- Vertex Pharmaceuticals said it plans to file for U.S. and EU approval of an experimental cystic fibrosis combination therapy in the third quarter after announcing positive results for two Phase 3 studies Tuesday night.
- Data from the studies suggest the treatment, a pairing of Vertex's Kalydeco (ivacaftor) with a second-generation corrector known as tezacaftor, will be a strong addition to the Boston-based biotech's existing cystic fibrosis (CF) portfolio. Shares in Vertex surged more than 20% Wednesday morning.
- The enthusiastic market reaction caps a quarter of steady gains as the biotech has shaken off worries over slower-than-expected growth from its newer CF drug Orkambi (lumacaftor/ivacaftor).
If approved later this year, the teza/iva combo should help Vertex strengthen its market position in CF ahead of rivals, while helping boost continued pipeline development of triple combination therapies.
The larger of the two studies, dubbed EVOLVE, tested the combo in CF patients aged 12 and older who have two copies of a mutation called F508del — a subgroup representing about 30% of the overall CF patient population.
After 24 weeks, treated patients saw an average improvement in lung function of 4 percentage points from baseline, compared to those on placebo. That level of improvement suggests teza/iva could be more effective than Orkambi in F508del patients. It also checked in a tick higher than market expectations, said analysts from Jefferies in a note.
The EXPAND study, meanwhile, also met its primary endpoint in patients with one copy of the F508del mutation and another mutation that results in residual function of a protein encoded by the CFTR gene. In that 8-week crossover study, patients treated with the combo experienced a mean absolute improvement of 6.8 percentage points compared to placebo.
Teza/iva was well tolerated and overall adverse and serious adverse event rates in the EVOLVE trial were higher for patients on placebo than those on teza/iva. Importantly, respiratory adverse events were seen in only 13% of teza/iva patients compared to 16% of those on placebo. As analysts from Jefferies note, this appears lower than that seen with Orkambi – another signal teza/iva could deliver a better efficacy and safety profile for F508del patients.
Such success from both studies also wipes out any lingering concerns from a clinical setback in another study testing the combo that read out last year. That trial looked at teza/iva in patients with one F508del mutation and another mutation that results in minimal function of a protein encoded by the CFTR gene.
Earlier this year, Vertex staked out cautious sales guidance for Orkambi and Kalydeco, forecasting conservatively after being forced to cut its sales estimates last year. With positive data in hand and a date with the FDA set for later this year, investors will likely be feeling more bullish about Vertex now than three months ago.