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Lisata Therapeutics Reports Promising 60% Response Rate in Pancreatic Cancer Trial with Certepetide

Orphan Drug
  • Lisata Therapeutics and WARPNINE completed enrollment in the Phase 1b/2a iLSTA trial evaluating certepetide in combination with standard-of-care therapy for locally advanced pancreatic ductal adenocarcinoma.
  • Updated preliminary data presented at ESMO-GI demonstrated a 60% overall response rate and 100% disease control rate, with 13 of 20 participants showing partial response and one complete response.
  • The study represents the first evaluation of certepetide in locally advanced non-resectable pancreatic cancer, an earlier disease stage where immunotherapy has been largely ineffective.
  • Certepetide is designed to enhance tumor penetration of co-administered anti-cancer drugs and has received multiple regulatory designations including Fast Track and Orphan Drug status.

LogiCare3PL Secures Exclusive Distribution Partnership for ChiRhoStim Diagnostic Product

Orphan DrugRare Disease
  • LogiCare3PL, a division of BioCare Inc., has entered into an exclusive third-party logistics partnership with ChiRhoClin Inc. for the distribution of ChiRhoStim (Human Secretin for Injection), effective July 14, 2025.
  • The partnership leverages LogiCare3PL's specialized expertise in orphan drug logistics and operates through two cGMP-compliant, NABP-accredited distribution centers in Arizona and Mississippi.
  • ChiRhoStim serves as a critical diagnostic product for pancreatic and gastrointestinal conditions, supporting ChiRhoClin's mission to provide timely access to diagnostic treatments for rare diseases.

AstraZeneca's Anselamimab Fails Primary Endpoint in Phase III AL Amyloidosis Trial, Shows Promise in Patient Subgroup

Monoclonal AntibodyRare DiseaseOrphan Drug
  • AstraZeneca's CARES Phase III clinical programme evaluating anselamimab in patients with Mayo stages IIIa and IIIb light chain amyloidosis did not achieve statistical significance for the primary endpoint of time to all-cause mortality and cardiovascular hospitalizations.
  • Despite missing the primary endpoint in the overall population, anselamimab demonstrated highly clinically meaningful improvement in survival and cardiovascular hospitalization rates in a prespecified patient subgroup compared to placebo.
  • The investigational anti-fibril monoclonal antibody represents a potentially first-in-class approach to clearing amyloid deposits in AL amyloidosis, a rare and life-threatening disorder affecting an estimated 74,000 patients worldwide.
  • Anselamimab was well tolerated with balanced adverse events between treatment and placebo arms, and Alexion plans to share full results with global health authorities and present data at upcoming medical meetings.

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)

NCT04512235Active, Not RecruitingPhase 3
Alexion Pharmaceuticals, Inc.
Posted 11/12/2020

A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis

NCT04304144CompletedPhase 2
Alexion Pharmaceuticals, Inc.
Posted 3/18/2020

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)

NCT04504825Active, Not RecruitingPhase 3
Alexion Pharmaceuticals, Inc.
Posted 2/2/2021

Lisata Therapeutics Secures Composition of Matter Patent for Certepetide Through 2040

Orphan Drug
  • Lisata Therapeutics received a new U.S. composition of matter patent for certepetide extending intellectual property protection through March 2040, with potential for further extension.
  • The patent covers certepetide's chemical structure, pharmacokinetic properties, manufacturing methods, and applications for treating solid tumor cancers.
  • Certepetide is an investigational cyclic peptide designed to enhance tumor penetration of co-administered anti-cancer drugs and has demonstrated favorable safety and clinical activity in pancreatic cancer trials.
  • The patent provides exclusive manufacturing and selling rights, strengthening Lisata's negotiating position with potential partners and delaying generic competition for nearly two decades.

Medera Treats First Patient in Groundbreaking Gene Therapy Trial for Duchenne Muscular Dystrophy Heart Failure

Orphan DrugRare Disease
  • Medera and University of Kansas Medical Center successfully treated the first patient in the MUSIC-DMD Phase 1b trial, marking the first-in-human gene therapy approach for DMD-associated cardiomyopathy.
  • The AAV1.SERCA2a gene therapy was delivered via minimally invasive intracoronary infusion, with the patient tolerating the procedure well and being discharged after overnight observation.
  • The trial addresses a critical unmet need as cardiac complications have become the leading cause of death in DMD patients, affecting nearly all patients by age 18.
  • The open-label study will enroll up to 12 adult males across low-dose, high-dose, and control groups to evaluate safety and efficacy of the one-time gene therapy treatment.

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

NCT06224660RecruitingPhase 1
Sardocor Corp.
Posted 10/2/2024

60 Degrees Pharmaceuticals Seeks FDA MUMS Designation for First Oral Canine Babesiosis Treatment

Orphan Drug
  • 60 Degrees Pharmaceuticals will submit a Minor Use Minor Species (MUMS) designation request to the FDA for tafenoquine to treat acute canine babesiosis in 2025.
  • Three independent clinical studies demonstrated that tafenoquine was well tolerated and appeared to facilitate recovery from acute canine babesiosis infection.
  • Currently no FDA-approved oral treatment exists for canine babesiosis, which affects several hundred to several thousand dogs annually in the United States.
  • The company's CEO noted that positive canine findings align with earlier research suggesting tafenoquine could be effective against babesiosis in humans.

Oral Tafenoquine Plus Standard of Care Versus Placebo Plus Standard of Care for Babesiosis

NCT06207370RecruitingPhase 2
60 Degrees Pharmaceuticals LLC
Posted 6/17/2024

Phase 2 Open Label Study of Tafenoquine for Treatment of Chronic Babesiosis Patients with Severe Fatigue

NCT06656351Not Yet RecruitingPhase 2
60 Degrees Pharmaceuticals LLC
Posted 2/1/2025

Expanded Use in Persistent (B. Microti) Babesiosis

NCT06478641available
60 Degrees Pharmaceuticals LLC

CEL-SCI Raises $5.7M to Fund Pivotal Multikine Trial for Head and Neck Cancer

Orphan Drug
  • CEL-SCI Corporation completed a $5.7 million stock offering to fund its confirmatory Phase 3 trial for Multikine, a neoadjuvant immunotherapy targeting head and neck cancer patients with low PD-L1 expression.
  • The company faces severe liquidity constraints with only $1.93 million in cash as of Q1 2025 and quarterly burn rate of approximately $7 million, creating an urgent need for additional funding.
  • Multikine previously demonstrated a 73% five-year survival rate versus 45% for standard care in a specific patient subset, earning FDA Orphan Drug designation.
  • The confirmatory trial will enroll 212 patients and represents CEL-SCI's final opportunity to validate decades of research in neoadjuvant immunotherapy for head and neck cancer.

Zorevunersen Shows Promising Cognitive and Behavioral Improvements in Dravet Syndrome Phase 3 Trial Analysis

Rare DiseaseOrphan Drug
  • Biogen and Stoke Therapeutics presented new data showing zorevunersen improved cognition and behavior at Week 68 in a Phase 3 dosing regimen analysis for Dravet syndrome patients.
  • The mixed-effects model analysis demonstrated improvements in multiple cognitive and behavioral measures that contrasted with natural history data from standard-of-care treatment.
  • The findings support the inclusion of five Vineland-3 Adaptive Behavior Scale sub-domains as key secondary endpoints in the ongoing Phase 3 EMPEROR study.
  • Zorevunersen represents a potential first-in-class disease-modifying treatment targeting the underlying genetic cause of Dravet syndrome, with no current approved therapies addressing cognitive and behavioral aspects.

Rhythm Pharmaceuticals' Setmelanotide Shows Breakthrough Results in Rare Hypothalamic Obesity Trial

Rare DiseaseOrphan Drug
  • Rhythm Pharmaceuticals' setmelanotide achieved a 19.8% BMI reduction at 52 weeks in the Phase 3 TRANSCEND trial for acquired hypothalamic obesity, with 80% of patients achieving clinically meaningful weight loss.
  • The company plans to submit regulatory applications to FDA and EMA by Q3 2025 for the first-ever approved therapy for acquired hypothalamic obesity, affecting approximately 50,000 patients globally.
  • With no current approved treatments for this rare condition caused by hypothalamic damage, setmelanotide could capture a market worth $1.5-2.5 billion in peak sales at rare disease pricing levels.

A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity

NCT06046443Active, Not RecruitingPhase 2
Rhythm Pharmaceuticals, Inc.
Posted 7/11/2024

Zhimeng Biopharma's CB03-154 Receives Chinese Regulatory Approval for ALS Phase 2/3 Trial

Orphan Drug
  • Shanghai Zhimeng Biopharma received approval from China's NMPA to initiate a Phase 2/3 clinical study of CB03-154, a next-generation KCNQ2/3 potassium channel opener for amyotrophic lateral sclerosis treatment.
  • CB03-154 previously received FDA Orphan Drug Designation and demonstrated significant preclinical efficacy in reducing motor neuron hyperexcitability and extending life expectancy in ALS models.
  • The drug represents a potential first-in-class therapy for ALS, addressing a significant unmet medical need in a disease with 2-5 year average survival and no current treatments capable of halting disease progression.

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