On November 1, 2022 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported that financial results for the third quarter ended September 30, 2022, and provided a corporate update (Press release, BioCryst Pharmaceuticals, NOV 1, 2022, View Source [SID1234622672]).

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"With ORLADEYO on a trajectory to more than double sales in its second year of launch after a very strong first year, we continue to demonstrate that what we are offering HAE patients is unique and fills their need for a therapy that offers both low burden of disease and low burden of treatment. We believe that our ability to successfully pursue challenging targets like plasma kallikrein is just the start, and we expect we will bring rare disease patients many more oral therapies to offer these patients the unique treatment options they are waiting for," said Jon Stonehouse, president and chief executive officer of BioCryst.

Program Updates and Key Milestones

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

U.S. Launch

The total number of patients on paid therapy grew by nine percent in the third quarter.

New patient starts remained strong in the third quarter, in line with the running six quarter average.

The ORLADEYO prescriber base grew by 11 percent in the third quarter.

These strong underlying growth trends were masked by accelerated direct shipments requested by patients late in the second quarter in June prior to the July 4 holiday weekend, plus a negative out of period reimbursement-related charge that was accounted for in the third quarter.
ORLADEYO: Global Updates

In the third quarter, ORLADEYO was approved in Saudi Arabia. The company expects continued approvals and launches in additional countries.
"With the strong underlying growth fundamentals we continued to see from patients and physicians in the third quarter, and the further growth dynamics we expect to see in the fourth quarter, we are on track to more than double ORLADEYO revenue in 2022 compared to 2021," said Charlie Gayer, chief commercial officer of BioCryst.

Complement Program

BCX9930 – Oral, Twice-daily Factor D Inhibitor

On August 4, 2022, the company announced that the U.S. Food and Drug Administration (FDA) had lifted its partial clinical hold on the BCX9930 program and that the company will resume enrollment in global clinical trials under revised protocols at a reduced dose of 400 mg twice daily of BCX9930. This includes the REDEEM-1 and REDEEM-2 pivotal trials in patients with paroxysmal nocturnal hemoglobinuria (PNH) and the RENEW proof-of-concept trial in patients with C3 glomerulopathy (C3G), immunoglobulin A nephropathy (IgAN) and primary membranous nephropathy (PMN).

Screening has begun for new patients to participate in the trials and the company expects to have data from approximately 15 newly-enrolled patients by the middle of 2023 to inform its decision to either fully invest in the pivotal program, or to discontinue the BCX9930 program.

BCX10013 – Oral, Once-daily Factor D Inhibitor with Best-in-class Potential

The company has begun a clinical program with BCX10013, a novel, potent and specific Factor D inhibitor, and expects to report preliminary data from healthy volunteers in the first quarter of 2023. The preclinical and early clinical profile from approximately 90 healthy volunteers to date suggests BCX10013 could have the properties of a once-daily oral therapy. A goal of the ongoing clinical program is to confirm this once-daily profile with healthy volunteer and patient data.

Additional Complement Targets

In addition to BCX9930 and BCX10013, which target 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. The goal of the company’s overall complement program is to advance several oral compounds across multiple pathways in the complement system to treat many complement-mediated diseases.
BCX9250 – Oral ALK-2 Inhibitor for Fibrodysplasia ossificans progressiva (FOP)

The company believes that patients with FOP, an ultra-rare disease, are likely to benefit from other oral ALK-2 inhibitors that currently are substantially ahead of BCX9250 in development. Considering the expectation that patients will be well-served by these other products, and the approximately $100 million in additional investment that would be required to advance BCX9250 to approval, the company is stopping the BCX9250 program and redirecting this investment to the other opportunities it has to serve patients with complement-mediated diseases.
Third Quarter 2022 Financial Results

For the three months ended September 30, 2022, total revenues were $75.8 million, compared to $41.0 million in the third quarter of 2021 (+85 percent year-over-year (y-o-y)). The increase was primarily due to $66.0 million in ORLADEYO net revenue in the third quarter of 2022.

Research and development expenses for the third quarter of 2022 increased to $52.7 million from $50.0 million in the third quarter of 2021 (+6 percent y-o-y), primarily due to additional investment in the HAE program and expenses for BCX9250 prior to its discontinuation, partially offset by reduced costs related to the BCX9930 studies.

Selling, general and administrative expenses for the third quarter of 2022 increased to $36.9 million, compared to $35.0 million in the third quarter of 2021 (+6 percent y-o-y). The increase was primarily due to increased investment to support the commercial launch of ORLADEYO.

Interest expense was $24.8 million in the third quarter of 2022, compared to $14.1 million in the third quarter of 2021 (+76 percent y-o-y). The increase was due to service on the royalty financings, which were completed in November 2021. The interest payment-in-kind (PIK) option on the Athyrium term loan has been exercised and $6.5 million has been added in the third quarter of 2022 and $32.4 million since issuance, to the $200 million principal.

Net loss for the third quarter of 2022 was $42.5 million, or $0.23 per share, compared to a net loss of $58.8 million, or $0.33 per share, for the third quarter of 2021.

Cash, cash equivalents, restricted cash and investments totaled $462.6 million at September 30, 2022, compared to $203.9 million at September 30, 2021. Operating cash use for the third quarter of 2022 was $29.4 million.

Financial Outlook for 2022

Based on the strength of the ORLADEYO launch through the first three quarters of 2022, the company expects full year 2022 net ORLADEYO revenue to be $255 million.

Based on the reduced spending on the BCX9930 program in the first three quarters of the year, and lower than projected spending on the program for the remainder of the year, the company now expects operating expenses for full year 2022, not including non-cash stock compensation, to be between $365 million and $370 million.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 866-374-5140 for domestic callers and 404-400-0571 for international callers and using conference ID 28663801#. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A replay of the call will be available on the company website.