On April 29, 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the first quarter ended March 31, 2020 and revised upward its full-year 2020 financial guidance for total cystic fibrosis (CF) product revenues (Press release, Vertex Pharmaceuticals, APR 29, 2020, View Source [SID1234556772]).
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"The COVID-19 pandemic has presented unprecedented challenges to societies, communities and businesses around the world, and while these global challenges will continue for some time to come, I am very proud of how Vertex has responded to ensure that we continue to deliver on our mission for patients, keep our employees safe and achieve our business goals," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "Importantly, our business continues to grow, and throughout the first quarter, thousands of patients initiated treatment with our medicines worldwide. The U.S. launch of TRIKAFTA has been remarkable, with the majority of eligible patients having now initiated treatment with this medicine. This strong interest reflects TRIKAFTA’s substantial benefits for patients and has resulted in significant increases in revenue to support continued investment in both our internal pipeline and business development efforts to support future growth. Looking ahead, we continue to be differentiated by our focus on serial innovation, investment in transformative medicines aimed at the underlying cause of disease, the breadth of our pipeline and capabilities and our financial strength."
Total product revenues increased 77% compared to the first 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 each increased more than 120% compared to the first quarter of 2019, largely driven by the strong growth in total product revenues.
Cash, cash equivalents and marketable securities as of March 31, 2020 were $4.2 billion, an increase of approximately $400 million 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 first 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 and Non-GAAP income taxes increased compared to the first quarter of 2019 primarily due to Vertex’s increased operating income. Refer to "Supplemental Income Tax Information" for discussion of the cash versus non-cash components of Vertex’s provision for income taxes.
Full-Year 2020 Financial Guidance
Vertex today revised upward its guidance for full-year 2020 CF product revenues and reiterated its guidance for GAAP and non-GAAP combined R&D and SG&A expenses and for its non-GAAP effective tax rate, as summarized below:
Key Business Highlights:
Approved Medicines:
Supply Chain for Approved CF Medicines
The global COVID-19 outbreak has not had any impact on the continuity of Vertex’s supply chain for its approved medicines.
Vertex remains highly confident in its ability to continue to supply all of its approved medicines to patients around the world.
TRIKAFTA (elexacaftor, tezacaftor and ivacaftor)
Vertex has seen rapid uptake of TRIKAFTA across all groups of eligible patients following approval of this medicine by the U.S. Food and Drug Administration (FDA) in October 2019. The majority of the approximately 18,000 eligible patients have now initiated treatment with TRIKAFTA.
In Europe, the Marketing Authorization Application for the elexacaftor, tezacaftor and ivacaftor triple combination in patients with at least one F508del mutation ages 12 and older continues to be under review with the European Medicines Agency (EMA).
Vertex also recently submitted applications for approval of the elexacaftor, tezacaftor and ivacaftor triple combination for patients with at least one F508del mutation ages 12 and older in Australia and Switzerland.
Vertex recently completed enrollment for a Phase 3 study evaluating the use of the elexacaftor, tezacaftor and ivacaftor triple combination regimen 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. Pending data from the study, Vertex plans to submit a supplemental New Drug Application (sNDA) to the U.S. FDA in the second half of 2020 for children ages 6-11 with at least one F508del mutation, followed by regulatory submissions in other countries.
KALYDECO (ivacaftor)
Vertex recently completed the submission of an sNDA to the U.S. FDA and Type 2 variation to the EMA for the use of KALYDECO in infants ages four to less than six months. KALYDECO is currently approved for use in infants as young as six months of age in both geographies.
Development Pipeline:
Vertex continues to progress its expanding pipeline of programs in the clinic, which span various diseases, modalities and stages of development. To ensure patient safety and reduce the burden on the healthcare system at a time of critical need, Vertex has temporarily paused or delayed enrollment in certain studies.
Alpha-1 Antitrypsin (AAT) Deficiency:
Vertex has temporarily paused screening and enrollment in the Phase 2 study of VX-814; however, the study remains active and Vertex continues to initiate new clinical trial sites to enable future patient enrollment.
Beta Thalassemia and Sickle Cell Disease
Vertex and its partner CRISPR Therapeutics remain on track to provide additional data from the two ongoing Phase 1/2 studies of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with transfusion-dependent beta thalassemia and in patients with severe sickle cell disease in 2020. New data expected in 2020 include initial data from additional patients dosed in each of the Phase 1/2 studies and longer duration follow-up data for the first patients dosed in each study. Screening, enrollment and mobilization in these studies is ongoing; however, no additional patients are scheduled to initiate conditioning or dosing at this time.
Focal Segmental Glomerulosclerosis (FSGS):
Vertex recently initiated a Phase 2 proof-of-concept study of VX-147 in people with FSGS.
The 13-week open-label Phase 2 study is designed to evaluate the reduction in proteinuria in people with FSGS after treatment with VX-147.
Type 1 Diabetes:
Vertex continues to advance its cell therapy program for the treatment of type 1 diabetes and expects to initiate clinical development in patients in late 2020 or early 2021.
Investments in External Innovation
Expanding Capabilities in Genetic Therapies: In April, Vertex entered into a collaboration with Affinia Therapeutics to gain access to a novel library of AAV capsids that will bolster Vertex’s ongoing research and development work in genetic therapies. The goal of the collaboration will be to develop genetic therapies for people affected by Duchenne muscular dystrophy (DMD), myotonic dystrophy 1 (DM1) and CF.
mRNA Therapies for CF: Based on promising preclinical data generated to date, Vertex and Moderna recently extended their research collaboration aimed at the discovery and development of mRNA therapeutics for the treatment of CF.