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Clinical Trial News

Applied Therapeutics Awaits FDA Decision on Govorestat for Galactosemia

  • Applied Therapeutics is seeking FDA approval for govorestat, a novel aldose reductase inhibitor, for the treatment of galactosemia, a rare metabolic disorder.
  • Govorestat aims to reduce the production of galactitol, a toxic metabolite that causes neurological damage in galactosemia patients, with no currently approved therapies.
  • Clinical data from Phase I/II and Phase III trials suggest govorestat can reduce galactitol levels and improve clinical outcomes in both adult and pediatric patients.
  • The FDA's decision on govorestat is anticipated by November 28, marking a potential first-ever approved treatment for this rare condition.

Sage Therapeutics' Dalzanemdor Fails in Huntington's Disease Trial, Pipeline Thins

  • Sage Therapeutics' dalzanemdor failed in the Phase II DIMENSION trial for Huntington's disease, leading to the discontinuation of its development.
  • This setback follows previous failures of dalzanemdor in Parkinson's and Alzheimer's, further impacting Sage's clinical pipeline.
  • Sage is now heavily reliant on Zurzuvae for postpartum depression, with strategic reorganizations aimed at supporting its commercial launch.
  • Analysts express concerns about Sage's path to profitability despite cost-cutting measures and the initial success of Zurzuvae.

FDA Approves BridgeBio's Attruby for Transthyretin Amyloid Cardiomyopathy

• The FDA has approved BridgeBio's Attruby, an oral medication, for treating transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and severe heart disorder. • ATTR-CM is characterized by the accumulation of defective transthyretin proteins in the heart, leading to significant health risks for affected individuals. • Attruby provides a new therapeutic option for adult patients with ATTR-CM, potentially improving outcomes in a treatment landscape previously dominated by Pfizer's Vyndaqel. • The approval marks a significant advancement for BridgeBio, offering hope to patients with this debilitating condition and expanding treatment accessibility.

Cardiol Therapeutics Advances CardiolRx™ for Recurrent Pericarditis to Phase 3 Trial

  • Cardiol Therapeutics is advancing its lead drug, CardiolRx™, to a Phase 3 clinical trial (MAVERIC) for patients with recurrent pericarditis at high risk of recurrence.
  • CardiolRx™ is uniquely positioned to address critical gaps in current treatment options for recurrent pericarditis, potentially offering a safer, more cost-effective, and disease-modifying approach.
  • Phase 2 trial results presented at the American Heart Association showed sustained pain reduction and decreased inflammation markers with CardiolRx™.
  • The FDA has granted Orphan Drug Designation to CardiolRx™ for the treatment of pericarditis, which includes recurrent pericarditis, potentially accelerating its path to market.

Impact of CardiolRxTM on Recurrent Pericarditis (MAvERIC-Pilot)

NCT05494788CompletedPhase 2
Cardiol Therapeutics Inc.
Posted 11/30/2022

Impact of CardiolRx on Myocardial Recovery in Patients With Acute Myocarditis

NCT05180240CompletedPhase 2
Cardiol Therapeutics Inc.
Posted 6/22/2022

Novotech Report Highlights Trends in Global Vaccine Clinical Trials

  • Novotech has released an in-depth whitepaper analyzing the global vaccine clinical trial landscape, offering strategic insights for biotech and pharmaceutical stakeholders.
  • The report highlights the expansion of mRNA platforms and innovative delivery methods, as well as efforts to ensure equitable vaccine access.
  • Key trends include adaptive trial designs, personalized vaccine trials, and a growing role for the Asia-Pacific region in therapeutic vaccine development.
  • The whitepaper also provides insights into venture funding, M&A activity, and streamlined regulatory processes aimed at expediting vaccine approvals.

iiCON-Supported Phase II Trial to Advance Novel Pneumococcal Vaccine, PnuBioVax

  • A new £3.2 million Medical Research Council-funded trial will advance the development of PnuBioVax, a novel vaccine against pneumococcal disease.
  • The phase II study will assess PnuBioVax's ability to prevent pneumococcal carriage in healthy adults via a controlled human infection model (CHIM) trial in Malawi.
  • PnuBioVax, developed by ImmBio, targets serotype 3 pneumococcus (SPN3), a dominant strain with increasing antimicrobial resistance, especially in low- and middle-income countries.
  • The trial aims to establish a low-cost vaccine that can be manufactured and distributed in Africa, potentially blocking community transmission of multiple serotypes.

Novo Nordisk's Semaglutide Shows Promise in NASH Trials

  • Semaglutide, developed by Novo Nordisk, has demonstrated positive results in clinical trials for the treatment of non-alcoholic steatohepatitis (NASH).
  • The competitive landscape for NASH treatments is intensifying, with multiple companies vying for market share in this therapeutic area.
  • Clinical trial activity in the Asia-Pacific region, particularly in China, is increasing, reflecting a growing focus on global pharmaceutical development.

Hanmi's BH3120 Shows Promise in Early Clinical Trials for Advanced Solid Tumors

  • Hanmi Pharmaceutical's BH3120, a novel dual-targeting immunotherapy, is progressing through Phase 1 trials, demonstrating a favorable safety profile in advanced solid tumor patients.
  • The ongoing Phase 1 trial evaluates BH3120 as a monotherapy, with no dose-limiting toxicities observed, suggesting potential for a safer anticancer treatment option.
  • A separate Phase 1 trial is underway, assessing BH3120 in combination with Merck's KEYTRUDA (pembrolizumab), aiming to enhance therapeutic efficacy in advanced or metastatic solid tumors.
  • BH3120 leverages Hanmi's Pentambody platform to target both PD-L1 on cancer cells and 4-1BB on immune cells, potentially bridging immune activation and tumor cell killing.

FDA Study Links Asthma Drug Montelukast to Neuropsychiatric Effects

  • An FDA study reveals that montelukast, commonly known as Singulair, significantly binds to brain receptors crucial for psychiatric function.
  • The research indicates montelukast can penetrate the brain, accumulating in regions associated with psychiatric effects, raising concerns about its impact.
  • Following numerous reports of neuropsychiatric episodes, the FDA added a black box warning in 2020, highlighting potential mental health risks like suicidal thoughts.
  • Scientists emphasize the need for further studies to understand how montelukast's brain binding leads to adverse effects and to identify at-risk individuals.

European Drug Shortages Hit Record Levels: Norway and Generics Most Affected

  • European pharmaceutical markets face unprecedented drug shortages in 2023-2024, driven by inflation, high operating costs, and rigid pricing systems, with Norway reporting 58% of total shortages across seven analyzed countries.
  • Generic medications are experiencing the most severe supply disruptions, with 11% of affected active ingredients reporting shortages in four or more European markets simultaneously.
  • Nervous system medications, particularly analgesics and psycholeptics, represent 25% of shortage-affected products, prompting various European governments to implement flexible pricing policies and regulatory adjustments.

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