On August 4, 2022 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the second quarter ended June 30, 2022, and provided a corporate update (Press release, BioCryst Pharmaceuticals, AUG 4, 2022, View Source [SID1234617575]).
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"With ORLADEYO firmly established in the marketplace as it steadily grows each quarter towards $1 billion in peak sales, our pipeline of other oral drugs for rare diseases and a balance sheet of nearly $500 million alongside our product revenues, BioCryst is uniquely positioned to bring multiple oral medicines for rare diseases to patients," said Jon Stonehouse, president and chief executive officer of BioCryst.
Program Updates and Key Milestones
ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks
U.S. Launch
ORLADEYO net revenue in the second quarter of 2022 was $65.2 million.
In the second quarter, more new physicians prescribed ORLADEYO than any quarter since the third quarter of 2021.
Prescribing from the top 500 HAE treaters increased in the second quarter with 60 percent of those physicians now having prescribed ORLADEYO. These top 500 physicians accounted for two-thirds of new patient prescriptions in the quarter.
The majority of patients continue to be well controlled on ORLADEYO. Even as the patient base has continued to grow steadily, discontinuations in the second quarter declined to their smallest number since the third quarter of 2021.
Patients continue to have broad and rapid access to ORLADEYO with the median time for a patient to receive reimbursed product following a prescription now under three weeks.
Based on the strong performance of ORLADEYO in the first half of 2022, and the steady quarterly ORLADEYO net revenue growth the company expects in the second half of 2022, the company now expects full year 2022 ORLADEYO net revenue to be between $255 million and $265 million. The company expects peak global net ORLADEYO sales of $1 billion.
"More than a year and a half into the launch of ORLADEYO, we see steady growth and strong momentum, which we expect to continue. The majority of HAE patients are very satisfied with their experience using oral, once-daily ORLADEYO and physicians continue to consistently express their strong intent for future prescribing, which we see translating directly to our growing patient numbers quarter after quarter," said Charlie Gayer, chief commercial officer of BioCryst.
ORLADEYO: Global Updates
In the second quarter, ORLADEYO was approved in Canada and Switzerland. The company expects approvals and launches in additional countries throughout the year.
Also in the second quarter, pricing was finalized in Germany, France and Switzerland.
On June 9, 2022, the company announced it had selected Pint Pharma as its commercial partner for ORLADEYO in Latin America.
Complement Oral Factor D Inhibitor Program – BCX9930
On August 4, 2022, the company announced that the U.S. Food and Drug Administration (FDA) has lifted its partial clinical hold on the BCX9930 program. The company will resume enrollment in global clinical trials under revised protocols at a reduced dose of 400 mg twice daily of BCX9930. This includes the REDEEM-1 and REDEEM-2 pivotal trials in patients with paroxysmal nocturnal hemoglobinuria (PNH) and the RENEW proof-of-concept trial in patients with C3 glomerulopathy (C3G), immunoglobulin A nephropathy (IgAN) and primary membranous nephropathy (PMN).
Clinical evidence and recent laboratory studies have informed the company’s hypothesis that crystals form in the kidneys of some patients. The company believes that lowering the dose to 400 mg and ensuring adequate hydration will dilute the concentration of drug in the urine below the threshold where crystals can form.
The company’s goal is to find a safe and effective dose for BCX9930. The company expects this can be accomplished in a reasonable time frame after resuming enrollment, in a relatively small number of patients given the rate and timing of the serum creatinine rises in patients prior to the enrollment pause.
If successful, the company plans to invest more significantly in BCX9930 to tap the full potential of reaching many patients suffering from a number of alternative pathway diseases, and, if not successful, the company will stop investment in BCX9930 and move on to other molecules in the pipeline.
Additional Updates
On April 27, 2022, the company announced the European Medicines Agency (EMA) had granted PRIME designation to BioCryst’s ALK-2 inhibitor, BCX9250, for the treatment of fibrodysplasia ossificans progressiva.
The EMA also has recently granted orphan drug designation and the FDA has granted fast track status for BCX9250.
Second Quarter 2022 Financial Results
For the three months ended June 30, 2022, total revenues were $65.5 million, compared to $50.0 million in the second quarter of 2021 (+31 percent year-over-year (y-o-y)). The increase was primarily due to $65.2 million in ORLADEYO net revenue in the second quarter of 2022, compared to $28.5 million in ORLADEYO net revenue in the second quarter of 2021 (+129 percent y-o-y). The $65.2 million of ORLADEYO net revenue in the second quarter of 2022 included approximately $2.2 million of non-repeating reimbursement related accrual releases.
Research and development expenses for the second quarter of 2022 increased to $62.0 million from $52.9 million in the second quarter of 2021 (+17 percent y-o-y), primarily due to increased investment in the development of our Factor D program, including BCX9930, as well as other research, preclinical and development costs.
Selling, general and administrative expenses for the second quarter of 2022 increased to $38.0 million, compared to $26.3 million in the second quarter of 2021 (+44 percent y-o-y). The increase was primarily due to increased investment to support the commercial launch of ORLADEYO and expanded international operations.
Interest expense was $24.0 million in the second quarter of 2022, compared to $13.5 million in the second quarter of 2021 (+78 percent y-o-y). The increase was due to service on the royalty financings, which were completed in November 2021.
Net loss for the second quarter of 2022 was $58.9 million, or $0.32 per share, compared to a net loss of $43.2 million, or $0.24 per share, for the second quarter of 2021.
Cash, cash equivalents, restricted cash and investments totaled $418.9 million at June 30, 2022, compared to $222.8 million at June 30, 2021. Operating cash use for the second quarter of 2022 was $27.9 million.
Additionally, on July 29, 2022, having achieved all required revenue-based milestones, the company drew the available $75 million tranche under its existing credit facility from Athyrium Capital Management. On a pro-forma basis, net of fees, this results in pro-forma cash of approximately $492 million.
Financial Outlook for 2022
Based on the strength of the ORLADEYO launch through the first half of 2022, and the continued steady growth from new patient demand the company expects for the remainder of the year, the company expects full year 2022 net ORLADEYO revenue to be between $255 million and $265 million.
Based on the reduced spending on the BCX9930 program in the first half of the year, and lower than projected spending on the program for the remainder of the year, the company now expects operating expenses for full year 2022, not including non-cash stock compensation, to be between $390 million and $400 million.
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 866-374-5140 for domestic callers and 404-400-0571 for international callers and using conference ID 68509725#. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A replay of the call will be available on the company website.