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Clinical Trial News

ABD-147 and PT217 Receive FDA Orphan Drug Designations for Neuroendocrine Carcinoma Treatment

  • The FDA granted Orphan Drug Designation to ABD-147, a radiopharmaceutical from Abdera Therapeutics, for neuroendocrine carcinoma, offering potential tax credits and market exclusivity.
  • PT217, a bispecific antibody developed by Phanes Therapeutics, also received Orphan Drug Designation for neuroendocrine carcinoma, building on its prior designation for small cell lung cancer.
  • Clinical trials are planned or underway to evaluate both ABD-147 and PT217 in patients with neuroendocrine carcinomas, including small cell lung cancer and large cell neuroendocrine carcinoma.

A Study of Peluntamig (PT217) in Patients With Neuroendocrine Carcinomas Expressing DLL3 (the SKYBRIDGE Study)

NCT05652686RecruitingPhase 1
Phanes Therapeutics
Posted 9/5/2023

FDA Approves Updated COVID-19 Vaccines Targeting Omicron Subvariants for 2024-2025 Season

  • The FDA has approved updated mRNA COVID-19 vaccines from Moderna (Spikevax) and Pfizer-BioNTech (Comirnaty) targeting the Omicron KP.2 subvariant for the 2024-2025 season.
  • Novavax's updated protein-based COVID-19 vaccine, targeting the Omicron JN.1 subvariant, has also received emergency use authorization for individuals 12 years and older.
  • The updated vaccines are formulated to provide improved protection against currently circulating variants, addressing waning immunity from previous exposure or vaccination.
  • Health officials recommend that everyone aged 6 months and older receive a COVID-19 vaccine, particularly those at higher risk of severe illness.

KalVista's Sebetralstat Advances Toward Potential Approval for Hereditary Angioedema

  • KalVista Pharmaceuticals' NDA for sebetralstat, an oral plasma kallikrein inhibitor, has been accepted by the FDA for on-demand treatment of hereditary angioedema (HAE) attacks.
  • The FDA has set a PDUFA goal date of June 17, 2025, for sebetralstat, marking a significant step toward potential market availability.
  • A pediatric clinical trial (KONFIDENT-KID) has been initiated to evaluate an orally disintegrating tablet formulation of sebetralstat in children aged 2 to 11 years.
  • KalVista anticipates filing for approval in the UK, Japan, and other countries later in 2024, expanding the global reach of sebetralstat.

Higher Dose of Spinraza Shows Significant Motor Function Improvement in Infants with SMA

  • Biogen's Spinraza, at a higher dose, demonstrated statistically significant motor function improvement in infants with spinal muscular atrophy (SMA).
  • The Phase II/III DEVOTE trial's Part B cohort showed a 26.19-point increase in CHOP-INTEND scores compared to the sham control group.
  • The higher dose regimen of Spinraza was generally well-tolerated, with a safety profile consistent with the established lower dose.
  • Biogen plans to pursue regulatory approval for the higher dose regimen of Spinraza, potentially reshaping SMA treatment paradigms.

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy

NCT02193074TerminatedPhase 3
Biogen
Posted 8/19/2014

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

NCT04089566CompletedPhase 3
Biogen
Posted 3/26/2020

A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)

NCT04488133Active, Not RecruitingPhase 4
Biogen
Posted 1/4/2021

Palisade Bio Identifies PDE4B as Potential Biomarker for Ulcerative Colitis Patient Stratification

  • Palisade Bio identifies PDE4B expression as a potential biomarker for patient stratification in ulcerative colitis (UC), with higher expression in colitis patients.
  • Data shows elevated PDE4B expression in 70% of adult and 90% of pediatric UC patients, suggesting its utility in identifying suitable candidates for targeted therapies.
  • PALI-2108, an oral, colon-specific PDE4 inhibitor prodrug, is being advanced by Palisade Bio for UC treatment, with Phase 1 trials expected to commence before year-end.
  • Preclinical studies in a DSS-induced UC mouse model demonstrated that PALI-2108 reduces colon tissue PDE4B expression and inflammatory cytokines, correlating with decreased disease activity.

Gilead's Lenacapavir Achieves 100% Efficacy in HIV Prevention Trial

• Gilead Sciences' lenacapavir, a twice-yearly injectable medication, demonstrated 100% efficacy in preventing HIV among cisgender women in Phase 3 trials. • This breakthrough surpasses the efficacy of daily oral medications like Truvada and Descovy, marking a significant advancement in HIV prevention strategies. • The study's findings could transform the landscape of HIV prevention, offering a more convenient and highly effective alternative for at-risk populations.

FDA Grants Fast Track Designation to IBI363 for Advanced Melanoma

  • The FDA has granted Fast Track designation to IBI363 for unresectable locally advanced or metastatic melanoma after progression on at least one prior systemic therapy, including a PD-(L)1 inhibitor.
  • IBI363 is a first-in-class PD-1/IL-2α bispecific antibody fusion protein showing encouraging efficacy and a favorable safety profile in melanoma patients who have previously received immunotherapy.
  • Clinical data from a phase 1/2 trial demonstrated an objective response rate of 29.7% and a disease control rate of 73.0% in melanoma patients previously treated with immunotherapy.
  • The Fast Track designation aims to expedite the development and review of IBI363, offering potential priority review and accelerated approval to address the unmet need in immunotherapy-failed melanoma.

Phase I/II Study Evaluating AUTO4 in Patients With TRBC1 Positive T Cell Lymphoma

NCT03590574CompletedPhase 1
Autolus Limited
Posted 8/30/2018

A Study Evaluating the Safety, Tolerability, and Initial Efficacy of IBI110 in Subjects With Advanced Malignant Tumors

NCT04085185RecruitingPhase 1
Innovent Biologics (Suzhou) Co. Ltd.
Posted 12/4/2019

A Study of Subcutaneous and Intravenous VELCADE in Patients With Previously Treated Multiple Myeloma

NCT00722566CompletedPhase 3
Millennium Pharmaceuticals, Inc.
Posted 7/1/2008

A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory T or B Cell Malignancies (COBALT-LYM)

NCT04502446TerminatedPhase 1
CRISPR Therapeutics AG
Posted 7/31/2020

Safe Pro AI Completes AI Minefield Analysis for UNDP in Ukraine

  • Safe Pro AI completed Phase III of its drone survey operation in Ukraine under a purchase order from the United Nations Development Programme (UNDP).
  • SpotlightAI™, powered by AWS, analyzed land in Kharkiv Oblast, providing detailed reports on items of interest detected by AI and ML models.
  • The technology evaluation program aims to enhance land release efficiency in agricultural areas, supported by multiple international entities and Ukraine's Ministry of Economy.
  • SpotlightAI™ utilizes advanced machine learning to identify over 150 types of land mines and UXO, significantly reducing analysis time and costs.

FDA Grants Orphan Drug Designation to Skyline Therapeutics' SKG1108 for Retinitis Pigmentosa

  • Skyline Therapeutics' SKG1108, a gene therapy for retinitis pigmentosa (RP), has received Orphan Drug Designation from the FDA.
  • SKG1108 uses an AAV vector to deliver light-activatable proteins to the retina, aiming to restore visual function by creating new photo-sensing cells.
  • The Orphan Drug Designation provides Skyline Therapeutics with incentives, including tax credits and potential market exclusivity.
  • SKG1108 compensates for the loss of photoreceptors in late-stage RP, regardless of the specific genetic mutation causing the condition.

University of Montana's Cancer Immunotherapy Enters Phase 1 Human Trials

  • Researchers at the University of Montana, in partnership with Inimmune, have advanced a novel cancer treatment into Phase 1 human clinical trials.
  • The immunotherapy drug, initially developed as an influenza vaccine, aims to retrain the body's immune system to target and destroy cancerous cells.
  • The treatment's development was supported by $33 million in grants and involves a multidisciplinary approach, from molecule design to safety testing.
  • The first patient in the trial is a 54-year-old woman with advanced breast cancer, marking a significant milestone for the research team.

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