It takes two for tango! Antibody-drug conjugates company and new kid in the block Pheon Therapeutics has emerged from stealth raising £68 million Series A. Paraphrasing the classics: biotech shall not live by blubird bio’s gene therapy pricing alone but from all therapeutic indications. On that front, GSK has just acquired an exclusive license for what could become the first oral carbapenem antimicrobial to receive marketing approval in the United States. In this edition of Cambridge Biocapital we spend a few lines on investment data in British biotech and pharma during the first 6 months of the year. Yes, indeed, it is not great but, bring in some context, it is not as awful as it may sound. Clock.bio has semi-emerged from stealth and Dr Mark Kotter and Mr Florian Schuster know something about it. The Innovators Club Accelerator program run by the Science Entrepreneurship Clubhas announced the participants for its Cohort III. In a nutshell: synthetic biology at its best. It will be interesting to watch how the patent landscape looks like in a few years. Dr Karin Schmitt has left Mogrify to become Chief Operating Officer at Mursla and Simon Dingemans has been appointed Chair of the Board at Genomics Plc after leaving GSK’s Financial Chair in 2019. Reasons to celebrate? VIVO Biobank has become the UK’s leading research resource for samples and data of children’s and young people’s cancers. In the Interesting readings section: Among others, Dr David Powell (CSO at LifeArc) on separating science from politics, Kate Bingham DBE on the future of brain biology, and Dr Coco Newton on navigating the oceans from academia to entrepreneurship and the options to do this within the Cambridge community. Music: JJ Cale. Let’s dive in!

London-based Pheon Therapeutics has emerged from stealth and raised $68 million series A. Pheon is developing antibody-drug conjugates (ADC) for hard-to-treat cancers .The round has been led by a Brandon Capital, Forbion and Atlas Venture, and participated by Research Corporation Technologies (seed investor). Pheon leadership team includes Betrand Damour (CEO; among other leadership positions, ex-CEO NBE Therapeutics, ex-CEO Mind NRG, acquired by Minerva Neurosciences.), Leigh Zawel (CSO; ex-CSO Cullinan Oncology (NASDAQ), ex-VP of Pfizer’s Centres for Therapeutic Innovation), and co-founders Paul Jackson(Vice President of R&D), and Prof David Thurston(Advisor and co-founder of Spirogen). Recently, Spirogen’s proprietary payload technology (Zynlonta) was approved by the US FDA as the first and only CD19-targeted antibody drug conjugate (ADC) as a single-agent treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (loncastuximab tesirine-lpyl). The drug was approved as part of ADC Therapeutics (NYSE: ADCT) portfolio. The proceedings of the Series A will be used to bring Pheon’s lead compound to Investigational New Drug (IND) stage in the next 18 months, which targets a novel cancer antigen. Overall, the company is developing monotherapies indicated for novel targets and/or novel payloads in solid tumours.

Oxford-based Optellum has raised $14 million Series A. The round has been led by Mercia and participated by Intuitive Ventures and Black Opal. Existing investors include IQ Capital, St John's College in the University of Oxford, and the family office of Sir Martin & Lady Audrey Wood. Optellum is a medtech startup developing an AI-driven platform to diagnose and treat early-stage lung cancer. The proceedings of the round will be used to scale up operations in the UK and US, and advance the AI-platform to enable personalised therapy decisions by integrating imaging data with molecular data, robotics, and liquid biopsies. Optellum is the first and only medtech company to attain FDA clearance, CE-MDR in the EU, and UKCA in the UK for its software platform Virtual Nodule Clinic. Since FDA clearance, Optellum has announced strategic partnerships in the US with GE Healthcare and the Lung Cancer Initiative at Johnson & Johnson, while the Virtual Nodule Clinic has been implemented in various healthcare systems across the US.

Cambridge-based Clock.bio has emerged from stealth but no further details have been disclose yet. Clock.bio has launched with the mission of decoding rejuvenation programs in pluripotent stem cells as a basis of a new class of health preserving therapies. The company is participated at least by Dr Mark Reinhard Kotter (Director; co-founder of bit.bio, Meatable, among others), Mr Florian Schuster (Director; co-founder and Chief Corporate Officer at bit.bio) and Dr Bernhard Klemen (Director; General Partner at Sarmayacar, Managing director at IOI Capital). They are joined by other co-founders Grant Belgard (Senior Director of Bioinformatics at bit.bio, CEO at The Bioinformatics CRO) and Dr Koby Baranes (Research Associate at the Dept. of Clinical Neuroscience at the University of Cambridge, who specialises in reprogramming methods to generate oligodendrocytes and other glial cells from human pluripotent stem cells). Lorraine Gibbs joins as Chief of Staff. More to come, likely before the end of the year.

Cresset has raised an undisclosed amount of growth capital from Scottish Equity Partners. Cresset is a drug discovery software scale up developing computational methods to describe molecules and accelerate drug discovery (virtual screening, hit discovery, lead optimisation, and QSAR analysis. According to company information, its computer-aided drug discovery (CADD) solutions are used by 8 of the top 10 pharma companies. The proceedings of the funding will be used to expand operations in US and advance the computational platform.

Let’s just use these few minutes of intimacy to look at investment data in British biotech and pharma during the first 6 months of the year. Drinks are at the back, tissues in the first drawer. Looking by sector (data from Pitchbook), the share of VC deal value (£B), has gone from £3.03b (2021) to £1.02 (Jan to June 2022). To say it all, 2021 was an exceptionally good year for biotech. Although looking at previous years the situation does not look that terrible, this house believes this is nothing to be content with: £0.78 (2017), £1.40 (2018), £0.83 (2019), £1.30 (2020). For context, sectors that have improved in comparison to last year are Energy (£0.43 in 2021, £0.80 in H1, 2022) and IT Hardware (£0.1.16 in 2021, £1.19 in H1, 2022). Looking at the share (%) of VC exit count by sector, biotech and pharma have gone from 10% (2020), 29% (2021) and 7% (H1 2022). Software keeps up with previous years, 65% (2020), 116% (2021) and 65% (H1 2022).

University of Edinburgh-spinout Stimuliver has emerged from stealth raising £1.7 million seed funding from the BioInnovation Institute and Vaekstfonden, Denmark’s state investment fund. Stimuliver, based in Denmark, is developing regenerative stem cell therapy to treat acute and chronic liver diseases. The company’s technology has been developed by tissue engineer Prof David Hay (CSO) and Dagmara Szkolnicka, PhD (COO) from the University’s Centre for Regenerative Medicine. The company has developed proof-of-concept data which demonstrates that these implants can be administered under the skin, rather into the liver, which, in chronic liver disease patients, is a hostile environment for cell therapies. The company formation was supported by Edinburgh Innovations, the University of Edinburgh’s commercialisation service.

The Innovators Club, run by non-profit Science Entrepreneur Club (SEC), has announced the 3rd cohort of synthetic biology startups. FTI Consulting and Gallagher are supporting this edition. SEC Cohort III includes the following startups. i) Alt Atlas: Machine learning-platform comprising 3 modules (stem cell, plant breeding and food design) that optimises R&D and production of alternative ingredients to meat and dairy industry; ii) CEXAL: non-living biosensor technology for make cost-effective molecular testing leveraging smartphone-based technology and ML pattern recognition; iii) Chronos Dx: high-performance, low-cost, scalable bioassay technology for drug screening and diagnostics at single-molecule detection level aiming to overcome disease heterogeneity; iv) EdenBio: ML-guided genetic engineering of microorganism strains to improve biological parameters aiming to develop vegan alternatives to traditional foods, therapeutics and materials; v) Evolutor: microbe development and optimisation company leveraging proprietary technology for bio-manufacturing a diverse portfolio of sustainable goods; and vi) Umkele: novel carbon-negative synthetic biology-based manufacturing process with initial focus on plastic-free packaging and alternative silk fabric.

NanoSyrinx has been named as the winner of the 2022 AbbVie UK Golden Ticketaccelerator programme. The company will have access to one year of free lab space, funded by AbbVie, at Pioneer Group’s BioCity Nottinghamsite, as well as support from AbbVie’s and Pioneer Group’s business and scientific networks. NanoSyrinx raised £6.2 millions of seed financing in June 2021 led by Octopus Ventures and participated by existing investor M Ventures. Previous investors included BioCity, the UK Innovation & Science Fund, IQ Capital, and Jonathan Milner. The company is developing protein-made ‘nanosyringes’ as a cell-selective non-viral peptide and protein delivery system, and proof-of-concept studies both for in vivo active pharmaceutical delivery and ex vivo cell engineering are currently being undertaken to optimise for target selection.

Allia’s Accelerator Challenge is open for applications. Age-tech, Med-tech start-ups across the UK are welcome to apply to the program. Up to £100,000 of investment are available from BRAN Investments, as well as further funding and network opportunities. The challenge has 3 phases (Venture Foundation, Pitch Perfect and Demo Day) that enable progression depending on the suitability of the venture’s idea, stage of growth and alignment with investor criteria. Deadline: October 14th.

Cambridge Enterprise, Cambridge Innovation Capital, and the University of Cambridge have launched Innovate Cambridge. This Greater Cambridge-wide initiative aims to collectively define an inclusive vision for the future of the region. A summit was held, and attendees were asked to sign the Innovate Cambridge Charter, pledging to support and enhance the Cambridge ecosystem.

The Early Cancer Institute (ECI) has been inaugurated in Cambridge becoming the first physical institute in the UK dedicated to early cancer. Prof Rebecca Fitzgeraldhas been appointed Director for the ECI, which will have over 120 scientists from a broad spectrum across the University. Certain cancer types remain to be defeated by treatment: only 5% of pancreatic cancer patients survive for ten or more years, and that figure rises to 10% in lung cancer, 12% in oesophageal cancer and liver cancer, and 15% in acute myeloid leukaemia. Thus, the Institute will be focusing on hard-to-treat cancers and those having very poor outcomes. ECI is looking to fundraise £20 million to carry out research and development activities.

Cancer Research UK (CRUK) has partnered with Blood Cancer UK to launch VIVO Biobank. By merging two existing biobanks – CCLG Tissue Bank and CellBank – VIVO Biobank has become the UK’s leading research resource for samples and data of children’s and young people’s cancers (including solid tumours and leukaemias), and it provides a single point of access for people and organisations involved in such research.

LifeArc has joined the Antimicrobial Resistance (AMR) Syndicate alongside the Cystic Fibrosis Trust and Medicines Discovery Catapult. The Syndicate aims to address unmet patient needs by bringing together experts from the NHS, industry, and academia. The end goal is to accelerate the discovery and development of cystic fibrosis antimicrobials, allowing for a faster reach to patients and improved infection diagnostics. More information about the syndicate can be found here.

Cambridge-based Domainex’s pre-clinical drug candidate DMXD-011 has shownefficacy in ex-vivo human studies. The drug could be indicated for treatment of interferonopathies (e.g. lupus, Sjögren’s syndrome, scleroderma, etc). Domainex has previously shown DMXD-011 efficacy in animal models of lupus and rheumatoid arthritis. The ex-vivo laboratory studies have used blood samples from hospital patients with interferonopathies, DMXD-011 was added, and the levels of inflammatory biomarkers in the blood were measured before and after stimulation. The company reports “DMXD-011 showed a strong dose-dependent reduction in the expression of these biomarkers” and points out “AstraZeneca showed a similar effect in the recently-approved type 1 interferon receptor antibody anifrolumab”. No further details have been disclosed.

Cardiff-based Cotton Mouton Diagnostics has secured ISO 9001 and ISO/IEC 17025 accreditation and has launched endotoxin testing service in collaboration with the team at FUJIFILM Wako Pyrostar. The CMD αBET system is a more rapid and sustainable solution for endotoxin testing using a reduced amounts of the FDA-licensed turbidimetric LAL reagent.

Fuljifilm Diosynth Biotechnlogies has expanded its Billingham-based large scale microbial manufacturing plant. The works have now started after announcement in December 2021. The new facility will include 20,000 sq. ft. modular clean room space, two purification suites, a column packing room, refolding suites, and two primary recovery suites. The expansion will allow the company to double down in its contract development manufacturing organisation operations. The expansion is expected to be complete late next year / early 2024 when Fuljifilm will aim to create 350 jobs.

bit.bio has added two new cell products for the study of neurodegenerative diseases. The portfolio expansion includes an ioGlutamatergic Neurons TDP-43 M337V disease model as well as early access to its ioMicroglia cell product. These aim to reduce the experimental variability in non-clinical studies and improve the translatability and reproducibility of research findings.

Genomics PLC has appointed Simon Dingemans as Chair of the Board. Simon was Chief Financial Officer at GSKwhere he served for 8 years. He led the acquisition of Novartis vaccine business and the creation of Haleon, the consumer healthcare company. Currently he is senior advisor to The Carlyle Group, focused on the healthcare sector as well as opportunities in the UK. Dingemans replaces outgoing Chair Dave Norwood, who remains part of the Board.

Cambridge-based Mursla has appointed Dr Karin Schmitt as Chief Operating Officer. Dr Schmitt joins from Mogrifyand has previously held senior leadership positions at US-based Exelixis and Millennium Pharmaceuticals and UK-based Thermo Fisher Scientific, Horizon Discovery Group and PredictImmune. She has played a key role in bringing 20 innovative products to market internationally, across a wide scientific spectrum, with recent emphasis on clinical products. Mursla is developing a suite of proprietary multi-omics methods to characterise exosomes. The aim is to develop a blood test that can detect early-stage hepatocellular carcinoma better than the current state of the art.

Keensight Capital has won Best Corporate Sustainability Strategy for a Management Company at the Private Equity Wire ESG AAA European Awards 2022. Keensight is one of the leading pan-European growth buyout investment firms. This award recognises the firm’s objective to direct financial flows towards responsible companies by considering their non-financial performance on a governance, social and environmental level.

London-based Complement Therapeutics has announced its Clinical and Scientific Advisory Board. The establishment of the Board will accelerate the development of the company’s pipeline including its lead product (CTx001), a gene therapy for dry age-related macular degeneration/geographic atrophy. Robyn Guymer, PhD is Professor of Ophthalmology at Melbourne University and he Deputy Director and Head of Macular Research at Centre for Eye Research Australia. She was named a Member in the General Division (AM) in the 2018 Queen’s Birthday Honours List. Jeff Heier, MD is the Director of the Vitreoretinal Service, President of the Retina Society, and serves on the Executive Committee of the American Society of Retina Specialists. Peter Kaiser, MD is the Director of Centre for Ocular Research and Evaluation and a RO1-funded principal investigator at the National Institute of Health. Prof Paul Morgan is Group Leader at the UK Dementia Research Institutein Cardiff, Fellow of the Academy of Medical Sciences, and has been President of the International Complement Society. Matthew Pickering, PhD is Professor of Rheumatology and Wellcome Trust Senior Fellow in Clinical Science at the Centre for Inflammatory Disease, Department of Immunology and Inflammation, Imperial College London. Last, Dr Nadia Waheed is a Professor in Ophthalmology at the Tufts University Department of Ophthalmology. She was the Chief Medical Officer at Gyroscope Therapeutics and previously the Director of the Boston Image Reading Center.

Prof Nick Fox has been awarded the European Grand Prix 2022 by the French Alzheimer Research Foundation. Prof Nick Fox’s research focuses on the early diagnosis and progression of neurodegenerative dementias as well as on biomarkers for testing potential treatments for Alzheimer's disease. He is Group Leader at the UK Dementia Research Institute at UCL, Honorary Consultant Neurologist at the National Hospital of Neurology and Neurosurgery, among other commitments. The award grants €100,000 and will be used to improve diagnosis in dementia, by validating an ultra-fast MRI protocol to reduce the time for an MRI brain scan to 5 minutes from the current 20 to 30 minutes.

University of Oxford-spinout PepGen (Boston-based) has appointed Habib Joseph Dable to its Board of Directors. Mr. Dable is the former President and Chief Executive Officer of Acceleron Pharma. Prior to his role at Acceleron, Mr. Dable worked at Bayer AG where, over the course of his 22-year tenure, he served in roles of increasing responsibility, including Global Head, Neurology and Ophthalmology and President of U.S. Pharmaceuticals. PepGen is a clinical-stage biotech developing oligonucleotide therapies aiming to transform the treatment of severe neuromuscular and neurological diseases.

Oxford University-spinout PepGen (NASDAQ: PEPG) has reported positive data from Phase 1 healthy normal volunteer (HNV) trial of PGN-EDO51. The drug is the company’s lead product candidate for the treatment of Duchenne Muscular Dystrophy (DMD) patients whose mutations are amenable to an exon 51 skipping approach. The trial consisted of administering a single ascending dose (SAD) to evaluate the safety and tolerability of PGN-EDO51 in 32 healthy adult males. Oligonucleotide tissue concentration and exon skipping were also assessed. PGN-EDO51 utilises PepGe’s proprietary Enhanced Delivery Oligonucleotide (EDO) technology, and it is designed to skip exon 51 of the dystrophin mRNA transcript. This is an established target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and enabling the production of a truncated, yet functional dystrophin protein. The company reported “PGN-EDO51 exhibited the highest levels of oligonucleotide delivery and exon skipping in a clinical study following a single dose when compared to publicly available clinical data for other exon 51 skipping approaches”. Phase 2a trial evaluating PGN-EDO51 in DMD patients in expected to begin during H1 2023.

Exscientia (NASDAQ: EXAI) has published research validating its AI Precision Medicine Platform for improving patient outcomes. The research has been published in Blood Cancer Discovery in collaboration with ETH Zurich, the Medical University of Vienna, and the Center for Molecular Medicine. The work, titled Deep Morphology Learning Enhances Precision Medicine by Image-Based Ex Vivo Drug Testing builds on the EXALT-1 trial by using deep learning algorithms to classify complex cell morphologies from patient cancer tissue samples into disease morphotypes. The outcomes generate clinically relevant insights into which treatments can deliver the most benefit to individual patients. Further evaluation would allow better understanding of which other patients may benefit too from similar treatments.

GSK plc (LSE/NYSE: GSK) has entered a $66 million exclusive licence agreement with Sphero Therapeutics, Inc(NASDAQ: SPRO) for tebipenem HBr. The exclusive licence will allow GSK to commercialise tebipenem HBr in all regions except for Japan and certain other Asian countries. Beyond the $66 million upfront, Spero Therapeutics will receive will receive further payments tied to commercial milestones. In addition, GSK has agreed to acquire $9 million in stocks of Sphero common stock. Tebipenem HBr is the first oral carbapenem antibiotic that could potentially treat complicated urinary tract infections (cUTI), including pyelonephritis, caused by certain bacteria. Spero will start a new phase III clinical trial in 2023, following encouraging US FDA regulatory feedback on clinical trial design. If approved, tebipenem HBr would be the first oral carbapenem antimicrobial to receive marketing approval in the United States.

London-headquartered Silence Therapeutics (NASDAQ: SLN) has announcedpreliminary single dose results from the GEMINI II Phase 1 study in Patients with Thalassemia. The study evaluated SLN124 in 24 adults with non-transfusion dependent thalassemia. Following a single dose, no serious adverse events or severe treatment emergent adverse events (TEAEs) that were SLN124 related or TEAEs leading to withdrawal were reported. No dose limiting toxicities or drug related liver injury were observed. SLN124 is a a siRNA targeting the TMPRSS6 gene, a gene ‘silencing’ therapy designed to temporarily block a specific gene’s message that would otherwise trigger an unwanted effect. SLN124 has rare pediatric disease and orphan drug designations for beta-thalassemia as well as orphan drug designation for polycythemia vera.

Connect Health Tech podcast has talked to Coco Newton, PhD. Great conversation on the entrepreneurship programs available within Cambridge such as Cambridge Judge Business School’s Enterprise Tech and Enterprise TechStar program.

“While we appreciate the UK government’s alternative funding proposals will go some way to make up for lost Horizon grants, it must be stressed that any national alternative will be unable to make up for the critical ability to collaborate with world leading European scientists and institutions at the forefront of ground-breaking research and innovation.“ Dr David Powell (CSO at LifeArc) has written a piece calling for action in regards to science policy.

This year 33 European digital health startups have been acquired or bought out, a figure that will almost certainly top the 2021 total of 35 and perhaps exceed the historic figure (44) set in 2017 and 2018. Sifted has published an article on why this may be happening, Sherlock.

“Major depressive disorder and generalised anxiety have an 80% overlap in disease definitions,” says Daniel Karlin, Chief Medical Officer of MindMed. The Economisthas published a fantastic piece on brain biology with interesting views from Kate Bingham, Amit Etkins and Paul Berns, among others.

A propos of bluebird’s $3 million gene therapy, insightful piece in Endpoints on drug pricing. https://endpts.com/are-3m-gene-therapies-the-new-normal-experts-say-bluebird-will-be-a-test-case-for-a-market-ready-for-prime-time/

Imperial College London is featuring spinout SPARTA Biodiscovery. The company’s technology is Single Particle Automated Raman Trapping Analysis (SPARTA) to accelerate the development and production of nanoparticles used in advanced medicines.