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BioWorld - Tuesday, March 24, 2026
Home » Newsletters » BioWorld

BioWorld

Feb. 28, 2019

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No limb-girdle MD hurdle for gene therapy; Sarepta pays $165M for Myonexus

Sarepta Therapeutics Inc. CEO Doug Ingram told Wall Street that the firm "has couple of things we've got to get done very soon and report back to you on," given early results in the first three-patient cohort of the MYO-101 study in patients with limb-girdle muscular dystrophy (LGMD) known as LGMD2E – strongly positive results that triggered an early exercise of the option to acquire Myonexus Therapeutics Inc. for $165 million. Read More

Blackstone launches CVD-focused Anthos with $250M investment

Anthos Therapeutics Inc., a new company formed with a $250 million investment from Clarus Ventures successor Blackstone Life Sciences, has in-licensed an antithrombotic agent shelved by Novartis AG. The asset, MAA-868, has the potential to prevent a variety of cardiovascular disorders (CVD) with minimal or no bleeding risk within a new long-acting treatment paradigm, the company said. John Glasspool, a former leader of Novartis' cardiovascular franchise, will lead the company as its CEO and Novartis will hold a minority stake in the company. Read More

Firms worry about lack of biosimilar policy support as China races ahead

HYDERABAD, India – As China surges ahead in the biosimilars space, Indian biopharma companies are increasingly concerned about their ability to keep pace due to lack of policy support and pricing controls. Read More

Brewer's yeast can make natural, and unnatural, cannabinoids

Synthetic biologists have engineered brewer's yeast to make cannabinoids, opening up the possibility of wider investigation of the pharmaceutical properties of chemicals that occur in very small amounts in the marijuana plant, Cannabis sativa. Read More

NIH study finds epigenetic 'clocks' can aid prediction of breast cancer risk

Genetics can only tell us so much when it comes to understanding cancer. It turns out that interrogating the myriad processes involved in the translation of DNA into actual bodily processes, as well as alterations over time, are crucial. To that end, researchers at the NIH are pursuing a more refined understanding of epigenetics, which is defined by how biochemical processes work to modify gene expression. Read More

Regulatory front

The U.S. Court of Appeals for the Third Circuit this week rejected the FTC's bid to expand its ability to pursue a temporary restraining order (TRO) or permanent injunction for violations of the FTC Act based solely on conduct that happened years ago.  Read More

Appointments and advancements

Notable Labs Inc., of Foster City, Calif., appointed Hiroomi Tada chief medical officer and Lloyd Mackenzie chief development officer. Read More

Financings

Aurinia Pharmaceuticals Inc., of Victoria, British Columbia, said it completed its at-the-market sale of approximately 4.6 million common shares at a weighted average price of $6.55 apiece to raise gross proceeds of approximately $30 million. Jefferies LLC managed the open market sale agreement. On Wednesday, Aurinia's shares (NASDAQ:AUPH) gained 10 cents to close at $7.10. Read More

Earnings

Horizon Pharma plc, of Dublin, reported its fourth-quarter net sales were $355.5 million, an increase of 30 percent, driven by continued strong growth of the company's orphan and rheumatology segment. For the year, Krystexxa (pegloticase) net sales were $258.9 million, representing a 65 percent year-over-year increase. Read More

Other news to note

Equillium Inc., of La Jolla, Calif., said it plans to develop EQ-001 for the treatment of lupus nephritis. Equillium remains on track to initiate the Equate phase Ib/II trial in acute graft-vs.-host disease in the first quarter of 2019. A proof-of-concept trial will start in severe asthma in the second quarter of 2019. Read More

Clinical data for Feb. 27, 2019

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Regulatory actions for Feb. 27, 2019

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