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

Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,100+ biopharma pros reading Endpoints daily — and it's free.

Ob­sE­va makes case for best-in-class hor­mone sup­pres­sive ther­a­py in pos­i­tive uter­ine fi­broid study

About a month after the Swiss biotech disclosed a failed late-stage study in its IVF program, ObsEva on Monday unveiled positive pivotal data on its experimental treatment for heavy menstrual bleeding triggered by uterine fibroids.

ObsEva in-licensed the drug, linzagolix, from Japan’s Kissei Pharmaceutical in 2015. Two doses of the drug (100 mg and 200 mg) were tested against a placebo in the 535-patient Phase III study, dubbed PRIMROSE 2, in patients who were both on and off hormonal add-back therapy (ABT).

Jake Van Naarden, Josh Bilenker, Nisha Nanda (Credit: Loxo, Aisling Capital)

Josh Bilenker and his Loxo crew are tak­ing the reins on on­col­o­gy R&D at Eli Lil­ly, culling the weak and map­ping a new path

Josh Bilenker, Jake Van Naarden and Nisha Nanda came out of Eli Lilly’s $8 billion Loxo Oncology buyout with a bundle of cash and plenty of choices on what they could do next. Start a new company, go public. Live on the beach in 5-star luxury. Contemplate the stars — in their own observatory.

So what are they doing?

They formed a new executive team that is taking over the management of Eli Lilly’s hundreds-strong oncology R&D group — essentially using Loxo as a base for a bold new experiment in Big Pharma R&D in an attempt to create a true biotech environment with the deep pockets of a top-15 industry player. They’ve recruited David Hyman from Memorial Sloan Kettering to join the team as chief medical officer. And the mandate includes culling out the oncology pipeline, highlighting their star prospects and going after new programs wherever they can find the best prospects.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,100+ biopharma pros reading Endpoints daily — and it's free.

Samit Hirawat. Bristol-Myers Squibb

Bris­tol-My­ers is mak­ing a bee-line to the FDA with pos­i­tive liso-cel da­ta — but is it too late in the CAR-T game?

Bristol-Myers Squibb came to ASH this past weekend with a variety of messages on the new cancer drugs they had acquired in the big Celgene buyout, including liso-cel, the lead CAR-T program picked up in the $9 billion Juno acquisition. And one of the most important was that they had the pivotal efficacy and safety data needed to snag an approval from the FDA next year, with the BLA on track for a filing this month.

J&J team shows off 'break­through' BC­MA CAR-T da­ta, and that could cause a big headache at blue­bird and Bris­tol-My­ers

Just hours after J&J’s oncology team bragged about scoring a breakthrough therapy designation for their BCMA CAR-T drug, they pulled the wraps off of the multiple myeloma data for JNJ-4528 that impressed the FDA. And it’s easy to see why they may well be on a short path to a landmark approval — which may well be making the rival team at bluebird/Bristol-Myers more than a little nervous.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,100+ biopharma pros reading Endpoints daily — and it's free.

J&J's Mathai Mammen at an Endpoints News event in Boston, June 2018 (Photo: Rob Tannenbaum for Endpoints News)

J&J fronts $750M cash to grab a failed can­cer drug that’s been re­pur­posed as a pow­er­ful an­ti-in­flam­ma­to­ry

J&J has stepped up with one of its blockbuster drug buys, agreeing to pay Austin-based XBiotech $XBIT $750 million in cash and up to $600 million more in milestones for their late stage-ready anti-inflammatory drug bermekimab — which some longtime biotech observers may recognize as a failed cancer therapy with a disaster-prone past.

The drug targets the IL-1a pathway. J&J $JNJ R&D chief Mathai Mammen is cutting a check for a drug that has produced positive mid-stage data in patients suffering from a skin condition called hidradenitis suppurativa with another mid-stage program underway for atopic dermatitis.

That puts J&J in charge of a drug on the threshold of pivotal — though pricey — R&D work for a broad patient group with other related fields to explore. And it’s a very busy development arena.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,100+ biopharma pros reading Endpoints daily — and it's free.

Sangamo CEO Sandy Macrae

Pa­tient #9 has been a con­cern, but Sang­amo and Pfiz­er are bull­ish about win­ning the marathon he­mo­phil­ia A gene ther­a­py race

Patient number 9 has given Sangamo and its partners at Pfizer some heart palpitations in their high profile hemophilia A gene therapy program.

After watching his Factor VIII level rise following treatment like the rest, the crucial efficacy gauge they track saw a sudden and significant plunge. At week 13, the FVIII level had dropped below normal. Then it began to rise again.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,100+ biopharma pros reading Endpoints daily — and it's free.

One of the 100 limited-edition framed certificates we're sending to the first group of companies that signed up for an Enterprise plan

Un­lock the full End­points ex­pe­ri­ence for your com­pa­ny — and sup­port our mis­sion of in­de­pen­dent bio­phar­ma re­port­ing

I want to give readers a quick update on the most important part of our business model — premium subscriptions. We have some crucial financial goals we hope to achieve by the end of the year, and the team here in Lawrence is ready to ship some swag to kick off this limited December promotion.

We offer two premium plans — Enterprise for companies ($1,000/year, unlimited people), and Insider for individuals ($200/year). This month of December will be the last chance to enroll at the original rates — which have remained flat since we launched them in 2017.

One of Wall Street’s most high-pro­file hedge funds push­es Alex­ion's CEO to the auc­tion block — and he's not budg­ing

Fresh off buying Barnes & Noble and prodding AT&T with some heavy-handed criticism after picking up a $3.2 billion stake in the company, the activist — and supremely high profile — hedge fund Elliott Management has stepped up with some M&A advice for Alexion’s management team.
And the execs on the team $ALXN are giving them a polite — but very firm — stiff arm Friday morning.
In a release out early Friday, the big biotech said that the Elliott team had been in touch to encourage them to sell the company. But that’s not on the agenda.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,100+ biopharma pros reading Endpoints daily — and it's free.