Sanofi’s Amlitelimab Demonstrates Promise in Atopic Dermatitis
Following the positive findings from the COAST 1 trial (NCT06130566) reported in September 2025, two additional global Phase 3 studies, SHORE (NCT06224348) and COAST 2 (NCT06181435), have further strengthened the clinical evidence supporting amlitelimab in patients aged 12 years and older with moderate-to-severe atopic dermatitis. Amlitelimab is a fully human monoclonal antibody that selectively inhibits OX40-ligand (OX40L) without depleting T cells. Across both studies, amlitelimab demonstrated favorable tolerability, with a safety profile consistent with previously reported results.
“These findings underscore the validity of amlitelimab’s differentiated mechanism of action, blocking OX40-ligand without T-cell depletion, and its potential to progressively rebalance immune function,” said Houman Ashrafian, Executive Vice President and Head of Research & Development at Sanofi. “The cumulative data to date reinforce our confidence in amlitelimab’s ability to provide Q12W dosing from treatment initiation while delivering increasing efficacy through Week 52. We look forward to presenting additional outcomes, including long-term data, as we advance toward global regulatory filings.”
In both the SHORE and COAST 2 Phase 3 trials, primary efficacy assessments were conducted at Week 24 in adolescents and adults with moderate-to-severe AD receiving amlitelimab either every four weeks (Q4W) or every 12 weeks (Q12W). In the US and US reference countries, the primary endpoint was the proportion of patients achieving a validated Investigator Global Assessment for AD (vIGA-AD) score of 0 (clear) or 1 (almost clear) with≥2-point improvement from baseline, analyzed using a non-responder imputation approach (US estimand). In the EU, EU reference countries, and Japan, the co-primary endpoints included both the proportion of patients achieving vIGA-AD 0/1 with a ≥2-point reduction from baseline and the proportion achieving at least a 75% improvement in Eczema Area and Severity Index (EASI-75), analyzed using a treatment-policy estimand (EU estimand).
Corcept Reports Positive Phase 3 ROSELLA Results with Overall Survival Endpoint Met in Platinum-Resistant Ovarian Cancer
Corcept Therapeutics Incorporated announced positive results from the ROSELLA pivotal Phase 3 study evaluating relacorilant in combination with nab-paclitaxel in patients with platinum-resistant ovarian cancer. The trial successfully achieved its primary endpoint of overall survival (OS).
In the ROSELLA study, the addition of relacorilant to nab-paclitaxel reduced the risk of death by 35% compared with nab-paclitaxel alone (hazard ratio: 0.65; p=0.0004). Median overall survival reached 16.0 months in the combination arm versus 11.9 months in the chemotherapy-only arm, representing an improvement of 4.1 months. The relacorilant and nab-paclitaxel regimen was generally well tolerated and aligned with the drug’s established safety profile. The incidence, nature, and severity of adverse events were comparable between the combination therapy and monotherapy groups, indicating that relacorilant delivered clinical benefit without adding to patients’ safety burden.
Previously, Corcept reported that ROSELLA also met its primary endpoint for progression-free survival as determined by blinded independent central review (PFS-BICR). Patients treated with relacorilant plus nab-paclitaxel demonstrated a 30% reduction in the risk of disease progression compared with those receiving nab-paclitaxel alone (hazard ratio: 0.70; p=0.008). These data were initially presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and published concurrently in The Lancet. Full study results will be shared at a forthcoming scientific conference.
CORXEL Strengthens Cardiometabolic Pipeline with $287M Series D1 Financing, Highlighting Oral GLP-1 Program
Corxel Pharmaceuticals Limited has announced the successful close of its Series D1 financing round, securing up to $287 million. The funds will primarily support the continued development of CX11, a differentiated oral small-molecule GLP-1 receptor agonist for the treatment of obesity and overweight. CX11 is currently being assessed in a Phase 2 clinical study led by CORXEL in the United States and a Phase 3 trial conducted by Vincentage in China. Additional proceeds will be allocated to advance other cardiometabolic programs, including those targeting acute ischemic stroke and hypertension.
The financing round attracted a strong group of new investors, including SR One, TCGX, RA Capital Management, HBM Healthcare Investments, SymBiosis, Adage Capital Management, Invus, SilverArc Capital, and other participants. Existing shareholders RTW Investments and Hengdian Group Capital also contributed, reflecting continued confidence in CX11’s differentiated clinical potential, CORXEL’s execution capabilities, and its long-term strategic vision.
Capital raised in this round will be used to:
- Progress CX11 through its ongoing Phase 2 clinical trial in the U.S. for obesity and overweight, initiate a planned global Phase 2 study in Type 2 Diabetes Mellitus (T2DM), and support early preparations for Phase 3 development;
- Advance the company’s broader pipeline through clinical development; and
- Further enhance CORXEL’s global development and operational infrastructure to enable multiregional clinical programs.
“This investment marks one of the most important milestones in CORXEL’s journey since its inception. It strengthens our ability to accelerate the global development of best-in-class therapies for cardiometabolic diseases while positioning the company for its next phase of transformative growth,” said Sandy Mou, Board Executive Director and Chief Executive Officer of CORXEL. “We deeply appreciate the trust shown by both our existing and new investors, and our determination to build CORXEL into a leading global biopharmaceutical company has never been stronger.”
IntraBio Announces Positive Pivotal Data Supporting Levacetylleucine in Ataxia-Telangiectasia
IntraBio Inc. announced positive topline findings from its pivotal Phase III IB1001-303 clinical study titled “Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Randomized, Placebo-Controlled, Double-Blind, Crossover Study” (NCT06673056). The trial evaluated N-acetyl-L-leucine (levacetylleucine) in both pediatric and adult patients with Ataxia-Telangiectasia.
The study’s primary endpoint assessed the impact of levacetylleucine on the Scale for the Assessment and Rating of Ataxia (SARA) versus placebo after 12 weeks of treatment. Results showed a statistically significant and clinically meaningful improvement, with a 1.88-point greater reduction in SARA score compared with placebo (-1.92 for levacetylleucine vs. -0.14 for placebo; p<0.001).
Secondary endpoints were also successfully achieved, demonstrating significant improvements on the International Cooperative Ataxia Rating Scale (ICARS) (-4.22 with levacetylleucine vs. -1.69 with placebo; p=0.003) and the Investigator’s Clinical Global Impression of Improvement (CGI-I) (-0.6 vs. -0.2, respectively; p=0.02). Levacetylleucine was found to be safe and well-tolerated, with no drug-related serious adverse events reported, in line with its known safety profile.
In light of these positive results, IntraBio intends to promptly proceed with regulatory filings with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and other regulatory agencies worldwide.
Otsuka’s Centanafadine NDA Accepted by FDA With Priority Review for ADHD Across Age Groups
Otsuka Pharmaceutical Co., Ltd., together with Otsuka Pharmaceutical Development & Commercialization, Inc., has announced that the FDA has granted priority review and accepted the New Drug Application (NDA) for centanafadine. Centanafadine is an investigational, once-daily extended-release oral capsule and a first-in-class norepinephrine, dopamine, and serotonin reuptake inhibitor (NDSRI) being developed for the treatment of attention-deficit hyperactivity disorder (ADHD) in pediatric, adolescent, and adult patients. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of July 24, 2026.
The NDA submission is based on data from four pivotal Phase 3 clinical studies that assessed the efficacy and safety of centanafadine across different age groups. In these trials, centanafadine produced statistically significant and clinically meaningful reductions in ADHD symptoms compared with placebo. Symptom improvement was evaluated using the ADHD Rating Scale-5 (ADHD-RS-5) in children and adolescents and the ADHD Investigator Symptom Rating Scale (AISRS) in adults. Across studies, centanafadine demonstrated a favorable tolerability profile. The most frequently reported adverse events in pediatric and adolescent patients included decreased appetite, nausea, rash, fatigue, abdominal pain, and somnolence, while adults most commonly experienced decreased appetite and headache.
“ADHD presents with considerable variability among individuals, underscoring the need for a range of treatment options,” said John Kraus, M.D., Ph.D., executive vice president and chief medical officer at Otsuka Pharmaceutical Development & Commercialization, Inc. “The FDA’s acceptance and priority review of the centanafadine NDA represents a meaningful step toward introducing a novel therapeutic approach for people living with ADHD. If approved, centanafadine would be the first NDSRI available, offering a new option for comprehensive symptom management. We are deeply thankful to the patients, caregivers, and clinical investigators whose contributions made this progress possible.”
While ADHD has traditionally been viewed as a pediatric condition, growing evidence indicates that a substantial proportion of individuals diagnosed in childhood continue to experience symptoms into adulthood, often resulting in significant functional impairment.
