On July 30, 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the second quarter ended June 30, 2020 and revised upward its full-year 2020 financial guidance for total cystic fibrosis (CF) product revenues (Press release, Vertex Pharmaceuticals, JUL 30, 2020, View Source [SID1234562615]).
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"This has been an exceptional first half for Vertex on all fronts and most importantly, in our efforts to bring our CF medicines to more people around the world. We have seen remarkable uptake of TRIKAFTA in the U.S., with the majority of eligible patients now taking this medicine; and in Europe, we secured a positive CHMP opinion earlier than expected and entered into a landmark expansion of our reimbursement agreement with NHS England that will give patients in England access to this medicine rapidly following European Commission approval," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "Additionally, despite the challenges of this unprecedented year, we have continued to make steady progress in our research programs and across our clinical development pipeline that will position us for continued growth into the future."
Total product revenues increased 62% compared to the second quarter of 2019, primarily driven by the uptake of TRIKAFTA in the U.S. and the uptake of our medicines outside the U.S. following the completion of key reimbursement agreements in 2019.
GAAP and Non-GAAP net income increased 213% and 110%, respectively, compared to the second quarter of 2019, largely driven by the strong growth in total product revenues.
Cash, cash equivalents and marketable securities as of June 30, 2020 were $5.5 billion, an increase of approximately $1.6 billion compared to $3.8 billion as of December 31, 2019.
Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to the second quarter of 2019, primarily due to the incremental investment to support the global use of Vertex’s medicines and the expansion of Vertex’s pipeline in CF and other new disease areas.
GAAP income taxes decreased compared to the second quarter of 2019. Non-GAAP income taxes increased compared to the second quarter of 2019 primarily due to Vertex’s increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex’s provision for income taxes.
Key Business Highlights:
TRIKAFTA/KAFTRIO (elexacaftor, tezacaftor and ivacaftor)
•The majority of the approximately 18,000 eligible patients have initiated treatment with TRIKAFTA.
•In June, the European Medical Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for KAFTRIO for the treatment of European CF patients ages 12 and older with one F508del mutation and one minimal function mutation or two F508del mutations.
•In June, Vertex also announced the expansion of its reimbursement agreement with the National Health Service (NHS) England to include KAFTRIO, ahead of the medicine’s anticipated approval by the European Commission. If approved, KAFTRIO will be available to people with CF in England ages 12 and older with one F508del mutation and one minimal function mutation or two F508del mutations.
•In July, Vertex announced positive Phase 3 study results for TRIKAFTA in people with CF ages 12 and older who have one copy of the F508del mutation and one gating or residual function mutation. This study was a post-marketing commitment and will be submitted to the U.S. FDA. In addition, the study data will be submitted to the EMA to support future indication expansion of the European Union (EU) label.
•Data from the Phase 3 study evaluating the use of the elexacaftor, tezacaftor and ivacaftor triple combination in children with CF ages 6 through 11 who have two copies of the F508del mutation or who have one F508del mutation and one minimal function mutation is expected in the second
half of 2020. Pending data from the study, Vertex will submit a supplemental New Drug Application (sNDA) to the U.S. FDA in the fourth quarter of 2020 for children ages 6 through 11 with at least one F508del mutation, followed by regulatory submissions in other countries.
SYMDEKO/SYMKEVI (tezacaftor and ivacaftor)
•The EMA review of the application for use of SYMKEVI in patients ages 6 through 11 in Europe is ongoing. If approved, this will be the first CFTR modulator to treat patients ages 6 through 11 with residual function mutations in the EU.
KALYDECO (ivacaftor)
•In June, Vertex announced that the European Commission granted approval of the label extension for KALYDECO for the treatment of children and adolescents ages 6 months and older who have the R117H mutation.
Development Pipeline:
Vertex continues to progress a broad pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is noted below:
Beta Thalassemia and Sickle Cell Disease:
•Vertex and its partner CRISPR Therapeutics provided new clinical data at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress from the two ongoing Phase 1/2 studies of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with transfusion-dependent beta thalassemia (TDT) and in patients with severe sickle cell disease (SCD). Data from two TDT patients demonstrated clinical proof-of-concept for CTX001 in this disease, and longer duration data from one SCD patient showed a durable effect on HbF levels and the patient was free of vaso-occlusive crises. Screening, enrollment and mobilization in these studies is ongoing; conditioning and dosing have been resumed following temporary COVID-19-related pauses in both studies. Vertex and CRISPR Therapeutics expect to report data from additional patients in the second half of 2020.
Alpha-1 Antitrypsin (AAT) Deficiency:
•Vertex is evaluating multiple compounds with the potential to correct the misfolding of Z-AAT protein in the liver, in order to increase the levels of functional AAT in the blood. Misfolded Z-AAT protein is the root cause of AAT deficiency.
•Enrollment and dosing have been re-initiated at some but not all sites following a temporary COVID-19-related pause in a Phase 2 proof-of-concept study designed to evaluate the levels of circulating, functional AAT protein after treatment with VX-814.
•A Phase 2 proof-of-concept study for a second Z-AAT corrector, VX-864, was initiated in July.
APOL1-mediated Kidney Diseases:
•Vertex is evaluating the potential for inhibitors of APOL1 function to reduce proteinuria in people with serious kidney diseases, including focal segmental glomerulosclerosis (FSGS).
•Enrollment is underway at multiple clinical trial sites in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated FSGS after treatment with VX-147.
Type 1 Diabetes (T1D):
•Vertex is developing a cell therapy designed to replace insulin-producing islet cells in people with T1D. Two opportunities exist for the transplant of these functional islets into patients: 1) transplantation of islet cells alone, using immunosuppression to protect the implanted cells and 2) implantation of the islet cells inside a novel immunoprotective device.
•Vertex plans to submit an Investigational New Drug (IND) application to the U.S. FDA for the islet cells alone program in late 2020 to support evaluation of this potential therapy in patients with T1D.