On February 25, 2021 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the fourth quarter and full year ended December 31, 2020, and provided a corporate update (Press release, BioCryst Pharmaceuticals, FEB 25, 2021, View Source [SID1234575596]).
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"With two approvals for ORLADEYO, our launch in the U.S., proof of concept data for BCX9930 in complement-mediated diseases, a successful phase 1 trial of BCX9250 for FOP and the addition of more than $425 million through our May and December financings, 2020 was a transformational year for BioCryst," said Jon Stonehouse, president and chief executive officer of BioCryst.
"Using our strong balance sheet as a foundation, we expect to continue this transformation in 2021 with ORLADEYO generating revenue in the U.S., Japan and Europe, and the opportunity to significantly advance the 9930 program as an oral monotherapy for both PNH, and renal complement-mediated diseases," Stonehouse added.
Program Updates and Key Milestones
ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks
BioCryst launched ORLADEYO in the United States following U.S. Food and Drug Administration (FDA) approval on December 3, 2020, and product shipments began on December 16, 2020.
On January 22, 2021, the company announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan had granted marketing and manufacturing approval for oral, once-daily ORLADEYO 150 mg for prophylactic treatment of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
ORLADEYO is the first and only prophylactic HAE medication approved in Japan and will be commercialized in Japan by BioCryst’s partner, Torii Pharmaceutical Co., Ltd. OrphanPacific, Inc. is BioCryst’s representative partner in Japan and holds the marketing authorization.
Torii will launch ORLADEYO in Japan following the successful completion of BioCryst’s pricing negotiations with the Japanese National Health Insurance System (NHI).
BioCryst is eligible to receive an additional milestone payment of $15 million from Torii upon receipt of a reimbursement price from the NHI in excess of the threshold specified in the agreement with Torii. In addition, BioCryst will receive tiered royalties ranging from 20 percent to potentially 40 percent of Japanese net sales.
In Europe, the Committee for Medicinal Products for Human Use (CHMP) is scheduled to review the ORLADEYO marketing authorization application this week. The company expects approval from the European Commission (EC) approximately 60 days following a positive opinion from the CHMP.
On October 30, 2020, the company announced that the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) had granted ORLADEYO a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). Under the EAMS, HAE patients in the UK aged 12 years and older can gain access to ORLADEYO for the routine prevention of recurrent attacks of HAE before the drug is granted marketing authorization by the EC.
In the fourth quarter of 2020, the company presented data in several manuscripts and abstracts.
On October 22, 2020, the company announced that data from the first 24 weeks of the Phase 3 APeX-2 trial of ORLADEYO in 121 HAE patients ages 12 years or older had been published online by the Journal of Allergy and Clinical Immunology.
On November 13, 2020, the company presented data in several abstracts at the 2020 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology across HAE patients, caregivers and treating physicians showing many patients experience a significant treatment burden associated with current prophylactic HAE therapies.
On November 30, 2020, the company announced that the journal Allergy had published data from the APeX-J trial, a randomized, placebo-controlled trial conducted in Japan evaluating ORLADEYO for the prophylactic treatment of HAE.
Complement Oral Factor D Inhibitor Program – BCX9930
The company has completed the enrollment of its ongoing dose ranging trial in treatment-naïve (no prior treatment with C5 inhibitors) paroxysmal nocturnal hemoglobinuria (PNH) patients, and PNH patients with an inadequate response to C5 inhibitors. The company plans to present data from the 16 enrolled PNH patients (10 treatment naïve and six inadequate C5 responders) at its upcoming R&D day on March 22.
In previously reported data from treatment-naïve (no prior treatment with C5 inhibitors) PNH patients receiving doses of oral BCX9930 through 400 mg bid, oral BCX9930 drove rapid and dose-dependent reductions in key biomarkers, including LDH, and increasing hemoglobin levels in all PNH patients in the trial. Increases in hemoglobin levels were maintained without transfusions. BCX9930 has been safe and well tolerated at all doses in the trial. No drug-related serious adverse events have been reported.
Additional Updates
On December 7, 2020, the company announced transactions totaling $325 million in funding for BioCryst, with $250 million available at closing, to support the launch of ORLADEYO in HAE and the development of BCX9930. Royalty Pharma provided BioCryst with an upfront cash payment of $125 million and will receive royalties on direct annual net sales of ORLADEYO up to $550 million, and a tiered percentage of sublicense revenue for ORLADEYO in certain territories. In addition, Royalty Pharma will receive a 1.0% royalty on global net sales of BCX9930, if approved. A fund managed by Athyrium Capital Management provided BioCryst with a $200 million credit facility, of which BioCryst drew $125 million at closing. The additional capital will be available in two tranches at BioCryst’s option, upon reaching defined revenue milestones. The credit facility bears interest at LIBOR + 8.25% (with a LIBOR floor of 1.75%) and is interest-only for the entire five-year term, with all outstanding principal due at maturity.
On December 21, 2020, the company announced that in a Phase 1 clinical trial with BCX9250, an oral activin receptor-like kinase-2 (ALK-2) inhibitor discovered and developed by BioCryst for the treatment of fibrodysplasia ossificans progressiva, BCX9250 was safe and well tolerated at all doses studied, with linear and dose-proportional exposure supporting once-daily dosing.
On February 3, 2021, the company announced that the FDA had approved a supplemental new drug application for RAPIVAB (peramivir injection) expanding the patient population of RAPIVAB for the treatment of acute uncomplicated influenza to include patients six months and older who have been symptomatic for no more than two days. Prior to this approval, RAPIVAB had been indicated for patients two years and older.
Fourth Quarter 2020 Financial Results
For the three months ended December 31, 2020, total revenues were $4.0 million, compared to $39.7 million in the fourth quarter of 2019. In the fourth quarter of 2019, we recognized revenue of $20.1 related to a one-time upfront milestone per the Torii agreement. Additionally, in that period we recognized $13.9 million of RAPIVAB product sales under our U.S. Department of Health and Human Services (HHS) contract, while in the fourth quarter 2020 we had no RAPIVAB product sales under our HHS contract. ORLADEYO revenues in the fourth quarter of 2020 were $0.1 million.
Research and development (R&D) expenses for the fourth quarter of 2020 increased to $35.4 million from $26.8 million in the fourth quarter of 2019, primarily due to increased investment in our Factor D and galidesivir programs, partially offset by a ramp down of clinical investment related to ORLADEYO, which launched commercially in the U.S. during December 2020.
Selling, general and administrative (SG&A) expenses for the fourth quarter of 2020 increased to $21.0 million, compared to $10.5 million in the fourth quarter of 2019. The increase was primarily due to increased investment in commercial activities to support the U.S. launch of ORLADEYO.
Interest expense was $5.6 million in the fourth quarter of 2020, compared to $3.1 million in the fourth quarter of 2019. This increase was due to service on the royalty and debt financings which were completed in December 2020. As part of those financings, there was also a loss on debt extinguishing related to the closing of our secured credit facility with MidCap Financial.
Net loss for the fourth quarter of 2020 was $60.5 million, or $0.34 per share, compared to a net loss of $2.6 million, or $0.02 per share, for the fourth quarter of 2019.
Cash, cash equivalents, restricted cash and investments totaled $302.6 million at December 31, 2020, and reflect an increase from $137.8 million at December 31, 2019. Cash, cash equivalents, restricted cash and investments include $250 million in cash received through transactions with Royalty Pharma and Athyrium Capital Management in December 2020. Operating cash use for the fourth quarter of 2020 was $49.9 million, and for the full year of 2020 was $147.9 million.
Full Year 2020 Financial Results
For the full year ended December 31, 2020, total revenues were $17.8 million, compared to $48.8 million in the full year ended December 31, 2019. In the fourth quarter of 2019 we recognized revenue of $20.1 related to a one-time upfront milestone per the Torii agreement, with the remaining amount of $1.9 million recognized in 2020. Additionally, in the fourth quarter of 2019 we recognized $13.9 million of RAPIVAB product sales under our HHS contract, while in the fourth quarter 2020 we had no RAPIVAB product sales under our HHS contract.
R&D expenses in full year 2020 increased to $123.0 million from $107.1 million in full year 2019, primarily due to increased investment in our Factor D and galidesivir programs, 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 2020 increased to $67.9 million, compared to $37.1 million in full year 2019. The increase was primarily due to increased investment in commercial activities to support the U.S. launch of ORLADEYO.
Interest and other income was $9.4 million in full year 2020, compared to $1.9 million in full year 2019. The increase was primarily due to the settlement of arbitration proceedings related to our Seqirus dispute in the first quarter of 2020.
Interest expense was $14.5 million in full year 2020, compared to $11.9 million in full year 2019. This was due to service on the royalty and debt financings which were completed in December 2020. As part of those financings, there was also a loss on debt extinguishing related to the closing of our secured credit facility with MidCap Financial.
Net loss for full year 2020 was $182.8 million, or $1.09 per share, compared to a net loss of $108.9 million, or $0.94 per share, for full year 2019.
Financial Outlook for 2021
In the launch period for ORLADEYO, the company is not providing specific revenue or operating expense guidance. Based on our expectations for revenue, operating expenses, and our option to access an additional $75 million from our existing credit facility, we believe our current cash runway takes us into 2023.
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 # 6779206. 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 # 6779206.