Having notched a second phase III win in major depressive disorder (MDD) with lumateperone, Intra-Cellular Therapies Inc. stands in good shape to submit a supplemental NDA in the second half of this year. First approved by the U.S. FDA as Caplyta in late 2019, the atypical antipsychotic is used for schizophrenia and bipolar depression. Abbvie Inc.’s drug in the same class, Vraylar (cariprazine), gained its MDD label expansion in December 2022, and has chalked significant sales upticks since. Vraylar was first cleared in September 2015 and is indicated for schizophrenia and bipolar I disorder along with MDD. The markets are closed for Juneteenth, but shares of New York-based Intra-Cellular (NASDAQ:ITCI) closed June 18 at $75.48, up $6.61, having traded as high as $78.53.

Kyverna’s CAR T improves mobility in stiff-person disease case study

Coming off a presentation at the European Alliance of Associations for Rheumatology meeting, where a single report of disease recurrence in a lupus patient overshadowed promising early data for Kyverna Therapeutics Inc.’s autoimmune candidate, KYV-101, the Emeryville, Calif.-based company rallied on news of a case report describing the first use of the CD19-targeting CAR T-cell therapy in a patient with stiff-person syndrome (SPS), a rare, progressive neurological autoimmune disorder for which there is limited treatment. Results from a 69-year-old patient with treatment-refractory SPS, reported in the Proceedings of the National Academy of Sciences, showed CAR T-cell infusion resulted in improvements in mobility, including a walking speed increase of more than 100%.

Companies to Watch: Seven ADC companies under the radar

The annual Companies to Watch report, which was just released, looks closely at seven companies flying under the radar that are developing antibody-drug conjugates (ADCs) for treating cancer. ADCs have become much sought after by big pharmas eager to incorporate the new and successful technology into their portfolios. Companies examined in the new report are Adcendo ApS, Araris Biotech AG, Go Therapeutics Inc., Heidelberg Pharma AG, Pheon Therapeutics Ltd., Tallac Therapeutics Inc. and Tubulis GmbH. The annual report, a companion to the annual Drugs to Watch report, comes from Clarivate plc, which publishes BioWorld. Previous Companies to Watch reports have highlighted RNA technology and artificial intelligence/machine learning companies.

Biotron’s BIT-225 meets endpoints in phase II HIV trial

Preliminary data show Biotron Ltd.’s lead compound BIT-225 met the primary objectives of the phase II BIT225-011 trial, a longitudinal, open-label trial designed to characterize the effect of the compound added to ongoing, suppressive antiretroviral (ARV) therapy in HIV-1-infected, treatment-experienced patients who achieved only partial immune reconstitution. The primary endpoints of the trial were to determine the impact of BIT-225, an HIV-1 Vpu inhibitor, when added to ARV treatment, on immune activation, inflammation and viral markers, as well as safety and tolerability in this patient population.

EHA 2024: Movers and shakers in the AML landscape

During the basic science morning track on the last day of this year’s Annual Congress of the European Hematology Association (EHA), the attention was focused on oncogenic transcription factors and complexes considered turning points within the acute myeloid leukemia (AML) arena. The first talk was given by Prof. Ruud Delwel from Erasmus MC Cancer Institute, who has been working on the EVI1 gene in the AML environment for decades. Delwel’s major discoveries include the unveiling of a subset of unique AML cases with aberrant expression of the EVI1 gene, demonstrating a specific mechanism of enhancer deregulation.

Anti-PD-1 therapy triggers microglia, causing CNS side effects

The adverse effects of PD-1 blockers on the CNS observed in cancer patients could occur through their effects on an enzyme that activates microglia. Pharmacological inhibition of the enzyme in mice reduced microglial activation and cognitive deficit without altering the antitumor capacity of the immunotherapy. “Immunotherapy approaches that rely on immune checkpoint inhibition have improved the outcome of cancer patients. The Nobel Prize was given to [Tasuku Honjo and James Allison], the discoverers of PD-1 and CTLA-4 [cytotoxic T-lymphocyte antigen-4], the main targets for checkpoint inhibition. However, there are also downsides to this therapy,” Robert Zeiser, a professor at the Department of Medicine I at the University of Freiburg, told BioWorld.

Also in the news

Ability, Advanz, Altus, Alvotech, Amgen, Astrazeneca, Avidity, Bavarian Nordic, Bridgebio, Bristol Myers Squibb, Celon, Cel-Sci, Clene, Clover, Dermtech, Enlaza, Ethris, Hikma, Iambic, Intra-Cellular Therapies, Ionis, Italfarmaco, Jasper, Junshi, Kalvista, Kyverna, Merck, Mitsubishi Tanabe, Otsuka, Primrose, Pyros, Recce, Relmada, Santhera, Spektar, Takeda, Taysha Gene Therapies, TME, Urogen, Xellia, Zentalis