Phase 1

A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.
Recent approvals for Corcept Therapeutics and Merck have injected momentum into the space, where GSK, Allarity Therapeutics, OSE Immunotherapies and others are advancing their own candidates.
Encouraging data for combination regimens of Revolution Medicines’ zoldonrasib “reinforce the path to leadership in PDAC” for the biotech, according to Truist Securities.
As uniQure prepares its closely watched Huntington’s disease gene therapy for FDA review, the biotech is also making waves in chronic epilepsy with another gene therapy that Stifel says is off to a “promising start.”
Following the mid-stage readout, Verastem Oncology has decided to move away from its investigational pancreatic cancer drug combo, instead putting resources behind its KRAS inhibitor pill for solid tumors.
Eli Lilly’s new JAK2 inhibitor—which it obtained from the recent acquisition of Ajax Therapeutics—reduced spleen volume by more than a third in 70% of patients with myelofibrosis.
DemeRx is launching the first U.S. clinical trial of an ibogaine-derived drug candidate, marking a pivotal moment for a controversial psychedelic long sidelined by safety concerns.
The RNA-based medicine is one of a handful of antibody-oligonucleotide conjugates that Novartis acquired last October when it took over neuromuscular-focused Avidity Biosciences.
All six non-Hodgkin lymphoma patients on Legend Biotech’s CAR T therapy responded to treatment—findings that could make the biotech an attractive takeover target, according to analysts at Oppenheimer.
Nearly 80% of patients saw tumor shrinkage after being treated with Kura Oncology’s darlifarnib plus Bristol Myers Squibb’s Krazati, findings Mizuho analysts say could open up a $2 billion opportunity for the biotech.
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