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shiota/ iStock Researchers in pharma and beyond have historically glommed onto a limited number of disease targets, limiting innovation. AI could change that. Proteins that fight tumor growth, gene exons that can be modified, and receptors on immune cells that control their cancer-killing powers. These are the types of mechanisms and bodily processes that could be key to curing or treating disease, and which form the targets of modern drug development. While the number of such targets is nearly infinite, a relatively small number have emerged as the most popular among pharmaceutical companies. In a phenomenon termed “herding,” numerous pharmaceutical firms, research organizations and even academic scientists are targeting not just the same narrow band of conditions, but the same limited set of drug targets. In 2020, 68% of targets pursued by the top 10 pharmaceutical firms were the basis of five or more R&D programs. Some firms were dedicating 80% or more of their research time and dollars to those popular targets. Chief among these targets are those associated with cancer; in 2022, there were nine assets per actively investigated oncology target among the top pharma firms, up from 1.8 in 2000, with most of the research dedicated to breast, hematological and brain cancer. While there are understandable reasons for herding and even advantages in certain situations, the overall effect of this “me-too” style of innovation is to set limits in drug development. But how can we break away from the crowd and forge our own path? As vice president of business development at Evogene, which offers an AI-driven computational chemistry platform, I believe artificial intelligence is the answer. AI has the power to transform drug development by identifying novel targets and compounds that can interact with them. The Drawbacks of Herding Well-known examples of “me-too” drugs include many of the selective serotonin reuptake inhibitors (SSRIs) used in treating depression—such as Prozac, Zoloft and Paxil—and various statins—think Lipitor and Crestor. One of the most comprehensive studies of drug targets curated 1,578 FDA-approved drugs acting on 893 unique molecular targets, meaning nearly 50% of FDA-approved drugs share common targets. Similarly, about 60% of drugs on the World Health Organization’s list of essential treatments fit into this “me-too” category, and more than 50% of the 50 novel drugs approved by the FDA in 2024 relied on pre-existing targets. In many ways, herding makes sense, and it has benefitted some patients and pharma companies. Many of the targets that experience herding, especially in the oncology space, are highly efficacious, and pharma firms want to follow what works and, ideally, create best-in-class treatments in these drug classes. Of course, there is a great demand—and a large need—for these improvements. In addition, when it comes to single-gene diseases such as thalassemia, Duchenne muscular dystrophy and Huntington’s, herding has emerged alongside progress in developing gene therapies. For example, instead of focusing solely on the mutant gene, companies might look at targeting cellular pathways disrupted by the mutant protein. Such approaches make up only 10%–30% of attempts to develop new therapies, while the majority still focus on the mutant gene itself. Such concentrated research efforts, exemplified by the focus on the CFTR gene in cystic fibrosis leading to therapies like Ivacaftor, can indeed accelerate progress in understanding and treating rare genetic diseases. But the focus on the gene itself and a small set of cellular pathways leaves other pathways under explored. This over-concentration diverts critical resources, exacerbating challenges in resource allocation and clinical trial design and ultimately hindering efforts to pursue novel targets and more innovative therapies. This is especially problematic for rare diseases, where the pool of trial participants is naturally low and standards limit patients to one trial at a time. If all or most of the patients are enrolled in trials testing drugs with the same target or few targets, there is little room to open a trial with a new target, further discouraging innovation. Innovation Through AI Pursuing new targets and therapies is essential to reversing the herding trend, and one of the tools for doing this is AI. AI offers the ability to navigate the vast chemical and biological landscape, uncovering hidden patterns and relationships that traditional methods overlook. By integrating data from genomics, proteomics and cheminformatics, AI can identify novel targets that have been underexplored, evaluating their potential with remarkable speed and accuracy. Over the past three years the market for AI platforms for the entire healthcare industry has tripled . As more companies— and regulators —embrace AI-based research, companies will be better equipped to break free from the constraints of herding and the redundancy of structurally similar drugs, shifting toward more innovative and diverse therapeutic strategies. McKinsey predicts that AI could increase the number of actionable drug targets by at least 30% . There are already signs of progress in de-herding as novel biological targets and molecules for drug development emerge. For example, AstraZeneca, in collaboration with BenevolentAI, has identified novel targets in chronic kidney disease (CKD) beyond the well-known renin-angiotensin system, opening pathways to treatments focused on kidney fibrosis and inflammation. AI-based research by Verve Therapeutics, meanwhile, has shifted from traditional statin targets to gene editing therapies for inherited cardiovascular conditions. And for rare diseases, including amyotrophic lateral sclerosis (ALS) and Duchenne muscular dystrophy, companies like Insilico have utilized AI-driven systems to identify specific genetic targets. In addition to finding new targets, deep learning enables the de novo design of novel ligands, the active molecules that are the basis of many potential new drugs. Generative architectures such as GANs (generative adversarial networks), VAEs (variational autoencoders) and GPTs (generative pre-trained models) allow researchers to create unique molecular structures that are specifically tailored to their targets. This capability is transformative because it avoids the redundancy of relying on existing compound libraries, which are often biased toward known targets. AI-driven de novo ligand generation explores new chemical spaces, ensuring structural diversity and reducing the risk of producing yet another version of an existing drug. Companies like Atomwise , Insilico and Recursion are using AI for structure-based drug design, and have discovered unique ligands for various indications. Evogene recently began to leverage its AI-driven computational chemistry platform, first developed for agricultural applications, to break into the world of novel ligand discovery and drug development. Despite its promise, AI-driven drug discovery faces challenges such as variable data quality and the “black box” nature of some models, which can complicate validation and regulatory approval. While the promise of AI-first drugs is yet to be fully realized—none have progressed beyond Phase II clinical trials due to long development timelines—it’s important to note that the most advanced drugs in the pipeline were created using technology that has since matured rapidly. These issues are recognized as part of an evolving field, with ongoing innovation addressing concerns like data standardization and model transparency. I have strong confidence that these hurdles will be overcome in time, ultimately enabling AI to revolutionize drug development and improve patient outcomes. AI use in the pharmaceutical industry is far from universal, but by collaborating with AI-first companies, pharmas can remain at the forefront of technological advancements, ensuring their pipelines are fueled by cutting-edge breakthroughs rather than incremental advances. As AI-driven research becomes the norm, fuelled by these strategic collaborations, we can anticipate transformative progress that will benefit pharmaceutical companies and patients alike.

Primary endpoint met, with TSND-201 demonstrating statistically significant placebo-adjusted CAPS-5 improvement of -9.64 points on Day 64 (p = 0.011) TSND-201 demonstrated rapid improvements, with a statistically significant placebo-adjusted CAPS-5 improvement of -8.00 points on Day 10 (p = 0.012), with placebo-adjusted improvements remaining statistically significantly for the duration of the trial Robust and statistically significant improvements were noted across multiple secondary endpoints on Day 64 TSND-201 was generally safe and well tolerated, with a safety profile consistent with its non-hallucinogenic pharmacology Company plans to align with FDA on Phase 3 program for PTSD NEW YORK, March 31, 2025 /PRNewswire/ -- Transcend Therapeutics, a clinical-stage biotechnology company that develops medicines to treat neuropsychiatric diseases, today announced positive results from the IMPACT-1 study (Investigation of Methylone for Post-Traumatic Stress Disorder [PTSD]). IMPACT-1 was a randomized, double-blind, placebo-controlled, Phase 2 clinical trial evaluating TSND-201 (methylone), a rapid-acting neuroplastogen, for the treatment of PTSD. This study enrolled 65 patients with severe PTSD (Clinician-Administered PTSD Scale for DSM-5 [CAPS-5] ≥ 35), all of whom had previously tried a PTSD treatment, to receive 4 oral doses of TSND-201 or placebo, with each dose separated by one week. Patients were followed for a total of 64 days after the first dose. Continue Reading CAPS-5 Scores Over Time; * p <0.05 On the primary endpoint, change from baseline to Day 64, TSND-201 demonstrated a statistically significant placebo-adjusted CAPS-5 improvement of -9.64 points on Day 64 (-23.28 points vs. -13.64 points; p = 0.011). TSND-201 also demonstrated rapid and statistically significant improvements from baseline compared to placebo on the CAPS-5 total severity score. Starting on Day 10, TSND-201 demonstrated a statistically significant placebo-adjusted CAPS-5 improvement of -8.00 points (-17.83 points vs. -9.83 points; p = 0.012), which was durable throughout the study until Day 64. "We are encouraged by the positive Phase 2 results demonstrating TSND-201's potential as a rapid-acting and durable treatment for patients with PTSD," said Transcend Therapeutics Co-Founder and CEO Blake Mandell. "We look forward to working with the FDA to align on a Phase 3 program in PTSD." TSND-201 demonstrated statistically significant improvements on multiple secondary endpoints, including response (≥ 50% improvement from baseline on CAPS-5; 57.1% vs. 19.2%; p = 0.002), remission (≤ 11 points on the CAPS-5; 32.1% vs. 11.5%; p = 0.036), and loss of PTSD diagnosis (60.7% vs. 30.8%; p = 0.014). Treatment with TSND-201 was generally safe and well tolerated. The majority of adverse events were transient, occurring on the day of dosing and resolving the same day. The most commonly occurring adverse events in the TSND-201 group were headache, decreased appetite, nausea, dizziness, blood pressure increase, dry mouth, insomnia, muscle tightness, and feeling abnormal. One serious adverse event of seizure occurred in the TSND-201 group; the event occurred 7 days after the last dose in a patient with a history of seizure and was considered unrelated to study drug. "It has been over two decades without a new treatment for PTSD. The results of this study suggest that TSND-201 may represent a significant clinical breakthrough — demonstrating clinically meaningful efficacy with a rapid onset," commented Murray B. Stein, M.D., M.P.H., Distinguished Professor of Psychiatry and Public Health at the University of California San Diego and Staff Psychiatrist at the Veteran Affairs San Diego Healthcare System. "Rapid-acting, episodic treatments could be a paradigm shift in the treatment of PTSD, providing hope for the many patients who have struggled to find effective relief." About Transcend Therapeutics Transcend Therapeutics, founded in 2021, is a clinical-stage, neuroscience-focused company developing rapid-acting treatments for neuropsychiatric diseases. The company's mission is to develop new drugs for the millions of people for whom current psychiatric medicines have not worked. Transcend's leadership team has made pivotal contributions to 14 FDA approvals and $7B in M&A and public company value. Transcend has raised approximately $50M to date. About TSND-201 TSND-201, a rapid-acting neuroplastogen, is a proprietary formulation of methylone developed by Transcend Therapeutics. TSND-201 has well-characterized primary pharmacology, with its primary site of action at the monoamine transporters, and no activity at 5HT-2a (i.e., not hallucinogenic). Transcend is developing TSND-201 as a rapid-acting and durable treatment for PTSD and other central nervous system (CNS) indications. TSND-201 is an investigational medication. About PTSD PTSD is a mental health condition that can occur after experiencing or witnessing a life-threatening or traumatic event. Symptoms may include flashbacks, nightmares, severe anxiety, and uncontrollable thoughts about the event. PTSD impacts approximately 13 million Americans each year and it is estimated that nearly 7% of U.S. adults will be diagnosed with PTSD during their lifetime. Only two medications are FDA-approved for the treatment of PTSD (sertraline and paroxetine), yet their efficacy is modest at best. New treatments for PTSD are urgently needed. Contact: [email protected] SOURCE Transcend Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED