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Clinical Trial News

Iovance Biotherapeutics Cuts Workforce by Nearly 20% Following Slow Amtagvi Cell Therapy Launch

Drug Approval
  • Iovance Biotherapeutics is reducing its workforce by less than 20% in a strategic restructuring following disappointing commercial performance of its TIL cell therapy Amtagvi.
  • The company slashed its 2024 revenue forecast by 40% in May, from $450-475 million to $250-300 million, citing challenging launch dynamics for the melanoma treatment.
  • Amtagvi, the first marketed tumor-infiltrating lymphocyte therapy priced at $515,000, represents a breakthrough in cellular medicine but faces adoption challenges due to complex manufacturing and logistics.
  • The restructuring aims to extend Iovance's cash runway while the company pursues potential approvals in Europe, UK, and Canada, plus late-stage lung cancer trials.

MAXONA Pharmaceuticals Submits IND Application for MAX-001, Novel Non-Opioid Pain Treatment

  • MAXONA Pharmaceuticals has submitted an Investigational New Drug (IND) application to the FDA for MAX-001, a non-opioid, non-NSAID oral therapy for acute and chronic pain treatment.
  • MAX-001 is an optimized extended-release formulation of nefopam that functions as a triple neurotransmitter re-uptake inhibitor, representing a novel mechanism of action for pain management.
  • Phase 1 clinical trial results demonstrated that MAX-001 was well tolerated with no serious adverse events, early discontinuations, or concerning safety findings reported.
  • The IND submission marks the fourth significant development milestone for MAX-001 in 2025, positioning the company to initiate Phase 2 studies upon FDA clearance.

NICE Recommends First NHS Treatment for Rare Eye Disease LHON, Ending Access Disparity in England

Drug ApprovalRare Disease
  • NICE has recommended idebenone (Raxone) as the first NHS treatment for Leber's hereditary optic neuropathy (LHON) in England, benefiting an estimated 250 eligible patients aged 12 and over.
  • The approval ends a disparity in UK access, as the drug has been available in Scotland, Wales, and Northern Ireland for several years, with NHS England set to provide treatment within three months.
  • Clinical evidence from RHODOS, LEROS, and PAROS trials demonstrates that idebenone can improve vision and reduce eyesight deterioration by restoring cellular energy production in affected eye cells.
  • LHON primarily affects young men and boys, causing rapid progression from painless central vision blurring to potential blindness in both eyes within months, with devastating impacts on independence and employment prospects.

Alexion Secures Canadian Access Agreement for Ultomiris in Two Rare Autoimmune Diseases

Rare Disease
  • Alexion, AstraZeneca's Rare Disease division, has reached a Letter of Intent with Canada's pan-Canadian Pharmaceutical Alliance for public reimbursement of Ultomiris (ravulizumab) for NMOSD and generalized myasthenia gravis patients.
  • The complement inhibitor demonstrated complete relapse prevention in all treated NMOSD patients at 72 weeks in pivotal trials, with improved dosing every two months compared to existing therapies.
  • Individual provinces and territories can now begin formulary listing processes, marking a significant milestone for Canadian patients with these debilitating rare autoimmune conditions.

Intas Pharmaceuticals and Accord BioPharma Acquire UDENYCA Biosimilar, Expanding Global Pegfilgrastim Market Presence

Oncology
  • Intas Pharmaceuticals and Accord BioPharma have completed the acquisition of UDENYCA (pegfilgrastim-cbqv), a biosimilar to Amgen's Neulasta, from Coherus BioSciences.
  • The acquisition positions the companies as one of the largest global suppliers of pegfilgrastim, significantly expanding their FDA-approved biosimilar portfolio.
  • UDENYCA is indicated to decrease infection incidence in cancer patients receiving myelosuppressive chemotherapy and offers three patient-friendly administration options.
  • Accord was the first company to commercialize a pegfilgrastim biosimilar in Europe and the only company to launch an autoinjector formulation.

Large-Scale Study Confirms Tamiflu Safety in Children, Debunks Neuropsychiatric Risk Concerns

Real World Evidence
  • A comprehensive study of over 692,000 children found that Tamiflu reduces neuropsychiatric events by 50% compared to untreated influenza, contradicting long-standing safety concerns.
  • The research analyzed Tennessee Medicaid data from 2016-2020, including 129,000 children treated for influenza, with 67% receiving oseltamivir treatment.
  • Findings published in JAMA Neurology demonstrate that influenza itself, not Tamiflu treatment, is associated with increased risk of seizures, mood disorders, and self-harm behaviors.
  • The study provides reassurance to healthcare providers and parents about Tamiflu's safety profile when used early in pediatric influenza treatment.

Apreo Health Raises $130 Million to Advance Pivotal Trial for Novel Emphysema Device

  • Apreo Health secured $130 million in Series B funding co-led by Bain Capital Life Sciences and Norwest to advance its BREATHE Airway Scaffold for severe emphysema treatment.
  • The funding will support BREATHE-3, a multicenter randomized controlled trial evaluating the bronchoscopically delivered implant designed to release trapped air in hyperinflated lungs.
  • More than three million Americans are affected by severe emphysema, yet fewer than 1% receive interventional treatment, highlighting significant unmet medical need.
  • The BREATHE Airway Scaffold received FDA Breakthrough Device Designation in May 2024, representing a fundamentally different approach to emphysema care.

Study to Assess Safety, Feasibility, and Preliminary Efficacy of the Apreo Implant for Severe Emphysema

NCT05949645Active, Not RecruitingNot Applicable
Apreo Health, Inc.
Posted 8/15/2023

pH-Responsive Graphene Nanomaterials Achieve Enhanced Tumor Targeting in Breakthrough Cancer Drug Delivery Study

  • Researchers from Okayama University and CNRS developed charge-reversible graphene oxide nanomaterials that remain undetected in neutral blood pH but become positively charged in acidic tumor environments.
  • The optimized GOPGNH60-DMMA variant demonstrated superior tumor cell uptake while avoiding healthy tissue binding, confirmed through mouse model studies.
  • This breakthrough in pH-responsive nanomedicine provides concrete guidelines for improving targeted drug delivery and opens new avenues for theranostic applications combining cancer diagnosis and treatment.

Mass General Brigham Launches Clinical Trials for Haystack MRD ctDNA Test in Head and Neck and Skin Cancers

  • Mass General Brigham has initiated two prospective clinical trials to evaluate Haystack MRD, a highly sensitive circulating tumor DNA test, for guiding postoperative treatment decisions in cutaneous squamous cell carcinoma and HPV-independent head and neck squamous cell carcinoma.
  • The trials aim to investigate the clinical utility of this minimally invasive liquid biopsy technology for risk stratification and early detection of residual disease in cancer types that currently lack robust monitoring options.
  • Previous research published in The New England Journal of Medicine demonstrated that Haystack MRD identified clinical complete response at a median of 1.4 months compared to over six months using traditional imaging methods.
  • The collaboration between Haystack Oncology, a Quest Diagnostics subsidiary, and renowned cancer centers represents a significant step toward personalized, proactive cancer care through molecular monitoring.

Minghui Pharmaceutical Secures $131 Million Pre-IPO Funding to Advance Oncology Pipeline and Commercial Launch

Oncology
  • Minghui Pharmaceutical closed a $131 million Pre-IPO financing round led by OrbiMed and co-led by Qiming Venture Partners to advance its late-stage clinical programs.
  • The funding will specifically support the company's PD-1/VEGF bispecific antibody development and combination strategies with antibody-drug conjugates (ADCs).
  • Proceeds will also enable the planned commercial launch of Minghui's topical JAK inhibitor currently under NDA review in China.
  • The company's pipeline includes multiple late-stage assets targeting oncology and immunology, including a B7-H3 ADC in Phase 3 trials for second-line small cell lung cancer.

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