FDA reveals real-world pilot for digital health tools

 The FDA is rolling out a pilot programme that could make it easier to bring digital health technologies (DHTs) for chronic diseases to market more quickly and easily.

In line with its deregulatory stance under new Commissioner Marty Makary, the US regulator has launched TEMPO – Technology-Enabled Meaningful Patient Outcomes – that will take the form of a voluntary pilot scheme "designed to promote access to certain digital health devices while safeguarding patient safety."

The TEMPO pilot will run within the Centers for Medicare and Medicaid Services (CMS) recently launched ACCESS model, which is testing an outcome-aligned payment approach for Medicare aimed at improving access to approved healthtech that is used for people with conditions like high blood pressure, diabetes and prediabetes, chronic musculoskeletal pain, and depression.

The FDA plans to select up to 10 device manufacturers in each of four clinical areas – which have not already gone through the usual premarket authorisation process – in the initial phase of the TEMPO project, and sponsors will be able to submit statements of interest starting in January 2026.

The four areas are early cardio-kidney-metabolic – which includes hypertension, dyslipidaemia, obesity/overweight, and prediabetes – along with later-stage cardio-kidney-metabolic (diabetes, chronic kidney disease, or atherosclerotic cardiovascular disease), chronic musculoskeletal pain, and behavioural health interventions of depression or anxiety.

"We are piloting an approach to encourage the use of digital technologies that meet people where they are," said Makary in a statement. "This pilot supports innovative tools and a healthcare delivery model that could improve care for millions of Americans managing chronic disease."

According to the regulator, participating manufacturers can request enforcement discretion for certain requirements, such as premarket authorisation and investigational device rules, as long as they target chronic conditions.

The FDA and CMS will work together on the TEMPO pilot, in which participating manufacturers will offer their DHTs for use while "collecting, monitoring, and reporting real-world performance data." The FDA will work with participants to identify the circumstances when enforcement discretion may be appropriate for a particular DHT.

The pilot will "allow us to responsibly encourage innovation while collecting real-world evidence that may help us better understand how these devices perform for patients in their everyday lives," commented Michelle Tarver, director of the FDA's Center for Devices and Radiological Health (CDRH).

Makary has said he aims to challenge with way medical products are regulated in the US while in office, taking the brakes off a system that he claims is holding the country back in an increasingly competitive international landscape. Other initiatives include the Commissioner's National Priority Voucher programme, which could see some medicines approved in weeks, rather than months, and a recently discussed move towards requiring only one pivotal clinical trial – rather than a pair – as the standard for showing safety and efficacy.

https://pharmaphorum.com/news/fda-reveals-real-world-pilot-digital-health-tools

ASH: Gilead preps filing for anito-cel on iMMagine-1 data

 Gilead Sciences' Kite unit has showcased three new CAR-T therapies at ASH, headed by multiple myeloma candidate anitocabtagene autoleucel (anito-cel) which is being prepared for a filing and potential launch in 2026.

The BCMA-targeted CAR-T could be a competitor to Johnson & Johnson and Legend Biotech's already approved Carvykti (ciltacabtagene autoleucel) and Bristol-Myers Squibb's Abecma (idecabtagene vicleucel) if approved for marketing.

At ASH, Kite presented the results of the iMMagine-1 trial of anito-cel, which met expectations of strong efficacy with an overall response rate (ORR) of 96%, with 74% of patients achieving a stringent complete response (sCR) or complete response (CR), and 95% of patients testing negative for minimal residual disease (MRD). The CAR-T was being tested in patients who had received at least three prior lines of therapy.

Close attention was paid to the safety data from the study, and in particular rates of neurotoxicities, which have been held up as one way for anito-cel to differentiate itself from its rivals, which are respectively approved as second- and third-line options for multiple myeloma.

At the moment, Carvykti is growing faster than Abecma on what is viewed as superior efficacy data, including a higher complete response rate, with quarterly sales currently running above $500 million.

iMMagine-1 showed a rate of neurotoxicity of 8%, and no cases of delayed neurotoxicities like Parkinsonism and neuropathies or immune effector cell-associated enterocolitis (IEC-EC), which are both recognised side effects with J&J and Legend's CAR-T and can happen weeks or months after treatment.

"These data are compelling and are an important advancement for patients living with multiple myeloma," said lead investigator Krina Patel of MD Anderson Cancer Center.

"We rely on therapies that deliver continued meaningful efficacy, a predictable safety profile, and reliable manufacturing," she added." Anito-cel demonstrates that it could become a significant new treatment option in our efforts to improve outcomes for patients."

Anito-cel uses technology developed by Kite's partner Arcellx that is designed to enable high CAR expression, with quick release from the BCMA target, to minimise toxicity. Kite's hope is that its profile will make it suitable for use in outpatient or even community settings.

Bicistronic CAR-Ts

ASH also gave Kite the opportunity to reveal new data on a pair of its so-called 'bicistronic' CAR-Ts, which are designed to target two antigens at the same time. That is a new area of CAR-T therapy that was also explored at ASH in the DURGA-1 study of AstraZeneca's multiple myeloma candidate AZD0120.

Kite reported the results of two CD19 and CD20-directed CAR-Ts – codenamed KITE-753 and KITE-363 – that also include co-stimulatory domains designed to boost their efficacy, reduce cancer cell escape from treatment, and prevent relapse. They can also be produced more quickly than conventional CAR-Ts.

The two studies, both in patients with relapsed/refractory large B-cell lymphoma (LBCL), showed high rates of complete response with KITE-753 (79%) and KITE-363 (70%-plus), with an encouraging safety profile and no dose-limiting toxicities.

https://pharmaphorum.com/news/ash-gilead-preps-filing-anito-cel-immagine-1-data

Dyne’s Exon Skipper Delivers ‘Best Ever’ Functional Outcomes in Early DMD Study

 

Dyne’s exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at six months and elicited functional improvements that are the “best ever” for this treatment class, Stifel analysts said.

Dyne Therapeutics’ investigational therapy zeleciment rostudirsen not only increased dystrophin levels in an early Duchenne muscular dystrophy study but also elicited what Stifel analysts called the “best ever” functional improvements for an exon skipper in this indication.

“The regulatory precedent is overwhelmingly in DYN’s favor,” Stifel added, noting that other Duchenne therapies have been approved based on “marginal” dystrophin effects, even with “limited evidence of clinical efficacy.”

While Dyne’s study was not powered to evaluate functional benefits on a statistical level, the analysts confirmed with the company that the analyses for these outcomes “match the rigor/standard of a registrational study.”

In the registrational expansion cohort of Dyne’s Phase I/II DELIVER study, presented Monday, patients given a monthly dose of 20-mg/kg zeleciment rostudirsen (z-rostudirsen) achieved mean dystrophin expression of 5.46% of normal, adjusted for the patients’ muscle content. This finding represents an approximately sevenfold increase in dystrophin at six months, Stifel said, giving z-rostudirsen a “highly differentiated” efficacy profile.

The analysts pointed out that Dyne’s dystrophin figures “far exceed” those of Sarepta Therapeutics’ exon skipper Exondys 51, which they said hit 0.3% of normal at six months. No head-to-head study between Exondys and z-rostudirsen has been conducted, however, and these cross-study comparisons may not accurately capture the relative efficacies of these therapies, Stifel noted.

Beyond dystrophin, patients on z-rostudirsen also saw notable functional improvements at six months—though DELIVER is not powered for statistical assessments of functional benefits versus placebo. A posthoc analysis nevertheless showed that patients on Dyne’s drug had better time-to-rise and 10-meter walk/run outcomes than placebo counterparts, with nominally significant effects. Z-rostudirsen also boosted participants’ stride velocity and upper limb performance, while also improving their lung function.

With these data, Dyne is planning to file a biologics license application for the drug in the second half of 2026, seeking accelerated approval. If all goes well, and if the FDA grants priority review, the biotech is eyeing an early 2027 launch. Also in the back half of next year, Dyne will launch a Phase III program for z-rostudirsen to validate clinical benefit and support global regulatory filings.

https://www.biospace.com/drug-development/dynes-exon-skipper-delivers-best-ever-functional-outcomes-in-early-dmd-study

Berkshire stock picker departs to lead new strategic investment group at JPMorgan

 Todd Combs, one of Warren Buffett's investing lieutenants and the CEO of GEICO, is departing Berkshire Hathaway (BRK-B, BRK-A) and joining JPMorgan Chase (JPM) in a new role as part of a major shake-up at both firms.

Combs is leaving Berkshire Hathaway and his role leading GEICO to run the bank's new investment group as part of its wider "security and resilience" initiative announced in October.

https://finance.yahoo.com/news/berkshire-hathaway-stock-picker-todd-combs-departs-to-lead-new-strategic-investment-group-at-jpmorgan-154015380.html

House Approves Bill Blocking IRS Fines Without Specific Supervisory Approval

 by Naveen Athrappully via The Epoch Times (emphasis ours),

The House of Representatives voted to pass two bills on Monday that increase protections for taxpayers in matters related to penalties imposed by the IRS and tax dispute proceedings in the U.S. Tax Court.

The first bill, HR 5346, the Fair and Accountable IRS Reviews Act, relates to supervisory approval for certain federal penalties imposed by IRS agents on taxpayers.

An IRS employee can impose certain federal penalties on taxpayers after gaining written authorization from their immediate supervisor.

Under present regulations, an immediate supervisor is broadly defined as an individual tasked with reviewing another employee’s proposed penalties, and not necessarily the supervisor to whom the IRS employee reports, according to a fact sheet from the House Committee on Ways and Means.

IRS agents can thus “shop around for sympathetic supervisors,” it said, which weakens taxpayer protections.

“This circular definition is so broad that IRS agents can obtain approval to apply tax penalties on taxpayers from virtually any other employee,” the fact sheet states.

The bill seeks to resolve the issue by defining an immediate supervisor “to be the person to whom the individual making the determination reports,” the fact sheet said.

In addition, the bill clarifies that supervisory approval of a penalty will only be considered timely if such approval is obtained in writing before a taxpayer is notified of such a penalty.

In a Dec. 3 statement, the House Ways and Means Committee said the bill will ensure that “rogue IRS agents are not levying fines and penalties on taxpayers without specific supervisory approval.”

The second bill, HR 5349, the Tax Court Improvement Act, seeks to expand the authority of the U.S. Tax Court in issuing subpoenas. The court will be authorized to extend certain petition deadlines and also institute other changes to court procedures.

The U.S. Tax Court is a federal trial court specializing in adjudicating disputes related to federal income tax, a process that typically takes place before formal tax assessments are conducted by the IRS, according to a fact sheet from the House Committee on Ways and Means.

The Tax Court is the only forum in the United States where taxpayers can litigate issues without having to first pay in full the taxes that are being disputed.

The bill aims to solve inefficiencies in the Tax Court structure, it said.

For instance, the court currently has limited pre-trial discovery powers, which cause “unnecessary delays in resolutions of cases” since there are limited options for parties in a trial to obtain documents relevant to a case in a timely manner, the fact sheet said.

The Tax Court Improvement Act solves this issue by expanding the court’s powers. It authorizes the court to sign subpoenas to produce relevant documents in the discovery phase of the case, ensuring that parties receive these documents prior to a hearing.

Since the bill allows the court to extend petition filing deadlines, it also resolves difficulties faced by taxpayers when timely filing is impractical and impossible, according to the fact sheet.

In addition, the Act will hold Tax Court judges to the same standards for disqualification that apply to other federal judges.

Other provisions of the bill include expanding the type of proceedings for which special trial judges may be appointed, authorizing special trial judges to impose a jail term of up to 30 days and a maximum fine of $5,000 for contempt of court, and requiring judges to recuse themselves in certain circumstances.

The bills have the backing of multiple groups, such as the National Taxpayers Union and the Taxpayers Protection Alliance. Both bills have been sent to the Senate.

Projected Revenues

The Congressional Budget Office (CBO) projects that the enactment of both bills will generate revenues over the coming decade.

In a Nov. 19 statement, the CBO said an analysis by the office and the Joint Committee on Taxation (JCT) estimates that enacting the Tax Court Improvement Act would increase revenues by a net $6 million between 2026 and 2035, with administrative costs rising minimally by less than $500,000 between 2026 and 2030.

CBO said in another Nov. 19 statement that the Fair and Accountable IRS Reviews Act is projected to boost revenues by $117 million between 2026 and 2035, with administration costs increasing by less than half a million dollars for the 2026–2030 period.

The passage of both bills comes as the IRS recently urged Americans to start preparing for filing taxes in the 2026 filing season, citing changes in tax laws and other factors.

The One Big Beautiful Bill Act, signed into law by President Donald Trump in July, will significantly affect federal taxes, deductions, and credits, according to agency officials.

“A little advance work preparing paperwork and organizing information now can help with filing tax returns quickly and accurately,” the agency said.

https://www.zerohedge.com/political/house-approves-bill-blocking-irs-fines-without-specific-supervisory-approval

'Disappointed' Trump Raps Zelensky Who 'Hasn't Yet Read' Peace Proposal

 President Trump has continued to speak frankly about Zelensky and the Ukrainian government, at a moment of a sense of stalled frustration on the peace plan with Russia unveiled last month. His frustration with Keiv continues to show, especially after high hopes for his 28-point peace plan were dashed by yet more roadblocks which saw his Thanksgiving deadline quickly come and go.

While speaking on the red carpet at the Kennedy Center Honors on Sunday night, Trump bluntly stated he was "a bit disappointed" that Ukrainian President Volodymyr Zelensky had not yet reviewed Washington's draft of the latest proposed peace plan to end the war.

"We've been speaking to President [Vladimir] Putin, and we've been speaking to Ukrainian leaders, including President Zelenskyy, and I have to say that I'm a little bit disappointed that President Zelensky hasn't yet read the proposal – that was as of a few hours ago," Trump stated.

He then said "His people love it, but he hasn’t. Russia’s fine with it." The somewhat negative assessment of the Ukrainian leader comes after Steve Witkoff and Jared Kushner on Friday again met with Rustem Umerov, secretary of the National Security and Defense Council of Ukraine, and Ukrainian negotiator Andriy Hnatov in Florida.

Trump at the same event went on to describe the war as "very tough, very nasty" and with mounting heavy casualties which have not abated.

As for Zelensky, he has freshly written on social media in response that "Ukraine is determined to keep working in good faith with the American side to genuinely achieve peace."

"We covered many aspects and went through key points that could ensure an end to the bloodshed and eliminate the threat of a new Russian full scale invasion, as well as the risk of Russia failing to honor its promises, as has happened repeatedly in the past," he continued.

Interestingly, while Trump issued critical remarks of Zelensky's stance, the Kremlin seemed to positively welcome the White House's new National Security Strategy memo, issued Friday:

Trump's criticism of Zelensky came as Russia on Sunday welcomed the Trump administration’s new national security strategy in comments by the Kremlin spokesman published by Russia’s Tass news agency.

Dmitry Peskov said the updated strategic document, which spells out the administration's core foreign policy interests, was largely in line with Moscow’s vision.

“There are statements there against confrontation and in favor of dialogue and building good relations,” he said, adding that Russia hopes this would lead to “further constructive cooperation with Washington on the Ukrainian settlement”.

Likely Moscow especially likes that the document characterizes Europe as weak while warning of an unpredictable, disunified atmosphere on the continent where in desperation European leadership could overreact and escalate war with Russia. For example FT has written of the document:

Donald Trump’s first NSS since returning to office blames European officials for thwarting US efforts to end the war in Ukraine and accuses governments of ignoring a "large European majority" who want peace.

Meanwhile, Trump's outgoing Ukraine envoy, Keith Kellogg, while addressing the Reagan National Defense Forum on Saturday claimed that the peace process was "in the last 10 meters." But it's a far different question and highly dubious whether the two warring sides actually see real lasting peace as so much as on the horizon at this point.

On Monday leaders from the United Kingdom, France, and Germany meeting with Zelensky in London. They are still trying to forge a more 'just' and 'fair' alternative or counter peace plan.

"We stand with Ukraine," UK Prime Minister Kier Starmer said while setting out his "principles".

He described, "If there is to be a ceasefire then it needs to be a just and lasting ceasefire; that is why it is so important we repeatedly set out the principle that matters about Ukraine are for Ukraine." He added at the start of the meeting: "We stand here to support you in the conflict and support you in the negotiations." The Europeans have by and large rejected and balked at the US proposal to give up the Donbas and Crimea.

https://www.zerohedge.com/political/disappointed-trump-raps-zelensky-who-hasnt-yet-read-peace-proposal

BiomX (NYSE American: PHGE) halts BX004 CF trial, to cut costs, weigh options

BiomX (NYSE American: PHGE) announced on December 8, 2025 that it will discontinue the Phase 2b trial of nebulized phage therapy BX004 in cystic fibrosis after an internal review and recommendations from the independent Data Monitoring Committee following unusually high rates of adverse events.

The company said it will implement cost‑cutting measures, including a significant workforce reduction, and review strategic alternatives while refocusing development efforts on its bacteriophage program BX011 for Staphylococcus aureus diabetic foot infections, subject to available resources.

https://www.stocktitan.net/news/PHGE/biom-x-announces-discontinuation-of-phase-2b-bx004-trial-following-gwoff9327dy3.html