CombiGeneâs and Neurochaseâs preclinical study provides valuable information for the upcoming long-term studies in toxicology and biodistribution
STOCKHOLM â On June 28, 2021, CombiGene (âCombiGeneâ, âthe Companyâ) announced that the Company, together with its partner Neurochase, had started the work to optimize the administration of CG01 in a preclinical pilot study in a large animal model. CombiGene can today announce that the practical part of the study has now been completed.
In the study, the administration of CG01 has been evaluated in terms of, among other things, injection volume and injection rate. The study provides valuable information of the Neurochase injection device for the upcoming long-term studies in toxicology and biodistribution in large animals.
âCombiGene continues to break new ground within the framework of the CG01 project,â said Pernilla Fagergren, Project Manager at CombiGene. âNo one has previously developed a gene therapy for the treatment of drug-resistant focal epilepsy. We have now gained new knowledge that is valuable for the upcoming long-term studies in toxicology and biodistribution in large animals.â
Since CombiGene and Spark Therapeutics in October 2021 entered an exclusive collaboration and licensing agreement for CG01, the two companies have jointly reviewed the future development of the project with the ambition to establish the best path forward.
The most significant outcome of this review is the decision to expand the clinical development program to include clinics in the U.S. as well as in Europe. The U.S. is the worldâs largest pharmaceutical market and to establish a clinical presence there adds much further strength to the CG01 project.
In order to prepare CG01 to meet the needs of an expanded submission that also includes the U.S., the remaining preclinical program will be expanded and, in some parts, complemented with additional studies. In practice, this means that the preclinical part of CG01 will take longer to finalize.
During the remaining part of the preclinical program, all CG01-related R&D activities that CombiGene is running, internal as well as external, will be agreed upon and approved by Spark, who also assumes all agreed costs. As CG01 enters the clinical phase, Spark will take over the responsibility for the continuation of the project and thus also bear all costs during this development phase.
About CG01
CG01 is a unique gene therapy candidate aimed at a large patient population to solve a global need in epilepsy treatment. Epilepsy is a major global medical problem with approximately 47,000 drug-resistant patients with focal epilepsy estimated to be added each year across the US, EU4, UK, Japan, and China. CG01 is in a late preclinical stage. The production platform, jointly developed by CombiGene and its partners Cobra Biologics and Viralgen, is scalable and designed to provide material for preclinical and clinical, and future commercial production. CombiGene has signed an exclusive collaboration and licensing agreement for CombiGeneâs CG01 project with Spark Therapeutics.
Horizon 2020
CombiGeneâs lead project CG01 has received funding from the European Unionâs Horizon 2020 research and innovation program under grant agreement No 823282.
About Spark Therapeutics
At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We currently have four programs in clinical trials. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit www.sparktx.com, and follow us on Twitter and LinkedIn.
About Neurochase
www.neurochase.com
Neurochase, founded by Professor Steven Gill, aims to bring transformative targeted therapies to patients with neurological diseases using state of the art technology. The companyâs team specializes in creating bespoke therapeutic strategies for the direct delivery of therapies using Convection Enhanced Delivery (CED). Neurochase provides accurate, targeted and safe direct drug delivery to the CNS and develop globally scalable treatment strategies and solutions for the pharmaceutical and biotech industry. Neurochase Ltd. is a Registered Company Number 12428919.
CombiGeneâs pilot study, which is aimed at optimising the administration of the drug candidate CG01, has given the company valuable insights for future development steps. The path towards clinical studies, which will take place with support from partner Spark Therapeutics, thus takes another step forward. BioStock contacted the companyâs CEO Jan Nilsson who talks about the collaboration with Spark, and how the company views its intensified investment in business development.
Ser ut som de har ambisjoner om Ă„ bredde ut pipen. Og vi vet at de er en interessant aktĂžr for partnerskap ref Spark-dealen.
Kan bli en multibagger med tiden det her
In recent months, in addition to focusing on concluding the preclinical programme, you have intensified your focus on business development. Can you elaborate on this?
â Our focus in identifying new possible projects is based on two things. Technologically, we are most interested in AAV vectors because this is an area where we consider ourselves very knowledgeable. In terms of indications, we primarily look at diseases within the central nervous system and metabolic diseases where we also have good knowledge. At the same time, we will keep a very open attitude towards other technologies and indications and assess each potential project on its own merits. Identifying projects that could be of interest to CombiGene is a high priority within the company right now.
Er det mulighet for godis her imorgen??
Forventer vel egentlig ikke sÄ veldig mye nytt, men kanskje litt videre informasjon om tidslinjer og eventuelt om det er noe vi kan forvente oss fremover. Blir ogsÄ etterhvert itneressant Ä se hvordan burnraten blir nÄr sÄpass mye av kostnadene vil dekkes av spark. Ser veldig frem til de oppnÄr den fÞrste milestonen for cg01.
FjĂ€rde kvartalet oktober â december 2021
⹠NettoomsÀttning: 84 042 (0) TSEK.
âą Ăvriga rörelseintĂ€kter: 1 098 (3 171) TSEK.
âą Resultat efter finansiella poster: 57 448 (-10 964) TSEK.
âą Resultat per aktie: 2,90 (-1,22) SEK.
Period januari â december 2021
⹠NettoomsÀttning: 84 042 (0) TSEK.
âą Ăvriga rörelseintĂ€kter: 7 478 (12 029) TSEK.
âą Resultat efter finansiella poster: 20 965 (-29 551) TSEK.
âą Resultat per aktie: 1,21 (-3,31) SEK.
⹠Likviditet vid periodens utgÄng: 136 744 (48 895) TSEK.
⹠Soliditet vid periodens utgÄng: 95 (89) %.
⹠Styrelsen föreslÄr att ingen utdelning lÀmnas för rÀkenskapsÄret
Hot felt on dagen!
Nytt nyhetsbrev fra CombiGene
Hva er Ärsaken til det kraftige fallet de siste dagene? Er det bare makro? Det er vel ikke sÄ vanlig at en aksje faller sÄpass mye uten grunn?
Kraftige rekyler rundt om i biotek nÄ, men denne virker som er helt glemt. Etter mine beregninger har de omtrent dagens kurs i ren cash i selskapet. SmÄtt utrolig nÄr man tenker pÄ at de har validert teknologien gjennom en big pharma avtale.
Men nÄr oppvÄkningen kommer sÄ blir den rÄtÞff og det kan bli vanskelig for mange Ä bli med sÄ hÞyt som den fortjener. Det er mange prosenter Ä hente her, men nÄr?
Ser ut som aktÞren(e) som har holdt ned nede begynner Ä gÄ tom for aksjer.
Noen som kjenner til om det har vĂŠrt stĂžrre endringer i aksjonĂŠrlisten?
Jeg vet jo alt om hvordan det fungerer i âtom for aksjerâ greia. Men i fĂžlge volumbarene i Nordnet sĂ„ skiftet hele aksje massen hender 2 ganger de 3 -4 fĂžrstte dagene etter Spark avtalen kom.
Det er 19,8 mill aksjer i selskapet og det ble omsatt
- 17 mill aksjer 12 okt
- 16 mill aksjer 13 okt
- 4,3 mill aksjer 14 okt
SÄ noen aktÞrer skulle ikke ha trengt Ä bruke et halvt Är pÄ Ä bli kvitt aksjene sine.
Men ellers har jeg ikke sett mye pÄ eier lister.
Hadde sikkert ikke kjent noen av dem heller 
Det ble vel bare mye trading og shorting antagelig og nÄ har volumet tÞrka sÄ fÊlt at de ser seg om etter andre aksjer Ä Þdelegge.
Men den negative trenden er jo brutt ogsÄ da! HÄper det er riktig sett
Av det jeg har sÄ er listene sist oppdatert 31 dec. sÄ ikke en aksjonÊrliste det er sÊrlig Ä fÞlge med pÄ.
Er vel sÄpass obskur at det skader vel ikke med en teaser pÄ hva borsdata.se kan by pÄ.
Vet det finnes de som fÞlger med pÄ avanza med antall eiere. finner det pÄ discord, sier meg ingenting (vet ikke hvor mange som har eid, nÄr dem har eid og fÞler ikke at det er viktig) er som nordnet kunder og kvitter seg med aksjene bare dem gjÞr rÞrer litt kjapt pÄ seg
Aasen er jo kjent fra PCIB og er stadig her pÄ forumet ogsÄ Hyggelig at han er med
Nyhetsbrev som gir hÄp om potensial i fremtiden:
CombiGene strÀvar efter en bredare pipeline - BioStock.
Utdrag:
För CombiGenes del verkar dock resan bara ha startat. I en kommentar till BioStock sÀger vd Jan Nilsson:
âJag förstĂ„r de som har sĂ„lt aktier lĂ€ngs vĂ€gen, det finns mĂ„nga privatekonomiska orsaker till att hĂ€mta hem en vinst. Ur mitt perspektiv befinner sig CombiGene fortfarande i början av en potentiellt mycket spĂ€nnande utveckling. Vi förvĂ€ntar oss att branschen som vi ingĂ„r i kommer utveckla mĂ„nga nya genterapiprodukter under de nĂ€rmaste Ă„ren. Vad gĂ€ller CG01 sĂ„ har Spark Therapeutics ett omfattande kunnande, finansiella muskler och en mĂ„lsĂ€ttning att ta kandidaten hela vĂ€gen till marknad. Under tiden ser jag framför mig ett scenario dĂ€r vi förhoppningsvis kan kommunicera nyheter som kan intressera marknaden och som inte nödvĂ€ndigtvis Ă€r kopplat till huvudkandidaten CG01. Genterapier har en enorm inneboende potential och mitt Ă€r mĂ„l att CombiGene skall vara en av de frĂ€msta drivande krafterna nĂ€r denna potential omsĂ€tts till mĂ€tbar patientnytta och nya innovativa behandlingsmetoderâ.
InnehÄllet i BioStocks nyheter och analyser Àr oberoende men BioStocks verksamhet Àr i viss mÄn finansierad av bolag i branschen. Detta inlÀgg avser ett bolag som BioStock erhÄllit finansiering frÄn.
Snart er hele oppgangen knyttet til dealen med Spark utradert.
Ser ikke ut som partneravtaler er drÞmmen for smÄ bioteks
Tenkte ogsÄ pÄ dette med milestones. Vet vi noe om hva som utlÞses nÄr?