The FDA’s approval of Agios Therapeutics Inc.’s Pyrukynd (mitapivat) for treating hemolytic anemia marks a turnaround from nearly a year ago. That’s when Agios sold its commercial, clinical and research-stage oncology portfolio to privately held Servier Pharmaceuticals LLC to concentrate on rare diseases. Agios received $1.8 billion cash up front and as much as $200 million in regulatory milestone payments for the investigational glioma treatment vorasidenib. Agios used the deal to accelerate and expand its genetically defined disease portfolio, including its work on Pyrukynd as a potential treatment for adults with pyruvate kinase deficiency, thalassemia and sickle cell disease. It paid off. The approval was announced on Feb. 17, the therapy’s PDUFA date. Pyrukynd is expected to launch in the U.S. within the next two weeks.

GSK pauses late-stage trial of potential blockbuster RSV vaccine

Glaxosmithkline plc has paused a late-stage trial of its potential respiratory syncytial virus (RSV) in pregnant women following a safety warning. In a brief statement, the London-based firm said it had voluntarily paused recruitment and vaccination in the phase III GRACE trial of its RSV maternal vaccine candidate, as well as two other trials investigating the drug in pregnant women. GSK said the decision was made following a recommendation from its independent data monitoring committee based on an undisclosed observation from a routine safety assessment.

HPT front and center as Wall Street awaits Ascendis phase III data 

Phase III data are due any day from Ascendis Pharma A/S, and Wall Street’s thoughts have turned to hypoparathyroidism, an indication fraught with questions in recent years due to the hurdles met by Takeda Pharmaceutical Co. Ltd.’s Natpara. Copenhagen-based Ascendis will roll out 26-week data from the study called Pathway with Transcon PTH, a prodrug of parathyroid hormone in development as a once-daily replacement therapy. A double-blind, placebo-controlled experiment in adults, Pathway is expected to enroll about 76 participants at sites in North America and Europe. Transcon PTH is stable at room temperature and given in a ready-to-use liquid formulation with a pen-injector.

EBV antibodies put to good use through retargeting

Researchers at Inserm have developed a method to direct pre-existing antibodies toward new targets. Their bimodular fusion proteins (BMFPs) could be a broadly useful method for expanding access to antibody therapy. In a study that appeared in the Feb. 11, 2022, issue of Science Advances, the teams showed that antibodies to Epstein-Barr virus (EBV), which are present in 95% of the global population, could be redirected to a target cell of their choosing by fusing an EBV antigen to a cellular targeting ligand. 

Newco news: Varsity jumps on POLQ train, licensing in novobiocin from Dana Farber

After a landmark clinical trial of the first POLQ inhibitor in cancer last year, a recently formed U.K. biotech is gearing up to bring a potential rival to the clinic in the coming months. Varsity Pharmaceuticals Ltd., of Cambridge, is planning to begin a phase I trial of novobiocin, a drug previously used as an antibiotic, which has also been found to inhibit the polymerase theta inhibitor (POLQ) pathway. This means the compound could be used to treat homologous recombination (HR)-deficient cancers, where the body is unable to repair double strand breaks in DNA due to mutations in genes such as BRCA1 and BRCA2.

EMA: Increase in NCEs approved in 2021 amid overall drop for the year

LONDON – Two years into the pandemic and the number of new drugs approved by the EMA fell from 97 approvals in 2020 to 92 in 2021. But both years are still well up on pre-COVID-19 times in 2019 when 66 products got the nod, according to the EMA’s annual human medicines report. Rather than referencing the drop in the overall approvals, the report published on Feb. 15 highlights a 35% increase in the number of new chemical entities (NCEs, or new active substances as the EMA calls them), with 53 NCEs approved in 2021 compared to 39 in 2020. The report also underlines the additional weight and stress that the pandemic has placed on the agency’s shoulders.

Livzon’s COVID-19 vaccine as a booster shot shows efficacy against omicron variant

Livzon Pharmaceutical Group Inc.’s key interim data from the phase III trials of its recombinant SARS-CoV-2 fusion protein vaccine, V-01, have shown efficacy against the omicron variant. Data showed that the person-year incidence rates were 6.73% for people who had a booster of V-01 and two doses of inactivated vaccine vs. 12.8% for those who received placebo after two doses of inactivated vaccine, with significant difference (p=0.0012). The absolute vaccine efficacy of V-01 as a booster was 61.35%. Meanwhile, 60 specimens of SARS-CoV-2 virus that were genotyped with the first generation of sequencing were infected with omicron, while the rest are going through second-generation sequencing. Data showed that a booster of V-01 is effective against the omicron variant, with no safety issues of concern.

European Besremi legal tussle continues

In the latest chapter in an ongoing contract dispute between AOP Orphan Pharmaceuticals GmbH and Pharmaessentia Corp., the German Federal Court of Justice this week set aside the €143 million (US$162.8 million) in damages awarded to AOP, citing procedural flaws in quantifying the product supply and damages. 

Holiday notice

BioWorld's offices will be closed in observance of Presidents Day in the U.S. No issue will be published Monday, Feb. 21.

BioWorld Insider Podcast: Could long COVID be the next public health crisis?

With the study of long COVID-19 continuing to evolve, the BioWorld team is taking a closer look at the condition – a complex syndrome affecting more than 100 million people globally – and what’s being done about it. In this episode, BioWorld Senior Science Editor Anette Breindl and Staff Writer Lee Landenberger, fresh off writing a pair of detailed stories about these issues, join us to talk about how consensus around what long COVID is has emerged.

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