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Clinical Trial News

EIB Provides €150 Million to Alfasigma for Rare Disease and Specialty Care Innovation

  • The European Investment Bank has signed a €150 million financing agreement with Italian pharmaceutical company Alfasigma to support R&D activities from 2025-2027.
  • The funding will focus on developing innovative therapies in rare diseases and specialty care, particularly in gastroenterology, hepatology, vascular medicine, and rheumatology.
  • This investment aims to accelerate the transformation of scientific advances into patient-centered solutions addressing unmet medical needs.
  • The agreement supports the EIB's strategy to strengthen European healthcare sector competitiveness and create highly skilled jobs in biopharmaceutical research.

Recursion Pharmaceuticals Acquires Full Rights to REV102, Potential First-in-Class Oral ENPP1 Inhibitor for Hypophosphatasia

Rare Disease
  • Recursion Pharmaceuticals acquired full rights to REV102, a potential first-in-class oral ENPP1 inhibitor for hypophosphatasia (HPP), from joint venture partner Rallybio for up to $25 million.
  • REV102 targets ENPP1 enzyme to restore inorganic pyrophosphate balance for proper bone mineralization, potentially offering the first oral disease-modifying therapy for HPP patients.
  • The drug candidate is currently in IND-enabling studies with Phase 1 clinical trials expected to begin in the second half of 2026.
  • HPP affects over 7,800 diagnosed patients across the US and EU5, with many having limited access to current enzyme replacement therapies.

JCR Pharmaceuticals Licenses JUST-AAV Gene Therapy Platform to Alexion in $825 Million Deal

  • JCR Pharmaceuticals has entered into a licensing agreement with Alexion, AstraZeneca Rare Disease, for its proprietary JUST-AAV capsid technology to develop genomic medicines for rare diseases.
  • The agreement allows Alexion to use the licensed capsids in up to five genomic medicine programs, with JCR eligible to receive milestone payments totaling up to $825 million plus tiered royalties.
  • JUST-AAV technology utilizes modified adeno-associated virus vectors with miniaturized antibodies for enhanced tissue-specific targeting, including liver-sparing, muscle-targeting, and brain-targeting variants.
  • This marks the third partnership between JCR and Alexion, building on previous collaborations involving J-Brain Cargo technology for neurodegenerative diseases and oligonucleotide therapeutics.

Certara Expands Clinical Technology Partnership with Merck to Streamline Data Management

  • Certara and Merck have expanded their clinical technology collaboration to include metadata repository and data standards workflow management through the Pinnacle 21 platform.
  • Phase III clinical trials now generate an average of 3.6 million datapoints, representing a threefold increase over the past decade according to Tufts Center data.
  • The expanded partnership aims to reduce time from data collection to validated, analysis-ready datasets in modern digital trials.
  • The collaboration builds upon Merck's existing use of Certara's technology platform for regulatory submissions support.

Circular Genomics Secures $250K ADDF Investment to Advance CircRNA Blood Biomarkers for Alzheimer's Diagnosis

Precision Medicine
  • Circular Genomics received $250,000 from the Alzheimer's Drug Discovery Foundation to develop circular RNA blood-based biomarkers for diagnosing and predicting Alzheimer's disease progression.
  • The company is developing the first blood-based tests using circRNA technology to enable personalized diagnosis and treatment for neurological conditions, offering potential advantages over current protein-based biomarkers.
  • The research partnership with Washington University will focus on improving diagnostic accuracy and distinguishing Alzheimer's disease from other dementias to reduce misdiagnosis and delayed diagnosis.
  • CircRNA biomarkers are mainly expressed in brains but can be measured in blood, providing a window into overall brain health and positioning them as novel diagnostic tools for brain disorders.

Hansa Biopharma Appoints Dr. Richard Philipson as Chief Medical Officer to Lead Next Development Phase

Rare Disease
  • Hansa Biopharma has appointed Dr. Richard Philipson as Chief Medical Officer effective July 14, bringing over 25 years of industry experience to the commercial-stage biopharmaceutical company.
  • Dr. Philipson's track record includes clinical leadership resulting in four product approvals, including rare disease and gene therapy treatments, with expertise spanning first-in-human to Phase 3 studies.
  • The appointment positions Hansa to advance its proprietary IgG-cleaving enzyme technology platform, including imlifidase for kidney transplantation and HNSA-5487 for autoimmune diseases.
  • Dr. Philipson will support upcoming regulatory submissions and help build strategic development plans for future indications across rare diseases, inflammation, and oncology.

Flightpath Biosciences Licenses Microbiome-Sparing Antibiotic Lolamicin for Gram-Negative Bacterial Infections

  • Flightpath Biosciences has licensed lolamicin, a novel antibiotic developed at the University of Illinois that targets gram-negative bacteria while preserving beneficial gut microbes.
  • The drug demonstrated 100% efficacy against drug-resistant septicemia and 70% efficacy against pneumonia in animal models without disrupting the gut microbiome.
  • Lolamicin killed up to 100% of multidrug-resistant clinical isolates of E. coli, K. pneumoniae, and E. cloacae at higher doses.
  • The company aims to begin human clinical trials as early as 2026 following additional preclinical studies and FDA approval.

Australian Scientists Develop PROTEUS Biological AI Platform to Accelerate Drug Discovery

AIDrug Discovery
  • Australian researchers have created PROTEUS, a groundbreaking "biological AI" platform that enables rapid evolution of molecules with new or enhanced functions directly inside mammalian cells.
  • The system represents a major breakthrough by allowing directed evolution within mammalian cells for the first time, previously limited to bacterial cells.
  • PROTEUS can reduce drug development processes from years to weeks by mimicking and accelerating natural selection.
  • The platform has been made open source to enable global adoption for developing advanced enzymes, molecular tools, therapeutics, and improved gene-editing medicines.

Predictive Oncology Secures $10M Equity Agreement to Advance AI-Driven Cancer Drug Discovery

AIDrug DiscoveryOncology
  • Predictive Oncology Inc. has entered into a $10 million standby equity purchase agreement with Yorkville Advisors to fund AI-driven drug discovery and repurposing initiatives.
  • The company's PEDAL AI platform demonstrates 92% accuracy in predicting tumor response to drug compounds, enabling more informed drug-tumor combination selection.
  • The flexible funding arrangement allows Predictive Oncology to control timing and amount of stock sales without minimum commitments or operational restrictions.
  • The partnership aims to accelerate drug discovery, biomarker discovery, and business development opportunities with leading biopharmaceutical companies.

Taiho's Pizuglanstat Fails to Meet Primary Endpoint in Phase III Duchenne Muscular Dystrophy Trial

Rare Disease
  • Taiho Pharmaceutical's pizuglanstat (TAS-205) failed to demonstrate significant improvement in time to rise from floor compared to placebo in the Phase III REACH-DMD study.
  • The randomized, double-blind trial enrolled 82 male DMD patients aged 5 years and older across 26 Japanese sites over three years.
  • This setback adds to recent DMD therapeutic failures, including Sarepta/Roche's Elevidys and Pfizer's withdrawn gene therapy program.
  • Pizuglanstat works by inhibiting hematopoietic prostaglandin D synthase to reduce muscle necrosis and inflammatory response in DMD patients.

A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）

NCT04587908Active, Not RecruitingPhase 3
Taiho Pharmaceutical Co., Ltd.
Posted 11/1/2020

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