De Monitor - Pharming Professor schreef op 6 mei 2021 14:06:
Bron: Biocryst
ORLADEYO™ (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks
ORLADEYO net revenue in the first quarter of 2021, the first full quarter of launch in the United States, was $10.9 million.
The majority of ORLADEYO revenue in the first quarter of 2021 came from new patients who switched to ORLADEYO from either injectable/infused prophylactic medications or from acute-only treatment. The remainder came from patients transitioning from clinical trials and the company’s early access program.
European Approvals and Launches
On April 30, 2021, the company announced that the European Commission (EC) had approved oral, once-daily ORLADEYO for the prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older. The EC approval of ORLADEYO is applicable to all European Union member states plus Iceland, Norway and Liechtenstein.
BioCryst has its European commercial team in place and expects to launch ORLADEYO in the second quarter in Germany, with launches in other European markets to follow. HAE patients in France currently have access to ORLADEYO through an Autorisation Temporaire d'Utilisation de cohorte (cohort ATU).
On March 2, 2021, the company announced the submission of a marketing authorization application (MAA) to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) seeking approval of ORLADEYO for the prevention of recurrent HAE attacks in HAE patients 12 years and older. The MAA was submitted under the MHRA’s new European Commission Decision Reliance Procedure. If approved, ORLADEYO would be the first oral, once-daily therapy in the United Kingdom to treat patients with HAE.
Japanese Approval and Launch
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 HAE in adults and pediatric patients 12 years and older.
On April 14, 2021, the company announced that the Japanese National Health Insurance System (NHI) approved the addition of oral, once-daily ORLADEYO™ (berotralstat) to the NHI drug price list on April 21, 2021. This triggered a $15 million milestone payment to BioCryst from Torii Pharmaceutical Co., Ltd., the company’s commercial partner in Japan.
ORLADEYO is the first and only prophylactic HAE medication approved in Japan. Torii launched ORLADEYO in Japan on April 23, 2021. BioCryst will receive tiered royalties ranging from 20 percent to 40 percent of Japanese net sales.
Complement Oral Factor D Inhibitor Program – BCX9930
BioCryst has reached agreement with the U.S. Food and Drug Administration (FDA) that the primary endpoint for the upcoming pivotal trials in paroxysmal nocturnal hemoglobinuria (PNH) is change from baseline in hemoglobin. On March 22, 2021 the company announced that BCX9930 increased hemoglobin from baseline by a mean of 3.3 g/dL in C5 inadequate response (no prior treatment with C5 inhibitors) patients and 3.5 g/dL in treatment-naïve patients and reduced transfusions in an ongoing dose-ranging trial in PNH patients. BCX9930 was safe and generally well-tolerated in the trial.
In the second half of 2021, the company plans to advance directly into PNH pivotal trials with oral BCX9930, at a dose of 500 mg bid, in patients naïve to C5 inhibitors, and patients with an inadequate response to C5 inhibitors. The goal of the pivotal trials is to achieve a broad indication for BCX9930 to treat PNH as oral monotherapy. Also in the second half of 2021, the company plans to initiate a proof of concept trial of oral BCX9930 (500 mg bid) in renal complement-mediated diseases.
Additional Updates
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.
On March 19, 2021, the company announced the appointment of Helen Thackray, M.D., FAAP, to the newly created position of chief research and development officer.
First Quarter 2021 Financial Results
For the three months ended March 31, 2021, total revenues were $19.1 million, compared to $4.8 million in the first quarter of 2020. The increase was primarily due to $10.9 million in ORLADEYO net revenue in the first quarter of 2021.
Research and development (R&D) expenses for the first quarter of 2021 increased to $42.4 million from $29.9 million in the first quarter of 2020, primarily due to increased investment in the development of BCX9930 as well as other research, preclinical and development costs, offset by a reduction in spend on the ORLADEYO program following our commercial launch in December 2020.
Selling, general and administrative (SG&A) expenses for the first quarter of 2021 increased to $22.1 million, compared to $15.9 million in the first quarter of 2020. The increase was primarily due to increased investment in to support the U.S. commercial launch of ORLADEYO and expanded international operations.
Interest and other income in the first quarter of 2021 was $6.4 million lower than the first quarter of 2020, primarily due to the partial arbitration award in the first quarter of 2020 related to our Seqirus dispute.
Interest expense was $12.9 million in the first quarter of 2021, compared to $3.0 million in the first quarter of 2020. The increase was due to service on the royalty and debt financings which were completed in December 2020.
Net loss for the first quarter of 2021 was $64.3 million, or $0.36 per share, compared to a net loss of $37.6 million, or $0.24 per share, for the first quarter of 2020.
Cash, cash equivalents, restricted cash and investments totaled $244.4 million at March 31, 2021, compared to $114.6 million at March 31, 2020. Operating cash use for the first quarter of 2021 was $60.0 million.