Ad­verum Bio of­fers a late no­ti­fi­ca­tion about FDA hold on lead gene ther­a­py for wet AMD

The FDA has some ques­tions for Ad­verum Biotech­nolo­gies about the CMC work re­lat­ed to their lead gene ther­a­py for wet, age-re­lat­ed mac­u­lar de­gen­er­a­tion, and reg­u­la­tors have put a clin­i­cal hold on their clin­i­cal de­vel­op­ment pro­gram un­til they get some an­swers.

The Men­lo Park, CA-based biotech $AD­VM re­port­ed Mon­day as the mar­ket opened that the agency had placed a hold on their drug “in ear­ly April” and were now re­view­ing a re­sponse to their query, sub­mit­ted last week. This was the first chance that in­vestors got to hear about this news from the pub­lic com­pa­ny, which re­served word about the FDA ac­tion un­til af­ter they not­ed they had a green light from the in­de­pen­dent da­ta mon­i­tor­ing com­mit­tee for the re­cruit­ment of their sec­ond co­hort of pa­tients for AD­VM-022 — which they say was the chrono­log­i­cal or­der of events.

This is their on­ly clin­i­cal-stage pro­gram. But the news seems to be sit­ting well with its share­hold­ers. The stock is up 7% in mid-af­ter­noon trad­ing.

I asked in a fol­low-up query to the com­pa­ny why they didn’t file any­thing with the SEC on the clin­i­cal hold when it oc­curred, or with­in a few days of the event. I haven’t heard back.

Leone Pat­ter­son

The treat­ment in­volves an afliber­cept cod­ing se­quence which is de­liv­ered to the eye via a com­pa­ny-owned vec­tor. Afliber­cept is a drug de­liv­ered to the eye via in­jec­tion, mak­ing any re­duc­tion in treat­ments some­thing most pa­tients would wel­come.

Ed­ward Nash at Sun­Trust says he’s been in touch with the ex­ec­u­tive crew at the biotech and not­ed that un­der the best case sce­nario the pro­gram would be de­layed a month. That, ob­vi­ous­ly, could stretch out if the FDA takes more time to suss things out.

Ad­verum was formed out of Avalanche Biotech­nolo­gies’ ac­qui­si­tion of Paris-based An­na­pur­na and their merg­er in­to a new gene ther­a­py com­pa­ny fol­low­ing some trou­ble at Avalanche. Late last year the biotech aban­doned their then lead, AD­VM-043, af­ter re­searchers de­ter­mined it was hav­ing an in­suf­fi­cient ef­fect on A1AT de­fi­cien­cy.

“We are work­ing with the FDA to re­solve this mat­ter as quick­ly as pos­si­ble,” said Ad­verum CEO Leone Pat­ter­son in a state­ment. “We are deeply com­mit­ted to the de­vel­op­ment of our nov­el gene ther­a­py AD­VM-022 for pa­tients with wet AMD. In the OP­TIC tri­al, the DMC re­viewed the safe­ty da­ta and unan­i­mous­ly agreed that we could pro­ceed to dos­ing the sec­ond co­hort. No pa­tient has ex­pe­ri­enced an SAE, with a fol­low-up pe­ri­od of up to five months. We look for­ward to shar­ing 24-week pri­ma­ry and sec­ondary out­comes from the first co­hort of pa­tients at a sci­en­tif­ic meet­ing in the sec­ond half of this year.”


Im­age: Shut­ter­stock

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

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Endpoints News writer Nicole DeFeudis has posted a snapshot of all the companies, universities and hospital-based groups now racing through the clinic, ranking them according to their place in the pipeline as well as the latest remarks available on timelines. And we’ll keep this lineup updated right through the end of the year, as the checkered flags start to fall, possibly as early as October.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

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Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

UP­DAT­ED: Bris­tol My­ers Squibb com­mits $300 mil­lion to com­bat racial dis­par­i­ties, but de­clines to re­lease own de­mo­graph­ic da­ta

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

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VBL an­nounces pos­i­tive PhI­II da­ta for ovar­i­an can­cer drug; Gene­Cen­tric part­ners with Janssen on blad­der can­cer

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