Nevro’s high-frequency neurostimulation cuts diabetic neuropathy pain by 80%

 Two years after a high-frequency version of Nevro’s neurostimulation system became the first spinal cord stimulator approved by the FDA with a specific indication to treat pain associated with diabetic neuropathy, the company has published long-term study data further backing up the regulator’s decision.

The study spanned two years and enrolled more than 200 people with painful diabetic neuropathy, and its results were published recently in the journal Diabetes Research and Clinical Practice, the company announced Wednesday.

Throughout the study period, a total of 142 of the participants were implanted with the high-frequency version of Nevro’s Senza system. It’s implanted near the spinal cord and operates at a frequency of 10 kHz—allowing it to send 10,000 electric pulses per second to a patient’s nervous system, with an aim of interrupting pain signals as they travel to the brain.

Not only does Nevro’s system operate at a significantly higher frequency than many of its competitors’ spinal cord stimulators, but the company also claims that the HFZ approach eliminates the uncomfortable tingling and buzzing sensations associated with lower-frequency neurostimulation.

The Senza system is rechargeable, with a battery designed to last up to 10 years.

Diabetic neuropathy affects about a quarter of all people with diabetes, according to statistics cited by Nevro, with often-painful symptoms including numbness, tingling, balance issues and a reduced response to sensory stimuli, among others.

The symptoms are typically treated with oral pain medications, which may not be overwhelmingly effective and can spark painful or otherwise debilitating side effects of their own, the company noted.

Meanwhile, after 24 months, users of the Senza system experienced an average pain reduction of 79.9%, and just over 90% of the study’s participants saw their pain levels slashed at least in half after being implanted with the neurostimulation device.

Beyond pain relief, Nevro’s technology also helped improve participants’ overall health-related quality of life. In the latter metric, the implanted patients saw an average improvement of 0.146 on the standard EuroQol 5-Dimension 5-Level scale, which is about three to five times higher than the minimal important difference specified for people with Type 2 diabetes.

They also reported boosts in neurological function: By the end of the study, about two-thirds of the participants were showing clinically meaningful improvements compared to their baseline neurological function. The majority of those improvements fell under the sensory function category, which Nevro said suggests that its technology could help restore protective sensation in people with painful diabetic neuropathy.

Throughout the two-year study period, five of the Senza systems—representing about 3% of those implanted in the study—had to be removed due to infection, the company reported, but none were removed because they weren’t working.

The data suggest that the high-frequency Senza system is a “viable, long-term relief solution for people with PDN, which is particularly encouraging for a condition that naturally worsens over time,” Erika Petersen, M.D., lead principal investigator of the study, said in this week’s announcement.

David Caraway, M.D., Ph.D., Nevro’s chief medical officer, added that the company is expecting the promising study results—along with those from future planned studies—to boost physician referrals for Nevro’s therapy and to “continue to support market access for high-frequency SCS for PDN patients.”

https://www.fiercebiotech.com/medtech/nevros-high-frequency-neurostimulation-cuts-diabetic-neuropathy-pain-80-2-year-study

Mustang First Data from Ongoing Multicenter Phase 1/2 Trial of CAR-T Cell Therapy Disappoints

 Initial data show clinical responses from four of four indolent lymphoma patients, including complete response in follicular lymphoma patient previously treated with CD19 CAR-T cell therapy

Aligns with ongoing results from investigator-sponsored trial at Fred Hutch that show ongoing complete remission for more than three years

Update on data presented at 5th iwCAR-T, Scottsdale, AZ

Safety Review Committee unanimously approved dose escalation of the indolent lymphoma arm

 Mustang Bio, Inc. (“Mustang” or the “Company”) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced the first data from the indolent lymphoma cohort of the Company’s ongoing multicenter Phase 1/2 clinical trial evaluating MB-106, a first-in-class CD20-targeted, autologous CAR-T cell therapy for the treatment of relapsed or refractory B-cell non-Hodgkin lymphomas (“B-NHL”) and chronic lymphocytic leukemia (“CLL”), demonstrating clinical responses as well as safety and efficacy consistent with the ongoing Phase 1/2 clinical trial taking place at Fred Hutchinson Cancer Center (“Fred Hutch”).

Initial data were presented by Mazyar Shadman, M.D., M.P.H., Study Chair, Associate Professor and physician at Fred Hutch and University of Washington, at the 5th International Workshop on CAR-T and Immunotherapies (“iwCAR-T”).

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “We are encouraged that the first data from the multicenter trial of our lead candidate, MB-106, show clinical responses and that the trial is on track to achieve results consistent with those from the ongoing trial taking place at Fred Hutch. Overall, MB-106 continues to exhibit high efficacy and a favorable safety profile compared to currently approved autologous CAR-Ts. We expect to provide an additional update on dose escalation and report response data at a major medical meeting later this year.”

https://finance.yahoo.com/news/mustang-bio-announces-first-data-120000586.html

US appeals court rules to restrict abortion pill use

 Access to the abortion pill mifepristone must be restricted, a U.S. appeals court ruled on Wednesday, ordering a ban on telemedicine prescriptions and shipments of the drug by mail, though the order will not immediately take effect.

The New Orleans-based 5th U.S. Circuit Court of Appeals stopped short of ruling that the drug must be pulled off the market altogether, as a lower court had done.

Mifepristone's availability remains unchanged for now, following an emergency order from the U.S. Supreme Court in April preserving the status quo during the appeal.

The U.S. Food and Drug Administration, which approved the pill, and lawyers for the anti-abortion groups challenging the drug's approval did not immediately respond to requests for comment.

The three-judge 5th Circuit panel was reviewing an order in April by U.S. District Court Judge Matthew Kacsmaryk in Amarillo, Texas. While it was a preliminary ruling that applied while the case was pending, Kacsmaryk said he was ultimately likely to make it permanent.

The ruling stems from a lawsuit brought by four anti-abortion groups headed by the recently formed Alliance for Hippocratic Medicine and four anti-abortion doctors who sued in November.

They contend the FDA used an improper process when it approved mifepristone in 2000 and did not adequately consider the drug's safety when used by minors.

All three judges on the panel are staunchly conservative, with a history of opposing abortion rights. One of them, Circuit Judge William Ho, said he would have gone further and pulled mifepristone off the market altogether.

Instead, the majority of the panel rolled back FDA actions that had made the drug easier to access in recent years. Those included allowing distribution by mail, approving its use up to 10 weeks of pregnancy instead of seven weeks, reducing the dosage and cutting the number of required in-person doctor visits from three to one.

The decision will almost certainly be appealed first to the full 5th Circuit and then to the U.S. Supreme Court, which last year overturned its landmark Roe v. Wade ruling that had legalized abortion nationwide.

Since then, at least 15 of the 50 states have banned abortion outright while many others prohibit it after a certain length of pregnancy, according to the Guttmacher Institute, a research organization that supports abortion rights.

Mifepristone is part of a two-drug regimen with misoprostol for medication abortions, which account for more than half of U.S. abortions.

Numerous medical studies and many years of real-world use have concluded that the drug is safe and effective.

Major medical associations, including the American College of Obstetricians and Gynecologists and the American Medical Association, have said in court filings that pulling mifepristone off the market would harm patients by forcing them to undergo more invasive surgical abortions.

Hundreds of biotech and pharmaceutical company executives have called or the reversal of Kacsmaryk's ruling, saying it ignores decades of scientific evidence on the drug's safety and undermines the FDA's authority, potentially creating chaos for the industry that relies on the agency.

https://news.yahoo.com/us-appeals-court-rules-restrict-184207899.html

FDA starts speedy review of Servier's Tibsovo in MDS

 Servier could become the first company to bring an IDH inhibitor to market in the US for patients with myelodysplastic syndromes (MDS) next year, after the FDA started a priority review of its Tibsovo drug in this indication.

Tibsovo (ivosidenib) is under review as a treatment for patients with IDH1-mutated relapsed or refractory MDS, which would add to the drug's existing indications in IDH1-mutant acute myeloid leukaemia (AML) and cholangiocarcinoma.

Servier acquired the IDH1 inhibitor as part of its $1.8 billion takeover of Agios Pharma's oncology business in 2021 and it has become a key driver of sales growth for the French pharma group, with sales up 180% to €256 million ($280 million) in the 2021/2022 fiscal year results reported in February.

MDS is a mixed group of haematopoietic stem cell disorders with a high tendency to transform into AML, with around 20,000 new cases reported each year in the US. IDH mutations are only seen in a minority of patients - estimates are around 3% - but are associated with a poor prognosis and increased chance of turning into AML.

Tibsovo has a breakthrough designation from the FDA for the treatment of IDH1-mutant MDS, and Servier said it has an opportunity to "shift the treatment landscape" for these patients if it gets approval for the new indication.

The MDS application is based on results from a subset of 18 patients in a pivotal phase 1, reported at the European Haematology Association (EHA) meeting in June, which showed that Tibsovo achieved an overall response rate of 83%, with nearly 40% of them achieving a complete response.

The median time to complete response was 1.87 months, and at that time the median duration of complete response had not been reached with a median overall survival of just under three years.

Furthermore, out of nine patients who had been dependent on transfusions with red blood cells or platelets at enrolment, seven were able to become transfusion independent, although that number had dipped to six in the latest update.

"While the novel use of targeted IDH inhibition has been proven across a number of difficult-to-treat cancers, there continues to be a significant unmet need for MDS patients within this molecularly defined subset, especially for those who experience disease progression," commented Amir Fathi of the Center for Leukemia at Massachusetts General Hospital and Harvard Medical School.

He added that the filing "provides further support for the potential efficacy and acceptable safety profile of Tibsovo in relapsed or refractory MDS and reinforces the importance of mutational testing in this patient population."

One other IDH-targeting drug has also been tested in MDS, Rigel Pharma's Rezlidhia (olutasidenib), although that was an investigator-led trial and the company is focusing its efforts in MDS on another drug, IRAK1/4 inhibitor R289.

Servier has made oncology a key part of its growth strategy, with sales in its last fiscal year rising more than a third to €848 million, accounting for more than 17% of its total revenues.

https://pharmaphorum.com/news/fda-starts-speedy-review-serviers-tibsovo-mds

CFPB considering new rules to crack down on data brokers

 The Consumer Financial Protection Bureau (CFPB) is considering new rules to crack down on data brokers amid a wider push to reduce risks from the rapidly expanding capabilities of artificial intelligence. 

CFPB Director Rohit Chopra said at a White House roundtable on Tuesday that the agency is looking to potentially bring data brokers — firms that harvest and sell consumer data — under the purview of the Fair Credit Reporting Act.

The Fair Credit Reporting Act grants various protections to consumers regarding background reports that are assembled with information about them, such as safeguards to ensure accurate information and restrictions on its use.

Under one proposal floated Tuesday by Chopra, data brokers would be defined as consumer reporting agencies and the sale of data about payment history, income and criminal records would be treated as a consumer report, triggering additional requirements under the law.

The CFPB is also considering a rule that would clarify whether identifying information contained in consumer reports produced by credit reporting companies, known as “credit header” data, is also considered a consumer report.

Such a rule could limit “the ability of credit reporting companies to impermissibly disclose sensitive contact information that can be used to identify people,” Chopra said.

The CFPB director warned Tuesday that artificial intelligence and other predictive decision-making technologies increasingly rely on mass amounts of data, which can be produced by data brokers and have “big implications.”

“This also has big implications when it comes to critical decisions, like whether or not we will be interviewed for a job or get approved for a bank account or loan,” Chopra said. “It’s critical that there’s some accountability when it comes to misuse or abuse of our private information and activities.”

https://thehill.com/policy/technology/4154371-cfpb-considering-new-rules-to-crack-down-on-data-brokers/

Vera started at Buy by Guggenheim

 Target $27

https://finviz.com/quote.ashx?t=VERA&ty=c&ta=1&p=d

Axsome upped to Neutral from Underperform by B of A

 Target to $81 from $59

https://finviz.com/quote.ashx?t=AXSM&ty=c&ta=1&p=d