BioCryst Reports Initial Clinical Data with Oral Factor D Inhibitor BCX10013 Supporting Development as a Once-daily Treatment for Complement-mediated Diseases

- Company also expanding its discovery platform in complement-mediated diseases, including potent, selective, oral molecules targeting C2—

RESEARCH TRIANGLE PARK, NC, USA I January 09, 2023 IBioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that initial data from ongoing phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) trials in healthy volunteers show rapid, sustained and >97 percent suppression of the alternative pathway (AP) of the complement system 24 hours following a single 110 mg dose. BCX10013 has been safe and generally well-tolerated at all doses studied to date. These data support the development of BCX10013 as a potential best-in-class, once-daily, oral Factor D inhibitor for multiple complement-mediated diseases.

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. Helen Thackray, chief research and development officer at BioCryst.

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.

About BioCryst Pharmaceuticals

BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States and many global markets. BioCryst has active programs to develop oral medicines for multiple targets across the complement system, including BCX10013, an oral Factor D inhibitor in clinical development. RAPIVAB® (peramivir injection) is approved in the U.S. and multiple global markets, with post-marketing commitments ongoing. For more information, please visit the company’s website at www.biocryst.com.

SOURCE: BioCryst Pharmaceuticals

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