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Clinical Trial News

Eleva Advances Factor H Therapy CPV-104 into First-in-Human Trial for Rare Kidney Disease C3 Glomerulopathy

Rare Disease
  • Eleva has administered the first dose of CPV-104, a recombinant human complement Factor H therapy, to healthy volunteers in a Phase 1 clinical study for C3 Glomerulopathy (C3G).
  • The therapy targets the abnormal regulation of the complement system that causes C3G, a rare renal disease, and has received Orphan Drug Designation in the European Union.
  • Preclinical data published in Frontiers in Immunology demonstrated CPV-104's ability to normalize serum C3 levels and rapidly degrade C3 deposits in the kidney.
  • This milestone represents Eleva's second proprietary program to advance into clinical trials, with the company also developing CPV-104 for dry AMD as a second indication.

PulseSight Therapeutics Doses First Patient in Phase I Trial of PST-611 for Dry AMD

  • PulseSight Therapeutics has successfully dosed the first patient in its Phase I clinical trial of PST-611, a first-in-class non-viral vectorized therapy for dry age-related macular degeneration and geographic atrophy.
  • PST-611 expresses human transferrin to restore iron homeostasis and address the dysregulation that leads to retinal cell death in dry AMD patients.
  • The first-in-human study will evaluate safety and tolerability in 6-12 patients, with preliminary results expected in early 2026.
  • The therapy utilizes electro-transfection technology to deliver DNA plasmids into the ciliary muscle, potentially requiring retreatment only every four to six months.

Amicus Therapeutics Secures Japan Approval for Pombiliti + Opfolda Combination Therapy for Late-Onset Pompe Disease

Drug ApprovalRare Disease
  • Japan's Ministry of Health, Labour and Welfare has approved Pombiliti + Opfolda for treating adult patients with late-onset Pompe disease, expanding global access to this innovative therapy.
  • The approval was based on data from the Phase 3 PROPEL study, which uniquely studied both treatment-naïve and treatment-experienced patients in a controlled setting.
  • Pombiliti + Opfolda is now approved in seven major markets including the US, EU, UK, Canada, Australia, Switzerland, and Japan.
  • The two-component therapy combines a recombinant human GAA enzyme with enhanced muscle cell uptake and an oral enzyme stabilizer designed to maintain enzyme activity in blood.

Emcure Pharmaceuticals Acquires Full Control of Zuventus Healthcare in ₹724.9 Crore Deal

  • Emcure Pharmaceuticals will acquire the remaining 20.42% stake in Zuventus Healthcare for ₹724.9 crore, making it a wholly-owned subsidiary.
  • The transaction is expected to close in the September quarter of FY26, with payment made in cash tranches.
  • Full ownership will enable complete financial consolidation and unlock synergies across Emcure's domestic business operations.
  • The company has also incorporated a new wholly-owned subsidiary, Emcure Wellness Private Limited, to expand its pharmaceutical operations.

Thailand Achieves Pharmaceutical Independence with First Domestically Produced Targeted Cancer Drugs

Targeted TherapyDrug Approval
  • Thailand has developed its first domestically produced targeted cancer drugs, IMCRANIB 100 and HERDARA, without foreign technology transfer, marking a historic milestone in pharmaceutical independence.
  • IMCRANIB 100 targets multiple cancers including chronic myeloid leukemia and gastrointestinal stromal tumors by inhibiting tyrosine kinase enzyme, while HERDARA serves as a cost-effective alternative to trastuzumab for breast cancer treatment.
  • The breakthrough addresses the financial burden of imported cancer drugs, with trastuzumab previously costing up to 1 million baht per course, significantly improving treatment accessibility under Thailand's universal healthcare system.
  • Production takes place at Thailand's first internationally certified cancer drug manufacturing facility, established in 2020 at the Royal Pharmaceutical Manufacturing Plant in Chonburi Province.

CDSCO Expert Committee Accepts Pfizer's Phase III Data for Abrocitinib in Atopic Dermatitis

  • India's CDSCO expert committee has accepted Pfizer's Phase III clinical trial data for abrocitinib, a selective JAK1 inhibitor for moderate-to-severe atopic dermatitis treatment.
  • The committee reviewed data for both 100 mg and 200 mg tablet formulations during their June 11, 2025 meeting without raising objections or additional conditions.
  • This regulatory milestone follows an earlier SEC recommendation from February 2022 and represents progress toward potential approval in India's systemic therapy market.
  • Abrocitinib targets patients with moderate-to-severe atopic dermatitis who are candidates for systemic therapy, addressing an important unmet medical need.

DRDO and AIIMS-Rishikesh Partner for Clinical Trials of Biomedical Devices in Extreme Environments

  • The Defence Research and Development Organisation (DRDO) and AIIMS-Rishikesh have signed a memorandum of understanding to conduct clinical trials and advanced research in biomedical devices for extreme environments.
  • The collaboration between DRDO's Defence Institute of Physiology and Allied Sciences (DIPAS) and AIIMS-Rishikesh aims to develop products, technologies, and strategies to mitigate adverse effects of extreme environments on human performance.
  • The partnership will focus on formulating biomedical devices and supplements specifically designed to enhance human performance in challenging environmental conditions.

Eli Lilly and Loxo Oncology Advance Phase 3 Trial Testing Pirtobrutinib Combination for Previously Treated CLL/SLL

Oncology
  • Eli Lilly and Loxo Oncology are conducting the BRUIN CLL-322 Phase 3 trial evaluating pirtobrutinib combined with venetoclax and rituximab versus standard venetoclax-rituximab therapy in previously treated chronic lymphocytic leukemia and small lymphocytic lymphoma patients.
  • The randomized, open-label study compares fixed-duration treatment with the experimental three-drug combination against the current two-drug standard of care, with pirtobrutinib and venetoclax administered orally and rituximab given intravenously.
  • The trial, which began in September 2021, is currently active but no longer recruiting participants, with the most recent update submitted in July 2025.
  • Results from this study could significantly impact the companies' market position in the competitive oncology landscape and influence treatment standards for CLL/SLL patients.

Andhra Pradesh Establishes First CDSCO-Notified Medical Device Auditing Body to Streamline Regulatory Compliance

  • Andhra Pradesh becomes home to its first CDSCO-notified auditing body for medical devices, with KIHT Certification Services (KCS) authorized to inspect Class A and Class B manufacturing facilities.
  • KCS operates as the only functional notified body among 14 nationwide in Andhra Pradesh and Telangana, housed within the AMTZ campus in Visakhapatnam.
  • The establishment eliminates the need for out-of-state audits for domestic manufacturers, particularly MSMEs, resulting in significant time and cost savings for regulatory compliance.
  • KCS offers comprehensive services including ISO 13485 certification, quality systems audits, risk management training, and CE-mark consultancy to meet global regulatory standards.

Abu Dhabi Partners with Children's National Hospital to Advance Pediatric Cell and Gene Therapy

  • The Department of Health – Abu Dhabi has signed a strategic MoU with Children's National Hospital to establish the Abu Dhabi – Children's National Hospital Cell and Gene Therapy Clinical Excellence Programme.
  • The partnership will focus on developing advanced pediatric cell and gene therapies including Virus-Specific T-cell therapies, with plans to establish GMP-compliant manufacturing facilities.
  • The collaboration includes comprehensive fellowship programs for Emirati clinician-scientists and specialized training curricula in pediatric cellular immunotherapy and clinical trial management.
  • The initiative aims to position Abu Dhabi as a global hub for life sciences innovation while ensuring sustainable access to cutting-edge treatments for children.

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