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Clinical Trial News

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

Ofichem Group Acquires Avivia to Expand Complex Formulation Development Capabilities

  • Ofichem Group has agreed to acquire Avivia B.V., a Netherlands-based contract development organization specializing in formulation development and analytical method development.
  • The acquisition strengthens Ofichem's integrated development offering, enabling broader support from early-phase development through GMP production with enhanced complex formulation capabilities.
  • Avivia brings a team of 10 scientists with expertise in dissolution profiling, excipient characterization, and stability testing to complement Ofichem's operations in Ter Apel and Leiden.
  • The strategic move supports Ofichem's ambition to evolve as a science-led CDMO offering end-to-end services from drug substance development through commercial-scale manufacturing.

Xaira Therapeutics Appoints Jeff Jonker as President and COO to Scale AI-Driven Drug Discovery Platform

AIDrug Discovery
  • Xaira Therapeutics has appointed biotech veteran Jeff Jonker as President and Chief Operating Officer to help scale its AI-enabled drug discovery platform and drive the next phase of growth.
  • Jonker brings over two decades of leadership experience, having served as CEO of Belharra Therapeutics and Ambys Medicines, and President of NGM Biopharmaceuticals where he formed strategic partnerships with Merck.
  • The appointment comes as Xaira aims to fundamentally reimagine drug discovery by integrating machine learning, biological insight, and therapeutic development to accelerate medicine discovery.
  • Xaira recently published the X-Atlas/Orion dataset, profiling over 8 million single cells as the largest publicly available genome-wide Perturb-seq dataset to power next-generation foundation models in biology.

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.

Anthem Biosciences Positions as China Alternative in $3.4 Billion IPO Amid Global Supply Chain Shift

  • Anthem Biosciences is launching a ₹3,395 crore IPO next week, positioning itself as a key alternative to Chinese CRDMOs as global pharma companies diversify supply chains amid geopolitical uncertainties.
  • The Bengaluru-based company has achieved 24.8% revenue CAGR over five years, reaching ₹1,844 crore in FY25 with a 36.8% EBITDA margin, making it one of India's fastest-growing CRDMOs.
  • Anthem serves over 550 pharma and biotech clients with specialized capabilities in complex modalities including peptides, antibody drug conjugates, and oligonucleotides, with 16 late-phase projects nearing commercialization.
  • The company is aggressively expanding capacity with ₹451.4 crore capex investment across multiple units to meet growing demand from global pharma seeking alternatives to Chinese suppliers.

Empagliflozin Demonstrates Cost-Effectiveness in Chronic Kidney Disease Treatment, EMPA-KIDNEY Analysis Shows

  • A new analysis from the EMPA-KIDNEY trial demonstrates that empagliflozin is cost-effective for chronic kidney disease treatment, improving quality-adjusted life years while reducing healthcare costs.
  • The study found that empagliflozin reduced hospital admissions by 14% and generated net healthcare cost savings of approximately £600 per patient over four years.
  • Cost-effectiveness probability increased from 43% at two years to 91% at four years, with benefits consistent across patient subgroups including those with and without diabetes.
  • The findings support current NICE recommendations and suggest even greater cost-effectiveness is anticipated with generic versions expected to reduce SGLT2 inhibitor costs significantly.

LEO Pharma's Tralokinumab Meets All Endpoints in Phase 3b Hand Atopic Dermatitis Trial

Monoclonal Antibody
  • LEO Pharma announced positive 16-week interim results from the phase 3b ADHAND trial, with tralokinumab meeting all primary and secondary endpoints for moderate to severe atopic dermatitis on the hands.
  • The IL-13 targeting monoclonal antibody showed statistically significant improvements in skin clearance, itch and pain reduction, and severity scores compared to placebo.
  • More than 50% of patients with moderate-to-severe atopic dermatitis have hand involvement, representing a significant unmet medical need in this difficult-to-treat area.
  • The treatment was generally well-tolerated with no new safety signals identified, with most adverse events being non-serious and mild to moderate in severity.

A 32-week Trial to Evaluate the Efficacy and Safety of Tralokinumab in Subjects With Moderate-to-severe Atopic Hand Eczema Who Are Candidates for Systemic Therapy

NCT05958407Active, Not RecruitingPhase 3
LEO Pharma
Posted 8/28/2023

MIT Develops Implantable Device for Automated Glucagon Delivery to Prevent Diabetic Hypoglycemia

  • MIT engineers have developed an implantable device that automatically administers glucagon to prevent life-threatening hypoglycemic episodes in diabetics, with testing in mice showing blood sugar stabilization within 10 minutes.
  • The quarter-sized device uses a 3D-printed polymer reservoir sealed with nickel-titanium shape-memory alloy and can be triggered by continuous glucose monitoring or remote activation.
  • Preclinical studies demonstrated four-week functionality with successful drug delivery despite fibrotic tissue formation, with human clinical trials projected to begin within three years.
  • The device showed versatility by also delivering epinephrine effectively, suggesting potential applications beyond diabetes for emergency medication administration.

Wearable Device Algorithm Detects COVID-19 Infections Seven Days Earlier Than Symptom-Based Screening

  • The COVID-RED trial, involving 17,825 participants, demonstrated that a wearable device algorithm combined with symptom reporting detected SARS-CoV-2 infections significantly earlier than symptom-only screening (median 0 vs 7 days before positive test).
  • The experimental algorithm achieved very high sensitivity (93.8-99.2%) but extremely low specificity (0.8-4.2%), generating many false positive alerts compared to the control algorithm's moderate performance (43.3-46.4% sensitivity, 66.4-65.0% specificity).
  • Researchers plan to refine the algorithm using improved machine learning methodologies and better alert thresholds to reduce false positives while maintaining early detection capabilities for COVID-19 and potentially other respiratory infections.

Sartorius Stedim Biotech Partners with Sensible Biotechnologies to Scale Cell-Based mRNA Manufacturing Platform

  • Sartorius Stedim Biotech and Sensible Biotechnologies have signed a Memorandum of Understanding to advance the industrialization of Sensible's first-in-class cell-based mRNA manufacturing platform.
  • The collaboration aims to enable clinical-grade production of cell-based mRNA by 2026 and unlock the next generation of mRNA medicines with unprecedented purity and performance.
  • The partnership will support Sensible's expansion from Slovakia to the US, UK, and broader Europe through process development, closed-system facility design, and GMP-compliant production capabilities.
  • Sensible's unique platform manufactures mRNA in living cells, promising superior quality needed for emerging mRNA therapeutics while supporting a more resilient biomanufacturing ecosystem.

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