On May 3, 2023 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the first quarter ended March 31, 2023, and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAY 3, 2023, View Source [SID1234630904]).

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"The strong new patient growth in the first quarter, building on our large patient base with ORLADEYO, positions us very well to achieve our expectations for 2023, and advances us on a trajectory to peak sales of $1 billion. This growing revenue stream, alongside our robust balance sheet, has dramatically reduced our reliance on the capital markets as we drive value with continued commercial execution and disciplined investment in our pipeline," said Jon Stonehouse, president and chief executive officer of BioCryst.

Program Updates

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

ORLADEYO net revenue in the first quarter of 2023 was $68.4 million (+37.6 percent year-over-year (y-o-y)).

The number of U.S. patients on drug at the end of Q1 2023 increased by 46 percent compared to the end of Q1 2022 and increased by eight percent compared to the end of Q4 2022.

The launch has recently surpassed the milestone of 1,000 patients in the U.S. on ORLADEYO therapy.

New patient growth remained strong, with a 20 percent increase in U.S. patient start forms in the first quarter of 2023 compared to the first quarter of 2022. There were more new U.S. patient start forms in the first quarter of 2023 than in three of the four quarters in 2022.

The launch of ORLADEYO outside the U.S. continues to accelerate. Sales from outside the U.S. contributed 11.1 percent of global ORLADEYO net revenues in the first quarter.

As expected, the percentage of U.S. ORLADEYO patients receiving free drug in the first quarter increased, primarily because many patients went through the annual re-authorization process with payors.
"As we enter our third year on the market, 1,000 of 7,500 U.S. HAE patients are already benefitting from ORLADEYO. Demand is very strong in the U.S. and in international markets and our market data from patients and physicians tells us that ORLADEYO is still in the early stages of its growth trajectory," said Charlie Gayer, chief commercial officer of BioCryst.

Rare Disease Pipeline

The goal with our pipeline is to continue bringing selected, highly differentiated rare disease products to the market, and to reproduce the commercial success we have delivered with ORLADEYO. We are investing to expand the ORLADEYO label with our ongoing pediatric trial (APeX-P); and pursuing BCX10013, a potential best-in-class once-daily, oral Factor D inhibitor, an oral C2 inhibitor in lead optimization, and other discovery programs targeting multiple complement and non-complement pathways.

Debt Refinancing

On April 17, 2023, the company entered into a $450 million loan agreement with Pharmakon which provided for an initial term loan of $300 million. The remaining $150 million can be drawn at the company’s option until September 2024. The initial proceeds were used to repay the outstanding indebtedness with Athyrium and provided approximately $26 million for other general corporate purposes.

First Quarter 2023 Financial Results

For the three months ended March 31, 2023, total revenues were $68.8 million, compared to $49.9 million in the first quarter of 2022 (+37.8 percent y-o-y). The increase was primarily due to $68.4 million in ORLADEYO net revenue in the first quarter of 2023, compared to $49.7 million in ORLADEYO net revenue in the first quarter of 2022 (+37.6 percent y-o-y).

Research and development (R&D) expenses for the first quarter of 2023 decreased to $48.4 million from $65.4 million in the first quarter of 2022 (-26.0 percent y-o-y), primarily due to reduced R&D investment following the discontinuation of the BCX9930 and BCX9250 programs.

Selling, general and administrative (SG&A) expenses for the first quarter of 2023 increased to $47.9 million, compared to $34.3 million in the first quarter of 2022 (+39.6 percent y-o-y). The increase was primarily due to increased investment to expand and enhance the U.S. commercial team and expanded international operations.

Interest expense was $27.4 million in the first quarter of 2023, compared to $23.8 million in the first quarter of 2022 (+14.9 percent y-o-y). The increase was due to service on the company’s royalty and debt financing agreements. $7.1 million was paid in cash following the expiration of the payment-in-kind (PIK) interest period under the Athyrium agreement.

Net loss for the first quarter of 2023 was $53.3 million, or $0.28 per share, compared to a net loss of $74.2 million, or $0.40 per share, for the first quarter of 2022.

Cash, cash equivalents, restricted cash and investments totaled $403.1 million at March 31, 2023, compared to $446.8 million at March 31, 2022. Operating cash use for the first quarter of 2023 was $40.8 million. Following the completion of the debt refinancing with Pharmakon announced in April 2023, net proceeds of the agreement of $26 million brought pro forma cash to approximately $429 million.

Financial Outlook for 2023

The company expects full year 2023 global net ORLADEYO revenue to be no less than $320 million. Operating expenses for full year 2023, not including non-cash stock compensation, are expected to be flat to 2022 at approximately $375 million. While flat year-over-year, we expect reductions in R&D spending in 2023 following the discontinuation of the BCX9930 and BCX9250 programs in 2022 and the delay in the BCX10013 clinical program, offset by increases in SG&A to support the U.S. launch and global expansion of ORLADEYO.

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 1-866-777-2509 for domestic callers and 1-412-317-5413 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at www.biocryst.com.