—Q1 2024 ORLADEYO net revenue grows 30 percent y-o-y to
—Full-year 2024 ORLADEYO revenue guidance adjusted to
—Pipeline programs, including BCX17725 for Netherton syndrome and avoralstat for DME, advancing on schedule into clinical trials—
“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
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 2024International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR).
“Our team made exceptional efficiency improvements in the
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.
- 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.
- 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.
First Quarter 2024 Financial Results
For the three months ended
Research and development (R&D) expenses for the first quarter of 2024 decreased to
Selling, general and administrative (SG&A) expenses for the first quarter of 2024 increased to
Operating loss for the first quarter was
Interest expense was
Net loss for the first quarter of 2024 was
Cash, cash equivalents, restricted cash and investments totaled
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
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
The company expects full year 2024 operating expenses to be between
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
Conference Call and Webcast
BioCryst management will host a conference call and webcast at
About
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement its commercialization plans for ORLADEYO, which could take longer or be more expensive than planned; BioCryst’s ability to successfully implement its plans for BCX10013, including that the results of the ongoing proof-of-concept trial may differ from BioCryst’s expectations and that BioCryst may not be able to successfully out-license the late-stage development and commercialization of BCX10013; BioCryst’s ability to successfully progress its pipeline development plans as described herein; risks related to the reduction in size of BioCryst’s R&D organization; the results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; risks related to government actions, including that decisions and other actions, including as they relate to pricing, may not be taken when expected or at all, or that the outcomes of such decisions and other actions may not be in line with BioCryst’s current expectations; the commercial viability of ORLADEYO, including its ability to achieve market acceptance; ongoing and future preclinical and clinical development of product candidates may take longer than expected and may not have positive results; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst’s ability to successfully commercialize its products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst’s business; timing for achieving profitability and positive cash flow may not meet management’s expectations; statements and projections regarding financial guidance and goals and the attainment of such goals may differ from actual results based on market factors and BioCryst’s ability to execute its operational and budget plans; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management's expected ranges. Please refer to the documents BioCryst files periodically with the
BCRXW
Contact:
+1 919 859 7910
jbluth@biocryst.com
CONSOLIDATED FINANCIAL SUMMARY
(In thousands, except per share)
Statements of Operations (unaudited)
Three Months Ended | |||||||
2024 | 2023 | ||||||
Revenues: | |||||||
ORLADEYO | $ | 88,867 | $ | 68,414 | |||
Other | 3,894 | 364 | |||||
Total revenues | 92,761 | 68,778 | |||||
Expenses: | |||||||
Cost of product sales | 1,265 | 931 | |||||
Research and development | 46,493 | 48,388 | |||||
Selling, general and administrative | 59,364 | 47,867 | |||||
Royalty | 127 | 7 | |||||
Total operating expenses | 107,249 | 97,193 | |||||
Loss from operations | (14,488 | ) | (28,415 | ) | |||
Interest income | 4,031 | 3,378 | |||||
Interest expense | (24,506 | ) | (27,396 | ) | |||
Foreign currency losses, net | (51 | ) | (229 | ) | |||
Loss before income taxes | $ | (35,014 | ) | $ | (52,662 | ) | |
Income tax expense | 365 | 671 | |||||
Net loss | $ | (35,379 | ) | $ | (53,333 | ) | |
Basic and diluted net loss per common share | $ | (0.17 | ) | $ | (0.28 | ) | |
Weighted average shares outstanding | 206,064 | 188,509 | |||||
Balance Sheet Data (in thousands)
(unaudited) | (Note 1) | ||||||
Cash, cash equivalents and investments | $ | 336,554 | $ | 388,987 | |||
Restricted cash | 1,798 | 1,804 | |||||
Receivables | 60,586 | 56,950 | |||||
Total assets | 467,892 | 516,960 | |||||
Secured term loan | 308,484 | 303,231 | |||||
Royalty financing obligation | 530,574 | 531,599 | |||||
Accumulated deficit | (1,716,538 | ) | (1,681,159 | ) | |||
Stockholders’ deficit | (476,167 | ) | (455,528 | ) | |||
Shares of common stock outstanding | 206,347 | 205,771 | |||||
Note 1: Derived from audited financial statements.
Reconciliation of Adjusted Net Loss and Adjusted Diluted Earnings Per Share (in thousands)
Three Months Ended | |||||||
2024 | 2023 | ||||||
GAAP net loss | $ | (35,379 | ) | $ | (53,333 | ) | |
Less: One-time R&D restructuring expense | (1,264 | ) | — | ||||
Adjusted net loss | $ | (34,115 | ) | $ | (53,333 | ) | |
GAAP basic and diluted net loss per common share | $ | (0.17 | ) | $ | (0.28 | ) | |
Adjusted basic and diluted net loss per common share | $ | (0.17 | ) | $ | (0.28 | ) |
Source:
2024 GlobeNewswire, Inc., source