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BioWorld - Saturday, December 13, 2025
Home » Topics » Clinical

Clinical
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Ionis shares sink as Roche quits dosing phase III HD drug following IDMC opinion

March 23, 2021
By Michael Fitzhugh
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.
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In the clinic for March 23, 2021

March 23, 2021
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Akero, Anokion, Frequency, Ideaya, Incyte, Ionis, Lineage, Neoimmunetech, Novartis, Omeros, Oramed, Precigen, Recordati, Regeneron, Roche, Sojournix.
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Diagram of Hemocath intelligent HF monitoring platform

Major test awaits less invasive device for detecting congestive heart failure

March 22, 2021
By David Godkin
TORONTO – Hemocath Ltd. is gearing up for a first-in-human trial of a device that addresses two critical metrics for the treatment of heart failure: pulmonary artery and central venous pressure. Hemocath’s pulmonary artery catheter (PAC) is part of a larger heart failure monitoring platform and is intended to assess fluid status in patients with severe or worsening congestive heart failure. The hope is to replace larger, more invasive catheters inserted into a patient’s neck.
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Savara goes back for seconds; inhaled fix for aPAP at last?

March 22, 2021
By Randy Osborne
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.
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Vaccine administration

Astrazeneca reports vaccine 79% effective in U.S. trial, prepares for FDA submission

March 22, 2021
By Nuala Moran
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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In the clinic for March 22, 2021

March 22, 2021
Clinical updates, including trial initiations, enrollment status and data readouts and publications: AIM Immunotech, Astrazeneca, Azurrx, Bioage, Biocryst, Biophytis, CTI, Curevac, Dare, Eucure, Faron, Genentech, Harbour, Leap, Neurorx, Rapt, Rocket, Terns.
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3D rendering of LIGHT system

Study shows potential of Advanced Oncotherapy’s LIGHT proton therapy for resistant cancers

March 19, 2021
By Meg Bryant
Advanced Oncotherapy plc reported new findings demonstrating the potential ability and superiority of its next-generation proton therapy system, LIGHT, to treat resistant cancers. The findings build on research at the Mayo Clinic that highlighted the benefits of combining proton therapy (PT) with targeted small molecule damage repair inhibitors.
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TLR illustration

At the crossroads: Idera ponders TLR9 phase III continuation

March 19, 2021
By Lee Landenberger
Following a missed primary endpoint in its phase III study in treating anti-PD-1-refractory advanced melanoma, Idera Pharmaceuticals Inc. is considering stopping the registrational trial of its synthetic Toll-like receptor 9 (TLR9) agonist or pressing on toward the clinical trial’s second primary endpoint.
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In the clinic for March 19, 2021

March 19, 2021
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Amyndas, Bluesky, Clovis, Eisai, Idera, Incyte, Merck & Co., Sarepta.
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Translate Bio shares fall on CF candidate's efficacy issues

March 18, 2021
By Michael Fitzhugh
Shares of mRNA therapeutics specialist Translate Bio Inc. (NASDAQ:TBIO) fell 30.9% to $17.76 on March 18 over concerns about a new interim analysis of phase I/II data for its cystic fibrosis (CF) candidate, MRT-5005. Several dosing regimens of the inhaled candidate failed to improve a key measure of lung function vs. placebo, in contrast to an earlier readout on other doses tested in the ongoing study, called Restore-CF.
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