#28 - Plummeting stocks, trial flops, & ebola shocks

The coffee break biotech roundup, by SomX.

Happy New Year!

While some of the best biotech startups are cracking medicine’s most challenging puzzles, elsewhere the news is not so jolly. But fear not – a stellar cast of clinical trials might help shock some life back into 2025!

Meanwhile, Sangamo's haemophilia hopes took quite the plunge, clever scientists caught Ebola red-handed playing hide-and-seek in human skin, Neumora's depression drug has stumbled, and Vertex is dropping some serious cash on a Orna's genetic bag of tricks.

Stay resolute, only 50 more weeks to go!

Dodo

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Discover 🔍

💊 Unravelling the most complex disease challenges in history: how AI startups are changing drug discovery (Startups Magazine): Three rather ingenious machine learning ventures are tackling medicine's thorniest puzzles by finessing and utilising human-centric techniques: Ochre Bio is using genomic data and donated livers to decode chronic liver disease; Qureight’s platform reimagines clinical trial data to analyse of disease progression; meanwhile, Antiverse uses generative AI to accelerate antibody discovery with higher predictive accuracy. Together, these pioneers are reshaping drug discovery for the modern age.

Our take: These data pioneers are displaying rather exemplary scientific judgement by prioritising human data over in vitro screens to recalibrate the fundamentals of drug discovery. By anchoring machine models in complex human data, they're addressing a long-standing Achilles heel of drug development: the disconnect between early promise and clinical reality – highlighting a fundamental shift in how pharma is targeting disease mechanisms.

📉 Sangamo’s stock plummets as Pfizer axes haemophilia gene therapy pact (Biospace): Sangamo finds itself in quite the financial pickle, with cash reserves dwindling and Pfizer's termination of their gene therapy partnership costing them a potential £220M in milestone payments. The news caused their shares to take quite the nosedive – down 56% on New Year's Eve. It appears the market for first-gen haemophilia gene therapies isn't quite the goldmine Pfizer imagined...we’ll have to wait and see what happens when the programme returns to Sangamo's hands this April.

Our take: It’s rather telling that Pfizer's pulling out despite positive Phase III data of the drug candidate giroctocogene fitelparvovec. The real story here is how patient preference for Roche’s Hemlibra's predictable efficacy has swept the commercial landscape for haemophilia gene therapy. This withdrawal, combined with BioMarin's tepid Roctavian launch, suggests the field might need a rethink – particularly around durability and pricing strategies.

🔥10 clinical trials to watch in the first half of 2025 (BiopharmaDrive): Amid the sector’s persistent volatility, the year ahead also holds promise. A fresh slate of trial readouts, targeting everything from metabolic disease to cancer and depression, suggests that 2025 could re-energise investors and validate scientific progress. These 10 upcoming trials involve both established players – like Eli Lilly’s Surpass-CVOT and Emerald-2, Novartis’ Lp(a) Horizon, Novo Nordisk’s Evoke – as well as smaller innovators, including Arvinas’ Veritac-2, Vera’s Origin 3, Verve’s Heart-2 and trials from Beam, Compass and Vertex.

Our take: Well, here’s a chance for biotech to break out of its economic doldrums. While 2025’s star-studded lineup might lure back the capital markets, there’s also a subtle litmus test at play: will regulators and payers keep up? Although Dodo’s main concern is that while big biotech’s might thrive, smaller biotechs on a shoestring could flounder if things don’t pick up. There’s no room for lacklustre results.

🦠 Ebola virus translocation model may clarify human skin transmission (GEN):
Researchers from the University of Iowa Health Care, along with chums at Texas Biomedical Research Institute and Boston University, have mapped out Ebola's crafty route through human skin. Using rather clever human skin explants that preserve both dermal and epidermal layers, they've shown the skin is be an active player in viral transmission: the virus, it seems, first infiltrates myeloid cells, endothelial cells, and fibroblasts in the lower dermis before sneaking into the epidermis through keratinocytes.

Our take: Beyond the immediate implications for outbreak protocols, this work rather brilliantly showcases the value of human tissue models in infectious disease research. The explant system's ability to maintain complex cellular architecture offers a splendid middle ground between oversimplified cell cultures and costly animal studies. A potential game-changer for therapeutic screening – particularly for cash-conscious biotechs.

🧠 Neumora's major depression drug flunks first of three Phase 3 trials (Endpoints): Navacaprant, a novel kappa opioid receptor antagonist that targets reward and dopamine pathways, has failed to distinguish itself from placebo in its KOASTAL-1 trial. Bad news for Neumora, who has teased multi-billion sales if the drug can move the needle on patient outcomes. With two more pivotal readouts (KOASTAL-2 and 3) penned for the first half of 2025, here’s hoping we can report more positive data soon.

Our take: With depression remaining a leading cause of disability – and treatments inadequate for many – pharma is casting about for new targets in this massive commercial market. Neumora is playing in the same mechanistic sandbox as J&J's aticaprant, which is also running Phase 3 studies for its major depressive disorder drug, and several novel products in mental health (including intranasal esketamine) have faced both clinical and reimbursement hurdles. The bar is high, and if navacaprant is viable, it could be a genuine alternative to the glut of SSRIs on the market.

And finally…

💸 Vertex pays Orna $65M to work on next-gen gene therapies for blood disorders (Investing News Network): Vertex is fortifying its blood disorder portfolio through a rather elegant partnership with Orna Therapeutics and its ReNAgade platform, which Orna acquired in May ‘24. Their lipid nanoparticles are set to advance gene editing therapies for sickle cell disease and transfusion-dependent beta-thalassemia. Beyond the initial $65M, Orna stands to collect up to $635M in milestones, with scope for an additional $365M per product across 10 potential indications.

Our take: The technological convergence here is rather brilliant. Orna's circular RNA platform offers extended stability and reduced immunogenicity compared to conventional mRNA, while their lipid nanoparticles are engineered for precise delivery to parts of the body, such as directly to the bone marrow, which opens the door to treatment for genetic diseases. This could render obsolete the complex conditioning regimens currently required, with profound implications for manufacturing costs and patient access.

Tune in

📊 Looking at 2025 Biotech Trends with SoftBank Vision Fund
Explore how funding strategies, AI applications, and emerging technologies are shaping biotech’s future, with insights on European markets, patient-focused innovation, and therapeutic trends.

💪 Empowering Others Through Life Sciences
Love hearing about other people’s journeys into Life Sciences? This episode focuses on a diverse career which includes time at small biotechs and pharmaceutical giants, non-profit initiatives in Africa, and advice on how to balance professional excellence and meaningful contributions in STEM.

🤖 The Role of Machine Learning in Clinical Trials

Learn how machine learning can predict trial outcomes, address diversity in research, navigate ethical concerns, and transform the landscape of AI-powered medicine.

Apply ✍️

🧬 Senior Staff Scientist, Wellcome Sanger: Love solving sequences? Design cutting-edge workflows, refine NGS technologies, and drive impactful discoveries in a collaborative lab setting.

🎯 Senior Director, Sales and Business Development, CGT, Fresenius Kabi: Ready to steer sales success? Oversee a dynamic team, develop key partnerships, and drive revenue growth in the ever-evolving world of cell and gene therapies.

🧪 Senior Scientist – Peptide Chemistry, AstraZeneca: Ready to mix things up? Design, synthesise, and purify complex peptides, collaborating with a dynamic team to advance cutting-edge drug development.

RSVP 📆

🇺🇸 13–15.01 | Biotech Showcase | San Francisco, USA: Dive into cutting-edge biotech as startups pitch, investors scout opportunities, and industry leaders forge collaborations to advance therapeutics and healthcare solutions.

🔎 29–30.01 | Festival of Genomics & Biodata | London, UK: Explore the expanding world of genomics and biodata, from multi-omics to AI, as industry leaders connect to tackle healthcare’s biggest challenges.

🍽️ 30.01 | BIA Gala Dinner 2025 | London, UK: Celebrate life sciences at the Roundhouse with fine dining, networking, fundraising, and live music alongside 760+ industry leaders and innovators.

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SomX is a communications and creative agency for healthcare. We are clinicians, scientists, creatives and communicators dedicated to serving our healthtech, biotech, pharma and public sector clients with services across strategy, content, PR, design, events and media production. Get in touch to learn more.