Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for
the Year Ended 31 December 2021
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
Company Information 1
Chairman’s Statement 3
Board of Directors and Senior Management 8
Strategic Report 10
Directors’ Report 18
Governance Report 24
Directors’ Remuneration Report 31
Independent Auditors Report 37
Consolidated Statement of Comprehensive Income 44
Consolidated Statement of Financial Position 45
Company Statement of Financial Position 46
Consolidated Statement of Changes in Equity 47
Company Statement of Changes in Equity 48
Consolidated Statement of Cash Flows 49
Company Statement of Cash Flows 50
Notes to the Financial Statements 51
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
Company Information
Dr Vladislav Sandler (Chief Executive Officer)
Professor Sir Marc Feldmann (Chairman)
Alexis Sandler (Non-Executive Director)
Peter Redmond (Non-Executive Director)
Company Secretary
Andrew Wright
Registered Office
5 Fleet Place London
Registered Number (England and Wales)
Joint Brokers
SP Angel Corporate Finance LLP
Prince Frederick House
35-39 Maddox Street
Peterhouse Capital Limited
80 Cheapside
Independent Auditor
PKF Littlejohn LLP
Statutory Auditor
15 Westferry Circus
Canary Wharf
E14 4HD
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
UK Solicitors
Cooley (UK) LLP
69 Old Broad Street
US Solicitors
Rubin & Rudman LLP
50 Rowes Wharf
Massachusetts 0211
Principal Bankers
Metro Bank plc
One Southampton Row
Computershare Investor Services plc
The Pavilions
Bridgwater Road
BS13 8AE
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
Chairman’s Statement
We achieved significant progress in our lead projects in 2021, having decided to focus on those
developments which we felt promised progression to human trials or other milestones most quickly. Key
to this was the work we have done on our CAR-T cell therapy for blood cancers, while we have also
taken our CDX antibody project forward following completion of the work carried out by Eli Lilly and
Company (“Lilly”) and the signing of a licensing agreement with Lilly for intellectual property developed
by it. Building on lines of research we had been developing previously, we have also filed a provisional
patent application for our Chimeric Bait Receptor (“CBR”) platform technology, a novel approach which
potentially has the capacity to cure most viral infections, both known and yet to appear, and certain
As a result of this programme of activity and outstanding scientific work the Company now has a strong
roster of assets, and is firmly on the path to taking them to market.
Financially, as the accounts show, our operating loss rose to approximately £2.7 million over the period,
up from approximately £2.2 million in 2020. Operating costs remained very low for a biotechnology
company developing such cutting-edge assets as ours, but we have made strong progress largely thanks
to the quality and effectiveness of our scientific team and the expertise of our consultants.
Overall, however, the Group made a loss of £5,108,310 (2020: £2,095,023 loss) during the period under
review. Approximately half this figure arose in connection with the convertible loan facility through
which we raised £12 million during the period, which consisted in part of introductory, legal and exchange
listing fees, and in part of the discount on the market value of the shares issued upon conversion of the
debt to shares; this latter component is a non-cash loss arising for accounting reasons and contributed to
the increased reported loss for the year.
The convertible loan facility did not operate as we expected. Consequently, we decided to terminate the
facility through a partial repayment and replacement of the facility balance with conventional equity,
thanks to the efforts of our brokers. While this has proved to be expensive and our share price has fallen
back substantially, conventional sources of finance on this scale were not available at the time and these
arrangements have crucially enabled us to raise sufficient funds to take our key projects significantly
forward and to bring our lead CAR-T product candidate close to being ready, subject to the work now
being carried out, for human trials in the coming months.
I review progress and developments on our lead projects below. I shall refer to our other projects more
briefly, with the reasons for the priorities that we have selected.
CAR-T cell therapy
Our chimeric antigen receptor T-cells are developed to be a treatment for relapsed/refractory (“R/R”)
Acute Myeloid Leukaemia (“AML”), the most common form of leukaemia and one for which there are
currently no fully effective treatments. AML has a five-year survival rate of less than 30% in adults and
is currently treated using chemotherapy, rather than the potentially more effective form of therapy being
developed by Hemogenyx Pharmaceuticals.
Our CAR-T therapy is expected to be a novel form of immunotherapy that programmes a patient’s own
T-cells to recognise an antigen expressed by cancerous cells and hence destroy these cells. The product
we are developing, which we refer to as HEMO-CAR-T, was constructed using our proprietary humanised
monoclonal antibody against a target, the FLT3 protein, that is over-expressed in AML cells and can be
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
found on their surface. Testing has demonstrated that HEMO-CAR-T is able to effectively programme
human T-cells to identify and destroy human AML-derived cells both in vitro (in non-animal studies) and
in vivo (in animal studies).
We have made strong progress over the past two years in the development of HEMO-CAR-T. The
programme has seen very encouraging results to date and promises to represent a major breakthrough in
the treatment of AML. The Company is now focusing its greatest efforts on this asset, which remains
proprietary and wholly owned by the Company.
In January 2021, we announced that we had entered into a Master Translational Research Services
Agreement with the University of Pennsylvania (“Penn”) to advance HEMO-CAR-T toward and through
clinical trials. The collaboration with Penn, the world’s leading experts in CAR-T therapies, is of vital
importance and demonstrates the potential value of our development. The intended outcome of the
relationship is clinical proof of concept for HEMO-CAR-T.
In preparation for human trials, the Company engaged Quality Systems LLC (“Quality Systems”) to
oversee chemistry, manufacturing and controls processes and to assist with the compilation of regulatory
filings. Quality Systems is an expert in this field and has taken more than 30 cell and gene-based therapies
to Phase I/II of clinical trials.
In December 2021, we engaged leading contract manufacturing organisation WuXi Advanced Therapies
ATU (“WuXi ATU”) for product development and manufacturing of the DNA plasmids and viral vectors
required to manufacture HEMO-CAR-T cells to support Phase I clinical trials. This partnership is key in
accelerating the timeline to deliver this innovative therapy to patients in need of a more effective treatment
for AML. Work on these precursors for the creation of HEMO-CAR-T is now well advanced.
We are now at an advanced stage of completing our application for Investigational New Drug (“IND”)
status. Work on manufacturability, quality, safety and other key parts of the development of HEMO-CAR-
T has continued and we expect to be in a position scientifically to commence trials once the IND
designation has been granted.
CDX antibody
CDX is a bispecific antibody targeting a majority of forms of relapsed/refractory AML, a subset of ALL,
and myelodysplastic syndrome; it may also be used in conditioning patients for bone marrow transplants
in a more benign way compared to traditional chemotherapy and/or radiotherapy protocols. CDX was the
subject of our development collaboration with Lilly which concluded in 2021. The partnership resulted
in the selection of a clone of the antibody that is ready for IND-enabling studies, the final step before
applying for a licence to conduct clinical trials.
Effective and non-toxic conditioning may extend the use of bone marrow transplantation to older and
more frail patients and potentially target additional indications including autoimmune diseases, such as
lupus and multiple sclerosis, for which the risk of conventional bone marrow transplantation has been a
major road-block.
The potential applications of the CDX antibody have far wider potential than initially anticipated, and we
now believe that CDX may be used not only for conditioning for bone marrow transplants but also as a
direct treatment, or when used in combination with other existing treatments, for AML and other blood
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
In October 2021 we secured an exclusive worldwide licence for all applications to intellectual property
developed by Lilly related to the final form of the CDX bispecific antibody. With patent protection now
secured (as described below), and following the valuable contributions made by Lilly, we believe that
CDX should attract significant interest from financial or strategic partners in taking the asset forward to
animal toxicology studies and ultimately clinical trials.
Following the year end, in January, the Company announced a partnership with Selexis SA (“Selexis”)
to leverage Selexis’s SUREtechnology Platform™ of protein expression technologies and modular
workflows to advance the Company’s CDX bispecific antibody toward human trials. The service
agreement will help the Company to reduce the time, effort, and costs associated with developing the cell
line for the antibody for the treatment of AML.
CBR is a novel platform technology that constitutes a new paradigm for treating viral infections and some
forms of cancer. The Company has been working on CBR since before the start of the COVID-19
pandemic, leveraging prior discoveries by the Company, and we have made considerable progress over
the course of 2021.
The essence of the CBR-based approach is to programme immune cells to destroy pathogens using a
novel type of modifiable synthetic receptor. We have ascertained that this approach can also potentially
be used to destroy malignant cells causing certain types of cancer, making it a broadly applicable and
exciting new class of therapy. Detailed work has been undertaken by our scientists to prove the efficacy
and widen the scope of the platform. We have achieved laboratory proof of concept for a CBR construct
that eliminates the SARS-CoV-2 virus responsible for COVID-19. The Company is planning to conduct
in vivo tests of the anti-SARS-CoV-2 CBR construct. Work also continues on a CBR construct for cancer.
Although work to date has been focussed on specific viruses, in particular SARS-CoV-2, the CBR
approach is applicable in principle to almost any form of virus. We believe it is likely to be of particular
value in combatting emerging or rare forms of viral infection, treating sufferers where effective vaccines
or anti-viral drugs have not yet been developed or have failed to be effective. These may include future
mutations of the SARS-CoV-2 virus and also new viruses that may cause new pandemics, referred to as
“Disease X”, which scientists have warned to be highly likely in the coming years.
Major advantages of our CBRs for combatting viral infections are: (1) the use of a bait makes CBRs
insensitive to mutations of the targeted virus, preventing the development of resistance; (2) CBRs are
made from naturally occurring receptors that are responsible for the function of immune cells and endow
the host’s own immune system with the ability to destroy invading pathogens; and (3) CBRs are modular
synthetic receptors that can be rapidly reconfigured to attack almost any virus, bacteria, or malignant
The Company has said relatively little about its work on CBR to date as it is a potentially broadly
applicable and highly valuable new method of creating immunotherapy treatments that is not comparable
to any other developments taking place, as far as we are aware. Now that we have achieved proof of
concept and a provisional patent application has been filed in relation to CBR we can consider business
development options. We believe that the technology should attract interest from a range of potential
partners with whom we can now hold more substantive conversations.
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
Other assets
The Company’s portfolio also includes its licence to the Hu-PHEC cell therapy approach that was the
subject of CEO and Co-founder Dr Vladislav Sandlers original discovery while working at Cornell
University, and its Advanced Hematopoietic Chimera (“AHC”) humanised mice that uniquely do not
suffer from graft-versus-host disease. These remain potentially valuable assets. Some work continues in
the background, including collaborations using our AHC as platforms for disease modelling. Shareholders
will be aware that we entered into joint ventures in 2018 to take these projects forward but these were
ultimately without result and the convertible loans to subsidiaries associated with them were repaid during
2021. However, our pragmatic focus has been on our lead assets and on becoming a clinical trial-stage
The Company continued to strengthen the legal protection of its pipeline assets through 2021.
In June 2021, the Company received US patent approval in relation to CDX, covering its method of use
for conditioning patients for bone marrow/hematopoietic stem cell transplantation. It also covers a subset
of sequences of monoclonal antibodies against target proteins existing on the surface of hematopoietic
stem cells/hematopoietic progenitors and/or a number of leukaemias. This patent solidifies the
Company’s leading position in the development of ground-breaking cancer-related therapies and protects
the Company’s intellectual property in this key asset.
In August 2021, the Company received US patent approval covering sequences of monoclonal antibodies
to the human FLT3/FLK2 receptor protein that is found on the surface of AML cells, hematopoietic (blood
forming) stem cells and progenitors, and dendritic cells. These monoclonal antibodies have allowed the
Company to develop both the bispecific CDX antibody and HEMO-CAR-T as treatments for AML as
well as potential treatments for other types of blood cancers, and CDX as a product for bone marrow
transplant conditioning.
The Company has made further patent applications in relation both to HEMO-CAR-T and to the CDX
bi-specific antibodies, the latter being a joint application with Lilly.
Following the period covered by these accounts, in March 2022, the Company filed a seminal provisional
patent application protecting its intellectual property rights in its CBR platform, as mentioned above.
Scientific and business advisers
The scientific advisory board, which I chair, continues to provide valuable guidance to the Company and
its scientists. During 2021 and into 2022 it has been particularly useful in shaping the Company’s intended
protocol for clinical trials of its CAR-T therapy, leveraging our advisers’ extensive experience. The
Company has now also bolstered its commercial expertise through the appointment in July 2021 of Dr
Alan E. Walts as a business adviser and board observer on an initial 12-month term. Dr Walts is a highly
qualified life sciences industry veteran, having served as a senior manager at Genzyme in a career there
lasting over 25 years. He is now a Venture Partner with Advent Life Sciences, a position he has held since
January 2014, and has positions with several other public, private and charitable life sciences
organisations. Dr Walts’s input has been instrumental in shaping the Company’s strategy for developing
its pipeline of assets, and in introducing the Company to several potential partners.
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
New laboratory premises
The Company recently occupied new premises in the Manhattanville Factory District of New York. The
premises, custom built to our requirements, are significantly larger than our original laboratory and
include two clean rooms, enabling us to carry out some procedures which we have until now had to
outsource to third parties. These will commence with the manufacturing of HEMO-CAR-T cells for
clinical trials in-house, a process that alone can save the Company over US$2 million compared with
outsourcing this work. They also open up the possibility of manufacturing cells for other organisations as
a potential source of revenue. The new laboratory will enable us to grow as we proceed to clinical trials
and commercialisation of our pipeline products, and has other advantages including proximity to
Columbia University and other life sciences institutions. It is gratifying that the Company’s highly
talented and dedicated scientific team now has the space and quality of facilities that it needs to further
its crucial work.
Overall, 2021 was an important year for Hemogenyx Pharmaceuticals in terms of product development
and strategic focus, and the present year has continued this positive progress. It remains for me to thank
our expert and committed team of scientists and our informed and influential group of scientific and
business advisers. We particularly look forward to taking our key CAR-T project toward human trials and
to seeking partnerships and other financing arrangements to further the development of our CDX and
CBR assets over the rest of 2022.
Prof Sir Marc Feldmann AC, FRS
MB BS, PhD, FRCP, FRCPath, FAA, F Med Sci
29 April 2022
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
Board of Directors and Senior Management
Professor Sir Marc Feldmann Non-Executive Director & Chairman appointed 9 April 2018
Professor Sir Marc Feldmann is a pre-eminent medically trained immunologist at the University of
Oxford where he was Head of the Kennedy Institute of Rheumatology until 2014 and now Emeritus
Professor. He trained in medicine at Melbourne University and then earned a Ph.D. in Immunology at the
Walter & Eliza Hall Institute with Sir Gus Nossal, before working in London at the Imperial Cancer
Research Fund. Sir Marc's main research interests are immunoregulation, understanding mechanisms of
autoimmunity and the role of cytokines in disease, and working out how to fill unmet medical needs.
His work in London led to the generation of a new hypothesis for the mechanism of autoimmunity, linking
upregulated antigen presentation and cytokine expression. Testing this hypothesis led to the discovery,
with colleague Sir Ravinder Maini, of the pivotal role of TNFα (Tumour Necrosis Factor alpha) in the
pathogenesis of rheumatoid arthritis. This major discovery has revolutionised therapy not only of
rheumatoid arthritis but other chronic inflammatory diseases (e.g. inflammatory bowel disease, psoriasis,
and ankylosing spondylitis), and helped change the perception of monoclonal antibodies from niche
products to mainstream therapeutics. Anti-TNF therapeutics are the current leading drug class with 2016
sales exceeding US$36 billion.
This has led to much scientific recognition, for example election to the Royal Society and Academy of
Medical Sciences in London, the National Academy of Sciences USA and the Australian Academy of
Science, and multiple major International prizes including the Crafoord Prize of the Royal Swedish
Academy of Sciences, the Albert Lasker Clinical Research Award (NY), the Ernst Schering Prize, the
Paul Janssen Award for Biomedical Research, and the Canada-Gairdner Award. He was also the first
recipient in biology or medicine of the EU/European Patent Office Inventor of the Year Award in the
Lifetime Achievement category. In addition, Sir Marc has advised more than 20 of the largest
pharmaceutical and biotech companies in the world and has mentored some of the most successful
scientists, many of whom have become senior figures in the commercial pharmaceutical world. Sir Marc
was knighted in the 2010 Queen's Birthday Honours, and was honoured in Australia with the knighthood
equivalent, the Companion of the Order of Australia.
Sir Marc has been at the forefront of promoting effective scientific-medical-pharmaceutical interactions.
He has built up a huge network of friends and collaborators who meet regularly in Oxford and who will
help Hemogenyx Pharmaceuticals to grow and enter clinical trials.
Dr Vladislav Sandler Chief Executive Officer appointed 4 October 2017
Dr Vladislav Sandler is the Co-Founder and CEO of Hemogenyx Pharmaceuticals and a research
Assistant Professor at the State University of New York (SUNY) Downstate. Dr Sandler is a widely
published stem cell scientist with decades of experience in scientific research. In particular, Dr Sandler
has extensive experience developing novel methods of direct reprogramming of somatic cells into
functional and engraftable hematopoietic stem cells, as well as developing novel sources of pluri- and
multi-potent cells.
Dr Sandler has conducted his research in Russia, Israel, Canada and the United States, including at the
Children's Hospital at Harvard Medical School, the Salk Institute for Biological Sciences, Harvard
University and Albert Einstein College of Medicine, among others. He also led a team of scientists at
Advanced Cell Technologies, Inc. and was most recently on the faculty of Weill Cornell Medical College.
While at Cornell, Dr Sandler made the significant discovery that the cells that give rise to blood stem
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
cells during mammalian development continue to exist after birth, and he developed the method of
isolation of these cells from humans. As a result of this important work, Dr Sandler was awarded the
inaugural Daedalus Fund Award for Innovation at Cornell. He went on to found Hemogenyx
Pharmaceuticals in order to further pursue this significant scientific discovery and his dedication to the
translation of science into clinical practice.
Dr Sandler has published numerous peer-reviewed papers and has received a number of awards and
fellowships for his scientific research. Dr Sandler received his PhD from the University of British
Columbia. He is a member of the International Society for Stem Cell Research.
Alexis Sandler Non-Executive Director appointed 4 October 2017
Alexis M. Sandler is the co-founder of Hemogenyx Pharmaceuticals, for which she has served as the
Chief Operating Officer. Ms Sandler is an attorney specialising in intellectual property, with 20 years of
experience representing a range companies and institutions.
Ms Sandler is the Vice President and General Counsel of Pace University. A talented and respected
attorney with a wide range of experience and expertise, Ms Sandler previously served for nearly a decade
as in-house counsel for The Museum of Modern Art. Prior to that, she worked as the director of business
and legal affairs for a major media and entertainment company, and in private practice for several
prominent law firms.
Ms Sandler received her AB from Harvard University and her JD from the UCLA School of Law and is
a member of the State Bar of New York and the State Bar of California.
Peter Redmond Non-Executive Director appointed 4 October 2017
Peter Redmond is a corporate financier with some 40 years’ experience in corporate finance and venture
capital. He has acted on and assisted a wide range of companies to attain a listing over many years on the
former Unlisted Securities Market, the Main Market of the London Stock Exchange and AIM, whether
by IPO or in many cases via reverse takeovers, across a wide range of sectors, ranging from technology
through financial services to natural resources and, in recent years has done so as a director and investor
of the companies concerned.
He was a founder director of Cleeve Capital plc (now BigBlu Operations Limited) and Mithril Capital
plc, both formerly listed on AIM prior to reverse takeovers, and of Silver Falcon plc, the Company into
which Hemogenyx Pharmaceuticals reversed, and he took a leading role in negotiating and effecting the
reverse takeover. He undertook the same role in the rescue, reconstruction and refinancing of AIM-quoted
3Legs Resources plc (now SalvaRx Group plc) and now Standard Listed URA Holdings plc and several
other companies, and took a significant active part in fundraising for the above companies.
He is currently a director of Standard Listed URA Holdings plc.
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
Directors Strategic Report for the year ended 31 December 2021
The Directors present their Strategic Report of Hemogenyx Pharmaceuticals plc for the year ended 31
December 2021.
This Strategic Report comprises a number of sections, namely: the Group’s objectives, the Group’s
strategy and business model, a review of the Group’s business using key performance indicators, and the
principal risks and uncertainties facing the business. The disclosures under s172 of the Companies Act
2006 are included in the Governance Report.
The Group’s objective is to develop breakthrough therapies for the treatment of blood and autoimmune
diseases and of viral infections.
Strategy and Business Model
The Group’s long-term strategy is to create a suite of products to address current problems associated
with the treatment of blood disorders such as cancers and autoimmune diseases, with viral infections, and
with bone marrow or hematopoietic stem cell transplants. The latter represents an important part of
the solution to treating blood-related diseases, with the opportunity to improve outcomes through reduced
blood stem cell transplant rejection and relapse, and if successful potentially provides long-term cures for
these diseases.
The Group’s business model aims to advance its therapies through clinical proof-of-concept, taking them
towards a final stage of development. A goal is the licensing of one or more of its therapies to partners in
return for potential upfront payments, research funding support, success milestone and royalty payments.
Operational Review and Outlook
The operational review and outlook are set out in the Chairman’s Statement.
Financial Review
The Group incurred a loss for the year to 31 December 2021 of £5,108,310 (31 December 2020 loss
of £2,095,023).
In the year to 31 December 2021 the loss mainly arose from operational expenses pursuing the Group’s
objectives listed above as well as salaries, consulting and professional fees, and general administration
expenses. These expenses have been met from the proceeds of the issue of convertible loans and equity
placings. The Group received other income of £99,943 (2020 £85,237) from collaborations with
partners, and £71,932 (2020: nil) from the forgiveness of a loan under the United States Payment
Protection Program in 2021 for a total of £171,875 (2020 £85,237). Finance costs connected with the
convertible loan arrangement have been expensed to profit or loss in ull in the year, following the
cessation of that acility, which would otherwise have been deferred and spread over the life of the
convertible loans.
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
Cash flow and cash position
Cash used in operations totalled £2,627,298 (31 December 2020: £1,798,404).
As at 31 December 2021, the Group had a cash balance of £6,840,969 (31 December 2020: £1,812,299).
Key Performance Indicators
The Directors have identified the KPIs below that they feel are the most vital measurements for the Group
to monitor given its current stage of development. KPIs are monitored on an annual basis to ensure that
they remain the most important and relevant measure of performance and progress.
Cash management
The Group supplemented its funding with proceeds totalling £12,000,000 resulting from the issuance of
convertible loans that took place in the year. The lender converted £10,400,000 of such loan into shares
of the Company and we repaid the remaining £1,600,000. We also repaid loans to Orgenesis Inc. during
2021, such that at 31 December 2021 there was no outstanding indebtedness. The cash position at 31
December 2021 was £6,840,969 (31 December 2020: £1,812,299).
The Group carefully plans expenditure with rolling cash flow forecasts and tight financial control. The
Group takes a collaborative cost sharing approach with business partners and avoids long-term
commitments as far as possible.
Intellectual property
The Group will focus on developing new conditioning treatments, drugs and cell therapy products for
blood and autoimmune diseases, HSC/BM transplantation, and viral infections. The Group, or its
licensors, has applied for patents to protect its proprietary technology and future products, which are in
varying stages of development.
The success of the Group will depend largely on the Groups ability to implement successful drug
development programmes, obtain the required regulatory approvals (in various territories), protect and
exploit its own intellectual property and know-how, and the intellectual property and know-how licensed
to it, and to generate a cash flow in accordance with the strategy of the Group. Intellectual property is
protected by the Group through taking a pro-active approach to filing patents over its products and
technologies, as well as the diligent maintenance and protection of such patents and licences.
The Group patent portfolio currently includes:
CDX bi-specific antibodies
The patent application relating to CDX bi-specific antibodies was filed by Hemogenyx Pharmaceuticals
LLC in the USA on 4 April 2016 ("CDX Patent") and awarded as Patent Number US 11,021,536 B2 on
1 June 2021. The invention summarised in the patent application is a method of eliminating hematopoietic
stem cells/hematopoietic progenitors ("HSC"/"HP") in a patient using bi-specific antibodies specifically
binding to a protein predominantly expressed on the surface of HSC/HP and to a protein uniquely
expressed on a surface of immune cells. The bound bi-specific antibodies redirect immune cells to
eliminate HSC/HP. The invention relates to the required conditioning of a patient prior to a BM/HSC
transplant. In this respect, the invention serves two main purposes:
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
it provides adequate immunosuppression of the patient and clears sufficient niche space in the
bone marrow for the transplant of HSC. This allows transplanted cells to engraft in the recipient;
it could potentially help to eradicate the source of malignancy.
On April 4 2017, a PCT (Patent Cooperation Treaty) application was filed by Hemogenyx
Pharmaceuticals which includes additional claims that extend the CDX Patent set out in the provisional
patent application. These claims protect specific sequences of several high-quality clones discovered and
validated by the Group. The claim extension transforms the original "method" provisional patent
application into a "composition of matter" PCT application. An additional composition of matter patent
application (covering novel sequences of the antibodies discovered and validated by the Company in
collaboration with Lilly) has been filed following completion of the Lilly collaboration agreement.
Monoclonal antibodies
In July 2019 the Group filed a composition of matter patent application entitled MONOCLONAL
ANTIBODIES TO HUMAN FLT3/FLK2 RECEPTOR PROTEIN in relation to newly-discovered
monoclonal antibodies against a target protein expressed on the surface of hematopoietic stem
cells/hematopoietic progenitors and a number of leukaemias, such as AML. The patent was granted on
31 August 2021 as Patent Number US 11,104,738. This patent covers composition of matter (sequences)
of monoclonal antibodies to the human FLT3/FLK2 receptor protein that is found on the surface of acute
myeloid leukemia (AML) cells, hematopoietic (blood forming) stem cells and progenitors (HSC/HP),
and dendritic cells. It also covers a method of application of the Group’s bi-specific CDX antibodies for
conditioning patients for bone marrow transplantation.
Hu-PHEC cell therapy
The patent relating to Hu-PHEC was filed by Cornell University ("Cornell Patent") in several
jurisdictions on 13 November 2014. The patent was approved and issued in the United States of America
on 25 February 2020 and published by the European Patent Office on 13 May 2020. The invention
summarises a method of isolation and identification of post-natal hemogenic endothelial cells, as well as
the provision of substantially purified populations of post-natal hemogenic endothelial cells,
compositions of post-natal endothelial cells and methods to utilise post-natal hemogenic endothelial cells
to regenerate the hematopoietic system in a patient.
Advanced Hematopoietic Chimeras
The provisional patent application relating to the Group’s proprietary humanised mouse model, the
Advanced Hematopoietic Chimera, is an application filed by Dr Sandler and Dr Rita Simone in the USA
on 20 February 2018 ("AHC Patent"). The invention summarised in the patent application is mice whose
hematopoietic system is at least 40% humanised and methods for preparing the same. The patent was
assigned to the Group’s subsidiary Immugenyx LLC on 24 May 2018. In June 2019 the Group announced
that Immugenyx LLC has further refined its work to develop the Advanced peripheral blood
Hematopoietic Chimera ("ApbHC") as a research and development tool. The major advantage of the
ApbHC compared to other humanised mouse models known to the Group is the absence of Graft versus
Host Disease, a disease that complicates and often renders impossible the efficient use of peripheral blood
mononuclear cells in transplanted mice. The ApbHC can potentially be used for testing multi-specific
antibodies, including its own bi-specific CDX antibody, as well as for the development and testing of
new cell therapies involving immune cell programming such as CAR-T. ApbHC can also potentially be
used for the modeling of autoimmune diseases, such as Systemic Lupus Erythematosus (aka Lupus), with
a goal of developing fundamentally new treatments for those diseases.
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
Chimeric Bait Receptor
In March 2022, the Company filed a seminal provisional patent application protecting its rights to the
intellectual property covering CBR.
Product development
The Group develops therapies to transform bone marrow and blood stem cell transplant procedures. These
therapies aim to replace the need for existing methods of preparation of patients for transplantation, such
as chemotherapy and radiation treatments, and at the same time address the problem of finding matching
stem cell donors whilst reducing the risk of blood stem cell rejection after transplantation.
The Group’s key products, CDX antibodies, CAR-T therapy, the CBR platform, and Hu-PHEC cell
therapy, are currently in preclinical development. In addition, the Group’s AHC product is currently the
subject of collaborations with other pharmaceutical companies to evaluate AHCs effectiveness as
platforms for disease modelling and drug discovery.
The Directors monitor product development through pre-clinical results. The CDX and CAR-T products
have been successfully evaluated in the Group’s proprietary humanised mouse model, achieving proof of
concept. Furthermore, we have achieved a notable demonstration of CDX’s activity versus AML cells in
vitro and in vivo. If successful, the Company may be able to use the CDX and/or CAR-T products to
eliminate R/R AML in patients who qualify for bone marrow transplantation. The Company is also
investigating the possibility of using its CDX antibodies in combination with other treatments for AML
to increase their effectiveness. A CBR construct designed to target SARS-CoV-2 has been tested in vitro.
Hemogenyx Pharmaceuticals is committed to workplace diversity which includes but is not limited to
gender, age, ethnicity and cultural background.
Hemogenyx Pharmaceuticals’ Diversity Policy defines initiatives which assist the Company in
maintaining and improving the diversity of its workforce. The table below highlights the proportion of
women engaged by the Group:
Organisation as a whole
Executive management team
Board of Advisors
The Group engages the services of a Board of Advisors who are highly experienced in both the clinical
development of treatments and regulatory processes to commercialisation. In addition to Professor Sir
Marc Feldmann, who runs the Board of Advisors in addition to his role as Chairman, the advisors are:
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
Dr H. Michael Shepard, Ph.D.
Led the discovery and development of many successful cancer treatments
including Herceptin/trastuzumab annual sales exceed $6.5 billion worldwide
Received Harvard Medical School's prestigious Warren Alpert Prize in recognition
of contributions to the field of cancer treatment research
Founded NewBiotics, Inc., acquired by Kiadis Pharma
Founded BioLogix, acquired by Symphogen
Dr Koen van Besien M.D.
Professor of Medicine and Director of the Stem Cell Transplant Program at NYP-Weill Cornell
College of Medicine
Developed novel methods of transplantation for those patients who lack matching donors
>200 publications in peer reviewed journals
Editor in Chief of the journal Leukemia and Lymphoma
Corporate Responsibility
We have defined the scope of our Group’s responsible business practices as falling within the following
key focus areas:
Health and Safety ensuring the safety and well-being of our staff
Environment managing our environmental impact areas of waste, energy and water
Employees supporting our people to develop and flourish within the business
Community positive interaction with the communities in which we operate
Ethical Standards operating to the highest ethical standards
We remain committed to ensuring these activities become embedded in how we operate and contribute
towards the success of our business. This includes not only identifying and managing business risk but
exploring opportunities to add value to the business.
Greenhouse Gas Emissions
Given the nature of its activities, there is limited scope for the Group to have a major impact on
environmental matters. Nevertheless, the Directors are mindful of their responsibilities in this regard and
strive to seek opportunities where improvements may be made; these are generally concentrated in areas
of energy conservation, recycling and waste control.
Principal Risks and Uncertainties
The Group operates in an uncertain environment and is subject to a number of risk factors. The Directors
have carried out a robust assessment of the principal risks facing the Group, including those that threaten
its business model, future performance, solvency or liquidity. They consider the following risk factors
are of particular relevance to the Group’s activities and to any investment in the Group. It should be noted
that the list is not exhaustive and that other risk factors not presently known or currently deemed
immaterial may apply.
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
The risk factors are summarised below:
Risks relating to the Group’s business strategy
The Group’s business is relatively undeveloped
The operations of Hemogenyx Pharmaceuticals are at a relatively early stage and, to date, no commercial
sales of its products have been made. The ability of the Group to achieve commercialisation is dependent
on a number of factors, many of which are outside of the Groups control. Examples of factors outside of
the Group’s control are capital market conditions, FDA approval and competition.
Business strategy of the Group
The development of clinical products for new medical treatments is inherently uncertain, with high failure
rates in clinical studies for both early and late stage development products and such clinical studies can
be expensive, time-consuming and complicated and there is no certainty as to the outcome of such
studies. Even once clinical studies have been successfully carried out, later phase trials may not
successfully replicate or improve on such outcomes.
Staffing and key personnel
The Group is reliant on a number of the key personnel, in particular Dr Vladislav Sandler who is the
founder of Hemogenyx Pharmaceuticals (refer to Corporate Governance Report for further detail). Whilst
the Group has endeavoured to ensure that it has contractual arrangements which include non-compete
restrictions in place with such persons to lessen the risk of them ceasing to be involved with the Group,
in the event that the Group was to lose the services of such individuals, its results could be adversely
Costs of commercialisation
The ability of the Group to bring its products to first commercial sale will be dependent in part on the
overall costs of manufacturing and the costs involved could be significant and there is no guarantee that
the sale prices achievable for its products will be viable and sustainable.
Clinical studies and timelines risk
Hemogenyx Pharmaceuticals is currently progressing its product candidates through preclinical
development. Although encouraging results have been achieved so far, there can be no certainty that
these results can be reproduced in clinical trials. The monies raised in the Placings and Subscriptions,
as well as the Orgenesis and Mint Capital convertible loans, are intended to support those preclinical
development activities.
The development of clinical products for new medical treatments is inherently uncertain, with high
failure rates in clinical studies for both early- and late-stage development products. Furthermore, such
clinical studies (Phase 1, Phase 2a/2b, Phase 3) are typically expensive, complex, can take considerable
time to complete and have uncertain outcomes.
Furthermore, as a result of adverse, undesirable, unintended or inconclusive results from any testing or
clinical trials (which have yet to be designed), the future progress, planning and potential treatment
outcome of the products and clinical programmes may be affected and may potentially prevent or limit
the commercial use of one, many or all of the Company's products. In addition, later phase clinical trials
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
may fail to show the desired safety and efficacy obtained in earlier studies, and a successful
completion of one stage of clinical development of an investigational clinical product does not ensure
that subsequent stages of clinical development will be successful.
Failure can occur at any stage of clinical development and, as a result, enforced delays to the clinical
development plan could delay or prevent commercialisation of the Company's product candidates.
Various factors associated with the potential failure or delay in completing a clinical programme include,
but are not limited to:
Delays in securing clinical investigators or clinical study sites;
Delays in securing any regulatory authority, hospital ethics committee, or institutional review
board approval or approvals necessary to commence a clinical study;
Delays or failure to recruit a sufficient number of clinical study participants in accordance with
the clinical study protocol;
Difficulty or inability to monitor subjects adequately during or after treatment;
Inability to replicate in Phase 3 controlled studies any safety and efficacy data obtained from
controlled Phase 2a/2b clinical studies;
Difficulty or inability to secure clinical investigator compliance to follow the approved clinical
study protocol; and
Unexpected adverse events or any other safety or related issues.
Research and development risk
The Group operates in the biotechnology and bio-pharmaceutical development sectors and carries out
complex scientific research. If the research or preclinical testing or clinical trials of any of Hemogenyx
Pharmaceuticals’ product candidates fail, meaning that these candidates will not be licensed or marketed,
this would result in a complete absence of revenue from these failed candidates. Positive results from
preclinical and early clinical studies do not guarantee positive results from clinical trials required to
permit application for regulatory approval. Furthermore, the Group may discontinue the development
of candidates if results are not positive or unlikely to further its progress towards a meaningful outcome
or collaboration.
Intellectual property (IP) infringement
The Group may be subject to future litigation concerning its own IP and the IP of others. Adverse
judgements in relation to its IP would likely have negative outcomes for its results of operations.
Intellectual property (IP) control
The Group is partially reliant on an exclusive, world-wide licence of a patent from Cornell University for
its Hu-PHEC line of business. The exclusivity and exploitable territory for this licence depend on the Group
meeting various developmental milestones.
Environmental and other regulatory requirements
The event of a breach with any environmental or regulatory requirements may give rise to reputational,
financial or other sanctions against the Group, and therefore the Board considers these risks seriously and
designs, maintains and reviews its policies and processes so as to mitigate or avoid these risks. Whilst the
Board has a good record of compliance, there is no assurance that the Group’s activities will always be
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
The Group’s ability to develop its product through to commercial sale will depend upon the Group’s
ability to obtain financing primarily through a further raising of new equity capital. Although the Group
has been successful in raising new equity capital, there can be no guarantee that it will be able to do so in
the future. The Group may not be successful in procuring the requisite funds on terms which are
acceptable to it (or at all) and, if such funding is unavailable, would raise questions over its ability to
further develop its products through to commercialisation. Further, Shareholders’ holdings of Ordinary
Shares may be materially diluted if debt financing is not available.
Market conditions
Market conditions, including general economic conditions and their effect on exchange rates, interest
rates and inflations rates, may impact the ultimate value of the Group regardless of its operating
performance. The Group also faces competition from other organisations, some of which may have
greater resources or be more established in a particular territory. The Board considers and reviews all
market conditions to try and mitigate any risks that may arise from these.
Political and country risk UK departure from the EU
The departure of the UK from the EU is now complete and impact on the business, whose current
operations are principally in the US, has been negligible. Any further changes in international trade, tariff
and import/export regulations as a result of Brexit or otherwise may impose unexpected duty costs or
other non-tariff barriers on the Group. The Company is monitoring matters and will seek advice, where
necessary, as to how to mitigate the risks arising.
Pandemic and business disruption risk
The Company may be affected by disruptions to its operations in one or more locations, particularly in
the near future in light of responses to the novel coronavirus or other potential pandemics. The Company’s
New York operations are classed as an essential business and have not been subject to closure, and work
has continued to date with prudent hygiene and distancing measures in place including limited work in
the laboratory on rota and work from home. All laboratory staff have been fully vaccinated. The Company
is allowing for extended delivery times for some supplies, and for slower progress with collaboration
partners. The Board and UK management continue to operate remotely, as usual. At present the Company
believes that there should be no significant material disruption to its work, but the Board continues to
monitor these risks and the Company’s business continuity plans.
Approved by the Board on 29 April 2022
Dr Vladislav Sandler
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
Directors’ Report for the year ended 31 December 2021
The Directors present their report with the audited financial statements of the Group for the year ended
31 December 2021.
The Company’s Ordinary Shares were admitted to listing on the London Stock Exchange under the name
Silver Falcon plc, on the Official List pursuant to Chapters 14 of the Listing Rules, which sets out the
requirements for Standard Listings, on 9 November 2015.
On 4 October 2017 the Company’s shareholders voted in favour of acquiring the biotechnology company
Hemogenyx Pharmaceuticals Limited, with shares being readmitted to trading on 5 October 2017 under
the name Hemogenyx Pharmaceuticals plc.
Principal Activity
The Group’s principal activity is the discovery, development and commercialisation of a suite of products
to address current problems associated with the treatment of blood disorders such as cancers and
autoimmune diseases, with viral infections, and with bone marrow, or hematopoietic stem cell,
transplants. The company's leading technologies aim to change the way in which bone
marrow/hematopoietic stem cell ("BM"/"HSC") transplants are performed and improve their efficacy.
Hemogenyx Pharmaceuticals’ distinct and complementary products include immunotherapy product
candidates for the treatment of AML and other blood malignancies and patient conditioning the CDX
bi-specific antibody and CAR-T therapy, and a cell therapy product for BM/HSC transplantation the
Hu-PHEC. Each of these products holds the potential to revolutionise the way BM/HSC transplants are
being performed or diseases of the blood are treated, offering solutions that mitigate the dangers and
limitations associated with the current standard of care. Additionally, the Group is developing CBR, a
novel platform technology potentially capable of programming immune cells to attract and destroy a wide
range of viruses and malignant (cancer-causing) cells.
The Group has three companies that are located outside of the UK. The principal laboratory of the Group
is located in Manhattan, New York, USA. The Group also had a subsidiary in Liège, Belgium that was
dissolved on 30 March 2022.
Results and Dividends
The Consolidated Statement of Comprehensive Income set out on page 44 shows a loss for the year
amounting to £5,108,310 (2020: loss of £2,095,023). The Directors do not propose a dividend in respect
of the year ended 31 December 2021 (31 December 2020: nil).
Directors and Directors’ Interests
The Directors who held office during the year and up to the date of this report were as follows:
Date Resigned
Professor Sir Marc Feldmann
Dr Vladislav Sandler
Alexis Sandler
Peter Redmond
Hemogenyx Pharmaceuticals plc
Annual Report & Financial Statements for the
Year Ended 31 December 2021
The Directors of the Company who held office at 31 December 2021 had the following beneficial interests
in the Ordinary shares of the Company at 31 December 2021 according to the register of directors’
At 31 December 2021
At 31 December 2020
Professor Sir Marc Feldmann
Peter Redmond*
Dr Vladislav Sandler
Alexis Sandler
* Peter Redmond holds the majority of these shares through Catalyst Corporate Consultants Ltd of which
he is the sole shareholder.
At the date of this report, there have been no further changes to the Directors’ beneficial interest in the
Ordinary shares of the Company as disclosed in the table above.
According to the Register of Directors’ Interests, no rights to subscribe for shares in or debentures of
Group companies were granted to any of the Directors or their immediate families, or exercised by them,
during the financial year, save for the annual grant of 10,000 ownership units in Immugenyx LLC due to
Dr Vladislav Sandler under the terms of his appointment as CEO and Chief Scientific Officer of that
company. Grants of options are as indicated below (see Note 20 for detail on option plans):
Date of grant
Number of
options at start
of year
Options granted
or acquired
during year
Options lapsed
during year
Number of
options at end of
Professor Sir
Marc Feldmann
9 Apr 2018
Dr Vladislav Sandler
20 August 2020