Blixeprodil

Malone quits CDC's vaccine panelBeam plots accelerated pathway for AATD base editorTakeda targets $1.3B in cost savingsBiogen signs up for Alteogen's subcutaneous techGilgamesh pockets $60M for neuropsychiatricsMalone quits CDC's vaccine panelRobert Malone, vice chair of the Centers for Disease Control and Prevention's Advisory Committee on Immunization Practices (ACIP), resigned this week."This was not an impulsive decision," he said in a text message quoted in The New York Times. "Hundreds of hours of uncompensated labour, incredible hate from many quarters, hostile press, internal bickering, weaponised leaking, sabotage…I have better things to do."His exit comes after a federal judge ruled last week that the committee — hand-picked by US Department of Health and Human Services (HHS) Secretary Robert F. Kennedy Jr. after summarily firing all 17 members of the previous panel last June — has moved away from making decisions through "a method scientific in nature and codified into law through procedural requirements." The decision suspended the new members' appointments.In his opinion, US District Judge Brian E. Murphy said the newly appointed ACIP members "appear distinctly unqualified" to make vaccine recommendations. He barred the panel from meeting or acting on prior decisions, which include rescinding recommendations for certain childhood vaccines (see – Spotlight On: Court restores vaccine status quo, but expert warns the battle is far from over). HHS has said it will appeal the court ruling, but offered no timeline.-Anna BratulicBeam plots accelerated pathway for AATD base editorBeam Therapeutics on Wednesday shared updated data from a Phase I/II trial evaluating its alpha-1 antitrypsin deficiency (AATD) base editing candidate that it said are sufficient to support an accelerated approval of BEAM-302. At the Feb. 10 data cutoff, findings from 28 evaluable patients showed that a single dose of BEAM-302 led to "rapid and durable" functional AAT increases, as well as decreases in mutant Z-AAT. Patients who received the 60-mg dose — selected as the optimal dose in future studies — achieved a mean steady-state circulating total AAT of 16.1 µM, and a steady-state mean reduction in Z-AAT of 84%.Next half, the biotech plans to enrol 50 patients with AATD-associated lung disease, with or without liver disease, into a pivotal expansion cohort of the open-label Phase I/II trial; the primary endpoint will evaluate AAT biomarkers over 12 months. Based on feedback from the FDA, Beam expects data from that cohort to support a BLA submission for BEAM-302 within the accelerated approval pathway.Beam's pursuit of accelerated approval for BEAM-302 seems aligned with the FDA's recent actions to streamline the regulatory pathway for rare disease drugs — with what appears to be a preference for gene editing candidates over gene therapies (see – Vital Signs: Is the FDA making gene therapy implausible?).-Elizabeth EatonTakeda targets $1.3B in cost savingsTakeda is targeting annual cost savings of JPY 200 billion ($1.3 billion) by fiscal year 2028 as part of plans to "strengthen competitiveness, enhance its long-term growth profile and accelerate launch execution." The move is part of an organisational transformation unveiled by the company in January.On Wednesday, Takeda said that the efficiencies will be achieved by streamlining corporate functions, bringing leadership and teams closer to patients and customers, whilst using advanced technologies to simplify processes. The drugmaker noted that the cost savings will "largely offset" investments needed to prepare for multiple drug launches, including oveporexton, rusfertide and zasocitinib, as well as advancing its late-stage pipeline and making strategic technology investments.While the impact of the transformation on Takeda's workforce was not revealed, the company expects to incur restructuring expenses of approximately JPY 150 billion ($940 million) in the current fiscal year, with lower costs in the following two years.-Matthew DennisBiogen signs up for Alteogen's subcutaneous techBiogen on Wednesday linked up with Alteogen to develop subcutaneous formulations for two of its biologics using the latter's recombinant human hyaluronidase, ALT-B4 (berahyaluronidase alfa).In exchange for exclusive development and commercialisation rights, Biogen will pay Alteogen $20 million upfront. The Korean firm is eligible for $10 million after it begins work on the second product and up to $549 million in combined development, regulatory and sales milestones across both programmes, plus royalties. Biogen also has an option to expand the partnership to a third asset. Developed with Alteogen's Hybrozyme technology, ALT-B4 enables the rapid dispersion and absorption of a co-administered therapeutic by temporarily depolymerising hyaluronan in the extracellular matrix.-Elizabeth EatonGilgamesh pockets $60M for neuropsychiatricsAfter last year off-loading psychedelic depression candidate bretisilocin to AbbVie in a deal worth up to $1.2 billion, Gilgamesh Pharma on Tuesday raised $60 million to fund the development of its remaining neuropsychiatric assets (see – Spotlight On: Next-gen psychedelics approach another inflection point). The series A was led by Satori Neuro with participation from Prime Movers Lab, which led earlier rounds for Gilgamesh prior to the spinout. The capital will support blixeprodil (GM-1020), its non-competitive NMDA receptor antagonist, as well as GM-3009, a cardio-safe ibogaine analogue. In January, Gilgamesh reported top-line data from a Phase IIa study in patients with major depressive disorder showing that blixeprodil led to a clinically meaningful and statistically significant reduction in the Montgomery-Åsberg Depression Rating Scale total score 24 hours after administration versus placebo.This year, the company plans to move blixeprodil into late-stage studies and begin Phase I testing of GM-3009.-Elizabeth Eaton