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.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, Congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Bio­gen, Ei­sai are push­ing for an­oth­er ac­cel­er­at­ed Alzheimer's OK — this time for BAN2401

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

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Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

UP­DAT­ED: Sanofi buys mR­NA play­er Trans­late Bio for $3.2B. And the price fits a pop­u­lar range for biotech M&A

Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio $TBIO a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines, Sanofi closed the deal with a buyout early Tuesday, spending $38 a share in a $3.2 billion buyout.

Translate’s stock $TBIO soared after the market closed Monday when Reuters reported the first word of the acquisition just hours ahead of the formal announcement. The wire service, though, didn’t have a price to report in its scoop, and investors chased the stock up 78% in the wild ride that followed. Once the price was announced, gains shriveled to 29% ahead of the bell.

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