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Clinical Trial News

Syntellix Secures NMPA Approval for Bioabsorbable Metallic Implants in China, Unlocking 1.4 Billion Patient Market

  • Syntellix AG received product approval from China's NMPA for its MAGNEZIX® bioabsorbable metallic implants, marking the company's most significant regulatory milestone and opening access to over 1.4 billion potential patients.
  • The Singapore-based manufacturing subsidiary now holds approvals in countries representing more than 5.5 billion inhabitants across 73 countries and regions on five continents, with China representing the largest single market opportunity.
  • The company's bioabsorbable metallic orthopedic implants have been documented in over 50 medical publications as "advantageous" and "clinically superior" to conventional titanium implants, with tens of thousands of successful surgeries completed globally.
  • Syntellix plans to leverage its Singapore production facility as an Asia-wide sales hub and is preparing for an IPO on the Singapore Exchange following this strategic market entry.

Kidney Cancer Research Consortium Advances Novel Combination Therapies Through Multi-Institutional Collaboration

  • The Kidney Cancer Research Consortium, a DoD-funded program led by Dr. Eric Jonasch, includes 7 institutions collaborating on hypothesis-testing clinical trials for novel kidney cancer therapeutic strategies.
  • The consortium has developed a portfolio of 10 clinical trials that combine established immune-modulating agents like nivolumab plus ipilimumab with novel therapeutic agents including LAG3 inhibitors and lutetium-177 girentuximab.
  • Researchers can conduct more mechanistic and scientifically driven investigations while collecting correlative samples including circulating tumor DNA, tissue samples, and quality of life data to acquire more information from fewer patients in less time.

Avalyn Pharma Raises $100M to Advance Inhaled Pulmonary Fibrosis Therapies

  • Avalyn Pharma secured $100 million in an oversubscribed Series D financing round to advance inhaled formulations of existing pulmonary fibrosis drugs pirfenidone and nintedanib.
  • The company is developing AP01 (inhaled pirfenidone) for Phase IIb trials and AP02 (inhaled nintedanib) for Phase II studies, aiming to reduce systemic side effects while maintaining efficacy.
  • Only 30% of the 300,000 U.S. pulmonary fibrosis patients take current oral medications due to severe gastrointestinal side effects including diarrhea, indigestion, and vomiting.
  • The inhaled delivery approach targets the lungs directly at lower doses, potentially improving tolerability for a disease with a median survival of 3-5 years.

FDA Approves First 3D-Printed Epilepsy Drug for Feeding Tube Administration

Drug Approval
  • The FDA has approved Spritam (levetiracetam), the world's first 3D-printed medication, for administration via nasogastric and gastrostomy tubes, making it the only levetiracetam formulation with this approval.
  • The drug uses Aprecia's proprietary ZipDose Technology Platform to create rapidly disintegrating tablets that can be dispersed in minimal liquid volumes for enteral administration.
  • This approval provides critical access for epilepsy patients who cannot take oral medications, particularly those with swallowing difficulties or requiring enteral feeding support.
  • Spritam is indicated for treating partial-onset seizures in patients 4 years and older, myoclonic seizures in juvenile myoclonic epilepsy patients 12 years and older, and generalized tonic-clonic seizures in patients 6 years and older.

Pacira BioSciences Partners with Johnson & Johnson to Expand Zilretta Access for Osteoarthritis Knee Pain

  • Pacira BioSciences has entered a strategic co-promotion agreement with Johnson & Johnson MedTech to expand distribution of Zilretta, the first FDA-approved extended-release intra-articular therapy for osteoarthritis knee pain.
  • The collaboration will leverage Johnson & Johnson's specialized sales team to target broader physician specialties including sports medicine, pain management, and rheumatology beyond traditional orthopedic practices.
  • Zilretta provides extended pain relief for up to 16 weeks and could potentially reach a significant portion of the seven million intra-articular knee injections administered annually in the U.S.
  • The partnership aligns with Pacira's 5x30 growth strategy and Johnson & Johnson's mission to advance orthopaedic care with non-opioid pain management solutions.

Stanford Researchers Develop Radiation-Free Stem Cell Transplant Protocol for Fanconi Anemia Patients

Monoclonal AntibodyRare Disease
  • Stanford Medicine researchers successfully conducted stem cell transplants in three Fanconi anemia patients using the anti-CD117 antibody briquilimab, eliminating the need for toxic radiation or busulfan chemotherapy.
  • All three pediatric patients achieved nearly 100% donor cell engraftment after two years, far exceeding the initial 1% target, with no graft rejection or major complications.
  • The innovative protocol addresses donor shortage by allowing transplants from half-matched donors like parents through alpha/beta T-cell depletion techniques.
  • Researchers are now conducting phase 2 trials and exploring applications for other genetic diseases including Diamond-Blackfan anemia and elderly cancer patients.

STRIDE Immunotherapy Achieves Unprecedented 19.6% Five-Year Survival Rate in Advanced Liver Cancer

  • A new exploratory analysis of the HIMALAYA phase III trial shows that 19.6% of patients with unresectable hepatocellular carcinoma treated with STRIDE immunotherapy remained alive after five years, compared to 9.4% with sorafenib.
  • STRIDE represents the first phase III study to report five-year overall survival data in advanced liver cancer, establishing a new survival benchmark for this devastating disease.
  • The combination therapy demonstrated sustained survival benefits across all clinically relevant patient subgroups with manageable safety and no new late-onset serious side effects.
  • These findings represent a clinical breakthrough that was considered unimaginable just a few years ago and will inform treatment decisions for years to come.

Study of Durvalumab and Tremelimumab as First-line Treatment in Patients With Advanced Hepatocellular Carcinoma

NCT03298451Active, Not RecruitingPhase 3
AstraZeneca
Posted 10/11/2017

HMNC Brain Health and Spruce Biosciences Launch Phase 2 Trial for Precision Depression Treatment

Precision Medicine
  • HMNC Brain Health and Spruce Biosciences have dosed the first patient in the Phase 2 TAMARIND trial, evaluating tildacerfont as a precision treatment for major depressive disorder.
  • The trial targets a biologically distinct subtype of MDD patients with HPA axis dysregulation, using HMNC's proprietary genetic marker-based patient selection tool.
  • Tildacerfont, a CRF1 receptor antagonist, has the potential to address up to 50% of MDD patients worldwide through personalized medicine approaches.
  • The randomized, double-blind, placebo-controlled study will enroll 88 adults across eight UK sites, with topline results expected in the first half of 2026.

Real-World Study Shows Adalimumab Biosimilar IBI303 Matches Humira Efficacy with Superior Cost Benefits in Ankylosing Spondylitis

Real World Evidence
  • A retrospective study of 106 patients with ankylosing spondylitis in China found IBI303 biosimilar demonstrated comparable safety to Humira with higher retention rates at 52 weeks (63% vs 44%).
  • Treatment costs were significantly lower for IBI303 patients compared to Humira at both 12 weeks ($762.20 vs $903.70) and 52 weeks ($2543.00 vs $2982.30).
  • Safety profiles were nearly identical between groups, with only 2 patients in each group experiencing mild infusion-related reactions and no statistically significant differences in adverse events.
  • The findings support IBI303 as a cost-effective first-line treatment option that could expand access to adalimumab therapy while maintaining clinical effectiveness.

Efficacy and Safety of IBI303 in Adult Patients With Active Ankylosing Spondylitis

NCT02893254CompletedPhase 3
Innovent Biologics (Suzhou) Co. Ltd.
Posted 9/22/2016

FDA Rejects Replimune's Melanoma Drug Amid Evolving Regulatory Standards

FDA ApprovalOncology
  • The FDA rejected Replimune's melanoma drug in a complete response letter, citing inadequate single-arm study design and patient enrollment differences as key concerns.
  • The agency questioned whether the single-arm study provided substantial evidence of effectiveness and raised issues with the company's confirmatory trial design.
  • Analysts interpret the rejection as evidence of changing FDA approval standards, with Replimune CEO expressing surprise at the decision given prior agency meetings.
  • The rejection highlights the evolving regulatory landscape for oncology drug approvals, particularly for single-arm studies in melanoma treatment.

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