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Clinical Trial News

Amgen Secures Expanded FDA Approval for Repatha® in Cardiovascular Risk Management

Drug Approval
  • Amgen received expanded FDA approval for Repatha®, broadening its use for adults at risk of major cardiovascular events.
  • The company reported significant Q2 earnings growth with revenue rising to $9,179 million during the same quarter.
  • The expanded approval may help Amgen counter biosimilar competition and patent expirations while potentially stabilizing revenue streams.
  • Amgen's current share price of $288.47 remains below the consensus price target of $311.88, reflecting an 8.12% discount.

European Regulators Approve First-in-Class Topical Anti-Androgen Therapy for Acne Treatment

Drug Approval
  • The European Medicines Agency's CHMP issued a positive opinion recommending approval of Winlevi® (clascoterone 1% cream) for facial acne treatment in adults and adolescents aged 12 to <18 years.
  • The approval follows a successful re-examination after an initial negative opinion, with the CHMP now concluding the benefit-risk profile is favorable for both age groups.
  • Winlevi® represents a first-in-class topical anti-androgen therapy that inhibits androgen receptors in the skin, reducing sebum production and inflammation.
  • The approval significantly expands access to topical anti-androgen treatment for European adolescents, who previously had no such therapeutic option available.

Novel Immunotherapy Combination Achieves 100% Disease Control in Recurrent Glioblastoma Pilot Study

  • A chemotherapy-free regimen combining nogapendekin alfa (Anktiva), NK cell therapy, and tumor treating fields achieved 100% disease control in five recurrent glioblastoma patients.
  • Three of five patients achieved objective responses, including two with near-complete responses, marking unprecedented results in this aggressive brain cancer.
  • The treatment successfully restored lymphocyte counts in all patients who had experienced lymphopenia following standard radiation and chemotherapy.
  • Based on these encouraging early findings, ImmunityBio plans to initiate a randomized Phase 2 trial for second-line glioblastoma patients.

N-803 and PD-L1 t-haNK Combined With Bevacizumab for Recurrent or Progressive Glioblastoma

NCT06061809RecruitingPhase 2
ImmunityBio, Inc.
Posted 8/7/2024

Comprehensive Clinical Trials Review Reveals Global Lennox-Gastaut Syndrome Research Landscape

Rare Disease
  • A new comprehensive report analyzes the global clinical trials landscape for Lennox-Gastaut Syndrome, providing critical data on trial numbers, enrollment figures, and regional distribution across G7 and E7 nations.
  • The analysis covers diverse aspects of clinical trials categorized by region, trial phase, status, sponsorship type, and endpoint analysis, highlighting prominent drugs currently under investigation.
  • Major pharmaceutical companies including Jazz Pharmaceuticals, Takeda, UCB, GSK, and Eisai are actively conducting trials in this rare epilepsy syndrome space.
  • The report aims to facilitate strategic business planning and investment decisions while identifying optimal locations for conducting efficient clinical trials in this challenging therapeutic area.

Allarity Therapeutics Receives FDA Fast Track Designation for Stenoparib in Advanced Ovarian Cancer

FDA Approval
  • Allarity Therapeutics received FDA Fast Track designation for stenoparib, an investigational dual PARP/WNT inhibitor for advanced ovarian cancer treatment.
  • The designation accelerates development timelines and enables more frequent FDA interactions with potential eligibility for expedited approval pathways.
  • The company has initiated a Phase 2 clinical trial building on prior data showing patients remaining on treatment for over 22 months.
  • ALLR stock surged over 119% intraday following the regulatory milestone, with trading volume increasing by 2,728% to $304.8 million.

iNtRON Bio Files Patent Applications for CRISPR-Cas9 Gene Editing Technology Targeting Pasteurella Bacteriophages

Gene Editing
  • iNtRON Bio has filed two patent applications for its IMPA™ gene editing technology that uses CRISPR-Cas9 and recombination methods to modify Pasteurella bacteriophages for therapeutic applications.
  • The technology enables precise genetic manipulation of bacteriophage capsids to create modular phages capable of carrying multiple therapeutic payloads, representing the first reported gene editing of Pasteurella bacteriophages worldwide.
  • The platform leverages an integrated database containing over 11,000 bacteriophage records and 600 whole genome sequences to develop targeted therapeutics, anticancer drugs, and vaccines.
  • The company plans to expand the technology beyond Pasteurella to other bacterial targets and develop various drug candidates including ADC drugs, anticancer agents, and vaccines.

PolarityBio Completes Phase III Enrollment for SkinTE in Diabetic Foot Ulcers, Targeting First BLA Approval in Chronic Wounds Since 1997

  • PolarityBio has completed enrollment of 120 patients in its pivotal Phase III COVER DFUS II trial for SkinTE, an autologous regenerative skin therapy for Wagner 1 diabetic foot ulcers.
  • The trial achieved rapid enrollment at 8.6 patients per month over 14 months, outpacing historical timelines for diabetic foot ulcer studies.
  • SkinTE received FDA Breakthrough Therapy designation and could become the first BLA-approved therapy for chronic wounds since 1997 if results are positive.
  • Final trial results are expected in Q1 2026, with the primary endpoint measuring complete wound closure within 12 weeks.

Curanex Pharmaceuticals Raises $15 Million in IPO to Advance Ulcerative Colitis Treatment

  • Curanex Pharmaceuticals completed its initial public offering, pricing 3.75 million shares at $4 per share to raise $15 million in gross proceeds.
  • The company will use IPO proceeds to develop Phyto-N, its lead product candidate for treating ulcerative colitis.
  • Curanex plans to conduct FDA-required GLP toxicology and pharmacokinetic studies and prepare an Investigational New Drug application for Phyto-N.
  • The company's shares began trading on Nasdaq under ticker CURX on August 26, with the offering expected to close on August 27.

EMA Grants First-Ever Orphan Drug Designation for Radiation Maculopathy Treatment

Rare DiseaseOncology
  • The European Medicines Agency has granted orphan drug designation to Roca Therapeutics' RCT002, marking the first formal recognition of radiation maculopathy as a distinct medical indication.
  • RCT002 is a first-in-class eye drop therapy designed to address resistant neovascularization, inflammation, fibrosis, and oxidative stress in patients experiencing vision loss after radiotherapy.
  • The designation opens pathways for accelerated regulatory support and targeted therapeutic development for a condition that currently has no approved treatment options.
  • Roca Therapeutics plans to initiate first-in-human clinical trials for RCT002 in 2026 while currently fundraising to support the program.

Qlaris Bio Initiates Phase II Trial for Novel Glaucoma Therapy QLS-111 in South Korea

  • Qlaris Bio has dosed the first patient in its Phase II Nightingale clinical trial evaluating QLS-111 for normal tension glaucoma in South Korea.
  • QLS-111 is a first-in-class ATP-sensitive potassium channel modulator that targets episcleral venous pressure to lower intraocular pressure through a novel mechanism.
  • The study builds on positive results from previous Phase II trials showing QLS-111 achieved 3.7 mmHg IOP reduction as monotherapy and additional 3.2-3.6 mmHg reduction when combined with latanoprost.
  • The trial addresses a significant unmet need in normal tension glaucoma patients who experience disease progression despite having intraocular pressures within normal ranges.

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