Wave Life Sciences Ltd. this week highlighted during its investor research day activin E and INHBE-targeting WVE-007, a GalNAc-siRNA therapy for obesity. The phase I study called Inlight turned up potent, durable, and dose-dependent activin E reductions with single doses of WVE-007 in the first three cohorts, the Cambridge, Mass.-based firm said. Benefiting from the “wave” of notice for the mechanism of action was Ibio Inc. with its early-stage activin E weight-loss prospect IBIO-610, for which the firm rolled out favorable pharmacokinetic findings.

Vesper's oral frontotemporal dementia drug positive in phase Ib/II

Neurodegeneration specialist Vesper Bio ApS announced positive results from the phase Ib/II trial of its oral sortilin inhibitor VES-001 in frontotemporal dementia (FTD). The small study involved six participants who had not progressed to symptomatic disease but were carriers of mutations in the GRN gene that codes for progranulin, a growth factor that is essential for neuronal health. Following daily dosing with VES-001 over three months, mean progranulin levels increased by more than 95% compared to baseline at the highest dose tested. There were dose-dependent increases in progranulin levels in both plasma and cerebrospinal fluid, demonstrating target engagement. “We were able to increase it by up to 100%, so two-fold. And that is really important, because we know that these patients are heterozygous mutants; they have around 50% of the protein levels of healthy individuals,” said Jacob Falck Hansen, Vesper CEO.

Boehringer licenses Kyowa Kirin small molecule in autoimmune disease

Boehringer Ingelheim GmbH has licensed an unnamed small-molecule preclinical candidate from Kyowa Kirin Co. Ltd. in the autoimmune disease space in a deal worth up to €640 million (US$739 million). Under terms of the deal, Tokyo-based Kyowa Kirin is eligible to receive up to €640 million, including an undisclosed up-front payment, as well as development, regulatory, and commercial-based milestone payments, and sales-based royalties. In exchange, Boehringer Ingelheim, of Ingelheim, Germany, gains exclusive global rights to develop the small-molecule program that has the potential to be first-in-class, according to the two companies.

Vera Therapeutics on the march, CD19 still in the limelight as well

As one analyst pegged Vera Therapeutics Inc.’s atacicept due to become “one of the big renal launches” in the year ahead, others on Wall Street are speculating about the prospects for the immunoglobulin A nephropathy (IgAN) drug lifting other ships, such as that of Climb Bio Inc., investigating CLYM-116, a preclinical-stage anti-APRIL monoclonal antibody (mAb) for patients with IgAN and other B-cell mediated afflictions. But a piece of the Climb story just as intriguing is budoprutug, a clinical-stage mAb designed to deplete CD19-positive B cells, including antibody secreting cells (plasma blasts), in order to directly reduce pathogenic autoantibodies in various immune-mediated disease. CD19 as a target has been the subject of recent deals and continues to hold appeal for developers.

Better DNA repair helps bowhead whales live longer, cancer free

Bowhead whales (Balaena mysticetus) live year-round in the icy or near-icy waters of the Arctic and sub-Arctic. Although they migrate with the seasonal cycles of ice formation and melting, they never reach the warmer waters visited by other large marine mammals. These whales can grow over 15 meters long, weigh up to 100 tons, and use their strong heads to break through ice up to one meter thick, like living icebreakers. Their adaptation to low temperatures may have also enabled them to live longer and avoid cancer, a disease closely linked to aging. A study on this cetacean highlights its remarkable DNA damage repair capacity and genomic stability as key factors behind this evolutionary success.

First mover in MYC condensate therapy showcased at AACR-NCI-EORTC

At the AACR-NCI-EORTC conference, researchers from Dewpoint Therapeutics Inc. presented advances in targeting MYC condensates, revealing a potential breakthrough strategy for treating cancers driven by MYC – a well-established oncogenic driver that is frequently overexpressed or amplified across a range of human cancers. “MYC is often considered a ‘holy grail’ target, one that has been notoriously difficult to drug due to its highly disordered structure,” Ann Boija, head of research of Dewpoint Therapeutics Inc., told BioWorld. Boija explained that the MYC program originated from the insight that many transcription factors act within biomolecular condensates, dynamic phase-separated structures that orchestrate critical cellular functions.

Also in the news

89bio, Aboleris, Akari, Alethio, Amnea, Anavex, Arbor, Argenx, Avobis, Brenig, Cartesian, Celltrion, Cocrystal, DBV, Dewpoint, Dianthus, Evaxion, Evommune, Everest Medicines, Helex, Innocare, Intellia, Johnson & Johnson, Mind Medicine, Neutrolis, Novartis, Oculis, Preveceutical, Roche, Savara, Sensei, Tectonic, Tolremo, Tubulis, Unicycive, Valanx, Vaxil, Vor, Wave