Roche seeks label expansion for Elevidys amid missing Phase III endpoint

 Roche and Sarepta Therapeutics’ have announced top-line results from the Phase III trial for Elevidys (delandistrogene moxeparvovec) in patients with Duchenne muscular dystrophy (DMD) aged 4-7 years.

The study failed to meet its primary endpoint of improvement in functional motor abilities, measured using the North Star Ambulatory Assessment (NSAA).

The companies were quick to note that although the drug failed to meet its primary endpoint, it met all the secondary endpoints, as per the October 30 press release.

Septra added that “the US Food and Drug Administration (FDA) has indicated openness to reviewing the data for label expansion based on the totality of evidence from Embark”.

DMD is a rare, early-onset genetic condition that causes gradual muscle damage and weakness due to the loss of the dystrophin protein.

Elevidys is a gene therapy that targets the DMD gene mutation (responsible for causing DMD). It received accelerated approval from the FDA for treating DMD patients aged 4-5 years and set a hefty wholesale acquisition price tag of $3.2m. GlobalData forecasts Elevidys to generate $5.6bn in global sales in 2029.

GlobalData is the parent company of Clinical Trials Arena.

Elevidys full approval is contingent on this Phase III (NCT05096221) trial data. Other functional secondary endpoints which were met include the time to rise from the floor and the 10m walk test.

Roche acquired the global (except the US) marketing rights for Elevidys in 2019. The Swiss company paid $1.15bn upfront, an additional $1.7bn payable based on regulatory and sales milestones, and royalties on net sales. Sarepta holds US marketing rights for the gene therapy.

Sarepta is also developing an RNA-targeted peptide-conjugated phosphorodiamidate morpholino oligomer (PMO), vesleteplirsen for treating DMD.

Roche invested in its neurological drug pipeline and signed a partnership and licensing agreement with Monte Rosa Therapeutics to discover and develop molecular glue degraders (MGDs) to target cancer and neurological diseases.

https://www.clinicaltrialsarena.com/news/roche-seeks-label-expansion-for-elevidys-amid-missing-phase-iii-endpoint/

Banned in Britain: Temu ads 'sexualize kids, objectify women'

 The online retailer Temu has had an advert banned for sexualising a child.

It showed a girl aged between eight and 11 wearing a bikini in a pose that was "was quite adult for a girl of her age", the Advertising Standards Authority (ASA) said.

It banned four other adverts for showing sexual images and pictures which objectified women.

Temu said the picture of the child violated their marketing policy and would not be shown again.

It disputed the other complaints but all were ruled to be inappropriate by the ASA.

Temu is a Chinese-owned online marketplace where sellers can upload pictures of their products.

The sellers have to follow rules against using pornographic, obscene or harassing pictures.

Since its UK launch earlier in 2023, Temu has become known for its wide range of products which are advertised at cheap prices.

It told the ASA that it has over a million photos uploaded to its Google Ads account but that it "did not get to decide the specific products advertised," or where they were shown.

The images which received complaints included those of women's torsos in low-cut dresses, a grey jockstrap and padded cycling underwear.

The ASA ruled that the jockstrap emphasised "the outline of the genitalia" while the cycling shorts "appeared as underwear with the bottom cut out".

The pictures of the dresses, which did not show the models' faces, meant "the women were presented as stereotypical sexual objects".

Furthermore, pictures of a facial roller and balloon ties "were phallic in shape and appeared sexual in nature" when shown alongside the other ads.

The ads were shown on a local news website, a chess website, a translation site and a puzzle app.

Temu said that the pictures of the models with their faces cut off were not meant to objectify women and argued that other retail platforms showed similar photos.

It also argued that the pictures of the other objects were accurate representations of the products on sale.

The chess website and the news website said that they would not advertise Temu products again.

The ASA said it had told Temu to make sure its future adverts were "prepared with a sense of responsibility to consumers and to society".

It has also told the company that under-18s should not be shown in a sexual way and that ads should be responsibly targeted.

https://www.bbc.co.uk/news/technology-67272541

PFAS ‘forever chemicals’ in drinking water can be removed — here’s how

 There’s something in the water — and you should get it out.

Scientists have sounded the alarm about the group of toxic chemicals commonly known as PFAS, or per-and polyfluoroalkyl substances.

PFAS are often called “forever chemicals” because they persist in water, plants, soil and elsewhere in the environment — including animal and human bodies.

The chemicals have been used for generations in a wide range of consumer and industrial materials to make them water-resistant, non-stick and fire-retardant, according to the Environmental Protection Agency.

PFAS also dissolve easily in water, and a 2023 report from the US Geological Survey revealed that PFAS have been found in 45% of drinking water in the US, including private wells and public water supplies.

The chemicals are implicated in a range of serious health concerns, from cancer, thyroid disease, liver damage and fertility problems to increased cholesterol levels, obesity, and hormonal disorders.

In March, the EPA proposed a restriction on PFAS in drinking water, which the agency believes could “prevent thousands of deaths and reduce tens of thousands of serious PFAS-attributable illnesses.”

Do water filters remove forever chemicals?

Until such a proposal becomes law, here are steps you can take to protect yourself and your family from PFAS in your drinking water.

A comprehensive new report by FoodPrint outlines inexpensive water filters that remove significant amounts of PFAS, including filters with activated carbon adsorption, ion exchange resins and high-pressure membranes.

These technologies can be whole-house filters installed at the point-of-entry, where water enters the house, or the point-of-use, such as in a kitchen sink or a shower. Point-of-use treatment tends to be less expensive than point-of-entry.

“To remove a specific contaminant like PFAS from drinking water, consumers should choose a water filtration device that is independently certified to remove that contaminant by a recognized lab,” said Jim Nanni of Consumer Reports.

Does boiling water remove forever chemicals?

Experts warn consumers that boiling water doesn’t remove PFAS. And while many homes have whole-house water-softening or iron-filtration systems, those systems don’t remove PFAS, either.

For a filter that removes PFAS, look for one with the code NSF/ANSI 53 (or NSF/ANSI 58 for reverse osmosis systems), followed by the manufacturer’s claim that the product can remove PFAS.

Remember that all filtration systems are effective only as long as they’re maintained, so follow the manufacturer’s recommendations for cleaning or replacing filters and other components.

For a list of recommended water filters that can remove PFAS, refer to Consumer Reports, this Environmental Science and Technology Letters article or the Environmental Working Group website for reviews and test results.

https://nypost.com/2023/10/31/lifestyle/pfas-forever-chemicals-in-drinking-water-can-be-removed-heres-how/

Hamas traps 1,000 Americans in Gaza as last US charter plane leaves Israel

Roughly 1,000 Americans are stuck in Gaza without a way out as Hamas terrorists refuse to open the region’s main exit route into Egypt, Secretary of State Antony Blinken told the Senate Appropriations Committee Tuesday.

The figure was revealed by America’s top diplomat the same day the State Department operated its final evacuation charter flight out of Israel.

Hamas has refused to let anyone seeking to leave its territory through the Rafah crossing, where Israeli officials had told Gazans to flee to avoid its burgeoning ground offensive.

Among those caught in the middle are about 400 Americans and their families — about 1,000 people in all, Blinken said, as well as an estimated 5,000 citizens from other countries.

“We’re working with various parties to try to facilitate their departure from Gaza,” he said. “The impediment is simple: it’s Hamas.”

Blinken added that his department was “focused on this intensively” and working with regional partners to hash out a way to get the US citizens out of Gaza.

“We’ve not yet found a way to get them out through whatever place and by whatever means that Hamas is not blocking, but we’re working that with intermediaries,” he said.

The department has been “in close communication as best we can with Americans who are stuck in Gaza,” but so far, it has been unable to secure their exits, Blinken went on.

“We’ve had about 5,500 communications that we’ve initiated – phone calls, emails, WhatsApp – to be in touch with them to try to guide them as best we can, and to work for their ability to leave,” he said.

But Sen. Jeff Merkley (D-Ore.) told the secretary of the plight of one Oregon family he is in touch with who “relayed to me how they three times have gotten a text message [or] email saying, ‘Drop everything [and] go to the Rafah crossing,’ and they waited until dark. But each time, nothing happened.”

“They weren’t interfered with by Hamas, just the gate never opened,” Merkley said. “With the trucks coming in [with humanitarian aid from Egypt], why can’t we at the same time … get those American families … out that gate?”

Blinken acknowledged that the department has “had occasions where we thought that we would be able to move forward … only to find that the necessary procedures that had to be put in place to actually make that work couldn’t go forward,” insisting that “Hamas has been the impediment to that.”

“We’re working on it with with Egypt. We’re working on it with Israel. We’re looking for ways to to get people out,” the secretary said. “But because Hamas controls what goes on for the most part inside of Gaza, unless it agrees, then it’s going to be very difficult to get that done.”

Other Americans are still trapped in Israel without flights home after major airlines canceled operations in the Jewish state following Hamas’ Oct. 7 terror spree that killed more than 1,400 people — including at least 33 US citizens.

After about a week of calls for the Biden administration to help evacuate Americans from Israel, the State Department announced it would begin sending charter flights to pick up US nationals.

However, Tuesday saw the last scheduled charter flight after State Department spokesman Matthew Miller said just five passengers had boarded one full-sized jetliner earlier this week.

Still, Miller left open the possibility that the department could restart the flights should demand increase.

https://nypost.com/2023/10/31/news/hamas-traps-1000-americans-in-gaza-as-last-us-charter-plane-leaves-israel/

How to Defeat Hamas Fast, Cheap and Easy

 As the war rages on, Gaza is getting destroyed, people are getting killed on both sides, and a resolution of the conflict seems as elusive as ever. On the contrary, the war is escalating and spreading to other theaters.

Israel is facing two challenging issues: the safe return of the hostages and the eradication of Hamas. Thus far, those issues seemed to be at odds with each other.

According to the reports, the IDF is evaluating two distinct options. The first option involved a full-scale assault on Gaza by infantry and Special Forces supported by tanks, heavy artillery, and aerial bombardment aimed at destroying Hamas positions, the infrastructure, and a wide network of tunnels with deep-penetrating bombs.

The problem with this approach is that the tunnels in Gaza are fairly sophisticated. They boast an impressive level of engineering, constructed from reinforced concrete, divided into separate self-contained sections by steel doors, and reaching depths up to 100 feet. The tunnels are also equipped with modern ventilation systems powered by generators. They were built under buildings, hospitals, schools, and other civilian projects.

To complicate the matter, many sections following intense bombardments now lie buried under the rubble of destroyed buildings. The rubble provides an additional layer of protection against concrete-buster ammunition. Even if the IDF successfully destroys the tunnels, and it is a big if, there is a fairly high possibility that the hostages will be lost.

The second option is to enforce the blockade and continue bombing while the Biden Administration negotiates with Hamas, using Qatar, a strong Hamas supporter, as a mediator. Under this scenario, the operation cannot begin until the hostages are released. It will be a long and tedious process. Hamas regards hostages as a commodity and a human shield and will not release them until its demands are met.

The terrorists currently hold more than 200 hostages. If they continue releasing two hostages per week, as we witnessed, and doing so keeps the invasion force at bay, it will take approximately two years to secure the release of all hostages. If history is a guide, an agreement negotiated by the US will be the product of Israel making concessions to please the Arabs.

Another point of concern is that Jews have, throughout history, found themselves marginalized and never accepted as an integral part of the global human community. Hamas understands that, in the long run, the world community will not support Israel. We are already witnessing mass demonstrations worldwide supporting Palestinians. Inevitably, international pressure will pave the way for establishing humanitarian corridors to facilitate aid delivery, evacuate sick and wounded, and provide electricity, water, and other essentials.

At the same time, Israel will face mounting challenges as it endeavors to sustain massive troops and materiel along the Gaza border. The prolonged commitment will weaken the economy, damage Israeli morale, erode confidence in the government, and end with a loss of strategic momentum. Indeed, time is on Hamas’s side.

So, what is to be done?

To eradicate Hamas and ensure safe passage for the hostages, the IDF should flood the tunnels with seawater.

If the complete plans of the tunnels with the location of the entrances are not available to IDF, it should not be too difficult to obtain them. This type of tunnel could only be built using professional engineering, procurement, and construction firms. The engineering firms engineered and designed the tunnels, construction firms built them, and various vendors supplied and installed equipment. All those firms have drawings of the tunnels and, in that part of the world, everything is for sale.

The flooding, if done correctly, will deprive Hamas of a safe harbor in the tunnels and force them out with the hostages where the IDF will be waiting. Even if, by some miracle, Hamas manages to avoid the IDF and leave tunnels undetected, its fighters will still lose the ammunition, rockets, and other military hardware.

At the outset of the operation, it is imperative to convey a clear message to Hamas that only those who get out with the hostages will be spared; the others will be executed on the spot. This time, Israel will manage to separate the so-called civilians from their murderous sons.

Hamas fighters do not wear a uniform or distinguish themselves from the civilian population in some other way, so they are not lawful combatants and are not protected by the Geneva Convention. Israelis should unequivocally convey their resolve by executing terrorists. Terror can be stopped only with greater terror.

The practice of flooding underground installations isn’t a novel concept, and it has historical precedents and a track record of effectiveness. During WWII, Germans used it in Odessa to flush out Soviet partisans concealed in the catacombs. Although the detailed descriptions and myriad techniques are beyond the scope of this article, the readers can find them in the Red Army military manual from 1939, which offers comprehensive insights into the subject.

For Israel, time is of the essence.

Alexander G. Markovsky is a senior fellow at the London Center for Policy Research, a think tank that examines national security, energy, risk analysis, and other public policy issues. He is the author of Anatomy of a Bolshevik and Liberal Bolshevism: America Did Not Defeat Communism, She Adopted It. Mr. Markovsky is the owner and CEO of Litwin Management Services, LLC. He can be reached at alexander.g.markovsky@gmai.com

https://www.americanthinker.com/articles/2023/10/how_to_defeat_hamas_fast_cheap_and_easy.html

FDA approves Amgen's biosimilar version of J&J's psoriasis drug

 The U.S. Food and Drug Administration on Tuesday approved Amgen's biosimilar version of Johnson & Johnson's blockbuster psoriasis treatment, Stelara, for multiple inflammatory diseases.

Despite the FDA approval, Amgen's treatment is expected to be launched in 2025 as part of a

legal settlement

between the two companies earlier this year to delay the entry of the therapy.

Biosimilars are close copies of complex biological drugs.

Stelara, introduced in 2009, has been J&J's top-selling drug since 2019, with sales reaching $9.7 billion in 2022.

https://finance.yahoo.com/news/rpt-1-u-fda-approves-225834589.html

Why Beam and Verve Therapeutics Shares Soared While Eli Lilly Soured

 A multimillion-dollar deal in the healthcare industry announced on Tuesday led to share price pops for two of the three parties involved. Shares of Beam Therapeutics (BEAM 10.62%) and Verve Therapeutics (VERV 27.95%) shot up 11% and 28%, respectively, on the day.

On the downside, their counterparty in the deal, Eli Lilly (LLY -2.08%), saw its share price slump by 2%.

Verve sold its gene therapy programs to Eli Lilly

This morning, Beam and Verve announced in separate press releases that the former had sold to Eli Lilly the product rights to three of Verve's cardiovascular gene-editing programs.

The deal is potentially worth up to $600 million; one-third of this takes the form of an up-front payment, while Lilly is also ponying up a $50 million equity investment in Beam.

Beam also stands to earn as much as $350 million if and when it hits certain clinical, regulatory, and other milestones.

In Beam's press release, CEO John Evans said that the deal "provides meaningful up-front capital to advance our portfolio of clinical- and research-stage programs, with significant additional value achievable as the Verve programs advance through development."

Although a total initial payout of $250 million isn't cheap, even for a major healthcare business with significant resources like Eli Lilly, it clearly believes it's money very well spent.

In Beam's press release, Eli Lilly's group president for diabetes, obesity, and cardiometabolic research, Ruth Gimeno, was quoted as saying that the new arrangement "expands the scope of Lilly's ongoing relationship with Verve and gives us exposure to the full breadth of potential with Beam's base editing platform."

She added that the single-course gene-editing solutions involved in the deal could end up being compelling new treatment options for people facing the risk of cardiovascular disease.

A cash infusion when it's needed most

Eli Lilly was apparently quite determined to make this deal. Industry website Fierce Biotech reported Evans' claim that the equity component of the agreement was the pharmaceutical giant's idea. He intimated that Lilly's ownership stake could grow the relationship between the two companies in the future.

Beam can definitely put the funds from the deal to good use. Earlier in October, the company announced that it would cut its employee head count by roughly 20%. The clinical-stage biotech continues to lose money, and as of the end of its second quarter, it carried a debt load of over $177 million.

https://www.fool.com/investing/2023/10/31/why-beam-and-verve-therapeutics-stock-soared-while/