Here are the latest stories being discussed in biopharma today:
## Silence’s siRNA Candidate for Cardiovascular Disease Continues to Deliver in Phase 2 Trial
Silence Therapeutics has shared promising data from a Phase 2 trial of its siRNA therapy candidate, zerlasiran, for patients at risk of cardiovascular events. Designed to lower lipoprotein(a) or Lp(a) levels—a significant genetic risk factor for cardiovascular disease—the candidate showed ‘highly significant’ reductions compared to placebo after 48 weeks. This adds to the positive outcomes seen at 36 weeks, where the treatment achieved a median reduction of 90% or more in Lp(a) levels.
## PTC Touts Phase 2 Biomarker Data for Huntington’s Drug, Showing Dose-Dependent Effect at One Year
PTC Therapeutics announced interim results from a Phase 2 study of PTC518 for Huntington’s disease, presenting compelling biomarker data. The drug demonstrated a dose-dependent reduction of the mutant huntingtin protein (mHTT). At a 10 mg dose, patients had a 43% reduction in bloodstream mHTT after one year, while those on the 5 mg dose saw a 22% reduction. The placebo group, contrasted by a 14% increase, underscored the drug’s potential impact.
## Jazz’s Stock Dips After Mid-Stage Fail in Essential Tremor
Jazz Pharmaceuticals faced a setback with its drug suvecaltamide failing to achieve a statistically significant improvement in essential tremor in a Phase 2b trial. The highest dosage of 30 mg did not show the expected changes compared to placebo on a severity scale. While numeric improvements were observed, the placebo response surpassed expectations, contributing to a stock dip of over 4%.
## Gilead Tested an Antibody Many Times. A Small Biotech Salvages It, with $40M and ‘Conviction’ Elsewhere
A small biotechnology company, āshibio, is resurrecting an antibody previously discarded by Gilead after several unsuccessful trials. With $40 million in initial funding, āshibio plans to repurpose andecaliximab (formerly GS-5745) to treat fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder affecting soft tissue and bone formation. After Gilead’s numerous attempts in various indications, āshibio is pushing forward with new hopes for this treatment.
## Aphaia Hits Primary Endpoint with Oral Glucose Drug in Mid-Stage Prediabetes Trial
Aphaia Pharma’s oral glucose treatment, APHD-012, has met its primary endpoint in a Phase 2 prediabetes trial. Intended to prevent the progression to type 2 diabetes, APHD-012 showed a statistically significant improvement in glucose tolerance over six weeks. The study included various patient groups and reported no serious adverse events, solidifying its potential as an early-intervention metabolic therapy.
## Forbion’s New €75M Fund; Cara Therapeutics to Cut Workforce After Trial Failure
– **Forbion’s New Fund:** Dutch VC firm Forbion has closed its €75 million BioEconomy Fund I to invest in sustainable startups across the food, agriculture, materials, and environmental tech sectors.
– **Cara Therapeutics Staff Cuts:** Following the failure of its pruritus drug in a mid-phase trial, Cara Therapeutics is reducing its workforce by approximately 70%. This strategic restructuring follows disappointing Phase 2/3 trial results for difelikefalin, prompting the company to discontinue the program and explore new strategic alternatives.