REG - Roquefort Theraptcs. - RNA Medicine Shows In Vitro Anti-Cancer Efficacy

Roquefort TherapeuticsAnnouncement

22/06/2023 07:00

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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 hepatocellular
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 Reginald (CEO)  +44 (0)20 3918 8633

 Hybridan LLP (Joint Broker)


 Claire Louise Noyce                            +44 (0)20 3764 2341

 Optiva Securities Limited (Joint Broker)
 Christian Dennis                               +44 (0)20 3411 1881

 Buchanan (Public Relations)

 Ben Romney / Jamie Hooper / George Beale       +44 (0)20 7466 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 (http://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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