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Clinical Trial News

Knight Therapeutics Files New Drug Submission for CREXONT in Canada Following Successful Phase 3 Trial

  • Knight Therapeutics has filed a New Drug Submission for CREXONT, a novel extended-release carbidopa/levodopa formulation for Parkinson's disease, which has been accepted for review by Health Canada.
  • The RISE-PD phase 3 trial with 630 patients demonstrated that CREXONT significantly improved daily "Good On" time by 0.53 hours compared to immediate-release formulations while reducing dosing frequency from five to three times daily.
  • CREXONT is expected to compete in markets valued at over $50 million in Canada and $120 million in Brazil, with Knight holding exclusive commercialization rights through a partnership with Amneal Pharmaceuticals.
  • The innovative formulation combines immediate-release granules with extended-release beads using mucoadhesive polymer technology to enable rapid onset and prolonged levodopa absorption in the gut.

Metastasis-Directed Radiotherapy Shows Durable Disease Control in Oligometastatic Renal Cell Carcinoma Without Systemic Therapy

  • A phase 2 trial demonstrated that metastasis-directed radiotherapy without concurrent systemic therapy achieved a median progression-free survival of 34.0 months in patients with oligometastatic clear cell renal cell carcinoma.
  • The treatment showed favorable safety profile with only 6.7% of patients experiencing grade 3 or higher adverse events, significantly lower than the 45-85% seen with standard systemic therapies.
  • Circulating tumor DNA biomarkers showed promise for patient selection, with MRD-negative patients demonstrating superior systemic therapy-free survival compared to MRD-positive patients.
  • The approach offers substantial cost advantages over frontline systemic therapies, with stereotactic radiation costing $15,000-$40,000 per round versus $150,000-$300,000 annually for immunotherapy or tyrosine kinase inhibitors.

Stereotactic Body Radiation Therapy in Treating Patients With Oligometastatic Renal Cell Carcinoma

NCT03575611SuspendedPhase 2
M.D. Anderson Cancer Center
Posted 6/14/2018

Lexicon Pharmaceuticals Presents Patient-Reported Data on Diabetic Peripheral Neuropathic Pain at ASPN 2025

  • Lexicon Pharmaceuticals presented patient survey data on diabetic peripheral neuropathic pain (DPNP) at the 7th Annual Meeting of the American Society of Pain and Neuroscience to inform development of pilavapadin.
  • The study aims to give voice to nearly 100 million people worldwide suffering from DPNP, which affects approximately 9 million patients in the U.S. and up to 50% of type 2 diabetes patients.
  • Pilavapadin (LX9211) is an investigational once-daily oral AAK1 inhibitor that demonstrated efficacy in Phase 2a trials and has received FDA Fast Track designation for DPNP treatment.

Belzutifan Demonstrates Superior Quality-Adjusted Survival Time Compared to Everolimus in Advanced Renal Cell Carcinoma

OncologyTargeted Therapy
  • Belzutifan showed a clinically meaningful 2.66-month improvement in quality-adjusted time without symptoms or toxicity (Q-TWiST) compared to everolimus in advanced renal cell carcinoma patients.
  • The phase 3 LITESPARK-005 trial demonstrated an 11.32% relative gain in Q-TWiST with belzutifan, despite numerically longer time with grade 3/4 toxicity.
  • Belzutifan patients experienced significantly longer time without symptoms and progression (10.73 vs 6.07 months) driven by prolonged periods without disease progression.
  • The analysis supports belzutifan's clinical benefit profile, with lead investigator Thomas Powles noting superior response rates, progression-free survival, and quality of life outcomes.

A Study of Belzutifan (MK-6482) Versus Everolimus in Participants With Advanced Renal Cell Carcinoma (MK-6482-005)

NCT04195750Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 2/27/2020

Processa Pharmaceuticals Advances PCS6422 in Phase 2 Breast Cancer Trial with $7 Million Funding Boost

Oncology
  • Processa Pharmaceuticals is developing PCS6422, a lead oncology asset currently in Phase 2 clinical trials for breast cancer treatment.
  • The company focuses on transforming chemotherapy agents into more effective cancer therapies while minimizing toxicity to patients.
  • Processa recently secured $7 million in funding to support its innovative approach to oncology drug development.
  • CEO George Ng will discuss the company's progress and corporate developments on Bloomberg TV's Small Stocks, Big Money show.

Aenova Expands Irish Facility to Tackle Drug Bioavailability Challenge with Advanced Spray Drying Technology

  • German pharmaceutical CDMO Aenova announces significant expansion of its Killorglin, Ireland facility to develop innovative drug delivery systems focusing on spray-dried amorphous solid dispersions and inhalation powders.
  • The strategic investment targets improving bioavailability of poorly soluble drugs, which constitute approximately 80% of compounds in global research pipelines.
  • The expansion will create 10 new positions in research and development, pharmaceutical technology, and analytics at the facility that currently employs 68 people.
  • The new spray drying technology aims to help pharmaceutical customers bring their products to market faster by addressing critical bioavailability challenges.

Hengrui Medicine Advances Cancer Pipeline with Breakthrough Therapy Designations and Clinical Trial Approvals

Orphan DrugOncologyMonoclonal Antibody
  • Jiangsu Hengrui Medicine secured inclusion of two products on China's proposed breakthrough therapy list, highlighting their potential to address unmet medical needs in gastric cancer treatment.
  • The company received US FDA orphan drug designation for trastuzumab for injection, providing development incentives including tax credits and market exclusivity for this gastric cancer therapy.
  • Hengrui obtained clinical trial approval for three oncology drugs, enabling advancement of its cancer treatment research and potential commercialization of innovative therapies.
  • The pharmaceutical company partnered with GlaxoSmithKline to develop up to 12 innovative medicines across respiratory, immunology, inflammation, and oncology therapeutic areas.

Bristol Myers Squibb's Reblozyl Fails Primary Endpoint in Phase III Myelofibrosis Trial Despite Secondary Benefits

  • Bristol Myers Squibb's anemia drug Reblozyl failed to meet its primary endpoint of red blood cell transfusion independence in the Phase III INDEPENDENCE trial for myelofibrosis patients.
  • Despite missing statistical significance (p=0.0674), the drug demonstrated meaningful improvements in secondary endpoints including a 50% reduction in transfusion burden and hemoglobin increases.
  • The company plans to approach FDA and EMA to discuss potential marketing applications based on the secondary endpoint benefits and unmet medical need in myelofibrosis anemia.
  • The mixed results highlight the challenge of treating myelofibrosis-associated anemia, where up to 60% of patients are affected and current JAK inhibitors provide limited benefit for this complication.

A Safety and Efficacy Study to Evaluate Luspatercept in Subjects With Myeloproliferative Neoplasm-associated Myelofibrosis Who Have Anemia With and Without Red Blood Cell-transfusion Dependence

NCT03194542CompletedPhase 2
Celgene
Posted 11/15/2017

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions

NCT04717414Active, Not RecruitingNot Applicable
Celgene
Posted 2/25/2021

Pediatric Hidradenitis Suppurativa Shows Superior Response to Adalimumab Over Infliximab in Real-World Study

  • A new study reveals adalimumab demonstrates superior drug survival compared to infliximab in pediatric patients with hidradenitis suppurativa, contrasting with adult patient data.
  • Biologic-naïve pediatric patients showed significantly higher drug survival rates than those previously exposed to biologics, highlighting the importance of early intervention.
  • Researchers developed an educational module to increase awareness and reduce stigma of hidradenitis suppurativa among children, addressing the average 10-year diagnostic delay.
  • The findings suggest earlier diagnosis and adalimumab initiation may improve treatment outcomes in pediatric hidradenitis suppurativa patients.

Telomir Pharmaceuticals' Lead Compound Shows Promise in Reversing Cancer Gene Silencing

  • Telomir-1 completely reversed epigenetic silencing of the STAT1 tumor suppressor gene in aggressive human prostate cancer cells during a 21-day preclinical study.
  • The compound outperformed established cancer drugs Paclitaxel and Rapamycin in reactivating critical cancer defense pathways through DNA methylation modulation.
  • Telomir Pharmaceuticals stock surged over 140% following the announcement, with the company preparing to file for its first IND application in the coming months.
  • The preclinical data represents a novel approach to cancer treatment by targeting epigenetic "off switches" that silence the body's natural tumor suppression mechanisms.

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