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Clinical Trial News

Novartis Secures Pan-Canadian Agreement for Leqvio Access in Familial Hypercholesterolemia

Drug ApprovalRare Disease
  • Novartis successfully concluded negotiations with the pan-Canadian Pharmaceutical Alliance for public reimbursement of Leqvio (inclisiran) to treat adults with heterozygous familial hypercholesterolemia.
  • The agreement represents a significant step toward addressing treatment gaps for patients with this genetic condition who face elevated cardiovascular risk from high LDL cholesterol levels.
  • While pCPA negotiations are complete, public reimbursement through provincial and territorial formularies has not yet been secured, requiring further collaboration with drug programs.
  • Since commercialization in 2022, Leqvio has treated more than 4,500 Canadians and 290,000 patients worldwide following Health Canada approval in 2021.

Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)

NCT03397121CompletedPhase 3
The Medicines Company
Posted 11/28/2017

Inclisiran for Participants With Atherosclerotic Cardiovascular Disease and Elevated Low-density Lipoprotein Cholesterol

NCT03399370CompletedPhase 3
The Medicines Company
Posted 12/21/2017

Inclisiran for Subjects With ASCVD or ASCVD-Risk Equivalents and Elevated Low-density Lipoprotein Cholesterol

NCT03400800CompletedPhase 3
The Medicines Company
Posted 11/1/2017

FDA Grants Orphan Drug Designation to Crinetics' Atumelnant for Congenital Adrenal Hyperplasia Treatment

Rare Disease
  • The FDA has granted orphan drug designation to atumelnant, Crinetics Pharmaceuticals' novel oral ACTH receptor antagonist for treating classic congenital adrenal hyperplasia.
  • Phase 2 TouCAHn trial results showed up to 80% mean reduction in androstenedione levels and meaningful improvements in clinical symptoms including resumption of menses.
  • The company plans to initiate Phase 3 CALM-CAH study in adults and Phase 2/3 BALANCE-CAH study in pediatrics in the second half of 2025.
  • Orphan drug status provides seven years of market exclusivity, FDA fee exemptions, and financial incentives for clinical development if approved.

ZEISS Receives CE Mark Approval for AI-Powered CIRRUS PathFinder OCT Assessment Tool

AI
  • ZEISS Medical Technology announced CE mark approval for CIRRUS PathFinder, an AI-powered clinical support tool that uses deep learning algorithms to automatically identify abnormal macular OCT B-scans.
  • The fully integrated AI decision support system flags B-scans requiring further review and allows assessment of hundreds of scans in real time without requiring third-party platforms.
  • The new CIRRUS software release includes AI-enhanced OCTA image quality, multi-layer segmentation, and an expanded Reference Database triple the size of the previous version.
  • Research indicates that physicians leveraging AI algorithms can be more effective than practitioners working alone, supporting more confident decision-making and efficient patient care.

Andelyn Biosciences Partners with Amplo Biotechnology to Advance AAV Gene Therapies for Neuromuscular Junction Disorders

Rare Disease
  • Andelyn Biosciences has partnered with Amplo Biotechnology to provide scalable manufacturing of clinical-grade AAV material using Andelyn's suspension AAV Curator® platform for neuromuscular junction disorders.
  • The collaboration aims to advance Amplo's AAV gene therapy programs toward clinical evaluation, with a focus on safety and efficacy for upcoming trials targeting genetic NMJ diseases.
  • Neuromuscular junction disorders typically result in muscle weakness and can be life-threatening, with genetic forms usually diagnosed in early childhood but potentially manifesting in adolescence or adulthood.
  • Amplo's lead program, AMP-101, is advancing toward a first-in-human trial in Dok7 congenital myasthenic syndrome, built on research from the University of Tokyo and Oxford.

Remote Weight-Loss Intervention Shows Significant Success in Breast Cancer Survivors at One-Year Mark

  • Breast cancer patients in a remote weight-loss intervention program lost an average of 4.7% of their baseline body weight after one year, while control group participants gained 1% of their baseline weight.
  • The phase III BWEL trial enrolled nearly 3,180 women from over 637 cancer treatment centers across the U.S. and Canada, demonstrating effectiveness across diverse patient populations and treatment factors.
  • Results showed 46.5% of intervention patients lost 5% of baseline weight and 22.5% lost 10%, compared to only 14.3% and 5% respectively in the control group.
  • The study sets the stage for ongoing research to determine whether weight loss after breast cancer treatment can reduce cancer recurrence risk and extend survival.

Johnson & Johnson Commits $55 Billion to U.S. Manufacturing Expansion in Strategic Reshoring Initiative

  • Johnson & Johnson announced a $55 billion U.S. manufacturing initiative in March 2025, representing 25% more capital allocation to domestic operations than the previous four years.
  • The expansion includes three new advanced manufacturing facilities, with a $2 billion biologics plant in Wilson, North Carolina already underway to produce oncology, immunology, and neuroscience therapies.
  • The initiative aims to reduce supply chain vulnerabilities exposed by geopolitical tensions, trade wars, and global disruptions while creating 500 permanent jobs and generating $3 billion economic impact in North Carolina over a decade.
  • The strategic move aligns with U.S. policy incentives including the Inflation Reduction Act and positions J&J to maintain stable supply of critical therapies like Darazalex and Carvykti amid international trade uncertainties.

Takeda Pharmaceutical Explores India for Global Clinical Trials to Accelerate Drug Launches

Oncology
  • Japanese drugmaker Takeda Pharmaceutical is exploring conducting global clinical trials in India to accelerate the launch of innovative drugs in the world's most populous nation.
  • The company aims to integrate India's research and development ecosystem into its global pipeline, expanding patient access to therapies in oncology, neuroscience, gastrointestinal health, and inflammation.
  • Takeda plans to launch key cancer drugs over the next two to three years in India, including a lung cancer drug ready to hit the market this year.
  • The company has established an innovation center in Bengaluru, expanding from 500 to 750 employees specializing in AI, data science, engineering, and design to support global digital transformation.

Strand Therapeutics Appoints Cancer Immunotherapy Pioneer Dr. Jason Luke as Chief Medical Officer

  • Strand Therapeutics has appointed Dr. Jason J. Luke, an internationally recognized physician-scientist in cancer immunotherapy, as Chief Medical Officer to strengthen its clinical development capabilities.
  • Dr. Luke brings extensive experience from over 100 clinical studies, including serving as global principal investigator for the FDA/EMA registration trial KEYNOTE-716 that established adjuvant therapy for stage II melanoma.
  • The appointment comes as Strand's lead programmable mRNA therapy STX-001 is showing unprecedented response rates in late-stage cancer patients in its ongoing first-in-human clinical trial.
  • Dr. Luke will oversee Strand's clinical development strategy and pipeline advancement, focusing on the company's programmable mRNA platform that enables logic-controlled expression and precision delivery.

Boehringer Ingelheim Partners with AnGes for HGF Gene Therapy Manufacturing Ahead of FDA Filing

FDA Approval
  • Boehringer Ingelheim has signed a contract development and manufacturing agreement with AnGes for the drug substance of their investigational hepatocyte growth factor gene therapy targeting peripheral arterial disease.
  • The partnership follows AnGes' completion of clinical trials and plans to submit a biologics license application to the FDA for their HGF gene therapy product.
  • Peripheral arterial disease affects 200 million people worldwide and can lead to severe complications including ulceration, infection, and limb amputation with mortality rates comparable to cancer.
  • The plasmid DNA molecule has been manufactured using proprietary microbial technology in E. coli at Boehringer's Vienna facility for nearly 20 years since the collaboration began.

Krystal Biotech Halts Melanoma Study of KB707 Following FDA Rejection of Similar Viral Immunotherapy

  • Krystal Biotech has discontinued its Phase I/II OPAL-1 study of KB707, a viral immunotherapy for melanoma, citing heightened regulatory uncertainty following the FDA's rejection of Replimune's similar therapy RP1.
  • Both KB707 and RP1 use modified herpes simplex virus type 1 vectors to trigger immune responses against tumors, but the FDA's controversial rejection of RP1 has complicated accelerated approval pathways for this drug class.
  • The company is now focusing on an inhaled version of KB707 for non-small cell lung cancer, which showed a 36% objective response rate in heavily pretreated patients and has secured an FDA End of Phase II meeting for October.
  • Over 20 melanoma experts have challenged the FDA's rejection of RP1 in an open letter, arguing that the agency's concerns about patient population heterogeneity actually better represent real-world treatment scenarios.

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