Cambridge, Mass.-based Korro Bio Inc. pulled down $91.5 million in a series A financing to advance its platform for precise, single-base, RNA editing to modulate protein function. The money will let Korro get its lead program to an IND filing and establish a portfolio of RNA-editing therapies. Wu Capital led the round with participation from current investors, Atlas Venture and New Enterprise Associates. New investors include Qiming Venture Partners USA, Surveyor Capital (a Citadel company), Cormorant Asset Management, MP Healthcare Venture Management and Alexandria Venture Investments.

Rockin’ the Casma: $50M series B comes its way

Casma Therapeutics Inc., of Cambridge, Mass., has completed a $50 million series B financing designed to develop its TRPML-1 agonist program for treating muscular dystrophy and refine its autophagy degrader platform. Autophagy is the natural process by which cells break down surplus or dysfunctional protein, organelles such as mitochondria, and viruses and other pathogens. The company said it wants to arrest or reverse the progression of lysosomal storage disorders, muscular disorders, cancer, inflammatory disorders and neurodegeneration by boosting autophagy. Third Rock Ventures launched Casma in 2018 and also participated in the series B.

Mina Therapeutics closes $30M A round for RNA-based gene expression activators

DUBLIN – Mina Therapeutics Ltd. raised £23 million (US$29.9 million) in a series A round to take forward a pipeline and a platform based on a novel regulatory RNA species, small activating RNAs, which selectively activate gene expression. The London-based firm is not exactly a newcomer – it was founded more than a decade ago and has been pursuing its present strategy for the last seven years. Its lead drug candidate, moreover, is already undergoing two separate combination trials. But it is taking on venture capital now following the recent publication of clinical proof-of-concept data for its lead drug, MTL-CEBPA, in advanced liver cancer.

Satsuma suffers phase III setback for acute migraine candidate

A pivotal test of Satsuma Pharmaceuticals Inc.'s dry powder acute migraine candidate, STS-101, found neither of two doses evaluated freed people from pain or their most bothersome symptom two hours post-administration, the study's co-primary endpoints. The outcome, confounding since the drug was thought to be fast-acting but performed better at a later time-point in the study, sent shares in the South San Francisco-based company (NASDAQ:STSA) down 66.4% by midday.

Constellation readying phase III with BET inhibitor; lengthened runway for Jakafi?

As Constellation Pharmaceuticals Inc. gears up to start the pivotal phase III test of CPI-0610 in myelofibrosis, Wall Street is mulling how likely are the stars to align for the Cambridge, Mass.-based company with its small-molecule inhibitor of bromodomain and extra-terminal (BET) proteins. In December at the American Society of Hematology meeting, Constellation offered data from the phase II effort called Manifest in MF, weighing CPI-0610 when combined with JAK inhibitor Jakafi (ruxolitinib) from Incyte Corp., of Wilmington, Del. H.C. Wainwright analyst Andrew Fein said in a report that the available data package “continues to support” the combo, and CPI-0610’s hematologic effects “should significantly extend and expand [Jakafi’s] clinical utility in the future.”

GTEx explores variation in genome bureaucracy

The Genotype-Tissue Expression (GTEx) project, a multiyear, multi-institutional attempt to catalog how expression quantitative trait loci (eQTL) and splicing quantitative trait loci (sQTL) affect protein levels, reported data from its final phase in 15 papers in the Sept, 10, 2020, online issues of the Science and Cell family of journals, as well as in Genome Biology. Collectively, the findings report multiple new insights into how protein expression levels can vary by genotype, sex, genetic ancestry, tissue type and other factors.

Hanmi considers options with diabetes candidate efpeglenatide back in-house

HONG KONG – Sanofi SA has finally ended its clinical development of Hanmi Pharmaceutical Co. Ltd.’s efpeglenatide as a diabetes treatment. Sanofi had informed Seoul, South Korea-based Hanmi of its intention to do so in May. The French pharma conglomerate was conducting phase III trials for the candidate on 6,000 patients at 300 clinical centers in 30 countries. The reason for the termination was apparently not related to the candidate’s potential, and Hanmi is now considering how to move on with developing the once-weekly GLP-1 receptor agonist that employs Hanmi's Lapscovery delivery technology.

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