Rapport Therapeutics, Inc.

Alto Neuroscience, a young biotechnology company developing medicines for the brain, lost much of its value Wednesday as negative results from a clinical trial raised doubts about the future of its most advanced research program.That program revolves around an experimental drug code-named ALTO-100, which Alto is testing against different kinds of depression. The new results, from a trial with just over 300 participants, show the drug was not significantly better than a placebo at improving symptoms of the most common type, known as major depression.Alto plans to analyze all the data from the study to determine the most appropriate next steps, if any, for ALTO-100 in major depressive disorder. Researchers are also evaluating the drug in mid-stage studies focused on post-traumatic stress disorder and bipolar depression, with high-level results in that latter indication expected in 2026.While the latest findings didnt align with the more positive data seen in earlier trials, we believe our approach to collecting and stratifying based on biomarkers represents an innovative approach to developing neuropsychiatric drugs, said Adam Savitz, Altos chief medical officer, in a statement Tuesday evening.Investors, though, may have lost some faith in Alto. The biotechs share price had sunk by two-thirds by Wednesday morning, to trade below $5.Founded in 2019 by current CEO Amit Etkin, Alto is attempting to make more precise brain drugs. Its a mission shared by other freshly launched startups like Nido Biosciences and Rapport Therapeutics, and one that has received sizable investments over the past few years. Neumora Therapeutics, for instance, raised $250 million late last summer through an initial public offering. And before that, it attracted more than $600 million from a lengthy list of investors that included Amgen and Arch Venture Partners.At Alto, the company aims to identify biological markers that can be used to design better brain drugs. One such biomarker, a verbal memory test, was used to enroll in the major depression study only participants with poor cognition who were most likely to respond to ALTO-100.To analysts, the studys failure, while disappointing, isnt a total indictment of Altos method of precision psychiatry drugmaking. Myles Minter, from the investment firm William Blair, wrote in a note to clients that the setback will surely cast investor doubt. Even so, we do not believe a single failure of the approach is indicative that the biomarker enrichment strategy is invalid.Ultimately our view is that the idea behind Alto Neuroscience still has a lot of merit, wrote Stifel analyst Paul Matties in his own note, adding that the companys other shots-on-goal for fundamentally different assets should not be written off.To be clear, we think other programs here are higher-risk, Matteis wrote. But from a company concept perspective, we still think what Alto is trying to do is highly logical and the failure of ALTO-100 shouldn't undermine this.Alto says it has enough cash to operate into 2027, by which point it will have data from other important studies, including two that are testing a couple of the companys other drugs. Data from those experiments should come in the first half of next year. The company reported having about $194 million worth of cash and cash equivalents as of June 30.The ALT-100 candidate is meant to enhance a protein that not only triggers the growth of new brain cells, but also regulates how those cells respond to stimulation. Alto thinks this effect could help people with depression, as research indicates that depressive disorders can actually shrink the brain especially parts like the hippocampus, which plays a vital role in learning, mood and memory. '

CAMBRIDGE, Mass.--( BUSINESS WIRE )--GC Therapeutics, Inc. (GCTx), announced its launch today to scale and unlock the next generation of cell therapy. Invented and developed by a world-class team of scientists in the lab of professor George Church, Ph.D., at Harvard Medical School and the Wyss Institute, GCTx’s induced pluripotent stem cell (iPSC) programming platform, TFome™ (Transcription-Factor-ome, pronounced as tee-eff-ome), integrates the latest advancements in synthetic biology, gene editing, cell engineering and machine learning to overcome the development and scaling complexities associated with cell therapy and improve patient access across a broad range of disease areas. GCTx completed a $65 million Series A financing led by Cormorant Asset Management with participation from Mubadala Capital, Andreessen Horowitz (a16z) Bio + Health, Medical Excellence Capital, Cercano Management and Pear VC, bringing total capital raised by GCTx to $75 million. “ Cell therapies have revolutionized the treatment of certain disease areas; however, challenges in scaling these therapies and generating high quality products limit their full potential and patient accessibility,” said Parastoo Khoshakhlagh, Ph.D., co-founder and chief executive officer of GCTx. “ Our novel cell programming approach enables the rapid and efficient production of fully optimized iPSC-based cell therapies through a streamlined, single-step process. This method enhances potency, efficiency, quality and cost-effectiveness compared to conventional approaches. We are excited to launch GCTx to broaden the accessibility of cell therapy and advance our pipeline of potential best-in-class treatments in various therapeutic areas starting with gastrointestinal, neurological and immunological diseases.” “ GCTx’s cell programming platform represents a major scientific advancement,” said George Church, Ph.D., professor of genetics at Harvard Medical School, core faculty member and synthetic biology lead at the Wyss Institute, and co-founder and senior advisor of GCTx. “ This approach harnesses the transformative potential of transcription factor biology to guide iPSC cell fate into any differentiated cell type in a single, seamless step. By transcending the limitations of natural processes, it holds the promise to create cells that surpass the capabilities of those found in nature, generating a groundbreaking new class of SuperCell™ medicines to address diverse disease areas and reduce healthcare burdens.” TFome: The world’s first “plug-and-play” iPSC cellular programming platform iPSC differentiation into distinct cell types depends on various signaling pathways that ultimately converge onto transcription factors (TFs), which control the sets of genes that are active in a cell. Cell fate is determined by specific TFs acting in concert. GCTx has built the world’s first complete collection of human TFs. Through sophisticated multidimensional screening modalities that integrate genome-scale experimental testing and powerful next-generation machine learning, TFome can identify fully optimized TF combinations to differentiate iPSCs into virtually any functional cell type. This allows for a single-step, four-day stem cell differentiation process with >90% efficiency with no requirement for cell type-specific optimization for microenvironmental factors. This has not been achieved previously via conventional small-scale testing. Through TF-based cell fate programming, TFome is designed to yield first-in-class and best-in-class off-the-shelf cell therapy products up to 100 times more rapidly than conventional methods of cell differentiation with improved potency, efficiency, quality and cost (SuperCells™). GCTx is positioned to be the first company in the iPSC-based cell therapy space to pursue multiple cell products from one starting point using a similar manufacturing strategy for more accelerated development and timelines. “ Unlike conventional iPSC approaches, TFome goes beyond traditional developmental biology by pushing the limits of the principle that cell identity is determined by the active gene regulatory networks governed by TFs. This approach enables a more direct and precise control over cell fate, thereby setting a new standard in cell programming,” said Alex Ng, Ph.D., co-founder and chief scientific officer of GCTx. “ We have validated our single-step cell fate programming approach across diverse cell types both in vitro and in vivo and have further engineered cells into SuperCells to tailor them for specific disease applications. This programming-centric paradigm achieves scalability, cost-effectiveness and streamlined clinical development while maintaining quality, safety and efficacy.” Originating from professor Church’s lab, Harvard’s Blavatnik Biomedical Accelerator (BBA) supported the project to advance the research toward commercialization opportunities. Harvard’s Office of Technology Development (OTD) protected the intellectual property associated with the founding research and licensed the technology exclusively to GCTx for further development. Building a leading, fully integrated cell therapy company with multiple pipeline programs advancing toward the clinic GCTx is committed to leading the field with first-in-class and best-in-class off-the-shelf cell therapies targeting gastrointestinal, neurological and immunological diseases. The versatility and efficiency of TFome also enables GCTx to broaden its reach beyond its core pipeline areas, unlocking strategic business development opportunities and harnessing the expansive capabilities of the company’s portfolio. “ GC Therapeutics’ groundbreaking approach to cell therapy has the potential to redefine the industry. By accelerating the production of iPSC-based therapies, the TFome platform unlocks unprecedented efficiency and scalability, which could lead to faster patient access to life-changing treatments. We are proud to partner with the GCTx team as they push the boundaries of what is possible in the development of next-generation cell therapies,” said Alaa Halawa, executive director and head of US Ventures at Mubadala Capital. Assembled world-class team of thought leaders in biopharma industry GCTx co-founders Parastoo Khoshakhlagh, Ph.D., chief executive officer Alex Ng, Ph.D., chief scientific officer Cory Smith, Ph.D., scientific co-founder and head of discovery platforms George Church, Ph.D., professor of genetics at Harvard Medical School and core faculty member and synthetic biology lead at the Wyss Institute GCTx board of directors and advisers Ray Kelleher, M.D., Ph.D., managing director, Cormorant Asset Management Vineeta Agarwala, M.D., Ph.D., general partner, a16z Bio + Health Alaa Halawa, MBA, executive director and head of U.S. ventures, Mubadala Capital John Maraganore, Ph.D., CEO and principal, JMM Innovations, LLC, founding CEO, Alnylam Pharmaceuticals, Inc. Steve Paul, M.D., former CEO, Karuna Therapeutics, former EVP, Eli Lilly and Company, co-founder and board chair, Rapport Therapeutics and Seaport Therapeutics “ GCTx’s TFome platform represents one of the most innovative approaches in the cell therapy space and has the potential to yield a revolutionary class of new SuperCell medicines that are tailored for specific patients and diseases,” said Ray Kelleher, M.D., Ph.D., managing director, Cormorant Asset Management. “ We are thrilled to support this exceptional team of scientists and company builders as we work to create the industry’s next leading cell therapy company.” About GC Therapeutics GC Therapeutics (GCTx) is a biopharmaceutical company created to scale and unlock the next generation of cell therapy-based medicines. GCTx’s induced pluripotent stem cell (iPSC) programming platform, TFome™ (tee-eff-ome), integrates the latest advancements in synthetic biology, gene editing, cell engineering and machine learning to overcome the therapeutic development and scaling complexities associated with cell therapy and improve patient access across a broad range of disease areas. GCTx’s initial focus is on developing first-in-class and best-in-class cell therapies for gastrointestinal, neurological and immunological diseases. Founded in 2019, GCTx is based in Cambridge, MA and has raised $75 million to date from leading life sciences investors. For more information, please visit us at www.gc-tx.com and follow us on LinkedIn and X .

In what’s shaping up to be another slow year for biotech IPOs — better, however, than the 2022 and 2023 doldrums — Jim Healy is somewhat of an outlier. Only four biotechs from this year’s IPO class are trading above their debut pricings: CG Oncology, ArriVent BioPharma, Rapport Therapeutics and Contineum Therapeutics. Healy is on the boards of two of those: cancer drugmaker ArriVent, which is in late-stage testing with a drug out of a China-based partner; and neuroscience startup Rapport, which spun out of Johnson & Johnson and quickly raised successive megarounds before landing on the Nasdaq. The Sofinnova Investments managing partner, a self-described venture capitalist of about 25 years, is also on the board of BioAge Labs, an anti-aging and clinical-stage obesity startup that is also in the process of an IPO. He’s also on the Seaport Therapeutics board, alongside many other Karuna Therapeutics veterans, and sees potential for a quick run to the public markets at that neuropsychiatric biotech as well. “I’ve never really seen the markets close for high-quality companies that have good clinical data and a good development plan that they can execute,” Healy said in an interview on Wednesday. BioAge is one of four biotechs lined up for Wall Street entries in the coming days and weeks, including Bicara Therapeutics , Zenas BioPharma and MBX Biosciences . The market reaction to their debuts could set the tone on investor appetite for the backlog of biotechs looking for their shining moment in New York. “This current market feels like it’s much more selective in terms of the profile of companies that go public,” Healy said. All but one of this year’s class was clinical-stage when it rang the opening bell, he noted. “And if I look over a longer time frame, clinical-stage companies have outperformed their preclinical peers significantly over the past eight to nine years, so it feels like it’s a migration to quality.” For biotech companies to go public in this environment, Healy said it’s important they hone in on a lead asset, which is where private and public investors will place “a majority of the value.” “You can get incremental benefit for a second or third indication, or a second drug in the pipeline and the potential of a platform, but the vast majority of the value is assigned to the lead program or lead product,” he said. Biotech startups have reeled in large financings this year to ensure they get clinical data on their lead programs, rather than raise multiple smaller rounds at various checkpoints prior to the big readout. That has led the industry to go on a tear with megarounds this year, with F2G’s $100 million Series H Thursday morning marking the 71st such round of 2024, according to an Endpoints News tally. Access to capital is a big risk in biotech, Healy said, and investors want to decrease the refinancing risk, so they’ll raise larger rounds now with crossover investors who can help carry them to a public offering. One of the megarounds Sofinnova participated in this year, neuropsychiatric biotech Seaport, could be one such example. The clinical-stage startup derives from PureTech Health, the company that also launched Karuna, a schizophrenia drugmaker that Bristol Myers Squibb acquired for $14 billion. “If I look over our current funds, over the past two funds in particular or past five years, our average time to IPO is less than a year,” Healy said. “I would hope that Seaport would fit within our investment [strategy], and it would be outstanding if it could follow the trajectory that Karuna had. But of course, no guarantees.” Biotechs shouldn’t get over their skis. Median pre-money valuations have been about $475 million to $500 million this year and last. “I think companies that are doing private financings that put them well north of that create a challenge for themselves,” Healy said. “If you look at the current class of IPOs that have gone public, most have a step-up from their last private financing of around 1.2x, so one needs to consider that in the valuation as well.” Healy said the current market environment feels “much more rational.” “There’s a correlation between the stage of development and the enterprise value of companies,” he said.