On February 23, 2022 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the fourth quarter and full year ended December 31, 2021, and provided a corporate update (Press release, BioCryst Pharmaceuticals, FEB 23, 2022, View Source [SID1234608873]).
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"The successful launch of ORLADEYO, the rapid advancement of our pipeline and the additional capital we acquired last year have transformed BioCryst. With more than $500 million on our balance sheet and ORLADEYO revenue growing to no less than $250 million in 2022, and $1 billion in global peak sales, we are focused on compounding the value of the company by allocating capital to grow ORLADEYO and advancing our complement program to get our oral drugs to patients suffering from many different rare diseases," 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 fourth quarter of 2021 was $46.2 million.
New patient demand for ORLADEYO remains strong and consistent, with a similar number of new patients added in Q4 2021 as in each of the previous three quarters of the year. Patients switching from other prophylactic therapies and acute-only therapy continue to drive the launch. More than half of patients new to ORLADEYO since launch had a previous prophylactic medicine prior to ORLADEYO and most of the remainder were from acute-only treatment.
Most patients are well-controlled on ORLADEYO and remain on therapy. Approximately 70 percent of patients starting ORLADEYO, including those switching from injectable prophylaxis, remain on ORLADEYO in the first year.
The ORLADEYO prescriber base continues to grow significantly as physicians gain real-world experience. In market research, 60 U.S. physicians, who treat an average of seven HAE patients each, reported that they expect to double their use of ORLADEYO, and that ORLADEYO will become their most prescribed prophylactic treatment in the next 12 months.
ORLADEYO is now covered by all major payors and pharmacy benefit managers, which will lead to more patients moving quickly to paid product.
ORLADEYO net revenue is expected to benefit from this wide coverage and continued new patient growth throughout 2022. Because of typical first quarter requirements from payors for prescription reauthorization of specialty products, like ORLADEYO, that can temporarily move patients from paid drug to free product, copayment assistance and Medicare D cost sharing dynamics, the company expects little to no ORLADEYO net revenue growth from Q4 2021 to Q1 2022. The company has accounted for this expected Q1 impact in its expectation that full year 2022 ORLADEYO global net revenues will double year over year to no less than $250 million.
"We are excited by the strong start to the ORLADEYO launch and the favorable experience most HAE patients are having controlling their HAE attacks with an oral, once-daily capsule. With a sizeable base of patients already on therapy, reimbursement in place for the full year in 2022, and more face-to-face opportunities for our commercial team to engage directly with patients and physicians, we are looking forward to more than doubling our revenue this year as we continue on our trajectory to become the market leader in HAE prophylaxis," said Charlie Gayer, chief commercial officer of BioCryst.
ORLADEYO: Global Updates
ORLADEYO has been launched in France, Germany, Japan, Norway, Sweden, the United Arab Emirates and the United Kingdom. The company expects launches in additional countries throughout the year.
Complement Oral Factor D Inhibitor Program – BCX9930
BioCryst is currently enrolling patients in two global pivotal trials, REDEEM-1 and REDEEM-2, with the company’s oral Factor D inhibitor, BCX9930 (500 mg bid), in patients with paroxysmal nocturnal hemoglobinuria (PNH). REDEEM-1 is a randomized, open-label, active, comparator-controlled comparison of the efficacy and safety of BCX9930 monotherapy in approximately 81 PNH patients with an inadequate response to a C5 inhibitor. REDEEM-2 is a randomized, placebo-controlled trial to evaluate the efficacy and safety of BCX9930 as monotherapy versus placebo in approximately 57 PNH patients not currently receiving complement inhibitor therapy. The primary endpoint for both trials is the change from baseline in hemoglobin, assessed at weeks 12 to 24 in REDEEM-1 and at week 12 in REDEEM-2.
The company also has begun patient enrollment for RENEW, a proof-of-concept (PoC) basket trial of oral BCX9930 (500 mg bid) in complement-mediated renal diseases. The trial is being conducted in patients with C3 glomerulopathy (C3G), IgA nephropathy (IgAN), and primary membranous nephropathy (PMN).
BioCryst plans to further advance and expand its Factor D program over the next two years by achieving the following by the end of 2023:
Complete and report data from REDEEM-1 and REDEEM-2
Prepare to submit regulatory approval filings in PNH
Complete the renal PoC basket trial and advance to pivotal trials in C3G, IgAN and PMN
Commence PoC trials in other complement-mediated diseases
Additional Updates
On November 22, 2021, the company announced financing transactions totaling $350 million in new funding for BioCryst from Royalty Pharma and OMERS Capital Markets.
On November 1, 2021, the company announced the appointment of Jinky Ang Rosselli as Chief Data and Insights Officer.
Fourth Quarter 2021 Financial Results
For the three months ended December 31, 2021, total revenues were $47.2 million, compared to $4.0 million in the fourth quarter of 2020. The increase was primarily due to $46.2 million in ORLADEYO net revenue in the fourth quarter of 2021.
Research and development (R&D) expenses for the fourth quarter of 2021 increased to $63.5 million from $35.4 million in the fourth quarter of 2020, primarily due to increased investment in the development of our Factor D program and other research, preclinical and development costs, offset by a slight reduction in spend on the ORLADEYO program following our commercial launch in December 2020.
Selling, general and administrative (SG&A) expenses for the fourth quarter of 2021 increased to $35.4 million, compared to $21.0 million in the fourth quarter of 2020. The increase was primarily due to increased investment to support the commercial launch of ORLADEYO and expanded international operations.
Interest expense was $18.8 million in the fourth quarter of 2021, compared to $5.6 million in the fourth quarter of 2020. The increase was due to service on the royalty and debt financings, which were completed in December 2020 and November 2021.
A one-time non-cash gain of $55.8 million related to the extinguishment of debt was recognized during the fourth quarter of 2021 to write-off the non-recourse PhaRMA Notes and related accrued interest payable.
Net loss for the fourth quarter of 2021 was $17.8 million, or $0.10 per share, compared to a net loss of $60.5 million, or $0.34 per share, for the fourth quarter of 2020. Non-GAAP net loss for the fourth quarter of 2021 was $73.6 million, or $0.40 per share when excluding the one-time non-cash gain on the extinguishment of the non-recourse PhaRMA Notes. A reconciliation between GAAP and non-GAAP net loss is provided in the table below.
Cash, cash equivalents, restricted cash and investments totaled $517.8 million as of December 31, 2021, compared to $302.6 million as of December 31, 2020. Operating cash use for the fourth quarter of 2021 was $29.0 million.
Full Year 2021 Financial Results
For the full year ended December 31, 2021, total revenues were $157.2 million, compared to $17.8 million in the full year ended December 31, 2020. The increase was primarily due to $122.6 million of ORLADEYO net revenue following our commercial launch in December 2020, recognition of a $15.0 million milestone payment from Torii related to the Japanese National Health Insurance System approval of ORLADEYO in Japan, and increased RAPIVAB revenues. These increases in revenue were partially offset by a reduction in royalty revenue (excluding those associated with ORLADEYO sales) of $4.2 million, a reduction in contract revenue of $3.3 million and the recognition of $1.9 million of deferred revenue in the prior year period compared to none in the current year period.
R&D expenses in full year 2021 increased to $208.8 million from $123.0 million in full year 2020, primarily due to increased investment in our Factor D program, and an increase in other research, preclinical and development activities, partially offset by a ramp down of clinical investment related to ORLADEYO, which launched commercially in the U.S. during December 2020.
SG&A expenses in full year 2021 increased to $118.8 million, compared to $67.9 million in full year 2020. The increase was primarily due to increased investment to support the U.S. commercial launch of ORLADEYO and expanded international operations.
Interest and other income was $0.1 million in full year 2021, compared to $9.4 million in full year 2020. The decrease was primarily due to the one-time settlement of arbitration proceedings related to our Seqirus dispute in the first quarter of 2020.
Interest expense was $59.3 million in full year 2021, compared to $14.5 million in full year 2020. The increase was associated with the $125.0 million Term A Loan under the Credit Agreement and Royalty financing obligations which were completed in December 2020 and November 2021.
A one-time non-cash gain of $55.8 million on extinguishment of debt was recognized in the full year 2021 related to the write-off of the non-recourse PhaRMA notes and related accrued interest payable.
Net loss for full year 2021 was $184.1 million, or $1.03 per share, compared to a net loss of $182.8 million, or $1.09 per share, for full year 2020. Non-GAAP net loss for full year 2021 was $239.9 million, or $1.34 per share when excluding the one-time gain on the extinguishment of the non-recourse PhaRMA Notes. A reconciliation between GAAP and non-GAAP net loss is provided in the table below.
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 will allow investors to more accurately understand our 2021 results and more easily compare them to future results. These non-GAAP pro forma measures also correspond with the way we expect Wall Street 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 fourth quarter 2021 "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 gain realized by the extinguishment of the debt from our PhaRMA notes.
Financial Outlook for 2022
Based on the strength of the ORLADEYO launch, and continued growth from new patient demand anticipated throughout the year, the company expects full year 2022 net ORLADEYO revenue to be no less than $250 million. Operating expenses for full year 2022, not including non-cash stock compensation, are expected to be in the range of $440 million to $480 million. The increase year over year is predominantly driven by additional investment in advancing the Factor D program across multiple indications.
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 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 6365545. 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 telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 6365545.