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#HVO hVIVO – Trading update
Orderbook growth and improved operational delivery lays strong foundations for future growth
· Record revenue, up 30% year-on-year to £50.6 million
· EBITDA margins of no less than 17%, significantly ahead of guidance
· Cash of £28.4 million as at 31 December 2022
· Strong contracted orderbook of £76m, up 65% year-on-year
· Board intention to declare shareholder distribution on publication of full audited accounts
· Over 95% of 2023 revenue guidance contracted and visibility into 2024
hVIVO plc (AIM & Euronext: HVO) (formerly Open Orphan plc), a rapidly growing specialist contract research organisation (CRO) and world leader in testing infectious and respiratory disease products using human challenge clinical trials, announces a trading update for the period ended 31 December 2022.
Significant double-digit revenue growth
The Group expects to report record full year revenues of £50.6 million (2021: £39 million), a 30% increase year-on-year. The significant growth in revenue provides further validation of the long-term sustainable growth in the human challenge trial market, for which hVIVO is the world leader.
EBITDA margins and cash ahead of expectations
The Group expects to report EBITDA margins of not less than 17% (2021: 7.4%), significantly ahead of previous guidance of 13-15%. The increase in EBITDA is driven by strong trading in H2 2022 and operational efficiencies leveraged on the concurrent conduct of multiple challenge trials. In addition, recognition of postponement and cancellation fees for an aggregate of over £1 million had a one-time positive impact on EBITDA. The advanced fees from orderbook growth and efficient operational delivery has resulted in a cash position of £28.4 million as at 31 December 2022 (2021: £15.7 million).
Capital distribution
The Board intends to make a shareholder distribution in respect of the financial performance achieved in FY22, details of which will be announced alongside publication of the Group’s audited results for FY22. The distribution reflects the Group’s exceptional cash generation in the year, in addition to its robust balance sheet.
Further contracted orderbook growth enhances revenue visibility
In 2022, the trend of larger contracts with biopharma clients continued, increasing the size of the Group’s orderbook to £76 million as at 31 December 2022, up 65% year-on-year (2021: £46 million), and over sixfold since 2020 (2020: £12 million).
There has been increased demand for hVIVO’s unique full-service human challenge offering (with three contracts signed in 2022) which also involves the manufacture of bespoke challenge agents to test products against specific infectious disease variants, including subvariants that are circulating in the population. There has also been an increase in average challenge trial contract value driven by client demand for larger volunteer cohorts as the biopharma market increasingly recognises the value of human challenge data to accelerate drug development timelines and de-risk later stage clinical trial programmes.
Current year outlook
The Group has entered 2023 well capitalised, debt free, and with record visibility into the current financial year. hVIVO is firmly placed to build on the growth in the human challenge trial market and further strengthen its position as the world leader in the field. The growing orderbook from new and existing Big Pharma and biotech clients provides excellent forward visibility with over 95% of forecasted revenue for 2023 contracted, and further revenue visibility into 2024. The Board is confident that the Group will continue to leverage its competitive position amidst favourable market dynamics and maintain its strong operational execution, orderbook revenue conversion and focus on profit generation into 2023 and beyond.
Yamin ‘Mo’ Khan, Chief Executive Officer of hVIVO, said: “The record numbers we have been able to announce today is the result of the hard work by everyone at hVIVO over the last 12 months. The goal for hVIVO has always been to establish a long-term sustainable growth model. We now have excellent profitable momentum, with full year 2022 EBITDA margin and cash significantly ahead of market expectations, while revenue shows substantial year-on-year growth. These results signify our successes of 2022 and also lay the foundations for FY23 and beyond. The record contracted orderbook not only covers over 95% of the FY23 revenue targets but also gives us visibility well into 2024.
“With the increasing prevalence and severity of infectious and respiratory diseases, there is a vital need for new vaccines and antivirals; as the human challenge partner of choice to the global biopharma industry, hVIVO is well placed to continue to help accelerate the development of these important new medicines.”
Investor presentation
Yamin ‘Mo’ Khan, Chief Executive Officer, and Stephen Pinkerton, Chief Financial Officer, will provide a live presentation via the Investor Meet Company platform on 25 January 2023 at 18:00 BST.
The presentation is open to all existing and potential shareholders. Investors can sign up to Investor Meet Company for free and add to meet hVIVO here.
#POLB Poolbeg Pharma – POLB sign AI discovery deal with OneThree Biotech for first human challenge trial
POLB’s CEO Jeremy Skillington & One Three Biotech‘s co-founder & COO Brad Pryde catch up with @proactive_UK to discuss the recent AI deal to identify new drugs & treatments for RSV.
#POLB Poolbeg Pharma – Final Results
Poolbeg Pharma (AIM: POLB, ‘Poolbeg’ or the ‘Company’) announces its audited results for the period ended 31 December 2021. These results follow the Company’s spin-out from Open Orphan plc and the subsequent listing on the AIM market of the London Stock Exchange in July 2021.
Poolbeg Pharma is a clinical stage infectious disease pharmaceutical company with a capital light model which is looking to develop multiple products faster and more cost effectively than the conventional biotech model. Poolbeg is targeting the growing infectious disease market and, in the wake of the COVID-19 pandemic, infectious disease is becoming one of the fastest growing markets with an expected value of c. $250 billion by 2025.
The Company aims to bring innovative infectious disease products through to a value inflection point by generating early human efficacy data before rapidly monetising through partnerships and licensing deals with pharma and biotech. Poolbeg’s model aims to significantly reduce spend and risk compared to the conventional biotech model and allows multiple opportunities for monetisation through its broad portfolio. The model also enhances investor returns as the out-licensing revenues will be reinvested into the pipeline of additional assets which in turn, can be further monetised rapidly and as such, substantially extending the runway from the original £25m raised at IPO.
Operational Highlights since IPO – significant strategic and operational progress
– POLB 001, a treatment for severe Influenza, remains on track to commence its bacterial lipopolysaccharide (LPS) human challenge trial in June 2022. Human data is expected before year end, at which point the Company aims to monetise by partnering or out licensing the product to pharma / biotech
– Continued expansion and diversification of the portfolio, reducing risk due to having multiple shots on goal;
o POLB 002 – in-licensed first-in-class, intranasally administered, RNA-based immunotherapy for respiratory virus infections
o Option to licence and evaluate an intramuscular vaccine to prevent Melioidosis (POLB 003), a disease predominately found in tropical and sub-tropical regions, and five additional bacterial vaccine candidates
o Oral Vaccine Delivery Platform – licensed access to use micro- and nanoencapsulation technology to develop oral vaccines for multiple disease indications
– In February 2022 the Company signed its first Artificial Intelligence (AI) deal with OneThree Biotech Inc. to identify new treatments for Respiratory Syncytial Virus (‘RSV’) through analysis of Poolbeg’s unique human challenge trial data and OneThree’s clinically validated biology driven platform
Financial & Corporate Highlights
– Well capitalised with a strong cash balance of £20.9m at period end, following the IPO fundraise of £23.2m (after expenses)
– Loss for the period amounted to £2.3m including the initial non-recurring costs of establishing the Group
– The Company has significant financial resources and will maintain its capital light approach to support its pipeline expansion and development
– Scientific Advisory Board strengthened with the appointment of Professor Daniel Hoft joining Professor Luke O’Neill and Dr Elaine Sullivan
2022 Pipeline
– Intention to dual-list on the OTC market in the US in Q2 2022 to provide additional liquidity in the stock
– Evaluating opportunities for non-dilutive grant funding to support the further development of existing pipeline
– Upon receipt of human data from POLB 001 study in H2 2022, the Company will look to commence monetisation to pharma and biotech companies via licensing or partnership agreements
– Generation of value through advancement of portfolio assets POLB 002, POLB 003, and Oral Vaccine Delivery Platform
– Preliminary outputs from the RSV Artificial Intelligence Discovery Programme are expected in H2 2022
– Continuous engagement and discussion with pharma and biotech companies around the Company’s portfolio including out-licensing and product rights deals
– Continue to identify and conduct diligence on numerous new innovative infectious disease products to add to the pipeline, leveraging the expertise and network of our management team and scientific advisory board. In addition, we continue to evaluate further AI partnerships
– Continue to position the Company firmly at the cutting edge of global R&D as the infectious disease market grows to c. $250bn by 2025
Investor presentation
Poolbeg’s Chairman, Cathal Friel and CEO, Jeremy Skillington will provide a live presentation via the Investor Meet Company platform on 3 March 2022 at 6:00pm.
The presentation is open to all existing and potential shareholders. Investors can sign up to Investor Meet Company for free and add to meet Poolbeg Pharma plc via: www.investormeetcompany.com/poolbeg-pharma-plc/register-investor
Jeremy Skillington, PhD, CEO of Poolbeg Pharma said:
“This has been a period of great progress at Poolbeg. In less than six months we have grown and diversified our portfolio of products and platforms targeting a range of infectious diseases. POLB 001 is on track to commence its LPS human challenge trial, we have in-licensed an intranasal RNA-based immunotherapy POLB 002, as well as partnering to develop an oral vaccine delivery platform and agreeing an option to licence a late pre-clinical Melioidosis vaccine, POLB 003.
“In addition, we are now using cutting edge Artificial Intelligence technology to exploit our unique human challenge data, having signed up leading AI drug development experts OneThree Biotech to analyse and interrogate our RSV data to identify new drug targets and treatments. This is the first time that AI is being used to analyse RSV human challenge study data and initial results are expected in H2 2022.
“As well as growing our operational team since July, we’ve also added invaluable international academic expertise to our Scientific Advisory Board. Our capital-light business model is working well, leaving us with significant financial resources to invest in growing our pipeline further and developing our offering to pharma, without any need for further investment.”
Cathal Friel, Chairman of Poolbeg Pharma said:
“As the single largest shareholder of the Company, I will make it my duty to ensure that the share price starts performing again. I want to make it clear that none of us are happy with where the share price is currently given the drop since IPO. Market conditions have been and remain extremely challenging however, I want to reassure you that we are actively working on creating demonstrable value as we drive the business forward.
My personal belief is that one of the biggest reasons for the pressure on the share price is the perceived overhang from the forthcoming end to the lock-in of the shares received as part of the spin-out, by Open Orphan shareholders. Many shareholders are concerned that the release of these shares may damage the share price. I personally feel that this perception is wrong and that we won’t see a large sell-off of these dividend in specie shares. However, I would like to reassure shareholders that we are looking to put in place a series of actions that will reduce or eliminate the potential for these shares to impact the market. In addition to these specific actions, Poolbeg is also at an advanced stage of dual listing on the OTC market in the USA with plans to complete this within Q2. This should help provide additional liquidity in the Company.
Furthermore, the distribution in specie shares issued to shareholders as part of the demerger from Open Orphan are treated as a distribution for UK tax purposes, which could be taxable as dividend income. However, as advance clearance for a statutory demerger was obtained from HMRC, the distribution is exempt for UK income tax purposes, and hence there should be no UK income tax liabilities for UK resident shareholders. The only time that UK resident shareholder will be subject to tax on these dividend in specie shares, will be in the event that they sell them, and in that event there will be a capital gains tax payment due. This is a further reason why I don’t see shareholders looking to sell these dividend in specie shares immediately upon the end of the lock in.
In summary, I am very optimistic that in the months ahead our share price will perform and more importantly, that we will continue to progress our business model as outlined at IPO while we rapidly grow Poolbeg into a unique publicly listed company which will be a ‘one-stop-shop’ for pharma and biotechs seeking a range of infectious disease assets to develop in the months and years ahead.”
The Company’s Annual Report and Accounts for the period ended 31 December 2021 will be posted to shareholders in due course together with the notice of the 2022 Annual General Meeting, and will be available on the Company’s website: www.poolbegpharma.com/investors/documents/
Footnote: c. $250bn infectious disease market made up of the: Diagnostics Market ( link ), Therapeutics Market ( link ), and Vaccines Market ( link ).
– Ends –
Enquiries
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Poolbeg Pharma Plc Jeremy Skillington, CEO Ian O’Connell, CFO
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+44 (0) 207 183 1499 |
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finnCap Ltd (Nominated Adviser & Joint Broker) Geoff Nash, James Thompson, Charlie Beeson, Richard Chambers, Sunila de Silva (ECM)
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+44 (0) 207 220 0500 |
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Arden Partners PLC (Joint Broker) John Lewellyn-Lloyd, Louisa Waddell
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+44 (0) 207 614 5900 |
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J&E Davy (Joint Broker) Anthony Farrell, Niall Gilchrist
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+353 (0) 1 679 6363 |
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Instinctif Partners Melanie Toyne Sewell, Rozi Morris, Tim Field
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+44 (0) 20 7457 2020 |
#POLB Poolbeg Pharma – Notice of Results
Poolbeg Pharma (AIM: POLB, ‘Poolbeg’ or the ‘Company’) a clinical stage infectious disease pharmaceutical company with a unique capital light clinical model , will announce its results for the period ended 31 December 2021 on Thursday 3 March 2022.
The Company will be hosting an online presentation for investors on the same day, at 6:00pm GMT on the Investor Meet Company platform.
The presentation is open to all existing and potential shareholders. Investors can register to attend here:
www.investormeetcompany.com/poolbeg-pharma-plc/register-investor
– Ends – Enquiries
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Poolbeg Pharma Plc Jeremy Skillington, CEO Ian O’Connell, CFO
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+44 (0) 207 183 1499 |
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finnCap Ltd (Nominated Adviser & Joint Broker) Geoff Nash, James Thompson, Charlie Beeson, Richard Chambers, Sunila de Silva (ECM)
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+44 (0) 207 220 0500 |
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Arden Partners PLC (Joint Broker) John Lewellyn-Lloyd, Louisa Waddell
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+44 (0) 207 614 5900 |
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J&E Davy (Joint Broker) Anthony Farrell, Niall Gilchrist
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+353 (0) 1 679 6363 |
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Instinctif Partners Melanie Toyne Sewell, Rozi Morris, Tim Field
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+44 (0) 20 7457 2020 |
#POLB Poolbeg Pharma – Artificial Intelligence deal with OneThree Biotech
Poolbeg Pharma (AIM: POLB, ‘Poolbeg’ or the ‘Company’) a clinical stage infectious disease pharmaceutical company with a capital light clinical model, has signed an agreement with OneThree Biotech, Inc . a biology-driven artificial intelligence (‘AI’) company, to identify new drug targets and treatments for Respiratory Syncytial Virus (‘RSV’).
Under the terms of the transaction and as aligned with Poolbeg’s strategy, OneThree Biotech’s state-of-the-art AI analysis tools will identify drug assets which target immune-response pathways, have a higher probability of clinical success and have the potential to prevent and / or treat infectious diseases. The analysis will prioritise drugs with existing Phase I safety data, reducing spend and risk, which can feed into Poolbeg’s rapid, capital light clinical development strategy and its expanding pipeline of assets. The analysis is expected to commence in Q1 2022 with preliminary outputs from this work expected in H2 2022.
OneThree Biotech is a clinically validated AI company with a proven technology platform which integrates chemical, biological, and clinical data with cutting-edge computational tools to answer complex questions surrounding disease biology and drug discovery. The team at OneThree will work closely with Poolbeg’s scientific team to build a tailored AI analysis model which can leverage the unique insights of human challenge trial data to identify disease-relevant cell signalling pathways which could lead to novel drug targets. OneThree will receive milestone payments based on candidate development and royalties on the sale of products derived from this partnership.
The Company believe that this partnership with OneThree Biotech is the first time that AI analysis has been undertaken on RSV human challenge trial data and samples to identify new drug targets. The unique nature of human challenge trials to produce disease progression data with high precision is expected to revolutionise the insights generated from this analysis. Poolbeg’s lead asset POLB 001, which is progressing towards its first human challenge trial in June 2022, was identified using such disease progression data. However, by utilising AI the Company aims to identify more targets, quicker and more cost efficiently than previously possible without this technology.
Poolbeg entered a partnership with Eurofins Genomics in October 2021 to complete RNA sequencing of Poolbeg’s RSV disease progression samples from human challenge trials. This work was a key step in Poolbeg advancing its AI analysis programme. This RSV discovery dataset has been specifically designed for incorporation into OneThree’s AI platform. The data sets within this project will leverage up to 140Gb of biological data per subject spread over baseline healthy measurements and the course of infection which is expected to unveil unprecedented insights.
RSV is a contagious virus that affects the respiratory tract of children and at-risk older adults; in severe cases, it can cause pneumonia and other life-threatening breathing difficulties. RSV is a significant public health threat affecting an estimated 50-million people annually, leading to 4 million global hospitalisations and up to 74,500 in-hospital deaths in children under the age of 5 years. An estimated 45% of these cases and deaths occur in new-born infants under the age of 6 months.
Jeremy Skillington, PhD, CEO of Poolbeg Pharma, said:
“OneThree Biotech’s AI analysis tools will allow us to break new ground in data-driven drug discovery, by allowing us to evaluate and interrogate human challenge trial data like never before. This is a key part of our growth strategy, as we’ll be able to identify and develop drugs in a more time and cost effective manner, compared with the traditional biotech model. It will enable us to identify potential new treatments which have a higher probability of clinical success and which can feed into our rapidly expanding pipeline in-line with our capital light model.
This will be the first time that AI tools will be used to analyse RSV human challenge trial disease progression data, which illustrates the significance of this deal as well as Poolbeg’s innovative model. We look forward to updating the market on the outputs from this cutting-edge AI analysis in due course.”
Neel S. Madhukar, PhD, co-founder and CEO of OneThree Biotech , said:
“At OneThree our mission has always been to unlock new therapeutic opportunities by understanding biology on a deeper level. The ability to combine our AI platform with the data and expertise from Poolbeg Pharma will enable just that, and create an opportunity to make meaningful progress in the treatment of infectious diseases.”
Brad Pryde, co-founder and COO of OneThree Biotech , concluded:
“We have been impressed with Poolbeg Pharma’s clear dedication towards using technology and data to create efficiencies in drug discovery and development. We’re excited to demonstrate the strength of this partnership as future developments arise.”
Footnote: During a human challenge study, a number of healthy volunteers are exposed to an infectious agent in a safe and controlled quarantine setting to assess the efficacy of vaccines or treatments. Human challenge trial data is unique in the depth of longitudinal virology, health, biomarker and symptom data collected during the course of disease from pre-infection, onset, progression and resolution of an infection .
– Ends –
Enquiries
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Poolbeg Pharma Plc Jeremy Skillington, CEO Ian O’Connell, CFO
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+44 207 183 1499 |
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finnCap Ltd (Nominated Adviser & Joint Broker) Geoff Nash, James Thompson, Charlie Beeson, Richard Chambers , Sunila de Silva (ECM)
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+44 (0) 20 7220 0500 |
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Arden Partners PLC (Joint Broker) John Lewellyn-Lloyd, Louisa Waddell
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+44 (0) 207 614 5900 |
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J&E Davy (Joint Broker) Anthony Farrell, Niall Gilchrist
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+353 (0) 1 679 6363 |
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Instinctif Partners Melanie Toyne Sewell, Rozi Morris, Tim Field
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+44 (0) 20 7457 2020 |
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Little Dog Communications for OneThree Jessica Yingling, Ph.D.
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+1 (858) 344-8091 |
#ORPH Open Orphan – Launch of STRiVE project
Respiratory viral strains suitable for challenge agents being collected from consenting volunteers
Open Orphan plc (AIM: ORPH), a rapidly growing specialist contract research organisation (CRO) and world leader in testing infectious and respiratory disease products using human challenge clinical trials, announces that hVIVO, a subsidiary of Open Orphan plc, has recently launched the STRiVE project (Seasonal Transmissible Respiratory Virus SurvEy) to collect respiratory viral strains suitable for challenge agents from consenting hVIVO employees. Collection of new respiratory viral strains circulating in the community will allow hVIVO to continually update and broaden its world leading portfolio of human challenge study models.
As part of STRiVE, volunteers with cold or flu like symptoms can send nasal swabs to hVIVO for analysis. Since starting the project, hVIVO has identified over 180 promising virus candidates that could be used in the manufacture of novel challenge agents for its human challenge trials. Viruses isolated include several strains of coronavirus, adenovirus, human metapneumovirus (HMPV), human rhinovirus (HRV), parainfluenza virus (PIV), influenza, and respiratory syncytial virus (RSV).
STRiVE ensures hVIVO can regularly update its challenge agent portfolio with relevant strains as viruses mutate over time. Manufacturing antigenically relevant strains on demand broadens hVIVO’s repository of viral pathogens and could help extend the range of human challenge models it currently offers.
The average UK adult has an estimated 2-4 respiratory infections each year, resulting in billions of pounds of lost output due to illness. Despite familiarity with the negative impacts of cold and flu viruses, remarkably little is understood about the hundreds of viruses responsible that cause illness, including transmission and markers of protective immunity. Using human challenge modelling to better understand such issues could enable future public health benefits and savings.
hVIVO has two decades of experience and expertise in safely conducting challenge studies across a range of respiratory and infectious diseases, including various strains of influenza, RSV, human rhinovirus (HRV – common cold virus), malaria, and asthma. This portfolio has also expanded to include the SARS-CoV-2 virus.
Cathal Friel, Executive Chairman of Open Orphan, said: “Despite only commencing recently, STRIVE has already been a remarkable success, with over 180 viruses identified and 12 different strains, with potential to be used as challenge agents in hVIVO human challenge trials. I would like to thank all hVIVO employees who have thus far participated and encourage more to do so in order to continue to build up this important repository for cold and flu research.
“Importantly, the STRIVE clinical research project will play a significant role in the Company’s efforts to broaden its world leading portfolio of human challenge models.”
Adrian Wildfire, Director of Scientific and Business Strategy at hVIVO, commented: “Access to viable strains of circulating respiratory pathogens is extremely limited and this often presents a significant obstacle to anyone seeking to grow a new viral challenge agent. To grow a new viral strain a virus must be collected from a human host, processed and stored in an adequate manner to keep the virus alive.
“STRIVE will allow the Company to use its expertise to monitor the viral strains of cold and flu that are circulating in the community, and potentially lead to the discovery of new, wild type challenge agents and the development of new human challenge models with unparalleled translation into the field.”
Interested in becoming a volunteer?
hVIVO recruits many of its volunteers for its challenge study clinical trials through its dedicated volunteer recruitment website, www.flucamp.com. By volunteering to take part in one of our studies in a safe, controlled, clinical environment under expertly supervised conditions you are playing your part to further medical research and help increase the understanding of respiratory illnesses.
Individuals interested in taking part in COVID-19 human challenge study research can learn more at www.UKCovidChallenge.com.
For further information please contact:
| Open Orphan plc | +353 (0) 1 644 0007 | |||
| Cathal Friel, Executive Chairman | ||||
| Arden Partners plc (Nominated Adviser and Joint Broker) | +44 (0) 20 7614 5900 | |||
| John Llewellyn-Lloyd / Louisa Waddell | ||||
| finnCap plc (Joint Broker) | +44 (0) 20 7220 0500 | |||
| Geoff Nash / James Thompson / Richard Chambers | ||||
| Davy (Euronext Growth Adviser and Joint Broker) | +353 (0) 1 679 6363 | |||
| Anthony Farrell | ||||
| Walbrook PR (Financial PR & IR) | +44 (0)20 7933 8780 or openorphan@walbrookpr.com | |||
| Paul McManus/ Sam Allen / Louis Ashe-Jepson | +44 (0)7980 541 893 / +44 (0) 7502 558 258 / +44 (0) 7747 515393 | |||
Tiziana Life Sciences #TILS Announces FDA Approval to Initiate Phase I Clinical Trial with Orally Administered Foralumab in Healthy Volunteers
THE INFORMATION CONTAINED IN THIS ANNOUNCEMENT IS DEEMED BY THE COMPANY TO CONSTITUTE INSIDE INFORMATION AS STIPULATED UNDER THE EU MARKET ABUSE REGULATION (596/2014). UPON PUBLICATION OF THE ANNOUNCEMENT VIA A REGULATORY INFORMATION SERVICE, THIS INFORMATION IS CONSIDERED TO BE IN THE PUBLIC DOMAIN.
Tiziana Life Sciences Announces FDA Approval to Initiate Phase I Clinical Trial with Orally Administered Foralumab in Healthy Volunteers.
A breakthrough approach for treatment of autoimmune and inflammatory diseases
London, 16 September, 2019– Tiziana Life Sciences plc (NASDAQ:TLSA and AIM: TILS), a clinical stage biotechnology company focused on developing targeted drugs for cancer and inflammatory diseases, is pleased to announce (further to the announcement made on 1 May 2019) that the U.S. Food and Drug Administration (FDA) has allowed the initiation of a Phase I clinical trial in healthy volunteers using a novel oral enteric-coated capsule formulation of Foralumab, a fully human monoclonal antibody (mAb), in collaboration with the Brigham and Women’s Hospital (BWH), Harvard Medical School, Boston, MA. We believe this is the first clinical trial in which Foralumab will be administered orally to healthy subjects. Our objective is to develop orally administered Foralumab for treatment of autoimmune and inflammatory diseases.
The scientific rationale for this approach was originally discovered by Dr. Howard Weiner, professor at the Brigham and Women’s Hospital, Harvard Medical School. Dr. Weiner discovered that oral or nasal administration of anti-CD3 mAb induces mucosal tolerance to upregulate T regulatory cells (Tregs) capable of providing site-targeted immunomodulation to suppress inflammation. Therefore, this scientific concept could be effective for the treatment of a variety of autoimmune and inflammatory diseases1-5.
“The therapeutic approach of oral administration with Foralumab greatly enhances our ability to treat neurodegenerative and inflammatory diseases. We have also explored the nasal administration of Foralumab for the treatment of progressive MS. We believe nasal and oral administration with Foralumab opens innovative avenues to treat inflammatory and autoimmune diseases by inducing different classes of Tregs. Thus, mucosal activation stimulating Tregs is a physiological mechanism which we think might be safer than other treatment approaches,”commented Dr. Weiner.
Cited References
- Wu HY, Maron R, Tukpah AM, Weiner HL. Mucosal anti-CD3 monoclonal antibody attenuates collagen-induced arthritis that is associated with induction of LAP+ regulatory T cells and is enhanced by administration of an emulsome-based Th2-skewing adjuvant. J Immunol. 2010; 185(6):3401-3407.
- Ochi, H.,et al.,Oral CD3-specific antibody suppresses autoimmune encephalomyelitis by inducing CD4+CD25-LAP+ T cells. Nature Medicine 2006: 12: (6); 627-635
- Lior Mayo, Andre Pires Da Cunha, Asaf Madi, Vanessa Beynon, Zhiping Yang,Jorge I. Alvarez, Alexandre Prat, Raymond A. Sobel, Lester Kobzik, Hans Lassmann, Francisco J. Quintana and Howard L. Weiner. IL-10-dependent Tr1 cells attenuate astrocyte activation and ameliorate chronic central nervous system inflammation. Brain 2016: 139; 1939–1957
- Chantal Kuhn, Rafael M. Rezende, Andre Pires da Cunha, Fabrice Valette, Francisco J. Quintana, Lucienne Chatenoud, Howard L. Weiner. Mucosal administration of CD3-specific monoclonal antibody inhibits diabetes in NOD mice and in a preclinical mouse transgenic for the CD3 epsilon chain. Journal of Autoimmunity (2016) 76: 1-8
- Ogura M, et al., Prevention of human xenograft rejection with oral anti-CD3 mAb. Clinical Immunology 183: 2017; 240-246
About Howard Weiner
Howard L. Weiner is the Robert L. Kroc Professor of Neurology at the Harvard Medical School, Director and Founder of the Partners Multiple Sclerosis (MS) Center and Co-Director of the Ann Romney Center for Neurologic Diseases at Brigham & Women’s Hospital in Boston. He has pioneered immunotherapy in MS and has investigated immune mechanisms in nervous system diseases including MS, Alzheimer’s disease, amyotrophic lateral sclerosis, stroke and brain tumours. He has also pioneered the investigation of the mucosal immune system for the treatment of autoimmune and other diseases and the use of anti-CD3 to induce regulatory T cells for the treatment of these diseases.
About Harvard Medical Centre
Brigham and Women’s Hospital (BWH, “The Brigham”) is located adjacent to Harvard Medical School, of which it is the second largest teaching affiliate. It is the largest hospital of the Longwood Medical and Academic Area in Boston, Massachusetts, USA. With Massachusetts General Hospital, it is one of the two founding members of Partners HealthCare, the largest healthcare provider in Massachusetts. Brigham and Women’s Hospital conducts the second largest hospital-based research program in the world, with an annual research budget of more than $630 million. Pioneering milestones include the world’s first successful heart valve operation and the world’s first solid organ transplant.
About Autoimmune Diseases and Foralumab
Autoimmune diseases constitute a major medical problem and include diseases such as multiple sclerosis, type 1 diabetes, rheumatoid arthritis and inflammatory bowel disease. Other diseases, that have inflammatory components include diseases such as NASH, atherosclerosis and stroke. The induction of regulatory cells at mucosal surfaces by the oral or nasal administration of antigens has been shown to treat a large variety of autoimmune and inflammatory diseases in animal models with minimal toxicity. Foralumab was developed by Novimmune and has been acquired by Tiziana Life Sciences PLC. Foralumab (formerly NI-0401) is thus far the only entirely human anti-CD3 mAb, shows reduced release of cytokines after IV administration in patients with Crohn’s disease with decreases in the classic side effects of cytokine release syndrome (CRS) and improves the overall safety profile of foralumab.
In a humanized mouse model (NOD/SCID IL2γc-/-) developed in Dr Kevan Herold’s laboratory, it was show that while targeting the T cell receptor, orally administered foralumab modulates immune responses of the T cells, enhances regulatory T cells and thus provides therapeutic benefit in treating inflammatory and autoimmune diseases without the occurrence of potential adverse events usually associated with parenteral mAb therapy (Ogura M. et al., 2017). Based on animal studies, the nasal and oral administration of foralumab offers the potential for the immunotherapy of autoimmune and inflammatory diseases in a safe manner by the induction of regulatory T cells.
About Tiziana Life Sciences
Tiziana Life Sciences plc is a UK biotechnology company that focuses on the discovery and development of novel molecules to treat human disease in oncology and immunology. In addition to milciclib, the Company is also developing foralumab for liver diseases. Foralumab is the only fully human anti-CD3 monoclonal antibody in clinical development in the world. This compound has potential application in a wide range of autoimmune and inflammatory diseases, such as non-alcoholic steatohepatitis (NASH), primary biliary cholangitis (PBS), ulcerative colitis, multiple sclerosis, type-1 diabetes (T1D), inflammatory bowel disease (IBD), psoriasis and rheumatoid arthritis, where modulation of a T-cell response is desirable.
Forward-Looking Statements
Certain statements made in this announcement are forward-looking statements. These forward-looking statements are not historical facts but rather are based on the Company’s current expectations, estimates, and projections about its industry; its beliefs; and assumptions. Words such as ‘anticipates,’ ‘expects,’ ‘intends,’ ‘plans,’ ‘believes,’ ’seeks,’ ‘estimates,’ and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company’s control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority
For more information go to http://www.tizianalifesciences.com
This announcement contains inside information for the purposes of Article 7 of EU Regulation 596/2014.
Contacts:
| Tiziana Life Sciences plc
Gabriele Cerrone, Chairman and founder
|
+44 (0)20 7495 2379 |
| Cairn Financial Advisers LLP (Nominated adviser)
Liam Murray / Jo Turner
|
+44 (0)20 7213 0883 |
| Shore Capital (Nominated brokers)
Antonio Bossi / Andy Crossley
|
+44 (0)20 7408 4050 |
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Tiziana Life Sciences #TILS Reports Phase 1 Clinical Data Demonstrating Nasal Treatment with Foralumab was Well-tolerated and Produced Positive Trend in Biomarkers
THE INFORMATION CONTAINED IN THIS ANNOUNCEMENT IS DEEMED BY THE COMPANY TO CONSTITUTE INSIDE INFORMATION AS STIPULATED UNDER THE EU MARKET ABUSE REGULATION (596/2014). UPON PUBLICATION OF THE ANNOUNCEMENT VIA A REGULATORY INFORMATION SERVICE, THIS INFORMATION IS CONSIDERED TO BE IN THE PUBLIC DOMAIN.
Tiziana Life Sciences plc
Tiziana Reports Phase 1 Clinical Data Demonstrating Nasal Treatment with Foralumab was Well-tolerated and Produced Positive Trend in Biomarkers of Immunomodulation and Anti-inflammation in Healthy Volunteers
New York, 10 September 2019 – Tiziana Life Sciences plc (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company focused on innovative therapeutics for inflammatory diseases and cancers, is pleased to report Phase 1 clinical data demonstrating that nasally administered Foralumab, a fully human anti-CD3 monoclonal antibody (mAb), was well-tolerated at all doses. Importantly, the treatment showed significant positive effects on the biomarkers for activation of mucosal immunity, which is capable of inducing site-targeted immunomodulation to elicit anti-inflammatory effects. These clinical data are consistent with the findings from numerous pre-clinical studies.1-3
This Phase 1 trial, conducted at the Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, was a single-site, double-blind, placebo-controlled, dose-ranging study with nasally administered Foralumab at 10, 50 and 250 µg per day, consecutively for 5 days in healthy volunteers for the treatment of progressive multiple sclerosis (pMS). 18 subjects received Foralumab treatment and 9 patient received placebo. All nasal doses were well tolerated. Biomarker analysis showed significant positive immune effects, that were most prominent in the 50 mg cohort with minimal immunomodulatory effects at the 10 µg and 250 µg doses.
Major Highlights
- Treatment was well-tolerated and no drug-related safety issues were reported at any of the doses.
- No drug-related changes were observed in vital signs among subjects at predose, during treatment and at discharge. The mean blood pressure (BP) during the 5 days of treatment were; Cohort A (10 µg/d):124/73, Cohort B (50 µg/d): 119/67 and Cohort C (250 µg/d):113/65 compared to placebo:118/67). Heart rates, respiratory rates and oral temperatures were unchanged among the 3 cohorts compared to the placebo.
- Nasally administered Foralumab at the 50 µg dose suppressed cytotoxic CD8+ as well as perforin secreting CD8+ cells, which have been implicated in neurodegeneration in multiple sclerosis (MS).
- Treatment at 50 mg stimulated production of anti-inflammatory cytokine IL-10 and suppressed production of pro-inflammatory cytokine IFN-γ.
- Taken together, these results suggest stimulation of Tregs that are needed to provide clinical benefits
“Nasal administration of Foralumab is a revolutionary approach to treat patients with neurodegenerative diseases such as progressive MS (pro-MS) and amyotrophic lateral sclerosis (ALS). Extensive data from animal studies with intranasal delivery of anti-CD3 demonstrate that this route of administration induces anti-inflammatory and immunomodulatory effects. This study demonstrates for the first-time that nasally administered Foralumab, at the identified optimal dose of 50 mg, induces immunomodulatory effects capable of providing clinical benefit to treated subjects.This is a major accomplishment providing the scientific rationale to move forward with further clinical development of nasally administered Foralumab in patients with neurodegenerative diseases,”commented Dr. Howard L. Weiner, a member of scientific advisory board of Tiziana Life Sciences. He added that “both oral and nasal administration routes are physiologic approaches to stimulate the mucosal immune system to induce disease modifying immunomodulation. Our immediate focus is on developing Foralumab for treatment of pro-MS.”
“The demonstration of the positive immunomodulatory effects provides the scientific rationale to move forward with further studies in the pro-MS population”,stated Dr. Tanuja Chitnis, the study PI at the Brigham and Women’s Hospital.
“We are very pleased with what we believe is the first-ever demonstration that nasally administered Foralumab is not only well-tolerated, but it also exhibited significantly positive immunomodulatory effects that are indicative of stimulation of Tregs. We are excited as these results provide the scientific rationale for the nasal and oral treatment with Foralumab, our core proprietary platform technologies which could potentially revolutionize treatment with antibodies”stated Kunwar Shailubhai, CEO & CSO of Tiziana.
The person who arranged for the release of this announcement on behalf of the Company was Dr Kunwar Shailubhai, CEO & CSO of Tiziana.
About Foralumab
Foralumab (formerly NI-0401), the only entirely human anti-CD3 mAb, shows reduced release of cytokines after IV administration in patients with Crohn’s disease with decreases in the classic side effects of cytokine release syndrome (CRS) and improves the overall safety profile of Foralumab. In a humanized mouse model (NOD/SCID IL2γc-/-), it was shown that while targeting the T cell receptor, orally administered Foralumab modulates immune responses of the T cells, enhances Tregs and thus provides therapeutic benefit in treating inflammatory and autoimmune diseases without the occurrence of potential adverse events usually associated with parenteral mAb therapy (Ogura M. et al., 2017). Based on animal studies, the nasal and oral administration of Foralumab offers the potential for the immunotherapy of autoimmune and inflammatory diseases in a safe manner by the induction of Tregs.
Preclinical studies on nasal and oral administration with Anti-CD3 mAbs
Preclinical and clinical studies have shown that mucosal induction of Tregs by oral or nasal administration of anti-CD3 mAbs is an innovative approach to treat autoimmune and anti-inflammatory diseases (Kuhn and Weiner 2016)4. Nasally administered anti-CD3 mAbs were shown to ameliorate disease in an animal model of multiple sclerosis by inducing IL-10+LAP+(latency-associated peptide) T cells3, demonstrating nasal anti-CD3 mAbs as a new approach to treat progressive forms of multiple sclerosis and other types of chronic CNS inflammation. Additionally, nasally administered anti-CD3 mAbs suppressed lupus in lupus-prone mice (BWF1) by inducing IL-10 and TGF-β dependent mechanisms associated with a suppression of IL-17 and IL-21 pro-inflammatory cytokines5.
About Tiziana Life Sciences
Tiziana Life Sciences plc is a UK biotechnology company that focuses on the discovery and development of novel molecules to treat human disease in oncology and immunology. In addition to Milciclib, the Company is also developing Foralumab for liver diseases. Foralumab is the only fully human anti-CD3 monoclonal antibody in clinical development in the world. This Phase 2 compound has potential application in a wide range of autoimmune and inflammatory diseases, such as nonalcoholic steatohepatitis (NASH), ulcerative colitis, multiple sclerosis, type-1 diabetes (T1D), crohn’s disease, psoriasis and rheumatoid arthritis, where modulation of a T-cell response is desirable.
Forward-Looking Statements
Certain statements made in this announcement are forward-looking statements. These forward-looking statements are not historical facts but rather are based on the Company’s current expectations, estimates, and projections about its industry; its beliefs; and assumptions. Words such as ‘anticipates,’ ‘expects,’ ‘intends,’ ‘plans,’ ‘believes,’ ’seeks,’ ‘estimates,’ and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company’s control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.
Cited References
- Weiner HL et al. Oral tolerance. Immunol Rev. 2011; 241(1):241-259
- Ochi H, Abraham M, Ishikawa H et al. New immunosuppressive approaches: Oral administration of CD3-specific antibody to treat autoimmunity. J Neurol Sci 2008; 274(1- 2):9-12
- Mayo, L et al. IL-10-dependent TrI cells attenuate astrocyte activation and ameliorate chronic central nervous system inflammation. Brain. 2016: 139; 1939-1957
- Kuhn C. and Weiner HL. Therapeutic anti-DC3 monoclonal antibodies: from bench to bedside. Immunotherapy 2016; 8(8):889-906
- Wu, H.Y, Quintana, F.J and Weiner, H.L. Nasal Anti-CD3 Antibody Ameleorates Lupus by Inducing an IL-10-Secreting CD4+CD25-LAP+ Regulatory T cell and Is Associated with Down-Regulation of IL-17+CD4+ICOS+CXCR5+ Follicular Helper T Cells. J Immunol 2008; 181:6038-6050
For further enquiries:
Tiziana Life Sciences plc +44 (0)20 7495 2379 Gabriele Cerrone, Chairman and founder
Cairn Financial Advisers LLP (Nominated adviser) +44 (0)20 7213 0883
Liam Murray / Jo Turner
| Shore Capital (Broker)
Andy Crossley / Antonio Bossi
|
+44 (0)20 7601 6125 |
Receive news and updates from Tiziana Life Sciences plc by signing up to get email alerts straight to you on https://ir.tizianalifesciences.com