Positive Phase 2b results in 2nd line melanoma

Highlight Therapeutics announces positive preliminary results from Phase 2b study of BO-112 + anti-PD1 in confirmed anti-PD1 progressor melanoma patients at SITC

Breakthrough cancer treatment shows best-in-class potential to open up 2^nd-line immunotherapy market to anti-PD1 resistant patients

• BO-112 demonstrates potential as best-in-class therapy to overcome anti-PD1 resistance in melanoma patients whose disease has progressed on prior anti-PD-1 treatment
• Primary endpoint met with a 27% Overall Response Rate (ORR), 8% Complete Responses (CR) & 65% Disease Control Rate (DCR) substantially exceeding current standard of care; further improvements anticipated over one year follow up
• Hard-to-treat mucosal melanoma patients achieved 66% ORR and 100% DCR
• Durable responses and manageable safety profile, with no patients discontinuing due to adverse events
• Preparation for pivotal trial based on FDA guidance underway; potential for use in multiple solid cancers resistant to anti-PD1 inhibitors, and with different anti-PD1 combinations

MADRID, Spain, Nov. 12, 2021 (GLOBE NEWSWIRE) -- Highlight Therapeutics, ("Highlight"), a clinical-stage biopharmaceutical company developing RNA-based therapies against cancer, today announced positive preliminary results of a Phase 2 study of intratumoral administration of BO-112 with pembrolizumab in patients with advanced melanoma whose disease had progressed on first-line anti-PD1-based therapy. BO-112 is a dsRNA agonist targeting anti-PD1 resistance, which has been successfully tested in several previous Phase 1b studies.

Initial results of the study were presented today (12^th November) in a late-breaking session at The Society for Immunotherapy of Cancer (SITC) 36th Annual Meeting in Washington DC, Nov. 10-14, 2021. (LBA Poster Abstract (961): Preliminary results of a phase 2 study of intratumoral administration of BO-112 with pembrolizumab in patients with advanced melanoma that have progressive disease on anti-PD-1-based therapy, presented in the Poster Hall in Washington, D.C. and the virtual ePoster Hall beginning Friday, Nov. 12 at 7 a.m. EST).

"These are potentially game-changing results showing that BO-112 can rescue melanoma patients who have failed first-line immune-therapy with anti-PD1," saidDrCarlos Paya, Executive Chairman of Highlight Therapeutics. "Anti-PD1 basedimmunotherapy has revolutionized oncology treatments, but only a fraction of patients initially respond and many of these patients progress thereafter. These initial Phase 2 results show that BO-112 combined with a leading PD1 inhibitor rescue around 65% of anti-PD1 failing patients, making many of them respond to the combined treatment. These much-anticipated outcomes demonstrate BO-112's potential as a best-in-class therapy for melanoma patients whose disease has progressed on prior anti-PD1 treatment, and we now look forward to further clinical studies, not only in melanoma but in other major tumor types in which anti-PD1 resistance is also an issue."

Anti-PD1 therapies are valued at approximately $24 billion¹ and are used across most solid tumors but currently fewer than 20% of all cancer patients benefit from first-line anti-PD1 treatment. BO-112 in combination with anti-PD1 therapy is designed to resensitize tumors to anti-PD1 treatment through improved antigen presentation, enhanced T-cell infiltration and increased MHC-1 and PDL1 expression by the tumor itself.

"This initial data is very encouraging and has the potential to change medical practice," said Dr. Marisol Quintero, CEO of Highlight Therapeutics. "The preliminary ORR of 27%, including 8% Complete Responses, already exceeds current standard of care, such as the use of anti-CTLA-4. Based on the mode of action and experience from previous BO-112 Phase 1 studies, additional follow-up of these patients is expected to deliver further improvements in ORR. We are also encouraged by the excellent safety profile, with no patients discontinuing the study due to adverse events. Highlight Therapeutics is in the planning stage of a pivotal Phase 3 trial, due to begin in 2022, and we look forward to opening discussions with potential partners to explorecombinations with anti-PD1s."

Highlight Therapeutics and Merck, known as MSD outside the United States and Canada, conducted an open-label, single arm study to evaluate the efficacy & safety of intra-tumoral administration of BO-112 + pembrolizumab in mucosal, acral and cutaneous melanoma patients whose disease had progressed, confirmed by two consecutive CT scans. The study recruited 42 patients in France and Spain, with recruitment completed by August 24, 2021. Patients included those with high LDH levels, which are often associated with poor response rates and have been excluded from comparable clinical trials.

The preliminary analysis shows:

• Primary endpoint (ORR by independent reviewer) has been met
• With a median follow up of three months, there is a clear clinical benefit in patients with confirmed anti-PD1-resistant melanoma, with a 27% ORR and a 65% DCR, superior to 2nd line Standard of Care in stage III/IV melanoma of ~8% (continuing with anti-PD1 Ab) or 13% (second line ipilimumab).
• Three hard-to-treat mucosal melanoma patients have achieved an ORR of 66% and DCR of 100%
• High baseline LDH levels (>3xULN) predict progressive disease
• Responses and SD are durable
• Study treatment has a manageable safety profile, with no patients discontinuing due to adverse events

Next steps in the development of BO-112 include:

• Initiation of a pivotal Phase 3 study in 2nd-line melanoma is planned in 2022 following discussions with regulatory agencies in the US and Europe
• Highlight Therapeutics has initiated strategic partnerships discussions with anti-PD1 companies interested in enhancing their anti-PD1 market potential
• Initial data from a sponsor-initiated Phase 1B trial by UCLA evaluating BO-112 + pembrolizumab in anti-PD1 resistant hepatocellular carcinoma currently recruiting patients is expected in 2022
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1.     IQVIA Global Oncology Report 2020

For more information, please contact:

Highlight Therapeutics S. L.	info@highlighttherapeutics.com
Marisol Quintero, CEO
Mo PR Advisory	Tel: +44 (0) 7876 444977 / 07860 361746
Mo Noonan/Jonathan Birt
Consilium Strategic Communications	Tel: +44 7921 697654
Chris Gardner	cgardner@consilium-comms.com

Notes to editors

About Highlight Therapeutics

Highlight Therapeutics, formerly known as Bioncotech Therapeutics, is a private, clinical-stage company dedicated to unlocking the full potential of immuno-oncology. Our lead drug candidate BO-112 is a best-in-class RNA-based therapy which has been demonstrated to initiate a powerful immune response, leveraging a unique multi-target approach to turn 'cold' tumors 'hot' and therefore visible to the immune system. It has the potential to rescue patients who are resistant to current checkpoint inhibitor therapy, a very large market opportunity. BO-112 is currently being investigated in a range of clinical trials as a monotherapy and in combination with checkpoint inhibitors. In addition to in-house research, Highlight Therapeutics has a number of external collaborators, including Merck & Co and UCLA.

For more information, please visit www.highlighttherapeutics.com

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