lead drug candidate MTX110 for the treatment of patients with recurrent glioblastoma (rGBM).
The company is planning to begin recruitment in the phase 1 study evaluating MTX110 in patients with recurrent glioblastoma (GBM).
Dmitry Zamoryakhin, CSO, said: "rGBM is a devastating cancer marked by short survival rate and universal recurrence. Receiving Fast Track designation for MTX110 is an important milestone for the development of the drug as it demonstrates the need for novel and effective treatment options for this currently universally fatal disease."
FDA Fast Track Designation is intended to expedite the development and review of new drugs to treat serious medical conditions that fill unmet medical needs.
has dosed first patient in the figHTN-CKD Phase 2 clinical study of its drug candidate Baxdrostat (CIN-107) for the treatment of patients with uncontrolled hypertension and chronic kidney disease (CKD).
The figHTN-CKD trial is a randomised Phase 2 clinical trial designed to assess the safety and efficacy of Baxdrostat (CIN-107) in patients with uncontrolled hypertension and CKD.
The company anticipates initial top-line data readout for figHTN-CKD clinical trial in second half of 2023.
Mason Freeman, M.D., Chief Medical Officer, commented: "Baxdrostat is a highly selective aldosterone synthase inhibitor that can significantly reduce aldosterone levels, offering the potential to not only improve uncontrolled blood pressure in CKD patients, but also ameliorate the negative impacts of aldosterone on kidney function."
Baxdrostat (CIN-107) is an oral small molecule inhibitor of aldosterone synthase, the enzyme responsible for the synthesis of aldosterone in the adrenal gland, in development for treatment-resistant hypertension and primary aldosteronism.
Centers for Disease Control and Prevention Director Rochelle Walensky warned that Pfizer’s COVID-19 pill Paxlovid can lead to a rebound in symptoms.
“If you take Paxlovid, you might get symptoms again,” Walensky told CBS News on Tuesday.
“We haven’t yet seen anybody who has returned with symptoms needing to go to the hospital. So, generally, a milder course.”
Another researcher who is not affiliated with the CDC said that he has observed such a scenario.
“People who experience rebound are at risk of transmitting to other people, even though they’re outside what people accept as the usual window for being able to transmit,” Dr. Michael Charness of the Veterans Administration Medical Center in Boston told CNN on Tuesday.
After a patient recovers from COVID-19, the aforementioned rebound has occurred between two and eight days later, according to the CDC. The agency, however, told CBS that the benefits of taking Paxlovid outweigh the risks of COVID-19, namely among those who are at a high risk of developing severe symptoms from the virus.
About a week ago, the agency issued an alert to health care providers about the rebound, saying that patients who took Paxlovid either test positive for the virus after having tested negative or will experience COVID-19 symptoms.
“A brief return of symptoms may be part of the natural history of SARS-CoV-2 infection in some persons, independent of treatment with Paxlovid and regardless of vaccination status,” the federal health agency said at the time. SARS-CoV-2 is another name for the CCP (Chinese Communist Party) virus, which causes COVID-19.
“Limited information currently available from case reports suggests that persons treated with Paxlovid who experience COVID-19 rebound have had mild illness; there are no reports of severe disease. There is currently no evidence that additional treatment is needed with Paxlovid or other anti-SARS-CoV-2 therapies in cases where COVID-19 rebound is suspected,” the CDC added.
The Epoch Times has contacted Pfizer for comment. Pfizer told CBS that it is observing a rebound rate of approximately 2 percent and is continuing to monitor patients.
“We have not seen any [COVID-19] resistance emerge to date in patients treated with Paxlovid,” a spokesperson for the company told Reuters this week.
In recent weeks, doctors have increasingly prescribed Paxlovid, which has been authorized to treat at-risk people. But some health care workers told Reuters they are putting off prescribing the medication.
“I am shying away from giving it to people who are very low- risk, and are not terribly ill, particularly people who are vaccinated and boosted,”said Dr. Bruce Farber, chief of public health and epidemiology for Northwell Health, in an interview with the news agency. He will still prescribe the bill to people who have significant health conditions or are aged 75 and older.
CTI BioPharma Corp. (Nasdaq: CTIC) today announced a poster presentation from the Company's pacritinib program at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting, being held inChicagoand virtually,June 3-7, 2022.
"These data reinforce pacritinib's role as a safe, differentiated JAK inhibitor. Our risk-adjusted analysis demonstrates that the safety profile of pacritinib 200 mg twice a day is comparable to best available therapy, including ruxolitinib, and that pacritinib 200 mg twice daily could be a full-dose therapeutic option for patients with myelofibrosis, including those who experience severe thrombocytopenia," said Adam Craig, President and Chief Executive Officer of CTI BioPharma. "Earlier this year, VONJOTM (pacritinib) at 200 mg orally twice daily received accelerated FDA approval, becoming the first approved therapy that specifically addresses the needs of patients with cytopenic myelofibrosis. The NCCN® Clinical Practice Guidelines in Oncology for Myeloproliferative Neoplasms also recently included VONJO as a recommended first- and second-line treatment. With a successful beginning to our commercial launch, these data reinforce VONJO's value as a new standard of care for cytopenic myelofibrosis patients with platelet counts <50 × 109/L who have been waiting for new treatment options."
Risk-adjusted safety analysis of pacritinib in patients with myelofibrosis (ASCO Poster #7058)
Pacritinib is a novel JAK2/IRAK1 inhibitor that has shown significant activity in patients with myelofibrosis, including those with platelet counts <50 × 109/L. This safety analysis focuses on toxicities of interest for patients treated with pacritinib 200 mg twice daily (BID) and best available therapy (BAT), including ruxolitinib, on the Phase 3 PERSIST-2 and Phase 2 PAC203 studies. Because the average treatment duration was longer for patients on pacritinib 200 mg BID on PERSIST-2 and PAC203 compared to BAT on PERSIST-2, this analysis presents adverse events rates in these patients corrected for duration of exposure.
This risk-adjusted analysis demonstrates that the safety profile of pacritinib 200 mg BID is comparable to BAT. In particular, rates of bleeding were not elevated on pacritinib 200 mg BID compared to BAT, both overall and in patients with PLT <50 x 109/L. Rates of fatal events, thrombosis, major adverse cardiac events (MACE) and non-melanoma skin cancer were higher on ruxolitinib than pacritinib. These results indicate that pacritinb 200 mg BID may represent a full-dose therapeutic option for patients with myelofibrosis, including those with thrombocytopenia.
NeuroOne, a medtech company based in Minnesota, has developed the Evo Cortical Electrode and the sEEG electrode, both of which are thin film electrodes for neural recording and stimulation. The small profile and flexible nature of the electrodes allows for less invasive insertion, and the low resistance they offer delivers improved signal quality.
The devices can combine both diagnostic and therapeutic functions, which could potentially reduce the number of procedures a patient has to undergo, as diagnosis and treatment can occur during the same procedure.
The company has also conducted simulations of long-term neural stimulation, and found evidence that the technology could provide stimulation for extended periods, which could make it useful for applications such as treating chronic back pain in patients who have undergone multiple failed surgeries.
Medgadget had the opportunity to speak with Dave Rosa, CEO of NeuroOne, about the technology.
Conn Hastings, Medgadget: Please give us an overview of neurostimulation/neuromodulation and how the technology has progressed to date.
Dave Rosa, NeuroOne: In the early 1960’s neuromodulation started with deep brain stimulation and then in the late 1960’s was followed by spinal cord stimulation. Sacral stimulation for urinary incontinence was developed in the early 1980’s finally receiving FDA clearance in 1997. The first single channel neurostimulator reportedly was implanted by in 1961 into two deaf patients but the first FDA cleared single channel device wasn’t released until 1984. Neuromodulation interacts with the nervous system using electrical stimulation to regulate, inhibit or modify neural activity. Today, the most common indications are for conditions such as: epilepsy, Parkinson’s disease, chronic back pain due to previously failed back surgeries, urinary incontinence, blindness, and peripheral pain. A colleague of mine told me a few years ago that while today we take medications to treat a variety of ailments, one day we would use electrical stimulation to treat the same conditions. I thought he was joking but it truly seems like this therapy has the potential to treat a plethora of conditions. Today with the public spotlight on mental health conditions, stimulation is being investigated as a potential solution to treat depression, OCD, short term memory loss and other neurocognitive disorders. One of the largest potential opportunities discussed is Alzheimer’s. There are promising diagnostic technologies such as SYNAPS Dx that are making progress in early identification of Alzheimer’s while deep brain stimulation is being investigated to treat the major inflow and output pathways in the brain that are reported to be impacted by Alzheimer’s.
In addition to new indications, there are a number of areas with respect to the technology that have made great progress.
While these procedures have generally been very invasive, required separate surgeries for diagnosis and treatment, and also relied on pre-determined stimulation settings that require the patients return to the doctor’s office to have adjustments, there are many advancements that have been made or that we are likely to see in the future. New minimally invasive or less invasive procedures now dominate the industry, wireless systems are in development to eliminate bulk and external wiring, closed loop systems now exist which make patient stimulation adjustments automatically, drug coated devices and multi-purpose devices that can perform both diagnostic and therapeutic functions are all advancing.
Medgadget: What inspired NeuroOne to develop the Evo Cortical Electrode?
Dave Rosa: NeuroOne initially set out to develop a thin film, high-definition electrode technology that had the capability to perform multiple diagnostic and therapeutic functions while utilizing advanced automated manufacturing procedures. While most companies race to get a single device or therapy to the finish line, we saw the opportunity to develop a platform of products but knew we had to start somewhere. We decided that the easiest development and manufacturing path would be to focus on a cortical electrode for recording and stimulating brain activity for less than 30 days. These devices were the gold standard for years for identifying the location of the brain that was causing irregular activity during a patient’s seizure. Doctors at the Mayo Clinic in Rochester Minnesota also partnered with us and provided essential feedback during the development phase. We are now working on the more popular sEEG electrode technology that require tiny holes to be drilled into the brain to perform a similar function as cortical electrodes but much less invasively. We expect to submit this product family to the FDA for 510(k) clearance in August 2022.
Medgadget: Please give us an overview of the electrode features, and its advantages compared with conventional electrodes.
Dave Rosa: The materials used for both our cortical and sEEG electrodes are very similar so both product families share similar features. In general, NeuroOne’s Evo cortical and sEEG electrodes are thin film devices. Given the thinness and flexibility of the devices, they are designed to enable less invasive insertion. They are also lighter weight, generally speaking, due to their thinness. These electrodes also have low resistance levels which help improve the signal quality for physicians. All our products utilize photolithography to manufacture the electrodes which provides a highly reproducible product versus most current commercially available electrodes that rely primarily on manual labor.
Medgadget: How is the electrode intended to be used? Does it have diagnostic and therapeutic applications?
Dave Rosa: The Evo cortical and sEEG electrodes are both designed for diagnostic purposes. However, one of the advantages of the technology is that it has the potential to be used for therapeutic purposes as well. Ultimately, we expect the devices to have the capability to monitor, ablate and stimulate targeted tissue all using the same device. We have devices in development for these multiple functions and will need to submit them for FDA clearance when development is completed.
Medgadget: Can the technology be used for long-term stimulation? What types of disease state would this be useful for?
Dave Rosa: We recently announced that we had successfully tested the devices in a bench top model to stimulate over a 5-year accelerated period. We also performed accelerated 5-year testing on the devices for recording. These tests suggest that the electrodes will function over a 5-year period.
The devices could be used for a variety of different conditions that have reported benefits from electrical stimulation. Parkinson’s disease, epilepsy, chronic back pain due to failed back surgeries, peripheral pain, incontinence, hypertension, and mental health disorders are either being used today to treat these conditions or are under investigation. We also will need to submit to FDA to receive clearance to market the devices for these functions.
Medgadget: Do you have any plans for other technologies in the future? Where do you see the company in five years?
Dave Rosa: We currently have a device in development that is intended to perform both a diagnostic and therapeutic function. It has the capability to record electrical activity in the brain in an effort to determine the problematic area(s) as well as ablate the problematic tissue. Today this is usually done with multiple surgeries and hospitalizations whereas our technology would intend to perform both functions in one hospitalization and surgery.
In addition, we have a separate program for spinal cord stimulation to treat chronic back pain due to multiple failed back surgeries. Given the properties of our thin film technology, we believe our electrodes have the potential to be placed less invasively along with providing additional benefits due to its uniqueness. In the future, I also expect these devices to help enable artificial intelligence to treat a variety of neurological conditions in addition to drug coated devices much like we have seen in the cardiovascular arena over the years. The neurovascular space reminds me of where interventional cardiology was 30 years ago and I believe it has similar exciting potential for future growth.
Sage Therapeutics has finally had some good news: its Gaba-A modulator zuranolone produced a convincing win in Skylark, its pivotal postpartum depression study. The question is how much this will impress investors, who have their eye on the bigger use of major depressive disorder – and here there arestill doubts about zuranolone’s approvability and commercial prospects. Sage and its partner Biogen have started a rolling submission in MDD, and plan a separate PPD filing early next year. One question is cost: Stifel analysts have speculated that Sage will price the drug for the larger MDD indication, although the company is staying tight-lipped for now. Sage estimates that around 500,000 women in the US have PPD, but only 28% of these are currently diagnosed; it hopes that an oral therapy will drive up this number. Sage already has a PPD drug in Zulresso, but that is given via a 60-hour infusion. The main downside in Skylark was a higher incidence of side effects like somnolence versus the lower-dose Robin trial, for arguably no additional clinical benefit. Sage reckons 50mg will be the go-to dose, with the option to reduce if required. The company’s stock climbed 4% this morning.
Flying high or wings clipped? Zuranolone's post-partum depression data
