Responding to safety concerns associated with roxadustat, Fibrogen Inc. officials told the FDA’s Cardiovascular and Renal Drugs Advisory Committee that it has proposed a change in the dosing regimen to reduce the risk of thrombosis. The company also has proposed enrolling 10,000 patients in a controlled postmarket study to confirm that the risk of cardiovascular events with the first-in-class drug is similar to that seen with erythropoiesis stimulating agents, the current standard of care in treating anemia due to chronic kidney disease in patients on dialysis. If approved, roxadustat also could be indicated for patients not on dialysis.

Biogen’s Aduhelm for AD goes through wringer again as ICER’s CTAF meets

The California Technology Assessment Forum, an independent evidence appraisal committee of the Institute for Clinical and Economic Review (ICER), convenes today for deliberation on Aduhelm (aducanumab), Biogen Inc.’s recently approved Alzheimer’s disease therapy. Voting questions involve clinical evidence and value vs. cost. The amyloid beta-targeting drug was cleared June 7 and priced at $56,000 per year. ICER has issued a statement declaring that the FDA “failed in its responsibility to protect patients and families from unproven treatments with known harms.”

Shape of RNA to come: Series B boosts gene therapy company

Shape Therapeutics Inc. has completed a $112 million series B financing to continue developing its RNA technology designed to correct genetic mutations while avoiding immunogenicity and DNA damage. The firm’s platforms include RNAfix, which enables direct in vivo targeting and modification of RNA by leveraging proteins such as ADARs, suppressor transfer RNAs, and engineered adeno-associated viruses. The round was co-led by Decheng Capital and Breton Capital. Willett Advisors, New Enterprise Associates and Mission Biocapital also participated. Late in 2020, the Seattle-based company raised $35.5 million in a series A round.

Innovent invests $50M in Ascentage with joint commercialization, development plans

Innovent Biologics Inc. and Ascentage Pharma Group Corp. Ltd. inked a three-component collaboration that comes with joint commercialization of an NDA-stage Bcr-Abl inhibitor and co-development of combination therapies, as well as Innovent taking a stake in Ascentage. The collaboration involves a deal worth up to $145 million and a $50 million investment. Ascentage CEO Dajun Yang called the partnership "the largest deal between two biopharma companies in China." He added that it not only includes the equity investment for a long-term partnership, but also a package deal to advance combination therapies using both companies' assets to deliver safer and more effective therapies to patients.

Cancer Research UK spin-offs bring in record investments in biotech bubble

Cancer Research UK (CRUK) took a significant hit when its U.K.-wide charity shop chain had to close last year because of the pandemic. But, according to latest figures, its commercial arm could help it to bounce back after seeing its income more than double in the recent biotech boom. The organization reported record levels of investment in its spinout companies, which more than doubled in 2020-2021 compared with the previous year, jumping from £400 million (US$555 million) to £822 million (US$1.14 billion). That took the total investment to date to £2.3 billion and the total number of spinouts created to more than 60, according to CRUK’s annual review of commercial partnerships activity,

Paq Therapeutics closes $30M series A round to harness autophagy for targeted degradation

Paq Therapeutics Inc. closed a $30 million series A round to take forward a novel approach to the targeted degradation and removal of a wide range of molecular substrates and defective organelles by developing drug molecules that can tap into autophagy, the cell’s general waste disposal and recycling system.

South Korea pledges $870M for bio data dam, makes progress on regulation

South Korea plans to create a bio data dam, a step toward creating the necessary industrial ecosystem in the country’s bid to become one the top seven players in the global medical device market by 2025. The aim of the project is to collect bio-health information from 1 million people, Minister of Economy and Finance Nam-Ki Hong said at a May 2021 government meeting on innovative growth. Hong also serves as an ex-officio deputy prime minister. The data dam could help power medical device and drug development projects that rely on big data and artificial intelligence.

Lepu’s NDA for anti-PD-1 antibody accepted ahead of Hong Kong IPO

The China NMPA’s Center for Drug Evaluation accepted Lepu Biopharma Co. Ltd.’s new drug application for anti-PD-1 antibody candidate HX-008 (pucotenlimab). That comes after the company filed for an IPO in Hong Kong. So far, HX-008 demonstrated efficacy and safety in phase I and II trials in multiple indications. The overall response rate (ORR) and disease control rate (DCR) in the phase II trial in melanoma reached 18.5% and 44.5%, respectively. The ORR and DCR in the phase II trial in MSI-H/dMMR solid tumors reached 44.8% and 66.7%, respectively. Lepu CEO Sui Ziye said HX-008 can win its market share, despite the fierce PD-1-targeted drugs market.

FDA clamps down on ad claims impacting biosimilars

The FDA took Amgen Inc. to task for promotions claiming Neulasta (pegfilgrastim) Onpro on-body injector is more effective than pegfilgrastim delivered via a prefilled syringe. Since all approved pegfilgrastim biosimilars use a prefilled syringe, promotions such as Amgen’s professional animated banner stating that patients receiving pegfilgrastim via a prefilled syringe have a higher incidence of febrile neutropenia than those receiving Neulasta Onpro could discourage the use of the biosimilars.

10x Genomics releases new high-throughput single-cell analysis platform

10x Genomics Inc. launched its latest platform for single-cell analysis, the Chromium X Series, to cost-effectively "enable massively large-scale experiments," according to the company. The system cuts the cost of single-cell analysis 83%, from 12 cents to 2 cents per cell, a significant step toward reducing the costs low enough to make genomic sequencing widely available.

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