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Leqembi's Slow Adoption: Challenges and Progress in Alzheimer's Treatment

Monoclonal AntibodyDrug ApprovalNeurology
  • Leqembi, the first FDA-approved drug shown to slow Alzheimer's progression, has experienced slower-than-expected adoption despite its groundbreaking status in treating the disease.
  • Healthcare systems face significant implementation challenges, including establishing diagnostic protocols, monitoring systems for side effects, and navigating complex insurance coverage issues for the $26,000-per-year treatment.
  • Despite modest clinical benefits and potential side effects like brain swelling and bleeding, some patients report improvements in short-term memory, offering hope as researchers continue developing easier administration methods and complementary treatments.

FDA Clears Path for Cingulate's CTx-1301 ADHD Treatment NDA Submission

Neurology
  • The FDA has confirmed that Cingulate can proceed with its New Drug Application for CTx-1301, a novel ADHD treatment, without completing additional Phase 3 studies.
  • CTx-1301 utilizes Cingulate's proprietary Precision Timed Release (PTR) technology to deliver three precisely timed doses of dexmethylphenidate in a single daily tablet, potentially benefiting over 60% of ADHD patients currently using booster doses.
  • Cingulate plans to submit the NDA in the first half of 2025 and is advancing discussions with potential commercialization partners while preparing to engage with payers for market access.

FDA to Convene Advisory Committee for Eli Lilly's Alzheimer's Drug Donanemab

Monoclonal AntibodyNeurologyPrecision Medicine
• The FDA has announced plans to convene a Peripheral and Central Nervous System Drugs Advisory Committee to review donanemab's efficacy and safety profile before making a final approval decision.
• Donanemab's TRAILBLAZER-ALZ 2 trial employed innovative biomarker strategies, using both Amyvid® and Tauvid™ PET scans to identify early-stage patients with confirmed amyloid plaques most likely to benefit from treatment.
• Experts view anti-amyloid therapies like donanemab as just the first line of defense, with approximately 75% of Alzheimer's drugs in development now exploring novel targets related to aging pathways including inflammation and vascular dysfunction.

Cingulate's Phase 3 Trial Shows Promising Results for Once-Daily ADHD Treatment CTx-1301

Neurology
  • Cingulate Inc. presented full results from its Phase 3 adult efficacy and safety trial of CTx-1301 (dexmethylphenidate) for ADHD at the 36th Annual Psych Congress, with the poster selected as a finalist for the inaugural Poster Awards.
  • CTx-1301 demonstrated a trend toward significant improvement in ADHD symptoms with rapid onset and entire active-day duration, potentially offering the first true once-daily stimulant medication with no mid-day dosing requirements.
  • The company has initiated additional Phase 3 studies in pediatric and adolescent populations, with plans to submit a New Drug Application to the FDA in the second half of 2024 under the Section 505(b)(2) pathway.

Phase 3 Efficacy and Safety Study in Adults With ADHD Using CTx-1301.

NCT05631626CompletedPhase 3
Cingulate Therapeutics
Posted 12/29/2022

First Veteran Receives FDA-Approved Qalsody for SOD1-ALS at Houston VA Medical Center

NeurologyRare DiseaseDrug ApprovalFDA Approval
  • Roy Swearingen, a 65-year-old Marine Corps veteran from Texas, became the first veteran in the United States to receive Qalsody, an FDA-approved medication for SOD1-mutated ALS.
  • Qalsody represents the first approved treatment targeting a genetic cause of ALS, specifically for patients with mutations in the superoxide dismutase 1 (SOD1) gene, affecting 1-2% of ALS patients.
  • The Houston VA ALS Center's rapid implementation of this cutting-edge therapy demonstrates the VA's commitment to providing veterans with timely access to innovative treatments.
  • Veterans face higher ALS risk compared to the general population, making early detection and specialized care crucial for optimal quality of life management.

U.S. Tuberous Sclerosis Drug Market Projected to Reach $1.25 Billion by 2030, Growing at 23.1% CAGR

Drug ApprovalNeurologyRare Disease
  • The U.S. tuberous sclerosis drug market is expected to grow from $237.2 million in 2022 to $1.25 billion by 2030, driven by FDA approvals of new treatments and increasing disease prevalence.
  • FDA-approved treatments include Afinitor (everolimus) for patients aged 2 and above, approved in 2018, and Epidiolex (cannabidiol) for patients aged 1 and above, approved in 2020.
  • mTOR inhibitors are gaining significant traction in treatment protocols, showing effectiveness for internal tumors and skin lesions, with FDA approval for treating lung complications like Lymphangioleiomyomatosis (LAM) in 2015.

FDA Approves Aduhelm: First Disease-Modifying Alzheimer's Treatment in Nearly Two Decades

Drug ApprovalMonoclonal AntibodyNeurology
  • The FDA granted accelerated approval to Biogen's aducanumab (Aduhelm) as the first disease-modifying Alzheimer's treatment in nearly two decades, despite divided expert opinions on its clinical efficacy.
  • Aduhelm, priced at $56,000 annually, works by reducing amyloid plaques in the brain and will require monthly intravenous infusions with comprehensive clinical and MRI monitoring for potential adverse effects.
  • As part of the accelerated approval, Biogen must conduct a Phase 4 confirmatory trial, while experts view this approval as a potential catalyst for developing more effective Alzheimer's treatments targeting multiple pathways.

A Study of Aducanumab in Participants With Mild Cognitive Impairment Due to Alzheimer's Disease or With Mild Alzheimer's Disease Dementia to Evaluate the Safety of Continued Dosing in Participants With Asymptomatic Amyloid-Related Imaging Abnormalities

NCT03639987TerminatedPhase 2
Biogen
Posted 12/20/2018

221AD302 Phase 3 Study of Aducanumab (BIIB037) in Early Alzheimer's Disease

NCT02484547TerminatedPhase 3
Biogen
Posted 9/15/2015

A Study to Evaluate Safety and Tolerability of Aducanumab in Participants With Alzheimer's Disease Who Had Previously Participated in the Aducanumab Studies 221AD103, 221AD301, 221AD302 and 221AD205

NCT04241068TerminatedPhase 3
Biogen
Posted 3/2/2020

221AD301 Phase 3 Study of Aducanumab (BIIB037) in Early Alzheimer's Disease

NCT02477800TerminatedPhase 3
Biogen
Posted 8/13/2015

Multiple Dose Study of Aducanumab (BIIB037) (Recombinant, Fully Human Anti-Aβ IgG1 mAb) in Participants With Prodromal or Mild Alzheimer's Disease

NCT01677572TerminatedPhase 1
Biogen
Posted 10/5/2012

ApTOLL: Novel Aptamer-Based Therapy Shows Promise for Acute Ischemic Stroke Treatment

Neurology
  • aptaTargets is advancing ApTOLL, an innovative aptamer-based immune modulator that reduces brain damage in acute ischemic stroke by targeting TLR4 receptors.
  • Preclinical studies demonstrated ApTOLL's ability to reduce brain damage by up to 65% with an effective treatment window of 12 hours post-stroke onset.
  • Following successful Phase I trials in healthy volunteers (NCT04742062), the company is now conducting a Phase Ib/IIa clinical trial (APRIL study, NCT04734548) to establish proof-of-concept in stroke patients.

First in Human Clinical Trial of ApTOLL in Healthy Volunteers

NCT04742062CompletedPhase 1
aptaTargets S.L.
Posted 7/18/2019

Phase Ib/IIa Clinical Study of ApTOLL for the Treatment of Acute Ischemic Stroke

NCT04734548CompletedPhase 1
aptaTargets S.L.
Posted 10/28/2020

Oasis Diagnostics Receives Michael J. Fox Foundation Grant for Saliva-Based Parkinson's Test

Neurology
  • Oasis Diagnostics has been awarded a research grant from The Michael J. Fox Foundation to validate their non-invasive, saliva-based rapid test for Parkinson's disease detection and diagnosis.
  • The company will collaborate with Dr. Charles Adler's team at Mayo Clinic in Scottsdale to study proprietary salivary biomarkers, with research suggesting that just three of 50 identified biomarkers can provide accurate diagnosis.
  • The VerOFy® Rapid Test Platform could potentially transform Parkinson's diagnosis, offering a non-invasive alternative to current expensive and invasive diagnostic methods while potentially accelerating clinical trials of disease-modifying treatments.

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