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Biohaven's Troriluzole Shows Promise in Slowing Spinocerebellar Ataxia Progression

  • Biohaven's troriluzole demonstrated a 50-70% slowing of disease progression in patients with spinocerebellar ataxia (SCA) over three years compared to untreated controls.
  • The study, designed with FDA input, utilized real-world evidence and achieved statistically significant improvements in the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA).
  • Biohaven plans to submit a New Drug Application (NDA) to the FDA in Q4 2024 for troriluzole to treat all SCA genotypes, potentially leading to commercialization in 2025.
  • Troriluzole has received Fast-Track and Orphan Drug designations from the FDA, which may expedite the review process for this rare and debilitating neurodegenerative disease.

Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control

NCT06529146Active, Not Recruiting
Biohaven Therapeutics Ltd.
Posted 3/18/2019

Troriluzole in Adult Participants With Spinocerebellar Ataxia

NCT03701399Active, Not RecruitingPhase 3
Biohaven Pharmaceuticals, Inc.
Posted 3/8/2019

FDA's Rare Disease Innovation Hub Aims to Accelerate Treatment Development

  • The FDA has launched a Rare Disease Innovation Hub to foster collaboration and accelerate the development of therapies for rare diseases.
  • The hub seeks to streamline the treatment-approval process, making it more accessible for patients and their families.
  • The initiative builds upon previous legislative efforts, such as the MD Care Act and the BENEFIT Act, to support rare disease research and patient involvement.
  • The hub will facilitate communication between teams developing various rare disease therapies, potentially expediting the availability of new treatments.

Apellis' Syfovre Faces Third Rejection by EU's CHMP for Geographic Atrophy

  • Apellis Pharmaceuticals' Syfovre (pegcetacoplan) received a third negative opinion from the CHMP for treating geographic atrophy (GA) in the EU.
  • The decision follows previous rejections and a procedural reset due to a European Court of Justice ruling on EMA's expert group organization.
  • Syfovre is approved in the U.S. for GA, demonstrating strong initial uptake with $292.1 million in sales in the first half of 2024.
  • Apellis continues to seek approval for pegcetacoplan in other countries, while also marketing it as Empaveli/Aspaveli for paroxysmal nocturnal hemoglobinuria.

Pancreatic Cancer Treatment Advances: New Trials and Therapies Offer Hope

  • Over 2,000 pancreatic cancer trials have been initiated since 2019, with the US and China leading research efforts, indicating a strong global focus on addressing this aggressive disease.
  • Pan-RAS inhibitors, particularly targeting the G12D mutation, are emerging as promising treatments, potentially altering therapeutic paradigms and improving outcomes for specific patient subgroups.
  • Combination therapies and targeted agents are gaining prominence in clinical trials, reflecting efforts to overcome tumor heterogeneity and resistance mechanisms in pancreatic cancer.
  • Updated guidelines from NCCN, ESMO, and Japan's Pancreas Society emphasize tailored treatment strategies, including FOLFIRINOX and gemcitabine-based therapies, to improve survival rates.

RNS60 Shows Promise in Extending Survival for ALS Patients in Phase 2 Trial

  • A post-hoc analysis of a Phase 2 trial reveals that RNS60, when added to standard ALS therapies, extends median survival by approximately six months compared to placebo.
  • The study identifies that patients with slower decline in lung function and lower levels of NfL and MCP-1 biomarkers experienced greater survival benefits from RNS60.
  • RNS60, an experimental therapy targeting inflammation and mitochondrial dysfunction, warrants further investigation in a Phase 3 clinical trial for ALS.
  • Revalesio's RNS60 has received orphan drug and fast track status from the FDA, potentially accelerating its development and review for ALS treatment.

The Effect of RNS60 on ALS Biomarkers

NCT03456882CompletedPhase 2
Mario Negri Institute for Pharmacological Research
Posted 5/30/2017

Novel PROTAC Degrader ACBI3 Shows Efficacy Against Multiple KRAS Mutant Cancers

  • A newly developed small molecule, ACBI3, has demonstrated efficacy against 13 of the 17 most common KRAS mutant-driven cancers, offering a potential treatment option for a broader range of patients.
  • ACBI3, a PROTAC-based degrader, works by tagging the mutant KRAS proteins for degradation, offering a novel approach compared to traditional KRAS inhibitors that only target the G12C mutation.
  • Preclinical studies in mouse models have shown that ACBI3 induces effective tumor regression, validating KRAS degradation as a promising therapeutic strategy.
  • Boehringer Ingelheim has made ACBI3 available to the global research community through its opnMe portal to accelerate innovation in cancer research.

Patient Education Boosts Deprescribing of PPIs, Gabapentin, and Diabetes Meds

  • A patient-directed education intervention, using medication-specific brochures, significantly increased deprescribing of proton pump inhibitors, high-dose gabapentin, and diabetes agents with hypoglycemia risk.
  • The study, a nonrandomized clinical trial across three VA medical centers, involved 5071 patients and 103 primary care practitioners.
  • Deprescribing rates in the intervention cohort were 29.5% compared to 25.8% in the control group, demonstrating a statistically significant improvement.
  • These findings suggest patient-directed education is a promising, low-tech strategy to promote deprescribing of potentially low-benefit and high-risk medications.

Engaging Patients to Promote Deprescribing

NCT04294901CompletedNot Applicable
VA Office of Research and Development
Posted 3/29/2021

Whooping Cough Cases Surge in the U.S., Especially Among Teens

  • The U.S. is experiencing a rapid increase in whooping cough cases, with 14,569 cases reported this year, more than four times higher than last year.
  • Breakthrough infections are rising among older children and adults due to less powerful pertussis vaccines used in recent decades.
  • High school students in states like Pennsylvania and teens aged 15-19 in New York are significantly contributing to the surge in cases.
  • The FDA is considering speeding up the development of more effective pertussis shots, with an advisory panel meeting to discuss potential booster vaccine trials.

Ideaya's Darovasertib Shows Promise in Uveal Melanoma, Advances to Phase 3

  • Ideaya Biosciences' darovasertib demonstrated encouraging Phase 2 results in neoadjuvant uveal melanoma (UM), with approximately 49% of patients showing >30% tumor shrinkage.
  • The trial also reported a ~61% eye preservation rate for enucleation patients, suggesting a potential for improved outcomes in this rare eye cancer.
  • Following a successful FDA Type C meeting, Ideaya is advancing darovasertib to a Phase 3 registrational trial with eye preservation and time to vision loss as primary endpoints.
  • The planned Phase 3 trial will enroll approximately 400 patients and aims to establish darovasertib as a new standard of care for neoadjuvant UM.

(Neo)Adjuvant IDE196 (Darovasertib) in Patients With Localized Ocular Melanoma

NCT05907954RecruitingPhase 2
IDEAYA Biosciences
Posted 7/3/2023

Neoadjuvant/Adjuvant Trial of Darovasertib in Ocular Melanoma

NCT05187884Active, Not RecruitingPhase 2
St Vincent's Hospital, Sydney
Posted 5/3/2022

IDE196 (Darovasertib) in Combination With Crizotinib as First-line Therapy in Metastatic Uveal Melanoma

NCT05987332RecruitingPhase 2
IDEAYA Biosciences
Posted 10/31/2023

Selexipag's Role in Pulmonary Arterial Hypertension: Timing Matters

  • A recent study suggests early exposure to selexipag, an oral prostanoid receptor agonist, is associated with decreased risk for all-cause hospitalization and PAH-related disease progression.
  • The research indicates that the benefits of selexipag are most pronounced when initiated within six months of starting endothelin receptor antagonist and PDE5 inhibitor therapy.
  • The study highlights the importance of timing in selexipag administration, with less benefit observed when added after 12 months of initial therapy for pulmonary hypertension.
  • Real-world data analysis reveals potential discrepancies between clinical trial populations and actual PAH treatment practices, emphasizing the need for careful patient selection.

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