AI up­start grabs $40M launch round; Sanofi backs a gene ther­a­py play­er; Ax­some hits a snag at the FDA

Boston AI-fo­cused biotech Deep­Cure closed a $40 mil­lion Se­ries A, ac­cord­ing to a com­pa­ny an­nounce­ment this morn­ing — adding more than five times the amount of mon­ey the com­pa­ny had be­fore: from $7 mil­lion raised since the com­pa­ny’s found­ing in 2018 to $47 mil­lion to­tal.

Their goal? To have an R&D process that has drugs ful­ly de­signed, syn­the­sized and test­ed by AI, ac­cord­ing to a com­pa­ny state­ment.

The round was led by Morn­ing­side Ven­tures — elic­it­ing par­tic­i­pa­tion from pre­vi­ous in­vestors such as TLV Part­ners, Sapir Ven­ture Part­ners, and Benon Group.

Kfir Schreiber

“We are de­light­ed to wel­come a top-tier syn­di­cate of lead­ing health­care in­vestors led by Morn­ing­side Ven­tures that sup­port our vi­sion of de­vel­op­ing an end-to-end drug dis­cov­ery pipeline de­signed, op­ti­mized, syn­the­sized, and an­a­lyzed by AI,” said Deep­Cure CEO and co-founder Kfir Schreiber.

The mon­ey will be used to ex­pand Deep­Cure’s pipeline with five ad­di­tion­al on­col­o­gy pro­grams and de­vel­op an au­to­mat­ed ro­bot­ic wet lab to fur­ther its goal of AI-in­volved R&D. The fund­ing will al­so be used to hire more drug dis­cov­ery sci­en­tists and tech­nol­o­gists — dou­ble what the com­pa­ny cur­rent­ly has in tan­dem with Deep­Cure’s planned glob­al ex­pan­sion with the launch of two new sites in Greece and Is­rael.

“This in­vest­ment po­si­tions us to ad­vance our first nov­el small mol­e­cule com­pound to file for IND, ad­vance our pipeline and re­al­ize our vi­sion of mak­ing a sig­nif­i­cant im­pact for pa­tients,” added Deep­Cure’s CSO and oth­er co-founder Joseph Ja­cob­son. — Paul Schloess­er

UK biotech Gy­ro­scope to re­ceive eq­ui­ty in­vest­ment from Sanofi six months af­ter abrupt­ly post­pon­ing IPO

Gy­ro­scope Ther­a­peu­tics an­nounced that Sanofi has com­mit­ted up to $60 mil­lion in an eq­ui­ty in­vest­ment.

The eye dis­ease-fo­cused gene ther­a­py biotech said this morn­ing in a state­ment that the French Big Phar­ma will in­vest $40 mil­lion as an ini­tial eq­ui­ty in­vest­ment in­to Gy­ro­scope ini­tial­ly, at a pre­mi­um to Gy­ro­scope’s pre­vi­ous $148 mil­lion Se­ries C fi­nanc­ing. The ad­di­tion­al $20 mil­lion will be in­vest­ed based on a fu­ture qual­i­fy­ing in­vest­ment round — and sub­ject to cer­tain clos­ing con­di­tions.

The biotech star­tled in­vestors and an­a­lysts af­ter post­pon­ing its IPO in May, cit­ing mar­ket con­di­tions.

Khurem Fa­rooq

“We are thrilled to wel­come Sanofi on­board as an in­vestor in Gy­ro­scope,” said Gy­ro­scope CEO Khurem Fa­rooq. “We be­lieve our lead in­ves­ti­ga­tion­al gene ther­a­py, GT005, has the po­ten­tial to be the first gene ther­a­py for ge­o­graph­ic at­ro­phy.”

Ge­o­graph­ic at­ro­phy is chron­ic pro­gres­sive de­gen­er­a­tion of the back of the eye and can be seen as part of late-stage age-re­lat­ed mac­u­lar de­gen­er­a­tion, or AMD.

An un­named Sanofi R&D ex­ec­u­tive will join Gy­ro­scope’s clin­i­cal ad­vi­so­ry board to ad­vise on mat­ters re­lat­ed to the de­vel­op­ment of GT005 for ge­o­graph­ic at­ro­phy. Ad­di­tion­al­ly, Gy­ro­scope has grant­ed Sanofi ex­clu­sive right of first re­fusal on cer­tain po­ten­tial fu­ture trans­ac­tions for GT005 in se­lect mar­kets.

Gy­ro­scope plans to use pro­ceeds from this in­vest­ment to sup­port fund­ing of on­go­ing clin­i­cal tri­als of GT005, as the drug has been grant­ed fast track des­ig­na­tion by the FDA. — Paul Schloess­er

Se­lec­ta shows promis­ing re­sults in gene ther­a­py de­liv­ery and re-dos­ing pa­tients in Phase I tri­al

Gene ther­a­py sci­en­tists see a ben­e­fit in re­dos­ing pa­tients af­ter the first dose of a ther­a­py. But the is­sue of an im­mune re­sponse against ade­no-as­so­ci­at­ed virus­es (AAVs) has pushed com­pa­nies to try and get around it.

Se­lec­ta Bio­sciences thought it found a way to tamp down the body’s im­mune re­sponse back in Oc­to­ber — and Take­da doled out more than $1 bil­lion to get in on the ac­tion.

Now to­day, the biotech re­vealed re­sults from a Phase I tri­al look­ing in­to the body’s im­mune re­sponse to their Imm­TOR plat­form — and the re­sults may be promis­ing.

With AskBio, Se­lec­ta un­veiled top-line re­sults this morn­ing from a Phase I ran­dom­ized, place­bo con­trolled, dou­ble blind, dose-es­ca­la­tion study. The goal is to eval­u­ate Se­lec­ta’s Imm­TOR plat­form in mit­i­gat­ing the for­ma­tion of neu­tral­iz­ing an­ti­bod­ies against an AAV8 cap­sid — which is used in gene ther­a­pies.

At day 30, in sub­jects who were ad­min­is­tered a sin­gle 0.3 mg/kg dose of Imm­TOR, Se­lec­ta ob­served a me­di­an an­ti-AAV8 neu­tral­iz­ing an­ti­body titer of 1:5 — 250 times low­er than that ob­served in sub­jects dosed with AAV8 cap­sid alone, ac­cord­ing to a Se­lec­ta state­ment.

“We look for­ward to lever­ag­ing these ex­cit­ing and nov­el find­ings in the clin­ic across our whol­ly owned gene ther­a­py pipeline and along­side our world class gene ther­a­py part­ners to achieve our goal of im­prov­ing the lives of those liv­ing with mono­genic dis­eases,” said Se­lec­ta pres­i­dent and CEO Carsten Brunn.

In the Phase I study, re­searchers eval­u­at­ed the ad­min­is­tra­tion of a sin­gle IV dose of a AAV8 emp­ty cap­sid con­tain­ing no DNA with and with­out a sin­gle dose of Imm­TOR in 23 sub­jects. — Paul Schloess­er

Ax­some de­pres­sion drug hits a CMC hur­dle at the FDA

Ax­some’s de­pres­sion drug AXS-05 has hit a snag at the FDA.

The biotech $AXSM re­port­ed Mon­day morn­ing that reg­u­la­tors no­ti­fied the com­pa­ny that they will not hit the PDU­FA tar­get ac­tion date — set all the way back on Aug. 22 — af­ter cit­ing CMC de­fi­cien­cies.

Ac­cord­ing to their state­ment:

The Com­pa­ny was re­cent­ly in­formed of two de­fi­cien­cies re­lat­ed to an­a­lyt­i­cal meth­ods in the Chem­istry, Man­u­fac­tur­ing, and Con­trols (CMC) sec­tion of the NDA, which must be ad­dressed pri­or to the FDA tak­ing ac­tion on the NDA. The Com­pa­ny be­lieves these de­fi­cien­cies are ad­dress­able and is con­firm­ing the de­tails of the re­quest with the FDA.

— John Car­roll

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

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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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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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.

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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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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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.