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Monday, September 11, 2023

Why Shares of Bicycle Therapeutics Were Rising

 Shares of Bicycle Therapeutics (BCYC 8.97%) were up 9% as of 1:30 p.m. ET on Monday after the healthcare stock climbed as high as 10.4% earlier in the day. The company's shares are still down more than 8% so far this year.

The clinical-stage biotech company focuses on short amino acid sequences that form stable loops called bicyclic peptides, or bicycles, to treat diseases that the company says are underserved by existing therapies. Many of the company's therapies are being looked at to treat solid tumors. These bicyclic peptides bind with molecules with high affinity and selection, making them good drug candidates for a variety of conditions.

On Monday, the company's shares rose when it said that, after discussions with the Food and Drug Administration (FDA), it is planning on a phase 2/3 investigational trial for BT8009 to treat metastatic bladder cancer. The trial is now expected to begin in the first quarter of 2024. The trial will have two cohorts; the first looks at the therapy in untreated bladder cancer and the second is for patients with previously treated bladder cancer.

"We are pleased to have reached alignment with the FDA on the registrational trial design, dose selection and clinical trial endpoints that could support potential accelerated approval in a broad metastatic bladder cancer population," said Santiago Arroyo, the chief development officer at Bicycle Therapeutics.

Bicycle already has a development deal that could be worth $1.7 billion with Roche Ag. The company also has several other ongoing collaborations, including ones with NovartisIonis Pharmaceuticals and Bayer. In the second quarter, the company reported that it had $340.4 million in cash, not counting a $45 upfront payment from Bayer in July, or the $215.5 million it received from a public stock sale in July. The company reported a net loss of $42.6 million in the quarter, but with its strong cash position, it has plenty of time to develop its pipeline, which includes 13 other assets besides BT8009.

https://www.fool.com/investing/2023/09/11/why-shares-of-bicycle-therapeutics-were-rising-on/

Hoth: FDA Accepts Pre-Investigational New Drug Submission for Treatment for Cancer

 


HT-KIT received Orphan Drug Designation from FDA for its mRNA Frame Shifting Cancer Therapeutic

Hoth intends to initially target mast cell neoplasms for development of HT-KIT, which is a rare, aggressive cancer with poor prognosis

https://www.biospace.com/article/releases/hoth-therapeutics-announces-fda-accepts-pre-investigational-new-drug-pre-ind-submission-for-ht-kit-treatment-for-cancer-patients/

New Data Validates Evaxion AI Platform to Reduce Risk, Cost in Infectious Disease Vaccine Development

  • Evaxion is the first organization to validate an AI model that outcompetes state-of-the-art vaccine development
  • Evaxion's AI model predicts vaccine efficacy in infectious disease models
  • These results mean using EDENTM will allow for a faster, cheaper, and lower-risk vaccine development compared to current approaches

FDA Takes Action on Updated mRNA COVID-19 Vaccines

 Today, the U.S. Food and Drug Administration took action approving and authorizing for emergency use updated COVID-19 vaccines formulated to more closely target currently circulating variants and to provide better protection against serious consequences of COVID-19, including hospitalization and death. Today’s actions relate to updated mRNA vaccines for 2023-2024 manufactured by ModernaTX Inc. and Pfizer Inc. Consistent with the totality of the evidence and input from the FDA’s expert advisors, these vaccines have been updated to include a monovalent (single) component that corresponds to the Omicron variant XBB.1.5.

What You Need to Know

  • Individuals 5 years of age and older regardless of previous vaccination are eligible to receive a single dose of an updated mRNA COVID-19 vaccine at least 2 months since the last dose of any COVID-19 vaccine. 
  • Individuals 6 months through 4 years of age who have previously been vaccinated against COVID-19 are eligible to receive one or two doses of an updated mRNA COVID-19 vaccine (timing and number of doses to administer depends on the previous COVID-19 vaccine received). 
  • Unvaccinated individuals 6 months through 4 years of age are eligible to receive three doses of the updated authorized Pfizer-BioNTech COVID-19 Vaccine or two doses of the updated authorized Moderna COVID-19 Vaccine.
  • The FDA is confident in the safety and effectiveness of these updated vaccines and the agency’s benefit-risk assessment demonstrates that the benefits of these vaccines for individuals 6 months of age and older outweigh their risks.
  • Individuals who receive an updated mRNA COVID-19 vaccine may experience similar side effects as those reported by individuals who previously received mRNA COVID-19 vaccines as described in the respective prescribing information or fact sheets.
  • The updated vaccines are expected to provide good protection against COVID-19 from the currently circulating variants. Barring the emergence of a markedly more virulent variant, the FDA anticipates that the composition of COVID-19 vaccines may need to be updated annually, as is done for the seasonal influenza vaccine. 
  • The U.S. Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices will meet tomorrow (Sept. 12), to discuss clinical recommendations on who should receive an updated vaccine, as well as further considerations for specific populations such as immunocompromised and older individuals. 
  • Manufacturers have publicly announced that the updated vaccines would be ready this fall, and the FDA anticipates that the updated vaccines will be available in the near future.

“Vaccination remains critical to public health and continued protection against serious consequences of COVID-19, including hospitalization and death,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “The public can be assured that these updated vaccines have met the agency’s rigorous scientific standards for safety, effectiveness, and manufacturing quality. We very much encourage those who are eligible to consider getting vaccinated.”

The updated mRNA vaccines are each approved for individuals 12 years of age and older and are authorized under emergency use for individuals 6 months through 11 years of age. As part of today’s actions, the bivalent Moderna and Pfizer-BioNTech COVID-19 vaccines are no longer authorized for use in the United States.

Data Supporting the Updated mRNA COVID-19 Vaccines (2023-2024 Formula)

The mRNA COVID-19 vaccines approved and authorized today are supported by the FDA’s evaluation of manufacturing data to support the change to the 2023-2024 formula and non-clinical immune response data on the updated formulations including the XBB.1.5 component. 

  • The updated mRNA vaccines are manufactured using a similar process as previous formulations. In studies that have been recently conducted, the extent of neutralization observed by the updated vaccines against currently circulating viral variants causing COVID-19, including EG.5 and BA.2.86, appears to be of a similar magnitude to the extent of neutralization observed with prior versions of the vaccines against corresponding prior variants against which they had been developed to provide protection. This suggests that the vaccines are a good match for protecting against the currently circulating COVID-19 variants.
  • The benefit-risk profile of previously authorized and approved mRNA COVID-19 vaccines is well understood as these vaccines have been administered to hundreds of millions of people in the United States. 

Based on an evaluation of the totality of the evidence, the benefit-risk profile is favorable for individuals 6 months of age and older to receive an updated COVID-19 mRNA vaccine. Although serious outcomes from COVID-19 are less common in younger individuals, they do occur, and it has been demonstrated that recently receiving a COVID-19 vaccine reduces the risk of such serious outcomes.

Additional Details on Today’s Actions

Specifically, today’s actions include:

  • Approval of Comirnaty (COVID-19 Vaccine, mRNA) to include the 2023-2024 formula, and a change to a single dose for individuals 12 years of age and older. Comirnaty was previously approved as a two-dose series for individuals 12 years of age and older. 
  • Approval of Spikevax (COVID-19 Vaccine, mRNA) to include the 2023-2024 formula, a change to a single dose for individuals 18 years of age and older, and approval of a single dose for individuals 12 through 17 years of age. Spikevax was previously approved as a two-dose series for individuals 18 years of age and older. 
  • Authorization of Moderna COVID-19 Vaccine for emergency use in individuals 6 months through 11 years of age to include the 2023-2024 formula and lower the age eligibility for receipt of a single dose from 6 years to 5 years of age. Additional doses are also authorized for certain immunocompromised individuals ages 6 months through 11 years, as described in the fact sheets.
  • Authorization of Pfizer-BioNTech COVID-19 Vaccine for emergency use in individuals 6 months through 11 years of age to include the 2023-2024 formula. Additional doses are also authorized for certain immunocompromised individuals ages 6 months through 11 years, as described in the fact sheets.

The approval of Comirnaty (COVID-19 Vaccine, mRNA) (2023-2024 Formula) was granted to BioNTech Manufacturing GmbH. The EUA amendment for the Pfizer-BioNTech COVID-19 Vaccine (2023-2024 Formula) was issued to Pfizer Inc.

The approval of Spikevax (COVID-19 Vaccine, mRNA) (2023-2024 Formula) was granted to ModernaTX Inc. and the EUA amendment for the Moderna COVID-19 Vaccine (2023-2024 Formula) was issued to ModernaTX Inc.

https://www.fda.gov/news-events/press-announcements/fda-takes-action-updated-mrna-covid-19-vaccines-better-protect-against-currently-circulating

The Government Censored Me and Other Scientists. We Fought Back—and Won

 When I was four, my mother took her first flight and first trip out of her native India to the U.S. with me and my younger brother in tow. We were going to meet my father, an electrical engineer and rocket scientist by training, who had won the U.S. visa lottery in 1970. He had moved to New York a year earlier. By the time we arrived he was working at McDonald’s because engineering jobs had dried up during a recession.

Both of my parents—children of the violent partition of India and East Pakistan (now Bangladesh)—had grown up in poverty, my mother in a Calcutta slum. They immigrated to this country because they believed in the American dream. That belief led to the success my father ultimately found as an engineer and my mother found running a family daycare business. 

Our family had indeed won the lottery. But coming to America meant something more profound than financial opportunity. 

I remember in 1975 when a high court found that then-prime minister of India Indira Gandhi had interfered unlawfully in an election. The ruling disqualified her from holding office. In response, she declared a state of emergency, suspended democracy, censored the opposition press and government critics, and threw her political opponents in jail. I remember the shock of these events and our family’s collective relief that we were in the U.S., where it was unimaginable that such things could happen.

When I was 19, I became an American citizen. It was one of the happiest days of my young life. The immigration officer gave me a civics test, including a question about the First Amendment. It was an easy test because I knew it in my heart. The American civic religion has the right to free speech as the core of its liturgy. I never imagined that there would come a time when an American government would think of violating this right, or that I would be its target. 

Unfortunately, during the pandemic, the American government violated my free speech rights and those of my scientist colleagues for questioning the federal government’s pandemic policies. 

My parents had taught me that people here could criticize the government, even over matters of life and death, without worry that the government would censor or suppress us. But over the past three years, I have been robbed of that conviction. American government officials, working in concert with big tech companies, have attacked and suppressed my speech and that of my colleagues for criticizing official pandemic policies—criticism that has been proven prescient. 

On Friday, at long last, the Fifth Circuit Court ruled that we were not imagining it—that the Biden administration did indeed strong-arm social media companies into doing its bidding. The court found that the Biden White House, the CDC, the U.S. Surgeon General’s office, and the FBI “engaged in a years-long pressure campaign [on social media outlets] designed to ensure that the censorship aligned with the government’s preferred viewpoints.” 

The judges described a pattern of government officials making “threats of ‘fundamental reforms’ like regulatory changes and increased enforcement actions” if we did not comply. The implication was clear. To paraphrase Al Capone: Nice company you have there. It’d be a shame if something were to happen to it.

It worked. According to the judges, “the officials’ campaign succeeded. The platforms, in capitulation to state-sponsored pressure, changed their moderation policies.”

In exposing this behavior—and in declaring it a likely violation of the First Amendment—the ruling is not just a victory for my fellow scientists and me, but for every single American.

The trouble began on October 4, 2020, when my colleagues and I—Dr. Martin Kulldorff, a professor of medicine at Harvard University, and Dr. Sunetra Gupta, an epidemiologist at the University of Oxford—published the Great Barrington Declaration. The Declaration called for an end to economic lockdowns, school shutdowns, and similar restrictive policies on the grounds that they disproportionately harm the young and economically disadvantaged while conferring limited benefits to society as a whole. 

The Declaration endorsed a “focused protection” approach that called for strong measures to protect high-risk populations while allowing lower-risk individuals to return to normal life with reasonable precautions. Tens of thousands of doctors and public health scientists signed our statement.

With hindsight, it is clear that this strategy was the right one. Sweden, which in large part eschewed lockdown and, after early problems, embraced focused protection of older populations, had among the lowest age-adjusted all-cause excess deaths than nearly every other country in Europe and suffered none of the learning loss for its elementary school children. Similarly, Florida has seen lower cumulative age-adjusted all-cause excess deaths than lockdown-obsessed California since the start of the pandemic. 

But at the time, our proposal was viewed by high government officials like Anthony Fauci and some in the Trump White House, including Deborah Birx, then-White House Coronavirus Response Coordinator, as a kind of heresy.

Federal officials immediately targeted the Great Barrington Declaration for suppression because it contradicted the government’s preferred response to Covid. Four days after the Declaration’s publication, then-director of the National Institutes of Health, Dr. Francis Collins, emailed Fauci to organize a “devastating takedown” of it. 

Almost immediately, social media companies such as Google/YouTube, Reddit, and Facebook censored mentions of the Declaration. 

As The Free Press revealed in its Twitter Files reporting, in 2021 Twitter blacklisted me for posting a link to the Great Barrington Declaration. YouTube censored a video of a public policy roundtable of me with Florida governor Ron DeSantis for the crime of telling him that the scientific evidence for masking children is weak. 

I have been a professor researching health policy and infectious disease epidemiology at a world-class university for decades. I am not a political person; I am not registered with either party. In part that is because I want to preserve my total independence as a scientist. I have always viewed my job as telling people honestly about the data issues, regardless of whether Democrats or Republicans liked the message. 

Yet at the height of the pandemic, I found myself smeared for my supposed political views, and my views about Covid policy and epidemiology were removed from the public square on all manner of social networks. I could not believe this was happening in the country I so love. 

In August 2022, my colleagues and I finally had a chance to fight back. The Missouri and Louisiana attorneys general asked me to join as a plaintiff in their case, represented by the New Civil Liberties Alliance, against the Biden administration. The aim of the suit was to end the government's role in this censorship—and restore the free speech rights of all Americans in the digital town square.

Lawyers in the Missouri v. Biden case deposed representatives, under oath, from many federal agencies involved in the censorship efforts, including Anthony Fauci. 

Broad discovery of email exchanges between the government and social media companies showed an administration willing to use its regulatory powers against social media companies that did not comply with censorship demands. 

The case revealed that a dozen federal agencies—including the CDC, the Office of the Surgeon General, and the Biden White House—pressured social media companies like Google, Facebook, and Twitter to censor and suppress even true speech contradicting federal pandemic priorities. For instance, in 2021, the White House threatened social media companies with damaging regulatory action unless it censored scientists who shared the demonstrable fact that the Covid vaccines do not prevent people from getting Covid. 

True or false, if speech interfered with the government’s priorities, it had to go.

On Independence Day this year, federal Judge Terry Doughty issued a preliminary injunction in the case, ordering the federal government to immediately stop coercing social media companies to censor protected free speech. In his decision, Justice Doughty compared the administration’s censorship infrastructure to an Orwellian Ministry of Truth. His ruling decried the vast federal censorship enterprise that dictated who and what social media companies could publish. 

The government appealed, convinced it should have the power to censor scientific speech. An administrative stay followed and lasted much of the summer. But on Friday, a three-judge panel of the U.S. Court of Appeals for the Fifth Circuit unanimously restored a modified version of the preliminary injunction, telling the government to stop using social media companies to do its censorship dirty work: 

Defendants, and their employees and agents, shall take no actions, formal or informal, directly or indirectly, to coerce or significantly encourage social-media companies to remove, delete, suppress, or reduce, including through altering their algorithms, posted social-media content containing protected free speech. That includes, but is not limited to, compelling the platforms to act, such as by intimating that some form of punishment will follow a failure to comply with any request, or supervising, directing, or otherwise meaningfully controlling the social media companies’ decision-making processes.

As I read the decision, I was overcome with emotion. I think my father, who died when I was 20, would be proud that I played a role in this. I know my mother is.

That is because the victory is not just for me but for every American who felt the oppressive force of this censorship industrial complex during the pandemic. It is a vindication for parents who advocated for some semblance of normal life for their children but found their Facebook groups suppressed. It is a vindication for vaccine-injured patients who sought the company and counsel of fellow patients online but found themselves gaslit by social media companies and the government into thinking their personal experience of harm was all in their heads. 

The decision provides some solace for scientists who had deep reservations about lockdowns but censored themselves for fear of the reputational damage that came with being falsely labeled misinformers. They were not wrong in thinking science wasn’t working right; science simply cannot function without free speech.

The decision isn’t perfect. Some entities at the heart of the government’s censorship enterprise can still organize to suppress speech. For instance, the Cybersecurity and Infrastructure Security Agency (CISA) within the Department of Homeland Security can still work with academics to develop a hit list for government censorship. And the National Institute of Allergy and Infectious Diseases (NIAID), Fauci’s old organization, can still coordinate devastating takedowns of outside scientists critical of government policy.

But the headline is a good one: the federal government can no longer threaten social media companies with destruction if they don’t censor on behalf of the government. 

The Biden administration, which has proven itself to be an enemy of free speech, will surely appeal the decision to the Supreme Court. But I am hopeful that we will win there, just as we have at every venue in this litigation. I am grateful for the resilience of the U.S. Constitution, which has withstood this challenge. 

But I can never go back to the uncomplicated faith and naive confidence I had in America when I was young. Our government is not immune to the authoritarian impulse. I have learned the hard way that it is only we, the people, who must hold an overreaching government accountable for violating our most sacred rights. Without our vigilance, we will lose them.

Jay Bhattacharya, MD, PhD, is a professor of health policy at Stanford University School of Medicine, where he researches epidemiology and health economics. He is a founding fellow of the Academy for Science and Freedom, a Hillsdale College initiative.


https://www.thefp.com/p/i-fought-government-censorship-and-won

Health Care Data – Think Globally, Act Locally

 Congress must pass reauthorization of the Pandemic and All Hazards Preparedness Act (PAHPA) before the end of the month.

COVID-19 was the worst pandemic in a century. The most fundamental obstacle we had in responding adequately to the virus was the lack of reliable, real-time data to guide public health officials and caregivers. Reauthorization is an opportunity to help fix this.

Throughout the COVID-19 pandemic, most states and local governments were operating off weeks-old data. This resulted in disjointed responses that failed to make effective use of available resources and confused public guidance.

The U.S. Centers for Disease Control and Prevention took a great deal of criticism for its lack of useful information during the pandemic – among other things. Much of that criticism is deserved.

However, we need to be cautious about avoiding a logic trap. We must not focus so much on the CDC that we assume the answer to timely, real-time health data can be found within the CDC. In fact, this is precisely what CDC leaders have proposed. They want all local health data to flow to the CDC first for the agency to analyze, and then spit back out to local health care agencies and providers.

A federal takeover of healthcare data would be the wrong approach. The CDC will never operate fast enough. Plus, it won’t be able to tailor the information to serve specific local needs. Instead, as the saying goes, we need to think globally, but act locally. We need a federated data system that serves local public health needs – but one that is nationally accessible with appropriate safeguards.

Fortunately, there were some bright spots during the pandemic that offered other models to follow. Nebraska had perhaps the best model of using health data to benefit patients, providers, and payers – while improving population health. It created a public-private partnership, now called CyncHealth, with a governance structure that allows providers, payers, patients, and the government a voice in how health data will be used. (At Gingrich 360, we consult with CyncHealth on communications strategy.)

Critically, CyncHealth does not think of itself as a health information exchange, which is the model in most states. Instead, its approach is to serve as a health data utility. Rather than simply moving information from one place to another, the health data utility model builds tools to be used by providers in the delivery of care, and payers (including the government) to monitor population health.

Stakeholder buy-in and client-customer relationships help overcome provider and payer resistance to the information-sharing requirements needed to make any system like this possible.

The benefits of this utility model were on full display during COVID-19. Within weeks of the virus coming to American shores, Nebraska public health officials had a command dashboard monitoring real-time ICU availability and other key data points. It took other states months to develop these capabilities. Some states still don’t have them.

This access to real-time data enabled by Nebraska’s Health Data Utility is one reason Nebraska ranked No. 2 in the National Bureau of Economic Research’s rankings of state responses to COVID-19. Real-time information allowed Nebraska public health officials to tactically deploy resources where they were needed. They didn’t have to rely as much on the blunt tool of lockdowns as states that were operating in the dark.

Nebraska’s Health Data Utility also generated better information for the CDC’s effort to monitor the virus nationally. But critically, Nebraska did not have to rely on the CDC.

This is the basic model we need for health data: infrastructure that serves a local utility purpose for public and private stakeholders that improves quality of care, conserves public resources, and gets appropriate communicable disease data to the CDC.

The advantages of this model go far beyond responding to public health emergencies. The model can also improve quality of care, make it easier for patients to move between providers, help states better manage their Medicaid programs, and more. There is virtually no aspect of health care that would not be improved with better real-time data.

States should take advantage of funds allocated during the pandemic to build their own health data utility infrastructure. States should not try to run it themselves. Instead, they should model their HDU after the successful public-private partnerships in Nebraska that give all healthcare stakeholders a seat at the table. However, to help make it successful, states should require that all health systems, pharmacies, and skilled nursing facilities (our nation’s largest Medicaid and Medicare spend areas) share health data with the newly established utility so they are equally invested in its success.

It is vital that CDC receive accurate, timely information to fulfill its mission. But the fact is, health care is delivered at the local level. Healthcare solutions must be designed to meet local needs. That can only be achieved with local leadership and local buy-in from providers, patients, and public health officials.

Congress should utilize PAHPA reauthorization to ensure states have the resources and guidance to set up appropriate systems.

EyePoint interim Phase 2 macular edema safety data

EyePoint Pharmaceuticals Inc 

 announced interim masked safety data for its lead product candidate EYP-1901 from its ongoing Phase 2 PAVIA trial of EYP-1901 as a potential nine-month treatment for moderately severe to severe non-proliferative diabetic retinopathy and DAVIO 2 trial as a potential six-month sustained delivery maintenance treatment for wet age-related macular degeneration

All treatment arms in the PAVIA trial have reached at least three months post-dosing follow-up as of September 1, 2023.

Approximately 170 patients have received EYP-1901 with a minimum of three months of follow-up post-injection from the ongoing Phase 2 PAVIA and DAVIO 2 trials and the completed DAVIO 1 trial with no reported drug-related ocular severe adverse events (SAEs) and no reported drug-related systemic SAEs.

In the PAVIA clinical trial, there have been no reported drug-related ocular SAEs and drug-related systemic SAEs. There were two ocular SAEs deemed unrelated to EYP-1901 by investigators:

  • Hemorrhagic posterior vitreous detachment (PVD) in a study eye eight weeks after dosing
  • Macular edema leading to vision loss in the non-study fellow eye

The company remains on track to share topline results from the DAVIO 2 trial in December of this year and the PAVIA trial in the second quarter of 2024.

https://www.benzinga.com/general/biotech/23/09/34446050/whys-going-on-with-eyepoint-pharmaceuticals-stock-today