TIDMROQ
RNS Number : 5063D
Roquefort Therapeutics PLC
22 June 2023
22 June 2023
Roquefort Therapeutics plc
("Roquefort Therapeutics" or the "Company")
RNA Medicine Shows In Vitro Anti-Cancer Efficacy
Further preclinical progress
Roquefort Therapeutics (LSE:ROQ), the Main Market listed biotech
company focused on developing first-in-class medicines in the high
value and high growth oncology market, is pleased to announce the
progress of its anti-cancer RNA oligonucleotide program targeting
Midkine expressing cancers producing >90% in vitro efficacy (at
the mRNA level) in human liver and neuroblastoma cancer cells.
Following the announcement on 5 December 2022 of the Company's
presentation of its Midkine RNA oligonucleotide program results at
the European Society of Medical Oncology Asia conference, the
development of its oligonucleotide portfolio has continued. This
work has been conducted through strategic research partnerships at
the Faculty of Medicine and Health at the University of Sydney and
the Immune Oncology Laboratory at the School of Biomedical
Sciences, University of New South Wales (UNSW).
The team led by Professor Mark Molloy at the University of
Sydney, detected a novel peptide corresponding to the truncated
Midkine protein from cancer cells. This in vitro proof-of-concept
experiment utilised advanced proteomic analysis using mass
spectrometry. The study confirmed that the Company's novel
anti-sense oligonucleotides produced a novel non-functional Midkine
protein . Non-functional Midkine protein has been shown to produce
>90% in vitro efficacy (at the mRNA level) in human liver cancer
and neuroblastoma cancer cells [1] .
The UNSW team led by Scientia Associate Professor Orazio
Vittorio and Dr. Filip Michniewicz has continued this line of
research to evaluate the optimal combination of oligonucleotides in
an in vitro model of h epatocellular carcinoma (HCC) liver cancer.
A proprietary combination of the Company's oligonucleotides
demonstrated in vitro efficacy in HCC cells producing a significant
reduction in full length Midkine and generated a novel
non-functional Midkine. HCC is the fourth-leading cause of cancer
mortality worldwide and accounts for 90% of liver cancers ([2])
with an estimated market size of $8 billion (7% CAGR) ([3]) .
Elevated Midkine has been associated with progression, metastasis
and chemotherapy resistance in liver cancer ([4]) and because of
the limited efficacy of conventional therapy ([5]) , the 5-year
survival rate is just 21% (American Cancer Society) ([6]) .
The Company's anti-cancer RNA oligonucleotide program will now
progress into in vivo studies which are planned to complete in Q4
2023.
Scientia Associate Professor Orazio Vittorio, Head of Immune
Oncology Lab, UNSW, commented:
"As researchers in cancer biology, our recent experiments have
unveiled a promising breakthrough in liver cancer treatment.
Through the utilization of these novel oligonucleotides, we have
achieved remarkable in vitro efficacy, successfully inducing a
significant reduction in full-length Midkine and generating a
non-functional Midkine variant within liver cancer cells. This
discovery holds immense potential for patients battling liver
cancer, offering a new avenue for therapeutic intervention. "
Ajan Reginald, Roquefort Therapeutics, Chief Executive Officer,
commented:
"Liver cancer is growing with incidences forecast to increase to
one million new patients per year by 2025[7]. It remains an area of
high unmet medical need with a 5-year survival of only 21% ([8])
because the existing medicines have limited effect ([9]) . Midkine
is associated with progression, metastasis and resistance ([10])
and we feel this is a cancer in which we should focus our portfolio
of Midkine targeting medicines including antibodies, mRNA and the
anti-sense oligonucleotides. These oligonucleotides attack a
different Midkine region versus the antibodies and mRNA, and this
diversity of targeting may be helpful in developing mono or
combination therapies.
We are delighted with the progress of the Midkine portfolio
through our accelerated development process which was completed on
time and in budget. The planned next steps for the oligonucleotide
program are in vivo studies, in parallel with our mRNAs in a highly
synergistic and efficient set of in vivo studies in Q3-4 2023.
The pre-clinical progress across all our programs is highly
encouraging and within budget and in-keeping with our strategy and
we look forward to updating shareholders on our pre-clinical and
business development progress in due course."
-ENDS-
Enquiries:
Roquefort Therapeutics plc
Stephen West (Chairman) / Ajan +44 (0)20 3918
Reginald (CEO) 8633
Hybridan LLP (Joint Broker)
Claire Louise Noyce
Optiva Securities Limited (Joint +44 (0)20 3764
Broker) 2341
+44 (0)20 3411
Christian Dennis 1881
Buchanan (Public Relations)
Ben Romney / Jamie Hooper / George +44 (0)20 7466
Beale 5000
LEI: 254900P4SISIWOR9RH34
About Roquefort Therapeutics
Roquefort Therapeutics (LSE:ROQ) is a Main Market listed biotech
company developing first in class drugs in the high value and high
growth oncology segment prior to partnering or selling to big
pharma.
Since listing in March 2021, Roquefort Therapeutics has
successfully acquired Lyramid Pty Limited, a leader in the
development of medicines for a new therapeutic target, Midkine (a
human growth factor associated with cancer progression), and most
recently acquired Oncogeni Ltd, founded by Nobel Laureate Professor
Sir Martin Evans, which has developed two families of innovative
cell and RNA oncology medicines.
Roquefort Therapeutics' portfolio consists of five fully funded,
novel patent-protected pre-clinical anti-cancer medicines. The
highly complementary profile of five best-in-class medicines
consists of:
-- Midkine antibodies with significant in vivo efficacy and toxicology studies;
-- Midkine RNA therapeutics with novel anti-cancer gene editing action;
-- Midkine mRNA therapeutics with novel anti-cancer approach;
-- STAT-6 siRNA therapeutics targeting solid tumours with significant in vivo efficacy; and
-- MK cell therapy with direct and NK-mediated anti-cancer action
For further information on Roquefort Therapeutics, please visit
www.roquefortplc.com and @RoquefortTherap on Twitter.
[1]
https://www.esmo.org/meeting-calendar/past-meetings/esmo-asia-congress-2022
([2]) https://www.nature.com/articles/s12276-020-00527-1
([3])
https://www.grandviewresearch.com/industry-analysis/liver-cancer-diagnostic-market
([4]) Gowhari Shabgah, A, Ezzatifar, F, Aravindhan, S, et al.
Shedding more light on the role of Midkine in hepatocellular
carcinoma: New perspectives on diagnosis and therapy. IUBMB Life.
2021; 73: 659- 669. https://doi.org/10.1002/iub.2458
([5]) Deng Z, Yang H, Tian Y, Liu Z, Sun F, Yang P. An OX40L
mRNA vaccine inhibits the growth of hepatocellular carcinoma. Front
Oncol. 2022 Oct 13;12:975408. doi: 10.3389/fonc.2022.975408. PMID:
36313716; PMCID: PMC9606466.
([6])
https://www.cancer.org/cancer/types/liver-cancer/detection-diagnosis-staging/survival-rates.html
[7] Lu K, Fan Q, Zou X. Antisense oligonucleotide is a promising
intervention for liver diseases. Front Pharmacol. 2022 Dec
9;13:1061842. doi: 10.3389/fphar.2022.1061842. PMID: 36569303;
PMCID: PMC9780395.
([8])
https://www.cancer.org/cancer/types/liver-cancer/detection-diagnosis-staging/survival-rates.html
([9]) Deng Z, Yang H, Tian Y, Liu Z, Sun F, Yang P. An OX40L
mRNA vaccine inhibits the growth of hepatocellular carcinoma. Front
Oncol. 2022 Oct 13;12:975408. doi: 10.3389/fonc.2022.975408. PMID:
36313716; PMCID: PMC9606466.
([10]) Gowhari Shabgah, A, Ezzatifar, F, Aravindhan, S, et al.
Shedding more light on the role of Midkine in hepatocellular
carcinoma: New perspectives on diagnosis and therapy. IUBMB Life.
2021; 73: 659- 669. https://doi.org/10.1002/iub.2458
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June 22, 2023 02:00 ET (06:00 GMT)
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