On Nov. 04, 2024 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported financial results for the third quarter ended September 30, 2024, and provided a corporate update (Press release, BioCryst Pharmaceuticals, NOV 4, 2024, View Source [SID1234648574]).

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"Our third quarter performance continues to build on the outstanding year we are having, with significant revenue growth, strong patient demand, pipeline advancement and operating profitability in the quarter. As we look ahead, we see robust and durable revenue growth, new opportunities for younger children to benefit from ORLADEYO, and data readouts from BCX17725 in Netherton syndrome and avoralstat in DME, all while moving closer to sustainable profitability," 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

"The real-world efficacy and convenience patients and physicians are experiencing with ORLADEYO are driving accelerated commercial momentum that translates to nearly 36 percent revenue growth in our fourth year on the market. Based on this established sales trajectory, and the continued, durable strength of patient demand we see for ORLADEYO, we are more confident than ever of achieving peak sales of $1 billion for ORLADEYO," said Charlie Gayer, chief commercial officer of BioCryst.

ORLADEYO net revenue in the third quarter of 2024 was $116.3 million (+35.7 percent year-over-year (y-o-y)).

Start forms in the U.S. are up 14.2 percent over the past 12 months compared to the prior 12 months; and 67 new U.S. prescribers were added during the third quarter, one of the highest totals over the most recent eight quarters.

The reimbursed product rate in the U.S. increased 0.4 percent in the third quarter to 74.8 percent.

Sales from outside the U.S. contributed 11.4 percent of global ORLADEYO net revenues in the third quarter.

Since launch, approximately half of patients who have started ORLADEYO have switched from another prophylactic therapy. The company has begun the observational Phase 4 APeX-T study, designed to generate real-world data to inform physicians on the best individual approaches to support transition to ORLADEYO.
Rare Disease Pipeline

"The goal with our pipeline is to build on our success with ORLADEYO by bringing the next highly differentiated product to patients living with rare disease. We are making excellent progress, with BCX17725 now in the clinic, avoralstat nearing the clinic and initial patient data from both programs expected next year. These programs would address significant unmet needs for patients with Netherton syndrome and DME, respectively," said Dr. Helen Thackray, chief research and development officer at BioCryst.

The company has advanced BCX17725, its KLK5 inhibitor for the treatment of Netherton syndrome, into clinical trials and expects initial data from the program in 2025.

Netherton syndrome is a serious, rare, lifelong genetic disorder affecting the skin, hair, and immune system, caused by lack of normal function of a natural inhibitor of KLK5. People with Netherton syndrome often have red, scaly, inflamed skin, fragile hair, and are more likely to develop skin infections, allergies, asthma and eczema. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there are no approved treatments for Netherton syndrome.

In 2025, the company plans to advance avoralstat, a plasma kallikrein inhibitor, into a clinical trial of patients with diabetic macular edema (DME).

DME is an important cause of vision loss in diabetes and is due to leakage from the blood vessels in the retina. While current treatments focus on VEGF inhibition, DME can develop from other mechanisms, such as the kallikrein-bradykinin pathway. This is supported by observations that many DME patients have an incomplete response to intravitreal anti-VEGF therapies that are administered every four to eight weeks. Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space as a depot formulation, is designed to provide high dose levels to the retinal vessels with long-lasting exposure, which could result in less frequent injections and a reduced burden on patients and the healthcare system.
Third Quarter 2024 Financial Results

"It is exciting to see continued ORLADEYO growth alongside the significant progress advancing our pipeline, and another quarter of operating profit. BioCryst is in the strongest financial position in its history," said Anthony Doyle, chief financial officer at BioCryst.

For the three months ended September 30, 2024, total revenues were $117.1 million, compared to $86.7 million in the third quarter of 2023 (+35.1 percent y-o-y). The increase was primarily due to $116.3 million in ORLADEYO net revenue in the third quarter of 2024, compared to $85.7 million in ORLADEYO net revenue in the third quarter of 2023 (+35.7 percent y-o-y).

Research and development expenses for the third quarter of 2024 decreased to $41.1 million from $46.9 million in the third quarter of 2023 (-12.4 percent y-o-y), primarily due to decreased investment following the discontinuation of the BCX10013 program, partially offset by investments to advance our early clinical and discovery programs and an increased investment in the APeX-P program in preparation for regulatory filings next year.

Selling, general and administrative expenses for the third quarter of 2024 increased to $65.1 million, compared to $50.6 million in the third quarter of 2023 (+28.7 percent y-o-y). The increase was primarily due to increased commercial investment to support our growing revenue, newly launched regions and expanded international operations. There was also an increase in general and administrative expenses to support commercial growth.

Interest expense was $24.8 million in the third quarter of 2024, compared to $27.3 million in the third quarter of 2023 (-9.2 percent y-o-y). The decrease was primarily due to a decrease in the amortization of interest associated with our royalty financing obligations. Due to the strong cash position of the company, we declined to elect the payment-in-kind (PIK) option related to the Pharmakon term loan for the third quarter of 2024, resulting in a cash interest payment of $10.2 million in the third quarter of 2024 compared to $4.9 million in the third quarter of 2023. The PIK option has now expired. Additionally, the company chose not to execute its option, which expired September 30, 2024, to draw the additional $150 million of debt available to it from Pharmakon.

GAAP operating profit for the third quarter of 2024 was $7.7 million, compared to a GAAP operating loss of $11.9 million for the third quarter of 2023. Non-GAAP operating profit, excluding stock-based compensation expense, was $24.9 million for the third quarter of 2024 compared to $0.4 million for the third quarter of 2023.

Net loss for the third quarter of 2024 was $14.0 million, or $0.07 per share, compared to a net loss of $36.1 million, or $0.19 per share, for the third quarter of 2023.

Cash, cash equivalents, restricted cash and investments totaled $351.7 million at September 30, 2024, compared to $399.2 million at September 30, 2023. Operating cash increased by $13.1 million in the third quarter of 2024.

Financial Outlook for 2024

Based on the strength of patient demand for ORLADEYO seen in the third quarter, and expected continued strength in the fourth quarter, the company is adjusting its outlook for full year 2024 global net ORLADEYO revenue to be between $430 million and $435 million (top end of prior range) and introducing full-year 2024 total revenue guidance of between $443 and $448 million based on additional 2024 RAPIVAB revenue.

Directly related to the revenue strength for both ORLADEYO and RAPIVAB, the company is revising its guidance for operating expenses, and now expects full year 2024 operating expenses to be between $380 million and $390 million. The increase is driven primarily by increased COGS related to new RAPIVAB sales, increased variable costs, including incentive compensation and distribution costs related to the continued strong revenue performance for ORLADEYO and increased expenses that are seasonally booked in Q4 related to our support for the HAE community, including charitable donations. This operating expense outlook does not reflect non-cash stock compensation expense, or one-time expenses related to the previously announced workforce reduction implemented in the first quarter of 2024.

Based on the company’s disciplined approach to capital allocation, and the strong performance of ORLADEYO, the company is confident that it will achieve a full-year operating profit in 2024 (not including non-cash stock compensation), be approaching quarterly positive earnings per share (EPS) and positive cash flow in the second half of 2025 (not including non-cash stock compensation) and be profitable on an EPS basis, with positive cash flow, for full year 2026. The company expects it can achieve these financial milestones without raising additional funds.