Biotech Vertex Pharmaceuticals VRTX is getting closer on an approval for its pain pill that could serve as a replacement for opioids since trials showed greater improvements in pain relief.
The company said on March 31 that it received positive results from two Phase 2 proof-of-concept studies that investigated treating patients with acute pain following abdominoplasty surgery or bunionectomy surgery with their drug known as VX-548.
The pain pill, VX-548, was generally “well tolerated in both studies,” the company said in a statement.
“Most adverse events (AEs) were mild to moderate and there were no serious adverse events (SAEs) related to VX-548,” Vertex said. “Fewer patients discontinued treatment in the mid- and high-dose VX-548 arms than in the placebo group or HB/APAP reference arm.”
The next step is for Vertex to advance the pain pill “into pivotal development in the second half of 2022, following discussions with regulators” and enter into Phase 3 of trials, the company said.
“The remarkable consistency in the safety, tolerability and efficacy results in these two studies demonstrate the potential of VX-548 to be a first-in-class non-opioid treatment for acute pain,” said Carmen Bozic, Vertex’s chief medical officer, in a statement.
“We are working with urgency to advance the program into Phase 3 with the goal of bringing forward the first novel pain treatment in decades to address the unmet needs of patients suffering from acute pain.”
Vertex Pipeline Has Several Other Promising Drugs
The company focuses on treatment of cystic fibrosis with its key drugs, Kalydeco, Orkambi, Symdeko and Trikafta/Kaftrio.
Vertex has increased its pipeline through gene-editing therapies such as CTX001 for beta-thalassemia and sickle-cell disease, small-molecule inhibitors targeting acute and chronic pain using nonopioid treatments, and small-molecule inhibitors of APOL1-mediated kidney diseases.
Vertex is also working on cell therapies to deliver a potential functional cure for type 1 diabetes.
Scroll to Continue
Is M&A on The Biotech Horizon?
Biotech stocks have slumped over the past 12 months, but their cheaper valuations could emerge as entry points for investors, experts say.
Many of them could be targets for acquisitions or merged with a larger biotech.
Investors could add AbbVie ABBV, Amgen AMGN, Incyte INCY Moderna MRNA and Vertex Pharmaceuticals to a portfolio, which CFRA currently has buy/strong buy ratings, Sam Stovall, chief investment strategist at CFRA, a New York-based investment research company, told TheStreet.
The price target for Vertex is $284, wrote CFRA equity analyst Stewart Glickman in a March 26 research report.
The pharmaceutical company is estimated to increase its revenue by 12% year-over-year to $8.5 billion after growth of 22% in 2021, he said.
The company’s cystic fibrosis drug, Trikafta, that launched in late 2019, produced 82% of VRTX’s product sales during the fourth quarter of 2021, and should continue to be “a key driver of sales growth as VRTX continues to expand commercial access to the drug and converts patients on old therapies to it,” Glickman wrote.
“Near-term catalysts include a plan to file for regulatory approval of CTX001, a sickle cell disease treatment (likely by year end); shifting to a pivotal development in the first quarter for VX-147, a treatment for APOL1-mediated kidney disease; and to file for an investigational new drug application for type 1 diabetes during 2022,” he added.
Jefferies analyst Michael Yee said Vertex remains “the cleanest story in large-cap biotech” and has a buy rating with a price target of $275.
Vertex has also shown positive news for its drug on treating kidney disease, VX-147.
The company said on March 22 it would open enrollment in March for a new pivotal study to study the efficacy of the drug.