Clinical Trial News
Stryker Receives FDA Clearance for OptaBlate BVN System to Treat Chronic Low Back Pain
• Stryker's OptaBlate basivertebral nerve ablation system has received FDA 510(k) clearance, offering a minimally invasive solution for chronic vertebrogenic low back pain.
• The system targets the basivertebral nerve and can achieve a 1 cm lesion in 7 minutes, featuring a steerable curved introducer and microinfusion technology to prevent tissue charring.
• This new addition to Stryker's pain management portfolio addresses an underserved population of patients who have not found relief through conventional treatments like physical therapy, injections, and medications.
FDA Questions Pfizer's Approach to Expand Talzenna's Indication in Prostate Cancer
• The FDA has raised concerns about Pfizer's strategy to expand Talzenna's indication to all castration-resistant prostate cancer patients, questioning the reliance on an "incompletely defined" subgroup without HRR gene mutations.
• Pfizer is seeking to broaden Talzenna's current first-line indication from only HRR-mutated castration-resistant prostate cancer to an all-comers population, representing a significant potential market expansion.
• The regulatory scrutiny highlights ongoing challenges in precision medicine approaches and biomarker-based treatment strategies in oncology drug development.
REGENXBIO Secures $250 Million Royalty Monetization Deal to Advance Gene Therapy Pipeline
• REGENXBIO has closed a non-dilutive royalty bond agreement with Healthcare Royalty for up to $250 million, receiving $150 million upfront that extends its cash runway into early 2027.
• The deal monetizes select royalties from ZOLGENSMA for SMA and payments from gene therapies for MPS disorders, while REGENXBIO retains other funding opportunities including a potential Priority Review Voucher.
• This strategic financing supports REGENXBIO's late-stage pipeline development, including RGX-121 for MPS II, RGX-202 for Duchenne muscular dystrophy, and ABBV-RGX-314 for wet AMD.
Virtual Trial Shows Combination Rescue Inhaler Reduces Severe Asthma Attacks by Nearly 50%
• A groundbreaking virtual phase III trial found that Airsupra, combining budesonide and albuterol, reduced severe asthma exacerbations by 44% compared to albuterol alone in mild asthma patients.
• The innovative fully remote trial design allowed 2,421 participants to receive inhalers by courier and meet investigators via telehealth, potentially revolutionizing future clinical research in pulmonary medicine.
• Beyond reducing severe attacks, Airsupra decreased the annualized exacerbation rate by 53% and reduced systemic steroid exposure by 63%, potentially sparing patients from steroid-related complications.
FDA Advisory Committee Rejects Pfizer's Talzenna Expansion for Broader Prostate Cancer Population
• Pfizer's application to expand Talzenna's indication to all castration-resistant prostate cancer (CRPC) patients was rejected by FDA's advisory committee due to insufficient data in biomarker-negative patients.
• The company sought to broaden Talzenna's current approval beyond patients with HRR gene mutations, but FDA questioned the reliance on an "incompletely defined" subgroup analysis.
• FDA oncology chief Richard Pazdur emphasized that chance effects could not be ruled out due to the lack of a formal efficacy analysis in patients without HRR mutations.
CASI Pharmaceuticals to Host Conference Call on May 21 to Provide Business and Clinical Updates
• CASI Pharmaceuticals will host a live conference call and webcast on May 21, 2025, to provide updates on its business operations and clinical pipeline developments.
• The biopharmaceutical company is focused on developing therapeutics for hematology oncology, organ transplant rejection, and autoimmune diseases, with operations spanning China, the United States, and globally.
• CASI is currently navigating legal challenges related to key pipeline products EVOMELA® and CNCT19, while advancing CID-103 for antibody-mediated rejection in organ transplantation.
NextCell Pharma Strengthens Commercial Strategy with Appointment of MSC Therapy Expert Dr. Eric Strati
• NextCell Pharma has appointed Dr. Eric Strati, former executive at Takeda, Mesoblast, and Novartis, to its Board of Advisors to advance commercial strategy for ProTrans, its type 1 diabetes cell therapy.
• Dr. Strati brings critical expertise from launching two approved MSC-based therapies (Alofisel and Ryoncil), strengthening NextCell's capabilities as it prepares for pivotal trials and partnership opportunities.
• Recent analysis from the ProTrans-Young study shows promising trends in preserving insulin production in patients aged 12-21 years, with full trial results expected in the second half of 2026.
Lexicon's SONATA-HCM Phase 3 Trial Design Unveiled at European Cardiology Congress
• Lexicon Pharmaceuticals has launched SONATA-HCM, the only ongoing Phase 3 trial evaluating sotagliflozin in both obstructive and non-obstructive hypertrophic cardiomyopathy, with enrollment active across 20 countries.
• The randomized, double-blind study will enroll 500 patients globally and measure improvement in symptoms using the Kansas City Cardiomyopathy Questionnaire, addressing a significant unmet need particularly for non-obstructive HCM patients.
• Sotagliflozin, a dual SGLT1 and SGLT2 inhibitor with established efficacy in heart failure, could potentially become the first approved therapy for non-obstructive HCM if trial results support a supplemental new drug application.
Eli Lilly Considers $5.9 Billion Biomanufacturing Facility in Houston
• Pharmaceutical giant Eli Lilly is exploring a $5.9 billion biomanufacturing plant at McCord Development's Generation Park in Northeast Houston, potentially creating 600 permanent jobs and 2,000 construction positions.
• The proposed 236-acre facility would be part of Eli Lilly's broader $50 billion U.S. manufacturing expansion since 2020, aimed at increasing production capacity for medicines including Mounjaro and Zepbound.
• If approved, the project would deliver an estimated $2.5 billion economic impact over 10 years and strengthen Houston's emerging life sciences sector, supported by local biotech workforce development initiatives.
Oligo Factory Expands Capabilities with New Low-Scale Synthesis Platform for Therapeutic and Diagnostic Development
• Oligo Factory has launched a new low-scale oligonucleotide synthesis capability, enabling researchers to order custom DNA and RNA oligos in volumes as small as 50 nmol while maintaining high quality standards.
• The company has appointed Luke Dannenberg as CEO and Baiju Parikh as VP of Commercial to drive expansion, while unveiling three specialized product portfolios: FactorTx™, FactorDx™, and FactorLS™ for therapeutics, diagnostics, and life science applications.
• With a new 13,000-square-foot facility in Holliston, Massachusetts, Oligo Factory has increased synthesis capacity 12-fold and purification capabilities 10-fold, positioning itself as an agile alternative to larger suppliers for specialized oligonucleotide needs.