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Tuesday, December 10, 2024

Cardiff Positive Initial Data from First-line Colorectal Cancer Trial

 - Initial results from randomized Phase 2 CRDF-004 trial evaluating onvansertib + standard of care in RAS-mut mCRC demonstrated 64% ORR in the 30mg onvansertib dose arm versus 33% ORR in the control arm -

- In the experimental arms, 30mg dose of onvansertib demonstrated a higher ORR compared to 20mg dose of onvansertib (64% vs. 50%) with deeper tumor regression in the 30mg arm -

- Onvansertib was well tolerated at both doses -

- Additional clinical data from CRDF-004 trial expected in 1H 2025 -

- Company will hold a conference call today at 8:00 a.m. ET / 5:00 a.m. PT -

Cardiff Oncology will host a conference call and live webcast at 8:00 a.m. ET / 5:00 a.m. PT on December 10, 2024. Individuals interested in listening to the live conference call may do so by using the webcast link in the "Events" section of the company's website. A webcast replay will be available in the investor relations section on the company's website following the completion of the call.

https://www.globenewswire.com/news-release/2024/12/10/2994304/0/en/Cardiff-Oncology-Announces-Positive-Initial-Data-from-First-line-RAS-mutated-mCRC-Clinical-Trial.html

Cumberland FDA Approval for Acetadote Dosing Change

 Thanks mostly to FDA approval of a supplementary New Drug Application (sNDA) for Acetadote (N-acetylcysteine for injection), Cumberland stock has been on a monumental run in December 2024. This approval streamlines the dose schedule, therefore improving safety and efficiency in treating liver damage connected to acetaminophen.


Hospitals all around have adopted the simplified strategy to lower medication mistakes and severe non-allergic anaphylactoid responses without sacrificing efficacy. This regulatory landmark has raised investor confidence, which has driven the stock price of the company much higher. Cumberland's development chances are significantly improved by the FDA Orphan Drug and Rare Pediatric Disease Designations for its Duchenne muscular dystrophy medication.


https://finance.yahoo.com/news/cumberland-stock-soars-90-fda-183912684.html

UniQure up on chance of speedy approval for Huntington’s therapy

 Shares of UniQure doubled Tuesday morning after the Netherlands-based biotechnology company trumpeted that its experimental treatment for Huntington’s disease could be eligible for a speedy kind of approval.

Known as AMT-130, the treatment is one of UniQure’s most advanced and closely watched assets. It’s a gene therapy designed to enter brain cells and block the genetic instructions for the protein that causes Huntington’s. UniQure has been periodically providing updates from two clinical trials testing multiple doses of AMT-130, and in July said the therapy appears to be slowing the progression of Huntington’s in patients who are early on in their disease.

Several months after disclosing those results, UniQure met with the Food and Drug Administration to discuss the program’s next steps. The company is now saying that during this late November meeting, the two parties agreed on some of the key elements UniQure would need if it planned to ask for “accelerated approval” — a type of clearance which allows drugs with limited supporting data to enter the market while their developers collect more evidence to confirm they are effective.

According to UniQure, the FDA said data from the ongoing trials of AMT-130 may serve as the main basis for an accelerated approval application, so long as the data are compared to an external control group of people who have early Huntington’s disease and are similar to the study participants. As such, the company would not need to conduct an additional trial before submitting its application.

UniQure said the two parties also agreed on ways to measure the effectiveness of AMT-130.

According to the company, FDA staff think a well-known scale for evaluating the progression of Huntington’s could be the main tool that shows AMT-130 is reasonably likely to benefit patients. The staff also believe reductions in “neurofilament light chain,” a protein tied to nerve cell damage, could be used as supportive evidence. Notably, Biogen’s Qalsody, a drug for ALS, won FDA approval last year because of its effects on neurofilament, which then encouraged other brain drug developers to prioritize measuring the protein in their studies.

Meetings like the one between UniQure and the FDA aren’t public, meaning the agency’s position on AMT-130 isn’t entirely clear. But the FDA’s stance, as described by UniQure, does seem fitting, given recent views expressed by top-ranking officials.


https://finance.yahoo.com/m/2523ee64-bc90-37eb-81a5-76b68bfcc35b/uniqure-shares-soar-on-chance.html

Chimerix Soars on FDA Filing Plans for Brain Cancer Therapy

 Tuesday after the biotech company revealed intentions to submit a New Drug Application to the U.S. Food and Drug Authority (FDA) seeking speedy clearance for dordaviprone, a therapy for recurrent H3 K27M-mutantdiffuse glioma, Chimerix (NASDAQ:CMRX) more than doubled, rising over 220% on Tuesday.The corporation will ask Priority Review and wants to turn in the application by the end of the year. Data from Phase 2 and Phase 3 studies, showing a 28% objective response rate and a median response duration of 10.4 months, will be included into the NDA.

Over 2,000 U.S. patients yearly suffer from severe brain cancer known as recurrent H3 K27M-mutant diffuse glioma. For this disorder, no FDA-approved treatment exists right now. Should the application be approved Priority Review Chimerix plans to start the treatment in the United States by the third quarterof 2025.

To speed patient access to the treatment, Chimerix said it had interacted withFDA, disease experts, and advocates. To assess safety and pharmacokinetics, the business is also doing Phase 1 research on another therapy, ONC206.


https://finance.yahoo.com/news/chimerix-soars-220-fda-filing-185109224.html

UK's "Open Border Experiment" Is Reversible

 by Sam Bidwell via The Critic,

As Keir Starmer lambasts the “open borders experiment” of the past few years, are Britain’s politicians finally waking up to the scale of our immigration crisis?

With the latest figures from the ONS showing that net migration to Britain stood at more than 700,000 in 2024, with total incoming migration standing at more than 1.2 million, it’s long past time that substantive action was taken. If, as the Prime Minister said last week, our experiment with mass migration has been a mistake, then politicians have a duty to atone for their wrongdoing. Naturally, that means revising our visa rules in the first place, making it more difficult for new immigrants to come to this country — but even if we change those rules, we are still left with an almighty conundrum.

Under the current immigration rules, most migrants on work and family visas will be eligible for Indefinite Leave To Remain (ILR) status after just five years. ILR status holders have a right to live and work indefinitely in the UK — and gain access to additional support from the state, in the form of services like universal credit. ILR status is also tricky to remove, without amending the Nationality, Immigration, and Asylum Act 2002.

Combine this legal mechanism with a migration wave of several million people, and the result is a slow-motion fiscal car crash. Fail to act now, and in less than five years, millions of low-wage, low-skilled workers will be eligible for a lifetime of support from the British state. After just a few short years in the workforce, the British taxpayer could be left on the hook for a migrant’s benefits, social housing costs, healthcare, state pension — and the cost of any dependents that they choose to bring to the UK. This would, effectively, lock in this migration wave — deepening the fiscal impact of migration, and making it more legally challenging to remove those who came here over the past few years.

At a time when the UK already has limited fiscal headroom thanks to years of sluggish growth, is it fair to add billions of pounds in additional costs to the burden carried by the British taxpayer?

As research from the excellent Karl Williams at the Centre for Policy Studies shows, just 5 percent of work visas handed out in 2022-23 were given to migrants likely to be net contributors — with 72 percent of “skilled work visa” holders earning less than the UK average salary.

The OBR’s analysis admits that the average low-wage migrant worker costs the taxpayer £465,000 by the time they reach the age of 81.

Now, imagine millions of these low-wage migrant workers, all gaining eligibility for a lifetime of support from the state — clearly, our current ILR rules are not fit for purpose. 

Fortunately, revision of those rules is actually relatively easy.

As I argue in my recent research for the Adam Smith Institute, the Home Secretary has the power to change the conditions for ILR by issuing a Statement Of Changes In Immigration Rules, using their powers under the Immigration Act 1971 to change rules around entry and settlement. Unless Parliament passes a resolution within forty days condemning the changes, then those new rules pass into law — it really is as simple as that.

The Home Secretary could change the standard eligibility criteria for ILR from five years to fifteen, while leaving the five-year rule in place for migrants from the United States, the European Union, the Anglosphere, and the Asian Tiger economies. This distinction would help to ensure that Britain remains a competitive destination for high-value, high-skilled migrants, while addressing the bulk of migrants, from countries like India, Nigeria, and Pakistan. As my oven-ready legislative framework makes clear, Parliament might also choose to signal its support for the Home Secretary by passing a bespoke ouster clause, shielding these changes from pernicious judicial review. In our increasingly litigious culture, one can never be too careful.

This wouldn’t be the first revision of ILR rules, either — in 2006, then-Home Secretary Charles Clarke changed the eligibility time from four years to five. Nor would it leave Britain as a European outlier; in recent years, the Netherlands has extended its own settlement eligibility period from five years to ten, and Sweden has created new rules to allow the Government to revoke settled status from migrants.

For those of us concerned about the long-term social and fiscal impact of migration, a revision of the ILR rules is the best short-term use of our energy; the savings to the taxpayer speak for themselves. It would also give the Government time and space to think about whether or not it wants to reissue visas to those who arrived over the last few years. After all, a five year visa grant does not entitle an individual to a lifetime of residence in the UK — access to this country is time-limited by design. If the Government feels that mass migration has been a mistake, then it would be well-within its rights to refuse to reissue visas to low-skilled workers who arrived since 2021. This becomes much easier if our ILR rules are revised, preventing long-term settlement in the first instance.

The question is not one of possibility — as my research lays out, this change is both practically possible and politically defensible. It would probably also be enormously popular with the public — at a time when trust in politicians is already low, this would represent the most substantive policy intervention on migration for decades.

The question is one of political will, and sincerity of conviction. If Keir Starmer and his colleagues are serious about addressing uncontrolled mass migration, then they have a duty to lay these changes before Parliament. If this experiment really has been a mistake, then why should the British people live with the impacts for decades to come? If they fail to do so, then we can only assume that their platitudes about addressing this issue were just that — platitudes, empty words designed to comfort a frustrated electorate.

As a country, we deserve better than that — we deserve real action. It’s time to address Britain’s impending ILR crisis.

https://www.zerohedge.com/political/uks-open-border-experiment-reversible

Grifols Names Two New Board Members in Win for Hedge Fund Mason

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Grifols SA, the drugmaker reeling from a short-seller attack earlier this year, named two new board members, as it faces pressure from a group of small shareholders led by hedge fund Mason Capital Management LLC.

Grifols will appoint Pascal Ravery, a former executive at Goldman Sachs Group Inc. and JPMorgan Chase & Co., and Paul S. Herendeen, who has served as an executive at several health firms, to fill two vacant seats on the board, the Spanish company said in a filing late Monday.

https://www.bloomberg.com/news/articles/2024-12-10/grifols-names-two-new-board-members-in-win-for-hedge-fund-mason

20 Largest Digital Exporters In The World

 Today, approximately half of the world’s service exports are made on digital platforms.

While the top exporting countries of digital services are largely concentrated in developed countries, export markets in India and China are rising significantly. In 2023, India’s digital service exports topped $257 billion, rising by 17% annually. Meanwhile, the value of China’s digital service exports grew by almost twofold between 2019 and 2023 to reach $207 billion.

This graphic, via Visual Capitalist's Dorothy Neufeld, shows the top 20 largest exporters of digital services in the world, based on data from the World Trade Organization.

Ranked: The Top Exporting Countries of Digital Services

To start, digital services are defined as those that are traded through electronic networks, such as apps or digital platforms.

They can encompass a broad range of activities including IT support, media streaming, R&D, and financial services. Given that a higher volume of business and consumer transactions are made online, digital services are making up a growing share of the global economy.

Here are the top exporting countries of digitally-delivered services in 2023:

With $649 billion in exports, the U.S. is the leading country for digitally-delivered services, at 15.3% of the world total in 2023.

Roughly two-thirds of service exports from America are conducted digitally. Financial services stands as the leading sector, while cloud services is among the fastest-growing overall. Moreover, advancements in AI are projected to boost digital exports looking ahead amid strong demand, cost savings, and increased innovation potential.

Ranking in second is the UK, where more than 80% of services exports are delivered through digital channels.

Following next in line is Ireland, one of the top exporting countries of computer services. Thanks to the country’s favorable tax policies, many big tech firms such as Google, Facebook, and Apple have their European headquarters in the country.

To learn more about this topic from a sector-based perspective, check out this graphic that breaks down different types of global digital service exports.

https://www.zerohedge.com/technology/these-are-20-largest-digital-exporters-world