Rodolphe Clerval, Coave Therapeutics

On a mis­sion to treat pa­tients not cov­ered by Lux­tur­na, Ho­ra­ma re­fu­els with a Se­ries B ex­pan­sion and a new name

About four years af­ter clos­ing its Se­ries B round the first time, Ho­ra­ma is adding an­oth­er $25.1 mil­lion to its cof­fers for its work on a next-gen gene ther­a­py that could treat re­tini­tis pig­men­tosa pa­tients not cov­ered by Roche’s Lux­tur­na — and it’s sport­ing a new name to boot.

The French biotech, now called Coave Ther­a­peu­tics, has ex­pand­ed its Se­ries B round, bring­ing its to­tal raise to $39 mil­lion, CEO Rodolphe Cler­val an­nounced ear­ly Wednes­day morn­ing. The lat­est round was led by Ser­o­ba Life Sci­ences, with a hand from Théa Open In­no­va­tion, eu­reKARE, Fund+, Omnes Cap­i­tal, V-Bio Ven­tures, Kur­ma Part­ners, Id­in­vest, GO Cap­i­tal, and Sham In­no­va­tion San­té/Turenne.

Why the name change? Coave stands for “con­ju­gat­ed AAV vec­tors,” which is ex­act­ly what the com­pa­ny in­tends to make, Cler­val told End­points News. 

Ho­ra­ma launched in 2015 based on work from the Uni­ver­si­ty of Nantes in the west of France. The com­pa­ny’s core tech­nol­o­gy — dubbed the AAV Lig­and Con­ju­gate, or ALI­GATER plat­form — is de­signed to en­hance the de­liv­ery and ef­fi­cien­cy of AAV vec­tors by bind­ing AAVs to lig­ands, sim­i­lar to the way sci­en­tists would link an an­ti­body to a pay­load to form an an­ti­body-drug con­ju­gate.

“It’s a tech­nol­o­gy that is in­spired from, for in­stance, an­ti­body drug con­ju­gates, where peo­ple are tak­ing chem­i­cal com­po­nents and us­ing chem­istry to bind them on pro­teins,” Cler­val said. “This is ex­act­ly what we are do­ing, but we are tak­ing the en­tire AAV… and we are de­vel­op­ing spe­cif­ic lig­ands and we are bind­ing co­va­lent­ly those lig­ands on­to the sur­face of the cap­sid.”

By do­ing so, Cler­val says the sci­en­tists are able to make three key changes: im­prov­ing the biodis­tri­b­u­tion of the cap­sid (how it’s able to trav­el through the tis­sue of an or­gan), im­prov­ing trans­duc­tion ef­fi­cien­cy (its abil­i­ty to trans­fer the pay­load in­side the cell), and pre­vent­ing the cap­sid from be­ing neu­tral­ized by the body’s im­mune re­sponse.

The com­pa­ny’s lead pro­gram, CTx-PDE6b, is cur­rent­ly in a Phase I/II tri­al for PDE6b in­her­it­ed reti­nal dy­s­tro­phies, in­clud­ing re­tini­tis pig­men­tosa. Lux­tur­na — which was de­vel­oped by Spark Ther­a­peu­tics be­fore it was bought out by Roche in 2019 — is cur­rent­ly ap­proved to treat mu­ta­tions in the RPE65 gene.

Re­tini­tis pig­men­tosa caused by PDE6b mu­ta­tions of­ten leads to blind­ness by midlife, and is char­ac­ter­ized by the pro­gres­sive loss of pho­tore­cep­tors.

“Those pa­tients ba­si­cal­ly will lose some pho­tore­cep­tors, lose their vi­sion, start­ing by the pe­riph­ery vi­sion to a shrink­age of their vi­su­al field,” Cler­val said.

Last Au­gust, Ocu­gen re­ceived or­phan drug des­ig­na­tion for its own gene ther­a­py, OCU400, to treat PDE6b-as­so­ci­at­ed reti­nal dis­eases. That can­di­date’s cur­rent­ly in IND-en­abling stages, ac­cord­ing to the com­pa­ny’s web­site.

To date, 15 of 17 pa­tients are en­rolled in Coave’s Phase I/II tri­al, and Cler­val ex­pects to read out da­ta in 2022. In ad­di­tion to ad­vanc­ing that pro­gram to­ward piv­otal tri­als, the 12-per­son team al­so has mul­ti­ple pre­clin­i­cal pro­grams in the works for rare CNS and oc­u­lar dis­eases, in­clud­ing  CTx-GBA1 for Parkin­son’s dis­ease and Gauch­er Dis­ease and CTx-AB­CA4 for Star­gardt’s dis­ease.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Radek Spisek, Sotio CEO (Cellestia)

A qui­et Czech biotech bags $315M to dri­ve its blos­som­ing can­cer pipeline through the clin­ic

In the rather insular world of biotech, most innovation inevitably comes from a cluster of R&D hubs — Cambridge, San Francisco, etc. But sometimes success stories sprout from rocky soil, which is most certainly the case with Prague-based Sotio Biotech and its suddenly jam-packed pipeline of cancer drugs.

After years in quiet development, Sotio now has $315 million in new funds to play with from parent company PPF Group, an investment group founded in the Czech Republic, as the biotech looks to advance its growing pipeline through early- and mid-stage trials.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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