Lund, Sweden (Press Release) – Active Biotech (NASDAQ STOCKHOLM: ACTI) announces that the Board of Directors, has approved a new direction for the com­pany. This de­ci­sion follows a detailed oppor­tu­ni­ty analysis of the com­pany’s wholly owned clin­i­cal lead assets, laquinimod and tasquinimod. Based on assess­ment of the many scientific pub­li­ca­tions and extensive pre-clinical and clin­i­cal data accumulated, an analysis of the commercial attractiveness of dif­fer­en­t clin­i­cal in­di­ca­tion oppor­tu­ni­ties to Active Biotech, as well as an ex­ternal advisory chal­lenge, new focused target in­di­ca­tions and corporate priorities have been defined.

Tasquinimod will be ad­vanced in a new academic part­ner­ship, as an immuno­modu­la­tory prod­uct with a novel mech­a­nism of action, for the treat­ment of multiple myeloma. Laquinimod will be ad­vanced, as an immuno­modu­la­tory prod­uct with a novel mech­a­nism of action, for use as a topical agent in inflammatory eye dis­eases and as an oral treat­ment of patients with Crohn’s dis­ease.

Going for­ward, Active Biotech has a project port­folio comprising naptumomab, partnered to NeoTX and cur­rently in Phase 1b/2 for the treat­ment of ad­vanced solid tumors, and the new clin­i­cal and pre-clinical pro­grams for tasquinimod and laquinimod.

No further work on the prior clin­i­cal pro­grams within multiple sclerosis or Huntington’s dis­ease for laquinimod or solid tumors for tasquinimod will be under­taken. In addi­tion, Active Biotech will for now put on hold the paquinimod and SILC-projects, to create a clear focus on the prioritized activities, and no further communication in rela­tion­ to these assets is ex­pec­ted.

Opportunity analysis and new pipe­line projects

Over the past 6 months, we have under­taken a detailed evaluation of tasquinimod and laquinimod from a tech­ni­cal as well as a commercial perspective, to assess poten­tial value-enhancing paths for­ward for the com­pany in devel­op­ing these assets. The analyses in­cluded updated scientific insights on the mech­a­nism of action of the com­pounds, as well as analyses of how best to leverage the existing clin­i­cal safety data. A broad network of inter­na­tional expert advisors has been engaged in this process, to ensure adequate ex­ternal chal­lenge of ideas and directions devel­oped. The new in­di­ca­tions now being ad­vanced for tasquinimod and laquinimod rep­re­sent dis­eases with sig­nif­i­cant unmet medical need and commercial poten­tial where IP has been secured or filed.

The new project port­folio com­prises poten­tial high-value in­di­ca­tion oppor­tu­ni­ties for which pre-clinical efficacy and safety results, in addi­tion to a com­pre­hen­sive CMC documentation, already has been generated. The com­pany´s new strat­e­gy aims at ad­vanc­ing the projects in well-defined focus areas by leveraging existing results in com­bi­na­tion with smaller proof-of-concept or con­firmatory phase 2 studies to enable early and cost-effective value crystallization to Active Biotech through partnering/out-licensing.

- Tasquinimod will be ad­vanced as a new prod­uct for treat­ment of multiple myeloma

Based on our analysis and ongoing business devel­op­ment activities, we today announce the for­ma­tion of an academic part­ner­ship with The Perelman School of Medicine, University of Pennsylvania for the devel­op­ment of tasquinimod as a new immuno­modu­la­tory prod­uct for the treat­ment of multiple myeloma. Extensive pre­clin­i­cal studies per­formed in col­lab­o­ration with the Wistar Institute in Philadelphia, during the past 24 months, provide clear sup­port for the ad­vancement of tasquinimod in multiple myeloma, these data will be pub­lished in peer-reviewed journals. The parties are preparing to ini­ti­ate a phase 1b/2a study in Q2-2020 and ex­pec­t the first patient treated in Q3-2020. This pro­gram has also re­ceived funding from the Leukemia & Lymphoma Society in United States.

- Laquinimod as a new prod­uct for use in eye disorders

Our analyses have revealed an exciting pre-clinical evi­dence base sup­port­ing use of laquinimod for treat­ment of the two eye disorders Wet AMD and Uveitis which has not yet been pub­lished. Our focus the coming 12 months will be, on a pre-clinical study basis, to in­crease our under­stand­ing of the thera­peutic poten­tial of laquinimod through addi­tional pre-clinical studies, and to define how best to de­vel­op laquinimod as a topical agent within these dis­eases, as sug­gested from the initial pre-clinical data.

- Laquinimod as a new prod­uct in Crohn’s dis­ease

We have also decided to ad­vance laquinimod for use in Crohn’s dis­ease, as an immuno-modulatory agent with a novel mech­a­nism of action, an in­di­ca­tion for which a prior clin­i­cal Phase 2a study provides compelling data. Our review of the extensive pre­clin­i­cal scientific profiling of laquinimod in models of gastro-intestinal disorders, further sup­ports a poten­tial role in treat­ment of Crohn’s dis­ease. We will during the coming 12 months refresh the prior regu­la­tory advice re­ceived from the FDA, and explore possible part­ner­ship modalities, in­clud­ing academic part­ner­ships, to ad­vance the evaluation of laquinimod in this in­di­ca­tion.

“After a com­pre­hen­sive analysis of our project port­folio, we are now ad­vanc­ing our lead projects in a new direction to maximize the value creation oppor­tu­ni­ty for Active Biotech. We are very enthusiastic about devel­op­ing laquinimod and tasquinimod further in these new in­di­ca­tions with unmet medical need and where existing data can be optimally used” says Helén Tuvesson, CEO of Active Biotech. “The commercial value that each of these in­di­ca­tions rep­re­sents is sub­stan­tial, and our focus is to realize this in a cost-effective manner leveraging academic part­ner­ships and our network”, con­tinues Helén Tuvesson.

Financial impact

At year-end 2019 avail­able cash is scheduled to finance ongoing activities in the new direction through 2021. In addi­tion, Active Biotech is eval­u­ating corporate devel­op­ment oppor­tu­ni­ties to broaden the share­holder base, and to strengthen the project port­folio.

“The extensive scientific profiling, and prior investments into large clin­i­cal pro­grams for laquinimod and tasquinimod, now enables a faster and more focused devel­op­ment of these projects for dif­fer­en­t clin­i­cal appli­ca­tions. We have leveraged this sub­stan­tial evi­dence base in a structured manner to sup­port setting our port­folio priorities, and look for­ward to executing on this new directions” says Michael Shalmi, Chairman of the Board of Active Biotech.

In conjunction with the Annual General Meeting of Active Biotech in May, the com­pany will invite interested parties to join a separate Investor Session, in which the medical need and scientific sup­port of the plans and activities now defined for tasquinimod and laquinimod, will be pre­sented. Further details will be communicated later.

About tasquinimod and laquinimod

Laquinimod, is an orally admin­istered small molecule with unique immuno­modu­la­tory properties that pre­vi­ously has been devel­oped primarily within neurodegenerative dis­eases. Tasquinimod is an immuno­modu­la­tory and anti-angiogenic com­pound, primarily studied in solid cancer in­di­ca­tions, that affects the tumor's ability to grow and spread. During its years of ad­vanced prod­uct devel­op­ment, clin­i­cal efficacy and safety data on laquinimod and tasquinimod was estab­lish­ed in more than 5000 and 1500 patients, rep­re­senting more than 14000 and 650 patient-years of exposure, re­spec­tive­ly. In addi­tion, extensive datasets spanning full-scale manu­fac­tur­ing and pre-clinical safety data, in sup­port of regu­la­tory filings of multiple sclerosis for laquinimod and ad­vanced prostate cancer for tasquinimod, has also been generated

About multiple myeloma

Multiple myeloma is an incurable blood cancer in which ab­nor­mal plasma cells in the bone mar­row grow un­con­trol­lably while other blood forming cells such as white and red blood cells and blood platelets are sup­pressed. This leads to anemia, in­fec­tions, destruction of bone tissue and progressive loss of renal function. Despite new treat­ments have greatly im­proved sur­vival of multiple myeloma patients, the biological heterogeneity of the dis­ease and the emergence of drug resistance is a major chal­lenge, and the medical need of inno­va­tive treat­ment modalities remains high.

In 2017, 81000 new cases of multiple myeloma were diag­nosed in the eight major mar­kets. The global sales* of drugs for multiple myeloma totaled USD 16 billion in 2017 and sales are ex­pec­ted to in­crease 48% by 2026.

About age-related macular degeneration and uveitis

Age-related macular degeneration (AMD) is caused by damage to the macula or retina, which results in blurred or loss of vision in the center of the visual field and the leading cause of blindness in the Western world. In the neovascular AMD, the "wet" form of ad­vanced AMD (wAMD), ab­nor­mal blood vessel growth in the eye leads to vision loss. Bleeding, leaking and scarring from these vessels cause irreversible damage and retinal detachment. About 10-20 % of late-stage AMD cases progress into wAMD, and is responsible for 90% of acute blindness due to age-related macular degeneration. Standard of care of wAMD is frequent in­vasive intravitreal injections of anti­bodies targeting the vascular endothelial growth factor (VEGF). However, even with these ther­a­pies, there is a great unmet need of new effective treat­ments with less in­vasive and more con­ve­nient de­livery modalities.

Around 3 million people in the seven major mar­kets were esti­mated to have wAMD in 2018. The global sales* of drugs for wAMD totaled USD 6.3 billion in 2018 and sales are ex­pec­ted to in­crease 67% by 2026.

Uveitis is the inflammation of the uveal tract (iris, ciliary body, & choroid), but can also cause inflammation of nearby tissues, such as the retina, the optic nerve and the vitreous humor. General vision problems, floaters-spots in the eye, eye pain and redness, photophobia, headache, small pupil, alteration of iris color are common symp­toms. If left untreated, uveitis can lead to severe eye problems, in­clud­ing blindness, cataracts, glaucoma, damage to the optic nerve, and detachment of the retina. In non-infectious uveitis there is a need for new ther­a­pies to avoid the abundant com­pli­ca­tions of long-term corticosteroid use.

Around 1 million people in the seven major mar­kets were esti­mated to have uveitis in 2017. The global sales* of drugs for uveitis totaled USD 615 million in 2017 and sales are ex­pec­ted to in­crease 70% by 2026.

About Crohn’s dis­ease

Crohn’s dis­ease is an auto­immune dis­ease, in which the auto­immune activity produces inflammation in the gastro­in­tes­ti­nal tract and is classified as an inflammatory bowel dis­ease (IBD). The symp­toms of the dis­ease can vary sig­nif­i­cantly among afflicted in­di­vid­uals. The main gastro­in­tes­ti­nal symp­toms are abdominal pain, diarrhea, or weight loss. Crohn’s dis­ease can also cause com­pli­ca­tions outside of the gastro­in­tes­ti­nal tract such as skin rashes, arthritis, and inflammation of the eye. Although treat­ment with pharma­ceu­tical agents and/or surgery can lead to clin­i­cally sig­nif­i­cant im­prove­ments in Crohn’s dis­ease, relapse is the major problem and there­fore the main­te­nance of tissue heal­ing remains the key chal­lenge.

During 2018 the number of treated patients with Crohn’s dis­ease in the seven major mar­kets amounted to approx­i­mately 0,5 million. The global sales* of drugs for Crohn’s dis­ease totaled USD 12.4 billion in 2018 and sales are ex­pec­ted to in­crease 32% by 2026.

* As esti­mated by Global Data

Source: Active Biotech.