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Ocugen's OCU400 Gene Therapy Shows Promise in Retinitis Pigmentosa Phase 1/2 Trial

• OCU400, a gene-agnostic therapy, demonstrated a favorable safety profile in a Phase 1/2 trial for retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). • Approximately 60% of RP patients in the Phase 1/2 study showed improvement in mobility, assessed via luminance-dependent navigation, after OCU400 treatment. • Ocugen's OCU400 has received orphan drug designation from the FDA and EMA for RP and LCA, and is currently in Phase 3 trials. • The Phase 3 trial aims to enroll 150 patients with RP, evaluating OCU400's efficacy across a broad range of genetic mutations.

A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa

NCT06388200RecruitingPhase 3
Ocugen
Posted 6/18/2024

Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

NCT05203939Active, Not RecruitingPhase 1
Ocugen
Posted 1/24/2022

Study Links Conversion Practices to Increased Mental Health Risks in LGBTQ+ Adults

  • A comprehensive US-based PRIDE study of 4,426 sexual and gender minority adults reveals significant associations between conversion practices and adverse mental health outcomes.
  • Research shows participants who underwent both sexual orientation and gender identity conversion practices experienced higher rates of PTSD and suicidality, with 95% confidence intervals.
  • The study, led by Stanford University researchers, demonstrates distinct mental health impacts: orientation-related practices linked to PTSD and gender identity-related practices associated with increased depression.

Incannex Healthcare Announces Positive Clinical Trial Results and Financial Updates

  • Incannex Healthcare reported positive Phase 2 results for PSX-001 in treating generalized anxiety disorder (GAD), showing significant anxiety reduction with synthetic psilocybin and psychotherapy.
  • Dosing has commenced in the Phase 2/3 RePOSA trial of IHL-42X for obstructive sleep apnea (OSA), with Phase 2 results expected in the first half of 2025.
  • The FDA cleared Incannex's IND application for PsiGAD2, a Phase 2b trial of PSX-001, to further evaluate its efficacy and safety in GAD patients.
  • Strategic financing with Arena Investors may provide Incannex up to $59.0 million to support clinical trials and general corporate purposes.

Sequana Medical Anticipates FDA Approval for Alfapump in Q1 2025, Highlights DSR Progress

  • Sequana Medical expects FDA approval for its Alfapump system in Q1 2025, with a US launch planned for H2 2025, targeting the $2.4 billion North American market.
  • The company's DSR therapy showed promise in treating cardiorenal syndrome, with RED DESERT and SAHARA data published in the European Journal of Heart Failure.
  • Sequana Medical secured up to EUR 6.1 million in shareholder financing to extend its cash runway towards Alfapump FDA approval and is exploring direct financing options.
  • MOJAVE study's DSMB approved the start of the randomized cohort, following positive data from the non-randomized cohort, pending additional fundraising.

Cartherics Secures $15 Million to Advance CAR-NK Cell Therapy for Ovarian Cancer

  • Cartherics has successfully raised over $15 million in an oversubscribed private financing round to support its cell therapy programs.
  • The funding will primarily advance the clinical trial of CTH-401, a CAR-NK cell therapy targeting TAG-72 for ovarian cancer treatment.
  • CTH-401 has demonstrated efficacy in preclinical studies, showing potent killing of ovarian cancer cells in vitro and in vivo.
  • The company is expanding its pipeline to include other disease indications beyond ovarian cancer with this new funding.

Drug Shortages in Australia Highlight Critical Need to Include Pregnant Women in Clinical Trials

  • Australia faces severe shortages of essential blood pressure medications for pregnant women, with only six registered drugs available, all over 30 years old, putting mothers and babies at risk.
  • Medical experts call for federal government intervention through a publicly funded entity to ensure stable supply of crucial pregnancy medications, backed by RANZCOG.
  • Global clinical trial data shows concerning trend with only 4% of trials in 2024 including pregnant women, while 36% explicitly exclude them, highlighting systemic issues in drug development.

Survey Reveals Low Intent for Respiratory Vaccinations Among US Adults for 2024-2025 Season

  • A new National Foundation for Infectious Diseases survey shows that only 26% of US adults plan to get updated COVID-19 vaccines, while 45% are hesitant about flu vaccination for the 2024-2025 season.
  • Healthcare provider trust remains high at 75%, yet vaccine hesitancy persists due to concerns about adverse events, with 40% citing safety worries for flu vaccines and 56% for COVID-19 vaccines.
  • Among high-risk populations, vaccination intent varies significantly, with 76% of adults aged 65 and older planning to get flu vaccines, while only 38% of RSV vaccine-eligible individuals intend to get vaccinated.

Ziftomenib Shows Promise in Treatment-Resistant Acute Myeloid Leukemia

  • Ziftomenib, an oral menin inhibitor, demonstrates efficacy in relapsed/refractory AML patients, offering a new treatment avenue.
  • The KOMET-001 trial reveals a partial or complete response in about a third of patients with multiple prior therapies.
  • Patients with NPM1 mutations treated with ziftomenib achieved a 35% complete remission rate, highlighting its potential in this subgroup.
  • FDA granted Breakthrough Therapy designation to ziftomenib, expediting its development and review for AML treatment.

A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, or 7+3 in Patients With AML

NCT05735184RecruitingPhase 1
Kura Oncology, Inc.
Posted 7/18/2023

A Study to Investigate the Safety and Tolerability of Ziftomenib Combinations in Patients with Relapsed/Refractory Acute Myeloid Leukemia

2023-510288-37-00RecruitingPhase 1
Kura Oncology Inc.

Key Medical Conferences Set to Shape Biopharma Industry in 2025

  • Major medical conferences in 2025 will showcase groundbreaking research across multiple therapeutic areas, with ASCO and ESMO remaining premier venues for oncology breakthroughs.
  • Neuroscience gatherings like AAN and CTAD will highlight progress in Alzheimer's research, while cardiovascular meetings ACC, ESC, and AHA will focus on obesity's impact on heart health.
  • The business side of biopharma will take center stage at J.P. Morgan Healthcare Conference, BIO CEO, and BIO International Convention, facilitating crucial industry partnerships and deal-making.

Talazoparib Plus Enzalutamide Demonstrates Overall Survival Benefit in mCRPC

  • The TALAPRO-2 trial showed that talazoparib combined with enzalutamide significantly improved overall survival in metastatic castration-resistant prostate cancer (mCRPC) patients.
  • The survival benefit was observed regardless of homologous recombination repair (HRR) gene mutation status, expanding the potential treatment population.
  • The combination therapy maintained clinically meaningful improvements in radiographic progression-free survival across both all-comer and HRR-deficient cohorts.
  • The safety profile of talazoparib plus enzalutamide was consistent with previous reports, supporting its use in mCRPC treatment.

Talazoparib + Enzalutamide vs. Enzalutamide Monotherapy in mCRPC

NCT03395197Active, Not RecruitingPhase 3
Pfizer
Posted 12/18/2017

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