—Q1 2022 ORLADEYO net revenue of
—ORLADEYO net revenue in 2022 expected to be no less than
—Company provides update on BCX9930 investigation—
“We are now over a year into the ORLADEYO launch and are excited to see strong and continuing patient demand and steady expansion in our prescriber base among both new and existing prescribers. These trends continued in the first quarter of 2022 and reinforce our confidence that we will achieve no less than
“We also have made substantial progress in our investigation with BCX9930. Based on our initial findings, we believe that both dose and dosing regimen could be contributing factors to the safety signal we have observed. By the end of the third quarter, we plan to discuss our proposed approach to resume the REDEEM trials, under a revised dosing protocol, with regulators,” Stonehouse added.
Program Updates and Key Milestones
ORLADEYO® (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks
- ORLADEYO net revenue in the first quarter of 2022 was
$49.7 million .
- Approximately 50 percent of patients currently on ORLADEYO have switched from another prophylactic therapy, and half of those patients have come from lanadelumab. These trends continued with new patients starting ORLADEYO in Q1 2022.
- In the first quarter, new patient prescriptions were evenly split between repeat prescribers and new prescribers, with an approximately equal number of new prescriptions coming from the top 500 HAE treaters and the broader set of other HAE treaters.
- The company has completed its latest quarterly survey of another 60 allergists who treat an average of eight HAE patients. These physicians were already using ORLADEYO on 13 percent of their patients and predicted growth to 23 percent over the next 12 months. These findings were in line with previously reported market research from
August 2021 .
- Approximately 80 percent of HAE patients in the
U.S. are insured by payors and pharmacy benefit managers that cover ORLADEYO. Despite prior authorization headwinds early in 2022, approximately 80 percent of patients on ORLADEYO were receiving paid product by the end of the first quarter, a substantial increase from approximately 66 percent of patients in the second half of 2021.
- Most patients are well-controlled on ORLADEYO and remain on therapy. Once payor prior authorization is complete, 78 percent of patients switching from lanadelumab and 73 percent of patients switching from subcutaneous C1 inhibitor remain on ORLADEYO for at least six months. About 70 percent of all patients who receive reimbursed product stay on ORLADEYO for at least 12 months, compared to 60 percent of patients who remain on long-term free product.
- The company expects steady quarterly ORLADEYO net revenue growth throughout the remainder of 2022, with total 2022 ORLADEYO net revenue of no less than
$250 million .
“We are very pleased that strong patient demand continued to drive growth in the first quarter and overcome the traditional Q1 reimbursement headwinds. With outstanding reimbursement in place, patients continuing to enjoy an excellent experience on ORLADEYO and the expansion of both our new and existing prescriber bases, we expect steady growth throughout the remainder of 2022 as we achieve no less than
ORLADEYO: Global Updates
- ORLADEYO has been launched in
Denmark ,France ,Germany ,Japan ,Norway ,Sweden , theUnited Arab Emirates and theUnited Kingdom . The company expects launches in additional countries throughout the year.
Complement Oral Factor D Inhibitor Program – BCX9930
On
Patients in the REDEEM-1, REDEEM-2 and RENEW clinical trials randomized to BCX9930 began those trials by starting immediately at a dose of 500 mg twice-daily. Patients receiving BCX9930 in the long-term extension trial started at lower doses as part of the proof-of-concept dose escalation regimen and were ultimately moved up to 500 mg twice-daily.
Preliminary evidence from the investigation points to both the 500 mg twice-daily dosing level and the immediate start of that dose, without a period at a lower dose first, as plausible contributory factors for the observed increases in serum creatinine.
Based on the initial results of the investigation, and the safety and efficacy data observed in the BCX9930 clinical program at 400 mg twice-daily, the company plans to discuss with regulators whether clinical trials with amended protocols could resume using stepped dosing to 400 mg twice-daily. The company expects to have discussions with regulators by the end of the third quarter.
During the ongoing investigation the company has observed the following:
- Three patients with PNH receiving BCX9930 in the REDEEM trials had early onset, and moderate or severe, elevations in their serum creatinine (2-4 xULN) after several weeks of dosing with 500 mg twice-daily. Two of these patients have been discontinued from therapy and one patient (who had the smallest increase in serum creatinine) continues on BCX9930 at this time.
- The company estimates that one-third of subjects randomized to BCX9930 in the REDEEM studies have had early increases in serum creatinine.
- The company also found a different pattern of slowly evolving, late onset, mild to moderate increases in serum creatinine in approximately 40 percent of patients in the long-term extension of the proof-of-concept trial, after those patients switched to the 500 mg twice-daily dose. This pattern was not observed during treatment with doses lower than 500 mg.
Subsequent to BioCryst voluntarily pausing trial enrollments, the
“As we complete our investigation, we will continue to be comprehensive and deliberate, with a primary focus on patient safety. After consultation with regulators, we will determine the next step for the BCX9930 program,” said Dr.
The company does not plan to provide additional updates on the BCX9930 program until it completes additional regulatory discussions and has more clarity on the next steps for the program.
Additional Updates
- On
February 8, 2022 , the company announced the appointment ofMachelle Sanders to its board of directors.
First Quarter 2022 Financial Results
For the three months ended
Research and development (R&D) expenses for the first quarter of 2022 increased to
Selling, general and administrative (SG&A) expenses for the first quarter of 2022 increased to
Interest expense was
Net loss for the first quarter of 2022 was
Cash, cash equivalents, restricted cash and investments totaled
Financial Outlook for 2022
Based on the strength of the ORLADEYO launch, and continued steady growth from new patient demand anticipated throughout the year, the company expects full year 2022 net ORLADEYO revenue to be no less than
The company had previously expected operating expenses for full year 2022, not including non-cash stock compensation, to be between
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: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst’s business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst’s and its partners’ development, regulatory processes and supply chains, negatively impact BioCryst’s ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst files periodically with the
BCRXW
Investors:
+1 919 859 7910
jbluth@biocryst.com
Media:
Catherine Collier Kyroulis
+1 917 886 5586
ckyroulis@biocryst.com
CONSOLIDATED FINANCIAL SUMMARY | ||||||||
(in thousands, except per share) | ||||||||
Statements of Operations (Unaudited) | ||||||||
Three Months Ended | ||||||||
2022 | 2021 | |||||||
Revenues: | ||||||||
Product sales | $ | 49,546 | $ | 17,871 | ||||
Royalty revenue | 347 | (897 | ) | |||||
Collaborative and other research and development | 30 | 2,085 | ||||||
Total revenues | 49,923 | 19,059 | ||||||
Expenses: | ||||||||
Cost of product sales | 236 | 5,923 | ||||||
Research and development | 65,360 | 42,435 | ||||||
Selling, general and administrative | 34,282 | 22,114 | ||||||
Royalty | 2 | (36 | ) | |||||
Total operating expenses | 99,880 | 70,436 | ||||||
Loss from operations | (49,957 | ) | (51,377 | ) | ||||
Interest and other income | 54 | 26 | ||||||
Interest expense | (23,837 | ) | (12,904 | ) | ||||
Foreign currency (losses) gains, net | (177 | ) | (29 | ) | ||||
Loss before income taxes | (73,917 | ) | (64,284 | ) | ||||
Income tax expense | 279 | - | ||||||
Net loss | $ | (74,196 | ) | $ | (64,284 | ) | ||
Basic and diluted net loss per common share | $ | (0.40 | ) | $ | (0.36 | ) | ||
Weighted average shares outstanding | 184,898 | 177,343 | ||||||
Balance Sheet Data (in thousands) | ||||||||
(Unaudited) | (Note 1) | |||||||
Cash, cash equivalents and investments | $ | 443,468 | $ | 514,430 | ||||
Restricted cash | 3,354 | 3,345 | ||||||
Receivables | 36,415 | 29,413 | ||||||
Total assets | 527,720 | 588,151 | ||||||
Secured term loan | 140,236 | 136,082 | ||||||
Royalty financing obligation | 464,225 | 449,375 | ||||||
Accumulated deficit | (1,281,700 | ) | (1,207,504 | ) | ||||
Stockholders’ deficit | (164,215 | ) | (106,986 | ) | ||||
Shares of common stock outstanding | 185,572 | 184,350 | ||||||
Note 1: Derived from audited financial statements. | ||||||||
Source:
2022 GlobeNewswire, Inc., source