Vertex Provides Pipeline and Business Updates in Advance of Upcoming Investor Meetings
“2023 was marked by continued strong performance in the cystic fibrosis business and acceleration of our development stage pipeline, propelling the company into 2024 with tremendous momentum," said
Disease Areas with Approved Medicines
Next-in-class triple combination: Completed three pivotal studies evaluating the next-in-class, triple combination CFTR modulator therapy vanzacaftor/tezacaftor/deutivacaftor compared to TRIKAFTA® in patients with CF ages 6 years of age and older (ages 12+ in SKYLINE 102 and 103 studies; ages 6-11 in the RIDGELINE study).
Vertexexpects to share the results of all three studies in early 2024.
VX-522: Completed dosing in the single ascending dose (SAD) portion of the Phase 1/2 study of VX-522 in patients with CF and initiated the multiple ascending dose (MAD) portion of the study. VX-522 is a CFTR mRNA therapeutic that
Vertexis developing in collaboration with Moderna for the >5,000 patients with CF who do not make any CFTR protein and cannot benefit from CFTR modulators.
Vertexrevised its estimates for the number of patients living with cystic fibrosis from ~88,000 to ~92,000 in the U.S., Europe, Australia, and Canada.
Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) – CASGEVY
Received regulatory approvals for CASGEVY in the fourth quarter of 2023 in the
U.S.for sickle cell disease, and in Great Britainand Bahrainfor the treatment of both SCD and TDT; also received positive CHMP opinion for CASGEVY for both SCD and TDT from the European Medicines Agency(EMA).
The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of
March 30, 2024, for CASGEVY in TDT. Additional regulatory submissions for CASGEVY are currently under review in Switzerlandand the Kingdom of Saudi Arabia, with submission in Canadaplanned for the first half of 2024.
- Completed enrollment in two global Phase 3 studies of CASGEVY in patients 5 to 11 years of age with SCD or TDT.
Signed an agreement with Synergie Medication Collective, a medication contracting organization founded by a group of
Blue Cross and Blue Shieldaffiliated companies covering approximately 100 million people, to provide access to CASGEVY.
Activated 9 authorized treatment centers (ATCs) in the
U.S.and 3 in Europe, with an ultimate goal of activating approximately 50 ATCs in the U.S.and 25 in Europe.
Vertexrevised its estimates for the number of patients living with severe sickle cell disease, from ~25,000 patients to ~30,000 patients in the U.S.and Europe, with additional patients in Bahrainand the Kingdom of Saudi Arabia. Vertex’s updated estimate for patients with transfusion-dependent beta thalassemia is revised from ~7,000 to ~5,000 patients in the U.S.and Europe, with additional patients in Bahrainand the Kingdom of Saudi Arabia.
Pipeline Disease Areas
VX-548: Completed three Phase 3 studies of VX-548 for the treatment of moderate to severe acute pain, including two randomized placebo-controlled studies: one following abdominoplasty and one following bunionectomy surgery, as well as a third single-arm safety and effectiveness study, which enrolled both surgical and non-surgical patients with moderate to severe acute pain.
Vertexexpects to share the results of all three studies in early 2024.
- Completed a Phase 1 study of an oral formulation of VX-993, a next-generation NaV1.8 inhibitor, and anticipates initiating a Phase 2 study for the treatment of moderate to severe acute pain in 2024.
Vertexalso anticipates initiating a Phase 1 study of an intravenous formulation of VX-993 for the treatment of moderate to severe acute pain in 2024.
Peripheral Neuropathic Pain (PNP)
December 2023release of positive Phase 2 results with VX-548 in diabetic peripheral neuropathy, which represents ~20% of peripheral neuropathic pain patients, Vertexplans to meet with regulators in the first quarter of 2024 and anticipates advancing VX-548 into pivotal development.
- Initiated a Phase 2 study of VX-548 in lumbosacral radiculopathy, another type of peripheral neuropathic pain and the largest patient segment (over 40%) within the PNP category.
- Following the
Vertexalso anticipates initiating a Phase 2 study with an oral formulation of VX-993 for the treatment of PNP in 2024.
APOL1-Mediated Kidney Disease (AMKD) – Inaxaplin (VX-147)
- Completed enrollment in the Phase 2B dose-ranging portion of the study of inaxaplin for the treatment of patients with AMKD.
Vertexexpects to select the inaxaplin dose for the Phase 3 portion of the Phase 2/3 pivotal trial and begin Phase 3 in the first quarter of 2024.
Type 1 Diabetes (T1D)
VX-880: Completed enrollment in Parts A, B, and C of the Phase 1/2 study of VX-880, an allogeneic, stem cell-derived, fully differentiated, insulin-producing islet cell therapy, used in conjunction with standard immunosuppression, in patients with T1D and impaired awareness of hypoglycemia and recurrent hypoglycemic events.
- As of the last data cut, all 14 patients dosed with VX-880 demonstrated islet cell engraftment and production of endogenous insulin.
All Part A and B patients, except for one patient who withdrew consent from the study, demonstrated glycemic control to target
ADArecommended levels with HbA1C <7% and no longer required exogenous insulin. Part C patients have trajectories similar to Part A and B patients.
- The safety profile of VX-880 to date is consistent with immunosuppressives, the perioperative period and past medical history.
Two patient deaths, both unrelated to VX-880, have occurred.
Vertexhas placed the study on a protocol-specified pause, pending review of the totality of the data by the independent data monitoring committee and global regulators.
VX-264: The clinical trial for VX-264, which encapsulates the same VX-880 cells in a device that is designed to eliminate the need for immunosuppressants, is a multi-part, Phase 1/2 study.
- Part A has initiated, enrolled and dosed multiple patients.
The study remains ongoing in multiple centers and countries as
Vertexprepares for Part B initiation.
Myotonic Dystrophy Type 1 (DM1) – VX-670
Received clearances from
Health Canadaand Medicines and Healthcare Products Regulatory Agency(MHRA – UK) for Clinical Trial Applications (CTA) for VX-670 for patients with DM1. Vertexinitiated the Phase 1/2 clinical trial in patients with DM1 in Canadaand will initiate the study in the UKnear-term.
Vertexalso submitted an Investigational New Drug (IND) application to the FDA for VX-670. The FDA requested additional information, which resulted in a clinical hold. Vertexis working to address FDA comments and initiate the study in the U.S.
VX-670 is an oligonucleotide that targets the underlying cause of DM1, linked to a cyclic peptide that promotes effective delivery into cells. DM1 is the most prevalent muscular dystrophy in adults with ~110,000 people living with the disease in the
Autosomal Dominant Polycystic Kidney Disease (ADPKD) – VX-407
- Completed IND-enabling studies of VX-407 for the treatment of ADPKD and anticipates beginning a Phase 1 clinical trial in healthy volunteers this year.
- VX-407 is a first-in-class small molecule corrector that targets the underlying cause of ADPKD in a subset of patients with responsive PKD1 mutations, estimated at ~25,000 (or ~10%) of the overall ~250,000 ADPKD patient population. ADPKD is the most commonly inherited kidney disease.
J.P. Morgan Healthcare Conference Presentation and Webcast
A live webcast of management's remarks will be available through the
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