Clinical Trial News

FDA approves Darzalex Faspro (daratumumab and hyaluronidase-fihj) in combination with bortezomib, lenalidomide, and dexamethasone (D-VRd) for induction and consolidation in newly diagnosed multiple myeloma (NDMM) patients eligible for autologous stem cell transplant (ASCT), supported by Phase 3 PERSEUS study data showing significant improvement in progression-free survival (PFS) and deeper responses with D-VRd compared to VRd.

The FDA extended the PDUFA action date for Syndax Pharmaceuticals' NDA for revumenib, a treatment for relapsed or refractory KMT2Ar acute leukemia, to December 26, 2024, due to a major amendment submission. Revumenib aims to address significant unmet needs in KMT2A-rearranged acute leukemia patients.

FDA approves Zunveyl (benzgalantamine) for mild-to-moderate Alzheimer's disease, a novel oral therapy with a dual mechanism of action designed to improve tolerability and efficacy, addressing therapy adherence issues due to GI side effects and insomnia.

Nuvalent's NVL-520, an oral, brain-penetrant, TRK-sparing ROS1 kinase inhibitor, entered Ph. II of ARROS-1 trial for advanced ROS1-positive NSCLC, showcasing a potential best-in-class profile.

Grifols' Xembify®, a 20% subcutaneous immunoglobulin, received FDA approval for an expanded label to include treatment-naïve patients with primary humoral immunodeficiencies (PI), becoming the first 20% SCIg with this extended label. The approval includes biweekly dosing, supported by phase 4 clinical trial data showing non-inferiority in total Ig levels compared to weekly administration. No unique safety issues were identified, and the tolerability profiles were consistent between biweekly and weekly administration. The phase 4 trial was a multicenter, single-sequence, open-label study that included 27 subjects across 18 U.S. sites.

Positive topline data from Phase 3 BROOKLYN trial shows obicetrapib significantly reduces LDL-C by 36.3% at 12 weeks and 41.5% at 52 weeks in HeFH patients, with well-tolerated safety profile. Over 50% achieved LDL-C below 70 mg/dl, and reductions in non-HDL-C, ApoB, and Lp(a) were statistically significant. The trial evaluated 10 mg obicetrapib vs. placebo in 354 patients with HeFH on maximally tolerated lipid-lowering therapy.

FDA approves Erzofri (paliperidone palmitate) for treating schizophrenia and schizoaffective disorder in adults, as monotherapy or adjunct to mood stabilizers/antidepressants. Erzofri, a long-acting injectable developed in China, is the first of its kind to be approved in the U.S., offering improved patient adherence and reduced dosing frequency. It is contraindicated in patients with dementia-related psychosis and known hypersensitivity to paliperidone or risperidone. Safety concerns include increased mortality in elderly patients with dementia-related psychosis, cerebrovascular adverse reactions, neuroleptic malignant syndrome, QT prolongation, tardive dyskinesia, metabolic changes, orthostatic hypotension, falls, leukopenia, neutropenia, agranulocytosis, hyperprolactinemia, cognitive and motor impairment, seizures, dysphagia, priapism, and disruption of body temperature regulation. Common adverse reactions include injection site reactions, somnolence, dizziness, akathisia, and extrapyramidal disorder.

Alector presented baseline characteristics for the INVOKE-2 Phase 2 trial of AL002 at AAIC® 2024, a global trial evaluating AL002's safety and efficacy in early Alzheimer’s disease. AL002, a TREM2 agonist, aims to slow disease progression. The trial enrolled 381 participants with early AD, with baseline characteristics confirming the intended population. Treatment-emergent brain MRI changes resembling ARIA were observed, leading to early discontinuation of homozygous APOE e4 carriers. Results are expected in Q4 2024.

FDA expands approval of Palforzia [Peanut (Arachis hypogaea) Allergen Powder-dnfp] to include individuals ages 1 through 3 years with peanut allergy to mitigate allergic reactions, including anaphylaxis, from accidental exposure. Palforzia, under a REMS, was initially approved in 2020 for ages 4 through 17 years. Users must still avoid peanuts in their diet.

FDA approves Leqselvi (deuruxolitinib), an oral JAK inhibitor for severe alopecia areata treatment.