A Swedish biotech is tar­get­ing throm­bo­sis us­ing the body's own de­fense sys­tem to pre­vent blood clots with low­er risk of bleed­ing as­so­ci­at­ed with cur­rent an­tithrom­bot­ic ther­a­pies

Every two sec­onds a per­son is af­fect­ed by throm­bo­sis. Blood clots form­ing and block­ing the cir­cu­la­tion in veins and ar­ter­ies is the num­ber one cause of death glob­al­ly, with com­pli­ca­tions such as my­ocar­dial in­farc­tion and stroke ac­count­ing for the ma­jor­i­ty of fa­tal­i­ties. Pa­tients at risk of de­vel­op­ing blood clots re­ceive pre­ven­tive treat­ment with an­tithrom­bot­ic agents of­ten called blood-thin­ning med­i­cines such as War­farin, as­pirin and the lat­er in­tro­duced NOACs. These drugs have been the stan­dard of care for decades, but they all come with a high price to pay: the risk of un­con­trolled bleed­ing.

Avail­able drugs on the mar­ket are es­sen­tial­ly de­signed to weak­en the body’s co­ag­u­la­tion sys­tem, the mech­a­nism that stops the bleed­ing when we cut our­selves. But by in­hibit­ing co­ag­u­la­tion the risk of bleed­ing is sig­nif­i­cant­ly in­creased. Up to 25% of pa­tients on cur­rent an­tithrom­botics ex­pe­ri­ence bleeds, out of which 3% are char­ac­ter­ized as se­vere.

Due to the risk of caus­ing se­ri­ous bleeds the dose lev­els of cur­rent an­tithrom­bot­ic drugs are gen­er­al­ly low, which in turn re­duces their clot-pre­ven­tive ef­fi­ca­cy. Thus, the med­ical need for ef­fi­cient drugs that could avoid putting pa­tients in a con­stant easy-to-bleed state is huge, as is the glob­al mar­ket.

In the US alone, throm­bo­sis pre­ven­tion drugs make up for a 10Bn dol­lar mar­ket. And ac­cord­ing to a re­cent­ly pub­lished re­port from Zion Re­search the glob­al throm­bo­sis mar­ket val­ue is es­ti­mat­ed to gen­er­ate rev­enue of up to 40.9Bn dol­lars by 2024, grow­ing at a CA­GR of 9.3% be­tween 2018 and 2024.

Fo­cus­ing on this po­ten­tial block­buster mar­ket is Cereno Sci­en­tif­ic, a mi­cro-cap biotech op­er­at­ing out of Swe­den’s lead­ing life sci­ence hub at As­traZeneca in Gothen­burg.

Start­ing out in 2012 with a team of car­dio­vas­cu­lar ex­perts from Gothen­burg’s Sahlgren­s­ka Hos­pi­tal/Acad­e­my, ul­ti­mate­ly re­cruit­ing for­mer As­traZeneca vet­er­ans and with CEO Sten R. Sörensen at the helm, Cereno brings a brand new ap­proach to pre­vent­ing throm­bo­sis. Rather than weak­en­ing the co­ag­u­la­tion sys­tem and putting pa­tients at risk of bleed­ing, its drug can­di­date CS1 is de­signed to boost the body’s nat­ur­al de­fense mech­a­nism against blood clots, called the fib­ri­nolyt­ic sys­tem.

Cereno’s con­cept is based on a new ad­vanced con­trolled re­lease for­mu­la­tion of val­proic acid, a well-known sub­stance which has been used in health care for treat­ing epilep­sy for decades. By stim­u­lat­ing the pro­duc­tion of t-PA, a sub­stance the body us­es to dis­solve blood clots and, si­mul­ta­ne­ous­ly de­creas­ing the lev­el of PAI-1, which is the main t-PA in­hibitor, CS1 would in­deed de­liv­er a dual im­pact on the clot­ting process.

Hav­ing se­cured fi­nanc­ing through a cou­ple of share is­sues fol­lowed by an IPO in 2016, the com­pa­ny has op­er­at­ed on an im­pres­sive­ly tight bud­get and yet com­plet­ed a phase I study on CS1. Pos­i­tive re­sults re­gard­ing safe­ty, phar­ma­co­ki­net­ic prop­er­ties and side ef­fects were re­port­ed in June this year.

In ad­di­tion to show­ing that the sub­stance is well tol­er­at­ed by pa­tients, one of the study’s key con­clu­sions was the ef­fect CS1 had on PAI-1, the bio­mark­er for risk of throm­bo­sis. The study clear­ly demon­strat­ed that CS1 sig­nif­i­cant­ly low­ers the lev­els of PAI-1 when at its high­est lev­el in the morn­ing. Ac­cord­ing to Cereno Sci­en­tif­ic, this con­firms that there is a very re­al pos­si­bil­i­ty of de­vel­op­ing a new, safe and ef­fi­ca­cious drug with a dou­ble mech­a­nism for fight­ing blood clots.

In mid-Sep­tem­ber Cereno Sci­en­tif­ic re­ceived a No­tice of Al­lowance from the US Patent Of­fice re­gard­ing in­tel­lec­tu­al prop­er­ty rights for the treat­ment con­cept and use of CS1 in the US.

The ap­proved patent pro­vides us with a plat­form for a sig­nif­i­cant mar­ket po­ten­tial in the US, the world’s largest drug mar­ket – a mar­ket that, for drug-re­lat­ed treat­ment of throm­bo­sis alone, has es­ti­mat­ed sales of ap­prox­i­mate­ly USD 10 bil­lion an­nu­al­ly and con­tin­ues to grow. We con­tin­ue to strength­en the pro­tec­tion for our treat­ment con­cept in line with the progress we make. For each mile­stone we achieve, the risk of our de­vel­op­ment re­duces while our tech­nol­o­gy and pipeline val­ue in­crease.

– Cereno Sci­en­tif­ic CEO Sten R. Sörensen

As of to­day, Cereno Sci­en­tif­ic is set to ad­vance its lead can­di­date in­to next phase and to this end has signed an agree­ment with OCT Group. Cereno Sci­en­tif­ic and OCT will col­lab­o­rate in car­ry­ing out a phase II study in­ves­ti­gat­ing the an­tithrom­bot­ic ef­fects of CS1 in pa­tients un­der­go­ing or­tho­pe­dic surgery. A frame­work is in place for the study and Cereno ex­pect to sub­mit all nec­es­sary ap­pli­ca­tions dur­ing the fourth quar­ter of 2018.

With re­gards to fi­nanc­ing, Cereno Sci­en­tif­ic has cho­sen to uti­lize con­vert­ible loans from the Eu­ro­pean High Growth Op­por­tu­ni­ties Se­cu­ri­ti­za­tion Fund. Un­der the agree­ment Cereno Sci­en­tif­ic can re­ceive up to 82 mil­lion SEK over a two-year pe­ri­od, with the pos­si­bil­i­ty to re­ceive an­oth­er 24.6 mil­lion SEK if war­rants are ex­er­cised. Al­though the arrange­ment has put pres­sure on the com­pa­ny’s stock price, man­age­ment’s ra­tio­nale for choos­ing this route has been to lock down a sta­ble source of cap­i­tal, thus se­cur­ing the re­sources to take CS1 through phase II.

Fol­low­ing com­ple­tion of the phase II study, Cereno Sci­en­tif­ic’s strat­e­gy is to out­li­cense CS1 to a larg­er phar­ma­ceu­ti­cal part­ner with the nec­es­sary re­sources to scale up the de­vel­op­ment process and bring­ing a safe an ef­fi­cient blood clot ther­a­py to­wards mar­ket launch – and to the mil­lions of pa­tients whom are still at risk of de­vel­op­ing life-threat­en­ing blood clots and se­ri­ous bleeds.

Cereno Sci­en­tif­ic is list­ed on Spot­light Stock Mar­ket

A pre­sen­ta­tion on CS1 from Cereno Sci­en­tif­ic can be viewed here.

For more in­for­ma­tion about the com­pa­ny, vis­it www.cereno­sci­en­tif­ic.se/en/