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Clinical Trial News

Elixirgen Therapeutics Receives FDA Rare Pediatric Disease Designation for EXG-34217 in Dyskeratosis Congenita

  • Elixirgen Therapeutics received FDA Rare Pediatric Disease Designation for EXG-34217, a treatment for dyskeratosis congenita and related telomere biology disorders (DC/TBD).
  • EXG-34217 consists of autologous CD34+ hematopoietic stem cells treated with EXG-001, encoding human ZSCAN4, using a Sendai virus vector.
  • The FDA's designation acknowledges the significant unmet need for DC/TBD therapies and the potential of EXG-34217 to address this rare condition.
  • Elixirgen is currently enrolling patients in a Phase 1/2 trial at Cincinnati Children’s Hospital Medical Center to evaluate EXG-34217.

Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

NCT04211714RecruitingPhase 1
Elixirgen Therapeutics, Inc.
Posted 4/8/2021

Safety and Immunogenicity of EXG-5003

NCT04863131CompletedPhase 1
Fujita Health University
Posted 4/28/2021

Cidara Therapeutics Presents Positive Clinical Data on CD388 for Influenza Prevention

  • Cidara Therapeutics presented safety and pharmacokinetic data from CD388 clinical trials at the OPTIONS XII conference.
  • The data supports CD388's safety profile across various doses and administration routes in healthy subjects.
  • Pharmacokinetic results suggest CD388 could provide season-long influenza A and B protection in diverse populations.
  • Cidara plans to advance CD388 into a Phase 2b trial for the Northern Hemisphere influenza season in 2024.

Palisade Bio Advances PALI-2108 for Ulcerative Colitis with Phase 1 Trial Imminent

  • Palisade Bio is set to initiate a Phase 1 clinical trial for PALI-2108, a PDE4 inhibitor prodrug, targeting moderate to severely active ulcerative colitis (UC).
  • The Phase 1 trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of PALI-2108 in healthy volunteers and UC patients.
  • Palisade Bio is developing precision medicine tests using PDE4B expression to identify patients most likely to respond to PALI-2108, potentially improving treatment outcomes.
  • A pre-Clinical Trial Application meeting with Health Canada has been completed, and the CTA for the Phase 1 study has been submitted.

Personalized Medicine and Genetic Markers Drive Major Advances in Pediatric Cancer Treatment

  • Pediatric cancer treatment has achieved remarkable success with cure rates reaching 85%, thanks to nationwide institutional collaboration and development of cooperative treatment groups.
  • Advanced genetic marker analysis enables personalized treatment approaches for pediatric cancers, leading to improved outcomes in brain tumors, kidney cancers, and hematologic malignancies.
  • Implementation of immunotherapy has allowed reduction in conventional therapy usage, with leukemia cure rates increasing from 85% to over 95% while minimizing long-term adverse effects.

CellBion's Lu-177-DGUL Shows Promise in Metastatic Prostate Cancer Treatment

• CellBion is advancing Lu-177-DGUL, a radiopharmaceutical, for metastatic castration-resistant prostate cancer (mCRPC), aiming for conditional approval in Korea by late 2025. • Phase 2 trial interim results indicate a 38.5% objective response rate for Lu-177-DGUL, potentially surpassing Novartis' Pluvicto, which reported 28.9%. • CellBion emphasizes Lu-177-DGUL's improved safety profile due to its enhanced metabolic stability and faster elimination from the body via kidney excretion. • The company plans global expansion through technology exports and is strategically securing patents in multiple countries to solidify its market position.

Mirai Bio Launches $50M AI Platform to Accelerate Genetic Medicine Development

  • Flagship Pioneering launches Mirai Bio with $50 million in funding to develop an AI-powered platform that optimizes the design, delivery, and manufacturing of genetic medicines.
  • The Cambridge-based company offers an open platform approach, enabling partnerships with biotech and pharmaceutical companies to streamline genetic medicine development across multiple therapeutic areas.
  • Led by Flagship veterans Geoffrey von Maltzahn and Hari Pujar, Mirai Bio aims to address critical technological challenges in nucleic acid-based medicine development through machine intelligence and collaborative innovation.

Repurposed HIV Drug Shows Promise in Treatment-Resistant Ovarian Cancer

• Researchers have identified an off-patent HIV drug that demonstrates potential efficacy against treatment-resistant ovarian cancer, warranting further clinical investigation. • A Phase 1B clinical trial, endorsed by clinicians and consumer advocates, awaits funding to assess the safety and efficacy of the repurposed HIV drug in approximately 40 patients. • Concurrently, the team is developing an early-detection blood test (liquid biopsy) to identify markers indicative of ovarian cancer in its nascent stages. • The early-detection blood test development involves combining datasets and collaboration with a cancer biobank to validate the sequencing test's accuracy in diagnosing ovarian cancer.

Aspirin and Heparin Combination Significantly Improves Live Birth Rates in Women with Recurrent Pregnancy Loss

  • A Kobe University-led study reveals that low-dose aspirin or heparin treatment significantly increases live birth rates in women with recurrent pregnancy loss who test positive for specific self-targeting antibodies.
  • The research indicates that treatment with aspirin or heparin led to an 87% live birth rate, compared to 50% in untreated women, showcasing a substantial improvement in pregnancy outcomes.
  • The study also found that the treatment reduced pregnancy complications from 50% to 6% among live births, highlighting the potential of these drugs to improve maternal and fetal health.
  • Women with only the newly discovered antibodies who received the treatment had a 93% live birth rate with no pregnancy complications, suggesting a targeted and highly effective approach.

INTREPID Trial: Fever Prevention Post-Stroke Fails to Improve Functional Outcomes

• The INTREPID trial demonstrated that targeted temperature management effectively reduced fever burden in ICU stroke patients compared to standard care. • Despite successful fever prevention, the study found no significant difference in functional recovery, as measured by the modified Rankin Scale (mRS) at 3 months. • Researchers suggest that the heterogeneous nature of cerebrovascular disease and variability in management options may contribute to the neutral outcome. • Experts emphasize the need for future research to identify specific patient subgroups that may benefit from fever prevention strategies after stroke.

GLP-1 Agonists Show Promise in Weight Management for Breast Cancer Patients

• A retrospective study at Memorial Sloan Kettering Cancer Center found that GLP-1 agonists led to an average weight loss of 5% in breast cancer patients. • Weight loss increased with longer duration of GLP-1 agonist treatment, with patients losing over 6 kg by the end of their treatment. • The study found no significant associations between breast cancer subtype, endocrine therapy, or diabetes status and the amount of weight loss achieved with GLP-1 agonists. • Experts suggest further clinical trials are needed to rigorously study the role of GLP-1 agonists in adverse event management and breast cancer outcomes.

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