AstraZeneca (LSE: AZN) said today it has decided to discontinue the STABILIZE-CKD and DIALIZE-Outcomes Phase III evidence trials for Lokelma (sodium zirconium cyclosilicate).
AstraZeneca explained that the decision was made due to substantially increased enrolment timelines and low event rates, respectively, which made it prohibitive to deliver study results within a timeframe to meaningfully advance clinical practice.
Phreesia is very pleased to announce that the Centers for Medicare and Medicaid Services (CMS) has included the Patient Activation Measure Performance Measure (PAM-PM) in its Merit-Based Incentive Payment System (MIPS), as well as in 15 specialty-specific measure sets and five MIPS Value Pathways (MVPs), subsets of measures for specific conditions.
The Patient Activation Measure (PAM) measures a patient’s knowledge, skills and confidence to self-manage their health. Insignia Health, a wholly owned subsidiary of Phreesia, is the measure steward of the PAM. Activated patients feel comfortable advocating for themselves and asking for support from their care team to manage their own health. But many patients who feel discouraged and overwhelmed need more targeted support and resources.
https://finance.yahoo.com/news/cms-includes-patient-activation-measure-110000611.html
Pfizer slumped lower Friday after the drugmaker said it would not advance a study of its two-dose obesity treatment, following a high level of side effects in patients involved in the Phase II trial.
Pfizer said it would instead focus on a single-dose option. Trials of its danuglipron treated showed good results in weight loss but induced vomiting and nausea in patients, which led to what it called "high discontinuation rates" compared with those taking a placebo.
Pfizer's Mikael Dolsten, president of worldwide research and development, in late October called the danuglipron study "our main opportunity here for getting data to review for obesity, in fact, to diabetes" in a call with investors.
The decision marks a significant setback in Pfizer's ambitions to enter the weight-loss drug market, which is currently dominated by Novo Nordisk's Wegovy. Pfizer rival Eli Lilly (LLY) - Get Free Report last month also won approval for its own weight-loss treatment, Zepbound, from the Food and Drug Administration.
“We believe an improved once-daily formulation of danuglipron could play an important role in the obesity treatment paradigm, and we will focus our efforts on gathering the data to understand its potential profile,” said Pfizer's chief science officer, Mikael Dolsten.
“Results from ongoing and future studies of the once-daily danuglipron modified release formulation will inform a potential path forward with an aim to improve the tolerability profile and optimize both study design and execution,” he added.
Pfizer shares were marked 4.17% lower in premarket trading to indicate a Friday opening bell price of $29.20 each, a move that would extend the stock's six-month decline to around 23%.
Pfizer slashed its full-year sales and profit forecasts in October amid a slump in demand for covid treatments, It told investors it would write off around $4.6 billion in inventories of Paxlovid, its oral covid treatment, while taking a further $5.5 billion noncash charge against its third-quarter earnings.
Japan’s medicines regulator has approved the first vaccine based on self-assembling RNA, a spin on the mRNA technology used in current shots, which is designed to deliver greater efficacy with a lower dose.
The Ministry of Health, Labour and Welfare (MHLW) gave the green light to CSL and Arcturus’ COVID-19 vaccine ARCT-154 – also known as Lunar-COV19 – which will be commercialised in Japan by partner Meiji Seika Pharma. Japan is the first country to clear the saRNA vaccine, which has also been filed for approval in Europe.
The lipid nanoparticle-formulated shot, given in two doses 28 days apart, has been shown to be effective against multiple ancestral strains of SARS-CoV-2, the virus that caused COVID-19. In phase 3 trials, it showed more than 95% efficacy in the prevention of severe COVID infections.
saRNA vaccines rely on RNA-dependent RNA polymerases (RDRP) derived from RNA viruses to amplify the delivered RNA, increasing the production of antigen proteins in the body. Studies suggest the approach can improve the durability of protection, in part because it prolongs the stimulation of antigen-presenting cells (APCs) important for long-term immunity.
CSL’s Seqirus subsidiary bought into the ARCT-154 programme last year as part of a wide-ranging collaboration for saRNA vaccines spanning COVID-19, influenza and other respiratory infections that included an upfront payment of $200 million and more than $4 billion in potential milestones.
The bottom has largely dropped out of the market for COVID-19 vaccines, with the two top producers of mRNA-based shots – Pfizer/BioNTech and Moderna – both reporting lowered sales expectations this year that could get worse in 2024 before stabilising in 2025 and beyond.
ARCT-154 targets ancestral variants of the coronavirus, so will likely not be an option for current vaccination campaigns. Nevertheless, the MHLW is an important milestone for the saRNA field and the Arcturus/CSL alliance, which has candidates against other variants in phase 3 testing – including the prevailing XBB.1.5 strain – as well as season and pandemic flu candidates in early development.
The approval “marks a historic and exciting milestone as the first [saRNA] vaccine in the world to be registered, and supports CSL’s promise to protect global public health,” said Jonathan Edelman, head of the company’s vaccines innovation unit.
“We are committed to working with health authorities around the world to ensure this important vaccine technology will be available to people at risk for COVID-19.”
https://pharmaphorum.com/news/csl-arcturus-get-first-approval-self-assembling-rna-jab
New data for Astellas Pharma’s non-hormonal menopause therapy fezolinetant has shown it remains effective to 24 weeks, consolidating the evidence of the drug as it starts to roll out in the US and Europe.
Fezolinetant was approved in the US in May as Veozah, becoming the first drug in the neurokinin 3 (NK3) receptor antagonist class to reach the market ahead of closest rival elinzanetant from Bayer. Last month, it was also recommended for approval in the EU under the Veoza brand name.
Those applications were based on clinical data showing efficacy at four and 12 weeks compared to placebo, but the new readout from the phase 3b DAYLIGHT study shows its benefits now last for much longer.
Astellas said the data – generated in more than 450 women who were unable or unwilling to take hormone replacement therapy (HRT) to alleviate symptoms of menopause – will support health technology assessments (HTA) for reimbursement negotiations.
In the study, oral dosing with fezolinetant at a dose of 45 mg per day achieved a statistically significant -1.93 reduction in the frequency of moderate to severe vasomotor symptoms (VMS) at 24 weeks compared to placebo, the primary endpoint.
Astellas’ drug also hit the key secondary endpoint of mean change from baseline in the severity of moderate to severe VMS, with a reduction of -0.39 compared to placebo, and also reduced sleep disturbances by -2.5 compared to the control group.
VMS, such as hot flushes, are reported by up to 80% of women at some point during menopause and the leading cause for seeking medical attention during this phase of a woman’s life.
The pharma company hopes to build momentum for fezolinetant in the marketplace before Bayer is in a position to launch elinzanetant, a dual neurokinin 1,3 antagonist that has shown strong signals for efficacy in phase 2 and is due to report phase 3 results from the OASIS trials programme before the end of this year.
Bayer is planning a launch in 2025 and has previously predicted $1 billion in annual sales for its drug. Astellas, meanwhile, has said fezolinetant could become a $2.2 billion to $3.4 billion product at its peak.
Astellas acquired fezolinetant as part of its 2017 takeover of Ogeda for €500 million (at the time equivalent to around $550 million) upfront, plus €300 million in potential milestones. Bayer, meanwhile, acquired elinzanetant as part of its $875 million takeover of UK biotech KaNDy Therapeutics in 2020.
https://pharmaphorum.com/news/astellas-builds-case-menopause-drug-fezolinetant
