Genetic eye disease specialist Proqr Therapeutics NV is entering new therapeutic areas with Eli Lilly and Co., specifically genetic disorders in the liver and nervous system. Proqr is collecting $50 million in the form of an up-front payment ($20 million) and an equity investment ($30 million), with the prospect of about $1.25 billion more if the arrangement hits research, development and commercialization milestones. The pair will use Proqr’s Axiomer RNA editing platform to develop editing oligonucleotides directed to as many as five targets. Shares of Proqr (NASDAQ:PRQR) were trading midday at $8.54, up $1.73, or 25%.

Humanigen's lenzilumab meets FDA refusal on emergency use for COVID-19

Initial efforts at Humanigen Inc. to win an FDA emergency use authorization (EUA) for treating hospitalized COVID-19 patients with its monoclonal antibody, lenzilumab, have failed to gain the regulator's buy-in, sending company shares (NASDAQ:HGEN) down 49% by midday. The FDA was "unable to conclude that the known and potential benefits of lenzilumab outweigh the known and potential risks" for treating COVID-19, Humanigen said. Regulatory filings in other markets, such as the U.K., remain underway. Meanwhile, a secondary path to an EUA in the U.S. may emerge from the ongoing NIAID-sponsored ACTIV-5/Big Effect Trial, Humanigen CEO Cameron Durrant said.

COVID-19 efforts carry infectious disease stocks amid UTI and HBV defeats

If there is one thing that potently illustrates the hope and despair of the COVID-19 pandemic, it is the zigzag of BioWorld’s Infectious Disease Index (BIDI). Up by nearly 64%, it is towering over both the Nasdaq Biotechnology Index and the Dow Jones Industrial Average, which are up by 12.4% and 15.5%, respectively, but it has experienced several downward swings this year as well. BIDI’s low points occurred at the end of March, May and July, just as the Delta variant emerged in India and began spreading throughout the world, reducing the effectiveness of available COVID-19 vaccines. Delays in Novavax Inc. filings to gain authorization for a fourth vaccine in the U.S. also weighed heavily during those drops. Sprinkled throughout the period, however, are several pandemic wins, such as robust phase III data of the Novavax protein-based vaccine NVX-CoV2373, as well as global emergency use authorizations and rolling submissions for vaccines that use Dynavax Technologies Corp.’s Toll-like receptor 9 agonist adjuvant, CPG 1018.

Astrazeneca eyes potential ‘rescue’ asthma inhaler after double phase III success

Astrazeneca plc is eyeing an addition to its respiratory diseases portfolio after a potential first-in-class “rescue” asthma inhaler, PT-027, met its goals in two phase III trials. The big U.K. pharma has a portfolio of respiratory drugs that are focused on longer-acting maintenance therapies. But, according to its development partner Avillion LLP, the combination of salbutamol and budesonide, respectively a short-acting beta2 agonist and an inhaled corticosteroid, could help treat the inflammation that is the underlying cause of the disease.

Selfmade antibodies could address vexing health questions

Monoclonal antibodies are a triumph of modern medicine. They are also too expensive to be a standard therapy in all but the wealthiest countries. And even in wealthy countries, their cost is unsustainable, and becoming more so as more antibodies are approved. According to a report by the health care analytics firm Iqvia, in 2017 biologics made up 2% of U.S. prescriptions, but accounted for nearly 40% of net drug spending. “Having 10% or 15% of your population on antibodies is not sustainable even in wealthy countries,” Rachel Liberatore told BioWorld. Liberatore is director of research and development at Renbio Inc., which is testing the intramuscular delivery of antibody-encoding DNA to prevent and treat infections including SARS-CoV-2 and HIV. Renbio as well as other companies and academic collaborators are working to expand the power of monoclonal antibodies by treating patients not with antibodies delivered not as proteins, but as DNA.

China’s new personal information protection law challenges company access to user data

China’s Personal Information Protection Law (PIPL) will take effect on Nov. 1, 2021, making it harder for tech firms, including health care companies, to access and use consumers’ personal information. The PIPL was officially passed by the National People’s Congress on Aug. 20, 2021. “With PIPL, China is moving from a relatively narrow and devolved regulation of personal information towards something that on the face of it could be one of the tightest regimes in the world,” Stephen Sunderland, a partner at strategy consultancy L.E.K. Consulting LLC, told BioWorld.

Novel theories of prosecution emerging among U.S. enforcement agencies

The COVID-19 pandemic has exerted a significant effect on enforcement in the U.S., but Ethan Davis, a partner at the law firm of King & Spalding, said on a Sept. 9 webinar that federal prosecutors have made use of several novel approaches to prosecution in the past year. One of the more novel prosecutorial moves in recent memory was to prosecute a drugmaker using the Racketeer Influenced and Corrupt Organizations Act, a portion of the statute typically reserved for prosecutions of organized crime and a signal that federal prosecutors are growing more aggressive in their pursuit of companies in the life sciences.

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