Qure Therapeutics (NASDAQ: QURE) is a biotechnology company that focuses on developing gene therapies for rare and life-threatening diseases.
Qure’s lead product candidate, AMT-061, is a potential cure for hemophilia B, a bleeding disorder that affects about 20,000 people in the US and 40,000 people worldwide. AMT-061 has received breakthrough therapy designation from the FDA and is currently in phase 3 clinical trials.
In this article, we will provide you with an ultimate guide to Qure stock, covering its history, fundamentals, growth prospects, risks, and valuation.
We will also give you some tips on how to invest in Qure stock wisely and profitably. By the end of this article, you will have a better understanding of Qure’s business, its competitive advantages, and its potential as a long-term investment.
Qure’s History and Background
Qure was founded in 1998 by a group of scientists from the University of Amsterdam, who had a vision to use gene therapy to treat incurable diseases.
Gene therapy is a technique that involves introducing a healthy copy of a gene into a patient’s cells to correct a genetic defect or enhance a biological function. Qure’s gene therapy platform is based on adeno-associated virus (AAV) vectors, which are harmless viruses that can deliver genes to specific tissues in the body.
Qure’s initial focus was on developing gene therapies for metabolic disorders, such as phenylketonuria (PKU) and glycogen storage disease type Ia (GSD-Ia). However, in 2014, Qure shifted its strategy to target blood disorders, such as hemophilia and sickle cell disease, which have a higher unmet medical need and a larger market opportunity.
Qure also partnered with Bristol-Myers Squibb (BMS) to develop gene therapies for cardiovascular diseases, such as congestive heart failure and coronary artery disease.
Qure went public in 2014, raising $82 million in its initial public offering (IPO) at $15 per share. Since then, Qure’s stock price has fluctuated significantly, reaching a high of $93.5 in 2018 and a low of $9.7 in 2020. As of December 28, 2023, Qure’s stock price is $37.2, giving it a market capitalization of $1.8 billion.
Qure’s Fundamentals and Financials
Qure’s main source of revenue is from its collaboration agreements with BMS and CSL Behring, a global leader in plasma-derived therapies. Under these agreements,
Qure receives upfront payments, milestone payments, and royalties on future sales of its gene therapy products. In 2020, Qure reported total revenues of $119.6 million, up 59% from $75.1 million in 2019. Qure’s net loss, however, widened to $173.4 million, or $3.64 per share, from $121.8 million, or $2.66 per share, in 2019.
Qure’s operating expenses consist mainly of research and development (R&D) and general and administrative (G&A) costs. In 2020, Qure spent $263.9 million on R&D, up 41% from $186.9 million in 2019. Qure’s R&D expenses reflect its investment in advancing its pipeline of gene therapy candidates, as well as expanding its manufacturing capabilities and intellectual property portfolio.
Qure’s G&A expenses, on the other hand, increased 29% to $29.1 million in 2020, from $22.6 million in 2019. Qure’s G&A expenses include personnel costs, legal fees, and other administrative expenses.
Qure’s balance sheet shows that it has a strong liquidity position, with $462.4 million in cash and cash equivalents as of December 31, 2020. Qure’s cash burn rate, or the amount of cash it spends to fund its operations, was $144.8 million in 2020, down from $111.8 million in 2019. Qure expects its cash burn rate to increase in 2021, as it plans to invest more in its clinical trials, manufacturing, and commercialization activities.
Qure’s Growth Prospects and Opportunities
Qure’s growth potential lies in its pipeline of gene therapy candidates, which target diseases with high unmet medical need and large market opportunity.
Qure’s most advanced product candidate, AMT-061, is a gene therapy for hemophilia B, a bleeding disorder caused by a deficiency of factor IX (FIX), a protein that helps blood clot. AMT-061 consists of an AAV5 vector that delivers a functional copy of the FIX gene to the liver cells of patients, where it produces a modified form of FIX that has enhanced clotting activity.
AMT-061 has demonstrated promising results in its phase 3 clinical trial, called HOPE-B, which enrolled 54 patients with severe or moderately severe hemophilia B.
The trial showed that AMT-061 increased the FIX activity levels of patients to a mean of 41.5% of normal at 26 weeks after a single infusion, compared to a baseline of less than 2%. AMT-061 also reduced the annualized bleeding rate of patients by 96%, from a median of 7.9 to 0.3, and eliminated the need for prophylactic FIX replacement therapy in 92% of patients.
Qure expects to submit a biologics license application (BLA) to the FDA for AMT-061 in the first half of 2021, and to receive a regulatory decision by the end of 2021. If approved, AMT-061 would be the first gene therapy for hemophilia B in the US, and the second gene therapy for hemophilia in the world, after BioMarin’s Roctavian, which was approved in Europe in 2020 for hemophilia A.
Qure estimates that the global market for hemophilia B gene therapy is about $2 billion, and that AMT-061 could capture a significant share of it, given its superior efficacy, safety, and convenience.
Qure’s other product candidates include AMT-130, a gene therapy for Huntington’s disease, a fatal neurodegenerative disorder that affects about 30,000 people in the US and 70,000 people worldwide; AMT-150, a gene therapy for spinocerebellar ataxia type 3 (SCA3), a rare and progressive movement disorder that affects about 10,000 people in the US and 50,000 people worldwide; and AMT-190, a gene therapy for Fabry disease, a rare and life-threatening metabolic disorder that affects about 5,000 people in the US and 10,000 people worldwide.
All of these product candidates are in early stages of clinical development, and have the potential to address unmet medical needs and large market opportunities.
Qure’s Risks and Challenges
Qure’s success depends largely on the approval and commercialization of AMT-061, which faces several risks and challenges. First, AMT-061 is not guaranteed to receive regulatory approval, as the FDA may require additional data, impose stricter safety requirements, or delay its decision. Second, AMT-061 may face competition from other gene therapies for hemophilia B, such as Pfizer’s fidanacogene elaparvovec and Sangamo’s SB-FIX, which are also in phase 3 clinical trials .
Third, AMT-061 may face pricing and reimbursement challenges, as gene therapies are typically very expensive and may not be covered by insurance companies or government agencies. Fourth, AMT-061 may face manufacturing and supply chain challenges, as gene therapies require complex and specialized production and distribution processes.
Qure’s other product candidates also face similar risks and challenges, as well as uncertainties regarding their efficacy, safety, and durability.
Qure’s gene therapy platform is based on AAV vectors, which may have limitations, such as immune responses, toxicity, or loss of expression. Qure’s intellectual property rights may also be challenged, invalidated, or infringed by competitors or third parties. Qure’s financial performance may also be affected by fluctuations in exchange rates, as it operates in multiple countries and currencies.
Qure’s Valuation and Outlook
Qure’s valuation is based on its future cash flows, which depend on its ability to develop, approve, and commercialize its gene therapy products. Qure’s stock price reflects the market’s expectations and sentiments regarding its growth potential and risk profile. Qure’s stock price may also be influenced by external factors, such as macroeconomic conditions, industry trends, and market movements.
Qure’s stock price has increased by 35% in the past year, outperforming the Nasdaq Biotechnology Index, which has gained 25% in the same period. Qure’s stock price has also recovered from its low of $9.7 in March 2020, when the COVID-19 pandemic caused a market crash.
Qure’s stock price may continue to rise if it achieves positive results in its clinical trials, receives regulatory approval for AMT-061, and successfully launches its gene therapy products in the and successfully launches its gene therapy products in the global market, creating value for patients, providers, payers, and shareholders. Qure’s vision is to become a leader in cell and gene therapy, delivering breakthrough innovative treatments for rare and life-threatening diseases. Qure’s mission is to empower patients with new hope and new possibilities, improving their quality of life and transforming their future.
