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Yahoo
23 minutes ago
- Yahoo
Novartis to pay Matchpoint up to $1 billion to develop anti-inflammatory therapies
(Reuters) -Swiss drugmaker Novartis will pay up to $1 billion to U.S. biotech Matchpoint Therapeutics to develop oral drugs for several inflammatory diseases. Matchpoint said on Thursday it will use its technology to develop drugs that block the activity of a specific protein, helping to lower the production of inflammation-causing signals. The company will lead the research and drug development process, using the funding from Novartis. If Novartis exercises its option to exclusively license the program, the drugmaker will have global rights to develop and commercialize all products resulting from the collaboration. Matchpoint said it will receive up to $60 million in upfront payment and research funding, with up to $1 billion in total potential payments, including option exercise fee, and development and commercial milestones. 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
Business Wire
an hour ago
- Business Wire
Aldeyra Therapeutics Receives Orphan Designation from the European Medicines Agency for ADX-2191 for the Treatment of Inherited Retinal Dystrophies including Retinitis Pigmentosa
LEXINGTON, Mass.--(BUSINESS WIRE)-- Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the European Medicines Agency (EMA) has granted Orphan Designation for ADX-2191 (methotrexate intravitreal injection) for the treatment of inherited retinal dystrophies of the rod-dominant phenotype, including retinitis pigmentosa. There are currently no approved drug treatments for patients with most forms of retinitis pigmentosa, a clinical group of rare genetic eye diseases characterized by retinal cell death and loss of vision. Retinitis pigmentosa affects more than one million people worldwide and remains a significant cause of inherited blindness. 'Retinitis pigmentosa is a serious and incurable sight-threatening disease that represents a major unmet need in the field of ophthalmology,' stated Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. 'The receipt of Orphan Designation from the EMA, in conjunction with the previously announced Orphan Drug Designation from the U.S. Food and Drug Administration, highlights the importance of developing a treatment option for patients suffering from retinitis pigmentosa.' The potential activity of ADX-2191 in retinitis pigmentosa is supported by results from a Phase 2 clinical trial, announced in 2023, which demonstrated improvements from baseline in retinal sensitivity following treatment. A planned Phase 2/3 clinical trial of ADX-2191 in retinitis pigmentosa is expected to initiate in 2025. The EMA grants orphan designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare, life-threatening, or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity in the European Union. About ADX-2191 ADX-2191 (methotrexate intravitreal injection) is a sterile, non-compounded intravitreal formulation of methotrexate for the potential treatment of specific rare retinal diseases, including primary vitreoretinal lymphoma and retinitis pigmentosa. The ADX-2191 intravitreal formulation is preservative-free, is designed to be vitreous-compatible, and is optimized for excipient composition, viscosity, density, tonicity, pH, concentration, and volume of administration. ADX-2191 has received U.S. Food and Drug Administration (FDA) Orphan Drug Designation for the treatment of primary vitreoretinal lymphoma and retinitis pigmentosa, and EMA Orphan Designation for the treatment of inherited retinal dystrophies of the rod-dominant phenotype, including retinitis pigmentosa. About Retinitis Pigmentosa Retinitis pigmentosa is a group of rare genetic eye diseases characterized by retinal cell death and loss of vision. There are currently no approved treatments for most forms of retinitis pigmentosa. In vivo preclinical research has identified the activity of methotrexate in inducing misfolded rhodopsin (a visual cycle protein) clearance, suggesting the potential of ADX‑2191 to treat genetic forms of retinitis pigmentosa that are characterized by misfolded rhodopsin. About Aldeyra Aldeyra Therapeutics is a biotechnology company devoted to discovering innovative therapies designed to treat immune-mediated and metabolic diseases. Our approach is to develop pharmaceuticals that modulate protein systems, instead of directly inhibiting or activating single protein targets, with the goal of optimizing multiple pathways at once while minimizing toxicity. Our product candidates include RASP (reactive aldehyde species) modulators ADX-629, ADX‑248, ADX-743, ADX-631, ADX-246, and chemically related molecules for the potential treatment of systemic and retinal immune-mediated and metabolic diseases. Our late-stage product candidates are reproxalap, a RASP modulator for the potential treatment of dry eye disease and allergic conjunctivitis, and ADX-2191, a novel formulation of intravitreal methotrexate for the potential treatment of primary vitreoretinal lymphoma and retinitis pigmentosa. Safe Harbor Statement This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Aldeyra's future expectations, plans, and prospects, including without limitation statements regarding: the goals, opportunity, and potential for ADX-2191; and the outcome and timing of any clinical trials of ADX-2191. Aldeyra intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. In some cases, you can identify forward-looking statements by terms such as, but not limited to, 'may,' 'might,' 'will,' 'objective,' 'intend,' 'should,' "could," 'can,' 'would,' 'expect,' 'believe,' 'anticipate,' 'project,' 'on track,' 'scheduled,' 'target,' 'design,' 'estimate,' 'predict,' 'contemplates,' 'likely,' 'potential,' 'continue,' 'ongoing,' 'aim,' 'plan,' or the negative of these terms, and similar expressions intended to identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions, and uncertainties. Aldeyra is at an early stage of development and may not ever have any products that generate significant revenue. All of Aldeyra's development timelines may be subject to adjustment depending on recruitment rate, regulatory review, preclinical and clinical results, funding, and other factors that could delay the initiation, enrollment, or completion of clinical trials. Important factors that could cause actual results to differ materially from those reflected in Aldeyra's forward-looking statements include, among others, the timing of enrollment, commencement and completion of Aldeyra's clinical trials, the timing and success of preclinical studies and clinical trials conducted by Aldeyra and its development partners; delay in or failure to obtain regulatory approval of Aldeyra's product candidates, including as a result of the FDA not accepting Aldeyra's regulatory filings, issuing a complete response letter, or requiring additional clinical trials or data prior to review or approval of such filings or in connection with resubmissions of such filings; the ability to maintain regulatory approval of Aldeyra's product candidates, and the labeling for any approved products; the risk that prior results, such as signals of safety, activity, or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or clinical trials involving Aldeyra's product candidates in clinical trials focused on the same or different indications; the scope, progress, expansion, and costs of developing and commercializing Aldeyra's product candidates; uncertainty as to Aldeyra's ability to commercialize (alone or with others) and obtain reimbursement for Aldeyra's product candidates following regulatory approval, if any; the size and growth of the potential markets and pricing for Aldeyra's product candidates and the ability to serve those markets; Aldeyra's expectations regarding Aldeyra's expenses and future revenue, the timing of future revenue, the sufficiency or use of Aldeyra's cash resources and needs for additional financing; the rate and degree of market acceptance of any of Aldeyra's product candidates; Aldeyra's expectations regarding competition; Aldeyra's anticipated growth strategies; Aldeyra's ability to attract or retain key personnel; Aldeyra's commercialization, marketing and manufacturing capabilities and strategy; Aldeyra's ability to establish and maintain development partnerships; Aldeyra's ability to successfully integrate acquisitions into its business; Aldeyra's expectations regarding federal, state, and foreign regulatory requirements; political, economic, legal, social, and health risks, public health measures, and war or other military actions, that may affect Aldeyra's business or the global economy; regulatory developments in the United States and foreign countries; Aldeyra's ability to obtain and maintain intellectual property protection for its product candidates; the anticipated trends and challenges in Aldeyra's business and the market in which it operates; and other factors that are described in the 'Risk Factors' and 'Management's Discussion and Analysis of Financial Condition and Results of Operations' sections of Aldeyra's Annual Report on Form 10-K for the year ended December 31, 2024, and Aldeyra's Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, which are on file with the Securities and Exchange Commission (SEC) and available on the SEC website at Additional factors may be described in those sections of Aldeyra's Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, expected to be filed with the SEC in the third quarter of 2025, and Aldeyra's other filings with the SEC. In addition to the risks described above and in Aldeyra's other filings with the SEC, other unknown or unpredictable factors also could affect Aldeyra's results. No forward-looking statements can be guaranteed and actual results may differ materially from such statements. The information in this release is provided only as of the date of this release, and Aldeyra undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
National Geographic
an hour ago
- National Geographic
Will psychedelics ever live up to their hype?
Psychedelic drugs, like MDMA, have garnered promising results in treating a long list of mental health conditions. But some researchers say the studies have some key shortcomings. Photograph by Daria Kulkova, Getty Images The mind-expanding drugs may help heal many mental health conditions. But studies haven't actually proved that yet. In recent years, the conversation around psychedelic medicine has shifted from hushed discussions to headline news, with many media articles trumpeting positive scientific findings. Studies have found psychedelics effective for a wide range of mental health conditions, including depression, post-traumatic stress disorder, and substance abuse, and even physical conditions such as chronic pain. Results have been so promising that three states, Oregon, Colorado, and New Mexico, have created pathways for psychedelics to be legally administered there. And at least in part because of the positive research news, illegal use of the drugs—a category that includes psilocybin (the active ingredient in magic mushrooms), lysergic acid diethylamide (LSD), ayahuasca, and other compounds that alter consciousness—has risen across the United States. For example, psilocybin use in people over 30 increased by nearly 200 percent between 2019 and 2023, with many saying they took the drug to improve a mental health condition or chronic pain. Momentum is building towards a possible psychedelic drug approval by the U.S. Food and Drug Administration (FDA) in the next few years. Yet some in the field say the research continues to have significant problems that make broad conclusions about their benefit premature. Despite all the hype, 'we cannot conclude anything as of yet about the safety or efficacy of psychedelic therapy' for many of the conditions it's been touted to treat, says Michiel van Elk, a cognitive neuroscientist at Leiden University in the Netherlands, who coauthored an analysis in 2023 of the research's shortcomings. Some of the red flags took the spotlight last year, when the FDA rejected one of the most well-studied of the drugs, methylenedioxy-methamphetamine (MDMA), as a treatment for post-traumatic stress disorder. Even MDMA requires another large clinical trial to address lingering concerns, van Elk says. Some issues plaguing psychedelic research stem from the youthfulness of the field. Although scientists preliminarily studied the drugs during the 1950s and 60s, most of the research has happened in the past decade. But other hurdles are specific to experimenting with mind-altering substances. Here are three of the major concerns. 1. Psychedelic studies have been shockingly small. Studying these compounds is admittedly challenging. The drugs must be administered in person, generally in the presence of two trained facilitators who stay with each clinical trial participant for the six to ten hours the substance remains active. People also generally talk to therapists several times in the weeks before and after the experience. Plus, the medicine is time-consuming to get, due to restrictive government regulations. All told, running a single participant through a psychedelic study can cost tens of thousands of dollars, van Elk estimates. (Who will guide your psychedelic medicine trip?) To make studies affordable, most psychedelic research has included a small number of participants. One widely touted study finding benefits of psilocybin for major depression involved just 27 participants. Only 39 people took part a study documenting LSD's advantages for treating anxiety. Even the two studies submitted to the FDA in 2024 seeking approval for MDMA included 90 and 104 participants, respectively. By contrast, when the diabetes drug Ozempic was approved in 2017, its manufacturer submitted results in more than 4,000 people. In studies this small, a few individuals having unique experiences can skew the findings, especially if the people differ from the average to start. 'People who agree to these trials may not reflect the general population: They may be more desperate, more resistant of conventional treatments, more willing to try something wildly different from the norm,' says Frederick Barrett, a neuroscientist and director of the Center for Psychedelic and Consciousness Research at Johns Hopkins University School of Medicine. Only larger studies can confirm positive results from these small trials. 'If you want to say anything meaningful, you need to increase sample sizes by hundreds of participants,' van Elk says. One way to bring up the numbers is to pool results. Researchers did this in 2024, combining nine clinical trials on psilocybin for depression, which together found the drug twice as effective as a placebo, the inert substance used for comparison in drug trials. But even this pooled analysis, while lending support to prior results, involved just 436 total participants. 2. People are bummed when they figure out that they didn't get the drug. The most reliable studies are 'double-blind,' where neither the person giving the medicine nor the person taking it knows whether they got the drug or the placebo. This helps keep people from incorrectly attributing health improvements to the medicine. But it's nearly impossible to keep study participants from the fact that they've taken a psychedelic when the room starts singing or their hands disappear before them. 'Psychedelic studies go through the motion, but it's functionally not blinded. Everybody figures it out,' says Balázs Szigeti, a clinical data scientist in the Translational Psychedelic Research program at the University of California, San Francisco, who studies this issue. People who don't get the drug seem to especially disappointed—more so than with other medications. 'Nobody wants to be in the placebo group,' Szigeti says. 'It's a great conversation when at cocktail parties you tell your friends you're going to be in a psychedelic drug trial. But imagine months later at the same social gathering when you have to tell them nothing happened.' This letdown may skew results, causing placebo-takers in psychedelic studies to underreport improvements. In a 2024 review, researchers found that people getting placebos in clinical trials testing the antidepressant escitalopram reported greater improvements in their depression levels than comparable studies for psychedelics. The wider gap between the psychedelic and placebo group artificially makes psychedelics look better, especially when they are compared to antidepressants, Szigeti says. In reality, the two treatments may be more evenly matched. Szigeti compared results from psychedelic and antidepressant studies without a placebo group, where everyone got an active treatment, and his unpublished data suggest they actually have similar effects. Of course, that may not be a bad thing. Antidepressant pills must be taken daily and come with side effects like weight gain and sexual disfunction. In comparison, one or two psychedelic sessions, which seem to convey lasting effects, could provide an appealing alternative for some patients. But such a finding would be far from the hype 'that psychedelics can cure depression,' Barrett notes. Scientists have been unable to find a placebo that does not tip off participants. They've tried a form of vitamin B that causes tingling and flushing, a cough medicine that can make people woozy, and even low doses of the psychedelic itself. But when people are asked during the clinical trial, most know they were not given the psychedelic. A more effective approach may be to randomize people earlier in the process so the placebo group never knows they could have gotten a psychedelic, Szigeti says. Studies could also be designed with a so-called cross-over design where everyone eventually gets the psychedelic, minimizing the disappointment of those who initially do not. 3. It's unclear how addictive some substances are. One of the biggest concerns raised by the FDA advisory committee assessing MDMA was the potential for abuse should the drug become legally available. Committee members complained that the company submitting the application, Lykos Therapeutics, had not asked study participants if they experienced euphoria or elation while on the drug—a sign some might seek to later abuse it. More than two million people in the U.S. illegally use MDMA, known on the street as Ecstasy or Molly—making it the fourth most common street drug after marijuana, cocaine, and methamphetamine. (Five recent scientific findings that change what we know about cannabis.) Psilocybin and LSD are less likely to be psychologically addictive than MDMA, Barrett says. In lab studies, animals exposed to an addictive drug like cocaine seek it out to the exclusion of food and water, but 'you have to bend over backwards' to get animals to take these psychedelics, he says. Still, the potential for abuse is rarely addressed in psychedelic research, even though critics think it always should be. 'Absence of data is not evidence of absence,' van Elk says. The potential for addiction is one reason drug companies are working to produce psychedelics that convey a health benefit without inducing the hallucinations or euphoria that might entice people to seek out these substances. (Scientists are creating psychedelics without the trips.) As the field of psychedelic science matures and seeks broader acceptance, including perhaps FDA approval of one or more of the drugs, a growing number of researchers are starting to take critiques of their work to heart. 'People are becoming aware that we potentially face a credibility crisis,' van Elk says, 'and that we need to do everything we can to make sure that we do the science according to the highest standards possible.'
