Shots:
OLE study assessed repinatrabit (JNT-517) in adolescents with PKU. Early time-point data remain embargoed to preserve parent-study blinding, with additional randomized-period results to be presented later
First cohort receiving repinatrabit (75mg, PO, BID) showed a 67% mean reduction in Phe from baseline (Day 56/OLE Month 1), with responses seen in all pts, incl. prior…
Shots:
Enodia has acquired Kezar’s Sec61-based discovery & development program assets, strengthening its understanding of Sec61 selectivity mechanisms & expanding translational insights to accelerate progress toward key clinical milestones
As per the deal, Kezar will receive a $1M upfront, ~$127M in development, regulatory, & commercial milestones, plus tiered royalties on future net sales
Enodia’s platform…
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PRISM has partnered with Receptor.AI to develop an AI-navigated, physics-guided platform for discovering orally available small molecules targeting intracellular protein–protein interactions (PPIs), membrane proteins & complex receptor systems
Collaboration will combine PRISM’s PepMetics Tech, 3D small molecules designed to mimic α-helix & β-turn motifs, with Receptor.AI’s AI-driven molecular design engine to identify hit, lead, &…
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Ultragenyx has reported the P-III (Enh3ance) study data assessing DTX301 AAV8 gene therapy vs PBO for the treatment of ornithine transcarbamylase (OTC) deficiency
DTX301 reduced 24hr. plasma ammonia by 18% vs PBO at Wk. 36, with ammonia levels maintained in the normal range, despite a 27% mean decrease in scavenger drugs & ~13% increase…
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Natera has reported the commercial launch of Zenith genomics, a next-generation whole genome sequencing (WGS) assay designed to enhance the detection of rare diseases in the US
The platform combines whole genome sequencing with long-read sequencing confirmation to detect genomic features such as tandem repeat expansions, supporting diagnosis of rare & ultra-rare conditions, &…
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Aplagon has dosed the first patient with APAC (IV) in its P-IIa (HEALING) trial in Finland for peripheral arterial occlusive disease (PAOD), leading to chronic limb-threatening ischemia (CLTI)
Trial will evaluate APAC in ~42 CTLI pts across 4 cohorts to assess safety, efficacy, & effects on thrombo-inflammatory biomarkers. Additionally, an associated PET imaging study with…
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Insulet has presented data from the EVOLUTION 2C feasibility study (n=24, 16-70yrs.) assessing its fully closed-loop (FCL) automated insulin delivery system (AID) for T2D at the ATTD’26
Trial showed that the final FCL algorithm increased TIR to an average of 68% with no boluses, a 24% improvement vs standard injection therapy, while TBR remained…
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The EMA has received the Type II variation application for Tecvayli monotx. (teclistamab) for the treatment of adults with r/r multiple myeloma (RRMM) who have received ≥1 prior therapy
Application was supported by the P-III (MajesTEC-9) trial assessing Tecvayli vs SoC of pomalidomide, bortezomib, & dexamethasone (PVd) or carfilzomib & dexamethasone (Kd) in 614…
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George Medicines has entered into an exclusive licensing & supply agreement with Ahngook Pharmaceutical, granting rights to seek regulatory approval & commercialize GMRx2 in Korea for the treatment of Hypertension
As per the deal, George Medicines will receive an upfront licensing fee, potential regulatory & commercial milestone payments, as well as stepped royalties on…
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C2N Diagnostics has entered into an agreement with BeauBrain Healthcare to offer PrecivityAD2 blood test for use in pts (≥50yrs.) with signs or symptoms of mild cognitive impairment or dementia in South Korea
Study in The JAMA showed PrecivityAD2 improved diagnostic accuracy of Alzheimer’s disease vs SoC, while research in npj Dementia showed that the test demonstrated 91%…

