Overall response rate of 90% in cohort with durable responses observed; one patient remains in complete remission at 31 months
All patients were heavily pretreated/refractory to BTK inhibitors, and only one patient has started new anti-WM treatment after MB-106
Outpatient administration was allowed and found to be feasible
Currently no FDA-approved CAR-T treatments for WM
Data presented at the European Hematology Association 2024 Hybrid Congress
Fans heading to Yankee Stadium hoping to pay in cash at the iconic ballpark for their favorite concessions have been thrown a curveball: go cashless or pay extra.
As the Wall Street Journal reports, Noa Khamallah, a 41-year-old New Yorker, found out the hard way. Looking to enjoy some popcorn and soda at a game, Khamallah was shocked to discover that his cash was as good as obsolete. Instead, he was directed to a "reverse ATM," where he inserted $200 only to receive a debit card with $196.50 - after he was hit with a $3.50 service fee for the 'convenience' of going cash-free.
"It's just not right," Khamallah told the outlet, echoing the sentiments of other New Yorkers shocked that what used to save you money - cash payments, now costs more. In some cases, transaction fees have soared more than $6 just for the privilege of spending your own funds.
Indeed, cashless venues and restaurants are popping up across the country, forcing cash lovers to either adapt or pay up as the war on cash continues.
Reverse ATMs like those at Yankee Stadium are now common at cashless venues and restaurants across the country as a way to cater to those who prefer paying in cash. People who want to pay their parking tickets, tolls, taxes or phone bills in cash, meanwhile, often learn that government agencies and businesses have outsourced that option to companies that usually charge a fee.
All that can amount to a penalty on the people who prefer paying cash. Though it is more common to buy things with cards and mobile devices, cash remains the third-most popular way to pay, accounting for 16% of all payments in 2023, according to the Federal Reserve. That’s down 2 percentage points from the year before, continuing a steady decline that accelerated during the pandemic. -WSJ
And it’s not just about convenience or the speed of transactions. Critics argue that the move sidelines those who rely on cash - often the young, the elderly, or the poor.
"To let my 13-year-old go buy a slushy at the amusement park, I’m already out $6," said Prudence Weaver, 41, who said she would rather be able to use cash on trips to the zoo and other venues vs. paying fees for debit cards. "I understand that there is a place for electronic payment, but I don’t think it should be the only option."
Despite the digital dominance, cash is still king for a significant chunk of Americans. According to the Federal Reserve, a full 16% of all payments in 2023 were made in cash, down 2% from 2022.
"It’s unbelievable that we actually have to tell retailers, ‘This is U.S. currency and it’s something that should be accepted,’" said Jonathan Alexander, executive director of the Consumer Choice in Payment Coalition, a group of businesses and nonprofits lobbying for the continued acceptance of cash.
The backlash has spurred some action. States like Colorado and Rhode Island have pushed back, banning cashless retail establishments. On Capitol Hill, lawmakers are batting around bills that would make it mandatory for businesses to accept cash for purchases under $500.
But the future of these efforts remains uncertain. In the meantime, companies like RedyRef are cashing in, literally, doubling their shipments of reverse ATMs to keep up with demand from businesses ditching traditional cash transactions.
"It has been a pretty wild shift," said Will Pymm, senior vice president. "Probably one of the biggest we’ve seen for a specific product, in such a short amount of time."
Even as venues pocket fees from these new systems, the debate rages on whether this shift truly serves the public or merely lines the pockets of corporate giants. At stadiums, amusement parks, and beyond, the cost of going cashless continues to climb - and it's everyday consumers who are footing the bill.
So next time you head out to a ball game, remember: bring your plastic, or be prepared to pay up.
– The Independent Data Monitoring Committee (IDMC) Recommends Continuation of Phase 3 REGAL Trial Without Any Modifications –
– No Safety or Futility Concerns Were Raised Based on theEfficacy and Safety Assessment of All REGAL Patients –
– Interim Analysis Anticipated by Q4 2024 –
With six target action dates lined up, the FDA has a very busy week ahead. The regulator is expected to release decisions on drug applications for a pneumococcal vaccine, a gene therapy for Duchenne muscular dystrophy and a label expansion for one of cancer’s cornerstone therapies.
Read below for more.
Merck is seeking approval for its investigational 21-valent vaccine, dubbed V116, to prevent invasive pneumococcal disease and pneumococcal pneumonia in adults. The FDA is expected to release its verdict on June 17.
The pharma is backing its regulatory bid with data from the Phase III STRIDE-3 trial, which in November 2023 showed that V116 induced non-inferior immune responses as compared with a 20-valent comparator vaccine. Merck is also supporting V116’s application with several other studies, including STRIDE-3, STRIDE-4, STRIDE-5 and STRIDE-6. Data from additional studies will be shared at future medical meetings.
If approved, Merck’s vaccine would become the first pneumococcal conjugate shot designed specifically for use in adults.
Argenx is proposing to use its antibody fragment Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) to treat the rare autoimmune disease chronic inflammatory demyelinating polyneuropathy (CIDP). The FDA’s decision is due on June 21.
The Netherlands-based biotech is supporting its supplemental Biologics License Application (sBLA) with data from the Phase II ADHERE study, which demonstrated a 61% reduction in the risk of relapse after treatment with Vyvgart Hytrulo versus placebo. In ADHERE’s open-label phase, 67% of patients showed evidence of clinical improvement.
Vyvgart Hytrulo is a subcutaneous formulation of the human IgG1 antibody fragment efgartigimod alfa, combined with the recombinant human hyaluronidase PH20, which allows its subcutaneous administration. The drug was approved in June 2023 for the treatment of generalized myasthenia gravis.
In February 2024, the FDA accepted Bristol Myers Squibb’s supplemental New Drug Application (sNDA) for its KRAS blocker Krazati (adagrasib), in combination with cetuximab, for the treatment of locally advanced or metastatic colorectal cancer (CRC). The deadline for the regulator’s decision is June 21.
The application, which seeks to make Krazati a treatment option for CRC patients with KRAS G12C-mutated CRC, is supported by data from the KRYSTAL-1 study. BMS reported data from this trial in March 2024, touting a 34% objective response rate at a median follow-up of 11.9 months. Median progression-free survival was 6.9 months, while median overall survival reached 15.9 months.
Krazati is an orally available small molecule inhibitor of G12C-mutated KRAS that won the FDA’s accelerated approval in December 2022. It was originally developed by Mirati Therapeutics, which BMS acquired in October 2023 for $4.8 billion.
Harmony Biosciences is proposing its oral drug Wakix (pitolisant) as a treatment for excessive daytime sleepiness in pediatric narcolepsy patients aged 6 years and above. The FDA’s deadline for a decision is June 21.
In its sNDA, Harmony provided data from a Phase III multicenter, randomized and placebo-controlled study evaluating the efficacy and safety of Wakix in narcolepsy patients ages six through 17 with or without cataplexy. The FDA granted the application its priority review status.
Wakix is an orally available histamine 3 receptor antagonist. It was approved by the FDA in August 2019 for the treatment of excessive daytime sleepiness of adult narcolepsy.
By June 21, the FDA is expected to release its verdict on Merck’s sBLA proposing to use its blockbuster PD-1 inhibitor Keytruda, in combination with standard of care chemotherapy, as a treatment for primary advanced or recurrent endometrial carcinoma.
Merck is supporting its bid for the additional indication with results from the Phase III NRG-GY018 study, which found that the Keytruda-based investigational regimen cut the risk of disease progression or death by 46% in patients whose disease was mismatch repair proficient. Meanwhile, those with mismatch repair deficient disease saw a 70% reduction in the risk of progression or death versus chemotherapy alone.
If approved, Keytruda would become the “first immunotherapy indicated for the frontline treatment of advanced endometrial cancer regardless of mismatch repair status,” Gursel Aktan, vice president of global clinical development at Merck Research Laboratories, said in a statement accompanying the sBLA’s acceptance in February 2024.
In February 2024, the FDA accepted an efficacy supplement to Sarepta Therapeutics’ sBLA seeking to convert the accelerated approval of its gene therapy Elevidys (delandistrogene moxeparvovec-rokl) into traditional approval, as well as broaden its label to include all Duchenne muscular dystrophy (DMD) patients with confirmed DMD gene mutations. The FDA’s decision is due on June 21.
Currently, Elevidys is only indicated for ambulatory pediatric patients aged 4 through 5 years of age. The FDA granted Elevidys accelerated approval in June 2023, a decision that has recently come under fire, particularly as the gene therapy failed its primary efficacy endpoint in part 1 of the SRP-9001-102 study, unable to significantly outdo placebo in terms of patients’ functional performance.
Sarepta’s bid for full approval is supported by the Phase III EMBARK study, which in October 2023 also fell short of its primary endpoint. In patients treated with Elevidys, scores on the North Star Ambulatory Assessment tool at 52 weeks were only 0.65 points higher than placebo comparators, an effect that did not satisfy statistical significance.
https://www.biospace.com/article/fda-action-alert-merck-amgen-sarepta-and-more/
Novo Nordisk CEO Lars Jørgensen has agreed to testify before the Senate Health, Education, Labor and Pensions Committee regarding the prices of the pharma’s top-selling semaglutide brands Ozempic and Wegovy, according to Sen. Bernie Sanders (I-Vt.).
Jørgensen will appear before the committee in a hearing dedicated to what Sanders calls the “outrageously high prices” of Ozempic and Wegovy in the U.S. The hearing is scheduled for early September 2024, though no exact date has been given yet.
Sanders, who is chair of the Health, Education, Labor and Pensions Committee, in a statement on Friday said that he “enjoyed” his conversation with Jørgensen, thanking him for “agreeing to voluntarily testify” at the hearing.
Last week, Sanders threatened to subpoena Novo Nordisk President Doug Langa to provide testimony about his company’s pricing for Ozempic and Wegovy. The vote on the subpoena, which was initially scheduled for June 18, has now been cancelled. “The scheduled subpoena vote is no longer necessary,” Sanders said on Friday.
Sanders launched the probe of Ozempic and Wegovy in April 2024, blasting the disparity in the prices of these drugs in the U.S. versus other developed countries. Ozempic, costs upwards of $900 per month in the U.S., while the treatment can be bought for $155 in Canada and $59 in Germany, according to Sanders.
Americans also pay more than $1,300 per month for Ozempic, while Novo sells the same product for $140 in Germany and $92 in the United Kingdom, Sanders contends.
“As important as these drugs are, they will not do any good for the millions of patients who cannot afford them,” Sanders said at the time, adding that their bloated price tags “have the potential to bankrupt Medicare, Medicaid and our entire health care system.”
The committee’s investigation also cited a March 2024 study, published in the Journal of the American Medical Association, which found that injectable semaglutide products can be profitably manufactured at less than $5 per month.
Sanders has launched an aggressive campaign to lower prescription drug prices in the U.S. In March 2023, the senator called on Sanofi and Lilly to follow Lilly’s example and lower insulin prices. The companies complied soon after.
In January 2024, Sanders also threatened to subpoena the CEOs of Merck and Johnson & Johnson, forcing them to participate in the committee’s broader investigation on drug prices in the U.S. The execs eventually agreed to testify at a February 2024 hearing, where they insisted that their prices reflect the costs of bringing products to market. In their respective testimony, the pharma companies pointed to pharmacy benefit managers as the main reason for inflated drug prices in the U.S.
Johnson & Johnson on Saturday posted promising data from the Phase II DAHLIAS study of its investigational FcRn blocker nipocalimab, demonstrating significant symptomatic improvement in patients with the chronic autoimmune Sjögren’s disease.
At 24 weeks, patients treated with nipocalimab saw a “statistically significant and clinically meaningful improvement” in the ClinESSDAI score, a composite scale specific for Sjögren’s disease that measures disease activity across 11 organ systems. Data were sparse but the pharma revealed that the improvement in ClinESSDAI scores had a p-value of 0.002 versus placebo.
In addition, patients showed response to nipocalimab as early as week 4 with the treatment effects persisting through 24 weeks of treatment.
Nipocalimab also elicited “clinically meaningful” improvements in key secondary endpoints including multiple organ and physician assessments, as well as other composite tools for clinical trial endpoints. DAHLIAS also recorded trends toward better outcomes in terms of important Sjögren’s disease symptoms, such as dryness of the eyes, mouth and vagina.
J&J presented these findings at a late-breaking session at last week’s 2024 Congress of the European Alliance of Associations for Rheumatology.
Terence Rooney, vice president of rheumatology at J&J, in a statement said that the data from DAHLIAS “demonstrate the potential of nipocalimab in a disease where patients have very few options,” noting that there is currently a “clear need” for “advanced therapies that target the underlying cause and systemic nature” of Sjögren’s disease.
Nipocalimab is an investigational monoclonal antibody that can bind with high affinity to FcRn receptor, which is commonly expressed in various cells of the immune system. This mechanism of action allows nipocalimab to lower circulating levels of IgG antibodies without triggering immune suppression and preserving the body’s overall immune function.
J&J secured access to nipocalimab in August 2020 when it dropped $6.5 billion to acquire Momenta Pharmaceuticals.
In addition to Sjögren’s disease, the pharma is testing nipocalimab in several immune-mediated indications. These include warm autoimmune hemolytic anemia, idiopathic inflammatory myopathy, rheumatoid arthritis and generalized myasthenia gravis.
In November 2023, J&J posted Phase IIa data for nipocalimab in moderate to severe rheumatoid arthritis demonstrating a significant reduction in circulating IgG antibodies, as well as numerical improvements in disease activity, treatment response and overall health.
In February 2024, the pharma released promising Phase III data for the investigational antibody in generalized myasthenia gravis. Nipocalimab treatment led to significant improvements in patients’ symptoms that affect their activities of daily living.
Gilead Sciences on Friday unveiled its final analysis of the Phase III ENHANCE study, providing more details surrounding the troubled development of its blood cancer therapy magrolimab.
In the late-stage trial—which compared magrolimab against placebo in nearly 540 higher-risk myelodysplastic syndromes (HR-MDS) patients who were also receiving standard-of-care azacitidine—Gilead’s antibody resulted in an approximately 20% higher risk of death versus placebo, though this effect was not statistically significant.
Overall survival in the magrolimab arm was also shorter by approximately three months.
Magrolimab’s objective response rate was 53.7%, which was lower than the observed rate of 58.7% in placebo comparators. Median progression-free survival was also slightly shorter in magrolimab-treated patients.
In addition to missing its efficacy endpoints, magrolimab was associated with excess side effects. Grade 3 or higher treatment-emergent adverse events arose in 92.8% of treated patients, compared to 79.2% of those in the placebo group. Overall, 84.8% of adverse events were found to be related to magrolimab, and 24% of patients had to discontinue the antibody due to toxicities.
Gilead presented these data at last week’s 2024 Hybrid Congress of the European Hematology Association.
According to ENHANCE’s abstract, its results are limited by several confounding factors including the imbalance in patients eligible for transplant between groups, as well as a partial clinical hold during its enrollment. Nevertheless, these findings “highlight challenges of developing anti-CD47 therapies and other new treatments” for HR-MDS, the abstract states.
Gilead gained the rights to magrolimab from its $4.9 billion acquisition of California-based biotech Forty Seven in 2020. The monoclonal antibody, which at the time was touted to have first-in-class potential, works by targeting and binding to the CD47 protein, which is commonly highly expressed in cancer cells. Through this mechanism of action, magrolimab is designed to boost the anti-cancer activity of the immune system and enable its phagocytic players to eliminate cancer cells.
However, magrolimab’s development has run into several safety and efficacy hurdles over the years. In January 2022, the FDA placed a partial clinical hold on studies evaluating the combination of magrolimab and azacitidine after unexpected safety signals. At the time, Gilead attributed the hold to an imbalance in serious adverse reactions.
Soon after, Gilead revealed that the hold had extended to two other magrolimab studies, which did not pair it with azacitidine. The FDA lifted all clinical holds in April 2022 following a comprehensive review of its safety data.
In July 2023, Gilead announced that it had discontinued ENHANCE after a planned interim analysis satisfied the criteria for futility. In its first-quarter 2024 business report, Gilead revealed that it had terminated all of its studies of magrolimab.
