QURE Stock Rockets After FDA Approves Its Most Expensive Drug Ever
The Food and Drug Administration just approved its most expensive drug ever — a hemophilia B gene therapy from Carlisle Companies (CSL) and UniQure (QURE) — and QURE stock popped Wednesday.
Meanwhile, BioMarin (BMRN) shares broke out after the FDA said it wouldn’t hold an advisory committee meeting to discuss its hemophilia A gene therapy. These advisories make nonbinding recommendations to the FDA after discussing the benefits and risks of experimental drugs. BioMarin’s Roctavian is already approved in Europe.
On today’s stock market, QURE stock jumped 14.6% to 26.36. That extended the stock’s run from a 7.1% surge on Tuesday. Shares topped a buy point at 25.91 out of a consolidation, according to MarketSmith.com.
BioMarin stock jumped 7.3% to 97.79. That pushed BioMarin stock to break out of a double-bottom base with an entry at 92.86.
QURE Stock: Price Is Above Expectations
UniQure’s infusion is approved for hemophilia B patients currently on a prevention regimen called Factor IX therapy, or who have a history of potentially deadly hemorrhages or serious bleeding episodes. At a one-time cost of $3.5 million, Carlisle and UniQure hope the drug is a cure.
Analysts say the approval is a best-case scenario for UniQure, the company that initially developed Hemgenix before partnering with Carlisle in later stages. The label does not limit the therapy to any specific pool of patients or include a “black box” warning.
“Given the uncertainty around hemophilia gene therapy approvals and investor caution, we think this is a major positive for UniQure and a key de-risking for the story,” UBS analyst Eliana Merle said in a report Wednesday.
The $3.5 million price tag is above a recommendation by the Institute for Clinical and Economic Review. The nonprofit said most hemophilia gene therapies would be cost effective at $2.5 million. The report examined prices between $2 million to $3 million based on executives’ comments.
SVB Securities analyst Joseph Schwartz said the higher-than-expected price likely accounts for the small hemophilia B patient population. Still, he views the approval as a positive for patients with frequent bleeding episodes and the need for “burdensome prophylaxis therapy.”
Patients will be monitored for three hours following the Hemgenix infusion. Then, they will need to undergo weekly monitoring for three months to watch for potential liver complications. Schwartz notes prevention treatment with Factor IX is typically given weekly.
“We believe these monitoring requirements make sense and will be viewed as a worthwhile sacrifice for the likely liberalization of factor therapy, bleeds and joint damage for a long time,” he said in a report.
He has an outstanding rating on QURE stock.
UniQure’s Other Gene Therapy Effects
Under the terms of the agreement, Carlisle will be responsible for commercializing Hemgenix. In return, UniQure will receive up to $1.5 billion in additional milestone payments and a royalty on sales in the midteens to low-20% range. UniQure also has manufacturing rights.
“We see (the) approval as a key de-risking event for UniQure’s future cash flow potential,” Schwartz said. “We also think this bodes well for the approval potential for BioMarin’s Roctavian in hemophilia A.”
The approval also likely lends credence to UniQure’s efforts in other gene therapy areas. Investors are probably more cautious about the company’s work in Huntington’s disease. Three out of 14 patients in UniQure’s study experienced serious side effects.
“We note the Phase 1 and Phase 2 update is still guided for in the second quarter of 2023 and could be an informative catalyst for the program with the first data on functional (goals),” said Merle, the UBS analyst.
She has bought rating on QURE stock and raised her price target to 42 from 40.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
YOU MAY ALSO LIKE:
Medtronic Hits Two-Year Low On Quarterly Lag — But Analysts Look For The Bright Spots
See Why Gilead’s Cancer Business Is Driving The Stock To A Fever Pitch
Get Timely Buy & Sell Alerts With IBD Leaderboard
Stocks To Buy And Watch: Top IPOs, Big And Small Caps, Growth Stocks
Find The Best Long-Term Investments With IBD Long-Term Leaders