Surfing MASH’s Post

OKYO Pharma Ltd (NASDAQ:OKYO) has shared encouraging results from a phase II trial of its treatment candidate for dry eye disease. The study involved 240 patients and showed that 68% of them had improved by meeting both key measures: reduced conjunctival staining and ocular pain. The recently released data comes from a randomized, double-masked, placebo-controlled trial. This means that neither the patients nor the researchers knew who was receiving the actual treatment or a placebo, which helps ensure unbiased results. The trial aimed to test the safety and effectiveness of OK-101 eye drops. More at #Proactive #ProactiveInvestors http://ow.ly/SXpe105A7wh

Sounds like another horrendous example of trial failure. Also sounds like patient groups were not consulted sufficiently, or listened to, nor of course sitting at the table when critical decisions were taken. It is devastating. We are starting to hear about wiser, more realistic companies planning their trials with real patient input and impact. When will the rest of them join and understand it is in their interest to devellop drugs differently ? #clinicaltrials #patientengagement #pharma

📰 NEWS  We are pleased to announce that our lead candidate FT011 has been granted the International Non-Proprietary Name (INN) of ‘asengeprast’ by the World Health Organization (WHO). #Asengeprast is a novel, first-in-class oral #GPR68 antagonist being developed for the treatment of chronic fibrosis in multiple organs. Phase I and IIa clinical studies in patients with systemic sclerosis (#SSc) have already demonstrated favourable efficacy, safety and pharmacokinetics.   Certa Therapeutics CEO Professor Darren Kelly said, “The granting of an INN for our lead drug candidate, #asengeprast is another important step in the development of this important therapy and follows the granting of EU and US Orphan Drug status and an FDA Fast Track Designation. We are continuing to drive the clinical development of asengeprast and believe it has the potential to address a critical need for people living with SSC, a debilitating condition with the highest mortality amongst rheumatic diseases.”   Certa has pioneered drug development targeting GPR68, an important but previously undrugged membrane GPCR receptor, showing it to be a master switch of fibrosis.  Press release 👇🏼 https://lnkd.in/gnZb5RUU   #fibrosis #drugdevelopment #clinicaltrial #scleroderma

In my new article, I talk about the incredible potential of a unique new drug, SPG302, developed by Spinogenix, Inc., for treating amyotrophic lateral sclerosis (ALS) and how the FDA has approved it for ongoing clinical trials. The drug regenerates the synapses damaged by the fatal disease. In Phase 1 trials conducted in Australia, the drug has proven safe and is now being trialled in people with ALS. It could be a watershed moment in ALS treatment. #ALS #synapse #smallmolecule https://lnkd.in/gbJhSmbx

There are no FDA/EMA/etc approved therapies for mitochondrial disease. We need to work together to thoughtfully collect and integrate data to inform clinically meaningful endpoints and de-risk clinical trial design. This task force is dedicated to advancing the regulatory science needs so that individuals living with mitochondrial and inherited metabolic diseases can finally see some drug development they so desperately need and want. #missiondriven #collaboration #publicprivatepartnership #regulatory

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> FDA adcomm backs Carvykti's benefit in earlier patients; Abecma vote to come: An FDA advisory committee voted unanimously on Friday that the benefits of Johnson & Johnson and Legend Biotech’s CAR-T therapy Carvykti outweigh its risks in earlier-stage multiple myeloma patients. Later Friday, the committee is expected to vote on Bristol Myers Squibb and 2seventy bio’s Abecma in earlier patients. Both CAR-Ts are currently approved as fifth-line treatments. But when asked to bring the therapies into earlier lines, FDA reviewers raised concerns in briefing documents about higher rates of death in the first months of treatment among patients who took the CAR-Ts compared with those who received standard regimens. That early imbalance of deaths was a key focus at the Oncologic Drugs Advisory Committee’s Thursday meeting. Daniel Spratt, professor at University Hospitals Seidman Cancer Center and Case Western Reserve University, said the FDA was “very appropriate” to ring the alarm. But after seeing the totality of Carvykti’s data, he said “the benefit outweighs the risk, and I think there’s a lot of learning lessons to continue to optimize this.” Jorge Nieva, associate professor of clinical medicine at the University of Southern California, said of Carvykti: “This is a case of front-loaded risk, much like we see with many other things in medicine. “As long as the patients understand the magnitude of the front-loaded risk, then I think these risks are acceptable,” Nieva added. Moving into earlier settings J&J and Legend Biotech have applied to bring Carvykti (cilta-cel) to second-line patients, while Bristol Myers and 2seventy want to bring Abecma (ide-cel) to the third-line setting. Both J&J and BMS said in briefing documents that many early deaths in the CAR-T arms of their studies happened before patients had received Carvykti or Abecma. BMS wrote that most early deaths were caused by disease progression. Mark Wildgust “This is a really tough group of patients,” Mark Wildgust, VP of global medical affairs for J&J’s oncology division, told Endpoints News ahead of the adcomm. He added that patients in the Carvykti arm also received lower dose intensities of bridging therapies, which were taken as the CAR-Ts were being manufactured. However, J&J noted in its briefing documents that “the extent of this impact is unclear.” “There’s one really important clinical finding from CARTITUDE-4, which is physicians should really be using the optimal bridging therapy,” Wildgust said, referring to Carvykti’s Phase III study in multiple myeloma patients who received one to three prior lines of therapy. Lola Fashoyin-Aje, a director at CBER’s Office of Clinical Evaluation, said during the adcomm that “we may never know why there was this increase [in] early deaths… #lucidquest #genetherapy #celltherapy

# Only put off until tomorrow what you are willing to die having left undone ## Embrace the Future of Healthcare: Invest in Zevra Therapeutics, Inc. \(ZVRA\) 🏥💰 Don't miss out on the opportunity to invest in Zevra Therapeutics, Inc. \(ZVRA\) as they obtained FDA approval for arimoclomol capsules, a groundbreaking treatment for Niemann-Pick disease type C \(NPC\). This significant milestone positions Zevra as a frontrunner in the healthcare industry. 💊 Zevra's arimoclomol capsules, branded as Miplyffa, have opened doors to advanced neurological care for patients suffering from NPC. The FDA has approved Miplyffa for the treatment of neurological manifestations when used in combination with miglustat, addressing the critical needs of adult patients fighting this disease. 📈 With a surging stock price close to 70% in just three months, Zevra is proving to be a promising investment opportunity. As a seasoned investment advisor, I strongly recommend considering ZVRA as part of your investment portfolio. 🚀 Don't let the Fear of Missing Out \(FOMO\) hold you back from potentially reaping the benefits. Harness the power of your Health Savings Account \(HSA\) and invest in Zevra to support cutting-edge advancements in healthcare. #healthcare #hsa #investing #health #family #wellness

We applaud the U.S. FDA Oncologic Drugs Advisory Committee (ODAC) unanimous recommendation (12-0) supporting minimal residual disease (MRD) as a viable endpoint in multiple myeloma clinical trials. This decision marks great news for the multiple myeloma community as it could potentially lead to the accelerated approval of innovative therapies and improved clinical outcomes for patients in the future. I’d like to commend the commitment of Johnson & Johnson Innovative Medicine scientists and clinicians who have worked collaboratively with investigators, academic centers, advocacy groups, across consortia and with the FDA and other Health Authorities to advance the science of multiple myeloma, with determination and focus in recognizing the important role MRD may play in improving outcomes for patients. #MyCompany #JNJOncology #MultipleMyeloma #OncologyResearch #ClinicalTrials #Innovation

Gene&Cell Therapy >> FDA adcomm backs Carvykti's benefit in earlier patients; Abecma vote to come: An FDA advisory committee voted unanimously on Friday that the benefits of Johnson & Johnson and Legend Biotech’s CAR-T therapy Carvykti outweigh its risks in earlier-stage multiple myeloma patients. Later Friday, the committee is expected to vote on Bristol Myers Squibb and 2seventy bio’s Abecma in earlier patients. Both CAR-Ts are currently approved as fifth-line treatments. But when asked to bring the therapies into earlier lines, FDA reviewers raised concerns in briefing documents about higher rates of death in the first months of treatment among patients who took the CAR-Ts compared with those who received standard regimens. That early imbalance of deaths was a key focus at the Oncologic Drugs Advisory Committee’s Thursday meeting. Daniel Spratt, professor at University Hospitals Seidman Cancer Center and Case Western Reserve University, said the FDA was “very appropriate” to ring the alarm. But after seeing the totality of Carvykti’s data, he said “the benefit outweighs the risk, and I think there’s a lot of learning lessons to continue to optimize this.” Jorge Nieva, associate professor of clinical medicine at the University of Southern California, said of Carvykti: “This is a case of front-loaded risk, much like we see with many other things in medicine. “As long as the patients understand the magnitude of the front-loaded risk, then I think these risks are acceptable,” Nieva added. Moving into earlier settings J&J and Legend Biotech have applied to bring Carvykti (cilta-cel) to second-line patients, while Bristol Myers and 2seventy want to bring Abecma (ide-cel) to the third-line setting. Both J&J and BMS said in briefing documents that many early deaths in the CAR-T arms of their studies happened before patients had received Carvykti or Abecma. BMS wrote that most early deaths were caused by disease progression. Mark Wildgust “This is a really tough group of patients,” Mark Wildgust, VP of global medical affairs for J&J’s oncology division, told Endpoints News ahead of the adcomm. He added that patients in the Carvykti arm also received lower dose intensities of bridging therapies, which were taken as the CAR-Ts were being manufactured. However, J&J noted in its briefing documents that “the extent of this impact is unclear.” “There’s one really important clinical finding from CARTITUDE-4, which is physicians should really be using the optimal bridging therapy,” Wildgust said, referring to Carvykti’s Phase III study in multiple myeloma patients who received one to three prior lines of therapy. Lola Fashoyin-Aje, a director at CBER’s Office of Clinical Evaluation, said during the adcomm that “we may never know why there was this increase [in] early deaths in the cilta-cel arm compared to the control arm.… #lucidquest #genetherapy #celltherapy

Simply devastating. On the 25th, Sanofi disclosed that the AMETHIST trial for venglustat is discontinued. It’s heartbreaking because so many families, including my own were waiting years on end or all the steps to be executed for a possible approved treatment. GM1 patients participated in the basket trial arm of the trial. The primary arm of the trial was for late-onset GM2. It’s devastating to see this FOURTH trial closure for GM1 studies in just a few years. While there was a tremendous burst of trials for GM1 starting in 2019, two companies went bankrupt and two companies shelved or discontinued their work. After many years in patient advocacy, It is abundantly clear that this problem of delivering a treatment involves far more than science alone. Meaningful engagement of the patients is critical at every step to ensure that the patients are truly being served and that the trial designs will be able to demonstrate a measurable effect. Collaboration and data sharing in rare diseases are critical. I’m grateful to all those whose hard work brought all the trials to fruition. I truly believe that it is possible to treat GM1, but it is also a fact that the regulatory pathways are not designed to work for ultra rare diseases, particularly neurodegenerative diseases. The newborn screening system is antiquated and prevents diagnoses early enough to qualify for the trials, making finding candidates extremely difficult. Our work at the Cure GM1 Foundation continues in the ever evolving and changing landscape. If anything, advocacy is most important when there are setbacks. It’s time to recharge, reset, and re-energize our efforts.