The Food and Drug Administration just approved its most expensive drug ever: a gene therapy for hemophilia B Carlisle Companies (CSL) and UniQure (QURE) – and QURE shares exploded on Wednesday.
Meanwhile, BioMarin (BMRN) shares erupted after the FDA said it would not hold an advisory committee meeting to discuss its hemophilia A gene therapy. These advisors make non-binding recommendations to the FDA after discussing the benefits and risks of the experimental drugs. BioMarin’s Roctavian is already approved in Europe.
In today’s stock market, QURE stock jumped 14.6% to 26.36. That extended the stock’s run by a 7.1% gain on Tuesday. The shares cleared a buy point at 25.91 from a consolidation, according to MarketSmith.com.
BioMarin shares jumped 7.3% to 97.79. This prompted BioMarin stock to break out of a double bottom basis with an entry at 92.86.
QURE Action: Price is higher than expected
The UniQure infusion is approved for patients with haemophilia B who are currently on a preventative regimen called factor IX therapy or who have a history of life-threatening bleeding or major 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 therapy to a specific patient pool or include a “black box” warning.
“Given the uncertainty surrounding gene therapy approvals for haemophilia and investor caution, we believe this is a major win for UniQure and a key risk-reducer to the story,” said UBS analyst Eliana Merle. in a report Wednesday.
The $3.5 million price tag is above an Institute for Clinical and Economic Review recommendation. The nonprofit said most hemophilia gene therapies would be affordable at $2.5 million. The report looked at prices between $2 million and $3 million based on executive comments.
SVB Securities analyst Joseph Schwartz said the higher-than-expected price likely explains the small haemophilia B patient population. However, he views the approval as positive for patients with frequent bleeding episodes and the need for a ” strenuous prophylactic therapy”.
Patients will be monitored for three hours after the Hemgenix infusion. Then, they will need to undergo weekly monitoring for three months to check for potential liver complications. Schwartz notes that preventive treatment with factor IX is usually given weekly.
“We believe these monitoring requirements make sense and will be seen as a worthwhile sacrifice for likely liberalization from factor therapy, hemorrhage and joint damage for a long time,” he said in a report.
It has a rating of outperforming the QURE stock.
UniQure’s other gene therapy efforts
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 mid-teen to low 20% range. UniQure also has the manufacturing rights.
“We see (the) approval as a key de-risk event to UniQure’s future cash flow potential,” said Schwartz. “We also think this bodes well for the approval potential of BioMarin’s Roctavian in hemophilia A.”
The approval also likely lends credence to UniQure’s efforts in other areas of gene therapy. Investors are likely more cautious about the company’s work in Huntington’s disease. Three out of 14 patients in the UniQure study experienced serious side effects.
“We note that the Phase 1 and Phase 2 update is still planned for Q2 2023 and could be an informational catalyst for the program with early data on functional (targets),” said Merle, an analyst at UBS.
It has a buy rating on QURE stock and has raised its price target to 42 from 40.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
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