BioSpace

FDA Reversals in Rare Disease Space Highlight Confusion Around External Controls

While recent FDA guidance speaks to the agency’s support of innovative trial designs—including the use of external ...
Medscape

Early Acoramidis Yields Long-Term Gains in ATTR-CM

Early and continuous treatment with acoramidis is linked to lower risks for death and heart-related hospitalization than delayed treatment in patients with transthyretin amyloid cardiomyopathy.

Open Label Outpatient Switch Study Demonstrates Symptom Stability During Transition from Oral Atypical Antipsychotics to Cobenfy™ (xanomeline and trospium chloride)

Open Label Outpatient Switch Study Demonstrates Symptom Stability During Transition from Oral Atypical Antipsychotics to Cobenfy.
Nasdaq

Supernus Announces Promising Data from Open-Label Phase 2a Study of SPN-820 in Adults with Major Depressive Disorder

“These Phase 2a data underscore our belief that SPN-820 has the potential as a novel treatment option for patients with depression, with the opportunity to decrease symptoms quickly and without ...
Managed Healthcare Executive

Attruby reduces risk of death by 49% in rare heart condition in long-term study

A long-term extension trial of 389 patients found Attruby significantly cut death risk in transthyretin amyloid ...

Palisade Bio Shares Phase 1b PALI-2108 Data Showing Safety, PK and Early Crohn’s Efficacy Signals

Palisade Bio (NASDAQ:PALI) said it generated encouraging safety, pharmacokinetic and early efficacy signals from an open-label Phase 1b study of PALI-2108 in fibrostenotic Crohn’s disease, a difficult ...
ascopubs.org

An open-label study to assess the safety and efficacy of naporafenib (ERAS-254) administered with trametinib in previously treated patients with locally advanced unresectable ...

177Lu-edotreotide versus everolimus in patients with advanced neuroendocrine tumors of lung or thymic origin: The phase 3 randomized LEVEL, GETNE-T2217 trial. This is an ASCO Meeting Abstract from the ...
Seeking Alpha

Atai Beckley N.V. (ATAI) Discusses BPL-003 Phase IIb Open-Label Extension Study Results in Treatment Resistant Depression Transcript

Thank you for standing by. My name is Tina, and I will be your conference operator today. At this time, I would like to welcome everyone to the Atai Beckley BPL-003 phase IIb open-label extension ...
Nasdaq

Larimar Therapeutics Announces Positive Data from Ongoing Long-term Open Label Study and Updates to Nomlabofusp Program for Friedreich’s Ataxia

BALA CYNWYD, Pa., Sept. 29, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare ...
ascopubs.org

STELLAR: Phase III, Randomized, Open-Label Study of Eflornithine Plus Lomustine Versus Lomustine Alone in Patients With Recurrent Grade 3 Astrocytoma

TBCRC 048 (Olaparib Expanded) Expansion Cohorts: Phase II Study of Olaparib Monotherapy for Patients With Metastatic Breast Cancer With Germline Mutations in PALB2 or Somatic Mutations in BRCA1 or ...
Seeking Alpha

Larimar Therapeutics Announces Positive Data from Ongoing Long-term Open Label Study and Updates to Nomlabofusp Program for Friedreich’s Ataxia

In 4 completed studies and the ongoing OL study, 65 participants received at least 1 dose of nomlabofusp, including 39 in the OL study, with 14 on treatment for at least 6 months and 8 for over 1 year ...