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BioCryst (BCRX) Begins Enrollment in PNH Study on BCX9930

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BioCryst Pharmaceuticals, Inc. (BCRX - Free Report) announced that it has enrolled the first patient in the pivotal REDEEM-2 study, evaluating its oral Factor D inhibitor, BCX9930, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (“PNH”), a rare blood disease.

The placebo-controlled REDEEM-2 study will evaluate the safety and efficacy of BCX9930 (500 mg) as a monotherapy versus placebo in around 57 PNH patients who are not receiving complement inhibitor therapy currently. The primary endpoint of the study is to see the change from baseline in hemoglobin, as assessed at week 12 of treatment.

Per the press release, BioCryst is planning to enroll patients in the pivotal REDEEM-1 study, a comparator-controlled comparison of the safety and efficacy of BCX9930 (500 mg) monotherapy in approximately 81 PNH patients who have an inadequate response to a C5 inhibitor. The company is also initiating a proof-of-concept study to investigate BCX9930 in renal complement-mediated diseases.

Please note that the FDA has already granted Fast Track status as well as Orphan Drug designation to BCX9930 for treating PNH.

Shares of BioCryst have rallied 67.1% so far this year against the industry’s decrease of 19%.

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We remind investors that BioCryst’s Orladeyo (berotralstat) is currently marketed for prophylaxis to prevent attacks of hereditary angioedema in adults and pediatric patients 12 years and older. The drug has seen solid uptake since its launch and is approved in the United States, the European Union, Japan, the United Arab Emirates and the United Kingdom.

The company also has a deep pipeline with several ongoing development programs. Apart from BCX9930, BioCryst’s pipeline includes galidesivir, a potential treatment for Marburg virus disease and Yellow fever; and BCX9250, an ALK-2 inhibitor which is being developed for the treatment of fibrodysplasia ossificans progressiva.

Zacks Rank & Stocks to Consider

BioCryst currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biotech sector include Sarepta Therapeutics, Inc. (SRPT - Free Report) , vTv Therapeutics Inc. (VTVT - Free Report) and Editas Medicine, Inc. (EDIT - Free Report) , all carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Sarepta Therapeutics’ loss per share estimates have narrowed 31.3% for 2021 and 26% for 2022, over the past 60 days.

Earnings of Sarepta Therapeutics have surpassed estimates in two of the trailing four quarters, and missed the same on the other two occasions.

vTv Therapeutics’ loss per share estimates have narrowed 21.7% for 2021 and 2.9% for 2022, over the past 60 days.

vTv Therapeutics’ earnings have surpassed estimates in three of the trailing four quarters and missed the same on the other occasion.

Editas Medicine’s loss per share estimates have narrowed 11.2% for 2021 and 4.6% for 2022, over the past 60 days.

Editas Medicine’s earnings have surpassed estimates in two of the trailing four quarters and missed the same on the other two occasions.