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Medscape
9 hours ago
- Health
- Medscape
New Guidance on the Use of Unproven Neurologic Therapeutics
A new position statement outlining guiding principles for navigating the use of unproven therapies for neurologic conditions that lack approval from the FDA or robust scientific evidence has been released by the American Academy of Neurology (AAN). The statement is intended to support informed conversations between clinicians, patients, and policymakers about emerging or unproven treatments. While it includes examples of such therapies, the document stops short of offering clinical recommendations. The use of emerging neurologic therapies not yet supported by science has been 'an ongoing concern,' with patients often asking providers about these therapies, the statement's lead author Larry B. Goldstein, MD, professor and chair of the Department of Neurology and associate dean for Clinical Research, University of Kentucky, Lexington, Kentucky, told Medscape Medical News. Developed in response to requests from AAN members seeking guidance on emerging therapeutics, the statement outlines guiding principles to support clinical and policy discussions. It was published online on June 25 in Neurology. Lack of FDA-Approved Options Many neurologic diseases have no available FDA-approved therapeutic options and no, or limited, evidence-based treatments. However, many potential treatments, both synthetic and naturally occurring, may be in varying stages of expert evaluation for neurologic conditions. Psychedelics have emerged as potential therapies for pain disorders such as cluster headache, as well as psychiatric conditions including major depression, posttraumatic stress disorder (PTSD), generalized anxiety, and substance use disorders. However, data on psychedelics' risks and benefits for these indications are limited. The AAN committee cites the example of midomafetamine combined with psychotherapy for the treatment of PTSD. Although this approach has generated interest, an FDA advisory committee recently recommended against its approval, citing significant methodological flaws in the supporting studies and advising the FDA to reject the Investigational New Drug Application. 'This illustrative example highlights the need for rigorous, placebo-controlled, double-blind trials to adequately test the effectiveness of these therapies while exhaustively documenting adverse events to characterize patient safety issues,' the statement's authors noted. The AAN supports the FDA-accelerated review of novel therapeutics for neurologic conditions 'when this process is appropriate,' said Goldstein. Off-Label Use The statement also addresses the expanded use of therapies beyond their original FDA-approved indications — for example, the off-label use of alteplase for thrombolysis in ischemic stroke patients treated 3-4.5 hours after they were last known to be well, as well as the use of tenecteplase in select cases of acute ischemic stroke. Goldstein noted that these examples reflect issues commonly encountered in hospitals nationwide. 'The AAN supports the off-label use of FDA-approved therapies in settings in which the high-quality evidence indicates that the benefit of the therapy outweighs the risks with shared decision-making between the patient and physician in the model of informed consent,' the statement authors noted. Another example is natalizumab, a humanized IgG4κ monoclonal antibody approved for the treatment of relapsing-remitting multiple sclerosis (MS). The drug was initially withdrawn from the market following reports of progressive multifocal leukoencephalopathy. However, it was later reintroduced, as some patients with MS have no alternative therapies to effectively manage their disease. Physician-patient discussions about unproven therapies might include situations where patients are considering their 'Right to Try.' Signed into law in 2018, the Right to Try Act allows individuals with a terminal illness who have tried all approved treatments and who are unable to participate in a clinical trial to receive an experimental treatment. The statement also addresses the issue of adverse events reported after FDA approval. For example, the statin cerivastatin was approved for cholesterol reduction but was withdrawn from clinical use following reports of deaths and hospitalizations. These cases, said Goldstein, illustrate the complexity of this issue. 'It's particularly challenging because healthcare providers must be constantly aware of new data that may become available as therapeutics enter general use after FDA approval,' said Goldstein. In cases where a therapy is approved but carries significant risks or an incomplete adverse event profile, the statement advises that the AAN should generally refrain from taking a definitive position until further review by the FDA is completed. Unproven 'Treatments' The authors also address the use of therapeutics with limited or no supporting data, many of which have been popularized on social media, to treat or prevent conditions such as dementia. Such use not only exposes patients to unknown risks but may also discourage them from pursuing evidence-based treatments or participating in clinical trials that could offer potential benefits, Goldstein noted. When seeing patients, healthcare providers should discuss potential participation in a relevant trial and ask more detailed questions about the use of unproven therapies, he said. 'Physicians should routinely not only confirm their patient's prescribed medications but also ask about any other substances they may be using. Some, including certain supplements, may have potential toxicities or interactions with prescribed medications.' Discussions between neurologists and patients about unproven therapies are becoming increasingly relevant. 'In the current climate of unfiltered, at times incorrect or misinterpreted information, having a trustworthy source of fact-based advice is critically important,' said Goldstein. 'The neurologist brings particular expertise and training related to neurological disorders and what is known about the risks and benefits of potential treatments to help inform patient decisions,' he added. The AAN policy statement offers 'a framework' to guide neurologists in their role as patient advocates, Goldstein added. Although it does not address specific treatments, it does provide a structure for conversations with patients, said Goldstein.
Yahoo
28-02-2025
- Business
- Yahoo
Psychedelic: GH Research, Compass Pathways report earnings results
In this week's 'Psychedelic,' The Fly's recurring series focused on psychedelic stock news, The Fly looks back on earnings, an analyst initiation and patent issuance. See what stocks are receiving Strong Buy ratings from top-rated analysts. Filter, analyze, and streamline your search for investment opportunities with TipRanks' Stock Screener. EARNINGS: On Thursday, GH Research (GHRS) reported a FY24 loss per share of (75c), which compared to analyst estimates of loss per share of (80c). The company also reported cash, cash equivalents, other financial assets and marketable securities at $182.6M as of December 31, compared to cash, cash equivalents, other financial assets and marketable securities of $222.7M as of December 31, 2023. The company said, 'As previously announced, our Investigational New Drug Application for GH001 administered using our proprietary aerosol delivery device has been placed on clinical hold by the U.S. Food and Drug Administration, with the FDA requesting that we provide an inhalation toxicology study in a non-rodent species and an additional inhalation toxicology study in rats, additional device design verification information and updates to our investigator brochure, to resolve the hold. We have recently announced the completion of all FDA requests to address IND hold. The requested additional inhalation toxicology study in a non-rodent species has now been completed. The pathology report concludes that there are no histology findings in the respiratory tract of any dogs at any dose level evaluated in the study. The requested additional inhalation toxicology study in rats has now been completed which showed histology findings consistent with our previously completed study in rats. This supports our position that these findings are rat specific. Based on previously announced FDA interactions, the response to their request for additional device design verification information is being prepared and, together with the completion of the inhalation toxicology studies, provides the final piece of information requested by the agency. We are preparing to engage with the FDA in advance of providing a full response to the IND hold which we are on track to submit in mid-2025.' Additionally on Thursday, Compass Pathways (CMPS) reported a fourth quarter loss per share of (63c), which compared to consensus of a loss per share of (62c). The company also reported cash and cash equivalents were $165.1M as of December 31, compared with $220.2M as of December 31, 2023. The company said FY25 net cash used in operating activities is expected to be in the range of $120M to $145M and the cash position at February 27 is expected to be sufficient to fund operating expenses and capital expenditure requirements at least through the planned 26-week data read-out from the COMP006 study, which is expected in 2H26. 'We are excited that the first data readout from our pivotal phase 3 COMP360 program in treatment resistant depression continues on track with top-line 6-week data expected next quarter,' said Kabir Nath, CEO. 'Building on last year's promising phase 2a data in PTSD and the resources now available from the January financing, we are advancing plans to develop a late-stage clinical program for PTSD with the goal of getting COMP360 to patients as quickly as possible. The need for a new approach to treatment remains significant in TRD and PTSD and we believe COMP360 represents a novel treatment option in these mental health conditions.' COMPASS PATHWAYS INITIATED WITH BUY: Stifel initiated coverage of Compass Pathways on Wednesday with a Buy rating and $11 price target. The firm sees 'major stakeholders,' including thought leaders and physicians coalescing around a view that psychedelics are likely to become a major drug class within neuropsychiatry and it believes the company is well positioned over the next 18 months to derisk COMP360 psilocybin across two Phase 3 studies. Shares trading 'well below' the IPO price offer 'an attractive opportunity' ahead of upcoming catalysts, the analyst said. QUANTUM BIOPHARMA COMPLETES PHASE 1 LUCID-21-302 TRIAL: Quantum BioPharma (QNTM) announced Wednesday that it has completed its trial entitled 'A Phase 1, Randomised, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate the Safety and Pharmacokinetics of Lucid-21-302 in Healthy Adult Participants.' A final safety review committee meeting was held after completion of the trial. The SRC found that Lucid-21-302 was well-tolerated with no safety concerns and no serious adverse events were reported during the trial. Lucid-MS is a non-immunomodulatory, neuroprotective compound for the treatment of multiple sclerosis. It is a patented new chemical entity that has a unique mechanism of action. As shown in preclinical models of MS, it can directly stabilize the myelin sheath surrounding nerve fibers and thus provide protection from demyelination. Zeeshan Saeed, CEO, said, 'We are excited about potential of Lucid-MS to protect myelin in MS patients as it represents a new direction in the treatment of this disease. By completing this trial and demonstrating safety in healthy participants, we are now closer to initiating a Phase 2 trial of Lucid-MS in people with MS. We are now looking ahead to our Phase 2 trial as we work towards our goals of drug approval and commercialization of Lucid-MS. We look forward to providing further updates as we execute on our milestones, driven by our mission to arrest demyelination in MS.' ENVERIC UNVEILS EVM401 SERIES OF COMPOUNDS: Enveric Biosciences (ENVB) announced Tuesday that the United States Patent and Trademark Office issued U.S. Patent No. 12,195,439 on January 14. Entitled 'Cl-Substituted Isopropylamine Fused Heterocyclic Mescaline Derivatives,' the '439 patent is the first Enveric patent to be issued for its new EVM401 Series. Further development of the EVM401 Series of compounds is intended to broaden Enveric's pipeline with additional non-hallucinogenic molecules and strengthen its ability to target addiction and neuropsychiatric disorders for patients with limited options. Preliminary testing of the EVM401 series of compounds and their metabolites has demonstrated unique patterns of brain receptor modulation emerging from the compounds' distinct molecular structures. Specifically, preliminary data suggest that these mescaline derivative compounds may interact with a variety of important neurological receptors, including the α2A adrenergic receptor, the 5-HT2C receptor, and the dopamine transporter. ADRA2A agonists are known to help manage the symptoms of opioid withdrawal and treat conditions such as ADHD, while the 5-HT2C receptor is an emerging therapeutic target in the treatment of neuropsychiatric diseases, including substance use disorders, anxiety and depression. Notably, DAT is also an important target in the treatment of substance use disorders. Additionally on Thursday, Enveric CEO Joseph Tucker issued a letter to shareholders, which read in part, 'This is a very exciting time in the growth and evolution of Enveric. We have successfully completed a strategic reshaping of the company to focus our business and development operations towards the advancement of EB-003 as our lead product. We believe EB-003 offers Enveric the greatest opportunity to drive shareholder value given its potential to be a first-in-class neuroplastogenic molecule that could address multiple, difficult-to-treat neuropsychiatric indications. We additionally recently unveiled an exciting new series of drug candidates in our pipeline, the EVM401 Series… As 2025 is underway, we believe the Company is well positioned to cement its leadership role in the industry by advancing the development of our innovative drug candidate, EB-003, for the treatment of neuropsychiatric conditions. We eagerly look forward to advancing toward clinical trials and ultimately receiving regulatory approval, so we can bring EB-003, and later our EVM401 Series candidates, to the market in support of our twin goals of improving the lives of patients and generating value for our shareholders.' SILO GRANTED PATENT FOR PTSD TREATMENT: Silo Pharma (SILO) announced Wednesday that the U.S. Patent and Trademark Office issued an Issue Notification for the previously filed patent application titled 'Pharmacological Prophylactics Against Stress-Induced Affective Disorders in Females.' The projected patent number is 12,239,614 and the issue date of such patent will be March 4. The patent reinforces protection for Silo's lead asset, SPC-15, an intranasal treatment targeting post-traumatic stress disorder. The patent includes newly added claims to U.S. Patent 11,491,120, which was issued to Silo in November 2022. 'Strategic IP and patent expansion is key to advancing our lead asset SPC-15 to clinical trials and creating value for our full pipeline of novel assets,' said Eric Weisblum, CEO. 'This new patent further supports our collaborative research with Columbia University and expands coverage for SPC-15.' OTHER PSYCHEDELIC STOCKS: Publicly-traded companies in the space include Algernon Pharmaceuticals (AGNPF), Allied Corp. (ALID), atai Life Sciences (ATAI), BetterLife (BETRF), Bright Minds Biosciences (DRUG), Clearmind (CMND), Cybin (CYBN), Entheon Biomedical (ENTBF), Filament Health (FLHLF), Incannex Healthcare (IXHL), Mind Medicine (MNMD), MIRA Pharmaceuticals (MIRA), Mydecine Innovations (MYCOF), NRx Pharmaceuticals (NRXP), Numinus Wellness (NUMIF), Optimi Health (OPTHF), Pasithea Therapeutics (KTTA), PharmAla Biotech (MDXXF), PharmaTher (PHRRF), Psyence Biomedical (PBM), Psyence Group (PSYGF), Relmada Therapeutics (RLMD), Revive Therapeutics (RVVTF), SciSparc (SPRC), Seelos Therapeutics (SEEL), Silo Pharma (SILO) and Synaptogenix (SNPX). Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See today's best-performing stocks on TipRanks >> Read More on GHRS: GH Research Reports 2024 Financial Results and Clinical Progress GH Research reports FY24 EPS (75c), consensus (80c) Psychedelic: Exclusive talk with biotech company Enveric Biosciences GH Research initiated with an Overweight at Cantor Fitzgerald GH Research PLC Raises $139.8 Million Through Share Offering Sign in to access your portfolio
