Last week, Mr. Trump received Regeneron Pharmaceuticals’ cocktail of monoclonal antibodies, an experimental therapeutic for coronavirus that is still undergoing testing and is not FDA approved. In a nearly five-minute video posted to Twitter on Wednesday, the president lauded its effects, calling it “the key.”

“I think this was a blessing from God that I caught [the virus], I think it was a blessing in disguise,” Mr. Trump said in the video. “I caught it, I heard about this drug, I said, ‘Let me take it’ … and it was incredible the way it worked.”

But the way in which the antibody cocktail was developed is at odds with the Trump administration’s position on stem cell research. The drug’s potency was tested in a lab using HEK 293T cells, a cell line originally derived from the kidney tissue of a fetus aborted in the Netherlands in the 1970s, said a spokesperson for Regeneron in an email to CBS News on Thursday. The cells “were used in testing the antibody candidates’ ability to neutralize the virus” and helped researchers “determine the ‘best’ two antibodies, which now make up the REGN-COV2 cocktail,” the spokesperson said.  

Doctor discusses President Trump’s health, treatments, and comments calling therapeutics a cure

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There is no fetal tissue present in the final product.

Remdesivir, an antiviral drug Mr. Trump received, also was tested using the HEK 293T cells.

Last year, the Trump administration said it would no longer support long-standing funding for medical research by government scientists using human fetal tissue, a move that countered advice from physicians and researchers. The decision was seen as a major victory for anti-abortion rights groups.

Because the fetal cells used in developing Regeneron’s antibody cocktail were originally derived from an abortion prior to the funding ban, a White House official told CBS News on Thursday that the therapeutic wasn’t in violation of the administration’s new policy.

“The Administration’s policy on the use of human fetal tissue from elective abortions in research specifically excluded ‘already-established (as of June 5, 2019) human fetal cell lines,” the official said. “Thus, a product made using extant cell lines that existed before June 5, 2019 would not implicate the Administration’s policy.”

Video: COVID Vaccine Trials Lacking Minority Participants (CBS Sacramento)

COVID Vaccine Trials Lacking Minority Participants

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Anti-abortion groups, which generally oppose the use of fetal tissue in pharmaceutical research, did not raise issue with the therapeutics used and promoted by the president.

“The president was not given any medicines to treat COVID-19 that involved the destruction of human life,” wrote David Prentice, Ph.D., and Tara Sander

COVID drug Trump touted as a “cure” was developed using cells derived from aborted fetal tissue

The antibody cocktail that President Trump received for his COVID-19 infection and touted on Wednesday evening as a “cure” for the deadly virus was developed using cells derived from aborted fetal tissue, a practice the White House and anti-abortion rights groups oppose.

Last week, Mr. Trump received Regeneron Pharmaceuticals’ cocktail of monoclonal antibodies, an experimental therapeutic for coronavirus that is still undergoing testing and is not FDA approved. In a nearly five-minute video posted to Twitter on Wednesday, the president lauded its effects, calling it “the key.”

“I think this was a blessing from God that I caught [the virus], I think it was a blessing in disguise,” Mr. Trump said in the video. “I caught it, I heard about this drug, I said, ‘Let me take it’ … and it was incredible the way it worked.”

But the way in which the antibody cocktail was developed is at odds with the Trump administration’s position on stem cell research. The drug’s potency was tested in a lab using HEK 293T cells, a cell line originally derived from the kidney tissue of a fetus aborted in the Netherlands in the 1970s, said a spokesperson for Regeneron in an email to CBS News on Thursday. The cells “were used in testing the antibody candidates’ ability to neutralize the virus” and helped researchers “determine the ‘best’ two antibodies, which now make up the REGN-COV2 cocktail,” the spokesperson said.  

There is no fetal tissue present in the final product.

Remdesivir, an antiviral drug Mr. Trump received, also was tested using the HEK 293T cells.

Last year, the Trump administration said it would no longer support long-standing funding for medical research by government scientists using human fetal tissue, a move that countered advice from physicians and researchers. The decision was seen as a major victory for anti-abortion rights groups.

Because the fetal cells used in developing Regeneron’s antibody cocktail were originally derived from an abortion prior to the funding ban, a White House official told CBS News on Thursday that the therapeutic wasn’t in violation of the administration’s new policy.

“The Administration’s policy on the use of human fetal tissue from elective abortions in research specifically excluded ‘already-established (as of June 5, 2019) human fetal cell lines,” the official said. “Thus, a product made using extant cell lines that existed before June 5, 2019 would not implicate the Administration’s policy.”

Anti-abortion groups, which generally oppose the use of fetal tissue in pharmaceutical research, did not raise issue with the therapeutics used and promoted by the president.

“The president was not given any medicines to treat COVID-19 that involved the destruction of human life,” wrote David Prentice, Ph.D., and Tara Sander Lee, Ph.D., of the Charlotte Lozier Institute, the research arm of the anti-abortion rights political group the Susan B. Anthony List, in a statement emailed to CBS News Wednesday afternoon. “No human embryonic stem cells or human fetal tissue were used to produce the treatments President Trump received – period.”

The researchers did not

Rheumatoid arthritis drug cuts coronavirus deaths in clinical trial, Eli Lilly says

Eli Lilly and Company said on Thursday that there were fewer deaths among coronavirus patients who were given a combination therapy involving its rheumatoid arthritis drug and Gilead Sciences Inc’s GILD.O remdesivir drug compared to those who only received remdesivir.

The company has previously shared that the drug, baricitinib, which is marketed as Olumiant, in combination with remdesivir, was shown to cut hospitalization time for coronavirus patients. The findings come from a National Institute of Allergy and Infectious Diseases-sponsored trial dubbed the Adaptive COVID-19 Treatment Trial (ACTT-2).

The company previously shared that the drug, in combination with remdesivir, was shown to cut hospitalization time for coronavirus patients. 

The company previously shared that the drug, in combination with remdesivir, was shown to cut hospitalization time for coronavirus patients. 
(Photo courtesy of Eli Lilly)

In the data shared on Thursday, the company said that the largest benefits were observed in coronavirus patients requiring supplemental oxygen, and those who required high-flow oxygen/non-invasive ventilation.

CORONAVIRUS RECOVERY TIME REDUCED BY RHEUMATOID ARTHRITIS DRUG AND REMDESIVIR, ELI LILLY SAYS

“Using the ordinal scale that ranged from recovered to death, the odds of improvement in clinical status at Day 15 were 30% greater in patients being treated with baricitinib in combination with remdesivir compared with remdesivir,” the company said in a news release. “A numerical decrease in death (35%) through Day 29 was observed in patients treated with baricitinib plus remdesivir compared to remdesivir in the overall population.”

In coronavirus patients receiving oxygen, the difference was more pronounced. Mortality at Day 29 was 60% lower in patients requiring supplemental oxygen, and 43% lower for patients requiring high-flow oxygen/non-invasive ventilation.

UCLA’S NEW $10 COVID TEST CAN PROCESS THOUSANDS OF RESULTS IN A DAY

Baricitinib is not currently approved by the FDA to treat COVID-19, although the company is gearing up to seek Emergency Use Authorization (EUA), according to the news release.

“We are excited that these results add to the potential role for baricitinib to treat hospitalized COVID-19 patients,” Ilya Yuffa, Lilly senior vice president and president of Lilly Bio-Medicines, said in a statement. “Lilly is committed to identifying impactful preventions and treatments, and we are engaged in discussions with the FDA regarding the potential to make baricitinib available to hospitalized patients as quickly as possible.”

CLICK HERE FOR COMPLETE CORONAVIRUS COVERAGE 

The company said it is working to have the full trial analysis completed and a peer-reviewed manuscript “available soon.”

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With new drug pricing order, Trump flirts with socialized medicine

President Trump’s recent executive order on drug prices gets almost everything right — except the solution. Ironically, that solution moves the United States toward socialized medicine, which the president vociferously opposes.

The order says, “Americans pay more per capita for prescription drugs than residents of any other developed country.” That’s certainly true for most brand name drugs, though Americans typically pay much less for generic drugs, which account for about 90 percent of all U.S. prescriptions — a fact often ignored in the health policy debates.

The EO is also correct that “Americans pay more for the exact same drugs, often made in the exact same places.” As a result, Americans “finance much of the biopharmaceutical innovation that the world depends on.” 

But Trump’s executive order won’t fix these problems. It will only make it as hard for American patients to obtain the newest, cutting-edge drugs as it is for many patients in foreign countries the president wants to emulate. 

The order forbids Medicare from paying more for drugs than the lowest price available in any member country of the Organization for Economic Cooperation and Development (OECD), after adjusting for per-capita income. Trump calls it a “most-favored-nation price.” 

The order claims those nations enjoy low drug prices because they “negotiate” with pharmaceutical manufacturers. But what the order describes as a negotiation is more akin to a hostage-taking — with their own citizens held for ransom. 

Bureaucrats in those nations’ systems – most of which are largely or completely controlled by the government – often refuse to cover drugs unless manufacturers sell the medicines far below fair-market prices. 

In Canada, for example, just 46 percent of new drugs approved worldwide between 2011 and 2018 are actually available to Canadian patients. And the average delay between approval and availability in Canada is 15 months. In the United Kingdom, it’s 59 percent and 18 months.

But in the United States it’s 87 percent and three months or less.

Those are months – and in some countries, years – that patients go without access to the newest treatments. Some drugs are never made available. 

The U.S. government doesn’t treat its people so callously — or at least it hasn’t. Medicare covers virtually every FDA-approved medicine, and it sets reimbursements based on prices in the commercial market. This market-based pricing ensures that the newest drugs are available and doctors, not government gatekeepers, decide which drugs to prescribe. 

It’s a shame that the president has adopted other countries’ socialized medicine prices because he so often criticizes foreign freeloading. 

Recall that when Trump took office, he saw that our NATO allies were not paying their fair share toward the alliance’s mutual defense, even though the members had for years committed to raising their defense spending to at least 2 percent of GDP to support the alliance. 

Trump did not respond to this inequity by swearing the United States would only spend as much as our stingiest ally. Instead, he called them out publicly and exhorted our allies

New Drug for Hypochondria Soon to Be OK’d (Satire)

Disclaimer: This post is from GomerBlog, a satirical site about healthcare.

Expect GiantDrugCo’s stock price to hit record levels soon — the company expects to launch the first-ever drug to treat hypochondria.

To be marketed as Placebadil, it’s an enteric-coated sucrose-(2R,3S,4S,5R,6R)-2-(hydroxymethyl)-6-[(2R,3S,4R,5R,6S)-4,5,6-trihydroxy-2-(hydroxymethyl)oxan-3-yl]oxy-oxane-3,4,5-trio, also known as “sugar” and “starch.”

In phase I and II trials, the drug was successful at treating myriad perceived illnesses such as 2-day-old sinus infections, COVID-19, brain cancers, and appendicitis. The trials were so successful that it was placed on the fast track to be approved before the end of the year.

One participant in the study was cured of her perceived endometriosis after only three doses. That study was conducted in a double-blind fashion with the participant not knowing if she was getting the placebo or the placebo. After she declared that she no longer suffered from endometriosis, she learned that she did indeed get the placebo.

Placebadil is slated to hit the market in the next 2 months. It will provide doctors with another tool in their armamentarium to treat hypochondriacs. Patients with perceived illnesses will no longer have to suffer in perceived silence.

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CytRx Highlights Use of Licensed Drug Aldoxorubicin in Treatment of Former Senate Majority Leader Harry Reid’s Pancreatic Cancer

Reid’s Stage IV Pancreatic Cancer is Reportedly in “Complete Remission” After Combination Immunotherapy That Included NantKwest’s PD-L1 t-haNK, ImmunityBio’s N-803 and Aldoxorubicin

ImmunityBio and NantKwest Announced in May 2020 That They Planned to Commence a Randomized Phase 2 Study of This Experimental Treatment for Pancreatic Cancer  

CytRx Corporation (OTCQB: CYTR) (“CytRx” or the “Company”), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today highlighted the use of its licensed drug – aldoxorubicin – in the combination immunotherapy used by ImmunityBio, Inc. and NantKwest, Inc. to treat former Senate Majority Leader Harry Reid’s stage IV pancreatic cancer. It was widely reported in June 2020 that former Senator Reid described himself as being in “complete remission” after receiving experimental treatment pioneered by the Chief Executive Officer of ImmunityBio and NantKwest.1

Earlier this year, CytRx highlighted that ImmunityBio and NantKwest announced the initiation of a Phase 2 randomized, two-cohort, open-label study for first and second-line treatment of locally advanced or metastatic pancreatic cancer (QUILT-88). The study received Food and Drug Administration (“FDA”) authorization and was slated to initially enroll 268 subjects across both cohorts. It has been indicated that enrollment was expected to begin in June 2020.

“We wish former Senator Reid the best now that he is reportedly in complete remission and hope that his combination immunotherapy treatment can become the basis for treating other individuals with pancreatic cancer,” said Steven A. Kriegsman, CytRx’s Chairman and CEO. “Although former Senator Reid is only one person and other comprehensive studies and trials are necessary and required, we continue to be encouraged with the progress and results of this promising pancreatic cancer treatment that includes aldoxorubicin.”

CytRx out-licensed global development, manufacturing and commercialization rights for aldoxorubicin to ImmunityBio in 2017. The Company has an agreement with ImmunityBio that can yield up to $343 million in potential milestone payments as well as prospective royalties on sales of aldoxorubicin.

About CytRx Corporation

CytRx Corporation (OTCQB: CYTR) is a biopharmaceutical company with expertise in discovering and developing new therapeutics principally to treat patients with cancer and neurodegenerative diseases. CytRx’s most advanced drug conjugate, aldoxorubicin, is an improved version of the widely used anti-cancer drug doxorubicin and has been out-licensed to ImmunityBio, Inc. In addition, CytRx’s other drug candidate, arimoclomol, was sold to Orphazyme A/S (Nasdaq Copenhagen exchange: ORPHA.CO) in exchange for milestone payments and royalties. Orphazyme is testing arimoclomol in four indications including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM). CytRx Corporation’s website is www.cytrx.com.

About Pancreatic Cancer

Pancreatic cancer kills an estimated 47,000 people annually; it is the fourth leading cause of cancer-related death in the U.S., and 57,600 new cases are expected in 2020. Less than 5% of these patients will live for more than five years after diagnosis, and the median survival prognosis is 5 to 8 months. Pancreatic cancer is a disease in which malignant (cancerous) cells form in the tissues of