Dive Brief:
- In its first quarter 2018 earnings announced on Thursday, Vertex Pharmaceuticals Inc. said cystic fibrosis (CF) product revenues rose to $638 million, a 33% increase year-on-year, driven by the launch of Symdeko and sales of Kalydeco and Orkambi.
- Net income fell by 16% to $210 million, in parallel with increases in combined R&D and SG&A expenses of 14%. The company predicts CF product revenue guidance of $2.65 billion to $2.8 billion for 2018.
- Vertex also announced the beginning of Phase 3 studies for the VX-445 triple combination CF treatment.
Dive Insight:
Vertex's aim is to treat the vast majority of CF patients with drugs from its portfolio. Symdeko, often referred to as tez/iva, combines tezacaftor and Kalydeco (ivacaftor) and was approved by the Food and Drug Administration in February this year. This made it Vertex's third approved CF therapy, alongside Kalydeco and Orkambi(ivacaftor lumacaftor).
One of Vertex's fastest moving areas is that of triple combination therapies that include a "next-generation corrector."
The two triplets Vertex is testing in Phase 3 trials use Symdeko as a backbone. The combination of Symdeko and VX-445 has just moved into a pair of late-stage studies in patients with CF who have one copy of the F508del mutation and one minimal function mutation, and in people with CF who have two copies of the F508del mutation — the most common form of the disease. A Phase 3 trial of Symdeko and VX659 in people with CF who have one F508del mutation and one minimal function mutation is currently enrolling, and the first patients have received their initial dose.
"Initiating Phase 3 studies with both VX-659 and VX-445 gives us the opportunity to generate data for two different triple-combination regimens and pick the best regimen to bring to patients as quickly as possible," Vertex's Chief Medical Officer Reshma Kewalramani said on the company's earnings call.
Vertex also reported results from its once-daily potentiator, VX-561, which it has assessed as part of a triple combination with VX-659 or VX-445 and tezacaftor in people with CF and one minimal function mutation. While the combinations were well-tolerated, the Food and Drug Administration has requested additional studies of VX-561, including as a monotherapy, which has delayed the start of late-stage evaluation.
CEO Jeffrey Leiden put the FDA's caution down to it treating VX-561 as a new chemical entity, despite it being a deuterated version of Kalydeco.
"Because our goal has always been to get the best regimen to patients as quickly as possible, we decided to move forward with VX-445 with Kalydeco because that's the quickest route and we didn't want to take a delay," Leiden said. "We do plan to take it forward into a once a day regimen. We're still in discussions with the FDA ... we'll work out a bridging strategy that allows us to bring VX-561 along with either VX-659 or VX-445 whichever one we choose to patients."
According to Leiden, there are a number of other potential next-generation correctors in the discovery and preclinical pipeline that could have greater efficacy and potency than VX-659 or VX-445, and could be formulated for once-daily dosing.
"If we feel we have a molecule that's significantly better than the other two, we'll take it forward into Phase 1, and certainly into early Phase 2. That's easy and quick to do. If we don't, we won't," he said.
A European approval decision on Symdeko is expected in the second half of 2018, and a Phase 3 study is underway in children aged between 6 and 11 years.