The Needle Issue #12


Welcome back to The Needle, a newsletter from Haystack Science to help you navigate the latest translational research, with a roundup of the latest news on preclinical biotech startups from around the world.

After a brief summer hiatus, we are delighted to be back. This week, in the context of all the chatter about in-licensing Chinese assets, we highlight a novel target originating from a Hangzhou City biotech. P-selectin glycoprotein ligand-1 (PSGL-1) is a new member of the growing target class of phagocytic checkpoints, which includes CD47/SIRPa, disialoganglioside (GD2), leukocyte immunoglobulin-like receptor subfamily B1 (LILRB1) and CD24 (Siglec10) among the examples with drug programs farthest in clinical development.

On the investment front, despite the funding doldrums, venture funds across the world purporting to seek “early-stage” investments keep on being raised. And the UK government also announced a big biotech push with its ‘Life Sciences Sector Plan’. It may be summer, but the financings (including a $216 megaround series A) and deals keep ticking over. Any that we missed, let us know (info@haystacksci.com).

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The Summer BIO report “The State of Emerging Biotech Companies: Investment, Deal, and Pipeline Trends” highlights how much China-based programs have contributed to the drug pipeline over the past 10 years.

A couple of weeks ago, Bloomberg also summarized deal data showing how the share of global licensing by Chinese biotech companies has jumped over the past two years.

Judging by a report listing 16 ‘high-value’ currently unlicensed assets from China being hawked by longtime Phalanx Investment Partners analyst David Maris, there is more licensing to come.

In this context, we read with interest a recent Science Immunology paper describing a monoclonal antibody (mAb) program targeting a novel phagocytic checkpoint under development at yet another Chinese biotech: MedimScience, founded in Hangzhou City in 2021. MedimScience is one of a growing cadre of companies, including LTZ Therapeutics, Dren Bio, Chengdu Kanghong, Antengene and ImmuneOnco, looking to develop novel myeloid cell engagers/phagocytic checkpoint inhibitors.

Phagocytic checkpoint inhibitors are drugs that circumvent the molecular cloaks that tumors throw around themselves to avoid uptake and destruction by myeloid cells, such as macrophages, monocytes, and neutrophils. The strategy first came to the fore through pioneering work on the ‘don’t eat me’ signal CD47, work carried out by Ravi Majeti and Irv Weissman at Stanford. Results from their preclinical studies spurred the launch of startup Forty Seven (subsequently acquired in 2020 by Gilead) and the first-in-class anti-CD47 IgG4 magrolimabprogram.Phase 1b trial results of magrolimab combined with azacitidine in acute myeloid leukemia (AML) patients were so impressive that, by 2022, more than 20 different companies had anti-CD47 programs in clinical development. This blew up spectacularly when early trials failed to be reproduced in larger efficacy trials of combinations — failure that was largely attributed to intolerability/anemia issues related to the target, slow action/early disease progression, and a failure to account for patient heterogeneity with regard to P53 mutation status. But the strategy is compelling and the hunt for new phagocytic checkpoints has continued with new antibody formats seeking to avoid these pitfalls.

Now, Cheng Zhong and his colleagues at MedimScience report the identification of a new evasion actor — PSGL-1 — that suppresses macrophage-mediated phagocytosis in a variety of hematological malignancies. PSGL-1, which was previously known largely for its role in cell adhesion, is highly expressed in various hematologic cancers, including AML, T-acute lymphoblastic leukemia (T-ALL) and multiple myeloma (MM).

Moreover, high PSGL-1 expression has been found to correlate with poor patient survival in AML, T-ALL and MM.

Using several mouse models, the researchers found that tumors lacking PSGL-1 show slower progression, increased macrophage infiltration, and higher rates of phagocytosis by macrophages, effects that were independent of T cells or dendritic cells.

Mechanistically, the team found that PSGL-1 disrupts the interaction between the cell-adhesion molecule ICAM-1 on tumor cells and the integrin LFA-1 (CD11a/CD18) on macrophages. And when they tested Novartis’ lifitegrast, an inhibitor of ICAM-1/LFA-1 binding, they found this largely abrogates the phagocytosis of PSGL-1 knockout tumor cells, confirming PSGL1’s role in impairing prophagocytic signaling and cytoskeletal reorganization required for effective tumor-cell engulfment.

The authors went on to develop a humanized mAb against PSGL-1 and show its ability to induce phagocytosis of human tumor cells in vitro and to reduce tumor burden in mouse models of AML, T-ALL, and MM. The antibody showed a good safety profile in non-human primates with no significant toxicity at high doses. Additionally, PSGL-1 blockade synergized with chemotherapy (doxorubicin) and antibody-based therapies (anti-CD47 and anti-CD38), further underscoring the translational potential of this strategy, particularly in treatment-resistant settings.

Papers: Best of the rest

Target biology

Procr+ chondroprogenitors sense mechanical stimuli to govern articular cartilage maintenance and regeneration | Cell

Nicotinamide N-methyltransferase inhibition in cancer-associated fibroblasts restores CD8+ T-cell antitumour activity in syngeneic ovarian, breast and colon tumour models | Nature

A mast cell receptor mediates post-stroke brain inflammation via a dural-brain axis | Cell

Hindbrain octadecaneuropeptide gliotransmission as a therapeutic target for energy balance control without nausea or emesis | Science Translational Med

G-protein-coupled receptor ADGRG1 drives a protective microglial state in Alzheimer’s disease through MYC activation | Neuron

The microRNA miR-30a blocks adipose tissue fibrosis accumulation in obesity | JCI

Sialyltransferase ST8Sia6 overexpression protects pancreatic β cells from spontaneous autoimmune diabetes in nonobese diabetic mice | JCI

Proof-of-concept studies

Nanobody therapy rescues behavioral deficits of NMDA receptor hypofunction in GluN1-knockdown mice modeling GRIN1 disease | Nature

Molecular glues that facilitate RAS binding to PI3Kα promote glucose uptake without insulin in Zucker fatty rats | Science

A molecular glue of RAS and cyclophilin A catalyzes covalent inhibition of the aspartic acid in RASG12D mutants in preclinical models of lung, pancreatic, and colorectal cancer | Science

AGPAT4 targeted covalent inhibitor potentiates targeted therapy to overcome cancer cell plasticity in hepatocellular carcinoma mouse models | Science Translational Med

New broad spectrum paenimicin antibiotic identified acting on lipid A in Gram-negatives and teichoic acids in Gram-positive bacteria, with no detectable resistance | Nat Comms

Small-molecule pyrvinium pamoate interacts with nuclear speckled scaffold protein (SON) to rehabilitate phase separation in nuclear speckles and restore proteostasis transcription factor levels to alleviate mouse models of retinal degeneration and tauopathy | Nat Comms

An oral heme oxygenase inhibitor targets immunosuppressive perivascular macrophages in preclinical models of hepatocarcinoma | Science Translational Medicine

A pan-KRAS inhibitor and its derived degrader elicit multifaceted anti-tumor efficacy in KRAS-driven pancreatic and lung cancers modeled in organoids and mice | Cancer Cell

Discovery and development of an oral analgesic inhibiting the α2B adrenoceptor validated in mice| PNAS

Restoration of hexosaminidase deficiency in mice through intracerebral injection of allogeneic Sca1- committed progenitor cells without myeloablative preconditioning | Nature

NSD2 inhibitors rewire chromatin to treat lung and pancreatic cancers in mice| Nature

Cysteinyl leukotrienes stimulate gut absorption of food allergens to promote anaphylaxis in mice | Science

Drughunter highlights notable recent patents on small-molecule against new targets in rare disease, oncology, inflammation, and pain.

Immunotherapy

Design of soluble Notch agonists that drive T cell development and boost immunity in mice | Cell

Intestinal mast cell–derived leukotrienes mediate the anaphylactic response to ingested antigens in mice | Science

OGG1 augments the transcriptional activation of Foxp3 to promote iTreg differentiation for IBD alleviation | PNAS

Deka Medicine’s IL-2/IL-10 fusion with an anti-EGFR scFv arm activates T cells/NK cells, eliciting antitumor activity without peripheral inflammatory toxicity or T-reg accumulation in mouse models and primates | Cell Reports Medicine

LARP4-mediated hypertranslation drives T cell dysfunction in tumors | Nature Immunol

De novo design and structure of a peptide–centric TCR mimic binding module | Science

De novo-designed pMHC binders facilitate T cell–mediated cytotoxicity toward cancer cells | Science

Design of high-specificity binders for peptide–MHC-I complexes | Science

Cancer immunology data engine reveals secreted AOAH as a potential immunotherapy | Cell

ACLY inhibition promotes tumour immunity and suppresses liver cancer | Nature

A CD4+ T lymphocyte–specific TCR/GSDMD/IL-2 axis facilitates antitumor immunity | JCI

CAR-T cells targeting CD155 reduce tumor burden in preclinical models of leukemia and solid tumors | JCI

Delivery platforms

Mannich reaction-based combinatorial libraries identify antioxidant ionizable lipids for mRNA delivery with reduced immunogenicity | Nature Biomed Engineering

Cationic ‘Discrete Immolative Guanidinium Transporters’ deliver mRNA to lung, spleen, and immature red blood cells | Nat Comms

Thermoreversibly assembled polymersomes for highly efficient loading, processing and delivery of protein and siRNA biologics | Nature Biomed Engineering

Transferrin receptor–targeted anti-amyloid antibody enhances brain delivery and mitigates ARIA | Science

Genome editing

Design of highly functional genome editors by modelling CRISPR–Cas sequences | Nature

Iterative recombinase technologies for efficient and precise genome engineering across kilobase to megabase scales | Cell

AI-generated MLH1 small binder improves prime editing efficiency | Cell

In vivo prime editing rescues alternating hemiplegia of childhood in mice | Cell

Without immunosuppressive drugs, allogeneic donor islet cells engineered to overexpress CD47, with CRISPR-Cas12b editing to B2M and CIITA, survive and produce C-peptide 12 weeks after intramuscular injection into a man with type 1 diabetes | NEJM

Startup news

The UK launched its Life Sciences Sector Plan:

Life Sciences Sector plan commits £520 million for life sciences manufacturing and £600 million investment to deliver a ‘Health Data Research Service’

Although university spinout funding is in the doldrums, venture funds are still raising dollars for “early-stage” biotech:

Frazier Life Sciences announces $1.3 billion fund XII, with ~40-50% going to ‘early-stage’ private therapeutic startups

Swiss-based Omega Funds rounds up $647 million for eighth fund for biotech startups

Biogen builds a ventures team internally to find, finance and build external assets

UK’s Cambridge Innovation Capital (CIC) pledges £100 million in Fund III to take more spin-outs out of Cambridge University

Since the 2021 peak during the COVID-19 pandemic, venture capital raises in the biopharma industry have declined. Money is creeping back to the sector via bigger raises at fewer funds:

The silver lining is especially evident in Asia/Oceania:

TCG Labs Soleil venture fund adds $400 million to its initial raise and opens a Shanghai unit for single-asset builds

National University of Singapore launches $78 million spinout fund for startups

Singapore-based Origgin launches venture fund II with $23 million for Asian university spinouts

Australian-based Brandon Capital raises $287 million for sixth fund for biotech startups

Recent weeks have seen several accelerator campaigns to attract startups:

BioLabs, TUM Venture Labs, and Eli Lilly today launches BioLabs|TUM, a new biotech innovation hub in central Munich to galvanize early-stage biotech startup activity

US startups working on respiratory illnesses should apply by Sept 30 for Biolabs/Chiesi Farmaceutica IMPULSE initiative, with offer of 1 yr of lab space, Biolabs/Chiesi mentoring, and access to Biolabs’ networks.

Freshly funded UK accelerator Slingshot invites applications from UK life science startups

U-Ploid Biotechnologies awarded sixth annual Pitch Battle at Oxford Bioescalator prize, with Granza Bio, Orfonyx Bio, and Infinitopes receiving commendations

Canadian adMare Academy releases report on Canadian biotech, with applications closing on Sept 5 for its Executive Institute for life science entrepreneurs interested in honing company building skills/network

Last, some news on grant awards for translational research around the world:

US Senate Appropriations Committee votes 26-3 to boost NIH’s budget by $400 million, rejecting Trump administration’s $17 billion cut and $15 million cap on research grant overheads

Publicly funded labs working on preclinical validation of putative drug targets should apply for The Singapore Therapeutics Development Review 2025 grant by August 28

UK research universities commission working group to advise on best practices to award £40 million in funding to accelerate proof-of-concept studies and spur university spinouts

Preclinical financings

Preclinical deals

Stay in touch

We hope you enjoyed this issue of The Needle and hit the button below to receive forthcoming issues into your inbox

If you’re interested in commercializing your science, get in touch. We can help you figure out the next steps for your startup’s translational research program and connect you with the right investor. Follow us on X, BlueSky and LinkedIn. Please send feedback; we’d love to hear from you (info@haystacksci.com).

Until next time,

Juan Carlos and Andy

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