Latest news with #Casgevy
Yahoo
a day ago
- Health
- Yahoo
Pfizer's sickle cell disease candidate fails Phase III trial
Pfizer is mulling its options after its investigational sickle cell disease drug failed to meet the primary endpoint in a Phase III trial. The THRIVE-131 study (NCT04935879) was evaluating inclacumab, an investigational P-selectin inhibitor, in patients 16 years of age and older with sickle cell disease. The trial failed to meet its primary endpoint of a significant reduction in the rate of vaso-occlusive crises (VOCs) after 48 weeks, placing the drug's future in jeopardy. Pfizer's chief inflammation and immunology officer Dr Michael Vincent, said: 'While the THRIVE-131 results did not meet our expectations, we remain committed to better understanding these results and sharing them with the medical and sickle cell community in the interest of advancing our collective understanding of sickle cell disease. We remain focused on our mission of bringing much-needed treatments to patients with sickle cell disease.' The therapy was generally well tolerated in THRIVE-131, with the most common adverse events (AEs) being anaemia, arthralgia, back pain, headache, malaria, sickle cell anaemia with crisis, and upper respiratory tract infection. Analyses of the data will be shared with the scientific and patient community in due course. The therapy is still being assessed in the THRIVE-133 OLE trial (NCT05348915) to evaluate the long-term safety of inclacumab. If approved, GlobalData predicts global sales of the drug to reach $267m in 2031. GlobalData is the parent company of Clinical Trials Arena. Pfizer's sickle cell disease woes This is the latest hit in sickle cell disease for Pfizer. In September 2024, it withdrew its therapy Oxbryta (voxelotor) in all approved markets after data suggested an imbalance in VOCs and fatal events. The company has reviewed and shared the data with the US Food and Drug Administration (FDA) and European Medicines Agency (EMA). The company is also awaiting the lifting of a partial clinical hold by the FDA on its Phase III trial of osivelotor, a haemoglobin S polymerisation inhibitor, in sickle cell disease. The FDA's order has paused enrolment into the trial, with Pfizer stating data will be shared as it becomes available. Approved therapies in the sickle disease treatment space are combating slow growth. Vertex and CRISPR Therapeutics' Casgevy (exagamglogene autotemcel) only reached revenue of $10m in 2024, with a $2.2m price tag per patient proving a barrier to initial uptake. Vertex said it expects the number of new patients initiating cell collection to grow significantly throughout 2025, with signals that this is already happening. GlobalData predicts Casgevy sales will increase significantly in 2025, with a forecast of $107m, with projections that the gene therapy will reach blockbuster status in 2030. The therapy gained approval by the National Institute for Health and Care Excellence (NICE) for use in sickle cell disease in January 2025, meaning it is now available for use on the UK's National Health Service (NHS). "Pfizer's sickle cell disease candidate fails Phase III trial" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. 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
Yahoo
2 days ago
- Business
- Yahoo
Here's How This Forgotten Healthcare Stock Could Generate Life-Changing Returns
Key Points CRISPR Therapeutics' first approved therapy, Casgevy, was a breakthrough. One of Casgevy's biggest achievements may be demonstrating the viability of CRISPR Therapeutics' strategy. The biotech company could soar if it can follow up that win with more clinical and regulatory milestones. 10 stocks we like better than CRISPR Therapeutics › Over the past few years, the market hasn't been kind to somewhat speculative, unprofitable stocks. CRISPR Therapeutics (NASDAQ: CRSP), a mid-cap biotech, fits that description. The company's shares are down by 24% since mid-2022. The S&P 500 is up 50% over the same period. Despite this terrible performance, there are reasons to believe that CRISPR Therapeutics could still generate life-changing returns for investors willing to be patient. Here's how the biotech could pull it off. CRISPR Therapeutics' first success CRISPR Therapeutics' first approval was for Casgevy, a treatment for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), which it developed in collaboration with Vertex Pharmaceuticals. Before Casgevy, no CRISPR-based gene-editing medicine had been approved. While it became the first, it still faces some challenges. Ex vivo gene-editing therapies require a complex manufacturing and administration process that can only be performed in authorized treatment centers (ATCs). Moreover, they're expensive. Casgevy costs $2.2 million in the U.S. Getting third-party payers on board for that is no easy feat. Still, CRISPR Therapeutics and Vertex Pharmaceuticals are making steady progress. As of the second quarter, CRISPR Therapeutics had achieved its goal of activating 75 ATCs. It had also secured reimbursement for eligible patients in 10 countries. The two companies estimate there are roughly 60,000 eligible SCD and TDT patients in the regions they have targeted. Let's say they continue to strike reimbursement deals and can count on third-party coverage for 70% of this target population (42,000 people), then go on to treat another 30% of that group in the next decade (12,600 patients). Assuming they could extend that $2.2 million price tag to those countries, Casgevy could generate more than $27.7 billion over this period. Based on its agreement with Vertex, 40% would go to CRISPR Therapeutics, or roughly $11.1 billion over a decade. That's not bad, but it's not that impressive either. So, while Casgevy could contribute meaningfully to CRISPR Therapeutics' results -- and may even reach blockbuster status at some point -- the medicine may primarily serve as a proof of concept to demonstrate that the biotech's approach can be effective. Substantial progress with its first commercialized product will help the stock price. But the company's performance will depend even more on future clinical and regulatory milestones, especially as it shows with Casgevy that it can manage the intricacies and complexities of marketing gene-editing medicines. Can the pipeline deliver? CRISPR Therapeutics has six candidates in clinical trials, which isn't bad at all for a mid-cap biotech company. One of its leading programs is CTX310, a potential therapy designed to help reduce low-density lipoprotein (LDL) cholesterol in patients with certain conditions. CTX310 is already producing encouraging clinical trial results. Additionally, it's an in vivo medicine, meaning it bypasses the need to harvest patients' cells to manufacture therapies; in vivo gene-editing treatments are easier to handle than their ex vivo counterparts. The company's path to creating life-changing returns hinges on its ability to deliver consistent clinical and regulatory wins over the next few years for CTX310 and other important candidates. If CRISPR Therapeutics can successfully launch several new products in the next five to seven years, its shares are likely to skyrocket. In the meantime, under this scenario, the company would succeed in making gene-editing medicines more mainstream. This would encourage third-party payers to get on board -- and healthcare institutions, and perhaps even governments, to help push for more ATCs, since there'd be a greater need to accommodate these treatments. Can CRISPR Therapeutics achieve this? In my view, the biotech stock is on the riskier side, but does carry significant upside potential. There's a (small) chance the gene-editing specialist will deliver life-changing returns in the next decade, but investors need to hedge their bets. It's best to start by initiating a small position in the stock, then progressively add more if CRISPR Therapeutics lands more wins. Should you invest $1,000 in CRISPR Therapeutics right now? 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 $668,155!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $1,106,071!* Now, it's worth noting Stock Advisor's total average return is 1,070% — a market-crushing outperformance compared to 184% for the S&P 500. Don't miss out on the latest top 10 list, available when you join Stock Advisor. See the 10 stocks » *Stock Advisor returns as of August 13, 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. Here's How This Forgotten Healthcare Stock Could Generate Life-Changing Returns was originally published by The Motley Fool
Yahoo
2 days ago
- Business
- Yahoo
Here's How This Forgotten Healthcare Stock Could Generate Life-Changing Returns
Key Points CRISPR Therapeutics' first approved therapy, Casgevy, was a breakthrough. One of Casgevy's biggest achievements may be demonstrating the viability of CRISPR Therapeutics' strategy. The biotech company could soar if it can follow up that win with more clinical and regulatory milestones. 10 stocks we like better than CRISPR Therapeutics › Over the past few years, the market hasn't been kind to somewhat speculative, unprofitable stocks. CRISPR Therapeutics (NASDAQ: CRSP), a mid-cap biotech, fits that description. The company's shares are down by 24% since mid-2022. The S&P 500 is up 50% over the same period. Despite this terrible performance, there are reasons to believe that CRISPR Therapeutics could still generate life-changing returns for investors willing to be patient. Here's how the biotech could pull it off. CRISPR Therapeutics' first success CRISPR Therapeutics' first approval was for Casgevy, a treatment for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), which it developed in collaboration with Vertex Pharmaceuticals. Before Casgevy, no CRISPR-based gene-editing medicine had been approved. While it became the first, it still faces some challenges. Ex vivo gene-editing therapies require a complex manufacturing and administration process that can only be performed in authorized treatment centers (ATCs). Moreover, they're expensive. Casgevy costs $2.2 million in the U.S. Getting third-party payers on board for that is no easy feat. Still, CRISPR Therapeutics and Vertex Pharmaceuticals are making steady progress. As of the second quarter, CRISPR Therapeutics had achieved its goal of activating 75 ATCs. It had also secured reimbursement for eligible patients in 10 countries. The two companies estimate there are roughly 60,000 eligible SCD and TDT patients in the regions they have targeted. Let's say they continue to strike reimbursement deals and can count on third-party coverage for 70% of this target population (42,000 people), then go on to treat another 30% of that group in the next decade (12,600 patients). Assuming they could extend that $2.2 million price tag to those countries, Casgevy could generate more than $27.7 billion over this period. Based on its agreement with Vertex, 40% would go to CRISPR Therapeutics, or roughly $11.1 billion over a decade. That's not bad, but it's not that impressive either. So, while Casgevy could contribute meaningfully to CRISPR Therapeutics' results -- and may even reach blockbuster status at some point -- the medicine may primarily serve as a proof of concept to demonstrate that the biotech's approach can be effective. Substantial progress with its first commercialized product will help the stock price. But the company's performance will depend even more on future clinical and regulatory milestones, especially as it shows with Casgevy that it can manage the intricacies and complexities of marketing gene-editing medicines. Can the pipeline deliver? CRISPR Therapeutics has six candidates in clinical trials, which isn't bad at all for a mid-cap biotech company. One of its leading programs is CTX310, a potential therapy designed to help reduce low-density lipoprotein (LDL) cholesterol in patients with certain conditions. CTX310 is already producing encouraging clinical trial results. Additionally, it's an in vivo medicine, meaning it bypasses the need to harvest patients' cells to manufacture therapies; in vivo gene-editing treatments are easier to handle than their ex vivo counterparts. The company's path to creating life-changing returns hinges on its ability to deliver consistent clinical and regulatory wins over the next few years for CTX310 and other important candidates. If CRISPR Therapeutics can successfully launch several new products in the next five to seven years, its shares are likely to skyrocket. In the meantime, under this scenario, the company would succeed in making gene-editing medicines more mainstream. This would encourage third-party payers to get on board -- and healthcare institutions, and perhaps even governments, to help push for more ATCs, since there'd be a greater need to accommodate these treatments. Can CRISPR Therapeutics achieve this? In my view, the biotech stock is on the riskier side, but does carry significant upside potential. There's a (small) chance the gene-editing specialist will deliver life-changing returns in the next decade, but investors need to hedge their bets. It's best to start by initiating a small position in the stock, then progressively add more if CRISPR Therapeutics lands more wins. Should you invest $1,000 in CRISPR Therapeutics right now? 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 $668,155!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $1,106,071!* Now, it's worth noting Stock Advisor's total average return is 1,070% — a market-crushing outperformance compared to 184% for the S&P 500. Don't miss out on the latest top 10 list, available when you join Stock Advisor. See the 10 stocks » *Stock Advisor returns as of August 13, 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. Here's How This Forgotten Healthcare Stock Could Generate Life-Changing Returns was originally published by The Motley Fool Sign in to access your portfolio
Boston Globe
04-08-2025
- Business
- Boston Globe
Vertex next-generation pain candidate fails Phase 2 trial
'We do not plan to advance VX-993 as monotherapy in acute pain, because we do not expect that it will be superior to our [existing] NaV1.8 inhibitors,' said CEO Reshma Kewalramani during a Monday afternoon earnings call with investors, using a scientific shorthand for the class of drugs. She noted that the company will continue a trial testing the drug in patients with diabetes who have chronic nerve pain. It was one of multiple setbacks the company disclosed for its pain franchise Monday, as it announced its second-quarter earnings. Advertisement Vertex also announced that longtime chief scientist David Altshuler was retiring next year, to be replaced by head of global research Mark Bunnage. Altshuler, an accomplished academic geneticist, took the post in 2015 and presided over the launch of its most important Advertisement Although Journavx was On Monday, though, the company disclosed that the the Food and Drug Administration 'indicated they do not see a path' for Vertex to obtain a broad approval for Journavx in 'peripheral neuropathic pain' — i.e., chronic pain caused by nerve damage or dysfunction. Instead the company will have to obtain approval in individual subsets of chronic pain. Accordingly, Vertex said it will cancel a planned Phase 3 trial in lumbosacral radiculopathy, also known as sciatica, which the company said affects about 4 million people. Journavx had already failed to outperform placebo in a Phase 2 sciatica trial. Instead it will launch a second Phase 3 trial in diabetic peripheral neuropathy, which affects about 2 million people, in the hope of first getting approval in that indication. At the same time, Vertex announced better-than-expected sales for Journavx in acute pain, with more than 110,000 prescriptions filled. Although the pill was hailed as a potential public health breakthrough for providing a non-addictive alternative to opioids, analysts questioned how widely they would be prescribed, given the availability of cheap alternatives. On Monday, Vertex said it earned $12 million from Journavx, compared to consensus Wall Street projections of $6.7 million. Advertisement Vertex also said it has now treated 29 patients with Casgevy, its gene editing therapy for sickle cell disease, including 16 in the last quarter. Casgevy, which has been slow to launch, earned $30.4 million from second-quarter sales.
Yahoo
29-07-2025
- Business
- Yahoo
The 3 Things That Matter for CRISPR Therapeutics Now
Key Points CRISPR Therapeutics' gene-editing treatments are highly customized and shockingly expensive. The market is watching to see if the underlying science can be applied to treat many diseases. The company has plenty of money right now, but its expenses could grow quite a bit from here. 10 stocks we like better than CRISPR Therapeutics › Biotechnology outfit CRISPR Therapeutics (NASDAQ: CRSP) isn't a name on many investors' radar -- and understandably so. Its market capitalization is a mere $6 billion. The company remains in the red largely because it's barely got any revenue to speak of. It's not likely to swing to a profit in the immediate future, either. Still, if you've got room in your portfolio for a little more risk paired with above-average upside potential, this is a stock worth adding to your watchlist (if not your portfolio) with three key things in mind. But first things first. What's CRISPR Therapeutics? Although the company was founded back in 2013, most of its time since then has been spent refining the work first done by Jennifer Doudna, Ph.D., and co-founder Emmanuelle Charpentier, Ph.D., who jointly figured out how to "edit" defective genetic code in a strand of DNA. By using a protein called Cas9 to find and remove a damaged portion of a genetic sequence and then replace it using a gene-editing biotechnology based on clustered regularly interspaced short palindromic repeats -- or CRISPR -- medical science is now able to do what was once unthinkable. It's still early days for the science -- very early. In fact, the FDA only made its first-ever approval of a gene-editing therapy in December of 2023. That's Casgevy, for the treatment of sickle cell disease, which was approved in the U.K. only a month earlier. The thing is, Casgevy is CRISPR Therapeutics' treatment, underscoring how well developed this biotech outfit's science is, and perhaps indirectly underscoring the fact that many rival drug developers are still well behind. Casgevy isn't the company's proverbial big Kahuna, however -- it's merely proof that gene editing can be successfully done. The heavy hitters in CRISPR's developmental pipeline are CTX310 for the treatment of certain cardiovascular diseases, and CTX131 and CTX112, both of which are taking aim at cancer using the very same CRISPR science. Although all of these drugs still have years of developmental work ahead of them, again, the underlying gene-editing technology works. Its potential applications are enormous. That's why CRISPR has a dozen or so others in clinical or pre-clinical trials also underway at this time. Three things to watch As time marches on, though, this stock's backstory is evolving from one broadly driven by an idea to one that increasingly hinges on some very specific factors. To this end, here are the three things that matter the most to current and prospective CRISPR Therapeutics shareholders right now -- and for the foreseeable future -- since they'll either drive the stock higher or let it slide into a sell-off. 1. Insurers and patients' acceptance of CRISPR-based medicine's cost While the science of using CRISPR to repair faulty cells is exciting, it's not exactly cheap or easy. See, Casgevy isn't a pill or an injection. It requires a sample of a patient's own blood stem cells to create a completely customized therapy, which is then infused back into that patient after he or she has undergone chemotherapy. All of this care can only be done at one of CRISPR's 65 authorized treatment centers. Total cost? A little over $2 million per patient. That's pretty steep for any therapy, but particularly for sickle cell disease, which at least has a handful of more affordable treatment options. The cost of treating life-threatening cancer is less of a stumbling block, even for insurers that may occasionally see bills nearly this size for even the most conventional of oncology treatment regimens. The price tag of this and any other future CRISPR-based therapy, however, is likely to remain in this ballpark, where payers may well balk. 2. The ongoing development of CTX310 Again, while Casgevy is approved to treat sickle cell disease, it's really more of a proving ground for the other drugs in CRISPR Therapeutics' developmental pipeline. This, of course, includes CTX131 and CTX112, but the company itself is putting the spotlight on CTX310 as a treatment for ANGPTL3 (angiopoietin-like 3), which is often associated with poorly regulated cholesterol, lipids, and triglycerides. If the company can demonstrate its solution is at least as good as (if not better than) alternative cholesterol-fighting drugs, investors might continue to support this stock. This information is coming. The company posted encouraging early results of CTX310's phase 1 clinical trial late last month, and says it intends to offer more detailed data at a healthcare conference scheduled for later in the year. If it's compelling enough, it could tamp down worries over the high cost of any CRISPR-based treatment. 3. Liquidity Finally, you'll want to keep an eye on CRISPR Therapeutics' liquidity, or the amount of cash it has on hand to cover its operating costs while it continues to develop CTX310 at the same time it's looking to expand Casgevy's commercialization. As of the end of the first quarter, CRISPR was sitting on $1.86 billion in cash, which is a lot for a company of this size. It's working through this money pretty quickly, though, shelling out nearly $150 million in operating expenses in Q1 alone. That doesn't include a full quarter's worth of the sort of costs the phase 1 trial of CTX310 is incurring now, or any other trials it starts or expands in the foreseeable future. Continued revenue growth from Casgevy should seemingly help offset some of this spending, even if not all of it. Indeed, the analyst community expects CRISPR's top line to soar from less than $50 million this year to more than $400 million in 2027, even if these same analysts believe the company will still be well in the red then. A few years' worth of losses isn't exactly unusual for a young biotech start-up. CRISPR Therapeutics would still lack much-needed scale even after such growth, though. The money it needs to spend just to get any meaningful degree of traction from Casgevy or maintain its clinical trials could far exceed any conceivable amount of revenue the newly approved drug might produce in the foreseeable future. Don't confuse what CRSP stock is So what's the call? There isn't one -- not this time. Buying or avoiding a stake in CRISPR Therapeutics is entirely up to you, depending on your risk tolerances and your ability to manage such a holding. If you're strictly a buy-and-hold investor, there's probably not enough certainty here yet to latch onto for the long haul. And there may never be. If you've got a speculative side that can tolerate risk in exchange for hype-driven reward, though, you could make a decent bullish case. Just don't confuse the two, or muddy the waters by trying to be both. The last thing you want to do here is talk yourself into a long-term position that requires you to ignore obvious red flags like the three potential ones discussed above. Should you buy stock in CRISPR Therapeutics right now? 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 $636,628!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $1,063,471!* Now, it's worth noting Stock Advisor's total average return is 1,041% — a market-crushing outperformance compared to 183% for the S&P 500. Don't miss out on the latest top 10 list, available when you join Stock Advisor. See the 10 stocks » *Stock Advisor returns as of July 28, 2025 James Brumley has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends CRISPR Therapeutics. The Motley Fool has a disclosure policy. The 3 Things That Matter for CRISPR Therapeutics Now 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
