Ted Benson’s Post

THOUSAND OAKS, Calif., Nov. 7, 2023. Amgen (NASDAQ:AMGN) today announced new data from its Phase 2 study evaluating dazodalibep, an investigational medicine, for the treatment of Sjögren’s. These results will be featured in presentations at the American College of Rheumatology (ACR) Convergence 2023, Nov. 10-15, in San Diego. Findings from the study demonstrate that dazodalibep may improve both the systemic and symptomatic disease burden of two different patient populations. The Phase 2 study of dazodalibep, a CD40 ligand antagonist in clinical development, was a randomized, double-blind, placebo-controlled crossover study evaluating two Sjögren’s populations: patients with moderate to severe systemic disease activity and those with moderate to severe symptomatology despite lacking additional organ involvement. In May 2023, presentations at the 2023 EULAR Congress reported that at Day 169, both patient groups treated with dazodalibep achieved the study’s primary endpoint. The presentations at ACR highlight results from the crossover period, when at Day 169, patients initially treated with dazodalibep transitioned to placebo, and patients that initially received placebo switched to dazodalibep. After administration of the last dose, patients were followed for an additional 12 weeks for safety. “To date, there are no FDA-approved disease-modifying treatments for Sjögren’s and the positive results from the Phase 2 trial provide evidence that dazodalibep may address the underlying causes of the disease by reducing systemic disease activity and improving the debilitating symptoms such as dryness and fatigue,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. The company is advancing a Phase 3 trial evaluating the benefit of dazodalibep in Sjögren’s. Patients with Moderate to Severe Systemic Disease ActivityThe first patient population included patients with moderate to severe systemic disease activity as defined by a EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI) score ≥5. Key findings include: Patients who transitioned from placebo to dazodalibep experienced an improvement in their disease activity from Day 169 (4.1-point reduction in total ESSDAI score) to Day 365 (6.3-point reduction). At Day 365, patients who transitioned to dazodalibep also showed greater improvements in ESSDAI response rate (3- to 4-point reduction), EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI) score and fatigue compared to those who transitioned to placebo. Dazodalibep was generally safe and well tolerated. Patients with Moderate to Severe SymptomatologyThe second patient population studied included those with moderate to severe symptomatology including dryness, fatigue and pain despite lacking additional organ involvement as defined by an ESSPRI score ≥5 and an ESSDAI score of <5. Key findings include: Patients who transitioned from placebo to dazodalibep experienced

Ultomiris (ravulizumab-cwvz) has been approved in the United States (US) as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). The approval by the US Food and Drug Administration (FDA) was based on positive results from the CHAMPION-NMOSD Phase III trial, which were published in the Annals of Neurology. In the trial, Ultomiris was compared to an external placebo arm from the pivotal Soliris PREVENT clinical trial. Ultomiris met the primary endpoint of time to first on-trial relapse as confirmed by an independent adjudication committee. Zero relapses were observed among Ultomiris patients with a median treatment duration of 73 weeks (relapse risk reduction: 98.6%, hazard ratio (95% CI): 0.014 (0.000, 0.103), p<0.0001). NMOSD is a rare and debilitating autoimmune disease that affects the central nervous system (CNS), including the spine and optic nerves. Most people living with NMOSD experience unpredictable relapses, characterised by a new onset of neurologic symptoms or worsening of existing neurologic symptoms, which tend to be severe and recurrent and may result in permanent disability. The diagnosed prevalence of adults with NMOSD in the US is estimated at approximately 6,000. Sean J. Pittock, MD, Director of Mayo Clinic's Center for Multiple Sclerosis and Autoimmune Neurology and of Mayo's Neuroimmunology Laboratory and lead primary investigator in the CHAMPION-NMOSD trial, said: “C5 inhibition has been proven to offer efficacy in reducing the risk of NMOSD relapses by blocking the complement system, a part of the immune system, from attacking healthy cells in the spinal cord, optic nerve and brain. With today’s FDA approval, patients now have the option of a long-acting C5 inhibitor treatment that showed zero relapses in the pivotal CHAMPION-NMOSD trial, supporting the primary goal of relapse prevention in treating NMOSD.” Marc Dunoyer, Chief Executive Officer, Alexion, said: “Alexion has been at the forefront of innovation in NMOSD, striving to offer patients a future without fear of life-altering or even fatal relapses. Building on the established efficacy of C5 inhibition for people living with AQP4 Ab+ NMOSD, we are proud to deliver a transformative, long-acting treatment option that has the potential to eliminate relapses with a convenient dosing schedule every eight weeks. We are grateful to the NMOSD community for their ongoing collaboration and input, which enables us to advance science for rare diseases.” Ultomiris is also approved for certain adults with NMOSD in Japan and the European Union (EU). Regulatory reviews are ongoing in additional countries. #neurology #neuroimmunology #raredisease #NMOSD #Ultomiris #Alexion #AstraZeneca #WhatScienceCanDo 👉 Ultomiris approved in the US for the treatment of adults with neuromyelitis optica spectrum disorder (NMOSD) (astrazeneca.com)

Sobi and Apellis Pharmaceuticals have shared positive late-stage results for their targeted C3 therapy in rare kidney disease patients. The phase 3 VALIANT trial has been evaluating the drug, pegcetacoplan, in patients with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). There are currently no treatments that target the underlying cause of either disease and approximately 50% of patients experience kidney failure within five to ten years of diagnosis. Pegcetacoplan is designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, and already holds approvals to treat the rare blood disorder paroxysmal nocturnal haemoglobinuria under the brand names Empaveli and Aspaveli. VALIANT met its primary endpoint, demonstrating a 68% proteinuria (excess of protein in the urine) reduction in patients treated with pegcetacoplan compared to placebo, both in addition to background therapy, at week 26. Results were consistent among adult and adolescent patients, and those with native and post-transplant kidneys, the companies noted. Pegcetacoplan also showed statistical significance on secondary endpoints including a composite measure combining proteinuria reduction and estimated glomerular filtration rate stabilisation. VALIANT lead principal investigator Carla Nester, University of Iowa Stead Family Children’s Hospital, said: “Currently, many patients living with these rare diseases will eventually require a kidney transplant or lifelong dialysis, so there is an urgent need for a treatment that targets the underlying cause of these diseases. This positive data is a major advance for the rare kidney disease community.” All patients who have completed the VALIANT study have now been enrolled into the VALE long-term extension study. Read more: https://lnkd.in/eQdjKGTR Stay in touch with all the leading stories, events and opportunities by subscribing to my weekly LinkedIn Newsletter here:  https://bit.ly/3KQs0mD

Innorna Announces U.S. FDA Rare Pediatric Disease Designation Granted to IN022 for the Treatment of Homocystinuria HONG KONG, SHENZHEN, NANJING, CHINA, July 08, 2024--Innorna, a clinical-stage biotech company pioneering its proprietary lipid nanoparticle (LNP) technology to develop novel RNA therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational therapy IN022 for the treatment of classic homocystinuria (HCU), a serious or life-threatening genetic disease. This RPDD will greatly facilitate IN022 clinical development and bring this potential therapy to patients as quickly as possible. The RPDD also means that FDA may award a Priority Review Voucher upon the approval of the IN022 product. About HCU and IN022 HCU is a rare autosomal recessive inherited disorder of sulfur amino acid metabolism primarily caused by deficiency of cystathionine beta synthase (CBS). HCU patients have a wide spectrum of age (older than three years) on presentation, with multisystemic clinical complications, particularly skeletal and connective tissue defects, osteoporosis, dislocated optic lenses, learning difficulties, and developmental delay or thromboembolism. Currently, there is no approved therapeutic drug for this disease. IN022 is designed to address the root cause of HCU resulting from deficiency in CBS. It is expected to restore the function of CBS enzyme, thereby normalizing homocysteine metabolism and potentially ameliorating symptoms in HCU patients.

Zydus Lifesciences Completes Enrolment For EPICS III Phase 2b/3 Trial Evaluating Saroglitazar Mg For Primary Biliary Cholangitis Zydus Group, a discovery-driven, global lifesciences company, has announced that it has completed enrolment of Phase 2b/3 EPICS III trial of Saroglitazar Mg in patients with Primary Biliary Cholangitis (PBC). PBC is a rare, progressive autoimmune disease which gradually destroys the bile ducts, resulting in an accumulation of bile in the liver which can result in fibrosis, cirrhosis, the need for liver transplantation or death. PBC disproportionately affects women, with 1 in 1,000 women over the age of 40 being afflicted, 9 times the rate for men. PBC is characterized by increases in biochemical markers, especially alkaline phosphatase (ALP), bilirubin and liver transaminases. Clinical symptoms include pruritus (itching) and fatigue, both of which can be severe. PBC is a life-long condition and only medications can be used to manage and slow its progression. Saroglitazar Mg is a potent and selective peroxisome proliferator-activated receptor alpha and gamma dual agonist. Results of phase 2, prospective multicentre randomized double-blind, placebo-controlled study to evaluate the safety, tolerability and efficacy of Saroglitazar Mg in patients with Primary Biliary Cholangitis (EPICS) was presented earlier at the Liver Meeting 2020, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD) [ClinicalTrials.gov Identifier: NCT03112681], and has been published in the ‘Journal of Hepatology’. Overall Principal Investigator for Saroglitazar Mg global development programme, Professor Chalasani Naga, David W. Crabb Chair in Gastroenterology and Hepatology, Indiana University School of Medicine, Indianapolis, USA lauded the Zydus team and investigators across 3 countries for achieving this milestone in a record time. The results from this pivotal trial holds potential to greatly improve the treatment choices available for this difficult to treat and rare liver disorder. Saroglitazar Mg is an investigational compound in the USA, and is yet to be approved by the U.S. FDA (USFDA) or European Medicines Agency (EMA). #zyduslifesciences #zyduslifesciencesnews #saroglitazar #primarybiliarycholangitis

AstraZeneca Ultomiris Wins FDA Approval for Rare Autoimmune Disease The FDA on Monday approved the label expansion for AstraZeneca’s complement inhibitor Ultomiris (ravulizumab-cwvz), allowing its use for the treatment of adult neuromyelitis optica spectrum disorder patients positive for anti-AQP4 antibodies. Monday’s approval makes Ultomiris the first and only long-acting C5 complement inhibitor for this indication that provides patients with the possibility of living relapse-free, according to the company’s announcement. Marc Dunoyer, CEO of AstraZeneca’s rare disease unit Alexion, in a statement called Ultomiris a “transformative” treatment option for neuromyelitis optica spectrum disorder (NMOSD) with the “potential to eliminate relapses with a convenient dosing schedule every eight weeks.” Ultomiris’ label expansion is supported by data from the Phase III CHAMPION-NMOSD trial, a global, open-label and multicenter trial with 58 NMOSD patients enrolled, all of whom were confirmed to be positive for anti-AQP4 antibodies and had had at least one attack or relapse in the 12 months before screening. Given the potential long-term consequences of NMOSD relapses, AstraZeneca and Alexion could not ethically include a direct placebo arm into CHAMPION-NMOSD. Instead, the company used data from the placebo arm of the PREVENT trial for its other NMOSD therapy Soliris (eculizumab). Findings from CHAMPION-NMOSD were published in March 2023 in the journal Annals of Neurology. Data showed that Ultomiris met its primary efficacy endpoint, preventing relapse in all 58 treated patients over a median follow-up period of 73.5 weeks, compared to 20 relapses in PREVENT’s placebo arm.

Ceres Brain Therapeutics SAS is proud to announce that the FDA has granted rare Pediatric Designation from our leading asset, CBT101, for the treatment of Creatine Transporter Deficiency Syndrome (CTD). This opens the door to potentially receiving a Priority Review Voucher, a key step in accelerating the development of our innovative treatments. Stay tuned for more updates! We remind you that CTD is an X-linked gene disease resulting in severe intellectual deficiency, autistic syndrome and seizure due to lack of creatine in brain neurons. CBT101, is a prodrug of creatine that delivers creatine to brain neurons by following the nose-to-brain pathway after nasal administration. CBT101 demonstrated particularly strong efficacy in two different mice models of CTD, increasing brain plasticity, improving brain metabolism and restoring cognition. https://lnkd.in/e4ka-7uY

𝐁𝐫𝐢𝐬𝐭𝐨𝐥 𝐌𝐲𝐞𝐫𝐬 𝐒𝐪𝐮𝐢𝐛𝐛 𝐀𝐧𝐧𝐨𝐮𝐧𝐜𝐞𝐬 𝐏𝐨𝐬𝐢𝐭𝐢𝐯𝐞 𝐓𝐨𝐩𝐥𝐢𝐧𝐞 𝐑𝐞𝐬𝐮𝐥𝐭𝐬 𝐟𝐫𝐨𝐦 𝐓𝐰𝐨 𝐏𝐢𝐯𝐨𝐭𝐚𝐥 𝐏𝐡𝐚𝐬𝐞 𝟑 𝐓𝐫𝐢𝐚𝐥𝐬 𝐄𝐯𝐚𝐥𝐮𝐚𝐭𝐢𝐧𝐠 𝐒𝐨𝐭𝐲𝐤𝐭𝐮 (𝐝𝐞𝐮𝐜𝐫𝐚𝐯𝐚𝐜𝐢𝐭𝐢𝐧𝐢𝐛) 𝐢𝐧 𝐀𝐝𝐮𝐥𝐭𝐬 𝐰𝐢𝐭𝐡 𝐏𝐬𝐨𝐫𝐢𝐚𝐭𝐢𝐜 𝐀𝐫𝐭𝐡𝐫𝐢𝐭𝐢𝐬 Bristol Myers Squibb (NYSE: BMY) Bristol Myers Squibb today announced results from POETYK PsA-1 (IM011-054) and POETYK PsA-2 (IM011-055), the pivotal Phase 3 trials evaluating the efficacy and safety of Sotyktu (deucravacitinib) in adults with active psoriatic arthritis (PsA). Both trials met their primary endpoint, with a significantly greater proportion of Sotyktu-treated patients achieving ACR20 response (at least a 20 percent improvement in signs and symptoms of disease) after 16 weeks of treatment compared with placebo. $BMY announced topline results from two pivotal Phase 3 studies for an investigational treatment in adults with #PsoriaticArthritis. Additionally, the POETYK PsA-1 and POETYK PsA-2 trials met important secondary endpoints across PsA disease activity at Week 16. The overall safety profile of Sotyktu through 16 weeks of treatment in the POETYK PsA-1 and POETYK PsA-2 trials was consistent with the established safety profile of Sotyktu observed in a Phase 2 PsA clinical trial and Phase 3 moderate-to-severe plaque psoriasis clinical trials. "Psoriatic arthritis is a heterogeneous disease that causes a range of symptoms, including joint pain and swelling, as well as psoriatic skin lesions. Despite available therapies, rheumatologists continue to express a need for a safe and effective oral treatment," said Roland Chen, MD, senior vice president and head, Immunology, Cardiovascular and Neuroscience development, Bristol Myers Squibb. "These POETYK PsA-1 and POETYK PsA-2 findings demonstrate that oral Sotyktu has the potential to be the first TYK2 inhibitor for people living with psoriatic arthritis and reinforce the established efficacy and safety profile of Sotyktu. We are encouraged by the positive data across both Phase 3 trials and look forward to discussing the results with health authorities." Bristol Myers Squibb will work with key investigators to present detailed results at upcoming medical congresses. These topline results represent the first Phase 3 clinical trials for Sotyktu in a rheumatic condition. Sotyktu is approved in numerous countries around the world for the treatment of adults with moderate-to-severe plaque psoriasis. Bristol Myers Squibb thanks the patients, investigators, and clinical trial sites who participated in these clinical trials. #PsoriaticArthritis #PsA #Sotyktu #Deucravacitinib #Immunology #Rheumatology #BristolMyersSquibb #Phase3Trials #AutoimmuneDisease #Psoriasis #AR20Response #TYK2Inhibitor

The United States Food and Drug Administration (US FDA) has extended the rare paediatric disease designation (RPDD) of the novel Race Oncology Ltd (ASX:RAC, OTC:RAONF) drug RC220 bisantrene for the treatment of paediatric subtypes of Acute Myeloid Leukaemia (AML). This designation was previously granted to RC110 bisantrene in 2018. RPDD is awarded for new treatments of serious or life-threatening diseases affecting fewer than 200,000 people in the US, primarily those under 18 years of age. Around 70% of rare diseases are exclusively paediatric in onset, with 95% having no approved treatments. The designation makes RC220 eligible for a priority review voucher (PRV) upon marketing approval, which can be transferred or sold. More at #Proactive #ProactiveInvestors #ASX #RAC #Oncology #Cancer #Biotech http://ow.ly/RXlG105xcvz

You are subscribed to Oncology (Cancer) / Hematologic Malignancies Approval. This information has recently been updated, and is now available. FDA grants accelerated approval to selpercatinib for pediatric patients two years and older with RET-altered metastatic thyroid cancer or solid tumors On May 29, 2024, the Food and Drug Administration granted accelerated approval to selpercatinib (Retevmo, Eli Lilly and Company) for pediatric patients two years of age and older with the following: advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy; advanced or metastatic thyroid cancer with a RET gene fusion, as detected by an FDA-approved test, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate); and locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options. #fda #approval #pediatric #oncology https://lnkd.in/gH5TdUVJ