On October 23, 2019 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the third quarter ended September 30, 2019 (Press release, BioMarin, OCT 23, 2019, View Source [SID1234542437]).
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Total Net Product Revenues for the third of quarter 2019 increased to $450.9 million, compared to $386.3 million for the third quarter of 2018. The increase in Net Product Revenues was attributed to the following:
Vimizim Net Product Revenues increased by $40.2 million, or 33%, driven primarily by increased sales volume driven by government orders in certain Middle Eastern countries, a large order from Brazil as well as smaller orders from other Latin American countries;
Palynziq Net Product Revenues increased by $20.0 million, driven by a combination of revenue from patients achieving maintenance dosing and new patients initiating therapy in the U.S. Palynziq received approval from the U.S. Food and Drug Administration (FDA) in May 2018 and launched in the third quarter of that year. Palynziq received European Medicines Agency (EMA) approval in May 2019 and commercial sales in Europe are expected to commence in the fourth quarter of 2019;
Brineura Net Product Revenues increased by $9.9 million, or 100%, due in large part by growth in the number of patients across all regions; and
Kuvan Net Product Revenues increased by $7.3 million, or 6%, primarily driven by an increase in the number of patients in North America; partially offset by
Naglazyme Net Product Revenues decreased by $8.7 million, or 8%, primarily due to decreased sales volume driven by government ordering patterns from certain Latin American and European countries; and
Aldurazyme Net Product Revenues decreased $4.8 million, due to the timing of customer acceptance for product shipped to Genzyme in the third quarter for which no revenue was recognized as of September 30, 2019.
The increase in GAAP Net Income for the third quarter of 2019, compared to GAAP Net Loss the same period in 2018 was primarily due to the following:
increased gross profits of $51.3 million driven by increased product sales;
increased tax benefit, which is primarily attributed to quarterly fluctuations in the mix and timing of our profits and losses on a territorial basis and reversals of certain tax reserves that were no longer required, partially offset by
higher selling, general and administrative (SG&A) expense related to pre-commercialization activities for valoctocogene roxaparvovec, support of the EU commercial launch and continued U.S. expansion of Palynziq, and increased general and administrative expense primarily attributed to personnel-related costs resulting from increased headcount to support our growth; and
higher research and development (R&D) expense related to preclinical activities for BMN 307 and clinical activities for the Company’s vosoritide and valoctocogene roxaparvovec development programs, partially offset by decreased R&D expense related to Palynziq for which we began capitalizing manufacturing costs upon FDA approval in May 2018, and a decrease in tralesinidase alfa clinical manufacturing costs. R&D expenses in the quarter were consistent with 2019 guidance despite the acceleration of the valoctocogene roxaparvovec development program and subsequent activities implemented to pursue an expedited regulatory path forward.
Key Program Highlights
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Valoctocogene roxaparvovec gene therapy for hemophilia A: The European Medicines Agency (EMA) recently granted BioMarin’s request for accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A. Accelerated assessment reduces the time-frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a Marketing Authorization Application (MAA) for an Advanced Therapy Medicinal Product (ATMP). Applications are eligible for accelerated assessment if the CHMP and CAT decide the product is of major interest for public health, particularly from the point of view of therapeutic innovation. Evaluating a MAA under the EMA centralized procedure can take up to 210 days, not counting clock stops when applicants are requested to provide additional information. On request, the CHMP and CAT can reduce the time-frame to 150 days if the applicant provides sufficient justification for an accelerated assessment. The decision to grant accelerated assessment has no impact on the eventual CHMP and CAT opinion on whether a marketing authorization should be granted.
On July 8, the Company announced that based on recent meetings with health authorities in the U.S. and Europe, it plans to submit marketing applications to both the FDA and the EMA in the fourth quarter of 2019 for valoctocogene roxaparvovec with the 6e13 vg/kg dose. The submissions will be based on the recently completed Phase 3 interim analysis and the updated three-year Phase 1/2 data of patients treated with valoctocogene roxaparvovec. Both submissions are expected to represent the first time a gene therapy product for any type of hemophilia indication will be reviewed for marketing authorization by health authorities.
Enrollment in the GENEr8-1 Phase 3 study is expected to be complete by R&D Day with 52-week results from the 130 subjects expected at the end of 2020. Although the trial is open label, BioMarin has implemented a data access plan designed to significantly mirror a blinded trial. This restricts the release of any ongoing data to a small group of medical personnel monitoring and managing the trial, and then, only to the extent necessary to perform their monitoring responsibilities.
The Company has chosen to cease development of the 4e13 vg/kg dose of valoctocogene roxaparvovec given the overwhelming preference by patients to be treated with the 6e13 vg/kg dose.
BioMarin intends to provide a 4 year update with the 6e13 vg/kg dose subjects and a 3 year update with the 4e13 vg/kg dose subjects from the ongoing Phase 2 study in mid-2020.
Palynziq for PKU: Palynziq, an injection to reduce blood Phe concentrations in adult patients with PKU, was added to BioMarin’s commercial product portfolio upon its U.S. approval May 2018. As of September 30, 2019, 670 patients were on reimbursed Palynziq, with an additional 153 naïve patients enrolled and awaiting their first treatment with commercial Palynziq. Of the 670 patients on therapy at the end of the third quarter, 528 were formerly naïve patients and 142 had transitioned from clinical studies. Of the 125 PKU clinics in the U.S., 97 unique clinics had at least one complete patient enrollment in the REMS program as of September 30, 2019.
On May 6, 2019, the European Commission (EC) granted marketing authorization for Palynziq at doses of up to 60 milligrams once daily, to reduce blood Phe concentrations in patients with PKU aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options. The Company is in the process of securing reimbursement on a country-by-country basis across the European Union and anticipates meaningful revenue contributions from this region in 2020.
Vosoritide for children with achondroplasia: The vosoritide development program includes four distinct areas of focus to support global approval, including a large contemporaneous natural history study which is underway. The global Phase 3 study is a randomized, double-blind placebo-controlled study of vosoritide in approximately 110 children with achondroplasia between the ages of 5 to 14 years. Data from this study is expected by year-end 2019.
The Company plans to share 54-month results from the ongoing Phase 2 study with vosoritide in children ages 5 to 14 years at the upcoming R&D Day planned for November 14. These data are expected to corroborate maintenance of effect at the time of anticipated marketing application submissions.
The fourth component of the Company’s global development program with vosoritide, includes a large Phase 2 study in infants and young children (newborn to 60 months old) with achondroplasia, to determine the impact of treatment in this age group. Three cohorts, segmented by age, are at various stages of enrollment in this study. Cohort 1 includes children ages 24 to 60 months old and has completed enrollment. Cohort 2 includes children ages 6 to 24 months old and will complete enrollment by year-end. Cohort 3, includes children ages 0 to 6 months old and began enrolling earlier this month.
BMN 307 gene therapy product candidate for phenylketonuria (PKU): On October 21, 2019, BioMarin was granted Orphan drug designation from the FDA for BMN 307 for the treatment of phenylketonuria. On September 26, 2019, the Company submitted a CTA with the medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K.
Preclinical data with BMN 307 demonstrated a lifetime Phe correction sustained at 80 weeks in mouse models. BMN 307 is an AAV vector containing the DNA sequence that codes for the phenylalanine hydroxylase enzyme that is deficient in people with PKU. Product to support clinical evaluation is being produced at BioMarin’s gene therapy manufacturing facility, where valoctocogene roxaparvovec is currently made, using a commercial scale manufacturing process to facilitate rapid clinical development.
Tralesinidase alfa (formerly referred to as BMN 250) for Mucopolysaccharidosis IIIB (MPS IIIB) or Sanfilippo Syndrome, Type B: On October 23, 2019, the Company announced that it had entered into a licensing agreement with Allievex Corp. (Allievex) for tralesinidase alfa, an investigational Enzyme Replacement Therapy (ERT) for MPS IIIB or Sanfilippo Syndrome Type B. Under the terms of the agreement, Allievex will receive a worldwide, exclusive license to tralesinidase alfa. BioMarin is entitled to receive a minority equity stake in Allievex, milestone payments if certain development, regulatory and sales milestones are met by Allievex and royalties on net sales of tralesinidase alfa. Upon closing of the transaction, Allievex will assume all financial obligations associated with the development and commercialization of tralesinidase alfa other than certain continued manufacturing activities that will be paid by BioMarin. BioMarin will transfer all tralesinidase alfa-related clinical and regulatory activity and responsibilities to Allievex during a transition period following closing of the transaction. Tralesinidase alfa is currently being evaluated in ongoing natural history and clinical trials.
R&D Day to be held in New York November 14, 2019: The Company plans to hold an investor event to discuss potential new product candidates, vosoritide, valoctocogene roxaparvovec status and other general corporate updates. Please email [email protected] for more information.
BioMarin will host a conference call and webcast to discuss third quarter 2019 financial results today, Wednesday, October 23, 2019 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.
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Conference ID: 6989536
Conference ID: 6989536