RECORDATI | MedPath

FDA Expands Osilodrostat (Isturisa) Indication for Broader Cushing's Syndrome Treatment

a month ago

• The FDA has approved an expanded indication for osilodrostat (Isturisa) to treat endogenous hypercortisolemia in adults with Cushing's syndrome for whom surgery is not an option or has not been curative. • This regulatory expansion broadens treatment beyond the previous indication limited to Cushing's disease (a subtype of Cushing's syndrome), addressing significant unmet needs for patients with other forms of the syndrome. • Clinical evidence supporting the approval includes data from the LINC studies involving over 350 patients, demonstrating osilodrostat's ability to rapidly normalize cortisol levels and maintain this effect with a manageable safety profile.

Bladder-Preserving Treatment Shows Promise: Radiation Plus Dual Immunotherapy Achieves 93% Complete Response in MIBC

3 months ago

• A novel combination of radiation therapy with durvalumab and tremelimumab immunotherapy achieved a 93% complete response rate in muscle-invasive bladder cancer patients, offering a promising bladder-preserving alternative. • The IMMUNOPRESERVE trial demonstrated strong efficacy with 30 out of 32 patients maintaining their bladders after a median follow-up of 27 months, with 84% two-year overall survival rate. • The treatment regimen showed manageable safety profile with 31% grade 3-4 adverse events, presenting a potential alternative to radical cystectomy for eligible patients.

Evorpacept Shows Promise in HER2-Positive Gastric Cancer: Updated ASPEN-06 Data

4 months ago

• ALX Oncology's ASPEN-06 Phase 2 trial evaluates evorpacept, a CD47-blocker, in HER2-positive advanced gastric cancer patients who have been previously treated. • Updated data from the ASPEN-06 trial were presented at the 2025 ASCO Gastrointestinal Cancers Symposium (ASCO GI). • The virtual event hosted by ALX Oncology on January 23, reviewed the ASPEN-06 data, focusing on evorpacept's potential in immuno-oncology. • The ASPEN-06 trial is a phase 2/3 study of evorpacept in patients with HER2-overexpressing gastric/gastroesophageal cancer.

Effective Sponsor Oversight Critical for CRO Partnerships in Clinical Trials, Industry Leaders Emphasize

6 months ago

• Clinical trial sponsors and CROs must establish optimal oversight balance, with experts warning that both excessive and insufficient monitoring can impede trial success and efficiency. • Industry leaders at Veeva's Clinical Operations Outsourcing 2024 Forum stress the importance of early collaboration agreements and transparent communication between sponsors and CROs. • As clinical trials become increasingly complex, particularly in specialized therapies, the need for efficient sponsor oversight becomes more crucial for ensuring trial quality and regulatory compliance.

Roche to Acquire Poseida Therapeutics; Merck, AstraZeneca, and Novartis Report Pipeline Success

6 months ago

• Roche will acquire Poseida Therapeutics for $1.0 billion, expanding its CAR-T therapy portfolio, targeting hematological malignancies, solid tumors, and autoimmune diseases. • Merck's Winrevair phase III study met its primary endpoint, showing a statistically significant reduction in morbidity or mortality events in advanced pulmonary arterial hypertension. • AstraZeneca's Truqap, combined with Zytiga and ADT, significantly improved radiographic progression-free survival in PTEN-deficient metastatic hormone-sensitive prostate cancer. • Novartis received European Commission approval for Kisqali to treat HR+/HER2- early breast cancer patients at high risk of disease recurrence, based on the NATALEE phase III trial.

Sanofi's Q3 Sales Surge 15.7% Driven by Vaccines and Dupixent; 2024 EPS Guidance Raised

7 months ago

• Sanofi reported a robust 15.7% sales growth in Q3, fueled by strong vaccine sales and the continued success of Dupixent, with sales up 23.8% to €3,476 million. • Pharma launches, including ALTUVIIIO, Nexviazyme, and Rezurock, saw a significant 67.1% increase, contributing €727 million to the overall sales growth. • The company's pipeline advanced with four regulatory approvals, including Dupixent for COPD in the US and China, and four positive Phase 3 data readouts, including tolebrutinib for nrSPMS. • Based on strong Q3 business performance, Sanofi has raised its 2024 business EPS guidance to at least a low single-digit percentage growth at CER.

Recordati Acquires Global Rights to Sanofi's CAD Treatment Enjaymo in $825M Deal

8 months ago

• Recordati has secured worldwide rights to Enjaymo, the first and only approved treatment for cold agglutinin disease (CAD), from Sanofi for an upfront payment of $825 million with potential milestone payments of $250 million. • Enjaymo, a humanized monoclonal antibody targeting C1s in the classical complement pathway, has generated approximately €100M in revenue over the past year and is projected to reach peak sales of €250-300M. • The acquisition strengthens Recordati's rare disease portfolio and addresses an unmet medical need, with Enjaymo already approved by regulatory authorities in the US, Europe, and Japan.

Relacorilant and Surgery Improve Hemostatic Markers in Cushing's Syndrome

8 months ago

• A study shows that hypercoagulopathy, a complication in Cushing's Syndrome (CS), improves with both relacorilant treatment and surgery. • Relacorilant, a selective glucocorticoid receptor modulator (SGRM), demonstrated improvements in coagulation markers after 3-4 months of treatment. • Surgical intervention also led to improvements in coagulation markers within an average of 6 months post-operation in CS patients. • The study suggests relacorilant may offer a beneficial treatment option for CS patients, particularly those at high risk of VTE or not suitable for surgery.

South Korea Accelerates Drug Reimbursement Process with New Pilot Program

a year ago

• South Korea is implementing reforms to reduce drug reimbursement times from approximately 4 years to align more closely with other advanced nations like the US, Japan, and Germany. • A new pilot program called "Approval-evaluation-negotiation linkage system" allows simultaneous review by health authorities, with Recordati's Qarziba and Ipsen's Bylvay being the first drugs included. • The reforms include risk-sharing agreements, waivers for cost-effectiveness analysis for pediatric, rare disease, and cancer treatments, and reduced price negotiation periods from 60 to 30 days.

Italian Pharmaceutical Market Faces $1.6B Decline as Government Pushes Generic Drug Adoption

12 years ago

• Italy's pharmaceutical market is projected to decline from $25.1 billion in 2012 to $23.5 billion by 2020, driven by government austerity measures and strict drug pricing policies. • The Italian Medicines Agency (AIFA) has implemented stringent pricing controls, refusing reimbursement for manufacturers who don't accept their suggested prices, while promoting generic drug adoption. • Despite having an underdeveloped generics market with only 20% volume share compared to 50% in other EU countries, Italy's recent reforms are expected to significantly boost generic drug usage.