The Food and Drug Administration has approved the most expensive drug ever – hemophilia B gene therapy Companies of Carlisle (CSL) and UniQure (QURE) – and QURE shares rose on Wednesday.
In the meantime, BioMarin ( BMRN ) shares fell after the FDA said it would not hold an advisory committee meeting to discuss hemophilia A gene therapy. These advisors make nonbinding recommendations to the FDA after discussing the benefits and risks of experimental drugs. BioMarin’s Roctavian is already approved in Europe.
QURE shares rose 14.6% to 26.36 on the market today. That extended the stock’s rally from Monday’s 7.1% gain. According to MarketSmith.com, shares crossed a buy point at 25.91 as a result of the consolidation.
BioMar shares rose 7.3% to 97.79. That pushed BioMar stock out of a double-bottom base with an entry at 92.86.
QURE Shareholder: The price is above expectations
UniQure infusion is currently approved for hemophilia B patients who are on a prophylaxis regimen called Factor IX therapy or who have had potentially fatal bleeding or serious bleeding episodes. With a one-time $3.5 million investment, Carlyle and UniQure hope the drug will be a cure.
Analysts say approval is the best-case scenario for UniQure, which initially developed Hemgenix before partnering with Carlisle in later stages. The label does not limit the therapy to any particular patient pool or contain a “black box” warning.
“Given the uncertainty surrounding the approval of the hemophilia gene therapy and investor caution, we think this is a big upside for UniQure and a key de-risk to the story,” UBS analyst Eliana Merle said in a report on Wednesday.
The $3.5 million price tag is higher than the Institute for Clinical and Economic Review’s recommendation. The nonprofit said most hemophilia gene therapies would cost $2.5 million. The report examined prices ranging from $2 million to $3 million based on comments from executives.
SVB Securities analyst Joseph Schwartz said the higher-than-expected price likely accounts for the smaller hemophilia B patient population. Still, he sees the approval as positive for patients who have frequent bleeding episodes and need “severe prophylactic therapy.”
After the Hemgenix infusion, patients will be observed for three hours. Then, they should undergo weekly monitoring for three months to watch for potential liver complications. Schwartz notes that preventive treatment with Factor IX is usually given weekly.
“We believe that these monitoring requirements are reasonable and will be viewed as a worthwhile sacrifice for possible liberalization from long-term factor therapy, bleeding, and joint injuries,” he said.
It has outperformed QURE stocks.
UniQure’s Other Gene Therapy Efforts
Under the terms of the agreement, Carlyle will be responsible for the commercialization of Hemgenix. In return, UniQure will receive up to $1.5 billion in additional milestone payments and royalties on sales at mid-terms of less than 20%. UniQure also owns the manufacturing rights.
“We view the approval as a key de-risking event for UniQure’s future cash flow potential,” Schwartz said. “We also think this bodes well for BioMar’s approval potential for Roctavian in hemophilia A.”
The approval likely also lends credence to UniQure’s efforts in other areas of gene therapy. Investors are probably more cautious about the company’s performance in Huntington’s disease. In UniQure’s study, three out of 14 patients experienced serious side effects.
“We note that the Phase 1 and Phase 2 update is still on track for the second quarter of 2023 and could be an informative catalyst for the program with the first data on functional (goals),” UBS analyst Merle said.
He has a buy rating on QURE stock and raised his price target to 42 from 40.
Follow Allison Gatlin on Twitter @IBD_AGatlin.
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