Latest news with #PeterMarks
Axios
15-07-2025
- Business
- Axios
DOGE makes its way into biotech IPO filings
DOGE cuts are leading to new warnings in filings from biotech and health-care companies looking to IPO stateside. The big picture: Uncertainty is a deal killer. According to data from Renaissance Capital, there have been just four biotech IPOs this year that have raised over $50 million — down from 10 in the same period last year. Venture bets are slowing too. Driving the news: Carlsmed, a personalized spine surgery company backed by B Capital Group and U.S. Venture Partners warned in its prospectus that "the current U.S. presidential administration" has issued policies to cut headcount and budgets in agencies including the FDA. "It remains unclear the degree to which these efforts may limit or otherwise adversely affect the FDA's ability to conduct routine activities," it continued. Sixth Street-backed cancer diagnostics company, Caris Life Sciences, which went public in late June, said this: "The current Trump administration is pursuing policies to reduce regulations and expenditures across government including at HHS, the FDA, CMS, and related agencies," Caris' prospectus read. "These actions ... may propose policy changes that create additional uncertainty for our business. Zoom out: Though Carlsmed says it's "unclear the degree to which" the cuts will affect the FDA's ability to conduct routine activity, those in the space already feel the effects. The FDA told KalVista Pharmaceuticals last month that the agency would miss a deadline in approving a drug for potentially life-threatening genetic disorders — marking what appears to be the first time a review had to be extended over DOGE cuts. Verve Therapeutics, in a filing explaining its rationale for selling to Eli Lilly for $1.3 billion, noted that Peter Marks resignation in March as director of the FDA played a role in its move to sell rather than raise additional capital.
CBS News
03-07-2025
- Health
- CBS News
RFK Jr.'s FDA officials overrode career staff to limit COVID shots
Top-ranking Food and Drug Administration officials picked by the Trump administration under Health and Human Services Secretary Robert F. Kennedy Jr. overrode career staff at the agency to limit the approvals of COVID-19 shots from Novavax and Moderna, according to records released by the FDA. In a memorandum following Novavax's approval in May, which narrowed the shot's label to only seniors ages 65 and older and others at-risk of severe disease, the FDA's Dr. Vinay Prasad said that he disagreed with the agency's career vaccine reviewers. Prasad was named earlier this year to replace Dr. Peter Marks, the longtime head of the agency's center regulating vaccines and other biologics, who was ousted under Kennedy. Prasad was later elevated to the FDA's chief medical and scientific officer as well, absorbing the roles of other top-ranking FDA officials who were removed or left under the Trump administration. Novavax had applied for a license for its shot to prevent COVID-19 for anyone over 12 years old, effectively converting it to a traditional approval from the emergency use authorizations the FDA had granted to the vaccine during the pandemic to allow its use at the time. Prasad wrote that he questioned the data submitted by Novavax, said COVID-19's risk of severe disease had dropped in the U.S. population, and cited safety concerns that he said "fundamentally alters the benefit-risk calculation in non-high-risk individuals." "The decrease in the chance of developing severe COVID-19, means that the potential for absolute benefit from vaccination has simultaneously decreased. Even rare vaccination-related harms, both known and unknown, now have a higher chance of outweighing potential benefits in non-high-risk populations," Prasad wrote. A separate memo released by the agency from Captain Edward Wolfgang, chair of the review team for Novavax's shot, said the changes for who should be approved to get the vaccine followed requests by Prasad and the special assistant to the FDA commissioner. The agency's career scientists had initially concluded on April 1 that Novavax's data submitted in its Biologics License Application, or BLA, and COVID-19's ongoing public health threat warranted broad approval for use in everyone ages 12 and older. "The data submitted with this BLA indicate the safety and efficacy of a single dose of Nuvaxovid (2024-2025 Formula) meet the statutory requirements to support its use in individuals 12 years of age and older to prevent COVID-19 caused by SARS-CoV-2," they concluded. Documents released last month about the approval of Moderna's new COVID-19 vaccine for only seniors ages 65 and older and others with at least one underlying condition down to age 12 outline a rationale similar to the one Prasad used in his decision about the Novavax shot. "The review team has done a commendable job in summarizing and analyzing the submission to date. Nevertheless, I disagree with certain aspects of their conclusions and instead reach the conclusion described below," Prasad wrote in his memo for Moderna's approval, dated May 30 and titled "CENTER DIRECTOR OVERRIDE MEMO." The New York Times previously reported that the FDA had released the documents. Trade publication Pink Sheet previously reported that Prasad and his aides had blocked agency plans to grant a broad license for Novavax's vaccine, delaying its approval. Under Marks, Prasad's predecessor, it was uncommon but not unheard of for the head of the Center for Biologics Evaluation and Research to intervene in the decisions to green-light vaccines. But it is virtually unprecedented for the FDA commissioner or his political appointees to step in to directly intervene or question traditional approval decisions, FDA officials have said. "The tradition in 99.95% of FDA decisions about individual products is that those decisions are made by career civil servants. The commissioner actually has no role in that, unless there's an internal dissent and an appeal, or in some cases, an external appeal that makes it all the way up to the commissioner level," Dr. Robert Califf, then the FDA commissioner under the Biden administration, had said at an event last year. In past criticism of the agency, FDA Commissioner Dr. Marty Makary has often cited a move by Marks to green-light booster shots during the COVID-19 pandemic that led to the ouster of two top career vaccines officials, who questioned the decision. One of those former FDA officials — Dr. Phil Krause — is also now criticizing moves by Makary and his aides as threatening to erode the agency's credibility. Agency leaders under Kennedy have made other unprecedented moves in recent months to wrest control over the nation's vaccines process. Months before Kennedy fired the Centers for Disease Control and Prevention's influential vaccine advisory committee and replaced them with his own picks, the FDA replaced the career official who usually works as liaisons to the CDC panel with Dr. Tracy Beth Hoeg. Hoeg is the special assistant to the FDA commissioner. Along with Makary, Hoeg questioned the agency's approach to approving vaccines during the COVID-19 pandemic. A spokesperson for the Department of Health and Human Services, which has largely replaced the agency's career media relations staff who were mostly laid off in April, rejected the characterization of Prasad overruling FDA scientists as "a distortion of the facts." "He evaluated the totality of the evidence and made a judgment rooted in gold standard science. That's not political — it's what principled leadership looks like," the spokesperson said in a statement. The spokesperson said the FDA "will continue to follow the science and use evidence-based decision-making," echoing frustrations voiced by Prasad and others picked by Kennedy about using long COVID to justify vaccine approvals, backing boosters for healthy young adults and the desire to bring U.S. vaccine policy in line with other countries. Makary and Prasad also made this argument in May, after they announced a new "framework" that would limit all COVID-19 vaccine approvals to only seniors and others at-risk, unless vaccine makers were able to generate new clinical trial data. "Dr. Prasad is correcting course with data, with transparency, and with the courage to say what others won't. That's how trust in science is rebuilt," the HHS spokesperson said. The FDA's approvals decide whether vaccinemakers are allowed to sell their shots in the U.S. market at all. Those approvals are usually followed by CDC recommendations on how they should be used, which are influential because they are tied to federal policies that enable access to vaccines, like guaranteeing insurance coverage. Health care providers are also allowed to give vaccines "off-label" outside of the FDA's label and CDC recommendations, once the FDA has approved sales, though that risks running afoul of insurance coverage and liability protections.
Boston Globe
16-06-2025
- Health
- Boston Globe
Sarepta reports second patient death after treatment with Duchenne gene therapy
Advertisement The company said early Sunday it was working with experts to come up with an enhanced immunosuppressive regimen that could make the therapy, called Elevidys, safer for non-ambulatory patients. It said it would talk with the Food and Drug Administration about the the proposed regimen. Sarepta also said it was pausing dosing in an ongoing clinical trial of Elevidys, called ENVISION, that's focused on older ambulatory and non-ambulatory patients. The company did not disclose the age of the person who died, but a statement indicated it was a male patient. Duchenne almost always affects boys, More than 900 patients have received Elevidys overall. 'We are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient's family and his care team during this incredibly difficult time,' Louise Rodino-Klapac, Sarepta's chief scientific officer, said in a statement. Advertisement 'We know how much hope families place in new treatment options like Elevidys — and we are committed to honoring that hope by acting swiftly, guided by scientific rigor and the insights of leading experts, to strengthen safety for all future patients,' she added. The death is certain to add additional scrutiny to Elevidys has become a key medicine caught up in the debate about regulatory flexibility — with patients and some government officials advocating for speeding treatments to patients with rare diseases who have few options — and effectiveness standards that drugs have traditionally had to clear to win approval. At the FDA, a top official, Peter Marks In the case of Elevidys, the dispute centered on efficacy, with the treatment appearing safe in clinical trials. However, Sarepta studied the therapy largely in young children, and the risks of gene therapy generally increase with age. When the FDA granted its approval, some experts raised concerns about the treatment's safety in older patients. FDA reviewers noted last year that Sarepta submitted data for only 16 patients over the age of 8 and only eight patients who were non-ambulatory. They highlighted that there were no significant safety differences in those older, non-ambulatory patients, but that 'the limited safety data limits meaningful assessment.' Advertisement The FDA's new leadership under the Trump administration
Yahoo
27-05-2025
- Business
- Yahoo
Here's Why Vaxcyte (PCVX) Sold Off in Q1
Carillon Tower Advisers, an investment management company, released its 'Carillon Eagle Small Cap Growth Fund' first quarter 2025 investor letter. A copy of the letter can be downloaded here. Small-cap stocks experienced a significant drop in Q1, with the Russell 2000 Growth Index (down 11.12%) trailing the Russell 2000 Value Index (down 7.74%). In the Russell 2000 Growth Index, real estate, which increased by 1.76%, outperformed all sectors both absolutely and relatively. The only other sector delivering positive returns was consumer staples, which rose by 0.67%. In addition, please check the fund's top five holdings to know its best picks in 2025. In its first-quarter 2025 investor letter, Carillon Eagle Small Cap Growth Fund highlighted stocks such as Vaxcyte, Inc. (NASDAQ:PCVX). Vaxcyte, Inc. (NASDAQ:PCVX) is a clinical-stage biotechnology vaccine company that focuses on developing novel protein vaccines to prevent or treat bacterial infectious diseases. The one-month return of Vaxcyte, Inc. (NASDAQ:PCVX) was -1.09%, and its shares lost 52.68% of their value over the last 52 weeks. On May 23, 2025, Vaxcyte, Inc. (NASDAQ:PCVX) stock closed at $32.54 per share with a market capitalization of $4.198 billion. Carillon Eagle Small Cap Growth Fund stated the following regarding Vaxcyte, Inc. (NASDAQ:PCVX) in its Q1 2025 investor letter: "Vaxcyte, Inc. (NASDAQ:PCVX) is a leading biotechnology company focused on developing vaccines. The company is developing VAX24/VAX-31 for the prevention of pneumococcal disease in infants and adults. Phase 2 trial results for VAX-24 in infants were announced, demonstrating non-inferiority to another drug on nearly all serotypes tested. While the results were promising enough to proceed to phase 3, they were not as robust as previous VAX-24 trials. Additionally, the resignation of a key U.S. Food and Drug Administration leader, Dr. Peter Marks, shortly before the results were unveiled, raised concerns in the vaccine industry and contributed to a significant selloff in the stock. Despite these challenges, the company remains optimistic about the future of VAX-24 in phase 3 trials." A research scientist in a laboratory holding a vial of a biotechnology drug. Vaxcyte, Inc. (NASDAQ:PCVX) is not on our list of 30 Most Popular Stocks Among Hedge Funds. As per our database, 45 hedge fund portfolios held Vaxcyte, Inc. (NASDAQ:PCVX) at the end of the first quarter, which was 50 in the previous quarter. While we acknowledge the potential of Vaxcyte, Inc. (NASDAQ:PCVX) as an investment, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns, and doing so within a shorter timeframe. If you are looking for an AI stock that is as promising as NVIDIA but that trades at less than 5 times its earnings, check out our report about the undervalued AI stock set for massive gains. In another article, we covered Vaxcyte, Inc. (NASDAQ:PCVX) and shared the list of stocks that will bounce back according to analysts. The fund stated in its Q4 2024 investor letter that Vaxcyte, Inc. (NASDAQ:PCVX) underperformed due to uncertainty surrounding its key vaccine candidates amid political shifts. In addition, please check out our hedge fund investor letters Q1 2025 page for more investor letters from hedge funds and other leading investors. READ NEXT: Michael Burry Is Selling These Stocks and A New Dawn Is Coming to US Stocks. Disclosure: None. This article is originally published at Insider Monkey.
Yahoo
27-05-2025
- Business
- Yahoo
Here's Why Vaxcyte (PCVX) Sold Off in Q1
Carillon Tower Advisers, an investment management company, released its 'Carillon Eagle Small Cap Growth Fund' first quarter 2025 investor letter. A copy of the letter can be downloaded here. Small-cap stocks experienced a significant drop in Q1, with the Russell 2000 Growth Index (down 11.12%) trailing the Russell 2000 Value Index (down 7.74%). In the Russell 2000 Growth Index, real estate, which increased by 1.76%, outperformed all sectors both absolutely and relatively. The only other sector delivering positive returns was consumer staples, which rose by 0.67%. In addition, please check the fund's top five holdings to know its best picks in 2025. In its first-quarter 2025 investor letter, Carillon Eagle Small Cap Growth Fund highlighted stocks such as Vaxcyte, Inc. (NASDAQ:PCVX). Vaxcyte, Inc. (NASDAQ:PCVX) is a clinical-stage biotechnology vaccine company that focuses on developing novel protein vaccines to prevent or treat bacterial infectious diseases. The one-month return of Vaxcyte, Inc. (NASDAQ:PCVX) was -1.09%, and its shares lost 52.68% of their value over the last 52 weeks. On May 23, 2025, Vaxcyte, Inc. (NASDAQ:PCVX) stock closed at $32.54 per share with a market capitalization of $4.198 billion. Carillon Eagle Small Cap Growth Fund stated the following regarding Vaxcyte, Inc. (NASDAQ:PCVX) in its Q1 2025 investor letter: "Vaxcyte, Inc. (NASDAQ:PCVX) is a leading biotechnology company focused on developing vaccines. The company is developing VAX24/VAX-31 for the prevention of pneumococcal disease in infants and adults. Phase 2 trial results for VAX-24 in infants were announced, demonstrating non-inferiority to another drug on nearly all serotypes tested. While the results were promising enough to proceed to phase 3, they were not as robust as previous VAX-24 trials. Additionally, the resignation of a key U.S. Food and Drug Administration leader, Dr. Peter Marks, shortly before the results were unveiled, raised concerns in the vaccine industry and contributed to a significant selloff in the stock. Despite these challenges, the company remains optimistic about the future of VAX-24 in phase 3 trials." A research scientist in a laboratory holding a vial of a biotechnology drug. Vaxcyte, Inc. (NASDAQ:PCVX) is not on our list of 30 Most Popular Stocks Among Hedge Funds. As per our database, 45 hedge fund portfolios held Vaxcyte, Inc. (NASDAQ:PCVX) at the end of the first quarter, which was 50 in the previous quarter. While we acknowledge the potential of Vaxcyte, Inc. (NASDAQ:PCVX) as an investment, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns, and doing so within a shorter timeframe. If you are looking for an AI stock that is as promising as NVIDIA but that trades at less than 5 times its earnings, check out our report about the undervalued AI stock set for massive gains. In another article, we covered Vaxcyte, Inc. (NASDAQ:PCVX) and shared the list of stocks that will bounce back according to analysts. The fund stated in its Q4 2024 investor letter that Vaxcyte, Inc. (NASDAQ:PCVX) underperformed due to uncertainty surrounding its key vaccine candidates amid political shifts. In addition, please check out our hedge fund investor letters Q1 2025 page for more investor letters from hedge funds and other leading investors. READ NEXT: Michael Burry Is Selling These Stocks and A New Dawn Is Coming to US Stocks. Disclosure: None. This article is originally published at Insider Monkey. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
