Latest news with #Casgevy
Yahoo
6 days ago
- Business
- Yahoo
As Interest Grows for New Agents to Treat Thalassemia, Hematologists Begin Early Identification of Ideal Patients for Gene Therapies Vs. New Therapeutic Options
Spherix Global Insights' new patient audit on transfusion-dependent thalassemia reveals strong physician interest in emerging treatments like mitapivat and etavopivat, and provides insight into ideal candidates for curative gene therapies. EXTON, PA, June 04, 2025 (GLOBE NEWSWIRE) -- Despite the promise of new curative strategies for beta thalassemia, such as Casgevy (Vertex) and Zynteglo (bluebird bio), most transfusion-dependent thalassemia patients remain tethered to long-term transfusion support. Transfusions, while providing symptom relief, in turn create logistical barriers and their own clinical challenges. New data from Spherix Global Insights' independent chart audit, Patient Chart Dynamix™: Transfusion Dependent Thalassemia 2025 (US), is based on insight from 81 real-world patient charts submitted by 49 US-based hematologists. These data paint a complex picture of therapeutic inertia, growing anticipation for emerging treatments, and deep-seated frustration with limitations of current treatment options. Most transfusion-dependent beta thalassemia patients follow some regular transfusion schedule. That said, many receive them at relatively extended intervals: often once a month or even less frequently, though a small subset still require more frequent transfusions. This variation in treatment patterns, along with persistent symptoms and unmet needs highlighted in the chart audit, points to a strong opportunity for new therapies to improve care for a broad range of patients. Among surveyed hematologists, mitapivat (Agios) currently holds a slight edge in name recognition over etavopivat (Novo Nordisk). Interestingly, despite this difference in familiarity, physicians identified slightly more patients as potential candidates for etavopivat, hinting at a broader perceived clinical fit once awareness and understanding of the agent catch up. Chart audit data allows users to probe further into specific patient types which physicians perceive as better fits for either agent, as well as those who they do not perceive as candidates. This growing interest in emerging oral therapies reflects a broader desire among hematologists for more accessible, lower-burden treatment options. While most patients are theoretically eligible for curative approaches like gene therapy, the market still favors lower-barrier alternatives. These alternatives include oral therapies that do not require referral to specialized centers or extensive pre-conditioning, and brands with which they are more familiar given other indications such as Reblozyl (Bristol Myers Squibb). Disease-modifying therapies that are easier to prescribe and integrate into routine practice have the promise to significantly improve the lives of patients who would not qualify or want gene therapy. Access barriers compound the issue, with over half of physicians voicing frustration over payer restrictions that make it difficult to initiate optimal care. These challenges highlight a critical need – not just for clinical innovation, but also for strong manufacturer support in navigating reimbursement and expanding access. As the beta thalassemia landscape continues to evolve, hematologists are increasingly looking toward emerging therapies that are easier to prescribe, carry fewer logistical burdens than gene therapy, and offer clear reductions in transfusion dependency. Products like mitapivat and etavopivat are well-positioned to address this gap, assuming familiarity, confidence, and access grow in tandem – but the goal to cure patients of hemoglobinopathies still carries heavy weight, with desire to treat with gene therapy consistently growing. Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the 'why' behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists' attitudinal & demographic data to highlight differences between stated and actual treatment patterns. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix's unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit or connect through LinkedIn. For more details on Spherix's primary market research reports and interactive dashboard offerings, visit or register here: NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight's analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. CONTACT: Sarah Hendry, Hematology Franchise Head Spherix Global Insights 4848794284 in to access your portfolio
Yahoo
02-06-2025
- Business
- Yahoo
The 2 Smartest Beaten-Down Biotech Stocks to Buy on the Dip
CRISPR Therapeutics has the potential to generate significant breakthroughs in gene editing. Regeneron's biggest growth driver is still performing well, and it is developing newer medicines. 10 stocks we like better than CRISPR Therapeutics › Even though broader equities have been highly volatile this year, it's still a good idea to invest in stocks for a straightforward reason. Holding shares of top companies for five years and beyond will usually allow anyone to earn superior returns. That's not a secret, but it's easy to forget when dealing with an uncertain near term. Even turbulent times don't change this fact. For investors still seeking companiies with attractive long-term prospects, let's consider two in the biotech industry: CRISPR Therapeutics (NASDAQ: CRSP) and Regeneron Pharmaceuticals (NASDAQ: REGN). These drugmakers have faced issues recently, and their shares have declined significantly over the trailing-12-month period. However, there may be considerable upside here for patient investors. Skepticism about CRISPR Therapeutics' prospects may be warranted. Though it has a product on the market, a gene editing therapy called Casgevy it developed with Vertex Pharmaceuticals, it is not yet generating much revenue despite first being approved in late 2023. Administering Casgevy to patients with sickle cell disease (SCD) or transfusion-dependent beta-thalassemia (TDT) -- two rare blood-related disorders -- is a complex and lengthy process. That's one of the issues with the company's gene-editing medicines. CRISPR also remains unprofitable, a significant concern for many investors given the challenging and uncertain economy we face. Even so, the company might be worth serious consideration. Though it is taking time to ramp up sales, Casgevy's potential is vast even without potential label expansions. There is also very little competition to speak of for the medicine. There were no one-time curative treatments for SCD and TDT before the advent of gene-editing therapies. Casgevy should eventually generate well over $1 billion in annual sales. Furthermore, despite its disadvantages, the field of gene-editing has the potential to lead to major breakthroughs. Casgevy was an example. CRISPR's pipeline features other potential gems. CTX-112 is being tested in B-cell malignancies, CTX-131 in solid tumors, and CTX-211 in type 1 diabetes. Also, CTX-310 and CTX-320 target high levels of cholesterol. Of these, CTX-112 earned the Regenerative Medicine Advanced Therapy designation from the U.S. Food and Drug Administration, which means it has demonstrated promising signs of efficacy for treatment of a serious condition for which there is an unmet clinical need. CRISPR Therapeutics is somewhat on the riskier side, but the biotech company could soar in the next five years as it continues to make substantial clinical and regulatory progress. Shares have declined by 32% over the past year. Now may be a good time to initiate a small position on the dip. Regeneron is facing biosimilar competition for Eylea, one of its biggest growth drivers. In the first quarter, the company's revenue decreased by 4% year over year to $3 billion. But there is some good news. Regeneron has long been planning for the Eylea patent cliff, and if not for the approval of a new high-dose (HD) formulation of the medicine in late 2023, it would be in a much worse position. U.S. sales of Eylea HD increased by 54% year over year to $307 million in the period. This version of Eylea should continue to steal some patients away from the old formulation due to the former's better dosing schedule. Furthermore, Eylea HD could potentially earn label expansions, helping to mitigate Eylea-related losses. Meanwhile, Regeneron's other main growth driver, eczema treatment Dupixent, is still performing well. Regeneron co-markets Dupixent with Sanofi. The medicine's total sales in the first quarter grew 19% year over year to $3.67 billion. Dupixent hasn't peaked yet. Recent label expansions, including in treating chronic obstructive pulmonary disease (COPD), which it earned in the U.S. in October, should allow it to continue growing its revenue for a while. Elsewhere, Regeneron is developing newer medicines. It has been making strides in its oncology business lately. Within the next year, it could earn several brand-new approvals or label expansions in this field. These will also help it overcome the Eylea-related troubles. Lastly, Regeneron has a robust share-buyback program and offers a dividend it initiated this year. The sell-off it experienced over the last 12 months might have been justified. The company tried to fend off Eylea biosimilars in court, but that didn't work, leading to a significant drop in its share price, which was justifiable. But at current levels -- down by 38% over the trailing-12-month period -- Regeneron's shares look attractive. Before you buy stock in CRISPR Therapeutics, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and CRISPR Therapeutics wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $651,049!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $828,224!* Now, it's worth noting Stock Advisor's total average return is 979% — a market-crushing outperformance compared to 171% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of May 19, 2025 Prosper Junior Bakiny has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends CRISPR Therapeutics, Regeneron Pharmaceuticals, and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy. The 2 Smartest Beaten-Down Biotech Stocks to Buy on the Dip was originally published by The Motley Fool
Yahoo
19-05-2025
- Business
- Yahoo
Prediction: This Beaten-Down Stock Could Double in The Next 5 Years
Shares of CRISPR Therapeutics have lost momentum in recent years. The company's lone marketed product isn't generating much revenue yet. That should change over time, and the biotech could notch further clinical wins. 10 stocks we like better than CRISPR Therapeutics › Despite making significant clinical progress, CRISPR Therapeutics (NASDAQ: CRSP) has been struggling financially in the past three years. The company's shares are down by 24% over this period, while the S&P 500 has gained 41%. The gene-editing specialist has, no doubt, faced some headwinds. However, important catalysts could double its stock price by 2030. For those keeping score at home, that would amount to a compound annual growth rate of about 14.9%, well above the market's historical average returns. CRISPR Therapeutics' focus on gene editing has some advantages and disadvantages. On the one hand, its revolutionary techniques can allow researchers to create therapies for otherwise untreatable (or difficult-to-treat) conditions. CRISPR proved as much when it created and developed Casgevy, a one-time treatment that provides a functional cure for two genetic blood disorders: sickle cell disease and transfusion-dependent beta-thalassemia. CRISPR shares the rights to Casgevy together with Vertex Pharmaceuticals. On the other hand, ex vivo gene-editing treatments are complex to manufacture and administer. The process involves collecting the patient's cells, editing their genes, and reinserting them into the patient. That's why, despite being approved since late 2023, Casgevy isn't meaningfully contributing to CRISPR Therapeutics' financial results yet. But it will eventually, and at its peak, is almost certain to far exceed the $1 billion per year in sales that is the milestone for blockbuster status. There are few competing treatments for sickle cell disease and transfusion-dependent beta-thalassemia, even in the U.S. The competitive landscape looks even better for CRISPR and Vertex in several countries in the Middle East, where they have received approvals for this product. Overall, the two estimate an addressable market of 58,000 patients in the regions they are targeting. At $2.2 million per treatment course in the U.S., the opportunity looks massive. It might take time to get there, but expect CRISPR Therapeutics' sales to grow at a good clip once money starts rolling in from Casgevy, and that should happen well before the end of the decade. Casgevy's slow uptake is one reason CRISPR Therapeutics stock has not performed well in recent years. Another is that clinical progress often drives the performance of small biotechs with few or no products on the market. Some investors take their profits and leave once they achieve significant clinical and regulatory milestones. That's what happened here. CRISPR Therapeutics' shares skyrocketed in the five years following its 2016 IPO, a period during which it more than doubled. CRSP data by YCharts Though it has experienced a significant pullback since 2021, CRISPR Therapeutics could bounce back as it shows substantial pipeline progress while making commercial headway with Casgevy. The company is working on several promising candidates for which it could release data readouts as early as this year. They include CTX112 and CTX131, two potential cancer medicines. The former received the Regenerative Medicine Advanced Therapy designation from the Food and Drug Administration, a sign that it has shown promising clinical evidence in treating a serious condition. CRISPR Therapeutics should also release data from an ongoing clinical trial for CTX320, a medicine being developed to lower lipoprotein(a) levels, which can cause a range of cardiovascular problems when too high. These programs and others -- such as CRISPR's investigational functional cure for type 1 diabetes -- could make significant headway in the next five years. They might not all pan out, but even a 50% success rate in getting products to market, after the smashing success it saw by laser-focusing on Casgevy, would be a significant win for the company. In the meantime, a series of clinical wins could eventually jolt the company's shares. Of course, there is the possibility that the biotech will run into severe setbacks, and given that it only has one product on the market and remains unprofitable for now, that could send the stock down even further. CRISPR Therapeutics carries above-average risk as an investment, but for those comfortable with the volatility, it is worth initiating a small position in the stock -- it could deliver monster returns in the next five years. Before you buy stock in CRISPR Therapeutics, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and CRISPR Therapeutics wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $642,582!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $829,879!* Now, it's worth noting Stock Advisor's total average return is 975% — a market-crushing outperformance compared to 172% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of May 12, 2025 Prosper Junior Bakiny has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends CRISPR Therapeutics and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy. Prediction: This Beaten-Down Stock Could Double in The Next 5 Years was originally published by The Motley Fool
Yahoo
13-05-2025
- Business
- Yahoo
MaxCyte, Inc. (MXCT): A Bull Case Theory
We came across a bullish thesis on MaxCyte, Inc. (MXCT) on Substack by OppCost. In this article, we will summarize the bulls' thesis on MXCT. MaxCyte, Inc. (MXCT)'s share was trading at $2.355 as of May 9th. A scientist in a lab conducting research on cell-based therapeutics and biotechnology. MaxCyte, Inc. is a commercial-stage cell-engineering company with a strong strategic position in the rapidly growing cell and gene therapy (CGT) industry. The company's core offering, the ExPERT™ platform, leverages its Flow Electroporation® technology to enable the non-viral engineering of cells used in therapeutic development and manufacturing. MaxCyte operates a dual revenue model, combining consistent income from the sale and lease of instruments and single-use processing assemblies (PAs) with a high-potential, variable revenue stream from Strategic Platform Licenses (SPLs). These licenses are granted to therapeutic developers and offer milestones and royalties, representing a key growth avenue for the company. Despite the promise of its technology, MaxCyte has faced challenges with revenue volatility, particularly due to the lumpiness of SPL-related revenue recognition. While core business revenue has demonstrated steady growth, primarily driven by recurring PA sales linked to an expanding instrument base, total revenue declined in 2023 and 2024. Despite this, MaxCyte maintains high gross margins of 85-90%. However, the company has incurred widening net losses over the past five years, largely driven by significant investments in research and development (R&D) and selling, general, and administrative (SG&A) expenses aimed at fueling growth and expanding its platform. Operationally, the company is still in a negative cash flow phase but benefits from a robust balance sheet with $174.7 million in cash and short-term investments as of March 31, 2025, and no outstanding debt, providing it with significant runway for future growth. Strategically, MaxCyte has made key developments, such as the divestiture of its CARMA asset and the acquisition of SeQure Dx to expand into gene editing safety assessment. The company's SPL model gained significant validation with the first FDA approval of a partner therapy, Casgevy, in late 2023. However, near-term SPL revenue guidance for 2025 remains modest at around $5 million. Despite this, the company's core business continues to grow, and the SPL strategy offers substantial long-term royalty potential as more developers adopt its technology. MaxCyte's valuation is highly sensitive to assumptions regarding long-term growth and profitability, particularly the timing and magnitude of SPL royalty streams. A Discounted Cash Flow (DCF) analysis suggests an intrinsic value of $4.15 per share, with a 5-year forward Internal Rate of Return (IRR) of approximately 18.9%. However, the company faces significant risks, including cash burn, unpredictability in SPL revenue, and the challenges of achieving profitability. Despite these hurdles, MaxCyte represents a high-risk, high-reward investment opportunity, suited for investors with a long-term horizon who believe in the transformative potential of the CGT market and MaxCyte's ability to execute its SPL strategy. MaxCyte, Inc. (MXCT) is not on our list of the 30 Most Popular Stocks Among Hedge Funds. As per our database, 14 hedge fund portfolios held MXCT at the end of the fourth quarter which was 12 in the previous quarter. While we acknowledge the risk and potential of MXCT as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns, and doing so within a shorter timeframe. If you are looking for an AI stock that is more promising than MXCT but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock. READ NEXT: 8 Best Wide Moat Stocks to Buy Now and 30 Most Important AI Stocks According to BlackRock. Disclosure: None. This article was originally published at Insider Monkey.
Yahoo
13-05-2025
- Business
- Yahoo
2 Biotech Stocks That Are Screaming Buys in May
These players share a product, one set to generate significant growth over time. Both of these stocks offer investors a bargain at their prices today. 10 stocks we like better than CRISPR Therapeutics › It's very tempting to pile into the stocks with the strongest positive momentum of the moment -- they've proven themselves and could continue along this path. But in many cases, you could set yourself up for a bigger win if you look to quality companies that have seen their stocks stumble in recent times. This is because you'll buy at a reasonable price and potentially win as the stock recovers and goes on to gain. Two biotech stocks make perfect candidates for this sort of strategy right now. One has declined over the past four years, while the other just slipped in recent weeks. Each represents a bargain buy right now. It's also important to note that these two players share a product, one that should start generating significant revenue in the quarters to come. Let's take a close look at these two biotech stocks that are screaming buys in May. CRISPR Therapeutics (NASDAQ: CRSP) focuses on the exciting field of gene editing, a technology that "fixes" faulty genes responsible for disease. The CRISPR technique involves cutting DNA at a particular location to launch a natural repair process. Importantly, this biotech has proven that its technology works, scoring the first ever product approval for a CRISPR-based therapy. This approval was for blood disorders treatment Casgevy, and rollout began last year. The launch and revenue growth processes take longer for such a product than for standard medicines because gene editing involves several steps. All this means that we'll just start seeing revenue pick up momentum later this year. CRISPR Therapeutics partnered with big biotech Vertex Pharmaceuticals (NASDAQ: VRTX) on Casgevy. Although Vertex takes a larger share of profit at 60%, this is worthwhile because Vertex has the solid commercial experience and infrastructure that could help make the product successful. Vertex also bears the biggest share of the costs. Meanwhile, CRISPR Therapeutics has other promising candidates involved in clinical trials. It just presented encouraging phase 1 data for CTX310. This candidate could address a huge patient population, as it aims to treat people with high cholesterol. The company said that potentially 40 million people in the U.S. alone could be helped by such a product. CRISPR Therapeutics also expects trial updates from other candidates in the areas of oncology and autoimmune disease this year. So, this could be a catalyst-rich year for this biotech, making now, after the stock's 80% four-year decline, a great time to buy and hold on as this story moves into its next chapters. Vertex, as mentioned, partners with CRISPR Therapeutics on Casgevy, and that product should represent a new stream of revenue growth for the company as of this year. On top of this, it's important to remember that Vertex dominates the global cystic fibrosis (CF) treatment market with blockbuster drug Trikafta -- and Vertex just launched a new CF drug, Alyftrek, that's even better than its predecessor. Now Vertex is shifting its Trikafta patients to Alyftrek. In its recent earnings call, the company said the launch of this product, as well as the rollout of another new release -- painkiller Journavx -- are going well. These drugs represent billion-dollar opportunities for Vertex, and Journavx is particularly interesting because it opens the door to an entirely new treatment area for the company. Journavx is a non-opioid pain drug approved for moderate to severe acute pain, and Vertex continues to usher it through clinical trials for other pain indications, including chronic pain. The Journavx approval shows that Vertex not only can be successful in CF, but in other major treatment areas as well. Vertex shares climbed 83% over three years through the start of May. But since Vertex's earnings report last week, when it spoke of a temporary illegal generic competition problem in Russia, the stock has retreated 15%. This has left Vertex trading for 23x forward earnings estimates, down from more than 28x just a few days ago. Considering Vertex's leadership in CF and its new growth drivers, biotech investors won't want to miss out on this top biotech at this bargain price. Before you buy stock in CRISPR Therapeutics, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and CRISPR Therapeutics wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $614,911!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $714,958!* Now, it's worth noting Stock Advisor's total average return is 907% — a market-crushing outperformance compared to 163% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of May 12, 2025 Adria Cimino has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends CRISPR Therapeutics and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy. 2 Biotech Stocks That Are Screaming Buys in May was originally published by The Motley Fool Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
