—Company also expanding its discovery platform in complement-mediated diseases, including potent, selective, oral molecules targeting C2—
BioCryst has initiated plans to advance BCX10013 into patient studies in mid-2023, including in patients with paroxysmal nocturnal hemoglobinuria (PNH), to evaluate once-daily dosing.
The company expects to confirm the optimal dosing for pivotal trials by the end of the year, move directly into a pivotal trial in patients with immunoglobulin A nephropathy (IgAN), and rapidly expand into pivotal trials in additional indications.
“We remain committed to bringing better options to patients with complement-mediated diseases and we are excited to see the immediate and durable effect of BCX10013 in suppressing the alternative pathway. Our next step is to gather data from a small number of patients, utilizing the excellent biomarkers in PNH, to quickly confirm optimal clinical dosing this year. We then plan to rapidly advance the program into pivotal trials in multiple complement-mediated diseases, beginning next year with IgAN. We believe BCX10013 has the potential to be a best-in-class treatment option with an oral, once-daily profile,” said Dr.
In the SAD assessment to date, cohorts of healthy volunteers received a single dose of 1 mg, 3 mg, 10 mg, 40 mg, 80 mg or 110 mg of oral BCX10013 or placebo. In the MAD assessment to date, cohorts of healthy volunteers received 20 mg, 40 mg or 80 mg of oral BCX10013 or placebo administered once daily for seven days (20 mg cohort) or 14 days (40 mg and 80 mg cohorts).
Following single BCX10013 dose administration, the onset of AP inhibition occurred within one hour and increased in a dose-dependent manner. At 110 mg, the highest dose studied to date, AP activity was suppressed by a mean of 97.8 percent at 24 hours post-dose. Suppression of AP activity by BCX10013 was assessed using the AP Wieslab® assay, which measures functional activity of the complement system. In the MAD studies with once-daily dosing, exposure to BCX10013 was approximately dose proportional over the studied dose range and steady-state was achieved in 7 to 14 days with modest accumulation.
Additional data from the trial can be found in slides at the investors section of the company website at www.biocryst.com.
Expanding Programs in Complement-mediated Diseases
In addition to BCX10013, which targets Factor D in the alternative pathway of complement, BioCryst is pursuing oral medicines directed at other targets across the classical, lectin and terminal pathways of the complement system, including C2, a critical upstream serine protease enzyme for activation of the classical and lectin pathways. The company has developed potent, selective molecules targeting C2, which are currently in lead optimization.
“We believe that our ability to develop oral medicines as monotherapy against targets across multiple complement pathways, in addition to the alternative pathway, could allow us to help more patients with distinctly different complement-mediated rare diseases. With our oral approach, BioCryst also has the opportunity to develop combination therapies to improve therapeutic options for those diseases affecting multiple pathways of the complement system,” Thackray added.
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Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding our plans and expectations for our complement program and other future results, performance or achievements. 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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Investor Contact:
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jbluth@biocryst.com
Media Contact:
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ckyroulis@biocryst.com
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