About Steen Bjerre

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So far Steen Bjerre has created 145 blog entries.

Positive results from Ionis’ HAE program

2021-05-06T16:17:41+02:00May 6, 2021|HAEi News|

Ionis Pharmaceuticals, Inc. reports its financial results for the first quarter of 2021 and recent business highlights. CEO Brett P. Monia, Ph.D., says: "In the first quarter, we delivered positive results from our IONIS-PKK-LRx program, demonstrating its potential to change the standard of care for patients with HAE." (Source: Ionis)

Intellia plans on submitting first-in-human regulatory filings

2021-05-06T16:10:06+02:00May 6, 2021|HAEi News|

Reporting the Intellia Therapeutics, Inc. operational highlights and financial results for the first quarter ended 31 March 2021, President and CEO John Leonard, M.D., says: “Intellia continues to build momentum as we progress our full-spectrum pipeline towards key upcoming milestones this year. We plan on submitting first-in-human regulatory filings for NTLA-2002 for HAE.” NTLA-2002 aims to [...]

Differentiated strategy has potential to create greater and greater value

2021-05-06T13:16:05+02:00May 6, 2021|HAEi News|

Announcing the BioCryst Pharmaceuticals, Inc. financial results for the first quarter ended 31 March 2021, President and CEO Jon Stonehouse says: “Our commercial team is off to an outstanding start with the U.S. launch of Orladeyo. In this highly competitive market, we are demonstrating what we have known for some time now, HAE patients have been [...]

European Commission Approves First Oral, Once-daily Therapy

2021-05-01T10:03:50+02:00May 1, 2021|HAEi News|

The European Commission (EC) has approved oral, once-daily Orladeyo (berotralstat) for the prevention of recurrent HAE attacks in patients 12 years and older. “As the first targeted oral prophylactic therapy approved in Europe, Orladeyo represents a major advance in treatment for HAE patients who have been waiting for a preventive therapy. Physicians will be delighted [...]

Collaboration to deliver new plasma collection platform

2021-04-20T15:38:56+02:00April 20, 2021|HAEi News|

Terumo Blood and Cell Technologies and CSL Plasma collaborate to deliver a new plasma collection platform at CSL Plasma U.S. collection centers. A clinical trial of the investigational plasmapheresis device began earlier in April 2021, and introduction of the new platform is subject to U.S. Food and Drug Administration device clearance, at which time the parties [...]

Regulatory Update for Clinical Trial

2021-04-20T13:11:48+02:00April 20, 2021|HAEi News|

The U.S. Food and Drug Administration (FDA) has notified KalVista Pharmaceuticals, Inc. in a letter that it has placed a clinical hold on the proposed Phase 2 clinical trial of KVD824. KVD824 is KalVista’s oral product candidate being developed for prophylactic treatment of HAE. An Investigational New Drug Application was submitted earlier in 2021 for a [...]

Japanese National Health Insurance System approves Orladeyo addition to drug price list

2021-04-14T15:23:32+02:00April 14, 2021|HAEi News|

The Japanese National Health Insurance System (NHI) has approved the addition of oral, once-daily Orladeyo (berotralstat) from BioCryst Pharmaceuticals, Inc. to the NHI drug price list on 21 April 2021. Oral, once-daily Orladeyo was approved in Japan in January 2021 for prophylactic treatment of HAE in adults and pediatric patients 12 years and older. Orladeyo [...]

Positive topline study results of antisense treatment

2021-03-30T08:08:48+02:00March 30, 2021|HAEi News|

Data from a Phase 2 clinical study of IONIS-PKK-LRx from Ionis Pharmaceuticals, Inc. meets its primary and secondary endpoints, achieving significant reductions in the number of attacks suffered by patients with HAE compared to placebo. The study demonstrated a mean reduction of 90% in the number of monthly HAE attacks in weeks one to 17 [...]

Catabasis expects Investigational New Drug application in 1H 2022

2021-03-12T15:04:35+02:00March 12, 2021|HAEi News|

“With our acquisition of Quellis and concurrent financing, we believe Catabasis is well positioned to advance the development of our lead program, QLS-215, as a differentiated and potential best-in-class new therapy for the chronic treatment of patients affected by HAE to prevent attacks,” says Jill C. Milne, Ph.D., CEO of Catabasis Pharmaceuticals, Inc., at the presentation [...]

Takeda submits New Drug Application for lanadelumab in Japan

2021-03-12T14:56:16+02:00March 12, 2021|HAEi News|

Takeda Pharmaceutical Company Limited has submitted a New Drug Application to the Ministry of Health, Labour and Welfare in Japan for lanadelumab subcutaneous injection, a monoclonal antibody therapy for prophylaxis against attacks of HAE. In Japan, it is estimated that between 2,000 and 3,000 people are living with HAE, but only approximately 450 have been [...]