REG - Roquefort Theraptcs. - Antibody Program Results & Orphan Drug Indication

Roquefort TherapeuticsAnnouncement

19/06/2023 07:00

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RNS Number : 0733D  Roquefort Therapeutics PLC  19 June 2023

19 June 2023

 

Roquefort Therapeutics plc

("Roquefort Therapeutics" or the "Company")

 

Antibody Program Results & Orphan Drug Indication

 

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
antibody programs targeting Midkine expressing cancers.

Following the announcement in January 2023 where the Company announced the
Midkine antibody programs, targeting metastatic breast cancer and metastatic
lung cancer, successfully demonstrated in vivo safety, Roquefort Therapeutics
has continued the development of its antibody portfolio with the Company's
research partners at La Trobe University. The Company is now releasing the
first in vivo efficacy results for its anti-Midkine antibodies CAB-101 (ROQA2)
and CAB-102 (ROQA1), including a new program for an osteosarcoma orphan drug
indication.

CAB-101 and CAB-102 are the lead programs in the Company's family of patented
humanised antibodies, which were developed in-house, to target
Midkine-expressing solid cancers.  The in vivo efficacy study tested the
anti-cancer killing ability of CAB-101 and CAB-102 in a validated experimental
model of osteosarcoma. Treatment with CAB-101 was found to produce a
statistically significant reduction in lung metastasis, and CAB-102 was found
to reduce proliferation (growth rate) of the primary tumour.  The more
detailed experimental results remain under embargo pending publication at a
leading cancer research conference.

CAB101 and CAB102 treat solid cancers, the market for which was estimated to
be $32 billion in 2021 and growing with a 7.8% CAGR 1  (#_ftn1) . Osteosarcoma
is the Company's first orphan drug indication and reflects the strategic
decision to target cancer niches in which, there remains a high unmet clinical
need (24% to 64% 5-year survival rate) 2  (#_ftn2) , an accelerated
development pathway 3  (#_ftn3) and the potential to offer a best-in-class
treatment in a significant market niche.

Medicines to treat an orphan drug indication may be awarded market exclusivity
for 7 years in the USA and 10 years in the EU and UK 4  (#_ftn4) , tax credits
for the clinical drug testing cost, fee reductions and, on average, will have
a significantly higher success rate in clinical trials 5  (#_ftn5) .

Osteosarcoma is the eighth-most common form of childhood cancer, comprising
2.4% of all malignancies in paediatric patients, and about 20% of all primary
bone cancers 6  (#_ftn6) , with a market estimated to reach $790 million by
2025 (6% CAGR) 7  (#_ftn7) . Midkine is over-expressed in osteosarcoma and the
level of Midkine expression is correlated with poor prognosis 8  (#_ftn8) and
therefore, represented a promising target for anti-Midkine medicines.

Commenting on the antibody program results, Ajan Reginald, Chief Executive
Officer, said:

"We decided to target metastatic osteosarcoma because of the high unmet
medical need. There is a very poor 5-year survival rate of just 24%, and this
poor survival is associated with over-expression of Midkine. Our rationale was
that by targeting Midkine, we would be able to attack both primary tumours and
metastasis. The in vivo results vindicate this approach and demonstrate the
potential for a best-in-class medicine. We look forward to providing further
information once the detailed results have been published.

This strategy to focus on areas of high unmet medical need is underpinned by
the orphan drug incentives (including market exclusivity, efficient clinical
trials and tax incentives) and the higher market valuation and return on
investment for the investors in companies with approved orphan medicines.
Finally, this approach seems to reduce risk, with significantly higher
clinical trial success rates particularly in phase 1 and 2 trials particularly
with a biomarker, which in our case is Midkine.

Therefore, this is a particularly promising scientific and commercial strategy
which was delivered on time and on budget. We expect to announce more updates
on our clinical progress and business development activities over the next
quarter."

-ENDS-

Enquiries:
 Roquefort Therapeutics plc
 Stephen West (Chairman) / Ajan Reginald (CEO)  +44 (0)20 3290 9339

 Hybridan LLP (Joint Broker)

 Claire Louise Noyce                            +44 (0)203 764 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  (#_ftnref1)
https://finance.yahoo.com/news/solid-tumor-testing-market-worth-103300895.html

 2  (#_ftnref2)
https://www.cancer.net/cancer-types/osteosarcoma-childhood-and-adolescence/statistics#:~:text=If%20the%20cancer%20has%20spread,relative%20survival%20rate%20is%2024%25.

 3  (#_ftnref3)
https://www.medpace.com/wp-content/uploads/2023/03/Article-Regulatory-Strategies-and-Considerations-for-Orphan-and-Pediatric-Drug-Designations.pdf

 4  (#_ftnref4)
https://www.propharmagroup.com/thought-leadership/orphan-drug-designations-in-the-u-s-and-eu

 5  (#_ftnref5)
https://www.sciencedirect.com/science/article/pii/S0040595720300093

 6  (#_ftnref6)
https://www.marketwatch.com/press-release/osteosarcoma-market-growth-2023-2030-2023-05-09

 7  (#_ftnref7)
https://www.barchart.com/story/news/14577891/osteosarcoma-market-size-share-revenue-and-structure-forecast-to-2030

 8  (#_ftnref8) Biochem Biophys Res Commun 2007 Jul 6;358(3):757-62.  doi:
10.1016/j.bbrc.2007.04.183. Epub 2007 May 7

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