RNA therapy developer Proqr Therapeutics NV’s phase I/II study of adults with Usher syndrome and non-syndromic retinitis pigmentosa met all its key objectives, prompting the company to plan two parallel pivotal phase II/III studies that could start by year-end. The company wasted no time as it has already discussed next steps with the FDA to support the therapy’s registration “as soon as possible,” Aniz Girach, Proqr’s chief medical officer, told investors during a March 24 investor call.
LONDON – A U.K.-wide study has found that seven out of 10 patients hospitalized with COVID-19 infection were continuing to suffer physical and mental health problems five months after discharge.
LONDON – Astrazeneca plc promised to publish the primary analysis of the phase III U.S. trial of its COVID-19 vaccine by the morning of March 25, after chastisement from the data safety monitoring board (DSMB) for using “outdated” information in the interim results published on March 22.
Like its distinctive name, Dragon Boat Biopharmaceutical Co. Ltd., of Shanghai, raced to victory with the Chinese IND approval for its anti-CSF-1R monoclonal antibody candidate BC-006, marking an important milestone for the company as it embraces a growing focus on innovative medicines.
A phase IIa study of Frequency Therapeutics Inc.’s lead candidate in treating hearing loss showed it fared no better than placebo, but the company said its faith in the candidate is bolstered by the belief the study was hampered by trial design issues and by data from another study.
Shares of Ionis Pharmaceuticals Inc. (NASDAQ:IONS) fell 21.7% to $43.59 on March 23 after its longtime partner, Roche Holding AG, decided to stop dosing the antisense oligonucleotide tominersen in a global phase III manifest Huntington's disease (HD) study. Roche subsidiary Genentech Inc. said the move was based on an independent data monitoring committee's preplanned assessment of the drug's risk-benefit profile.
As the firm doubles down on a therapy for the rare and terrible lung disease called autoimmune pulmonary alveolar proteinosis (aPAP), Savara Inc. met with little trouble raising money, disclosing March 15 a public offering that grossed $130 million.
LONDON – Positive interim data from the U.S. trial of Astrazeneca plc’s COVID-19 vaccine have added yet more evidence it is effective in older adults and quelled concerns about serious thrombotic events that led countries in Europe to pause use of the vaccine earlier this month. Overall, the vaccine showed 79% efficacy in preventing symptomatic COVID-19, and was 100% effective in preventing serious disease and hospitalization. The effect was comparable across different ethnicities and age groups, with 80% efficacy in participants over 65 years of age. The analysis is based on 141 cases of COVID-19 infection.
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