BIOCRYST PHARMACEUTICALS, INC. Clinical evidence and recent laboratory studies have informed the company’s hypothesis that crystals form in the kidneys of some patients. The company believes that lowering the dose to 400 mg and ensuring adequate hydration will dilute the concentration of drug in the urine below the threshold where crystals can form.
On
“Now that the FDA has lifted the partial clinical hold, we can take the next step toward our goal of finding a safe and effective dose for BCX9930. We expect this can be accomplished in a reasonable time frame after resuming enrollment, in a relatively small number of patients given the rate and timing of the SCr rises in patients prior to the enrollment pause. If we are successful, we will invest more significantly in BCX9930 to tap the full potential of reaching many patients suffering from a number of alternative pathway diseases, and, if we are not successful, we will stop investment in BCX9930 and move on to other molecules in our pipeline,” said
PNH patients naive to C5 inhibitor therapy who received 400 mg twice daily in the Phase 1 clinical program (n=7) experienced a mean change from baseline increase in hemoglobin of 4.3 g/dL and required no transfusions, and no patients experienced an elevation in their SCr during dose-ranging at the 400 mg twice daily dose. All observed cases of SCr elevations occurred at 500 mg twice daily.
About the BCRX9930 Clinical Trial Program
REDEEM-1 is a randomized, open-label, active comparator-controlled comparison of the efficacy and safety of BCX9930 monotherapy in approximately 81 PNH patients with an inadequate response to a C5 inhibitor. In part 1 of this trial, patients who have not had an adequate response to a C5 inhibitor will be randomized 2:1 to discontinue their C5 inhibitor and receive BCX9930 as monotherapy or to continue receiving their C5 inhibitor for 24 weeks. All patients will receive BCX9930 in part 2 (weeks 25-52) to assess the long-term safety, tolerability and effectiveness of BCX9930. Patients who are randomized to C5 inhibitor therapy in part 1 will discontinue that therapy at the week 24 visit and start BCX9930 for part 2. The primary endpoint of REDEEM-1 is change from baseline in hemoglobin, as assessed at weeks 12 to 24.
REDEEM-2 is a randomized, placebo-controlled trial evaluating the efficacy and safety of BCX9930 as monotherapy versus placebo in approximately 57 PNH patients not currently receiving complement inhibitor therapy. In part 1 of this trial, patients will be randomized 2:1 to receive BCX9930 or placebo under double-blind conditions for 12 weeks. All patients will receive BCX9930 in part 2 (weeks 13-52) to assess the long-term safety, tolerability and effectiveness of BCX9930, with patients randomized to placebo in part 1 switching to BCX9930 at the week 12 visit. The primary endpoint of REDEEM-2 is change from baseline in hemoglobin, as assessed at week 12.
RENEW is an open-label, multicenter, proof-of-concept study designed to evaluate the safety, tolerability and therapeutic potential of BCX9930 administered for 24 weeks in approximately 42 adult patients (14 patients in each disease) with either C3G, IgAN or PMN. All patients will be enrolled into one of three parallel study cohorts, based on confirmation of diagnosis and disease activity in a recent kidney biopsy, and will receive BCX9930 for the 24-week treatment period. The primary endpoint of RENEW is percent change from baseline in 24-hour urine protein-to-creatinine ratio (uPCR), as assessed at week 24.
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Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding BioCryst’s plans and expectations for its BCX9930 program. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst’s business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst’s and its partners’ development, regulatory processes and supply chains, negatively impact BioCryst’s ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst files periodically with the
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