On May 6, 2024 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the first quarter ended March 31, 2024, and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAY 6, 2024, View Source [SID1234642667]).

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"We are off to a fantastic start to the year with outstanding ORLADEYO revenue growth and our prioritized pipeline programs advancing on schedule. We are focused on continuing this momentum as we see strong patient demand for ORLADEYO and more pipeline programs advancing into the clinic, starting later this year," said Jon Stonehouse, president and chief executive officer of BioCryst.

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

ORLADEYO net revenue in the first quarter of 2024 was $88.9 million (+30 percent year-over-year (y-o-y)).

In the first quarter, the U.S. commercial team accelerated patients going through annual reimbursement reauthorization from free drug to paid drug faster than in previous years, generating higher than expected ORLADEYO revenue.

New patient growth remained strong, with the past two quarters having the most new prescriptions in the United States since the first two quarters of the launch.

Sales from outside the U.S. contributed 10 percent of global ORLADEYO net revenues in the first quarter, as the number of patients treated with ORLADEYO continued to grow strongly and consistently in new and existing markets.

New real-world evidence showing significant reductions in healthcare resource utilization among patients with HAE following ORLADEYO initiation to be presented in a poster on May 8 at the 2024 International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR).
"Our team made exceptional efficiency improvements in the U.S. prescription reauthorization process, and new prescriptions remained very strong as physicians and patients increasingly understand that ORLADEYO provides excellent efficacy and convenience. These two successes helped us exceed our revenue projections for the quarter and, as a result, we are increasing our revenue guidance for 2024," 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. Milestones in the next 18 months include:

The ongoing proof-of-concept trial with BCX10013, an oral Factor D inhibitor, remains on track. The company expects to partner or discontinue the program later this year.

Enrollment is complete in the APeX-P pediatric trial. Data from the trial will support a regulatory filing in 2025 to expand the ORLADEYO label to enable children as young as two years of age to receive ORLADEYO. ORLADEYO would be the first oral prophylactic therapy for children with HAE.

The company expects to advance BCX17725, its KLK-5 inhibitor for the treatment of Netherton syndrome, into the clinic by the end of 2024.

Netherton syndrome is a rare, lifelong genetic disorder that often presents in neonates or infancy with red, scaly and inflamed skin and susceptibility to recurrent skin infections. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there is no approved treatment 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 the most common cause of vision loss in individuals with diabetes and at least one-third of patients have persistent DME despite anti-VEGF therapies, which are administered via monthly injection. By delivering avoralstat with Clearside’s SCS microinjector directly into the suprachoroidal space of the eye in the clinical trial, avoralstat could inhibit plasma kallikrein at the sites of edema formation in DME disease, the retinal and choroidal vascular endothelium. With its low solubility, the drug could persist in the eye at the site of disease for a long duration, resulting in less frequent injections.
"It is very exciting to be advancing our pipeline of first-in-class/best-in-class molecules into the clinic to generate proof-of-concept data across multiple programs and diseases, and to be so close to delivering the first oral prophylactic option to children with HAE," said Dr. Helen Thackray, chief research and development officer of BioCryst.

First Quarter 2024 Financial Results

For the three months ended March 31, 2024, total revenues were $92.8 million, compared to $68.8 million in the first quarter of 2023 (+34.9 percent y-o-y). The increase was primarily due to $88.9 million in ORLADEYO net revenue in the first quarter of 2024, compared to $68.4 million in ORLADEYO net revenue in the first quarter of 2023 (+30 percent y-o-y).

Research and development (R&D) expenses for the first quarter of 2024 decreased to $46.5 million from $48.4 million in the first quarter of 2023 (-3.9 percent y-o-y), primarily due to decreased spending on BCX10013 due to our plan to either out-license or discontinue late-stage development and commercialization, which was announced in January 2024, as well as the discontinuation of the BCX9930 program, which was announced in December 2022. These reductions were partially offset by increased investment in BCX17725 and other discovery programs, and ORLADEYO label expansion and life cycle investments, such as our ongoing ORLADEYO pediatric trial.

Selling, general and administrative (SG&A) expenses for the first quarter of 2024 increased to $59.4 million, compared to $47.9 million in the first quarter of 2023 (+24.0 percent y-o-y). The increase was primarily due to increased investment to expand and enhance the U.S. commercial team and expand and support international operations.

Operating loss for the first quarter was $14.5 million, adjusted to $0.8 million excluding non-cash stock compensation.

Interest expense was $24.5 million in the first quarter of 2024, compared to $27.4 million in the first quarter of 2023 (-10.6 percent y-o-y). The decrease was primarily due to a decrease in the amortization of interest associated with our royalty financing obligations, partially offset by an increase in interest expense associated with the Pharmakon debt refinancing secured in April 2023.

Net loss for the first quarter of 2024 was $35.4 million, or $0.17 per share, compared to a net loss of $53.3 million, or $0.28 per share, for the first quarter of 2023. Non-GAAP net loss for the first quarter of 2024 was $34.1 million, or $0.17 per share when excluding one-time costs associated with the R&D restructuring, totaling $1.3 million. A reconciliation between GAAP and non-GAAP net loss is provided in the table below.

Cash, cash equivalents, restricted cash and investments totaled $338.4 million at March 31, 2024, compared to $403.1 million at March 31, 2023. Net cash utilization for the first quarter of 2024 was $52.4 million, which was driven by debt service, royalty payments and one-time first quarter compensation expense. We expect, as in previous years, that the first quarter will be the highest quarter of operating cash use for the year, and that operating cash use for the remaining quarters of 2024 will normalize around $10-$12 million per quarter. We expect that total cash at the end of 2024 will be above $300 million.

Non-GAAP Pro forma Financial Measures

The information furnished in this release includes non-GAAP pro forma financial measures that differ from measures calculated in accordance with generally accepted accounting principles in the United States of America ("GAAP"), including financial measures labeled as "non-GAAP" or "adjusted."

We believe providing these non-GAAP measures, which show our pro forma results with these items adjusted, is valuable and useful since they allow the company and investors to better understand the company’s financial performance in the absence of these one-time events and allowed investors to more accurately understand our current period results and more easily compare them to future results. These non-GAAP pro forma measures also correspond with the way we expect investors and financial analysts to compare our results. Our non-GAAP pro forma measures should be considered only as supplements to, and not as substitutes for or in isolation from, our other measures of financial information prepared in accordance with GAAP, such as GAAP revenue, operating income, net income, and earnings per share.

Our references to our first quarter 2024 "non-GAAP pro forma" financial measures of adjusted net loss and adjusted earnings per share constitute non-GAAP financial measures. They refer to our GAAP results, adjusted to show the results without the one-time costs associated with the R&D restructuring. A reconciliation between GAAP and non-GAAP net loss is provided in the table below.

Financial Outlook for 2024

Based on the team’s success in accelerating ORLADEYO patients through the U.S. reauthorization process faster than anticipated, the company is adjusting its outlook for full year 2024 global net ORLADEYO revenue to be between $390 million and $400 million, the top end of its prior guidance range.

The company expects full year 2024 operating expenses to be between $365 million and $375 million, flat to full year 2023 operating expenses.

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 adjusted revenue guidance for ORLADEYO, the company is even more 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 and does not intend to draw the additional $150 million of debt available to it from Pharmakon.

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 1-844-481-2942 for domestic callers and 1-412-317-1866 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.