Ulefnersen, an RNA-targeted therapy being developed by Ionis Pharmaceuticals and Otsuka Pharmaceutical, improved measurements of functional impairment and survival in patients with a rare, genetic ALS subtype in a Phase 3 trial. The partners plan to approach the FDA with the results to determine potential pathways for approval.

Original title: “Ionis-Otsuka offer 'groundbreaking' improvement in function and survival for genetic type of ALS”

New option for patients with systemic lupus erythematosus

Original title: “Roche welcomes MHRA approval of Gazyvaro”

Albany New York September 22 2026 Las Vegas Nevada United States As per DelveInsight s assessment globally Multiple Sclerosis pipeline constitutes 75 key companies continuously working towards developing 80 Multiple Sclerosis treatment therapies analysis of Clinical Trials Therapies Mechanism of ... Press release - ABNewswire - Multiple Sclerosis Pipeline 2026: FDA Updates, Therapy Innovations, and Clinical Trial Landscape Analysis by DelveInsight | Biogen, Immune Response BioPharma, Celgene, Novartis, Sanofi, Actelion, Baye - published on openPR.com While there is no cure, a wide range of disease-modifying therapies can help reduce relapse rates, slow disease progression, and improve quality of life. Explore the latest Multiple Sclerosis pipeline insights 2026, including emerging therapies, clinical trials, and market opportunities. * Multiple Sclerosis Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. * Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Multiple Sclerosis market. The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc. (Albany, New York) - September 22, 2026: The Key Hyperphosphatemia Companies in the market include - Ardelyx, Sanofi, Takeda Pharma, Unicycive Therapeutics, Taisho Pharma, Alebund Pharmaceuticals, FRESENIUS MEDICAL CARE RENAL PHARMACEUTICALS, Astellas pharma, Shield Therapeutics, Kyowa Kirin Co., Ltd., Shanghai Alebund Pharma, Sanofi, Bayer, Keryx Biopharma, Kissei Pharma, Taisho Pharma, Astellas Pharma Inc, Novartis, Sanofi, Mitsubishi Tanabe Pharma, and others. DelveInsight's "Hyperphosphatemia Market Insights, Epidemiology, and Market Forecast-2036 report offers an ... (Albany, New York) -

Original title: “Multiple Sclerosis Pipeline 2026: FDA Updates, Therapy Innovations, and Clinical Trial Landscape Analysis by DelveInsight | Biogen, Immune Response BioPharma, Celgene, Novartis, Sanofi, Actelion, Baye”

Dazodalibep, acquired in Amgen’s big buyout of Horizon Therapeutics, succeeded in the first of two late-stage trials against the tough-to-treat autoimmune disorder.

Original title: “Amgen reports a Phase 3 win with Sjögren’s drug candidate”

Citing the urgent unmet need for patients with advanced biliary tract cancer, Compass Therapeutics will continue to seek an approval path for its drug after the FDA recommended the biotech conduct a new clinical study.

Original title: “Compass vows to press on with FDA application despite new trial request”

Ulefnersen met its primary end point in the phase 3 FUSION trial, providing the first placebo-controlled evidence for a genetically targeted therapy in FUS-ALS. “We are profoundly grateful to the patients, carers, families, investigators and study teams whose commitment made this study possible,” Kunal Gandhi, MD, MPH, Vice President, Head of Global Clinical Development, at Otsuka, told NeurologyLive®. “These findings represent a major advancement for people living with FUS-ALS, a rare and often rapidly progressive genetic form of ALS. There are currently no approved therapies specifically targeting the underlying genetic cause of FUS-ALS, underscoring the significant unmet need for effective treatment options. As the first FUS-ALS clinical trial to meet its primary endpoint, FUSION provides compelling evidence that a targeted genetic approach may help alter the course of disease.” Otsuka Pharmaceutical and Ionis Pharmaceuticals announced positive topline results from the phase 3 FUSION trial (NCT04768972) evaluating ulefnersen, an investigational antisense oligonucleotide (ASO), in patients with a rare genetic subtype of amyotrophic lateral sclerosis (ALS) driven by mutations in the fused in sarcoma (FUS) gene, known as FUS-ALS.1 · According to the companies, the trial met its primary end point, showing a statistically significant improvement on a composite measure of functional decline and survival, marking the first placebo-controlled clinical evidence for an agent designed to target the underlying genetic cause of the disease. “Today’s Phase 3 FUSION topline results mark a major milestone for people living with FUS-ALS, re-shaping what is possible for a community that has long faced this devastating disease with limited treatment options,” John Kraus, MD, PhD, executive vice president and chief medical officer at Otsuka, said in a statement.1 “As the first FUS-ALS clinical trial to meet its primary endpoint, FUSION provides compelling evidence that a targeted genetic approach may help alter the course of disease.” FUS-ALS accounts for an estimated 0.6% of ALS cases overall but a disproportionate share of pediatric and juvenile-onset disease, with FUS variants implicated in an estimated 43% to 52% of cases in that population.2 The disease typically presents with rapid progression, and early-onset cases can progress to respiratory failure and death in 1 to 2 years of symptom onset. No therapy targeting the underlying FUS mutation has previously been approved.

Original title: “Ulefnersen Meets Primary End Point in Phase 3 FUSION Trial for FUS-ALS | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

Though barzolvolimab displayed what one analyst described as the best efficacy results to date against chronic skin hives, treatment was also associated with two life-threatening anaphylaxis cases.

Original title: “Celldex sinks as safety concerns cloud immune drug results”

While Vertex is laser-focused on an upcoming FDA decision for povetacicept in immunoglobulin A nephropathy, the APOL1 inhibitor inaxaplin has demonstrated potential “best-in-class” data in a different kidney disease.

Original title: “Vertex opens up larger kidney disease opportunity with mid-stage readout”

Regulator supports earlier multiple myeloma treatment option

Original title: “CHMP backs expanded use of teclistamab”

The Dermatomyositis clinical trial analysis report delivers important insights into ongoing research of 10 pipeline Dermatomyositis drugs clinical strategies upcoming therapeutics and commercial analysis Albany US September 21 2026 DelveInsight s Dermatomyositis Pipeline Insight 2026 report ... The Dermatomyositis clinical trial analysis report delivers important insights into ongoing research of 10 pipeline Dermatomyositis drugs clinical strategies upcoming therapeutics and commercial analysis Albany US September 21 2026 DelveInsight s Dermatomyositis Pipeline Insight 2026 report provides comprehensive insights about ... Press release - ABNewswire - Dermatomyositis Pipeline Gains Momentum: 10+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight - published on openPR.com Image: http://www.abnewswire.com/pressreleases/wp-content/uploads/2026/09/2026 The Buerger's Disease clinical trial analysis report delivers important insights into ongoing research of 3+ pipeline Buerger's Disease drugs, clinical strategies, upcoming therapeutics, and commercial analysis. Projected Growth and Market Size Outlook for Dermatomyositis Treatment The dermatomyositis treatment market is anticipated to experience substantial expansion, reaching a valuation of ... Dermatomyositis is a rare, chronic autoimmune condition characterized by muscle inflammation and distinctive skin rashes. The disease impacts both adults and children, with complications that include interstitial lung disease and increased cancer risk.

Original title: “Dermatomyositis Pipeline Gains Momentum: 10+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight”

By delivering the genetic instructions ... T-cell-engaging therapeutics with the potential convenience and durability of a one-time treatment. VNX-101 has received U.S. FDA Fast Track, Orphan Drug, and Rare Pediatric Disease designations. ... The SENTRY-CD19 Clinical Trial [NCT06533579] ... By delivering the genetic instructions for therapeutic protein production directly in vivo, Vironexis is developing an approach intended to combine the potency of T-cell-engaging therapeutics with the potential convenience and durability of a one-time treatment. VNX-101 has received U.S. FDA Fast Track, Orphan Drug, and Rare Pediatric Disease designations. ... The SENTRY-CD19 Clinical Trial [NCT06533579] is a Phase 1/2, first-in-human, open-label, dose-escalating study designed to assess the safety and efficacy of VNX-101 in patients with relapsed or refractory CD19 positive hematologic malignancies. Visit the clinicaltrials.gov listing for more information on the trial. ... Vironexis Biotherapeutics is a clinical-stage biotechnology company developing in vivo therapies designed to enable the body to produce therapeutic proteins following a single administration. Our platform is being developed across oncology and other serious diseases with the goal of transforming medicines that traditionally require repeated administration into durable, potentially one-time therapies. Mr. Varma continued, “Further, we believe VNX-101 has achieved an important first for the field: demonstrating that a systemically delivered, one-time in vivo therapy can translate into clinical antitumor activity in patients with blood cancer and extramedullary disease.” “These three patients have tried multiple lines of therapy and, sadly, exhausted available treatment options. We believe the depth, consistency, and emerging durability of these responses provide important clinical validation of our in vivo therapeutic protein platform, and compelling proof of concept for its potential to transform the body into a meaningful, durable source of highly active cancer therapeutics.”

Original title: “Vironexis Announces Complete and Durable Responses in Relapsed/Refractory ALL Patients Treated with VNX-101 - BioSpace”

According to IMARC Group the global virtual clinical trials market size reached USD 252 5 Million in 2025 and is projected to reach USD 379 0 Million by 2034 expanding at a CAGR of 4 48 during 2026 2034 This ... Press release - IMARC Group - Virtual Clinical Trials Market Size, Share, In-Depth Analysis, Trends and Forecast 2026-2034 - published on openPR.com Advances in targeted therapies, immunotherapies, and precision medicine are reshaping cancer care and pushing sponsors toward trial designs that can accommodate more individualized treatment protocols. The growing understanding of tumor genetics is also expanding demand for niche trials targeting rare and genetically defined cancers, where virtual recruitment models help overcome the patient-scarcity challenges that limit traditional site-based enrollment. This steady growth reflects the deepening integration of advanced imaging technology into veterinary practice, driven by rising pet humanization, escalating chronic disease burden in animals, and the rapid proliferation of specialized veterinary hospitals and clinics globally.

Original title: “Virtual Clinical Trials Market Size, Share, In-Depth Analysis, Trends and Forecast 2026-2034”

Delivering therapeutics directly to tumors, as in the case of Replimune’s recently approved Tudriqev and Amgen’s Imlygic, holds advantages, especially when cancer is caught early, but challenges remain before the method becomes a mainstay in treatment.

Original title: “Citing Replimune approval, experts say localized cancer meds could redefine care”

Single-patient antisense oligonucleotide for rare CHCHD10 mutation shows functional gains over 12 months Single-patient antisense oligonucleotide for rare CHCHD10 mutation shows functional gains over 12 months ... When a personalized RNA therapy was administered to a patient with amyotrophic lateral sclerosis (ALS) caused by a genetic mutation, the patient’s physical function improved. A joint research team from the Department of Molecular Medicine at Mayo Clinic in Rochester, USA, and the Department of Genetics at Orlando Health, USA, developed and administered an individualized antisense oligonucleotide (ASO) therapy to treat a single clinical trial participant, and reported that the patient showed significant symptom improvement one year later. The results were published on the 14th in the international journal Med. ALS is also known as Lou Gehrig’s disease. The research team stated, “We have shown that it is possible to develop a personalized ASO therapy for CHCHD10-related ALS,” adding that this provides “evidence suggesting the potential for therapeutic benefit in the clinic.” ... Copyright ⓒ DongA Science. All rights reserved. ... An ALS patient, unable to move the muscles required for speech, has successfully translated their thoughts into words on a digital device using a tech · New Treatment Strategy Discovered for Lou Gehrig's Disease, Which Paralyzes Muscles The case suggests personalized RNA therapies may be feasible even for rare hereditary forms of ALS.

Original title: “Mayo Clinic develops personalized RNA therapy that improves ALS motor scores - DongA Science”