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Personalized ALS Drug Shows Breakthrough Results, Slowing Disease Progression by More Than Half

Rare DiseasePrecision Medicine
  • A custom-built antisense oligonucleotide drug targeting the CHCHD10 gene mutation has slowed ALS progression by more than half in Dr. Rakesh Parekh, marking the first treatment for this specific genetic variant.
  • The personalized therapy, developed by the n-Lorem Foundation, cost $1.2 million and took three years to create but can now be adapted for other patients with the same mutation.
  • This breakthrough represents a new era of "N-of-1" medicine for ALS, with gene-targeting therapies showing potential to transform treatment for the 15-20% of patients with known genetic mutations.

ACROBiosystems and DAAN Biotherapeutics Form Strategic Alliance to Accelerate Precision Immuno-Oncology Development

Precision Medicine
  • ACROBiosystems and DAAN Biotherapeutics signed a strategic MOU to establish a multi-faceted collaboration framework for developing precision immunotherapies targeting drug-resistant cancers.
  • The partnership combines ACROBiosystems' GMP-grade bioreagent expertise with DAAN's proprietary immune-modulation platform that selectively activates T cells within tumor microenvironments.
  • DAAN's lead TCR-T cell therapy pipeline has completed preclinical development and is preparing for global clinical trials, with platform applications expanding to autoimmune and rare diseases.
  • The collaboration aims to overcome current immunotherapy limitations including toxicity and therapeutic resistance through enhanced precision targeting and improved manufacturing capabilities.

Mosaic Neuroscience Partners with iXCells to Pioneer Patient-Specific ALS Disease Models

Precision Medicine
  • Mosaic Neuroscience and iXCells Biotechnologies launched a groundbreaking pilot project to develop patient-specific models of sporadic ALS, which accounts for 90% of ALS cases.
  • The initiative aims to address the 99% failure rate of ALS treatments by creating "neurobiopsies" - lab-grown neurons that replicate individual patient disease patterns.
  • Project Mosaic adapts precision medicine methods from cancer research to transform ALS drug development, moving away from familial ALS models that represent fewer than 10% of patients.
  • The pilot will use patient-derived stem cell lines to generate transcriptomic signatures unique to each sporadic ALS patient, potentially enabling personalized drug matching.

Controlled Release Drug Delivery Market Expands with Major Pharma Investment in Precision Medicine Technologies

Precision Medicine
  • Major pharmaceutical companies including AbbVie, AstraZeneca, and Merck are actively developing controlled drug release systems to enhance treatment of chronic diseases, cancer, and neurological disorders.
  • The market is shifting toward personalized and targeted drug delivery mechanisms driven by biotechnology advances and precision medicine approaches.
  • Non-invasive delivery methods such as implantable systems and transdermal patches are gaining traction for improved patient convenience and compliance.
  • Clinical trials span multiple therapeutic areas from pain management and oncology to neurological diseases and metabolic disorders, validating safety and efficacy of these advanced delivery systems.

Zephyr AI Names Watson Pharmaceuticals Founder Dr. Allen Chao as CEO, Acquires Aster Insights to Accelerate Cancer Drug Development

AIPrecision MedicineReal World EvidenceOncology
  • Zephyr AI has appointed Dr. Allen Chao, founder of Watson Pharmaceuticals, as Chief Executive Officer, bringing over 40 years of biopharmaceutical leadership experience.
  • The company simultaneously acquired Aster Insights, gaining access to the world's largest observational cancer study dataset with over 400,000 lifetime-consented patients.
  • The combined entity creates a formidable real-world evidence platform that integrates AI-driven precision medicine with comprehensive genomic, clinical, and transcriptome data.
  • Integration efforts are already underway focusing on biomarker discovery, clinical trial optimization, and AI-enabled companion diagnostics development.

Oxcia's OXC-101 Receives Dual Orphan Drug Designation from EMA and FDA for Acute Myeloid Leukemia Treatment

Precision Medicine
  • Oxcia's first-in-class mitotic MTH1 inhibitor OXC-101 has received Orphan Drug Designation from both the European Medicines Agency and the US FDA for acute myeloid leukemia treatment.
  • The dual approvals provide up to 10 years of market exclusivity in the EU and 7 years in the US, along with accelerated regulatory processes and significant cost savings.
  • Preclinical studies demonstrate that OXC-101 significantly reduces tumor growth and prolongs survival in AML models, with clinical benefits observed in ongoing Phase I/II trials.
  • The company is currently conducting the MAATEO expansion study combining OXC-101 with idarubicin in relapsed/refractory AML patients, showing partial responses and stable disease up to 5 months.

MAATEO: A phase 1 study in patients with hematological malignancies to evaluate safety, tolerability and efficacy of Karonudib

2024-513766-20-00RecruitingPhase 1/2
Oxcia AB
Posted 12/3/2019

Incyclix Bio Raises $11.25 Million to Advance CDK2 Inhibitor INX-315 for Treatment-Resistant Breast and Ovarian Cancers

Precision Medicine
  • Incyclix Bio secured $11.25 million in Series B extension funding from investors including Eli Lilly and Company, Eshelman Ventures, and Pharmacosmos to advance its Phase 1/2 clinical trial.
  • The funding will support completion of the ongoing INX-315-01 trial evaluating the novel CDK2 inhibitor in patients with CDK4/6 inhibitor-resistant ER+/HER2- breast cancer and CCNE1-amplified solid tumors.
  • INX-315 represents a precision treatment approach targeting aberrant cell cycle proliferation in advanced and treatment-resistant cancers, with study completion expected by mid-2026.
  • The company has already presented interim clinical data from the dose escalation portion at the 2024 San Antonio Breast Cancer Symposium.

Open-Label Study to Evaluate the Safety, Tolerability, PK, and Efficacy of INX-315 in Patients With Advanced Cancer

NCT05735080RecruitingPhase 1
Incyclix Bio
Posted 3/28/2023

Oculis Strengthens Leadership with Appointment of Two World-Renowned Ophthalmology Experts as Chief Medical Advisors

Precision Medicine
  • Oculis appointed Mark Kupersmith, M.D., as Chief Medical Advisor for Neuro-Ophthalmology following positive Phase 2 ACUITY trial results with Privosegtor (OCS-05) in acute optic neuritis.
  • Sebastian Wolf, M.D., Ph.D., was named Chief Medical Advisor for Ophthalmology as the company advances its Phase 3 DIAMOND trials of OCS-01 eye drops in diabetic macular edema.
  • The appointments strengthen Oculis' scientific leadership as the company progresses toward potential regulatory filings, with topline results from Phase 3 trials expected in Q2 2026.
  • Both advisors bring decades of clinical expertise, with Kupersmith having 40 years in neuro-ophthalmology and Wolf authoring over 400 peer-reviewed publications in retinal diseases.

Sarepta Therapeutics Refinances $700M Convertible Notes to Strengthen Financial Position

Precision Medicine
  • Sarepta Therapeutics exchanged approximately $700 million of 1.25% convertible notes due 2027 for $602 million in new 4.875% notes due 2030, extending debt maturity by three years.
  • The refinancing includes up to 6.7 million shares of common stock and $123.3 million in cash, with new notes having a conversion price of approximately $60.00 per share.
  • CEO Doug Ingram stated the transaction enhances balance sheet flexibility and positions the company to fund its pipeline including siRNA programs while meeting near-term obligations.
  • Following the exchange, approximately $450 million in aggregate principal amount of the original convertible notes will remain outstanding with unchanged terms.

Tempus AI Acquires Paige to Build World's Largest Oncology Foundation Model

AIPrecision MedicineOncologyDrug Discovery
  • Tempus AI has acquired Paige, an AI company specializing in digital pathology with FDA-cleared cancer detection applications and nearly 7 million digitized pathology slides.
  • The $81.25 million stock acquisition positions Tempus to build the largest oncology foundation model in history by combining datasets and AI capabilities.
  • The strategic move strengthens Tempus' position in the digital pathology market projected to reach $10 billion by 2030, enhancing drug discovery and personalized medicine capabilities.
  • Paige's global dataset from 45 countries and partnership with Memorial Sloan Kettering Cancer Center adds significant credibility and diversity to Tempus' AI platform.

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